PharmaCyte Biotech has submitted an Investigational New Drug application (IND) to the U.S. Food and Drug Administration (FDA) for a planned Phase 2b clinical trial in locally advanced, inoperable pancreatic cancer (LAPC).

PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, commented, “Submitting this IND is the most important milestone the Company has met thus far in the clinical development of our leading product candidate. That’s our therapy to combat what is truly a global unmet medical need for those patients with LAPC whose tumors no longer respond to the first line treatments of either Abraxane plus gemcitabine or FOLFIRINOX.

“Now that the IND has been submitted, we must wait a minimum of 30 calendar days before initiating our clinical trial. During this time, the FDA has an opportunity to review the IND to ensure that it’s complete and that the planned clinical trial research patients will not be subject to unreasonable risk. It also gives the FDA time to ask for more information and clarification about the information submitted.

“Completing and submitting our IND is the culmination of many years of hard work and dedication to ensure that we’ve dotted every ‘I’ and crossed every ‘T’ by our partner, Austrianova, all of our committed consultants and our team at PharmaCyte, including our Chief Operating Officer, Dr. Gerald W. Crabtree, our Consulting Chief Medical Officer, Dr. José Iglesias, and the Chairman of our Medical and Scientific Advisory Board, Dr. Matthias L?hr.”

PharmaCyte’s Consulting Chief Medical Officer, Dr. José Iglesias, stated, “I am pleased that PharmaCyte has submitted its IND to the FDA. The entire PharmaCyte team can now begin work to prepare for the clinical trial. Combining a unique biologic that consists of encapsulated genetically altered human cells with a well-known cancer drug to treat LAPC, PharmaCyte has the possibility of changing the way LAPC is treated in the future. This novel treatment modality may also hold the potential for extending the life span of patients with LAPC.”

The proposed multicenter, randomized, open-label Phase 2b clinical trial is intended to evaluate the efficacy and safety of CypCaps (genetically engineered human cells encapsulated using the Cell-in-a-Box technology) in combination with low doses of the chemotherapy prodrug, ifosfamide, as compared to chemoradiation therapy with capecitabine plus external beam radiation therapy (“EBRT”) or stereotactic body radiation therapy (“SBRT”) alone. The study population will consist of approximately 100 patients. Patients will be randomized in a 1:1 ratio to either treatment with the study therapy or a comparator. The randomization will be stratified by previous treatment (Abraxane plus gemcitabine or FOLFIRINOX) and the control arm choice (capecitabine/EBRT or SBRT alone).

The primary objective will be determined by progression free survival (“PFS”). The secondary objectives for this study are to determine if CypCaps plus low-dose ifosfamide will: (i) increase overall survival (“OS”); (ii) increase objective response rate; (iii) increase the rate of conversion of the pancreatic tumor from inoperable to operable; (iv) decrease the pancreatic cancer tumor marker CA 19-9; and (v) improve a patient’s quality of life. In addition, this clinical trial will assess the safety and tolerability of CypCaps plus low dose ifosfamide.