Expanded Access Policy

X4 Pharmaceuticals is developing novel therapeutics designed to improve immune cell trafficking and reverse immune suppression, which are fundamental to treating cancer and certain rare primary immunodeficiency diseases. We are motivated and committed to developing our agents with the goal of improving and extending patients’ lives. Currently, we are conducting clinical trials to assess the safety and efficacy of our investigational medicines. If both safety and efficacy can be proven, we should be able to obtain the necessary marketing approvals from regulatory authorities to provide patients with broad access to these medicines.

Providing access to our investigational treatments through Compassionate Use or Expanded Access Programs is a complex matter to which we have given a lot of thought. The clinical development process involves controlled testing in humans to ensure both safety and efficacy. Because it is not known during clinical development whether an investigational medicine is safe or effective, compassionate use may present numerous risks for the patient and for the clinical development program. For patients, treatment with an investigational drug with unknown side effects may pose a serious safety risks or provide a false sense that the product will provide benefits or a cure for their disease. For the clinical development program, access to investigational products outside of clinical trials may delay or jeopardize the approval of a new medicine sought by many, by reducing the supply of study drug or adversely affecting the data collection process. It is only through the regulatory review and approval process that we ultimately will help the largest number of patients.

Accordingly, our current focus is on conclusive demonstrations of the safety and efficacy of our agents through clinical trials so that we may obtain regulatory approval and ensure the widespread availability of our medicines to all patients who may benefit from them. As a result, we must limit access to our investigational products to these ongoing and critical clinical trials until they have been completed successfully. Therefore, at this time no investigational products of X4 Pharmaceuticals are available under expanded access programs. We encourage all eligible patients to speak to their physicians about the possibility of enrolling in clinical trials in order to gain access to investigational agents and to also support the development of promising new therapies.

We are pushing forward to develop and deliver innovative therapies to the largest number of patients in the shortest amount of time, and we will continue to focus on our singular goal of changing the course of devastating diseases. We will be re-evaluating this policy periodically.

If you have questions about X4 Pharmaceutical’s Compassionate Use Policy, please contact us at patientinfo@x4pharma.com.

Updated: February 27, 2017