ONC201 EXPANDED ACCESS - FREQUENTLY ASKED QUESTIONS

  • When will the ONC201 Expanded Access Program open?

The Expanded Access program has been made available to select hospitals across the United States, though accrual is not yet open to patients. A list of centers that are in the process of initiating the program can be found on clinicaltrials.gov along with their contact information: click here. Patients and their families can reach out directly to these centers to discuss treatment options and enrollment timelines, as enrollment activities must be completed by the treating physician and their respective institutions in collaboration with Oncoceutics before a patient can be treated.

  • How do patients apply to the ONC201 Expanded Access Program?

Requests for Expanded Access need to be submitted by a U.S. based physician familiar with the patient’s medical history. Families and patients can reach out directly to participating hospitals to evaluate clinical trial options. Contact information for these centers can be found on clinicaltrials.gov: click here.

  • Can doctors from any hospital apply for a patient to be accepted into the ONC201 Expanded Access Program? Or, do we need to coordinate with certain doctors/sites to apply?

Oncoceutics is working with select hospitals that are capable of supporting the special requirements on this program to provide access to ONC201. As a result, not all clinical centers are able to participate in the program, and the quickest way to access ONC201 is typically by referral to a center that is already participating in the program. A list of participating hospitals can be found on clinicaltrials.gov: click here.

  • How can I find out if a patient qualifies for the ONC201 Expanded Access Program?

Eligibility criteria and a list of participating hospitals for all of Oncoceutics’ clinical trials and for the Expanded Access program are available on clinicaltrials.gov: click here. Eligibility for a specific patient is always determined by the treating physician in tandem with Oncoceutics’ medical team. Please reach out directly to a participating hospital to determine eligibility.

  • Is there an age requirement to participate in the ONC201 Expanded Access Program?

Patients two and older are eligible for the Expanded Access program.

  • Is the patient required to have a biopsy to be eligible for the ONC201 Expanded Access Program? Does the patient need to have the H3K27M mutation to be eligible for the ONC201 Expanded Access Program?

H3 K27M status does not have to be known for DIPG patients to be eligible for the ONC201 Expanded Access program, and therefore a biopsy is not required for these patients.

For patients who have a glioma diagnosis other than DIPG, the H3 K27M mutation needs to be known prior to enrollment. The presence of the mutation may be demonstrated by a tumor or liquid biopsy to be eligible for the ONC201 Expanded Access program.

  • Does the ONC201 Expanded Access Program include newly-diagnosed patients? Does it include patients in progression?

The ONC201 Expanded Access program allows DIPG patients to start ONC201 as soon as 14 days after completion of radiation therapy, regardless of whether their disease has progressed or not. Patients who have a glioma diagnosis other than DIPG who are otherwise are eligible for the ONC201 Expanded Access program may start ONC201 after completion of radiation and after disease progression.

  • Is there a required washout period from other therapies before starting the ONC201 Expanded Access Program?

 Yes, there is a washout period from other therapies required before starting the ONC201 Expanded Access Program. The length of the washout period depends on the specific prior therapy and varies according to how long they last in the body. Please consult your treating physician on the washout period that will be needed based on your current or prior treatments.

 What is the cost to the patient for the ONC201 Expanded Access Program?

Oncoceutics provides ONC201 free of charge to participating hospitals, and hospitals are not allowed to charge for ONC201 itself. However, the cost of medical care for the patient may vary, depending on insurance and the local hospital. Please contact one of the participating centers to discuss the cost you may incur from your care provider.

  • Is information available on the safety and effectiveness of ONC201?

ONC201 is in clinical trials that are evaluating its safety and efficacy. ONC201 is not approved by the FDA. Available clinical results with ONC201 have been published at conferences and medical journals that can be accessed online for further information:  https://oncoceutics.com/publications/

  • Our family is located outside of the United States. Can we apply for the ONC201 Expanded Access Program?

Oncoceutics only distributes ONC201 to U.S.-based hospitals participating in clinical trials or the Expanded Access program. Oncoceutics does not provide ONC201 outside of these programs. International patients are eligible for the Expanded Access program as long as they can be treated at one of the participating U.S.-based hospitals. All applications for the EAP need to be submitted by a U.S. based physician.

  • I have heard there is ONC201 available outside the US. Is that the same drug as what is available through the ONC201 Expanded Access Program in the US?

Oncoceutics only distributes ONC201 to U.S.-based hospitals participating in clinical trials or the Expanded Access program. Oncoceutics does not provide ONC201 outside of these programs and cannot comment on compounds claiming to be ONC201 that are distributed by other organizations. We are aware of claims by third parties about substances that they call ONC201. When we are able to find contact information for third parties claiming to sell ONC201, we request that those parties stop selling those products and stop using the name ONC201 to describe any product other than drug that has been manufactured for Oncoceutics by our suppliers in the United States.

  • Can I apply for a spot for a patient in the ONC201 Expanded Access Program now so we can ensure they get accepted when it opens?

The EAP does not allow holding a spot in advance. We recommend speaking with your treating physician about the possibility of participating in the ONC201 Expanded Access Program and how to make arrangements to be referred to a clinical center that is participating in the program. A list of hospitals that are participating in the Expanded Access Program can be found on clinicaltrials.gov. Applications for the Expanded Access program need to be submitted a U.S.-based physician and eligibility for the program needs to be determined by the treating physician and the Oncoceutics medical team.