Paxmedica Inc.’s shares (NASDAQ:PXMD) surged by 179% in early trading Nov. 7 after reporting the publication of phase II autism spectrum disorder results for PAX-101 (suramin) intravenous infusions. Suramin, an anti-trypanosomal and anti-purinergic agent introduced in 1923 for East African sleeping sickness, showed a statistically significant improvement at the 10-mg/kg dose in a secondary endpoint of Clinical Global Impressions – Improvement scale vs. placebo.
Having rolled out less-than-stellar phase II data with oral TYK2 inhibitor VTX-958 in moderate to severe plaque psoriasis, Ventyx Biosciences Inc. faces Wall Street speculation regarding the odds for the ongoing effort in Crohn’s disease (CD), due for an interim analysis in the first quarter of next year.
Top-line results from Moonlake Immunotherapeutics AG’s phase II study of sonelokimab treating active psoriatic arthritis encouraged the company but discouraged investors.
Kymera Therapeutics Inc.’s KT-333 new phase I data, while early in the STAT3 targeted protein degrader’s development, showed results that cheered the company’s stock. It didn’t hurt that the company also took in a $40 million milestone from partner Sanofi SA in their collaboration to develop the IRAK4 degrader KT-474.
Shares of Werewolf Therapeutics Inc. (NASDAQ:HOWL) reflected enthusiasm on Wall Street for the prospect, albeit early stage, of systemically delivering a masked native interleukin-2 (IL-2) cytokine to cancer patients.
Among the avalanche of abstracts for the upcoming American Society of Hematology (ASH) annual conference released early on Nov. 2 were data from Arcellx Inc.’s phase I study of CART-ddBCMA in patients with relapsed or refractory multiple myeloma. The results caught investors’ attention as the company’s stock (NASDAQ:ACLX) jumped 18.5% upward to close at $40.77 per share.
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.
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.
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.
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.
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