September 2015 Spotlight

SARTORIUS AG (WWW.SARTORIUS.COM)

Sartorius Opens New Applications Center

In June 2015, Sartorius opened an application center at its North American headquarters in Bohemia, NY. Among the guests were representatives from local government, customers and business partners, and industry media including BPI’s publisher, Brian Caine. The New York Sartorius Application Center provides customers with access to multiple single-use systems for upstream and downstream processing. Demonstration laboratories present equipment by application and to show how individual systems work together for complete process solutions.

This 40,000-ft² facility was designed to cultivate innovation and enhance customer experience in four areas:

Mary Lavin, president of the Sartorius companies in North America, acknowledged the strategic importance of opening this center: “With this new resource, we can offer customers and employees the opportunity to see and use many of our innovative products. Our US-based R&D team is close to an important market.”

In 1988, Sartorius North America had 40 employees and generated US$20 million in revenue. By 2015, it has >800 employees and will generate >$350 million.

BEBPA: 23–25 September 2015 in Budapest

BPI advisor emeritus Laureen Little is founder of the Biopharmaceutical Emerging Best Practices Association (BEBPA) and a chair of its eighth annual meeting this month in Budapest, Hungary. In a recent interview with the organizers, she discussed the upcoming event and industry challenges related to bioassay development.

“With more commercial providers entering the market of ready-to-use commercial kits, I think more companies will find themselves trying to determine how to assess the usefulness of these kits for their products. I have come across several cases where kits that appear to work well for some products are too sensitive to small changes in structure for others. Or the putative mechanism of action isn’t really ‘typical’ for a given product even though at first it seems that it should be appropriate. Availability of these kits may have given us a false sense of the amount of time and resources we need to invest in developing methods using them.”

Interactive surveys have been popular at BEBPA meetings. In Barcelona last year, attendees were surveyed on the trends in validating bioassays. “One thing I am asked about all the time is how many product lots are used,” said Little. “Traditionally, validation is considered to be only about the method, so most companies were using only a single lot for each type of product tested (e.g., one drug substance and one drug product if the method was used to release both). But lately, more companies are asked to include at least two lots; now about half report using more than one lot.”

Two preconference workshops will cover bioassay basics and monotoring programs. The latter workshop starts with a tutorial about the use of tolerance intervals, confidence intervals, and how to set limits with small data sets. It will end with some nonideal case studies about how to approach nonnormalized data sets and skewed data. Problems can arise, Little explained, from simply applying a log transformation to establish limits.

Look for a summary position paper emerging from this important bioassay conference in an upcoming issue of BioProcess International.

North American Drug Market Shifts Focus

Cost-containment measures are shifting the decision- making focus of North America’s pharmaceutical market from prescribers to payers, says research and consulting firm GlobalData. Its June report, PharmaSphere: North American Market Access Strategy, states that pricing and reimbursement hurdles are particularly challenging there.

Industry analyst Adam Dion says that overall health- care spending slowed during the recession. New drugs and medical technologies have received increased attention for potential cost savings. “The US population is largely covered through private health insurance, but public-sector coverage is further complicated by third- party administrators, dispensers, and others. Canada’s single-payer system has sweeping cost-containment measures and stricter pricing and reimbursement.” As market-access regulations become more stringent, he explains, product commercialization strategies require launching in countries with good domestic demand, pricing, and reimbursement conditions. Efforts continue toward lowering prescription drugs spending (~10% of the US healthcare budget), but the number of drugs that cost >$100,000/year has increased over the past five years. The rise in premium-priced drugs has been most notable in oncology and infectious disease segments.

Rare-Disease Awareness

by Cheryl Scott

In May, the Rare Genomics Institute (RGI, www. raregenomics.org) announced its new Amplify Hope Initiative, a study of how crowd-funding can promote scientific research to help rare-disease patients. RGI is an international nonprofit organization that provides patients with access to state-of-the-art genomics sequencing technology, connections to scientists and researchers, and help in paying for services through grants, insurance, and crowd-funding.

“Crowd-funding not only will help raise much-needed funds to uncover the cause of a child’s illness,” said RGI president Jimmy Lin. “It also can educate an entire community about this latest revolution in medicine.”

Rare Genomics invites patients, families, friends, and industry partners to support this initiative. Thanks to a grant from the Templeton Foundation, the project will measure effectiveness, scientific impact, and community engagement of different crowd-funding strategies. Scientific partners include Ambry Genetics and Baylor Miraca Genetics Laboratories. Crowd-funding partners include CrowdRise, Indiegogo Life, and YouCaring. Communications software is provided by xTeros.

Patients will be led through different strategies and coached through different methods of crowd-funding as the study tracks funds raised and other parameters. Anyone can donate and share these campaigns through social media to spread the word about genetic sequencing and help families affected by rare ailments. Participants and donors will be asked to complete surveys that enable RG to create additional resources, support, and education tailored to rare-disease patients and caregivers. Find more information about the Amplify Hope Initiative online at http://amplifyhope. raregenomics.org.

RGI also offers both printed and electronic editions of its rare-disease guide, Rare Diseases: Diagnosis, Therapies and Hope. Covering scientific developments in genomics and advanced treatments alongside inspiring stories of families overcoming the incredible odds, the e-book is free, and print copies can be ordered from Amazon.com.

“We are part of a growing movement to raise global awareness of rare diseases,” said Lin’s coauthor, Ana Sanfilippo. “It’s been a privilege to work with so many experts to share their deep insights for rare-disease parents. And it’s been rewarding to work with the courageous parents who have opened their homes and shared their stories to help other families.”

Meanwhile, BioXcel Corporation (a provider of cloud-based “big data” solutions) and Takeda Development Center Americas (a medical research company) are partnering to repurpose assets for orphan diseases. In April, they announced a deal providing Takeda with BioXcel’s integrated product-discovery engine and PharmGPS orphan-disease software suite for drug-discovery and repurposing.

BioXcel’s chair and CEO Vimal Mehta said that “Discovering novel applications for existing molecules is an increasingly attractive strategy to address patient needs while capitalizing on previous investments and derisking clinical development. Our Big Data Innovation Lab facilitates the repurposing of the most attractive candidates applicable to a given disease.” His company’s proprietary suite covers more than 9,000 rare and ultrarare diseases, with up to 5,000 associated genes, 1,500 disease pathways, and distinct target- indication tiles for antibody, protein, RNA, small-molecule, and gene- therapy modalities.

CORRECTION: A June 2015 Article Reference

In BPI’s June 2015 issue, “Optimization and Scale-Up of HCIC-Based MAb Purification Processes, Part 2” by Stephanie Chaudoreille, Natalie Bohrer, Patrick Santambien, Warren Schwartz, and Steven P. Allen, a reference was incorrectly cited on page 41:

“Although this work focused on resolution of antibody from less- strongly retained impurities, HCIC control based on both pH and the presence of a binding-promoting salt also has been used to facilitate resolution of monomeric antibody from more strongly retained impurities: antibody aggregates (5).”

The cited reference should be (1) Chen J, et al. The Distinctive Separation Attributes of Mixed- Mode Resins and Their Application in Monoclonal Antibody Downstream Purification Process. J. Chromatogr. A 1217(2) 2010: 216–224. We apologize for any confusion. The article has been corrected in our online archives.