Good morning. My name is Ayala, and I will be your conference operator today. At this time, I would like to welcome everyone to the Keriopharm Therapeutics First Quarter Twenty Eight Financial Results Conference Call. There will be a 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. Christopher Primiano, Chief Business Officer of Keriopharm Therapeutics.
Thank you, Ayala, and thank you all for joining us on today's conference call to discuss Karyopharm's first quarter 2018 financial results. This is Chris Permiano, and I'm joined today by Doctor. Michael Kaufman, Chief Executive Officer Mr. Michael Falvey, Chief Financial Officer Doctor. Sharon Schacko, our Founder, President and Chief Scientific Officer and Doctor.
Jatin Shah, Senior Vice President, Clinical Development. On the call today, Michael Kaufman will make some introductory comments that Mike Balby will provide an overview of the first quarter of 20 18 financial results. Doctor. Kaufman will then discuss our key upcoming milestones and provide some summary remarks. We'll then open the call up for questions for which Sharon, Jayden and I will also be available.
Earlier this morning, we issued press release detailing Keriopharma's results for the first quarter of 2018. This release is available on our website at karyopharm.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 under the Private Securities Litigation Reform Act of 1995. These include statements about our future expectations, clinical developments and 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 including those discussed in the Risk Factors section of our annual report on Form 10 K for the year ended December 31, 2017, which was filed with the SEC on March 15, 2018, and any other filings we may make with the SEC, including our quarterly report on Form 10 Q for the quarter ended March 31, 20 18, which we expect to file later today.
Any forward looking statements represent our views as of today only and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change, therefore, not rely on these forward looking statements as representing our views as of any date subsequent to today. In addition, please note that any references we make to clinical trial data during today's discussion referred to interim unaudited site data unless otherwise specified. I'll now turn the call over to Doctor. Michael Kauffman, Chief Executive Officer of Karyopharm.
Thank you, Chris, and good morning, everyone. Thank you for joining us on today's call. Last week, we reported exciting top line results from part 2 of the Phase 2b STORM study evaluating selinexor in 122 patients with heavily pretreated pantry refractory myeloma. For the STORM study's primary objective, oral selinexor achieved a 25.4 percent overall response rate assessed by the independent review committee. In 29 partial are very good partial responses.
1 of the stringent complete responses was negative for minimal residual disease or MRD, which is particularly significant in this penta refractory population. The median duration of response, a key secondary objective was 4.4 months. Responding patients had a significantly prolonged overall survival as compared with non responders. To our knowledge, This was the 1st large study in patients with enter refractory myeloma and these results are an important advance for myeloma patients their families and for their physicians and caregivers
who provide
treatment for this difficult disease. Oral selinexor demonstrated a predictable and manageable safe tolerability profile, consistent with that previously reported from Part 1 of the STORM study and from other selinexor studies. No new safety signals were identified. When occurring, adverse effects were often reversible, transient and manageable with dose modification and or standard supportive care. We look forward to submitting a detailed storm study results for presentation at an upcoming medical oncology meeting.
As you may know, selinexor was recently granted Fast Track designation by the FDA for the panel refractory population evaluated in the STORM study. We believe this is an acknowledgment from the FDA that the patient population in STORM represents a true unmet medical need where new therapies remain critical. As a reminder, patients on storm with pembrol refractory myeloma have disease that progressed after receiving some of the best anti cancer agents available in all of oncology. Including Revlimid, Kamalist, Kyprolis, Velcade, Alkylating agents as well as DARZALEX. Moreover, we required that all patients entering this study have myeloma that is to their last immunomodulatory drug, their last proteasome inhibitor and SEDARZALEX as well as progressing on their most recent therapy indicating that their disease is unlikely to benefit from retreatment with these classes of anti myeloma drugs.
Looking ahead, we plan to submit a new drug application to the FDA during the second half of twenty eighteen with a request for accelerated approval for oral seladexers and new treatments for patients with Phenorefactory multiple myeloma. We also plan to submit a marketing authorization application to the European Medicines Agency in early 2019 with a request for conditional approval for selinexor in the same indication. On the commercial front, we are actively designing and building our infrastructure and preparing our first potential selinexor product launch United States. In parallel, we are exploring strategic commercial collaborations with potential partners in Europe and other key markets. Importantly, the activity in the STORM patient population further support the ongoing pivotal randomized Phase III BOSTON study where selinexor is being evaluated in earlier lines of therapy.
