Important Announcement Regarding ABT-003 Clinical Trial

To Our MPS and ML Community,
 
We remain ever mindful that all patients deserve improved treatment and management of their disease, regardless of where an individual is on the MPS or ML path.
 
With this in mind we ask you all to come together in support of the MPS IIIA community as we share news that the Abeona study ABT-003 (Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease), closed enrollment and study site activities with a “Global End of Trial” date effective yesterday, March 10, 2022.
 
This trial involved the extension of the groundbreaking intravenous AAV9 gene therapy with Abeona’s drug designated ABO-102 to older patients with more advanced neurocognitive disease. This approach to treating MPS IIIA was pioneered in Abeona’s Transpher A Study (ABT-001). To be clear, based on our most recent communication, the Transpher A Study and its associated extension study are ongoing.
 
All of us here at the Society and in the MPS community are deeply concerned regarding this news. In their communication to us, Abeona detailed that this difficult course of action was based on a recent review of available neurocognitive and biomarker data, which they concluded did not demonstrate improvements in neurocognitive function. This was the basis of their decision. Interested and impacted members of the Sanfilippo A community are encouraged to communicate with Abeona at [email protected] for more information and guidance.
 
In the coming weeks, as we learn more about this trial and its closure, we will plan to host an interactive webinar for the Sanfilippo A community. Until that time, if you are in need of support please contact Terri Klein at the following email address: [email protected].
 
The Society will continue to advocate and provide support and research directed at improving development, review, and approval of therapies for all unmet needs, including needs of those in our community who live with neurocognitive disease and its challenges. Until we have effective treatments for all in our community, this news is reminder of both the need to support our Sanfilippo A and allied communities and to redouble our efforts in research, family support, and advocacy, to ensure the discoveries, reforms, and supports are available to all those who are in need.
 
With a wish to all that you find both courage and hope in your lives,
 
Matthew
 
N. Matthew Ellinwood
Chief Scientific Officer
National MPS Society