Disclosures Regarding Forward-Looking Statements





The following should be read in conjunction with the unaudited condensed
consolidated financial statements and the related notes that appear elsewhere in
this report as well as in conjunction with the Risk Factors section in our
Annual Report on Form 10-K for the year ended September 30, 2019 as filed with
the United States Securities and Exchange Commission ("SEC") on January 10,
2020. This report and our Form 10-K include forward-looking statements made
based on current management expectations pursuant to the safe harbor provisions
of the Private Securities Litigation Reform Act of 1995, as amended.



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 Part II, Item 1A - Risk Factors of this Quarterly
Report and in Part I, Item 1A - Risk Factors of our Annual Report. In light of
the significant risks and uncertainties inherent in the forward-looking
statements included in this Quarterly Report on Form 10-Q and in our 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 the
SEC.



Overview



Recent Developments


Announcement of Positive Preclinical Data and Target Milestones

On March 31, 2020, we announced positive preclinical data from our pharmacokinetics studies in non-human primates ("NHPs") and in vitro pharmacodynamics data in patient-derived cell lines. Our pharmacokinetics studies in NHPs demonstrated, among other things:

· rapid uptake of our PATrOL™-enabled compound out of the body's circulation


    after single-dose systemic intravenous administration, with a half-life in
    circulation of approximately 1.5 hours;



· penetration by our PATrOL™-enabled compound in every organ system studied,


    including the central nervous system and skeletal muscle; and



· retention of therapeutically relevant doses for greater than one week after


    single-dose injection.



Our pharmacodynamics studies in patient-derived cell lines demonstrated, among other things:

· activity in engaging target disease-causing transcripts and knocking-down


    resultant malfunctioning mutant huntingtin ("mHTT") protein levels
    preferentially over normal huntingtin ("HTT") 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.




In addition, PATrOL™ 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.



We believe that the data from these studies support the advancement of the
Company's HD and DM1 programs into lead optimization and subsequent IND-enabling
studies. We announced the following target milestones for our NT0100 and NT0200
programs:



                                       14





NT0100 Program for HD



  · Lead candidate selection by the end of the calendar year 2020




· Initiation of IND-enabling studies during the first half of the calendar year


    2021




  · IND filing by the end of the calendar year 2021




  · Clinical data by the end of the calendar year 2022




NT0200 Program for DM1

· Lead candidate selection during the first half of the calendar year 2021

· Initiation of IND-enabling studies during the second half of the calendar year


    2021




  · IND filing during the second half of the calendar year 2022




The intersection of the NHP pharmacokinetic data and the in vitro
patient-derived pharmacodynamic data provides a roadmap to create a pipeline of
therapeutic candidates which can reach target tissues of interest after systemic
administration and achieve the desired activity at that dose.



Description of the Company



We are a biotechnology company focused on developing next-generation therapies
to treat rare genetic diseases and cancer caused by mutant genes. Our modular
peptide-nucleic acid antisense oligonucelotide ("PATrOL™") platform is designed
to improve upon current gene silencing treatments by combining the specificity
of antisense oligonucleotides ("ASOs") with the broad tissue distribution
capabilities of small molecules. Given that every human disease may have a
genetic component, we believe that our differentiated platform technology has
the potential for broad impact. We plan to use our platform to address genetic
diseases and we are initially focused on Huntington's disease ("HD") and
myotonic dystrophy type 1 ("DM1").



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-messenger ribonucleic acid ("mRNA"), 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.



We are developing 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 peptide-nucleic acids ("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, is 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 with attached nucleobases that are linked
together into a specific sequence that matches (complements) the target RNA
sequence; however, 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 intended therapeutic
effect. Our PNAs assume predominantly a single conformation with any
constellation of nucleobases 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.



                                       15





In addition to the backbone conformational purity that allows for more efficient
discovery of drug product candidates, we also have a kit of proprietary
bi-facial (also known as 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 as compared to the normal RNA.
Enhanced selectivity for mutant RNAs as compared to 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, including across the blood brain barrier and into the central
nervous system.



