Daisy Program Offers Positive Results
On May 24, 2010, Neurocrine Bio (NBIX) announced that the Daisy PETAL (Study-901) program provided statistically significant results in all top-line primary and secondary endpoints testing the company’s proprietary, orally-active non-peptide Gonadotropin-Releasing Hormone (GnRH) receptor antagonist, elagolix, in patients with endometriosis.
In addition, the company confirmed that the daily scales for menstrual and non-menstrual pelvic pain, developed with extensive input from the U.S. FDA and patients, functioned well in a clinical trial setting. The Daisy PETAL (901) study enrolled 137 endometriosis subjects into one of two treatment groups; elagolix 150 mg (n=66) or placebo (n=66) once daily for two months of treatment, in a double-blind design.
Top-line results confirm that elagolix is associated with statistically significant reduction in the two co-primary endpoints, Dysmenorrhea and Non-Menstrual Pelvic Pain, and the exploratory endpoint, Dyspareunia, when compared to placebo based on the ITT population.
Management also conducted a responder analysis that demonstrated clinically meaningful improvement of 30% or greater from baseline on all three endpoints (ITT population). Secondary endpoints also demonstrated statistically significant improvement from elagolix subjects versus the placebo on validated efficacy outcome assessments.
During the phase II program, elagolix demonstrated encouraging safety and tolerability. Discontinuation due to adverse events (AEs) was low at 4.4% for elagolix vs. 1.4% for the placebo. The most common adverse event reported more often with elagolix than with placebo was nausea (7.4% elagolix; 2.9% placebo). This was consistent with what we saw in previous clinical studies.
Importantly, there were no elagolix treatment-related serious adverse events (SAEs). We note that subjects are continuing for an additional four months of open-label elagolix treatment and assessments program. This 24-week open-label data should be available in late August 2010.
Next Step – Meet with the FDA
Now that the top-line data from Daisy PETAL has been reported, management will request an end of phase II (Type-B) meeting with the Division of Reproductive Products at the FDA. We expect this request (filing) will take place in late June 2010. The FDA usually responds within 60 days, and our best guess is that the meeting will take place in late August / early September 2010.
In total, Neurocrine should be able to sit down with the FDA and offer up data from 6 phase II programs (2 phase IIa and 4 phase IIb) on roughly 1,000 patients. Concurrent to the end of phase II meeting request, management will file a draft proposal for a special protocol assessment (SPA) for the phase III design.
The company believes that the first round of discussions on the proposed SPA will take place with the agency at the end of phase II meeting. The final SPA agreement should be in hand by late in the Q3-2010. This should allow management, and its partner, to begin a phase III program before the end of the year.
We expect that the pivotal phase III program will include two large-scale (n~500) patient programs with co-primary Dysmenorrhea and Non-Menstrual Pelvic Pain endpoints. The exploratory Dyspareunia endpoint may also be tested in the phase III program. There will also be a long-term (n~400) safety program. Depending on management’s European strategy, one of the two phase III programs may include a head-to-head non-inferiority program vs. Lupron for the European submission.
Neurocrine is actively seeking a development and commercialization partner on elagolix. Our best guess is that management will be able to secure this partnership this summer, in-time for the partner to get involved in the end of phase II meeting and SPA discussions with the FDA.
This is the most significant catalyst to drive the shares higher. Potential partners are firms that have strong Women’s health focus, including several large-cap pharmaceutical players. Although, we note the product could also be very effectively marketed by a specialty pharmaceutical company given that a small specialty Ob/Gyn sales force of 200 to 250 representatives can effectively target greater than 90% of the market.
Neurocrine Bio is one bright spot today in an otherwise down market. We think things are positing well for the company with elagolix. Once a partnership has been secured and the phase III trial initiated, Neurocrine becomes an attractive investment opportunity in 2011 based on the release of the pivotal data.
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