Lineage Cell Therapeutics, Inc. (LCTX) Q1 2019 Earnings Call Transcript
Published at 2019-05-10 00:09:06
Welcome to the BioTime, Inc., First Quarter 2019 Earnings Conference Call. At this time, all participants are in a listen-only mode. An audio webcast of this call is available on the Investors section of BioTime's website at www.biotimeinc.com. This call is subject to copyright property of BioTime, Inc. and recordings, reproduction or transmission of this call without the expressed written concert of BioTime is strictly prohibited. As a reminder, today’s call is being recorded. I would now like to introduce your host for today, Ms. Ioana Hone, Director of Investor Relations at BioTime. Ms. Hone, you may begin.
Thank you, Nova. Good afternoon and thank you for joining us. A press release reporting our first quarter 2019 financial results was issued earlier today May 9, 2019 and can be found on the Investors section of our website. Please note that today's conference call and webcast will contain forward-looking statements within the meaning of federal securities laws, including statements regarding our strategy, goals, product candidates and clinical trials, expected synergies and benefits of the Asterias acquisition and financing matters. Such statements are subject to significant risks and uncertainties, including those described in our press release issued on May 9, 2019 and our SEC filings on Form 8-K, Form 10-K, and Form 10-Q. Actual results or performance may differ materially from the expectations indicated by our forward-looking statements, due to those risks and uncertainties. We caution you not to place undue reliance on any of the forward looking statement, which speak only as of today. Joining us today are our Chief Executive Officer, Brian Culley; our Chief Financial Officer, Brandi Roberts; our Chief Medical Officer, Ed Wirth; and our Senior Vice President of Clinical and Medical Affairs, Gary Hogge. The executives will provide prepared remarks, then take questions from analysts and institutional holders. With that, I'd like to turn the call over to Brian Culley, our CEO.
Thank you, Ioana. Good afternoon, everyone, and thank you for joining today's call. We have continued to pave the way toward our objective of becoming the leading cell therapy company. With the successful close of the Asterias acquisition, we now have three clinical stage cell therapy assets in our pipeline, which means 2019 will be an important year in terms of completing our open trials and planning for our next ones. I’ll now highlight some recent developments with OpRegen, OPC1 and Renevia before asking Brandi to review our updated financials. Beginning with OpRegen, last week one of our study investigators presented updated results from our ongoing open label Phase I/IIa clinical study of OpRegen for the treatment of dry-AMD with geographic atrophy. This work was presented at the 2019 Association for Research in Vision and Ophthalmology annual meeting also known as ARVO. And we're pleased to report that treatment with OpRegen continues to be well tolerated and in some patients signs of structural improvement in the treated areas of the retina, has been observed. Importantly, the initial data from the better vision Cohort 4 patients, those who have less advanced dry-AMD and smaller areas of GA remain encouraging with indications of the continued presence of the transplanted opportune cells and some improvements in visual acuity. The updated and detailed data from this ongoing study is available at the Events and Presentation section of our website. There you can see evidence of rapid healing of the injection site, details on the systemic and ocular safety of OpRegen and walk through a number of case studies, which collectively give us confidence that we're on the right track as we continue to enroll better vision patients for Cohort 4. Our next step with OpRegen is to deploy an improved delivery system in at least the next six patients. We believe more precise delivery of cells to the back of the eye will lead to a reproducible safer and ultimately an overall superior procedure with an increased likelihood of positive clinical outcomes. And as you'll recall, in January, we entered into an exclusive agreement with Orbit Biomedical to use their subretinal delivery system for more precise administration of OpRegen cells to the eye. I would like to highlight that Orbit is a specialty medical device company founded through the acquisition of the leading surgical platform for self-retinal delivery and the transfer of key individuals from the Janssen biotech unit of Johnson & Johnson to the Orbit team. The Orbit subretinal delivery system received the FDA 510(k) clearance in November of 2018 and we announced that we had entered into an agreement with them in January of 2019. Orbit's focus is to transform the accuracy safety and consistency of delivering therapeutics to the sub retinal space of patients suffering from site threatening diseases. And we believe, there is no comparable device available to deliver cells in the precise and careful way which Orbit provides. We have already submitted