NYSE American: PFNX Corporate Presentationpfenex.investorroom.com/download/Pfenex+Corporate... ·...
Transcript of NYSE American: PFNX Corporate Presentationpfenex.investorroom.com/download/Pfenex+Corporate... ·...
Corporate PresentationNYSE American: PFNX
Safe Harbor StatementPfenex
This presentation (the “Presentation”) includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, which are based on current expectations, estimates and projections based on information currently available to management. These forward-looking statements include, among others, statements regarding the future potential of Pfenex's product candidates, including future plans to advance, develop, manufacture and commercialize its product candidates; Pfenex’s plan to execute on its strategy, including to focus on and deliver key programs, selectively expand its partnered portfolio, increase its business development focus, and targeted and lean execution; potential market opportunities for PF582, PF708, PF529, PF690, Px563L/RPA563 and Pfenex’s other product candidates; Pfenex's expectation to receive data from the PF708 clinical program in the first half of 2018 and its expectation to submit an NDA in the third quarter of 2018; the expected patent expiration timelines for Lucentis, Forteo, and other branded reference drugs; developments and projections relating to competitors and the industry, including support of the US administration for development of medical countermeasures; expected milestones for Px563L/RPA563, including the timing of the Phase 2 study initiation, options triggered by the U.S. Government to fund the progression of manufacturing, non-clinical development, and preparatory activities for the Phase 2 trial; the potential of Pfenex’s anthrax vaccine candidates to fulfill the strategic national stockpile, including the potential of Pfenex to obtain a procurement contract if within 10 years of FDA approval; expectations with regard to future milestone and royalty payments from Pfenex’s collaboration with Jazz Pharmaceuticals; expectations with respect to Pfenex’s ability to receive future payments under its government contracts; Pfenex’s expectations regarding the use of abbreviated regulatory pathways for the approval of its product candidates, including use of the 505(b)(2) regulatory pathway for PF708 and the 351(k) pathway for PF529; Pfenex's expectations regarding the timing and advancement of clinical trials and the types of future clinical trials for its product candidates, including PF708 and Px563L/RPA563; and Pfenex's expectation for potential strategic partnership opportunities for advancement of PF582, PF529, and its other product candidates. Forward-looking statements are typically identified by words like “believe,” “anticipate,” “could,” “should,” “estimate,” “expect,” “intend,” “plan,” “project,” “will,” “forecast,” “budget,” “pro forma,” and similar terms. Factors that could cause the Company’s results and expectations to differ materially from those expressed in forward-looking statements include, without limitation, our need for additional funds to support our operations; our success being dependent on PF708, Px563L/RPA563, and our other product candidates; our reliance on our collaboration partner’s performance over which we do not have control; failure to achieve favorable results in later clinical trials for PF708, Px563L/RPA563, or our other product candidates or receive regulatory approval; delays in our clinical trials or in enrollment of patients in our clinical trials; failure to market PF708, Px563L, or our other product candidates due to the existence of intellectual property protection owned or controlled by a third party and directed to PF708, Px563L/RPA563, or our other product candidates; PF708, Px563L/RPA563 and our other product candidates may cause serious adverse side effects or have properties that delay or prevent regulatory approval or limit their commercial profile; if approved, risks associated with market acceptance, including pricing and reimbursement; our ability to enforce our intellectual property rights; adverse market conditions; and changes to laws and government regulations involving the labelling, approval process, funding and other matters affecting biosimilars, therapeutic equivalents to branded products and vaccines. Forward-looking statements represent our management’s beliefs and assumptions only as of our November 9, 2017 press release announcing results for the quarter ended September 30, 2017. You should read our Annual Report on Form 10-K for the year ended December 31, 2016, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2017 and our subsequent reports filed with the SEC, including the Risk Factors set forth therein, completely and with the understanding that our actual future results may be materially different from what we expect. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.
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► Pfenex Inc. is a clinical-stage biotechnology company developing complex therapeutic proteins. Using the patented Pfenex Expression Technology® platform, the company has created an advanced pipeline of therapeutic equivalents, vaccines, biologics and biosimilars
► Extensive competencies from early R&D to regulatory filing and partnership management
► San Diego-based, listed on NYSE American: PFNX
OverviewPfenex
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Proven success developing complex therapeutic proteins with Pfenex Expression Technology™ platform
Proven Platform
Near-term Catalysts
Strong Management
PF708 top-line immunogenicity data readout expected 1H18, anticipated NDA submission in 3Q18; Anthrax vaccine candidates developed under contract with the US Government moving towards potential Phase 2 study initiation end ‘18
Recently appointed Evert (Eef) Schimmelpennink as CEO; added Jason Grenfell-Gardner (Chairman) and Sigurdur (Siggi) Olafsson to Board of Directors
PfenexCompany Overview
Attractive Portfolio Differentiated portfolio of therapeutic equivalents, vaccines, biologics and biosimilars
Scalable Technology
Opportunity to select additional partnered and wholly owned products for development up to various stages in cycle
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Evert (Eef) Schimmelpennink(Aug 2017)CEO, President and Secretary
Patrick K. LucyChief Business Officer
Hubert ChenChief Medical & Scientific Officer
Seni
or
Man
agem
ent
Boar
dof
Dire
ctor
s
PfenexNew and Established Leadership Aligned to Drive Growth
Jason Grenfell-GardnerMay 2017 John TaylorDennis Fenton Philip SchneiderRobin Campbell
Mayda MercadoVice President, Quality
Sigurdur OlafssonMay 2017
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Focus on and deliver key programs
Increase business development focus
Clear Strategy and Streamlined Execution
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Selectively expand Pfenex Expression Technology-based partnered portfolio
Targeted and lean execution
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Building on Core Competencies
Platform and Products in Development
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1 2 3Advanced Strain Engineering Accelerated Development Robust Analytics
Our proprietary protein production platform, based on Pseudomonas fluorescens, allows for rapid, high quality production and analysis of difficult-to-produce proteins.
