Myovant Sciences and Pfizer Receive FDA Approval for MYFEMBREE®, the First Once-Daily Treatment for Heavy Menstrual Bleeding Associated With Uterine Fibroids

BASEL, Switzerland and NEW YORK, May 26, 2021 (GLOBE NEWSWIRE) — Myovant Sciences (NYSE: MYOV) and Pfizer Inc. (NYSE: PFE) today announced that the U.S. Food and Drug Administration (FDA) has approved MYFEMBREE® (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg), the first once-daily treatment for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women, with a treatment duration of up to 24 months. The approval is supported by efficacy and safety data from the Phase 3 LIBERTY 1 and LIBERTY 2 studies, which were published in the New England Journal of Medicine. Under the terms of their previously announced collaboration, Myovant and Pfizer will jointly commercialize MYFEMBREE in the U.S. MYFEMBREE is expected to be available in June 2021.

Media Snippet accompanying this announcement is available by clicking on the image or link below:

“With MYFEMBREE, we can offer women with uterine fibroids a non-invasive treatment that provides clinically meaningful symptom relief for heavy menstrual bleeding with one pill, once-a-day,” said Ayman Al-Hendy, M.D., Ph.D., Professor of Obstetrics and Gynecology, University of Chicago, and LIBERTY Program Steering Committee Member. “The FDA approval of MYFEMBREE represents a significant milestone in expanding treatment options for uterine fibroids, a chronic and debilitating disease for many women in the U.S.”

“Uterine fibroids affect millions of women in the U.S. and account for over 250,000 hysterectomies each year, with heavy menstrual bleeding being one of the most bothersome symptoms,” said David Marek, Chief Executive Officer of Myovant Sciences, Inc. “The approval of MYFEMBREE represents the second FDA product approval for Myovant in less than one year. This is an important step forward as we seek to redefine care for women and for men, not only through new medicines but through continued collaboration with the community.”

“MYFEMBREE’s approval is a testament to the shared commitment between Myovant and Pfizer to support women living with uterine fibroids,” said Nick Lagunowich, Global President, Internal Medicine at Pfizer. “We are excited to offer this new treatment option which will help provide much needed symptom relief with the convenience of an oral, once-daily tablet.”

The Phase 3 LIBERTY studies each met the primary endpoint, with 72.1% and 71.2% of women in the MYFEMBREE groups achieving the responder criteria compared with 16.8% and 14.7% of women in the placebo groups at Week 24, respectively (both p < 0.0001). A response was defined as a menstrual blood loss volume of less than 80 mL and a 50% or greater reduction from baseline in menstrual blood loss volume during the last 35 days of treatment measured using the alkaline hematin method. Women receiving MYFEMBREE experienced reductions of 82.0% and 84.3% in menstrual blood loss from baselines, respectively (both p < 0.0001 compared to placebo). Adverse reactions occurring in at least 3% of women treated with MYFEMBREE and at a greater incidence than placebo were hot flush, abnormal uterine bleeding, alopecia, and decreased libido. There were no pregnancies reported in the MYFEMBREE groups in either study.

Myovant and Pfizer are committed to supporting women in the U.S. who are prescribed MYFEMBREE throughout their treatment journeys. The MYFEMBREE Support Program provides services, including insurance benefits checks, prior authorization support, co-pay support for commercially insured patients, and patient assistance for qualifying uninsured patients. Program terms and conditions apply. For more information and additional resources, please contact 833-MYFEMBREE (833-693-3627), 8 a.m.–8 p.m. Eastern Time, Monday–Friday.

Myovant Conference Call and Webcast
Myovant will hold a conference call on Friday, May 28, 2021 at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time to discuss the FDA approval of MYFEMBREE®. Investors and the general public may access a live webcast of the call by visiting the investor relations page of Myovant’s website at investors.myovant.com. Investors and analysts may also participate in the conference call by dialing 1-800-532-3746 in the U.S. or +1-470-495-9166 from outside the U.S. A replay of the webcast will be archived on Myovant’s investor relations website.

About Uterine Fibroids
Uterine fibroids are noncancerous tumors that develop in or on the muscular walls of the uterus and are among the most common reproductive tract tumors in women. In addition to an individual’s genetic predisposition, estrogens are well known to play an important role in the regulation of fibroid growth.

Although uterine fibroids are benign tumors, they can cause debilitating symptoms such as heavy menstrual bleeding (frequently resulting in anemia and fatigue), pain (including painful periods, abdominal pain, painful intercourse, backache), increased abdominal girth and bloating, urinary frequency or retention, constipation, pregnancy loss, and, in some cases, infertility. These symptoms can also lead to loss of productivity at work, limitations in normal activities of daily living, and social embarrassment.

An estimated five million women in the U.S. suffer from symptoms of uterine fibroids, and an estimated three million women are inadequately treated by current medical therapy and require further treatment.

About MYFEMBREE®
MYFEMBREE (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) is the first once-daily treatment for heavy menstrual bleeding associated with uterine fibroids in premenopausal women approved by the U.S. Food and Drug Administration, with a treatment duration of up to 24 months. MYFEMBREE contains relugolix, which reduces the amount of estrogen (and other hormones) produced by ovaries, estradiol (an estrogen) which may reduce the risk of bone loss, and norethindrone acetate (a progestin) which is necessary when women with a uterus (womb) take estrogen.

For full prescribing information including Boxed Warning and patient information, please click here.

Indications and Usage

MYFEMBREE is indicated for the management of heavy menstrual bleeding associated with uterine leiomyomas (fibroids) in premenopausal women. Limitations of Use: Use of MYFEMBREE should be limited to 24 months due to the risk of continued bone loss which may not be reversible.

Important Safety Information

BOXED WARNING: THROMBOEMBOLIC DISORDERS AND VASCULAR EVENTS 

Estrogen and progestin combination products, including MYFEMBREE, increase the risk of thrombotic or thromboembolic disorders including pulmonary embolism, deep vein thrombosis, stroke and myocardial infarction, especially in women at increased risk for these events.

MYFEMBREE is contraindicated in women with current or a history of thrombotic or thromboembolic disorders and in women at increased risk for these events, including women over 35 years of age who smoke or women with uncontrolled hypertension.

CONTRAINDICATIONS

MYFEMBREE is contraindicated in women with any of the following: high risk of arterial, venous thrombotic, or thromboembolic disorder; pregnancy; known osteoporosis; current or history of breast cancer or other hormone-sensitive malignancies; known hepatic impairment or disease; undiagnosed abnormal uterine bleeding; known hypersensitivity to components of MYFEMBREE.

WARNINGS AND PRECAUTIONS

Thromboembolic Disorders: Discontinue immediately if an arterial or venous thrombotic, cardiovascular, or cerebrovascular event occurs or is suspected. Discontinue at least 4 to 6 weeks before surgery associated with an increased risk of thromboembolism, or during periods of prolonged immobilization, if feasible. Discontinue immediately if there is sudden unexplained partial or complete loss of vision, proptosis, diplopia, papilledema, or retinal vascular lesions and evaluate for retinal vein thrombosis as these have been reported with estrogens and progestins.

Bone Loss: MYFEMBREE may cause a decrease in bone mineral density (BMD) in some patients, which may be greater with increasing duration of use and may not be completely reversible after stopping treatment. Consider the benefits and risks in patients with a history of low trauma fracture or risk factors for osteoporosis or bone loss, including medications that may decrease BMD. Assessment of BMD by dual-energy X-ray absorptiometry (DXA) is recommended at baseline and periodically thereafter. Consider discontinuing MYFEMBREE if the risk of bone loss exceeds the potential benefit.

Hormone-Sensitive Malignancies: Discontinue MYFEMBREE if a hormone-sensitive malignancy is diagnosed. Surveillance measures in accordance with standard of care, such as breast examinations and mammography are recommended. Use of estrogen alone or estrogen plus progestin has resulted in abnormal mammograms requiring further evaluation.

Depression, Mood Disorders, and Suicidal Ideation: Promptly evaluate patients with mood changes and depressive symptoms including shortly after initiating treatment, to determine whether the risks of continued therapy outweigh the benefits. Patients with new or worsening depression, anxiety, or other mood changes should be referred to a mental health professional, as appropriate. Advise patients to seek immediate medical attention for suicidal ideation and behavior and reevaluate the benefits and risks of continuing MYFEMBREE.

Hepatic Impairment and Transaminase Elevations: Steroid hormones may be poorly metabolized in these patients. Instruct women to promptly seek medical attention for symptoms or signs that may reflect liver injury, such as jaundice or right upper abdominal pain. Acute liver test abnormalities may necessitate the discontinuation of MYFEMBREE use until the liver tests return to normal and MYFEMBREE causation has been excluded.

Gallbladder Disease or History of Cholestatic Jaundice: Discontinue MYFEMBREE if signs or symptoms of gallbladder disease or jaundice occur. For women with a history of cholestatic jaundice associated with past estrogen use or with pregnancy, assess the risk-benefit of continuing therapy. Studies among estrogen users suggest a small increased relative risk of developing gallbladder disease.

Elevated Blood Pressure: For women with well-controlled hypertension, monitor blood pressure and stop MYFEMBREE if blood pressure rises significantly.

Change in Menstrual Bleeding Pattern and Reduced Ability to Recognize Pregnancy: Advise women to use non-hormonal contraception during treatment and for one week after discontinuing MYFEMBREE. Avoid concomitant use of hormonal contraceptives. MYFEMBREE may delay the ability to recognize pregnancy because it alters menstrual bleeding. Perform testing if pregnancy is suspected and discontinue MYFEMBREE if pregnancy is confirmed.

Risk of Early Pregnancy Loss: MYFEMBREE can cause early pregnancy loss. Exclude pregnancy before initiating and advise women to use effective non-hormonal contraception.

