Posts Tagged ‘MedImmune’

at #JPM16 – Moderna Therapeutics turns away an extra $200 million: with AstraZeneca (collaboration) & with Merck ($100 million investment)

Reporter: Aviva Lev-Ari, PhD, RN


per SOURCES quoted:


AstraZeneca, for one, has sent an important validating signal to outsiders by continuing to invest in 29 Moderna drug candidates at last count. The financial community can’t get enough. As ambitious as Moderna has been with a dream to disrupt conventional small molecule drugs and protein therapies, it recognizes it can only do so much. Moderna turned away an extra $200 million of investment that would have made its $500 million round a $700 million round. The company didn’t need that much. “It’s bizarre,” Bancel said. “I used to spend my time begging for money. Now I had to go to my board and say ‘We’re going to turn down $200 million.”




AstraZeneca and Moderna Therapeutics announce new collaboration to co-develop and co-commercialise immuno-oncology mRNA therapeutics™

PUBLISHED 11 January 2016

Moderna to lead preclinical development; AstraZeneca to lead clinical development; Moderna to co-commercialise and share profits on resulting products in the US

11 January 2016

AstraZeneca, along with its global biologics research and development arm, MedImmune, and Moderna Therapeutics today announced a new collaboration to discover, co-develop and co-commercialise messenger RNA (mRNA) therapeutic candidates for the treatment of a range of cancers. The collaboration is in addition to the agreement announced by the companies in 2013 to develop mRNA Therapeutics™ for the treatment of cardiovascular, metabolic and renal diseases as well as selected targets in oncology.

The collaboration will combine MedImmune’s protein engineering and cancer biology expertise with Moderna’s mRNA platform. mRNA-based therapies are an innovative treatment approach that enables the body to produce therapeutic protein in vivo, opening up new treatment options for a wide range of diseases that cannot be addressed today using existing technologies.




Merck Joins Messenger RNA Frenzy, Betting $100M On Moderna Therapeutics

I’m the founder and editor of Timmerman Report.

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Spirogen’s Cytotoxic Warheads IP Taken over for $400 Million by AstraZeneca PLC (AZN)’s MedImmune (AZN) for Avoidance Capabilities of Emergent Drug Resistance

Reporter: Aviva Lev-Ari, PhD, RN

AstraZeneca PLC (AZN)’s MedImmune (AZN) Takes OverSpirogen Ltd. for Up to $400 Million to Bolster Oncolology Portfolio

10/15/2013 7:54:15 AM


AstraZeneca PLC Oncology Portfolio Strengthened as MedImmune Acquisition of Spirogen Ltd. Boosts Antibody-Drug Conjugate Capability 

AstraZeneca PLC Oncology Portfolio Strengthened as MedImmune Acquisition of Spirogen Ltd. Boosts Antibody-Drug Conjugate Capability 

Tuesday, 15 October 2013 — AstraZeneca today announced that MedImmune, its global biologics research and development arm, has acquired Spirogen, a privately-held biotech company focused on antibody-drug conjugate technology for use in oncology.

MedImmune has also entered into a collaboration agreement with ADC Therapeutics to jointly develop two of ADC Therapeutics’ antibody-drug conjugate programmes in preclinical development. MedImmune will also make an equity investment in ADC Therapeutics, which has an existing licensing agreement with Spirogen.

MedImmune will acquire 100 per cent of Spirogen’s shares for an initial consideration of $200 million and deferred consideration of up to $240 million based on reaching predetermined development milestones. Existing out-licensing agreements and associated revenue streams are excluded from this acquisition.

MedImmune will also pay $20 million for an equity investment in ADC Therapeutics, which will be matched by Auven Therapeutics, the majority shareholder in both ADC Therapeutics and Spirogen. The collaboration agreement will include an upfront payment with predetermined development milestones for two programmes from a defined list and a cost- and profit-sharing arrangement with MedImmune representing the majority share. ADC Therapeutics will also have the option to co-promote one of the products in the US.

Antibody-drug conjugates are a clinically-validated cancer drug technology that offers both high potency and selective targeting of cancer cells. Spirogen’s proprietary pyrrolobenzodiazepine (PBD) technology attaches highly potent cytotoxic agents, or ‘warheads’ to specific cancer-targeting antibodies using biodegradable ‘linkers’. This targeting optimises the delivery of the cancer drug to the tumour cells only and provides the greatest degree of tumour killing while minimising the toxicity to the patient.

“Antibody-drug conjugates are ground-breaking technologies with the potential for directly targeting many types of cancer tumours while safeguarding healthy cells. The cutting-edge technologies developed by Spirogen and ADC Therapeutics complement MedImmune’s innovative antibody engineering capabilities, enabling us to accelerate antibody-drug conjugates into the clinic,” said Dr. Bahija Jallal, Executive Vice President, MedImmune.