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. We 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 the NT0100 program, targeted at
HD, a repeat expansion disorder, and the NT0200 program, targeted at DM1.
Preclinical studies are being conducted to evaluate the PATrOL™ platform
technology and program candidates in the areas of pharmacokinetics,
pharmacodynamics, and safety, and we reported results from certain of those
studies in the first calendar quarter of 2020. We expect to report the results
of certain additional studies in the fourth quarter of calendar year 2020 via
press release or in a scientific publication or presentation at a scientific
conference. 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 believe we can create ASOs that have distinct
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 predominantly
only one stereoisomer; the ability of the PNA backbone to invade, open up
secondary 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 nucleobases that increase
selectivity to specific target sequences including secondary and tertiary
structures that has been licensed exclusively from Carnegie Mellon University
("CMU"); technology to allow self-assembly of our small PNAs at the RNA target
to increase selectivity which has been licensed exclusively from CMU; the
ability to modulate cell permeability and be broadly distributed throughout the
body after systemic administration including into the brain; the lack of innate
or acquired immune responses of similar PNAs in preclinical models; and
potential minimal toxicity based on previous in-vivostudies 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.



Product Pipeline



Huntington's Disease



HD is a devastating rare neurodegenerative disorder. After onset, symptoms such
as uncontrolled movements, cognitive impairments and emotional disturbances
worsen over time. HD is caused by toxic aggregation of mutant huntingtin
protein, leading to progressive neuron loss in the striatum and cortex of the
brain. The wildtype huntingtin gene ("HTT") has a region in which a three-base
DNA sequence, CAG, is repeated many times. When the DNA sequence CAG is repeated
26 or fewer times in this region, the resulting protein behaves normally. While
the wildtype function of HTT is largely uncharacterized, the protein is known to
be essential for normal brain development. When the DNA sequence CAG is repeated
40 times or more in this region, the resulting protein becomes toxic and causes
HD. Every person has two copies, or alleles, of the HTT. Only one of the alleles
(the "mutant" allele) needs to bear at least 40 CAG repeats for HD to occur. HD
is one of many known repeat expansion disorders, which are a set of genetic
disorders caused by a mutation that leads to a repeat of nucleotides exceeding
the normal threshold. Current therapies for patients with HD can only manage
individual symptoms. There is no approved therapy that has been shown to delay
or halt disease progression. There are approximately 30,000 symptomatic patients
in the U.S. and more than 200,000 at-risk of inheriting the disease globally.



                                       16




NT0100 Program - PATrOL™ Enabled PNA for Huntington's Disease

We are initially focused on HD, a fatal rare genetic repeat expansion disorder with no viable treatment options.


One especially important advantage of the PATrOL™ platform that makes it
promising for the treatment of repeat expansion disorders like HD is the ability
of our small ASOs to potentially self-assemble within an RNA hairpin. As the
number of repeats increases, the PATrOL™ oligonucleotides bind more tightly to
each other and the mutant RNA. This allows our therapies to potentially
inactivate mutant HTT mRNA before it can be translated into harmful protein via
selective binding to the expanded CAG repeats while leaving the normal HTTmRNA
largely unbound to drug and producing functional protein. Achieving mutant
allele selectivity would be a key advantage for any RNA-based approach aiming to
treat HD. The PATrOL™-enabled NT0100 program is currently in preclinical
development for the treatment of HD.



NT0200 Program - PATrOL™ Enabled PNA for Myotonic Dystrophy Type 1





Our pipeline also contains a second potentially transformative medicine, which
we believe has significant potential for DM1, a severe and rare trinucleotide
repeat disease. DM1 is a multisystem disorder that primarily affects skeletal
and smooth muscle. DM1 is caused by expansion of a CTG trinucleotide repeat in
the noncoding region of the DM1 Protein Kinase gene (DMPK), which captures and
sequesters splice proteins. Sequestered splice proteins cannot then fulfill
their normal functions. The diagnosis of DM1 is suspected in individuals with
characteristic muscle weakness and is confirmed by molecular genetic testing of
DMPK. CTG repeat length exceeding 34 repeats is abnormal. Molecular genetic
testing detects pathogenic variants in nearly 100% of affected individuals. It
is estimated that the global prevalence of DM1 is 1 in 20,000 individuals. Our
development strategy targets the DM1 expanded allele with PATrOL™-enabled drug
candidates to disrupt and/or open the mutant hairpin and allow release of
sequestered splice proteins.



Additional Indications



In addition, we are in the process of building an early stage pipeline of other
therapies that focus on the unique advantages of our technology across a variety
of diseases.


