Vonjo (pacritinib)

P2, N=78, Recruiting, Swedish Orphan Biovitrum | Active, not recruiting --> Recruiting

P2, N=27, Recruiting, University of Washington | Not yet recruiting --> Recruiting

P2, N=58, Recruiting, Karyopharm Therapeutics Inc | N=118 --> 58 | Trial primary completion date: Apr 2026 --> Jun 2027

P1/2, N=25, Not yet recruiting, Thomas Jefferson University

P2, N=78, Active, not recruiting, Swedish Orphan Biovitrum | Trial completion date: Aug 2027 --> May 2028 | Trial primary completion date: Aug 2027 --> Dec 2026

Equally, there is a major focus on next generation JAK inhibitors and mutant calreticulin antibodies. There is also increasing conversation around the need for novel endpoints, as the limitations of symptom assessment, in particular, become apparent and candidate biomarkers of disease modification emerge.

P2, N=78, Active, not recruiting, Swedish Orphan Biovitrum | Recruiting --> Active, not recruiting

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.

P2, N=32, Not yet recruiting, Medical College of Wisconsin

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.

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.