SENTYNL’S EXPANDED ACCESS POLICY FOR INVESTIGATIONAL DRUG CUTX-101 (COPPER HISTIDINATE) FOR TREATMENT OF MENKES DISEASE

Sentynl Therapeutics, Inc. (Sentynl) is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare disease. Under the 21st Century Cures Act, the manufacturer or distributor of one or more investigational drugs for the treatment of one or more serious diseases or conditions is required to make available its policy on how it evaluates and responds to requests submitted under section 561(b) of the Federal Food, Drug, and Cosmetic Act for provision of such a drug.  Described below is Sentynl’s policy for reviewing and responding to requests for individual early patient access to the investigational drug CUTX-101 (copper histidinate) for the treatment of Menkes disease.  Please note that the policy is in no way a guarantee of access.

Sentynl’s Expanded Access Policy

CUTX-101 is under investigation for the treatment of Menkes disease and related disorders.  It has not yet been established as safe and effective for any such treatment by the U.S. FDA or any other regulatory authorities.   As such, the benefits and risks should be carefully evaluated by the treating physician(s) and the caregiver(s). CUTX-101 is, however, available through an Expanded Access Protocol to obtain further safety and efficacy data that may be used to support the approval of the product and subsequent wider accessibility to Menkes disease patients. Sentynl may consider providing CUTX-101, as an investigational drug, under an Expanded Access Protocol.

General Criteria

Sentynl will review and respond to a request that it receives on a case-by-case basis, applying the following criteria:

  1. Only patients residing in the U.S. will be considered for enrollment into the study.
  2. There is no guarantee that Sentynl will provide CUTX-101 for any request.
  3. The patient must have a diagnosis of Menkes disease, verified by the treating physician.
  4. The enrollment of the patient into the Expanded Access Protocol must be in full compliance with applicable ICH guidelines, FDA regulations and local requirements.
  5. The Expanded Access Protocol may be discontinued by the company at any time or upon approval of CUTX-101 by the FDA for Menkes disease.
  6. The treating physician, if eligible as an Investigator, must be willing to comply with the assessments in the Expanded Access Protocol.

Contact Information

A treating physician must submit questions or requests regarding expanded access to the following: StudyInfo@sentynl.com

Additional information may be obtained from the U.S. Food and Drug Administration at: https://www.fda.gov/downloads/newsevents/publichealthfocus/expandedaccesscompassionateuse/ucm504494.pdf

Request Procedures

Requests for participation in the Expanded Access Protocol must be submitted to Sentynl by the licensed treating physician in the U.S. and should include sufficient supporting de-identified patient information to enable Sentynl and the Principal Investigator of the study to evaluate the expanded access request.

Please include contact information so Sentynl can follow-up with the physician directly (i.e., physician’s name/institution, address, phone number, e-mail, and IRB information). Requests must relate to a single patient. No protected health information shall be submitted to Sentynl.

If the physician’s request to enroll the patient is approved, the requesting physician would be expected to become an Investigator to the Expanded Access Protocol.

Anticipated Timing

Sentynl will attempt to acknowledge receipt of a request for expanded access within 2 business days, if possible.

Clinicaltrials.gov Hyperlink

The clinical trial record containing information on the Expanded Access Protocol (CYP-001) is available on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT04074512

Additional Information

As authorized by the 21st Century Cures Act, Sentynl may revise this expanded access policy at any time.  Additionally, the posting of this policy by Sentynl shall not serve as a guarantee of access to CUTX-101 and/or enrollment into the Expanded Access Protocol for any patient.