Chelsea Therapeutics International Ltd
Chelsea Therapeutics International — Investor Presentation (October 2011)
30 slides from Chelsea Therapeutics International's investor presentation, Oct 19, 2011
Slide 1: Chelsea Therapeutics Presentation, October 2011
Exhibit 99.1 Exhibit October © 2004-2011 99.1 2011 Chelsea Therapeutics, Inc.
Slide 2: Forward-Looking Statement
Forward-Looking appropriateness This materially. clinical Exchange 2NASDAQ: presentation trials, Commission. These CHTP intellectual orrisks isStatement contains suitability being andproperty provided forward-looking uncertainties of anyrisks, for investment. informational include andstatements the
Slide 3: Chelsea Overview
Chelsea Overview Two Norepinephrine Northeratm Neurogenic Intradialytic (NOH)* Freezing Other Non-Metabolized CH-4051 Rheumatoid Psoriasis *Approved 3NASDAQ: Major norepinephrine Therapeutics of Crohn’s &CHTP Platforms indication Gait* (droxidopa) Orthostatic others Arthritis Hypotension* Replacemen
Slide 4: Robust Clinical Pipeline
RobustQ2 PRECLINICAL Northera: NOH Study Intradialytic Fibromyalgia Metabolically Rheumatoid Other CH-4051: Data: 4NASDAQ: Q3 Q1 Autoimmune 302: 306A: Clinical 12 09 Norepinephrine Study PD, CHTP Arthritis Hypotension Data: PD Inert MSA, Pipeline PHASE 202 101 Study Year Q3 Antifolates Diseases CH-1
Slide 5: Northera: Oral Norepinephrine Replacement Therapy
Northera™: Oral Within •Well 5NASDAQ: Replenishes Unique Only Marketed Orthostatic Freezing Hypotension “Prodrug” Documented Chronic Autonomic Mechanism ofEx CHTP Norepinephrine Hypotension Gait Diminished associated US Oral of in Norepinephrine: Safety (Japan) Nervous Therapy Parkinson’s of Action
Slide 6: Neurogenic Orthostatic Hypotension (NOH)
Neurogenic Caused •Poor 6NASDAQ: 500–1000 Reduced Some perfusion patients by ability diminished CHTP mL Orthostatic toof cannot to the blood push brain synthesis stand Hypotension shifts blood leads unaided toback and/or to thedizziness, (NOH): lower to forrelease the more heart body Sudden, lighthe
Slide 7: The Symptomatic NOH Patient Prevalence
The Symptomatic Parkinson’s Multiple •Pure 7NASDAQ: Prevalence NOH Autonomic Systems Patient CHTP Disease ofNOH disease: NOH: Failure Atrophy affects 18% 75% 100% 120patients 5-15 10-30 tocases cases 30% per 100,000 per per with 100,000 100,000 primary population population autonomic failure, a grou
Slide 8: NOH: Therapeutic Market Landscape
NOH: •Midodrine 8NASDAQ: Alpha NeverCurrent Provides Black piloerection Poor $55-60M patient penetration box agonist satisfied constant/undifferentiated (ProAmatine®)…alpha peak CHTP warning (goose persistence Therapeutic side sales FDA’s ofeffects for bumps, PD (priced requirement supine Market and
Slide 9: Northera: Attractive Commercial Opportunity
Northera: Attractive Limited Personal •Non-Personal 9NASDAQ: Accounts Chronic Price: Compliance: 10% ~4,000 No Promotional Medical Journal Direct Sampling Competing of$30/day Sales Mail Physicians Promotion: Advertising Midodrine Treatment US Science US for Program Programs CHTP Educational Commerci
Slide 10: Northera NDA Status
Northera •New •Study NASDAQ: 10 Anticipating Fast Drug StudyTrack 301: 302: 304: 305: 303: NDA: Pivotal Supporting CHTP Application: Status 24Priority Long-Term HR Neurogenic Proof Blood Review, Phase Filed Safety ofPressure Efficacy Orthostatic III September Estimated Extension Efficacy Monitoring
Slide 11: Study 301: Northera Favors Placebo Symptoms of NOH
Study 301: NASDAQ: Favors 11 Northera Placebo Symptoms CHTP of NOH in PD, MSA & PAF
Slide 12: Study 301: OHQ Composite Statistical Significance
Study In NASDAQ: 12 Addition 301: OHQ CHTP to Statistical CompositeSignificance of Single Endpoint, FDA Examines the Cumulative Distribution Function Between Arms to Discern Treatment Effect
Slide 13: Study 301: Change in Dizziness and Standing SBP
Study 301: Change NASDAQ: 13 Dizziness CHTP in and OHSA Standing Standing #1SBP (Dizziness) SBP
Slide 14: Pooled Meta-Analysis of 301 & 302 OHQ Composite
Pooled 301 CHTP Meta-Analysis NASDAQ: 14 &302 of 301 Analysis: & 302OHQ Data composite Support Treatment Effect Demonstrated in Study 301
Slide 15: Pooled 301 & 302 Analysis: Dizziness and Standing SBP
Pooled 301 CHTP NASDAQ: 15 &302 Analysis: Dizziness and Standing SBP
Slide 16: Northera: Most Common Adverse Events and Safety Profile
Exhibit 99.1 Northera: Most Efficacy •Clinical Placebo System Preferred pts) Adverse Nervous Headache Dizziness Loss General administration Fatigue Injury, procedural Fall Infections Urinary NASDAQ: 16 Associated Headache: Falls: Safety 1% Infrequent ECGnts 9of incidence common (6.8) study poisoning
