Dendreon is a biotechnology company focused on developing immunotherapies for cancer. It was founded in 1992 as Activated Cell Therapy and is now focused on developing Provenge, an autologous cellular immunotherapy for prostate cancer. Provenge received FDA approval in 2010 based on results from the IMPACT trial showing a 4 month increase in median survival. For expansion into Europe, Dendreon must obtain regulatory approval based on the IMPACT data and negotiate reimbursement given the high cost and modest survival benefits shown in the trial.
Professor Akseli Hemminki public presentation on oncolytic viruses (May 25, 2...Oncos Therapeutics
This document discusses gene therapy for cancer and summarizes the current state of clinical evidence. It describes how gene therapy works by delivering genes into cells to compensate for mutations or produce therapeutic proteins. Several clinical trials using gene therapy approaches like mutation compensation, prodrug converting enzymes, and oncolytic viruses are summarized. Personalized oncolytic adenovirus treatments have been administered to 190 late-stage cancer patients in Finland as part of an access program, showing promise for this targeted gene therapy approach.
This document discusses chronic prostatitis and its potential relationship to prostate cancer. It suggests that long-term chronic inflammation can potentially lead to prostate cancer through several mechanisms, including the production of growth factors and mutagenic compounds. It also describes a pre-cancerous lesion called Proliferative Inflammatory Atrophy (PIA) that has been observed adjacent to prostate cancer tumors. Several studies have found an association between past prostatitis and increased prostate cancer risk. Effective treatment of chronic bacterial prostatitis may help reduce this risk.
This document summarizes interim analysis results from a randomized phase 3 study of abiraterone acetate plus prednisone versus placebo plus prednisone in chemotherapy-naïve patients with metastatic castration-resistant prostate cancer. The study showed a statistically significant improvement in radiographic progression-free survival with a median of not reached for abiraterone acetate versus 8.3 months for placebo. There was also a strong trend towards improved overall survival with a median of not reached for abiraterone acetate versus 27.2 months for placebo and a 25% reduction in risk of death. Benefits were seen across patient subgroups.
ASCO 2015 Melanoma Immunotherapy
Thomas Olencki, DO Division of Medical Oncology Department of Internal Medicine The Ohio State University Wexner Medical Center Columbus, Ohio
Dr. Patrick Hwu presents the latest information on immunotherapies for melanoma at the MRF's Patient Symposium at MD Anderson Cancer Center on January 31, 2015.
The management of advanced prostate cancer has been revolutionized by the translation of the rapid progress made in understanding the biology of the disease into new therapies. While these developments are unprecedented, many challenges remain, including our need to understand how best to utilize these new treatments, how to integrate them into current treatment paradigms and how to demonstrate the most cost effective ways to use these new drugs. Dr. Dreicer briefly reviews the most important of the new developments, progress to date in integrating these new therapies, and speculates how these developments will translate into improvements in patient outcomes.
An automated donor management system
Collections
The Trima Accel and other automated blood collection devices increase efficiency and flexibility for mobile and in-center blood drives.
Processing
Terumo's automated blood processing systems like the Spectra Optia improve lab efficiency, blood component quality, and process control.
Pathogen Reduction
Terumo is working to make transfusions safer for patients through pathogen reduction technologies with simple procedures for operators.
Dendreon is a biotechnology company focused on developing immunotherapies for cancer treatment. Their first product, Provenge, received FDA approval in 2010 for the treatment of asymptomatic or minimally symptomatic metastatic castrate-resistant prostate cancer. Provenge involves extracting a patient's antigen-presenting cells and combining them with a recombinant prostate antigen to stimulate an immune response against prostate cancer cells. Dendreon is working to expand Provenge's market and develop new immunotherapy treatments targeting antigens like HER2/neu, CA-9, and CEA expressed on other cancer types. They face challenges in scaling up manufacturing and achieving reimbursement to fully commercialize Provenge in the US and expand into Europe.
Professor Akseli Hemminki public presentation on oncolytic viruses (May 25, 2...Oncos Therapeutics
This document discusses gene therapy for cancer and summarizes the current state of clinical evidence. It describes how gene therapy works by delivering genes into cells to compensate for mutations or produce therapeutic proteins. Several clinical trials using gene therapy approaches like mutation compensation, prodrug converting enzymes, and oncolytic viruses are summarized. Personalized oncolytic adenovirus treatments have been administered to 190 late-stage cancer patients in Finland as part of an access program, showing promise for this targeted gene therapy approach.
This document discusses chronic prostatitis and its potential relationship to prostate cancer. It suggests that long-term chronic inflammation can potentially lead to prostate cancer through several mechanisms, including the production of growth factors and mutagenic compounds. It also describes a pre-cancerous lesion called Proliferative Inflammatory Atrophy (PIA) that has been observed adjacent to prostate cancer tumors. Several studies have found an association between past prostatitis and increased prostate cancer risk. Effective treatment of chronic bacterial prostatitis may help reduce this risk.
