Julie Rezler – Director-Investor Relations and Corporate Communication Mark J. Murray – Chief Executive Officer and President Bruce Cousins – Chief Financial Officer and Executive Vice President Michael J. Abrams – Chief Discovery Officer and Executive Vice President.
Judy Leung – RBC Capital Markets Jason McCarthy – Maxim Group Michael Schmidt – Leerink Partners LLC David Novak – Clarus Securities Stephen Willey – Stifel Nicolaus.
Good day, ladies and gentlemen, and welcome to the Tekmira Corporate Update and Q3 Results Conference Call. At this time, all participants are in a listen-only mode. Later we will be having a question-and-answer session and instructions will follow at that time. (Operator Instructions) As a reminder, this conference call is being recorded.
I’d now like to turn the call over to Julie Rezler, Director of Investor Relations and Corporate Communication. Please proceed..
Thank you, Nicolas. Good afternoon everyone and thank you very much for joining us as we provide our corporate update and report the third quarter results for Tekmira Pharmaceuticals Corporation. Joining me on today’s call is Dr. Mark Murray, Tekmira’s President and CEO; and Bruce Cousins, Executive Vice President and Tekmira’s Chief Financial Officer.
I would like to remind everyone that there are a number of statements made in this conference call that constitute forward-looking statements or forward-looking information under applicable securities laws.
Forward-looking statements and information discussed in this conference call include, but are not limited to, those with respect to the expected developments and key milestones for our RNAi antiviral product platform programs including TKM-HBV and TKM-Ebola, as well as our strategy, future operations, clinical trials, prospects, and the plans of management.
Forward-looking statements and information are current predictions only, which are based on certain assumptions, and include, but are not limited to, the effectiveness of our products as treatments for cancer, chronic hepatitis B infection, other infectious diseases, alcohol use disorder or other diseases, and our ability to demonstrate the safety and efficacy of our drug candidates on a timely basis.
While we consider these assumptions to be reasonable, these assumptions are inherently subject to significant business, economic, competitive, market, and social uncertainties and contingencies.
Forward-looking statements and information involve known and unknown risks, uncertainties and other factors that may cause the actual results to be materially different from any future results expressed or implied by such forward-looking statements and information.
A more complete discussion of the risks and uncertainties facing Tekmira appears in our 10-K for the year ended December 31, 2013, which is filed on EDGAR and SEDAR and is available online under the Investors section of our website.
We do not expect to update forward-looking statements and information continually as conditions change, except as required by law. This conference call is being webcast live and the archive will be available on our website at tekmira.com following today’s call.
Mark will begin by providing a corporate update and Bruce will then outline some key financial highlights. Please note that we do report in U.S. dollar and we are in compliances with U.S. GAAP. At the end of the prepared comments, we will open the call up for your questions. So now, over to you, Mark..
Thanks, Julie. Good afternoon everyone, and thank you for joining our call today. This afternoon, I will highlight some recent developments in our business and provide an update on our clinical development programs along with key milestones to be reached by the year’s end.
I’d like begin by focusing on our RNAi antiviral product platform, specifically hepatitis B infection and Ebola virus disease. Our most compelling development program is TKM-HBV, targeting hepatitis B surface antigen expression in chronically infected hepatitis B patients.
This represents a significant unmet need in many thousands of patients chronically infected with hepatitis B virus. Our HBV therapeutic is designed to block surface antigen expression in these chronically infected patients.
Blocking surface antigen expression is indented to reduce the pathology associated with its expression in these patients, enable chronically infected patients to mount a full immune response against the virus or undergo what’s referred to as seroconversion, and finally result in the so-called functional cure.
In order to achieve this clinical objective, we’ve designed TKM-HBV to effectively block HBV surface antigen across multiple genotypes by implying multiple RNAi triggers in the third generation LNP formulation, the most potent available. The results of our preclinical studies with TKM-HBV indicate that we have achieved this objective preclinically.
These results were presented at the 10th annual meeting of the Oligonucleotide Therapeutics Society last month. Our results demonstrated the rapid and important reduction of hepatitis B surface antigen by TKM-HBV in several well validated models.
In these models TKM-HBV treatment resulted in reductions in both Intrahepatic and Serum, surface antigen, as well as several other viral markers, such as hepatitis B, hepatitis E and hepatitis C antigens. HBV DNA and Covalently Closed Circular or CCC DNA.
These data strongly support the utility of TKM-HBV as a new therapeutic option for treating patient with chronic HBV. Our next steps are to file our regulatory documents this year and initiate clinical trials in early next year. With these studies we look forward to generating more evidence of TKM-HBVs benefit in an area of significant and that need.
