Participant screening begins at first clinical trial site in the United States

Dear Canavan Community Members,

Today we are excited to announce the initiation of our first clinical trial for BBP-812, Aspa’s investigational gene therapy for Canavan disease.

The Phase 1/2 clinical trial, called CANaspire, will evaluate the safety and efficacy of a one-time, intravenous (IV) dose of BBP-812 in children with Canavan disease.

Participant screening will begin immediately at our first clinical trial site in the United States, Massachusetts General Hospital in Boston, Massachusetts, and at first will be limited to US residents. We are working as fast as we can to enable recruitment from outside the United States and are also working to add additional clinical sites in the US and abroad.

This milestone is a result of years of groundbreaking research and development by Dr. Guangping Gao at the University of Massachusetts Medical School, who has made the fight against Canavan disease a central focus of his scientific career. Aspa’s program has included extensive collaboration with Dr. Gao and his team including Dr. Dominic Gessler, along with other leading experts in gene therapy and Canavan disease.  The combined efforts contributed to the completion of robust preclinical safety and efficiacy studies in animals, including mice and monkeys. Most importantly, this milestone reflects the ongoing support and insights all of you in the Canavan community have generously shared with us through our collaboration with patient organizations and families.

We want to highlight a few important details about the study:

• BBP-812 will be administered as a one-time intravenous (IV) infusion. This is a non-invasive procedure.
• CANaspire will enroll children with a confirmed diagnosis with Canavan disease who are 30 months of age or younger on the expected date the investigational gene therapy would be given.
• We anticipate enrolling approximately 18 participants.
• We plan to begin screening participants who live outside the US as soon as possible, and will alert the community when this occurs.
• There is no cost to participants or families for participating in the study.
• Aspa will pay all travel costs to and from treatment sites for patients and their families.

We recognize that parents and caregivers of older children are also very interested in this treatment.  Once we are able to see data in some of our initial patients and have a chance to review that data, we will work together with regulators and study investigators to create opportunities for older Canavan patients to receive BBP-812 as well.

We understand the urgency for all patients, and are proud that BBP-812 has been granted Fast Track designation by the FDA.

If you are interested in learning more about CANaspire, please visit clinicaltrials.gov to find the CANaspire listing. There you will find additional details on the inclusion and exclusion criteria.  You can also contact the Canavan Disease Study Team at +1.833.764.2267 or +1.617.861.4617 or email CANaspire@aspatx.com.

Investigators at the clinical sites will be responsible for participant screening and selection. Members of the Aspa team are not involved in the selection of individual trial participants.

As with any investigational therapy, there are risks that your child’s physicians and the study investigators will discuss with you. The initiation of CANaspire marks an important step forward in our mission to develop a potential treatment that will improve the lives of as many children with Canavan disease as possible.

As we move forward in this journey, we will share relevant information and program updates whenever we are able.  Visit treatcanavan.com to stay connected and to learn about gene therapy.

On behalf of the Aspa Therapeutics Team,

Eric M David MD JD
CEO