Q2 2020 Karyopharm Therapeutics Inc Earnings Call

Operator today.

I will.

At this time I would like to welcome everyone to Karyopharm Therapeutics second quarter Twentytwenty 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. in car.

Karyopharm Senior Vice President Investor and public Relations. Please go ahead.

Thank you Debbie you all for joining us on todays conference call to discuss Karyopharm second quarter 2020 financial results and business update.

Car and I'm joined today by Dr., Michael Kauffman, Chief Executive Officer, Dr., Sharon Shopko, President and Chief Scientific Officer, Mr., Mike Mason, Chief Financial Officer, Mr., John Demeritt, Chief Commercial Officer Mr. Christopher for me on our Chief business Officer in General Counsel.

Oh, sorry, Michael John will provide an overview of key recent corporate developments and an update on our commercial.

Mike <unk> second quarter 2020 financial results will conclude with extremely fortunate we call.

Earlier. This morning, we issued a press release detailing karyopharm results for the second quarter of 2020. This release along with a slide presentation that we plan to reference are available on our website at <unk> Dot com.

Before we get a final comments are wide at various remarks, we'll make today constitute forward looking statements for purposes of the Safe Harbor provisions under the private Securities Litigation Reform Act 99.

These statements about our future expectations clinical development and regulatory matters timelines the potential success for products and product candidates.

Patients regs are the commercialization Fabio financial projections, and our plans and prospects actual results may differ materially from those indicated by these forward looking statements as result of various important factors, including those discussed in the risk factor section. Most recent quarterly report on form 10-Q, which is on file with the FCC. Another five that we may make with yes.

Future.

These statements represent our views of today.

Well, we made I like to update these forward looking statements at some point in the future. We specifically disclaim any obligation to do so even if our views changed therefore, you should not one of these forward looking statements.

Representing our views they tell us what you today. Your question. Please know that any references we make the clinical trial data drug today's discussion refer to internet site data unless otherwise specified I'll now turn the call are Dr. Mike.

Chief Executive Officer, Thank you and and good morning, everyone before I, specifically, what you dairy farms performance in the second quarter I'd like to put into some context, where karyopharm is on its journey, becoming a leading global oncology focused pharmaceutical company or those following along in the presentation. Please turn to slide four.

Dr. Shrotriya coming on founded the company over 12 years ago, We had a vision that one day, we pre medicines that are approved the lives of patients suffering from cancer and other serious diseases. We spent the first fiber some years building the organization, probably besides body of scientific and clinical evidence around actually go on inhibition.

To the approval first in class oral compound exposure.

And in July of last year, we affectionately transition toward commercial company, but the initial FTC approval and launch of exposure in relapsed or refractory multiple myeloma.

This year, we achieved an additional important milestone on our journey.

We already see get second after FDA approval at the end of June became the first branded therapy to be approved in both myeloma Andy.

As we look out towards next year and beyond we expect to expand our commercial reach and advanced robust clinical development pipeline, including into solid tumor indications all in support of the patients and families you need a new treatment options are devastating cancers.

Now turning to slide five.

In the second quarter, we achieved record quarterly exposed meal sales of $18.6 million a strongest quarter since our July 2019 watch total revenue for the quarter reached 33.5 million, which included most of the payment from one of our international partners MPG in order for our expanded territory.

Earlier this year, the 16% increase then exposure beyond that sales compared to the first quarter up 2020 was driven by increased demand for both academic and community based physicians.

Importantly, we saw more than 170, new physician accounts prescribing spoke out for the first time and an order even as in person activities were limited due to regional restrictions related to the ongoing called <unk>.

This represents more new accounts added in Q2 that in Q1, despite the ongoing but that anda.

The second quarter was also marked by the FDA ramping accelerated approval installed meal or the treatment of patients with excuse part b cell lymphoma, or do you all bcl, including deal Bcl arising from molecular lymphoma. After at least two lines of systemic therapy. This represents a second is approved indication for its Fabio we were able to launch immediately.

Leveraging our existing commercial infrastructure, given the overlap between oncologists treating myeloma and deal Bcl, particularly in the community setting.

The annualized still meeting in May we presented the detailed results in a highly successful phase three Boston study in patients with previously treated myeloma Boston is our randomized trial once weekly selinexor with once weekly Velcade, along with dexamethasone compared to standard twice weekly Okay index. So the trial met its pre specified primary.

Endpoint and based on these results we put we submitted a supplemental new drug application or sndk to the FDA are exposed we opened the treatment of myeloma after at least one prior lines of therapy.

FDA has filed the application and assigned an action date of March 19, 2021 under the prescription drug user fee I do.

I'm also pleased to announce that we have now completed enrollment in our phase three study of Selinexor in patients with heavily pretreated de differentiated like the sarcoma and expect topline data in the second half of this year.

These data are positive we believe this will be a very important step towards our goal upbringing Oh, yeah to patients battling solid tumors. In addition to those looking at a large malignancies.

We also made important progress in our clinical pipeline outside of cancer and today, we are providing an update on our placebo controlled phase two trial up logo selinexor for patients with severe cold 19 that is patients who are hospitalized hypoxic and require supplemental oxygen.

This program was prioritizing karyopharm and rapidly advance into the clinic and her matter of weeks due to the encouraging anti viral and anti inflammatory activity like stelvio inhibition with Selinexor.

Recall that in addition to its roles in cancer XP. All one may also be an important targeted viral infections.

Okay, and then transport of several key viral proteins from the nucleus of the host sell to decide on class.

At the planned interim analysis of the randomized phase two study with approximately 120 patients. The results indicated that selinexor is unlikely to demonstrate statically significant efficacy benefit across the entire population of patients hospitalized and see or severe over 19, where study however.

Selinexor does appear to confer clinical benefit and some population of patients that represents about three quarters of the whole severe cobot 19 population.

Based on these results we have discontinued the current study and expected future clinical development of mono Selinexor.

Oh, and 19, well focus on a sub population we saw the significant defects I'll provide some additional details on these specific results later in todays presentation.

On the corporate development front, we were excited to announce our entry into a cooperative research and development agreement or credo, but the National Cancer Institute cancer therapy evaluation program for the research of exposure. We are in various oncology indication, which we hope you have additional opportunities for us and patients in the future.

Finally on the financial front, we ended the quarter with a strong cash position of approximately $348 million and we expect will be sufficient to fund our plan operations into the middle of 2022.

Let's now turn to slide six well ask John Demeritt, our chief commercial officer to provide some additional details onyx ws outperformance during the second quarter John.

Thank you Michael Good morning, everyone as Michael noted earlier, we were pleased with the commercial momentum theme during the quarter, despite being impacted by some of the challenges associated from the carbon 19 pandemic, including limited in person sales visits to our customers I commend the care for our commercial organization for driving our education.

Initiatives for work virtually during these challenging times in support of our community of patients families and caregivers our virtual initiative for both multiple myeloma DLP CEO include digital tools to facilitate continued sales force nurse liaison air team engagement with customers.

Agenda in marketing and media placement and virtual peer to peer programs. Additionally, we believe expose product profile offer some distinct advantages for our patients during the pandemic, including oral administration at home delivery, which can eliminate the need to travel to hospitals and clinics as frequently as commonly.

Used peripheral therapies.

As of June Thirtyth, approximately 3200 exposure you know prescriptions have been filled in the U.S. That's large at approximately 950 prescriptions were filled in the second quarter prescription demand increased by 12% compared to the first quarter and as Michael highlighted earlier net sales increased.

By 16% in the quarter the second quarter was the strongest quarter to date for both net sale and patient demand its growth was particularly exciting as the overall demand in the U.S. for a number of other multiple myeloma drugs declined in the second quarter as a result at the ongoing pandemic further underscore.

The strong performance for exposure.

There was also incremental added some incremental inventory added at our distribution partners in Q2, driven by initial stocking at the end of June three new exposure package sizes to support the commercial watch patients with deal Bcl.

Turning to slide seven I will provide some additional color I patient metrics that we follow closely and believe further reinforce the positive experience many physicians and patients are having with exposed the treatment.

The graph on the website at this slide highlights the average treatment cycles or prescriptions per patient based on data from our participating specialty pharmacies.

You will notice is that this number has increased steadily each quarter since March as we believe physicians are getting more comfortable treating patients with exposed the out at effectively managing its potential side effects.

The end of June average number of prescriptions per patient was 2.7 as compared to 2.0, yes, but 2019.

On the right side of this slide the graph shows the prescription refill rate overtime for both the first and second prescription refills are those patients eligible. These numbers have also continued to improve significantly each quarter since launch as an example in September 2019, which marked the end of the.

First quarter studio became commercially available roughly 42% of patients were going on to get a second prescription.

The end of June 2020 that number stood at 61 first at an impressive improvement.

Increasing refill rates, coupled with the increasing average number of treatment cycles further reinforced the positive feedback we've received for patients and physicians regarding their experience and usage of exposure.

