Karyopharm Therapeutics (NASDAQ: KTPI) Q1 2019 Earnings Call May 9, 2019, 8:30 a.m. ET

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Prepared RemarksQuestions and AnswersCall ParticipantsPrepared Remarks:

Operator

Good morning. My name is Gigi and I will be your conference operator. At this time, I would like to welcome everyone to the Karyopharm Therapeutics first quarter 2019 financial results conference call. There will be a question and answer session to follow. Please be advised this call is being recorded at the company's request.

I would now like to turn the call over to Ian Karp, Karyopharm's Vice President of Investor and Public Relations.

Ian Karp — Vice President of Investor Relations and Public Relations

Thank you, Gigi and thank you all for joining us on today's conference call to discuss Karyopharm's first quarter 2019 financial results and business updates. This is Ian Karp and I'm joined today by Dr. Michael Kauffman, our Chief Executive Officer, Mr. Mike Mason, Chief Financial Officer, Mr. Christopher Primiano, Chief Business Officer and General Counsel, and Mr. Anand Varadan, our Chief Commercial Officer.

On the call today, Michael Kauffman will provide an overview of some of our recent corporate developments, then Mike Mason will provide an overview of the first quarter 2019 financial results. Dr. Kauffman will discuss our key upcoming milestones and provide some summary remarks. We will then open the call for your questions.

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Earlier this morning, we issued a press release detailing Karyopharm's results for the first quarter of 2019. The release is available on our website at Karyopharm.com. Before we make our formal comments, I'll remind you that various remarks we will make today constitute forward-looking statements.

For purposes of the safe harbor provision under the Private Securities Litigation Reform Act of 1995. These include statements about our future expectations, clinical development, 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 factors, including those discussed in the risk factors section of our most recent annual report in form 10-K, which is on file with the SEC, and in other filings that we may make with the SEC in the future. Any forward-looking statements represent our views of today only.

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, you should not rely on these forward-looking statements as representing our views on any date subsequent to 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 Dr. Michael Kauffman, Chief Executive Officer.

Michael Kauffman — Chief Executive Officer

Thank you, Ian. Good morning, everyone. Thank you for joining us on today's call. I will begin with a quick overview of recent company events and developments. First, in mid-March, the FDA extended the PDUFA action data for the Selinexor new drug application by three months to July 6th, 2019.

This extension followed the FDA Oncologic Drug Advisory Committee meeting that was convened on February 26th to review data supporting our NDA requesting accelerated approval for oral Selinexor in combination with low-dose dexamethasone for patients based on the results of the single-arm Phase 2b STORM study.

At the conclusion of the ODAC meeting in a vote of 8 to 5, the committee recommended that the FDA wait for the results from Karyopharm's randomized open-label Phase 3 BOSTON study evaluating Selinexor in patients with previously treated myeloma before making a final decision regarding approval.

After the conclusion of the ODAC meeting, Karyopharm continued to have productive discussions with the FDA regarding the Selinexor new drug application. At the request of the FDA, Karyopharm submitted additional existing clinical information as an amendment to the NDA. Following a review of these additional data, the FDA extended the PDUFA action date for three months.

Since then, we have been working closely with FDA as they complete their review of the Selinexor new drug application. We remain deeply committed to bringing Selinexor into the hands of physicians treating patients with refractory multiple myeloma.

While the FDA will consider the recommendations of the advisory committee, the final decision regarding approval of the product is made solely by the agency. The recommendations of the advisory committee are non-binding. As a reminder, the BOSTON study, which was fully enrolled this past January, is evaluating Selinexor in combination with Velcade, also known as Bortezomib and dexamethasone, indicating dexamethasone alone in patients with myeloma who have had one to three prior lines of therapy. As progression-free survival is the primary endpoint, we expect to have topline results from that study by the end of 2019 or into 2020 depending on the recurrence of the progression.

At the recent meeting of the Data Safety Monitoring Board, no new safety signals were identified on the study and based on the first interim analysis, the board recommended proceeding with the study as originally planned with no changes in patient numbers.

As we now await the conclusion of the FDA's review, we have also been very active in preparing for the potential commercial arm to Selinexor in the United States. In January, we hired a commercial salesforce of approximately 60 sales representatives and eight nurse liaisons. Should Selinexor receive FDA approval by the updated July 6th PDUFA date, our commercial team will be an excellent position to support a successful United States launch.

Parallel to the US activities, we announced in early January that we had submitted a marketing authorization application to the European Medicines Agency requesting conditional approval for Selinexor in the same purple class refractory myeloma application in the United States. As a customary part of the marketing application review process, we received a consolidated list of questions from the EMA in early May 2019.

