To no great surprise, the U.K.’s health technology assessment body has found that the benefits of the first two approved Alzheimer’s disease drugs are too small to justify the costs. Neither Kisunla (donanemab, Eli Lilly & Co. Inc.) or Leqembi (lecanemab, Eisai Co. Ltd.) “demonstrate sufficient benefit to justify their high cost, including the cost of administering them,” the National Institute of Health and Care Excellence (NICE) concluded after an extended appraisal of the two amyloid neutralizing antibodies. The drugs “should not be provided on the National Health Service (NHS), as they are not good value for money,” NICE’s appraisal committee said. “For us to be able to approve a medicine for use in the NHS, it must not only represent a step forward in treatment, but it must also represent a good use of NHS resources and taxpayers’ money.”

CBER shakeup points to new FDA alignment in rare diseases

When asked by BioWorld if the Center for Biologics Evaluation and Research’s Office of Therapeutic Products director and deputy director had been forced out and if so, why, an HHS spokesperson responded on background with a single sentence: “Center directors deserve to be supported by managers that are aligned with aggressive goals to expeditiously advance therapeutics for rare diseases using the gold standard of science.” The Alliance for Regenerative Medicine said it is “deeply disappointed to learn” that director Nicole Verdun and deputy director Rachael Anatol “have been placed on administrative leave.” The alliance praised the two for having “modernized the FDA’s regulatory approach” to cell and gene therapy.

CHMP recommends approval of Rezdiffra, Ogsiveo and others

One year after the FDA’s nod, the EMA is following on and recommending conditional approval of Madrigal Pharmaceuticals Inc.’s Rezdiffra (resmetirom) as the first drug in Europe for treating noncirrhotic metabolic dysfunction-associated steatohepatitis. And less than two months after Merck KGaA announced it was to acquire Springworks Therapeutics Inc., for $3.9 billion, the Stanford, Conn-based company met with CHMP’s approval of Ogsiveo (nirogacestat) for the treatment of non-malignant desmoid tumors that form in fibrous tissue.

FDA scrutinizing trials sending US patient cells to China

Using informed consent to do what Congress couldn’t, the U.S. FDA is flexing its regulatory authority to halt clinical trials that involve sending cells from American patients to China or other adversarial nations for genetic engineering and subsequent infusion back into the patient. In announcing an immediate review of the trials Wednesday, the agency cited “mounting evidence that some of these trials failed to inform participants about the international transfer and manipulation of their biological material and may have exposed Americans’ sensitive genetic data to misuse by foreign governments.”

More HAE prospects in the works as CSL celebrates Andembry approval

CSL Behring LLC’s regulatory win June 16 with humanized anti-factor XIIa monoclonal antibody Andembry (garadacimab CSL-312) to prevent attacks of hereditary angioedema (HAE) sharpened focus on the space, where drugmakers continue to push their pipelines along. Oral on-demand therapies as well as long-term prophylaxis agents for HAE are among the options. The U.S. FDA go-ahead for Andembry came on the heels of approvals in a handful of other markets

Drug Farm harvests new immunomodulators with AI-gene editing tech

Shanghai Yao Yuan Biotechnology Ltd., also known as Drug Farm, is advancing a portfolio of immune-modulating therapies for various indications including hepatitis B virus, hepatocellular cancer and a relatively new rare genetic disease called ROSAH, an acronym for retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache. The assets are built on the company’s AI and gene editing technology platforms, according to Drug Farm Chief Operating Officer Tony Xu, who cofounded the Shanghai and Albany, N.Y.-based biotech in October 2015 with CEO Tian Xu.

Keymed launching Stapokibart, advancing pipeline, with $125M raise

Keymed Biosciences Co. Ltd. has raised HK$982 million (US$125 million) in a placement on the Hong Kong Stock Exchange (HKEX:2162) to commercialize its IL-4Rα monoclonal antibody CM-310 branded as Stapokibart, and to advance its larger pipeline. The placement consisted of 21.6 million shares priced at HK$45.48 per share. Morgan Stanley was the lead bookrunner. Keymed said 30% of the funds raised would be used to commercialize Stapokibart, 35% would go toward R&D expenses to advance CM-512 and CM-518D1 and other projects, 25% would go toward capital expenditures for manufacturing and R&D facilities, with the remaining 10% going toward corporate purposes.

Immuno Cure, Pharmjet partner on needle-free HIV vaccine trial

Immuno Cure Biotech Ltd. is collaborating with Pharmajet Inc. to evaluate the safety and immunogenicity of its HIV therapeutic DNA vaccine, Icvax, delivered via Pharmajet's innovative Tropis needle-free injection system. The two companies signed a material transfer agreement at the 2025 Biotechnology Innovation Organization’s International Convention in Boston. Icvax employs Immuno Cure's PD-1-enhanced DNA vaccine technology that aims to achieve sustained, immune-mediated HIV-1 virological control without the need for antiretroviral therapy (ART). Although highly efficacious, ART does not cure HIV/AIDS. Globally, there are more than 39 million people living with HIV, and more than 40 million people have died since HIV/AIDS was discovered in 1981.

Holiday notice

BioWorld's offices were closed in observance of Juneteenth in the U.S. No issue was published Thursday, June 19.

Also in the news

Abbvie, Actio, Acurx, Aligos, Applied, Aptevo, Atalanta, Avalo, Bavarian Nordic, Bioaegis, Biomea Fusion, Camurus, Cantargia, Cardiff, Climb, Cynaptec, Diagonal, F2G, Galderma, Grin, Lobe Sciences, Medicovestor, Moleculin, Nanocell, Naya, Nervgen, Neurvati, Nob Hill, O2nix, Oncoinvent, Polyactiva, Rewind, Scholar Rock, Senti, Shionogi, Sinovac, Sling, Synfini, Tolremo