Business and Science for BD & others
#62, August 2011
House members trying to shorten 12 years of biologics exclusivity
Seven US House Democrats, led by Rep. Henry Waxman (D-CA), are trying to have the 12-year data exclusivity period for biologics (under the US biosimilar approval legislation) excluded from a multi-lateral free trade deal (the TPP). The trade deal is currently being negotiated by Australia, Brunei, Chile, Malaysia, New Zealand, Peru, Singapore, the United States, and Vietnam (and possibly to be joined by other countries). Obama’s proposed 2012 budget would also cut to 7 years the exclusivity for the originator’s biologic against a biosimilar using the new approval pathway. In a letter to the President, the House members stated that "[w]ere the TPP to ultimately contain a 12 year biologics exclusivity provision, it would impede the ability of Congress to achieve the Administration’s proposed 7 year change without running afoul of U.S. trade obligations." http://bilaterals.org/spip.php?page=print&id_article=20083
Tufts: CNS drugs are tough to develop
A CNS drug, the group at Tufts found, will spend 8.1 years in human testing—more than two years longer than the average for all agents. Regulatory approval takes 1.9 years, compared with 1.2 years average for all drugs. Only 8.2% of CNS drug candidates that begin clinical trials will reach the marketplace, compared with 15% overall. Only 46% of CNS candidates succeed in phase III trials, compared with 66% on average for all drugs. As a result, the cost of developing a CNS drug is among the highest of any therapeutic area. http://www.scientificamerican.com/article.cfm?id=a-dearth-of-new-meds
M&A up in the first half of 2011- returns to VCs up too
The HBM Pharma and Biotech report covering US, Canada and European small molecule and biotech therapeutics companies (not diagnostics, devices, tools, etc) found both number and dollar volume of M&A deals were up in the first half of 2011. 18 Companies sold for at least $200M. Of companies with VC/PE backing, the Takeda takeover of Nycomed set records, but even without including that deal, the first half is definitely up. Sanofi did the most deals, 5 deals (including Genzyme) but Gilead and Valeant did the most acquisitions of VC/PE backed companies (with 3 and 2, respectively). In 10 US sales of VC/PE backed companies, the upfronts totaled $2.8B, while excluding the spectacular Nycomed deal, the 4 European acquired VC/PE backed had upfronts totaling $673M. For VC backed companies, the average ratio of exit deal values (without contingent values) to amount invested jumped to 2.9x up from 1.6x in 2010 and 2.1x in 2009. May these trends continue! http://www.hbmpartners.com/report/HBM%20Pharma%20Biotech%20M&A%20Report/HBM%20Pharma%20Biotech%20M&A%20Report%20-%20H1%202011.pdf
What makes a great partnering meeting?
We all wrestle with the balance of cost and benefit of attending meetings. It is sometimes hard to decide because the costs are upfront and the benefits hopefully come afterwards. In thinking about this for myself, I want a good mix of in-licensing and out-licensing interests represented, with enough companies present so that I’m sure to meet with someone new. I also want lots of chances for accidental meetings at receptions and coffee breaks to just maybe make the unexpected partnering connection. However, the software to let find and plan for meetings with potential partners is key too. A defined vocabulary helps make sure you are not missing things in the searches. I tend to book meetings for every available slot and it is great to have easy access to food throughout the day. I do like getting some big picture perspective on our industry and latest trends. Good talks and panels not just with the same people over and over again is a plus. And it is great having some talks not overlapping with my time in the partnering booths. Friendly smart staff can make the whole experience smooth. When all this comes together, I have found that the meetings lead to discussions and typically a number of CDAs and then every once in a while, a deal I can trace back to the partnering meeting, giving a great ROI. All-in-all, I think no one does partnering meetings better than the EBD Group and their Biopharm America meeting http://www.ebdgroup.com/bpa/index.php in Boston Sept 7-9th, is coming up FAST! I’d love to see you there – seeing friends is one more thing I really like in a meeting!
