New collaboration targets protein buildup linked to brain diseases
Deal advances small-molecule drugs designed to remove harmful proteins
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- Origami and Ipsen collaborate on therapies for neurodegenerative diseases such as ALS.
- They develop small molecules designed to remove or correct abnormal protein buildup.
- This approach targets toxic protein clumps thought to damage motor neurons in ALS.
Origami Therapeutics is collaborating with Ipsen to develop experimental therapies designed to remove or correct abnormal proteins that build up in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS).
The partnership will focus on small molecules known as protein degraders and correctors. These compounds are designed to help cells remove toxic, misfolded proteins or help those proteins return closer to their normal shape.
Because abnormal protein buildup is a key feature of ALS and many neurodegenerative diseases, this approach aims to target underlying disease biology.
Inside Origami’s protein-targeting drug approach
Origami is developing compounds designed to destroy or correct misfolded proteins linked to many neurological disorders. Under the terms of the agreement, Ipsen has an exclusive option to license the drug development program for a rare, inherited neurodegenerative disorder following successful drug candidate nomination. If Ipsen exercises that option, it would become responsible for global development and commercialization.
“This collaboration with Ipsen is a strong validation of Origami’s science and of the potential of targeted protein degradation to address the root causes of rare genetic neurodegenerative diseases,” Beth Hoffman, PhD, Origami’s founder and CEO, said in a company press release.
“Ipsen brings deep expertise in rare neuroscience and global development, making them an ideal partner as we advance this program toward the clinic. Together, we share a commitment to translating cutting-edge biology into meaningful therapies for patients who currently have limited or no treatment options,” Hoffman added.
A hallmark of many neurodegenerative diseases is the abnormal folding and aggregation, or clumping, of certain proteins inside nerve cells. In ALS, these toxic protein clumps are thought to damage motor neurons, the nerve cells that control movement.
Origami’s approach is designed to help remove these abnormal proteins or help them return closer to their normal shape. The company uses its ORICISION drug discovery platform to identify small molecules that target disease-causing proteins while preserving the function of the normal proteins.
How these experimental drugs are designed to work
Candidates are designed to leverage the body’s natural protein disposal systems and are tested in cell models of neurodegenerative diseases. The most promising compounds will be selected based in part on their ability to enter the brain and potentially limit the spread of disease-causing proteins to other neighboring cells.
The goal is to develop compounds that can be delivered orally, though other routes of administration may also be considered.
Under the collaboration, Origami is eligible to receive upfront payments as well as additional payments tied to development, regulatory, and commercial milestones. The company may also receive royalties if a product reaches the market.
“We are delighted to join forces with Origami whose pioneering work in developing protein degraders targeting genetic neurodegenerative diseases represents a powerful new frontier in rare neuroscience,” said Steve Glyman, Ipsen’s senior vice president and head of neuroscience research and development.
“Their approach aligns seamlessly with Ipsen’s strong rare neuroscience heritage and expertise in small‑molecule innovation, enabling us to advance first‑ and best‑in‑class therapies for people living with debilitating conditions where treatment options remain limited,” Glyman added.
