Access to Investigational Drugs Outside a Clinical Study

Kura Oncology is committed to realizing the promise of precision medicines for the treatment of cancer. To do this, we conduct clinical studies to assess the safety and efficacy of investigational drugs, and the data generated from these studies allows us to obtain the necessary approvals from Health Authorities which allow for broad access to medicines.

At Kura Oncology, we believe that wherever possible, use of an investigational drug for a patient as part of a clinical study is preferable as clinical studies can generate data that may lead to the approval of drugs and, consequently, to wider patient availability.

A medical professional holding the hand of their patient.

However, we also recognize that, when participating in a clinical study is not possible, patients facing serious or life-threatening conditions may seek access to investigational drugs outside of a clinical study setting.

Consistent with applicable laws and regulations, Kura Oncology may provide patients with access to investigational drugs through Expanded Access/Compassionate Use Programs when there is sufficient evidence of the safety profile and clinical activity of the investigational drug to support its use in a particular circumstance. The patient’s physician will perform an assessment as to the patient’s suitability for the treatment.

At this time, Kura Oncology is only providing expanded access to our investigational drugs as follows:

Tipifarnib for eligible adult patients with HRAS mutations or Peripheral T-Cell Lymphoma (PTCL) in the United States as permitted under local laws and regulatory requirements. Additional information regarding access to our investigational drug tipifarnib outside of clinical studies can be found at clinicaltrials.gov (NCT04809233).
Ziftomenib for eligible adult patients with (a) KMT2A-rearranged Acute Lymphoblastic Leukemia (ALL), (b) NPM1-mutant Acute Myeloid Leukemia (AML), or (c) KMT2A-rearranged AML, in each case in the United States as permitted under local laws and regulatory requirements. In the case of patients with KMT2A-rearranged AML, Expanded Access to ziftomenib will only be provided for use in combination with FDA-approved therapies currently under clinical investigation at Kura Oncology (e.g., venetoclax + azacitidine, cytarabine + daunorubicin). Additional information regarding access to our investigational drug ziftomenib outside of clinical studies can be found at www.clinicaltrials.gov (NCT05738538).

Visit Kura Oncology Clinical Trials for specific details on our current clinical studies and, for a more general list of clinical studies currently recruiting patients, please visit www.clinicaltrials.gov.

It is important to note that the terminology used can vary from country to country and be dependent on the way that the drug is accessed. Although often referred to as Expanded Access Programs or Compassionate Use Programs, these types of access can also be known as Early Access, Emergency Use, Individual Patient Expanded Access, Named Patient Program and Treatment Protocols.

Eligibility Criteria

All requests for access to investigational drugs outside of a clinical study will be assessed against the following criteria.

Patient Eligibility Criteria

To be eligible for access to an investigational drug outside of a clinical study, a patient must meet the following criteria:

Diagnosed with a serious or immediately life-threatening disease or condition.
Has exhausted appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
Is ineligible for participation in any ongoing clinical study of the investigational drug, which includes lack of access due to geographic limitations.
Meets any other pertinent medical criteria for access to the investigational drug, as established by Kura Oncology.

Investigational Drug Criteria

In addition to the patient eligibility requirements, the investigational drug must meet the following criteria:

The drug is under investigation in one or more clinical studies.
There is sufficient evidence that the investigational drug will have an acceptable safety profile for the intended patient population.
There is biological rationale for the disease or condition to be treated, coupled with adequate human clinical data to support an assessment that the potential patient benefit justifies the potential risks of the treatment use, and those potential risks are not unreasonable in the context of the disease or condition to be treated.
The provision of the investigational drug will not interfere with or compromise the clinical development of the drug.
Adequate supply of the investigational drug exists to perform necessary clinical studies in addition to providing access to patients who do not have alternative treatment options.

Treating Physician Criteria and Responsibilities

The physician attending to the patient receiving an investigational drug outside of a clinical study must be properly licensed and fully qualified to administer the drug.

The physician must agree in writing to comply with:

Any applicable country-specific legal and regulatory requirements related to providing an investigational drug under expanded access; and,
Any Kura Oncology requirements in terms of medical criteria, safety reporting, drug supply/use, and protection of intellectual property.

How to Request Access:

All requests should be submitted by a medically qualified physician. Please initiate the request by emailing ExpandedAccess@kuraoncology.com. Your request should include the patient diagnosis and treatment history. Do not include any patient identifiers in the request. Kura Oncology is committed to providing a fair and equitable evaluation of all the requests we receive; therefore, all decisions are based on clinical circumstances. Whenever possible, patients will be referred to ongoing clinical studies as the primary way to access investigational drugs. We will review requests on a case-by-case basis, and the fact that our treatment is made available to one patient does not guarantee it will be made available to future patients. We continually evaluate the benefit-risk profile of our investigational drugs and, based on evolving clinical data, we may require additional information from a treating physician to fully evaluate a request. We cannot guarantee that all requests for access will be granted, even when eligibility criteria are met. We regularly monitor the email box that these requests may be submitted to and we will acknowledge each submitted request within one (1) business day after receipt. You can find further contact details on the Contact Us page of our website.

The publication of this policy by Kura Oncology is not a guarantee of access to any specific investigational drug by any individual patient. Kura Oncology may revise this Expanded Access Policy at any time.