Good morning. My name is Crystal and I will be your conference operator today. At this time, I would like to welcome everyone to Karyopharm Therapeutics Second Quarter 2018 Results Conference Call. There will be question and answer session to follow. Please be advised that this call is being recorded at the company's request.
I would now like to turn the call over to Mr. Ian Clark, Karyopharm's Vice President, Investor And Public Relations.
Thank you, Krystal, and thank you all for joining us today us on today's conference call to discuss Cariopharm's second quarter 2018 financial results. This is Ian Karp, and I'm joined today by Michael Kauffman, Chief Executive Officer Mr. Michael Falvey, Chief Financial Officer Doctor. Sharon Schockham, our Founder, President and Chief Scientific Officer and Mr. Chris Primiano, Chief Business Officer.
On the call today, Michael Kaufman will make some introductory comments that Mike Valvey will provide an overview of the second quarter 2018 financial results. Doctor. Kaufman will then discuss our upcoming key milestones and provide some summary remarks. We will then open the call up for questions for which Sharon Chris and I will also be available. Earlier this morning, we issued a press release detailing Karyopharm's results for the second quarter 2018.
Additionally, we issued a separate release yesterday afternoon announcing the completion of our rolling submission to the U. S. FDA for a new drug application for selinexor, our lead clinical candidate. Both releases are now available on our website at carriopharm.com. Before we begin our formal comments, I'll remind you that various remarks we will make today constitute forward looking statements for purposes of the Safe Harbor provisions to the Private Securities Litigation Reform Act of 1995.
These include statements about our future expectations, clinical developments, regulatory matters and timelines, the potential success of our product candidates, financial projections, and our plans and prospects. Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed in the Risk Factors section of our most recent quarter report on Form 10 Q, which is on file with the SEC and in other filings that we make with the SEC in the future. Any forward looking statements represent our views as of today only. While we may elect to update these forward looking statements at some point, we specifically disclaim any obligations to do so, even if our use change. Therefore, you should not rely on these forward looking statements as representing our views as of any date subsequent of today.
In addition, Please note that any references we make to clinical trial data during today's discussion refer to interim unaudited site data unless otherwise specified. I'll now turn the call over to Doctor. Michael Kaufman, Chief Executive Officer of Caryopharm.
Thank you, Ian, and good morning, everyone. Thank you for joining us on today's call. Over the past few months after receiving Fast Track Designation, we have made tremendous progress advancing our lead selinexor program and completed the submission of our first new drug application, the United States Food And Drug Administration, seeking accelerated approval for selinexor in multiple myeloma. The NDA is supported by data from our Phase 2b STORM study which evaluated selinexor in low dose dexamethasone to treat patients with pentor refractory disease. These patients have previously received 2 proteasome inhibitors velcating Kyprolis, the 2 immunomodulatory drugs Revlimid and POMALISSE and the anti CD38 monoclonal antibody DARZALEX.
As well as alkylating agents and their disease is also refractory to at least one proteasome inhibitor at least one image DARZALEX and their most recent therapy. There are about 120,000 patients living with myeloma in the United States and over 30,000 new cases expected this year. Unfortunately, are also expected this year. This is the first new drug application submission of a novel mechanism for the treatment of relapsed refractory myeloma since DARZALEX was approved in 2015. It is an important achievement for both carryout farm and for patients battling this difficult to treat cancer.
Their storage continue to hire us every day and we're grateful to all of the physicians, caregivers, patients and families who've contributed to the selinexor program to date. We also greatly appreciate the FDA's collaboration and support during the to better serving the needs of patients with pemter refractory myeloma. We also believe that our recent NDA submission marks only the beginning for Karyopharm. We remain convinced that selinexor has the potential to become an important option for myeloma patients in earlier lines of therapy as well as for patients suffering from other forms of cancer sir. On the commercial front, we have been making exciting progress building our commercial infrastructure in preparation for the first potential selinexor product launch in the United States During the first as well as our new Chief Commercial Officer, Anon Baraden, formerly of Qiasma, Amgen, and Procter and Gamble.
Anon is a twenty five year old industry a 25 year industry veteran with a strong track record of building successful marketing teams and commercializing novel medicine and we are delighted to welcome him for the executive team as we continue building our commercial foundation for the future and for the record, Anand is more than twenty five years old. These regulatory and commercial initiatives are supported by the positive top line results reported during the quarter from part 2 of the Phase 2b STORM study evaluating twice weekly oral selinexorpluslow dose dexamethasone or cell Dex in patients with pentorefractory myeloma. For the STORM studies primary objective, oral seldex achieved 25.4% overall response rate as assessed by an independent review committee. In addition to the 29 partial or very good partial responses, there were 2 stringent complete responses, which were negative for minimal residual disease. The median duration of response is 4.4 months and patients with any response to Celldex including minimal partial, very good partial and complete responses had a significantly prolonged overall survival as compared with patients who did not respond.
