☒ | ANNUAL 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 |
Delaware | 04-3508648 | ||
(State or other jurisdiction of incorporation or organization) | (I.R.S. Employer Identification No.) | ||
Four Tower Bridge 200 Barr Harbor Drive, Suite 200 West Conshohocken, Pennsylvania | 19428 | ||
(Address of Principal Executive Offices) | (Zip Code) |
Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||
Common Stock, $0.0001 Par Value Per Share | MDGL | The NASDAQ Stock Market LLC |
Large accelerated filer | ☒ | Accelerated filer | ☐ | |||
Non-accelerated filer | ☐ | Smaller reporting company | ☐ | |||
Emerging growth company | ☐ |
Auditor Firm Id: 238 | Auditor Name: PricewaterhouseCoopers LLP | Auditor Location: Philadelphia, Pennsylvania |
Page | ||||||
Item 1. | ||||||
Item 1A. | ||||||
Item 1B. | ||||||
Item 2. | ||||||
Item 3. | ||||||
Item 4. | ||||||
Item 5. | ||||||
Item 6. | ||||||
Item 7. | ||||||
Item 7A. | ||||||
Item 8. | ||||||
Item 9. | ||||||
Item 9A. | ||||||
Item 9B. | ||||||
Item 10. | ||||||
Item 11. | ||||||
Item 12. | ||||||
Item 13. | ||||||
Item 14. | ||||||
Item 15. | ||||||
Item 16. | ||||||
75 |
Item 1. | Business |
Resmetirom 80 mg | Resmetirom 100 mg | Placebo | ||||
Safety population | (N=327) | (N=324) | (N=318) | |||
At least one TEAE | 289 (88.4) | 279 (86.1) | 260 (81.8) | |||
At least one Serious TEAE | 20 (6.1) | 24 (7.4) | 20 (6.3) | |||
TEAE ≥ Grade 3 Severity | 26 (8.0) | 29 (9.0) | 29 (9.1) | |||
AE discontinuations from study | All treatments combined, n=21; (2.17%) | |||||
Maximum NCI CTCAE Severity Grade | ||||||
Grade 1 | 99 (30.3) | 99 (30.6) | 92 (28.9) | |||
Grade 2 | 164 (50.2) | 151 (46.6) | 139 (43.7) | |||
AEs over 10% | ||||||
Diarrhea | 76 (23.2) | 101 (31.2) | 44 (13.8) | |||
Nausea | 38 (11.6) | 59 (18.2) | 25 (7.9) |
Resmetirom 100 mg OL | Resmetirom 80 mg | p-value | Resmetirom 100 mg | p-value | Placebo | |||||||
LDLc %CFB (SE) (Week 24) | -21 (1.9) | -12.7 (2.1) | <.0001 | -14.4 (2.1) | <.0001 | -1.7 (2.0) | ||||||
ApoB %CFB (SE) (Week 24) | -22 (1.5) | -14.6 (1.5) | <.0001 | -16.6 (1.6) | <.0001 | -0.1 (1.5) | ||||||
MRI-PDFF %CFB (Week 16) | -49% | -41% | <.0001 | -48% | <.0001 | -6% | ||||||
Liver volume PDFF correction %CFB | -60% | |||||||||||
MRI-PDFF %CFB (Week 52) | -53% | -43% | <.0001 | -48% | <.0001 | -8% | ||||||
Liver volume PDFF correction %CFB | -61% | |||||||||||
Triglycerides baseline >150 mg/dL, CFB (SE) | -65 (8.3) | -55.6 (8.6) | NA | -59 (6.5) | NA | -6.9 (16.1) | ||||||
Triglycerides baseline >150 mg/dL (geomean) %CFB (95% CI) | -25 (3.1) | -19.5 (-27.0 to -11.1) | =.0005 | -21.5 (-28.0 to -14.3) | <.0001 | -2.1 (-10.6 to 7.4) |
MGL-3196 | MGL-3196 MRI-PDFF Responders(1) | Placebo | ||||||||||
Number of patients with baseline and end-of-study liver biopsies(2) | 73 | 46 | 34 | |||||||||
> 2 Point Decrease in NAS | 56% | 70% | 32 | % | ||||||||
p=0.02 | p=0.001 | |||||||||||
NASH Resolution | 27% | 39% | 6 | % | ||||||||
p=0.02 | p=0.001 |
Resmetirom | Resmetriom MRI-PDFF Responders(1) | Placebo | ||||
Number of patients with baseline and end-of-study | 73 | 46 | 34 | |||
>2 Point Decrease in NAS | 56% | 70% | 32% | |||
p=0.02 | p=0.001 | |||||
NASH Resolution | 27% | 39% | 6% | |||
p=0.02 | p=0.001 |
(1) | resmetirom MRI-PDFF Responders = resmetirom treated patients with >=30% relative fat reduction on Week 12MRI-PDFF |
(2) | does not include one end-of-study |
intended to establish the overall risk-benefit ratio of the product candidate and provide, if appropriate, an adequate basis for product approval and product labeling. In most cases, the FDA requires two adequate and well controlled Phase 3 clinical trials to demonstrate the efficacy of the product candidate, although a single Phase 3 clinical trial with other confirmatory evidence may be sufficient in certain instances. |
Item 1A. | Risk Factors |
Item 1B. | Unresolved Staff Comments |
Item 2. | Properties |
Item 3. | Legal Proceedings |
Item 4. | Mine Safety Disclosures |
Item 5. | Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchase of Equity Securities. |
Item 6. | Selected Financial Data |
Years Ended December 31, | ||||||||||||||||||||
2019 | 2018 | 2017 | 2016 | 2015 | ||||||||||||||||
(in thousands, except per share amounts) | ||||||||||||||||||||
Consolidated Statements of Operations Data: | ||||||||||||||||||||
Revenues: | ||||||||||||||||||||
Total revenues | $ | — | $ | — | $ | — | $ | — | $ | — | ||||||||||
Operating expenses: | ||||||||||||||||||||
Research and development | 72,324 | 25,389 | 24,390 | 15,933 | 2,427 | |||||||||||||||
General and administrative | 22,648 | 15,293 | 7,672 | 9,290 | 806 | |||||||||||||||
Total operating expenses | 94,972 | 40,682 | 32,062 | 25,223 | 3,233 | |||||||||||||||
Loss from operations | (94,972 | ) | (40,682 | ) | (32,062 | ) | (25,223 | ) | (3,233 | ) | ||||||||||
Interest income (expense), net | 11,024 | 7,671 | 558 | (1,165 | ) | (3,612 | ) | |||||||||||||
Other income | — | 200 | 350 | — | — | |||||||||||||||
Net loss | $ | (83,948 | ) | $ | (32,811 | ) | $ | (31,154 | ) | $ | (26,388 | ) | $ | (6,845 | ) | |||||
Net loss per common share: | ||||||||||||||||||||
Basic and diluted net loss per common share | $ | (5.45 | ) | $ | (2.22 | ) | $ | (2.54 | ) | $ | (5.07 | ) | $ | (40.03 | ) | |||||
Basic and diluted weighted average number of common shares outstanding | 15,394,659 | 14,796,712 | 12,244,939 | 5,204,644 | 171,012 |
As of December 31, | ||||||||||||||||||||
2019 | 2018 | 2017 | 2016 | 2015 | ||||||||||||||||
(in thousands) | ||||||||||||||||||||
Consolidated Balance Sheet Data: | ||||||||||||||||||||
Cash, cash equivalents and marketable securities | $ | 439,045 | $ | 483,718 | $ | 191,527 | $ | 40,500 | $ | 306 | ||||||||||
Total assets | 442,056 | 485,428 | 192,313 | 41,210 | 364 | |||||||||||||||
Total liabilities | 25,491 | 8,444 | 10,054 | 4,800 | 49,277 | |||||||||||||||
Accumulated deficit | (223,220 | ) | (139,272 | ) | (106,461 | ) | (75,307 | ) | (48,920 | ) | ||||||||||
Total stockholders’ equity (deficit) | $ | 416,565 | $ | 476,984 | $ | 182,259 | $ | 36,410 | $ | (48,913 | ) |