Moving forward, we plan to continue to engage with the medical community and reinforced the potential benefits of exposed the out through virtual and in person engagements where possible. We will continue to fully leverage the newly approved indication and Dl Bcl access our key stakeholders educate.

On this new indication it also generate increasing excitement in multiple myeloma to help more patients impacted by these diseases.

Now I'd like to turn the call back over to Michael to discuss our key clinical development activity than priorities, which began on slide eight Michael Thank you John.

Critical milestone for us in the second quarter included a formal presentation of the positive Boston study results. As a reminder, this was the first large phase three study and previously treated myeloma to directly compare a once weekly triplet regimen with standard twice weekly Velcade.

We were thrilled that the study met its primary endpoint statistically and clinically significant improvement in PFS or the 47% increase in the median progression free survival on the SPD arm as compared to the BDR, representing a 4.47 month improvement.

The median progression free survival in the SPDR was 13.93 months compared to 9.46 months into VTR, where the hazard ratio of 0.70.

SPD was superior to be across other key efficacy endpoints such as overall response rate the rate a very good partial response or better and the duration of response the results for consistent across most of the sub populations, including patients over the age of 65 patients who are frail those with prior Lenalidomide Street.

And those with high risk cytogenetics.

You know verse all survival on SPDR has not yet and reached but it was 25 months on the BDR. Moreover, there where numerically fewer deaths on the SPDR then on the VDR.

We were also pleased to report the rate of peripheral neuropathy on the SPD arm was significantly lower than the rate in the BD controller.

Remember that peripheral neuropathies among the most common causes treatment limitation and discontinuation of BD and combination BD regiments and as a significant differentiator for the Boston regimen any future regulatory approval, but they also show no new safety signals in the SPD aren't relative previously reported adverse events.

Other selinexor myeloma trials.

In summary, we were able to conclude from the Boston study that in patients with multiple myeloma, who have received one of the three prior therapies, including prior lenalidomide or protease inhibitor. Once weekly SPD offers patients on effective convenient triplet regimen, acquiring approximately 40% Youre clinic business and a reduced rate of peripheral nerves.

The pending future regulatory approval of course.

On slide nine I'll provide a brief update on our key upcoming regulatory activities. Our Boston SNG I was accepted for filing in July So isn't expected by March 19, 2021 in Europe, we plan to submit the final additional monitoring data requested by M&A from a storm study in patients with advanced Refrac.

Hey, myeloma in September.

We then expect to submit the Boston data to the remain a fourth quarter. This year exact timing of potential regulatory decisions based on both the storm and Boston data will be determined following feedback from me on that.

As we turn to slide 10.

I will highlight our recent accelerated approval for scobey out for the treatment of adult patients with relapsed or refractory DLP see out in the sales study with served as the basis for approval exposure you know demonstrated an overall response rate of 29%, including a complete response rate at 13%.

Responses were seen in all sub groups, regardless of age gender prior therapy, GCB or ABC subtype or prior stem cell transplant therapy.

Importantly responses were associated with longer survival underscoring the potential of oral SPL, one inhibition as a simple non terminal therapeutic option for patients with recurrent DLP seal.

Well, we have just kicked off our commercial launch in this indication we believe its phobias product profile addresses several key treatment considerations, but it's difficult to treat disease as displayed on slide 11.

Operator: Operator today. I will.

Physicians, commonly examination history, and Z subtype previous therapies use and treatment efficacy and durability when deciding on an appropriate treatment pathway. The simple administration single agent and novel mechanism of action of exposure provides additional advantages for use with a manageable safety profile.

Operator: At this time, I would like to welcome everyone to Karyopharm Therapeutics' second quarter 2020 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. Ian Karp, Karyopharm's Senior Vice President, Investor and Public Relations. Please go ahead.

Next please turn to slide 12 hour you the updates from our covert 19 study.

Ian Karp: Thank you, Debbie. And thank you all for joining us on today's conference call to discuss Karyopharm's second quarter 2020 financial results and business update. This is Ian Karp, and I'm joined today by Dr. Michael Kaufman, Chief Executive Officer; Dr. Sharon Shacham, President and Chief Scientific Officer; Mr. Mike Mason, Chief Financial Officer; Mr. John Demary, Chief Commercial Officer; and Mr. Christopher Firmiano, Chief Business Officer and General Counsel. On the call today, Michael and John will provide an overview of key recent corporate developments and an update on our commercial progress, and then Mike Mason will highlight the We will conclude with the Q&A portion of the call.

Based on Andy stars chronic viral activity in vitro and anti inflammatory activity in vitro and Nvvault Karyopharm began enrollment in a placebo controlled randomized phase two study to evaluate low dose oral selinexor in hospitalized patients with severe cobot 19 in April this year these patients require supplemental oxygen.

And our at high risk for clinical worsening.

Many of them had already received from Descovy or steroids convalescent plasma and other agents used to treat covered 19.

As mentioned previously the data safety monitoring board for the study concluded trial is unlikely to demonstrate a statistically significant efficacy benefit across the entire population of severe patients who are I'm roll. However, they also concluded that the trial was likely to show a benefit in the subpopulation uptake.

Ian Karp: Earlier this morning, we issued a press release detailing Karyopharm's results for the second quarter of 2020. This release, along with a slide presentation that we plan to reference, is available on our website at karyopharm.com. Before we begin our formal comments, I'll remind you that the various remarks we will make today constitute forward-looking statements for purposes of the State Department provisions under the Private Securities Litigation Reform Act of 1995.

Ian Karp: These include statements about our future expectations, clinical developments, regulatory matters and timelines, the potential success of our products and product candidates, including our expectations related to the commercialization of Expovia, 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 quarterly report on Form 10-Q, which is on file with the SEC, and another filing that we may make with the SEC in the future. Any forward-looking statements represent our views of today only, but 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.

Thanks for less than 75 years, all and have a Cobra cobot Graham non hybris for which represented approximately 75% of the entire population enrolled in the study.

Preliminary results from the unaudited site data indicate that in this specific sub population on the primary endpoint for the entire study, which was a two point improvement in ordinal score a day 14.

Statistically significant results were obtained.

In addition on day 28.

Improvement in normal score by two points also reached statistical significance with P. is less than or equal to <unk> 0.05.

And very importantly array of hospital discharge by day 14 was significantly higher with Selinexor as compared with placebo in other words patients treated with selinexor stopped requiring supplemental oxygen more rapidly than those on placebo and could leave the hospital sooner.

Ian Karp: Therefore, you should not rely on these forward-looking statements as we're 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 refer to interim, unaudited site data, unless otherwise specified. I'll now turn the call over to Dr. Michael Kaufman, Chief Executive Officer. Thank you, Ian, and good morning, everyone.

Finally, why all patients entering the study were required to have positive Sars koby to PCR results conversion into Sars koby to PCR negative status was significantly higher on the selinexor arm as compared with the placebo arm across the entire population.

Michael P. Mason: Before I specifically review Kareo Pharm's performance in the second quarter, I'd like to put into some context where Kareo Pharm is on its journey to becoming a leading, global, oncology-focused pharmaceutical company. For those following along in the presentation, please turn to slide four. When Dr. Sharon Shackham and I founded the company over 12 years ago, we had a vision that one day we would create medicines that improve the lives of patients suffering from cancer and other serious diseases. We spent the first five or so years building the organization, growing the body of scientific and clinical evidence around XPO1 inhibition, leading to the approval of the first-in-class oral compound Expovio. And in July of last year, we officially transitioned to a commercial company with the initial FDA approval and launch of Expovio for refractory multiple myeloma.

How are these were similar into subpopulation mentioned above.

While the rate of pay talent ease in the study were imbalanced and the patients 75 years, our older over the call, we'd Graham high risk score.

Michael P. Mason: This year, we achieved an additional important milestone on our journey as Expovio received its second FDA approval at the end of June and became the first branded therapy to be approved in both myeloma and DLPCL. And as we look out towards next year and beyond, we expect to expand our commercial reach and advance a robust clinical development pipeline, including into solid tumor indications, all in support of patients and families in need of new treatment options for devastating cancer. Please now turn to slide five.

SMB considered that all Desama study were due to severe cobot, 19 disease and or underlying a morbidities without a clear contribution of selinexor.

Moving forward and based on these data we are following the SMB recommendation to discontinue the current trial and plan to analyze the data to further characterize as specific sub population that will likely benefit from selinexor.

We plan to work with the FDIC and other regulatory bodies to identify a path forward for future clinical development. Even these initial positive findings.

Furthermore, we plan to engage with potential partners and seek external funding to advanced future clinical development in covert 19.

Before turning the call over to Mike Mason I do want to take a moment just sincerely. Thank all the patients investigators who participated in this study and support of our collective efforts to make positive difference in the ongoing pandemic.