PfenexExpression Technology Platform
► Proven success with Pfenex Expression Technology™
► Worked with 60+ biopharmaceutical companies with our protein expression platform
► 80% success rate where other previously-used systems failed
► Therapeutic equivalents, vaccines, biologics and biosimilars
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Products in DevelopmentPipeline Highlights
PF7081
Teriparatide (Forteo®)PF582Ranibizumab (Lucentis®)
PF529Pegfilgrastim (Neulasta®)
PF690Pegaspargase (Oncaspar®)
Jazz PartnershipMultiple Hematology Products
REGULATORYSUBMISSION
PRECLINICAL PHASE 1 PHASE 2
PX563LAdjuvanted Anthrax Vaccine
EXISTING USGTHIRD-PARTY
ANTHRAX VACCINE CONTRACT
5 YEARS - $911MM3RPA563Non-Adjuvanted Anthrax Vaccine
FULLY FUNDED BYUS GOVERNMENT
POTENTIAL PROCUREMENT
$1.5B
REFERENCE DRUG 2016 GLOBAL SALES2
$3.2B
$4.6B
$0.2B
INITIAL | CONFIRMATORYUS FDA BIOSIMILARINITIAL ADVISORY
MEETING
PRECLINICAL ANALYTICAL SIMILARITY
COMPARATIVE CLINICAL STUDY
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► Forteo® (teriparatide) rDNA protein and pen combination product
► Indicated for treatment of high fracture risk osteoporosis
► 2016 global sales of $1.5B of reference drug4
► Pfenex has achieved:
► high titer protein production
► low cost of goods
► Functionally equivalent pen device
► Demonstrated bioequivalence in healthy subjects
Plas
ma
Terip
arat
ide
(pg/
mL)
Products in DevelopmentPF708: Therapeutic Equivalent Candidate to Forteo®
Mean ± Standard Deviation
Time (hr)
Bioequivalence results in healthy subjects
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► Ongoing phase 3 osteoporosis study
► Fully enrolled with 181 subjects
► Primary endpoint for immunogenicity
► Secondary endpoints in pharmacokinetics, bone mineral density and bone turnover markers
► Interim results indicate comparable PK profiles between PF708 and Forteo®
► Complements prior bioequivalence results in healthy subjects
Products in DevelopmentPF708: Therapeutic Equivalent Candidate to Forteo®
Mean ± Standard Deviation
Comparable PK results in osteoporosis patients
Osteoporosis study design
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► Key Upcoming Expected Milestones:
► Primary endpoint of immunogenicity anticipated in first half of 2018
► 505(b)(2) NDA filing expected second half 2018
► Latest expiry of Orange Book-listed teriparatide method of treatment, formulation and/or API patent expected in 2019 in US5
Products in DevelopmentPF708: Therapeutic Equivalent Candidate to Forteo®
Pfenex PF708
PF708 (teriparatide [rDNA] injection)
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► Awarded a BARDA contract in August 2015 of up to $143.5MM to fund advanced development
► Anthrax recombinant Protective Antigen (rPA) vaccine candidates:► Demonstrated long term stability
data from toxicology lot showing the maintenance of high purity at 5°C at 40 months.
