orBec

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orBec (oral beclomethasone dipropionate)
NDA 22-062

• DOR BioPharma, Inc.
• Oncologic Drugs Advisory Committee
• May 9, 2007

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Christopher Schaber, Ph.D

• President CEO
• DOR BioPharma, Inc.

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Agenda

• Introduction
• orBec beclomethasone dipropionate
• Acute Graft-vs-Host Disease (GVHD)
• Rationale for oral BDP
• Randomized, placebo-controlled trials of oral BDP
• Summary of clinical trial results
• Benefit/Risk

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Presenter

• George B. McDonald, MD
• Professor of Medicine, University of Washington
• Head, Gastroenterology/Hepatology Section, Fred
  Hutchinson Cancer Research Center

Moderator
Timothy C. Rodell, MD Medical MonitorDOR
BioPharma, Inc.
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External Advisors
David Hockenbery, MD Member at the Fred Hutchinson Cancer Research Center Lead Investigator, Study ENT 00-02
Theodore Gooley, PhD Member at the Fred Hutchinson Cancer Research Center Lead Statistician, Study 875
Keith Sullivan, MD Duke University Medical Center Chief, Medical Oncology and Transplantation
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DOR BioPharma, Inc.

• Focused on treatments for
• Life-threatening side effects of cancer
  treatments
• Serious GI diseases
• Enteron Pharmaceuticals is a wholly owned
  subsidiary of DOR BioPharma, Inc.

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Beclomethasone Dipropionate

• Diester of beclomethasone, a potent synthetic
  corticosteroid
• Anti-inflammatory and immunosuppressive effects
• Widely used in topical applications
• Inhaled
• Intranasal
• Enema

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Nomenclature

• BDP
• Beclomethasone dipropionate
• Oral BDP
• Formulation
• Immediate-release (IR) tablet (1 mg)
• Delayed-release, enteric-coated (EC) tablet (1
  mg)
• Proposed dosing
• 1 IR and 1 EC 4 times daily
• orBec
• Proposed trade name for oral BDP

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Development History
1991 Oral BDP development began (Investigator-Initiated IND)
1991 Development funded by FDA Orphan Drugs Division
1995 Phase 1 trial completed (Study 615)
1998 Phase 2 trial completed (Study 875)
1998 Orphan Indication Designation
1999 Ownership was transferred to Enteron Pharmaceuticals
2000 Fast Track Designation
2005 Pivotal Phase 3 trial completed under Special Protocol Assessment (SPA), Division of Gastrointestinal and Coagulation Drug Products (Study ENT 00-02)
2006 NDA 22-062 submitted September 21, 2006
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Studies of Oral BDP in Patients with GI GVHD
Patients enrolled Patients enrolled Patients enrolled
Study Phase Description Sponsor Placebo BDP Total
615 1 Uncontrolled Investigator initiated 0 42 42
1500 1 Uncontrolled Investigator initiated 0 16 16
875 2 Single-center, randomized, placebo-controlled Investigator initiated 29 31 60
ENT 00-02 3 Multicenter, randomized, placebo-controlled Enteron Pharmaceuticals (subsidiary of DOR BioPharma, Inc.) 67 62 129
Total 96 151 247
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Basis for Approval

• Approval is merited based on a favorable safety
  profile and clinical benefits as measured by
  reductions in
• GVHD treatment failure
• Mortality at transplant Day 200
• Mortality 1 year post-randomization

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Proposed Indication for orBec (oral BDP)

• orBec is indicated for the treatment of graft
  versus host disease (GVHD) involving the
  gastrointestinal (GI) tract in conjunction with
  an induction course of high-dose prednisone or
  prednisolone.

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