Neurona Therapeutics Expanded Access Program

Neurona Therapeutics (Neurona) is dedicated to developing cellular therapies that have a positive impact on patient health for serious or life-threatening illnesses/conditions of the central nervous system. Consistent with the company mission, Neurona’s development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational products, and to obtain regulatory approvals to make the therapies available broadly to patients as quickly as possible. Neurona is privileged to collaborate with both clinical investigators and patients who participate in Neurona’s clinical studies to develop new, safe, and effective therapies. Neurona believes this is the best approach to combine strong scientific, legal, and ethical principles to the evaluation of investigational cellular therapies to serve patients with serious or life-threatening illnesses/conditions. 

Expanded Access Program (also referred to as “compassionate use”, “early access”, or “Right to Try”) allows possible access to investigational treatments outside of clinical trials while the investigational product is still under development. Neurona Therapeutics is developing allogeneic inhibitory interneurons, a cell type found in the central nervous system that dampens the excitability of the nervous system, for patients with drug-resistant epilepsy. Consistent with our commitment to bring innovative, safe, and effective products to patients, participation in one of our clinical trials is the best and preferred route to access these investigational products. Neurona does not currently have any active Expanded Access protocols and does not anticipate providing expanded access to the investigational therapies under development until clinical trials indicate that the therapy has benefit to patients with acceptable safety. We encourage patients and their healthcare providers to discuss if participation in one of our planned or ongoing clinical trials is appropriate. Patients, families, caregivers, healthcare professionals, and patient advocacy organizations may find information about our clinical studies on www.clinicaltrials.gov, consistent with FDA regulations, and ex-US clinical trial registries for global trials.

Treating physicians may request information about Neurona’s Expanded Access policy by contacting our clinical trials team by sending an email using the earlyaccess@neuronatx.com address. Neurona will respond to inquiries within 5 business days from receipt.

Although we currently do not have an expanded access program, all requests will be evaluated on a case-by-case basis in a fair and equitable manner. In the future if our policy changes, the evaluation will consider availability of alternative treatments, the potential risks and benefits of the investigational product to the patient, adequacy of investigational product supply, and the potential interference of the decision with the ongoing clinical trials.

As a part of our strong commitment to patients and our focus on developing transformative cellular therapies for illnesses/conditions of the central nervous system, Neurona will regularly review this Expanded Access Policy and make revisions as appropriate, in accordance with the 21st Century Cures Act of 2016.