Overall, results from the phase III Valor trial testing Lyme disease vaccine PF-07307405 “strengthen confidence” in the candidate, according to partners Pfizer Inc. and Valneva SE, with the big pharma now looking toward regulatory submissions. Investors, however, focused on the fact that the study, which demonstrated more than 70% efficacy in preventing Lyme disease in individuals, ages 5 and older, failed to hit the statistical criterion for the first prespecified analysis.
Facing increasing competition to PI3Kα inhibitor Piqray (alpelisib) in breast cancer, Novartis AG looks to bolster its pipeline with next-generation programs, including phase I/II-stage candidate SNV-4818, by way of a potential $3 billion deal with Synnovation Therapeutics LLC.
Any lingering disappointment in the delay for Xenon Pharmaceuticals Inc.’s readout of the phase III X-Tole2 study testing azetukalner in focal onset seizures (FOS) appeared thoroughly extinguished as the company’s KV7 potassium channel opener yielded better-than-expected data, even besting earlier phase IIb findings and positioning the drug for an NDA submission later this year.
Investors looking for midteen percentage weight loss findings in Zealand Pharma A/S’s phase II study of once-weekly amylin analogue petrelintide were clearly disappointed by the actual 10.7% mean body weight reduction, as shares (Copenhagen:ZEAL) fell 36% March 6. But from the company’s standpoint, the combination of efficacy and safety data from the Zupreme-1 study bode well for a phase III trial expected to start later this year.
Can-Fite Biopharma Ltd.’s namodenoson met the safety endpoint in its phase IIa open-label study in advanced pancreatic ductal adenocarcinoma patients, a readout that left investors hopeful for survival data, expected to be disclosed later this year.
Uniqure NV is the latest firm to get caught between the FDA’s shifting demands for “gold standard” science and regulatory flexibility for rare disease therapies. The company disclosed in its latest earnings report that U.S. regulators are calling for a sham-controlled study before they will consider approval of gene therapy AMT-130 in Huntington’s disease, a requirement that could set the program back by two to three years and raises potential ethical issues.
The EMA’s Committee for Medicinal Products for Human Use recommended approval of Moderna Inc.’s Mcombriax (mRNA-1083), positioning it to potentially become the world’s first combination vaccine for both seasonal influenza and COVID-19.
Coming off a U.S. FDA approval of the first GLP-1 in pill format, Novo Nordisk A/S leaned further into oral drug delivery efforts, partnering with Vivtex Corp. to develop next-generation oral formulations of peptide and protein therapeutics in a potential $2.1 billion deal that marks the highest-profile news for Vivtex since the firm spun out of MIT in 2018.
An “outsized placebo response” is not stopping Gossamer Bio Inc. from seeking a path to potential approval of inhaled tyrosine kinase inhibitor seralutinib in pulmonary arterial hypertension (PAH), but the missed primary endpoint in the phase III Prosera study sent shares of the company (NASDAQ:GOSS) tumbling 80% and left investors skeptical going forward.
Vanda Pharmaceuticals Inc. gained U.S. FDA approval for Bysanti (milsaperidone), cleared for use in acute bipolar I disorder and schizophrenia and ensuring continuation of the firm’s atypical antipsychotic franchise, with Fanapt (iloperidone) set to start losing patent protection in 2027. It also offered a rare straightforward regulatory win for Vanda, which has tussled with the FDA in recent years.
