BioCryst Pharmaceuticals,Inc. (BCRX)

[JAG626]

BioCryst Pharmaceuticals
Update for Investors
A il62010April 6, 2010
Forward-Looking Statement
BioCryst’s presentation may contain forward-looking statements, including statements regarding future results, unaudited and forward-
looking financial information and company performance or achievements. These statements are subject to known and unknown
risks and uncertainties which may cause our actual results, performance or achievements to be materially different from any future results or
performances expressed or implied in this presentation. You should not place undue reliance on the forward-looking statements. For additional
information, including important risk factors, please refer to BioCryst’s documents filed with the SEC and located at
// / / fhttp://investor.shareholder.com/biocryst/sec.cfm
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We Have the Key Elements to Build an Enduring, Successful Company
Transformed biotechnology company with product revenue and two late-stage development candidates
Hospitalized seasonal influenza is a sizeable and sustainable opportunity
Emergency intravenous (i.v.) peramivir use offers current revenue and upside to sustainable seasonal flu franchise
PNP ith f ll ll d i t l t i l f CTCL(1) d i ifi tPNP programs with a fully enrolled pivotal trial for CTCL(1) and a significant potential for gout
Nondilutivefinancingmitigatescashburnandallowsinvestment inR&D(2)Non-dilutive financing mitigates cash burn and allows investment in R&D (2)
Experienced and proven team focused on delivering
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Substantial Progress in Past 12 months
Positive i.v. peramivir Shionogi Phase 3 results, leading to January 2010 approval & launch in Japanpp p
Green Cross filed for peramivir regulatory approval in South Korea
Emergency Use Authorization (EUA) issued
–U.S. Government purchased 10,000 courses of i.v. peramivir ($22.5M order)
Additional $77.2 M in HHS funding awarded for i.v. peramivir development and initiation of Phase 3 studies to support U.S. filing
Forodesine CTCL study fully enrolled
Phase 2 study of BCX4208 initiated in patients with gout
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Significant Value Creating Events to Come
Clinical EventsClinical Events Regulatory EventsRegulatory Events Commercial EventsCommercial Events
Forodesine data from pivotal CTCL study and
Phase 2 CLL study
Potential approval of peramivir in South
Korea, other countries
Stockpiling partners provide virtually global
coveragey
Data from two Phase 2 studies of BCX4208 in
gout
DatafromPhase3
Potential filing for peramivir approval in
other countries
g
Potential additional government orders for
i.v. peramivir
Potential royaltiesfromData from Phase 3 studies in i.v. peramivir
in hospitalized, seasonal influenza
Potential royalties from ex-U.S. approvals
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Advancing Late-Stage Pipeline in Multiple Indications
INFECTIOUS DISEASE
PRE-CLINICAL PHASE 1 PHASE 2 PIVOTAL FILED APPROVED
Peramivir Outpatient Flu(Seasonal Influenza / i.v.)
Peramivir Inpatient Flu(Acute Influenza / i.v.)
CANCER
PRE-CLINICAL PHASE 1 PHASE 2 PIVOTAL FILED APPROVED
Forodesine(CTCL)(CTCL)
Forodesine(CLL)
Forodesine(ALL)(ALL)
INFLAMMATORY DISEASE
PRE-CLINICAL PHASE 1 PHASE 2 PIVOTAL FILED APPROVED
BCX4208
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BCX4208(Gout)
PERAMIVIR FOR INFLUENZA
Peramivir(RAPIACTA) Approved & Launching in Japan
First marketing approval of a BioCryst discovered drug –January 2010g y
Broad Indication
–Seasonal—uncomplicated flu
–Seasonal—patients with high risk factors
One of fastest review periods ever by Japanese authorities
Japan is one of world’s largest markets for influenza anti-virals
–Over 10 million people treated annually
G ’–Gov’tpanel recommendation: grow anti-viral stockpile to cover 60 million citizens
Attractive economics for BioCryst
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New Influenza Anti-Virals are Needed
Seasonal influenza requiring hospitalization is a serious medical problem
–200,000 hospitalizations
–36,000 deaths in the U.S. annually
No anti-viral is approved for hospitalized influenza patients
Currently approved anti-virals are dosed orally or inhaled
Intravenousdeliveryoftherapyisthepreferredroutetotreat infectionsin
This medical need can be addressed by a potent, rapidly delivered, safe,
Intravenous delivery of therapy is the preferred route to treat infections in the hospital setting
y p , p y , ,effective i.v. anti-viral
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Key Requirements for U.S. NDA Filing
Task StatusTask Status
Two adequate & well-controlled efficacy studies One completedOne in progress
1,500 subjects for safety data base > 1,000 already complete
Commercial scaleCMC NDAlotscompletedCommercial scale CMC NDA lots completed
Drug product stability Currently > 3 years
Placebo-controlled Shionogi Phase 2 study is 1 of 2 well-controlled studies needed for approval
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pp
PeramivirPhase 3 Program Enrolling
Parameter Study 301 Study 303
Dose Groups PVR 600mg QD x 5 days + Std of Carevs. PVR 300mg BID x 5 days: orCare vs.
Placebo + Std of Care
orPVR 600mg QD x 5 days
Allocation ratio 2:1 1:1
Power 90% N/A
Hazard ratio 0.67 N/A
Endpoint Time to Clinical Resolution Reduction in viral titer
NTotal Sufficienttoconfirm306inITTI 300NTotal Sufficient to confirm 306 in ITTI (est. 445) 300
Patient Population Requires hospitalization and presence of one or more risk
factors
Broad inclusion
factors
Seasons NH / SH ’09/’10, NH ’10/’11 NH / SH ’09/’10, NH ’10/’11
Additional studies to provide further evidence of efficacy are underdiscussionwithFDA&HHS
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under discussion with FDA & HHS
Sustainable Revenue Opportunity—Seasonal Flu in the Hospital
Average U.S. flu season: Global flu season:
–200,000 hospitalizations –3-5 million severe illnesses
–36,000 deaths –250-500,000 deaths
The first i.v. anti-viral to market
Pharmacoeconomic benefits in reducing hospital and ICU stays
Base price established through initial Government orders
Patent protection to 2018
Revenue generation for BioCryst from Shionogi
–$7 million milestone for approval in 1Q 2010
–Royalties on net sales of 10-20%
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–Potential future commercial milestones of up to $95 million
Preparedness for PeramivirDemand
Emergency use authorization issuedOctober 23, 2009
Initial U.S. Government order received November 4
$–10,000 courses at $2,250
–Up to 30,000 additional courses may be ordered under the 2 year
contractcontract
Supply chain capability fully established: API to finished product
Packagedatotal of130000courses–Packaged a total of 130,000 courses
–Additional active pharmaceutical ingredient (API) available to produce up to 350,000 more courses
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Stockpiling Partners Provide Virtually Global Coverage
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Near-Term Peramivir Revenue Opportunities
Potential additional orders
fromHHSfrom HHSGovernment
Stockpiling Potential ex-U.S.
