1. Organization MyoKardia, Inc. (the Company) is a clinical-stage biopharmaceutical company pioneering a precision medicine approach to discover, develop and commercialize targeted therapies for the treatment of serious and neglected rare cardiovascular diseases. The Companys initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. The Company has used its precision medicine platform to generate a robust pipeline of therapeutic programs for the chronic treatment of the two most common forms of heritable cardiomyopathyhypertrophic cardiomyopathy (HCM), and dilated cardiomyopathy (DCM). The Company was incorporated on June 8, 2012 in Delaware and its corporate headquarters and operations are located in South San Francisco, California. Liquidity The Company has incurred significant operating losses since inception and has an accumulated deficit of $186.9 million as of September 30, 2018. The Company has relied on its ability to fund its operations through private and public equity financings, and to a lesser extent through a license and collaboration arrangement with its collaboration partner, Sanofi S.A. (Sanofi) through its subsidiary, Aventis, Inc. The Company has not yet received regulatory approval to commercialize or sell any product and does not have customers. Management expects operating losses and negative operating cash flows to continue for the foreseeable future. As the Company continues to incur losses, the transition to profitability is dependent upon the successful development, approval, and commercialization of its products and product candidates and the achievement of a level of revenues adequate to support its cost structure. The Companys ultimate success depends on the outcome of its research and development activities. The Company expects to incur additional losses and negative cash flows for the foreseeable future and it anticipates the need to raise additional capital to fully implement its business plan. The Company intends to raise such capital through the issuance of additional equity, debt and/or strategic alliances with partner companies. On March 8, 2018, the Company filed a Registration Statement on Form S-3ASR (the 2018 Shelf Registration Statement) covering the potential offering, issuance, and sale of an indeterminate amount of common stock, preferred stock, debt securities, warrants and/or units. In May 2018, the Company completed a follow-on offering under the 2018 Shelf Registration Statement pursuant to which the Company issued 3,750,000 shares of common stock at a price of $49.00 per share. In addition, in June 2018, the Company sold an additional 211,174 shares of common stock directly to the underwriters when they partially exercised their over-allotment option at the price of $49.00 per share. Consequently , the Company received proceeds totaling approximately $181.9 million from the offering, net of underwriting discounts and commissions and offering expenses, which includes approximately $9.7 million received due to the underwriters partial exercise of their over-allotment option. As of September 30, 2018, the Company had $411.6 million of cash, cash equivalents and short and long-term investments and management believes that these amounts will be sufficient to meet the Companys anticipated operating and capital expenditure requirements for the twelve months following the date of this Form 10-Q. Managements belief with respect to its ability to fund operations is based on estimates that are subject to risks and uncertainties. If actual results are different from managements estimates, the Company may need to seek additional funding.