Medicenna is developing MDNA55, a targeted immunotherapy for the treatment of recurrent glioblastoma and other cancers. Phase 1/2 clinical trials of MDNA55 showed no drug-related serious adverse events and signs of anti-tumor activity. Medicenna is currently enrolling patients in a Phase 2b clinical trial to further evaluate the safety and efficacy of MDNA55 in recurrent glioblastoma. Medicenna is also using its Superkine platform to develop new immunotherapies for cancer and other diseases by engineering interleukins to have improved anti-tumor effects.
Medicenna corporate presentation renmark fm printablemedicenna2016
This corporate presentation provides an overview of Medicenna Biopharma Corp., a clinical-stage biopharmaceutical company focused on developing targeted immunotherapies for cancer. Key points include:
- MDNA55 is Medicenna's lead candidate for treating recurrent glioblastoma and has received orphan drug designation from the FDA and EMA. Phase 1/2 clinical data showed compelling efficacy and safety results.
- Medicenna is currently enrolling patients in a Phase 2b clinical trial of MDNA55 for recurrent glioblastoma and plans to expand into other brain cancer indications.
- The company is developing a pipeline of "superkine" versions of cytokines like IL-2 and IL-4 with potential applications across multiple
This corporate presentation by Medicenna outlines their lead candidate MDNA55 for the treatment of glioblastoma and other IL4R-expressing cancers. MDNA55 is a targeted immunotherapy currently in Phase 2 clinical trials for recurrent glioblastoma. The presentation highlights compelling efficacy data from previous studies of MDNA55 and outlines plans to improve outcomes through second generation convection enhanced delivery. It also discusses Medicenna's pipeline of IL-2 and IL-4 superkines and preclinical programs, as well as upcoming clinical milestones and financial position.
Medicenna is a clinical-stage biotech company developing MDNA55, a targeted immunotherapy for the treatment of glioblastoma and other IL4R-expressing cancers. MDNA55 has shown promising efficacy and safety results in Phase 2 clinical trials for recurrent glioblastoma. Medicenna is currently enrolling patients in a Phase 2b clinical trial evaluating MDNA55 for recurrent glioblastoma and plans to initiate additional clinical trials in brain and other cancers in 2018. The company has a deep pipeline of IL-2 and IL-4 superkines and is well funded through non-dilutive grants and an equity financing to advance its programs over the next two years.
Professor of Neurosurgery at Stanford University;
Inventor of MDNA55
Sam Denmeade, MD
Johns Hopkins University:
Chief Scientific Officer; Expert in Prostate Cancer Therapeutics
David M. Nanus, MD
Memorial Sloan Kettering Cancer Center:
Expert in Immunotherapy for Solid Tumors
David Spetzler, MD, PhD
CSO, Xencor:
Expert in Protein Engineering and Antibody Therapeutics
John Sampson, MD, PhD
Duke University:
Principal Investigator and Expert in Drug Delivery to the Brain
17
One in 8 U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. Following breast cancer surgery in the adjuvant setting, a HER2/neu 3+ patient typically receives Herceptin® in the first year, with the hope that their breast cancer will not recur, and with the odds of recurrence slowly decreasing over the first 5 years after surgery. Herceptin® has been shown to reduce recurrence rates from 25% to 12% in the adjuvant setting. In the neoadjuvant setting, a patient receives treatment before surgery and based on the results of a biopsy at surgery, will receive the same or more potent treatment after surgery. Kadcyla® has been shown to reduce recurrence rates from 22% to 11% in the neoadjuvant setting. Accordingly, we believe that GP2 may be used to address the 50% of recurring patients who do not respond to either Herceptin® or Kadcyla®.
Greenwich LifeSciences (the “Company”) is a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery. GP2 is a 9 amino acid transmembrane peptide of the HER2/neu (human epidermal growth factor receptor 2) protein, a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or “over-expressor”) levels.
Aridis Pharmaceuticals is a late-stage clinical development company, leading the creation of transformative, first-in-class anti-infectives for life-threatening viral and bacterial respiratory infections. The company’s lead drug candidate for acute pneumonia met all endpoints for its phase 2 clinical trial and the Company is now preparing for a phase 3 study. Its pipeline of novel mechanism antibacterial and antivirals, sprung from its proprietary technology platforms, are designed to combat the growing public health threat of viral pandemics and antimicrobial resistant (AMR) bacteria. Its anti-toxin monoclonal antibody approach is a proven Mechanism of Action and has shown efficacy as a combination therapy in reducing acute pneumonia patients time in ICU as compared to the current standard of care, which is antibiotics. In addition to its phase 3 program, ARDS also has a phase 2 asset for the treatment of Bacteria HAP/VAP LPS 011. It also recently began enrolling for a phase 2 clinical trial for the treatment of Cystic Fibrosis patients with acute pneumonia, a program funded by the Cystic Fibrosis Foundation for $7.5 million. Importantly, ARDS is also preparing to launch a Phase 1/2 clinical trial in the second half of 2021 with its novel inhalation antibody technology for emerging COVID-19 mutated variants. The expansion of COVID virus strain coverage, combined with the product's self-administered, at-home treatment modality, further differentiates the company's AR-712 COVID treatment offering.
MedChemica - Automated Extraction of Actionable Knowledge from Large Scale in...Al Dossetter
This document discusses the importance of stereochemistry in medicinal chemistry research and drug development. It notes that many approved drugs are chiral molecules where the specific stereochemistry is important. Better rules for medicinal chemistry could help reduce high drug development costs by improving predictions of properties like absorption, distribution, metabolism, and excretion. The document advocates mining large datasets of in vitro pharmacology data to extract actionable knowledge about stereochemistry and its effects on important drug properties and clinical outcomes. This could help medicinal chemists design safer and more effective compounds with lower attrition rates in development.
Medicenna corporate presentation renmark fm printablemedicenna2016
This corporate presentation provides an overview of Medicenna Biopharma Corp., a clinical-stage biopharmaceutical company focused on developing targeted immunotherapies for cancer. Key points include:
- MDNA55 is Medicenna's lead candidate for treating recurrent glioblastoma and has received orphan drug designation from the FDA and EMA. Phase 1/2 clinical data showed compelling efficacy and safety results.
- Medicenna is currently enrolling patients in a Phase 2b clinical trial of MDNA55 for recurrent glioblastoma and plans to expand into other brain cancer indications.
- The company is developing a pipeline of "superkine" versions of cytokines like IL-2 and IL-4 with potential applications across multiple
This corporate presentation by Medicenna outlines their lead candidate MDNA55 for the treatment of glioblastoma and other IL4R-expressing cancers. MDNA55 is a targeted immunotherapy currently in Phase 2 clinical trials for recurrent glioblastoma. The presentation highlights compelling efficacy data from previous studies of MDNA55 and outlines plans to improve outcomes through second generation convection enhanced delivery. It also discusses Medicenna's pipeline of IL-2 and IL-4 superkines and preclinical programs, as well as upcoming clinical milestones and financial position.
Medicenna is a clinical-stage biotech company developing MDNA55, a targeted immunotherapy for the treatment of glioblastoma and other IL4R-expressing cancers. MDNA55 has shown promising efficacy and safety results in Phase 2 clinical trials for recurrent glioblastoma. Medicenna is currently enrolling patients in a Phase 2b clinical trial evaluating MDNA55 for recurrent glioblastoma and plans to initiate additional clinical trials in brain and other cancers in 2018. The company has a deep pipeline of IL-2 and IL-4 superkines and is well funded through non-dilutive grants and an equity financing to advance its programs over the next two years.
Professor of Neurosurgery at Stanford University;
Inventor of MDNA55
Sam Denmeade, MD
Johns Hopkins University:
Chief Scientific Officer; Expert in Prostate Cancer Therapeutics
David M. Nanus, MD
Memorial Sloan Kettering Cancer Center:
Expert in Immunotherapy for Solid Tumors
David Spetzler, MD, PhD
CSO, Xencor:
Expert in Protein Engineering and Antibody Therapeutics
John Sampson, MD, PhD
Duke University:
Principal Investigator and Expert in Drug Delivery to the Brain
17
One in 8 U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. Following breast cancer surgery in the adjuvant setting, a HER2/neu 3+ patient typically receives Herceptin® in the first year, with the hope that their breast cancer will not recur, and with the odds of recurrence slowly decreasing over the first 5 years after surgery. Herceptin® has been shown to reduce recurrence rates from 25% to 12% in the adjuvant setting. In the neoadjuvant setting, a patient receives treatment before surgery and based on the results of a biopsy at surgery, will receive the same or more potent treatment after surgery. Kadcyla® has been shown to reduce recurrence rates from 22% to 11% in the neoadjuvant setting. Accordingly, we believe that GP2 may be used to address the 50% of recurring patients who do not respond to either Herceptin® or Kadcyla®.
Greenwich LifeSciences (the “Company”) is a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery. GP2 is a 9 amino acid transmembrane peptide of the HER2/neu (human epidermal growth factor receptor 2) protein, a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or “over-expressor”) levels.
Aridis Pharmaceuticals is a late-stage clinical development company, leading the creation of transformative, first-in-class anti-infectives for life-threatening viral and bacterial respiratory infections. The company’s lead drug candidate for acute pneumonia met all endpoints for its phase 2 clinical trial and the Company is now preparing for a phase 3 study. Its pipeline of novel mechanism antibacterial and antivirals, sprung from its proprietary technology platforms, are designed to combat the growing public health threat of viral pandemics and antimicrobial resistant (AMR) bacteria. Its anti-toxin monoclonal antibody approach is a proven Mechanism of Action and has shown efficacy as a combination therapy in reducing acute pneumonia patients time in ICU as compared to the current standard of care, which is antibiotics. In addition to its phase 3 program, ARDS also has a phase 2 asset for the treatment of Bacteria HAP/VAP LPS 011. It also recently began enrolling for a phase 2 clinical trial for the treatment of Cystic Fibrosis patients with acute pneumonia, a program funded by the Cystic Fibrosis Foundation for $7.5 million. Importantly, ARDS is also preparing to launch a Phase 1/2 clinical trial in the second half of 2021 with its novel inhalation antibody technology for emerging COVID-19 mutated variants. The expansion of COVID virus strain coverage, combined with the product's self-administered, at-home treatment modality, further differentiates the company's AR-712 COVID treatment offering.
MedChemica - Automated Extraction of Actionable Knowledge from Large Scale in...Al Dossetter
This document discusses the importance of stereochemistry in medicinal chemistry research and drug development. It notes that many approved drugs are chiral molecules where the specific stereochemistry is important. Better rules for medicinal chemistry could help reduce high drug development costs by improving predictions of properties like absorption, distribution, metabolism, and excretion. The document advocates mining large datasets of in vitro pharmacology data to extract actionable knowledge about stereochemistry and its effects on important drug properties and clinical outcomes. This could help medicinal chemists design safer and more effective compounds with lower attrition rates in development.
Greenwich LifeSciences (the “Company”) is a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery. GP2 is a 9 amino acid transmembrane peptide of the HER2/neu (human epidermal growth factor receptor 2) protein, a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or “over-expressor”) levels.
How we Built a Large Scale Matched Pair Analysis Engine (MCPairs) using OpenE...Al Dossetter
MCPairs performed Matched Molecular Pair Analysis on large scale to build databases of exploitable knowledge which is accessible for Drug Discovery to accelerate research projects. The talk describes how we did this and some of the challenges.
MedChemica BigData What Is That All About?Al Dossetter
A light look at the world of BigData for the lay person - a look at a couple of examples and what we do in MedChemica to speed up drug discovery. First presented at Macclesfield SciBar, and then Knutsford SciBar.
MedChemica Active Learning - Combining MMPA and MLAl Dossetter
Describes MedChemica research on combining Matched Molecular Pair Analysis (MMPA) and Machine Learning (ML) into a closed loop to find and optimize new hits for drug discovery. The talks describes the MMPA and Regression Forest models and how they were combined and some early conclusion. Of these permutative MMPA is the clear winner (Free Wilson ++)
Accelerating multiple medicinal chemistry projects using Artificial Intellige...Al Dossetter
The technical methods and results of Matched Molecular Pair Analysis (MMPA) applied from a small, individual assay scale through large pharma scale, to multiple pharma data sharing scale have been published and reviewed. The drive behind these efforts has been to derive a medicinal chemistry knowledge base (i.e. definitive textbook) that can be applied to drug discovery projects. The aim is to greatly decrease the time in lead identification and optimization by the synthesis of fewer compounds. Such a system suggests compound designs to expert chemists to triage; such a process is Artificial Intelligence (AI). Given this context, how does this work on projects? How do the chemists make decisions? What are the results? The talk will answer these questions through project examples where MMPA has been applied and how this led to drug candidates. The projects disclosed are from multiple organisations and describe Cathepsin K inhibitors, Glucokinase Inhibitors, 11β-Hydroxysteroid Dehydrogenase Type I Inhibitors (11β-HSD1), Ghrelin inverse antagonists and Tubulin Polymerization inhibitors. An overview of MMPA will be presented and each project will be briefly described with a focus on how the chemists used MMPA to understand SAR and design compounds. The impact of project progress to CD will be quantified.
Immuron Limited is a clinical stage biopharmaceutical company developing oral immunotherapies to treat inflammatory and infectious diseases. It has two lead clinical assets in Phase 2 development for non-alcoholic steatohepatitis (NASH) and Clostridium difficile infection. Immuron's lead candidate, IMM-124E, is being studied in three Phase 2 clinical trials for NASH, alcoholic steatohepatitis, and pediatric non-alcoholic fatty liver disease, with data expected in late 2017 and 2018. IMM-124E works through broad anti-inflammatory effects and has demonstrated a strong anti-fibrotic effect in preclinical models of liver disease.
The talk describes the science and results of a consortium of multiple pharmaceutical companies extracting medicinal chemistry knowledge from research data and the application to real drug design projects. A new technique for automating pharmacophore / toxophore finding from public data is disclosed.
The document summarizes the 2016 annual meeting of stockholders held on July 14, 2016. It includes a forward-looking statement noting risks and uncertainties in projections. The development pipeline outlines several investigational products in various phases of clinical trials for breast cancer, gastric cancer, gynecological cancers, and hematological conditions. An overview of the current status of NeuVax clinical trials in breast cancer is provided, along with collaborations. Summaries are also given for GALE-301 and GALE-401 clinical programs, corporate details, leadership, and motivation.
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead program, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis (SPMS). If successful, Tcelna has the potential to address an unmet medical need as there is currently only one approved drug for SPMS that is not suitable for chronic use due to side effects. Opexa has an agreement with Merck Serono to develop Tcelna for MS indications worldwide, excluding Japan. Topline results from the Phase 2b SPMS trial are expected in late 2016.
Analysis of DNA methylation and Gene expression to predict childhood obesitysahirbhatnagar
Recent advances in genomic technologies have made it feasible to measure, on the same individual, multiple types of genomic activity such as genotypes, gene expression, DNA copy number, methylation and microRNA expression. However, in order to benefit from the increasing amounts of heterogeneous data and to obtain a more complete view of genomic functions, there is a great need for statistical and computationally efficient methods that allow us to combine this information in an intelligent way. Challenges with prediction models in this setting arise from the high-dimensional non-linear nature of the data, the large number of measurements compared to the few samples for whom they are collected, and the presence of complex interactions between the different types of data. Methods such as sparse regression, hierarchical clustering and principal component analysis can address any one of these challenges, but can not do so simultaneously. Kernel methods, which use matrices measuring the similarity between two individuals, offer a powerful way of simultaneously addressing these challenges without significantly increasing the computational burden. In this work, we investigate the benefits and challenges that arise from using kernel methods in the context of integrating DNA methylation, gene expression and phenotypic data in a sample of mother-child pairs from a prospective birth cohort. The goal of this study is to identify epigenetic marks observed at birth that help predict childhood obesity.
AMBS is a regenerative medicine company developing new treatments for CNS disorders and regenerative medicine. It has several programs and subsidiaries developing treatments for conditions like Parkinson's disease, burns, cancer, and retinal diseases. Its lead programs include Eltoprazine for Parkinson's disease levodopa-induced dyskinesia through its subsidiary Elto Pharma, and recombinant MANF protein for retinal conditions like retinitis pigmentosa through its subsidiary MANF Therapeutics. AMBS is currently trading at very low prices but has achieved much higher values in the past, and it believes further progress in its clinical programs could increase its valuation.
Opexa Therapeutics August 2015 OPXA Corporate PresentationOpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and key programs. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown signs of stabilizing disease progression. Tcelna works by reducing myelin reactive T-cells that damage the myelin sheath. Opexa has an option agreement with Merck Serono for the development and commercialization of Tcelna in multiple sclerosis. Additionally, Opexa is developing OPX-212 for neuromyelitis optica, an orphan indication with no approved therapies.
Opexa Therapeutics December 2015 Corporate PresentationOpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and lead programs. Their T-cell immunotherapy Tcelna is in Phase 2b clinical trials for secondary progressive multiple sclerosis. Preclinical studies of OPX-212 showed a reduction of AQP4 reactive T-cells in a murine model of neuromyelitis optica, an orphan disease with no approved therapies. Opexa secured funding to advance OPX-212 into a Phase I/II clinical trial in 2016 pending regulatory approval. Their proprietary ImmPath platform allows for personalized T-cell therapies tailored for individual patient's disease profiles.
Catasys provides a virtual, scalable, and data-driven behavioral health program called OnTrak to help address the high costs of untreated behavioral health conditions like substance abuse, depression, and anxiety. OnTrak uses predictive analytics to identify avoidant patients, engages them in a 52-week outpatient treatment program with care coaching support, and integrates medical and psychosocial care. This approach aims to reduce health plan costs by around 50% while providing full reimbursement. Catasys has signed agreements with several major health insurance companies to provide OnTrak and is seeing growing enrollment.
Genetic Technologies Limited is a diversified molecular diagnostics
company developing tools for the prediction and assessment of cancer
risk to help physicians proactively manage patient health. The
Company’s lead products, ‘GeneType for Breast Cancer’ and
‘GeneType for Colorectal Cancer’, are clinically validated risk
assessment tests that are first in their class. The Company’s
development pipeline includes new tests for COVID-19, Type 2
diabetes, cardiovascular disease, prostate cancer, and melanoma.
Listed on the ASX in 2000 and NASDAQ in 2005, Genetic
Technologies has been a leader in the development and
commercialization of genetic risk assessment technology for 20 years.
Invitae Corporation is a genetic information company focused on bringing comprehensive genetic testing into mainstream medical practice. Some key points:
- Invitae's market cap is $2.052B with revenue expected to hit $500M by 2020 growing at triple digit rates annually.
- Their goal is to become the "Amazon of genetics" by making genetic testing as easy and affordable as online shopping.
- They offer a variety of genetic testing services including carrier screening, non-invasive prenatal testing, and testing related to miscarriages and IVF.
- Invitae aims to build a global "genome network" to share genetic information and advance healthcare. They have acquired several companies to expand their
Catasys provides an integrated virtual healthcare program called OnTrak that identifies and treats behavioral health conditions like substance abuse and depression. OnTrak uses predictive analytics to identify high-cost patients with behavioral health issues who rarely seek treatment. Patients enroll in a 52-week virtual treatment program with care coaching support. Studies show OnTrak significantly reduces medical costs and healthcare utilization for enrolled members. Catasys contracts with health plans to provide OnTrak and is paid a monthly fee per enrolled member.
SciSparc is a specialty pharmaceutical company developing unique cannabinoid technologies for treatment of central nervous system disorders. The proprietary compounds capitalize on the biochemistry of receptors that specialize in modulating the central nervous system (CNS) to create therapeutics that mitigate the adverse symptoms associated with CNS disorders.
One in 8 U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. Following breast cancer surgery in the adjuvant setting, a HER2/neu 3+ patient typically receives Herceptin® in the first year, with the hope that their breast cancer will not recur, and with the odds of recurrence slowly decreasing over the first 5 years after surgery. Herceptin® has been shown to reduce recurrence rates from 25% to 12% in the adjuvant setting. In the neoadjuvant setting, a patient receives treatment before surgery and based on the results of a biopsy at surgery, will receive the same or more potent treatment after surgery. Kadcyla® has been shown to reduce recurrence rates from 22% to 11% in the neoadjuvant setting. Accordingly, we believe that GP2 may be used to address the 50% of recurring patients who do not respond to either Herceptin® or Kadcyla®.
This document provides a summary of key information from a Glaukos Corporation presentation:
- Glaukos is transforming glaucoma therapy through micro-scale injectable therapies like iStent that restore and enhance the eye's natural drainage system.
- Clinical trials show iStent used with cataract surgery significantly lowers intraocular pressure up to 3 years while reducing medication needs by over 80%.
- US retrospective studies of over 100 eyes found iStent with cataract surgery lowered pressure 22% and medications 56% at 2 years.
- An international study demonstrated iStent's pressure lowering ability increases with 1, 2, or 3 stents implanted as a standalone procedure.
This corporate presentation from Target: infinite Hope outlines their lead product candidate MDNA55 for the treatment of cancers with Interleukin-4 Receptor (IL4R) biomarker expression. MDNA55 has shown promising clinical efficacy in recurrent glioblastoma patients in Phase 1/2 trials with an objective response rate of 34%. The presentation highlights the significant market opportunity for MDNA55 in brain cancers, which affect over 133,000 patients annually. Target: infinite Hope is pursuing accelerated approval for MDNA55 in recurrent glioblastoma in 2018 based on its Phase 2b trial and end of Phase 2 meeting with the FDA. The company has a pipeline of next-generation IL4-empowered cytokines and $14.
This corporate presentation discusses Cidara's antifungal drug rezafungin and antiviral drug development platform Cloudbreak. For rezafungin, positive Phase 3 data was reported showing non-inferiority to caspofungin for the primary endpoints of all-cause mortality and global cure. Cidara plans to submit an NDA for rezafungin in mid-2022. The Cloudbreak platform is progressing a Phase 1 trial for influenza starting early 2022 with expansion to other viruses and oncology planned. Cidara's pipeline targets multiple unmet medical needs in infectious disease and oncology.
Greenwich LifeSciences (the “Company”) is a clinical-stage biopharmaceutical company focused on the development of GP2, an immunotherapy to prevent breast cancer recurrences in patients who have previously undergone surgery. GP2 is a 9 amino acid transmembrane peptide of the HER2/neu (human epidermal growth factor receptor 2) protein, a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or “over-expressor”) levels.
How we Built a Large Scale Matched Pair Analysis Engine (MCPairs) using OpenE...Al Dossetter
MCPairs performed Matched Molecular Pair Analysis on large scale to build databases of exploitable knowledge which is accessible for Drug Discovery to accelerate research projects. The talk describes how we did this and some of the challenges.
MedChemica BigData What Is That All About?Al Dossetter
A light look at the world of BigData for the lay person - a look at a couple of examples and what we do in MedChemica to speed up drug discovery. First presented at Macclesfield SciBar, and then Knutsford SciBar.
MedChemica Active Learning - Combining MMPA and MLAl Dossetter
Describes MedChemica research on combining Matched Molecular Pair Analysis (MMPA) and Machine Learning (ML) into a closed loop to find and optimize new hits for drug discovery. The talks describes the MMPA and Regression Forest models and how they were combined and some early conclusion. Of these permutative MMPA is the clear winner (Free Wilson ++)
Accelerating multiple medicinal chemistry projects using Artificial Intellige...Al Dossetter
The technical methods and results of Matched Molecular Pair Analysis (MMPA) applied from a small, individual assay scale through large pharma scale, to multiple pharma data sharing scale have been published and reviewed. The drive behind these efforts has been to derive a medicinal chemistry knowledge base (i.e. definitive textbook) that can be applied to drug discovery projects. The aim is to greatly decrease the time in lead identification and optimization by the synthesis of fewer compounds. Such a system suggests compound designs to expert chemists to triage; such a process is Artificial Intelligence (AI). Given this context, how does this work on projects? How do the chemists make decisions? What are the results? The talk will answer these questions through project examples where MMPA has been applied and how this led to drug candidates. The projects disclosed are from multiple organisations and describe Cathepsin K inhibitors, Glucokinase Inhibitors, 11β-Hydroxysteroid Dehydrogenase Type I Inhibitors (11β-HSD1), Ghrelin inverse antagonists and Tubulin Polymerization inhibitors. An overview of MMPA will be presented and each project will be briefly described with a focus on how the chemists used MMPA to understand SAR and design compounds. The impact of project progress to CD will be quantified.
Immuron Limited is a clinical stage biopharmaceutical company developing oral immunotherapies to treat inflammatory and infectious diseases. It has two lead clinical assets in Phase 2 development for non-alcoholic steatohepatitis (NASH) and Clostridium difficile infection. Immuron's lead candidate, IMM-124E, is being studied in three Phase 2 clinical trials for NASH, alcoholic steatohepatitis, and pediatric non-alcoholic fatty liver disease, with data expected in late 2017 and 2018. IMM-124E works through broad anti-inflammatory effects and has demonstrated a strong anti-fibrotic effect in preclinical models of liver disease.
The talk describes the science and results of a consortium of multiple pharmaceutical companies extracting medicinal chemistry knowledge from research data and the application to real drug design projects. A new technique for automating pharmacophore / toxophore finding from public data is disclosed.
The document summarizes the 2016 annual meeting of stockholders held on July 14, 2016. It includes a forward-looking statement noting risks and uncertainties in projections. The development pipeline outlines several investigational products in various phases of clinical trials for breast cancer, gastric cancer, gynecological cancers, and hematological conditions. An overview of the current status of NeuVax clinical trials in breast cancer is provided, along with collaborations. Summaries are also given for GALE-301 and GALE-401 clinical programs, corporate details, leadership, and motivation.
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead program, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis (SPMS). If successful, Tcelna has the potential to address an unmet medical need as there is currently only one approved drug for SPMS that is not suitable for chronic use due to side effects. Opexa has an agreement with Merck Serono to develop Tcelna for MS indications worldwide, excluding Japan. Topline results from the Phase 2b SPMS trial are expected in late 2016.
Analysis of DNA methylation and Gene expression to predict childhood obesitysahirbhatnagar
Recent advances in genomic technologies have made it feasible to measure, on the same individual, multiple types of genomic activity such as genotypes, gene expression, DNA copy number, methylation and microRNA expression. However, in order to benefit from the increasing amounts of heterogeneous data and to obtain a more complete view of genomic functions, there is a great need for statistical and computationally efficient methods that allow us to combine this information in an intelligent way. Challenges with prediction models in this setting arise from the high-dimensional non-linear nature of the data, the large number of measurements compared to the few samples for whom they are collected, and the presence of complex interactions between the different types of data. Methods such as sparse regression, hierarchical clustering and principal component analysis can address any one of these challenges, but can not do so simultaneously. Kernel methods, which use matrices measuring the similarity between two individuals, offer a powerful way of simultaneously addressing these challenges without significantly increasing the computational burden. In this work, we investigate the benefits and challenges that arise from using kernel methods in the context of integrating DNA methylation, gene expression and phenotypic data in a sample of mother-child pairs from a prospective birth cohort. The goal of this study is to identify epigenetic marks observed at birth that help predict childhood obesity.
AMBS is a regenerative medicine company developing new treatments for CNS disorders and regenerative medicine. It has several programs and subsidiaries developing treatments for conditions like Parkinson's disease, burns, cancer, and retinal diseases. Its lead programs include Eltoprazine for Parkinson's disease levodopa-induced dyskinesia through its subsidiary Elto Pharma, and recombinant MANF protein for retinal conditions like retinitis pigmentosa through its subsidiary MANF Therapeutics. AMBS is currently trading at very low prices but has achieved much higher values in the past, and it believes further progress in its clinical programs could increase its valuation.
Opexa Therapeutics August 2015 OPXA Corporate PresentationOpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and key programs. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown signs of stabilizing disease progression. Tcelna works by reducing myelin reactive T-cells that damage the myelin sheath. Opexa has an option agreement with Merck Serono for the development and commercialization of Tcelna in multiple sclerosis. Additionally, Opexa is developing OPX-212 for neuromyelitis optica, an orphan indication with no approved therapies.
Opexa Therapeutics December 2015 Corporate PresentationOpexaTherapeutics
Opexa Therapeutics presented information on their precision immunotherapy platform and lead programs. Their T-cell immunotherapy Tcelna is in Phase 2b clinical trials for secondary progressive multiple sclerosis. Preclinical studies of OPX-212 showed a reduction of AQP4 reactive T-cells in a murine model of neuromyelitis optica, an orphan disease with no approved therapies. Opexa secured funding to advance OPX-212 into a Phase I/II clinical trial in 2016 pending regulatory approval. Their proprietary ImmPath platform allows for personalized T-cell therapies tailored for individual patient's disease profiles.
Catasys provides a virtual, scalable, and data-driven behavioral health program called OnTrak to help address the high costs of untreated behavioral health conditions like substance abuse, depression, and anxiety. OnTrak uses predictive analytics to identify avoidant patients, engages them in a 52-week outpatient treatment program with care coaching support, and integrates medical and psychosocial care. This approach aims to reduce health plan costs by around 50% while providing full reimbursement. Catasys has signed agreements with several major health insurance companies to provide OnTrak and is seeing growing enrollment.
Genetic Technologies Limited is a diversified molecular diagnostics
company developing tools for the prediction and assessment of cancer
risk to help physicians proactively manage patient health. The
Company’s lead products, ‘GeneType for Breast Cancer’ and
‘GeneType for Colorectal Cancer’, are clinically validated risk
assessment tests that are first in their class. The Company’s
development pipeline includes new tests for COVID-19, Type 2
diabetes, cardiovascular disease, prostate cancer, and melanoma.
Listed on the ASX in 2000 and NASDAQ in 2005, Genetic
Technologies has been a leader in the development and
commercialization of genetic risk assessment technology for 20 years.
Invitae Corporation is a genetic information company focused on bringing comprehensive genetic testing into mainstream medical practice. Some key points:
- Invitae's market cap is $2.052B with revenue expected to hit $500M by 2020 growing at triple digit rates annually.
- Their goal is to become the "Amazon of genetics" by making genetic testing as easy and affordable as online shopping.
- They offer a variety of genetic testing services including carrier screening, non-invasive prenatal testing, and testing related to miscarriages and IVF.
- Invitae aims to build a global "genome network" to share genetic information and advance healthcare. They have acquired several companies to expand their
Catasys provides an integrated virtual healthcare program called OnTrak that identifies and treats behavioral health conditions like substance abuse and depression. OnTrak uses predictive analytics to identify high-cost patients with behavioral health issues who rarely seek treatment. Patients enroll in a 52-week virtual treatment program with care coaching support. Studies show OnTrak significantly reduces medical costs and healthcare utilization for enrolled members. Catasys contracts with health plans to provide OnTrak and is paid a monthly fee per enrolled member.
SciSparc is a specialty pharmaceutical company developing unique cannabinoid technologies for treatment of central nervous system disorders. The proprietary compounds capitalize on the biochemistry of receptors that specialize in modulating the central nervous system (CNS) to create therapeutics that mitigate the adverse symptoms associated with CNS disorders.
One in 8 U.S. women will develop invasive breast cancer over her lifetime, with approximately 266,000 new breast cancer patients and 3.1 million breast cancer survivors in 2018. Following breast cancer surgery in the adjuvant setting, a HER2/neu 3+ patient typically receives Herceptin® in the first year, with the hope that their breast cancer will not recur, and with the odds of recurrence slowly decreasing over the first 5 years after surgery. Herceptin® has been shown to reduce recurrence rates from 25% to 12% in the adjuvant setting. In the neoadjuvant setting, a patient receives treatment before surgery and based on the results of a biopsy at surgery, will receive the same or more potent treatment after surgery. Kadcyla® has been shown to reduce recurrence rates from 22% to 11% in the neoadjuvant setting. Accordingly, we believe that GP2 may be used to address the 50% of recurring patients who do not respond to either Herceptin® or Kadcyla®.
This document provides a summary of key information from a Glaukos Corporation presentation:
- Glaukos is transforming glaucoma therapy through micro-scale injectable therapies like iStent that restore and enhance the eye's natural drainage system.
- Clinical trials show iStent used with cataract surgery significantly lowers intraocular pressure up to 3 years while reducing medication needs by over 80%.
- US retrospective studies of over 100 eyes found iStent with cataract surgery lowered pressure 22% and medications 56% at 2 years.
- An international study demonstrated iStent's pressure lowering ability increases with 1, 2, or 3 stents implanted as a standalone procedure.
This corporate presentation from Target: infinite Hope outlines their lead product candidate MDNA55 for the treatment of cancers with Interleukin-4 Receptor (IL4R) biomarker expression. MDNA55 has shown promising clinical efficacy in recurrent glioblastoma patients in Phase 1/2 trials with an objective response rate of 34%. The presentation highlights the significant market opportunity for MDNA55 in brain cancers, which affect over 133,000 patients annually. Target: infinite Hope is pursuing accelerated approval for MDNA55 in recurrent glioblastoma in 2018 based on its Phase 2b trial and end of Phase 2 meeting with the FDA. The company has a pipeline of next-generation IL4-empowered cytokines and $14.
This corporate presentation discusses Cidara's antifungal drug rezafungin and antiviral drug development platform Cloudbreak. For rezafungin, positive Phase 3 data was reported showing non-inferiority to caspofungin for the primary endpoints of all-cause mortality and global cure. Cidara plans to submit an NDA for rezafungin in mid-2022. The Cloudbreak platform is progressing a Phase 1 trial for influenza starting early 2022 with expansion to other viruses and oncology planned. Cidara's pipeline targets multiple unmet medical needs in infectious disease and oncology.
Cidara is developing long-acting therapeutics designed to improve the standard of care for patients facing serious diseases. The Company’s portfolio is comprised of drug candidates intended to transform existing treatment and prevention paradigms. Its lead Phase 3 antifungal candidate, rezafungin, will report Phase 3 data at the end of 2021. The potential peak sales opportunity for rezafungin in the US is ~$750M. In addition, the Company is developing Drug-Fc Conjugates (DFCs) targeting viral and oncology diseases from Cidara’s proprietary Cloudbreak® platform.
This document provides a summary of Galena Biopharma's Q3 2016 financial results and corporate update. It discusses interim results from the Phase 3 PRESENT trial of NeuVax showing a potential delay in disease-free survival and highlights the risk of pseudoprogression in cancer immunotherapy trials. It also reviews the company's immunotherapy development pipeline, including NeuVax programs in breast and gastric cancer and GALE-301/302 programs in ovarian and breast cancer. Finally, it discusses the company's cash position and expected milestones for the remainder of 2016.
Cidara is developing long-acting therapeutics designed to improve the standard of care for patients facing serious diseases. The Company’s portfolio is comprised of drug candidates intended to transform existing treatment and prevention paradigms. Its lead Phase 3 antifungal candidate, rezafungin, will report Phase 3 data at the end of 2021. The potential peak sales opportunity for rezafungin in the US is ~$750M. In addition, the Company is developing Drug-Fc Conjugates (DFCs) targeting viral and oncology diseases from Cidara’s proprietary Cloudbreak® platform.
This presentation provides an overview of Interpace Diagnostics Group (IDXG), a commercial company that provides molecular diagnostic tests and pathology services for cancer evaluation. IDXG operates two CLIA-certified labs and has four proprietary molecular diagnostic tests for pancreatic cysts and thyroid nodules that assess cancer risk. The tests have high margins and barriers to entry due to reimbursement and complexity. Recent accomplishments include raising funds, improving financials, expanding insurance coverage and launching international distribution. The molecular diagnostic market is large and growing due to advantages over drug development. IDXG's tests establish new standards in cancer risk assessment for pancreatic cysts and thyroid nodules compared to current guidelines.
This presentation provides an overview of Interpace Diagnostics Group (IDXG), a commercial company that provides molecular diagnostic tests and pathology services. Key points:
- IDXG has proprietary molecular diagnostic tests for pancreatic cysts (PancraGEN), thyroid nodules (ThyGenX/ThyraMIR), and Barrett's esophagus (BarreGEN).
- Clinical studies show PancraGEN more accurately determines cancer risk of pancreatic cysts compared to current guidelines. ThyGenX/ThyraMIR combination testing can accurately rule in or rule out thyroid cancer risk.
- The tests have significant market opportunities and address unmet clinical needs to avoid unnecessary surgeries and
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date. Piclidenoson received approval for COVID-19 clinical trial in Israel in April 2020 and is expected to file its IND in the US in the near-term.
Improving Management of Non-Metastatic Castration-Resistant Prostate Cancer (...Carevive
This document summarizes an educational initiative on improving management of non-metastatic castrate resistant prostate cancer (nmCRPC). The initiative consisted of presentations on evolving evidence-based treatment paradigms for nmCRPC and supportive care for men with CRPC. Key studies discussed included the SPARTAN and PROSPER trials which found that the addition of apalutamide or enzalutamide to androgen deprivation therapy significantly prolonged metastasis-free survival in men with nmCRPC. The document reviewed results, adverse events, and implications for sequencing of these new standard treatments in nmCRPC.
Glaukos investor presentation for q2 2017 for website and 8_kglaukos
This document discusses Glaukos Corporation and its glaucoma treatment technologies. It contains the following key points:
- Glaukos is transforming glaucoma therapy with micro-scale injectable treatments that restore eye's natural drainage and reduce intraocular pressure, the only proven treatment for glaucoma.
- Their flagship product, iStent, is a single-use micro-bypass stent that has demonstrated long-term IOP reduction and medication reduction when used with cataract surgery based on multiple clinical studies.
- New products in development include iStent Inject for combination cataract/glaucoma procedures, iStent Supra to access a secondary drainage pathway, and iDose for targeted
- Frexalimab demonstrated significantly reduced disease activity in relapsing multiple sclerosis in a phase 2 trial, meeting its primary endpoint with an 89% reduction in new gadolinium-enhancing lesions. It was well tolerated across all dose arms. Pivotal trials are planned to start in 2024.
- Amlitelimab showed statistically significant improvements in atopic dermatitis symptoms compared to placebo in a phase 2b trial. Biomarker results support effects on both type 2 and non-type 2 inflammation pathways. It was well tolerated. Pivotal trials are planned for 2024.
- A phase 1b trial of SAR443765, an IL-13/TSL
- Sanofi reported Q2 2023 results with total sales up 3.3% driven by double-digit growth of Specialty Care led by Dupixent and strong growth in Vaccines, while CHC was impacted by a Q1 inventory build.
- Specialty Care sales increased 11.8% led by 34.2% growth of Dupixent which saw demand-driven growth across its five approved indications. Rare Diseases grew 9.6% mainly due to patient accruals in Fabry and Pompe franchises and the strong launch of Nexviazyme. Neurology, Oncology and Rheumatology declined 35.1% due to LOE impact on Aubagio.
-
1) Marker Therapeutics is developing a multi-tumor associated antigen (MultiTAA) T cell therapy approach that targets multiple tumor antigens to address limitations of current CAR T and TCR therapies such as relapse due to antigen escape.
2) In clinical trials to date involving over 50 lymphoma patients, MultiTAA T cell therapy has shown durable complete responses in over 50% of patients with active lymphoma with no serious adverse events or cytokine release syndrome observed.
3) For patients treated while in remission, over 80% have maintained a continued complete response for durations ranging from 3 to 37 months to date.
Glaukos Corporation is an ophthalmic medical technology company transforming glaucoma treatment. Their minimally-invasive micro-scale injectable therapies, including the iStent, provide more effective treatment options compared to eye drops and traditional surgeries. Clinical trials demonstrate the iStent used with cataract surgery significantly lowers intraocular pressure up to 3 years post-op while reducing medication use. Additional studies show promise for combination treatments and sustained efficacy of micro-scale injectable therapies.
Targovax Next generation immune activators for solid tumorsRoarFredriksen1
Targovax (OSE:TRVX) is a clinical stage immuno-oncology company developing immune activators to target hard-to-treat solid tumors. Targovax’s focus is to activate the patient’s immune system to fight cancer, and to bring benefit to cancer patients with few available treatment alternatives. Targovax is developing its product candidates in different cancer indications, including melanoma, mesothelioma, and multiple myeloma, and has demonstrated a favorable safety and tolerability profile.
Targovax’s lead clinical candidate, ONCOS-102, is a genetically modified oncolytic adenovirus, which has been engineered to selectively infect cancer cells and activate the immune system against the tumor. Following very encouraging clinical data in several indications, both as monotherapy and in combinations, ONCOS-102 is progressing into a randomized phase 2 trial in melanoma patients resistant to PD-1 checkpoint inhibitor treatment.
Building on successful clinical studies which have provided deep mechanistic insights into the tumor biology and the human immune systems, Targovax is researching circular RNA (circRNA) as novel cancer medicines. In addition, Targovax has a KRAS immunotherapy program, with lead cancer vaccine candidate, TG01, expected to enter the clinic in an enhanced format in the second half of 2022. Together this provides Targovax with a rich pipeline of innovative future immunotherapy product candidates to follow ONCOS-102.
Can-Fite BioPharma Ltd. (NYSE American: CANF) is an advanced clinical stage drug development company with a platform technology that addresses multi-billion-dollar markets in the treatment of autoimmune inflammatory diseases including Psoriasis, and liver diseases including advanced liver cancer and NASH. Can-Fite’s drugs have an excellent safety profile with experience in over 1,000 patients. Can-Fite’s intellectual property portfolio consists of 13 patent families issued and pending. Piclidenoson and Namodenoson have been out-licensed in select territories with approximately $18 million received to date. Piclidenoson received approval for COVID-19 clinical trial in Israel in April 2020 and is expected to file its IND in the US in the near-term.
- IDXG provides molecular diagnostic tests for cancer risk assessment and prognosis.
- Recent accomplishments include new product launches, reimbursement from Aetna for ThyraMir, and achieving approval in New York State.
- The presentation provides financial information, with revenue growing but losses continuing from investments in sales, R&D and administrative expenses.
- Olivier Brandicourt, CEO of Sanofi, presented at the JP Morgan Healthcare Conference in San Francisco on January 9, 2018.
- The presentation outlined Sanofi's progress on its 2020 strategic roadmap and focused on building leadership in specialty care and strengthening its diabetes portfolio.
- Sanofi is sustaining innovation in R&D to support long-term growth through advancing its pipeline of over 70 projects, with 9 potential regulatory submissions expected in the next 18 months.
Idxg Corporate Presentation Global Online Growth Conference - Oct 2016RedChip Companies, Inc.
The corporate presentation discusses IDXG's molecular diagnostic tests for evaluating cancer risk in pancreatic cysts, thyroid nodules, and Barrett's esophagus. It provides an overview of the company's history and key tests, the clinical need they address, clinical evidence and performance data of the tests. It also reviews reimbursement and billing details such as payor coverage, contracts, and realized revenue for the tests.
Opexa Therapeutics is developing personalized T-cell immunotherapies for autoimmune diseases. Their lead candidate, Tcelna, is in Phase 2b clinical trials for secondary progressive multiple sclerosis and has shown signs of stabilizing disease progression in previous trials. Opexa has an option agreement with Merck Serono for the development and commercialization of Tcelna in multiple sclerosis. Recent milestones include completing enrollment in the Tcelna Phase 2b trial for SPMS and receiving a $3 million payment from Merck Serono. Opexa is also developing OPX-212 for neuromyelitis optica and expects to file an IND by the end of 2015.
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2. Forward-Looking Statements
May 2018 Medicenna Corporate Overview
Certain statements in this presentation are “forward-looking statements. Any statements that express or involve discussions with respect to
predictions, expectations, beliefs, plans, projections, objectives, assumptions or future events or performance (often, but not always using words
or phrases such as “expect”, “seek”, “endeavour”, “anticipate”, “plan”, “estimate”, “believe”, “intend”, or stating that certain actions, events or results
may, could, would, might or will occur or be taken, or achieved) are not statements of historical fact and may be “forward-looking statements”.
Forward-looking statements are based on expectations, estimates and projections at the time the statements are made that involve a number of risks
and uncertainties which would cause actual results or events to differ materially from those presently anticipated. Forward-looking statements are
based on expectations, estimates and projections at the time the statements are made and involve significant known and unknown risks, uncertainties
and assumptions. A number of factors could cause actual results, performance or achievements to be materially different from any future results,
performance or achievements that may be expressed or implied by such forward-looking statements. These include, but are not limited to, the risk
factors discussed in the public filings made by Medicenna with the applicable securities commissions in Canada, including the Annual Information
Form dated June 15, 2017. Should one or more of these risks or uncertainties materialize, or should assumptions underlying the forward-looking
statements prove incorrect, actual results, performance or achievements could vary materially from those expressed or implied by the forward-looking
statements contained in this document. These factors should be considered carefully and prospective investors should not place undue reliance on
these forward-looking statements.
Although the forward-looking statements contained in this document are based upon what Medicenna currently believes to be reasonable assumptions,
Medicenna cannot assure prospective investors that actual results, performance or achievements will be consistent with these forward-looking
statements. Except as required by law, Medicenna does not have any obligation to advise any person if it becomes aware of any inaccuracy in or
omission from any forward-looking statement, nor does it intend, or assume any obligation, to update or revise these forward-looking statements to
reflect new events or circumstances.
2
3. May 2018 Medicenna Corporate Overview 3
MDNA55 — LEAD PROGRAM SUPERKINE PLATFORM CORPORATE SNAPSHOT
COMPELLING
DATA
Phase 1/2 data in
rGBM with MDNA55
ORPHAN/
FAST TRACK
Orphan Drug (FDA, EMA)
Fast Track (FDA)
IL-2; IL-4; IL-13
Tunable cytokines
13 PATENT
FAMILIES
Strong technology
platform protection
10 TOP-RANKED
CENTERS
Currently enrolling a Phase 2b rGBM
patients
4,000
Brain tumor patients can
be treated with 1 gram
of MDNA55
GROWING
PIPELINE
Oncology, autoimmune and
inflammatory
EXPERIENCED
LEADERSHIP
Biotech and
drug development
250,000
Annual incidence
of glioblastoma and
metastatic brain cancer2
2 BILLION
Potential market of
MDNA55 market for
brain cancer ($US)1,3
VALIDATED
TARGETS
Industry transactions support further
development
$18 Million
Non-dilutive financing
1. BioXcel Strategic Analysis Report, 2014.
2. Globocan 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide 2012.
3. Decision Resources, Inc Glioblastoma Report, Sept 2013.
6. MDNA55
Treatment
Direct infusion
into tumor
convection enhanced
delivery (CED)
75%
INOPERABLE rGBM
Treatment Pathway for Glioblastoma (GBM)
May 2018 Medicenna Corporate Overview 6
* Expression of the DNA repair protein O6-methylguanine-DNA methyltransferase (MGMT) is responsible for resistance to alkylating agents used in GBM treatment.
STANDARD OF CARE FOR PATIENTS WITH GBM
DIAGNOSIS
ADJUVANT CHEMOSURGERY
(85-90%) 55% of GBM
Chemo-Resistant*
RADIOTHERAPY CHEMOTHERAPY
RELAPSE
25%
OPERABLE rGBM
GBM IS UNIFORMLY FATAL – VIRTUALLY ALL TUMORS WILL RECUR (rGBM)
7. MDNA55: Targeted Dual-Action Immunotherapeutic
May 2018 Medicenna Corporate Overview 7
Tumor
Targeting Domain
Circularly Permuted
Interleukin-4 (cpIL-4)
Tumor Killing
“Cytotoxic” Domain
Catalytic domain of
Pseudomonas Exotoxin A (PE)
Proven payload efficacy - identical to
Medimmune's anti-CD22 immunotoxin,
Moxetumomab Pasudotox, met primary endpoint
for Hairy Cell Leukemia (Priority Review)
Potently toxic to tumor cells with a wide therapeutic window
Bypass the blood brain barrier through localized convection enhanced delivery (CED)
Simultaneously purges the tumor microenvironment (TME) and un-blinds the immune system to cancer cells
A POWERFUL MOLECULAR TROJAN HORSE
8. Mechanism of Action of MDNA55
May 2018 Medicenna Corporate Overview 8
ENDOCYTOSIS
FURIN PROTEASE
ADP RIBOSYLATION
NUCLEUS
Efficient intracellular
delivery of toxin payload
Inhibit Protein Synthesis
CELL DEATH
NUCLEUS
9. 72 Patients Treated to Date
9
MDNA55: Clinical Study Summary
May 2018 Medicenna Corporate Overview
Consolidated Safety Profile
• No deaths attributed to MDNA55
• No systemic toxicity at any dose
• No clinically significant laboratory abnormalities
• Most adverse events were due to local effects and
similar to those typically seen in this patient
population
• Manageable inflammation and edema associated
with tumor cell death
• MTD established at 240 μg
STUDY PATIENT DOSE(µg)
Investigator Initiated
(U.S.)
Recurrent GBM
(n=9)
6–720
Multi-Center
(U.S./Germany)
Phase 1/2
Recurrent HGG
No-Resection
(n=25 GBM+6 AA)
240–900
Multi-Center
(U.S./Germany)
Phase 1/2
Recurrent GBM
+ Resection
(n=32)
90–300
10. Open-Label Single Arm Study in 52 Patients
10
Phase 2b Study Design Summary
May 2018 Medicenna Corporate Overview
PRIMARY OBJECTIVES
ORR
SECONDARY OBJECTIVES
MOS | Safety | PFS-6
TERTIARY OBJECTIVES
Correlate IL4R expression with efficacy
DIAGNOSIS PLANNING TREATMENT FOLLOW UP
11. Statistical Design and Sample Size
11
Efficacy Analysis
May 2018 Medicenna Corporate Overview
PRIMARY ENDPOINT
Objective Response Rate (ORR) per RANO (Response Assessment for Neuro-Oncology) based criteria
TEST HYPOTHESIS
Null hypothesis that ORR is 6% (kill) versus the alternative hypothesis (pursue) that ORR is 18% following treatment
with MDNA55. Hypothesis based on ORR from previous studies1
PRIMARY ANALYSIS
Assessed according to a single-arm, single-stage binomial design at 1-sided alpha =2. A total of 52 Subjects will
provide >95% power. A total of 25 subjects from the revised protocol will provide greater than 80% power.
1 Levin VA, Tonge PJ, Gallo JM, et al. CNS Anticancer Drug Discovery and Development Conference White Paper. Neuro-Oncology, v17:1–26, 2015
Type of Treatment Number of Clinical Trials Number of Patients
Case Number of Weighted
Mean ORR (Range)
Cytotoxic Agents 21 1745 6% (0–17%)
Non-Cytotoxic/Non-Antiangioneic 18 1239 4% (0–9%)
12. 2nd Generation CED Improves Tumor Drug Coverage
May 2018 Medicenna Corporate Overview 12
PAST STUDIES
1st Generation CED
Inaccurate catheter placement
Drug leakage due to backflow
Inadequate tumor coverage
CURRENT STUDIES
HIGH FLOW CED
TUMOR
CAVITY
TUMOR
INFUSION AT 3 HOURS
TUMOR
INFUSION AT 24 HOURS
64%
GREATER TUMOR COVERAGE WITH HIGH FLOW CED
Tumor coverage using new
high flow CED
Image-guided catheter
placement
New catheters
prevent backflow
Real-time
monitoring ensures
tumor coverage
13. MDNA55 Survival Results Consistent with Previous Studies
May 2018 Medicenna Corporate Overview 13
Overall Survival:
MDNA55-05 vs. Legacy Combined Data
Overall Survival:
MDNA55-05 (1.5µg/mL vs. 3.0µg/mL)
0 100 200 300 400 500
0
50
100
Days elapsed
Percentsurvival
MDNA55-05
Legacy combined
0 100 200 300 400
0
50
100
Days elapsed
Percentsurvival
1.5 µg/mL
3.0 µg/mL
14. Tumor Response Following Progression
May 2018 Medicenna Corporate Overview 14
Baseline
11.3 cm3
Day 30
19.6 cm3
Day 60
15.2 cm3
Day 120
13.8 cm3
Day 180
11.1 cm3
Day 240
6.4 cm3
Day 300
9.6 cm3
Day 330
12.3 cm3
15. Advanced Imaging to Determine Progression or Response
May 2018 Medicenna Corporate Overview 15
Baseline Day 60 Day 90 Day 120
Necrotic Tumor Active Tumor
16. Advanced Imaging to Determine Progression or Response
May 2018 Medicenna Corporate Overview 16
17. 17
Protocol Amendments Recommended by Safety
and Clinical Advisory Committees
May 2018 Medicenna Corporate Overview
Improving the
likelihood of
success with
MDNA55 in
patients with
rGBM
SAFETY SUMMARY
Ph 1
(n=25)
Ph 2
(n=32)
Ph 2b
(n=27)
Related Serious Adverse Events 24 28 8
• Dosing up to 240 µg (MTD)
• Volume personalized based on tumor size
• Include advanced imaging and/or biopsy to discriminate between pseudo-
progression and true-progression
• Second dose of MDNA55, if appropriate
• Patients requiring surgical resection will continue on study if evidence of tumor
necrosis and no residual tumor
• Allow sub-therapeutic dose of Avastin if intolerant to steroids
18. >2000 Patient Biopsies Analyzed Consistently Show IL4R Over-Expression
18
Future Opportunity: 1 Million IL4R Cancers Annually
May 2018 Medicenna Corporate Overview
Glioblastoma
76%
Bladder
73%
Breast
82%
Colorectal
89%
Head and Neck
75%
NSCLC
79%
Mesothelioma
96%
Ovarian
60%
Pancreatic
60%
20. 20
Extensive Library of Tunable Superkines
May 2018 Medicenna Corporate Overview
IL-2, IL-4 and IL-13 Superkines were engineered by directed evolution to have unique properties
Super-agonist or super-antagonist
SELECTION
22. Product Concept: Improved Version of Proleukin® (Aldesleukin)
22
Rationale for a IL-2 Superkine Agonist (MDNA109)
May 2018 Medicenna Corporate Overview
• Proleukin first targeted immunotherapy — approved for metastatic
melanoma and renal cancer
• Effector T cells and NK cells are relatively insensitive to IL-2 due to low
expression of CD25
• High dose IL-2 is required to stimulate effector cells
• CD25 expression on endothelium and Tregs limits use of IL-2 due to:
• Vascular leak syndrome
• Pulmonary edema
• Stimulation of Tregs blunts anti-tumor response
• IL-2 Superkine (MDNA109) signal independently of CD25 thereby:
• Preferentially activating effector T cells while limiting stimulation of Tregs
• Derive therapeutic benefits in the clinic without the underlying toxicity
Atkins et. al, Cancer J Sci Am., 2000
ORR = 16%; 6% CR, 10% PR
Survival of Melanoma
Patients Treated with IL-2
23. A High Potency Agonist for CD122: Preferentially Activates Effector Cells
23
MDNA109: A Best-in-Class IL-2 Cytokine for Cancer Immunotherapy
May 2018 Medicenna Corporate Overview
Comparison of the Stimulatory Effects on Key Immune Cell Populations
Proliferation
Immunosuppressive
Regulatory
T Cell
CD25
CD122 IL-2Rɣc
Proliferation
Immuno-stimulatory
Naïve T cell
CD8 Memory
T cell
NK cell
CD122 IL-2Rɣc
Proleukin® +++ +
ALKS 4230 + +
NKTR-214 + +
MDNA109 + +++
24. MDNA109 is More Potent and Less Toxic than Wild-type IL-2
May 2018 Medicenna Corporate Overview 24
Dose-Response of IL-2 Superkines
on CD25-YT Cells
Levin, Bates, and Ring et. al, Nature, 2012
Log[IL-2 (ng/mL)]
MDNA109 MDNA109
Selective Expansion of
CD8+ T Cells in Vivo
Reduced Adverse Effects
in Vivo
MDNA109
25. 5 10 15 20 25 30 35 40 45
0
500
1000
1500
2000
Days Post-Implant
MeanTumorVolume(mm3)
PBS
anti-PD-1
MDNA109 (5 ug q.d.)
MDNA109 (25 ug q.d.)
anti-PD-1 + MDNA109 (5 ug q.d.)
anti-PD-1 + MDNA109 (25 ug q.d.)
9/10 cures
1/10 cures
0/10 cures
MC38 colon cancer:
* 10 mg/kg IV q4dx3
25
MDNA109 Synergizes with
Anti-PD-1 Immunotherapy
May 2018 Medicenna Corporate Overview
Combination Therapy Produces Robust Responses
in a Dose-Dependent Fashion
• MDNA109 and anti-PD-1*
produce limited efficacy
alone
• Combination treatment
sufficient to cure most
mice
• Increased efficacy of
combination was well-
tolerated
27. Medicenna’s Engineered Superkines — Unique Differentiated Profile
27
Industry Leading Validation of Pre-Clinical Pipeline
May 2018 Medicenna Corporate Overview
SUPERKINE PROPERTIES COMPARATORS
MDNA209
IL-2 Super-Antagonist;
Blocks IL-2 signalling and
NK cell activity
• Broad and potent inhibition of IL2R expressing
cells compared to DEL106 and Daclizumab
• Efficient internalization enables payload fusion
or conjugation
DEL106 (IND stage)
$300 M upfront ($775 M total)
Acquired by Celgene (Jan 2017)
Daclizumab (marketed)
MDNA413
IL4/13 Dual Super-
Antagonist; Blocks Type 2
IL-4 and IL-13 signalling
• Unique MOA focused on modulating Th2
effector cells through Type II IL4/IL13 receptor
compared to Dupilumab and PRS-060
• Amenable to unique aerosol formulations like
PRS-060
Dupilumab (marketed)
PRS-060 (IND stage)
$2.1B partnership with AstraZeneca
(May 2017)
MDNA132
IL-13 Superkine;
Selective for IL13Ra2
• Higher selectivity and affinity than IL-13 mutant
used as targeting moiety of MB-101
• Proprietary fully human toxin fusion enables
efficient tumor killing with low predicted toxicity
MB-101 (Phase I in GBM)
$94.5 M private placement (Feb 2017)
29. 29
Financial Snapshot
May 2018 Medicenna Corporate Overview
• Cash balance at December 31, 2017: CDN$6.4 million
• Available to be drawn under CPRIT grant: US$6.5 million
• Expected cash burn: CDN$1 million per month
• Lead program fully funded: CDN$14.8 million available at December 31, 2017
Issued and Outstanding
24,344,048
Fully Diluted*
29,595,296
* Fully diluted includes 3,294,105 warrants with a CND$2.00 exercise price and 1,957,143 stock options with a weighted average exercise price of CDN$2.00
TSX: MDNA
OTCQX: MDNAF
30. Seasoned Management and Experienced Board
May 2018 Medicenna Corporate Overview
Management Team
Fahar Merchant, PhD Chairman, President & CEO
Former CEO Sophiris Bio (TSX); Former Director, President & CTO at KS Biomedix
(LSE); Founder, President & CEO of Avicenna Medica and IntelliGene Expressions
Martin Bexon, MD Head of Clinical Development
Former Medical Director at CSL Behring; Medical Director at Hoffman La Roche (UK and
Switzerland)
Shafique Fidai, PhD Head of Corp Development
Former VP of Business Development at Sophiris Bio; Formerly with Xenon Pharma,
Chromos
Elizabeth Williams, CPA,CA Chief Financial Officer
Former VP Finance & Admin and interim CFO at Aptose (TSX and Nasdaq); Previously
with Ernst & Young
Nina Merchant, MESc. Chief Development Officer
Former SVP Development at Sophiris Bio; Formerly VP Development at KS Biomedix
(LSE); Previously at Avicenna Medica, IntelliGene, Pharmacia and Sanofi Pasteur
Board of Directors
Fahar Merchant, PhD Chairman, President & CEO
Albert Beraldo, CPA, CA Independent Director
Founder, President and CEO of Alveda Pharmaceuticals until its acquisition by Teligent, Inc.
(NASDAQ: TLGT); Former President and CEO of Bioniche (TSX).
William W. Li, M.D. Independent Director
CEO, President and Co-Founder of the Angiogenesis Foundation. Executive strategic
consultant to pharma in drug development and major investment banks. Director of Leap
Therapeutics (NASDAQ)
Chandra Panchal, PhD Independent Director
Founder, Chairman and CEO of Axcelon; Former Co-Founder, President and CEO of Procyon
Biopharma Inc. (TSX); Former Senior Executive VP of Business Development at Ambrilia
Biopharma Inc. (TSX).
Andrew Strong, JD Independent Director
Partner at Pillsbury Winthrop Shaw Pittman — leading the Life Sciences Team in Houston,
TX. Formerly CEO of Kalon Biotherapeutics. Director of Ashford Hospitality Prime (NYSE)
Nina Merchant, M.E.Sc Director, Chief Development Officer
30
31. World Class Advisors and Collaborators
May 2018 Medicenna Corporate Overview 31
Clinical and Scientific Advisors Collaborators and Inventors
John Sampson, MD, PhD, MBA
Duke University
Principal Investigator and Expert in Drug Delivery to the Brain
Sam Denmeade, MD
John Hopkins University
Professor of Oncology: Targeted therapies for cancer
Nicholas Butowski, MD
University of California San Francisco
Principal Investigator; Novel therapies for brain cancer
Guido Kroemer, MD, PhD
University of Paris
Chair: SAB and Expert in Cancer Immunotherapy
Ralph Smalling, MSc
Regulatory Advisor
Former VP Regulatory Affairs at Amgen; Filed 40 INDs; 5 NDAs
Raj Puri, MD
USFDA
Director at CBER
Inventor of MDNA55
Aaron Ring, MD, PhD
Yale University
Asst. Prof Immunobiology & Cancer Biology
Co-Inventor of IL-2 Superkines
Chris Garcia, PhD
Stanford University
Co-Inventor of IL-2, IL-4 and IL-13 Superkines
Haya Loberboum Galski, PhD
Hebrew University of Jerusalem
Inventor of Fully Human Payloads)
32. 32
Multiple Near-Term Value Inflection Milestones
May 2018 Medicenna Corporate Overview
MDNA55
• Complete enrollment in Phase 2b rGBM trial
• Report rGBM Phase 2b interim top-line results
• End of Phase 2 meeting with FDA
• Commence Phase 2a clinical trial in other types of brain cancer
• Phase 2 clinical results for rGBM
MDNA109
• Complete manufacture of GLP API and formulation of drug product
• Complete dose range finding studies for pre-IND meeting with FDA
• Commence IND enabling studies in preparation for Phase 1 clinical trial
Phase 2 clinical
results for MDNA55
in rGBM
MDNA109 to be IND
Ready
33. May 2018 Medicenna Corporate Overview 33
MDNA55 — LEAD PROGRAM SUPERKINE PLATFORM CORPORATE SNAPSHOT
1 MILLION
Annual incidence of
IL4R positive cancers1
20
Number of cancers
known to over-express
the IL4R1
INTERLEUKIN
2,4,13
Tunable cytokines
13 PATENT
FAMILIES
Strong technology
platform protection
250,000
Annual incidence
of glioblastoma and
metastatic brain cancer2
ORPHAN/
FAST TRACK
Orphan Drug (FDA, EMA)
Fast Track (FDA)
GROWING
PIPELINE
Oncology, autoimmune and
inflammatory
EXPERIENCED
LEADERSHIP
Biotech and
drug development
4,000
Brain tumor patients can
be treated with 1 gram
of MDNA55
2 BILLION
Potential market of
MDNA55 market for
brain cancer ($US)1,3
VALIDATED
TARGETS
Industry transactions support further
development
$18 Million
Non-dilutive financing
1. BioXcel Strategic Analysis Report, 2014.
2. Globocan 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide 2012.
3. Decision Resources, Inc Glioblastoma Report, Sept 2013.
34. Thank You!
Fahar Merchant
President and Chief Executive Officer
fmerchant@medicenna.com
medicenna.com
Liz Willaims
Chief Financial Officer
ewilliams@medicenna.com