ADNP Kids Research Foundation
  • About Us
    • What is ADNP
    • Our Story
    • Our Mission
    • Our Team >
      • Families >
        • Newly Diagnosed
        • ADNP Patient Registry
        • Contact Registry
        • Worldwide Parent Ambassadors
        • contact us
        • ADNP Family Conference >
          • HIGHLIGHTS- ADNP Syndrome Family Conference
          • TSA Travel Help
          • #CureADNP
    • Medical and Scientific Advisory Board
    • Inclusivity Statement
  • RESEARCH
    • Research Projects
    • Research Strategy
    • Ketamine Trial
    • Ketamine Trial Publication
    • Ketamine Update
    • Path to a cure
    • publications
  • NEWS
    • NET Collaboration Study
    • Ketamine Trial Phase 2 Announcement
    • Mount Sinai Launches First Drug Trial for ADNP Syndrome
    • Seaver Midtown Partnership
    • Biomarker Discovered
    • NEWS - p.Tyr719* Case Study
    • NEWS HIGHLIGHTS- ADNP Syndrome Family Conference
  • ADNP STORE
  • DONATE/SUPPORT
    • Boston Marathon
    • Third Party Fundraising
  • Warrior Fun Run 2022
    • Past Fun Run Pictures
    • Sponsorship Opportunity
  • Link Page
  • New Page
Picture


​RESEARCH AND TREATMENT
DEVELOPMENT
UPDATES


RESEARCH AND DRUG DEVELOPMENT TIMELINE
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.


SCROLL DOWN TO THE BOTTOM OF THE PAGE FOR THE LASTEST UPDATES

NAP-peptide/CP201/Davunetide (NAP) - 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.  

In 2012, following the unfortunate unsuccessful outcomes in both FDA Phase 3 clinical trials, her company dissolved. 
 
In 2015, following the discovery that children with mutations in ADNP have conditions that Dr. Gozes began conversations with us regarding the use of this peptide in children with ADNP Syndrome.
 
In 2017 Dr. Gozes formed the company Coronis Neurosciences (Coronis).
 
In 2018, February - Coronis received FDA Orphan Drug Designation status for CP201 for ADNP Syndrome.
 
In 2018, November - Coronis, Mount Sinai, University of Washington and the ADNP Kids Research Foundation attended a successful PRE-IND meeting at the FDA.
 
In mid 2019 Mount Sinai and the ADNP Kids Research Foundation, in an effort to jump start the much needed 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.  ​

In late 2019 the foundation obtained the support of NCATS/NIH in our effort to start the NAP drug trial as soon as possible at Mount Sinai as planned with no more delay.  In conversations with the foundation, NCATS, Coronis and Mount Sinai, the group agreed to move forward with the nasal administration deliver NAP drug trial with Mount Sinai as the Sponsor.
2019/2020 - In the interest of expediting the study initiation, Coronis agreed to work with the Seaver team at Mount Sinai and grant the use of NAP 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.  
This will include:
  • 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.​

​2020-2021 Coronis Nuerosciences has not transferred any of the peptide to the team at Mount Sinai.  Without any information validated outside of the Gozes lab, the foundation has not been able to provided the needed information for investment/grant applications.  

In April of 2021 the foundation was informed that the University of Tel Aviv Tech Transfer office has pulled the license from Coronis Neurosciences.  Information has been changing weekly, as soon as we hear any validated information we will update. 

The foundation connected the Tel Aviv team to Ultragenyx, a rare disease pharmaceutical company who we believe have the recourses and knowledge, in addition to the trail expertise from Mount Sinai,  to run a successful clinical trail in the fastest period of time.   We are awaiting an update and will post here when one arrives. 

We will add additional information as we learn more.

KETAMINE - CURRENT INFORMATION: 
​
In early 2019 Sandra Sermone and Matt Davis, after investigating and collecting a large amount of clinical data, brought the idea of low dose 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. 

Sermone and Kolevzon announced at the November 2019 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.​

Picture
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.  ​

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.

UPDATE: 1/1/2020

After multiple requests from the foundation and other academics, the Gozez Lab at Tel Aviv University refuses to share their validated ADNP mouse model.  This is highly surprising to all in the research community and rather than waiting, the foundation has just paid Mount Sinai to begin a new mouse model through a company called OZGENE.  One the line is developed, Mount Sinai will confirm/validate the mouse model and then the line will be stored at JAX Labs and shared with ALL collaborative researchers interested in ADNP.  (update summer 2020, due to the pandemic, the project has come to a stop and will start as soon as the lab is back up and running)  

UPDATE: 1/22/2020

Today marks a significant step forward on the road to finding effective treatments for ADNP Syndrome.  The FDA has just given the team at Mount Sinai approval to begin the Phase 2 study on Ketamine.  Press release is attached below and more information should be out in the near future about the cost and trial design.  We enthusiastically welcome the first clinical trial for children with ADNP syndrome. 
PRESS RELEASE:  https://www.adnpfoundation.org/ketamine.html

UPDATE: 2/4/2020

Ketamine Trail Update:  The team at Mount Sinai is hopeful that we will begin the ketamine study by May 1st. The enrollment and visit details will follow as soon as the Mount Sinai IRB is approved.

