Record Attendance Expected! Don't Miss IBC's 18th Annual TIDES

Register by Friday, April 15th and save $300! To save, use the code B16180BLOG100

Experts Provide Key Success Factors for Drug Development and Manufacturing:

Mixed-Incretin Receptor Biochemical Signaling at Multiple Receptors
Richard DiMarchi, Ph.D. Standiford H. Cox Professor of Chemistry, Indiana University

Drisapersen: Case Study
Robert Baffi, Ph.D. Executive VP, Technical Operations, BioMarin Pharmaceuticals

Quark's Long History of Oligonucleotide Development
Danny Zurr, Ph.D. Chairman and CEO, Quark Pharmaceuticals Inc. 

Technologies for Targeting Molecules to Sites of Diseases
John Reed MD, Ph.D Global Head of Roche Pharma Research and Early Development, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd.


TIDES 2016 is on pace for record attendance with 800+ global attendees expected to attend this year’s meeting, held May 9-12, 2016 in Long Beach, California – will you be there?

Register your team today and access the science, technologies and contracts you need to accelerate your oligonucleotide and peptide products through the clinic towards commercial success.

Your registration grants you access to:

• 800+ attendees representing 25 countries help you forge new business and scientific collaborations
• 100+ speaker presentations across 5 tracks provide a customized agenda of solutions for your company’s current challenges
• 70+ exhibitors demonstrate the latest products and technologies to accelerate your therapeutic to market
• 40+ poster presentations keep you abreast of cutting-edge industry and academic research

Plus, be sure to register this week to gain access to Attendee Connect powered by partneringONE, the digital networking platform that allows you to view the full attendee list, pre-schedule meetings, and send private messages to attendees before, during and after the event.

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Exclusive Interview with Dr. Bruce Riser, CEO, BLR-Bio, LLC

IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Bruce L. Riser, Ph.D., CEO, BLR-Bio, LLC for an exclusive interview that delves into the topics he plans to discuss at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA.

What are the biggest challenges of bringing new agents with novel targets to the market?

BR: There are really two big challenges when bringing new agents with novel targets to the market. First, many investors and potential partners are not familiar with the target, or they do not have the comfort that exists with an established target. Second, there can be misperceptions in the scientific community and in the biopharmaceutical industry where an agent is associated with another type of development creating a negative halo. On the first challenge, it is easy to see a proven target and to line up, jumping on the bandwagon with others trying to either win the race to launch first or to attempt to find a niche in an already established area. The problem of course is that lining up with everyone else does not provide the opportunity to develop and launch a true breakthrough therapy that can change the treatment paradigms and have a breakthrough impact on peoples' lives. Instead, investors and companies that line up for the established target often find they are trailing others to the market and are never able to successfully establish themselves in the market. It really takes a visionary to see the potential of a new target and have the insight and strength to invest in it. The second challenge, dealing with miss-associations of a novel therapy with other developments can be truly vexing. Our peptides target the novel CCN signaling pathway, leveraging the unique qualities of CCN3 to target the control of ECM changes in response to injury, blocking fibrosis and creating an environment for re-establishment of complete tissue homeostasis. By creating peptide libraries and conducting diverse in vitro and in vivo screens we have identified specific regions with the CCN3 protein responsible for blocking single, or multiple, pathways to fibroblast and cancer growth, and fibrosis formation. Unlike other products such as antibodies directed against the pro-fibrotic CCN2, that appear to partially block a single pathway, our novel therapy regulates vs. simply blocking, thus allowing reestablishment of the body's healthy equilibrium.

With several peptide products in the pipeline in preclinical development and a wide range of possible disease applications, how have you selected your lead indication target(s)?

