NEW YORK – Obsidian Therapeutics this week said the US Food and Drug Administration has granted regenerative medicine advanced therapy (RMAT) designation to OBX-115, its investigational treatment for immune checkpoint therapy-resistant unresectable or metastatic melanoma. The autologous, tumor-infiltrating lymphocyte cell therapy is engineered with membrane-bound IL15 regulated by the FDA-approved small molecule ligand acetazolamide. The treatment in July received fast-track designation from the FDA, which allows a sponsor to meet with regulators more frequently for advice on developing agents, submit data on a rolling basis, and apply for accelerated approval or priority review. RMAT status is granted to regenerative medicine therapies that aim to treat, modify, reverse, or cure a serious or life-threatening disease, and the designation affords similar benefits to fast-track status.
Skyline Therapeutics this week announced that its investigational gene therapy for retinitis pigmentosa, SKG1108, received orphan drug designation from the US Food and Drug Administration. SKG1108 is an adeno-associated virus-based gene therapy administered directly to the retina that delivers single-stranded DNA encoding proteins that are light-activated. Skyline aims to demonstrate that SKG1108 can generate new photo-sensing cells and improve or restore visual function in retinitis pigmentosa patients, regardless of the specific gene or genetic mutation causing the inherited retinal disease.
Mainz Biomed said this week that it has expanded its collaboration with Liquid Biosciences, originally announced last November, to pancreatic cancer. The companies now plan to use Liquid Biosciences' AI analysis technology platform, called Emerge, to select biomarkers for Mainz's PancAlert test for the detection of pancreatic cancer. The companies recently completed the first phase of their original collaboration, around Mainz's colorectal cancer screening test. For the second phase, they plan to include microbiome biomarkers and to extend the AI algorithm, completing their analysis in Q4 of this year.
Novartis said this week that it plans to expand its in-house radioligand production capacity by opening a new facility in Carlsbad, California. The firm also said it broke ground on a new facility at its existing Indianapolis site to produce the radioisotopes used in the firm's radiopharmaceutical therapies, including Pluvicto (lutetium vipivotide tetraxetan) and Lutathera (lutetium Lu 177 dotatate). Following regulatory approvals, the isotopes that Novartis produced with the expanded manufacturing capacity will be used in Pluvicto, Lutathera, and investigational radioligand therapies in the firm's pipeline.
Synthekine this week said the US Food and Drug Administration has granted fast-track designation to SYNCAR-001 + STK-009, its investigational therapy comprising autologous CD19 CAR T cells and orthogonal IL-2. The Menlo Park, California-based firm is developing SYNCAR-001 + STK-009 as a treatment for patients with severe, refractory systemic lupus erythematosus (SLE) who aren’t undergoing lymphodepletion. The FDA recently cleared Synthekine’s investigational new drug application to begin a Phase I trial of this treatment in patients with non-renal SLE and lupus nephritis. The company is also testing the treatment in CD19-positive hematologic malignancies in a Phase I trial.
Vivet Therapeutics this week said the European Commission has designated VTX-806 an orphan drug for treating cerebrotendinous xanthomatosis (CTX). The rare neurodegenerative disease is due to CYP27A1 genetic mutations that impede the body’s ability to properly metabolize cholesterol and bile acids, causing them to build up in the brain, tendons, eyes, and arteries. This buildup then leads to the formation of nodules that damage the brain tissue. VTX-806 uses an adeno-associated viral vector to deliver a normal copy of CYP27A1, which Paris-based Vivet is hoping will stabilize or reverse symptoms in CTX patients.
Vivet earlier this year received from the French government €4.9 million, which it will use to research, manufacture, and identify response-related neurological biomarkers for VTX-806. Receiving orphan status from European regulators affords the company certain benefits, such as protocol and scientific assistance with the development program, reduced fees, and 10 years of market exclusivity should VTX-806 garner approval. Vivet CEO and Cofounder Jean-Philippe Combal said the company hopes to seek orphan drug status in the US as well and begin clinical development in the latter part of 2025.
The US Food and Drug Administration this week granted orphan drug designation to Abdera Therapeutics' radiopharmaceutical agent ABD-147 in patients with DLL3-expressing neuroendocrine cancer. The agent is designed to target DLL3-expressing cancer cells and deliver the radioactive isotope actinium-225. The firm takes an approach to developing its radiopharmaceuticals that involves using a corresponding imaging agent to select the right patients for treatment. 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.
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.