On the safety front, oral selinexor demonstrated a predictable and managed tolerability profile, consistent with that previously reported from Part 1 of the STORM study with no new safety signals identified. When occurring, the adverse effects were often reversible, transient and manageable with dose modification and or standard supportive care. We plan to present the detailed storm study results upcoming medical oncology meeting. In addition to serving kinds of refractory population, our development strategy is aimed at advancing selinexor to reach patients in earlier lines treatment. We are conducting the pivotal randomized Phase III BOSTON study to investigate once weekly oral cell Dex.
In combination with once weekly Velcade compared to standard twice weekly Velcade Dex alone in patients with myeloma who had 1 to 3 prior line therapy. To our knowledge, this is the first combination regimen utilizing once weekly subcutaneous felting on the experimental arm. This regimen could provide greater convenience with once weekly physician office visits as well as reduced rates of Velcade associated side effects versus the usual twice weekly VELCADE regimens. We expect to complete enrollment in Boston this year and report top line data in 2019. Assuming a positive outcome, we believe the data from as a evaluating sel dex in combination with several standard approved therapies, including Revlimid, pamilist, Velcade, Tekrelis, and DARZALEX, in patients with relapsed or refractory myeloma and in combination with Revlimid in patients with newly diagnosed myeloma.
In June at EHA 2018, we reported updated data from the VELCADE, POMALISSE and DARZALEX arms. All three of presented arms continue to show robust anti myeloma activity and manageable tolerability when combined with these standard approved therapies. Given the observed synergistic activity of selinexor with these anti myeloma therapies, we believe oral selinexor has the potential to be a future backbone therapy in myeloma. In addition to myeloma, we are also developing selinexor for the treatment of diffuse large B cell lymphoma or DLBCL. We remain on using single agent selinexor in patients with relapsed or refractory DLBCL who are not candidates for stem cell transplantation.
If the final results of the SADAL study are positive, we plan to submit an NDA for accelerated approval in the first half of twenty nineteen for this indication. Finally, in addition to the key clinical and regulatory highlights I just outlined, we also made significant progress towards expanding our geographic reach for both selinexor as well as our other pipeline assets. In May, we entered into a strategic partnership with Antigene Corporation for the development and commercialization of selinexor and additional pipeline assets Eltonixor, Vertonixor and KPT-nine thousand two hundred and seventy four in China and other important regions in Asia. Entin gene has a strong clinical regulatory expertise and capabilities in China and the covered territories. This partnership, which carries a total deal value up to $162,000,000 plus royalties creates an important alliance, which nice to complement our existing partnership with Omar Pharmaceutical for Japan and certain other Asian countries.
This impressive set of partners will be an important part of the global advancement of our novel oral drug candidates in these important markets With that
earlier today outlining our full financial results, I'll just review our second quarter 2018 financial highlights. As of June 30, 2018, cash, cash equivalents and investments, including restricted cash, totaled $250,500,000 compared to $176,400,000 as of December 31, 2017. In May 2018, we completed an underwritten book offering of just over 10,500,000 shares of our common stock at a price to the public of $14.75 per share. The net proceeds Karyopharm from the offering after deducting for underwriting discounts and commissions and other estimated offering expenses were $145,700,000. For the quarter ended June 30, 2018, Karyopharm recognized $19,900,000 in revenue compared to a small amount of grant revenue 3 months ended June 30, 2017.
The increase in revenue was primarily the result of recognizing $19,700,000 of revenue related fulfilling an obligation under our license agreement with Ono. The cash related to this revenue was part of the upfront payment received from Ono in October 2017. For the second quarter of 2018, research and development expense was $44,700,000 compared to $23,100,000 for the same period in 2017. General and administrative expense for the second quarter of 2018 was $9,500,000 compared 17. Comparing the second quarter of 2018 to the prior quarter, the first quarter of 2018, R and D expense increased by $3,400,000 reflecting increased spending on activities associated net loss of $33,700,000 or $0.60 per share compared to a net loss of $29,400,000 or $0.64 per share the second quarter of 2017.
