Fort Collins, CO, September 17, 2019

Cetya Therapeutics, Inc. announces the award of a $1,000,000 Phase II STTR grant from the National Heart, Lung and Blood Institute of NIH to support the in vivo assessment of its histone deacetylase (HDAC) inhibitor CT-101 for up-regulation of the fetal hemoglobin gene as a potential treatment for sickle cell disease (SCD). This is a two-year grant with a start date of September 4, 2019 and an end date of August 31, 2021 as stated in the Notice of Grant Award from NIH.

Research has shown that small increases in the level of fetal hemoglobin can mitigate the severity of symptoms associated with sickle cell disease. This award follows the successful completion of a $240,000 Phase I STTR grant in collaboration with Dr. Susan P. Perrine, formerly of the Boston University School of Medicine.

Sickle cell disease and related conditions including thalassemia, are among the most common inherited genetic disorders globally, affecting millions of people. Seven percent of the world’s population carries an abnormal hemoglobin gene and annually 400,000 newborns are afflicted with a severe hemoglobinopathy. Sickle cell disease affects 100,000 individuals in the US, who incur $3B in annual medical costs. Fetal hemoglobin is down-regulated shortly after birth, but if present could compensate for deficient or defective adult hemoglobin. It is thus a natural remedy – but requires activation. HDACs are a family of epigenetic enzymes that are important in regulating the expression of many genes, and our data suggests that the fetal hemoglobin gene can be activated by Cetya’s HDAC inhibitor, CT-101 without significant toxicity.

“I am excited by the potential of Cetya’s CT-101 based on initial data obtained in a transgenic mouse model carrying the human hemoglobin locus where fetal hemoglobin was activated. I am eager to expand on our initial results to the preclinical sickle cell disease transgenic mouse model to be examined under this Phase II STTR grant”, stated Dr. Betty S. Pace of the Medical College of Georgia at Augusta University and co-Principal Investigator for the grant along with Dr. Robert M. Williams of Colorado State University and Dr. Louis H. Junker of Cetya Therapeutics.

Cetya’s proprietary compounds represent an exciting opportunity to develop these potent HDAC inhibitors that hold significant promise to mitigate dose-limiting toxicities and serious adverse events that have plagued HDAC inhibitor drug development. Cliff Hendrick, CEO of Cetya stated: “The Phase II STTR grant with Dr. Pace and Dr. Williams is a tremendous opportunity for Cetya to showcase CT-101, its lead candidate HDAC inhibitor for treating hemoglobinopathies such as sickle cell disease, and gain supporting evidence that these analogs could be useful outside of the currently approved HDAC inhibitor indications within oncology.”

About Medical College of Georgia/Augusta University
The Medical College of Georgia is the state’s public medical school established in 1828 and based in Augusta, Georgia. The Medical College of Georgia is Georgia’s only public medical school. It was one of the nation’s first medical schools and continues to make an impact today by optimizing health care in Georgia and beyond through education, discovery and service.

About Cetya Therapeutics
Cetya Therapeutics, Inc. is a development stage pharmaceutical company developing histone deacetylase inhibitors structurally based on the marine natural product Largazole. Cetya has the exclusive worldwide rights to the histone deacetylase (HDAC) inhibitor platform and patent estate developed in the laboratory of Dr. Williams, University Distinguished Professor of Chemistry at Colorado State University, with co-inventors Dr. James E. Bradner, formerly of the Dana-Farber Cancer Research Institute and Harvard Medical School and Prof. Olaf G. Wiest of the University of Notre Dame.

Cetya has a portfolio of analogs to draw upon for selection of a lead candidate for each of the therapeutic indications under study, including treatment of sickle cell disease. Initial data confirms up-regulation of fetal hemoglobin expression in the absence of significant inhibition of the growth and maturation of erythroid precursor cells.

Cetya is seeking corporate partners to assist in the advancement of its analog portfolio in a number of different therapeutic indications. For more information on Cetya, please visit our website at www.cetyatherapeutics.com or contact Cliff Hendrick at chendrick@cetya-therapeutics.com