AT-007 continues to be safe and well tolerated to date. If not, the study will continue in blinded format to 24 months, where clinical outcomes will be assessed again in a final statistical analysis. If the primary clinical outcome measure (10-meter-walk/run) reaches statistical significance at 12 months, the study will be completed and unblinded. The INSPIRE study will continue in blinded format to the 12-month interim clinical outcomes assessment. The Company is working with the FDA to determine the appropriate regulatory path forward, as well as data required for an NDA submission, with the shared goal of bringing a safe and effective treatment to patients with SORD Deficiency as expeditiously as possible. Taken together with the biology of the disease and strong supportive animal model data, the Company believes that compelling data exists demonstrating that sorbitol reduction is reasonably likely to predict effect on clinical outcomes over time. In the INSPIRE trial, a baseline cross-sectional analysis of the relationship between sorbitol level, age (or duration of disease) and clinical outcome measures demonstrated a statistically significant correlation between sorbitol level and key clinical outcome measures, including 10-meter-walk/run speed, 4-stair climb speed, and sit-to-stand test (p<0.05). placebo) in patients with SORD Deficiency.Īt baseline, the mean blood sorbitol level of SORD patients was approximately 29,000ng/ml, with a range of approximately 22,000ng/ml-38,000ng/ml. In a pre-specified interim analysis of the ongoing Phase 3 INSPIRE trial, AT-007 reduced sorbitol levels by a mean of approximately 52% (or approximately 16,000ng/ml) over 90 days of treatment (p<0.001 vs. AT-007 (govorestat) is a central nervous system penetrant Aldose Reductase Inhibitor, which blocks conversion of glucose to sorbitol, and has previously been shown to reduce sorbitol levels in an open-label pilot study in patients with SORD Deficiency. SORD Deficiency (also called SORD Neuropathy or CMT-SORD) is a debilitating hereditary axonal neuropathy caused by mutations in the Sorbitol Dehydrogenase gene, leading to an inability to metabolize the sugar sorbitol, and resulting in accumulation of high levels of toxic sorbitol, which causes motor neuron degeneration and loss of mobility and motility. The INSPIRE trial is a Phase 3 double-blind placebo-controlled registrational study evaluating the effect of once-daily oral AT-007 in approximately 50 patients age 16-55 with SORD Deficiency in the US and Europe. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced positive sorbitol reduction data from the ongoing global Phase 3 INSPIRE trial. 16, 2023 (GLOBE NEWSWIRE) - Applied Therapeutics, Inc. Company to host conference call to discuss results at 8:30am Eastern.AT-007 reduced sorbitol by a mean of 52%, or approximately 16,000 ng/ml, over a 90 day period, which was highly statistically significant vs.placebo in patients with SORD Deficiency on sorbitol reduction as well as clinical outcomes in approximately 50 patients age 16-55 in the US and Europe The ongoing Phase 3 INSPIRE trial is evaluating the effect of AT-007 vs.
0 Comments
Leave a Reply. |
AuthorWrite something about yourself. No need to be fancy, just an overview. ArchivesCategories |