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Lack of efficacy brought the development of two investigational agents for amyotrophic lateral sclerosis (ASL) to a halt over the past week. On May 23, Wave Life Sciences Inc. disclosed that its stereopure antisense oligonucleotide WVE-004 failed to demonstrate clinical benefit after 24 weeks of treatment on a phase Ib/IIa trial in familial ALS patients or frontotemporal dementia patients. And on May 25, Apellis Pharmaceuticals Inc. and its partner, Swedish Orphan Biovitrum International AB, said that pegcetacoplan failed to meet its primary endpoint of a one-year phase II trial in patients with sporadic disease.
The U.K. government has announced £121 million (US$148 million) in funding to reboot the commercial clinical trials system after a sharp decline saw the number of industry-sponsored studies falling by 44% from 2017 to 2021.
Providing no details, Mirati Therapeutics Inc. failed to meet its overall survival primary endpoint in the phase III Sapphire trial with sitravatinib. The receptor tyrosine kinase inhibitor was tested in combination with anti-PD-1 checkpoint inhibitor Opdivo (nivolumab, Bristol Myers Squibb Co.) vs. docetaxel in patients with second- or third-line advanced nonsquamous non-small-cell lung cancer. According to Mirati, this patient population consists of about 70,000 people in the U.S. and Europe who have derived prior benefit from a PD-(L)1 inhibitor.
Mixed phase III study results have Bioxcel Therapeutics Inc. moving ahead to complete the clinical trial while withstanding a hammering from investors. BXCL-501 (dexmedetomidine), a sublingual film being developed to treat bipolar disorders- or schizophrenia-associated agitation, produced clinically meaningful efficacy results in part 1 of the pivotal study with half of the approved dose, but the primary efficacy endpoint was not statistically significant at two hours (p=0.077). BXCL-501 separated from placebo at four hours (p=0.049).
On the strength of upside in best corrected visual acuity (BCVA) in patients with geographic atrophy (GA), Annexon Inc. officials will meet with the U.S. FDA to talk about what’s next for ANX-007, after the C1q inhibitor missed the phase II trial’s primary endpoint: mean rate of change, or slope, in the GA lesion area compared to sham.
Cansino Biologics Inc. reported positive data in a phase IIb trial evaluating the heterologous mRNA vaccine CS-2034 booster compared to an inactivated vaccine to prevent SARS-CoV-2 infections.
“We clearly see an active drug here,” PTC Therapeutics Inc. CEO Matthew Klein said of the 15-lipoxygenase inhibitor vatiquinone for Friedreich’s ataxia (FA), tested in a phase III study called Move-FA that missed the primary endpoint of statistically significant change in modified FA Rating Scale score at 72 weeks. The company will “take one step at a time” decisions about the drug, analyzing the results and then consulting with the U.S. FDA regarding how to proceed, he said. Meanwhile, Wall Street wasn’t happy, and South Plainfield, N.J.-based PTC’s shares (NASDAQ:PTCT) closed May 24 at $46.95, down $11.46, or 19.6%.
While pricing a $67.8 million registered direct offering, Icosavax Inc. also released positive top-line interim data from a phase I study of IVX-A12 against respiratory syncytial virus (RSV) and human metapneumovirus in older adults.
Long-term brain recordings from four patients with chronic pain have led investigators at the University of California at San Francisco to identify brain signals that could serve as biomarkers for each individual patients’ pain.
Hanall Biopharma Co. Ltd.’s phase III trial VELOS-3 of tanfanercept did not show effects on the central cornea of the eye or in eye dryness in the treatment of moderate to severe dry eye disease, the primary endpoints. Data from the trial did show improvements in tear production, a secondary endpoint.