In 2013, AstraZeneca will be advancing three of its cancer drugs to Phase 3 clinical development, the global biopharmaceutical company reports. Leukemia, ovarian cancer, and lung cancer are the targets of the three compounds currently in development.

One patient has already been enrolled in the Phase 3 clinical trial for moxetumomab pasudotox, which will be evaluated on adult patients with hairy cell leukemia who have not responded to or relapsed after standard therapy. This Phase 3 trial is sponsored by the Cancer Therapy Evaluation Program, a program within the Division of Cancer Treatment and Diagnosis at the U.S. National Cancer Institute. Phase 1 test results for the drug showed significant response rates with a manageable safety profile.

"We believe that targeted therapies which address the underlying mechanisms of disease are the future of personalized healthcare, to help meet the unmet needs in treating cancer patients," said Dr. Bahija Jallal, Executive Vice President of MedImmune.

During 2013, the company is also planning to progress olaparib, intended as a maintenance treatment for platinum-sensitive relapsed ovarian cancer patients with BRCA mutations, onto Phase 3. The drug exploits DNA repair pathway deficiencies to preferentially kill cancer cells, which gives it the potential for activity in a range of tumor types.

Finally, the company announced that selumetinib will also move forward within the FDA approval process. A selective MEK kinase inhibitor, selumetinib has already been tested in patients with advanced cutaneous melanoma that harbors a mutation of the BRAF gene. In the second half of 2013, AstraZeneca plans to commence a Phase 3 study of selumetinib in combination with docetaxel as a second-line therapy for patients with KRAS mutation-positive and metastatic nonsmall cell lung cancer.

Accelerating the development of new molecular entities is a priority, the company noted, as oncology has become one of its core therapy areas. AstraZeneca's primary treatment areas include cardiovascular, metabolic, respiratory, inflammation, autoimmune, infection, and neuroscience diseases.

In addition to trials, the company will present new Phase 2 data for olaparib as well as data from a separate study on selumetinib at the American Society of Clinical Oncology Congress in Chicago within the next three weeks.

What is the FDA approval process?

It is reported that, of 5,000 compounds discovered in the pre-clinical stage, only about five will make it through the entire Food and Drug Administration approval process. On its website, the FDA notes that the average review time for an innovative new drug is now only six months.

In the preclinical, also known as the drug discovery phase, researchers identify potential new compounds to treat targeted diseases. Once a compound has been refined, its toxicology is tested in animals and living tissue. and the company then files an Investigational New Drug Application (IND) with the FDA. Fast track designation, an expedited review of a drug, is granted by the FDA when a drug addresses an unmet medical need; the FDA can designate fast track status at any point after an IND has been approved.

Phase 1 studies are usually conducted in healthy volunteers with an emphasis on safety. The goal here is to determine what the drug's most frequent side effects are and, often, how the drug is metabolized and excreted. The number of subjects typically ranges from 20 to 80.

Phase 2 studies begin if the Phase 1 trial does not reveal unacceptable toxicity. Typically, the number of subjects in Phase 2 studies ranges from a few dozen to about 300. With an emphasis on effectiveness, this phase aims to obtain preliminary data on whether the drug works in people who have a certain disease or condition. For controlled trials, patients receiving the drug are compared to similar patients receiving a different treatment - usually an inactive substance (placebo).

Phase 3 studies begin if evidence of effectiveness is shown in Phase 2. Preceding Phase 3, the FDA and the drug's sponsors come to an agreement on how large of a scale the studies should be conducted. Phase 3 trials gather more information about safety and effectiveness, studying different populations and different dosages and using the drug in combination with other drugs. The number of subjects in a Phase 3 study usually ranges from several hundred to about 3,000 people.

Having completed the necessary trials, the company then sends FDA's Center for Drug Evaluation and Research (CDER) the data from all its tests to prove its proposed drug is safe and effective for intended use. A team of CDER physicians, statisticians, chemists, pharmacologists, and other scientists reviews the data and proposed labelling. If this review establishes that a drug's health benefits outweigh its known risks, the FDA approves the drug for sale in the U.S.