Critical Accounting Estimates and Policies





The preparation of financial statements in accordance with United States
generally accepted accounting principles ("U.S. GAAP") requires management to
make estimates and assumptions that affect the amounts reported in our unaudited
condensed consolidated financial statements and accompanying notes. Management
bases its estimates on historical experience, market and other conditions, and
various other assumptions it believes to be reasonable. Although these estimates
are based on management's best knowledge of current events and actions that may
impact us in the future, the estimation process is, by its nature, uncertain
given that estimates depend on events over which we may not have control. If
market and other conditions change from those that we anticipate, our unaudited
condensed consolidated financial statements may be materially affected. In
addition, if our assumptions change, we may need to revise our estimates, or
take other corrective actions, either of which may also have a material effect
in our unaudited condensed consolidated financial statements. We review our
estimates, judgments, and assumptions used in our accounting practices
periodically and reflect the effects of revisions in the period in which they
are deemed to be necessary. We believe that these estimates are reasonable;
however, our actual results may differ from these estimates.



Our critical accounting policies and estimates are discussed in our Annual
Report on Form 10-K for the fiscal year ended September 30, 2019 and there have
been no material changes to such policies or estimates during the nine months
ended June 30, 2020.


Recent Accounting Pronouncements

Please refer to Note 2, Significant Accounting Policies-Recent Accounting Pronouncements, in Item 1, Financial Statements for a discussion of recent accounting pronouncements.





                                       17







Results of Operations


Results of operations for the quarter ended June 30, 2020 reflect the following changes from the quarter ended June 30, 2019:





                                                    Three Months Ended June 30,
                                                       2020               2019            Change
OPERATING EXPENSES
General and administrative expenses               $     2,331,283     $  1,655,189     $    676,094
Research and development expenses                       1,492,881         

305,204        1,187,677
TOTAL OPERATING EXPENSES                                3,824,164        1,960,393        1,863,771
LOSS FROM OPERATIONS                                   (3,824,164 )     (1,960,393 )     (1,863,771 )
OTHER INCOME (EXPENSE)
Interest expense                                           (1,573 )        (60,944 )         59,371

Change in fair value of warrant liabilities              (285,284 )        (13,240 )       (272,044 )
Equity in losses on equity method investment             (101,238 )              -         (101,238 )
Other income                                              412,371                -          412,371
Total income (expenses), net                               24,276         

(74,184 )         98,460
NET LOSS                                          $    (3,799,888 )   $ (2,034,577 )   $ (1,765,311 )




During the quarter ended June 30, 2020, our operating loss increased by $1.9
million compared to the quarter ended June 30, 2019. Our net loss increased by
$1.8 million for the quarter ended June 30, 2020, as compared to the quarter
ended June 30, 2019. Until we are able to generate revenue from product sales,
our management expects to continue to incur net losses.



General and Administrative Expenses





General and administrative expenses consist primarily of legal and professional
fees, wages and stock-based compensation. General and administrative expenses
increased by $0.7 million for the quarter ended June 30, 2020, as compared to
the quarter ended June 30, 2019, primarily due to an increase in stock-based
compensation expense, employee head count and need for legal and professional
services.


Research and Development Expenses





Research and development expenses consist primarily of professional fees,
research, development, and manufacturing expenses, and wages and stock-based
compensation. Research and development expenses increased by $1.2 million for
the quarter ended June 30, 2020, as compared to the quarter ended June 30, 2019,
primarily due to an increase in stock-based compensation, employee head count
and the ramp up of research and development activities.



Interest Expense



Interest expense consists primarily of interest on convertible notes and notes
payable. Interest expense decreased by $0.06 million for the quarter ended
June 30, 2020, as compared to the quarter ended June 30, 2019 primarily due to a
decrease in outstanding convertible notes. No convertible notes were outstanding
during the quarter ended June 30, 2020.



Change in fair value of warrant liabilities





Change in fair value of warrant liabilities reflects the changes in the fair
value of outstanding warrants which is primarily driven by changes in our stock
price. Change in fair value of warrant liabilities was a loss of $0.3 million
for the quarter ended June 30, 2020, as compared to a loss of $0.01 million for
the quarter ended June 30, 2019, due to the change in valuation of warrants
acquired in the Merger with Ohr, which did not exist in the comparative prior
year period.


Equity in losses on equity method investment


The Company accounts for its investment in DepYmed common shares using the
equity method of accounting and records its proportionate share of DepYmed's net
income and losses. Equity in losses for the three months ended June 30, 2020 was
approximately $0.1 million. No such investment existed in the comparative prior
year period.



