On February 14, 2023 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, 2022, and other recent developments (Press release, NeuBase Therapeutics, FEB 14, 2023, View Source [SID1234627177]).
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"We plan on leveraging our PATrOL platform to perform ‘nuclease free’ in vivo gene editing to restore healthy gene function. This technology complements the field of CRISPR/Cas editors, base editors, and prime editors, with the potential to address the majority of disease-causing mutations. We believe the high fidelity and lack of immunogenicity of our editing approach offer the possibility to address tissue turnover by redosing. Throughout calendar year 2023, we anticipate sharing data on ex vivo and in vivo editing results against high-value genetic mutations, together with associated performance metrics, such as fidelity and efficiency. In addition to focusing on our internal programs, which we plan to announce in more detail over the coming months, we recently announced a research agreement with a global healthcare company to evaluate editing against three monogenic genetic disease-causing genes. Since announcing this initial agreement, we have held additional discussions with other leading healthcare companies on potential collaborations. This is truly an exciting time at NeuBase and we look forward to keeping you apprised of our progress," stated Dietrich A. Stephan, Ph.D., Founder and Chief Executive Officer of NeuBase.
"As previously announced, we are actively pursuing collaborative initiatives, including partnerships, for our gene silencing programs in myotonic dystrophy type 1 (DM1), Huntington’s disease (HD) and cancers driven by common KRAS gene mutations. We believe this is the best approach for these programs to keep building momentum as they move into the clinic and beyond," concluded Dr. Stephan.
First Quarter of Fiscal Year 2023 and Recent Operating Highlights
Gene Editing Program:
The Company is advancing development of the differentiated gene editing capabilities of its PATrOL platform, including identifying and evaluating multiple indications for possible future development.
Details of the gene editing pipeline expected to be provided during calendar year 2023.
Gene Editing Research Agreements:
Announced a research agreement with a global healthcare company to evaluate the PATrOL platform for three monogenic genetic diseases and collaborate with NeuBase on the evaluation of drug candidates for three undisclosed indications. The global healthcare company will have the exclusive opportunity, subject to certain terms and conditions, to license and develop the drug candidates created under this research evaluation agreement.
Engaged in discussions with other healthcare companies on potential for additional research agreements.
Gene Silencing Pipeline Collaborations:
Actively pursuing collaborative initiatives, including partnerships, for the Company’s DM1, HD, and KRAS (G12D and G12V) programs, which are expected to support future development of these programs.
Financial Results for the Fiscal Quarter Ended December 31, 2022
As of December 31, 2022, the Company had cash and cash equivalents of approximately $17.4 million, compared with approximately $23.2 million as of September 30, 2022.
NeuBase estimates its current cash and cash equivalents are sufficient to fund currently planned operating and capital expenditures into the second quarter of calendar year 2024.
For the fiscal quarter ended December 31, 2022, the Company reported a net loss of approximately $4.4 million, or a net loss of $0.13 per share, compared with a net loss of approximately $7.7 million, or a net loss of $0.24 per share, for the same period last year.
For the fiscal quarter ended December 31, 2022, total operating expenses were approximately $4.6 million, consisting of approximately $2.6 million in general and administrative expenses, $1.3 million in research and development expenses, and $0.7 million in restructuring costs. This compares with total operating expenses of approximately $7.3 million for the same period last year, consisting of approximately $2.9 million in general and administrative expenses and $4.4 million in research and development expenses.