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Clinical readouts

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

391 picksRelatedSafety signalsRegulatoryOncology

Latest picks

41–60 of 391
Oct 15Thu
  1. BioSpace58

    Verastem reports ‘robust’ response rate in pancreatic cancer subgroup where Rasonque struggled

    VS-7375, an oral selective KRAS G12D dual ON/OFF inhibitor, produced a 50% confirmed ORR in second-line metastatic pancreatic ductal adenocarcinoma at 900 mg daily, versus 24% for Rasonque in G12D patients in the Phase 3 RASolute 302 trial. In third- and fourth-line mPDAC the confirmed ORR was 36%, with 43% confirmed ORR in NSCLC at 600 mg and 33% unconfirmed ORR in third-line colorectal cancer combined with anti-EGFR therapy. Discontinuation due to adverse events was 2% across the 295-patient TARGET-D 101 trial, and Verastem will start a Phase 3 first-line mPDAC trial by year-end.

    Why it matters: Allele-specific KRAS G12D inhibition may outperform the newly approved pan-RAS agent in the subgroup where Rasonque underperforms.

Oct 14Wed
  1. Nature62

    Talazoparib plus enzalutamide in high-volume metastatic hormone-naive prostate cancer: a randomized phase 2 trial

    Adding the PARP inhibitor talazoparib to enzalutamide improved prostate-specific antigen response in patients with high-volume metastatic hormone-sensitive prostate cancer in the randomized phase 2 ZZ-FIRST trial. Spatial transcriptomic analysis of paired biopsies showed that androgen receptor inhibition suppresses homologous recombination repair-related programs, and this suppression correlated with proliferation arrest rather than functional HRR deficiency.

    Why it matters: Links AR inhibition to HRR program suppression, giving a mechanistic basis for combining PARP inhibition with androgen receptor inhibitors.

  2. Medical Xpress88

    Triple-action drug retatrutide delivers major weight loss for people with diabetes, Phase III trial finds

    In the Phase III TRIUMPH-2 trial, 1,152 patients with obesity and type 2 diabetes received Eli Lilly's triple agonist retatrutide at weekly doses of 4, 9, or 12 mg for 80 weeks. Mean weight loss was 11.9%, 16.8%, and 18.8% respectively, versus 5.1% for placebo. In the 12 mg group, 75% of participants lost at least 10% and nearly one-third lost at least 25%.

    Why it matters: The article reports weight loss and HbA1c improvements across three doses, enabling direct comparison with semaglutide and tirzepatide in the same indication.

  3. Medical Xpress65

    Targeted radioactive therapy may help delay spread of recurrent prostate cancer

    Adding two cycles of the PSMA-targeted radioligand 177Lu-PNT2002 before SBRT cut the risk of progression or needing additional hormone therapy by 62% versus SBRT alone in 92 men with oligorecurrent hormone-sensitive prostate cancer, followed for a median of 31.9 months. At 24 months, 33% of the combination group had no detectable disease versus 12% with SBRT alone, and fewer developed widespread disease or castration-resistant prostate cancer. The phase 2 LUNAR results suggest treating microscopic disease while delaying hormone therapy.

    Why it matters: Longer LUNAR follow-up shows a radioligand plus SBRT combination may reach undetectable disease and delay hormone therapy.

  4. Medical Xpress58

    Drug tapering noninferior to treatment continuation for rheumatoid arthritis with low disease activity

    Structured tapering of biologic or targeted synthetic DMARDs was noninferior to continuation for rheumatoid arthritis patients with sustained low disease activity in a 24-week randomized trial of 171 patients assigned to tapering and 177 to continuation. Low disease activity was maintained in 88.2% of the tapering group versus 89.6% of the continuation group, a risk difference of −1.43 percentage points (95% CI, −8.9 to 5.23), meeting the noninferiority criterion. Most patients who worsened regained low disease activity after protocol-guided treatment re-escalation.

    Why it matters: It supports dose reduction for RA patients with sustained low disease activity without losing disease control at 24 weeks.

  5. Medical Xpress59

    Scientists identify clues to making immunotherapy more effective against glioblastoma

    Neoadjuvant nivolumab plus ipilimumab extended median survival to about 13.5 months versus roughly nine months with nivolumab alone in a phase 2 trial of 63 patients with recurrent glioblastoma. Five patients, about 15% of the combination group, became long-term survivors, with none on nivolumab alone. Tumor T-cell density independently predicted overall survival, and early pseudoprogression on MRI correlated with longer survival, pointing to biomarkers for patient selection.

    Why it matters: Dual checkpoint blockade produced long-term survivors in recurrent glioblastoma, a cancer largely resistant to immunotherapy.

