jefferies 2017 global healthcare conference...confidential information lems –lambert-eaton...

Confidential Information

Jefferies 2017Global Healthcare Conference

June 6, 2017


Safe HarborThis presentation contains forward-looking statements that are subject to a number of risks and uncertainties, many of which are outside our control. All statements regarding our strategy, future operations, financial position, estimated revenues or losses, projected costs, prospects, plans and objectives, other than statements of historical fact included in our filings with the U.S. Securities and Exchange Commission (“SEC”), are forward-looking statements. When used in this presentation or in answers given to questions asked today, the words “may,” “will,” “could,” “would,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “potential,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. You should not place undue reliance on forward-looking statements. While we believe that we have a reasonable basis for each forward-looking statement that we make, we caution you that these statements are based on a combination of facts and factors currently known by us and projections of future events or conditions, about which we cannot be certain. Forward-looking statements in this presentation should be evaluated together with the many uncertainties that affect our business, and particularly those mentioned in the “Risk Factors” section of our Annual Report on Form 10-K filed with the SEC, reporting our financial position and results of operations as of and for the year ended December 31, 2016, as well as subsequent reports filed with the SEC. In addition, market and industry statistics contained in this presentation are based on information available to us that we believe is accurate. This information is generally based on publications that are not produced for purposes of securities offerings or economic analysis. All forward-looking statements speak only as of the date of this presentation. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.

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EAP=Expanded Access Program ISI=Investigator Sponsored IND1 Orphan Drug Designation2 Breakthrough Therapy Designation3 If development continues, it would likely be for adult refractory Tourette’s Disorder

Pipeline

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Technology Platform &

Indication

Access Development Stage

EAP ISI Preclin Phase 1 Phase 2 Phase 3 Regulatory

Neuronal Potassium Channel Blocker (Firdapse®)

Lambert-Eaton Myasthenic Syndrome (LEMS)1,2

Congenital Myasthenic Syndromes (CMS)1

MuSK-Myasthenia Gravis (MuSK-MG)1

Downbeat Nystagmus

GABA-AT Inhibitor (CPP-115 and CPP-109)

Infantile Spasms1

Tourette’s Disorder3

Generic Sabril® (Vigabatrin, 500 mg) NDA Phases Not applicable to ANDAs

Clinical program not yet defined


Catalyst Pharmaceuticalsis dedicated to advancing therapies targeting rare neuromuscular and neurological diseases, including:

Lambert-Eaton Myasthenic Syndrome (LEMS)

Congenital Myasthenic Syndromes (CMS)

MuSK-Myasthenia Gravis (MG)

Infantile Spasms


Lead ProgramsFirdapse®

Amifampridine Phosphate(3,4-Diaminopyridine

Phosphate Salt)

NH2N

NH2

•H3PO4


Firdapse® Positioned for Commercial Success• Potentially life-altering therapy for rare neuromuscular diseases

– Lambert-Eaton Myasthenic Syndrome (LEMS)• Lead indication• Marketed in the EU by BioMarin for LEMS

– Recommended first line therapy in the EU for LEMS– Congenital Myasthenic Syndromes (CMS)– Myasthenia Gravis, MuSK antibody positive subtype (MuSK-MG)

• Exclusivities– LEMS: Breakthrough Therapy and Orphan Drug Designations– CMS: Orphan Drug Designation– MG: Orphan Drug Designation– New chemical entity (NCE) exclusivity on approval

• Estimated U.S. prevalence– ~3,000 patients for LEMS– ~1,000-1,500 patients for CMS– ~3,000-4,800 patients for MuSK-MG

• Clinical development– Successfully completed a positive Phase 3 trial for treating LEMS with Firdapse®

• Second Phase 3 trial to complete 2nd half 2017– Phase 3 trial for CMS to complete 2nd half 2017– Double blind, placebo controlled proof of concept trial for MuSK-MG successfully

completed• Planning to start a phase 3 trial for MuSK-MG subject to the availability of funding

