Business and Science for BD & others
#58, April 2011
How long does a patent take?
The coming power of genome sequencing in cancer
The whole genome sequencing, validation, analyses, and interpretation for better understanding an individual’s cancer cost around $40,000 per tumor-normal genome pair and took 7 weeks. So it is not quite ready for broad use. http://www.genomeweb.com/sequencing/cancer-studies-highlight-clinical-potential-genome-sequencing
Is Orphan Drug exclusivity tied to pricing? Does exclusivity apply to compounding?
The Orphan Drug Act provides 7 years of exclusivity for the specified indication to manufacturers who win FDA approval for drugs that affect fewer than 200,000 people. A recent case of Orphan Drug exclusivity has gotten some press coverage, not all of it clear on the issues.
The off-patent active ingredient, hydroxyprogesterone has long been “compounded” for an individual patient by pharmacists and used “off-label” to prevent premature births, without FDA approval, and without a controlled clinical trial to show its efficacy. In the case of hydroxyprogesterone, KV Pharmaceuticals got Orphan Drug status and FDA approval for hydroxyprogesterone (to be known as Makena) after submitting clinical trial data to the FDA. There were protests about the high price ($1500 per injection versus as low as $20 for a compounded form), and the FDA said, “At this time and under this unique situation, FDA does not intend to take enforcement action."
This FDA decision seems to be based on price and a contradiction of its generally regulatory position on compounding. Compounding is intended to be for tailoring drugs for individual patients needs (such as altering the route of administration or making a non-allergenic form). Compounding is not supposed to be a route around an approved drug’s manufacturer.
On Friday, KV announced that it would slash its price for Makena, to $690 per dose. KV defended the price by pointing out that the FDA had insisted it conduct a long and expensive clinical trial.
Without the incentives of market exclusivity, there would have been little incentive to study the use of the off-patent compound. And case by case decisions do not allow for the predictability needed to invest in those clinical trials.
According to the FDA, the 1983 “ODA [Orphan Drug Act] has been very successful—more than 200 drugs and biological products for rare diseases have been brought to market since 1983. In contrast, the decade prior to 1983 saw fewer than ten such products come to market.” After the ODA was passed (but not before) mortality rates for people with orphan diseases decreased faster than mortality rates for those with more common diseases. http://www.mttlr.org/volfifteen/lichtenberg&waldfogel.pdf http://marginalrevolution.com/marginalrevolution/2011/03/makena-and-the-orphan-drug-act.html
Normal and cancer cells can spontaneously convert to stem cells and cancer stem cells
Quoting from the unusually clear PNAS abstract, “Current models of stem cell biology assume that normal and neoplastic stem cells reside at the apices [top] of hierarchies and differentiate into nonstem progeny in a unidirectional manner. Here we identify a subpopulation of basal-like human mammary epithelial cells that departs from that assumption, spontaneously dedifferentiating into stem-like cells. Moreover, oncogenic transformation enhances the spontaneous conversion, so that nonstem cancer cells give rise to cancer stem cell (CSC)-like cells in vitro and in vivo.” http://dx.doi.org/10.1073/pnas.1102454108.
Dr. Chaffer found breast cells that survived when detached, with 3 genetic markers of stem cells, a weakly active CD24 gene, a missing ESA gene and a strongly active CD44 gene. The cell, when transplanted into mice, could produce complicated structures like milk ducts. When Chaffer transformed the floating breast cells into cancer cells, she found that the low-CD44 ones could transform into high-CD44 cancer stem cells around 2-5 times more quickly than their normal cousins, making more aggressive tumors. The ability of cancer cells to make new cancer stem cells seems to mean that tumors may come back unless therapies kill existing cancer stem cells and other cancer cells. http://blogs.discovermagazine.com/notrocketscience/2011/04/12/breast-cells-naturally-transform-into-stem-cells/
Biotech IPOs continue to be priced low
Tranzyme Inc. became the fifth venture-backed biotech to go public this year, but also the fifth such company to see its offering price slashed ahead of its offering due to weakening demand. Tranzyme’s underwriters had projected the company would sell 5 million shares at $11 to $13 apiece, but the price ultimately was cut to $4 a share to get investors interested, while lifting the number of shares offered to 13.5 million. This had the effect of giving Tranzyme the funding it needed to support clinical trials, but also diluted the venture investors’ stakes.
Weak demand for biotech IPOs is restricting access to capital, depressing valuations of late-stage venture rounds, and giving corporations the edge in partnership and merger talks. http://blogs.wsj.com/venturecapital/2011/04/04/biotech-ipos-continue-bleeding-money/
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