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DRUG CLASS:

ACVR1 inhibitor

8d
Trial initiation date
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STAT5A (Signal Transducer And Activator Of Transcription 5A)
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Vonjo (pacritinib)
12d
PACIFICA: A Phase 3 Study of Pacritinib in Patients With Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post-Essential Thrombocythemia Myelofibrosis (clinicaltrials.gov)
P3, N=407, Active, not recruiting, Swedish Orphan Biovitrum | Recruiting --> Active, not recruiting | Trial completion date: Jul 2027 --> Oct 2028
Enrollment closed • Trial completion date
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Jakafi (ruxolitinib) • Vonjo (pacritinib) • hydroxyurea
13d
Enrollment change
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Ojjaara (momelotinib)
18d
FALKON: A Study to Assess the Effectiveness and Safety of 2 Dosage Regimens of Oral Fidrisertib (IPN60130) for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP). (clinicaltrials.gov)
P2, N=113, Terminated, Clementia Pharmaceuticals Inc. | Trial completion date: Mar 2029 --> Mar 2026 | Completed --> Terminated | Trial primary completion date: Mar 2029 --> Mar 2026; Following the planned FALKON Part A primary analysis, the study did not meet its primary endpoint of reducing heterotopic ossification (HO) volume compared with placebo and met predefined futility criteria.
Trial completion date • Trial termination • Trial primary completion date
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ACVR1 (Activin A Receptor Type 1)
20d
A Study of Momelotinib in Participants With Low-risk Myelodysplastic Syndrome (clinicaltrials.gov)
P2, N=80, Recruiting, GlaxoSmithKline | N=59 --> 80 | Trial completion date: Mar 2029 --> Dec 2027
Enrollment change • Trial completion date
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Ojjaara (momelotinib)
22d
ODYSSEY: Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis (clinicaltrials.gov)
P2, N=68, Recruiting, GlaxoSmithKline | Trial completion date: Nov 2026 --> Mar 2028 | Trial primary completion date: Oct 2026 --> Mar 2027
Trial completion date • Trial primary completion date
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Reblozyl (luspatercept-aamt) • Ojjaara (momelotinib)
27d
Enrollment change
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Ojjaara (momelotinib)
27d
RNA dicing promotes the expression of an oncogenic JAK1 isoform. (PubMed, Cell Rep)
This results in a better response to the JAK1 inhibitor momelotinib, highlighting RNA dicing's role in patient stratification. Our findings show that RNA dicing diversifies mRNA products, significantly impacting biological functions.
Journal
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IFNG (Interferon, gamma) • JAK1 (Janus Kinase 1)
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Ojjaara (momelotinib)
1m
A pharmacological profile of pacritinib for the treatment of myelofibrosis. (PubMed, Expert Rev Clin Pharmacol)
While no JAK inhibitor has demonstrated clear disease-modifying effects in MF, pacritinib's non-myelosuppressive profile, unique activity against IRAK1, and potential anemia benefit via ACVR1 inhibition suggests potential utility as a backbone for future combination strategies. Ongoing and future studies will be critical to further define its role in phenotype-driven MF management.
Review • Journal
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JAK2 (Janus kinase 2) • JAK1 (Janus Kinase 1) • ACVR1 (Activin A Receptor Type 1) • IRAK1 (Interleukin 1 Receptor Associated Kinase 1)
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Vonjo (pacritinib)
1m
Trial completion
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ACVR1 (Activin A Receptor Type 1)