January 2017 J.P. Morgan 36 th Annual Management Healthcare Presentation Conference (San Francisco, CA) January 8, 2018
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TOPICS FOR DISCUSSION OUR LEAD ASSET: AVATROMBOPAG LAUNCH READINESS PLAN AVATROMBOPAG VS. LUSUTROMBOPAG DOVA PIPELINE FINANCIALS
AVATROMBOPAG: THE OPPORTUNITY TO UNIQUELY CHANGE THE WAY CHRONIC LIVER DISEASE PATIENTS ARE BEING MANAGED TODAY CHRONIC LIVER DISEASE PTS. with thrombocytopenia prior to a procedure ROBUST EFFICACY Superiority consistently demonstrated across two Phase 3 trials (p<0.0001 to p=0.0006) with AE profile similar to placebo 5-DAY DOSING 3-5 days to platelet production following initial dose* DOUBLES PLATELET COUNT* 10-days after initial dose to 50K for most patients thus averting need for platelet transfusion * Based on data from clinical trials. These are not labelled claims since avatrombopag has not yet been approved by the Food and Drug Administration
CLINICALLY SIGNIFICANT RISKS AND HIGH COSTS MAY LIMIT OR PREVENT THE USE OF PLATELET TRANSFUSION RISKS $9,000 ESTIMATED TRANSFUSION COSTS RISK OF ANTIBODY DEVELOPMENT May lead to refractoriness in up to ~50% of patients INCONVENIENT ADMINISTRATION Administration time AE management SHORT DURATION OF EFFECT AND LIMITED SUPPLY Short life of platelets Transfusion must be given same day as procedure INFECTION & ADVERSE REACTIONS Immune reaction Febrile non-hemolytic reactions Sepsis AE MGMT $1,500 (17%) OVERHEAD $1,500 (17%) LABOR $3,000 (33%) PLATELETS $3,000 (33%) Note: Company derived estimates based on third party sources PATIENTS WILL RESPOND, BUT YOU RISK INFECTIONS AND SIGNIFICANT PLATELET VOLUME HEPATOLOGIST, EU THE PROBLEM WITH PLATELET TRANSFUSIONS IS THE PATIENTS TEND TO BECOME REFRACTORY HEPATOLOGIST, EU
PHYSICIANS EXERCISE DISCRETION REGARDING PLATELET COUNT LEVELS REQUIRED PRIOR TO A PLANNED MEDICAL PROCEDURE 70K - 210K* SCHEDULED PROCEDURES FOR CHRONIC LIVER DISEASE PATIENTS WITH PLATELET COUNTS < 50,000 / µl 60%** RECEIVE PLANNED TRANSFUSIONS NO APPROVED THERAPEUTIC AGENT FOR THE TREATMENT OF THROMBOCYTOPENIA PRIOR TO PLANNED PROCEDURES *Source: one procedure/patient/year from single center database analysis. two procedures/patient/year from Oliver Wyman Understanding Barriers to Entry Ahead of the Curve, May 2012. three procedures/patient/year from IMS Health Acute Thrombocytopenia in Chronic Liver Disease Assessment. **Source: Triangle Insights Group, results of n=155 quantitative responses. Conducted May 2017
AVATROMBOPAG NDA HAS RECEIVED FDA PRIORITY REVIEW THE REVIEW CLASSIFICATION FOR THIS APPLICATION IS PRIORITY. THEREFORE, THE USER FEE GOAL IS MAY 21, 2018-11/21/2017
OUR LEAD ASSET: AVATROMBOPAG LAUNCH READINESS PLAN AVATROMBOPAG VS. LUSUTROMBOPAG DOVA PIPELINE FINANCIALS
