Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Merger or Acquisition (June 2018)
36 slides from Biocryst Pharmaceuticals's merger presentation, Jun 21, 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 mergers, Nautilus Holdco, Inc. (“Holdco”) has filed with the U.S. Securities and Exchange Commission (the “SEC”), and the SEC has declared effective on May 23, 2018, a Post-Effec
Slide 2: Forward-Looking Statements
Forward-Looking Statements 3 These materials contain forward-looking statements within the meaning of the federal securities laws, including Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements involve known and u
Slide 3: Combination Creates Substantial Value
Combination Creates Substantial Value 4 Complementary Assets and Platforms Enhance Market Opportunities and Accelerate Value Creation Maximizing Value and Market Potential Robust, Rare Disease Focused Pipeline Synergistic Discovery Engines Proven Clinical and Commercial Track Record Increased Financ
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 Recent Positive Data on Key Late-Stage Program Lead Candidate: Tilsotolimod PD-1 Refractory Melanoma Developing Oral Therapies for Life Threatening Rar
Slide 6: 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 Tilsotolimod – PD-1 Refractory Melanoma in combination with ipilimumab BCX7353 – HAE Prophylaxis (Capsule) BCX7353 – HAE Acute (Liquid) Tilsotolimod – Sol
Slide 7: Tilsotolimod Data from ILLUMINATE-204 Trial
8 Tilsotolimod Data from ILLUMINATE-204 Trial Trial Objectives Assess preliminary clinical activity of tilsotolimod in combination with ipilimumab at the respective recommended Phase 2 dose (RP2D) in patients with metastatic melanoma that is not responsive to PD-1 inhibitor therapy, using Response E
Slide 8: Tilsotolimod Data from ILLUMINATE-204 Trial
Tilsotolimod Data from ILLUMINATE-204 Trial 38.1% Response Rate / 71.4% Disease Control Rate 9
Slide 9: Tilsotolimod Data from ILLUMINATE-204 Trial
Tilsotolimod Data from ILLUMINATE-204 Trial 38.1% Response Rate / 71.4% Disease Control Rate 10
Slide 10: Results Reinforce Clinical Attractiveness of Treatment
Tilsotolimod Data from ILLUMINATE-204 Trial Results Reinforce Clinical Attractiveness of Treatment 11 Tilsotolimod + ipilimumab revives the immune response in anti-PD-1-resistant tumors resulting in altering the tumor microenvironment and conversion of cold (noninflamed) to hot (inflamed) tumors Thi
Slide 11: Phase Asset with Real Utility in I/O Toolkit
Tilsotolimod Data from ILLUMINATE-204 Trial Phase 3 Asset with Real Utility in I/O Toolkit 12 Tilsotolimod data continues to be very clinically meaningful even after doubling the number of patients Response rate of 38.1% in Idera’s trial is approximately triple that of response rates of ipilimumab a
Slide 12: Phase 3 Trial Design for Tilsotolimod
13 Tilsotolimod Data from ILLUMINATE-204 Trial Results Reinforce Clinical Attractiveness of Treatment Ipilimumab 3 mg/kg Q3wks for 4 doses Ipilimumab (same, beginning wk 2) + intratumoral tilsotolimod, wks 1, 2, 3, 5, 8, 11, 16, 20, 24 Unresectable or metastatic melanoma w/ confirmed radiologic prog
Slide 13: Merger Upside: Maximizing Value and Market Potential
Merger Upside: Maximizing Value and Market Potential 14 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
Slide 14: Synergistic Discovery Engines with Enhanced Development Opportunities
Synergistic Discovery Engines with Enhanced Development Opportunities 15 KIDNEYS LIVER Idera’s oligonucleotide chemistry primarily targets the kidney and liver. BioCryst’s structure-based small molecule design combined with Idera’s oligo chemistry may enable delivery of oligo treatments to additiona
Slide 15: Solid Capital Position & Meaningful Operational Synergies
Solid Capital Position & Meaningful Operational Synergies 17 * Unaudited pro-forma cash balance as of March 31, 2018 Strong Combined Financial Profile with Opportunities to Generate Non-Dilutive Capital ~$204 million net cash balance* Capital for continued clinical development through next major mil
