Advancing Rare Disease Through Disciplined Execution

The Structural Gap Between Discovery and Delivery

The UK and Europe are home to world-class biomedical science and clinical expertise. Yet promising rare and orphan-designated assets frequently stall prior to meaningful patient access.

The causes are structural rather than scientific:

• Long development timelines before value inflection

• Capital intensity ahead of mid-stage clinical data

• Fragmented funding pathways

• Misalignment between patient urgency and traditional biotech financing cycles

For rare disease patients, time is not theoretical. Development efficiency matters.

A Capital-Efficient Development Model

Ambrose Healthcare was established to address this execution gap. Our strategy focuses on:

• Advancing late-stage and clinically de-risked assets

• Structured Phase 2 progression designed around clear inflection milestones

• Leveraging appropriate early-access pathways, including Named Patient Supply where suitable

• Aligning long-horizon capital with disciplined operational delivery

This approach is being applied across targeted programmes addressing significant unmet need, including:

• Gastrointestinal manifestations of cystic fibrosis, where symptom burden remains substantial despite progress in CFTR modulation

• Oncology indications with orphan characteristics, where defined patient subsets may benefit from mechanistically rational therapeutic approaches

These programmes exemplify our philosophy: select assets where clinical rationale is established, development pathways are definable, and execution discipline can accelerate meaningful progress.

Early Access and Responsible Acceleration

For certain rare and orphan conditions, regulatory frameworks allow structured early-access pathways under appropriate governance.

When clinically appropriate, we evaluate mechanisms such as Named Patient Supply to:

• Enable responsible patient access

• Generate real-world insight

• Support sustainable development economics

Such pathways do not replace rigorous clinical development. They complement it — when applied with regulatory integrity and operational control.

Building a UK Rare Disease Ecosystem

Rare disease development requires collaboration. Ambrose is committed to contributing to a scalable UK rare disease ecosystem connecting:

• Clinical centres of excellence

• Translational research institutions

• Regulatory expertise

• Long-term, impact-aligned capital

The UK possesses the scientific depth and regulatory maturity to lead globally in rare disease delivery. What is required is alignment, coordination and disciplined capital deployment.

Rare Disease Day is an opportunity to reinforce that ambition.

A Long-Term Commitment: on Rare Disease Day 2026, Ambrose Healthcare reaffirms its mission

Behind every rare condition is a family navigating uncertainty and constrained options. Rare disease development requires patience, structure and sustained commitment.

To advance rare and orphan therapies through capital-efficient, clinically rigorous development and to help build a world-class rare disease platform anchored in UK expertise.

We welcome dialogue with clinicians, partners and long-horizon investors aligned to structured rare disease impact.