Full Press Release Details
A Leader in Developing Immunocellular Therapeutics for Cancer
Analyst Briefing Document
Investment Highlights
CVac immunotherapy is a potential game changer in the treatment of ovarian cancer:
Maintenance therapy for ovarian cancer after second line treatment is a significant unmet medical need
Favorable competitive environment for immunotherapy agents to treat
Randomized phase 2 identified a clear development path and target patient profile (n=20)
Robust randomized phase 2 (n=210) proof of concept underway
Orphan designation for ovarian cancer in the U.S. and Europe
Prima has addressed production costs and manufacturing scale up globally
Funded for 2+ years through multiple data catalysts
Significant non-dilutive grant support (Saxony Development Bank in Germany has provided nearly EUR 8 million
in grants to support development)
Potential for expanded cancer indications in large pilot
pancreatic cancer trial underway
Notice: Forward looking statements
This document contains forward-looking statements. Such forward-looking statements are based on current expectations and
involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed.
Among other risks, there can be no guarantee that CVac will become a commercially successful product or that it will receive regulatory approval in the U.S. or other markets. Forward-looking statements in this press release should be evaluated
together with the many uncertainties that affect Prima BioMed s business, particularly those identified as Risk Factors in our Annual Report on Form 20-F for the year ended June 30, 2103, and in our filings to both the Australian
Securities Exchange and to the U.S. Securities and Exchange Commission. This document should not be relied on as a recommendation or forecast by Prima BioMed. Nothing in this presentation should be construed as either an offer to sell or a
solicitation of an offer to buy or sell securities in any jurisdiction. Prima BioMed undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise.
Briefing Document PRIMA BIOMED
Investment Highlights 1
Executive Summary. 4
Company Background 5
Recent Performance 7
Corporate Development 7
Orphan designation &
breakthrough designation 7
Personalized immunocellular therapeutics. 7
Early clinical history 9
CVac s place in treatment
of ovarian cancer and new agents in development 17
Treatment of Ovarian Cancer 17
Second-line therapy and second remission maintenance (Consolidation) 19
Agents in development for consolidation therapy 19
CVac s target patient profile & the CR2 ovarian cancer market 21
Pancreatic cancer 22
Mucin 1 background 23
Personalized immune cell
Dendritic cell immunotherapy therapy background 26
Considerations for cancer vaccines 29
Complex mixtures or purified antigens 29
selection, immunogenicity, and cross reactions 29
Checkpoint blockades 30
Briefing Document PRIMA BIOMED
Prima intellectual property 30
Cash position and guidance 31
Figure 1. Prima milestones and catalysts 8
Figure 2. CVac clinical trials accomplished and planned objectives 9
Figure 3. Immune responses generated in mice and in humans using mannan-mucin 1 (MUC1) with or without dendritic cells
Figure 4. Summary CAN-001 results 10
Figure 5. Ovarian cancer patient A09 case study from CAN-001 trial. Plot of CA-125 over time with arrows indicating
CVac injection points 11
Figure 6. Confirmed responders on CAN-002 12
Figure 7. Summary of best T cell response for CVac treated patients on CAN-003 13
Figure 8. T cell stimulation summary for 17 patients who completed 56 weeks on CAN-003, data corrected for baseline and
unstimulated PBMC controls 14
Figure 9. Kaplan Meier estimates of PFS for CAN-003 patients in second remission
Figure 10. Natural History of Ovarian Cancer 17
Figure 11. Summary of median survival outcomes depending on residual disease (RD) after primary debulking surgery in
Figure 12. Typical management course for ovarian cancer consolidation therapy 19
Figure 13. CVac s place in ovarian cancer management 21
Figure 14. Aberrant glycosylation of mucin 1expressed on tumor cells 23
Figure 15. CVac production and mechanism 24
Figure 16. Prima s approach to CVac manufacturing optimization 25
Figure 17. Screen shot of Prima s web-based automated logistics software 25
Figure 18. B cells cannot kill directly instead, they make antibodies which flag a pathogen or foreign cell for destruction by other elements of the immune system 26
Figure 19. Activated T lymphocytes can bind to and directly destroy cells expressing a foreign antigen 26
Figure 20. Passive immunotherapy with monoclonal antibodies 27
Figure 21. Dendritic cells attack cancer 28
Figure 22. NCI prioritization of cancer antigens 29
Figure 23. Prima balance sheet 31
Figure 24. Income Statement 31
Analyst Briefing Document
Executive Summary. Prima Biomed is a company with a viable product candidate in CVac for second remission ovarian cancer a product with minimal toxicity, good (but early phase)
randomized trial data, focused in a market of significant medical need and little competition.
robust revised clinical plan for ovarian cancer, the potential of treating additional mucin 1 overexpressing tumors such as pancreatic cancer, and Prima s current financial situation, there is significant upside to CVac.
CVac has demonstrated the ability to induce a robust T cell response specific for the mucin 1 cancer antigen. This is a
significant milestone in the cancer vaccine space, as it presages clinical efficacy (e.g. survival). With the recent phase 2 data, Prima identified a relatively homogeneous patient population (second remission ovarian cancer) and now has a clear
development pathway to bring CVac to market.
Most other ovarian cancer products in development are targeting
different mechanisms than CVac and could even be beneficial in combination with CVac.
Prima holds a leadership
position in the development of personalized immunocellular therapeutics a space that should become increasingly exciting in the coming months and years as the cancer immunotherapy market starts maturing.
As Dr. Drew Pardoll, Director of the Cancer Immunology Program in Johns Hopkins University s Sidney Kimmel
Comprehensive Cancer Center, was recently quoted talking about potential combinations of immunotherapies, . . . the real excitement lies with cancer vaccines. Their stock is very low right now, but they are going to come roaring back.
It is becoming increasingly evident that cancer immunotherapy will work best in combinations especially
the checkpoint inhibitors (like PD-1 and PDL-1 inhibitors) in combination with T cell stimulating products like CVac. As the market realizes the magnitude increase in efficacy that checkpoint blockades combined with therapeutic vaccines will provide
to patients, we see promising future for development of products such as CVac.
Analyst Briefing Document
Prima BioMed Ltd. was spun out
in 2001 from the Austin Research Institute (since merged with the Burnet Institute) in Melbourne, Australia, a well-regarded immunology research center.
The focus on the company is developing personalized immunocellular therapeutics to treat cancer with a clinical stage program for CVac in ovarian and pancreatic cancers.
Over the past year, in conjunction with Mr. Lehman taking over as CEO, CVac entering later stage trials, and the listing
of ADRs on NASDAQ, Prima has transitioned to a more US and European focused development stage company.
operates in the US, Germany, and Australia, and has accomplished the challenging tasks of successfully transferring manufacturing technology and scaling up production cost effectively for autologous cell based products in three regions.
Prima s primary listing is in Australia on the ASX (ticker PRR) and has an ADR listed on NASDAQ in 2012 (ticker
PBMD). Each 1 ADR = 30 ordinary shares.