It has been a long time coming, but Fabre-Kramer Pharmaceuticals Inc. finally received U.S. FDA approval for its major depressive disorder candidate, Exxua (gepirone hydrochloride extended-release tablets). The approval comes three months after the June 23 PDUFA date (as a result of amendments filed by the company in April and May), but 24 years after the original NDA was filed in 1999.
Positive top-line data from the phase IIb study of BNC-210 to treat post-traumatic stress disorder (PTSD) supercharged Bionomics Ltd.’s stock. Shares (NASDAQ:BNOX) closed trading a dramatic 243.8% higher at $3.37 each on Sept. 28. The oral, selective negative allosteric modulator of the alpha7 nicotinic acetylcholine receptor stumbled last December in a phase II trial for treating social anxiety disorder but now has regained momentum.
Brainstorm Cell Therapeutics Inc. said it’s exploring all its options in the wake of a Sept. 27 U.S. FDA advisory committee vote, in which the committee overwhelmingly disagreed with the company that the data it presented supported the effectiveness of Nurown (debamestrocel) for the treatment of mild to moderate amyotrophic lateral sclerosis.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified 3-alkynyl carboxamides acting as legumain (asparaginyl endopeptidase; LGMN) inhibitors.
Immuther Pharmtech (Shanghai) Co. Ltd. has patented lysophosphatidic acid LPA5 receptor (GPR92) antagonists reported to be useful for the treatment of neuropathic pain, cancer, neurological disorders, atherosclerosis, inflammatory and autoimmune diseases, fibrosis and obesity, among others.
Gro Biosciences Inc. has presented successful proof-of-concept results in two preclinical programs for autoimmune disease and immunogenicity. The company’s genomically recoded organism (GRO) platform enables precise placement of non-standard amino acids (NSAAs) within a protein.
A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.
Brainstorm Cell Therapeutics Inc.’s Nurown got a thumbs down from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Sept. 27, as the committee voted 1-17, with one abstention, that the data presented demonstrated substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis.
Arialys Therapeutics Inc. launched this month with $58 million in seed funding, an experimental compound it is developing for autoimmune encephalitis and autoimmune psychosis, and high aspirations for its field. “Yes, I want to treat these patients, I want these patients to have a better life. But I also want drug discovery and development folks to think differently about discovering new drugs for the CNS,” Jay Lichter told BioWorld.
Acurastem Inc. said on Sept. 25 that it struck an out-licensing deal potentially worth $580 million with Takeda Pharmaceutical Co. Ltd. to develop drugs for amyotrophic lateral sclerosis (ALS) and other PIKfyve gene-targeting therapeutics. Under the terms, Tokyo-headquartered Takeda obtains exclusive worldwide rights to Acurastem’s PIKfyve-targeting therapeutics, including Acurastem’s lead AS-202 asset, an antisense oligonucleotide therapy to treat ALS.
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