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.
MEET THE CEO & FOUNDER OF NEUBASE
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.
RECENT NEWS FROM NEUBASE
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.
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.