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Brief description/objective

For patients with cancer cachexia new mechanism-based pharmacological interventions are required, in addition to a standardized palliative cancer care and basic cachexia management approach. The melanocortin system is one of the important targets for drugs against cancer cachexia, others include ghrelin-analogues, anti-inflammatory drugs and those substances directly acting on the muscle. The MC4 receptor antagonist scFv 1E8a is a new agent, which can be intravenously applied once per week. It has three actions, anti-MSH-activity, inverse agonist activity promoting basal activity (this feature do oral MC4-antagonists not have) and third it potentiates h-erp. The work of Obexia, a small start-up company based on an academic research team, demonstrated the effect of scFv 1E8a in rats with increasing nutritional intake and body mass. As next steps a first in human study is planned. As further preparatory steps Obexia will perform an additional characterization of the compound by comparative studies with oral MC4 antagonists and by exploring whether scFv 1E8a influences also the muscle, an important target of anti-cachexia drugs.
In order to prepare a competitive CTI grant application, the fist-in-human study requires protocol writing including involvement of statisticians, clinical trialists experienced in phase 1 and in cancer cachexia, and medical pharmacologists. Potential endpoints need to be prepared, namely autonomic function (sympathetic nerve actions), and measures of muscle function and eventually gene expression related to cachexia.
Market arguments need to be summarized, acknowledging the market situation of cancer cachexia and the situation of competitors. The goal is to submit in spring 2010 a grant application for CTI for the single dose intravenous first in human proof of concept study, applying a double-blind, placebo-controlled, intra-patient crossover design with the primary endpoint safety and as secondary endpoints nutritional intake and appetite, pharmacokinetics and pharmacodynamics, muscle function, and cachexia-associated symptoms.