Eculizumab

Today, a brief rundown of news involving Foghorn Therapeutics and Wave Life Sciences, as well as updates from Varda Space Industries and Hatteras Venture Partners that you may have missed.Foghorn Therapeutics is cutting 40% of its staff after a cancer drug it was developing with Eli Lilly failed to meet efficacy benchmarks to advance into further testing. In a Thursday announcement, the biotechnology firm said it is discontinuing its synthetic lethality program FHD-909, as well as a second unnamed experimental medicine targeting the SMARCA2 gene and is ceasing its collaboration with Lilly, which dates back to 2021. Its the latest stumble for Foghorn, whose stock first plunged in 2022 after regulators placed a study of its leukemia drug on hold following patient deaths. Shares are now worth a fraction of their value six years ago, when Foghorn debuted on Wall Street. This is disappointing news, given FHD-909 was the company's lead value driver and most advanced program in clinical development, Guggenheim Securities analyst Michael Schmidt wrote in a note to clients. Shares fell 22% on Thursday.The Food and Drug Administration gave Wave Life Sciences the all-clear for a pathway to accelerated approval following a two-year trial of its RNA editing treatment for alpha-1 antitrypsin deficiency. Waves Chief Medical Officer, in a Thursday announcement, said a registrational trial design that offers an efficient path to full approval, without the need for a separate confirmatory trial, is advantageous. In May, Wave presented data showing its treatment, called WVE-006, helped AATD patients produce therapeutically relevant levels of a protein their bodies dont make. That therapy is in mid-stage testing, and additional data is expected by the end of 2026.Samsung Bioepis and Teva Pharmaceutical have expanded their partnership to develop half a dozen biosimilar drugs, including copycats to FasenraandIlaris. According to a Thursday announcement, Samsung Bioepis will handle R&D and manufacturing, while Teva will commercialize those drugs globally. Previously, the South Korean and Israeli drugmakers had commercialized two biosimilars:Opuviz, a mimic of Eylea, and Epysqli, a drug similar to Soliris. Samsung Bioepis is also developing, on its own, biosimilars for cancer drugs such as Keytruda and immune drugs like Entyvio, Tremfya and Dupixent.Drug discovery biotech Varda Space Industries announced Wednesday it added $251 million to its war chest via a Series D round led by Lux Capital and Natural Capital. Varda hypothesizes that due to the absence of gravity, materials such as the active pharmaceutical ingredients in medicines can be crystallized in ways that would be impossible on Earth, enabling novel drug formulations that can benefit patients. The biotech said it has more than a dozen launches and reentries planned in the next two years, but has disclosed little about the pharmaceuticals it's developing.Hatteras Venture Partners is padding its ranks, adding former GSK and UnitedHealth Group CEO Andrew Witty as its chairman and former Acting FDA Commissioner Ned Sharpless as managing director of a drug discovery initiative. Witty is returning to the North Carolina biotech backer after spending two years as a venture partner there from 2017 to 2019. Sharpless, meanwhile, launched G1 Therapeutics with the aid of Hatteras and is coming to help run Hatteras Discovery Innovation. The venture firm also said it marked the first closing of Hatteras Opportunity Fund II, which will spread $100 million in capital across its existing biotech investments. '

European drug regulators just wrapped another stack of reviews , and the EU bloc now has a sizeable list of likely new treatments to show for it. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has signed off on eight new medicines, two biosimilars and two generics, while also recommending label extensions for 11 other therapies. Among the most notable developments is a positive recommendation for Novo’s Frehemgo, also known as denecimig, to treat or prevent bleeding episodes in adults and children with hemophilia A. Novo is positioning the drug as a next-generation factor VIIIa mimetic. The bispecific antibody works by binding Factor IXa and Factor X to increase thrombin production and improve clotting. Frehemgo is in direct competition with Roche’s blockbuster and current market leader Hemlibra, which made 4.8 billion Swiss francs ($5.81 billion) in sales last year. The CHMP recommendation comes just days after Frehemgo’s first approval in Saudi Arabia and makes way for a possible approval from the European Commission in the coming weeks. It’s a welcome win for Novo after suffering a string of late-stage disappointments in its cardiovascular pipeline amid mounting competition in metabolic medicine markets. The Danish drugmaker expects an initial launch of Frehemgo by years' end, with a broader rollout in early 2027. AstraZeneca’s rare disease business Alexion also snagged a recommendation from the CHMP, setting up dual-binding nanobody C5 inhibitor Klygefa for a formal approval from the EC. Klygefa, also known as gefurulimab, received the CHMP go-ahead as a treatment for adult patients with generalized myasthenia gravis (gMG), a chronic autoimmune disease marked by muscle weakness caused by the immune system’s attack on the connection points between the nerves and muscles. The treatment marks AstraZeneca’s continued investment in the C5 inhibitor class after Soliris and Ultomiris. But unlike those earlier infused drugs, Klygefa is a self-administered injection, offering patients more control and flexibility. If approved, Europe will be Klygefa’s second approval after Japan earlier this month and it will be the first dual-binding nanobody C5 inhibitor approved for gMG patients. Beyond those meds, new offerings from SFL Pharmaceuticals, Corcept, Incyte Biosciences and Sanofi also made the cut at CHMP this week. SFL scored double with a positive recommendation for both Gevalka and Sepalna. Gevalka was recommended in adults with anaplastic lymphoma kinase-positive advanced non-small cell lung cancer, a version of the disease more common in young patients who have never smoked. And Sepalna was recommended as a maintenance treatment of advanced epithelial high-grade ovarian, fallopian tube, or primary perineal cancer. Corcept’s Lifyorli, meanwhile, snagged a recommendation as a combo treatment with chemotherapy for platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer. Incyte Biosciences’ Povofortay will take a positive review to the European Commission for acne inversa. And rounding out the list of endorsements was Sanofi’s rabies vaccine VaxRabeo. Alfasigma’s Jyseleca, AstraZeneca and Daiichi Sankyo's Enhertu, Merk & Co.'s Keytruda, Novartis’ Cosentyx, Leo’s Anzupgo, and Roche’s Ocrevus, among others, took positive opinions for their proposed label expansions. In a setback for MaaT Pharma, the CHMP elected—after reexamination—to uphold its decision to recommend against Xervyteg as a treatment for acute graft-versus-host disease, a life-threatening condition in which donor bone marrow or stem cells attack the patient. The agency maintained that MaaT’s single-arm trial did not show a sufficient benefit-risk profile. In response, the pharma proposed a phase 3 trial evaluating Xervyteg against a best available therapy to satisfy regulator concerns. All CHMP recommendations now head for the European Commission for final sign-off.