In 2010, issues that had been previously resolved resurfaced, like funding embryonic stem cells and patenting genes. Some other age-old themes like heightened regulatory scrutiny and cancer treatment advances plus technology breakthroughs and new diseases to consider also warrant mention in our look back at the year gone by. Here’s a round-up of the biggest life science headlines from 2010.
Third-generation sequencing made its debut last year, pushing this revolutionary technology a step further with the ability to produce more data types. Platforms can now generate DNA sequences as well as epigenetic information with single-molecule sensitivity in real time. Helicos, Illumina, Life Technologies, and Pacific Biosciences (PacBio) are all embroiled in a patent-infringement case that pits Helicos against the other three.
Helicos was delisted from Nasdaq in November 16. PacBio had a successful IPO 2010, and GenProbe invested $50 million in the firm in June 2010. Complete Genomics also has third-generation sequencing capabilities, and NHGRI bankrolled $18 million worth of grants last year to spew further innovation.
In May 2010, J. Craig Venter Institute created the first fully synthetic, self-replicating bacterial cell, which was named Synthia. While the U.S. government has plugged $430 million into synthetic biology since 2005, most of it has gone toward developing alternative fuels. Some firms are now starting to leverage the technology for medical purposes. Novartis along with Synthetic Genomics (SGI), which was co-founded by Craig Venter, Ph.D., is working on vaccines.
The first products, however, will likely be synthetic genomic-based tools for synthesis and DNA assembly. In April Life Technologies invested in SGI and then in June purchased about 59% of Geneart, a gene synthesis firm. And on December 22, the Presidential Commission for the Study of Bioethical Issues submitted a review of the synthetic biology field. Several recommendations were made, but all in all it took a fairly neutral stance devoid of fear-mongering.
Genes have been a primary driver of the biotech industry for the last few decades, but a recent court decision may change the way business is done. The issue of gene patenting resurfaced in the courts during 2009, and in March 2010, a New York District Court ruled that the patents on BRCA1 and 2 are invalid.
Myriad Genetics, which owns patents for these genes, is appealing the ruling, and experts believe that it will be reversed, at least in part. The case was initially filed by American Civil Liberties Union (ACLU) and the Public Patent Foundation (PUBPAT) against Myriad, the University of Utah Research Foundation, and the USPTO. In November 2010, the U.S. Justice Department filed a friend-of-the-court brief supporting ACLU and PUBPAT.
Another vacillating item is federal funding of embryonic stem cells (ESC). 2009’s good run for those in favor of government support for ESC research came to a screeching halt. In August 2010, about 17 months after President Barack Obama repealed President George W. Bush’s ban on federal dollars for ESC research, a federal district judge put a temporary block back on. The issue is once again in limbo while the controversy makes its way through the courts.
Geron is having a similar seesaw experience with advancing a Phase I trial of its ESC therapy GRNOPC1. This candidate for spinal cord injury has been “entering” a Phase I trial since 2008. Two clinical holds and subsequent lifts later, the company enrolled the first patient in October 2010. Adult stem cell therapies have had better luck in the clinic, with Osiris and TCA Cellular Therapy moving their products into Phase III testing.
A look at last year’s stories reveals much to-do about the anti-aging potential of sirtuins, that beloved chemical in red wine. Most significantly, GlaxoSmithKline (GSK) reported bad news related to a $720 million investment made two years ago: Its lead sirtuin activator, which was obtained through the 2008 acquisition of Sirtris Pharmaceuticals, showed minimal efficacy in multiple myeloma patients and exacerbation of kidney complications in a Phase IIb trial.
This candidate was the industry’s most advanced sirtuin-based drug. GSK and others continue to develop both sirtuin activators and inhibitors. If there’s anything basic research published in 2010 on sirtuins has taught us, it is that, like most things new in biotech, sirtuins are not well understood.
Cancer is certainly not new, but there are few diseases more complicated. While a cure still eludes scientists, a therapeutic vaccine did make it through to the market. Cancer vaccines was a hot topic in 2009 as well with companies reporting late-stage clinical data on their candidates.
Dendreon won the race and got Provenge approved to treat prostate cancer in April. Now companies are hoping to duplicate Dendreon’s regulatory success, with both autologous and nonautologous vaccines moving through mid- and late-stage trials last year.
Anti-obesity therapies seemed to have stalled at the Phase III stage, nonetheless, the obesity issue has moved from an interesting trend in 2009 to being a key event in 2010. This October two drugs received complete response letters (CRLs) that raised clinical and efficacy concerns: lorcaserin (Arena Pharmaceuticals and Eisai) and Qnexa (Vivus). Arena and Eisai completed a review with the FDA in December and expect to resubmit the lorcaserin NDA by end 2011. Vivus said it will meet with the Endocrine and Metabolic Division of the FDA to discuss its resubmission in January 2011. This is the same division that voted against the sanction of lorcaserin and Qnexa.
On December 7, however, the panel recommended the approval of a third anti-obesity candidate: Orexigen Therapeutics’ Contrave. We should know by January 31 if we’ll have a new approved treatment for obesity. Currently there is one therapy that is sold OTC (Alli/Orlistat), and a second, Abbott Laboratories’ Meridia, was pulled from the market in 2010.
Besides the aforementioned key events in biotech during 2010, we’d also like to point out certain topics that we will be watching closely in 2011.
Genzyme’s manufacturing issues that started in 2009 and shut down a plant in Allston, MA, bled into its operations last year. In the wake of dipping share prices, sanofi-aventis made an unsolicited $18.5 billion bid for Genzyme. While the takeover battle is ongoing, Genzyme is divesting three business units as per strategic plans announced in May. In September LabCorp bought Genzyme Genetics for $925 million, and in November Sekisui Chemical paid $265 million for the diagnostics products business, leaving only the pharmaceutical intermediates division up for grabs.
Intensified regulatory vigilance of marketed drugs was another binding theme for 2010. Avandia, GSK’s type 2 diabetes drug, was pulled from the European market and use was restricted in the U.S. The changes were made in response to data that suggests an elevated risk of cardiovascular events and stroke in patients treated with Avandia. Avastin, Roche/Genentech’s cancer drug, has also been subjected to a higher level of post-marketing scrutiny. The company will have a hearing with the FDA to address the agency’s request to remove breast cancer from Avastin’s label entirely. The EMA, on the other hand, has recommended to the EC that Avastin only be used with paclitaxel and not with docetaxel. Avastin’s potential to be used in multiple cancer settings was touted as being a big part of why Roche doled out $46.8 billion to take full control of Genentech, which discovered the drug.
Medicinal marijuana was definitely in the news a lot. Six states put the issue on the November ballot, taking steps to decriminalize and in some cases legalize marijuana, however, all fell through. Some companies trying to pharmaceuticalize this green weed are having better luck, with GW Pharmaceuticals and Bayer winning approval in Canada, Spain, and the U.K. for its cannaboid Sativex as a treatment for spasticity in multiple sclerosis.
Sam Waksal, Ph.D., has re-entered the biotech industry. After serving about five years of a seven-year, three-month sentence for insider trading, the ex-CEO of ImClone was released in 2009. He got busy quickly, forming a private venture called Kadmon Pharmaceuticals, which is investing in the HCV market.