Curated news and analysis on clinical trials, drug approvals, and medical research.
Ocular Therapeutix stock surged 30% in premarket trading as the company prepared to release topline Phase 3 data for AXPAXLI in wet AMD. The SOL-1 trial results will be presented at the Macula Society meeting later in February. The company plans to file for FDA approval using one-year data if results are favorable.
The FDA issued final guidance advising drug companies to set duration limits for medically important antibiotics in livestock feed without legal obligation. Public health advocates criticize the guidance as insufficient to combat antibiotic resistance, which causes over 2.8 million infections and 35,000 deaths annually in the U.S.
The FDA agreed uniQure's existing trial data could support an accelerated approval filing for Huntington's therapy AMT-130, a reversal from earlier positions. Meanwhile, UK-based Harness Therapeutics selected HRN001 as its lead experimental candidate, targeting the FAN1 protein to address somatic expansion of Huntington's-causing genetic repeats.
The American College of Sports Medicine has published the first evidence-based exercise guidelines for children and adolescents with cancer, recommending safe, supervised activity during treatment. A new clinical trial is also evaluating structured exercise for young sarcoma patients undergoing chemotherapy to improve outcomes and quality of life.
NMD Pharma reported Phase 2a trial data showing ignaseclant improved muscle strength and motor function in adults with Charcot-Marie-Tooth disease, with benefits lasting seven days after treatment. The study met key secondary endpoints but missed its primary endpoint of six-minute walk test improvement. The drug has received FDA orphan drug designation for CMT.
Two neuroscience studies reveal brain mechanisms behind anxiety and compulsive behavior. One found restoring balance in an amygdala circuit reversed anxiety in mice, while another showed inflammation in the striatum makes behavior more deliberate.
The FDA has approved WAKIX for cataplexy in pediatric narcolepsy patients, making it the first non-scheduled treatment for all ages. Harmony Biosciences reported 2025 WAKIX revenue of $868.5 million and projects 2026 sales over $1 billion, nearing blockbuster status.
Acurx Pharmaceuticals is advancing ibezapolstat to international Phase 3 clinical trials for C. difficile infection following final regulatory guidance from FDA and EMA. The company launched a new clinical trial program for recurrent CDI and presented structural biology research demonstrating ibezapolstat's mechanism of action. Acurx reported year-end 2025 cash of $7.6 million.
Sensei Biotherapeutics has changed its name to Faeth Therapeutics following its February 2026 acquisition of the biotechnology company. The name change accompanies a $200 million private placement financing to advance PIKTOR, an investigational all-oral multi-node inhibitor targeting the PI3K/AKT/mTOR pathway in endometrial and breast cancer. Topline Phase 2 data in endometrial cancer is anticipated in the second half of 2026.
The U.S. FDA has accepted Gilead's application for an investigational once-weekly oral lenacapavir for HIV prevention, with a review date set for February 2027. Concurrently, PEPFAR and The Global Fund are expanding access to the twice-yearly injectable version to a total of 3 million people by 2028. Gilead will present new data on both lenacapavir and a novel bictegravir/lenacapavir combination at CROI 2026.
HHS and CMS issued a proposed rule to codify the Medicare Drug Price Negotiation Program, largely formalizing existing guidance. The rule includes new provisions, such as clarifications for new drug formulations. The proposal is now open for public comment.
The FDA has approved three new targeted therapies for different stages of advanced prostate cancer: Truqap for PTEN-deficient tumors, rucaparib for BRCA-mutated castration-resistant disease, and Akeega for BRCA2-mutated castration-sensitive cancer.
Grifols has launched FESILTY (fibrinogen, human-chmt) in the U.S., a fibrinogen concentrate FDA-approved in December 2025 for acute bleeding episodes in patients with congenital fibrinogen deficiency. The highly purified product, developed by Biotest, enables rapid fibrinogen restoration and can be reconstituted in approximately three minutes.
A genetic framework using Mendelian randomization identified and prioritized drug targets for major depression from over 525,000 cases. The study found drugs targeting genetically supported proteins are 2.6 times more likely to succeed and prioritized 54 repurposing opportunities using existing medications for other conditions.
The FDA has granted accelerated approval to Denali Therapeutics' Avlayah (tividenofusp alfa-eknm), the first therapy targeting neurological symptoms of Hunter syndrome. The approval was based on a surrogate endpoint measuring heparan sulfate reduction in cerebrospinal fluid, with confirmatory study results required for full approval. The global Hunter syndrome treatment market is projected to reach $2.6 billion by 2033.
The FDA has cleared an IND for Amphista's novel BRD9 degrader AMX-883 for AML, approved Lantheus' new prostate cancer imaging agent Pylarify TruVu, and accepted Biofrontera's sNDA for Ameluz PDT in superficial basal cell carcinoma. These regulatory milestones span hematologic and solid tumor areas, addressing significant unmet medical needs.
Akeso reported overall survival benefit for ivonescimab plus chemotherapy in Phase III lung cancer study. The company enrolled first patient in Phase Ib/II trial of next-generation HER3 ADC AK138D1 for advanced breast cancer. Akeso also received China approval to start Phase II trials for AK139 bispecific antibody.
New research shows posture-driven retinal vascular changes can predict diabetic retinopathy progression, with smartphone-based imaging detecting early microvascular dysfunction. A five-year study found abnormal arteriolar tortuosity during postural shifts increased progression risk over two-fold.
The U.S. FDA has accepted for filing a New Drug Application for tirabrutinib under the accelerated approval pathway for relapsed or refractory primary central nervous system lymphoma, setting a PDUFA action date of December 18, 2026. The submission is supported by Phase 2 PROSPECT study results showing a 67% overall response rate and 44% complete response rate. If approved, tirabrutinib would be the first BTK inhibitor commercially available in the U.S. for this indication.
At the ADA Scientific Sessions, Novo Nordisk's Wegovy pill surpassed 3 million prescriptions within five months, while Structure Therapeutics and AstraZeneca shared mid-stage GLP-1 data targeting a 2029 launch. The US FDA approved TRUQAP as the first targeted treatment for PTEN-deficient metastatic prostate cancer, reducing disease progression risk by 19% in the CAPItello-281 trial. Meanwhile, two major genomic atlas projects—a 4,098-genome infant gut bacteria catalog and Basecamp Research's Trillion Gene Atlas—aim to advance precision probiotics and AI-driven drug discovery, respectively.
