Dr. Patrice Rioux reports

THIOGENESIS RECEIVES FDA RARE PEDIATRIC DISEASE DESIGNATION FOR TTI-0102 FOR THE TREATMENT OF LEIGH SYNDROME

The United States Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation to Thiogenesis Therapeutics Corp.'s lead candidate, TTI-0102, for the treatment of Leigh syndrome.

The FDA grants RPD designation to therapies intended to treat serious or life-threatening diseases that primarily affect children from birth through 18 years of age. The designation provides the company with the potential, upon approval of a future new drug application (NDA), to receive a priority review voucher (PRV), which may be redeemed to obtain priority review of another marketing application or sold to another sponsor. Historically, PRV's have represented valuable strategic assets for biotechnology companies.

"Receiving rare pediatric disease designation is an important regulatory milestone for our Leigh syndrome spectrum program and further expands Thiogenesis's regulatory portfolio," said Patrice Rioux, MD, PhD, chief executive officer of Thiogenesis. "The designation recognizes the significant unmet medical need in Leigh syndrome and reinforces our confidence in the potential of TTI-0102 to address that need.

"Since receiving FDA clearance of our IND, we have strengthened our phase 2 protocol by incorporating important pharmacokinetic learnings from our MELAS clinical program. We are now completing final study start-up activities and look forward to initiating the phase 2 trial and generating clinical data."

About Thiogenesis Therapeutics Corp.

Based in San Diego, Calif., Thiogenesis Therapeutics is a clinical-stage biotechnology company developing TTI-0102, a patented new chemical entity designed to deliver sustained cysteamine exposure through proprietary prodrug chemistry rather than conventional controlled-release formulation technology. This differentiated approach is intended to improve tolerability, convenience and therapeutic exposure relative to existing cysteamine therapies. The company's lead program is in nephropathic cystinosis, where TTI-0102 is advancing toward late-stage clinical development. Thiogenesis is also developing TTI-0102 for primary mitochondrial diseases, including Leigh syndrome spectrum, and is preparing to initiate a phase 2 clinical trial.

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