FDA grants orphan drug status to PEP-010 for pancreatic cancer
Phase 1b trial in France now testing therapy's safety, antitumor activity
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Pep-Therapy‘s PEP-010, a potential first-in-class treatment for pancreatic cancer.
The therapy is now undergoing testing in CLEVer-PEPtide, a Phase 1b clinical trial (NCT04733027) enrolling an estimated 100 adults with pancreatic ductal adenocarcinoma, known as PDAC —the most common type of pancreatic cancer — or platinum-resistant ovarian cancer.
The study is recruiting patients with recurrent and/or metastatic tumors at three sites in France, with a fourth expected to open soon. It aims to assess PEP-010’s safety, pharmacological properties, and preliminary antitumor activity as either a single agent or in combination with the chemotherapies paclitaxel or gemcitabine.
Orphan drug status is awarded to investigational therapies intended to prevent, diagnose, or treat a rare disease, defined in the U.S. as those affecting fewer than 200,000 people. The designation offers certain incentives to companies developing such drugs, specifically, tax credits for clinical trials, exemptions from regulatory fees, and up to seven years of market exclusivity if the treatment is ultimately approved.
“Receiving [the] FDA’s [orphan drug designation] is an important milestone for Pep-Therapy,” Antoine Prestat, CEO and cofounder of Pep-Therapy, said in a company press release. “It will support our objective to accelerate the development of PEP-010 toward delivering an innovative solution for challenging-to-treat cancers.”
Award of orphan drug status called ‘a significant milestone’ by developer
PDAC, which accounts for more than 90% of pancreatic cancer cases, starts in the pancreatic ducts, a series of tubes that extend from the pancreas and supply enzymes to aid digestion.
Surgery to remove pancreatic tumors is the only potentially curative treatment option. However, just about 20% of patients are eligible for the procedure, and, even with it, the recurrence rate reaches 80% within two years of surgery. Chemotherapy is also available to control symptoms when surgery is not possible, and is used to help shrink tumors before the procedure or to stop the cancer from recurring afterward.
Hatem Azim, MD, PhD, chief medical officer of Pep-Therapy, noted that patients now “have limited treatment options.” Further, Azim said, “the growing incidence and mortality of pancreatic cancer underscores the urgency for new therapies.”
PEP-010 is a first-in-class bifunctional peptide — a short chain of amino acids, which are proteins’ building blocks — designed to penetrate cells and disrupt the interaction between caspase-9 and PP2A. These two proteins are involved in apoptosis, a form of programmed cell death. When their interaction is disrupted, normal apoptosis is restored in cancer cells, causing them to die.
The therapy has shown a good safety profile and antitumor activity in preclinical studies, stopping tumor growth by as much as 85% in patient-derived breast and ovary tumor models, per the company’s website. Adding chemotherapies boosted PEP-010’s effects.
[Orphan drug designation] along with encouraging initial data from our ongoing Phase 1 study strengthen the advancement of PEP-010 as a potential novel alternative [for treating pancreatic cancer].
Data from the Phase 1a part of the CLEVer-PEPtide study, involving 34 patients with recurrent and/or metastatic solid tumors, showed favorable safety and tolerability profiles. Preliminary antitumor activity efficacy was also observed, with 13 patients showing a partial response or stable disease. Two of the confirmed partial responses occurred with PEP-010 in combination with paclitaxel in ovarian and pancreatic cancers, the company stated.
“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,” Azim said. “[Orphan drug designation] along with encouraging initial data from our ongoing Phase 1 study strengthen the advancement of PEP-010 as a potential novel alternative.”
Prestat added: “We look forward to reporting updated clinical data.”
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