Uterine Fibroid Prolapse or Expulsion: Advise women with known or suspected submucosal uterine fibroids about the possibility of uterine fibroid prolapse or expulsion and instruct them to contact their physician if severe bleeding and/or cramping occurs.

Alopecia: Alopecia, hair loss, and hair thinning were reported in phase 3 trials with MYFEMBREE. Consider discontinuing MYFEMBREE if hair loss becomes a concern. Whether the hair loss is reversible is unknown.

Effects on Carbohydrate and Lipid Metabolism: More frequent monitoring in MYFEMBREE-treated women with prediabetes and diabetes may be necessary. MYFEMBREE may decrease glucose tolerance and result in increased blood glucose concentrations. Monitor lipid levels and consider discontinuing if hypercholesterolemia or hypertriglyceridemia worsensIn women with pre-existing hypertriglyceridemia, estrogen therapy may be associated with elevations in triglycerides levels leading to pancreatitis. Use of MYFEMBREE is associated with increases in total cholesterol and LDL-C.

Effect on Other Laboratory Results: Patients with hypothyroidism and hypoadrenalism may require higher doses of thyroid hormone or cortisol replacement therapy. Use of estrogen and progestin combinations may raise serum concentrations of binding proteins (e.g., thyroid-binding globulin, corticosteroid-binding globulin), which may reduce free thyroid or corticosteroid hormone levels. Use of estrogen and progestin may also affect the levels of sex hormone-binding globulin, and coagulation factors.

Hypersensitivity Reactions: Immediately discontinue MYFEMBREE if a hypersensitivity reaction occurs.

ADVERSE REACTIONS
Most common adverse reactions for MYFEMBREE (incidence ≥3% and greater than placebo) were hot flush/hyperhidrosis/night sweats, abnormal uterine bleeding, alopecia, and decreased libido. These are not all the possible side effects of MYFEMBREE.

DRUG INTERACTIONS
P-gp Inhibitors: Avoid use of MYFEMBREE with oral P-gp inhibitors. If use is unavoidable, take MYFEMBREE first, separate dosing by at least 6 hours, and monitor patients for adverse reactions.

Combined P-gp and Strong CYP3A Inducers: Avoid use of MYFEMBREE with combined P-gp and strong CYP3A inducers.

LACTATION
Advise women not to breastfeed while taking MYFEMBREE.

About Myovant Sciences 
Myovant Sciences aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. Founded in 2016, we have two FDA-approved products. ORGOVYX™ (relugolix) was approved by the U.S. Food and Drug Administration in 2020 as the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist for the treatment of adult patients with advanced prostate cancer, and relugolix is also under regulatory review in Europe for men with advanced prostate cancer. MYFEMBREE® (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) was approved in the U.S. in 2021 as the first once-daily treatment for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women. Relugolix combination tablet (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) is under regulatory review in Europe for women with uterine fibroids, has completed Phase 3 registration-enabling studies for women with endometriosis, and is being assessed for contraceptive efficacy in healthy women ages 18-35 years who are at risk for pregnancy. We are also developing MVT-602, an oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd., is our majority shareholder. For more information, please visit our website at www.myovant.com. Follow @Myovant on Twitter and LinkedIn.

About Pfizer: Breakthroughs That Change Patients’ Lives 
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer NewsLinkedInYouTube and like us on Facebook at Facebook.com/Pfizer.

Myovant Sciences Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In this press release, forward-looking statements include, but are not limited to, all statements reflecting Myovant Sciences’ expectations, including: statements regarding Myovant’s aspiration to redefine care for women and for men; Myovant’s expectations regarding the potential benefits of MYFEMBREE; Myovant’s expectations regarding the potential commercial launch of MYFEMBREE by Myovant and Pfizer in the United States; Myovant’s expectation that MYFEMBREE will become available in June 2021; the plan to offer a MYFEMBREE support program for patients; and the features of such program. 

Myovant Sciences’ forward-looking statements are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties, assumptions and other factors known and unknown that could cause actual results and the timing of certain events to differ materially from future results expressed or implied by the forward-looking statements, including unforeseen circumstances or other disruptions to normal business operations arising from or related to the COVID-19 pandemic. Myovant Sciences cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur and actual results could differ materially from those expressed or implied by these forward-looking statements. Factors that could materially affect Myovant Sciences’ operations and future prospects or which could cause actual results to differ materially from expectations include, but are not limited to the risks and uncertainties listed in Myovant Sciences’ filings with the United States Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in Myovant Sciences’ Annual Report on Form 10-K filed on May 11, 2021, as such risk factors may be amended, supplemented or superseded from time to time. These risks are not exhaustive. New risk factors emerge from time to time, and it is not possible for Myovant Sciences’ management to predict all risk factors, nor can Myovant Sciences assess the impact of all factors on its business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. You should not place undue reliance on the forward-looking statements in this press release, which speak only as of the date hereof, and, except as required by law, Myovant Sciences undertakes no obligation to update these forward-looking statements to reflect events or circumstances after the date of such statements.

Pfizer Disclosure Notice
The information contained in this release is as of May 26, 2021. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about MYFEMBREE® (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) for the treatment of heavy menstrual bleeding associated with uterine fibroids in premenopausal women, and a collaboration between Pfizer and Myovant Sciences to develop and commercialize relugolix in advanced prostate cancer and women’s health, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of MYFEMBREE; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical studies; whether and when applications for MYFEMBREE may be filed in any other jurisdictions for any potential indications for MYFEMBREE; whether and when regulatory authorities in any other jurisdictions may approve any such applications for MYFEMBREE that may be pending or filed, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether MYFEMBREE will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of MYFEMBREE; whether our collaboration with Myovant Sciences will be successful; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

Myovant Sciences Contacts
Ryan Crowe, Investors
+1 (650) 781-9106
investors@myovant.com

Albert Liao, Media
+1 (650) 410-3055
media@myovant.com

Pfizer Contacts
Media Relations
Steve Danehy
+1 (212) 733-1538
PfizerMediaRelations@pfizer.com  

Investor Relations
Chuck Triano
+1 (212) 733-3901
Charles.E.Triano@Pfizer.com

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Sumitovant Biopharma Has a Strong Fourth Quarter with Significant Clinical Development and Corporate Achievements Across its Portfolio of Companies

LONDON and NEW YORK, May 24, 2021 /PRNewswire/ — Sumitovant Biopharma, Inc. today announced that its portfolio of four wholly owned subsidiary companies (UrovantEnzyvantAltavant and Spirovant )  and Myovant Sciences (NYSE: MYOV), a publicly listed company that is majority owned by Sumitovant, achieved significant clinical and corporate milestones in the company’s fourth quarter ending on March 31, 2021. 

“We were delighted to see tremendous strides across the Sumitovant portfolio of companies in the fourth quarter including progress across clinical development, regulatory and commercialization milestones.   Significant corporate accomplishments included Urovant transitioning from publicly traded to a wholly owned subsidiary of Sumitovant Biopharma and the appointment of executives in key roles for two of our subsidiary companies,” said Myrtle Potter, CEO of Sumitovant Biopharma.  “In addition, progress was made in supporting health equity and access and formed collaborations to support educational and clinical initiatives.  This was an exciting quarter and I’m proud of what we’ve accomplished.”

CLINICAL HIGHLIGHTS

Myovant Sciences (NYSE: MYOV), a majority-owned subsidiary
On March 29, the European Medicines Agency (EMA) validated Myovant Science’s Marketing Authorization Application (MAA) for relugolix for the treatment of advanced prostate cancer. The validation of the application confirms the submission is sufficiently complete for the EMA to begin the formal review process. If approved, relugolix would be the first and only oral androgen deprivation therapy for advanced prostate cancer in Europe.

On March 24, Myovant Sciences and its partner, Pfizer, announced positive data from Phase 3 LIBERTY randomized withdrawal study of once-daily relugolix combination therapy in women with uterine fibroids.

On February 17, Myovant Sciences and Pfizer announced publication in the New England Journal of Medicine of Phase 3 LIBERTY studies of once-daily relugolix combination therapy in women with uterine fibroids.

On January 26, Myovant Sciences and its partner, Pfizer, announced positive one-year data from its Phase 3 SPIRIT extension study of once-daily relugolix combination therapy in women with endometriosis 

Urovant Sciences
On February 11, Urovant Sciences announced the continuation of a Phase 2a trial with URO-902, a novel gene therapy product in patients with overactive bladder and urinary incontinence following a positive recommendation from the study’s Data and Safety Monitoring Board. 

Altavant Sciences
On February 18, Altavant Sciences announced initiation of a chemical lung injury program in collaboration with BARDA and NIAID. The in vivo nonclinical proof-of-concept pilot study will evaluate ALTA-2350, a novel inhaled formulation of a recombinant IL-1Ra for the treatment of acute and chronic lung injuries 

CORPORATE HIGHLIGHTS

Myovant Sciences
On March 23, Myovant Sciences announced recipients of “Forward for Health Equity” grants to improve healthcare access in prostate cancer and uterine fibroids.

On February 16, Myovant Sciences and HealthyWomen, the nation’s leading independent, nonprofit health information source for women, launched “Voices of Periods”, an educational campaign to fight menstrual stigma. 

On January 5, Myovant Sciences announced the U.S. availability of ORGOVYX™ (relugolix) for the treatment of advanced prostate cancer. ORGOVYX is a gonadotropin-releasing hormone (GnRH) receptor antagonist indicated for the treatment of adult patients with advanced prostate cancer.  More information about ORGOYYX, including the full product prescribing information, can be found here

On January 4, Myovant Sciences announced the appointment of industry veteran David Marek as its new Chief Executive Officer and as a member of its board of directors. 

Urovant Sciences
On February 12, Urovant announced that the Urovant Board authorized and approved the definitive merger agreement in which Sumitovant Biopharma would acquire all outstanding shares of Urovant that are not held by Sumitovant Biopharma. 