Boston evaluates the oral seldex regimen in combination with once weekly Velcade. Compared to standard twice weekly Beldex alone in patients with myeloma who have had 1 to 3 prior lines of therapy. 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 the BOSTON study will support a full approval for Celldex in combination with Velcade as a second line treatment for myeloma. In addition, the storm data supports variety of selinexor combinations being evaluated in the ongoing Stump study in patients with relapsed or newly diagnosed myeloma.
I also want to mention that we are on track to report top line results and another important unmet medical need population by the end of this year. Patients with relapsedrefractory diffuse large B cell lymphoma who are currently being evaluated in our ongoing SADAL study. If the final results of the SADAL study are consistent with the interim data presented last year at the European Hematology Association annual meeting, we plan to file a request for accelerated approval in the first half of twenty nineteen for this indication. Secured approximately $155,000,000 in gross proceeds to carry a farm. We are grateful for the continued support of our existing holders would also like to extend a sincere thank you and welcome to our newest shareholders.
With that, I'll turn the call over to Mike.
Thank you, Michael. Since we issued a press release earlier today outlining our full financial results, I'll just review our first quarter 2018 financial highlights. As of March 31, 2018, cash, cash equivalents and investments, including restricted cash, totaled $141,500,000, compared to $176,400,000 as of December 31, 2017. As Michael mentioned, on May 7, 2018, Carrier Farm completed an underwritten public offering of just over 10,500,000 shares of its common stock at a price to the public of $14.75 per share. The gross proceeds to carry a farm from the offering were $155,300,000 and after deducting the underwriting discounts to commissions and other estimated offering expenses, our net proceeds were $145,600,000.
For the quarter ended March 31, 2018, Karyopharm recognized $10,000,000 in revenue compared to $100,000 for the 3 months ended March 31, 2017. The increase in revenue was the result of the upfront payment received from the asset sale of KTT-three fifty to Biogen in January of 2018. For the first quarter 2018, research and development expense was $41,300,000 compared to $24,100,000 for the same period in 2017. General and administrative expenses for the first quarter of 2018 was $7,600,000 compared to $6,300,000 for the same period in 2017. Comparing the first quarter of 2018 to the prior quarter, the fourth quarter of 2017, R and D expense increased by $6,500,000 reflecting increased spending on our late stage clinical trials.
For the first quarter of 2018, we reported a net loss of $38,500,000 or $0.78 per share compared to a net loss of $29,900,000 or $0.71 per share for the first quarter of 2017. Net loss includes stock based compensation expense of $4,200,000 $5,900,000 for the first quarters of 2018 2017, respectively. Carrier Farm expects its operating cash burn including research and development and general and administrative expenses for the year ended December 31, 2018 to be in the range $175,000,000 to $185,000,000. Based on our current operating plans, we expect that our existing cash cash equivalents and investments will be sufficient to fund our operations into the third quarter of 2019. And importantly, this runway takes a through the planned launch of selinexor in the first half of next year.
These plans include the continued clinical development of selinexor in our lead indications, submitting an potential launch of selinexor in the United States. I'll now turn the call back over to Michael Kaufman for concluding remarks. Michael?
Teams recently achieved, and we continue to believe that 2018 will be a transformational year for Karyopharm. We have several upcoming milestones that I'd like to highlight. During the second half of the year, we plan to submit an NDA for oral selinexor as a new treatment for patients with pantry refractory myeloma. Following that, we plan to submit to the EMA for conditional approval in the same indication. For our SADAL study in DLBCL, we expect report top line data by the end of this year, which could support a regulatory filing in 2019.
For the pivotal Phase III BOSTON study, we expect to complete enrollment this year with top line data expected in 2019 and regulatory filings in 2020. For the STOMP study, common in myeloma, we will continue to update at appropriate medical meetings and we look forward to initiating a new arm of stop evaluating the all oral regimen of selinexor plus rev Dex in patients with newly diagnosed myeloma. Beyond hematologic malignancies, the ongoing seal and C ENDO Phase III trials in liposarcoma and endometrial cancer, respectively, continued to advance. In closing, I'd just like to reiterate on behalf of the entire Karyopharm team that we are extremely enthusiastic about the data reported last week from the STORM study. We believe these data are great news for all of our stakeholders, including the patients battling myeloma, their families, physicians and caregivers, the foundations that have supported and believed in us from the beginning as well as the many new investors that recently purchased shares.
