The Food and Drug Administration (FDA) has granted orphan drug designation to PEP-010 — a first-in-class bifunctional therapeutic peptide — for the treatment of patients with pancreatic cancer, according to a recent news release from the drug’s manufacturer, PEP-Therapy.
The orphan drug designation is a status granted by the U.S. FDA to drugs and biologics intended to treat rare diseases or conditions, according to the official FDA website, fda.gov, which noted that the regulatory designation is a separate process from seeking approval or licensing. According to the news release, these rare diseases or conditions are defined as conditions affecting no more than 200,000 people in the United States.
Glossary:
Bifunctional therapeutic peptide: a type of drug that can perform two distinct functions in the body, in this case, targeting cancer cells in a specific way to trigger cell death.
Maximum tolerated dose (MTD): the highest dose of a drug that can be given to patients without causing unacceptable side effects.
Pharmacokinetics: the study of how a drug is absorbed, distributed, metabolized and eliminated from the body.
Apoptosis: a process of programmed cell death, which is a natural mechanism used by the body to remove damaged or unnecessary cells.
Caspase-9: a protein involved in initiating apoptosis, particularly in response to cellular damage or stress.
PP2A: a protein phosphatase involved in regulating cell growth and apoptosis, and its interaction with Caspase-9 can be disrupted by PEP-010 to restore normal cell death in cancer cells.
“Obtaining orphan drug designation for PEP-010 marks a significant milestone in our efforts to develop our drug candidate for pancreatic cancer and highlights the significant unmet medical need that exists for these patients” Dr. Hatem Azim, chief medical officer of PEP-Therapy, said in the release. “They currently have limited treatment options, and the growing incidence and mortality of pancreatic cancer underscores the urgency for new therapies. ODD along with encouraging initial data from our ongoing phase I study strengthen the advancement of PEP-010 as a potential novel alternative."
The news release highlights that pancreatic cancer is the fourth leading cause of cancer-related death in the U.S., with 66,000 diagnoses and 51,000 deaths annually. Surgical resection is the only curative option, but over 80% of patients are ineligible. Even after resection and therapy, median survival is 3 to 4 years, and only 3% survive advanced cases. Despite limited progress in treatment, chemotherapy remains the primary therapy, underscoring the need for novel options that improve survival without compromising quality of life.
More Information on PEP-010 Therapy, Including Ongoing Trials
PEP-010 functions by entering cells and disrupting the interaction between Caspase-9 and PP2A, two key proteins in the apoptotic pathway, as per the release. When released, PP2A and Caspase-9 restore normal apoptosis in cancer cells.
Moreover, PEP-010 is PEP-Therapy's first-in-class bifunctional therapeutic peptide and is being evaluated in a phase 1b trial for safety, tolerability, pharmacokinetics and preliminary anti-tumor activity. The trial combines PEP-010 with chemotherapy (Taxol [paclitaxel] or Gemzar [gemcitabine]) in patients with metastatic pancreatic ductal adenocarcinoma or advanced metastatic ovarian cancer. This follows safety and tolerability results, with early signs of efficacy, from the phase 1a CLEVer-PEPtide dose escalation trial. Four sites in France are actively recruiting patients for treatment on the trial, including the Institut Curie, Gustave Roussy, Centre François Baclesse, and Institut de Cancérologie de l'Ouest.
According to a news release from BioSpace, the active phase 1b trial consists of two cohorts. The first patient cohort evaluates PEP-010's efficacy at the recommended phase 2 dose with Taxol in patients with metastatic pancreatic ductal adenocarcinoma, while the second determines the maximum tolerated dose and recommended phase 2 dose of PEP-010 with Gemzar in patients with metastatic pancreatic ductal adenocarcinoma or advanced/metastatic ovarian cancer. The target patient enrollment for the ongoing trial is 53 patients.
"Receiving FDA’s [orphan drug designation] is an important milestone for PEP-Therapy,” Antoine Prestat, chief executive officer and co-founder of PEP-Therapy, added in the release. “It will support our objective to accelerate the development of PEP-010 toward delivering an innovative solution for challenging-to-treat cancers. We look forward to reporting updated clinical data.”
The news release from PEP-Therapy concludes by stating that the Company will be presenting at BioSpring from March 17 to March 19, where officials will be available to discuss the company’s latest advancements, including PEP-010.
For more news on cancer updates, research and education, don’t forget to subscribe to CURE®’s newsletters here.