Years Ended December 31, | ||||||||||||||||||||
2021 | 2020 | 2019 | 2018 | 2017 | ||||||||||||||||
(in thousands, except per share amounts) | ||||||||||||||||||||
Consolidated Statements of Operations Data: | ||||||||||||||||||||
Revenues: | ||||||||||||||||||||
Total revenues | $ | — | $ | — | $ | — | $ | — | $ | — | ||||||||||
Operating expenses: | ||||||||||||||||||||
Research and development | 205,164 | 184,809 | 72,324 | 25,389 | 24,390 | |||||||||||||||
General and administrative | 37,318 | 21,864 | 22,648 | 15,293 | 7,672 | |||||||||||||||
Total operating expenses | 242,482 | 206,673 | 94,972 | 40,682 | 32,062 | |||||||||||||||
Loss from operations | (242,482 | ) | (206,673 | ) | (94,972 | ) | (40,682 | ) | (32,062 | ) | ||||||||||
Interest income (expense), net | 363 | 4,329 | 11,024 | 7,671 | 558 | |||||||||||||||
Other income | 273 | 100 | — | 200 | 350 | |||||||||||||||
Net loss | $ | (241,846 | ) | $ | (202,244 | ) | $ | (83,948 | ) | $ | (32,811 | ) | $ | (31,154 | ) | |||||
Net loss per common share: | ||||||||||||||||||||
Basic and diluted net loss per common share | $ | (14.63 | ) | $ | (13.09 | ) | $ | (5.45 | ) | $ | (2.22 | ) | $ | (2.54 | ) | |||||
Basic and diluted weighted average number of common shares outstanding | 16,535,188 | 15,446,638 | 15,394,659 | 14,796,712 | 12,244,939 |
Years Ended December 31, | ||||||||||||||||||||
2021 | 2020 | 2019 | 2018 | 2017 | ||||||||||||||||
(in thousands) | ||||||||||||||||||||
Consolidated Balance Sheet Data: | ||||||||||||||||||||
Cash, cash equivalents and marketable securities | $ | 270,346 | $ | 284,149 | $ | 439,045 | $ | 483,718 | $ | 191,527 | ||||||||||
Total assets | 273,332 | 286,995 | 442,056 | 485,428 | 192,313 | |||||||||||||||
Total liabilities | 77,225 | 47,025 | 25,491 | 8,444 | 10,054 | |||||||||||||||
Accumulated deficit | (667,310 | ) | (425,464 | ) | (223,220 | ) | (139,272 | ) | (106,461 | ) | ||||||||||
Total stockholders’ equity | $ | 196,107 | $ | 239,970 | $ | 416,565 | $ | 476,984 | $ | 182,259 |
Item 7. | Management’s Discussion and Analysis of Financial Condition and Results of Operations. |
Year ended December 31, | Increase / (Decrease) | |||||||||||||||
2019 | 2018 | $ | % | |||||||||||||
Research and Development Expenses | $ | 72,324 | $ | 25,389 | 46,935 | 185 | % | |||||||||
General and Administrative Expenses | 22,648 | 15,293 | 7,355 | 48 | % | |||||||||||
Interest (Income) | (11,024 | ) | (7,671 | ) | 3,353 | 44 | % | |||||||||
Other (Income) | — | (200 | ) | (200 | ) | (100 | %) | |||||||||
$ | 83,948 | $ | 32,811 | 51,137 | 156 | % |
Year ended December 31, | Increase / (Decrease) | |||||||||||||||
2021 | 2020 | $ | % | |||||||||||||
Research and Development Expenses | $ | 205,164 | $ | 184,809 | 20,355 | 11 | % | |||||||||
General and Administrative Expenses | 37,318 | 21,864 | 15,454 | 71 | % | |||||||||||
Interest (Income) | (363 | ) | (4,329 | ) | (3,966 | ) | (92 | %) | ||||||||
Other (income) | (273 | ) | (100 | ) | 173 | 173 | % | |||||||||
$ | 241,846 | $ | 202,244 | 39,602 | 20 | % |
Year ended December 31, | Increase / (Decrease) | |||||||||||||||
2020 | 2019 | $ | % | |||||||||||||
Research and Development Expenses | $ | 184,809 | $ | 72,324 | 112,485 | 156 | % | |||||||||
General and Administrative Expenses | 21,864 | 22,648 | (784 | ) | (3 | %) | ||||||||||
Interest (Income) | (4,329 | ) | (11,024 | ) | (6,695 | ) | (61 | %) | ||||||||
Other (income) | (100 | ) | — | 100 | 100 | % | ||||||||||
$ | 202,244 | $ | 83,948 | 118,296 | 141 | % |
Year ended December 31, | Increase / (Decrease) | |||||||||||||||
2018 | 2017 | $ | % | |||||||||||||
Research and Development Expenses | $ | 25,389 | $ | 24,390 | 999 | 4 | % | |||||||||
General and Administrative Expenses | 15,293 | 7,672 | 7,621 | 99 | % | |||||||||||
Interest (Income) | (7,671 | ) | (558 | ) | 7,113 | 1275 | % | |||||||||
Other (Income) | (200 | ) | (350 | ) | (150 | ) | (43 | %) | ||||||||
$ | 32,811 | $ | 31,154 | 1,657 | 5 | % |
Year ended December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
Net cash used in operating activities | $ | (41,624 | ) | $ | (25,507 | ) | $ | (22,317 | ) | |||
Net cash (used in) provided by investing activities | 30,707 | (380,076 | ) | (22,006 | ) | |||||||
Net cash provided by financing activities | 235 | 314,335 | 173,805 | |||||||||
Net (decrease) increase in cash and cash equivalents | $ | (10,682 | ) | $ | (91,248 | ) | $ | 129,482 |
Year ended December 31, | ||||||||||||
2021 | 2020 | 2019 | ||||||||||
Net cash used in operating activities | $ | (183,917 | ) | $ | (157,561 | ) | $ | (41,624 | ) | |||
Net cash (used in) provided by investing activities | (5,055 | ) | 159,780 | 30,707 | ||||||||
Net cash provided by financing activities | 171,237 | 5,088 | 235 | |||||||||
Net (decrease) increase in cash and cash equivalents | $ | (17,735 | ) | $ | 7,307 | $ | (10,682 | ) |
Payments Due by Period | ||||||||||||||||||||
Contractual Obligations | Total | Less Than 1 Year | 1 - 3 Years | 4 - 5 Years | More Than 5 Years | |||||||||||||||
Operating Leases | 765 | 363 | 402 | — | — | |||||||||||||||
Total Contractual Obligations | 765 | 363 | 402 | — | — | |||||||||||||||
Payments Due by Period | ||||||||||||||||||||
Contractual Obligations | Total | Less Than 1 Year | 1 - 3 Years | 4 - 5 Years | More Than 5 Years | |||||||||||||||
Operating Leases | 836 | 436 | 400 | — | — | |||||||||||||||
Total contractual Obligations | 836 | 436 | 400 | — | — | |||||||||||||||
Item 7A. | Quantitative and Qualitative Disclosures About Market Risk. |
Item 8. | Financial Statements and Supplementary Data. |
Item 9. | Changes in and Disagreements with Accountants on Accounting and Financial Disclosure. |
Item 9A. | Controls and Procedures. |
Item 9B. | Other Information. |
Item 10. | Directors, Executive Officers and Corporate Governance. |
Item 11. | Executive Compensation. |
Item 12. | Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters. |
Item 13. | Certain Relationships and Related Transactions, and Director Independence. |
