After a difficult start, there’s been more progress in PIK3 drug class, with FDA approval of Novartis AG’s Vijoice (alpelisib) for the rare condition PIK3CA-related overgrowth spectrum (PROS).
In a first of its kind phase I trial, Rejuvenate Biomed NV has secured ethical clearance to induce acute sarcopenia in 42 healthy subjects, aged 65 to 75 years old. That will be achieved by placing a cast on one leg to induce sarcopenia through muscle disuse. The volunteers in the double-blind trial will then receive either RJx-01, an oral small molecule for treating sarcopenia, or placebo.
The challenging financial environment of 2022 will lead to an increase in partnerships, according to representatives of the two big Swiss biopharma companies, Roche Holding AG and Novartis AG.
Moonlake Therapeutics AG is making headway on its mission to establish a broad dermatology and rheumatology franchise around its sole product, sonelokimab, announcing it is poised to start a phase II study in hidradenitis suppurativa.
The U.S. FDA has approved Novartis AG’s Pluvicto (lutetium Lu 177 vipivotide tetraxetan, formerly referred to as 177Lu-PSMA-617) for treating adults with metastatic prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer. The treatment is the indication’s first FDA-approved targeted radioligand therapy that contains a radioisotope.
Targeted radiopharmaceutical specialist Precirix NV has closed an €80 million (US$87.8 million) series B financing, enabling it to complete phase II development of CAM-H2, a potential treatment for HER2-positive metastatic breast cancer and gastric cancer. The round follows initial data showing there were no dose limiting toxicities in the first cohort of patients in the phase I/II trial, and a positive review from the safety committee.
Though the Ukraine war has had an impact on the availability of medical devices and diagnostics, a number of companies based in the U.S. and Europe have announced measures to ensure their products will reach the war-torn nation. Device companies are donating millions to nongovernmental organizations for humanitarian assistance, while the Advanced Medical Technology Association (Advamed) said its member companies are tracking the situation and are eager to pitch in with desperately needed supplies and medical equipment.
Novartis AG, an early and active player in bringing gene therapies to market, has agreed to pay Voyager Therapeutics Inc. $54 million up front and up to $1.7 billion in fees and milestone payments for options to license up to five next-generation adeno-associated virus (AAV) capsids to use as gene therapy vectors for neurological diseases.
Third Harmonic Bio Inc. CEO Natalie Holles said the company’s $105 million in series B money will push THB-001 – a first-in-class, highly selective, oral inhibitor of wild-type KIT – “well past the first proof-of-concept study in inducible urticaria [hives].”
LONDON – “I really believe we can start using the word cure,” said the pioneer of chimeric antigen receptor T-cell (CAR T) therapy Carl June, as he revealed two leukemia patients he treated in a phase I trial have now been in remission for 10 years. Both patients with chronic lymphocytic leukemia achieved complete remission shortly after treatment in 2010. The genetic modification has remained detectable in their CAR T cells for more than 10 years of follow-up, June said, describing details of the case studies published in Nature Feb. 2.
