Foresee Pharmaceuticals announced that the company will present the clinical design of the FP-045 Phase 1/2 clinical Fanconi anemia clinical study (FuschiA study) currently being initiated, at the Fanconi Anemia Research Fund (FARF) 2023 Scientific Symposium taking place in Vancouver, BC, Canada from September 28-October 1, 2023. The oral presentation entitled: "A Clinical Trial Evaluating the Safety & Biological Activity of FP-045, an ALDH2 Activator, in Fanconi Anemia Patients" details the clinical design of the FuschiA Phase 1/2 study that will be conducted in collaboration with clinical experts in the area of Fanconi anemia (FA) from Stanford University, Memorial Sloan Kettering Cancer Center and the University of Minnesota. Details of the oral presentation are as follows: Title: A Clinical Trial Evaluating the Safety & Biological Activity of FP-045, an ALDH2 Activator, in Fanconi Anemia Patients.

Date: Saturday, September 30, 2023. Time: 11:10 - 11:25 PST. Location: Sheraton Vancouver in Grand Ballroom AB.

FP-045 is a first-in-class, potent oral aldehyde dehydrogenase 2 (ALDH2) activator. A dominant-negative allele of ALDH2 in Fanconi anemia (FA) patients is associated with accelerated progression of bone marrow failure, suggesting ALDH2 activation might be a potential therapeutic approach in FA. Laboratory studies demonstrate that FP-045 decreases aldehyde-induced toxicity in lymphoblastoid cell lines from FA patients.

Preclinical animal experiments also demonstrated that sustained activation of ALDH2 can prevent Hematopoietic stem cell (HSC) loss in FA, resulting in improved HSC numbers and functions. To allow for clinical testing in FA patients, single ascending dose (SAD), multiple ascending doses (MAD) 7 days studies, and a 28 days dosing study of FP-045 have been conducted in healthy human subjects that showed safety and tolerability of FP-045 following oral administration. Dose-proportionality and time-stability of the pharmacokinetics of FP-045 in humans were also established.

Based on these favorable results from healthy subjects and further discussions with regulators, Foresee is initiating the FuschiA study, a first-in-patient Phase 1/2 clinical trial to test the safety and tolerability of FP-045 and to obtain data on preliminary biological activity in FA patients. ALDH2 (Aldehyde Dehydrogenase 2) is a mitochondrial matrix enzyme and key regulator of mitochondrial quality control systems/health and regulator of reactive aldehydes/carbonyls, oxidative stress, inflammation, and fibrosis. Activation of ALDH2 is a compelling therapeutic strategy for improving mitochondrial quality and regulatory mechanisms for treating the treatment of rare/orphan diseases and severe diseases of aging.

The key role of ALDH2 in disease is supported by strong genetic/GWAS evidence related to a dominant-negative ALDH22 polymorphism.