Sio Gene Therapies had a few big announcements last week, including a Fast Track Designation from the U.S. Food and Drug Administration and the reorganization of its R&D group.
Sio Gene Therapies had a few big announcements last week, including a Fast Track Designation from the U.S. Food and Drug Administration (FDA) and the reorganization of its R&D group.
FDA Grants Fast Track Designation to Investigational Gene Therapy
The clinical-stage company announced that the FDA granted Fast Track Designation to AXO-AAV-GM1, its adeno-associated viral vector 9-based gene therapy candidate for Type I and Type II GM1 gangliosidosis.
“Receiving Fast Track Designation is a critical step in our mission to develop the first potential treatment for all pediatric forms of this rare, terminal disease. This designation joins both the Orphan Drug Designation and Rare Pediatric Disease Designation assigned to AXO-AAV-GM1 by the FDA, which we believe further demonstrates the potential impact of this work on the patient community,” said Pavan Cheruvu, M.D., chief executive officer of Sio Gene Therapies.
The designation was granted with the goal of getting new drugs to patients faster for the medical condition, which currently has an unmet need.
Phase I/II Trial Interim Data Announcement
On top of the Fast Track Designation announcement, Sio also presented interim data from its ongoing Phase I/II trial of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis.
The interim data, which was presented at the European Society of Gene & Cell Therapy Virtual Congress, showed that the drug candidate was generally well-tolerated at both low and high doses, and there have been no serious adverse events attributed to gene therapy in patients.
“Our team continues to lead the industry in the development of a new, potentially disease-modifying therapeutic option for GM1 gangliosidosis. We are extremely proud of these data, representing our broadest dataset generated thus far, which support a dose response and a favorable safety and tolerability profile at both low and high doses,” said Gavin Corcoran, M.D., chief R&D officer of Sio Gene Therapies.
The company is expecting to share more data from Stage 1 of the study in the first half of 2022.
Reorganization of R&D Team
The same day as the previously mentioned news, the company also announced changes it is making to its R&D team. Effective November 12, Chief Research and Development Officer Gavin Corcoran, M.D., will leave the company. Sio CEO and President Pavan Cheruvu, M.D. will take over the position’s responsibilities upon its vacancy.
Corcoran leaving isn’t the only executive shift the company is undergoing. Sio is establishing two new leadership positions: chief medical officer and senior vice president of early development and scientific affairs.
“With the continued progress of our gene therapy programs in the clinic, including positive data announced earlier today in GM1 gangliosidosis, and plans to expand our pipeline by investing in next-generation genetic medicines, the addition of a CMO and SVP of Early Development and Scientific Affairs will strategically position us for the advancement and expansion of our pipeline,” Cheruvu said.
