Expanded Access Policy

This policy provides general information on how to request access to Alkeus Pharmaceuticals, Inc. (“Alkeus Pharmaceuticals” or “Alkeus”) investigational drugs prior to their commercial availability (“expanded access”). Please note that:

(a)Expanded access requires significant resources and efforts

(b)There is never a guarantee of access to any specific investigational drug by any individual patient

(c)This policy only concerns patients located in the United States

(d)There is no equivalent policy available for patients residing in other countries: in such cases, requests will be considered individually, recognizing that such international requests may be harder to grant

(e)This policy may be revised at any time.

Expanded access, frequently called “compassionate use”, is a process regulated by the United States Food and Drug Administration (FDA), which allows but does not obligate, drug manufacturers such as Alkeus Pharmaceuticals, to provide investigational drugs to patients with serious diseases or conditions, and who otherwise cannot participate in a clinical trial.

Contact Information for Expanded Access Requests

(to be submitted under Section 561(B))

Email (preferred method of communication)

Phone Number

Procedures for making expanded access requests

To make a request for expanded access, the patient or the patient’s physician should send a message to the email address provided above. Please include a phone number in the email so that we are able to reach you.

General criteria used to evaluate and respond to requests for expanded access

Each request will be considered individually after a thorough review of all available information. Alkeus Pharmaceuticals will only consider allowing expanded access if all of the following are true:

(a)The patient has a serious or life-threatening condition with no satisfactory treatment option
(b)The potential benefits outweigh the known or the anticipated risks, based on clinical and scientific knowledge of the requested investigational drug
(c)Granting expanded access does not directly or indirectly, interfere with, or delay the initiation, conduct, or completion of any Alkeus-sponsored clinical trials
(d)Alkeus Pharmaceuticals has an adequate supply of the requested investigational drug, and is able and willing to provide that investigational drug.
(e)The potential benefits outweigh the known or the anticipated risks, based on clinical and scientific knowledge of the requested investigational drug

After review, if a patient is deemed to qualify or is likely to qualify for one of Alkeus’ ongoing or upcoming clinical trials, we will most likely deny the request and redirect the patient to the clinical trial site closest to them. A request will also generally be denied when authorizing expanded access could interfere or delay any of our clinical studies.

Evaluating and making a decision on expanded access is complex: each decision will be made impartially and fairly, and based upon the patient’s clinical file, status of the clinical development of the requested study drug, study drug availability, Alkeus’ internal and external resources required to support the safe use of expanded access, existing medical and scientific data on the requested investigational drug, and multiple other factors.

In general, the earlier the stage of development, the more challenging it will be to grant the request, because little data on the risks or benefits are available. If Alkeus Pharmaceuticals is unable to authorize the request at a given time, that decision may be revisited in the future, for example in case of changes in policy, new results on the risks and benefits, etc.

Also, if expanded access is allowed by Alkeus Pharmaceuticals, further approval by appropriate regulatory authorities (eg: FDA) will most likely be required, in addition to applicable institutional review boards (IRB) or ethics committees (EC). These approvals are independent, and their decision may differ from that of Alkeus.

Length of time anticipated to acknowledge receipt of request for expanded access

It may take up to one month to acknowledge receipt of request for expanded access.

Hyperlink or reference to the expanded access clinical trial record

Expanded access programs may be listed on to the extent required by FDAAA 801.