Pittsburgh-based NeuBase Therapeutics (NASDAQ: NBSE) is a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines which have been shown to be able to increase, decrease and change gene function, as appropriate, to resolve causal genetic defects in living systems. NeuBase’s designer PATrOL™ therapies are centered around its proprietary drug scaffold to address genetic diseases at the source by combining the highly targeted approach of traditional genetic therapies with the broad organ distribution capabilities of small molecules. With an initial focus on silencing disease-causing mutations in debilitating neurological, neuromuscular and oncologic disorders, NeuBase is committed to redefining medicine for the millions of patients with both common and rare conditions.
This week, Neubase reported its financial results for the three-month period ended December 31, 2020.
“In 2020 we validated that our PATrOL™ platform technology can deliver compounds that are broadly biodistributed, mutant allele-specific and well tolerated, including in non-human primates (NHPs). Thereafter we finalized screening compound libraries and moved into in vivo efficacy and tolerability studies that demonstrated that administration of a PATrOL™-enabled compound resolves the causal genetic insult in an established transgenic animal model of myotonic dystrophy type 1 (DM1), a severe genetic disease with no effective therapies. This momentum is being carried forward into 2021 as we set the stage to enter the clinic in CY2022,” said Dietrich A. Stephan, Ph.D., chief executive officer of NeuBase. “Predicated on our progress, we recently announced an agreement to acquire additional gene modulating technology to consolidate the intellectual property to protect and enhance value creation with this unique therapeutic modality. We look forward to providing more data and insights during an investor R&D day in the first half of CY2021, including updates on platform innovations, continued progress in Huntington’s disease (HD) and DM1 and new pipeline programs. This event will provide an opportunity for us to introduce the expanded team, including Dr. Curt Bradshaw, Ph.D. chief scientific officer, who has overseen several development programs into the clinic and complements a world-class team of technical experts and drug developers.” stated Dietrich A. Stephan, Ph.D., chief executive officer of NeuBase.
First Quarter of Fiscal Year 2021 and Recent Operating Highlights
- Announced positive preclinical in vitro and in vivo data for PATrOL™-enabled anti-gene for the treatment of DM1, which further validate the Company’s core technological foundation as well as the potential of its proprietary approach to comprehensively treat the underlying cause of DM1 and many other genetically driven diseases
- Further strengthened the management team with a key appointment to the role of chief scientific officer and the addition of the original inventor of peptide nucleic acid chemistry to the Scientific Advisory Board
- Entered a binding agreement to acquire gene modulating technology to consolidate and expand the capabilities within our technology platform which we expect to close in Q1 CY2021
- Initiated the buildout of a new headquarters with office and lab space that will support and enable the Company’s expanding development activities around its rapidly advancing pipeline of PATrOL™-enabled therapies
Financial Results for the Fiscal Quarter Ended December 31, 2020
- At December 31, 2020, the Company had cash and cash equivalents of approximately $28.0 million, compared with cash and cash equivalents of approximately $32.0 million at September 30, 2020. NeuBase estimates its cash and cash equivalents are sufficient to fund the currently planned operating and capital expenditures into the first quarter of CY2022
- For the three-month period ended December 31, 2020, the Company reported a net loss of approximately $4.1 million, or a net loss of $0.18 per share, compared with a net loss of approximately $4.5 million, or a net loss of $0.26 per share, for the three-month period ended December 31, 2019
- For the three-month period ended December 31, 2020, total operating expenses were approximately $4.7 million, consisting of approximately $2.6 million in general and administrative expenses and $2.0 million of research and development expenses. This compares with total operating expenses of $3.8 million for the three-month period ended December 31, 2019, which was comprised of approximately $2.6 million in general and administrative expenses and $1.2 million in research and development expenses.
In December, I hosted a FREE Presentation & Q&A Event for our sister organization Tribe Public with the CEO & Founder of NeuBase Therapeutics, Dietrich Stephan, Ph.D., who owns ~25% of NeuBase’s total shares. He is an industry veteran who is considered one of the fathers of the field of precision medicine. The presentation is titled “Accelerating the Genetic Revolution with a New Class of Synthetic Medicines.” You can view the recording at the Tribe Public Channel.