Seeking Alpha
We cover over 5K calls/quarter
Profile| Send Message|
( followers)  

Synthetic Biologics Inc. (NYSEMKT:SYN)

Q1 2014 Earnings Conference Call

May 15, 2014 8:30 am ET

Executives

Jeff Riley – President, Chief Executive Officer

Evan Ballantyne – Chief Financial Officer

Kris Maly – Vice President, Corporate Communications

Analysts

Jason Kolbert – Maxim

Keay Nakae – Ascendiant Capital

Keith Markey – Griffin Securities

Operator

Good morning and welcome to Synthetic Biologics First Quarter 2014 Investor Conference call. All participants will be in listen-only mode. Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today’s presentation, there will be an opportunity to ask questions. To ask a question, you may press star then one on your touchtone phone. To withdraw your question, please press star then two. Please note this event is being recorded.

At this time, I would like to turn the call over to Kris Maly, Vice President, Corporate Communications at Synthetic Biologics. Kris?

Kris Maly

Thank you Betty, and good morning everyone. Welcome to Synthetic Biologics’ First Quarter 2014 Investor Conference call. Today I’m joined by our CEO, Jeff Riley, and our CFO, Evan Ballantyne. Pre-market this morning, Synthetic Biologics issued a press release reporting its first quarter 2014 financials and summarizing recent operational highlights. That release can be found on the Investors section of our website. During our call today, Jeff will begin with an overview of our business objectives and review the recent Phase II data from our MS program. Evan will then provide a brief overview of our financials for the three months ended March 31, 2014. Jeff will conclude our prepared remarks with a progress update on our anti-infective programs and on the advances we are making toward our upcoming milestones. After the formal portion of the call, we will offer an opportunity for Q&A.

In addition to the phone line today, this call is being streamed live over the internet and the webcast replay will be archived on our website for 30 days. During the call, we will be making forward-looking statements regarding Synthetic Biologics current expectations and projections about future events. Generally, the forward-looking statements can be identified by terminology such as may, should, expects, anticipates, intends, plans, believes, and estimates, and similar expressions. These statements are based upon current beliefs and expectations and assumptions and are subject to a number of risks and uncertainties, including those set forth in Synthetic Biologics filings with the SEC, many of which are difficult to predict. No forward-looking statements can be guaranteed and actual results may differ materially from such statements. This information in this call is provided only as of the date of the call, and Synthetic Biologics undertakes no obligation to update any forward-looking statements contained on this call on account of new information, future events or otherwise, except as required by law.

With that, I’d like to turn the call over to Jeff.

Jeff Riley

Thanks Kris, and good morning everyone. Since the beginning of the year, we have continued to achieve milestones and advance Synthetic Biologics programs. We remain focused on developing our core pipeline of novel anti-infectives and we are committed to executing our aggressive R&D strategy. We are proud to say that after two years of working to build a world-class organization at Synthetic Biologics, we are now on the doorstep of advancing some of the most innovative anti-infective programs into the clinic. These include our C. diff, our constipation form of IBS, and pertussis programs, each of which we believe represent multi-billion dollar opportunities for the company. I’d like to thank our long-term investors for supporting us along the way.

We also believe we have added significant value to Synthetic Biologics by successfully demonstrating the therapeutic potential of oral estriol in the Phase II exploratory trial. Our goal continues to be entering into a strategic partnership for this program. We are in active business development discussions with potential partners at this time and I will discuss how the recent positive data support our efforts to monetize this asset. Given the market’s reaction to the Phase II MS data, it seems there may be some lingering confusion about the findings of the study that could be impacting the value of our stock. While Synthetic Biologics is focused on building shareholder value by advancing our pipeline, we are also committed to protecting shareholder value, which is why I want to discuss the Trimesta data at the onset of this call.

To quickly summarize recent events, the study’s lead investigator, Dr. Rhonda Voskuhl from UCLA, presented positive top-line efficacy and safety results from the Phase II trial evaluating adjunctive Trimesta in women with relapsed remitting MS, the most common form of MS, at the American Academy of Neurology’s Annual Meeting two weeks ago. Subsequently, Synthetic Biologics and Dr. Voskuhl hosted a conference call on April 30 to discuss the top line findings in more detail. The UCLA-led Phase II study was designed to show statistical significance at 12 months for the MS relapse rate reduction in patients treated with Trimesta plus Copaxone compared to patients given placebo plus Copaxone. The trial was only powered to trend – I repeat, to trend – towards statistical significance at the 24-month time point. According to the protocol, the results of the top line data demonstrate that Trimesta met the pre-specified goal of the study with rapid onset of activity observed for Trimesta plus Copaxone compared to placebo plus Copaxone. Furthermore, because the burden is so high for showing efficacy as an adjunctive therapy on top of an already effective standard of care, Dr. Voskuhl and her team hope to see an approximately 29% reduction in MS relapse rate, per the study protocol.

So to put the preliminary data in context, the fact that we saw a statistically significant 47% decrease in relapse rates at 12 months of therapy – that’s a P value of 0.03 powered for significance level of 0.05 – as well as a clear trend toward a 32% reduction at 24 months – that’s a P value of 0.15 powered for significance at 0.10 – far surpassed our investigators’ expectations, and they are extremely happy with the study results, as are we. These findings are highly encouraging not only from a clinical perspective; they are also very positive for advancing our discussions with potential partners which, from a strategic business standpoint, has always been the primary purpose of completing this study. We have successfully created a strong clinical case for advancing Trimesta further as a novel treatment for women with MS, which may drive higher valuations among potential partners. Indeed, since the data were presented, we have accelerated and expanded our partnership discussions as a result.

To that end, it is worth reiterating that the Trimesta study also demonstrated a clinically significant near-normalization of cognitive scores at 12 months of therapy in women taking Trimesta plus Copaxone. This outcome, as you may imagine, is of high importance for MS specialists and patients, and we believe it is the result of oral estriol’s unique neuroprotective effect. No other MS drug currently on the market has demonstrated significant neuroprotection and the potential positive impact on cognition. Dr. Voskuhl and her potential partners are very intrigued. In fact, Dr. Voskuhl emailed me yesterday, stating only further emphasis is the fact that this data leads the field into new paths of treatment for MS that are not merely anti-inflammatory but also neuroprotective. Such neuroprotective effects of Trimesta may ultimately be relevant to Alzheimer’s disease and other neurodegenerative diseases characterized by cognitive decline – unquote. We look forward to evaluating this exciting clinical result as well as exploring potential unique regulatory strategies going forward.

In summary, the multiple positive efficacy signals from this placebo-controlled study are strong. Neurologists and MS specialists saw exactly what they hoped to see from this study and more in the area of cognitive improvement. Dr. Voskuhl, Synthetic Biologics, as well as several potential partners believe we are on the right track in developing Trimesta as an adjunctive MS therapy for women. We look forward to updating shareholders on our discussions with potential MS partners as well as our plans to advance Trimesta in the clinic based on these clinically significant Phase II results.

In parallel, we are enrolling women into a separate placebo-controlled Phase II study focused exclusively on cognition in MS patients. Women in this trial receive either oral estriol or a matching placebo in addition to an FDA-approved standard MS treatment to include Copaxone, Avonex, Betaseron, Extavia, Rebif, Gilenya, Aubagio, and Tecfidera.

You now see that the fundamentals of Synthetic Biologics’ business have been strengthened by the recent MS data. We believe we have added significant intrinsic value to the company by demonstrating the therapeutic potential of oral estriol, which further supports our efforts to attract a strong strategic partner to accelerate development of this innovative therapy for relapsing remitting MS.

I will now hand the call over to Evan to review our first quarter financial results, after which I will provide an overview of our recent achievements and expected milestones for our world-class pipeline of novel pathogen-specific anti-infectives. Evan?

Evan Ballantyne

Jeff – thanks, and thanks everybody for attending the call today. Synthetic Biologics’ first quarter 2014 financials were included in a press release which was distributed over the newswire earlier this morning. The company’s March 31, 2014 10-Q will be filed with the SEC later this afternoon.

For both the three months ended March 31, 2014 and March 31, 2013, our general and administrative expenses were $1.1 million. Included in these numbers were non-cash charge related to stock-based compensation of $255,000 for the three months ended March 31, 2014 compared to $353,000 for the same period a year ago in 2013. Research and development expenses increased to $2.7 million for the three months ended March 31, 2014 compared to $1.1 million for the same period in 2013. The increase of 143% is primarily the result of increased program costs associated with expanded research, development and manufacturing activities within our anti-infective pipeline, including our C. diff, CIBS and pertussis programs. Non-cash charges related to stock-based compensation were $107,000 for the three months ended March 31, 2014 compared to $104,000 for the same period a year ago.

I’d like to add that our Phase II MS trial evaluating Synthetic’s oral estriol drug candidate, Trimesta, which Dr. Voskuhl from UCLA recently reported positive top line data, is being conducted under an investigator-initiated IND and is funded by $8 million in grants received from the MS Society and the NIH. I was extremely pleased with the investigator-initiated Phase II MS trial results. These results will allow the company to pursue potential partnering opportunities or to move forward into Phase III trials with potential collaborators. In my opinion, that is the definition of a successful trial. Also, our pertussis and acinetobacter monoclonal antibody development programs are being funded from a prepaid account established with Intrexon Corporation in 2012.

Cash and cash equivalents as of March 31, 2014 were $11.2 million compared to $14.6 million as of December 31, 2013. As CFO of Synthetic Biologics and in my previous position as CFO of Clinical Data, which coincidentally was acquired by Forest Labs for $1.3 billion, I have had the privilege of working with some very novel drug candidates which have the potential to have an impact on human health.

To conclude my part of this discussion is, as Jeff said, we believe that our C. diff and CIBS programs represent multi-billion dollar program opportunities for Synthetic Biologics. These clinical programs are amongst the most impressive opportunities I have ever seen, and I am extremely excited by the company’s near and longer term prospects.

At this time, I’ll turn the call back to Jeff for our overview of our core products.

Jeff Riley

Thanks Evan – couldn’t agree more with your assessment. In fact, it’s my hope that the details I’m about to review will further support the assessment and spread management’s excitement to our shareholders and other listeners on the call this morning.

Our promising portfolio of novel anti-infective candidates continues to represent our core focus and the primary mid to long-term growth drivers for Synthetic Biologics business. As noted earlier, we have been extremely successful in building a robust pipeline over the last two years of truly innovative biologic and drug candidates, each designed to address specific high needs serious infections and diseases. We continue to operate at the forefront of today’s research by employing novel approaches and technologies to target specific pathogens such as Clostridium difficile, methanogens, pertussis and acinetobacter. Our plan is to remain aggressive in terms of preclinical and clinical R&D, leveraging the strength and commitment of our academic and corporate collaborators.

Through these efforts, I’m proud to say that we have continued to make important strides and we are on track to complete our ambitious goal of moving three of our novel anti-infective development programs into the clinic over the next 12 months. I’d like to begin with an overview of our Clostridium difficile project.

Synthetic Biologics’ second generation oral enzyme therapy, SYN-004, is designed to protect the normal gastrointestinal microflora or microbiome from the unintended effects of IV antibiotics and potentially preventing the devastating effects of hospital-acquired C. diff. C. diff is a multi drug-resistant bacterium that has surpassed MRSA as the number one hospital acquired infection in the United States. In fact, in 2013 the CDC identified C. diff as an urgent public health threat, particularly given its resistance to many drugs used to treat other infections. As media coverage increases regarding the devastating effects of C. diff infection, Synthetic Biologics is working to address the significant unmet medical need.

SYN-004 is the first point of care therapy designed to prevent C. diff infection and works by neutralizing the antibiotics in the gut. It is intended to protect and maintain the balance of bacterial flora in the GI tract, thereby preventing C. diff infection from happening in the first place. SYN-004 is believed to have a similar profile to its first generation predecessor which successfully demonstrated protection of the gut microbiome in successful Phase II trials in addition to eliminating antibiotic-associated diarrhea.

Last week, we announced additional international patents providing composition of matter coverage for various aspects of the C. diff program. Specifically, we had two patents validated in Europe and received a Notice of Allowance for an additional patent in Australia. These new patents strengthened Synthetic Biologics’ C. diff IP portfolio, which now includes more than 25 issued U.S. and international patents. We remain on schedule to initiate a 28-day bridging tox study next month. Following the results of this study, we are expecting to file our IND and initiate Phase Ia and Phase Ib clinical trials during the second half of this year, with preliminary top line data expected by year-end of 2014. A Phase II efficacy study is expected to begin in the first half of next year.

As we announced previously, we have initiated CGMP manufacturing of SYN-004 to support these preclinical and clinical studies. There are currently no therapies – no therapies – approved to prevent C. diff infections, and we look forward to continuing our efforts to develop SYN-004 to address this multi-billion dollar potential market. This is a huge opportunity to make a significant positive impact on the global public health system and to save lives. When disruptive innovation is introduced into an existing paradigm, the rewards can always be very great.

The next program is our pertussis program. Next, I’d like to discuss the antibody-based therapy for pertussis, also known as whooping cough, which is a potentially deadly disease to newborns and the elderly and infects nearly 50 million people worldwide. Even though most adults receive a vaccine, the incidence of pertussis has continued to rise in the United States, so the need for new therapies remains high. There are no existing therapeutics for pertussis, so Synthetic Biologics’ SYN-005 candidate will be the first one.

Tragically, by the time infants present with the characteristic whooping or worse symptoms, antibiotics can do little to change the course of the disease, and the only available therapy is supportive care. Worldwide, as noted earlier, there are approximately 50 million cases of pertussis each year, leading to about 300,000 deaths, primarily in infants. Part of the reason antibiotics are ineffective is that while they can eliminate the pertussis bacteria from the respiratory tract, they do not neutralize the toxins secreted by the bacteria which play a major role in the pathology of the disease. Multiple lines of evidence suggests that neutralizing the pertussis toxins may mitigate the course of the disease, shorten ICU and hospital stays, prevent long-term disabilities, and most importantly diminish mortality.

In collaboration with Intrexon Corporation as well as our academic partner, the University of Texas at Austin, Synthetic Biologics is advancing this combination antibody therapy SYN-005, which demonstrated effectiveness against the pertussis toxin in both in vitro and in vivo studies. The two humanized antibodies that comprise SYN-005 function through complementary mechanisms and appear to be highly synergistic in neutralizing the pertussis toxin. In previous mouse studies that utilized a virulent pertussis strain isolated from a critically ill newborn, treatment with our antibodies was believed to both individually and in combination diminish the bacterial burden in the lungs and completely mitigate elevation of the white blood cell count that is characteristic of the disease.

A few weeks ago, we reported additional positive efficacy data from two non-human primate studies that further support the findings from the mouse studies. SYN-005 was associated with a favorable decrease in white blood cell count which was observed as early as two days after the treatment with animals achieving nearly normal white blood cell level counts within one week. Synthetic Biologics’ antibody combo has a significant effect on disease progression, and we are moving this program forward towards human studies.

In terms of specific next steps, we intend to file an Investigational New Drug – IND – application to support expeditious Phase I clinical trial, and we expect this first in man study to initiate during the first half of 2015. Top line data should be available within 90 days of the study initiation. A Phase II trial is intended to follow in the second half of next year. We intend to request an orphan drug designation for SYN-005 for the treatment of pertussis later this year. In the meantime, we have filed an additional patent application around pertussis antibodies and we intend to move this into CGMP manufacturing to support human clinical trials. Synthetic Biologics’ intellectual property for the pertussis program, like the C. diff product, is robust.

Our third potential anti-infective blockbuster is for constipation predominant irritable bowel syndrome, or CIBS, toward preclinical studies. This program is being developed under a worldwide exclusive license agreement with the Cedars Sinai Medical Center in Los Angeles and represents Synthetic Biologics’ second development program to leverage recent discoveries relating to the human microbiome. Under the agreement, Synthetic Biologics obtained the rights to develop therapeutic and prophylactic treatments for CIBS and other GI-related diseases. Dr. Mark Pimentel, a GI motility expert at Cedars Sinai, is leading the development of our CIBS program and it is his team’s critical discoveries related to the impact of methane-producing organisms on conditions such as CIBS that made this a particularly attractive pipeline addition for Synthetic Biologics. Rather than treat CIBS symptoms, the development of SYN-010 is intended to treat the underlying cause of gas pain and constipation associated with CIBS.

Synthetic Biologics recently appointed Dr. Pimentel as Chairman of our newly formed clinical advisory board for IBS, and we look forward to expanding this focused advisory board by adding prominent researchers in this field. We are committed to building this strategic infrastructure in order to leverage the expertise of these thought leaders and increase our opportunity for success with this program as we prepare to enter the clinic.

For those of you not familiar with Dr. Pimentel, he is a world noted GI specialist who recently identified the clinical significance of a unique antibiotic in treating the diarrheal form of IBS in collaboration with Salix, and which has been a principal driver in Salix’s monumental growth into a $6.5 billion market cap company, so we feel very privileged to have him on the side of Synthetic Biologics as we clinically develop his treatment candidate for the constipation form of IBS, a completely separate IBS condition. Of the 40 million IBS patients in the United States, approximately 13.2 million have that form of IBS.

Based on continued preclinical work, Synthetic Biologics expects to initiate a Phase II clinical trial in constipation IBS during the second half of this year under a corporate IND with top line data expected in 2015; and as a quick reminder, we expect development of our CIBS program to be significantly accelerated based on an anticipated 505b2 pathway. We anticipate being able to share additional development details for the CIBS program, including the specific API, in the coming few months. To summarize, the CIBS program will move into the clinic toward the latter part of this year and represents an incredibly novel way of treating the underlying cause of CIBS rather than merely it’s symptoms.

To conclude my formal remarks today, we are focused on successful clinical program execution. Each of our core anti-infective programs has significant key clinical milestones on the way in the near term. For C. diff, we expect to file our IND and initiate Phase I and Ib clinical trials during the second half of this year with preliminary top line data expected by year-end. A Phase II efficacy study is expected to begin in the first half of 2015. For pertussis, we expect to initiate a Phase I trial early next year with top line data available within 90 days of the study initiation. A Phase II trial is intended to follow in the second half of 2015 subject to positive Phase I data. We expect to file for orphan drug status later this year. For constipation predominant IBS, we expect to initiate a Phase II clinical trial during the second half of this year under a corporate IND with top line data expected mid-next year.

We believe our successful efforts in clinical development over the next couple of quarters will drive significant shareholder value. We look forward to keeping you up to date on our programs. Finally as I mentioned earlier in this call, based on the clinically significant results demonstrated in the Phase II Trimesta study, Synthetic Biologics and our collaborators at UCLA expect the Trimesta program to advance towards FDA approval. The specific next steps will be determined based on further discussions with Dr. Voskuhl’s team at UCLA as well as our active conversations with interested parties on the partnering front. We look forward to accelerating the clinical development of this exciting adjunctive therapy for women with multiple sclerosis.

At this time, I’ll turn the call back to Kris.

Kris Maly

Thank you, Jeff. We’d now like to open the lines to questions. Betty, would you please describe the procedure to ask questions for our listeners?

Question and Answer Session

Operator

[Operator instructions]

Our first question comes from Jason Kolbert of Maxim. Please go ahead, sir.

Jason Kolbert – Maxim

Good morning, Jeff. Thanks for the overview – I really appreciate it. Just a couple of questions. First of all on estriol, is it my understanding now that the search would be on for a partner and it would only be with a partner that you would move to the next phase of clinical development, and what would that next phase look like?

Jeff Riley

Hi Jason, glad to have you on the call this morning. The current thought is that we are in multiple partnering discussions at the moment. We are looking for somebody that can either move it forward together with us in unison or that will move it in collaboration into the Phase III trials. There were some incredibly interesting results that came out of this trial, in particular in the cognition piece. We currently have an ongoing Phase II enrollment study going for cognition specifically, and some of the discussions are around do we expand that current—the existing clinical trial in cognition, and what would it look like if we go into a Phase III trial specifically for relapse and remitting. We did have a discussion with the FDA late last year and we do have guidance on what that parallel Phase III, those two pivotals would look like.

That’s about all I can say at this time, but it’s fairly well defined what we would need to do.

Jason Kolbert – Maxim

Okay, well what I hear you saying, though, is that cognition may become one of the endpoints that you’ll be looking at, and that would be new and novel in the world of MS.

Jeff Riley

That’s correct. I mean, there are no other drugs out there today that have a label for cognition specifically; and Dr. Voskuhl, as I said, in her quote seems to believe that it may actually work for other central nervous system disorders, not just MS alone.

Jason Kolbert – Maxim

Thank you. Listen, I really appreciate the breakdown on the pertussis and the constipation the C. difficile programs. I think that’s really helpful. I just wonder if I could get you to expand a little bit on each of those programs on what the size of the Phase II trials might look like, and in terms of the endpoints both in pertussis and in the CIBS program. And on the CIBS program, given the fact that this is a chronic disease that’s occurred over a long period of time, how long will it take before you really would see symptomatic relief? And sorry – one more question, if you can keep this in your head. Since it’s 505b2 and there is an API that’s been used, people have been exposed to that, so is there a lot of historical data that suggests proof of concept is in hand already?

Jeff Riley

Okay, let me—

Jason Kolbert – Maxim

Sorry – many questions.

Jeff Riley

No worries. Let’s start with C. diff. So the C. diff Phase Ia and Phase Ib are fairly quick. Obviously we’re looking at normal volunteers to begin with. That’s not a large number of folks that we need to dose. We have the CRO lined up – it’s all ready to go. The Phase Ib will be roughly the same size but will be inpatient specifically, so we’ll be looking at safety in the Phase Ia and safety plus efficacy in the Phase Ib. Again, not a huge number of patients – well under 50, probably, for each of those two. As you can imagine, it’s a fairly quick readout on these particular patients. Primary endpoint will likely be C. diff with a secondary being antibiotic-associated diarrhea, so fairly straightforward trial.

The Phase II is a different beast. We need to be informed from the Phase I to figure out how large and how powered the Phase II should look like. We are in discussions with a variety of folks that are experts in this field, to include the CDC, to really determine what that would look like. Again, it’s a preventative approach, so it’s never been done; but it’s also point of care, so a patient would take one of our 50 milligram pills or two of our 50 milligram pills at the same time as they go into the hospital and get an IV antibiotic, and they would stay on those pills while they’re on the IV antibiotic and probably a couple days after they go home to make sure that that IV antibiotic is not doing damage in the intestinal tract. So that’s the C. diff program.

Jason Kolbert – Maxim

So what I hear you saying is that by the second half of this year, we should have a little bit more clarity on just what the Phase II design and structure will be as you kind of start this—you know, look forward to prep that for first half 2015.

Jeff Riley

Absolutely, and what we didn’t—you know, we’ll obviously have a pre-IND meeting with the FDA as well to really discuss what that would look like, so towards the latter part of the third quarter or early fourth quarter, we should have a much more well delineated plan going forward on getting through proof of concept.

