Coronis Neurosciences Receives FDA Orphan-Drug Designation for Davunetide.
A Historic Moment in ADNP Syndrome History!
A Historic Moment in ADNP Syndrome History!
February 25, 2018
The ADNP Kids Research Foundation is excited to announce that Coronis Neurosciences has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for CP201 (davunetide). This marks the first ever possible treatment for patients with ADNP syndrome.
The ADNP Kids Research Foundation is excited to announce that Coronis Neurosciences has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for CP201 (davunetide). This marks the first ever possible treatment for patients with ADNP syndrome.
ADNP Syndrome, also known as Helsmoortel-VanDerAa Syndrome, is a rare neurodevelopmental disorder caused by a mutation in the ADNP (Activity Dependent Neuroprotective Protein) gene. This gene affects brain formation and development, as well as brain function. It also affects many other major organs and functions of the body. It can cause the neurological, cardiovascular, endocrine, immune, musculoskeletal and gastrointestinal systems, as well as vision, hearing, growth, feeding and sleep. It can also cause mild to severe delays in intelligence, speech and motor planning, (including gross motor, fine motor and oral motor). It causes behavior disorders such as Autism Spectrum Disorder (ASD) in a substantial proportion of cases. In fact, it is estimated: ADNP to be mutated in at least 0.17% of genetic autism cases, making it one of the most frequent ASD-associated genes known to date.
|
Devunetide is derived from a naturally occurring neuroprotective brain protein known as activity-dependent neuroprotective protein (ADNP). CP201 (devunetide) is a small fragment of Activity Dependent Neuroprotective Protein (ADNP); it is an important nerve cell protecting snippet that enhances ADNP activity. It has been extensively studied in more than 20 cell assays and 25 animal models for autism, neuropsychiatric and neurodegenerative diseases. The developers websites claims that CP201 improves learning and memory, increases nerve cell plasticity, protects the cell scaffold and transport system, and restores the autophagic function. The full description can be found on coronisns.com.
|
“This is a major achievement for Coronis which secures the US market for CP201 for the treatment of ADNP syndrome, one of the three most common genetically dependent neurodevelopmental impairments within the Autism spectrum.” Dr. Eric Messika, President and CEO of Coronis. |
Dr. Eric Messika, President and CEO of Coronis, said “this is a major achievement for Coronis which secures the US market for CP201 for the treatment of ADNP syndrome, one of the three most common genetically dependent neurodevelopmental impairments within the Autism spectrum.”
“This marks a significant step on the road to effective approved treatments for ADNP,” said Sandra Sermone, ADNP-KRF President. “Patients with ADNP syndrome currently do not have one single FDA approved drug for treatment of ADNP syndrome. The ADNP Kids Research Foundation is proud to be working with the team at Coronis on this monumental novel treatment project.”
Based on this granted status Coronis is now planning the US portion of their upcoming ADNP clinical trial. The group plans to perform a prospective observational study in a small group of ADNP children to generate descriptive statistics and inform assumptions for several end-point assessments planned to be used in a subsequent pivotal interventional study. These studies will be performed the Seaver Autism Center at Mount Sinai Hospital in New York City, Directed by Prof. Joseph Buxbaum and the Autism Center at the Seattle Hospital in Washington, directed by Prof. Raphael Bernier.
If you are interested in information and future participation on this and other studies, please fill out the contact information form on our RESEARCH page.
Forward Looking Statements
Statements contained herein, other than those which are strictly statements of historical fact may include forward looking information. Such statements will typically contain words such as "believes", "may", "plans", "will", "estimate", "continue", "anticipates", "intends", "expects", and similar expressions. While forward-looking statements represent management's outlook based on assumptions that management believes are reasonable, forward-looking statements by their nature are subject to known and unknown risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by them.
OFFICIAL PRESS RELEASE
FOR IMMEDIATE RELEASE: February 25, 2018
Ness Ziona, Israel--(February 25, 2018) – Coronis Neurosciences (Coronisns.com) announced today that its lead neuroprotective drug candidate, CP201 (davunetide), has been granted Orphan-Drug Status by the U.S. Food and Drug Administration for the treatment of the activity-dependent neuroprotective protein (ADNP) syndrome in the United States.
Dr. Eric Messika, President and CEO of Coronis, said the FDA designation provides both the scientific validation and the potential market for CP201 (davunetide) as a treatment for the ADNP syndrome as well as other neurodevelopmental as well as degenerative brain diseases with similar pathologies.
“Extending CP201 Davunetide's potential use for ADNP syndrome is a significant business opportunity for Coronis, made even more attractive by achieving Orphan Drug Designation in the U.S one of the world's largest and fast-growing markets for the syndrome" said Messika. Professor Illana Gozes, Chief Scientific Officer of Coronis and the discoverer of ADNP, CP201, at Tel Aviv University has demonstrated a strong scientific and clinical rationale for the potential efficacy of CP201 (davunetide) in the ADNP syndrome.
