Organon stops working on endometriosis medication after phase 2 test failure
An experimental organic pore drug used to alleviate pain caused by endometriosis failed to meet the primary goals of mid-term clinical trials, causing the company to stop developing candidates for the product, a key part of its growth strategy previously described.
Organon’s Tuesday announcement provided little details about the trial’s results, which recruited 354 women aged 18 to 49 who experienced moderate to severe pain associated with endometriosis. The main goal of the Phase 2A/B study was to measure changes in overall pelvic pain scores after 16 weeks. Jersey, New Jersey-based Organon said only that its drug OG-6219 did not beat the placebo, as evaluated by the numerical scale used to measure pelvic pain.
In endometriosis, the endometrium grows outside the uterus, causing abdominal pain. Standard treatment for this chronic disease includes over-the-counter painkillers, such as nonsteroidal anti-inflammatory drugs. Because hormone changes can worsen the pain of endometriosis, drugs that limit or block hormones offer another therapeutic option. But hormone therapy carries a risk of side effects. For severe or advanced cases of endometriosis, surgery is another treatment option.
“This is a priority area of disease for us because of the limited treatment options for women with endometriosis at present,” Organon said in his annual report.
With OG-6219 being a twice-daily pill, Organon hopes to introduce a new non-hormonal option for the treatment of endometriosis pain. The drug is a small molecule designed to inhibit type 1 hydroxysterol 17-β dehydrogenase type 1 (HSD17B1), an enzyme that plays a role in regulating the growth of uterine tissue. This approach is designed to provide local effects against endometriotic tissue without affecting systemic circulation.
On a February call to discuss 2024 financial results, Juan Camilo Arjona Ferreira, head of R&D and chief medical officer, said Organon has a backup plan for OG-6219, “this supports our goal to deliver products under this new mechanism.” Tuesday’s announcement did not discuss the status or plans for the backup plan.
Organon started from Merck in 2021 on an independent way, and is composed of publicly traded companies whose portfolio consists of the drug giant’s former women’s health business and non-favorite drugs. Although these products provide stable revenue, Organon also aims to acquire and develop new drugs that provide growth opportunities.
Merck’s spin-offs did not bring much drug discoverers and infrastructure. Organon has turned to business development as a way to build drug pipelines for candidates at various stages of development. OG-6219 is part of the acquisition of its developer, Women’s Healthcare Company, in 2021. Organon paid $75 million in advance and assumed $9 million in biotech debt. Another $600 million is related to achieving milestones.
Forendo also brought another HSD17B1 inhibitor to Organon, now coded as OG-7191. This preclinical drug is being used as a treatment for polycystic ovary syndrome (PCOS), a chronic disease that causes menstrual cycle disruption and infertility. In its annual report, the company said there are currently no FDA-approved therapies for PCOS, another priority area of disease in Organon.
Organon’s growth strategy goes beyond women’s health. Last year, the company paid $175 million to acquire Dermavant, a subsidiary of Roivant Sciences whose main asset is VTAMA, a topical drug and received FDA approval in plaque psoriasis and atopic dermatitis.
Photo: Tang Yanjun/China News Service/VCG, via Getty Images
