Elizaria Named Patient Programme Feasibility Assessment | India A comprehensive evaluation of market access potential and patient demand forecasting for early access pathway implementation

OBJECTIVE Strategic Assessment Framework This assessment evaluates the feasibility and potential demand for Elizaria under a Named Patient Programme (NPP) in India, providing critical insights for both short-term NPP planning and long-term registration strategy development. Core Evaluation Areas Potential patient population sizing and segmentation Annual treatment volume projections across multiple scenarios Key assumptions, data gaps and strategic uncertainties NPP implementation feasibility and resource requirements

BACKGROUND Understanding Named Patient Programmes in India What is an NPP? An early access pathway for unapproved or not- yet-registered medicines, designed specifically for serious, life-threatening or rare diseases where patients face limited treatment alternatives. Target Patient Profile NPPs typically serve severe or refractory patients with demonstrated medical need, those who have exhausted available treatment options, and cases where no suitable therapeutic alternatives exist. Local Agent Role In India, the local agent manages importation and supply handling, coordinates regulatory requirements, facilitates patient-specific distribution, and provides essential medical and pharmacovigilance support. Key insight: NPP demand is fundamentally driven by unmet medical need rather than broad disease prevalence, making patient selection and clinical criteria critical success factors.

Elizaria Clinical Context & Treatment Assumptions The following estimates are based on planning assumptions developed for feasibility assessment purposes. Final parameters will require validation with clinical and regulatory experts. Assumed Product Profile Indicated for a rare, severe or refractory condition requiring specialist intervention Chronic or long-term treatment paradigm with sustained therapy requirements Prescription limited to tertiary or quaternary care centres with appropriate diagnostic capabilities Treatment Setting Characteristics Limited number of eligible hospitals with necessary infrastructure and expertise High disease awareness concentrated amongst key opinion leaders (KOLs) and specialist physicians Centralised diagnosis enabling controlled patient identification and referral pathways Initial Symptoms Patient notices concerning signs Specialist Referral Primary care refers to specialist Confirmed Diagnosis Diagnostic tests confirm condition NPP Access Patient receives product access This patient funnel represents the critical pathway from disease presentation to NPP treatment initiation, highlighting key decision points and potential attrition stages.

EPIDEMIOLOGY Disease Epidemiology Estimates for India 1.4B India Population Total population base for prevalence calculations 1-5 Cases per 100,000 Estimated prevalence range for rare condition 14K-70K Prevalent Patients Total estimated patient population in India Disease Characteristics Based on the assumed rare or orphan disease classification, we estimate prevalence between 1 and 5 cases per 100,000 population. Whilst this represents low prevalence by absolute measures, India's substantial population base amplifies these figures significantly. The resulting prevalent patient population ranges from approximately 14,000 to 70,000 individuals nationwide. However, the actual NPP-eligible subset will be considerably smaller due to diagnosis rates, disease severity criteria, and access constraints. Critical consideration: Large population base transforms even rare diseases into meaningful patient volumes, but access remains highly concentrated in urban tertiary care centres.

NPP-Eligible Patient Pool Analysis Total Prevalent Patients 14,000370,000 nationwide prevalence estimate Diagnosed Patients 30340% diagnosis rate due to awareness and access barriers Severe/Refractory Cases 20330% of diagnosed patients meet severity criteria NPP Eligible 10320% final eligibility after access and affordability screening Resulting NPP Patient Pool Estimate Following this stepwise attrition funnel, we estimate approximately 300 to 2,000 patients annually would be eligible for and able to access Elizaria through the NPP pathway. This represents a highly select patient subset characterised by confirmed severe disease, treatment failure on available alternatives, access to specialist centres, and ability to navigate NPP requirements. NPP access is fundamentally constrained by diagnosis rates, specialist referral pathways, and the concentration of appropriate treatment facilities in major urban centres.

SCENARIO MODELLING Annual Patient Volume Scenarios & Treatment Assumptions 0 800 1,600 2,400 Low Medium High Scenario Framework Three distinct scenarios capture the range of potential NPP demand, reflecting uncertainties in diagnosis rates, specialist referral patterns, and patient access capabilities. Treatment Regimen Assumptions Chronic therapy requiring sustained long-term treatment Standard dosing: 1 treatment unit per patient per month Annual requirement: 12 units per patient per year Average treatment duration: 12 months with minimal discontinuation Volume projections scale directly with treatment duration assumptions. In the NPP setting, discontinuation rates are expected to remain low given careful patient selection and close monitoring protocols.

PROJECTIONS Annual Product Volume Requirements & Strategic Implications Scenario Patients Units per Patient per Year Total Units per Year Low 300 12 3,600 Medium 800 12 9,600 High 2,000 12 24,000 Conclusion & Key Takeaways NPP Feasibility Confirmed Estimated 30032,000 patients annually represents clinically and operationally viable volumes for controlled NPP implementation with manageable supply chain requirements. Volume Requirements Annual product volume ranging from 3,600 to 24,000 units enables appropriate supply planning whilst maintaining programme exclusivity and control. Strategic Value NPP provides critical early patient access, generates valuable real-world evidence, facilitates KOL engagement, and establishes foundation for full registration strategy. Critical next steps: Validate epidemiology assumptions with Indian medical experts, map key tertiary treatment centres nationwide, and initiate early regulatory engagement to establish clear NPP pathways and requirements.