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Bio Blurb November 26, 2013 Dear Readers, Please find below the lateBio Blurb. Please feel free to contact me at the listed email with any questions, comments or contributions that you may have regarding the newsletter. I highly encourage attorneys, law students, and industry professionals to submit biotech-related stories of interest. Chad Brooker Krista Hessler Carver, JD
Wasim K. Bleibel, JD
Chad Brooker, JD
, according to Genetic Engineering and Biotechnology News. (Nov. 19, 2013).
RESEARCH, BREAKTHROUGHS, NEW DIRECTIONS

“Genomic Health, Almac to Develop Chemotherapy Benefit Test.” Genomic Health will exclusively license
Almac Group’s technology and intellectual property to further develop and commercialize a multi-gene test to
predict benefit from DNA damage-based chemotherapy drugs, such as the commonly used anthracycline-based
regimens, in breast cancer. Genomic Health made an up-front payment of $9 million and will pay additional
milestones as certain clinical and commercial endpoints are achieved in the future. Upon successful
commercialization of the test, Genomic Health will pay additional royalties to Almac, according to Genetic
Engineering and Biotechnology News. (Nov. 26, 2013)
“Orexigen shares soar as long-delayed weight drug clears PhIII safety hurdle.” More than two years after
Orexigen Therapeutics ($OREX) began a forced march back through a $100 million Phase III study for its weight
drug Contrave, the biotech is back with the news that it scored the positive interim results it needs for a return trip
to the FDA. And the drug developer says it can look ahead to a potential approval as early as June 2014. Orexigen
was forced to go back to the drawing board as its rivals--Arena ($ARNA) and Vivus ($VVUS)--were eventually
allowed to begin their own troubled marketing efforts for the first new weight drugs in more than a decade.
Orexigen, though, found the front door at the FDA slammed shut early in 2011 as regulators demanded a pricey
study to determine if the therapy triggered any dangerous heart trouble. Investigators had to recruit 10,000
patients for the Phase III safety study, according to Fierce Biotech. (Nov. 25, 2013)
“Study Identifies Protein Essential for Immune Recognition, Response to Viral Infection.” A Massachusetts General Hospital (MGH)-led research team has identified an immune cell protein that is critical to setting off the body's initial response against viral infection. The report that will be published in an upcoming issue of Nature Immunology and is receiving early online release describes finding that a protein called GEF-H1 is essential to the ability of macrophages -- major contributors to the innate immune system -- to respond to viral infections like influenza, according to Science Daily. (Nov. 24, 2013). “Gene-Silencing Study Finds New Targets for Parkinson's Disease.” Scientists at the National Institutes of Health have used RNA interference (RNAi) technology to reveal dozens of genes which may represent new therapeutic targets for treating Parkinson's disease. The findings also may be relevant to several diseases caused by damage to mitochondria, the biological power plants found in cells throughout the body, according to Science Daily. (Nov. 24, 2013) “Novartis sneaks up on Pfizer with CDK4/6 cancer challenger.” Pfizer ($PFE) and its much-discussed palbociclib lead the way in the race to cash in on the blockbuster potential of a new pathway to treat breast cancer, but Novartis' ($NVS) rival drug has quietly closed the gap, promising to compete much sooner than expected. Novartis revealed that LEE011, which inhibits the CDK 4 and 6 enzymes, will enter Phase III in December, rocketing it into contention with Pfizer's drug, which is in midst of late-stage studies in breast cancer. Much like Pfizer is doing with palbociclib, Novartis will test LEE011 in combo with its own letrozole in ER-positive, HER2-negative breast cancer, the company said, according to Fierce Biotech. (Nov. 22, 2013) “Limit to Flu’s Vaccine Evasion Abilities Found.” Predicting the flu’s antigenic ducking and weaving from year to year may become easier in light of a recent discovery, enabling the timely development of seasonal vaccines that won’t be caught flatfooted by the flu’s feints. The discovery emerged after researchers created and evaluated viruses that had a variety of amino acid substitutions—and different combinations of substitutions. This work revealed that seasonal flu escapes immunity and