Oncology is a core therapy area for AstraZeneca spanning both small molecule and biologics research and development. MedImmune is developing a comprehensive portfolio with an emphasis on two key areas in oncology development: antibody-drug conjugates and immune-mediated cancer therapy, which aims to harness the power of the patient’s own immune system to fight cancer. Together, immune-mediated cancer therapies and antibody-drug conjugates have the potential to treat cancer in a way that current therapies are unable to do.

“This deal reflects the very significant progress made by our scientists, most notably over the last two years, as we have applied our warhead and linker technologies to the development of highly potent and specific antibody-drug conjugates,” said Dr. Chris Martin, Chief Executive Officer, Spirogen. “We believe that pyrrolobenzodiazepine-armed antibody-drug conjugates will emerge as a critical component in the next generation of cancer biologics with the potential to make a difference for oncologists and their patients. We look forward to combining our world-class capabilities in this area with MedImmune’s ability to develop this exciting class of oncology drugs.”

About Antibody-Drug Conjugates

An antibody-drug conjugate is a three-component system consisting of a potent cytotoxic agent, or ‘warhead’, a biodegradable linker and a monoclonal antibody. The antibody binds to specific markers at the surface of the cancer cell. The whole antibody-drug conjugate is then internalised within the cancer cell where the active drug is released. Antibody-drug conjugates have extensive potential therapeutic applications in several disease areas, particularly in cancer. The principle can also be applied beyond antibodies, with the possibility of linking warheads to antibody fragments, peptides, vitamins and hormones.

About Spirogen

The Spirogen group was founded in 2001 as a spin-out from several institutions including University College London and with partial funding by Cancer Research UK. It is majority owned by Auven Therapeutics. It has developed a novel class of highly potent cytotoxic warheads based on its proprietary pyrrolobenzodiazepines (PBDs), DNA minor groove binding agents, which bind and cross-link specific sites of DNA of the cancer cell. This blocks the cancer cells’ division without distorting its DNA helix, thus potentially avoiding the common phenomenon of emergent drug resistance. Spirogen has been developing its PBD technology for more than ten years, including a standalone PBD agent in a Phase II study in acute myeloid leukemia. Its business model has been to partner its technology with pharma and biotech for use in the development of novel drugs. It has a number of industry collaborations, including collaborations with Genentech announced in 2011 and with ADC Therapeutics announced in 2012. For further information, please visit: http://www.spirogen.com.

About ADC Therapeutics

ADC Therapeutics (ADCT) is a Swiss-based oncology drug development company that specialises in the development of proprietary antibody-drug conjugates (ADCs) targeting major cancers such as breast, lung, prostate, renal and blood. The company’s ADCs are highly targeted drug constructs which combine monoclonal antibodies specific to particular types of tumour cells with a novel class of highly potent PBD-based warheads. The company was launched in 2012 with a $50m commitment from private equity firm Auven Therapeutics. ADCT has access to warhead and linker chemistries via existing agreements with Spirogen. It operates a virtual business model based in Lausanne, Switzerland. For further information, please visit: http://www.adctherapeutics.com.

About Auven Therapeutics

Auven Therapeutics was founded by Stephen Evans-Freke and Dr. Peter B Corr in 2007 with an innovative investment strategy that enables it to operate as a drug development company while remaining structured as a private equity fund. Auven Therapeutics has a portfolio of biologic and small molecule drug candidates for a range of therapeutic indications including cancer, ophthalmic conditions, women’s health and orphan diseases. Auven manages its drug development activities from its bases in Lausanne, Switzerland, New York, USA and Hamilton, Bermuda. Auven Therapeutics Management LLLP, based in the US Virgin Islands, serves as its Investment Advisor. For further information, please visit: http://www.auventx.com.

About MedImmune

MedImmune is the worldwide biologics research and development arm of AstraZeneca. MedImmune is pioneering innovative research and exploring novel pathways across key therapeutic areas, including respiratory, inflammation and autoimmunity; cardiovascular and metabolic disease; oncology; neuroscience, and infection and vaccines. The MedImmune headquarters is located in Gaithersburg, MD, one of AstraZeneca’s three global R&D centres. For more information, please visit http://www.medimmune.com.

About AstraZeneca

AstraZeneca is a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of cardiovascular, metabolic, respiratory, inflammation, autoimmune, oncology, infection and neuroscience diseases. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information please visit: http://www.astrazeneca.com




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Curator: Aviva Lev-Ari, PhD, RN

If Biologics will help increase HDL in wide market penetration, the market share of Statins will be negatively impacted.

The biologics was developed by NIH funding, as reported on 2/14/2012, see last section, below.

NHLBI SMARTT Program Awards AlphaCore Pharma Funding to Manufacture Potential Treatment for Familial Lecithin-Cholesterol Acyltransferase (LCAT) Deficiency


In an Interview I had with the VP of Scientific Affairs at AstraZenaca on 3/18/2013, the Executive Dr. D.S., MD, PhD, told me that the Cardiovascular Therapeutic Area at AstraZeneca is at present and in the future, probably the most important one of all of its businesses to date, thus, the position he is interviewing for, Director of Scientific Affairs Cardiovascular, will be the most powerful one within the Scientific Affairs Office.

Per my discussion of BRILINTA (ticagrelor), referring the VP to my post on this topic on 12/28/2012,

PLATO Trial on ACS: BRILINTA (ticagrelor) better than Plavix® (clopidogrel bisulfate): Lowering chances of having another heart attack

VP said, “the position will be beyond BRILINTA, or Cardiovascular.” A candidate not found yet. AZ keeps on calling, Keeps searching.

AstraZeneca – The Biggest R&D Spenders In Biopharma

Company: AstraZeneca

2011 spending: $5.5 billion
2010 spending: $5.3 billion
Change: +3.6%
Percentage of revenue: 16.3%

Like several other top 10 pharma companies, AstraZeneca ($AZN) saw its R&D expenses climb somewhat in 2011. But this year, as CEO David Brennan unveiled the annual results for 2011, he started with a new restructuring plan. And R&D is intended to bear some of the biggest cuts.

Hit with sliding profits and eviscerated by analysts for one of the weakest late-stage pipelines in the Big Pharma business, Brennan had to do something significant. Of more than 7,000 pink slips being readied, 2,200 were being reserved for R&D as the company moved to shutter R&D facilities in Soedertaelje in Sweden and Montreal. Neuroscience, once a key feature in the pipeline, is being scaled way back, with plans to field a “virtual” team in key hubs.

AstraZeneca became the poster child for the R&D quagmire when Forbes‘ Matthew Herper concluded that AstraZeneca had the worst ratio of R&D costs to approvals in the industry. For a company that went 6 years without a drug approval ahead of the 2009 OK for Onglyza, accumulated setbacks have reached a breaking point.

AstraZeneca, though, can’t cut its way to a turnaround in R&D. That’s going to take new programs and new technologies. It only began to address the issue with a licensing pact for a slate of Amgen antibodies. Research chief Martin Mackay was quick to follow up by telling Reuters‘ Ben Hirschler that more deals were coming. And indeed just weeks later, AstraZeneca acquired a late-stage gout drug with the $1.26 billion buyout of Ardea. The fact that AstraZeneca didn’t bother to stick with its disease strategy, and quickly indicated that it wouldn’t in the future, underscored just how crucial it is to move fast.

Nevertheless, AstraZeneca will find it hard to shake its legacy of failures. Just weeks ago the company was forced to wash its hands of a billion-dollar deal with Targacept ($TRGT) for a prospective depression drug that failed 4 out of 4 late-stage studies. And as criticism mounted, Brennan has been forced to adopt a defensive posture.

“I read and hear and see lots of things, but we’re here trying to change policy, make good decisions and execute our strategy,” the CEO told Bloomberg, vowing to stick to the game plan. “Maybe somebody sees something different, but spending more money does not have a linear increase in the number of returns you get from a research and development perspective.”

AstraZeneca – The Biggest R&D Spenders In Biopharma – FierceBiotech http://www.fiercebiotech.com/special-reports/biggest-rd-spenders-biopharma/astrazeneca-biggest-rd-spenders-biopharma#ixzz2PQN8is2U

On April 3, 2013, FierceBiotech reported that


3 April 2013

AstraZeneca today announced that MedImmune, its global biologics research and development arm, has acquired AlphaCore Pharma, an Ann Arbor, Michigan-based biotechnology company focused on the development of ACP-501, a recombinant human lecithin-cholesterol acyltransferase (LCAT) enzyme.

LCAT, an enzyme in the bloodstream, is a key component in the reverse cholesterol transport (RCT) system, which is thought to play a major role in driving the removal of cholesterol from the body and may be critical in the management of high-density lipoprotein (HDL) cholesterol levels.  The LCAT enzyme could also play a role in a rare, hereditary disorder called familial LCAT deficiency (FLD) in which the LCAT enzyme is absent.

Cardiovascular and metabolic disease is a core therapy area for AstraZeneca’s small and large molecule research.

“As the science in this area continues to evolve, we are committed to exploring unique pathways that could lead to new combination or standalone therapies for patients living with chronic and acute cardiovascular diseases,” said Dr. Bahija Jallal, Executive Vice President, MedImmune. “Cardiovascular disease is projected to remain the single leading cause of death worldwide over the next decade and beyond. Through novel approaches like LCAT, we hope to shift the treatment paradigms in this area to help prevent and treat these conditions.”

In 2012, results from a Phase I clinical trial of ACP-501 met the primary safety and tolerability endpoints.  No serious adverse events were reported.  ACP-501 also met the study’s secondary endpoints by rapidly and substantially elevating HDL cholesterol.  The data from this study support ongoing clinical development of ACP-501.