a protocol amendment to the FDA to add the Orbit device to our protocol and pending the 30 day waiting period. We expect to begin dosing pit subjects with the Orbit device this quarter. We also plan to introduce our new thaw and inject formulation which should be much easier to use and eliminate the need for extensive dose preparation. Consequently, we expect to be in a position to provide important updated data on the OpRegen Orbit treated patients and our new formulation no later than at the American Academy of Ophthalmology Annual Meeting in October. Moving to our second program OPC1 which I'll just remind everyone is the delivery of oligodendrocyte progenitor cells to treat spinal cord injury. Just a few days ago, we announced the issuance of a patented from the U.S. Patent and Trademark Office for a method of reducing spinal cord injury due to preventable cavitation. Cavitation is a destructive process that occurs within the spinal cord following injuries and typically leads to permanent loss of motor and sensory function. A key finding from our Phase 1/2a study of OPC1 was that 95% of subjects showed evidence by MRI scans that OPC1 cells and grafted at the injury site and helped to prevent cavitation. We think preventing cavitation is one of the key mechanisms by which OPC1 can help improve outcomes in SCI patients, and notably the recently issued patented would have a term that expires no earlier than 2036 and further solidifies our competitive advantage in this space. Also pertaining to our OPC1 program, last month our Chief Medical Officer, Dr. Edward presented additional findings from our SCI clinical trial at the 26th Annual Conference of The American Society for Neural Therapy and Repair. The primary goals of the study were met and they included observing the safety of OPC1 in cervical SCI patients, metrics related to the optimal timing of the OPC1 injection. The tolerability of the immunosuppression regimen, the engraftments of OPC1 cells and rates of motor recovery among different study subpopulations. These data provide us with the information needed to design the next clinical study. For example, we observed that among the 25 subjects treated in the study, the two worst performing subjects both also had post-operative cord compression. We do not think that this is a coincidental finding and it highlights the kind of situation that we will seek to mitigate in the next trial through careful protocol design and patient selection criteria. If you'd like to review these encouraging results for yourself, we've provided some patient level data on our website in the events and presentation section just as we did for the interim data from the OpRegen study. Although, we do still have a number of additional analysis to do on the OPC1 trial and we must meet with the FDA to propose our next steps. We do expect our next study to be randomized and to be designed to provide evidence that our OPC1 cells provide a clinically meaningful benefit to SCI patients. Lastly, we appreciate the generous support of the California Institute for Regenerative Medicine or CIRM whose grant funding has helped not only to advance the clinical development of our OPC1 program, but also to generate these encouraging clinical results and from whom we intend to request additional support for the OPC1 program later this year. Turning next to Renevia, our medical aesthetics program for HIV lipoatrophy. We submitted our CE Mark application for Renevia last year and its review is still ongoing. We previously estimated that we would receive a response during the second half of this year. But based on recent information from our regulatory contacts in Europe, we believe we now are in the final stages of the review process and we currently estimate that we will receive a response this quarter. Although a successful CE Mark registration reflects well upon this organization's ability to obtain marketing authorizations. I've explained previously that Renevia itself does not fit strategically into our new focus on cell therapy treatments. So if and when our application is approved, we intend to seek an external partner for the commercialization of our Renevia in Europe. So to sum up, we remain focused on setting up BioTime for long term success. Key actions we've taken toward this goal include spinning off our early stage assets into AgeX, gaining two new clinical programs by the Asterias acquisition and hiring a brand new management team including a CEO, CFO and new General Counsel who starts later this month. Importantly, we are now solely focused on making clinical progress with our three cell therapy programs. We also intend to establish a new brand and corporate image, which will highlight our commitment to cell therapy, so you can expect to hear about those updates in the coming months. Overall, we're pleased to report on another successful quarter featuring clinical data updates, accelerated guidance and additional steps, which we believe will position the company for durable success. I now will hand things over to Brandi to review our financials and discuss some additional plans for this year.
