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Clofarabine ODAC PresentationClofarabine ODAC PresentationClofarabine ODAC PresentationClofarabine ODAC Presentation
Pediatric Acute Leukemia
December 1, 2004
Pediatric Acute Leukemia
December 1, 2004
Clofarabine ODAC PresentationClofarabine ODAC PresentationClofarabine ODAC PresentationClofarabine ODAC Presentation
Steve Weitman, MD, PhDChief Medical OfficerILEX Oncology, Inc.
Steve Weitman, MD, PhDChief Medical OfficerILEX Oncology, Inc.
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Clofarabine ODAC ConsultantsClofarabine ODAC Consultants
Sima Jeha, MDSt. Jude Children’s Research Hospital
Ka Wah Chan, MDM.D. Anderson Cancer Center
Laurel Steinherz, MDMemorial Sloan-Kettering Cancer Center
Robert Arceci, MD, PhDSidney Kimmel CCC at Johns Hopkins
Sima Jeha, MDSt. Jude Children’s Research Hospital
Ka Wah Chan, MDM.D. Anderson Cancer Center
Laurel Steinherz, MDMemorial Sloan-Kettering Cancer Center
Robert Arceci, MD, PhDSidney Kimmel CCC at Johns Hopkins
Peter Steinherz, MDMemorial Sloan-Kettering Cancer Center
Stephen Sallan, MDDana-Farber Cancer Institute
Varsha Gandhi, PhDM.D. Anderson Cancer Center
Eric Sandler, MDNemours Children’s Clinic
Peter Adamson, MDChildren’s Hospital of Philadelphia
Peter Steinherz, MDMemorial Sloan-Kettering Cancer Center
Stephen Sallan, MDDana-Farber Cancer Institute
Varsha Gandhi, PhDM.D. Anderson Cancer Center
Eric Sandler, MDNemours Children’s Clinic
Peter Adamson, MDChildren’s Hospital of Philadelphia
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AgendaAgendaIntroduction Steve Weitman, MD, PhD
Pediatric Leukemia: Robert Arceci, MD, PhDNeed for New Treatment Options Sidney Kimmel CCC at
Johns Hopkins
Clofarabine Pivotal Studies Steve Weitman, MD, PhD
Clinician’s Perspective Stephen Sallan, MDDana-Farber Cancer Institute
Clofarabine Development Plan Steve Weitman, MD, PhD
Introduction Steve Weitman, MD, PhD
Pediatric Leukemia: Robert Arceci, MD, PhDNeed for New Treatment Options Sidney Kimmel CCC at
Johns Hopkins
Clofarabine Pivotal Studies Steve Weitman, MD, PhD
Clinician’s Perspective Stephen Sallan, MDDana-Farber Cancer Institute
Clofarabine Development Plan Steve Weitman, MD, PhD
The ChallengeThe Challenge
Robert Arceci, MD, PhDRobert Arceci, MD, PhD
Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsDepartments of Oncology and Pediatrics
Pediatric Leukemia:Pediatric Leukemia:Need for New Treatment OptionsNeed for New Treatment OptionsPediatric Leukemia:Pediatric Leukemia:Need for New Treatment OptionsNeed for New Treatment Options
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Treatment of Patients with Pediatric LeukemiasTreatment of Patients with Pediatric Leukemias
Treatments for newly diagnosed patients with ALL & AML use aggressive multi-drug regimens
Overall survival for pediatric ALL and AML
has improved, but is approaching a plateau
20% ALL and 50% AML have disease recurrence
Treatments for newly diagnosed patients with ALL & AML use aggressive multi-drug regimens
Overall survival for pediatric ALL and AML
has improved, but is approaching a plateau
20% ALL and 50% AML have disease recurrence
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Common Pediatric CancersCommon Pediatric Cancers
De Novo ALL & AML (3,000 cases)
CNS (2,200 cases)
Relapsed ALL & AML (900 cases)
Hodgkins (875 cases)
Neuroblastoma (650 cases)
Wilms' (500 cases)
Osteosarcoma (400 cases)
Rhabdomyosarcoma (350 cases)
Ewing's (200 cases)
Hepatic (150 cases)
De Novo ALL & AML (3,000 cases)
CNS (2,200 cases)
Relapsed ALL & AML (900 cases)
Hodgkins (875 cases)
Neuroblastoma (650 cases)
Wilms' (500 cases)
Osteosarcoma (400 cases)
Rhabdomyosarcoma (350 cases)
Ewing's (200 cases)
Hepatic (150 cases)
De NovoALL & AML
CNS
Source: SEER Pediatric MonographAssumes: 75% ALL cases cured and 50% AML cases cured
Relapsed/ RefractoryALL & AML
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Challenges in Pediatric Relapsed & Refractory LeukemiasChallenges in Pediatric Relapsed & Refractory Leukemias
