Vanda Pharmaceuticals' experimental drug VQW-765 for social anxiety disorder failed the main goal of a late-stage study in female patients, prompting the company to put the program on hold pending further analysis.
Immunovant has discontinued development of IMVT-1402 for cutaneous lupus erythematosus after the anti-FcRn antibody failed to meet its primary endpoint in a proof-of-concept study of 57 patients. The company's stock fell 6% following the announcement.
Roche's experimental RNA drug sefaxersen significantly reduced proteinuria in a late-stage trial for IgA nephropathy, positioning the company to bring a new treatment to market for the autoimmune kidney disease that currently has limited approved therapies.
Amgen reported positive Phase 3 results for dazodalibep in moderate-to-severe Sjögren's disease, a chronic autoimmune condition with no currently approved therapies. The drug was acquired through Amgen's $28 billion Horizon Therapeutics buyout.
Vertex Pharmaceuticals announced positive results from its phase 2b AMPLIFIED study of inaxaplin in people with APOL1-mediated kidney disease (AMKD), including patients with modest proteinuria and those with type 2 diabetes. The company is positioning the drug for a broad kidney disease label ahead of a key readout next year.
First-line ivonescimab significantly prolonged overall survival compared with pembrolizumab in patients with PD-L1-positive advanced non-small cell lung cancer, according to updated analysis from a clinical trial.
Beacon Therapeutics reported its experimental gene therapy improved low-light reading ability in a late-stage trial for X-linked retinitis pigmentosa (XLRP), a rare inherited eye disease that can cause blindness. The company succeeded where larger drugmakers including Johnson & Johnson and Biogen have failed.
Otsuka Pharmaceutical presented new phase 3b clinical trial results for SIMTRIYO (centanafadine) at Psych Congress 2026, demonstrating consistent improvements across ADHD symptoms, anxiety, executive function, and emotional dysregulation in adults with ADHD and comorbid anxiety disorders.
Once-daily injectable vosoritide demonstrated significantly larger increases in annualized growth velocity compared with placebo among children with hypochondroplasia, according to phase 3 trial data published in NEJM Evidence.
French biotech Axoltis Pharma announced that its Phase 2 clinical trial for an experimental ALS treatment failed to meet its primary endpoints. The company suggested it may have found a small signal to potentially continue development.
Definium Therapeutics reported that its oral LSD-based treatment DT120 reduced symptoms of generalized anxiety disorder in a second successful late-stage study, marking the company's third Phase 3 win this year for the candidate.
AstraZeneca announced that its breast cancer pill failed to meet the primary endpoint of improving progression-free survival in a late-stage clinical trial. The setback came just one week after the treatment received U.S. regulatory approval.
Ultragenyx Pharmaceuticals' apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The failure of what was once considered a potential blockbuster therapy is forcing Ultragenyx to examine significant cost cuts.
AbbVie announced positive Phase 3 results for etentamig, a dual-acting T cell engager for multiple myeloma, showing survival benefits with a cleaner safety profile compared to competing BCMA-targeting therapies from Johnson & Johnson, Pfizer and Regeneron. The company is seeking to enter the crowded multiple myeloma treatment market.
Tezspire succeeded in a Phase 3 trial for eosinophilic esophagitis, potentially becoming the drug's third approved indication and positioning it to compete with Sanofi's Dupixent, currently the only approved biologic for the condition.
Innovent Biologics has initiated a registrational phase 3 study evaluating IBI363, a PD-1/IL-2α bispecific fusion protein, combined with bevacizumab in 550 patients with treatment-refractory colorectal cancer.
Taiho Oncology and Cullinan Therapeutics announced their next-generation EGFR inhibitor zipalertinib significantly improved progression-free survival in a registrational phase 3 trial for first-line treatment of advanced non-small cell lung cancer. The results position the drug as a competitor to multiple approved medicines in the EGFR-mutated lung cancer market.
Definium Therapeutics announced its experimental LSD-based pill significantly reduced anxiety symptoms in a late-stage study, meeting the trial's main goal. The results mark the company's second major win in two months and sent shares up more than 20% in premarket trading.
MoonLake Immunotherapeutics announced its experimental drug sonelokimab met all primary and secondary endpoints in the Phase 3 IZAR-1 trial for psoriatic arthritis in bio-naïve patients, achieving a 42.1% ACR50 response rate at Week 16. Despite the positive clinical results, the company's stock gapped down on Monday, opening at $17.24 after closing at $18.26.
Swedish Orphan Biovitrum (Sobi) and Innate Pharma announced a strategic partnership worth up to $580 million, with $75 million upfront, to advance lacutamab into the TELLOMAK-3 Phase 3 confirmatory trial for cutaneous T-cell lymphoma, specifically Sézary syndrome. Innate Pharma also appointed Markus Jensen as Chief Medical Officer.
AbCellera Biologics reported that its non-hormonal menopause treatment ABCL-635 significantly reduced the frequency and severity of hot flashes in a 92-person Phase 2 trial, with the drug meeting both primary endpoints after four weeks. The results sent the company's shares surging nearly 40% in early trading.
Silence Therapeutics announced that its Phase 2 SANRECO trial of divesiran met its primary endpoint in patients with polycythemia vera, a rare blood cancer, with data showing the treatment stabilized red blood cell levels and reduced phlebotomies to 0.2 per patient over 36 weeks. The company's stock rallied significantly on the news.