BR: Having several products covering a wide variety of diseases is great and choosing lead indications to pursue is a wonderful challenge to have. In reality, the two lead indications that we have chosen to pursue have as much to do with the research that we have done with my team as it does the extreme health care need. My long background in academia, clinical nephrology/public health, and pharmaceutical drug R&D has driven the way we do drug discovery at BLR Bio. First, working to clearly understand the pathways to disease before selecting a target, and trying not to get caught up by targets in vogue. Then, testing our peptides in multiple related diseases before moving into the clinic. Our pipeline was created out of our long-term interest in fibrosis and the extracellular matrix in biology and disease, particularly in renal fibrosis as a complication of diabetes an area of great medical need. This has led to the selection of diabetic nephropathy as a main target for one of our lead products. Since our target, and the peptide inhibitor of our target, are involved in many other forms of fibrosis and other disease involving the miss-regulation of matricellular signaling, this creates other exciting therapeutic indications down the road. These include NASH, IPF, MD, and scleroderma to name a few.

I began my career in cancer research, and we know now that the extracellular matrix and matricellular signaling proteins such as the CCNs are critical also in the formation and resolution of tumors. The pro-fibrotic CCNs, CCN2 and CCN4 in particular have been shown to be important for the establishment and likely invasion and metastases in numerous tumors. The opportunity to use CCN3-based therapy to, not only halt the cancer, but also to target the microenvironment is an exciting one. Our company is particularly excited about the potential in pancreatic cancer. We have an established relationship with thought leaders in the cancer field and are moving forward with development work, partnering with some of these world experts. To add to the excitement of the therapies, our company also is developing a diagnostic/theranostic based on the same technology platform. It holds the promise of either stand-alone or companion diagnostics for development.

What are the key advantages of peptide therapeutics over small molecules, antibodies and other protein therapeutics?

BR: As you know, peptide-based drugs are one of the most rapidly growing areas in therapeutic development. More than 100 peptide-based drugs are on the market with several effective in cancer treatment. There is good reason for this. In size, falling between small molecule drugs and "biologics", they have many of the benefits of both while shedding many of the deficiencies. For example, because of their small size they gain many delivery benefits over biologics. First, better penetration of tissue at the site of action. This is crucial in fibrotic disease and cancer where there is a very strong barrier to penetration. Second, it allows for many more delivery options, including oral formulation. The earlier assumption of poor metabolic stability of peptides has just not proven to be the case. Although there is often rapid clearance from circulation, the previous assumptions about required PK by classic definition has also proven to be not relevant, since a specific and sustained biological activity at the target receptor is characteristic of many peptides. Unlike small molecules they are based on sequences that have evolved to react with specific receptors and often at very low concentrations. This results in very low off target effects. Last, since they can be chemically synthesized the ease and cost to manufacture are more nearly like that of small molecules. The majority of approved peptides are in the 15 amino acids (aa) range or smaller, and they treat a broad range of diseases.

How do you see academia and industry collaborating in the future to advance promising peptide therapeutics into commercially viable products?

BR: First, we think that academia will play a significant role in the creation of new peptide-based therapies in the future. Since the last decade has seen a decline in the number of newly approved drugs from large pharma, and recently there has been a strong trend for these same companies to dramatically reduce their discovery effort, this creates a large opportunity for novel peptide therapies and academic-initiated translational research. This does not necessarily mean that the new paradigm will be that large strategic companies will license early stage assets directly from Universities. Rather, we anticipate that the most promising discoveries will lead to the creation of start-up companies and the building of defendable IP. Then, large pharmaceuticals will court these start-ups as they progress to become true biotechnology companies with completion of phase I, phase II clinical trials or beyond. We think that the best of these emerging biotech companies will then expand their ability to do discovery and early stage development. They will also be able to clearly evaluate and in-license additional opportunities in academia. These are exactly the skills now lacking in big pharma. These successful discovery biotech's will become the future pipelines for drugs that will go to the mid and large pharma companies centered on late development and marketing. I envision BLR Bio as becoming one of these discovery companies able to contribute significantly to filling this gap.

Want to hear more from Dr. Riser? Join him this May in Long Beach, CA for the 18th annual TIDES - Save $300 when you register using the code B16180BLOG100. Click here to register. 

