Positive data from Novo Nordisk A/S’s pivotal phase IIIa study of once weekly and once monthly doses of its hemophilia treatment are prompting the company to say it will submit the first regulatory approval request toward the end of this year. Mim8, a next-generation human IgG4 bispecific antibody bridging factor IXa/X, hit its co-primary endpoints by showing a statistically significant and superior reduction of treated bleeding episodes in those ages 12 and older. Roche Holding AG’s Hemlibra (emicizumab), a bispecific factor IXa- and factor X-directed antibody for hemophilia A, was approved in 2017 by the U.S. FDA. Biomarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec) was approved by the FDA in June 2023 as the first gene therapy for the indication.

Immatics mulls registrational trial with melanoma prospect

As part of its quarterly update, Immatics NV had good news in the melanoma space with IMA-203. The candidate emerged from the ACTengine platform, set up to formulate a personalized therapy in which a patient’s own T cells are collected, genetically modified to make TCR-T cells, and then reinfused. Immatics, with offices in Tuebingen, Germany, and Houston, offered data with IMA-203 as a monotherapy that targets preferentially expressed antigen in melanoma, or PRAME, from an ongoing phase I trial testing what’s been established as the recommended phase II dose of 1-10x10⁹ TCR-T cells in 30 heavily pretreated metastatic melanoma patients who were evaluable for efficacy. Results were pleasing enough that Immatics plans a potentially registration-enabling randomized phase II/III trial with the compound in second-line-plus disease this year, after talks with the U.S. FDA. Shares (NASDAQ:IMTX) were trading at $11.96, up 96 cents.

Shionogi’s COVID-19 drug misses endpoint in global phase III

Osaka, Japan-based Shionogi & Co. Ltd. said on May 13 that ensitrelvir fumaric acid (Xocova), its oral antiviral for COVID-19, showed no statistical difference against placebo in completely resolving 15 common COVID-19-related symptoms in a global phase III Scorpio-HR trial. Shifting the endpoint to predefined supportive analysis, defined as resolution of six COVID-19-related symptoms, however, yielded a statistically significant difference with ensitrelvir compared to placebo for the time to resolution of symptoms.

BIOSECURE Act officially begins trek through US Congress

The BIOSECURE Act is now more than just a topic of conversation in the halls and meeting rooms of the U.S. Capitol. Reps. Brad Wenstrup (R-Ohio) and Raja Krishnamoorthi (D-Ill.) formally introduced the legislation in the House Friday as a defense against China’s national security laws requiring all Chinese firms to share any requested data with the Chinese Communist Party, including biotechnology companies that collect, test and store American genomic data. Besides prohibiting federal agencies from contracting with certain biotech providers in most situations, the bill charges the Office of Management and Budget with publishing a list of biotechnology companies of concern. That list is to include BGI (formerly Beijing Genomics Institute), BGI subsidiaries MGI and Complete Genomics, Wuxi Apptec, and Wuxi Biologics, according to the legislation.

DNA, mRNA, peptides, cells … everything’s possible in cancer vaccines

Immunotherapy-based cancer vaccines could permanently kill tumors by stimulating immune cells in multiple ways. At the 27th Annual Meeting of the American Society of Gene & Cell Therapy, researchers presented their advances in this field with different techniques in the scientific symposium, “Novel nucleic acid and cell-based vaccines for cancer,” organized by the infectious diseases and vaccines committee.

Court: Bystolic patent settlements not antitrust

Patent settlements involving Forest Laboratories LLC’s blood pressure drug Bystolic were not illegal pay-for delay deals, the U.S. Court of Appeals for the Second Circuit said yesterday in affirming a lower court’s dismissal, twice, of an antitrust suit brought by several payers. The ruling relied on the 2013 Supreme Court decision in FTC v. Actavis Inc. in which the high court said reverse payments do not violate antitrust laws if they “represent fair value for goods or services exchanged as part of a bona fide commercial relationship,” according to the Second Circuit. The appellate court found that, in this case, the plaintiffs failed to plausibly allege that any of Forest’s reverse payments to generic companies involved in the settlements were unjustified or unexplained. The ruling was a defeat for the FTC, which had filed an amicus brief on behalf of the plaintiffs.

BioWorld reader feedback needed: Cyberattack impact survey

The recent cyberattack on Change Healthcare, a part of the Unitedhealth Group, crippled reimbursement claims processing for thousands of providers for several weeks and potentially exposed troves of patient data. Congressional committees are investigating the attack, its scope, and Unitedhealth’s response. Are there downstream impacts that are being overlooked in the biopharmaceutical and/or medical technology sectors? Please take a few minutes to fill out this short survey to help BioWorld understand how your company is responding to these threats. You can click through to the questions here.

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