Otsuka, Eikonizo Collaborating on ALS, Rare Disease Therapies

The McQuade Center for Strategic Research and Development (MSRD) and Eikonizo Therapeutics have entered a collaboration to advance potential therapies for rare diseases, including amyotrophic lateral sclerosis (ALS).

“We are dedicated to seeking out and supporting novel treatments for complex and rare diseases,” Robert McQuade, PhD, president of MSRD and chief strategic officer at Otsuka Pharmaceutical, said in a press release.

MSRD, established in 2019 by the Otsuka Pharmaceutical business in the U.S., seeks to identify early-stage therapies with the potential to change the treatment paradigm in areas of unmet need.

“Eikonizo and MSRD share values of innovation, scientific rigor and urgency to address unmet medical needs and thus make a meaningful impact on patients’ lives,” said Janice Kranz, PhD, co-founder, president and CEO of Eikonizo.

Eikonizo Therapeutics is a biotechnology company focused on the development of small molecules that are able to enter the brain and reach specific therapeutic targets. The company’s lead program is directed at neurodegenerative diseases and involves inhibiting specific enzymes called histone deacetylases (HDACs).

HDACs are enzymes that remove certain chemical groups from proteins, and are important therapeutic targets in cancer, inflammatory disease, and pulmonary disease. Inhibiting the HDAC6 enzyme, in particular, is thought to rescue the transport of several molecules within nerve cells, and to lessen disease-associated symptoms in ALS.

Eikonizo is developing selective HDAC6 inhibitors for neurodegenerative diseases such as Alzheimer’s disease and ALS, while also creating a companion tracker for imaging exams that measures how much of HDAC6 is being targeted, which will help monitor the effectiveness of these inhibitors.

“Eikonizo is discovering and developing novel small molecule therapeutics and leveraging companion target engagement technology to de-risk and accelerate development,” added Kranz.

“We believe our approach and our brain-penetrant HDAC6 inhibitors could provide a transformative therapy for ALS,” she said.

Under the agreement, Eikonizo and MSRD will collaborate to advance these HDAC6 inhibitors through preclinical and Phase 1b clinical trials. The companies might opt to expand their collaboration after those studies are complete.

“Based on our pre-clinical evaluation, we are hopeful that Eikonizo’s compounds can open up new treatment options for patients living with ALS and other rare diseases of high unmet need,” said McQuade.

“We are delighted to collaborate with MSRD, honored that they recognize the potential of Eikonizo’s strategy and HDAC6-targeting platform, and excited to leverage MSRD’s experience and support to markedly accelerate development of our first therapeutic candidate into the clinic,” Kranz said.