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First Example in Respiratory Area

First Example in Respiratory Area. Steroid Nasal Spray Seasonal Allergic Rhinitis, Age  12 yr Randomized, Double-Blind, Parallel-Group 5-21 Days of Screening Followed by 2 Weeks of Treatment Placebo, 50mcg, 100mcg, 200mcg, 400mcg, ~ 125 Patients/Treatment.

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First Example in Respiratory Area

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  1. First Example in Respiratory Area • Steroid Nasal Spray • Seasonal Allergic Rhinitis, Age12 yr • Randomized, Double-Blind, Parallel-Group • 5-21 Days of Screening Followed by 2 Weeks of Treatment • Placebo, 50mcg, 100mcg, 200mcg, 400mcg, • ~ 125 Patients/Treatment.

  2. Primary Efficacy & Key Safety Endpoints • Mean change from baseline over the entire treatment period in daily, reflective total nasal symptom scores (rTNSS) • Adverse events • 24-hour urinary cortisol excretion

  3. Primary Efficacy EndpointMean Change in Daily rTNSS over Treatment Period Day

  4. Primary Efficacy Endpoint Mean Change in Daily rTNSS over Entire Treatment Period

  5. Onset of ActionMean Change in AM Pre-dose iTNSS Hour

  6. “Important” Efficacy Endpoint Mean changes in AM pre-dose iTOSS over Entire Treatment Period

  7. Adverse Events during Treatment Period

  8. 24-hour Urinary Cortisol ExcretionChange from Baseline to Week 2 (UC pop)

  9. Dose Selection • Which dose would you choose? • Why?

  10. Discussion • Flat Dose Response Pattern for steroids, once a particular does starts to work, all higher doses work? • Why not study more lower doses, e.g. 25 mcg, or even 12.5 mcg? • Why not use active control (market leader) to better asses relative efficacy and safety? • Why not wait until the final formulation and final device are developed?

  11. Second Example in Respiratory Area • A Novel Compound • COPD (Chronic Obstructive Pulmonary Disease) – Smoker’s Lung • Randomized, Double-Blind, Parallel-Group, >40 yrs • 2 Weeks Run-in Followed by 4 Weeks of Treatment • Placebo, 5mg, 10mg, 15mg. • ~105 Patients/Treatment

  12. One of the Primary Efficacy Endpoints Change from baseline in trough FEV1 at Endpoint • Measured in AM prior to dosing

  13. Phase II Study Results Dose 5mg 10mg 15mg FEV1(Trt – Plb) 60ml 10ml 140ml (Endpoint at 4 wks)

  14. Phase III Study Results Study 1 2 3 4 FEV1(15mg-Plb) 40ml 20ml 30ml 30ml (Average Over Time) FEV1(15mg-Plb) 80ml 40ml 30ml 40ml (Endpoint at 24 wks)

  15. Discussion • A successful phase II does ranging study does not guarantee phase III successes – Duh! • Is it Preventable? • If you say yes, then how?

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