Celgene International Sarl's successful multiple myeloma drug, Revlimid (lenalidomide), met an important benchmark in newly diagnosed patients in a Phase III study.
In that large, randomized trial, 1,623 newly diagnosed patients who were ineligible for autologous stem cell transplant received continuous oral Revlmid plus low-dose dexamethasone, or melphalan, prednisone and thalidomide (MPT). The trial met its primary endpoint of improving overall survival.
Revlimid is currently approved in nearly 70 countries for patients who have already received at least one prior therapy.
Greg Geissman, director of public relations for Celgene, told BioWorld today that the potential approval in the newly diagnosed indication "would open availability for a number of patients," and would account for about $500 million in additional revenue in 2017.
Patients in the study received Revlimid plus low-dose dexamethasone for 18 28-day cycles or MPT combination for up to 12 42-day cycles. In addition to progression-free survival (PFS), the study also assessed overall survival (OS), response rate, quality of life and safety.
"We're planning to submit an abstract for the American Society of Hematology meeting this year. We're working with investigators to put that abstract together," Geissman said.
Shares of Celgene (NASDAQ:CELG) gained $9.84, or 7.9 percent, Thursday to close at $134.92.
In addition to multiple myeloma, Revlimid also is approved for transfusion-dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities, and for mantle cell lymphoma in patients who have relapsed or progressed after two prior therapies.
"We expect [Revlimid plus dexamethasone] to likely show a clear trend for OS improvement, with potential for a significant benefit, given the lack of crossover in the study," wrote Piper Jaffray analyst Ian Somaiya. "We believe the data will support regulatory approval in the U.S. and Europe, and could lead to rapid cannibalization of MP-based regimens, unlocking a $3 billion peak sales opportunity in front-line/maintenance in Europe." He added that Celgene's stock is "underowned" relative to other large-cap biotech stocks.
Somaiya modeled a slow rate of adoption in front-line use in Europe, and a gradual increase in treatment duration globally in spite of evidence of OS benefit in Revlimid maintenance treatment.
According to Somaiya, the MM-020 study was 80 percent powered to show 25 percent improvement in PFS, which he predicted could be as high as 38 percent improvement, or about 10 months.
Wells Fargo analyst Brian Abrahams noted that the study was generally expected to meet the PFS endpoint. "Though this was generally expected by us and, we believe, the market, based on historical data for [Revlimid plus dexamethasone regimen] and MPT, we see this as an important de-risking event – reducing concerns about potential disruption to future EU treatment dynamics," Abrahams wrote. He added that he expects "upside" for Celgene's shares on that de-risking event, "which we believe should set the stage for them to ultimately capture a more meaningful part of the EU market."
Robyn Karnauskas, of Deutsche Bank, noted that the result "removes a key overhang" for Celgene, as with these data "they can now file for approval in the EU."
"We do not know the magnitude of benefit with [the] continuous Revlimid arm, but as we noted before we expect the PFS in this arm to be 35-36 months. Notably, we expect [the] control arm to do 25-26 months in this study," Karnauskas wrote.
The new trial results build on other recent advances for Revlimid.
In June, Summit, N.J.-based Celgene disclosed results from two studies evaluating the combination of Revlimid and Rituxan (rituximab, Roche AG and Biogen Idec Inc.) in various non-Hodgkin's lymphomas. Those results showed that in patients with previously untreated follicular lymphoma in a Phase II study yielded, in 54 patients evaluable, an overall response rate of 92.6 percent.
Revlimid with R-CHOP (R2CHOP) in diffuse large B-cell lymphoma or Grade IIIB follicular lymphoma, tested in a Phase II trial, yielded an overall response rate in 63 patient evaluable.
Also in June, the European Commission amended the marketing authorization application for Revlimid to add the indication for transfusion-dependent anemia due to low or intermediate-1 risk myelodysplastic syndromes associated with isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.