Monday December 9th, Moberg Pharma announced results from the first study in the Phase 3 program for MOB-015, where the primary endpoint and the two key secondary endpoints were met. However, the results were somewhat contradictory and the company has clarified the various study parameters as well as their view of the current status and next step.
For example, the company has stated that “the primary endpoint was met and provided that the second study also produces positive results, both studies could form a basis to register the product. However, the outcome of the first study as a whole is more difficult to interpret.”
83 percent of the patients reported improvement
Treatment of onychomycosis involves two main parameters: curing the fungal infection (mycological cure) and restoring the nail’s normal appearance (clinical cure), where the former is objectively determined through laboratory results and the latter is determined subjectively by the investigators. The fact that MOB-015 achieved mycological cure in 70 percent of the patients in this study is exceptional and exceeded our expectations. The study also showed that MOB-015 significantly outperformed the vehicle for the key endpoints and confirmed the product’s ability to rapidly achieve visible improvement. Already by the first follow-up visit, 83 percent of the patients reported improvement.
Clinical assessment of the nails’ appearance was not as positive
The investigators’ clinical assessment of the nails’ appearance was not as positive, however, the company states. “The outcome of the study is surprising, since a high mycological cure is normally over time followed by normalization of the nail’s appearance.”
“To cure 70 percent of the patients from their fungal nail infection is a superior result and better than expected for a topical product. We were however expecting a higher complete cure rate, following the high mycological cure, despite the well-known challenges in demonstrating completely cleared nails at 52 weeks even after the fungal infection is cured. The study confirms the rapid visible improvement experienced by patients, where four out of five patients reported some improvement already at the first follow up visit, and at the end of the study, 33 percent of patients rated their nail to be cured or almost cured”, said Anna Ljung, CEO of Moberg Pharma AB.
Expecting further insight from the ongoing European study
The company is now engaged in reviewing the study results in detail, including all nail photos, to better understand the reasons for the outcome. “We also expect further insight from the ongoing European study, where MOB-015 is being compared to the most commonly used topical treatment. Lessons learned from both studies can later be used to optimize how the product will be used,” they state.
It is too early to draw any firm conclusions from the first study in the Phase 3 program, Moberg Pharma continues, and the company plans to provide an update once additional information is available.
The stock dropped 50 percent
After the announcement on Monday, Moberg Pharma’s stock fell 52 percent and then improved slightly up to 50 percent.
About the trial
The purpose of this randomized, multicenter, controlled clinical Phase 3 study was to evaluate the efficacy and safety of MOB-015 in patients with mild to moderate distal subungual onychomycosis (DSO) affecting 20-60 percent of the great toenail. The study was conducted at 32 sites in the U.S. and Canada and included 365 patients, 246 patients receiving MOB-015 and 119 patients receiving vehicle. Patients received treatment during 48 weeks and had the last follow-up assessment at week 52.
The primary endpoint, the proportion of patients achieving complete cure of their target toenail at 52 weeks, was achieved in 4.5 percent of the patients for MOB-015 and in none of the patients receiving vehicle (p=0.019). Complete cure is a composite endpoint that requires both a completely clear nail and mycological cure. Mycological cure, defined as both negative KOH and negative dermatophyte culture, was achieved in 70 percent of the patients (p<0.0001). Treatment success (mycological cure and almost or completely clear great toenail) assessed by the investigator was achieved in 15.4 percent of the patients (p= 0.0018). In the patients’ self-assessments, a clear majority (83 percent) of the patients completing the study reported improvement from MOB-015 as early as 12 weeks after starting treatment, and at week 52, 33 percent reported their treated toenails were cured or almost cured.
MOB-015 was generally well tolerated. The number of patients with treatment related adverse events was similar for MOB-015 and vehicle, and consistent with our past experience. No safety issues were identified in the trial and no serious adverse events related to MOB-015 were reported.
Photo of Anna Ljung: Moberg Pharma