This document summarizes interim analysis results from a randomized phase 3 study of abiraterone acetate plus prednisone versus placebo plus prednisone in chemotherapy-naïve patients with metastatic castration-resistant prostate cancer. The study showed a statistically significant improvement in radiographic progression-free survival with a median of not reached for abiraterone acetate versus 8.3 months for placebo. There was also a strong trend towards improved overall survival with a median of not reached for abiraterone acetate versus 27.2 months for placebo and a 25% reduction in risk of death. Benefits were seen across patient subgroups.
ASCO 2015 Melanoma Immunotherapy
Thomas Olencki, DO Division of Medical Oncology Department of Internal Medicine The Ohio State University Wexner Medical Center Columbus, Ohio
Dr. Patrick Hwu presents the latest information on immunotherapies for melanoma at the MRF's Patient Symposium at MD Anderson Cancer Center on January 31, 2015.
The management of advanced prostate cancer has been revolutionized by the translation of the rapid progress made in understanding the biology of the disease into new therapies. While these developments are unprecedented, many challenges remain, including our need to understand how best to utilize these new treatments, how to integrate them into current treatment paradigms and how to demonstrate the most cost effective ways to use these new drugs. Dr. Dreicer briefly reviews the most important of the new developments, progress to date in integrating these new therapies, and speculates how these developments will translate into improvements in patient outcomes.
An automated donor management system
Collections
The Trima Accel and other automated blood collection devices increase efficiency and flexibility for mobile and in-center blood drives.
Processing
Terumo's automated blood processing systems like the Spectra Optia improve lab efficiency, blood component quality, and process control.
Pathogen Reduction
Terumo is working to make transfusions safer for patients through pathogen reduction technologies with simple procedures for operators.
Dendreon is a biotechnology company focused on developing immunotherapies for cancer treatment. Their first product, Provenge, received FDA approval in 2010 for the treatment of asymptomatic or minimally symptomatic metastatic castrate-resistant prostate cancer. Provenge involves extracting a patient's antigen-presenting cells and combining them with a recombinant prostate antigen to stimulate an immune response against prostate cancer cells. Dendreon is working to expand Provenge's market and develop new immunotherapy treatments targeting antigens like HER2/neu, CA-9, and CEA expressed on other cancer types. They face challenges in scaling up manufacturing and achieving reimbursement to fully commercialize Provenge in the US and expand into Europe.
This document discusses targeted cancer therapy. It begins with an introduction to cancer classification and targeted therapy. It then discusses epidemiology, signs and symptoms, and risk factors of cancer. It describes the goals and challenges of targeted therapy development. Targets for targeted therapy include monoclonal antibodies and small molecule inhibitors that target proteins involved in cancer signaling pathways. Treatment involves administration of targeted drugs through intravenous methods. Side effects can include skin, gastrointestinal and blood problems. Limitations include cancer cells developing resistance. Present therapies also include immunotherapy and nanotechnology.
This document discusses targeted cancer therapy. It begins with an introduction to cancer classification and targeted therapy. It then discusses the epidemiology, signs and symptoms, and risk factors of cancer. It describes the goals and challenges of targeted therapy development. Targets for targeted therapy include monoclonal antibodies and small molecule inhibitors that target proteins involved in cancer signaling pathways. Treatment involves targeted drugs administered through pills or IV. Side effects and limitations of targeted therapy are also discussed. The document concludes that targeted therapies provide a more selective treatment approach compared to chemotherapy.
This document discusses targeted cancer therapy. It begins with an introduction to cancer classification and targeted therapy. It then discusses the epidemiology of cancer in India, signs and symptoms, and risk factors. It describes the goals and challenges of targeted therapy development. Targets for targeted therapy include monoclonal antibodies and small molecule inhibitors that target proteins involved in cancer signaling pathways. Treatment involves administration of targeted drugs through pills or IV. Side effects and limitations of targeted therapy are also discussed. The document concludes that targeted therapies provide more selective treatment compared to chemotherapy.
New Modern Way to Approach Cancer - 5th International Biobran WorkshopSheldon Stein
New Modern Way to Approach Cancer - 5th International Biobran Workshop Krakow , Poland June 9-11, 2017. Professor Jurasunas discusses the problems of conventional oncology and offers insight into less toxic and more efficient methods of cancer treatment. He offers case histories and reviews protocols
using Biobran and other modern methods against cancer, focusing on Strategic Immunotherapy, Apoptosis and Angiogenesis. He shows how Biobran improves cancer survival rates and metastases prevention and disease recurrence.
Cancer chemoprevention uses natural or laboratory-made substances to prevent cancer from developing. It is typically used by people at higher risk of cancer, such as those with a family history or previous cancer. Some chemopreventive agents studied include tamoxifen, raloxifene, aspirin and other NSAIDs. While chemoprevention may lower cancer risk, it also carries risks of side effects that must be weighed against the individual's cancer risk. Clinical trials test chemopreventive agents' safety and efficacy in delaying or preventing cancer. Targeted drug delivery seeks to concentrate medication in tissues of interest while reducing side effects by specifically targeting cancer cells over normal cells. Strategies include passive,
This document discusses targeted gene therapy and provides an overview of the topic. It begins with an introduction and then covers the history of gene therapy. The main types of gene therapy are described as germline therapy, ex vivo somatic gene therapy, and in vivo somatic gene therapy. The key steps in gene therapy are outlined, including identification of the affected gene, cloning the healthy gene, loading the vector, delivering the gene into target cells, and integration into the DNA. Various vector agents and routes of administration are discussed. Applications of gene therapy for cancer are summarized, including tumor suppressor genes, suicide genes, and anti-angiogenic therapy. The conclusion states that gene therapy has the potential to revolutionize medicine in the future.
