Full Press Release Details
Execute development plans : Data will be the main driver for future growth in company value Data establishes safety, efficacy, and commercial viability of our products Data is the basis for global regulatory approvals physician use Focus on data quality Focus on data integrity Corporate Strategy As Dr. Frazer says . . . . . . everyone else bring data (the US FDA s unofficial motto)
Incremental research innovation : Identify optimal patient populations Support development of portfolio products Improve product manufacturing processes Lower costs of production and delivery Corporate Strategy 3. Expand internal resource capacity : Focus in San Francisco a key area for hiring in our field Review production and laboratory capacity Enhance value of the company Add security to supply chain Maximize control over programs Support increased development activity Corporate Strategy 4.
Enhance business development : Identify assets and technology complimentary to Prima s existing advantages to develop additional products in an expanded pipeline Review a variety of arrangements that maximize potential upside acquisitions, collaborations, licenses Expand CVac development to other cancers pending data and financing Marc Voigt recently appointed as CBO 2.
PBS) CVac Potential Market CVac Safety CAN 003X Study (ASX:PRR, NASDAQ:PBMD) Neil Frazer, MB ChB Chief Medical Officer TM Autologous dendritic cell approach Comprised of a patient s own cells Normal Mucin 1 is produced by cells lining body tissues exposed to the outside world, and only at their tips that line these organs (airways, gut, mouth and others) Killer T Cells do not normally enter these tubes Killer T Cells target abnormal Mucin 1 Intradermal route is inherently a safe way to deliver the dendritic cells to optimize migration to lymph nodes Why Cvac should be well tolerated Studies recruited patients with rapidly progressing disease Studies did not have an arm with patients who did not receive CVac Multiple courses of chemotherapy, surgery and radiation therapy for all patients Cvac was very well tolerated Adverse events recorded were almost always associated with the disease, not Cvac Investigator assessment: some injection site reactions (similar to mosquito bites), rare temporary flu like illness related to Cvac CAN 001 and CAN 002 safety Independent safety reviews by a safety monitoring board to date support progression of the study Serious adverse events (for example requiring time in hospital) are uncommon, and almost always considered disease related One death (cerebral hemorrhage) in a patient in the standard of care arm while on study, obviously unrelated to CVac Pain on injection and/or injection site reaction (mild) in 7 of 36 patients receiving Cvac Full safety analysis anticipated after completion of dosing of all CAN 003 patients Safety Profile in CAN 003 Newer protocol only for CAN 003 patients Allows patients who progress during the CAN 003 study to: receive CVac for the first time (if they had been in the standard of care arm), or continue to receive a full course of Cvac if they had not completed the course on CAN 003 The study is ongoing and one death due to disease progression has occurred Data outcomes: Explore safety of combining CVac with standard chemotherapy Additional data for overall safety CAN 003X CVac Development for ovarian cancer (ASX:PRR, NASDAQ:PBMD) Sharron Gargosky, PhD Chief Technology Officer TM Apheresis MNC s cultured DC s pulsed with M FP Mucin 1 internalized CVac in vial CVac overview Objective stimulation of the immune system to target and destroy tumors, leaving normal tissue undamaged Mucin1 selected as the target for immunotherapy Up to a 40 fold increase in the amount of mucin1 present in cancer cells compared with normal cells Alteration in cellular distribution of mucin1 in cancer cells, with mucin1 being found ubiquitously throughout the cell rather than at the secreting pole May also be an alteration in the structure of mucin1 such that new carbohydrate and peptide epitopes, which are not detected in normal tissues, are exposed i.e. naked protein is exposed Mu cin 1 Immunotherapy CAN 001 Study 10 patients with terminal cancer (3 6 months life expectancy), broad range of adenocarcinomas including renal, breast, ovarian, fallopian tube, colon, lung and oesophageal Objectives: Primary: assess toxicity Secondary: assess anti tumour efficacy, immune response and procedure feasibility Results: No treatment related toxicity All patients produced desired cellular immune response Patient s cells could be successfully cryopreserved Protocol: 28 patients (21 evaluable) enrolled ovarian cancer and rising CA 125 levels ( 25% over baseline within one month) Patients received 3 injections of CVac at one monthly intervals, followed by 4 injections at 10 week intervals 9 of 21 evaluable patients received 3+ doses Objectives: Primary: disease response as defined by CA 125 Secondary: safety Results Summary: 4 of 21 evaluable patients responded (19%) to CVac (stabilization or reduction in CA 125 level from baseline) All 4 of the responding patients had received 3+ doses (i.e. 44% response in this group of 9) CVac well tolerated; no therapy related toxicity CAN 002 Study CAN 003 Study Protocol: 63 epithelial ovarian cancer patients enrolled in complete remission after successful 1 or 2 line chemotherapy 36 randomized to CVac ; 27 to observation standard of care Objectives: Primary: progression free survival (PFS) as defined by CA 125 overall survival (OS) prove biological activity by intracellular cytokine staining (ICS) establish comparability between 2 global manufacturing sites confirm safety of CVac in patients in remission Results: Manufacturing comparability established CVac well tolerated to date; no therapy related toxicity Dosing complete for all patients in 4 quarter 2012 Within the next 1 2 months, Prima will outline a calendar for full data release planned over the next year (ICS, PFS, OS) th st nd CANVAS CAN 004 Study CANVAS( CAN cer VA ccine S tudy) is multinational, multi center, randomized, double blinded, placebo controlled for a robust analysis Stage III/IV patients eligible after optimal debulking 1000 patients randomized 1:1 CVac:placebo to obtain 800 evaluable patients Patients must be in complete clinical and radiologic remission after chemotherapy to continue to dosing 6 doses in 44 weeks Dosing start as soon as possible (~2 3 weeks after chemo) About 120 130 centers globally (USA, Australasia, Europe) First patient in Q1 2012 Last patient in expected by Q1 2014 CAN VA S Data Points Development Milestones Manufacturing Intracellular cytokine staining (ICS) to add knowledge of biologic mechanism of action Companion screening test Safety confirmation Establish CVac effects on progression free survival Establish CVac effects on overall survival Collect pharmacoeconomic and quality of life data Qualification and training of cell collection centers worldwide Validation of automated scheduling and logistic management software Comparability and scale up of 3 global production centers Validation of quality control measure Finalization of product specifications Manufacturing milestones ICS is a method to see if CVac, after administration, induces a T cell response specific to mucin 1 Validation of CVac s mechanism of action (how it is expected and intended to work biologically) Data would be a key point for Prima to go into exploratory studies in other cancer types that overexpress mucin 1 Intracellular Cytokine Staining CVac is intended to target mucin 1 overexpressing tumor cells Important for efficacy and commercial reasons to only treat patients with mucin 1 overexpressing tumors Prima has developed a mucin 1 screening test in collaboration with Fraunhofer Institute in Germany Plan to approach development partners to help validate the test for commercial use Companion Screening Tool The time between when a patient responds to 1 st line therapy and is in remission until the tumor comes back (relapse) Extending the time in remission can confer significant improvement in quality of life, reduces the burden on the medical system by deferring further medical intervention, and may indicate an improvement in later overall survival Median time in remission for our target patients group is about 16 20 months Goal is to obtain a clinically meaningful improvement in this time Progression Free Survival st Gold standard of cancer research Most objective measure of efficacy Some weaknesses: Can be confounded due to multiple treatments Much longer time point to observe Secondary objective of CVac trials is to obtain an improvement in overall survival of patients Overall Survival For regulators and payors, quality of life is of increasing importance Goal is to assess the patient s ability to work and live a normal standard of life Key point of any future pricing negotiations with health plans Goal of CVac is to also improve life quality Quality of Life Looking forward Data catalysts (all subject to exact timing of patient outcomes): CAN 003 final safety analysis CAN 003 intracellular cytokine staining data (ICS) CAN 003 progression free survival data CAN 003 overall survival data Development catalysts: Comparability with 3 rd manufacturing center (Europe) All blood collection centers globally qualified and approved All CANVAS sites globally initiated Initiation of exploratory trial in new cancer indication(s) pending confirmatory biological activity from ICS CVac catalysts next 12 months rd Corporate Strategy 1.
(2010) 70 75% diagnosed in late stage ~44,400 p.a. 70 80% of those patients are mucin 1 positive and achieve remission ~33,300 p.a. (Total estimated present market size in Australia major markets) CVac potential market share price depends on: Competitive products for maintenance of remission currently there are none in advanced development Clinical trial results (quantification of benefit) Negotiations with payors health plans (e.g.
Interesting target for potential antibodies Prima tested 3 antibodies generated at the Burnet Institute (Melbourne) Strong binding to Cripto 1; however, no detectable direct killing of cancer cells While it is still an interesting target, potential applications would require substantial early phase research work not Prima s focus Cripto 1 EUR 4.1 million grant from Saxony, Germany supports European development CAN 003 study completed recruiting 63 patients in 3 quarter 2011 CANVAS trial commenced recruitment in 1 quarter 2012; Australia US sites open Major advances in control scale up of manufacturing, distribution, supply chain CVac rd st TM CVac Intellectual Property Licensed patents and unique mucin 1 sequence demonstrated to stimulate high levels of immune activity in nonclinical and clinical studies Composition of matter patent on Cvac and other key antigens Method patent for processing DCs with the antigen Orphan status (in ovarian cancer) in USA and EU Protection under biosimilars legislation Immense process and logistics know how CVac development and IP has established Prima s leadership position in personalized bio therapeutics Target Patient Profile: Stage III/IV epithelial ovarian cancer patients who are in complete remission after optimal debulking surgery and platinum taxane containing chemotherapy = New Market Annual incidence of EOC in Australia major markets (USA, Japan, UK, Germany, France, Italy, Spain) ~61,283 p.a.
