Are We Reaching the Biotech Singularity?


This opinion piece presents the opinions of the author. It does not necessarily reflect the views of BioSpace.

I'll admit upfront that the above title is a bit of hyperbole. The "singularity" is a term favored by futurists like Google exec Ray Kurzweill, typically in reference to computer technology. The (rather overheated) idea is that we'll reach a point in artificial intelligence where further advances happen at a snowballing pace, fueled by intelligent machines themselves, while humanity undergoes an unprecedented and unfathomable transformation in a short period of time. Kurzweil himself is famous for believing he can live essentially forever if only he can make it to the singularity, because the problems of human biology and aging will be quickly solved (or at least improved at a fast enough pace to keep him alive).

Anyone who has followed the biotech sector will probably be a little less than sanguine about the 69-year-old Kurzweil's chances of immortality.

Nevertheless, the past couple of years have led to a startling number of advances in biotechnology that promise many more to follow–perhaps even at a snowballing pace. While we can quibble about the numbers of FDA approvals for novel drugs, the level of novelty is rising rapidly.

Recall that for many years, the biotech industry was more-or-less synonymous with recombinant proteins. These might now be regarded as the low-hanging fruit of biologics, but they built an industry from the ground up. Amgen got FDA approval for Epogen in 1989 and still sold over $1 billion of this drug last year.

Then came monoclonal antibodies. Discovered in 1975 by Georges Köhler, César Milstein, and Niels Kaj Jerne, subject of the 1984 Nobel in Medicine, the first monoclonal was approved in 1986. Orthoclone OKT3 wasn't terribly good as a medicine, and the failure of some further drugs led many to believe this was a blind alley. But monoclonals became the main drivers of the biotech industry throughout the mid-1990s and beyond. That's true even today–there have been about 75 novel monoclonal antibodies approved over the years, and 17 of those approvals came this year or last year.

The other big driver has been kinase inhibitors. These are novel not so much for their form–they are traditional small molecule drugs–but for the rational design behind them and the targets they seek. Since the approval of Novartis' Gleevec in 2001, dozens of kinase inhibitors, along with related proteasome inhibitors, have driven billions in sales. These remain, along with monoclonals, the mainstay of the industry, and there's little reason to believe that will change soon.

But look at how much has changed in just the past year or so. We've seen multiple approvals for PARP inhibitors, a new class of small molecule. We've seen multiple approvals for antisense drugs. And while the latter is not exactly new–Ionis won approval for Vitravene in 1998–this is the first time that there have been antisense drugs of commercial and clinical significance. Then there's the somewhat related field of RNAi. Subject of a 2006 Nobel Prize, it looks very likely that the first RNAi drug–Alnylam's Patisiran–will be approved in 2018.

Gene therapy has, after a number of fumbles in the early 2000s, also started to snowball. In the narrower sense of gene sequences introduced by viral vectors, there have already been a few approvals, but it looks as if diseases like beta thalassemia, sickle cell anemia, and maybe many more illnesses will soon be addressed by gene therapy in a significant way.

Look more broadly to cancer immuno-oncology–particular areas like CAR-T–and the progress is even more stunning. Not only were there groundbreaking approvals for Novartis and Kite/Gilead in blood cancers, but exciting progress in CRISPR, other gene editing techniques, and of course around checkpoint inhibitors.

The larger point is that each of these areas could drive subsectors of their own. Biotech grew to an industry with over a hundred billion in sales on the backs of recombinant proteins, monoclonals, and (later) kinase inhibitors. Each one of these started off a bit fumblingly and then picked up speed. In just the past couple years, we have multiplied available therapeutic approaches, and it seems that much of the fumbling has already been done.

 To be sure, the recent successes in biotech have led to an enormous–and sometimes overdone–surge of enthusiasm. We watched Axovant Sciences climb to a $3 billion valuation on the basis of a cast-off Alzheimer's drug with no evidence of efficacy, before crashing back down to earth. (Although its current market cap of nearly $600 million still speaks to some pretty…ummm…remarkable optimism). Just this month was saw Odonate Therapeutics file for a $173 million IPO on the basis of a rejected taxane drug originally developed by Genta, a biotech that liquidated in 2012.

There is plenty of froth, no doubt. But there are also signs that biotech innovation may soon accelerate. And that's something we can all be thankful for.

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