January 29, 2018
The Senate voted 55 to 43 Jan. 24 to confirm former pharmaceutical industry executive Alex Azar as HHS secretary. Support for Azar, who previously served as the department’s deputy secretary during the George W. Bush administration, was divided, with critics voicing concerns over his time as president of pharmaceutical giant Eli Lilly’s U.S. division. He also served as a member of BIO’s board of supervisors.
FDA Guidance on Hypertension Combinations Offers Alternatives to Larger Trials
Sponsors can prove the effectiveness of combination treatments for hypertension by testing the highest-approved dosage, rather than designing larger trials that examine multiple doses and components, according to a new FDA draft guidance for product development. Alternatives for demonstrating the components’ independent contributions include: comparing the effect of the combination to the separate components at their highest-approved doses, or by comparing them at the highest doses planned for the fixed combination. While the guidance assumes two components, the FDA said it can be applied to combinations of three or more. Sponsors should select previously approved drugs that have mechanisms of action distinct enough to demonstrate the combination’s effects. Phase III clinical studies should involve populations where initiating therapy with two drugs is appropriate, with primary endpoints possibly including systolic, diastolic or average blood pressure, the agency said. Read the full draft guidance here: https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm593825.pdf.
Celgene Acquires CAR-T Developer Juno Therapeutics
Celgene acquired Juno Therapeutics for approximately $9 billion. Juno was an early leader in the development of CAR-T and T cell receptor therapeutics, and holds a broad portfolio in multiple targets and cancer indications. Juno’s JCAR017 (lisocabtagene maraleucel; liso-cel), a CD19-directed CAR-T therapy currently being studied in relapsed or refractory diffuse large B-cell lymphoma, will be added to Celgene’s lymphoma program. The companies said they expect regulatory approval in in 2019 with $3 billion in potential global sales. JCARH125, meanwhile will support Celgene’s work against BCMA (B-cell maturation antigen), a key target in multiple myeloma. In collaboration with Juno’s team in Seattle, Celgene plans to expand its existing center of excellence for immuno-oncology translational medicine by leveraging Juno’s research and development facility in Seattle as well as Juno’s manufacturing facility in Bothell, Wash.
China Issues Guidelines for Submitting Foreign Medical Device Trial Data
The Chinese Food and Drug Administration (CFDA) released technical guidelines for accepting clinical data for trials of medical devices conducted overseas, in a move to help speed approvals — with sponsors no longer having to conduct an additional trial within the country before moving forward. The clinical trial registration guidelines largely cover good clinical practices for devices and in vitro diagnostics, as well as ethical and legal principles. They also clarify data and technical requirements. In addition, sponsors will need to confirm that data can be extrapolated to China’s population. Any differences in GCPs should be documented and explained, CFDA said. Clinical trials conducted abroad may not always conform with China’s requirements, the agency warned, specifically when it comes to data integrity, clinical design and protection of research subjects. Applicants also need to fully describe study endpoints, calculation methods and study design considerations.
UPenn-Based Tmunity Therapeutics Raises $100 Million in Series A Financing
Tmunity Therapeutics announced the closing of $100 million in global Series A financing from Ping An Ventures, the Parker Institute for Cancer Immunotherapy, Gilead Sciences, Be The Match BioTherapies, the University of Pennsylvania, Lilly Asia Ventures and others. Tmunity, focused on developing next-generation T cell immunotherapies, plans to use the proceeds to advance its product portfolio, first in the treatment of cancer. The company was founded on an exclusive collaboration and license agreement with the University of Pennsylvania. “Tmunity is unlike any other cell-based immunotherapy start-up because of the unrivaled expertise of its scientific founders and leadership team in cell and gene therapy and its proven translational and manufacturing success in T cell medicine,” said Jiang Zhang, managing partner of Ping An Ventures. “We were also attracted to the global potential of the pipeline, especially the T cell therapies in oncology in China, as well as the scope beyond oncology into autoimmune and infectious diseases, as we begin to expand our investment portfolio.”
—by Conor Hale @conorhale