Advaxis is developing a personalized neoepitope immunotherapy called ADXS-NEO to target mutations specific to a patient's cancer. Recent advances in genomics, immunotherapy, and cancer biology have enabled a new understanding of cancer as unique to each patient. ADXS-NEO uses DNA sequencing to identify tumor-specific mutations, engineers these neoepitopes into a bacterial vector, and administers this to activate the patient's immune system to target and eliminate the cancer. Advaxis has several clinical programs testing this approach across multiple cancer types with the goal of empowering each patient's immune system to fight their own unique form of cancer.
The document discusses cancer vaccines and their potential as anti-cancer drugs. It begins with background information on cancer types and stages. It then covers diagnostic and treatment options, including classification of anti-cancer drugs. The bulk of the document focuses on vaccines, describing how they can boost the immune system to treat existing cancers or prevent cancer development. It provides examples of specific cancer vaccines, discusses limitations and future prospects. The conclusion is that vaccines may be a promising biotechnological approach to curing, treating and preventing cancer.
Our fifth webinar in the MDC Connects Series 2021 | A Guide to Complex Medicines.
This slide deck takes a closer look at physicochemical characterisation new and novel approaches to understand the pharmacokinetics of complex drugs.
Juliana Maynard (MDC)
Immunotherapy is a treatment method for brain tumors that works by activating or suppressing the immune system. There are several types of immunotherapy including monoclonal antibody therapy, CAR T-cell therapy, checkpoint inhibitors, dendritic cell vaccines, and oncolytic viruses. Monoclonal antibodies target specific proteins on cancer cells to help the immune system find and kill them, while CAR T-cell therapy uses modified T cells to recognize and bind to tumor cells. Checkpoint inhibitors block proteins that stop the immune system from attacking cancer cells. Despite ongoing research, many immunotherapy techniques still face challenges in overcoming the blood-brain barrier and immunosuppressive environment of brain tumors.
Chemotherapy is the main treatment for disseminated cancers. It involves using multiple drugs in cycles to target rapidly dividing cancer cells. Common drugs include alkylating agents, antimetabolites, microtubule inhibitors, and monoclonal antibodies. Combination chemotherapy aims to maximize responses while avoiding overlapping toxicities. Doses are based on body surface area and adjusted for individual factors. Treatment intervals allow time for normal tissues to recover between cycles. Toxicities include myelosuppression, nausea/vomiting, and alopecia. Response is evaluated based on tumor shrinkage or progression.
Oncoceutics leerink global healthcare 2015oncoceutics
- Oncoceutics has discovered a novel class of compounds called ONC201 that shows compelling efficacy against aggressive and refractory tumors in preclinical studies.
- Phase I/II clinical trials are underway at leading cancer centers to evaluate ONC201's safety and efficacy in hematological malignancies and solid tumors.
- ONC201 engages multiple critical cancer pathways without toxicity, demonstrating potential as a first-in-class therapeutic for treatment-resistant cancers.
Cancer vaccines aim to stimulate the immune system to recognize and destroy cancer cells. There are two major types: specific vaccines target individual cancer types, while universal vaccines target common cancer antigens. Cancer vaccines work by exposing the immune system to tumor antigens, stimulating immune cells to develop a memory and mount an attack against cancer cells displaying those antigens. Currently, many types of cancer vaccines are under clinical trials, including antigen vaccines, dendritic cell vaccines, and DNA vaccines targeting cancers like melanoma, prostate cancer, and lung cancer. The goal is to develop safe and effective personalized or broad-spectrum vaccines to help treat and prevent cancer.
Cancer vaccines aim to stimulate the immune system to recognize and destroy cancer cells. There are two major categories: specific vaccines target individual cancer types, while universal vaccines can target many cancer types. Cancer vaccines work by exposing the immune system to tumor antigens, stimulating immune cells to produce a targeted attack on cancer. Current clinical trials are testing vaccines for cancers like melanoma, lung cancer, and prostate cancer, with some showing improved survival rates.
We can aid decision making from the pre-clinical to the clinical setting, supporting line of sight to the clinic, by identifying and translating crucial biomarker approaches into the real world.
This presentation focuses on the science of Gene Therapy, the techniques of germ-line and somatic gene therapy and the mechanism of curing diseases and disorders using gene therapy. The presentation starts by discussing some common basic terms from genetics and moves on to the historical development of gene therapy techniques in chronological order. The different types of gene therapy techniques and their mechanisms have been discussed in detail subsequently. In concluding slides, some commercially available gene therapy products are mentioned and challenges of gene-therapy techniques have been highlighted.
