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 |
(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) | |
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(Address of principal executive offices) |
(Zip Code) |
Title of each class |
Trading symbol(s) |
Name of each exchange on which registered | ||
Large accelerated filer | ☐ | ☒ | ||||
Non-accelerated filer |
☐ | Smaller reporting company | ||||
Emerging growth company |
Page |
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PART I |
3 |
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Item 1. |
3 |
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3 |
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4 |
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5 |
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6 |
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7 |
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Item 2. |
19 |
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Item 3. |
27 |
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Item 4. |
27 |
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PART II |
28 |
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Item 1A. |
28 |
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Item 6. |
50 |
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51 |
Item 1. |
Financial Statements |
June 30, |
December 31, |
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2020 |
2019 |
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Assets |
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Current assets: |
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Cash and cash equivalents |
$ | $ | ||||||
Marketable securities |
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Accrued interest receivable |
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Prepaid research and development expenses |
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Other prepaid expenses and 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 |
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Other assets |
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Total assets |
$ | $ | ||||||
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Liabilities and stockholders’ equity |
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Current liabilities: |
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Accounts payable |
$ | $ | ||||||
Accrued research and development expenses |
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Accrued restructuring |
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Other accrued liabilities |
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Total current liabilities |
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Long-term portion of operating lease liability |
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Total liabilities |
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Commitments and contingencies |
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Stockholders’ equity: |
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Preferred stock, $ |
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Common stock, $ |
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Additional paid-in capital |
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Accumulated other comprehensive income |
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Accumulated deficit |
( |
) | ( |
) | ||||
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Total stockholders’ equity |
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Total liabilities and stockholders’ equity |
$ | $ | ||||||
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Three Months Ended |
Six Months Ended |
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June 30, |
June 30, |
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2020 |
2019 |
2020 |
2019 |
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Operating expenses: |
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Research and development |
$ |
$ | $ | $ | ||||||||||||
General and administrative |
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Restructuring charges |
( |
) | — | ( |
) | — | ||||||||||
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Total operating expenses |
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Loss from operations |
( |
) | ( |
) | ( |
) | ( |
) | ||||||||
Other income: |
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Interest income |
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Total other income |
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Net loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
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Other comprehensive income: |
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Unrealized gain on marketable securities |
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Total other comprehensive income |
$ | |||||||||||||||
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Comprehensive loss |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
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Basic net loss per common share |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Diluted net loss per common share |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Weighted average common shares outstanding used to calculate basic net loss per common share |