We have evolved from an entrepreneur and an idea and to a well integrated team of close to two hundred people, all working passionately to prepare the regulatory submissions for selinexor in our first potential indication. And to refractory multiple myeloma. At Karyopharm, our mission since inception has been to advance the treatment of cancer through the discovery and development of novel oral therapy beginning with selinexor. We are very proud to be developing the 1st in class nuclear export inhibitor with the potential for activity across a number of hematologic solid tumor ligence. As we look ahead, we will continue to foster innovation, courage, urgency, resilience and energy, our IQOR culture, we are actively working to recruit and hire talented professionals to embody our ideals and lay the important groundwork for our future growth and commercial success.
I will now turn
Our first question is from Brian Abrahams with RBC Capital Markets. Your line is now open.
Hi, good morning. This is Rick on for Brian who's on a flight at the moment. Thanks for taking our questions. First off, could you please walk us through what preparations for commercialization are currently underway and maybe speak to what you hope to accomplish in the coming months?
Sure. So we announced on our last earnings call that we had hired 3 very seasoned VPs into our commercial organization, VP of sales, VP of marketing and VP of market access. What you'll see over the course of the rest of the year is that those three leaders together with Bill Hathfield, our Head of Commercial, will be building out and blocking out our launch plans and starting to fill in the senior the rest of the senior positions in our commercial organization. And then also begin to do the work towards preparing for the launch. So this includes preparing our marketing materials for the launch, which, as you know, need to be submitted with our NDA.
We'll start to build out our distribution channels. We'll hire agency to help with our marketing campaigns. And then as we get into the fourth quarter, you'll see us taking steps like purchasing data to understand the market's deeper, building our CRM systems and really laying all of the foundation that will lead to do a full fledged launch in the United States. But the one
thing that we'll be missing
to support that launch would be the hiring of the sales force. Which we plan to pull the trigger on immediate or probably about 3 months before the launch of selinexor in the middle of next year.
Great. That's very helpful. Next, I wanted to just maybe get your most recent thoughts on partnering and Have you thought about at all since you received the positive storm data last week?
Yes. Hey, this is Chris. So, give you a couple of the highlights on how we're thinking about partnering. So I think it would be somewhat arbitrary to give a timing expectation around partnering, but I think it's probably more instructive to think about, what does that collaboration look like and what kind of a partner are we looking for? So as you know, we recently, at the towards the end of last year, established the collaboration with Ono Pharmaceuticals or some of the Asian territories.
And I think that's probably a good, guideline for how you might see a collaboration come together for some of the ex U. S. Ex ono territories. And you could expect to see something of a multiple of what you see in that particular arrangement in terms of the upfront the milestones, the cost sharing around clinical development expenses and those types of things. But I think even more important than that is the kind of partner that we'd be looking for.
And obviously one of the key territories that we're discussing around an ex U. S. Ex owner territory. Partner is experiencing in hemonc and solid tumors broadly In particular, with respect to the reimbursement landscape and the commercial landscape across Europe, which is an increasingly complicated area in which launch a new product. And in addition to that, we're looking for someone who's really philosophically aligned with us in the way that we view selonexor and the long term development plan for selinexor.
This is not as we've talked about many times simply a drug in myeloma and lymphoma that actually has a much longer and broader development plan behind it. And we'd want someone who's philosophically aligned with us on that perspective as well. So that's how we're thinking about partnering.
All right. Got it. And one more question, if I can. How long do you expect the overall survival data and the flexibility to take some sure. Could we expect these data to be included into maybe a rolling NDA submission?
And will we see these data at a medical later this year? Thank you. I'll hop back in the queue.
It's a little hard to tell when the data will mature. But part 1 is already published as you're aware and importantly show that the response rate, which we saw there, which is a little bit lower, was associated with an improved survival of compared to the non responders, which is a really key point as you evaluate the overall activity of the drug. We mentioned on our call as well as today that in part 2, we're seeing the same kind of actually statistically significant differences that the patients who respond with any response to selinexor have as clinically and statistically significant improved survival over the patients who do not respond indicating that response is associated with longer survival and also that there really isn't a good rest therapy available for those who do not respond. We are given the fast track designation, we are in ongoing discussions with FDA about planning our submission. And we are strongly considering a fast track roll use of the fast track rolling submission guidelines in order to facilitate the review of Selinexion.
We'll keep you posted.
Our next question is from Maury Raycroft with Jefferies. Your line is now open.
To start, for ASCO, it seems like you're going to have Phase II, the Phase II liposarcoma seal data there. And I was wondering what we should expect as far as the details go. And if there's any possibility for the HR to change or should we expect that that will hold up in the update?
So the data is to ask about phase 2, as we have seen in India. The abstract of the current results and that includes the vast majority of the patients, reaching their event of PFS. So no significant changes are expected based on this. And as you know, we have moved into the Phase III and the Phase III is enrolling. So, and this was based on the results of the Phase II, as you can see them, in the abstracts.
Got it. And, well, can you comment on whether that will be a presentation or poster?
It's in the
public domain. I don't think we can comment. No, it's on the public domain.
And then, next, I was just based on the recent approval of DARZALEX and frontline. I'm just wondering if you can provide thoughts or perspectives on how this could influence the current treatment paradigm And if you have any expectations to fold a newly diagnosed cell plus dara combo arm into the STOMP study?
Yeah, I will take it and then I'll turn it to Jason. So we're thrilled to see DARZALEX moving up to frontline We also thought this would happen. We also know that the DARZALEX REF Dex combination is being evaluated in front line and we anticipate that will also be successful. And it was part of the strategy here with the Boston study that we're doing that frontline would consist of a direct red along with Revlimid, and that we would come in as a Velcade based regimen, Selveldex is one of the potentially the most convenient regimen in this is successful with once a week dosing, a low rate of neuropathy of the Phase III data hold up. And really a nice regimen for patients who unfortunately after they progress from frontline need a second line therapy.
And I'll just let I'll turn it over to Jatin a minute. But the other point about Dara moving in front line is that it really opens up the tenter refractory population. This means that patients with 2 or 3 lines of therapy, could very well meet criteria for Petrobrasactory disease as they get Dara in earlier and earlier lines In fact, people are even using Dara with Revlimid and Velcade in frontline. So you can imagine that second line could be Carfil palm regimen in that third line could be a pentor refractory situation. So we think that this is going to continue to push the pentor refractory population earlier and open up the number of patients with enter refractory disease.
I'll turn it to Jake and to add and then talk about the frontline strategy on side. So thanks, Michael. So I
fully agree. I think that that's a good thing for patients, giving access to DARZALEX earlier, but really positions us well as patients gets dollars less in combination either of the BNP with the approved, indication now or in the future with Lendal and Mite Index. I think it positions the combination in from the BOSTON study well and that, second line therapy, as well as for the current potential indication of Pantor refractory setting. So I think that's, we're well positioned with DARZLux moving
on the frontline setting.
I think regarding your second question, regarding, newly diagnosed myeloma. So in our current stop study, will be looking at selinexor in newly diagnosed myeloma in combination with lenalidomide and to understand that there's still an appetite for an all oral combination in newly diagnosed myeloma. We'll be exploring that combination in the current stop study.
Our next question is from Jonathan Chang with Leerink Partners. Your line is now open.
Maybe first just for the fatal study. Can you talk about how you do the BAR for approval in a 3rd line plus DLBCL?
So can you repeat that?
Yes. So the bar for
potential approval in SADAL. Please remember that there are still currently no drugs that are actually approved for the treatment of DLBCL since rituximab was approved for frontline combination. With CHOP back in 2006. So there's just other than CAR T, which is approved as a cell therapy, for very specific patients who are transplant eligible. There's nothing available now for patients in 3rd line.
There's no approved second line. There's pretty standard of care. For transplant eligible and ineligible patients. We believe the bar, is a response rate in the 2025% to 25% or better. And that the duration of response again should be about 4 to 5 months, much the way it is in last line myeloma.
And we think that the FDA is and physicians and patients are interested in drugs that are also somewhat agnostic to the subtype. So we've made sure that our SADAL study includes both GCB and ABC types of DLBCL. And the interim data, I'll remind you that we presented last year at EHA showed a response rate of 33% and a duration of response of greater than 7 months. We think those easily meet the bar. And we think this could provide a really convenient oral option for patients who have refractory DLBCL and really no approved therapies.
Thanks. And just one more question. How do you see CAR T impacting the DLBCL landscape and the positioning of HelenX are in this questions? Thank you.
Great question. So this is Jatin. So regarding CAR T cell, so as Michael mentioned, CAR T cells is really limited to a small subset of patients who are transplant eligible. We know the those trials are highly selective and as we move into the commercial space, it approved now for the last since fall of 2017. It's really Luna for those patients who are transplant eligible, and younger patients is also at the major academic medical centers that can support a CAR T cell, which is limited.
