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CPTR 2017 Workshop: Summaries, Slides, and Videos — Day 2

Welcoming Remarks

Mel Spigelman (TB Alliance)

Mel Spigelman opened the second day of the workshop by introducing the day’s agenda and drawing attention to the personal stories of TB patients, reinforcing the driving force behind CPTR and all TB research and development efforts.

Video

 

Driving Progress Through Regulatory Innovation – Learnings from Oncology and Translatability to TB Regimen Development

Session Intro

Martha Brumfield (Critical Path Institute)

Video

 

Roundtable

Moderators:

Interview and Q&A with:

This session focused on extrapolating regulatory learnings from other fields for use in TB. Richard Pazdur discussed the evolution of oncology drugs and the explosion of new drugs and how drug development strategies popular in oncology may relate to TB. Martha Brumfield identified the development of biomarkers a gamechanger for oncology research and treatment, noting similar advances could do the same for TB. Pazdur noted a lack of coordination in biomarker development in oncology – this is something CPTR is helping address in TB. He remarked that oncology represents both the best and worst in pharma – great progress and innovation, but lack of coordination because competition trumps cooperation. Edward Cox emphasized that this is a potential strength of CPTR, as cooperation can enable the limited resources for TB research to be used most effectively and efficiently.

Video (Richard Pazdur)

Video (Edward Cox)

Video (Question and Answer)

 

TB Patient Perspectives

Session Intro

Erica Lessem (Treatment Action Group)

Video

 

Roundtable

Moderators:

Interview and Q&A:

Phumeza Tisile (TB Proof)

Phumeza Tisile shared her harrowing story being treated for TB, MDR-TB, pre-XDR-TB, and finally XDR-TB. Her treatment lasted almost four years total. Tisile was taking 20 – 30 pills daily, plus injections for a substantial length of time. She lost her hearing while sick, but was ultimately able to regain it with cochlear implants years later. Tisile was ultimately cured with the help of an unapproved TB drug – linezolid – which is now undergoing additional development. Tisile has since dedicated herself to advocating for better treatments for TB, more investment, and greater inclusion of the patient perspective in the research process. She delivered a strong message about ensuring new treatments reach those in need – asking what good are new and improved therapies if those suffering can’t afford them?

Video

 

Steve Bradley (TB Alert)

Steve Bradley shared his story, which included lengthy delay in getting accurately diagnosed with TB and terrible side effects of TB therapy. Bradley lost his sight and experienced permanent reduced sensitivity in his legs due to complications with first line treatment. Bradley spoke of the devastating effects of loss of independence and ability to work, as well as the terrible neglect of TB and stigma associated with it. He continues to share his story to raise awareness for TB and research, and remains optimistic that breakthroughs to improve therapy may be on the horizon.

Video

 

Question and Answer video

 

 

TB Drug Co-Development Roundtable

Moderator: Carl Mendel (TB Alliance)

 

Session Introduction

Carl Mendel (TB Alliance)

One of the signature sessions of the annual workshop, The TB Drug-Co-Development Roundtable was introduced the by Carl Mendel, who drew specific attention to the CTB2  database as an important new resource in the field.

Video

Presentation

 

Brief Industry Updates (10 minute presentations)

Jeffrey Hafkin (Otsuka)

Jeffrey Hafkin delivered an update on Otsuka’s programs to provide access to delamanid. The drug is currently available through the Global Drug Facility, and an access program in South Africa was unveiled on World TB Day, 2017. Efforts to expand access in several additional countries in Asia are ongoing. Overall, treatment has been provided for more than 2,400 patients in 45 countries. Additionally, the pediatric delamanid program has been expanded, following a WHO endorsement of use of delamanid in certain pediatric populations in October of 2016. Hafkin also provided an update on the ongoing development of the OPC-167832 compound.

Video

Presentation

 

Tine De Marez (Janssen)

Tine De Marez presented on the latest in the bedaquiline clinical development program. Areas covered included post-marketing commitments – a phase 3 study and pediatric study. De Marez covered locations where bedaquiline has already received approval and where submissions for approval have been completed. The update also noted that the pediatric trial of bedaquiline is underway, specifying that adolescents in the trial receive adult tablets and younger children receive a pediatric formulation of the drug. De Marez closed by providing an update on additional studies that include bedaquiline within drug combinations.

Video

Presentation

 

Dan Everitt (TB Alliance)

Dan Everitt gave an overview TB Alliance’s clinical portfolio. New and promising compounds discussed included TBAJ-587, a second generation diarylquinolone, and TBI-223 an oxazolidione that shows increased safety and a favorable efficacy profile in mice. Everitt also gave an encouraging update on the Nix-TB trial, which treats patients with XDR-TB or who have failed MDR-TB therapy with a fully novel regimen. As of the presentation, 31 patients had completed therapy and six months of follow-up. Interim results are quite promising. Finally, Everitt presented plans for TB Alliances upcoming NC-007 combination trial.

