Winter 1-2017 Newsletter

Winter 1-2017 Newsletter


Dear colleagues,

In this edition of the Kinexum newsletter issued in our still young year, we want to highlight the highly significant changes at FDA and unprecedented number of draft guidances issued just in the past three months, as well as the arrival of the 21st Century Cures Act.  These and other developments are discussed in the roundtable web conference held on January 20.  Many of you have requested the audio file of the conference, and it is available at the link in the attached newsletter. We will soon provide an edited transcript.  We barely scratched the surface in that call.  Do expect follow-up roundtable discussions and other special presentations. 

In short, we are entering an era in which the ecology of health product development and regulation is changing at an unprecedented rate.  We at Kinexum join many of you and your organizations in striving to stay on top of these changes and working toward a healthier environment for product development to address unmet need. 

Also in addition are two commentaries by my colleagues Brian Harvey and Sue Manley.  They bring enormous experience from their careers.  I am also proud to introduce you to colleagues who have recently joined Kinexum.  These professionals add to our ability both to serve our clients and to foster communication and understanding among a wide variety of stakeholders.

Best wishes,



“Wow or Yeow!?”

We are now in a new era of therapeutic development and regulation

A January 20, 2017

Special Roundtable Discussion


Timothy Franson, M.D., Chief Medical Officer, Your Encore

David Fox, J.D., Partner, Hogan Lovells

Brian Harvey, M.D, PhD., Distinguished Advisor, Kinexum

Moderated by

Alexander Fleming, M.D., CEO, Kinexum

Click here to listen,

Conference starts at minute 9 mark

Winter Newsletter 1-2017

FDA E-Submission

As we move further into 2017, we want to remind all our clients that the FDA is now requiring their e-submission system be used for most submissions. Kinexum now offers a complete set of e-submission services starting with the development and authoring of documents to the final submission through the FDA’s electronic gateway.

Kinexum continues to offer the highest level of regulatory document preparation based on each client’s specific regulatory strategy.   Our submission development teams are supported by highly experienced regulatory, CMC, pre-clinical, clinical consultants as well as medical writers to ensure a clear narrative and succinct presentation of the data. Our goals are to deliver the highest quality submission possible. Want to learn more about Kinexum submission capabilities? Contact Steve Casey

NASH Regulatory Perspectives From FDA Staff

By Brian Harvey, MD, PhD  Formerly Director, Division of Gastroenterology Products, CDER

This is a summary of the important concepts and current regulatory thinking presented by FDA Medical Officer Dr. Lara Dimick-Santos at the most recent Liver Forum held just prior to the American Association for the Study of Liver Diseases (AASLD) Meeting. The Liver Forum is an opportunity to bring together individuals from academics, government, industry, as well as medical practitioners and patient advocates, to learn about the current state of research in various liver diseases, as well as discuss potential paths forward for increasing knowledge of liver disease pathophysiology that help generate data supporting the development of safe and effective treatments.

This FDA presentation focused upon Non-Alcoholic Steatohepatitis (NASH), with an emphasis on appropriate clinical trial design that may increase the odds for generating data that will meet the FDA threshold for drug or biologic product approval.  The critical aspect of this is the choice of endpoints for the proposed pivotal clinical trial.  There are several potential pathways which involve the use of traditional clinical endpoints, intermediate clinical endpoints, as well as surrogate endpoints reasonably likely to predict clinical benefit in patients.

Continue to the full article


Recent Kinexum Publications

William T Cefalu, John B. Buse, Jaakko Tuomilehto, G. Alexander Fleming, Ele Ferrannini, Hertzel C. Gerstein, Peter H. Bennett, Ambady Ramachandran, Itamar Raz, Julio Rosenstock, Steven E. Kahn. Update and Next Steps for Real-World Translation of Interventions for Type 2 Diabetes Prevention; Reflections from a Diabetes Care Editor’s Expert Forum. Diabetes Care Jul 2016, 39 (7) 1186-1201.

Regulatory Operations Success

By Susan Manley  Formerly, Executive Director and Head, Global Regulatory Operations, Novartis Pharmaceuticals

As a former head of Regulatory Operations for a global pharmaceutical company, I have learned some keys to success that I would like to share.   While these keys may seem like common sense, it is easy to lose focus in a fast-moving organization.  Operational success is based on a clearly defined strategy, great planning and open communication to customers and project staff at all levels.

Strategy should guide you in concentrating on the end goal and the steps along the way.  Sometimes the grand design could change during a project, but this should be avoided as it can result in delays.  For example, if the short-term plan to start a clinical study in the US in indication A is changed to move the study to Japan in indication B, there will certainly be delays from the original schedule.

Continue to the full article

David C. Klonoff, G. Alexander Fleming, Douglas B. Muchmore. Brian M. Frier. Hypoglycemia Evaluation and Reporting in Diabetes: Importance for the Development of New Therapies. Diabetes MetabolismResearch and Review. Published online at  January 2017


The newest members of the Kinexum team

We would like to introduce:


Richard Paul, MD

Clinical Development & Regulatory Strategy

To learn more about Rich



Elizabeth Whalley-Buono, RN, JD, MBA

Process and Innovation Management


To learn more about Liz

Susan Manley

Regulatory Operations

To learn more about Sue



Robert Kaster, RPh


To learn more about Rob

Sandor Bernath, PhD, MBA

Medical Writing

To learn more about Sandor



Diane Paul, MS, RN

Project Management & Clinical Operations

To learn more about Diane

Peggy Hagerty

Medical Writing.

To learn more about Peggy

Contact us:

Kinexum Services LLC

PO Box 1260

550 East Ridge Street

Harpers Ferry, WV 25425

P - 304-535-3037

F - 304-535-3166