UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
(Mark One) | |
QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 | |
For the quarterly period ended | |
OR | |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 | |
For the transition period from to | |
Commission File Number:
(Exact name of registrant as specified in its charter)
(State or other jurisdiction of | (I.R.S. Employer |
(Address of principal executive offices) | (Zip Code) |
(
(Registrant’s telephone number, including area code)
N/A
(Former name, former address and former fiscal year, if changed since last report)
Securities registered pursuant to Section 12(b) of the Act: | ||||
Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||
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Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days.
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files).
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
Large accelerated filer ☐ | Accelerated filer ☐ |
Smaller reporting company | |
Emerging growth company |
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes
The number of shares of the registrant’s common stock, $0.001 par value per share, outstanding at November 5, 2021 was
Table of Contents
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PART I - FINANCIAL INFORMATION | ||
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4 | ||
6 | ||
Balance Sheets as of September 30, 2021 and December 31, 2020 | 6 | |
Statements of Operations for the three and nine months ended September 30, 2021 and 2020 | 7 | |
8 | ||
Statements of Cash Flows for the nine months ended September 30, 2021 and 2020 | 9 | |
10 | ||
Management’s Discussion and Analysis of Financial Condition and Results of Operations | 21 | |
36 | ||
37 | ||
PART II OTHER INFORMATION | ||
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37 | ||
83 | ||
83 | ||
83 | ||
83 | ||
84 | ||
85 | ||
2
FORWARD-LOOKING STATEMENTS
This Quarterly Report on Form 10-Q contains forward-looking statements that involve risks and uncertainties. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this Quarterly Report on Form 10-Q are forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “may,” “will,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “potential,” “continue” or the negative of these terms or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:
| ● | our plans relating to clinical trials for BXCL501, BXCL701 and our other product candidates; |
| ● | our plans for 505(b)(2) regulatory path approval; |
| ● | our plans to research, develop and commercialize our current and future product candidates; |
| ● | our plans to seek to enter into collaborations for the development and commercialization of certain product candidates; |
| ● | the potential benefits of any future collaboration; |
| ● | the timing of and our ability to obtain and maintain regulatory approvals for our product candidates; |
| ● | the rate and degree of market acceptance and clinical utility of any products for which we receive marketing approval; |
| ● | our commercialization, marketing and manufacturing capabilities and strategy; |
| ● | our intellectual property position and strategy; |
| ● | our estimates regarding expenses, future revenue, capital requirements and need for additional financing; |
| ● | developments relating to our competitors and our industry; |
| ● | the impact of government laws and regulations; |
| ● | the impact of COVID-19 on our business, including our preclinical studies and clinical trials: and |
| ● | our relationship with BioXcel LLC. |
These forward-looking statements are based on management’s current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Factors that may cause actual results to differ materially from current expectations include, among other things, those listed under “Summary Risk Factors” Part II, Item 1A. “Risk Factors,” and Part I, Item 2. “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in this Quarterly Report on Form 10-Q. Given these uncertainties, you should not rely on these forward-looking statements as predictions of future events. Except as required by law, we assume no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future.
This Quarterly Report on Form 10-Q also contains estimates, projections and other information concerning our industry, our business, and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data and similar sources.
As used in this Quarterly Report on Form 10-Q, unless otherwise specified or the context otherwise requires, the terms “we,” “our,” “us,” the “Company” or “BTI” refer to BioXcel Therapeutics, Inc. and “BioXcel LLC” refers to the Company’s former parent company and significant stockholder, BioXcel LLC and its predecessor, BioXcel Corporation. All brand names or trademarks appearing in this Quarterly Report on Form 10-Q are the property of their respective owners.
We may use our website as a distribution channel of material information about the Company. Financial and other important information regarding the Company is routinely posted on and accessible through the Investors sections of its website at www.bioxceltherapeutics.com. In addition, you may automatically receive email alerts and other information about the Company when you enroll your email address by visiting the “Email Alerts” option under the News / Events menu of the Investors section of ours website at www.bioxceltherapeutics.com.
3
SUMMARY RISK FACTORS
Our business is subject to numerous risks and uncertainties, including those described in Part II, Item 1A. “Risk Factors” in this Quarterly Report on Form 10-Q. You should carefully consider these risks and uncertainties when investing in our common stock. The principal risks and uncertainties affecting our business include the following:
● | We have a limited operating history and have never generated any product revenues, which may make it difficult to evaluate the success of our business to date and to assess our future viability. |
● | We have incurred significant operating losses since inception and anticipate that we will continue to incur substantial operating losses for the foreseeable future and may never achieve or maintain profitability. |
● | We will need substantial additional funding, and if we are unable to raise capital when needed, we could be forced to delay, reduce or eliminate our product development programs or commercialization efforts. |
● | We have limited experience in drug discovery and drug development, and we have never had a drug approved. |
● | In the near term, we are dependent on the success of BXCL501 and BXCL701. If we are unable to complete the clinical development of, obtain marketing approval for or successfully commercialize BXCL501, BXCL701 and our other product candidates, either alone or with a collaborator, or if we experience significant delays in doing so, our business could be substantially harmed. |
● | Interim “top-line” and preliminary data from our clinical trials that we announce or publish from time to time may change as more patient data become available and are subject to audit and verification procedures that could result in material changes in the final data. |
● | The regulatory approval processes of the FDA and comparable foreign authorities are lengthy, time consuming, expensive and inherently unpredictable, and if we are ultimately unable to obtain regulatory approval for our product candidates, our business will be substantially harmed. |
● | Clinical trials are expensive, time-consuming and difficult to design and implement, and involve an uncertain outcome. |
● | Our product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following marketing approval, if any. |
● | BioXcel LLC’s approach to the discovery and development of product candidates based on EvolverAI is novel and unproven, and we do not know whether we will be able to develop any products of commercial value. |
● | If we are required by the FDA or similar regulatory authorities to obtain approval (or clearance, or certification) of a companion diagnostic device in connection with approval of one of our product candidates, and we do not obtain or face delays in obtaining FDA approval (or clearance, or certification) of a companion diagnostic device, we will not be able to commercialize the product candidate and our ability to generate revenue will be materially impaired. |
● | Even if we obtain regulatory approval for BXCL501, BXCL701 or any product candidate, we will still face extensive and ongoing regulatory requirements and obligations and any product candidates, if approved, may face future development and regulatory difficulties. |
● | If our products do not gain market acceptance, our business will suffer because we might not be able to fund future operations. |
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● | Even if we obtain regulatory approvals to commercialize BXCL501, BXCL701 or our other product candidates, our product candidates may not be accepted by physicians or the medical community in general. |
● | We continue to depend on BioXcel LLC to provide us with certain services for our business. |
● | We are substantially dependent on third parties for the manufacture of our clinical supplies of our product candidates, and we intend to rely on third parties to produce commercial supplies of any approved product candidate. Therefore, our development of our products could be stopped or delayed, and our commercialization of any future product could be stopped or delayed or made less profitable if third party manufacturers fail to obtain approval of the FDA or comparable regulatory authorities or fail to provide us with drug product in sufficient quantities or at acceptable prices. |
● | Our failure to find third party collaborators to assist or share in the costs of product development could materially harm our business, financial condition and results of operations. |
● | It is difficult and costly to protect our proprietary rights, and we may not be able to ensure their protection. If our patent position does not adequately protect our product candidates, others could compete against us more directly, which would harm our business, possibly materially. |
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PART I. FINANCIAL INFORMATION
Item 1. Financial Statements
BIOXCEL THERAPEUTICS, INC.
BALANCE SHEETS
(amounts in thousands)
September 30, | December 31, | |||||
| 2021 |
| 2020 | |||
(unaudited) | ||||||
ASSETS |
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Current assets |
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Cash and cash equivalents | $ | | $ | | ||
Prepaid expenses and other current assets |
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Total current assets |
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Property and equipment, net |
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Operating lease right-of-use assets | | | ||||
Other assets | | | ||||
Total assets | $ | | $ | | ||
LIABILITIES AND STOCKHOLDERS' EQUITY |
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Current liabilities |
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Accounts payable | $ | | $ | | ||
Accrued expenses |
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Due to related party |
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Other current liabilities | | | ||||
Total current liabilities |
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Long-term portion of operating lease liabilities | |
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Total liabilities |
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Commitments and contingencies (Note 11) | ||||||
Stockholders' equity |
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Common stock, $ |
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Preferred stock, $ | ||||||
Additional paid-in-capital |
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Accumulated deficit |
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Total stockholders' equity |
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Total liabilities and stockholders' equity | $ | | $ | | ||
The accompanying notes are an integral part of these financial statements.
