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.
Vistagen announced topline results from a repeat dose study of fasedienol for acute treatment of social anxiety disorder, showing favorable safety and tolerability profiles along with efficacy signals across primary and secondary endpoints in both repeat and single dose administrations.
Tarsus Pharmaceuticals agreed to acquire privately-held Alkeus Pharmaceuticals for $450 million upfront and up to $800 million total, gaining gildeuretinol (ALK-001), a Phase 3 oral therapy for Stargardt disease. The rare genetic eye condition affects over 36,000 people in the U.S. and currently has no FDA-approved treatment, with pivotal trial data expected in late 2029.
British researchers have conducted the first successful clinical trials of vaccines that do not require refrigeration, a breakthrough that could address the problem of half of all vaccines being discarded due to cold chain failures.
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.
A University of Colorado Anschutz-led randomized clinical trial found that an automated oxygen delivery system kept hospitalized patients in their target oxygen range 85% of the time, compared to 63% with manual adjustments by healthcare staff.
Novo Nordisk's experimental inflammation-targeting drug failed to reduce the risk of heart attacks and strokes in a closely watched large clinical trial, a setback for the drugmaker's growth prospects beyond obesity and diabetes.
Latigo has announced successful mid-stage clinical trial results for its non-opioid pain medication, positioning it as a potential competitor to Vertex Pharmaceuticals' pain drug Journavx in a field historically marked by development failures.
Hansoh Pharmaceutical announced positive results from the ARTEMIS-011 Phase III trial in China, with its experimental drug Ris-Rez meeting the primary endpoint of progression-free survival in relapsed bone cancer patients. This marks the second Phase III success for the drug, strengthening GSK's oncology portfolio through its licensing partnership with the Chinese biotech.
Innocare Pharma announced its experimental oral drug for moderate-to-severe plaque psoriasis showed consistent treatment effects in late-stage clinical trials, entering a competitive market where recent head-to-head studies have compared efficacy of various psoriasis treatments.
AstraZeneca announced Monday that its rare disease drug Ultomiris failed a late-stage trial for blood vessel complications following stem cell transplants, while a separate trial of its Claudin 18.2-targeting antibody-drug conjugate in stomach cancer succeeded. The Ultomiris setback adds to concerns about the drugmaker's pipeline following a series of setbacks this year.
Phase III CLARITY-Gastric01 trial results showed sonesitatug vedotin (Sone-Ve) achieved statistically significant overall survival improvement in CLDN18.2-positive advanced gastric, gastroesophageal junction, or esophageal adenocarcinoma patients in second-line and later settings, meeting its dual primary and key secondary endpoints.
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.
The European Commission has approved AstraZeneca's Etcamah (camizestrant) in combination with a CDK4/6 inhibitor for first-line treatment of adults with ER-positive, HER2-negative advanced breast cancer with emergent ESR1 mutations. The approval is based on SERENA-6 Phase III trial results showing a 56% reduction in risk of disease progression or death.
GSK discontinued development of an experimental chronic cough treatment after it failed to meet key goals in two late-stage clinical trials, with the company stating it was unlikely to transform patient care. Shares fell about 4%.
The Serum Institute of India has signed an agreement with the Gates Medical Research Institute to manufacture M72/AS01E, a novel tuberculosis vaccine candidate currently in late-stage clinical trials. If approved, it would be the first new TB vaccine in over 100 years, with Serum Institute planning to invest over $100 million in production.
Researchers found that semaglutide, the active ingredient in Ozempic and Wegovy, slowed biological aging markers in adults with HIV, marking the first clinical evidence that the drug may influence human aging beyond its metabolic effects.
An experimental drug called diranersen demonstrated the ability to lower levels of tau protein in the brain and slow cognitive decline in early Alzheimer's patients, offering a new treatment approach distinct from current therapies. Clinical trial data released July 14, 2026, showed the drug can reduce tau tangles, one of the hallmark proteins associated with Alzheimer's disease.
Researchers report safety and preliminary efficacy results from a phase Ib clinical trial testing personalized neoantigen-pulsed autologous dendritic cell vaccination in patients with newly diagnosed glioblastoma, a deadly brain cancer with limited treatment options.
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.
BeOne Medicines announced positive Phase 3 results from the MANGROVE trial, showing Brukinsa plus rituximab reduced the risk of disease progression or death by 43% compared to bendamustine plus rituximab in frontline mantle cell lymphoma patients. The combination represents a potential chemotherapy-free treatment option.
The EPCORE DLBCL-4 Phase 3 trial showed that epcoritamab (Epkinly) combined with lenalidomide significantly improved progression-free survival compared to R-GemOx chemotherapy in adults with relapsed or refractory diffuse large B-cell lymphoma who received at least one prior line of therapy. The results bring the bispecific antibody closer to a potential label expansion.
Texas-based biotech Ollin Biosciences secured $330 million in funding to advance late-stage clinical studies of its experimental eye disease treatment, representing one of the largest recent funding rounds in ophthalmology.
The United States has supplied doses of an experimental antibody drug from Mapp Biopharmaceutical to support clinical trials addressing the expanding Ebola outbreak in the Democratic Republic of Congo, according to a Health Department spokesperson.
Eli Lilly provided its experimental 'triple-G' weight loss drug retatrutide to a 79-year-old patient through the FDA's compassionate use program, marking an extremely rare instance of pre-approval access to an obesity medication.
The U.S. Food and Drug Administration announced a series of measures designed to speed up drug research across all stages, from early investigational studies through late-stage clinical trials, in an effort to compete with China and develop treatments more quickly.
The FDA has approved the combination of belzutifan (Welireg) and pembrolizumab (Keytruda) as adjuvant treatment for patients with clear cell renal cell carcinoma at high risk of recurrence following nephrectomy, based on data from the phase 3 LITESPARK-022 trial.
