Preleukemia

Roivant knew RVT-2001 was among their "higher risk studies," a company spokesperson told Fierce Biotech. Roivant’s Hemavant has discontinued development of its only asset, a former Eisai drug dubbed RVT-2001 that was being studied in a midstage trial for patients with myelodysplastic syndromes (MDS). “Unfortunately, the data generated in that phase 1/2 study did not meet our bar for progressing,” Roivant CEO Matt Gline said on a Feb. 13 earnings call. “We spent a reasonable, modest, low double-digit million-dollar sum on the program. And I think sometimes these things don't work out the way you want scientifically. And so, we're trying to be efficient in making those decisions.” The interim data analysis comes from an open-label trial that was set to enroll a total of 200 patients and had a primary completion date of September 2024, according to ClinicalTrials.gov.   Patients enrolled had MDS, a condition in which a group of cancers occur because the bone marrow can’t produce enough healthy blood cells. Also known as preleukemia, MDS can be treated with azacitidine (sold under brand names Vidaza or Onureg) and decitabine (Dacogen), while stem cell transplants offer curative potential.  A Roivant spokesperson said the company “always knew” RVT-2001 was “among our higher risk studies.” “The existing clinical landscape set the bar for success, and it did not look to us like RVT-2001 was able to clear it,” the spokesperson explained. RVT-2001 was a small-molecule modulator of splicing factor 3B subunit 1 and the lifeblood of Hemavant. The Roivant spinout launched almost exactly two years ago, built around the asset that Roivant licensed from Eisai in late 2021.  Back in 2016, Eisai launched a phase 1 trial evaluating the oral candidate among patients with MDS and other blood cancers but saw no complete or partial responses in a 2019 data readout of the first 84 participants. Roivant still saw promise in some of the data generated by Eisai, prompting the Big Biotech to license the asset and zero in on an endpoint that suggested RVT-2001 could have a future in lower-risk, transfusion-dependent MDS patients, a bet that has now proved to be fruitless. Though Hemavant is "largely synonymous" with RVT-2001, the company's team is mainly staffed by Roivant personnel who will be redeployed, according to Roivant's spokesperson, who said the company doesn't expect any "meaningful team or corporate impact" beyond the trial discontinuation.