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Depression pipeline hits Phase 2 setback
Johnson & Johnson’s JNJ-5120, a depression program developed with Contineum Therapeutics, missed its primary endpoint in a Phase 2 trial. The failure further complicates the outlook for the...
Regulatory approvals: first-in-class SMA therapy cleared by FDA
The FDA approved Scholar Rock’s Isembyld (apitegromab-mstn), a myostatin inhibitor for spinal muscular atrophy, marking a first-in-class, muscle-targeted treatment approach. The clearance is for...
FDA policy: reassessing rare-disease trial criteria
The FDA is seeking outside input on whether to reassess criteria for rare disease trials, including endpoint selection and the level of evidence needed to support efficacy and safety. The agency’s...
China Phase 3: B7-H3 ADCs post overall survival wins in relapsed SCLC
China-based Phase 3 trials reported overall survival wins for B7-H3 antibody-drug conjugates in relapsed small-cell lung cancer. MediLink’s tambotatug pelitecan (tam-peli; YL-201) and risvutatug...
Oncology: Roche partner MediLink posts Phase 3 OS benefit for Tam-Peli in China
Roche collaborator MediLink released interim Phase 3 results for tambotatug pelitecan (Tam-Peli; YL201) in Chinese patients with relapsed small-cell lung cancer. In the TAISHAN-302 trial, Tam-Peli...
Biotech partnerships and platform: discovery transcriptomics CRO acquisition
Single Cell Discoveries completed the acquisition of TATAA Biocenter to create a European CRO able to move molecular target work from discovery transcriptomics through GLP/GCLP-compliant...
Regulatory submissions: AI platform for clinical trial data-to-filing acceleration
nPhase launched an AI platform aimed at accelerating regulatory submissions for clinical trials by converting raw study data into submission-ready information. The company claims the workflow can...
Clinical trial results in neuromuscular disease: DM1 Phase 3 misses primary endpoint
Novartis said its global Phase 3 HARBOR study of del-desiran in myotonic dystrophy type 1 did not meet the primary endpoint of video hand opening time (vHOT) versus placebo. The company reported...
Platform therapeutics pivot: Cellectis exits CAR-T for in vivo gene editing
Cellectis said it is exiting its CAR-T programs and pivoting toward “in vivo” gene editing, citing a shifting landscape and changes in how the company wants to deploy resources. The French biotech...
Regulatory approvals in rare neuromuscular disease
The FDA cleared Scholar Rock’s myostatin inhibitor apitegromab (Isembyld) for spinal muscular atrophy (SMA) in adults and children 2 years of age and older, positioning a new, muscle-targeted...
Phase 3 oncology readouts reshape second-line SCLC strategy
MediLink reported interim Phase 3 results for tambotatug pelitecan (Tam-Peli; YL-201), a B7-H3-targeted antibody-drug conjugate with a dual release payload, beating topotecan on overall survival...
Phase 3 competitive pressure in first-line breast cancer SERD strategy
AstraZeneca’s Etcamah (camizestrant) failed to significantly delay disease progression in SERENA-4, a Phase 3 trial evaluating the oral SERD as part of a standard first-line regimen for advanced...
Emerging oncology biomarkers and dual-acting PD-1/VEGF competition
Akeso and Summit Therapeutics moved to reinforce the clinical case for ivonescimab with new HARMONi-2 results presented at the World Conference on Lung Cancer. The dual-acting PD-1/VEGF...
FDA action for rare disease clinical trial design standards
The FDA signaled it is rethinking how it evaluates rare-disease trial criteria, including endpoint selection and the amount of efficacy and safety evidence required for approval. As more therapies...
Regulatory AI tooling for clinical submissions
nPhase launched an AI platform aimed at accelerating regulatory submission preparation for clinical trials, claiming it can convert raw study data into submission-ready materials 75% to 80% faster...
Imaging innovation reaches the US market for glioma
Telix Pharmaceuticals won U.S. FDA approval for Pixclara (floretyrosine F-18, 18F-FET), marking the first FDA-approved FET-PET imaging agent for glioma. The approval also makes Pixclara the first...
Drug development outcomes in neuromuscular antibody-oligonucleotide conjugates
Novartis updated its Phase 3 HARBOR program for del-desiran (del-desiran; an antibody oligonucleotide conjugate) in myotonic dystrophy type 1 (DM1), reporting it did not meet the trial’s primary...
Biotech CRO consolidation to move targets from discovery to validation
Single Cell Discoveries completed the acquisition of TATAA Biocenter, combining discovery transcriptomics capabilities with qPCR/dPCR and GLP/GCLP-compliant bioanalytical and assay development...
Company pivots and platform bets in gene editing and muscle biology
Cellectis announced it is exiting CAR-T programs and shifting toward “in vivo” gene editing, citing changes in the cell-therapy landscape and the need to reallocate resources. The move underscores...
FDA approval—first myostatin-targeted therapy expands SMA options
The FDA cleared Scholar Rock’s Isembyld (apitegromab, myostatin inhibitor) for spinal muscular atrophy in adults and in children at least 2 years old who are receiving a survival motor neuron...