Anthera Pharmaceuticals' (ANTH) Craig Thompson on Clinical Updates Call Transcript

Anthera Pharmaceuticals Inc. (NASDAQ:ANTH)

Clinical Updates Conference Call

June 29, 2016 08:30 ET

Executives

May Liu - SVP & Finance Administration

Paul Truex - CEO

Craig Thompson - President & COO

James Pennington - CMO

Renee Martin - SVP, Medical Science

Jonathan Barratt - Reader, Department of Infection Immunity & Inflammation, University of Leicester & Honorary Consultant Nephrologist

Analysts

Yigal Nochomovitz - Citigroup

Chris Howerton - Jefferies

Operator

Good day, ladies and gentlemen, and welcome to the Anthera's Conference Call. All lines have been placed on mute to prevent any background noise. After the speakers' remarks, there will be a question-and-answer session. As a reminder, this conference is being recorded.

I would now like to turn the call over to May Liu, Senior Vice President and Finance Administration.

May Liu

Thank you. Welcome to Anthera's conference call. We issued a press release that provided updates for our Blisibimod and Sollpura clinical programs on June 28, 2016. This press release is available on our website. For additional data, allow me to refer to you our corporate presentation, also available on our website under Events & Presentations at www.anthera.com.

During the course of this conference, we will state our beliefs and make projections and other forward-looking statements regarding future events. We wish to caution you that such statements or predictions and expectations and actual results may differ materially. We refer you to our publicly filed SEC disclosure documents for a detailed description of the risk factors affecting our business, most recently, our Annual Report on Form 10-K for the year-ended December 31, 2015 and Quarterly Report on Form 10-Q for the quarter ended March 31, 2016.

This document identifies the important factors that could cause our actual results to differ materially from our projections and other forward-looking statements. These risk factors include regulatory, operating expenses, intellectual property, clinical development and other risk factors relating to the business. During today's call, we will be discussing the results of the BRIGHT study and CHABLIS 7.5 study for Blisibimod and the SOLUTION study with Sollpura. We will then open the call to your questions.

Joining me on the call today is Mr. Truex, our Chief Executive Officer; Mr. Craig Thompson, our President and Chief Operating Officer; Dr. Pennington, Jim Pennington, our Chief Medical Officer; and Dr. Renee Martin, our Senior Vice President, Medical Science. As well, we've asked Dr. Jonathan Barratt to join today's call. Dr. Barratt is a leader in the Department of Infection Immunity and Inflammation at the University of Leicester and an Honorary Consultant Nephrologist in the John Walls Renal Unit, Leicester General Hospital. Dr. Barratt has been involved with the BRIGHT study as an investigator and has helped improve [ph] our strategy in an IgA nephropathy.

I will now hand the call over to Mr. Craig Thompson. Craig?

Craig Thompson

Thank you May, and good morning to everyone. Yesterday we issued a press release outlining the interim outcome from the BRIGHT SC clinical study and other updates on our clinical program including the early completion of screening and the SOLUTION study and initial screening in our second Phase 3 study for Blisibimod and Severe Lupus. In addition to today's call we have provided supplemental details as May mentioned on our corporate presentation that can be viewed at www.anthera.com.

Let me start today's call with what we believe are encouraging results from interim analysis of the Phase 2 BRIGHT SC proof-of-concept study of Blisibimod and patients with IgA nephropathy or IgAN. The purpose of the study is to determine the safety and efficacy of sub-cutaneous Blisibimod compared with placebo and patients with biopsy proven IgAN. The study enrolled 57 patients, 47 of whom completed assessments through a minimum of 24 weeks. Patients with persistent proteinuria between 1 grams and 6 grams per 24 hours, prior to enrollment, were randomized to receive either Blisibimod or matching placebo over background of stable blood pressure therapy.

This Phase 2 clinical study was intended to determine if Blisibimod could meaningfully reduce levels of patient's proteinuria as either a partial or complete response. Dr. Pennington will discuss the specific findings of the study. However, having seen all of the data from the BRIGHT study in this patient population with proteinuria and established kidney disease we have encouraging data on proteinuria and other biomarkers to suggest a potential for slowing, maybe even preventing disease progression in Blisibimod treated patients or those on placebo continue to worsen.

I'll now hand it over to Jim to review the clinical trial results. Jim?

