Rhythm Pharmaceuticals Reports Second Quarter 2022 Financial Results and Business Update

-- Received FDA approval for IMCIVREE® (setmelanotide) as the first-ever therapy for patients with Bardet-Biedl syndrome --

-- U.S. launch off to a strong start with more than 50 IMCIVREE prescriptions written for patients with BBS in first six weeks since FDA approval --

-- EMA’s CHMP recommended expansion of IMCIVREE marketing authorization to include treatment of obesity and control of hunger in patients with genetically confirmed BBS; EC approval anticipated in the fourth quarter of 2022 --

-- Announced positive interim results from Phase 2 trial in hypothalamic obesity and plans to initiate Phase 3 trial in early 2023 --

-- Entered into non-dilutive revenue interest financing agreement with Healthcare Royalty Partners for up to $100 million, extending cash runway into second half of 2024 --

-- Management to host conference call today at 8:00 a.m. ET --

BOSTON, Aug. 02, 2022 (GLOBE NEWSWIRE) --  Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) today reported financial results and provided a business update for the second quarter ended June 30, 2022.

“Rhythm is executing on our global mission to transform the lives of patients and families living with hyperphagia and severe obesity caused by rare MC4R pathway diseases,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “Our commercial launch of IMCIVREE® (setmelanotide) in Bardet-Biedl syndrome (BBS) is off to a strong start, and we continue to make meaningful progress towards securing market access in Europe, for both our initial indications and, more recently, BBS.”

Dr. Meeker added, “We are highly encouraged by the interim data from our Phase 2 trial evaluating setmelanotide in hypothalamic obesity and the potential role it may play in transforming how this disease is treated. We look forward to reviewing these data with the U.S. Food and Drug Administration (FDA) and seeking input on a pivotal Phase 3 trial, which we plan to initiate in the first half of 2023. We are excited to add a potential Phase 3 trial in hypothalamic obesity to our broad setmelanotide clinical development program, which also includes the ongoing Phase 3 EMANATE trial, Phase 2 DAYBREAK trial, Phase 3 switch study evaluating a weekly formulation of setmelanotide and our Phase 3 pediatrics trial, which has completed enrollment.”

Second Quarter and Recent Business Highlights:

Regulatory and Commercial Updates: Bardet-Biedl Syndrome:

• Today, Rhythm announced that more than 35 physicians have written more than 50 prescriptions for IMCIVREE for patients with BBS in the first six weeks since IMCIVREE was approved by the FDA on June 16, 2022 for chronic weight management in adult and pediatric patients 6 years of age and older with obesity due to BBS.
• In July 2022, Rhythm announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending to expand the current indication for IMCIVREE to include the treatment of obesity and control of hunger in adult and pediatric patients 6 years of age and older with genetically confirmed BBS.
• In July 2022, Rhythm announced that the French National Agency for Medicines and Health Products Safety (ANSM) and Haute Autorité de santé (HAS) granted post-marketing authorization AP1 (autorisation d’accès précoce -- early access authorization) for setmelanotide for the treatment of obesity and control of hunger in patients with genetically confirmed BBS. AP1 allows for early access to innovative therapies in France prior to regulatory approval when a positive benefit/risk ratio is recognized and when no other therapeutic alternatives are available.

POMC and LEPR Deficiency Obesities:

• In July 2022, Rhythm announced that the National Institute for Health and Care Excellence (NICE) issued guidance recommending IMCIVREE as an option for treating obesity and controlling hunger caused by POMC, PCSK1 or LEPR deficiency in people 6 years of age or older. With this recommendation under the Highly Specialised Technologies (HST) pathway, IMCIVREE will be funded and available for use within 90 days in the National Health Service.
• In June 2022, Rhythm launched IMCIVREE in Germany for patients with POMC, PCSK1 or LEPR deficiency obesity. IMCIVREE is the first precision medicine designed to induce weight loss and control of hunger that has received a lifestyle exemption from the German Federal Joint Committee (G-BA).
• Also in June 2022, Rhythm announced that the European Commission (EC) authorized a variation for IMCIVREE that allows for dosing in patients aged 6 years or older with POMC or LEPR deficiency who have mild, moderate or severe renal impairment.

Clinical Development Updates:

• Today, Rhythm announced it completed enrollment in the Phase 3, open-label trial evaluating one year of setmelanotide therapy in pediatric patients with MC4R pathway deficiencies between the ages of 2 and 6 years old.
• In July 2022, Rhythm announced positive interim results from the Phase 2 study in patients with hypothalamic obesity. All 11 evaluable patients achieved the primary endpoint of at least 5% reduction in body mass index (BMI) (P