On March 23, Urovant shareholders voted to approve the definitive merger agreement with Sumitovant Biopharma by an affirmative vote of holders of a majority of Urovant’s outstanding shares, and by holders of a majority of Urovant’s outstanding shares that were not held by Sumitovant Biopharma. 

On March 29, Urovant and Sumitovant Biopharma jointly announced the completion of the merger in which Sumitovant acquired all outstanding shares of Urovant. Urovant became a wholly owned subsidiary of Sumitovant Biopharma and Urovant’s stock ceased trading on the Nasdaq stock market.

Spirovant Sciences
On January 26, Spirovant Sciences announced that the leasing of new and expanded headquarters and laboratories in Philadelphia’s University City and that the company expanded its leadership team with the appointments of Eric Pastor as Senior Vice President of Technology Development and Operations and Maria Limberis, PhD, as Vice President of Research.

About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is the majority shareholder of Myovant (NYSE: MYOV) and wholly owns Urovant, Enzyvant, Spirovant, and Altavant. Sumitovant’s promising pipeline is comprised of early-through late-stage investigational medicines across a range of disease areas targeting high unmet need. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma. For further information about Sumitovant, please visit https://www.sumitovant.com. Follow Sumitovant on LinkedIn.

About Sumitomo Dainippon Pharma Co., Ltd. 
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China, and the European Union. Sumitomo Dainippon Pharma was formed pursuant to the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com.

About Altavant Sciences
Altavant Sciences is a clinical-stage biopharmaceutical company focused on elevating patient-centric drug development in rare respiratory diseases. Altavant is currently developing two pipeline candidates: rodatristat ethyl and ALTA-2530. Rodatristat is a tryptophan hydroxylase (TPH) inhibitor in Phase 2 development for patients with pulmonary arterial hypertension. By reducing serotonin production via TPH inhibition rodatristat may play a role in halting or reversing the vascular remodeling associated with PAH, offering a novel treatment option for patients living with this disease. ALTA-2530 is an inhaled interleukin-1 receptor antagonist under development for bronchiolitis obliterans syndrome (BOS), a life-threatening form of chronic lung allograft dysfunction (CLAD) that may present following lung transplantation. ALTA-2530’s unique mechanism of action may offer a novel treatment option for patients who suffer from BOS, a disease where there are currently no approved therapies.

Altavant is a wholly owned subsidiary of Sumitovant Biopharma Ltd. For more information, please visit https://altavant.com

About Myovant Sciences 
Myovant Sciences (Myovant) aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. ORGOVYX™ (relugolix) was approved by the FDA in 2020 as the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist for the treatment of adult patients with advanced prostate cancer, and relugolix is also under regulatory review in Europe for men with advanced prostate cancer. Myovant’s lead product candidate, relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg), is under regulatory review in the U.S. and Europe for women with uterine fibroids, has completed Phase 3 registration-enabling studies for women with endometriosis, and is being assessed for contraceptive efficacy in healthy women ages 18-35 years who are at risk for pregnancy. Myovant is also developing MVT-602, an oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd., is Myovant’s majority shareholder. For more information, please visit Myovant’s website at www.myovant.com. Follow @Myovant on Twitter and LinkedIn.

About Spirovant Sciences, Inc.
Spirovant is a gene therapy company focused on changing the course of cystic fibrosis and other respiratory diseases. The company’s current investigational gene therapy technologies are designed to overcome the historical barriers that have prevented effective genetic treatments for cystic fibrosis. Spirovant’s lead programs are in development for cystic fibrosis. Spirovant is a wholly owned subsidiary of Sumitovant Biopharma Ltd., which is itself a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd.  Spirovant is located in Philadelphia, PA. More information is available at https://www.spirovant.com/.

About Urovant Sciences
Urovant Sciences is a biopharmaceutical company focused on developing and commercializing innovative therapies for urologic conditions. The company’s lead product, GEMTESA® (vibegron), is an oral, once-daily (75 mg) small molecule beta-3 agonist approved by the U.S. FDA in December 2020 for the treatment of adult patients with overactive bladder (OAB) with symptoms of urge urinary incontinence, urgency and urinary frequency. GEMTESA is also being evaluated for the treatment of OAB in men with benign prostatic hyperplasia (OAB+BPH). The company’s second product candidate, URO-902, is a novel gene therapy being developed for patients with OAB who have failed oral pharmacologic therapy. Urovant Sciences, a subsidiary of Sumitovant Biopharma Ltd., intends to develop novel treatments for additional urologic diseases. Learn more about us at www.urovant.com.

Forward-Looking Statements 
This press release contains forward-looking statements that are based on management’s assumptions and beliefs in light of information available up to the day of announcement and thus involve both known and unknown risks and uncertainties. Actual financial results and other situations of the future may differ materially from those presented in this press release due to various factors.

For more information with respect to Myovant Sciences, including disclosure regarding the risks and uncertainties related to any forward-looking statements, please refer to Myovant Sciences’ filings with the United States Securities and Exchange Commission (“SEC”), including under the heading “Risk Factors” in Myovant Sciences’ Quarterly Report on Form 10-Q filed on February 10, 2020, as such risk factors may be amended, supplemented or superseded from time to time.

This press release contains “forward-looking statements” concerning the development and commercialization of Altavant’s products, the company’s business development efforts and its expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements. These statements are made as of the date of this press release. Actual results may vary. Altavant undertakes no obligation to update any forward-looking statements for any reason.

Media Contacts:

Sumitovant Biopharma
Mary Stutts
SVP, Corporate Relations
media@sumitovant.com

Myovant Investor Contact:
Frank Karbe
President, Chief Financial Officer
Myovant Sciences, Inc.
investors@myovant.com

Myovant Media Contact:
Albert Liao 
Director, Corporate Communications
Myovant Sciences, Inc.
media@myovant.com

Urovant Investor Contact:
Ryan Kubota
ryan.kubota@urovant.com

Urovant Media Contact:
media@urovant.com

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Myovant Sciences Receives Positive CHMP Opinion for RYEQO® (Relugolix Combination Tablet) for the Treatment of Women With Uterine Fibroids

BASEL, Switzerland, May 21, 2021 (GLOBE NEWSWIRE) — Myovant Sciences (NYSE: MYOV) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agencyhas adopted a positive opinion recommending the approval of RYEQO® (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age. The European Commission will review the CHMP recommendation, and a final decision on the Marketing Authorization Application is expected to be available in approximately two months. The decision will be applicable to all 27 European Union member states plus Iceland, Norway, and Liechtenstein.

“Over 25% of women of reproductive age develop uterine fibroids. This chronic disease can cause debilitating symptoms that have a significant impact on quality of life and require long-term treatment, yet there are currently limited treatment options in Europe and many women are faced with the decision to undergo surgery to alleviate symptoms,” said Roberta Venturella, M.D., Ph.D., Associate Professor, Magna Græcia University of Catanzaro and investigator in the LIBERTY program. “The CHMP’s positive opinion further validates RYEQO’s potential to effectively address heavy menstrual bleeding and pain associated with uterine fibroids and serve as an important new treatment option for patients and physicians.”

“This positive CHMP opinion represents an important step in advancing our mission to redefine care for women living with uterine fibroids,” said David Marek, Chief Executive Officer of Myovant Sciences, Inc.“We look forward to Gedeon Richter’s launch of RYEQO, if approved, as a new treatment option for uterine fibroids.”

The positive opinion recommending approval is based on safety and efficacy data from the Phase 3 LIBERTY program, which consisted of two replicate, 24-week, multinational clinical studies (LIBERTY 1 and LIBERTY 2), a one-year extension study, and a randomized withdrawal study assessing the safety and efficacy for up to two years of relugolix combination therapy (relugolix 40 mg plus estradiol 1.0 mg and norethindrone acetate 0.5 mg). Results from the LIBERTY 1 and LIBERTY 2 studies were published in the New England Journal of Medicine in February 2021.

In March 2020, Myovant and Gedeon Richter entered into an exclusive license agreement for Gedeon Richter to commercialize relugolix combination tablet for uterine fibroids and endometriosis in Europe, the Commonwealth of Independent States including Russia, Latin America, Australia, and New Zealand. Under the terms of the agreement, Myovant continues to lead the global development of relugolix combination tablet while Gedeon Richter is responsible for local clinical development, manufacturing, and all commercialization for its territories.

Relugolix combination tablet for the treatment of uterine fibroids is also under review by the U.S. Food and Drug Administration, with a target action date of June 1, 2021.

About Uterine Fibroids
Uterine fibroids are noncancerous tumors that develop in or on the muscular walls of the uterus and are among the most common reproductive tract tumors in women. In addition to an individual’s genetic predisposition, estrogens are well known to play an important role in the regulation of fibroid growth.

Although uterine fibroids are benign tumors, they can cause debilitating symptoms such as heavy menstrual bleeding (frequently resulting in anemia and fatigue), pain (including painful periods, abdominal pain, painful intercourse, backache), increased abdominal girth and bloating, urinary frequency or retention, constipation, pregnancy loss, and, in some cases, infertility. These symptoms can also lead to loss of productivity at work, limitations in normal activities of daily living, and social embarrassment.

About RYEQO®
RYEQO (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is being evaluated for the treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age. RYEQO contains relugolix, which reduces the amount of estrogen (and other hormones) produced by ovaries, estradiol (an estrogen), which may reduce the risk of bone loss, and norethindrone acetate (a progestin), which is necessary when women with a uterus (womb) take estrogen.

RYEQO is not approved for any indication in any geography.