Thank you, Brian. Let me start the financial discussion with some balance sheet highlights. At March 31st, 2019, BioTime's cash, cash equivalents, and marketable securities totaled $27.1 million. Our investment in OrthoCyte was value at $58 million as of March 31st, 2019. Our investment in OrthoCyte grew by more than $35 million in the first quarter due to significant stock appreciation based on positive data OrthoCyte announced in January 2019. OrthoCyte continues to do well and accordingly, our investment in OrthoCyte was worth approximately $66 million as of yesterday. Our promissory note due to us from Juvenescence had an outstanding balance principle plus accrued interest of $22.5 million as of March 31st, 2019. If Juvenescence completes an IPO with proceeds of at least $50 million prior to our note maturity, then the note will convert into Juvenescence stock. The conversion is subject to an upward adjustment if the 20-day vwap of AgeX stock is above $3. Based on yesterday's closing price, the 20-day vwap of AgeX is $4.57. If the conversion were to take place now, the number of shares to be issued would be adjusted upward by roughly 52% and the shares at the time of issuance would be worth more than $32 million based on yesterday's closing price. If the promissory note is converted, our Juvenescence stock will be a marketable security that we may use to supplement our liquidity needs. If the promissory note is not converted, it is payable in cash plus accrued interest at 7% per year, which will be about $24.6 million at maturity in August of 2020. We believe that Juvenescence is a credit-worthy debtor and will be able to service this payment. We also added new intangible assets to our balance sheet in the first quarter of 2019 due to the Asterias merger with a fair value of OPC1 and VAC2 were recorded as $31.7 million and $14.8 million respectively. As I get ready to highlight the statement of operations, I'd like to remind everyone that beginning on August 30th, 2018, BioTime ceased recognizing revenues and expenses related to AgeX and its subsidiaries due to the AgeX deconsolidation on that date. Total revenues for the three months ended March 31st, 2019 were $900,000, an increase of $200,000 compared to $700,000 for the same period in 2018. The increase was primarily related to a $400,000 increase in grant revenues offset by a $200,000 reduction in subscription and advertisement revenues attributable to the AgeX T consolidation in 2018. Total operating expenses, which are comprised of research and development expenses and G&A expenses for the three months ended March 31, 2019 were $13.6 million as reported and $7.9 million as adjusted. In addition to stock-based compensation and depreciation and amortization expense, there were also $3.5 million of Asterias merger transaction related costs that were excluded from total operating expenses as adjusted. The reconciliation between GAAP and non-GAAP operating expenses by entity is provided in the financial tables included with our earnings release. Research and development expenses for the three months ended March 31, 2019 were $5 million, a decrease of $900,000 compared to $5.9 million for the same period in 2018. The decrease was primarily related to a $1.6 million decrease from the AgeX T consolidation and the absence of AgeX research and development expenses incurred after August 30 2018, offset by a net increase of $600,000 in BioTime programs, primarily related to an increase of $800,000 in OpRegen related expenses, an increase of $600,000 in OPC1 and VAC2 expenses, and reduction of $800,000 in Renevia and high-stem expenses. General and administrative expenses for the three months ended March 31 2019 were $8.7 million, an increase of $2.7 million compared to $6 million for the same period in 2018. The increase was primarily attributable to increases of $3.5 in severance, legal and related costs related to Asterias merger and $500,000 in non-cash stock-based compensation expense due to additional equity award grants. These increases were fully offset by a decrease of $1.3 million from the AgeX T consolidation and the absence of AgeX general administrative expenses incurred after August 30 2018. Other income or expense nets for the three months ended March 31, 2019 reflected other income net of $47.7 million compared to other expense net of $51.5 million for the same period in 2018. The increase was primarily related to changes in the value of equity investments in office site and Asterias for the applicable periods. The net income attributable to BioTime for the three months ended March 31, 2019 was $39.3 million or $0.30 per share basic and diluted compared to a net loss attributable to BioTime of $63.5 million or $0.50 per share basic and diluted for the same period in 2018. As we mentioned on our last earnings call