Treatments & Trials
Drugs, therapies and clinical trials, with the trial phase and who reported the results.
Lilly's Retatrutide Beats Semaglutide on A1C and Weight in Head-to-Head Phase 3 Trial
Company-reported TRANSCEND-T2D-2 results in 1,298 adults with type 2 diabetes also showed higher discontinuation rates for side effects on the investigational triple agonist.
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More in Treatments & Trials
Pfizer's Elrexfio Meets Phase 3 Progression-Free Survival Goal in Relapsed Myeloma
Topline MagnetisMM-32 results compared the BCMA bispecific antibody with three standard regimens in patients previously treated with anti-CD38 therapy and lenalidomide.
FDA Approves GSK's Hibsago, First Antisense Drug for Chronic Hepatitis B
The six-month injectable course is added to existing antiviral therapy, and in Phase III trials some patients were able to stop all hepatitis B treatment.
Disc's Bitopertin Meets All Endpoints in Pivotal Phase 3 Trial After FDA Rejection
The company plans to resubmit its application for the oral drug in erythropoietic protoporphyria by the end of 2026, months after the FDA turned down an accelerated approval request.
FDA Approves Genentech's Enspryng as Subcutaneous Treatment for Thyroid Eye Disease
The approval follows a Phase III program in which one of two identically designed trials missed its primary endpoint, according to the company.
Bristol Myers' Alpha-Emitting RYZ101 Meets Phase 3 Goal in Neuroendocrine Tumors
An interim analysis of the ACTION-1 trial showed improved progression-free survival after lutetium-177 therapy, the company said, but it did not release the size of the benefit.
FDA Approves Tecentriq With Chemotherapy After Surgery for Stage III dMMR Colon Cancer
Genentech said the approval rests on the NCI-sponsored Phase III ATOMIC trial, in which adding the immunotherapy to chemotherapy halved the risk of recurrence or death.
Vaxcyte's 31-Valent Pneumococcal Vaccine Meets Primary Goals in Phase 3 OPUS-1 Trial
The company-reported topline data compare VAX-31 with Pfizer's Prevnar 20 and Merck's Capvaxive in more than 4,000 U.S. adults and will anchor a planned FDA filing.
FDA Approves AbbVie's Juvmo, Which It Says Is the First D1/D5 Agonist for Parkinson's
The once-daily tavapadon tablet can be taken with or without levodopa, and AbbVie expects it to reach U.S. patients in October.
Kodiak's Zenkuda and Tabirafusp-ted Match Aflibercept in Phase 3 Wet AMD Trial
Both eye drugs met the DAYBREAK trial's noninferiority goal for vision at one year, Kodiak said, and the company plans to file Zenkuda for approval in the fourth quarter.
FDA Approves Lilly's Onswik, a Once-Weekly Basal Insulin for Type 2 Diabetes
The approval rests on four Phase 3 QWINT trials against daily insulins and makes Onswik the second weekly basal insulin approved in the U.S. after Novo Nordisk's Awiqli.
Otsuka and Ionis' Ulefnersen Meets Primary Goal in Phase 3 FUS-ALS Trial
The antisense drug improved a combined measure of function and survival versus placebo in a rare genetic form of ALS, the companies said, and they plan to discuss an expedited filing with the FDA.
FDA Approves Ultragenyx's Fayuvi, First Gene Therapy for Sanfilippo Syndrome Type A
The one-time AAV9 infusion received full rather than accelerated approval, and Ultragenyx set a wholesale price of $3.95 million.
Definium's LSD-Based DT120 Meets Goal in Second Phase 3 Anxiety Trial
A single dose cut anxiety scores 5.1 points more than placebo at 12 weeks in the Panorama study, the company said, and it plans to file for approval in the first half of 2027.
FDA Approves Scholar Rock's Isembyld, First SMA Therapy Aimed at Muscle Loss
The myostatin-blocking antibody is added to existing SMN2-targeted treatment, and the approval comes after a 2025 rejection tied to a contract manufacturing plant.
Amgen's Imdelltra With Imfinzi Improves Survival in Phase 3 First-Line SCLC Trial
The DeLLphi-305 study met its overall survival goal at an interim analysis, Amgen said, though the company has not yet released the numbers.
Novartis' Pelacarsen Misses Primary Goal in Phase 3 Lp(a)HORIZON Outcomes Trial
The antisense drug lowered lipoprotein(a) but did not reduce major cardiovascular events against placebo in 8,323 patients with established heart disease, the company said.
FDA Approves Ionis' Zanvastro, First Treatment for Alexander Disease
The antisense drug zilganersen, injected into the spinal canal every three months, is approved for children and adults on data from a randomized study that measured walking speed and motor skills.
