Notes from NPC Therapy Accelerator Meeting – 3/16/2021

Sean Kassen, APMRF
Justin Hopkin, NNPDF
Phil Marella, Danas Angels Research Trust, NPC Parent
Cristin Davidson, NPC Scientist
Dave Zook, Faegre Drinker
Tim Franson, Faegre Drinker

The main topic of the conversation was the Parseghian Fund had a 1 hr meeting with Mallinckrodt Pharmaceuticals, which included Elissa Cote, Josh Schafer, and Vadim Rechanskiy and below are points that were shared with the team:

  • Mallinckrodt has been spending a lot of time reconsenting patients
  • There is still no timeline for a formal meeting with the NTA due to scheduling and information gathering
  • Mallinckrodt is looking to identify a new sponsor to take over the IND for Adrabetadex
  • Mallinckrodt indicated to the Parseghians that it was best for the community to reach out to the FDA outside of MNK.  Though our consultants confirmed that the FDA could only discuss the issues around Adrabetadex with the sponsor.
  • Discussed the costs associated with running the Adrabetadex program

The group also discussed conversations around Miglustat with Edenbridge and continue to work with them as they have discussions with the FDA.

We also are coordinating a call with Biographene, a company with a novel approach to look at NPC disease.

  1. Our next steps were to continue to share information across the group in regards to Adrabetadex
  2. Send a note to the NPC community about the NTA
  3. Continue to engage MNK as they plan a path for Adrabetadex.

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