ECONOMIC DEVELOPMENT: CALIFORNIA WITHOUT POLICIES TO CAPTURE BENEFIT OF BIOTECH PRODUCTS HEADING TOWARD COMMERCIALIZATION
California’s life sciences companies are on the verge of investing close to $50 billion over the next five years to manufacture 230 treatments in late-stage clinical trails and create more than 12,000 jobs per year in the process, according to a new report from the Northern California biotechnology trade association BayBio. The report warns, though, that the State of California has no long-term strategy to capture what the group called a “massive, unprecedented economic opportunity.” It called on California to take action in the areas of tax policy, infrastructure, education, and regulatory affairs to help capture the return on California’s three decades of investment in life sciences research.

 

RNAI: USPTO ALLOWS OF KEY PATENT FOR INTRADIGM
Palo Alto, California-based Intradigm, a leading developer of targeted, systemic RNA interference or RNAi therapeutics, said the United States Patent and Trademark Office has allowed a key patent that claims methods of enhancing the RNA silencing activity of an RNAi agent in a mammalian or plant cell. The allowed subject matter is not only focused on claims to siRNAs, but also includes specific coverage for micro RNAs (miRNAs), miRNA precursors, primary miRNA transcripts and short hairpin RNA (shRNA). The allowed application is one of several applications disclosing certain efficacy-enhancing structural elements of RNAis that Intradigm exclusively licensed from the University of Massachusetts Medical School.

 

San Francisco-based The J. David Gladstone Institutes, an independent non-profit biomedical research organization, has entered into a major research collaboration and licensing agreement with iZumi Bio to focus on applications for induced pluripotent stem cells or iPS. The iPS cells are reprogrammed adult cells that have similar properties and potential to those of human embryonic stem cells, yet do not raise the same ethical concerns and can be generated in a patient-specific manner to avoid immune rejection.

Elements of the broad partnership include iZumi Bio taking a license to certain Gladstone patents and sponsoring research related to iPS cells and cardiovascular disease. Deepak Srivastava, director of the Gladstone Institute of Cardiovascular Disease, will lead the research and join the iZumi Bio scientific advisory board.

 

REGENERATIVE MEDICINE: RESEARCHERS IDENTIFY KEY REGULATORY PATHWAYS THAT CONTROL REPAIR AND REPLACEMENT OF DAMAGED TISSUE
In a study published in an online issue of the journal Nature, researchers at the University of California, Berkeley identified two key regulatory pathways that control how well adult stem cells repair and replace damaged tissue. The researchers then tweaked how those stem cells reacted to those biochemical signals to revive the ability of muscle tissue in old mice to repair itself nearly as well as the muscle in the mice's much younger counterparts. Because the findings relate to adult stem cells that reside in existing tissue, the researchers said this approach to rejuvenating degenerating muscle eliminates the ethical and medical complications associated with transplanting tissues grown from embryonic stem cells. They believe it could set the path for research on new treatments for age-related degenerative conditions such as muscle atrophy or Alzheimer’s and Parkinson’s diseases. The researchers focused on the interplay of two competing molecular pathways that control the stem cells, which sit next to the mature, differentiated cells that make up our working body parts. When the mature cells are damaged or wear out, the stem cells are called into action to begin the process of rebuilding. The researchers, however, warn against interpreting this research as the cure-all for aging.

 

INFECTIOUS DISEASE: GSK SIGNS SUPERBUG DRUG ALLIANCE WITH MPEX PHARMACEUTICALS
San Diego-based Mpex Pharmaceuticals and GlaxoSmithKline said that they have entered into a worldwide strategic alliance for the discovery, development, and commercialization of novel medicines for bacterial diseases. The collaboration provides GSK with access to Mpex’ novel efflux pump inhibitors (EPI) and related proprietary technology for use in combination with a variety of antibiotics. Bacterial efflux pumps are an intrinsic defense mechanism in gram-negative bacteria that expel toxins such as antibiotics that have permeated the outer-cell membrane before they can reach their targets and kill the bacteria. Higher intracellular antibiotic concentrations achieved through efflux pump inhibition can increase the potency and pharmacological barrier against other drug resistance mechanisms (e.g., target-based mutations). There are currently no drugs approved to inhibit the activity of these efflux pumps and restore the potency of otherwise effective antibiotics. The collaboration will focus on the discovery and development of novel drug regimens comprising Mpex’ EPIs combined with GSK’s novel development stage compounds as well as existing commercial antibiotics to improve potency and broaden the spectrum of antibacterial activity. Under the terms of the agreement, Mpex will grant GSK rights to product candidates developed under the collaboration that are directed to three different target product profiles and with the potential to deliver up to seven treatment options. Mpex will receive an $8.5-million upfront payment and a $6.5-million equity financing commitment from GSK. Mpex is eligible to receive development, regulatory and commercial milestones ranging up to $200 to $250 million for each product candidate.

