Sio Gene Therapeutics (NASDAQ: SIOX) Offers Updates About Prioritization Of The GM1 And GM2 Gangliosidosis Programs

Sio Gene Therapeutics (NASDAQ: SIOX) has offered an update disclosing the prioritization of its clinical-stage AAV gene therapies, AXO-AAC-GM2 and AXO-AAV-GM1, for GM2 and GM1 gangliosidosis.

Sio has a cash runway of up to 2H 2023

The portfolio prioritizing enhances the company’s expected cash runway through multiple critical clinical targets for the gene therapy initiatives into 2H 2023. As of December 31, 2021, the company had cash and equivalents of $82 million. In addition, the company expects to discontinue its licensing arrangement with Oxford Biomedica for AXO-Lenti-PD, a lentiviral gene therapy initiative for Parkinson’s disease, as a result of the prioritization.

With the departure of its CEO, Dr. Pavan Cheruvu, who is quitting the company to explore new possibilities, Sio has named David Nassif, the Chief Financial Officer, and Chief Legal, as its interim CEO and a Board of Directors member. With the help of a top executive search company, a Sio Board search committee will interview both external and internal candidates to choose a permanent new CEO.

Nassif commented, “I believe our programs in GM1 and GM2 have the ability to transform the care for patients and deserve this heightened focus. We will continue to actively consider a variety of initiatives and options to further improve our financial position in order to provide the necessary capital to continue to pursue our key priorities for the longer term.”

Outgoing CEO Dr. Cheruvu stated that serving as the company’s CEO in the past four years has been a massive experience. He said that he is proud of the efforts to turn Sio into a leader in central nervous system gene therapies.

Sio to present Stage 1 Phase 1/2 study results in 1H 2022

In the first half of 2021, the company plans to present data from Stage 1 of the Phase 1/2 trial that includes the first look at type 1 patients in the low dose group and long-term findings from the Type II patient group. The company is also planning to engage the FDA in reviewing Stage 1 data and discussing the next clinical development steps.