BIOCOM Builds Outreach Efforts

With 2009 being BIOCOM's Year of the Member, we want to really drive the point home that this association is for and about its members. I travel regularly on behalf of our membership, spreading the good word about the efforts of our Southern California life science community, and we want to make sure we do everything we can to raise the visibility of this industry and its efforts to improve healthcare and quality of life.

With today's edition of the BCQ (our nickname for the Biocommunique), we're launching three new Web site features to help ensure our readers and members are up to date on news. First off, each month, we're going to focus on a disease area that our member companies are working in, giving a simple overview of what our companies are doing to advance the cause of healthcare. I'm sure many of you are familiar with our Deals and Data column, which provides an one-stop shop for news from our industry members; the monthly disease focus will be a quick way to see what our members are doing in specific disease areas that we will have accessible on the "About Southern California" section of our Web site.

Second, we are launching our new CEO Corner, which will be a place where I will communicate our CEO-level advocacy efforts, activities and the tidbits we see in our travels, including brief observations on the JP Morgan conference, the inauguration and other musings. Traditionally, we haven't disclosed much publicly about our CEO-level events, but this will be a place where we can have information on those activities and interviews from our member company CEOs. This month, we're excited to have a few quick questions from Randy Woods, our incoming board chair, and a letter from me welcoming everyone to our Web site. Feel free to email CEOCorner@biocom.org with any comments.

Finally, we're going to be including links to more regular news items in all of our email blasts and communications with our membership, giving our readership a regular, less formal way to keep updated on the news from BIOCOM and the community. There's a lot of activity going on and articles written about our members; we want to make sure that everyone sees these stories.

It's no secret that 2009 is going to be a challenging time for every industry; we want you to know that BIOCOM is here doing its best to help our members in the life science community in their efforts to create innovative science and technology. We hope you come back to our Web site often to see how.

Regards,

Joe Panetta
President and CEO
BIOCOM

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Spotlight on Oncology

With this issue of the Biocommunique, BIOCOM is launching a new feature to take a monthly look at drugs and devices in development to treat that disease from member companies. Following our 2009 "Art of Research" Calendar, each month, we'll look at a new disease and our companies efforts in it, with the information eventually posted to the "About Southern California" section of our Web site.

For our first disease spotlight, we'll take a look at companies with products and devices in development to treat cancer. There are more than 65 companies in Southern California. However, this year, more than 560,000 Americans will die of cancer, making it the second most common cause of death. In addition, more than 1.4 million people will be diagnosed with it. With the American Cancer Society estimating the disease is likely to kill one in four Americans, nearly everyone’s life is touched by cancer.

Please click here to download a LifeLines magazine PDF with a cover story focusing on oncology efforts in Southern California.

The following companies responded to our request for summaries of their oncology efforts. Is your company not on the list? Please email Kira Jenkins and we'll update you on our site.

Next month's spotlight will be on companies working to combat diseases of the heart. Is your company on the list? Please contact Kira Jenkins or Tim Ingersoll with any questions or to make sure your company is on the list.

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Spotlights

Adventrx Pharmaceuticals is a biopharmaceutical company focused on in-licensing, developing and commercializing proprietary product candidates primarily for the treatment of cancer and infectious disease. The company seeks to improve the performance and commercial potential of existing treatments by addressing problems associated with these treatment regimens.

Adventrx is currently focused on commercializing two late-stage product candidates in the U.S., both of which are reformulations of currently approved products and are designed to improve the safety profile of the approved product without affecting its efficacy. ANX-514 (docetaxel emulsion) is a reformulation of the blockbuster chemotherapeutic agent, Taxotere. In 2007, the aggregate worldwide market for Taxotere was in excess of $3 billion. ANX-530 (vinorelbine emulsion) is a reformulation of Navelbine which, despite being a generic product for a number of years, still sells in excess of $200 million a year world-wide. Both of the company’s product candidates have the potential to be on the market in 2010.