  6. WeChat72

    BioNTech BNT113 Plus Pembrolizumab Misses PFS Endpoint in AHEAD-MERIT HPV16-Positive HNSCC Trial

    BNT113 plus pembrolizumab failed to significantly improve progression-free survival versus pembrolizumab alone in first-line HPV16-positive head and neck squamous cell carcinoma, according to first topline results from the phase 2/3 AHEAD-MERIT trial (NCT04534205). PFS was a dual primary endpoint. BNT113 is an mRNA tumor immunotherapy encoding the HPV16 E6 and E7 proteins.

    Why it matters: First phase 3 failure for a BioNTech mRNA cancer vaccine candidate in a key indication.

  7. JAMA Network67

    Cold and Room-Temperature Platelets in Cardiac Surgery

    A noninferiority randomized clinical trial compared cold-stored versus room-temperature platelets in patients transfused for active bleeding from cardiac surgery. It determined the maximum cold-storage duration and assessed hemostatic efficacy of cold-stored platelets against the room-temperature standard.

    Why it matters: Directly compares cold-stored and room-temperature platelets for hemostatic efficacy in cardiac surgery bleeding.

  8. BioPharma Dive53

    First Tracks climbs on ‘impressive’ celiac study data

    First Tracks Biotherapeutics reported that its celiac disease candidate ANB033 met the primary endpoint in an early-stage trial of 32 participants, showing a statistically significant improvement over placebo on a measure of intestinal mucosal barrier protection. Two other disease severity assessments and symptom control during a gluten challenge also hit prespecified targets. The trial saw no treatment-related serious adverse events and no participants withdrew due to side effects.

    Why it matters: Analysts compared ANB033 with Teva's celiac results reported last month and judged the former stronger, letting readers weigh the two early CD122-pathway signals.

Oct 13Tue
  1. 顶刊论文（社媒热度）65

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

    Adding intravenous VCN-01, a hyaluronidase-expressing oncolytic adenovirus, to gemcitabine and nab-paclitaxel improved outcomes as first-line therapy in metastatic pancreatic ductal adenocarcinoma. In the full analysis set, median overall survival was 10.8 versus 8.6 months (HR 0.57) and progression-free survival was 7.0 versus 4.6 months (HR 0.55, P = 0.011); response duration was 11.2 versus 5.4 months. The phase 3 evaluation is warranted.

    Why it matters: First randomized evidence that an intravenous oncolytic virus plus chemotherapy extends survival in metastatic pancreatic cancer.

  2. BioSpace50

    Syndax ends fibrosis program for Niktimvo after mid-stage disappointment

    Niktimvo (axatilimab) failed its primary endpoint in the Phase 2 MAXPIRe study, missing on slowing the annualized rate of decline in forced vital capacity at 26 weeks among 145 patients with idiopathic pulmonary fibrosis, and Syndax has discontinued the drug's development in IPF. The monoclonal antibody remains approved for chronic graft-versus-host disease after at least two prior lines of treatment, and two ongoing combination studies in that indication read out later this year and in early 2028.

    Why it matters: A Phase 2 failure ends a CSF-1R inhibitor's development in IPF while the approved GVHD program continues.

  3. WeChat73

    Bristol Myers Squibb's RYZ101 Hits PFS Primary Endpoint in Phase 3 ACTION-1 for GEP-NETs

    Bristol Myers Squibb's radiopharmaceutical RYZ101 (225Ac-DOTATATE) met the primary endpoint of progression-free survival in the phase 3 ACTION-1 trial. Blinded independent central review showed a statistically significant and clinically meaningful improvement over investigator's choice of standard therapy, with safety consistent with the previously known profile.

    Why it matters: The phase 3 ACTION-1 trial met its PFS endpoint in SSTR+ GEP-NETs patients who progressed after prior Lu-177 therapy, enabling comparison with existing treatments in this line.

  4. WeChat84

    Epcoritamab plus R-CHOP cuts progression or death risk by half in untreated high-risk DLBCL

    Adding epcoritamab to R-CHOP cut the risk of disease progression or death by 51% in previously untreated DLBCL patients with IPI scores of 3 to 5 (HR 0.49; 95% CI, 0.35-0.69; P<0.0001), meeting the primary endpoint of the phase 3 EPCORE DLBCL-2 trial. The benefit was matched in the overall IPI 2 to 5 population (HR 0.49; 95% CI, 0.36-0.67; P<0.0001). This is the first positive phase 3 study of a bispecific antibody combination in first-line DLBCL.

    Why it matters: First phase 3 evidence that a bispecific antibody plus R-CHOP improves PFS in front-line DLBCL.