The first participant has been treated in a groundbreaking clinical trial testing cellular reprogramming technology designed to rejuvenate aging cells. This marks the first time such reverse-aging therapy has been administered to a human.
AstraZeneca announced results from a phase II trial showing its oral GLP-1 therapy reduced body weight by 10.5%, positioning the company to compete with Eli Lilly in the race to develop convenient pill-based obesity treatments. The drug is advancing to late-stage trials.
ADC Therapeutics shares plunged 53% in premarket trading after a late-stage study of its blood cancer drug showed more deaths among treated patients, despite meeting its main goal. The safety signal represents a significant setback for the company's development program.
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.
J&J's Erleada used with hormone-blocking therapy six months before and after prostate surgery improved cancer elimination rates and reduced the risk of cancer spread and death in a late-stage trial for prostate cancer patients.
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.
Novartis reported early data showing its experimental actinium-based radiopharma drug demonstrated anti-tumor activity against prostate cancer, including in patients previously treated with its older therapies.
Bristol Myers Squibb announced its experimental oral drug mezigdomide successfully delayed disease progression in a late-stage trial of multiple myeloma patients whose illness had relapsed or not responded to prior treatments.
Agios Pharmaceuticals announced it will stop developing its experimental drug for a form of blood cancer after a mid-stage trial failed to show sufficient benefit, marking a setback for the rare disease program.
Eli Lilly reported that a high dose of its gene-editing therapy from Verve Therapeutics reduced cholesterol levels by 62% in participants in an early-stage clinical trial, representing a significant advance in gene-editing treatments for cardiovascular disease.
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.
Five years after a disastrous trial where some boys died while others had astonishing recoveries, Astellas is returning with a revised gene therapy treatment for X-linked myotubular myopathy (XLMTM), offering new hope to a devastated rare disease community.
Johnson & Johnson's closely watched inflammatory bowel disease therapy failed to show statistically significant improvement in patients during clinical trials, representing a setback for the pharmaceutical giant's gastroenterology pipeline.
Setidegrasib, an investigational targeted therapy drug, demonstrated ability to slow cancer growth and shrink tumors in a first-in-human clinical trial published in the New England Journal of Medicine, targeting a previously difficult-to-treat cancer mutation.
A clinical trial of 108 adults with obesity and alcohol use disorder found that once-weekly semaglutide injections reduced heavy drinking days in the past 30 days by an average of roughly 12 days, representing a 50% reduction compared to placebo.
A major international clinical trial found that a minimally invasive heart procedure may be a better first-line treatment than medication for people with advanced forms of atrial fibrillation, offering improved outcomes for patients with this common heart rhythm disorder.
Two studies report that targeted, noninvasive radiation therapy (proton beam therapy) reduced episodes of life-threatening cardiac arrhythmias by nearly 80% in critically ill patients, representing the first large-scale and first-in-human evidence for this approach.
Intellia Therapeutics announced that its CRISPR-based gene editing treatment reduced swelling attacks in a pivotal trial for hereditary angioedema, a rare genetic disease. The results advance the CRISPR field's one-and-done treatment approach toward potential regulatory approval.
A major clinical trial found that real-time continuous glucose monitoring (CGM) significantly improves blood glucose management in adults with type 2 diabetes treated with basal insulin, offering evidence for expanded use of the technology beyond type 1 diabetes.
Two major clinical trials led by St. Michael's Hospital and Oxford University found that Paxlovid does not reduce hospital admissions or deaths in vaccinated adults at higher risk of severe COVID-19, though it helps them recover faster.
A global clinical trial found that GMRx2, a single pill combining three low-dose blood pressure medications, significantly reduced the risk of recurrent stroke in patients with intracerebral hemorrhage and high blood pressure by approximately 40%.
International clinical trial results show that gene therapy for a rare form of genetic deafness successfully restored hearing in 90% of participants, with improvements lasting years after treatment. The study was co-led by Mass General Brigham and China's Eye & ENT Hospital of Fudan University.
Swiss drugmaker Roche presented latest clinical trial data for its experimental multiple sclerosis drug at a major conference, setting the stage for regulatory approval submissions, though safety questions remain under scrutiny.
Kyverna Therapeutics announced plans to submit its one-time CAR-T cell therapy for stiff person syndrome to the FDA after late-stage trial showed improvements in mobility and reduced disabilities in patients with the rare neurological disease.
Clinical trial results demonstrate that immunotherapy as first-line treatment provides clinical benefit for patients with advanced colorectal cancer, offering an alternative to chemotherapy for a disease increasingly affecting younger patients.
BioAge Labs announced its investigational pill for cardiovascular risk prevention significantly reduced inflammation markers in an early-stage clinical study, representing potential progress in heart disease prevention.
Early trial results presented at AACR show CAR-T cell therapy may prevent smoldering multiple myeloma from progressing to active disease in high-risk patients, representing a potential preventive approach to cancer treatment.
A landmark trial presented at ESCMID Global 2026 demonstrates that improving oral hygiene for hospital patients can reduce the risk of non-ventilator-associated hospital-acquired pneumonia by 60%, offering a simple intervention to prevent a serious complication.
India has approved new tuberculosis vaccines for adolescents, backed by a large, independent Phase III clinical trial led and funded by public science institutions. The vaccines represent a significant breakthrough in TB prevention, though officials note they are not a complete solution.
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.
Global phase III TROPION-Breast02 trial led by National Cancer Centre Singapore shows datopotamab deruxtecan significantly improves survival outcomes in triple-negative breast cancer patients, representing a major breakthrough for this aggressive cancer type.