Archive for the ‘Pharmaceutical Discovery’ Category

More Than 25 Percent of the Novel New Drugs Approved by FDA in 2015 are Personalized Medicines

Reporter: Aviva Lev-Ari, PhD, RN

A new analysis from the Personalized Medicine Coalition (PMC) documents an upward trend in the number of personalized medicine approvals at FDA, with personalized medicines accounting for more than 1 in 4 novel new drugs (NNDs) approved in 2015.

The analysis, titled 2015 Progress Report: Personalized Medicine at FDA, lists the 13 personalized medicines approved as NNDs in 2015, which represent 28 percent of the 45 NNDs the agency approved overall. The new approvals accelerate a trend PMC first noted in 2014, when the Coalition classified 21 percent of the year’s NNDs as personalized medicines.

PMC Science Policy Vice President Daryl Pritchard, Ph.D., said the momentum is driven by scientific validation of personalized medicine’s ability to improve patient outcomes.

“The scientific community has established personalized medicine as a successful approach to treating many diseases,” Pritchard said. “The increasing number of approvals for these drugs reflects that progress.”



2015 Progress Report Personalized Medicine at FDA


More Than 25 Percent of the Novel New Drugs Approved by FDA in 2015 are Personalized Medicines

The transformation of health care from one-size-fits-all, trial-and-error medicine to a targeted approach utilizing an individual patient’s molecular information continues to accelerate as the U.S. Food and Drug Administration (FDA) more regularly and rapidly approves new personalized medicines. FDA’s Center for Drug Evaluation and Research (CDER) approved 45 novel new drugs (NNDs), either

new molecular entities or new therapeutic biologics, in 2015. Of these 45 NNDs, 13 of them — more than 25 percent — were personalized medicines as classified by the Personalized Medicine Coalition (PMC), thus continuing a trend that began last year when nine of 41 NNDs were classified as personalized medicines.





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Applying Pharmacology to New Drug Discovery, April 22, 2016 in San Diego, CA by CHI

Reporter: Aviva Lev-Ari, PhD, RN


Applying Pharmacology to New Drug Discovery, April 22, 2016 in San Diego, CA by CHI

The system-independent quantification of molecular drug properties for prediction of therapeutic utility

April 22, 2016

Over the past 6 six years, the primary cause of new drug candidate failures (50%) has been failure of therapeutic efficacy. Put another way, drug discovery programs do everything right, get the defined candidate molecule, only to have it fail in therapeutic trials. Among the most prevalent reasons proposed for this shortcoming is the lack of translation of in vitro and recombinant drug activity to therapeutic in vivo whole systems. Drug activity in complete systems can be characterized with the application of pharmacological principles which translate drug behaviors in various organs with molecular scales of affinity and efficacy.

Pharmacological techniques are unique in that they can convert descriptive data (what we see, potency, activity in a given system) to predictive data (molecular scales of activity that can be used to predict activity in all systems including the therapeutic one, i.e. affinity, efficacy). The predicted outcome of this process is a far lower failure rate as molecules are progressed toward clinical testing.


Terry Kenakin presently is a Professor of Pharmacology in the Department of Pharmacology, University of North Carolina School of Medicine. The course is taught from the perspective of industrial drug discovery; Dr. Kenakin has worked in drug industry for 32 years (7 at Burroughs-Wellcome, RTP, NC and 25 at GlaxoSmithKline, RTP. NC). He is Editor-in-Chief of the Journal of Receptors and Signal Transduction and Co-Editor-in-Chief of Current Opinion in Pharmacology and is on numerous journal Editorial Boards. In addition, he has authored over 200 peer reviewed papers and reviews and has written 10 books on Pharmacology.

Course Material

Summary sheets, exercises with answers, relevant papers are included as well as a pdf of all slides. The course is based on the book A Pharmacology Primer: Techniques for More Effective and Strategic Drug Discovery. 4th Edition, Elsevier/Academic Press, 2014.

This course will describe pharmacological principles and procedures to quantify affinity, efficacy, biased signaling and allostery to better screen for new drugs and characterize drug candidates in lead optimization assays.

1. Assay Formats/Experimental Design

  • Binding
  • Functional Assays
  • Null Method Assays

2. Agonism

  • Agonist Affinity/Efficacy
  • Black/Leff Operational model

3. Biased Signaling (Agonism)

  • Mechanism of Biased Signaling
  • Quantifying Biased Agonism
  • Therapeutic application(s)

4. Orthosteric Antagonism (I)

  • Competitive
  • Non-Competitive/Irreversible

5. Orthosteric Antagonism (II)

  • Partial Agonism
  • Inverse Agonism

6. Allosteric Modulation (I)

  • Functional Allosteric Model
  • Negative Allosteric Modulators (NAMs)

7. Allosteric Modulation (II)

  • Positive Allosteric Modulators (PAMs)
  • Allosteric Agonism

8. Drug-Receptor Kinetics

  • Measuring Target Coverage
  • Allosteric Proof-of-Concept
  • Application of Real-Time Kinetics

9. Drug Screening

  • Design of Screening Assays
  • Screening for Allosteric Modulators

Cambridge Healthtech Institute’s Eleventh Annual Drug Discovery Chemistry is a dynamic conference for medicinal chemists working in pharma and biotech. Focused on discovery and optimization challenges of small molecule drug candidates, this event provides many exciting opportunities for scientists to create a unique program by going back and forth between concurrent meeting tracks to hear presentations most suited to one’s personal interests. New for 2016 is the addition of three symposia on Friday covering the blood-brain barrier, biophysical approaches for drug discovery, and antivirals.

