Biocryst Pharmaceuticals, Inc. · NASDAQ:BCRX
Biocryst Pharmaceuticals — Merger or Acquisition (February 2018)
34 slides from Biocryst Pharmaceuticals's merger presentation, Feb 22, 2018
Slide 1: Forward-looking statements
This press release contains forward-looking statements within the meaning of the federal securities law. Such statements are based upon current plans, estimates and expectations that are subject to various risks and uncertainties. The inclusion of forward-looking statements should not be regarded as
Slide 2: Additional information and participants in the solicitation
In connection with the proposed merger, Idera and BioCryst plan to file with the SEC and mail or otherwise provide to their respective stockholders a joint proxy statement/prospectus regarding the proposed transaction. BEFORE MAKING ANY VOTING DECISION, IDERA’S AND BIOCRYST’S RESPECTIVE STOCKHOLDERS
Slide 3: Combination Creates Substantial Value
A unique player in rare diseases with scale Diversified late-stage pipeline Synergistic potential with best-in-class people, facilities and commercial know-how in rare diseases Experienced development capabilities across organization Active and complementary discovery engines Financial strength Comb
Slide 4: 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 ~$243 million net cash balance* Opportunities to add cash through par
Slide 5: Phase 3 programs create a financially strong foundation
Phase 3 Programs Create a Financially Strong Foundation to Support a Robust Rare Disease Pipeline 6 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 at mo
Slide 6: Merger Upside: Maximizing Value and Market Potential
Merger Upside: Maximizing Value and Market Potential 7 Value Driver Merger Amplifier Commercializing BCX7353 Idera management HAE launch experience Investing in commercial launch and ongoing pipeline development Capital from IMO-2125 out licensing opportunities Expanding market opportunity + diversi
Slide 7: Maximizing Value, Minimizing Risk, Multiplying Opportunity
Maximizing Value, Minimizing Risk, Multiplying Opportunity 8 BCX7353 HAE Prophylaxis (Capsule) BCX7353 HAE Acute (Liquid) Gen II kallikrein inhibitors (HAE & Other Indications) BCX9250 BCX9499 (FOP) Small Molecule Rare Disease Discovery Engine IMO-2125 PD-1 Refractory Melanoma IMO-8400 Dermatomyosit
Slide 8: 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 9: Late-stage 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 10: 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 11: Combination Creates Opportunities for Innovative Medicines
Extensive experience in both discovery approaches within one organization Combining technologies expands ability beyond stand-alone Combination therapy of small molecule and oligo may create more effective and potent treatments for rare diseases Combination Creates Opportunities for Innovative Medic
Slide 12: Solid Capital Position & Meaningful Operational Synergies
~$243 million net cash balance* Capital for continued clinical development beyond next milestone events Commercial launch planning and preparation Additional $20+ million (non-dilutive) procurement contract anticipated in 2018 Opportunities to generate non-dilutive capital through non-strategic asse
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: Combining Capabilities to Serve More Patients with Rare Diseases
Extraordinary drug discovery, development and commercialization so patients can have a better quality of life + Combining Capabilities to Serve More Patients with Rare Diseases 15
Slide 15: Combination Creates Substantial Value
A unique player in rare diseases with scale Diversified late-stage pipeline Synergistic potential with best-in-class people, facilities and commercial know-how in rare diseases Experienced development capabilities across organization Active and complementary discovery engines Financial strength Comb
Slide 16: Combining Capabilities to Serve More Patients with Rare Diseases
Combining Capabilities to Serve More Patients with Rare Diseases +
Slide 17: 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 approximately 51.6% of combined company and Idera stockholders to
Slide 18: APeX-1: Dose Provided Consistent Reductions in Attack Rate
APeX-1: 125 mg Dose Provided Consistent Reductions in Attack Rate 20 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 19: APeX-1: Angioedema Quality of Life
APeX-1: Angioedema Quality of Life (AE-QoL): LS Mean Change from BL at Day 29, PP 21 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 20: 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 21: APeX-1: Exposure Comparisons of BCX7353 and SC C1INH
APeX-1: Exposure Comparisons of BCX7353 and SC C1INH 23 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 22: Predictable PK Supports Second Dose in Phase 3
Predictable PK Supports 175 mg as Second Dose in Phase 3 24 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 23: APeX-2: Phase 3 Trial Design
APeX-2: Phase 3 Trial Design 25 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 o 32 BCX7353 150 mg QD* Screening Visit Screening / Run-in Blinded Treatment 24 weeks
Slide 24: APeX-2: Safety Extension
APeX-2: Phase 3 Trial Design – Safety Extension 26 N o 32 BCX7353 150 mg QD* Screening / Run-in Blinded Treatment 24 weeks N o 32 BCX7353 110 mg QD* N o 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
Slide 25: APeX-S: Long-term Safety Study Design
APeX-S: Long-term Safety Study Design 27 Endpoints: Long term safety of BCX7353 Durability of response Quality of Life APeX-1 subjects eligible N o 80 BCX7353 150 mg QD 48 Weeks Treatment N o 80 BCX7353 110 mg QD *Doses in Phase 2 APeX-1 were shown as the dihydrochloride salt: 150 mg = 175 mg dihydr
Slide 26: Phase 1/2 Study in Anti-PD-1 Refractory Melanoma
Phase 1/2 Study in Anti-PD-1 Refractory Melanoma 28 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 27: Time on Study: Best RECIST Response
Time on Study: Best RECIST v1.1 Response and Largest Percentage Decrease in Target Lesions (8mg subjects) 29
Slide 28: 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 30
Slide 29: Phase 1/2 Conclusions
Phase 1 Conclusions 31 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 30: Phase 2 Expansion Update
Phase 2 Expansion Update 32 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 31: Phase 3 Trial Design
Phase 3 Trial Design 33 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 32: Phase 3 Readiness
Phase 3 Readiness (FPFV 1Q18) 34 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 33: Growth/Partnering Opportunities
Growth/Partnering Opportunities 35 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 34: Growth/Partnering Opportunities
Growth/Partnering Opportunities INTRODUCE EXPAND TRANSFORM Unresectable metastatic “Cold” tumors unaddressable with current /O Emerging 0 ressable tumor: pris addressable tumors. + High need in anti-PD1- + Moderate response to + Significant opportunity refractory patients ccomerstone anti-PD1 in tum
BioCryst Pharmaceuticals — Merger or Acquisition (February 2018) on 6ix