May 30, 2014
Angiochem announced today that the Food & Drug Association (FDA) has granted both orphan drug and fast track designation to ANG1005 a novel paclitaxel-peptide drug conjugate leveraging the low density lipoprotein receptor-related protein 1 (LRP-1) pathway to cross the blood-brain barrier (BBB) and enter cancer, for the treatment of glioblastoma multiforme (GBM).
“We are pleased that the FDA supports our applications for both fast track and orphan drug designation for ANG1005 for the treatment of glioblastoma multiforme. These are important regulatory milestones for the program,” commented Dr. Jean Paul Castaigne MD CEO of Angiochem. “We will continue to work closely with the FDA as we advance ANG1005 through clinical development and the associated regulatory processes.”
Fast track designation is granted by the FDA to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Fast Track designation confers some or all of the following benefits: more frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval, more frequent written correspondence from FDA about such things as the design of the proposed clinical trials and use of biomarkers, eligibility for accelerated approval and priority review, if relevant criteria are met, and rolling review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed.
Orphan drug status is granted by the FDA to promote the development of promising therapeutics for the treatment of rare diseases affecting fewer than 200,000 people in the US annually. Orphan drug designation includes benefits such as a seven-year period of marketing exclusivity in the United States after approval. Other potential advantages come in the form of protocol assistance, the ability to apply for research funding, tax credits for certain research expenses, and fee waivers for the regulatory procedures.
ANG1005 is a novel paclitaxel-peptide drug conjugate that represents the first oncology product to leverage the low density lipoprotein receptor-related protein 1 (LRP-1) pathway to cross the blood-brain barrier (BBB) and enter cancer cells is currently in phase 2 clinical development in high-grade gliomas and brain metastasis from breast cancer.
About Glioblastoma multiforme (GBM)
Glioblastoma multiforme (GBM) (also called glioblastoma) are fast-growing, invasive types of glial tumors, commonly spreading to nearby brain tissue. The National Cancer Institute estimates that there will be 23,380 new cases of brain and other nervous system tumors diagnosed in 2014. It also estimates that in 2014, 14,320 people will die of this disease.
ANG1005 is a novel paclitaxel-peptide drug conjugate that represents the first oncology product to leverage the low density lipoprotein receptor-related protein 1 (LRP-1) pathway to cross the blood-brain barrier (BBB) and enter cancer cells. ANG1005 has been studied in over 200 patients in three clinical studies; two phase 1 studies where the product has shown tolerability similar to paclitaxel and indications of activity, and a Phase 2 study for which the intent-to-treat (ITT) analysis demonstrated encouraging signs of anti-tumor activity and was reported at the 2013 AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference. A multi-study Phase 2 clinical program is underway to further confirm the clinical activity of ANG1005 observed in these earlier studies, including a Phase 2 clinical study in patients with primary brain cancers such as recurrent glioblastoma multiforme (GBM) and anaplastic glioma and a Phase 2 clinical study in HER2+ breast cancer patients.
Angiochem is a clinical-stage biotechnology company discovering and developing new breakthrough peptide drug conjugates that leverage the LRP-1 mediated pathway to cross the BBB to treat neurological diseases. These new compounds have the potential to address significant medical needs, many of which are insurmountable due to the fundamental physiological challenge posed by the BBB.
Angiochem is developing a focused product pipeline, including small molecules and biologics, for the potential treatment of a wide range of CNS diseases, including primary brain cancer, brain metastases, lysosomal storage diseases and pain. Founded in 2003, Angiochem maintains headquarters in Montreal, Canada. For additional information about the Company, please visit www.angiochem.com.