RHYTHM PHARMACEUTICALS, INC

The FDA expanded Rhythm Pharmaceuticals' obesity drug IMCIVREE's indication to include children as young as 2, targeting MC4R pathway diseases. The approval is based on the drug's ability to reduce excess body weight and maintain long-term weight reduction, though it comes with side effects. Rhythm Pharmaceuticals has seen a stock surge and maintains strong liquidity.

Rhythm Pharmaceuticals announces FDA approval of IMCIVREE for children as young as 2 with syndromic or monogenic obesity due to Bardet-Biedl syndrome or POMC, PCSK1, or LEPR deficiency, targeting hyperphagia and obesity via the MC4R pathway.

Rhythm Pharmaceuticals' IMCIVREE received FDA approval for treating children as young as 2 with rare obesity disorders, targeting the MC4R pathway to address hyperphagia and early-onset obesity, marking a significant advancement in precision medicine for these conditions.

Rhythm Pharmaceuticals announces FDA approval of expanded indication for IMCIVREE® (setmelanotide) to include children as young as 2 years old for syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency. IMCIVREE targets the hypothalamic MC4R pathway, reducing excess body weight and maintaining weight reduction long-term.

The FDA is set to decide on ten drug applications, including Ionis' olezarsen for familial chylomicronemia syndrome, Lexicon's sotagliflozin for type 1 diabetes, AstraZeneca and Daiichi Sankyo's Dato-DXd for non-squamous NSCLC, Zealand Pharma's glepaglutide for short bowel syndrome, Rhythm Pharmaceuticals' Imcivree for genetic obesity in children, Checkpoint Therapeutics' cosibelimab for cutaneous squamous cell carcinoma, Mirum's chenodiol for cerebrotendinous xanthomatosis, Bristol Myers Squibb's subcutaneous Opdivo, and Neurocrine's crinecerfont for congenital adrenal hyperplasia.

Rhythm Pharmaceuticals published Phase 3 VENTURE trial results of setmelanotide in children aged 2-5 with Bardet Biedl syndrome or POMC, PCSK1, or LEPR deficiency, showing 83% reduction in BMI Z-score and 91% reduction in hunger. Setmelanotide generally well-tolerated with no new safety signals. IMCIVREE received EU authorization for BBS or POMC, PCSK1, or LEPR deficiency in children as young as 2. Rhythm submitted an sNDA to the FDA for expanded U.S. label, with a PDUFA goal date of December 26, 2024.

Rhythm Pharmaceuticals published Phase 3 VENTURE trial results of setmelanotide in children aged 2-5 with Bardet Biedl syndrome or POMC, PCSK1, or LEPR deficiency, showing clinically meaningful reductions in hunger and body weight, published in The Lancet Diabetes & Endocrinology.

Pamela J. Cramer sold 9,375 Rhythm Pharmaceuticals shares for $585,940, exercised options for 9,375 shares at $19.02 each, and holds 13,500 shares. Rhythm reported a 48% Q3 revenue increase to $33.3M, reduced 2024 operating expense guidance, and has $298.4M in cash reserves. The company expects top-line Phase III trial data in H1 2025 and is optimistic about completing trial enrollments by year-end. RYTM stock has shown 133.08% total return over the past year, but faces financial challenges with an operating income margin of -235.95%.

Rhythm Pharmaceuticals reports Q3 2024 success with $33.3 million in global net revenue from IMCIVREE, a 48% YoY increase. The company anticipates funding operations into 2026, with plans to complete Phase II trials for bivamelagon and Phase I for RM-718 in early 2025. Rhythm expects to expand IMCIVREE's label to include children ages two to six by December 26, 2024, and forecasts European revenue growth in 2025.