NEW YORK – AstraZeneca said this week that the Scottish Medicines Consortium accepted the PARP inhibitor Lynparza (olaparib) for use within National Health Service (NHS) Scotland as a treatment for patients with HER2-negative, locally advanced or metastatic breast cancer with germline BRCA1 or BRCA2 mutations after chemotherapy. The consortium based its decision on positive results from the Phase III OlympiAD trial in which patients on Lynparza had a 42 percent reduction in risk of disease progression or death compared to those on chemotherapy.
Arrowhead Pharmaceuticals this week said it has closed a licensing and collaboration agreement with Sarepta Therapeutics, which was announced in November. As part of the deal, Sarepta will obtain rights to multiple small interfering RNA (siRNA) therapeutics in clinical, preclinical, and discovery-stage development. Sarepta will pay Arrowhead $500 million upfront, $325 million through the purchase of Arrowhead common stock, and $250 million to be paid in annual installments of $50 million over five years, among potential other milestone payments and tiered royalties on commercial sales.
The US Food and Drug Administration has granted regenerative medicine advanced therapy (RMAT) designation to Immix Biopharma's NXC-201, an autologous CAR T-cell therapy it's developing for relapsed/refractory amyloid light-chain (AL) amyloidosis, the Los Angeles-based firm said this week. RMAT designation is for treatments that meet the FDA's definition of a cell or gene therapy that are intended to treat, modify, or cure a serious or life-threatening disease and that may address unmet needs in that disease setting. In the US, Immix currently is evaluating NXC-201 within the Phase Ib/II NEXICART-2 trial.
The US Food and Drug Administration has granted rare pediatric disease and orphan drug designations to EN-374, a drug that Ensoma is developing as a treatment for X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder caused by mutations in the CYBB gene, the Boston-based company said this week. Ensoma said it expects to submit an investigational new drug (IND) application for EN-374, which is its lead program, in the first half of this year. A sponsor with an approved drug with rare pediatric disease designation may qualify for a voucher that it can redeem to receive priority review for a different product, or transfer or sell the voucher to another sponsor. The FDA grants orphan designation to drugs for rare diseases, which provides sponsors tax credits for clinical trials, exemption from user fees, and seven years of market exclusivity for the drug after regulatory approval.
Baltimore-based Elixirgen Therapeutics this week said a gene therapy it is developing for telomere biology disorders, EXG-34217, has received regenerative medicine advanced therapy designation from the US Food and Drug Administration. Elixirgen is evaluating EXG-34217 in patients with these rare and inherited disorders, which are characterized by short telomeres, and resulting in bone marrow failure within an ongoing Phase I/II trial.
Ideaya Biosciences said this week it has entered into a clinical study collaboration and supply agreement with Gilead Sciences to evaluate its MAT2A inhibitor, IDE397, with Gilead's TROP2-directed antibody-drug conjugate Trodelvy (sacituzumab govitecan-hziy) in MTAP-deletion non-small cell lung cancer. The collaboration expands on an existing partnership from 2023 between Ideaya and Gilead to study IDE397 and Trodelvy in MTAP-deletion bladder cancer. Under the expanded agreement, the Phase I/II trial of the combination treatment in bladder cancer will expand to include a cohort of NSCLC patients.
London-headquartered skin microbiome testing company Sequential Skin and Singapore-based Amili have been awarded the UK-Singapore Collaborative R&D Grant to support a $1.8 million project to characterize the interplay between the skin and gut microbiomes. The overarching goal of the research, which is also supported by Innovate UK and Enterprise SG, is to develop better approaches to evaluating inflammatory skin disorders such as atopic dermatitis and psoriasis, the companies said this week. Sequential maintains a database of over 25,000 clinical skin microbiome samples and has offered commercial skin microbiome at-home tests since 2019. Amili has focused on developing and analyzing Asia's largest multiethnic gut microbiome database to map the relationships and mechanistic pathways between changes in the gut microbiome and health conditions.
In Brief This Week is a selection of news items that may be of interest to our readers but had not previously appeared on Precision Medicine Online.