Final confirmation of successful safety read-out in the Phase I Extension study

Oslo (Norway), 8 April 2019 – PCI Biotech (OSE: PCIB), a cancer focused clinical-stage company developing innovative therapeutics that address significant unmet medical needs today announced final confirmation of successful safety read-out for the Phase I Extension study in inoperable bile duct cancer patients which currently are left without effective local treatment options. The appointed Cohort Review Committee (CRC) completed a formal review confirming the Company’s preliminary report that no adverse reactions have been reported that would limit the delivery of up to two fimaCHEM treatments in the pivotal RELEASE study with registration intent. The Phase I Extension study is now completed and recruitment will be formally closed.

The clinical data from the Phase I Extension study include a total of seven patients receiving fimaCHEM treatment and five of these received two treatments. Three of the five patients that received two fimaCHEM treatments had measurable disease at baseline and one of these had an overall partial tumour response according to RECIST at 6 months, one had stable disease and one had progressive disease due to the appearance of new lesions.

A post-study follow-up of the patients is ongoing. Five patients were alive at last censoring (December 2018 – mid-February 2019), which all had been treated with two fimaCHEM procedures. Patient numbers are limited and it is too early to put any significance to the emerging survival data with 5 of 7 patients still being alive, but currently this translates to an interim median Overall Survival of approximately 12 months including all 7 patients, i.e. similar to best comparable published data on gemcitabine/cisplatin treatment alone.

Per Walday, CEO of PCI Biotech, said: "With this positive safety clearance for repeated treatments, PCI Biotech is now fully focused on execution of the pivotal phase and we are eager to see the benefits fimaCHEM hopefully can provide to the patients in need of better local treatment options."

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. Based on today’s positive safety data the RELEASE study will be initiated with up to two fimaCHEM treatments and include a seamless safety review by an Independent Data Monitoring Committee (IDMC) when eights patients have completed two treatments.

About the RELEASE studyThe 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 RELEASE study is expected to start during the first half of 2019; the interim analysis of PFS and objective response rate (ORR) for potential accelerated/conditional marketing approval is expected to be available approximately 36 months after study initiation, while the final analysis is expected approximately 50 months from initiation. The pivotal study will be executed in clinical sites that first will open in Europe, followed by a roll-out in the U.S.

Contact information:
Per Walday, CEO
pw@pcibiotech
Mobile: +47 917 93 429

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 fimaCHEM programme consists of a clinical Phase I/II clinical 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. 

Forward-looking statements

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.

This information is subject to the disclosure requirements pursuant to section 5-12 of the Norwegian Securities Trading Act.