Recently, Peter J. deSilva, CMTRF Board member and Chair of the ENDGAME Capital Campaign to end CMT1A and Susan Ruediger, CMT patient, co-founder, and Chief Mission Officer of the CMTRF, hosted a private call with donors to update them on the progress being made in CMT1A drug development and ENDGAME, the campaign to end CMT1A.
The result of the call was an immediate pledge of $520,000 in new donations to ENDGAME, bringing the total the campaign has raised to over $6.6 million.
The program included an overview of the 9 projects funded directly by ENDGAME and the resulting 5 pre-clinical candidates for CMT1A that include: DTx/Novartis (candidate: DTx-1252); Oryzon Genomics (ORY-4001) Armatus Bio (ARM-101); Shift Pharmaceuticals (SHC1-012); and Augustine Therapeutics (AGT-216).
Dr. Paul August, who serves on both CMTRF’s Scientific Advisory Board and its governing Board, is the Chief Scientific Officer at ReviR Therapeutics and is a leader in the development of therapeutics for neurodegenerative diseases, discussed the remarkable progress CMTRF has made in a very short period of time. “In just five years CMTRF has become the most significant force in the CMT research arena, funding 19 projects that have all advanced the mission of finding a treatment or cure,” noted Dr. August.
One of the early programs CMTRF funded was DTx Pharma, a young company that had developed a platform for RNA therapeutics called Fatty Acid Ligand Conjugated Oligonucleotide. CMTRF executives suggested DTx’s platform, developed for another indication, could be relevant to the delivery challenge of drugs to Schwann cells. CMTRF then funded an initial project to validate that in January 2020. The success of that work earned DTx a grant from the NIH and supported their raising more than $115 million based on the development of DTx-1252, a first-in-class FALCON siRNA therapeutic for the treatment of CMT1A. DTx was recently acquired by Novartis for $1 billion.
Dr. Rachel Salzman, Global Head of Corporate Strategy at Armatus Bio outlined the impact in the scientific and pharma industries of the Novartis acquisition noting that “the publicity of the sale was noticed by the entire CMT ecosystem from patients to phama to the investors in effect derisking additional investments in CMT research. The Novartis deal will definitely encourage other pharma companies to get into or stay in CMT drug development.”
“I continue to be humbled and gratified by the response to the CMT1A Endgame Campaign. Our generous donors enable us to identify and fund the most promising CMT1A research in search of treatments and cures,” says Mr. deSilva, author of Taking Stock: 10 Life and Leadership Principles from My Seat at the Table. “We will not rest until the needed funds have been fully raised and deployed.”