So it's really limited to these special centers and not really applicable for 2 things. 1, for older patients, which vast majority of large cell lymphoma. And number 2, the vast majority of patients out in the community, even if they are younger patients, So I think that's where selinexor is well positioned in oral therapy that can be given widely.
Our next question is from Eric Joseph with JP Morgan.
Hey guys, congrats on all the progress and thanks for taking the question. Just looking for, I'm framing expectation for EHA, whether you can provide us with an update around hatchback conditions, specifically the lead
rig here for storm.
As well as any follow-up presentations related to the strong combination of
Sure. We've assembled the data where we're hopeful that we can have a late breaker at EHA and it'll really depend on what else is what's there. So if we don't make it there, we'll certainly make it at the next meeting. We're excited about the data. You've heard the top line the details will come out when they can.
Okay. So I mean, I guess with abstracts being released next week, we shouldn't expect, abstracts from the soft combinations.
That's right. Yes. So there are several stop abstracts that were submitted to EHA. So we'll hear about those soon.
Our next question is from Mike King with JMP Securities. Your line is now open.
Hi, good morning guys. Thanks for taking my question and I'm asking on behalf of couple of friends of mine. I just wanted to maybe follow-up on the Revdexcel frontline indication. I'm just wondering if, Michael, do you think about it or Jatin, do you think about this as far as transplant eligible versus ineligible or is that definition not as clear cut anymore? Because I'm thinking that maybe you could still go frontline with cell rev decks, but without DARZALEX just because there may be a population that does not want to or can't tolerate the IV infusion of DARZALEX.
Yes. So to be clear, so right now, we're looking at the combination of selinexor route Dex in newly diagnosed myeloma. And it's a bit, I think it's quite controversial when you look across, globally in terms of when folks are using transplant. And so you can clearly go in the transplant ineligible patient population or even the delayed transplant. So those questions, those patients who are still transplant ineligible.
Some centers still widely delayed approach as opposed to, induction therapy. So I think that this can be both situations, it doesn't have to be limited to the ineligible because there's still additions for transplant eligible.
You can
get it, getting their cells collected into a delayed transplant. So I think that that line is a blurring a bit now as we think about thediagnosis problem and how centers approach it.
Okay. And then just on enrollment in both Boston and SADAL, have you guys achieved full enrollment in SADAL yet?
Always said, Mike, is that we'll have top line data by the end of the year. We're comfortable with that.
Our next question is from Ying Huang with Bank of America. Your line is now open. Hey guys, it's Jenny. Thank you for taking your questions.
Just in terms of your commercial preparations, have you guys thought about the academic versus community settings and how you would differently approach each of those? And also beyond SVD. So the other stop indications, would you bring any into a pivotal trial or just use stomp for like NOA after you're approved in Henterofactory? Thank you.
Yes. Thanks. So I'll I'll start. For the approach in the academic versus the community setting, one of the great unique things about selling answer is. It clearly has applicability in both settings.
Obviously, the majority is the vast majority of the storm patients came from the academic setting they were treated in academic centers. And the fact that these centers are seeing this this level of activity in patients who've exhausted available therapies really sets us up nicely. But on the other hand, selinexor is a very convenient twice a week oral therapy. Which makes it really uniquely suited for treatment in the community as well. So we're very much looking forward to a 2 pronged approach where we focus on selinexor's activity in the heavily, heavily pretreated patients with no real options in the academic center and for the ease of use situation in patients with myeloma out in the community who are perhaps less heavily pretreated, but still meet the criteria for penter refractory disease.
Think it's a great and a unique opportunity, in this setting. On the, question about whether we move any of our other SOP combinations, those data will continue to mature. We're certainly excited about a lot of those combinations, the DARZALEX combo, the POMALIST combo, in particular, will be studying Kyprolis as well coming up. Obviously, Velcade has already started and the Revlimid combo is moving into frontline. So lots of good stuff happening.
We will pick likely pick 1 or more of these to move towards a phase 3. We may do that with a consortium. And we'll have to think about it the data merged, but we really have a unique situation there where essentially every combination has shown some level of additivity or Frank's synergy going forward.
Got it. Great. Thanks guys. Michael Kaufman, CEO, for any further remarks.
Just take one more time and thank everybody for your great questions. And we look forward to speaking with many of you in the coming weeks and to keeping you updated on our continued progress. Thank you.
Ladies and gentlemen, thank you for participating in today's conference.