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 |
(State or other jurisdiction of incorporation or organization) |
(I.R.S. Employer Identification No.) | |
(Address of Principal Executive Offices) |
(Zip Code) |
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered | ||
☒ | Accelerated filer | ☐ | ||||
Non-accelerated filer |
☐ | Smaller reporting company | ||||
Emerging growth company |
Auditor Firm Id: |
Auditor Name: |
Auditor Location: |
Page |
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Item 1. |
6 | |||||
Item 1A. |
29 | |||||
Item 1B. |
57 | |||||
Item 2. |
57 | |||||
Item 3. |
57 | |||||
Item 4. |
57 | |||||
Item 5. |
58 | |||||
Item 6. |
60 | |||||
Item 7. |
61 | |||||
Item 7A. |
69 | |||||
Item 8. |
69 | |||||
Item 9. |
69 | |||||
Item 9A. |
69 | |||||
Item 9B. |
70 | |||||
Item 10. |
71 | |||||
Item 11. |
71 | |||||
Item 12. |
71 | |||||
Item 13. |
71 | |||||
Item 14. |
71 | |||||
Item 15. |
72 | |||||
Item 16. |
74 | |||||
75 |
• | Anticipated or estimated future results, including the risks and uncertainties associated with our future operating performance and financial position, |
• | Our possible or assumed future results of operations and expenses, business strategies and plans (including ex-U.S. launch/partnering plans), capital needs and financing plans, market trends, competitive position, industry environment and potential growth opportunities, |
• | Our ability to delay certain research activities and related clinical expenses as necessary, |
• | Our clinical trials, including the anticipated timing of disclosure, presentations of data from, or outcomes from our trials, |
• | Research and development activities, and the timing and results associated with the future development of our lead product candidate, resmetirom (formerly known as MGL-3196), including projected market size and sector leadership, |
• | The timing and completion of projected 2022 clinical milestone events, including enrollment, additional studies, top-line data and open label projections, |
• | Plans, objectives and timing for making a Subpart H (Accelerated Approval of New Drugs for Serious or Life-Threatening Illnesses) submission to FDA, |
• | Our primary and secondary study endpoints for resmetirom, and the potential for achieving such endpoints and projections, including non-alcoholic steatohepatitis (“NASH”) resolution, safety, fibrosis treatment, cardiovascular effects and lipid treatment with resmetirom, |
• | Optimal dosing levels for resmetirom and projections regarding potential NASH or nonalcoholic fatty liver disease (“NAFLD”) and potential patient benefits with resmetirom, including future NASH resolution, safety, fibrosis treatment, cardiovascular effects, lipid treatment and/or biomarker effects with resmetirom, |
• | The potential efficacy and safety of resmetirom for non-cirrhotic NASH patients and cirrhotic NASH patients, |
• | Ex-U.S. launch/partnering plans, |
• | The predictive power of resmetirom liver fat reduction, as measured by non-invasive tests, on NASH resolution and/or fibrosis reduction or improvement, and potential NASH or NAFLD patient risk profile benefits with resmetirom, |
• | The predictive power of liver fat, volume or fibrosis reduction with resmetirom using non-invasive tests, |
• | The predictive power of non-invasive tests generally, including for purposes of diagnosing NASH, monitoring patient response to resmetirom, or recruiting and conducting a NASH clinical trial, |
• | Market demand for and acceptance of our products, |
• | Research, development and commercialization of new products, |
• | Obtaining and maintaining regulatory approvals, including, but not limited to, potential regulatory delays or rejections, |
• | Risks associated with meeting the objectives of our clinical studies, including, but not limited to our ability to achieve enrollment objectives concerning patient numbers (including an adequate safety database), outcomes objectives and/or timing objectives for our studies, any delays or failures in enrollment, the occurrence of adverse safety events, and the risks of successfully conducting trials that are substantially larger, and have patients with different disease states, than our past trials, |
• | Risks related to the effects of resmetirom’s mechanism of action and our ability to accomplish our business and business development objectives and realize the anticipated benefit of any such transactions, and |
• | Assumptions underlying any of the foregoing. |
• | We have limited operating history, we have incurred significant operating losses since inception and we expect to incur significant operating losses for the foreseeable future. We may never become profitable or, if achieved, be able to sustain profitability. |
• | Our business depends on the success of resmetirom, which is still in clinical development and has not completed a pivotal trial. If we are unable to obtain regulatory approval for and successfully commercialize resmetirom, or we experience significant delays in doing so, our business will be materially harmed. |
