Enemsa Pharma Secures Rights from UChicago to Develop Novel Therapeutic
Enemsa Pharma has been granted exclusive patent rights from the University of Chicago to use a family of small molecules for the treatment of demyelinating diseases.
Under the terms of the agreement, the France-based biotechnology company receives exclusive rights to the U.S. Patent No. 10,905,663 patent family, in exchange for equity, maintenance fees, development milestone payments, and royalties on future net sales.
The licensed intellectual property covers Enemsa Pharma’s lead multifunctional drug candidate – Icerguastat – for use in demyelinating diseases, including Charcot‑Marie‑Tooth (CMT) diseases, a group of rare inherited debilitating neuropathies for which no disease‑modifying therapies currently exist.
“We are excited that this molecule will be advanced through our license with Enemsa Pharma, accelerating its development for patients with demyelinating disorders and broader neuromuscular diseases who are in great need of treatment options,” said Kenneth Onishi, senior manager, business development and licensing at the University of Chicago’s Polsky Center for Entrepreneurship and Innovation, which manages the University’s IP portfolio.
Enemsa Pharma CEO Pierre Miniou called the license agreement an important milestone for the company, as it will enable them to advance icerguastat into clinical development across multiple CMT subtypes in the U.S.
An orally available multifunctional small molecule, icerguastat selectively prolongs the body’s natural protective integrated stress response (ISR). The goal is to delay or halt disease progression by preserving vulnerable cells.
“Icerguastat’s unique mechanism of action, based on the selective enhancement of the ISR, offers a differentiated disease-modifying approach designed to protect and preserve both myelin-forming Schwann cells and neurons,” said Minious. “We believe this strategy has the potential to address the underlying biology of several genetic forms of CMT and bring meaningful benefits to patients who currently have no approved disease-modifying treatment options.”
Preclinical studies with icerguastat have demonstrated its broad therapeutic potential across several CMT subtypes. It also has demonstrated a favorable safety and tolerability profile in a Phase 1 clinical trial in healthy volunteers and been evaluated in an exploratory Phase 2 clinical trial in patients with bulbar‑onset amyotrophic lateral sclerosis (ALS).
Recognizing its potential to fill a critical unmet need, Icerguastat has received Orphan Drug Designation from the US Food and Drug Administration and the European Medicines Agency for all CMT subtypes.
// Technologies at the University of Chicago are available for licensing across several industries, from therapeutics, diagnostics, and medical devices, to quantum sciences, advanced materials, and more.