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Palifermin (Kepivance™)

Palifermin (Kepivance™). Recombinant Human Keratinocyte Growth Factor Oncologic Drugs Advisory Committee Pediatric Subcommittee October 20, 2005 Amgen Inc. Palifermin Pediatric Development. Pre-registration Phase. Sept 2000 End of Phase 2. Adult studies conducted. Sept 03 Pre- BLA.

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Palifermin (Kepivance™)

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  1. Palifermin (Kepivance™) Recombinant Human Keratinocyte Growth Factor Oncologic Drugs Advisory Committee Pediatric Subcommittee October 20, 2005 Amgen Inc.

  2. Palifermin Pediatric Development Pre-registration Phase Sept 2000 End of Phase 2 Adult studies conducted Sept 03 Pre- BLA Jun 04 Adult BLA submission 2000 2001 2002 2003 2004 Early Discussions Pediatric Phase 1/2 design Pediatric PMC agreed BLA: Biologics License Application PMC: Post-Marketing Commitment

  3. Palifermin Pediatric Program: Former Proposal Phase 1/2 Study with COG* • Patients: B-NHL receiving COP/COPADM/CYM • Age 3-16 years • Palifermin i.v. 3 consecutive days before chemotherapy Phase 1 Phase 2 • Palifermin vs. Placebo • Safety • Efficacy • Dose escalation, • 4 dose cohorts • Safety, Pharmacokinetics *COG: Children’s Oncology Group. B-NHL: B-cell Non-Hodgkin’s Lymphoma, stage 1-2 COP/COPADM/CYM: multi-agent chemotherapy regimen

  4. Post-registration Phase Palifermin Pediatric Development Dec 04 Adult BLA approved Mar 05 Jun 05 Sept 05 2004 2005 Pediatric PMC Change in Standard Therapy Revised Study Proposal Further Study Specification

  5. Palifermin Pediatric Program: Current Proposal Separate Studies with PBMTC and COG* • Patients: Acute leukemias, neuroblastoma • Age 1-16 years, 3 age groups • Palifermin i.v. 3 consecutive days before + after chemo Phase 1 Phase 2 • Dose escalation, 4 dose cohorts • Safety • Pharmacokinetics • Safety • Homogeneous • population • Age 1-6 years • Efficacy, Safety • Randomized • Broad population • Age 1-16 years *PBMTC Pediatric Blood and Marrow Transplant Consortium COG: Children’s Oncology Group

  6. Palifermin Pediatric ProgramPhase 1 with PBMTC* Sites • Objectives: Dose finding, safety, pharmacokinetics • Patients: ALL or AML requiring TBI and high-dose chemotherapy with alloHSCT • 36-72 patients • 3 age groups (1-2, 3-11 and 12-16 years) • Conventional dose escalation • 4 dose cohorts (20 – 40 – 60 – 80 g/kg/d) given 3 consecutive days before and after chemotherapy • Dose escalation decisions for each age group *PBMTC Pediatric Blood and Marrow Transplant Consortium. ALL: Acute Lymphoblastic Leukemia, AML: Acute Myeloid Leukemia, TBI: Total Body Irradiation, alloHSCT: allogeneic hematopoietic stem cell transplant.

  7. Palifermin Pediatric ProgramProposed Phase 2 with COG*: Safety • Objective: Safety • Patients: Homogeneous patient population • 60-80 patients, randomized • Age group < 6 years • Autologous transplant setting • Neuroblastoma • Doses established in Phase 1 *COG: Children’s Oncology Group

  8. Palifermin Pediatric ProgramProposed Phase 2 with COG*: Efficacy • Objectives: Efficacy, Safety • Patients: • 200 patients, broad population, randomized to placebo • Age 1-16 years • Hematological malignancies or neuroblastoma undergoing either allogeneic or autologous HSCT • Endpoints: • Incidence and duration of WHO Grade 3-4 oral mucositis • Acute and long-term safety • Doses established in Phase 1 *COG: Children’s Oncology Group

  9. Palifermin Pediatric Development Common to All Drugs in Pediatric Oncology • Similar to pegfilgrastim Palifermin-Specific Issues • Change in standard therapy • Competition with therapeutic agent trials • Need to establish long-term safety

  10. Palifermin Pediatric DevelopmentSummary and Conclusions • Positive benefit/risk profile based on approved adult indication • Severe mucositis is an unmet medical need in the pediatric transplant population • Pediatric development program with common and product-specific challenges • Amgen shares the goal of making this drug available to children

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