• Plan to file NDA 2017

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LEMS – Lambert-Eaton Myasthenic Syndrome

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• Proximal muscles most

commonly affected

• Commonly affected areas

shown in red (below)

• Gradually progresses

• Can be life shortening

Clinical Characteristics Epidemiology

• Typically diagnosed > age 40

• Can occur in children

• Antibodies bind to calcium

channels of nerves

• Typically diagnosed > age 50

• SCLC comorbidity

• Antibodies to SCLC

carcinoma also bind to

calcium channels of nerves

Autoimmune

~50% Paraneoplastic

~50%


Current Treatment Options Are Not AdequateLEMS – There Is No Cure

• Concerns about safety and efficacy ofcurrent therapies– Off label use: IVIG, plasmapheresis,

steroids, immune suppressants– All with poor efficacy

• Expanded access to Firdapse® or 3,4-DAP– Catalyst Pharmaceuticals-sponsored IND– Jacobus Pharmaceutical’s drug through

investigator-sponsored expanded access INDs• Less stable freebase form

• Compounding of 3,4-DAP– Minimal, and decreasing– FDA Pharmacy Compounding Advisory

Committee recommended againstcompounding of this drug• With Passage of Drug Quality & Security

Act in 2013, compounding of 3,4-DAPprevented by regulation in most cases

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Treatment With Firdapse®

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FDA Regulatory Status

• Key regulatory events– February 2016: Refusal to file letter– April 2016: Meeting with FDA regarding refusal to file letter– October 2016: Special Protocol Assessment (SPA) reviewed

and agreed to by FDA

• Final FDA requirements– A second well-controlled trial will be required

• Underway and expected to finish 2nd half 2017

– Additional toxicology studies are required• Required due to evolving FDA position on pre-clinical studies

for all CNS drugs

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Firdapse® LEMS Trial (LMS-003)

• Double-blinded, parallel design– Catalyst’s existing EAP patients by invitation– Out patient study– Clinical trial sites: Miami and LA

• www.clinicaltrials.gov (NCT02970162)• Top-line results in second half of 2017

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EAP Firdapse®

Firdapse®

Placebo

EAP

Randomization

~28 Subj, ~1:1

Enter from EAP Screening Randomization Period Exit to EAP

N/A 1-7 days 4 days N/A

Treatment success and on stable dose

Baseline Efficacy and Safety Assessments

Efficacy Assessments on Days 4 Exit Safety Assessments

1°: QMG-Quantitative Myasthenia Gravis ScoreSGI-Subject Global Impression

2°: CGI-I-Clinical Global ImpressionExploratory: 3TUG-Triple Timed Up and Go

Most bothersome symptom severity

http://www.clinicaltrials.gov/


CMS Market Expansion Opportunity• Clinical characteristics

– Muscle weakness resulting from inadequate neuromuscular junction transmission

– Similar clinical presentation to LEMS, but genetic in origin• 24 mutations known, CHRNE, COLQ, RAPSN, DOK7 most common

– No approved therapies– Early onset: 2/3 adolescents and children

• Estimated addressable US prevalence: 1,000-1,500 patients• Evidence of amifampridine efficacy

– Published open-label studies of efficacy and small blinded study– CMS patients successfully being treated in Catalyst EAP program

• Orphan Drug Designation granted March 2015 for CMS• FDA reviewed protocol and provided comments

– FDA comments incorporated into ongoing study• Top-line results in second half of 2017

– If successful, will add CMS indication to any NDA we file for Firdapse

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Firdapse® CMS Trial

• Adults and pediatric patients– EAP patients and newly recruited naïve patients– Five clinical trial sites