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BIOXCEL THERAPEUTICS, INC.
STATEMENTS OF OPERATIONS
(amounts in thousands, except per share amounts)
(unaudited)
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||
| 2021 |
| 2020 |
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Revenues | $ | $ | $ | $ | ||||||||
Operating expenses |
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Research and development |
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General and administrative |
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Total operating expenses |
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Loss from operations |
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Other income (expense) |
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Interest income |
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Interest expense | ( | ( | ( | ( | ||||||||
Net loss and comprehensive loss | $ | ( | $ | ( | $ | ( | $ | ( | ||||
Basic and diluted net loss per share attributable to common stockholders | $ | ( | $ | ( | $ | ( | $ | ( | ||||
Weighted average shares outstanding - basic and diluted |
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The accompanying notes are an integral part of these financial statements.
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BIOXCEL THERAPEUTICS, INC.
STATEMENTS OF CHANGES IN STOCKHOLDERS’ EQUITY
(amounts in thousands)
(unaudited)
Additional | ||||||||||||||
Common Stock | Paid in | Accumulated | ||||||||||||
| Shares |
| Amount |
| Capital |
| Deficit |
| Total | |||||
Balance as of December 31, 2019 | | $ | | $ | | $ | ( | $ | | |||||
Issuance of common stock, net of issuance costs of $ | | | | — | | |||||||||
Purchase and cancellation of shares from BioXcel Corporation | ( | — | ( | — | ( | |||||||||
Stock-based compensation | — | — | | — | | |||||||||
Exercise of stock options | | — | | — | | |||||||||
Net loss | — | — | — | ( | ( | |||||||||
Balance as of March 31, 2020 | | $ | | $ | | $ | ( | $ | | |||||
Stock-based compensation | — | — | | — | | |||||||||
Exercise of stock options | | — | | — | | |||||||||
Net loss | — | — | — | ( | ( | |||||||||
Balance as of June 30, 2020 | | $ | | $ | | $ | ( | $ | | |||||
Issuance of common stock, net of issuance costs of $ | | | | — | | |||||||||
Stock-based compensation | — | — | | — | | |||||||||
Exercise of stock options | | — | | — | | |||||||||
Net loss | — | — | — | ( | ( | |||||||||
Balance as of September 30, 2020 | | $ | | $ | | $ | ( | $ | | |||||
Additional | ||||||||||||||
Common Stock | Paid in | Accumulated | ||||||||||||
Shares |
| Amount |
| Capital |
| Deficit |
| Total | ||||||
Balance as of December 31, 2020 | | $ | | $ | | $ | ( | $ | | |||||
Stock-based compensation | — | — | | — | | |||||||||
Exercise of stock options | | | | — | | |||||||||
Net loss | — | — | — | ( | ( | |||||||||
Balance as of March 31, 2021 | | $ | | $ | | $ | ( | $ | | |||||
Issuance of common stock, net of issuance costs of $ | | | | — | | |||||||||
Stock-based compensation | — | — | | — | | |||||||||
Exercise of stock options | | — | | — | | |||||||||
Net loss | — | — | — | ( | ( | |||||||||
Balance as of June 30, 2021 | | $ | | $ | | $ | ( | $ | | |||||
Stock issuance costs | — | — | ( | — | ( | |||||||||
Stock-based compensation | — | — | | — | | |||||||||
Exercise of stock options | | — | | — | | |||||||||
Net loss | — | — | — | ( | ( | |||||||||
Balance as of September 30, 2021 | | $ | | $ | | $ | ( | $ | | |||||
The accompanying notes are an integral part of these financial statements.
8
BIOXCEL THERAPEUTICS, INC.
STATEMENTS OF CASH FLOWS
(amounts in thousands)
(unaudited)
Nine months ended September 30, | ||||||
| 2021 |
| 2020 | |||
CASH FLOWS FROM OPERATING ACTIVITIES: |
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Net loss | $ | ( | $ | ( | ||
Reconciliation of net loss to net cash used in operating activities |
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Depreciation and amortization |
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Loss on disposal of equipment | | — | ||||
Stock-based compensation expense |
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Changes in operating assets and liabilities: |
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Prepaid expenses and other assets |
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Operating lease right of use assets | | | ||||
Accounts payable, accrued expenses, and other liabilities |
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Operating lease liabilities | ( | ( | ||||
Net cash used in operating activities |
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CASH FLOWS FROM INVESTING ACTIVITIES: |
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Purchases of equipment and leasehold improvements |
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Net cash used in investing activities |
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CASH FLOWS FROM FINANCING ACTIVITIES: |
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Proceeds from issuance of common stock, net of issuance costs |
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Purchase and cancellation of shares from BioXcel LLC |
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Exercise of stock options |
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Net cash provided by financing activities |
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Net increase in cash and cash equivalents |
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Cash and cash equivalents, beginning of the period |
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Cash and cash equivalents, end of the period | $ | | $ | | ||
Supplemental cash flow information: |
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Purchases of equipment and leasehold improvements included in accounts payable and accrued expense as of December 31, 2020, but paid in the current period | | — | ||||
Operating lease ROU assets obtained in exchange for operating lease liabilities | — | | ||||
The accompanying notes are an integral part of these financial statements.
9
BIOXCEL THERAPEUTICS, INC.
NOTES TO FINANCIAL STATEMENTS
(amounts in thousands, except per share amounts)
(unaudited)
Note 1. Nature of the Business
BioXcel Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on drug development that utilizes artificial intelligence to identify improved therapies in neuroscience and immuno-oncology. BTI's drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indices. BTI's two most advanced clinical development programs are BXCL501, a proprietary, orally dissolving, thin film formulation of the adrenergic receptor agonist dexmedetomidine (“Dex”), for the treatment of agitation and opioid withdrawal symptoms, and BXCL701, an orally administered, systemic innate immune activator for the treatment of a rare form of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors.
As used in these financial statements, unless otherwise specified or the context otherwise requires, the terms the “Company” or “BTI” refer to BioXcel Therapeutics, Inc., and “BioXcel LLC” refers to BioXcel LLC and, its predecessor, BioXcel Corporation.
The Company was incorporated under the laws of the State of Delaware on March 29, 2017. The Company’s principal office is in New Haven, Connecticut.
The Company incurred losses of $
Impact of COVID-19 Pandemic
In March 2020, the World Health Organization declared the outbreak of COVID-19, a novel strain of coronavirus, a global pandemic. This pandemic has caused and is continuing to cause major disruptions to businesses and financial markets worldwide. This may affect the Company’s operations and those of third parties on which the Company relies, including causing disruptions in the supply of the Company’s product candidates and the conduct of current and planned preclinical and clinical studies. The Company may need to limit its operations and may experience limitations in employee resources. There are risks that the COVID-19 pandemic, or the spread of the variants of the disease, may be more difficult to control than currently anticipated in which case the risks described herein could increase significantly. The extent to which the COVID-19 pandemic impacts the Company’s results will depend on future developments, which are highly uncertain and cannot be predicted, including new information which may emerge concerning the prevalence of variants of the disease, the efficacy and adoption of vaccines, and actions to contain the coronavirus or treat its impact, among others.
Additionally, while the potential economic impact brought by, and the duration of, the COVID-19 pandemic is difficult to assess or predict, the impact of the coronavirus on the global financial markets may reduce the Company’s ability to access capital, which could negatively impact the Company’s short-term and long-term liquidity, and the Company’s ability to complete its preclinical and clinical studies on a timely basis, or at all. The ultimate impact of COVID-19 is highly uncertain and subject to change. The Company does not yet know the full extent of potential delays or impacts on its business, financing, preclinical and clinical trial activities or the global economy as a whole. However, these effects could have a material, adverse impact on the Company’s liquidity, capital resources, operations and business and those of the third parties on which the Company relies.
