Pitch & Partner


Pitch and Partner, Monday 17 August 2020

Ulrich Thienel
Pitch and Partner

Pitch 1: Development of RLS-0071 in Hypoxic Ischemic Encephalopathy

Ulrich Thienel, Chief Executive Officer, ReAlta Life Sciences
Rashida Karmali
Pitch and Partner

Pitch 7: Phase IB trial of Carboxyamidotriazole Orotate (Pravitinib™) in glioblastoma patients

Rashida Karmali, CEO, Tactical Therapeutics Inc
Samarendra Mohanty
Pitch and Partner

Pitch 8: Multi Characteristic Opsin for vision restoration in patients with retinitis pigmentosa

Samarendra Mohanty, Founder And President, NanoScope Technologies
Ngeh Toyang
Pitch and Partner

Pitch 9: Targeting pancreatic cancer with Caflanone

Ngeh Toyang, Chief Executive Officer, Flavocure Biotech
David Young
Pitch and Partner

Pitch 10: Processa Pharmaceuticals, Inc. – combining High Growth with Lower Risk

David Young, Chief Executive Officer, Processa Pharmaceuticals, Inc.
Nawal Ouzren
Pitch and Partner

Pitch 11: Sensorion Innovative therapies to treat, prevent and restore within the field of hearing loss disorders

Nawal Ouzren, Chief Executive Officer, Sensorion Pharma
Prashant Kohli
Pitch and Partner

Pitch 12: Grace Therapeutics: Innovative Products for Orphan CNS Conditions

Prashant Kohli, Vice President Commercial Operations, Grace Therapeutics
Therese Kinsella
Pitch and Partner

Pitch 13: NTP42, an antagonist of the thromboxane receptor, attenuates experimentally induced pulmonary arterial hypertension (PAH)

Therese Kinsella, Founder And Chief Scientific Officer, ATXA Therapeutics
William Xiang
Pitch and Partner

Pitch 14: Crossborder Orphan Drug Development: Differences in China and the US

William Xiang, Director, Strategy And Operations, XW Laboratories
Alan Cash
Pitch and Partner

Pitch 15: Reversing the Warburg Effect for Glioblastoma Treatment

Alan Cash, Chief Executive Officer And Co-Founder, MetVital
Robert Lodder
Pitch and Partner

Pitch 16: BSN175 for the treatment of Prader-Willi Syndrome

Robert Lodder, President, Biospherics
Sean Ekins
Pitch and Partner

Pitch 17: Using machine learning to discover Pyronaridine for the orphan disease Ebola

Sean Ekins, Founder And Chief Executive Officer, Collaborations Pharmaceuticals
Jarrod Longcor
Pitch and Partner

Pitch 18: CLR 131 a novel targeted radiotherapeutic for the treatment of multiple myeloma, lymphoplasmacytic lymphoma, and pediatric neuroendocrine tumors, sarcomas and brain tumors

Jarrod Longcor, Chief Business Officer, Cellectar Biosciences Inc
Randy Milby
Pitch and Partner

Pitch 19: Salinomycin QUATRAMER for Small Cell Lung Cancer (SCLC

Randy Milby, Chief Executive Officer, Hillstream Biopharma
Alon Ben-Noon
Pitch and Partner

Pitch 21: Elements of creativity in orphan drug development

Alon Ben-Noon, CEO, NeuroSense Therapeutics
last published: 25/Aug/20 17:55


Pitch and Partner, Monday 24 August 2020



Ellen Coleman

Chairperson’s opening remarks

Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
Jeremy Levin

Keynote Address: Rare diseases in the time of Covid-19

Jeremy Levin, Chairman and Chief Executive Officer, Chairman, BIO, Ovid Therapeutics
Emil Kakkis

Keynote Address: The future of rare diseases: developing and accessing the next generation of therapies

Emil Kakkis, Chief Executive Officer, Ultragenyx Pharmaceutical
Neena Nizar

Keynote Panel: Patient involvement in drug development for rare diseases – examining how patients are driving meaningful development and what companies are doing to incorporate patient input throughou

Neena Nizar, President, The Jansens Foundation
Jessica Scott, Head of R&D Patient Engagement Office,, Takeda Pharmaceuticals
Amy Mckee, Vice President, Regulatory, Parexel Consulting, Parexel International
Dara Richardson-Heron, Chief Patient Officer, Pfizer
Yann Le Cam, Chief Executive Officer, EURORDIS




Amanda Finlayson

ROUNDTABLE 1: Balancing the Patient Experience & ROI – understanding when to incorporate patient accommodations into study programs and balancing short and long term financial goals

Amanda Finlayson, Therapeutic Strategy Lead, ClinEdge
David Lapidus

ROUNDTABLE 2: Forecasting & epidemiology - rare-disease epidemiology methods and resources to support commercial forecasts

David Lapidus, President, Lapidus Data
Sam Lucas

ROUNDTABLE 3: Expanded Access Programs- How EAPs can complement rare disease drug development and optimize commercial launch

Sam Lucas, SVP, Expanded Access Programs, Durbin
Julia Campbell, VP, Client Strategies and Services, Durbin
Todd Galles, VP, US Business Development, EAPs, Durbin
Janet Maynard

ROUNDTABLE 4: FDA updates - Office of Orphan Product Development

Janet Maynard, Director, Office Of Orphan Products Development, Food And Drug Administration, U.S. Food and Drug Administration
Lewis Fermaglich, Medical Advisor, Office of Orphan Products Development,, FDA


Oodaye Shukla

Keynote Panel: Examining industry strategies for leveraging patient data and AI to advance rare disease patient identification, R&D, and commercialization

Moderator: Oodaye Shukla, Chief Data Officer, HVH Precision Analytics, now EVERSANA
Thomas Abbott, Head, Real World Data And Evidence, Astellas Pharma
Sonalee Agarwal, Vice President Of Value And Evidence Strategy, Alnylam Pharmaceuticals
Sean Khozin, Global Head Of Data Strategy, Johnson & Johnson
last published: 25/Aug/20 17:55