Oslo (Norway), 23 May 2019 – PCI Biotech (OSE: PCIB), a cancer focused clinical-stage company developing innovative therapeutics that address significant unmet medical needs today announced first patient enrolled in the fimaChem pivotal RELEASE study with registration intent in inoperable bile duct cancer patients. The pivotal RELEASE study has the potential of accelerated/conditional marketing approval as a first-line treatment given the rare disease status and high unmet medical need in this patient population, which currently are left without any approved effective treatment options.
Prof. Dr. med. Jörg Trojan, coordinating investigator for the study in Europe, commented: “Bile duct cancer is a devastating disease with a very clear need of better treatment methods. fimaChem represents a novel local treatment approach for inoperable perihilar bile duct cancer patients and the efficacy data from Phase I are encouraging when compared to standard treatment options. It will be exciting to explore fimaChem’s performance in a randomised setting and I look forward to working with my fellow investigators on this pivotal study.”
Per Walday, CEO of PCI Biotech, said: “We appreciate our investigators’ dedication to patients who are underserved with currently available treatment options and the formal kick-off of recruitment in the RELEASE study represents a major milestone for PCI Biotech. The company is now fully focused on execution of the pivotal phase and we are eager to see the benefits we believe fimaChem can provide to the patients in need of better local treatment options.“
About the RELEASE study
The pivotal RELEASE study design is based on the outcome of meetings with the two leading regulatory authorities European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA). The study programme consists of a single open randomised two-arm study with 186 patients (93 patients per arm), having a control arm with the standard of care (SoC) treatment of up to eight cycles of the chemotherapies gemcitabine and cisplatin, and an experimental arm with up to two fimaChem treatments in addition to SoC. The study’s primary endpoint is progression free survival (PFS), with overall survival (OS) as a key secondary endpoint. The study includes an interim analysis of PFS followed by analysis of objective response rate (ORR), with the potential of accelerated/conditional marketing approval. In addition, the study contains several other secondary endpoints that provide the opportunity to generate robust comparative data of importance for market acceptance of fimaChem as a first-line treatment for inoperable bile duct cancer.
The interim analysis of PFS and ORR for potential accelerated/conditional marketing approval is expected to be available in approximately 36 months, while the final analysis is expected in approximately 50 months. The pivotal study will be executed in clinical sites that first opens in Europe, followed by a roll-out in the U.S.
A Phase I study with fimaChem has provided data on safety and tolerability as well as selection of the dosing regimen for further clinical studies. A total of 23 patients were enrolled in Phase I, which provided encouraging early signs of efficacy and safety results that support further clinical development in this orphan indication. Based on these data, the pivotal RELEASE study is being initiated with up to two fimaChem treatments and will include a seamless safety review by an Independent Data Monitoring Committee (IDMC) when eight patients have completed two treatments in the pivotal RELEASE study.
About bile duct cancer and the fimaChem technology
Bile duct cancer (cholangiocarcinoma) is a cancer that affects the cell lining of the bile duct and represents a patient population with a high unmet medical need. It is a rare disease with an incidence rate of 1-2 per 100,000 in the western world, indicating a total patient population of close to 15,000 per year. The incidence rates are increasing worldwide. The immediate target for PCI Biotech is inoperable patients with perihilar disease. Across Europe and USA approximately 3,000 patients annually are assumed to be eligible for fimaChem treatment. Possible upsides to the targeted patient population include perihilar patients with more extensive metastatic spread, as well as distal bile duct disease. There may also be potential for restaging of patients from inoperable to operable disease by the fimaChem treatment. There is a potential for obtaining a significant uptake in the identified eligible market due to the anticipated benefits, such as no competing marketable treatment alternatives, limited development pipeline, greater efficacy due to local chemotherapy boosts and fimaChem being an add-on to the current standard of care with easy light access through established standard procedures.
Overall survival at 5 years is dismal at less than 10%. Resection is today the only potential cure but only possible in 10-35% of the incidents. Most patients die of local effects of the tumour and the cancer shows remarkable resistance to chemotherapy. A chemotherapy combination of gemcitabine and cisplatin has proven activity in this disease and become standard treatment in several regions. Gemcitabine’s anti-cancer effect is significantly enhanced by the fimaChem technology in preclinical studies.
The first line fimaChem treatment regimen consists of an intravenous injection of fimaporfin, followed four days later by an intravenous infusion of gemcitabine and a laser light application in the bile duct easily administered through endoscopic methods used routinely in these patients. The patients then follows the standard background treatment with up to 8 chemotherapy cycles of gemcitabine + cisplatin. The fimaChem treatment may be repeated during the background chemotherapy treatment cycles. Local tumour response in the bile duct is important to maintain biliary drainage and loco-regional control may therefore be more important for patient outcome than would be the case for many other cancers. The fimaChem treatment boosts the chemotherapy effect locally in the bile duct, thereby directly targeting this area.
Bile duct cancer is an orphan indication with a range of development and market incentives. PCI Biotech has obtained orphan drug designation (ODD) for this disease in both EU and the US, meaning that regulatory authorities may expedite a market approval process, and that a market exclusivity period can be secured under the orphan drug legislations in both regions. ODD is a significant regulatory milestone and it recognises the therapeutic benefits fimaChem seek to bring to the bile duct cancer patients in need of better local treatments.
Per Walday, CEO
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About PCI Biotech
PCI Biotech is a biopharmaceutical company focusing on development and commercialisation of novel therapies for the treatment of cancer through its innovative photochemical internalisation (PCI) technology platform. PCI is applied to three distinct anticancer paradigms: fimaCHEM (enhancement of chemotherapeutics for localised treatment of cancer), fimaVACC (T-cell induction technology for therapeutic vaccination), and fimaNAc (nucleic acid therapeutics delivery).
Photochemical internalisation induces triggered endosomal release that is used to unlock the true potential of a wide array of therapeutic modalities. The company’s lead programme fimaCHEM consists of a pivotal study in bile duct cancer, an orphan indication with a high unmet need and without approved products. fimaVACC applies a unique mode of action to enhance the essential cytotoxic effect of therapeutic cancer vaccines, which works in synergy with several other state-of-the-art vaccination technologies. fimaNAc utilises the endosomal release to provide intracellular delivery of nucleic acids, such as mRNA and RNAi therapeutics, thereby addressing one of the major bottlenecks facing this emerging and promising field.
This announcement may contain forward-looking statements, which as such are not historical facts, but are based upon various assumptions, many of which are based, in turn, upon further assumptions. These assumptions are inherently subject to significant known and unknown risks, uncertainties and other important factors. Such risks, uncertainties, contingencies and other important factors could cause actual events to differ materially from the expectations expressed or implied in this announcement by such forward-looking statements. PCI Biotech disclaims any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.