Myths, Presumptions, and Facts about Obesity Steven B. Heymsfield 2

Size: px

Start display at page:

Download "Myths, Presumptions, and Facts about Obesity Steven B. Heymsfield 2"

Juniper Oliver
6 years ago
Views:

H eld o n Translational the 3 rd Thursday of Medicine each month Grand Rounds A joint educational program of Pennington Biomedical Research Center and Kinexum to support the development

1 H eld o n Translational the 3 rd Thursday of Medicine each month Grand Rounds A joint educational program of Pennington Biomedical Research Center and Kinexum to support the development and commercialization of promising discoveries. Myths, Presumptions, and Facts about Targeting Novel Indications for Metabolic Drugs 12 noon CST, February 21, 2013 Alexander Fleming, M.D

2 Myths, Presumptions, and Facts about Obesity Steven B. Heymsfield 2

3 Today A mile wide, a mile high, and inch deep Consider products all categories of regulated health Intend to get to a better understanding about how we achieve preventative and r estorative therapies* Want to emphasize that t his about T2DM and obesity is not just Touch on tools for supporting evelopment of preventative therapies d 3

4 Diabetes and Obesity Population Targets for Therapeutic Development: Metabolic Syndrome Pre- Diabetes Aging Diabetes and obesity are the easily identifiable populations that we treat when they reach late stages. Let s look at it another way- in 4

5 Cancer Aging Alzheimer's Depression Diabetes Cardio vascular Disease Obesity Increasing evidence points to the importance of metabolic and nutritional factors as root contributors to most causes of mortality. These risk factors can be eliminated or modified. 5

constellation of metabolic and nutritional disorders,

6 Cancer Aging Alzheimer's Depression Diabetes Cardio vascular Disease Obesity What could we call this constellation of metabolic and nutritional disorders, which could be targeted with one or more therapies? How about?. M etabesity 6

7 A Conversation about Metabesity 7

8 OXIMA Sometime? ( nicotinic amidoxime derivative) Tablet 200 mg R INDICATIONS OXIMA is indicated as an adjunct to metformin/diet to lower the blood glucose patients with type 2 diabetes whose hyperglycemia cannot be controlled by standard therapy. in.for prevention of type 2 diabetes in people with metabolic syndrome mellitus 8

9 OXIMA ( nicotinic Tablets 200 Where amidoxime are d erivative) mg and 600 mg we headed? R INDICATIONS Adjunct to metformin/diet to lower the blood glucose i n patients with T 2D whose hyperglycemia cannot be controlled by standard therapy. Reduction in major adverse cardiovascular stroke, and death) in people with T2DM events (MI, Prevention of type 2 diabetes metabolic syndrome mellitus in people with 9

10 Regulatory Pathways Health Products for Marketing of in the USA DSHEA FD&C Act Dietary Supplement FD&C Act PHS Act Devices Food Biologics Medical Food Drugs Botanical Drug 10

11 FDA regulation of is health products based on Intended Use A F ood is consumed orally for nutritive value. taste, aroma, A D ietary Supplement is taken to supplement the diet to produce effects on structure and function of the human body A D rug is used to diagnose, or prevent disease. cure, treat, or mitigate, A M edical Food i s a specially formulated p roduct intended for the dietary management of a patient w ith impaired capacity to ingest, digest, 11 a bsorb, or m etabolize o rdinary foodstuffs.

12 Many health products are not regulated 12

13 A dietary supplement can never be developed as a drug. This is a myth Facts Niacin Niaspan Omega-3 fish oils products Lovaza (DHA+EPA) Resveratrol Sirtris product 13

14 A medical food can carry a disease claim and require a prescription. This is true Presumptions: The medical food route is a viable commercialization pathway A lower level of evidence is required of medical food products than for drug products Myth: FDA is not likely to notice medical food a noncompliant 14

15 15

David Yates President Nestle HealthCare Nutrition 10801 Red Circle Drive Minnetonka, Minnesota 55343 WARNING LETTER Re: CFSAN-OC-10-04 Dear Mr.

