UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(d) OF
THE SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of Earliest Event Reported): February 11, 2013
CATALYST PHARMACEUTICAL PARTNERS, INC.
(Exact Name Of Registrant As Specified In Its Charter)
Delaware | 001-33057 | 76-0837053 | ||
(State or other jurisdiction of incorporation) |
(Commission File Number) |
(I.R.S. Employer Identification No.) | ||
355 Alhambra Circle Suite 1500 Coral Gables, Florida |
33134 | |||
(Address of principal executive offices) | (Zip Code) |
Registrants telephone number, including area code: (305) 529-2522
Not Applicable
Former Name or Former address, if changed since last report
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
¨ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
¨ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
¨ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR240.14d-2(b)) |
¨ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Item 7.01 | Regulation FD Disclosure |
On February 11, 2013, the Company posted on its website the presentation materials from the Companys presentation at the 15th Annual BIO CEO & Investor Conference, held at the Waldorf Astoria Hotel in New York City, New York. The presentation is furnished as Exhibit 99.1 to this Form 8-K and is incorporated herein by reference.
The information in Item 7.01 of this Current Report on Form 8-K, including the presentation attached as Exhibit 99.1 hereto, shall not be deemed filed for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the Exchange Act), or otherwise subject to the liabilities under that Section. The information in Item 7.01 of this Current Report shall not be incorporated by reference into any filing or other document pursuant to the Securities Act of 1933, as amended, or the Exchange Act except as shall be expressly set forth by specific reference in such filing or document.
Item 8.01 | Other Events |
On February 11, 2013, the Company issued a press release updating the market on the status of the research and development pipeline for its product candidates Firdapse, CPP-115 and CPP-109. The press release is attached to this Current Report on Form 8-K as Exhibit 99.2 and is incorporated herein by reference.
Item 9.01 | Financial Statements and Exhibits. |
(c) | Exhibits |
99.1 | Information posted on Companys website on February 11, 2013 | |
99.2 | Press Release issued by the Company on February 11, 2013 |
2
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
Catalyst Pharmaceutical Partners, Inc. | ||
By: | /s/ Alicia Grande | |
Alicia Grande | ||
Vice President, Treasurer and CFO |
Dated: February 11, 2013
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Exhibit 99.1 |
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Copyright
©
2013 Catalyst Pharmaceutical Partners, Inc.
This 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 (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
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, 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 |
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©
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 Tourettes 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 |
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©
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
Q2
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 |
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2013 Catalyst Pharmaceutical Partners, Inc.
Announced strategic alliance with BioMarin (in October) for
the
development
of
Firdapse
TM
North
American
license
for
Firdapse
TM
to
treat
neuromuscular diseases including LEMS
Strategic fit with our other orphan drug programs
CPP-115 for Infantile Spasms and Tourettes 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
TM
/BioMarin
Alliance |
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©
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
TM
: Lambert-Eaton Myasthenic Syndrome (LEMS)
CPP-115: Complex Partial Seizures
CPP-109: Tourettes Disorder*
CPP-115: Infantile Spasms
Potassium
Channel
Blocker
GABA-AT
Inhibitors
CPP-115: Dyskinesia
in Parkinsons
CPP-115: MS
Product Pipeline |
Firdapse
TM
Potassium Channel Blocker
Amifampridine
Phosphate
(3,4-Diaminopyridine) |
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©
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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
TM
in
Europe
(2Q10)
for
the
treatment of LEMS
EFNS
recommends
Firdapse
TM
as
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
TM
Opportunity
Summary |
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©
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
TM
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
TM
Treatment |
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©
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 |
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©
2013 Catalyst Pharmaceutical Partners, Inc.
Study
N
Dose, duration
Efficacy Outcomes
McEvoy, 1989
12
Up to 100 mg/day
3 day crossover
Significant on CMAP, disability score,
arm/leg strength
Sanders, 1993
18
Up to 100 mg/day
8 day crossover
Significant on QMG
Sanders, 2000
26
60 mg/day
6 day parallel arm
Significant
on
QMG,
CMAP
Wirtz, 2009
9
Single IV dose, 10
mg
Significant on isometric force, CMAP
Oh, 2009
8
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 |
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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
TM
U.S. Phase III Clinical Trial |
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2013 Catalyst Pharmaceutical Partners, Inc.
