MeiraGTx(MGTX) - 2019 Q2 - Quarterly Report

PART I. FINANCIAL INFORMATION Financial Statements (Unaudited) This section presents the unaudited condensed consolidated financial statements for MeiraGTx Holdings plc, including balance sheets, statements of operations, and cash flows Condensed Consolidated Balance Sheets Total assets and liabilities significantly increased due to higher cash and deferred revenue from a related party Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2019 | December 31, 2018 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $204,273 | $68,080 | | Total Current Assets | $212,875 | $74,652 | | Total Assets | $253,076 | $96,895 | | Liabilities & Equity | | | | Total Current Liabilities | $40,790 | $15,499 | | Deferred revenue—related party | $67,197 | $0 | | Total Liabilities | $123,513 | $15,835 | | Total Shareholders' Equity | $129,563 | $81,059 | Condensed Consolidated Statements of Operations and Comprehensive Loss Net loss decreased in Q2 and H1 2019 due to new license revenue and lower general and administrative expenses Statement of Operations Summary (in thousands) | Metric | Q2 2019 | Q2 2018 | H1 2019 | H1 2018 | | :--- | :--- | :--- | :--- | :--- | | License revenue—related party | $1,982 | $0 | $2,767 | $0 | | General and administrative | $13,437 | $17,378 | $21,937 | $28,500 | | Research and development | $9,772 | $7,791 | $22,748 | $14,718 | | Loss from operations | $(21,227) | $(25,169) | $(41,918) | $(43,218) | | Net loss | $(20,822) | $(29,981) | $(38,804) | $(46,384) | | Basic and diluted adjusted net loss per share | $(0.63) | $(2.29) | $(1.26) | $(4.27) | Condensed Consolidated Statements of Cash Flows Positive operating cash flow in H1 2019, driven by deferred revenue, significantly increased cash and equivalents Cash Flow Summary for the Six-Month Period Ended June 30 (in thousands) | Activity | 2019 | 2018 | | :--- | :--- | :--- | | Net cash provided by (used in) operating activities | $60,422 | $(36,425) | | Net cash used in investing activities | $(2,207) | $(1,318) | | Net cash provided by financing activities | $77,561 | $131,470 | | Net increase in cash, cash equivalents and restricted cash | $135,776 | $93,726 | Notes to Condensed Consolidated Financial Statements Notes detail accounting policies, liquidity, lease standard adoption, and the $100 million Janssen collaboration - The company is a clinical-stage biotech company focused on developing gene therapies for ocular diseases, xerostomia, and neurodegenerative diseases like Parkinson's and ALS[28] - As of June 30, 2019, the company had cash and cash equivalents of $204.3 million and believes these funds are sufficient to cover expenses for at least the next twelve months, largely due to a $100 million upfront payment from Janssen[32] - The company adopted the new lease accounting standard (ASC 842) on January 1, 2019, resulting in the recognition of a right-of-use asset of $10.8 million and a corresponding lease obligation of $3.7 million upon implementation[67] - On January 30, 2019, the company entered into a collaboration agreement with Janssen, receiving a non-refundable upfront fee of $100 million for the development and commercialization of gene therapies for inherited retinal diseases[94] Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses financial condition, operating results, and liquidity, including clinical developments and funding Overview The clinical-stage gene therapy company incurred significant losses, financed primarily by equity sales and a Janssen payment - MeiraGTx is a vertically integrated, clinical-stage gene therapy company with six programs in clinical development, focusing on ophthalmology, salivary gland, and neurodegenerative diseases[146] Financial Snapshot as of H1 2019 | Metric | Value (in millions) | | :--- | :--- | | Net Loss (H1 2019) | $38.8 | | Net Loss (H1 2018) | $46.4 | | Accumulated Deficit (as of June 30, 2019) | $187.1 | | Cash and Cash Equivalents (as of June 30, 2019) | $204.3 | Highlights and Recent Development Recent clinical progress includes positive AAV-RPE65 data, planned FDA meetings, and increased personnel - AAV-RPE65 for RPE65-Deficiency: Reported positive 6-month data from the Phase 1/2 trial, achieving the primary safety endpoint and demonstrating statistically significant improvement in secondary endpoints. The company plans to meet with global regulatory authorities in 2019 to discuss the path to approval[158] - AAV-GAD for Parkinson's Disease: The company anticipates meeting with the FDA in 2019 to define the clinical pathway for regulatory approval[159] - Corporate Growth: The company has substantially increased its number of key personnel and now has more than 100 full-time employees to advance its pipeline[162] Results of Operations Net loss decreased in Q2 and H1 2019 due to license revenue and lower G&A, offsetting increased R&D Comparison of Results for the Three Months Ended June 30 (in millions) | Item | 2019 | 2018 | Change | | :--- | :--- | :--- | :--- | | License revenue—related party | $2.0 | $0.0 | $2.0 | | General and administrative | $13.4 | $17.4 | $(3.9) | | Research and development | $9.8 | $7.8 | $2.0 | | Net loss | $(20.8) | $(30.0) | $9.2 | Comparison of Results for the Six Months Ended June 30 (in millions) | Item | 2019 | 2018 | Change | | :--- | :--- | :--- | :--- | | License revenue—related party | $2.8 | $0.0 | $2.8 | | General and administrative | $21.9 | $28.5 | $(6.6) | | Research and development | $22.7 | $14.7 | $8.0 | | Net loss | $(38.8) | $(46.4) | $7.6 | Liquidity and Capital Resources The company had $204.3 million in cash, with management expecting funds to support operations into 2022 - The company had $204.3 million in cash and cash equivalents as of June 30, 2019[197] - Management estimates that current cash and expected research funding will be sufficient to fund operating expenses and capital expenditure requirements into 2022[205] Cash Flow Summary for the Six Months Ended June 30 (in millions) | Activity | 2019 | 2018 | | :--- | :--- | :--- | | Net cash from operating activities | $60.4 | $(36.4) | | Net cash used in investing activities | $(2.2) | $(1.3) | | Net cash from financing activities | $77.6 | $131.5 | Quantitative and Qualitative Disclosures About Market Risk No material changes in market risk have occurred since the prior fiscal year-end - There have been no material changes in the company's market risk since the end of the previous fiscal year[212] Controls and Procedures Disclosure controls were effective as of June 30, 2019, with no material changes in internal control - Management, including the CEO and CFO, evaluated the effectiveness of disclosure controls and procedures and concluded they were effective at a reasonable assurance level as of June 30, 2019[214] - No changes in internal control over financial reporting occurred during the quarter that have materially affected, or are reasonably likely to materially affect, the company's internal control[215] PART II. OTHER INFORMATION Legal Proceedings The company is not currently subject to any material legal proceedings - The company is not subject to any material legal proceedings[216] Risk Factors This section details numerous risks affecting the company's business, financial condition, and operations - The company has a history of significant losses, with an accumulated deficit of $187.1 million as of June 30, 2019, and anticipates continued losses for the foreseeable future[218] - The business is heavily dependent on the success of its most advanced product candidates, which are still in development and may never receive regulatory approval or be successfully commercialized[229] - Gene therapy is a novel field with an uncertain and evolving regulatory landscape, which makes it difficult to predict the time and cost of obtaining regulatory approval[237] - The company faces significant competition from larger, more established pharmaceutical and biotechnology companies with greater financial resources and expertise[323] Unregistered Sales of Equity Securities and Use of Proceeds All $65.3 million net proceeds from the June 2018 IPO have been fully utilized - The company completed its IPO on June 12, 2018, receiving $65.3 million in proceeds after expenses[467] - As of June 30, 2019, the company had used all of the net proceeds from its IPO[467] Defaults Upon Senior Securities No defaults upon senior securities occurred during the period - None[468] Other Information Information on new lease agreements in New York and London is incorporated by reference - Information regarding a new sublease in New York and a new lease in London is incorporated by reference from Notes 9 and 11 of the financial statements[469] Exhibits This section lists exhibits filed, including amended articles, agreements, and officer certifications Signatures The report is duly signed and authorized by the CEO and CFO on August 7, 2019