Aminex Therapeutics’ immunotherapy approach combines the company’s novel, patented polyamine uptake inhibitor, AMXT 1501, with an approved, off-patent polyamine synthesis inhibitor, DFMO.
As demonstrated in multiple animal trials, AMXT 1501 + DFMO, supports activation of the immune system to attack and clear cancer primary tumors and inhibit their metastases. (See Publications).
AMXT 1501 + DFMO negates two major immune system suppressors — polyamines and myeloid-derived suppressor cells. Further, it compromises the cancer-driving roles of both the MYC and RAS oncogenes, thought to be involved with more than sixty percent of solid tumors.
DFMO is a special drug, saving more than 100,000 lives by curing African Sleeping sickness since FDA approval in 1990 for this high mortality disease. More recently, DFMO is being used as a component of neuroblastoma treatment, is of research interest in colorectal cancer preventionand in glioblastoma treatment. It has also demonstrated potential activity in Alzheimer’s animal models.
AMXT 1501 has been specifically developed to enhance and extend the polyamine depletion capabilities of DFMO. Polyamine depletion stops the growth and division of malignant cells and leads to the immune system being able to recognize and attack multiple types of solid tumor cancers.
Based on the promising results to date, Aminex Therapeutics has advanced AMXT 1501 + DFMO into a Phase 1, dose-escalation clinical trial designed to evaluate its safety and determine optimal dosing of the therapy in cancer patientsCurrent Clinical Trial
Expanded Access Policy for Treatment with Investigational Products
Aminex is a small pharmaceutical company currently developing new therapies to treat patients. Patients who meet specific pre-defined criteria may be eligible to participate in Aminex’s clinical trials to access investigational drugs before the drugs have been approved by FDA. Aminex recognizes that patients and their physicians may believe that a patient with a serious or immediately life-threatening disease could benefit from Aminex’s investigational drugs, even if the patient does not qualify for participation in one of Aminex’s clinical trials.
An Expanded Access Program, also known as “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product for treatment outside of clinical trials. More information is available on the FDA website (https://www.fda.gov/news-events/public-health-focus/expanded-access).
How We Evaluate Requests
As a small company, Aminex will consider requests to provide Expanded Access outside of the currently ongoing clinical trials in a very limited context. Expanded access requests submitted to Aminex will be considered on a case-by-case basis and evaluated based on criteria including, but not limited to:
- The patient must have diffuse intrinsic pontine glioma (DIPG) or diffuse midline glioma (DMG), which is considered an immediately life-threatening condition. The patient must have exhausted the standard of care and/or all available therapies typically used to treat the disease, and be ineligible for any relevant clinical trials.
- The requesting physician must:
- Be appropriately qualified and licensed with expertise and facilities suitable for administering the investigational drug and for managing the patient and reporting side effects.
- Be currently participating in an Aminex clinical trial at the time of request.
- Prepare and submit an individual patient Expanded Access Protocol to the appropriate regulatory authority under a new IND and obtain IRB approval to proceed. Aminex must review and approve the protocol prior to submission.
- Routinely update Aminex on patient status.
- Sufficient efficacy and safety data must exist to enable Aminex to determine whether the potential benefit to the patient outweighs the collective potential risks to the patient.
- Supplies of investigational drug are often limited and Aminex must be able to provide it in an equitable manner and ensure it can maintain an adequate supply of the investigational drug without impairing the timely completion of clinical trials or ongoing drug development that could lead to broader patient availability.
- The access must be permitted under applicable laws and regulations.
Please Note: meeting the above expanded access criteria does not guarantee access to an Aminex investigational drug.
How to Submit a Request
Patients interested in seeking expanded access to an Aminex investigational drug should talk to their physician. Requests on behalf of a patient can only be considered when initiated by a licensed physician qualified to administer and oversee the investigational treatment and comply with safety requirements, including safety reporting responsibilities. Additionally, the requesting physician must be currently participating in an ongoing Aminex trial at the time of request.
- Any requests for expanded access must be initiated and submitted to Aminex through a patient’s treating physician at the following email address: EAPrequest@aminextx.com
- The requesting physician must be willing to obtain approval from the relevant Institutional Review Board, obtain an Investigational New Drug Application (IND) from FDA, if necessary, and otherwise comply with federal, state, and local regulations.
- The request must include contact information for the treating physician and sufficient supporting documentation to allow Aminex to evaluate the request.
Response Time and Additional Information
Aminex will make their best efforts to acknowledge and/or respond to expanded access requests within 3 US business days and, after all relevant information has been received, and will provide a final decision within 5 -10 US business days.
For more information about US expanded access programs, please visit:
- U.S. Food and Drug Administration website Expanded Access: Information for Patients for additional information for patients, caregivers and patient advocates.
- U.S. Food and Drug Administration Website Expanded Access: Information for Physicians for additional information for physicians.