Neurizon, Elanco sign deal to advance ALS therapy NUZ-001
Elanco to provide animal and manufacturing data, future supply of monepantel
Neurizon Therapeutics has entered into a global licensing deal with Elanco Animal Health to advance the development of NUZ-001, an investigational therapy for amyotrophic lateral sclerosis (ALS).
Monepantel, the active ingredient in NUZ-001, is an oral deworming agent commonly used in veterinary medicine, and Elanco, a company that produces medicines and vaccinations for pets and livestock, has collected extensive data from its use in animals.
Under the new agreement, Neurizon will have access to Elanco’s animal safety studies and manufacturing data, which may help meet some regulatory requirements to advance NUZ-001 into clinical studies and allow the company to avoid repeating early-stage research, thereby reducing development costs and timelines.
“This license agreement with Elanco marks a watershed moment in Neurizon’s journey,” Michael Thurn, PhD, Neurizon’s managing director and CEO, said in a company press release. “This strategic milestone provides exclusive access to a comprehensive package of non-clinical studies and manufacturing data, dramatically reducing near-term development costs and accelerating development timelines.”
Elanco to provide Neurizon with long-term supply of monepantel
The companies have also outlined a future supply agreement. Under it, Elanco would provide Neurizon with long-term supply of monepantel that’s compliant with good manufacturing practice guidelines, which help ensure consistent product quality and safety, to support clinical trials, regulatory submissions, and commercialization of NUZ-001.
ALS is a progressive disease that affects motor neurons, the nerve cells that control movement. A number of mechanisms is believed to contribute to the gradual damage and death of motor neurons, including the accumulation of toxic protein clumps.
Beyond its deworming use, monepantel has an off-target effect of inhibiting a signaling pathway known as mTOR, which helps regulate a process called autophagy, or the process by which cells break down unneeded or damaged components. By boosting this cleanup process, NUZ-001 may help remove the toxic protein clumps that are thought to drive ALS progression.
In a small Phase 1 trial called MEND (NCT04894240), 12 people with ALS were treated with NUZ-001 at a low or high dose. The treatment was well tolerated and slowed functional decline by 39%-58%, compared with a matched group of untreated patients from an external database. It also reduced lung function decline by 48%.
After completing the trial, participants continued to receive NUZ-001 via a compassionate use program, and later entered an open-label extension study (NCT06177431), where all received daily NUZ-001 for up to a year. After four months in the extension, patients continued to experience slower disease progression compared with the external control group, and their risk of death was was significantly reduced, by 80.3%.
NUZ-001 is also being added to the HEALEY ALS platform trial (NCT04297683), a multi-arm study that is testing multiple ALS candidates simultaneously against a shared placebo group. The goal is to accelerate the development of potential therapies, with new treatments added as they become available.
The recent licensing deal includes an undisclosed upfront payment to Elanco, and $9.75 million in milestone payments for the first approved product, plus $5.2 million for additional products or uses. Elanco could also receive up to $65 million in sales-based milestones, and will receive tiered single-digit royalties on global net sales.
“This agreement not only strengthens our clinical and regulatory position but also reflects our strong commitment to accelerating the development of meaningful therapies for people living with neurodegenerative diseases,” said Sergio Duchini, non-executive chairman of Neurizon. “We are also very happy to be making this important step in formalising our relationship with Elanco and look forward to building on this foundation as we progress towards finalising a supply agreement later this year.”
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