Clinical development of ILX002 for SCD now fueled by new investment
Zynext invests in Illexcor therapy targeting root cause of sickle cell disease
Zynext Ventures is investing in Illexcor Therapeutics to advance the clinical development of ILX002, its lead, novel oral candidate therapy for sickle cell disease (SCD), the companies announced.
The financial details of the investment by Zynext Ventures, the venture capital arm of Zydus Lifesciences, were not disclosed.
“This investment is consistent with Zynext Ventures’ objective of identifying and fostering transformative early-stage healthcare innovations,” Jay Kothari, director of Zynext Ventures, said in a Zydus press release. “Beyond the financial backing, Zynext Ventures will leverage its unique data analytics and strategic expertise to accelerate Illexcor’s path to delivering this potentially life-changing therapy globally.”
Now in preclinical development, ILX002 is designed to directly tackle the root cause of SCD by preventing faulty hemoglobin proteins from sticking together. This occurs due to a process known as hemoglobin polymerization, which leads to the formation of rigid, sickle-shaped red blood cells that clog blood vessels, cause painful crises, and damage organs in SCD.
Andrew Fleischman, CEO of Illexcor, said the company is “excited to partner with Zynext Ventures to advance our lead drug ILX002 into clinical trials later this year.” No details on any potential trials were provided.
“We are hopeful that ILX002 will be a transformative treatment not only for SCD patients in the U.S. but also for millions around the globe. Zynext Ventures and Zydus Lifesciences are in a strategic position to help us achieve these goals,” Fleischman said.
Development of ILX002 expected to soon move into clinical trials
SCD is caused by mutations in the HBB gene. Those mutations lead to the production of a defective version of hemoglobin — the protein that carries oxygen in red blood cells — called hemoglobin S. The faulty proteins tend to clump together, causing red blood cells to take on a sickle-like shape. Such misshaped cells break down prematurely, resulting in chronic anemia, pain, inflammation, and severe complications affecting organs throughout the body.
While a bone marrow transplant and gene therapies can functionally cure SCD, such treatments are costly, can be risky, and generally are reserved for patients with the most severe forms of the disease.
An oral medication that can turn SCD into a manageable chronic illness could be a game changer, but currently available therapies have thus far provided only modest improvements.
ILX002, discovered in partnership with scientists from Virginia Commonwealth University, Children’s Hospital of Philadelphia, and King Abdulaziz University in Saudi Arabia, aims to change that.
The therapy works by directly binding to hemoglobin S and preventing hemoglobin polymerization.
According to Illexcor, ILX002 is the most potent hemoglobin S polymerization inhibitor discovered to date, and can effectively prevent red blood cell sickling for more than two hours, even in oxygen-deprived conditions, as shown in preclinical studies.
In a mouse model of human SCD, ILX002 restored normal hemoglobin levels, and reduced the number of immature red blood cells, or reticulocytes, from 50% to less than 20%. The levels of bilirubin, a byproduct of red blood cell breakdown, were reduced by 60%, while neutrophils, immune cells that rapidly respond to inflammatory cues, had their levels cut by 85%.
Recognizing the profound impact of sickle cell disease on patients’ lives, we are pleased to support Illexcor in [its] efforts to develop a novel therapeutic solution addressing this critical unmet medical need.
The therapy has shown an excellent safety profile thus far in preclinical studies, even at doses three times higher than the expected therapeutic level, according to the developer. Illexcor is now completing toxicology and safety tests, in preparation for ILX002’s potential entry into human clinical trials.
If the preclinical findings are reproduced in human clinical trials, ILX002 could become the first oral therapy to deliver benefits comparable to or better than those provided by gene therapies.
Sharvil Patel, MD, managing director of Zydus Lifesciences, said the company’s investment “reflects our commitment to patients battling rare and orphan diseases” such as SCD.
“Recognizing the profound impact of sickle cell disease on patients’ lives, we are pleased to support Illexcor in [its] efforts to develop a novel therapeutic solution addressing this critical unmet medical need,” Patel said.
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