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. Based on secondary endpoints showing statistical significance and a positive meeting with the U.S. FDA, Sarepta could continue to market it under its current label for 4- and 5-year-old ambulatory Duchenne muscular dystrophy (DMD) patients. A better fate would be an expansion into 6- and 7-year-old ambulatory patients, and possibly to all patients, regardless of age or ambulatory status. The third option, however, is a complete withdrawal from the U.S. market – a possibility that greatly threatened investors on Oct. 31, with Sarepta’s shares (NASDAQ:SRPT) plummeting by 40% to $64.07 at midday, having reached its 52-week low of $55.25 earlier in the day. “We acknowledge that the Embark data were disappointing and that there may be emotional, structural and perceived credibility considerations at play here as well,” said analyst Brian Abrahams, of RBC Capital Markets, “but trying to take an objective, fundamental view of the company’s valuation indicates that at this point virtually all Elevidys promise is washed out of the stock here – in our view, an overreaction and a buying opportunity.”

Lianbio says hit-and-miss for co-primary endpoints in Xdemvy’s China trial

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 FDA-approved eyedrop hitting just one co-primary endpoint in the Libra trial on Chinese patients. The multicenter, randomized, double-blind, vehicle-controlled registrational Libra study evaluated TP-03’s safety and efficacy in 163 Chinese adult patients with Demodex blepharitis. Lianbio also ran a single-arm, open-label pharmacokinetics substudy to evaluate TP-03’s systemic PK parameters. Top-line readout of the study in China showed that TP-03 significantly improved one of the co-primary endpoints concerning mite eradication, defined as density of zero mites per lash, in patients with Demodex blepharitis compared to vehicle at day 43 (p<0.001). But TP-03 did not reach statistical significance (p=0.15) for the co-primary endpoint of complete collarette cure at day 43, defined as a collarette score of zero, although the trend was positive.

Kintara shifts its focus after disappointing glioblastoma data

Disappointing preliminary top-line data from Kintara Therapeutics Inc.’s phase II/III study of VAL-083 are causing the company to shift its attention and resources to another program, REM-001. Results showed that VAL-083’s performance in the study of treating glioblastoma was no better than the current standards of care. REM-001 is a second-generation photodynamic therapy photosensitizer agent for treating cutaneous metastatic breast cancer. It was granted U.S. FDA fast track designation for the indication in November 2022. The data mightily shook up Kintara’s stock (NASDAQ:KTRA) at midday, with shares trading 78% downward at 85 cents each. 

Clinical trial data show 1.79% growth year-on-year through Q3 2023 

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. 

Biden administration seeks information on AI algorithms during algorithm training 

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 an algorithm, which makes clear that AI that is regulated by the FDA will now be subject to additional government scrutiny in the premarket phase.

Also in the news

Aavantgarde, Affamed, Alfasigma, Amgen, Annovis, Avalo, Avenue, Basilea, Beam, Biofrontera, Bristol Myers Squibb, Calidi, Cambrium, Catalyst, Cognito, Coherus, Day One, Dren, Engine, Epic, Evecxia, Galapagos, Galera, GSK, Imagine, Immpact, Junshi, Kite, Laverock, Maplight, Nurexone, Octapharma, Okyo, Oncoinvent, Paxmedica, Phathom, Protagonist, Rentschler, Sanguine, Scinai, Sellas, Third Harmonic, Transcode, Turnstone, Verve, Vir, Viridian, Vivos