Karyopharm Therapeutics (NASDAQ:KPTI) Q2 2020 Earnings Call

0:00 Good morning. My name is Debbie, and I will be your conference operator today. 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, Kerrio Farms, Senior Vice President, Investor and Public Relations. Please go ahead.

0:38 Thank you, Debbie, and thank you all for joining us on today's conference call to discuss Kerrio Farms' 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 Desmarais, Chief Commercial Officer, and Mr. Christopher Firminano, 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 second quarter 2020 financial results. We will conclude with the Q&A portion of the call. 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, are available on our website at carrierpharm.com.

1:27 Before we begin our formal comments, I'll remind you that various remarks we will make today constitute forward-looking statements for purposes of the safe harbor provisions under the private securities litigation reform act of 1995. These include statements about our future expectations, clinical developments, and regulatory matters and timelines, the potential success of our 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 factor 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.

2:13 We specifically disclaim any obligation to do so, even if our views change. Therefore, we 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.

2:36 Thank you, Ian, and good morning, everyone. Before I specifically review Karyopharm's performance in the second quarter, I'd like to put into some context where Karyopharm is on its journey to becoming a leading global oncology-focused pharmaceutical company. When Dr. Sharon Shacham 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 and growing the body of scientific and clinical evidence around XPO1 inhibition, leading to the approval of the first-in-class oral compound, Expobio. and in July of last year, we officially transitioned to a commercial company with the initial FDA approval and launch of Expobio and relapsed or refractory multiple myeloma.

3:26 This year, we achieved an additional important milestone on our journey as Expobio received its second FDA approval at the end of June and became the first branded therapy to be approved in both myeloma and DLBCL. 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 the patients and families in need of new treatment options for devastating cancers. Please now turn to slide five. 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.

4:16 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 physicians. Importantly, we saw more than 170 new physician accounts prescribe Expovio for the first time in a quarter Thank you. Thank you. 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 3 Boston study in patients with previously treated myeloma.

5:25 Boston is our randomized trial of once-weekly Selenexer with once-weekly Velcade along with dexamethasone, and Dexamethasone. 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, or PDUFA. I'm also pleased to announce that we have now completed enrollment in our Phase 3 study of Selenixer in patients with heavily pre-treated, de-differentiated liposarcoma and expect top-line data in the second half of this year.

6:10 If these data are positive, we believe this will be a very important step towards our goal of bringing Expovio to patients battling solid tumors, in addition to those with hematologic 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 2 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 Keriopharm and rapidly advanced into the clinic in a matter of weeks due to the encouraging antiviral and anti-inflammatory activity of Expobio 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.

7:03 At the planned interim analysis of the randomized Phase II study with approximately 120 patients, the results indicated that Selenoxir is unlikely to demonstrate a statistically significant efficacy benefit across the entire population. 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 Selinexa for patients with COVID-19 will focus on a subpopulation where we saw the significant effects. I'll provide some additional details on these specific results later in today's presentation.

7:53 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 Expobio in various oncology indications, which we hope yields 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 that we expect will be sufficient to fund our planned operations into the middle of 2022. Please now turn to slide six, where I'll ask John Demery, our Chief Commercial Officer, to provide some additional details on Expobio sales performance during the second quarter. Thank you, Michael, and good morning, everyone.

8:38 As Michael noted earlier, we were pleased with the commercial momentum seen during the quarter, despite being impacted by some of the challenges associated from the COVID-19 pandemic, including limited in-person sales visits to our customers. I commend the Caryopharm 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 DLVCL include digital tools to facilitate continued sales force, nurse liaison, and payer team engagement with customers, search engine marketing and media placement, and virtual peer-to-peer programs. 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 therapies.

9:40 As of June 30th, approximately 3,200 Expobio 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. 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 US 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 Expobio. There was also some incremental added, some incremental inventory Thank you for joining us.

11:00 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 Exposio 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. 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 Exposio became commercially available, roughly 42% of patients were going on to get a second prescription.

11:57 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. We will continue to fully leverage the newly approved indication in DLVCL to access our key stakeholders, educate on this new indication, and also generate increasing excitement in multiple myeloma to help more patients impacted by these diseases.

12:46 Now I'd like to turn the call back over to Michael to discuss our key clinical development activities and priorities, which begin on slide eight. Michael?

12:54 Thank you, John. A critical milestone for us in the second quarter included 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 Velcade. 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 a 4.47-month improvement. The median progression-free survival in the SVD arm was 13.93 months compared to 9.46 months in the VD arm with a hazard ratio of 0.70.

