Targeted Cancer Therapies Wedbush PacGrow Healthcare Conference, August 16 th , 2016
Safe Harbor Statement Except for statements of historical fact, any information contained in this presentation may be a forward-looking statement that reflects the Company’s current views about future events and are subject to risks, uncertainties, assumptions and changes in circumstances that may cause events or the Company’s actual activities or results to differ significantly from those expressed in any forward-looking statement. In some cases, you can identify forward-looking statements by terminology such as “may”, “will”, “should”, “plan”, “predict”, “expect,” “estimate,” “anticipate,” “intend,” “goal,” “strategy,” “believe,” and similar expressions and variations thereof. Forward-looking statements may include statements regarding the Company’s business strategy, potential growth opportunities, clinical development activities, the timing and results of preclinical research, clinical trials and potential regulatory approval and commercialization of product candidates. Although the Company believes that the expectations reflected in such forward-looking statements are reasonable, the Company cannot guarantee future events, results, actions, levels of activity, performance or achievements. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including those described under the heading “Risk Factors” in documents the Company has filed with the SEC. These forward-looking statements speak only as of the date of this presentation and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof. Certain information contained in this presentation may be derived from information provided by industry sources. The Company believes such information is accurate and that the sources from which it has been obtained are reliable. However, the Company cannot guarantee the accuracy of, and has not independently verified, such information. Trademarks: The trademarks included herein are the property of the owners thereof and are used for reference purposes only. Such use should not be construed as an endorsement of such products. 2
ProNAi Therapeutics A drug development • We are an ambitious oncology drug development company oriented to company focused on registration and commercialization. advancing targeted cancer • We have a world-class management therapies team with a proven track record in oncology drug development. • We intend to build a broad and NASDAQ: DNAI diverse pipeline of promising Headquarters: Vancouver, BC oncology assets against emerging Development: San Francisco, CA targets on the leading edge of cancer biology. IPO: July 2015 • We have a healthy cash balance, that Shares (30/6/16): 30.2M outstanding we expect will allow us to achieve 34.6M fully diluted meaningful development milestones. Cash on hand (30/6/16): $130.6M 3
Proven Leadership in Oncology Development Nick Glover, PhD President and CEO Barbara Klencke, MD Chief Development Officer Angie You, PhD Chief Business & Strategy Officer and Head of Commercial Sukhi Jagpal, CA, CBV, MBA Chief Financial Officer Chandra Lovejoy Keith Anderson, PhD Senior Vice President, Global Regulatory Affairs Senior Vice President, Technical Operations and Head of Quality Wendy Chapman Emma McCann Senior Vice President, Clinical Operations Senior Vice President, Program Management Diane Gardiner Gregg Smith, PhD, MBA Senior Vice President, Human Resources and Senior Vice President, Preclinical Administration Christian Hassig, PhD Senior Vice President, Research 4
PNT2258 and DNAi • Interim assessment of PNT2258 at ASCO: modest efficacy observed with mostly limited duration of response. • Clinical data were not trending to an outcome supportive of registration as monotherapy in late line DLBCL. • Wolverine and Brighton studies were closed to further enrollment of subjects. • All development of PNT2258 was suspended. • Halted all further investment in our DNAi research program. • No further investment in PNT2258 or DNAi currently contemplated. • Closed our research facility in Plymouth, Michigan, which supported these programs. 5
Building a Pipeline of Promising Oncology Assets We intend to build a broad and diverse pipeline: • PNT141 is our first asset. • Two additional promising assets under exclusive option. • Actively engaged in business development with multiple assets under evaluation. Our strategic business development focus: • Small molecules and next generation biologics with exemplary target selectivity. • Foundation of strong, cancer-critical biology. • Emerging targets; opportunity to be first-in-class or fast follower/best-in-class. • Potential as monotherapy and in combinations, across a variety of cancer indications. • Possibility of proprietary combinations. 6
