Retinopathy of Prematurity

Retinopathy of prematurity (ROP) is the leading cause of preventable childhood blindness. With the improved survival of early born babies in both high- and low-income countries ROP has become a global health issue. With 1 million infants at risk every year, 200,000 develop any form of ROP with 60,000 going on to severe vision threatening ROP and a high risk of becoming blind. The cause of ROP is abnormal blood vessel development (pathological neovascularization) in the retina of the prematurely born infant. AP-001 in relevant models is shown to prevent such neovascularization, and,  importantly, to stimulate normal blood vessel outgrowth. The data support the development of AP-001 as an eye drop for safe and convenient use from birth through to full term age to prevent severe ROP in high risk infants. There is currently no approved drug treatment to prevent the development of ROP.

Neurotrophic keratitis

Neurotrophic keratitis is a rare progressive eye disease leading to corneal scarring and vision loss. The cause is damage to the branches of the fifth (trigeminal) sensory nerve that innervates the cornea. To protect from harmful insults the cornea is the most densely innervated organ of the body but its innervation additionally provides necessary trophic support to maintain structural integrity and  trans-parency. With loss of innervation, most commonly caused by viral (herpes simplex or herpes zoster) infections, surgery or trauma, trophic support is lost and corneal integrity threatened. AP-001 is experimentally shown to restore corneal innervation and normalize sensation following surgical trauma. AP-001 is as potent as nerve growth factor (NGF) in stimulating trigeminal nerve fiber growth and additionally, and independently thereof, stimulates corneal healing and dampens inflammation commonly associated with neurotrophic keratitis. AP-001 will be an easy to administer once or twice daily eye drop.

Drug-induced hearing loss 

Four to five children of every thousand born alive will suffer lack of brain oxygenation, develop brain ventricular hemorrhage or stroke in the perinatal situation. Survivors will grow up with permanent handicaps, which may be severe, including cerebral palsy, cognitive impairment, and chronic epilepsy, and for many individuals with a life- long depen-dency to function in society. In models of neonatal brain ischemia and stroke, as well as adult stroke, AP-001 treatment reduced loss of brain tissue and improved post-stroke function, with treatment beginning after the insult. AP-001 was further shown to be neuroprotective, to promote neurogenesis and to prevent epileptogenesis. Current treatment of babies at risk following an ischemic event is limited to cooling but is insufficient, and no drug treatment exists to further reduce brain damage. For babies suffering a stroke or ventricular bleeding no treatment is available.

Per Gjorstrup, MD PhD

President and Chief Executive Officer

Co-founder Resolvyx Pharmaceuticals.
Senior management positions: Micromet,
Biogen, Pharmacia (Pfizer), Astra

Thomas P. Sakmar, MD

Research

Richard M. and Isabel P. Furlaud
Professor, and Senior Physician,
Rockefeller University, New York

John C. Kapeghian, PhD 

Preclinical development and toxicology

President Preclinical Safety Associates;
Senior positions with Ciba-Geigy, Charles
River Labs and their Navigator Group

Raymond E. Dagger, PhD

Chemistry and formulation

President, Stonehedge Pharmaceutical
Consulting; Senior positions PPD, Sequoia
Pharmaceuticals, Eisai Research Institute

Ulrich Granzer, PhD

Regulatory affairs

Founder Granzer Regulatory Consulting;
Senior regulatory management positions
at Knoll, Bayer, Glaxo-Wellcome

Issi Rozen, MBA

Business Development

Chief Business Officer, Broad Institute;
Senior management positions at Resolvyx
Pharmaceuticals, EMD Serono

Donna M. Ferriero, MD MS

Distinguished Professor

Departments of Pediatrics and Neurology
UCSF Weill Institute for Neuroscience

John A. Germiller, MD PhD

Assoc. Prof. Pediatric Otorhinolaryngology

Director, Clinical Research, Pediatric
Otorhinolaryngology, The Children’s Hospital
Of Philadelphia, Perelman School of Medicine

Henrik Hagberg, MD PhD

Professor and Physician in Chief

Centre for Perinatal Medicine and Health
Institute for Clinical Sciences,
Sahlgrenska Academy, Gothenburg University

Ann Hellström, MD PhD

Professor in Pediatric Ophthalmology

Queen Silvia Hospital,

Sahlgrenska Academy, Gothenburg University

Anne G. M. Schilder, MD

Professor Paediatric Otorhinolaryngology

University College London, NIHR Research

Professor and Director evidENT, UCL Ear

Institute

David K. Wallace, MD MPH

Marilyn K. Glick Professor Ophthalmology

Chair Department of Ophthalmology,

Indiana University School of Medicine