A world-class allergy & asthma specialty biopharma business - Corporate presentation - Circassia
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Corporate presentation July 2015 A world-class allergy & asthma specialty biopharma business
Disclaimer
Neither this presentation nor any verbal communication shall constitute, or form part of, any offer, invitation or
inducement to any person to underwrite, subscribe for, or otherwise acquire or dispose of, any shares or other
securities in Circassia Pharmaceuticals plc (“Circassia”).
Forward-looking statements
This presentation and information communicated verbally to you may contain certain projections and other
forward-looking statements with respect to the financial condition, results of operations, businesses and
prospects of Circassia. The use of terms such as “may”, “will”, “should”, “expect”, “anticipate”, “project”,
“estimate”, “intend”, “continue”, “target” or “believe” and similar expressions (or the negatives thereof) are
generally intended to identify forward-looking statements. These statements are based on current expectations
and involve risk and uncertainty because they relate to events and depend upon circumstances that may or
may not occur in the future. There are a number of factors which could cause actual results or developments to
differ materially from those expressed or implied by these forward-looking statements. Any of the assumptions
underlying these forward-looking statements could prove inaccurate or incorrect and therefore any results
contemplated in the forward-looking statements may not actually be achieved. Nothing contained in this
presentation or communicated verbally should be construed as a profit forecast or profit estimate. Investors or
other recipients are cautioned not to place undue reliance on any forward-looking statements contained herein.
Circassia undertakes no obligation to update or revise (publicly or otherwise) any forward-looking statement,
whether as a result of new information, future events or other circumstances.
2
Circassia overview
Building an allergy & asthma champion
Strong broad-based specialty biopharma business
– 2 currently marketed products sold to allergy / asthma specialists
– 12 products in development for allergy, asthma and COPD
– Lead allergy candidate in phase III (data expected H1 2016)
– Lead asthma product filed Q3 2014
– Potential for 8 product launches by end 2021
– Strong IP across portfolio
Commercial infrastructure focused on allergy / asthma specialists
– Focused commercialization strategy: direct to specialists in key markets and partner in primary care
– Direct sales currently targeting key customers in US and Germany
– Market access and payor expertise in place
– Scalable infrastructure to optimize launch of lead allergy product and broader portfolio
Strong growth platform
– Immediate revenues, near-term pipeline and high-value specialty products
– Novel short-course immunotherapies have potential to revolutionize multi-$bn allergy market
– Fully funded to deliver pipeline (estimated cash1 ~£230m June 2015)
1 Cash, cash equivalents and short-term bank deposits
3
Circassia’s strategy
Building a self-sustaining specialty biopharma company
Deliver the pipeline
Market novel products
Independently in N America
and major EU markets
Partnerships elsewhere
Build broad and
balanced portfolio
4Strong, deep and balanced pipeline
Product 2015 2016 2017 2018 2019 2020 2021
NIOX MINO Launched
NIOX VERO Launched
PSX1001* UK1 UK1 EU1
Flixotide® substitute approval launch launch
PX1439* UK UK EU
Serevent® substitute filing launch launch
PSX2005 UK UK EU US US
Seretide® substitute filing launch launch filing launc
Ph III EU / US EU / US
Cat-SPIRE data filing launch
PSX1050* Partnered – timelines
Flovent® substitute not disclosed publicly
Ph III EU / US EU / US
Grass-SPIRE data filing launch
House Dust Mite-SPIRE Ph II Ph III EU / US EU / US
data data filing launch
PSX3001 PK Ph III US US
Novel triple presentation study data filing launc
Ph II Ph III US US
Ragweed-SPIRE data data filing launc
PSX1002 Ph II Ph III US
Novel LAMA formulation data data filing
Pipeline does not show earlier-stage programmes: Birch-SPIRE, Japanese cedar-SPIRE, Alternaria-SPIRE and home use NIOX device
Fully funded to deliver pipeline
*Partnered
1Approval / launch reflects estimates of MHRA review and decentralized procedure timelines only
All timelines are forward-looking projections that involve risks and uncertainties – please see the disclaimer on slide 2 for further details 5Focused commercialization strategy