With that I'll now turn the call over to Mike for a review of the financials, Mike. Thank you Michael since we issued a press release earlier today with a full financial results I will just focus on the highlights which began on slide 14 net product revenue for the second quarter of 2020 was 18.6 million total revenue was 33.5 million.

Michael P. Mason: In the second quarter, we achieved record quarterly Expovio sales of $18.6 million, the strongest quarter since our July 2019 launch. Total revenue for the quarter reached $33.5 million, which included most of the payment from one of our international partners, Antigene, in the quarter for our Expanded Territory Agreement, which we announced earlier this year. The 16% increase in Expovio net sales compared to the first quarter of 2020 was driven by increased demand from both academic and community-based positions. Importantly, we saw more than 170 new physician accounts prescribe Expovio for the first time in a quarter, even as in-person activities were limited due to regional restrictions related to the ongoing COVID pandemic. This represents more new accounts added in Q2 than in Q1, despite the ongoing pandemic.

As previously mentioned second quarter sales were driven by patient demand from academic and community based positions with minimal channel inventory build in the second quarter.

Related to our deal Bcl launch in late June.

The estimated gross to net discount for exposure in the second quarter was approximately 16%. We continue to expect a gross to net discount to fall between 15 and 20% throughout the remainder of this year.

Research and development expense for the second quarter of 2020 was 42.6 million compared to 26.5 billion for the second quarter of 2019.

Michael P. Mason: The second quarter was also marked by the FDA granting accelerated approval of Expovio for the treatment of patients with diffuse large B-cell lymphoma, or DLBCL, including DLBCL arising from follicular lymphoma after at least two lines of systemic therapy. This represents the second approved indication for Expovio. We were able to launch immediately by leveraging our existing commercial infrastructure, given the overlap between oncologists treating myeloma and DLBCL, particularly in a community setting. At the annual ASCO meeting in May, we presented the detailed results from the highly successful Phase III Boston study in patients with previously treated myeloma. Boston is our randomized trial of once-weekly telonexer with once-weekly Velcade, along with dexamethasone, compared to standard twice-weekly Velcade and dexamethasone.

The increase in R&D expenses compared to the second quarter 2009.

It was mainly due to the rapid initiation enrollment or cold in 19 study, which I'll discuss will now be discontinued and we expect R&D costs to be relatively consistent for the remainder of 2020 on a quarterly basis.

For the second quarter of 2020, SJ expense was 30.8 million compared to 24.7 million for the second quarter of 2019, the increased unless you had expenses compared to the prior year was primarily due to activities to support the U.S. commercialization of exposure and preparations for the launch of exposure as a treatment for patients with relapsed.

Refractory DLP steel.

Cash cash equivalents restricted cash and investments as of June 30, 2020 toll of 340 million compared to 265.8.

Michael P. Mason: The trial met its pre-specified primary endpoint, and based on these results, we quickly submitted a Supplemental New Drug Application, or SNDA, to the FDA for expovio for the treatment of myeloma after at least one prior line of therapy. FDA has filed the application and assigned an action date of March 19, 2021 under the Prescription Drug User Fee Act. I'm also pleased to announce that we have now completed enrollment in our Phase 3 study of Stelinexer in patients with heavily pre-treated, de-differentiated liposarcoma and expect top-line data in the second half of this year. If these data are positive, we believe this will be a very important step towards our goal of bringing exfolio to patients battling solid tumors, in addition to those with hematologic malignancies.

Our first 2019.

Based on our current operating plans, we expect our non-GAAP R&D and SGN expenses, which excludes stock based compensation expense for the full year clients wanting to be on a previously projected range of 240 to 206 million.

In addition, as shown on slide 15, we currently expect that our existing cash cash equivalents and investments and the revenue we expect to generate permits overview product sales will be sufficient to fund our plant operations in the middle of 2022, which puts us in a terrific position to further advance our strategic aspirations I will now.

Michael P. Mason: We have also made important progress in our clinical pipeline outside of cancer, and today, we are providing an update on our placebo-controlled phase two trial of low-dose Selenexer for patients with severe COVID-19. That is, patients who are hospitalized, hypoxic, and require supplemental oxygen. This program was prioritized at Karyopharm and rapidly advanced into the clinic in a matter of weeks due to the encouraging antiviral and anti-inflammatory activity of expovial inhibition with Selenexer. Recall that in addition to its roles in cancer, XPO1 may also be an important target in viral infections by facilitating the transport of several key viral proteins from the nucleus of the host cell to the cytoplasm.

I'll turn the call back over to Michael.

Michael P. Mason: At the planned interim analysis of the randomized phase two study with approximately 120 patients, the results indicated that Selen X-ray is unlikely to demonstrate a statistically significant efficacy benefit across the entire population of patients hospitalized with severe COVID-19 who were studied. However, Selinexer does appear to confer clinical benefit in a subpopulation of patients that represents about three quarters of the whole severe COVID-19 population. Based on these results, we have discontinued the current study and expect that future clinical development of low-dose Selinex for patients with COVID-19 will focus on a subpopulation where we saw significant effects. I'll provide some additional details on these specific results later in today's presentation. On the corporate development front, we were excited to announce our entry into a Cooperative Research and Development Agreement, or CRADA, with the National Cancer Institute's Cancer Therapy Evaluation Program for the research of Expovio in various oncology indications, which we hope will yield additional opportunities for us and patients in the future.

Including remarks idle.

Thank you Mike before moving to the QNX, Let me highlight some of the key clinical and regulatory milestones that we expect for the remainder of 2020 shown on slide 16.

As you can see we've done a terrific job so far in 2020, achieving many of the goals we set forth at the start of the year, but we have some important goal still to accomplish.

First we plan to submit additional storm data and all Boston data TDMA in coming months, we hope to have greater clarity on the timing of future regulatory decisions following feedback from the agents.

Next we expect to share topline phase three data from the steel study evaluating selinexor and life a sarcoma assuming positive results on the primary endpoint of PFS.

Subsequent regulatory submissions and finally, we anticipate initiating a confirmatory phase three study how big Toby on deal Bcl in support of our recent accelerated approval.

Of course, we will work diligently through the remainder of the year to support our Sndk based on the Boston study as well as explore potential next steps for Selinexor development in patients with severe cobot 19.

As we look forward to the next several years, we will increasingly expand our activities in solid tumors, both as a single agent and in combination. These include uterine cancer with the ongoing phase three CNDO study.

Obama multi farming lawn combination.

Colon cancer and lung cancer.

We appreciate your ongoing support and look forward to keeping you updated on our 2020 progress I'll now turn the call over to the operator for questions operator.

Okay.

We will now begin the question and answer session.

To ask a question. Please press Star then one on your telephone keypad. If you are using a speakerphone. Please pick up your handset before pressing the keys.

Michael P. Mason: Finally, on the financial front, we ended the quarter with a strong cash position of approximately $348 million that we expect will be sufficient to fund our planned operations into the middle of 2022. Please now turn to slide 6, where I'll ask John Demery, our Chief Commercial Officer, to provide some additional details on Expovio's sales performance during the second quarter.

To withdraw your question. Please press Star then to.

Please limit questions to one question and one follow up question.

At this time, we will pause momentarily to assemble our roster.

The first question comes from Mari Ray Crock with Jefferies. Please go ahead.

John Mascarenhas: Thank you, Michael. And good morning, everyone. As Michael noted earlier, we were pleased with the commercial momentum seen during the quarter, despite being impacted by some of the challenges associated with the COVID-19 pandemic, including limited in-person sales visits to our customers. I commend the Karyopharm Commercial Organization for driving our educational initiatives forward virtually during these challenging times in support of our community of patients, families, and caregivers. Our virtual initiatives for both multiple myeloma and DLV-CL include digital tools to facilitate continued sales force, nurse liaison, and pair team engagement with customers; Search Engine Marketing and Media Placement, and Virtual Peer-to-Peer Programs.

Hi, good morning, everyone and thanks for taking my questions and congrats on the progress.

I had a question about the Koby 19 study so for that the less than 75 year old patients it better Graham on non high res a patients where you saw this benefit can you go and can you go into more details on now what you know so far about how other medications are treatments could have impacted results and then it seems like.

This is the the larger population of Coca 19 patients do you expect additional funding it support to come from external pharma partner or would it be for my governments.

Yes. The second part is where were in ongoing and expect additional discussions.

John Mascarenhas: Additionally, we believe Expovio's product profile offers some distinct advantages for our patients during the pandemic, including oral administration and home delivery, which can limit the need to travel to hospitals or clinics as frequently as commonly used parenteral therapy. As of June 30th, approximately 3,200 Expovio prescriptions have been filled in the U.S. since launch, and approximately 950 prescriptions were filled in the second quarter. Prescription demand increased by 12% compared to the first quarter, and as Michael highlighted earlier, net sales increased by 16% in the quarter.

For for all options in terms of next steps up given all the things on our own played in oncology. We believe these strong proof of concept results will lead to some kind of a partnership to make sure. This moves along but we don't we will not be able to fund that.

In terms of the the.

Risk factors for response, or if you will or better prognosis with selinexor. It. So far we've not found that prior therapy really impacts the outcomes in this population where there was a significant benefit in fact, what was I think more surprising was many of the patients we received including in that that ticket growth.

John Mascarenhas: The second quarter was the strongest quarter to date for both net sales and patient demand. This growth was particularly exciting as the overall demand in the U.S. for a number of other multiple myeloma drugs declined in the second quarter as a result of the ongoing pandemic, further underscoring the strong performance for Expovio. There was also some incremental added inventory added at our distribution partners in Q2, driven by the initial stocking at the end of June of three new Expovio package sizes to support the commercial launch in patients with DLBCL. Turning to slide seven, I will provide some additional color on patient metrics that we follow closely and believe further reinforce the positive experience many physicians and patients are having with Expovio treatment.

Already gone through and more worsening on the available therapy. So we kind of got a our relapsed or refractory population, but so far there is no specific.

Prior therapies that mattered and that's that's somewhat consistent with the novel mechanism of Selinexor in this disease.

Got it and maybe one quick question on a commercial update so you mentioned the new package sizes.

Can you just go into any more specifics on on how the deal Bcl package size could impact multiple myeloma and then for inventory. If you can provide any more specifics on what you saw in inventory smarter.

Sure.

Michael Yes, we introduced three new package sizes at the end of this quarter to match the label dosing requirements for patients with DLP CEO. We introduced a 60 milligrams twice a week 40 milligrams twice a week to 40 milligram.

John Mascarenhas: The graph on the left side of the slide highlights the average number of treatment cycles or prescriptions per patient based on data from our participating specialty pharmacies. What you will notice is that this number has increased steadily each quarter since launch as we believe physicians are getting more comfortable treating patients with expovio and effectively managing its potential side effects. As of the end of June, the average number of prescriptions per patient was 2.7 as compared to 2.0 at the end of 2019.

Once per week to address the patient dosing within the context of the package insert of course. These package sizes could also be used if a doctor chose to use them into context of multiple myeloma also but those three package sizes were specifically introduced to address the need and deal PCL and I'll turn it over to Mike to address the inventory sure.

We are.

John Mascarenhas: On the right side of the slide, the graph shows the prescription refill rate over time for both the first and second prescription refills for those patients eligible. These numbers have also continued to improve significantly each quarter since launch. As an example, in September of 2019, which marked the end of the first quarter that Expovio became commercially available, roughly 42% of patients were going on to get a second prescription. At the end of June 2020, that number stood at 61%, an impressive improvement. These increasing refill rates, coupled with the increasing average number of treatment cycles, further reinforce the positive feedback we have received from patients and physicians regarding their experience and usage of Expovio. Moving forward, we plan to continue to engage with the medical community and reinforce the potential benefits of Expovio through virtual and in-person engagements where possible.

So we reported 18.6 million on sales for the quarter.

About 5% of that was inventory build related the DLP launched to the board approved so ladies and gentlemen, 95% was driven by patients Matt.

Great. Thanks, everyone.

The next question comes from Mike Oh with Baird. Please go ahead.

Hey, guys. Thanks for taking the question congrats on the progress as well.

Just a question on exposure you I guess, just just post presentation. The Boston data at ASCO and maybe you can provide some color on the types of feedback you've gone from physicians. There and then separately you have you noticed any increase in off label use post Vasco presentation. Thanks.

Yeah. Thanks, Mike.

The feedback has really been uniformly very positive.

I'd say, there's sort of that the several camps out there there were the people that always believed and where early adopters that were very gratified to see the results and said they were not surprise there where the people are somewhat on the fans and using the drug more cautiously we're quite excited and actually impressed.

John Mascarenhas: We will continue to fully leverage the newly approved indication and DLVCL to access our key stakeholders, educate them on this new indication, and also generate increasing excitement in multiple myeloma to help more patients impacted by these diseases. Now I'd like to turn the call back over to Michael to discuss our key clinical development activities and priorities, which began on slide eight.

The with the drug and then the group of people that were really skeptical and perhaps not using the drug yet all sort of not it and said it's time to learn how to use this drug and you can you can easily see some tweaks for some folks that have started converted and they are using the drug it's very difficult to tell.

Michael P. Mason: Thank you, John. A critical milestone for us in the second quarter was the formal presentation of the positive Boston Study results. As a reminder, this was the first large Phase III study in previously treated myeloma to directly compare a once-weekly triplet regimen with standard twice-weekly VelCase. We were thrilled that the study met its primary endpoint of a statistically and clinically significant improvement in PFS with a 47% increase in the median progression-free survival on the SVD arm as compared to the VD arm, representing The median progression-free survival in the SBDR was 13.93 months compared to 9.46 months in the VDR with a hazard ratio of 0.70. SVD was superior to VD across other key efficacy endpoints, such as overall response rate, the rate of very good partial response or better, and the duration of response.

Michael P. Mason: The results were consistent across most of the subpopulations, including patients over the age of 65, patients who were frail, those with prior lenalidomide treatment, and those with high-risk fetogenetics. However, median overall survival on the SVD arm has not yet been reached, but it was 25 months on the VD arm. Moreover, there were numerically fewer deaths on the SVD arm than on the VD

How the drug is being adopted immediately.

Michael P. Mason: We were also pleased to report the rate of peripheral neuropathy in the SVD arm was significantly lower than the rate in the BD control arm. Remember that peripheral neuropathy is among the most common causes of treatment limitation and discontinuation of VD and combination VD regimens and is a significant differentiator for the Boston regimen pending future regulatory approval. The data also showed no new safety signals in the SBD arm relative to previously reported adverse events from other felon extra myeloma trials. In summary, we are able to conclude from the Boston study that in patients with multiple myeloma who have received one to three prior therapies, including prior lenalidomide or proteasome inhibitor therapy, once weekly SBD offers patients an effective, convenient triplet regimen requiring approximately 40% fewer clinic visits On slide nine, I'll provide a brief update on our key upcoming regulatory activities. Our Boston SNDA was accepted for filing in July, with a decision expected by March 19, 2021.

We suspect that there may be increase used in combination but in general.

This is in the patients who've already gone through the available therapies and are in need of something new.

Good news is that once weekly combination therapy does seem to be well tolerated and we do believe there is some increased use of that but again, it's Adam advanced refractory population.

We have submitted to NCCN guidelines and we are waiting.

Responses from the NCCN and hopefully we'll be able to see.

The option of Boston regimen in earlier lines of therapy and of course, the PDUFA date early next year.

Okay. Thank you.

The next question comes from Peter Lawson with Barclays. Please go ahead.

Hey, thanks.

A question.

Maybe around.

I think impact.

For the than any of the dynamics you could talk through around new script growth patient growth.

This concludes would be.

Which appreciated during the quarter.

John you want to take that sure two parts to that first I'll address.

New patient growth, we continue to add new patients each quarter, what are the key drivers of our sales, especially when you consider that sub patients will also come off expose their each quarter. We're encouraged by recent trends in new patient starts that we don't breakout our sales figures by the new patient starts compared to existing patients, but we did see acute.

Okay with increasing total demand as mentioned earlier up 12% even in the context of the fall even in the context of the pandemic. The increase was driven primarily by multiple myeloma as the new deal VCR indication was approved at the ended the quarter.

In terms of the impact of the Cobot nice team had data on our commercial efforts. We believe the Kobe 19 impact, it's having multiple effects as reported by many other companies as well and secondary data sources patient visits to their doctors continue to be lower than pre pandemic levels in the context of this environment.

We were pleased to be able to growth demand for extol the over the quarter. We've also seen as an impact in person visits by our Salesforce scarcely AIDS odds and payer teams to be extremely limited based on secondary data at our own experience. We estimate only about 15% ahgps can be seen in in person visits.

Michael P. Mason: In Europe, we plan to submit the final additional monitoring data requested by EMA from the STORM study in patients with advanced refractory myeloma in September. We then expect to submit the Boston data to the EMA in the fourth quarter of this year. The exact timing of potential regulatory decisions based on both the STORM and Boston data will be determined following feedback from EMA.

We also seeing of course Vasco is virtual this year, which limited the ability to have a number of engagements with academic and community based psychologist. We are doing a number of things specifically to offset the impact of being virtual continuing to grow exposed go back for the.

Michael P. Mason: As we turn to slide 10, I will highlight our recent accelerated approval for Exscovio for the treatment of adult patients with relapsed or refractory DLV-PL. In the SATEL study, which served as the basis for approval, Exscovio demonstrated an overall response rate of 29%, including a complete response rate of 13%. Responses were seen in all subgroups regardless of age, gender, prior therapy, GCP or ABC subtype, or prior stem cell transplant therapy. Importantly, responses were associated with longer survival, underscoring the potential of oral XPO1 inhibition as a simple, non-therapeutic option for patients with recurrent DLB-PL.

Yes, we planned on and executed virtual watch we've increased our level of Dodd personal promotion and digital content, including implementing virtual meeting technology for field force engagement watching emailing mail campaigns direct professional and through the field force increasing search engine marketing spend for both HCP inpatient audiences.

Increasing media placements launching new web sites and executing exposed you education at the point of diagnosis and DMR systems. So just some examples of things we're doing to offset the ongoing impact from the code 19 pandemic.

Okay. Thanks, and then.

Good any sense of wed so they can use then theyll be Seattle and multiple I know what lines.

Michael P. Mason: Well, we have just kicked off our commercial launch in this indication. We believe Expovio's product profile addresses several key treatment considerations for this difficult-to-treat disease, as displayed on slide 11. Physicians commonly examine patient history and disease subtype, previous therapies used, and treatment efficacy and durability when deciding on an appropriate treatment pathway. The simple administration, single agent, and novel mechanism of action of Expovio provide additional advantages for use with a manageable safety profile. Next, please turn to slide 12, where I will discuss the updates from our COVID-19 study. Based on anti-SARS coronaviral activity in vitro and anti-inflammatory activity in vitro and in vivo, Karyopharm began enrollments in a placebo-controlled, randomized Phase 2 study to evaluate low-dose oral Selenixer in hospitalized patients with severe COVID-19 in April of this year.

John So as Michael just mentioned before we see the most to use that exposure beyond the context of the indicated lights. So in the context of multiple myeloma most of usage in that penta refractory setting where indicated and while we just have limited data given the early launch in.

No we see it being used there and after two or more lines of therapy, where its indicated as well.

Great. Thanks, much thanks for taking the questions.

Thanks for your question Peter.

Michael P. Mason: These patients require supplemental oxygen and are at high risk for clinical worsening. Many of them have already received remdesivir, steroids, convalescent plasma, and other agents used to treat COVID-19. As mentioned previously, the Daily Safety Monitoring Board for the study concluded that the trial was unlikely to demonstrate a statistically significant efficacy benefit across the entire population of severe patients who were enrolled. However, they also concluded that the trial was likely to show a benefit in the subpopulation of patients who are less than 75 years old and have a COVID-GRAM non-high risk score, which represented approximately 75% of the entire population enrolled in the study Preliminary results from the unaudited site data indicate that in this specific sub-population, on the primary endpoint for the entire study, which was a two-point improvement in ordinal score at day 14, statistically significant results were obtained.

The next question comes from Eric Joseph J.P. Morgan. Please go ahead.

Hi, Good morning, guys and thanks for taking my question really appreciate all the.

Got a commercial color Onyx Bobo today.

I'm just wondering if you could comment on the latest trends in terms of.

Discontinuation in the myeloma.

Indication how that split between progression of disease.

Versus tolerability and whether you see much more headroom to the expansion of time on therapy.

From the current duration here 2.7 months.

Hi, John I'll take that.

Thanks to the question, Eric the discontinuation rate due to adverse events remains 13%. The same number we reported last quarter since our launch we attribute this in part due to the emphasis our commercial organization has had and discussing with physicians and their teams. The proactive measures that can be taken to prevent and mitigate some of exposure you have potential side effects.

Particularly the recommended supportive care guidelines in dose modifications included in the product label. We've also enhanced we also offer enhanced nurse service offerings to our specialty pharmacy partners to ensure patients have a positive experience with exposure.

Michael P. Mason: In addition, on day 28, improvement in ordinal score by two points also reached statistical significance with p = 0.05. And, very importantly, the rate of hospital discharge by day 14 was significantly higher with Selenexer as compared with placebo. In other words, patients treated with Selenexer stopped requiring supplemental oxygen more rapidly than those on placebo and could leave the hospital sooner. Finally, while all patients entering the study were required to have positive SARS-CoV-2 PCR results, conversion to SARS-CoV-2 PCR negative status was significantly higher on the Selenexor arm as compared with the placebo arm across the entire population. Fatalities were similar in the subpopulation mentioned above.

Additionally, we're finding that physicians as physicians gain more personal experience with xtremio, they're becoming much more familiar with how to best support keep their patients on therapy.

In terms of the duration of therapy.

We would expect that as.

Well give group of patients to continue to improve overtime as we would expect longer duration of therapy in the DLP CLL patients and then what the future indication pending FDA approval longer duration of therapy at earlier stages of myeloma.

Of course got it okay and just a housekeeping question here regarding the provided cash guidance can you talk little about what that anticipates in terms of exposure here sales in 2021. Thanks.

We're not going to give us specific numbers for 25 themselves, but obviously includes.

Michael P. Mason: While the rate of fatalities in the study was imbalanced in patients 75 years or older or with a COVID-gram high-risk score, the DSM-D considered that all deaths in the study were due to severe COVID-19 disease and or underlying comorbidities without a clear contribution of cell inactivity. Moving forward, and based on these data, we are following the DSMB recommendation to discontinue the current trial and plan to analyze the data to further characterize the specific subpopulation that will likely benefit from spell-inexecution. We plan to work with the FDA and other regulatory bodies to identify a path forward for future clinical development given these initial positive findings. Furthermore, we plan to engage with potential partners and seek external funding to advance future clinical development of COVID-19. Before turning the call over to Mike Mason, I do want to take a moment to sincerely thank all of the patients and investigators who participated in this study in support of our collective efforts to make a positive difference in the ongoing pandemic. I'll now turn the call over to Mike for a review of the financials. Mike?

The currently improved Collier approved indications and that obviously at some point if all goes all Boston approval.

Which we believe today's March twice.

Yes, Okay got it thanks for taking the questions guys.

The next question comes from Jonathan Chang with S. C. B lean Nick Please go ahead.

Good morning, and thanks for taking my questions. My first question can you provide any color on lucky outstanding questions and remind the during data are from the European regulators.

Yeah, I mean that they're just requested that all efficacy data the re monitored for all the responders and it's not an unreasonable request. The problem. We ran into what was in something that would have normally take and 48 weeks.

It was impossible during during the cobot problem because a lot of the sites were completely shut down was zero access at all.

Michael P. Mason: Thank you, Michael. Since we issued a press release earlier today with a full financial result, I'll just focus on the highlights, which began on slide 14. Net product revenue for the second quarter of 2020 was $18.6 million, and total revenue was $33.5 million. As previously mentioned, second quarter sales were driven by patient demand from academic and community-based positions with minimal channel inventory bills in the second quarter related to our DLVCL launch. The estimated growth to net discount for Expovio in the second quarter was approximately 16%.

Michael P. Mason: We continue to expect the growth in net discount to fall between 15 and 20% throughout the remainder of this year. Research and development expense for the second quarter of 2020 was $42.6 million, compared to $26.5 million for the second quarter of 2019. The increase in R&D expenses compared to the second quarter of 2019, Unknown Speaker, Unknown Attendee, Detlef Winckelmann, Reshma Rangwala, Elhan Webb, Sohanya Cheng, for the remainder of 2020 on a quarterly basis. For the second quarter of 2020, SG&A expense was $30.8 million compared to $24.7 million for the second quarter of 2019.

So we're just providing all efficacy data and any updated data that are available for those patients who responded to the drug and that will all be completed and submitted in September.

Got it thank you.

And second question for the Expo feel launch I give a sense whether things are trending up and should lie for instance June are there any metrics you can share with us today. Thank you.

Yes, I mean, I think what we would say as again, we tend not to.

Project monthly data, but certainly with the launch certainly there's a lot of positive momentum and were without doubt seeing prescriptions generated from the obvious from a de obviously all indications. So certainly we are seeing more patients coming into the system in July the June certainly by the fact that we now have the LTL patient.

I'm being prescribed exposure, but I would say in general terms, we continue to be pleased with the momentum.

That we're seeing based on April one do you have in Seattle, Boston data being presented and exposure being added to NCCN guidelines et cetera, et cetera, and we think that bad momentum will continue to build as we get out into the.

Michael P. Mason: The increase in SG&A expenses compared to the prior year was primarily due to activities to support the U.S. commercialization of Expovio and preparations for the launch of Expovio as a treatment for patients with prolapse or refractory DLVCO. Cash, cash equivalents, restricted cash, and investments as of June 30, 2020, total of $348.2 million compared to $265.8 million as of December 31, 2019. Based on our current operating plans, we expect our non-GAAP R&D and SG&A expenses, which exclude stock-based compensation expense, for the full year of 2020 to be in the previously projected range of $240 to $260 million. In addition, as shown on slide 15, we currently expect that our existing cash, cash equivalents, and investments and the revenue we expect to generate from Expovio product sales will be sufficient to fund our planned operations into the middle of I'll now turn the call back over to Michael for some concluding remarks. Thank you.

