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NeuBase Therapeutics, Inc. (NBSE)

NasdaqCM - NasdaqCM Real-time price. Currency in USD
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7.19-0.26 (-3.49%)
At close: 4:00PM EST
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Previous close7.45
Open7.46
Bid7.10 x 1100
Ask7.18 x 1000
Day's range7.08 - 7.64
52-week range4.52 - 11.78
Volume174,886
Avg. volume199,982
Market cap166.647M
Beta (5Y monthly)N/A
PE ratio (TTM)N/A
EPS (TTM)-3.26
Earnings date24 Mar 2021 - 29 Mar 2021
Forward dividend & yieldN/A (N/A)
Ex-dividend dateN/A
1y target est15.67
  • NeuBase Therapeutics to Present at the H.C. Wainwright Virtual BioConnect Conference
    GlobeNewswire

    NeuBase Therapeutics to Present at the H.C. Wainwright Virtual BioConnect Conference

    PITTSBURGH, Jan. 11, 2021 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (NASDAQ: NBSE) (“NeuBase” or the “Company”), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, announced today that Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, will present a corporate overview during the H.C. Wainwright Virtual BioConnect Conference, which is being held from January 11 - 14, 2021. A webcast of the on-demand presentation will be available beginning today at 6:00 a.m. Eastern Time and archived on the NeuBase website, www.neubasetherapeutics.com.   About NeuBase Therapeutics NeuBase is accelerating the genetic revolution using a new class of synthetic medicines. 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. To learn more, visit www.neubasetherapeutics.com.NeuBase Investor Contact: Dan Ferry Managing Director LifeSci Advisors, LLC Daniel@lifesciadvisors.com OP: (617) 430-7576NeuBase Media Contact: Cait Williamson, Ph.D. LifeSci Public Relations cait@lifescipublicrelations.com OP: (646) 751-4366

  • NeuBase Therapeutics Reports Business Update and Financial Results for Fiscal Year 2020
    GlobeNewswire

    NeuBase Therapeutics Reports Business Update and Financial Results for Fiscal Year 2020

