Sumitovant Biopharma Highlights Significant Clinical, Regulatory and Commercial Achievements Across its Portfolio of Companies in the Second Quarter of FY2021

LONDON and NEW YORK, Dec. 10, 2021 /PRNewswire/ -- Sumitovant Biopharma, Inc. today announced that its portfolio of four wholly-owned subsidiary companies (Urovant, Enzyvant, Altavant and Spirovant) and Myovant (NYSE: MYOV), a publicly listed company that is majority-owned by Sumitovant, achieved significant clinical, regulatory and commercial milestones during the company's second quarter of fiscal year 2021 ending in October.

"The second quarter of 2021 was marked by progress on significant regulatory milestones. Enzyvant gained FDA approval of RETHYMIC^® for pediatric patients with congenital athymia, a rare immune disorder. Also, majority-owned Myovant received FDA acceptance of MYFEMBREE^®'s supplemental New Drug Application in pain associated with endometriosis," said Myrtle Potter, CEO of Sumitovant Biopharma. "In addition, Sumitovant companies also made significant clinical contributions to the scientific community, with key data presentations at prominent medical meetings which continue to underscore the therapeutic potential of our collective pipeline in a variety of therapeutic areas with high unmet needs."

Milestone Highlights

Enzyvant

On October 8, Enzyvant announced the U.S. Food and Drug Administration (FDA) approved RETHYMIC^® (allogeneic processed thymus tissue-agdc), a one-time regenerative tissue-based therapy for immune reconstitution in pediatric patients with congenital athymia.

Myovant

On September 9, Myovant and its partner, Pfizer, announced that the U.S. Food and Drug Administration (FDA) accepted for review a supplemental New Drug Application (sNDA) for MYFEMBREE^® (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) for the investigational use of the management of moderate to severe pain associated with endometriosis. The FDA set a target action date of May 6, 2022 for the sNDA under the Prescription Drug User Fee Act (PDUFA).

Additional Highlights

Myovant

On October 19, Myovant and its partner, Pfizer, presented data on relugolix combination therapy (relugolix 40 mg plus estradiol 1.0 mg and norethindrone acetate 0.5 mg) from clinical studies in uterine fibroids and endometriosis at the American Society for Reproductive Medicine (ASRM) 2021 Congress. The presentations included two-year efficacy and safety data in women with heavy menstrual bleeding associated with uterine fibroids and pooled safety and tolerability data.  

In August, the FDA informed Myovant that the partial clinical hold for the Phase 3 SERENE study evaluating relugolix combination therapy for the prevention of pregnancy was lifted following study protocol amendments. The amended SERENE study protocol will evaluate the contraceptive efficacy of relugolix-CT in women with heavy menstrual bleeding associated with uterine fibroids or endometriosis-associated pain who are 18 to 50 years of age and at risk for pregnancy, and includes bone mineral density monitoring for patients during and after treatment. Patient screening with this updated protocol began in September 2021, with initial patients dosed in October 2021.

Urovant

On September 13, Urovant presented at the 2021 American Urologic Association (AUA) annual meeting new data from a dedicated ambulatory blood pressure study showing that vibegron 75mg in overactive bladder patients was not associated with statistically significant or clinically meaningful effects on blood pressure or heart rate. In addition to this presentation, further data presented at AUA included a review of the efficacy of vibegron in the OAB dry population from the EMPOWUR phase 3 pivotal trial. Urovant also presented data collected from a Director of Nursing population related to the impact of overactive bladder on residents and caregivers in the long-term care community. These data highlights were presented at the National Association of Directors of Nursing Administration (NADONA) and the Gerontological Advanced Practice Nurses Association (GAPNA) annual meetings in September 2021.

Altavant

On September 13, Altavant presented an e-poster at the European Respiratory Society annual congress highlighting the protocol design of the ongoing double-blind, placebo-controlled ELEVATE 2 (NCI NCT04712669) Phase 2b study of rodatristat ethyl (rodatristat) in patients with pulmonary arterial hypertension (PAH).

Enzyvant

On August 26, Enzyvant announced data from two first-ever studies revealing extreme clinical, emotional, social, and financial burdens on pediatric patients with congenital athymia and their families, and extraordinary costs of supportive care to healthcare systems. Posters of each of the studies were presented at the Clinical Immunology Society 2021 Annual Meeting.

On August 4, Enzyvant announced results from clinical trials of the Company's investigational RVT-802 (allogeneic processed thymus tissue-agdc), engineered human thymus tissue implanted in 105 pediatric patients, published in The Journal of Allergy and Clinical Immunology.