                                       18





Other income



Other income reflects the Company's license, and completed transfer, of certain
clinical trial data during the three months ended June 30, 2020. No such license
and data transfer occurred during the comparative prior year period.



Results of operations for the nine months ended June 30, 2020 reflect the following changes from the nine months ended June 30, 2019:





                                                       Nine Months Ended June 30,
                                                          2020              2019            Change
OPERATING EXPENSES

General and administrative expenses                  $    7,624,984     $  3,991,567     $  3,633,417
Research and development expenses                         4,336,576          343,206        3,993,370
Research and development expense- license acquired                -       

1,046,965       (1,046,965 )
TOTAL OPERATING EXPENSES                                 11,961,560        5,381,738        6,579,822
LOSS FROM OPERATIONS                                    (11,961,560 )     (5,381,738 )     (6,579,822 )
OTHER INCOME (EXPENSE)
Interest expense                                             (3,226 )       (119,195 )        115,969

Change in fair value of warrant liabilities                (909,474 )        (51,942 )       (857,532 )
Loss on disposal of fixed asset                              (3,230 )              -           (3,230 )
Equity in losses on equity method investment               (218,589 )      

       -         (218,589 )
Other income                                                412,371                -          412,371
Total other expenses, net                                  (722,148 )       (171,137 )       (551,011 )
NET LOSS                                             $  (12,683,708 )   $ (5,552,875 )   $ (7,130,833 )
During the nine months ended June 30, 2020, our operating loss increased by $6.6
million compared to the nine months ended June 30, 2019. Our net loss increased
by $7.1 million for the nine months ended June 30, 2020, as compared to the nine
months ended June 30, 2019. Until we are able to generate revenue from product
sales, our management expects to continue to incur net losses.



General and Administrative Expenses





General and administrative expenses consist primarily of legal and professional
fees, wages and stock-based compensation. General and administrative expenses
increased by $3.6 million for the nine months ended June 30, 2020, as compared
to the nine months ended June 30, 2019, primarily due to an increase in
stock-based compensation expense, employee head count and need for legal and
professional services.


Research and Development Expenses





Research and development expenses consist primarily of professional fees,
research, development, and manufacturing expenses, and wages and stock-based
compensation. Research and development expenses increased by $4.0 million for
the nine months ended June 30, 2020, as compared to the nine months ended
June 30, 2019, primarily due to an increase in stock-based compensation,
employee head count and the ramp up of research and development activities.

Research and Development Expense- licenses acquired


Research and development expense- licenses acquired during the nine months ended
June 30, 2019 consists of the license acquired from CMU. Research and
development expense- licenses acquired decreased by $1.0 million, for the nine
months ended June 30, 2020, as compared to the nine months ended June 30, 2019,
due to our acquisition of license rights in the 2019 period.



Interest Expense



Interest expense consists primarily of interest on convertible notes and notes
payable. Interest expense decreased by $0.1 million for the nine months ended
June 30, 2020, as compared to the nine months ended June 30, 2019, primarily due
to a decrease in convertible notes. No convertible notes were outstanding during
the nine months ended June 30, 2020.



                                       19




Change in fair value of warrant liabilities





Change in fair value of warrant liabilities reflects the changes in the fair
value of outstanding warrants which is primarily driven by changes in our stock
price. Change in fair value of warrant liabilities was $0.9 million for the nine
months ended June 30, 2020, as compared to $0.05 million for the nine months
ended June 30, 2019, due to the change in valuation of warrants acquired in the
Merger with Ohr, which did not exist in the comparative prior year period.

Equity in losses on equity method investment


The Company accounts for its investment in DepYmed common shares using the
equity method of accounting and records its proportionate share of DepYmed's net
income and losses. Equity in losses for the nine months ended June 30, 2020 was
approximately $0.2 million. No such investment existed in the comparative prior
year period.



Other income



Other income reflects the Company's license, and completed transfer, of certain
clinical trial data during the nine months ended June 30, 2020. No such license
and data transfer occurred during the comparative prior year period.



Liquidity, Capital Resources and Financial Condition





We have incurred substantial operating losses since our inception and expect to
continue to incur significant operating losses for the foreseeable future and
may never become profitable. As of June 30, 2020, we had an accumulated deficit
of $39.7 million. We are reliant, at present, upon our capital reserves for
ongoing operations and have no product revenue.