Heterogeneous population
Multi-drug resistance is common in leukemia cells, particularly at relapse
Dose intensification with combination therapies has resulted in significant co-morbidities and organ dysfunction
Transplant is the best curative option but requires disease control and time to identify donor
Heterogeneous population
Multi-drug resistance is common in leukemia cells, particularly at relapse
Dose intensification with combination therapies has resulted in significant co-morbidities and organ dysfunction
Transplant is the best curative option but requires disease control and time to identify donor
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0%
50%
100%
0 1 2 3 4 5 6Years Followed
Pro
bab
ilit
y o
f S
urv
ival
Survival in Relapsed/ Refractory Pediatric Leukemia Survival in Relapsed/ Refractory Pediatric Leukemia
Weitman, et. al. J Pediatr Hematol Oncol. 1997;17:197-207
Acute Lymphoid Leukemia
Acute Myeloid Leukemia
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New Agents Needed for Remission Induction in Relapsed/ Refractory PatientsNew Agents Needed for Remission Induction in Relapsed/ Refractory Patients
Few agents have been approved for pediatric leukemiaMost commonly used agents approved many years ago
Methotrexate (1953) 6-Mercaptopurine (1953)Vincristine (1963)Ara-C (1969)Doxorubicin (1974)
Development of new pediatric oncology agents has lagged behind adult oncology drug development
Few agents have been approved for pediatric leukemiaMost commonly used agents approved many years ago
Methotrexate (1953) 6-Mercaptopurine (1953)Vincristine (1963)Ara-C (1969)Doxorubicin (1974)
Development of new pediatric oncology agents has lagged behind adult oncology drug development
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ConclusionsConclusions
Relapsed leukemia is the third most common childhood cancer
Successful treatment of relapsed and refractory pediatric leukemias remains a major challenge
Patients with multi-drug resistant leukemia are refractory to chemotherapy and have accumulated organ toxicities
New, well tolerated and effective agents are urgently needed
Relapsed leukemia is the third most common childhood cancer
Successful treatment of relapsed and refractory pediatric leukemias remains a major challenge
Patients with multi-drug resistant leukemia are refractory to chemotherapy and have accumulated organ toxicities
New, well tolerated and effective agents are urgently needed
Clofarabine NDAClofarabine NDA
Steve Weitman, MD, PhDSteve Weitman, MD, PhD
Development OverviewClinical Study ResultsDevelopment OverviewClinical Study Results
ILEX Oncology, Inc.ILEX Oncology, Inc.
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Clofarabine Development OverviewClofarabine Development Overview
Adult Phase I studies started in 1999
Pediatric Phase I studies started in 2000Striking activity in heavily pretreated population with acceptable toxicity profile
Compelling results in pediatric patients with an unmet medical need
Propelled development in pediatrics at a faster rate than adults
Increased the request for expanded access due to lack of alternative studies
Adult Phase I studies started in 1999
Pediatric Phase I studies started in 2000Striking activity in heavily pretreated population with acceptable toxicity profile
Compelling results in pediatric patients with an unmet medical need
Propelled development in pediatrics at a faster rate than adults
Increased the request for expanded access due to lack of alternative studies
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Clofarabine Development TimelineClofarabine Development Timeline
Adult StudiesAdult
Studies
Pediatric Studies
Pediatric Studies
Phase ILeukemia
Phase I/ IICLO/ ARA-CAML, MDS
Phase IIAML
Phase IIAML, ALL, MDS, CML
Phase II/ IIICLO/ ARA-C
AMLIn Development
1999199919991999
2000200020002000
2001200120012001
2002200220022002
2003200320032003
2004200420042004
20052005
Phase IIAML
Phase ILeukemia Phase II
ALLCOG Phase II CLO/ ARA-C
AML, ALLIn Development
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Phase I Pediatric ALL & AML Phase I Pediatric ALL & AML
Twenty-five patients
Dose levels from 11-70 mg/m2/day x 5
MTD 52 mg/m2/day IV
DLT was reversible increases in bilirubin and skin rash
ResponseCR 5/25 (20%)PR 3/25 (12%)
7 of 25 patients went to transplant
Twenty-five patients
Dose levels from 11-70 mg/m2/day x 5
MTD 52 mg/m2/day IV
DLT was reversible increases in bilirubin and skin rash
ResponseCR 5/25 (20%)PR 3/25 (12%)
7 of 25 patients went to transplant Jeha, et. al. Blood. 2004;103:784-789
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Pivotal Pediatric ALL & AML Pivotal Pediatric ALL & AML
Planned Study DesignPrimary Endpoint is Overall Response Rate (CR+CRp)
Fleming 2 stage design - (40% response rate of interest was based on 1 to 2 prior regimens)
Current Clinical PracticePopulation was heavily pretreated, having highly resistant leukemia (up to 6 prior regimens)
Patients went to transplant before count recovery
The study size was expanded in collaboration with FDA to understand the true response rate
Planned Study DesignPrimary Endpoint is Overall Response Rate (CR+CRp)
Fleming 2 stage design - (40% response rate of interest was based on 1 to 2 prior regimens)
Current Clinical PracticePopulation was heavily pretreated, having highly resistant leukemia (up to 6 prior regimens)
Patients went to transplant before count recovery
The study size was expanded in collaboration with FDA to understand the true response rate
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0
2
4
6
8
10
12
14
16
18
20
1 3 5 7 9 11 13 15 17 19 21 23 25 27 29 31 33 35 37 39 41 43 45 48
Pivotal Pediatric ALL Number of Unique Prior AgentsPivotal Pediatric ALL Number of Unique Prior Agents
Num
ber
of A
gent
s
Patients
Chemo Drugs Transplant TBI
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Pivotal Pediatric AMLNumber of Unique Prior AgentsPivotal Pediatric AMLNumber of Unique Prior Agents
Num
ber
of A
gent
s
Patients
Chemo Drugs Transplant TBI
20042003200220012000
Post-Induction RegimenPost-Induction RegimenL-Asparaginase, Vincristine, CytarabineCyclophosphamide, Etoposide, Thioguanine
Transplant Prep RegimenTransplant Prep RegimenTBI, Thiotepa, Fludarabine Peripheral Blood SCT
4 Year Old AML Patient4 Year Old
AML Patient
Alivewith
NED
Induction Regimen 3Induction Regimen 3L-AsparaginaseCytarabine
Induction Regimen 4Induction Regimen 4Gemcitabine, TopotecanThiotepa, Vinorelbine
Induction Regimen 5Induction Regimen 5CytarabineIdarubicin
Induction Regimen 2Induction Regimen 2Etoposide , Daunorubicin, Cytarabine, Thioguanine
Clofarabine Cycle 1 Clofarabine Cycle 2 Clofarabine Cycle 3 Clofarabine Cycle 4 Clofarabine Cycle 5 Off Study Bone Marrow Transplant
ClofarabineClofarabine
Induction Regimen 1Induction Regimen 1Prednisone, Vincristine, Methotrexate
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NDA Efficacy/ Safety PopulationNDA Efficacy/ Safety Population
Efficacy data base N=84Efficacy data base N=84
Integrated safety data base N=113Integrated safety data base N=113
ALL AML
Pivotal Studies
49 35
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Pivotal Study EndpointsPivotal Study Endpoints
Key Efficacy Endpoints: Independent Response Review Panel (IRRP) determined response: CR, CRp, PR
Duration of remission
Post-treatment transplants
Survival
Safety
Key Efficacy Endpoints: Independent Response Review Panel (IRRP) determined response: CR, CRp, PR
Duration of remission
Post-treatment transplants
Survival
Safety
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Pivotal Pediatric ALL Characteristics of Study GroupPivotal Pediatric ALL Characteristics of Study Group
Number of Patients 49
Median Age in Years 12 (1 - 20)
Median # Prior Regimens 3 (2 - 6)
Patients Refractory to Most Recent Regimen 30 (61%)
Patients with Prior Transplants 15 (31%)
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Pivotal Pediatric ALLIRRP Determined Best Objective ResponsePivotal Pediatric ALLIRRP Determined Best Objective Response
