Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Merger or Acquisition (February 2018)
36 slides from Biocryst Pharmaceuticals's merger presentation, Feb 27, 2018
Slide 1: Additional Information and Where to Find It
2 Additional Information and Where to Find It Additional Information and Where to Find It In connection with the proposed merger, Nautilus Holdco, Inc. (“Holdco”) has filed with the U.S. Securities and Exchange Commission (the “SEC”) a Registration Statement on Form S-4 (as may be amended from time
Slide 2: Forward-Looking Statements
Forward-Looking Statements 3 These materials contain forward-looking statements within the meaning of the federal securities law, regarding, among other things, future events or the future financial performance of Idera and BioCryst. Such statements are based upon current plans, estimates and expect
Slide 3: Combination Creates Substantial Value
A unique player in rare diseases, with scale and strengthened competitive position More opportunities for success through diversified late-stage pipeline, variety of early stage programs and supporting assets Synergistic discovery engines with enhanced development opportunities, including through jo
Slide 4: Terms
Terms Stock for stock transaction Each share of BioCryst to be converted into 0.50 shares of new company stock Each share of Idera to be converted into 0.20 shares of new company stock Ownership at Closing BioCryst stockholders to own 51.6% of new company and Idera stockholders to own 48.4%, on a fu
Slide 5: Creating a Leader in Innovative Rare Disease Therapies
Creating a Leader in Innovative Rare Disease Therapies 6 Rare Disease Company with Strong Immuno-Oncology Assets Oligo Rare Disease Discovery Engine 2 Late Stage Programs Lead Candidate: IMO-2125 PD-1 Refractory Melanoma Developing Oral Therapies for Life Threatening Rare Diseases Small Molecule Rar
Slide 6: Robust Pipeline, Complementary Leadership, Financial Strength
Robust Pipeline Complementary Leadership Proven commercial team; launched 1st prophylactic HAE product Extensive clinical development experience Significant experience with 2 distinct engines Expands number of rare disease targets beyond standalone capabilities Creates opportunities for differentiat
Slide 7: Phase 3 Programs Create Financially Strong Foundation
Phase 3 Programs Create Financially Strong Foundation to Support Robust, Rare Disease Focused Pipeline 8 BCX7353 Prophylactic HAE IMO-2125 PD-1 Refractory Melanoma in Combination with ipilimumab Once a day oral (capsule) Competitive attack rate reduction 73% Safety & tolerability similar to placebo
Slide 8: Merger Upside: Maximizing Value and Market Potential
Merger Upside: Maximizing Value and Market Potential 9 Value Driver Merger Amplifier Commercializing BCX7353 Idera management HAE launch experience Investing in commercial launch and ongoing pipeline development Capital from out licensing opportunities Expanding market opportunity + diffusing risk B
Slide 9: Synergistic Discovery Engines with Enhanced Development Opportunities
Ability to leverage both structure-guided small molecule design and nucleic acid/oligonucleotide chemistry within one organization Combination therapy of small molecule and oligo may create more effective and potentially unique treatments for rare diseases Combining technologies expands number of ra
Slide 10: Robust Rare-Disease Focused Pipeline
+ Lead optimization Pre-clinical Ph 1 Ph 2 Ph 3 Filed Approved STRATEGY: Discover and develop novel therapies for life-threatening, rare diseases IMO-2125 – PD-1 Refractory Melanoma in combination with ipilimumab BCX7353 – HAE Prophylaxis (Capsule) IMO-8400 – Dermatomyositis BCX7353 – HAE Acute (Liq
Slide 11: Innovative Portfolio of Rare Disease Programs
+ Oral (capsule) Kallikrein Inhibitor for Hereditary Angioedema One pill, once a day – fulfilling patient needs HAE market expected to exceed $2B in global sales Robust quality of life data BCX7353 Prophylactic HAE IMO-2125 PD-1 Refractory Melanoma in Combination with ipilimumab Subcutaneous TLR 7,8
Slide 12: Proven Rare Disease Clinical & Commercial Track Record