Government orders th hBi C t dthrough BioCryst and
regional partners
Korea&otherex-U.S.Fll R l t A l Korea & other exU.S. potential approvalsFull Regulatory Approval
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PNP INHIBITORS
FORODESINE FOR LYMPHOMA/LEUKEMIA
BCX4208 FOR GOUT
PNP Development Strategy Is Advanced and Targeted
CANCER
PRECLINICAL PHASE1 PHASE2 PIVOTAL FILED APPROVEDPRE-CLINICAL PHASE 1 PHASE 2 PIVOTAL FILED APPROVED
Forodesine(CTCL)
Forodesine(CLL)(CLL)
Forodesine(ALL)
GOUTGOUT
PRE-CLINICAL PHASE 1 PHASE 2 PIVOTAL FILED APPROVED
BCX4208
Multiple pre-clinical compounds available for near-term development
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Existing CTCL Treatments Have Significant Limitations
ManypatientswithCTCLhaveaprolongedcourseofillnessandqualityofMany patients with CTCL have a prolonged course of illness and quality of life is an important consideration
–Treatment should be effective, safe, convenient and tolerable for long-term chronic use
Majority of current treatment options have unfavorable side effect profiles and are inconvenient
Approved oral agents have dosing challenges and difficult side effect profilesprofiles
Forodesine profile meets patient needs in the CTCL market
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oodes epo e eetspate t eeds t eCC a et
Proof of Concept for Forodesine in CTCL
Best mSWATfrom Baseline in All Subjects vs. 80mg/m2 (Blue) Outcome Measure 80 mg/m2N=36
Overall RR 14(39%)
j g ( )
50
100
Percent of Change in mSWAT
Overall RR 14 (39%)
CR 2 (6%)
PR 12 (33%)
0
50 SD 15 (42%)
Time to Response, Days (95% CI) 42 (29.0 –58.0)
Response Duration, Days (95% CI) 127 (71.0, N/A)
(100)
(50)
Duvic, M. et al, ASH 2007 abstract #122
IndividualSubjects
p y ( ) ( )
Long-term treatment data presented at ASCO 2009
demonstratedacceptablesafetyprofileandefficacyin
Solid Phase 1 –2 results support pivotal study
Data from BCX1777-105 study
Individual Subjects
Subjects Not on 80mg/m2 Subjects on 80mg/m2
demonstrated acceptable safety profile and efficacy in
CTCL subjects treated for 12+ months
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Sod ase esutssuppotp ota study
Forodesine CTCL Pivotal Trial Nearing Completion
Pivotal study is ongoing under special protocol assessment by FDA
Single arm Forodesine 200mg daily
Differentiated profile
Study Design
Intervention & Outcome Measure Commercial Rationale
Stages 1b through 4
Three or more failed treatments
daily
Primary outcome measure is objective
response rate
Market addressable by small sales force
Composition of matter IPthrough2017
Prior therapy must include bexarotene
p IP through 2017
Potential price premium
~$80M sales of approved CTCL
products over the last 12 months
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Pivotal CTCL study fully enrolled, data expected 2H:10
CLL is an Attractive Indication for Forodesine
MostofthecurrentlyavailableMost of the currently available drugs have significant toxicity
concerns and i.v. dosing
People living with CLL in U.S., 2008: 90,179
Forodesinefor CLL
–Novel mechanism of action
New CLL diagnoses in U.S., 2008: 15,110
–Generally safe and well-tolerated in clinical trials
–Orally administered $35K-$75K /patient spent on CLL therapy annually
–Suitable for combination and potentially synergistic with
existing therapies
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Incidence data: American Cancer Society
Gout is a Growing Market with Significant Unmet Needs
Gout is now the most common inflammatory arthritis in males
Over last 40 years, the prevalence has increased in most Westernized countries by 200-300%
Growth in prevalence of gout appears to be caused by several factors including:
–Alarming rise in obesityAgingpopulation–Aging population
–Increase in prevalence of kidney failure & hypertension–Widespread use of prescription drugs and alcohol
Issueswithcurrenttherapies:efficacyallergicreactionsandsideeffectsIssues with current therapies: efficacy, allergic reactions and side effects
Phase 2 program initiated in September 2009; Datafrom two studies
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p g p ;expected in 2010
Phase 2 Psoriasis Study -Rationale for Gout
