NeuBase Therapeutics, Inc. announced the presentation of preclinical pharmacokinetics (PK) and biodistribution data for its lead development candidate, NT-0231.F, supporting a differentiated whole body treatment solution for myotonic dystrophy type 1 (DM1). These new data are being presented today in a poster session at the American Society of Gene and Cell Therapy (“ASGCT”) 25th Annual Meeting, taking place virtually and in person in Washington, D.C., May 16-19, 2022. Following a single intravenous (IV) injection of 30 mg/kg in wild-type BALB/c mice, NT-0231.F was cleared rapidly from the systemic compartment and demonstrated rapid and wide distribution into tibialis anterior muscle, heart muscle, and brain tissues.

NT-0231.F rapidly cleared the plasma, and each tissue evaluated displayed an extended elimination phase with tissue concentrations measurable for at least four weeks following a single IV dose administration. Patients with DM1 suffer from cognitive deficits and muscle pathology caused by a trinucleotide expansion in the DMPK gene which, when transcribed, results in an RNA hairpin structure that sequesters RNA splice proteins. NeuBase's DM1 investigational genetic therapy, NT-0231.F, targets mutant DMPK pre-mRNA with a novel peptide-nucleic acid (PNA) pharmacophore and is designed to selectively engage with the toxic RNA hairpin structure, release the splicing proteins, and restore RNA splicing and downstream protein production.

The PNA pharmacophore is conjugated to NeuBase's novel delivery technology that is designed for broad distribution, including into the deep brain, with the potential for a whole body, disease-modifying solution for DM1.