2020 Spectrum Corporate Deck June
Transcript of 2020 Spectrum Corporate Deck June
June 2020 | Investor Presentation
Spectrum Pharmaceuticals
A Biopharmaceutical Company Developing Targeted and Novel Therapies in Oncology
Joe Turgeon | President and CEO
2
Safe Harbor StatementThis presentation contains forward-looking statements regarding future events and the future performance of Spectrum Pharmaceuticals that involve risks and uncertainties that could cause actual results to differ materially. These statements are based on management’s current beliefs and expectations. These statements include but are not limited to statements that relate to our business and its future, our strategy, the success of our drug candidates, the safety and efficacy of our drug products, product approvals, market potential, product sales, revenue, development, regulatory and approval timelines, product launches, product acquisitions, capital resources and any statements that relate to the intent, belief, plans or expectations of Spectrum or its management, or that are not a statement of historical fact.
Risks that could cause actual results to differ include the possibility that our existing and new drug candidates may not prove safe or effective, the possibility that our existing and new drug candidates may not receive approval from the FDA and other regulatory agencies in a timely manner or at all, the possibility that our existing and new drug candidates, if approved, may not be more effective, safer or more cost efficient than competing drugs, the possibility that price and other competitive pressures may make the marketing and sale of our drugs not commercially feasible, the possibility that our efforts to acquire or in-license and develop additional drug candidates may fail, our lack of sustained revenue history, our limited experience in establishing strategic alliances, our limited marketing experience, our customer concentration, the possibility for fluctuations in customer orders, evolving market dynamics, our dependence on third parties for clinical trials, manufacturing, distribution, information and quality control and other risks that are described in further detail in the Company's reports filed with the Securities and Exchange Commission. We do not plan to update any such forward-looking statements and expressly disclaim any duty to update the information contained in this presentation except as required by law.
3
Targeted & Novel MedicinesSpectrum’s Pipeline
Focused InterferonTherapeutics (FIT)Phase 1 Dose Escalation Study
POZIOTINIB
Cohort 2 Results Mid-2020
ROLONTIS®
(eflapegrastim)
PDUFA Date of October 24, 2020
4
ROLONTIS® (eflapegrastim)
BLA has been accepted with a PDUFA of Oct 24th 2020
5
ROLONTIS is a Novel Product with a Unique Molecular Structure
6
Robust Data Set Features Head to Head Data vs Neulasta
* Non-inferiority of ROLONTIS versus pegfilgrastim in Duration of Severe Neutropenia in Cycle 1** 95% confidence intervals and p-value obtained from bootstrap sampling method
ADVANCE (-301) RECOVER (-302)
ROLONTISN=196
PegfilgrastimN=210
ROLONTISN=118
PegfilgrastimN=119
DSN (days)Mean (SD)Median (Range)
0.20 (0.503)0 (0, 3)
0.35 (0.683)0 (0, 3)
0.31 (0.688)0 (0,3)
0.39 (0.949)0 (0,7)
Δ DSN (SPI-2012 –pegfilgrastim)95% Confidence Interval**Non-inferiority p-value**
-0.148(-0.264, -0.032)
<0.0001
-0.074(-0.292, 0.129)
<0.0001
643 Patients in Two Phase 3 Trials
Met Primary
Endpoint*
Met All Secondary Endpoints
Similar AE Profile
7
Integrated Phase 3 Results Presented at ASCO 2019
Incidence of Severe Neutropenia from Two Phase 3 ROLONTIS Trials
8
Long Acting G-CSF Market Presents a Compelling Opportunity
Customer Behavior
Reimbursement Dynamics
Market Size
>$3B total US market value Rational pricing behavior among competitors NCCN Guidelines recently revised to recommend
increased use of G-CSF
Willingness to change products based on value Onpro® volume declining with pre-filled syringe growth Providers receptive to incremental contracting opportunities
Unique reimbursement for novel products is a competitive advantage
Value based care creates additional complexity in customer’s decision matrix
