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Corporate Presentation May 2017
• 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 timing of the initiation of our anticipated clinical trials and studies for Px563L and our other product candidates; expectations with regard to future milestone and royalty payments from our collaboration with Jazz Pharmaceuticals; expectations with respect to our ability to receive future payments under our government contracts; potential market opportunities for PF582, PF708, PF529 and our other product candidates; developments and projections relating to competitors and the industry, including the rate and degree of market acceptance of biosimilars by stakeholders, payors and physicians; the potential timing of our clinical trial results for PF708 and our other product candidates; the expected patent expiration timelines for Lucentis, Forteo, and other branded reference drugs; our expectations regarding the use of abbreviated regulatory pathways for the approval of our product candidates, including our use of the 505(b)(2) regulatory pathway for PF708 and the 351(k) pathway for PF529; and our expectations with regard to our potential to obtain a government procurement contract for Px563L if within ten years of FDA approval. 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 PF582, PF708, and our other product candidates; our reliance on our collaboration partners’ performance over which we do not have control; failure to achieve favorable results in later clinical trials for PF582, PF708, 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 PF582, PF708, or our other product candidates due to the existence of intellectual property protection owned or controlled by a third party and directed to PF582, PF708, or our other product candidates; PF582, PF708, 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 May 8, 2017 press release announcing results for the quarter ended March 31,2017. You should read our Annual Report on Form 10‐K for the year ended December 31, 2016, our Quarterly Report on Form 10‐Q forthe quarter ended March 31, 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
Safe Harbor Statement
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Pfenex Today
• 67 full time employees (15 PhDs/MDs)*
• 47,000 square feet of lab and office space with 17,000 square feet housing state of the art molecular biology, fermentation, purification, analytical and pilot plant capabilities*
• Three wholly-owned clinical stage biosimilar and therapeutic equivalent product candidates targeting $5.9B of 2015 branded product sales
• Up to $181M partnership with Jazz Pharmaceuticals to develop hematology/oncology products
• Up to $143.5M contract with the US government to develop a next generation anthrax vaccine
• Platform enables robust pipeline of product candidates
*As of May 3, 2017
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Programs in our development pipeline include four programs in clinical development including three biosimilar candidates and one vaccine candidate
Corporate Overview & Investment Highlights
Q2 2016 Q3 2016 Q4 2016 1H 2017 2H 2017 1H 2018 2H 2018 2019 2020
PF708: Positive topline study results reported
Jazz Pharmaceuticals partnership announced
PF582: Phase 1/2 completed
Px563L: Positive Phase 1 Day 70analysis completed
PF708: Pivotal study initiated
PF529: Regulatory feedback received
PF708: ExpectedForteo® patent expiry(with respect to API, MOT and/or formulation patents in US)
PF582: ExpectedLucentis® patent expiry in US
Px563L: Consultation with BARDA
PF708
Px563L
PF582
PF529
PF708: PK study results anticipated
PF708: Immunogenicity study results anticipated
Px563L: Potential phase 2 study initiation
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Pfenex Pipeline
PRECLINICAL/ BIOANALYTICAL CHARACTERIZATION
PF708 – teriparatide (Forteo®)1
PF582 – ranibizumab (Lucentis®)
Preclinical Analytical Similarity US FDA BiosimilarInitial Advisory
Meeting
Comparative Clinical Study
Initial I Pivotal
2015 Sales of Third Party Branded Reference
Product
PF529– pegfilgrastim (Neulasta®)
Multiple HemOnc Products
PF444 - HGHPF694– PegInfAlpha (Pegasys®)
Fully funded by US
Government
$1.4B2
$3.6B2
$4.7B2
Preclinical Phase 1 Phase 2
Px563L – Adjuvanted Anthrax Vaccine
RPA563 – Non-Adjuvanted Anthrax Vaccine
$403M2
$3.5B4
PF690 –pegaspargase(Oncaspar®)
$121M3
PF688 –certolizumab pegol (Cimzia®)
Wholly Owned $1.2B2
PF530 – Interferon beta -1b (Betaseron®) $900M2
$11.9B
1 Being developed via the 505(b)(2) pathway in the United States2 Based on publicly available 2015 sales data for the third party branded pharmaceutical company.3 Approximate 2015 global branded sales of third‐party reference drug per IMS data accessed May 4, 2016. 4 Approximate 2015 aggregate global branded sales of third‐party growth hormone products per IMS data accessed May 4, 2016
…………………………………………………
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• Our unique Expression Technology allows for rapid, high quality production of therapeutics and vaccines and a revolutionary advancement in clinical technology.
Pfenex Expression Technology
GOAL:HIGH QUALITY, HIGH TITER
TRADITIONAL: TRIAL AND ERROR
PRIMARY STRUCTUREAmino acids are the primary structures of a protein, linked together by peptide bonds, which form a polypeptide. Biosimilars are first compared at the polypeptide level.