Item 14. | Principal Accountant Fees and Services |
Item 15. | EXHIBITS AND FINANCIAL STATEMENT SCHEDULES | ||
Item 15(a) | The following documents are filed as part of, or incorporated by reference into, this Annual Report on Form 10-K: | ||
Item 15(a)(1) and (2) | The Consolidated Financial Statements beginning on page F-1 are filed as part of this Annual Report onForm 10-K. Other financial statement schedules have been omitted because the information required to be presented in them is not applicable or is shown in the financial statements or related notes. | ||
Item 15(a)(3) | We have filed, or incorporated into this Annual Report on Form 10-K by reference, the exhibits listed on the accompanying Exhibit Index. | ||
Item 15(b) | See Item 15(a)(3) above. | ||
Item 15(c) | See Item 15(a)(2) above. |
Exhibit Number | Exhibit Description | Filed Herewith | Reference herein from Form or Schedule | Filing Date | SEC File / Registration Number | |||||||||||||||
2.1 | ||||||||||||||||||||
DEFA14A; 8-K (Exhibit 2.1) | 4/14/2016 | 001-33277 | ||||||||||||||||||
3.1 | ||||||||||||||||||||
Form 10-K (Exhibit 3.1) | 3/31/2017 | 001-33277 | ||||||||||||||||||
3.2 | ||||||||||||||||||||
Form 8-K (Exhibit 3.1) | 6/21/2017 | 001-33277 | ||||||||||||||||||
3.3 | ||||||||||||||||||||
DEFA14A; 8-K (Exhibit 3.1) | 4/14/2016 | 001-33277 | ||||||||||||||||||
4.1 | ||||||||||||||||||||
Form 10-K (Exhibit 4.1) | 2/26/20 | 001-33277 | ||||||||||||||||||
4.2 | ||||||||||||||||||||
Form 8-K (Exhibit 10.1) | 6/21/2017 | 001-33277 | ||||||||||||||||||
Equity Agreements | ||||||||||||||||||||
10.1 | Sales Agreement, dated June 1, 2021, by and between Madrigal Pharmaceuticals, Inc. and Cowen and Company, LLC (concerning at-the-market offerings of Madrigal common stock). | Form 8-K (Exhibit 1.1) | 6/1/2021 | 001-33277 |
Exhibit Number | Exhibit | Filed Herewith | Reference herein from Form or Schedule | Filing Date | Registration Number | |||||||||||||||||||||
Agreements with Respect to Collaborations, Licenses, Research and Development | ||||||||||||||||||||||||||
10.2 | ||||||||||||||||||||||||||
| Form 10-Q (Exhibit 10.5) | 11/14/2016 | 001-33277 |
Number | Herewith | ||||||||||||||||||||||||||||||
Equity Compensation Plans | |||||||||||||||||||||||||||||||
10.3* | Amended 2015 Stock Plan | | Definitive Proxy Statement (Annex A) | | 4/30/2021 | 001-33277 | |||||||||||||||||||||||||
10.4* | |||||||||||||||||||||||||||||||
| Form 10-K (Exhibit 10.10) | | 3/31/2017 | 001-33277 | |||||||||||||||||||||||||||
10.5* | |||||||||||||||||||||||||||||||
| Form 10-K (Exhibit 10.11) | | 3/31/2017 | 001-33277 | |||||||||||||||||||||||||||
10.6* | |||||||||||||||||||||||||||||||
| Form 10-K (Exhibit 10.13) | | 3/31/2017 | 001-33277 | |||||||||||||||||||||||||||
10.7* | |||||||||||||||||||||||||||||||
| Form 10-Q (Exhibit 10.1) | | 5/10/2016 | 001-33277 | |||||||||||||||||||||||||||
10.8* | Non-Employee Director Equity Compensation Policy | | Form 10-Q (Exhibit 10.1) | | 5/6/2021 | 001-33277 | |||||||||||||||||||||||||
Agreements with Executive Officers and Directors | |||||||||||||||||||||||||||||||
10.9* | |||||||||||||||||||||||||||||||
| Form 8-K (Exhibit 10.2) | | 001-33277 | ||||||||||||||||||||||||||||
10.10* | |||||||||||||||||||||||||||||||
| Form 8-K (Exhibit 10.3) | | 7/22/2016 | 001-33277 | |||||||||||||||||||||||||||
10.11* | |||||||||||||||||||||||||||||||
| Form 8-K (Exhibit 10.4) | | 7/22/2016 | 001-33277 | |||||||||||||||||||||||||||
21.1 | List of Subsidiaries. | ||||||||||||||||||||||||||||||
X | |||||||||||||||||||||||||||||||
23.1 | |||||||||||||||||||||||||||||||
X |
Exhibit Number | Exhibit Description | Filed Herewith | Incorporated by Reference herein from Form or Schedule | Filing Date | SEC File / Registration Number | |||||||||||||||
31.1 | ||||||||||||||||||||
X | ||||||||||||||||||||
31.2 | ||||||||||||||||||||
X | ||||||||||||||||||||
32.1** | ||||||||||||||||||||
X | ||||||||||||||||||||
101.INS | Inline XBRL Instance Document. | X | ||||||||||||||||||
101.SCH | ||||||||||||||||||||
Inline XBRL Taxonomy Extension Schema Document. | X | |||||||||||||||||||
101.CAL | ||||||||||||||||||||
Inline XBRL Taxonomy Extension Calculation Linkbase Document. | X | |||||||||||||||||||
101.DEF | ||||||||||||||||||||
Inline XBRL Taxonomy Extension Definition Linkbase Document. | X | |||||||||||||||||||
101.LAB | ||||||||||||||||||||
Inline XBRL Taxonomy Extension Label Linkbase Document. | X | |||||||||||||||||||
101.PRE | ||||||||||||||||||||
Inline XBRL Taxonomy Extension Presentation Linkbase Document. | X | |||||||||||||||||||
104 | ||||||||||||||||||||
Inline XBRL for the cover page of this Annual Report on Form 10-K, included in the Exhibit 101 Inline XBRL Document Set. | X |
* | Indicates a management contract, compensatory plan or arrangement. |
** | The certifications attached as Exhibit 32.1 that accompany this Annual Report on Form 10-K are not deemed filed with the SEC and are not to be incorporated by reference into any filing of the registrant under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date of this Annual Report on Form10-K, regardless of any general incorporation language contained in any filing. |
† | Confidential portions of these documents have been filed separately with the Securities and Exchange Commission pursuant to a request for confidential treatment. |
Item 16. | Form 10-K Summary. |
MADRIGAL PHARMACEUTICALS INC. | ||||||||
Date: February 24, 2022 | By: | /s/ PAUL A. FRIEDMAN, M.D. | ||||||
Paul A. Friedman, M.D. | ||||||||
(Principal Executive Officer) |
Signatures | Title | Date | ||||
/s/ PAUL A. FRIEDMAN, M.D. Paul A. Friedman, M.D. | Chairman of the Board of Directors and Chief Executive Officer (Principal Executive Officer) | February | ||||
/s/ | February | |||||
/s/ REBECCA TAUB, M.D. Rebecca Taub, M.D. | Director | February | ||||
/s/ FRED B. CRAVES, PH.D. Fred B. Craves, Ph.D. | Director | February | ||||
/s/ KENNETH M. BATE Kenneth M. Bate | Director | February | ||||
/s/ KEITH R. GOLLUST Keith R. Gollust | Director | February |