FDA Approves Revolution Medicines' Rasonque for Metastatic Pancreatic Cancer
The once-daily RAS inhibitor daraxonrasib extended median overall survival to 13.2 months from 6.7 months on chemotherapy in a 500-patient Phase 3 trial, and the agency acted 6.5 months before its user-fee deadline.
FDA Approves J&J's Imaavy for Warm Autoimmune Hemolytic Anemia
The FcRn-blocking antibody nipocalimab, already approved for generalized myasthenia gravis, won its second indication on Phase 2/3 data showing about three times as many durable hemoglobin responses as placebo, the company said.
FDA Grants Accelerated Approval to Ultragenyx's Genglycos Gene Therapy for GSDIa
The one-time AAV8 gene therapy, the first approved treatment for glycogen storage disease type Ia according to the FDA, was approved on a 31% reduction in daily cornstarch intake.
Merck and Moderna's Intismeran Meets Phase 3 Recurrence Goal in Resected Melanoma
The individualized mRNA therapy plus Keytruda improved recurrence-free and distant metastasis-free survival over Keytruda alone at an interim analysis, the companies said, without yet disclosing the size of the effect.
FDA Approves Bristol Myers' Zenbexus Combination for Relapsed Multiple Myeloma
The accelerated approval of oral iberdomide with daratumumab and dexamethasone rests on minimal residual disease data from the Phase 3 EXCALIBER-RRMM trial, and continued approval depends on confirmatory trials.
Tenax's Oral Levosimendan Misses Primary Goal in Phase 3 LEVEL Heart Failure Trial
TNX-103 did not improve six-minute walk distance over placebo in pulmonary hypertension due to HFpEF, the company said, and it will ask the FDA about an enriched trial population.
FDA Approves Replimune's Tudriqev for Advanced Melanoma After Two Rejections
The oncolytic virus therapy, given with nivolumab, received accelerated approval based on a 24% response rate in the single-arm IGNYTE trial, a week after advisers voted 10 to 3 in its favour.
FDA Approves Takeda's Orzeyful, First US Orexin Drug for Narcolepsy Type 1
The twice-daily oveporexton tablet activates the orexin receptor 2; it will reach patients after the DEA decides how to schedule it, which Takeda expects within 90 days.
Novo Nordisk's Ziltivekimab Fails to Cut Cardiovascular Events in Phase 3 ZEUS Trial
The IL-6 inhibitor lowered inflammation markers but did not reduce heart attacks, strokes or cardiovascular deaths versus placebo in more than 6,300 patients, the company said.
Atea's Hepatitis C Regimen Meets Noninferiority Goal Against Epclusa in Phase 3
Bemnifosbuvir/ruzasvir achieved sustained virologic response in 93.9% of patients versus 94.8% for sofosbuvir/velpatasvir in the 905-patient C-BEYOND trial, with an eight-week course for those without cirrhosis, the company said.
Arrowhead's Plozasiran Meets Phase 3 Goals in Severe Hypertriglyceridemia
The quarterly injection cut triglycerides by a median 79% and 81% at 12 months in SHASTA-3 and SHASTA-4 and reduced acute pancreatitis events, the company said.
Gilead and Merck's Once-Weekly HIV Pill Matches Daily Therapy in Two Phase 3 Trials
Week 48 data from ISLEND-1 and ISLEND-2 show islatravir/lenacapavir kept the virus suppressed in adults who switched from daily regimens, and the companies plan regulatory filings.
FDA Approves Merck's Lipfendra, the First Oral PCSK9 Inhibitor Approved in the US
The once-daily enlicitide tablet cut LDL cholesterol by a placebo-adjusted 56% and 59% in two Phase 3 trials; whether it reduces heart attacks and deaths is still being studied.
Biogen Details Phase 2 CELIA Data for Anti-Tau Drug Diranersen, Plans Phase 3
The lowest dose slowed decline by 26% on the CDR-SB scale at 18 months, but the trial missed its dose-response primary endpoint and Biogen shares fell about 8% on the day.
AstraZeneca and Ionis' Wainua Misses Primary Goal in Phase 3 ATTR-CM Heart Trial
Adding the gene-silencing drug to standard care did not significantly cut cardiovascular deaths and recurrent events in the 1,432-patient CARDIO-TTRansform trial, the companies said.
Compass Pathways Reports Six-Month Phase 3 Data for Psilocybin Drug in Resistant Depression
The company said 39% of patients on two 25 mg doses of COMP360 responded by week 6 and kept that response through week 26, as it completes a rolling FDA application.