The Alzheimer’s disease (AD) space took another blow along with the shares of Cassava Sciences Inc. (NASDAQ:SAVA), which plummeted by 83.8%, or $22.19, to end Nov. 25 at $4.30 after the firm unveiled top-line data from the phase III Rethink-Alz study with simufilam. The study tested simufilam in mild to moderate AD, where results fell short of each of the prespecified co-primary, secondary and exploratory biomarker endpoints.
Autoimmunity Biosolutions has closed a seed financing to support its work advancing a next-generation, immuno-corrective therapy for autoimmune diseases. The company’s approach targets a branch of the interleukin-7 (IL-7)/interleukin-7 receptor (IL-7R) pathway controlled by the soluble IL-7R (sIL-7R), a critical amplifier of autoimmune reactions.
Gruenenthal GmbH has patented new cis-8-(3,5-difluorophenyl)-8-(dimethylamino)-1,3-diazaspiro[4.5]decan-2-one derivatives acting as nociceptin receptor (OPRL1; KOR3; ORL1) agonists and thus reported to be useful for the treatment of pain.
Voyager Therapeutics Inc. has selected a lead development candidate, VY-1706, for its tau silencing gene therapy program in Alzheimer’s disease. The company anticipates filing an IND application with the FDA and a clinical trial application (CTA) with Health Canada for VY-1706 in 2026.
China’s drug regulator approved Alpha Biopharma Ltd.’s EGFR tyrosine kinase inhibitor (TKI), Zorifer (zorifertinib hydrochloride tablets; AZD-3759), as a first-line treatment for EGFR-mutated non-small-cell lung cancer (NSCLC) with brain metastases.
Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
The bad news keeps piling up for Sage Therapeutics Inc. Having absorbed other study stumbles in the past few months, the company now has halted development of dalzanemdor in treating Huntington’s disease after top-line phase II data showed it missed a statistically significant difference compared to placebo on the primary endpoint.
Aeovian Pharmaceuticals Inc. has prepared tricyclic fused imidazole compounds acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors reported to be useful for the treatment of neurodegeneration, inflammation, fibrosis, systemic scleroderma and more.
Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).
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