    Data presented throughout 2020 have validated the potential of the PATrOL™ platform to develop highly targeted therapies that increase, decrease or change causal protein function Plan to provide updates on development pipeline, including the myotonic dystrophy type 1 (DM1) and Huntington’s disease (HD) programs, at an R&D day in the first half of CY2021PITTSBURGH, Pa., Dec. 23, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) (“NeuBase” or the “Company”), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, today reported its financial results for the fiscal year ended September 30, 2020.“Throughout 2020, we successfully executed against our development strategy, most notably with the generation of two very exciting datasets supporting our initial therapeutic pipeline, including our DM1 and Huntington’s disease programs. These initial data are a clear validation of our transformative platform and provide a strong foundation on which to build our IND-enabling studies for both programs,” said Dietrich A. Stephan, Ph.D., chief executive officer of NeuBase. “In addition, these data support the broad potential of our PATrOL™ platform as a viable synthetic approach to genetic medicine. We believe the platform’s unique capabilities, which include increasing, decreasing and changing protein function, have the potential to redefine treatment for a multitude of patients suffering from both common and rare genetic conditions with insufficient or no therapeutic options.”“As we plan for the future, we’ve expanded our team with several key hires in the second half of the year, including the appointments of Drs. Curt Bradshaw and William Mann as chief scientific officer and chief operating officer, respectively. The expanded capabilities across our entire clinical team are expected to be a positive driver of our development activities as we enter the new year, and advance and expand our pipeline. In order to support this larger team, we recently signed a lease for a new headquarters which will offer more space and state-of-the-art lab facilities to support the rapid development of our pipeline. We look forward to providing greater insight into these activities during an investor R&D day expected to take place in the first half of CY2021,” continued Dr. Stephan.Fourth Fiscal Quarter of 2020 and Recent Operating Highlights * Announced positive preclinical in vitro and in vivo data for PATrOL-enabled anti-gene for the treatment of myotonic dystrophy type 1 (DM1), which further validate the potential of the Company’s proprietary platform to develop highly targeted genetic therapies * Appointed Curt Bradshaw, Ph.D., seasoned industry veteran and former chief scientific officer at Arrowhead Pharmaceuticals, as the new chief scientific officer of NeuBase * Further strengthened the management team with the appointment of William Mann, Ph.D., MBA, an experienced executive with a track record that spans the biopharma life cycle, as chief operating officer * Expanded the Company’s Scientific Advisory Board (SAB) with the appointments of Peter Nielsen, Ph.D., inventor of peptide nucleic acid technology, Eriks Rozners, Ph.D., an expert in alternative binding modes of peptide nucleic acids, and Randy Davis, MBA, a leader in biotech development, which complement the SAB’s existing team of renowned experts * Signed a lease for a new headquarters with office and lab space in Pittsburgh that will offer more space to support the Company’s expanding development activities around its rapidly advancing pipeline of PATrOL-enabled therapies Financial Results for the Fiscal Year Ended September 30, 2020: * At September 30, 2020, the Company had cash and cash equivalents of approximately $32.0 million, compared with cash and cash equivalents of approximately $10.3 million at September 30, 2019. 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 fiscal year ended September 30, 2020, the Company reported a net loss of approximately $17.4 million, or a net loss of $0.89 per share, compared with a net loss of approximately $26.1 million, or a net loss of $3.16 per share, for the fiscal year ended September 30, 2019; and * For the fiscal year ended September 30, 2020, total operating expenses were approximately $17.1 million, consisting of approximately $10.1 million in general and administrative expenses and $6.9 million of research and development expenses. This compares with total operating expenses of $25.5 million for the fiscal year ended September 30, 2019, which was comprised of approximately $9.1 million in general and administrative expenses, $3.4 million in research and development expenses, and $13.0 million in research and development-license acquired expenses. About NeuBase Therapeutics NeuBase is accelerating the genetic revolution using a new class of synthetic medicines. 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. To learn more, visit www.neubasetherapeutics.com.Use of Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the potential significance and implications of the Company’s positive in vitro and in vivo preclinical data for its PATrOL™-enabled anti-gene therapies for the treatment of myotonic dystrophy and our plan to provide updates on development pipeline, including the myotonic dystrophy type 1 (DM1) and Huntington’s disease (HD) programs, at an R&D day in the first half of CY2021. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the risks that prior data will not be replicated in future studies; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.NeuBase Investor Contact: Dan Ferry Managing Director LifeSci Advisors, LLC daniel@lifesciadvisors.com OP: (617) 430-7576NeuBase Media Contact: Cait Williamson, Ph.D. LifeSci Communications cait@lifescicomms.com OP: (646) 751-4366  NeuBase Therapeutics, Inc. and Subsidiaries Consolidated Balance Sheets           September 30,    2020  2019  ASSETS         CURRENT ASSETS         Cash and cash equivalents $31,992,283  $10,313,966  Prepaid insurance  521,617   449,583  Other prepaid expenses and current assets  294,640   265,686  Total Current Assets  32,808,540   11,029,235            EQUIPMENT, net  1,166,934   430,995            OTHER ASSETS         Intangible assets, net  -   145,833  Investment  323,557   586,418  Long-term prepaid insurance  145,250   338,916  Total Other Assets  468,807   1,071,167            TOTAL ASSETS $34,444,281  $12,531,397            LIABILITIES AND STOCKHOLDERS’ EQUITY         CURRENT LIABILITIES         Accounts payable $1,505,042  $1,477,152  Accrued expenses and other current liabilities  555,883   405,599  Warrant liabilities  950,151   496,343  Insurance note payable  138,557   122,919  Total Liabilities  3,149,633   2,502,013  COMMITMENTS AND CONTINGENCIES (Note 16)                   STOCKHOLDERS’ EQUITY         Preferred stock, $0.0001 par value; 10,000,000 shares authorized; no shares issued and outstanding as of September 30, 2020 and 2019  -   -  Common stock, $0.0001 par value; 250,000,000 shares authorized; 23,154,084 and 17,077,873 shares issued and outstanding as of September 30, 2020 and 2019, respectively  2,315   1,708  Additional paid-in capital  74,850,935   36,201,758  Accumulated deficit  (43,558,602)  (26,174,082) Total stockholders’ equity  31,294,648   10,029,384            TOTAL LIABILITIES AND STOCKHOLDERS’ EQUITY $34,444,281  $12,531,397    NeuBase Therapeutics, Inc. and Subsidiaries Consolidated Statements of Operations     Year Ended September 30,    2020  2019  OPERATING EXPENSES         General and administrative $10,123,298  $9,095,674  Research and development  6,946,008   3,447,201  Research and development expense- license acquired  -   12,967,415  TOTAL OPERATING EXPENSES  17,069,306   25,510,290            LOSS FROM OPERATIONS  (17,069,306)  (25,510,290)           OTHER INCOME (EXPENSE)         Interest expense  (7,686)  (128,951) Change in fair value of warrant liabilities  (453,808)  (492,889) Loss on disposal of fixed asset  (3,230)  -  Equity in losses on equity method investment  (262,861)  -  Other income  412,371   -  Total other income (expenses), net  (315,214)  (621,840)           NET LOSS $(17,384,520) $(26,132,130)           BASIC AND DILUTED LOSS PER SHARE $(0.89) $(3.16)           WEIGHTED AVERAGE SHARES OUTSTANDING:                   BASIC AND DILUTED  19,620,291   8,271,707

