ACTUATE THERAPEUTICS EXPANDED ACCESS POLICY
Actuate Therapeutics (“Actuate”) is dedicated to developing transformative clinical outcomes for patients suffering from advanced, hard-to-treat cancers, many of which are fatal with limited or no treatment options. We are committed to developing innovative therapies as quickly as our clinical programs, data review, and regulatory requirements allow. Through the inhibition of glycogen synthase kinase-3 beta (GSK-3β) we believe elraglusib will improve treatment outcomes for patients and families who suffer from these devastating diseases for which there are few or no treatment options. The purpose of this policy is to describe the requirements for expanded access to the Actuate investigational product elraglusib for the indication of metastatic pancreatic adenocarcinoma (mPDAC) and Ewing Sarcoma outside of a clinical study.
Our Commitment to Clinical Research
There is an urgent need for new therapeutic strategies that combine novel targeted therapies with new mechanisms of action to expand existing chemotherapy regimens to improve patient quality of life and outcomes. Designing cancer focused clinical trials is as unique and variable as the patients we treat, depending on demographics, age, stage of disease, and other health factors. To ensure that we meet our responsibility to deliver safe and effective treatments to patients, we collaborate with physician investigators and health authorities worldwide in the design and conduct of stage appropriate clinical trials. We believe evaluating the safety and efficacy data from our clinical trials to obtain regulatory approval is key to ensuring safe and timely access to our therapies so that we can meet the needs of patients.
As a clinical-stage company, Actuate recommends that the best way for patients to access our investigational therapies is through clinical trial participation. Actuate is currently designing new clinical studies to evaluate elraglusib in the treatment of patients with mPDAC and Ewings Sarcomas, to ensure that it is safe and effective for patients and to generate data. Actuate clinical trials are designed and conducted by investigator physician’s familiar with investigational therapies and the potential side effects, to help ensure the safety of each participant. Participation in a clinical trial will allow Actuate to accelerate the development of elraglusib for the benefit of patients now and in the future by generating data that potentially demonstrates the safety and efficacy of elraglusib prior to submission for approval for marketing by regulatory authorities.
For information about our clinical trials, please visit https://clinicaltrials.gov/ or contact compassionateuse@actuatetherapeutics.com
Expanded Access Policy
We understand that there may be circumstances under which patients, family members, or physicians may request treatment with our investigative therapies. Investigational therapies are those that have not been approved for marketing by the U.S. FDA or other Regulatory Agencies. The use of investigational therapies outside of a clinical trial is sometimes called “expanded access,” “compassionate use,” or “early access.”
According to the U.S. FDA’s published guidelines, expanded access may be appropriate when all the following apply:
- Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.
- There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.
- The patient cannot get the medical product under another investigational medical product study or protocol.
- The possible benefits to the patient justify the possible risks of the treatment, and those possible risks are not unreasonable given the disease or condition to be treated.
- Providing the investigational medical product will not interfere with the clinical investigations that could support marketing approval of the expanded access use or otherwise compromise the potential development of the expanded access product.
Initial requests for expanded access will be referred to study investigators to determine a patient’s eligibility for one of our ongoing clinical studies. If a patient does not qualify for one of our ongoing clinical studies, Actuate, under very limited circumstances, may consider making investigational medicines available for qualifying patients outside of a clinical trial. Access to investigational therapies must balance our clinical development planning with fair and sustainable access for patients. Patients participating in our clinical trials will be prioritized for access to elraglusib as drug supply is limited.
Criteria used by Actuate to evaluate and respond to an expanded access request:
- The request is made by a licensed physician/medical practitioner who is qualified and knowledgeable in the treatment of mPDAC or Ewing Sarcoma. Patients who are interested in understanding access options should speak with the physician overseeing their care.
- The patient has exhausted all available treatment options and is not eligible to participate in an Actuate sponsored clinical trial.
- Assessment of Risk/Benefit to patient participation.
- Actuate has adequate investigational medicine to meet patient needs.
- The request for expanded access, does not impact ongoing or planned clinical trials or regulatory decision making.
- Expanded access requests may only be approved in countries where we have ongoing clinical trials and plan to seek regulatory approval.
- The requesting physician is familiar with the requirements for applying for and managing expanded access studies with regulatory agencies.
- The physician must agree in writing to comply with:
- Any applicable country-specific legal and regulatory requirements related to providing an investigational product under expanded access.
- Any Actuate requirements in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property.
Focusing on developing and enrolling patients into our current and future clinical trials is the fastest path to regulatory approval and ensuring future treatment options to meet patient needs. As we complete and analyze data from our clinical trials we will evaluate our expanded access program.