Commercial payers increasingly important stakeholders in decision-making
Neulasta 73%
Udenyca, 21%
Fulphila, 6%
Estimated LA-GCSF Market Share: Current 3 months through Jan 2020
Market Landscape
9
Pricing: Q2 2020
Neulasta WAC $6,231
Neulasta ASP$3,758
Biosimilars ASPs are 3-8% below
Neulasta
Biosimilars ASP
10
Poziotinib
Tyrosine Kinase Inhibitor targeting mutations in lung
cancer
Results from ZENITH20 cohort 2 in mid-2020
Results from ZENITH20 cohort 3 in 2H 2020
11
Unique Structure Provides Irreversible Binding
Poziotinib
Even with the restricted binding pocket of exon 20 mutations, preclinical studies have shown that poziotinib is able to overcome steric hindrance allowing for irreversible binding
12
Cohort 1 Results
Best Overall Response Intent to treat (N=115)N (%)
Objective Response Rate (ORR) by independent review committee (IRC)95% Confidence Interval
17 (14.8%)(8.9 - 22.6%)
Disease Control Rate (DCR=CR+PR+SD)95% Confidence Interval
79 (68.7%)(59.4 - 77.0%)
Duration of Response, Median (months)95% Confidence Interval
7.4(3.7, 9.7)
Progression-free Survival, Median (months)95% Confidence Interval
4.2(3.7, 6.6)
13
ZENITH 20 Cohort 1 Waterfall PlotBest Percent Change from Baseline in Sum of Longest Diameter by Treatment Group
*Indicates subject is ongoing treatment
14
Safety: Treatment-related Adverse Events
Drug interruption rate 88%
Dose reduction rate 68%
Permanent discontinuation due to
treatment-related AE = 12 (10%)
No Grade 5 treatment related AE
Most common treatment-related
AEs (any grade) include:
Rash
Diarrhea
Stomatitis
Paronychia
Treatment-related AEN=115
Any Grade n (%)
Grade 3 n (%)
Grade 4 n (%)
Any TRAE 114 (99) 70 (61) 2 (2)Diarrhea 91 (79) 29 (25) 1 (1)Rash 69 (60) 32 (28) 0Stomatitis 60 (52) 10 (9) 0Paronychia 52 (45) 7 (6) 0Nausea 44 (38) 3 (3) 0Decreased appetite 36 (31) 2 (2) 0Mucosal inflammation 34 (30) 8 (7) 0Dry skin 33 (29) 3 (3) 0Vomiting 33 (29) 1 (1) 0Alopecia 31 (27) 0 0Dermatitis acneiform 30 (26) 8 (7) 1 (1)Fatigue 30 (26) 6 (5) 0Pruritus 29 (25) 5 (4) 0
15
Efficacy: Duration of Response
Response ProgressionTreatment ongoing
16 mg 14 mg12 mg10 or less
0 28 56 84 112 140 168 196 224 252 280 308 336 364 392
Days
Median duration of response:
7.4 months
(95% CI 3.7,9.7)
Responses occurred early and were durable
16
Simulated Poziotinib Plasma Concentrations8 mg BID vs. 16 mg QD
Pozi plasma ½ life is 7.9 hours
BID dosing
Decreases Cmax
Maintains Ctroughabove IC50
Pozi IC50 4nM (2ng/ml)-T790m
00
10
20
30
40
50
60
70
Subject US010-002(Simulated Concentrations)
Time (Days)1 2 3 4 5
16 mg QD8 mg Q12H
IC50
17
ZENITH20: Continued Enrollment with New Cohorts
Key Eligibility Criteria
NSCLC EGFR or HER2 exon20 insertions
Point mutations, including T790M, are not allowed
Brain mets are allowed if stable
Cohort 1EGFR - previously treated
Cohort 2HER2 - previously treated Primary Endpoint
Objective Response Rate (RECIST 1.1)
Secondary Endpoints Duration of
response Disease control rate PFS (exploratory) Safety
NSCLC patients with EGFR or HER2 Mutations
Cohort 3EGFR – treatment naive
Cohort 4HER2 – treatment naive
16mg QD
Cohort 5EGFR or HER2 exon 20
Cohort 6EGFR osimertinib failure
Cohort 7Atypical EGFR or HER2
mutations
8mg BID
Osimertinib-resistant with
EGFR mutationsAtypical EGFR or HER2
mutations
Randomized to 10mg QD, 6mg BID, 8mg BID
Oral daily dose 28-day cycle
8mg BID
18
Exon 20 Mutations in Various Tumor Types
Prevalence of Exon20 NSCLC in Key Markets
Region Mutation Exon 20 Frequency (%)
Total Number of Exon-20 NSCLC Patients/year
USEGFR 2.1%
3.6% 7,700HER2 1.5%
EUEGFR 2.1%
3.6% 8,000HER2 1.5%
JapanEGFR 2.4%
6.4% 5,000HER2 3.9%
TOTAL Patients 20,700
Prevalence of Exon20 In Other Tumors
Region MutationExon 20
Frequency (%)
Total Exon 20 (non-Lung) Patients/year
US
EGFR 3710 (0.2%)
0.6% 8,400
HER2 4691 (0.4%)
Heymach J. Phase II trial of poziotinib for EGFR and HER2 exon 20 mutant NSCLC Presented at IASLC 19th World Conference on Lung Cancer; Sept. 2018 Toronto CAN
N= 390,000 patients
Total US Patients 8,400Total Global Patients 20,700
19
Focused Interferon Therapeutics
IFN is an approved treatment for cancer
