RESEARCH AND DRUG DEVELOPMENT
The ADNP Kids Research Foundation believes that collaboration and transparency are key in accelerating treatment research and development in the quest to find viable treatments to help our children with ADNP Syndrome. We intend to keep families informed of the research and development on this page.
This page will be a working document with a living timeline and will be updated as changes are made. Clinical trials are complex but we believe that it is important that families are kept informed. Please check back frequently for updates.
The ADNP Kids Research Foundation believes that collaboration and transparency are key in accelerating treatment research and development in the quest to find viable treatments to help our children with ADNP Syndrome. We intend to keep families informed of the research and development on this page.
This page will be a working document with a living timeline and will be updated as changes are made. Clinical trials are complex but we believe that it is important that families are kept informed. Please check back frequently for updates.
CP201/NAP (also known as Davunetide) - HISTORY:
CP201/NAP is a potential treatment for ADNP Syndrome derived within a peptide of eight amino acids called NAP. This peptide has highly potent neuroprotective activity. It has been used in the past prior to the discovery of ADNP Syndrome. In 2007, Professor Illana Gozes formed Allon Therapeutics where she began and ran large scale clinical trials in both Alzheimer’s and Schizophrenia patients. Unfortunately, those two drug trials washed out in their FDA Phase 3 clinical trial in 2012 and the company dissolved.
In 2015, following the discovery that children with mutations in ADNP have conditions that Dr. Gozes felt could be therapeutically improved with CP201/NAP, Dr. Gozes began conversations with us regarding the use of this peptide in children with ADNP Syndrome, which of course, we have been excited about ever since.
Dr. Gozes recently formed the company Coronis Neurosciences (Coronis) in 2017 and in early 2018 the FDA granted Coronis “Orphan Drug Designation” for CP201.
In February of 2018 Coronis received FDA Orphan Drug Designation status for CP201 for ADNP Syndrome.
In November of 2018, Coronis, Mount Sinai, University of Washington and the ADNP Kids Research Foundation attended a successful PRE-IND meeting at the FDA.
Mid 2019 Mount Sinai and the ADNP Kids Research Foundation, in an effort to jump start the much needed CP201/NAP clinical trial, proposed fully funding and conducting a pilot study to Coronis but an agreement with Coronis was not obtained.
After struggling to raise funding, Coronis begin plans to change the mechanism of delivery from nasal administration to injection to satisfy investors and announced that they intended to perform a small 5-10 patient 2-week injection safety study to be conducted in Germany.
CP201/NAP is a potential treatment for ADNP Syndrome derived within a peptide of eight amino acids called NAP. This peptide has highly potent neuroprotective activity. It has been used in the past prior to the discovery of ADNP Syndrome. In 2007, Professor Illana Gozes formed Allon Therapeutics where she began and ran large scale clinical trials in both Alzheimer’s and Schizophrenia patients. Unfortunately, those two drug trials washed out in their FDA Phase 3 clinical trial in 2012 and the company dissolved.
In 2015, following the discovery that children with mutations in ADNP have conditions that Dr. Gozes felt could be therapeutically improved with CP201/NAP, Dr. Gozes began conversations with us regarding the use of this peptide in children with ADNP Syndrome, which of course, we have been excited about ever since.
Dr. Gozes recently formed the company Coronis Neurosciences (Coronis) in 2017 and in early 2018 the FDA granted Coronis “Orphan Drug Designation” for CP201.
In February of 2018 Coronis received FDA Orphan Drug Designation status for CP201 for ADNP Syndrome.
In November of 2018, Coronis, Mount Sinai, University of Washington and the ADNP Kids Research Foundation attended a successful PRE-IND meeting at the FDA.
Mid 2019 Mount Sinai and the ADNP Kids Research Foundation, in an effort to jump start the much needed CP201/NAP clinical trial, proposed fully funding and conducting a pilot study to Coronis but an agreement with Coronis was not obtained.
After struggling to raise funding, Coronis begin plans to change the mechanism of delivery from nasal administration to injection to satisfy investors and announced that they intended to perform a small 5-10 patient 2-week injection safety study to be conducted in Germany.
CP201/NAP - BREAKING NEWS 11/15/19:
In November, the ADNP Kids Research Foundation obtained the support of NCATS/NIH in our effort to start the CP201/NAP drug trial as soon as possible at Mount Sinai as planned with no more delay. In conversations with the ADNP Kids Research Foundation, NCATS, Coronis Neurosciences and Mount Sinai, the group agreed to move forward with the nasal delivered CP201 drug trial with Mount Sinai as the Sponsor.
In November, the ADNP Kids Research Foundation obtained the support of NCATS/NIH in our effort to start the CP201/NAP drug trial as soon as possible at Mount Sinai as planned with no more delay. In conversations with the ADNP Kids Research Foundation, NCATS, Coronis Neurosciences and Mount Sinai, the group agreed to move forward with the nasal delivered CP201 drug trial with Mount Sinai as the Sponsor.
CP201/NAP - CURRENT INFORMATION:
In the interest of expediting the study initiation, Coronis has agreed to work with the Seaver team at Mount Sinai and grant the use of CP201/NAP peptide for the intranasal administration study. Unfortunately, the Coronis manufacture is no longer in business and Coronis only has the peptide - not formulated or filled. This will be time consuming and will require the Seaver team, with the assistance of NCATS, to send requests for proposal, obtain and manage drug formulation and filling for the study. All of this needs to happen before any drug trial can begin. The team will work diligently to move forward without delay so that we can initiate the CP201/NAP study.
