Gene therapy, CRISPR, synthetic biology and emerging platforms. Separating what is real from what is hype, and what actually translates.
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Sep 24, 2026The result fell short of investor expectations, with a p-value of 0.0603 and an effect size below what TD Cowen had modeled. Acadia and BMO still see enough efficacy and safety support to keep developing remlifanserin, while dropping the lower 30-mg arm from the ongoing phase 3 program.
Sep 23, 2026The Fusion study met its primary endpoint, with patients on ulefnersen performing significantly better than placebo on a Week 72 measure that combined functional impairment and survival. The readout matters scientifically because it targets a defined disease driver, but its business impact is likely to be limited by the very small patient population.
Sep 22, 2026The Embarq CSU1 and CSU2 studies enrolled 1,939 patients and showed significant improvements in itch and hive scores at week 12, along with higher complete response rates versus placebo. For Celldex, the readout shifts attention back to mast cell biology in CSU and toward a planned FDA submission in 2027.
Sep 21, 2026The midstage dataset gives Cue a clinical basis for advancing an asset it licensed for ex-China rights in April for $15 million. While the Xolair comparison was not statistically tested, the longer follow-up is the part most likely to shape whether CUE-221 can claim a distinct profile in CSU.
Sep 21, 2026In the 85-patient Vista study, 24.1% of patients in the low-dose arm and 31% in the high-dose arm met the responder definition for improved visual acuity under low light, versus none in the control group. Beacon said it plans to start and finish a rolling biologics license application to the FDA before the year’s end, while also pursuing approval in the European Union and the U.K.
Sep 19, 2026Artelo’s preclinical update suggests a non-incretin mechanism may have activity in obesity without affecting body composition in lean mice. The strategic question is whether that signal is strong enough to turn a repurposed oncology-supportive asset into a partnerable obesity program alongside GLP-1 drugs.
Sep 18, 2026The biotech is continuing treatment for patients already enrolled in X-Nova2 in major depressive disorder and X-Ceed in bipolar depression. Management said the safety events were mostly mild or moderate and expects to modify dosing over the coming months after notifying the FDA.
Sep 14, 2026In the Panorama study, a 100 microgram dose of DT120 reduced Hamilton Anxiety Rating Scale scores by 9.8 points at week 12, versus 4.7 points for placebo. Definium also used a 50 microgram arm to address functional unblinding concerns that have become a live regulatory issue in psychedelic development.
Sep 14, 2026The Rezilient3 interim analysis showed statistically significant progression-free survival and higher response rates for zipalertinib combined with chemotherapy in previously untreated advanced non-small cell lung cancer with EGFR exon 20 insertion mutations. With a later-line FDA decision expected next February, Taiho is now planning discussions with global health authorities and the FDA about the front-line data.
Sep 11, 2026The study focused on cisplatin and carboplatin in children who received platinum therapy followed by surgery to remove liver tumors. Its immediate implication is not a change to current use of these drugs, but a stronger basis for long-term monitoring and, eventually, for designing treatments that spare healthy tissue more effectively.
Sep 9, 2026The new company was created specifically to move a next-generation menin inhibitor into diabetes, with funding led by BVF Partners and participation from Eli Lilly, Kura and other investors. Caspian also plans to advance a second menin program for diabetes and cardiometabolic conditions while preparing KO-7246 for IND-enabling work and initial clinical proof-of-concept.
Sep 8, 2026In CHAPTER-3, Pharvaris reported an 83% reduction in monthly hereditary angioedema attack rate versus placebo after 24 weeks, rising to 87% in patients with HAE type 1 or 2. The result strengthens a two-formulation strategy built around immediate-release deucrictibant for on-demand use and an extended-release version for prevention.
Machine learning, predictive models and computational biology: how AI is changing discovery workflows and probability of success.
Drug development, clinical trials and industry dynamics. What changes timelines, risk and competitive positioning.
Where capital is flowing and why the tape moves, across funding rounds, M&A, IPOs and market analysis.
Digital health, diagnostics and medical devices, and the signals that matter for adoption, reimbursement and outcomes.
FDA actions, approvals, policy shifts and compliance: how the rules change timelines and probability.