Althea is working to integrate molecular diagnostics into oncology both as diagnostic products and as markers in clinical trials to identify which patients will respond to each therapeutic option. Cancer is a disease caused by genetic destabilization and, as a consequence, the genetic state of a tumor can vary dramatically from individual to individual. It is therefore no coincidence that there is no magic drug to cover all cancer cases. Althea works directly with drug developers to identify and integrate DNA and RNA-based genetic markers that can identify the specific patient populations that are most likely to respond to their therapy. These markers help to create more efficient clinical trials involving fewer individuals and can dramatically improve the response rates to the selected therapy. Upon successful approval of the drug the biomarkers can be launched as companion diagnostics. In addition to its pharmaceutical services, Althea is also working on the development of novel diagnostics in prostate cancer that can be used to more accurately diagnose the disease and to help guide therapy by identifying patients that have a more aggressive form of the disease.

bioTheranostics discovers, develops, and commercializes new molecular diagnostic (MDx) tests in oncology, enabling physicians to personalize cancer treatment through better understanding of the molecular biology underlying a patient’s tumor. bioTheranostics currently markets two MDx assays: THEROS CancerTYPE ID (CTID) improves cancer classification, especially in patients diagnosed with metastatic cancer where the origin is uncertain or unknown. Studies have demonstrated that CTID quickly and accurately predicts cancer type and origin with an overall success rate of 86 percent, and a success rate of over 90 percent for 22 cancer types. Ongoing research is exploring patients’ therapeutic response given correct tumor classification. The company’s second diagnostic test, THEROS Breast Cancer Index (BCI), combines its H/I and MGI assays to provide a more complete picture of recurrence risk and therapeutic options for patients with ER-positive, node-negative breast cancer. Ongoing studies are exploring the ability of Theros BCI to predict individual patient response to various therapeutic regimens. bioTheranostics has several MDx tests in development, including an assay to assist with prostate cancer profiling. This test is designed to provide non-subjective, quantitative data that will help physicians better define indolent vs. aggressive cases.

In 2008, Celgene Corporation continued advancing clinical programs for its marketed therapies, as well as its deep pipeline in the areas of hematology and oncology. Revlimid, its lead product for previously-treated multiple myeloma is currently being studied in a number of additional indications including newly-diagnosed multiple myeloma where studies demonstrated an unprecedented three-year survival rate. In non-Hodgkin’s lymphoma, Revlimid was selected by the premier international lymphoma cooperative group, GELA, for a Phase III trial in diffuse large b-cell lymphoma in first-line maintenance. The therapy has also received approval for two Phase III special protocol assessment trials in chronic lymphocytic leukemia. Additionally, Vidaza, the company’s newest marketed therapy, demonstrated a 9.4 month advantage in overall survival compared to conventional care regimens in an international Phase III study. The company’s deep pipeline of compounds also advanced as pomalidomide, the most recent IMiDs compound demonstrated promising activity and manageable safety in previously treated myeloma and myelofibrosis. In addition, Amrubicin, the company’s candidate for small-cell lung cancer received orphan drug designation in the United States and Europe. Finally, research collaborations advanced as ACE-011, a compound being developed in conjunction with Acceleron Pharma entered Phase II research in bone loss associated with multiple myeloma.

Cylene Pharmaceuticals, Inc. is a Phase II-stage small-molecule oncology company that was co-founded by Daniel Von Hoff, MD, one of the world’s leading oncology clinical investigators.•Cylene has created a diverse portfolio of product candidates using two proprietary discovery platforms: the SERTIB platform creates first-in-class inhibitors of Serine/Threonine protein kinases, and the Nucleolus Targeting Technology creates novel Nucleolus Targeting Agents (NTAs). Cylene’s Nucleolus Targeting Technology has generated Quarfloxin, a first in class agent in Phase II development for the treatment of carcinoid/neuroendocrine tumors. Quarfloxin and other NTAs disrupt the abnormal nucleolar functions of cancer cells, thereby eliminating a critical source of support for tumor cells and selectively causing cell death. Another NTA, CX-5461, is a compound designed as an oral inhibitor of the RNA polymerase I complex, which is found exclusively in the nucleolus and is deregulated in cancer cells. Cylene’s SERTIB kinase inhibitor platform has generated the molecule CX-4945, as a highly-selective oral inhibitor of CK2, a protein kinase that promotes cell survival and is directly linked to tumorigenesis. CX-4945 is in Phase I clinical development against CK2-driven tumors. Additionally, the SERTIB platform has delivered CX-5011, another CK2 inhibitor, and CX-6258, an oral inhibitor of the PIM-1,2,3 kinases.