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Weighted average common shares outstanding used to calculate diluted net loss per common share |
Six Months Ended |
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June 30, |
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2020 |
2019 |
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Operating activities |
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Net loss |
$ | ( |
) | $ | ( |
) | ||
Adjustments to reconcile net loss to net cash used in operating activities: |
||||||||
Depreciation and amortization |
||||||||
Stock-based compensation expense |
||||||||
Net accretion and amortization of investments in marketable securities |
( |
) | ( |
) | ||||
Changes in assets and liabilities: |
||||||||
Interest receivable and other current assets |
( |
) | ||||||
Prepaid expenses |
( |
) | ||||||
Other assets |
— | |||||||
Accounts payable |
( |
) | ||||||
Accrued restructuring charges |
( |
) | — | |||||
Accrued liabilities |
( |
) | ||||||
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Net cash used in operating activities |
( |
) | ( |
) | ||||
Investing activities |
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Purchases of property and equipment |
— | ( |
) | |||||
Purchases of marketable securities |
( |
) | ( |
) | ||||
Proceeds from maturities of marketable securities |
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Proceeds from sale of marketable securities |
— | |||||||
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Net cash provided by (used in) investing activities |
( |
) | ||||||
Financing activities |
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Proceeds from issuance of common stock, net of issuance costs |
— | |||||||
Proceeds from issuance of common stock pursuant to equity award plans |
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Net cash provided by financing activities |
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Net increase (decrease) in cash and cash equivalents |
( |
) | ||||||
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Cash and cash equivalents at beginning of period |
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Cash and cash equivalents at end of period |
$ | $ | ||||||
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Supplemental disclosure |
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Cash paid for amounts included in the measurement of lease liabilities |
$ | $ | ||||||
Supplemental non-cash investing and financing activities |
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Accrued financing costs |
$ | $ | — |
Three and Six Months Ended June 30, 2020 |
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Accumulated |
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Additional |
Other |
Total |
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Common Stock |
Paid-in |
Comprehensive |
Accumulated |
Stockholders’ |
||||||||||||||||||||
Shares |
Amount |
Capital |
Income (Loss) |
Deficit |
Equity |
|||||||||||||||||||
Balances as of December 31, 2019 |
$ | $ | $ | $ | ( |
) | $ | |
||||||||||||||||
Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
Net loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
Net unrealized loss on marketable securities |
— | — | — | ( |
) | — | ( |
) | ||||||||||||||||
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Balances as of March 31, 2020 |
$ | $ | $ | ( |
) | $ | ( |
) | $ | |||||||||||||||
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Issuance of common stock upon exercise of stock options |
— | — | — | |||||||||||||||||||||
Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
Net loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
Net unrealized gain on marketable securities |
— | — | — | — | ||||||||||||||||||||
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Balances as of June 30, 2020 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
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Three and Six Months Ended June 30, 2019 |
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Accumulated |
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Additional |
Other |
Total |
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Common Stock |
Paid-in |
Comprehensive |
Accumulated |
Stockholders’ |
||||||||||||||||||||
Shares |
Amount |
Capital |
Income (Loss) |
Deficit |
Equity |
|||||||||||||||||||
Balances as of December 31, 2018 |
$ | $ | |
$ | ( |
) | $ | ( |
) | $ | |
|||||||||||||
Issuance of common stock upon exercise of stock options and incentive awards |
— | — | — | |||||||||||||||||||||
Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
Issuance of common stock, net of $ |
— | — | ||||||||||||||||||||||
Net loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
Net unrealized gain on marketable |
— | — | — | — | ||||||||||||||||||||
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Balances as of March 31, 2019 |
$ | $ | $ | $ | ( |
) | $ | |||||||||||||||||
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|
|||||||||||||
Issuance of common stock upon exercise of stock options and incentive awards |
— | — | — | |||||||||||||||||||||
Stock-based compensation expense |
— | — | — | — | ||||||||||||||||||||
Net loss |
— | — | — | — | ( |
