Quoin Pharmaceuticals (NASDAQ: QNRX) announced Friday August 28, 2026 a securities purchase agreement with new and existing healthcare-focused institutional investors (Sirenia Capital Management LP, Sphera Healthcare, AIGH Capital Management, Nantahala Capital, StemPoint Capital LP, and Stonepine Capital Management among others) for up to approximately $50.0 million in gross proceeds. Structure: approximately $30.8 million in initial upfront funding plus up to approximately $19.2 million from cash exercise of accompanying ordinary warrants. The private placement is expected to close on or about August 31, 2026. Proceeds will fund general corporate purposes including completion of clinical development of QRX003 (topical serine protease inhibitor) for Netherton Syndrome (a rare severe congenital ichthyosis caused by SPINK5 mutations that leaves affected patients with a defective skin barrier and life-threatening infections in infancy). Aggregate net proceeds (assuming full warrant exercise) are expected to fund Quoin into H2 2029.
Regeneron Pharmaceuticals (NASDAQ: REGN) received FDA approval Thursday August 20, 2026 for Pasatru (garetosmab-grts, an anti-activin A monoclonal antibody administered as subcutaneous injection) for the reduction of the formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP). FOP is a rare autosomal-dominant genetic disease affecting roughly 1 in 2 million people globally (approximately 800 patients in the US) in which skeletal muscle and connective tissue progressively turn into bone through extra-skeletal ossification triggered by activin A signaling through mutated ACVR1 (activin receptor A type 1) receptors. Patients typically develop the first flare-ups in early childhood, with progressive immobilization by adulthood as ossification advances across major joints. Pasatru's mechanism: garetosmab binds activin A and blocks its signaling through the mutated ACVR1 receptors, reducing the flare-up frequency and slowing new HO lesion formation. The approval marks the second FDA-approved therapy for FOP after Ipsen's Sohonos (palovarotene, a retinoic acid receptor gamma agonist small molecule) approved in 2023. Pasatru offers a mechanistically distinct alternative for patients who cannot tolerate palovarotene or who need combination or sequential therapy. The Pasatru approval extends Regeneron's growing rare-disease commercial portfolio alongside Eylea (aflibercept for wet AMD), Dupixent (dupilumab for atopic dermatitis and asthma), and multiple antibody-drug conjugate programs in development. Pricing and launch details have not been publicly disclosed but rare-disease pricing typically runs $300,000-$500,000 per patient per year.
Amylyx Pharmaceuticals on May 5 launched a U.S. Expanded Access Program (EAP) for up to 250 adults with post-bariatric hypoglycemia (PBH) following Roux-en-Y gastric bypass surgery. The investigational compound, avexitide, is a first-in-class peptide GLP-1 *receptor antagonist* — designed to bind GLP-1 receptors on pancreatic islet beta cells and block the exaggerated GLP-1-driven insulin response characteristic of PBH, reducing inappropriate insulin secretion and stabilizing blood glucose. Avexitide carries FDA Breakthrough Therapy Designation for both PBH and congenital hyperinsulinism, plus Orphan Drug Designation. Topline data from the pivotal Phase 3 LUCIDITY trial are anticipated in Q3 2026. The program inverts the standard GLP-1 narrative — for this population, GLP-1 signaling is the problem.
Rhythm Pharmaceuticals reported Q1 2026 results May 5: setmelanotide (IMCIVREE) net product revenue of $60.1M, up from $37.7M a year earlier ($36.9M US, $23.2M ex-US), driven by 150+ new US start forms in acquired hypothalamic obesity following the FDA approval and the EU Marketing Authorization for the same indication; Japan's PMDA accepted the NDA for review. The MC4R-agonist peptide is the only commercial therapy for hypothalamic obesity. Offsetting the topline beat: the Phase 3 EMANATE trial in genetically caused MC4R-pathway diseases failed its primary endpoint in all four independent substudies, narrowing the indication-expansion runway. Net loss was $56.7M; cash $340.6M for 24+ months runway.