Maplight Therapeutics Inc. is betting that the next competitive battleground in schizophrenia will be less about whether muscarinic agonists work and more about whether patients can stay on them and whether they offer benefits beyond psychosis.
Otsuka Pharmaceutical Co. Ltd. will likely look next to expand the label of Simtriyo (centanafadine) into patients with comorbid anxiety, having won the U.S. FDA’s green light as a once-daily extended-release capsule for attention deficit hyperactivity disorder (ADHD) in adults plus pediatrics age 6 years and older who weigh at least 20 kg (about 44 lbs.).
With its stock price falling as much as 70% July 27, Capricor Therapeutics Inc. took the rare step of issuing a news release to explain the negative tone of the U.S. FDA’s briefing document for the July 29 Cellular, Tissue and Gene Therapies Advisory Committee meeting to discuss the company’s BLA for deramiocel to treat cardiomyopathy in Duchenne muscular dystrophy.
In a rare event for the European biotech sector, Dutch/Belgian immunology specialist Argenx SE is to acquire a U.S. counterpart, Forte Biosciences Inc., in an all cash deal valued at $2.2 billion. The price of $77 per share is a premium of about 86% to Forte’s volume-weighted average price since it reported positive phase Ib data for the lead program FB-102 in vitiligo on July 9.
Sanofi SA will not seek approval of amlitelimab in atopic dermatitis (AD) as previously planned, stating that the OX40-ligand monoclonal antibody “would not represent a meaningful improvement to the standard of care” for patients with moderate to severe disease. The company said the decision, which marks an about-face as Sanofi had been looking to amlitelimab as a possible successor to its Regeneron Pharmaceuticals Inc.-partnered blockbuster AD drug Dupixent (dupilumab), comes as part of an ongoing assessment of the pipeline.
Medtronic plc’s recent launch of the Viaverte basivertebral nerve ablation system and completion of the acquisition of SPR Therapeutics Inc. broadens its chronic pain offering and strengthens its ability to treat patients across the pain care continuum.
Continuing the year’s strong IPO activity, Scribe Therapeutics Inc. priced an upsized offering at the high end of its price range, offering 8.6 million shares at $15 apiece for gross proceeds of about $129 million. The company, which is developing CRISPR-based therapies targeting cardiovascular and metabolic diseases, began trading on Nasdaq under the ticker SCTX, with shares getting a warm welcome, opening at $25 and ending the day at $21.65, up 44.3%.
Zevra Therapeutics Inc. will try to follow in the footsteps of Acadia Pharmaceuticals Inc. with Rett syndrome drug Daybu (trofinetide) by asking the EMA’s Committee for Medicinal Products for Human Use (CHMP) to rethink arimoclomol for Niemann-Pick disease type C (NPC). Boston-based Zevra will request a second look at the MAA after CHMP gatekeepers ruled that efficacy in NPC has not been sufficiently proven.
After a decade of research, the NIH 4D Nucleome program on July 23 delivered a wealth of new insights into how spatial changes in chromatin structure and DNA methylation regulate gene expression in health and disease. In a collection of eight papers published in Science and Science Advances, researchers describe how they have applied single-cell level multi-omics data and tools and techniques developed in the first part of the 4D Nucleome program to track changes in the noncoding part of the genome that are involved in heart disease, Alzheimer’s disease, brain aging, retinal health and immune cell development.