CytRx Oncology has a rich pipeline of clinical candidates exemplified by tamibarotene and INNO-206. Tamibarotene is a synthetic retinoid designed to avoid toxic side effects of ATRA, the current first-line APL treatment. FDA has granted Orphan Drug and Fast Track Designations for tamibarotene in patients with relapsed or refractory APL. CytRx is actively enrolling patients in a Phase 2 trial. In addition, we are evaluating strategies for developing tamibarotene as front-line APL therapy. INNO-206 is a novel targetable pro-drug for the commonly prescribed chemotherapeutic doxorubicin. It works by reacting with Cysteine-34 of human albumin but this linkage is acid sensitive, which is usually the case in the tumor microenvironment. Both preclinical and clinical data suggested a better safety profile of INNO-206 compared to doxorubicin. CytRx believes that it could be effective in a wide variety of cancers.

Gen-Probe’s focus in Oncology has been prostate cancer, the second leading cancer in men WW. Several publications of its initial molecular marker, PCA3, have shown that the PCA3 score from post-DRE urine can be used to predict likelihood of a positive prostate biopsy and can be correlated with tumor volume, stage and Gleason Score. Additional clinical research is underway to confirm its prognostic value, assess PCA3 in combination with other markers and to expand clinical utilities (e.g., therapeutic monitoring). A second-generation multiplex PCa3 assay is also in development on a new, fully automated instrument. Gen-Probe has also begun research and development of an assay for the gene fusions of TMPRSS2:ERG. Early research studies have demonstrated that such fusions may be associated with more “aggressive” neoplasms, and are highly specific to prostate cancer. Because the fusion is one of an androgen gene to an oncogenic gene, current research is ongoing to evaluate its utility for prognosis and treatment selection and monitoring. The Gen-Probe oncology pipeline is also exploring next-generation multiplexing technologies to facilitate creation of biomarker panels with greater diagnostic accuracy to help in the management of prostate cancer.

Ichor Medical Systems is dedicated to the clinical application and commercialization of its electroporation technology for the delivery of DNA therapeutics and vaccines to treat and prevent debilitating or life threatening diseases such as cancer and chronic viral infections. Ichor's TriGrid Delivery System integrates several patented technologies into an automated handheld device enabling safe, efficient, and reproducible administration that requires minimal clinical training (see short movie at http://www.ichorms.com/movies/Ichor.swf). In collaboration with the Memorial Sloan-Kettering Cancer Center (MSKCC), a Phase I clinical evaluation of TriGrid electroporation based delivery of a DNA vaccine is underway in patients with Stage IIB-IV melanoma. A second Phase I clinical trial, in collaboration with the Aaron Diamonds AIDS Research Center for a preventative HIV DNA vaccine delivered by the TriGrid system, has completed subject recruitment. Initial data shows clinical acceptance and safety of the procedure. Ichor and its partners have several additional DNA vaccine candidates moving toward clinical investigation in the coming year, for both prophylactic and therapeutic indications.

IDM Pharma is focused on the development of innovative cancer products that either destroy cancer cells by activating the immune system or prevent tumor recurrence by triggering a specific adaptive immune response. The company’s lead product, mifamurtide (L-MTP-PE), recently received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) recommending grant of a centralized marketing authorization or approval for L-MTP-PE by the European Commission for the treatment of patients with non-metastatic, resectable osteosarcoma, a rare and often fatal bone tumor that typically affects children and young adults. The European Commission approval is expected in the first quarter of this year and L-MTP-PE will be the first approved new treatment in more than 20 years for patients with osteosarcoma. Results from the Phase 3 trial, which was the largest study completed in osteosarcoma, enrolling approximately 800 patients, demonstrated that overall survival after six years of follow-up in patients treated with chemotherapy and L-MTP-PE was 78 percent, compared to 70 percent in patients treated with chemotherapy alone. The addition of L-MTP-PE to chemotherapy resulted in approximately a 30 percent decrease in the risk of death.

Inovio Biomedical is focused on developing DNA vaccines for cancers and infectious diseases using its novel electroporation-based DNA delivery technology, which uses brief, controlled electrical pulses to enhance cellular uptake and immunogenicity of DNA vaccines. Merck is conducting a Phase I clinical study using Inovio’s technology to deliver a DNA vaccine encoding hTERT, targeting non-small cell lung and prostate cancers. Final results from a Phase I clinical study conducted by Moffitt Cancer Center showed that a DNA-based immunotherapy encoding for IL-12 was safe, tolerable, demonstrated CD4+ and CD8+ lymphocytic infiltrate in the treated lesions, and showed durable partial and complete tumor regression in locally treated and distant untreated metastatic melanoma lesions, suggesting a systemic response. Interim data from a Phase I/II study conducted by the University of Southampton has shown that a DNA vaccine encoding for PSMA27 against prostate cancer, delivered using electroporation, has been safe and well-tolerated, and induced significant increases in antibody and anti-DOM CD4 responses. Inovio’s technology is protected by an extensive patent portfolio covering in vivo electroporation.

