Failing to meet the primary endpoint in its confirmatory phase III Embark trial, Sarepta Therapeutics Inc.’s gene therapy, Elevidys (delandistrogene moxeparvovec), which received accelerated approval in June and was priced at $3.2 million, has one of three fates in its future, all of which are dependent on how the U.S. FDA perceives the data. Based on secondary endpoints showing statistical significance and a recent positive meeting with the agency, Sarepta could continue to market Elevidys under its current label for 4- and 5-year-old ambulatory Duchenne muscular dystrophy (DMD) patients; Sarepta is filing the postmarketing requirement needed to transition from accelerated to full approval. Read More
Lianbio Co. Ltd. announced mixed phase III top-line results on Oct. 30 for its in-licensed Demodex blepharitis treatment called TP-03, with the U.S. FDA-approved eyedrop hitting just one co-primary endpoint in the Libra trial on Chinese patients. Read More
Disappointing preliminary top-line data from Kintara Therapeutics Inc.’s phase II/III study of VAL-083 (dianhydrogalactitol) is causing the company to shift its attention and resources to another cancer program. In the meantime, the results wreaked havoc on the stock and sent the company on a mission to look at its options. Read More
Clinical trial data from January-September 2023 was up 1.79% compared to the same time period last year. In the first three quarters of this year, BioWorld reported on 2,611 drugs in phase I-III, compared to 2,565 in Q1-Q3 2022. The number of trial updates is down 13.74% from 3,027 in 2021 and also down from the 2,738 in 2020, but up from 2,251 updates noted in 2019. Read More
The Biden administration has issued an executive order (EO) for artificial intelligence (AI), which addresses not only national security considerations, but public health considerations as well. One of the features of the EO is that any company developing AI for public health and safety must notify the federal government when training a foundation AI algorithm, which suggests that AI that is regulated by the U.S. FDA will now be subject to additional government scrutiny in the premarket phase. Read More
New hires and promotions in the biopharma industry, including: Aavantgarde, Calidi, Cognito, Dren, Evecxia, Immpact, Protagonist, Rentschler, Third Harmonic, Turnstone, Vir, Viridian. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Allyx, Aptose, Aqilion, Coya, Deciphera, Geovax, GSK, Junshi, Leo, Novartis, Novelmed, Reviva, University of Oxford, Volastra, Vyne. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: BMS, Coherus, Day One, Junshi, Nurexone, Oncoinvent, Phathom, Sellas. Read More
