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.

Chemotherapy-induced hearing loss

​

Treatment of childhood cancer is highly successful with cure rates above 80%,

but for many survivors come at a price of life-long ailments due to the side-effects

of chemotherapy. The most devastating is permanent hearing loss with its impact on learning and social skills; up to 50% of children with even minimal hearing loss
may not pass 9th grade. Platinum-based drugs are extremely toxic to the inner ear but preferred in most treatment combi-nations of childhood solid tumors. Up to 70% of children treated with cisplatin 

experience permanent hearing loss and

for at least half of them the loss is severe, poorly compensated for by hearing aids. AP-001, as intended prophylactic treat-ment, is experimentally shown to protect from cisplatin-induced hearing loss. AP-001 will be delivered locally to the ear to not compete with the cancer therapy and can become the first such approved treatment.

Hypoxic-ischemic encephalopathy (HIE) and neonatal stroke

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