Once-monthly subcutaneous injections of CSL Ltd.’s CSL-312 (garadacimab) significantly reduced the rate of heart attacks in patients with hereditary angioedema (HAE) compared to placebo, meeting both primary and secondary endpoints in the pivotal phase III Vanguard trial. Based on the trial results, CSL will file global regulatory submissions later in 2023. A humanized anti-factor XIIa monoclonal antibody, garadacimab is self-administered by subcutaneous administration once monthly, which is a huge convenience for patients, Andrew Nash, CSL’s chief scientific officer and senior vice president for research, told BioWorld.

Fed Circuit orders Jazz to delist REMS patent

While the FDA has yet to take a stance on whether risk evaluation and mitigation strategy (REMS) patents can be listed in its Orange Book, the U.S. Court of Appeals for the Federal Circuit weighed in Feb. 24 with a decisive “no” when those patents only claim systems. In affirming a lower court decision in Jazz Pharmaceuticals Inc. v. Avadel CNS Pharmaceuticals LLC, the appeals court gave Jazz 14 days to delist the ’963 patent related to the REMS program for its narcolepsy drug, Xyrem (sodium oxybate). The court’s precedential opinion could impact other drug companies that have REMS patents listed in the Orange Book, which provides patent and exclusivity information about approved drugs with therapeutic equivalence evaluations.

Ethical considerations open FDA’s Rare Disease Day

The U.S. FDA is marking the 40^th anniversary of the Orphan Drug Act with Rare Disease Day 2023. Robert Califf, the agency’s commissioner, opened the day by expressing his wonder and accompanying concern regarding gene editing and gene therapy. The new technology is dramatically beneficial, Califf said, but the long-term benefits are still a big unknown. He recommended more follow-up studies for research involving the genome and he cited recent guidance on ethical considerations in studies that include children. This kind of expansion, he added includes extending these considerations into the curriculum of medical schools. The Orphan Drug Act, which helps speed approval for rare disease treatments, has seen the approval of more than 550 unique drugs and biologics for more than 1,100 indications.

Newco news: Aqemia making waves in computational drug discovery

For Aqemia SA, the year got off to a good start, as one of its pharma partners, Les Laboratoires Servier SAS, extended an existing collaboration to drug a supposedly undruggable immuno-oncology target, using its Launchpad artificial intelligence platform. The Paris-based company has started to make waves with a novel approach to identifying small-molecule binders to proteins, which is based on a highly efficient algorithm that emerged from academic physics research conducted by its CEO and co-founder Maximilien Levesque.

Transthera raises $38M, propelling TT-0420 toward market

Transthera Sciences Inc., a Nanjing, China-based company developing small-molecule therapies in cancer, inflammation and cardiovascular disease, raised ¥260 million (US$38 million) in a series D+ funding round to advance its first drug product. “Following the financing, we will speed up the commercialization and the expansion into the global market,” said Wu Yongqian, founder and chairman of Transthera. So far, Transthera has seven candidates in the pipeline, including lead asset TT-0420 (tinengotinib), which has fast track and orphan drug designations in the U.S. where it is in a phase II trial for cholangiocarcinoma, a type of cancer that forms in the bile ducts.

HIV cure, a less uncertain journey

HIV research is a winding road where one obstacle leads to another, slowing down success. The first barrier to getting the cure starts before one can even talk about it. “Cure may be too powerful and promising a term. Remission is probably better,” said John Mellors. His work led to the universal use of plasma HIV-1 RNA and CD4+ T-cell counts in HIV-1 infection. He is one of the parents of antiretroviral therapy and a professor in the Department of Immunology at the University of Pittsburgh. “Cure means maintaining an undetectable viral load off antiretroviral treatment. That means you cannot transmit it to people,” Sharon Lewin told BioWorld. Lewin is the director of The Peter Doherty Institute for Infection and Immunity in Melbourne, and the president of the International AIDS Society.

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