A new hormonal therapy designed to treat uterine fibroids called relugolix has finished a Phase 3 study in Japan, and the results are positive.
That's welcome news for a SoftBank-based company based in Basel, Switzerland called Roivant Sciences that's separately dealing with much lousier news.
But let's back up a bit first.
The Phase 3 trial was one of several Phase 3 trials of relugolix that are ongoing, including in the U.S. and Europe, where the drug is also being tested as a means to treat endometriosis-associated pain and to help men with advanced prostate cancer by suppressing their testosterone levels. The result out of Japan isn't enough to win it FDA approval, but Myovant, the biopharmaceutical company behind it, is expected to use the result from Japan to support its approval here in the U.S.
Why does it matter? Well, for one thing, a win would be meaningful for Myovant, which staged the biggest biotech IPO of last year, shortly after recruiting Lynn Seely as its CEO. (Seely was formerly the chief medical officer of Medivation, which sold to Pfizer last year for $14 billion). In other words, expectations are high.
Myovant is also a subsidiary of Roivant, a young holding company whose 32-year-old founder, Vivek Ramaswamy, believes will become a giant holding company for dozens of independent biopharmaceutical companies.
Ramaswamy sold SoftBank on that vision over the summer, in fact, with SoftBank leading a $1.1 billion investment in the company (and getting a steep discount on its privately held shares in the process, we're told).
Then, disaster. More specifically, early last week, Axovant -- another of Roivant's holding companies that was taken public and wound up becoming the biggest biotech IPO of 2015 -- received news that its much-hyped, experimental Alzheimer’s drug, interperdine, doesn’t work. (We wrote about that here.) It was a huge blow to the company, whose shares plummeted 75 percent that day and have remained flat. It was also a black mark for Roivant, which has told a convincing story until now about using data more effectively than big pharma companies that have exponentially more employees and resources.
Now, to regain his place as a wunderkind of the biotech world, Ramaswamy -- a Harvard-educated biology major with a law degree from Yale -- needs some positive momentum.
Relugolix is a smaller bet for Roivant, but it's not insignificant. According to one study, 70 percent of white women, and between 80 and 90 percent of black women, will develop fibroids by age 50, and 90 percent of women who are diagnosed with them seek medical or surgical treatment of their fibroids within a year. (Many others don't realize they have them.)
The less invasive route is, of course, medicine. In fact, reloguolix is hoping to grab market share of a hormone therapy already in the market: Leuprorelin, a product that first received FDA approval in 1985 and is currently used in the treatment of hormone-responsive cancers like prostate cancer and breast cancer, as well as to treat estrogen-dependent conditions, including uterine fibroids and endometriosis. (It's marketed under different brands by different companies. The German multinational conglomerate Bayer sells it as Viadur; The U.S. company Abbott Laboratories markets it as Lupor.)
There is still work to do; the other Phase 3 studies for relugolix were kicked off fairly recently, meaning it could be until closer to 2019 before the drug receives approval -- if it's able to win approval at all. In the meantime, another biopharma giant, AbbVie, is developing a competing drug called elagolix, so the race is on.
In the meantime, Ramaswamy, who worked formerly as hedge fund analyst, is hedging his bets. Axovant, for example, is seeking to find other uses for inteperdine, part of which involves an ongoing Phase 2 test to treat a progressive brain disorder called Lewy body dementia. (The market is much smaller than for Alzheimer's patients. The disease impacts 1.3 million Americans, for example, compared with Alzheimer's, which impacts 5.5 million.)
Another Roivant subsidiary, Enzyvant, is also seeking FDA approval to treat a rare pediatric disease called DiGeorge Syndrome and last month received a designation from the FDA that should move it through the review process more quickly. (The path is more streamlined in the case of rare diseases with life or death consequences.)
Roivant appears to be focusing more nakedly on getting third party biotech companies to partner with it on drug development, too. The idea, presumably, is that Roivant can bring them efficiencies by opening up its infrastructure to them.
Earlier this week, for example, Roivant plugged $116 million into a company in which it already owned a stake: Arbutus, a hepatitis B-focused biotech firm. It's was a sizable gamble on the company. Then again, it's chasing a big market. Approximately 350 million people in the world are believed to have the disease. Arbutus will now rely on Roivant for functions that it would otherwise have had to build in-house.
Whether Roivant's various moves will pay off enough will require more time to learn. In the meantime, Roivant -- which has now raised more than $2 billion from investors, including from Founders Fund, and the hedge funds Viking Global Investors and QVT Financial -- will have to hope its investors remain patient.
We reached out to Ramaswamy for this story; through a spokesman, he declined to comment.