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10-2012-313

Pioneering Protein Replacement Therapy for ADNP Syndrome and Neurodevelopmental Disorders

UNMET NEED

ADNP, discovered by Professor Gozes, is subject to “spontaneous” mutations resulting in the ADNP syndrome (debilitating outcomes, developmental delays, motor and cognitive dysfunctions, autistic traits). ADNP is a leading gene mutated in autism, interacting and regulating hundreds of other autism genes. To-date, there is no autism disease modifying drug

OUR SOLUTION
• ADNP, essential for brain formation and function. Smallest active fragment of ADNP (NAP) showed clinical efficacy in schizophrenia and mild cognitive impairments patients (preceding Alzheimer’s disease) coupled to a high safety profile
• In two world leading unique mouse models, Adnp+/- and genome-edited ADNP mutated mouse recapitulating the most abundant ADNP mutation NAP corrects ADNP-related deficits, in a precise mechanism of action, which is at the core of development and degeneration
• Short term NAP mouse treatment corrects electrophysiological damage mimicking the human condition and providing an innovative biomarker
• NAP activities in cellular and animal models mimics activities in patient-derived cells, providing biochemical biomarkers

• Clinical-Stage Status:
• GMP Ready Batch for Fill and Finish and Clinical Trials
• Selected Investigator-initiated clinical sites and patient recruitment encompassing reasonable costs.
• NAP received orphan drug and rare pediatric disease designation to treat the ADNP syndrome (FDA), meaning that upon planned successful clinical trial, a tradeable priority review voucher will be awarded.
• Regulating hundreds of key genes, ADNP is at the intersection of development and aging, NAP success in the ADNP syndrome will allow developments in multiple orphan indications, extending to blockbuster indications.
• The investigational drug was proven to be safe in humans, in Phase I and Phase II/III clinical trials for cognitive impairment, schizophrenia and other indications (>500 patients showing bioavailability and protection of cognitive scores/activities of daily living).
INTELLECTUAL PROPERTY (SELECTED)
7,960,334; 8,618,043; 7,452,867; 8,143,221; 8,377,875; 8,586,548; 9,518,994; 2521919; 10,118,943;
PUBLISHED EFFICACY IN MICE AND HUMANS (SELECTED)

Mutated Tyr mouse mimicking life-long ADNP syndrome reveals novel biomarkers

 

 

Deficits corrected by the ADNP fragment, drug candidate, NAP

PLEASE CITE THIS ARTICLE AS: A DOUBLE-BLIND, PLACEBO-CONTROLLED, ASCENDING-DOSE, RANDOMIZED STUDY TO EVALUATE THE SAFETY, TOLERABILITY AND EFFECTS ON COGNITION OF AL-108 AFTER 12 WEEKS OF INTRANASAL ADMINISTRATION IN SUBJECTS WITH MILD COGNITIVE IMPAIRMENT.
MORIMOTO BH, SCHMECHEL D, HIRMAN J, BLACKWELL A, KEITH J, GOLD M; AL-108-211 STUDY.DEMENT GERIATR COGN DISORD. 2013;35(5-6):325-36. DOI: 10.1159/000348347. EPUB 2013 APR 13.PMID: 23594991 CLINICAL TRIAL.

AL-108 = NAP SHOWING SAFETY AND EFFICACY IN HUMANS, ENHANCING SHORT-TERM MEMORY