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DRUG CLASS:

IRAK-1 inhibitor

14d
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
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) (clinicaltrials.gov)
P2, N=78, Recruiting, Swedish Orphan Biovitrum | Active, not recruiting --> Recruiting
Enrollment open
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Vonjo (pacritinib)
2ms
Enrollment open
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azacitidine • Inqovi (decitabine/cedazuridine) • Vonjo (pacritinib)
3ms
MDS: Pacritinib With Aza for Upfront Myelodysplastic Syndrome (clinicaltrials.gov)
P1/2, N=25, Not yet recruiting, Thomas Jefferson University
New P1/2 trial
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azacitidine • Vonjo (pacritinib)
4ms
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) (clinicaltrials.gov)
P2, N=78, Active, not recruiting, Swedish Orphan Biovitrum | Trial completion date: Aug 2027 --> May 2028 | Trial primary completion date: Aug 2027 --> Dec 2026
Trial completion date • Trial primary completion date
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Vonjo (pacritinib)
4ms
A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS) (clinicaltrials.gov)
P2, N=78, Active, not recruiting, Swedish Orphan Biovitrum | Recruiting --> Active, not recruiting
Enrollment closed
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Vonjo (pacritinib)
5ms
Masking as Myelofibrosis: A Case of Tp53 Mutated Acute Erythroid Leukemia Presenting With Pancytopenia and Bone Pain. (PubMed, Case Rep Hematol)
He was started on JAK inhibitor therapy with pacritinib but clinically declined over the next several days with worsening diffuse pain and pancytopenia...Shortly after, the patient presented to an outside hospital with septic shock, at which point the patient expired. This case illustrates the aggressive nature of the disease, the need for confirmatory testing when diagnosis is suspected and the difficulty in management as the prognosis is poor and requires aggressive treatment that can lead to life-threatening sequelae.
Journal
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TP53 (Tumor protein P53) • JAK2 (Janus kinase 2)
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TP53 mutation
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Vonjo (pacritinib)
6ms
Real-world experience with pacritinib for patients with myelofibrosis refractory to ruxolitinib: a report of three cases. (PubMed, Hosp Pract (1995))
Adverse events, including gastrointestinal symptoms, weight loss, and transient voice changes, were manageable through dose adjustments and supportive care, enabling continued therapy. Our cases contribute to the growing body of evidence supporting pacritinib's role in the evolving treatment landscape of MF.
Journal • Real-world evidence
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JAK2 (Janus kinase 2) • JAK1 (Janus Kinase 1) • IRAK1 (Interleukin 1 Receptor Associated Kinase 1)
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Jakafi (ruxolitinib) • Vonjo (pacritinib)
6ms
Interleukin-1 receptor-associated kinase-1 is a therapeutic target for gastric cancer. (PubMed, Discov Oncol)
Notably, we found that gastric cancer cells display marked sensitivity to IRAK1/4 inhibitor and pacritinib, the latter targeting JAK2 and IRAK1 specifically, over the pan-JAK inhibitor tofacitinib. Collectively, our results underscore IRAK1 as a promising therapeutic target in gastric cancer. Furthermore, the pharmacological blockade of IRAK1 by pacritinib, an established drug for myelofibrosis and severe thrombocytopenia treatment, holds potential for repurposing in gastric cancer therapy.
Journal
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JAK2 (Janus kinase 2) • IRAK1 (Interleukin 1 Receptor Associated Kinase 1)
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Vonjo (pacritinib) • tofacitinib