Gene therapy specialist Meiragtx Holdings plc got a market bump courtesy of newly released top-line data from its phase II bridging study in Parkinson’s disease. The six-month, three-arm randomized, double-blind, sham controlled trial of AAV-GAD, a one-time infusion, demonstrated significant and clinically meaningful improvements in key efficacy endpoints. The primary objective was evaluating the therapy’s safety and tolerability. The study of participants with idiopathic disease showed the therapy was safe and well-tolerated with no serious adverse events. Meiragtx is pursuing approvals in the U.S., Europe and Japan.

Booster Therapeutics is ready to open up a new arm of the proteasome after raising $15 million in seed funding to advance small molecules it says can degrade multiple types of harmful proteins. Rather than tagging single disease proteins with a ubiquitin marker for degrading via 26S proteasomes, these compounds directly activate 20S proteasomes that naturally recognize disordered proteins without the need for ubiquitin tagging.

Bad news has buffeted Sage Therapeutics Inc. twice in the past few months. Now its placebo-controlled phase II Lightwave study of dalzanemdor in Alzheimer's disease has missed the primary outcome measure, prompting the company to stop development of the NMDA receptor positive allosteric modulator in the indication.

Aviadobio Ltd. has entered a potential $2.18 billion license and commercialization agreement for its frontotemporal dementia gene therapy, AVB-101, with Astellas Pharma Inc. Astellas is making a $20 million equity investment in London-based Aviadobio and will pay up to $30 million up front in advance of deciding whether or not to exercise the exclusive option to worldwide rights.

Shares of Scholar Rock Holding Corp. (NASDAQ:SRRK) soared $26.86, or 362%, to close Oct. 7 at $34.28, after the Cambridge, Mass.-based firm disclosed positive top-line data from the phase III Sapphire study testing apitegromab in patients with spinal muscular atrophy (SMA). Apitegromab, which Wainwright analyst Andres Maldonado said will “transform SMA” therapy, met the primary endpoint with statistically significant and clinically meaningful improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded.

Loqus23 Therapeutics Ltd. has raised £35 million (US$46.6 million) in a series A to take forward small molecules it has discovered for the treatment of Huntington’s disease and other conditions that are driven by DNA mismatch repair (MMR). MMR fixes DNA insertions, deletions and misincorporation errors that occur during transcription and/or cellular replication. Smaller repairs are directed by MutSalpha, a protein that binds single base mismatches, while MutSbeta handles larger insertion/deletion loops. Huntington’s and other triplet repeat diseases are caused when trinucleotide repeats accumulate in somatic DNA to the extent that they interfere with protein expression.

A collaboration led by the Flywire Consortium and comprising hundreds of scientists has completed a whole map of the adult fruit fly brain after several decades of collaborative work. By using electron microscopy and three-dimensional reconstruction supported by AI tools, the researchers have revealed the neural wiring of the Drosophila melanogaster brain, a connectome of 140,000 neurons with 50 million synaptic connections. In the future, researchers could possibly use this map as an artificial in silico model to study the brain as a simulator through its connections, though a lot of work remains to be done for this.

The FDA has approved Cobenfy, a dual M1/M4 muscarinic agonist that offers a fundamentally different approach to treating schizophrenia. The fixed dose combination of xanomeline-trospium is the first to act via a novel mechanism for the serious psychiatric disorder in over 50 years, finally expanding the treatment options beyond dopamine-targeted therapies. Bristol Myers Squibb Co., which acquired Cobenfy developer Karuna Therapeutics Inc. for $14 billion in a deal that closed in March 2024, said the drug will be available in the U.S. from late October.

Nine years on from securing $3.84 million for a phase I clinical trial to test the formulation, with results showing it overcame side effects that had confounded its forerunner, the schizophrenia treatment Karxt met its PDUFA date Sept. 26 with no decision by midday. If approved, the fixed combination of xanomeline-trospium will be the first in a new drug class, and as a dual M1/M4 muscarinic agonist, the first new therapy to act via a novel mechanism for the serious psychiatric disorder in over 50 years.

China’s National Medical Products Administration has approved Sino Biopharmaceutical Ltd.’s rivastigmine transdermal patch to treat mild to moderate Alzheimer’s disease. Developed by Sino Biopharm, the patch is the first domestically produced rivastigmine transdermal patch approved for marketing. Rivastigmine is a cholinesterase inhibitor used for the treatment of Alzheimer’s disease.