Plenary Keynotes


A New Model for Academic Translational Research

Peter G. Schultz, Ph.D., The Scripps Research Institute

Cell-Penetrating Miniproteins

Gregory L. Verdine, Ph.D., Harvard University

April 19-20

April 20-21

April 22

Inflammation Inhibitors

Kinase Inhibitor Chemistry

Brain Penetrant Inhibitors

Protein-Protein Interactions

Macrocyclics & Constrained Peptides

Biophysical Approaches

Epigenetic Inhibitor Discovery

Fragment-Based Drug Discovery


Short Courses

Make the most of your time in San Diego by adding on one or more short courses*. Topics include trends in physical properties, GPCRs, peptide therapeutics, immunology, phenotypic screening, crystallography, ligand-receptor molecular interactions, inhibitor design, macrocycles, FBDD, and covalent inhibitors.

* separate registration required for short courses


From: Deborah Shear <>

Date: Friday, January 8, 2016 at 11:42 AM

To: Aviva Lev-Ari <>

Subject: Training Seminar: Applying Pharmacology to New Drug Discovery

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J.P. Morgan 34th Annual Healthcare Conference & Biotech Showcase™ January 11 – 15, 2016 in San Francisco

Reporter: Aviva Lev-Ari, PhD, RN

J.P. Morgan 34th Annual Healthcare Conference


January 11, 2016 – January 14, 2016 (all-day)


San Francisco, CA, USA

Conference AGENDA 


UPDATED on 1/10/2016

The definitive guide to the J.P. Morgan Healthcare Conference


[Image courtesy of Flickr user Ryan McDonald]


MAJOR BioTech Conferences in San Francisco –

The CAPITAL of BioTech for 2nd week in January 2016


  • Festival Start up Health

  • Biotech Showcase 2016

  • JP Morgan HealthCare Conference



  • The 9th Annual OneMedForum, San Francisco 2016 – A Private Investment Conference for OneMed Research Clients


  • RESI@JPM — Redefining Early Stage Investments – Life Science Nation (LSN)


Venture Valkyrie (and capitalist) Lisa Suennen rightly pointed out that JPM is typically a male-dominated affair, which is why she’s written “JP Morgan: Where the Boys are… And not the Girls.”

In a field where women hold many senior positions in actual US healthcare corporations, they are drowned out at this conference by the advancing horde of finance guys in red ties and the CEOs that love them.

Signal Podcast on STAT

Listen to Episode 5: By LUKE TIMMERMANandMEG TIRRELL

San Francisco 2016

Union Square: Maps & Resources by MacDougall Biomedical Communications.

Biotech Showcase™ 2016 Program Overview

Sunday, January 10, 2016
1:00–5:00 pm
Additional Program
Biotech Showcase™ pre-event

This workshop is focused on delivering results and securing funding at All Levels: Boards, Angels, VCs, Corporate Partners and Other Sources of Funds, with four hours of intensive and interactive discussion, on-your-feet sessions, war stories and insights aimed at folks looking for financing. It is designed to accelerate your funding activities and eliminate unnecessary noise.

Preregistration is required, more information can be found here.

3:00–6:00 pm
Level 4, Cyril Magnin Foyer

All Biotech Showcase attendees are invited to pick up name badges prior to the beginning of the conference on Monday.

Monday, January 11, 2016
7:00 am
Level 4, Cyril Magnin Foyer
Registration Opens and Continental Breakfast
8:00–8:55 am
8:00 am–6:00 pm
One-to-one Meetings ►

Hilton Union Square
333 O’Farrell Street
Level 2, Ballroom

8:00–9:50 am

Regenerative Medicine and Advanced Therapies State of the Industry Briefing

8:00 am–12:00 pm

Company Presentations ►

Private Biotech
Public Biotech

12:00–1:30 pm

Lunch Plenary

1:45–5:30 pm

Company Presentations ►

Private Biotech
Public Biotech

Tuesday, January 12, 2016
7:00 am
Level 4, Cyril Magnin Foyer
Registration Opens and Continental Breakfast
8:00–8:55 am
8:00 am–6:00 pm
One-to-one Meetings ►

Hilton Union Square
333 O’Farrell Street
Level 2, Ballroom

8:00–9:15 am

Medtech Showcase State of the Industry Report

8:00 am–12:00 pm

Company Presentations ►

Private Biotech
Public Biotech

12:00–1:30 pm

Lunch Plenary

1:45–5:30 pm

Company Presentations ►

Private Biotech
Public Biotech

4:30–5:30 pm

Medtech Showcase Workshop


Wednesday, January 13, 2016
7:00 am
Level 4, Cyril Magnin Foyer
Registration Opens and Continental Breakfast
8:00–8:55 am

8:00 am–5:00 pm
One-to-one Meetings ►

Hilton Union Square
333 O’Farrell Street
Level 2, Ballroom

8:00–9:00 am

Digital Health Showcase State of the Industry Report

8:00 am–12:00 pm

Company Presentations ►

Private Biotech
Public Biotech

10:00–11:00 am

Digital Health Showcase Workshop

11:00–11:45 am

11:45 am–12:15 pm

Digital Health Showcase Discussion

12:00–1:30 pm

Lunch Plenary

1:00–1:45 pm

Digital Health Showcase Workshop

4:00–5:00 pm

Digital Health Showcase Workshop

1:45–5:00 pm

Company Presentations ►

Private Biotech
Public Biotech

5:00–6:00 pm

Level 4, Cyril Magnin Foyer
Closing Reception



About Biotech Showcase™ 2016

Previous conferences ►

Biotech Showcase™ 2016
January 11–13, 2016, San Francisco

Biotech Showcase™ 2015 Highlights

  • 232 company presentations
  • 2,100 attendees
  • 1,276 companies
  • 37 countries represented
  • 4,277 one-to-one meetings
  • 14 workshops and panels

Photos of Biotech Showcase 2015 ►

Biotech Showcase™ is an investor and networking conference devoted to providing private and public biotechnology and life sciences companies with an opportunity to present to, and meet with, investors and potential strategics in one place during the course of one of the industry’s largest annual healthcare investor conferences. Investors and biopharmaceutical executives from around the world gather in San Francisco during this critical week which is widely viewed as setting the tone for the coming year.

Now in its eighth year, this rapidly growing conference features multiple tracks of presenting companies, plenary sessions, workshops, networking, and an opportunity to schedule one-to-one meetings.

Biotech Showcase delegates include investors in private and public companies, sector analysts, bankers and industry professionals, as well as biopharmaceutical and life science company executives.

Biotech Showcase is produced by Demy Colton Life Science Advisors and EBD Group. Both organizations have a long history of producing high quality programs that support the biotechnology and broader life sciences industry.

Biotech Showcase™ 2016 Press Releases

J.P. Morgan 34th Annual Healthcare Conference


January 11, 2016 – January 14, 2016 (all-day)


San Francisco, CA, USA

Conference AGENDA 




Whether you’re a conference veteran or a rookie, we hope this light-hearted guide helps you survive the week of life science mayhem in San Fransisco. At Chempetitive Group, we have a deep passion for everything life science—its people, its processes and its promise for the future. As life science marketers, this passion takes us to the industry’s biggest events every year, including the J.P. Morgan Healthcare Conference and related conferences each January. Over the years, we’ve learned our way around San Francisco’s Union Square—places we like to frequent.


January 11-14 San Francisco RAMP UP

Over 12,000 attendees

Over 15,000 meetings

Over 1,500 companies presenting

Over 40 countries represented Projected value of this year’s deals: unlimited

Surviving the J.P. Morgan Healthcare Conference [Plus Insider’s Guide]


Each January, the J.P. Morgan Healthcare Conference – perhaps the life science industry’s largest and most frenzied conference of the year – reliably draws thousands of investors and executives across the healthcare sector to San Francisco’s Union Square neighborhood as hundreds of companies present their latest innovations and dreams in an attempt to pique the interest of venture capitalists and potential partners. In addition to J.P. Morgan, parallel events Biotech Showcase, OneMedForum and RESI Conference ensure that there is a high density of biotech brainpower and capital in the City by the Bay.

The conference week is a mix of long days of presentations and lively evenings of cocktail parties and networking events. With more than 50 networking receptions, days of sessions, and still a volume of work to manage while away from the office, you might need some guidance on where to take your client or potential partner for a meeting, where to refuel or caffeinate, or simply where to hide from the chaos. For these reasons, we decided to let you into our world by creating this simple guide to surviving the 2016 J.P. Morgan Healthcare Conference week.

Download it and, if you happen to find yourself in one of our favorite spots, let us know with a direct message on Twitter at @chempetitive. Safe travels, have fun, and get some deals done.

JP Morgan 2016 Healthcare Conference Participants

The following organizations have released announcements of their participation in the 34th Annual JP Morgan Healthcare Conference:



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Two New Drugs for Inflammatory Bowel Syndrome Are Giving Patients Hope

Reporter: Stephen J. Williams, Ph.D.

Actavis Receives FDA Approval for VIBERZI (eluxadoline) for the Treatment of Irritable Bowel Syndrome with Diarrhea (IBS-D) in Adults -First in class treatment for IBS-D treats hallmark symptoms of IBS-D; abdominal pain and diarrhea

DUBLIN, May 27, 2015 /PRNewswire/ — Actavis plc (NYSE: ACT) announced today that VIBERZI™ (eluxadoline) was approved by the Food and Drug Administration (FDA) as a twice-daily, oral treatment for adults suffering from irritable bowel syndrome with diarrhea (IBS-D). VIBERZI (eluxadoline) has mixed opioid receptor activity, it is a mu receptor agonist, a delta receptor antagonist, and a kappa receptor agonist.

Logo –

“The FDA’s approval of VIBERZI is the first step to providing physicians with a new, evidence-based, treatment option for their adult patients with IBS-D,” said David Nicholson, Executive Vice President, Actavis Global Brands R&D. “At Actavis, we are dedicated to providing new treatment options, and the development of new agents that help address the most bothersome symptoms of IBS-D. We are very pleased to be working with the FDA to advance this IBS-D treatment and we eagerly await DEA scheduling determination later this year.”

IBS-D is a multifactorial disorder marked by recurrent abdominal pain or discomfort and altered bowel function that affects as many as 15 million adult Americans, impacting about twice as many women as men.i,ii,iii There are few treatment options available for IBS-D, particularly options that relieve both the diarrhea and abdominal pain associated with IBS-D.

“The unpredictable symptoms experienced by patients with IBS-D can have a significant impact on everyday life,” said William D. Chey, MD, Nostrant Professor of Gastroenterology at the University of Michigan Health System. “It’s exciting when physicians are able to add an additional treatment option like VIBERZI to their toolbox for patients with IBS-D.”

The FDA has recommended that VIBERZI be classified as a controlled substance. This recommendation has been submitted to the U.S. Drug Enforcement Administration (DEA).  Once VIBERZI receives final scheduling designation, the updated label will be available. Pending final scheduling designation, product launch is anticipated in Q1 2016.


VIBERZI is an orally active compound indicated for the treatment of irritable bowel syndrome with diarrhea (IBS-D) in men and women. VIBERZI (eluxadoline) has mixed opioid receptor activity, it is a mu receptor agonist, a delta receptor antagonist, and a kappa receptor agonist.

Efficacy was established in two Phase III clinical studies, demonstrating significant superiority over placebo on the composite endpoint of simultaneous improvement in both abdominal pain and diarrhea at both 75 mg and 100 mg twice daily doses. The primary efficacy responder endpoint was evaluated over the duration of double-blind, placebo-controlled treatment. Response rates were compared based on patients who met the daily composite response criteria (improvement in both abdominal pain and stool consistency on the same day) for at least 50% of the days from weeks 1 to 12 (FDA endpoint) and weeks 1 to 26 (European Medicines Agency endpoint).

The most common adverse events in the two Phase III clinical trials were constipation (7% and 8% for eluxadoline 75 mg and 100 mg; 2% for placebo) and nausea (8% and 7% for eluxadoline 75 mg and 100 mg; 5% for placebo). Rates of severe constipation were less than 1% in patients receiving 75 mg and 100 mg eluxadoline. Rates of discontinuation due to constipation were low for both eluxadoline and placebo (≤2%) and similar rates of constipation occurred between the active and placebo arms beyond 3 months of treatment. A total of 2,426 subjects were enrolled across the two studies.

For more information including full prescribing information about VIBERZI at

About IBS-D

Irritable bowel syndrome with diarrhea (IBS-D) is a functional bowel disorder characterized by chronic abdominal pain and frequent diarrhea, which affects approximately 15 million patients in the U.S.  Although the exact cause of IBS-D is not known, symptoms are thought to result from a disturbance in the way the gastrointestinal tract and nervous system interact.

IBS-D can be debilitating and there are limited therapeutic options for managing the chronic symptoms. IBS-D is associated with economic burden in direct medical costs and indirect social costs such as absenteeism and lost productivity, along with decreased quality of life.

About Actavis
Actavis plc (NYSE: ACT), headquartered in Dublin, Ireland, is a unique, global pharmaceutical company and a leader in a new industry model—Growth Pharma. Actavis is focused on developing, manufacturing and commercializing innovative branded pharmaceuticals, high-quality generic and over-the-counter medicines and biologic products for patients around the world.

Actavis markets a portfolio of best-in-class products that provide valuable treatments for the central nervous system, eye care, medical aesthetics, gastroenterology, women’s health, urology, cardiovascular and anti-infective therapeutic categories, and operates the world’s third-largest global generics business, providing patients around the globe with increased access to affordable, high-quality medicines. Actavis is an industry leader in research and development, with one of the broadest development pipelines in the pharmaceutical industry and a leading position in the submission of generic product applications globally.

With commercial operations in approximately 100 countries, Actavis is committed to working with physicians, healthcare providers and patients to deliver innovative and meaningful treatments that help people around the world live longer, healthier lives.

Actavis intends to adopt a new global name – Allergan – pending shareholder approval in 2015.

For more information, visit Actavis’ website at

Actavis Cautionary Statement Regarding Forward-Looking Statements

Statements contained in this communication that refer to Actavis’ estimated or anticipated future results, including estimated synergies, or other non-historical facts are forward-looking statements that reflect Actavis’ current perspective of existing trends and information as of the date of this communication. Actual results may differ materially from Actavis’ current expectations depending upon a number of factors affecting Actavis’ business. These factors include, among others, the timing and success of product launches; the difficulty of predicting the timing or outcome of product development efforts and regulatory agency approvals or actions, if any; market acceptance of and continued demand for Actavis’ products; difficulties or delays in manufacturing; and such other risks and uncertainties detailed in Actavis’ periodic public filings with the Securities and Exchange Commission, including but not limited to Actavis plc’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2015 and from time to time in Actavis’ other investor communications. Except as expressly required by law, Actavis disclaims any intent or obligation to update or revise these forward-looking statements.

i Camilleri M. Current and future pharmacological treatments for diarrhea-predominant irritable bowel syndrome. Expert Opinion on Pharmacotherapy. 2013;14:1151.

ii Grundmann O, Yoon SL. Irritable bowel syndrome: epidemiology, diagnosis, and treatment: an update for health-care practitioners. Journal of Gastroenterology and Hepatology. 2010;25:691–699.

iii Eluxadoline Xifaxin Summary Final. November 2014.

Lisa DeFrancesco
(862) 261-7152

David Belian
(862) 261-8141

SOURCE Actavis plc


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Synergy’s Looming FDA Filing Makes It Pharma of the Month

By James Passeri Follow

| Jan 05, 2016 | 8:39 AM EST  | 0

Keep an eye on Synergy Pharmaceuticals (SGYP) this month: Analysts like it, its shares have waned since a big spike this summer, and the official filing of its star product is expected any day.

When the New York-based pharmaceutical company, which specializes in gastrointestinal therapy, announced that it passed clinical trials on its flagship drug plecanatide this summer, shares rocketed 95%.

But today analysts appear mystified at why the stock has receded 45% from its July high, especially with plecanatide’s new drug application with the Food and Drug Administration expected this month. (It’s currently trading below $6, and the consensus price target is over $13, according to data provided by Bloomberg.)

Synergy should be raking in $600 million from plecanatide, a daily tablet that treats patients with irritable bowel syndrome (IBS), within five years of obtaining FDA approval (expected in 2017, according to equity research firm BTIG. Synergy currently has a market capitalization of just $645 million.

BTIG’s $11 price target is also buoyed by roughly $142 million on the balance sheet, as well as newly appointed management including CFO Gary Sender and COO Troy Hamilton, both former executives at pharma success story Shire (SHPG). Though Shire shares are down just under 4% over the past 12 month, they have rocketed 112% over the past two years.

Synergy also stands to benefit from a growing demand for gastrointestinal treatments, feeding the appetite of Big Pharma for potential acquisitions, according to BTIG.

“With about 45 million Americans suffering from chronic constipation and IBS, and major companies like Allergan(AGN) and Valeant (VRX) focusing their marketing efforts on GI treatments, it seems logical to imagine SGYP as a takeover candidate,” BTIG analyst Timothy Chiang wrote in a November report.

Whether or not this leads to a buyout or another stock surge, Synergy certainly can be counted on for a healthy dose of small-cap volatility as its chief product takes the final steps toward reaching its customers.



Synergy Pharmaceuticals Announces Successful End-of-Phase 2 Meeting with FDA for Plecanatide in Irritable Bowel Syndrome with Constipation

Download PDF

Pivotal Phase 3 IBS-C Program to be Initiated in the Fourth Quarter of 2014

NEW YORK– Synergy Pharmaceuticals Inc. (NASDAQ:SGYP) today announced that it has successfully completed an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) on its lead drug plecanatide for the treatment of irritable bowel syndrome with constipation (IBS-C). Agreement was reached with the FDA for the plecanatide pivotal phase 3 IBS-C clinical development program that is scheduled to begin in the fourth quarter of this year.

“We are very pleased with the outcome of our meeting with the FDA and have a clear path forward to start the IBS-C registration program with plecanatide this year,” said Dr. Gary S. Jacob, Chairman and CEO of Synergy. “The pivotal phase 3 IBS-C trials will include both 3.0 mg and 6.0 mg plecanatide, which are consistent with the doses currently being evaluated in our phase 3 chronic idiopathic constipation (CIC) program. Plecanatide has demonstrated a clinical dose-response for efficacy with an excellent tolerability profile that is observed across trials. This is an important advantage as we look to bring two doses to market in both indications and provide physicians with options for addressing individual patient needs.”

Synergy’s pivotal phase 3 IBS-C clinical development program will consist of two registration trials, each including 1,050 patients who will receive either placebo, 3.0 mg or 6.0 mg plecanatide. IBS-C patients successfully completing either of the 12-week placebo-controlled registration trials will be offered enrollment into a long-term safety trial in order to complement and support the ongoing long-term safety database for the CIC indication.

About Plecanatide

Plecanatide is Synergy’s lead uroguanylin analog in late-stage clinical development to treat patients with CIC and IBS-C. Uroguanylin is a natural gastrointestinal (GI) hormone produced by humans in the small intestine and plays a key role in regulating the normal functioning of the digestive tract through its activity on the guanylate cyclase-C (GC-C) receptor. The GC-C receptor is known to be a primary source for stimulating a variety of beneficial physiological responses. Orally administered plecanatide mimics uroguanylin’s functions by binding to and activating the GC-C receptor to stimulate fluid and ion transit required for normal bowel function. Synergy has successfully completed a phase 2b trial of plecanatide in 951 patients with CIC and is currently enrolling patients in two pivotal phase 3 CIC trials. The company also recently announced positive top-line data results from a phase 2b dose-ranging study with plecanatide in patients with IBS-C.

About Synergy Pharmaceuticals

Synergy Pharmaceuticals (NASDAQ:SGYP) is a biopharmaceutical company focused on the development of novel therapies based on the natural human hormone, uroguanylin, to treat GI diseases and disorders. Synergy has created two unique analogs of uroguanylin – plecanatide and SP-333 – designed to mimic the natural hormone’s activity on the GC-C receptor and target a variety of GI conditions. SP-333 is currently in phase 2 development for opioid-induced constipation and is also being explored for ulcerative colitis. For more information, please visit


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Genomics’ Proprietary Statistical Analysis Tools and Integrated Multi-Phenotype Database to be used to Support Research and Development at Vertex Pharmaceuticals

Reporter: Aviva Lev-Ari, PhD, RN



Press release

04 January 2016

Genomics and Vertex Collaborate to Identify Target Therapeutic Pathways


Genomics’ Proprietary Statistical Analysis Tools and Integrated Multi-Phenotype Database to be used to Support Research and Development at Vertex Pharmaceuticals


Oxford, UK, 04 January 2016: Genomics plc (“Genomics”), a leading analysis company developing algorithms, data resources, and software solutions to uncover the relationships between genetic variation and human disease, today announced that Vertex Pharmaceuticals will use Genomics’ integrated database and state-of-the-art analysis tools to inform its drug research and development.  These tools aim to provide confidence in the rationale for targeting Vertex’s pathways of interest for the treatment of certain diseases and to identify potential safety concerns and repositioning opportunities.


Genomics has developed a unique analytical platform for genome analysis and interpretation. The platform combines proprietary algorithms and software with the Company’s integrated genome-phenome database and analytical expertise to learn about human biology.  Genomics has several existing partnerships with large pharmaceutical companies, and in clinical genomics is a Platform Partner for Genomics England, the company undertaking the 100,000 Genomes Project in the UK.


John Colenutt, CEO, Genomics plc, said: “Pharmaceutical and biotech companies are increasingly using human genetic data in research to increase the chance of success in drug development.  We are excited that Vertex has chosen to use Genomics’ proprietary technology, integrated database and tools to support them in this aim.”


