Organon finally offers a larger pharma model for women's health. But 'one company can't do it by itself'

When it comes to getting women's health therapies to market, nothing is ever straightforward. From encouraging women to "step forward" for clinical trials to battling for R&D funding, it remains a tough arena—yet Organon is building a pipeline to mount a challenge.

“Every woman has a story to tell,” Organon’s R&D head Sandy Milligan, M.D., told Fierce Biotech in an interview. “I just absolutely love the fact that we are lowering that sort of taboo culture of not being able to talk about it. We need more women to tell us about their experience, to come forth and raise their hands.”

While interest in women’s health has risen in recent years, Organon is still one of the few companies with a pipeline nearly wholly built around healthcare innovations for women specifically. In 2020, only 1% of biopharma R&D investment was in female-specific conditions excluding oncology, according to a McKinsey report. For female-specific cancers, clinical-phase investments sat at 4%. Even with so little research in women's health, Milligan has found trial recruitment a challenge.

Organon wants to change that, starting in-house.

The Merck & Co. spin-out encourages its own workers to speak up, offering internal forums where all employees can talk about personal challenges related to women’s health. Milligan said this gives employees a face—potentially even one of a leader or executive—to match with the conditions or diseases the company is trying to treat.  

Like most women, Organon wears many hats with its three core areas: women’s health, biosimilars and legacy products. In the last 20 months, the company has looked at over 400 potential pipeline candidates, snapping up eight. Most are preclinical, with the majority of the pipeline consisting of early-stage assets, including ObsEva’s ebopiprant for pre-term labor inflammation and uterine contractions, as well as Cirqle’s non-hormonal on-demand contraceptive.

While contraception isn’t technically a female-specific condition, society has pushed the brunt of it upon the female body. Organon wants to balance out the burden by exploring contraceptive options for both females and males.

With so many early-stage candidates, Organon has a lopsided pipeline and few nearer-term assets to push forward. Milligan said this somewhat mirrors broader trends, noting that there aren’t a lot of new women’s health treatments expected to come to market anytime soon in general. Over the years, many Big Pharmas have deemphasized their investment in women’s health, according to Milligan, which has contributed to the dearth of late-stage assets.

Organon is instead looking to medtech devices to fill the pipeline gap.

“If you're really focused just on women's health, you almost can't stay in just a pharmaceutical therapeutic option,” according to Milligan. 

Everything that happens to women is basically treated like it’s a hormonal imbalance." — Sandy Milligan, M.D., Organon

At the beginning of this year, Organon invested in Claria Medical’s device that aims to make hysterectomies a minimally invasive, laparoscopic procedure that would be safer and faster than the current standard. Back in 2021 before Organon’s launch, corporate parent Merck paid out $240 million to acquire Alydia Health and set up the spin-out's portfolio. The acquired assets include a system called Jada designed to stem blood loss during postpartum hemorrhage.

On the biopharma side, Organon is particularly interested in understanding the root of disease instead of just controlling symptoms with hormones—the most common band-aid given for many women-specific conditions.

“Everything that happens to women is basically treated like it’s a hormonal imbalance,” Milligan said, citing heavy menstrual periods or pain as a prime example.

“The Holy Grail, frankly, would be investing in therapeutics that are not just specific to the target, but specific to that woman's expression of the target,” Milligan explained. “We're years away from that, but we need to start investing now or we'll never get there.”

As head of R&D, her main focuses are conditions with a high unmet medical need, such as severe peri- or post-menopausal symptoms—which affects 10 million women, according to Milligan—and can include hot flashes, mood changes, anxiety and changes in sexual function.

Endometriosis is another. The tissue growth disorder causes the uterine lining to grow outside of the uterus, such as on the ovaries, fallopian tubes or intestines. It can cause chronic pain and infertility and impacts about 1 in 10 females of reproductive age. There are few effective treatments available, with the main options being hormone therapy or surgery.

One of Organon’s more advanced pipeline meds for the disorder is OG-6219, a midphase asset obtained from Forendo Pharma in a 2021 acquisition. The drug lowers estrogen production in endometriotic lesions and is currently being evaluated in a clinical study dubbed Elena. The global phase 2a/b trial is still recruiting women who have been surgically diagnosed with endometriosis and have moderate or severe pain. The estimated enrollment goal is 380 participants and completion is expected for the end of 2024.   

While Milligan is optimistic the trial will hit its enrollment goals, she said recruitment has been challenging. For instance, fewer women have returned to the clinic since the pandemic began. The company is attracting participants via word-of-mouth and public campaigns.

“We need women to step forward, wave their arms and say, ‘You know what? I want to further science,’” Milligan said.

Another place Organon needed people to step forward was on staffing. Milligan was worried about attracting talent after the split from Merck. Milligan herself is no stranger to Big Pharma, having clocked in time at Amgen, Genentech and Merck (all after getting her M.D. and J.D.), She was worried that a smaller company focused on women’s health might deter people from joining. However, she’s been pleasantly surprised at how many people have been attracted to the company for its mission, with a current staff roster boasting 10,000 names. 

While Milligan is excited about all Organon is working on, the reality of a women’s health-focused company can still be daunting. Engagement in the space has been rising recently, but Milligan said interest—and investment—is still extremely fragmented.  

“We're just part of the ecosystem,” she said. “And one company can't do it by itself.”

The R&D leader wants to see “the silos go down,” noting that more seamless interactions between pharmas, organizations and health agencies could help move innovation along faster. Organon is working to break those barriers down with more messaging, communication and outreach.

Right now, the company is still “trying to find that unifying theme” for outreach, one that can pull people together and drive women’s healthcare forward. But as Organon has found in listening to women, the unifying theme might be right in front of us.