Unlocking the potential of genomic medicine to identify and treat patients with life-altering diseases.
Medgenics is focused on fulfilling the promise of genomic medicine to improve the lives of children and adults with rare and difficult-to-treat diseases.
The company's core capabilities, including its internal research and development and ongoing sponsored research and licensing agreements with a well-respected pediatric academic medical center, endow Medgenics with deep insight into the underlying genetic pathway of disease and guides us in pursuing solutions for patients with life-altering diseases.
Medgenics Acquires Phase 2-Ready CNS Program: NFC-1
PHILADELPHIA (September 9, 2015) – Medgenics, Inc. (NYSE MKT: MDGN) today announced the acquisition of neuroFix Therapeutics, LLC, the developer of NFC-1, a first-in-class, non-stimulant metabotropic glutamate receptor (mGluR) neuromodulator entering Phase 2/3 for the treatment of mGluR network mutation positive Attention Deficit Hyperactivity Disorder (mGluR+ ADHD), as well as neuropsychiatric symptoms resulting from a related rare genetic disorder, 22q11.2 Deletion Syndrome (22q11.2 DS). Hakon Hakonarson, M.D., Ph.D., Professor and Director of the Center for Applied Genomics (CAG) at The Children's Hospital of Philadelphia (CHOP) founded neuroFix to pursue development of NFC-1 following a breakthrough genetic discovery. Medgenics acquired all outstanding shares of neuroFix for upfront consideration of $2 million cash, a series of performance-based milestone payments and sales royalties.
"This acquisition represents the first of a pipeline of opportunities resulting from our collaboration with the Center for Applied Genomics," said Mike Cola, Chief Executive Officer, Medgenics. "Additionally, it expands our company focus beyond gene therapy to broader genomic capabilities, allowing us to identify and treat pediatric patients with serious, unmet medical needs."
A recently completed five-week Phase 1b dose-escalation study in 30 patients of NFC-1 demonstrated strong efficacy signals in several validated ADHD scales in mGluR+ adolescents with ADHD symptoms. The treatment effect of NFC-1 appeared more robust over time and at higher doses. NFC-1 was well tolerated, with no treatment-related serious adverse events reported. Additionally, 20 of the 30 enrolled patients elected to continue in a long-term safety trial in order to maintain access to therapy. The results of the study will be presented at the 2015 American Academy of Child and Adolescent Psychiatry (AACAP) Annual Meeting on October 26-31 in San Antonio, Texas.
"In the NFC-1 GREAT study we observed improvement not only in ADHD symptoms including inattention and hyperactivity, but also in other neurobehavioral symptoms including anxiety, mood and sleep disturbance," said Study Principal Investigator, Josephine Elia, MD, Neuroscience Center, Department of Child and Adolescent Psychiatry, Nemours/Alfred I. duPont Hospital for Children. "These results are meaningful for patients and their families and the data suggest a compelling clinical profile for NFC-1 for these impairing comorbidities."
NFC-1 also has the potential to help the thousands of patients around the world with a rare and severe genetic disorder known as 22q11.2 Deletion Syndrome. Among many other abnormalities, these patients suffer from severe neuropsychiatric disorders. "Surgery can correct the structural abnormalities, such as congenital heart disease and palatal abnormalities, in many of these patients. In contrast, existing therapeutic options for these neuropsychiatric disorders are often inadequate in their effectiveness and may cause unwanted side effects. The discovery that disruptions in the mGluR network underlie these disorders and the promise of NFC-1 to treat them provides new hope for these patients and their families," said Donna M. McDonald-McGinn, MS, CGC, Associate Director of Clinical Genetics and Program Director of the 22q and You Center at CHOP.
"We are elated to have the opportunity to move NFC-1 into Phase 2/3 development. The science underlying this new program allows us to identify the patients who can benefit most with high specificity," stated Dr. Garry Neil, Chief Scientific Officer, Medgenics.