PERTH, Australia – Neuren Pharmaceuticals Ltd.’s stock nearly doubled on positive top-line phase III results for trofinetide in Rett syndrome, meeting all primary and secondary endpoints in the pivotal phase III trial run by partner Acadia Pharmaceuticals Inc.
LONDON – Sosei Heptares is to get $100 million up front in a potential $2.6 billion deal with Neurocrine Biosciences Inc., in which the pair will develop muscarinic receptor agonists in the treatment of schizophrenia, dementia and other neuropsychiatric disorders. San Diego-based Neurocrine is taking rights to a portfolio of small molecules against specific subtypes of the muscarinic receptor family, targeting M1 and M4 alone, plus a dual M1/M4 agonist.
PERTH, Australia – Australian researchers have debunked previous research that suggests autism spectrum disorder behavior may be caused by differences in the composition of the gut microbiota.
Investigators at Northwestern University’s Feinberg School of Medicine have used a new mouse model of Parkinson’s disease to confirm a causal role for mitochondrial dysfunction in Parkinson’s disease. More surprisingly, the same model has called into question previously uncontroversial notions about the motor features that are PD’s most conspicuous feature.
Zai Lab Ltd. and Blueprint Medicines Corp. have inked an exclusive collaboration and license agreement for the development and commercialization of two candidates for the potential treatment of patients with EGFR-driven non-small-cell lung cancer (NSCLC) in greater China, where Clarivate forecasts total NSCLC market sales are poised to grow from $3.2 billion in 2020 to $9.5 billion in 2030.
Aculys Pharma Inc. closed its $60 million series A financing round, with the funds to be used to develop pitolisant (Wakix), a selective histamine H3 receptor antagonist/inverse agonist, in Japan.
Cerecin Inc. has raised $40 million in an oversubscribed round of financing, paving the way for a potential listing in South Korea. Proceeds of the financing will fund the expansion of the company’s current studies and support the planning and initiation of a global phase III study of its lead candidate, tricaprilin, in Alzheimer’s disease.
PERTH, Australia – After meeting with the FDA, Medlab Clinical Ltd. is reformulating its cannabinoid pain product into a synthetic formulation because the agency indicated it was more likely to register a synthetic compared to a botanic.
Sumitomo Dainippon Pharma Co. Ltd., its U.S.-based subsidiary Sunovion Pharmaceuticals Inc. and Otsuka Pharmaceutical Co. Ltd. have inked a global collaboration agreement to jointly advance four new compounds for serious neuropsychiatric conditions. Sunovion granted Otsuka rights to collaboratively develop and commercialize the four compounds worldwide. In return, Sunovion will receive an up-front payment of $270 million and up to $620 million in development milestone payments for the agents, with the potential for more depending on additional indications. It could also receive sales milestones from Otsuka.
Just months after the controversial FDA approval of Alzheimer’s disease drug Aduhelm (aducanumab), partners on that medicine, Eisai Co. Ltd. and Biogen Inc., are advancing a BLA for another possible AD therapy, lecanemab. Formerly known as BAN-2401, the Bioarctic AB-originated antibody is designed to neutralize and eliminate soluble, toxic amyloid beta for the treatment of early AD. The rolling submission, in pursuit of an accelerated approval, was primarily based on data from a phase IIb trial in people with early AD and confirmed amyloid pathology.