• www.clinicaltrials.gov (NCT 02562066)• Top-line results in second half of 2017

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Firdapse®

Firdapse®

Placebo Firdapse®

PlaceboFirdapse®

Firdapse®

EAP

Randomization

~20 Subj, ~1:1

Screening Run-in Period 1 Re-stabilization Period

Period 2 Exit to EAP

1-3 weeks 1-4 weeks 8 days ~ 2 weeks 8 days N/A

Baseline Assessments & Screening

Baseline and Safety Assessments & dose stabilization

Efficacy Assessments on Days 1 and 7

Baseline and Safety Assessments

Efficacy Assessments on Days 21 and 28

Exit Safety Assessments

1°: MFM 20 or 32, depending on age andSGI or caregiver assessment, depending on age

2°: CGI-I and CGI-S

http://www.clinicaltrials.gov/


MuSK-Myasthenia Gravis• Clinical Characteristics

– Affects primarily head and neck musculature– Episodic respiratory crises requiring hospitalization– Generally unresponsive to current MG standard of care

• Pyridostigmine and/or steroids– Reports of efficacy for treating MuSK-MG using Rituxan

• Most doctors seem reluctant to use it for MG• Estimated addressable US Prevalence: 3,000-4,800 patients

– Well defined population diagnosed with readily available commercial antibody test

• Evidence of amifampridine efficacy– Successful proof of concept trial

• Investigator-sponsored well controlled clinical trial• Dr. Renato Mantegazza, Milan Italy, Carlo Besta Neurological Institute

– Pre-clinical and indirect clinical evidence of efficacy• Orphan Drug Designation in September 2016 for Myasthenia Gravis• Planning multicenter phase 3 trial

– Will seek FDA input• Indication will be a supplement to an approved NDA for Firdapse

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MuSK-MG Proof of Concept Trial• N=7, double blind, placebo controlled, double crossover

“switchback” design– Switchback design permits mathematical elimination of carryover

while still getting the power benefits of a crossover design• Trial outcome

• Other notable findings– Very little carry over– Very homogeneous population and response

• All 7 patients had large, clinical significant, benefit

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End point Effect Size Clinically Significant

P-value

MGC -11.5 ≥ 3 change


Intellectual Property• Exclusivities

– ODE for LEMS indication: 7 years from indication approval– ODE for CMS indication: 7 years from indication approval– ODE for MG indication: 7 years from indication approval– NCE for amifampridine: 5 years from approval of the drug for any and all

indications– Pediatric for CMS: 6 months appended to ODE for respective indication– NCE prevents filing of ANDAs and 505(b)(2) applications for the exclusivity

period– ODE and Pediatric exclusivity prevents approval of an ANDA or 505(b)(2) for

the covered indication• Patents (pending)

– Use of Firdapse® to treat LEMS– Method of use (dosing regimen) related to variability of metabolism in the

human population• Life cycle management

– 3-4 times a day dosing of current product would support development of, and switch to, a sustained release product

– New dosage form exclusivity: 3 years– Patents related to new dosage form will be pursued

• Patent term is 20 years from the date of filing

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Firdapse® Expanded Access Program

• Ensures eligible patients have access to amifampridine phosphate (FIRDAPSE®) during product development– Catalyst sponsored IND

• Minimize regulatory workload on physicians to the extent allowed by law

• Currently provided in U.S. only– Patient access to therapy while FDA approval is pursued– Provided free of charge– Drug shipped direct to patients

• Diseases treated in EAP:– Lambert-Eaton Myasthenic Syndrome (LEMS)– Congenital Myasthenia Syndromes (CMS)

• Geno-types known not to respond to amifampridine excluded• Per FDA EAP guidance, MuSK-MG not included, pending

completion of clinical trials for MuSK-MG

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Firdapse® Expanded Access Program (EAP)Patient Outreach

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MDA Quest Magazine –targeting patientsand caregivers


Patient Outreach and Meetings

• Global Genes– Catalyst provided grant for LEMS

patient meeting• January 2017

– LivingwithLEMS.org • Patient visits to Catalyst office• Individual patient discussions• Patient email updates• Myasthenia Gravis Foundation of

America (MGFA)– Working with MGFA on webinar

sponsorship– Attended annual patient summit

• LEMS and CMS disease awareness campaign

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Firdapse® Addressable Market

