Expanded Access to PCNAT-01 for Patients With Resected Pancreatic Ductal Adenocarcinoma Following Surgical Resection and Adjuvant Therapy

Expanded Access Program for PCNAT-01, a Personalized Tumor Neoantigen Peptide Vaccine, in Patients With Resected Pancreatic Ductal Adenocarcinoma Following Surgical Resection and Completion of Adjuvant Chemotherapy

This expanded access program provides a potential pathway for eligible patients with resected pancreatic ductal adenocarcinoma (PDAC) to receive PCNAT-01, an investigational, personalized tumor neoantigen peptide vaccine, outside of the ongoing clinical trial when participation in the clinical trial is not possible or feasible. PCNAT-01 is intended for use in disease-free patients following surgical resection and completion of standard adjuvant therapy to reduce the risk of recurrence. Access is subject to Sponsor review, regulatory authorization, institutional review board approval, physician oversight, successful patient-specific vaccine manufacture, and confirmation that treatment under expanded access will not interfere with the ongoing clinical development program.

PCNAT-01 is an investigational, personalized tumor neoantigen peptide vaccine composed of patient-specific synthetic peptides and Poly-ICLC. PCNAT-01 is being evaluated in Study PCNAT-01-2024-104, a Phase 1, multicenter, open-label study in disease-free patients with resected PDAC after completion of standard adjuvant chemotherapy. The ongoing Phase 1 study evaluates the safety, tolerability, immunogenicity, and preliminary clinical activity of PCNAT-01 and includes a Phase 1a dose-escalation part followed by a Phase 1b dose-expansion part.

This expanded access program is related to Study PCNAT-01-2024-104, "A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of PCNAT-01 in Patients With Pancreatic Cancer Following Surgical Resection and Adjuvant Therapy" [NCT number to be added once assigned]. Whenever feasible, participation in the clinical trial should be considered before expanded access.

This expanded access program is intended for eligible patients who may have a serious disease history of resected PDAC and are at risk of recurrence after standard therapy but are unable to participate in the ongoing PCNAT-01 clinical trial. Whenever feasible, participation in the clinical trial should be considered before expanded access. Expanded access may be considered only when the requesting physician determines that the patient has no comparable or satisfactory alternative options and that the potential benefit justifies the potential risks.

Because PCNAT-01 is personalized, access depends on the availability of adequate tumor tissue, successful sequencing and neoantigen identification, and successful manufacture and release of the individualized vaccine. Patients must also meet protocol-defined clinical, safety, laboratory, disease-status, and tumor-marker requirements before receiving PCNAT-01. Requests will be reviewed on a case-by-case basis by the Sponsor in collaboration with the treating physician. Treatment may proceed only after all applicable regulatory, institutional, informed consent, and product-release requirements are satisfied.

Eligibility for expanded access will be determined on a case-by-case basis by the Sponsor and treating physician. Key criteria may include the following:

Inclusion Criteria

1. Adult patients aged 18 years or older.
2. Histologically confirmed pancreatic ductal adenocarcinoma.
3. Prior R0 or R1 surgical resection of PDAC.
4. Completion of standard adjuvant therapy, unless discontinued early due to intolerable toxicity as permitted after Sponsor and physician review.
5. No evidence of disease recurrence before PCNAT-01 administration.
6. ECOG performance status of 0 or 1, or otherwise acceptable functional status based on treating physician and Sponsor assessment.
7. Adequate organ and marrow function to receive PCNAT-01.
8. Availability of adequate tumor tissue for sequencing and neoantigen identification.
9. Successful identification of sufficient patient-specific neoantigens for PCNAT-01 manufacture.
10. Ability to provide informed consent and comply with expanded access procedures.
11. For patients of reproductive potential, willingness to comply with pregnancy testing and contraception requirements.

Exclusion Criteria

1. Ability to participate in the ongoing PCNAT-01 clinical trial, if trial participation is feasible and appropriate.
2. Evidence of recurrent, metastatic, or unresectable disease before planned PCNAT-01 administration, unless specifically accepted after Sponsor and physician review.
3. Known hypersensitivity or intolerance to PCNAT-01, Poly-ICLC, or any component of the vaccine.
4. Active uncontrolled infection or clinically significant viral infection.
5. Active or suspected autoimmune disease requiring systemic treatment, except protocol-permitted conditions.
6. Immunodeficiency disease, organ transplantation requiring immunosuppression, or chronic systemic immunosuppressive therapy.
7. Clinically significant cardiovascular disease, uncontrolled hypertension, clinically significant arrhythmia, or other uncontrolled medical condition.
8. Recent live or live-attenuated vaccination, recent prohibited anticancer therapy, or recent major surgery within a timeframe considered unsafe by the treating physician and Sponsor.
9. Unresolved clinically significant toxicity from prior anticancer therapy.
10. Pregnancy or breastfeeding.
11. Any condition that, in the opinion of the treating physician or Sponsor, would make expanded access treatment unsafe, interfere with patient monitoring, or compromise the ongoing clinical development program.