Disclosures Regarding Forward-Looking Statements
This report includes "forward-looking statements" within the meaning of Section 21E of the Exchange Act. Those statements include statements regarding the intent, belief or current expectations of the Company and its subsidiaries and our management team. Any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, and actual results may differ materially from those projected in the forward-looking statements. These risks and uncertainties include but are not limited to those risks and uncertainties set forth in Item 1A of this Annual Report on Form 10-K. In light of the significant risks and uncertainties inherent in the forward-looking statements included in this Annual Report on Form 10-K, the inclusion of such statements should not be regarded as a representation by us or any other person that our objectives and plans will be achieved. Further, these forward-looking statements reflect our view only as of the date of this report. Except as required by law, we undertake no obligations to update any forward-looking statements and we disclaim any intent to update forward-looking statements after the date of this report to reflect subsequent developments. Accordingly, you should also carefully consider the factors set forth in other reports or documents that we file from time to time with theSEC . 65 Overview As a result of the Merger, our going-forward operations will be primarily those of Legacy NeuBase. Accordingly, the results of operations reported for the fiscal years endedSeptember 30, 2019 and 2018, in this Management's Discussion and Analysis are not indicative of the results of operations expected for future years due to the transition of our historic business operations to primarily those of Legacy NeuBase. We are a biotechnology company focused on developing next generation therapies to treat rare genetic diseases and cancer caused by mutant genes. Given that perhaps every human disease has a genetic component, we believe that our differentiated platform technology has the potential for broad impact. Mutated proteins resulting from errors in deoxyribonucleic acid ("DNA") sequences cause many rare genetic diseases and cancer. DNA in each cell of the body is transcribed into pre-RNA, which is then processed (spliced) into mRNA which is exported into the cytoplasm of the cell and translated into protein. This is termed the "central dogma" of biology. Therefore, when errors in a DNA sequence occur, they are propagated to RNAs and can become a damaging protein. The type of therapies that we are developing are termed ASO therapies. ASOs are short single strands of nucleic acids (traditionally thought of as single stranded RNA molecules) which will bind to defective RNA targets in cells and inhibit their ability to be translated into defective proteins. We believe we are a leader in the discovery and development of the class of RNA-targeted ASO drugs called PNAs. Our proprietary PATrOL™ platform allows for a more efficient discovery of drug product candidates, potentially transforming the treatment paradigm for people affected by rare genetic diseases and cancer. The PATrOL™ platform allows for a potentially more efficient discovery of drug product candidates because of manufacturing consistency and because we are not constrained by folded regions of the target RNA molecule (secondary structures). The peptide backbone of our ASOs is rigid, and once linked together to form a series of backbone subunits, forms a single pre-organized structure. At a more detailed level, each subunit of the peptide backbone has only a single chiral center - a point in the chemical structure where the conformation of the backbone could fluctuate - and this chiral center is locked into one conformation and thus pre-organized to form only a single conformation or stereoisomer. A stereoisomer is a term used in the ASO therapeutics field to mean a string of backbone subunits (usually sugars or modified sugars) with nuclear bases attached that are linked together into a specific sequence that matches (complements) the target sequence, but because of the nature of the backbone subunits used, the drug assumes various conformations often with varying affinity for the target sequence. These stereoisomers often require a manufacturing step to purify the heterogeneous mixture of conformations into a more homogenous mixture or even a single conformation of the drug in order to obtain the hoped-for therapeutic effect. Our PNAs assume only a single conformation with any constellation of nuclear bases added to the backbone or any oligomer length. This backbone also has a neutral charge, as opposed to the negatively charged backbones of DNA and RNA. This neutral charge allows our ASO to open up RNAs which are folded upon themselves and bind to their target sequence. This potentially accelerates identification of drug candidates which have the desired activity. In addition to the backbone conformational purity which allows for a more efficient discovery of drug product candidates, we also have a kit of proprietary bi-facial or bi-specific nucleotides (traditional nucleotides only have a single binding face and thus are restricted to only binding single-stranded RNA targets) which can be used in any combination to access RNA secondary structures (double stranded RNA targets which are folded upon themselves) such as hairpins. This allows us to potentially access regions of the target transcript which may be unique in secondary structure to allow enhanced selectivity for the target (mutant) RNA vs. the normal RNA. Enhanced selectivity for mutant RNAs vs. normal RNAs is critical as normal RNAs are likely required for effective functioning of the cell. These bi-specific nucleotides can also target genomic loci and microRNAs in their double-stranded form.
