Mount Sinai Grants Exclusive License to Plexcera Therapeutics to Develop Treatments for Farber Disease and Cystic Fibrosis

September 10, 2013 –  Mount Sinai Innovation Partners (MSIP), part of the Icahn School of Medicine at Mount Sinai, has granted an exclusive license to  Plexcera Therapeutics, LLC  to commercially develop recombinant human acid ceramidase (rhAC) to treat diseases caused by genetic or disease-induced deficiencies in the enzyme rhAC.

Mount Sinai is promoting the scientific discoveries of its faculty by facilitating the establishment and supporting incubator companies to accelerate the discovery of treatments for devastating diseases, including those that often affect relatively small populations.

Plexcera was founded by Edward H. Schuchman, PhD, Genetic Disease Foundation – Francis Crick Professor Professor of Genetics and Genomic Sciences at the Icahn School of Medicine, and Ivan Galanin, a pharma industry veteran and advisor to MSIP, in collaboration with QOL Medical LLC, a specialty pharma company focused on rare pediatric diseases.

Two devastating childhood diseases are caused by recessive inherited mutations in the gene encoding rhAC: Farber disease, characterized by severe joint pain, inflammation and arthritis, and, a form of spinal muscular atrophy with epilepsy (SMA-PME), found in adolescents, and characterized by progressive muscle weakness. There is no therapy for either condition. In addition, in cystic fibrosis, excess ceramide accumulates in the lungs. Treatment with inhaled rhAC may address lung cell death, inflammation and susceptibility to infection seen in these patients.

“The name ‘Plexcera’ comes from the concept that rhAC is an enzyme with multiple uses. Farber disease is our first target,” said Dr. Schuchman. “We hope to launch a clinical trial of rhAC within the next 18 months.”

The licensed technology is based on more than 20 years of research conducted by Dr. Schuchman, who will serve as Plexcera’s Chief Scientific Officer, and Erich Gulbins, PhD, from the Center for Medical Biotech at the University of Duisburg-Essen, Germany, who will serve on Plexcera’s Scientific Advisory Board.  Dr. Schuchman has extensive research and development experience with these disorders and enzyme replacement therapy specifically. Dr. Gulbins has identified a central role for excess ceramide accumulation in cystic fibrosis, as well as other pulmonary diseases.

“Mount Sinai has a strong track record of developing breakthrough products for rare diseases.  Dr. Schuchman has worked with key scientific, clinical and industry thought leaders for many years and can call on their expertise and commitment,” said Mr. Galanin, CEO of Plexcera.  “The collaboration with QOL Medical gives us access to key infrastructure components such as clinical, regulatory and manufacturing expertise.”

As part of the license, Mount Sinai received equity in the new company, as well as royalties.  This is the second major license agreement negotiated this year by MSIP in the field of orphan diseases, both originating from research conducted by the Department of Genetic and Genomic Sciences.

About Mount Sinai Innovation Partners
Mount Sinai Innovation Partners (MSIP), part of the Icahn School of Medicine at Mount Sinai, facilitates the transfer of discovery from the laboratory to the marketplace, acting as the interface with commercial entities.

MSIP is responsible for the full spectrum of commercialization activities required to bring the Icahn School of Medicine’s inventions to life. These activities include evaluating, patenting, marketing, and licensing new technologies, while also negotiating agreements for sponsored research, material transfer, and confidentiality. For more information on MSIP, visit: https://www.ip.mountsinai.org.

About Plexcera Therapeutics, LLC
Plexcera Therapeutics was founded with the goal of bringing life-saving therapies to patients who have either a genetic deficiency in the acid ceramidase enzyme or a disease-driven imbalance between the enzyme and its substrate.  The company is headquartered in Vero Beach, FL, with facilities in New York City.  For more information on Plexcera Therapeutics, please visit:  https://www.plexcera.com.

About QOL Medical LLC
QOL Medical LLC, based in Vero Beach, is a specialty biopharmaceutical company focused on pediatric products for genetic and rare orphan diseases.  QOL invests in clinical stage and marketed products and currently markets 2 approved products: Sucraid (sacrosidase) Oral Solution for Congenital Sucrase-Isomaltase Deficiency and Ethamolin (Ethanolamine Oleate) Injection 5% for bleeding esophageal varices.

About The Mount Sinai Medical Center
The Mount Sinai Medical Center encompasses both The Mount Sinai Hospital and Icahn School of Medicine at Mount Sinai. Established in 1968, the Icahn School of Medicine is one of the leading medical schools in the United States, and is noted for innovation in education, biomedical research, clinical care delivery, and local and global community service. It has more than 3,400 faculty in 32 departments and 14 research institutes, and ranks among the top 20 medical schools both in National Institutes of Health (NIH) funding and by U.S. News & World Report.

The Mount Sinai Hospital, founded in 1852, is a 1,171-bed tertiary- and quaternary-care teaching facility and one of the nation’s oldest, largest and most-respected voluntary hospitals. In 2012, U.S. News & World Report ranked The Mount Sinai Hospital 14th on its elite Honor Roll of the nation’s top hospitals based on reputation, safety, and other patient-care factors.

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Media contact:  Mount Sinai Press Office, 212-241-9200, newsmedia@mssm.edu