in March due to our extensive redundancy across our existing GMP facility in Jerusalem and our recently acquired GMP facility in Fremont, we plan on realizing significant synergies from Asterias merger. We are still in the process of transitioning manufacturing activities from Fremont to Jerusalem, but the integration is going well and according to our timeline. By June 30, we expect to complete our transfer of activities and a reduction of force of more than 85% Asterias employees as their roles and tasks are transferred to others at bio time in the U.S. and Israel. In terms of guidance, we anticipate that our cash burn for 2019 will be in the range of $32 million to $34 million. We believe that our 2019 net expense budget will be in the range of $36 million to $38 million excluding non-cash items. The difference of $4 million between our cash burn and the net expense budget is related to $2 dollars of net cash that was obtained from Asterias in the merger, as well as another $2 million collected from OncoCyte for prior year shared services in February 2019. Our projected 2019 net expenses of $36 million to $38 million despite absorbing one-time charges of more than $8 million related to the Asterias acquisition and the license of the Orbit device are significantly less than the 2018 expenses of approximately $45 million for the combined companies. This extensive savings is attributable to the sale and distribution of our preclinical assets pertaining to AgeX, the consolidation of manufacturing efforts in Jerusalem and the new management team’s commitment to substantial oversight of costs. In coordination with completing our integration of Asterias into BioTime, we are also evaluating the best course of action for bringing in additional capital to the company. We are committed to making BioTime a premier cell therapy company and to improving our chances of success by ensuring it is well-funded. We currently have multiple funding options available to us. We can seek to leverage our investments in OncoCyte or AgeX. We can seek to leverage the promissory note due to us from Juvenescence. As of March 31, 2019, the value of our cash, marketable securities, equity positions and our affiliate companies and the balance of the promissory note due to us in August of 2020 was worth more than $100 million. We understand that we need additional capital to progress our clinical stage programs and we will seek to make the best move or combination of moves at the opportune time. With the changes made in the last eight months, we believe that we have stabilized the company's cost structure and are poised to capitalize on the three clinical stage assets that we are currently developing. We will also continue to evaluate funding and strategic alliance opportunities through our existing and future potential partners. With that, I will turn the call back over to Brian.
All right. Thanks Brandi. As you have seen, we have been rapidly transforming BioTime into a leading cell therapy company through strategic transactions on the corporate development, clinical and operational fronts and we have no plans to slow the pace of progress. We will remain focused on advancing our clinical stage programs in a thoughtful and cost effective manner throughout 2019 and we'll update our investors on our timelines achievements and regulatory plans. Some milestones to look forward to this year include the initiation of dosing with the Orbit device and a new thaw and inject formulation in our ongoing clinical study of OpRegen which is expected this quarter. Decision on BioTime CE Mark application for Renevia which is now expected also this quarter, continuing the clinical advancement of the OPC1 program including manufacturing improvements and our plans to meet with the FDA to discuss next steps in the clinical development of that program, strengthening the partnerships with CIRM and Cancer Research UK for the ongoing support of the OPC1 in fact two programs. Additional milestones include completion of patient’s enrollment in our Phase 1, 2 clinical study of opportune which is anticipated by the end of this year, evaluating the development of OPC1 as a candid for multiple sclerosis and the ischemic stroke through some ongoing research collaborations we have with major universities and lastly an increased presence and engagement with the patient, physician and advocacy communities. So to conclude, we believe that BioTime is heading in an exciting new direction and our goal is to build awareness and support for a reinvigorated and repositioned company. This management team’s priority and focus in 2019 will be to deliver on our stated milestones and continue to increase our visibility in the investment community through continuous communication and engagement. With that operator, the team here is ready for questions.