In addition to the backbone and modified nuclear bases, the platform toolkit also includes linker technology which, when added to both ends of the PNAs, allow cooperative binding between individual drug molecules once they are engaged with the target RNA to form longer and more tightly bound drugs.
The final component of the platform is a chemical moiety, which is used to decorate the peptide backbone in a proprietary manner and allows the PNAs to penetrate cell membranes and distribute throughout the body when administered systemically. 66
This relatively simple toolkit of components forms the PATrOL™ platform and allows us to manufacture genome and transcript-specific PNAs quickly for screening.
We are currently focused on therapeutic areas in which we believe our drugs will provide the greatest benefit with a significant market opportunity and intend to utilize our technology to build out a pipeline of custom designed therapeutics for additional high-value disease targets. We are developing several preclinical programs using our PATrOL™ platform, including: NT0100, targeted at HD, a repeat expansion disorder, and NT0200, targeted at myotonic dystrophy ("DM1"). Preclinical studies are being conducted to evaluate the PATrOL™ platform technology and program candidates in the areas of pharmacokinetics and pharmacodynamics, and we expect to report results from those studies beginning in the first calendar quarter of 2020 and in the second calendar quarter of 2020. In addition, the emerging pipeline of other assets that target primary and secondary RNA structure and genomic DNA allows a unique market advantage across a variety of rare diseases and oncology targets. Using our PATrOL™ platform, we can create ASOs that have distinct potential advantages over other chemical entities currently in the market or in development for gene silencing applications. These advantages include, among others: a backbone that has only one chiral center and thus forms only one stereoisomer; the ability of the PNA backbone to intercalate, open up secondary (RNA folded upon itself) and tertiary structures (RNA molecules that interact with other RNA molecules in the cell) and bind within these double-stranded RNA in a highly selective manner; a proprietary set of engineered nuclear bases that increase selectivity to specific target sequences including secondary and tertiary structures that has been licensed exclusively from CMU; technology to allow self-assembly of our small gamma-PNA at the RNA target to increase selectivity which has been licensed exclusively from CMU; the ability to modulate cell permeability and the ability to pass the blood-brain barrier when administered systemically; the lack of innate or acquired immune responses of similar gamma-PNAs in preclinical models; and potential minimal toxicity based on previous in-vivo studies in rodent models. With these advantages, our PATrOL™ platform-enabled therapies can potentially address a multitude of rare genetic diseases and cancer, among other indications. Merger OnJuly 12, 2019 , Ohr completed the Merger. At the closing of the Merger, each outstanding share of Legacy NeuBase's capital stock was converted into the right to receive 1.019055643 shares of our common stock. Shares of our common stock commenced trading on the Nasdaq Capital Market under the ticker symbol "NBSE" as of market open onJuly 15, 2019 . Our previous ticker symbol was "OHRP". Results of Operations
Results of operations for the fiscal year endedSeptember 30, 2019 reflect the following changes from the period from inception (August 28, 2018 ) toSeptember 30, 2018 : Period Ended September 30, 2019 2018 Change General and administrative$ 9,095,674 $ 28,393 $ 9,067,281 Research and development 4,273,318 12,819 4,260,499 Research and development expense- licenses acquired 12,967,415 - 12,967,415 Total Operating Expenses 26,336,407 41,212 26,295,195 Operating Loss (26,336,407 ) (41,212 ) (26,295,195 ) Interest expense (128,951 ) (740 ) (128,211 )
Change in fair value of warrant liabilities (492,889 ) -
(492,889 ) Net Loss$ (26,958,247 ) $ (41,952 ) $ (26,916,295 )
During the fiscal year endedSeptember 30, 2019 , we had no revenues and our operating loss increased by$26,295,195 compared to the period from inception toSeptember 30, 2018 . Our net loss increased by$26,916,295 for the fiscal year endedSeptember 30, 2019 , as compared to the period from inception toSeptember 30, 2018 . Until we are able to generate revenues, our management expects to continue to incur net losses.
General and Administrative Expense
General and administrative expense consists primarily of legal and professional fees, wages and stock-based compensation. General and administrative expenses increased by$9,067,281 for the fiscal year endedSeptember 30, 2019 , as compared to the period from inception toSeptember 30, 2018 , primarily due to an increase in stock-based compensation expense, employee head count and need for legal and professional services.
