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Clinical readouts

Phase III and pivotal trial results — positive and negative, treated alike.

Latest picks

141–159 of 390
Oct 1Thu
  1. The Lancet62

    [Articles] 8 weeks versus 12 weeks of sofosbuvir–velpatasvir for treatment-naive, non-cirrhotic, chronic hepatitis C (RESOLVE): a multicentre, open-label, non-inferiority, randomised controlled trial in India

    Eight weeks of sofosbuvir-velpatasvir was similar in efficacy to the standard 12-week regimen in treatment-naive, non-cirrhotic patients with chronic hepatitis C in the RESOLVE multicentre, open-label, non-inferiority, randomised controlled trial in India. The finding supports shortening treatment duration in this population.

    Why it matters: A randomised non-inferiority trial supporting 8-week sofosbuvir-velpatasvir instead of 12 weeks for non-cirrhotic chronic HCV.

  2. The Lancet55

    [Articles] Cardiovascular safety of orforglipron versus insulin glargine in adults with type 2 diabetes at increased cardiovascular risk (ACHIEVE-4): a phase 3, event-driven, randomised, open-label, non-inferiority, active comparator trial

    Orforglipron was non-inferior to insulin glargine for MACE-4 in adults with type 2 diabetes at increased cardiovascular risk, establishing its cardiovascular safety in the event-driven ACHIEVE-4 trial. Gastrointestinal adverse events were the most frequent adverse event and the most common reason for orforglipron discontinuation, while clinically significant hypoglycaemia occurred less often with orforglipron than with insulin glargine. The findings support orforglipron as a once-daily oral option with established cardiovascular safety in this population.

    Why it matters: Cardiovascular safety data for an oral daily drug versus insulin glargine in high-risk type 2 diabetes.

  3. Medical Xpress69

    Trevogrumab (anti-myostatin) can help preserve lean mass during and after semaglutide-induced weight loss

    Regeneron reported Phase II data (N=975) at the EASD meeting and in The Lancet: trevogrumab combined with semaglutide reduced lean mass loss by about 50% by DXA and muscle volume loss by about 70% by MRI.

    Why it matters: Provides lean mass and muscle retention percentages by both DXA and MRI for comparison with existing interventions such as exercise.

Sep 30Wed
  1. STAT57

    STAT+: Eli Lilly’s Zepbound combined with amylin drug led to significant weight loss in mid-stage trial

    Combining Eli Lilly's Zepbound with eloralintide, an amylin-targeting candidate, produced 23.3% weight loss at 48 weeks in people with obesity and type 2 diabetes at the highest dose, outperforming Zepbound alone (14.8%) and eloralintide alone (11.1%). High discontinuation rates may raise tolerability questions. It shows next-generation combination therapy can achieve greater weight loss even in patients with diabetes, who typically lose less on medications.

    Why it matters: Mid-stage trial shows a Zepbound-eloralintide combination beats either drug alone on weight loss in diabetes.

  2. Medical Xpress62

    Experimental obesity drug targets appetite through amylin, offering potential alternative to GLP-1 therapies

    Weekly injections of petrelintide produced roughly 10% weight loss over 42 weeks in the Phase II ZUPREME 1 trial, with a permanent discontinuation rate due to gastrointestinal events of only 1.5%. Results were presented at the EASD annual meeting and published in The Lancet Diabetes & Endocrinology.

    Why it matters: The article gives 42-week weight-loss figures by dose and gastrointestinal discontinuation rates that readers can compare directly with real-world GLP-1 discontinuation data.

  3. Nature70

    Intravenous hyaluronidase-expressing oncolytic adenovirus with chemotherapy in metastatic pancreatic cancer: a randomized phase 2b trial

    Intravenous zabilugene almadenorepvec (VCN-01), a hyaluronidase-expressing oncolytic adenovirus, plus gemcitabine and nab-paclitaxel prolonged overall survival over chemotherapy alone in the randomized phase 2b VIRAGE trial in treatment-naive metastatic pancreatic cancer. The finding supports adding an oncolytic virus to standard first-line chemotherapy in this setting.

    Why it matters: First randomized evidence that an intravenous oncolytic adenovirus improves survival in metastatic pancreatic cancer.

  4. The Lancet56

    [Articles] Retatrutide in adults with obesity and type 2 diabetes (TRIUMPH-2): a double-blind, parallel-group, randomised, placebo-controlled, phase 3 trial

    Retatrutide produced substantial improvements in bodyweight and improved glycaemic control in adults with obesity and type 2 diabetes in the phase 3 TRIUMPH-2 trial, a double-blind, randomised, placebo-controlled comparison. Its safety profile was generally consistent with other molecules with GLP-1 receptor agonism, supporting retatrutide as a possible treatment for obesity in people with type 2 diabetes.

    Why it matters: Phase 3 evidence that retatrutide addresses both obesity and glycaemic control in type 2 diabetes.

  5. Reddit88

    Phase 3 results for Retatrutide

    NEJM published Eli Lilly's phase 3 TRIUMPH-1 trial of retatrutide. Among 2339 patients with obesity but without diabetes, after 80 weeks of randomization, the 4 mg/9 mg/12 mg dose groups lost an average of 17.6%/23.7%/25.0% of body weight versus 3.9% with placebo (differences of -19.8 and -21.0 percentage points, both P<0.001).

    Why it matters: Provides 80-week weight loss up to 25% for direct comparison with semaglutide's roughly 15% and tirzepatide's roughly 20%.