About Myovant Sciences
Myovant Sciences aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. ORGOVYX™ (relugolix) was approved by the U.S. Food and Drug Administration in 2020 as the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist for the treatment of adult patients with advanced prostate cancer, and relugolix is also under regulatory review in Europe for men with advanced prostate cancer. Our lead product candidate, relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg), is under regulatory review in the U.S. and Europe for women with uterine fibroids, has completed Phase 3 registration-enabling studies for women with endometriosis, and is being assessed for contraceptive efficacy in healthy women ages 18-35 years who are at risk for pregnancy. We are also developing MVT-602, an oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd., is our majority shareholder. For more information, please visit our website at www.myovant.com. Follow @Myovant on Twitter and LinkedIn.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In this press release, forward-looking statements include, but are not limited to, all statements reflecting Myovant Sciences’ expectations, including: statements regarding Myovant’s aspiration to redefine care for women and for men; the expectations of commercialization and launch of RYEQO® for uterine fibroids in Europe by Gedeon Richter, if approved; the expectations of the European Commission’s review of the CHMP recommendation and the timeline of its final decision on the Marketing Authorization Application and the application of such decision in the European Union; and Myovant’s expectations regarding the potential benefits of RYEQO® and it serving as an important new treatment option for uterine fibroids, if approved.

Myovant Sciences’ forward-looking statements are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties, assumptions and other factors known and unknown that could cause actual results and the timing of certain events to differ materially from future results expressed or implied by the forward-looking statements, including unforeseen circumstances or other disruptions to normal business operations arising from or related to the COVID-19 pandemic. Myovant Sciences cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur and actual results could differ materially from those expressed or implied by these forward-looking statements. Factors that could materially affect Myovant Sciences’ operations and future prospects or which could cause actual results to differ materially from expectations include, but are not limited to the risks and uncertainties listed in Myovant Sciences’ filings with the United States Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in Myovant Sciences’ Annual Report on Form 10-K filed on May 11, 2021, as such risk factors may be amended, supplemented or superseded from time to time. These risks are not exhaustive. New risk factors emerge from time to time and it is not possible for Myovant Sciences’ management to predict all risk factors, nor can Myovant Sciences assess the impact of all factors on its business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. You should not place undue reliance on the forward-looking statements in this press release, which speak only as of the date hereof, and, except as required by law, Myovant Sciences undertakes no obligation to update these forward-looking statements to reflect events or circumstances after the date of such statements.

Investor Contact
Ryan Crowe, Investors
+1 (650) 781-9106
investors@myovant.com

Media Contact
Albert Liao, Media
+1 (650) 410-3055
media@myovant.com

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Altavant Sciences Reports Rodatristat Ethyl PK/PD Analysis at ATS Supporting Doses Selected for Phase 2b ELEVATE 2 Study

CARY, N.C. and BASEL, Switzerland — May 20, 2021 — Altavant Sciences, a clinical-stage biopharmaceutical company focused on patient-centric drug development in rare respiratory diseases, presented an update on the clinical development of rodatristat ethyl, the company’s lead candidate for the treatment of pulmonary arterial hypertension (PAH), at the recent meeting of the American Thoracic Society (ATS). This update included findings from a dose-response model, which simulated daily administration of rodatristat ethyl to identify optimal doses for the Phase 2b clinical trial.

In a 28-day preclinical study, a 40% reduction in serotonin biosynthesis significantly improved vascular remodeling and pulmonary vascular resistance in animal models of PAH. Altavant developed a dose-response model that accounted for variabilities in drug exposure and drug response observed in previous clinical studies. After conducting 10,000 simulations, doses of 300mg and 600mg BID were identified as having a high probability of achieving the targeted 40% reduction in serotonin biosynthesis. The company is assessing these doses in PAH patients in the randomized, double-blind Phase 2b ELEVATE 2 (NCT04712669) study, which is now enrolling patients at sites in the US, and later this year in the EU, and Canada. The e-poster, A Pharmacokinetic / Pharmacodynamic-Based Rationale for Dose Selection of the TPH Inhibitor Rodatristat Ethyl in ELEVATE–2 – a Phase 2b Study in Pulmonary Arterial Hypertension can be found on Altavant’s website.

Bill Symonds, Pharm.D., Chief Executive Officer of Altavant, explained, “In preparation for initiating ELEVATE 2, we conducted extensive nonclinical and clinical research on the pharmacokinetics and pharmacodynamics of rodatristat ethyl. As we reported at ATS in 2020, we performed nonclinical studies to assess the drug candidate’s penetration into lung tissue as well as exposure in the periphery and central nervous system; the latter was performed to ensure that brain levels of serotonin are preserved. Now that we have identified the optimal dose levels, we are excited to begin enrolling patients into the ELEVATE 2 study.”

ELEVATE 2 will test the ability of rodatristat to reduce the peripheral production of serotonin through its unique mechanism of tryptophan hydroxylase (TPH) inhibition in order to improve PAH symptoms. The pathology underlying PAH appears to be driven, in part, by the unchecked production of peripheral serotonin, which drives the proliferation and constriction of the smooth muscle cells in the wall of the pulmonary arteries that is a hallmark of the disease.

Clinicians and others interested in learning more about rodatristat’s unique mechanism of action are encouraged to watch the archived video symposium: Serotonin Pathobiology in Pulmonary Arterial Hypertension and Phase 2b Investigation of the Novel Therapy Rodatristat Ethyl (RE) in WHO Group 1 PAH. This event was hosted by Altavant on May 18, 2021 from 5:30 – 6:00 p.m. Eastern Time and featured a presentation by Marc Humbert, M.D., Ph.D., Director of the French Pulmonary Hypertension Referral Center & Professor of Respiratory Medicine at the Université Paris-Saclay.

In addition, Lyn Baranowski, Altavant’s Chief Operating Officer gave a presentation focused on the company’s patient-centered clinical development strategy, titled: Bench to Bedside: The Importance of the Patient Voice in the Drug Discovery Pipeline. Ms. Baranowski discussed several aspects of the company’s approach, including: the use of advisory boards and one-on-one interviews to capture and address patient concerns, the development of patient-focused materials and trial summary reports, and partnerships with organizations like Greater Gift.

Pre-recorded presentations of the e-poster and the patient-centered clinical development strategy are available on demand on the ATS virtual platform until July 2, 2021, and all presentations are archived on the Altavant website in compliance with the congress’ embargo policy.

About Altavant Sciences
Altavant Sciences is a clinical-stage biopharmaceutical company focused on elevating patient-centric drug development in rare respiratory diseases. Altavant is currently developing two pipeline candidates: rodatristat ethyl and ALTA-2530. Rodatristat is a tryptophan hydroxylase (TPH) inhibitor in Phase 2 development for patients with pulmonary arterial hypertension. By reducing serotonin production via TPH inhibition rodatristat may play a role in halting or reversing the vascular remodeling associated with PAH, offering a novel treatment option for patients living with this disease. ALTA-2530 is an inhaled interleukin-1 receptor antagonist under development for bronchiolitis obliterans syndrome (BOS), a life-threatening form of chronic lung allograft dysfunction (CLAD) that may present following lung transplantation. ALTA-2530’s unique mechanism of action may offer a novel treatment option for patients who suffer from BOS, a disease where there are currently no approved therapies.

Altavant is a wholly owned subsidiary of Sumitovant Biopharma Ltd. For more information, please visit https://altavant.com

About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sumitovant is the majority shareholder of Myovant, and wholly owns Enzyvant, Spirovant, Urovant, and Altavant. Sumitovant’s pipeline is comprised of early- through late-stage investigational medicines across a range of disease areas targeting high unmet need.

Sumitovant Biopharma Ltd. is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. For further information about Sumitovant please visit https://www.sumitovant.com/

About Sumitomo Dainippon Pharma Co., Ltd.
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China and the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com/.

Forward-Looking Statements
This press release contains “forward-looking statements” concerning the development and commercialization of Altavant’s products, the company’s business development efforts and its expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements. These statements are made as of the date of this press release. Actual results may vary. Altavant undertakes no obligation to update any forward-looking statements for any reason.

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Myovant Sciences Announces Corporate Updates and Financial Results for Fourth Fiscal Quarter and Fiscal Year Ended March 31, 2021


BASEL, Switzerland, May 11, 2021 (GLOBE NEWSWIRE) — Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women and for men, today announced corporate updates and financial results for the fourth fiscal quarter and fiscal year ended March 31, 2021.

“The ORGOVYX launch is off to a strong start and its differentiated clinical profile has the potential to redefine care for men with advanced prostate cancer. ORGOVYX demand accelerated over the course of the quarter, reflecting our ongoing efforts to educate urologists and medical oncologists about ORGOVYX while improving access and reimbursement for patients. In partnership with Pfizer, we continue to execute on our long-term goal of establishing ORGOVYX as the new standard of care androgen deprivation therapy,” said David Marek, Chief Executive Officer of Myovant Sciences, Inc. “I am also pleased with the progress we have made in preparing for the U.S. launch of relugolix combination tablet in women with uterine fibroids, which is expected this June. In addition, we have advanced relugolix combination tablet toward a U.S. regulatory submission in endometriosis and our European regulatory submission for relugolix monotherapy for the treatment of advanced prostate cancer was validated by the European Medicines Agency.”

Fourth Fiscal Quarter 2020 and Recent Corporate Updates

ORGOVYX

Relugolix Monotherapy

Relugolix Combination Tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg)

Executive Appointments

Expected Upcoming Milestones

Fourth Fiscal Quarter and Fiscal Year Ended March 31, 2021 Financial Summary

Total revenues for the three months and year ended March 31, 2021, were $24.6 million and $59.3 million, respectively. There were no revenues recorded in the comparable prior year periods.

Cost of product revenue for the three months and year ended March 31, 2021, was $0.3 million related to the cost of goods sold and royalty expense payable to Takeda pursuant to the Takeda License Agreement. There were no such expenses for the comparable prior year periods.

Collaboration expense to Pfizer for the three months and year ended March 31, 2021, was $1.7 million, reflecting Pfizer’s 50% share of net profits from sales of ORGOVYX in the U.S., pursuant to the Pfizer Collaboration and License Agreement. There were no such expenses for the comparable prior year periods.

Research and development (R&D) expenses for the three months ended March 31, 2021, were $21.6 million compared to $41.7 million for the comparable prior year period. R&D expenses for the year ended March 31, 2021, were $136.7 million compared to $192.6 million for the prior fiscal year. The decrease in R&D expenses reflects a reduction in clinical study costs as a result of the wind down of Myovant’s Phase 3 LIBERTY, HERO, and SPIRIT studies and cost share reimbursements from Pfizer for certain R&D expenses in the fiscal 2020 periods. This decrease was partially offset primarily by an increase in personnel expenses, mainly driven by the continued expansion of Myovant’s medical affairs organization to support the U.S. commercial launch of ORGOVYX and the potential U.S. commercial launches of relugolix combination tablet for the women’s health indications, if approved, as well as regulatory expenses and incremental spend on new relugolix development programs.

Selling, general and administrative (SG&A) expenses for the three months ended March 31, 2021, were $78.0 million compared to $22.4 million for the comparable prior year period. SG&A expenses in the year ended March 31, 2021, were $181.4 million compared to $82.3 million for the prior fiscal year. The increase was primarily due to higher expenses related to commercial activities to support the ORGOVYX U.S. launch and commercial readiness activities for the potential U.S. launch of relugolix combination tablet, higher personnel-related costs primarily due to the hiring of Myovant’s commercial operations, marketing, and market access teams, as well as the oncology sales force, higher share-based compensation expense, and general overhead expenses to support Myovant’s organizational growth. Share-based compensation expense for the three months and year ended March 31, 2021 includes incremental expense of $25.7 million related to the acceleration, modification, and remeasurement of our former Principal Executive Officer’s outstanding equity awards. SG&A expenses for the year ended March 31, 2020 included $10.2 million in share-based compensation expense related to the accelerated vesting of certain equity awards as well as a $3.6 million capital tax accrual.

Interest expense was $3.5 million for the three months ended March 31, 2021, compared to $1.4 million for the comparable prior year period. Interest expense was $10.4 million for the year ended March 31, 2021, compared to $12.7 million for the prior fiscal year. The decrease in interest expense, despite higher outstanding loan balances, was primarily driven by the significantly lower interest rates associated with the Sumitomo Dainippon Pharma Loan Agreement as compared to Myovant’s previously outstanding debt obligations, which were repaid in December 2019.

There was no loss on extinguishment of debt for the year ended March 31, 2021. For the year ended March 31, 2020, Myovant recorded a $4.9 million loss resulting from the early repayment of Myovant’s previously outstanding debt obligations in December 2019.

Interest income for the three months ended March 31, 2021, was less than $0.1 million compared to $0.2 million for the comparable prior year period. Interest income for the year ended March 31, 2021, was $0.2 million compared to $2.6 million for the prior fiscal year. The decrease was primarily due to decreases in interest rates.

Foreign exchange loss (gain) for the three months ended March 31, 2021, was a loss of less than $0.1 million compared to a gain of $0.5 million for the comparable prior year period. Foreign exchange gain for the year ended March 31, 2021, was $16.2 million compared to $1.6 million for the prior fiscal year. The increase for the year ended March 31, 2021 was primarily due to a larger foreign currency exchange gain on Myovant’s outstanding balance under the Sumitomo Dainippon Pharma Loan Agreement during the year ended March 31, 2021 compared to the prior year.

Net loss for the three months ended March 31, 2021, was $81.4 million compared to $64.9 million for the comparable prior year period. Net loss for the year ended March 31, 2021, was $255.1 millioncompared to $289.0 million for the prior fiscal year. On a per common share basis, net loss was $0.89 and $0.73 for the three months ended March 31, 2021 and 2020, respectively, and $2.83 and $3.37 for the year ended March 31, 2021 and 2020, respectively.

Capital resources: Cash, cash equivalents, marketable securities, and amounts available under the Sumitomo Dainippon Pharma Loan Agreement totaled $726.2 million as of March 31, 2021, and consisted of $684.9 million of cash, cash equivalents, and marketable securities and $41.3 million of available borrowing capacity under the Sumitomo Dainippon Pharma Loan Agreement.

Conference Call
As previously announced, Myovant will hold a webcast and conference call at 8:30 a.m. Eastern Time (5:30 a.m. Pacific Time) today, May 11, 2021, to discuss corporate updates and financial results for its fourth fiscal quarter and fiscal year ended March 31, 2021. Investors and the general public may access a live webcast of the call by visiting the investor relations page of Myovant’s website at investors.myovant.com. Institutional investors and analysts may also participate in the conference call by dialing 1-800-532-3746 in the U.S. or +1-470-495-9166 from outside the U.S. The webcast will be archived on Myovant’s Investor Relations website following the call.

About Relugolix
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces testicular testosterone, a hormone known to stimulate the growth of prostate cancer, and ovarian estradiol, a hormone known to stimulate the growth of uterine fibroids and endometriosis. Relugolix monotherapy (120 mg) is FDA-approved as ORGOVYX™ for the treatment of adult patients with advanced prostate cancer and is under regulatory review in Europe for the treatment of men with advanced prostate cancer. Relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is under regulatory review in the U.S. and Europe for women with uterine fibroids, has completed Phase 3 registration-enabling studies for women with endometriosis, and is being assessed for contraceptive efficacy in healthy women ages 18-35 years who are at risk for pregnancy.

About Myovant Sciences
Myovant Sciences (Myovant) aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. ORGOVYX™ (relugolix) was approved by the FDA in 2020 as the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist for the treatment of adult patients with advanced prostate cancer, and relugolix is also under regulatory review in Europe for men with advanced prostate cancer. Myovant’s lead product candidate, relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg), is under regulatory review in the U.S. and Europe for women with uterine fibroids, has completed Phase 3 registration-enabling studies for women with endometriosis, and is being assessed for contraceptive efficacy in healthy women ages 18-35 years who are at risk for pregnancy. Myovant is also developing MVT-602, an oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd., is Myovant’s majority shareholder. For more information, please visit Myovant’s website at www.myovant.com. Follow @Myovant on Twitter and LinkedIn.

About Sumitomo Dainippon Pharma Co., Ltd.
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including the U.S., Japan, China, and the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com.

About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is the majority shareholder of Myovant, and wholly owns Urovant Sciences, Enzyvant Therapeutics, Spirovant Sciences, and Altavant Sciences. Sumitovant’s pipeline is comprised of commercialized and investigational medicines across a range of disease areas targeting high unmet need. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma. For further information about Sumitovant, please visit https://www.sumitovant.com.

Forward-Looking Statements 
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In this press release, forward-looking statements include, but are not limited to, all statements reflecting Myovant Sciences’ expectations, including: statements regarding Myovant’s aspiration to redefine care for women and for men; certain statements with respect to expectations of commercialization of ORGOVYX in Mr. Marek’s quote; Myovant’s expectations regarding status of its publicly announced milestones; Myovant’s expected timelines of coverage decisions by commercial and Medicare Part D payors; the timing of Myovant’s regulatory submissions, anticipated regulatory review results, and U.S. launch of relugolix combination tablet in women with uterine fibroids; the design and any expectation of the results of the SERENE study; and those statements under the caption “Expected Upcoming Milestones.”

Myovant Sciences’ forward-looking statements are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties, assumptions and other factors known and unknown that could cause actual results and the timing of certain events to differ materially from future results expressed or implied by the forward-looking statements, including unforeseen circumstances or other disruptions to normal business operations arising from or related to the COVID-19 pandemic. Myovant Sciences cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur and actual results could differ materially from those expressed or implied by these forward-looking statements. Factors that could materially affect Myovant Sciences’ operations and future prospects or which could cause actual results to differ materially from expectations include, but are not limited to the risks and uncertainties listed in Myovant Sciences’ filings with the United States Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in Myovant Sciences’ Annual Report on Form 10-K to be filed on May 11, 2021, as such risk factors may be amended, supplemented or superseded from time to time. These risks are not exhaustive. New risk factors emerge from time to time and it is not possible for Myovant Sciences’ management to predict all risk factors, nor can Myovant Sciences assess the impact of all factors on its business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. You should not place undue reliance on the forward-looking statements in this press release, which speak only as of the date hereof, and, except as required by law, Myovant Sciences undertakes no obligation to update these forward-looking statements to reflect events or circumstances after the date of such statements.

