Best available therapy

Phase 3Small molecule

Novartis

Best available therapy is one of 244 small molecule programs targeting Polycythemia Vera. It is currently in Phase 3.

Indications

Total Trials

Active Trials

243

Competitors

Pipeline

Status by Indication[3][1]

Indication

Polycythemia VeraActive, not recruiting

ThrombocytopeniaActive, not recruiting

MortalityActive, not recruiting

Acute Graft Vs. Host DiseaseActive, not recruiting

Chronic Graft Versus Host DiseaseActive, not recruiting

MyelofibrosisActive, not recruiting

Multiple SclerosisActive, not recruiting

Enterobacteriaceae InfectionsActive, not recruiting

Essential ThrombocythemiaActive, not recruiting

Polycythemia VeraPhase 3

Active, not recruiting

ThrombocytopeniaPhase 3

Active, not recruiting

MortalityPreclinical

Active, not recruiting

Acute Graft Vs. Host DiseasePhase 3

Active, not recruiting

Chronic Graft Versus Host DiseasePhase 3

Active, not recruiting

MyelofibrosisPhase 3

Active, not recruiting

Multiple SclerosisPhase 3

Active, not recruiting

Enterobacteriaceae InfectionsPhase 3

Active, not recruiting

Essential ThrombocythemiaPhase 3

Active, not recruiting

Competition

Competitive Landscape

560 competitors in Multiple Sclerosis

#DrugCompanyPhaseTrials

Best available therapy

Novartis

Phase 3

1 2

Kesimpta

Novartis

Preclinical

15 3

Avonex

EMD Serono

Preclinical

22 4

Campath

GGenzyme, a Sanofi Company

Preclinical

12 5

Flomax

Sanofi

Preclinical

1 6

Novantrone

Pred Forte

Myfortic

Circadin

NNeurim Pharmaceuticals Ltd.

Preclinical

3 10

Wellcovorin

GGlaxo Dba Glaxosmithkline

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 3Active, not recruiting

Myelofibrosis

NCT04468984

Q2 2027

Phase 3Recruiting

Enterobacteriaceae Infections

NCT07089186

Q2 2028

Phase 3Active, not recruiting

Myelofibrosis

NCT04576156

Q3 2028

Phase 2Not yet recruiting

Acinetobacter Baumannii

NCT07431307

Clinical

Trials by Indication[1]

Myelofibrosis6 trials

Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis

Terminated· NS Pharma logo

NS Pharma·Phase 2·NCT04854096·7 enrolled

Pacritinib Versus Best Available Therapy to Treat Myelofibrosis

Terminated·CCTI BioPharma·Phase 3·NCT01773187·327 enrolled

KRT-232 Versus Best Available Therapy for the Treatment of Subjects With Myelofibrosis Who Are Relapsed or Refractory to JAK Inhibitor Treatment

Unknown status·KKartos Therapeutics, Inc.·Phase 2/3·NCT03662126·385 enrolled

Show 3 more trials

Study of Oral Navitoclax Tablet in Combination With Oral Ruxolitinib Tablet Versus Best Available Therapy to Assess Change in Spleen Volume in Adult Participants With Relapsed/Refractory Myelofibrosis

Active, not recruiting· AbbVie logo

AbbVie·Phase 3·NCT04468984·330 enrolled

Controlled Myelofibrosis Study With Oral Janus-associated Kinase (JAK) Inhibitor Treatment-II: The COMFORT-II Trial

Completed· Novartis logo

Novartis·Phase 3·NCT00934544·219 enrolled

A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients With Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK) Inhibitor

Active, not recruiting·GGeron Corporation·Phase 3·NCT04576156·327 enrolled

Infections2 trials

Ceftazidime-Avibactam for the Treatment of Infections Due to Ceftazidime Resistant Pathogens

Completed· Pfizer logo

Pfizer·Phase 3·NCT01644643·345 enrolled

Efficacy, Safety, Tolerability of Vabomere Compared to Best Available Therapy in Treating Serious Infections in Adults

Completed·MMelinta Therapeutics, Inc.·Phase 3·NCT02168946·77 enrolled

Polycythemia Vera2 trials

AOP2014 vs. BAT in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study.

