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GENERATION
PHARMACEUTICALS

A clinical-stage drug development company advancing first-in-class, orally bioavailable vasopressin 1a receptor (V1aR) antagonists for stress-related CNS disorders, traumatic brain injury (TBI), and neurodevelopmental diseases

Neuropsychiatric & Neurodevelopmental Disorders

Pathological stress and fear responses are central drivers of symptoms across neuropsychiatric and neurodevelopmental conditions. Dysregulated arginine vasopressin (AVP) signaling within fear-related neural circuits contributes to aggression, anxiety, emotional dysregulation, and impaired social behavior.

Azevan’s selective vasopressin 1a receptor (V1aR) antagonists are designed to modulate these circuits directly, offering a targeted, mechanism-based approach to symptom control without broad CNS suppression.

Traumatic Brain Injury

Traumatic brain injury triggers an injury cascade over hours to days following the initial insult. This cascade includes blood–brain barrier disruption and dysregulated arginine vasopressin (AVP) signaling, resulting in neuronal damage and cell death, post-concussion syndrome, and increased long-term risk of neurodegenerative disease.

Our focus is on mild TBI, which accounts for 90% of TBI cases annually. Vasopressin 1a receptor (V1aR) antagonist treatment during the acute period after injury represents a well-rationalized, mechanism-based, injury-modifying approach to TBI. By attenuating AVP-driven secondary injury pathways, Azevan’s selective, orally bioavailable V1aR antagonists are designed to minimize neuronal damage, prevent post-concussion syndrome, reduce neuroinflammation, and limit the pathological processes that drive chronic neurological issues following brain injury.

Markets

The Company’s development strategy targets indications with significant unmet medical need, including rare diseases, where approved therapies are absent or offer limited clinical benefit. Clinical validation in these settings supports expansion into larger indications, including neurodegenerative diseases and additional neurodevelopmental disorders.

Neuropsychiatric Symptoms in Huntington’s Disease.

Anger and aggression are commonly observed in a high percentage of patients with Huntington’s disease. These neuropsychiatric symptoms are a leading cause of institutionalization and significantly impact patients, families, and caregivers. Existing pharmacologic approaches are off label, have limited efficacy, and carry meaningful side effect risks. There are no approved treatments. The underlying fear/threat-response circuitry implicated in these symptoms is also dysregulated across other neurodegenerative disorders, supporting the potential for broader application in diseases such as Alzheimer’s disease.

Traumatic Brain Injury (TBI)

The leading cause of death and disability in adults under 35 and the elderly, TBI is a growing global problem and there are no approved drug treatments. Vasopressin antagonists are a novel mechanism for preventing or reducing the cerebral edema that underlies the poor prognosis and outcomes in TBI.

Fragile X Syndrome

Anxiety and aggression are prominent and persistent features of Fragile X syndrome and are major contributors to impaired functioning across developmental stages. These symptoms interfere with learning, social interaction, and daily activities, creating substantial and ongoing burden for individuals with Fragile X syndrome, their families, and caregivers. There are no approved treatments and currently used therapeutics are off-label and often limited by tolerability concerns. Clinical success in Fragile X syndrome may support broader relevance to autism spectrum disorder.

Azevan Facts

  • Anger/Aggression in Huntington’s Disease: Phase 2 Clinical Trial

    The National Institute of Neurological Diseases and Stroke awarded the Company a major grant to conduct a Phase 2 trial with SRX246 in Huntington's Disease patients exhibiting irritability and aggression, a major neuropsychiatric symptom and a leading cause of institutionalization. The trial was completed in collaboration with the NINDS NeuroNext network across 22 sites nationwide. The primary and secondary endpoints were met and the exploratory efficacy endpoints demonstrating clinical benefit. SRX246 was granted an Orphan Designation based on these results.

  • Intermittent Explosive Disorder Phase II Clinical Trial

    A Phase 2 clinical trial with SRX246 for the treatment of Intermittent Explosive Disorder was completed. The results showed excellent tolerability and safety with clinical benefit observed on multiple scales, including a reduction in severe aggressive episodes and lessened disability due to the disorder.

  • Fragile X Syndrome Phase 2 Trial: Treating Anxiety and Aggression

    The Department of Defense recently awarded the Company a grant to test the effect of SRX246 on anxiety and aggression in persons with Fragile X Syndrome. The study will start enrolling participants in late Q1/early Q2 and is being conducted in collaboration with leading investigators in the field at 3 sites, Rush University, University of Cincinnati Children’s Hospital, and University of California-Davis. The design will include home visits to allow participation by individuals previously unable to participate in clinical trials. An orphan designation is in process for use in Fragile X Syndrome.