COMPASSIONATE USE

Compassionate use policy – VBL Therapeutics – February 2017

VBL Therapeutics is a clinical biopharmaceutical company focused on the discovery, development and commercialization of first in class treatments for cancer. VBL Therapeutics’ lead oncology product candidate, VB-111 (ofranergene obadenovec) is a targeted anti-cancer gene-based biologic agent that is positioned to treat a wide range of solid tumors with current clinical trials in recurrent Glioblastoma (rGBM), ovarian cancer and thyroid cancer.

We are committed to developing safe and effective therapies for patients, and strive to put patients first. Our goal is to provide first in class therapies to patients, as soon as possible, through thoughtful and efficient drug development.

In general, VBL believes that participating in clinical trials is the best way for patients to access medicines prior to approval. In some extreme circumstances when this is not possible, patients with life-threatening diseases or conditions may seek special access to investigational medicines outside of a clinical trial setting.

We recognizes the importance of developing a Compassionate Use Policy and support the need for compassionate use programs. Our goal is to provide study drug access, at the appropriate time and in the correct context, for patients with life threatening diseases.

A number of aspects are considered to determine whether or not to support compassionate use during the development process:

  • Sufficient clinical safety and efficacy data that suggest a favorable benefit/risk profile for the proposed use.
  • Granting access to compassionate use will not interfere with current clinical trials or regulatory filings so as not to jeopardize the ongoing development work VBL is doing to bring VB-111 to market as quickly as possible and to as many patients as possible.
  • Adequate drug supply for both clinical development program (current and future trials) and compassionate use.
  • Evidence to reasonably assess safety and effectiveness to be provided in a less controlled environment than a clinical trial.

In the USA, there is a specific type of compassionate use program called the ‘Expanded Access Program’ (EAP).

For patients who cannot participate in a clinical trial of an investigational drug but have a serious disease or condition that may benefit from treatment with the drug, FDA regulations allow manufacturers of such drugs to provide those patients with access to the drug under certain situations, known as “expanded access”.

VBL has submitted an expanded access request for VB-111 or VB-111 in combination with bevacizumab in May 2016 for intermediate-size patient population that will allow treatment of rGBM patients that do not qualify for the on-going Phase 3 study VB-111-215 (the `GLOBE` study) with VB-111.

VBL has assessed the aspects listed above and determined that the Company currently does not have sufficient drug to provide VB-111 to patients outside of the on-going and near-future planned clinical studies.

If and when VBL will provide compassionate use VB-111 to any of its indications, the company intends to review each individual request for compassionate use and respond to the physician who makes the request.

We will then need to ensure that:

  • Granting access will not interfere with current clinical trials or regulatory filings
  • The patient does not qualify for any ongoing or planned clinical trials
  • The request is made through a clinician expert in the therapeutic area who has weighed the benefit/risk of providing the investigational drug to the patient
  • The patient(s) for whom treatment with an unauthorized medicinal product is sought:
    1. Is suffering from a severely debilitating or life-threatening disease which cannot be treated satisfactorily by an authorized drug in the country where the request has originated.
    2. Has received appropriate standard treatment without success (or no standard treatment exists for the patient’s condition) and no satisfactory alternative drug is available.
    3. Is ineligible or, due to geographical limitations, cannot participate in any ongoing clinical study.
  • Meets inclusion and exclusion criteria.
  • The treatment was approval by the relevant ethics committee.

Once a regulatory agency approves VB-111 for commercial use, existing expanded access programs will be phased out.