Addressing Critical Patient Needs

PRX-P4-003 represents a thoughtfully designed therapeutic approach targeting two significant areas of unmet medical need: ADHD and apathy in Alzheimer's Disease. Our development strategy focuses on delivering meaningful improvements in patient care through innovative solutions that address fundamental limitations of current treatments. With its unique mechanism of action and patient-centric design features, PRX-P4-003 is positioned to potentially transform treatment paradigms in both indications.

Addressing Treatment Needs in Global ADHD Care

ADHD affects approximately 4.4% of the population worldwide, with more adults today than children receiving diagnosis¹¹. While current stimulant medications are effective, they present significant challenges that PRX-P4-003 aims to address:

Overview of ADHD Global Patient Population and Treatment Gaps

ADHD represents a significant global health challenge, affecting millions of individuals across all age groups. Despite available FDA-approved treatments, substantial unmet needs persist, highlighting the opportunity for improved therapeutic options:

Clinical Impact and Disease Burden

ADHD imposes substantial burdens on individuals, healthcare systems, and society:

• Annual economic burden in the U.S. estimated between $143-266 billion
• Additional family-related costs of $33-43 billion from spillover effects
• Ranked 84th among causes of global disability-adjusted life years (DALYs)
• Significantly impacts academic performance and occupational success
• Affects interpersonal relationships and social functioning
• Associated with higher rates of comorbid mental health conditions
• May lead to reduced life expectancy in adults
• Increases risk of substance misuse

Economic costs encompass healthcare services, productivity losses, educational expenses, and significant family-related burden.

Current Treatment Landscape and Unmet Needs

Despite available treatments, significant challenges persist that impact patient outcomes and quality of life:

• Suboptimal Symptom Control: Many patients continue to experience functional impairments despite treatment
• Duration of Action: Limited effectiveness periods create gaps in symptom control
• Side Effects and Tolerability: Common adverse effects lead to treatment discontinuation
• Access Barriers: Schedule II status of first-line stimulant therapies creates significant hurdles in treatment access, compounded by DEA manufacturing quota restrictions leading to drug shortages; additional geographic and socioeconomic disparities further limit care access
• Healthcare Disparities: Underdiagnosis and undertreatment in underserved regions

PRX-P4-003: Addressing Critical Treatment Gaps

PRX-P4-003 represents a differentiated approach to ADHD treatment through several key innovations:

• Balanced Mechanism: Unique pharmacological profile bridging the gap between reuptake inhibitors (methylphenidate) and releasing agents (amphetamines), offering potential for optimized efficacy
• Enhanced Safety Profile: Built on fencamfamine's foundation of well-tolerated clinical use, with additional safeguards through innovative prodrug design
• Reduced Abuse Potential: Prodrug design prevents common routes of misuse (snorting, injection), supporting potential Schedule IV classification with lower regulatory restrictions compared to Schedule II stimulants
• Extended Duration: Once-daily dosing providing ~15 hours of consistent symptom control, supporting improved daily functioning and treatment compliance

Strategic Market Position

PRX-P4-003's differentiated profile, particularly its balanced mechanism and built-in abuse deterrence, positions it to address significant unmet needs in ADHD treatment while maintaining the benefits of extended duration. This innovative approach could help transform ADHD treatment globally, especially in regions where current stimulant options face regulatory or access limitations due to scheduling restrictions.