24, 2022 | ||||||
/s/ DAVID MILLIGAN, PH.D. David Milligan, Ph.D. | Director | February 24, 2022 |
Signatures | Title | Date | ||||
/s/ RICHARD S. LEVY, M.D. Richard S. Levy, M.D. | Director | February | ||||
/s/ JAMES M. DALY James M. Daly | Director | February |
Page | |||||
F-2 | |||||
F-5 | |||||
F-6 | |||||
F-7 | |||||
F-8 | |||||
F-9 | |||||
F-10 |
December 31, 2019 | December 31, 2018 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 46,697 | $ | 57,379 | ||||
Marketable securities | 392,348 | 426,339 | ||||||
Prepaid expenses and other current assets | 1,152 | 1,483 | ||||||
Total current assets | 440,197 | 485,201 | ||||||
Property and equipment, net | 1,184 | 227 | ||||||
Right-of-use asset | 675 | — | ||||||
Total assets | $ | 442,056 | $ | 485,428 | ||||
Liabilities and Stockholders’ Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 1,178 | $ | 2,487 | ||||
Accrued expenses | 23,637 | 5,957 | ||||||
Lease liability | 315 | — | ||||||
Total current liabilities | 25,130 | 8,444 | ||||||
Long term liabilities: | ||||||||
Lease liability | 361 | — | ||||||
Total long term liabilities | 361 | — | ||||||
Total liabilities | 25,491 | 8,444 | ||||||
Stockholders’ equity: | ||||||||
Preferred stock, par value $0.0001 per share authorized: 5,000,000 shares at December 31, 2019 and December 31, 2018; 1,969,797 shares issued and outstanding at December 31, 2019 and December 31, 2018 | — | — | ||||||
Common stock, par value $0.0001 per share authorized: 200,000,000 at December 31, 2019 and December 31, 2018; 15,429,154 and 15,409,023 shares issued and outstanding at December 31, 2019 and December 31, 2018, respectively | 2 | 2 | ||||||
Additional paid-in-capital | 639,567 | 616,573 | ||||||
Accumulated other comprehensive gain (loss) | 216 | (319 | ) | |||||
Accumulated deficit | (223,220 | ) | (139,272 | ) | ||||
Total stockholders’ equity | 416,565 | 476,984 | ||||||
Total liabilities and stockholders’ equity | $ | 442,056 | $ | 485,428 | ||||
December 31, 2021 | December 31, 2020 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 36,269 | $ | 54,004 | ||||
Marketable securities | 234,077 | 230,145 | ||||||
Prepaid expenses and other current assets | 1,338 | 1,014 | ||||||
Total current assets | 271,684 | 285,163 | ||||||
Property and equipment, net | 851 | 1,047 | ||||||
Right-of-use | 797 | 785 | ||||||
Total assets | $ | 273,332 | $ | 286,995 | ||||
Liabilities and Stockholders’ Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 21,380 | $ | 1,017 | ||||
Accrued expenses | 55,048 | 45,222 | ||||||
Lease liability | 410 | 318 | ||||||
Total current liabilities | 76,838 | 46,557 | ||||||
Long term liabilities: | ||||||||
Lease liability | 387 | 468 | ||||||
Total long term liabilities | 387 | 468 | ||||||
Total liabilities | 77,225 | 47,025 | ||||||
Stockholders’ equity: | ||||||||
Preferred stock, par value $0.0001 per share authorized: 5,000,000 shares at December 31, 2021 and December 31, 2020; 1,969,797 shares issued and outstanding at December 31, 2021 and December 31, 2020 | 0— | — | ||||||
Common stock, par value $0.0001 per share authorized: 200,000,000 at December 31, 2021 and December 31, 2020; 17,103,395 and 15,508,146 shares issued and outstanding at December 31, 2021 and December 31, 2020, respectively | 2 | 2 | ||||||
Additional paid-in-capital | 863,495 | 665,385 | ||||||
Accumulated other comprehensive gain (loss) | (80 | ) | 47 | |||||
Accumulated deficit | (667,310 | ) | (425,464 | ) | ||||
Total stockholders’ equity | 196,107 | 239,970 | ||||||
Total liabilities and stockholders’ equity | $ | 273,332 | $ | 286,995 | ||||
Year Ended December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
Revenues: | ||||||||||||
Total revenues | $ | — | $ | — | $ | — | ||||||
Operating expenses: | ||||||||||||
Research and development | 72,324 | 25,389 | 24,390 | |||||||||
General and administrative | 22,648 | 15,293 | 7,672 | |||||||||
Total operating expenses | 94,972 | 40,682 | 32,062 | |||||||||
Loss from operations | (94,972 | ) | (40,682 | ) | (32,062 | ) | ||||||
Interest income | 11,024 | 7,671 | 558 | |||||||||
Other income | — | 200 | 350 | |||||||||
Net loss | $ | (83,948 | ) | $ | (32,811 | ) | $ | (31,154 | ) | |||
Net loss per common share: | ||||||||||||
Basic and diluted net loss per common share | $ | (5.45 | ) | $ | (2.22 | ) | $ | (2.54 | ) | |||
Basic and diluted weighted average number of common shares outstanding | 15,394,659 | 14,796,712 | 12,244,939 |
Year Ended December 31, | ||||||||||||
2021 | 2020 | 2019 | ||||||||||
Revenues: | ||||||||||||
Total revenues | $ | 0 | $ | 0 | $ | 0 | ||||||
Operating expenses: | ||||||||||||
Research and development | 205,164 | 184,809 | 72,324 | |||||||||
General and administrative | 37,318 | 21,864 | 22,648 | |||||||||
Total operating expenses | 242,482 | 206,673 | 94,972 | |||||||||
Loss from operations | (242,482 | ) | (206,673 | ) | (94,972 | ) | ||||||
Interest income | 363 | 4,329 | 11,024 | |||||||||
Other income | 273 | 100 | 0 | |||||||||
Net loss | $ | (241,846) | $ | (202,244) | $ | (83,948) | ||||||
Net loss per common share: | ||||||||||||
Basic and diluted net loss per common share | $ | (14.63) | $ | (13.09) | $ | (5.45) | ||||||
Basic and diluted weighted average number of common shares outstanding | 16,535,188 | 15,446,638 | 15,394,659 |
Year Ended December 31, | ||||||||||||
2019 | 2018 | 201 7 | ||||||||||
Net Loss | $ | (83,948 | ) | $ | (32,811 | ) | $ | (31,154 | ) | |||
Other comprehensive income (loss): | ||||||||||||
Unrealized gain (loss) on available-for-sale securities | 535 | (288 | ) | (56 | ) | |||||||
Comprehensive loss | $ | (83,413 | ) | $ | (33,099 | ) | $ | (31,210 | ) | |||
Year Ended December 31, | ||||||||||||
2021 | 2020 | 2019 | ||||||||||
Net Loss | $ | (241,846 | ) | $ | (202,244 | ) | $ | (83,948 | ) | |||
Other comprehensive income (loss): | ||||||||||||
Unrealized gain (loss) on available-for-sale | (127 | ) | (169 | ) | 535 | |||||||
Comprehensive loss | $ | (241,973 | ) | $ | (202,413 | ) | $ | (83,413 | ) | |||