To provide adequate time evaluate the application and allow us to respond to the questions and feedback, the AMA has switched the application from an accelerated review to a traditional review. We expect to receive a decision on the application by the end of 2019.

Shifting gears now to our Selinexor development programs beyond myeloma. In diffuse large-B-cell lymphoma following a presentation of positive topline results from the Phase 2b SADAL study at ASH 2018 and the receipt of fast-track designation during the fourth quarter of 2018, we are now working toward a new drug application and marketing authorization application submissions requiring accelerating and conditional approvals requesting accelerated and conditional approvals, respectively, for patients with relapsed or refractory DLBCL who have received at least two prior multi-agent therapies and who are ineligible for stem cell transplantation, including CAR T therapies.

We are working with both the FDA and EMA to determine the appropriate timelines for these submissions and expect to have some greater clarity following the FDA's decision on our new drug application requesting accelerated approval for oral Selinexor in myeloma.

Next, I'd like to discuss some recent developments for our Selinexor program in endometrial cancer, where there are currently no approved therapies for treatment of patients in a maintenance setting following the initial chemotherapy.

During the first quarter, an investigational new drug application was submitted and accepted by the FDA for a randomized blinded Phase 2/3 study titled the SIENDO study evaluating Selinexor versus placebo as a maintenance therapy in patients with advanced or recurrent endometrial cancer following our initial platinum-based treatments.

The primary endpoint in the SIENDO study is progression-free survival. This study was previously an investigator-sponsored trial and has subsequently transitioned to a company-sponsored study. We are targeting complete enrollment in this study in 2020.

We've also seen progress across a number of additional clinical programs. Two Selinexor abstracts have been selected for presentation at the upcoming American Society of Clinical Oncology or ASCO meeting 2019 in late May and early June in Chicago. The first will be an early presentation describing results from the Phase 2b KING study evaluating single-agent Selinexor in patients with recurring glioblastoma. The second will be a poster presentation with discussion describing additional overall survival from the STORM study in patients with heavily pre-treated myeloma.

Finally, on the corporate front, we recently appointed Tina Clark Beamon as Chief Compliance Officer. Tina formerly served as Executive Director of Compliance and Ethics at Alexion Pharmaceuticals. She brings to the company 21 years of healthcare industry experience and her appointment reflects our ongoing commitment to maintaining the highest compliance and ethics standards as we transition into a commercial stage organization.

With that, I'll now turn the call over to Mike Mason for an overview of the first quarter financials. Mike?

Mike Mason — Chief Financial Officer

Thank you, Michael. Since we issued a press release earlier today with full financial results, I will just focus on the highlights. As of March 31, 2019, cash, cash equivalents, and investments, including restricted cash, totaled $265.1 million compared to $330.9 million as of December 31st, 2018.

For the first quarter of 2019, license and other revenue was $0.2 million compared to $10 million for the first quarter in 2018. The revenue in 2018 was from the $10 million upfront payment for the asset sale of KPT-350 to Biogen in the first quarter of 2018.

For the first quarter of 2019, research and development expense was $38 million compared to $41.3 million for the first quarter of 2018. We expect R&D expense to decrease going forward in 2019 compared to the first quarter of 2019 largely due to reduced spend from the BOSTON and SADAL trials.

General and administrative expense for the first quarter 2019 was $27.1 million compared to $7.6 million for the first quarter of 2018. The increase was due primarily to the hiring of the Karyopharm commercial team and related commercial launch preparation activities to support the potential US commercial launch of Selinexor. Assuming a commercial launch by July, we expect G&A expenses for the remainder of 2019 on a quarterly basis to be relatively consistent with Q1 2019.

For the first quarter of 2019, we reported a net loss of $66.2 million or $1.09 per share compared to a net loss of $38.5 million or $0.78 per share for the first quarter of 2018. Net loss includes stock-based compensation expense of $3.9 million and $4.2 million for the first quarters of 2019 and 2018, respectively.

Based on current operating plans, which assume a Selinexor commercial launch by July of 2019, we expect our full year operating expense to be between $200 million and $215 million excluding stock-based compensation. Additionally, we expect our existing cash, cash equivalents, and investments will be sufficient to fund operations into the second half of 2020.

If the FDA decides to delay its approval decision for Selinexor until the BOSTON data are available, we will reevaluate our spending expectations for 2019 and update the investment community at the appropriate time.

I'll now turn the call back over to Michael for concluding remarks.