I will now shift my focus to our TKM-Ebola product development program and the company’s involvement in the current outbreak. Early this quarter the genetic sequence of the Ebola virus Guinea strain became available. This viral variant is responsible for the Ebola outbreak in West Africa.
With this sequence information we designed a new RNAi therapeutic which we call TKM-Ebola-Guinea, specifically targeting the Guinea strain. We have initiated GMP manufacture of TKM-Ebola-Guinea therapeutic and GMP product will be available in early December of this year. To further support continued development of anti-Ebola therapeutics, the U.S.
Department of Defense recently exercise an option in our current contract to allow for the manufacture of a modified RNAi therapeutic targeting the Ebola-Guinea strain. We have been awarded the option for scale-up in GMP manufacture of approximately 500 treatment courses of product which is valid at $7 million.
Now with all of our development programs, our objective is well with for TKM-Ebola is to seek license of a novel therapeutic which meets an un-met need. At the same time, we remain committed to making responsible contributions in this global public health crises.
To this end we are collaborating with an International Consortium led by the International Severe Acute Respiratory and Emerging Infections Consortium also referred to as ISARIC to conduct expedited clinical trials of promising therapeutic agents, which may include RNAi based investigational therapeutic agents in West Africa.
The Consortium was recently awarded $5.1 million from the Wellcome Trust to fund this initiative. We’ve also provided TKM-Ebola under the FDAs expended access program to treat several patients with confirmed or suspected Ebola virus infection. Now I want to underscore that in the interest of patients and institutions.
We are holding this information confidential and we do not disclose who has received TKM-Ebola. And further must be kept in mind that any uses of the product under expanded access, do not constitute control clinical trials. However, the data collected from this use in patient, will be provided to the FDA under Tekmira’s IND.
Now with recent developments, such as the production of a new product candidate for clinical trials in West Africa and the emergency use of our TKM-Ebola product under expanded access protocols. The clinical development pathways for our Ebola products have evolved and may continue to evolve.
So we may not resolve the partial clinical hold of the healthy volunteer multiple ascending dose portion of the Phase I trial of TKM-Ebola of this year. Now let me switch to PLK1. We remain on track with our clinical studies in oncology.
Our Phase IIa study with TKM-PLK1 is ongoing in two specific cancer indications, GI-NET or Gastrointestinal Neuroendocrine Tumors and ACC or Adrenocortical Carcinoma. In this study, our objective is to confirm and expand the clinical benefit observed in patients with these diseases.
Today, we achieved our enrollment targeted patients with both GI-NET and ACC Tumors. So these patients will continue treatment and be followed to determine if TKM-PLK1 results in meaningful clinical benefit. We plan to provide a status report on this study by year end.
Our second TKM-PLK1 study is a Phase I/II clinical trial in patients with hepatocellular carcinoma. ACC is associated with one of the poorest survival rates in oncology. Our study is designed to determine the safety, tolerability and clinical benefit of TKM-PLK1 and it’s being conducted at multiple sides in North America and Asia.
We have completed the enrollment of the first cohort in this study. Now with respect to expanding our pipeline, we continue to evaluate pre-clinical candidates with potential and diverse therapeutic areas, even the extremely high efficiency of delivery, for third-generation liver centric LNP formulations.
We are focused on rare diseases where the molecular target is found in the liver and where early clinical proof of concept can be achieved. As well where there may be accelerated development opportunities. We remain on track to nominate an additional candidate for clinical development by the year’s end.
I’d also like to take this opportunity today to mention Alnylam’s ALN-TTR02 program based on Tekmira’s proprietary LNP technology.
Alnylam recently reported results from its ongoing Patisiran Phase II open-label extension study and demonstrated sustained knockdown of serum TTR of up to 90% and a favorable tolerability profile in patients treated out to one year.
These data further validate our LNP technology as the industry gold standard and associated LNP with positive outcomes and clinical benefit. Now, turning briefly to our partnering and business development activities. We received an additional $1.5 million payment from Monsanto following the achievement of specified program developments.
Within this collaboration, Tekmira’s proprietary delivery technology is being applied to agricultural applications. The potential value to Tekmira could reach up to $86.2 million following the successful completion of all of the program milestones.
Finally, I would like to remind everyone on the call today that we’re hosting an Analyst Day in New York City on November 21st. I hope this provides a brief overview of Tekmira’s key developments over the past quarter. RNAi interference is one of the most promising and rapidly advancing frontiers in drug discovery and development.
