Pliant Therapeutics Inc., which offered few details earlier this week when it voluntarily paused enrollment in the phase IIb Beacon-IPF trial testing bexotegrast in idiopathic pulmonary fibrosis (IPF), has taken an unusual step as it assembles a panel of outside experts to review unblinded data from the study with the goal of providing an independent recommendation.
Snagging its second U.S. FDA approval since being spun out of Pfizer Inc. in 2017, Springworks Therapeutics Inc. is aiming to position Gomekli (mirdametinib), cleared for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) not amenable to complete resection, as a first-in-class drug for adults and potentially best-in-class option for children. The priority approval, which came after market close Feb. 11, more than two weeks ahead of the Feb. 28 PDUFA date, marks the second MEK1/2 inhibitor to hit the market for NF1-PN, a rare, genetic condition.
Touting its novel approach for treating glaucoma, Qlaris Bio Inc. reported promising top-line data from two phase II studies testing QLS-111 in patients with primary open-angle glaucoma and ocular hypertension, showing the drug met all primary and secondary endpoints, reducing intraocular pressure with a clean safety profile that could encourage patients to remain on treatment.
An effort two decades in the making, Vertex Pharmaceuticals Inc.’s suzetrigine gained U.S. FDA approval as the first drug targeting the NaV1.8 pain signal. Branded Journavx, the oral small molecule is cleared for use as a non-opioid option for treating moderate to severe acute pain.
In an indication that has proved difficult for biopharma to conquer, Regulus Therapeutics Inc. disclosed further positive data from its ongoing phase Ib study testing RGLS-8429 in autosomal dominant polycystic kidney disease and laid out its plans to move straight into a phase III trial later this year, with the potential for an accelerated U.S. approval.
What a difference 60 weeks can make. That’s the lesson Akero Therapeutics Inc. shared with the rollout of what executives called “unprecedented” data from the phase IIb Symmetry trial testing efruxifermin (EFX), its FGF21 receptor agonist, in patients with compensated cirrhosis due to metabolic dysfunction-associated steatohepatitis (MASH). While earlier findings had shown only a trend in improvement at 36 weeks, full 96-week results showed more than doubling of earlier effect size, hitting the primary endpoint and sending shares of Akero (NASDAQ:AKRO) up 97% to close Jan. 27 at $51.71.
Medexus Pharmaceuticals Inc. looks set for its U.S. launch of bifunctional alkylating agent treosulfan in the first half of 2025 following a long-awaited FDA approval of the drug, branded Grafapex, for use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adults and pediatric patients, 1 and older, with acute myeloid leukemia or myelodysplastic syndrome.
With positive data in hand from its phase IIb trial testing immunotherapy candidate OST-HER2 in osteosarcoma, OS Therapies Inc. anticipates a regulatory filing this year seeking accelerated approval from the U.S. FDA, putting the firm on track to receive a potentially profitable rare pediatric disease priority review voucher that could help fund further R&D work. Should OST-HER2 go on to win approval, it would mark the first new therapy in more than 40 years for osteosarcoma, an aggressive bone cancer characterized by high rates of metastases, often to the lungs, and disease recurrence.
Joining the dealmaking spree to kick off the 2025 J.P. Morgan Healthcare Conference, Eli Lilly and Co. announced it was picking up an early clinical-stage PI3Kα inhibitor program from Scorpion Therapeutics Inc. in a deal that could be worth up to $2.5 billion, while GSK plc is adding to its cancer pipeline with the acquisition of Idrx Inc. for $1 billion up front.
Though Pfizer Inc.’s work on a PD-1-targeting antibody has trailed far behind that of its fellow big pharmas, the company could be the first to market in treatment-naïve, high-risk non-muscle invasive bladder cancer on the back of solid phase III data showing subcutaneously administered sasanlimab hit its endpoint of event-free survival. Pfizer anticipates meeting with regulatory agencies to discuss potential filings for what could be the first new treatment for that NMIBC population in decades.
