Roivant Sciences, a company that acquired neglected or shelved drug assets from major pharmaceutical companies and formed subsidiaries to develop them, has launched its newest division, a biotechnology that aims to translate Eisai molecules into treatments for rare diseases caused by rare diseases. New treatments for types of anemia. blood cancer. Roivant said the new assets from its new subsidiary Hemavant have potential advantages over the Bristol Myers Squibb drug currently being marketed for the indication.
Hemavant’s drug is being developed to treat patients with myelodysplastic syndrome (MDS), a cancer in which the bone marrow produces abnormal blood cells. The resulting low red blood cell levels lead to anemia, making the patient dependent on blood transfusions. The disease is thought to be caused by mutations that lead to altered mRNA splicing, Roivant said in an article. Investor introduction.
First-line treatment of MDS includes drugs that stimulate red blood cell production. But these treatments don’t work for 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 sells Acceleron Pharma drug Rebrozyl, which is Approved in 2020 For the treatment of low-to-moderate risk MDS unresponsive to first-line therapy. In his presentation, Roivant noted that the two BMS drugs are only approved for a specific subset of patients with MDS, and they carry a dangerous risk of side effects.
Eisai has advanced its molecule H3B-8800 (now renamed RVT-2001 by Roivant) into a Phase 1 study enrolling 84 patients, including high-risk and low-risk MDS cases.result post The 2019 study showed that while the drug was safe and well tolerated, no patients responded to the treatment. However, decreased red blood cell or platelet transfusions were observed in 12 patients.
Roivant focused on 19 low-risk, transfusion-dependent patients in the Eisai study who had been previously treated with Revlimid or other drugs to control gene expression. Of the 19 patients, Roivant said RVT-2001 resulted in a red blood cell transfusion independence rate of more than 30 percent. In the Phase 2 trial of Rebrozyl, transfusion independence was observed in 13% of patients; for Revlimid, improvement in red blood cell measurements was observed in 12% of patients.
It is important to note here that these are small studies and there are no head-to-head tests comparing RVT-2001 to Rebrozyl or Revlimid. Nonetheless, Roivant believes that the Eisai drug has the potential to provide better treatment options for low-risk MDS patients who do not respond to first-line therapy. Last month, Roivant licensed Global rights to Eisai Molecule; financial terms not disclosed.
RVT-2001 is a small molecule designed to target SF3B1, a gene that is mutated in certain subsets of MDS patients. In laboratory and preclinical studies, Roivant said the drug corrected a splicing defect caused by mutations in SF3B1 in the mRNA instruction that encodes a protein thought to be involved in the development of MDS.
By focusing on patients whose disease is defined by the SF3B1 gene, Roivant believes it can achieve better clinical trial results than Eisai. An open-label expansion of the Phase 1/2 clinical trial is planned, but this study will selectively enroll patients with the mutation. These patients account for about 30 percent of MDS cases, according to Roivant. The expansion of the Phase 1/2 study is scheduled to begin in the first half of this year; the company said it expects to release preliminary data in 2023.
The unveiling of the Hemavant took place in Roivant Report its financial results for the third quarter of fiscal 2021. Roivant went public last year in a merger with a special purpose acquisition companyAt the time, company executives explained how the biotech’s business model goes beyond acquiring drug assets and forming subsidiaries (which it calls “vants”) to fuel their growth. Roivant’s computational drug discovery efforts are also producing internally developed drug assets.
“RVT-2001 is Hemavant’s lead product candidate and is a great example of our pipeline expansion plans, which will come from licensing and our internal computational discovery work,” Roivant CEO Matt Glenn said in announcing the financial results.
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