Targeting direct sales in US & major EU markets
Circassia US & EU commercial operations
Corporate administration
(Finance, HR, IT)
KOLs
Business analytics Direct
sales
Compliance (legal, regulatory)
force
Market access
Medical affairs
Marketing Allergists Pulmos
Sales
Supply chain / distribution
Top prescribers primary care
Partner
Partner elsewhere sales
Primary care
force
Outside US and Europe
Primary care
61
Marketed products
2
Asthma / COPD pipeline
3
Novel allergy immunotherapies
4
Summary
7Products marketed in over 40 countries
Direct sales infrastructure in US and EU’s largest allergy market
Direct sales targeting allergy /
asthma specialists in key markets
Opportunity to expand in EU
Broad international distribution
network
Novel products Direct sales Distributors
8Leadership in FeNO asthma diagnosis & management
Meeting key clinical need in major therapeutic market
Only point-of-care device available across major markets to measure FeNO to assist diagnosis
and control of airways disease
– Strong IP with 72 granted patents in US, EU & Japan with protection currently to 2026
Asthma is one of largest healthcare burdens
– 25 million asthmatics in US
– 14 million physician office / 1.8 million ER visits in US with asthma as primary diagnosis
– >$50bn medical cost of asthma in US in 2007
Clinical evidence shows FeNO measurement improves asthma management
– Improves diagnosis
– Improves determination of inhaled steroid responsiveness
– Improves control through tailoring inhaled steroid use
– Improves monitoring of treatment compliance
– Potential to reduce exacerbations
Extensive big pharma use in asthma clinical studies
– Validates the importance of FeNO in asthma
– Establishes FeNO in market and trains physicians in use of products
9Only point-of-care product available across major markets
Roll-out of next generation product in US and Japan
Transition to next
generation product
provides major
opportunity
NIOX® MINO® NIOX® VERO®
EU 2004, US 2008, EU 2013, US 2014,
China 2010, Japan 2013 Japan 2015
For ages 4+ in EU; 7+ in US For ages 4+ EU; 7+ in US
10 second test; 90 second result Fully portable; enhanced screen interface
Monitor lasts 3 years or 3,000 tests 6 and 10 second test; 60 second result
Limited portability Monitor lasts 5 years or 15,000 tests
Improved margin on consumables
10Endorsement from key allergy / asthma organizations
Included in ATS treatment guidelines and NICE recommendation
11Significant market opportunity
Exemplifies Circassia’s commercialization strategy
US specialist opportunity Robust global
revenue growth
$190m Direct ‒ 2014 revenues
sales KOLs $24.3m1
force
‒ 18% CAGR over
last 5 years
‒ Strong forecast
Allergists Pulmos growth
US primary care opportunity
Top prescribers primary care
Partner
sales
$610m force Primary care
Long-term upside potential from FeNO home use device currently in planning
1 Average FX rate for year ended 31 December 2014 SEK/$ = 0.1462 12Potential to accelerate growth
Foundations in place
2015 positioned for growth
Significant progress in establishing new market category and changing existing paradigm
FeNO accepted by KOLs and specialists
Many major guidelines include FeNO
Scientific evidence / publications support use of FeNO
Reimbursement established (64% US coverage; targeting 75% by 2016)
Next generation VERO® device offers significant improvements over predecessor
VERO® US and Japanese launches H1 2015
Chinese VERO® approval expected shortly
131
Marketed products
2
Asthma / COPD pipeline
3
Novel allergy immunotherapies
4
Summary
14Near-term pipeline & longer-term novel formulations
Device types Significant pricing potential
‒ 73.5% of pre-entry brand
Focus on pMDI market segment price for first to market
generic in US during
exclusivity1
‒ 47.8% of pre-entry brand
Generic directly substitutable products pMDI DPI price for only on market
generic in US1
– No requirement for significant commercial
infrastructure
– Limited development
– Rapid route to market; near-term revenue
Direct
– Challenging to achieve for respiratory products KOLs
sales
force
– Non-substitutable competitors require promotion
Novel combinations / products Allergists Pulmos
– Longer more extensive development
Top prescribers primary care
– Majority of market in primary care Partner
sales
– Circassia to target allergy / asthma specialists Primary care
force
– Partner for phase III and targeting primary care
1 Bureau of Economics, Federal Trade Commission, Working Paper No 317. The effect of generic drug competition on generic drug prices during the Hatch-Waxman 180-day exclusivity period. April 2013.