Net loss included stock based compensation expense of $4,400,000 $5,100,000 for the second quarters of 2018 2017, respectively. Aeriopharm expects its operating cash burn including research and development in general and administrative expenses for the year ended December 31, 2018 to be in the range of $75,000,000 to $185,000,000. Based on our current operating explant plans, we expect that our existing cash, cash equivalents and investment will be sufficient to fund our operations into the third quarter of 2019. Importantly, this runway takes us through the potential launch of selinexor in the first half of twenty nineteen. These plans include the continued clinical development of selinexor in our lead indications and preparing the commercial infrastructure, including hiring a sales force support the potential launch of Selinexor in the U.
S. I'll now turn the call back over to Michael Kaufman for concluding remarks.
Yeah. Thank you, Mike. Before I review our upcoming milestones and move to the Q And A, I'd just like to take a moment to formally welcome our newest member of the team. Our Vice President, Investor And Public Relations, Ian Karp. Ian brings over 20 years of Investor Relations, Corporate Development And Strategic Communications Experience.
And I'm confident that he will serve as a key resource to our investors as we continue the significant progress we are making. Moving on now to our upcoming milestones. We are extremely pleased stones we expect to achieve. For our fatal study in DLBCL, we expect to report top line data by the end of 2018, which would support regulatory filings in first half of twenty nineteen. For the pivotal Phase III BOSTON study, we expect to complete enrollment by the end of this year with top line data expected in we plan to submit an MAA to the EMA for conditional approval of selinexor based on the data from the STORM study.
For the Stomp combination study in myeloma, we will continue to provide data updates at appropriate medical meetings. And for our solid tumor development programs, the ongoing phase 3 seal and C ENDO studies continue to advance in patients with liposarcoma and endometrial cancer, respectively. In closing, I would just like to take a moment to reflect upon what has truly been a remarkable time for us at Kario Farm. Completion of our first NDA submission is a landmark event for the company and bring selinexor 1 step closer to the many patients battling myeloma today. Should the FDA grant our request for accelerated approval, selinexor could be available to patients in the first half of twenty nineteen.
On behalf of our entire management team and Board of Directors, I want to express our sincere gratitude to the entire Karyopharm team for advancing the selinexor NDA so quickly. In just over 3 months following reporting the top line storm data, The team was able to successfully and effectively prepare and submit our first NDA, and I cannot be any prouder of the professionalism and dedication I've witnessed over the past few months. We are committed to working closely with the FDA during this review process, and we are looking forward to the exciting prospect of potentially introducing The first ever FDA approved Exportin-one inhibitor to help these patients who have exhausted all other available therapies of likely clinical benefit. I will now turn the
Our first question comes from Brian Abrahams from RBC Capital. Your line is open.
Hi, all. Thanks for taking our question. This is actually Rick on the line for Brian. Congrats on the NDA submission and all the regulatory progress. First, my question is, could you describe some of the work that is being done around the submission of the marketing authorization application to the EMA.
And if you anticipate any potential rate limiting steps for getting this submitted by early 2019?
Sure. This is Michael. As you're probably aware, the common technical document, which forms the basis of both the NDA and the MAA are quite similar, hence the name common technical document. We're moving forward in converting the various appropriate sections of the Ctd into the MAA format and we'll be moving that as expeditiously as possible with our plan filing in the first half of next year.
Great. And as far as the potential for conditional approval in your goes, in your view, is the EMA aligned with the FDA in terms of the unmet need in the pemter refractory population and what the expected bar for efficacy would be for these patients?
In general, FDA has allowed a couple more accelerated approvals, but in all cases of novel mechanisms, namely with Velcade and with DARZALEX more recently, the EMA has been open to that and of course, we're discussing this with EMA and they are certainly open to discussions around this novel mechanism of action in a population of patients that exhausted all EMA approves therapies as well.
All right. And just one more Europe question for me and then I'll hop back in the queue. Can we get the latest updates for potential commercial partners in Europe, with the potential submission in early 2019 and the potential for approval in the back half of twenty nineteen, what are timelines for establishing a partnership and how much lead time do you think a potential partner would need to support a launch in the back half of 2019?
Yes, I'll turn that over to Chris Permianna.
Sure. Hi. This is Chris Permano, the Chief Business Officer here at TerraForm. So it's a great question and I also want to just echo what Michael said at the end of our prepared remarks, which is that the company really undertook an amazing feat to file this or to submit the NDA so quickly. And all of the folks here at Keriopharm had patients in the forefront of their minds as the driving force to, to accomplish that so quickly.
