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Aspa Therapeutics, a BridgeBio company, shares an update on the CANinform natural history study

October 16, 2023

Dear Members of the Canavan Community,

Aspa Therapeutics, a BridgeBio company and the sponsor of the CANinform natural history study, would like to extend our appreciation and gratitude for families’ participation in CANinform. We are pleased to provide the community with an update on the ongoing study and share how the information has contributed to our understanding of Canavan disease.

The CANinform natural history study began in November 2019. To date, the study has enrolled 59 children in 14 countries.

Group (age) at Enrollment

Number of Participants

0-18 months

18

18-36 months

10

36-60 months

7

More than 60 months

19

Deceased

5

The fundamental goal of the CANinform study is to gain insight into how Canavan disease first presents and how it evolves over time. This information is advancing Canavan disease knowledge among researchers and clinicians and is being applied productively to the CANaspire gene therapy trial. Natural history data from the CANinform study can help identify the most informative ways to assess the effects of treatment and to measure what those effects are. Using the CANinform study as a comparison for the CANaspire clinical trial allows all participants in the CANaspire trial to receive the investigational treatment. No child will receive a placebo.

We are in ongoing dialogue with the FDA about the use of a natural history comparator for CANaspire and are continuing to recruit new participants to CANinform. The more natural history data we can gather, the more informed we’ll be regarding how to assess the participants in CANaspire.

From current analyses of CANinform data, several important learnings have emerged:

  • Head control, sitting ability, reaching, grasping, and visual tracking – functions flagged by the Canavan community for years – are showing potential as meaningful measures of change in CANaspire.
  • N-acetylaspartic acid (NAA) levels in urine are helping to characterize natural differences in NAA levels in patients with Canavan disease. This approach may allow less burdensome measurement of treatment effects in CANaspire.
  • The range of ASPA gene mutations and the in-depth clinical characterization of participants in the natural history study support CANinform as an appropriate comparator to CANaspire, further reducing the potential need for a placebo.

The selfless contributions of families participating in CANinform are deepening the medical community’s understanding of Canavan disease and supporting encouraging progress in the development of future treatment options for children and their families.

With sincere gratitude,

Aspa Therapeutics

Aspa Therapeutics
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