  5. WeChat73

    Hansoh's HS-10380 Beats Placebo and Aripiprazole on PANSS in Phase 2b Schizophrenia Trial

    Hansoh Pharmaceutical's oral drug HS-10380 met the primary endpoint in a Phase 2b trial in acute schizophrenia, with every dose group beating placebo on PANSS total score at day 42. Placebo-corrected least-squares mean differences were -16.7, -13.2 and -14.7 points, all p<0.0001, against -12.1 points for the active comparator aripiprazole. The results, presented at the ECNP congress in Munich, support advancing HS-10380 to a further late-stage trial.

    Why it matters: Every HS-10380 dose beat aripiprazole on PANSS reduction in acute schizophrenia, supporting further late-stage development.

  6. medRxiv41

    FORESEE: A prospective feasibility trial of functional precision oncology for metastatic breast cancer patients

    Organoid-based drug testing plus genomic characterization was feasible but limited by PDO establishment in HER2-negative metastatic breast cancer. Of 15 patients enrolled, 10 biopsies had adequate tumor content and PDOs were established in 4, an overall success rate of 40%, missing the primary endpoint of at least 46%. Results returned within 12 weeks informed treatment selection in all evaluable cases, and physicians used the information in 100% of successful cases.

    Why it matters: Quantifies the real-world success rate of functional precision oncology in metastatic breast cancer, showing PDO establishment as the bottleneck.

  7. 顶刊论文（社媒热度）58

    Optogenetic Therapy for Restoring Aspects of Visual Function.

    In the open-label PIONEER trial, 10 patients with advanced retinitis pigmentosa received a single intravitreal injection of an AAV vector expressing ChrimsonR, used together with light-stimulating goggles. Safety was acceptable, and 6 participants achieved a clinically meaningful improvement in light sensitivity.

    Why it matters: Small open-label trial in 10 patients with advanced retinitis pigmentosa gives preliminary clinical reference for optogenetic therapy.

  8. BioPharma Dive53

    First Tracks climbs on ‘impressive’ celiac study data

    ANB033, a CD122-blocking antibody from First Tracks Biotherapeutics, produced a statistically significant improvement over placebo in protecting the intestinal lining from gluten-induced damage in an early-stage celiac disease trial. The 32 participants received three subcutaneous doses or placebo over four weeks, then a two-week gluten challenge equivalent to about two slices of bread daily; the drug hit significance on three biological measures with no related serious adverse events. A second cohort reports in early 2027, with a late-stage trial planned that year.

    Why it matters: First positive clinical data for a CD122 blocker in celiac, an autoimmune disease with no approved treatment.

  9. The Journal of dermatological treatment37

    Comparative efficacy of topical garlic extract and 20% podophyllin solution in the treatment of female genital warts: a triple-blind randomized clinical trial

    Topical 5% garlic extract worked as well as 20% podophyllin solution for female genital warts in a triple-blind randomized trial of women treated weekly for four weeks. Mean wart size fell from 1.61 to 0.60 mm with garlic and from 2.96 to 0.62 mm with podophyllin, with no significant between-group difference (p = 0.756). Complete response occurred in 51.4% of each group, offering a low-cost alternative where no single therapy gives consistent clearance.

    Why it matters: It shows a low-cost topical garlic extract matches a standard genital wart treatment in a randomized trial.

Oct 12Mon
  1. BioSpace68

    BMS’ Phase 3 win for lung drug clouded by high-dose miss

    Bristol Myers Squibb's LPA1 antagonist admilparant met the primary endpoint in the Phase 3 ALOFT-IPF study, with the 60-mg dose showing a statistically significant improvement over placebo in absolute change in FVC from baseline at week 52, while the 120-mg dose missed significance on the same measure. The drug was well tolerated across both doses, with discontinuations due to adverse events comparable to placebo. Analysts said the results could support approval and validate the LPA1 mechanism, but the lack of dose response raises questions; BMS plans to meet health authorities ahead of regulatory submissions.

    Why it matters: Phase 3 success for a first-in-class LPA1 antagonist in IPF, but the high-dose miss and prior liver-injury death complicate the regulatory picture.

  2. BioSpace69

    BMS’ $4.1B RayzeBio bet delivers Phase 3 radiopharma win

    Bristol Myers Squibb's RYZ101, an actinium-225-based radiopharmaceutical acquired in the $4.1 billion RayzeBio deal, met the primary endpoint in an interim analysis of the Phase 3 ACTION-1 study, showing a statistically significant and clinically meaningful improvement in progression-free survival by blinded independent central review. The trial enrolled patients with advanced somatostatin receptor-expressing GEP-NETs that progressed after Lutathera, and these are the first Phase 3 results for an alpha-isotope targeted radiopharmaceutical therapy in any indication.

    Why it matters: First Phase 3 readout for an actinium-225 alpha-isotope radiopharmaceutical, a potential new option after Lutathera progression in GEP-NETs.

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