develops into new strains typically by just a single amino acid substitution. What’s more, these single amino acid changes occur at only seven places on its surface. That the flu depends on changes at so few sites to alter its outer coat, and thereby evade vaccines, comes as something of a surprise. Previously, it was thought that the flu could change amino acids at as many as 130 sites, according to Genetic Engineering and Biotechnology News. (Nov. 22, 2013) “Sanofi, Regeneron Show Promising Phase III Results for Sarilumab.” Sanofi and Regeneron today trumpeted successful results of the first Phase III trial for their rheumatoid arthritis (RA) drug candidate sarilumab, saying that the first fully human monoclonal antibody directed against the interleukin-6 receptor (IL-6R) in combination with methotrexate (MTX) therapy improved disease signs and symptoms as well as physical functionw while inhibiting progression of joint damage in adults with RA who saw little improvement through MTX therapy alone, according to Genetic Engineering and Biotechnology News. (Nov. 22, 2013) “Gene Silencing and Dietary Restrictions Shown to Impact Aging.” Scientists at Brown University have found a way to measure the effects of aging by measuring chromatin, a structure along strands of DNA that either silences or permits gene expression. In several newly published experiments they show that gene silencing via chromatin in fruit flies declines with age. They also demonstrated that administering life span-extending measures to the flies, such as switching them to a lower-calorie diet or increasing expression of the protein Sir2, restores the observed loss of gene silencing due to age, according to Genetic Engineering and Biotechnology News. (Nov. 21, 2013) “Insight On Cell Migration, Movement of Cancer Cells.” The migration of groups of cells in order to form tissues is common during the development of an organism. Discovering how these multiple movements are achieved is not only crucial to understand the basic principles of development but provides new information and insights for further research into processes associated with the spread of cancer. Jordi Casanova, head of the "Morphogenesis in Drosophila" lab at IRB Barcelona and CSIC research professor, and Gaëlle Lebreton, postdoctoral fellow in the
same group, have published a study performed using Drosophila melanogaster in the Journal of Cell Science. This
work reveals that in a multiple movement, a single cell can act as the leader and can drag the rest with it. The
scientists have studied the tracheal development of Drosophila in vivo and describe the morphological
characteristics of the leading cell and provide molecular details about how it drives the movement, according to
Science Daily. (Nov. 21, 2013)
“Pfizer, GSK to Start Phase I/II Study of Combo Melanoma Therapy.” Pfizer and GSK have agreed to collaborate
on a Phase I/II study to explore the anticancer efficacy and safety of GSK’s trametinib (GSK1120212) combined
with Pfizer’s palbociclib (PD-0332991) in patients with advanced/metastatic melanoma. The study, named Study
200344, will be conducted by GSK. It is a dose-escalation, open-label study designed to determine the
recommended combination regimen for the combination therapy. The study will also evaluate the effect of the
combination on tumor biomarkers, safety, and anticancer activity in patients with BRAFV600 wild-type
melanoma, including those with NRAS mutations, according to Genetic Engineering and Biotechnology News.
(Nov. 21, 2013)
“Oncolytics tanks on 'confounding' PhIII for cancer drug.” Canada's Oncolytics Biotech ($ONCY) trumpeted
some promising late-stage results for its viral head and neck cancer therapy, but a distortion in the company's
overall survival data rattled investors, sending its shares down as much as 8.6% on Thursday, according to Fierce
Biotech. (Nov. 21, 2013)
“Alzheimer’s Gene Variant Effects May Suggest a Therapeutic Path Forward.” In research results published in
the November 20 issue of Science Translational Medicine, investigators from Massachusetts General Hospital,
Harvard Medical School, the Harvard School of Public Health, and the University of Iowa showed that even low
levels of the Alzheimer's disease (AD) APOE4 protein can increase the number and density of amyloid beta (A-
beta) brain plaques, neuronal damage, and the amount of toxic-soluble A-beta within the brains of mice that serve
as models of the human disease, according to Genetic Engineering and Biotechnology News. (Nov. 20, 2013).

“Linking risk factors, disease origins in breast cancer.” Researchers have found that epigenetic changes to DNA
are associated with aging in disease-free breast tissues and are further altered in breast tumors. Epigenetic changes
describe heritable alterations caused by mechanisms other than by changes in DNA sequence. The discovery
illustrates how cancer and aging are tightly interconnected processes by identifying epigenetic alterations present
in the normal aging breast that may increase disease risk in cancer-free individuals, according to Science Daily
(Nov. 20, 2013)
LAW, POLITICS, & GOVERNMENT

“In Warning Letter, FDA Orders 23andMe to Stop Selling Saliva Kit.” The FDA ordered 23andMe to stop selling
its Saliva Collection Kit and Personal Genome Service (PGS), accusing the genetic test developer of violating the
Federal Food, Drug, and Cosmetic (FD&C) Act of marketing the test without first obtaining clearance or approval
from the agency. In a warning letter to 23andMe, the FDA’s Center for Devices and Radiological Health (CDRH)
said it considered PGS as being “intended for use in the diagnosis of disease or other conditions or in the cure,
mitigation, treatment, or prevention of disease, or is intended to affect the structure or function of the body.”
CDRH cited intended uses for PGS listed on 23andMe’s website during a November 6 visit, which it said
included providing “health reports on 254 diseases and conditions,” including categories such as “carrier status,”
“health risks,” and “drug response,” and specifically as a “first step in prevention” that enables users to “take
steps toward mitigating serious diseases" such as diabetes, coronary heart disease, and breast cancer,” according
to Genetic Engineering and Biotechnology News. (Nov. 22, 2013)
“Portola shoots for early FDA approval after nabbing 'breakthrough' status.” South San Francisco-based Portola
Pharmaceuticals ($PTLA) has nabbed boasting rights to the FDA's new breakthrough drug designation for
andexanet alfa (PRT4445), its Factor Xa inhibitor antidote designed to hit the brake on blood thinners when they turn dangerous. And the biotech says that the BTD helps set the stage for its campaign to win an accelerated approval while pursuing a 2014 launch of a registration trial. This antidote to anticoagulants is meant to complement Portola's oral Factor Xa inhibitor betrixaban, now in a late-stage study for venous thromboembolism prevention in acute medically ill patients. And its effect is being studied on Eliquis, Xarelto, Lovenox and Lixiana, according to Fierce Biotech. (Nov. 25, 20 “FDA reviews weight's affect on emergency contraceptive efficacy.” The FDA is considering the question of whether new labeling is needed to reflect research questioning the effectiveness of morning-after pills for women weighing 165 pounds or more. European health regulators ordered HRA Pharma, a French drugmaker, to change the label for its emergency contraceptive Norlevo. Similar drugs are marketed in the U.S. as Plan B One-Step and as generics.(11/25),(Nov. 25, 2013) “Drug-device combo by Avedro is granted priority new-drug review by FDA .” Avedro's new-drug application for its riboflavin ophthalmic agent and KXL system has received priority review from the FDA. The company said the drug-device combination, if approved, would be the first such product to be OK'd by the FDA for treating patients with corneal ectasia and keratoconus.(Nov. 25, 2013) “Limits on rosiglitazone-containing meds overturned by FDA.” Some restrictions on the prescribing and use of rosiglitazone-containing diabetes drugs, including Avandia, Avandamet and Avandaryl, were lifted Monday by the FDA based on data showing that they didn't raise the chances of heart attacks. "Although some scientific uncertainty about the cardiovascular safety of rosiglitazone medicines still remains, in light of the new re-evaluation of the Rosiglitazone Evaluated for Cardiovascular Outcomes and Regulation of Glycemia in Diabetes (RECORD) trial, our concern is substantially reduced," the agency said. The move means patients will not be required to sign up in a special registry to access the drugs, which will now be sold at most pharmacies.(Nov. 25, 2013(Nov. 25, 2013) “Clarify that FDA fees are exempt from sequester, lawmakers urge.” Twenty-two members of the U.S. House of Representatives asked in a letter last week that congressional budget negotiators state explicitly the intention of Congress to exempt all FDA user fees from sequestration cuts in the final deal. Negotiators were also urged to stop the Office of Management and Budget from sequestering user fees in the future. "By applying sequestration to fee-based agencies, we are going back on the deal we made with those users when we asked them to pay higher fees in exchange for improved services," the letter said. (Nov. 25, 2013) “Pioneering H5N1 flu vaccine from GSK receives FDA approval.” The FDA approved the use of GlaxoSmithKline's H5N1 bird flu vaccine for people 18 and older who could be exposed to the virus. The vaccine is the first with an adjuvant to be cleared in the U.S. for H5N1. It will not be sold commercially and instead will be kept in the national stockpile. "This vaccine could be used in the event that the H5N1 avian influenza virus develops the capability to spread efficiently from human to human, resulting in the rapid spread of disease across the globe," said Dr. Karen Midthun, director of the FDA's Center for Biologics Evaluation and Research.(Nov. 24, 2013). “J&J-Medivir's hepatitis C drug wins FDA approval.” The FDA approved Johnson & Johnson and Medivir's Olysio, or simeprevir, in combination with interferon and ribavirin, as a treatment for chronic hepatitis C infection. Data from clinical trials showed that the virus could not be detected in about 8 in 10 patients after treatment with Olysio. The drug is the third protease inhibitor to be cleared by the FDA for this indication.(Nov. 20, 2013),(Nov. 20, 2013) FDA approves wider use of Bayer-Onyx's Nexavar for thyroid cancer Bayer HealthCare Pharmaceuticals received the FDA's approval to market Nexavar, or sorafenib, as a treatment for metastatic differentiated thyroid cancer. The approval was based on data from a clinical trial that showed Nexavar improved progression-free survival by about 41% compared with placebo. The drug, made by Bayer and Onyx Pharmaceuticals, was previously cleared for treatment of unresectable liver cancer and advanced kidney cancer.(Nov. 22, 2013), “FDA sets up roadblock for Forest, Richter, rejecting schizophrenia drug.” The FDA set up a potentially serious roadblock for Forest Laboratories ($FRX) and its partner Gedeon Richter, rejecting their application for the schizophrenia/bipolar drug cariprazine and likely delaying any possible approval until the agency gets additional clinical trial data. The move into regulatory limbo is a clear setback for Forest Laboratories, which has billed this new therapy as one of its top late-stage prospects as it pushed a full slate of studies for schizophrenia and bipolar disease. Analysts' estimates on peak sales have varied widely, with numbers ranging from $200 million to a blockbuster-sized market. And in the past Richter executives--who struck a deal with Forest dating back to 2005--have estimated top annual sales at up to $2 billion, according to Fierce Biotech. (Nov. 21, 2013). “Pfizer's lung cancer drug Xalkori receives approval from FDA.” Pfizer obtained the FDA's full approval to market Xalkori, or crizotinib, as a treatment for nonsmall-cell lung cancer. Studies showed crizotinib improved progression-free survival compared with chemotherapy.(Nov. 21, 2013). “Regadenoson and adenosine linked to adverse events, FDA warns.” The FDA has issued a safety warning for cardiac nuclear stress test agents regadenoson and adenosine after reports of adverse events such as myocardial infarction and death turned up in the FDA Adverse Event Reporting System database. The FDA recommends that cardiac resuscitation equipment and trained staff be available before administration of both agents and that the drugs should not be used in patients who exhibit signs of cardiovascular instability or unstable angina.(Nov. 20, 2013) “25 drugmakers agree to settle drug-price litigation in La. for $88.4M.” Louisiana Attorney General Buddy
Caldwell announced Wednesday that 25 drugmakers agreed to settle for $88.4 million a state-filed lawsuit over
marketing and the prices they charged the state's Medicaid program. The latest settlements bring the total
recovered by the state from more than 100 drugmakers to $238 million. State Department of Health and Hospitals
Secretary Kathy Kliebert said 50% to 75% of the funds would go to the federal government.(Nov. 21, 2013),(Nov. 20, 2013),(Nov. 20, 2013)
“BioMarin skates through FDA panel for rare disease drug.” The Bay Area's BioMarin Pharmaceutical ($BMRN)
had an easy time at the FDA, winning the support of an agency panel for its latest rare-disease treatment and
feeding expectations for a near-term approval. A committee of agency advisers voted 20-1 in favor of approval
for Vimizim, which treats the rare enzyme deficiency Morquio A. The majority of panelists said BioMarin's drug
demonstrated convincing safety and efficacy, and their recommendation, while nonbinding, likely spells a full
approval once BioMarin's PDUFA date comes around in February, according to Fierce Biotech. (Nov. 19, 2013).