MEDIMMUNE, ASTRAZENECA’S BIOLOGICS ARM, ACQUIRES ALPHACORE PHARMA – FierceBiotech http://www.fiercebiotech.com/press-releases/medimmune-astrazenecas-biologics-arm-acquires-alphacore-pharma#ixzz2PQL73X3f

 AstraZeneca gambles on cardio therapy in AlphaCore buyout

By Ryan McBride

In another early-stage bet, AstraZeneca’s MedImmune unit acquired the biotech AlphaCore Pharma. The deal comes as AstraZeneca ($AZN) reboots a floundering R&D effort and adds a recombinant LCAT enzyme therapy from AlphaCore that could combat cardiovascular disease.

MedImmune, the biologics division of Astra, faces years of additional development before AlphaCore’s ACP-501 becomes part of the London-based pharma group’s late-stage pipeline, which has many holes yet to be filled. Last year, Ann Arbor, MI-based AlphaCore touted Phase I work on ACP-501, reporting that the enzyme therapy was well-tolerated and quickly boosted levels of HDL or “good” cholesterol in patients.

AZ CEO Pascal Soriot

New AstraZeneca CEO Pascal Soriot has signaled his desire to wager on new science amid an overhaul of R&D announced last month that will cost 1,600 research jobs across the company and after the ouster of former R&D chief Martin Mackay in January. Bahija Jallal, executive vice president of MedImmune, survived the round of cutbacks and plans to pursue new biologics such as ACP-501, which she stated could treat both acute and chronic cardiovascular disease.

“Cardiovascular disease is projected to remain the single leading cause of death worldwide over the next decade and beyond,” Jallal said. “Through novel approaches like LCAT, we hope to shift the treatment paradigms in this area to help prevent and treat these conditions.”

The ACP-501 is an engineered version of the natural LCAT enzyme from the liver that plays a role in ridding the body of cholesterol and keeping up levels of beneficial HDL cholesterol. The candidate could aid millions of patients with cholesterol problems as well as those with a rare inherited disease called familial LCAT deficiency that robs the body of the enzyme.

Bahija Jallal, EVP of MedImmune

The AlphaCore buyout comes on the heels of AstraZeneca’s sizable $240 million upfront payment to Moderna Therapeutics to get in early on the startup’s preclinical programs that use messenger RNA to turn cells in the body into makers of healing proteins. The financial details of the AlphaCore buyout weren’t disclosed.

Still, analysts expect Soriot to pull the trigger on larger deals to bolster the late-stage pipeline or even provide marketed products as AstraZeneca faces the impact of patent expirations on blockbuster cholesterol pill Crestor and the heartburn med Nexium. As Reuters noted, the company has only 6 drugs in late-stage development and aims to double that number by 2016.

February 14, 2012 09:17 AM Eastern Daylight Time

NHLBI SMARTT Program Awards AlphaCore Pharma Funding to Manufacture Potential Treatment for Familial Lecithin-Cholesterol Acyltransferase (LCAT) Deficiency

ANN ARBOR, Mich. & ROCKVILLE, Md.–(BUSINESS WIRE)–AlphaCore Pharma, a biopharmaceutical company, and Advanced Bioscience Laboratories (ABL), a biomedical contract research and manufacturing company, today announce funding from the National Institutes of Health, National Heart, Lung and Blood Institute (NHLBI) “Science Moving towards Research Translation and Therapy” (SMARTT) program, to manufacture recombinant human lecithin-cholesterol acyltransferase (rhLCAT) for the treatment of familial LCAT deficiency.

“This is a significant step towards developing a treatment for familial LCAT deficiency. We are pleased by the strong support from the NHLBI and ABL and look forward to advancing this program.”

Also known as ACP-501, rhLCAT represents a promising new approach in the fight against atherosclerotic cardiovascular disease, and has demonstrated preclinical efficacy in promoting HDL maturation and cholesterol flux, a natural process by which cholesterol is removed from the body. Currently, ACP-501 is in Phase 1 clinical development with the eventual goal of reducing the risk of cardiovascular events in patients presenting with acute coronary syndrome. Manufacturing support from the NHLBI SMARTT program will enable production of additional material that will be used to determine the safety and efficacy of rhLCAT enzyme replacement therapy for patients with familial LCAT deficiency – a potentially life-threatening illness for which there is no FDA-approved treatment.

“This is a significant step towards developing a treatment for familial LCAT deficiency. We are pleased by the strong support from the NHLBI and ABL and look forward to advancing this program.” said AlphaCore President, Bruce Auerbach.

The enzyme, rhLCAT, will be produced by ABL in its Rockville, MD biologics production facility under a contract from the NHLBI SMARTT program. Dr. Thomas VanCott, ABL’s President and Chief Executive Officer stated, “ABL is privileged to be working with AlphaCore Pharma in support of their ACP-501 (rhLCAT) program. Research in rare genetic diseases can encounter funding hurdles, yet through this NHLBI-sponsored manufacturing project we have the potential to advance an urgently needed enzyme replacement therapy. This effort further demonstrates ABL’s expertise of partnering with the NIH to support major development programs and our commitment to deliver the highest quality cGMP biologics to our clients in a cost-effective manner.”

