LION. Corporate Presentation June 2016 BIOTECHNOLOGIES. Leadership & Innovation in Oncology

Transcription

1 LION BIOTECHNOLOGIES Leadership & Innovation in Oncology Corporate Presentation June 2016

2 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that can be identified with words such as expects, plans, projects, potential, suggests, may, or similar expressions. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. In particular, management s expectations regarding future results could be affected by, among other things, uncertainties relating to clinical trials and product development; unexpected regulatory delays or government regulation generally; the Company s ability to obtain or maintain patent and other proprietary intellectual property protection; and competition in general. For more detailed information about the risks and uncertainties that could cause actual results to differ materially from those implied by, or anticipated in, these forward-looking statements, please refer to the Risk Factors section of the Company s Annual Report on Form 10-K and subsequent updates that may be contained in the Company s Quarterly Reports on Form 10-Q and current reports on Form 8-K on file with the SEC. Forwardlooking statements speak only as to the date they are made. The Company does not undertake to update forward-looking statements to reflect circumstances or events that occur after the date the forward looking statements are made. 1

3 Leveraging and enhancing the power of tumor infiltrating lymphocytes (TILs) to treat solid tumors 2 2

4 Power of TIL: Compelling Results in Late-Stage Disease 3 Clinical Cancer Research December 15, : 6122

5 4 TILs: Regressions in Late-Stage Disease

6 Investment Highlights: LBIO (NASDAQ) Broad pipeline of programs using TILs for treatment of solid tumors 24% complete remission rate and 45% ORR in heavily pretreated metastatic melanoma patients (NCI) as presented at ASCO and JCO Lead product candidate LN-144 in Phase 2 trial for refractory metastatic melanoma Orphan designation Second product candidate entering Phase 2 trials for cervical and head and neck cancer by end of 2016 Exclusive licenses from NIH for TIL in melanoma, lung, breast, bladder and HPV-associated cancers MedImmune research collaboration for TIL and PDL-1 combination studies 5 Recently completed ~$100 million equity financing

7 Tumor-Infiltrating Lymphocytes (TILs) Unique Mechanism in Immuno-oncology TILs target neoantigens unique to tumor cells Capture tumor heterogeneity and overcome I/O challenge Large experience base in solid tumors Over 600 patients treated in the US Universal approach to solid tumors: Creating patient specific T-cells 6 ells

8 Robust TIL Pipeline/Lion and Collaborators Program (n) Indication Preclinical Phase 1 Phase 2 Milestones LN-144 (n=25) Melanoma Phase 2 trial enrolling patients, 2 cohort study, TIL, TIL+Durva TIL ± TBI (n=101) Melanoma Trial completed, 45% ORR, 24% CR TIL + Keytruda (n= 170) TIL + Nivolumab (n=12) LN-145 LN-145 Melanoma Melanoma Cervical cancer Head & neck cancer Currently enrolling Currently enrolling Phase 2 trial to initiate by year-end 2016 Phase 2 trial to initiate by year-end 2016 Preclinical Lung cancer Preclinical Ovarian cancer Preclinical Bladder cancer 7 Lion NCI Moffitt IST

9 LN-144: Phase 2 Trial in Refractory Metastatic Melanoma Enrollment ongoing in multi-center, single-arm, 2 cohort study in 25 patients Metastatic melanoma Refractory to at least one systemic treatment Sequential treatment with durvalumab Following a lympho-depleting preparative regimen, patients are treated with a single infusion of LN-144 followed by IL-2 Objectives Safety Feasibility Anti-tumor activity and other measures of efficacy 8

10 LN-144: Impressive Survival Benefit: 2 nd & 3 rd Line Durable remissions in melanoma regardless of prior therapies (Median Follow-up > 62 months) 19 of 20 complete responders are ongoing at 7 to >10 years 9 Steven A. Rosenberg, James C. Yang, Richard M. Sherry, et al. Immunotherapy with Metastatic Melanoma Using T Cell Transfer Durable Complete Responses in Heavily Pretreated Patients. April 15, 2011

11 NCI Study: Durable Remissions in Metastatic Melanoma Trial Combined Randomized 101 patient trial to determine effect of TBI Chemoablation + TIL + IL-2 Chemoablation + TBI + TIL + IL-2 CR* 24 (24%) NR 47 (47%) Objective response rates (ORR) = 54% in both combined arms Significant improvement over data from recent clinical studies of ipilimumab (ORR 10-15%) and anti-pd-1 therapy (ORR 31-41%) PR 30 (30%) Median follow-up about 41 months *23 CRs ongoing months 10 Updated June 2016 Data presented at ASCO June 2016

12 NCI Study: Excellent Survival in Melanoma Survival of patients in TIL ± TBI study 11

13 Tumor Reduction in Majority of Patients Metastatic Melanoma Patients MD Anderson N= Radvanyi L G et al. Clin Cancer Res 2012;18:

14 Tumor Responses to TIL: Melanoma Change in tumor burden over time after TIL infusion 100 Percent of initial tumor burden Time after TIL infusion (months) 13 Radvanyi et al., Clin. Cancer Res., 2012

15 NCI Published Study: Cervical Cancer and TIL Treatment Data Ability of infusion of HPV tumor-infiltrating lymphocytes (HPV-TIL) to induce regression of advanced HPV-positive cancers 9 patients with metastatic refractory or recurrent cervical cancer Widely metastatic disease 8/9 patients had prior radiotherapy and all patients had cisplatin Results: 3 objective tumor responses 2 CRs (duration 15+ and 22+ months) 1 PR (duration 3 months) No acute toxicities related to cell infusion No autoimmune AEs 14 Christian S. Hinrichs et al. J Clin Oncol 2015, 33 (15)

16 Manufacturing: Commercial Feasibility Lympho-depletion 15

17 Next Generation TILs Enable More Efficient and Effective Therapeutics Pre-sorted TILs offer many benefits Select higher potency TIL Need lower cell numbers Shorter manufacturing Lower COGS Stronger IP protection Genetic engineering of TIL Expression of certain cytokines to increase potency Modulation of PD-1/CTLA-4/LAG-3 on cell surface Persistence 16

18 Competitive Advantages of TIL in Solid Tumors PD-1s TCR CAR-T vs. TIL Long-term irreversible impacts Long maintenance period New therapy Genetic modification Intensive process to ID neoantigens Target only specific neoantigens No examples of utility in solid tumors Potentially immunogenic One time treatment: no maintenance therapies needed after TIL and IL-2 Less chance for unpredicted on-target, off-tissue effects TIL can now be successfully prepared from > 90% of melanoma patients (NCI, Moffitt) Target multiple neoantigens Autologous 17

19 18 Corporate Summary

20 Key Collaborations and Partnerships Preclinical and clinical collaboration with MedImmune CRADA with NCI/Rosenberg Option to license exclusive rights for new TIL therapy to treat lung, triple-negative breast, bladder and HPV-associated cancers Intellectual property license Exclusive licenses for TILs in metastatic melanoma, lung, breast, and HPV-associated cancers Manufacturing agreements 19

21 Management Team Maria Fardis, PhD, MBA President and CEO Molly Henderson, CPA CFO Michael Lotze, MD CSO Steven Fischkoff, MD CMO Howard Johnson, MBA VP, Corporate Development James Bender, PhD, MPH VP, Product Dev & Mfg 20

22 Scientific Advisory Board Dr. Mario Sznol Dr. Jeffrey Weber Dr. James Mulé Dr. Patrick Hwu Dr. Cassian Yee Dr. Daniel Powell 21

23 Financial Summary - First Quarter 2016 Total Common Shares Outstanding 48.6 million Preferred Shares as converted to Common Shares 0.8 million Warrants/Options 9.9 million Cash $99.2 million Debt 0 Data current as of March 31, 2016 Recent equity financing of $100 million 22

24 LION BIOTECHNOLOGIES Leadership & Innovation in Oncology