Preferred stock | Common stock | Additional paid-in Capital | Accumulated other comprehensive income (loss) | Accumulated deficit | Total stockholders’ equity | |||||||||||||||||||||||||||
Shares | Amount | Shares | Amount | |||||||||||||||||||||||||||||
Balance at December 31, 2016 | — | $ | — | 11,951,866 | $ | 1 | $ | 111,691 | $ | 25 | $ | (75,307 | ) | $ | 36,410 | |||||||||||||||||
Issuance of common and preferred shares in equity offerings, net of transaction costs | 1,969,797 | — | 2,275,768 | — | 173,805 | — | — | 173,805 | ||||||||||||||||||||||||
Compensation expense related to stock options for services | — | — | — | — | 3,254 | — | — | 3,254 | ||||||||||||||||||||||||
Unrealized loss on marketable securities | — | — | — | — | — | (56 | ) | — | (56 | ) | ||||||||||||||||||||||
Net loss | — | — | — | — | — | — | (31,154 | ) | (31,154 | ) | ||||||||||||||||||||||
Balance at December 31, 2017 | 1,969,797 | $ | — | 14,227,634 | $ | 1 | $ | 288,750 | $ | (31 | ) | $ | (106,461 | ) | $ | 182,259 | ||||||||||||||||
Issuance of common shares in equity offering, excluding to related parties, net of transaction costs | — | — | 1,079,580 | 1 | 311,824 | — | — | 311,825 | ||||||||||||||||||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs | — | — | 101,809 | — | 2,510 | — | — | 2,510 | ||||||||||||||||||||||||
Compensation expense related to stock options for services | — | — | — | — | 13,489 | — | — | 13,489 | ||||||||||||||||||||||||
Unrealized loss on marketable securities | — | — | — | — | — | (288 | ) | — | (288 | ) | ||||||||||||||||||||||
Net loss | — | — | — | — | — | — | (32,811 | ) | (32,811 | ) | ||||||||||||||||||||||
Balance at December 31, 2018 | 1,969,797 | $ | — | 15,409,023 | $ | 2 | $ | 616,573 | $ | (319 | ) | $ | (139,272 | ) | $ | 476,984 | ||||||||||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs | — | — | 20,131 | — | 235 | — | — | 235 | ||||||||||||||||||||||||
Compensation expense related to stock options for services | — | — | — | — | 22,759 | — | — | 22,759 | ||||||||||||||||||||||||
Unrealized gain on marketable securities | — | — | — | — | — | 535 | — | 535 | ||||||||||||||||||||||||
Net loss | — | — | — | — | — | — | (83,948 | ) | (83,948 | ) | ||||||||||||||||||||||
Balance at December 31, 2019 | 1,969,797 | $ | — | 15,429,154 | $ | 2 | $ | 639,567 | $ | 216 | $ | (223,220 | ) | $ | 416,565 | |||||||||||||||||
Additional paid-in Capital | Accumulated other comprehensive income (loss) | Accumulated deficit | Total stockholders’ equity | |||||||||||||||||||||||||||||
Preferred stock | Common stock | |||||||||||||||||||||||||||||||
Shares | Amount | Shares | Amount | |||||||||||||||||||||||||||||
Balance at December 31, 2018 | 1,969,797 | $ | — | 15,409,023 | $ | 2 | $ | 616,573 | $ | (319 | ) | $ | (139,272 | ) | $ | 476,984 | ||||||||||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs | — | — | 20,131 | — | 235 | — | — | 235 | ||||||||||||||||||||||||
Compensation expense related to stock options for services | — | �� | — | — | — | 22,759 | — | — | 22,759 | |||||||||||||||||||||||
Unrealized gain on marketable securities | — | — | — | — | — | 535 | — | 535 | ||||||||||||||||||||||||
Net loss | — | — | — | — | — | — | (83,948 | ) | (83,948 | ) | ||||||||||||||||||||||
Balance at December 31, 2019 | 1,969,797 | $ | — | 15,429,154 | $ | 2 | $ | 639,567 | $ | 216 | $ | (223,220 | ) | $ | 416,565 | |||||||||||||||||
Issuance of common shares in equity offering, excluding to related parties, net of transaction costs | — | — | 39,607 | 0 | 4,421 | — | — | 4,421 | ||||||||||||||||||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs | — | — | 39,385 | — | 667 | — | — | 667 | ||||||||||||||||||||||||
Compensation expense related to stock options for services | — | — | — | — | 20,730 | — | — | 20,730 | ||||||||||||||||||||||||
Unrealized loss on marketable securities | — | — | — | — | — | (169 | ) | — | (169 | ) | ||||||||||||||||||||||
Net loss | — | — | — | — | — | — | (202,244 | ) | (202,244 | ) | ||||||||||||||||||||||
Balance at December 31, 2020 | 1,969,797 | $ | — | 15,508,146 | $ | 2 | $ | 665,385 | $ | 47 | $ | (425,464 | ) | $ | 239,970 | |||||||||||||||||
Issuance of common shares in equity offering, excluding to related parties, net of transaction costs | — | — | 1,584,169 | — | 170,207 | — | — | 170,207 | ||||||||||||||||||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs | — | — | 11,080 | — | 1,030 | — | — | 1,030 | ||||||||||||||||||||||||
Compensation expense related to stock options for services | — | — | — | — | 26,873 | — | — | 26,873 | ||||||||||||||||||||||||
Unrealized loss on marketable securities | — | — | — | — | — | (127 | ) | — | (127 | ) | ||||||||||||||||||||||
Net loss | — | — | — | — | — | — | (241,846 | ) | (241,846 | ) | ||||||||||||||||||||||
Balance at December 31, 2021 | 1,969,797 | $ | — | 17,103,395 | $ | 2 | $ | 863,495 | $ | (80 | ) | $ | (667,310 | ) | $ | 196,107 | ||||||||||||||||
Years Ended December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
Cash flows from operating activities: | ||||||||||||
Net loss | $ | (83,948 | ) | $ | (32,811 | ) | $ | (31,154 | ) | |||
Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||||||
Stock-based compensation expense | 22,759 | 13,489 | 3,254 | |||||||||
Depreciation and amortization expense | 112 | 96 | 77 | |||||||||
Changes in operating assets and liabilities: | ||||||||||||
Prepaid expenses and other current assets | 332 | (998 | ) | 502 | ||||||||
Accounts payable | (1,309 | ) | 558 | 917 | ||||||||
Accrued expense | 16,783 | (2,168 | ) | 4,087 | ||||||||
Accrued interest, net of interest received on maturity of investments | 3,647 | (3,673 | ) | — | ||||||||
Net cash used in operating activities | (41,624 | ) | (25,507 | ) | (22,317 | ) | ||||||
Cash flows from investing activities: | ||||||||||||
Purchases of marketable securities | (619,303 | ) | (614,358 | ) | (70,211 | ) | ||||||
Sales and maturities of marketable securities | 650,182 | 234,304 | 48,330 | |||||||||