) | ( |
) | ||||||||||||||||
Net unrealized gain on marketable |
— | — | — | — | ||||||||||||||||||||
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Balances as of June 30, 2019 |
$ | $ | $ | |
$ | ( |
) | $ | ||||||||||||||||
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As of June 30, 2020 |
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Level 1 |
Level 2 |
Level 3 |
Total |
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Cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | — | $ | — | |||||||||||
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Total cash equivalents |
— | — | ||||||||||||||
Marketable securities: |
||||||||||||||||
U.S. and foreign commercial paper |
— | — | ||||||||||||||
U.S. and foreign corporate debt securities |
— | — | ||||||||||||||
Asset-backed securities |
— | — | ||||||||||||||
U.S. treasury securities |
— | — | ||||||||||||||
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Total marketable securities |
— | — | ||||||||||||||
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Total assets measured at fair value |
$ | $ |
$ | — | ||||||||||||
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As of December 31, 2019 |
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Level 1 |
Level 2 |
Level 3 |
Total |
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Cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | — | $ |
— | |||||||||||
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|
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|
|
Total cash equivalents |
— | — | ||||||||||||||
Marketable securities: |
||||||||||||||||
U.S. and foreign commercial paper |
— | — | ||||||||||||||
U.S. and foreign corporate debt securities |
— | — | ||||||||||||||
Asset-backed securities |
— | — | ||||||||||||||
U.S. treasury securities |
— | — | ||||||||||||||
|
|
|
|
|
|
|
||||||||||
Total marketable securities |
— | — | ||||||||||||||
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|
|
|
|
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|
|
|
|
|
Total assets measured at fair value |
$ | $ | $ |
— | ||||||||||||
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Gross |
Gross |
|||||||||||||||
Amortized |
Unrealized |
Unrealized |
Estimated |
|||||||||||||
Cost |
Gains |
Losses |
Fair Value |
|||||||||||||
As of June 30, 2020: |
||||||||||||||||
Cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | — | $ | — | $ | ||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total cash equivalents |
— | — | ||||||||||||||
Short-term investments: |
||||||||||||||||
U.S. and foreign commercial paper |
— | — | ||||||||||||||
U.S. and foreign corporate debt securities |
— | |||||||||||||||
Asset-backed securities |
— | |||||||||||||||
U.S. treasury securities |
— | |||||||||||||||
|
|
|
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|
|||||||||
Total short-term investments |
— | |||||||||||||||
|
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|
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Total marketable securities |
$ | $ | $ | — | $ | |||||||||||
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|
|
Gross |
Gross |
|||||||||||||||
Amortized |
Unrealized |
Unrealized |
Estimated |
|||||||||||||
Cost |
Gains |
Losses |
Fair |
|||||||||||||
As of December 31, 2019: |
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Cash equivalents: |
||||||||||||||||
Money market funds |
$ | $ | — | $ | — | $ | ||||||||||
|
|
|
|
|
|
|
|
|||||||||
Total cash equivalents |
— | |||||||||||||||
Short-term investments: |
||||||||||||||||
U.S. and foreign commercial paper |
— | — | ||||||||||||||
U.S. and foreign corporate debt securities |
— | |||||||||||||||
Asset-backed securities |
— | |||||||||||||||
U.S. treasury securities |
— | |||||||||||||||
|
|
|
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|
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Total short-term investments |
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|
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Total marketable securities |
$ | $ | $ | — | $ | |||||||||||
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|
|
|
|
|
Three Months Ended June 30, |
Six Months Ended June 30, |
|||||||||||||||
2020 |
2019 |
2020 |
2019 |
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Numerator: |
||||||||||||||||
Net loss allocated to common stock—basic |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Net loss allocated to common stock—diluted |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Denominator: |
||||||||||||||||
Weighted average number of common stock shares outstanding—basic |
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Dilutive securities: |
||||||||||||||||
Weighted average number of common stock shares outstanding—diluted |
||||||||||||||||
Net loss per share—basic: |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) | ||||
Net loss per share—diluted: |
$ | ( |
) | $ | ( |
) | $ | ( |
) | $ | ( |
) |
Three Months Ended June 30, |
Six Months Ended June 30, |
|||||||||||||||
2020 |
2019 |
2020 |
2019 |
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Common stock options |
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Incentive awards |
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Total |
June 30, 2020 |
December 31, 2019 |
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Accrued compensation |
