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.
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.
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.
(Disclosure/reminder: I work at a genomic data company.)
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.
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.
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.
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.
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.
Also on Forbes:
Read more:
It's A Drug! It's A Pipeline! It's A New Blockbuster Model!
- Podcast 9 Athens part 3 and news [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Podcast 10 Athens part 4 [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Podcast 11 Athens Part 5 [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 12 Cardiac Nuclear Medicine in the UK [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Podcast 13 Choline PET and prostate cancer [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode14 ED function and Dysponea [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- ep 15 Collimator Choice and Reconstruction algorithm [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 16 Dr Strauss on vulnerable plaque [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode17 Chemical Stress [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 18 Myocardial Perfusion Imaging and Diabetes [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 19 The INSPIRE trial [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- 20 Dual isotope with a difference [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 21 The real effect of stress [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 22 D-SPECT [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 23 VQ Reprise [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 24 Hepatobiliary Nuclear Medicine [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 25 Sleep Apnea [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 26 Imaging Post Traumatic Stress Disorder [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 27 Cardiac Stem Cells [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 28 Molly Supply [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 29 Melanoma therapy interview with Dr Ekaterina Dadachova [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 30 Viral Therapy [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 31 breast radiotherapy using radioisotopes [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 32 news from snm [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 33 EBV Imaging and Therapy [Last Updated On: November 8th, 2009] [Originally Added On: November 8th, 2009]
- Episode 34 Dr Lele SNM India 2009 [Last Updated On: December 14th, 2009] [Originally Added On: December 14th, 2009]
- Episode 35 Dr Andrew Scott from the Ludwig institute [Last Updated On: January 10th, 2010] [Originally Added On: January 10th, 2010]
- Episode 36 CardiArc [Last Updated On: February 25th, 2010] [Originally Added On: February 25th, 2010]
- Episode 37 Dr Modder Radiosynoviorthesis (Radiation Synovectomy) [Last Updated On: March 31st, 2010] [Originally Added On: March 31st, 2010]
- Episode 38 Indian Nuclear Medicine With Dr Malhotra and Dr Krishna [Last Updated On: May 28th, 2010] [Originally Added On: May 28th, 2010]
- Episode 39 Prof Ora Israel The Utility of Hybrid Imaging [Last Updated On: July 13th, 2010] [Originally Added On: July 13th, 2010]
- Episode 40 New V/Q SPECT developments [Last Updated On: October 11th, 2010] [Originally Added On: October 11th, 2010]
- Episode 41 scintimun [Last Updated On: February 14th, 2011] [Originally Added On: February 14th, 2011]
- Episode 42 Radio-Isotope Supply into the future [Last Updated On: March 6th, 2011] [Originally Added On: March 6th, 2011]
- Podcast 4: DNA therapy [Last Updated On: April 24th, 2011] [Originally Added On: April 24th, 2011]
- Sorry about the delay a new podcast will be out soon! [Last Updated On: April 24th, 2011] [Originally Added On: April 24th, 2011]
- Podcast 5 Flash 3D [Last Updated On: April 24th, 2011] [Originally Added On: April 24th, 2011]
- Podcast 6 Affibody [Last Updated On: April 24th, 2011] [Originally Added On: April 24th, 2011]
- Podcast 7 EANM 2006 Athens Part 1 [Last Updated On: April 24th, 2011] [Originally Added On: April 24th, 2011]
- Podcast 8 Athens Part 2 [Last Updated On: April 24th, 2011] [Originally Added On: April 24th, 2011]
- Episode 43 Rapidscan Part 1, 720P high quality video version [Last Updated On: May 1st, 2011] [Originally Added On: May 1st, 2011]
- Episode 43 Rapidscan Part 1, Ipod quality video [Last Updated On: May 1st, 2011] [Originally Added On: May 1st, 2011]
- Episode 43 Rapidscan Part 1, Audio version [Last Updated On: May 1st, 2011] [Originally Added On: May 1st, 2011]
- Episode 45 Mummies with cardiovascular disease video [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- Episode 45 Mummies with cardiovascular disease audio edition [Last Updated On: May 21st, 2011] [Originally Added On: May 21st, 2011]