LEMS

CMS

MG-MuSK

~3,000

*Based on market research

Addressable

Patients*

~1,000-1,500

~3,000-4,800

Completed Phase 3 Pivotal Trial

Second Phase 3 In Progress

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~8,000 Total Patients

Phase 3 Trial In Progress

POC Completed

Phase 3 planned


CPP-115 for Neurological Disorders

Next-Generation GABA-Aminotransferase Inhibitor

NH2

OH

O

F

F


CPP-115, A Novel GABA-AT Inhibitor

• A new molecular entity– Safer, more potent analog of vigabatrin

• Invented by Richard Silverman, Ph.D.– Inventor of Lyrica® (pregabalin);

~$4B in annual sales for Pfizer– Rationally designed drug

• Exclusive worldwide license to commercialize new GABA-AT inhibitors, August 2009

• Includes composition of matter patents to a new class of inhibitors– Protection through 2028 with patent extensions allowed under Patent

Term Restoration Act• Pending application to protect CPP-115 in ex-U.S. markets

– Issued in U.S., pending in EU• Use patent issued in U.S. for Tourette’s Disorder• Catalyst expects to make CPP-115 “Phase 2 ready”

– Catalyst will determine if CPP-115 will be partnered or continued by Catalyst

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CPP-115 for Infantile Spasms

• Large market opportunity for Infantile Spasms (IS)• Oral therapy with better safety & tolerability than vigabatrin• Infant with refractory IS successfully

treated with CPP-115• Orphan drug designation in US & EU• Leading therapies are not adequate

– Sabril® exhibits somnolence and VFD side effects– Acthar® Gel is difficult to administer and exhibits

hyperglucocorticoid side effects• Affects 10,000-20,000 infants globally

– 5,000-10,000 in US– About half are refractory to all current treatments

• Phase 1 study completed– Large increases in brain GABA observed by MRI– Somnolence was observed during phase 1 studies

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Generic Sabril®

Vigabatrin Tablets andPowder for Oral Solution


Generic Sabril® (Vigabatrin, 500 mg)

• One time opportunity to leverage pipeline product• Catalyst is uniquely qualified to develop generic Sabril®• Sabril® sales $193MM (2016), +36% YOY

– 2017 estimate $250MM, +27% YOY– Sales starting to accelerate due to relaxed REMS program

• Leverage Catalyst’s experience with vigabatrin– Vigabatrin (CPP-109) has been part of Catalyst’s pipeline for a

number of years– Catalyst team has previous generic drug industry experience

• Generic version of sachet formrecently approved (Par-Endo)

• Likely no competition for tablets• Initiated a formal process for

evaluating potential partners

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Catalyst Milestones for Near-Term Value Creation

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Timing Milestone

Q4 2016 Initiate (first patient in) second required LEMS trial (LMS-003)

H1 2017 Top-line data from investigator sponsored MuSK-MG trial (Italy)• Update on Vigabatrin (Sabril®) ANDAs

H2 2017 • Last patient enrolled in LMS-003• Top-line data from LMS-003 trial• Top-line data from CMS-001 trial• Resubmit Firdapse® NDA (LEMS and CMS indications)• Firdapse® NDA acceptance for filing by FDA• Subject to funding, expect to initiate MuSK-MG phase 3 trial


Stock Information

• Market Cap: ~$190MM (as of 6/2/17)• Common S/O: 82.9MM* shares• Cash and investments: $36.5MM*• Debt: None• NASDAQ trading symbol: CPRX• Many high quality life science

institutional investors

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*Per 3/31/17 Form 10-Q


www.catalystpharma.com

Patrick McEnany, CEO(305) [email protected]

Investor RelationsBrian KorbThe Trout Group, LLC(646) [email protected]

Public RelationsDavid SchullRusso Partners, LLC(212) [email protected]
http://www.catalystpharma.com/mailto:[email protected]:[email protected]:[email protected]

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