Rhythm Pharmaceuticals has appointed Manher "AJ" Joshi as Chief Medical Officer, effective 17 August. Dr Joshi brings over 20 years of experience spanning drug discovery, pivotal trials, regulatory processes and lifecycle management. Dr Joshi joins from Soleno Therapeutics, where he served as Chief Development Officer. He previously held the Chief Medical Officer position at Neurona Therapeutics and executive roles at Atara Biotherapeutics. His career also includes leadership positions at Allergan Medical, Synageva BioPharma, and Genzyme. Rhythm is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases. Its lead asset, IMCIVREE (setmelanotide), is approved in the US and Europe for treating certain forms of obesity. The company is advancing next-generation MC4R agonists through late-stage development.

Rhythm Pharmaceuticals announced that the UK's Medicines and Healthcare products Regulatory Agency has expanded the marketing authorisation for IMCIVREE (setmelanotide) to include treatment of obesity and control of hunger in adults and children aged 4 and older with acquired hypothalamic obesity due to hypothalamic injury or impairment. The MHRA also granted IMCIVREE orphan drug designation for acquired HO. The approval is based on results from the Phase 3 TRANSCEND trial, which met its primary endpoint with a statistically significant 19.8% placebo-adjusted reduction in body mass index. The global trial enrolled 120 patients. IMCIVREE initially received marketing authorisation in the UK in 2021 for treatment of obesity in patients with POMC, PCSK1 or LEPR deficiency, with subsequent expansions for other rare obesity conditions.

Rhythm Pharmaceuticals reported preliminary results showing its experimental weekly injectable drug RM-718 helped reduce body weight in patients with acquired hypothalamic obesity, a rare condition caused by brain damage from tumour surgery or treatment. The Boston-based company's shares rose 5% in premarket trading. Among 11 enrolled patients, seven who completed 16 weeks of treatment saw their body mass index fall by an average of 11.6%. The drug works by activating the MC4R brain receptor, which controls hunger and metabolism. Two patients discontinued treatment due to side effects including nausea and injection site problems. Rhythm also sells setmelanotide for the same condition under the brand name Imcivree. Separately, the company reported second-quarter revenue of $71.3 million, up 46.9% from $48.5 million a year earlier.