$ |
$ | ||||||
Accrued professional fees and other |
||||||||
Operating lease liability |
||||||||
Total other accrued liabilities |
$ | $ | ||||||
Termination Benefits |
Contract Termination Costs |
Total |
||||||||||
Balances as of December 31, 2019 |
$ | $ | $ | |||||||||
Restructuring charges |
— | |||||||||||
Reductions for cash payments |
( |
) | — | ( |
) | |||||||
Balances as of March 31, 2020 |
||||||||||||
Restructuring charges |
( |
) | — | ( |
) | |||||||
Reductions for cash payments |
( |
) | — | ( |
) | |||||||
Balances as of June 30, 2020 |
$ | $ | $ | |||||||||
Lease Payments |
||||
Year ending December 31, |
||||
2020 (July through December) |
$ | |||
2021 |
||||
2022 |
||||
2023 |
||||
2024 |
||||
|
|
|||
Total undiscounted future minimum lease payments |
$ | |||
Less: imputed interest |
||||
|
|
|||
Total operating lease liability |
$ | |||
Less: current portion of operating lease liability (included in other accrued liabilities) |
||||
|
|
|||
Long-term portion of lease liability |
$ | |||
|
|
Three Months Ended June 30, |
Six Months June 30, |
|||||||||||||||
2020 |
2019 |
2020 |
2019 |
|||||||||||||
Research and development |
$ | $ | $ | $ | ||||||||||||
General and administrative |
||||||||||||||||
|
|
|
|
|
|
|
|
|||||||||
Restructuring charges |
|
|
|
|
|
|
— |
|
|
|
|
|
|
|
— |
|
Total stock-based compensation expense |
$ | $ | $ | $ | ||||||||||||
|
|
|
|
|
|
|
|
Item 2. |
Management’s Discussion and Analysis of Financial Condition and Results of Operations |
• | primary biliary cholangitis (PBC), a rare, chronic autoimmune disease that causes progressive destruction of the bile ducts in the liver resulting in impaired bile flow (cholestasis) and inflammation. The FDA has granted seladelpar Breakthrough Therapy Designation for the treatment of early stage PBC. |
• | primary sclerosing cholangitis (PSC), a rare, chronic cholestatic liver disease characterized by diffuse inflammation and fibrosis of the intrahepatic and extrahepatic bile ducts. |
• | Nonalcoholic steatohepatitis (NASH), a prevalent and serious chronic liver disease caused by excessive fat accumulation in the liver that results in inflammation and cellular injury that can progress to fibrosis and cirrhosis, and potentially liver failure and death. |
• | Remote workforce operations |
• | Clinical trial and drug manufacturing operations COVID-19 related travel restrictions and reduced access to work facilities through the use of remote working technologies and other measures as they continue to progress toward completion of our existing clinical trials. However, in the future, as we look to restart the clinical development of seladelpar and initiate other programs, our research and development employees and contractors may not be able to sufficiently access their applicable work facilities as a result of continued facility closure orders and the possibility that governmental authorities might further modify such restrictions. Furthermore, patients we expect to enroll in our future clinical trials may also be impacted by any ongoing travel and facility access restrictions. Although we and our contractors continue to plan for and develop pandemic-related risk mitigation strategies, it is uncertain whether these plans will continue to be sufficient to fully offset the potential impact that travel and facility access restrictions (or other currently unanticipated impediments) may have on our ability to execute our research and development activities. |
• | Drug regulator interactions |
• | Financial reporting and compliance |
Three Months Ended June 30, |
Change Q2 2020 vs 2019 |
Six Months Ended June 30, |
Change Q2 YTD 2020 vs 2019 |
|||||||||||||||||||||
2020 |
2019 |
2020 |
2019 |
|||||||||||||||||||||
($ in thousands) |
||||||||||||||||||||||||
Operating expenses: |
||||||||||||||||||||||||
Research and development |
$ | 7,942 | $ | 21,119 | $ | (13,177 | ) | $ | 17,451 | $ | 39,707 | $ | (22,256 | ) | ||||||||||
General and administrative |
3,398 | 4,529 | (1,131 | ) | 7,745 | 10,192 | (2,447 | ) | ||||||||||||||||
Restructuring charges |
(188 | ) | — | (188 | ) | (117 | ) | — | (117 | ) | ||||||||||||||
|
|
|
|
|
|
|
|
|||||||||||||||||
Total operating expenses |
11,152 | 25,648 | (14,496 | ) | 25,079 | 49,899 | (24,820 | ) | ||||||||||||||||
|
|
|
|
|
|
|
|
|||||||||||||||||
Loss from operations |
(11,152 | ) | (25,648 | ) | 14,496 | (25,079 | ) | (49,899 | ) | 24,820 | ||||||||||||||
Other income: |
||||||||||||||||||||||||
Interest income |
426 | 1,610 | (1,184 | ) | 1,265 | 2,786 | (1,521 | ) | ||||||||||||||||
|
|
|
|
|
|
|
|
|||||||||||||||||
Total other income |
426 | 1,610 | (1,184 | ) | 1,265 | 2,786 | (1,521 | ) | ||||||||||||||||
|
|
|
|
|
|
|
|
|||||||||||||||||
Net loss |
$ | (10,726 | ) | $ | (24,038 | ) | $ | 13,312 | $ | (23,814 | ) | $ | (47,113 | ) | $ | 23,299 | ||||||||
|
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|
|
|
Three Months Ended June 30, |
Change Q2 2020 vs 2019 |
Six Months Ended June 30, |
Change Q2 YTD 2020 vs 2019 |
|||||||||||||||||||||
2020 |
2019 |
2020 |
2019 |
|||||||||||||||||||||
Operating expenses: |
||||||||||||||||||||||||
Research and development |
$ | 7,942 | $ | 21,119 | $ | (13,177 | ) | $ | 17,451 | $ | 39,707 | $ | (22,256 | ) | ||||||||||
General and administrative |
3,398 | 4,529 | (1,131 | ) | 7,745 | 10,192 | (2,447 | ) | ||||||||||||||||
Restructuring charges |
(188 | ) | — | (188 | ) | (117 | ) | — | (117 | ) | ||||||||||||||
|
|
|
|
|
|
|
|
|||||||||||||||||
Total operating expenses |
$ | 11,152 | $ | 25,648 | $ | (14,496 | ) | $ | 25,079 | $ | 49,899 | $ | (24,820 | ) | ||||||||||
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
Change Q2 2020 vs 2019 |
Six Months Ended June 30, |
Change Q2 YTD 2020 vs 2019 |
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2020 |
2019 |
2020 |
2019 |
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Project costs: |
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Seladelpar PBC clinical studies |
$ | 3,142 | $ | 9,954 | $ | (6,812 | ) | $ | 8,726 | $ | 18,798 | $ | (10,072 | ) | ||||||||||
Seladelpar NASH clinical studies |
572 | 2,315 | (1,743 | ) | 1,469 | 5,358 | (3,889 | ) | ||||||||||||||||
Seladelpar PSC clinical studies |
2 | 976 | (974 | ) | 254 | 976 | (722 | ) | ||||||||||||||||
Seladelpar drug manufacturing & development |
— | 2,079 | (2,079 | ) | — | 3,407 | (3,407 | ) | ||||||||||||||||
Seladelpar other studies |
225 | 922 | (697 | ) | 350 | 1,542 | (1,192 | ) | ||||||||||||||||
Non-seladelpar studies |
726 | 154 | 572 | 1,193 | 270 | 923 | ||||||||||||||||||
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Total project costs |
4,667 | 16,400 | (11,733 | ) | 11,992 | 30,351 | (18,359 | ) | ||||||||||||||||
Internal research and development costs |
3,275 | 4,719 | (1,444 | ) | 5,459 | 9,356 | (3,897 | ) | ||||||||||||||||
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Total research and development |
$ | 7,942 | $ | 21,119 | $ | (13,177 | ) | $ | 17,451 | $ | 39,707 | $ | (22,256 | ) | ||||||||||
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• | expenses incurred under agreements with contract research organizations, investigative sites and consultants that conduct our clinical trials and a substantial portion of our preclinical activities; |
• | the cost of acquiring and manufacturing clinical trial and other materials; and |
• | other costs associated with development activities, including additional studies. |
Six Months Ended June 30, |
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2020 |
2019 |
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Net cash used in operating activities |
$ | (22,457 | ) | $ | (46,648 | ) | ||
Net cash provided by (used in) investing activities |
93,632 | (82,116 | ) | |||||
Cash provided by financing activities |
7 | 107,850 | ||||||
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Net increase (decrease) in cash and cash equivalents |
$ | 71,182 | $ | (20,914 | ) | |||
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Item 3. |
Quantitative and Qualitative Disclosures About Market Risk |
Item 4. |
Controls and Procedures |
• | We are currently completing the shutdown of several clinical trials in geographies which are affected by the COVID-19 pandemic and we are in the early planning stages of restarting clinical development of seladelpar. While we have not experienced significant impacts to our clinical activities through June 30, 2020, we believe that the COVID-19 pandemic could potentially have an impact on various aspects of our clinical activities in the future. For example, our employees, representatives from our clinical research organization partners, and study investigators may be unable to efficiently collaborate to conduct investigator site activities in-person at the sites (as per standard practice) and may be required to delay, or alter their approach to complete this work due to diversion of resources at clinical sites or continued government-imposed limitations on travel. Furthermore, such restrictions could impact the rate of patient enrollment in any new clinical studies and the ability to efficiently treat such patients at investigator sites. Lastly, our employees and representatives from our contract manufacturing organizations may experience unanticipated challenges producing and distributing sufficient quantities of clinical drug supplies for use in our clinical trials. |
• | We have closed our corporate office and requested that most of our personnel, including all of our administrative employees, work remotely, and restricted on-site staff to only those personnel and contractors who must perform essential activities that must be completed on-site. The COVID-19 pandemic could disrupt our ability to secure supplies for our operations and to provide personal protective equipment for our employees. The safety, health and well-being of our workforce is of primary concern and we may need to enact further precautionary measures to help minimize the risk of our employees being exposed to the novel coronavirus. |
• | Our increased reliance on personnel working from home may negatively impact productivity, or disrupt, delay, or otherwise adversely impact our business. In addition, this could increase our cyber-security risk, create data accessibility concerns, and make us more susceptible to communication disruptions, any of which could adversely impact our business operations, or delay necessary interactions with regulators, contract manufacturers, contract research organizations, clinical trial sites, and other important agencies and contractors. |
• | Our employees and contractors involved in conducting our research and development activities may not be able to access their applicable work facilities for an extended period of time as a result of facility closure orders and the possibility that governmental authorities further modify such access restrictions. |
• | The FDA and comparable foreign regulatory agencies may experience operational interruptions or delays, which could impact timelines for regulatory meetings, submissions, trial initiations, and regulatory approvals. |
• | the rate of progress and cost of our clinical studies; |
• | the need for additional or expanded clinical studies; |
• | the rate of progress and cost of our Chemistry, Manufacturing and Control development, registration, validation and commercial programs; |
• | the timing, economic and other terms of any licensing, collaboration or other similar arrangement into which we may enter; |
• | the costs and timing of seeking and obtaining U.S. Food and Drug Administration (FDA) and other regulatory approvals; |
• | the extent of our other development activities; |
• | the costs of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; and |
• | the effect of competing products and market developments. |
• | successful enrollment and completion of clinical trials; |
• | receipt of marketing approvals from the FDA and regulatory authorities outside the United States for the product candidate; |
• | establishing commercial manufacturing capabilities by making arrangements with third-party manufacturers; |
• | launching commercial sales of the product, whether alone or in collaboration with others; |
• | acceptance of the product by patients, the medical community and third-party payors; |
• | effectively competing with other therapies; |
• | a continued acceptable safety profile of the product following marketing approval; and |
• | obtaining, maintaining, enforcing and defending intellectual property rights and claims. |
• | regulators or institutional review boards may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site; |
• | the clinical study protocol may require one or more amendments delaying study completion; |
• | clinical trials of our product candidates may produce negative or inconclusive results, and we may decide, or regulators may require us, to conduct additional clinical trials or abandon product development programs; |
• | the number of subjects required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinical trials may be insufficient or slower than we anticipate, we may have to compete with other clinical trials to enroll eligible subjects, or subjects may drop out of these clinical trials at a higher rate than we anticipate; |
• | clinical investigators or study subjects fail to comply with clinical study protocols; |
• | trial conduct and data analysis errors may occur, including, but not limited to, data entry and/or labeling errors; |
• | our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner, or at all; |
• | we might have to suspend or terminate clinical trials of our product candidates for various reasons, including a finding that the subjects are being exposed to unacceptable health risks; |
• | regulators or institutional review boards may require that we or our investigators suspend or terminate clinical research for various reasons, including noncompliance with regulatory requirements; |
• | the cost of clinical trials of our product candidates may be greater than we anticipate; |
• | the supply or quality of our clinical trial materials or other materials necessary to conduct clinical trials of our product candidates may be insufficient or inadequate; and |
• | our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators to suspend or terminate the trials. |
• | inability to raise funding necessary to initiate or continue a trial; |
• | delays in obtaining regulatory approval to commence a trial; |
• | delays in reaching agreement with the FDA or other regulatory authorities on final trial design; |
• | imposition of a clinical hold following a reported safety event; |
• | an inspection of our clinical trial operations or trial sites by the FDA or other regulatory authorities; |
• | delays in reaching agreement on acceptable terms with prospective contract research organizations (CROs) and clinical trial sites; |
• | delays in obtaining required institutional review board (IRB) approval at each site; |
• | delays in recruiting suitable patients to participate in a trial; |
• | delays in having subjects complete participation in a trial or return for post-treatment follow-up; |
• | delays caused by subjects dropping out of a trial due to side effects or otherwise; |
• | changes to treatment guidelines or the introduction of a new standard of care; |
• | delays caused by clinical sites dropping out of a trial; |
• | time required to add new clinical sites; |
• | delays by our contract manufacturers to produce and deliver sufficient supply of clinical trial materials; and |
• | delays in importing clinical trial materials into foreign countries where our clinical trials are being conducted. |
• | regulatory authorities may withdraw their approval of the product or impose restrictions on its distribution in a form of a risk evaluation and mitigation strategy (REMS) plan; |
