Gilead Sciences Inc. tallied a phase III interim analysis win showing that the Foster City, Calif.-based company’s twice-yearly injectable HIV-1 capsid inhibitor, lenacapavir, yielded 100% efficacy for the investigational use of HIV prevention in cisgender women. The effort called Purpose 1 met its key efficacy endpoints of superiority of twice-yearly lenacapavir to once-daily oral Truvada (emtricitabine 200 mg and tenofovir disoproxil fumarate 300 mg) and background HIV incidence. It’s the first phase III HIV prevention study ever to show zero infections. Based on the findings, the independent data monitoring committee recommended that Gilead stop the blinded phase of the trial and offer open-label lenacapavir to all participants. Shares (NASDAQ:GILD) didn’t react much, trading at $66.33, up $3.18.

Ionctura aiming to reopen PI3K-delta space, adds €80M in series B

Ionctura SA closed a €80 million (US$86 million) series B round as it prepares to accelerate development of lead asset roginolisib in the treatment of uveal melanoma. That follows publication of phase Ib data showing roginolisib, an orally available allosteric modulator of the delta isoform of PI3K (phosphatidylinositol-4,5-bisphosphate kinase), is distinguished from other drugs in this class, in being well-tolerated over long periods, with patients having been treated for up to 38 months. Thirteen of 16 patients, all of whom had received one or two prior therapies, remained alive and on therapy at 12 months.

With new Huntington’s data, FDA lifts partial hold on PTC phase II

A nearly two-year-old partial clinical hold has been lifted by the U.S. FDA on PTC Therapeutics Inc.’s phase II study of Huntington’s disease. The agency paused enrollment in October 2022, saying it wanted more data on PTC-518, an orally bioavailable small-molecule splicing modifier, before enrollment could continue. Newly released interim results showed that at month 12, the therapy produced dose-dependent lowering of mutant huntingtin protein in blood and cerebrospinal fluid. The interim data also showed favorable trends on assessments including Total Motor Score and the Composite Unified Huntington’s Disease Rating Scale. The FDA liked the new data enough to lift the partial hold, according to PTC.

Alzheon nabs $100M in series E to advance oral Alzheimer’s drug

Alzheon Inc. has raised $100 million in a series E financing round to push its oral drug candidate for early Alzheimer’s disease, ALZ-801 (valiltramiprosate), through a late-stage, Apolloe4 study. On the brink of a data readout in the third quarter of 2024, Framingham, Mass.-based Alzheon is approaching regulatory filings in the U.S. this year, Martin Tolar, founder, president and CEO of Alzheon, recently told BioWorld.

China’s NMPA clears Dizal’s golidocitinib in T-cell lymphoma

China’s National Medical Products Administration (NMPA) approved Dizal Pharmaceutical Co. Ltd.’s golidocitinib for treating adults with relapsed or refractory (r/r) peripheral T-cell lymphoma (PTCL) whose disease has progressed or was refractory to at least one prior systemic therapy. The approval of golidocitinib (DZD-9008, AZD-4205), which marks the first global approval for a JAK1 inhibitor for the treatment of r/r PTCL, is supported by the multinational pivotal Jackpot-8B study that demonstrated superior and durable clinical benefits and a favorable safety profile compared with existing treatment options.

Informed by experience, FDA updating interchangeability guidance

Now that it has some experience under its belt, the U.S. FDA is proposing an update to its guidance on demonstrating interchangeability with a reference biologic. In lieu of the switching studies currently required, the draft guidance being issued this week allows sponsors to provide an assessment of why the comparative analytical and clinical data provided in the BLA or supplement support a showing that the switching standard has been met. The current guidance was released before the FDA had received or reviewed an interchangeable application. Now that the agency has approved several interchangeable biosimilars, its scientific approach to when a switching study or studies may be needed has evolved, according to a notice to be published in tomorrow’s Federal Register.

Africa looks to do for itself with vaccine effort

The African Vaccine Manufacturing Accelerator is up and running, with more than $1 billion already pledged to the effort aimed at supporting the sustainable growth of Africa's manufacturing base and contributing to the African Union's goal of producing most vaccines required by African countries on the continent. A major goal of the accelerator, which launched today in Paris, is to diversify global vaccine suppliers with at least four African vaccine manufacturers entering the market in a sustainable way. “The African Vaccine Manufacturing Accelerator is the next step forward in achieving the African ambition of producing 60% of vaccines locally,” said Jutta Urpilainen, EU commissioner for international partnerships.

Also in the news

Abbvie, Aurion, Aurobac, Brainstorm Cell, Cellxlife, Daewoong, Dizal, ITM, Kactus, Lexicon, Longeveron, Medicinova, Neurona, Opthea, Puretech, Sobi, Woolsey, Xbiotech, Yoltech