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3 Day Thiarabine

Phase 1

Leukemia | Small molecule | Oncology |Abeona Therapeutics Inc.|Last Updated: Jun 17, 2014

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDBiomarker
Total Trials1
Total Enrollment31
FDA Designations
ORPHAN_DRUG
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT011391514'-Thio-araC (Thiarabine) in Advanced Hematologic MalignanciesPHASE1 COMPLETED 31Aug 1, 2010Nov 1, 2013Jun 17, 20141 United States
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Study Endpoints
Primary Endpoints
Maximum Tolerated Dose (MTD) of 4'-thio-araC
3 weeks

The MTD is the highest dose level in which \<2 patients of six develop first cycle dose-limiting toxicity (DLT).

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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Group 1 - 3 Day ThiarabineEXPERIMENTALThiarabine 3 days in a row in each cycle.
Group 2 - 5 Day ThiarabineEXPERIMENTALThiarabine 5 days a row in each cycle.
Interventions
NameTypeDescription
3 Day ThiarabineDRUGStarting dose 70 mg/m\^2 IV over 1 hour (±15 minutes) daily x 3
5 Day ThiarabineDRUGStarting dose 40 mg/m2 IV over 1 hour (±15 minutes) daily x 5
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Eligibility Criteria
Age Range16 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Patients must have relapsed/refractory leukemias for which no standard therapies are anticipated to result in a durable remission. Patients with poor-risk myelodysplasia (MDS) \[i.e. refractory anemia with excess blasts (RAEB-1 or RAEB-2) by WHO classification\] and chronic m...

Countries:United States
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Competitive Landscape -Leukemia 332 trials
CompanyTickerTrialsLead PhaseDrugs
Amgen Inc.AMGN8PHASE3Blinatumomab, Low-intensity chemotherapy regimen, HyperCVAD, Omitted Doxorubicin, Omitted Vincristine+Dexamethasone pulses
Eli Lilly and CompanyLLY10PHASE3Pirtobrutinib, Idelalisib, Bendamustine, Rituximab, Venetoclax
AstraZeneca PLCAZN20PHASE3Acalabrutinib, Rituximab, Chlorambucil, Venetoclax, Obinutuzumab
Merck & Co., Inc.MRK7PHASE3Nemtabrutinib, Fludarabine, Cyclophosphamide, Bendamustine, Rituximab
BeOne Medicines Ltd. Sponsored ADRONC14PHASE3Sonrotoclax, Zanubrutinib, Venetoclax, Obinutuzumab, Bendamustine
AbbVie, Inc.ABBV24PHASE3Venetoclax, Acalabrutinib, Obinutuzumab, Fludarabine, Cytarabine
Novartis AG Sponsored ADRNVS21PHASE3Imatinib, Nilotinib, Bosutinib, Dasatinib, Asciminib
Johnson & JohnsonJNJ12PHASE3Ibrutinib, Venetoclax, Chlorambucil, Obinutuzumab, Bleximenib
Nurix Therapeutics, Inc.NRIX5PHASE3NX-5948, Pirtobrutinib, venetoclax, rituximab, obinutuzumab
Kura Oncology, Inc.KURA5PHASE3Ziftomenib, Venetoclax, Azacitidine, Daunorubicin, Cytarabine
Syndax Pharmaceuticals IncSNDX4PHASE3Revumenib, Intensive Chemotherapy Regimen, revumenib, cobicistat, SNDX-5613
Takeda Pharmaceutical Co. Ltd. Sponsored ADRTAK1PHASE3Ponatinib, Imatinib, Vincristine, Dexamethasone, Cytarabine
SELLAS Life Sciences Group, Inc.SLS1PHASE3Galinpepimut-S, Azacitidine, Venetoclax, Decitabine, Cytarabine
Grifols, S.A. Sponsored ADR Class BGRFS1PHASE3Xembify
Bristol-Myers Squibb CompanyBMY7PHASE2Azacitidine, CC-486, AG-120, AG-221, BMS-986497
Pfizer Inc.PFE6PHASE2Gemtuzumab ozogamicine - Cytarabine - Gilteritinib, Inotuzumab ozogamicin, ALLR3, SEA-CD70, azacitidine
Incyte CorporationINCY6PHASE2Ruxolitinib, Conditioning Regimen A, Conditioning Regimen B, Conditioning Regimen C, Conditioning Regimen D
Tango Therapeutics, Inc.TNGX7PHASE3AG-120, Azacitidine, Ivosidenib, Ivosidenib + azacitidine, BN104 monotherapy
Ascentage Pharma Group International Unsponsored ADRAAPG11PHASE3Olverembatinib, Imatinib, Lisaftoclax, Acalabrutinib, Fludarabine
Actinium PharmaceuticalsATNM2PHASE3Iomab-B, Conventional Care, 131I-apamistamab, Fludarabine, Cyclophosphamide
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