MabVax Therapeutics, Inc. is an immunotherapeutics discovery and development company focused on developing and commercializing novel vaccines and human antibodies for the treatment of cancer. Last year, MabVax completed two licensing agreements with Sloan-Kettering Institute for Cancer Research, the research arm of Memorial Sloan-Kettering Cancer Center (MSKCC), for exclusive development and commercialization rights to multiple vaccines against selected recurrent cancers. MSKCC has already initiated Phase I clinical trials with some of the vaccines to demonstrate immunogenicity and tolerability. MabVax plans to enter Phase II clinical trials with the licensed vaccines against sarcoma and small cell lung cancer in 2009. The NIH, through the Gynecologic Oncology Group consortium is sponsoring yet another Phase II trial of a licensed vaccine to begin in 2009 as well. Concurrently, MabVax will use its expertise to isolate anticancer antibodies from immunized patients participating in the vaccine clinical trials resulting in a pipeline of fully human monoclonal antibody products. MabVax is working to address a significant unmet medical need, to prevent recurrence in certain types of cancers and prolong patient survival, by targeting residual circulating cancer cells and micrometastasis with passively administered or vaccine induced antibodies. For additional information, contact David Hansen, President and CEO, at dhansen@mabvax.com.

Nereus Pharmaceuticals pursues novel sources of chemical diversity to discover and develop new therapeutics. Using its unmatched expertise in marine microbiology to identify unique biologically active compounds, Nereus has two oncology drug candidates in multiple Phase 1 and Phase 2 clinical trials. NPI-2358 is a potent, selective tumor vascular disrupting agent (VDA) with best-in-class characteristics that is an analogue of a molecule with novel chemistry and activity derived from a marine fungus. NPI-2358 was successfully evaluated in a Phase 1 clinical trial and demonstrated an advantageous safety profile and therapeutic window along with an excellent pharmacodynamic effect.• NPI-2358 is being evaluated in a randomized Phase 2 study in combination with docetaxel in patients with NSCLC having failed prior therapy.• NPI-0052 is a highly potent potentially best in class proteasome inhibitor derived from a novel marine actinomycete. NPI-0052 is being evaluated for the treatment of multiple myeloma, solid tumors, lymphomas and leukemias in three Phase 1 clinical trials. NPI-0052 is also being evaluated in a Phase 1/2 clinical trial for the treatment of melanoma, pancreatic cancer, NSCLC, and NHL in combination chemotherapy with Zolinza. The company expects NPI-0052 to progress into Phase 2 studies in multiple indications during 2009. In addition to the two clinical programs, Nereus internal discovery portfolio includes potential drug candidates for cancer, infectious diseases and inflammation.

NovaRx Corporation is a late-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of novel cell-based therapeutic vaccines for the treatment of cancer. The company was originally founded in San Diego, California in May of 1997 in order to commercialize the award-winning research conducted between 1990 and 1997 by Dr. Habib Fakhrai and his associates at the Sidney Kimmel Cancer Centerin San Diego and at the UCLA School of Medicinein Los Angeles. The proprietary core technology has been exclusively licensed to NovaRx on a worldwide basis. The company’s headquarters and GMP manufacturing operations are located in a state-of-the-art facility on Nancy Ridge Drive in San Diego. At present, the company has over 60 employees, nine of whom hold advanced scientific degrees. The company’s lead product candidate, Lucanix, a whole cell-based vaccine comprised of four allogeneic cell lines, is currently being tested in an international, double-blind, placebo-controlled pivotal Phase III clinical trial for the treatment of advanced non-small cell lung cancer . Future plans call for the company to initiate a Phase III clinical trial to test Glionix, an allogeneic therapeutic vaccine to treat advanced stage glioblastoma multiforme.

