Synlogic, Inc. (NASDAQ:SYBX) Q1 2019 Results Earnings Conference Call May 9, 2019 5:00 PM ET
Elizabeth Wolffe - Head, IR and Corporate Communications
Aoife Brennan - President, CEO, and CMO
Scott Plevy - CSO
Todd Shegog - CFO
Conference Call Participants
Mark Breidenbach - Oppenheimer & Co Inc.
Joseph Schwartz - SVB Leerink
Samantha Semenkow - Citigroup
Ted Tenthoff - Piper Jaffray & Co.
Edward Marks - H.C. Wainwright & Co.
Chris Howerton - Jefferies
Julian Harrison - BTIG
Taylor Feehley - Chardan
Good afternoon. Welcome to the Synlogic's First Quarter 2019 Conference Call and Webcast. At this time, all participants are in a listen-only mode. There will be a question-and-answer session at the end of this call. Please be advised that this call is being recorded.
I would now like to turn the call over to Dr. Elizabeth Wolffe, Head of Investor Relations and Corporate Communications. Please proceed.
Thank you, Angela. Good afternoon and thanks for joining us on today's conference call. This afternoon, we issued a press release, which outlines our first quarter 2019 financial results and several other topics that we plan to discuss today. The release is available on the Investor section of our website at www.synlogictx.com.
Joining me on this call today are Aoife Brennan, President and Chief Executive Officer; Todd Shegog, Chief Financial Officer; Antoine Awad, Head of Technical Operations, and joining us for the first time in his new role as our Chief Scientific Officer, Dr. Scott Plevy.
During the call, Aoife will provide a brief outline of our recent progress and introduce Scott. Todd will summarize our financial results for the quarter and finally Aoife will discuss the data that were recently presented at the ASGCT Annual Meeting and their implications for the development of our Synthetic Biotic platform before summarizing on our upcoming milestones. Following our prepared remarks, we'll open up the call for questions.
As we begin, I'd like to remind everyone the comments today may include forward-looking statements made under the Private Securities Litigation Reform Act of 1995. These forward-looking statements are made as of the date hereof and are subject to numerous factors, assumptions, risks and uncertainties which change over time.
Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including those described under the heading Forward-Looking Statements in Synlogic's press release from earlier today or under the heading Risk Factors in Synlogic's most recent Form 10-K, or in later filings with the SEC. Synlogic cautions you not to place undue reliance on any forward-looking statements.
Now, I'd like to turn the call over to Aoife.
Thanks Liz. Good afternoon everyone and thank you for joining us in our call to discuss our first quarter 2019 financial results, progress in 2019, and what's shaping up to be a busy time for Synlogic as the year progresses.
At Synlogic, we're designing for life, using the tools and principles of synthetic biology to rationally engineer living medicines that have the potential to dramatically change the lives of patients.
We recognized a significant unmet need in diseases with complex biology, which provides an opportunity for innovative therapeutic approaches such as our Synthetic Biotic platform.
We are using our platform to develop novel class of living medicines based on a strain of non-pathogenic bacteria and engineering them to perform therapeutic functions that may be missing or impaired in patients.
For new modalities, it's critical to understand where they may be most effective. And as we started to develop the platform, we made a strategic decision to focus on programs that required the bacteria to be active at different sites in the body.
In that way, we would learn about the kinetics of our Synthetic Biotic medicines at different sites of action and use this information to choose additional programs to advance.
Our initial programs were there before designed to be active in the small intestine, colon and tumor. SYNB1020, a strain of E. Coli Nissle has been engineered to consume ammonia within the colon where approximately 50% of total body ammonia originates.
In hyperammonemia, imperative function our genetic mutation in one of the enzymes of urea cycle means that patients cannot effectively metabolize ammonia. Blood ammonia levels elevate and this can have serious health consequences potentially leading to coma and death.
Next, for SYNB1618, small intestinal activity is most important, as that is the site where most phenylalanine is present within the GI tract. SYNB1618 is designed for the treatment of phenylketonuria or PKU.
In PKU, defects in phenylalanine hydroxylase enzyme meet the elevated levels of phenylalanine or Phe in the blood, which can result in cognitive impairment, seizures, and other health issues.
SYNB1891 is designed to activate the immune system in tumors that are not responsive to checkpoint inhibitors. For this, Synthetic Biotic medicines were just delivering bacteria into the tumor microenvironment directly.
In addition to executing these programs, we've also been investing in additional programs that we will advance based on data and information gained from our initial clinical trials. We also see potential for our Synthetic Biotic medicines in broad disease areas.