This will include:
*Mount Sinai is interested in expediting the study start date as quickly as possible and will work with the ADNP Kids Research Foundation and families to do everything possible for no more delays.
In the interest of expediting the study initiation, Coronis has agreed to work with the Seaver team at Mount Sinai and grant the use of CP201/NAP peptide for the intranasal administration study. Unfortunately, the Coronis manufacture is no longer in business and Coronis only has the peptide - not formulated or filled. This will be time consuming and will require the Seaver team, with the assistance of NCATS, to send requests for proposal, obtain and manage drug formulation and filling for the study. All of this needs to happen before any drug trial can begin. The team will work diligently to move forward without delay so that we can initiate the CP201/NAP study.
This will include:
- CP201/NAP peptide (intranasal administration) to test safety in pediatric patients with ADNP Syndrome and assess preliminary efficacy signals. This study would be conducted in collaboration with Coronis.
- Drug formulation and filling (to be contracted through a third-party vendor; funding still required) 3-6 months
- Stability testing – 3 months
- IND (Investigation New Drug) package preparation and submission to the FDA 1-3 months
- FDA review – 1 month
- Some of these can be done concurrent and the Seaver team estimates the FDA first-in patient study timeline to start approximately next fall but updates in this timeline could change and we will post the information as we learn them. We truly hope we can help accelerate this start date if possible.
- The study duration is planned as 24-28 weeks. The study design and timeline could change upon regulatory review.
*Mount Sinai is interested in expediting the study start date as quickly as possible and will work with the ADNP Kids Research Foundation and families to do everything possible for no more delays.
KETAMINE - CURRENT INFORMATION:
After investigating and collecting a large amount of clinical data, in early 2019 Sandra Sermone and Matt Davis brought the idea of Ketamine as a potential treatment option for ADNP Syndrome. Dr. Alex Kolevzon and the Seaver team then thoroughly investigated that clinal data which suggests that low dose Ketamine increases ADNP expression and determined that it looks promising as a possible treatment for ADNP Syndrome. Dr. Kolevzon announced at the ADNP Syndrome Family Conference, that Mount Sinai will be moving forward into a clinical trial study for Ketamine.
After investigating and collecting a large amount of clinical data, in early 2019 Sandra Sermone and Matt Davis brought the idea of Ketamine as a potential treatment option for ADNP Syndrome. Dr. Alex Kolevzon and the Seaver team then thoroughly investigated that clinal data which suggests that low dose Ketamine increases ADNP expression and determined that it looks promising as a possible treatment for ADNP Syndrome. Dr. Kolevzon announced at the ADNP Syndrome Family Conference, that Mount Sinai will be moving forward into a clinical trial study for Ketamine.
- This will involve testing safety in pediatric patients with ADNP Syndrome and assess preliminary efficacy signals.
- This study would be independent of the CP201 study and the Seaver team is currently working on the protocol and regulatory submission of Ketamine as a repurposed drug for treatment with the FDA.
- Dr. Kolevzon plans on submitting to the FDA by the end of the year, with a couple month turn around time with the FDA, and hopes to start the study by March of 2020.
- If the FDA comes back sooner, the trial can start sooner.
- The study duration is planned as 8-12 weeks. The study design and timeline could change upon regulatory review.
ADDITIONAL INFORMATION:
In regards to these study proposals, timelines and cost for clinical trials; the Seaver team is still working on the clinical protocols and will share more detailed information as soon as possible and we will update this page when we receive that information.
In general, they intend to enroll 20-30 patients in each placebo-controlled study. Each patient will ultimately receive the study drug either as part of the cross-over design or in the open-label extension.
In regards to these study proposals, timelines and cost for clinical trials; the Seaver team is still working on the clinical protocols and will share more detailed information as soon as possible and we will update this page when we receive that information.
In general, they intend to enroll 20-30 patients in each placebo-controlled study. Each patient will ultimately receive the study drug either as part of the cross-over design or in the open-label extension.
COST:
It is difficult to estimate the overall cost of drug development through regulatory approval for any of these treatments until the Seaver team has preliminary efficacy signal in ADNP children. Depending on a multitude of factors, the path to approval could differ as well as the cost. The Seaver team will provide cost estimates for the studies described above as soon as the design and timelines are finalized. When we receive those estimates we will update that information on this page.
It is difficult to estimate the overall cost of drug development through regulatory approval for any of these treatments until the Seaver team has preliminary efficacy signal in ADNP children. Depending on a multitude of factors, the path to approval could differ as well as the cost. The Seaver team will provide cost estimates for the studies described above as soon as the design and timelines are finalized. When we receive those estimates we will update that information on this page.
*The ADNP Kids Research Foundation's number one priority will be to work with Mount Sinai and NCATS to support these very important studies and to fund raise for research and clinical trial expenses. We are a grassroots nonprofit organization ran by 100% volunteers and we need support, prayers and as much fundraising as possible in order to help fund our mission to support those living with ADNP Syndrome by spreading awareness, advocating, educating and supporting research for treatments and a cure. BELIEVE THERE IS HOPE FOR A CURE! #CureADNP