Investor Presentation

Transcription

1 Fr persnal use nly Investr Presentatin Dr. Marie Rskrw, CEO & Managing Directr September 2014 ASX: PAB

2 Fr persnal use nly Safe Harbur Statement This presentatin cntains frward-lking statements that are subject t risks and uncertainties. Such statements invlve knwn and unknwn risks that may cause the actual results, perfrmance r achievements f Patrys Limited t be materially different frm the statements in this presentatin. Actual results culd differ materially depending n factrs such as the availability f resurces, the results f clinical studies, the timing and effects f regulatry actins, the strength f cmpetitin and the effectiveness f the Cmpany's patent prtectin. 2

3 Fr persnal use nly Highlights f rd Quarter (July September): Released details f the planned PAT-SM6 & carfilzmib Phase Ib/IIa clinical trial due t start end f 2014 Reprted significant prgress n all preclinical prgrammes Prgressed the GMP manufacturing f PAT-SM6 2 nd Quarter (April June): Reprted the granting f a third US patent fr PAT-SM6 Released clinical data shwing that a patient with end-stage multiple myelma respnded psitively t treatment with PAT-SM6 in cmbinatin with ther marketed drugs Reprted the first plant-based prductin f PAT-SM6 1 st Quarter (January t March): Reprted final data frm successful Phase I/IIa PAT-SM6 single-agent clinical trial Rights issue cmpleted: Ttal fundraising f $7.7 millin PAT-SC1 10yr fllw-up clinical trial data in patients with gastric cancer published 3

4 Fr persnal use nly Vlume (m) Share Price A$ Crprate Overview KEY STATS & FINANCIALS 17 SEPT (AUD$) MANAGEMENT & BOARD ASX Cde PAB Share price $ week high $ week lw $0.021 Shares n issue 697,060,986 Market capitalisatin $14.6m 30/6/14 $8.6m Funding Since Listing $54m Marie Rskrw, BSc (Hns), MBBS, PhD Rger McPhersn, CPA, GAICD Frank Hensel, PhD Jhn Read, BSc (Hns), MBA, FAICD Mike Strk, BBA Suzy Jnes Managing Directr, CEO CFO, Cmpany Secretary Vice President R&D Nn-Executive Chairman Nn-Executive Directr Nn-Executive Directr CURRENT YEAR SHARE PERFORMANCE $0.061 $0.051 $0.041 $0.031 $0.021 $ Jan Jan Feb Mar Mar Apr May May Jun Jul Jul Aug 14 4

5 Fr persnal use nly Pipeline Prduct (Target) Discvery Preclinical Phase I Phase 2a PAT-SM6 (GRP78) Melanma Phase I Cmpleted PAT-SM6 (GRP78) Multiple Myelma Single Agent Phase I/IIa Cmpleted 2013 PAT-SM6 (GRP78) Multiple Myelma Cmbinatin Carfilzmib Phase Ib/IIa Cmmence 2014 PAT-LM1 (NONO) PAT-SC1 (CD55) PAT-SM3 PAT-SM4 PAT-H12 Slid Tumurs Gastric Cancer Phase I/IIa Cmpleted Out- Licensing Candidate 5

6 Fr persnal use nly PAT-SM6

7 Fr persnal use nly Patrys Lead Antibdy: PAT-SM6 PAT-SM6: IgM istype, λ-light chain Islated frm stmach cancer patient Targets tumur specific epitpe n GRP78 Binds als t xidised LDL and VLDL Mde f Actin: Internalisatin upn binding f xidised LDL & GRP78 PAT-SM6 Internalisatin triggers apptsis In viv & In vitr Reactivity: Effective in multiple xengraft mdels Expressin data shw specific expressin in wide range f tumurs incl. melanma and myelma 7

8 Fr persnal use nly Multiple Myelma - Opprtunity Multiple Myelma Opprtunity Cancer f the plasma cells in bne marrw. Cells grw ut f cntrl and frm tumurs in slid bne, cause damage t ther rgans Estimated t be mre than 220,000 cases wrldwide and incidence increasing 5 year survival f ~30% Market expected t increase frm $8B (2013) t >$15B (2018) Market currently dminated by 3 prducts: Revlimid (net sales $4.28B 2013) Velcade (net sales $3.77B 2013) Kyprlis (carfilzmib) (net sales $272M 2013) Several MAbs currently in clinical develpment but nne apprved t date. Likely t be used in cmbinatin therapies Significant interest in MM frm bth large pharmaceutical and bitechnlgy cmpanies 8

9 Fr persnal use nly Multiple Myelma Pathlgy Abnrmal plasma cells (myelma cells) secrete lts f useless antibdies (M prteins) Abnrmal prteins in serum detected by electrphresis Myelma cells crwd ut ther bld cells resulting in anaemia, thrmbcytpenia (bleeding) and leucpenia (infectins) Abnrmal prteins (Bence Jnes) detected in urine Myelma cells 9

