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2014 ANNUAL REPORT Catalyst Is Committed to Changing the Lives of Patients with Rare, Debilitating Diseases

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2014 ANNUAL REPORT Catalyst Is Committed to Changing the Lives of Patients with Rare, Debilitating Diseases 2014 ANNUAL REPORT Catalyst is Committed to Changing the Lives of Patients With Rare, Debilitating Diseases • Successfully completing the largest phase 3 clinical trial in LEMS patients • Introduced the Firdapse Expanded Access Program (EAP) for patient suffering with LEMS or CMS • Catalyst continues to provide strong support to both patient and professional associations including AANEM, AAN, ANA, MDA and NORD Example of the Firdapse EAP patient announcement as seen in MDA Quest Magazine ©2015 Catalyst Pharmaceutical Partners, Inc. (Catalyst Pharmaceuticals )CAT-15005 All Rights Reserved. Printed in the USA Dear Stockholders, Last year was a transformative year for Catalyst Pharmaceuticals, as we successfully completed our pivotal Phase 3 clinical trial for our lead program and began laying the groundwork to become a fully integrated biopharmaceutical company with a direct marketing organization. In September 2014, we reported positive results from a Phase 3 clinical trial evaluating Firdapse® for the treatment of Lambert-Eaton Myasthenic Syndrome, otherwise known as LEMS. As I am sure you are aware, LEMS is a rare neuromuscular, autoimmune disorder frequently associated with small cell lung cancer. There is currently no FDA approved treatments for patients with LEMS. In our trial, both co-primary endpoints, quantitative myasthenia gravis score and subject global impression, demonstrated that Firdapse® was significantly superior to placebo, as did a secondary endpoint for the physician's clinical global impression of improvement. Following on from this, we initiated an expanded access program (EAP) available on a compassionate use basis under which patients with LEMS and Congenital Myasthenic Syndrome (CMS) who meet the inclusion and/or exclusion requirements have access to Firdapse® via the EAP at no charge. Having already been granted Orphan Drug Designation and Breakthrough Therapy Designation by the FDA for Firdapse®, in early 2015 we continued our collaborative discussions with the agency about the best pathway to bring Firdapse® to market for the benefit of patients suffering from LEMS and from certain types of CMS. We expect to complete a full NDA submission for Firdapse® for the treatment of LEMS in Q3 2015. We have begun to build a commercial organization ahead of a potential launch of the product in 2016. Our symposium at the 2014 Annual Meeting of the American Association of Neuromuscular and Electrodiagnostic Medicine (AANEM), a key meeting for specialists treating the LEMS patient community, had a large an enthusiastic audience. I think I speak for the entire Catalyst team when I say that I am humbled to be a part of this dedicated physician and patient advocate LEMS community. Earlier this year we promoted David D. Muth to Chief Commercial Officer as we continue to accelerate pre- commercial activities. In 2014, we also expanded our team with the addition of David J. Caponera to the newly created position, Vice President, Patient Advocacy and Reimbursement. In that role, David is actively working to develop a patient advocacy strategy and appropriate reimbursement and patient assistance initiatives. In addition to LEMS, we also believe that Firdapse® has the potential to treat other neuromuscular disorders such as certain forms of CMS and myasthenia gravis caused by antibodies to muscle-specific tyrosine kinase (MuSK MG), and we plan to explore these additional indications for Firdapse®. We are also continuing to make progress with the rest of our drug pipeline. We are currently developing CPP-115, a novel GABA-aminotransferase inhibitor, which has potential in a broad range of central nervous system indications, such as infantile spasms, epilepsy, Tourette disorder and Post Traumatic Stress Disorder (PTSD). In September 2014, we initiated our second Phase 1 safety and tolerance study of CPP-115 (a multiple ascending dose study), and we expect to report top-line data from this Phase 1(b) study during the 2nd quarter of 2015. We are committed to the development of our pipeline and have plans to explore other selected diseases in which modulation of GABA levels by CPP-115 might be beneficial, providing further opportunities to expand the market and serve additional patients with unmet medical needs. We believe that Catalyst controls all current intellectual property for GABA-aminotransferase inhibitors, and most recently, a patent was issued for the Reduction or Elimination of Visual Field Defects by Treating Patients with CPP-115. We are currently supporting an academic investigator proof-of-concept study being conducted at Mount Sinai Hospital in New York City evaluating our other GABA-aminotransferase inhibitor, CPP-109 (our formulation of vigabatrin) for the treatment of Tourette disorder, a large orphan indication, and we expect to report top-line results from this study during the 2nd quarter of 2015. If the results of this study show evidence of reduced numbers of tics, we will likely seek to develop CPP-109 or CPP-115 for this indication. Over the past year, we have significantly strengthened our balance sheet. In April 2014, we raised net proceeds of approximately $26.7 million through a sale of 13,023,750 shares of our common stock, and, in February 2015, we raised net proceeds of approximately $34.7 million from a sale of 11,500,000 shares of our common stock. On a pro forma basis, including the net proceeds of the February 2015 offering, we had cash and investments of approximately $74 million as of December 31, 2014, giving us the capital to support our operations through 2016, including our currently anticipated drug development activities, our development of a commercial infrastructure that can market Firdapse® if we receive an approval for the product and, hopefully, a successful launch of the product during that period. Finally, during 2014 and into 2015, we have been pleased to see the substantial improvement in the quality and number of institutional investors who have made significant investments in our company. In the midst of all the success we are seeing at Catalyst, I would like to take the time to fondly remember Hubert E. Huckel, M.D., a founding shareholder and director of Catalyst, who sadly passed away at the end of last year. His contributions to our company were invaluable, and he will be greatly missed. I would like to thank all of the subjects in our clinical studies, our clinical investigators, our strategic partner, BioMarin Pharmaceuticals, our employees and stockholders like yourself, all of whom are helping bring new drugs to market to treat unmet medical needs. We are committed to the LEMS patient community, as well as to other patient populations with unmet medical needs. We will work tirelessly in 2015 and 2016 to bring Firdapse® to market and to build value through our pipeline. We look forward to keeping you updated on our progress. Regards, Patrick J. McEnany Chairman, President and CEO Catalyst Pharmaceuticals March 30, 2015 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, 2014 OR TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File No. 001-33057 CATALYST PHARMACEUTICAL PARTNERS, 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 1500 Coral Gables, Florida 33134 (Address of principal executive offices) (Zip Code) Registrant’s telephone number, including area code: (305) 529-2522 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 i ((§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, 2014, 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 $155,035,298.
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