On May 12, 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 March 31, 2022, and other recent developments (Press release, NeuBase Therapeutics, MAY 12, 2022, View Source [SID1234614371]).

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"We are pleased with the progress being made across our development pipeline of therapeutic programs to treat DM1, HD, and KRAS-driven cancers. The new data we’ve announced to date this year have further validated the use of our PATrOL platform to design novel genetic medicines that target and rescue gene dysfunctions, with the potential for clinically impactful outcomes in both rare and common diseases," said Dietrich A. Stephan, Ph.D., Founder, Chief Executive Officer, and Chairman of NeuBase. "We continue to execute the development strategy for our DM1 program, which includes a series of IND-enabling studies scheduled to report data throughout CY2022. Last quarter, we presented pharmacodynamic data that illustrated a single intravenous (IV) dose or multiple subcutaneous (SC) doses of our DM1 development candidate resolves the genetic defect and myotonia in skeletal muscle of the gold-standard mouse model of the disease. Building off these results, we plan on announcing at ASGCT (Free ASGCT Whitepaper) additional pharmacokinetic (PK) data, which will illustrate the exposure levels of our development candidate when administered via systemic administration in skeletal muscles, heart, and brain, tissues that are affected in DM1. We expect these data to support further advancement of our lead candidate for DM1 and validate a differentiated whole-body solution for this disease. Considering this progress, we believe the submission of an IND application to the FDA is on track for the fourth quarter of CY2022."

Second 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 IV and SC routes of administration, exploratory and IND-enabling Good Laboratory Practice (GLP) toxicology, and mechanism of action studies. In addition, Good Manufacturing Practice (GMP) of NeuBase’s development candidate to support Phase 1/2 clinical trials has been successfully implemented via contract manufacturing organizations.
In March 2022, the Company presented a robust data package through posters and presentations at the 2022 MDA Clinical & Scientific Conference demonstrating that systemic administration of the Company’s lead DM1 candidate, NT-0231.F, in the HSALR model achieves clinically relevant molecular and functional rescue, including genetic target engagement, displacement of sequestered MBNL1, resolution of nuclear aggregates, rescue of the spliceopathy, and reversal of myotonia (delayed muscle relaxation after contraction). In PK studies of NT-0231.F in wild-type BALB/C mice, a single IV or SC dose showed high volume of distribution, suggesting wide tissue distribution.
The Company announced that two abstracts have been accepted for presentation at the ASGCT (Free ASGCT Whitepaper) 25th Annual Meeting. The presentations will include new preclinical data on the biodistribution in key tissues of NT-0231.F.
With continued positive results from in vitro and in vivo preclinical studies, the Company expects to file an IND application for NT-0231.F in the fourth quarter of CY2022.
Huntington’s Disease (HD) Program: The HD program is currently in preclinical development. In CY2022, NeuBase expects to present new preclinical data describing the pharmacology of a candidate compound in the brain after systemic administration, nominate a development candidate and initiate scale-up and toxicology activities.
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). Existing in vivo data show activity illustrating allele-selective engagement of mutant KRAS at the DNA and RNA levels, with abrogation of downstream hyperactive signaling through multiple RAS pathway members, resulting in anti-tumor activity.
Financial Results for the Second Fiscal Quarter Ended March 31, 2022

As of March 31, 2022, the Company had cash and cash equivalents of approximately $39.0 million, compared with approximately $52.9 million as of September 30, 2021.
For the fiscal quarter ended March 31, 2022, the Company reported a net loss of approximately $9.9 million, or a net loss of $0.30 per share, compared with a net loss of approximately $5.5 million, or a net loss of $0.24 per share, for the same period last year.
For the fiscal quarter ended March 31, 2022, total operating expenses were approximately $9.9 million, consisting of approximately $3.1 million in general and administrative expenses and $6.8 million of research and development expenses. This compares with total operating expenses of approximately $5.9 million for the same period last year, consisting of approximately $2.7 million in general and administrative expenses and $3.2 million in research and development expenses.
Financial Results for the Six-Month Period Ended March 31, 2022

For the six-month period ended March 31, 2022, the Company reported a net loss of approximately $17.7 million, or a net loss of $0.54 per share, compared with a net loss of approximately $9.6 million, or a net loss of $0.41 per share, for the same period last year.
For the six-month period ended March 31, 2022, total operating expenses were approximately $17.2 million, consisting of approximately $6.0 million in general and administrative expenses and $11.2 million of research and development expenses. This compares with total operating expenses of approximately $10.6 million for the same period last year, consisting of approximately $5.4 million in general and administrative expenses and $5.2 million in research and development expenses.