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: BioCryst + Idera: Valuation Perspectives
BioCryst + Idera: Valuation Perspectives 19 Similar expected value from modestly higher risk profile but significantly larger value potential plus meaningful upsides in other tumor types Risk-adjusted equity value $1.2bn $1.2bn Strong data for 7353 HAE Prophylactic, with uncertain read-through to ef
Slide 18: Combination Creates Substantial Value
Combination Creates Substantial Value 20 Complementary Assets and Platforms Enhance Market Opportunities and Accelerate Value Creation Maximizing Value and Market Potential Robust, Rare Disease Focused Pipeline Synergistic Discovery Engines Proven Clinical and Commercial Track Record Increased Finan
Slide 19: Combination Creates Substantial Value
Combination Creates Substantial Value DEERE MEE © Creates a unique player in rare diseases, with scale New Data Market Potential and strengthened competitive position Presented on PRES © More opportunities for success through diversified Tilsotolimod at 2 late-stage pipeline, variety of early-stage
Slide 20: APeX-1: Overall Angioedema Attack Rate per Week
APeX-1: Overall Angioedema Attack Rate per Week, PP Population, Weeks 2-4 and 1-4 22 62.5mg 125mg 250mg 350mg WEEKS 2-4 % Difference, Active-PBO -9% -74% -47% -58% p-Value 0.657 <0.001 0.005 <0.001 WEEKS 1-4 % Difference, Active-PBO -4% -70% -54% -50% p-Value 0.818 <0.001 <0.001 <0.001 Attack Rate:
Slide 21: APeX-1: Dose Provided Consistent Reductions in Attack Rate
APeX-1: 125 mg Dose Provided Consistent Reductions in Attack Rate 23 1 Least squares mean calculated using an ANCOVA model with qualifying attack rate as covariate Final Data Analysis n LS mean1 Attacks per Week Difference vs Placebo Percentage Reduction vs Placebo p-Value vs Placebo BCX7353 125 mg
Slide 22: 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 24 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 23: 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 24: APeX-1: Exposure Comparisons of BCX7353 and SC C1INH
APeX-1: Exposure Comparisons of BCX7353 and SC C1INH 26 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 25: Predictable PK Supports Second Dose in Phase 3
Predictable PK Supports 175 mg as Second Dose in Phase 3 27 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 26: APeX-2: Phase 3 Trial Design
APeX-2: Phase 3 Trial Design 28 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 27: APeX-2: Phase 3 Trial Design – Safety Extension
APeX-2: Phase 3 Trial Design – Safety Extension 29 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 28: APeX-S: Long-term Safety Study Design
APeX-S: Long-term Safety Study Design 30 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 29: Phase 1/2 Study in Anti-PD-1 Refractory Melanoma
Phase 1/2 Study in Anti-PD-1 Refractory Melanoma 31 Phase 2 Expansion with Ipilimumab Enrolling Dosing: Tilsotolimod 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 interven
Slide 30: 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) 32
Slide 31: 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 33
Slide 32: Phase 2 Conclusions
Phase 1 Conclusions 34 The combination of tilsotolimod with ipilimumab was tolerable at all dose levels studied Dendritic cell activation, detectable within 24 hours of the first tilsotolimod injection, is evidence for target acquisition at the Recommended Phase 2 Dose (8mg) Tilsotolimod with ipilim
Slide 33: Phase 2 Expansion Update
Phase 2 Expansion Update 35 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 34: Phase 3 Readiness
Phase 3 Readiness (FPFV 1Q18) 36 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 commenced1Q18 Commercial presentatio
Slide 35: Growth/Partnering Opportunities
Growth/Partnering Opportunities 37 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 EXPAND TRANSFORM Unresectable metastatic Emerging VO ‘Cold” tumors unaddressable with addressable tumors. current UO melanoma + High need in ant-PD1- + Moderate response to + Significant opportunity refractory patients ‘comerstone anti-PD1 in tumors with + P
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