Research and Development Expense
Research and development expense consist primarily of professional fees, manufacturing expenses, wages and stock-based compensation. Research and development expenses increased by$4,260,499 for the fiscal year endedSeptember 30, 2019 , as compared to the period from inception toSeptember 30, 2018 , primarily due to an increase in stock-based compensation, employee head count and the ramp up of research and development before and after the close of the Merger withOhr Pharmaceutical .
Research and Development Expense- licenses acquired
Research and development expense- licenses acquired consists of licenses acquired from CMU and in the Merger with Ohr. Research and development expense- licenses acquired increased by$12,967,415 , for the fiscal year endedSeptember 30, 2019 , as compared to the period from inception toSeptember 30, 2018 , primarily due to our acquisition of license rights in 2019. 67 Interest Expense Interest income consists primarily of interest on convertible notes and notes payable. Interest expense increased by$128,211 for the fiscal year endedSeptember 30, 2019 , as compared to the period from inception toSeptember 30, 2018 , primarily due to the issuance of convertible notes.
Change in fair value of warrant liabilities
Change in fair value of warrant liabilities reflects the changes in warrant liabilities primarily due to changes in our stock price. Change in fair value of warrant liabilities increased by$492,889 for the fiscal year endedSeptember 30, 2019 , as compared to the period from inception toSeptember 30, 2018 , primarily due to warrants issued in connection with the CMU License Agreement.
Liquidity, Capital Resources and Financial Condition
We have limited working capital reserves with which to fund our continuing operations. We are reliant, at present, upon our capital reserves for ongoing operations and has no revenues.
Net working capital increased from the fiscal year endedSeptember 30, 2018 to the fiscal year endedSeptember 30, 2019 by$8,319,118 (to$8,527,222 from$208,104 ) primarily due to cash proceeds from financings completed inJuly 2019 . Our quarterly cash burn has increased significantly compared to the prior period from inception toSeptember 30, 2018 due to increased research and development activities and costs associated with the Merger. We anticipate that our cash needs will increase relative to prior periods as we achieve progress with our research and development objectives. Alongside the closing of the Merger, inJuly 2019 , we completed two financings raising gross proceeds of approximately$14 million , and we believe that our current cash balance will provide sufficient capital to continue operations to the end of fiscal 2020. At present, we have no bank line of credit or other fixed source of capital reserves. Should we need additional capital in the future, we will be primarily reliant upon private or public placement of our equity or debt securities, or a strategic transaction, for which there can be no warranty or assurance that we may be successful in such efforts.
Contractual Obligations and Commitments
Leases From inception through the year endedSeptember 30, 2018 , Legacy NeuBase utilized the services ofLifeX Labs LLC . These services included accounting consultation and office space rental. This agreement was terminated onJanuary 8, 2019 .Dietrich Stephan , our Chief Executive Officer ("CEO"), was on the board and acting as CEO ofLifeX Labs LLC untilDecember 28, 2018 , when he resigned all positions within LifeX. During the period fromAugust 28, 2018 (inception) toSeptember 30, 2018 and the year endedSeptember 30, 2019 ,LifeX Labs was paid$1,575 and$10,628 , respectively, by Legacy NeuBase. OnMarch 12, 2019 , we entered into a sublease agreement with StartUptown. The monthly rent on the one year sublease is$2,532 and we provided a security deposit of$2,532 upon signature of the agreement. The total sublease liability for the term of the sublease is$30,381 excluding the security deposit. The sublease includes an option to extend the agreement for up to six months. OnMay 21, 2019 , we entered into an amendment to the sublease agreement with StartUptown to increase our office space, whereby the monthly rent on the one year sublease was increased from$2,532 to$4,521 per month. All other material terms remained the same. OnJuly 29, 2019 , we entered into a second amendment to the sublease agreement with StartUptown to increase our office space, whereby the monthly rent on the sublease was increased from$4,521 to$6,883 per month. All other material terms remained the same. Cash Flow Summary The following table summarizes selected items in our consolidated statements of cash flows: For the Year Ended For the Period FromAugust 28 ,September 30, 2018 (Inception) toSeptember 30, 2019 2018
Net cash used in operating activities $ (2,845,488 ) $ (455 ) Net cash used in investing activities (685,225 ) - Net cash provided by financing activities 13,595,079 250,055 Net increase in cash and cash equivalents $ 10,064,366
$ 249,600
Operating Activities from Continuing Operations
Net cash used in operating activities from continuing operations was approximately$2.8 million for the fiscal year endedSeptember 30, 2019 and net cash used in operating activities from continuing operations was approximately$455 for the period from inception toSeptember 30, 2018 . Net cash used in operating activities from continuing operations in 2019 was primarily as a result of our net loss, offset by our stock-based compensation expense and research and development expense-licenses acquired.
