# Presentation: 750081

##  orBec® (oral beclomethasone dipropionate) NDA 22-062

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

**5030.01**

## Christopher Schaber, Ph.D

**President & CEO**
**DOR BioPharma, Inc.**

**5031.01**

## 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

**5032.01**

## 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.

**5158.01**

## 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 |

**5033.01**

## 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.

**5034.01**

## Beclomethasone Dipropionate

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

**5035.01**

## 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

**5036.01**

## Development History

**5037.01**

| 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 |

## Studies of Oral BDP in Patients with GI GVHD

|  |  |  |  | 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 |

**5038.01**

## 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

**5039.01**

## 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.

**5040.01**