GlycoMimetics(GLYC) - 2019 Q4 - Annual Report

Part I Business GlycoMimetics is a clinical-stage biotechnology company developing glycomimetic drugs for rare diseases Company Overview and Drug Candidates The company develops novel glycomimetic drugs, with lead candidate Uproleselan targeting Acute Myeloid Leukemia (AML) - Uproleselan, a specific E-selectin inhibitor for treating Acute Myeloid Leukemia (AML), has been granted Breakthrough Therapy designation by the FDA and orphan drug designation in both the U.S. and Europe[17] - GMI-1359, a dual inhibitor of E-selectin and CXCR4, is in a Phase 1b trial for HR+ breast cancer patients with bone metastases[23] - Pfizer terminated its license agreement for rivipansel in February 2020 after the Phase 3 trial failed to meet its endpoints[28] Strategy and Platform The company's strategy focuses on completing the Uproleselan Phase 3 trial and leveraging its glycomimetic platform - The company's primary strategic goal is to complete the clinical development of uproleselan for relapsed/refractory AML, with enrollment in the pivotal Phase 3 trial expected to be completed in the second half of 2021[31] - A collaboration with the National Cancer Institute (NCI) is underway to evaluate uproleselan in older adults with previously untreated AML[31] - The company entered into an exclusive license agreement with Apollomics in January 2020 to develop and commercialize uproleselan and GMI-1687 in Greater China[31] Clinical Development Pipeline The clinical pipeline is led by Uproleselan in Phase 3 for AML, with other candidates in earlier stages - The pivotal Phase 3 trial for uproleselan in relapsed/refractory AML is designed to enroll approximately 380 adult patients, with overall survival as the primary endpoint[59][60] - A Phase 1b trial for GMI-1359 in HR+ breast cancer patients with bone metastases was initiated in Q4 2019, with the first patient dosed in January 2020[68] Uproleselan Phase 1/2 Trial Key Results | Cohort | Key Metric | Result | | :--- | :--- | :--- | | Relapsed/Refractory AML | CR/CRi Rate | 41% | | Relapsed/Refractory AML | Median Overall Survival | 8.8 months | | Newly Diagnosed AML | CR/CRi Rate | 72% | | Newly Diagnosed AML | Median Overall Survival | 12.6 months | Collaborations, IP, and Commercialization The company manages its pipeline through strategic collaborations, protects its IP, and relies on third-party manufacturing - The collaboration with Apollomics for uproleselan and GMI-1687 in Greater China includes a $9.0 million upfront payment, up to $180.0 million in potential milestones, and tiered royalties[72] - The company has issued patents for uproleselan expected to expire between 2032 and 2033, and for GMI-1359 expected to expire in 2036[74] - The company does not have manufacturing facilities and relies on third parties for preclinical, clinical, and potential commercial manufacturing[82] Competition and Government Regulation The company faces competition from established AML therapies and is subject to extensive government regulation - The company faces competition from several recently FDA-approved therapies for AML, including those from Novartis (RYDAPT®), Celgene (IDHIFA®), and Jazz Pharmaceuticals (VYXEOS)[91] - The drug development process is heavily regulated by the FDA, requiring preclinical studies, an effective IND, and adequate and well-controlled clinical trials[97][98] - Uproleselan has received Fast Track, Breakthrough Therapy, and Orphan Drug designations, which may expedite review and provide market exclusivity[114][119][159] Risk Factors The company faces significant financial, clinical development, and operational risks, including a history of losses - The company has a history of significant operating losses, with an accumulated deficit of $258.4 million as of December 31, 2019[171] - The company will need substantial additional funding and believes its current cash will only fund operations into 2022[178] - Clinical drug development is a lengthy, expensive, and uncertain process, with potential delays in patient enrollment for its trials[193][196] - The business relies heavily on third parties for manufacturing, conducting clinical trials, and commercialization in certain territories[201][207][211] Unresolved Staff Comments The company reports no unresolved staff comments from the SEC - None[320] Properties The company leases approximately 42,000 square feet of office space in Rockville, Maryland - The company leases approximately 42,000 square feet of office space in Rockville, Maryland, under a lease agreement that expires in October 2023[321] Legal Proceedings The company is not currently a party to any material legal proceedings - The company is not currently a party to any material legal proceedings[322] Mine Safety Disclosures This item is not applicable to the company's business - Not applicable[323] Part II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities The company's common stock trades on Nasdaq under "GLYC" and it does not anticipate paying dividends - Common stock is listed on The Nasdaq Global Market under the symbol "GLYC"[325] - The company has never declared or paid dividends and does not plan to in the foreseeable future[325] Selected Financial Data This section summarizes key financial data from 2015 to 2019, showing consistent net losses and rising R&D expenses Selected Financial Data (2015-2019) | (in thousands) | 2019 | 2018 | 2017 | 2016 | 2015 | | :--- | :--- | :--- | :--- | :--- | :--- | | Revenue | $ — | $ — | $ — | $ 18 | $ 20,071 | | Research and development expense | 47,029 | 40,092 | 24,100 | 23,282 | 25,050 | | General and administrative expense | 14,360 | 11,413 | 9,832 | 8,650 | 7,805 | | Net loss | $(57,892) | $(48,274) | $(33,281) | $(31,810) | $(12,769) | | Cash and cash equivalents (End of Period) | $158,201 | $209,918 | $123,925 | $40,042 | $46,803 | | Total assets (End of Period) | $167,970 | $214,839 | $128,583 | $42,388 | $48,462 | | Total stockholders' equity (End of Period) | $154,201 | $205,464 | $119,701 | $35,301 | $40,472 | Management's Discussion and Analysis of Financial Condition and Results of Operations The company's 2019 net loss increased to $57.9 million due to higher R&D and G&A expenses Results of Operations (2019 vs. 2018) The net loss increased by $9.6 million in 2019, driven by higher clinical trial and personnel costs Results of Operations (in thousands) | | 2019 | 2018 | Change | | :--- | :--- | :--- | :--- | | Revenue | $ — | $ — | $ — | | Research and development expense | 47,029 | 40,092 | 6,937 | | General and administrative expense | 14,360 | 11,413 | 2,947 | | Loss from operations | (61,389) | (51,505) | (9,884) | | Interest income | 3,497 | 3,231 | 266 | | Net loss | $(57,892) | $(48,274) | $(9,618) | - Research and development expense increased by $6.9 million (17%) in 2019, primarily due to increased clinical costs for the ongoing global Phase 3 trial of uproleselan[383] - General and administrative expense increased by $2.9 million (26%) in 2019, mainly due to higher personnel-related costs and legal expenses[384] Liquidity and Capital Resources The company had $158.2 million in cash as of year-end 2019, sufficient to fund operations into 2022 - As of December 31, 2019, the company had $158.2 million in cash and cash equivalents[386] - The company expects its existing cash and cash equivalents to fund operating expenses and capital expenditure requirements into 2022[400] Summary of Cash Flows (in thousands) | | 2019 | 2018 | | :--- | :--- | :--- | | Net cash used in operating activities | $(51,984) | $(43,331) | | Net cash used in investing activities | (145) | (126) | | Net cash provided by financing activities | 413 | 129,450 | Quantitative and Qualitative Disclosures About Market Risk The company's primary market risk is interest rate sensitivity on its cash equivalents, which is considered immaterial - The company's primary market risk is interest rate sensitivity on its $158.2 million of cash and cash equivalents as of December 31, 2019[410] - Due to the short-term maturities and low-risk profile of its investments, the company does not believe interest rate changes would have a material effect on its financial position[410] Financial Statements and Supplementary Data This section references the location of the company's financial statements within the Form 10-K - The financial statements required for this item are listed in Part IV, Item 15 of the report[411] Changes in and Disagreements with Accountants on Accounting and Financial Disclosure The company reports no changes in or disagreements with its accountants - None[412] Controls and Procedures Management concluded that the company's disclosure controls and internal controls were effective as of year-end 2019 - As of December 31, 2019, the company's CEO and CFO concluded that disclosure controls and procedures were effective at a reasonable assurance level[414] - Management concluded that the company's internal control over financial reporting was effective as of December 31, 2019, a conclusion audited and affirmed by Ernst & Young, LLP[415][416] Other Information The company reports no other information for this item - None[418] Part III Directors, Executive Officers and Corporate Governance Information is incorporated by reference from the company's 2020 proxy statement - Information is incorporated by reference from the 2020 Proxy Statement[421] Executive Compensation Information is incorporated by reference from the company's 2020 proxy statement - Information is incorporated by reference from the 2020 Proxy Statement[422] Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters Information is incorporated by reference from the company's 2020 proxy statement - Information is incorporated by reference from the 2020 Proxy Statement[422] Certain Relationships and Related Transactions, and Director Independence Information is incorporated by reference from the company's 2020 proxy statement - Information is incorporated by reference from the 2020 Proxy Statement[423] Principal Accounting Fees and Services Information is incorporated by reference from the company's 2020 proxy statement - Information is incorporated by reference from the 2020 Proxy Statement[423] Part IV Exhibits, Financial Statement Schedules This section lists the financial statements and exhibits filed as part of the Form 10-K - This item lists the financial statements and exhibits filed with the Form 10-K[425] Form 10-K Summary This item is not applicable - Not applicable[430]