Sarepta Therapeutics Inc.’s next-generation peptide-conjugated PMO therapy, SRP-5051 (vesleteplirsen), looks set to stake its claim in the Duchenne muscular dystrophy (DMD) space, as phase II data unveiled dystrophin expression that outperforms first-generation exon-skipping drug Exondys 51 (eteplirsen). The question is what that space might look like in the wake of a U.S. FDA decision whether to expand labeling and convert to full approval Sarepta’s DMD gene therapy, Elevidys (delandistrogene moxeparvovec).
For full year 2023, clinical trial updates saw a 5.23% increase compared to the corresponding period last year. In December, BioWorld tracked 281 phase I-III clinical trial updates, a decline from the 354 noted in November and 416 reported in October.
Shares of Corbus Pharmaceuticals Inc. (NASDAQ:CRBP) doubled on opening Jan. 26 and ended the day up a whopping 249% as investors got a look at data from a first-in-human study testing next-generation Nectin-4-targeted antibody-drug conjugate (ADC) CRB-701. Licensed from CSPC Pharmaceutical Group for $7.5 million up front in February 2023, CRB-701 has IND clearance from the U.S. FDA, and Corbus plans to start its own clinical testing in the first quarter of 2024.
Wall Street got what it wanted from Vera Therapeutics Inc. with atacicept in IgA nephropathy (IgAN) and rewarded shares of the Brisbane, Calif.-based firm (NASDAQ:VERA), pushing them to close Jan. 25 at $25.31, up $8.29, or 49%.
The hearing has returned for the first person who has received gene therapy for treating genetic hearing loss in the U.S. Initial results from Akouos Inc.’s phase I/II study showed that within 30 days of receiving AK-OTOF-101, pharmacologic hearing was restored to an 11-year-old who had profound hearing loss from birth.
About two months after reporting that enrollment of its phase II/III study with buntanetap in Alzheimer’s disease had completed enrollment, Annovis Bio Inc. disappointed Wall Street by saying that findings from the phase III in Parkinson’s disease – hoped for by the end of this month – would be delayed for data-cleaning reasons.
Sciwind Biosciences Co. Ltd.’s oral ecnoglutide (XW-004) produced strong weight loss results after short-term treatments, up to a 6.8% body weight decrease in obese and overweight participants after once-daily dosing for six weeks, according to data from the first four cohorts of the phase I trial.
Biontech SE and Duality Biologics Co. Ltd. have progressed BNT-323/DB-1303 to pivotal phase III trials, beginning in China in patients with hormone receptor-positive and HER2-low metastatic breast cancer.
What had been viewed as a major catalyst for Gilead Sciences Inc. going into 2024 turned into a disappointment on the stock market, as antibody-drug conjugate Trodelvy (sacituzumab govitecan) failed to meet the overall survival primary endpoint in the phase III Evoke-01 study in previously treated metastatic non-small-cell lung cancer (NSCLC).
Sagimet Biosciences Inc. stock (NASDAQ:SGMT) more than doubled Jan. 22 as shares closed 170% higher at $18.42 each as a phase IIb study of lead candidate denifanstat performed well against nonalcoholic steatohepatitis (NASH) compared to placebo. Top-line data from the 52-week, randomized, double-blind Fascinate-2 trial showed the oral fatty acid synthase inhibitor resulted in statistically significant improvements in biopsy-confirmed NASH patients with stage 2 or stage 3 fibrosis, which is moderate to severe disease, at week 52.
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