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Analytical Controls in an Evolving Technology and Regulatory Landscape

 
IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Rachel Orr, Senior Scientist, GlaxoSmithKline R&D, United Kingdom for an exclusive interview that delves into analytical controls in an evolving technology and regulatory landscape, the topic of her presentation at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA.


1. What key analytical challenges do oligonucleotide developers need to overcome in order to achieve regulatory approval?

Oligonucleotides are inherently difficult to analyze - they are neither traditional, small nor large molecules, and the techniques and well established methodologies for pharmaceutical analysis are all focused on these distinct classes. In addition, there is very limited regulatory guidance when it comes to oligonucleotides, so there are no set rules to follow. There are often discussions as to whether oligonucleotide analysis should follow small molecule rules and regulations or align with biopharmaceuticals; opinions are divided.

With limited experience across the industry in marketing applications it is difficult to assess what is needed in order to gain regulatory approval and this is one of the biggest challenges in CMC oligonucleotide development. There is no recent precedence of global oligonucleotide marketing applications and as such it is difficult to ascertain what may and may not be acceptable. Close engagement with the regulators is something which helps to form strategies and controls and hopefully this will increase the likelihood of success of regulatory approvals going forwards.


2. How have advancements in technology improved the analytical process? Can you provide examples?

Since the last successful oligonucleotide marketing application there have been considerable enhancements in technologies. UHPLC is now common throughout the industry and techniques such as high resolution NMR and mass spectrometry are much more affordable and quick. These advances in technology mean that the resolution and robustness of methodologies can be much improved, however has a downside that the regulators may well expect us to present them with these improvements by first intent.

Increased understanding of non "typical" molecules throughout the industry has enabled wider thinking and collaboration between different pharmaceutical companies, with more people working together on the problem progress will inevitably come quicker. As more people engage with the concept of oligos and as technology continues to evolve, I anticipate a large shift in the way we look to control oligonucleotides analytically in coming years.


3. What are the most common mistakes that oligonucleotide drug developers encounter during CMC submissions?

Again, due to there being so few oligonucleotides which have gone through the full CMC process I think it is difficult to make too many sweeping statements, however the key issues that I have come across all stem from attempting to fit oligonucleotides into a box. Whether it is the small molecule group or the biopharmaceutical groups who take on the challenge of oligonucleotides, it is generally only a few "experts" within that team who work on the molecules. It is impossible to fit existing small or large molecule platforms onto oligonucleotides and a lot of time, resources and money can be wasted trying to do so! Due to the small pool of people working on the oligo projects it can also be challenging when it comes to elements such as wider reviews of submissions as the reviewers are not always as well informed about the challenges associated with oligonucleotide analysis. Education of these reviewers and key stakeholders is an activity that cannot be underestimated in time or importance! This emphasizes the benefits of cross-pharmaceutical company collaborations.


Want to hear more from industry experts like Rachel? See our exclusive TIDES speaker Q&A series here.

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From Critical Quality Attribute Assessment to Specification and Characterization

"The reason for assessing the critical quality attributes is not only to fulfill an expectation, but also because it serves as a better understanding of the product under development"

IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Renata Varga, Ph.D., Scientist - Characterization, Analytical Sciences, Global Biological CMC, Teva Pharmaceutical for an exclusive interview that delves into the topics he plans to discuss at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA.

In this interview, Dr. Varga discusses;

• The key regulatory challenges facing peptide developers
• What regulators look for when establishing CQAs in genetics, and any variation between generics and novel therapeutics.
• Peptide innovation in the next 5 years

From Critical Quality Attribute Assessment to Specification and Characterization
Characterization and comparability assessment of peptide based generic products is driven by critical quality attributes (CQAs). Standard CQAs are identified based on regulatory requirements. Additional CQAs are determined by understanding the molecule itself: sensitive amino acids or which are involved in receptor-binding or mechanism of activation, are identified and based on uncertainty and impact the criticality of that attribute is defined. All standard- and additional CQAs are included in the specification while potential CQAs are further characterized by orthogonal techniques.