  6. Endpoints News37

    Roche cans muscle-preserving obesity drug, hands it back to Chugai

    Roche has discontinued development of a muscle-building drug and ended a trial testing it in combination with an anti-obesity injectable, returning the experimental medicine to Chugai. The move removes one candidate from the muscle-preserving approach to obesity treatment.

    Why it matters: A major pharma exiting a muscle-preserving obesity program shifts the competitive landscape for that strategy.

Sep 29Tue
  1. BioSpace54

    UniQure crashes as Huntington’s gene therapy shows less slowing of disease at 4 years

    Four-year data on UniQure's Huntington's gene therapy AMT-130 showed a 44% slowing of disease progression on the cUHDRS in 12 high-dose patients, non-significant versus natural history controls and down from the 75% slowing reported at three years; the stock fell as much as 38% to $24.11. Total functional capacity slowing was 61% and significant, and a three-year analysis of all 15 high-dose patients showed 80% slowing on cUHDRS and 67% on TFC, the timepoint anchoring the submitted BLA. Five high-dose patients, 17%, had treatment-related serious CNS inflammation events, all resolved.

    Why it matters: Four-year efficacy regression on the primary measure complicates the pending FDA review of the first Huntington's gene therapy BLA.

  2. BioSpace40

    Roche cuts muscle-sparing obesity drug after mid-stage disappointment

    Roche has stopped developing emugrobart, an anti-myostatin antibody intended to spare lean mass during weight loss, and rights revert to Chugai, which plans to resume development for spinal muscular atrophy and advance it into Phase 3. Roche had been testing the drug in the Phase 2 GYMINDA trial in combination with tirzepatide. The exit removes Roche's myostatin-targeting approach from the obesity field, leaving enicepatide and petrelintide as its main obesity assets.

    Why it matters: A major pharma abandoning a muscle-preserving obesity mechanism reshapes the competitive landscape and returns the asset to Chugai for Phase 3 in SMA.

  3. Nature54

    Werner helicase inhibitor for advanced microsatellite instability solid tumors: a phase 1 trial

    RO7589831, a first-in-class inhibitor of the synthetic lethal target Werner syndrome helicase, showed some partial responses in the dose-escalation stage of a phase 1 trial in patients with microsatellite instability and/or DNA mismatch repair-deficient solid tumors. The maximum tolerated dose was not reached.

    Why it matters: First clinical data for a Werner helicase inhibitor, a new synthetic lethal strategy in MSI tumors.

  4. Nature54

    Oral small-molecule GLP-1 receptor agonist safiglipron in early type 2 diabetes: a randomized, double-blind, placebo-controlled trial

    Once-daily oral small-molecule GLP-1 receptor agonist safiglipron reduced HbA1c by up to 1.45% versus placebo over 32 weeks in people with early type 2 diabetes not taking glucose-lowering medication, in the randomized OUTSTAND-1 trial. Body weight effects were modest. It offers an oral option with glycemic efficacy in this population.

    Why it matters: Oral small-molecule GLP-1 receptor agonists would replace injectable dosing for type 2 diabetes.

  5. The Lancet64

    [Articles] Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial

    Garetosmab, an activin A-blocking antibody, substantially reduced the number of new heterotopic bone lesions in patients with fibrodysplasia ossificans progressiva in the randomised, double-blind, placebo-controlled phase 3 OPTIMA trial. The results support its potential as an efficacious and generally well tolerated disease-modifying therapy for FOP.

    Why it matters: Phase 3 evidence that blocking activin A can modify disease course in FOP.

  6. BioCentury84

    Kodiak challenges Eylea with a six-month dosing interval in wet AMD

    Zenkuda tarcocimab tedromer met the primary endpoint in the Phase III DAYBREAK study in wet AMD, showing non-inferiority to Eylea aflibercept on best corrected visual acuity at one year. More than half of patients reached a six-month dosing interval, a point of differentiation in retinal disease, and Kodiak Sciences shares rose 178% on the news.

    Why it matters: Phase III non-inferiority to Eylea plus six-month dosing data position a new wet AMD therapy against a market leader.

  7. BioPharma Dive57

    Kodiak skyrockets on positive study data for 2 eye drugs

    Kodiak Sciences' Zenkuda matched Regeneron's Eylea in a Phase 3 trial in wet age-related macular degeneration, improving visual acuity by 7.2 letters versus 7.6 for Eylea, with 54% of participants dosed only once every six months under an individualized strategy. Tabirafusp alfa tedromer was also non-inferior, gaining 6.3 letters. Kodiak shares more than doubled and its market value rose by more than $3 billion; an FDA filing for Zenkuda is planned in the fourth quarter of 2026.

    Why it matters: Phase 3 non-inferiority to Eylea with six-month dosing positions a cheaper durable threat to a leading eye drug.

Sep 28Mon
  1. BioPharma Dive47

    An ‘off-the-shelf’ CAR-T therapy from Adicet shows promise against lupus

    Adicet's donor-derived gamma delta CAR-T therapy prula-cel drove lupus into remission in an early-stage trial. After a year of follow-up, 50% of evaluable lupus nephritis patients had a complete kidney response and 54% met criteria for remission, with all stopping immunosuppressive drugs and no serious cytokine release syndrome or neurological side effects. A pivotal lupus study is planned for the fourth quarter.

    Why it matters: It suggests an off-the-shelf cell therapy could match personalized CAR-Ts in lupus without their manufacturing and safety drawbacks.

CBACBA Frontier Insights (CFI)Chinese Biopharmaceutical Association (CBA)www.cba-usa.org