MYOVANT SCIENCES LTD.
Condensed Consolidated Statements of Operations
(Unaudited, in thousands, except share and per share data)

 Three Months Ended March 31,Years Ended March 31,
 2021202020212020
Revenues:
Product revenue, net$3,630$3,360
Collaboration revenue20,97522,354
License and milestone revenue33,333
Total revenues24,60559,317
Operating costs and expenses:  
Cost of product revenue 301301
Collaboration expense to Pfizer1,6641,664
Research and development (1)21,553 41,713136,713192,560
Selling, general and administrative (1)78,03622,430181,42382,327
Total operating costs and expenses101,554 64,143320,101274,887
Loss from operations(76,949)(64,143)(260,784)(274,887)
Interest expense3,493 1,425 10,40112,663
Loss on extinguishment of debt —4,851
Interest income(33) (247) (211)(2,552)
Foreign exchange loss (gain)2 (470)(16,176)(1.621)
Loss before income taxes(80,411)(64,851)(254,798)(288,228)
Income tax expense95262336761
Net loss$(81,363)$(64,913)$(255,134)$(288,989)
Net loss per common share — basic and diluted$(0.89)$(0.73)$(2.83)$(3.37)
Weighted average common shares outstanding — basic and diluted91,018,20489,130,80690,036,45985,839,303

(1) Includes the following share-based compensation expenses:

Research and development$2,989$2,959$14,049$14,524
Selling, general and administrative28,9413,11439,62725,727
Total share-based compensation expense $31,930$6,073$53,676$40,251

MYOVANT SCIENCES LTD.
Condensed Consolidated Balance Sheet
(Unaudited, in thousands)

 March 31, 2021March 31, 2020
Assets  
Current assets:  
Cash and cash equivalents$674,493 $76,644
Accounts receivable, net3,570
Marketable securities10,435 2,997
Inventory2,611
Prepaid expenses and other current assets13,536 8,269
Total current assets704,645 87,910
Property and equipment, net3,300 2,497
Operating lease right-of-use asset9,655 11,146
Other assets7,427 4,373
Total assets$725,027 $105,926
Liabilities and Shareholders’ (Deficit)  
Current liabilities:  
Accounts payable$17,809 $15,334
Accrued expenses and other current liabilities 44,612 29,060
Share-based compensation liabilities 21,636
Deferred revenue100,56440,000
Amounts due to collaboration partnership 1,954
Cost share advance from collaboration partner92,415
Operating lease liability1,807 1,515
Amounts due to related parties54315
Total current liabilities281,34085,925
Deferred revenue, non-current397,369
Cost share advance from collaboration partner, non-current29,447
Long-term operating lease liability9,18910,996
Long-term debt, less current maturities (related party)358,700 113,700
Other2,9473,582
Total liabilities1,078,992 214,203
Total shareholders’ deficit(353,965)(108,277)
Total liabilities and shareholders’ deficit$725,027 $105,926

Investor Contact: 
Ryan Crowe
Vice President, Investor Relations
Myovant Sciences, Inc.
+1 (650) 781-9106
investors@myovant.com

Media Contact:
Albert Liao
Director, Corporate Communications
Myovant Sciences, Inc.
+1 (650) 410-3055
media@myovant.com

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Altavant Sciences Advances Rodatristat Ethyl into Phase 2b Clinical Trial for Pulmonary Arterial Hypertension

Cary, N.C. and Basel, Switzerland, May 06, 2021 (GLOBE NEWSWIRE) — Altavant Sciences, a clinical-stage biopharmaceutical company focused on patient-centric drug development in rare respiratory diseases, announced today that the company has initiated its ELEVATE 2 Study, a Phase 2b clinical trial of its lead product candidate, rodatristat ethyl (“rodatristat”), for the treatment of patients with pulmonary arterial hypertension (PAH).  

Rodatristat is an orally bioavailable, direct and reversible tryptophan hydroxylase (TPH) inhibitor designed to block peripheral serotonin production. In an earlier study in healthy volunteers, levels of 5-hydroxyindoleacetic acid (5-HIAA), a biomarker of peripheral serotonin, were reduced after 14 days of once-daily oral rodatristat administration. The degree of serotonin lowering was consistent with that observed in preclinical models of PAH that generated marked improvements in vessel remodeling.

“Dysregulated, excessive serotonin signaling in the lungs of patients with PAH is a life-threatening condition, causing excessive growth of pulmonary-artery smooth muscle cells as well as the release of proinflammatory and profibrotic molecules, all of which constrict pulmonary blood vessels,” stated Marc Humbert, M.D., Ph.D., Professor of Respiratory Medicine at the Université Paris-Saclay and an investigator in the ELEVATE 2 Study. “Decades of research point to the critical role of serotonin in PAH disease progression and support the hypothesis that by reducing levels of the hormone in the periphery, we may be able to improve, and potentially even reverse, pulmonary remodeling in these patients.”

Altavant’s ELEVATE 2 Study is a dose-ranging, randomized, double-blind, placebo-controlled trial in approximately 90 adults with confirmed diagnoses of symptomatic PAH belonging to one of several subtypes, including idiopathic, heritable or toxin-induced PAH, referred to as WHO Group 1 PAH. Enrolled patients will be randomized to receive one of two doses of rodatristat or placebo twice daily, while continuing their existing prescribed PAH treatments. Safety, pharmacokinetics and efficacy will be assessed at various points in the study’s screening, treatment and follow-up phases via in-clinic, home and telemedicine visits. Efficacy will be assessed as the percent change from baseline of pulmonary vascular resistance (PVR), as measured by right heart catheterization. Secondary efficacy endpoints include change in 5-HIAA, change in validated PAH scales, and change in six-minute walk test.

“Extensive in vivo and early clinical testing strongly support the rodatristat mechanism as a potential disease-modifying treatment for conditions characterized by excessive production of serotonin, including PAH,” added William T. Symonds, Pharm.D., chief executive officer of Altavant. “We are excited to advance our lead candidate into Phase 2b development, and by the opportunity to provide meaningful benefit to patients with this disease.”

“Tufts Medical Center, a PHA Center of Comprehensive Care, strives to be a leader in the diagnosis and treatment of patients with PAH,” stated Dr. Nicholas Hill, Chief Pulmonary, Critical Care and Sleep at Tufts Medical Center and an investigator in the ELEVATE 2 Study. “As a pulmonologist, it is particularly rewarding to be involved in the advancement of new treatments, especially those that have the potential to be disease modifying, like rodatristat.”

More information may be found on the Altavant Sciences website and on www.elevatePAHstudy.com.

About Pulmonary Arterial Hypertension
Pulmonary arterial hypertension (PAH) is a rare, progressive disorder characterized by vasoconstriction, cellular proliferation and remodeling in the small pulmonary arteries. These changes lead to high arterial pressure, right heart strain, and ultimately, right heart failure and death. Although there are approved treatments used for PAH, they mainly help alleviate symptoms, primarily via vasodilation, and none reverse the disease process. Long-term survival rates for PAH are poor, with less than 40 percent survival at five years for high-risk patients.

About Rodatristat 
Rodatristat is a tryptophan hydroxylase (TPH) inhibitor designed to reduce the body’s peripheral production of serotonin. A significant body of scientific evidence supports dysregulated peripheral serotonin production as a trigger of aberrant proliferation and constriction of the smooth muscle cells in the wall of the pulmonary arteries, causing them to restrict blood flow in pulmonary arterial hypertension (PAH). By lowering circulating serotonin levels, it is believed that rodatristat may halt or reverse the pathology of diseases that are driven by excessive serotonin production, such as PAH, idiopathic pulmonary fibrosis (IPF) and sarcoidosis. Altavant is currently testing this mechanism of action in the ELEVATE proof-of-concept Phase 2 study of rodatristat in patients with pulmonary arterial hypertension (PAH).

About Altavant Sciences
Altavant Sciences is a clinical-stage biopharmaceutical company focused on elevating patient-centric drug development in rare respiratory diseases. Altavant is currently advancing two pipeline candidates: rodatristat and ALTA-2530. Rodatristat is in Phase 2 development for patients with pulmonary arterial hypertension. A tryptophan hydroxylase (TPH) inhibitor, rodatristat may play a role in halting or reversing the vascular remodeling associated with PAH, offering a novel treatment option for patients living with this disease. ALTA-2530 is an interleukin-1 receptor antagonist under development for bronchiolitis obliterans syndrome (BOS), a life-threatening form of chronic lung allograft dysfunction (CLAD) that may present following lung transplantation. ALTA-2530’s unique mechanism of action may offer a novel treatment option for patients who suffer from BOS, a disease where there are currently no approved therapies.

Altavant is a wholly owned subsidiary of Sumitovant Biopharma Ltd. For more information, please visit https://altavant.com

About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sumitovant is the majority shareholder of Myovant, and wholly owns Enzyvant, Urovant, Spirovant and Altavant. Sumitovant’s pipeline is comprised of early- through late-stage investigational medicines across a range of disease areas targeting high unmet need.

Sumitovant Biopharma Ltd. is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. For further information about Sumitovant please visit https://www.sumitovant.com/

About Sumitomo Dainippon Pharma Co., Ltd.
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China and the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com/.

Forward-Looking Statements
This press release contains “forward-looking statements” concerning the development and commercialization of Altavant’s products, the company’s business development efforts and its expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements. These statements are made as of the date of this press release. Actual results may vary. Altavant undertakes no obligation to update any forward-looking statements for any reason.

Contact:

Aline Sherwood
Scienta Communications, LLC
asherwood@scientapr.com

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Aceragen Launches with Acquisition of Enzyvant‘s Investigational Therapy for Farber Disease and $35 Million Product Financing with NovaQuest

Research Triangle Park, NC and Basel, CH – May 3, 2021 – Aceragen, Inc., a biopharmaceutical company focused on advancing transformational therapeutics for rare and ultra-rare diseases, today announced the acquisition of Enzyvant’s RVT-801 (now ACG-801), an investigational enzyme replacement therapy (ERT) for acid ceramidase deficiency presenting as Farber disease, a lysosomal storage disease with a unique, severe inflammatory phenotype for which no disease-specific therapy exists. Enzyvant will receive an upfront payment and development and sales-based milestones up to $226 million, as well as tiered royalties on net sales. In addition, Aceragen entered into a $35 million product financing agreement with NovaQuest Capital Management to fund the development program into a potential registrational study. 

Aceragen was recently founded by a team of industry and medical veterans with a long record of accomplishment in product development and commercialization in biopharmaceuticals, much of which has been in the rare disease field. Co-founder John Taylor will lead the company as President and Chief Executive Officer, having previously held the same roles with Spyryx Biosciences. 

“The Aceragen team is delighted to announce the strategic transaction with Enzyvant and the relationship we have established with NovaQuest to continue the development of ACG-801,” Taylor stated. “This program is based on the foundational work of Dr. Ed Schuchman at the Icahn School of Medicine at Mount Sinai, establishing the potential to address the underlying pathology of Farber disease, a genetic deficiency of acid ceramidase. In addition to the intellectual property and regulatory designations, Aceragen has also acquired from Enzyvant a robust preclinical package including several completed toxicology studies, a quantitative patient research study and the first ever natural history study in patients with Farber disease that documented and quantified the features, impact and progression of this devastating condition.  Aceragen’s relationship with NovaQuest, an experienced investor in the area of pharmaceutical development, will enable us to advance through the planned clinical study and associated regulatory submissions with the goal of delivering a disease-specific therapy to patients who are in desperate need.”