Access the complete interview

Register for TIDES now and save $500 - to save, use the code B16180BLOG100

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Efficient Chemoenzymatic Peptide Synthesis (CEPS) in Water

"When peptides are ligated in water, the crude reaction mixture can immediately be loaded on the preparative HPLC column to purify the product" 

IBC's 18th Annual TIDES is the industry's #1 forum for oligonucleotide and peptide leaders to build successful partnerships and accelerate products from early discovery through late-stage development and commercialization. The TIDES team recently talked to Timo Nuijens, Ph.D., Lead Scientist, EnzyPep BV, The Netherlands for an exclusive interview that delves into the topics he plans to discuss at the upcoming Oligonucleotide and Peptide Therapeutics event in Long Beach, CA.

Efficient Chemoenzymatic Peptide Synthesis (CEPS) in Water
We describe a novel, organic cosolvent-stable peptide ligase (peptiligase), which couples peptide fragments with exceptional efficiency in water. Because peptiligase does not need a particular recognition motif, this novel technology is traceless and very broadly applicable. Besides fragment condensation, the enzyme is able to efficiently catalyze head-to-tail cyclization of peptides to create macrocyclics. It has also been shown to couple peptide esters efficiently to proteins. Peptiligase could be a useful tool for the (industrial) synthesis of numerous (pharmaceutical) peptides and conjugates.

In this interview, Dr. Nuijens also discusses;

• The importance of fragment condensation of peptides in water
• What the future holds for the Netherlands in the global peptide drug development market
• The factors to consider when determining the optimal synthesis strategy when screening an enzyme

Access the complete interview

Register for TIDES now and save $500 - To save, use the code B16180BLOG100

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Impurity Guidelines to Oligonucleotides | An Interview with Andrew Teasdale of AstraZeneca

In a recent interview with TIDES, Andrew Teasdale, AZ Impurities Advisory Group Chair, AstraZeneca, United Kingdom examines the scope and applicability of both new guidance (ICH Q3D and ICH M7) and existing guidance (ICH Q3A / Q3B) to Oligonucleotides, looking into practical challenges and how they might be addressed and the potential application of generic, platform risk assessment approaches.


When discussing the impurities that face oligonucleotide developers, Andrew said, "The most common impurities in oligonucleotides are still related substances (oligomeric impurities)". However, this still begs to question what does one need to consider when mitigating risk for these impurities? Access the complete interview now.

Access the complete interview

To learn more from Andrew about oligonucleotide impurities, join him at IBC's 18th Annual TIDES event this May in Long Beach, CA where he will be speaking in his session titled "Assessment of the Relationship of Impurity Guidelines to Oligonucleotides - Opportunities for Rationale Approaches to Management of Risk". Download the brochure to access the complete agenda and speaking faculty.



Register for TIDES now and save $500 - To activate your savings, use the code B16180BLOG100

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Are You Keeping Up with Your Competition at TIDES?

Over 70 leading and emerging oligonucleotide and peptide companies will be Exhibiting, providing cutting-edge Spotlight Presentations and/or Sponsoring networking sessions at TIDES 2015, coming to San Diego this May. What do they know?

They know:

·         TIDES is the largest gathering of the oligonucleotide and peptide community where networking and business gets done. Over 50% of the attendees are President’s, VPs, C-Level, Directors and Managers that hold the true buying decision power in their hands. Meeting face-to-face with these decision makers will accelerate business, partnerships and sales.

·         Whether in the Exhibit Hall, between sessions or during the scheduled Networking Receptions, you have direct access to hundreds of leading minds and businesses.

TIDES is the leading source of advances in technology, strategies and case studies that span from R&D through Commercialization. Your company needs to be an important part of this community and conversation.

Why let your competition have all the glory?

·         Secure one of the remaining 10 exhibit spaces left >> Request an updated floor plan

·         Sponsor a Spotlight Presentation and prove that your company is a thought-leader in the industry. We have a few select spots remaining in the agenda. Contact me for subject and topic areas.

