Expanded Access Policy

The development of new drugs involves a long process of clinical trials and interactions with the Food and Drug Administration (FDA) in the United States, with the European Medicines Agency (EMA) in the EU, and with other regulatory bodies around the world. Patients with serious diseases may seek access to investigational medicines outside of this process.

This policy describes how Leal Therapeutics evaluates and responds to requests for expanded access to its investigational products outside a clinical trial. Expanded access may also be called compassionate use, early access, named-patient access, or other terms depending on the country.

Leal Therapeutics is committed to developing new therapies for diseases with high unmet needs, based on rigorous science, novel approaches, and most importantly, a commitment to patient safety and well-being. Our programs are still considered investigational, and none of our products has been approved by health authorities as safe or effective for use in humans. Our energy and resources are currently devoted to conducting clinical trials that are carefully designed to evaluate the safety and efficacy of our investigational products, and our goal is to follow the most efficient path to meet requirements from FDA, EMA, and other regulatory agencies around the world. We believe that this approach is the best way for us to provide regulatory authorities with the information they need to determine if our products are safe and effective. If you feel that you might be a candidate for one of our clinical trials, please check here for trials that are currently enrolling. This website may also provide contact information for the sites where the trials are being conducted.

At present, Leal does not operate a broad expanded-access program. We may consider individual-patient expanded-access requests in limited circumstances, subject to the criteria in this policy and the availability of products and operational resources.

Who may be considered
Submission of a request does not create an obligation for Leal to provide an Investigational Product. Leal may decline a request at its sole discretion, including when the criteria in this policy are not met or when product supply, manufacturing, safety, operational, clinical development, or regulatory considerations do not permit access.

A request may be considered when all of the following general conditions are met:

• The patient has a serious or immediately life-threatening disease or condition.
• The patient has no comparable or satisfactory treatment alternative.
• The patient is not eligible for, or cannot reasonably participate in, an appropriate ongoing clinical trial—for example, because there are no trial sites accessible to a patient.
• The potential benefit of treatment justifies the potential risks for the individual patient, and those risks are not unreasonable in the context of the patient’s disease or condition.
• The patient meets applicable medical eligibility and safety-monitoring requirements.
• Providing an investigational product will not compromise the conduct, enrollment, completion, or regulatory development of our clinical trials.
• Adequate investigational product supply, manufacturing capacity, clinical oversight, and operational support are available.

Before treatment begins, all applicable regulatory, ethics committee or institutional review board, informed-consent, importation, pharmacy, and other local requirements must be satisfied. Requirements vary by country and by the nature of the request.

Leal may establish additional product-specific eligibility and safety requirements based on emerging safety findings, route of administration, laboratory parameters, respiratory status, concomitant medications, or other relevant clinical factors.

How to submit a request
Expanded access requests must be submitted by the patient’s treating physician. Patients, caregivers, and advocates who want to submit an expanded access request should ask their treating physician to contact us.

Please send requests to:

Medical Information / Expanded Access Contact
Email: clinical.trials@LealTx.com

The treating physician should include sufficient supporting detail to help us evaluate whether the request is consistent with the conditions above. The treating physician should also include contact information to enable us to follow up with the physician directly. Please do not include personally identifiable patient information in an initial email.

Timing of our response
Leal aims to acknowledge receipt of requests promptly, generally within five business days. Leal will make reasonable efforts to provide a substantive response or a status update after receiving the information needed for review. Timing will depend on the completeness and urgency of the request, product availability, safety considerations, regulatory requirements, and the need for medical, operational, manufacturing, or legal review.

Acknowledgement of a request does not indicate that Leal will provide the investigational product.

For time-sensitive requests, the treating physician should clearly indicate the clinical urgency and provide a direct telephone number for prompt follow-up.

Requests involving potential emergency expanded access will be assessed on a case-by-case basis and remain subject to applicable regulatory and institutional requirements. Leal cannot guarantee product availability or authorization, even in emergency situations.

How we evaluate requests
Each request is evaluated by qualified medical, safety, regulatory, clinical operations, and supply-chain personnel, as appropriate. We may consider:

• The patient’s diagnosis, disease severity, clinical history, and current condition
• Known and potential risks of the requested investigational product
• The available clinical and nonclinical evidence for the requested investigational product
• Whether a suitable clinical trial is available and feasible
• Whether the patient can meet necessary monitoring and follow-up requirements
• The ability of the treating physician and institution to administer the product and report safety information
• Product supply and manufacturing capacity limitations
• Potential impact on ongoing clinical development

A decision not to provide expanded access does not reflect a judgment about the patient’s worthiness or the seriousness of their condition. It may reflect safety, supply, scientific, operational, or regulatory considerations.