Sperogenix Acquires Rights in China to Develop ABSK021

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by Marta Figueiredo, PhD |

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ABSK021 and ALS therapy/ALS News Today/pharma partnership announced


A subsidiary of Sperogenix Therapeutics has acquired exclusive rights to develop and commercialize ABSK021, an investigational oral therapy for amyotrophic lateral sclerosis (ALS) and other rare neurological diseases in mainland China, Hong Kong, and Macao.

Under the terms of the agreement between Sperogenix MedTech and Abbisko Therapeutics, the therapy’s developer, Abbisko will receive an upfront payment from Sperogenix and will be eligible for additional milestone and royalty payments. The company will retain rights to ABSK021 in all the other territories and for other indications.

Sperogenix will first focus its efforts on ALS, and will be responsible for all preclinical studies, proof-of-concept and pivotal clinical trials, and post-marketing studies, as well as for the potential registration and commercialization of ABSK021 as allowed.

“Rare neurological diseases such as ALS have a serious impact on patients’ survival time and quality of life, which places a heavy burden on patients’ families and society,” Yan Zhiyu, Sperogenix’s co-founder, chairman and CEO, said in a press release.

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“A series of innovative therapies are urgently needed to improve the status of this disease field,” Zhiyu added.

“Sperogenix has impressive clinical research, development and commercialization capabilities in the field of rare diseases, and we are confident that this cooperation will further accelerate the development process of ABSK021 and benefit patients with rare diseases in China,” added Xu Yaochang, Abbisko’s founder and CEO.

ABSK021 is an orally available small molecule that works by potently and selectively binding to and blocking the colony-stimulating factor 1 receptor (CSF-1R), which is involved in the development and survival of certain immune cells.

These include microglia, immune cells of the central nervous system (the brain and spinal cord) that are overly active in people with ALS, promoting further neuroinflammation and neurodegeneration.

Previous preclinical studies have shown that suppressing CSF-1R can prevent ALS progression in several animal models of the disease.

Abbisko is also developing ABSK021 as a potential therapy for certain types of cancer, and for preventing immune reactions against transplants.

The therapy’s safety, tolerability, and pharmacokinetics (movement into, through, and out of the body) are currently being evaluated in cancer patients in Phase 1b clinical trials in both the U.S. (NCT04192344) and China. Preliminary data showed that ABSK021 was well-tolerated, had a favorable pharmacokinetic profile, and effectively suppressed its target, CSF-1R.

“This cooperation between Abbisko and Sperogenix is encouraging and demonstrates the great potential of ABSK021, an innovative CSF-1R drug candidate in the field of neurological diseases,” Yaochang said.

According to an analysis by the ALS cooperative group of the Neurology Society of Chinese Medical Association, 1.62 new ALS cases per 100,000 people are identified in China every year, and ALS affects annually 2.97 per 100,000 people in the country.