• | Clinical trials are very expensive, time-consuming and difficult to design and implement and involve uncertain outcomes. The results of preclinical studies and early clinical trials are not always predictive of future results. Any product candidate that we advance into clinical trials may not have favorable results in later clinical trials or receive regulatory approval. |
• | Because resmetirom has not yet received regulatory approval for any indication, it is difficult to predict the time and cost of development and our ability to successfully complete clinical development and obtain the necessary regulatory approvals for commercialization and our ability to realize a benefit, if any, from Fast Track designation or any Subpart H application may be limited or unavailable. |
• | If clinical trials or regulatory approval processes for our product candidates are prolonged, delayed or suspended, we may be unable to commercialize our product candidates on a timely basis, which would require us to incur additional costs and delay our receipt of any revenue from potential product sales. |
• | If we inadvertently fail to comply with foreign regulatory requirements governing human clinical trials and marketing approval for drugs, we could be prevented from selling our drug candidates in foreign markets. |
• | We depend on enrollment of patients in our clinical trials for our product candidates. If we encounter difficulties enrolling patients in our clinical trials, COVID-19 , |
• | Any product candidate in our current or future clinical trials may cause unacceptable adverse events or side effects or have other properties that may delay or prevent its regulatory approval or commercialization or limit its commercial potential. |
• | Our product candidates will remain subject to ongoing regulatory review even if they receive marketing approval, and if we fail to comply with continuing regulations, we could lose these approvals and the sale of any approved commercial products could be suspended. |
• | We operate in a highly competitive and rapidly changing industry, and our product candidates may become obsolete. |
• | If the FDA or other applicable regulatory authorities approve generic products that compete with any of our or any of our partners’ product candidates, the sales of our product candidates would be adversely affected. |
• | If physicians and patients do not accept our future products or if the market for indications for which any product candidate is approved is smaller than expected, we may be unable to generate significant revenue, if any. |
• | The continuation or worsening of the COVID-19 pandemic could affect our ability to complete our ongoing clinical trials, disrupt regulatory activities and delay or disrupt commercialization of resmetirom, and may have other adverse effects on our stock price and business operations. |
• | As we evolve from a company that is primarily involved in clinical development to a company that is also involved in commercialization, we may encounter difficulties in expanding our operations successfully. |
• | The uncertainty associated with pharmaceutical reimbursement and related matters may adversely affect our business. |
• | If we fail to develop and commercialize other product candidates, we may be unable to grow our business. |
• | Our internal computer systems, or those of our third-party collaborators, service providers, contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our development programs and have a material adverse effect on our reputation, business, financial condition or results of operations. |
• | Our rights to develop and commercialize our product candidates are subject in part to the terms and conditions of a license to resmetirom granted to us by Hoffmann-La Roche. |
• | We may fail to comply with any of our obligations under agreements pursuant to which we license rights or technology, which could result in the loss of rights or technology that are material to our business. |
• | Our success depends on our ability to protect our intellectual property and our proprietary technologies. |
• | Claims by third parties that we infringe their proprietary rights may result in liability for damages or prevent or delay our developmental and commercialization efforts. |
• | We may be involved in lawsuits to protect or enforce our patents or the patents of our licensors, which could be expensive, time consuming, and unsuccessful. Further, our issued patents could be found invalid or unenforceable if challenged in court. |
• | We may not be successful in obtaining or maintaining necessary rights to our product candidates through acquisitions and in-licenses. |
• | We may not be able to protect our intellectual property rights throughout the world. |
• | If we fail to obtain the capital necessary to fund all of our planned operations, we may be unable to successfully develop and commercialize resmetirom and other future product candidates. |
• | Our ability to use net operating loss and tax credit carryforwards and certain built-in losses to reduce future tax payments may be limited by provisions of the Internal Revenue Code. |
