World Orphan Drug Congress LIVE: Pitch and Partner Sessions

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Pitch & Partner has been quite beneficial for both sides. We’ve heard wonderful pitches and saw plenty of interests. Here are some of the pitches at World Orphan Drug Congress Conference Day 2.

Pioneering the development of gene therapy based treatments for retinal degenerative diseases. Jean Philippe Combal, Chief Operations Officer, GenSight Biologics

  • About 40-45 million people are blind
  • Geosight platform->degenerative retinal diseases

Jean Philippe explains GS010 and GS030. GS010 : AAV2/2-ND4 is to preserve or restore vision. It is a transformative therapy for LHON and is currently passing Phase I//II a safety & tolerability study. GS030 has significant potential in RP and AMD. Ultimately GenSight is looking into a sequential process to restore retinal function through optogenetics

Oral biological candidate for the treatment of PKU. Gjalt Huisman, VP, Pharmaceutical Technology & Innovation, Codexis

  • PKU is the inability to convert Phe to Tyr with about 1 in 15,000 cases
  • Ideal situation is to use oral enzyme therapy for PKU treatment
  • Codexis holds world-class protein Engineering Platform
  • Codexis is engineering an Oral-ePAL for PKU treatment

IFB-088, a breakthrough therapeutic approach to treat orphan degenerative diseases caused by misfolded protein accumulation. Pierre Miniou, Chief Business Officer, InFlectis BioScience

IFB-088 is in the late preclinical development. It is a small molecule entity, orally available, has validated mechanism of action, safe, potent, and a strong patent protection. Pierre gives compelling data that support a de-risked project.

InFlectis has interests in ALS, CMT-1 (charcot-Marie-Tooth disease 1), Tretinal Ciliopathies and their primary focus is on orphan diseases. The goal is to have IFB-088 capitalize on orphan disease incentives and have the support of patient advocacy groups.

Some orphan diseases are only skin deep: the role of Granzyme B. Alistair Duncan, CEO, viDA Therapeutics

  • viDa Therapeutics is the only company that supports tissue injury repair via novel targets and inhibitors. They have a library of first-in-class granzyme inhibitors
  • Granzymes is an exciting new extracellular target. There is a new paradigm: extracellular GzmB- perforin INDEPENDENT mechanism of action.
  • Alistair explains how GzmB-deficient mice exhibited reduced skin aging and improved collagen remodeling. UV induces GzmB in mouse/human skin; GzmB deficiency prevents wrinkling, improved collagen remodeling. Ultimately, this can treat Discoid Lupus Erythematosus: an Orphan Disease triggered by sunlight.

Delivery of enzyme replacement therapies across the blood brain barrier to treat lysosomal storage diseases. Derek Kelaita, VP, Business Development, ArmaGen

ArmaGenHas a proprietary platform and a robust pipeline of therapies for devastating, previously unaddressed neurological symptom.

There is a Blood-Brain Barrier dilemma. BBB protects brain from bacteria and foreign substances.

  • The ArmaGen Solution- ArmaGen leverages body’s natural active transport systems present on BBB –it utilizes antibody and therapeutic fusion protein. ArmaGen transports a wide range of therapies to the human brain by targeting insulin receptors.
  • AGT-182 can be distributed extensively to all regions of the brain within 2 hours of IV injection in Rhesus monkey

AeroVanc’s successful Phase II clinical trial for Cystic Fibrosis. Taneli Jouhikainen, Chief Operating Officer, Savara Pharma

Savar Pharmaceuticals is an emergimg specialty pharmaceutical company focused on rare respiratory diseases.

There are limited treatment options for MRSA.

AeroVanc is the first inhaled MRSA antibiotic and has completed phase II –proof of concept study. The primary endpoint of the study is to reduce MRSA.

Selectively target signaling pathways that lead to tumor-promoting inflammation.  John Maki, President and CEO, Vicus Therapeutics

A couple of key points to take away:

  • Vicus is the next generation immuno-oncology company
  • VT-122 is the first multi-pathway neuro-immune modulator for cancer
  • Vicus Therapeutics is focused on advanced liver cancer, non-resectable liver cancer and more.
  • Top KOLS will lead two approval directed studies in 2016
  • NDAs are expected in 2018(pancreatic) and 2019 (liver)

A Phase 3 study of L-glutamine therapy for sickle cell anemia and sickle β◦-Thalassemia. Charles Stark, SVP, R&D, Emmaus Life Science

Phase 3 study included a 48-week trial. They found that safety data similar between L-glutamine and placebo arms of the trial.

Living Rare: Families finding help and hope for Menkes Disease. Jamie Eckman, President, The Menkes Foundation

Menkes is a rare fatal genetic disorder. If not treated a patient could pass away within the first 3 years of their life. It’s important to diagnose the disease as soon as possible to ensure the treatments can work effectively. It’s the only hope out there.

SCENESSE as an innovative therapy for EPP. Nicoletta Muner, Director, Global Regulatory Affairs, Clinuvel

  • Scenesse is a first-in-class melanocortin-1 receptor (MC1R) agonist

SCENESSE’s clinical program focuses on:

  • Erythopoietic protoporphyria (EPP) – an orphan metabolic disease (heme biosynthesis)
  • Vitiligo- depigmentation disorder of unknown etiology

All the presenters of the Pitch & Partner conference did a spectacular job and took full advantage of this networking opportunity.

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