2023年1月26日 · AKAVA’S lead compound, AKV9, is a protein aggregation inhibitor that was identified to be the first compound to improve the health of upper motor neurons that degenerate in motor neuron diseases, such as amyotrophic lateral sclerosis (ALS), primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), and ALS/frontotemporal lobar ...

2023年8月28日 · Akava Therapeutics will soon launch a Phase 1 clinical trial to test AKV9, its investigational therapy for amyotrophic lateral sclerosis (ALS), in healthy people. The trial follows the clearance of Akava’s investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA).

2022年6月10日 · AKAVA Therapeutics ’ experimental therapy AKV9, formerly NU-9, was superior to approved therapies for amyotrophic lateral sclerosis (ALS) at improving the health of lab-grown upper motor neurons from a mouse model of the disease, a study shows.

AKV-9 is the inhibitor for mutated Cu/Zn superoxide dismutase (SOD1), that inhibits SOD1-induced protein aggregation and exhibits protective efficacy against SOD1-induced cytotoxicity in PC-12 with an EC50 of 0.3 μM. AKV-9 ameliorates amyotrophic lateral sclerosis in mouse models and extends their lifespan [1].

2023年9月2日 · On July 3 rd, 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).

2022年5月25日 · 正在推进 NU-9 分子（在公司称为 AKV9）的研究工作，目前正在开展临床前动物安全性试验，包括剂量和毒性评估，以向 FDA 申报 IND。 AKV9 也是当前该公司进展最快的在研管线。 据 透露，如果动物研究进展顺利并且 FDA 批准， AKV9 有望在 2023 年初进入 I 期临床试验，试验将从健康志愿者开始。 根据 FDA 对临床 I 期结果的审查，该药物分子的临床 II 期结果可能于 2024 年初启动，在人体中测试其治疗 ALS 的有效性。 “发现一种新药并将其推向市场 …

AKV9 was one of the best of the optimized compounds and was administered orally to both SOD1 and TDP-43 mouse models of ALS that are well-characterized and that closely mimic human pathology, including progressive UMN loss. The SOD1 mouse model expresses the human G93A mutation and displays misfolded SOD1 toxicity.

Protein aggregation is a hallmark of all neurodegenerative diseases, including ALS and other upper motor neuron (UMN) diseases. AKV9 is the first compound reported to improve the health of diseased upper motor neurons. UMNs degenerate early in ALS; inhibition of protein aggregation caused by various gene mutations maintains the health of UMNs.

Dr. Ozdinler is an expert in upper motor neuron cellular biology. Her team has done extensive work on a small molecule, known as AKV9 (NU-9), which is showing great promise in the treatment of upper motor neuron diseases. The compound is thought to improve the structural stability of the cell through the endoplasmic reticulum.

2023年8月22日 · On July 3 rd, 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).