CATALYST PHARMACEUTICALS, INC. - FORM 8-K - EX-99.1

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8-K - FORM 8-K - CATALYST PHARMACEUTICALS, INC.	d482585d8k.htm
EX-99.2 - EX-99.2 - CATALYST PHARMACEUTICALS, INC.	d482585dex992.htm

Exhibit 99.1

Page 2

2013 Catalyst Pharmaceutical Partners, Inc.

This presentation contains forward-looking statements that are subject to a number of risks and uncertainties,

many

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 (the

“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

the

“Risk

Factors”

section

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, 2011, in the Registration Statement on Form S-1 that we filed with the

SEC on April 6, 2012, as well as subsequent reports filed with the SEC during 2012. 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.

Safe Harbor

Page 3

2013 Catalyst Pharmaceutical Partners, Inc.

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 Gables, FL

NASDAQ Capital Market:

CPRX

Shares outstanding:

41,420,687

Share price (2/7/13):

$0.54

Market capitalization:

$22.4M

Cash and investments:

$17M*

Lead institutional investors:

Federated, Millenium, Sophrosyne

Strategic investment:

BioMarin (16% and joint development

agreement)

*As of 9/30/12, pro-forma for BioMarin $5MM investment

Catalyst Overview

Page 4

2013 Catalyst Pharmaceutical Partners, Inc.

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

–

2013

–

Meet with NIDA to discuss

complete findings

–

Expect to present data at conference later this year

–

Trials

for

cocaine

addicts

difficult

patient

population

Catalyst will not continue

development of addiction drugs

A Glance In The Rear View Mirror

Page 5

2013 Catalyst Pharmaceutical Partners, Inc.

Announced strategic alliance with BioMarin (in October) for

the

development

Firdapse

North

American

license

for

Firdapse

treat

neuromuscular diseases including LEMS

Strategic fit with our other orphan drug programs

–

CPP-115 for Infantile Spasms and Tourette’s disorder

BioMarin invested $5 million for a 16.6% equity stake

Joint development agreement for several remaining

studies-

sharing costs 50/50

Several milestones to be paid later and a mid-teen royalty

payment

Firdapse

/BioMarin

Alliance

Page 6

2013 Catalyst Pharmaceutical Partners, Inc.

Phase I

Preclinical

Phase II

Phase III

Note: *Investigator sponsored study, CPP-109 is a model for CPP-115 to treat this disorder

Firdapse

: Lambert-Eaton Myasthenic Syndrome (LEMS)

CPP-115: Complex Partial Seizures

CPP-109: Tourette’s Disorder*

CPP-115: Infantile Spasms

Potassium

Channel

Blocker

GABA-AT

Inhibitors

CPP-115: Dyskinesia

in Parkinson’s

CPP-115: MS

Product Pipeline

Firdapse

Potassium Channel Blocker

Amifampridine

Phosphate

(3,4-Diaminopyridine)

Page 8

2013 Catalyst Pharmaceutical Partners, Inc.

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. (10 per 1M prevalence)

Continuing pivotal Phase III trial designed and initiated by BioMarin with FDA

input

Orphan drug designation in the U.S.

BioMarin

launched

Firdapse

Europe

(2Q10)

for

the

treatment of LEMS

–

EFNS

recommends

Firdapse

the

first-line

symptomatic treatment for LEMS

–

European annual cost of therapy ~$60,000 USD

Independent market research indicates

annual peak U.S. sales of ~$100 million

Opportunities for label expansion

Pending composition of matter patent

Firdapse

Opportunity

Summary

Page 9

2013 Catalyst Pharmaceutical Partners, Inc.

Insufficient ACh release due to

antibodies to the pre-synaptic P/Q type

voltage gated calcium channel

LEMS Disease:

–

Proximal muscle weakness

–

Straight forward differential diagnosis

–

Can be disabling

–

Often worsens after diagnosis

–

In some cases, may be life threatening

–

~50% of cases associated with SCLC, which has a

12-24 month life expectancy

Firdapse

Treatment:

