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FARA Announces Catabasis Pharmaceuticals as the Recipient of the Kyle Bryant Translational Research Award to Evaluate CAT-4001 as a Potential Therapy for Friedreich’s Ataxia

Downingtown, PA and Cambridge, MA (January 19, 2016) - The Friedreich’s Ataxia Research Alliance (FARA) and Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB) announce that Catabasis Pharmaceuticals is the recipient of the Kyle Bryant Translational Research Award. Catabasis is a clinical-stage drug development company built on a pathway pharmacology technology platform. The two year award will be for the Evaluation of CAT-4001 in Frataxin-deficient mouse models and dorsal root ganglia neurons to enable its therapeutic development for Friedreich's ataxia. This work will be led by Dr. Andrew Nichols at Catabasis along with collaborators Dr. Mark Payne at Indiana University and Dr. Jordi Magrane at Weill Cornell College of Medicine who are expected to perform testing in the Friedreich’s ataxia (FA) animal models.

Catabasis is developing CAT-4001 as a potential treatment for neurodegenerative diseases such as FA. CAT-4001 is designed to activate Nrf2 and inhibit NF-kB, both of which are implicated in FA pathophysiology. Catabasis has shown that CAT-4001 modulates the Nrf2 and NF-kB pathways in both cellular assays and animal models.

“We are excited to support this research as it brings a potential new candidate to the FA treatment pipeline and the research development team advancing the work is an example of FARA’s private public partnership strategy. We believe that we make the most meaningful research advancements when FARA, our academic investigators and our pharmaceutical partners take a collaborative approach to answering research questions,” said FARA Executive Director, Jennifer Farmer.

The Kyle Bryant Translational Research Award specifically focuses on pre-clinical and clinical investigations that target treatments for FA. The award is named for a young man diagnosed in his early teens with FA, Kyle Bryant, who started a cycling program called rideATAXIA with his family in 2007. The named award not only honors Kyle’s initiative to do his part to advance FA research but also recognizes all FA families involved in fundraising for research.

“We greatly appreciate the support of the Friedreich’s Ataxia Research Alliance as we advance the preclinical development of CAT-4001 as a candidate to treat FA,” said Andrew Nichols, PhD, Catabasis Senior Vice President, Research and Non-Clinical Development. “We are sensitive to the unmet medical need for patients affected by Friedreich’s ataxia and believe partnerships such as this could make a meaningful difference in discovering and developing therapies for them.”

About FA
Friedreich’s ataxia is a rare, degenerative, life-shortening neuro-muscular disorder that affects children and adults, and involves the loss of strength and coordination usually leading to wheelchair use; diminished vision, hearing and speech; scoliosis (curvature of the spine); increased risk of diabetes; and a life-threatening heart condition. There are no FDA-approved treatments.

About FARA
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3), non-profit, charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich's ataxia.

About CAT-4001
Catabasis is developing CAT-4001 as a potential treatment for neurodegenerative diseases such as Friedreich’s ataxia (FA) and amyotrophic lateral sclerosis (ALS). CAT-4001 is a small molecule that activates Nrf2 and inhibits NF-kB, two pathways that have been implicated in FA and ALS. Catabasis has shown that CAT-4001 modulates the Nrf2 and NF-kB pathways in both cellular assays and animal models.

About Catabasis
Catabasis Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics using its proprietary Safely Metabolized And Rationally Targeted, or SMART, linker technology platform. The Company's SMART linker technology platform is based on the concept of treating diseases by simultaneously modulating multiple targets in one or more related disease pathways. The Company engineers bi-functional product candidates that are conjugates of two molecules, or bioactives, each with known pharmacological activity, joined by one of its proprietary SMART linkers. The SMART linker conjugates are designed for enhanced efficacy and improved safety and tolerability. The Company’s focus is on treatments for rare diseases. The Company is also developing other product candidates for the treatment of serious lipid disorders. For more information on the Company's technology and pipeline of drug candidates, please visit

Felicia DeRosa Program Director, Friedreich's Ataxia Research Alliance

Andrea Matthews
Catabasis Pharmaceuticals, Inc.

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