A Drug Repositioning Approach for Parkinson's Disease Using Patient Derived Cells
In the talk Dr. Mortiboys will outline a drug repositioning screening approach for Parkinson's Disease, starting with and validating in various patient derived cellular systems. This will include one example of ursodeoxycholic acid (UDCA) which is now in clinical trials for Parkinson's Disease, followed by more recent repositioning screens highlighting multiple compound classes with different novel mechanisms.
Heather Mortiboys, Ph.D., Senior Lecturer/Associate Professor, University of Sheffield
Rethinking Translational Drug Discovery and Repurposing Through the Use of Small Model Organisms
Small animal models have been extensively used to better understand basic and fundamental aspects of human biology. Now, they are proving to be valuable tools in our understanding of the genetics, and the underlying causes, of human disorders and in the efforts to accelerate drug development for them. Their small size and ease of manipulation have shown that small animal models can be in vivo models of choice to catalyze preclinical drug discovery efforts, and drive therapies into a clinical setting.
James Doyle, Ph.D., Co-Founder and CEO, Modelis
Repositioning in the Time of Covid-19: Mapping the Opportunities
Long a tool in pharma development, repositioning is enjoying focused interest in Covid-19 times. What are lessons learned from Covid-19 repositioning? What is the opportunity map for the field going forward? Specific examples and technology approaches will be discussed.
Aris Persidis, Ph.D., President, Biovista
COVID-19 Brought Worldwide Attention to Repurposing - Will VCs and Pharma Rethink the Value of the 505(b)(2) Pathway?
Federal funding for COVID-19 treatments required product availability within six months for testing in human clinical trials. Given this rapid timeline, repurposing came to the forefront of all research efforts. Big pharma rapidly embraced various approaches for assessing opportunities within their portfolio of approved products and initiated studies with COVID-19 patients in record time. Without accepting repurposing as a development strategy, pharma would not have been able to introduce any treatments for the pandemic.
John Seman, Chief Executive Officer, Revitale Pharma
Drug Repurposing Roadmap That Optimizes Patient Impact Through Collaboration
The Cure Drug Repurposing Collaboratory (CDRC) is a US FDA funded public-private partnership initiated in June 2020 and led by the non-profit Critical Path Institute (C-Path). The Collaboratory brings together a diverse group of global stakeholders to advance drug repurposing by systematically capturing real-world data from off-label drug usage to inform future clinical trials for diseases of high unmet medical need. The ultimate goal is to provide an open and transparent roadmap that supports regulatory submissions to expand drug labels for diseases with limited or no treatment options.
Marco Schito, Ph.D., Executive Director, CURE Drug Repurposing Collaboratory (CDRC), Critical Path Institute
11:45 am Artificial Intelligence Based Drug Repurposing for Better Therapeutic Effect
In this presentation, Dr. Sharma will discuss the traditional approach and demerits of AI-based drug repurposing. He will then cover Innoplexus' AI based approach for Drug Repurposing. He will go over relevant Data and NLP based approaches for data extraction for ML/DL as well as graph theory for drug repurposing. Finally he will outline an important drug repurposing case study of a BTK inhibitor.
OM Sharma, Ph.D., Vice President, Bioinformatics, Innoplexus
1:05 pm The Benefits of Collaborative Business Efforts on 505(b)(2) Repurposing
The pandemic has demonstrated how important repurposed products can be in producing rapid action and innovation to meet market needs. However, sadly many repurposed products never gain the support needed to make it to market. As we move into the next stage, how can we use collaboration to expand the number of repurposed and repositioned products that make it to market? In this session Mr. Casey will discuss achieving success through meaningful collaborations. Learn how to create more innovation, efficient processes, increased success, and improve communication by:
♦ Building partnerships with competitors, other businesses, non-profits and government regulators to move your repurposing business forward
♦ Using collaboration to amplify your voice
♦ Collaborating to create rapid action and innovation in days and weeks instead of months
♦ Using collaboration to problem solve
Stephen Casey, Founder & CEO, The 505(b)(2) Platform
1:40 pm Creating Impact in Minority and Underserved Communities Through Repurposing
The value of repurposing is more evident today than ever before, not just in finding possible treatments for the COVID-19 pandemic, but also its ability to serve a broader purpose. Currently, there is increased attention on both the health disparities in communities of color as well as a racial gap in the funding of minority researchers in the US. Cures Within Reach’s Diversity, Equity and Inclusion efforts are designed to help address both health disparities and building the pipeline of fundable minority researchers through proof-of-concept repurposing trials.
Clare Thibodeaux, Ph.D., Director of Scientific Affairs, Cures Within Reach
Social Impact Investing: New Ideas for Deprioritized Drug Programs
Drug programs are often deprioritized in development due to reasons other than efficacy. The truth is: most of these deprioritized or “shelved” drug programs can help patients, and can generate financial returns for drug developers and their investors in the process. We propose to unleash the full promise of shelved programs for patients by improving market efficiency for these deprioritized programs. We are partnering with traditional and non-traditional stakeholders to achieve our mission.