15Novel technology provides sophisticated API control
Technology #1
Technology controls Significant potential benefits
‒ Size Directly substitutable products
‒ Shape ‒ Potential first to market with
unique combination of therapeutic
‒ Aerodynamics equivalence, all strengths, similar
‒ Surface properties device, same formulation & cost-
effective
Engineered API ‒ Manufacturability
Novel products
‒ Product stability
‒ Optimized combinations and
‒ Product performance
novel formulations
Technology #2
Established at commercial scale in cGMP compliant FDA-approved facilities
Broad IP protecting apparatus to 2022 in US & 2019 in EU; patents pending will extend
product and process protection to 2030 in US and 2028 in EU
16Lead product filed in EU
Collaboration with Mylan PSX1001
Flixotide®
PSX1050
Flovent®
substitute (EU) substitute (US)
EU filing Mylan collaboration
Product candidate targeting substitution for
GSK’s Flixotide® pMDI (Flovent® pMDI in US) Technology validation by leading company
Filing validated and under assessment Q3 2014 Certain marketing rights retained in specific EU
territories
Filing review under EU orally inhaled products
Full rights retained in China, South America,
guidelines that allow approval based on in vitro
Middle East and Africa
equivalence data only
Mylan has marketing rights in agreement territory1
Decentralised procedure - MHRA reviewing file
FDA guidelines require PK and PD studies in US
Decision on first approval expected 2H 2015
Estimated $930m originator sales ($680m in US; $250m ex-US)
1 USA, Canada, Australia and New Zealand, India, Japan, Europe (including the EU and EFTA states (Iceland, Liechtenstein, Norway and Switzerland)), Turkey, Russia and CIS)
Originator sales estimate based on GSK Annual Reports 2011 and 2014 and selected IMS data 2011 and 2012
17Strong pipeline of follow-up products
PX1439 PSX2005
Serevent® Seretide®
substitute substitute
Serevent® pMDI substitute
– First registration batches in place
– UK filing anticipated H2 2015
– Estimated originator sales $60m1
– Partnered in UK / Ireland
Seretide® pMDI substitute
– Global rights retained
– Initial registration batches in place
– UK filing anticipated H1 2016
– Originator sales estimated $1.8bn1
Targeting nearly $2bn market
1 Originator sales estimates based on GSK Annual Reports 2011 and 2014 and selected IMS data 2011 and 2012
18Longer-term high value novel products
PSX1002
Novel LAMA
formulation
Optimized glycopyrronium bromide formulation
– Potential Spiriva® competitor targeting predicted >$3bn1 opportunity
– Compelling phase IIa results
Significant efficacy vs placebo; once daily dosing potential
0.20
0.15
0.10
Mean FEV1
0.05
0.00
-0.05
-0.10
-0.15
0 5 10 15 20 25 30
Time (h)
PSX1002 12.5μg PSX1002 25μg PSX1002 50μg PSX1002 100μg Placebo
1 Respiratory Market 2025: Taking A Deep Breath And A Deep Dive – Jefferies 2013 Equity Research
19Significant potential benefits for combination
products PSX3001
Novel triple
presentation
Conventional formulation Proprietary multi-component particles
results in random mixture provide consistency
ICS/LABA
(eg Seretide®)
DPI
MCPTM
LAMA
(eg Spiriva®)
Proprietary MCPTM technology enables constant ratio of multiple APIs in each particle
Provides dose on label at individual particle level
Potential for dual & triple combinations (ICS/LABA, LABA/LAMA, ICS/LABA/LAMA)1
Extensive testing already undertaken
