Cash and cash equivalents totaled $30.4M as of June 30, 2025 vs. $79.4M at December 31, 2024.”The first half of 2025 has been marked by a period of important execution as we’ve strengthened our internal clinical operations and made significant progress in addressing priorities while aligning on future planning across our pipeline. We are positioned for forward momentum as we engage with the FDA in the third quarter regarding a potential NDA submission for govorestat for the treatment of CMT-SORD. Our commitment to the CMT-SORD community is highlighted by our newly launched sponsored sorbitol assay, which is available to healthcare providers for patients at no-cost, removing potential barriers to diagnosis and treatment,” said Les Funtleyder, interim CEO and CFO. “As we look to the remainder of the year, we are focused on regulatory alignment across our pipeline. Our unwavering priority continues to be transforming the lives of patients with rare diseases, specifically CMT-SORD, Classic Galactosemia and PMM2-CDG.”
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