1st prophylactic treatment of HAE Grew to ~$400M in N.A. annual sales in 5 years Treatment of C. difficile-associated diarrhea (CDAD) Grew to ~$300M in annual sales Multiple global and U.S. rare disease launches Led launch for 5 global brands that drive ~70% of CSL’s current revenue Grew U.S. Hizent
Slide 13: 2018: Significant Near-Term Value-Building Events
Initiate APEX-2 Ph 3 Pivotal Trial in HAE prophylaxis BCX 7353 Q1 Initiate ILLUMINATE 301 Ph 3 Pivotal Trial in PD-1 Refractory Metastatic Melanoma in combination with ipilimumab IMO-2125 Q1 Data available from PIONEER Phase 2 Trial in Dermatomyositis IMO-8400 Q2 Complete enrollment in ILLUMINATE 20
Slide 14: Solid Capital Position & Meaningful Operational Synergies
Solid Capital Position & Meaningful Operational Synergies 15 * Unaudited pro-forma cash balance as of December 31, 2017 Strong Combined Financial Profile with Opportunities to Generate Non-Dilutive Capital ~$243 million net cash balance* Capital for continued clinical development through next major
Slide 15: Wall Street Analysts Recognize Value of Combination
Wall Street Analysts Recognize Value of Combination 16 “We view the merger favorably for creating pipeline "critical mass" (and risk diversification) versus clear clinical synergies, a direct correlation between the respective programs. We believe the merger adds strength in the form of 1) broader p
Slide 16: Engaged, Well-Advised Boards
Engaged, Well-Advised Boards BioCryst and Idera Boards comprised of highly experienced directors with extensive industry knowledge BioCryst Board of Directors met numerous times over last two years to discuss value enhancing opportunities for BioCryst Both Boards retained financial and legal advisor
Slide 17: Combination Creates Substantial Value
A unique player in rare diseases, with scale and strengthened competitive position More opportunities for success through diversified late-stage pipeline, variety of early stage programs and supporting assets Synergistic discovery engines with enhanced development opportunities, including through jo
Slide 18: Combination Creates Substantial Value
v v Combination Creates Substantial Value A unique player in rare diseases, with scale and strengthened competitive position More opportunities for success through diversified late-stage pipeline, variety of early stage programs and supporting assets Synergistic discovery engines with enhanced devel
Slide 19: APeX-1: Overall Angioedema Attack Rate per Week
APeX-1: Overall Angioedema Attack Rate per Week, PP Population, Weeks 2-4 and 1-4 20 1.0 0.5 0 62.5mg 125mg 250mg 350mg WEEKS 2-4 % Difference, Active-PBO -7% -73% -46% -58% p-Value 0.715 <0.001 0.006 <0.001 WEEKS 1-4 % Difference, Active-PBO -6% -69% -53% -48% p-Value 0.747 <0.001 <0.001 <0.001 WEE
Slide 20: APeX-1: Dose Provided Consistent Reductions in Attack Rate
APeX-1: 125 mg Dose Provided Consistent Reductions in Attack Rate 21 1 Least squares mean calculated using an ANCOVA model with qualifying attack rate as covariate Analysis n LS mean1 Attacks per Week Difference vs Placebo Percentage Reduction vs Placebo p-Value vs Placebo BCX7353 125 mg Placebo Con
Slide 21: APeX-1: Angioedema Quality of Life (AE-QoL)
APeX-1: Angioedema Quality of Life (AE-QoL): LS Mean Change from BL at Day 29, PP 22 Difference in adjusted least square means are shown (Active treatment minus Placebo). ANCOVA Model includes terms of treatment and adjusted qualifying attack rate. Reductions (negative changes from BL) represent imp
Slide 22: APeX-1: Treatment-Emergent Adverse Event Summary
1 TEAE- treatment-emergent adverse event. 2 GI infection- investigator assessed as unrelated to study drug. Abdominal symptoms similar to several previous non-HAE-attack episodes occurring over past 3 years resulting in severe vomiting and diarrhea. Pt presented to ER and hospitalized for IV fluids
Slide 23: APeX-1: Exposure Comparisons of BCX7353 and SC C1INH
APeX-1: Exposure Comparisons of BCX7353 and SC C1INH 24 1 Longhurst, H. et al. N Engl J Med 376, 1131-1140 (2017). Box plots represent median, 25th and 75th percentiles, minimum and maximum values. CSL-830 and BCX7353 data are from distinct clinical trials and no head to head study has been conducte
