Roche and Ionis hit main endpoint in Phase 3 kidney disease trial
The partnered monthly antisense injection sefaxersen significantly lowered protein in the urine of patients with IgA nephropathy at an interim check, the companies announced.
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Thursday, October 15, 2026Thu, Oct 15
Updated 7:01 PM PT
The partnered monthly antisense injection sefaxersen significantly lowered protein in the urine of patients with IgA nephropathy at an interim check, the companies announced.
Immunovant has halted development of its anti-FcRn antibody IMVT-1402 in cutaneous lupus erythematosus after the drug failed a 57-patient Phase 2 trial against placebo.
Celldex reported that its monoclonal antibody barzolvolimab met primary and secondary endpoints in two randomized Phase 3 trials enrolling 1,939 patients with chronic spontaneous urticaria.
The injectable fusion protein dazodalibep met its primary endpoint in the first of two pivotal Phase 3 trials in patients with Sjögren's disease.
Viking Therapeutics reported that patients who received its dual GLP-1 and GIP receptor agonist VK2735 maintained 85% of their weight loss after switching to once-monthly dosing.
Vertex Pharmaceuticals reported that inaxaplin, an oral small molecule designed to inhibit APOL1, improved measures on a urine test across two different patient populations with kidney disease.
Otsuka and Ionis Pharmaceuticals reported that their Phase 3 Fusion trial of ulefnersen met its primary endpoint in 73 patients with amyotrophic lateral sclerosis caused by FUS gene mutations.
Weekly injections of enicepatide, an injectable dual GLP-1/GIP receptor agonist acquired through Roche's $2.7 billion purchase of Carmot Therapeutics, reduced HbA1c by 2.65% and body weight by 15.5% at week 48 in adults with Type 2 diabetes.
Beacon reported that its RPGR gene therapy, laru-zova, met its primary endpoint in an 85-patient pivotal study for X-linked retinitis pigmentosa, an inherited condition that causes progressive blindness.
The FDA has approved IntraBio's Aqneursa, an orally administered modified amino acid known as levacetylleucine, to treat neurological symptoms in patients with ataxia-telangiectasia.
Cue Biopharma reported that its injected allergy drug CUE-221 met its primary endpoint in a 145-patient Phase 2 trial for chronic spontaneous urticaria, a severe hives condition.
Alkermes reported that its oral orexin agonist ALKS 7290 reduced symptom scores in a 50-patient Phase 1b trial in adults with attention deficit hyperactivity disorder.
Hansoh Pharma's risvutatug rezetecan, partnered with GSK, and MediLink Therapeutics' tambotatug pelitecan, partnered with Roche, both cut the risk of death by 54% compared to standard topotecan chemotherapy in late-stage relapsed small-cell lung cancer trials.
Xenon Pharmaceuticals voluntarily stopped new patient enrollment in its Phase 3 trials of azetukalner, an oral Kv7 potassium channel opener, following reports of confusion, ataxia, and rare cases of psychosis.
Bristol Myers Squibb has terminated development of ORM-6151, an antibody designed to deliver a protein degrader to CD33-expressing leukemia cells, which it bought from Orum Therapeutics for $100 million in 2023.
The confirmatory Phase 3 trial evaluated Lunsumio (mosunetuzumab), a bispecific antibody that redirects T cells to kill cancerous B cells by targeting CD20 and CD3, in earlier-line patients with follicular lymphoma.
Longeveron is reviewing strategic options after its investigational cell therapy laromestrocel failed to beat control surgery in a Phase 2b trial of 40 infants with hypoplastic left heart syndrome.
Phase 3 clinical trial results demonstrate that Vera Therapeutics' dual BAFF and APRIL inhibitor atacicept, branded as Trutakna, successfully preserved kidney function in patients with IgA nephropathy.
Skyhawk Therapeutics reported that patients receiving SKY-0515 showed statistically significant clinical improvements over a control group from nine months through 15 months of treatment in an ongoing mid-stage trial.
Forbion led the Series C financing alongside TPG Life Sciences Innovations and Sectoral Asset Management to fund a 120-patient trial of linsitinib.
Novartis halted development of VHB937 in amyotrophic lateral sclerosis after the TREM2 stabilizer and activator missed both primary and secondary endpoints in a 251-patient Phase 2 trial against placebo.
BioMarin has halted development of its once-daily injected bone-growth peptide, vosoritide, for children with Noonan syndrome, attributing the decision to study feasibility and a changing commercial landscape rather than safety or efficacy.
Johnson & Johnson's oral M1 muscarinic receptor antagonist, JNJ-5120, failed its primary efficacy endpoint in a Phase 2 trial of 107 patients with major depressive disorder, showing no improvement over placebo on a standard depression rating scale at day five.
Novo has ended its metabolic disease collaboration with Ascendis Pharma less than two years after committing up to $285 million in milestones to develop a once-monthly weight-loss therapy.
Connect Biopharma reported that its IL-4R alpha antibody rademikibart missed the primary endpoint in a 160-patient Phase 2 trial in individuals with asthma and type 2 inflammation.
Vera Therapeutics reported that its dual BAFF and APRIL inhibitor Trutakna met all prespecified endpoints in the pivotal Phase 3 ORIGIN 3 trial for IgA nephropathy, an autoimmune kidney disorder.
In a Phase 3 trial in China, MediLink and Roche's antibody-drug conjugate tambotatug pelitecan extended median overall survival in relapsed small cell lung cancer to 13.3 months compared with 9.4 months on topotecan chemotherapy.
In an early-stage clinical trial, Corbus Pharmaceuticals' experimental pill CRB-913 achieved up to 5% placebo-adjusted weight loss after 12 weeks without the prominent neuropsychiatric safety issues that derailed earlier drugs in its class.
Camizestrant, an oral selective estrogen receptor degrader, failed to outperform standard care at delaying disease progression when paired with another therapy in a pivotal Phase 3 trial for advanced breast cancer.
Cellectis is seeking partners to offload its clinical-stage donor-derived cell therapies for leukemia and lymphoma, lasme-cel and eti-cel, effectively ending its pioneering work in allogeneic CAR-T treatments.