Jason Kolbert – Maxim

Okay. Same type of question on pertussis and on constipation in terms of Phase II.

Jeff Riley

Okay, so pertussis is probably going to be in the market before all of our other drugs. It’s going to be fairly quick. The initial Phase I study is very easy – you know, it’s probably 10 to 15 normal volunteers. We’re going to inject the antibodies into them and see what happens. Typically there’s almost no effect safety-wise from an antibody, a monoclonal. We’ll then jump directly into the Phase II study. The Phase II study, at the moment we’re looking at a pediatric label for this product, so we’re going to be looking for infants that have pertussis that we’ll dose. We don’t think that we need more than 50 to 60 total to get all the way through the Phase II and the Phase III for that particular product. Again, we’re looking for efficacy and we’ll see that within the first few days, as well as safety; and again, if we see that we’ve not—you know, our first discussion with the regulatory folks seems to point that we may be able to get a compassionate use label for that drug, so we’re looking at next year, towards the latter part of next year when we’ll probably be into a Phase III and then driving forward that we may have a compassionate use designation before that. Again, there’s no drug, there’s no therapeutic available for these infants that get this condition.

Where we’re going to do the trials is still open to question at this time. We’ll do the Phase I here in the United States, but there are other countries outside the United States that have significantly higher rates of pertussis and obviously recruitment becomes much easier in those countries. So that’s pertussis.

Jason Kolbert – Maxim

Thank you, that’s really helpful. And constipation?

Jeff Riley

Last of the Mohicans. So the IBS program – again, the API itself, we can’t talk about it at this time, the reason being is we’re building additional—we end licensed 10-plus patents out of Cedars Sinai around this particular drug and treatment and use, and all of that. We just felt that, and our IP attorneys felt that we needed to build some more IP just to make sure, and that’s what we’re doing today. So we’re doing in vitro and in vivo work with a specific API. The drug itself for the other indication has been around for a long time – very effective, huge database of safety. We should have no issues. The differences are going to be in systemic exposure – again, we’re trying to keep the majority of our drugs, C. diff included, in the intestinal tract with no systemic exposure. That’s the goal. So that is really a unique way of attacking this problem and that’s what we’re doing today, as well as potentially longer lasting within the intestinal tract itself.

So that’s the goal. Again, we’re treating the underlying cause of the disease. We’re not treating the symptoms of the disease, right? We’re trying to knock or prevent the methanogens, which are a form of archaea, which is a different tree of life, but we’re trying to inhibit those guys from making methane in the gut. Not killing them – just keeping them from interrupting that cycle.

The other question you had is how long until we see results. You see results in the first few days. It’s very rapid. You remove the methane gas, you remove the problem that the patients are having. The patients would likely be on a once a day dosage of this pill for the rest of their lives. You take them off the pill, the methane gas comes back fairly quickly, so this would be a chronic treatment for the rest of their lives.

Jason Kolbert – Maxim

Yes, terrific. Thank you for the comprehensive overview, Jeff. We’re excited by all the catalysts we see ahead. Thanks.

Jeff Riley

Thanks Jason.

Operator

Our next question comes from Keay Nakae from Ascendiant Capital. Please go ahead.

Keay Nakae – Ascendiant Capital

Yes, good morning. Jeff, back to Trimesta. So I guess two questions – the first one is are we still looking at mid-2015 to get the top line results from the cognition study?

Jeff Riley

Answer is yes at this point in time, unless we increase the number of patients, which is under discussion at this time.

Keay Nakae – Ascendiant Capital

Okay, and as you move Trimesta forward for MS, if you’re thinking about Phase III studies with cognition as the primary endpoint, it would seem like you’re going to need to or would want to wait until you see that top line data from the cognition study to be able to better inform any tweaks you might want to make to your Phase III study. So is that sort of the timing that one would be thinking about?

Jeff Riley

I would think so. I mean, it really is going to depend on the partner, Keay, to be honest with you. A couple of the partners are very focused on the cognition component and a couple are focused more on the relapse and remitting adjunctive therapy component, so I don’t have a clear answer for that. That will be driven by the guys with the deeper pockets, I think, and how fast they want to move it forward.

Keay Nakae – Ascendiant Capital

Okay. Do you have a better sense at this time when you might be able to release more data analysis from the Phase II study?

Jeff Riley

Well, I think there’s two time points. One, we are tentatively planning an MS investor analyst day like we had last year sometime this summer, where we’ll bring Dr. Voskuhl to New York and we’ll answer any questions or she’ll answer any questions that we may have. The database itself was locked on March 15 so there was only roughly a month of data crunching that UCLA was able to do before Rhonda presented last month, so it’s been a fairly short period of time, which is why we keep saying top line results. Her intention continues to be to present again, I want to say either mid-September or mid-October at the next neurology conference, which is another big one; and that is where she was planning to present the deeper dive – you know, the abstract, all the bells and whistles that came with the program itself.

We have not looked at what’s happened in the 18 to 25 versus the 25 to 50-year-old range. We’ve not looked at a lot of the cognition data up to this point. We’ve not looked at in detail a lot of the MRI data that was also part of the trial, so that is ongoing work that’s being done and funded by UCLA and by Synthetic Biologics. We hope to have a much better view, I think, when we have the investor day in the summer, but I think you’ll have the full-blown soup-to-nuts view in the September-October time frame at the next neurology conference.

Keay Nakae – Ascendiant Capital

Okay, thanks. That’s all I have.

Jeff Riley

Thank you, Keay.

Operator

Again, if you have a question, please press star then one. Our next question comes from Keith Markey of Griffin Securities. Please go ahead.

Keith Markey – Griffin Securities

Morning Jeff, morning Evan. A couple questions. A little bit more on the Trimesta MS cognition information. I was just wondering if you feel that the neuroprotection or neuroprotective properties of Trimesta might be identified through the MRI data on the grey matter.

Jeff Riley

That is absolutely correct.

Keith Markey – Griffin Securities

Okay. Will that be more or less kept away from the public until Dr. Voskuhl makes her presentation at the neurology meeting?

Jeff Riley

That is correct. What was looked at was the white matter, so the data that was in the presentation was white matter only. We will be going back and looking now at all the grey matter over the next few months. We’ll probably under CDA show that to certain partners going forward, and then of course she’ll present it in a more academic fashion later this year.

Keith Markey – Griffin Securities

Sure. Then I was just wondering about the cognition data, did she provide any information about how often the patients were tested for cognitive function during the trial?

Jeff Riley

She’s not released that information yet, no.

Keith Markey – Griffin Securities

Yeah, okay. Secondly, I was just wondering – I understand that a former director sold stock in the company around the time of the Trimesta data being released. Can you address that and tell us whether or not—what the status of that investor’s present interest is in the company?

Jeff Riley

Sure, I think you’re referring to Steve Kanzer. Steve was the founder of this company back in the day it was called Pipex. He had roughly 7 million shares up until recently. He resigned his position in the company on February 26 of this year, and he reported to the SEC sales of approximately 5 million shares on April 29, April 30, right in that time frame. So like a lot of the folks on this call, he’s now just a private investor and he’s not privy to any of our ongoing—since February, he’s not been privy to any of our internal management discussions. He’s under 5% and not required to report any future sales going forward.

Keith Markey – Griffin Securities

Right, okay. Thanks. One final question about the CIBS program. Do you have a sense at this point of how large that trial might have to be, and whether or not you would have to have rather a prolonged clinical trial, maybe in the neighborhood of two to three years, to determine safety and efficacy on a chronic basis?

Jeff Riley

Well, without giving away the API, I’ll try to answer that question. The existing API for the existing condition is given chronically and is given over the lifetime of a patient.

Keith Markey – Griffin Securities

Okay, so it’s not a major leap forward.

Jeff Riley

Right. Our product is the same product, essentially, but we’re reducing the systemic exposure so that there will be no systemic exposure or as little as possible. So I would guess from an FDA regulatory perspective, they would view that in a positive light, that we’re taking an API that’s been known to be exceptionally safe for many, many years and given over the lifetime of a patient, we’re now taking that and reducing the dosage, probably, and reducing the systemic exposure so it doesn’t get in the bloodstream. So it really is resident only in the intestinal tract.

With respect to how many patients and how long would it be, the trials are not two years. This is like a six month trial at the most. Again, we see effects, or Dr. Pimentel has seen effects within the first few days of giving the patient the drug. I mean, you’re looking at constipation, right, so you’re looking at number of bowel movements, you’re looking at bloating, you’re looking at pain – very easy to measure, typically, in any of these particular patients. So we don’t think it’s going to be a long haul to really look at the data. We’re going to start the latter part of this year and we’ll have data sometime in the middle of next year, so six to 12 months, let’s say, from the time we start enrollment to the time we get final data.

The other thing is we don’t know how many patients it’s going to take. It really depends on discussions with the FDA. Again, this API is a very well known drug, a huge safety database. It gets signaled very, very quickly in these patients, so the question to the FDA is do you run a large Phase I that could potentially be a pivotal trial? Do you run directly into a Phase II, which is our current thought process, and do you power the Phase II high enough where it could be accepted as one of the two pivotal trials? Possibly. We just don’t know at this stage of the game, but I would guess that well under 300 – 200 to 300 patients would be more than sufficient for us to have a high powered program in this area.