Slide 24: Predictable PK Supports Second Dose in Phase 3
Predictable PK Supports 175 mg as Second Dose in Phase 3 25 Given the predictable PK of BCX7353, simulations are helpful in selecting an intermediate dose of BCX7353 to study that is between 125 mg and 250 mg. These simulations suggest a relatively small increase in dose above 125 mg should achieve
Slide 25: APeX-2: Phase 3 Trial Design
APeX-2: Phase 3 Trial Design 26 Primary endpoint at Week 24: Rate of Investigator-confirmed HAE attacks through entire treatment period Study powered at >90% to detect a ≥50% reduction in attack rate over placebo N 32 BCX7353 150 mg QD* Screening Visit Screening / Run-in Blinded Treatment 24 weeks N
Slide 26: APeX-2: Phase 3 Trial Design – Safety Extension
APeX-2: Phase 3 Trial Design – Safety Extension 27 N 32 BCX7353 150 mg QD* Screening / Run-in Blinded Treatment 24 weeks N 32 BCX7353 110 mg QD* N 32 Placebo QD BCX7353 150 mg QD BCX7353 110 mg QD 24 weeks Safety Extension *Doses in Phase 2 APeX-1 were shown as the dihydrochloride salt: 150 mg = 175
Slide 27: APeX-S: Long-term Safety Study Design
APeX-S: Long-term Safety Study Design 28 Endpoints: Long term safety of BCX7353 Durability of response Quality of Life APeX-1 subjects eligible N 80 BCX7353 150 mg QD 48 Weeks Treatment N 80 BCX7353 110 mg QD *Doses in Phase 2 APeX-1 were shown as the dihydrochloride salt: 150 mg = 175 mg dihydrochl
Slide 28: Phase 1/2 Study in Anti-PD-1 Refractory Melanoma
Phase 1/2 Study in Anti-PD-1 Refractory Melanoma 29 Phase 2 Expansion with Ipilimumab Enrolling Dosing: IMO-2125 is given as a single intratumoral agent week 1,2,3,5,8,11,15,19,23 Ipilimumab and pembrolizumab are administered per label beginning week 2 Deep injections are permitted with intervention
Slide 29: Time on Study: Best RECIST v1.1 Response
Time on Study: Best RECIST v1.1 Response and Largest Percentage Decrease in Target Lesions (8mg subjects) 30
Slide 30: Patient 004 Remains a CR since May 2016
Post-Therapy 08/2016 Pre-Therapy 03/2016 Injected Lesion Distant Lesions Patient 004 Remains a CR since May 2016 31
Slide 31: Phase 2 Conclusions
Phase 1 Conclusions 32 The combination of IMO-2125 with ipilimumab was tolerable at all dose levels studied; Dendritic cell activation, detectable within 24 hours of the first IMO-2125 injection, is evidence for target acquisition at the Recommended Phase 2 Dose (8mg); IMO-2125 with ipilimumab showe
Slide 32: Phase 2 Expansion Update
Phase 2 Expansion Update 33 Ipilimumab Combination Phase 2 Trial Expansion – Targeting approximately 60 patients with PD-1 refractory metastatic melanoma treated with 8mg 21 patients enrolled 10 Centers (5 sites currently enrolling) MD Anderson, Roswell Park, Vanderbilt, Huntsman, Uni. of Arizona Op
Slide 33: Phase 3 Trial Design
Phase 3 Trial Design 34 Ipilimumab 3 mg/kg Q3wks for 4 doses Ipilimumab (same, beginning wk 2) + intratumoral IMO-2125, wks 1, 2, 3, 5, 8, 11, 16, 20, 24 Unresectable or metastatic melanoma w/ confirmed radiologic progression on or after a PD-1 inhibitor: >21d from most recent aPD-1 and no interveni
Slide 34: Phase 3 Readiness
Phase 3 Readiness (FPFV 1Q18) 35 Agreement with FDA and MHRA on design and path forward for regular and accelerated approval (one study) Fast Track Designation Granted by U.S. FDA in Q4 2017 Global trial (US, Can, EU, Aus) ~300 patients ~70 sites planned CMC work on track for 1Q18 start Commercial p
Slide 35: Growth/Partnering Opportunities
Growth/Partnering Opportunities 36 Expand Transform Unresectable metastatic melanoma High need in anti-PD1-refractory patients Peak year sales estimate > $500 million Emerging I/O addressable tumors “Cold” tumors unaddressable with current I/O Moderate response to cornerstone anti-PD1 Increasing num
Slide 36: Growth/Partnering Opportunities
Growth/Partnering Opportunities INTRODUCE Unresectable metastatic melanoma EXPAND Emerging 0 addressable tumors. TRANSFORM “Cold” tumors unaddressable with current /O + High need in anti-PD1- refractory patients + Peak year sales estimate > $500 million patient population at 2025! IL BPDI-elractory
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