Number (%) 95% CI
Overall response (CR+CRp) (N=49)
10 (20%) 10 - 34%
Patients who achieved at least PR (N=49)
15 (31%) 18 - 45%
Patients who were refractory to most recent regimen (CR+CRp) (N=30)
5 (17%) 6 - 35%
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0
25
50
75
100
0 5 10 15 20 25 30
Duration (Weeks)
Patie
nts
in R
emis
sion
(%)
Pivotal Pediatric ALLDuration of RemissionPivotal Pediatric ALLDuration of Remission
CR + CRp(N=10)Median 20.2 wks
CR + CRp(N=10)Median 20.2 wks
CR + CRp + PR(N=15)Median 9.7 wks
CR + CRp + PR(N=15)Median 9.7 wks
Censored Value
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Pivotal Pediatric ALLPost-Clofarabine TransplantsPivotal Pediatric ALLPost-Clofarabine Transplants
14% of patients went to transplant (7/49)
2 CR, 2 CRp, 2 PR, 1 NE
Median time to transplant was 32 days (range 16 - 77)
Median number cycles was 2 (range 2 - 3)
5 of 7 patients alive post-transplant
14% of patients went to transplant (7/49)
2 CR, 2 CRp, 2 PR, 1 NE
Median time to transplant was 32 days (range 16 - 77)
Median number cycles was 2 (range 2 - 3)
5 of 7 patients alive post-transplant
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0
25
50
75
100
0 10 20 30 40 50 60 70
Survival (Weeks)
Patie
nt S
urvi
val (
%)
Pivotal Pediatric ALLOverall SurvivalPivotal Pediatric ALLOverall Survival
CR + CRp(N=10)Median 58.6 wks
CR + CRp(N=10)Median 58.6 wks
CR + CRp + PR(N=15)Median 42 wks
CR + CRp + PR(N=15)Median 42 wks
All Pts(N=49)Median 11.7 wks
All Pts(N=49)Median 11.7 wks
Censored Value
30
Pivotal Pediatric AML Characteristics of Study GroupPivotal Pediatric AML Characteristics of Study Group
Number of Patients 35
Median Age in Years 12 (2 - 22)
Median # Prior Regimens 3 (1 - 5)
Patients Refractory to Most Recent Regimen 22 (63%)
Patients with Prior Transplants 18 (51%)
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Pivotal Pediatric AML IRRP Determined Best Objective ResponsePivotal Pediatric AML IRRP Determined Best Objective Response
Number (%) 95% CI
Overall response(CR+CRp) (N=35)
1 (3%) 0 - 15%
Patients who achieved at least PR(N=35)
9 (26%) 12 - 43%
Patients who were refractory to most recent regimen (CR+CRp, PR) (N=22)
4 (18%) 5 - 40%
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0
25
50
75
100
0 10 20 30 40 50 60 70 80
Duration (Weeks)
Patie
nts
in R
emis
sion
(%)
Pivotal Pediatric AMLDuration of RemissionPivotal Pediatric AMLDuration of Remission
CR + CRp + PR(N=9)Median 16.2 wks
CR + CRp + PR(N=9)Median 16.2 wks
Censored Value
33
Pivotal Pediatric AMLPost-Clofarabine TransplantsPivotal Pediatric AMLPost-Clofarabine Transplants
34% of patients went to transplant (12/35)
1 CRp, 6 PR, 3 NE, 2 TF
Median time to transplant was 38 days (range 21 - 75)
Median number of cycles was 2 (range 1 - 5)
7 of 12 patients alive post-transplant
34% of patients went to transplant (12/35)
1 CRp, 6 PR, 3 NE, 2 TF
Median time to transplant was 38 days (range 21 - 75)
Median number of cycles was 2 (range 1 - 5)
7 of 12 patients alive post-transplant
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0
25
50
75
100
0 10 20 30 40 50 60 70 80 90 100
Survival (Weeks)
Patie
nt S
urvi
val (
%)
Pivotal Pediatric AMLOverall SurvivalPivotal Pediatric AMLOverall Survival
CR + CRp + PR(N=9)Median 39 wks
CR + CRp + PR(N=9)Median 39 wks
All Pts(N=35)Median 21 wks
All Pts(N=35)Median 21 wks
Censored Value
35
Pivotal ALL & AML Efficacy Summary Pivotal ALL & AML Efficacy Summary
Recurrent pediatric acute leukemia is a substantial unmet medical need, especially for patients with AML
Impressive response rates with Clofarabine for pediatric patients with ALL and AML that has become cross-resistant to most currently available agents
The duration of remission was more than sufficient to allow patients with donors the opportunity to proceed to transplant
Long-term survival was observed in patients with ALL and AML who responded to Clofarabine
Recurrent pediatric acute leukemia is a substantial unmet medical need, especially for patients with AML
