Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
FDA approves first RAS-targeting drug for pancreatic cancer
The FDA has approved Revolution Medicines’ Rasonque (daraxonrasib) for adults with advanced pancreatic adenocarcinoma, marking the first approved therapy designed to directly target a genetic driver of the disease. The once-daily oral RAS inhibitor is approved for patients with metastatic pancreatic cancer who have received at least one prior systemic therapy or are not candidates for multiagent treatment. In a randomized Phase 3 trial involving 500 previously treated patients, Rasonque more than doubled median overall survival compared with standard chemotherapy, extending it to 13.2 months versus 6.7 months. Although pancreatic cancer accounts for only around three percent of cancer diagnoses in the US, it is responsible for approximately eight percent of cancer deaths, with more than 50,000 people dying from the disease each year. Rasonque targets RAS proteins, which drive tumor growth in most pancreatic adenocarcinomas, and was granted Breakthrough Therapy, Orphan Drug, and Priority Review designations. “The approval was granted 6.5 months before the user fee deadline, demonstrating the FDA’s commitment to accelerating the approval of new cancer treatments for patients with serious and life-threatening conditions,” said Angelo de Claro, Director of the FDA’s Oncology Center of Excellence, in the press release. – Bree Foster
Another win for Amgen and AstraZeneca’s Tezspire
On Thursday, AstraZeneca and Amgen announced that their monoclonal antibody drug, Tezspire, hit all Phase 3 trial primary and key secondary endpoints in patients with eosinophilic esophagitis, a chronic immune mediated inflammatory disease of the esophagus. At both doses, Tezspire led to histologic remission and improvements in swallowing compared to placebo through 52 weeks. The results show the potential for the drug to have broad applications for epithelial-driven inflammatory diseases following its prior FDA approvals to treat severe asthma in 2021 and chronic rhinosinusitis with nasal polyps (CRSwNP) in 2025. “Epithelial science represents an important and rapidly evolving area in respiratory and immunology medicine, and we look forward to sharing these results at an upcoming medical meeting and with regulatory authorities as quickly as possible,” said Sharon Barr, Executive Vice President, BioPharmaceuticals R&D at AstraZeneca, in the press release. The results suggest Tezspire may soon be poised to compete with Dupixent from Regeneron and Sanofi, which currently has nine FDA approvals across allergic and inflammatory conditions. – Allison Whitten
Roche and Eli Lilly win FDA approval for Alzheimer’s blood test
The FDA has cleared another blood test to aid Alzheimer’s disease assessment, as blood-based biomarkers increasingly move into clinical practice. Developed by Roche in collaboration with Eli Lilly, the Elecsys pTau217 test measures phosphorylated tau 217 (pTau217), a biomarker associated with amyloid pathology, and is intended for adults aged 55 and older with signs or symptoms of cognitive decline. The test provides positive, intermediate, or negative results indicating the likelihood of amyloid pathology, potentially helping clinicians determine which patients may need further evaluation with PET imaging or cerebrospinal fluid testing. The clearance adds to a growing field of FDA-cleared Alzheimer’s blood tests. Fujirebio’s Lumipulse became the first in 2025, followed by Roche and Lilly’s pTau181 test later that year, and C2N Diagnostics’ PrecivityAD2 last week. Roche describes the pTau217 assay as the first blood test for Alzheimer’s disease that is designed to support both rule-in and rule-out assessment of amyloid pathology using a single biomarker and validated cutoffs, potentially simplifying Alzheimer’s testing across primary and specialty care. However, the test is not intended to diagnose Alzheimer’s disease on its own, with results requiring interpretation alongside clinical information and other diagnostic findings. – Bree Foster
New once-daily HIV pill clears FDA
The FDA approved a novel once-daily single-tablet HIV regimen this week, and it's a notable one: It's the first and only single-tablet option for virologically suppressed patients on complex, multi-pill regimens who couldn't switch to existing single-tablet therapies due to resistance or tolerability issues. The tablet pairs a guideline-recommended integrase strand transfer inhibitor with a first-in-class capsid inhibitor that has no cross-resistance to other antiretrovirals, giving doctors a new tool for patients with extensive treatment histories. Approval was based on two Phase 3 trials, one of which enrolled the oldest patient population in a Phase 3 HIV-1 treatment trial to date (median age 60, with a median 28 years of treatment experience). Both trials showed comparable efficacy in maintaining viral suppression through Week 48, no new safety concerns, and the most common side effects (headache, nausea, diarrhea) each hit under five percent of participants. Patients start with a two-day initiation dose paired with an existing injectable before moving to the new pill alone. For now, it's approved only in the US, with the company signaling more long-acting HIV options in its pipeline – Andrea Corona
SK Biopharmaceuticals to license Biohaven’s epilepsy therapy in $795M deal
In a deal worth up to $795 million in upfront and milestone payments, SK Biopharmaceuticals will partner with Biohaven with the goal of pushing their potassium channel opener, opakalim, towards a US commercial launch in 2029. Ahead of an important Phase 2/3 readout for the drug, the current deal hands SK the global license to opakalim and Biohaven’s full program of Kv7 voltage-gated potassium channel drugs. The deal cements SK’s commitment to the epilepsy space, with their drug Xcopri approved for focal seizures in 2019. “As this news has been long awaited, the agreement to in-license opakalim carries immense strategic significance to our company,” said Donghoon Lee, CEO of SK Biopharmaceuticals, in the statement. "This propels SK Biopharmaceuticals to another level – transitioning into a multi-product company that directly commercializes therapies in the U.S. market.” – Allison Whitten
FDA approves first ever targeted therapy for rare muscle and skin disease
The FDA has approved the first targeted therapy for dermatomyositis, a rare autoimmune disease marked by progressive muscle weakness and painful skin lesions, which has traditionally been managed with chronic high-dose steroids. The once-daily pill, a dual TYK2/JAK1 inhibitor, cleared trials on the strength of the largest dermatomyositis study ever conducted, testing two doses against placebo in 241 patients over a year. The high-dose group saw significantly greater improvement on a standard myositis scoring measure than placebo, with benefits appearing as early as four weeks and holding through the trial's end; more than half of treated patients hit meaningful improvement while cutting steroid use, compared to roughly a third on placebo. As a JAK-family drug, it carries an expected boxed warning for serious infections, death, malignancy, cardiovascular events, and thrombosis, with more common side effects including respiratory infections, headache, and fatigue. It's the developer's first FDA approval, and the company is now pushing the same molecule into late-stage trials for two additional rare inflammatory conditions. – Andrea Corona