Triple combination ICS + LABA/LAMA1 targeting emerging ~$8bn2 market opportunity expected
to enter clinic H2 2015
1 Inhaled corticosteroid (ICS) / long-acting beta agonist (LABA) / long-acting muscarinic antagonist (LAMA)
2 Respiratory Market 2025: Taking A Deep Breath And A Deep Dive – Jefferies 2013 Equity Research
20Approach exploits market dynamics
Opportunity to capture modest share of significant markets
Global key inhaled maintenance respiratory market1 (excludes ICS monotherapy market: >US$2bn in 2014)
25
Monotherapy ICS PSX1001 / PSX 1050 targeting
Flixotide® / Flovent® substitution
20
Global Sales (US$bn)
15 Triple fixed dose combination PSX3001
Monotherapy LAMA PSX1002
10
Monotherapy LABA PSX1439 targeting
5
Serevent® substituion
LABA/ICS PSX2005 targeting Seretide®
substitution
0
2008A 2010A 2012A 2014E 2016E 2018E 2020E 2022E 2024E
LABA/ICS LABA LAMA and SAMA LABA/LAMA LABA/LAMA/ICS
Potential near term approvals with high value follow up products
1 Respiratory Market 2025: Taking A Deep Breath And A Deep Dive – Jefferies 2013 Equity Research 211
Marketed products
2
Asthma / COPD pipeline
3
Novel allergy immunotherapies
4
Summary
22Allergic rhinitis is a global healthcare problem
Affects 10-20% of global population
Allergic diseases affect over 1 billion people worldwide 3
Allergic rhinitis is the world’s most prevalent chronic non-communicable disease3
Allergy is medical condition with greatest impact on work productivity in US4
Allergy is a precursor of asthma; treatment with immunotherapy halts “allergic march”
USA Europe
Skin prick test positive Skin prick test positive
Rank Allergen Rank Allergen
(% Popln) (million)1 (% Popln) (million)2
1 House dust mite 28 86 1 House dust mite 22 82
2 Perennial rye 27 84 2 Grass pollen 17 63
3 Short ragweed 26 82 3 Cat 8-10 30-37
4 Cockroach 26 82 4 Birch pollen 6 22
5 Bermuda grass 18 57 5 Mould 4 15
6 Cat 17 53 6 Olive pollen 3 11
Source: Arbes et al. J Allergy Clin Immunol. 2005 Aug;116(2):377-83. Source: Bousquet et al. Allergy. 2007: 62: 301-9
Targeted by Circassia Future potential targets
Immunotherapy is the only way to treat the underlying disease
1 US Census Bureau, 2012 3 EAACI Global Atlas of Allergy 2014
2 World Bank, 2012 4 Gemson & Eng, August 2004
23Moderate to severe allergy is inadequately addressed
by current therapies
Allergen avoidance: not feasible in majority of cases
Symptomatic drugs: (anti-histamines, nasal corticosteroids etc) limited efficacy
– Prescription market estimated at approximately $7bn1
Whole allergen Subcutaneous Sublingual
immunotherapy Immunotherapy (SCIT) Immunotherapy (SLIT)
Targets cause of allergy Allergen injected Allergen under the tongue
leading to tolerance of Lengthy treatment 3 - 5yrs Lengthy treatment 1 - 3yrs
allergens Poor patient adherence Low adherence (7% complete 3yrs2)
Non-standardized dosing US requires EpiPen prescription
Reduces “allergic march” to
asthma High frequency of side effects incl High frequency of side effects incl
potential for anaphylaxis potential for anaphylaxis
Total 5 year cost: ~$3,600 – $6,000* Total 1 year cost: $1,400 - $2,700**
Total 3 year cost: ~$9,000**
Majority of allergic rhinitis patients consulting a GP have moderate to severe symptoms3
1 Bloomberg * Based on Circassia’s estimates
2 J Allergy Clin Immunol. 2013 Aug;132(2):353-60.e2. doi: 10.1016/j.jaci.2013.03.013. Epub 2013 May ** Based on Merck/ALK and Stallergenes published US prices for SLIT treatments