Professor Gozes said that “ADNP is essential for brain formation and function: No ADNP - No Brain. In mice, half the content of the ADNP protein results in severe cognitive impairments, which in mice, are treatable with nasal CP201 (davunetide) treatment. ADNP mutations in children result in an autism spectrum disorder – ADNP syndrome.”
Professor Gozes added that the mechanism of action of CP201 (davunetide) involves microtubules, the cellular scaffold and transport system, especially important for brain cell connections providing for cognitive, motor and sensory functions, which are partially impaired in the ADNP syndrome.
"We are pleased to have reached this important milestone and look forward to working with the US regulators as we plan the US portion of our upcoming ADNP clinical trial," said Dr. Saar Oz, Coronis’ VP Drug Development.
About the ADNP Syndrome
* ADNP syndrome is a currently untreated newly discovered (Helsmoortel, Kooy, Van Der Aa, 2014) ultra rare neuro-genetic disease caused by de novo (non-inherited) mutations in the ADNP gene
* Symptoms manifest early and include abnormalities in a wide range of sensory, motor and cognitive functions:
About US Orphan Drug Designation
Orphan drug designation in the US is granted to medicinal products intended for the diagnosis, prevention and treatment of life-threatening diseases that affect not more than 200,000 people in the US and for which no effective therapy or only a sub-optimal therapy exists.
US Orphan Drug Designation qualifies Coronis for guidance from the FDA on preparing and executing a clinical trial program that will meet FDA marketing approval requirements, for financial assistance for development costs and reduced regulatory fees, plus seven years of marketing exclusivity if davunetide is eventually approved as a treatment for the ADNP syndrome.
It should be noted that orphan drug designation does not limit a drug to use in less common diseases. The drug may, in parallel or afterwards, be developed for more common diseases.
About CP201 (davunetide)
Davunetide is derived from a naturally occurring neuroprotective brain protein known as activity-dependent neuroprotective protein (ADNP). Professor Gozes laboratory and animal studies at Tel Aviv University, have shown that CP201 (davunetide) improves cognition in a number of disease models through a mechanism believed to involve effects on structures in the brain - known as microtubules which are critical to communication between brain cells and the structure of individual cells.
In 2008, Allon Therapeutics reported Phase IIa clinical trial results showing that davunetide had a statistically significant positive impact on memory function in patients with amnestic mild cognitive impairment (aMCI), a precursor to Alzheimer's disease (AD). The data was presented July 28 and July 30, 2008 to the International Conference on Alzheimer's Disease and Related Disorders (ICAD 2008).
On December 7, 2009, Allon Therapeutics reported Phase IIa clinical trial results showing that davunetide had a statistically significant positive impact on the ability of schizophrenia patients to carry out important activities in their daily lives. These data was presented at the annual meeting of the American College of Neuropsychopharmacology.
About Coronis
Coronis Neurosciences is a specialty bio-pharmaceutical company, focused on the development of innovative therapeutics for neurological, neurodegenerative and neuropsychiatric diseases. Coronis is licensing its core technology from Ramot at Tel Aviv University. For more information about Coronis, please visit the company WEB site www.coronisns.com
Forward Looking Statements
Statements contained herein, other than those which are strictly statements of historical fact may include forward looking information. Such statements will typically contain words such as "believes", "may", "plans", "will", "estimate", "continue", "anticipates", "intends", "expects", and similar expressions. While forward-looking statements represent management's outlook based on assumptions that management believes are reasonable, forward-looking statements by their nature are subject to known and unknown risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by them.
Press Release and Coronis Neurosciences Contact:
Dr. Eric J. Messika
President & CEO
Coronis Neurosciences
eric@coronisns.com
www.coronisns.com
_________________________________________
ADNP-KRF Patient Organization Contact:
Sandra Sermone
President
ADNP Kids Research Foundation
sandra@adnpfoundation.org
www.adnpfoundation.org
Ness Ziona, Israel--(February 25, 2018) – Coronis Neurosciences (Coronisns.com) announced today that its lead neuroprotective drug candidate, CP201 (davunetide), has been granted Orphan-Drug Status by the U.S. Food and Drug Administration for the treatment of the activity-dependent neuroprotective protein (ADNP) syndrome in the United States.
Dr. Eric Messika, President and CEO of Coronis, said the FDA designation provides both the scientific validation and the potential market for CP201 (davunetide) as a treatment for the ADNP syndrome as well as other neurodevelopmental as well as degenerative brain diseases with similar pathologies.