• | regulatory authorities may require the addition of labeling statements, such as black box or other warnings or contraindications that could diminish the usage of the product or otherwise limit the commercial success of the affected product; |
• | we may be required to change the way the product is administered or to conduct additional clinical studies; |
• | we may choose to discontinue sale of the product; |
• | we could be sued and held liable for harm caused to patients; or |
• | our reputation may suffer. |
• | the efficacy and safety, as demonstrated in clinical studies; |
• | the risk/benefit profile of our product candidates; |
• | the prevalence and severity of any side effects; |
• | the clinical indications for which the product is approved; |
• | acceptance of the product by physicians, other health care providers and patients as a safe and effective treatment; |
• | the potential and perceived advantages of product candidates over alternative treatments; |
• | the safety of product candidates seen in a broader patient group, including if physicians prescribe our products for uses outside the approved indications; |
• | the cost of treatment in relation to alternative treatments; |
• | the timing of market introduction of competitive products; |
• | the availability of coverage and adequate reimbursement by third party payors and government authorities; |
• | relative convenience and ease of administration; and |
• | the effectiveness of our or our partners’ sales, marketing and distribution efforts. |
• | we may be unable to demonstrate to the satisfaction of regulatory authorities that a product candidate is safe and effective for any indication; |
• | regulatory authorities may not find the data from nonclinical studies and clinical studies sufficient or may differ in the interpretation of the data; |
• | regulatory authorities may require additional nonclinical or clinical studies; |
• | the FDA or foreign regulatory authority might not approve our third party manufacturers’ processes or facilities for clinical or commercial product; |
• | the FDA or foreign regulatory authority may change its approval policies or adopt new regulations; |
• | the FDA or foreign regulatory authority may disagree with the design or implementation of our clinical studies; |
• | the FDA or foreign regulatory authority may not accept clinical data from studies that are conducted in countries where the standard of care is potentially different from that in the United States; |
• | the results of clinical studies may not meet the level of statistical significance required by the FDA or foreign regulatory authorities for approval; |
• | we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks; and |
• | the data collection from clinical studies of our product candidates may not be sufficient to support the submission of a new drug application (NDA), marketing authorization or other equivalent submission, or to obtain regulatory approval in the United States or elsewhere. |
• | issue an untitled or warning letter asserting violation of the law; |
• | seek an injunction or impose civil or criminal penalties up to and including imprisonment or monetary fines; |
• | suspend or withdraw regulatory approval; |
• | suspend any ongoing clinical trials; |
• | refuse to approve a pending NDA or supplements to an NDA; or |
• | request recall and/or seize product. |
• | the inability to meet our product specifications, including product formulation, and quality requirements consistently; |
• | a delay or inability to procure or expand sufficient manufacturing capacity; |
• | manufacturing and product quality issues, including those related to scale-up of manufacturing; |
• | costs and validation of new equipment and facilities required for scale-up; |
• | a failure to comply with cGMP and similar quality standards; |
• | the inability to negotiate manufacturing agreements with third parties under commercially reasonable terms; |
• | termination or nonrenewal of manufacturing agreements with third parties in a manner or at a time that is costly or damaging to us; |
• | the reliance on a limited number of sources, and in some cases, single sources for key materials, such that if we are unable to secure a sufficient supply of these key materials, we will be unable to manufacture and sell our product candidates in a timely fashion, in sufficient quantities or under acceptable terms; |
• | the lack of qualified backup suppliers for those materials that are currently purchased from a sole or single source supplier; |
• | operations of our third-party manufacturers or suppliers could be disrupted by conditions unrelated to our business or operations, including the bankruptcy of the manufacturer or supplier; |
• | disruption of the distribution of chemical supplies between the U.K. and E.U. due to Brexit; |
• | carrier disruptions or increased costs that are beyond our control; and |
• | the failure to deliver our products under specified storage conditions and in a timely manner. |
• | demonstration of clinical safety and efficacy in our clinical trials; |
• | the risk/benefit profile of our product candidates; |
• | the relative convenience, ease of administration and acceptance by physicians, patients and health care payors; |
• | the prevalence and severity of any side effects; |
• | the safety of product candidates seen in a broader patient group, including its use outside the approved indications; |