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Note 2. Basis of Presentation
The accompanying unaudited financial statements do not include all of the information and footnotes required by Generally Accepted Accounting Principles in the United States of America (“GAAP”). The accompanying year-end balance sheet was derived from audited financial statements but does not include all disclosures required by GAAP. The unaudited interim financial statements have been prepared on the same basis as the audited annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s financial position as of September 30, 2021, the results of its operations for the three and nine months ended September 30, 2021 and 2020 and its cash flows for the nine months ended September 30, 2021 and 2020, respectively. The results for the three and nine months ended September 30, 2021 are not necessarily indicative of results to be expected for the year ending December 31, 2021, any other interim periods or any future year or period. The accompanying unaudited financial statements of the Company should be read in conjunction with the audited financial statements and notes as of and for the year ended December 31, 2020 included in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2020 filed with the Securities and Exchange Commission (the “SEC”) on March 12, 2021.
The preparation of financial statements in accordance with GAAP requires management to make estimates and assumptions that affect amounts reported in the financial statements and notes thereto. The Company believes that its existing cash and cash equivalents will be sufficient to cover its cash flow requirements for at least the next twelve months from the issuance of these financial statements. However, the Company’s future requirements may change and will depend on numerous factors.
Note 3. Summary of Significant Accounting Policies
Use of Estimates
The preparation of our financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity and expenses. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making judgments about the carrying values of assets, liabilities and equity and the amount of expenses.
Cash and Cash Equivalents
The Company considers all highly liquid investments with an original maturity of three months or less at the date of purchase to be cash equivalents. As of September 30, 2021 and December 31, 2020, cash equivalents were comprised primarily of U.S. government money market funds. Cash and cash equivalents held at financial institutions may at times exceed federally insured amounts. We believe we mitigate such risk by investing in or through major financial institutions.
Property and Equipment
Property and equipment are recorded at cost and depreciated and amortized over the shorter of their remaining lease term or their estimated useful life on a straight-line basis as follows:
Equipment
Furniture
Leasehold improvements Lesser of life of improvement or lease term
Expenditures for maintenance and repairs which do not improve or extend the useful lives of respective assets are expensed as incurred. When assets are sold or retired, the related cost and accumulated depreciation are removed from their respective accounts and any resulting gain or loss is included in income (loss) from operations.
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The Company follows the guidance provided by the Financial Accounting Standards Board (“FASB”) ASC Topic 360-10, Property, Plant, and Equipment. Long-lived assets are reviewed for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset to undiscounted future net cash flows expected to be generated. Impairment charges are recognized at the amount by which the carrying amount of an asset exceeds the fair value of the asset. Assets to be disposed of are reported at the lower of the carrying amount or the fair value less costs to sell.
Leases
We determine if an arrangement is a lease at inception. Operating leases are included in operating lease right-of-use (“ROU”) assets, other current liabilities, and the long-term portion of operating lease liabilities in our balance sheet.
ROU assets represent our right to use an underlying asset for the lease term and lease liabilities represent our obligation to make lease payments arising from the lease. Operating lease ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. As our lease did not provide an implicit rate, we used an incremental borrowing rate based on the information available at commencement date in determining the present value of lease payments. We use the implicit rate when readily determinable. The operating lease ROU asset also includes any prepaid lease payments made and excludes lease incentives. Our lease terms may include options to extend or terminate the lease when it is reasonably certain that we will exercise that option. Renewal options were not included in our calculation of the related asset and liability. Lease expense for lease payments is recognized on a straight-line basis over the lease term.
Stock-Based Compensation
The Company accounts for stock-based compensation in accordance with ASC 718, “Compensation—Stock Compensation,” which requires the measurement and recognition of compensation expense based on estimated fair market values for all share-based awards made to employees, directors, and non-employees, including stock options. The Company’s 2017 Equity Incentive Plan (the “2017 Plan”) became effective in August 2017. The Company’s 2020 Incentive Award Plan (the “2020 Plan”) became effective in May 2020. Following the effective date of the Company's 2020 Plan, the Company ceased granting awards under the 2017 Plan, however the terms and conditions of the 2017 Plan continue to govern any outstanding awards granted thereunder.
The Company’s stock option awards are valued at fair value on the date of grant and that fair value is recognized over the requisite service period. The estimated fair value of stock option awards was determined using the Black-Scholes option pricing model on the date of grant. Significant judgment and estimates were used to estimate the fair value of awards, granted prior to the Company’s IPO in March 2018. Stock awards granted by the Company subsequent to the IPO are valued using market prices at the date of grant.
ASC 718 requires companies to estimate the fair value of share-based awards on the date of grant using an option-pricing model. The Black-Scholes option-pricing model was used as its method of determining fair value. This model is affected by the Company’s stock price as well as assumptions regarding a number of subjective variables. These subjective variables include, but are not limited to, the expected stock price volatility over the term of the awards, and projected employee stock option exercise behaviors. The value of the award is recognized as an expense in the statement of operations over the requisite service period. The Company has elected to account for forfeitures as they occur, by reversing compensation cost when the award is forfeited.
Research and Development Costs
Research and development expenses include wages, benefits, facilities, supplies, external services, clinical study and manufacturing costs and other expenses that are directly related to the Company’s research and development activities. At the end of the reporting period, the Company compares payments made to third party service providers to the estimated progress toward completion of the research or development objectives. Depending on the timing of payments to the service providers and the progress that the Company estimates has been made as a result of the service
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provided, the Company may record net prepaid or accrued expense relating to these costs. Such estimates are subject to change as additional information becomes available. The Company expenses research and development costs as incurred.
Expenses Accrued Under Contractual Arrangements
As part of the process of preparing our financial statements, we are required to estimate our accrued expenses. This process involves reviewing open contracts and purchase orders, communicating with our applicable personnel to identify services that have been performed on our behalf and estimating the level of service performed and the associated cost incurred for the service when we have not yet been invoiced or otherwise notified of actual cost. The majority of our service providers invoice us monthly in arrears for services performed. We make estimates of our accrued expenses as of each balance sheet date in our financial statements based on facts and circumstances known to us at that time. We periodically confirm the accuracy of our estimates with the service providers and make adjustments if necessary.
We base our expenses related to clinical trials on our estimates of the services received and efforts expended pursuant to contracts with multiple research institutions and contract research organizations that conduct and manage clinical trials on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contract and may result in uneven payment flows. Payments under some of these contracts depend on factors such as the successful enrollment of patients and the completion of clinical trial milestones. In accruing expenses, we estimate the time period over which services will be performed and the level of effort to be expended in each period, which is based on an established protocol specific to each clinical trial. If the actual timing of the performance of services or the level of effort varies from our estimate, we adjust the accrual accordingly. Although we do not expect our estimates to be materially different from amounts actually incurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and may result in us reporting amounts that are too high or too low in any particular period.
Patent Costs
Costs related to filing and pursuing patent applications are recorded as general and administrative expense and expensed as incurred since recoverability of such expenditures is uncertain.
Fair Value of Financial Instruments
The Company applies the provisions of ASC 820, “Fair Value Measurements and Disclosures” for financial assets and liabilities measured on a recurring basis which requires disclosure that establishes a framework for measuring fair value. ASC 820 defines fair value as the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. ASC 820 establishes a fair value hierarchy that distinguishes between (1) market participant assumptions developed based on market data obtained from independent sources, or observable inputs, and (2) an entity’s own assumptions about market participant assumptions developed based on the best information available in the circumstances, or unobservable inputs. The fair value hierarchy consists of three broad levels, which gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1) and the lowest priority to unobservable inputs (Level 3). ASC 820 requires that fair value measurements be classified and disclosed in one of three categories:
Level 1: Quoted prices (unadjusted) in active markets that are accessible at the measurement date for assets or liabilities. The fair value hierarchy gives the highest priority to Level 1 inputs.
Level 2: Directly or indirectly observable inputs as of the reporting date through correlation with market data, including quoted prices for similar assets and liabilities in active markets and quoted prices in markets that are not active. Level 2 also includes assets and liabilities that are valued using models or other pricing methodologies that do not require significant judgment since the input assumptions used in the models, such as interest rates and volatility factors, are corroborated by readily observable data from actively quoted markets for substantially the full term of the financial instrument.
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Level 3: Unobservable inputs that are supported by little or no market activity and reflect the use of significant management judgment. These values are generally determined using pricing models for which the assumptions utilize management’s estimates of market participant assumptions.
In determining fair value, the Company utilizes valuation techniques that maximize the use of observable inputs and minimize the use of unobservable inputs to the extent possible, as well as considering counterparty credit risk in its assessment of fair value.
The carrying amounts of cash and accounts payable approximate fair value due to the short-term nature of these instruments.
As of September 30, 2021 and December 31, 2020, the Company had $
Earnings (Loss) per Share
Basic earnings (loss) per share (“EPS”) is calculated in accordance with ASC 260, “Earnings Per Share,” by dividing net income or loss attributable to common stockholders by the weighted average common stock outstanding. Diluted EPS is calculated by adjusting weighted average common shares outstanding for the dilutive effect of common stock options and warrants. In periods in which a net loss is recorded, no effect is given to potentially dilutive securities, since the effect would be antidilutive. Securities that could potentially dilute basic EPS in the future were not included in the computation of diluted EPS because to do so would have been antidilutive.
Segment Information
The Company operates in a single segment. Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker in making decisions regarding resource allocation and assessing performance. To date, our chief operating decision maker has made such decisions and assessed performance at the company level as
Recent Accounting Pronouncements
Recently adopted accounting pronouncements
In December 2019, the FASB issued ASU No. 2019-12, Income Taxes (Topic 740) which amends the existing guidance relating to the accounting for income taxes. ASU No. 2019-12 is intended to simplify the accounting for income taxes by removing certain exceptions to the general principles of accounting for income taxes and to improve the consistent application of GAAP for other areas of accounting for income taxes by clarifying and amending existing guidance. ASU No. 2019-12 is effective for interim and annual periods beginning after December 15, 2020. The Company adopted ASU No. 2019-12 effective January 1, 2021. The adoption of ASU No. 2019-12 did not have a material impact on the Company’s financial statements.
In August 2018, the FASB issued ASU No. 2018-15, Intangibles—Goodwill and Other—Internal-Use Software (Subtopic 350-40): Customer’s Accounting for Implementation Costs Incurred in a Cloud Computing Arrangement That Is a Service Contract. We adopted this standard effective January 1, 2020. ASU No. 2018-15 requires that certain implementation costs for cloud computing arrangements are capitalized and amortized over the term of associated hosted cloud computing arrangement service and that capitalized implementation costs are classified in prepaid expenses and other assets. ASU No. 2018-15 also provides classification guidance on these implementation costs as well as additional quantitative and qualitative disclosures. The adoption of ASU No. 2018-15 did not have an effect on the Company’s financial statements.
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Accounting Pronouncements effective in future periods
In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments-Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments and subsequent amendments to the initial guidance: ASU No. 2018-19, ASU No. 2019-04 and No. ASU 2019-05 (collectively, “Topic 326”). Topic 326 requires measurement and recognition of expected credit losses for financial assets held. Topic 326 was to be effective for reporting periods beginning after December 15, 2019, with early adoption permitted. In November 2019, the FASB issued ASU No. 2019-10, Financial Instruments - Credit Losses (Topic 326), Derivatives and Hedging (Topic 815), and Leases (Topic 842) Effective Dates, which deferred the effective dates for the Company, until fiscal year 2023. The Company does not expect that the adoption of ASU No. 2016-13 will have a material impact on its financial statements.
Note 4. Financing Activities
In June 2021, the Company sold in a registered offering
In May 2021, the Company entered into an Open Market Sale Agreement (the “Sale Agreement”) with Jefferies LLC (“Jeffries”) pursuant to which the Company could offer and sell shares of its common stock, par value $
In July 2020, the Company sold in a registered offering
In February 2020, the Company sold in a registered offering
Note 5. Transactions with BioXcel LLC
The Company entered into a Separation and Shared Services Agreement with BioXcel LLC that took effect on June 30, 2017, as amended and restated, (the “Services Agreement”), pursuant to which services provided by BioXcel LLC through its subsidiaries in India and the United States will continue indefinitely, as agreed upon by the parties. These services are primarily for drug discovery, chemical, manufacturing and controls cost, and administrative support.
Service charges recorded under this agreement for the three and nine months ended September 30, 2021 and 2020 were comprised as follows:
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||
2021 | 2020 | 2021 | 2020 | |||||||||
Research and development |
| $ | | $ | | $ | | $ | | |||
General and administrative |
| | |
| | | ||||||
Total | $ | | $ | | $ | | $ | | ||||
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As of September 30, 2021, $
Under the Services Agreement, the Company has an option, exercisable until March 12, 2023, to enter into a collaborative services agreement with BioXcel LLC pursuant to which BioXcel LLC shall perform product identification and related services for us utilizing EvolverAI. As of September 30, 2021, this option has not been exercised.
Note 6. Earnings (Loss) Per Share
Basic earnings (loss) per share (“EPS”) is calculated in accordance with ASC 260, “Earnings Per Share,” by dividing net income or loss attributable to common stockholders by the weighted average common stock outstanding. Diluted EPS is calculated by adjusting weighted average common shares outstanding for the dilutive effect of common stock options and warrants. In periods in which a net loss is recorded, no effect is given to potentially dilutive securities, since the effect would be antidilutive. Securities that could potentially dilute basic EPS in the future were not included in the computation of diluted EPS because to do so would have been antidilutive. The calculations of basic and diluted net loss per share are as follows (in thousands, except per share amounts):
Three Months Ended | Nine Months Ended | |||||||||||
| September 30, | September 30, | ||||||||||
2021 |
| 2020 | 2021 |
| 2020 | |||||||
Net loss (numerator) | $ | ( | $ | ( | $ | ( | $ | ( | ||||
Weighted average share, in thousands (denominator) | | | | | ||||||||
Basic and diluted net loss per share | $ | ( | $ | ( | $ | ( | $ | ( | ||||
Potentially dilutive securities outstanding consists solely of stock options. The Company had options outstanding to purchase
Note 7. Property and Equipment, net
Property and Equipment, net consisted of the following
| September 30, |
| December 31, | |||
| 2021 |
| 2020 | |||
Computers and related equipment | $ | | $ | | ||
Furniture | | | ||||
Leasehold improvements | | | ||||
Work in process | — | | ||||
| | |||||
Accumulated depreciation | ( | ( | ||||
$ | | $ | | |||
Depreciation expense was $
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Note 8. Accrued Expenses and Other Current Liabilities
Accrued expenses consist of the following:
| September 30, 2021 |
| December 31, 2020 | |||
Research and development expenses | $ | | $ | | ||
Accrued compensation and benefits | | | ||||
Accrued professional expenses |
| |
| | ||
Accrued taxes | | | ||||
Other accrued expenses |
| |
| | ||
$ | | $ | | |||
Other current liabilities as of September 30, 2021 and December 31, 2020 consists of $
Note 9. Stock-Based Compensation
2017 Equity Incentive Plan
The Company’s 2017 Plan became effective in August 2017. Following the effective date of the Company's 2020 Plan (as defined below), the Company ceased granting awards under the 2017 Plan, however the terms and conditions of the 2017 Plan continue to govern any outstanding awards granted thereunder.
2020 Incentive Award Plan
The Company’s 2020 Plan was approved and became effective at the Company’s 2020 annual meeting of stockholders on May 20, 2020 and unless, earlier terminated by the Board of Directors, will remain in effect until March 26, 2030. The 2020 Plan originally authorized for issuance the sum of (i)
Options granted under the 2020 Plan have a term of
As of September 30, 2021, there were
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A summary of the status of the Company’s stock option activity for the nine months ended September 30, 2021 is presented below (in thousands, except per share amounts):
Number | Weighted Average | ||||
of | Exercise | ||||
| Shares |
| Price per Share | ||
Outstanding as of January 1, 2021 |
| | $ | | |
Granted | | $ | | ||
Forfeited | ( | $ | | ||
Cancelled | ( | $ | | ||
Exercised | ( | $ | | ||
Outstanding as of September 30, 2021 | | $ | | ||
Options vested and exercisable as of September 30, 2021 |
| | $ | | |
As of September 30, 2021, the intrinsic value of options outstanding was $
The total intrinsic value of stock options exercised for the nine months ended September 30, 2021 and 2020 was $
The weighted average grant date fair value of options granted during the nine months ended September 30, 2021 and 2020 was $
The weighted average grant date fair value of options vested at September 30, 2021 was $
The weighted average remaining contractual life is
Stock-Based Compensation
The fair value of options granted during the nine months ended September 30, 2021 and 2020 was estimated using the Black-Scholes option-pricing model with the following assumptions.
For the | For the | ||||||||||||
Nine Months Ended | Nine Months Ended | ||||||||||||
| September 30, 2021 | September 30, 2020 | |||||||||||
Expected Term | years | - | years | years | - | years | |||||||
Expected stock price volatility | % | - | % | % | - | % | |||||||
Risk-free rate of interest | % | - | % | % | - | % | |||||||
Expected dividend | % | - | % | % | - | % | |||||||
Prior to the Company’s IPO, it did not have a history of market prices of its common stock and, as such, volatility was estimated using historical volatilities of similar public companies. In 2021, the Company began using a combination of the historical volatility of similar public companies and the limited historical information related to the Company’s common stock. The expected term of the employee awards is estimated based on the simplified method, which calculates the expected term based upon the midpoint of the term of the award and the vesting period. The Company uses the simplified method because it does not have sufficient option exercise data to provide a reasonable basis upon which to estimate the expected term. The expected dividend yield is
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Unrecognized compensation expense related to unvested awards as of September 30, 2021 was $
Total stock-based compensation charges for the three and nine months ended September 30, 2021 and 2020 were comprised as follows:
Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||
2021 | 2020 | 2021 | 2020 | |||||||||
Research and development |
| $ | | $ | | $ | | $ | | |||
General and administrative |
| | |
| | | ||||||
Total | $ | | $ | | $ | | $ | | ||||
2020 Employee Stock Purchase Plan
The Company’s 2020 Employee Stock Purchase Plan (the “ESPP”) was also approved and became effective at the Company’s 2020 annual meeting of stockholders on May 20, 2020. The ESPP is designed to assist eligible employees of the Company with the opportunity to purchase the Company’s common stock at a discount through accumulated payroll deductions during successive offering periods. The aggregate number of shares that may be issued pursuant to rights granted under the ESPP is
Note 10. Leases
In August 2018, the Company entered into an agreement to lease approximately
In August 2020, the Company entered into an amendment to the 12th Floor Lease wherein the Company leased an additional
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The future minimum annual lease payments under these operating leases as of September 30, 2021 are as follows:
Year ending December 31, |
| Amount | |
Remainder of 2021 | | ||
2022 | | ||
2023 | | ||
2024 | | ||
2025 | | ||
Thereafter | | ||
Total lease payments |
| | |
Less imputed interest | ( | ||
Total lease liability | | ||
Less current portion of lease liability | ( | ||
Long-term portion operating lease liability | $ | | |
The portion of the Company’s operating lease liability of $
The Company recorded lease expense of $
The Company has an
Note 11. Commitments and Contingencies
From time to time, in the ordinary course of business, the Company may be subject to litigation and regulatory examinations as well as information gathering requests, inquiries and/or investigations. The Company is not currently subject to any matters where it believes there is a reasonable possibility that a material loss may be incurred.
The Company received a demand letter pursuant to Section 220 of the Delaware General Corporation Law (“DGCL”) from a stockholder seeking disclosure of certain of the Company's records. The Company responded to those demands, stating its belief that the demand letter failed to fully comply with the requirements of Section 220 of the DGCL. On June 15, 2021, the stockholder filed a complaint in Delaware Chancery Court seeking to compel inspection of books and records pursuant to Section 220 of the DGCL. Pursuant to a negotiated settlement agreement, the matter was dismissed with prejudice on August 10, 2021.
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Item 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS
You should read the following discussion and analysis of our financial condition and results of operations together with our financial statements and related notes appearing elsewhere in this Quarterly Report on Form 10-Q and the audited financial statements and related notes contained in our Annual Report on Form 10-K for the year ended December 31, 2020. Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report, such as information with respect to our plans and strategy for our business and expectations related to the clinical development of our product candidates, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the “Summary Risk Factors” and “Risk Factors” sections of this Quarterly Report, our actual results could differ materially from the results described in, or implied by, the forward-looking statements contained in the following discussion and analysis. All dollar amounts in the below Management’s Discussion and Analysis of Financial Condition and Results of Operations are presented in U.S. dollars, and all dollar amounts are presented in thousands, unless otherwise noted or the context otherwise provides. All share amounts are also presented in thousands.
Overview
We are a clinical stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology. Our drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indices. We believe this differentiated approach has the potential to reduce the cost and time of drug development in diseases with a substantial unmet medical need.
Our two most advanced clinical development programs are BXCL501, an investigational proprietary, orally dissolving, thin film formulation of the adrenergic receptor agonist dexmedetomidine (“Dex”), for the treatment of agitation associated with psychiatric and neurological disorders, and BXCL701, an investigational orally administered systemic innate immune activator for the treatment of a rare form of prostate cancer and advanced solid tumors that are refractory or treatment naïve to checkpoint inhibitors.
During the first quarter of 2020 the novel coronavirus disease, or COVID-19, was declared a pandemic and has continued to spread to multiple regions across the globe, including the United States and Europe. The pandemic and government measures taken in response have significantly impacted, both directly and indirectly, businesses and commerce, as worker shortages have occurred; supply chains have been disrupted; facilities and production have been suspended; and demand for certain goods and services, such as medical services and supplies, has spiked, while demand for other goods and services, such as travel, has fallen.
In 2020 and 2021, we took steps in line with guidance from the U.S. Centers for Disease Control and Prevention (“CDC”) and the State of Connecticut to protect the health and safety of our employees and community. In particular, we implemented a work-from-home policy for all employees. We continue to assess the impact of the COVID-19 pandemic to best mitigate risk and continue business operations. Beginning late in the first quarter of 2021, and in line with State of Connecticut guidelines, we opened our office on a voluntary basis up to full capacity. In May 2021 the State of Connecticut lifted all business and office restrictions in the State. We have instituted a return to the office policy and continue to evaluate that policy.
We continue to work closely with our clinical sites to monitor the potential impact of the evolving COVID-19 pandemic and the spread of its variants. We remain committed to our clinical programs and development plans. Through September 30, 2021, we have not experienced any significant delays to our ongoing or planned clinical trials, except for challenges in accessing elderly care facilities and ICU settings. However, this could rapidly change.
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Our Clinical Programs
The following is a summary of the status of our clinical development programs as of the date of this Quarterly Report on Form 10-Q:
BXCL501 Neuroscience Program
BXCL501 is an investigational, proprietary, orally dissolving, thin film formulation of Dex, a selective alpha-2a receptor agonist, targeting symptoms from stress-related behaviors such as agitation and opioid withdrawal symptoms. BXCL501 is our most advanced neuroscience clinical program, currently being developed for the acute treatment of agitation related to schizophrenia, bipolar disorders, dementia and delirium, the symptoms associated with opioid withdrawal as well as for the treatment of major depressive disorder (MDD) in conjunction with the use of Selective Serotonin / Norepinephrine Reuptake Inhibitors (“SS/NRIs”). As a selective adrenergic agent with a sublingual or buccal route of administration, BXCL501 is designed to be easily administered and has shown a rapid onset of action in multiple clinical trials, including clinical trials studying patients with schizophrenia, bipolar disorders, dementia and opiate use disorder. We believe results from these studies suggest that BXCL501 has the potential to generate a calming effect without producing excessive sedation. We also believe BXCL501 is highly differentiated from antipsychotics, which often produce unwanted side effects such as excessive sedation or extra pyramidal motor effects, currently used as a standard of care to treat agitation. Managing patient agitation in neuropsychiatric and neurodegenerative disorders represents a significant challenge for physicians and caregivers. We believe BXCL501 has the potential to address these challenges while providing an efficient treatment regimen for patients.
BXCL501 Clinical Trials
SERENITY I and SERENITY II
In May 2021, we announced the FDA had notified us that our NDA submission was accepted for filing. The FDA assigned a Prescription Drug User Fee Act (“PDUFA”) date of January 5, 2022. During the quarter we continued to address information requests from the FDA. We also have plans underway to submit a Marketing Authorization Application (“MAA”) to the European Medicines Agency (“EMA”) for BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders I & II in the first half of 2022.
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TRANQUILITY
In January 2021, we announced topline results from the TRANQUILITY trial, a phase 1b/2 randomized, placebo-controlled, adaptive ascending dose-finding study that enrolled 54 patients with agitation related to dementia. Overall, BXCL501 was well tolerated and demonstrated statistically significant, clinically meaningful, rapid, and durable reductions in agitation with the 60 mcg dose as measured by multiple scales.
With positive results from TRANQUILITY, we have been advancing the BXCL501 dementia clinical program in close consultation with the FDA.
We held an end of Phase 2 meeting with the FDA in May 2021 to discuss the BXCL501 program for the acute treatment of agitation in dementia patients. Over the last several months we have had multiple interactions with the FDA, to further discuss development plans and align on key design features for the pivotal Phase 3 program. We currently plan to commence the BXCL501 Phase 3 program for the acute treatment of agitation in patients with Alzheimer’s disease in the fourth quarter of 2021. Alzheimer’s disease is the most prevalent type of dementia in the United States.
RELEASE
The RELEASE trial was a multicenter, randomized, double-blind, placebo-controlled, ascending dose Phase 1b/2 trial designed to evaluate the safety, pharmacokinetics, tolerability, and efficacy of escalating doses of BXCL501 versus placebo, following discontinuation of morphine maintenance in patients with opioid use disorder who are physically dependent on opioids.
BXCL501 was well tolerated, with no severe or serious adverse events reported across all doses evaluated. Although improvements were not observed in group mean total efficacy scores, the study showed numerical improvements in retention rates in multiple BXCL501 dose cohorts compared to the placebo.
We believe the favorable tolerability results observed in multiple dose regimens within the RELEASE trial provide valuable insights that support investigation across additional indications and treatment settings.
PLACIDITY
Agitation associated with delirium is a serious condition that affects patients in many hospital settings: ICUs, surgical and medical wards, and emergency departments. Currently, there are no FDA-approved medications for delirium or agitation associated with delirium.
On February 25, 2021, we announced initiation of the Phase 2 PLACIDITY trial of BXCL501. PLACIDITY enrollment was voluntarily paused to assess challenges posed in opening relevant clinical sites and enrolling delirium patients in ICU settings. These challenges include the burden COVID-19 has placed on the ICU. The PLACIDITY trial status has not changed.
PEDIATRIC STUDY
In June 2021, we initiated a global clinical trial designed to determine the safety and efficacy of BXCL501 in agitation associated with pediatric schizophrenia and bipolar disorder. The trial has been reviewed by the FDA to ensure we meet the Pediatric Equity Research Act (“PREA”) requirements as well as the EMA to fulfill commitments to study the effects of BXCL501 in children, ages 10-17. The multisite double-blind placebo controlled parallel group trial will enroll patients with schizophrenia, schizoaffective disorder, bipolar I and bipolar II disorder. Similar to the SERENITY I and II trials, the primary endpoint is the change from baseline PEC total score at 2 hours. The trial has been initiated in the U.S. and patients are currently enrolling in various sites. Clinical trial authorizations and ethics committee approvals have been obtained in Europe and sites are now being activated.
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Major Depressive Disorder (MDD)
We have recently expanded the market potential of BXCL501 as an adjunctive treatment for Major Depressive Disorder (“MDD”). In October 2021, we held a pre-IND meeting with the FDA to discuss the clinical strategy to evaluate the use of BXCL501 in conjunction with patients receiving SS/NRIs. We are preparing to submit an IND to the FDA and expect to initiate the first clinical trial in the first half of 2022.
Additional Opportunities
In December 2020, the VA Connecticut Healthcare System and Yale University Medical School were awarded a grant by the U.S. Department of Defense’s Congressionally Directed Medical Research Programs to evaluate BXCL501 in patients suffering from post-traumatic stress disorder related to alcohol and substance abuse disorder. For the first time we will be investigating BXCL501 as a potential chronic treatment.
We continue to explore the use of a wearable device to measure early signs of agitation in subsets of patients who are part of BXCL501 clinical trials. Data is obtained by the wearable device, collected on other electronic devices such as phones, and transmitted to our service vendor for analysis. Feasibility studies demonstrated that patients were able to wear these devices and that these devices were able to transmit data to our service vendor. Along with our service vendor, we are establishing methods to use these data to construct algorithms designed to predict when a patient is becoming agitated. The goal of this program is to establish a predictive algorithm that allows providers to dose patients before they become highly agitated.
Other Neuropsychiatric / Neurodegenerative Indications and Formulations
Given the differentiated design of BXCL501 and its selective mechanism of action, we believe BXCL501 has the potential for broad applicability across several indications where agitation is a symptom of a condition or underlying disease. There are additional neuropsychiatric disorders where agitation is a symptom that requires treatment.
We recently identified and initiated the development of a second neuropsychiatric drug candidate, BXCL502. We plan to evaluate BXCL502 initially as a monotherapy and possibly as a combination with BXCL501 for the chronic treatment of agitation in patients with dementia. The active pharmaceutical ingredient (“API”) underlying BXCL502 is designed to be a potent and selective antagonist of a G-protein coupled receptor (“GPCR”) that affects serotonergic signaling in the brain. Our preclinical data and machine learning results suggests BXCL502 has potential to treat agitation and stress related neuropsychiatric symptoms in dementia. In previously published third party clinical trial data, daily administration of the API of BXCL502 demonstrated improvement in agitation using a well-established, clinically validated symptom scale. Formulation and clinical development planning are currently underway with BXCL502.
Commercial and Launch Readiness
We continue to advance our preparations for commercial and launch readiness if our NDA for BXCL501 is approved by the FDA. We are optimizing the design and recruitment of our sales force and market access and pricing strategy for the potential commercial launch of BXCL501 for the acute treatment of agitation associated with schizophrenia and bipolar disorders I & II. This work will be a crucial foundation to launching additional potential follow-on indications, paving the way for our expanding neuroscience business.
During the third quarter of 2021, we fully launched our unbranded disease education campaign to promote awareness around the treatment of agitation in schizophrenia and bipolar disorders. In addition, we fully deployed our Medical Science Liaison and Medical Managed Care teams who continued to actively engage with healthcare professionals and payors to provide key insights to support our commercial strategy.
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BXCL701 Immuno-Oncology Program
BXCL701 is a potential first-in-class, oral, small-molecule immunomodulator designed to stimulate the innate and acquired immune systems by inhibiting DPP 8/9 (dipeptidyl peptidases). DPP 8/9 behave as "checkpoints" of the innate immune system. We believe that BXCL701, if successfully developed and approved, may establish a differentiated immuno-oncology platform by modulating multiple steps in the cancer immunity cycle and, when combined with other checkpoint inhibitors and/or immune activating agents, may be able to convert immuno-resistant tumors to immuno-sensitive tumors (“cold” to “hot” tumors).
BXCL701 Clinical Trials
BXCL701 is currently being evaluated in two combination therapy clinical trials:
In April 2020, we announced the initiation of the Phase 2 efficacy portion of the Phase 1b/2 trial evaluating BXCL701 in combination with KEYTRUDA® (pembrolizumab, a PD-1 inhibitor) for small-cell neuroendocrine carcinoma (“SCNC”) / neuro endocrine prostate cancer (“NEPC”). The Phase 1b safety assessment of BXCL701 identified a split dose totaling 0.6 mg per day as the recommended dose when used in combination with KEYTRUDA.
In addition to the efficacy cohort in SCNC patients, in August 2020, we opened a separate cohort for adenocarcinoma, a subtype of metastatic castration resistant prostate cancer (“mCRPC”) patients who have failed taxane-based chemotherapy and up to two lines of second-generation androgen pathway blockers. The Phase 1b portion of this trial was presented at the Society for Immunotherapy of Cancer’s 35th Anniversary Annual Meeting in 2020, and an efficacy update was recently presented at the American Society of Clinical Oncology Genitourinary Symposium in February 2021.
In October 2021, we announced the SCNC / NEPC cohort of the ongoing Phase 1b/2 trial of BXCL701 and KEYTRUDA® in aggressive forms of prostate cancer met the protocol specified threshold to move to stage two. We expect to report updated data from the Phase 2 portion of this trial in SCNC or NEPC and adenocarcinoma or mCRPC cohorts in Q1 2022.
In May 2021, we announced the adenocarcinoma cohort of the ongoing Phase 1b/2 trial of BXCL701 and KEYTRUDA® in aggressive forms of prostate cancer met the protocol specified efficacy threshold to move to stage two. Data from the Phase 2 portion was presented at the European Society for Medical Oncology conference in September 2021. The trial is now continuing towards full enrollment.
The MD Anderson-led Phase 2 open-label basket trial is designed to evaluate the response rate of orally administered BXCL701, combined with KEYTRUDA, in two arms: Arm A is enrolling checkpoint naïve patients (where checkpoint therapy is indicated: “hot tumors”); and Arm B is enrolling patients who have progressed following checkpoint therapy alone. As of August 2020, the efficacy threshold had been met for both arms, allowing the trial to advance to completion. Preliminary data was presented at the June 2021 American Society of Clinical Oncology meeting. In the first half of 2022, we expect to present additional efficacy data from the trial.
Other Immuno-oncology Indications
In addition to CRPC and checkpoint inhibitor (“CPI”) refractory tumors, we plan to leverage our existing preclinical and clinical data to identify other cancer types with high unmet medical need that we believe would benefit from BXCL701’s novel potential mechanism of action. We are prioritizing indications where the immuno-suppressive microenvironment is driven by the potential molecular and cellular targets of BXCL701 and where the single agent activity of approved immune checkpoint inhibitors is limited. In January 2021, we announced that BXCL701 was granted Orphan Drug Designation for the treatment of soft tissue sarcoma.
In addition, we believe BXCL701, if successfully developed and approved, may provide a platform for combination with immunotherapy modalities that go beyond currently approved immune checkpoint agents targeting
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the PD-1/PD-L1 axis. Following our proof-of-concept trials, we plan to conduct clinical trials covering a broad range of additional combinations with other immunotherapy agents, including:
| ● | immune checkpoint inhibitors (other than PD-1/PD-L1); |
| ● | cellular therapies (CAR-T and chimeric antigen receptor natural killer cells); and |
| ● | ADCC-driven monoclonal antibodies. |
Recent Research Findings
On November 4, 2021, the Journal of Immunotherapy of Cancer reported data findings suggesting BXCL701 may enhance immunotherapy efficacy in ‘cold’ tumor types such as pancreatic cancer. These findings also highlighted the potential importance of natural killer (NK) cells along with T cells in regulating pancreatic cancer tumor growth.
Other Product Candidates
Neuroscience Program
We are targeting neuropsychiatric disorders with high unmet medical needs. Our focus is on treating stress related symptoms, such as agitation, that are responsible for higher levels of institutionalized care. We are also using machine learning to identify new approaches for rare neurological diseases.
We utilize proprietary algorithms to identify associated mechanisms with existing pharmacology to test whether these agents can improve the disease profile in the animal model either through disease modification or symptomatic manner. The agents identified must be those we believe can enter the clinic with the potential for an efficient development path.
Immuno-oncology Program
Our immuno-oncology program is based on utilizing a comprehensive map of all currently known relationships that link immuno-evasion and immuno-activation pathways and targets with thousands of pharmacological agents and tumor indications. This comprehensive map has permitted us to select a potential pipeline of candidates based on our ability to alter the tumor micro-environment and the potential to address relevant unmet medical needs for various tumor types.
Finally, we continually leverage the artificial intelligence platform to select and prioritize additional development opportunities to expand the current portfolio and broaden the addressable market for our lead programs through the identification of new indications. This includes exploring additional combination therapy approaches to expand BXCL701’s target indications beyond NEPC and CRPC.
Intellectual Property
Our policy is to protect and enhance the proprietary technologies, inventions, and improvements that are commercially important to our business by filing patent applications in the United States and other jurisdictions related to our proprietary technology, inventions, improvements, and product candidates. We also rely on trademarks, trade secrets, and know-how relating to our proprietary technologies and product candidates, continuing innovation, and in-licensing technology and products. This reliance is expected to develop, maintain, and strengthen, our proprietary position for novel therapeutics and novel formulations of existing therapeutics across multiple therapeutic areas. We also plan to rely on data exclusivity, market exclusivity, and patent term extensions when available.
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Patent Portfolio
As of October 18, 2021, our patent portfolio included 5 Patent Cooperation Treaty (PCT) applications, 11 U.S. utility applications, 1 issued U.S. utility patent, 13 U.S. provisional patent applications, 77 pending non-U.S. applications, 8 allowed or granted non-U.S. patents, 1 design patent application, which is a U.S. design application, and 34 allowed or registered design patents. U.S. Pat. No. 10,792,246, directed to our proprietary sublingual thin-film formulation of Dex, was issued on October 6, 2020 and has a term set to expire no earlier than 2039. We plan to list the U.S. patent in the FDA's Approved Drug Products with Therapeutic Equivalence Evaluations, or Orange Book. We have filed applications in the core patent family protecting BXCL501 in the United States, Taiwan, and other major markets. We expect that patents issuing from these applications, if any, will expire no earlier than 2039. We have also filed applications in additional patent families that are relevant to BXCL501. We have applications pending in the United States, Europe and Japan directed to methods of treating insomnia using sublingual Dex. We expect that patents issuing from these applications, if any, will expire no earlier than 2035. We also have applications filed in 15 regions, including the United States, Europe, Japan, and China, directed to methods of treating agitation. We expect that patents issuing from these applications, if any, will expire no earlier than 2037. We have 1 U.S. application and 1 European application directed to intravenous administration of Dex. We expect that patents issuing from these applications, if any, will expire no earlier than 2039. We continue to file new applications on an ongoing basis, including provisional applications directed to treating mania and dementia. If patents issue from those cases, we expect them to expire no earlier than 2041 and 2042, respectively.
We have multiple patent families filed to protect our BXCL701 program, including our core patent family directed to methods of using BXCL701 with immune checkpoint inhibitors, which is filed in the United States and 14 other countries. Any patents issuing from that family should expire no earlier than 2036. We have a PCT application directed to combination therapies using BXCL701 with immune checkpoint inhibitors and approaches for modifying T-cell activity. We expect any patents issuing from this family to expire no earlier than 2038. Additional PCT and ex-US applications are directed to administering BXCL701 in combinations with various other molecules and dosing regimens. We expect that patents issuing from these applications, if any, will expire no earlier than 2039. Finally, we have multiple provisional applications directed to various dosing regimens and combination therapies. Any patents issuing from those applications are expected to expire between 2039 and 2041 at the earliest.
The term of individual patents depends upon the legal term for patents in the countries in which they are obtained. In most countries, including the United States, the patent term is 20 years from the earliest filing date of a non-provisional patent application. Depending upon the timing, duration, and specifics of FDA approval of our product candidates, a U.S. patent we own or license may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Act of 1984, referred to as the Hatch-Waxman Act. The act permits a patent restoration term of up to five years as compensation for patent term lost during product development and the drug approval regulatory review process. However, patent term restoration cannot extend the remaining term of a patent beyond a total of 14 years from the product’s approval date. The patent term restoration period is generally one-half the time between the effective date of an IND, and the submission date of a NDA, plus the time between the submission date of an NDA and the approval of that application. Only one patent applicable to an approved drug is eligible for the extension, and the application for extension must be made prior to patent expiration. The U.S. Patent and Trademark Office, in consultation with the FDA, reviews and approves the application for any patent term extension or restoration. In the future, we intend to apply for restorations of patent term for some of our currently owned or licensed patents to add patent life beyond their current expiration date, depending on the expected length of clinical trials and other factors involved in the submission of the relevant NDA.
The patent positions of companies such as ours are generally uncertain and involve complex legal and factual questions. No consistent policy regarding the scope of claims allowable in patents in the field of method of use patents or reformulation patents has emerged in the United States. Relevant patent laws and their interpretation outside of the United States are also uncertain. Changes in either the patent laws or their interpretation in the United States and other countries may diminish our ability to protect our technology or product candidates and enforce the patent rights that we license, and also could affect the value of such intellectual property. In particular, our ability to stop third parties from making, using, selling, offering to sell, or importing products that infringe our intellectual property will depend in part on our success in obtaining and enforcing patent claims that cover our technology, inventions, and improvements. With
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respect to both licensed and company owned intellectual property, we cannot guarantee that patents will be granted with respect to any of our pending patent applications or with respect to any patent applications we may file in the future, nor can we be sure that any patents that may be granted to us in the future will be commercially useful in protecting our products, the methods of use, or the manufacture of those products. Patent and other intellectual property rights in the pharmaceutical and biotechnology space are evolving and involve many risks and uncertainties. For example, third parties may have blocking patents that could be used to prevent us from commercializing our product candidates and practicing our proprietary technology, and the issued patents that we in-license and those that may issue in the future may be challenged, invalidated, or circumvented, which could limit our ability to stop competitors from marketing related products or could limit the term of patent protection that otherwise may exist for our product candidates. In addition, the scope of the rights granted under any issued patents may not provide us with protection or competitive advantages against competitors with similar technology. Furthermore, our competitors may independently develop similar technologies outside the scope of the rights granted under any issued patents that we own or exclusively in license. For these reasons, we may face competition with respect to our product candidates. Moreover, because of the extensive time required for development, testing, and regulatory review of a potential product, it is possible that, before any particular product candidate can be commercialized, any patent protection for such product may expire or remain in force for only a short period following commercialization, thereby reducing the commercial advantage the patent provides.
Basis of Presentation
The Company’s financial statements are prepared in accordance with Generally Accepted Accounting Principles in the United States of America (“GAAP”). All amounts are presented in thousands.
Components of Our Results of Operations
Revenues
We have not recognized any revenue since inception.
Operating Costs and Expenses
Research and Development
Our research and development expenses reflect costs incurred for the research and development of our clinical and pre-clinical product candidates. Research and development expense primarily consist of salary, benefits and non-cash stock-based compensation for our research and development personnel, costs incurred under agreements with contract research organizations (“CROs”) and sites that conduct our non-clinical studies and clinical trials, costs of outside consultants engaged in research and development activities, including their fees, stock-based compensation and travel expenses, the cost of acquiring, developing and manufacturing pre-clinical and clinical trial materials and lab supplies, and depreciation and other expenses.
We expense research and development costs to operations as incurred.
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Our research and development costs by program for the three and nine months ended September 30, 2021 and 2020 were as follows:
Three Months Ended | Nine Months Ended | ||||||||||
September 30, | September 30, | ||||||||||
2021 | 2020 | 2021 | 2020 | ||||||||
Direct external costs | |||||||||||
BXCL501 | $ | 2,652 | $ | 9,114 | $ | 12,613 | $ | 30,524 | |||
BXCL701 |
| 3,312 | 1,948 |
| 8,520 | 4,917 | |||||
Other research and development programs |
| 564 | 181 |
| 1,231 | 538 | |||||
Total direct external costs | 6,528 | 11,243 | 22,364 | 35,979 | |||||||
Internal personnel costs | 4,660 | 4,393 | 16,080 | 9,067 | |||||||
Sub-total direct costs | 11,188 | 15,636 | 38,444 | 45,046 | |||||||
Indirect costs and overhead |
| 772 | 681 | 2,101 | 1,549 | ||||||
Research and development tax credit | (27) | — | (362) | — | |||||||
Total research and development expenses | $ | 11,933 | $ | 16,317 | $ | 40,183 | $ | 46,595 | |||
General and Administrative
General and administrative expenses primarily consist of salaries, benefits and non-cash stock-based compensation for our executive and administrative personnel. General and administrative expenses also include legal expenses to pursue patent protection of our intellectual property, professional fees for audit and tax and insurance charges.
We expect that our general and administrative expenses will increase to enable us to support our operations as we continue to expand our clinical programs. We also expect increased administrative costs resulting from our clinical trials and the potential commercialization of our product candidates. We believe that these increases will likely include increased costs for director and officer liability insurance, hiring additional personnel to support future market research and future product commercialization efforts and increased fees for outside consultants, attorneys and accountants. We may also incur increased costs to comply with corporate governance, internal controls, investor relations and similar requirements applicable to public companies.
Recently Issued Accounting Pronouncements
A description of recently issued accounting pronouncements that may potentially impact our financial position and results of operations is set forth in Note 3 to the financial statements included in this Quarterly Report on Form 10-Q.
Summary of Results of Operations
Comparison of the Three Months Ended September 30, 2021 and 2020
Revenues
We have not recognized any revenues since inception.
Research and Development Expense
Research and development expenses for the three months ended September 30, 2021 and 2020 were $11,933 and $16,317, respectively. Research and development expenses for the three months ended September 30, 2021 and 2020 were comprised as follows:
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Three Months Ended | |||||||||||||
September 30, | |||||||||||||
| 2021 |
| 2020 |
| Change | % Change | |||||||
Personnel and related costs | $ | 3,710 | $ | 2,190 | $ | 1,520 | 69 | % | |||||
Non-cash stock-based compensation |
| 951 |
| 2,203 |
| (1,252) | (57) | % | |||||
Professional fees |
| 2,856 |
| 2,269 |
| 587 | 26 | % | |||||
Clinical trials expense |
| 3,483 |
| 8,479 |
| (4,996) | (59) | % | |||||
Chemical, manufacturing and controls cost ("CMC") |
| 353 |
| 859 |
| (506) | (59) | % | |||||
Travel and other costs |
| 607 |
| 317 |
| 290 | 91 | % | |||||
Research and development tax credit | (27) | — | (27) | (100) | % | ||||||||
Total research and development expenses | $ | 11,933 | $ | 16,317 | $ | (4,384) | (27) | % | |||||
The decrease of $4,384 for the three months ended September 30, 2021 is primarily attributable to:
| ● | Decreased non-cash stock-based compensation due to the reversal of expense related to forfeitures during the period. |
| ● | Decreased clinical trial expense primarily related to decreased costs related to our SERENITY I and II, TRANQUILTY, RELEASE and BXCL501 bioavailability clinical trials, partially offset by increased costs related to our BXCL701 prostate cancer clinical trial. |
| ● | Decreased CMC costs largely related to decreased BXCL501 manufacturing costs. |
These decreases were partially offset by:
| ● | Increased personnel costs related to our efforts to enlarge our clinical team as we expand our clinical programs and in preparation of the potential commercial launch of BXCL501 in the U.S. |
| ● | Increased professional fees primarily due to increased fees related to toxicology studies and consulting fees related to BXCL501. |
| ● | Increased travel and other costs to support the growing clinical team as we expand our clinical programs. |
The State of Connecticut provides companies with the opportunity to exchange certain research and development credit carryforwards for cash in exchange for foregoing the carryforward of the research and development credit. The program provides for such exchange of the research and development credit at a rate of 65% of the annual research and development credit. The benefit for such exchange is recorded as a reduction of research and development expenses.
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General and Administrative Expense
General and administrative expenses for the three months ended September 30, 2021 and 2020 were $14,879 and $8,451, respectively. General and administrative expenses for the three months ended September 30, 2021 and 2020 were comprised as follows:
Three Months Ended |
| ||||||||||||
September 30, |
| ||||||||||||
| 2021 |
| 2020 |
| Change |
| % Change | ||||||
Personnel and related costs | $ | 2,702 | $ | 1,022 | $ | 1,680 | 164 | % | |||||
Non-cash stock-based compensation |
| 3,935 | 3,065 |
| 870 | 28 | % | ||||||
Professional fees |
| 2,575 | 1,361 |
| 1,214 | 89 | % | ||||||
Commercial | 4,299 | 963 | 3,336 | 346 | % | ||||||||
Insurance |
| 560 | 442 |
| 118 | 27 | % | ||||||
Travel and other costs |
| 808 | 1,598 |
| (790) | (49) | % | ||||||
Total general and administrative expenses | $ | 14,879 | $ | 8,451 | $ | 6,428 | 76 | % | |||||
The increase of $6,428 for the three months ended September 30, 2021 is primarily attributable to:
| ● | Increased personnel costs due to our continuing efforts to expand our teams in preparation of the potential commercial launch of BXCL501 in the U.S. |
| ● | Increased non-cash stock-based compensation primarily resulting from an increase in personnel. |
| ● | Increased professional fees due to the expanding growth of our operations and was primarily related to increased corporate legal fees and increased consulting costs related to our preparation for the potential commercial launch of BXCL501 in the U.S. |
| ● | Increased commercial related fees primarily related to our unbranded agitation awareness campaigns, planned BXCL501 marketing campaigns and overall preparation for the potential commercial launch of BXCL501 in the U.S. |
| ● | Increased insurance costs primarily related to an increase in Director and Officer liability premiums. |
These increases were partially offset by:
| ● | Decreased travel and other costs. An e-mail-based wire fraud incident in September 2020, which led to the misappropriation of approximately $1,927. Of this amount, $774 was subsequently recovered. As such, $1,153 was included in travel and other costs in 2020. This was partially offset by an increase in travel and other overhead expense to support the growing company and preparation for the potential commercial launch of BXCL501 in the US. |
Comparison of the Nine Months Ended September 30, 2021 and 2020
Revenues
We have not recognized any revenues since inception.
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