James Pennington

Thank you, Craig and good morning everyone. As Craig suggested, we find the data from the BRIGHT study highly informative and encouraging that Blisibimod may in fact slow and/or potentially prevent progression of proteinuria over a longer period of time.

In summary, 24 week interim data from the BRIGHT SC study demonstrated a numerical reduction in proteinuria of Blisibimod versus placebo treated patients from week 1 through 24. But the study did not meet the pre-defined statistical primary endpoint complete or partial response with the 24-week time point. However, data from the study beyond week 24 and through week 108, almost two years, demonstrated an increasingly large separation in proteinuria, favoring the Blisibimod treated group compared to placebo.

While the finding was not statistical, the magnitude of the difference at the end of the study is quite large. Supporting these findings, patients treated with Blisibimod demonstrated statistical improvements in total B-cell counts and consistent reductions in the Immunoglobulin A, G and M, as early as week 8 which is similar to our previous studies in Lupus.

Given the increasing separation of the proteinuria data after 24 weeks of dosing and the clear improvement on immunological markers relevant to IgAN, after treatment with Blisibimod, we have elected to continue the study through weeks 48 time point to gain further information about the potential of Blisibimod to prevent these patients. Blisibimod was safe and well tolerated with the most common adverse effects being common cold, upper respiratory tract infection, back pain and rash at the injection sight. The study did have two patients who progressed to end-stage renal disease, one in the Blisibimod arm and one in the placebo arm.

Encouragingly, our patients stayed on Blisibimod therapy than placebo throughout the two-year portion of the study. Dr. Barratt, perhaps you could provide your thoughts as well.

Jonathan Barratt

Thank you, Jim. So the BRIGHT study hypothesized that patients with IgA nephropathy would benefit by reducing the immunological element of the disease specifically, reducing B-cell population numbers and the associated immunoglobulin levels, so thus by effecting these pathogenic pathways this could lead to a benefit in patients by reducing proteinuria.

Now based on the data from the BRIGHT study that is available, this hypothesis is still intact, yet we may need to consider the BAFF inhibition will prevent or slow worsening of the disease which is still a worthwhile impact for patients with limited treatment options. The longer term proteinuria observations based beyond week 24 through to two years seems to suggest with the patients with IgA nephropathy treated with placebo may continue to suffer disease worsening which is what we would expect and in this case demonstrating an increase of 1.5 grams per 24 hours in proteinuria over the two year follow-up period.

Conversely, those patients who were treated with Blisibimod demonstrated slight reduction of around 0.2 grams per 24 hours in proteinuria from baseline during the same time while the latter time points are very small and non-statistical due to the small sample size, the magnitude of difference is large and would be clinically meaningful if validated in the larger parent study. Hence, I agree Anthera's decision to collect additional data to determine if this effect of Blisibimod is in fact real. Back to you, Jim.

James Pennington

Thank you, John. Potentially holding disease progression, in IgA nephropathy where Blisibimod particularly in those with advanced renal impairment could be a breakthrough for those patients. We've learned a lot from the BRIGHT study including that our selected primary endpoint remission did not contemplate the hypothesis that Blisibimod may prevent worsening of the disease in a population for which worsening is the norm. As I mentioned in the press release, the longer term proteinuria and immunological data from the study is encouraging and provides a strong rationale to continue the study.

Finally, we conducted additional retrospective analysis of the data from BRIGHT in light of emerging research regarding measurements of efficacy and IgA nephropathy. According to recently published article in nature by Dr. Jürgen Floege which is called Time Average Proteinuria is thought to be the most reliable predictor of outcomes in IgA nephropathy. Time Average Proteinuria data from the BRIGHT study is also directionally consistent with our other findings. Taken together all of this data has led to our optimism, that should these trends continue for all patients, Blisibimod could prove meaningful therapy to slow the progression of IgA nephropathy.

Let us cover the rest of the Anthera release and then we will open up for any questions regarding our plans for IgA nephropathy. Craig?

Craig Thompson

Thank you, Jim for the update on the BRIGHT study. Last night we also announced that we have closed patients screening for the Phase 3 SOLUTION clinical study evaluating efficacy and safety of Sollpura compared to an approved porcine-derived pancreatic enzyme replacement therapy with the treatment of exocrine pancreatic insufficiency in Cystic Fibrosis patients. Screening on the study was closed ahead of schedule and we expect top line data by the end of this year. As this is the only clinical study required for responding to the U.S. FDA complete response letter, we anticipate re-submitting the BLA to the U.S. FDA in 2017 pending completion of the manufacturing program.

