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1 NASDAQ: XBIO Enhancing Lives with Transformative Therapies Corporate Presentation May 2018

2 Forward Looking Statements This presentation contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of All statements contained in this presentation other than statements of historical facts may constitute forward-looking statements within the meaning of the federal securities laws. These statements can be identified by words such as "expects," "plans," "projects," "will," "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding the Company s ability to develop and customize the Company s PolyXen platform technology to improve the clinical utility of protein and peptide drugs; the Company s anticipated corporate development strategies and pursuit of current and future collaborations to co-develop new product candidates; its ability to add new programs to its pipeline and expand the development of its current product candidates into new indications; the initiation, timing, progress, enrollment and reporting of results of its preclinical programs and clinical trials; the Company s potential for future growth and creation of shareholder value; the Company s expectation of cash flow from the royalties under the Right to Sublicense Agreement; and the Company s liquidity and ability to fund its future operations;. Any forward-looking statements contained herein are based on current expectations, and are subject to a number of risks and uncertainties. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These risks and uncertainties include those described in the "Risk Factors" section of the Company s Annual Report on Form 10-K for the fiscal year ended December 31, 2017 and filed with the Securities and Exchange Commission on March 30, 2018, and subsequent reports that it may file with the Securities and Exchange Commission. In addition, forwardlooking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new product candidates and indications, manufacturing issues that may arise, patent positions and litigation, among other factors. The forward-looking statements contained in this presentation speak only as of the date the statements were made, and the Company does not undertake any obligation to update forwardlooking statements, except as required by law. 2

3 Overview Clinical-stage biopharmaceutical company focused on discovery, research and development of next-generation biologic drugs and novel orphan oncology therapeutics XBIO lead program in Phase 2 clinical development for treatment of progesterone resistant endometrial cancer (EC) - FDA Orphan Drug designation for progesterone receptor-negative (PrR-) endometrial cancer in conjunction with progestin therapy - Planning a biomarker study of XBIO-101 for the treatment of triple negative breast cancer (TNBC) PolyXen - proprietary polysialic acid (PSA) drug delivery platform - Enables next generation biologic drugs by improving their half-life and other pharmacological properties - Planning to pursue business development activities to explore partnerships utilizing PolyXen delivery platform 3

4 Management Team Jeffrey F. Eisenberg Chief Executive Officer - Life Sciences executive with over 20 years of successful track record in value creation in both private and public companies 4 Curtis Lockshin, Ph.D. Chief Scientific Officer - 18 years Biotech/Pharma management experience, including discovery, preclinical and clinical development and commercial manufacturing James F. Parslow, MBA, CFA Chief Financial Officer - Over 30 years of experience providing financial and business leadership to biotech, manufacturing, technology, business-to-business e-commerce and cleantech industries

5 Board of Directors Adam Logal - Chairman - Over 15 years of experience in the biopharmaceuticals industry - Senior Vice President, Chief Financial Officer, Chief Accounting Officer and Treasurer of OPKO Health, Inc. Jeffrey F. Eisenberg - Chief Executive Officer James Eric Callaway, Ph.D. - Over 30 years of experience in the execution of product development operations for biotherapeutics - Seasoned CEO within the venture-backed biotech community and over the course of his career he has built and operated two companies, transforming each from research companies to clinical stage operating entities Firdaus Jal Dastoor, FCS - Fellow Member of The Institute of Company Secretaries of India - Board Member, Serum Institute of India Limited Dmitry Genkin - Also serves on the Company s Scientific Advisory Board - Headed a number of Russia's largest pharmaceutical companies including Pharmavit, which had 27% of the Russian pharmaceutical market Roman Knyazev - Senior Investment Director at Rusnano Roger Kornberg, Ph.D. - Nobel Prize Laureate in Chemistry - Member of the U.S. National Academy of Sciences and the Winzer Professor of Medicine in the Department of Structural Biology at Stanford University 5

6 Development Pipeline Internal Development Programs Program Indication Preclinical Phase 1 Phase 2 Phase 3 Next Catalyst Progesterone Resistant Endometrial Cancer Preliminary Phase 2 data expected in 2018 XBIO-101 Triple Negative Breast Cancer Planning a biomarker study 6

7 7 XBIO-101

8 Hormone Receptor-Negative Cancers Receptor negativity can arise from: - Biological state of cell (multifactorial origins) - Desensitization from prior hormone therapy Receptor-negative patients are not candidates for hormonal therapy Examples of receptor-negative tumor types: - Progesterone receptor negative (PrR-) endometrial cancer - Triple negative breast cancer (PrR-, ER-, HER2-) 8

9 XBIO-101 (sodium cridanimod) for Endometrial Cancer (EC) Scientific and clinical evidence indicates that loss of PrR expression during EC progression is linked with EC invasion, metastasis and worsening clinical outcomes Loss of PrR expression appears to be part of a sequence of molecular events leading to progression Progestin therapy is limited to the PrR+ patient population Efficacy of hormone therapy is limited, due to: - Loss of PrR expression in PrR+ tumors following progestin exposure - False positive PrR+ EC phenotypes - Predominance of PrR- status in metastatic sites in patients with PrR+ primary EC tumors 9

10 10 Progesterone Receptor-Negative Endometrial Cancer

11 11 Endometrial Cancer Statistics (US)

12 XBIO-101: Phase 2 Candidate for Treatment of PrR- EC XBIO-101: lead program in Phase 2 clinical development for treatment of PrR- EC Commercial and clinical history: years of market history in Russia and FSRs as Neovir - Used in complex or monotherapy for immunodeficiency and infectious diseases - Principal mechanism of action: interferon induction In in vivo cancer models, sodium cridanimod induces expression of progesterone receptors in PrR- EC tumors - Restoration of PrR expression may sensitize endometrial tumor tissue to progestin therapy in unresponsive tumors Granted US Orphan Drug designation for treatment of PrR- EC Potential to treat a range of cancers, including PrR- EC and TNBC 12