Slide 17: Expanding Label: Falls Injuries in Parkinson's Disease
Expanding Injuries #1 2nd •Estimated NASDAQ: 17 leading 1/3 NOH reason most of (symptomatic PD sustained cause common for 20% the CHTP related patients Label of in death patients reason nursing falls in & being Parkinson’s in non-symptomatic) are for with the home admitted aPD elderly common Parkins
Slide 18: Study 306A: Total Falls
Studyfalls Northera Total Number NASDAQ: 18 306A: of (n=24) =Days Falls CHTP 197 79 Cumulative Placebo of Therapy (n=27) Falls
Slide 19: Study 306A: Reduction in Falls per Patient/Week
Study •Falls 2.3 Mean NASDAQ: 19 Allows Detailed Fold per Falls 306A: or for patient/week discussion 60% CHTP / distribution Patient Reducing Reduction / of Week pre-specified Falls metric of falls inper Falls and to patient/week be analysis per asstandardized efficacy Patient/Week in SAP, criteria
Slide 20: Study 306A: Change in Hoehn & Yahr Scores
Study •Commonly Baseline Change NASDAQ: 20 Heavily 306A: intoweighted HY CHTP End-of-Treatment used Improving Score 5-point toward Hoehn rating postural scale & Yahr instability characterizing Scores as thethe primary progression index Parkinson’s of disease severity disease
Slide 21: Study 306A: Change in UPDRS
StudyI: Assessing Change Part NASDAQ: 21 I Part 306A: Non-Motor in the II Component CHTP Part Response severity III Experiences Part of Score inIV motor Parkinson” of and Daily non-motor s Disease Living symptoms Part II: Motor of Parkinson’s Experiencesdisease of Daily Living Part III: Motor Examin
Slide 22: Study 306A: Efficacy in Repeat Fallers
Study 306A: >40% NASDAQ: of patients CHTP Efficacy in Study in Repeat 306A Fallers experienced 2 or more falls in 10 weeks
Slide 23: Northera Preliminary Data and Study 306B
Northera Preliminary Study Data: •Future NASDAQ: 23 Anticipate Target To Preliminary, Data FDA Powering beQ2 306A: 306B: safety Confirmed Studies initiated Enrollment: sNDA: 2012 for CHTP top Data Supporting Phase monitoring unblinded to approximately line post-approval, Reduction Support Reported S
Slide 24: Droxidopa Future Growth Opportunities
Norepinephrine Intradialytic Fibromyalgia Adult 15-25% Droxidopa Chronic 2nd 4.4% Debilitating CHTP: Investigator: Completed Data: Future NASDAQ: 24 Most of YE 2011 Attention Growth Phase of US Fatigue Pain 11 Common all +CHTP adults Fatigue Hypotension Phase Carbidopa Disorder II hemodialysis Oppor
Slide 25: Non-Metabolized Novel Antifolates
Non-Metabolized Novel •Methotrexate NASDAQ: 25 Additional Non-metabolized Potential Limitations Nausea Kidney Undergoes Est. 1/3 Antifolate and ofFirst CHTP MTX High significant are Diarrhea Liver (MTX) Line AEs with Antifolates Potential Patients Oral Toxicity and remains and Blockbuster metabolism
Slide 26: Developing a Better, Safer Methotrexate
Developing MTX Polyglutamylated 7-OH CH-4051 (no NASDAQ: 26 change!) MTXCHTP a Better, MTX Safer and/or Methotrexate
Slide 27: CH-4051: Phase I Proof-of-Concept
CH-4051: Phase 8-fold Superior Key Demonstrated 20-fold Improved 2-fold NASDAQ: 27 preclinical findings II Improvement Higher Proof-of-Concept Efficacy: Safety/toxicity: Tolerability pharmacokinetics: Greater CHTP from Comparable safety Doses findings Efficacy, Superior phase in : Than inBioavailabi
Slide 28: CH-4051 Phase II Study Design in RA
CH-4051 •Staggered Un-blinded Randomization Screening N= Baseline 0.3 3.0 20 Hybrid 2Up + 412 MTX NASDAQ: 28 Enroll Open weeks Follow mg/wk weeks to mg 50Washout 2N= All ACR CH-4051/daily weeks 10 Visit Up Phase start: MTX 50 Arms Patients CHTP Interim Score N=Double-Blind Visit II: +After 50FOLATE
Slide 29: Financial Capitalization Summary
Financial Capitalization Common Fully Cash Warrants March NASDAQ: 29 and Diluted 2012 3,241,550 Expiring Short-Term Summary Stock CHTP –70,786,680 (as Exercise 61,847,700 of (cash Options 7/26/11) Investments price exercise): 5,697,430 $5.66—Anticipated ( as of 6/30/11)$4.5 $67.1 million million
Slide 30: Looking Ahead: Upcoming Milestones
Looking •Operational Upcoming Initiate Interim File Un-blinded Anticipated Top-line NASDAQ: 30 Advance Drive Execute Northera CH-4051 Northera Analysis: Ahead Results Strategic, CH-4051 Milestones CHTP Interim US Focus NDA: Approval Droxidopa Northera Registration PII: NOHEfficacy Cash NOH inRA RA S
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