Rhythm Pharmaceuticals

Rhythm Pharmaceuticals' Imcivree Shows Significant Weight Loss in Phase III Hypothalamic Obesity Trial

a month ago

• Rhythm Pharmaceuticals' Imcivree (setmelanotide) demonstrated a 19.8% placebo-adjusted reduction in BMI over 52 weeks in patients with acquired hypothalamic obesity, a rare condition caused by hypothalamic damage. • The TRANSCEND Phase III trial met its primary endpoint with 83% of treated patients achieving at least 5% BMI reduction, while placebo patients experienced a 3.3% BMI increase, supporting potential regulatory filings in Q3 2025. • If approved for hypothalamic obesity, analysts project Imcivree could exceed $1 billion in annual sales by 2028, significantly expanding its current indications for certain genetic obesity disorders.

GSK Expands Neurodegenerative Disease Portfolio with $2.5B+ ABL Bio Partnership

a month ago

• GSK has committed to a potential $2.5 billion partnership with South Korea's ABL Bio, providing an upfront payment of £38.5 million to develop novel treatments for neurodegenerative diseases. • The collaboration centers on ABL Bio's proprietary Grabody-B platform, which utilizes bispecific antibodies targeting insulin-like growth factor 1 receptors to facilitate the transport of therapeutic molecules across the blood-brain barrier. • This agreement represents GSK's continued strategic expansion into neuroscience, following its recent $35 million partnership with Muna Therapeutics focused on Alzheimer's disease targets.

Rhythm Pharmaceuticals Regains Global Control of IMCIVREE by Reacquiring Rights in China

2 months ago

• Rhythm Pharmaceuticals has terminated its 2021 licensing agreement with RareStone Group Ltd., reacquiring rights to IMCIVREE (setmelanotide) in mainland China, Hong Kong, and Macau for $6.3 million. • The reacquisition aligns with Rhythm's global strategy as the company prepares for topline data readout from its Phase 3 trial in acquired hypothalamic obesity in Q2 2025, with studies conducted across North America, Europe, and Japan. • IMCIVREE, an MC4R agonist, is currently approved in the US and Europe for treating obesity in patients with specific genetic conditions including Bardet-Biedl syndrome and certain genetic deficiencies.

Setmelanotide Receives Orphan Drug Designation in Japan for Hypothalamic Obesity Treatment

2 months ago

• Rhythm Pharmaceuticals' setmelanotide has received orphan drug designation from Japan's Ministry of Health for the treatment of acquired hypothalamic obesity, a rare condition affecting an estimated 5,000-8,000 patients in Japan. • Setmelanotide, a melanocortin-4 receptor agonist, is currently being evaluated in a global Phase 3 trial with topline data expected in Q2 2025, potentially offering the first therapy targeting the underlying biology of this disease. • The designation follows similar status in Europe, strengthening Rhythm's global strategy to address significant unmet medical needs for patients with hypothalamic obesity caused by brain tumors, traumatic injury, stroke, or inflammation.

Rhythm Pharmaceuticals Announces Positive IMCIVREE® Sales and Pipeline Advancements

4 months ago

• Rhythm Pharmaceuticals reports preliminary unaudited net revenues of approximately $42 million for Q4 2024 and $130 million for full year 2024 from global IMCIVREE® sales. • Topline data from the Phase 3 trial of setmelanotide in acquired hypothalamic obesity (HO) is expected in the first half of 2025. • A new Phase 2 trial exploring setmelanotide in Prader-Willi syndrome (PWS) is planned to commence in the first quarter of 2025. • Enrollment is completed in the Phase 3 EMANATE trial substudies for genetically-caused MC4R pathway diseases, with topline data anticipated in H1 2026.

FDA Expands Setmelanotide Approval to Include Children Aged 2-5 with Rare Obesity Forms

5 months ago

• The FDA has expanded the approval of setmelanotide (Imcivree) to include children aged 2 to 5 years with rare genetic forms of obesity. • The expanded indication is based on the VENTURE phase 3 trial, which demonstrated significant BMI reduction in young children with POMC, LEPR deficiencies, and Bardet-Biedl syndrome. • Setmelanotide is the first precision medicine to target the MC4R pathway, addressing the root cause of hyperphagia and obesity in these rare genetic conditions. • The most common adverse events observed in clinical trials were mild to moderate, including skin hyperpigmentation and injection site reactions.

Rhythm Pharmaceuticals Targets Rare Obesity Disorders with Promising Treatments

5 months ago

Rhythm Pharmaceuticals Inc. is making strides in the treatment of rare obesity disorders, with its drug Imcivree showing significant revenue growth. Oppenheimer analysts are optimistic about the company's future, especially with upcoming Phase 3 trial results for hypothalamic obesity treatment and the potential expansion of Imcivree's uses.

FDA Wraps Up 2024 with Key Approvals for Drugs Targeting Various Conditions

6 months ago

• The FDA approved Vertex's Alyftrek for cystic fibrosis, offering improved dosing and potential market exclusivity. • Novo Nordisk's Alhemo was approved for hemophilia A and B, providing a new option for patients with inhibitors. • Bristol Myers Squibb's Opdivo Qvantig gained approval as a subcutaneous formulation, offering faster administration for various solid tumors. • Eli Lilly's Zepbound secured approval for obstructive sleep apnea in obese adults, marking the first prescription medicine for this condition.

Setmelanotide Shows Promise in Reducing Obesity in Young Children With Rare Genetic Deficiencies

6 months ago

• A Phase 3 trial of setmelanotide in children aged 2-5 years with Bardet-Biedl syndrome or POMC, PCSK1, or LEPR deficiency demonstrated clinically meaningful reductions in BMI Z-score. • 83% of patients achieved a ≥0.2-point reduction in BMI Z-score after 52 weeks, with a mean percent change in BMI from baseline of -18%. • Caregivers reported a 91% reduction in patient hunger compared to baseline, alongside a reduced personal burden. • Setmelanotide was generally well-tolerated, with no new safety signals and common adverse events including skin hyperpigmentation and vomiting.