3 Bousquet et al. J Allergy Clin Immunol. 2006 Jan;117(1):158–62
24Proprietary ToleroMune® technology
Designed to treat underlying disease with minimal side-effects
Whole
allergen
ToleroMune® identifies T cell epitopes
– Short linear stretches of amino acids in allergen sequence
– Binds to antigen presenting cells to induce regulatory T cells
– Identified from blood of allergic individuals
SPIREs – Synthetic Peptide Immuno-Regulatory Epitopes
Short treatment designed to provide efficacy without the safety issues
– Regulatory T cells down-regulate allergic response T cell
epitopes
– Lack of B cell epitopes avoids cross-linking of mast cells eliminating selected
early response / no need to dose escalate
Final product is a
– Synthetic manufacture – no extraction from whole allergens room temperature
stable, lyophilized
Broadly applicable across range of allergies vial containing a
mix of 7 peptides
– Allergens already identified; no research required for injection
Initial development of new SPIRE candidate takes ~18 months
Modern, synthetic, rationally-designed pharmaceuticals
25Technology validated with clinical proof-of-concept
in four programmes
Development
Next milestone Phase III data Key findings
stage
Pilot pediatric Proof-of-concept in
Phase III field study
Cat-SPIRE safety study H1’16 multiple products
(n = 1,409)
completes H2 ‘15
Phase II asthmatic Short-course treatment
Grass- Phase IIb study
study reports H2’17
SPIRE (n = 282)
H1‘15
Efficacy persists over time
House Dust Phase IIb study Fully recruit phase
Mite (HDM) - H1’19 Enhanced efficacy in more
(n = 172) IIb field study
SPIRE symptomatic subjects
Evaluate in follow-
Ragweed- Phase IIb study
up & higher-dose H1’20 Safety profile similar to
SPIRE (n = 280) placebo
study
Japanese Cedar-SPIRE, Birch-SPIRE and Alternaria-SPIRE in early stage development
26Cat-SPIRE phase IIb
Proof-of-concept
Skin prick +ve Cat:
Randomised, placebo-controlled parallel group chamber study US: 17%1 (53m)
– Commercial-scale room-temperature stable formulation EU: 8-10%2 (30-37m)
202 subjects randomised
– 2 dosing regimens and placebo
Primary objective: evaluate efficacy in cat allergic subjects
following cat allergen challenge
Subjects in chamber 3 hours per day for 4 days at baseline and at
post-treatment challenge
– Controlled levels of cat dander (similar to house that has a cat)
– Symptoms recorded every 30 minutes
Comparison of symptom scores at challenge 5 months after Exposure
chamber
starting treatment to baseline Toronto
1 Arbes et al. J Allergy Clin Immunol. 2005 Aug;116(2):377-83
2 Bousquet et al. Allergy. 2007: 62: 301-9
27Cat-SPIRE phase IIb (n=202)
Confirmed efficacy
Total Rhinoconjunctivitis Symptoms Score (“TRSS”)
Patient self-rated scores used as primary efficacy Overall TRSS improvement
measure of 2.1 vs. placebo (p = 0.05)
Scoring system required by regulators
‒ Used for approval of intranasal steroids,
antihistamines etc
Scores measured on 4-point rating scale
‒ 0: absent
‒ 1: mild, barely noticeable
‒ 2: moderate, annoying / troublesome
‒ 3: severe, incapacitating
SPIRE studies use 8 symptoms = 24-point scale;
Cat-SPIRE used sneezing & runny / blocked / itchy
nose & itchy / watery / red / sore eyes
TRSS score of 8 could be 8 “mild / barely
noticeable scores”
TRSS score of 12 could be 4 “mild / barely
noticeable” and 4 “moderate / annoying” scores
Note: Based on non-asthmatic subjects
