• BIO Announces San Diego to Host the World’s Largest Industrial Biotechnology Conference in 2016

    Washington, DC (September 8, 2015) – The Biotechnology Industry Organization (BIO) today officially announced that logo_BIOthe 2016 World Congress on Industrial Biotechnology will be held April 17-20, 2016 in San Diego, California at the San Diego Convention Center. BIO is also announcing the call for papers for the event. Leaders from the biotechnology, bioenergy, chemical, consumer products manufacturing, and agricultural industries as well as academia and financial sectors are invited to submit proposals and individual papers beginning September 17, 2015.  

    “More than 400 biotechnology companies call San Diego their “home,” which makes it an exciting venue to host the world’s largest conference dedicated to promoting next-generation biobased products, renewable chemicals and biofuels,” stated Brent Erickson, Executive Vice President, Industrial and Environmental at BIO. “With California’s growing industrial biotech sector, BIO is excited to host the 2016 World Congress on Industrial Biotechnology in the center of it all.”

    The 2015 World Congress on Industrial Biotechnology, held in Montréal, drew 1,200 industry leaders from 725 companies, 50 countries and 37 states, as well as the District of Columbia. Further, the 2015 World Congress hosted more than 1,400 partnering meetings, a 40% increase from the 2014 BIO World Congress.

    The call for papers will open September 17 to invite submissions for breakout panels, breakout papers, posters, and Green Tech investor sessions. Leaders from the biotechnology industry, academia, and policy community are invited to offer an abstract or a proposal for a 15 minute presentation in any of the following program tracks:

    • Algae, Biomass Supply and Specialty Crops
    • Specialty Chemicals, Food & Nutritional Ingredients
    • Synthetic Biology and Genomics Research
    • Research Presentations
    • Technical Presentations
    • Renewable Chemicals and Biobased Materials
    • Advanced Biofuels and Biorefinery Platforms
    • Growing Global Biobased Markets

    Proposed papers and presentations should address any in a range of topics, including renewable chemicals, algae for fuels and bioproducts, and chemicals, biobased products, biomass utilization, cellulosic biofuels, metabolic engineering, new chemical pathways, regional economic development, synthetic biology, regulatory issues, new feedstock crops and feedstock sustainability, aviation biofuels, genetically engineered crops, project finance, biopathways to bulk chemicals, enzyme development, green plastics, work force training and specialty chemicals. 

    For more information on the conference or to submit a proposal, please visit http://www.bio.org/worldcongress

    For assistance, please contact worldcongress@bio.org.

  • Pathway Genomics Launches First Liquid Biopsy Test To Detect Cancer-Associated Mutations In High-Risk Patients

     
    pathway-logo1CancerIntercept-Detect-Monitor_header_-600x202

     

     

     

    SAN DIEGO – Sept. 10, 2015 – Pathway Genomics, a global precision medical diagnostics company, announced the launch of CancerInterceptTM, its first liquid biopsy, a non-invasive screening test designed for early cancer detection and monitoring, for as low as $299. 

     

    The test detects mutations that are commonly associated with lung, breast, ovarian, colorectal cancers and melanoma, as well as mutations that occur less frequently in other cancer types (such as pancreatic, head and neck, thyroid, gastric and prostate cancers). View an animation of howCancerIntercept works here.

    The test is offered for two general populations: CancerInterceptTM Detect is the first liquid biopsy designed to detect tumor DNA in high-risk but otherwise healthy patients; CancerInterceptTMMonitor monitors patients with active or previously diagnosed cancer. Both programs use advanced DNA analysis to identify small DNA fragments that are shed from cancer cells and released into the bloodstream. The tests analyze the presence of 96 frequently occurring DNA mutations in nine cancer genes.

    “Early detection is the single most important factor in ensuring successful treatments and improved survival rates,” said Jim Plante, CEO and founder of Pathway Genomics. “Cancer patients and those at risk for the disease can take proactive steps to safeguard their health and fight back against some of the most virulent forms of the disease.”

    In addition, with CancerIntercept Monitor, physicians are able to supplement more invasive tissue biopsies and scans with liquid biopsies to monitor cancer treatment efficacy, disease progression and recurrence. CancerIntercept Monitor can also be ordered with personalized Clinical Trial Matching for later stage cancer patients.

    “Rising levels of tumor DNA may indicate progression of the cancer before there is clinical or imaging evidence of tumor growth” said Dr. Glenn Braunstein, MD and Chief Medical Officer of Pathway Genomics. “Our liquid biopsy tests may also detect new mutations that occur over time and signal that the patient is becoming resistant to current therapy.”

    Testing can be initiated through the patient’s treating physician or through Pathway’s online physician referral network. For patients and physicians requesting repeat testing on a scheduled basis, a deeply discounted subscription service is available.

    CancerIntercept Detect and CancerIntercept Monitor are offered through Pathway’s integrated system, which streamlines the entire testing process from initial order to delivery of test reports. Key components of the system include physicians who will review online requisitions and order the tests; mobile phlebotomists to draw blood samples at the patient’s home or office; and Pathway’s medical oncology support team, who will discuss results with the patient’s treating physician upon a positive result. All positive results are released via the patient’s treating physician. Results are delivered approximately two to three weeks after testing.

    About Pathway Genomics

    Since its founding in 2008, Pathway Genomics has rapidly become a leader in the commercial healthcare industry. Pathway Genomics’ success lies in its commitment to deliver innovative healthcare solutions. The company’s program with IBM Watson is the first of its kind. The program is a smartphone app that merges artificial intelligence and deep learning with personal genetic information. The app provides users with personalized health and wellness information based on the individual’s health history.

    Based in San Diego, the company’s CLIA and CAP accredited clinical laboratory provides physicians and their patients in more than 40 different countries with actionable and accurate precision healthcare information to improve, or maintain, health and wellness. Pathway Genomics’ testing services cover a variety of conditions including cancer riskcardiac healthinherited diseasesnutrition and exercise response, as well as drug response for specific medications including those used in pain management and mental health. For more about Pathway Genomics, visit www.pathway.com or follow us on TwitterLinkedIn and Facebook.

     

    Read SD Union Tribune article:Sdut-logo

     

    New blood test helps detect cancer early

     

     

     

     

     

     

  • Celgene buys Receptos for $7.3 billion

     

    In one of the largest sales of a San Diego County biotech company to date, Receptos has agreed to a $7.32 billion cash purchase by Celgene, a New Jersey-based cancer drug maker with an office in San Diego.

    Celgene will gain a much-anticipated drug in clinical testing by Receptors for multiple sclerosis and ulcerative colitis. The drug, called ozanimod, is in a Phase 3 trial for relapsing multiple sclerosis.

    Formerly called RPC1063, the drug's potential has helped propel Receptos into becoming a multibillion-dollar company since its initial public offering in 2013. Receptos shares have surged more than fivefold over the last year as ozanimod progressed in clinical testing.

    The company agreed to pay $232 per share for Receptos, a 12 percent premium from Tuesday's closing price. The deal is expected to close this year.

    Receptos and Celgene valued the deal at $7.2 billion excluding Receptos' cash on hand. That's larger than all but a few local biotech purchases, the biggest of which was the $13.6 billion purchase of Carlsbad's Life Technologies by Thermo Fisher Scientific in 2014.

    Faheem Hasnain, Receptos' CEO, is highly regarded in the industry. Before Receptos, Hasnain led Facet Biotech, sold for $450 million cash to Abbott Laboratories in March 2010.

    Facet developed Zenapax, an injectable drug for multiple sclerosis. The Receptos drug is taken orally.

    Celgene, based in Summit, New Jersey, employs several hundred people in San Diego, said spokesman Greg Geissman. The workforce performs early stage research on identifying potential new drugs.

    "We're very excited about this," Geissman said. "This is a great opportunity for us to continue to bolster this inflammation and immunology franchise."

    Receptos has 68 employees, according to a profile on Yahoo Finance. Celgene is interested in keeping Receptos employees, but it's not clear how many will be retained, Geissman said.

    Receptos reported about $2 billion in first-quarter sales of its drugs, which include the multiple myeloma treatments Revlimid and Pomalyst and breast and lung cancer drug Abraxane.

    Revlimid, its top seller, is also used to treat severe anemia and mantle cell lymphoma.

    The purchase of Receptos is part of a push to boost its sales of treatments for inflammatory and immune diseases. In 2014 Celgene launched its first drug in that category, the psoriasis and psoriatic arthritis treatment Otezla.

    Receptos doesn't have any approved drugs on the market. The company expects results from late-stage trials of ozanimod in 2017 and 2018 and hopes to get the drug approved as a treatment for multiple sclerosis in 2018.

    In addition to ulcerative colitis, Receptos is also studying ozanimod as a treatment for inflammatory bowel disease. The companies said they think its annual sales could reach $6 billion.

    In extended trading, Receptos shares added 10 percent, or $21.17, to $228.35. The stock closed at $37.75 a year ago.

    Celgene shares rose nearly 6 percent, or $7.07, to $129.92 in late trading. Last month Celgene agreed to invest about $1 billion in drug developer June Therapeutics Inc., mostly by buying stock. The companies plan to develop cancer and autoimmune disease treatments.

    Source: UT San Diego By Bradley J. Fikes | 7:10 p.m. July 14, 2015

  • Illumina Non-Invasive verifi® Prenatal Test May Reflect Presence of Maternal Cancer

    Study of Illumina's Non-Invasive verifi® Prenatal Test Suggests that Test Results, When Discordant from Fetal Karyotyping, May Reflect Presence of Maternal Cancer

    – Results Published in Journal of the American Medical Association –

    SAN DIEGO–(BUSINESS WIRE)–

    Illumina, Inc. (ILMN) today announced that a preliminary study retrospectively correlated 10 cases of occult maternal cancer among pregnant women receiving an “aneuploidy detected” or “aneuploidy suspected” positive results on the verifi® noninvasive Prenatal Test (NIPT)1 to discordant results of fetal karyotypes for the women, suggesting that discordant NIPT test results may be indicative of maternal cancer.

    Maternal cancers sometimes leave tell-tale abnormal cell free DNA fragments in plasma. In 8 of 10 reported cancer cases, Illumina scientists and physicians reviewed all the genome-wide sequencing data and identified nonspecific copy-number changes across multiple chromosomes, suggesting that these changes, when present in a woman whose fetal karyotype tests as normal, might represent a signal to the clinician to probe for the presence of cancer.

    The results of the study, entitled “Noninvasive Prenatal Testing and Incidental Detection of Occult Malignancies,” are available online today in The Journal of the American Medical Association and the full article can be accessed at broadcast.jamanetwork.com. The paper will be included in the July 14 print edition of JAMA.

