Impressive data from an interim readout of Bridgebio Pharma Inc.’s BBP-418 in limb-girdle muscular dystrophy type 2I/R9 has the company prepping to meet with the U.S. FDA to discuss plans for the upcoming NDA filing, including the possibility for seeking full approval for what could be the first therapy for the rare muscular disease.
Much-awaited detailed data from Exelixis Inc.’s phase III Stellar-303 study of zanzalintinib, presented at the European Society for Medical Oncology meeting, showed the third-generation, oral tyrosine kinase inhibitor in combination with Tecentriq (atezolizumab, Roche AG) reduced the risk of death by 20% vs. Stivarga (regorafenib, Bayer AG) in patients with previously treated non-microsatellite instability (MSI)-high metastatic colorectal cancer and are expected to pave the way for an NDA filing later this year.
The U.S. FDA named the first nine recipients of the recently unveiled commissioner’s national priority voucher (CNPV) program aimed at addressing unmet public health needs by shortening regulatory review times to as little as one to two months. For one of those firms, Disc Medicine Inc., which submitted an NDA for bitopertin for rare genetic disorder erythropoietic protoporphyria in September, that could mean a potential approval before the end of 2025.
Touting a science-driven, regenerative medicine-based treatment for hair loss “designed for the 21st century,” Pelage Pharmaceuticals Inc. drew a solid group of investors to the table in an oversubscribed $120 million series B round to fund an upcoming phase III program for PP-405, a topical small molecule targeting hair follicle stem cells.
More than a decade after the last idiopathic pulmonary fibrosis (IPF) treatment gained U.S. FDA approval, Boehringer Ingelheim GmbH’s Jascayd (nerandomilast) is set to hit the market following the agency’s green light on Oct. 7. While expected to offer a modest benefit over existing therapies, Jascayd, an orally administered preferential inhibitor of phosphodiesterase 4B with breakthrough therapy status, is viewed by analysts as the first of several potential advancements in the IPF space over the next few years.
A top-line readout of the 26-week phase IIa Cbeyond trial showed nimacimab, Skye Bioscience Inc.’s peripherally restricted CB1 inhibitor antibody for weight loss, fell short of statistical significance as a monotherapy vs. placebo on the primary endpoint of weight loss, sending the company’s shares down 60%. Skye executives, however, offered a more optimistic outlook for the findings, which they said provide clear direction for moving forward.
Patients with chronic spontaneous urticaria who remain symptomatic despite H1 antihistamine treatment now have a new oral treatment option, with the U.S. FDA’s approval of Novartis AG’s Rhapsido (remibrutinib), a Bruton’s tyrosine kinase inhibitor administered by pill twice daily.
Pfizer Inc. has become the first drugmaker to agree to provide its products at most-favored nation (MFN) pricing, an effort aimed at lowering the costs of U.S. drug prices by bringing them in line with the prices paid in other developed nations.
Genmab A/S is paying $8 billion to acquire Merus NV, gaining rights to the latter’s phase III-stage petosemtamab, a bispecific antibody initially in development for head and neck cancer, and continuing the firm’s strategy to transition from a royalty-based operation to a fully integrated biopharma.
As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61.