Purchases of property and equipment, net of disposals | (172 | ) | (22 | ) | (125 | ) | ||||||
Net cash provided by (used in) investing activities | 30,707 | (380,076 | ) | (22,006 | ) | |||||||
Cash flows from financing activities: | ||||||||||||
Proceeds from issuances of stock, excluding related parties, net of transaction costs | — | 311,825 | 173,805 | |||||||||
Proceeds from the sale of related party stock and exercise of common stock options, net of transaction costs | 235 | 2,510 | — | |||||||||
Net cash provided by financing activities | 235 | 314,335 | 173,805 | |||||||||
Net increase (decrease) in cash and cash equivalents | (10,682 | ) | (91,248 | ) | 129,482 | |||||||
Cash and cash equivalents at beginning of period | 57,379 | 148,627 | 19,145 | |||||||||
Cash and cash equivalents at end of period | $ | 46,697 | $ | 57,379 | $ | 148,627 | ||||||
Supplemental disclosure of cash flow information: | ||||||||||||
Obtaining a right-of-use asset in exchange for a lease liability | $ | 900 | $ | — | $ | — | ||||||
Purchases of property and equipment in accounts payable or accrued expense at period end | 897 | — | 250 |
Years Ended December 31, | ||||||||||||
2021 | 2020 | 2019 | ||||||||||
Cash flows from operating activities: | ||||||||||||
Net loss | $ | (241,846 | ) | $ | (202,244 | ) | $ | (83,948 | ) | |||
Adjustments to reconcile net loss to net cash used in operating activities: | ||||||||||||
Stock-based compensation expense | 26,873 | 20,730 | 22,759 | |||||||||
Depreciation and amortization expense | 405 | 471 | 112 | |||||||||
Changes in operating assets and liabilities: | ||||||||||||
Prepaid expenses and other current assets | (325 | ) | 138 | 332 | ||||||||
Accounts payable | 20,363 | (161 | ) | (1,309 | ) | |||||||
Accrued expense | 9,826 | 21,585 | 16,783 | |||||||||
Accrued interest, net of interest received on maturity of investments | 787 | 1,920 | 3,647 | |||||||||
Net cash used in operating activities | (183,917 | ) | (157,561 | ) | (41,624 | ) | ||||||
Cash flows from investing activities: | ||||||||||||
Purchases of marketable securities | (394,120 | ) | (329,342 | ) | (619,303 | ) | ||||||
Sales and maturities of marketable securities | 389,274 | 489,456 | 650,182 | |||||||||
Purchases of property and equipment, net of disposals | (209 | ) | (334 | ) | (172 | ) | ||||||
Net cash provided by (used in) investing activities | (5,055 | ) | 159,780 | 30,707 | ||||||||
Cash flows from financing activities: | ||||||||||||
Proceeds from issuances of stock, excluding related parties, net of transaction costs | 170,207 | 4,421 | — | |||||||||
Proceeds from the sale of related party stock and exercise of common stock options, net of transaction costs | 1,030 | 667 | 235 | |||||||||
Net cash provided by financing activities | 171,237 | 5,088 | 235 | |||||||||
Net increase (decrease) in cash and cash equivalents | (17,735 | ) | 7,307 | (10,682 | ) | |||||||
Cash and cash equivalents at beginning of period | 54,004 | 46,697 | 57,379 | |||||||||
Cash and cash equivalents at end of period | $ | 36,269 | $ | 54,004 | $ | 46,697 | ||||||
Supplemental disclosure of cash flow information: | ||||||||||||
Obtaining a right-of-use | $ | 376 | $ | 451 | $ | 900 | ||||||
Purchases of property and equipment in accounts payable at period end | — | — | 897 |
As of December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
Common stock options | 1,461,987 | 1,132,618 | 976,777 | |||||||||
Unvested restricted common stock | — | 52,063 | 104,127 | |||||||||
Preferred stock | 1,969,797 | 1,969,797 | 1,969,797 |
As of December 31, | ||||||||||||
2021 | 2020 | 2019 | ||||||||||
Common stock options | 2,301,574 | 1,837,540 | 1,461,987 | |||||||||
Unvested restricted common stock | 0 | 0 | 0 | |||||||||
Preferred stock | 1,969,797 | 1,969,797 | 1,969,797 |
December 31, 2019 | ||||||||||||||||
Cost | Unrealized gains | Unrealized losses | Fair value | |||||||||||||
Cash and cash equivalents: | ||||||||||||||||
Cash (Level 1) | $ | 1,772 | $ | — | $ | — | $ | 1,772 | ||||||||
Money market funds (Level 1) | 44,925 | — | — | 44,925 | ||||||||||||
Corporate debt securities due within 3 months of date of purchase (Level 2) | — | — | — | — | ||||||||||||
Total cash and cash equivalents | 46,697 | — | — | 46,697 | ||||||||||||
Marketable securities: | ||||||||||||||||
Corporate debt securities due within 1 year of date of purchase (Level 2) | 309,365 | 220 | (40 | ) | 309,545 | |||||||||||
Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) | 82,767 | 39 | (3 | ) | 82,803 | |||||||||||
Total cash, cash equivalents and marketable securities | $ | 438,829 | $ | 259 | $ | (43 | ) | $ | 439,045 | |||||||
December 31, 2018 | ||||||||||||||||
Cost | Unrealized gains | Unrealized losses | Fair value | |||||||||||||
Cash and cash equivalents: | ||||||||||||||||
Cash (Level 1) | $ | 2,004 | $ | — | $ | — | $ | 2,004 | ||||||||
Money market funds (Level 1) | 43,401 | — | — | 43,401 | ||||||||||||
Corporate debt securities due within 3 months of date of purchase (Level 2) | 11,974 | — | — | 11,974 | ||||||||||||
Total cash and cash equivalents | 57,379 | — | — | 57,379 | ||||||||||||
Marketable securities: | ||||||||||||||||
Corporate debt securities due within 1 year of date of purchase (Level 2) | 426,658 | 14 | (333 | ) | 426,339 | |||||||||||
Total cash, cash equivalents and marketable securities | $ | 484,037 | $ | 14 | $ | (333 | ) | $ | 483,718 | |||||||
December 31, 2021 | ||||||||||||||||
Cost | Unrealized gains | Unrealized losses | Fair value | |||||||||||||
Cash and cash equivalents: | ||||||||||||||||
Cash (Level 1) | $ | 18,877 | $ | — | $ | — | $ | 18,877 | ||||||||
Money market funds (Level 1) | 17,392 | — | — | 17,392 | ||||||||||||
Total cash and cash equivalents | 36,269 | — | — | 36,269 | ||||||||||||
Marketable securities: | ||||||||||||||||
Corporate debt securities due within 1 year of date of purchase (Level 2) | 228,348 | 6 | (66 | ) | 228,288 | |||||||||||
Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) | 5,809 | — | (20 | ) | 5,789 | |||||||||||
