Doctors and investors are getting their first glimpse of the data that compelled Takeda Pharmaceutical to make a $6 billion bet on an experimental pill. The drug, which it acquired from Boston biotech Nimbus Therapeutics last month, blocks a key lever in the immune system that Takeda believes has the potential to treat numerous autoimmune and inflammatory diseases.
In an intermediate-stage clinical study of 259 people with moderate-to-severe plaque psoriasis, the highest dose of the daily pill completely cleared itchy and painful patches of skin in one-third of the patients after 12 weeks. “We’re offering the potential for a functional cure,” Andrew Plump, Takeda’s president of research and development, said in an interview.
The results were impressive for other patients too. Nearly half of the patients on the high dose had 90 percent of their psoriasis cleared. And two-thirds of the patients had 75 percent of their psoriasis cleared — compared with 6 percent of patients who received a placebo. Takeda announced the results Saturday morning and will present them at a medical conference for dermatologists in New Orleans over the weekend.
The drug was not without side effects. Mild or moderate adverse events occurred in 53 to 62 percent of patients who got Takeda’s drug, depending on the dose, and 44 percent of placebo recipients experienced side effects. The company didn’t provide details in its press statement, but Plump said the most common side effects from the drug were increased risk of mild respiratory viral infections, including COVID-19, and acne.
Takeda will start an advanced study of the drug in psoriasis later this year, and is planning intermediate stage studies for lupus, Crohn’s disease, and ulcerative colitis, Plump said. The company also expects results from an intermediate study of the drug in psoriatic arthritis this year, and if it is positive, a larger study could begin next year.
Simultaneously testing a single drug across five diseases is unusual, with the exception of cancer drugs, which are routinely assessed against multiple types of tumors. Yet that list is only the beginning. Plump said his company has compiled up to 20 autoimmune and inflammatory conditions that the pill might be able to treat because it selectively blocks an important protein that ignites immune responses.
“This has the potential to be one of the most broadly indicated mechanisms — outside of oncology — of any oral medicine that’s ever been developed,” Plump said. “I think in 10 years, we’ll look back and say that this was a bargain.”
Nimbus designed the drug to target a protein known as TYK2 that helps immune cells kick into high gear when they sense signs of danger. While that response can be helpful for fighting infections, it can be harmful if over-activated in an autoimmune or inflammatory disease.
Curiously, people born with genetic mutations that hinder TYK2 are less likely to develop those diseases. That observation triggered a race among drug companies replicate those benefits in a pill — a task that proved difficult because blocking closely related proteins may increase the risk of heart attack, stroke, blood clots, and cancer.
Last summer, Bristol Myers Squibb was the first to win approval for a TYK2 inhibitor. The pharmaceutical giant’s drug, Sotyktu, is sold for treating moderate-to-severe plaque psoriasis, and the company is planning tests in several more diseases.
Nimbus, a small biotech company with about 80 employees, thought there was room for improvement. The company claimed that its compound was far better at only inhibiting TYK2 and sparring its cousins, suggesting that it may be more effective and have fewer risks.
The results that Takeda saw from the intermediate study, finally divulged this weekend, led the firm to announce in December its intention to acquire the drug. Nimbus earned $4 billion when the deal closed in February and could reap $2 billion more in potential milestone payments.
A larger study will be needed for approval, and to better compare the drug’s safety and effectiveness against Sotyktu. Plump is confident in Takeda’s prospects. “It’s a very competitive space. And so our intent is to go all out,” he said.” This is our top priority.”
Ryan Cross can be reached at firstname.lastname@example.org. Follow him on Twitter @RLCscienceboss.