Impressive response rates with Clofarabine for pediatric patients with ALL and AML that has become cross-resistant to most currently available agents
The duration of remission was more than sufficient to allow patients with donors the opportunity to proceed to transplant
Long-term survival was observed in patients with ALL and AML who responded to Clofarabine
37
All Grade 3 & 4 Adverse Events in > 10% of Pediatric Patients (N=113)
Integrated SafetyIntegrated Safety
54.0%
15.0% 15.0%6.2% 4.4%10.6%14.2%
6.2%7.1%7.1%0.9%
4.4%
0%
25%
50%
75%
100%
FebrileNeutropenia
Nausea Pyrexia Epistaxis HypotensionNOS
Sepsis Anorexia
Grade 3 Grade 4
38
Drug-Related Grade 3 & 4 Adverse Events in > 5% of Pediatric Patients (N=113)
Integrated SafetyIntegrated Safety
8.0%28.3%
8.8% 5.3%6.2%2.7%8.0%0.9%6.2%
0.9%
0%
25%
50%
75%
100%
FebrileNeutropenia
Nausea Pyrexia DiarrheaNOS
Neutropenia VomitingNOS
DermatitisNOS
Grade 3 Grade 4
39
All Grade 3 & 4 Hepato-Biliary/ Renal Lab AbnormalitiesAll Grade 3 & 4 Hepato-Biliary/ Renal Lab Abnormalities
Grade 3 Grade 4
37.3% 33.3%
11.6% 3.5% 1.0%1.8%3.6%
6.4%4.8%
0%
25%
50%
75%
100%
Elevated SGPT(ALT)
Elevated SGOT(AST)
Elevated TotalBilirubin
ElevatedCreatinine
Elevated AlkalinePhosphatase
40
Deaths During StudyDeaths During Study
ALL (N=67)
AML (N=46)
Disease Progression 7 (10%) 4 (9%)
Non Drug-related AE 6 (9%) 5 (11%)
Drug-related AE 3 (4%) 1 (2%)
Includes deaths within 30 days of last dose of study drug
41
Integrated Safety SummaryIntegrated Safety Summary
Heavily pretreated population
Many adverse events were consistent with underlying leukemia
Events are not unexpected for a cytotoxic agent
Most adverse events were reversible
Heavily pretreated population
Many adverse events were consistent with underlying leukemia
Events are not unexpected for a cytotoxic agent
Most adverse events were reversible
Stephen Sallan, MDStephen Sallan, MD
Clinician’s PerspectiveClinician’s Perspective
Dana-Farber Cancer Institute
43
Childhood Leukemia Childhood Leukemia
ALL 75 - 80% cured with multi-drug chemotherapy
AML 40 - 50% are cured
Successful treatment of relapsed and refractory pediatric leukemias remains a major challenge
ALL 75 - 80% cured with multi-drug chemotherapy
AML 40 - 50% are cured
Successful treatment of relapsed and refractory pediatric leukemias remains a major challenge
44
Survival in Pediatric LeukemiasSurvival in Pediatric Leukemias
0
20
40
60
80
100
1950 1960 1970 1980 1990 2000Year
Fiv
e Y
ear
Rel
ativ
e S
urvi
val R
ates
Kersey, Blood. 1997; 90(11):4243-4251
ALLALL
AMLAML
45
CR After Single Agents in Childhood ALLCR After Single Agents in Childhood ALLAdapted from Holland and Frei Cancer Medicine 1974
De Novo ALL CR
6-Mercaptopurine 25%
Methotrexate 20%
Cytarabine 30%
Multi-drug Resistant ALL CR+CRp
Clofarabine 20%
46
What Impresses Me As A ClinicianWhat Impresses Me As A Clinician
ALL Clofarabine results
1 in 5 children with multi-drug resistant ALL achieved a CR, affording them a transplant option
AML Clofarabine results
1 in 3 children with multi-drug resistant AML went to transplant
Transplant is the curative therapeutic option in drug-resistant childhood ALL and AML
ALL Clofarabine results
1 in 5 children with multi-drug resistant ALL achieved a CR, affording them a transplant option
AML Clofarabine results
1 in 3 children with multi-drug resistant AML went to transplant
Transplant is the curative therapeutic option in drug-resistant childhood ALL and AML
47
What Impresses Me As A ClinicianWhat Impresses Me As A Clinician
Clofarabine is Well Tolerated
No overlapping toxicities
Clofarabine provides Clinical Benefit
In a heterogeneous population
No meaningful Alternatives
Clofarabine is Well Tolerated
No overlapping toxicities
Clofarabine provides Clinical Benefit
In a heterogeneous population
No meaningful Alternatives
Commitment to Pediatric Clinical DevelopmentCommitment to Pediatric Clinical Development
Steve Weitman, MD, PhDSteve Weitman, MD, PhD
ILEX Oncology, Inc.ILEX Oncology, Inc.