Michael Kauffman — Chief Executive Officer

Thank you, Mike. Before we move to the Q&A, I'd like to provide an overview of our key upcoming milestones. For our first Selinexor NDA, we continue to work collaboratively with the FDA toward the July 6th, 2019 PDUFDA date and prepare for a potential commercial launch by mid-year. We are continuing to work closely with the EMA in support of our MAA requesting conditional approval for Selinexor in patients with triple-class refractory myeloma. We're expecting a decision by the end of 2019.

For our next potential indication in relapsed or refractory DLBCL, we plan to submit an NDA to the FDA with a request for accelerated approval and will be working with the FDA over the coming months to determine the most appropriate timing for submission. We also plan to submit an MAA with a request for conditional approval for oral Selinexor in this patient population.

For the pivotal Phase 3 BOSTON study, enrollment was completed in January and topline data are expected by the end of 2019 or the end of 2020 depending on the occurrence of progression events on the trial. If positive, these data could support regulatory submissions in 2020 in second line myeloma.

Next, the various arms of the Phase 1b/2 STOMP study continue in myeloma and we look forward to providing updates on the various combination arms at future medical meetings. Finally, we will continue to progress our solid tumor progress in liposarcoma and endometrial cancer.

In closing, I'd just like to say that we remain deeply committed to working with the patients, physicians, regulators, and the overall myeloma community with the goal of bringing Selinexor to the market as a new treatment option for patients who are in dire need of novel therapies to treat their highly refractory disease.

I will now turn the call over to the Operator for questions. Operator?

Questions and Answers:

Operator

Ladies and gentlemen, at this time, if you have a question, please press the * then the number 1 key on your touch-tone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the # key. To prevent any background noise, we ask that you please place your line on mute once your question has been stated.

Our first question is from Brian Abrahams from RBC Capital Markets. Your line is now open.

Brian Abrahams — RBC Capital Markets — Analyst

My first question — what efficacy data would you be able to provide to the FDA on the ongoing BOSTON study without comprising that trial? What mechanisms and processes might be in place to do that? What degree of maturity of data would be available just based on the trajectory of enrollment, which I know completed in January ahead of the July PDUFA?

Michael Kauffman — Chief Executive Officer

So, point one and very important is the FDA has specifically asked us not to disclose what data they requested and what data they reviewed in their review of the application. Separately, what we have said is our Data Safety Monitoring Board, which meets periodically throughout the trial did review and go through the first interim analysis, which includes the safety review, including an analysis of fatalities on each arm of the trial and in addition, certain efficacy data, which is assembled by a third-party for which we are blinded as a company.

Based on the safety review, they found no new safety findings and no concerns about potential imbalances on the trial and on the efficacy side, what we know is the DSMB did not recommend any alteration to the sizing of the trial and they did not indicate the trial was futile. So, the trial is proceeding along the lines for which it was originally designed.

Brian Abrahams — RBC Capital Markets — Analyst

Could you provide more detail around the parameters around that interim with respect to what the bar might have been for futility or for resizing?

Michael Kauffman — Chief Executive Officer

We don't disclose that data, but what you can glean from the statistical parameters in the study is that we're looking for approximately a 30% improvement in the overall progression-free survival in the study and that's the primary endpoint. As you know, the key secondary endpoint is the overall response rate.

Brian Abrahams — RBC Capital Markets — Analyst

One more from me — I'm wondering if you could give us anymore detail on how the EMA review process is going and maybe just qualitatively the nature of their questions. Were they asking some of the same questions from some of the same data that FDA was? What might we think of the European processes being different given their preference for oral drugs in this disease?

Michael Kauffman — Chief Executive Officer

I think generally speaking, there are no surprise questions at all from the Europeans. They largely follow the FDA's questions. Given the size of this application and the time it's coming back, we'll be able to turn this around in a timeframe that gives us an answer by the end of the year.

Operator

Thank you. Our next question is from Maury Raycroft from Jefferies. Your line is now open.

Mitchell — Jefferies — Analyst

Hey, guys. This is Mitchell on for Maury. Thank you for taking our questions. I wanted to ask if you're able to give any context on what the FDA was looking for. Is it more on the safety side or efficacy side as well?

Michael Kauffman — Chief Executive Officer

The FDA was quite responsive to us after conclusion of the ODAC meeting. One of the lines that I'm happy to share with you is they not only look at the total votes, but they look at the context of the comments in the votes. I think there were several members of the ODAC committee that seemed to understand myeloma in great detail and had some comments that seemed to resonate with the FDA regarding the Selinexor efficacy and safety data. I think the FDA was looking for additional reassurance that some of these comments were correct.

