Catalyst Pharmaceuticals, Inc. · NASDAQ:CPRX
Catalyst Pharmaceuticals — Catalyst Pharmaceutical Partners — BIO CEO Investor Conference 2013
27 slides from Catalyst Pharmaceuticals's conference presentation, Feb 14, 2013
Slide 1: BIO CEO Investor Conference
ry catalyst PHARMACEUTICAL PARTNERS BIO CEO Investor Conference February 11, 2013 Patrick McEnany, CEO Steven Miller, PhD, COO/CSO
Slide 2: Safe Harbor
Safe Harbor This presentation contains forward-looking statements that are subject to a number of risks and uncertainties, manyofwhichareoutsideourcontrol.Allstatements regardingourstrategy,futureoperations, financial position, estimated revenues or losses, projected costs, prospects, plans and obje
Slide 3: Catalyst Overview
Catalyst Overview Catalyst Pharmaceutical Partners is focused on the development and commercialization of prescription drugs targeting rare (orphan) neurological diseases and disorders, including Lambert-Eaton Myasthenic Syndrome (LEMS), infantile spasms, and Tourette’s disorder Headquarters: Coral
Slide 4: A Glance In The Rear View Mirror
A Glance In The Rear View Mirror CPP-109 for Cocaine Addiction – Pivotal Phase II(b) trial showed no statistical difference for primary and secondary end points – Disappointing for all Catalyst stakeholders – Full data set available Q2 – 2013 – Meet with NIDA to discuss complete findings – Expect to
Slide 5: Firdapse/BioMarin Alliance
TM Firdapse /BioMarin Alliance Announced strategic alliance with BioMarin (in October) for TM the development of Firdapse TM North American license for Firdapse to treat neuromuscular diseases including LEMS Strategic fit with our other orphan drug programs – CPP-115 for Infantile Spasms and Tourett
Slide 6: Product Pipeline
Product Pipeline Preclinical Phase I Phase II Phase III Potassium TM Channel Firdapse : Lambert-Eaton Myasthenic Syndrome (LEMS) Blocker CPP-115: Infantile Spasms CPP-115: Complex Partial Seizures GABA-AT CPP-109: Tourette’s Disorder* Inhibitors CPP-115: Dyskinesia in Parkinson’s CPP-115: MS Note: *
Slide 7: Firdapse Potassium Channel Blocker
TM Firdapse Potassium Channel Blocker Amifampridine Phosphate (3,4-Diaminopyridine)
Slide 8: Firdapse Opportunity Summary
TM Firdapse Opportunity Summary Lambert-Eaton Myasthenic Syndrome (LEMS) is a rare autoimmune disease caused by auto-antibodies that inhibit acetylcholine release from nerve terminals – Chronic, often severly disabling and progressive – Usually managed by neurologists – ~3,000 patients in the U.S. (
Slide 9: LEMS and Firdapse Treatment
TM LEMS and Firdapse Treatment Insufficient ACh release due to LEMS Disease: antibodies to the pre-synaptic P/Q type voltage gated calcium channel – Proximal muscle weakness – Straight forward differential diagnosis – Can be disabling – Often worsens after diagnosis – In some cases, may be life thre
Slide 10: U.S. LEMS Product Need
U.S. LEMS Product Need Unserved U.S. population due to no FDA approved therapy – Off-label therapies include pyridostigmine and immunosuppressants – IVIg and plasmapheresis are also part of the standard of care Amifampridine is currently available to patients via investigator sponsored INDs and expa
Slide 11: Amifampridine Proven Efficacy and Safety in LEMS
Amifampridine Proven Efficacy and Safety in LEMS Study N Dose, duration Efficacy Outcomes McEvoy, 1989 12 Up to 100 mg/day Significant on CMAP, disability score, 3 day crossover arm/leg strength Sanders, 1993 18 Up to 100 mg/day Significant on QMG 8 day crossover Sanders, 2000 26 60 mg/day Significa
Slide 12: Firdapse U.S. Phase III Clinical Trial
TM Firdapse U.S. Phase III Clinical Trial Trial design – FDA concurred with design in June, 2010 meeting with BioMarin – Ethical design accepted by KOLs and FDA FDA requires one randomized, placebo-controlled, treatment discontinuation trial in LEMS patients – Compares amifampridine efficacy to plac
Slide 13: Firdapse U.S. Phase III Clinical Trial
TM Firdapse U.S. Phase III Clinical Trial Randomization N=30, 1:1 Last Patient ~March 2014 Firdapse™ Firdapse™ Firdapse™ Dose Taper Placebo Screening Open Label Run-In Double-Blind Treatment Phase Open-Label Safety Extension Days 1-4 Days 7-91 Day 1-7 Day 8-14 Up to 2 years Efficacy/Baseline Efficac
Slide 14: Firdapse Expansion Opportunities
