Jennifer Farmer, MS, CGC, Executive Director at FARA, commented, "We are excited to award a second research grant to STATegics. The data generated from their previous grant is encouraging and this additional funding builds on their excellent progress. FARA recognizes the importance of public-private partnership in getting compounds through discovery and pre-clinical development. FARA is grateful for STATegics' commitment to advancing new therapeutics for Friedreich's ataxia and we look forward to seeing these compounds advance through pre-clinical development to human studies.
“We have made significant progress in our program and are grateful for FARA’s continued support. Our recent results have illustrated the potential of STATegics’ compounds to protect neurons and organs from cellular damage and to increase frataxin protein levels both in vitro and in vivo. This new grant allows for more detailed characterization of the mechanisms associated with frataxin increase, which will support the preclinical advancement of STATegics’ lead compounds and may also identify new targets for future drug development programs,” stated Juha Punnonen, MD, PhD, Chief Executive Officer of STATegics.
About FRDA and small molecule erythropoietin receptor agonists
FRDA is an inherited neurodegenerative disease caused by a single gene defect resulting in reduced expression of the mitochondrial protein frataxin. The symptoms of FRDA typically start at the age of 5 to 15 and include progressive loss of strength and coordination, vision and hearing impairment, slurred speech, diabetes and enlargement of the heart with the potential of leading to a heart failure. Because reduced frataxin is the underlying cause of the disease, drug candidates that enhance frataxin expression are considered to have significant therapeutic potential.
STATegics’ erythropoietin receptor agonists are small molecules designed to activate the tissue-protective erythropoietin receptor. The lead compounds have demonstrated promising effects in protecting neurons from various insults and increase frataxin protein levels in both normal and frataxin-deficient cells, without the erythropoietic activity of recombinant erythropoietin. In addition to increasing frataxin levels, STATegics’ compounds offer several other potential advantages when compared to alternative approaches due to their small size, tissue-availability, cytoprotective activity and feasibility for oral dosing.
About The Friedreich’s Ataxia Research Alliance (FARA)
FARA is a national, public, 501(c)(3), non-profit, tax-exempt organization dedicated to curing FRDA through research. FARA grants and activities provide support for basic and translational research, pharmaceutical/
About STATegics, Inc.
STATegics, Inc. is a privately-held biopharmaceutical company in Menlo Park, CA, committed to the discovery and development of orally available small molecule modulators of cytokine receptors. The company’s lead programs are focused on small molecule agonists of erythropoietin and thrombopoietin receptors for the treatment of central nervous system diseases and thrombocytopenia, respectively. The lead indication for the erythropoietin receptor agonists is FRDA, an orphan disease with no treatments currently available. Preclinical studies performed by STATegics and its collaborators have indicated superior properties of the lead compounds when compared to alternative approaches. STATegics is also developing screening technologies for efficient and rapid identification of small molecules targeting allosteric sites of cytokine receptors. STATegics’
Contact: Juha Punnonen, CEO, STATegics, Inc.: juha.punnonen@