SECONDARY STRUCTUREPolypeptides are then coiled into a helix - this is the secondary structure that is compared for biosimilarity.
TERTIARY STRUCTUREThese helixes of polypeptides then fold together in a specific manner. This resulting tertiary structure is then considered for biosimilarity.
QUATERNARY STRUCTUREThese polypeptide folds interact to form a functional protein. This is the quaternary structure considered for biosimilarity.
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Biosimilars Market Landscape
Globally Biosimilars Are Gaining Momentum
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EU Biosimilar Product Reviews as of April 2017
51 Marketing Authorization Applications (MAA) submitted
17 MAAs under review
34 MAAs reviewed
31approved
7 accelerated Biologics License Applications (aBLA) submitted
66 programs in the FDA
Biosimilar Biological Product
Development program
7 aBLA’s reviewed
5 approved
US Biosimilar Product Reviews as of April 2017
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FDA and EMA Biosimilars Guidance: Focus Is on Analytical Comparison
Phase 3
Phase 1/2
BiologicalCharacterization
AnalyticalCharacterization
Novel Drugs
Analytical Characterization
BiologicalCharacterization
Pharmacokinetics
Pharmacodynamics,Immunogenicity
Biosimilars
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Biosimilars Expand Patient Access to Therapy
Filgrastim Uptake in the EU
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• NOR‐SWITCH Study: Physicians more comfortable switching patients to a biosimilar
• United Healthcare, Express Scripts, CVS Health have excluded Neupogen from their formulary in favor of its biosimilar Zarxio. Healthcare systems establishing biosimilars only tiers. (OPERS)
Stakeholders are Responding
FirstWord Pharma Physician Views Poll, November 2016
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Our Products in Development
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• Forteo® (teriparatide) indicated for treatment of high fracture risk osteoporosis• Reference product produced via E. coli• Forteo global sales in 2015: $1.4 billion5
• Expected section 505(b)(2) regulatory approval pathway for PF708• Latest expiry of Orange Book‐listed teriparatide method of treatment, formulation and/or API patent expected in 2019 in US
• Pfenex has achieved high titer protein production; low cost of goods• Completed bioequivalence in healthy subjects that met endpoints • Pivotal immunogenicity/pharmacokinetic study in subjects with osteoporosis began at end of 2016
• PK study results anticipated in second half of 2017• Immunogenicity study results anticipated in first half of 2018
PF708: Therapeutic Equivalent Candidate to Forteo®
Study Design
Results
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• Latest known composition of matter patent expiry: USA 2020; EU 2022• 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
• Evaluating strategic options
PF582: Biosimilar to Lucentis®
Figure 1: No significant differences in best corrected visual acuity (BCVA)
Figure 2: Comparable decreases in central retinal thickness
Figure 3: Comparable immunogenicity (anti‐drug antibody formation)
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• Neulasta® (pegfilgrastim) is indicated for the prevention of febrile neutropenia in patients receiving cytotoxic chemotherapy
• Neulasta ® global sales in 2015: $4.7 billion6
• Regulatory feedback for PF529 was received in Q4 2016 and may support the feasibility of development under the 351(k) biosimilar pathway.
• Pfenex continues to evaluate the potential resource requirements and timeline for development.
PF529:Biosimilar Candidate to Neulasta®
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• In August 2015 awarded a BARDA contract of up to $143.5MM to fund advanced development
• Anthrax recombinant Protective Antigen (rPA) vaccine candidates:• Phase 1a Day 70 analysis demonstrated that
Px563L was well‐tolerated and conferred potentially superior protection after only 2 doses
• Potential procurement contract if within 10 years of FDA approval
• In addition to the base period, BARDA has exercised an additional two of the eight option periods effective January 2017, allowing for the continuing development of Px563L.
• The Phase 2 study could initiate in 2018, provided the program continues to successfully advance with the support of BARDA.
Px563L: Next Generation Anthrax Vaccine Candidate
Figure 1: Toxin Neutralizing Antibody NF50 Results
Table 1: Positive Day 70 Immunogenicity Results for Px563L
• Regulatory Threshold For Post Exposure Prophylaxis Indication:• For the percentage of subjects with TNA NF50 value ≥0.56, the
lower limit of the 95% confidence interval should be ≥40%.