Solid financial position and unencumbered programs allow flexibility to leverage future development opportunities Prima s technology platform Cryopreserved cell formulation minimizes cell collections (leukapheresis procedures) from patients and reduces cost Intradermal injections maximises delivery of maturing dendritic cells to drain to lymph nodes and migrate to tumor cells Automated logistics labeling with bar codes and digital management of the entire supply chain minimizes errors and optimizes use of human resources and facilities Cell collection expertise 40+ qualified and trained cell collection centers worldwide to provide material for high quality products Global breadth experience in tech transfers, comparability protocols, and regulatory licensing of manufacturing facilities in Australia, USA, and Germany Mucin 1 multiple opportunities mucin 1 is overexpressed on numerous other cancer types; Prima has a unique immunogenic sequence and companion diagnostic to move in other indications New product opportunities when supported by data and financing, at the right time, Prima has the opportunity leverage company strengths with new products Recent Accomplishments Business Updates SEC process in US significant validation of transparency compliance of the business In line with Prima s strategy of global visibility and operations Important step to facilitate larger and institutional investors in USA and Europe Expect to increase interest and liquidity in the stock over time especially behind clinical trial data that drives US analysts US German ADR Listings Was an interesting opportunity for a pilot commercialization project The initial assumptions of the business case were too optimistic Too challenging to reliably treat patients and too long to achieve profitability in Dubai to continue the business CVac and Prima can come back to the region at a more appropriate time Dubai Operations Embryonic gene implicated in carcinogenesis; re expresses at high levels in several human tumor types.
CVac potential: mucin 1 is also present in other cancers such as breast, colon, pancreas, lung and stomach. Technology: global platform (formulation, manufacturing, regulatory, distribution) for development of personalized bio therapeutics Prima BioMed: Introduction Legal form Public listed company Active since 2001 Headquarters Sydney, Australia Shares 1,065 million Market cap (13 Jun 2012) ~AU$ 126 million ~US$ 125 million ~EUR 100 million Headcount ~20 Prima BioMed is a world leader in the development of personalized bio therapeutics Senior Management Introduction Matthew Lehman Chief Operating Officer CEO Designate 12 years in industry clinical development Hundreds of clinical programs Sharron Gargosky Chief Technology Officer PhD biochemist with 18 years in industry Responsible for several FDA approvals Neil Frazer Chief Medical Officer Physician with 26 years in industry Led development programs in US EU Marc Voigt Chief Business Officer Corporate development in biotech for 15 years Led investment and partnering for several assets Ian Bangs Chief Financial Officer Company Secretary 25 years in senior financial positions Company secretary and CFO for a number of ASX companies 11 11 9 5 7 6 13 Prima BioMed: Global Footprint CANVAS research sites Prima offices Prima BioMed: Key Strengths Management team with a proven product development and commercial track record in oncology Clear development plan for CVac in ovarian cancer with an opportunity to expand CVac in other cancer indications Significant commercial opportunities to treat patients in remission Broad intellectual property position for CVac (patents, process know how, orphan status in USA and EU) Strong technology and global platform for development of personalized bio therapeutic products (cell therapy, immunology) Liquid stock on the Australian Securities Exchange (ASX:PRR); American depository receipts (1 ADR: 30 shares) listed on NASDAQ (NASDAQ: PBMD) and Deutsche B rse.
Frazer CVac development S. Gargosky Looking forward M. Lehman Questions answers Mission: developing novel and high value personalized bio therapeutic products with a focus in cell therapy for oncology. Lead product: CVac is made of autologous dendritic cells pulsed with mannan mucin 1 fusion protein. In advanced clinical development for ovarian cancer.
This presentation should not be relied on as a recommendation or forecast by Prima BioMed Ltd. Nothing in this presentation should be construed as either an offer to sell or a solicitation of an offer to buy or sell shares in any jurisdiction. Important Notice Agenda Intros to Prima and management M. Lehman Recent accomplishments updates M. Lehman CVac safety CAN 003X N.
Important factors that could cause actual results to differ materially from assumptions or expectations expressed or implied in this presentation include known and unknown risks. Because actual results could differ materially to assumptions made and Prima BioMed Ltd s current intentions, plans, expectations and beliefs about the future, you are urged to view all forward looking statements contained in this presentation with caution.
No representation or warranty is made as to the accuracy, completeness or reliability of the information. Any forward looking statements in this presentation have been prepared on the basis of a number of assumptions which may prove incorrect and the current intentions, plans, expectations and beliefs about future events are subject to risks, uncertainties and other factors, many of which are outside Prima BioMed Ltd s control.
Accordingly, these slides and the information they contain should be read in conjunction with past and future announcements made by Prima BioMed Ltd and should not be relied upon as an independent source of information. Please contact the Company and/or refer to the Company's website for further information. The views expressed in this presentation contain information derived from publicly available sources that have not been independently verified.
Prima BioMed Ltd. Shareholder Briefing 15 June 2012 (ASX:PRR, NASDAQ:PBMD) Matthew Lehman Chief Operating Officer CEO Designate Exhibit 99.1 The purpose of the presentation is to provide an update of the business of Prima BioMed Ltd ACN 009 237 889 (ASX:PRR and NASDAQ:PBMD). These slides have been prepared as a presentation aid only and the information they contain may require further explanation and/or clarification.