Y-mAbs(YMAB) - 2020 Q4 - Annual Report

Part I Business Y-mAbs is a commercial-stage biopharmaceutical company focused on antibody-based cancer therapeutics, with its first FDA-approved product DANYELZA and a pipeline including omburtamab, bispecific antibodies, and radioimmunotherapy platforms - On November 25, 2020, DANYELZA® (naxitamab-gqgk) was approved by the FDA for the treatment of pediatric and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow[30] - The company received a Refusal to File letter from the FDA in October 2020 for its omburtamab BLA, citing the need for more detail in the CMC and Clinical modules, with a resubmission planned for the second or third quarter of 2021[34][45] - The company is advancing novel platforms, including Y-BiClone for T-cell engaging bispecific antibodies and the SADA (Liquid Radiation™) technology for a two-step radioimmunotherapy approach[36][37][55] - Y-mAbs has exclusive rights to its current product candidates through license agreements with Memorial Sloan Kettering Cancer Center (MSK) and the Massachusetts Institute of Technology (MIT)[57][54] Our Pipeline The company's pipeline features the FDA-approved DANYELZA, lead candidate omburtamab with a planned BLA resubmission, and early-stage assets including bispecific antibodies and SADA technology candidates Key Pipeline Status | Product Candidate | Target | Key Indication | Status/Next Milestone | | :--- | :--- | :--- | :--- | | DANYELZA | GD2 | Relapsed/Refractory High-Risk Neuroblastoma | Approved in November 2020 | | Omburtamab | B7-H3 | CNS/Leptomeningeal Metastases from Neuroblastoma | Q2/Q3 2021 BLA resubmission | | Nivatrotamab | GD2xCD3 | Small Cell Lung Cancer | Ongoing Phase 2 trial | | huCD33-BsAb | CD33xCD3 | AML (Pediatric) | IND planned for 2021 | | GD2-SADA | GD2 | GD2 Positive Solid Tumors | IND planned for 2021 | DANYELZA (naxitamab-gqgk) DANYELZA is an FDA-approved humanized monoclonal antibody for relapsed/refractory high-risk neuroblastoma, offering a shorter infusion time and being explored for additional indications - DANYELZA is administered in approximately 30 to 60 minutes in an outpatient setting, a significant advantage over other GD2-targeting therapies that require 10 to 20 hours of infusion and hospitalization[43][94] - The FDA approval was based on interim data from Study 201 and Study 12-230 for high-risk relapsed/refractory neuroblastoma, with Study 201 ongoing to satisfy post-marketing requirements[101][110] - The product has a boxed warning for serious infusion reactions and neurotoxicity, with common adverse events including infusion-related reactions, pain, tachycardia, and vomiting[90][459] Omburtamab Omburtamab is a murine monoclonal antibody targeting B7-H3 for pediatric CNS/leptomeningeal metastases from neuroblastoma, with a BLA resubmission planned following promising clinical data - Omburtamab targets B7-H3, an immune checkpoint molecule widely expressed in various cancer types, and has received Breakthrough Therapy Designation for pediatric CNS/LM from neuroblastoma[157] - In an analysis of 107 patients with pediatric CNS/LM from neuroblastoma (Study 03-133), treatment with 131I-omburtamab demonstrated a median overall survival of 50.8 months, compared to a historical median of approximately six to nine months[45][166][185] - The company is also developing 177Lu-omburtamab-DTPA, a Lutetium-177 conjugated version, for medulloblastoma and B7-H3 positive CNS/LM tumors in adults, with Phase 1 trials expected to begin enrollment in Q1 2021[49][229] Bispecific Antibody Programs Y-mAbs is developing T-cell engaging bispecific antibodies using its Y-BiClone platform, with lead candidate nivatrotamab in Phase 2 trials and a CD33-BsAb in preclinical development - The lead bispecific antibody, nivatrotamab, targets both GD2 on tumor cells and CD3 on T-cells, with an IND for a Phase 2 trial in Small Cell Lung Cancer cleared in January 2021[242][260] - The company's BsAbs are designed in an IgG-like format to have a longer serum half-life, potentially avoiding the need for continuous infusion required by smaller BiTE constructs[257][86] - A second BsAb candidate targeting CD33 for hematological cancers is in preclinical development, with plans to file an IND in 2021[244][261] Manufacturing and Commercialization The company utilizes a third-party manufacturing model with key CMOs and has established a focused U.S. commercial team alongside international distribution partnerships for its products - Y-mAbs relies on contract manufacturing organizations (CMOs) for all manufacturing and does not own or operate any facilities[270] - Key manufacturing partners include Patheon/Thermo Fisher for DANYELZA drug substance and product, and EMD/Merck for omburtamab drug substance with radiolabeling performed at SpectronRx[278][279] - The company has established commercial partnerships for DANYELZA and omburtamab in Greater China (SciClone), Israel (Takeda), and Eastern Europe (Swixx BioPharma)[285][287] Intellectual Property The company's intellectual property strategy involves in-licensing key patent portfolios from MSK and MIT, covering DANYELZA, omburtamab, and the SADA technology with various expiration dates - The DANYELZA patent portfolio, licensed from MSK, includes patents covering composition of matter with expected expiration dates in June 2031 and March 2034[295] - The omburtamab patent portfolio, licensed from MSK, includes patents covering composition of matter and methods of use, with expected expiration dates ranging from October 2021 to March 2028[295] - The SADA radioimmunotherapy platform portfolio is exclusively licensed from MSK and MIT, with patent families expected to expire between February 2030 and July 2039[297][299] MSK Agreements Y-mAbs maintains a critical relationship with Memorial Sloan Kettering Cancer Center (MSK) through license agreements for DANYELZA, omburtamab, and SADA technology, involving fees, milestones, and royalties - The 2015 MSK License grants worldwide, sub-licensable rights for key product candidates including DANYELZA and omburtamab, in exchange for upfront payments, stock, milestones, and royalties[308] - Under the MSK License, MSK is entitled to 40% of the income from the sale of the first Priority Review Voucher (PRV) and 33% from subsequent PRVs[312] - The SADA License Agreement with MSK and MIT, executed in April 2020, involves upfront cash and stock payments, and potential future milestones and royalties for the SADA radioimmunotherapy platform[331][332] Risk Factors The company faces substantial risks including a limited operating history, dependence on DANYELZA's commercial success, regulatory uncertainties for omburtamab, reliance on third parties, and identified material weaknesses in internal controls - The company has a limited operating history, has incurred significant losses since inception (net loss of $119.3 million in 2020), and may never achieve profitability[402][403] - Management has identified material weaknesses in internal control over financial reporting, specifically a lack of sufficient trained professionals for accounting, controls over complex matters, and segregation of duties[424][425][426] - The business is highly dependent on the successful commercialization of DANYELZA and obtaining regulatory approval for omburtamab, which faces uncertainty after the FDA's Refusal to File letter[542][543] - The company relies on third-party CMOs for all manufacturing, creating risks related to supply chain disruptions, quality control, and regulatory compliance of facilities[513][514] Properties The company leases its corporate headquarters in New York, NY (4,312 sq. ft.), a combined office and laboratory space in Nutley, NJ (4,783 sq. ft.), and an office space for its Danish subsidiary in Hørsholm, Denmark (15,087 sq. ft.) - Corporate headquarters are leased in New York, NY[724] - A combined office and laboratory space is leased in Nutley, New Jersey[724] - The wholly owned Danish subsidiary leases approximately 15,087 square feet of office space in Hørsholm, Denmark[725] Legal Proceedings The company is not currently subject to any legal proceedings that are expected to have an adverse effect on its business, operating results, or financial condition - As of the filing date, the company is not a party to any material legal proceedings[727] Part II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities Y-mAbs' common stock trades on NASDAQ under "YMAB"; the company has never paid cash dividends and recently completed a secondary public offering to bolster liquidity - The company's common stock trades on the NASDAQ Global Select Market under the symbol "YMAB"[731] - The company has never declared or paid cash dividends and intends to retain future earnings to finance business growth[734] - In February 2021, the company completed a secondary public offering, selling 2,804,878 shares at $41.00 per share for gross proceeds of $115.0 million[742] Selected Financial Data This section presents selected consolidated financial data for the fiscal years ended December 31, 2020, 2019, and 2018, highlighting increased net loss, rising operating expenses, and the recognition of first license revenue in 2020 Consolidated Statement of Operations Data (in thousands) | | 2020 | 2019 | 2018 | | :--- | :--- | :--- | :--- | | License revenue | $20,750 | $0 | $0 | | Research and development | $93,697 | $63,492 | $34,269 | | General and administrative | $44,785 | $19,512 | $8,961 | | Net loss | $(119,337) | $(81,028) | $(43,274) | | Net loss per share | $(2.97) | $(2.30) | $(1.50) | Consolidated Balance Sheet Data (in thousands) | | Dec 31, 2020 | Dec 31, 2019 | Dec 31, 2018 | | :--- | :--- | :--- | :--- | | Cash and cash equivalents | $114,634 | $207,136 | $147,840 | | Total assets | $132,047 | $216,366 | $151,924 | | Total stockholders' equity | $105,836 | $198,903 | $140,527 | Management's Discussion and Analysis of Financial Condition and Results of Operations In 2020, Y-mAbs transitioned to a commercial-stage company with DANYELZA's approval, reporting $20.8 million in license revenue and an increased net loss of $119.3 million, with subsequent financing strengthening liquidity Comparison of Results of Operations (Years Ended Dec 31, in thousands) | | 2020 | 2019 | Change | | :--- | :--- | :--- | :--- | | License revenue | $20,750 | $0 | $20,750 | | Research and development | $93,697 | $63,492 | $30,205 | | General and administrative | $44,785 | $19,512 | $25,273 | | Loss from operations | $(119,935) | $(83,004) | $(36,931) | | Net loss | $(119,337) | $(81,028) | $(38,309) | - The increase in R&D expenses was primarily due to a $13.2 million increase in milestone and license acquisition costs related to the SADA agreement and a $13.4 million increase in personnel-related costs[805][806] - The increase in G&A expenses was mainly driven by a $12.7 million increase in commercial expenses for the DANYELZA launch and an $8.9 million increase in employee-related costs[807] - Net cash used in operating activities increased to $91.2 million in 2020 from $73.5 million in 2019, primarily due to the higher net loss and increased operating expenses[812][814] Quantitative and Qualitative Disclosures About Market Risk The company's primary market risks are interest rate risk and foreign currency exchange risk, both of which are considered immaterial due to the short-term nature of cash equivalents and limited foreign currency exposure - The company's exposure to interest rate risk is considered immaterial as cash equivalents are held in highly rated securities and are short-term in nature[844] - Foreign currency exchange risk is primarily related to the Danish Kroner (DKK) due to the company's Danish subsidiary, but as of December 31, 2020, the exposure was not material[845] Financial Statements and Supplementary Data This section presents audited consolidated financial statements, with PricewaterhouseCoopers LLP issuing an adverse opinion on internal controls due to material weaknesses, reflecting a $119.3 million net loss in 2020 - The independent auditor, PricewaterhouseCoopers LLP, issued an adverse opinion on the company's internal control over financial reporting as of December 31, 2020, due to material weaknesses[848][849] Consolidated Balance Sheet Highlights (in thousands) | | Dec 31, 2020 | Dec 31, 2019 | | :--- | :--- | :--- | | Current Assets | | | | Cash and cash equivalents | $114,634 | $207,136 | | Total Assets | $132,047 | $216,366 | | Current Liabilities | $19,535 | $13,586 | | Total Liabilities | $26,211 | $17,463 | | Total Stockholders' Equity | $105,836 | $198,903 | Consolidated Statement of Net Loss (in thousands) | | Year Ended Dec 31, 2020 | Year Ended Dec 31, 2019 | | :--- | :--- | :--- | | License revenue | $20,750 | $0 | | Total operating expenses | $140,685 | $83,004 | | Net Loss | $(119,337) | $(81,028) | Controls and Procedures Management concluded that disclosure controls were ineffective as of December 31, 2020, due to material weaknesses in internal control over financial reporting, with remediation efforts currently underway - Management concluded that disclosure controls and procedures were not effective as of December 31, 2020[989] - The ineffectiveness was due to material weaknesses, including a lack of sufficient trained accounting professionals, inadequate controls for complex accounting matters (like license arrangements and share-based compensation), and insufficient segregation of duties[992] - Remediation efforts are underway, including hiring more accounting staff, implementing new business process-level controls, and enhancing controls over journal entries and account reconciliations[995][996] Part III Directors, Executive Compensation, and Corporate Governance Information required for Items 10 through 14, covering directors, executive officers, corporate governance, executive compensation, security ownership, related transactions, director independence, and principal accounting fees, is incorporated by reference from the company's Definitive Proxy Statement for its 2021 Annual Meeting of Stockholders - Information regarding Directors, Executive Officers, Corporate Governance, Executive Compensation, Security Ownership, Certain Relationships, and Principal Accounting Fees is incorporated by reference from the forthcoming 2021 Proxy Statement[1001][1002][1003] Part IV Exhibits, Financial Statement Schedules This section lists the financial statements and exhibits filed as part of the Annual Report on Form 10-K, including corporate governance documents, material contracts, and executive certifications - This section contains the index of all exhibits filed with the Form 10-K, including corporate governance documents, material contracts, and executive certifications[1009]