NeuBase Therapeutics presents new preclinical data at MDA 2022 for its Myotonic Dystrophy Type 1 program demonstrating splice rescue, resolution of nuclear aggregates and reversal of myotonia

NeuBase Therapeutics, Inc.

  • only one intravenous NeuBase NT-0231.F Myotonic Dystrophy Type 1 (DM1) development candidate dose, powerfully rescues splicing, restorations function of the chloride channel (Clcn1) and permanently reverses myotonia in HSAL/R model

  • Multiple subcutaneous doses also lead to molecular and functional rescue in SAHL/R model demonstrating the efficacy in a dose-dependent manner and the potential feasibility of this patient-friendly route of administration

  • Company remains on track to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in Q4 2022

  • NeuBase management to host conference call and webcast tomorrow, March 14 at 8:00 a.m. EDT

PITTSBURGH and CAMBRIDGE, Mass., March 13, 2022 (GLOBE NEWSWIRE) — NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a Drugging the Genome™ biotechnology platform company to combat the disease at the basic level using a new class of precision genetic drugs, today announced new preclinical data for its lead development candidate, NT-0231.F, to treat myotonic dystrophy type 1 (DM1) . These data are presented as posters and oral presentations at the MDA 2022 Clinical and Scientific Conference, taking place virtually and in person in Nashville, Tennessee, March 13-16, 2022. These data will also be presented at RNA Leaders World Congress in Basel on March 17.

Sandra Rojas-Caro, MD, NeuBase Chief Medical Officer, said, “DM1 is a significant unmet medical need characterized by myotonia, muscle weakness and wasting, and cognitive impairment. We are excited to present new preclinical data for our lead candidate, NT-0231.F, to support our differentiated approach for DM1. Systemic administration of NT-0231.F achieves clinically relevant molecular and functional rescue in muscle from SAHL/R model, which reproduces many components of the disease. Initial pharmacokinetic data support a comprehensive solution to the disease, and additional preclinical work is underway to assess biodistribution in key tissues. These data are an important milestone in our DM1 development program and support our quest for a first-in-class therapeutic profile for the disease. »

A single intramuscular dose confirmed that NT-0231.F is pharmacologically active in muscle and results in molecular and functional rescue in SAHL/R model, including splice rescue, resolution of nuclear aggregates, and reversal of myotonia (delayed muscle relaxation after contraction). A single intravenous (IV) dose of NT-0231.F or multiple subcutaneous (SC) doses over a 28-day period largely rescued splicing, including the chloride channel (Clcn1) transcription and reverse myotonia in the model. A single IV dose of NT-0231.F provides initial splice rescue at around two weeks, with significant splice rescue around three weeks. Reversal of myotonia was achieved after about four weeks, with effects lasting for at least six weeks, the longest period tested to date. A time course of multiple SC doses at increasing concentrations of NT-0231.F was also investigated and showed splice rescue and reversal of myotonia in a dose-responsive manner, illustrating the feasibility of the pathway. Differentiated and patient-friendly SC. In pharmacokinetic studies of NT-0231.F in wild-type BALB/C mice, a single IV or SC dose showed a high volume of distribution, suggesting wide tissue distribution.

Dietrich A. Stephan, Ph.D., Founder, CEO and President of NeuBase, said, “We now have preclinical data in the reference animal model for our development candidate demonstrating robust reversal of myotonia, as measured by muscle relaxation in these studies. We believe we are the only company to have demonstrated improved muscle relaxation after systemic routes of administration. HSAL/R The model also sets a high bar for human disease in that it contains at least 10 times more mutant gene CUG repeat targets than patients, giving us additional confidence in the robustness of our approach. Not only does this data support the continued advancement of our lead program in DM1 and keep us on track to submit an Investigational New Drug Application to the United States Food and Drug Administration by 4Q CY2022, it also validates that we can use our PATrOLMT platform to design new genetic drugs that target and rescue many other genetic dysfunctions, with the potential for clinically meaningful results in rare and common diseases.

Conference call and webcast details
NeuBase Therapeutics, Inc. will discuss this data in a webcast conference call accompanied by slides tomorrow, March 14, 2022 at 8:00 a.m. EDT. To access the webcast, please Click here. An archived recording of this presentation will be available after the call via the Investor Relations Calendar page in the Investors section of the Company’s website,

About the NeuBase DM1 program
Patients with DM1 suffer from cognitive deficits and muscle pathologies caused by an expansion of trinucleotides in the DMPK gene which, when transcribed, results in an RNA hairpin structure that sequesters RNA splicing proteins. NeuBase’s DM1 Experimental Gene 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 hairpin structure of RNA, release splicing proteins, and restore splicing of RNA and downstream protein production. The PNA pharmacophore is conjugated to NeuBase’s novel delivery technology which is designed for broad distribution, including in the deep brain, with the potential of a whole-body disease modification solution for DM1. For more information, visit

About NeuBase Therapeutics
NeuBase is accelerating the genetic revolution by developing a new class of precision genetic drugs, Drug the Genome™. The Company’s therapies are based on a proprietary platform called PATrOL™ which encompasses a novel peptide-nucleic acid antisense oligonucleotide technology combined with a novel delivery shuttle that overcomes many barriers to selective mutation engagement, repeated administration and systemic administration of genetic drugs. With an initial focus on inactivating pathogenic mutations in debilitating neurological, neuromuscular and oncological disorders, NeuBase is committed to redefining medicine for the millions of patients with common and rare diseases, who currently have little or no of treatment options. To learn more, visit

Use of forward-looking statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by the use of words such as “will”, “would”, “anticipate”, “expect”, “believe”, “design”, “plan” or “intend”. , the negative of such terms and similar references to future periods. These forward-looking statements include, among others, those related to the plan to provide updates on the Company’s development pipeline, including Myotonic Dystrophy Type 1 (DM1) at the MDA 2022 Clinical and Scientific Conference and the Global RNA Leaders Congress 2022, and the potential and outlook for the Company’s proprietary PATrOL™ platform and the Company’s DM1 program. These opinions involve risks and uncertainties that are difficult to predict and, therefore, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including risk factors contained in our filings with the United States Securities and Exchange Commission (“SEC”), may cause our actual results to differ from those expressed in the forward-looking statements. The Company may not actually achieve the plans, achieve the intentions or meet the expectations or projections disclosed in any forward-looking statements, and you should not place undue reliance on such forward-looking statements. Because these statements address future events and are based on the Company’s current expectations, they are subject to various risks and uncertainties, and the actual results, performance or achievements of the Company could differ materially from those described or underlined. understood by the statements in this press release. publication, including: the Company’s plans to develop and commercialize its product candidates; the timing of the initiation of the Company’s planned clinical trials; the risks that past data will not be replicated in future studies; the timing of any anticipated investigational new drug application or new drug application; the Company’s plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company’s product candidates; the Company’s sales, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company’s ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as the risk factors contained in our filings with the SEC. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

NeuBase Investor Contact:
Dan Ferry
General manager
LifeSci Advisors, LLC
[email protected]
PO Box: (617) 430-7576

NeuBase media contact:
Jessica Yingling, Ph.D.
Little Dog Communications Inc.
(858) 344-8091
[email protected]