Finally, yesterday we announced initiation of the CHABLIS 7.5 clinical study, our second Phase 3 study examining the efficacy and safety of subcutaneous Blisibimod in subjects with Severe Systemic Lupus or SLE, with or without nephritis, and we have successfully screened our first patient. The target enrollment population for this study is informed by responder traits identified in our Phase 2 PEARL study, as well as large Phase 3 programs with our fast inhibitors. In these data patients with high levels of double stranded DNA and low levels of complement, T3 and T4 specifically, consistently demonstrated even higher levels of clinical response. Importantly, in a disease where physicians have difficulty identifying the best patients to treat with the B-cell activating inhibitor like Blisibimod, these standard markers of disease will provide an additional objective diagnostic on which physician can rely on for optimizing therapy. We are making great progress on the data analysis on the CHABLIS SC1 study.

The results from the BRIGHT SC clinical certainly indicate we are on target with B-cell reductions, immunological reductions and trends towards reduced proteinuria with no new safety findings. Our preparations for topline safety and efficacy data for CHABLIS SC1 is targeted for third quarter which efficacy data from the population of patients that meet the CHABLIS 7.5 enrollment criteria to follow shortly thereafter.

In summary, we are very excited from the emerging data from the BRIGHT SC study and continue the program in an expansive way to further our understanding of how Blisibimod could become a meaningful therapy for IgA. The SOLUTION study has completed screening and we anticipate having the data by the end of the year. Following the immunological and proteinuria data from the BRIGHT study we're increasingly excited to see the CHABLIS SC1 data in Q3.

Operator, at this time we would be happy to take few questions. Can you please open the line for audience questions?

Question-and-Answer Session

Operator

Thank you. [Operator Instructions] Our first question comes from Yigal Nochomovitz with Citigroup. Your line is now open. Please go ahead.

Yigal Nochomovitz

Hi, thanks. Could you just clarify some of the details on the data? So I think the physician mentioned 0.2 nanograms for 24 hours. So is that -- what exactly does that mean? Is that linear, meaning we just multiply that by the number of days and say what -- just to say again, what was the rate of decrease on the placebo?

Craig Thompson

So Jim, would you like to take that question?

James Pennington

Sure, I'd be happy to. Just to clarify what we're talking about, we're looking at proteinuria measurements over -- up to a two year period. Patients come in every few months and have their urines analyzed for protein, and as you know, protein in the urine is a sign of renal disease; more protein, worse renal disease. And what we found over the course of the study was that the Blisibimod treated group, it was basically flat over this period of time. They've had a small, maybe 0.2 grams reduction in their proteinuria and to give you an idea of what that means, the proteinuria values are expressed as per 24 hours or using a protein-creatinine ratio surge to measure, it basically gives you the same type of data.

Whereas the placebo group over this period of time continuously rose, each time it was measured the proteinuria was higher. There was no crossing of lines, it was consistently elevating overtime and by the end of the two year period, although the numbers are very small, and of course will be being increased as we continued the study, the group that received placebo had increased their proteinuria by about 1.5 gram value and that is clinically meaningful whereas the Blisibimod treated group had basically stayed below, stayed at their starting point or 0.2 below which is small but it is not up suggesting at an idea of those curves they are diverging with placebo going up continuously.

Yigal Nochomovitz

Okay, got it. Now do you know that if it's that big at -- I mean do you know if it's at 48 weeks from what you are seeing in the longer term data?

James Pennington

Just to be clear, are you asking what the value is? Oh, I see, what you're asking really is where we will see it when we do the next analysis in 48 weeks? Do we expect to see these trends strengthen, if anything? Is that what you are asking?

Yigal Nochomovitz

Yes, I think you referenced that you had even patients up to a 108 weeks. So I am just wondering if you have enough power, enough patients who could have made it to 48 weeks to run the statistics and see where you stand?

James Pennington

I see your question, okay, good. Yes, the statistics have been run at every time point, all the way up to two years and while some of them come close, none of them reached statistical significance at this time. Keep in mind the numbers are pretty small at this point.

