While Spruce Biosciences Inc.’s tildacerfont missed its phase II primary endpoint in classic congenital adrenal hyperplasia (CAH) last March, the U.S. FDA has now approved Crenessity (crinecerfont) from Neurocrine Biosciences Inc. for treating pediatric and adult CAH patients. The nonsteroidal corticotropin-releasing factor type 1 receptor antagonist had a priority NDA along with fast track, breakthrough therapy and orphan drug designations. Crenessity is the first new treatment in 70 years to treat classic CAH, a rare disease that affects the adrenal glands. With the approval, H.C. Wainwright analysts said the clean label and high unmet need could result in a blockbuster.

Checkpoint cleared: FDA approves Unloxcyt in skin cancer

A year after manufacturing issues resulted in a complete response letter for cosibelimab, the U.S. FDA approved Checkpoint Therapeutics Inc.’s drug, branded Unloxcyt, as the first anti-PD-L1 antibody for use in metastatic or locally advanced cutaneous squamous cell carcinoma patients who are not candidates for curative surgery or curative radiation.

Newron stock soars on €117M EA Pharma deal for schizophrenia drug

Newron Pharmaceuticals SpA scored €44 million (US$46.26 million) up front in a potential €117 million licensing deal with EA Pharma Co. Ltd. to pad the clinical runway of its late-stage oral schizophrenia asset, evenamide (NW-3509), sending company stock prices up near 20%. The agreement announced Dec. 13 grants EA Pharma rights to develop and commercialize evenamide in Japan and other Asian territories, including Brunei, Cambodia, Indonesia, Laos, Malaysia, Myanmar, Philippines, Singapore, Thailand and Vietnam.

Puretech’s modified pirfenidone impresses in phase IIb IPF trial

Puretech Health plc has announced phase IIb data showing its reformulation of pirfenidone has improved the tolerability and increased the efficacy of the marketed antifibrotic in the treatment of idiopathic pulmonary fibrosis. The tweak on the original molecule involved substituting three hydrogen atoms with three of the heavier counterpart, deuterium, improving stability, whilst retaining the antifibrotic effect. “These changes are designed to retain the pharmacology and clinically validated efficacy of the molecule, but with a differentiated pharmacokinetic and tolerability profile. I’m delighted to share that [we] clearly demonstrated this favorable profile in our phase IIb trial,” Bharatt Chowrira, Puretech CEO told analysts on a call held to discuss the data.

Recce moves to pivotal trials in Indonesia for diabetic foot ulcers

Recce Pharmaceuticals Ltd. is progressing synthetic antibiotic Recce-327 to pivotal phase III trials in Indonesia for the treatment of diabetic foot infections. The trial, which is assessing Recce-327 as a topical gel (R327G), is expected to begin in the first quarter of 2025, Recce CEO James Graham told BioWorld. “What is unique about Indonesia is that it is one of the world's largest diabetes patient populations. Indonesia’s population is about 280 million, and 11% have diabetes,” he said.

November’s 10 billion-dollar deals cap 2024 as record year

Biopharma deal activity in November reached $29.18 billion, up from $22.29 billion in October. Year-to-date deal values have increased 3.6%, from $194.13 billion in the first 11 months of 2023 to $201.19 billion through November this year, overtaking 2023 to be the highest year on record through the month.

Also in the news

Acoramidis, Actimed, Akari, Aptevo, ARS, Ascendis, Assertio, Bicycle, Boundless, Candel, Corcept, Crinetics, EA Pharma, Editas Medicine, Elevation Oncology, Eli Lilly, Esperion, Fairjourney, Fibrogen, Galderma, Geron, Gilead, Incyclix, Inovio, Iteos, Jaguar, Kalvista, Kineta, Lakeshore, Merck, Neophore, Neural, Newron, Nintx, Palisade, Repare, Roche, Sanofi, Tuhura, Vectory, Xilio