Takeda hits FDA snag on Ninlaro, but its growth still helps drive revenue hike

In Takeda’s last quarterly report as a separate company from Shire, things seem to be going its way—key drugs are growing nicely, Shire integration is on track, and cash-raising sell-offs are underway, executives said.

But all is not rosy. Takeda's rising multiple myeloma star and key growth driver Ninlaro just hit a snag at the FDA.

The Japanese pharma said on Friday that it decided to withdraw an application to expand Ninlaro’s use as a maintenance therapy in post-transplant patients, after conversations with the FDA showed that the agency wants to know how long Ninlaro can extend patients’ lives, data that Takeda doesn’t yet have.

"After only 31 months of median follow-up, there were not enough deaths accrued in the study to assess any significant difference in overall survival, or even in fact imply a trend," Christopher Morabito, Takeda’s head of R&D portfolio strategy, said during a conference call on Friday.

Morabito admitted that the speed bump shouldn’t have been a surprise, because the FDA previously telegraphed that overall survival (OS) data is a critical endpoint for their final analysis. Celgene's Revlimid, for example, is one of the drugs that faced that hurdle.

Only two months ago at the American Society of Hematology annual meeting, Takeda touted data showing Ninlaro could cut the risk of disease progression or death by 28% compared with placebo. Ninlaro data has also indicated it could be safer than its blockbuster predecessor Velcade, and that it could be "a very good combination partner with other classes," Jay Humphrey, VP of U.S. marketing, told FiercePharma at the time.

Full OS data for myeloma maintenance therapy could take years to accumulate, if Revlimid's case is any indication. A meta-analysis published in the Journal of Clinical Oncology in 2017 turned up a seven-year survival rate of 62% among post-transplant patients on the Celgene drug. Morabito said Takeda will be in active talks with regulators "as to how much OS data they need to see to feel comfortable with the endpoint."

Takeda can’t yet predict when it will be able to resubmit the app, Morabito said, though he assured investors another study that involves newly diagnosed patients who haven’t undergone a transplant remains on track, with readouts expected in the second half of 2019.

Ninlaro has been a new favorite at Takeda since Velcade dropped off the patent cliff. In the quarter that ended in December, Ninlaro sales rose 33.2%, to 17.1 billion Japanese yen ($157 million). Another important growth driver is bowel disorder therapy Entyvio: During the first nine months, Entyvio added 194.4 billion Japanese yen to Takeda’s top line, up 35.1%. These two drugs helped Takeda deliver 4.8% revenue growth over the three quarters.

Takeda wrapped up its $58 billion deal to acquire Shire in January, but it won’t be until next quarter that Shire’s numbers are consolidated with Takeda’s. Sell-offs to raise cash for paying down deal-related debt, however, are already under way. Takeda just announced on Monday that it would sell 21 assets, including its old Osaka headquarters building, and aims to rack up 38 billion yen in sale proceeds by the end of March.

CEO Christophe Weber has also said the company would look to sell up to $10 billion worth of noncore assets to fill the huge debt hole it incurred from the Shire buyout. Takeda has drawn a line around five key areas it wants to keep: gastroenterology, oncology, neuroscience, rare diseases, and plasma-based therapies and vaccines, which together represent 75% of the combined business, according to CFO Costa Saroukos. "All other areas are noncore, basically," he said.

Executives again stressed their focus on a smooth integration and driving down its net debt/EBITDA ratio to 2.0x in the midterm. "At the moment, it’s very much about … keeping the business momentum and not disrupting our R&D and pipeline momentum," Weber said.

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