10 Fr persnal use nly Multiple Myelma Clinical Presentatin Bne disease and hypercalcaemia Abnrmal prtein depsits Bne marrw failure and infectins 10

11 Fr persnal use nly Therapies fr Multiple Myelma Prtesme inhibitrs Brtezmib (Velcade) Carfilzmib (Kyprlis) Immunmdulatrs (IMIDs) Lanalidmide (Revlimid) Pmalyst (Pmalidmide) Thalidmide Stem cell transplantatin Autlgus Allgeneic Clinical studies Small mlecules Antibdies, peptides Immuntherapeutics Chemtherapeutics Melphalan Cisplatin Cyclphsphamide Dxrubicin 11

12 Fr persnal use nly Antibdies in Clinical Trials fr MM Daratumumab (Genmab/Janssen Bitech,Phase III) MOR 202 (MrphSys/Celgene, Phase I/IIa) PAT-SM6* (Patrys, Phase Ib/IIa) Eltuzumab (BSM/Abbtt, Phase III) GRP78 BHQ880 (Nvartis, Phase II) Antibdies in all stages f clinical develpment *Upcming trial t begin end f 2014 with PAT-SM6 in cmbinatin with carfilzmib and dexamethasne CD138 txin- cnjugated (BT062, Bitest AG, Phase I/IIa) 12

13 Fr persnal use nly PAT-SM6 Mntherapy Trial 12 patients treated with 4 dses PAT-SM6 in 4 dse chrts (0.3mg/kg, 1mg/kg, 3mg/kg and 6mg/kg/dse) evaluated at day +36 (end f trial) 10 male, 2 female: median age 71 yrs Received average 5 prir lines f therapy PAT-SM6 safe in all patients. N dse limiting txicity (DLT), n related serius adverse events (SAE) and n related adverse event grade 4 N evidence f immungenicity. PK analysis shwed half-life f 7 hrs PAT-SM6 specifically targets MM cells in viv and stimulates significant immune respnses 4/12 patients had stable disease (day +36 pst treatment) with a significant reductin in prtein M levels in the peripheral bld Patients wh received prir treatment with prteasme inhibitrs respnded best t PAT-SM6 Mean time t next therapy is 51 days (clinically significant). Tw patients had stable disease fr >130 days pst treatment 13

14 Fr persnal use nly Increase/Decrease Clinical Data Preliminary Efficacy M Prtein Changes frm Baseline (%) 140% 120% 114% 100% 80% Stable disease Stable disease Stable disease Stable disease 81% 60% 40% 42% 54% 36.30% 48% 40% 20% 0% -20% -40% 13% 7% 1.40% 1% % 0.3 mg/kg 1 mg/kg 3 mg/kg 6 mg/kg 4 patients shwed stable disease accrding t the IMWG criteria 14

15 Fr persnal use nly Clinical Data anti CD138 Istype Cntrl PAT-SM6 PAT-SM6 Istype Cntrl Immunhistchemical staining with PAT- SM6 n paraffin embedded tumur bipsies frm MM patients The psitive cntrl (anti-cd138) and PAT- SM6 bund t patient tissues PAT-SM6 Istype Cntrl Flw cytmetry n MM patient bne marrw Cells frm MM patients displayed cell surface binding fr PAT-SM6 suggestive the target (GRP78) is present n the MM cells anti-id CD138 merge Detectin f PAT-SM6 n circulating MM cells Befre and after dsing, patient bld was cllected and circulating MM cells were purified using CD138 magnetic beads. PAT-SM6 binding was cnfirmed using an anti-iditype antibdy specific fr PAT- SM6 (anti-id) by cnfcal micrscpy. In the psttreatment sample, red stained MM cells indicate the presence f PAT-SM6. CD138 was used as a psitive cntrl Pre-treatment Pst-treatment 15

16 Fr persnal use nly PAT-SM6 & Carfilzmib Cmbinatin Trial Endpints: 16 Primary: The safety and tlerability f PAT-SM6, given in cmbinatin with carfilzmib and dexamethasne in patients with multiple myelma wh are refractry and/r intlerant t brtezmib, a currently-marketed prteasme inhibitr Secndary: Overall respnse rate, duratin f respnse, time t prgressin and a series f wellestablished assays will measure immunlgical and disease parameters Design: Single-arm study with a 2-stage design: after treating 9 pts in the 1 st stage, if > 2 respnd (a partial respnse r better), the trial will cntinue t the 2nd stage and a ttal f 24 pts will be treated Each pt will be treated with up t 4 cycles f PAT-SM6 (6 mg/kg/dse) + carfilzmib (20-27 mg/m 2 ) + dexamethasne (40 mg) Each cycle will cnsist f 3 dses f PAT-SM6, 6 dses f carfilzmib and 4 dses f dexamethasne administered ver ne mnth Timelines: Trial prjected t start end f 2014 at 2 clinical centres in Germany (Würzburg & Dresden) Prgressive recruitment f up t 24 pts estimated t take mnths. Data frm first 9 patients will be released