Yigal Nochomovitz

Okay. And how many -- what's the size of the sample size at 48 weeks right now?

James Pennington

Renee, I don't have that number right in front of me, do you happen to have the chart?

Renee Martin

So the total sample size right now at week 48 is 33 subjects. So it's pretty small.

Yigal Nochomovitz

Okay. And the 48 week analysis, was that the sort of, part of the initial clinical trial protocol or was that something -- is that an amendment to the initial structure of the study?

James Pennington

The protocol prospect that we called for the serial proteinuria to be collected, so all of this is prospectively defined.

Yigal Nochomovitz

Okay.

Craig Thompson

Yigal, I would also refer you to our website where we have updated the corporate presentation and provided a lot of the data as well.

Yigal Nochomovitz

Okay, alright, perfect. And then on Sollpura, can you just provide any quick updates on how things are going with the manufacturing with respect to improving the volume of reducing the [indiscernible] for Sollpura?

Craig Thompson

So from a manufacturing perspective of Sollpura, as we mentioned the SOLUTION trial, we should have results from that by the end of this year. The SIMPLICITY trial is still on-track with data expected in Q1. And then from a manufacturing perspective, we are also on-track from a manufacturing perspective and are getting ready to begin full-scale manufacturing in the near-term but all plans are in place and everything is looking good.

Yigal Nochomovitz

Okay, thanks very much. Appreciate it.

Craig Thompson

Thanks, Yigal.

Operator

Thank you. Our next question comes from Chris Howerton with Jefferies. Your line is now open. Please go ahead.

Chris Howerton

Hey, good morning. I think you got to several of the questions I was going to ask, but I guess, just to clarify, and perhaps you could remind us what the definition of complete and partial response was in the context of proteinuria? And kind of help us understand how these flat to 2.0 gram reduction relative to the 1.5 gram increase in placebo could be couched within those definitions?

Craig Thompson

Sure. Thanks Chris for the question and Renee can cover the complete and partial response for you.

Renee Martin

Sure. So the definitions that were used were for partial response. Patients needed to meet a proteinuria of less than 1.0 gram because complete response is actually dependent on your baseline proteinuria value. So if you came in with a proteinuria of 1.0 gram to less than 2.0 grams, then you'd be a complete responder if you achieved less than 1.0 gram and 50% reduction in proteinuria. If you came in with a baseline of 2.0 grams or high -- sorry, yes, 2.0 grams or higher, then you'd be a complete responder if you had achieved less than 1.0 gram or 50% reduction. That makes sense?

Chris Howerton

Yes, right. So I mean it's basically, you're not giving extra care for people that already had higher baseline levels. So I guess how should we think about the numerical values that -- I apologize, I forgot what the doctor's name was -- in terms of describing the reduction or the increase, is that really driven by patients that had the higher baseline values or is that pretty evenly spread across the different kinds of presentation type?

Craig Thompson

Jim, would you like to answer that question?

James Pennington

Sure, thanks Craig. I think what we're doing here is adjusting our hypothesis about how Blisibimod treats this disease. Our original hypothesis was that Blisibimod treatment would lower the proteinuria. In fact, we had some hope of course that it might even cure the disease. I think that our observations so far with the data in hand suggests a different hypothesis and that is, that Blisibimod holds -- puts a finger in the dyke [ph], it holds the patient from progression but it may be asking too much to bring them down to normal. Meanwhile the placebo group, not receiving treatment goes along with deterioration. And so what we appear to be doing here is preventing progression of the disease, and that's a good thing and as I mentioned, the magnitude of the data suggests this could be the real thing to help patients but we need more information, larger numbers, and we need to fill in the statistical issues as the study winds out for the later months.

Chris Howerton

Sure, okay. Now that makes sense. Okay, and then, I guess just another quick one. Is there any kind of data that you would point to that you've seen with this interim analysis that gives you more hope or makes you more positive on Blisibimod and Lupus patients?

Craig Thompson

Jim, would you like to take that as well because I think we have a lot of encouraging data there.

James Pennington

Yes, I'll take it initially but Renee did some of the relief seminal [ph] analyses on the proteinuria and the renal issues in Lupus so she is our true expert on this. But yes, these are both autoimmune diseases, they both have immune complexes attacking the kidney, leading to damage as indicated by proteinuria. Again IgAN, it's the IgA immune complexes and Lupus, it's more likely IgG. But yes, I think there really is mechanistically some overlap and we are encouraged. Renee, would you like to add?

Renee Martin

Jim, I think you nailed it. The only thing I would add to that is the data here show that we absolutely are nailing the BAFF mechanisms. The signature that we see for the effect on B-cells is exactly what you'd expect for BAFF inhibitions that tells you, you have a relevant dose and the effects are happening very quickly and are profoundly different from placebo in the B-cell signature. And that effect is echoed in the immunoglobulin signals. So I think combined with the proteinuria data, I think all of these points towards an encouraging position to be waiting for the CHABLIS trial to read out.

Chris Howerton

Okay, great. I appreciate you taking my questions. Thank you.

Craig Thompson

Thanks for the questions, Chris.

Operator

Thank you. [Operator Instructions] Our next question comes from the line of Edward Nash with SunTrust. Your line is now open. Please go ahead.

Unidentified Analyst

Hi, this is Jung Song [ph] for Edward. Thank you for taking the questions. So just first, a follow-up question about the 48 week time point for the data. How many patients do you expect to have at 48 weeks?

Craig Thompson

So Renee, could you answer that question, please?

Renee Martin

We should expect something more like 20 per arm, thereabouts at the week 48 time point. But also bear in mind as we carry the cohort forward, it means all of the major time points are also increasing in sample size.

Unidentified Analyst

Okay. So if I remember correctly, I think the original target of enrollment for this study was higher than 57, and that was before the termination of the Zenyaku program -- sorry, agreement. And you cited the small study size for the lack of significance. So I wonder if you had been able to enroll other patients, would you have been able to meet the statistical significance based on the current magnitude of change?

Paul Truex

Craig, its Paul. I certainly can help with that. Hey Jung [ph], how are you? It's Paul Truex. Yes, the short answer is, given the magnitude of the effect -- and again, I would refer you to the slide deck that's been recently updated on the website that contains a lot of the data that we're referring here to -- referring to here today. I think if you saw that magnitude of effect and we had 100 patients an arm, there would be no doubt that the statistical significance would be achieved given that magnitude. And that part of the validation or the thought process around extending the studies through 48 weeks is just getting more comfort that even in a slightly larger cohort that data remains consistent throughout that one-year time point. So great question, and yes, I think if we had a 200 patient study that we anticipated doing with Zenyaku, in fact that would have hit.

Unidentified Analyst

Okay. And the last question is about your plan for the program. So assume that you see even more convincing promising data at the 48-week time point, are you going to proceed without an agreement -- or sorry, a collaboration agreement or you still probably will secure a collaboration before you move forward?

Craig Thompson

Great question. So we're having general discussions around Blisibimod, the compound both for Lupus trial as well as IgA nephropathy, those discussions continue to move forward. I think when our partners see this data, they will also be very excited about the data and the prospect of moving forward. So our anticipation would be that we would eventually find a partner for the asset to finish the clinical development program.

Unidentified Analyst

Okay, great. Thank you for taking the questions.

Operator

Thank you. Our next question comes from the line of Yigal Nochomovitz with Citigroup. Your line is now open. Please go ahead.

Yigal Nochomovitz

Okay, hi, thanks. I just have one quick follow-up. When you run the analysis at 48 weeks, are you going to continue to adhere to the p is less than 0.1 threshold as you say in the press release or are you going to switch to a more difficult 0.05? Thanks.

Craig Thompson

Renee, can you cover the statistical powering at the 48-week analysis?

Renee Martin

Sure. So really the plan is to continue to conduct the analysis as we've already done it, so just to extend onwards. So we'd retain the p-value of one-sided P1. Obviously, we will report the values so if you see a value that's less than 0.05, it's even better. But yes, that's the plan.

Yigal Nochomovitz

Okay, thanks a lot.

Operator

Thank you. Ladies and gentlemen, this is all the time we have for questions today. I will now turn the call back over to Craig Thompson, President and Chief Operating Officer for closing comments.

Craig Thompson

So thank you very much for dialing-in today. We're very excited about the data from BRIGHT, the early enrollment of the SOLUTION trial, and the overall programs continue in the right direction. We look forward to catching up with everybody on our next call. And thank you for calling-in today.

Operator

Ladies and gentlemen, this does conclude today's program. And you may all disconnect. Everybody have a wonderful day.

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