Expanded Access Program

Expanded access, sometimes known as compassionate use, may be an option for a limited number of individuals to gain access to an investigational medicine(s) outside of a clinical trial.

Over the course of developing a new investigational medicine(s) for a serious or life-threatening condition, Elevar Therapeutics, Inc. (Elevar) may obtain sufficient clinical information about that investigational medicine to identify a population of patients for whom expanded access may be appropriate. In these cases, Elevar may establish an expanded access program for a cohort, or group, of patients who meet specific eligibility criteria. These programs are managed by Elevar and follow a specific protocol, which is developed in consultation with a regulatory agency for the use of the investigational medicine.

At Elevar, in alignment with our core values, we support expanded access programs in those cases where (i) clinical trials are not an option, (ii) the patient has a serious illness and has exhausted all available options, and (iii) we have the medical and scientific evidence to support that the use of the investigational medicine may provide a benefit to these patients.

In rare cases, and with approval from the FDA, Elevar may, in response to a physician request, provide investigational medicine(s) to an individual patient deemed suitable to receive drug based on Elevar’s and FDA’s criteria. Individual patient expanded access must be managed by the patient’s physician and with strict adherence to FDA rules and guidance. It is the requesting physician’s responsibility to submit all the appropriate regulatory paperwork to the FDA.

Making decisions about expanded access requests is always complex. We strive to make these decisions as ethically and appropriately as possible, considering the patient’s case, the available medical and scientific information about the investigational medicine(s), and the probability and timing of a regulatory approval. Our primary priority is to ensure that expanded access is conducted to ensure we are minimizing the potential risk of harm to patients.

Elevar will consider granting expanded access to an investigational medicine(s) if all of the following criteria are met (as codified in 21 CFR Part 312.305):

• Patient has a serious or life-threatening illness with no comparable or satisfactory alternative therapies
• Patient is ineligible for, or otherwise unable to, participate in a clinical trial
• Patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational medicine and the benefit outweighs the known or anticipated risks
• Providing the investigational medicine for the requested use will not interfere with the initiation, conduct, or completion of active clinical trials
• The patient is either being enrolled as a part of a treatment protocol (“cohort” expanded access) or the FDA has approved the use of the investigational medicine(s) for the individual patient

The investigational medicine(s) made available through expanded access have not yet received regulatory approval; therefore, their potential safety and efficacy have not yet been approved by the US Food and Drug Administration (FDA). Doctors and patients should consider all possible benefits and risks when seeking access to investigational medicine(s) prior to regulatory approval.

Once a regulatory agency approves the investigational medicine for commercial use and the medicine is commercially available, existing expanded access programs for that medicine will be phased out.

We encourage patients to speak with their physician first about their eligibility to enroll in a clinical trial. If a treating physician believes expanded access may be the only option for a patient, the physician should contact Elevar to make a formal request on behalf of the patient. This will enable the physician to work with the Company to determine the best course of action.

The request for access to an Elevar investigational medicine can only be considered if the patient’s treating physician is committed to, and supportive of, the requested treatment.

To find open Cohort Expanded Access programs, search “expanded access programs” on www.clinicaltrials.gov.

For more information from the FDA about expanded access in the U.S., click here.

Physicians seeking help on behalf of their patients may contact Elevar at 1-801-303-7440 x300. Once Company has received the request and required medical information, we will respond within 3 business days.