Spirovant

On October 28, Spirovant announced plans to deliver oral and poster presentations highlighting pre-clinical data for its compound, SP-101, an investigational novel recombinant adeno-associated virus (AAV) gene therapy selected for its tropism to human airway epithelia, at the 2021 North American Cystic Fibrosis Conference in November.

About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company leveraging data-driven insights to rapidly accelerate development of new potential therapies for unmet patient conditions. Through our unique portfolio of companies—wholly-owned Urovant, Enzyvant, Spirovant, Altavant, plus majority-owned Myovant (NYSE: MYOV)—and use of embedded computational technology platforms to generate business and scientific insights, Sumitovant has supported the development of FDA-approved products and advanced a promising pipeline of early-through late-stage investigational assets for other serious conditions. Sumitovant is a wholly-owned subsidiary of Sumitomo Dainippon Pharma. For more information, please visit our website at www.sumitovant.com or follow us 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 Japan, the U.S., China, and other Asian countries. 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 7,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com.

About Urovant Sciences
Urovant is a biopharmaceutical company focused on developing and commercializing innovative therapies for areas of unmet need, with a dedicated focus in Urology. The Company's lead product, GEMTESA^®(vibegron), is an oral, once-daily (75 mg) small molecule beta-3 agonist for the treatment of adult patients with overactive bladder (OAB) with symptoms of urge urinary incontinence, urgency, and urinary frequency. GEMTESA was approved by the U.S. FDA in December 2020 and launched in the U.S. in April 2021. GEMTESA is also being evaluated for the treatment of OAB in men with benign prostatic hyperplasia. 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 wholly-owned subsidiary of Sumitovant Biopharma Ltd., intends to bring innovation to patients in need in urology and other areas of unmet need. Learn more about us at www.urovant.com

About GEMTESA^®

GEMTESA is a prescription medicine for adults used to treat the following symptoms due to a condition called overactive bladder:

• urge urinary incontinence: a strong need to urinate with leaking or wetting accidents
• urgency: the need to urinate right away
• frequency: urinating often

It is not known if GEMTESA is safe and effective in children.

IMPORTANT SAFETY INFORMATION
Do not take GEMTESA if you are allergic to vibegron or any of the ingredients in GEMTESA. Before you take GEMTESA, tell your doctor about all your medical conditions, including if you have liver problems; have kidney problems; have trouble emptying your bladder or you have a weak urine stream; take medicines that contain digoxin; are pregnant or plan to become pregnant (it is not known if GEMTESA will harm your unborn baby; talk to your doctor if you are pregnant or plan to become pregnant); are breastfeeding or plan to breastfeed (it is not known if GEMTESA passes into your breast milk; talk to your doctor about the best way to feed your baby if you take GEMTESA).

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

What are the possible side effects of GEMTESA?
GEMTESA may cause serious side effects including the inability to empty your bladder (urinary retention). GEMTESA may increase your chances of not being able to empty your bladder, especially if you have bladder outlet obstruction or take other medicines for treatment of overactive bladder. Tell your doctor right away if you are unable to empty your bladder.
The most common side effects of GEMTESA include headache, urinary tract infection, nasal congestion, sore throat or runny nose, diarrhea, nausea, and upper respiratory tract infection. These are not all the possible side effects of GEMTESA. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please click here for full Product Information for GEMTESA.

About Myovant Sciences
Myovant aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. Founded in 2016, Myovant has executed five successful Phase 3 clinical trials across oncology and women's health leading to two regulatory approvals by the U.S. Food and Drug Administration for men with advanced prostate cancer and women with heavy menstrual bleeding associated with uterine fibroids, respectively, as well as a European Commission approval for women with symptomatic uterine fibroids. Additionally, Myovant has two regulatory submissions under review, a Marketing Authorization Application in advanced prostate cancer and a supplemental New Drug Application in endometriosis-associated pain. Myovant is conducting a Phase 3 study to evaluate the prevention of pregnancy in women with uterine fibroids or endometriosis. Myovant is also developing MVT-602, an investigational 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 www.myovant.com. Follow @Myovant on Twitter and LinkedIn.

About MYFEMBREE®
MYFEMBREE (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) is the first once-daily oral 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 worsens. In 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.
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 July 28, 2021, as such risk factors may be amended, supplemented or superseded from time to time.

About Enzyvant Therapeutics
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 RETHYMIC^® (previously RVT-802) is a one-time tissue-based regenerative therapy approved by the U.S. Food and Drug Administration for pediatric congenital athymia, an ultra-rare and life-threatening pediatric immunodeficiency. For more information about Enzyvant, visit Enzyvant.com. Follow @Enzyvant on Twitter, Facebook, and LinkedIn.