This document summarizes key information about the diagnosis and management of metastatic ocular melanoma. It discusses imaging and biopsy techniques used to detect metastasis, as well as liver-directed therapies like surgery, radioactive beads, and ablation. Systemic therapies covered include chemotherapy, immunotherapy like checkpoint inhibitors, and targeted therapies. Emerging treatments mentioned are IMCgp100 immunotherapy, hepatic perfusion chemotherapy, tumor infiltrating lymphocytes, and novel targeted therapies or combinations currently in clinical trials.
High Ki67 expression is an independent good prognostic marker in colorectal cancer. The study analyzed 1800 colorectal cancer specimens and found that tumors with high Ki67 expression (>25% of cells staining positive) were associated with improved patient survival outcomes. While most studies find high Ki67 expression correlates with poorer prognosis in other cancers, this study and others have found the opposite relationship in colorectal cancer. The reasons for this difference are unclear. The results suggest patients with low Ki67 expression CRC tumors may benefit most from adjuvant chemotherapy.
This document summarizes a grant-funded project called STEMVAC that aims to develop new immunotherapies for breast cancer. The project brings together 5 partners including academic institutions and pharmaceutical companies. It will identify antigens specifically expressed by breast cancer stem cells using transcriptomic and proteomic analysis. These antigens will then be developed into cancer vaccines and targeted monoclonal antibody therapies with the goal of entering clinical trials. The project has the potential to create new treatment options for breast cancer patients and generate economic benefits for the region through job creation.
This document discusses targeted cancer therapy. It begins with an introduction to cancer classification and targeted therapy. It then discusses epidemiology, signs and symptoms, and risk factors of cancer. It describes the goals and challenges of targeted therapy development. Targets for targeted therapy include monoclonal antibodies and small molecule inhibitors that target proteins involved in cancer signaling pathways. Treatment involves administration of targeted drugs through intravenous methods. Side effects can include skin, gastrointestinal and blood problems. Limitations include cancer cells developing resistance. Present therapies also include immunotherapy and nanotechnology.
This document discusses targeted cancer therapy. It begins with an introduction to cancer classification and targeted therapy. It then discusses the epidemiology, signs and symptoms, and risk factors of cancer. It describes the goals and challenges of targeted therapy development. Targets for targeted therapy include monoclonal antibodies and small molecule inhibitors that target proteins involved in cancer signaling pathways. Treatment involves targeted drugs administered through pills or IV. Side effects and limitations of targeted therapy are also discussed. The document concludes that targeted therapies provide a more selective treatment approach compared to chemotherapy.
This document discusses targeted cancer therapy. It begins with an introduction to cancer classification and targeted therapy. It then discusses the epidemiology of cancer in India, signs and symptoms, and risk factors. It describes the goals and challenges of targeted therapy development. Targets for targeted therapy include monoclonal antibodies and small molecule inhibitors that target proteins involved in cancer signaling pathways. Treatment involves administration of targeted drugs through pills or IV. Side effects and limitations of targeted therapy are also discussed. The document concludes that targeted therapies provide more selective treatment compared to chemotherapy.
New Modern Way to Approach Cancer - 5th International Biobran WorkshopSheldon Stein
New Modern Way to Approach Cancer - 5th International Biobran Workshop Krakow , Poland June 9-11, 2017. Professor Jurasunas discusses the problems of conventional oncology and offers insight into less toxic and more efficient methods of cancer treatment. He offers case histories and reviews protocols
using Biobran and other modern methods against cancer, focusing on Strategic Immunotherapy, Apoptosis and Angiogenesis. He shows how Biobran improves cancer survival rates and metastases prevention and disease recurrence.
Cancer chemoprevention uses natural or laboratory-made substances to prevent cancer from developing. It is typically used by people at higher risk of cancer, such as those with a family history or previous cancer. Some chemopreventive agents studied include tamoxifen, raloxifene, aspirin and other NSAIDs. While chemoprevention may lower cancer risk, it also carries risks of side effects that must be weighed against the individual's cancer risk. Clinical trials test chemopreventive agents' safety and efficacy in delaying or preventing cancer. Targeted drug delivery seeks to concentrate medication in tissues of interest while reducing side effects by specifically targeting cancer cells over normal cells. Strategies include passive,
This document discusses targeted gene therapy and provides an overview of the topic. It begins with an introduction and then covers the history of gene therapy. The main types of gene therapy are described as germline therapy, ex vivo somatic gene therapy, and in vivo somatic gene therapy. The key steps in gene therapy are outlined, including identification of the affected gene, cloning the healthy gene, loading the vector, delivering the gene into target cells, and integration into the DNA. Various vector agents and routes of administration are discussed. Applications of gene therapy for cancer are summarized, including tumor suppressor genes, suicide genes, and anti-angiogenic therapy. The conclusion states that gene therapy has the potential to revolutionize medicine in the future.
Advaxis is developing a personalized neoepitope immunotherapy called ADXS-NEO to target mutations specific to a patient's cancer. Recent advances in genomics, immunotherapy, and cancer biology have enabled a new understanding of cancer as unique to each patient. ADXS-NEO uses DNA sequencing to identify tumor-specific mutations, engineers these neoepitopes into a bacterial vector, and administers this to activate the patient's immune system to target and eliminate the cancer. Advaxis has several clinical programs testing this approach across multiple cancer types with the goal of empowering each patient's immune system to fight their own unique form of cancer.
The document discusses cancer vaccines and their potential as anti-cancer drugs. It begins with background information on cancer types and stages. It then covers diagnostic and treatment options, including classification of anti-cancer drugs. The bulk of the document focuses on vaccines, describing how they can boost the immune system to treat existing cancers or prevent cancer development. It provides examples of specific cancer vaccines, discusses limitations and future prospects. The conclusion is that vaccines may be a promising biotechnological approach to curing, treating and preventing cancer.
Our fifth webinar in the MDC Connects Series 2021 | A Guide to Complex Medicines.
This slide deck takes a closer look at physicochemical characterisation new and novel approaches to understand the pharmacokinetics of complex drugs.
Juliana Maynard (MDC)
Immunotherapy is a treatment method for brain tumors that works by activating or suppressing the immune system. There are several types of immunotherapy including monoclonal antibody therapy, CAR T-cell therapy, checkpoint inhibitors, dendritic cell vaccines, and oncolytic viruses. Monoclonal antibodies target specific proteins on cancer cells to help the immune system find and kill them, while CAR T-cell therapy uses modified T cells to recognize and bind to tumor cells. Checkpoint inhibitors block proteins that stop the immune system from attacking cancer cells. Despite ongoing research, many immunotherapy techniques still face challenges in overcoming the blood-brain barrier and immunosuppressive environment of brain tumors.
Chemotherapy is the main treatment for disseminated cancers. It involves using multiple drugs in cycles to target rapidly dividing cancer cells. Common drugs include alkylating agents, antimetabolites, microtubule inhibitors, and monoclonal antibodies. Combination chemotherapy aims to maximize responses while avoiding overlapping toxicities. Doses are based on body surface area and adjusted for individual factors. Treatment intervals allow time for normal tissues to recover between cycles. Toxicities include myelosuppression, nausea/vomiting, and alopecia. Response is evaluated based on tumor shrinkage or progression.
Oncoceutics leerink global healthcare 2015oncoceutics
- Oncoceutics has discovered a novel class of compounds called ONC201 that shows compelling efficacy against aggressive and refractory tumors in preclinical studies.
- Phase I/II clinical trials are underway at leading cancer centers to evaluate ONC201's safety and efficacy in hematological malignancies and solid tumors.
- ONC201 engages multiple critical cancer pathways without toxicity, demonstrating potential as a first-in-class therapeutic for treatment-resistant cancers.
Cancer vaccines aim to stimulate the immune system to recognize and destroy cancer cells. There are two major types: specific vaccines target individual cancer types, while universal vaccines target common cancer antigens. Cancer vaccines work by exposing the immune system to tumor antigens, stimulating immune cells to develop a memory and mount an attack against cancer cells displaying those antigens. Currently, many types of cancer vaccines are under clinical trials, including antigen vaccines, dendritic cell vaccines, and DNA vaccines targeting cancers like melanoma, prostate cancer, and lung cancer. The goal is to develop safe and effective personalized or broad-spectrum vaccines to help treat and prevent cancer.
Cancer vaccines aim to stimulate the immune system to recognize and destroy cancer cells. There are two major categories: specific vaccines target individual cancer types, while universal vaccines can target many cancer types. Cancer vaccines work by exposing the immune system to tumor antigens, stimulating immune cells to produce a targeted attack on cancer. Current clinical trials are testing vaccines for cancers like melanoma, lung cancer, and prostate cancer, with some showing improved survival rates.
We can aid decision making from the pre-clinical to the clinical setting, supporting line of sight to the clinic, by identifying and translating crucial biomarker approaches into the real world.
This presentation focuses on the science of Gene Therapy, the techniques of germ-line and somatic gene therapy and the mechanism of curing diseases and disorders using gene therapy. The presentation starts by discussing some common basic terms from genetics and moves on to the historical development of gene therapy techniques in chronological order. The different types of gene therapy techniques and their mechanisms have been discussed in detail subsequently. In concluding slides, some commercially available gene therapy products are mentioned and challenges of gene-therapy techniques have been highlighted.
This document summarizes key information about the diagnosis and management of metastatic ocular melanoma. It discusses imaging and biopsy techniques used to detect metastasis, as well as liver-directed therapies like surgery, radioactive beads, and ablation. Systemic therapies covered include chemotherapy, immunotherapy like checkpoint inhibitors, and targeted therapies. Emerging treatments mentioned are IMCgp100 immunotherapy, hepatic perfusion chemotherapy, tumor infiltrating lymphocytes, and novel targeted therapies or combinations currently in clinical trials.
High Ki67 expression is an independent good prognostic marker in colorectal cancer. The study analyzed 1800 colorectal cancer specimens and found that tumors with high Ki67 expression (>25% of cells staining positive) were associated with improved patient survival outcomes. While most studies find high Ki67 expression correlates with poorer prognosis in other cancers, this study and others have found the opposite relationship in colorectal cancer. The reasons for this difference are unclear. The results suggest patients with low Ki67 expression CRC tumors may benefit most from adjuvant chemotherapy.
This document summarizes a grant-funded project called STEMVAC that aims to develop new immunotherapies for breast cancer. The project brings together 5 partners including academic institutions and pharmaceutical companies. It will identify antigens specifically expressed by breast cancer stem cells using transcriptomic and proteomic analysis. These antigens will then be developed into cancer vaccines and targeted monoclonal antibody therapies with the goal of entering clinical trials. The project has the potential to create new treatment options for breast cancer patients and generate economic benefits for the region through job creation.
2. HISTORY
1992
Biotechnology company called:
Activated Cell Therapy
Moutain View, California
Dr Edgar Dr Samuel
Engleman Strobber
•First activity : isolating hematopoietic stem cells from blood for use in patients with cancer
who require transplantation.
2
3. HISTORY
After several years : Activated Cell Therapy
• Activity shift : developing therapeutic products that fight cancer
by manipulating aspects of the immune system
Seattle, Washington state
« Targeting Cancer, Transforming Lives »
• Today:
o CEO : Mitchell H. Gold
o 650 employees (April 2010)
o Main Manufacturing facility in Morris Plain (NJ)
3
4. FUNDING
•DENDREON came public in 2000 (NASDAQ) : $10 per share
•Major shareholders:
o Mutual Fund : Fidelity Growth Company Fund: 9.50% of shares held
o Individual Investors : David L Urdal (Executive vice president of Dendreon): 0.36% of
shares held
February 13, 2011: $35.15
4
5. ACTIVITY
DENDREON is focused on the discovery, development and commercialization:
• of novel therapeutics
• that may significantly improve cancer treatment options for patients
Philosophy of Dendreon :
produce Active Cellular Immunotherapy products
stimulate an immune response against a variety of tumor types
5
6. CANCER THERAPIES
• Cancer is characterized by abnormal cells that grow and proliferate,
• forming masses called tumors
• Cancer therapies must
eliminate or
the growth of the cancer
control
Chemotherapy Hormone treatments Surgery Radiation
•BUT :
may not have the desired therapeutic effect
may result in significant detrimental side effects
New approach to Cancer Treatment:
IMMUNOTHERAPIES 6
8. RESEARCH ACTIVITY
•First step : to find antigens expressed on cancer cells that are suitable targets for cancer therapy
Internal antigen License
discovery program agreements
8
9. RESEARCH ACTIVITY
Second step: to engineer antigens designed to stimulate and maximize cell-mediated immunity
Creation of the “Antigen Delivery Cassette™”
= The key to robuste immune response
Aim : to raise the quality and the quantity of the immune response
9
11. ESTIMATED NEW CASES
PROSTATE
•Estimated new cases and deaths in 2010 (US) :
New cases: 217,730
Deaths: 32,050
11
The second most common type of cancer among men in the USA
12. PROSTATE CANCER
Diagnosis Symptoms Stages
•Average age when •Often asymptomatic •Low growth
diagnosed:70 years at the beginning
•Physical examination •Pain •Hormono-
dependant
• Dosing •Difficulty in urinating
Prostate
Specific •Problems during
Antigen sexual intercourse
•Biopsy => Gleason •Erectile dysfunction •Hormono-
score independant
• Such as Benign after one to three years and resume
growth despite hormone therapy.
Prostatic Hyperplasia
12
13. PROVENGE®: Active Cell Immunotherapy
applied to Prostate Cancer
3 actors:
Recombinant antigen: composed of
Prostatic Acid Phosphatase (expressed in more than 95% of
prostate cancers cells)
GM-CSF : Granulocyte-macrophage colony-stimulating factor
Antigen Presenting Cell: white blood cells removed from the
patient through LEUKAPHERESIS
T-Cells: actived by the APC-PAP-GM-CSF, attack the tumor cells
13
25. PROVENGE SALES
Total in 2010 : $48 M
• About only 500 patients were treated in 2010 (in 8 months)
• Expectations (2009) : to treat 8% of the Asymptomatic Metastatic AIPC market7300 patients
we can expect a large progression
25
26. What’s next for PROVENGE in the
USA?
Expectations in 2014:
•Market shares = 35%
•Patients treated= 38,628
BUT can DENDREON provide enough
Provenge® to meet demand?
•Enough Recombinant Prostatic Antigen?
•Enough Infusions Centers?
•Enough Manufacturing capacity?
2011: A YEAR OF GROWTH
26
27. Supply of the recombinant
Antigen
• DENDREON doesn’t produce
the antigen itself
• The company utilizes third party suppliers to
manufacture and package the recombinant antigene
• First collaboration :
• Since September 2010 , second supplier :
27
28. MANUFACTURING FACILITIES
Los Angeles
Mid 2011
• New-Jersey : additional capacity expected in march 2011
• Atlanta & LA: additional capacity starting in mid-2011
28
30. INFUSIONS CENTERS
Increase of number of Infusion centers by 9 fold in 2011 for DENDREON to be
near their patients 30
31. OTHER ISSUES FOR 2011
SALES FORCES
• Increase of the sales forces to approximatly 100 reps to service 450 centers by the end of 2011
Significant increase in outreach to maximize the additional capacity
REIMBURSEMENT
• Will CMS recommend and provide national reimbursement?
•Date of national decision : March, 30,2011
•But some local Medicare contractors already reimburse PROVENGE®
Good clue for a positive decision by CMS
31
32. EMERGING COMPETITORS FOR
PROSTATE CANCER
Trade name Type of treatment Company Phase
PROSTVAC® Viral vector Bavarian Nordic Phase II completed
GVAX GM-CSF gene- BioSante Phase III
transfected cell Pharmaceuticals
vaccines
DCVAX Prostate Cellular vaccine Northwest Phase III
Biotherapeutics
TROVAX® Viral vector Oxford Biomedical Phase III
IPILIMUMAB Monoclonal antibodies BMS Phase III
ABIRATERONE Hormonotherapy Janssen-Cilag Phase III
32
33. PIPELINE
Extension of Indication: Androgen Dependent Prostate Cancer
(phase 3 : awaiting data on overall survival)
Potential Raise of the market for PROVENGE® 33
36. HER2/neu
= Human Epidermal Growth Factor Receptor 2
Membrane Glycoprotein involved in cell growth and differenciation
Composed of:
• an extracellular domain for binding ligands
• a single transmembrane segment
• an intracellular domain carrying tyrosine-kinase activity
36
37. BREAST CANCER and HER2/neu
Total : 207,090 (USA)
Total : 207,090
The HER2 protein is overexpressed in about 30% of all breast
cancers
37
38. BREAST CANCER and HER2/neu
One drug already targetting HER2 : Trastuzumab HERCEPTIN®
Recombinant humanised IgG1 monoclonal antibody
38
39. HER2/neu
Opportunities in different solid tumors expressing HER2/neu
• Breast
• Ovarian
• Colorectal
• Bladder
Initiate Phase 2 trial 1Q 2011
Lapuleucel-T NEUVENGE®
39
40. BLADDER CANCER & HER2/neu
Bladder cancer : 70,530 new cases in 2010 (USA)
The 4th most frequent cancer in men
HER2 expression in bladder cancer : very variable between the different studies (from 9 to
81%)
WHY TO CONDUCT A CLINICAL TRIAL IN BLADDER CANCER AND NOT IN BREAST
CANCER?
HER2 Cancer market : HERCEPTIN® No indication in the bladder cancer
Neuvenge® targeting HER2 in breast Neuvenge® targeting HER2 in
cancer : Vs HERCEPTIN? bladder cancer : Vs placebo?
40
42. CA-9
•In-licensed from Bayer Corporation, Business Group Diagnostics
= Carbonic Anhydrase IX
•Transmembrane protein involved in cell proliferation
the only tumor-associated carbonic anhydrase isoenzyme known
Tumors over-expressing CA-9:
•Colon
•Cervical
•Kidney
CA-9 is overexpressed in 75% of Renal cell
Carcinoma
phase 1 in Renal Cell Carcinoma planned in 2011
42
44. CEA
• In-licensed from Bayer Corporation, Business Group Diagnostics
=CarcinoEmbryonic Antigen : glycoprotein involved in cell adhesion
• Not usually present in healthy adults, although levels are raised in heavy smokers
Cancers expressing CEA :
• Breast (65%)
• Lung (70%) Phase 1 expected in 2012
• Colon
44
45. DIFFICULTIES FOR ACI
PRODUCTS DEVELOPMENT
Long
studies
Manufacturing Huge
Antigens logistic
ACI
Exclusion
Ethic : vs
of HIV,
placebo?
HepB- C
45
46. Small Molecule Active Cellular Immunotherapies
Pre-clinical
Phase 2
Phase 3
Market
46
47. TRPM8
=Transient Receptor Potential Cation Channel subfamily M member 8
• transmembrane cation channel identified through Dendreon’s internal antigen discovery
program
Patent on the gene in 2001
Over-expressed in :
• 100% of prostate cancers
• 71% of breast cancers
• 93% of colon cancers Attractive target
• 80% of lung cancers
Synthesis of small molecule agonists
47
48. TRPM8: Mechanism of Action
Activation by agonist induces Ca++ to flow into cells APOPTOSIS
•This small molecule agonist is orally available
Clinical phase 1 trial ongoing
48
52. DENDREON’S FIRST TARGET
EUROPE « Europe is our first ex-US opportunity »
•Market for metastatic AIPC patients = 1.5X to 2X US
•Overall market characteristics similar to US
Both urologists & oncologists are involved in treatment
Treatment paradigms similar
Significant therapeutic unmet need remains
•DENDREON CEO Mitch Gold :
« Low rates of PSA testing in Europe meant that many men
arrived in their physicians office with metastatic disease »
52
54. WHAT ABOUT LICENSING?
1998: license agreement : rights for
PROVENGE in Asia and Pacific countries
2003 : released its
rights for
PROVENGE
54
55. LICENSING?
Advantages
• Greater local knowlege of the Regulatory agencies by the licensee.