“Extending CP201 Davunetide's potential use for ADNP syndrome is a significant business opportunity for Coronis, made even more attractive by achieving Orphan Drug Designation in the U.S one of the world's largest and fast-growing markets for the syndrome" said Messika. Professor Illana Gozes, Chief Scientific Officer of Coronis and the discoverer of ADNP, CP201, at Tel Aviv University has demonstrated a strong scientific and clinical rationale for the potential efficacy of CP201 (davunetide) in the ADNP syndrome.
Professor Gozes said that “ADNP is essential for brain formation and function: No ADNP - No Brain. In mice, half the content of the ADNP protein results in severe cognitive impairments, which in mice, are treatable with nasal CP201 (davunetide) treatment. ADNP mutations in children result in an autism spectrum disorder – ADNP syndrome.”
Professor Gozes added that the mechanism of action of CP201 (davunetide) involves microtubules, the cellular scaffold and transport system, especially important for brain cell connections providing for cognitive, motor and sensory functions, which are partially impaired in the ADNP syndrome.
"We are pleased to have reached this important milestone and look forward to working with the US regulators as we plan the US portion of our upcoming ADNP clinical trial," said Dr. Saar Oz, Coronis’ VP Drug Development.
About the ADNP Syndrome
* ADNP syndrome is a currently untreated newly discovered (Helsmoortel, Kooy, Van Der Aa, 2014) ultra rare neuro-genetic disease caused by de novo (non-inherited) mutations in the ADNP gene
* Symptoms manifest early and include abnormalities in a wide range of sensory, motor and cognitive functions:
- Intellectual disability
- Severe speech delay
- Motor development delay
- Sensory processing disorder
- Insensitivity to pain
- Sleep disorders
About US Orphan Drug Designation
Orphan drug designation in the US is granted to medicinal products intended for the diagnosis, prevention and treatment of life-threatening diseases that affect not more than 200,000 people in the US and for which no effective therapy or only a sub-optimal therapy exists.
US Orphan Drug Designation qualifies Coronis for guidance from the FDA on preparing and executing a clinical trial program that will meet FDA marketing approval requirements, for financial assistance for development costs and reduced regulatory fees, plus seven years of marketing exclusivity if davunetide is eventually approved as a treatment for the ADNP syndrome.
It should be noted that orphan drug designation does not limit a drug to use in less common diseases. The drug may, in parallel or afterwards, be developed for more common diseases.
About CP201 (davunetide)
Davunetide is derived from a naturally occurring neuroprotective brain protein known as activity-dependent neuroprotective protein (ADNP). Professor Gozes laboratory and animal studies at Tel Aviv University, have shown that CP201 (davunetide) improves cognition in a number of disease models through a mechanism believed to involve effects on structures in the brain - known as microtubules which are critical to communication between brain cells and the structure of individual cells.
In 2008, Allon Therapeutics reported Phase IIa clinical trial results showing that davunetide had a statistically significant positive impact on memory function in patients with amnestic mild cognitive impairment (aMCI), a precursor to Alzheimer's disease (AD). The data was presented July 28 and July 30, 2008 to the International Conference on Alzheimer's Disease and Related Disorders (ICAD 2008).
On December 7, 2009, Allon Therapeutics reported Phase IIa clinical trial results showing that davunetide had a statistically significant positive impact on the ability of schizophrenia patients to carry out important activities in their daily lives. These data was presented at the annual meeting of the American College of Neuropsychopharmacology.
About Coronis
Coronis Neurosciences is a specialty bio-pharmaceutical company, focused on the development of innovative therapeutics for neurological, neurodegenerative and neuropsychiatric diseases. Coronis is licensing its core technology from Ramot at Tel Aviv University. For more information about Coronis, please visit the company WEB site www.coronisns.com
Forward Looking Statements
Statements contained herein, other than those which are strictly statements of historical fact may include forward looking information. Such statements will typically contain words such as "believes", "may", "plans", "will", "estimate", "continue", "anticipates", "intends", "expects", and similar expressions. While forward-looking statements represent management's outlook based on assumptions that management believes are reasonable, forward-looking statements by their nature are subject to known and unknown risks, uncertainties and other factors that may cause the actual results, events or developments to be materially different from any future results, events or developments expressed or implied by them.
Press Release and Coronis Neurosciences Contact:
Dr. Eric J. Messika
President & CEO
Coronis Neurosciences
eric@coronisns.com
www.coronisns.com
_________________________________________
ADNP-KRF Patient Organization Contact:
Sandra Sermone
President
ADNP Kids Research Foundation
sandra@adnpfoundation.org
www.adnpfoundation.org
Coronis NeuroSciences is a specialty pharma company focused on the development of Innovative therapies for Neurocognitive and Neurodegenerative disorders.
