Atuzaginstat

The FDA has granted fast track designation to Quince Therapeutics’ Phase III drug candidate EryDex to treat rare disease ataxia-telangiectasia. Credit: grandbrothers via Shutterstock. California-based biotech Quince Therapeutics has won fast track designation from the US Food and Drug Administration (FDA) for its ataxia-telangiectasia (A-T) drug EryDex. Recommended Buyer's Guides Buyer's Guide Leading Guide to Packaging Materials, Containers and Containment Services for the Pharmaceutical Industry Buyer's Guide Leading Guide to Clinical Packaging Companies in contract Manufacturing for the Pharmaceutical Industry According to the 3 June announcement, fast track designation was awarded to address a high unmet medical need for patients with A-T. The status allows for earlier interactions with the FDA as Quince seeks accelerated approval, and also the possibility to undergo rolling reviews. A-T is a rare disease of the nervous and immune systems, affecting motor movement and speech. EryDex utilises autologous intracellular drug encapsulation (AIDE) technology to administer dexamethasone sodium phosphate (DSP) into a patient’s red blood cells. Red blood cells filled with DSP are then reinfused into the patient, allowing for the slow release of steroids over several weeks, without the long-term toxicity commonly associated with chronic administration. The FDA lifted a partial clinical hold that was on the drug in October 2023, advancing it into the Phase III NEAT study (NCT06193200). The FDA removed the partial clinical hold after requesting additional information on plastics used in the single-use EryKit, which is part of EryDex. Changes were made to comply with recent European regulations. The NEAT study will enrol approximately 86 six- to nine-year-old patients with A-T as the primary analysis population, as well as 20 patients over the age of ten years. Participants will be randomised between EryDex or placebo and treatment will involve six infusions once every 21 to 30 days. The primary efficacy endpoint will be measured by the change from baseline to last visit completion in the rescored modified International Cooperative Ataxia Rating Scale (RmICARS). See Also: FDA hearing set to discuss advisory committee members’ conflicts and more Be Bio’s haemophilia B treatment gains FDA orphan drug status Quince shared in an April 2024 business update that it anticipates topline results in H2 2025 with a potential NDA submission in 2026. Quince inherited EryDex in July 2023 when it acquired biotech EryDel in a $485m deal in October 2023. Formerly known as Cortexyme, Quince changed its name in 2022 and focused on rare diseases following several setbacks, including a clinical hold on its Alzheimer’s disease drug atuzaginstat. In the announcement accompanying the fast track, Quince CEO Dirk Thye said: “The granting of fast track status for EryDex System marks another important milestone in our endeavour to identify a beneficial therapeutic solution for patients with A-T. “We have initiated our pivotal Phase III NEAT clinical trial, which is being conducted in the US, UK, and the European Union.”

Phase 1 single ascending and multiple ascending dose studies previously completed demonstrate safety, tolerability and dosing to reach optimal target engagement SAN FRANCISCO, Nov. 16, 2023 /PRNewswire/ -- Lighthouse Pharmaceuticals, a clinical-stage biopharmaceutical company pioneering precision medicine for the treatment of Alzheimer's and other diseases of aging, today announced clearance by the U.S. Food and Drug Administration (FDA) of the Investigational New Drug (IND) application for LHP588, the company's next-generation gingipain inhibitor, for the treatment of P. gingivalis-positive Alzheimer's disease. The "Study May Proceed" letter from the FDA was based on review of the comprehensive IND package including safety, chronic toxicology, manufacturing, Phase 1 human data, and the planned Phase 2 SPRING (Stopping PRogression of P. gINGivalis-positive Alzheimer's disease) trial protocol. LHP588 is a next-generation, brain penetrant small-molecule gingipain inhibitor that demonstrated safety and tolerability in the completed Phase 1 study, along with plasma levels sufficient to engage the target with just once-daily oral dosing. The Phase 2 SPRING clinical trial is a randomized, double-blind, placebo-controlled study to assess the efficacy and safety of once-a-day dosing of LHP588 for the treatment of P. gingivalis-positive mild to moderate Alzheimer's disease. 300 patients will be enrolled in the trial and randomized into three arms (placebo, 25 and 50 mg) for 48 weeks of treatment. Patients must have mild to moderate Alzheimer's disease and evidence of P. gingivalis infection, based on a saliva test. The GAIN trial of the first-generation gingipain inhibitor, atuzaginstat, demonstrated that patients with mild to moderate AD and a positive P. gingivalis saliva test had a dose-dependent, 57% slowing of cognitive decline on the prespecified proof of efficacy endpoint, ADAS-Cog11, compared to the placebo group (p = 0.02). "We are very pleased to have FDA clearance to initiate the SPRING Phase 2 clinical trial. Previous studies of this mechanism indicated promise of the precision medicine approach being leveraged by Lighthouse going forward. The clinical efficacy and safety data provided by the GAIN trial has enabled us to significantly de-risk the mechanism of action, maximize target engagement with higher exposures of LHP588, and implement the optimal clinical study design," said Casey Lynch, CEO of Lighthouse Pharmaceuticals. "LHP588 is a next-generation gingipain inhibitor that has been optimized for selectivity and metabolism, demonstrating an excellent safety profile in both human and toxicology studies. With FDA clearance in hand, we look forward to working with our clinical advisors and investors to move this important clinical study forward." About Lighthouse Pharmaceuticals Lighthouse Pharma is a clinical stage private biopharmaceutical company pioneering precision medicine approaches to major unmet medical needs based on expertise in small molecule protease inhibition. Lighthouse's lead program, LHP588, a Phase 2 ready orally bioavailable small molecule, targets a specific, infectious pathogen, Porphyromonas gingivalis, which is associated with chronic degenerative and inflammatory disorders including dementia, periodontal disease, atherosclerosis, and orodigestive cancers. For more information, visit . SOURCE Lighthouse Pharmaceuticals