17 Fr persnal use nly Cell Lines Used in Manufacturing Antibdies PER.C6 Cell Line: Patrys uses PER.C6 human cell line fr GMP prductin: Only human cell line cmmercially available Over 6300 patients have been treated with PER.C6 prducts safe prfile PER.C6 cell line has received regulatry apprval in all majr markets PER.C6 derived prducts have nt yet gained marketed apprval: Currently in Phase III Capable f prducing high yields f bth IgM and IgG antibdies. Cst f gds similar t CHO system Chinese Hamster Ovary (CHO) Cell Line: CHO is a cmmnly used cell nn-human cell line: CHO is mre familiar t big Pharma / Bitech cmpanies Numerus antibdy therapeutics prduced in CHO are n the market Patrys has initiated a feasibility study with CHO cells in rder t deliver multiple ptins t ptential partners 17

18 Fr persnal use nly PAT-LM1

19 Fr persnal use nly PAT-LM1 Antibdy & Target PAT-LM1: IgM istype, λ-light chain Islated frm a lung cancer patient Targets tumur-specific epitpe f surfaceexpressed NONO (nn-pou-dmain-cntaining ctmer binding prtein) Mde f Actin: NONO mainly fund in nucleus: invlved in transcriptinal & pst-transcriptinal gene regulatin Unknwn mechanism-f-transprt t cell membrane In Viv & In Vitr Reactivity: Effective in several xengraft mdels Expressin data shw specific expressin in a wide range f tumrs incl. lung, pancreas, cln, leukaemias Crystal structure f NONO with PSPC1 Passn et al PNAS

20 Tumur vlume mm 3 Fr persnal use nly OD655 PAT-LM1 Preclinical Data Lung Squamus Cell Carcinma + cntrl - cntrl PAT-LM withut C1q cating with 5μ/ml C1q cating 0.3 Cln Adencarcinma Esphagus Squamus Cell Carcinma Breast Ductal Adencarcinma μg 10μg 5μg 1μg 0.5μg IHC staining with PAT-LM1 n tumur tissues Lung cancer xengraft (n=10 per grup) Saline IgM PAT-LM1 20 A) Individual (symbls) and mean (bar) tumr vlume H&E (200µg) Appttic cells A) Individual (symbls) and mean (bar) tumr vlume Regressin PAT-LM1 binds C1q, suggestive f CDC Cytkeratin Cntrl Cntrl tumr (n=10 per tumr PAT-LM1 grup) LM-1 treated treated tumr tumr Cytkeratin Necrsis (200µg) B) Histlgy n excised tumurs H&E LM1 Cntrl Cntrl tumr tumr PAT-LM1 reduced tumur vlume & induced cell-death Appttic cells Regressin Necrsis PAT-LM1 LM-1 treate treate B) Histlgy n excised tumurs

21 Fr persnal use nly PAT-SC1

22 Fr persnal use nly PAT-SC1 (Gastric Cancer) PAT-SC1: 22 Pentameric IgM istype, λ-light chain Islated frm a stmach cancer patient Targets isfrm f CD55 (Decay Accelerating Factr) expressed n surface f multiple types f cancer cells POC Trial Results: Phase I/IIa (POC) pen-label trial cnducted (Germany) Safe in 51 pts receiving single 20 mg dse PAT-SC1 Significant 10 year survival benefit fr 30 pts with minimal residual disease (R0) pstsurgery vs. untreated pts (histric cntrl) Current Stage/Cmpetitin: Currently in ut-licensing prcess Cnversin frm hybridma t recmbinant PER.C6 cell line cmpleted Orphan designatin by the FDA fr use in gastric cancer N ther knw clinical prducts targeting CD55 Intellectual Prperty: Key patent granted in varius jurisdictins

23 Fr persnal use nly 2014 Key Milestnes Key Milestne Prjected Timing (CY) PAT-SM6: Final data frm mntherapy MM trial Cmplete GMP manufacturing fr cmbinatin MM trial Cmmence cmbinatin Phase Ib/IIa MM trial Preclinical and clinical data published 1Q, H, Q, Q, 2014 PAT-LM1: Cmplete manufacturing prcess develpment ready fr scale-up Preclinical data published PAT-SC1: Preclinical & clinical trial (gastric cancer) data published Out-licensing deal 2H, H, Q, Discvery Abs: Target wrk n PAT-SM3, PAT-SM4, PAT-H

24 Fr persnal use nly Fr Further Infrmatin Cntact Details: Dr. Marie Rskrw, Chief Executive Officer / Managing Directr Mr. Rger McPhersn, Chief Financial Officer Ph: inf@patrys.cm Website: