An Eisai drug that produced disappointing data in a myelodysplastic syndrome study is getting another go under newly formed Roivant Sciences subsidiary Hemavant. Roivant executives say the small molecule could be a safer and more effective alternative to the MDS therapies being marketed by Bristol Myers Squibb.

Roivant Sciences, a company that acquires overlooked or shelved drugs from big pharmaceutical companies and sets up subsidiaries to develop them, has launched its new entity, a biotech aimed at turning an Eisai molecule into a new treatment for anemia that can be caused by a rare type. of blood cancer. According to Roivant, the new active ingredient from its new subsidiary Hemavant has potential advantages over the Bristol Myers Squibb drugs that are currently marketed for this indication.

The drug Hemavant is being developed to treat patients with myelodysplastic syndrome (MDS), a type of cancer in which the bone marrow produces abnormal blood cells. The resulting low levels of red blood cells cause anemia, making patients dependent on blood transfusions. The disease is believed to be caused by mutations that cause changes in mRNA splicing, Roivant said in a presentation to investors.

The first line of treatment for MDS involves drugs that stimulate the production of red blood cells. But these therapies don't work in more than half of patients, Roivant said in the presentation. Low-risk MDS is one of the indications for Bristol Myers Squibb's cancer drug Revlimid. BMS also markets Acceleron Pharma's drug Reblozyl, which was approved in 2020 for the treatment of low-to-moderate risk MDS unresponsive to first-line treatment. Roivant noted in his presentation that both BMS drugs are only approved for certain subgroups of MDS patients and are associated with dangerous side effects.

Eisai had advanced its H3B-8800 molecule (now renamed RVT-2001 by Roivant) in a phase 1 study that enrolled 84 patients, both high- and low-risk MDS cases. The results, published in 2019, showed that while the drug was safe and well tolerated, no patients responded to treatment. However, a reduction in red blood cell or platelet transfusions was observed in 12 patients.

Roivant focused Eisai's study on a subset of 19 low-risk transfusion-dependent patients who had previously been treated with Revlimid or other drugs used to control gene expression. Roivant said that RVT-2001 resulted in a red blood cell transfusion independence rate of more than 30% in these 19 patients. In the phase 2 trials of Reblozyl, transfusion independence was observed in 13% of patients; With Revlimid, improvements in red blood cell counts were seen in 12% of patients.

The caveat here is that these are small studies and there has been no direct comparison evidence between RVT-2001 and Reblozyl or Revlimid. However, Roivant sees the potential for Eisai to offer a better treatment option for low-risk MDS patients who do not respond to first-line therapies. Last month, Roivant licensed the worldwide rights to the molecule from Eisai; Financial terms were not disclosed.

RVT-2001 is a small molecule designed to target SF3B1, a gene that is mutated in certain subgroups of MDS patients. In preclinical and laboratory research, Roivant said the drug corrected splicing defects caused by SF3B1 mutations in mRNA instructions that encode proteins that are thought to be linked to the development of MDS.