Myovant to Seek FDA Approval for Uterine Fibroid Treatment Following Successful Trial

Myovant is preparing to seek regulatory approval for its treatment of uterine fibroids following positive Phase III results that showed a 71.2% response rate in patients.

This morning, Switzerland-based Myovant, a subsidiary of Roivant Sciences, said its Phase III LIBERTY 2 trial hit its primary endpoint, as well as six secondary endpoints in women with uterine fibroids. The trial assessed Myovant’s once-daily relugolix combination therapy. In addition to the strong response rates in patients, Myovant said there was also an 84.3% reduction in menstrual blood loss. The results from the Phase III LIBERTY 2 trial confirm the positive Phase III LIBERTY 1 trial, Myovant said. Like LIBERTY 2, LIBERTY 1 met its primary endpoint and six key secondary endpoints

According to the LIBERTY 2 data, 71.2% of women who received the once-daily relugolix combination therapy achieved the responder criteria compared with 14.7% of women receiving placebo. Menstrual blood loss was one of the key secondary endpoints of the trial. A response was defined as a menstrual blood loss volume of less than 80 milliliters and a 50% or greater reduction from baseline in menstrual blood loss volume during the last 35 days of treatment measured using the alkaline hematin method. On average, women receiving relugolix combination therapy experienced a highly significant 84.3% reduction in menstrual blood loss from baseline. In addition, a significantly greater proportion of women suffering from moderate-to-severe pain from uterine fibroids at baseline experienced no pain or minimal pain during the last 35 days of treatment with relugolix combination therapy compared with women on placebo.

In addition to the menstrual blood reduction, other key secondary endpoints hit in the trial include reduction in pain, improvement in quality of life, amenorrhea, defined as no or negligible blood loss, an improvement in anemia in those women with anemia at baseline and reduction in uterine volume.

Uterine fibroids are noncancerous tumors that develop in or on the walls of the uterus. Although benign, the symptoms include abnormal uterine bleeding, heavy or painful periods, anemia, abdominal pain, backache, increased abdominal girth and bloating, urinary frequency or retention, constipation or painful defecation, pregnancy loss, painful intercourse and sometimes infertility.

In addition to the LIBERTY 2 data, Myovant said a separate clinical study of single-tablet relugolix combination therapy met all required and pre-specified FDA criteria for bioequivalence. That will provide the data necessary to include the once-daily, single-tablet regimen in the NDA submission for approval of the treatment for uterine fibroids, the company said.

Lynn Seely, president and chief executive officer of Myovant Sciences, said the two Phase III LIBERTY studies demonstrate that relugolix combination therapy has a “distinctive constellation of attributes, including substantial symptom relief with a well-tolerated safety profile” in a once-per-day pill.

“No one else has achieved this for women with uterine fibroids. With these results, our team is focused on submitting the NDA by the end of the year and continuing to build the organization and capabilities to efficiently and successfully deliver this treatment to women in need,” Seely said in a statement.

The overall incidence of adverse events in the relugolix combination and placebo groups was comparable, 60.3% vs. 58.9%, Myovant said. In the relugolix combination therapy group, 1.6% of women discontinued treatment early due to adverse events compared with 4.7% in the placebo group.