This company’s novel and lonely development of a new fertility drug belies the industry’s current uninterest in the field.
Nora Therapeutics is about the only game in town for development of novel-MOA (mechanism of action) fertility drugs. Its sole product in the current pipeline, a proprietary G-CSF (granulocyte-colony stimulating factor), targets miscarriages in women undergoing in-vitro fertilization (IVF) as well as recurrent miscarriages among other women. Nora’s two main Phase 2 trials, THRIVE and RESPONSE, address IVF-related and other miscarriages, respectively.
WHAT’S AT STAKE
Historically, fertility epitomizes areas fit for specialty care drugs, where the problems and solutions are more complex than in diseases treatable with a simple pill. (See “EMD Serono — The Culture of Specialty Care,” in this issue.) The usual emphasis in specialty care is on the patient-support programs offering services such as education and reimbursement assistance. But the other side of patient support is answering special unmet needs to expand the therapeutic bounds. Nora Therapeutics may stand alone in completing the circle of treatment for women miscarrying with IVF or suffering repeated, idiopathic miscarriages.
In the words of the company’s president and CEO Jeffrey Tong, “We often use the analogy of pregnancy requiring both a good seed (embryo) as well as soil (endometrium). The industry has invested heavily in drugs, diagnostics, and procedures to optimize the seed. We are the only company now researching ways to optimize the soil.”
It is a fact that current fertility drugs and almost all R&D in the area since IVF became available in 1978 have concentrated on maximizing the quantity and quality of eggs and fertilized embryos. Nora’s drug, NT100, a unique form of G-CSF, is meant to improve a mother’s immune tolerance to implantation and maintenance of a fertilized embryo and thereby reduce the chance of miscarriage. Miscarriages among women who have undergone multiple unsuccessful IVF procedures have grown apace with the procedure. Recurrent miscarriages also affect a growing population.
“While many miscarriages are due to early embryonic defects, women who suffer from a history of multiple miscarriages are, in fact, less likely to have embryonic issues, suggesting a potential maternal issue,” Tong explains. “In fact, these women have been shown to have an imbalance in maternal-fetal immune tolerance.”
Nora appears to have no real competition, either in development or on the market. “Fertility drug pipelines appear to be quite scarce, particularly given the significant unmet need. We don’t view any of the existing fertility drugs as competitive. In fact, they would be highly complementary,” says Tong.
He calls for more industry attention to infertility and miscarriage. If successful, NT100 would be the first drug in 20 years with a new mechanism of action approved to treat infertility. “We hope that in the future we will see more companies developing drugs for this significant unmet need.”
Although infertility may affect as many as one in ten women of childbearing age, or about 6 million in the United States, there are only about 450 IVF clinics in the country. With a specialty care indication, Nora may or may not be a candidate for commercial partnership. Tong gives no sign that it is one, but he does infer the company could, at least initially, go it alone: “Only 250 clinics represent 90 percent of the cycle volume in the United States, and so a targeted, specialty sales force would be ideal to serve the needs of this market.”
Nora wisely keeps a narrow focus. Its single-minded concentration on developing one product for two closely related medical needs befits a company of its size and maturity. Nora is a good example of how small companies can identify and occupy an area evidently left abandoned by the pioneers in the field. Just often enough, the most obvious avenues of innovation are the ones most overlooked.
Headquarters: Palo Alto, CA
Finances: $53M raised to date over two rounds (Series A, Series B)
Partnerships: Major investors: Burrill & Co., Novo Ventures, Prospect Venture Partners, Rho Ventures, Vivo Capital
June 2014: Initiation of Phase 2 RESPONSE study, a randomized, placebo-controlled safety and efficacy trial of NT100 in women with a history of unexplained recurrent miscarriage
April 2014: Close of $18M Series B Financing