After a delay in November that resulted in a new PDUFA date, Apellis Pharmaceuticals Inc.’s Syfovre (pegcetacoplan injection) received U.S. FDA approval for treating geographic atrophy (GA) secondary to age-related macular degeneration. The treatment’s Feb. 17 approval of the priority NDA came more than a week ahead of its Feb. 26 PDUFA. Syfovre, the first and only FDA-approved treatment for GA, is priced at $2,190 per vial, with dosing regimens staggered every 25 to 60 days. The company said the price reflects the treatment’s clinical profile as the first approved therapy for the indication, while also remaining in line with the price of existing anti-VEGF treatments. The Waltham, Mass.-based company’s stock (NASDAQ:APLS) surged at midday, trading up by about 14% at $63 per share.

Iomab-B radiotherapy data hold strong for Actinium in phase III AML trial

Actinium Pharmaceuticals Inc. unveiled full data from the phase III study Sierra testing Iomab-B in patients age 55 and above with active relapsed or refractory acute myeloid leukemia (AML). The radiotherapy met the primary endpoint of durable complete remission of six months following initial complete remission after bone-marrow transplant with a high degree of statistical significance. First data rolled out last October. Shares of the firm (NYSE:ATNM) fell 16% by midday and were trading at around $11.50.

US CMS told to reconsider Alzheimer’s drug coverage

Now that the U.S. FDA has granted accelerated approval for Biogen Inc./Eisai Co. Ltd.’s early Alzheimer’s drug, Leqembi, the Centers for Medicare & Medicaid Services (CMS) is being pressured to rethink its coverage of amyloid-targeting monoclonal antibodies (MAbs). Under its current national coverage determination, CMS limits coverage for MAbs granted accelerated approval to treat Alzheimer’s and other dementia to beneficiaries enrolled in randomized clinical trials. “This decision creates a barrier to care for older Americans, especially individuals living in rural and underserved areas,” according to a bipartisan letter 20 U.S. senators sent to CMS Administrator Chiquita Brooks-LaSure and Health and Human Services Secretary Xavier Becerra on Feb. 17. Unless CMS reconsiders its decision, “access to disease-modifying therapy for Alzheimer’s disease will be extremely limited or nearly nonexistent,” the senators added.

Fauci at CROI 2023: Viral spillover is forever, but pandemics are preventable

Anthony Fauci has retired from his position as director of the National Institute of Allergy and Infectious Diseases and as chief medical advisor to the U.S. president. But Fauci, who has advised every president since Ronald Reagan, continues to share his encyclopedic knowledge with the HIV research community, as he has since the beginning of the HIV pandemic. Fauci co-founded the first National Conference on Human Retroviruses and related infections in 1993. At the Opening Session of the 30th edition of the Conference on Retroviruses and Opportunistic Infections (CROI), he highlighted the advances that have collectively extended the life expectancy of newly diagnosed patients by decades. At the meeting, Fauci sat down with BioWorld to discuss these major advances.

Carephar’s P-CAB drug gets China nod for duodenal ulcer, reflux esophagitis

Carephar Pharmaceutical Co. Ltd. obtained marketing approval for keverprazan (KFP-H008) from China’s NMPA for use in duodenal ulcer and reflux esophagitis treatments, allowing it to tap an increasingly competitive market. Keverprazan is a P-CAB with a strong acid-suppressive capacity designed for acid-related diseases. P-CABs provide an alternative option for patients, offering more rapidly acting, acid-stable and increasingly potent antisecretory agents.

Hemab, ‘an Alexion five years from now,’ closes $135M series B round

Hemab Therapeutics ApS raised $135 million in a series B funding round to continue its broad effort to build a company focused on developing prophylactic therapies for neglected bleeding and thrombotic disorders. The new cash brings its total equity raise to $190 million. Hemab closed a $55 million series A round in July 2021 after licensing a portfolio of antibodies from Bagsvaerd, Denmark-based Novo Nordisk A/S. The Copenhagen, Denmark-based firm moved its lead drug candidate, HBM-001, into a phase I/II trial in Glanzmann thrombasthenia, last month, and it expects an initial data readout in the second half of this year, CEO Benny Sorensen told BioWorld.

Bridge Biotherapeutics’ BBT-401 misses primary endpoint in phase II ulcerative colitis trial

Bridge Biotherapeutics Inc.’s small-molecule Pellino-1 inhibitor, BBT-401, failed to meet the primary efficacy endpoint in a phase II trial in ulcerative colitis (UC). The phase II mid-to-high dose cohort study included 38 patients with active UC enrolled at 37 clinical sites across the U.S., New Zealand, South Korea, Poland and Ukraine. “Although the phase IIa mid-to-high dose clinical trial did not meet the primary efficacy endpoint, we have confirmed that the drug delivery profile has improved drastically,” a Bridge Biotherapeutics spokesperson told BioWorld.

Biogen terminates collaboration with Innocare on orelabrutinib

Innocare Pharma Ltd. said it is open to new collaborations after partner Biogen Inc. decided to terminate a global deal to develop and commercialize BTK inhibitor orelabrutinib, in development for multiple sclerosis (MS) and other autoimmune diseases. Notice of the termination, disclosed in Biogen’s fourth-quarter 2022 financial report, was “based on the contract term of ‘terminate for convenience,’” Innocare told BioWorld. In July 2021, Biogen acquired global rights to orelabrutinib for MS and certain autoimmune diseases with a $125 million up-front payment, as well as $812.5 million in potential development milestones and commercial payments. It was the first mega out-licensing deal for the Chinese firm.

Holiday notice

BioWorld's offices were closed in observance of Presidents Day in the U.S. No issue was published Monday, Feb. 20.

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