And patients are one of our stakeholders and as our investors and caregivers positioned, patient advocacy groups, regulatory authorities and the like. And so when it comes to European partnering and a commercial launch launch in Europe or anywhere ex U. S, we're going to do the right thing by all of these stakeholders and do the right thing strategically the company and for patients. So I'm a little bit reluctant to attach any particular timelines to a commercial partner in Europe. We're constantly evaluating the regulatory and reimbursement landscape in Europe and otherwise ex U.
S. And there are always opportunities to do things that actually have an even better strategic fit Cario farm. So I think it would be, a little bit, unnecessary to probably provide specific timeline, but I would say to the other part of your question around how long does a partner need to get sort of prepared for a launch. I would think that 6 months is probably a reasonable amount of time. Obviously, the longer the better, but but the trick in Europe, as you know, is not so much around regulatory approval, but around, understanding and navigating your way through the reimbursement landscape.
And so that's where a lot of the timelines will lie. So I think takeaway here is that this is obviously an important topic for us and one that we're subject to ongoing evaluation here. I wouldn't put specific timelines on a European partner or any other strategic decisions we'd make ex U. S. Around commercialization, but just know that we will do the right thing by all of these various stakeholders with respect to any commercial launch.
And our next question comes from Arlinda Lee from Canaccord. Your line is open.
Hi, guys. Thanks for taking my questions. And I'd like to add my congratulations on completing your first NDA filing. I wanted to maybe follow-up on the EMA filing. And I'm wondering that if it's a common technical document, if it takes guess 6 months ish to, convert that?
Or are you potentially waiting for the sales data to kind of incorporate that into a filing or and or a European partner? Thanks.
Hi, it's
Michael. Yes, I think we're trying to be very careful about what we submit. Obviously, first impressions are lasting. And remember that it's not in Europe, it's not just across of submitting the documentation. There's a set of meetings that occur with various country experts in these diseases.
So we have to carry out all of those meetings and then there's touring co rapid care selection, which is done through the EMA process. So, we feel it's a very safe bet that this filing will be done in the beginning of next year.
Okay, great. And then, as I follow-up on the EU filings for SADAL, you mentioned that the U. S. Filing would be in the beginning of next year. Or next year, is that timeline going to be the same for the European filing?
And then maybe you can talk about, what specific commercialization efforts you guys are undertaking in the
we're not talking about the MAA filing for SADO quite yet. It's a little premature given everything else we have on our plate, but rest assured that as we move this Storm NDA expeditiously. We will be working hard to get the proper documentation together for the SADAL NDA filing as well as for Europe as well. The commercialization in general is basically good because the target audiences are quite a bit overlapping between lymphoma doctors and myeloma. Obviously, at academic institution, these are generally separate departments, but all of the institutions have both departments.
And for the more community positions and the large group practices, these will be largely same position. So we won't have to increase our marketing efforts too much as we move into lymphoma.
Thank you. And our next question comes from Eric Joseph from JP Morgan. Your line is open.
Congrats on the progress and let me also extend my welcome to Ian. Thanks for taking the questions. I guess the just a couple from us. The first on the NDA submission, I'm wondering if you requested or if it's reasonable to fact a review along priority timelines? And then as it relates to SADAL, I'm not sure if I missed
it, but can you give us
a sense of just where you are in terms of enrollment on the way to the target 130 patients and how you might be thinking about median duration of a follow-up when the data readout later this year? Thanks.
Yes. Hi. On the question of, on the priority review, part of Fast Track Designation, rolling submission prior to review and accelerated approval are all components of that, but to be Clear Fast Track allows you to request those. The accelerated approvals, I'm not aware of any accelerated approval that goes in without a Fast Track request. So in general, one would expect that.
And as we receive the information back from FDA, we'll update you on the status of that. Can you repeat the second part of the question?
Worst, sorry, where part 2 stable enrollment stands on the way to completion, how to think about medium follow-up at the time of data read read out year end?
Yes, we feel comfortable that by year end, we'll have strong top line data. We regards to the key endpoints of response rate and duration of response that will be sufficient to provide a good amount of clarity on how that filing would go.
Great. Thanks for taking the questions.
Thank you. And our next question comes from Maury Raycroft from Jefferies. Your line is open.
Hi, good morning and congrats on the update and, milestone NDA filing. First for for the U. S. Sales force for multiple myeloma, you mentioned 1H19, you'll start hiring. Is that enough time?
And are you doing any work before that to educate physicians people are launching?
Yes, let me turn that answer over to Chris or Mike.
Sure. Sure. So, as we noted, with the successful completion of submission this week on track for the first half launch in multiple myeloma in the U. S. And we plan to commercialize that by ourselves.
