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Table 1 Selected studies on the novel single agent targeted therapeutic on myelofibrosis from the 2022 ASH annual meeting

From: Novel agents and evolving strategies in myelofibrotive neoplasm: an update from 2022 ASH annual conference

Name

Target

Route

Trial phase

Inclusion criteria (prior treatments; subject age; spleen size)

Subjects Baseline Characteristics

Study duration (current /planned)

Accrual

(current accrual #/target accrual #)

Efficacy

Adverse Events

Clinicaltrials.gov Registration

References

MMB

JAK1/2/ACVR1

PO

III

One prior JAKi for ≥ 90 days; ≥ 18 years; palpable spleen ≥ 5 cm

N/A

27/96 months

195/195

TI-R (31%); SVR (100%) at W24

Thrombocytopenia

Anemia

Infection

Peripheral neuropathy

NCT04173494

[3]

Pacritinib

JAK2/ACVR1

PO

III

JAKi-naïve patients; ≥ 18 years; palpable spleen ≥ 5 cm

All patients had platelet counts ≤ 100 × 109/L

40/40 months

327/327

TL-R (24% on SIMPLIFY criteria, 37% on Gale criteria) at W24

Not reported

NCT01773187

[4]

TP-3654

PIM1

PO

I/II

At least one prior JAKi; ≥ 18 years; palpable spleen ≥ 5 cm or SV ≥ 450 cm3

Median age of 70 years; median spleen volume of 2370 cm3

32/55 months

8/60

SVR (83%); TSS50 (66%); at W12

Nausea

Vomiting

Diarrhea

NCT04176198

[5]

IMG-7289

LSD1

PO

II

Prior JAKi or JAKi-naïve patients; ≥ 18 years;

Median age of 68 years; median spleen volume of 1354 cm3

56/56 months

89/89

SVR (66%); TSS50 (19%); TI-R (90%); BMF (53%) at W24

Dysgeusia

Diarrhea

NCT03136185

[6]