Tekmira continues to be at the forefront of many breakthroughs in the RNAi therapeutic field. It is truly an exciting and busy time at Tekmira and we are pleased with our progress today. We look forward to reaching our forecast and milestones in finishing the year off strongly.
At the same time my team has remained fully focused on advancing our important clinical programs and key milestones to assure that we are achieving our promises to our investors, shareholders, and stakeholders like. I’ll pause here and turn things over to Bruce, who will provide some further detail on key financial milestones and achievements.
Bruce?.
Thanks Mark. Tekmira’s net loss from operations so excluding other income and expenses was $6.8 million for Q3 2014, as compared to $9.4 million loss for Q2 2014. Improvement and profitability in the quarter is entirely revenue related with total operating expenses tracking consistently quarter-on-quarter.
Terkmira’s net loss including other income and expenses was $8.6 million for Q3 2014 as compared to $6.1 million for Q2 2014. There were significant fluctuations in the fair value of outstanding warrants and foreign exchange gains and losses in Q2 and Q3.
Our net loss in Q3 2014 includes a non-cash charge of $5.1 million due to an increase in the fair value of our extending warrants. Conversely, our Q2 2014 net loss included a $5.8 million gain due to a decrease in the fair value of our warrants.
The change in warrant fair value are primarily related to movements in our share price from quarter-to-quarter. As far our foreign exchange gains and losses for operational purposes, we now hold large U.S. dollar cash reserves, so expect to continue to see foreign exchange gains and losses in line with movements in the U.S. Canadian exchange rates.
Looking up revenue in more detail. In Q3 2014 revenue was $4.4 million as compared to $1.8 million in Q2 2014. In Q3 2014 we’ve recorded $1.5 million from our U.S. Department of Defense contracts developed TKM-Ebola as compared to only $0.9 million in revenue in Q2 2014.
To the end of this contract increased as we have treated several patients with confirmed or suspected Ebola virus infections under an emergency IND authorization by the FDA. Our IND for TKM-Ebola under the DoD contract remains on partial clinical hold at this time.
We also recorded $1.6 million from the release of deferred BMS revenue with the exploration of our agreement and the end of obligations for whole parties. In addition, we recorded $1 million in Monsanto revenue in the quarter.
Turning now to expenses, total research development, collaborations and contract expenses remain consistent with Q2 2014 at $9.3 million. We continue to incur significant costs related to our HBV product candidate and we have ongoing cost for other programs including the commencement of our HCC trial for TKM-PLK1.
As at the end of Q3 2014, we have cash and investments balance up $120.5 million as compared to $129.5 million at the end of Q2. Our cash used in operations for this quarter was $8 million as compared to $6.7 million in Q2 2014.
The increase in cash use this quarter primarily relates to our efforts in preparation for filing an HBV, IND or equivalent by the end of this year. I’d now like to turn the call over to the operator who will manage the question-and-answer portion of the call.
Operator?.
(Operator Instructions). And our first question comes from the line of Michael Yee with RBC Capital Markets. Your line is now open. Please proceed with your question..
Hi, good afternoon. This is Judy Leung on for Michael Yee from RBC. Thank you very much for taking my questions. I have two questions today.
First one is, if you could provide a few more details on your Ebola study? What is the design and timing is for example? And my second question is on your upcoming Analyst Events on November 21, you had stated to expect some data from your oncology studies by your end as well as the nomination of additional candidate by your end, but could we expect that as soon as November 21 or what could we expect in terms of new information for the Analyst Day?.
Sure, go ahead..
So, firstly on the Analyst Event, both of those milestones, the additional development candidate, sorry….
In the PLK-1 update..
It remains somewhat variable at this time, it is not clear to us that we will have that data in time for the Analyst Day. It is certainly our objective, but it is not a firm commitment at this point, but we do remain on track for both of those milestones for this year..
Okay, great. Thank you..
With respect to the design of a clinical study in Ebola, you recall it that we are part of a collaboration involving the WHO and (indiscernible) Organization. And they are experts at conducting clinical studies in this area. We, a final protocol has not developed and so I think, it would be premature for me to comment on that at this moment..
Okay, great. Thank you..
Our next question comes from the line of Jason Kolbert with Maxim Group. Your line is now open. Please proceed with your question..
Hi, this is actually Jason McCarthy for Jason Kolbert. I had two questions; one of this is going to throughout my Ebola question. I just wanted to know if the emergency is patients were in the U.S. or if they were overseas. And my second question, it’s a little more complex.
I wanted to know there if you are if there are PLK-1 levels that are increased in late-stage HBV induced lever damaged and if there is potential to use the TKM-PLK and TKM-HBV together, these are in preclinical stage and you can kind of do this in animal models.
