FDA approval of Crinetics Pharmaceuticals’ Palsonify makes it the first oral first-line therapy for acromegaly, a rare endocrine disorder. The once-daily pill will compete with blockbuster acromegaly drugs from Novartis and Ipsen that are administered as injections.
UniQure plans a first quarter 2026 FDA submission for AMT-130 as a treatment for Huntington’s disease. This gene therapy delivers microRNA designed to silence the mutated gene that produces a protein driving this disorder, which so far has no FDA-approved therapies.
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Ionis Pharmaceuticals plans a 2026 FDA submission for zilganersen as a treatment for Alexander disease, an ultra-rare inherited neurological disorder. The antisense oligonucleotide developer has been moving toward commercializing drugs on its own rather than seeking partners.
Pfizer is acquiring Metsera and its pipeline of obesity medications that could offer dosing and tolerability advantages for patients as well as a manufacturing edge for the pharma giant. Key data Phase 2 readouts for Metsera’s lead GLP-1 drug candidate are expected in 2026.
Keytruda Qlex, an injectable version of Merck’s cancer immunotherapy, can be administered in minutes compared to the 30 minutes needed to dose the original infused formulation. The FDA approval is based on clinical trial results showing the injectable formulation was comparable to infused Keytruda.
Roche is buying 89bio to get pegozafermin, a drug currently in two Phase 3 studies for the fatty liver disease MASH. Beyond the $2.4 billion payable when the deal closes, another $1.1 billion in contingent value right payments is tied to the achievement of commercial milestones for the projected blockbuster seller.
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AllRock Bio’s drug is a small molecule pan-ROCK inhibitor ready for Phase 2 testing in pulmonary arterial hypertension and pulmonary hypertension with interstitial lung disease. The startup is led by the management team that steered CinCor Pharma to a $1.3 billion acquisition by AstraZeneca.
With positive Phase 1 results for the metabolic disorder drug MZE782, Maze Therapeutics plans to advance the small molecule to Phase 2 tests in phenylketonuria and chronic kidney disease. Following the data readout, Maze raised $150 million in a private placement.
LB Pharmaceuticals found enough investor interest to boost the size of its IPO, which will finance clinical trials in schizophrenia and bipolar depression. The biotech has modified an old Sanofi drug with changes intended to reduce side effects and allow less frequent dosing.
A Johnson & Johnson cancer treatment that can spare certain bladder cancer patients from the radical step of surgical removal of the organ now has FDA approval. J&J’s drug/device combination product, Inlexzo, came from the 2019 acquisition of Taris Biomedical.
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Novartis is getting Tourmaline Bio’s pacibekitug, a Phase 3-ready antibody drug in development for atherosclerotic cardiovascular disease (ASCVD). Tourmaline licensed pacibekitug from Pfizer, which remains eligible for milestone payments tied to the progress of the drug.
Takeda Pharmaceutical’s oveporexton is designed to treat the root cause of narcolepsy. Statistically significant Phase 3 results for the twice daily pill were presented Monday during the World Sleep 2025 Congress in Singapore.
Despite achieving statistically signficant improvement in progression-free survival in North American and European patients, Summit’s drug ivonescimab fell short of the other Phase 3 goal measuring overall survival. It’s the first Phase 3 readout for an emerging class of bispecific antibodies that treat cancer by blocking two targets, PD-1 and VEGF.
FDA Commissioner Marty Makary said publishing complete response letters recognizes public interest in the transparency and credibility of agency decision-making about drugs. He also said the move complies with an executive order that covers the communication of scientific data.
Eschewing the traditional model of financing biotech startups milestone to milestone to support a lead asset, Treeline Biosciences’ well-funded pipeline is comprised of multiple complementary programs. Select investors have supported the approach of Treeline, which said it has raised more than $1.1 billion to date.