While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, investors focused on three serious adverse events related to the drug, driving shares (NASDAQ:DYNE) down by 31% on Sept. 3.
With a successful phase III study of plozasiran in hand, Arrowhead Pharmaceuticals Inc. plans to file an NDA with the U.S. FDA for treating the rare genetic disease familial chylomicronemia syndrome. While the data are strong, the company is playing catch-up to Ionis Pharmaceuticals Inc., which has a December 2024 PDUFA for its candidate, olezarsen, in the same indication.
George Medicines Ltd. has published positive data from two phase III studies showing GMRx2, a low-dose triple-drug combination treatment for hypertension, is superior to dual combinations of its components.
Sanofi SA’s brain-penetrant Bruton's tyrosine kinase (BTK) inhibitor, tolebrutinib, met the primary endpoint in the phase III Hercules trial in non-relapsing secondary progressive multiple sclerosis (nrSPMS). The first compound to show reduction in disability accumulation in MS, tolebrutinib delayed the time to onset of confirmed disability progression in people with nrSPMS, a population for which there are currently no approved therapies.
Potential implications for others in the space, including Pfizer Inc. and Bridgebio Pharma Inc., became the subject of buzz after Alnylam Pharmaceuticals Inc. provided details from the Helios-B phase III study with RNAi candidate vutrisiran, in the works for ATTR amyloidosis with cardiomyopathy.
After initially reporting that Xanamem failed to meet the primary endpoint in cognitive attention in the phase IIa Xanacidd cognition and depression trial, Actinogen Medical Ltd. CEO Steven Gourlay said a new analysis shows clinically and statistically significant benefits in depression.
Neurocrine Biosciences Inc.’s top-line phase II data, though billed as promising, wasn’t enough to please observers on Wall Street, who stacked the data in schizophrenia with NBI-1117568 (NBI-'568) against others in the space. Shares (NASDAQ:NBIX) closed Aug. 28 at $123.76, down $28.79, or 18.9%.
Yunovia Co. Ltd. gained clearance in South Korea to start a multiple ascending dose phase I study for ID-110521156 – a novel, orally available, small-molecule, glucagon-like peptide-1 (GLP-1) agonist.
Microbiotica Ltd. is poised to advance two of its microbiome-derived products into the clinic after securing regulatory approval and fresh finance. The first live bacterial therapeutic, MB-097, will be tested in combination with Keytruda (pembrolizumab) in patients with advanced melanoma who have not responded to treatment with immune checkpoint inhibitors. The second product, MB-310, is a once-daily oral therapy for treating the inflammatory bowel disease, ulcerative colitis.
About two months after Astrazeneca plc said its application for sipavibart (AZD-3152) had been accepted by the EMA for pre-exposure prophylaxis (PrEP) against COVID-19 in immunocompromised people, Invivyd Inc. unveiled positive 180-day exploratory efficacy data from the company’s ongoing Canopy phase III trial with Pemgarda (pemivibart) in the same indication – and made known less happy news from regulators on the other side of the pond.
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