Message From CMTRF CEO:

This morning, DTx Pharma announced that it has been acquired by Novartis for an upfront payment of $500 million and additional payments of up to $500 million upon completion of pre-specified milestones. This is a very exciting next step in our long support of DTx as Novartis brings substantial resources and drug development experience to help accelerate the process of bringing DTx-1252 through clinical trials and into the marketplace to benefit an estimated 150,000 patients living with CMT1A in the U.S. and Europe.

Novartis’ acquisition of DTx represents a seminal moment in Charcot-Marie-Tooth disease drug development. It is a significant proof of concept for CMT Research Foundation’s out-of-the-box, entrepreneurial approach to finding a cure for all forms of CMT. We hope it will encourage many others in biotech and pharma to take more substantial positions in finding treatments and cures for this difficult disease.

CMTRF was launched just five years ago with an entrepreneurial spirit that challenged the status quo in CMT research. For too long, patients have waited for treatments and the hope of a cure. CMTRF is building on the work of scientists who came before and is identifying new platforms, therapies, and technologies that could solve the specific challenges in drug development for CMT. Of the 19 projects we have funded, five already have clinical candidates, a remarkable record the Foundation and its supporters can be proud of.

One of the early programs we funded was DTx Pharma, a young company that had developed a platform for RNA therapeutics called Fatty Acid Ligand Conjugated Oligonucleotide or (FALCON™).

CMTRF executives suggested DTx’s FALCON platform, developed for another indication, could be relevant to the delivery challenge of drugs to Schwann cells. We 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 Pharma is currently conducting safety studies in animals to support an Investigational New Drug (IND) application to the FDA for a CMT1A treatment. To prepare for clinical trials in humans – which are expected to start in late 2023 or early 2024 – CMTRF introduced DTx to scientific and clinical experts in CMT, key opinion leaders and the national clinical network that can support the trials of DTx-1252.

To the families who have funded this work, we say thank you. Financial support from the families and friends of the CMTRF created this opportunity and development, furthering the proof that philanthropy can change the outlook of patients and families who struggle with a rare disease and hope for treatment.

Cleary Simpson, CEO, CMT Research Foundation

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