Just as it is for terminally ill cancer patients, time is of the essence for people in the early stages of Alzheimer’s disease. Thus, the clinical meaningfulness of Eli Lilly and Co.’s donanemab is the time it gives patients before the disease progresses, Reisa Sperling, a neurology professor at Harvard Medical School and director of the Center for Alzheimer Research and Treatment at Brigham and Women’s Hospital, told the U.S. FDA’s Peripheral and Central Nervous System Drugs Advisory Committee yesterday. Presenting on behalf of Lilly, Sperling said patients in the donanemab trials gained an average of five to seven months before disease progression and there’s potential for an even greater gain over the long term. While the adcom voted unanimously in support of donanemab, the question is whether the FDA and payers, especially the Centers for Medicare & Medicaid Services, will be as flexible in approving and covering Alzheimer’s treatments in the future as they are with cancer drugs.

Ipsen wins FDA nod for Iqirvo in primary biliary cholangitis

Ipsen SA picked up U.S. FDA accelerated approval for its Genfit SA-licensed elafibranor, making it the first new drug in eight years for treating primary biliary cholangitis (PBC), though a potential competitor lurks just around the corner. Elafibranor, branded Iqirvo, which has breakthrough therapy designation, is cleared for use in second-line PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as a monotherapy in those unable to tolerate UDCA. It is expected to be available immediately for eligible patients. Continued approval may be contingent on confirmatory data, specifically looking at improvement in survival and prevention of liver decompensation events. A long-term study, Elfidence, is ongoing.

Newco news: Syntis Bio launches for oral obesity, rare disease drugs

Focused on oral therapies for obesity, diabetes and rare diseases, a new Boston-based company built out of Massachusetts Institute of Technology (MIT) research has launched to advance its synthetic tissue-lining technology and a pipeline of candidates. Syntis Bio Inc., which raised $15.5 million through seed funding last year, was co-founded by Rahul Dhanda, who serves as its president and CEO, and two MIT engineers Robert Langer and Giovanni Traverso. The company’s lead program, SYNT-101, is in human trials as a once-daily oral therapy for obesity. It also has two first-in-class oral enzyme replacement therapies for homocystinuria and maple syrup urine disease, acquired in April from Codexis Inc., of Redwood City, Calif. Both of the enzyme therapies are ready for IND-enabling studies.

Acepodia’s antibody cell conjugation platform could disrupt CAR Ts  

Acepodia Inc.’s antibody cell conjugation platform (ACC) could change the way CAR T-cell therapies are manufactured, clearing the way for an off-the-shelf model to treat numerous cancers that is safer and cheaper than current CAR Ts, Acepodia CEO Sonny Hsiao told BioWorld. The ACC platform is based on chemistry developed by Chief Scientific Adviser Carolyn Bertozzi, who won the Nobel Prize in chemistry in 2022 for discovering a novel form of click chemistry – known as bioorthogonal chemistry – that integrates cellular functions without disrupting the normal functions of a cell. Acepodia applied click chemistry to create an off-the-shelf, non-genetically engineered version of CAR T-cell therapy that is more easily scaled and avoids cytokine release storms, neurotoxicity and other side effects associated with CAR T-cell therapies.

Adcytherix aims for innovation as startup in crowded ADC space

Fresh from the sale of one antibody-drug conjugate (ADC) company, Jack Elands has formed another, raising €30 million (US$32.2 million) in seed funding for Adcytherix SAS. The newco brings together the same cast as Elands’ former company, Emergence Therapeutics, which was sold to Eli Lilly and Co. for an undisclosed sum in a deal that closed in August 2023. Marseille, France-based Adcytherix also has the same backers as Emergence, with the seed round led by Pontifax Venture Capital and supported by RA Capital Management, KKR and Pureos Bioventures

Also in the news

4DMT, 4moving, Actinium, Adverum, Almirall, Arcturus, Astrazeneca, Cantargia, Cybin, Eisai, Eli Lilly, Entos, GC, GSK, Lipocine, Longboard, Maia, Moderna, Moonlake, Novel, Oculis, Pacylex, Pepgen, Prokidney, Quralis, Roche, Santhera, Sellas, Shattuck Labs, Sisaf, Skye, Skyhawk, Surrozen, Tharimmune, Thermosome, Vertex