When AbbVie AbbVies CEO Rick Gonzalez described his recently acquired product ibrutinib (Imbruvica) as offering a pipeline in a drug, I imagine every business development executive in biopharma chuckled. No question each of them has used this exact line at some point to characterize a partner-ready product that has been developed for one indication, yet just might be useful in a range of others. <\/p>\n
Its tempting to dismiss this concept as slick salesmanship of the Ginsu Knife but wait, theres more variety but dont. While pipeline in a pill may sound like a marketing slogan, it captures important emerging concepts within molecular medicine and pharmaceutical strategy. <\/p>\n
Progress in understanding illness at a molecular level has revealedthat seemingly different conditions may share common molecular elements. For example, two very different cancers may be driven, in part, by a common oncoprotein (a signaling molecule stuck in the on position, say), suggesting a drug that effectively targeted this aberrant molecule could find use in a range of different cancers. This framework is one reason why sequencing has secured such a strong foothold in oncology (think Foundation Medicine) the possibility of identifying a drugable target in an otherwise inscrutable cancer. <\/p>\n
(Disclosure\/reminder: I work at a genomic data company.) <\/p>\n
Outside of oncology, perhaps the most common variation on this theme is the use of a single drug to treat a range of seemingly unrelated conditions, especially illnesses that are caused by some sort of autoimmune response (essentially, the body attacking itself). Perhaps the most common therapeutic here are glucocorticoids (steroids like prednisone) to treat conditions ranging from asthma to arthritis. To be sure, no one would consider steroids an example of an especially precise medicine, given their notoriously pleotropic effects. Not surprisingly, theres been a huge amount of effort trying to develop treatments that more selectively restrain the immune system. <\/p>\n
One example of such a product, which has enjoyed considerable commercial success, is adalimumab (Humira), a biologic that acts by sequestering a powerful secreted (soluble) mediator of immune response called TNF. Consequently, the medicine has been approved for the treatment of several arthritic conditions, several inflammatory bowel conditions, and couple of other autoimmune diseases. <\/p>\n
AbbVies newly-acquired ibrutinib (see here for my detailed story of the medicines wild history) represents yet another variation on this theme, targeting a signalling molecule within an immune cell specifically, a protein involved in the activation of B-cells (which are responsible for the production of antibodies). Ibrutinib was initially considered as a potential treatment for rheumatoid arthritis, an autoimmune disease with incompletely understood pathophysiology that seems to include an antibody component. However, researchers also recognized that a drug blocking B-cell activation might be useful in the treatment of B-cell cancers; this proved to be the indication for which iburtinib ultimately was developed and approved. In a sense, Gonzalezs apparent interest in pursuing the autoimmune possibilities of ibrutinib has a sense of completing the circle, returning the development of the drug to the indication from which it began. <\/p>\n
Of course, development doesnt always proceed as anticipated; AstraZeneca AstraZeneca partnered with Rigel to evaluate a similar inhibitor of B-cell activation, fostamatinib, in the treatment of rheumatoid arthritis. However, when a phase 2b study suggested the new product was likely to be less effective than adalimumab, AstraZeneca returned the rights to Rigel and absorbed a $140M writedown in the process. The potential of a precisely targeted molecule to be used for a range of indications reflects an evolution of the blockbuster ambition. Rather than relying on a single, broadly defined indication (think statins to treat high cholesterol), the industry now often looks to demonstrate the efficacy of a pathway-targeted product in a range of conditions involving the implicated pathway This distinction was elegantly pointed out (as Ive discussed) at a recent Xconomy forum by Rick Morrison of Comprehend Systems, who suggested the increasing need to identify promising indications represented an important application for big data and sophisticated analytics. <\/p>\n
Bottom line for pipeline in a pill: while overused as a sales pitch, the concept reflects a legitimate scientific aspiration that is often pursued, and increasingly realized. <\/p>\n
Also on Forbes: <\/p>\n
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Read more:<\/p>\n
It's A Drug! It's A Pipeline! It's A New Blockbuster Model!<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" When AbbVie AbbVies CEO Rick Gonzalez described his recently acquired product ibrutinib (Imbruvica) as offering a pipeline in a drug, I imagine every business development executive in biopharma chuckled. No question each of them has used this exact line at some point to characterize a partner-ready product that has been developed for one indication, yet just might be useful in a range of others Continue reading