MARKET

“Bayer Makes $2.4B Takeover Bid for Zofigo Partner Algeta.” Algeta said today it received a “preliminary
acquisition proposal” from Bayer worth NOK 14.8 billion ($2.42 billion), as the German pharma and chemical
conglomerate seeks to acquire its partner in developing the recently approved prostate cancer drug Xofigo (also
known as radium Ra 223 dichloride or alpharadin), according to Genetic Engineering and Biotechnology News.
(Nov. 26, 2013).
“SillaJen Buys Jennerex in Potentially $150M Deal.” CRO SillaJen is acquiring San Francisco-based Jennerex, a biotech firm that develops targeted oncolytic immunotherapies for solid tumors. The firms say the total consideration for the transaction—made in all cash—could reach approximately $150 million, including potential future milestone payments, according to Genetic Engineering and Biotechnology News. (Nov. 26, 2013). Jennerex will become a subsidiary of SillaJen after the merger, but it will keep its name and remain headquartered in San Francisco, CA. The acquisition is expected to close in the first quarter of 2014. “Avalon jumpstarts first biotech in $500M GlaxoSmithKline portfolio deal.” Avalon Ventures is kick-starting the first biotech enterprise in its half-billion dollar portfolio deal with GlaxoSmithKline ($GSK), launching a startup named Sitari Pharmaceuticals which will pursue research work out of Stanford University with an eye to developing a new drug for celiac disease, according to Fierce Biotech. (Nov. 22, 2013) “GlaxoSmithKline politely dumps $230M Fabry drug deal with Amicus.” Slightly less than a year after GlaxoSmithKline ($GSK) and Amicus Therapeutics reported that the first step in their Phase III program for the Fabry disease drug Amigal--or migalastat HCI--had ended in failure, the pharma giant has bowed out of its development collaboration. GSK is handing over a check for $3 million to Amicus ($FOLD) for a small equity stake and giving up its codevelopment and commercialization rights in exchange for a royalty on any future sales of the drug, if it eventually gains approval. And the biotech simultaneously announced a deal to acquire a preclinical program while cutting its staff as it drummed extra cash and debt financing, according to Fierce Biotech. (Nov. 20, 2013) “Clovis bets up to $420M on targeted cancer drug.” After reportedly striking out in an effort to find a buyer, clovis Oncology ($CLVS) has struck up a deal of its own, agreeing to buy Italy's Ethical Oncology Science (EOS) and its mid-stage targeted cancer drug lucitanib for up to $420 million. Under the deal, Clovis will hand over $200 million up front for the U.S. and Japanese rights to the dual-selective inhibitor, paying $190 million in stock and $10 million in cash. EOS is up for $65 million if lucitanib picks up FDA approval, plus another $155 million in long-term milestone payments. EOS had already signed over European and rest-of-world rights to Servier, and now Clovis is entitled to up to $470 million in milestones from its inherited partner, in addition to royalties on its Servier's eventual lucitanib sales, according to Fierce Biotech. (Nov. 20, 2013). Click here to join theto receive this free member benefit weekly.

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