 

Vertex Pharmaceuticals and the Los Angeles-based CHDI Foundation said they have established a collaboration aimed at developing assays for use in discovering novel compounds for the treatment of Huntington’s disease, a genetic neurodegenerative disease that affects approximately 30,000 people in the United States. CHDI is a non-profit foundation committed to accelerating the discovery and development of new drugs that delay the onset or slow the progression of Huntington’s disease. As part of the collaboration, CHDI will provide Vertex up to approximately $4 million of research funding through the first quarter of 2010. Under the agreement, Vertex will work closely with CHDI using those elements of CHDI’s extensive network of academic researchers and Huntington’s disease research assets that have direct relevance to this collaboration. Vertex will retain rights to all discoveries that are the result of the collaboration and will license related tools back to CHDI for use in further Huntington’s disease research. Researchers at Vertex’s San Diego-based research and development site will conduct the majority of the Huntington’s disease research using protein folding technologies and capabilities unique to that facility.

THYROID CANCER: EXELIXIS REACHES AGREEMENT WITH FDA ON SPECIAL PROTOCOL ASSESSMENT FOR PIVOTAL LATE-STAGE TRIAL OF COMPOUND
South San Francisco-based Exelixis said it has reached an agreement with the U.S. Food and Drug Administration on the late-stage registration trial of XL184, a small molecule anticancer compound targeting MET, RET, and VEGFR2, via the agency’s special protocol assessment process. XL184 inhibits MET, RET, and VEGFR2, which are key drivers of tumor growth, metastasis, survival, and angiogenesis. Exelixis said it has also discussed the trial design with European regulatory agencies. Exelixis is planning to initiate the late-stage clinical trial of XL184 as a potential treatment for medullary thyroid cancer this summer. This will be a randomized, placebo-controlled, double-blinded study of XL184 as single-agent therapy in 315 patients with unresectable, locally advanced, or metastatic MTC. The primary endpoint will be duration of progression-free survival. It is expected that approximately 100 sites in 20 countries will participate in this study. The planned initiation of this trial is based on encouraging data that were presented earlier this month at the 2008 annual meeting of the American Society of Clinical Oncology.

 

Emeryville, California-based Bionovo said results from in-vitro studies in neurons of its drug candidate MF101 showed that in neurons expressing native estrogen receptors differentiated from mouse stem cells, it’s drug candidate regulated calcium levels, which care related to temperature regulation. The data, presented at the Endocrine Society’s 90th Annual Meeting, showed that changes in calcium levels in neurons, an important biochemical pathway to temperature regulation, are mediated selectively by the activation of estrogen receptor beta. MF101 demonstrated this selective regulation. The study was conducted by investigators at The Center for Reproductive Sciences in the Department of Obstetrics, Gynecology and Reproductive Sciences at the University of California, San Francisco in collaboration with scientists at Bionovo. The company said that in a mid-stage clinical trial, MF101 was effective in the treatment of hot flashes. But unlike estrogen, it did not adversely affect the uterus.

IMMUNOLOGY: LA JOLLA INSTITUTE DISCOVERS NEW CELLULAR MECHANISM
San Diego-based La Jolla Institute for Allergy & Immunology scientists have discovered a previously unknown mechanism in how the body fights a virus. The finding, reported in the journal Immunity, runs counter to traditional scientific understanding of this process and will provide scientists a more effective method for developing vaccines, the researchers said. The research grew out of an effort to determine why there are good antibody responses to some parts of pathogens, but not others. It was known previously that getting a good antibody response requires two different cells of the immune system—B cells and CD4 T cells, both soldiers in the immune system’s defensive army. Antibody responses are important because they help the body fight off viruses and they also are critical to vaccine development. Surprisingly, the researchers found that B cells and CD4 T cells recognize the same piece of the virus. Previously, it was thought that the CD4 T cell could react to any part of the virus, but now we realize it must be specific to the same part as the B cell. The researchers said scientists will now be able to more easily figure out the most important viral pieces to focus on in developing a vaccine—the pieces that elicit a response from both the B cells and CD4 T cells.

 

BIOFUELS: MENDEL BIOTECHNOLOGY AND MMR GENETICS ANNOUNCE SORGHUM COLLABORATION

Hayward, California-based Mendel Biotechnology said it entered into a collaborative agreement with MMR Genetics and Richardson Seed to jointly research, develop, and breed new cultivars for the emerging second-generation biofuels market. MMR Genetics and Mendel will jointly establish a bioenergy nursery, which will be used to breed new, high-performing sorghum cultivars with unique biofuels characteristics. Richardson Seeds will produce commercial quantities of selected cultivars. Under the terms of the agreement, MMR Genetics will contribute germplasm, breeding know-how and services, and nursery operations; Mendel will provide funding and research and breeding assistance and will have exclusive commercial rights to selected cultivars. Richardson Seeds will provide seed production research and produce commercial quantities of selected cultivars. The financial terms were not disclosed.