Regulus Therapeutics is a biopharmaceutical company created to discover, develop, and commercialize microRNA therapeutics. Greater than 700 microRNAs, a new category of RNA, have been identified that regulate the expression of an estimated 30 percent of all human genes. microRNAs control, in a dose-dependent manner, cellular states or responses rather than collections of functionally unrelated proteins. This feature makes these molecules attractive candidates or targets for future medical therapies. Regulus technology allows for the modulation of these natural mechanisms by either inhibiting or replacing microRNAs and thus has the potential to deliver a range of new therapies for multiple diseases. Unusual levels of various microRNAs for example have been shown to be diagnostic of pathology in a number of different cancers, such as cancers of the liver, prostate, lung and breast. Together with collaborators, we have identified microRNAs in several cancer types that provide beneficial effects when either inhibited or replaced and we are currently evaluating the potential of these candidates in preclinical models. Regulus is building a strong oncology program and plans to have drugs in clinical trials for cancer, as well as other indications, in the near future.

Tracon Pharmaceuticals is a biotechnology company developing targeted therapies for cancer and related diseases. The Tracon team initiated four clinical trials of three first-in-class molecules between July 2007 and June 2008, including two monoclonal antibodies for the treatment of cancer that reported Phase 1 data at the NCI-AACR-EORTC meeting in Geneva in October 2008 (the poster presentations are available online at www.traconpharma.com). Tracon’s drug candidates include: TRC093: a monoclonal antibody to cleaved collagen that blocks angiogenesis and tumor invasion. This antibody inhibits the growth of human melanoma, breast and pancreatic cancers and improves the activity of chemotherapy and targeted agents in preclinical models. Phase 1 enrollment is nearly complete. TRC105: a monoclonal antibody that binds CD105, a protein expressed on the surface of endothelial cells that is essential for angiogenesis. TRC105 is expected to address multiple solid tumor types including breast and lung cancer, as well as age-related macular degeneration (AMD).•Phase 1 enrollment is nearly complete. TRC102: a base excision repair inhibitor that reverses resistance to chemotherapy, including agents used in the treatment of melanoma and lung cancer.•Two Phase 1 studies are ongoing, one in combination with Temodar and a second in combination with Alimta.

VentiRx Pharmaceuticals Inc. is a clinical stage biopharmaceutical company committed to the development and commercialization of novel medicines for the treatment of cancer, respiratory, autoimmune/inflammation and skin diseases. Since its inception, VentiRx has been able to position itself in the forefront of the of toll-like receptor (TLR) therapeutics with a clear focus in the small molecule TLR8 agonists and antagonists for the treatment of serious diseases. VentiRx currently has 2 distinct TLR8 programs in development and expect to quickly expand its TLR8 franchise into additional therapeutic areas via strategic partnerships. In November 2008, we announced the initiation of a Phase I study with VTX-2337 in cancer patients in 2 clinical sites in the US. In addition, we continue to advance VTX1463 for allergic rhinitis towards the clinic. Our goal is to commence clinical trials with VTX-1463 in the first half of 2009. Founded in June 2006 by Michael Kamdar and Robert Hershberg, VentiRx has raised $28.9 million in funding from ARCH Venture Partners, Domain Associates, and Frazier Healthcare Ventures. VentiRx is a privately held organization headquartered in San Diego with operations in Seattle.

Vical Incorporated is currently conducting a pivotal Phase 3 trial comparing Allovectin-7 with physician’s choice of dacarbazine (DTIC) or temozolomide (TMZ). The Allovectin-7 Immunotherapeutic for Metastatic Melanoma (AIMM) study is currently recruiting subjects in 12 countries. This trial includes chemotherapy naïve patients with at least one cutaneous, subcutaneous, or nodal melanoma lesion, normal LDH, ECOG performance status of 0-1, and melanoma stage III-IV 1b.The primary endpoint is to compare durable response rate at ≥24 weeks after randomization in the Allovectin-7® arm versus the control (DTIC/TMZ) arm. Approximately 375 subjects with metastatic melanoma will be randomized in a 2:1 allocation to receive Allovectin-7 (~250 subjects) or physician’s choice of DTIC or TMZ (~125 subjects). The treatment course recommended for patients who receive Allovectin-7 is a minimum of 16 weeks. Each cycle consists of weekly injections of Allovectin-7 for six weeks followed by two weeks of observation and assessments. For patients who receive the chemotherapy, the treatment course will follow standard dosing. During the trial all patients' tumors will be closely monitored. Patients whose melanoma does not clinically progress will be encouraged to continue on the treatment and be assessed for up to two years. Additional information is available at www.vical.com.

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