I was just curious if you’ve ever seen data like that?.
So Jason, with respect to the second question, I don’t know that we’re aware of that data. I’ll look into that, I don’t know if PLK is elevated in chronically infected patients..
And with respect to your first question Jason, we’ve not disclosed as Mark noted earlier details on the patients treated. I would say that the patients treated under the expanded access are primarily in the U.S..
Great thank you very much..
Our next question comes from the line of Michael Schmidt with Leerink. Your line is now open please proceed with your question..
Thanks for taking my questions. So I had a question on the DoD option exercise on the five hundred courses of the Guinea Ebola product is that in telling to the U.S. that’s part of the expanded access program or is that product that will also be used in the potential trunked study in Africa..
So Michael, good to hear from you, its Bruce Cousins, the DoDs intentions with that product are not been declared at this time..
Okay, and I guess on the clinical hold of your phase one, you said you won’t be able to resolve that issue this quarter.
Is that a question of resource allocation on your part or is there something else going on?.
It’s really a question of focus and potential relevant. We’re focused now as you can see I think on evaluating the products in patients in there is less need to evaluate the material in healthy volunteer subjects..
Okay, understood. And then I guess on the HBV program I know it’s early but I was just wondering if based on the preclinical data combined with the experience of the TKM Ebola which is based on the same platform technology.
If based on the data if you’ve a sense of what the repeating diseases would be needed to achieve a therapeutic effect in humans and obviously striking them on human primates’ studies here. And I was just wondering how confident you’re in the tolerability profile of the product in light of the experience in the Ebola Program..
So, I think the another which we can’t exactly predict the therapeutic dose as you know right.
We fully expected to be based on experiencing variety of programs to be under one milligram per kilogram, a well tolerated range and yes that’s – if I answered your question?.
I guess. So what I’m just wondering in the Phase I Ebola study you saw as opposed to cytokine release, dose is starting at 0.25 milligrams per gig.
And I was wondering if that somewhat translates to the HBV – the plan HBV study as well given that is the same delivery technology?.
Not. So it’s – I’m not – be careful not to, it’s a similar generation of technology, because, it may not be exactly the same. And remember that we – the Ebola program is being done without steroid cover in a product that is lay-off lives. And I don’t want you to directly compare that to what we would be doing in HBV.
I think it that’s potentially misleading..
Okay, understood. Okay, great thanks and congrats on the progress..
Thanks..
Our next question comes from the line of David Novak with Clarus Securities. Your line is now open. Please proceed with your question..
Hi guys, and thanks for taking the call. I have a few questions here. So, I’ll try to get through them as quickly as possible and let me practice by saying I understand your approach to TKM-Ebola. Your sensitivity to be over promotional and might have the tradigic situation currently unfolding.
However, I think its imperatives to highlight the importance of this program. And specifically what you get you to derisk, the terrific science that you’re currently carrying out, which internally have much of the implications for rest of your pipeline. For example, it could potentially be the most immediately monetizable asset that you have.
And furthermore, it could be the first FDI approved RNAi therapeutic in history utilizing LNP to suit. So, I definitely think it deserves a bit more attention.
So let me launch on from there, with respect to TKM-Ebola GI-NET, have you changed the trigger technology at all, I believe that the original TKM-Ebola was using encapsulated siRNA’s and we are now on to UNAs or we start using encapsulated..
No. So we are still using encapsulated. And just let me remind you that, sort of the product is comprised with two RNAi triggers targeting different viral protein products.
So the new product is the same – has the same target sites on the virus, but the – as made a couple of sequence changes to make it fully safe full to recognize GI-NET sequence in that zone. And the chemistry and the modifications are the same between both products..
So I guess you haven’t screen the Oligo design at all to potentially weed out those that are conferring immune stimulation.
So looking for a potentially Oligos in our less immunostimulatory for example? Or have you made any changes in reiteration at LNPs which could be L&T which is being used to mitigate the immune stimulation effect?.
So for the time being we’re using the same LNP, right. And you know that immune stimulation is driven on the payload end is driven primarily by the sequence, sequence motif. The modifications made – are made to mitigate that the activity of those sequence motif. So it’s done essentially bioinformatically..
So you have in fact looks for Oligos that potentially would be less stimulatory? Is that fair to say?.
Well, so we have not gone back and re-screened. We are targeting the same sites on the virus, but we have adapted them to know be fully homologous where there may have been a difference between the original strain and getting strain..
Okay..
I’m hope, I’m being clear here..
