RARE Daily

Abbisko and Sperogenix Partner to Develop ABSK021 for ALS, Other Rare CNS Diseases in Greater China

July 6, 2021

Chinese pharmaceutical companies Sperogenix Therapeutics and Abbisko Therapeutics entered into an exclusive licensing agreement to develop and commercialize ABSK021 for rare neurological indications, with amyotrophic lateral sclerosis as the first indication.

Photo: Yan Zhiyu, co-founder, chairman and CEO of Sperogenix

Under the agreement, Sperogenix subsidiary Sperogenix Medtech will have the exclusive right to develop, manufacture and commercialize ABSK021 in mainland China, Hong Kong, and Macao, for non-oncology rare neurological diseases indications. Abbisko will receive an upfront payment and a series of milestone payments as well as royalties on annual net sales from Sperogenix and reserves the rights for all the other territories and indications. Sperogenix will be responsible for the development of ABSK021 in ALS, including preclinical studies, proof-of-concept clinical trials, pivotal clinical trials, and post-marketing studies, as well as the registration and commercialization of the product in the Sperogenix territory.

“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,” said Xu Yaochang, founder and CEO of Abbisko.

Amyotrophic lateral sclerosis (ALS) is a rare neurological disorder characterized by progressive limb weakness, and muscular atrophy that eventually leads to death due to nutrition depletion and respiratory failure. According to an analysis of the national medical insurance data by the ALS cooperative group of the Neurology Society of Chinese Medical Association, the annual incidence of ALS in China is 1.62 per 100,000, and the annual prevalence rate is 2.97 per 100,000. At present, there is no specific treatment for the disease, and the prognosis is poor. The median survival time is only 3 to 5 years, which imposes heavy burden on families and society physically, emotionally, and economically.

ABSK021 is an orally bioavailable, potent, and selective colony-stimulating factor 1 receptor (CSF-1R) antagonist. Phase 1b clinical trials on safety, tolerability, and pharmacokinetics in oncology patients are being initiated both in China and the United States. Preliminary clinical data has demonstrated that ABSK021 was well tolerated, with favorable pharmacokinetic properties, and effective modulation of the target. CSF-1R signal transduction is involved in the development and survival of microglia. The presence of a large number of activated microglia is one of the hallmarks of neurodegeneration in ALS patients. Preclinical studies have shown that inhibition of CSF-1R could prevent disease progression of ALS in a variety of animal models.

“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,” said Yan Zhiyu, co-founder, chairman and CEO of Sperogenix.  “The strong cooperation with Abbisko will accelerate the clinical development and commercialization process of ABSK021, further strengthen our product portfolio in this field, and establish Sperogenix’s leading position in the space of rare neurological diseases in China.

Author: Rare Daily Staff

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