·         Brand your company through sponsorship of the TIDES Lounge or other branding opportunities.

·         Capitalize on this unique opportunity to highlight your company's expertise to 800+ qualified attendees from 25+ countries.   As our valued event sponsor/exhibitor, your company's brand awareness extends beyond our attendees to our thousands of targeted email subscribers, website visitors and event prospects.   Give me a call or shoot me an email today to discuss how your participation at this year's TIDES meeting will help you achieve your company's targeted growth goals.

Join us in San Diego next month. Register for TIDES today: http://bit.ly/1OBPwgA

Best,
The TIDES Team

Futurebiopharma.blogspot.com

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See who is attending TIDES

Will you be joining these confirmed colleagues at IBC’s 16th Annual TIDES meeting, taking place May 12-15 2014 in Providence, Rhode Island?

Drive your discovery and manufacturing programs forward at TIDES by hearing a world-class group of speakers provide you with the latest science, technology and development updates.

Benchmark your current portfolio by hearing new reports on leading therapeutic candidates in development from Amgen, GSK, Teva, Alnylam, Ophthotech, AstraZeneca and Giuliani SpA. Also, ensure regulatory success by hearing FDA speaker Jeffrey Baker provide insight on how to prepare for the FDA Safety and Innovation Act.

Plus, be the first to access novel technologies and cutting-edge science in our poster and exhibit hall, featuring over 75 exhibitors and 30 peer-submitted posters.

Keynote Speakers Provide Cutting-Edge Science to Accelerate Your Current Project

• Peptide R&D: Advances and Opportunities Nader Fotouhi, Ph.D., Vice President, Head of Discovery Technologies, Hoffmann-La Roche

• Pharmaceutical Protein and Peptide Engineering Lars F. Iversen, Ph.D., Corporate Vice President, Diabetes Protein Engineering, Novo Nordisk, Denmark

• Aptamer Nanotechnology for Therapeutic Nanoparticles Omid Farokhzad, M.D., Associate Professor; Director, Laboratory of Nanomedicine and Biomaterials, Brigham and Women's Hospital, Harvard Medical School

• Peptide-Based Vaccines in the New Era of Cancer Immunotherapy Eric von Hofe, Ph.D., President, Antigen Express, Inc.

• PeptiDream: From Bench to IPO Patrick C. Reid, Ph.D., Chief Scientific Officer, PeptiDream Inc., Japan and U.S.A.

To learn more, download our brochure.

Register with code XB14180BLOG to receive 20% off of the standard rate. If you have questions about the event, feel free to contact our group manager, Kate Devery (Kdevery@iirusa.com), or visit our webpage. We look forward to seeing you May 12-15 in Providence, RI!

Cheers,
The TIDES Team

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See who is attending TIDES 2014

Accelerate your oligo and peptide discovery, development and manufacturing efforts at IBC's 16th Annual TIDES meeting, taking place May 12-15, 2014 in Providence, RI. With four concurrent tracks offering 100 speaker presentations TIDES is the premier event to drive your business forward by helping you understand evolving technologies and regulatory expectations.

Why Do Attendees Return to TIDES Year After Year?

• Improve your manufacturing efforts by hearing how industry leaders are implementing new process validation guidelines and overcoming formulation challenges

• Meet with all of the key players in the industry during dedicated networking breaks, luncheons and cocktail receptions - we will also be rolling out a new partnering tool which allows you to view the attendee list and pre-schedule onsite meetings

• Benchmark your CMC portfolio against industry leaders by hearing case study updates on what they are doing to ensure regulatory success

• Discover new targets and discovery strategies for both oligonucleotides and peptides

• Design effective discovery and clinical studies by gaining updates on the latest preclinical and clinical oligo and peptide projects under development

• Understand evolving technologies and network with major vendors and service providers in the industry in our exhibit and poster hall

• Overcome regulatory expectations and improve your changes for clinical success by attending one of four interactive full-day workshops

To learn more, download our brochure.

