Medgenics Acquires Phase 2-Ready CNS Program: NFC-1
- Strong Efficacy Signal Established in Genetically Identified Subset of ADHD Patients
- Development will Include Neuropsychiatric Symptoms of 22q11.2 Deletion Syndrome
"This acquisition represents the first of a pipeline of opportunities resulting from our collaboration with the
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
"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,
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
"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.
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About the NFC-1 GREAT trial
The NFC-1 GREAT trial was a 30 patient Phase 1b study in adolescents with ADHD and disruptions in the metabotropic glutamate receptor (mGluR) gene network. The objectives of the study were to evaluate the safety, tolerability, and pharmacokinetics of NFC-1 and to evaluate the effect of NFC-1 on ADHD during four weeks of continuous treatment following one week of placebo therapy. The study was conducted at
Subjects were stratified by mGluR mutations. In addition to several exploratory measures, efficacy was assessed using the Clinical Global Impressions of Severity (CGI-S) and Improvement (CGI-I) scales, and the Vanderbilt Parent Assessment Score. Clinical improvement based on CGI-S, CGI-I scores and
NFC-1 was well tolerated, with no treatment-related serious adverse events reported.
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Medgenics, Inc. John Leamanjohn.firstname.lastname@example.org Brian PiperBrian.email@example.com Stern Investor Relations Beth DelGiacco212-362-1200 Beth@sternir.com