Total cash, cash equivalents and marketable securities | $ | 270,426 | $ | 6 | $ | (86 | ) | $ | 270,346 | |||||||
December 31, 2020 | ||||||||||||||||
Cost | Unrealized gains | Unrealized losses | Fair value | |||||||||||||
Cash and cash equivalents: | ||||||||||||||||
Cash (Level 1) | $ | 716 | $ | — | $ | — | $ | 716 | ||||||||
Money market funds (Level 1) | 53,288 | — | — | 53,288 | ||||||||||||
Total cash and cash equivalents | 54,004 | — | — | 54,004 | ||||||||||||
Marketable securities: | ||||||||||||||||
Corporate debt securities due within 1 year of date of purchase (Level 2) | 227,172 | 80 | (36 | ) | 227,216 | |||||||||||
Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) | 2,926 | 4 | (1 | ) | 2,929 | |||||||||||
Total cash, cash equivalents and marketable securities | $ | 284,102 | $ | 84 | $ | (37) | $ | 284,149 | ||||||||
December 31, 2019 | December 31, 2018 | |||||||
C ontract research costs | $ | 13,775 | $ | 571 | ||||
Compensation and benefits | 2,779 | 1,797 | ||||||
Professional fees | 1,177 | 1,062 | ||||||
Other | 5,906 | 2,527 | ||||||
$ | 23,637 | $ | 5,957 | |||||
December 31, 2021 | December 31, 2020 | |||||||
Contract research organization costs | $ | 38,349 | $ | 31,646 | ||||
Other clinical study related costs | 3,957 | 3,901 | ||||||
Compensation and benefits | 6,769 | 4,686 | ||||||
Professional fees | 2,455 | 830 | ||||||
Other | 3,518 | 4,159 | ||||||
Total accrued liabilities | $ | 55,048 | $ | 45,222 | ||||
Shares | Weighted average exercise price | Weighted average remaining contractual life (years) | Aggregate intrinsic value (in thousands) | |||||||||||||
Outstanding at January 1, 2019 | 1,132,618 | $ | 48.52 | |||||||||||||
Options granted | 378,500 | 118.70 | ||||||||||||||
Options exercised | (20,131 | ) | 11.67 | |||||||||||||
Options cancelled | (29,000 | ) | 175.78 | |||||||||||||
Outstanding at December 31, 2019 | 1,461,987 | $ | 64.67 | 7.37 | $ | 68,604 | ||||||||||
Exercisable at December 31, 2019 | 943,399 | $ | 40.38 | 6.94 | $ | 63,995 |
Shares | Weighted average exercise price | Weighted average remaining contractual life (years) | Aggregate intrinsic value (in thousands) | |||||||||||||
Outstanding at January 1, 2021 | 1,837,540 | $ | 71.80 | |||||||||||||
Options granted | 687,059 | 109.03 | ||||||||||||||
Options exercised | (11,080 | ) | 92.96 | |||||||||||||
Options cancelled | (211,945 | ) | 114.34 | |||||||||||||
Outstanding at December 31, 2021 | 2,301,574 | $ | 78.89 | 6.51 | $ | 62,566 | ||||||||||
Exercisable at December 31, 2021 | 1,432,144 | $ | 62.66 | 5.38 | $ | 60,932 |
Shares | Weighted average exercise price | |||||||
Outstanding at January 1, 2019 | 52,063 | $ | 9.45 | |||||
Vested | (52,063 | ) | 9.45 | |||||
Outstanding at December 31, 2019 | — | $ | — | |||||
Years Ended December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
Stock-based compensation expense by type of award: | ||||||||||||
Stock options | $ | 22,487 | $ | 12,997 | $ | 2,662 | ||||||
Restricted stock | 272 | 492 | 592 | |||||||||
Total stock-based compensation expense | $ | 22,759 | $ | 13,489 | $ | 3,254 | ||||||
Effect of stock-based compensation expense by line item: | ||||||||||||
Research and development | $ | 8,277 | $ | 3,707 | $ | 883 | ||||||
General and administrative | 14,482 | 9,782 | 2,371 | |||||||||
Total stock-based compensation expense included in net loss | $ | 22,759 | $ | 13,489 | $ | 3,254 | ||||||
Years Ended December 31, | ||||||||||||
2021 | 2020 | 2019 | ||||||||||
Stock-based compensation expense by type of award: | ||||||||||||
Stock options | $ | 26,873 | $ | 20,730 | $ | 22,487 | ||||||
Restricted stock | 0 | 0 | 272 | |||||||||
Total stock-based compensation expense | $ | 26,873 | $ | 20,730 | $ | 22,759 | ||||||
Effect of stock-based compensation expense by line item: | ||||||||||||
Research and development | $ | 10,698 | $ | 8,833 | $ | 8,277 | ||||||
General and administrative | 16,175 | 11,897 | 14,482 | |||||||||
Total stock-based compensation expense included in net loss | $ | 26,873 | $ | 20,730 | $ | 22,759 | ||||||
Operating Leases | ||||
2020 | $ | 363 | ||
2021 | 371 | |||
2022 | 31 | |||
Thereafter | — | |||
Total minimum payments | $ | 765 | ||
Less: imputed interest | 89 | |||
Present value of lease liabilities | $ | 676 |
Operating Leases | ||||
2022 | 436 | |||
2023 | 400 | |||
Thereafter | — | |||
Total minimum payments | $ | 836 | ||
Less: imputed interest | 39 | |||
Present value of lease liabilities | $ | 797 |
For the years ended December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
Deferred Tax Liabilities | ||||||||||||
Unrealized gains on investments | $ | 62 | $ | — | $ | — | ||||||
Total deferred tax liabilities | $ | 62 | $ | — | $ | — | ||||||
Deferred Tax Assets | ||||||||||||
Charitable contributions | $ | 15 | $ | 13 | $ | 4 | ||||||
Accrued expenses | 759 | 498 | 421 | |||||||||
Intangibles | 983 | 477 | 579 | |||||||||
Stock compensation | 10,943 | 4,395 | 605 | |||||||||
Property, plant & equipment | 13 | 9 | — | |||||||||
Unrealized loss on investment | — | 92 | 9 | |||||||||
Net operating losses | 17,635 | 14,851 | 9,229 | |||||||||
Capitalized R&D | 29,364 | 15,108 | 8,671 |
For the years ended December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
R&D credit | $ | 6,141 | $ | 3,505 | $ | 1,901 | ||||||
Total deferred tax assets before valuation allowance | 65,853 | 38,948 | 21,419 | |||||||||
Valuation allowance | (65,791 | ) | (38,948 | ) | (21,419 | ) | ||||||
Total deferred tax assets | 62 | — | — | |||||||||
Net deferred tax assets | $ | — | $ | — | $ | — | ||||||
For the years ended December 31, | ||||||||||||
2021 | 2020 | 2019 | ||||||||||
Deferred Tax Liabilities | ||||||||||||
Unrealized gains on investments | $ | — | $ | 14 | $ | 62 | ||||||
Total Deferred Tax Liabilities | $ | — | $ | 14 | $ | 62 | ||||||
Deferred Tax Assets | ||||||||||||
Charitable contributions | $ | 53 | $ | 51 | $ | 15 | ||||||
Accrued expenses | 1,857 | 1,318 | 759 | |||||||||
Intangibles | 783 | 883 | 983 | |||||||||
Stock compensation | 24,335 | 16,812 | 10,943 | |||||||||