But systemic IFN therapy has limitations due to dose limiting toxicity
Focused IFN Therapeutics (FIT) Technology seeks to overcome the toxicity while maintaining efficacy
By attaching IFN to an antibody, FIT targets delivery of IFN to tumor microenvironment
FIT Platform
Targeted Antibody-InterferonFusion Technology
FIT – Fusion of Interferon (IFNα) and Targeted Antibodies
Unaltered binding to Fc receptor
Growth inhibition Turn cold tumors into HOT Direct Apoptosis
Proteolysis-resistant short peptide LINKER
Interferon(IFN)
20
21
Mechanism of Action
NO Apoptosis/Growth arrest
Ab-IFN binds poorly to IFN-R
No IFN-Rsignaling
IFN Target antigen
Antibody does not bind cell surface antigen
Normal Cells without Antibody Target
Apoptosis/Growth arrest
1
Antibody binds target
IFN binds IFN-R
IFN-Rsignaling
IFN Target antigen
32
Tumor Cells with Antibody Target
22
Lead Asset in Phase 1 Study
Phase 1, Open-Label, Dose-Escalation Study in Subjects with Refractory Non-Hodgkin Lymphoma⁻ Evaluate the Safety, Tolerability, and PK of Multiple IV Doses with Weekly
Administration⁻ Planned for up to 20 subject at maximum tolerated dose
Primary Outcome Measures⁻ Safety and tolerability (weekly for 6 months)⁻ Determine the maximum tolerated dose
Secondary Outcome Measures⁻ Characterize PK/PD profile of ascending doses⁻ Anti-tumor activity
Clinical Trial Sites⁻ UCLA, University of Florida, MD Anderson Cancer Center
23
Targeted & Novel MedicinesSpectrum’s Pipeline
Focused InterferonTherapeutics (FIT)Phase 1 Dose Escalation Study
POZIOTINIB
Cohort 2 Results Mid-2020
ROLONTIS®
(eflapegrastim)
PDUFA Date of October 24, 2020
24
Thank you
24
Non-GAAP Financial Measures (from Continuing Operations)
In this press release, Spectrum reports certain historical results that have not been prepared in accordance with generally accepted accounting principles (GAAP), including non-GAAP selling, general and administrative expenses, non-GAAP research and development expenses, non-GAAP net loss and non-GAAP net loss per share. Non-GAAP financial measures are reconciled to the most directly comparable GAAP financial measures in the tables of this press release and the accompanying footnotes. The non-GAAP financial measures contained herein are a supplement to the corresponding financial measures prepared in accordance with GAAP. The non-GAAP financial measures presented exclude the items summarized in the below table.
Management believes that adjustments for these items assist investors in making comparisons of period-to-period operating results and that these items are not indicative of the company's on-going core operating performance. Management uses non-GAAP net income (loss) in its evaluation of the company's core after-tax results of operations and trends between fiscal periods and believes that these measures are important components of its internal performance measurement process. Management believes that these non-GAAP financial measures are useful to investors in providing greater transparency to the information used by management in its operational decision-making. Management believes that the use of these non-GAAP financial measures also facilitates a comparison of the company’s underlying operating performance with that of other companies in its industry, which use similar non-GAAP measures to supplement their GAAP results.
The non-GAAP financial measures presented herein have certain limitations in that they do not reflect all of the costs associated with the operations of the company's business as determined in accordance with GAAP. Therefore, investors should consider non-GAAP financial measures in addition to, and not as a substitute for, or as superior to, measures of financial performance prepared in accordance with GAAP. In addition, other companies, including other companies in our industry, may calculate non-GAAP financial measures differently than we do, limiting their usefulness as a comparative tool. Investors and potential investors are encouraged to review the reconciliation of our non-GAAP financial measures contained within this news release with our GAAP financial results.
25
26
27
28