28Cat-SPIRE phase IIb
Sustained benefit at 1 and 2 years with no additional dosing
1 year follow-up study: 2 years follow-up study:
Efficacy enhanced over time Efficacy persists at 2 years
Overall TRSS improvement
of 3.9 vs. placebo (p = 0.01) Overall TRSS improvement
of 3.9 vs. placebo (p=0.13)
Secondary endpoint: TRSS
improvement at end of day 4:
5.1 vs. placebo (p=0.02)
Tolerance persists at least 2 years without further dosing
Published: J Allergy Clin Immunol. 2013 Jan;131(1):103-9.e1-7 / Clin Exp Allergy. 2015 May;45(5):974-81. doi: 10.1111/cea.12488
29Cat-SPIRE represents therapeutic step change
More effective and more convenient
Product/Study3 Treatment Difference Active vs. Placebo
TRSS
Cat-SPIRE chamber study1 4 doses 4 weeks apart 3.9
ALK-Abelló Grazax® pivotal field study2 Daily 16 weeks before and during
1.0
(licensed in Europe) SLIT tablets season
Stallergenes Oralair® grass field study2 Daily 16 weeks before and during
1.4
(licensed in Europe) SLIT tablets season
Allergy Therapeutics Pollinex® Quattro
grass field study2 (filed Germany in
4 administrations 1 week apart 1.1
2009, not yet approved) adjuvanted
whole allergen IT
GSK fluticasone furoate perennial
Once daily for 4 weeks 0.86
rhinitis field study2 intranasal steroid
Sanofi fexofenadine cat chamber study2 180 mg 2 hours before chamber (ie
1.3
antihistamine pre-symptoms)
1 Based on the 4 x 6 nmol dose of Cat-SPIRE in CP005A
2 Source: Summary of product characteristics for each product, except i) Fexofenadine: Ann Allergy Asthma Immunol. 2006 Feb;96(2):327-33 and ii) Pollinex Quattro: EAACI XXVIII Congress 2009 Poster presentation
3 TRSS scoring ranges from 16 – 24 points for these studies
30HDM-SPIRE phase IIb (n=172)
Skin prick +ve HDM:
Efficacy demonstrated at 1 year US: 28%1 (86m)
EU:22%2 (82m)
Overall TRSS improvement of 2.8
vs. placebo (p = 0.02) at one year
Increasing symptom severity
Excellent data – Treatment effect maintained in more
similar to Cat-SPIRE at 1 year symptomatic subjects
1 Arbes et al. J Allergy Clin Immunol. 2005 Aug;116(2):377-83
2 Bousquet et al. Allergy. 2007: 62: 301-9
Selected for oral presentation at AAAAI 2014
31HDM-SPIRE phase IIb 2 year follow-up study
Improvement maintained; enhanced effect in more symptomatic
Matched subjects at year 1 and 2 Subjects with baseline TRSS >12
Overall TRSS improvement of 1.4 vs Overall TRSS improvement of
placebo at two years 3.0 vs. placebo
Overall TRSS improvement of
1.4 vs placebo at one year
Symptom improvement sustained at same level in same patients
32Grass-SPIRE phase IIb (n=282) Skin prick +ve
Efficacy demonstrated after first grass season Grass:
US: 27%1 (84m)
EU: 17%2 (63m)
Subjects with mean baseline TRSS ≥8 Subjects with mean baseline TRSS ≥12
Overall TRSS improvement of Overall TRSS improvement of
1.6 vs. placebo (p = 0.035) 2.0 vs. placebo (p=0.040)
Increasing treatment effect over time Enhanced efficacy in the more symptomatic
1 Arbes et al. J Allergy Clin Immunol. 2005 Aug;116(2):377-83 (Perennial rye)
2 Bousquet et al. Allergy. 2007: 62: 301-9 (Grass pollen)
33Grass-SPIRE phase IIb long-term follow-up studies
Symptom improvement confirmed in same subjects
Matched subjects (8 x 6 nmol group) Matched subjects (4 x 12 nmol group)
TRSS -5.0 vs.
placebo (p = 0.004) TRSS -4.5 vs.
placebo (p = 0.008)
TRSS -2.9 vs. TRSS -4.0 vs.
placebo (p = 0.075) placebo (p = 0.016)
TRSS -3.4 vs. TRSS -4.1 vs.
placebo (p = 0.033) placebo (p = 0.010)
Initial treatment effect maintained after three grass pollen seasons despite no further doses
34Ragweed-SPIRE phase IIb (n=275)
Skin prick +ve
Proof-of-concept demonstrated (2011) Ragweed:
US: 26%1 (82m)
Subjects with mean baseline TRSS ≥8 Subjects with mean baseline TRSS ≥12
Overall TRSS improvement of Overall TRSS improvement
1.7 vs. placebo (p = 0.066) of 2.9 vs. placebo (p = 0.044)
Stronger efficacy in more symptomatic subjects
1 Arbes et al. J Allergy Clin Immunol. 2005 Aug;116(2):377-83
J. Allergy Clin. Immunol. 2012 Feb 129, Issue 2, Supplement , Page AB368
35Ragweed-SPIRE phase IIb
Comparison treatment effect 2014 vs 2011 study
2014 (mean baseline TRSS ≥12) 2011 (mean baseline TRSS ≥12)
Overall TRSS improvement Overall TRSS improvement
1.2 vs. placebo (p = 0.149) 2.9 vs. placebo (p = 0.044)
Mean change in TRSS (baseline minus follow-up)
Marked placebo response
28 of 70 placebo-treated
subjects (40%) had >25%
reduction in symptom score
Greater placebo effect in 2014 study vs 2011 study on days 3 & 4
36Ragweed-SPIRE phase IIb (2014)
Field score endpoint
Mean change in Combined Score from pre- Mean Combined Score and mean ragweed
season to peak season pollen count
Placebo 8 x 12 nmol
ITT population 68 69
Mean change in CS 0.79 0.53
p value vs placebo - 0.090
Field endpoint: combined TRSS (0-24 scale) and
rescue medication use (RMS) score (0-3 scale)
- Combined Score = (TRSS / 8) + (RMS); 0-6 scale
Treatment effect 33% vs placebo
- FDA requires at least 15% treatment effect1
- World Allergy Organization: at least 20% treatment
effect clinically meaningful
1 With upper bound of 95% confidence interval minimum10%
37Potential to revolutionize immunotherapy market
Current immunotherapy
Key characteristics
Subcutaneous Sublingual
Short course immunotherapy giving clearly superior efficacy
Efficacy for at least a year with single course
Two year follow-up data encouraging¹
Very good safety and well-tolerated
Safety profile similar to placebo
No patients with anaphylaxis
Standardized dose
No need for dose escalation
No need for doses tailored to individual patients
State-of-the-art synthetic production process
No natural whole allergen
No potency variation between vials
Micro-needles
Patient-friendly administration
1 Demonstrated for Cat-SPIRE, HDM-SPIRE and Grass-SPIRE
38Full global rights retained
Ideal fit with focused commercialization strategy
Commercialization strategy US Cat-SPIRE target population
Independent in key markets Cat-allergic individuals ~24 million1
– Scalable infrastructure in place
– Train on Cat-SPIRE in preparation for launch Consulting an allergist / ~1.3 million Secondary
specialist focus
– Build relationships with allergists early Patients not
offered IT
– Map out customers & key accounts in advance
– Current sales support field force build well ahead of launch Offered IT ~1.0 million
– Target steeper sales curve with higher peak
Primary focus
Patients
Sales force plan declining IT
– 100 in N America initially targeting 3,500 allergists
– 90 in EU targeting high prescribers among 6,600 allergists /
specialists
Accept IT Secondary
(~378k) focus
Subsequently target other high prescribers Patients failing to
complete IT
– Selected ENT physicians
– Special interest primary care physicians
Complete
Partner in other markets IT (~60k)
1 Kantar Health Quantitative Cat Allergy Report 2010
39Multiple studies demonstrate significant
market opportunity
Selected sizing and pricing studies Opportunity for Cat-SPIRE
Illustrative peak sales of
US market research for Cat-SPIRE (Kantar / 2010) c.$500-700mm for US and EU
– 93 allergists, 82 PCPs, 8 payers
– US opportunity: $0.5-1.0 billion peak annual sales US: 200,000 x $2,600 = $520mm
US pricing research for Cat-SPIRE (Bridgehead / 2011)
– 101 allergists, 105 patients, 35 payers Equals 5 of 34 new cat
US pricing: Supported
