Agreement enables the development of therapeutics, including AcuraStem’s AS-202
Candidate, an antisense oligonucleotide that suppresses levels of the PIKFYVE kinase.
MONROVIA, Calif., Sept. 25, 2023 — AcuraStem, a patient-based biotechnology
company pioneering how treatments are developed for neurodegenerative diseases,
announced today that it has entered into a license agreement with Takeda to develop
and commercialize AcuraStem’s PIKFYVE targeted therapeutics including AS-202, an
innovative antisense oligonucleotide (ASO) for the treatment of Amyotrophic Lateral
Sclerosis (ALS).
PIKFYVE is a novel therapeutic target for ALS and may also be relevant for treating
additional TDP-43 proteinopathies such as Frontotemporal Dementia (FTD).
AcuraStem’s therapeutic strategy for PIKFYVE focuses on addressing
neurodegeneration by expelling toxic protein aggregates and protecting healthy
neuronal function.
This novel therapeutic mechanism was initially discovered at the lab of AcuraStem’s co-
founder, Justin Ichida, PhD, using patient-derived disease models, is exclusively
licensed to AcuraStem by the USC Stevens Center for Innovation, and was further
developed on AcuraStem’s iNeuroRx® disease modeling platform (Shi Y et al Nat Med
2018, Hung S-T et al Cell 2023). The AcuraStem researchers further demonstrated that
ASO-mediated suppression of PIKFYVE can restore motor function, reduce
neurodegeneration, and improve survival in multiple in vivo models of both ALS and
FTD.
Under the terms of the agreement, Takeda will receive an exclusive, worldwide license
to AcuraStem’s PIKFYVE program. AcuraStem will receive an upfront and milestone
payments totaling up to approximately $580 million if all future clinical, regulatory, and
commercial milestones are achieved during the term of the agreement plus tiered
royalties on potential net sales of any commercial products resulting from this license.
“Our mission at AcuraStem is to get our promising therapeutics to the patients who
need them as quickly as possible,” said Sam Alworth, MS, MBA, CEO of AcuraStem.
“We are delighted to have secured a partner in Takeda who has the world class clinical
development and commercial capabilities to deliver on our mission.”
AcuraStem will be responsible for certain activities to help advance AS-202 IND
enabling studies and characterizing potential backup ASOs. Takeda will be responsible
for all other development activities including clinical development, regulatory affairs, and
global commercialization.
“We aim to develop transformative treatments for some of society’s most debilitating
neurological diseases including ALS. Whilst recent treatment advances have brought
new hope to some patients, there remains a significant unmet need,” said Sarah
Sheikh, BM BCh, MSc, MRCP, Head, Neuroscience Therapeutic Area Unit at Takeda.
“We believe AS-202 has the potential to address this unmet need through its unique
dual mechanism of action, which addresses TDP-43 aggregation and improves TDP-43
function, the pathological hallmark of ALS and other TDP-43 proteinopathies including
certain forms of dementia. We look forward to building on AcuraStem’s work to progress
this exciting science into development and to patients.”
AcuraStem, co-founded by Alworth, Ichida, Paul August, PhD, and Qing Liu, PhD, in
2016, remains committed to continually improving its patient-based iNeuroRx®
technology platform, discovering ever more effective therapeutic approaches across
neurodegenerative indications, and rapidly advancing its therapeutic programs to
patients including its existing SYF2 and UNC13A programs for ALS and FTD.
“I’m very pleased that AcuraStem is partnering with Takeda around their PIKFYVE
program,” said Justin Ichida, PhD. “We originally identified PIKFYVE as a novel
therapeutic target for ALS using nerve cells derived from patient stem cells, and this
illustrates the power of using this modern approach. I am grateful for the funding and
help we received from philanthropic and government agencies that enabled us to better
understand the therapeutic potential of targeting PIKFYVE. AcuraStem has done an
outstanding job of generating best-in-class molecules to target PIKFYVE in humans,
and I am excited to see this move forward.”
Destum Partners acted as transaction advisor to AcuraStem, and Foley Hoag acted as
legal counsel to AcuraStem.