Myriad gene patent upheld in appeals court
The new ruling by the US Appeals Court said that isolated DNA molecules are not products of nature and are patentable, but did not give Myriad the method claims on comparing the gene sequences.
The court ruled that in isolating cDNA, the genes are no longer part of the bigger DNA polymer and chromosomes, and so are transformed and patentable. The test is whether “human intervention has given ‘markedly different,’ or ‘distinctive,’ characteristics.” One of the 2 judges (Judge Alan D. Lourie, with a PhD in chemistry) writing in support of gene patenting said,
“They have to be chemically cleaved from their chemical combination with other genetic materials. In other words, in nature, isolated DNAs are covalently bonded to such other materials. Thus, when cleaved, an isolated DNA molecule is not a purified form of a natural material, but a distinct chemical entity. In fact, some forms of isolated DNA require no purification at all, because DNAs can be chemically synthesized directly as isolated molecules.”
The court also affirmed patents for the Myriad screening process to identify inhibitors, which is based on changes in cell growth rates of transformed cells. However, in throwing out a number of diagnostic methods claims, the Appeals Court decided that the diagnostic comparison of the isolated sequence to one without mutations in the BRCA genes was an information-focused, mental process and not eligible for patenting.
It is not over yet. The original plaintiffs can petition for a rehearing before the same three-panel of judges; petition for a rehearing before the entire Court of Appeals for the Federal Circuit; or petition for the U.S. Supreme Court to hear the case. http://patentlawcenter.pli.edu/2011/08/02/foley-lardner-on-the-aclumyriad-decision/
Big Buzz on Great Results with Genetically Modified T cells, but….
I saw the story of the impressive results with the genetically engineered tumor reactive T cells on TV, in newspapers and multiple newsletters. It is impressive data. The paper in the New England Journal of Medicine describes a p53-deficient, heavily-pretreated patient with chronic lymphocytic leukemia (CLL) who had a complete remission associated with the tumor lysis syndrome after adoptive immunotherapy with second-generation anti-CD19 chimeric antigen receptor–modified T cells.
The patient’s own lymphocytes were depleted with chemotherapy and then he received the genetically engineered T cells. These were peripheral blood T cells infected with a self-inactivating lentiviral vector carrying genes for the chimeric antigen receptor to recognize the tumor and turn on the T cells. The genes included 1) an antibody portion to target CD19, a surface antigen on B cells and on the malignant CLL cells derived from the B cells, 2) a CD3-Zeta T cell receptor portion to couple recognition of the antigen to activation of the T cells, and 3) a 4-IBB region to send the 2nd signal (co-stimulatory signal) and prevent the T cells from shutting down in anergy.
It worked. There was an increase at day 22 in the number of circulating chimeric antigen receptor-positive T cells to a level nearly 1000x that detected the day after the infusion. Eight months after therapy, chimeric antigen receptor–positive T cells persisted, and the patient had no evidence of disease on physical examination or on computed tomographic, flow-cytometric, or cytogenetic analysis. A second patient who experienced a complete response after the infusions also remains in remission 1 year after therapy. "The third patient had a dramatic but partial response," said Dr. Porter. "He has relatively stable disease."
But as noted in the accompanying NEJM editorial, it may be a long time before we see these sorts of results in most tumor types. Few tumor types have antigens that are not also on vital normal tissues, unlike the restricted expression of CD19. And the persistence of autoreactive T cells to most antigenic targets may mean unacceptable toxicity.
See you at the next meeting?
4400 Paseo Santa Rosa, Newbury Park, CA 91320
This newsletter is a free service of Pullan Consulting. About 3300 readers, representing VCs, BD and CEOs of biotech and pharma companies, are on the list. Back issues are on the website www.PullanConsulting.com . Please add the email to your safe sender list to avoid it being treated as junk. To subscribe or unsubscribe, just send me an email.