World Orphan Drug Congress LIVE: Pitch and Partner Sessions Day 1

In Clinical Development, Orphan Drug Congress, Orphan Drugs Live, Partnering & Investment by Freya Smale

The presentations today in our pitch and partner conference room had much success.

Mark Varney, Co-Founder, President and Chief Executive Officer, Neurolixis speaks about NLX-101 for the treatment of Rett Syndrome.

Rett Syndrome affects about 1 in 10,000 females. The disease is caused by a mutation in the MeCP2 gene causing most commonly breathing and movement disorders. NLX-101 is a selective and efficacious 5-HT 1A receptor “biased agonist.” The 5-HT 1A receptors stabilizes breathing.  NLX-101 improves respiratory disorders in transgenic Rett Mice.

Marc Hedrick, Chief Executive Officer, Cytori Therapeutics talks about the treatment of patients with hand and finger involvement due to scleroderma and symptomatic knee osteoarthritis.

Some main points included Cytori Therapeutics’ technology, which involves automated bedside manufacturing system. Their development of ECCS-50 for treatment of Scleroderma is at Phase 3 pivotal trial.

Ann Tsukamoto, Executive Vice President, Scientific and Strategic Alliances, StemCells Inc. on Human Neural Stem Cells and possible orphan applications.

HuCNS-SC- First in Class Biologic. Some pre-clinical observations reflected in early clinical results addressed the Lysosomal Storage Disease, Myelin disorder, and also Spinal Cord Injury and Dry Age. These cells are a platform technology for rare CN?S diseases.

Teresa Leezer, Chief Executive Officer, Rhinocyte Inc. addresses Stem Cell treatments for orphan designations.

Teresa introduces Rhinocyte advantages plus their clinical targets which are Spinnal Cord Injury and Parkinson’s Disease.

Antonio Lee, Chief Executive Officer and Managing Director, Medipost America speaks about Pneumostem for the prevention of Bronchopulmonary Dysplasia (BPD).

BPD has about 10,000 cases in the US. Pneumostem is up to phase II and III in trial testings.

David Pernock, Chief Executive Officer, Fibrocell Science on rare skin and connective tissue diseases. 

Fibrocell Science is transforming lives of patients with Recessive Dystrophic Epidermolysis Bullosa, which holds about 1,100-1,500 cases in the US. They are pre-clinical so far.

Jan Rosenbaum, Chief Scientific Officer, Airway Therapeutics on AT-100 and the prevention of bronchopulmonary dysplasia. 

AT-100 received the orphan designation for prevention of BPD in the US and Europe. It is a novel targeted therapy in development. The results stabilize the lungs and improves immunity.

Jeet Mahal, Head of Corporate Development, Portola speaks about Andexanet Alfa: a breakthrough-designated Factor Xa Inhibitor antidote.

The use of novel oral anticoagulants is large and growing rapidly. Current reversal strategies were designed for Warfarin and have not shown effective reversal of novel fXa agents. Andexanet alfa is a rationally designed modified factor Xa Decoy. It’s been granted orphan designation and is committed to doing a phase 4- ongoing open-label, single-arm study.

J. Michael French, President & CEO, Marina Bio on nucleic acids as promising candidates for rare disease treatments.

Marina Bio has a novel and proprietary chemistries as well as delivery systems. Their delivery capability is unparalleled within the sector. They have two separate and distinct delivery technologies in clinical development: SMARTICLS and tkRNAi. CEQ508-FAP is currently in Phase I. Myotonic Dystrophy and Duchenne Muscular Dystrophy in lead selection.

Margaret Lee, VP, Product Leadership & Biology, Karyopharm Therapeutics discusses promising orphan therapeutics: an overview of Karyopharm’s SINE™ compounds

Selinexor (KPT-330) is granted orphan designation by the FDA in Multiple Myeloma. There has been 700+ patients treated to date.

Christopher M. Cashman, Chairman & CEO, Marinus Pharmaceuticals  speaks to us about PCDH19 for the treatment of pediatric epilepsy.

Ganaxolone is the first-in-class therapeutic for epilepsy and neuropsychiatric disorders. Ganaxolone is designed to modulate GABA through synaptic and extrasynaptic receptors. Marinus Pharmaceuticals have currently taken this to phase 2 proof-of-concept study ongoing in 2-10 year old females. 135 unique pediatric subjects have already been dosed with ganaxolone in children with epilesy

 Informative sessions in the Pitch & Partner workshops overall; also heard great feedback of the pitches from Opsona Therapeutics, Andarix, Valley Fever Solutions, Recursion Pharmaceuticals, RASopathies Network USA, and Sixera Pharma AB.