The Phase 2 study is estimated to cost approximately $130,000, but a hard number will be available soon. As soon as we get the final information from Mount Sinai, we will be launching a global fundraiser. If anyone finds any grant opportunities, please forward the information to admin@adnpfoundation.org.

UPDATE: 5/7/2020

Our team at Mount Sinai just received the IRB approval to start recruiting for the Ketamine study. Very soon we will post information about the study but our team is working from home so we anticipate it taking a week or two. The plan is to open the trial for recruitment while launching a fundraising campaign.

UPDATE: 5/14/2020

Ketamine Trail Update: Enrollment 
​
Enrollment will be posted soon on clinicaltrials.gov - study to begin August 2020 

UPDATE 7/1/2020

Ketamine Trial Update:  "WARRIOR FUN RUN" Fundraiser

The amazing and supportive families of children with ADNP Syndrome participated in our 1st ever virtual fun run, and raised almost $150,000 to fund the Phase 2 ketamine study in one month!
​
Thank you so much to all of the participants and generous donors.  Because of your, the first ever drug trial for ADNP Syndrome will begin!

UPDATE 9/10/2020

We are incredibly happy to announce that the very first ADNP patient has received the drug for our low dose ketamine phase 2 drug trial today at the Seaver Autism Center at Mount Sinai in New York.  The family will come back for 4 weeks for evaluations and then the team will see the next patient.  We are hopeful for a smooth clinical trail that will result in helpful information about the safety and efficacy of low dose ketamine as treatment for ADNP syndrome.

UPDATE 2/1/2021

Ketamine Trial Update:  HALFWAY MARK!

We are halfway through our Ketamine trial at the Seaver Autism Center at Mount Sinai in New York, led by Dr. Alex Kolevzon.  The trial is running smoothly and we would like to recognize and thank the 10 families who have enrolled their children in this very important study.

UPDATE 4/30/2021

ADNP Kids Research Foundation has fully funded an important iPSC study costing $95,000. 
​The study aims to:
  1. Phenotypic characterization of human glutamatergic neurons derived from isogenic ADNP mutation iPSC's.
  2. Compound testing in rescuing morphology deficits of the ADNP-syndrome in vitro models.
  3. Synergistic effects of ketamine and NAP in rescuing morphology deficits in ADNP-syndrome in vitro models.
  4. Validation of ketamine in rescuing morphology deficits in neurons derived from iPSC lines from ADNP-syndrome patients.
Privacy Policy  |  Terms of Services  |  Other Policies  
All rights reserved. No portion of the content of this website may be reproduced in any manner without the written permission of the author/creator.   Any information used from this website must also have credit noted to ADNP Kids Research Foundation / www.adnpfoundation.org. The information on this website is not substitute for personal diagnosis or medical care advise. Families and patients should consult a medically qualified clinician in all matters relating to genetic diagnosis, management and health. Information on ADNP changes is a very fast-moving field and while the information in this guide is believed to be the best available at the time of publication, some facts may later be updated or change. Please see additional disclosures on our PRIVACY POLICY page.
  • About Us
    • What is ADNP
    • Our Story
    • Our Mission
    • Our Team >
      • Families >
        • Newly Diagnosed
        • ADNP Patient Registry
        • Contact Registry
        • Worldwide Parent Ambassadors
        • contact us
        • ADNP Family Conference >
          • HIGHLIGHTS- ADNP Syndrome Family Conference
          • TSA Travel Help
          • #CureADNP
    • Medical and Scientific Advisory Board
    • Inclusivity Statement
  • RESEARCH
    • Research Projects
    • Research Strategy
    • Ketamine Trial
    • Ketamine Trial Publication
    • Ketamine Update
    • Path to a cure
    • publications
  • NEWS
    • NET Collaboration Study
    • Ketamine Trial Phase 2 Announcement
    • Mount Sinai Launches First Drug Trial for ADNP Syndrome
    • Seaver Midtown Partnership
    • Biomarker Discovered
    • NEWS - p.Tyr719* Case Study
    • NEWS HIGHLIGHTS- ADNP Syndrome Family Conference
  • ADNP STORE
  • DONATE/SUPPORT
    • Boston Marathon
    • Third Party Fundraising
  • Warrior Fun Run 2022
    • Past Fun Run Pictures
    • Sponsorship Opportunity
  • Link Page
  • New Page