– Supports pricing of $2,000-3,000 allergy patients / month
by third party research
EU market and pricing research for Cat-SPIRE (PRMA / 2011) already coming to allergist
– 27 specialists, 28 PCPs, 27 payers
– Supports Grazax as the likely benchmark
US market overview for 4 lead SPIRE products (LEK / 2009) EU: 50,000 x $1,500 (€1,100) = $75mm
– $2.6bn opportunity in US
1.5 million patients in EU pricing: Discount to
US and European research for 4 lead SPIREs (GfK / 2014)
EU already on allergy Grazax cost of €2.5-5.3k
– $2,600 pricing in US immunotherapy over 3+ years
Consistent assessment of commercial opportunity
401
Marketed products
2
Asthma / COPD pipeline
3
Novel allergy immunotherapies
4
Summary
41Strong newsflow
News Date* Description
Grass-SPIRE support study H1‘15 Observational study (TG003) reports (n=108)
Grass-SPIRE phase II results H1‘15 Phase II controlled asthmatic study (TG004) reports (n=54)
NIOX MINO® / VERO® sales data H2’15 Interim results with H1’15 sales results
Flixotide® substitute approval outcome1 H2’15 MHRA response to filing1
Cat-SPIRE safety study complete H2‘15 Pilot pediatric safety study (CP009) completes (n≥12)
Ragweed-SPIRE phase IIb complete H2’15 Phase IIb follow-up field study (TR006A) completes (n≤280)
HDM-SPIRE study recruitment H2’15 Complete phase IIb field study (TH005) recruitment (n=660)
Serevent® substitute filing H2’15 EU decentralized procedure filing to MHRA
Flixotide® substitute launch1 (if approved) H1’16 UK launch1 (if approved)
Seretide® substitute filing H1’16 EU decentralized procedure filing in UK
NIOX MINO® / VERO® sales data H1’16 Year end results with FY’15 sales
Cat-SPIRE phase III results H1‘16 Phase III study (CATALYST) reports (n=1,409)
Grass-SPIRE phase III start H1‘16 Enrolment starts into phase III field study
NIOX MINO® / VERO® sales data H2’16 Interim results with H1’16 sales results
Cat-SPIRE filing H2‘16 File for marketing approval
*To be included in announcements as appropriate and in-line with financial calendar including half-year / full-year results
1Approval / launch reflects estimate of MHRA review timelines only
42Delivering on our strategy
Delivering the pipeline
– Flixotide® substitute approval anticipated H2 2015
– Two regulatory filings anticipated by end H1 2016
– On track to report Cat-SPIRE phase III in H1 2016
– Strong clinical progress in multiple late-stage allergy programmes
Commercializing products independently in key markets
– Direct sales targeting allergy / asthma specialists in US and Germany
– Scalable infrastructure to optimize first allergy product launch
– Market access and payor expertise in place
– Leverageable across broader portfolio
Building broad and balanced portfolio
– Pipeline of 12 products in development for allergy, asthma and COPD
– Potential for 8 product launches by end 2021
World-class allergy & asthma specialty biopharma business fully funded to deliver portfolio
43A world-class specialty biopharma business
Marketed
products
Direct sales in US
and Germany
Broad international
distribution
network
Strong pipeline
Direct sales Distributors
44Contact us: Investors Financial and Corporate
Communications
Circassia Ltd Steven Harris, CEO FTI Consulting
Northbrook House Julien Cotta, CFO 200 Aldersgate
Robert Robinson Avenue Aldersgate Street
The Oxford Science Park t: +44 (0) 1865 405560 London EC1A 4HD
Oxford, OX4 4GA
t: +44 (0) 20 3727 1000
w: www.circassia.com e: circassia@FTIConsulting.com
e: ir@circassia.com
45Unaudited group financial information
Revenues £21.9m1,2
Operating loss £66.4m1,2
Estimated cash assuming completion June 2015 ~£230m3
1 Circassia & Aerocrine year ended 31 December 2014; Prosonix year ended 31 March 2014
2 SEK:£ average FX rate for year ended 31 Dec 2014 11.29:1
3 Director’s estimate; excludes £30m consideration contingent on UK approval
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