Second half of this year into early next year, which progression of publications medical meetings as well as hopefully an approval by the first quarter of next year.

The next question comes from Brian Abrahams with RBC capital markets. Please go ahead.

Hi, there congrats on all the progress and thanks for taking my questions.

I was wondering if you could talk a little bit more about.

Maybe how the FDA views on expose benefit risk profile, maybe evolving at all following the up the Boston presentation and submission now that randomized.

Detailed safety and efficacy data are available do you expect we could see label expansion even sooner than the standard review PDUFA in March and then also curious about the potential timeline an impact you might expect from getting on the NCCN guidelines. Thanks.

Sure I mean look we recognize we all recognize that the FDA inundated and they're under the same difficult working conditions at the rest of us or our end. So they have a lot on their play.

Michael P. Mason: Thank you, Mike. Before moving to the Q&A, let me highlight some of the key clinical and regulatory milestones that we expect for the remainder of 2020, as shown on slide 16. As you can see, we have done a terrific job so far in 2020, achieving many of the goals we set forth at the start of the year, but we have some important goals still to accomplish. First, we plan to submit additional STORM data and the full Boston data to EMA in the coming months. We hope to have greater clarity on the timing of future EMA regulatory decisions following feedback from the agency.

Myeloma with one to three prior therapies has always been a standard review time.

But we're hopeful that our rapid responsiveness to their information requests and there.

Strong knowledge about exposed the all in both myeloma deal Bcl will help facilitate review and of course, we're hopeful they can get it done sooner, but right now we had to stick with the new per day.

Remember that for the original approval for storm. They did look at the early data from Boston and those data, which we were not aware of have clearly borne out and.

Michael P. Mason: Next, we expect to share top-line Phase III data from this field study evaluating Selinexer and Leiblitzar columns. Assuming positive results on the primary endpoint of PFS, we plan to file subsequent regulatory submissions. And finally, we anticipate initiating a confirmatory phase 3 study of Expovio and DLVCL in support of our recent accelerated approach.

So I guess, there, they're probably gratified that they fall into the phase three trends back then and we think that they will they if they have now felt comfortable with their decision to get this drug not one but to accelerated approvals.

Michael P. Mason: And, of course, we will work diligently through the remainder of the year to support our SNDA based on the Boston study, as well as explore potential next steps for cell and gene therapy development in patients with severe COVID-19. As we look forward to the next several years, we will increasingly expand our activities in solid tumors, both as a single agent and in combination. These include uterine cancer with the ongoing Phase 3 C-endo study, glioblastoma multiforme, both alone and in combination, colon cancer, and lung cancer.

In terms of the.

The decode the NCCN situation now we've submitted to the NCCN.

And we're hopeful that we'll see a action from the NCCN sooner Interestingly, we submitted our deal Bcl data at approximately the same time and not immediately moved.

Onto the NCCN guidelines it really in myeloma, just dependent upon the myeloma group and NCCN get NCCN getting together rendering decision.

Michael P. Mason: We appreciate your ongoing support and look forward to keeping you updated on our 2020 progress. I'll now turn the call over to the operator for questions. Operator?

Really helpful. Thanks, a lot.

The next question comes from David Liebowitz with Morgan Stanley. Please go ahead.

Operator: We will now begin the question and answer session. To ask a question, please press star and then one on your telephone keypad. If you are using a speakerphone, please pick up your handset before pressing the key.

Oh would you be able to give us any thoughts on cadence of exposed to a sales going into the second half of the or.

John or Mike.

Operator: To withdraw your question, please press star then two. Please limit questions to one and one follow-up question. At this time, we will pause momentarily to assemble our roster. The first question comes from Maury Raycroft with Jeffreys. Please go ahead. Hi, good morning, everyone.

Yes, I mean, I think what you saw in Q2 was let's see.

18.6 million revenue, but 95% of that as we mentioned was this demand versus 5% was channel and we expect the losses continued strong.

And the mm sales in the second half of the or were you specific color.

Sorry numerically on the call.

Michael P. Mason: And thanks for taking my questions and congrats on the progress. I had a question about the COVID-19 study. So for the less than 75-year-old patients that are GRAM, non-high-risk patients, where you saw this benefit, can you go into more details on what you know so far about how other medications or treatments could have impacted results? And then it seems like this is the larger population of COVID-19 patients.

Yes, I think that.

And with respect to be upcoming the folks that comment data could you tell us I guess run us through what your expectation.

For that study is given.

What occurred in the phase two part of the trial.

And what your thoughts are on what that data might mean for the broader topic of solid tumors.

Michael P. Mason: Do you expect additional funding and support to come from an external pharma partner, or would it be from the government? Yeah, the second part is we're in ongoing discussions about all options in terms of next steps. Given all of the things on our own plate in oncology, we believe these strong proof-of-concept results will lead to some kind of partnership to make sure this moves along, but we don't, we will not be able to fund that. In terms of the risk factors for response, or, if you will, or better prognosis, we'll sell an excerpt. So far, we've not found that prior therapy really impacts outcomes in this population where there is a significant benefit.

Yeah I think this the importantly, this as a strategic it's not a shift I would say, but it's definitely a focus area that we'll be focusing considerably more of our resources in solid tumors and liposarcoma represents an extreme unmet medical need a relatively small population with differentiated liposarcoma, but unfortunately.

Pretty much uniformly fatal almond only scheme parental chemotherapy is our available there's no oral treatment there.

So demonstrating activity in these very large tumors that are chemo refractory. We think is that does an excellent precedent and an excellent first first potential approval for exposure in solid tumors. The phase two study as you may recall showed a hazard ratio just shy of 0.7, which is what the famous power to do.

And we hope to see a similar kind of result.

Michael P. Mason: In fact, what was, I think, more surprising was many of the patients we received, including in that particular group, had already gone through and were worsening on the available therapies. So we kind of got a relapsed or refractory population. But so far, there's no specific prior therapy that mattered. And that's somewhat consistent with the novel mechanism of Selinexer in this disease.

And if we do then we will be filing that both the U.S. and.

The regulatory bodies, and we think that facility and important draw for life, what sarcoma and our and also in general showing that exposure has activity not only in hematologic malignancies, but also in solid tumors, particularly chemo refractory tumors. Following that we have RC Endo study that should read out the.

John Mascarenhas: And maybe one quick question on the commercial update. You mentioned the new package sizes. Can you just go into any more specifics on how the DLBCL package size could impact multiple myeloma? And then for inventory, can you provide any more specifics on what you saw in inventory?

End of next year in uterine cancer and the adjuvant settings. So that's after first line completion of chemotherapy and then try to delay progression after chemotherapy in patients with advanced uterine cancer and we have additional trials ongoing in Glioblastoma Multiforme you. Both now in the front line in combination with radio.

One plus or minus Temozolomide and in the second line in combination with Alkylator therapy.

John Mascarenhas: Sure. We introduced three new package sizes at the end of this quarter to match the labeled dosing requirements for patients with DLV-CL. So we introduced 60 milligrams twice a week, 40 milligrams twice a week, and 40 milligrams once a week to address patient dosing within the context of the package insert. Of course, these package sizes could also be used if a doctor chose to use them in the context of multiple myeloma also, but those three package sizes were specifically introduced to address the need in DLV-CL.

We have additional studies that are also ongoing now both investigator and company sponsored in colorectal cancer and in lung cancer, and we hope to be sharing some additional data in other solid tumors towards the end of this year.

So long story that they did company, we'll be focusing to a large extent on solid tumors, while continuing to move expeditiously in both myeloma Dion deal Bcl.

Thank you for the uptick.

The next question comes from had point with H.C. Wainwright. Please go ahead.

Michael P. Mason: And I'll turn it over to Mike to address the inventory. Sure. So we reported 18.6 million in sales for the quarter.

Michael P. Mason: About 5% of that was inventory billed related to the DLV-CL launch because it got approved so late in June, and 95% was driven by patient demand. Great. Thanks, everyone.

Good morning, Thanks for taking my questions. So maybe just staying on solid tumors Michael.

If you can just give us a.

A little update on the Glioblastoma trial, you had the first patient enrolled in June.

Can you give us any further enrollment update or time into the phase one data and then also on the colorectal cancer lung cancer studies.

Michael P. Mason: The next question comes from Mike Olmst with Baird. Please go ahead. Hey guys, thanks for taking the question and congrats on the progress as well. Question on Expovio, I guess, just post-presentation of the Boston data at ASCO, maybe you can provide some color on the types of feedback you've gotten from physicians there, and then separately, have you noticed any increase in off-label use post-ASCO presentation? Yeah, thanks, Mike. Look, the feedback has really been uniformly very positive. I would say there's sort of several camps out there.

In Israel just are they progressing is has there been any impact on any of these studies.

For with the Cobot pandemic. Thank you.

