Pacylex Pharmaceuticals, Inc.

At this time, based on the evidence generated to date, Pacylex has decided not to offer Expanded Access (EA) to any of our investigational products and will not approve EA requests. We will re-evaluate the status of our policy based on data generated from ongoing and future clinical trials.
This will be re-evaluated based on the criteria listed below.

3.1 Criteria
Where there is a possible need for patients to gain early access to a new medicine that is not generally available for prescription, Pacylex will consider the need for early access using the following criteria:

• Where it is expected that there is a need for ongoing treatment with one of our new medicines, we recognize that this must be through normal health services once the new medicine is available for prescription. As such we will only conduct clinical studies and provide early access outside of studies, in those countries where we intend to make the medicine available through normal prescription channels after its approval.
• We recognize that we must have a minimum level of evidence that the new medicine is likely to work before considering early access and must not provide excessive risk to any patients who may receive the new medicine early.
• While addressing this patient need, we will ensure that if we agree to provide early access to our new medicines, that there is sufficient medicine available to ensure the supply will not run out for those patients receiving it early, or for those patients in our clinical studies. This is because we want to ensure that when providing early access, it does not prevent us completing clinical studies and regulatory approval that would lead to wider access through normal routes of prescription.
• Any early access programs we provide will be in line with the laws and requirements of the country involved.

3.2 Continued treatment after clinical studies
To be sure that our new medicines will work effectively and be safe enough to be prescribed for the diseases patients are suffering from, we study them rigorously to see whether they provide adequate relief. The results of these clinical studies allow the regulatory authorities to independently assess whether to allow the new medicines to be prescribed. As this can take several years, patients who participate in our clinical studies may have to wait for a new medicine to become available on prescription.
Where there may be a benefit for a patient to continue to be treated after the clinical study has ended, but before it is available on prescription, we will decide if we have enough information to justify early access. This will depend on the severity of the disease we are studying, how much evidence we have that the medicine works as expected and is sufficiently safe, and whether alternative effective medicines are already available on prescription. If we plan for continued treatment after a clinical study, we will include this information in our study documents so that it is clear to each patient whether this is a possibility before they start in the study.
We understand that it can be difficult to decide to participate in a clinical study, so will ensure we make this information as clear as possible. To be sure we can assess whether the new medicine continues to work effectively and is safe enough to continue to be used during early access, we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine.

3.3 Compassionate or emergency use outside of clinical studies
The best way to see if a new medicine is sufficiently safe and effective is to run clinical studies. As such we prefer that, where possible, patients can participate in our clinical studies. We recognize however that not everyone has this opportunity. Where patients who cannot enroll in a clinical study are suffering from serious or immediately life-threatening diseases, and there are no satisfactory alternative treatments available, we will consider early access if there is enough evidence available in their condition (as described above) and whether there is a possibility of them being able to access the medicine on prescription in the future.
In the same way as after a clinical study, we want to be sure we can assess whether the new medicine continues to work and is safe enough to continue to be used. As such we will ask for the patient’s consent for their physician to collect some relevant information from them on an ongoing basis while they receive the medicine.

3.4 Discontinuation of early access
When a new medicine receives approval from the regulatory authorities for the disease we are studying, and is available for prescription in the patient’s country, we will phase out the early access to allow the health system to prescribe the new medicine in the normal manner.
If, however, the medicine is approved but not available for prescription for some reason, and there is no alternative medicine available, we will work with country authorities to discuss if we can jointly set up a patient-assistance program to allow patients who are benefitting from it, who do not have alternative treatment options, to continue to receive the new medicine. Patient safety is our primary concern, and we respect patient safety by continually evaluating all the evidence that we have available to ensure that early access use of new medicines remains effective and appropriately safe. If, however, the evidence demonstrates that the new medicine is not going to be as sufficiently effective or safe as we had hoped, we will discontinue all early access and work with patients and their physicians to help them safely transition patients to other treatments. When regulatory authorities do not approve the use of the new medicine for prescription, because it is considered insufficiently safe or effective, and Pacylex discontinues its work to achieve regulatory approval, we will work with patients and their physicians to help them safely transition patients to other treatments.