- Episode 46 Dr Gordon DePuey Choosing Reconstruction clinical perspective [Last Updated On: August 7th, 2011] [Originally Added On: August 7th, 2011]
- Episode 46 Dr Gordon DePuey Choosing Reconstruction clinical perspective audio version [Last Updated On: August 7th, 2011] [Originally Added On: August 7th, 2011]
- Episode 47 Cardiac Image Fusion With Dr Kaufmann [Last Updated On: September 18th, 2011] [Originally Added On: September 18th, 2011]
- Episode 47 Cardiac Fusion with Dr Kaufmann video version [Last Updated On: September 18th, 2011] [Originally Added On: September 18th, 2011]
- Dear Steve, I am and many more like me are your one more thing. [Last Updated On: October 9th, 2011] [Originally Added On: October 9th, 2011]
- EPISODE 48 PET MRI camera from Siemens with Dr Georgi audio [Last Updated On: October 23rd, 2011] [Originally Added On: October 23rd, 2011]
- EPISODE 48 PET MRI camera from Siemens Dr Georgi video [Last Updated On: October 23rd, 2011] [Originally Added On: October 23rd, 2011]
- Silicon Biosystems to Present Single-Circulating Tumor Cell Molecular Characterization at the Fourth World CTC Summit [Last Updated On: April 25th, 2012] [Originally Added On: April 25th, 2012]
- Leukaemia cells have a remembrance of things past [Last Updated On: April 25th, 2012] [Originally Added On: April 25th, 2012]
- Technologist Education Requirements Can Help Cut Repeat Scans [Last Updated On: April 25th, 2012] [Originally Added On: April 25th, 2012]
- HKU Collaborative Research Discovers A Novel Molecular Mechanism Of A New Anti-HIV-1 Drug Candidate [Last Updated On: April 25th, 2012] [Originally Added On: April 25th, 2012]
- Focal Segmental Glomerulosclerosis and the Nephrotic Syndrome - Part 1 Clinical - Video [Last Updated On: May 4th, 2012] [Originally Added On: May 4th, 2012]
- Electro-Medicine : Biological Physics - The Molecular Basis of Alzheimers Disease - Video [Last Updated On: May 4th, 2012] [Originally Added On: May 4th, 2012]
- osdd heterocyclic compounds and molecular medicines - Video [Last Updated On: May 4th, 2012] [Originally Added On: May 4th, 2012]
- Molecule to Medicine: The Biomarker Frontier - Video [Last Updated On: May 4th, 2012] [Originally Added On: May 4th, 2012]
- Fulfilling the Promise of Molecular Medicine in a Developmental Brain Disorder - Video [Last Updated On: May 4th, 2012] [Originally Added On: May 4th, 2012]
- Focus on Stefanie Dimmeler - Video [Last Updated On: May 4th, 2012] [Originally Added On: May 4th, 2012]
- Dundee [Last Updated On: May 4th, 2012] [Originally Added On: May 4th, 2012]
- Nanotechnology In Medicine: Huge Potential, But What Are The Risks? [Last Updated On: May 5th, 2012] [Originally Added On: May 5th, 2012]
- Cutting Edge Technology Coming To DMH [Last Updated On: May 5th, 2012] [Originally Added On: May 5th, 2012]
- This Week in Experimental and Molecular Pathology [Last Updated On: May 5th, 2012] [Originally Added On: May 5th, 2012]
- Researchers' discovery offers hope for cancer, heart disease miracle drugs [Last Updated On: May 7th, 2012] [Originally Added On: May 7th, 2012]
- Penn State student Zachary Hostetler from Garnet Valley is being honored as a student marshal [Last Updated On: May 7th, 2012] [Originally Added On: May 7th, 2012]
- NCKU Student Wins Prestigious Award for Anti-Cancer Research [Last Updated On: May 10th, 2012] [Originally Added On: May 10th, 2012]
- In-Vitro Diagnostics (IVD) Market (Clinical Chemistry, Immunoassays, Molecular Diagnostics, Hematology Analyzers ... [Last Updated On: May 10th, 2012] [Originally Added On: May 10th, 2012]
- Pitt team uses genomics to identify a molecular-based treatment for a viral skin cancer [Last Updated On: May 10th, 2012] [Originally Added On: May 10th, 2012]
- Genomics used to identify a molecular-based treatment for a viral skin cancer [Last Updated On: May 10th, 2012] [Originally Added On: May 10th, 2012]
- 2 molecular biologists get $500K medical prize [Last Updated On: May 13th, 2012] [Originally Added On: May 13th, 2012]
- 2 molecular biologists share $500K medical prize [Last Updated On: May 13th, 2012] [Originally Added On: May 13th, 2012]
- Lab21 Unveils New Molecular Analysis Services at Greenville Site [Last Updated On: May 13th, 2012] [Originally Added On: May 13th, 2012]
- 2 molecular biologists from NYC to share $500K medical prize for pioneering research on cells [Last Updated On: May 13th, 2012] [Originally Added On: May 13th, 2012]
- Two Molecular Biologists Get $500K Medical Prize [Last Updated On: May 13th, 2012] [Originally Added On: May 13th, 2012]
- Pair of molecular biologists receive Albany Medical Center Prize [Last Updated On: May 13th, 2012] [Originally Added On: May 13th, 2012]
- CNIO scientists successfully test the first gene therapy against aging-associated decline [Last Updated On: May 16th, 2012] [Originally Added On: May 16th, 2012]
- Cancer Institute of New Jersey Aims to Advance Personalized Cancer Treatments Through 'Precision Medicine' [Last Updated On: May 16th, 2012] [Originally Added On: May 16th, 2012]