Japan’s Takeda Pharmaceutical has joined forces with American biotech company AcuraStem to embark on the development of a groundbreaking treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. AcuraStem recently announced its partnership with Takeda, revealing a licensing agreement to advance and commercialize targeted therapies utilizing PIKFYVE, including AS-202, an innovative antisense oligonucleotide (ASO) specifically designed for the treatment of amyotrophic lateral sclerosis.
AS-202 focuses on inhibiting PIKFYVE kinase levels, a novel therapeutic target for treating amyotrophic lateral sclerosis. Furthermore, it holds potential in treating other TDP-43 proteinopathies, such as frontotemporal dementia (FTD). AcuraStem’s treatment strategy centers on combatting neurodegeneration by eliminating toxic protein aggregates and safeguarding the functionality of healthy neurons.
This groundbreaking treatment mechanism was initially discovered by Dr. Justin Ichida, co-founder of AcuraStem, while working with patient-derived disease models at the University of Southern California. It then underwent further development utilizing AcuraStem’s iNeuroRx disease modeling platform. Impressive results from Acculastem’s researchers demonstrate that antisense oligonucleotide-mediated PIKFYVE inhibition can restore motor function, reduce neurodegeneration, and enhance survival rates in various in vivo models of amyotrophic lateral sclerosis and frontotemporal dementia.
Under the terms of the agreement, Takeda will secure an exclusive worldwide license to AcuraStem’s PIKFYVE program. AcuraStem stands to receive approximately $580 million in upfront and milestone payments, in addition to royalties based on net product sales. These figures are contingent upon the achievement of clinical, regulatory, and commercial milestones throughout the agreement’s duration.
The preclinical studies of AS-202 and the characterization of potential follow-on ASOs will be conducted by AcuraStem, with Takeda taking charge of all development activities encompassing clinical development, regulatory affairs, and global commercialization.
“AcuraStem’s primary objective is to swiftly provide promising treatments to those in need,” stated Sam Alworth, CEO of AcuraStem. “It brings me great satisfaction to fulfill this mission.”
Sarah Shayk, Head of Neuroscience Therapeutics at Takeda, expressed the company’s dedication to developing innovative treatments for life-altering neurological disorders such as ALS. While recent advancements have brought hope to some patients, there is still a significant unmet need. AS-202 addresses this need with a unique dual mechanism of action that tackles TDP-43 aggregation, a key pathological feature of ALS and other TDP-43 proteinopathies, while improving TDP-43 functionality.
With AcuraStem’s research as a foundation, the hope is to further develop this exciting science and bring it to patients in need.
Targeted treatment was secured by PIKFYVE…clinical development and commercialization plan
[의약뉴스] Japan’s Takeda Pharmaceutical teamed up with American biotech company AcuraStem to develop a treatment for amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease).
On the 25th (local time), Acurastem announced that it had signed a licensing agreement with Takeda to develop and commercialize PIKFYVE targeted therapies, including AS-202, an innovative antisense oligonucleotide (ASO) for the treatment of amyotrophic lateral sclerosis.
▲ Takeda plans to develop AS-202, an antisense oligonucleotide that inhibits PIKFYVE kinase levels, through a licensing agreement with Acurastem.
PIKFYVE is a novel therapeutic target for amyotrophic lateral sclerosis and may be relevant for the treatment of additional TDP-43 proteinopathies such as frontoamerobic dementia (FTD).
Acurastem’s treatment strategy focuses on addressing neurodegeneration by expelling toxic protein aggregates and protecting the function of healthy neurons.
This new treatment mechanism was first discovered by Acurastem co-founder Dr. Justin Ichida from the University of Southern California using patient-derived disease models and further developed it with Acurastem’s iNeuroRx disease modeling platform.
Acculastem researchers showed that PIKFYVE inhibition mediated by an antisense oligonucleotide can restore motor function, reduce neurodegeneration, and improve survival in multiple in vivo models of amyotrophic lateral sclerosis and frontotemporal dementia.
Under the terms of the agreement, Takeda will receive an exclusive worldwide license to Acurastem’s PIKFYVE program. Acurastem is eligible to receive a total of approximately $580 million in upfront and milestone payments, plus royalties based on net product sales, contingent upon the achievement of all clinical, regulatory and commercial milestones during the term of the agreement.
Acurastem will conduct preclinical studies of AS-202 and conduct specific activities to help characterize potential follow-on ASOs. Takeda will be responsible for all development activities, including clinical development, regulatory affairs, and global commercialization.
“Acurastem’s mission is to get promising treatments to the patients who need them as quickly as possible,” said Sam Alworth, CEO of Acurastem. our mission. “I’m happy to do that,” he said.
“Our goal is to develop innovative treatments for some of society’s most debilitating neurological diseases, including ALS,” said Sarah Shayk, Head of Neuroscience Therapeutics at Takeda. “Recent advances in treatments have brought new hope to some patients, but there remains a significant unmet need.”
“AS-202 addresses this unmet need through a unique dual mechanism of action that addresses TDP-43 aggregation, a pathological hallmark of ALS and other TDP-43 proteinopathies, by include some types of dementia, and improves the function of TDP-43. it has potential,” he said, adding, “Based on Acurastem’s research, I look forward to developing this exciting science and bringing it to patients.”
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