16 David Yates President Nestle HealthCare Nutrition Red Circle Drive Minnetonka, Minnesota WARNING LETTER Re: CFSAN-OC Dear Mr. Yates: This is to advise you that the Food and Drug Administration (FDA) reviewed your w ebsites at the Internet addresses Nutrition.com, a nd in November Your BOOST Kid Essentials Nutritionally Complete D rink ( Vanilla, Chocolate, and Strawberry flavors) is promoted on your websites as a "medical 16 f ood," and t he labeling claims on your websites represent the product as a m edical food for the medical condition of " failure

17 S hort term clinical dietary supplement This is a probably a s tudies to address c linical need. presumption and myth. can enable unmet a Presumptions: Consumers will become committed long term users of a product based on data The medical community will recommend these therapies. Competing products market will not embrace enter the and 17

18 The Top- Selling Herbal Supplements Drug, and Mass Market Channel in the for 2011 in the Food, United States 18

19 FDA can only approve indications for conditions that are universally embraced by t he clinical community. This is a myth based on: Presumptions: Therapeutic indications must be based on thorough mechanistic understanding ADA has to formally support any new metabolic indication for FDA to adopt it. 19

20 20

21 21

A Study With A leglitazar in Patients With a Recent Syndrome and Type 2 Diabetes Mellitus Acute Coronary PURPOSE This double-blind, parallel, two- arm study will evaluate the potential to reduce

22 A Study With A leglitazar in Patients With a Recent Syndrome and Type 2 Diabetes Mellitus Acute Coronary PURPOSE This double-blind, parallel, two- arm study will evaluate the potential to reduce cardiovascular risk, the tolerability and long- term safety profile of aleglitazar compared to placebo on top of standard care in patients with recent acute coronary syndrome (ACS) and type 2 diabetes mellitus. Patients will be randomized to receive either a leglitazar or placebo once daily as oral doses. The study will last until at least 950 events occur, but time on study treatment w ill be for at least 2.5 years. 22

25 Implications for P harma: but E vent driven pivotal trials cost increase 2-3X for Phase 3; in the range of $ million G iven the cost and time this investment must deliver positive benefit Neutral/equivocal data will be punished by the market (e.g. Z etia) All risk is back loaded investment is front loaded

26 D rug products m atter without IP cannot composition of be s uccessfully developed. This is a presumptions based on: 5 years market exclusivity in the US (10 years in the EU is insufficient to warrant investment Use and formulation patents strongly defended. But, there are Metformin (Glucophage) Niacin (Niaspan) Coming soon--biosimilars cannot be many exceptions: 26

27 How do we get the evidence for preventive indications? Faster Less expensive More creditable 27

75% per year rate* Low CV Risk Total T2DM Population at Risk

28 Concept of the Large Simple Trial High CV Risk High risk 4.0% per year event rate* Moderate : 0.75% per year rate* Low CV Risk Total T2DM Population at Risk N=5,000 N=25,000 Conventional Enriched Trial Approach (RCT) MACE events Large Simple Trial Approach (LST)

29 How Can the LST Reduce T ime to Results? Recruitment Conventional Enriched Trial Approach (RCT) Time on Drug Analysis 1 year 1.5 years.3 years Recruitment Time on Drug Analysis 4-8 weeks 1.5 years.3 years Large Simple Trial Approach (LST) Drug Information Association 29

30 Opportunities to participate in trials are offered to physicians and patients through multiple web based sites and e- mail. A s many clinical sites ( 1,000-10,000) can be registered as needed from a vast pool of physicians (1-10 patients per site). I nvestigators are q ualified and recruited simple web- based form P atients enroll by web, of medical office staff phone, or A ssigned drug is distributed by mail central pharmacy Drug Information Association How Can the LST Reduce Recruitment Time? with the from by a help 30

31 How do we get there? PROGENSA R (Resenoprenol) Tablets 500 mg INDICATION Progensa is indicated for persons 40 years age and older as a means of increasing life span and slowing age- related decline in quality of life and function. of 31

Where Do We Stand? Unraveling the FDA Regulatory Pathway for Diabetes and Obesity Drug Approval. Alexander Fleming, MD

Where Do We Stand? Unraveling the FDA Regulatory Pathway for Diabetes and Obesity Drug Approval Alexander Fleming, MD 1 Presenter Disclosure Alexander Fleming, M.D. Board Member/Advisory Panel/Consultant: