QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
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(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) |
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
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The |
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☒ |
Accelerated Filer |
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Non-Accelerated Filer |
☐ |
Smaller Reporting Company |
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Emerging Growth Company |
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• |
our ability to raise substantial additional capital to fund our planned operations in the near term; |
• |
estimates regarding our expenses, use of cash, timing of future cash needs and anticipated capital requirements; |
• |
the development of our product candidates, including statements regarding the initiation, timing, progress and results of our preclinical clinical studies, clinical trials and research and development programs; |
• |
our ability to advance our product candidates through various stages of development, especially through pivotal safety and efficacy trials; |
• |
the risk that final trial data may not support interim analysis of the viability of our product candidates; |
• |
our expectation regarding the safety and efficacy of our product candidates; |
• |
the timing, scope or likelihood of regulatory filings and approvals from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies for our product candidates and for which indications; |
• |
our ability to license additional intellectual property relating to our product candidates from third parties and to comply with our existing license agreements; |
• |
our ability to enter into partnerships or strategic collaboration agreements, our ability to achieve the results contemplated and the potential benefits to be derived from relationships with collaborators; |
• |
our ability to maintain and establish collaborations and licenses; |
• |
developments and projections relating to competition from other pharmaceutical and biotechnology companies or our industry; |
• |
our estimates regarding the potential market opportunity for our product candidates; |
• |
the anticipated rate and degree of commercial scope and potential, as well as market acceptance of our product candidates for any indication, if approved; |
• |
the anticipated amount, timing and accounting of contract liability (formerly deferred revenue), milestones and other payments under licensing, collaboration or acquisition agreements, research and development costs and other expenses; |
• |
our intellectual property position, including the strength and enforceability of our intellectual property rights; |
• |
our ability to attract and retain qualified employees and key personnel; |
• |
the impact of government laws and regulations in the United States and foreign countries; |
• |
our expectations regarding the impact of the ongoing coronavirus disease 2019, or COVID-19, pandemic, including the expected duration of disruption and immediate and long-term impact and effect on our business and operations; |
• |
the diversion of healthcare resources away from the conduct of clinical trials as a result of the ongoing COVID-19 pandemic, including the diversion of hospitals serving as our clinical trial sites and hospital staff supporting the conduct of our clinical trials; |
• |
the interruption of key clinical trial activities, such as clinical trial site monitoring, due to limitations on travel, quarantines or social distancing protocols imposed or recommended by federal or state governments, employers and others in connection with the ongoing COVID-19 pandemic; and |
• |
other risks and uncertainties, including those listed under Part I, Item 1A, “Risk Factors”. |
Page |
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Part I - Financial Information |
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Item 1. |
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4 |
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5 |
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6 |
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7 |
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8 |
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Item 2. |
24 |
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Item 3. |
33 |
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Item 4. |
33 |
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Part II - Other Information |
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Item 1. |
35 |
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Item 1A. |
35 |
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Item 2. |
73 |
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Item 3. |
73 |
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Item 4. |
73 |
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Item 5. |
73 |
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Item 6. |
74 |
March 31, 2020 |
December 31, 2019 |
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ASSETS |
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Current assets: |
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Cash and cash equivalents |
$ | $ | ||||||
Receivables |
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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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Right of use asset |
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Deposits |
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Other non-current assets |
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Total assets |
$ | $ | ||||||
LIABILITIES AND STOCKHOLDERS’ EQUITY |
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Current liabilities: |
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Accounts payable |
$ | $ | ||||||
Accrued expenses |
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Lease liability - current portion |
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Total current liabilities |
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Lease liability - noncurrent portion |
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Total liabilities |
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Commitments and contingencies (Note 6 ) |
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Stockholders’ equity: |
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Common stock, $ |
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Additional paid-in capital |
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Accumulated deficit |
( |
) | ( |
) | ||||
Total stockholders’ equity |
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Total liabilities and stockholders’ equity |
$ | $ | ||||||
For the Three Months Ended March 31, |
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2020 |
2019 |
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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 |
( |
) | ( |
) | ||||
Other income, net |
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Net loss |
$ | ( |
) | $ | ( |
) | ||
Basic and diluted net loss per share |
$ | ( |
) | $ | ( |
) | ||
Weighted average common shares outstanding used to compute basic and diluted net loss per share |
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For the Three Months Ended March 31, 2019 |
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Common Stock |
Additional Paid In Capital |
Accumulated Deficit |
Total Stockholders’ Equity |
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Shares |
Amount |
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Balance at December 31, 2018 |
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$ | |
$ | |
$ | ( |
) | $ | |
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Stock-based compensation |
— |
— |
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— |
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Issuance of restricted common stock |
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— |
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Cancelled restricted common stock |
( |
) | — |
— |
— |
— |
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Repurchase of restricted common stock |
( |
) | — |
( |
) | — |
( |
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Net loss |
— |
( |
) | ( |
) | |||||||||||||||
Balance at March 31, 2019 |
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$ | |
$ | |
$ | ( |
) | $ | |
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For the Three Months Ended March 31, 2020 |
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Common Stock |
Additional Paid In Capital Common Stock |
Accumulated Deficit |
Total Stockholders’ Equity |
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Shares |
Amount |
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Balance at December 31, 2019 |
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$ | |
$ | |
$ | ( |
) | $ | |
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Stock-based compensation |
— |
— |
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— |
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Exercise of employee stock options |
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— |
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— |
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Issuance of restricted common stock |
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( |
) | — |
— |
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Issuance of common stock in connection with a public offering, net of commissions and expenses of $ |
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— |
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Issuance of common stock in connection with an at the market offering, net of commissions and expenses of $ |
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— |
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Net loss |
— |
— |
— |
( |
) | ( |
) | |||||||||||||
Balance at March 31, 2020 |
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$ | |
$ | |
$ | ( |
) | $ | |
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For the Three Months Ended March 31, |
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2020 |
2019 |
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Cash flows from operating activities: |
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Net loss |
$ | ( |
) | $ | ( |
) | ||
Adjustments to reconcile net loss to net cash used in operating activities: |
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Depreciation |
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Stock-based compensation |
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Bonus paid in common stock, net |
— |
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Change in operating assets and liabilities |
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(Increase) decrease in: |
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Receivables |
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Prepaid expenses and other current assets |
( |
) | ||||||
Right of use asset |
( |
) | ||||||
Deposits |
— |
( |
) | |||||
Other noncurrent assets |
( |
) | ||||||
Increase (decrease) in: |
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Accounts payable |
( |
) | ||||||
Accrued expenses |
( |
) | ||||||
Deferred rent |
— |
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Lease liabilities |
( |
) | ||||||
Net cash used in operating activities |
( |
) | ( |
) | ||||
Cash flows from investing activities: |
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Purchases of property and equipment |
( |
) | ( |
) | ||||
Net cash used in investing activities |
( |
) | ( |
) | ||||
Cash flows from financing activities: |
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Proceeds from exercise of stock options |
— |
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Issuance of common stock in connection with a public offering, net |
— |
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Issuance of common stock in connection with at the market offerings, net |
— |
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Net cash provided by financing activities |
— |
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Net increase (decrease) in cash and cash equivalents, and restricted cash |
( |
) | ||||||
Cash and cash equivalents, and restricted cash, beginning of period |
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Cash and cash equivalents, and restricted cash, end of period |
$ | $ | ||||||
Supplementary disclosure of cash flow information: |
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Compensation paid in restricted stock, gross |
$ | — |
$ | |||||
Property and equipment included in accrued expenses |
$ | $ | — |
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• |
Clinical trial expenses; |
• |
Collaboration agreements; |
• |
Fair value measurements of stock-based compensation; and |
• |
Income taxes |
• | Level 1 - Quoted prices in active markets for identical assets or liabilities. |
• | Level 2 - Inputs other than Level 1 that are observable, either directly or indirectly, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. |
• | Level 3 - Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. |
($ in thousands) |
Fair Value Measurements at Reporting Date Using |
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Description |
Balance as of March 31, 2020 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) |
Significant Other Observable Inputs (Level 2) |
Significant Unobservable Inputs (Level 3) |
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Assets: |
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Cash equivalents |
$ | $ | $ | — |
$ | — |
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($ in thousands) |
Fair Value Measurements at Reporting Date Using |
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Description |
Balance as of December 31, 2019 |
Quoted Prices in Active Markets for Identical Assets/Liabilities (Level 1) |
Significant Other Observable Inputs (Level 2) |
Significant Unobservable Inputs (Level 3) |
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Assets: |
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Cash equivalents |
$ | $ | $ | — |
$ | — |
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March 31, |
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2020 |
2019 |
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Stock options |
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Inducement stock options |
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Unvested restricted stock |
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Warrants |
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Three Months Ended March 31, |
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(in thousands) |
2020 |
2019 |
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Operating lease cost |
$ | $ | ||||||
Total lease cost |
$ | $ | ||||||
Weighted-average remaining lease term (years) |
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Weighted-average discount rate |
% | % |
Maturity of Lease Liabilities |
Operating Leases |
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2020 (excluding the three months ended March 31, 2020) |
$ |
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2021 |
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2022 |
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2023 |
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2024 |
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Thereafter |
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Total lease payments |
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Less: Imputed Interest and Adjustments |
( |
) | ||
Present value of lease payments |
$ | |||
For the three months ended March 31, |
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(in thousands) |
2020 |
2019 |
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Research and development |
$ | |
$ | |
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General and administrative |
|
|
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Stock-based compensation expense |
$ | |
$ | |
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For the three months ended March 31 , |
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2020 |
2019 |
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Risk-free interest rate |
- |
- |
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Expected life in years |
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Expected volatility |
- |
- |
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Expected dividend yield |
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(in thousands, except share and per share data) |
Number of Shares |
Weighted- Average Exercise Price |
Weighted- Average Contractual Term (Years) |
Aggregate Intrinsic Value |
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Outstanding, December 31, 2019 |
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$ | |
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Granted |
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Exercised |
( |
) | |
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Cancelled |
( |
) | |
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Outstanding, March 31, 2020 |
|
$ | |
|
$ | |
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Options exercisable, March 31, 2020 |
|
$ | |
|
$ | |
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Options exercisable, December 31, 2019 |
|
$ | |
|
$ | |
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Options available for future grant |
|
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Number of Shares |
Weighted-Average Grant Date Fair Value |
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Non-vested, December 31, 2019 |
|
$ | |
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Granted |
|
|
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Vested |
|
|
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Cancelled |
|
|
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Non-vested, March 31, 2020 |
|
$ | |
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• | continue to undertake clinical trials for product candidates; |
• | seek regulatory approvals for product candidates; |
• | work with regulatory authorities to identify and address program-related inquiries; |
• | implement additional internal systems and infrastructure; |
• | hire additional clinical, scientific and management personnel; and |
• | scale-up the formulation and manufacturing of our product candidates. |
Three months ended March 31, |
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($ in thousands) |
2020 |
2019 |
Change |
|||||||||||||
Research and development |
$ | 12,706 |
$ | 9,476 |
$ | 3,230 |
34 |
% |
Clinical Phase |
Estimated Completion Period |
|||
Phase 1 |
1 - 2 years |
|||
Phase 2 |
2 - 3 years |
|||
Phase 3 |
2 - 4 years |
• | The number of clinical sites included in the trials; |
• | The length of time required to enroll suitable patents; |
• | The number of patients that ultimately participate in the trials; |
• | The duration of patient follow-up to ensure the absence of long-term product-related adverse events; and |
• | The efficacy and safety profile of the product. |
Three months ended March 31, |
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($ in thousands) |
2020 |
2019 |
Change |
|||||||||||||
General and administrative |
$ | 5,954 |
$ | 4,145 |
$ | 1,809 |
44 |
% |
Three months ended March 31, |
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($ in thousands) |
2020 |
2019 |
Change |
|||||||||||||
Other income, net |
$ | 367 |
$ | 187 |
$ | 180 |
96 |
% |
Three months ended March 31, |
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($ in thousands) |
2020 |
2019 |
||||||
Net cash provided by (used in): |
||||||||
Operating activities |
$ | (9,906 |
) | $ | (10,232 |
) | ||
Investing activities |
(513 |
) | (10 |
) | ||||
Financing activities |
101,680 |
— |
||||||
Net increase (decrease) in cash, cash equivalents, and restricted cash |
$ | 91,261 |
$ | (10,242 |
) | |||
• | Non-cash operating items such as depreciation and stock-based compensation; and |
• | Changes in operating assets and liabilities, which reflect timing differences between the receipt and payment of cash associated with transactions and when they are recognized in results of operations. |
• | the scope, number, initiation, progress, timing, costs, design, duration, any potential delays, and results of clinical trials and nonclinical studies for our current or future product candidates; |
• | changes in the focus, direction and pace of our development programs; |
• | the effect of competitive and technical advances and market developments; |
• | costs associated with the development of our product candidates; |
• | our ability to establish and maintain partnering, collaborations or similar arrangements on favorable terms and whether and to what extent we retain development or commercialization responsibilities under any new licensing, collaboration or similar arrangement; |
• | diversion of healthcare resources away from the conduct of clinical trials as a result of the ongoing COVID-19 pandemic, including the diversion of hospitals serving as our clinical trial sites and hospital staff supporting the conduct of our clinical trials; |
• | the interruption of key clinical trial activities, such as clinical trial site monitoring, due to limitations on travel, quarantines or social distancing protocols imposed or recommended by federal or state governments, employers and others in connection with the ongoing COVID-19 pandemic; |
• | our need and ability to hire additional management and scientific and medical personnel; |
• | the costs of acquiring, licensing or investing in businesses, product candidates and technologies; |
• | costs of filing, prosecuting, defending and enforcing any patent claims and any other intellectual property rights, or other developments; and |
• | other matters identified under Part II, Item 1A. “Risk Factors.” |
($ in thousands) |
Total |
Less than 1 year |
2 - 3 years |
4 - 5 years |
More than 5 years |
|||||||||||||||
Operating leases |
$ | 2,594 |
$ | 962 |
$ | 727 |
$ | 450 |
$ | 455 |
||||||||||
CRADA |
5,000 |
3,125 |
1,875 |
— |
— |
|||||||||||||||
Royalty and license fees |
3,900 |
850 |
700 |
700 |
1,650 |
|||||||||||||||
Total |
$ | 11,494 |
$ | 4,937 |
$ | 3,302 |
$ | 1,150 |
$ | 2,105 |
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• | continue to undertake clinical trials for product candidates; |
• | scale-up the formulation and manufacturing of our product candidates; |
• | seek regulatory approvals for product candidates; |
• | work with regulatory authorities to identify and address program-related inquiries; |
• | implement additional internal systems and infrastructure; and |
• | hire additional personnel. |
• | obtaining regulatory approval from the FDA and other regulatory authorities that have very limited experience with the commercial development of genetically modified and/or unmodified T-cell therapies for cancer; |
• | identifying and manufacturing appropriate TCRs from patient and from third parties that can be administered to a patient; |
• | developing and deploying consistent and reliable processes for engineering a patient’s and/or donor’s T-cells ex vivo T-cells back into the patient; |
• | possibly conditioning patients with chemotherapy in conjunction with delivering each of the potential products, which may increase the risk of adverse side effects of the potential products; |
• | educating medical personnel regarding the potential side effect profile of each of the potential products, such as the potential adverse side effects related to cytokine release; |
• | addressing any competing technological and market developments; |
• | developing processes for the safe administration of these potential products, including long-term follow-up for all patients who receive the potential products; |
• | sourcing additional clinical and, if approved, commercial supplies for the materials used to manufacture and process the potential products; |
• | developing a manufacturing process and distribution network with a cost of goods that allows for an attractive return on investment; |
• | establishing sales and marketing capabilities after obtaining any regulatory approval to gain market acceptance; |
• | developing therapies for types of cancers beyond those addressed by the current potential products; |
• | maintaining and defending the intellectual property rights relating to any products we develop; |
• | and not infringing the intellectual property rights, in particular, the patent rights, of third parties, including competitors, such as those developing T-cell therapies. |
• | developing drugs and biopharmaceuticals; |
• | undertaking preclinical testing and human clinical trials; |
• | obtaining FDA and other regulatory approvals of drugs and biopharmaceuticals; |
• | formulating and manufacturing drugs and biopharmaceuticals; and |
• | launching, marketing, and selling drugs and biopharmaceuticals. |
• | the scope of rights granted under the applicable license agreement and other interpretation-related issues; |
• | whether and the extent to which our technology and processes, and the technology and processes of PGEN, MD Anderson, the NCI and our other licensors, infringe intellectual property of the licensor that is not subject to the applicable license agreement; |
• | our right to sublicense patent and other rights to third parties pursuant to our relationships with our licensors and partners; |
• | whether we are complying with our diligence obligations with respect to the use of the licensed technology in relation to our development and commercialization of our potential products under the MD Anderson License, the License Agreement with PGEN and our patent license agreement with the NCI; and |
• | whether or not our partners are complying with all of their obligations to support our programs under licenses and research and development agreements; and |
• | the allocation of ownership of inventions and know-how resulting from the joint creation or use of intellectual property by our licensors and by us. |
• | Additional nonclinical data requests by regulatory agencies; |
• | Unforeseen safety issues; |
• | Determination of dosing issues; |
• | Lack of effectiveness during clinical trials; |
• | Slower than expected rates of patient recruitment and enrollment; |
• | Inability to monitor patients adequately during or after treatment; |
• | Inability or unwillingness of medical investigators or patients to follow our clinical protocols or attend follow-up visits, including as a result of the recent COVID-19 pandemic; and |
• | Regulatory determinations to temporarily or permanently cease enrollment for other reasons not related to patient safety. |
• | Continuing to undertake preclinical development and clinical trials; |
• | Participating in regulatory approval processes; |
• | Formulating and manufacturing products; and |
• | Conducting sales and marketing activities. |
• | Decreased demand for our product candidates; |
• | Injury to our reputation; |
• | Withdrawal of clinical trial participants; |
• | Withdrawal of prior governmental approvals; |
• | Costs of related litigation; |
• | Substantial monetary awards to patients; |
• | Product recalls; |
• | Loss of revenue; and |
• | The inability to commercialize our product candidates. |
• | Delay commercialization of, and our ability to derive product revenues from, our product candidates; |
• | Impose costly procedures on us; and |
• | Diminish any competitive advantages that we may otherwise enjoy. |
• | regulatory authorities may withdraw approvals of such product; |
• | regulatory authorities may require additional warnings on the label; |
• | we may be required to create a risk evaluation and mitigation strategy plan, which could include a medication guide outlining the risks of such side effects for distribution to patients, a communication plan for healthcare providers, and/or other elements to assure safe use; |
• | we could be sued and held liable for harm caused to patients; and |
• | our reputation may suffer. |
• | We may be unable to identify manufacturers on acceptable terms or at all because the number of potential manufacturers is limited and the FDA must approve any replacement contractor. This approval would require new testing and compliance inspections. In addition, a new manufacturer would have to be educated in, or develop substantially equivalent processes for, production of our products after receipt of FDA approval, if any. |
• | Our third-party manufacturers might be unable to formulate and manufacture our products in the volume and of the quality required to meet our clinical needs and commercial needs, if any. |
• | Our future contract manufacturers may not perform as agreed or may not remain in the contract manufacturing business for the time required to supply our clinical trials or to successfully produce, store, and distribute our products. |
• | Biopharmaceutical manufacturers are subject to ongoing periodic unannounced inspection by the FDA, the Drug Enforcement Administration and corresponding state and foreign agencies to ensure strict compliance with current good manufacturing practices, or cGMP, and other government regulations and corresponding foreign standards. We do not have control over third-party manufacturers’ compliance with these regulations and standards. |
• | If any third-party manufacturer makes improvements in the manufacturing process for our products, we may not own, or may have to share, the intellectual property rights to the innovation. |
• | Third party manufacturers may also encounter difficulties in achieving volume production, quality control, and quality assurance and also may experience shortages in qualified personnel and obtaining materials for our product candidates, including delays or shortages due to limited supply or capacity of production facilities as a result of the recent COVID-19 pandemic. |
• | Our third-party manufacturers may not be able to comply with cGMP regulations or similar regulatory requirements outside the United States. Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in sanctions being imposed on us, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of products, operating restrictions and criminal prosecutions, any of which could significantly and adversely affect supplies of our products. |
• | Litigation involving patients taking our product; |
• | Restrictions on such products, manufacturers or manufacturing processes; |
• | Restrictions on the labeling or marketing of a product; |
• | Restrictions on product distribution or use; |
• | Requirements to conduct post-marketing studies or clinical trials; |
• | Warning letters; |
• | Withdrawal of the products from the market; |
• | Refusal to approve pending applications or supplements to approved applications that we submit; |
• | Recall of products; |
• | Fines, restitution or disgorgement of profits or revenues; |
• | Suspension or withdrawal of marketing approvals; |
• | Damage to relationships with existing and potential collaborators; |
• | Unfavorable press coverage and damage to our reputation; |
• | Refusal to permit the import or export of our products; |
• | Product seizure; or |
• | Injunctions or the imposition of civil or criminal penalties. |
• | Developing drugs and biopharmaceuticals; |
• | Undertaking preclinical testing and human clinical trials; |
• | Obtaining FDA and other regulatory approvals of drugs and biopharmaceuticals; |
• | Formulating and manufacturing drugs and biopharmaceuticals; and |
• | Launching, marketing, and selling drugs and biopharmaceuticals. |
• | Perceptions by members of the healthcare community, including physicians, about the safety and effectiveness of our products; |
• | Pharmacological benefit and cost-effectiveness of our products relative to competing products; |
• | Availability of coverage and adequate reimbursement for our products from government or other third-party payors; |
• | Effectiveness of marketing and distribution efforts by us and our licensees and distributors, if any; and |
• | The price at which we sell our products. |
• | Created an annual, nondeductible fee on any entity that manufactures or imports certain branded prescription drugs and biologic agents, apportioned among these entities according to their market share in certain government healthcare programs; |
• | Increased the statutory minimum rebates a manufacturer must pay under the Medicaid Drug Rebate Program to 23.1% and 13% of the average manufacturer price for most branded and generic drugs, respectively; |
• | Created a new Medicare Part D coverage gap discount program, in which manufacturers must now agree to offer 70% point-of-sale discounts off negotiated prices of applicable brand drugs to eligible beneficiaries during their coverage gap period, as a condition for the manufacturer’s outpatient drugs to be covered under Medicare Part D; |
• | Extended manufacturers’ Medicaid rebate liability to covered drugs dispensed to individuals who are enrolled in Medicaid managed care organizations; |
• | Created new methodologies by which rebates owed by manufacturers under the Medicaid Drug Rebate Program are calculated for drugs that are inhaled, infused, instilled, implanted or injected, and for drugs that are line extensions; |
• | Expanded eligibility criteria for Medicaid programs by, among other things, allowing states to offer Medicaid coverage to additional individuals with income at or below 133% of the Federal Poverty Level, thereby potentially increasing both the volume of sales and manufacturers’ Medicaid rebate liability; |
• | Expanded the entities eligible for discounts under the Public Health Service pharmaceutical pricing program; |
• | Created a new requirement to annually report drug samples that certain manufacturers and authorized distributors provide to physicians; |
• | Expanded healthcare fraud and abuse laws, including the False Claims Act and the federal Anti-Kickback Statute, new government investigative powers, and enhanced penalties for noncompliance; |
• | Created a licensure framework for follow-on biologic products; |
• | Created new requirements under the federal Physician Payments Sunshine Act for certain drug manufacturers to annually report information related to payments and other transfers of value made to physicians, as defined by such law, and teaching hospitals as well as ownership or investment interests held by physicians and their immediate family members; |
• | Created a Patient-Centered Outcomes Research Institute to oversee, identify priorities in, and conduct comparative clinical effectiveness research, along with funding for such research; and |
• | Established a Center for Medicare & Medicaid Innovation at the Centers for Medicare & Medicaid Services, or CMS, to test innovative payment and service delivery models to lower Medicare and Medicaid spending, potentially including prescription drug spending. |
• | The federal Anti-Kickback Statute, which regulates our business activities, including our marketing practices, educational programs, pricing policies, and relationships with healthcare providers or other entities, by prohibiting, among other things, soliciting, receiving, offering or paying remuneration, directly or indirectly, to induce, or in return for, either the referral of an individual or the purchase or recommendation of an item or service reimbursable under a federal healthcare program, such as the Medicare and Medicaid programs; |
• | Federal civil and criminal false claims laws, including the False Claims Act which permits a private individual acting as a “whistleblower” to bring actions on behalf of the federal government alleging violations of the False Claims Act, and civil monetary penalty laws, which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment from Medicare, Medicaid, or other third-party payors that are false or fraudulent; |
• | The federal Health Insurance Portability and Accountability Act of 1996, or HIPAA, which created new federal civil and criminal statutes that prohibit, among other things, executing a scheme to defraud any healthcare benefit program or making false statements relating to healthcare matters; |
• | HIPAA, as amended by the Health Information Technology for Economic and Clinical Health Act of 2009, or HITECH, and their implementing regulations, which imposes certain requirements relating to the privacy, security and transmission of individually identifiable health information on entities and individuals subject to the law including certain healthcare providers, health plans, and healthcare clearinghouses, known as covered entities, as well as individuals and entities that perform services for them which involve the use, or disclosure of, individually identifiable health information, known as business associates; |
• | Requirements under the Physician Payments Sunshine Act to report annually to CMS certain financial arrangements with physicians, as defined by such law, and teaching hospitals, as defined in the ACA and its implementing regulations, including reporting any “transfer of value” made or distributed to teaching hospitals, prescribers, and other healthcare providers and reporting any ownership and investment interests held by physicians and their immediate family members and applicable group purchasing organizations during the preceding calendar year; and |
• | State and foreign law equivalents of each of the above federal laws, such as anti-kickback and false claims laws which may apply to items or services reimbursed by any third-party payor, including commercial insurers; state laws that require pharmaceutical companies to comply with the industry’s voluntary compliance guidelines and the applicable compliance guidance promulgated by the federal government that otherwise restricts certain payments that may be made to healthcare providers and entities; state laws that require drug manufacturers to report information related to payments and other transfer of value to physicians and other healthcare providers and entities; state laws that require the reporting of information related to drug pricing; state and local laws that require the registration of pharmaceutical sales representatives; and state and foreign laws governing the privacy and security of health information in certain circumstances, many of which differ from each other in significant ways and often are not preempted by HIPAA, thus complicating compliance efforts. |
• | The degree and range of protection any patents will afford us against competitors, including whether third parties will find ways to invalidate or otherwise circumvent our patents; |
• | If and when patents will be issued; |
• | Whether or not others will obtain patents claiming subject matter related to or relevant to our product candidates; or |
• | Whether we will need to initiate litigation or administrative proceedings that may be costly whether we win or lose. |
• | Price and volume fluctuations in the overall stock market; |
• | Changes in operating results and performance and stock market valuations of other biopharmaceutical companies generally, or those that develop and commercialize cancer drugs in particular; |
• | Market conditions or trends in our industry or the economy as a whole; |
• | Preclinical or clinical trial results; |
• | Public concern as to the safety of drugs developed by us or others; |
• | The financial or operational projections we may provide to the public, any changes in these projections or our failure to meet these projections; |
• | Comments by securities analysts or changes in financial estimates or ratings by any securities analysts who follow our common stock, our failure to meet these estimates or failure of those analysts to initiate or maintain coverage of our common stock; |
• | The public’s response to press releases or other public announcements by us or third parties, including our filings with the SEC, as well as announcements of the status of development of our products, announcements of technological innovations or new therapeutic products by us or our competitors, announcements regarding collaborative agreements and other announcements relating to product development, litigation and intellectual property impacting us or our business; |
• | Government regulation; |
• | FDA determinations on the approval of a product candidate BLA submission; |
• | The sustainability of an active trading market for our common stock; |
• | Future sales of our common stock by us, our executive officers, directors and significant stockholders; |
• | Announcements of mergers or acquisition transactions; |
• | Our inclusion or deletion from certain stock indices; |
• | Developments in patent or other proprietary rights; |
• | Changes in reimbursement policies; |
• | Announcements of medical innovations or new products by our competitors; |
• | Announcements of changes in our senior management; |
• | General economic, industry, political and market conditions, including those resulting from pandemic, epidemic or disease outbreaks, such as the ongoing COVID-19 pandemic, or geo-political actions, including war and incidents of terrorism, natural disasters or responses to these events; |
• | Changes in accounting principles; and |
• | Other risks detailed in our SEC filings. |
• | Delaying, deferring or preventing a change in control; |
• | Impeding a merger, consolidation, takeover or other business combination involving us; or |
• | Discouraging a potential acquirer from making a tender offer or otherwise attempting to obtain control of us. |
Exhibit Number |
Description | |||
3.1 |
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3.2 |
||||
3.3 |
||||
10.1 |
||||
31.1+ |
||||
31.2+ |
||||
32.1++ |
||||
101.INS+ |
Inline XBRL Instance Document (the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document). | |||
101.SCH+ |
Inline XBRL Taxonomy Extension Schema Document | |||
101.CAL+ |
Inline XBRL Taxonomy Extension Calculation Linkbase Document | |||
101.DEF+ |
Inline XBRL Taxonomy Definition Linkbase Document | |||
101.LAB+ |
Inline XBRL Taxonomy Extension Label Linkbase Document | |||
101.PRE+ |
Inline XBRL Taxonomy Extension Presentation Linkbase Document | |||
104+ |
Cover Page Interactive Data File—the cover page interactive data is embedded within the Inline XBRL document or included within the Exhibit 101 attachments |
+ |
Filed herewith. | |||
++ |
This certification is deemed not filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, or otherwise subject to the liability of that section, nor shall it be deemed incorporated by reference into any filing under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended whether made before or after the date hereof, regardless of any general incorporation language in such filing. |
ZIOPHARM ONCOLOGY, INC. | ||
By: | ||
/s/ Laurence J.N. Cooper | ||
Laurence J.N. Cooper, M.D., Ph.D. | ||
Chief Executive Officer | ||
(Principal Executive Officer) | ||
Dated: May 7, 2020 | ||
By: | ||
/s/ Satyavrat Shukla | ||
Satyavrat Shukla | ||
Executive Vice President and Chief Financial Officer | ||
(Principal Financial Officer) | ||
Dated: May 7, 2020 |
EXHIBIT 31.1
CERTIFICATION
I, Laurence J.N. Cooper, certify that:
1. I have reviewed this quarterly report on Form 10-Q of ZIOPHARM Oncology, Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrants other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrants disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
(d) Disclosed in this report any change in the registrants internal control over financial reporting that occurred during the registrants most recent fiscal quarter (the registrants fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrants internal control over financial reporting; and
5. The registrants other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrants auditors and the audit committee of the registrants board of directors (or persons performing the equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrants ability to record, process, summarize and report financial information; and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrants internal control over financial reporting.
Date: May 7, 2020
/s/ Laurence J.N. Cooper |
Laurence J.N. Cooper, M.D., Ph.D. Chief Executive Officer |
(Principal Executive Officer) |
EXHIBIT 31.2
CERTIFICATION
I, Satyavrat Shukla, certify that:
1. I have reviewed this quarterly report on Form 10-Q of ZIOPHARM Oncology, Inc.;
2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to make the statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by this report;
3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respects the financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report;
4. The registrants other certifying officer(s) and I are responsible for establishing and maintaining disclosure controls and procedures (as defined in Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have:
(a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under our supervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by others within those entities, particularly during the period in which this report is being prepared;
(b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed under our supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements for external purposes in accordance with generally accepted accounting principles;
(c) Evaluated the effectiveness of the registrants disclosure controls and procedures and presented in this report our conclusions about the effectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and
(d) Disclosed in this report any change in the registrants internal control over financial reporting that occurred during the registrants most recent fiscal quarter (the registrants fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely to materially affect, the registrants internal control over financial reporting; and
5. The registrants other certifying officer(s) and I have disclosed, based on our most recent evaluation of internal control over financial reporting, to the registrants auditors and the audit committee of the registrants board of directors (or persons performing the equivalent functions):
(a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which are reasonably likely to adversely affect the registrants ability to record, process, summarize and report financial information; and
(b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrants internal control over financial reporting.
Date: May 7, 2020
/s/ Satyavrat Shukla |
Satyavrat Shukla Executive Vice President and Chief Financial Officer |
(Principal Financial Officer) |
EXHIBIT 32.1
CERTIFICATION
In connection with the Quarterly Report on Form 10-Q of ZIOPHARM Oncology, Inc. (the Company) for the quarter ended March 31, 2020, as filed with the Securities and Exchange Commission on the date hereof (the Report), we Laurence J.N. Cooper, the Principal Executive Officer of the Company and Satyavrat Shukla, the Principal Financial Officer of the Company, hereby certify, pursuant to 18 U.S.C. 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, that, to our knowledge:
(1) | the Report fully complies with the requirements of Section 13(a) or 15(d), as applicable, of the Securities Exchange Act of 1934, as amended, and |
(2) | the information in the Report fairly presents, in all material respects, the financial condition and results of operations of the Company. |
Dated: May 7, 2020
/s/ Laurence J.N. Cooper |
Laurence J.N. Cooper, M.D., Ph.D. Chief Executive Officer |
(Principal Executive Officer) |
/s/ Satyavrat Shukla |
Satyavrat Shukla Executive Vice President and Chief Financial Officer |
(Principal Financial Officer) |