Swedish biotech Atrogi says its oral drug ATR-258 increased lower-extremity muscle power by about 13% after eight weeks in a study of 13 people with overweight or obesity. Participants also lost an average of 1.1 kg of fat mass and gained 0.8 kg of lean mass.
uniQure’s shares fell about 40% in early trading on September 29 after its Huntington’s gene therapy missed statistical significance on the main disease progression measure after four years. The Dutch biotech reported continued slowing of decline with AMT-130, but the update complicates the durability picture as the company seeks regulatory approval.
Roche’s sefaxersen significantly reduced proteinuria in a Phase 3 IgA nephropathy (IgAN) trial, but the Swiss pharma stopped short of disclosing the size of the effect.
Agomab Therapeutics’ inhaled idiopathic pulmonary fibrosis (IPF) drug AGMB-447 showed lung-restricted exposure and inhibited its intended target in its first patient trial. The company plans to move the ALK5 inhibitor into phase 2 before the end of the year.
French biotech Axoltis Pharma’s NX210c missed the primary endpoint of a Phase 2 trial in amyotrophic lateral sclerosis (ALS), but post-hoc analyses showing slower functional decline are giving the company enough encouragement to plan further development.
Two B7-H3-targeted antibody-drug conjugates licensed from Chinese biotechs have now delivered positive phase 3 survival data in relapsed small-cell lung cancer, strengthening the oncology bets GSK and Roche made in China.
Danish biotech Teitur Trophics completed its phase 1 study and said its experimental Parkinson’s disease treatment TT-P34 was safe and reached the central nervous system, supporting plans to move the peptide into phase 2 testing in 2027.
Switzerland-based Pharvaris’ once-daily oral deucrictibant XR reduced hereditary angioedema (HAE) attacks by 83% versus placebo in a pivotal Phase 3 trial, putting the experimental prophylactic into roughly the same efficacy range as several leading injected treatments.
Novartis has suffered another pipeline setback after del-desiran, an antibody-oligonucleotide conjugate acquired with Avidity Biosciences, failed to significantly improve hand function over placebo in the Phase 3 HARBOR study in myotonic dystrophy type 1.
Novartis’ pelacarsen failed to reduce cardiovascular events in the first Phase 3 outcomes trial of an Lp(a)-lowering drug, despite successfully lowering the genetically driven cardiovascular risk factor.