In inflammatory bowel disease or IBD, there's an opportunity to explore the activity of our strains in the colon, and this is a disease where there is a need for a combination approach, a key capability of our living medicines. We are developing a Synthetic Biotic medicine for IBD in collaboration with AbbVie.
Ultimately, our goal is to develop therapeutic applications in which we design Synthetic Biotic medicines that are engineered to sense disease states and respond with a combination of therapeutic responses to the disease symptoms.
We had a busy start to the year and have made progress on all of these fronts. Importantly, for the development of the organization, we expanded our scientific leadership of the company with the appointment of Dr. Patricia Hurter to our Board of Directors; and Dr. Scott Plevy as our Chief Scientific Officer. Both have significant experience in drug developments and will be very helpful to us as we move our programs forward.
I'd like to take this opportunity to introduce Scott. He joined us full time this week. I think this is day four him. As many of you know from the press release that we issued recently, Scott is a physician scientist, a gastroenterologist by training, who enjoyed a successful career in academia before moving to industry and most recently served as Vice President in Gastroenterology Disease Area Leader at Janssen Research & Development.
Let me hand over to Scott to say a few words about his background, why he joined us and what he is excited the most. Scott?
Thanks Aoife and good afternoon everyone. It has been a whirlwind few days, but I am delighted to have joined Synlogic and to finally be here at the company getting to know the team. I've been very impressed by everyone I have met with the company. Their accomplishments over the past four years speak for themselves.
People have asked me why I moved to Synlogic. In addition to the high quality of the science, I really captured my imagination was the transformational potential of this Synthetic Biotic platform and the approach that the company is taking to really understand and develop the platform.
As a clinician scientist, I have always been driven by unmet needs for patients with debilitating diseases. There is a growing awareness in the biopharma industry that the ability to deliver combinations of therapies to the right location at the right time is the way of the future, particularly for the treatment of complex diseases.
As I learned more about Synlogic, I gravitated towards the Synthetic Biotic platform as an exciting potential therapeutic solution for a broad range of systemic, metabolic, and inflammatory diseases.
My experience includes research in areas from disease specific targets to basic immunology and molecular biology. I have also carried out translational research to advance the understanding of novel immunological interventions in inflammatory bowel disease or IBD, other inflammatory conditions and microbiome-related diseases, and I have served as the lead investigator on multiple early phase clinical trials.
At Janssen Pharmaceuticals, I was responsible for the end-to-end GI disease area strategy in the immunology therapeutic area, but also led the strategy across therapeutic areas, including oncology, neuroscience, and metabolic diseases for understanding and inhibiting the IL-23 cytokine pathway.
With this experience, I believe that my skills are highly relevant to Synlogic and I look forward to leading the team to even greater accomplishments as we continued to build our platform and advance additional programs into development based on data from our initial pipeline programs. I also look forward to getting out and to meet our analysts and investors once I've found my feet here at the company.
Now, let me hand it back to you Aoife.
Great. Thanks Scott. Don't worry, Liz is going to have you out on the road in no time. As I mentioned, it's been a busy year so far. We've made visible progress in orally administered programs, which are currently both being evaluated in patients and we're on track to present top line data in mid-year specifically in the third quarter of 2019.
At the beginning of the year, Science Translational Medicine published the first-in-human data from a Synthetic Biotech medicine and the supporting preclinical data from our SYNB1020 programs to treat hyperammonemia. As is all of our publications and presentations the paper can be accessed from the Publications & Presentations section of our website.
More recently at the Annual Meeting of the American Society of Gene and Cell Therapy, we were delighted to be able to share data from our process to develop a solid formulation of SYNB1618.
I will discuss these data and our manufacturing updates later in this call, but suffice it to say we've demonstrated a robust and reproducible process to manufacture solid material that will be used in our future clinical trials and provides a path to commercialization.
We remain on track to file an investigational new drug or IND application for our program in immuno-oncology, SYNB1891, in the second half of 2019. I look forward to providing more detail at our clinical trial plan for that program on future calls.
Finally, in March, we announced that we advanced our collaboration with AbbVie to develop a Synthetic Biotic medicine for IBD to the lead optimization stage and we received a $2.5 million milestone payment. So, lots of progress on all fronts.
Before I describe our ASGCT data in more detail, let me hand over to Todd who will summarize our financial results for the quarter. Todd?
Thank you, Aoife and good afternoon everyone. Earlier today, we released our financial results for the first quarter ended March 31, 2019, and I'm happy to review the highlights of those results with you now.
For the first quarter of 2019, Synlogic reported a consolidated net loss of $12.9 million or $0.51 per share compared to a net loss of $11.1 million or $0.55 per share for the corresponding period in 2018.
The increase in net loss is primarily due to increases in compensation-related expenses related to increased headcount, as well as increases in research and development expenses to support Synlogic's advancing clinical programs.
The total operating expenses for the first quarter of 2019 were $14.0 million compared to $12.0 million for the same period in 2018. Research and development expenses were $10.4 million for the three months ended March 31, 2019, compared to $8.4 million for the same period in 2018.
The increase was primarily due to an increase in compensation-related expenses associated with increased headcount and increased expenses associated with manufacturing preclinical and clinical studies of Synlogic's Synthetic Biotic programs.
General and administrative expenses for the first quarter of 2019 were $3.7 million compared to $3.6 million for the corresponding period in 2018. Revenue was $0.3 million for the three months ended March 31, 2019, compared to $0.4 million for the same period in 2018. Revenue for both periods associated with services performed under Synlogic's collaboration with AbbVie to developed Synthetic Biotic medicine for the treatment of IBD.
Turning to the balance sheet. Synlogic ended the quarter of 2019 with $109.8 million in cash, cash equivalents, and short-term investments. I'm pleased to say that Synlogic is in a solid financial position with cash that will take us through 2020 under the current plans and we look forward to executing on that plan to advance our pipeline of Synthetic Biotech medicines.
I'd like to thank you and I will now turn the call back over to Aoife.
Thanks Todd. With the solid cash position to see us through 2020, 2019 will be a year of significant achievements for the company in terms of program progress and gaining an understanding of our platform.
Essential punitive [ph] synthetic biology is the idea of design, build, test; rapid cycles of evaluation and refinement to our strain to maximize activity. This is a concept that we've embraced with the company and which is informed not just our strain engineering development but our early chemical strategy.
As I just described, we are evaluating our Synthetic Biotic medicines at three different sites of activity, small intestine colon, and tumor, to determine where the best first applications of our platform lie.
We decided to move into the clinic as quickly as possible with an early liquid formulation of our Synthetic Biotic medicine to determine if we had a feasible approach to address unmet needs in patients.
We wanted to quickly understand if, one, our engineered bacteria were safe; two, if they could perform the functions we had programmed into them in humans as well as they did in in vivo at preclinical models; and three, how their activity in these models predicted activity and healthy volunteers and patients?
We answered the first of these questions in clinical trials of both SYNB1020 and SYNB1618 in healthy volunteers demonstrating that both strains were safe, genetically stable, and cleared from subjects upon cessation of dosing.
By measuring the production of biomarkers, we were able to demonstrate that they were biologically active in the GI tracts, in large and small intestine, respectively. We will answer the last question in the third quarter of this year when we expect to have readouts from our Phase Ib/IIa clinical trial of SYNB1020 in patients with cirrhosis and elevated ammonia and from the expansion cohort of our SYNB1618 study in PKU patients.
The early frozen liquid formulation of our Synthetic Biotic medicines enabled us to rapidly gain important understanding and the feasibility and therapeutic potential of our platform. However, our intention has always been to develop a solid formulation that could be readily taken by patients at home.
Therefore, in tandem with our early clinical studies, we've been developing, manufacturing an appropriate formulation processes and capabilities to deliver a solid formulation that could be stable at room temperature or at the refrigerated product. This has been an exciting undertaking requiring the patient's work as a team with Synlogic and I'm pleased to be able to discuss our progress today.
In addition to investing and developing our fermentation, downstream processing and lyophilization processes, we've taken a cost effective route to expanding manufacturing capabilities to enable in-house GMP manufacturing of liquid and solid forms for our medicines.
This has been a game changer for us as a company as we advance clinical development, as it maximizes our ability to move quickly into the next phase of clinical testing of our Synthetic Biotic medicines with a commercialization and patient-friendly formulation.
We announced this initiative less than six months ago and I'm pleased to report that the first clinical trial material was manufactured at our new facility. This is the liquid formulation of 1891, our first immuno-oncology program for inter-tumor injection.
In terms of oral delivery, we've continue to build that this week that will be used for GMP production of lyophilized material and expect to begin manufacturing lyophilized GMP materials for our next SYNB1618 clinical trials within the month.
We were very pleased to be able to present data at the ASGCT meeting on the characterization of SYNB1618 made using our improved fermentation and lyophilization process.
The data highlights several important advances that we've made over the past year or so. The original poster is available in the Presentations & Publications section of our website, and I would summarize the main takeaway in slide three through five.
First, we've improved the fermentation process to produce material that retains viability and activity when lyophilized. When we began our solid formulation work, we were warned by others that we might have to accept a significant loss of viability and thus for a living medicine, a loss of activity.
However, and this has been a testament to the great work of the team here, we've demonstrated that our lyophilization process reproducibility results in minimal loss of viability and activity.
I should note that we've determined that not all live cells give rise to colonies and that's the important measure for our living medicines is the number of life cells rather than the total cell number of CFU.
Even with an early development batch, when we compared a frozen liquid formulation to lyophilized material, we saw very similar activities of liquid and solid formulations when we compared equivalent numbers of life cells from each preparation and their production of biomarkers of SYNB1618 activity. This was demonstrated across in vitro assays, an animal model of disease and in healthy mice and non-human primates.
We've also measured several physical characteristics such as free protein and viscosity in addition to cell viability and activity. The batches made using our new process are less viscous and most have lower free protein levels suggesting that there is less cell breakage. This is very encouraging and we look forward to evaluating the effect of these improvements in humans.
If you recall from our healthy volunteers, while the product was safe, we saw some tolerability issues at high doses of the liquid formulation, notably nausea and vomiting in the first few days of dosing.
We did not know the exact mechanism. However, one possibility is that this is caused by cell fragments and debris in the preparation. When we compared the liquid presentations of both SYNB1020 and SYNB1618, it was notable that subjects reported tolerability issues at lower doses of SYNB1618. And we saw more cell breakage in this preparation than in the SYNB1020 liquids.
Having said this, we don't have a preclinical model for tolerability and so we will need to assess the impact of the improved fermentation process and solid formulation in healthy volunteers directly, which we will do in a bridging study. We may also be able to evaluate the effectiveness of a dose ramp to improve tolerability.
As the tolerability issues were have only observed in the first days of administration, we may be able to alternate subjects to the bacteria at a low starting dose and then increase the dose itself.
Our lyophilization process is robust and reproducible. As we show in slide four, we demonstrated reproducible viability and in vivo levels of activity in healthy mice across three different batches made at the 30-liter scale.
An important factor for us is also stability, and while these studies ongoing, our initial data suggests that the lyophilized material is stable at 2 degree centigrade to 8-degree centigrade for at least three months and for more than 30 days at room temperature as seen on slide five.
This property is very important, not only as we consider the ability to execute larger trials of longer duration but for patients having a medicine that's easy to store and transport on a day-to-day basis is critical.
Importantly, this also gives us the preparation of SYNB1618 that is suitable for commercialization and can be formulated in sachets or capsules depending on the patient demographics.
Even with the current competitive landscape in PKU, we believe that there's a pressing need for a safe oral therapy that's suitable for all patients with PKU, regardless of their type of mutation or their age.
And we are particularly looking at younger patients who have the fewest therapeutic options, which brings me to our ongoing study in SYNB1618 in patients with PKU and probably expect to report in the third quarter.
First, we will gain an understanding of the safety and tolerability of the liquid formulation in patients. We will also be focused on the levels of biomarkers of 1618 activity and high levels in patients compared to those in healthy volunteers.
Our intention going forward is to use lyophilized material in future clinical studies and so we will first evaluate tolerability and dosing and a bridging study in healthy volunteers. I expect to provide more detail as to our plans when we release data from our ongoing study in the third quarter.
In our ongoing SYNB1020 HE study in patients with cirrhosis and elevated ammonia, we're evaluating safety and tolerability in this population, measuring ammonia lowering and evaluating several exploratory endpoints.
We also expect to have data to share from this program in the third quarter of this year. As we've indicated before with ammonia lowering data and other supportive outcomes in these endpoints, we will make decision on the development path for SYNB1020.
We remain on track for filing our first IND for our IO program in the second half of 2019. As I mentioned earlier, we've already made the clinical trial material for this program and I look forward to sharing more information around the specific clinical trial plan in a not too distant future. We're also working on a number of new pipeline programs and I look forward to providing more detail on those later in this year also.
To sum-up, with approximately $110 million at the end of the first quarter, we're in a solid cash position to take us through 2020 and enable us to execute our current plans beyond our upcoming clinical milestones.
I look forward to updating you on our progress throughout the year. So, thank you. Angela, I now open the call for questions.
First question comes in the line of Mark Breidenbach with Oppenheimer. Please go ahead caller.
Hey good afternoon.
Just a couple of quick ones. Now, that you've demonstrated that your lyophilized formulation can have similar viability and metabolic capacity as the liquid suspension version, I wonder if you can translate for us what a dose level would look like, not in terms of number of live cells or colonies but in terms of like grams of actual powder that would be required for patients to take each day? Thank you.
Yes. So, that's a great question. I think it really depends on the dose and because we're moving from the liquid to the solid oral, our plan, Mark, is that we would do a bridging study in healthy volunteers with a solid oral where we would measure not just safety and tolerability, but also look at the PD biomarkers, and with that data and understanding how healthy volunteer is compared to PKU patients based on the current study, we would develop either one or two doses to take forward into a Phase II trial.
And I think it's only after we kind of understand what the dose level that likely to be the optimal dose level that we would be able to give you an estimate of what that would be in terms of grams or number of capsules or sachets or some of those other details. So, it's likely that we'll still have a little bit of dose finding to do.
But I think the great news is that now that we have the lyophilized product, we have lots of options and because we can move into lots of kind of other preparations and presentations to address pediatric or adult indications, so it gives us lots of optionality.
And a quick follow-up, it sounds like those two very important readouts coming up in the third quarter both for the PKU project program and for SYNB1020, and it sounds like we'll be dealing with relatively small numbers of symptomatic patients in both readouts. First of all, I am wondering if this information -- this top line data will be released by a press release or if this will be in the context of medical conferences.
And second, do you anticipate that will have enough information from these small numbers of patients to sort of gauge metabolic turnover in terms of the capacity of the cells in symptomatic patients that actually process the buildup of toxic metabolites? Thank you.
Yes, it's great question. The first one is easy. I think we would issue a press release and a call once we have the data and underlined -- and have interpreted us. And then, we would look at the next opportunity to present a full data set as an academic meeting.
I think we've always moved very aggressively to publish and disclose all the data that we collect in the trials. So, this time will be no different from that. So, you can expect to see the press release first followed by full disclosure of the data set for both readouts.
And I think the second one in terms of the interpretation in the small numbers, these are really studies that were designed around kind of biomarkers and based on our healthy volunteer experience certainly with the PKU, because we're using this stable isotope, we're generally able to give -- we can get pretty good precision with the data. So what we would be able to say is the bacteria is working or not and how the activity compares healthy volunteers to PKU patients.
I think the second component that will aim to provide is how those data are contextualized in terms of an ability to achieve our product profile. So, we'll be providing both the numbers, but also our interpretation of those in terms of how good they are, based on how we'd like to achieve in patients with PKU.
So, -- and that's what we're working towards here with the team, the data, the context, but also then the guidance -- providing guidance around the upcoming milestones and activities for both programs. So, that's I think what you can expect to get from when we disclosure the data in the third quarter.
Okay. Thank you very much for taking the questions and congrats on the progress.
Yes. It's good to speak to you, Mark.
And our next question comes from the line of Joseph Schwartz with Leerink. Caller, please go ahead.
Hi there. Congratulations on all the progress. I was wondering with the new solid formulation of the SYNB1618, what kind of a dosing regimen would you anticipate? Would it differ in any way from the current frozen liquid and be something other than a thrice a day?
Yes. So, that's a good question. I think the dosing regimen for us is not so much dependent on the formulation or presentation, it really depends on the site of activity and the kinetics.
So, that's the reason that we took these three different sites of action kind of programs forward, is that the kinetics will be very different. So for instance, in the small intestine, we know that the bacteria are really there for four hours to six hours based on both small intestinal transit time and also from the data that we got in the healthy volunteers. So, I think regardless of solid or liquid, it's likely that that product would need to be taken three times a day.
For colonic programs, there's potential that it may be taken less frequently given that we know our bacteria spend between 24 and 48 hours in the large intestine and so there may be potential for once daily dosing with colonic applications based on what we know now.
And then I think finally for the inter-tumor programs, because they're injected into tumor, there will be a completely different dosing regimen there. But that's I think a very different context.
So, we think the kinetics will depend both on the size of action and the mechanism that we're engaging for all the programs, but we'll probably not be influenced by the formulation work.
Okay that makes sense. So, could you give us an update on the status of your work to develop a solid oral formulation of 1020? And is that process quite similar or other differences that we should keep in mind?
So, we think that we'll be able to apply the lessons that we've learned from 1618 across all of the oral programs. It's likely that there will be some tweaks to the process, but I think certainly we've seen a lot of benefits in kind of what we've learned from one and been able to apply that knowhow and learning to other programs.
So, we haven't provided any guidance in terms of next steps or timelines. I think we would aim to do that when we disclose the data. But we anticipate that there'll be lots of learnings from 1618 that can be applied to all of the oral programs.
Great, very helpful. Thanks for taking my questions.
And our next question comes from the line of Yigal Nochomovitz with Citi. Please go ahead.
Hi Yigal. I also have first name that's difficult to pronounce, so you're in good company.
Yes, happens all the time. Actually this is Samantha on Yigal. But, you're right. [Indiscernible]. Anyway thanks for taking my questions. I also have a last name that is hard to pronounce. So, I understand. And congrats on the progress on the solid formulation.
I'm curious, obviously, the stability that you've achieved for 90 days for 2 degrees to 8 and 30 days for room temperature is an achievement. I'm wondering if you have any room there to potentially optimize it further. Or do you think that, that durability is sufficient for patient expectation?
Yes. So, our aim is to eventually have up to one to two years and has the stability studies work that we put a batch in stability then we put samples at intervals over a period of time. So, we have only posed samples up to the three-month time, but that study is ongoing and will continue to develop. We have no way to expedite that, it is just an issue of every month that goes by we get additional information about stability.
But what I will say is that if you look at the graphic on the slides, the trends are very, very positive. So, even as you know, up to 90 days, we're not seeing any kind of drift off. So, we're very confident based on these early kind of trends that we're seeing up to 90 days that it looks like -- it's looking good for longer periods of time. But you're absolutely correct, our goal would be to have a year or two years of stability. Is that a perfect commencement?
Yes, that's perfect. And then, I guess just a little bit on the biology just going back on to 1618. Is there -- one -- is there a difference between PAL and LAAD consumption for phenylalanine in terms of -- is one more important than the other outside of the location of where they are in the cell and your biotic or did they contribute fully to phenylalanine consumption?
Yes, that's a great question. As you know, we have -- so they both consume phenylalanine. The PAL enzyme produces TCA, which we can measure directly; the LAAD large enzyme produces phenylpyruvate, which is very difficult to measure from numerous reasons and in humans. We believe that both contribute to phenylalanine consumption in vivo.
The real question is how much each contributes and how we can build the model to be able to kind of showcase here's where we are falling in terms of our therapeutic target. And but we've done a lot of work on developing biomarkers for the LAAD enzyme activity, so that we can help make some estimate of how much phenylalanine is being consumed by the strain in vivo. And but you're correct both to function independently and consume phenylalanine in different ways.
Great. Thanks so much for taking the question.
And your next question comes line of Ted Tenthoff with Piper Jaffray. Please go ahead.
Great. Thank you very much and Scott welcome, that's a great background. I think you're going to bring a lot of expertise to the team. I wanted to check on sort of next steps, if I could and I know you -- I don't want to get too far out here ahead of the curve, but assuming positive data in PKU, what were the next or what could the next study look like? What do you think you need to demonstrate? Thank you.
So, that's great. I think you just set expectations for analyst community in terms of what to expect in the third quarter, we'll be presenting the data from the PKU cohort and we'll be presenting kind of our plans for the program in terms of next steps and guidance and we'll be also providing some context in terms of how to interpret the biomarker data.
I think in terms of next steps that would include doing a bridging study in healthy volunteers to make sure that the -- we think we have improve tolerability based on the quality parameters that we've measured, but we'll be validating that in a short healthy volunteer study and then moving -- if that all looks good, moving directly into a Phase II trial in patients with PKU.
And obviously, once we've gotten that all, tied up as a bow, we'll be able to give a lot more detail in terms of specific study design and timing to set some expectations about that but we'll be moving forward pretty quickly with the program.
All right, cool. Awesome. Looking forward to data in the third quarter.
Awesome. Thanks Ted.
And our next question comes from the line of Raghuram Selvaraju with H.C. Wainwright. Please go ahead.
Hi, this is Edward Marks on for Ram. You just mentioned that bridging study for 1618, I'm just wondering if you could go into a little detail on whether bridging studies would also be necessary for 1020 and 1891?
I think -- so, I will start far from the end backwards, 1891 product is given by inter-tumor, products at a minus 80, liquid, because it's given intertumorly and it's possible that if we get great data that formulation and presentation can be taken all the way through registration, because we anticipate that would be given in an inpatient setting in a cancer hospital kind of scenario where products that have sufficient efficacy and tolerability and the context of cancer and that's certainly viable to take all the way through.
In terms of the oral programs, I think based on our experience to-date, everything we do with one program allows us to do less and to be more efficient with subsequent programs. I think we would make a decision at the time and disclose that.
But I think I would anticipate we learned a lot from the bridging study in the PKU program regarding how the preclinical data translates into the clinic and it was novel platform, every time we do a trial in humans, we're learning more and more that can be applied to subsequent programs and then have a good experience with regulatory so far been able to cross-reference at some of the experience that we've had already also. So, we'll certainly be working as efficiently as possible as we move the programs forward.
Excellent. Thank you for that clarity. And then when we look at 1618 itself, are you saying then categorically, there would be no further clinical study using a liquid formulation?
Okay. And then again looking out a little bit further, if you can comment just looking at 1891, what kind of combination regimens or cancer types might it be deployed in?
So, we will be providing this specific study design I think at a future date, but what we've disclosed in our ASGCT Presentation is that we'll be pursuing solid tumors that have been resistant to all available treatment modalities and it will be given by inter-tumor injection.
It's likely that the study will contain a monotherapy arm and then a combination therapy component and as soon as we have the kind of full details around its specific inclusion criteria and the regimen for the combination arm, we'll be certainly providing an update. So, watch this space.
Yes. I'll be looking forward to it. And then finally just talking about the AbbVie collaboration, when might enter the clinic, you mentioned filing an IND, but also just talking about whether they're interested in the solid oral formulation as well?
I think I can guarantee they'll be interested in our solid oral work and are watching closely. I think I shy away from making forward-looking statements when I'm in control. I'm even more leery about it when somebody else is in the driver's seat.
So, I think it would be very difficult for us to anticipate an IND filing date for that program right now and just based on that collaborative nature and some of this will obviously be subject at the governance structure, but as soon as we get close, we will certainly be updating you.
All right. Well, thank you very much. I appreciate taking the questions.
And your next question comes from the line of Chris Howerton with Jefferies. Please go ahead.
Hey thanks for taking the questions. I think at this point most of them have been asked, but I think I want to touch upon the idea of what the quality of the measure of viable cell count versus CFU and why and do you believe that that's a better measure of activity?
Yes, I think that's a great question. It's not just our hypothesis, it is actually demonstrated data head-to-head to show that for this program and for our product, live cells actually predict in vivo activity in an animal model and much more precisely than CFU. So, we believe that the right strength and potency measure is live cells and that CFU are actually not a correct measure. And it's not so relevant to the activity.
I think is what is the benefits of having our Synthetic Biotech platform where we actually understand mechanism and can measure in vivo activity that we can really evaluate what are the critical quality attributes and compare that and present a signed package to support the critical quality attributes.
I think it's a nice feature of our platform that we can do that. I'm not sure if we disclosed this data but we will at some point if we haven't. But they do support live cells, as being the right measure.
Got it. Okay. And then for the potential work on 1020 for a solid formulation or lyophilized formulation, will those activities be gated by these critical clinical data or those going on right now?
Yes. So, we have done some work preliminarily to [Indiscernible] and less based on clinical data and just more based on our capacity here internally and as we've been able to fit this in and around activities on the other programs. But as soon as we have clinical data, we'll be able to provide kind of next step in and set expectations during timing. So, that would be the plan.
Okay. And then I guess one last question just with respect to kinetics in a different biological compartments that you are exploring, do you think there's any opportunities to alter those kinetics whether that's a formulation perspective "from a synthetic biology perspective"? So, for example, if you observe that there is better within the mouse testing, are there opportunities to increase the growth area under the curve, let's say, activity there?
Yes, we have done some kind of preliminary brainstorming and thought process around that, particularly as it pertains to small intestinal transit time. And we've talked about ways we could engineer bacteria to hang around a little bit longer at the small intestine. I think I discussed here with the gastroenterologist and he'll be a digging in on some of that work.
And Chris, we engineer these to be off the trough, so we can think about other ways maybe in the colonic compartments to enables them to stay around a little bit longer. What was most important for us is to make sure that we had good safety and genetic stability before contemplating some of those kind of what you might call next-gen approaches and they're very much kind of an exploratory research type of stage right now, but nothing meaningful or concrete to discuss, but an interesting thought experiment.
Okay. That make sense. And then the last thing when you think about the translation of liquid formulation to lab [Indiscernible], are there additional [Indiscernible] that were added to drug trials that would constitute any kind of meaningful biological differences between the two?
No, we haven't -- a lot of are kind of special cells knowhow and we're maintaining it's a trade secret; you'll understand because a lot of work is to be able to preserve so much vitality to lyo, we are kind of I think one of the first to invest in this area; I think there can be lack of investment.
So, right now we're kind of keeping that secret stuff secret. I think suffice it to say that we've tested a lot of different, it depends on components and none of them would have any impact on the safety or benefit risk profile of the product and we'll provide enough data on an ongoing basis.
Well, if it has a hand, there will be a mouth.
I was just saying -- I can tell you, but then I have to kill you.
Okay. All right. Well, I appreciate you taking the questions and I think I'd ease it down at this point. [Indiscernible]
Yes, you do, you do.
All right. Well thanks so much and have a wonderful evening.
And your next question comes from the line of Tom Shrader with BTIG. Please go ahead.
Hi there, this is Julian on for Tom actually.
Hey Julian, how are you?
Pretty good. How are you guys?
So, I just had two quick questions. First on the heels of ASGCT, just wondering if you could comment on how important it is to have a dose full of regimen as opposed to I guess one-and-done gene therapy for a condition like PKU.
I guess what I'm really trying to get at is, how tight is the normal range? Is it generally difficult to stay within 120 micromolar to 360 micromolar, I guess, in the clinic and do you see this as a possible driver of interest in alternatives to gene therapy for the condition?
Yes. So, I think, gene therapy -- for PKU, gene therapy is still in a very early stage. I think they'll be days and maybe next year from some of the programs, but absolutely an interesting approach.
Our understanding of the gene therapy programs right now is that they're really targeting the adult segments and they'll be targeting -- our patients will be eligible who you don't have preexisting antibodies to the whatever capsid been used in those programs.
So, we're watching the data and we're hopeful at some point in the future that so be a gene therapy option for a subset of patients with PKU. But we also see tremendous opportunity for chronic overall product, particularly in the pediatric segments where I think these kids really struggle to maintain adequate protein intake and maintain their Phe levels in the normal range.
So, I think there is a normal range where all of those, those of us not born with a PAH mutation maintain blood Phe pretty tightly and then the recommended range based on the treatment guidance, which is a Phe level of less than 360 micromolar. We know from some cross-sectional data that a minority of patients actually stay -- get within that recommended target range.
So, I think there's a big unmet need. I think there is a need particularly in the pediatric segment and even an adult in the context of gene therapy programs we see that there's an interesting opportunity to do something meaningful.
So, we're watching the space for it. We're learning about PKU all the time and are moving the program forward aggressively.
Okay, great. Thanks, that's helpful. And then Todd, sorry if I missed this, but are you still guiding a runway through 2020?
Yes. Julian, that's correct. Yes, through 2020.
Okay, great. Thank you very much.
And our final question comes from the line of Taylor Feehley with Chardan. Please go ahead.
Hi thanks for taking the question. I just want to clarify one of your statements, particularly on 1020. So, you mentioned that based on the results of the ammonia lowering in the third quarter, you'll determine further development.
I was just wondering if you could give a little more color on what that means. Should we think about that more in terms of formulation and the bridging study or patient populations that are most amenable to the therapy? Anything else you can provide would be great.
Yes. So, I think as we said earlier and I probably should have been more explicit in my remarks today. I think the path forward for 1020 program depends on the strength of the data but also some work that we've been doing to really understand the development path in UCD and the various segments within the liver disease population.
So, we've been doing a lot of work to map out what the next studies could look like, what the development feasibility and unmet need is across the various applications for the 1020 program could pay.
So, I think it was both in connection with the target patient population for future development as well as the guidance around when that next study would be likely to start. So, it really is kind of a holistic development plan path forward that we would be sharing with the data in hand. Does that make sense?
Absolutely. That's really helpful. Thank you so much. And I think all my other questions have been answered. So, that's it for me today.
And I'm showing no further questions at this time. I would now like to turn the call back to our host for closing remarks.
Thank you, operator. We'd like to thank everyone for joining us on today's call and we look forward to updating you on progress across our developing pipeline in the coming months. We'll be available throughout the evening if anyone would like to reach back out for a follow-up call. Thanks so much for your questions and your engagement. Thank you.
Ladies and gentlemen, this concludes today's conference. Thank you for your participation and have a wonderful day. You may all disconnect.