NEW YORK – Tokyo-based Eisai and Cambridge, Massachusetts-based Biogen this week said the Ministry of Health and Prevention in the United Arab Emirates has approved their beta-amyloid-targeting drug Leqembi (lecanemab) as a treatment for patients with early Alzheimer's disease. Eisai leads development and regulatory submissions globally for Leqembi, with both companies co-commercializing and co-promoting the product. However, Biogen will commercialize Leqembi in the UAE.
Leqembi is also approved in the US, Japan, China, South Korea, Hong Kong, and Israel, and is being marketed in the US, Japan, and China.
The US Food and Drug Administration has granted rare pediatric disease designation to ATSN-201, a gene therapy candidate that Atsena Therapeutics is developing as a treatment for X-linked retinoschisis, an inherited disease that causes retinal degeneration and vision loss, the company said this week. The Durham, North Carolina-based company is currently studying the safety and tolerability of the gene therapy in the open-label, dose-escalation Phase I/II LIGHTHOUSE trial.
Emeryville, California-based Kyverna Therapeutics this week said it has received regenerative medicine advanced therapy designation from the US Food and Drug Administration for its autologous anti-CD19 CAR T-cell therapy KYV-101 as a treatment for progressive myasthenia gravis, after the FDA reviewed clinical outcomes of patients treated with KYV-101 in Germany under a named-patient treatment option. In addition to progressive myasthenia gravis, Kyverna is evaluating KYV-101 in multiple autoimmune conditions, including multiple sclerosis, systemic sclerosis, and lupus nephritis.
The FDA this week granted fast-track designation to Accutar Biotechnology's investigational agent AC699 for estrogen receptor-positive, HER2-negative, ESR1-mutated advanced breast cancer. Accutar is evaluating the estrogen receptor α degrader in a Phase I trial. With fast-track designation, a sponsor can meet with the FDA more frequently to get advice on developing fast-track designated agents, submit data on a rolling basis, and apply for accelerated approval or priority review.
Watertown, Massachusetts-based Abata Therapeutics this week said it has received an equity investment of an undisclosed amount from Bristol Myers Squibb. The funding will support Abata as it advances a pipeline of regulatory T-cell therapy candidates it's developing for autoimmune and inflammatory diseases, including plans to launch a Phase I trial of the company's lead product candidate ABA-101 in progressive multiple sclerosis. Abata's second development program in type 1 diabetes, ABA-201, is in investigational new drug (IND)-enabling studies.
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.