Claudia Pluess, the company’s senior regulatory affairs manager, called the April 27 decision “another important regulatory milestone, validating the potential therapeutic use of PQR309” for such patients, she said in a press release. In December 2016, the U.S. Food and Drug Administration (FDA) granted the medication similar orphan drug status for the treatment of primary central nervous system lymphoma (PCNSL).
PQR309 is a competitive inhibitor of the PI3K-mTOR signaling pathway, believed to be among the most highly mutated pathways in all cancers.
Through genetic mutations and amplifications, an overactive PI3K-mTOR pathway drives cancer cell proliferation, survival and resistance to commonly used chemotherapeutics. PQR309 acts by directly binding to and inhibiting both P13K and mTOR. This dual action makes PQR309 more potent than therapeutics that only target one of such component. Researchers conducted a Phase 1 clinical trial (NCT01940133) assessing PQR309’s safety and toxicity in 2015.
Between 10 and 15 percent of DLBCL patients do not respond to therapy. Of those who do, 20 to 25 percent will ultimately suffer a recurrence of the disease, highlighting the need for better therapeutics.
The EMA’s orphan drug designation goes only to medicines intended to treat conditions whose prevalence in the 28-member European Union is under 5 in 10,000. It allows PIQUR to benefit from EU financial incentives to develop the drug to treat DLBCL.
“PIQUR will continue to work with physicians and regulatory agencies to further define the clinical development strategy to bring a potential new treatment option to patients suffering from this disease,” said PIQUR’s CEO, Dr. Vladimir Cmiljanovic, who co-founded the company in August 2011 as a spinoff of the University of Basel.
PGR309 is the focus of several ongoing clinical trials, including a Phase 1 trial for advanced solid tumors (NCT02483858), two Phase 2 studies for relapsed or refractory lymphoma patients (NCT03127020 and NCT02249429), and a Phase 2 trial for relapsed or refractory PCNSL (NCT02669511). All are now recruiting participants.
The PIQHASSO Phase 1/2 trial (NCT02723877), also recruiting patient,s is evaluating the combination of PQR309 with Halaven (eribulin) in metastatic HER2-negative and triple-negative breast cancer. PIQUR is also studying PQR309 in an ongoing Phase 2 trial for glioblastoma multiforme patients (NCT02850744).
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