Aug 13, 2012 - Bayer's New Antibody-Drug Combo Earns Orphan Drug Designation
The Food and Drug Administration has granted orphan drug status to Bayer’s new anti-cancer drug cryptically title BAY94-9343. The drug is a mesothelin-targeting antibody-drug conjugate and is currently undergoing Phase I clinical trials designed to assess its safety as a mesothelioma treatment before progressing on to Phase II trials.
Antibody-drug conjugates (ADCs) are a new type of drug that scientists are looking at very closely as potential treatments for various forms of cancer including mesothelioma. The conjugates are composed of an antibody or an antibody fragment which is saddled onto a cytotoxic drug. This combination, it is thought, will lead to much better results than single line therapy ever could. The antibody effectively targets only cancerous cells while the cytotoxic drug is designed to destroy them.
While officials at Bayer have been quite tight lipped about their potential new drug, attaining orphan status is a very big achievement indeed. Essentially orphan drug status is an incentive for pharmaceutical companies to develop curative rather than palliative treatments for rare diseases. These companies would be granted exclusive rights to manufacture these drugs in addition to huge tax breaks should the treatment be successfully developed.
The Phase I trials of this new drug began back in In September of 2011, with Bayer enrolling patients in an escalation study to find out at what levels the drug becomes unsafe and how well it is tolerated within various populations. These tests will establish the pharmacokinetics and pharmacodynamics of BAY 94-9343 as well.
BAY 94-9343 is currently designated for the treatment of any cancer which causes the growth of solid tumors, a category of which mesothelioma falls under.
Scientists have proven that mesothelin, a surface protein, is expressed in abnormally high levels on the surface of mesothelioma cells. By attaching a fully human mesothelin antibody to its new drug, Bayer is in hopes that their treatment will affect only diseased cells. This means that it could potentially be a more effective mesothelioma treatment than traditional tried methods, such as chemotherapy and radiation. Indeed, everything about this drug is cutting edge. Bayer cut an unprecedented agreement to use maytansinoid TAP (Targeted Antibody Payload) technology developed by ImmunoGen, Inc. under a special contract to deliver their cytotoxic drug on target.
It is important to keep in mind that orphan status does not mean the drug is approved for use. It is still classified as an investigative agent and must pass through multiple phases of testing and regulatory review before it can be approved. That process may take years but from the early indications it appears that Bayer may soon be developing this mesothelioma drug on a larger scale.
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