NEW YORK – Kaiseraugst, Switzerland-based chemistry, manufacturing, and control analytics firm Solvias this week said it has inked a long-term agreement with Vertex Pharmaceuticals to perform analytical release testing services for the company's CRISPR gene-editing therapy Casgevy (exagamglogene autotemcel), which late last year received approval in the US as a treatment for sickle cell disease. Solvias said it has worked with Vertex for several years to develop and validate test methods for the final release of patients' autologous edited cells as part of the therapy.
Bristol Myers Squibb this week completed its acquisition of Mirati Therapeutics. The deal, worth $4.8 billion, was announced in October. The acquisition added Mirati's KRAS G12C inhibitor Krazati (adagrasib) to BMS’s portfolio, along with a pipeline of other oncology targeted therapy assets including a KRAS G12D inhibitor, a PRMT5 inhibitor, and an SOS1 inhibitor. With the deal complete, Mirati shares have ceased trading on the Nasdaq, and the firm is now a wholly owned subsidiary of BMS.
Apollo Cancer Centers this week said it has launched India's first AI-precision oncology center. The center will include services for diagnosis, cancer risk assessment, treatment, and continuity of care including patient management based on genomic, clinical, and pathological profiles.
The US Food and Drug Administration this week notified manufacturers of BCMA- and CD19-directed genetically modified autologous CAR T-cell immunotherapies that they must revise product package inserts to include a boxed warning about the risk of T-cell malignancies with serious outcomes and update the medication guide for the products to identify the possibility of increased risk of cancer. In November, the FDA warned patients receiving autologous CAR T-cell therapies targeting BCMA and CD19 that they may be at risk of developing new T-cell malignancies based on reports of T-cell cancers within clinical trials and in the post-market setting.
Tokyo-based Kyowa Kirin this week completed its acquisition of London-based gene therapy developer Orchard Therapeutics, which was first announced in October. As part of the deal, Orchard is now a wholly owned subsidiary of Kyowa Kirin. The acquisition will bolster Kyowa Kirin's portfolio with Orchard's gene therapy for early-onset metachromatic leukodystrophy, Libmeldy (atidarsagene autotemcel), which has been approved in the EU and UK. Orchard is also developing gene therapies in mucopolysaccharidosis type I Hurler's syndrome and mucopolysaccharidosis type IIIA.
The US Food and Drug Administration has granted Emeryville, California-based 4D Molecular Therapeutics rare pediatric disease designation for aerosolized 4D-710, an experimental treatment for cystic fibrosis lung disease that delivers a functional CFTR transgene, the firm said this week. Cystic fibrosis is a progressive disease caused by mutations in the CFTR gene and that can lead to impaired lung function, and lung disease is the leading cause of morbidity and mortality in people with cystic fibrosis. The treatment is currently being tested in a Phase I/II study called AEROW, and 4DMT said it expects to publish interim data from the trial midyear.
Merck said this week that its board of directors has declared a quarterly dividend of $.77 per share payable on April 5 to Merck shareholders of record as of the close of business on March 15.
Repare Therapeutics said this week that it earned a $40 million milestone payment under its licensing agreement with Roche for ATR inhibitor camonsertib. The payment was triggered by the first patient being dosed in the Phase II TAPISTRY platform trial, conducted by Roche. The trial includes two arms evaluating camonsertib: one in patients with ATM loss of function tumors and the other in SETD2 loss of function tumors. Roche licensed the drug from Repare in 2022 in an agreement that included a $125 million upfront payment and up to $1.2 billion in milestone payments.
Tempus said this week that it has signed agreements for in-network provider status with Humana. Under the agreement, Humana’s over 13 million members will have improved access to Tempus’ portfolio of molecular profiling tests. The company said that in-network coverage of its assays will support broader access to precision cancer care and may lead to lower out-of-pocket costs for patients.
Spanish biotech firm Amadix said this week that its colorectal cancer blood test PreveCol has received breakthrough device designation from the US Food and Drug Administration. According to the company, the assay has been shown to have better diagnostic efficacy than currently approved colorectal cancer screening tests. Breakthrough device designation offers companies expedited assessment by the FDA for premarket approval of tests.
Jupiter Medical Center, a not-for-profit medical center in Jupiter, Florida, said this week that it has partnered with My Gene Counsel, a digital health company, to scale its genetic counseling and testing for cancer. Under the collaboration, JMC will use My Gene Counsel's Living Lab Report for all current and past patients who have been seen at the center for genetic counseling. The report will provide JMC patients with updated genetic counseling information as new disease details are discovered, therapies developed, guidelines change, or genetic variants are reclassified.
Novato, California-based Ultragenyx Pharmaceutical this week said it has treated all patients receiving its investigational gene therapy candidate for Wilson disease, UX701, across three dose-escalation cohorts in the first stage of the ongoing Phase I/II/III Cyprus2+ study. The firm expects to publish safety and initial efficacy data from all three stage 1 dose cohorts in the first half of 2024 and start the dose-selection and pivotal randomized-controlled stage 2 portion of the study in the second half of the year.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared in Precision Medicine Online.