49
Commitment to Pediatric Clinical DevelopmentCommitment to Pediatric Clinical Development
This represents a new paradigm in pediatric drug developmentThe sponsor commits to further develop this drug in children:
Continue survival follow-up on pivotal studiesMoving to less heavily pretreated ALL and AML patientsProceed to a randomized study in pediatric patientsOur commitment includes working closely with the Children’s Oncology Group and CTEP
This represents a new paradigm in pediatric drug developmentThe sponsor commits to further develop this drug in children:
Continue survival follow-up on pivotal studiesMoving to less heavily pretreated ALL and AML patientsProceed to a randomized study in pediatric patientsOur commitment includes working closely with the Children’s Oncology Group and CTEP
50
Commitment to Pediatric Clinical DevelopmentCommitment to Pediatric Clinical Development
Children’s Oncology Group (COG)/ CTEPAML Ara-C/Clofarabine Combination Study
First relapsed patients
Study chairs: Razzouk and Cooper
ALL Cytoxan/Clofarabine and Etoposide/Clofarabine Combination Study
– Second relapsed patients– Study chair: Hijiya
Children’s Oncology Group (COG)/ CTEPAML Ara-C/Clofarabine Combination Study
First relapsed patients
Study chairs: Razzouk and Cooper
ALL Cytoxan/Clofarabine and Etoposide/Clofarabine Combination Study
– Second relapsed patients– Study chair: Hijiya
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Commitment toAdult Clinical Development ProgramCommitment toAdult Clinical Development Program
ObjectivesFocused on AML and MDSCombination studies with Ara-CInterest in elderly population of patients
Adult Leukemia StudiesCLO-141 Phase I/II Combination Ara-C/Clofarabine in AMLRandomized studies planned
Phase II/III Ara-C/Clofarabine in elderly patients with De Novo AMLPhase II/III Ara-C/Clofarabine in non-elderly adult patients with relapsed AML
Adult Development Discussion with FDA Ongoing
ObjectivesFocused on AML and MDSCombination studies with Ara-CInterest in elderly population of patients
Adult Leukemia StudiesCLO-141 Phase I/II Combination Ara-C/Clofarabine in AMLRandomized studies planned
Phase II/III Ara-C/Clofarabine in elderly patients with De Novo AMLPhase II/III Ara-C/Clofarabine in non-elderly adult patients with relapsed AML
Adult Development Discussion with FDA Ongoing
52
Overall Risk/ Benefit of Clofarabine inPediatric LeukemiaOverall Risk/ Benefit of Clofarabine inPediatric Leukemia
Acceptable safety profile in this heavily pre-treated patient populationImpressive benefits
Provided meaningful clinical response and cytoreduction (CR, CRp, and PR) Overall, 23% of patients proceeded to transplant (19/84)
14% of patients with ALL34% of patients with AML
Patients alive at data cutoff - ALL 22%, AML 26% (Median follow-up 29 weeks)
Meets an urgent unmet medical need in highly resistant pediatric leukemia
Acceptable safety profile in this heavily pre-treated patient populationImpressive benefits
Provided meaningful clinical response and cytoreduction (CR, CRp, and PR) Overall, 23% of patients proceeded to transplant (19/84)
14% of patients with ALL34% of patients with AML
Patients alive at data cutoff - ALL 22%, AML 26% (Median follow-up 29 weeks)
Meets an urgent unmet medical need in highly resistant pediatric leukemia