A COMPREHENSIVE MEDICAL AND COMMERCIAL APPROACH TO BECOME THE NEW STANDARD OF CARE EDUCATE HEPATOLOGIST 10 PERSON EXPERIENCED MSL TEAM DEDICATED TO COMMUNICATING RISKS OF CURRENT STANDARD OF CARE AND CLINICAL DATA ON AVATROMBOPAG INFLUENCE HEAD OF SALES HIRED BEGINNING TO HIRE A 30-PERSON SALES FORCE STARTING IN JANUARY DOVA REIMBURSEMENT SUPPORT CENTER
A COMPREHENSIVE MEDICAL AND COMMERCIAL APPROACH TO BECOME THE NEW STANDARD OF CARE SPECIALTY PHARMACY DISTRIBUTORS HEPATOLOGIST DOVA REIMBURSEMENT SUPPORT CENTER MANAGED CARE ORG. 5 PERSON ACCOUNT TEAM FOCUSED ON DRIVING FORMULARY ADOPTION
OUR LEAD ASSET: AVATROMBOPAG LAUNCH READINESS PLAN AVATROMBOPAG VS. LUSUTROMBOPAG DOVA PIPELINE FINANCIALS
PATIENTS (%) PATIENTS (%) COMPARISON OF AVATROMBOPAG IN PATIENTS WITH BASELINE PLATELET COUNT < 35K ACHIEVING PRIMARY ENDPOINT PROPORTION OF PATIENTS WHO DID NOT REQUIRE PLATELET TRANSFUSION OR ANY RESCUE PROCEDURE FOR BLEEDING ADAPT-1 / ADAPT-2 (<40K / µl) ADAPT-1 / ADAPT-2 ( 40K / µl to <50K / µl) ADAPT-1 / ADAPT-2 (<35K / µl) P < 0.0001 88% P < 0.0001 67% P < 0.0001 63% 28% 36% 27% PLACEBO AVATROMBOPAG PLACEBO AVATROMBOPAG PLACEBO AVATROMBOPAG *Terrault N, et al. AASLD; 2017 Oct 20-24; Washington DC.
AVATROMBOPAG S DOSING SCHEDULE IN PHASE 3 TRIALS COMPARED TO LUSUTROMBOPAG <50K PHYSICIAN DISCRETION AVATROMBOPAG PHASE 3* (ADAPT-1 & ADAPT-2) 5-DAY DOSING (40MG. OR 60 MG.) 5-DAY WAIT PROCEDURE (DAY 10-13) >50K <50K <50K PHYSICIAN DISCRETION LUSUTROMBOPAG PHASE 3** (L-PLUS-2) 5-DAY DOSING (3 MG.) <50K DAY 6 DOSING 3-DAY WAIT PROCEDURE (DAY 9-14) DAY 7 DOSING 2-DAY WAIT >50K AND 20K OVER BASELINE PROCEDURE (DAY 9-14) 1-DAY WAIT >50K AND 20K OVER BASELINE PROCEDURE (DAY 9-14) >50K AND 20K OVER BASELINE *Terrault N, et al. AASLD; 2017 Oct 20-24; Washington DC **Afdhal N, et al. AASLD ; 2017 Oct 20-24; Washington DC.
OUR LEAD ASSET: AVATROMBOPAG LAUNCH READINESS PLAN AVATROMBOPAG VS. LUSUTROMBOPAG DOVA PIPELINE FINANCIALS
AVATROMBOPAG: POTENTIAL TO ADDRESS VARIOUS TYPES OF THROMBOCYTOPENIA Acute treatment of thrombocytopenia Thrombocytopenia in CLD patients scheduled to undergo a procedure 40,000 125,000 platelet transfusions Thrombocytopenia in broader population of patients scheduled to undergo surgery (1) 125,000 platelet transfusions Chemotherapy-Induced Thrombocytopenia (CIT) 125,000 platelet transfusions PRECLINICAL PHASE 1 PHASE 2 PHASE 3 NEXT MILESTONE PDUFA: May 21, 2018 First Patient To Be Enrolled: Q1 2018 First Patient To Be Enrolled: Q2 2018 Chronic treatment of thrombocytopenia Immune Thrombocytopenic Purpura (ITP) snda to be submitted: H2 2018 1 Surgery includes spectrum of minimally invasive to highly invasive medical procedures. For highly invasive surgeries such as vascular, cardiac, brain or spine surgeries, many medical professional association guidelines recommend that patients have at least 100K platelets / µl
