We are proud to announce that on October 19, 2026, the first three patients with Congenital Erythropoietic Porphyria (CEP) enrolled in the ATL001-PII-CEP clinical trial received their first dose of ATL-001, our investigational oral drug. It is the first time ATL-001 has been given to people with this severe, ultra-rare disease, for which no drug is specifically approved.
The three patients started treatment after completing the initial natural history observation phase and will continue taking a daily dose of ATL-001 oral solution over the coming months. ATL001-PII-CEP is an open-label, prospective Phase 1/2 trial with an N-of-1 design. Approximately six patients are expected to take part, each treated for about 48 weeks. The study is being conducted in the United States by Dr. Robert Desnick at the Icahn School of Medicine at Mount Sinai, Dr. Angelika Erwin at Cleveland Clinic, and Dr. Hetanshi Naik at Stanford University School of Medicine.
We would like to thank the patients and their families for their trust and commitment, and the investigators and clinical teams for their dedication. We will continue to work with rigor, caution, and transparency.