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New Data To Be Presented By Neubase Therapeutics On Myotonic Dystrophy Type 1 Program At #MDA2022 In Nashville- $NBSE $XBI

By John F. Heerdink, Jr.
Patients with myotonic dystrophy type 1 (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.  

On Monday, Feb 28, NeuBase Therapeutics, Inc. (Nasdaq: NBSE), a biotechnology platform company Drugging the Genome™ to address disease at the base level using a new class of precision genetic medicines, 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). 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. The Conference, the largest of its kind, highlights unprecedented research advancements and clinical achievement in NMD.

Expanding upon initial data presented in June 2021, Neubase’s 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 HSALR mouse model following single and repeated subcutaneous, intravenous, and intramuscular administration. The HSALR 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.

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

The Muscular Dystrophy Association (MDA) is transforming the lives of people with muscular dystrophy, ALS and related neuromuscular diseases, through their funding of research, care, and advocacy for over 300,000 families in the United States. For more information visit mda.org.


On February 3rd, our sister organization, Tribe Public hosted a Webinar Presentation and Q&A Event titled “Drugging The Genome.” The event was co-hosted by Dietrich A. Stephan, Ph.D, CEO, & Founder of NeuBase Therapeutics (NASDAQ: NBSE). The event video may now be viewed at the Tribe Public YouTube Channel at this link and has been over 22k times.


On Feb. 10, NeuBase reported its financial results for the three-month period ended December 31, 2021, and other recent developments. Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase stated,  “We are making significant progress in advancing the IND-enabling studies for the development candidate for our myotonic dystrophy type 1 (DM1) program, and we expect to file an IND with the FDA in Q4 CY2022. These studies are on track for data readouts to occur throughout CY2022, with the first presentation of rodent pharmacokinetic and bioavailability data to occur at an upcoming scientific meeting. We expect these data to illustrate the differentiated potential for our candidate to be a whole-body solution to treat DM1 and the unique ability of our delivery shuttle for distribution into the brain. The ability to cross the blood brain barrier and reach the deep brain is also especially relevant for our Huntington’s disease program, where we are planning to initiate scale-up and toxicology activities this year. In addition, I’m especially excited to have welcomed Todd to the executive team as Chief Financial Officer. The team and science are strong, and I believe we are at a pivotal moment for NeuBase as we are building a robust data package that is expected to support bringing our first candidate into the clinic for DM1, validate our genetic medicine technology platform to efficiently deliver genetic medicines with broad tissue distribution, including into the deep brain, and to precisely engage genetic mutations in a manner that is well-tolerated with the potential for sustained efficacy.” READ THE BALANCE OF THE RELEASE HERE. 

On Jan. 10, NeuBase announced the appointment of Todd P. Branning as Chief Financial Officer (CFO). Mr. Branning has more than 25 years of experience leading corporate finance and accounting, tax, financial planning and analysis, and investor relations for several publicly traded pharmaceutical companies. Prior to joining NeuBase, Mr. Branning was CFO of Phathom Pharmaceuticals, Inc., a publicly traded late clinical-stage biopharmaceutical company. Before that, he was Senior Vice President, CFO of Amneal Pharmaceuticals, Inc., a publicly traded pharmaceutical company, where he helped to build, leverage, and optimize infrastructure following the completion of a transformational merger. Prior to joining Amneal, he was Senior Vice President, CFO of the global generic medicines division at Teva Pharmaceutical Industries Ltd., a multinational generic pharmaceuticals company, where he led the finance function and served on the leadership team responsible for managing the day-to-day operations of Teva’s largest multi-billion-dollar commercial unit. Mr. Branning has also held financial leadership roles at Allergan plc, PricewaterhouseCoopers LLP, PPG Industries, Inc., and Merck & Co., Inc. Mr. Branning received his BBA from the University of Miami and MBA from Carnegie Mellon University. Mr. Branning is also a Certified Public Accountant and has completed a CFO certification program at The Wharton School at the University of Pennsylvania.
On Jan. 5, Neubase announced the appointment of Eric J. Ende, M.D., to the Company’s Board of Directors. Dr. Ende has nearly 25 years of experience in advising biotechnology and life sciences companies to optimize corporate strategy and structure and maximize shareholder value. “Dr. Ende has the experience and perspective to recognize the opportunity ahead for NeuBase as it plans for the clinical development of its potentially transformational new class of precision genetic medicines,” said Dietrich A. Stephan, Ph.D., Founder, CEO and Chairman of NeuBase. “We welcome Dr. Ende’s strategic insight as we begin to scale our therapeutic candidate pipeline from our new precision genetic medicines platform technology. In addition to his broad experience, he also shares in our Company’s goal of helping millions of patients with both common and rare conditions that currently have limited or no treatment options.” “I believe NeuBase has a game-changing technology that overcomes the limitations of early precision genetic medicines by delivering mutation selectivity, repeat dosing, and systemic administration in a modular precision medicine platform with the potential to efficiently scale to treat a wide variety of diseases that are currently undruggable,” said Dr. Ende. “I look forward to working closely with NeuBase’s leadership team and Board of Directors to elevate strategy and operations in order to create exceptional value for patients and shareholders.” Dr. Ende currently is the President of Ende BioMedical Consulting Group. He also is a member of the Board of Directors of Matinas BioPharma, where he is the Chairman of the Compensation Committee and serves on the Audit and the Nomination & Governance Committees, and of Avadel plc, where he is the Chairman of the Nomination & Corporate Governance Committee and serves on the Audit and Compensation Committees. Dr. Ende previously served on the Board of Directors of Progenics (acquired by Lantheus Holdings) and Genzyme (acquired by Sanofi-Aventis for $20 billion). During his time on Genzyme’s Board of Directors, Dr. Ende was a member of the Audit and Risk Management Committees. Prior to Genzyme, Dr. Ende was a biotechnology analyst, previously serving at Merrill Lynch, BofA Securities, and Lehman Brothers. Dr. Ende received an M.B.A. from NYU Stern School of Business, an M.D. from the Icahn School of Medicine at Mount Sinai, and a B.S. in biology and psychology from Emory University.


Neubase Therapeutics’ stock trades on the Nasdaq under the ticker NBSE. Shares of NBSE closed trading on Monday, Feb 28 at $1.48/sh, +4.23%. The 52-wk range is $1.21- $10.05 and the 3-month daily trading average is 161,463 shares. 



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