Sun Sentinel Palm Beach Edition
Boca firm awaits menopause treatment OK
Hot flashes, night sweats and painful sex are common complaints among menopausal women — and not much new is available to address them.
But a Boca Raton company, TherapeuticsMD, has been developing treatments and now has two drug candidates that could be cleared for marketing this year by the Food and Drug Administration.
TherapeuticsMD’s mission is women’s health because there are “so many things [in medicine] that women need, and they just don’t have them,” CEO Robert Finizio said.
The two potential new drugs have finished clinical trials and have been submitted to the FDA for review. If approved for sale, one could be available to women through their doctor’s office as early as July, and the other by midnext year.
Ten years into drug development, TherapeuticsMD’s CEO is careful to avoid making any claims that could jeopardize the new drug applications before the FDA. “They’re the boss,” he said.
At risk is the continued health of TherapeuticsMD, which went public in 2016, and has a 52-week trading range of $3.50 to $8.30 a share on the New York Stock Ex-
change.
After pouring about $500 million into research and development, and building a corporate structure to support and market new drugs, the company hopes to bring new options to millions of women worldwide.
Regulatory approval would mean the company ramping up its internal salesforce to 500 over the next year. The company already has plans to hire more than 100 sales representatives. TherapeuticsMD employs 210 people, 130 of whom are in Boca Raton. They include scientists, doctors and corporate staff.
TherapeuticsMD management and investors will be watching for possible regulatory action around these dates:
May 29: The company expects to hear from the FDA on whether it can market its first proposed drug designed to treat vaginal dryness and painful intercourse.
End of October: TherapeuticsMD’s second drug candidate, for hot flashes, could be cleared by the FDA; the company is expecting an early indication in March. The proposed hot flashes treatment is in the form of one soft-gel capsule of estradiol, a form of estrogen, and progesterone. Today, many women have to take two different medications to treat hot flashes and other menopausal symptoms.
A 10-year timeline for TherapeuticsMD’s new drug development is “not bad,” said Dr. Jackson Streeter, CEO of the Florida Institute for the Commercialization of Public Research in Gainesville, which provides seed, or startup capital. The institute has no connection to TherapeuticsMD, which is funded by private and public investors.
The drug developer is in “a high-risk, high-reward kind of industry,” said Streeter, a former Navy doctor who led biotech companies in California before joining the institute. “It takes a long time, and it takes a lot of money to drive these things through.”
But TherapeuticsMD is keeping its eye on the prize: huge potential demand. The menopause symptoms affect more than 30 million women in the United States alone, resulting in a market opportunity of $20 billion to $25 billion for each proposed drug, based on pricing of current drugs on the market, the company said in a regulatory filing.
Julia Amadio, chief product officer for TherapeuticsMD, said the number of women entering menopause and in post-menopause continues to grow every month. “Boomers are aging, and we’re the prime target,” she said. “And the next generation [in their 40s] also is moving into the phase.”
The FDA warns women that hormones should be used in the lowest dose that helps, and for the shortest time, because all hormones carry some risks, including blood clots, heart attacks, strokes, breast cancer and gall bladder disease.
TherapeuticsMD conducted a 12-month clinical trial of its proposed drug to treat hot flashes, with 1,750 women at 100 sites across the country. The study compared four different dose combinations of the two hormones, based on what women typically take for their age and symptoms, along with a placebo.
Dr. Brian Bernick, chief clinical officer and co-founder of the company, said menopausal women seeking hormone therapy are typically prescribed between 0.5 and 1 milligram of estradiol to be taken with 100 milligrams of progesterone. (Before TherapeuticsMD, he was a practicing obstetrician-gynecologist in Boca Raton and delivered the Finizios’ two children.)
Bernick said TherapeuticsMD’s clinical study is important beyond its support of a new drug application.
“No one had established how much progesterone you needed on a daily basis to oppose estrogen. Patients were using these alternative products by chance, and doctors were hoping they were giving patients the right combinations,” Bernick said.
Dr. JoAnn Pinkerton, executive director of the North American Menopause Society based in Cleveland, Ohio, said TherapeuticsMD’s clinical trial showed “the frequency and severity of hot flashes was reduced over a oneyear period, with no serious adverse events and with uterine protection from the progesterone.”
Of the proposed drug, Pinkerton said: “I think it’s very much needed ... having something combined in one pill will be very attractive.”
Amadio said one of the problems with prescribing two hormone medications to be taken together is that some women think progesterone makes them feel “worse,” so they may only take the “good-feeling” estrogen, she said.
Pinkerton, who is also division director of midlife health at the University of Virginia Health System in Charlottesville, Va., warns that progesterone is needed as well, to protect women taking estrogen from endometrial cancer, or cancer of the uterine lining.
Meanwhile, the company had a small setback last year with its vaginal drug candidate. In May, it received a letter in which the FDA sought more data on long-term endometrial safety because the proposed drug contains a low dose of estrogen. Finizio said the FDA initially denied the new drug application, saying it had asked for a 12-month trial. But the FDA had only requested a 12-week trial, which is what TherapeuticsMD submitted.
“The FDA confirmed it was an ‘oversight’ on their part. They did not tell us to do a 12-month trial,” Finizio said.
Following the FDA letter and a hit to TherapeuticsMD’s stock price, some stockholders filed suit, alleging the company made “false and misleading statements” that caused the delay and violated securities law. But the lawsuits have since been dismissed, according to Finizio and a regulatory filing.
In November, the FDA allowed the company to resubmit its application, with the caveat that TherapeuticsMD conduct a post-marketing study to ensure safety. “It’s a more cost-effective way to get the data,” Finizio said.
But even If TherapeuticsMD receives FDA marketing approval, the company will still face some big-name competition in the pharmaceutical industry. In the vaginal product category, competitors could include Allergan’s Estrace Cream, Pfizer’s Premarin Cream and Novo Nordisk’s Vagifem. For hot flashes, the FDA has approved patches, gels and pills containing either estradiol (estrogen) or progestin (progesterone).
Meanwhile, TherapeuticsMD is generating revenues with its prenatal vitamins, VitaMedMD, which already have FDA approval. Revenues were just over $19 million in 2016, while losses totaled nearly $90 million, reflecting the investment in its hormone therapy drug candidates. The company’s 2017 financial report is scheduled for release on Feb. 20.
TherapeuticsMD has begun hiring a sales force to eventually take any approved drugs to market. The company has raised nearly $70 million in a stock offering toward a nationwide sales force. Finizio said an internal sales force is the only way to go because it allows the company to properly control information being presented to physicians and their patients.
If TherapeuticsMD’s drug candidates are approved, Finizio and Bernick say they will work to ensure their new drugs are accessible to all consumers. The industry has come under pressure in recent years for the “sticker shock” of new cancer and other drugs.
“We’ve made it an important priority for this company that the out-of-pocket cost is going to be affordable,” Bernick said.