• | limitations or warnings contained in the FDA and other regulatory authorities approved label for the relevant product candidate; |
• | acceptance of the product by physicians, other health care providers and patients as a safe and effective treatment; |
• | the potential and perceived advantages of product candidates over alternative treatments; |
• | the timing of market introduction of competitive products; |
• | pricing and cost-effectiveness; |
• | the effectiveness of our or any future collaborators’ sales and marketing strategies; |
• | our ability to obtain formulary approval; |
• | our ability to obtain and maintain sufficient third-party coverage or reimbursement, which may vary from country to country; and |
• | the effectiveness of our or any future collaborators’ sales, marketing and distribution efforts. |
• | different regulatory requirements for drug approvals in foreign countries; |
• | reduced protection for intellectual property rights; |
• | unexpected changes in tariffs, trade barriers and regulatory requirements; |
• | differing payor reimbursement regimes, governmental payors or patient self-pay systems and price controls; |
• | economic weakness, including inflation, or political instability in particular foreign economies and markets; |
• | compliance with tax, employment, immigration and labor laws for employees living or traveling abroad; |
• | foreign taxes, including withholding of payroll taxes; |
• | foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, and other obligations incident to doing business in another country; |
• | workforce uncertainty in countries where labor unrest is more common than in the United States; |
• | production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and |
• | business interruptions resulting from geopolitical actions, including war and terrorism, pandemics, or natural disasters including earthquakes, typhoons, volcanic eruptions, floods and fires. |
• | research and development resources, including personnel and technology; |
• | regulatory experience; |
• | experience in pharmaceutical development and commercialization; |
• | ability to negotiate competitive pricing and reimbursement with third-party payors; |
• | experience and expertise in the exploitation of intellectual property rights; and |
• | capital resources. |
• | decreased demand for our product candidates; |
• | impairment to our business reputation; |
• | withdrawal of clinical study participants; |
• | distraction of management’s attention from our primary business; |
• | substantial monetary awards to patients or other claimants; |
• | the inability to commercialize our product candidates; and |
• | loss of revenues. |
• | adverse or inconclusive results or delays in preclinical testing or clinical trials; |
• | inability to obtain additional funding; |
• | any delay in filing an Investigational New Drug (IND) application or NDA for any of our future product candidates and any adverse development or perceived adverse development with respect to the FDA’s review of an IND or NDA; |
• | failure to maintain our existing collaborations or enter into new collaborations; |
• | failure of our collaboration partners to elect to develop or commercialize product candidates under our collaboration agreements or the termination of any programs under our collaboration agreements; |
• | failure by us or our licensors and collaboration partners to prosecute, maintain or enforce our intellectual property rights; |
• | failure to successfully develop and commercialize our future product candidates; |
• | changes in laws or regulations applicable to future products; |
• | changes in the structure of payment systems; |
• | inability to obtain adequate product supply for our future product candidates or the inability to do so at acceptable prices; |
• | adverse regulatory decisions; |
• | introduction of new products, services or technologies by our competitors; |
• | failure to meet or exceed financial projections we may provide to the public; |
• | failure to meet or exceed the estimates and projections of the investment community; |
• | the perception of the pharmaceutical industry by the public, legislatures, regulators and the investment community; |
• | announcements of significant acquisitions, strategic partnerships, joint ventures or capital commitments by us, our collaboration partners or our competitors; |
• | announcements of significant or potential equity or debt sales by us; |
• | announcements of clinical trial plans or results by us; |
• | disputes or other developments relating to proprietary rights, including patents, litigation matters and our ability to obtain patent protection for our technologies; |
• | additions or departures of key scientific or management personnel; |
• | significant lawsuits, including patent or stockholder litigation; |
• | changes in the market valuations of similar companies; |
• | sales of our common stock by us or our stockholders in the future; and |
• | trading volume of our common stock. |
Item 6. |
Exhibits |
CYMABAY THERAPEUTICS, INC. | ||
By: | /s/ Sujal Shah | |
Sujal Shah | ||
President and Chief Executive Officer | ||
(Principal Executive Officer) | ||
Date: | August 10, 2020 | |
By: | /s/ Daniel Menold | |
Daniel Menold | ||
Vice President, Finance | ||
(Principal Financial Officer) | ||
Date: | August 10, 2020 |