Despite numerous marketed anti-seizure medications in the U.S., patients with epilepsy continue to experience breakthrough seizures and tolerability issues compounded with drug interactions and side effects, leaving room for companies like Praxis Precision Medicines Inc. to fill treatment gaps. The Boston-based company presented phase IIa proof-of-concept data March 26 showing that in an evaluation of photo paroxysmal response, its once-daily oral small-molecule treatment, PRAX-628, resulted in a complete response in three epilepsy patients receiving 45 mg of the drug and four patients receiving 15 mg for a combined complete response rate of 88%.
Neuromuscular disease specialist NMD Pharma A/S has been given U.S. FDA approval for a phase IIb trial of NMD-670, after demonstrating proof of mechanism for the orally-available chloride ion channel-1 inhibitor in generalized myasthenia gravis.
Top-line phase II results of privately held Ocuterra Therapeutics Inc.’s novel selective small-molecule RGD integrin inhibitor, nesvategrast (OTT-166), showed it failed to meet primary and key secondary endpoints in patients with diabetic retinopathy, prompting the Boston-based company to seek strategic alternatives.
The topical combination therapy miconazole plus domiphen bromide (MCZ-DB) resolved signs and symptoms of acute vulvovaginal candidiasis (VVC) and eradicated the yeast Candida albicans from vaginal swabs to a greater extent than miconazole (MCZ) alone in a phase II study, according to Hyloris Pharmaceuticals SA and Purna Female Healthcare.
Allakos Inc. terminated all development of its lead drug, anti-Siglec-8 antibody lirentelimab (AK-002), and is cutting its workforce in half, following phase II failures for atopic dermatitis and chronic spontaneous urticaria. The San Carlos, Calif.-based company will now focus on phase I trials for anti-Siglec-6 antibody AK-006 as part of a restructuring that stretches the runway into mid-2026. Shares (NASDAQ:ALLK) sank by 60.2%, down $1.80, to close at $1.19 on Jan. 16.
Lumos Pharma Inc.’s novel approach with LUM-201 in pediatric growth hormone deficiency paid off in top-line results, and the company is laying the groundwork for a phase III study next year. Pisit “Duke” Pitukcheewanont, senior vice president of global clinical development and medical affairs for Lumos, told BioWorld that the ability of LUM-201 to generate hormone at the normal range, promoting growth comparable to standard-of-care daily or weekly injections is “groundbreaking.”
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.
While Ventyx Biosciences Inc.’s oral S1P1 receptor modulator, VTX-002, achieved primary and secondary endpoints in a phase II ulcerative colitis (UC) trial, the company’s shares (NASDAQ:VTYX) fell 25.9%, or $7.78, closing Oct. 10 at $22.22, possibly due to a higher-than-expected placebo response and a clinical remission rate that was similar to Pfizer Inc.’s etrasimod.
Alnylam Pharmaceutical Inc. hopes to tap into Roche Holding AG’s global footprint with its early-stage hypertension RNAi therapeutic, zilebesiran, selling development and commercialization rights to the pharma giant for up to $2.8 billion with a view to extending the drug’s future reach beyond the U.S market.
SK Bioscience Ltd. and Sanofi Pasteur SA announced June 30 positive results from a phase II study for its 21-valent pneumococcal conjugate vaccine (PCV) candidate called GBP-410, or SP-0202 under Sanofi.