    “Abnormal tumor DNA, shed from maternal malignancies, can cause highly unusual NIPT results, including the findings of more than one chromosome abnormality detected,” commented Diana W. Bianchi, M.D., Executive Director of the Mother Infant Research Institute at Tufts Medical Center, and lead author of the publication. “All abnormal NIPT results should be confirmed with a diagnostic test, such as amniocentesis or chorionic villus sampling (CVS). If there is a difference between the fetal diagnostic test and the NIPT results, maternal cancer can be a rare but important underlying explanation. In order to provide the best maternal clinical care, this possibility should be considered, especially when multiple abnormalities of chromosome number are identified on the NIPT report.”

    In a retrospective analysis of 125,426 noninvasive verifi® Prenatal Tests, 3,757 (three percent) were positive for one or more aneuploidies involving chromosomes 13, 18, 21, X or Y. As part of Illumina’s standard procedures, the laboratory contacts the referring physician to discuss all positive test results and to recommend a diagnostic procedure to obtain a confirmatory fetal karyotype. From this group of 3757 positive cases, 10 cases of maternal cancers were subsequently (between 3 and 39 weeks after NIPT) reported to the laboratory. At the time of NIPT these women were not diagnosed with cancer. In 3 cases the abnormal NIPT findings prompted a search for an underlying malignancy. Seven of these eight women had diagnostic tests that indicated a chromosomally-normal fetus, discordant with the NIPT test result. One woman did not undergo a diagnostic procedure.

    Maternal cancers were most frequently associated with the rare NIPT finding of multiple aneuploidies that were discordant with the fetal karyotype. The cancer types were clinically diverse, including three cases of B cell lymphoma and single cases of T-cell leukemia, Hodgkin’s lymphoma, unspecified adenocarcinoma, leiomyosarcoma, and neuroendocrine, colorectal and anal carcinomas. The cancers ranged from stage II to metastatic disease (IV). In one case, after completion of treatment, the abnormal DNA signature became undetectable in follow-up testing.

    “This study shows that NIPTs that use whole genome sequencing techniques may have the ability to detect some cancer signatures under certain conditions,” said Dr. Rick Klausner, Illumina’s Chief Medical Officer. “The sensitivity and specificity of the verifi Prenatal Test to detect cancer is not currently known, and further studies will be required to develop a test for this specific purpose. We have published these results to help clinicians improve overall patient care by considering the possibility of maternal cancer if there is discordance between positive NIPT results and a normal fetal karyotype. We also want to encourage health care providers to contact the clinical sequencing laboratory with follow-up information on abnormal NIPT results that show concordance as well as discordance with fetal karyotype, including cancers diagnosed in pregnant women, so that we can better understand the nonspecific patterns of DNA changes that precede clinical symptoms.”

    About the verifi® Prenatal Test

    The Illumina verifi® Prenatal Test analyzes genetic material (cfDNA) from a pregnant woman’s blood to look for too few or too many copies of chromosomes in the mother and baby, or babies in the case of twins. Missing or extra copies of chromosomes are referred to as “aneuploidies” and may be related to conditions in pregnancy such as trisomy 21 (Down syndrome), trisomy 18 (Edwards syndrome), trisomy 13 (Patau syndrome) or sex chromosome (X and Y) associated conditions.

    Source: Results from Journal of the American Medical Association, published by BusinessWire

  • IPC & Innate Pharma collaborate on Translational Research for Novel Immune Checkpoint Inhibitors

    IPC, The comprehensive Cancer Center of Marseille, and Innate Pharma collaborate on Translational Research for Novel Immune Checkpoint Inhibitors

    The Paoli Calmettes Institute (IPC), a private not-for-profit comprehensive cancer center in Marseille, France, has initiated a collaboration with Innate Pharma SA (Euronext Paris: FR0010331421 – IPH), a biopharmaceutical company based in Marseille, to conduct translational research aimed at identifying specific populations of patients with hematological cancers who may benefit most from treatment with Innate Pharma’s novel proprietary antibodies, and to identify associated biomarkers.

    innate-logoNicolai Wagtmann, Chief Science Officer of Innate Pharma, said: “This agreement strengthens our translational research capabilities, at the core of our drug development process. IPC is a leading center in hematology-oncology with deep understanding in immunology and capabilities in immune monitoring. Early-stage testing of Innate Pharma’s promising antibodies on patient samples will greatly optimize subsequent clinical development by better identifying the most relevant indications, patient populations and biomarkers”.

    logo-ipc-bigPr. Patrice Viens, Chief Executive Officer of IPC, stated that “This new R&D collaboration with Innate Pharma further expands a long-lasting relationship which started with lirilumab, a fully human monoclonal antibody blocking the interaction between Killer-cell immunoglobulin-like receptors (KIR) on NK cells with their ligands on tumor cells. IPC conducted the “first-in-man study” of lirilumab and continues to be involved in the development of this drug. IPC and Innate Pharma are also involved in other partnerships within the frame of the Marseille SIRIC* and of Marseille Immunopole **”.

    The collaboration will operate under the direction of Pr. Daniel Olive, Head of the Immunity and Cancer research team and of the immune monitoring platform at IPC, and Pascale André, Senior Director, R&D, at Innate Pharma. It will also involve drug-discovery research teams and experts in translational research at IPC, including Pr. Norbert Vey and Pr. Anthony Gonçalvès, and other scientists with expertise in converting promising drug discoveries into clinical treatments for cancer patients.