• | The price of our common stock has been, and may continue to be, volatile. |
• | A small number of our stockholders own a substantial amount of our outstanding common stock and may be deemed to have substantial control over us; therefore, your ability to influence corporate matters may be limited. |
• | Anti-takeover provisions in our charter documents and under Delaware law could make an acquisition of our company, which may be beneficial to our stockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management. |
• | Future sales and issuances of our common stock or rights to purchase common stock could result in additional dilution of the percentage ownership of our stockholders and could cause our share price to fall. |
• | Sales of a significant number of shares of our common stock in the public markets or significant short sales of our common stock, or the perception that such sales could occur, could depress the market price of our common stock and impair our ability to raise capital. |
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) |
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 12 MRI-PDFF |
(2) |
does not include one end-of-study |
• | Sustained, highly statistically significant (p<0.0001) reduction in liver fat compared with placebo on 36-week MRI-PDFF and mean relative fat reduction of 37% with resmetirom treated patients in contrast with 8.5% with placebo patients; |
• | Sustained, statistically significant reductions (p<0.0001) in resmetirom compared to placebo treated patients in low-density lipoprotein cholesterol (LDL-C) and apolipoprotein B (ApoB) of more than 20%, triglycerides (TG) of 36% and lipoprotein(a) of 37%; |
• | Statistically significant reductions in liver enzymes (ALT, AST and GGT) relative to placebo (all p=0.002) and a 40% reduction in ALT in patients with elevated baseline levels (p=0.01); |
• | Statistically significant reductions in fibrosis biomarkers in resmetirom treated patients as compared with placebo patients; |
• | On liver biopsy, fibrosis was reduced by at least one point in 23% of placebo patients and 29% of resmetirom treated patients; |
• | Very good all subject tolerability: mostly mild and a few moderate adverse events, or AEs, which were balanced between drug treated patients and placebo patients; and |
• | An increase in incidence of a transient mild diarrhea at beginning of study, often a single episode, in resmetirom treated patients compared with placebo patients. |
• | Percentages of patients with NASH resolution at Week 36 increased with greater PDFF reduction (agreement between two independent pathologist central biopsy readers) |
• | In resmetirom-treated patients with ≥ 50% fat reduction at Week 12, 64% had NASH resolution with a component response driven primarily by ballooning and inflammation |
• | PDFF reduction ≥ 30 and ≥ 50% at Week 12 was associated with improvement in all NAS components including fibrosis reduction on subsequent liver biopsy and achievement of both endpoints: NASH resolution and ≥ 1 point fibrosis reduction PDFF reduction ≥ 30 and ≥ 50% at Week 12 was also associated with fibrosis reduction on subsequent liver biopsy |
• | Complete clinical development and seek regulatory approval of resmetirom in NASH. first-in-class |
• | Establish commercial capabilities to market resmetirom as a leading treatment for NASH. |
• | Grow our pipeline through additional indications for resmetirom potentially including orphan indications. |
• | Our potential competitors may have substantially greater financial, technical, and personnel resources than us. In addition, many of these competitors have significantly greater commercial infrastructures. Our ability to compete successfully will depend largely on our ability to leverage our collective experience in drug discovery, development and commercialization to: |
• | discover and develop medicines that are differentiated from other products in the market, |
• | obtain patent and/or proprietary protection for our products and technologies; |
• | obtain required regulatory approvals; |
• | obtain a commercial partner; |
• | commercialize our drugs, if approved; and |
• | attract and retain high-quality research, development and commercial personnel. |
• | completion of preclinical laboratory tests, animal studies and formulation studies, some in accordance with the FDA’s current Good Laboratory Practices, or GLP, the Animal Welfare Act administered and enforced by the United States Department of Agriculture, and other applicable regulations; |
• | submission to the FDA of an IND which must become effective before human clinical trials may begin; |
• | approval by an institutional review board, or IRB, before each trial may be initiated at each clinical site; |
• | performance of adequate and well-controlled human clinical trials under protocols submitted to the FDA and reviewed and approved by each IRB, conducted in accordance with federal regulations and according to Good Clinical Practices, or GCP, to establish the safety and efficacy of the proposed drug for its intended use; |