Completed·AAOP Orphan Pharmaceuticals AG·Phase 3·NCT02218047·170 enrolled

Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.

Completed· Novartis logo

Novartis·Phase 3·NCT02038036·149 enrolled

Essential Thrombocythemia2 trials

An Efficacy and Safety Study of Fedratinib Compared to Best Available Therapy in Subjects With DIPSS-intermediate or High-risk Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis and Previously Treated With Ruxolitinib

Completed· Bristol-Myers Squibb logo

Bristol-Myers Squibb·Phase 3·NCT03952039·202 enrolled

Efficacy of Momelotinib Versus Best Available Therapy in Anemic or Thrombocytopenic Subjects With Primary Myelofibrosis (MF), Post-polycythemia Vera MF, or Post-essential Thrombocythemia MF

Completed·—·Phase 3·NCT02101268·156 enrolled

Treatment1 trial

Calcifediol in the Treatment of COVID 19

Completed·MMaimónides Biomedical Research Institute of Córdoba··NCT05819918·728 enrolled

8 more indications

Severe Infection1 trial

Study of Cefiderocol (S-649266) or Best Available Therapy for the Treatment of Severe Infections Caused by Carbapenem-resistant Gram-negative Pathogens

Completed· Shionogi logo

Shionogi·Phase 3·NCT02714595·152 enrolled

Chronic Graft Versus Host Disease1 trial

A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy

Active, not recruiting· Incyte logo

Incyte·Phase 3·NCT06821542·9 enrolled

Acute Graft Vs. Host Disease1 trial

Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs. Host Disease After Allogeneic Stem Cell Transplantation

Completed· Novartis logo

Novartis·Phase 3·NCT02913261·310 enrolled

Multiple Sclerosis1 trial

Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)

Recruiting·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 3·NCT04047628·156 enrolled

Acinetobacter Baumannii1 trial

Safety, Pharmacokinetics and Efficacy of BV100 Plus Low Dose Polymyxin B Plus Ceftazidime/Avibactam, or Plus Cefiderocol in Patients With Pulmonary and Extrapulmonary Infections Due to Carbapenem-resistant Acinetobacter Baumannii-calcoaceticus Complex

Not yet recruiting·BBioVersys AG·Phase 2·NCT07431307·120 enrolled

Mortality1 trial

Impact of Ceftazidime / Avibactam Treatment vs Better Available Therapy on Mortality of Patients With Infections Caused by Carbapenem-resistant Enterobacteria

Completed·MMaimónides Biomedical Research Institute of Córdoba··NCT04167228·348 enrolled

Enterobacteriaceae Infections1 trial

Study to Assess the Efficacy and Safety of Meropenem and Pralurbactam in Carbapenem-Resistant Enterobacteriaceae Infections

Recruiting· Qilu Pharmaceutical Co., Ltd. logo

Qilu Pharmaceutical Co., Ltd.·Phase 3·NCT07089186·80 enrolled

Thrombocytopenia1 trial

Pacritinib Versus Best Available Therapy to Treat Patients With Myelofibrosis and Thrombocytopenia

Terminated·CCTI BioPharma·Phase 3·NCT02055781·311 enrolled

Sources

References

[1]
ClinicalTrials.gov — 21 trial records for Best available therapy. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Best available therapy compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Best available therapy. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Novartis company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: Novartis

Best available therapy

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q2 2027

Q2 2028

Q3 2028

Trials by Indication[1]

Myelofibrosis6 trials

Infections2 trials

Polycythemia Vera2 trials

Essential Thrombocythemia2 trials

Treatment1 trial

Severe Infection1 trial

Chronic Graft Versus Host Disease1 trial

Acute Graft Vs. Host Disease1 trial

Multiple Sclerosis1 trial

Acinetobacter Baumannii1 trial

Mortality1 trial

Enterobacteriaceae Infections1 trial

Thrombocytopenia1 trial

References

Drug Info

Quick Facts

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