    Under the terms of the collaboration agreement, IPC will test Innate Pharma’s new therapeutic antibodies in immuno-oncology using IPC’s extensive biological resource collection. Innate Pharma’s antibodies are designed to block immune checkpoints, one of the most promising classes of drugs in oncology. These immune checkpoints interfere with the natural defense mechanisms of our immune system against cancer and regulate immunosuppressive mechanisms. Their blockade can unleash the patient’s immune system to recognize and eliminate cancer cells. However, only a fraction of patients respond to current immune checkpoint inhibitors, and identifying the population of patients most likely to respond to various checkpoint blockers would be of great interest. This R&D collaboration will facilitate the development of novel therapeutic antibodies by identifying the best suited patient population and indications for a given drug.

    * The Marseille SIRIC is the site for integrated cancer research focused on breast cancer, leukemia and myelodystrophy, glioma and pancreatic cancer funded by the French National Cancer Institute, which brings together   IPC, the Assistance Publique – Hôpitaux de Marseille (AP-HM) and their associated research centers.

    ** Marseille Immunopole is a unique collaborative ecosystem for therapeutic innovation in immunology, integrating all the skills from academic to clinic. It aims to make Marseille the European capital of the R&D of immune-based therapy

  • AN UNEXPECTED SURPRISE: EUROPE TO MOVE AWAY FROM FDA-STYLE APPROVAL PROCESS FOR MEDICAL DEVICES

    European FlagsLast week provided breaking news in regards to a highly anticipated and much-talked about topic – the future of the medical device approval process in Europe.

    The 2011 breast implant controversy, where the French company PIP produced defective breast implants made from cheap industrial silicone, has since put the Medical Device Approval process in the EU under the microscope. As a result of this scrutiny, many had raised the question of whether a European-wide, “FDA” style approval process should be adopted for medical devices.

    With the unforeseen leak of the draft report from the Council of Europe on June 11th, it seems that the answer behind a proposal for centralisation is now a resounding ‘no.’ The proposed regulation  put forward by the Latvian presidency and corroborated by many of the Member States discussed the placing, making available and putting into service medical devices on the market. This will be seen as welcome news for American companies seeking EU commercialisation while they are undergoing the arduous FDA process.

    CE-markFor quite some time, it had been suggested that EU institutions should look into ways that would help tighten safety of medical devices. Some lawmakers had therefore felt that in order to solve the problem of a lax EU system, Europe needed a model which resembles the FDA; a US-centralized approval model rather than the current decentralized one, where each country sets its own legal and safety requirements, would be the only answer.

    It seems that leaving the decision and approval process in the hands of Notified Authorities is still plausible. As stated in Article 28 of the proposed regulation “a member state that intends to designate a conformity assessment body as a notified body […] to carry out conformity assessment activities […] shall nominate an authority […] for the assessment, designation and notification” of medical devices.

    This is further reiterated in Annex VIII allowing the nominated authority to still oversee and work with notified bodies, who will in turn work with manufacturers on the approval process.

    The outlined proposals in Chapter VI in regards to clinical evaluations and investigations also stress a more thorough process with the addition that “the Commission, in collaboration with the Member States, shall […] set up, manage and maintain an electronic system”.

    The discussions that took place on June 19th were set to begin the formal review of the proposal into the Trilogue between the Commission, EU Parliament and Member States.

    Although it may seem at first glance an approach to allow the ‘old ways’, it actually paves a way into making a leap from the current directive into differentiating itself from the pharmaceutical industry so that it is, in its own right, specific for medical devices.

    The proposal does not ‘shy away’ from a centralized system because of bureaucracy or financial implications, but merely that a bulky process like that of the FDA is not necessarily the correct model for the EU. It instead provides a means for tighter regulations without necessarily adding delays to its nimble approval process.

    Going forward, the Trilogue will most likely begin in September for proposed adoption by mid-2016 and full implementation in all Member States by 2019.

    SOURCE: http://www.medeuronet.com/medeuronet/an-unexpected-surprise-europe-to-move-away-from-fda-style-approval-process-for-medical-devices/

    June 22, 2015  by Dr. Sunni Patel, medeuronet clinical scientist 

  • 2015 BIO International Convention Closes in Philadelphia – New Record for Partnering Meetings & Back to San Diego in 2017

     

    logo-BIOBIO 2015 Sets Record – 29,279 Partnering Meetings in Philadelphia. Biotechnology Industry Organization to Become Biotechnology Innovation Organization in Early 2016. 15,858 Industry Leaders Attended World's Largest Biotech Gathering

    Philadelphia, PA (June 18, 2015) – The 2015 BIO International Convention, where the global biotech community meets, connecting the people, companies and innovations that help to fulfill the promise of biotechnology through healing, fueling, and feeding the world, concluded  today at the Pennsylvania Convention Center. This year’s Convention hosted 29,279 partnering meetings, a new record for the event. Organized by the Biotechnology Industry Organization (BIO), the event drew 15,858 industry leaders from 69 countries and 47 states, as well as the District of Columbia and Puerto Rico.

    “This year’s BIO International Convention was a tremendous success, bringing to Philadelphia leaders in industry, government and academia from across the globe to pursue biotechnology’s potential to solve some of society’s most pressing problems,” said BIO President & CEO Jim Greenwood.  “From our outstanding keynote speakers, to the record number of partnering meetings, the 2015 Convention was a resounding success.  This event continues to provide biotech leaders with the opportunity to showcase the best of our industry and form connections and partnerships that will drive growth in the sector for years to come.”

