#74

Pullan's Pieces - #74 August 2012

Science and Business in Biotech and Pharma

In This Issue

Genes CAN be patented

Diarrhea drug 1st oral for extra Medicare payment

What we did not know - fun new science

DEAL: Novartis rewards U Penn's high-risk translational work

DEAL: Is RNA hot? RNA deal of Regulus and AstraZeneca

Genes can be patented, says Appeals Court

The Supreme Court sent the gene patenting case of AMP versus Myriad to the Federal Court of Appeals, following the decisions on Mayo versus Prometheus (a case on the patentability of methods in diagnostics).

On August 16th, 3 judges of the Court of Appeals preserved decades of gene patents, and decided that isolated DNA, including cDNA, was patentable as new compositions of matter not existing in nature. The majority ruled that as the structures of the isolated molecules, with their different ends and removal of histones, do not exist in nature (and that all new chemical entities are derived from natural materials), they are the result of human ingenuity and skilled labor. The informational nature of DNA does not change the fact that it is a new chemical entity when isolated. Mayo versus Prometheus was not found to be relevant to the patentability.

The Court rejected the patentability of comparing and analyzing sequences to see the mutations as difference, as there was no transformative step (consistent with the Mayo Prometheus case).

But the Court of Appeals upheld the patentability of screening cancer therapeutics by measuring their effects on cells with altered BRCA genes compared to cells without BRCA gene mutations. Growing the man-made transformed cells and determining their growth rate is not merely a natural process.

It may still go to the whole Court of Appeals and/or to the Supreme Court, but, for the moment, genes are patentable and methods must be more than mental analyses to be patentable. Myriad's gene patents stand (at least until their expiration at the end of 2015).

look at the court ruling

Diarrhea drug is 1st oral med to get extra from Medicare as new technology

Medicare pays hospitals a fixed, prospectively determined amount for each in-patient hospitalization, based on severity diagnosis-related groups (MS-DRGs). Each MS-DRG has a payment weight assigned to it, based on the average resources used to treat Medicare patients in that MS-DRG.

But there is a mechanism (New Technology Add-on Payments) designed to allow extra reimbursement for a new technology that provides a substantial new clinical benefit.

Optimer, just managed to get the Centers for Medicare & Medicaid Services to agree to an NTAP payment to provide hospitals with a payment beyond the standard-of-care DRG reimbursement, of up to 50% of the cost of their drug , DIFICID for traveler's diarrhea, for a maximum of $868. The NTAP payment is available to hospitals only for individual patient cases that are more costly than the average DRG charges.

see Optimer press release

With governments controlling how much is paid for a new drug, demonstration of the added value and mastery of reimbursement pathways becomes ever more important. This is the first oral drug to get NTAP payments.

What we did not know!

Part 1. Protein takes 2 very different structures and 2 jobs

The transcription factor RfaH binds to DNA to determine if genes are transcribed or not. But once done, its helical structure unravels to refold into a barrel structure where it helps in translation, recruiting the ribosome to the mRNA. It is not yet clear if the structural change can go in the other direction, nor if dual structures and roles occur in other proteins.

more on brain plumbing system

DEAL: Novartis rewards U Penn's high-risk translational work

Novartis (NVS) is rewarding University of Pennsylvania's

Translational biology:

Universities are playing an increasing role in translating their basic science closer to useful drugs and technologies as funds are scarce in either young companies or in big pharma for such essential work.

Immunotherapy:

increasingly a promising path for cancer therapies.

Personalized medicine:

the vision of the future but a very challenging business model, with patient specific therapies meaning a processing center not a drug in the bottle.

in a deal paying for a $20M cell processing center.

The deal follows encouraging results from a pilot clinical trial of a patient-specific immunotherapy, CART-19 for chronic lymphocyte leukemia.

The personalized immunotherapy uses ex-vivo genetic engineering to put on the surface of the patient's T cells, a tumor antigen recognition domain of an antibody (here anti-CD19), along with a signal (the CD3-zeta chain) for T cell activation. After the processing, the T cells are returned to the same cancer patient. When tumor antigen is encountered, CAR-modified T cells become activated and kill in an antigen-dependent manner. There is no need for different therapies for HLA-subtypes, as would be true for a peptide antigen approach. But it is still personalized medicines and more of a procedure than the drug in the bottle of the pharma industry.