SINO BIOPHARM (01177) announced that the group's self-developed Class 1 innovative drug, Rovaxicitinib tablets (brand name: Anxu®), has received market approval from the National Medical Products Administration (NMPA) of China. The drug is indicated for the first-line treatment of adult patients with Intermediate-2 or high-risk primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF).
Rovaxicitinib is a globally first-in-class small molecule inhibitor targeting both JAK and ROCK. It achieves dual therapeutic effects of anti-inflammation and anti-fibrosis through the synergistic action of the JAK/ROCK dual pathways. On one hand, by inhibiting the JAK1/2–STAT3/5 signaling pathway, it reduces the high levels of inflammatory cytokines produced by myeloid cells, exerting an anti-inflammatory effect and improving splenomegaly and systemic symptoms. On the other hand, by inhibiting ROCK1/2, it lowers the polarization level of helper T cells and the burden of pro-inflammatory cytokines in myelofibrosis patients, further enhancing the anti-inflammatory effect and providing support for long-term disease control.
In a multicenter, randomized, double-blind, double-dummy, active-controlled Phase II clinical study (TQ05105-II-01), Rovaxicitinib demonstrated superior efficacy and a favorable safety profile compared to hydroxyurea in treating Intermediate-2 and high-risk myelofibrosis patients. The study enrolled a total of 107 patients, who were randomized in a 2:1 ratio to receive either Rovaxicitinib 15mg or hydroxyurea 0.5g, administered orally twice daily.
In terms of efficacy, the proportion of subjects in the Rovaxicitinib group achieving a ≥35% reduction in spleen volume from baseline (SVR35) at week 24, as assessed by an Independent Review Committee (IRC), was 58.33%. The proportion of subjects achieving SVR35 at any time point reached 63.89%, with a mean SVR35 duration of 8.31 months. The rate of achieving a ≥50% improvement in the Total Symptom Score (TSS50) was as high as 77.78%.
Regarding safety, Rovaxicitinib was generally well-tolerated. In the relevant study, the incidence of Grade ≥3 adverse reactions was approximately 40%, the incidence of anemia was around 40%, and the treatment discontinuation rate was only 6.7%, all of which were significantly lower than those associated with ruxolitinib.
Beyond myelofibrosis, Rovaxicitinib has also shown breakthrough potential in the treatment of chronic graft-versus-host disease (cGVHD). Currently, the development of this product for cGVHD is progressing smoothly: in China, it has entered Phase III clinical trials and was included in the NMPA's Center for Drug Evaluation (CDE) Breakthrough Therapy Designation program in August 2025; in the United States, it has been approved to initiate Phase II clinical studies.