TÜBINGEN, GERMANY, January 29th, 2024 — Mireca Medicines GmbH, an ophthalmology company formed to develop treatments for inherited retinal diseases today announces it received a USD 989,000 Translational Research Acceleration Program Award from the renowned Foundation Fighting Blindness. The funds will permit Mireca to further advance the preclinical development of its lead product MM238. 

By becoming an awardee of this competitive funding, the Foundation recognizes Mireca’s leading contribution to unlocking the potential of targeting the cGMP pathway to treat inherited retinal diseases (IRD) like Retinitis Pigmentosa (RP), Leber Congenital Amaurosis, and Stargardt’s disease. A wide range of mutations in at least 30% of these patients trigger excess levels of cGMP, thus over-activating the enzyme protein kinase G (PKG) which further down the signaling cascade causes the loss of photoreceptors. Mireca has developed proprietary cGMP-analogs that inhibit PKG, thereby halting photoreceptor degeneration which preserves retinal structure and function.  

During the TRAP project Mireca will continue developing its proprietary lead program MM238, a cGMP-analog in a tailored PLGA-based formulation, which is protected by a strong intellectual property estate. The formulation has previously shown an intraocular retention and release profile that supports life-long patient compliance. Product development and manufacturing will be followed by scientific advice meetings, safety, toxicology and translational proof-of-concept studies, all aimed at an Investigational New Drug (IND) filing. In performing these efforts, Mireca may tap into the Foundation’s strong network across all research and development stages, especially by consulting Dr. Cheryl L. Rowe-Rendleman, a regulatory and clinical senior executive, who the Foundation Fighting Blindness has assigned as project mentor. 

“Our research and development journey started with understanding the photoreceptor cell death mechanism. It took us through developing a novel class of small molecules that can regulate this disease pathway in combination with drug delivery formulations that are suitable for chronic ocular treatment. We have demonstrated proof-of-concept in preclinical IRD-models, with a favorable safety profile and tolerability window, and upscaled the small molecule manufacturing to GMP-grade. Being awarded with funds, network and mentoring from the Foundation Fighting Blindness to push our treatment approach further in its preclinical development is a significant reward and confirmation of our efforts. I am honored to be leading this TRAP project as principal investigator,” stated Prof. Dr. Francois Paquet-Durand, Mireca’s Scientific Founder. 

“Mireca’s pioneering pan-mutation approach is convincing as it has the potential to treat a high percentage of patients suffering from inherited retinal degenerations, who have hardly any treatment options today,” said Chad Jackson, Ph.D., Senior Director at the Foundation Fighting Blindness. 

About the Foundation Fighting Blindness
Established in 1971 in the Unites States of America, the Foundation Fighting Blindness is the world’s leading private funding source for retinal degenerative disease research. The Foundation has raised more than $915 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the spectrum of blinding retinal diseases including: Retinitis Pigmentosa, macular degeneration, and Usher syndrome. Visit FightingBlindness.org for more information. 

About the Translational Research Acceleration Program
The Translational Research Acceleration Program (TRAP) awards will accelerate the movement of preclinical research toward an Investigational New Drug filing and into clinical trials to provide a robust and diverse pipeline of potential therapies to fight inherited retinal diseases (IRD) and dry age-related macular degeneration. 

About Inherited Retinal Diseases (IRD)
Grouped under the term “Inherited Retinal Diseases,” Retinitis Pigmentosa, Leber’s congenital amaurosis, and Stargardt’s disease often result in the degeneration and loss of the light-sensitive cells in the retina, the so-called photoreceptors. This leads to severe visual impairment and eventually blindness. IRD are caused by a genetic defect in a single gene, out of more than 280 possible genes. Patients with these conditions may develop symptoms in early childhood, including night blindness or the inability to see parts of the world in front of them. They can take abnormally long periods of time to adjust to changes in lighting, and some patients may even find light uncomfortable. Individuals affected by IRD will gradually lose their sight and, unfortunately, most forms of IRD remain untreatable to this day.