Property, plant & equipment | 80 | 68 | 13 | |||||||||
Unrealized loss on investment | 23 | — | — | |||||||||
Net operating losses | 47,864 | 27,933 | 17,635 | |||||||||
Capitalized R&D | 112,848 | 71,128 | 29,364 | |||||||||
R&D credit | 23,799 | 14,205 | 6,141 | |||||||||
Total deferred tax assets before valuation allowance | 211,642 | 132,398 | 65,853 | |||||||||
Valuation allowance | (211,642 | ) | (132,384 | ) | (65,791 | ) | ||||||
Total deferred tax assets | — | 14 | 62 | |||||||||
Net deferred tax assets | $ | 0 | $ | — | $ | — | ||||||
For the years ended December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
Tax benefit at U.S. federal statutory rate | $ | (17,629 | ) | $ | (6,890 | ) | $ | (10,592 | ) | |||
Stock based compensation | (47 | ) | (3,415 | ) | 138 | |||||||
Effect of tax reform, change in federal tax rate | — | — | 9,260 | |||||||||
Other n ondeductiblee xpenses | 14 | — | 1 | |||||||||
State income taxes benefit before valuation allowance, net of federal benefit | (6,613 | ) | (5,460 | ) | (704 | ) | ||||||
Increase in domestic valuation allowance | 26,843 | 17,529 | 2,880 | |||||||||
Research and development credit | (2,636 | ) | (1,604 | ) | (825 | ) | ||||||
Other adjustments | 68 | (160 | ) | (158 | ) | |||||||
Income tax expense (benefit) | $ | — | $ | — | $ | — | ||||||
For the years ended December 31, | ||||||||||||
2021 | 2020 | 2019 | ||||||||||
Tax benefit at U.S. federal statutory rate | $ | (50,788 | ) | $ | (42,471 | ) | $ | (17,629 | ) | |||
Stock based compensation | — | (68 | ) | (47 | ) | |||||||
Other nondeductible expenses | 5 | 1 | 14 | |||||||||
State income taxes benefit before valuation allowance, net of federal benefit | (19,622 | ) | (16,580 | ) | (6,613 | ) | ||||||
Increase in domestic valuation allowance | 79,258 | 66,593 | 26,843 | |||||||||
Research and development credit | (9,002 | ) | (7,472 | ) | (2,636 | ) | ||||||
Other adjustments | 149 | (3 | ) | 68 | ||||||||
Income tax expense (benefit) | $ | 0— | $ | 0 | $ | 0 | ||||||
Three months ended | ||||||||||||||||
March 31, 2019 | June 30, 2019 | September 30, 2019 | December 31, 2019 | |||||||||||||
Revenues: | ||||||||||||||||
Total revenues | $ | — | $ | — | $ | — | $ | — | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 12,373 | 15,594 | 19,447 | 24,910 | ||||||||||||
General and administrative | 5,746 | 7,110 | 4,748 | 5,044 | ||||||||||||
Total operating expenses | 18,119 | 22,704 | 24,195 | 29,954 | ||||||||||||
Loss from operations | (18,119 | ) | (22,704 | ) | (24,195 | ) | (29,954 | ) | ||||||||
Interest income | 3,039 | 3,005 | 2,766 | 2,214 | ||||||||||||
Other income | — | — | — | — | ||||||||||||
Net loss | $ | (15,080 | ) | $ | (19,699 | ) | $ | (21,429 | ) | $ | (27,740 | ) | ||||
Net loss per common share: | ||||||||||||||||
Basic and diluted net loss per common share | $ | (0.98 | ) | $ | (1.28 | ) | $ | (1.39 | ) | $ | (1.80 | ) | ||||
Basic and diluted weighted average number of common shares outstanding | 15,364,465 | 15,368,986 | 15,415,096 | 15,429,154 |
Three months ended | ||||||||||||||||
March 31, 2021 | June 30, 2021 | September 30, 2021 | December 31, 2021 | |||||||||||||
Revenues: | ||||||||||||||||
Total revenues | $ | — | $ | — | $ | — | $ | — | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 45,770 | 51,632 | 54,873 | 52,889 | ||||||||||||
General and administrative | 7,209 | 10,110 | 8,287 | 11,712 | ||||||||||||
Total operating expenses | 52,979 | 61,742 | 63,160 | 64,601 | ||||||||||||
Loss from operations | (52,979 | ) | (61,742 | ) | (63,160 | ) | (64,601 | ) | ||||||||
Interest income | 160 | 91 | 60 | 52 | ||||||||||||
Other income | 273 | 0 | 0 | 0 | ||||||||||||
Net loss | $ | (52,546) | $ | (61,651) | $ | (63,100) | $ | (64,549) | ||||||||
Net loss per common share: | ||||||||||||||||
Basic and diluted net loss per common share | $ | (3.32) | $ | (3.72) | $ | (3.79) | $ | (3.78) | ||||||||
Basic and diluted weighted average number of common shares outstanding | 15,840,401 | 16,571,322 | 16,639,776 | 17,074,543 |
Three months ended | ||||||||||||||||
March 31, 2020 | June 30, 2020 | September 30, 2020 | December 31, 2020 | |||||||||||||
Revenues: | ||||||||||||||||
Total revenues | $ | — | $ | — | $ | — | $ | — | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 33,400 | 44,688 | 53,292 | 53,429 | ||||||||||||
General and administrative | 4,605 | 5,639 | 5,494 | 6,126 | ||||||||||||
Total operating expenses | 38,005 | 50,327 | 58,786 | 59,555 | ||||||||||||
Loss from operations | (38,005 | ) | (50,327 | ) | (58,786 | ) | (59,555 | ) | ||||||||
Interest income | 1,870 | 1,204 | 823 | 432 | ||||||||||||
Other income | 0 | 100 | 0 | 0 | ||||||||||||
Net loss | $ | (36,135) | $ | (49,023) | $ | (57,963) | $ | (59,123) | ||||||||
Net loss per common share: | ||||||||||||||||
Basic and diluted net loss per common share | $ | (2.34) | $ | (3.18) | $ | (3.75) | $ | (3.82) | ||||||||
Basic and diluted weighted average number of common shares outstanding | 15,429,154 | 15,433,348 | 15,448,425 | 15,475,291 |
Three months ended | ||||||||||||||||
March 31, 2018 | June 30, 2018 | September 30, 2018 | December 31, 2018 | |||||||||||||
Revenues: | ||||||||||||||||
Total revenues | $ | — | $ | — | $ | — | $ | — | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 5,198 | 5,109 | 6,211 | 8,871 | ||||||||||||
General and administrative | 1,871 | 2,717 | 5,122 | 5,583 | ||||||||||||
Total operating expenses | 7,069 | 7,826 | 11,333 | 14,454 | ||||||||||||
Loss from operations | (7,069 | ) | (7,826 | ) | (11,333 | ) | (14,454 | ) | ||||||||
Interest income | 705 | 1,166 | 2,821 | 2,979 | ||||||||||||
Other income | — | 200 | — | — | ||||||||||||
Net loss | $ | (6,364 | ) | $ | (6,460 | ) | $ | (8,512 | ) | $ | (11,475 | ) | ||||
Net loss per common share: | ||||||||||||||||
Basic and diluted net loss per common share | $ | (0.45 | ) | $ | (0.45 | ) | $ | (0.56 | ) | $ | (0.75 | ) | ||||
Basic and diluted weighted average number of common shares outstanding | 14,127,868 | 14,383,720 | 15,307,872 | 15,348,358 |