Expanded Access Policy Statement

Policy Overview

OKYO Pharma is committed to developing safe and effective treatments for patients with Neuropathic Corneal Pain. Our primary goal is to make our investigational treatments widely available to patients as quickly as possible through clinical trials and regulatory approval.

Expanded Access Programs

Expanded Access (sometimes called “compassionate use”) refers to the use of an investigational drug outside of a clinical trial by patients with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial or otherwise do not have access to investigational drugs through participation in clinical trials.

FDA Expanded Access Policy for OKYO Pharma’s Urcosimod

OKYO Pharma is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness.

Consistent with OKYO’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe, and effective therapies. We believe this approach will serve patients who could be helped by the treatments we are developing. At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, OKYO will consider providing a requesting physician with pre-approval access to a specific OKYO investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:

• The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available.
• The patient’s enrollment in a clinical trial is not possible.

We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis.

OKYO is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; OKYO may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by OKYO, whose decisions are final.

Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to expandedaccess@okyopharma.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 5 business days after receipt.

Physicians located in the U.S. may find additional information regarding expanded access to investigational products by visiting the FDA website: Expanded Access: Information for Physicians.

For information on OKYO clinical trials, please visit www.clinicaltrials.gov.

Eligibility Criteria:

• Patient Condition: Patients must have been diagnosed with neuropathic corneal pain as confirmed by Confocal Microscopy.
• Medical Necessity: The patient’s condition must be serious or life-threatening, and there must be no satisfactory alternative therapies available.

Investigational Drug:

• Drug Name: urcosimod
• Manufacturer: OKYO Pharma
• Indication: Treatment of neuropathic corneal pain
• Status: Investigational drug not yet approved by the FDA

Application Process:

• Physician Responsibility: The treating physician must submit an inquiry or formal written request to OKYO Pharma via email for expanded access on behalf of the patient. If the request for expanded access is granted by OKYO, the physician must then apply for an expanded access IND (Investigational New Drug) application with the FDA on behalf of the patient.
• Contents of Application: The application should include patient medical records, diagnosis via Confocal Microscopy, rationale for using Urcosimod, and details of the treatment plan.

FDA Review and Approval:

• Evaluation: The FDA will review the application to ensure patient safety, assess the potential benefits and risks of urcosimod, and evaluate whether the patient meets eligibility criteria.
• Approval Criteria: Approval is based on a determination that the potential benefit justifies the potential risks of the investigational treatment.

Treatment and Monitoring:

• Treatment Plan: The treating physician is responsible for administering urcosimod as outlined in the approved treatment plan.
• Monitoring: Ongoing monitoring of the patient’s health and response to treatment is essential. The physician must report any adverse events to the FDA.

Patient Consent:

• Informed Consent: Patients (or their legal guardians) must provide informed consent for participation in the expanded access program, understanding the investigational nature of urcosimod.

Conclusion:

• Duration of Treatment: Access to urcosimod through expanded access continues until the drug is approved for commercial use, the patient withdraws from the program, or other circumstances outlined by the FDA.

• Reporting: OKYO Pharma and the treating physician must comply with FDA reporting requirements regarding patient outcomes and safety.

Disclaimer:

The existence of this expanded access policy does not guarantee that expanded access to an investigational product will be provided to a patient. OKYO Pharma reserves the right to modify this policy at any time.