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2016 Annual Report

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2016 Annual Report 2016 ANNUAL REPORT Dear Stockholders: As you are aware, 2016 was a critical year for Catalyst Pharmaceuticals. In February 2016, we received a “Refusal to File” (RTF) letter from the FDA with respect to the new drug application that we had submitted for Firdapse® in late 2015. Following receipt of the RTF letter, we worked diligently with the FDA to resolve the open issues raised in the letter and to obtain clarity around the clinical trials and studies that the FDA would require in order to accept for filing an NDA for Firdapse®. Our goal was to accomplish this as quickly as possible, in order to bring Firdapse® to market for the treatment of patients suffering with Lambert–Eaton myasthenic syndrome (LEMS) and congenital myasthenic syndromes (CMS). We were pleased in June to reach an agreement with the FDA on a confirmatory Phase 3 study protocol for Firdapse® and then, in October, to reach an agreement with FDA under a Special Protocol Assessment (SPA) for the protocol design, clinical endpoints, and statistical analysis approach in a second Phase 3 study evaluating Firdapse® for the treatment of LEMS. This agreement was a major milestone that provided us with a clearly defined development and regulatory pathway for Firdapse®. Catalyst is currently conducting its second Phase 3 trial evaluating Firdapse® for the treatment of LEMS at clinical trial sites in Miami, Florida and Los Angeles, California. This double-blind, placebo controlled withdrawal trial will include approximately 28 subjects and will have the same co-primary endpoints as Catalyst's first Phase 3 trial evaluating Firdapse® for the treatment of LEMS. Further, the FDA has agreed to allow Catalyst to enroll patients from its Expanded Access Program (EAP) as study subjects in this second trial. In December, we announced that the first patient had been enrolled into this trial. Based on the positive results seen in our previous Phase 3 study, we look forward to reporting top-line results from this trial in the second half of 2017. Additionally, we were pleased to enhance and expand our clinical study evaluating Firdapse® for the treatment of CMS to include adults with CMS, increasing the total number of participants in our trial to approximately 20, as we evaluate the efficacy and safety of Firdapse® in children and adult patients diagnosed with certain genetic mutations of CMS. Further, there are now a total of five sites participating in this U.S. study. Last month, we were pleased to report positive top-line results from the investigator-sponsored trial evaluating Firdapse® as a treatment for myasthenia gravis patients with anti-MuSK antibodies (MuSK- MG). Not only were the results statistically significant, but more importantly, the investigators observed a large clinical benefit to the patients. While several effective treatment options exist for the anti- acetylcholine receptor form of myasthenia gravis (AcHR-MG), MuSK-MG has been particularly refractory to current MG treatment options and represents an unmet medical need in the MG community of patients. If approved for this indication, Firdapse® has the potential to become the first line standard of care for MuSK-MG. We plan to continue discussions with the FDA and external experts about a regulatory path forward for a MuSK-MG pivotal, U.S. multi-center trial. This past August, the FDA also granted Orphan Drug designation to Firdapse® for myasthenia gravis and we expect that this will provide us with a number of benefits through the development and commercialization process. This year, we remain sharply focused on completing the current clinical trials required for LEMS and CMS, having an NDA submitted and accepted by the FDA for filing, and reinitiating our pre- commercialization activities for a potential 2018 launch of Firdapse®. The rest of our scientific pipeline remains important to us, and during 2017 we are continuing to further the development of CPP-115 for the treatment of refractory infantile spasms and, perhaps, adult refractory patients with Tourette's disorder, and a generic version of Sabril® (vigabatrin). We are also hopeful that ongoing discussions for the partnering of either or both of these programs will lead to meaningful transactions later this year. Lastly, Catalyst remains focused on its goal of improving access to treatments for patients with these significant unmet medical needs. Catalyst continues to grow its EAP with participating physicians and eligible patients suffering with LEMS and CMS. We recently launched our new EAP program website designed to make it even easier and transparent for patients to learn about Catalyst's EAP program and the possibility of getting access to Firdapse® at no cost if they are eligible. As part of our ongoing commitment to patients, we hope this website enables more patients with LEMS and CMS to understand our program and to learn more about this investigational treatment option for these debilitating diseases. On behalf of Catalyst’s Board of Directors, I would like to thank you for your investment and continued confidence in our company. Our board and management remain committed to serving the interests of all of our stakeholders, including the patients in our clinical studies, our clinical investigators, our corporate and institutional partners, our employees, and, of course, our stockholders. Regards, Patrick J. McEnany Chairman & Chief Executive Officer Catalyst Pharmaceuticals, Inc. April 14, 2017 2 UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K [Mark One] ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2016 OR TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File No. 001-33057 CATALYST PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) Delaware 76-0837053 (State of jurisdiction of incorporation or organization) (IRS Employer Identification No.) 355 Alhambra Circle, Suite 1250 Coral Gables, Florida 33134 (Address of principal executive offices) (Zip Code) Registrant’s telephone number, including area code: (305) 420-3200 Securities Registered Pursuant to Section 12(b) of the Act. Common Stock, par value $0.001 per share Nasdaq Capital Market (Title of each class) (Name of exchange on which registered) Securities registered pursuant to Section 12(g) of the Act.: None Indicate by check mark if registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes No Indicate by check mark if registrant is not required to file reports pursuant to Rule 13 or Section 15(d) of the Act. Yes No Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such report(s), and (2) has been subject to such filing requirements for the past 90 days. Yes No Indicate by check mark whether the registrant has submitted electronically and posted on its corporate web site, if any, every Interactive Data File required to be submitted and posted pursuant to rule 405 of Regulation S-T ((§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit and post such files). Yes No Indicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K (§229.405 of this chapter) is not contained herein, and will not be contained, to the best of registrant’s knowledge, in definitive proxy or information statements incorporated by reference in Part III of this Form 10-K or any amendment to this Form 10-K. Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer or a smaller reporting company. See the definitions of “large accelerated filer”, “accelerated filer” and “smaller reporting company” in Rule 12b-2 of the Exchange Act (Check one): Large accelerated filer Accelerated filer Non-accelerated filer (Do not check if a smaller reporting company) Smaller reporting company As of June 30, 2016, the last business day of the Registrant’s most recently completed second quarter, the aggregate market value of all voting, and non-voting common equity held by non-affiliates was $54,581,032. Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes No Indicate the number of shares outstanding of each of the issuer’s classes of common stock, as of the latest practicable date: 82,972,316 shares of common stock, $0.001 par value per share, were outstanding as of March 10, 2017. Part III incorporates certain information by reference from the registrant’s definitive proxy statement for the 2016 annual meeting of stockholders. The proxy statement with respect to the 2017 annual meeting of stockholders will be filed no later than 120 days after the close of the registrant’s fiscal year ended December 31, 2016. Table of Contents Page PART I........................................................................................................................................................................... 1 Item 1. Business………………………………………………………………………………………………………. 4 Item 1A. Risk Factors………………………………………………………………………………………………… 40 Item 1B. Unresolved Staff Comments……………………………………………………………………………….. 61 Item 2 Properties……………………………………………………………………………………………………… 61 Item 3. Legal
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