Takeda’s ongoing bid to expand the audience for its cancer drug Ninlaro has suffered some setbacks this year, but now the company can claim one big win—and investors are cheering.
Takeda announced that in a phase 3 trial, patients who had not undergone stem cell transplants and were taking Ninlaro as a first-line maintenance treatment had significant survival benefits over those on a placebo. The trial is part of Takeda’s bid to make Ninlaro the first drug approved for “switch” maintenance in multiple myeloma, meaning it could be prescribed to patients after they’ve been treated for six months to a year with different medicines.
Takeda did not provide details about the results but vowed to submit them for presentation at an upcoming medical meeting. Still, the news was enough to send the company’s shares up more than 6% in premarket trading to nearly $21.
As Takeda’s multiple myeloma blockbuster Velcade fades in the face of generic competition, the company is counting on Ninlaro to help pick up the slack. But two pieces of bad news this year raised doubts about whether the new entrant could live up to expectations.
In February, Takeda withdrew an application to the FDA for approval of Ninlaro as maintenance therapy in patients who have undergone stem-cell transplants. The agency cited the lack of data on overall survival—figures that could take years for the company to collect. The FDA’s dismissal was a surprise, considering Takeda had just presented data showing Ninlaro had slashed the risk of disease progression or death by 28% in multiple myeloma patients post-transplant.
And in June, Takeda called off a phase 3 trial of Ninlaro in patients with systemic light-chain amyloidosis, a rare disorder that can occur in patients with blood cancers like multiple myeloma. An analysis showed that combining Ninlaro with dexamethasone in patients who failed previous treatments was no more effective than using standard chemotherapy.
Ninlaro is still managing to chart impressive growth; sales of the product grew nearly 33% in the most recent quarter. And the company cited the drug as a key growth driver that contributed to the decision to hike profit guidance for the full year ending in March of 2020.
But any opportunity to expand Ninlaro’s market will only help Takeda cushion the impact of challenges facing its other franchises. The company’s rare disease unit—despite being bolstered by its $59 billion purchase of Shire—was the only weak spot in its six-month earnings report. Revenue from that unit fell 11% year over year as competition put pressure on its hemophilia drugs.
Ninlaro is an oral drug that was approved in 2015 in combination with Celgene’s Revlimid to treat multiple myeloma patients who had previously received at least one other therapy. The company's growth strategy for the product extends well beyond switch maintenance. It has more than four ongoing clinical trials of Ninlaro in a variety of combinations and treatment settings.