CSL Ltd. inked a potential $2.1 billion deal with Dutch biotech company, Varmx BV, to develop VMX-C001 as a new treatment to restore blood coagulation in patients taking a Factor Xa inhibitor. Under terms of the deal, CSL will pay $117 million up front to Varmx for an exclusive option to acquire the company. CSL will have the right to exercise the option after it receives phase III data for VMX-C001. Subject to certain milestones and regulatory clearances, Varmx will receive payments of up to $388 million upon launch of VMX-C001, with a potential $1.7 billion tied to commercial milestones.

Animosity toward mRNA COVID vaccines puts basic science at risk

Part two of a three-part series in mRNA vaccine research looks at an ongoing concern for scientists of across-the-board funding cuts. This is already happening in mRNA research, where reductions affected coronavirus-related projects. During the pandemic, efforts focused on this pathogen, and once the health emergency was over, grants for antivirals were eliminated. However, these drugs could stem future outbreaks. Despite the cuts, recent research continues to demonstrate the potential of mRNA, not only for the development of antivirals, but also for obtaining more effective and longer-lasting vaccines.

Areteia’s dexpramipexole phase III win in eosinophilic asthma

Three-year-old Areteia Therapeutics Inc. hit the primary endpoint in its Exhale-4 phase III study of dexpramipexole for eosinophilic asthma, bringing the oral small molecule one step closer to the U.S. market, where it could challenge currently approved injectable anti-IL-5/5R biologics. A repurposed eosinophil maturation inhibitor, dexpramipexole was originally developed for neurological indications by Biogen Inc. and Knopp Biosciences LLC, which created Areteia in 2022 with Population Health Partners. Dexpramipexole was well-tolerated and showed a statistically significant improvement in lung function in the 150-mg twice-daily dexpramipexole group and a statistically significant reduction in blood absolute eosinophil count in the 150-mg and the 75-mg twice-daily groups, averaged over weeks 20 and 24, compared to placebo.

Novo’s amylin analogue cagrilintide encouraging in obesity phase III

Novo Nordisk A/S rolled out positive data from a sub-analysis of the phase III Redefine-1 trial with long-acting amylin analogue cagrilintide at the European Association for the Study of Diabetes congress in Vienna, Austria. The work evaluated the efficacy and safety of cagrilintide 2.4 mg administered once per week as monotherapy, plus lifestyle intervention, for adults with obesity or overweight and a weight‑related comorbidity without diabetes. Novo said the average weight loss was 12.5 kg (11.8% body weight reduction) with cagrilintide compared to 2.5 kg (2.3%) with placebo, after 68 weeks.

Dualitas launches with $65M to advance bispecifics for I&I

Dualitas Therapeutics Inc. has exited stealth mode with a $65 million series A investment. The company has developed a method for screening antibody fragments linked as pairs to discover novel bispecific antibodies. Dualitas has already used the platform to identify two undisclosed novel combinations of targets, one for inflammatory allergic diseases and another for autoimmune diseases.

Advisory committee meetings becoming a rarity at the FDA?

With the U.S. FDA convening only seven advisory committee meetings so far this year for drugs and medical devices, last week’s comments from CDER Director George Tidmarsh that seemed to suggest the agency, or at least CDER, was backing away from the use of the expert panels for individual product approvals could foreclose an important avenue for patients with rare diseases, and the doctors specializing in those diseases, to engage with the FDA. “The science is hard enough” when it comes to developing diagnostics and treatments for rare diseases, John Cox, a partner and co-chair of life sciences at Barnes & Thornburg LLP, told BioWorld. Reducing opportunities to educate FDA staff about a rare disease, makes it even more difficult, he added.

Halt of biopharma investment prompts emergency UK hearing

The U.K.’s failure to join the dots from discovery research to patient access to innovative drugs was cited repeatedly by pharma leaders, who were called to an emergency hearing in parliament on Tuesday to explain decisions to pull out of promised capital investments. The spur was Merck & Co. Inc.’s announcement it is stopping construction of a $1.2 billion research lab in the London constituency of British Prime Minister Keir Starmer, closing a laboratory based at the Francis Crick Institute, and axing all discovery research in the U.K. with the loss of 125 jobs. That urgency was intensified when Astrazeneca plc subsequently said it was pausing a £200 million investment at its global R&D site in Cambridge, and then Eli Lilly and Co said it is holding off on a plan announced in October 2024 to invest £279 million (US$380 million) in the U.K.

Allrock snags $50M for its in-licensed ROCK inhibitor

Allrock Bio Inc. secured $50 million in a series A round to advance ROC-101, an oral pan-rho-associated protein kinase (ROCK) inhibitor to treat pulmonary arterial hypertension and pulmonary hypertension with interstitial lung disease. The phase II-ready asset comes to the startup via a license from Sanofi SA, which acquired the asset through its 2021 buyout of Kadmon Holding Inc.

Also in the news

Acadia, Alterity, Altimmune, Amo, Atavistik, Atyr, Avicanna, Avid, Cadrenal, Catalym, Corstasis, Curanex, Daiichi, Exithera, Fore, Geovax, Humacyte, Kodiak Sciences, Krystal, Kwangdong, Merck, Monopar, Novo Nordisk, Ocugen, Pencil, Som, Theratechnologies