NeuBase Therapeutics to Present New Preclinical Data for Myotonic Dystrophy Type 1 Program at the 2022 MDA Clinical & Scientific Conference

Rhea-AI Summary

NeuBase Therapeutics (Nasdaq: NBSE) announced the presentation of new preclinical data on its myotonic dystrophy type 1 (DM1) program at the 2022 MDA Clinical & Scientific Conference, occurring from March 13-16. The presentations include details on pharmacology, biodistribution, and tolerability of its PATrOL™-enabled investigational genetic therapy. Results from the HSA^LR mouse model highlight the therapy's effectiveness in targeting toxic RNA structures and restoring RNA splicing, crucial for DM1 treatment, which may modify disease impact on patients.

Positive

• Presentation of promising preclinical data at a key conference, indicating progress in DM1 treatment.
• New pharmacokinetic data showing broad distribution of investigational therapy, including brain and muscle absorption.
• Demonstrated molecular and functional pharmacological activity of the therapy in animal models.

Negative

• None.

PITTSBURGH and CAMBRIDGE, Mass., Feb. 28, 2022 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology platform company Drugging the Genome™ to address disease at the base level using a new class of precision genetic medicines, today announced that new preclinical data from its myotonic dystrophy type 1 (DM1) program will be featured in presentations at the 2022 MDA Clinical & Scientific Conference. MDA 2022 will be taking place virtually and in-person in Nashville, Tennessee, from March 13-16, 2022, and the abstracts will be available on the meeting website.

Details of the presentations are as follows:

Title: Pharmacology, Biodistribution and Tolerability of a PATrOL™-Enabled Investigational Genetic Therapy for Myotonic Dystrophy, Type 1
Type: Poster Presentation
Date: Available for viewing Sunday, March 13 through Tuesday, March 15

Title: A PATrOL™- Enabled Investigational Genetic Therapy for DM1: Mouse Pharmacokinetics, Biodistribution, and CNS Penetration after Systemic Administration
Type: Oral Presentation
Date: Wednesday, March 16, 9:30am-9:45am CST

Expanding upon initial data presented in June 2021, the presentations will include new data of a PATrOL™-enabled investigational genetic therapy for DM1. NeuBase will present molecular and functional pharmacological activity of its DM1 investigational genetic therapy in the HSA^LR mouse model following single and repeated subcutaneous, intravenous, and intramuscular administration. The HSA^LR model carry the long repeat (LR) length of the DMPK repeat expansion and recapitulates many aspects of the clinical presentation of DM1. Additional pharmacokinetic data will be presented of NeuBase’s DM1 investigational genetic therapy in wild-type mice demonstrating distribution and pharmacologic activity throughout the body, including the brain and muscle, following systemic administration.

Patients with DM1 suffer from cognitive deficits and muscle pathology caused by a trinucleotide expansion in the DMPK gene. NeuBase’s DM1 investigational genetic therapy targets DMPK pre-mRNA with a novel peptide-nucleic acid (PNA) pharmacophore and is designed to selectively engage with the toxic RNA hairpin structure and release the splicing proteins to 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. For more information, visit https://www.neubasetherapeutics.com/pipeline/.

About NeuBase Therapeutics
NeuBase is accelerating the genetic revolution by developing a new class of precision genetic medicines that Drug the Genome™. The Company’s therapies are built on a proprietary platform called PATrOL™ that encompasses a novel peptide-nucleic acid antisense oligonucleotide technology combined with a novel delivery shuttle that overcome many of the hurdles to selective mutation engagement, repeat dosing, and systemic delivery of genetic medicines. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular, and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions, who currently have limited to no treatment options. To learn more, visit www.neubasetherapeutics.com.

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 use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the potential and prospects of our myotonic dystrophy type 1 (DM1) program. These views involve risks and uncertainties that are difficult to predict and, accordingly, 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 those risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the risks that prior data will not be replicated in future studies; the timing of any planned 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 commercialization, 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 those risk factors contained in our filings with the U.S. Securities and Exchange Commission. 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.

Source: NeuBase Therapeutics, Inc.

NeuBase Investor Contact:
Dan Ferry
Managing Director
LifeSci Advisors, LLC
daniel@lifesciadvisors.com
OP: (617) 430-7576

NeuBase Media Contact:
Jessica Yingling, Ph.D.
Little Dog Communications Inc.
(858) 344-8091
jessica@litldog.com

FAQ

What are the new findings presented by NeuBase Therapeutics at the 2022 MDA Clinical & Scientific Conference?

NeuBase presented new preclinical data on its investigational genetic therapy for myotonic dystrophy type 1, highlighting pharmacological activity and biodistribution.

When will NeuBase's presentations on DM1 be available for viewing?

The presentations will be available from March 13 through March 15, 2022, with an oral presentation on March 16 at 9:30am CST.

How does NeuBase's therapy aim to treat myotonic dystrophy type 1?

The therapy targets DMPK pre-mRNA using a peptide-nucleic acid pharmacophore to engage toxic RNA structures, aiming to restore proper RNA splicing.

What animal model is used in NeuBase's research for DM1?

NeuBase utilizes the HSA^LR mouse model, which replicates characteristics of myotonic dystrophy type 1.

What is the significance of NeuBase's PATrOL™ technology in its treatments?

PATrOL™ technology enhances the delivery of genetic medicines, facilitating engagement with disease-causing mutations and improving therapeutic outcomes.