NEW YORK – Clarity Pharmaceuticals said this week that its Phase I/II radiopharmaceuticals trial has completed cohort 1 and advanced to cohort 2. Through the SECuRE trial, Clarity is evaluating its targeted copper theranostic 64Cu/67Cu SAR-bisPSMA for identifying and treating patients with prostate specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer. The version of the agent with the 64Cu radioisotope is used as a diagnostic agent to visualize PMSA-expression tumors, while the version with 67Cu is a therapeutic agent. The second cohort of the trial includes an escalated dose.
Regenxbio this week announced it has received Regenerative Medicine Advanced Therapy designation from the US Food and Drug Administration for RGX-121, an investigational gene therapy the firm is developing for mucopolysaccharidosis type II, a rare disorder also known as Hunter syndrome and caused by deficiency of the enzyme iduronate-2-sulfatase. RGX-121 is an adeno-associated virus (AAV) vector-based therapy that delivers a copy of the IDS gene. The firm plans to file a biologics license application for the gene therapy next year through the accelerated approval pathway.
The US Food and Drug Administration has granted Rocket Pharmaceuticals a Regenerative Medicine Advanced Therapy designation for its investigational gene therapy for pyruvate kinase deficiency, a rare monogenic red blood cell disorder, the biotechnology company said this week. The firm's RP-L301 is a lentiviral-based therapy that delivers a corrected copy of the PKD gene through a patient's own genetically modified CD34-positive hematopoietic stem cells. The FDA granted RMAT designation based on data from an ongoing Phase I clinical trial. Rocket Pharmaceuticals plans to launch a pivotal Phase II trial in Q4.
Eisai said it has submitted a marketing authorization application for Leqembi (lecanemab) to the UK Medicines and Healthcare products Regulatory Agency in Great Britain as a treatment for patients with early Alzheimer's disease and confirmed amyloid pathology. Leqembi, which was codeveloped by Eisai and Biogen, is a monoclonal antibody treatment that attacks beta-amyloid clusters in the brain. The therapy was granted accelerated approval in the US in January.
AstraZeneca this week announced Ultomiris (ravulizumab), a treatment to prevent relapses in patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder, has been approved by the Japanese Ministry of Health, Labour, and Welfare. Ultomiris is a long-acting C5 complement inhibitor that was developed by Alexion, a pharmaceutical company focused on rare diseases that AstraZeneca acquired in 2021. Ultomiris recently received approval in the EU to treat the same condition.
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.