Communications

Community Update,
November, 2021

To The NPC1 Community,

First, we want to express our gratitude to all the members of the community who continue to work hard to ensure the voices and experiences of patients and families are heard. We would like to take this opportunity to recognize the patients, parents, and advocacy organizations working to build greater awareness and understanding of NPC, and the clinicians and researchers further demonstrating the value of the tools and datasets we have for use in NPC today.

Next, we share our thanks to the Janssen Pharmaceutical Companies of Johnson & Johnson for their support in providing access to unused vials to support our efforts to build additional supply of adrabetadex*.

Finally, we turn to providing another brief update on our continued progress with adrabetadex. This is a period of transition for many, as final treatment appointments in the VTS-270 clinical trials were completed in October. We are now working to support uninterrupted treatment access through the transfer and enrollment of patients into the Expanded Access Program (EAP) in November for those who meet the eligibility criteria and continue to receive benefit.

As part of our monthly communication to help keep the community up to date on our Expanded Access Program efforts, we are happy to share our most current dashboard summary below.

Summary DASHBOARD of EAP Efforts (as of November 1, 2021)

Current Sites

13 distinct sites are currently active in EAP

New/Pending Sites

6 pending new sites for EAP

Current patients enrolled in Expanded Access Program

45 patients

Development

The effort and resource required to undertake responsibility for the clinical trials and simultaneously demonstrate our commitment to the EAP program so that it could continue beyond October 20th has been very successful. Mandos can now provide additional focus towards the advancement of the development of adrabetadex.

We continue to work with several different organizations to gain access to additional data and to analyze the totality of that data. We are encouraged by the engagement from the community, with many members having spent days or weeks of their time sharing their expertise and insights with us.

A critical part of our efforts is documenting potential markers of clinical response from physicians and families. It’s important to us that we understand what physicians see in the clinic, and how those observations translate into what patients and families experience at home and in their everyday activities. If you are a physician with experience treating NPC1 patients or a family with experience with adrabetadex that we’ve not heard from yet, please reach out to us via our website’s “Contact Us” page.

We know there is more work to do in understanding which symptoms most impact patients and their families, and how we can best capture and present the information that demonstrates how the progression of those symptoms may change in response to treatment. We look forward to updating you all again soon on our efforts.