Investing Activities from Continuing Operations
Net cash used in investing activities from continuing operations was approximately$685 thousand for the fiscal year endedSeptember 30, 2019 and net cash used in investing activities from continuing operations was$0 for the period from inception toSeptember 30, 2018 . Net cash used in investing activities in 2019 was primarily as a result of transaction costs paid in connection with the Merger with Ohr and the acquisition of the CMU License as well as the purchase of laboratory and office equipment. These expenditures were partially offset by cash acquired in connection with the Merger with Ohr. 68
Financing Activities From Continuing Operations
Net cash provided by financing activities was approximately$13.6 million for the fiscal year endedSeptember 30, 2019 and net cash provided by financing activities was approximately$250 thousand for the period from inception toSeptember 30, 2018 . Net cash provided by financing activities in 2019 reflects the proceeds from the issuance of common stock in our pre-acquisition and private placement financings. Net cash provided by financing activities in 2019 also reflects the proceeds from the issuance of convertible notes.
Off-Balance Sheet Arrangements
As of
Recent Accounting Pronouncements
InJune 2018 , theFinancial Accounting Standards Board (the "FASB") issued Accounting Standard Update ("ASU") 2018-07, Compensation- Stock Compensation (Topic 718) Improvements to Nonemployee Share-Based Payment Accounting, which expands the scope of Topic 718 to include share-based payment transactions for acquiring goods and services from nonemployees. The guidance also specifies that Topic 718 applies to all share-based payment transactions in which a grantor acquires goods or services to be used or consumed in a grantor's own operations by issuing share-based payment awards. The Company early adopted this standard as ofOctober 1, 2018 . The Company did not grant share-based payment awards during the period fromAugust 28, 2018 (inception) throughSeptember 30, 2018 . Accordingly, the adoption of this standard did not have a material effect on the Company's consolidated financial statements and related disclosures. InMay 2014 , the FASB issued ASU 2014-09, Revenue from Contracts with Customers (Topic 606), which requires entities to recognize revenue in a way that depicts the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. The new guidance also requires additional disclosure about the nature, amount, timing and uncertainty of revenue and cash flows arising from customer contracts. The FASB subsequently issued ASU 2016-10, Revenue from Contracts with Customers: (Topic 606) Identifying Performance Obligations and Licensing, to address issues arising from implementation of the new revenue recognition standard. The Company adopted the standard using the full retrospective method. The Company does not currently have a revenue stream. Accordingly, the adoption of this standard did not have a material effect on the Company's consolidated financial statements and related disclosures. InFebruary 2016 , the FASB issued ASU 2016-2, Leases. The new standard establishes a right-of-use ("ROU") model that requires a lessee to record a ROU asset and a lease liability on the balance sheet for all leases with terms longer than 12 months. Leases will be classified as either finance or operating, with classification affecting the pattern of expense recognition in the income statement. The new standard is effective for fiscal years beginning afterDecember 15, 2018 , including interim periods within those fiscal years. A modified retrospective transition approach is required for lessees for capital and operating leases existing at, or entered into after, the beginning of the earliest comparative period presented in the financial statements, with certain practical expedients available. The Company plans to adopt this standard onOctober 1, 2019 . The adoption of the standard is not expected to have a material effect on the Company's consolidated financial statements and related disclosures as the Company does not currently have leases with terms longer
than 12 months.