Peter Greenleaf, chief executive of MedImmune. (Jeffrey MacMillan – JEFFREY MACMILLAN)Peter Greenleaf is stepping down down as president of Gaithersburg-based biotechnology giant MedImmune, according to a company spokesman, to take the helm of parent company AstraZeneca’s Latin America business.

He will be replaced by Bahija Jallal, who currently serves as MedImmune’s executive vice president of research and development. Jallal joined the company in 2006 as vice president of translational sciences.

The leadership change comes as MedImmune was formally designated a biologics research and development site for AstraZeneca, meaning Jallal will report directly to AstraZeneca chief executive Pascal Soriot, said company spokesman Mike O’Brien.

He added that MedImmune’s commercial organization will now report into AstraZeneca’s North American business and its manufacturing group will be folded into AstraZeneca’s global operations group.

“There’s no new news on jobs today,” O’Brien said. “The driver for these changes is not cost but even faster decision-making in key areas of the business and a need to reduce complexity.”

O’Brien said Greenleaf will continue to be based in Maryland, where he has become a figure­head of sorts for the life sciences industry.

Greenleaf was an advocate for Democratic Gov. Martin O’Malley’s InvestMaryland initiative, which allocates state money for investment in local upstarts. He serves as chairman of the Maryland Venture Fund Authority, a nine-member board assigned to oversee its implementation.

MedImmune has long been an anchor of Maryland’s biotechnology hub along the Interstate 270 corridor. The company was purchased by AstraZeneca in 2007 for $15.6 billion, a sales price that some industry observers still question.

The Washington Business Journal reported the personnel changes earlier.



Mineo C, Yuhanna IS, Quon MJ, Shaul PW., (2003). HDL-induced eNOS activation is mediated by Akt and MAP kinases. J. Biol. Chem., 278:9142–9149.

Shaul, PW and Mineo, C, (2004). HDL action on the vascular wall: is the answer NO? J Clin Invest., 15; 113(4): 509–513.

Other related articles to this topic on the Open Access Online Scientific Journal include the following:

Aviva Lev-Ari, PhD, RN, 4/7/2013


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Reporter: Aviva Lev-Ari, PhD, RN

Demonstrate Biosimilarity with 100% confidence. Everytime

While offering greater flexibility, new FDA biosimilar guidelines lack specificity, nothing in the development can be taken for granted.

Develop biosimilars that exhibit comparability in every required category and avoid ever having a biosimilar application rejected. With so much at stake, are you 100% confident of demonstrating comparability every time? 

Demonstrate Biosimilarity is your opportunity to get first hand case studies from the leading biosimilar and generic drug developers as well as the FDA on the best approaches to…

  1. Interpret regulatory ambiguity and determine how much similarity must be demonstrated to gain approval and achieve interchangeability
  2. Ensure stability and analytical comparability as well as comparability in safety and PK/PD by optimizing your quality assessment strategy
  3. Reduce the impact of immunogenicity in your biosimilar drug and discover how to translate this regulatory expectation into a clinical trial concept
  4. Optimize your approaches to protein characterization with practical guidance from industry leading analytical scientists and service providers

Demonstrate Biosimilarity will tackle burning issues surrounding naming and pricing policiesoriginators defence strategiesmanufacturing challenges andhow to identify the best target for biosimilar development. Achieve technical insights and obtain cutting edge intelligence from 16 pharma case studies showcasing biosimilar best practise.

Attend and gain first hand testimony from the FDA, that combined with this depth of insight will give you the information you need to move quickly and decisively to get your biosimilar approved.

What you will learn? 

Attend Demonstrate Biosimilarity to:

  • Learn how to effectively monitor the higher-order structure and associated structural dynamics of your biosimilar drug molecules by optimizing your approach to comparability studies with practical guidance from Biogen Idec
  •  Expand your knowledge of how protein aggregates interact with the immune system and how this understanding can be used to reduce immunogenicitywith the Chief Medical Research Officer at the FDA
  •  More effectively determine the extent of physiochemical, pre-clinical and clinical characterization required to demonstrate biosimilarity and gain approval with case studies from Novartis and Sandoz
  • Enhance harmonization of your biosimilar data with a comprehensive comparison of the EMEA Vs USA regulatory landscape with Sandoz, Pfizerand Wokhardt
  • Get insight from MedImmune to achieve comparability when test methods are changed during the product life cycle and a non-inferior, equivalent, or superior replacement study model has been selected
  • Understand fully the applications of bioassays to determine product potency, aid biological characterization, test comparability, and determine stability with expert advice and clinical data from Teva Pharmaceuticals
  • Get a first hand guide from the FDA regarding quantitation and characterization of protein aggregates in biosimilars to ensure quality and safety of products,consistency of manufacture between lots and comparability when the manufacturing process has been changed

Does the FDA biosimilar guidance go far enough? Not if you want to operate with 100% confidence.

In fact the FDA still wants to take a cautious case by case approach. Complex biosimilar comparability studies are inherently risky and the consequences of a rejected submission are dramatic. This means that an open dialogue with FDA & other developers is essential.

Our recent industry survey highlighted two key desires for drug developers:


To know exactly how other drug developers are tackling challenges such as structural comparability, immunogenicity and interchangeability

What the FDA is currently thinking and how this would apply to their development programs

In response to this demand for insight and support we’ve assembled the FDA alongside the industry’s best at Demonstrate Biosimilarity Washington DC (13-14th February).


Learn from the testimony of 18 industry speakers including Sandoz, Teva, Wokhardt, GSK, Pfizer, MedImmune, Biogen Idec, Amgen, Novartis, Merck and senior FDA representatives dedicated to answering these questions.  This will ensure that you can effectively achieve comparability , demonstrate biosimilarity and avoid costly failed submissions. View the agenda now.

Overcome challenges such as:


  • How do I assess the level of biological purity needed to match my innovator product and avoid making several submission to regulators?
  • Is it truly possible to achieve interchangeability and how can I convince patients and physicians of the equivalence of my biosimilar and ensure market access?
  • What are the best practises to decrease immunogenicity risk to increase my chances of achieving comparability?
  • Where can I obtain a suitable reference product to allow me to begin characterization?
  • What is the FDA’s current thinking on the criteria for analytical, stability, safety and PK/PD comparability?

Download the brochure now to view the full agenda, workshops & speaker line up.


This meeting will provide you with unrivalled access to 15 case study lead presentations from pioneering biosimilar and generic drug developers sharing their innovative new approaches.

Learn how your peers are decreasing the clinical work required, minimising the impact of immunogenicity, achieving interchangeability and how they define targets for biosimilar development.

If that isn’t enough, the FDA will be giving keynote presentations on the regulatory expectations regarding aggregates and comparability as well as minimizing the impact of immunogenicity by understanding the role of protein aggregates in unwanted immunogenicity.




Day One

13th February, 2013

8.00 Registration

8.55 Chair’s Opening Remarks 

Optimizing Quality Assessments: Biochemical and Physiochemical Properties

9.00 Regulatory Expectations Regarding Aggregates and Comparability

• A guide from the FDA regarding quantitation and characterization of protein aggregates to ensure quality and safety, consistency of manufacture and comparability when the manufacturing process is being changed

• Overcoming challenges associated with characterization of the aggregates’ full size range to maximize safety information

Ewa Marszal, Chemist, Laboratory of Plasma Derivatives, Division of Haematology, CBER, FDA

9.30 An Industry Perspective: Optimizing Approaches to Protein Characterization in the Development of Biosimilars

• Practical guidance for optimizing analytical characterization of commercial products

• Understanding the innovation required in both technical development and clinical development

• Devising a systematic engineering approach to match biosimilar to reference product

Roxana Butoi, Manager, Biosimilars, GSK

10.00 Solution Spotlight: Analytical methods for monitoring biosimilar glycosylation

Scott Barksdale, Director, Business Development, Procognia

10.15 Speed Networking & Morning Refreshments

11.45 CASE STUDY: Implementing Advanced Analytical Methods and Regulatory Approved Comparability Strategies

• Numerous case studies demonstrating when test methods are changed during the product life cycle and a non-inferior, equivalent, or superior replacement study model has been selected based on the intended use of the new test method

• How to set risk-based acceptance criteria from product specifications and existing manufacturing process knowledge

Stephan Krause, PDA Task Force Leader for Analytical Methods, and Principal Scientist, Analytical Biochemistry, MedImmune

12.15 Application of Hydrogen/Deuterium Exchange with Mass Spec Detection (H/DX-MS) to Assess Comparability 

• Applications of instrumental hardware and computer software to enable H/DX-MS to be employed in a practical and routine way to assess biosimilarity

• Optimizing comparability studies to monitor the higherorder structure and associated dynamics of biosimilar drugs

Steven Berkowitz, Principal Investigator, Analytical Development, Biogen Idec

12.45 Effective use of Bioassays to Streamline Biosimilar Development 

• Applications of bioassays to determine product potency, aid biological characterization, and test comparability

• Bioassays as a bioanalytical tool in support of pre clinical and clinical studies throughout the span of biosimilar development

• Developing an assay strategy and clear understanding of regulatory expectations, development and implementation of validated biological assays to ensure approval

Patrick Liu, Senior Director and Global Head of Bioassays, Teva Pharmaceuticals

1:15 Lunch & Networking

2.15 CASE STUDY: Correcting Biases in Light Obscuration and Light Scattering Measurements of Protein Particles 

• A demonstration of the applications of optical models for measuring of protein particles

• Several case studies involving protein aggregates, examining size errors and the practicality of corrections for biosimilar development

Dean Ripple, Leader, Bioprocess Measurements Group, National Institute of Standards and Technology

Demonstrating Biosimilarity: Biochemical and Physiochemical Properties

Roxana Butoi, Manager, Biosimilars, GSK 

Stephan KrausePDA Task Force Leader for Analytical Methods, and Principal Scientist, Analytical Biochemistry, MedImmune