• | submission to the FDA of an NDA; |
• | completion of registration batches and validation of the manufacturing process to show ability to consistently produce quality batches of product; |
• | satisfactory completion of an FDA Advisory Committee review, if applicable; |
• | satisfactory completion of an FDA inspection of the manufacturing facility or facilities at which the drug is produced to assess compliance with current good manufacturing practice, or cGMP, to assure that the facilities, methods and controls are adequate to preserve the drug’s identity, strength, quality and purity; and |
• | FDA review and approval of the NDA. |
• | Phase 1: |
• | Phase 2: |
• | Phase 3: |
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 |
• | we may not have sufficient financial and other resources to complete the necessary clinical trials for resmetirom, including, but not limited to, our planned registrational clinical trials to obtain drug approval; |
• | the mechanism of action of resmetirom is complex and we do not know the degree to which it will translate into a therapeutic benefit, if any, in NASH or any other indication, and we do not know the degree to which the complex mechanism of action may contribute to long term safety issues or adverse events, if any, when resmetirom is taken for prolonged periods such as in the treatment of NASH or any other indication; |
• | delay or inability to reach agreement with the FDA or comparable foreign regulatory authorities on acceptable clinical trial design; |
• | regulators, IRBs or ethics committees may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial at a prospective trial site; |
• | we may not be able to obtain adequate evidence from clinical trials of efficacy and safety for resmetirom in NASH or any other indication; |
• | we do not know the degree to which resmetirom will be accepted as a therapy by physicians, patients and payors, even if approved; |
• | in our clinical programs for resmetirom, we may experience variability in patients, adjustments to clinical trial procedures and the need for additional clinical trial sites, which could delay our clinical trial progress; |
• | the results of our clinical trials may not meet the level of statistical or clinical significance required by the FDA or comparable foreign regulatory bodies for marketing approval; |
• | patients in our clinical trials may die or suffer other adverse effects for reasons that may or may not be related to resmetirom, which could delay or prevent further clinical development; |
• | the standards implemented by clinical or regulatory agencies may change at any time; |
• | we cannot be certain what efficacy endpoints clinical or regulatory agencies may require in a Phase 3 clinical trial of NASH or for approval of our product candidates; we also cannot be certain if we will be able to gain Subpart H approval of any of our product candidates based on surrogate endpoints; |
• | foreign clinical or regulatory agencies may require efficacy and safety endpoints for Phase 3 clinical trials that may not be favorable to us and different from the results we have observed to date in our current trials; |
• | other differences in the design of our ongoing and planned Phase 3 clinical trials of the treatment of NASH, including the use of a new tablet formulation of resmetirom and/or the inclusion of patients with more advanced NASH, could cause the results of our Phase 3 trials to be less favorable than the results we observed in our Phase 2 trials in NASH; |
• | if we obtain Subpart H approval of resmetirom based on a surrogate endpoint, consistent with our ongoing Phase 3 trial, we will be required to conduct a post-approval clinical outcomes trial to confirm the clinical benefit of the product candidate and if the post-approval trial is not successful we may not be able to continue marketing the product; |
• | we cannot be certain of the number and type of clinical trials and non-clinical studies that FDA or other regulatory agencies will require in order to approve resmetirom for NASH; |
• | if approved for NASH, resmetirom will likely compete with the off-label use of currently marketed products and other therapies in development that may reach approval for NASH prior to resmetirom; and |
• | we may not be able to obtain, maintain or enforce our patents and other intellectual property rights. |
• | conditions imposed on us by the FDA or other regulatory authorities regarding the scope or design of our clinical trials; |
• | insufficient supply of our product candidates or other materials necessary to conduct and complete our clinical trials; |
• | slow enrollment and retention rate of subjects in our clinical trials; |
• | challenges in identifying or recruiting sufficient study sites or investigators for clinical trials; and |
• | serious and unexpected drug-related side effects related to the product candidate being tested. |
