PITCH & PARTNER

 

Pitch & Partner is the only rare disease focused platform for biotech and academia to pitch their orphan drug candidates and rare disease programs to prospective investors, pharma R&D, partners, grant agencies, and licensing companies. Pitch & Partner will feature 30 biotechs pitching their orphan drug candidates as well as investors, accelerators and funding organizations showcasing their funding and business models

 

Key Speakers

 

Pitch and Partner, Wednesday 10 April 2019

last published: 13/Feb/19 21:55

Pitch and Partner, Thursday 11 April 2019

Ellen Coleman
08:25

Chairperson's opening remarks

Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
John Maraganore
08:30

Keynote Address: Advancing RNAi therapeutics as a whole new class of medicines

Christopher Austin
08:55

Keynote Address: Re-thinking and reshaping “rare” – continuing the march towards curative therapies for the rare disease community

Arndt Rolfs
09:20

Keynote Address: Accelerating Orphan Drug development and improving outcomes, by unlocking genetic information

10:30

10:30 SPEED NETWORKING FOLLOWED BY MORNING NETWORKING BREAK

Pitch and Partner
11:50

Development, Sanofi Walter Kowtoniuk, Principal, Third Rock Ventures Heather Behanna, Principal, Sofinnova Ventures Debra Miller, President & CEO, Cureduchenne

Imran Babar
Pitch and Partner
12:15

How a venture-backed orphan drug accelerator is advancing promising therapies for rare diseases

Alain Vicari
Pitch and Partner
12:40

Pitch 1: Humanized monoclonal antibody targeting Interleukin-15 for the treatment of Eosinophilic Esophagitis

Alan Cash
Pitch and Partner
12:50

Pitch 2: Anhydrous Enol-Oxaloacetate as a novel platform drug for CNS Intercellular Glutamate reduction and rare brain diseases

John Freshley
Pitch and Partner
13:00

Pitch 3: ONL1204, First-in-Class inhibitor of Fas (CD95) signaling to protect vision from retinal disease

13:05

1:05 NETWORKING LUNCH

2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min) – SPONSORED ROUNDTABLES AVAILABLE

Tiina Urv

ROUNDTABLE 10: Data ownership – addressing the need for increased data sharing while also considering company/patient data "ownership" through clinical trials and beyond

Tara Mathiesen

ROUNDTABLE 11: Educating HCPs – when considering the absence of newborn screening lessons learned from Metachromatic Leukodystrophy (MLD)

ROUNDTABLE 12: Chilling investment? – the potential implications of increased public scrutiny of rare disease drug prices in the US

Vipul Kashyap

ROUNDTABLE 13: Cross-Ecosystem collaborations – mutual value propositions and collaborations for pharma, payers and providers to collaborate in the rare diseases space

Harsha Rajasimha

ROUNDTABLE 14: Rare in India – engaging patients with rare diseases in India with global clinical trials and resources

Paul Melmeyer

ROUNDTABLE 15: Federal Policy – update on rare disease federal policy and NORD initiatives to engage capital hill

Clive Whitcher

ROUNDTABLE 17: Patient Access in Europe & Beyond - Enabling access for rare disease therapies exploring alternative pathways for patients in need

Clive Whitcher, Vice President Head of Global Patient Access, Inceptua Commercial Products
Kelly Fearn, Principal Consultant, Inceptua Medicines Access
Susan Robinson
14:30

ROUNDTABLE 18: Market Access & commercialization – how specialty services and options should vary for patient populations of 20, 2,000, and 20,000

Leone Atkinson
14:30

ROUNDTABLE 19: Update on patient centricity in research – preliminary survey results from patient advocacy group leaders

Anne Cropp

ROUNDTABLE 1: Global expanded access – applying artificial intelligence and access innovation for expanded access programs

David Lapidus

ROUNDTABLE 3: Epidemiology & forecasting – value, challenges, and methods for rare diseases

Joseph Musumeci

ROUNDTABLE 4: BD & forecasting – how to build forecasts based on available epidemiological information and considerations for pricing and access

Scott Schliebner

ROUNDTABLE 5: Patient-focused research – new approaches to bring clinical trials directly to patients and change the patient-centric trial paradigm in rare diseases

Jamie Arnott

ROUNDTABLE 6: Patient-centered Development: Early patient input, understanding patient goals in clinical trials, and furthering relationships between all stakeholders

Lisa Dilworth

ROUNDTABLE 7: Meaningful endpoints – navigating the differences in what it means to patients, caregivers, physicians and regulators during advanced therapies clinical development

Kate Grady

ROUNDTABLE 8: Driving diagnosis – steps industry can take to improve the diagnosis of rare disease

14:30

ROUNDTABLE 9: Sponsored by Catalent Pharma Solutions

Pitch and Partner
14:30

BIOTECH PITCHES

Ray Lee
Pitch and Partner
14:30

Pitch 4: Induction of tumor necrosis in hepatocellular carcinoma to enhance the efficacy of immune checkpoint inhibitors by combining a hypoxia-activation agent with embolization

Maurizio Chiriva
Pitch and Partner
14:40

Pitch 5: Next generation of immunotherapeutic target discovery platform for rare diseases

Martin Devenport
Pitch and Partner
14:50

Pitch 6: CD24Fc for the prevention of GVHD and damage mediated inflammation

ROUND 2 (select a second roundtable to attend from the options above)

Frank Sorgi
Pitch and Partner
15:00

Pitch 7: FLAG-003 as an oral therapy for the treatment of glioma

Manohar Katakam
Pitch and Partner
15:10

Pitch 8: ST-001 for treatment of Cushing’s syndrome

Joe Young
Pitch and Partner
15:20

Pitch 9: Cibinetide for treatment of sarcoidosis-related small fiber neuropathy

Kieran Rooney
Pitch and Partner
15:30

Pitch 10: AP101 for the treatment of Epidermolysis bullosa

Vincent Forster
Pitch and Partner
15:40

Pitch 11: VS-01 for the treatment of overt hepatic encephalopathy and Acute-on-Chronic Liver Failure

Manijeh Goldberg
Pitch and Partner
15:50

Pitch 12: Nanoengineered chemotherapy patch for oral cavity cancer

Johan Drott
Pitch and Partner
16:00

Pitch 13: Treatment of diffuse large B-Cell Lymphoma (DLBCL) VAL001 - a phase IIb/III ready drug

Mingxin Qian
Pitch and Partner
16:10

Pitch 14: TL-010 for treatment of schistosomiasis, foodborne trematodiases and taeniasis/neurocysticercosis

Patrick Lu
Pitch and Partner
16:20

Pitch 15: Development of RNAi therapeutics for treatment of Cholangiocarcinoma (an orphan indication)

Matthew Kalnik
Pitch and Partner
16:30

Pitch 16: First-in-class nicotine-blocker in development for the treatment of Buerger’s Disease: ATI-1013, a human anti-nicotine monoclonal antibody

Shawn Ritchie
Pitch and Partner
16:40

Pitch 17: Synthetic plasmalogen precursor PPI-1040 for the treatment of Rhizomelic Chondrodysplasia Punctata (RCDP) and other rare pediatric peroxisomal disorders

Linda Hogan
Pitch and Partner
16:50

Pitch 18: Lixivaptan for the treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)

17:00

5:00 AFTERNOON NETWORKING BREAK

18:30

6:30 NETWORKING DRINKS RECEPTION – Sponsored by Orchard Therapeutics

last published: 13/Feb/19 21:55