Keith Markey – Griffin Securities

That sounds great. One of the things that is diagnostic for this particular condition, from what I understand, is the presence of methane in the breath in the patient. I assume you’re going to be measuring that on a periodic basis during, prior to and then subsequent to treating the patient. How is that done?

Jeff Riley

It’s just a breath test. It’s just like if you were pulled over by a policeman and got hit by a breath test. I’m not saying you’ve had that, Keith! I’m just—you know, we definitely have—it’s very straightforward. We likely would not use it at the beginning. We wouldn’t use it to enrich the patient population. We don’t want it to be used in that way, but we would do it as we went along to correlate reduction in methane gas with a reduction in the symptoms.

Keith Markey – Griffin Securities

Are you saying that you’re not going to identify the patients who have methane being expressed as the ones that you’ll enroll?

Jeff Riley

Well, we won’t use that as an enriching patient population choice, and there’s a variety of reasons for that, the primary one being it’s just—it wouldn’t be used that way out there in the real world, right? I mean, physicians will prescribe this without doing the methane test many, many times, so we’re trying to look at it is what would the real world situation be like, and Dr. Pimentel kind of goes back and forth should we use the breath test upfront with every single patient and use that as a determinant as to whether that patient is enrolled, or do we not? At this time, it’s sort of in between. My guess is that he will take all comers in the Phase II trials and then we would use the breath test as just a marker going forward.

Keith Markey – Griffin Securities

Okay. Thank you very much.

Operator

Thank you, and as that is all the time we have for questions, I would now like to turn the conference call back over to Jeff Riley for any closing remarks.

Jeff Riley

Everybody, thank you for—if you’re still on the call, for lasting with us for the 45 minutes. I’m sorry – it was a pretty long one, but we felt it important to really address the MS results with more clarity. Thank you, Betty, for guiding this call.

I want to reiterate one last time that the fundamentals of Synthetic Biologics’ business are strengthened given results of Trimesta. We are on schedule to hit all of our anti-infective milestones and we’ve engaged in partnering discussions on Trimesta. We remain strategically focused on developing novel anti-infectives, which is our core strategy.

As noted earlier, we expect our C. diff and CIBS programs to move into the clinic later this year, and pertussis early next year. We also believe we have added significant value to the company by demonstrating the therapeutic potential of oral estriol in the Phase II exploratory trial, which further supports Synthetic Biologics’ efforts to attract a strategic partner to accelerate development of this innovative therapy for relapsed and remitting MS. Again, I’d like to thank our long-term investors that have been with us for the last couple of years, and we look forward to a very, very fruitful end of this year and next year.

Thanks again for everyone joining us this morning. We look forward to updating you at an MS investor analyst day sometime this summer and later in the year at an anti-infective investor analyst day, and of course our quarterly call next quarter. Thanks a lot.

Operator

The conference has now concluded. Thank you for attending today’s presentation. You may now disconnect your lines.

Copyright policy: All transcripts on this site are the copyright of Seeking Alpha. However, we view them as an important resource for bloggers and journalists, and are excited to contribute to the democratization of financial information on the Internet. (Until now investors have had to pay thousands of dollars in subscription fees for transcripts.) So our reproduction policy is as follows: You may quote up to 400 words of any transcript on the condition that you attribute the transcript to Seeking Alpha and either link to the original transcript or to www.SeekingAlpha.com. All other use is prohibited.

THE INFORMATION CONTAINED HERE IS A TEXTUAL REPRESENTATION OF THE APPLICABLE COMPANY'S CONFERENCE CALL, CONFERENCE PRESENTATION OR OTHER AUDIO PRESENTATION, AND WHILE EFFORTS ARE MADE TO PROVIDE AN ACCURATE TRANSCRIPTION, THERE MAY BE MATERIAL ERRORS, OMISSIONS, OR INACCURACIES IN THE REPORTING OF THE SUBSTANCE OF THE AUDIO PRESENTATIONS. IN NO WAY DOES SEEKING ALPHA ASSUME ANY RESPONSIBILITY FOR ANY INVESTMENT OR OTHER DECISIONS MADE BASED UPON THE INFORMATION PROVIDED ON THIS WEB SITE OR IN ANY TRANSCRIPT. USERS ARE ADVISED TO REVIEW THE APPLICABLE COMPANY'S AUDIO PRESENTATION ITSELF AND THE APPLICABLE COMPANY'S SEC FILINGS BEFORE MAKING ANY INVESTMENT OR OTHER DECISIONS.

If you have any additional questions about our online transcripts, please contact us at: transcripts@seekingalpha.com. Thank you!

Source: Synthetic Biologics' (SYN) CEO, Jeff Riley on Q1 2014 Results - Earnings Call Transcript
This Transcript
All Transcripts