PLANNED INVASIVE SURGICAL PROCEDURES STUDY DESIGN Multi-center, open-label study in patients with thrombocytopenia undergoing invasive surgery (will select 2-3 surgical types) requiring a platelet count >100K / µl N = 60-90 PRIMARY ENDPOINT SCREENING TREATMENT DAY 1-5 PROCEDURE DAY 10-13 Proportion of subjects achieving a platelet count of 100 / µl on Procedure Day, without transfusion MEAN BASELINE PC 50 to <100K 60MG AVATROMBOPAG 5 8 DAYS AFTER LAST DOSE OF STUDY DRUG SECONDARY ENDPOINTS Change from baseline platelet count to Procedure Day Proportion of subjects without platelet transfusion and any other treatment for bleeding after the Baseline Visit and up to 7 days following Procedure Day INTERIM DATA REVIEWS AT N=15 AND N=30 TO ASSESS DOSE SELECTION
CHEMOTHERAPY-INDUCED THROMBOCYTOPENIA (CIT) Confidential & Proprietary STUDY DESIGN Phase 3 randomized, double-blind, placebo-controlled study of the efficacy and safety of oral avatrombopag in subjects with active non-hematological cancers (i.e., ovarian, NSCLC) who develop CIT (platelet count <50K / µl in the previous cycle of chemo) N = 120 (2:1 RDZ) CYCLE X +1 CYCLE X +2 SCREENING PC < 50K DURING CYCLE X OF CHEMO TREATMENT DAY 1-5 60MG AVA PLACEBO CHEMO DAY TREATMENT DAY 1-5 60MG AVA PLACEBO STUDY DRUG NOT ADMINISTERED: OBSERVATION CYCLE ONLY TO DETERMINE OUTCOME OF PRIMARY ENDPOINT
POTENTIAL FOR EXPANSION INTO MORE INVASIVE SURGICAL PROCEDURES AND CHEMOTHERAPY- INDUCED THROMBOCYTOPENIA (CIT) 50% OF PLATELET TRANSFUSIONS ARE ADMINISTERED IN AN EMERGENT SETTING 1.2 MM PLATELET TRANSFUSIONS EVERY YEAR IN THE U.S. Total Addressable Market ~$2.5 BILLION PLATELET TRANSFUSIONS FOR NON-EMERGENT PROCEDURES ~125K PLATELET TRANSFUSIONS FOR MORE INVASIVE PLANNED SURGICAL PROCEDURES* ~125K PLATELET TRANSFUSIONS FOR CHEMOTHERAPY-INDUCED THROMBOCYTOPENIA (CIT)* * Figures are estimated based on historical data from third-party sources
AVATROMBOPAG: POTENTIAL TO ADDRESS VARIOUS TYPES OF THROMBOCYTOPENIA Acute treatment of thrombocytopenia Thrombocytopenia in CLD patients scheduled to undergo a procedure 40,000 125,000 platelet transfusions Thrombocytopenia in broader population of patients scheduled to undergo surgery (1) 125,000 platelet transfusions Chemotherapy-Induced Thrombocytopenia (CIT) 125,000 platelet transfusions PRECLINICAL PHASE 1 PHASE 2 PHASE 3 NEXT MILESTONE PDUFA: May 21, 2018 First Patient Enrolled: Q1 2018 First Patient Enrolled: Q2 2018 Chronic treatment of thrombocytopenia Immune Thrombocytopenic Purpura (ITP) $600 Million (U.S.) / $1.2 Billion WW snda to be filed H2 2018 based on completed study data 1 Medical procedure refers to a non-emergent, minimally to moderately invasive medical procedure 2 Surgery includes spectrum of minimally invasive to highly invasive medical procedures. For highly invasive surgeries such as vascular, cardiac, brain or spine surgeries, many medical professional association guidelines recommend that patients have at least 100K platelets / µl
