Program Description

The small patient populations associated with rare diseases reduce the incentive for companies to invest time and money to develop treatments. To make investing in developing new therapies for these rare diseases, so-called orphan drugs, more attractive, the U.S. and other major countries have created programs specifically for rare diseases, changing the cost/benefit equation by offering incentives that can reduce cost and time to market and increase the revenue potential. This program will feature a representative of the FDA’s Office of Orphan Products Development to set the stage for the discussion by a panel of entrepreneurs with the unique challenge of developing a drug for a rare disease.

Program Moderator

Jules Fried, CEO & President, Vasogenesis

Jules Fried is a serial entrepreneur, co-founder, and Chief Executive officer of Vasogenesis, a clinical-stage drug company repurposing an off patent biologic to treat vascular diseases, some of which are rare diseases.

Keynote

Lewis Fermaglich, MD, MHA, Medical Officer, Office of Orphan Products Development, FDA

Dr. Fermaglich is a board-certified pediatrician with a passion for working with children with rare diseases and their families. He has been working as a Medical Officer in The Office of Orphan Products Development (OOPD) since September 2019.

Program Panelists

Timothy R. Coté, M.D., CEO, Silk Road Therapeutics

Dr. Timothy R. Coté is co-founder and CEO of Silk Road Therapeutics, founded to develop a treatment for Behcet’s Disease, a rare disease for which he received orphan drug designation and Fast Track status.

Christopher Leo Ph.D., MBA, Managing Director, Back Bay Life Science Advisors

Chris focuses his practice on product/market strategies and diligence support across pharma, biotech, and specialty pharma.

John Parker, Founder, Springhood Ventures

John founded Springhood Ventures to invest in young companies solving the biggest problems in children’s health.

Cesare Spadoni, Ph.D., MBA, Founder, COO & Board Member, Oncoheroes Biosciences

Cesare co-founded Oncoheroes Biosciences Inc., which is exclusively focused on pediatric oncology drug development.

Join this timely keynote presentation, and blue-ribbon panel discussion focused on opportunities in orphan indications. Ask questions and contribute your insights during the interactive audience segment.

Who should attend?

C-level executives, board members, investors, and other senior leaders who want a better understanding of opportunities in orphan indications.

Speaker Bios

Timothy R. Coté, M.D., Co-founder and CEO, Silk Road Therapeutics

Dr. Timothy R. Coté is CEO and co-founder of Silk Road Therapeutics, founded to develop a treatment for Behcet’s Disease, a rare disease for which he received orphan drug designation and Fast Track status which recently completed a successful Phase 2 trial in Turkey. He is also founder and CEO of Only Orphans Cote, a consultancy focused on rare diseases and orphan drug development. Between 2007 – 2011, he was Director of the Office of Orphan Product Development (OOPD) of the FDA, leading the agency’s efforts in implementing the Orphan Drug Act and personally signing decisions on over 1400 requests for orphan drug designation. As a scientist, entrepreneur and advisor to a broad spectrum of biotech and pharmaceutical clients across Asia, Europe and the US, his experience spans the various stages of drug development and approval pathways in the US, the UK and Europe.

An anatomic pathologist and medical epidemiologist, Dr Coté has published 80 peer reviewed articles on a variety of medical topics including HIV/AIDS-related malignancies, typhoid fever epidemics, infectious diseases in conflict-affected countries. He received a Bachelor’s degree from Syracuse University and MD from Howard University College.

Lewis Fermaglich, MD, MHA, Medical Officer, Office of Orphan Products Development, FDA

Dr. Fermaglich is a board-certified pediatrician with a passion for working with children with rare diseases and their families. He has been working as a Medical Officer in The Office of Orphan Products Development (OOPD) since September 2019. He received his undergraduate degree at Wesleyan University and then an M.D. from the University of Kentucky College of Medicine. He completed his pediatric residency at Children’s National Medical Center in Washington, DC, where he was Chief Resident. After residency, he was a practicing general pediatrician for 10 years – first as a military physician, and then in private practice in Rockville, MD. Lewis came to FDA in 2017, originally assigned to the Division of Clinical Review (DCR) in the Office of Generic Drugs (OGD). Additionally, in 2018, he completed an Executive Master of Health Administration at the George Washington University.

Jules Fried, CEO & President, Vasogenesis

Jules Fried is a serial entrepreneur, co-founder, and Chief Executive officer of Vasogenesis, a clinical stage drug company repurposing an off patent biologic to treat vascular disease without surgical intervention. Before Vasogenesis, Jules was CEO of Interactive Motion Technologies, a manufacturer of robotic rehabilitation equipment for stroke victims which he sold to a microcap public company. He was also a Principal at Atlantic VIC, a technology venture development company licensing technology out of local research institutions, and founder and board member of First Commons Bank, one of the fastest growing community banks in the country, sold last year to Brookline Bank. Earlier in his career, he built and led a two-time Inc. 500 digital printing peripheral equipment manufacturer as a member of the founding management team. Before that, Jules was a partner in the law firm of McDermott, Will & Emery, having co-founded a boutique health care law firm that became their Boston office. Mr. Fried started his legal career in the Antitrust Division of the U.S. Department of Justice and was part of the team responsible for the litigation resulting in the breakup of AT&T. He has a J.D cum laude from the University of Michigan Law School and is a graduate of Princeton University and has led many successful NEHEN programs.

Christopher Leo Ph.D., MBA, Managing Director, Back Bay Life Science Advisors

Christopher Leo, PhD, is a Managing Director, Back Bay Life Science Advisors. He joined the firm at its founding in 2010; focus areas include business unit/product/market strategies and diligence support across large pharma, biotech, and specialty pharma. He has significant expertise at the interface of industry and academia around strategies for collaborating and advancing novel technology.

Dr. Leo joined Back Bay Life Science Advisors from Genzyme, where he led strategic & competitive planning projects for the company’s $2 billion Rare Genetic Disease and $500 million Biosurgery business units. Before Genzyme, he was a Director with Leerink Swann’s Strategic Advisory group and a Principal in Wood Mackenzie’s Life Science consulting practice.

Dr. Leo was also Director of the Belfer Cancer Genomics Center at the Dana Farber Cancer Institute, under the leadership of Drs. Ronald DePinho and Lynda Chin. He has managed an Oncogenomics research group at Variagenics/Nuvelo.

Dr. Leo completed his doctorate in Biochemistry and Molecular Pharmacology at the University of Massachusetts Medical School and holds an undergraduate degree in Biology, cum laude, from The College of the Holy Cross.

John Parker, Founder, Springhood Ventures

John founded Springhood Ventures to invest in young companies solving the biggest problems in children’s health and to capture attractive venture returns in the process. In parallel to this role, he established and manages CH Innovations, the impact-first venture program of the Charles H. Hood Foundation, a Boston-based private foundation that supports pediatric research, where he also serves as a trustee.

John is an observer on the boards of Nicolette, Argus Cognitive, Anida Pharma, Mesentech, Inc., BioROSA Technologies, 149 Medical, and Prapela, Inc. He also serves on the advisory boards of multiple nonprofit entities, largely around pediatric health and mission-driven healthcare investing, and a number of pre-launch pediatric-focused startups. He is a frequent speaker on pediatric innovation, investment in child health solutions, and mission investing.

John has a BA from Dartmouth College and an MBA from Dartmouth’s Tuck School of Business.

Cesare Spadoni, Ph.D., MBA, Founder, Chief Operating Officer & Member of Board of Directors, Oncoheroes Biosciences

Cesare has more than 20 years of experience in the drug development field, in both scientific and commercial roles. He is the co-founder and Chief Operating Officer of Oncoheroes Biosciences Inc, a Boston-based biotech company exclusively focused on pediatric oncology drug development. Prior to that he held senior positions at AMRI, Aptuit Laurus, ThalesNano and Auxiliis BV. Cesare started his career as a research scientist in a pharmaceutical company (Eisai, London) and in academia (Institute of Enzymology, Budapest).

Following the death of his first daughter Laura to cancer, Cesare set up aPODD Foundation, a London-based charity focused on accelerating pediatric oncology drug development. As aPODD’s chairman and trustee he is actively involved on a pro-bono basis in drug repurposing projects, advocacy efforts and research collaborations with other childhood cancer charities.

Cesare holds an MSc in Applied Molecular Biology and a PhD in Neurosciences from UCL, University of London, as well as an MBA from the Central European University, Budapest.

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