Clinical Trials
Ultragenyx to weigh ‘significant’ cost cuts as Angelman drug fails key study
Ultragenyx's stock nearly halved after its Phase III study of the Angelman syndrome candidate drug GTX-102 (apazunersen) failed to meet primary and secondary endpoints. The company plans to evaluate the drug program and seek significant cost reductions, focusing on marketed products, aiming for profitability by 2027.
Akeso, Summit cancer drug confirms lung cancer survival benefit
Akeso Biopharma and Summit Therapeutics announced on Thursday that their co-developed bispecific antibody, ivonescimab, met the key secondary endpoint of overall survival in an interim analysis of a Phase 3 clinical trial (HARMONi-2) in Chinese patients with non-small cell lung cancer. Results showed that patients treated with ivonescimab had significantly prolonged overall survival compared to those treated with Merck's pembrolizumab (Keytruda). Full data are expected to be presented at a lung cancer conference later this month. Although the trial results do not directly serve as the primary basis for a U.S. marketing application, they bolster industry confidence in positive outcomes from another global head-to-head trial.
Novartis says MS pill succeeds in two large studies
Novartis announced on Tuesday that its oral BTK inhibitor remibrutinib met primary endpoints in two Phase III clinical trials for relapsing multiple sclerosis, significantly reducing annualized relapse rates and brain inflammatory lesions, with no liver safety concerns observed. Following this news, Novartis shares rose more than 4%.
Alumis’ TYK2 drug fails in key lupus study
Alumis's TYK2 inhibitor envudeucitinib missed primary and secondary endpoints in a Phase IIb study of systemic lupus erythematosus, but the high interferon gene signature subgroup showed a "robust" response. The company still plans to move forward and will discuss the Phase III trial path with regulators. Shares plunged nearly 55% after the announcement.
ESC ‘26: Cytokinetics’ sell-off, Arrowhead’s edge and a heart drug autopsy
At the 2026 European Society of Cardiology (ESC) Annual Meeting, several major pharmaceutical and biotechnology companies presented detailed study data on drugs for various heart diseases. This article reviews three closely watched clinical data sets and their impact on investors and analysts: detailed data from Cytokinetics' Myqorzo Phase III study in non-obstructive hypertrophic cardiomyopathy showed robust efficacy, but the disclosure of specific heart failure events led to a stock price decline of over 7%; Arrowhead's plozasiran demonstrated comparable efficacy and potentially better safety versus Ionis' Tryngolza in two Phase III studies for severe hypertriglyceridemia, with a marketing application expected by year-end; and the detailed analysis following the Phase III failure of eplontersen, developed by AstraZeneca and Ionis, for transthyretin amyloid cardiomyopathy, sparked renewed discussion on the positioning of existing therapies and the prospects of Alnylam's investigational drug.
Spyre tumbles as immune drug misses mark in rheumatoid arthritis
Spyre Therapeutics announced results from a Phase 2 trial of SPY072 in rheumatoid arthritis, where the drug failed to meet the company's predefined development threshold, prompting a shift to other autoimmune indications. Shares dropped 13% in response, with a market value loss exceeding $1 billion.
Cancer vaccine win restores Wall Street’s faith in Moderna
Moderna and Merck's cancer vaccine intismeran met its primary endpoint in a Phase 3 melanoma trial, a first for cancer vaccines. The news boosted Moderna's shares by more than 170%, adding nearly $45 billion in market value, but some analysts caution the market reaction may be overblown given limited disclosed data.
Moderna and Merck cancer vaccine returns ‘landmark’ result in melanoma
Moderna and Merck reported that their personalized mRNA cancer vaccine, intismeran, combined with Keytruda, significantly improved disease-free survival in patients with resected melanoma in a Phase 3 trial. The interim analysis met pre-specified criteria, prompting analysts to project billions in sales. Full results are pending presentation.
Amylyx's GLP-1 Blocker Bet Yields Positive Late-Stage Data
Amylyx Pharmaceuticals announced positive late-stage trial results for avexitide, a potential first-in-class treatment for post-bariatric hypoglycemia. The drug reduced serious-to-severe hypoglycemic events by 55% versus placebo and appeared safe. Amylyx intends to file for FDA approval before year-end, marking a comeback after withdrawing its ALS drug in 2024.
Argenx Shares Surge on Positive Phase 3 Data for Vyvgart in Rare Muscle Disease
Argenx announced positive Phase 3 results for Vyvgart Hytrulo in myositis, leading to a 13% stock surge. The trial met its primary endpoint overall, with strong efficacy in IMNM but not in DM. Analysts see expansion opportunity despite mixed subset data.