• | restrictions on marketing or manufacturing of our products, withdrawal of the product from the market; |
• | holds on clinical trials; |
• | warning letters or untitled letters; |
• | civil or criminal penalties; |
• | fines; |
• | injunctions; |
• | product seizures or detentions; |
• | pressure to initiate voluntary product recalls; |
• | suspension or withdrawal of regulatory approvals; and |
• | refusal to approve pending applications for marketing approval of new products or supplements to approved applications. |
• | timing of market introduction of competitive products; |
• | demonstration of clinical safety and efficacy compared to other products; |
• | cost-effectiveness; |
• | limited or no coverage by third-party payers; |
• | convenience and ease of administration; |
• | prevalence and severity of adverse side effects; |
• | restrictions in the label of the drug; |
• | other potential advantages of alternative treatment methods; and |
• | ineffective marketing and distribution support of its products. |
• | decreased demand for any of our future approved products; |
• | injury to our reputation; |
• | withdrawal of clinical trial participants; |
• | termination of clinical trial sites or entire trial programs; |
• | significant litigation costs; |
• | substantial monetary awards to or costly settlements with patients or other claimants; |
• | product recalls or a change in the indications for which products may be used; |
• | loss of revenue; |
• | diversion of management and scientific resources from our business operations; and |
• | the inability to commercialize our product candidates. |
• | the scope of rights granted under the license agreement and other interpretation-related issues; |
• | the extent to which our technology and processes infringe on intellectual property of the licensor that is not subject to the licensing agreement; |
• | the sublicensing of patent and other rights; |
• | our diligence obligations under the license agreement and what activities satisfy those diligence obligations; |
• | the ownership of inventions and know-how resulting from the joint creation or use of intellectual property by us and our licensors and collaborators; and |
• | the priority of invention of patented technology. |
• | the USPTO and various foreign governmental patent agencies require compliance with a number of procedural, documentary, fee payment and other provisions during the patent process and after a patent has issued. There are situations in which noncompliance can result in abandonment or lapse of a patent or patent application, resulting in partial or complete loss of patent rights in the relevant jurisdiction. In such an event, competitors might be able to enter the market earlier than would otherwise have been the case; |
• | patent applications may not result in any patents being issued; |
• | patents may be challenged, invalidated, modified, revoked, circumvented, found to be unenforceable or otherwise may not provide any competitive advantage; |
• | we and our licensor(s) may not have been the first to make the inventions covered by pending patent applications or issued patents; |
• | we and our licensor(s) may not have been the first to file patent applications for our product candidates or the compositions developed, or for their uses; |
• | others may independently develop identical, similar or alternative products or compositions and uses thereof; |
• | we and our licensor(s)’ disclosures in patent applications may not be sufficient to meet the statutory requirements for patentability; |
• | others may design around our owned and licensed patent claims to produce competitive products which fall outside of the scope of the patents; |
• | others may identify prior art or other bases which could invalidate our or our licensor(s)’ patents; |
• | our competitors might conduct research and development activities in the United States and other countries that provide a safe harbor from patent infringement claims for certain research and development activities, as well as in countries where us and our licensor(s) do not have patent rights, and then use the information learned from such activities to develop competitive products for sale in major commercial markets; |
• | there may be significant pressure on the United States government and international governmental bodies to limit the scope of patent protection both inside and outside the United States for disease treatments that prove successful, as a matter of public policy regarding worldwide health concerns; and |
• | countries other than the United States may have patent laws less favorable to patentees than those upheld by United States courts, allowing foreign competitors a better opportunity to create, develop and market competing product candidates. |
• | result in costly litigation; |
• | divert the time and attention of our technical personnel and management; |
• | cause development delays; |
• | prevent us from commercializing resmetirom for NASH or our other product candidates until the asserted patent expires or is held finally invalid or not infringed in a court of law; |