Now I'll turn it to Sharon. So this study is progressing nicely on most of the arms that Michael just mentioned indeed.

In newly diagnosed and no first line as well as an inpatient.

As.

LNG BM.

Michael P. Mason: There were the people that always believed and were early adopters that were very gratified to see the results and said they were not surprised. There were the people that were somewhat on the fence and using the drug more cautiously who were quite excited and actually impressed with the drug. And then the group of people that were really skeptical and perhaps not using the drug yet, all sort of nodded and said, "It's time to learn how to use this drug." You can easily see some tweets for some folks that have sort of converted, and they are using the drug. It's very difficult to tell how the drug is being adopted immediately.

And we hope to be yet.

But then.

That is fantastic making.

Yes.

Okay. Thank you.

Again, if you have a question. Please press Star then one.

The next question comes from Arlinda Lee with Canaccord. Please go ahead.

Hi, Good morning, it's Ben Shim, calling in for Arlinda, Thanks for taking my questions.

Congrats on the strength of mix Toby ourselves and we really appreciate all the details guys provider sales metrics just a just two questions.

Michael P. Mason: We suspect that there may be increased use in combination, but in general, the use is in patients who've already gone through the available therapies and are in need of something new. The good news is that the once-weekly combination therapy does seem to be well-tolerated, and we do believe there's some increased use of that, but again, it's in the advanced refractory population. We have submitted to NCCN guidelines, and we're awaiting responses from the NCCN, and hopefully, we'll be able to see adoption of the Boston Regimen and earlier lines of therapy and, of course, the PDUFA date early next year. Okay, thank you.

Have you re thought or are you thinking about your EU commercialization strategy and oncology Q partner.

And I'll ask my follow up after that.

Chris for me I'll take that serve hey, Ben it can be hard to get satisfying incremental updates when it comes to the types of things, but as you know we continued George.

Numerous options for potential commercialization in Europe now that we have regained the development and commercial rights in Japan are we think those potential options for us have increased one option and probably the most likely one could involve a partnership.

And this scenario, we book for a partner ever at a partnership mutually beneficial for for both parties maximizing the potential of Selinexor in the European market.

John Mascarenhas: The next question comes from Peter Lawson with Barclays. Please go ahead. Hey, thanks for taking my question, maybe around. The impact of COVID and any of the dynamics you could talk through around new script growth, patient growth, and level of discounting would be much appreciated and around the corner. John, you want to?

We've said offered that we're looking for a partner who aligns with our philosophy regarding our long term development and commercialization plans for Celebrex or.

Has the expertise and marketing Hematological malignancy drugs, you and then obviously building towards a solid tumor presence as well in second option could be to potentially launch sold extra select for our own and select European countries, where reimbursement negotiations and decisions.

John Mascarenhas: Sure. There are two parts to that. First, I'll address the growth in new patient growth. We continue to add new patients each quarter, which is one of the key drivers of our sales, especially when you consider that some patients will also come off exfoliotherapy each quarter. We're encouraged by recent trends and new patient starts, but we don't break out our sales figures by new patient starts compared to existing patients. What we did see in Q2 was increasing total demand, as mentioned earlier, up 12%, even in the context of the pandemic. The increase was driven primarily by multiple myeloma, as the new DLVCO indication was approved at the end of the quarter. In terms of the impact of the COVID-19 pandemic on our commercial efforts, we believe the COVID-19 impact is having multiple effects. As reported by many other companies as well as secondary data sources, patient visits to their doctors continue to be lower than pre-pandemic levels.

We are occur more quickly and other acceptable levels, but in either scenario. Our belief is that the value of selinexor were built over time, particularly as we progress through the regulatory process in myeloma and you know just as an aside in myeloma, obviously, the most significant opportunity in Europe or com following.

Potential approval based on the data from a Boston study and I. Just also know that there are opportunities to leverage approvals in the U.S. for paid named patient program I'm in certain territories, where we wouldn't anticipate any impact to reference pricing. So we may also avail ourselves.

Both of these opportunities.

As well subject of course to the always evolving our global pricing and reimbursement landscape.

John Mascarenhas: In the context of this environment, we were pleased to be able to grow demand for Expovio in the quarter. However, we've also seen an impact on in-person visits by our sales force, nurse liaisons, and payer teams to be extremely limited. Based on secondary data and our own experience, we estimate only about 15% of HCPs can be seen in in-person visits.

Thanks.

Great that's very helpful.

Switching gears to covert 19, I guess some of this may touch upon potential partnering in oncology.

Will you be pursuing emergency use authorization use authorization pure and kind of wonder what the implications will be for pricing.

John Mascarenhas: We've also seen, of course, that ASCO was virtual this year, which limited the ability to have a number of engagements with academic and community-based oncologists. We are doing a number of things specifically to offset the impact of being virtual and continuing to grow Expovio back to the DLV-CL launch we planned on and executed a virtual launch. We've increased our level of non-personal promotion and digital content, including implementing virtual meeting technology for field force engagement, launching email and mail campaigns direct to professionals and through the field force, increasing search engine marketing spend for both HCP and patient audiences, increasing media placement, Launching New Websites, and Executing Expovio Education at the Point of Diagnosis in EMR Systems.

Both in a pandemic and post pandemic world. Thanks.

Yeah, I think I think both those questions in parts are a little premature I mean, our intention is to demonstrate prospectively that we have this what appears to be a significant benefit now in this specific sub population is important large sub population and once we get there. We'll we'll work with all of the different constituents to do the right.

This is not a time too.

Start playing around people people are suffering greatly and well do the right thing what we've got to demonstrate prospectively that this is correct.

And on and replicate the results and then we'll we'll move ahead and we don't believe this is a low dose of Selinexor as 20 milligrams given three times a week.

John Mascarenhas: So just some examples of things we're doing to offset the ongoing impact from the COVID-19 pandemic. And then, did you get any sense of where Steli is being used in DOBCL and multiple myeloma? What lines? So, as Michael just mentioned before, we see the most use of Expovio in the context of the indicated lines.

I don't believe this will have implications for the.

Calls you franchise.

Okay. Thanks, very much for the color and good luck you guys.

Thank you thanks Ben.

This concludes our question and answer session.

I would like to turn the conference back over to C O Michael Kauffman for any closing remarks.

Thanks, very much and thank you all for joining todays call. We look forward to updating you on our progress in the future best.

John Mascarenhas: So, in the context of multiple myeloma, most of the use is in that petrorefractory setting where indicated. And, while we just have limited data given the early launch of DLVCL, we see it being used there and after two or more lines of therapy where it's indicated as well. Great. Thank you so much.

The conference has now concluded. Thank you for attending today's presentation you may now disconnect.

John Mascarenhas: Thanks for taking the question. Thanks for your questions, Peter. The next question comes from Eric Joseph with JP Morgan. Please go ahead. Hi, good morning, guys.

John Mascarenhas: Thanks for taking the questions and I really appreciate all the added commercial color on Expovio today. I'm just wondering if you could comment on the latest trends in terms of discontinuation in the myeloma indication, how that splits between progression of disease versus tolerability, and whether you see much more headroom for expansion of time on therapy from the current duration here at 2.7 months. All right, John, I'll take that.

John Mascarenhas: Thanks for the question, Eric. The discontinuation rate due to adverse events remains 13%, the same number we reported last quarter since our launch. We attribute this in part to the emphasis our commercial organization has had on discussing with physicians and their teams the proactive measures that can be taken to prevent and mitigate some of Expovio's potential side effects, particularly the recommended supportive care guidelines and dose modification included in the product label. We also offer enhanced nurse service offerings to our specialty pharmacy partners to ensure patients have a positive experience with Expovio. And additionally, we're finding that as physicians gain more personal experience with Expovio, they're becoming much more familiar with how to best support and keep their patients on therapy. In terms of the duration of therapy, we would expect that as a collective group of patients to continue to improve over time, as we would expect longer duration of therapy in DLBCL patients. And then, with the future indication pending FDA approval, longer duration of therapy in earlier stages of myeloma. Of course, I got it.

Michael P. Mason: Okay, and just a housekeeping question here regarding the cash guidance provided. Can you talk a little bit about what that anticipates in terms of exposed US dollars in 2021? Um, you know, we're not going to give a specific number for 2021 sales, but it obviously includes, you know, the currently improved, currently approved indications. And then obviously, at some point, it puzzles all the Boston approvals, which we, you know, produced with AIDS in March of 2012. Yeah, okay. I got it. Thanks for taking the question, guys. The next question comes from Jonathan Chang with SVB LEMIC.

Michael P. Mason: Please go ahead. Good morning, and thanks for taking my question. First question, can you provide any color on what the outstanding questions and re-monitoring data are from the European regulators? Yeah, I mean, they just requested that all efficacy data be remonitored for all the responders, and it's not an unreasonable request. The problem we ran into was that something that would normally take four to eight weeks was impossible during the COVID problem because a lot of the sites were completely shut down with zero access at all.