AVATROMBOPAG HAS BEEN STUDIED IN 128 ITP PATIENTS WITH AN AVERAGE DURATION OF SEVEN MONTHS TWO PHASE 2 STUDIES PHASE 3 STUDY 49 RANDOMIZED 17 PLACEBO 32 AVATROMBOPAG 16 DC ED 10 DC ED 1 COMPLETE 22 COMPLETE
PHASE 3 STUDY OF AVATROMBOPAG IN ITP: CUMULATIVE NUMBER OF WEEKS OF PLATELET RESPONSE > 50K / µl 15 P < 0.0001 12 WEEKS 10 WEEKS 5 0 0 WEEKS PLACEBO (N = 17) AVATROMBOPAG (N = 32)
PHASE 3 STUDY OF AVATROMBOPAG IN ITP PLACEBO (N = 17) AVATROMBOPAG (N = 32) PLATELET COUNT > 50K / µl AT DAY 8 0 (0%) 21 (66%) P < 0.0001 REDUCTION OF CONCOMITANT ITP MEDICATION 0 (0%) 5 (33%) P - NS DURABLE PLATELET RESPONSE (PLATELET RESPONSE FOR 6 OF THE LAST 8 WEEKS OF TREATMENT) 0 (0%) 11 (34%) P = 0.009 All measures at a 95% confidence interval
THE US citp MARKET IS $600 MILLION GROWING AT 15% A YEAR. GLOBALLY, THE MARKET IS ESTIMATED AT $1.2 BILLION. 12,000 MONITOR 21,000 STABLE 60,000 ADULTS 48,000 FIRST LINE THERAPY 27,000 SECOND LINE THERAPY 16,000 STABLE / MORTALITY 11,000 THIRD LINE THERAPY Source: U.S. PROMACTA Research and Analysis (February 2015); Putnam Analysis, GSK Forecast model
THE CHRONIC ITP SEGMENT IS A $1.2 BILLION MARKET AND AVATROMBOPAG IS DIFFERENTIATED FROM THE OTHER TWO MAIN PLAYERS HEPATOTOXICITY DOSING FOOD EFFECT AVATROMBOPAG NO HEPATOTOXICTY ORAL DOSING CONVENIENT ADMINISTRATION WITH FOOD MAY INCREASE THE RISK OF SEVERE HEPATOTOXICITY. MONTHLY LIVER FUNCTION TESTING REQUIRED ORAL DOSING ABSORPTION IMPACTED BY FOOD NO HEPATOTOXICTY SUBCUTANEOUS N/A Note: Avatrombopag s proposed indication is acute treatment of thrombocytopenia for patients with chronic liver disease undergoing procedures; Promacta s indication is chronic ITP, pediatric chronic ITP, severe aplastic anemia and chronic hepatitis C; Nplate s indication is chronic ITP Avatrombopag has not yet been approved by the FDA. This represents studies completed to date.
OUR LEAD ASSET: AVATROMBOPAG LAUNCH READINESS PLAN AVATROMBOPAG VS. LUSUTROMBOPAG DOVA PIPELINE FINANCIALS
DOVA PHARMACEUTICALS WILL POTENTIALLY BEGIN GENERATING REVENUE IN THE SECOND HALF OF 2018 $100 MILLION CASH AND CASH EQUIVALENTS (AS OF 9/30) $30 MILLION (NOTE PAYABLE TO EISAI: DUE 3/30/18) $6.3 MILLION OPERATING CASH BURN (NINE MONTHS ENDED 9/30/2017). ANTICIPATED BURN WILL INCREASE WITH COMMERCIAL ACTIVITIES
LATE STAGE ASSET WITH SIGNIFICANT MARKET POTENTIAL 2018 POTENTIAL PRODUCT LAUNCH ROBUST PIPELINE LARGE US MARKET OPPORTUNITY FAVORABLE IP STRONG CASH POSITION May 21 st 2018 PDUFA date Well- differentiated versus competition Surgery and CIT Phase 3 studies to commence in H1 2018 snda for ITP in H2 2018 MAA filing expected in H1 2018 $800 Million in first indication $1.7 Billion in follow-on acute indications $600 Million in ITP ($1.2 Billion WW) Composition of matter patents expire in 2025 with potential patent term extension to 2030 $100 million in cash (as of 9/30/17) Potentially revenue generating in H2 2018