On May 14, 2020 NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company developing next-generation antisense oligonucleotide (ASO) therapies using its scalable PATrOL platform to address genetic diseases, reported its financial results for the three and six month periods ended March 31, 2020 (Press release, NeuBase Therapeutics, MAY 14, 2020, View Source [SID1234558036]).
Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:
Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing
Schedule Your 30 min Free Demo!
"We have generated a solid foundation of data that we expect will enable us to produce mutation-specific genetic medicines for a multitude of diseases. The positive non-human primate pharmacokinetic (biodistribution) and patient-derived cell line pharmacodynamic (activity) data released on March 31st illustrate our ability to create a new class of mutation silencing therapies. In addition, our proprietary targeting technology enables our PATrOL compounds to achieve therapeutically relevant drug concentrations throughout the body, including in the brain," said Dietrich A. Stephan, Ph.D., chief executive officer of NeuBase.
"Our therapeutic development programs continue to drive forward, despite the difficult macroenvironment, as evidenced by the recent positive platform validation data and well-received public offering. As we look ahead, our initial focus is to continue advancing our Huntington’s disease ("HD") and myotonic dystrophy type 1 ("DM1") programs through lead optimization and IND-enabling studies. We expect to announce the lead candidate for the NT0100 Program for HD by the end of calendar year 2020, followed by the initiation of IND-enabling studies during the first half of calendar year 2021. Finally, our unique biodistribution profile allows us the opportunity to develop therapies against targets and organ systems that we believe other antisense companies cannot currently reach in the body, including into the brain after systemic delivery, which is one of the grand challenges in drug delivery of macromolecules. We believe this unique ability validates our large opportunity in the antisense space," continued Dr. Stephan.
Second Fiscal Quarter of 2020 and Recent Operating Highlights
·Announced positive, preclinical pharmacokinetic ("PK") and pharmacodynamic ("PD") data validating the first-in-class genetic therapy PATrOL platform:
oPK studies of the PATrOL-enabled compound in non-human primates (NHPs) demonstrated, among other things, rapid uptake of the compound out of the body’s circulation after systemic intravenous administration, penetration by the compound into every organ and tissue system studied, and retention of therapeutically relevant doses for greater than one week after single-dose injection;
oPD studies in patient-derived cell lines demonstrated, among other things, activity in engaging target disease-causing transcripts and knocking-down resultant malfunctioning mutant protein levels preferentially over normal protein knock-down; and dose-limiting toxicities were not observed relative to a control either at or above the doses demonstrating activity in human cells in vitro; and
oPATrOL enabled compounds were generally well-tolerated in vivo after systemic administration, both after single dose administration in NHPs and multi dose administration in mice for over a month.
Financial Results for the Fiscal Quarter Ended March 31, 2020:
·At March 31, 2020, the Company had cash and cash equivalents of approximately $5.8 million, compared with cash and cash equivalents of approximately $10.3 million at September 30, 2019. Subsequent to the end of the fiscal second quarter of 2020, the Company completed a public equity offering that generated net proceeds of approximately $33.3 million. The Company believes that its current cash balance will provide sufficient capital to fund operations into the second calendar quarter of 2022;
·For the three month period ended March 31, 2020, the Company reported a net loss of approximately $4.4 million, or a net loss of $0.26 per share, compared with a net loss of approximately $2.0 million, or a net loss of $0.33 per share, for the same period last year; and
·For the three month period ended March 31, 2020, total operating expenses were approximately $4.4 million, consisting of approximately $2.8 million in general and administrative expenses and $1.6 million of research and development expenses. This compares with total operating expenses of $1.9 million for the same period last year, which was comprised of approximately $1.9 million in general and administrative expenses and $0.03 million in research and development expenses.
Financial Results for the Six Month Period Ended March 31, 2020:
·For the six month period ended March 31, 2020, the Company reported a net loss of approximately $8.9 million, or a net loss of $0.52 per share, compared with a net loss of approximately $3.5 million, or a net loss of $0.59 per share, for the same period last year; and
·For the six month period ended March 31, 2020, total operating expenses were approximately $8.1 million, consisting of approximately $5.3 million in general and administrative expenses and $2.8 million of research and development expenses. This compares with total operating expenses of $3.4 million for the same period last year, which was comprised of approximately $2.3 million in general and administrative expenses and $1.1 million in research and development and research and development- license acquired expenses.