    BIO’s new Board Chair, Ron Cohen, CEO of Acorda Therapeutics, announced during Wednesday afternoon’s Keynote Luncheon that the Biotechnology Industry Organization would soon become the Biotechnology Innovation Organization, effective in early 2016.

    The Convention featured dynamic keynotes from legendary journalist Tom Brokaw, leading digital health expert Eric Topol and internationally acclaimed jazz musician Wynton Marsalis. Educational programming included over 750 speakers and 125 sessions in 16 educational tracks which addressed the latest business opportunities, breakthroughs in medicine, diagnostics, the environment, energy production, food and agriculture and more. For session highlights and editorial, visit www.biotech-now.org/.

    The top 15 largest international delegations included (in alphabetical order): Australia, Belgium, Brazil, Canada, China, France, Germany, India, Japan, Netherlands, South Korea, Spain, Switzerland, Taiwan, and the United Kingdom.

    Several U.S. Governors attended this year’s event including Pennsylvania Governor Tom Wolf, Delaware Governor Jack Markell, and South Dakota Governor Dennis Daugaard. New Jersey Lieutenant Governor Kim Guadagno was also in attendance as well as numerous legislators from around the country.

    The BIO Business Forum hosted a record-breaking 29,279 scheduled one-on-one partnering meetingsbetween more than 3,100 companies, and featured 207 company presentations. View highlights here.

    The BIO Exhibition featured more than 1,800 exhibitors and covered over 150,000 square feet with 47 state, regional and international pavilions. The Innovation Zone, featuring SBIR grantees from the National Institutes of Health (NIH) and the National Science Foundation (NSF), as well as the Digital Health Zone, both doubled in size this year.

    BIO would like to thank Philadelphia Mayor Michael Nutter and Pennsylvania Governor Tom Wolf for their hospitality and support leading up to and during the 2015 BIO International Convention. BIO also thanks our regional partners: BioNJ, the Delaware BioScience Association and Pennsylvania Bio.

    “With the support of BioNJ, the Delaware BioScience Association and Pennsylvania Bio, our tri-state region was able to make this year's BIO 2015 convention in Philadelphia a tremendous success,” said Jack Ferguson, Philadelphia Convention & Visitors Bureau President & CEO. “We were grateful for the opportunity to showcase our wonderful Pennsylvania Convention Center, the hotels and hospitality offerings of our city and our region's life sciences assets, and we hope BIO comes back again in the near future.”

    For BIO 2015 video, photos, and more, visit convention.bio.org.

    The 2016 BIO International Convention will take place June 6-9, 2016 in San Francisco, California at the Moscone Center. The 2017 BIO International Convention will take place June 19-22, 2017 in San Diego, California at the San Diego Convention Center. The 2018 BIO International Convention will take place June 4-7, 2018 in Boston, Massachusetts at the Boston Convention & Exhibition Center.

    “I would like to thank the Philadelphia Convention & Visitor's Bureau and the Pennsylvania Convention Center for their leadership and assistance in making the 2015 BIO International Convention such a resounding success,” said Scott Whitaker, President of the BIO International Convention. “The 2015 BIO International Convention may be wrapping-up, but I know the entire biotech community is excited to be back in San Francisco in 2016, where we expect another great BIO Convention.”  

    The BIO International Convention helps support BIO programs and initiatives. BIO works throughout the year to create a policy environment that enables the industry to continue to fulfill its vision of bettering the world through biotechnology innovation.

    Source: https://www.bio.org/media/press-release/2015-bio-international-convention-closes-philadelphia-event-sets-new-record-part

  • Top 10 European Biopharma Clusters

    Source: GEN

    Top 10 European Biopharma ClustersGenetic Engineering & Biotechnology News Logo

    Europe Is Home to Numerous Regional Communities.

    Find Out How They Rank on This GEN List

    Top 10 European Biopharma Clusters

    European countries compete vigorously not just in soccer but also in their efforts for attract and build up life science clusters. [© aberenyi/Fotolia]

    • The FDA’s approval March 6 of the first biosimilar drug for U.S. use helped shine a spotlight on European biotechnology, and not just because the biosimilar in question—Zarxio™, a variation of Amgen’s Neupogen (filgrastem)—will be marketed by the Sandoz unit of Swiss-based Novartis. As of March 10, Europe has 19 biosimilars authorized for patient use since 2006 (including Zarxio, which was approved as Zarzio in 2009).

      Yet there’s much more to biotechnology in Europe than biosimilars. The continent, like the U.S., is home to numerous regional communities or “clusters” of biotechs, pharmaceutical employers, universities, and independent research institutions. As GEN remarked last year, Europe’s biotech leaders enjoy more than one strong region, while smaller nations market their entire country as a hub for life sciences activities. Thus, unlike the U.S., European clusters cannot practically be ranked solely by region.

      And as in the U.S., there’s no central clearinghouse for statistical information using a single standard. So a list of European clusters is likely to include numerous larger bioclusters, though a few smaller clusters are notable enough to deserve mention: Norway’s Oslo Cancer Cluster, for example, is home to 66 biotech, pharma, and life sciences employers. That number is expected to increase once the cluster’s Innovation Park opens on August 24. The park will integrate the Oslo Cancer Cluster Incubator, Ullern High School, The Norwegian Cancer Registry, the Pathology unit, and bioinformatics unit at Oslo University Hospital and several biopharma businesses, from startups to bigger companies.

    • #10—Ireland

      What Ireland appears to lack in patents (14th with six) and EU research funding (13th with 86 grants), the country more than makes up for in venture capital (seventh at $103.232 million in 2013) and number of companies (seventh with 120). “Close to 50,000” are employed in biopharma (IDA Ireland). Yet when it comes to biotech in Ireland, perhaps the best known number is 12.5%—one of the lowest corporate tax rates in the Western world, and no small reason why the Emerald Isle has proven attractive to biopharmas from the U.S. and continental Europe. Under criticism from the U.S. and other nations, Ireland agreed to phase out by 2020 the “Double Irish” practice that has long allowed multinational companies to cut their tax bills legally. Yet Ireland continues to attract jobs from biopharmas based in and outside the country; last month Horizon Pharma opened its new Dublin HQ, with plans to add 60 jobs over three years, while Bristol-Myers Squibb in November said it would build a new biologics manufacturing facility in Cruiserath, County Dublin, designed to support up to 400 manufacturing jobs.

    • #9 – Sweden

      Sweden’s strengths include its number of pure biotechs (136 of some 1,500 companies) and EU research funding (211 grants). More than 30,000 employees work in the industry (SwedenBIO). But the country dips to ninth in 2014 patents (30) and places only 13th in venture capital ($16.230 million in 2013). That’s not to say Swedish companies aren’t finding money; Göteborg-based Cortendo last month completed a $26 million private placement, growing to $37 million the amount of financing raised by the developer of treatments for orphan endocrine disorders since October. And Stockholm-based NeuroVive raised its presence in Asia by establishing a subsidiary that collected $3.255 million from Taiwanese investors, ahead of a planned initial public offering in Taiwan. Sweden’s life-sci strengths remain its heritage of pharmas such as Astra (now AstraZeneca) and Pharmacia (acquired by Pfizer)—and a present-day research base that includes the Karolinska Institute as well as Lund, Uppsala, and Göteborg Universities. Karolinska and Lund joined a home-grown company, Newron Sweden, last month in reporting that a growth factor applied to the brains of a 12 patients resulted in increased signaling of dopamine—a discovery that researchers hope can someday aid in the development of a treatment for Parkinson's disease.

    • #8—Denmark

      Denmark places fifth in venture capital ($135.492 million in 2013), sixth in patents (67), and 10th in both EU research funding (151 grants) and number of core biotechs (88). The industry employs 40,000, according to the Ministry of Foreign Affairs. Yet the country’s drug developers have been generating no small share of attention; Kvistgaard-based Bavarian Nordic earlier this month licensed its Phase III Prostvac® prostate cancer vaccine to Bristol-Myers Squibb for up to $975 million. Prostvac is indicated for asymptomatic or minimally symptomatic metastatic castration-resistant prostate cancer (mCRPC).  And on March 7, Novo Nordisk presented positive Phase IIIa data at The Endocrine Society's 97th Annual Meeting for its Saxenda® (liraglutide [rDNA origin] injection) treatment, which won FDA approval in December. A rich history in agriculture spawned a research industry that has grown into a full-fledged biopharma cluster, anchored in part by the more than 100 companies within the Medicon Valley consisting of Greater Copenhagen and Sweden’s Scania region.

    • #7— Belgium

      Belgium is perhaps Europe’s most consistent biotech sector, placing seventh in EU research funding (228 grants), 2014 patents (45), and number of companies (more than 140), but faring worse in venture capital (ninth at $61.294 million). More than 30,000 people worked in the industry as of 2013 (Embassy and Consulates of Belgium in the U.S.). Biopharmas are concentrated in a few regions—from Leuven, Mechelen, and Walloon Brabant (South East of Brussels), to Ghent, where home-grown ActoGeniX agreed last month to be acquired by Intrexon for $60 million, in a deal that will combine the companies’ technologies to create new oral biologic drugs. Expansion works both ways with Belgian biotechs. Earlier this month, Cardio3 BioSciences based in Mont-Saint-Guibert, Walloon Brabant, selected as the site of its first U.S. facility Rochester, MN—home to the Mayo Clinic, which holds a 3% stake in the Cardio3 BioSciences, and licenses its intellectual property to the company.

    • #6—Italy

      Italy fared best in core biotechs (fourth with 264 of a total 422 biotechs) and EU research funding (fifth with 322 grants), but lagged somewhat on 2014 patents (eighth with 31) and lagged especially on venture capital (a mere 12th with $25.808 million in 2013). The industry had 6,626 R&D employees in 2013, trade group Assobiotec Federchimica and the Italian Trade Agency with Farmindustria reported last year. The country has built a new generation of biotechs by leveraging its heritage chemical and pharmaceutical businesses, especially near Milan. But least one U.S. biotech has taken notice of Italy’s research: Biogen Idec agreed in January to develop gene therapies for hemophilia A and B with the San Raffaele-Telethon Institute for Gene Therapy (TIGET), a joint venture of Italy’s Fondazione Telethon and Ospedale San Raffaele.

    • #5—Switzerland

      Switzerland’s combination of corporate R&D, university research, and a strong financial sector helps explain its numbers: The country finished third in 2014 patents (94) and venture capital ($172.591 million in 2013), but placed sixth in core biotechs (146) and ninth in EU research funding (165 grants). Swiss Biotech counted 14,131 employees in 2013. The numbers also reflect the presence of a new generation of biotechs emerging from the lengthy shadows of heritage pharma giants Novartis and Roche, both of which are still headquartered within the Swiss Confederation. For example, the €30 million ($33 million) biotech startup support program launched by Merck Serono when it closed its Geneva R&D hub has its first success, with Prexton Therapeutics last month closing on an €8.7 million ($9.4 million) Series A financing round toward developing new small molecules targeting the metabotropic Glutamate Receptor 4 (mGluR4) through Phase I trials. Sunstone Capital and Ysios Capital co-led the round, with MS Ventures participating.

    • #4—The Netherlands

      The Netherlands ranked highest in EU research funding (fourth with 328 grants), and was middle-of-the-list in both 2014 patents (76) and number of core biotechs (114), dipping only slightly in venture capital (sixth with $148.396 million in 2013). The country’s biotech sector employed nearly 35,000 in 2013 (Commission on Genetic Modification or COGEM), and remains among the more diverse in Europe, with tools giant Qiagen, contract research organization Quintiles, and Royal DSM, a drug developer that expanded into contract manufacturing and development (through its DPx joint venture with JLL Partners). Yet a Dutch biotech, Lead Pharma, garnered attention last month by launching a partnership with Sanofi to develop and commercialize small molecule therapies for a broad range of autoimmune disorders by targeting the nuclear receptors known as retinoic acid receptor-related orphan receptor gamma (RORγt).

    • #3—France

      France has made some impressive strides in biotech the past few years, climbing to second in both 2014 patents (124) and venture capital ($188.721 million in 2013), and to third in both EU research funding (438) and core biotechs (224 according to EY; 255 according to France Biotech). Industry employment last year stood at 110,006 (European Federation of Pharmaceutical Industries and Associations or EFPIA) Yet in December, France Biotech cited several obstacles to investment in the smallest startups—from “excessive paperwork and administrative tasks when dealing with multiples layers of academic institutions” to delays in review of applications for the CIR or Research Tax Credit, to the need for more understanding of industry needs by regulators. The most closely watched drug developer is heritage pharma Sanofi, whose new CEO Olivier Brandicourt, M.D., comes from France—unlike predecessor Christopher Viehbacher, whose move from Paris to Boston and pursuit of job cuts in the Republic contributed to his ouster by the board. Dr. Brandicourt will begin work on April 2. The Ile de France region that includes Paris remains the center of France’s life sciences activity, followed by Rhône-Alpes.

    • #2—United Kingdom

      The U.K.’s world-renowned universities and their biotech spinouts keep the kingdom first in EU research funding (516 grants) and in venture capital ($446.801 million in 2013), while finishing second in number of core biotechs (309 by EY, among 4,980 companies identified by the government in 2013) and fourth in 2014 patents (87). A total 175,761 people worked in the industry as of 2013 (UK government), though the figure includes 76,723 “medical technology” staffers in a category broad enough to include in vitro diagnostics and wound care. Like Germany, the U.K. benefits from two powerhouse clusters—the triangle formed by London, Cambridge, and Oxford and sometimes known as “East of England;” and Edinburgh, Scotland, institutions and companies focused on stem cell research (capitalizing on since-lifted restrictions in the U.S.) and industrial biotech. The U.K. also benefits from ambitious public-private efforts; in July 2014, the government joined Illumina and The Wellcome Trust in committing £311 million ($470 million) to map 100,000 human genomes by 2017–and thus catapult the kingdom to global leadership in genetic research into cancer and rare diseases.

    • #1—Germany

      Germany leads Europe in biopharma activity by 2014 patents (267) and number of “core” biotechs dedicated exclusively to the industry (428), while placing second to the U.K. in public funding, thanks to the presence of two dynamic regional clusters, North-Rhine Westphalia (NRW) and Bavaria. VC activity lags behind other Euro-giants (fourth at $169.365 million in 2013), in large part because of Germany’s unfavorable tax environment for investments. Industry employment was reported last year at 110,006 (EFPIA). About half of Germany’s companies are in the country’s two strongest clusters: North-Rhine Westphalia (more than 400, including 87 core biotechs and another 15 “additionally biotech active” companies, mostly global giants such as Bayer) and Bavaria (333 companies, including 160 to 170 core biotechs). 

  • FDA approves first biosimilar product Zarxio


    FDA approved Zarxio (filgrastim-sndz), the first biosimilar product approved in the United States.

    Biological products are generally derived from a living organism. They can come from many sources, including humans, animals, microorganisms or yeast. A biosimilar product is a biological product that is approved based on a showing that it is highly similar to an already-approved biological product, known as a reference product. The biosimilar also must show it has no clinically meaningful differences in terms of safety and effectiveness from the reference product. Only minor differences in clinically inactive components are allowable in biosimilar products.

    Sandoz, Inc.’s Zarxio is biosimilar to Amgen Inc.’s Neupogen (filgrastim), which was originally licensed in 1991. Zarxio is approved for the same indications as Neupogen.

     

    For more information, see below:

    The U.S. Food and Drug Administration today approved Zarxio (filgrastim-sndz), the first biosimilar product approved in the United States.

    Biological products are generally derived from a living organism. They can come from many sources, including humans, animals, microorganisms or yeast.

    A biosimilar product is a biological product that is approved based on a showing that it is highly similar to an already-approved biological product, known as a reference product. The biosimilar also must show it has no clinically meaningful differences in terms of safety and effectiveness from the reference product. Only minor differences in clinically inactive components are allowable in biosimilar products.

    Sandoz, Inc.’s Zarxio is biosimilar to Amgen Inc.’s Neupogen (filgrastim), which was originally licensed in 1991. Zarxio is approved for the same indications as Neupogen, and can be prescribed by a health care professional for:

    - patients with cancer receiving myelosuppressive chemotherapy;
    - patients with acute myeloid leukemia receiving induction or consolidation chemotherapy;
    - patients with cancer undergoing bone marrow transplantation;
    - patients undergoing autologous peripheral blood progenitor cell collection and therapy; and
    - patients with severe chronic neutropenia.

    “Biosimilars will provide access to important therapies for patients who need them,” said FDA Commissioner Margaret A. Hamburg, M.D. “Patients and the health care community can be confident that biosimilar products approved by the FDA meet the agency’s rigorous safety, efficacy and quality standards.”

    The Biologics Price Competition and Innovation Act of 2009 (BPCI Act) was passed as part of the Affordable Care Act that President Obama signed into law in March 2010. The BPCI Act created an abbreviated licensure pathway for biological products shown to be “biosimilar” to or “interchangeable” with an FDA-licensed biological product, called the “reference product.” This abbreviated licensure pathway under section 351(k) of the Public Health Service Act permits reliance on certain existing scientific knowledge about the safety and effectiveness of the reference product, and enables a biosimilar biological product to be licensed based on less than a full complement of product-specific preclinical and clinical data.

    A biosimilar product can only be approved by the FDA if it has the same mechanism(s) of action, route(s) of administration, dosage form(s) and strength(s) as the reference product, and only for the indication(s) and condition(s) of use that have been approved for the reference product. The facilities where biosimilars are manufactured must also meet the FDA’s standards.

    The FDA’s approval of Zarxio is based on review of evidence that included structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamics data, clinical immunogenicity data and other clinical safety and effectiveness data that demonstrates Zarxio is biosimilar to Neupogen. Zarxio has been approved as biosimilar, not as an interchangeable product. Under the BPCI Act, a biological product that that has been approved as an “interchangeable” may be substituted for the reference product without the intervention of the health care provider who prescribed the reference product.

    The most common expected side effects of Zarxio are aching in the bones or muscles and redness, swelling or itching at injection site. Serious side effects may include spleen rupture; serious allergic reactions that may cause rash, shortness of breath, wheezing and/or swelling around the mouth and eyes; fast pulse and sweating; and acute respiratory distress syndrome, a lung disease that can cause shortness of breath, difficulty breathing or increase the rate of breathing.

    For this approval, the FDA has designated a placeholder nonproprietary name for this product as “filgrastim-sndz.” The provision of a placeholder nonproprietary name for this product should not be viewed as reflective of the agency’s decision on a comprehensive naming policy for biosimilar and other biological products. While the FDA has not yet issued draft guidance on how current and future biological products marketed in the United States should be named, the agency intends to do so in the near future.

    Sandoz, a Novartis company, is based in Princeton, New Jersey. Neupogen is marketed by Amgen, based in Thousand Oaks, California.

    The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

     

    Source: FDA News Release

     

  • FDA Clears Sale of First Direct-to-Consumer Genetic Test

     
    The US Food and Drug Administration (FDA) announced today that they have authorized marketing of a direct-to-consumer genetic test from 23andMe.The test is designed to identify healthy individuals who carry a gene that could cause Bloom Syndrome in their offspring.
    The agency also noted that it will now classify carrier screening tests, such as this one, as class II products, which means they are subject to general and special controls. Special controls can include performance standards, postmarket surveillance, patient registries, special labelling requirements and guidelines.
    The agency, however, specifically noted that they will not require premarket reviews for direct-to-consumer carrier screening tests. The agency plans to announce their intention of exempting these products and will have a 30-day public comment period regarding that decision.
    "The FDA believes that in many circumstances it is not necessary for consumers to go through a licensed practitioner to have direct access to their personal genetic information," said Alberto Gutierrez, PhD, director of the Office of In Vitro Diagnostics and Radiological Health in the FDA's Center for Devices and Radiological Health, in the agency press release.
    "Today’s authorization and accompanying classification, along with FDA’s intent to exempt these devices from FDA premarket review, supports innovation and will ultimately benefit consumers.… These tests have the potential to provide people with information about possible mutations in their genes that could be passed on to their children," he said.
    Bloom Syndrome is an autosomal recessive disorder, which means that carriers may be unaware that they carry a mutation in the BLM gene, but their child could be affected. The syndrome is characterized by short stature, usually under five feet, sun sensitivity, and an increased risk for cancer.
    The syndrome is rare among the general population but is more common in the Central and Eastern European (Ashkenazi) Jewish population. In that group, approximately one in 50,000 people are affected.
     
    Labeling Requirements
     
    The agency notes that while it is not regulating who can purchase or use the tests, the company is required to provide adequate information in the label such that users will understand what the test mean, and, if sold over the counter, how they can reach a board-certified clinical molecular geneticist or equivalent for pre- and post-test counseling.
    The approval is a significant shift in direction for the agency. In November 2013, the FDA sent 23andMe a warning letter directing the company to stop selling a Personal Genome Service. The letter said the company had not obtained proper approval or demonstrated that their tests were accurate and clinically meaningful.
    In the press release today, the agency notes that two studies, involving 228 samples in a total of four laboratories, have been done to show that the Bloom Syndrome test is accurate. The company also performed usability testing to ensure that consumers could collect an adequate saliva sample.