No you are. I just I know there are various bioinformatic techniques that you could use in silicon to determine whether or not they could potentially be stimulatory or not.
Right, so that might not have been a concern in the first generation so to speak, but with what you’ve seen with the cytokines, so I wonder if you took that into consideration, but yes..
No. we’re trying to make this – it’s close to the original product as we possibly can..
Got you, then….
We’re obviously reasoned..
Fair enough. So at the FDA given you any guidance in terms of how they were treat this new formulation. I mean will they allow the data from the first iteration, the second iteration and the guinea iteration.
Do they see them as comparable?.
Well it’s premature I would say. We are in regular discussions with them about it. That will be a novel approach. Will it be an interesting approach? I don’t know that one could achieve that..
Okay. And I mean a number of authorities have come for DoD (indiscernible) or even the Nebraska medical clinic who have implied that TKM-Ebola has been difficult in one manner and another to administer. I know that we’ve gotten word from you that it’s been well tolerated in all circumstances, I mean.
Is there anything further you can say this is just the first dose that it’s typically a little bit difficult to administer – any clarity you can get to us on this would be great..
So, I would just say that that anything you hear is entirely anecdotal. I mean these are individual patients being treated – most of them are quite sick, most of them are being treated in very different ways with – by very different medical practitioners with a combination of potential therapeutics.
So I think to draw a conclusion – any conclusion at all is way too premature..
Fair enough. So I didn’t catch Michael’s first question, you might have touched on this. With respect to the DOD funding – these inventories.
The owners now lay on them to approve using all cases and furthermore do they have to approval to disclose any data or observations emanating from this usage?.
Well, so when they purchase a product like this, they own the product, and they have – they will of course have a lot to say with how it is used and what’s done with the data..
I will move on to other aspects. You guys can breathe some size of relief on Ebola for now..
Okay..
Any further clarity around the Phase 1 for HBV, where it will take place, will it be in Europe, will be in China, any ideas with respect to that?.
Well, as you know, we haven’t disclosed that yet and I think we will tell you a lot more about that in the Analyst Day..
Okay great and nothing specific to update with respect to (indiscernible) endeavors using LNP for up regulation transcription by mRNA therapeutics?.
Well, you do know it’s an area that we’re very interested in and we continue to explore and I think we’ll update you on that at the Analyst Day as well..
I’m looking forward to seeing you guys at the Analyst Day then..
Okay, excellent. .
Thank you very much..
Our next question comes from the line of Steven Willey with Stifel. Your line is now open. Please proceed with your question..
Yes, hi. Thanks for taking my question. I think in one of your responses to prior question drawing the distension between Ebola and HBV. I think you kind of commented that Ebola was administered without steroid cover.
Would that been assume that HBV is going to be administered in the context of the steroid cover?.
No Steve, I hope I didn’t – thanks for asking the clarifying question. We don’t anticipate using steroid cover in the HBV program..
Okay. And then I know that you’ve talked about new products candidate reveals here that are going to be happening maybe at the Analyst Day and I guess in the little close to the next year or so.
And just given the fact that there is a bit of a final universe of rare disease liver centric targets and the landscape for those targets is kind of growing increasingly competitive I’m just kind of curious as to whether or not is if that competitiveness has kind of changed the way that you have think about disclosing what some of this novel candidates are?.
Well, I think that it causes us to – before we disclose we want to be sure that we can as much as possible differentiate ourselves somewhat else might be out there Steve. So maybe that’s the way of seeing it.
We are not trying to keep others from knowing what we are doing, we are just trying to be clear about what we are doing and how it’s differentiated when we do it.
Did I answer your question?.
Yes, I guess. And then, so I guess the lack of resolution around the clinical hold for TKM-Ebola by year-end.
I mean how much of that is quote unquote this evolving clinical development corporate and how much of that is more of an emphasis being placed on the guinea strain product?.
Well, I think there’s sort of hand-in-hands, right. We are – the current outbreak is being driven by guinea. So we are focused on that product and collecting data with that product. And we think some of that data may read over onto the other product.
It’s just pushing forward in the next level of the healthy volunteer study, it’s just not terribly meaningful at this moment..
Okay. Thanks for the color..
Thanks, Steve..
Thank you. And that concludes our Q&A session for today. I would like to turn the call over to Julie Rezler for any closing remarks..
Thank you, Nicolas. We appreciate everybody’s participation in the call today, and we look forward to sharing our updates on our continuing progress with you in the months ahead. So this concludes our call. Thank you very much. Bye, bye..
Ladies and gentlemen thank you for participating in today’s conference. This does conclude the program and you may all disconnect. Have a good day, everyone..