ACG-801
ACG-801 is an investigational form of recombinant human acid ceramidase (rhAC) designed to address the genetic deficiency of the naturally occurring enzyme which is the cause of Farber disease and Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA-PME).  Preclinical development for rhAC has been completed, including in vivo proof of concept in a mouse model of Farber disease, and IND-enabling GLP toxicology studies. Data in animal models has also been published supporting the potential of rhAC as a treatment for cystic fibrosis. 

Patients with Farber disease have been involved in the development through a quantitative research study and the natural history study. Data from these studies support the clinical investigation of ACG-801 and significant progress has been made in harmonized regulatory discussions with the FDA and the EMA. ACG-801 has been granted Rare Pediatric Disease and Fast Track designations by the FDA, as well as Orphan Drug designations by the FDA and EMA.

“Breakthrough therapies for patients battling devastating rare diseases often result from extraordinary collaborations between academic discovery teams and dedicated developers like Enzyvant and Aceragen,” said Rachelle Jacques, CEO of Enzyvant.  “We are delighted that Aceragen, with strong capabilities, a commitment equal to our own and a singular focus, will rapidly advance this important therapy to address the significant unmet needs of Farber disease patients and their families.”

Concurrent with the closing of the transaction, NovaQuest’s Managing Partner Ron Wooten and Vice President Stephen Lesser have joined Aceragen’s board of directors.

“We congratulate John and the Aceragen team for identifying this exceptional rare disease opportunity in Farber disease and for choosing NovaQuest’s Product Finance approach among the alternatives,” said Lesser. “It was a pleasure for all of us at NovaQuest to conclude that at-risk, non-dilutive financing is possible as a company-enabling resource for development funding within the realm of ultra-rare disease. NovaQuest believes that opportunities, like ACG-801, offer an attractive area for investment, combining a significant unmet medical need with a treatment that has the potential to deliver a clinically meaningful benefit and improved quality of life for patients.” 

“Our product financing agreement with Aceragen furthers NovaQuest’s core strategy and focus of providing innovative funding for a wide range of biopharma companies and products,” Wooten added. “We are pleased to join the Aceragen board and contribute to the team’s efforts to provide benefits to patients with Farber disease.”

Wedbush Securities acted as strategic financial advisor, Hutchison PLLC was corporate counsel, and Hogan Lovells provided IP counsel to Aceragen. Arnold & Porter was legal counsel to Enzyvant, and Wyrick Robbins Ponton & Yates LLP acted as legal counsel to NovaQuest.

About Farber disease and Acid Ceramidase Deficiency
Farber disease is caused by mutations in the ASAH1 gene, resulting in a deficiency of acid ceramidase, a naturally occurring lysosomal enzyme. The enzyme normally acts to metabolize ceramide, a highly inflammatory and apoptotic lipid. Lack of adequate acid ceramidase function results in accumulation of ceramide and causes a severe inflammatory disease associated with macrophage-filled subcutaneous granulomas, joint damage and deformity, and life-threatening respiratory complications among other severe symptoms. Patients with the most rapidly progressive form of Farber disease usually do not live beyond the first few years of life.  A portion of the patient population also have progressive central nervous system involvement, leading to developmental delay and regression. Acid ceramidase deficiency also causes Spinal Muscular Atrophy with Progressive Myoclonic Epilepsy (SMA-PME) and dysregulation of ceramide metabolism has been implicated in a number of other diseases and conditions, including cystic fibrosis.

About Aceragen
Aceragen is a biopharmaceutical company focused on the development of innovative therapeutics for rare and ultra-rare diseases. The Company is advancing ACG-801 (rhAC) as an investigational enzyme replacement therapy for the treatment of patients with Farber disease and potentially other diseases associated with the dysregulation of ceramide metabolism.  

For more information, please visit www.aceragen.com

About NovaQuest Capital Management
Founded by a team of accomplished industry professionals who began working together in 2000, NovaQuest Capital Management is a premier biopharma and life sciences investment firm. NovaQuest pioneered a Product Finance solution for the industry, providing at-risk, nondilutive funding that enables partner companies to advance pivotal clinical trials, launch new brands, license products, and acquire accretive products or companies. NovaQuest has invested in scores of biopharmaceutical assets across therapeutic areas with a clinical success rate significantly higher than the industry average. Currently managing more than $2.2 billion in capital, NovaQuest is actively investing from the $1.2 billion Pharma Opportunities Fund V, evaluating global opportunities with financing needs that range from $30-100 million.

For more information, please visit www.novaquest.com.

About Enzyvant
Enzyvant, a wholly owned subsidiary of Sumitovant Biopharma Ltd. (wholly owned by Sumitomo Dainippon Pharma Co., Ltd.), is a biotechnology company dedicated to developing novel, transformative regenerative therapies for people with devastating rare diseases. Enzyvant’s lead asset is the investigational tissue-based regenerative therapy, RVT-802, for congenital athymia, an ultra-rare and life-threatening pediatric immunodeficiency. RVT-802 has been granted multiple regulatory designations, including the US Food and Drug Administration designation as a Regenerative Medicine Advanced Therapy (RMAT).

For more information about Enzyvant, visit www.Enzyvant.com. Follow @Enzyvant on Twitter, Facebook and LinkedIn.

Contact:
Aceragen, Inc.
Jtaylor@aceragen.com

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ENZYVANT Resubmits Biologics Licensing Application (BLA) to FDA for RVT-802 for Pediatric Congenital Athymia

CAMBRIDGE, Mass., April 27, 2021 (GLOBE NEWSWIRE) — Enzyvant today announced the resubmission of the Biologics Licensing Application (BLA) to the U.S. Food and Drug Administration (FDA) for RVT-802, cultured human thymus tissue. RVT-802 is a one-time tissue-based regenerative therapy for the treatment of pediatric patients with congenital athymia.

The FDA made several regulatory requests related to Chemistry, Manufacturing and Controls (CMC) in a December 2019 Complete Response Letter (CRL) that followed the initial RVT-802 BLA submission in April 2019. Enzyvant has been working since that time to address each of the FDA requests. The expected action date provided by the FDA under the Prescription Drug User Fee Act (PDUFA) is October 8, 2021.

“Resubmission of the RVT-802 BLA is important progress in the mission to bring a desperately needed treatment option to families battling the dire consequences of congenital athymia,” said Rachelle Jacques, CEO of Enzyvant. “Thank you to everyone involved in the RVT-802 development program, including families who participated in clinical trials, for your efforts to achieve this milestone and make a difference in the lives of families who will face this condition in the future.”

An ultra-rare condition in which children are born without a thymus, congenital athymia can lead to profound immunodeficiency, immune dysregulation and high susceptibility to infections. With only supportive care, patients with congenital athymia typically die from infections or autoimmune manifestations by age two or three. Currently, there are no FDA-approved treatments available for congenital athymia.

“The bleak prospects of children with congenital athymia have driven our work over the past two decades to develop a therapy that will provide a functioning thymic environment and enable patients to fight off fatal infections,” said Louise Markert, M.D., Ph.D., principal investigator for RVT-802 clinical trials and Professor of Pediatrics and Immunology at the Duke University School of Medicine. “Resubmission of the RVT-802 BLA is a reason for families to be optimistic that an approved treatment option for congenital athymia may soon be available.”

About Congenital Athymia
Children with congenital athymia are born without a thymus, making them severely immunodeficient and unable to fight infections. Athymia is initially detected by T-cell deficiency observed in newborn screening for SCID (severe combined immune deficiency), which is now required in all 50 U.S. states. SCID and congenital athymia are both primary immunodeficiency disorders but they are distinct conditions. Congenital athymia is associated with multiple conditions such as complete DiGeorge Anomaly (cDGA), CHARGE syndrome, FOXN1 deficiency, TBX1 gene mutation and diabetic embryopathy. Pediatric congenital athymia is ultra-rare with an estimated incidence in the U.S. of ~17 to 24 live births each year.

About Investigational RVT-802 
Investigational RVT-802 is a novel one-time tissue-based regenerative therapy that has been studied by researchers at Duke University across 10 clinical studies for more than 25 years. Investigational RVT-802 is cultured human thymus tissue for the treatment of pediatric congenital athymia and is implanted in a single surgery. It is designed to establish a functioning thymic environment where T cells can develop the ability to correctly identify and interact with antigens and fight infections. Investigational RVT-802 has been granted multiple FDA designations: Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, Rare Pediatric Disease and Orphan Drug. The European Medicines Agency (EMA) has granted Orphan Drug designations and the Advanced Therapy Medicinal Product (ATMP) designation for RVT-802.

About Enzyvant
Enzyvant, a wholly owned subsidiary of Sumitovant Biopharma Ltd. (wholly owned by Sumitomo Dainippon Pharma Co., Ltd.), is a biotechnology company dedicated to developing novel, transformative regenerative therapies for people with devastating rare diseases. Enzyvant’s lead asset is the investigational tissue-based regenerative therapy, RVT-802, for congenital athymia, an ultra-rare and life-threatening pediatric immunodeficiency. RVT-802 has been granted multiple regulatory designations, including the U.S. Food and Drug Administration designation as a Regenerative Medicine Advanced Therapy (RMAT).

For more information about Enzyvant, visit Enzyvant.com. Follow @Enzyvant on Twitter, Facebook and LinkedIn.