Paul de Bakker, Ph.D., Head of Computational Genomics for Vertex said: “Vertex is focused on advancing research programs where disease mechanisms are validated by human biology.  Our collaboration with Genomics is aimed at obtaining insights into the genetic underpinnings of specific targets and diseases to help predict which potential medicines may have success moving from discovery research toward patients.”




Photo: John Colenutt, CEO, Genomics plc. For a high resolution image please contact


For further information please contact:


Zyme Communications

Lorna Cuddon

Tel: +44 (0)7811996942



About Genomics plc

Genomics was founded by four leading Oxford academics, including Professor Peter Donnelly, Director of The Wellcome Trust Centre for Human Genetics, and Professor Gil McVean, Director of The Big Data Institute. The Company has developed a unique platform for genomic sequence data analysis and interpretation which combines world-leading expertise in statistical analysis and data mining with a unique integrated database linking genotypes and phenotypes. Genomics England, the company running the UK project to undertake whole genome sequencing of 100,000 patients in the National Health Service, has appointed Genomics plc as a Platform Partner and has also awarded the Company three SBRI grants. Genomics plc is also working with four major pharmaceutical companies to bring the benefits of genomic analysis to their drug development processes. The Company is supported by major investors, including IP Group, Invesco Perpetual, Woodford Investment Management and Lansdowne Partners.


From: Lorna Cuddon <>

Reply-To: <>

Date: Monday, January 4, 2016 at 4:11 AM

To: Aviva Lev-Ari <>

Subject: Genomics and Vertex Collaborate to Identify Target Therapeutic Pathways

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Top Seven big Pharma in Thomson Reuters 2015 Top 100 Global Innovators

Reporter: Aviva Lev-Ari, PhD, RN


Abbott USA






Brinstol-Myers Squibb USA





Novartis Switzerland


Roche Switzerland



2012, 2011


Introducing the Thomson Reuters 2015 Top 100 Global Innovators Organization Country Industry Previous Winners

New in 2015:

Top Bay Area Innovators For the first time, Thomson Reuters analysts studied Silicon Valley, known as the technology and innovation corridor in the US, to see which companies are leading there. Following a methodology similar to that of the Top 100 Global Innovators, except for the Volume criteria, all companies headquartered or with a major subsidiary in that region were investigated. The Top Bay Area Innovators list can be found on page 19. There are 11 companies that overlap with the Top 100 Global Innovators; meaning 31 percent of the leading US innovators and 11 percent of the world’s top innovators are located in the Bay Area.


The United Kingdom is absent from the list yet again this year. Innovation incentives introduced in the UK, such as Patent Box legislation, do not have enough legacy yet to have had an impact. Additionally, the UK spends much less on R&D as a percentage of Gross Domestic Product (GERD) than the Top 100 Global Innovator countries do. The UK’s GERDis 1.63 percent, whereas, for example, Japan’s is 3.47 percent.5 The region’s underuse of its patent system and lack of significant commercialization keep the UK from making the list once again.

China is also absent from the 2015 list. It joined the innovation-leader ranks in 2014, for the first time, via Huawei, however wasn’t able to replicate that performance to join again in 2015. A big factor contributing to China’s shortcoming is the fact that most of its innovation is domestic and therefore is not realized outside of its borders. In fact, only about six percent of China’s innovation activity is protected, and commercialized, outside of China. In order for China to see more organizations join this prestigious group, it will need to think more internationally and look to bring its inventions to market around the world. There are 27 companies that dropped from the prior year (see Table 1 on page 12), including AT&T, IBM, Siemens and Xerox. While these companies are still innovating at noteworthy levels, their respective scores across all of the metrics did not advance them to the Top 100. It’s expected that we will see them again in the future.

Patent Reform

There’s been some influential intellectual property legislation that is shaping how companies innovate, where they seek protection and when. Some of these initiatives include the America Invents Act and the Patent Trial & Appeal Board; the European unitary patent and unified patent court; the UK’s Patent Box legislation; and impactful court rulings, such as Alice 101 in the US. The landscape is ripe with reform as patent offices and filers grapple with how best to implement these changes given their goals and needs. Despite these changes, one thing remains certain: the patent system is vital to protecting innovation and to the economic wellbeing of organizations, nations and our world. OECD statistics confirm that nations with higher GDPs have similarly high patent filing rates (aka strong patent infrastructures), whereas the converse holds equally true. One way for developing nations to propel their economies forward is to invest in innovation and building a reliable intellectual property infrastructure.


The Thomson Reuters Top 100 Global Innovator methodology analyzes patent and citation data across four main criteria:

  • volume,
  • success,
  • globalization and
  • influence

using Thomson Reuters solutions including Derwent World Patents Index (DWPI), Thomson Innovation and Derwent Patent Citations Index (PCI).


Volume is the first criteria. An organization must have at least 100 unique inventions protected by a granted patent over the most recent five year period to advance for further analysis. A unique invention is defined as one instance of a published application or granted patent for an idea for which protection is sought. In DWPI, these are called “basic” patents. DWPI provides access to 50 patentissuing authorities. Subsequent filings for the same invention are recorded as equivalents and collated into patent families which, for this analysis, were not included. Once an organization passes the volume stage gate, it is measured across the next three criteria: success, globalization and influence.


The success metric covers the ratio of inventions described in published applications (those patents which are filed and publicly published by the patent office but not yet granted) to inventions protected with granted patents over the most recent five years. Not all patent applications pass through the examination process and are granted.