• Better manufacturing capacity of the licensee
• No administrative expenses and no cost of good solds for Dendreon
(PROVENGE® commercialization needs much money)
Disadvantage
•DENDREON will depend on the skills, abilities and ressources of the licensee as a
source of revenue dependence
55
56. DENDREON’S CHOICE : to go
alone in EUROPE
Why this choice?
•2 hypothesis: They want 100% of worldwide rights
Own will of DENDREON
They don’t want to share their revenues
Corporate image of growth
Choice by default: they didn’t find any partners?
No certitude to get an european
•Too risky? approval
Reimbursement?
Provenge « is not just a pill
in a bottle » 56
57. WHAT DO THEY NEED?
TRIALS MONEY
IMPACT D9901 D9902 Provenge Senior Notes
58. TRIALS & REGULATORY
• Advanced Therapy Medicinal Product (ATMP) Annex IV of directive
2003/63/CE
Cellular Therapy
Via Centralised Procedure
Same dossier as for a medicinal product with technical
adaptations
• DENDREON wants to rely on its IMPACT trial, conducted in the US
BUT: Will it be acceptable in EU?
58
61. IMPACT TRIAL:
NEGATIVE POINTS?
IMPACT TRIAL
Primary endpoint: Overall survival
trials done versus placebo: ethic problems, same efficiency
versus taxotere?
patients having metastases: what medicine did they take before? (docetaxel
approval for prostate cancer by FDA: 19/05/2004)
ethnic population isn’t the same and ethny changes impact of the
disease
Secondary endpoint: Time to objective disease survival
FDA agreed to allow Dendreon to amend the design of the IMPACT study
61
62. REIMBURSEMENT CHALLENGE
Market access and reimbursement success is key to realizing full product potential in
E.U.
Key factors influencing reimbursment:
• overall survival is the « gold standart » for payers
IMPACT: 4 months survival benefits against placebo…
• total cost of care is taken into account
$93,000/ complete cost treatement for Provenge VS $18,000/ 6 cycles
of treatment for taxotere
lack of required premedication and supportive care costs compared to
Taxotere
62
63. WHERE TO HAVE A PLACE?
• Find a strategic place in EUROPE
wich must be:
Near airport and road network
In a reasonable distance from each European capital
Able to cover the majority of the market
• DENDREON’s decision to build its manufacturing site: GERMANY
50% of patients live in less than 8 hours to this site in car or flight
63
64. WHERE TO HAVE A PLACE?
• During DENDREON submits an European authorization (late 2011/early 2012)
Initial manufacturing through a Contract Manufacturing
Organization (CMO):
Qualifying a CMO can be done faster than plant construction
Dendreon expects to save 12 to 18 months by outsourcing to support filing.
• Concurrently DENDREON will build its first manufacturing site:
Initiate built out in 2011
Huge expenses!!
64
65. WITH WICH MONEY?
Revenues from Provenge : $48 millions in 2010
January, 14th 2011: Dendreon announced the pricing of a publing offering of $540
million convertible senior notes
Raise the equity : in the beginning of 2010 : public offering of 15 Million shares
65
67. Strengths Weaknesses
- ACI : revolutionary therapeutic approach - Huge logistic :
Expensive
- ACI : less AEs than chemotherapy Restrictions for Clinical trials
- One drug on the market at least : Provenge - Provenge sales too low compared to
revenues expectations
- Huge logistic : difficult to copy for generics - Increase of debts (senior notes)
- No profit yet
SWOT
Opportunities Threats
- Expansion
-Decision for reimbursment of Provenge
In the USA
expected in March
In Europe : similarity with US
market
- Emerging competitors
-Provenge : new indication in development
- At the mercy of the EMA for the approval of
Provenge (clinical trial vs. Taxotere?)
- ACI : repeatible with new Antigens
other cancers targeted
67
71. WOULD WE JOIN
DENDREON?
Cancer treatment is a « noble » domain
Working for a revolutionary process as ACI must be exciting
Transition from a total R&D to a fully-integrated
commercial company
Development of domains corresponding to our professional
expectations
Regulatory Challenge in Europe Development of new ACI products
New jobs in Regulatory Affairs Evaluation of new markets
71
CMS = CENTERS FOR MEDICARE AND MEDICAID SERVICE Svt les assurances privees suivent les tx de remboursement de medicaid et medicare plus de patients pourront avoir acces au ttmt
entraine l'internalisation du récepteurs HER2, ce qui le rend inactif ; bloque leur dimérisation donc aucune activité kinase n’est possible ; stimule la formation de tétramère de protéine Her2, une conformation non propice à l’activité kinasique. Chacun de ces trois mécanismes empêchent l'activation des récepteurs HER2 et donc la prolifération cellulaire