Register with code XB14180BLOG to receive 20% off of the standard rate. If you have questions about the event, feel free to contact our group manager, Kate Devery (Kdevery@iirusa.com), or visit our webpage.

We look forward to seeing you May 12-15 in Providence, RI!

Cheers,
The TIDES Team

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Follow us on Twitter
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Introducing TIDES: Oligonucleotide and Peptide® Therapeutics

Over the last 15 years, the TIDES Summit has cemented its status as the community’s premier meeting place for the oligonucleotide and peptide discovery, development and manufacturing industries.

Join 750 of professionals at this must attend oligonucleotide and peptide discovery, development and manufacturing event. This is your chance to get the information and contacts you need to overcome the toughest challenges you are facing and move your research, development and process chemistry.

View the full conference line-up. Download the agenda today!

Tides will take place May 12-15, 2013 in Boston.  If you'd like to join us, as a reader of this blog, you receive 20% off the standard rate when you register to join us and mention code TIDES13JP.  If you have any questions, feel free to email Jennifer Pereira.

Keynote Presentations Include:

• MIPOMERSEN CLINICAL ADVANCES
• ˃Presenter: Mary P. McGowan, M.D., Senior Medical Director, Clinical Research, Genzyme, A Sanofi Company
• EPITOPE TARGETING PCC AGENTS
• ˃Presenter: James R. Heath, Ph.D., Professor, Chemistry and Chemical Engineering, California Institute Of Technology
• SPHERICAL NUCLEIC ACIDS (SNAS)
• ˃Presenter: Chad A. Mirkin, Ph.D., Professor, Chemistry, Northwestern University
• VC FUNDING FOR OLIGOS AND PEPTIDES
• ˃Presenter: Bruce Booth, D.Phil., Partner, Life Sciences, Atlas Venture
• DELIVERY AND DEVICE STRATEGIES FOR PEPTIDES
• ˃Presenter: William J. Lambert, Ph.D., Fellow, Drug Delivery and Device Development, MedImmune, Inc.

Hear Comprehensive Updates Spanning the Entire Development Process:

• OLIGONUCLEOTIDE MANUFACTURING, TECHNOLOGY AND PRODUCT DEVELOPMENT
• • Hear first-hand reviews from regulatory and industry thought leaders on quality and purity specifications to answer your pressing questions about impurities specifications
• • Implement continued process verification with latest perspective of industry consortium
• • Modernize your process science with cutting-edge reports in the new process chemistry session
• PEPTIDE MANUFACTURING, TECHNOLOGY AND PRODUCT DEVELOPMENT
• • FDA perspective and scale up report on peptide vaccines
• • Plan for the future of novel synthesis technologies with new data from Lonza, Bachem and EMD
• • Learn how to assess and prevent aggregation to avoid late stage problems that can hold up product release
• OLIGONUCLEOTIDE THERAPEUTICS DISCOVERY
• • Discover new developments in single stranded RNAs, non-coding RNAs and non-hepatic cell and tissue targeting
• • Find out what it really takes to bring an oligonucleotide drug to market and learn about the phase-specific strategies you should be implementing
• • Explore emerging solutions to the oligonucleotide delivery challenge and hear the latest preclinical and clinical data to inform your own oligonucleotide therapeutic projects
• PEPTIDE DISCOVERY AND DEVELOPMENT
• • Discover the latest advances in peptide-like scaffolds and structures and peptides as protein-protein interaction modulators to accelerate your discovery efforts
• • Hear new preclinical and clinical data from multiple peptide drugs in development to provide you lessons for your own projects
• • Find novel synthetic approaches to modify peptides and strategies for intracellular peptide delivery to improve the drug-like properties of your molecules
• NUCLEIC ACIDS TECHNOLOGIES FOR MOLECULAR DIAGNOSTICS
• • Learn new technologies for sample prep and for the diagnostics of the future
• • Ensure supply chain for labels and modifications to avoid interruptions in distribution

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