Akava Therapeutics, Inc. Announces FDA Clearance of Investigational New Drug Application for the Treatment of Amyotrophic Lateral Sclerosis

2023-08-22

临床1期临床申请

WINNETKA, Ill.--(BUSINESS WIRE)-- Akava Therapeutics, Inc., a pioneering biopharmaceutical company focused on developing first-in-class small molecule therapeutics that act through inhibition of key pathways in neural crest-derived targets for a variety of neurodegenerative diseases and cancers, celebrates a significant achievement. On July 3rd, the U.S. Food and Drug Administration cleared Akava’s Investigational New Drug (IND) application for the treatment of amyotrophic lateral sclerosis (ALS) to proceed with a Phase I first-in-human study in healthy subjects using Akava’s investigational new drug, AKV9 (formerly NU-9). The study will evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of AKV9. This pivotal milestone reinforces the company’s commitment to advancing the treatment landscape for ALS and other related neurodegenerative disorders. Dr. Richard Silverman, founder of Akava Therapeutics, expressed his enthusiasm, stating, "The FDA's IND approval of AKV9 is a testament to our team's dedication and the remarkable potential of our approach. We are thrilled to take this important step forward in the development of a groundbreaking therapy for ALS patients. Our goal is to make a meaningful difference in the lives of those affected by neurodegenerative diseases, and we are excited about the potential impact of AKV9 in addressing this devastating disease." ALS is a rare neurodegenerative disease characterized by selective premature degeneration and death of upper (cortical) and lower (spinal) motor neurons, leading to atrophy of muscles controlling voluntary actions, such as walking and breathing. The disease leads to progressive paralysis of muscles and loss of control of vital functions, typically resulting in death in 2-5 years (average) from diagnosis, although 20% of patients live more than 5 years and 10% more than 10 years. ALS is a highly heterogeneous disease. The sporadic form of ALS occurs without a known family history and represents about 90% of the cases. The familial form comprises about 10% of the cases. One common feature found in both forms of ALS is abnormal protein aggregation. There continues to be a critical unmet medical need for treatments that slow the progression of this fatal motor neuron disease. In preclinical models, unlike other drugs, AKV9 was found to act on multiple cellular degeneration pathways that are critical to the progression of the disease, by inhibiting protein aggregation, restoring cellular integrity, and improving the health of the mitochondria, endoplasmic reticulum, apical dendrites, and axons. About Akava Therapeutics, Inc. Akava Therapeutics ( ) was established in 2019 by Dr. Silverman as a platform to develop the deep pipeline of preclinical drug candidates discovered in the Silverman lab at Northwestern University. For 47 years, Dr. Silverman has been responsible for groundbreaking research, including the invention of the blockbuster drug Lyrica®, marketed by Pfizer for fibromyalgia, neuropathic pain, spinal cord injury pain, and epilepsy. He is also the inventor of CPP-115, which successfully completed a Phase I clinical trial. Under a compassionate use IND, this drug has been taken for the last eight years by a child with infantile spasms, who was refractory to all other medications. Silverman also is the inventor of OV329, which has been licensed to Ovid Therapeutics for epilepsy and is currently in clinical trials.

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