NeuBase Therapeutics Reports Third Quarter Fiscal 2021 Financial Results and Recent Operating Highlights

  • Further demonstrated the wide potential of the new genetic medicine platform with in vivo data in three diseases driven by different genetic disease mechanisms

  • Presentation of preclinical data showing that compounds activated with proprietary NeuBase delivery technology are well tolerated at pharmacologically active doses and are delivered beyond the liver after systemic administration

  • Recent data supports the advancement of the development of the myotonic dystrophy type 1 (DM1) program; IND filing expected in the fourth quarter of CY 2022

  • The treasury track is expected to finance the operating and capital expenditure currently planned until the first quarter of the year 2023

PITTSBURGH, August 12, 2021 (GLOBE NEWSWIRE) – NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology platform company Drugging the Genome ™ to treat disease at the grassroots level using a new class of precision genetic drugs, today announced its financial results for the three and nine month periods ended June 30, 2021.

“In June, we presented in vivo data on new compounds demonstrating the selective silencing of disease-causing mutations at the DNA or RNA level in three diseases, each caused by a different underlying genetic mechanism. These new data further illustrate the broad applicability of our genetic medicine platform.,Said Dietrich A. Stephan, Ph.D., Founder, CEO and Chairman of NeuBase. “After intravenous or subcutaneous administration, these compounds were well tolerated at pharmacologically active doses. In addition, the compounds have achieved targeted delivery to the brain and muscles, further confirming our claim to offer the unique ability to deliver genetic drugs throughout the body. “

“For our lead program in DM1, recent data support a differentiated therapeutic approach to maintain DMPK function while selectively silencing the disease causing mutation. With this positive data in hand, we believe we have a clear path to entry into the clinic and expect an IND filing in the fourth quarter of calendar year 2022, ”continued Dr Stephan. “We continue to advance our therapeutic program for Huntington’s disease and believe that our proprietary delivery technology will allow our compounds to go beyond intrathecal delivery, overcoming challenges encountered with other programs. .

Dr Stephan concluded: “Finally, we have shown that we can silence the activation KRAS point transfers in vivo to inhibit protein production, which has the potential to target G12D and G12V, the two most common and historically “non-drug” KRAS conductive mutations which represent the majority of tumors induced by KRAS. This paves the way for the generation of novel precision genetic drugs capable of selectively targeting mutations at a single base level to treat rare and common diseases. “

Third Quarter of Fiscal 2021 and Recent Operating Highlights

  • Completion of an oversubscribed public offering led by renowned investors in fundamental health for net proceeds of $ 42.6 million, offering a cash flow path through 2023.

  • Presentation of preclinical data demonstrating:

    • Proprietary Genetic Medicines Platform Generates New Compounds that Selectively Suppress Genetic Mutations That Cause Disease in vivo without permanently modifying the genome

    • Functional rescue of myotonic dystrophy type 1 (DM1) phenotype in vivo after subcutaneous administration, as well as tolerance to pharmacologically active doses

    • In vivo proof of concept in Huntington’s disease with knock-down of an allele-selective mutant protein after administration of compounds across the blood-brain barrier after subcutaneous administration

    • Pharmacological activity against historically “non-drug” KRAS pilot mutations in a variety of patient-derived tumor lines in xenograft models

    • Penetration of the blood-brain barrier with pharmacological activity in the CNS and wide tissue distribution and activity of compounds after subcutaneous administration

    • Compounds are well tolerated and consistent with historical data

  • Expansion of the management team with the appointment of Sandra Rojas-Caro, MD, as Medical Director responsible for overseeing the preclinical and clinical development, medical and regulatory strategy of the Company’s pipeline; and Kia Motesharei, Ph.D., as Director of Business and Strategy to oversee business development and alliance management and work with the CEO on corporate strategy

Financial results for the third fiscal quarter ended June 30, 2021

  • As at June 30, 2021, the Company had cash and cash equivalents of approximately $ 58.8 million, compared to approximately $ 32.0 million as at September 30, 2020

  • NeuBase believes its current cash and cash equivalents are sufficient to fund current planned operating and capital expenses in the first quarter of CY2023

  • For the three-month period ended June 30, 2021, the Company reported a net loss of approximately $ 8.7 million, or a net loss of $ 0.29 per share, compared to a net loss of approximately 3 , $ 8 million, or a net loss of $ 0.18 per share. , for the three-month period ended June 30, 2020

  • For the three-month period ended June 30, 2021, total operating expenses were approximately $ 8.8 million, of which approximately $ 3.5 million were general and administrative expenses, $ 2.5 million in research and development costs and $ 2.9 million in research and development – Vera acquisition costs. This compares to total operating expenses of approximately $ 3.8 million for the three-month period ended June 30, 2020, comprised of approximately $ 2.3 million in general and administrative expenses and $ 1.5 million. million dollars in research and development costs.

Financial results for the nine-month period ended June 30, 2021

  • For the nine-month period ended June 30, 2021, the Company reported a net loss of approximately $ 18.3 million, or a net loss of $ 0.72 per share, compared to a net loss of approximately 12 , $ 7 million, or a net loss of $ 0.69 per share. , for the same period last year

  • For the nine-month period ended June 30, 2021, total operating expenses were approximately $ 19.4 million, of which approximately $ 8.8 million were general and administrative expenses, $ 7.7 million in research and development costs, $ 2.9 million in research and development – Vera acquisition costs. This compares to total operating expenses of about $ 12.0 million for the same period last year, or about $ 7.6 million in general and administrative costs and $ 4.3 million in maintenance costs. research and development.

About NeuBase Therapeutics
NeuBase is accelerating the genetic revolution by developing a new class of precision genetic drugs that can be designed to increase, decrease, or modify gene function, as needed, to resolve genetic defects that lead to disease. NeuBase’s PATrOL ™ Targeted Therapies are centered on its proprietary drug scaffold to treat genetic diseases at the DNA or RNA level by combining the highly targeted approach of traditional genetic therapies with the broad organ delivery capabilities small molecules. By initially focusing on silencing disease-causing mutations in debilitating neuromuscular, neurological and oncological disorders, NeuBase is committed to redefining medicine for the millions of patients with common and rare diseases. 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”, “designed”, “plan” or “intend” , the negative of these terms and similar references to future periods. These forward-looking statements include, among others, those relating to the outlook for DM1 and the Company’s expectation to file an IND filing for DM1 in the fourth quarter of CY 2022, the Company’s therapeutic program for Huntington’s disease, the ability of the Company to target the G12D and G12V and the Company’s expectation that its liquidity will fund operating and capital expenditures currently forecast through the first quarter of CY 2023. These views involve risks and uncertainties that are difficult to ascertain. forecast 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 the risk factors contained in our filings with the United States Securities and Exchange Commission (the “SEC”), may cause our actual results to differ. those expressed in forward-looking statements. The Company may not actually carry out the plans, achieve the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on such forward-looking statements. Since these statements deal with 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 under- heard by statements in this press. publication, including: the Company’s plans to develop and market its product candidates; the timing of the launch of the clinical trials planned by the Company; the risks that past data may not be reproduced in future studies; the timing of any investigational new drug request or new drug request; the Company’s plans to research, develop and market its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company’s product candidates; the Company’s marketing, 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 documents filed with the SEC. Unless otherwise provided by law, the Company disclaims any intention or obligation to update or revise forward-looking statements, which are only valid as of the date hereof, whether as a result of new information, events or future circumstances or otherwise.

Contact for NeuBase investors:
Dan Ferry
General director
LifeSci Advisors, LLC
[email protected]
OP: (617) 430-7576

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

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