Personalized medicine, which refers to tailoring treatments to a patient's specific condition, is a revolutionary concept in the field of healthcare, very different from the 'one-size-fits-all' approach to the diagnosis and treatment of disease. One of the companies pursuing this innovative approach of personalized medicine is Canadian drugmaker Aeterna Zentaris Inc. (AEZS) (AEZ.TO).
For readers who are new to Aeterna, here's a brief overview of its product pipeline and what to expect in the coming months...
Aeterna is a late-stage drug development company specialized in oncology and endocrine therapy. The company has two drug candidates in phase III testing - Perifosine for advanced metastatic colorectal cancer and multiple myeloma, and AEZS-130, a diagnostic test for growth hormone deficiencies.
Perifosine is an alkylphospholipid that works by blocking signaling pathways that control cell survival, causing cell death. Aeterna licensed the rights to this compound to Keryx BioPharmaceuticals (KERX) for North America, Yakult Honsha for Japan, Handok for Korea and Hikma for the Middle East and North Africa.
In the colorectal cancer trial, Perifosine in combination with Roche's Xeloda (capecitabine) is compared with placebo and Xeloda. The phase III trial dubbed X-PECT is being conducted by Aeterna's partner Keryx pursuant to a Special Protocol Assessment with the FDA and with Fast Track Designation. The primary endpoint of the X-PECT study is overall survival, with secondary endpoints including overall response rate, progression-free survival and safety.
The trial, involving over 465 patients, is scheduled to be completed during the first quarter of 2012.
In what is perhaps a first in the clinical development of Perifosine in Japan, Aeterna's Japanese partner, Yakult Honsha initiated a phase 1/2 trial early this month to assess the safety and efficacy of Perifosine, in combination with capecitabine, in patients with refractory advanced colorectal cancer.
A phase III registration trial comparing the efficacy and safety of Perifosine vs. placebo when combined with Velcade and dexamethasone in patients with advanced multiple myeloma was initiated by Aeterna's partner Keryx in December 2009. The primary endpoint is progression-free survival and secondary endpoints include overall response rate, overall survival and safety.
The trial, which is designed to enroll about 450 patients, is currently recruiting participants and is expected to be completed in early 2013.
Perifosine has also secured Orphan Medicinal Product designation from the EMA in multiple myeloma, and has received positive Scientific Advice from the EMA for both the advanced colorectal cancer and multiple myeloma programs, with ongoing phase III trials for these indications expected to be sufficient for registration in Europe.
Aeterna also plans to explore development of Perifosine in new indications.
Yet another late-stage drug candidate of Aeterna is AEZS-130, a novel synthetic small molecule acting as a ghrelin agonist that stimulates the secretion of growth hormone. AEZS-130 has been granted orphan-drug designation by the FDA for use as a diagnostic test for growth hormone deficiency. A phase III trial with the compound for use as a simple oral diagnostic was successfully completed last August. The company plans to file an NDA for AEZS-130 in first half of 2012.
AEZS-130 could become the first approved oral test for the diagnosis of AGHD, or adult growth hormone deficiency, a condition which arises when pituitary gland is missing or does not function correctly.
Last November, the FDA granted approval for the initiation of a phase 2A trial with AEZS-130 to assess its safety and efficacy in patients with cancer cachexia. Cachexia, which is characterized by diminished appetite and food intake in cancer patients, results in loss of weight, muscle atrophy, fatigue and weakness.
The other clinical compounds in the company's pipeline are:
* AEZS-108, which has completed phase II trials in advanced endometrial cancer and advanced ovarian cancer, and under phase 1/2 studies in castration refractory prostate cancer and refractory bladder cancer. A pivotal study with AEZS-108 in advanced endometrial cancer is planned in first half of 2012. AEZS-108 has been granted orphan-drug designation by the FDA and orphan medicinal product designation from the EMA for the treatment of ovarian cancer. The worldwide rights to AEZS-108 are with Aeterna.
Earlier this month, Aeterna entered into a collaboration agreement with Ventana Medical Systems Inc., a member of the Roche Group, to develop a companion diagnostic for the immunohistochemical determination of luteinizing hormone-releasing hormone, or LHRH, receptor expression for AEZS-108.
According to Aeterna, AEZS-108 specifically targets LHRH receptors and therefore, could prove to be more efficient in treating patients with LHRH-receptor positive cancers like endometrial, ovarian, breast, bladder, prostate and pancreatic tumors.
* AEZS-112, which is in phase I testing for solid tumors.
The preclinical compounds of Aeterna include, AEZS-115 for multiple cancers, benign prostatic hyperplasia and endometriosis; AEZS-120 for prostate cancer and melanoma; AEZS-129, AEZS-131 and AEZS-132 whose target indications are yet to be revealed; AEZS-127 for multiple cancers; AEZS-123 for obesity and related pathologies and AEZS-137 for solid tumors.
The company has one marketed product - Cetrotide, indicated for use in in-vitro fertilization. Cetrotide, which is an injectable hormone to prevent premature ovulation in women, is marketed worldwide by Merck Serono, except for Japan where it is marketed by Nippon Kayaku and Shionogi.
A quick look at the company's balance sheet...
In the third quarter ended September 30, 2011, Aeterna earned $1.1 million or $0.01 per share, reversing a loss of $9.9 million or $0.12 per share in the year-ago quarter. Revenues increased to $9.5 million in the third quarter of 2011 from $5.7 million in the comparable quarter a year ago, helped by higher-than-normal deliveries of Cetrotide to Merck Serono and comparative strengthening of the euro against the US dollar.
The company has no debt and ended the third quarter of 2011 with $48.1 million in cash, sufficient to take it through to planned NDA filings for AEZS-130 and Perifosine.
Shares of Aeterna are listed on the Nasdaq as AEZS and on the TSX as AEZ. AEZS closed Friday's trading down 1.79% at $1.65. On the TSX, the stock closed Monday's trading up 4.09% at C$1.78.
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