Innovation in Biotech R&D

By Taren Grom

The pharmaceutical industry has dramatically shifted its R&D focus from its historical concentration on small molecule drugs to include a rapidly increasing number of biotechnology products, according to a recent analysis from the Tufts Center for the Study of Drug Development.

Biotech products, which accounted for only 7% of revenue generated by the 10 top selling pharmaceutical-biotech products worldwide in 2001, accounted for 71% of the 10 top selling products in 2012. The transformation of big pharma has been driven as much by new technologies that have enabled development of new products that improve disease outcomes and command high prices, as by the expiring patents on many top-selling small molecule drugs, according to Tufts CSDD Director Kenneth Kaitin.
Biotech products and pipelines, which are extremely attractive pharma companies, are helping to drive the innovation evolution. Novel technology platforms have been developed over the last 30 or so years, some of which according to Tufts has spurred an extensive pipeline of products across a wide range of therapeutic areas. For example, in 1989, only 13 biotechnology products were commercially available. By 2012, that number had grown to 210.

I asked several C-suite biotech executives to identify the biotech innovations that they believe have had the biggest impact, not surprisingly rooted in complex science.

Lloyd Everson, M.D., CEO of MolecularHealth, says Dr. Janet Rowley’s identification of the causal –chromosome rearrangement in chronic myelogenous leukemia (CML) is the poster child for what the whole panomic/genomic movement is all about. Dr. Rowley’s discovery eventually led to the development of Gleevec, the first molecular biomarker targeted therapy for leukemia.

“This paints a tremendous picture of the potential medicine can, and should, evolve into,” Dr. Everson says. “We’re at the beginning of a revolution in medicine; how we understand disease, how we diagnose it, and how the fingerprint of a particular disease paired with the DNA of an individual can accurately spell therapeutic options. There is no greater or more pressing example of this than in cancer care. We are just now beginning to unravel the heterogeneity of cancer and how it behaves in a human being.”

Gary Rabin, CEO and Chairman of Advanced Cell Technology, notes that even with all the fanfare around the Nobel Prize winning technology of induced pluripotent stem cells (iPSC), the overall progression of iPSC technology into the clinic has been hampered by reports that iPSC lines may have epigenetic memory and replicative capacity issues, perhaps as a consequence to incomplete dedifferentiation using current iPS techniques.

“Jacob Hanna and his colleagues at the Weizmann Institute of Science have recently developed a method for generating human iPS cells in a true naïve ground state and maintaining them in that form,” Mr. Rabin says. “If this method has in fact solved the stability issue for iPSC lines, than it opens up the opportunities for utilizing this important technology in regenerative medicine therapies.”

Szilard Voros, M.D., CEO and Co-Founder of Global Genomics Group (G3), is focusing his attention on the large systems-biology-based studies that are currently ongoing.

He believes breakthroughs will come from trials such as the GLOBAL study, which involves the integration of data from up to 10,000 patients who will undergo coronary computed tomographic (CT) angiography (an advanced imaging technology for precisephenotyping) with pan-omicanalyses to identify diagnostic biomarkers and disease pathways for cardiovascular diseases.

For more information about biotechnolgy innovation,  read the complete article in the PharmaVOICE Digital Edition.