Interim phase II data of Wave Life Sciences Ltd.’s oligonucleotide, WVE-N531, revealed “impressive” dystrophin expression, solid safety and the potential for once-monthly dosing for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. The findings drove Wave Life’s stock (NASDAQ:WVE) up by 53.4%, or $2.85, to close Sept. 24 at $8.19, after peaking earlier in the day at $8.35, its 52-week high.
Despite a phase IIa failure in 2018 for lupus, Biogen Inc. and UCB Inc. maintained their collaboration and now have new positive data for their anti-CD40L candidate. Top-line phase III data show dapirolizumab pegol, along with the standard of care (SOC), met the primary endpoint in demonstrating an improvement in moderate to severe systemic lupus erythematosus compared to placebo and SOC at 48 weeks.
A little more than a year after the U.S. FDA refused to review the NDA for Biohaven Ltd.’s ultra rare disease treatment, new and positive phase III data have changed the treatment’s momentum. The upbeat results came as a surprise to analysts and investors, with the stock having a strong day and the company prepping an NDA for a fourth quarter submission.
What the results might mean for the future of other developers in the cannabinoid receptor 1 (CB1) weight-loss arena came into question after Novo Nordisk A/S unveiled phase IIa findings with monlunabant, a small-molecule oral inverse agonist, formerly INV-202.
Ascletis Pharma Inc. is entering the obesity space, announcing it has begun two phase I trials for ASC-30, a small-molecule glucagon-like peptide-1 (GLP-1) receptor agonist that can be dosed once monthly subcutaneously and once daily orally for treating obesity.
The specter of elevated liver enzymes, a known problem with the drug class, became a topic of talk with regard to Sanofi SA’s tolebrutinib, the central nervous system-penetrating Bruton's tyrosine kinase (BTK) inhibitor for multiple sclerosis (MS). Meanwhile, another player in the space, Immunic Inc., has caught the eye of Wall Street lately.
After disclosing data from the phase II Herald study, Aligos Therapeutics Inc. may sign a partner to help advance ALG-055009, a thyroid hormone receptor (THR) beta agonist, in subjects with metabolic dysfunction-associated steatohepatitis (MASH, formerly nonalcoholic steatohepatitis, or NASH).
Positive data from two studies boosted Edgewise Therapeutics Inc.’s market share and elevated analyst enthusiasm for the company and its treatment of obstructive hypertrophic cardiomyopathy. Top-line data from the phase I and phase II studies of EDG-7500 in treating the genetic disease that results in thickened heart muscles showed the therapy was well-tolerated in healthy volunteers and produced meaningful improvements in those with the disease.
Wall Street apparently wanted more from Prelude Therapeutics Inc.’s phase I data with SMARCA2 enzyme degrader PRT-3789 in cancer, which rolled out Sept. 13 during the recent European Society of Medical Oncology Congress in Barcelona, but hopes are still high for other prospects in the class pushed forward by various developers.
Innovent Biologics Inc. unveiled for the first time safety and efficacy data of IBI-354, an HER2 monoclonal antibody-camptothecin derivative conjugate that shows promising efficacy signals across a range of advanced solid tumors, during the European Society of Medical Oncology (ESMO) 2024 Congress. An HER2-targeted antibody-drug conjugate developed using Innovent's topoisomerase inhibitor NT3 platform, IBI-354 combines a hydrophilic linker design with a hydrophobic payload to enhance the bystander effect, targeting adjacent antigen-low or negative tumor cells.
RSS