CONTACT
Eliza Schleifstein
917-763-8106
Eliza@schleifsteinpr.com

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Altavant Sciences Finds Nebulized ALTA-2530 Retains Potency and Reaches Target Lung Tissues, Supporting Continued Investigation for Treatment of BOS

Cary, N.C. and Basel, Switzerland – April 26, 2021 – Altavant Sciences, a clinical-stage biopharmaceutical company focused on patient-centric drug development in rare respiratory diseases, presented results of in vitro and in vivo studies for nebulized ALTA-2530, a novel inhaled formulation of recombinant human IL-1 receptor antagonist (rhIL-1Ra) currently in nonclinical development for the treatment of bronchiolitis obliterans syndrome(BOS). These studies demonstrated that ALTA-2530 achieved and maintained pharmacologically relevant exposure levels in lung epithelial lining fluid and distributed to epithelial cells of the lung’s distal airways. Additional studies demonstrated the rhIL-1Ra protein was stable and retained full potency during nebulization. Together, these findings support the further investigation of ALTA-2530 for the treatment of BOS, a life-threatening form of chronic lung allograft dysfunction (CLAD) that leads to fibrosis of the small airways (bronchioles) and eventual partial or total obstruction of the distal airways. 

During the ISHLT virtual poster session, Altavant presented results from studies in animal models that showed that nebulized ALTA-2530 reached the lung epithelial lining fluid at concentrations ~2,500-fold higher than those measured in serum. Further, concentrations in the epithelial lining fluid 24 hours after administration were greater than 29-fold the half maximal inhibitory concentration (IC50), a measure of a drug’s potency. In non-human primates, immunohistochemistry demonstrated ALTA-2530 distributed to the epithelial cells of the distal bronchioles. ALTA-2530 was well tolerated in all acute and repeat-administration toxicity studies performed.

“BOS is a life-threatening complication that sadly develops all too frequently following lung transplantation. Current therapies, including cyclosporine A or tacrolimus that modulate the adaptive immune system following transplant, are not approved for the treatment of BOS. BOS treatment likely requires new mechanisms to address the underlying dysregulation of the innate immune system that is potentially mediated by interleukin-1 (IL-1) signaling” said Steve Wring, Ph.D., Altavant’s VP of R&D and author of the ISHLT poster. “Our findings show that the nebulized ALTA-2530 formulation generates particles of an optimal size for delivery to the small airways of the lung, and that pharmacological levels persist in lung for at least 24 hours. These findings reinforce our confidence in ALTA-2530’s potential to quell the inflammatory cascade in the deep tissues of the lung, where BOS manifests.”

The Altavant poster, Inhalation of IL-1 Receptor Antagonist (ALTA-2530) Achieves Stable and Prolonged Pulmonary Exposure in Nonclinical Studies Supportive of Development for Bronchiolitis Obliterans Syndrome, is available on the Altavant Publications webpage.  

In addition, Altavant provided an update on its clinical program for its lead candidate, rodatristat ethyl, during a non-CME symposium titled: Serotonin Pathobiology in Pulmonary Arterial Hypertension (PAH) and Phase 2b Investigation of the Novel Therapy Rodatristat Ethyl (RE) in WHO Group 1 PAH. Altavant is currently screening patients for enrollment in ELEVATE 2 (NCT identifier: NCT04712669), a Phase 2b study for the treatment of PAH, in the US, Canada, and EU. More information on ELEVATE 2 may be found on the study’s website at elevatepahstudy.com and on the Altavant website. 

About Altavant Sciences
Altavant Sciences is a clinical-stage biopharmaceutical company focused on elevating patient-centric drug development in rare respiratory diseases. Altavant is currently advancing two pipeline candidates: rodatristat ethyl and ALTA-2530. Rodatristat ethyl is in Phase 2 development for patients with pulmonary arterial hypertension. A tryptophan hydroxylase (TPH) inhibitor, rodatristat may play a role in halting or reversing the vascular remodeling associated with PAH, offering a novel treatment option for patients living with this disease. ALTA-2530 is a recombinant human interleukin-1 receptor antagonist under development for chemical lung injury and bronchiolitis obliterans syndrome (BOS), a life-threatening form of chronic lung allograft dysfunction (CLAD) that may present following lung transplantation. ALTA-2530’s unique mechanism of action may offer a novel treatment option for patients who suffer from BOS, a disease where there are currently no approved therapies.

Altavant is a wholly owned subsidiary of Sumitovant Biopharma Ltd. For more information, please visit https://altavant.com

About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sumitovant is the majority shareholder of Myovant, and wholly owns Enzyvant, Spirovant, Urovant and Altavant. Sumitovant’s pipeline is comprised of early- through late-stage investigational medicines across a range of disease areas targeting high unmet need.

Sumitovant Biopharma Ltd. is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. For further information about Sumitovant please visit https://www.sumitovant.com/

About Sumitomo Dainippon Pharma Co., Ltd.
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China and the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com/.

Forward-Looking Statements
This press release contains “forward-looking statements” concerning the development and commercialization of Altavant’s products, the company’s business development efforts and its expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements. These statements are made as of the date of this press release. Actual results may vary. Altavant undertakes no obligation to update any forward-looking statements for any reason.

Contact:

Aline Sherwood
Scienta Communications, LLC
asherwood@scientapr.com

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Altavant Sciences Highlights Pipeline Progress in PAH and BOS at the International Society for Heart & Lung Transplantation Meeting

Cary, N.C. and Basel, Switzerland – April 21, 2021 – Altavant Sciences, a clinical-stage biopharmaceutical company focused on patient-centric drug development in rare respiratory diseases, announced today that the company would be presenting information on its two pipeline candidates, ALTA-2530 and rodatristat ethyl, at the upcoming virtual meeting of the International Society for Heart & Lung Transplantation (ISHLT) being held April 24-28, 2021. 

Results of in vivo studies measuring lung exposure of a novel nebulized formulation of ALTA-2530, a IL-1 receptor antagonist (IL-1Ra) in development for the treatment of bronchiolitis obliterans syndrome (BOS), will be presented in a poster (#936) at ISHLT titled: Inhalation of IL-1 Receptor Antagonist (ALTA-2530) Achieves Stable and Prolonged Pulmonary Exposure in Nonclinical Studies Supportive of Development for Bronchiolitis Obliterans Syndrome. Sustained exposure to the distal regions of the lungs is an important consideration for the efficacious treatment of BOS. Further, treatment via a handheld nebulizer may provide a convenient portable intervention option for lung transplant patients experiencing BOS. Poster 936 will be available on demand from April 24 – July 28, 2021 on the ISHLT virtual platform in the session: “Lung Posters: Pharmacology and Therapeutics” and will be available on the Altavant website following the conference embargo. 

BOS is a life-threatening form of CLAD, commonly observed following lung transplantation. The condition is caused by an unchecked upregulation of pro-inflammatory chemical signals, including interleukin-1 (IL-1), which leads to fibrosis of the small airways (bronchioles) and eventual partial or total obstruction of the airways. ALTA-2530, a recombinant human IL-1Ra, binds competitively to the IL-1 receptor to attenuate the inflammatory response. ALTA-2530 is currently in preclinical development at Altavant for the treatment of BOS and chemical lung injuries.

In addition, Altavant will be hosting a non-CME symposium on April 25, 2021 from 2:30 – 3:30 p.m. Eastern Time, entitled: Serotonin Pathobiology in Pulmonary Arterial Hypertension (PAH) and Phase 2b Investigation of the Novel Therapy Rodatristat Ethyl (RE) in WHO Group 1 PAH. Altavant is currently screening patients for enrollment in a Phase 2b study (NCT identifier: NCT04712669) of rodatristat ethyl for the treatment of PAH, a rare lung disorder driven by excessive production of peripheral serotonin. Rodatristat works by inhibiting the production of peripheral serotonin with the goal of preventing and potentially reversing the arterial remodeling that restricts blood flow in patients with PAH. This mechanism of action will be reviewed during the ISHLT symposium, which may be accessed from the society’s virtual meeting platform.  Investigators interested in learning more about this ongoing Phase 2b study, which includes clinical sites in US, Canada, and Europe, are encouraged to join this presentation.

About Altavant Sciences
Altavant Sciences is a clinical-stage biopharmaceutical company focused on elevating patient-centric drug development in rare respiratory diseases. Altavant is currently advancing two pipeline candidates: rodatristat ethyl and ALTA-2530. Rodatristat, a tryptophan hydroxylase (TPH) inhibitor, may play a role in halting or reversing the vascular remodeling associated with PAH, offering a novel treatment option for patients living with this disease. Rodatristat ethyl is in Phase 2 development for patients with pulmonary arterial hypertension (PAH). ALTA-2530 is a recombinant human interleukin-1 receptor antagonist under development for bronchiolitis obliterans syndrome (BOS), a life-threatening form of chronic lung allograft dysfunction (CLAD) that frequently presents following lung transplantation. ALTA-2530’s unique mechanism of action may offer a novel treatment option for patients who suffer from BOS, a disease where there are currently no approved therapies.

Altavant is a wholly owned subsidiary of Sumitovant Biopharma Ltd. For more information, please visit https://altavant.com

About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. Sumitovant is the majority shareholder of Myovant, and wholly owns Enzyvant, Spirovant, Urovant and Altavant. Sumitovant’s pipeline is comprised of early- through late-stage investigational medicines across a range of disease areas targeting high unmet need.

Sumitovant Biopharma Ltd. is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. For further information about Sumitovant please visit https://www.sumitovant.com/

About Sumitomo Dainippon Pharma Co., Ltd.
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China and the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com/.

Forward-Looking Statements
This press release contains “forward-looking statements” concerning the development and commercialization of Altavant’s products, the company’s business development efforts and its expectations regarding its prospects. Forward-looking statements are subject to risks, assumptions and uncertainties that could cause actual future events or results to differ materially from such statements. These statements are made as of the date of this press release. Actual results may vary. Altavant undertakes no obligation to update any forward-looking statements for any reason.

Contact:

Aline Sherwood
Scienta Communications, LLC
asherwood@scientapr.com

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