Thank you. [Operator Instructions] Our first question comes from the line of Kevin DeGeeter from Oppenheimer. Q – Kevin DeGeeter: Good afternoon, guys and thanks so much for taking my question. With regard to the gating factors enroll over the next six patients for OpRegen, I mean, appreciate the granularity of calling out the protocol amendment for the Orbit device and thaw and inject, which of those appears to be the gating timeline factor to initiate that? And against that backdrop, can you also provide us an updated thinking in terms of your expectation of whether DSMB would -- maybe comfortable allowing you enroll patients concurrently or will continue to seek full safety follow-up on these patients before enrolling the next patient?
Yeah. Hi, Kevin. I think it's a good question for Gary Hogge who runs that program. So, Gary please.
Yeah. Thanks, Kevin for the question. So the independent Data Safety Monitoring Board has already given approval to proceed with the protocol as it was submitted to the agency. Basically that would involve a gating on the first two subjects, provided that goes well, the DSMB is going to reconsider simultaneous or open enrolment at that point provided things go well. In the FDA, we're waiting for the period of comment – no comment to expire.
Right. And with regard to the amendment pertaining to the device versus thaw and inject, which – how should we think about the steps so each one of those to permit you to reinitiate testing?
So, the thaw and inject formulation has already been gone through the CMC amendment back in January timeframe to the FDA, and they had no objections to it at that time. So essentially we've just added that to the over device, which again is cleared already. So we don't anticipate and again it's the same target population as was previously involved with the prior amendments and method of delivery. So it is our expectation that we shouldn't receive any objection.
And one more question if I may then I'll get back in the queue, and that pertains to OPC1, appreciate the granularity in terms of how to think about a potential coming meeting with FDA. But I think you specifically called out endpoints in future studies that may confer or allow clear evaluation of clinical benefit in these patients. Can you expand on your current thinking as to, which endpoint to what general series of endpoint, do you think ultimately are the most clinically actionable for these patients in quite limited current treatment options?
Yeah as you know, there's no regulatory presence, there's no approved agent. So let Ed address the -- Dr. Wirth, our CMO will address the current state of affairs with respect to endpoints in spinal cord injury.
Yeah. Thanks. That's a good question. So this is an area of very active research and development within the SCI community. Just within the past couple of years there have been some important new outcome measures that we think will be potentially useful for demonstrating clinically meaningful function from OPC1. So just to call it a couple for example, one is a new welcome measure that was published called the capabilities of upper extremity test which allows functional assessment of the entire arm and hand which would that -- we complement the motor recovery that we've been looking at previously. Another one is a patient reported outcome that's been validated and published called the spinal cord injury functional index. And again as patient -- report outcome by definition, we believe that that's a clinically meaningful improvement if you see improvement on that as well. So these are a couple of things that would be incorporated into the protocol in the design of the next randomized study. So we're feeling pretty good about where the field is moving in this regard and we will look at how those relapse occur in the next study.
Extremely helpful. Congratulations on all the progress.
Our next question comes from the line of Jason McCarthy from Maxim.
Hi guys. Thanks for taking the questions. I want to stick with OPC, the OPC1 program. First question is, you had mentioned that the two I guess worst performing patients in the Phase I/IIa study had post-operative compression. Is that something that you would look to possibly screen out in the next study, in the next randomized study? What do these patients potentially benefit from a higher dose of cells? And my next question is related to the OPC1 manufacturing. Now that you've brought everything in-house from mysterious, do you expect it to be a pretty seamless transition to the site -- manufacturing site in Israel?
It’s one of few two part A and two part B.
Okay. Thanks Brian. Sounds good.
Yes. So with regard to the cord compression, it's almost certainly do and again based on the MRI scans to a fluid that's commonly is found outside the spinal cord, but under the skin after surgical procedures. This is common to virtually every surgical procedure. And the surgeons tend to manage it very differently. And in talking with all of our surgeons, it's -- they, almost unanimously, agree that something like this could be pretty easily preventable in future trials, simply by requiring that all the surgeons insert a drain tube overnight after surgery to pull off that fluid that tends to accumulate soon afterward. And in some cases that we've seen, can potentially be compressed the spinal cord. So we think that that's a very straightforward and easily accessible issue in future studies.
