Four months after Vertex Pharmaceuticals Inc.’s U.S. FDA nod for Journavx (suzetrigine) as the first drug targeting NaV1.8 for treating pain, Eli Lilly and Co. is joining the potential competition via a buyout of Siteone Therapeutics Inc., a privately held firm developing small-molecule sodium channel inhibitors, including a phase II-ready NaV1.8 inhibitor.
Mirroring results reported in a phase IIb study, Apnimed Inc.’s first of two pivotal trials testing AD-109 as an oral therapy in obstructive sleep apnea hit primary and secondary endpoints. Should similar findings emerge from a second phase III trial, expected to read out in a couple of months, the privately held firm anticipates a U.S. NDA filing in early 2026.
It’s been a big week for Inozyme Pharma Inc. On the heels of a promising interim readout for phase III-stage enzyme replacement therapy (ERT) candidate INZ-701 in ENPP1 deficiency, the firm agreed to be acquired by Biomarin Pharmaceuticals Inc. in a deal valued at about $270 million, putting the rare disease ERT in the hands of an experienced commercial team.
Only a few days out of the European Association for the Study of the Liver annual meeting, the metabolic dysfunction-associated steatohepatitis (MASH) space continues to grab headlines, with GSK plc shelling out $1.2 billion up front to acquire phase III-ready efimosfermin alfa in a deal with Boston Pharmaceuticals Inc. that could end up totaling about $2 billion.
Theriva Biologics Inc.’s stroma-targeting oncolytic virus approach yielded positive findings in metastatic pancreatic ductal adenocarcinoma, with the top-line readout of phase IIb data showing VCN-01 (zabilugene almadenorepvec) in combination with chemotherapy bested chemotherapy alone on primary and secondary endpoints, which included overall survival.
Regulus Therapeutics Inc. CEO Jay Hagan told investors in a January call the company had no interest in “simply out-licensing” rights to lead candidate farabursen, an oligonucleotide targeting autosomal dominant polycystic kidney disease set to start phase III testing on a path to a potential accelerated approval. And now, there’s no need to, as Regulus found a buyer for the whole company in a deal with Novartis AG valued at about $1.7 billion.
Shares of CG Oncology Inc. gained 25% April 28 on the back of phase III data presented at the American Urological Association meeting over the weekend showing its cretostimogene grenadenorepvec yielded durable response rates, as well as an enviable safety profile, in patients with high-risk non-muscle invasive bladder cancer unresponsive to Bacillus Calmette Guerin treatment with carcinoma in situ with or without Ta or T1 disease.
Synthetic Design Lab Inc., which emerged from stealth with a $20 million seed round and a platform technology aimed at advancing the antibody-drug conjugate (ADC) space, began with its founders identifying a single clear goal: how to deliver more payload to a target cancer cell.
Synthetic Design Lab Inc., which emerged from stealth with a $20 million seed round and a platform technology aimed at advancing the antibody-drug conjugate (ADC) space, began with its founders identifying a single clear goal: how to deliver more payload to a target cancer cell.
Pfizer Inc. is ending work on oral GLP-1 candidate danuglipron for weight loss following the report of a single potentially drug-induced liver injury, a move that appears to open the door for other firms working on oral therapies in the high-dollar obesity space, even as industry watchers seek further details to determine whether similar safety signals could emerge for those competitors.
