Celgene (NASDAQ:CELG) got the win it needed for Revlimid, showing that its blood-cancer pill significantly increased progression free survival [PFS] compared to chemotherapy in newly diagnosed multiple myeloma. The New Jersey group said the findings of the MM-020 study will be used to support regulatory submissions to expand the immunomodulator’s label into first-line treatment and cement its hold in the space.
Shares jumped 8% to a record high of $134.92 on the news, fueled by hopes that the findings will widen adoption and payer acceptance of early use, which is already taking place in the U.S. Detailed data are being held back for medical meetings, with the American Society of Hematology a likely forum, meaning it may be several months before the sector learns the magnitude of the survival benefit and whether Revlimid improved overall survival.
Clearing the air
MM-020 tested Revlimid and low-dose dexamethasone against melphalan, prednisone and thalidomide; the former two being the standard of care before Johnson & Johnson and Takeda’s Velcade launched in 2003. Velcade plus melphalan and prednisone has achieved PFS of 18.3 months when compared to the latter two treatments, providing a benchmark against which Revlimid will no doubt be compared (Event – Celgene aims to solidify Revlimid’s hold in multiple myeloma, July 1, 2013).
It will also be measured against the findings of the MM-015 trial, which found Revlimid plus melphalan and prednisone significantly extended PFS to 31 months, when compared to the 13 months of the older drugs, but only if a maintenance phase of Revlimid was added following an induction phase of nine 28 day cycles of Revlimid plus melphalan and prednisone. When the nine-cycle regimen, a normal schedule for the older drugs, was used with no Revlimid maintenance phase, there was no significant difference in PFS (Celgene's Revlimid data prompts questions but high hopes remain, December 9, 2009).
The MM-015 trial had been one of three submitted for European first line approval, but because secondary cancers have been a worry EU regulators were looking for balancing data on overall survival (Celgene data need to grow up to give Revlimid European success, October 11, 2012). There is some thought that the combination with oral melphalan may be behind the secondary malignancy issue.
Thus, the 1,600-patient MM-020 trial will most likely give more robust answers to the lingering questions, as it is one of the biggest ever in multiple myeloma and nearly four times the size of MM-015.
Hurry up and wait
The share price jump of 8% came around the middle of the range expected from Leerink Swann, a sign that investors may want a little more information from Celgene before viewing MM-020 as a complete success. No information on the overall survival endpoint, the gold standard of cancer treatment, has been disclosed.
Moreover, MM-020’s active treatment arms include one that continues until disease progression and one that ends after 18 four-week cycles. ISI Group analyst Mark Schoenebaum wrote that if the 72-week regimen is as effective, it has the potential to limit Revlimid’s sales in this setting – every extra month could be worth $40m in sales. Thus investors will be anxious to see a comparison between those two arms, as well as overall survival data and the secondary cancer trends.
Celgene management believes the label expansion is worth $500m in sales in 2017, compared with 2013’s forecast of $4.16bn in sales. Getting the first-line efficacy win is not necessarily essential to Revlimid’s success, but it would be a useful addition and help maintain momentum for Celgene.