  • NeuBase Therapeutics Announces Positive Preclinical In Vivo Data for PATrOL™-enabled Anti-gene for the Treatment of Myotonic Dystrophy Type 1
    GlobeNewswire

    NeuBase Therapeutics Announces Positive Preclinical In Vivo Data for PATrOL™-enabled Anti-gene for the Treatment of Myotonic Dystrophy Type 1

    In vivo data after single-dose IV administration demonstrate engagement with DMPK mRNA and broad rescue of mis-splicing across key transcripts Findings provide support for hypothesized mechanism of action of anti-gene, which is designed to not degrade the DMPK transcriptData further validate the potential of the PATrOL™ platform to develop highly targeted therapies that increase, decrease or change causal protein functionNeuBase management to hold conference call and webcast today, December 16, at 8:00 a.m. ESTPITTSBURGH, Pa., Dec. 16, 2020 (GLOBE NEWSWIRE) -- NeuBase Therapeutics, Inc. (Nasdaq: NBSE) ("NeuBase" or the "Company"), a biotechnology company accelerating the genetic revolution using a new class of synthetic medicines, today announced positive in vitro and in vivo preclinical data for its PATrOL™-enabled anti-gene therapies for the treatment of myotonic dystrophy type 1 (DM1). These new data show that PATrOL-enabled Compound A can rapidly resolve mis-splicing without negatively impacting DMPK protein levels. They also support the potential of NeuBase’s anti-gene approach to comprehensively treat the underlying cause of DM1.“Despite the fact that the genetic basis of DM1 is well understood today, there is still an urgent need to find the first genetically-targeted, disease-modifying treatment option for affected patients,” said Curt Bradshaw, Ph.D., Chief Scientific Officer of NeuBase. “DM1 is caused by a genetic mutation in the DMPK gene leading to mis-splicing of a broad spectrum of genes and DMPK protein insufficiency. A treatment option that addresses mis-splicing while retaining functional DMPK protein levels may be key to treating all aspects of DM1.”Dietrich A. Stephan, Ph.D., Chief Executive Officer of NeuBase, added, “Using our proprietary PATrOL platform, we have designed a first-in-class anti-gene candidate that selectively binds mutant DMPK mRNA and opens its hairpin secondary structure, as opposed to a mechanism of action that explicitly degrades the mutant and wild-type transcripts indiscriminately, making it a unique option for the treatment of DM1. These in vitro and in vivo data both support our hypothesized mechanism of action and demonstrate rapid and broad resolution of the mis-splicing that is the primary cause of DM1.“This is the second set of positive data that we’ve announced in 2020 for our PATrOL-enabled therapies, which we believe serves as proof of concept that further validates our technologic foundation. With a single unified platform, we believe we can increase, decrease or change protein function of potentially any nucleic acid target, unique among genetic medicine approaches. We are excited by the progress we have made and look forward to providing additional updates on our platform and pipeline of programs at an R&D day in the first half of 2021.”In vitro data highlights in DM1 patient-derived fibroblasts:•Compound A traffics to the nucleus, engages and normalizes DMPK mRNA.   •Compound A rescues mis-splicing of two key DM1 dysregulated transcripts (MBNL1 and MBNL2) within two days after initial treatment. Notably, induction of rescue continues to improve through day 9, the latest time point analyzed.   •Compound A significantly induces broad correction of global exon inclusion levels of mis-spliced transcripts.     ºStatistically significant improvement in global splicing as measured by the human differential splice inclusion (hDSI) statistic.   ºMore than 175 dysregulated human transcripts achieved statistically significant improvement in splicing, many with completely normalized exon usage. •DMPK protein levels remain unchanged 5 days after a single Compound A dose, supporting the hypothesized mechanism of action maintaining DMPK.   In vivo data highlights in the HSALR transgenic mouse model of DM1 that expresses high levels of mutant CUG-repeat-containing mRNA (HSA) in skeletal muscle: •A single intravenous (IV) injection of 29 mg/kg of Compound A traffics to the nucleus and engages HSA mRNA within 24 hours in tibialis anterior (TA) skeletal muscle. •A single intravenous (IV) injection of Compound A significantly induces broad correction of global exon inclusion levels of mis-spliced transcripts in HSALR TA skeletal muscle at day 13. •Statistically significant improvement in global splicing as measured by the murine differential splice inclusion (mDSI) statistic.   ºMore than 50 unique dysregulated murine transcripts achieved statistically significant improvement in splicing post-treatment, with many achieving complete normalization of appropriate exon usage. •Compound A was well tolerated after single dose administration at the dose demonstrating activity in vivo. DM1 is a rare, autosomal dominant repeat expansion disorder characterized by progressive muscle wasting and weakness. It also affects the central nervous system (CNS) and heart. DM1 is caused by CTG nucleic acid repeats in the DMPK gene that produce a hairpin structure in the transcribed DMPK mRNA. The hairpin structure sequesters critical splice regulators and results in the mis-splicing of multiple gene transcripts. Furthermore, the binding of splice regulators traps the mutant DMPK mRNA in the nucleus, resulting in DMPK protein haploinsufficiency, or half the level of protein that is needed for normal function, which is thought to exacerbate the CNS and cardiac symptoms that are characteristic of DM1 (as knock-out mice for Dmpk show both severe cardiac conduction defects as well as issues with neuronal cytoskeletal remodeling manifesting in aberrant long-term potentiation). The prevalence of DM1 is >5/100,000 in the general population. There are currently no approved treatments for DM1. Conference Call and Webcast Details NeuBase Therapeutics, Inc. will discuss these data during a webcasted conference call with slides today, December 16, 2020, at 8:00 a.m. EST. To access the webcast, please click here. An archived recording of this presentation will be available following the call through the IR Calendar page on the Investors section of the Company’s website, www.neubasetherapeutics.com.About NeuBase Therapeutics, Inc. NeuBase is accelerating the genetic revolution using a new class of synthetic medicines. 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. To learn more, visit www.neubasetherapeutics.com.Use of Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act. These forward-looking statements are distinguished by use of words such as "will," "would," "anticipate," "expect," "believe," "designed," "plan," or "intend," the negative of these terms, and similar references to future periods. These forward-looking statements include, among others, those related to the potential significance and implications of the Company’s positive in vitro and in vivo preclinical data for its PATrOL™-enabled anti-gene therapies for the treatment of myotonic dystrophy. These views involve risks and uncertainties that are difficult to predict and, accordingly, our actual results may differ materially from the results discussed in our forward-looking statements. Our forward-looking statements contained herein speak only as of the date of this press release. Factors or events that we cannot predict, including those risk factors contained in our filings with the U.S. Securities and Exchange Commission, may cause our actual results to differ from those expressed in forward-looking statements. The Company may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements, and you should not place undue reliance on these forward-looking statements. Because such statements deal with future events and are based on the Company's current expectations, they are subject to various risks and uncertainties, and actual results, performance or achievements of the Company could differ materially from those described in or implied by the statements in this press release, including: the Company's plans to develop and commercialize its product candidates; the timing of initiation of the Company's planned clinical trials; the risks that prior data will not be replicated in future studies; the timing of any planned investigational new drug application or new drug application; the Company's plans to research, develop and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of the Company's product candidates; the Company's commercialization, marketing and manufacturing capabilities and strategy; global health conditions, including the impact of COVID-19; the Company's ability to protect its intellectual property position; and the requirement for additional capital to continue to advance these product candidates, which may not be available on favorable terms or at all, as well as those risk factors contained in our filings with the U.S. Securities and Exchange Commission. Except as otherwise required by law, the Company disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.NeuBase Investor Contact: Dan Ferry Managing Director LifeSci Advisors, LLC daniel@lifesciadvisors.com OP: (617) 430-7576NeuBase Media Contact: Cait Williamson, Ph.D. LifeSci Communications cait@lifescicomms.com OP: (646) 751-4366