13 XBIO-101 (sodium cridanimod) Clinical History Twenty-one foreign controlled clinical studies involving 750 patients have been conducted with sodium cridanimod administered by intramuscular injection. No SAEs were reported Sodium cridanimod has been marketed in former USSR countries since 1995 with over 10,000,000 doses sold under trade name Neovir 13 Pharmacovigilance reports verify that no unexpected serious adverse effects, unexpected non-serious adverse effects, expected serious adverse effects, expected non-serious adverse effects, or drug interactions were reported from the original date of marketing in the following territories: - Azerbaijan (Original Marketing Authorization 2007) - Armenia (Original Marketing Authorization 2009) - Belarus (Original Marketing Authorization 1995) - Kazakhstan (Original Marketing Authorization 2009) - Russia (Original Marketing Authorization 1995) - Ukraine (Original Marketing Authorization 2004)

14 XBIO-101 Effect on the Progesterone Receptor (PrR) In animal models XBIO-101 makes progesterone therapy effective in PrR- tumors; upregulation of PrR- expression has been confirmed at the molecular level In a biomarker study of EC patients, 100% of PrR- patients were converted to PrR+ status following XBIO-101 exposure 14

15 Upregulation of Progesterone Receptor by XBIO-101 Phase 2 Study: Open-label comparative study of the neoplastic tissue morphology and receptor status changes following a course of neoadjuvant therapy with XBIO-101 and Provera in patients with primary EC cancer. (50 patients evaluated) PrR+ > 10 fmol/mg PrR- <= 10 fmol/mg 15

16 Endometrial Cancer Xenograft Animal Models: XBIO MPA PrR- EC tumor xenografts PrR+ EC tumor xenografts Survival Benefit from XBIO-101 in PrR- tumors No survival benefit from XBIO-101 in PrR+ tumors Group (dose/animal) Mean Survival (d) p_ 1mg MPA + 6mg XBIO ± 7 <0.05 1mg MPA + 3mg XBIO ± 8 <0.05 1mg MPA 33 ± 3 <0.05 Control 38 ± 5 <

17 NEOPRO Trial: Interim Results An exploratory, open-label study to evaluate efficacy of a sodium cridanimod and medroxyprogesterone acetate (MPA) combination therapy in women with advanced or recurrent endometrial carcinoma who fail or become resistant to MPA monotherapy 17

18 XBIO-101: Phase 2 Study Highlights in Hormone-Resistant EC 18 XBIO-101 dose level is twice that of the NEOPRO trial (500mg vs. 250mg)

19 XBIO-101 Protocol Design Phase 2, Single Arm, Two Period Study of Sodium Cridanimod in Conjunction with Progestin Therapy in Patients with Endometrial Carcinoma 19

20 XBIO-101 Summary Phase 2 clinical development for treatment of hormone-resistant endometrial cancer Hormone-resistant endometrial cancer: significant unmet medical need with currently no FDA-approved treatment Granted US Orphan Drug designation for PrR- endometrial cancer in conjunction with progestin therapy Potential to expand into multiple high value indications 20

21 21 PolyXen PSA Technology Platform

22 PolyXen: Enhancing Protein Drugs Polysialylation employs the natural polymer polysialic acid (PSA) to modulate the pharmacokinetic and pharmacodynamic profiles of protein drugs Modelled on the multi-billion dollar success of PEGylation, which uses the synthetic polymer polyethylene glycol (PEG) Cost-effective strategy focuses on leveraging existing corporate partners, including Pharmsynthez and the Serum Institute Key Features: Half-life extensions Retention of native protein conformation Non-immunogenicity Biodegradability Fewer injections Improved protease stability Improved thermal stability Broad patent cover PolyXen Versatile: Designed to improve the clinical utility of most protein and peptide drugs Applicable to franchise extensions as well as candidates in development Potential use for delivering small molecule drugs 22

23 PolyXen: Next Generation Platform Technology Transforming Biologic Drug Delivery Seeking to build a pipeline of partnerships utilizing PolyXen, our proven biologic delivery platform 23

24 24 Corporate Summary

25 Capitalization: NASDAQ: XBIO March 31, 2018 Share Ownership % (000 s) Issued Shares PJSC Pharmsynthez 61.6% 5,365 OPKO Health Inc. 3.5% 309 Serum Institute of India Limited 7.2% 630 Shire Inc. 4.5% 395 Executive officers and directors 0.4% 32 All other common shareholders 22.8% 1,986 8,717 Potential Shares Equity Incentive Plan pre-ipo awards (WAEP: $5.76) 615 Equity Incentive Plan post-ipo awards (WAEP: $3.39) 1,277 Warrants (WAEP: $12.89) 646 Warrants associated with retired bridge notes (WAEP: $4.95) 1,098 Convertible Preferred A shares (held by Synbio LLC) 970 Convertible Preferred B shares (held by Pharmsynthez) 1,455 Convertible Preferred B shares (held by OPKO) 485 Convertible Preferred B shares (held by Alpha Capital) 181 Insider warrants associated with Preferred B IPO shares (WAEP: $4.00) 1,940 Non-insider warrants associated with Preferred B IPO shares (WAEP: $4.00) 485 9,152 Insiders own 8,667 of all potential shares Total Fully Diluted (Issued and Potential) Shares 17,869 25

26 NASDAQ: XBIO Enhancing Lives with Transformative Therapies Phone: IR: