Autism Spectrum Disorder (ASD) is a neurodevelopmental disorder characterized by restricted, repetitive, and inflexible patterns of behavior, interests, or activities and persistent deficits in social communication and reciprocal social interaction.
Many children with Autism exhibit impaired protein digestion, which may or may not manifest in self-restricted diets. The inability to digest protein affects the availability of essential amino acids in the body. Essential amino acids play a critical role in the expression of several genes important to neurological function and serve as precursors to key neurotransmitters such as serotonin and dopamine.
According to the CDC, the prevalence of Autism has increased from 1 in 150 to 1 in 31 children over the past 20 years. In 2021, the Lancet Commission on the Future of Care and Clinical Research in Autism called for targeting treatment for ages of 0-6 when the neuroplasticity is greatest.
There is currently no FDA approved drug on the market that treats the core symptoms of Autism.
At Curemark, we are working to change that. CM-AT, our lead drug candidate, is designed to enhance protein digestion thereby potentially restoring the pool of essential amino acids in children as young as 3. In 2010, CM-AT received “Fast Track” designation from the U.S. Food and Drug Administration (FDA). We have completed our Phase III, placebo-controlled, randomized clinical trials and continue to advance the program towards commercialization. If approved, CM-AT could represent a crucial evolution in the treatment paradigm by offering a first-line, FDA-approved therapy for children with autism.
Curemark is a clinical stage biopharmaceutical company focused on developing novel therapies for the treatment of neurological and other disorders.
Curemark development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational products.
The purpose of this policy is to describe the Expanded Access Policy for Curemark investigational products.
SCOPE
This policy applies to provision of access to a Curemark investigational product that is not approved for any purpose in the country from which the request is intended to be used. This also includes the time period between regulatory approval of an investigational product and its commercial availability in a country.
POLICY STATEMENTS
Any use of a Curemark investigational product outside of a clinical study in a country must be in accordance with local laws and regulations governing such programs, including Curemark Policies and procedures.
Currently Curemark is not making any of its investigational medicines available on an Expanded Access basis prior to regulatory approval anywhere in the world.
Curemark may reconsider making one or more of its investigational medicines available through an expanded Access program in the future.
As authorized by the 21st Century Cures Act, Curemark may revise this Expanded Access policy at any time. Should Curemark decide to offer Expanded Access in the future it will follow all FDA defined criteria for Expanded Access programs.
The Blüm™ Study enrolled children at over 30 sites around the US.