Intellia Therapeutics announced second quarter 2026 financial results alongside positive Phase 3 clinical data for lonvo-z in hereditary angioedema (HAE), with FDA biologics license application acceptance anticipated in late 2026 and U.S. commercial launch planned for first half of 2027.
UK regulators have authorized the INTERCEPT-Lynch Phase 1/2 trial of mRNA-4194, a preventive cancer vaccine developed by Moderna and the University of Oxford targeting people with Lynch syndrome, an inherited condition affecting 1 in 300 people with up to 80% lifetime cancer risk. The trial will begin dosing in summer 2026.
Merck's anti-TL1A antibody from its 2023 Prometheus Biosciences acquisition delivered positive mid-stage results in hidradenitis suppurativa but failed to meet endpoints in a rare lung disease trial. The company disclosed the mixed Phase 2 results alongside its second-quarter earnings.
The LiBBY clinical trial found that a combination of CBD and THC rapidly and significantly reduced agitation in hospice-eligible dementia patients, with nearly 90% of participants showing behavioral improvement in the nationwide study.
A major U.S. clinical trial found that silver diamine fluoride, a liquid brushed onto cavities in seconds, stopped decay in more than half of treated cases. The treatment could spare many young children from pain, infection, and surgery.
Eli Lilly announced positive results from two additional Phase 3 trials of its experimental obesity drug retatrutide (a 'triple-G' GLP-1 receptor agonist), showing it did not increase overall heart risk. The company plans to submit an FDA application in Q1 2027.
A Phase 1 clinical trial in Japan reported no tumor formation or graft-related adverse events after transplanting iPSC-derived neural stem/progenitor cells into four patients with subacute complete cervical spinal cord injuries during two to four years of follow-up. The small, uncontrolled study was designed primarily to assess safety and was not able to establish treatment efficacy.
Gilead Sciences and Merck announced that their experimental once-weekly HIV pill combining islatravir and lenacapavir successfully maintained viral suppression at 48 weeks in two Phase 3 trials (ISLEND-1 and ISLEND-2) involving 1,209 adults with virologically suppressed HIV. The results support regulatory filings for what could become the first once-weekly oral HIV treatment regimen.
Roche announced positive results from a head-to-head Phase 3 trial showing its KRAS G12C inhibitor divarasib demonstrated superior efficacy compared to competing treatments from Amgen and Bristol Myers Squibb in patients with KRAS-driven lung cancer.
The New England Journal of Medicine retracted the main clinical trial used to approve Amgen's $500 million drug Tavneos, following allegations of data manipulation. The European Medicines Agency had previously recommended revoking the drug's approval over data integrity concerns.
Longevity-focused biotech startup NewLimit announced a $435 million Series C funding round as it prepares to launch its first clinical trial of a liver medicine, marking one of the largest recent raises in the longevity space.
New clinical trial results presented at the American Society of Clinical Oncology show that Moderna's experimental personalized mRNA vaccine, combined with immunotherapy, significantly reduced the risk of deadly melanoma returning after five years. The vaccine is custom-made for each patient based on their specific tumor.
Ivonescimab, an experimental drug combining two immunotherapy mechanisms, reduced the risk of death by 34% compared to standard immunotherapy in patients with advanced squamous non-small cell lung cancer in a closely watched Chinese trial. The drug helped patients live 15% longer than BeOne Medicines' Tevimbra.
Clinical trial results show that daraxonrasib, an experimental daily pill, nearly doubled overall survival rates for patients with advanced pancreatic cancer compared to traditional chemotherapy. Medical experts are calling the results a 'game changer' for treating one of the deadliest forms of cancer.
GSK reported that its experimental chronic hepatitis B drug helped nearly one in five patients achieve a functional cure in late-stage studies, vastly outpacing current treatments and raising hopes for a finite treatment course as an alternative to lifelong therapy.
Eli Lilly announced positive results from its Phase 3 TRIUMPH-1 trial showing retatrutide, an experimental triple-agonist obesity drug, achieved average weight loss of 28.3% at 80 weeks and 30.3% at 104 weeks on the highest dose, matching weight reduction levels typically seen with bariatric surgery.
AstraZeneca's VOLGA trial confirms that the Imfinzi EV combination significantly improves overall survival for cisplatin-ineligible bladder cancer patients, representing a major advancement in treatment for this difficult-to-treat population.
UPMC and University of Pittsburgh researchers successfully weaned multiple liver transplant patients off all immunosuppressant drugs for over three years using a pre-transplant cell therapy approach, representing a potential breakthrough in organ transplantation.
A major multi-site clinical trial co-led by Washington University School of Medicine demonstrates that a procedure can relieve post-thrombotic syndrome, a common and painful complication following deep vein thrombosis.
An experimental drug designed to silence a gene strongly linked to Parkinson's disease showed encouraging effects in reducing the target protein by up to 60% in a first-in-human clinical trial published in Nature Medicine.
A phase 1 clinical trial demonstrated that stem cell transplantation using gene-edited cells rapidly and effectively restored hemoglobin production in five patients with beta-thalassemia, allowing them to discontinue blood transfusions.
