The drug can now be tested for safety, tolerability, and efficacy in a phase 1 study.
The FDA has cleared Akava Therapeutics’ Investigational New Drug (IND) application for AKV9 to treat patients with amyotrophic lateral sclerosis (ALS), allowing the company to proceed with a phase 1 study in healthy subjects to evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of the drug, according to a statement released by the company.
AKV9 was observed in preclinical research to act on multiple cellular degeneration pathways that are critical to the progression of ALS. This is done by inhibiting protein aggregation, restoring cellular integrity, and improving the health of the mitochondria, endoplasmic reticulum, apical dendrites, and axons.
ALS is a rare, neurogenerative disease that is characterized by selective premature degeneration and death of upper and lower motor neurons, which leads to atrophy of muscles controlling voluntary actions such as walking and breathing. It leads to progressive paralysis of muscles, as well as a loss of control of vital function. It typically results in death 2 to 5 years after diagnosis.
The disease is highly heterogeneous; the sporadic form of ALS occurs without a known family history and represents about 90% of all cases, whereas the familial form comprises around 10% of the cases. A common feature found in both forms is abnormal protein aggregation.
"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,” said Dr. Richard Silverman, founder of Akava Therapeutics. “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."
BusinessWire. Akava Therapeutics, Inc. announces FDA clearance of investigational new drug application for the treatment of amyotrophic lateral sclerosis. News release. Accessed August 23, 2023. Published on August 22, 2023. Accessible at https://www.businesswire.com/news/home/20230816654411/en.