Wednesday, May 25 2022
  • Enabling Investigational New Drug (IND) studies for the Myotonic Dystrophy Type 1 (DM1) program development candidate continue as planned; invited to present new pharmacokinetic (PK) and bioavailability data in rodents during an oral presentation at an upcoming scientific meeting; plans to submit an IND application to the United States Food and Drug Administration (FDA) in the fourth quarter (Q4) of calendar year (CY) 2022
  • Good Manufacturing Practices (GMP) manufacturing to support phase 1/2 clinical trials for successfully implemented DM1 program
  • Plans to nominate a development candidate and initiate scale-up and toxicology activities for a systemically administered selective allele candidate for the Huntington’s disease (HD) program in CY2022 with the goal of filing a IND request with FDA in CY2023
  • Advanced KRAS G12D and G12V oncology programs supported with mechanical work and in vivo pharmacology
  • Strengthening of the management team with the appointment of Todd P. Branning as Chief Financial Officer (CFO)

PITTSBURGH and CAMBRIDGE, Mass., Feb. 10 28, 2022 (GLOBE NEWSWIRE) — NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a Drugging the Genome™ biotechnology platform company to fight disease at the grassroots level using a novel class of precision genetic drugs, today announced its financial results for the three-month period ended December 31, 2021, along with other recent developments.

“We are making significant progress in advancing IND-enabling studies for the development candidate in our myotonic dystrophy type 1 (DM1) program, and expect to file an IND with the FDA in Q4 CY2022” , said Dietrich A. Stephan, Ph.D., founder, CEO and president of NeuBase. “These studies are on track for data readouts to occur throughout CY2022, with the first presentation of rodent pharmacokinetics and bioavailability data at an upcoming scientific meeting. We expect these data to illustrate the differentiated potential of our candidate as a whole-body solution to treat DM1 and the unique ability of our delivery shuttle to distribute in the brain. The ability to cross the blood-brain barrier and reach the deep brain is also particularly relevant to our Huntington’s disease program, where we plan to launch scale-up and toxicology activities this year.

Dr Stephan added: “Furthermore, I am particularly pleased to have welcomed Todd to the management team as Chief Financial Officer. The team and the science are strong, and I believe we are at a pivotal moment for NeuBase as we build a robust dataset that should support the introduction of our first candidate into the clinic for DM1, validate our platform. technological form of genetic medicine to efficiently deliver genetic drugs with wide tissue distribution, including in the deep brain, and to precisely engage genetic mutations in a well-tolerated manner with the potential for long-lasting efficacy.

First quarter of fiscal 2022 and recent operational highlights

  • Myotonic Dystrophy Type 1 (DM1) Program: NeuBase is making steady progress in advancing studies enabling IND for its development candidate in the DM1 program, which includes pharmacokinetics, absorption, distribution, metabolism and excretion (ADME) and bioavailability via intravenous (IV) and subcutaneous (SQ) routes of administration, exploratory toxicology and IND enabling good laboratory practice (GLP) and mechanism of action studies. Additionally, GMP manufacturing of the NeuBase development candidate to support Phase 1/2 clinical trials has been successfully implemented through contract manufacturing organizations. The company plans to announce a strong set of data through posters and presentations at scientific meetings and peer-reviewed publications throughout CY2022. NeuBase expects these data to support the submission of an IND dossier to the FDA in Q4 CY2022.
  • Huntington’s Disease (HD) Program: The HD program is currently in preclinical development. In CY2022, NeuBase plans to nominate a development candidate and initiate scale-up and toxicology activities to support an IND filing with the FDA in CY2023.
  • kras Oncology program: The Company leads in vitro mechanistic studies and live pharmacology studies for kras program (kras G12V and G12D mutations).
  • Appointment of a new CFO: The company has named Todd P. Branning as chief financial officer. Mr. Branning brings over 25 years of experience leading corporate finance and accounting, tax, financial planning and analysis, and investor relations for several publicly traded pharmaceutical companies. .

Financial results for the fiscal quarter ended December 31, 2021

  • As of December 31, 2021, the Company had cash and cash equivalents of approximately $47.3 million, compared to approximately $52.9 million as of September 30, 2021.
  • NeuBase estimates that its current cash and cash equivalents are sufficient to fund currently planned operating and capital expenditures through the first quarter of calendar year 2023.
  • For the fiscal quarter ended December 31, 2021, the Company reported a net loss of approximately $7.7 million, or a net loss of $0.24 per share, compared to a net loss of approximately $4.1 million, or a net loss of $0.18 per share, for the same period last year.
  • For the fiscal quarter ended December 31, 2021, total operating expenses were approximately $7.3 million, consisting of approximately $2.9 million in general and administrative expenses and $4.4 million in expenses. of research and development. This compares to total operating expenses of approximately $4.7 million for the same period last year, comprised of approximately $2.7 million in general and administrative expenses and $2.0 million in dollars in research and development costs.

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™ that encompasses a novel peptide-nucleic acid antisense oligonucleobase 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 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 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 our expectations to submit an IND to the FDA for our DM1 program by 4Q CY2022 and our other expectations for our DM1 program, our expectation to initiate scale-up activities and toxicology for the development of a systemically administered allele-selective NT-0100 program to treat HD in CY2022 and targeting an IND filing for this program with the FDA for CY2023, the potential of our therapeutic program for HD and the potential of our PATrOL™-compatible silence-activating compounds kras point mutations live to inhibit protein production. These views 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, may cause our actual results to differ from those expressed in the statements. prospective. 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; 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 Securities and Exchange Commission of United States. Except as 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]

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