Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Merger or Acquisition (January 2018)
36 slides from Biocryst Pharmaceuticals's merger presentation, January 2018
Slide 1: Combining Capabilities to Serve More Patients with Rare Diseases
Filed by BioCryst Pharmaceuticals, Inc. pursuant to Rule 425 Under the Securities Act of 1933 And Deemed Filed Pursuant to Rule 14a-12 Under the Securities Exchange Act of 1934 Subject Company: BioCryst Pharmaceuticals, Inc. Commission File No. of Subject Company: 000-23186 Combining Capabilities to
Slide 2: Additional Information and Where to Find It
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 to
Slide 3: Forward-Looking Statements
Forward-Looking Statements 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 expectat
Slide 4: Combination Creates Substantial Value
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 dev
Slide 5: Combination Highlights
Combination Highlights • Stock for stock transaction Terms • 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 sto
Slide 6: Creating a Leader in Innovative Rare Disease Therapies
Creating a Leader in Innovative Rare Disease Therapies Developing Oral Therapies for Life Rare Disease Company with Strong Threatening Rare Diseases Immuno-Oncology Assets Small Molecule Rare Disease Oligo Rare Disease Discovery Discovery Engine Engine 2 Late Stage Programs 2 Late Stage Programs Lea
Slide 7: Patient-Centric Rare Disease Culture and Approach
Patient-Centric Rare Disease Culture and Approach Synergistic Complementary Robust Pipeline Financial Strength Discovery Engines Leadership • 2 Phase 3 orphan- • Significant experience • $30 million in annual • Proven commercial designated programs with 2 distinct engines pre-tax cost synergies team
Slide 8: Phase 3 Programs Create Financially Strong Foundation to Support Robust, Rare Disease Focused Pipeline
Phase 3 Programs Create Financially Strong Foundation to Support Robust, Rare Disease Focused Pipeline IMO-2125 Compelling Data PD-1 Refractory Melanoma driving 2 Phase 3 BCX7353 in Combination with Prophylactic HAE programs ipilimumab • Novel agent designed to induce abscopal anti-tumor immune resp
Slide 9: Merger Upside: Maximizing Value and Market Potential
Merger Upside: Maximizing Value and Market Potential Value Driver Merger Amplifier Idera management HAE launch Commercializing BCX7353 experience Investing in commercial launch and Capital from out licensing ongoing pipeline development opportunities BCX7353 and IMO-2125 Phase 3 Expanding market opp
Slide 10: Synergistic Discovery Engines with Enhanced Development Opportunities
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 uniqu
Slide 11: Robust Rare-Disease Focused Pipeline
Robust Rare-Disease Focused Pipeline Idera BioCryst Lead Pre-clinical Ph 1 Ph 2 Ph 3 Filed Approved optimization STRATEGY: Discover and develop novel therapies for life-threatening, rare diseases IMO-2125 – PD-1 Refractory Melanoma in Orphan-Designation combination with ipilimumab BCX7353 – HAE Prop
Slide 12: Innovative Portfolio of Late-Stage Programs
Innovative Portfolio of Late-Stage Programs IMO-2125 BCX7353 PD-1 Refractory BCX7353 IMO-8400 Prophylactic HAE Melanoma in Combination Acute HAE Dermatomyositis with ipilimumab • Oral (capsule) Kallikrein • Intratumoral TLR9 • Oral (liquid) Kallikrein • Subcutaneous TLR Inhibitor for Hereditary Agon
Slide 13: Proven Rare Disease Clinical & Commercial Track Record
Proven Rare Disease Clinical & Commercial Track Record • 1st prophylactic • Multiple global and U.S. • >245 HAE patients dosed and studied • Treatment of C. treatment of HAE rare disease launches • CMOs clinical development/launch difficile- • Grew to ~$400M • Led launch for 5 global experience: Ara
Slide 14: 2018: Significant Near-Term Value-Building Events
2018: Significant Near-Term Value-Building Events Q1 BCX 7353 BCX 7353 Initiate APEX-2 Ph 3 Pivotal Trial in HAE prophylaxis Data from ZENITH-1 Phase 2 Study in Acute HAE Q1 IMO-2125 Initiate ILLUMINATE 301 Ph 3 Pivotal Trial in PD-1 Refractory Metastatic Melanoma in IMO-2125 combination with ipilim
Slide 15: Solid Capital Position & Meaningful Operational Synergies
Solid Capital Position & Meaningful Operational Synergies ◆ ~$243 million net cash balance* • Capital for continued clinical development through next major milestone events and into Q3 2019 • Capital for commercial launch planning and preparation • Multiple options for non-dilutive capital through r
Slide 16: Wall Street Analysts Recognize Value of Combination
Wall Street Analysts Recognize Value of Combination “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 “…We believe [the combination] will provide enhanced HAE regula
Slide 17: BioCryst & Idera Boards Carefully Evaluated Strategic Options
BioCryst & Idera Boards Carefully Evaluated Strategic Options ◆ 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 opportu
Slide 18: Combination Creates Substantial Value
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 dev
