Business and Science for BD & others
#71, May 2012
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Clinical development success rates
BIO and Biomedtracker have updated their analysis of clinical development success rates to include 2011 data for 835 companies. They report that the overall likelihood of approval for a Phase 1 is 10.4%, but if you look at lead indications, that rate is 15.4%. For all diseases, efficacy continues to be the challenge, responsible for 57% of suspensions or failures after Phase 3.
Biologics do better than small molecules, with a 21% likelihood of approval from a lead indication Phase 1 versus 12% for a small molecule NME. (Pharma companies recognize that difference - Novartis had more than twice as many patents for biological drugs compared to small molecules in 2009). http://www.genengnews.com/keywordsandtools/print/3/26751/).
Infectious disease programs have the highest success rate, with 19.3% of lead indication phase 1 trials making it to approval.
Oncology does worst overall with 6.7% of all Phase 1 trials leading to approval, but if you look at lead indications, that rate increases to 13.2%, suggesting that the lead indication does indeed have a stronger rationale. Oncology continues to have a lower rate of success from Phase 3 to NDA or BLA than other indications, presumably a reflection of the transition from tumor shrinkage or progression free survival in Phase 2, to overall survival endpoints in Phase 3. It is apparently not due to the change from open label or single arm trials to randomized trials (http://www.biotech-now.org/business-and-investments/2012/02/oncology-clinical-trials-secrets-of-success).
VC investing falls. The NCVA reports that VCs invested in biotechnology in the 1st quarter of 2012, with $780 million going into 99 deals, a 43% fall in dollars, and a 14% decrease in deals from the prior quarter. http://www.nvca.org/ The drop was seen in Europe too. Healthcare companies completed 40 deals that raised euro 137 million in the first quarter, a 25% drop in deals and a 67% decrease in investment. Deal activity and investment fell in all sectors of the industry. Biopharmaceuticals accounted for the majority of the industry's investment with 26 deals raising euro 108 million, a 16% decline in deals and 66% decline in investment. http://www.fiercebiotech.com/press-releases/venture-capital-investment-continues-fall-europe?utm_medium=nl&utm_source=internal#ixzz1vLnc6tkD However, every year, the first quarter is down from the quarter below.
Big new funds. Kleiner Perkins has closed on $525 million for its 15th fund (aimed at digital, green tech, and life sciences) and New Enterprise Associates is taking in about $2.5 billion for its new fund (to invest in IT, energy and healthcare). http://techcrunch.com/2012/05/17/kleiner-perkins-closes-on-525-million-kpcb-15/
A bit of life in new IPO’s. Four biotech companies—
have all held their own in their early days of trading as public stocks this year. And 7 more are in the line-up. http://www.xconomy.com/national/2012/04/30/biotech-ipos-start-to-show-some-modest-signs-of-life/
Big bucks in M&A, with GSK acquiring Cellzome and chasing HGS, AstraZeneca bidding for Ardea, Roche chasing Illumina, Agilent taking over Dako (diagnostics) among others. And my client ImmunoCellular Therapeutics with its Phase 2 glioblastoma vaccine as its lead program is speculated to be a target. http://www.investorplace.com/2012/04/cue-the-biotech-ma-attack-hgsi-gsk-ilmn-rhhby-imuc-amln/
Hubbub on disease definitions for psychosis, autism.
A few miracle patients get ALK diagnostic adopted, but what about cost effectiveness?
I’ve written in the past about the low use of some companion diagnostics.
But it appears that personally seeing the “miracles” with ALK inhibitor Xaltori in non-small cell lung cancer means that 54% of advanced NSCLC patients are getting the test, despite only about 5% likely to be positive.
But payers such as Express Scripts may not be happy with the cost-effectiveness; they have already produced a list of 10 companion diagnostics (including those for warfarin and plaxil) deemed not cost effective. http://www.biopharmatoday.com/2012/04/genetic-testing-for-tamoxifen-warfarin-plavix-lacks-clear-value-express-scripts-says.html?utm_source=feedburner&utm_medium=feed&utm_campaign=Feed%3A+BiopharmaToday+%28BioPharma+Today%29
16% of cancers from infection – Now where did that number come from?
A fun analysis of the numbers for the proportion of disease caused by a risk factor reminds us there are no individuals where we know what caused the cancer. Rather there are statistical models based on how common the risk factor is (what proportion of cancer patients have had the infection) and the size of the effect (comparing cancer rates in infected and uninfected people or looking at antibodies to the infectious agents in cancer patients) that lead to Population Attributable Fractions (PAFs). These PAFs don’t add up; that is, the cancers in a patient with infection may have also had other “causes”. And given the different methods and studies in the statistical analysis, PAFs from different years or different risk factors are not comparable. http://blogs.discovermagazine.com/notrocketscience/2012/05/10/what-does-it-mean-to-say-that-something-causes-16-of-cancers/
See you at the next meeting?
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