TM Firdapse Expansion Opportunities Congenital Myasthenic Syndrome (CMS) – Prevalence of ~1,500 patients in the U.S. • Eligible for orphan drug designation • Prevalence may be under reported due to complexities of diagnosis – No approved therapy – Differential diagnosis complex – Confirmation of dia
Slide 15: Firdapse Regulatory Pathway
TM Firdapse Regulatory Pathway File NDA Estimated FDA Approval Phase III Top-Line Commercial Results Launch 2013 2014 2015 2016 H1 H2 H1 H2 H1 H2 H1 H2 Phase III Clinical Trial Phase III Safety Extension Clinical Safety Studies Pre-Clinical Studies Prepare NDA FDA Review Copyright© 2013 Catalyst Pha
Slide 16: Firdapse Commercialization Strategy
TM Firdapse Commercialization Strategy First FDA approved drug for LEMS Patient tracking and LEMS support groups to identify patients Market access through private and public payors – Market access research indicates drug will be widely reimbursed Specialty sales force – Initial sales force estimate
Slide 17: Beyond Firdapse: CPP-115
TM Beyond Firdapse : CPP-115 Next Generation GABA-AT Inhibitor
Slide 18: CPP-115 Innovation
CPP-115 Innovation Invented by Richard Silverman, Ph.D. – Inventor of Lyrica® (pregabalin); ~$4B in annual sales for Pfizer – Rationally designed drug with enhanced potency, specificity, and safety Exclusive worldwide license to commercialize new GABA-AT inhibitors Includes composition of matter pat
Slide 19: CPP-115 Targeted Indications
CPP-115 Targeted Indications IS Infantile spasms and complex partial seizures are proven Proven indications for inhibition of Indications GABA-AT. CPP-115 could be a safer and more effective CPS alternative. Tourette’s Potential New HypoGABAergic signaling Indications PTSD pathways are known to play
Slide 20: CPP-115 Superior Visual Safety
CPP-115 Superior Visual Safety Risk of visual field deficits with vigabatrin use – Black box warning on label and REMS program – Occurs in 1/3 to 1/2 of patients – Permanent loss of some peripheral vision Comparative vision safety study in rats Effective Vision Vision 45 Day Retinal 90 Day Retinal D
Slide 21: Infantile Spasms Opportunity
Infantile Spasms Opportunity Infantile spasms, or West Syndrome, is a Infantile Spasms Epidemiology (2010) catastrophic form of epilepsy for infants Incidence Prevalence Affects 10K – 20K infants worldwide, with 6,000 nearly one-half of them in the U.S. and 4,000 Europe 2,000 60-70% of patients have
Slide 22: CPP-115 Infantile Spasms Screening
CPP-115 Infantile Spasms Screening “Multiple-Hit Model” for ACTH-refractory infantile spasms (Albert Einstein College of Medicine) – Widely Respected model of infantile spasms – CPP-115 (0.1-1 mg/kg/day i.p.) suppressed spasms at 1/100th the dose of vigabatrin, with better tolerance than 1 vigabatri
Slide 23: CPP-115 Development Strategy
CPP-115 Development Strategy Met with FDA Oct 2011 to discuss IS development plans – Agreed on development strategy through phase II – Relatively standard development pathway to enter phase II • No unusual phase 1 or preclinical requirements, other than preclinical work in juvenile animals for IS in
Slide 24: CPP-115: Other Potential Indications
CPP-115: Other Potential Indications Indications for which no predictive animal models exist – Tourette’s disorder • 6-10 patient, open label, phase I/II study in progress • Top-line results Q4 2013 – Post Traumatic Stress Disorder (PTSD) – CPP-109 (vigabatrin) used as a research “surrogate” for CPP
Slide 25: Catalyst Milestones
Catalyst Milestones Recent Milestones Expected Milestones TM Acquired Firdapse , a phase III asset, Q1 2013 for development and commercialization – Firdapse TM DMC meeting results Completed $5,000,000 strategic Q4 2013 investment by BioMarin TM – Complete enrollment of Firdapse Initiated Phase I/II
Slide 26: Contact Information
Contact Information Catalyst Pharmaceutical Partners, Inc. Investor Relations 355 Alhambra Circle, Suite 1500 Rx Communications Coral Gables, FL 33134 Melody Carey (305) 529-2522 (917) 322-2568 mcarey@rxir.com Patrick J. McEnany Chairman and Chief Executive Officer pmcenany@catalystpharma.com Copyri
Slide 27: Catalyst Pharmaceutical Partners
yy catalyst PHARMACEUTICAL PARTNERS www.catal harma.com
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