Esperion (ESPR) hit a grand slam with triple therapy data, submitting an NDA filing and enrollment ahead of schedule, asserts biotech expert John McCamant, editor of The Medical Technology Stock Letter.
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The Phase II triple combo trial—bempedoic acid(BA)/ezetimibe combo pill plus 20mg atorvastatin (the “038” study) randomized 63 patients 2:1 to the triple combination or placebo—was wildly successful.
The triple combination resulted in a significant 64% reduction (154 mg/dL to 56 mg/dL) in LDL cholesterol from baseline compared to just a 3% reduction on placebo.
The onset of reduction was notably faster with the 3 drug combo than any single agent and the triple combination was well tolerated with no liver enzyme increases and no muscle related AEs distinguishable from placebo.
The safety profile, like the efficacy of this combo trial, was no less than remarkable and represents an excellent first step in this once-daily, triple therapy pill.
Even more impressive, more than 95% of patients saw their LDL levels decline by at least 50% and 90% had their LDL levels down by to at least 70 mg/dL.
This result was highly consistent across patients (shown via waterfall plot). In addition, a significant 48% drop in Creactive protein (sCRP) was seen by the triple therapy.
With the faster than expected enrollment in the clinical programs, management revised their timeline for the bempedoic acid NDA submission by a quarter to Q1:19, up from H2:19 previously. All four global pivotal Phase III studies will be fully enrolled this month.
Three of them, including the 52 week safety study, will readout in Q2:18. The final study, a 52 week efficacy study on top of maximal statin therapy, will read out in Q3:18.
Based on FDA feedback, Esperion will initiate a randomized, double-blind, placebo-controlled bridging Phase III trial in patients (n~350) with hypercholesterolemia and with ASCVD and/or HeFH in Q4:17. Data are expected by YE:18.
Esperion plans to submit an NDA under the 505(b)(2) pathway for the bempedoic acid/ezetimibe combo by Q1:19. This schedule is the same time as the monotherapy filing.
Esperion has plans for a a large pharma partner, but is not beholden to any timeline. The firm remains confident in completing a global and/or geographic deal on favorable terms. However, management stressed that they are not on anyone’s clock to do so sooner versus later.
With the library of positive data being assembled, in our view, time is on their side and with each additional positive data point, the terms will further benefit shareholders.
Lastly, the company raised $150 million in a follow-on offering after the data, bringing their cash position to a healthy $330 million.
ESPR continues to execute at a "Big Pharma, Big Bio" level with a small, emerging company focus. After the successful trial, the company raised $150 million at $49 per share. We reiterate our buy under $55 rating with a $70 target price.
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