Venture firm Flagship Pioneering Inc. and Pfizer Inc. are each investing $50 million in a pact that could result in the development of 10 programs, each worth a potential $700 million in milestones. Altogether, if all products are successfully commercialized, the deal could be worth $7 billion, with the milestone money going to some of Flagship’s 45 bioplatform companies. Flagship was founded in 2000 and has since launched companies like Denali Therapeutics Inc., Foghorn Therapeutics Inc., Generate Biomedicines, Moderna Inc., Omega Therapeutics Inc., Sana Biotechnology Inc., Seres Therapeutics Inc., Tessera Therapeutics and a host of others.

Lilly’s phase III of donanemab in Alzheimer’s slows decline

Strong and complete phase III results for Eli Lilly and Co.’s donanemab for treating early Alzheimer's disease (AD) will no doubt inspire more comparisons with recently approved Leqembi (lecanemab). Newly released data for donanemab show it significantly slowed cognitive and functional decline for those with amyloid-positive early symptomatic AD, which lowered the disease-progression risk. A subpopulation analyses showed participants at the earliest stage of disease had a greater benefit, with 60% seeing their decline slow when compared to placebo. Leqembi, the latest amyloid beta-targeting antibody to receive U.S. FDA approval, reduced clinical decline by 27% vs. placebo at 18 months (p=0.-00005) in a phase III study that led to its approval.

Apellis stock flops on retinal vasculitis reports with newly launched Syfovre

Apellis Pharmaceutical Inc.’s shares nosedived by 37% July 17 after the American Society of Retina Specialists (ASRS) wrote to ophthalmologists warning them of the risk of serious intraocular inflammation events in patients following injection of the company’s geographic atrophy drug, Syfovre (pegcetacoplan). The ASRS’s Research and Safety in Therapeutics (ReST) committee received reports during late May and early June of six cases of occlusive retinal vasculitis shortly after (between seven and 13 days) patients were given their first injection of the drug. Retinal vasculitis is a rare but known risk factor associated with intravitreal therapies, and the occurrences were of particular concern to the committee given the drug was only launched in February.

Genequantum strikes deal for its ADC conjugation technology with Inxmed

On the heels of a licensing deal last week, Genequantum Healthcare Co. Ltd. has struck another deal, this time out-licensing its conjugation technology to Inxmed Co. Ltd. to support development of next-generation targeted antibody-drug conjugates (ADCs). Under the terms, Genequantum will provide Inxmed with site-specific glycan conjugation technology, stable linker small-molecule technology and its intelligent continuous conjugation production platform. This will support Inxmed, which works in the field of tumor resistance and metastasis, in the development of innovative ADC drugs. In exchange, Genequantum will receive an undisclosed up-front payment and will be eligible for milestone payments related to specific development and sales milestones as well as sales-based royalties.

CIDP phase II scores for Argenx; read-through to Immunovant

Argenx SE rolled out stock-boosting news July 17 from the phase II Adhere study with Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in chronic inflammatory demyelinating polyneuropathy (CIDP). Adhere met its primary endpoint (p=0.000039), turning up a significantly lower risk of relapse with the drug compared to placebo. The results have Wall Street speculating about other developers in CIDP as well as those such as Immunovant Inc. with similar mechanisms of action to Vyvgart Hytrulo, a neonatal Fc receptor blocker and endoglycosidase combination. Bearing the same active ingredient, Vyvgart (efgartigimod alfa-fcab) was cleared by the U.S. FDA in December 2021 for generalized myasthenia gravis (gMG). Vyvgart Hytrulo, given subcutaneously, was given the go-ahead in June for gMG and deploys Halozyme Therapeutics Inc.’s Enhanze technology. Shares of Argenx (NASDAQ:ARGX) jumped 28%, or $105.35, to close at $484.43 on the day that the trial data became public, and were trading midday July 18 at $522.44, up $38.01.

HLB Group subsidiary, Elevar, confirms FDA to review rivoceranib by 2024

Elevar Therapeutics Inc. said that the U.S. FDA accepted its NDA for oral tyrosine kinase inhibitor rivoceranib in combination with PD-1 inhibitor camrelizumab (Airuika) as a first-line treatment for liver cancer. The FDA stamped an official PDUFA target action date of May 16, 2024. “Rivoceranib plus camrelizumab demonstrated the potential to improve lives of those confronted with unresectable hepatocellular carcinoma,” Elevar CEO Saeho Chong said. “With the FDA’s acceptance of our new drug application, we are pleased to reach another milestone for this combination therapy.”

US House committee keen on faster Medicare coverage, but CMS resources a sticking point

A committee of the U.S. House of Representatives met to review the Medicare coverage procedure for innovative drugs and devices, an event that seemed to gin up support for legislation that would help to streamline those processes. The bigger problem for drug and device makers, however, may be that the Centers for Medicare & Medicaid Services continues to labor under a flat appropriations picture that is eroding daily thanks to inflation.

US CMS proposes loosening the leash on amyloid PET scans

Responding to medical advances and new standards of care in Alzheimer’s, the U.S. Centers for Medicare & Medicaid Services (CMS) is proposing to end its 10-year-old coverage with evidence development policy that has limited Medicare payment of amyloid PET scans to once in a lifetime for beneficiaries. The proposal released yesterday would do away with the blanket coverage imposed in 2013, leaving the decision to Medicare administrative contractors of whether to cover PET beta amyloid imaging more than once per patient, as well as its use within or outside of a CMS-approved study.

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