We've noted that we'll be bringing on a sales course in early 2019. And yes, there's a whole host of activities that will take place before before we bring the sales force on board. Michael noted that we're pleased to bring on board an environment to lead our commercial effort joins a team of very experienced vice presidents that we hired earlier in the year. And they have our e started work on our marketing efforts, which includes branding, preparing the launch materials. And then I think also just beginning a general education campaign, which will gather for us, when we're able to share the full results from the STORM study.
So there's a number of commercial activities, educational in nature that take place prior to the launch and we've begun to put in place the plans to execute that. And I think you'll start to see those activities begin in a little bit later this year, Q4 and then into Q1, Q2 prior to launch.
Got it. Very helpful. And what are next steps for the thrombo studies and any perspective or update on enrollment in the newly diagnosed setting revenue.
Hi. We'll just continue to update as appropriate when we have an data to present it in abstract and hopefully, presentation format. As times go on, we're moving along expeditiously. People are excited about this. And I just remind everybody that the STOMP study uses once weekly selinexor with all the different combinations.
And we've seen additive or synergistic activity across the board so far. So we're quite excited about it and we'll just continue to update as we have recently.
And our next question comes from Jonathan Chang from Leerink Partners. Your line is open.
Hi. This is David Rusch. I'm dialing in for Jonathan. Thank you for taking my questions and congratulations again on the submission. Two questions here.
Following the positive data from the steel study, how should investors be thinking about the opportunity in liposarcoma? And could you provide any guidance on additional plans in that population? And then also with the ongoing progress of the Boston study, with Velcade. Is there any context that you can provide on the other combination data that was presented at EHA moving forward Thank you.
Sure. For liposarcoma, we're in the midst of the Phase III study and we intend to provide data by the end of next year. That's a randomized Phase III study, 2 to 1 randomization and moving along. In terms of the as I mentioned on the other combinations, we'll just continue to update things as we move forward. One of our good problems to have is that selinexor has activity across a large variety of cancers and choosing whether we continue to expand deeply into myeloma, which we will do at some point or we also bring selinexor to other cancers including liposarcoma.
And as we mentioned earlier in endometrial cancer, we'll have to make some choices as we go forward. But the broad activity of selinex really allows us to explore it across a number of different tumors. And we're just going to have to make some decisions on market sizes and unmet medical need across the board.
And our next question comes from Constantino Eprilakis from JMP Securities. Your line is open.
Hey guys, good morning. Thanks for taking my questions. And let me add my congrats on the progress. So assuming the SRT combination looks good in frontline multiple myeloma, do you know what kind of trial you need to run to get approval in that setting? And the response rate you're seeing in future reader patients is already pretty high, especially in LEN naive patients.
Do you need to see additional CRs cell and export to bolster confidence?
These are great questions and really you have to look at the tow of the data as the FDA likes to talk about. The main issue in frontline myeloma is tolerability and long term disease control. Deep responses are great, but as we all know, deep responses that last a short time are probably less important than prolong disease control. So part of it will have to be to mature some of the data and of course, the big advantage of Srd cell rev decks in frontline is it's an oral regimen and it utilizes once weekly once weekly Selinexor in combination with Revlimid. So as the data become available, we'll be able to update.
And then lastly, there are some operations going on with FDA amongst a lot of people to use surrogate endpoints in the frontline setting because of the very prolonged PFS that now and perhaps response rate or deep responses would be looked at, but there may be other ways to look at this. So we'll have to decide as we move forward. Okay.
And our next question comes from Ying Huang from Bank of America. Your line is open.
Hey guys, Jenny on for Ying. Congrats on all the progress in filing your NDA. Just a question for us in terms of the NDA, what was included in the safety package? Was it just the Phase 2b storm or did you have
a lot of the other trials also in
there for the safety package? Thank you so much.
Typically, in an NDA, the is being discussed with the FDA and we are following the FDA guidance and what is included in the NDA and including most of the data in dermatological cancer and for the safety information.
Thank you. And I am showing no further questions from the phone line. I would now like to turn the call back over to Michael Kaufman for any closing remarks.
Hi, everyone. At Karyopharm, our mission since inception has been to advance the treatment of cancer through discovery and development of novel oral therapies, beginning with selinexor. We are extraordinarily proud to be developing this 1st in class oral nuclear export inhibitor with the potential for activity across a number of hematologic and solid tumor malignancies. We are working with passion and urgency to foster innovation and lay the important groundwork for our future growth and commercial success. I thank everyone for your time today and we look forward to keeping you updated on our continued progress.
Have a good day.
Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program. You may all disconnect. Everyone have a wonderful day.