PNT141: Targeting Cdc7
Targeting the DNA Damage Response Network Cell Viral Oxygen Replication DNA Damage Response Metabolism Infection Radicals Drugs Stress Radiation (DDR) DDR is a network of cellular • pathways that monitor and DNA Damage repair DNA damage. • Our DNA is continuously subject to The DDR comprises cell-cycle • damage through a variety of checkpoints, which temporarily endogenous and exogenous inhibit replication to repair mechanisms. damaged DNA. 8
DDR: An Achilles Heel of Cancer • Malignant cells tolerate substantially Certain cancer cells survive and • replicate - despite accumulating DNA greater levels of DNA damage than damage - via an over-reliance on the healthy cells. DDR network. • Significant and persistent DNA Targeted inhibition of the DDR may be • damage is evident in tumors selectively lethal to cancer cells and of carrying genetic mutations such as potential benefit in the treatment of BRCA1/2, MYC and p53. certain cancers. Synthetic lethality is possible by • • Many standard chemotherapeutic inhibiting DDR in context of pre- agents and radiotherapy also induce existing genetic mutations. DNA damage in order to kill cancer cells. There also exists potential for synergy • between standard therapies and DDR targeting agents. 9
Burgeoning Scientific Validation for Targeting DDR Focus Issue: DNA Damage Repair June 2016 June 2016 10
Industry Validation of DDR’s Potential in Cancer May 2016 11
PNT141: Selective Small Molecule Targeting Cdc7 PNT141: highly-selective and • potent Cdc7 inhibitor. Cdc7: key regulator of DNA • replication and DNA damage response. Broad development scope in • solid and liquid tumors. Mono- and combo- therapy • development potential. Clinical studies expected to begin • by the end of 2017. 12
Cdc7: Key Function in the Cell Cycle Potential to exploit tumor-specific genetic defects and/or combination therapies to effect cancer cell death via ‘synthetic lethality’. 13
Cdc7 Inhibition: Potential Therapeutic Window Preclinical data and published literature suggest a variety of indications with potential for response to Cdc7 inhibitors: • Solid tumors: breast, ovarian, pancreatic, melanoma, colorectal, uterine, thyroid, etc. • Hematological malignancies: AML, DLBCL, etc. 14
Cdc7: Broad Potential Applications in Oncology Chemotherapy Combinations with DNA damaging chemotherapy DDR Monotherapy DDR Combinations Exploit replicative Rational stress and genetic combinations to instability in maximize DNA ‘synthetically lethal’ DNA Damage damage monotherapy Response Radiotherapy Immuno-Oncology Synergy with ionizing DDR coupled to innate radiation immune activation 15
PNT141: First-In-Class/ Best-In-Class Opportunity • PNT141’s selectivity profile offers possible differentiation and potential safety and efficacy advantages. • A biomarker-driven patient selection strategy focusing on drivers of replication stress, genomic instability and proliferation (e.g. p53, BRCA, MYC, KRAS, H2AX mutation/expression) will help facilitate clinical trial execution. • ProNAi plans to pursue a robust program of preclinical studies to further evaluate tumor responses across a variety of indications and dosing regimens to inform clinical development plans and patient selection strategies. • Clinical studies expected to begin by the end of 2017. 16
ProNAi: Advancing Targeted Cancer Therapies • We are an ambitious oncology drug development company oriented towards registration and commercialization. • We have a world-class management team with a proven track record in oncology drug development. • We intend to build a broad and diverse pipeline of promising oncology assets against emerging targets on the leading edge of cancer biology. • Our first new asset is PNT141, a highly-selective and potent Cdc7 inhibitor with broad development scope and first-in-class / best-in-class potential. Cdc7 is a key regulator of both DNA replication and DNA damage response. • We have a healthy cash balance, that we expect will allow us to achieve meaningful development milestones. 17
Targeted Cancer Therapies
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