Skyhawk Therapeutics Inc. signed on to work with Ipsen SA in a neurological disease-focused deal worth potentially $1.8 billion to discover and develop novel small molecules that modulate RNA. The deal is an option agreement for exclusive global rights to two candidates. Once the parties have identified and validated those candidates, Paris-based Ipsen will take on all further development and commercialization.
Newco Outrun Therapeutics Ltd. has raised $10 million in a seed round to develop small-molecule E3 ubiquitin ligase inhibitors that prevent programmed protein degradation. The lead program targeting an E3 ligase that tags a tumor suppressor protein for destruction will now advance to preclinical development.
Evotec SE and Variant Bio Inc. have signed a collaboration agreement to identify treatments for diseases caused by fibrosis. The partnership leverages Variant Bio’s genomic discovery capabilities and VB-Inference platform as well as Evotec’s expertise in antifibrotic drug discovery.
South Korea’s SK Biopharmaceuticals Co. Ltd. and Shanghai-based Ignis Therapeutic Co. Ltd. signed a ₩804 billion (US$58 million) licensing deal on April 18, granting the latter global rights to a non-narcotic pain treatment candidate dubbed SKL-22544. A sodium channel blocker, SKL-22544 is in late discovery.
Newco Outrun Therapeutics Ltd. has raised $10 million in a seed round to develop small-molecule E3 ubiquitin ligase inhibitors that prevent programmed protein degradation. The lead program targeting an E3 ligase that tags a tumor suppressor protein for destruction will now advance to preclinical development.
Cerevel Therapeutics Inc.’s positive results from the long-shot pivotal phase III Tempo-3 trial with tavapadon – the first D1/D5 receptor partial agonist being studied as a once-daily treatment for Parkinson’s disease – added gravy to the $8.7 billion takeover by Abbvie Inc., disclosed late last year. The buyout’s centerpiece was the late-stage asset emraclidine, a positive allosteric modulator of the muscarinic M4 receptor, touted as a potential best-in-class, next-generation antipsychotic for schizophrenia, which strikes more than 5 million people in the G7 (U.S., France, Germany, Italy, Spain, U.K. and Japan).
Nxera Pharma Co. Ltd. announced that its partner, Neurocrine Biosciences Inc., is advancing NBI-1117568 to phase II trials for treatment of schizophrenia and other neuropsychiatric disorders.
With no drugs approved by the U.S. FDA for treating Becker muscular dystrophy, Edgewise Therapeutics Inc. reported positive two-year, phase Ib data looking at patients’ ability to physically function, plus biomarker data.
Preclinical data of rabbits having convulsions has prompted the U.S. FDA to place a clinical hold on Neumora Therapeutics Inc.’s phase I study of NMRA-266 in healthy adults. Neumora said about 30 participants had been dosed so far in the single ascending and multiple ascending dose study, with no evidence of convulsions seen.
With the recent removal of its amyotrophic lateral sclerosis drug from the market, Amylyx Pharmaceuticals Inc. is looking to revive investor interest with interim data from its phase II Helios study testing the same drug, AMX-0035 (sodium phenylbutyrate plus taurursodiol), in Wolfram syndrome, a rare indication in which Amylyx could be leading the charge.
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