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CPTR 4th Workshop 2013 Report Day 2

October 3, 2013

Day Two opened with a drug development roundtable that provided participants with updates and future plans for various TB drugs and regimens in the development pipeline, followed by a Q&A session with presenters. Participants included representatives from AstraZeneca (Scott Butler), Bayer (Janet Herrington), Janssen/J&J (Myriam Haxaire-Theeuwes), Otsuka (Jeffrey Hafkin), Sanofi (Isabelle Cieren-Puiseux), Sequella (Carol Nacy) and TB Alliance (Dan Everitt).  The second panel discussion of the morning showcased various modeling efforts that may help to improve the knowledge base and selection of novel regimens for development. This panel discussion was led by David Hermann (BMGF) and included presentations from Tawanda Gumbo (UT-Southwestern), Peter Vis (LP&P), Rada Savic (Uppsala University), and Klaus Romero (C-Path).

Dr. Dalene von Delft gave the closing remarks, sharing her personal experience as a TB patient who was able to access Sirturo (bedaquiline) against MDR-TB. Dr von Delft has been working since that time to expand access to new TB therapies. Jan Gheuns wrapped up the workshop by sharing his personal perspectives on CPTR’s progress since its first workshop in 2009.

TB Drug Co-Development Roundtable

The Co-Development Roundtable provided the opportunity for drug developers to share status updates and field questions about new drugs or regimens in the pipeline. It was followed by a Q&A session with the audience that covered a variety of topics including drug pricing, the regimen development pathway, and challenges and perceived gaps in the drug development arena.

Moderator: Carl Mendel, Senior VP Research and Development, TB Alliance

Scott Butler, Infection Innovative Medicines Group, AstraZeneca R&D, gave an update about Phase 2 trials for AZD5847: an oxazolidinone for the treatment of TB. See Scott Butler’s presentation and video.

Janet Herrington, Director of Global Regulatory Affairs at Bayer, presented a status report from Bayer and TB Alliance’s REMoxTB study, intended to demonstrate that moxifloxacin shows potential to shorten treatment duration for TB to 4 months as part of  an optimized combination regimen. See Janet Herrington’s presentation and video.

Myriam Haxaire, Compound Development Team Leader, Janssen Global Public Health, gave an overview of current status and use of bedaquiline as a treatment for MDR-TB, as well as an update on the Phase 3 trial and goals moving forward. SeeMyriam Haxaire’s presentation and video.

Jeffrey Hafkin, Associate Director, Otsuka Novel Products – TB, discussed the clinical development process for delaminid, sharing the status of trials and an update on regulatory progress for the drug. He also shared information on DDI studies and addressed a query about the prospects for use of delaminid as part of a novel combination. See Jeffrey Hafkin’s presentation and video.

Isabelle Cieren-Pusiseux, Sanofi, provided an update on Sanofi’s clinical activities for latent and active TB, an update on industrial activities, and an overview of the Sanofi portfolio. See Isabelle Cieren-Pusiseux’s presentation presentation and video.

Carol Nacy, CEO, Sequella, shared an overview of the Sequella TB portfolio, including SQ109 and sutezolid. She was then joined by Bob Wallis, who provided details about delamanid and rifabutin  combinations currently being examined that could benefit the MDR TB population. See Carol Nacy’s presentation and video. See Bob Wallis’ presentation and video.

Daniel Everitt, MD, Senior Medical Officer, TB Alliance, gave an overview of the most critical components for TB drug development in benefiting TB patients around the world. He presented the TB Alliance’s approach to novel regimen development – including the ideal product/regimen profile – and provided examples of two-week and two-month regimen studies. See Daniel Everitt’s presentation and video.

See the TB Drug Co-Development Roundtable Q&A video.

Quantitative Tools for TB Combination Regimens Development

This session provided an opportunity for learning and discussion on various modeling work being undertaken to develop better forecasting tools for TB drug and drug regimen development, particularly in the area of clinical trials.

David Hermann, VP & Lead Scientist for Great Lakes Drug Development / Certara and Consultant to Quantitative Sciences, Bill & Melinda Gates Foundation, shared an overview of CPTR’s expanded role in developing models and tools that can help scientists and drug developers “connect the dots” between early- and late-stage clinical trials data. See David Hermann’s presentation and video.

Tawanda Gumbo, Administrative Director of Research Programs in Global Health, University of Texas Southwestern Medical Center, shared findings from a recent CPTR initiative to evaluate the accuracy of the hollow fiber model in forecasting therapeutic concepts, hypotheses and parameters for TB. See Tawanda Gumbo’s presentation and video.

Rada Savic, PhD, Assistant Professor, UC San Francisco, provided results from a modeling and clinical trial simulation intended to provide guidance for Phase 3 trials for new TB regimens. See Rada Savic’s presentation and video.

Peter Vis, LAP&P Consultants, reviewed various modeling techniques and examined gaps with applying current approaches to TB Clinical Trials Simulation (CTS). He also provided guiding criteria for arriving at clinically relevant PK-PD models for TB CTS. See Peter Vis’ presentation and video.

Klaus Romero, Director of Clinical Pharmacology, Critical Path Institute provided an update on the proposed expanded CPTR Modeling and Simulation Workgroup’s approach and goals. He then shared learnings from a successful model developed to help optimize clinical trial design for mild and moderate Alzheimer’s Disease, and described how that experience might be relevant to a TB CTS model. See Klaus Romero’s presentation and video.

A discussion examining the opportunities and challenges of modeling for the future of regimen identification and development – including various approaches and knowledge gaps – followed the presentations.

See the Quantitative Tools for TB Combination Regimens Development Q&A video.

Closing Remarks

In the final workshop session, Dr. Dalene von Delft, TB Proof, shared her personal story as a TB patient, including the fact that she took a treatment course that included bedaquiline, which may have saved her life, to emphasize the critical need to develop new drugs and regimens. She urged participants to accelerate momentum by designing innovative models for new drugs, conducting fast-track efficacy testing through novel regimens, being creative in partnerships with National Departments of Health and NGO’s, and involving patients and communities. See Dalene von Delft’s presentation and video.

Workshop Wrap-Up

Jan Ghuens, Bill & Melinda Gates Foundation, closed the workshop by thanking organizers, speakers and attendees. He commended the group for the progress made in the field over the last three years, and challenged them to continue to move forwards towards the goal of new drugs and regimens for patients suffering from TB. See Jan Ghuens’ video.


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