13:42 SVD was superior to VD across other key efficacy endpoints such as overall response rate, 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 cytogenetics. 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 arm. We were also pleased to report the rate of peripheral neuropathy on the SVD arm was significantly lower than the rate in the VD control arm. Remember that peripheral neuropathy is among the most common causes of treatment limitation and discontinuation of VD and combination VD regimens and a significant differentiator for the Boston regimen pending future regulatory approval.

14:40 The data also showed no new safety signals in the SVD arm relative to previously reported adverse events from other cell and 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 a proteasome inhibitor, once weekly SVD offers patients an effective, convenient triplet regimen requiring approximately 40% fewer clinic visits and a reduced rate of peripheral neuropathy pending future regulatory approval, of course. 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 19th, 2021. 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. Exact timing of potential regulatory decisions based on both the STORM and Boston data will be determined following feedback from EMA.

15:49 As we turn to slide 10, I will highlight our recent accelerated approval for ExFovio for the treatment of adult patients with relapsed or refractory DLBCL. In the SADL study, which served as the basis for approval, 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, GCB 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-therapy option for patients with recurrent DLBCL. While we have just kicked off our commercial launch in this indication, we believe Expobio'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.

16:51 The simple administration, single agent, and novel mechanism of action of Expobio provides additional advantages for use with a manageable safety profile. Next, please turn to slide 12, where I will view the updates from our COVID-19 study. Based on anti-SARS-coronavirus activity in vitro and anti-inflammatory activity in vitro and in vivo, Keriopharm began enrollment in a placebo-controlled randomized phase 2 study to evaluate low-dose oral selenexer in hospitalized patients with severe COVID-19 in April of this year. These patients require supplemental oxygen and are at high risk for clinical worsening. Many of them had already received remdesivir, steroids, convalescent plasma, and other agents used to treat COVID-19.

17:36 As mentioned previously, the Data Safety Monitoring Board for the study concluded that the trial is 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 unauded site data indicate that in this specific subpopulation, 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.

18:25 In addition, on day 28, Improvement in ordinal score by two points also reached statistical significance with P as less than or equal to 0.05. And very importantly, the rate of hospital discharge by day 14 was significantly higher with Selinexer as compared with placebo. In other words, patients treated with Selinexer 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 Selinexer arm as compared with the placebo arm across the entire population.

19:11 Fatalities were similar in the subpopulation mentioned above. While the rate of fatalities in the study were imbalanced in the patient 75 years or older or with a COVID-gram high-risk score, The DSMB considered that all deaths on the study were due to severe COVID-19 disease and or underlying comorbidities without a clear contribution of Selinexer. 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 Selinexer. 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.

19:58 Furthermore, we plan to engage with potential partners and seek external funding to advance future clinical development in 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? Thank you, Michael.

20:23 Since we issued a press release earlier today with the 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, and total revenue was $33.5 million. As previously mentioned, second quarter sales were driven by patient demand from academic and community-based physicians, with minimal channel inventory bills in the second quarter related to our DLBCL launch in late June. The estimated gross to net discount for Expovio in the second quarter was approximately 16%. We continue to expect the 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 million for the second quarter of 2019.

21:16 The increase in R&D expenses compared to the second quarter of 2019 was mainly due to the rapid initiation and enrollment in our COVID-19 study, which, as discussed, 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, SG&A expense was $30.8 million compared to $24.7 million for the second quarter of 2019. The increase in SG&A expenses compared to the prior year was primarily due to activities to support the U.S. commercialization of Expobio and preparations for the launch of Expobio as a treatment for patients with relapse or refractory DLBCL. Cash, cash equivalents, restricted cash and investments as of June 30, 2020, totaled $348.2 million compared to $265.8 million as of December 31, 2019.

22:05 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 $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 from Expovio product sales, will be sufficient to fund our plant operations into the middle of 2022, which puts us in a terrific position to further advance are strategic aspirations. I'll now turn the call back over to Michael for some concluding remarks.

22:49 Michael. 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 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. Next, we expect to share top-line Phase III data from the SEAL study evaluating Selenexor and liposarcoma. Assuming positive results on the primary endpoint of PFS, we plan to file subsequent regulatory submissions. And finally, we anticipate initiating a confirmatory Phase III study of Expobio and DLBCL in support of our recent accelerated approval.

23:43 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 x-ray 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 III Siendo study, Glioblastoma multiforme, both alone and in 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? We will now begin the question and answer session.

24:28 To ask a question, please press star then 1 on your telephone keypad. If you are using a speakerphone, please pick up your handset before pressing the keys. To withdraw your question, please press star then two. 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 Raycroft with Jefferies. Please go ahead.

25:04 Hi, good morning everyone 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. Do you expect additional funding and support to come from an external pharma partner or would it be from governments?

25:44 Yeah, the second part is we're ongoing and expect additional discussions for 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 a partnership to make sure this moves along, but we will not be able to fund that. In terms of the risk factors for response, or if you will, or better prognosis with SELEN-X, so far we've not found that prior therapy really impacts the outcomes in this population where there was Thank you for joining us.

26:54 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, if you can provide any more specifics on what you saw in inventory this quarter.

27:12 Sure. We introduced three new package sizes at the end of this quarter to match the label dosing requirements for patients with DLBCL. So we introduced a 60 milligram twice a week, a 40 milligram twice a week, and a 40 milligram once per week to address the patient dosing within the context of the package insert. 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 DLBCL. I'll turn it over to Mike to address the inventory. Sure.

27:52 As we said, we reported $18.6 million in sales for the quarter. About 5% of that was inventory billed related to the DLBCL launch because it got approved so late in June, and 95% was driven by patient demand.

28:08 Great. Thanks, everyone. The next question comes from Mike Ulz with Baird. Please go ahead.

28:19 Hey, guys. Thanks for taking the question, and congrats on the progress as well. Just a 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? Thanks. Yeah.

28:43 Thanks, Mike. Look, the feedback has really been uniformly very positive. I'd say there's sort of the several camps out there. 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 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 and you can either It's very difficult to tell how the drug is being adopted immediately. We suspect that there may be increased use in combination, but in general, the use is in the patients who've already gone through the available therapies and are in need of something new.

29:40 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 Regiment and early lines of therapy and, of course, the PDUFA date early next year.

30:07 Okay. Thank you. The next question comes from Peter Lawson with Barclays. Please go ahead.

30:18 Hey, thanks for taking my questions. Maybe around kind of 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 around the quarter.

30:38 John, you want to take that?

30:40 Sure. Two parts of that. First, I'll address the 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 expoviotherapy each quarter. We're encouraged by recent trends and new patient starts, but we don't break out our sales figures by the 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 full, even in the context of the pandemic. The increase was driven primarily by multiple myeloma as the new DLVCL 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.

31:29 As reported by many other companies as well on 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 grow demand for Expobio in the quarter. We've also seen as an impact 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 HTPs can be seen in in-person visits. We've also seen, of course, 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. In fact, for the DLBCL 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 engagements, launching email and mail campaigns direct to professional and through the field force, increasing search engine marketing spend for both HCP and patient audiences, increasing media placements, launching new websites, and executing Expobio education at the point of diagnosis in EMR systems. So just some examples of things we're doing to offset the ongoing impact from the COVID-19 pandemic.

32:51 Thank you. And then did you get any sense of where STELI has been used in DOBCL and multiple myeloma? What lines?

33:04 John? So as Michael just mentioned before, we see the most use of Expobio in the context of the indicated lines. So in the context of multiple myeloma, most of the uses in that pentarefractory setting were indicated. And while we just have limited data given the early launch in DLVCL, we see it being used there and after two or more lines of therapy where it's indicated as well.

33:31 Great. Thank you so much. Thanks for taking the questions.

33:34 Thanks for your questions, Peter. The next question comes from Eric Joseph with JP Morgan. Please go ahead.

33:44 Hi, good morning, guys. Thanks for taking the questions and really appreciate all the 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 to expansion of time on therapy from the current duration here at 2.7 months.

34:15 All right, John, I'll take that. 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 due to the emphasis our commercial organization has had in 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 modifications included in the product label. We also offer enhanced 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 the DLBCL patients, and then with the future indication pending FDA approval, longer duration of therapy in earlier stages of myeloma.

35:29 Of course, got it. Okay, and just a housekeeping question here regarding the provided cash guidance. Can you talk a little bit about what that anticipates in terms of exposure sales in 2021? Thanks.

35:37 You know, we're not going to give a specific number for 2021 sales, but, you know, obviously includes, you know, the currently approved indications, and then obviously at some point it follows all Boston approval, you know, which we, you know, producer dates March 2021.

35:59 Yeah, okay, got it. Thanks for taking the questions, guys. The next question comes from Jonathan Chang with SVB-Linic. Please go ahead.

36:12 Good morning, and thanks for taking my questions. First question, can you provide any color on what the outstanding questions and remonitoring data are from the European regulators?

36:27 Yeah, I mean, they were 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 in something that would have normally taken 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. 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.

37:04 Got it. Thank you. And second question, for the ExpoVio 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.

37:21 Yeah, I mean, I think what we would say is, again, we tend not to project monthly data, but certainly there's a lot of positive momentum, and we're without doubt seeing prescriptions generated from a DLBCL indication. So certainly we are seeing more patients coming into the system in July than June, certainly by the fact that we now have DLBCL patients being prescribed Expobio. But I would say in general terms, We continue to be pleased with the momentum that we're seeing based on the approval in the LBCL, the Boston data being presented, and Expoio 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 into early next year with the progression of publications, medical meetings, as well as hopefully an approval by the first quarter of next year.

38:27 The next question comes from Brian Abrahams with RBC Capital Markets. Please go ahead.

38:35 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, 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 then I'm also curious about the potential timeline and impact you might expect from getting on the NCCN guidelines. Thanks.

39:10 Sure. I mean, look, we recognize, 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. Myeloma with one to three prior therapies has but we're hopeful that our rapid responsiveness to their information requests and their strong knowledge about Expobio in both myeloma and DLBCL 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 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 so I guess they're probably gratified that they followed the phase three trends back then and we think that they've now felt comfortable with their decision to give this drug not one but two accelerated approvals.

40:12 In terms of the code, the NCCN situation, we've submitted to the NCCN and we're hopeful that we'll see The next question comes from David Liebowitz with Morgan Stanley. Please go ahead.

40:52 Would you be able to give us any thoughts on cadence of ExpoVO sales going into the second half of the year? John?

41:03 Mike? Yeah, I mean, I think what you saw in Q2 with the 18.6 million of revenue, about 95% of that, as we mentioned, was demand versus 5% was channel. And we expect the launch to continue strong. and the MM sales in the second half of the year. But we're not going to give specific color, you know, as far as numerically on the call.

41:34 And with respect to the upcoming liposucoma data, could you tell us, I guess, run us through what your expectation for that study is given what occurred in the Phase II part of the trial? and what your thoughts are on what that data might mean for the broader topic of solid tumors.

41:59 Yeah, I think importantly this is a strategic, not a shift I would say, but it's definitely a focus area that we'll be focusing considerably more of our research Thank you very much. The Phase 2 study, as you may recall, showed a hazard ratio just shy of 0.7, which is what the Phase 3 was powered to do.

42:46 And we hope to see a similar kind of result. And if we do, then we will be filing that to both the U.S. and other regulatory bodies. And we think that this will be an important drug for liposarcoma and also, in general, showing that Expovio has activity not only in hematologic malignancies, but also in solid tumors, particularly chemorefractory tumors. Following that, we have our Ciendo study that should read out 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 multiforme both now in the front line in combination with radiation plus or minus temozolomide and in the second line in combination with alkylator therapy.

43:42 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 the company will be focusing to a large extent on solid tumors while continuing to move expeditiously in both myeloma and DLVCL.

44:09 Thank you for the update. The next question comes from Ed White with HC Wainwright. Please go ahead.

44:21 Good morning. Thanks for taking my questions. So maybe just staying on solid tumors, Michael. If you can just give us a little update on the glioblastoma trial. You had the first patient enrolled in June. Can you give us any further enrollment update or timing to the phase one data? And then also on the colorectal cancer and lung cancer studies in Israel, just are they progressing? Has there been any impact on any of these studies with the COVID pandemic? Thank you.

44:56 Yeah, I'll turn that to Sharon.

44:58 So the study is progressing nicely on all three arms that Michael just mentioned in the newly diagnosed first line as well as in patients that have current GBMs. And we hope to be able to present the results from this study at scientific meetings next year.

45:28 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.

45:44 Hey, good morning. It's Ben Shim calling in for Arlinda. Thanks for taking my questions. Congrats on the strength of Expovia sales, and we really appreciate all the details that you guys provide on the sales metrics. Just two questions. Have you rethought or are you thinking about your EU commercialization strategy and oncology partner? And I'll ask my follow-up after that.

46:10 Chris Permian, I'll take that. Sir, hey, Ben. You know, it can be hard to get satisfying incremental updates when it comes to these types of things. But, you know, 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. In this scenario, we'd look for a partner and a partnership that is mutually beneficial for both parties, maximizing the potential of Selinex in the European market. 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 Selinexor and has the expertise in marketing hematologic malignancy drugs in the EU and then obviously building towards a solid tumor presence as well. A second option could be to potentially launch Selinexor on our own in select European countries where reimbursement negotiations and decisions typically occur more quickly and at accessible levels.

47:19 But in either scenario, our belief is that the value of Selinexor will build 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 will come following a potential approval based on the data from the Boston study. And I just also know that there are opportunities to leverage approvals in the U.S. for paid name patient programs in certain territories where we wouldn't anticipate any impact to 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.

48:08 Great. That's very helpful. Switching gears to COVID-19, I guess some of this may touch upon potential partnering 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. Thanks.

48:31 Yeah, I think both those questions and 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 subpopulation, this important large subpopulation. 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 This concludes our question and answer session. I would like to turn the conference back over to CEO Michael Kaufman for any closing remarks. Thanks very much and thank you all for joining today's call.

49:36 We look forward to updating you on our progress in the future. Best. The conference is now concluded. Thank you for attending today's presentation. You may now disconnect.