On February 11, 2022 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, reported its financial results for the three-month period ended December 31, 2021, and other recent developments (Press release, NeuBase Therapeutics, FEB 11, 2022, View Source [SID1234607987]).

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"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," said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase. "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."

Dr. Stephan added, "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."

First Quarter of Fiscal Year 2022 and Recent Operating Highlights

Myotonic Dystrophy Type 1 (DM1) Program: NeuBase is making steady progress advancing IND-enabling studies for its development candidate in the DM1 program, which includes PK, absorption, distribution, metabolism, and excretion (ADME), and bioavailability via intravenous (IV) and subcutaneous (SQ) routes of administration, exploratory and IND-enabling Good Laboratory Practice (GLP) toxicology, and mechanism of action studies. In addition, GMP manufacturing of NeuBase’s development candidate to support Phase 1/2 clinical trials has been successfully implemented via contract manufacturing organizations. The Company plans to announce a robust data package through posters and presentations at scientific meetings and peer-reviewed publications throughout CY2022. NeuBase expects these data to support the submission of an IND filing to the FDA in the Q4 CY2022.
Huntington’s Disease (HD) Program: The HD program is currently in preclinical development. In CY2022, NeuBase expects to nominate a development candidate and initiate scale-up and toxicology activities to support an IND filing to the FDA in CY2023.
KRAS Oncology Program: The Company is conducting in vitro mechanistic studies and in vivo pharmacology studies for the KRAS program (KRAS G12V and G12D mutations).
Appointed New Chief Financial Officer: The Company appointed Todd P. Branning as CFO. Mr. Branning brings 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.
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 with approximately $52.9 million as of September 30, 2021.
NeuBase estimates its current cash and cash equivalents are sufficient to fund currently planned operating and capital expenditures into the first quarter of CY2023.
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 with 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 of research and development expenses. This compares with total operating expenses of approximately $4.7 million for the same period last year, consisting of approximately $2.7 million in general and administrative expenses and $2.0 million in research and development expenses.