Boston Children's Hospital to invest drug patent earnings back into pediatric medical research

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Kristen Dattoli, Kristen.dattoli@childrens.harvard.edu, +1 617-919-3110

(BOSTON) ­— Today, Boston Children’s Hospital announced that it has received proceeds from the sale of royalties related to Vonvendi, a drug developed at Boston Children’s and marketed by Shire Pharmaceuticals, in a $51.5 million transaction with Barings Alternative Investments, part of Barings LLC, on behalf of institutional investors. Boston Children’s portion of proceeds from the transaction will be directed back into its charitable mission including its pediatric medical research pipeline.

Vonvendi, a recombinant drug used to treat a common bleeding disorder called von Willebrand disease, is based on patented technology developed by researchers at Boston Children’s, hematologists Stuart Orkin, MD, and David Ginsburg, MD. Vonvendi was brought to market through licensing agreements between the Boston Children’s Technology Innovation and Development Office (TIDO) and Baxalta, which merged with Shire in 2016.

“The success of Vonvendi reflects the tremendous impact of Boston Children’s research on patient health and exemplifies how a partnership with industry can expand the reach of treatments developed here to benefit the broadest set of patients,” says Irene Abrams, Vice President of Technology and New Ventures at Boston Children’s TIDO, which builds partnerships with life sciences industry to accelerate the translation of academic research to life-saving products.

“Scientific research has always been a cornerstone of Boston Children’s and so it’s quite fitting that these proceeds, stemming from a drug that is already improving the wellbeing of patients today, will go toward discovery of future therapeutics for a wide range of children’s diseases that our scientists assiduously investigate,” says Sandra L. Fenwick, President and Chief Executive Officer of Boston Children’s.

The research enterprise at Boston Children’s, the largest pediatric medical center of its kind in the world, comprises more than 3,000 scientists who conduct research across all scopes of the basic, translational and clinical research funnel. Their efforts are advancing the prevention, diagnosis and treatment of childhood and, in some cases, adult disease.

“The investment return, so-to-speak, provides new funding to accelerate the translation of emerging therapies from the laboratory bench to our patients, where it can make real impact on children and their families,” says David A. Williams, Senior Vice President and Chief Scientific Officer of Boston Children’s and President of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “It’s extremely rewarding to see Stu and David’s research continue to contribute to pediatric medical research in such a substantial and meaningful way. It is also important to note that many adults with von Willebrand’s Disease will also benefit from this new therapy.”

At Boston Children’s, Orkin and Ginsburg first cloned the gene for von Willebrand factor, an essential blood clotting protein, and filed a patent for their recombinant form of it in 1985. In 2015, the drug Vonvendi — based on Orkin and Ginsburg’s discovery — was approved after the required safety testing by the FDA. Today, patients with von Willebrand disease who are prescribed Vonvendi receive blood transfusions containing the recombinant product to help compensate for their body’s deficient production of natural von Willebrand clotting factor. Over the years, beyond the technology behind Vonvendi, Orkin and Ginsburg’s research has made huge strides in our understanding of von Willebrand disease and its genetic and molecular underpinnings.

“All the hard work is made worthwhile by the knowledge that our efforts are having a positive outcome on patients,” says Orkin, who is currently a Howard Hughes Medical Institute (HHMI) Investigator at Boston Children’s and the David G. Nathan Distinguished Professor of Pediatrics at Harvard Medical School. He was formerly Chairman of Pediatric Oncology at Dana-Farber Cancer Institute.

Ginsburg, who continues to study von Willebrand disease, is currently an HHMI investigator and the James V. Neel Distinguished Professor at the University of Michigan Life Sciences Institute.

MTS Health Partners acted as exclusive financial advisor and Covington & Burling LLP acted as special transactional counsel to Boston Children’s during the transaction with Barings.

About Boston Children’s Hospital

Boston Children’s Hospitalthe primary pediatric teaching affiliate of Harvard Medical School, is home to the world’s largest research enterprise based at a pediatric medical center. Its discoveries have benefited both children and adults since 1869. Today, more than 3,000 scientists, including 10 members of the National Academy of Sciences, 18 members of the National Academy of Medicine and 12 Howard Hughes Medical Investigators comprise Boston Children’s research community. Founded as a 20-bed hospital for children, Boston Children’s is now a 415-bed comprehensive center for pediatric and adolescent health care. For more, visit our Vector and Thriving blogs and follow us on social media @BostonChildrens@BCH_InnovationFacebook and YouTube.

About Barings Alternative Investments

Barings Alternative Investments (BAI), part of Barings LLC, is a 450+ associate team located across 11 countries that manages $52.3 billion* in client capital. BAI seeks differentiated sources of returns by incorporating decades of investment experience in alternative assets offering investors access to a diverse range of opportunities across private equity, real assets, asset-based investments and the four quadrants of real estate. We serve as a trusted partner to clients, leveraging our global presence and robust origination capabilities to identify the most attractive risk-adjusted return opportunities. The Barings Private Equity and Real Assets team focuses on control positions in actively-managed, thematically-driven asset-based investments that generate both current income and opportunity for capital appreciation. Led by a research intensive investment process that identifies long-term investment trends, the team invests across eight specialist sectors in four asset classes: Capital Assets, Infrastructure, Intangible Assets and Natural Resources.

About Barings

Barings is a $305+ billion* global financial services firm dedicated to meeting the evolving investment and capital needs of our clients. We build lasting partnerships that leverage our distinctive expertise across traditional and alternative asset classes to deliver innovative solutions and exceptional service. Part of MassMutual, Barings maintains a strong global presence with over 1,800 professionals and offices in 16 countries. Learn more at www.barings.com.

*As of March 31, 2018