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.
Contact information:
Per Walday, CEO
pw@pcibiotech.no (mailto:pw@pcibiotech.no)
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 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.
For further information, please visit: www.pcibiotech.com
(http://www.pcibiotech.com)
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.