–

Potassium channel blocker

–

Delays neuron repolarization

–

Voltage gated calcium channels remain open

longer

–

Increased calcium influx causes more

acetylcholine to be released to the innervated

muscle cells

–

Restoration of lost muscle strength

LEMS

and

Firdapse

Treatment

Page 10

2013 Catalyst Pharmaceutical Partners, Inc.

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 expanded access INDs

–

Difficult for patients to obtain drug

•

Even with legally allowed options, many physicians are

unwilling or unable to utilize them

–

Unclear if/how safety reporting is being done

–

Inadequate control of product manufacturing

•

Uniformity of potency

•

Stability

U.S. LEMS Product Need

Page 11

2013 Catalyst Pharmaceutical Partners, Inc.

Study

Dose, duration

Efficacy Outcomes

McEvoy, 1989

Up to 100 mg/day

3 day crossover

Significant on CMAP, disability score,

arm/leg strength

Sanders, 1993

Up to 100 mg/day

8 day crossover

Significant on QMG

Sanders, 2000

60 mg/day

6 day parallel arm

Significant

QMG,

CMAP

Wirtz, 2009

Single IV dose, 10

Significant on isometric force, CMAP

Oh, 2009

75 –

80 mg/day

3-8 day crossover

Significant on symptom severity,

QMG, muscle strength and CMAP

Amifampridine Proven Efficacy and Safety in LEMS

Significant database of exposure in the literature

–

1174 patient exposures for all uses

–

169 in LEMS patients

Page 12

2013 Catalyst Pharmaceutical Partners, Inc.

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 placebo at the end of a 14-day

discontinuation period

–

Primary endpoint: Muscle strength (Quantitative Myasthenia Gravis score

[QMG])

–

Secondary endpoint: Walking speed (Timed 25-foot walking test)

–

Tertiary endpoint: Compound Muscle Action Potential (CMAP)

Approximately 1/3 enrolled

7 active sites (4 U.S./3 Europe) with up to 20 to be added to accelerate

the study

Data Monitoring Committee (DMC) review Q1 2013

Expect to complete double blind stage of trial around end of Q1 2014

Firdapse

U.S. Phase III Clinical Trial

Page 13

2013 Catalyst Pharmaceutical Partners, Inc.

Randomization

N=30, 1:1

Dose Taper

Last Patient

~March 2014

Firdapse

U.S. Phase III Clinical Trial

Firdapse™

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

Assessments

Screening

Efficacy/Eligibility

Assessments and Dose

Adjustments

Efficacy

Assessments

on Day 1

Efficacy Assessments

on Days 8 and 14

Safety Assessments

and Dose

Adjustments

1 : Quantitative Myasthenia Gravis (QMG)

2 : Timed 25 Foot Walk

3 : Compound Muscle Action Potential

Not required for NDA

filing and approval

Page 14

2013 Catalyst Pharmaceutical Partners, Inc.

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 diagnosis from genetic screening for one or more

of the 14 known genetic defects

Myasthenia Gravis (MG)

–

Prevalence of ~60,000 patients in the U.S.

•

Potential to treat a few thousand refractory patients with Firdapse

–

First-line therapy is Mestinon (pyridostigmine), an ACh inhibitor

approved before 1982 that is not promoted

Firdapse

Expansion Opportunities

Page 15

2013 Catalyst Pharmaceutical Partners, Inc.

Phase III Clinical Trial

Phase III Safety Extension

Clinical Safety Studies

Pre-Clinical Studies

Prepare NDA

FDA Review

File NDA

Estimated FDA

Approval

Commercial

Launch

Phase III Top-Line

Results

Firdapse

Regulatory

Pathway

2013

2014

2015

2016

Page 16

2013 Catalyst Pharmaceutical Partners, Inc.

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 of 20 sales representatives

Orphan drug pricing

Patient-assistance program

Registry support patients

Product education through KOLs

Expansion to new indications

Firdapse

Commercialization Strategy

Beyond Firdapse

: CPP-115

Next Generation GABA-AT Inhibitor

Page 18

2013 Catalyst Pharmaceutical Partners, Inc.

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 patents to a new

class of inhibitors

–

Protection through 2028 with patent extensions

allowed under Patent Term Restoration Act

Filed PCT application seeking to protect CPP-

115 in ex-U.S. markets

CPP-115 Innovation

Page 19

2013 Catalyst Pharmaceutical Partners, Inc.

CPP-115 Targeted Indications

Proven

Indications

CPS

Potential New

Indications

Tourette’s

PTSD

Movement

Disorders

Infantile spasms and complex

partial seizures are proven

indications for inhibition of

GABA-AT. CPP-115 could be a

safer and more effective

alternative.

HypoGABAergic signaling

pathways are known to play a

role in these conditions.

