Eligen B12 Human Clinical Results. Michael V. Novinski President and Chief Executive Officer

Transcription

1 Eligen B12 Human Clinical Results Michael V. Novinski President and Chief Executive Officer July 29, 2008

2 Safe Harbor Safe Harbor Statement Regarding Forward-Looking Statements The statements in this presentation and oral statements made by representatives of Emisphere relating to matters that are not historical facts (including without limitation those regarding potential third party collaborations, future performance or financial results, the timing or potential outcomes of research collaborations or clinical trials, any market that might develop for any of Emisphere's product candidates and the sufficiency of Emisphere's cash and other capital resources) are forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of Such statements involve risks and uncertainties, including, but not limited to, the likelihood that one or more potential third party collaborations may not be consummated, that actual performance or results could materially differ, that future research will prove successful, the likelihood that any product in the research pipeline will receive regulatory approval in the United States or abroad, the ability of Emisphere and/or its partners to develop, manufacture and commercialize products using Emisphere's drug delivery technology, or Emisphere's ability to fund such efforts with or without partners. Emisphere undertakes no obligation to update any of these statements. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as to the date hereof. Accordingly any forwardlooking statements should be read in conjunction with the additional risks and uncertainties detailed in Emisphere's filings with the Securities and Exchange Commission, including those factors discussed under the caption "Risk Factors" in Emisphere's Annual Report on Form 10-K for the fiscal year ended December 31, 2007, filed on March 17,

3 Agenda Introductory Remarks B12 and Emisphere B12 Clinical Data Expert Commentary Wrap-up 3

4 Introductory Remarks 4

5 Emisphere Objective Commercialize the Eligen Technology Use B12 as a possible avenue 5

6 Why B12? Vitamin B12 is an essential vitamin, but a poorly absorbed molecule/nutrient For those who are B12 deficient or at-risk, current options are injections (painful ), or megadoses in tablet form (poorly absorbed with high uncertainty) Currently 5 million people in the United States are taking 40 million injections annually, with 250 million injections estimated worldwide At least an estimated 600 million tablets are purchased annually by an additional 5 million people 6

7 B12 Program Animal studies High dose one species Physiological dose one species Physiological dose second species Human Studies High dose absorption and rate Lower dose absorption and rate versus IV and commercial tablet 7

8 Eligen B12 Human Clinical Trial Study conducted in 20 healthy males, with a single administration of Eligen B12 well tolerated with no adverse reactions Eligen B12 formulation bypasses normal absorption process Eligen B12 formulation demonstrates 240% improvement in bioavailability versus standard oral tablet in healthy males Absorption time with Eligen B12 was 30 minutes, versus 6.5 hours for standard oral tablet Eligen B12 bioavailability of 5% 8

9 What Does This Mean? Reduces uncertainty of megadose oral therapy and oral therapy in general Patients at-risk Individuals concerned about levels of this essential vitamin Reduce dependence on injections taken by millions Improve compliance More convenient dosage formulation Highly effective treatment through consistently enhanced bioavailability Moving away from injections broadens application for oral therapy as a solution or approach to the problems associated with B12 supplementation 9

10 Eligen B12 Human PK Study Pharmacokinetic study conducted in 20 healthy male subjects divided into four arms Four patients received 1mg B12 intravenously Four patients received 10mg Eligen B12 Six patients received 5mg Eligen B12 Six patients received commercially available 5mg B12 tablet 10mg Eligen group served essentially as a pilot arm to determine appropriate dose for pharmacokinetic purposes Following these results, dosage reduced by 50% to a level that is being used in certain commercially available formulations Expect to be able to reduce dose further based on the data received and the linear relationship between the two Eligen groups 10

11 Eligen B12 Human Clinical Data Mean B12 peak blood levels were more than 10 times higher for the Eligen B12 formulation than the 5mg commercial tablet pg/ml and 1239 pg/ml, respectively Time to reach peak concentration reduced by more than 90%, where mean Tmax was 0.5 hours for the 5mg Eligen B12 and 6.8 hours for the commercial 5mg dose Mean AUC (24h) values were hr* pg/ml for Eligen B12 and hr* pg/ml for the commercial 5mg product 11

12 Eligen B12 Mean PK Data Treatment Cmax (pg/ml) Tmax (hr) AUC 24 (hr* pg/ml) 5 mg B12 Commercial Formulation 1239± ± ± mg B12 Eligen Formulation 12847±6613* 0.5±0.2 * 54609±16405 * *p<0.05, t-test 12

13 Eligen B12 Human Clinical Data Mean (± S.D.) Serum B12 Concentrations Concentrations in Healthy Male Subjects Serum Cyanocobalamin Concentration (pg/ml mg Eligen B12 5 mg Commerical B12 10 mg Eligen B12 1 mg IV Commerical B Time (hr) 13

14 Eligen B12 Human Clinical Data Mean Serum B12 Concentrations in Healthy Male Subjects Serum Concentration (pg/ml mg Commercial B12 5 mg Eligen B Time (hr) 14

15 Eligen B12 Human Clinical Data Mean Cmax Values Following Oral B12 Treatments mg Eligen B12 5 mg Eligen B12 5 mg commercial B12 Cmax (pg/ml) Mean AUC Values Following Oral B12 Treatments mg Eligen B12 5 mg Eligen B12 5 mg commecial B12 AUC (hr*pg/ml)

16 Eligen B12 Clinical Update Conclusions Eligen significantly enhances B12 delivery in humans Eligen utilized a new mechanism of B12 delivery B12 animal studies proved highly predictive of the observed human response Next steps Clinical investigation of Eligen B12 pharmacokinetics at lower doses in target populations (Q3/2008) Clinical investigation of Eligen B12 therapeutic efficacy in target populations (Q4/2008) 16

17 Eligen B12 Why It s s Important for Patients This presents a potential paradigm shift in the way we approach B12 deficiency and at-risk people Emisphere s carriers reduce the potential uncertainty associated with oral megadoses of B12 New formulation may prevent the substantial number of B12 injections given worldwide This may be an effective recourse for people who are at-risk of B12 deficiency, are already identified as B12 deficient, and for people who are concerned about this important vitamin 17

18 Eligen B12 Commercial Application Food fortification Supplementation for those individuals concerned about B12 Supplementation for the at-risk populations Supplementation for B12 deficient segments 18

19 Eligen B12 Commercial Application Clinical strategy to support all segments Regulatory strategy to allow access to all segments NDI GRAS Evaluate each segment to maximize the potential valuation 19

20 Eligen B12 Summary All research indicates that Eligen B12 presents a significant improvement to help approach the problem of B12 supplementation, regardless of level of supplementation that may be required Emisphere is committed to commercializing Eligen B12 as an opportunity for all defined segments These data only further demonstrate the value of the Eligen Technology as a platform to improve the bioavailability for difficult to absorb molecules; including, but not limited to, other essential vitamins and minerals, prescription drug products, and even other possible molecules 20

21 Q&A 21