Randomization
N=30, 1:1
Dose Taper
Last Patient
~March 2014
Firdapse
TM
U.S. Phase III Clinical Trial
Firdapse™
Firdapse™
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 |
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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
TM
First-line therapy is Mestinon (pyridostigmine), an ACh inhibitor
approved before 1982 that is not promoted
Firdapse
TM
Expansion Opportunities |
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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
TM
Regulatory
Pathway
2013
2014
2015
2016
H1
H2
H1
H2
H1
H2
H1
H2 |
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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
TM
Commercialization Strategy |
Beyond
Firdapse TM
: CPP-115
Next Generation GABA-AT Inhibitor |
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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 |
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CPP-115 Targeted Indications
Proven
Indications
IS
CPS
Potential New
Indications
Tourettes
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. |
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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
1
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
~1
~30-60%
~45-60%
CPP-115
<
1
mg/kg
1
20 mg/kg
> 20
~5-30%
~10-35% |
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0
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
Questcors
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:
0
10
20
30
40
50
60
$70
$80
Acthar Gel
Sabril |
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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
vigabatrin
1
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
1
Briggs SW, Ono T, Moshé
SL, Galanopoulou AS (2011): presented at the American Epilepsy Society
Meeting CPP-115 Infantile Spasms Screening
(December 2011) |
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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 |
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Indications for which no predictive animal models exist
Tourettes 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 Parkinsons Disease
Multiple Sclerosis
CPP-115: Other Potential Indications |
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Acquired
Firdapse
TM
,
a
phase
III
asset,
for development and commercialization
Completed $5,000,000 strategic
investment by BioMarin
Initiated Phase I/II study for Tourettes
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
TM
DMC
meeting
results
Q4 2013
Complete
enrollment
of
Firdapse
TM
phase III clinical trial
Top-line results from Tourettes
Disorder study
Q2 2014
Top-line
results
from
Firdapse
TM
phase III clinical trial
Q1 2015
File
Firdapse
TM
NDA
Catalyst Milestones
Recent Milestones
Expected Milestones |
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©
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 |
|
Exhibit 99.2
NEWS RELEASE | FOR IMMEDIATE RELEASE | |||
For Further Information Contact: | ||||
Patrick J. McEnany | Melody Carey | |||
Catalyst Pharmaceutical Partners | Rx Communications Group | |||
Chief Executive Officer | Co-President | |||
(305) 529-2522 | (917) 322-2571 | |||
pmcenany@catalystpharma.com | mcarey@rxir.com |
Catalyst Pharmaceutical Partners Provides Update on
Research and Development Pipeline
CORAL GABLES, FL February 11, 2013 Catalyst Pharmaceutical Partners, Inc. (Nasdaq: CPRX), a specialty pharmaceutical company focused on the development and commercialization of novel prescription drugs targeting rare (orphan) neurological diseases and disorders, today provided an update on its research and development pipeline.
We are providing this information today to update our shareholders, patients, physicians, key opinion leaders and the financial community on our drug development activities. We are primarily focused on rapidly advancing the development of FirdapseTM for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), which is our lead product candidate, said Patrick J. McEnany, Chief Executive Officer of Catalyst.
Portfolio update
Firdapse
In October 2012, Catalyst acquired the North American rights to Firdapse, a proprietary form of amifampridine phosphate (3-4 diaminopyridine or 3-4 DAP), from BioMarin Pharmaceutical Inc. (BioMarin). Firdapse was approved in December 2009 by the European Medicines Agency for the treatment of Lambert-Eaton Myasthenic Syndrome (LEMS), a rare and sometimes fatal autoimmune disease characterized by muscle weakness. Firdapse has been granted orphan drug designation by the U.S. Food & Drug Administration, (FDA) for the treatment of LEMS, making the product eligible to obtain seven-year marketing exclusivity, if Catalyst is the first pharmaceutical company to obtain approval of an NDA for its formulation of amifampridine.
As part of its license agreement with BioMarin, Catalyst is taking over the sponsorship of their ongoing Phase III clinical trial evaluating amifampridine phosphate for the treatment of LEMS. The trial:
| is designed as a randomized double-blind, placebo-controlled discontinuation trial as recommended by FDA to BioMarin; |
| has a goal to enroll approximately 30 LEMS patients (approximately one third enrolled currently); |
| currently has 7 active sites (expected to be increased to approximately 25 in the near future); |
| has defined as a primary endpoint-change in muscle strength during the 2-week, double-blind discontinuation period as determined using a validated questionnaire (Quantitative Myasthenia Gravis score); and |
| has defined as a secondary endpoint-change in walking speed (timed 25-foot walk test) during the discontinuation period. |
For further details on this trial, please go to: www.clinicaltrials.gov; Search amifampridine phosphate.
With respect to the trial, Catalyst expects:
| to complete enrollment by the end of 2013; and |
| to report top-line results from the double-blind portion of this clinical trial during the second quarter of 2014. |
Assuming positive results are obtained from the trial, Catalyst hopes:
| to file an NDA for Firdapse in the first quarter of 2015; |
| to obtain approval from the FDA of such NDA by the end of 2015; and |
| to commercially launch this product sometime in the first half of 2016. |
Firdapse may also be an effective treatment for other neuromuscular orphan indications:
| Congenital Myasthenic Syndrome; and |
| Myasthenia Gravis. |
Catalyst believes Firdapse can achieve peak annual revenues from sales in the United States of approximately $100 million.
CPP-115
On August 27, 2009, Catalyst entered into a license agreement with Northwestern University (Northwestern), under which it acquired worldwide rights to commercialize new GABA aminotransferase inhibitors and derivatives of vigabatrin which were discovered and patented by Northwestern. Catalyst has designated the lead compound to be developed under this license as CPP-115. CPP-115 has been granted orphan drug designation by the FDA for the treatment of infantile spasms and orphan medicinal product designation in the European Union (EU) for Wests syndrome (a form of infantile spasms). This means this product will be eligible to obtain the seven-year and ten-year marketing exclusivities available from the FDA and the EU, respectively, if Catalyst is the first pharmaceutical company to obtain approval of an NDA/MAA for CPP-115.
Based on the results of pre-clinical studies to date, Catalyst believes CPP-115 is:
| more potent; and |
| may have fewer side effects (e.g., visual field defects, or VFDs) than vigabatrin. |
Page 2
In October 2011, a pre-IND meeting was conducted with the FDA, during which preclinical and clinical requirements were defined that would allow Catalyst to complete a development program through Phase II of CPP-115 for the treatment of infantile spasms.
During the fourth quarter of 2011, Catalyst completed its IND-enabling studies, filed an IND, and began a Phase I(a) human trial of CPP-115 to evaluate its safety. On May 22, 2012, Catalyst reported positive results from this double-blind, placebo-controlled, clinical trial evaluating the safety, tolerability and pharmacokinetic profile of CPP-115. The key findings were:
| CPP-115 was well tolerated at all six doses administered in the study; there were no significant adverse events, and no cardiovascular or respiratory events were reported in the study; and |
| CPP-115 was rapidly absorbed (time to peak blood concentration was about 30 minutes). |
Subject to the availability of funding, Catalyst hopes to begin further human clinical trials evaluating CPP-115 later in 2013. To fund such trials and studies, Catalyst intends to pursue grants from NIH and foundations. In addition, Catalyst hopes to identify a strategic partner to work with it in the development and future commercialization of CPP-115.
CPP-109
Catalyst, as a co-inventor, with scientists at New York University and the Feinstein Institute for Medical Research, recently filed a provisional patent application with the U.S. Patent and Trademark Office for the use of GABA aminotransferase inhibitors, including CPP-109 and CPP-115, in the treatment of Tourettes disorder. Catalyst also recently entered into a license agreement with NYU and the Feinstein Institute granting it worldwide rights with respect to such patent.
Catalyst is currently providing CPP-109 and financial support for a small Phase I/II trial being undertaken at Mt. Sinai School of Medicine in New York to evaluate the use of CPP-109 in treating Tourettes disorder. This is a 6-10 patient, open-label trial, from which Catalyst anticipates top line results during the fourth quarter of 2013. If the results of the study show evidence of reduced numbers of tics, Catalyst hopes to develop CPP-109 (and/or CPP-115) for this indication, subject to the availability of additional funds. The Company believes that this indication should qualify for orphan drug designation from the FDA.
Key development milestones
| Q1 2013 |
| Report Firdapse Data Monitoring Committee meeting results |
| Q4 2013 |
| Complete enrollment of Firdapse phase III clinical trial |
| Report results from Tourettes Disorder study |
| Q2 2014 |
| Report top-line results from Firdapse phase III clinical trial |
| Q1 2015 |
| File NDA for Firdapse |
Page 3
Project discontinuation
CPP-109 for addiction
For several years, Catalyst has been conducting its own clinical trials and studies, as well as supporting investigator-sponsored trials and studies, evaluating CPP-109 for the treatment of cocaine and methamphetamine addiction. However, based on the previously announced top-line results obtained from its most recent Phase II(b) trial of CPP-109 in cocaine dependent subjects, Catalysts management and Board of Directors have determined not to focus its future product development efforts on evaluating CPP-109 for the treatment of drug addictions. Catalyst is disappointed for all its stakeholders, including patients, investigators, families and advocacy groups, but believes this is the correct decision for the company under the circumstances.
About Catalyst Pharmaceutical Partners
Catalyst Pharmaceutical Partners, Inc., is a specialty pharmaceutical company 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 Tourettes disorder. Catalysts lead candidate, FirdapseTM for the treatment of LEMS, is currently undergoing testing in a global, multi-center, pivotal phase III trial. Catalyst is also developing a potentially safer and more potent vigabatrin analog (designated CPP-115 by Catalyst) to treat infantile spasms, and epilepsy, as well as other neurological conditions associated with reduced GABAergic signaling, like Tourettes disorder, post-traumatic stress disorder, and movement disorders associated with the treatment of Parkinsons Disease.
Forward-Looking Statements
This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks and uncertainties, which may cause the Companys actual results in future periods to differ materially from forecasted results. A number of factors, including whether the Phase III trial of Firdapse will be completed on the timeline described above and will be successful, whether the Company will, even if the Phase III trial is successful, be permitted to file an NDA for Firdapse, whether Catalyst will obtain funding to support future development efforts of CPP-115 and/or CPP-109, whether any of the Companys product candidates will ever be approved for commercialization and the timing of any such approvals, the level of potential sales that can be achieved by any of the Companys product candidates that are approved for commercialization, and those factors described in the Companys filings with the U.S. Securities and Exchange Commission (SEC), could adversely affect the Company. Copies of the Companys filings with the SEC are available from the SEC, may be found on the Companys website or may be obtained upon request from the Company. The Company does not undertake any obligation to update the information contained herein, which speaks only as of this date.
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