1
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Dose of BCX4208, mg/day
0 20 120
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Dose related reduction in uric acid sustained for the duration of drug exposure
Placebo 20mg/d BCX4208 120mg/d BCX4208
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Consistent finding in studies of PNP inhibitors
Uric acid elevated in gout and is the cause of the disease
BCX4208 Gout Development Strategy & Commercial Rationale
Gout prevalence in the U.S. and Europe
rangesfrom0814%
Study Design Goal
Primary
Outcome
Measure
Top-line
Data
Expected
3 treatment Dose-Monotherapy
ranges from 0.8-1.4%
U.S. demographic lifestyle trends could
arms
Placebo-
controlled
response
+
Safety
Reduction
in uricacid
2Q:10
Monotherapy
Dose-ranging
Phase 2a
lead to 3.8-5.2M with gout in 2020
~$2000annuallyfor
Controlled
(BCX4208
+/-
Allopurinol)
Dose-
response
+
Safety
Reduction
in uric acid
4Q:10
Combination
therapy
Phase 2b
$2000 annually for current gout therapy
(febuxostat)
N d t fNew products for refractory gout may
price as high as $20,000/yr
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Phase 2a Gout Study –Dosing Design and Research Goals
Part 1: Parallel GroupN=60 Part 2: Dose EscalationN=60
BCX4208
BCX420840mg/day EligibleSubjects BCX4208160mg/day or PBO
Data Review –Decision to ContinueBCX4208
80mg/day
BCX4208120mg/day
EligibleSubjects R Eligible
Subjects
BCX4208240mg/day or PBO
DataReview–DecisiontoContinue
g y
Placebo
EligibleSubjects BCX4208320mg/day or PBO
Data Review Decision to Continue
Study GoalsDetermine dose response for uric acid reduction (efficacy)
Determinedoseresponseforreductioninlymphocytes(safety)
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Determine dose response for reduction in lymphocytes (safety)Select a dose(s) for Phase 2b trial
Selectively Investing in our Discovery Platform
Target
Illustrative
Indications
Hepatitis C RNA Ch i H titi C
Management has focused on advancing late-stage products
Successful clinical progressandp
Polymerase
Chronic Hepatitis C
Kallikrein
Hereditary
angioneuroticedema
Successful clinical progress and non-dilutive funding allow the
opportunity to invest in early stage assets
angioneurotic edema
Complement
Age related macular
degeneration
Multiple promising candidates in development
JAK
Psoriasis;
Rheumatoid arthritis
Management is taking a focused and financially disciplined approach
to R&D
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Financial Summary
Potential revenue opportunities exist from:
–Royalty payments from strategic partnerships–RevenuefromperamivirstockpilingRevenue from peramivir stockpiling
Recent secondary offering & product revenue have significantly strengthened BioCryst’s balance sheet
Current cash allows us to fund clinical development plans for the next 2-3 years
Common shares outstanding 43.9 M as of 12/31/09
C h h i l t & iti $943M f12/31/09Cash, cash equivalents & securities $94.3 M as of 12/31/09
Underlying 2009 cash use* $37.2 M
Anticipated 2010 cash use $25-30 M
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*Excludes proceeds from equity offering and sale of peramivir to HHS
2010 Events
Additionalstockpilingpartnerscoversmajormarketsglobally
Clinical Events
Additional stockpiling partners covers major markets globally
Update regarding forodesine CLL study
ForodesineCTCL study reaches full enrollment (1Q:10)
Data from Phase 2 monotherapy study of BCX4208 in gout (2Q:10)
Provide update: plans for BCX4208/allopurinolcombo study in gout (mid-10)
Data from pivotal forodesine CTCL study (2H:10)
Data from Phase 2 BCX4208/allopurinol combo study in gout (4Q:10)
Dt f Ph 2CLL t d (2H10)Data from Phase 2 CLL study (2H:10)
R l t dC i lE t
Peramivirmarketing authorization in Japan
Potential for additional regulatory approvals and revenue events
Regulatory and Commercial Events
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Potential for royalty payment on seasonal sales of peramivir ex-U.S.
Endnotes
1. CTCL: Cutaneous T-Cell Lymphoma2. Non-dilutive financing sources include HHS, Shionogi and Mundipharmag , g p
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