Effective Date: December 16, 2024

1. Introduction

Cambium Bio Limited ("Cambium Bio") is a clinical-stage regenerative medicine company committed to developing innovative biologics for ophthalmology and tissue regeneration applications. Cambium Bio will comply with all applicable laws and regulations related to expanded access programs, including the 21st Century Cures Act and FDA regulations. Our lead product candidate, Elate Ocular®, is an investigational drug currently under development for the treatment of dry eye disease. Elate Ocular® is a topical eye drop derived from human platelets. Dry eye disease affects millions of people worldwide and is characterized by insufficient lubrication and moisture on the surface of the eye. This can lead to discomfort, irritation, and blurred vision. Elate Ocular® is designed to address this unmet medical need by providing a novel therapeutic approach to dry eye disease.  

This Expanded Access Policy (the "Policy") outlines Cambium Bio's approach to evaluating and responding to requests for expanded access to Elate Ocular®. This Policy is intended to meet the requirements of the 21st Century Cures Act and provide clear guidance to healthcare providers and patients seeking access to Elate Ocular® outside of clinical trials.

2. Expanded Access to Elate Ocular®

Expanded access, sometimes referred to as "compassionate use," allows patients with serious or immediately life-threatening diseases or conditions who lack satisfactory therapeutic alternatives to access investigational drugs outside of clinical trials.

While Cambium Bio recognizes the critical need for new treatment options for patients with dry eye disease, we are not currently offering expanded access to Elate Ocular®. This decision is based on several factors:

• Ongoing Clinical Trials: Cambium Bio is currently preparing for registration-enabling clinical trials to assess its safety and efficacy of Elate Ocular® . Our primary focus is on completing these trials to generate the necessary data to support potential regulatory approval and make Elate Ocular® available to a broader patient population.

• Limited Drug Supply: The current supply of Elate Ocular® is limited, and prioritizing its use for clinical trials is essential to ensure the timely completion of these studies.

• Resource Constraints: Managing expanded access programs requires significant resources, including personnel, manufacturing capacity, and regulatory oversight. At this stage of Elate Ocular®'s development, our resources are primarily dedicated to advancing the clinical trial program.

For eligible patients, participation in clinical trials offers several potential benefits, including access to investigational therapies, close monitoring by medical professionals, and the opportunity to contribute to scientific knowledge that may benefit others.  

3. Future Considerations

Cambium Bio will periodically review its expanded access policy for Elate Ocular® as the clinical development program progresses. Factors that may influence future decisions regarding expanded access include:

• Clinical Trial Progress: The availability of Elate Ocular® for expanded access may be considered upon successful completion of key clinical trials and demonstration of a positive benefit-risk profile.

• Drug Supply: Increased manufacturing capacity and drug supply may allow for expanded access in the future.

• Regulatory Guidance: Cambium Bio will continue to monitor and adhere to relevant regulatory guidance on expanded access programs.

4. Alternative Treatment Options

While expanded access to Elate Ocular® is not currently available, we encourage patients with dry eye disease to discuss available treatment options with their healthcare providers. Several approved therapies can effectively manage dry eye symptoms and improve ocular surface health. These may include artificial tears, lubricating ointments, anti-inflammatory medications, and lifestyle modifications.

5. Clinical Trial Information

Information about Elate Ocular® clinical trials can be found on ClinicalTrials.gov. A Phase 1/2 clinical trial has shown promising results in terms of safety and efficacy.  The trial publication can be found here.

6. Contact Information

For inquiries regarding this Expanded Access Policy or Elate Ocular® clinical trials, please contact:

Cambium Bio Limited
16 Goodhope Street
Paddington, New South Wales 2021
Australia
Phone: 1 300 995 098
Email: info@cambium.bio  

7. Conclusion

Cambium Bio is dedicated to advancing the development of Elate Ocular® as a potential new treatment option for dry eye disease. We appreciate the interest in expanded access and will continue to evaluate the feasibility of providing access to Elate Ocular® outside of clinical trials in the future as the clinical development program progresses. We are committed to finding effective treatments to address the needs of patients with dry eye disease.