I will add that the underlying theme with what was ongoing at the time with venetoclax in its Phase 3 with venetoclax versus Velcade-dex, the FDA was conducting its own analysis throughout our review process and during our ODAC process. About two weeks after ODAC, the FDA released a clinical hold or implemented a clinical hold on venetoclax. I think there's been a heightened sensitivity around efficacy and safety in myeloma and the FDA was doing appropriate due diligence on all new drugs being developed in this area.

Mitchell — Jefferies — Analyst

Thank you. That's helpful. Are you able to say if they request additional data beyond BOSTON?

Michael Kauffman — Chief Executive Officer

I can't speak to any of the data, specifically what they requested. It was a very wholesome review of the entire data set. Again, with the backdrop of venetoclax and other results in myeloma, they were doing their appropriate diligence to make sure they understood the behavior of our compound.

Operator

Our next question is from Jonathan Chang from SVB Leerink. Your line is open.

David Ruch — SVB Leerink — Analyst

This is David Ruch on for Jonathan. Thanks for taking our question. Could you help set investor expectations on the KING data coming at ASCO?

Michael Kauffman — Chief Executive Officer

Yeah. The KING study was conducted in patients with previously treated and progressive glioblastoma multi-forming QVM, which is a type IV glioma. These patients really have no known treatment options of benefit. This was a single-agent Selinexor study. What we're looking for is both responses into destabilization and those will be reported at ASCO in the oral presentation.

David Ruch — SVB Leerink — Analyst

Could you provide any further context on the EMA decision to switch from the accelerated review to traditional? Do you expect the EMA's decision by the end of 2019 regardless of what happens in the interim?

Michael Kauffman — Chief Executive Officer

We think the EMA certainly watched ODAC very carefully, but they had their own set of questions. This was a large application with over 1,000 patients treated with hematological malignancies in the safety database for Selinexor. So, I think it's the large amount of data. It's the diversity of diseases, a large number of questions, a lot of which are very similar to what the FDA has answered. We weren't surprised at all.

David Ruch — SVB Leerink — Analyst

Regarding timing?

Michael Kauffman — Chief Executive Officer

We think the Europeans are pretty good about making sure they stay on their timelines. Remember, the EMA process does depend on the company's ability to return information in a timely fashion. They had a large number of questions, none of which were unexpected, but they wanted to make sure the company had plenty of time to return the answers.

Operator

Our next question is from Eric Joseph from JP Morgan. Your line is now open.

Turner — JP Morgan — Analyst

This is Turner on for Eric. A couple questions from us — Michael, I think you've previously cited mature duration response as a factor to filing. How should we be thinking about the level of sensitivity around DOR that we initially saw at ASH in 2018 given the number of censored events? Then the second part to that — what would be viewed as clinically meaningful with respect to DOR and PFS, looking toward the first half '20 submission?

Michael Kauffman — Chief Executive Officer

Remember that in an accelerated approval, response rate and duration of response are important. There have been no small molecule drugs approved in DLBCL period. The small molecule drugs that are sometimes used such as Revlimid and to some extent ibrutinib are mainly restricted to the ADC subtype of the disease and unfortunately have single-agent duration of response in the four-month range. I would comment on the side that in myeloma, four months in a refractory population is considered quite important.

In lymphoma, typically they're looking for at least six months' duration of response. We consider that our goal in this study and then looking at response rate that are above the 20% to 25% rate in these patients who have no other options. Those are the bases. I think we will update in the future, I hope, on the stable results because one of the reasons the FDA asked us to hold on the application until we had mature data was because of the number of censored events on the DOR.

We want to make sure we cross that six-month line with the vast majority of patients having crossed that line as opposed to being early on. We'll have to wait to see in terms of public disclosure of data, but we are excited and remain confident that these durations that are north of seven months for our drug with response rates in the 20% to 30% are real and will hold up and we can submit the application.

Turner — JP Morgan — Analyst

And then one last one — SIENDO is now a company-sponsored trial, which requires a certain amount of investment. Has it been incorporated into prior guidance? How has your thinking changed with respect to the probability of success in the indication to convert to the company-sponsored trial versus an IST?

Michael Kauffman — Chief Executive Officer

I'll start and then turn it over to Mike for the budgetary question. We're increasingly excited about potential for this drug in that indication and we want to move it faster in an investigator-sponsored trial. That is the reason for what was done. The lead investigator is [inaudible] in Europe and he's been a huge supporter of this program and will continue to be. Mike?

Mike Mason — Chief Financial Officer

The costs for SIENDO are in the expense guidance we gave this morning, which is, again, between $200 million and $215 million of opex for 2019.

Michael Kauffman — Chief Executive Officer

Just to add to that quickly, I think it's important to take any of these, especially these new programs, into the context of what potential market size we're looking at. There are currently no maintenance therapies approved for patients with endometrial cancer. This is in stark contrast to ovarian cancer, where CARP inhibitors have been important and life-expanding therapies in the maintenance setting. So, we're looking to do that.

As you know, our drug has a broad mechanism of action. So, we don't have any particular genotypes specified for uterine cancer. By following initial platinum therapy, our drug would come as maintenance therapy for those patients, which is the vast majority who obtain at least a partial response or a complete response to their initial therapy. We estimate the size of these markets are well north of $500 million and could be as high worldwide as $1 billion. This is a substantial market with a substantial unmet medical need.

Operator

Thank you. Our next question is from Ying Huang from Bank of America Merrill Lynch. Your line is now open.

Alec — Bank of America Merrill Lynch — Analyst

Hey, guys. This is Alec on for Ying. My first question is on Selinexor in DLBCL. Do you view the NDA submission in DLBCL is contingent upon approval in multiple myeloma given they are quite different diseases? SADAL, like STORM, is a single-arm study, so, how do you plan to frame the NDA given the FDA's preference for randomized control studies?

Michael Kauffman — Chief Executive Officer

Let me start with the second question. The difficulty in DLBCL is that in patients who are not eligible for transplant or those that have progressed following transplant or CAR T therapy, there are no approved therapies and no standard of care. A quick look at the NCCN guidelines will verify that. It's virtually impossible to design a study that has an appropriate control arm. People have tried in the past. They've either died dealers' choice or physicians' choice control arms. They just don't accrue and there's little interest.

So, we designed the SADAL study with the goal to show single-agent Selinexor activity, which in and of itself is a very important demonstration. The reason for us giving a little bit of a wait and see on the exact SADAL filing is we don't want to end up with another ODAC around the single-arm trial in DLBCL like we did in myeloma. We are working with the FDA to make sure we're all on the same page as we review this.

I should mention about 80% of the NDA for SADAL has already been reviewed by the FDA in terms of safety data. What they're really reviewing is the efficacy data from the SADAL study and any updated data from ongoing trials we would provide. We want to make sure we're completely walking together with FDA and not end up with a difficult ODAC. If we do have to delay it until the BOSTON topline data are available, we could, but if we have good discussions with FDA and move forward with the myeloma approval, they would be open to independent SADAL NDA coming in before the BOSTON data.

Alec — Bank of America Merrill Lynch — Analyst

Thank you. As you're in a holding period waiting for the approval in multiple myeloma, how are you managing the prior commercial ramp? Mostly, I guess, how are you utilizing the sales team right now? If the PDUFA were to be delayed further, how do you plan to manage G&A expense the rest of the year?

Michael Kauffman — Chief Executive Officer

I'm going to turn it over to Anand Varadan, our Chief Commercial Officer.

Mike Mason — Chief Financial Officer

Thank you for the question. First of all, our full field organization is hired and then placed and trained to the extent that's possible on the disease state and in terms of the customer base and the like. We're using this timeframe before approval to engage with customers in an appropriate way, so, to educate them around the mechanism of disease. They have materials in order to do that, to really make sure they understand the role of SINE technology and around nuclear export as it plays a role within cancer.

That's a new area in terms of mechanism that's not necessarily familiar to this customer base. They're also using this time to profile the customers and try to understand what the movement of patients are through their various practices, where they're likely to be given the label that we would anticipate or the kinds of labels we might be able to anticipate for Selinexor or when it's approved. So, we can be in the strongest posture to have a strong ramp for the commercial uptake after approval.

Operator

Thank you. At this time, I am showing no further questions. I would like to turn the call back to Michael Kauffman for closing remarks.

Michael Kauffman — Chief Executive Officer

Thank you, everybody for joining today's call and have a great day.

Operator

Ladies and gentlemen, thank you for your participation in today's conference. This concludes the program. You may now disconnect.

Duration: 34 minutes

Call participants:

Ian Karp — Vice President of Investor Relations and Public Relations

Michael Kauffman — Chief Executive Officer

Mike Mason — Chief Financial Officer

Anand Varadan — Chief Commercial Officer

Brian Abrahams — RBC Capital Markets — Analyst

Mitchell — Jefferies — Analyst

David Ruch — SVB Leerink — Analyst

Turner — JP Morgan — Analyst

Alec — Bank of America Merrill Lynch — Analyst

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