• | require us to develop non-infringing technology, which may not be possible on a cost-effective basis; or |
• | require us to enter into royalty or licensing agreements. |
• | the type, number, scope, progress, expansion costs, results of and timing of our future clinical trials and projected product label or the need for additional clinical trials of resmetirom for NASH and dyslipidemia or any of our other product candidates which we are pursuing or may choose to pursue in the future; |
• | the costs of obtaining, maintaining and enforcing our patents and other intellectual property rights; |
• | the costs and timing of obtaining regulatory approval for resmetirom for NASH and any of our other potential product candidates; |
• | the costs and timing of obtaining or maintaining manufacturing for resmetirom for NASH and any of our other product candidates, including commercial manufacturing if any product candidate is approved; |
• | the costs and timing of establishing sales, marketing and reimbursement capabilities and enhanced internal controls over financial reporting; |
• | the terms and timing of establishing and maintaining collaborations, license agreements and other partnerships, including for ex-US resmetirom opportunities; |
• | costs associated with any new product candidates that we may develop, in-license or acquire; and |
• | the effect of competing technological and market developments. |
• | the losses we may incur, including increased losses resulting from costs associated with increases in our clinical trial activity; |
• | developments in patent or other proprietary rights owned or licensed by us, our collaborative partners or our competitors; |
• | the progress and results of our clinical trials; |
• | public or regulatory concern as to the safety and efficacy of NASH products developed by us or others or public safety generally; and |
• | litigation. |
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, |
||||||||||||||||||||
2021 |
2020 |
2019 |
2018 |
2017 |
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(in thousands, except per share amounts) |
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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 | |||||||||||||||
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|
|
|
|
|
|
|
|
|
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Total operating expenses |
242,482 | 206,673 | 94,972 | 40,682 | 32,062 | |||||||||||||||
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|
|
|
|
|
|
|
|
|
|||||||||||
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 | ) | |||||
|
|
|
|
|
|
|
|
|
|
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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 |
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(in thousands) |
||||||||||||||||||||
Consolidated Balance Sheet Data: |
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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. |
• | salaries and related expense, including stock-based compensation; |
• | external expenses paid to clinical trial sites, contract research organizations, laboratories, database software and consultants that conduct clinical trials; |
• | expenses related to development and the production of nonclinical and clinical trial supplies, including fees paid to contract manufacturers; |
• | expenses related to preclinical studies; |
• | expenses related to compliance with drug development regulatory requirements; and |
• | other allocated expenses, which include direct and allocated expenses for depreciation of equipment and other supplies. |
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, |
||||||||||||
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 |
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 on Form 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 |
Incorporated by Reference herein from Form or Schedule |
Filing Date |
SEC File / Registration Number | |||||
31.1 | Certification of Principal Executive Officer required by Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002. | X | ||||||||
31.2 | Certification of Principal Financial Officer pursuant to Rule 13a-14(a) or Rule 15d-14(a) of the Securities Exchange Act of 1934, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002. | X | ||||||||
32.1** | Certifications of Principal Executive Officer and Principal Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002. | 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 Form 10-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. | ||||||
Chief Executive Officer | ||||||
(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 24, 2022 | ||
/s/ ALEX G. HOWARTH Alex G. Howarth |
Chief Financial Officer (Principal Accounting and Financial Officer) |
February 24, 2022 | ||
/s/ REBECCA TAUB, M.D. Rebecca Taub, M.D. |
Director |
February 24, 2022 | ||
/s/ FRED B. CRAVES, PH.D. Fred B. Craves, Ph.D. |
Director |
February 24, 2022 | ||
/s/ KENNETH M. BATE Kenneth M. Bate |
Director |
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