Montreal, Canada and Geneva, Switzerland - AngioChem, Inc., a clinical-stage biotechnology company dedicated to creating and developing new drugs to treat brain diseases, announced today that its lead product, ANG1005, is safe and well tolerated in patients with brain cancer treated to date. ANG1005 consists of a proprietary peptide vector (Angiopep) that transports drugs across the blood-brain barrier (BBB) conjugated to three molecules of the anti-cancer agent paclitaxel. The results, from ongoing Phase I/2 studies in the US and preclinical vector delivery data, were presented today at the 20th EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics (Geneva, Switzerland).
"This preliminary safety and tolerability data is very encouraging but we are still in the early stages of clinical development and dose-escalation continues. We know from our preclinical data that our platform Angiopep vector technology is able to deliver not only paclitaxel to the target brain cells but also any other type of drug, including peptides , monoclonal antibodies siRNAs and other biologicals," said Dr. Jean-Paul Castaigne, Chief Executive Officer of AngioChem. "Brain disease is a huge market, worth around US$65 billion a year, yet there still remains a significant unmet medical need for effective treatments. In many diseases effective drugs candidates have being identified; the issue is getting them into the brain so that they are able to work effectively. We believe our Angiopep technology has the ability to transport these drugs candidates across the blood-brain barrier, thus treat many diseases of the central nervous system."
AngioChem is conducting two parallel phase 1/2 studies involving approximately 30 patients per study: one study treating recurrent glioblastoma and a second study treating brain metastasis. The primary endpoint of both studies is safety, tolerability and determination of the maximum tolerated dose (MTD). Secondary endpoints include determination of the pharmacokinetic profile of ANG1005, a preliminary assessment of efficacy, as well as the measurement of ANG1005 levels in surgically removed tumors from glioblastoma patients. Both studies are being conducted at multiple sites in the United States. Top-line data from the studies are expected before the end of 2008.
In the first clinical trial presented (Abstract 424), data from 22 patients with advance solid tumors with brain metastases showed that doses of ANG1005 up to 500 mg/m2 were safe and well tolerated. ANG1005 is administered by intravenous infusion for 1 hour every 21 days. Doses of the drug continue to be increased as part of the ongoing clinical study.
The second trial presented (Abstract 425) included data from the first 12 patients with malignant glioma treated to date. The data showed that ANG1005 administered by intravenous infusion for 1 hour every 21 days is safe and tolerable up to and including doses of 105 mg/m2. The dose of the drug continues to be incressed as part of the ongoing clinical trial.
Tests in mice and rats presented (Abstract 117) by AngioChem and collaborators in the US and Canada showed that ANG1005 is transported rapidly across the blood-brain barrier approximately 100-fold higher than free paclitaxel and 10-fold higher than temozolomide, currently the only drug available to treat primary brain cancer. In mice with glioblastoma, administration of ANG1005 resulted in a 27% increase in survival and caused significant shrinking of these tumors in rats.
A second preclinical poster (Abstract 139), where a scientist in France studied ANG1005 in cancer cell lines and mice, revealed the drug to have the same anti-cancer properties to paclitaxel. In rats, the researchers found that ANG1005 significantly reduced brain cancer growth. This effect however was not observed when paclitaxel alone was administered.
About ANG1005 and the AngioPep vector technology
The key challenge to treating brain diseases is the blood-brain barrier (BBB), which prevents more than 95% of drugs from penetrating the brain. AngioChem has developed a physiological way for drugs to reach the brain by engineering a family of peptides that acts as vectors. AngioChem believes that its AngioPep vectors will allow many drugs (i.e. from small molecules to monoclonal antibodies) to get past the BBB hurdle and ultimately express their full treatment potential. Accordingly, AngioChem conjugated one of its vectors to paclitaxel, a widely-used drug over the past 10 years in a number of cancer treatments. AngioChem believes that combining paclitaxel with an AngioPep vector will unlock the potential of this drug to treat brain cancer. In addition, choosing paclitaxel as a first conjugate offers a number of potential advantages in terms of quicker time to market and lower development risk, including potential for fast track status.
About brain disease and brain cancer
The global brain diseases market is projected to grow from $65B today to over $105B by 2015. Conditions encompass diseases not optimally treated, such as Alzheimer's disease, brain cancer, psychiatric disorders, pain, and many others. The increased incidence of neurodegenerative diseases associated with an aging population is one of the key drivers of the 11% CAGR of this drug class.
Brain metastasis occurs in 10-15% of cancer patients, with breast, lung and melanoma being the most common cancers to spread to the brain. The predominant, and most aggressive, form of primary brain tumors is glioblastoma. Once diagnosed, the patients' life expectancy is measured in months. Each year, more than 200,000 people in the United States are diagnosed with a primary (20,000) or metastatic brain tumor. Treatment options are scarce and prohibitive, making the cure rate significantly lower than that of many other types of cancer.
To find solution for these diseases the pharmaceutical and biotech industry faces a major challenge: how to get drugs or drug candidates across the natural guardian of the brain, the blood-brain barrier (BBB).
AngioChem is a Canadian clinical-stage biotechnology company dedicated to creating and developing new drugs to treat brain diseases. The Company leverages a breakthrough platform technology capable of physiologically transporting any drug or drug candidate to the brain across the Blood-Brain Barrier. This approach creates patentable New Chemical Entities (NCEs). While validating its platform technology in clinical trials with ANG1005 for the treatment of brain cancer, the company is also leveraging this technology to create a pipeline of other NCEs with small molecules, peptides, monoclonal antibodies and siRNA addressing brain diseases such as metabolic diseases and neurodegenerative diseases. A detailed description of AngioChem's activities is available at www.angiochem.com.