Slide 19: Appendix
Appendix +
Slide 20: APeX-1: Overall Angioedema Attack Rate per Week, PP Population, Weeks 2-4 and 1-4
APeX-1: Overall Angioedema Attack Rate per Week, PP Population, Weeks 2-4 and 1-4 Attack Rate: LS Mean Attacks/Week 1.0 62.5mg 125mg 250mg 350mg WEEKS 2-4 % Difference, Active-PBO -7% -73% -46% -58% 0.5 p-Value 0.715 <0.001 0.006 <0.001 WEEKS 1-4 % Difference, Active-PBO -6% -69% -53% -48% p-Value 0
Slide 21: APeX-1: 125 mg Dose Provided Consistent Reductions in Attack Rate
APeX-1: 125 mg Dose Provided Consistent Reductions in Attack Rate LS mean1 Attacks per Week Percentage p-Value Difference vs Analysis n Placebo Reduction vs vs BCX7353 125 mg Placebo Placebo Placebo Confirmed attacks, 13 0.248 0.932 -0.684 73% <0.001 Weeks 2-4 PP population Confirmed attacks, 14 0.2
Slide 22: APeX-1: Angioedema Quality of Life (AE-QoL): LS Mean Change from BL at Day 29
APeX-1: Angioedema Quality of Life (AE-QoL): LS Mean Change from BL at Day 29, PP Total Score Functioning Fatigue/Mood Fears/Shame Nutrition 0 MCID QOL Score Placebo -10 Improved BCX7353 62.5mg QD BCX7353 125mg QD BCX7353 250mg QD -20 * * BCX7353 350mg QD * p<0.05 -30 ** p<0.005 * ** ** ** -40 ** Di
Slide 23: APeX-1: Treatment-Emergent Adverse Event Summary
APeX-1: Treatment-Emergent Adverse Event Summary BCX7353 62.5 mg 125 mg 250 mg 350 mg Placebo Category N=7 N = 14 N = 14 N = 18 N = 22 Subjects with any TEAE1, n (%) 4 (57) 7 (50) 11 (79) 14 (78) 15 ( 68.2) Subjects with any Serious AE, n (%) 0 0 1 (7)2 0 0 Subjects with Drug-Related Grade 3/4 AE, n
Slide 24: APeX-1: Exposure Comparisons of BCX7353 and SC C1INH
APeX-1: Exposure Comparisons of BCX7353 and SC C1INH CSL-830 Phase 3 Study BCX7353 APeX-1 & Phase 1 Study C1INH levels at baseline and after SC BCX7353 plasma concentrations at 24 hours post-dose dosing with CSL-8301 1 Longhurst, H. et al. N Engl J Med 376, 1131-1140 (2017). Box plots represent medi
Slide 25: Predictable PK Supports 175 mg as Second Dose in Phase 3
Predictable PK Supports 175 mg as Second Dose in Phase 3 % > 4 x EC50 % > 6 x EC50 % > 8 x EC50 • Given the predictable PK of BCX7353, Dose, simulations are helpful in selecting an mg QD intermediate dose of BCX7353 to study Predicted Actual Predicted Actual Predicted Actual that is between 125 mg a
Slide 26: APeX-2: Phase 3 Trial Design
APeX-2: Phase 3 Trial Design Blinded Treatment Screening / Run-in 24 weeks • Primary endpoint at Week 24: • Rate of Investigator-confirmed HAE N ≅ 32 BCX7353 150 mg QD* attacks through entire treatment period Screening • Study powered at >90% to detect N ≅ 32 BCX7353 110 mg QD* Visit a ≥50% reductio
Slide 27: APeX-2: Phase 3 Trial Design – Safety Extension
APeX-2: Phase 3 Trial Design – Safety Extension extension Blinded Treatment Screening / Run-in 24 weeks Safety Extension 24 weeks N ≅ 32 BCX7353 150 mg QD* BCX7353 150 mg QD Screening N ≅ 32 BCX7353 110 mg QD* BCX7353 110 mg QD Visit BCX7353 150 mg QD N ≅ 32 Placebo QD BCX7353 110 mg QD Final analys
Slide 28: APeX-S: Long-term Safety Study Design
APeX-S: Long-term Safety Study Design • Endpoints: 48 Weeks Treatment • Long term safety of BCX7353 • Durability of response • Quality of Life N ≅ 80 BCX7353 150 mg QD • APeX-1 subjects eligible N ≅ 80 BCX7353 110 mg QD ◆ Safety database: • Up to 100 subjects at each dose level • Combination of APeX
Slide 29: Phase 1/2 Study in Anti-PD-1 Refractory Melanoma
Phase 1/2 Study in Anti-PD-1 Refractory Melanoma 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 interventional
Slide 30: Time on Study: Best RECIST v1.1 Response and Largest Percentage Decrease in Target Lesions
Time on Study: Best RECIST v1.1 Response and Largest Percentage Decrease in Target Lesions (8mg subjects) + 30
Slide 31: Patient 004 Remains a CR since May 2016
Patient 004 Remains a CR since May 2016 Pre-Therapy Post-Therapy 03/2016 08/2016 Injected Lesion Distant Lesions + 31
Slide 32: Phase 1 Conclusions
Phase 1 Conclusions • 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 sh
Slide 33: Phase 2 Expansion Update
Phase 2 Expansion Update • 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 Arizo
Slide 34: Phase 3 Trial Design
Phase 3 Trial Design Unresectable or metastatic Ipilimumab 3 mg/kg Q3wks for 4 melanoma w/ confirmed doses radiologic progression on or after a PD-1 inhibitor: • ≥21d from most recent No cross-over aPD-1 and no intervening systemic Tx • No prior ipi (except Ipilimumab (same, beginning wk 2) adjuvant
Slide 35: Phase 3 Readiness (FPFV 1Q18)
Phase 3 Readiness (FPFV 1Q18) • 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 • C
Slide 36: Growth/Partnering Opportunities
Growth/Partnering Opportunities INTRODUCE EXPAND TRANSFORM Unresectable “Cold” tumors Emerging I/O metastatic unaddressable with addressable tumors melanoma current I/O • High need in anti-PD1- • Moderate response to • Significant opportunity refractory patients cornerstone anti-PD1 in tumors with:
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