Dr. Patrice Rioux reports

THIOGENESIS ANNOUNCES FDA CLEARANCE OF IND FOR NEPHROPATHIC CYSTINOSIS

The U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for TTI-0102 in nephropathic cystinosis. On Oct. 16, 2026, the FDA issued a "study may proceed" letter for IND 184780. The IND is now in effect and supports the investigator-initiated study announced on Feb. 2, 2026.

The study, TTI-CYST-001, will be conducted at Emory University School of Medicine and Children's Healthcare of Atlanta under Dr. Larry Greenbaum, whose site previously participated in the development of delayed-release cysteamine for cystinosis. It is a single-site, open-label pilot in approximately six adults who will switch from their standard cysteamine therapy to once-daily TTI-0102 for seven days. Safety and tolerability are the primary objectives and pharmacokinetics are secondary. White-blood-cell (WBC) cystine will be evaluated across 24 hours as an exploratory end point, using the established surrogate biomarker as the measure of cystine control.

In its letter, the FDA provided comments on the protocol, and the company will incorporate them into a revised protocol, including additional safety and pharmacology assessments, before submission to the FDA and the institutional review board. Assuming timely study initiation, the company anticipates top-line data in the first quarter of 2027. Subject to results, the data are intended to inform dose selection and longer-term safety evaluation ahead of a registrational trial, which the company plans to design in agreement with the FDA under the 505(b)(2) regulatory pathway.

"This clearance allows us to move TTI-0102 into patients with cystinosis for the first time," said Dr. Patrice Rioux, chief executive officer of Thiogenesis. "Patients live every day with the dosing frequency, pill burden and side effects of current therapy, and TTI-0102 was designed with that burden in mind. We appreciate the FDA's review and are grateful to Dr. Greenbaum and his team."

"The burden of frequent dosing remains a daily reality for patients with cystinosis and their families. We are pleased to work with Thiogenesis to evaluate the safety and pharmacokinetics of a once-daily powder and explore whether it can maintain cystine control over 24 hours," said Dr. Larry Greenbaum, professor of pediatrics at Emory University School of Medicine and principal investigator.

About nephropathic cystinosis

Nephropathic cystinosis is a rare, life-threatening genetic disorder in which the amino acid cystine accumulates inside lysosomes, leading to kidney failure in childhood and multiorgan damage. Cysteamine is the standard of care. There are just over 600 diagnosed patients in the United States and an estimated addressable market of $360-million.

About TTI-0102

TTI-0102 is a cysteamine-pantetheine mixed disulphide pro-drug, converted in the gastrointestinal tract into two molecules of cysteamine and one of pantothenic acid (vitamin B5). It is a water-soluble powder designed for once-daily oral dosing, with the potential to eliminate the need to swallow capsules. TTI-0102 has completed a phase 2 trial in MELAS (mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes), has an IND-cleared phase 2a trial in Leigh syndrome with initiation planned for the fourth quarter of 2026 and is in clinical development for cystinosis.

About Thiogenesis Therapeutics Corp.

Thiogenesis Therapeutics is a clinical-stage biopharmaceutical company with operations based in San Diego, Calif. The company is publicly traded on the TSX Venture Exchange and in the United States on the OTCQB. Thiogenesis is developing sulphur-containing pro-drugs that act as precursors to previously approved thiol-active compounds, with the potential to treat serious pediatric diseases with unmet medical needs.

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