DTx Pharma Receives FDA Orphan Drug Designation for DTx-1252 for the Treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A)

SAN DIEGO, June 7, 2023 /PRNewswire/ -- DTx Pharma, a biotechnology company addressing the delivery challenges of oligonucleotide therapeutics with its Fatty Acid Ligand Conjugated OligoNucleotides (FALCON™) platform, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to DTx-1252, an investigational FALCON small interfering RNA (siRNA) therapeutic for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A). CMT1A is a progressive, neuromuscular, autosomal-dominant disease that leads to life-long loss of muscle function and disability.

"We are pleased to receive Orphan Drug Designation from the FDA for DTx-1252. Patients living with CMT1A are in urgent need of treatment and this represents a significant step forward for the patients and the DTx team as we advance DTx-1252 toward the clinic," said Artie Suckow, Ph.D., co-founder and CEO of DTx Pharma. "DTx-1252 targets the underlying genetic lesion of the disease and leverages our FALCON platform to unlock the promise of RNAi therapeutics. We look forward to the continued development of DTx-1252."

The FDA Office of Orphan Products Development (OOPD) provides orphan status to drugs and biologics which are intended to treat, diagnose or prevent rare diseases that affect fewer than 200,000 people in the U.S.

Currently, there are no approved therapeutics addressing PMP22, the underlying genetic cause of CMT1A, for the estimated 150,000 patients living with the debilitating disease in the U.S. and Europe. DTx-1252, is a novel, potential first-in-class candidate targeting PMP22.  The asset boasts a robust preclinical package, demonstrating reversal of disease in preclinical rodent models and translation to higher species with IND-enabling studies near completion.

About CMT1A
CMT1A is the most common inherited neuromuscular disease, affecting 150,000 patients in the U.S. and EU. Currently, there are no approved therapies for patients. CMT1A is driven by overexpression of the PMP22 gene in Schwann cells, which prevents myelination of peripheral nerves and causes progressive muscle wasting, neuropathic pain, difficulty walking and, eventually for many, an inability to live independently, leading to a significant and debilitating impact on patient lives.

About DTx-1252
DTx-1252 is a potential first-in-class FALCON™ siRNA therapeutic for treatment of CMT1A. By repressing PMP22, DTx-1252 reverses CMT1A in a mouse model that faithfully recapitulates the genetic and clinical manifestations of the disease. DTx-1252 treatment induces remyelination of axons to normal levels, improves relevant electrophysiological measurements and increases muscle mass, grip strength, coordination and agility in preclinical studies.

About DTx Pharma 

DTx Pharma, Inc. is a privately held biotechnology company, addressing the challenges associated with delivering RNA-based therapeutics beyond the liver with the company's proprietary Fatty Acid Ligand Conjugated OligoNucleotides (FALCON™) technology platform. The FALCON platform leverages fatty acids for enhanced biodistribution and cellular uptake to tissues and cell types throughout the body. In preclinical studies, FALCON siRNAs have demonstrated potent and durable repression of target genes in the peripheral nervous system (PNS), skeletal muscle, heart, skin and central nervous system (CNS). FALCON siRNAs can be delivered by intravenous, subcutaneous and intrathecal routes of administration and can be manufactured at low cost. The company has a pipeline focused on PNS, neuromuscular and CNS diseases. DTx Pharma has raised more than $115M in combined investment from several of the world's leading healthcare investors including RA Capital Management and Access Biotechnology, and research foundations such as the CMT Research Foundation (CMTRF).

To learn more about DTx Pharma, please visit www.dtxpharma.com and follow us on LinkedIn and Twitter. 

Company Contact
Christina Trout
DTx Pharma
[email protected]

Media Contact
Ignacio Guerrero-Ros, Ph.D.
Russo Partners
646-942-5604
[email protected]

SOURCE DTx Pharma

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