Critical Accounting Estimates and Policies
Our management's discussion and analysis of our financial condition and results of operations is based on our financial statements, which have been prepared in accordance withUnited States generally accepted accounting principles ("GAAP"). The preparation of these financial statements requires us to make estimates, judgments and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities as of the date of the balance sheet and the reported amounts of expenses during the reporting period. In accordance with GAAP, we evaluate our estimates and judgments on an ongoing basis. The most significant estimates relate to the valuation of share-based compensation, the valuation of licenses, the fair value of warrant liabilities and the valuation allowance of deferred tax assets resulting from net operating losses. We base our estimates and assumptions on current facts, our limited historical experience from operations and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying value of assets and liabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. We define our critical accounting policies as those accounting principles that require it to make subjective estimates and judgments about matters that are uncertain and are likely to have a material impact on our financial condition and results of operations, as well as the specific manner in which we apply those principles. While our significant accounting policies are more fully described in Note 2 to our financial statements appearing elsewhere in this Annual Report on Form 10-K, we believe the following are the critical accounting policies used in the preparation of our financial statements that require significant estimates and judgments: Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the financial statements and the reported amounts of revenue and expenses during the reporting period. Actual results could differ from those estimates. 69 Cash and Cash Equivalents We consider all highly-liquid investments purchased with short term maturities to be cash equivalents. We had cash equivalents as ofSeptember 30, 2019 and did not have any cash equivalents as ofSeptember 30, 2018 .
Fair Value of Financial Instruments
In accordance with Accounting Standards Codification ("ASC") 820, the carrying value of cash and cash equivalents, accounts payable and notes payable approximates fair value due to the short-term maturity of these instruments. ASC 820 clarifies the definition of fair value, prescribes methods for measuring fair value, and establishes a fair value hierarchy to classify the inputs used in measuring fair value as follows: ? Level 1 - Quoted prices in active markets for identical assets or liabilities on the reporting date. ? Level 2 - Pricing inputs are based on quoted prices for similar instruments in active markets, quoted prices for identical or similar
instruments in markets that are not active and model-based valuation
techniques for which all significant assumptions are observable in the market or can be corroborated by observable market data for substantially the full term of the assets or liabilities. ? Level 3 - Pricing inputs are generally unobservable and include situations where there is little, if any, market activity for the investment. The inputs into the determination of fair value require management's judgment or estimation of assumptions that market participants would use in pricing the assets or liabilities. The fair
values are therefore determined using factors that involve considerable
judgment and interpretations, including but not limited to private and public comparables, third-party appraisals, discounted cash flow models and fund manager estimates.
As of
As of
Research and Development Research and development expenses are expensed in the statement of operations as incurred in accordance with theFinancial Accounting Standards Board ("FASB") ASC 730, Research and Development. Research and development expenses include patent and certain legal fees. We incurred net research and development expenses of$12,819 from inception toSeptember 30, 2018 . During the fiscal year endedSeptember 30, 2019 , we incurred$4,273,318 in research and development expenses. Share-Based Compensation
We follow the provisions of ASC 718 - Stock Compensation which requires all share-based payments to employees, including grants of employee stock options, be recognized in the income statement based on their fair values. We have early adopted Accounting Standard Update ("ASU") 2018-07, which expands the scope of ASC 718 to include share based payments granted to nonemployees and supersedes the guidance in ASC 505-50. The Company estimates the fair value of stock option grants using the Black-Scholes option pricing model, and the assumptions used in calculating the fair value of stock-based awards represent management's best estimates and involve inherent uncertainties and the application of management's judgment.
Stock-based compensation expense is recognized in our financial statements on a straight-line basis over for each separately vesting portion of the award. The stock-based compensation awards generally vest over a period of up to four years. All stock-based compensation costs are recorded in general and administrative or research and development costs in the consolidated statements of operations based upon the underlying individual's role at the Company. Income taxes Income taxes are recorded in accordance with Accounting Standards Codification Topic 740, Income Taxes ("ASC 740"), which provides for deferred taxes using an asset and liability approach. We recognize deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. We account for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, we recognize the tax benefit of tax positions to the extent that the benefit would more likely than not be realized assuming examination by the taxing authority. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. NeuBase recognizes any interest and penalties accrued related to unrecognized tax benefits as income tax expense. The Tax Cuts and Jobs Act (the "Act") was enacted onDecember 22, 2017 . The Act reduces the US federal corporate tax rate from 35% to 21%, and among other changes, eliminates net operating loss carrybacks for losses arising in taxable years beginning afterDecember 31, 2017 . Further, operating losses arising in tax years afterDecember 31, 2017 , are carried forward indefinitely. The Act did not result in any material changes to our financial statements or results of operations. 70
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