Steven Berkowitz, Principal Investigator, Analytical Development, Biogen Idec

Patrick Liu, Senior Director and Global Head of Bioassays, Teva Pharmaceuticals

3.15 Afternoon Refreshments & Networking  

Achieving Interchangeability with a Biosimilar Product

3.45 Application of Biophysical Techniques in Comparability Exercises: Quantitative Assessment of Spectral Similarity

• Effective use of biophysical tools, including circular dichroism spectroscopy to provide qualitative assessment of the similarity in higher order structure for biological molecules

• Using statistical analysis to provide a more quantitative evaluation of spectral similarity

Qin Zou, Senior Principal Scientist, BioProcess Analytics, Pfizer  

4.15 The Momenta Approach to Developing Biosimilars and Potentially Interchangeable Biologics

• Establishing biosimilarity and potential interchangeability by focussing on “comparison” between RPP and biosimilar

• An overview of Momenta’s key takeaways from the recently issued FDA draft guidance

Jim Roach, SVP, Development and Chief Medical Officer, Momenta Pharmaceuticals

4.45 Comparability and Biosimilarity – Two Sides of the Same (or a Different) Coin? 

• Compare and contrast the comparability and biosimilarity paradigms, with consideration for the impact of Quality by Design on these product development strategies

• The challenges and future directions of product characterization for biosimilars

Brent Kendrick, Director of Analytical Sciences, Amgen

5.15 Chair’s Closing Remark


Day Two

14th February, 2013

8.15 Registration

9.10 Chair’s opening remarks

9.15 Analysis of the Biosimilar Development Pipeline

• Gain a better understanding of how the biosimilar market will develop and evolve in light of the biosimilarity guidelines

• Understand when products will begin entering the U.S. and European markets

• What different development approaches are companies taking?

• What will be the involvement of companies in the US/EU

vs. those in developing countries?

Ronald Rader, President, Biotechnology Information Institute

9.45 Minimizing the Impact of Immunogenicity Understanding the Role of Protein Aggregates in Unwanted Immunogenicity

• How subvisible protein aggregates may interact with the immune system, their potential impact on product safety and efficacy

• An FDA perspective on the current regulatory considerations pertaining to the control of these particulates

Jack Ragheb, Chief Medical Research Officer, CDER, FDA

10.15 Morning Refreshments & Networking

Navigating the Regulatory and Legal Environment for Biosimilars

11.00 Comparison of the EMEA and USA Regulation for Biosimilar Development

• A review of the current state of initiatives for biosimilars in both EMEA and USA

• Perspective on how regulatory uncertainty could be reduced in the implementation of quality by design arguments

• A comprehensive comparison of the EMEA Vs USA landscape an the intricacies of harmonization

Ajaz Hussain, Chief Scientific Officer, Workhardt

11.30 An Industry Perspective: USA Current and Future Regulatory Setting for Biosimilars 

• Understanding the need to maximize productivity of FDA biosimilars development meetings

• Identifying and justifying differences between structural and functional characterization

• Negotiating regulatory landscape for clinical development and determining the required magnitude of the program

• Pursuing interchangeability for your biosimilar product without FDA guidance

John Pakulski, Senior Director and Head US Biopharmaceutical Regulatory Affairs, Sandoz

12.00 Biosimilar Regulation Roundtable Session 

• An opportunity for delegates to discuss with the regulators and regulatory experts the recent FDA biosimilar guidelines

• Collaboratively discuss the impact of the regulation and strategies to comply with it

12.45 Lunch & Networking

2.00 A Practical Guide and Overview of Current Strategies of Biocomparability and Biosimilarity 

• Industry perspective on the current guidance from EMEA and FDA for biocomparability

• Current strategies to assess the the extent of physiochemical, pre-clinical and clinical characterization based on the stage of development

• Emerging guidance on biosimilarity and the implications on the pre-clinical and clinical development programs for biosimilars

• Use of biomarkers in the biocomparability exercise: Are we there yet?

Shefali Kakar, Senior Fellow, Clinical Pharmacology, Oncology Business Unit, Novartis

2.30 Understand the Nuances and Implications of the FDA Guidelines on Biosimilars

• A detailed examination of the content of the recently released FDA guidelines and how to best understand the requirements for quantity of data, sources of material and types of studies permitted

• What kind of potential exists for increased efficiency, collaboration and cost-effectiveness in the U.S?

• The repercussions of important FDA decisions that have been made regarding user fees to be paid by companies in order to submit an application for a biosimilar

Helen Hartman, Regulatory Affairs Strategist, Pfizer

3.00 Afternoon Refreshments & Networking

Optimizing Approaches for Biosimilar Production and Manufacture

3.30 CASE STUDY: Evaluation of Comparability due to Changes in Scale-up, Process, Manufacturing Site, and Formulation 

• A case study of a preliminary comparability study used to evaluate the changes due to scale-up, manufacturing site, process and formulation of batches used in phases I/II and III

• A detailed overview of the extended characterization results from this study

• Preliminary assessment of comparability studies to establish the type of analytical testing required to correlate manufacturing changes to the product characteristics in the final comparability testing

Soundara Soundararajan, Principal Scientist, Bioprocess Development, Merck

4.00 Biosimilars: The Age of Post-Patent Medicine

• How will the thorny issues of safety, efficacy and cost (and in that order) impact the role of biosimilars in 21st century healthcare

• A detailed look at the challenges associated with demonstrating biosimilarity to the relevant regulatory bodies

Peter Pitts, President, Center for Medicine in the Public Interest

4.30 Chair’s Closing Remarks



Pre Conference Workshops: 12th February 2013

Workshop A) 08.00 – 11.00: Measurement, Characterization and Impactof Impurities for Biosimilars

Biotechnology and biosimilar products have substantial differences fromchemical entities in their starting materials, manufacturing processes, productcharacteristics, stability profiles, and interactions with containers and closures.Each of these can impact the nature of the impurities present in the finalproduct.

This workshop will provide an overview of these differences with links tothe current regulatory expectations and notes on current ‘best practices’for impurities assessment during development.

  • Impurities in Biotechnology/Biosimilar Products – What Makes themCritical?

– What are the specific guidance requirements for biosimilar productimpurities?

– What elements impact meaningful, reliable specifications for processand product impurities?

– How are the risks of impurities managed during development?

  • Example of Critical Process-Related Impurities: Host Cell Proteins

– What are the key requirements for measuring host cell proteins fromvarious expression systems?

– How should host cell protein assays be selected, optimized, and validated?

  • Example of Critical Product-Related Impurities: Particulates

– What types of particulates are of concern?

– How do we measure particulates?

  • Example of Critical Container/Closure Related Impurities: Extractables/Leachables

– How do we identify the extractables and leachables?

– How can extractables or leachables affect the product?

You will leave this workshop with a detailed understanding of the impact of key impurities in biosimilar development, through case study examples ofeach, to review the current and emerging issues for biotechnology productsassociated with each.

Led By: Nadine Ritter, Senior CMC Consultant, Biologics Consulting Group


Workshop B) 12.00 – 15.00: Strategies for an Abbreviated Clinical Programfor Biosimilar mAbs

This workshop is designed to give you a practical guide to develop theoptimum clinical strategy for developing a biosimilar to minimize the size ofthe clinical program require to demonstrate biosimilarityIn this workshop you will:

  • Understand how to design an abbreviated clinical program for biosimilardrugs
  • Learn to optimize phase I by developing a streamlined FIM PK study usingreference product and PK equivalence as endpoints
  • Discuss best practices for phase III dose and time response design
  • Develop solutions to challenges associated with safety, immunogenicity, interchangeability and extrapolation across multiple indications

You will leave this workshop with a step by step guide to taking a biosimilarthrough clinical development that will minimize the amount of clinical workrequired without compromising the quality or quantity of clinical evidence.

Led by: Partha Roy, Principle Consultant, PAREXEL Consulting

Please note that workshop B runs at the same time as workshop C – so you cannot attend both.

Workshop C) 12.00 – 15.00: Biosimilars: Is the Risk worth the Reward?

This workshop will demonstrate how you can capitalize on the recentregulatory developments, and offer all of the information you need tomove quickly and decisively to turn the streamlined biosimilars processinto a lucrative commercial opportunity.In this workshop you will:

  • Gain an understanding of the potential of the biosimilar market andassess the potential entry routes available for your business
  • Get an overview via numerous case studies of the latest progress inthe biosimilar field and discover how you can capitalize on the latestdevelopments
  • Understand the complex issue of biosimilar ROI and discusspractical strategies to maximize your return
  • Hear both provider and payer perspectives to gain a betterunderstanding of how you can cater to both of their needs

You will leave this workshop with all the information you need todevelop an effective commercially viable biosimilar development strategy.

Led by: James Harris, Chief Executive Officer, Healthcare Economics LLC

Please note that workshop B runs at the same time as workshop C – so you cannot attend both.


Workshop D) 15.30 – 18.30: Gain a Biosimilar Market Overview:Present and Future Challenges

Biosimilars present a new set of challenges for regulatory authoritieswhen compared with conventional generics. After many years in theslow lane, changes are driving new momentum in the market forbiosimilarsIn this workshop you will:

  • Analyze with industry leaders the present and future actions of themain players in biosimilar market in different regions of the world tohelp identify best practises from varying geo-specific approaches
  • Get an understanding of the patents used for the first generation ofapproved biopharmaceuticals and discover how to capitalize onpatents about to expire to open up new opportunities in the biosimilarmarket
  • Understand the critical issues for healthcare professionals surroundingthe use of biosimilars to make informed treatment decisions

You will leave this workshop with a greater understanding of thebiosimilar market place and an actionable biosimilar strategyincorporating the best practises discussed during this case study anddiscussion lead workshop.

Led by: Leandro Mieravilla, Global Market Manager mAbs, Cassara Biotech


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