Our lead program targets impaired executive function in Down syndrome (DS-EF), with expansion potential in ADHD and autism.
Down syndrome (DS) is the most common chromosomal abnormality characterized by three copies of chromosome 21 since birth, with more than 5 million individuals worldwide. A defining and consistently documented challenge for children and adults with Down syndrome is impairment of executive function, yet no therapy dedicated to improving executive function in this population exists.
of individuals with DS have significant EF impairment
experience sleep disturbance, which further worsens EF
have communication impairment, which is closely linked to EF deficits
What executive function is
Executive function (EF) is the brain's control system: it governs working memory, inhibition, cognitive control, planning, and attention. In individuals with DS, these networks are significantly disrupted, driving functional disability across daily life.
Current treatments fall short
All existing options (stimulants, re-uptake inhibitors, α2A agonists, psychotropics) target monoamines, not the underlying prefrontal network dysfunction driving DS-EF. None is specifically designed to stabilize the impaired neuronal firing affecting this population. None improves sleep.
Impaired EF in DS impacts
88% felt they wish for the loved one with Down syndrome to be as independent as possible, and only 6% felt that independence was not a priority.
Down syndrome specialists describe a successful executive-function therapy as a potential blockbuster, and say better executive function is among the very top priorities families ask for.
Sources: de Graaf et al., Genet Med 2017; Eur J Hum Genet 2022; Skotko et al. estimates; Santoro et al., Genet Med 2020; Edgin et al., Neurosci Biobehav Rev 2015; Diamond 2013; Fidler 2005, 2009; Churchill et al., Sleep Med Rev 2015.
Over three decades, co-founder Mario Sanchez systematically observed that compounds stabilizing neuronal firing produced consistent improvements in executive function across DS, ADHD, and autism, while standard neurotransmitter-targeting drugs did not. This hypothesis led directly to identification of AKA1315.
The driver of DS-EF
EF impairment in DS reflects a pattern of unstable prefrontal cortex (PFC) network activity characterized by:
Our mechanism
AKA1315 directly stabilizes neuronal firing via dual-state ion channel modulation (Nav fast and slow inactivation):
AKA1315 is the only drug in development for DS-EF with pilot EF, sleep, and mood data, and the lowest regulatory risk of any active DS program.
AKA1315's mechanism targets the EF network layer shared across multiple neurodevelopmental conditions, establishing a clear path to franchise expansion after DS-EF proof of concept.
Anaka is building the first dedicated DS-EF franchise: a high-need, unserved population with no therapy approved specifically for DS-EF. AKA1315 is a clinical-stage asset with human efficacy signals in Down syndrome, ADHD, and autism, and known and favorable safety.
Development stage
Clinical Stage
Preparing for Phase 1
Expansion areas · shared EF mechanism
Expansion area
ADHD
~15M individuals in US
EF network dysfunction is central to ADHD. Shared mechanistic rationale supports expansion post DS-EF proof of concept.
Expansion area
Autism spectrum
~5.5M individuals in US
EF impairment is a core feature of autism spectrum conditions. Shared neuronal firing instability provides a compelling mechanistic rationale.
Expansion area
Other EF conditions
EF deficits are a shared feature across a range of neurodevelopmental and psychiatric conditions, representing a broad long-term franchise opportunity.
Our team brings together deep Down syndrome expertise, more than 100 years of drug development, and 30+ years of clinical insights across DS, ADHD, and autism -- built specifically to execute in DS and other neurodevelopmental conditions.
President · CEO · Co-founder
Chief Medical Officer · Chief Development Officer · Co-founder
Chief Scientific Officer
Head of Formulation & Manufacturing
Scientific Originator · Co-founder
In memory of Mario Sanchez, who passed in April 2026. His clinical insight is the foundation of this work.
Clinical Prof. of Psychiatry, SUNY Upstate; PI on 400+ CNS & ADHD clinical trials
Distinguished Prof. & Exec. Dir., UCSD Down Syndrome Center; DS neurobiology leader
Chief of Translational Science, Loulou Foundation; ex-Roche DS & autism clinical lead
Pediatric neuropsychiatrist; ex-Autism Speaks & GSK; CMO across multiple CNS biotechs
We welcome conversations with potential investors, partners, and collaborators aligned with our mission to address the significant unmet need of executive function impairment in individuals with Down syndrome.
Lexington, MA · Greater Boston Area
Hampus Hillerstrom
President, CEO, Co-founder