AI Therapeutics Announces that a Common LAM-002 Mechanism in Cancer and Neurodegenerative Diseases Shows Antitumor Activity in the Clinic and Hope for ALS

Company Receives FDA Guidance for Pivotal Trial Design in Follicular Lymphoma to Support Accelerated and Full Approval

GUILFORD, Conn.--(BUSINESS WIRE)-- AI Therapeutics is a clinical-stage biopharmaceutical company that has created an artificial intelligence-driven drug development platform for matching drugs to new indications. The company has made significant recent progress with its four clinical assets and proprietary Guardian Angel™ algorithm.

LAM-002 is a first-in-class, PIKfyve kinase inhibitor that activates transcription factor EB (TFEB), the master regulator of lysosomal biogenesis.¹ TFEB activation clears toxic aggregates that drive neurodegenerative disorders.^2-4 Dr. Murat Gunel, Nixdorff-German Professor of Neurosurgery and Professor of Genetics and of Neuroscience at Yale University and a member of AI Therapeutics’ Scientific Advisory Board indicated, “A wealth of new data points to the accumulation of toxic proteins as a common mechanism in neurodegenerative conditions disorders including amyotrophic lateral sclerosis (ALS), Parkinson’s disease and Alzheimer’s disease. We look forward to bringing the most advanced PIKfyve inhibitor into clinical development for patients suffering from ALS. We are pleased that our Guardian Angel^TM platform made the connection of LAM-002 to ALS and that the finding was validated in our laboratories and with our collaborators, as well as in independent research published in Nature Medicine.⁵”

Through its action on the lysosome, LAM-002 also selectively kills tumor cells. AI Therapeutics has recently completed enrollment in a clinical trial with patients previously treated for follicular lymphoma. Efficacy has been observed with LAM-002 as a single agent and when combined with rituximab or atezolizumab. LAM-002 appears to be well-tolerated with patients on continuous treatment for well over a year. Dr. Sarah Rutherford, Assistant Professor at Weill Cornell Medicine noted “LAM-002 represents a novel targeted approach to treat patients with follicular lymphoma. Our patients have shown durable objective responses while on LAM-002 therapy without the adverse effects often associated with other drugs.”

AI Therapeutics is also pleased to announce that it had a Type C meeting with the U.S. Food and Drug Administration (FDA) to discuss the registrational program for LAM-002 in patients with previously treated follicular lymphoma. At this meeting, AI Therapeutics reached concurrence on the design of a pivotal trial that could support accelerated approval. The clinical data to support full approval in patients with previously treated follicular lymphoma was also discussed and a path forward was detailed.

LAM-002 has received Fast Track status and Orphan Drug Designation from the FDA for the therapy of follicular lymphoma. The safety and clinical data for LAM-002 are expected to be presented at the American Society of Clinical Oncology (ASCO) conference in 2020.

¹Gayle, S. et al., Blood. 2017 Mar 30;129(13):1768-1778.

²Polito, V. et al., EMBO Mol Med. 2014 Sep;6(9):1142-60.

³Xiao, Q. et al., J Neurosci. 2014 Jul 16;34(29):9607-20.

⁴Decressac, M. et al., Proc Natl Acad Sci U S A. 2013 May 7;110(19):E1817-26.

⁵Shi Y., et al. Nat Med. 2018 Mar;24(3):313-325.

About AI Therapeutics

AI Therapeutics has built a state-of-the-art deep learning platform, Guardian Angel^TM, that synthesizes public and proprietary data on drugs and diseases and finds new indications for our library of new chemical entities. Aided by Guardian Angel^TM we now have four drugs in the clinic, including drugs that shrink cancers, the most promising candidate to treat ALS (Lou Gehrig’s disease), and drugs with applications for longevity.

AI Therapeutics is a 4Catalyzer company, and benefits from the advantages of being part of an incubator with >300 scientists and engineers, and offices in Connecticut, New York, Silicon Valley, and Taiwan. The mission of 4Catalyzer is to save the lives of people we love and make healthcare more accessible.

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