BBDF-101 Update

Dear Family Partners,

I hope you are enjoying the holidays. In lieu of a family research call before year-end, we wanted to touch base and provide an update on the clinical development of BBDF-101

As you know from our previous announcement ^[1] and family call, BBDF-101 has been cleared for clinical trial by the FDA. We are initiating Phase I/II now for patients age 17 and older to gather safety data. The trial has been submitted to www.clinicaltrials.gov. Interested patients (17 and older) are encouraged to reach out to Dr. Gary Clark, Principal Investigator at Texas Children’s Hospital.

In order to proceed with Phase III for patients under 17 we will need to provide the Phase I/II safety data. That data won’t be available until after all Phase l/ll patients are all enrolled and on the protocol for 18 weeks. We will also need to have a meeting with the FDA in the new year to discuss efficacy endpoints.

Data from the chronic tox study in dogs, as well as statistics from the Unified Batten Disease Rating Scale (UBDRS) are being prepared now to support our efficacy endpoints discussion with the FDA. Special thanks to the 38 families that participated in the Engage Health survey, Understanding the Functional Burden of CLN3 Through the Eyes of Patients and Families. This information is more valuable data for our meeting with the FDA. Because of your quick responses and willingness to help, we were able to accelerate our timeline and we will submit the meeting request to the FDA early in the new year. The agency will have 75 days to schedule the meeting. These are our next major milestones.

We will plan to schedule a quarterly family research call as soon as we have new information to share. In the meantime, I hope you have plenty of meaningful time with family and friends during the holidays. We count each of you in our blessings and wish you health and happiness in 2022.

All my best,
Mary Beth
President & CEO[/vc_column_text][/vc_column][/vc_row]