Page 20

2013 Catalyst Pharmaceutical Partners, Inc.

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

For infantile spasms in Multiple Hit Model, and dose shown to inhibit GABA-AT in other studies

CPP-115, at 20 times its effective dose, is safer than vigabatrin at

its effective

dose

CPP-115, at its effective dose, will likely be even safer

–

Potentially no Visual Field Defect (VFD) risk

CPP-115 Superior Visual Safety

Effective

Dose

(Rats)

Vision

Study

Dose

Vision

Safety

Margin

45 Day Retinal

Function Loss

(ERG)

90 Day Retinal

Function Loss

(ERG)

Vigabatrin

300 mg/kg

200 mg/kg

~30-60%

~45-60%

CPP-115

mg/kg

20 mg/kg

> 20

~5-30%

~10-35%

Page 21

2013 Catalyst Pharmaceutical Partners, Inc.

2,000

4,000

6,000

U.S.

Europe

Incidence

Prevalence

Infantile spasms, or West Syndrome, is a

catastrophic form of epilepsy for infants

Affects 10K –

20K infants worldwide, with

nearly one-half of them in the U.S. and

Europe

60-70% of patients have underlying

disorder

Leading

therapies

are

Acthar

Gel

and

Sabril

; generate ~$100M in U.S. sales

–

In spite of significant side effects

CPP-115 has U.S. and EU orphan drug

designations

CPP-115 will be a new first-line therapy,

as well as for non-responders to existing

therapies

Opportunity valuation

–

Lundbeck

paid

~$300M

for

Sabril

rights

–

Oppenheimer

values

Questcor’s

Acthar

Gel infantile spasm franchise at ~$300M

Infantile Spasms Opportunity

Infantile Spasms Epidemiology (2010)

310M

Source: Epilepsia 2010; Population Reference Bureau 2010;

Company Reports; Catalyst Estimates

U.S. Infantile Spasms Sales (2011E)

Sales ($M)

739M

Population:

$70

$80

Acthar Gel

Sabril

Page 22

2013 Catalyst Pharmaceutical Partners, Inc.

“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

1/100th

the

dose

vigabatrin,

with

better

tolerance

than

vigabatrin

–

CPP-115 more effective than vigabatrin

•

Magnitude and duration of seizure reduction greater than

vigabatrin

•

CPP-115 causes no sedation in contrast to vigabatrin which

causes severe sedation at therapeutic doses

Briggs SW, Ono T, Moshé

SL, Galanopoulou AS (2011): presented at the American Epilepsy Society Meeting

CPP-115 Infantile Spasms Screening

(December 2011)

Page 23

2013 Catalyst Pharmaceutical Partners, Inc.

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 indication

–

Agreed on phase II design

•

Escalating dose study, N=25-30 infants

Utilizing experts Jack Pellock, MD and Don Shields, MD as consultants

–

Widely respected KOLs for infantile spasms

–

Accompanied Catalyst to FDA meeting

Phase 1 studies (supports any indication)

–

Phase 1 SAD study completed Q2 2012

–

Phase 1 MAD study designed (includes MRI efficacy biomarker)

Phase 2 enabling toxicology studies are needed

Will seek additional development funding

–

Potential partners

–

NIH

CPP-115 Development Strategy

Page 24

2013 Catalyst Pharmaceutical Partners, Inc.

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-115

•

CPP-115 and CPP-109 have same mechanism of action

•

Extended duration use in man for CPP-109 allowed with frequent vision testing

•

Will use CPP-109 until CPP-115 is developed sufficiently to support use in

phase 2 studies

Indications for which predictive animal models exist

–

Dyskinesia in Parkinson’s Disease

–

Multiple Sclerosis

CPP-115: Other Potential Indications

Page 25

2013 Catalyst Pharmaceutical Partners, Inc.

Acquired

Firdapse

phase

III

asset,

for development and commercialization

Completed $5,000,000 strategic

investment by BioMarin

Initiated Phase I/II study for Tourette’s

Disorder

Met with FDA to define development plan

for CPP-115 to treat infantile spasms

Granted orphan medicinal product

designation in EU for CPP-115 for

treatment of West Syndrome (infantile

spasms)

Reported CPP-115 Phase I(a) study

results

Filed U.S. provisional patent application

for GABA-AT inhibitor use in treatment of

Tourette Syndrome

Completed common stock public offering

Q1 2013

–

Firdapse

DMC

meeting

results

Q4 2013

–

Complete

enrollment

Firdapse

phase III clinical trial

–

Top-line results from Tourette’s

Disorder study

Q2 2014

–

Top-line

results

from

Firdapse

phase III clinical trial

Q1 2015

–

File

Firdapse

NDA

Catalyst Milestones

Recent Milestones

Expected Milestones

Page 26

2013 Catalyst Pharmaceutical Partners, Inc.

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

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