Net working capital increased from September 30, 2019 to the period ended
June 30, 2020 by $24.9 million (to $33.4 million from $8.5 million) primarily
due to our April 2020 underwritten public offering of 6,037,500 shares of common
stock, at a price to the public of $6.00 per share, offset, in part, by costs
for the development of our PATrOL™ platform technology and lead programs. We
received net proceeds from the offering of approximately $33.3 million, after
deducting the underwriting discounts and commissions and other estimated
offering expenses payable by the Company. Our cash burn for the period increased
significantly compared to prior periods due to increased research and
development activities. We anticipate that our cash needs in the future will
increase relative to prior periods as we proceed with our research and
development objectives.



We believe that our current cash balance will provide sufficient capital to
continue operations into the second quarter of calendar 2022. In particular, we
expect that these funds will allow us to achieve certain milestones for our
NT0100 program for HD and our NT0200 program for DM1, but we expect that we will
need to obtain additional funding to obtain clinical data for our NT0100 program
and to submit an IND for our NT0200 program. Despite these expectations, our
forecast of the period of time through which our financial resources will be
adequate to support our operations is a forward-looking statement that involves
risks and uncertainties, and actual results could vary materially. We have based
this estimate on assumptions that may prove to be wrong, and we could use our
capital resources sooner than we expect. We will continue to assess our working
capital requirements, and, if circumstances warrant, we will make appropriate
adjustments to our operating plan. Furthermore, we are closely monitoring
ongoing developments in connection with the COVID-19 pandemic, which may
negatively impact our commercial prospects in fiscal 2020 and beyond. Please see
Part II, Item 1A - Risk Factors-"Our operations may be adversely affected by the
coronavirus outbreak, and we face risks that could impact our business" for
further discussion of the effect of the COVID-19 pandemic on our operations.



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 a 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. If the Company is unable to maintain
sufficient financial resources, its business, financial condition and results of
operations will be materially and adversely affected. This could affect future
development and business activities and potential future clinical studies and/or
other future ventures. Failure to obtain additional equity or debt financing
will have a material, adverse impact on the Company's business operations. There
can be no assurance that the Company will be able to obtain the needed financing
to achieve its goals on acceptable terms or at all.



                                       20





Cash Flow Summary


The following table summarizes selected items in our unaudited condensed consolidated statements of cash flows:





                                                               Nine Months Ended June 30,
                                                                  2020               2019
Net cash used in operating activities                        $    (7,142,340 )    $ (394,779 )
Net cash used in investing activities                               (362,915 )      (379,380 )
Net cash provided by financing activities                         

33,116,045 592,656 Net increase (decrease) in cash and cash equivalents $ 25,610,790 $ (181,503 )






Operating Activities



Net cash used in operating activities was approximately $7.1 million for the
nine months ended June 30, 2020, as compared to approximately $0.4 million for
the nine months ended June 30, 2019. Net cash used in operating activities in
the nine months ended June 30, 2020 was primarily the result of our net loss,
offset by our stock-based compensation expense, the change in fair value of
warrant liabilities, depreciation and amortization expense and the loss on
equity method investment. Net cash used in operating activities in the nine
months ended June 30, 2019 was primarily the result of our net loss, offset by
research and development expense-license acquired, stock based compensation and
changes in operating assets and liabilities.



Investing Activities



Net cash used in investing activities was approximately $0.4 million for the
nine months ended June 30, 2020, as compared to $0.4 million for the nine months
ended June 30, 2019. Net cash used in investing activities in the nine months
ended June 30, 2020 was primarily the result of purchases of laboratory
equipment. Net cash used in investing activities in the nine months ended
June 30, 2019 was primarily the result of purchases of laboratory equipment and
cash consideration and expenses paid in connection with the acquisition of

the
CMU License.



Financing Activities



Net cash provided by financing activities was approximately $33.1 million for
the nine months ended June 30, 2020, as compared to $0.6 million for the nine
months ended June 30, 2019. Net cash provided by financing activities for the
nine months ended June 30, 2020 reflects the proceeds from the issuance of
common stock of $33.3 million, net of issuance costs, and the principal payments
of financed insurance. Net cash provided by financing activities for the nine
months ended June 30, 2019 primarily reflects the net proceeds received from the
issuance of convertible notes.



Off-Balance Sheet Arrangements

As of June 30, 2020, we did not have any off-balance sheet arrangements as defined in Item 303(a)(4)(ii) of Regulation S-K.





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