Marrying diagnosis and treatment
DARMSTADT, Germany—Glioblastoma multiforme (GBM) is the most common and most aggressive malignant primary brain tumor in humans, accounting for 52 percent of all functional tissue brain tumor cases and 20 percent of all intracranial tumors. The prognosis for glioblastoma is extremely poor since recurrence is almost universal (more than 90 percent) following primary standard treatment. About 50 percent of glioblastoma patients die within one year, while 90 percent die within three years. Two companies have joined forces to do something about it.
R-Biopharm AG, a global provider of innovative clinical diagnostics, has entered into a research and development cooperation agreement with Apogenix GmbH, a next-generation immuno-oncology company. The agreement is for the development of companion diagnostic tests for Apogenix’s lead product APG101, a novel targeted anticancer therapy.
Apogenix’s APG101 is a CD95 ligand-binding fusion protein. Final data from the randomized, controlled Phase 2 efficacy trial in patients with recurrent glioblastoma demonstrated the clinical efficacy of APG101 in combination with radiotherapy in all study endpoints compared to treatment with radiotherapy alone, according to the company. Retrospective analysis of data from a Phase 2 proof-of-concept trial showed that glioblastoma patients who express a certain biomarker associated with the CD95 ligand experienced the greatest benefit from treatment with APG101, and biomarker-positive patients survived longer.
“Therefore, Apogenix searched for a strong partner with longstanding experience and expertise in the development of companion diagnostic tests,” said Dr. Juergen Gamer, vice president of business development for Apogenix. “Companion diagnostic tests will allow for discrimination between responders and non-responders and reduce clinical development risk.”
“The partnership strengthens our position in the field of companion diagnostics,” said Dr. Frank Apostel, vice president of companion diagnostics for R-Biopharm. “It provides options for collaboration across therapeutic areas, samples types and technologies.”
Objectives of the agreement are to develop patient- stratifying companion diagnostic tests to identify glioblastoma patients benefiting the most from treatment with APG101 and development of in-vitro diagnostic tests to determine the status of the CD95 ligand in other solid tumors. R-Biopharm will develop and technically validate diagnostic tests for investigational use to support pivotal clinical trials of APG101. Apogenix will validate companion diagnostic tests in a pivotal Phase 2/3 trial in glioblastoma patients, which is currently in preparation. Test technologies will include immunohistochemistry and polymerase chain reaction.
R-Biopharm’s key role will be the technical validation and regulatory approval of companion diagnostic tests. Apogenix’s primary role will be the clinical validation of companion diagnostic tests.
Apogenix believes that its highly qualified scientific team with proven expertise in protein engineering and clinical development is its strong suit. “The team has built a promising pipeline of immuno-oncology drug candidates that target different tumor necrosis factor superfamily (TNFSF)-dependent signaling pathways,” said Gamer. “These TNFSF modulators restore the immune response against tumors.”
Gamer added that APG101 is in clinical development for the treatment of a variety of solid tumors and malignant hematological diseases. It has demonstrated a statistically significant efficacy in a controlled Phase 2 proof-of-concept trial in recurrent glioblastoma. APG101’s mode of action offers broad applicability in other tumor indications.
“R-Biopharm brings a seamless chain of competence from biomarkers to market-ready companion diagnostics for research-driven pharmaceutical and biotech companies,” said Apostel. “It is a fully integrated diagnostic company, with a dedicated corporate division for companion diagnostics.”
He added that R-Biopharm has been developing and commercializing globally successful in-vitro diagnostic products for more than 25 years. It has an experienced team dedicated to the preparation of products for FDA submission and highly skilled scientists in an expanding product development team, a quality management group (quality assurance, quality control and validation) geared toward FDA compliance.
Because the CD95 ligand is expressed in many types of cancer, both companies believe that APG101 has tremendous potential in a large number of oncological indications beyond glioblastoma, including ovarian, colon, prostate, breast, bladder and kidney cancer. Consequently, APG101 has significant commercial potential in the oncology field. The combined annual peak sales potential of APG101 and companion diagnostic test is in excess of $1 billion in major markets for glioblastoma alone.