Globalization has to do with the value an organization places on an invention by protecting it across the major world markets. The premise being that inventions protected in all four of the Thomson Reuters Quadrilateral Patent Index authorities: the Chinese Patent Office, the European Patent Office, the Japanese Patent Office and the United States Patent & Trademark Office, are deemed to be of significant value to the organization. A ratio is created of the inventions protected across the Quadrilateral Patent Index authorities versus the total volume for that period. Influence Finally,


influence is the downstream impact of an invention, measured by how often it is cited by other organizations. Via the Derwent Patent Citation Index, citations to an organization’s patents are counted over the most recent five years, excluding self citations. Scores for each of these areas are tallied and combined to produce the Top 100 Global Innovator list.

Top 100 Global Innovator list

3M Company USA Chemical 2011, 2012, 2013, 2014

Abbott Laboratories USA Pharmaceutical 2013, 2014

Advanced Micro Devices USA Semiconductor & Electronic Components 2011, 2012, 2013, 2014

Air Products USA Chemical 2013

Aisin Seiki Japan Automotive 2014

Alcatel-Lucent France Telecommunication & Equipment 2011, 2012, 2013, 2014

Alstom France Electrical Power

Amazon USA Media Internet Search & Navigation Systems

Analog Devices USA Semiconductor & Electronic Components 2011, 2012, 2013

Apple USA Telecommunication & Equipment 2011, 2012, 2013, 2014

Arkema France Chemical 2011, 2012, 2013, 2014

Avago Technologies (previously LSI) USA Semiconductor & Electronic Components 2011,2012, 2013, 2014

BASF Germany Chemical 2011, 2014

Bayer Germany Pharmaceutical 2011

Becton Dickinson USA Medical Devices

Blackberry Canada Telecommunication & Equipment 2013, 2014

Boehringer Ingelheim Germany Pharmaceutical

Boeing USA Aerospace 2011, 2012, 2013, 2014

Bridgestone Japan Automotive

Bristol-Myers Squibb USA Pharmaceutical 2011

Canon Japan Imaging 2011, 2012, 2013, 2014

Casio Computer Japan Computer Hardware 2014

Chevron USA Oil & Gas 2011, 2012, 2013

CNRS, The French National Center for Scientific Research France Scientific Research 2011, 2012, 2013, 2014

CEA–The French Alternative Energies and Atomic Energy Commission France Scientific Research 2011, 2012, 2013, 2014

Daikin Industries Japan Industrial 2011, 2014

Dow Chemical Company USA Chemical 2011, 2012, 2013, 2014

DuPont USA Chemical 2011, 2012, 2013, 2014

Emerson Electric USA Electrical Products 2012, 2013, 2014

Ericsson Sweden Telecommunication & Equipment 2011, 2012, 2013, 2014

Exxon Mobil USA Oil & Gas 2011, 2012, 2013

Fraunhofer Germany Scientific Research 2013, 2014

Freescale Semiconductor USA Semiconductor & Electronic Components 2013, 2014

Fujifilm Japan Imaging 2012, 2013, 2014

Fujitsu Japan Computer Hardware 2011, 2012, 2013, 2014

Furukawa Electric Japan Electrical Products 2014

General Electric USA Consumer Products 2011, 2012, 2013, 2014

Google (now Alphabet Inc.) USA Media Internet Search & Navigation Systems 2012, 2013, 2014

Hitachi Japan Computer Hardware 2011, 2012, 2013, 2014

Honda Motor Japan Automotive 2011, 2012, 2013, 2014

Honeywell International USA Electrical Products 2011, 2012, 2013, 2014

Idemitsu Kosan Japan Oil & Gas

IFP Energies Nouvelles France Scientific Research 2011, 2012, 2013, 2014

Intel USA Semiconductor & Electronic Components 2011, 2012, 2013, 2014

InterDigital USA Telecommunication & Equipment

Japan Science and Technology Agency (JST) Japan Scientific Research

Johnson & Johnson USA Pharmaceutical 2013, 2014

Johnson Controls USA Automotive

JTEKT Japan Automotive Kawasaki Heavy Industries Japan Industrial

Kobe Steel Japan Primary Metals 2014

Komatsu Japan Industrial 2014

Kyocera Japan Electrical Products 2014

LG Electronics S Korea Consumer Products 2011, 2012, 2013, 2014

Lockheed Martin USA Transportation Equipment 2012, 2013, 2014

LSIS S Korea Electrical Power 2011, 2012, 2013, 2014

Makita Corporation Japan Machinery

Marvell USA Semiconductor & Electronic Components 2012, 2013, 2014

MediaTek Taiwan Semiconductor & Electronic Components 2014

Medtronic USA Medical Devices 2014

Micron USA Semiconductor & Electronic Components 2012, 2013, 2014

Microsoft USA Computer Software 2011, 2012, 2013, 2014

Mitsubishi Electric Japan Electrical Products 2011, 2012, 2013, 2014

Mitsubishi Heavy Industries Japan Machinery 2012, 2013, 2014

Mitsui Chemicals Japan Chemical NEC Japan Computer Hardware 2011, 2012, 2013, 2014

Nike USA Consumer Products 2012, 2013, 2014

Nippon Steel & Sumitomo Metal Japan Primary Metals 2012, 2013, 2014

Nissan Motor Japan Automotive 2013, 2014

Nitto Denko Japan Chemical 2011, 2012, 2013, 2014

Novartis Switzerland Pharmaceutical 2014 2015

NTT Japan Telecommunication & Equipment 2011, 2012, 2013, 2014

Olympus Japan Healthcare Products 2011, 2012, 2013, 2014

Oracle USA Computer Software 2013, 2014

Panasonic Japan Consumer Products 2011, 2012, 2013, 2014

Philips Netherlands Electrical Products 2011, 2013, 2014

Qualcomm USA Semiconductor & Electronic Components 2011, 2012, 2013, 2014

Roche Switzerland Pharmaceutical 2011,2012,2013, 2014

Safran France Transportation Equipment 2013, 2014

Saint-Gobain France Industrial 2011, 2012, 2013, 2014

Samsung Electronics S Korea Semiconductor & Electronic Components 2011, 2012, 2013, 2014

Seagate USA Computer Hardware 2012, 2013, 2014

Seiko Epson Japan Imaging 2011, 2012, 2013, 2014

Shin-Etsu Chemical Japan Chemical 2011, 2012, 2013, 2014

Showa Denko Japan Chemical

Solvay Belgium Chemical 2012

Sony Japan Consumer Products 2011, 2012, 2013, 2014

Sumitomo Electric Japan Industrial 2011, 2013, 2014

Symantec USA Computer Software 2011, 2012, 2013, 2014

TE Connectivity Switzerland Semiconductor & Electronic Components 2011, 2012, 2013, 2014

Thales France Transportation Equipment 2012, 2013

Toray Japan Chemical

Toshiba Japan Computer Hardware 2011, 2012, 2013, 2014

Toyota Motor Japan Automotive 2011, 2012, 2013, 2014

Valeo France Automotive 2012, 2013

Xilinx USA Semiconductor & Electronic Components 2012, 2013, 2014

Yamaha Japan Consumer Products 2011, 2014

Yamaha Motor Japan Automotive

Yaskawa Electric Japan Industrial

Yazaki Japan Automotive




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Monoclonal Antibody Drug Immunogenicity

Larry H. Bernstein, MD, FCAP, Curator



Immunogenicity assessment of monoclonal antibodies
Category: Abstracted Scientific Content
Author(s):    Michelle Derbyshire, PhD, GaBI Online Editor

GaBI 2015; 4(4).

The most critical safety concern relating to biologicals (including biosimilars) is immunogenicity. This is especially important for monoclonal antibody (mAb) biologicals, which are large molecules with complex structures and functions and which represent the largest class of biologicals.

Immunogenicity is the ability to induce a humoral and/or cellmediated immune response. Most biologicals induce immune responses, because they are polypeptides or proteins and might therefore be recognized by the immune system as foreign. However, in most cases, the presence of antibodies is harmless and has little clinical consequence. The problem is that some cases of immunogenicity can cause problems or even be fatal, such as in the case of pure red cell aplasia. Such cases raise concerns about the potential clinical consequences of extensive use of biologicals and biosimilars. Given that biologicals may induce such unwanted immune responses it is essential to investigate the immunogenicity of a biological prior to marketing approval. This is especially important when considering that the problem with immunogenicity is that it is impossible to predict.

The immune response is influenced by many factors and data generated in pre-licensure studies may prove difficult to assess for regulators. Immunogenicity can be influenced by the product itself, e.g. structure, aggregation, dose, duration, but can also be affected by the patient, e.g. age, gender, ethnicity, immune status, genetic make-up. The knowledge and expertise required for assessment of immunogenicity requires a thorough understanding of animal and human immunology as well as specific product characteristics, including mechanism of action, antibody assays and assessment of results in a given clinical context. The appropriate interpretation of immunogenicity data is of critical importance for defining the safety profile of an mAb.

At the World Health Organization (WHO) implementation workshop on Evaluation of Biotherapeutic Products, held in Seoul, Republic of Korea, in May 2014, regulators and manufacturers participated in a workshop evaluating two case studies mimicking a real situation evaluating immunogenicity studies for two fictitious mAb products.

It was expected that after completing the workshop, participants would have an understanding of how immunogenicity studies are conducted and assessed. In addition, how the information obtained is used to make decisions relating to the appropriateness of the studies and how the observed immunogenicity impacts on the clinical use of the mAbs was also covered.

Predictive immunogenicity modelling algorithms, such as in silico and T cell studies, are showing promise for identification of potential immunogenic T cell epitopes. However, despite the promise of these predictive tests, human clinical data is still needed for determining immunogenicity. This cannot be replaced by use of animal or in vitro or in silico tools.

Suitability of the assays for immunogenicity assessment was highlighted as a topic of critical importance for conducting the case studies. In the case of biosimilars, the methods used to measure the incidence of immunogenicity and the immunogenic potential of biosimilars and reference biologicals can significantly impact the comparability of the two molecules, and therefore great care must be taken in the development and execution of assays to measure immunogenicity. The value of reviewing raw data for each individual subject in order to assess the impact of immunogenicity on effi cacy and safety was also clearly demonstrated in the case studies.

WHO guidelines state that the monitoring period for immunogenicity assessment depends on the intended duration of treatment and the expected time of antibody development. However, one of the examples in the case studies illustrated that a longer period of observation may be necessary to increase accuracy in assessing immunogenicity.

Discussion on additional indications and the need for additional immunogenicity studies revealed that the expectations in terms of the size and design of such studies differ among regulators and manufacturers. However, there was a consensus that the original case studies were limited and that additional data needed to be generated.

When it comes to biosimilar mAbs, it becomes an even more sophisticated exercise, and includes the challenge of addressing correlation between bioanalytical signals and clinical endpoints. The case studies highlighted the need to assess the methods used for appropriateness for use for their intended purpose and to interpret the data generated, taking into account their limitations.




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