About RETHYMIC
RETHYMIC (allogeneic processed thymus tissue-agdc) is a novel one-time tissue-based regenerative therapy used for immune reconstitution in pediatric patients with congenital athymia. RETHYMIC is engineered human thymus tissue designed to regenerate the thymic function children with congenital athymia are missing and does not require donor-recipient matching. RETHYMIC has been studied across 10 clinical trials for more than 25 years and was granted multiple U.S. Food and Drug Administration (FDA) designations including Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, Rare Pediatric Disease, and Orphan Drug. It also has been granted the Orphan Drug designation and the Advanced Therapy Medicinal Product (ATMP) designation by the European Medicines Agency (EMA). RETHYMIC is the first and only treatment approved by the FDA for immune reconstitution in pediatric patients with congenital athymia. Please see full prescribing information.

Important Safety Information (ISI)

Indication
RETHYMIC® (allogeneic processed thymus tissue–agdc) is indicated for immune reconstitution in pediatric patients with congenital athymia.

Limitations of Use
RETHYMIC is not indicated for the treatment of patients with severe combined immunodeficiency (SCID).

Important Safety Information
Immune reconstitution sufficient to protect from infection is unlikely to develop prior to 6-12 months after treatment with RETHYMIC. Given the immunocompromised condition of athymic patients, follow infection control measures until the development of thymic function is established as measured through flow cytometry. Monitor patients closely for signs of infection including fever. If a fever develops, assess the patient by blood and other cultures and treat with antimicrobials as clinically indicated. Patients should be maintained on immunoglobulin replacement therapy until specified criteria are met, and two months after stopping, IgG trough level should be checked. Prior to and after treatment with RETHYMIC, patients should be maintained on Pneumocystis jiroveci pneumonia prophylaxis until specified criteria are met.

RETHYMIC may cause or exacerbate pre-existing graft versus host disease (GVHD). Monitor and treat patients at risk for the development of GVHD. Risk factors for GVHD include atypical complete DiGeorge anomaly phenotype, prior HCT and maternal engraftment. GVHD may manifest as fever, rash, lymphadenopathy, elevated bilirubin and liver enzymes, enteritis, and/or diarrhea.

Treatment with RETHYMIC may increase the risk of autoimmune-mediated conditions. These events included: thrombocytopenia, neutropenia, proteinuria, hemolytic anemia, alopecia, hypothyroidism, autoimmune hepatitis, autoimmune arthritis, transverse myelitis, albinism, hyperthyroidism, and ovarian failure. Monitor for the development of autoimmune disorders, including complete blood counts with differential, liver enzymes, serum creatinine, urinalysis, and thyroid function.

Pre-existing renal impairment is a risk factor for death.

In the clinical studies of RETHYMIC, 3 out of 4 patients with pre-existing cytomegalovirus infection died. The benefits/risks of treatment should be considered prior to treating patients with pre-existing CMV infection.
Because of the underlying immune deficiency, patients who receive RETHYMIC may be at risk of developing post-treatment lymphoproliferative disorder. Patients should be monitored for the development of lymphoproliferative disorder.

Transmission of infectious disease may occur because RETHYMIC is derived from human tissue and because product manufacturing includes porcine- and bovine-derived reagents.

Immunizations should not be administered in patients who have received RETHYMIC until immune-function criteria have been met.

All patients should be screened for anti-HLA antibodies prior to receiving RETHYMIC. Patients testing positive for anti-HLA antibodies should receive RETHYMIC from a donor who does not express those HLA alleles. HLA matching is required in patients who have received a prior hematopoietic cell transplantation (HCT) or a solid organ transplant. Patients who have received a prior HCT are at increased risk of developing GVHD after RETHYMIC if the HCT donor did not fully match the recipient.

Of the 105 patients in clinical studies, 29 patients died, including 23 deaths in the first year (< 365 days) after implantation.

The most common (>10%) adverse events related to RETHYMIC included: hypertension, cytokine release syndrome, rash, hypomagnesemia renal impairment / failure thrombocytopenia, and graft versus host disease. 

To report suspected adverse reactions, please contact the FDA at 1-800-FDA-1088 or https://www.fda.gov/safety/medwatch.

About Spirovant Sciences
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 Altavant Sciences
Altavant 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 ethyl is a tryptophan hydroxylase (TPH) inhibitor in Phase 2 development for patients with pulmonary arterial hypertension. By reducing serotonin production via TPH inhibition rodatristat ethyl 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. 

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