And then, Jason, with respect to manufacturing, in fact, although, I would argue that tech transfer is never a simple process. The benefit and one of the reasons that we did this acquisition is that we have a highly skilled stem cell therapy manufacturing team at our GMP facility in Israel. They have successfully demonstrated things such as moving from two dimensional growth on plates of plastic to growing cells reproducible in multi-liter bioreactors. So those are the kinds of features and improvements and enhancements that we want to do with the OPC1 process as it stood, when we got it from Asterias. We want to get it into the hands of our accomplished team and introduce some of those, what I call, pre-commercial attributes and then introduce that -- those improvements into an ongoing clinical trial.
Last question. I know, strategic focus is not Renevia. Once you get to CE Mark, you mentioned that you would seek a partnership for commercialization in Europe. Would you do that while retaining right to the U.S. or would U.S. rights go to a potential European partner as well?
The correct answer is, it depends. If there is an entity which is interested in global rights and there's a fair price for that, then we're delighted to do that. If there's an entity that is only interested in regional -- limited regional rights such as Europe and the price is right and we're open to that. I think what we would intend to do is, take a very broad approach, a broad business development campaign and figure out what's out there for us and then try and choose the best deal for the company.
Our next question comes from the line of Reni Benjamin from Raymond James.
Hey, good afternoon, guys. Thanks for taking the questions. Maybe just on the thaw and inject formulation. Can you talk a little bit about what sort of comparability tests were done? And has they -- have the cells need to have been modified in any way to kind of make sure that the data that we've seen so far and what we're going to get with this new formulation, are going to equivalent?
Yes, Reni. That will go back to Dr. Hogge.
Yeah, Reni. So the OpRegen is OpRegen. Essentially AgeX simply means that the media in which it is frozen in and its store is an FDA approved product using other cell therapy procedures gone in clinical trials to-date and we've prepared extensive compatibility testing to show that it performs as well as the previous version of OpRegen which again the cells are unchanged simply the way they're frozen down. And we've also completed all those compatibility tests with the Orbit device. Previously we administered them through a [Indiscernible]. So, we're quite confident that the OpRegen is the same. It's simply the way it's frozen down and able to be delivered to sites.
And just as a follow-up regarding the Orbit device, is there any specific training or special training that needs to occur is that also again in fact are not just to starting but then also to continue expand in those patients?
Right. So the surgeons who are involved – clinically involved and trained, specifically by an Orbit train facility. So we are good to go from that perspective. We don't intend to involve additional sites at this time. We think that the sites that are involved will complete the study within the timeline is outlined. So we're good to go from that perspective. But yes, additional training is required. But it is simply a one day training that is relatively routine and the surgeons come out very enthusiastic about the ease in which the cells are delivered.
Got it. And just one final question for me regarding Renevia speaking with potential external partnership, have there been any discussions to-date, has there been any interest? And I guess, if you could kind of characterize what looks likely I am imaging to be distribution type deal what is the ideal deal look like?
I would of course want some form of an auction situation. So we want to bring multiple parties to the table. It doesn't lend itself to a simple answer, because it's going to be heavily dependent on the label. If it is a device that's limited to HIV Lipoatrophy, it's got a very different look and feel than if it's more of a broad label. And we would be interested in knowing what sort of capabilities and commitments to further studies that a potential partner would bring to the table. So the process here Reni is to engage a European based business development group, or individual have them do. I would call a conventional process, bring opportunities to the table and let the organization negotiate those and try and find a deal that works well for us.
Great. Thanks for taking the questions.
You bet. Thank you, Reni.
Thank you. And at this time, this concludes our Q&A session. And I would now like to turn the conference back over to Brian Colley your CEO. Sir?
Great. Thanks. I appreciate everyone joining us this afternoon. I'm obviously excited about our plans. Hope we'll be able to keep this positive momentum going for a long time. Thank you and have a great afternoon and evening.
Ladies and gentlemen. Thank you for participating in today's conference. This does conclude the call. You may now disconnect. Everyone have a wonderful day.