Video

Presentation

 

Charles Wells (Sanofi)

Charles Wells presented on the status of rifapentine, which was WHO prequalified in 2017. Registration has been achieved in Asia, with additional applications for approval submitted in several other regions. Wells gave updates on the TBTC Study 31 update, assessing a shortened regimen for active TB, and on the studies dedicated to rifapentine as part of fixed dose combinations. Wells also identified two recent preclinical drug candidates discovered by Sanofi.

 

June Kim (Qurient)

June Kim offered info on Qurient’s drug candidate, Q203. Kim reviewed the compound’s key attributes and noted that it is scheduled to enter an EBA study later in 2017. Development is continuing with a partner; Phase 1 has taken place in Russia.

Video

Presentation

 

Norbert Heinrich (University of Munich)

Norbert Heinrich presented an update on the drug candidate, BTZ2043. Heinrich provided background on the compound and summarized Phase 1 and 2 development plans, plus presented the slated trial design for an upcoming Phase 2a study.

Video

Presentation

 

Bern-Thomas Nyang’wa (TB-PRACTECAL)

Bern-Thomas Nyang’wa presented plans and goals for an ongoing late stage trial treating drug-resistant patients using combinations of bedaquiline, pretomanid, linezolid, moxifloxacin, and clofazimine. The trial consists of two stages. Patients will be observed after 8 weeks, and those who received regimens that do not perform up to standard will discontinue experimental treatment and receive standard therapy, while better performing regimens will be continued for six months and follow-up, seeking cure. The trial commenced in January of 2017. Nyang’wa articulated plans to expand the trial to additional sites.

Video

Presentation

 

Alison Webster (GlaxoSmithKline)

Alison Webster provided a concise overview of GSK’s TB drug portfolio. Webster discussed the organization’s strategies and approaches to drug discovery and development, including the role of beta-lactams. She also presented on GSK’s newest anti-TB compound, GSK070.

Video

Presentation

 

Panel Discussion

Moderator: Carl Mendel (TB Alliance)

Video

 

Next Generation Sequencing for TB Diagnostics: Advances, Opportunities and Necessary Steps

Moderator: Angela Starks (Centers for Disease Control and Prevention)

 

Session Introduction

Angela Starks (Centers for Disease Control and Prevention)

Video

 

 

Target Product Profiles for TB Diagnostics

Tim Rodwell (FIND)

Tim Rodwell discussed the need for new diagnostics and features important for them to possess to support technical and operational needs for use in low income settings considering new and upcoming TB drug regimens.

Video

Presentation

 

The Role of Genome Sequencing in Global Surveillance of Anti-Tuberculosis Drug Resistance

Matteo Zignol (World Health Organization)

Matteo Zignol discussed efforts to use genomic sequencing in surveilling global TB drug resistance. Zignol explained the operations of the program and associated challenges and solutions. Surveillance is performed using Xpert, and often smear microscopy, but the transportation of samples remains a challenge, losing up to 20% of samples in the process. Sequencing was posed as a solution to the loss of samples due to transportation. Zignol presented the results of using sequencing data for surveillance. Suboptimal sensitivity remains an issue, and there are ongoing efforts to develop models to adjust for misclassification. In particular, moxifloxacin is prone to sensitivity issues under sequencing methods. The first such project was successful; additional studies are ongoing and planning for later in 2017.

Video

Presentation

 

Advances in Targeted Sequencing

Dave Engelthaler (Translational Genomics Research Institute)

Dave Engelthaler discussed the novel methods in which sequencing-based assays can be implemented to address clinical questions. This included points on for identifying minor drug resistant subpopulations using nested amplicon next generation sequencing and interpretation. Engelthaler discussed the potential of these strategies to potentially identify early cases of drug resistance while addressing the needs to lower rates of sequencing error.

 

Microdilution Based MIC Detection for Old and New Anti-TB Drugs: Preliminary Evaluation of the Layout Proposed for the CRyPTIC Project

Daniela Cirillo (San Raffaele Scientific Institute)

Daniela Cirillo presented the work performed by the CRyPTIC consortium on evaluating a new microtiter plate designed to contain existing, new and repurposed TB drugs. The topics discussed included a validation study regarding reproducibility by assessing percentage of interpretable plates by reading day and by drug, agreement between readers and reading method. Various plate reading methods were also assessed and future work is planned on evaluating machine learning algorithms.

 

Panel Discussion

Moderator: Angela Starks (Centers for Disease Control and Prevention)

Video

 

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