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• Agreement signed in July 2016
• License and option agreement granting Jazz Pharmaceuticals worldwide rights to develop and commercialize multiple early stage hematology product candidates
• Partnership details:• Up to $181 MM in combined 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
• Jazz obtains exclusive option to PF690, Pfenex’s Pegaspargase biosimilar candidate to Oncaspar®
Jazz Pharmaceuticals/Pfenex Collaboration
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Programs in our development pipeline include four programs in clinical development including three biosimilar candidates and one vaccine candidate
Corporate Overview & Investment Highlights
Q2 2016 Q3 2016 Q4 2016 1H 2017 2H 2017 1H 2018 2H 2018 2019 2020
PF708: Positive topline study results reported
Jazz Pharmaceuticals partnership announced
PF582: Phase 1/2 completed
Px563L: Positive Phase 1 Day 70analysis completed
PF708: Pivotal study initiated
PF529: Regulatory feedback received
PF708: ExpectedForteo® patent expiry(with respect to API, MOT and/or formulation patents in US)
PF582: ExpectedLucentis® patent expiry in US
Px563L: Consultation with BARDA
PF708
Px563L
PF582
PF529
PF708: PK study results anticipated
PF708: Immunogenicity study results anticipated
Px563L: Potential phase 2 study initiation
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Appendix
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Senior Management
Patricia Lady, MBA, CPAChief Accounting Officer
Patrick K. LucyInterim CEO & Chief Business Officer
Paul Wagner, PhD, CFAChief Financial Officer
Hubert C. Chen, MDChief Medical & Scientific Officer
Steven S. SandovalChief Manufacturing Officer
Mayda MercadoVice President of Global Quality
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• Jason Grenfell‐Gardner, M.B.A. (Chairman)Jason Grenfell‐Gardner joined the board of directors in 2017. Mr. Grenfell‐Gardner has served as the President and Chief Executive Officer and a member of the Board of Directors of Teligent, Inc., a specialty generic pharmaceutical company, since 2012. From 2008 to 2012, Mr. Grenfell‐Gardner served in various management roles, and most recently as Senior Vice President of Sales and Marketing of Hikma Pharmaceuticals, PLC and its subsidiaries, including West‐Ward Pharmaceuticals, a pharmaceutical company.
• Robin D. Campbell, Ph.D.Dr. Robin D. Campbell joined the board of directors in September 2014. He has over 25 years of experience in pharmaceutical and biotechnology sales, marketing, product development and general management in both the U.S. and in international markets. Currently he serves as Chairman of the Board of Aptitude Medical Systems, an early stage company creating high performance aptamers for research, diagnostic and therapeutic use.
• Phillip M. Schneider, M.B.A.Phillip M. Schneider joined the board of directors in connection with Pfenex’s initial public offering in July 2014. Most recently, Mr. Schneider held various positions with IDEC Pharmaceuticals Corporation, a biopharmaceutical company, from 1987 to 2003, including: Senior Vice President and Chief Financial Officer from 1997 to 2003; and Director of Finance and Administration from 1992 to 1997.
• John Taylor, J.D.John Taylor joined the board of directors in April 2015. He is the President and Principal of Compliance and Regulatory Affairs at Greenleaf Health LLC., and has over 24 years of experience working on food and drug related issues at the U.S. Food and Drug Administration (FDA) and in private industry.
• Dennis Fenton, Ph.D.Dr. Dennis Fenton joined the board of directors in September 2015, deepening the manufacturing and product development expertise of our company. Dr. Fenton, an industry pioneer, has over three decades of experience in the biotechnology industry. Dr. Fenton retired after a lengthy and distinguished career with Amgen, where he held a variety of notable roles, including Executive Vice President, Operations.
• Sigurdur (Siggi) Olafsson, M.S.Sigurdur (Siggi) Olafsson joined the board of directors in 2017. Mr. Olafsson served as President and Chief Executive Officer, Global Generic Medicines Group of Teva Pharmaceuticals Ltd., a pharmaceutical company, from 2014 until his retirement in March 2017. Mr. Olafsson previously served as President of Actavis Pharma, Inc., a pharmaceutical company, from 2012 to 2014, Executive Vice President, Global Generics, at Actavis plc (Watson) from 2010 to 2012 and CEO of the Actavis Group from 2008 to 2010.
Board of Directors
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The Pfenex Toolbox
Elements combined to generate >100 rapid cloning, off the shelf, expression vectors covered by our numerous issued/allowed patents**As of March 7, 2016
Our patent protected ability to enable rapid strain engineering for optimal protein production accelerates proof of concept, product development and long‐term cost of goods advantage.
Cell Model
Our model enables 5,400,00+ opportunities for strain development.
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• Biosimilars in the US: Progress and Promise DIA Biosimilars 2016 John Jenkins, M.D. Director Office of New Drugs Center for Drug Evaluation and Research October 27, 2016
• BCC Research. “Biologic Therapeutic Drugs: Technologies and Global Markets,” Jan. 2015, p. 2.• IMS Health, “Shaping the biosimilars opportunity: A global perspective on the evolving biosimilars landscape.” Dec. 2011, p. 6. • Bayer Annual Report 2015, p. 156.• Eli Lilly Annual Report 2015, p. 40.• Amgen Annual Report 2015, p. 44• Medicines for Europe, “Biosimilar Medicines: Did You Know?” accessed Apr. 2016.• Generics and Biosimilars Initiative Journal, “Saving Money in the European Healthcare Systems with Biosimilars,” 2012, p. 124.
References
Note: All trademarks mentioned herein are property of their respective owners.
© 2017 Pfenex Inc. All rights reserved.
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