► Phase 1a Day 70 analysis demonstrated that Px563L and RPA563 were well-tolerated and afforded immunogenicity protection with fewer doses than the currently licensed product
Products in DevelopmentPx563L/RPA563: Next Gen Anthrax Vaccine Candidates
0.03
0.3
3
30
0 14 28 42 56 70
PBO
10 mcg
50 mcg
80 mcg
0.03
0.3
3
30
0 14 28 42 56 70
PBO
10 mcg
50 mcg
80 mcg
Toxin Neutralizing Antibody NF50 Results
Toxi
n N
eutr
alizi
ng A
ntib
ody
NF 5
0 (G
eom
etric
Mea
n Ti
ter ±
95%
Con
fiden
ce In
terv
als)
Time (Day)
Time (Day)
RPA563
Px563L
REGULATORY THRESHOLDNF50 ≥ 0.56
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► In addition to the amounts originally granted, BARDA exercised two options in January 2017, allowing for continued development
► Key Upcoming Expected Milestones:
► Potential triggering of options in our development contract which will fund:
► the progression of manufacturing
► nonclinical development
► preparatory activities for the Phase 2 trial
► The Phase 2 study could initiate late 2018, provided the program continues to successfully advance with the support of BARDA as described above
Products in DevelopmentPx563L/RPA563: Next Gen Anthrax Vaccine Candidates
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► US administration has demonstrated strong support for development of medical countermeasures (MCMs)
► Current sole sourced contract is valued at close to $1B for a 5 year supply6
► Potential procurement contract if within 10 years of FDA approval
► We believe Pfenex is the only company funded by the US Dept. of Health and Human Services via BARDA developing a next generation rPA based anthrax vaccine that is able to potentially fulfill the needs of the Strategic National Stockpile (SNS)
Products in DevelopmentPx563L/RPA563: Next Gen Anthrax Vaccine Candidates
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► License and option agreement signed in July 2016granting Jazz Pharmaceuticals worldwide rights to develop and commercialize multiple early-stage hematology product candidates
► Collaboration details:
► Up to $181 MM in upfront and potential milestone payments including up to $41 MM non-sales related
► Tiered royalties on net sales► Collaboration governed by Joint Development
Committee with equal representation from each company
► Process development milestone achieved in Q3 2017
► Jazz obtains exclusive option to PF690, Pfenex’s pegaspargase biosimilar candidate to Oncaspar®
Products in DevelopmentJazz Pharmaceuticals/Pfenex Collaboration
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► Global Lucentis® market was $3.2 billion in 20167
► Latest known composition of matter expected patent expiry: USA 2020; EU 20228
► Phase 1/2 first-in-human study completed:
► Met primary objective of demonstrating similar safety and tolerability between PF582 and Lucentis
► Demonstrated consistent pharmacological activity between PF582 and Lucentis
► Pfenex considering strategic partnership opportunities for advancement to maximize value
Pipeline ProductsPF582: Biosimilar to Lucentis®
Best
Cor
rect
ed V
isual
Acu
itySc
ore
±SD
PF582 Lucentis
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► Neulasta® (pegfilgrastim) is indicated for the prevention of febrile neutropenia in patients receiving cytotoxic chemotherapy
► Neulasta global sales in 2016: $4.6 billion9
► US FDA feedback for PF529 was received in Q4 2016 supporting the feasibility of development under the 351(k) biosimilar pathway
► Pfenex considering strategic partnership opportunities for advancement to maximize value
Pipeline ProductsPF529: Biosimilar Candidate to Neulasta®
Q216 Q316 Q416 1H17 2H17 1H18 2H18 2019 2020
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Programs in our pipeline include five programs in clinical development, including one therapeutic equivalent, two vaccine candidates and two biosimilar candidates.
PfenexOverview & Investment Highlights
JAZZ PHARMACEUTICALSPARTNERSHIP ANNOUNCED
PF708 - teriparatide Px563L – Anthrax vaccine PF582 - ranibizumab PF529 - pegfilgrastim
PF708:POSITIVE TOPLINE STUDY RESULTS ANNOUNCED
PF708:PIVOTAL STUDY INITIATED
PF708:IMMUNOGENICITY STUDY RESULTS ANTICIPATED
PF708:POTENTIAL NDAFILING
PF708:INTERIM PK STUDY RESULTSANNOUNCED
PF708:EXPECTED FORTEO® PATENT EXPIRY (WITH RESPECT TO API, MOT AND/OR FORMULATION PATENTS IN US)
PX563L:POSITIVE PHASE 1 DAY 70 ANALYSISCOMPLETED
PX563L:CONSULTATION WITH BARDA
PX563L:POTENTIAL
OPTION TRIGGERED FOR CLINICAL
MANUFACTURING
PF582:PHASE 1/2 COMPLETED
PF582:EXPECTED LUCENTIS®PATENT EXPIRY IN US
PF529:REGULATORY FEEDBACKRECEIVED
PX563L:POTENTIAL
PHASE 2 STUDYINITIATION
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Proven Platform
Near-term Catalysts
Strong Management
Pfenex
Attractive Portfolio
Scalable Technology
INNOVATIVE SOLUTIONS FOR GLOBAL HEALTH
© 2017 Pfenex Inc. All rights reserved.
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1. Being developed via the 505(b)(2) pathway in the United States2 Based on publicly available 2016 sales data for the third party branded pharmaceutical company3 https://www.sec.gov/Archives/edgar/data/1367644/000136764416000084/form8kearningsq3q2016.htm4 Eli Lilly Annual Report 2016, Page 245 Forteo® teriparatide patent, U.S. patent no. 7,351,414 B26 https://www.sec.gov/Archives/edgar/data/1367644/000136764416000084/form8kearningsq3q2016.htm7 2016 Roche Annual report8 GaBI Online, http://www.gabionline.net/Biosimilars/General/Biosimilars-of-ranibizumab9 Amgen Annual Report 2016
PfenexReferences