Breaking with its long-held, oft-recited mantra that observational studies are great for generating hypotheses but not as evidence for approval, the U.S. FDA is initiating the approval of leucovorin calcium tablets for patients with cerebral folate deficiency, a neurological condition that affects folate transfer into the brain.
As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61.
Shape Therapeutics Inc. could bring in as much as $1.2 billion in a new option and license deal with Vectory Therapeutics BV. It’s another collaboration for both companies that are known for working with large and small pharmas. Vectory is getting the exclusive option to evaluate Shape’s brain-penetrating adeno-associated virus capsid, SHP-DB1, against three targets, including mHTT, TDP-43 for Huntington’s disease and phosphorylated tau for Alzheimer’s disease.
Loss of function variants in the lipid transporter gene ATP-binding cassette ABC transporter A7 (ABCA7) nearly double the risk of developing Alzheimer’s disease (AD), which makes ABCA7 the strongest AD genetic risk factor after ApoE4.
Remynd NV has turned in positive phase IIa data for a new target in Alzheimer’s disease, showing that restoring calcium homeostasis in neurons reduces levels of pathological tau proteins, increases dopamine levels and has a positive effect on cognition in symptomatic patients.
Two months after starting the phase I/II Synrgy trial with its gene therapy, CAP-002, enrolling 12 pediatric patients with rare disease STXBP1 encephalopathy, Capsida Biotherapeutics paused the study following the death of the trial’s first patient.
LB Pharmaceuticals Inc. became the first traditional biopharma company to price an IPO in the U.S. since February, raising $285 million through the upsized sale of 19 million shares at $15 apiece. Shares, now listed on Nasdaq under the ticker symbol LBRX, rose 15% to close Sept. 11 at $17.30.
The U.S. FDA issued new guidance for the development of non-opioid analgesics for chronic pain indications, with specific details on trial design, patient populations and meaningful outcomes, including reducing the nation’s reliance on opioids.
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
Medicxi Ventures’ asset-focused investment model has scored another hit, in a $450 million deal with Servier SA, for KER-0193, an oral small molecule that is ready for phase II development in the treatment of the inherited autism spectrum disorder, fragile X.
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