Michael P. Mason: So we're just providing all efficacy data and any updated data that are available for those patients who responded to the drug, and that will all be completed and submitted in September. Got it, thank you. And second question, for the Expovial launch, do you have a sense of whether things were trending up in July versus June? Are there any metrics you can share with us today? Thank you. Yeah, I mean, I think what we would say is, again, we tend not to project monthly data, but certainly, with the law, there's certainly a lot of positive momentum, and we're, without doubt, seeing prescriptions generated from the DLVCL indication. So certainly, we are seeing more patients coming into the system in July than June, certainly because of the fact that we now have DLVCL patients being prescribed Expovio.

Michael P. Mason: But I would say in general terms, we continue to be pleased with the momentum that we're seeing based on, you know, the approval in DLVCL, the Boston data being presented, Expovio being added to NCCN guidelines, et cetera, et cetera, and we think that that momentum will continue to build as we get out into the second half of this year and early next year with the progression of publications, medical meetings, as well as hopefully an approval by the first quarter of next year. The next question comes from Brian Abrahams with RBC Capital Markets. Please go ahead. Hi there.

Michael P. Mason: Congratulations on all the progress and thanks for taking my questions. I was wondering if you could talk a little bit more about, you know, maybe how the FDA's views on Expovio's benefit-risk profile may be evolving at all following the Boston presentation and submission, now that randomized detailed safety and efficacy data are available. Do you expect we could see label expansion even sooner than the standard review PDUFA in March? And I'm also curious about the potential timeline and impact you might expect from getting on the NCCN guidelines. Thanks.

Michael P. Mason: Sure. I mean, look, we all recognize that the FDA is inundated, and they're under the same difficult working conditions that the rest of us are in. So they have a lot on their plate.

Michael P. Mason: Myeloma with one to three prior therapies has always been a standard review time, but we're hopeful that our rapid responsiveness to their information requests and their strong knowledge about Expovio in both myeloma and DLBCO will help facilitate the review. And, of course, we're hopeful they can get it done sooner. But right now, we have to stick with the PDUFA date. Remember that for the original approval of STORM, they did look at early data from Boston, and those data, which we were not aware of, have clearly borne out. And so I guess they're probably gratified that they followed the phase three trends back then.

Michael P. Mason: And, you know, we think that they have now felt comfortable with their decision to give this drug not one but two accelerated approvals. In terms of the, to code, the NCCN situation. You know, we've submitted to the NCCN, and we're hopeful that we'll see action from the NCCN sooner. Interestingly, we submitted our DLBCL data at approximately the same time, and that immediately moved onto the NCCN guidelines. It really is, in myeloma, just dependent upon the myeloma group and NCCN getting together and rendering a decision. Really helpful. Thanks a lot.

Michael P. Mason: The next question comes from David Leibowitz with Morgan Stanley. Please go ahead. Would you be able to give us any thoughts on the pace of Expovio sales going into the second half of the year? John, Mike.

Michael P. Mason: Yeah, I mean, I think what you saw in Q2 with the 18.6 million in revenue, but 95% of that, as we mentioned, was demand versus 5% was channel. And we expect the launch to continue strong and MM sales in the second half of the year, but we're not going to give specific color, first numerically on the call. Unknown Speaker.

Michael P. Mason: And with respect to the upcoming liposarcoma data, could you tell us, I guess, run us through what your expectation for that study is given what occurred in the phase two part of the trial and what your thoughts are on what that data might mean for the broader topic of solid tumors. Yeah, I think this is, importantly, this is a strategic, not a shift, I would say, but it's definitely a focus area that we And liposarcoma represents an extreme unmet medical need, a relatively small population with deep differentiated liposarcoma, but unfortunately, pretty much uniformly fatal. And only parenteral chemotherapies are available; there's no oral treatments there. So demonstrating activity in these very large tumors that are chemorefractory, we think is an excellent precedent and an excellent first potential approval for expovio in solid tumors.

Michael P. Mason: The phase two studies, you may recall, showed a hazard ratio just shy of 0.7, which is what phase three was powered to do. And we hope to see a similar kind of result. And if we do, then we will be filing that for approval in both the U.S. and... other regulatory bodies. And we think that this will be an important drug for life with sarcoma and also, in general, showing that Expovio has activity not only in hematologic malignancies but also in solid tumors, particularly chemorefractory tumors.

Michael P. Mason: Following that, we have our Siendo study that should read out at the end of next year in uterine cancer in the adjuvant setting, so that's after first line, completion of chemotherapy, and then to try to delay progression after chemotherapy in patients with advanced uterine cancer. And we have additional trials ongoing in glioblastoma and multiforme, both now in the front line in combination with radiation, plus or minus temozolomide, and in the second line in combination with alkylated therapy. We have additional studies that are also ongoing now, both investigator-sponsored in colorectal cancer and in lung cancer, and we hope to be sharing some additional data in other solid tumors towards the end of this year. So the long story is that the company will be focusing to a large extent on solid tumors while continuing to move expeditiously in both myeloma and DLV-CL. Thank you for the update. The next question comes from Ed White with HC Wainwright. Please go ahead. Good morning.

Michael P. Mason: Thanks for taking my questions. So maybe just staying on solid tumors, Michael, if you could just give us a little update on the glioblastoma trial. You had the first patient enrolled in June.

Unknown Attendee: Can you give us any further enrollment update or timing for the Phase I data? And then also on the colorectal cancer and lung cancer studies in Israel, just how are they progressing? Has there been any impact on any of these studies with the COVID pandemic?

Michael P. Mason: Thank you. Yeah, I'll turn it up to Sharon. So the study is for working nicely on all three arms that Michael just mentioned in the... For the newly diagnosed and first line as well as an inpatient that has a current UDM.

Unknown Attendee: And we hope to be able to present the results of this study at scientific meetings. Okay, thank you. Again, if you have a question, please press star and then one. The next question comes from Arlinda Lee with Canaccord. Please go ahead. Hey, good morning. It's Ben Shim calling in for our Linda.

Christopher Firmiano: Thanks for taking my question. Congratulations on the strength of expobia cells. And we really appreciate all the details that you guys provide on the cells. Just two questions. Have you rethought, or are you thinking about your EU commercialization strategy and oncology, do you think you've part... And I'll ask my follow-up. Chris Permian, I'll take that.

Christopher Firmiano: Sir, hey Ben, you know, it can be hard to get satisfying incremental updates when it comes to these types of things, but as you know, we continue to assess numerous options for potential commercialization in Europe. Now that we've regained the development of commercial rights in Japan, we think those potential options for us have increased. One option, and probably the most likely one, could involve a partnership.

Christopher Firmiano: In this scenario, we look for a partner and a partnership that is mutually beneficial for both parties, maximizing the potential of Selenexor in the European market. You know, we've said often that we're looking for a partner who aligns with our philosophy regarding our long-term development and commercialization plans for Selenexor and has the expertise in marketing hematologic malignancy drugs in the EU, and then, obviously, building towards You know, a second option could be to potentially launch Selenexor on our own in select European countries, where reimbursement negotiations and decisions typically occur more quickly and at accessible levels. But in either scenario, our belief is that the value of Selenexor will build over time, particularly as we progress through the regulatory process in myeloma.

Christopher Firmiano: And, you know, just as an aside, in myeloma, obviously, the most significant opportunity in Europe will come following a potential approval based on the data from the Boston study. And I just also note that there are opportunities to leverage approvals in the U.S. for paid inpatient programs in certain territories where we wouldn't anticipate any impact on reference pricing. So we may also avail ourselves of these opportunities as well, subject, of course, to the always evolving global pricing and reimbursement landscape. Great, that's very helpful. Switching gears to COVID-19, I guess some of this may touch upon potential partnership in oncology. Will you be pursuing emergency use authorization here? And I kind of wonder what the implications will be for pricing, both in a pandemic and a post-pandemic world. Yeah, I think I think both those questions are, in part, a little premature. I mean, our intention is to demonstrate prospectively that we have this what appears to be a significant benefit in this specific subpopulation. Now, in this specific subpopulation, it's an important large subpopulation.

Michael P. Mason: And once we get there, we'll work with all of the different constituents to do the right thing. And this is not a time to just start playing around with people. People are suffering greatly, and we'll do the right thing. But we've got to demonstrate prospectively that this is correct and replicate the results. And then we'll move ahead. And I don't believe this is a low dose of Selenexer. It's 20 milligrams given three times a week.

Michael P. Mason: We don't believe this will have implications for the oncology franchise. Okay, thanks very much for the color, and good luck to you guys. Thank you. Thanks, Ben. This concludes our question and answer session. I would like to turn the conference back over to CEO Michael Kauffman for any closing remarks. Thanks very much, and thank you all for joining today's call. We look forward to updating you on our progress in the future. Best. The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.