Rachel Rubin, Vice President for Programs, Roivant Social Ventures
The Opportunities and Challenges of Licensing Academic Repurposing Assets for Industry
Dr. Bruce Bloom, Founder, Cures Within Reach, Chief Collaboration Officer, Healx
Investment Transactions in 505(b)(2) Product Pathways: An Investor’s Perspective
In the session, you’ll learn about:
Which asset classes are deploying capital to develop products and companies
Why investors allocate capital into projects that offers competitively differentiated products and market exclusivity which address unmet medical needs in significant markets
Why investors view innovation as a key success factor that must lead to sufficient ROI at an acceptable risk to warrant a capital investment
David H. Crean, Ph.D., MBA, President & CEO, Coast Bioventures
PANEL SESSION: Investing in Repurposing: Strategies for Influencing the Capital Markets' Perception of Drug Repurposing
David H. Crean, Ph.D., MBA, President & CEO, Coast Bioventures
Michael Derby, Managing Partner, TardiMed Sciences
Sven Jacobson, MBA, CEO, Woolsey Pharmaceuticals, Founder & CEO, Martin Pharmaceuticals
Drug Repurposing Strategies for Global Health: Screening of the ReFRAME Library to Identify COVID-19 Interventions
The ReFRAME library is a comprehensive repurposing library of 12,000 compounds generated with support from the Bill & Melinda Gates Foundation to accelerate discovery of treatments for global health. With the onset of the COVID-19 pandemic, Calibr shared the library with virology labs around the world to accelerate the discovery of therapeutic interventions. They also rapidly developed in-house screens to identify drugs that could be repurposed as treatments for SARS-CoV-2 infection and as advanced starting points for medicinal chemistry optimization. In this talk Dr. Bakowski will discuss the development and results of their anti-SARS-CoV-2 screening cascade.
Malina Bakowski, Ph.D., Principal Investigator, Calibr at Scripps Research
Repurposing Beta-lactams for Buruli Ulcer Therapy, From the Bench to the Clinic
Drug discovery and development for NTDs (Neglected Tropical Diseases) is not an attractive activity to the pharma industry due to low returns compared to pharmaceutical standards, as such, new approaches need to be implemented. Repurposing clinically approved might help to speed up the development of new therapies for NTDs. Beta-lactams are one of the largest groups of antibiotics available today with an exceptional record of clinical safety in humans. Dr. Ramon-Garcia and his team recently demonstrated that beta-lactams combined with rifampicin and clarithromycin are synergistic in vitro against M. ulcerans They thus focused on amoxicillin/clavulanate, which is oral, suitable for treatment in adults and children, and readily available with an established clinical pedigree.
In this presentation, he will discus the scientific rational behind the progression of amoxicillin/clavulanate as a new anti-BU treatment in combination with current oral BU therapy, rifampicin and clarithromycin, and the steps undertaken towards its validation, including a clinical trial in Benin.
Santiago Ramon-Garcia, Ph.D., ARAID Investigator, Research and Development Agency of Aragon (ARAID) Foundation
Fast-Tracking Affordable Cancer Treatments Using AI
Reboot Rx is the tech nonprofit startup dedicated to saving the lives of cancer patients using repurposed generic drugs. Repurposing FDA-approved generics represents a major opportunity to quickly improve outcomes for cancer patients and reduce healthcare costs. Hundreds of non-cancer generics have already shown promise for treating cancer across thousands of published studies, but it would take years to manually review the data and identify the most promising drugs for repurposing. Reboot Rx is building AI technology for rapid evidence synthesis and prioritization of drug candidates. Dr. Kleiman will discuss how Reboot Rx is partnering with cancer patients and oncologists to identify the top candidates to advance into the clinic.
Laura Kleiman, Ph.D., Founder & CEO, Reboot Rx
Drug Repositioning as a Therapeutic Strategy to Slow the Trajectory of Neurodegeneration in Patients with Parkinson’s disease; a Large Global Program
For the past decade our organisation has facilitated a a major drug repositioning initiative which is currently running many (<20) neuroprotective clinical trials each involving a different (mostly repurposed) therapeutic. A committee of >21 global experts in Parkinsons meet regularly to select which drugs will newly enter long-term clinical trials (typically of 1-2 years duration). Their drug selection process is assisted by running drug screens (of up to 230,000 compounds) to interrogate relevant potency on biological targets, and also typically underpinned by a variety of preclinical studies. This presentation will review lessons learnt in running a multinational drug repositioning program involving so many different therapeutic classes, and the excellent progress so far.
Richard Wyse, MD, Director of Research & Development, The Cure Parkinsons Trust
Drug Repositioning as a Pharmaceutical Strategy: The Obvious Benefits are Real but Beware of Pitfalls That May be Less Apparent
For over 15 years drug-repositioning has been a recognized approach towards bringing new therapeutics to market and has continued to grow in popularity over this time frame. Melior Discovery is a company with a founding mission around drug repositioning. The speaker shares his perspective on lessons learned over 15 years of conducting drug repositioning efforts. He will discuss advantages and disadvantages that he has encountered using this approach and why, overall, this means of drug discovery provides a compelling business rationale, particularly if one approaches with an “eyes wide open” awareness of pitfalls.
Andrew Reaume, Ph.D., MBA, President & CEO, Co-Founder, Melior Discovery
Intellectual Property Strategies for Repurposed Products
This topic will lay the foundation for why patents are a critical component for any repurposed product. It will explore the regulatory framework and how intellectual property is addressed in this framework and also discuss strategies for developing patent portfolios to maintain a competitive edge.
Matthew Fedowitz, JD, Shareholder, Buchanan Ingersoll & Rooney