Q1 2021 Karyopharm Therapeutics Inc Earnings Call
Good morning, My name is Jason and I'll be your conference operator today.
Jason: My name is Jason, and I will be your conference operator today. At this time, I'd like to welcome everyone to the Karyopharm Therapeutics first quarter 2021 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 in Public Relations.
At this time I would like to welcome everyone to the carrier farm Therapeutics first quarter 2021 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 carrier farms, Senior Vice President Investor and public relations.
Great. Thank you and thank you all for joining us on today's conference call to discuss to discuss carrier forums first quarter financial results and business update and let me be the first one to officially welcome our new President and Chief Executive Officer, Mr. Richard Paulson to our quarterly earnings call. This is Ian Karp and today. In addition to the bulk Michael Kauffman and Richard Paulson.
Ian Karp: Thank you. And thank you all for joining us on today's conference call to discuss Karyopharm's first quarter financial results and business update. And let me be the first one to officially welcome our new President and Chief Executive Officer, Mr. Richard Paulson, to our quarterly earnings call. This is Ian Karp.
Ian Karp: And today, in addition to both Michael Kaufman and Richard Paulson, I'm also joined by Mr. Mike Mason, our Chief Financial Officer, Mr. John Demery, our Chief Commercial Officer, and Mr. Stephen Michener, our Chief Business Officer. On the call today, Michael will provide an overview of key recent corporate developments and an update on our commercial progress, followed by an update on one of our key pipeline opportunities in endometrial cancer. Mike Mason will then provide an overview of the first quarter financial results.
I'm also joined by Mr. Mike Mason, our Chief Financial Officer, Mr. John Demaree, Chief Commercial Officer, and Mr. Stephen Michener, Chief business Officer.
On the call today, Michael will provide an overview of key recent corporate developments and an update on our commercial progress followed by an update on one of our key pipeline opportunities in endometrial cancer. Mike Mason will then provide an overview of the first quarter financial results. We will conclude with some thoughts from Richard on carry forms future and then we will move to the Q&A portion of the.
Ian Karp: We will conclude with some thoughts from Richard on Karyopharm's future, and then we will move to the Q&A portion of the call. Earlier this morning, we issued a press release detailing Karyopharm's results for the first quarter of 2021 and the appointment of Richard Paulson as our next president and CEO. These releases, along with a slide presentation that we plan to reference on today's call, are available on our website at karyopharm.com. Before we begin our formal statements, I'll remind you that various remarks we make today constitute forward-looking statements for purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995 and as outlined on slide number three.
A call.
Earlier. This morning, we issued a press release detailing carrier farm's results for the first quarter of 2021 and the appointment of Richard Paulson as our next president and CEO.
These releases along with a slide presentation that we plan to reference on today's call are available on our website at carrier form Dot com before we begin our formal statements I'll remind you that various remarks, we make today constitute forward looking statements for purposes of the Safe Harbor provisions under the private Securities Litigation Reform Act of 1995 and as outlined on slide number.
Three these include statements about our future expectations clinical development and regulatory matters and timelines the potential success of our products and product candidates, including our expectations related to the commercialization of ex Bovino index, Bobbio financial projections, and our plans and prospects actual results may differ materially from those indicated by these forward looking statements as a result of.
Ian Karp: These include statements about our future expectations, clinical development, regulatory matters, and timelines, the potential success of our products and product candidates, including our expectations related to the commercialization of Expovio and Nexpovio, 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 annual report on Form 10-K, which is on file with the SEC, and in other filings we may make with the SEC in the future.
Various important factors, including those discussed in the risk factors section of our most recent annual report on form 10-K, which is on file with the FCC and in other filings we may make with the SEC in the future any forward looking statements represent our views as of today only while we may elect to update these forward looking statements at some point in the future, we specifically disclaim any obligation to do so.
Ian Karp: Any forward-looking statements represent our views as of today only, and while we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change. Therefore, you should not rely on these forward-looking statements as representing our views as of any date subsequent to today.
So even if our views change therefore, you should not rely on these forward looking statements as representing our views as of any date subsequent to today I'll now turn the call over to Dr. Michael Kauffman co founder of carry a farm and our first CEO and now please turn to slide number four.
Michael P. Mason: I'll now turn the call over to Dr. Michael Kaufman, co-founder of Karyopharm and our first CEO, and now please turn to slide number four. Thank you, Ian, and good morning, everyone. Let me begin first by saying how thrilled I am to also be joined here today by Richard Paulson, our company's new CEO, and we'll begin on slide four. When Dr. Sharon Chatham and I started Karyopharm over 12 years ago, our mission was really quite simple.
Thank you Ian and good morning, everyone. Let me begin first by saying how thrilled I am also joined here today by Richard Paul.
Company's new CEO, who will begin on slide four.
And Dr. Sharon Sharon.
Are you starting to carry a farm over 12 years ago, Our Michigan was really quite simple you wanted to do everything we possibly could.
Michael P. Mason: We wanted to do everything we possibly could to make a difference in the lives of patients battling cancer. More specifically, we sought to develop novel drugs that exploited a fundamental pillar of oncogenesis, namely the reactivation of tumor suppressor proteins by inhibiting their export out of the cell nucleus, which, as you may know, is actually where the name Karyopharm comes from. Now, with our lead medicine Expovio having received three separate FDA approvals and one marketing authorization in Europe, over 450 employees, and a robust pipeline of programs across both hematologic and solid tumor indications, I could not be more proud of the work we've accomplished on behalf of patients, their families, and healthcare providers.
From the lives of patients battling cancer.
More specifically, we start to develop novel drugs that explain a fundamental market got it.
Namely the reactivation of tumor suppressor proteins by inhibiting their export out of the zone nucleus, which as you may know as accident, whether they carry a farm.
Now what I read Madison exposure.
<unk> reached our current FDA approvals and one marketing authorization in Europe over 450 employees and a robust pipeline of programs.
Both hematologic and solid tumor indications I could not be more proud of the work we've accomplished on behalf of patients and their families and health care providers.
Michael P. Mason: But as our company is increasingly focused on commercial execution and competing in the global cancer marketplace, the time is right for a new leader, particularly one with a strong track record of building successful oncology commercial brands. Richard, of course, is no stranger to Karyopharm, having served on our board of directors since February 2020, and prior to his new role here, he served as Chief Executive Officer of ZipStim North America and is the former Vice President and General Manager of Oncology at Amherst. Richard will provide some of his thoughts regarding Karyopharm's future at the end of today's review of our Q1 2021 earnings and business update. Please now turn to slide five.
But as our company is increasingly focused on commercial execution and competing in the global cancer marketplace. At the time is right for a new leader, particularly one with strong track record of building.
Festival oncology commercial brands.
And of course is no stranger to carrier from having served on our board of directors in February 2000, and prior to his new role here Richard served as Chief Executive Officer, North America and is the former Vice President and general manager of oncology at Amgen.
Richard will provide some of his thoughts regarding carry a farm future at the end of today's review of our Q1, 2020, one earnings and business update.
Please now turn to slide five.
Michael P. Mason: Total revenues were $23.3 million, with Expovio net product sales of $21.7 million in the quarter. Importantly, Expovio prescription demand increased 17% in Q1 2021 as compared to Q4 2020, following the expanded FDA indication granted in December of 2020. We also saw more than 160 new physicians or accounts prescribing Explovio for the first time in the quarter. This quarterly growth occurred when many myeloma brands were flat or declining.
Total revenues were $23 $3 million net exposure net product sales of $21 7 million in the quarter importantly, exposing a prescription demand increased 17% in Q1 2021 as compared to Q4 2020. Following the expanded FDA indication granted in December of 2020.
We also saw more than 160, new physicians or accounts prescribing you slow for the first time in the quarter.
This quarterly growth occurred when many myeloma brands from flat or declining.
Michael P. Mason: Moving to our pipeline progress, we recently announced that in March 2021, the European Commission granted a conditional marketing authorization for Nexpovio in combination with dexamethasone for the treatment of patients with multiple myeloma who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents, and an anti-CD38 monoclonal antibody, and who Additionally, we also announced that we have now submitted the clinical data from our phase three Boston study as part of a type two mark variation marketing authorization application, previously requesting an expansion of our indicated label in Europe. This submission has now been validated, and we expect to have a decision on this application from the European Commission in the fourth quarter of 2021.
Moving to our pipeline progress, we recently announced that in March of 2021, the European Commission granted conditional marketing authorization for <unk> in combination with dexamethasone in the treatment of patients with multiple myeloma, who have received at least four prior therapies and whose diseases refractory to at least two proteasome inhibitors two amino module.
Tori agents and an anti <unk> monoclonal antibody and who have demonstrated disease progression on the last therapy.
Additionally, we also announced that we have now submitted the clinical data from our phase III Boston study as part of the type two variation marketing authorization application.
Formerly requesting an expansion of our indicated label in Europe.
This submission has now been validated and we expect to have a decision on this application from the European Commission in the fourth quarter of 2021.
Turning now to some other program updates from our ongoing Hematological and solid tumor clinical studies. We are pleased to see the first set of data from our phase III study evaluating <unk> in patients with differentiated life of sarcoma were recently published in the first patients have now been dosed in a phase III study in diffuse large b cell and Paul.
Michael P. Mason: Turning now to some other program updates from our ongoing hematological and solid tumor clinical studies, we are pleased to see the first set of data from our phase three SEAL study evaluating expovio in patients with de-differentiated liposarcoma recently published, and the first patients have now been dosed in a phase three study in diffuse large B cell lymphoma. Finally, on the financial front, we ended the quarter with a strong cash position of approximately $233.6 million that, along with expected future revenues, we anticipate will be sufficient to fund our planned operations into late 2022.
Finally on the financial front, we ended the quarter with a strong cash position of approximately $233 $6 million that along with expected future revenues, we anticipate will be sufficient to fund our planned operations into late 2022.
Let's now turn to slide six where I'll provide additional details on exposure.
Performance first quarter net exposure sales increased by 7% as compared to the fourth quarter of 2020, while prescriptions increased by approximately 17%.
Michael P. Mason: Let's now turn to slide six, where I'll provide additional details on Expovio's recent sales performance. First quarter net expovio sales increased by 7% as compared to the fourth quarter of 2020, while prescriptions increased by approximately 17%. This increase was primarily driven by new multiple myeloma patient starts. Additionally, we've seen strong payer coverage since the expanded approval we received from the FDA in December. I will note that much of the difference between the sales growth and prescription growth was driven by higher gross net discounts, which are fairly typical in the first quarter of the year as commercial and Medicare patients' out-of-pocket payment requirements reset for the new year.
This increase was primarily driven by multiple myeloma, new patient starts. Additionally, we've seen strong payer coverage since the expanded approval received from the FDA in December I will note that much of the difference between the sales growth in prescription growth was driven by higher gross to net discounts, which are fairly typical in the first quarter of the year as commercial and Medicare patients.
Out of pocket payment requirements reset for the new year.
There were also some additional stocking within our distributor network at the end of 2020 and preparation for the launch of exposure.
Expanded indications.
Well, we expected higher demand growth. We are encouraged that sales in prescription demand returned to growth from the first quarter and we remain confident exposing us long term commercial potential and our ability to further increase utilization Richard is top priority in the coming weeks and months ahead of us will be to help our organization further accelerate the growth trajectory.
In the U S market. We believe some of this will occur naturally as we expect to see the benefit of longer duration of exposure treatment in the second half of 2021, and we also believe our teams will have better access to customers in the second half of the year, which will increase promotional impact.
Michael P. Mason: There will also be some additional stocking within our distributor network at the end of 2020 in preparation for the launch of Explodio's Expanded Indication. While we expected higher demand growth, we are encouraged that sales and prescription demand returned to growth in the first quarter, and we remain confident in Expovio's long-term commercial potential and our ability to further increase utilization. Richard's top priority in the coming weeks and months ahead of us will be to help our organization further accelerate the growth trajectory of expovio in the US market.
Please now turn to slide seven.
Here you can see a chart a monthly prescriptions for the first three months of 2021 importantly, we saw a significant increase in March and we'll be working hard to further expand utilization and penetration into the earlier line treatment setting in the near future.
Moving now to slide eight the graph here shows the prescription refill rate breaks volume for both the first and second retails for those patients eligible for these rebuilds. These numbers have remained encouraging throughout 2020 and into 2021 and are significantly higher as compared to our initial launch period from 2019. These refill rates coupled with average.
Michael P. Mason: We believe some of this will occur naturally as we expect to see the benefit of longer duration of expovio treatment in the second half of 2021. And we also believe our teams will have better access to customers in the second half of the year, which will increase promotional impact. Please now turn to slide seven.
An average of nearly three treatment cycles per patient further reinforced the positive feedback we've received from patients and physicians regarding their experience and uses of exposure.
Michael P. Mason: Here you can see a chart of monthly Expovio prescriptions for the first three months of 2021. Importantly, we saw a significant increase in March and will be working hard to further expand utilization and penetration into the earlier line treatment setting in the near future. Moving now to slide 8, the graph here shows the prescription refill rate for Exfobio for both the first and second refills for those patients eligible for these refills.
Importantly, we do expect the average duration of treatment to increase throughout 2021 as more and more patients are being treated with a once weekly <unk> dose and as part of a combination triplet regimen.
Additionally, patient discontinuation rates due to side effects remains relatively low at 12%, which we believe is a testament to more and more physicians gaining comfort in helping their patients prevent and manage side effects from exposure with proper prophylactic therapies and dose modifications.
Michael P. Mason: These numbers have remained encouraging throughout 2020 and into 2021 and are significantly higher as compared to our initial launch period in 2019. These refill rates, coupled with an average of nearly three treatment cycles per patient, further reinforce the positive feedback we've received from patients and physicians regarding their experience and use of exfovio. Importantly, we do expect the average duration of treatment to increase throughout 2021 as more and more patients are being treated with a once-weekly exfovio dose and as part of a combination triplet regimen.
On slides nine and 10, you can see our most current robust clinical development plan for <unk>.
In both Hematological malignancies and solid tumors. This.
This includes our phase III <unk> study in patients with endometrial cancer, where we expect to have top line data before the end of this year and which I'll highlight in more detail in a few moments.
I will note that there are two important clinical trials, we expect to initiate in 2021, which we believe will help further define our broad clinical utility of exposure.
Total partner of choice with other activating cancel rates.
Michael P. Mason: Additionally, patient discontinuation rates due to side effects remain relatively low at 12%, which we believe is a testament to more and more physicians gaining comfort in helping their patients prevent and manage side effects from exfoliation with proper prophylactic therapy and dose modification.
First we expect to start a new randomized phase III study evaluating <unk> in combination with analysts and dexamethasone in patients with previously treated myeloma that will begin in 2021 as a result of this trial is positive this regimen from represent a potent oral drug option to patients with refractory myeloma.
Michael P. Mason: On slides 9 and 10, you can see our most current robust clinical development plan for exfovio in both hematological malignancies and solid tumors. This includes our phase 3 C-endo study in patients with endometrial cancer, where we expect to have top-line data before the end of this year, and which I'll highlight in more detail in a few moments. I will note that there are two important clinical trials we expect to initiate in 2021, which we believe will help further define the broad clinical utility of Expovio as a potential partner of choice with other active anti-cancer agents.
Actually we plan to initiate a new phase II study evaluating <unk> in combination with keytruda in patients with newly diagnosed or recurrent metastatic melanoma.
We're particularly excited about this study based on some encouraging data from an investigator sponsored trial from MD Anderson evaluating this combination regimen, which was presented at the annual ESMO conference in 2020.
Moving to slide 11, I'll highlight the potential opportunity to explore beyond patients with endometrial cancer, where we're currently conducting our phase III <unk> study.
Michael P. Mason: First, we expect to start a new randomized phase 3 study evaluating expovio in combination with pomelos and dexamethasone in patients with previously treated myeloma that will begin in 2021. If the results of this trial are positive, this regimen could represent a potent all oral drug option for patients with refractory myeloma. Next, we plan to initiate a new Phase 2 study evaluating expoglio in combination with Keytruda in patients with newly diagnosed or recurrent metastatic melanoma.
Endometrial cancer is the most common gynecologic cancer in the U S with over 65000, new cases, and unfortunately over 12000, new debt in 2020.
While most women are diagnosed with early stage disease and have a good prognosis. After surgery alone approximately 14000 patients each year in the United States have advanced or metastatic disease and our strength.
With combination chemotherapy in the frontline setting when their disease progresses. These patients are typically treated with additional chemotherapy immunotherapy and our targeted agents. However, currently there are no approved drugs in the maintenance setting for patients who've had a response to their frontline chemotherapy.
Michael P. Mason: We're particularly excited about this study based on some encouraging data from an investigator-sponsored trial from MD Anderson evaluating this combination regimen, which will be presented at the annual ESMO conference in 2020. Moving to slide 11, I'll highlight the potential benefit of Expovia in patients with endometrial cancer, where we're currently conducting our phase three CIANDO study. Endometrial cancer is the most common gynecologic cancer in the U.S., with over 65,000 new cases and, unfortunately, over 12,000 new deaths in 2020.
This is a setting in which we're currently studying exposure in the CNS done.
A similar approach was taken with PARP inhibitors for patients with ovarian cancers and their use in this maintenance setting in that disease has been quite dramatic.
To put our potential and opportunity in endometrial cancer and perspective.
About two thirds of frontline patients responding chemotherapy, there could be 3000, or so patients treated each year in the maintenance setting by capturing approximately 30% of this market.
Now as we move to slide 12, you will see highlights from our previous Phase II study published in 2019, which evaluated selinexor and 114 patients with heavily pretreated and actively progressing gynecologic cancers, including 23 patients with heavily pretreated endometrial cancer, who have previously received a median of up.
Michael P. Mason: While most women are diagnosed with early-stage disease and have a good prognosis after surgery alone, approximately 14,000 patients each year in the United States have advanced or metastatic disease and are treated with combination chemotherapy in the frontline setting. When their disease progresses, these patients are typically treated with additional chemotherapy, immunotherapy, or targeted aging. However, currently, there are no approved drugs in the maintenance setting for patients who've had a response to their frontline chemotherapy. This is the setting in which we're currently studying Expovio and Ciendis.
Of two and up to five fire lines of therapy.
In this population with growing cancer patients treated with single agent Selinexor demonstrated a disease control rate of 35% and a confirmed partial response rate of 9%.
Michael P. Mason: A similar approach was taken with PARP inhibitors for patients with ovarian cancer, and their use in this maintenance setting in that disease has been quite dramatic. To put our potential opportunity in endometrial cancer in perspective, assuming about two-thirds of front-line patients respond to chemotherapy, there could be 3,000 or so patients treated each year in the maintenance setting by capturing approximately 30% of this mark. Now, as we move to slide 12, you will see highlights from a previous phase 2 study published in 2019, which evaluated telonexer in 114 patients with heavily pre-treated and actively progressing gynecologic cancers, including 23 patients with heavily pre-treated endometrial cancer who previously received a median of 2 and up to 5 lines of therapy.
Most common side effects were similar to other selling extra studies and included nausea, fatigue decreased appetite vomiting weight loss anemia, thrombocytopenia, Dysgeusia and blurred vision and were primarily grade one or two and reversible.
On the right hand side of the slide you can see a waterfall plot of the best per cent change in the sum of all the target lesions from screening for 19, evaluable patients with endometrial cancer.
This encouraging clinical data in a population with highly refractory.
Regressive endometrial cancer gave us the confidence to conduct a <unk> study in the frontline maintenance setting which is summarized on slide 13.
CNS is enrolling approximately 248 patients randomized two to one to receive either 80 milligram selinexor once weekly or matching placebo.
Eligible patients included those who have completed a single line of at least 12 weeks of Taxane platinum combination chemotherapy and achieve either a partial or complete response.
Michael P. Mason: In this population with growing cancer, patients treated with single agent telonexor demonstrated a disease control rate of 35% and a confirmed partial response rate of 9%. The most common side effects were similar to other cell anexure studies and included nausea, fatigue, decreased appetite, vomiting, weight loss, anemia, thrombocytopenia, dysgousia, and blurred vision, and were primarily grades one and two and reversible.
Primary endpoint of the trial is improvement in progression free survival from the time of randomization until death or disease progression.
In November of 2020, we announced the trial passed as planned interim futility analysis and so the study continues as planned with no modifications and we expect the top line data by the end of this year.
We remain highly encouraged by this study and the opportunity for patients should the trial met its primary endpoint and we look forward to providing additional updates from the future.
Michael P. Mason: On the right-hand side of the slide, you can see a waterfall plot of the best percent change in the sum of all the target lesions from screening for 19 invaluable patients with endometrial cancer. This encouraging clinical data in a population with highly refractive, progressive endometrial cancer gave us the confidence to conduct the CIENDO study in the Frontline Maintenance Study, which is summarized on slide 13. She ended up enrolling approximately 248 patients, randomized 2 to 1, to receive either 80mg Selenex once weekly or matching placebo.
With that I'll now turn the call over to Mike Mason to review the quarterly financials Mike.
Michael since we issued a press release earlier today with the full financial results I'll just focus on the highlights which begin on slide 15.
Net product revenue for the first quarter of 2021 was $21 7 million compared to $16 1 million from the first quarter of 2020.
The estimated gross to net discount for ex <unk> in Q1, 2021 was slightly higher at 21% from the top of our expected range of 15% to 20%.
Michael P. Mason: Eligible patients include those who have completed a single line of at least 12 weeks of taxade-platinum combination chemotherapy and achieved either a partial or complete response. The primary endpoint of the trial is improvement in progression-free survival from the time of randomization until death or disease progression. In November of 2020, we announced the trial had passed its planned interim futility analysis, and so the study continues as planned with no modifications, and we expect top-line data by the end of this year.
License and other revenue for the first quarter of 2021 was $1 5 million compared to $2 $1 million per the first quarter of 2020.
R&D expenses for the first quarter of 2021 were $37 1 million compared to 34 million from the first quarter of 2020.
The increase in R&D expenses in 2021 compared to 2020 was primarily attributable to costs incurred related to our ongoing clinical trials and regulatory activities.
Selling general and administrative expense for the first quarter of 2021 was $37 7 million compared to $30 7 billion from the first quarter of 2012, the increase in SG&A expenses compared to the prior year was due primarily to activities to support the U S commercialization of exposure.
Michael P. Mason: We remain highly encouraged by this study and the opportunity for patients should the trial meet its primary end point, and we look forward to providing additional updates in the future. With that, I'll now turn the call over to Mike Mason to review the quarterly financials.
On slide 16, you can see that cash cash equivalents restricted cash and investments as of March 31, 2021, total $233 6 million compared to $276 seven zone as of December 31, 2012.
Michael P. Mason: Thank you, Michael. Since we issued a press release earlier today with the full financial results, I will just focus on the highlights, which begin on slide 15. Net product revenue for the first quarter of 2021 was $21.7 million, compared to $16.1 million for the first quarter of 2020.
Finally based on our current operating plans carrier products like non-GAAP, R&D, and SG&A expenses, which exclude stock based compensation expense for the full year 2021 to be in the range of shorter 8 million to $300.
The company expects that its existing cash cash equivalents and investments and the revenue are expected to generate from COVID-19 product sales and other license revenues will be sufficient to fund planned operations into late 'twenty target zone.
Michael P. Mason: The estimated gross net discount for Expovio in Q1 2021 was slightly higher at 21%, and the top of our expected range is 15 to 20%. License and other revenue for the first quarter of 2021 was 1.5 million, compared to 2.1 million for the first quarter of 2020. R&D expenses for the first quarter of 2021 were $37.1 million, compared to $34 million for the first quarter of 2020. The increase in R&D expenses in 2021 compared to 2020 was primarily attributable to costs incurred related to ongoing clinical trials and regulatory activities. Telling general and administrative expenses for the first quarter of 2021 were $37.7 million compared to $30.7 million for the first quarter of 2020.
We are not providing revenue guidance for 2021 today is we remain in the initial launch phase of exposure and its expanded indication, but do expect to see meaningful growth in 2021 relative to 2020 with a ramp of sales increasing in the second half of the year as we expect to see the benefit of a longer duration of treatment for multiple myeloma patients being prescribed exposure.
Earlier in the treatment of force <unk> in combination with Velcade.
I'll now turn the call over to Richard for some of his thoughts about carrot bombs Richard.
Thank you Mike.
Let me begin by saying how excited I am to be leading the current pharm organization and such inspiring times.
Of course carry apartment is not new to me and I serve on its board of directors since February of 2020.
And first I would like to acknowledge and thank Dr. Michael Kauffman for his immense contribution to the scientific and initial commercial success achieved by Gary requirement.
Dr Kauffman, both as a clinician at drug developer.
Truly enabled our company to reach amazing scientific and clinical achievements.
During my tenure on the board I've been extraordinarily impressed with book this amazing companies being able to do.
Michael P. Mason: The increase in SG&A expenses compared to the prior year was due primarily to activities to support the U.S. commercialization of exposures. On slide 16, you can see that cash, cash equivalents, restricted cash, and investments as of March 31, 2021 totaled $233.6 million compared to $276.7 million as of December 31, 2021. Finally, based on our current operating plans, Karyopharm expects non-GAF R&D and SG&A expenses, which exclude stock-based competition expenses, for the full year 2021 to be in the range of $280 million to $300 million.
In such a short period of time.
Now with three separate FDA approvals and one conditional European marketing authorization branch at.
Oh, having being achieved in just the past few years.
Of course, all of these approvals were preceded by many years of careful and thoughtful clinical development per exposure.
And carryover from remains deeply rooted and innovative science with a passion for improving the lives of patients battling cancer.
That is very near and Dear to me is a passion I worked towards.
Importantly.
I believe the best is yet to come from both carryover arm and for the patients we aim to serve.
However, I know that a lot of work still remains.
<unk> is now effectively transitioned to a commercial stage organization.
I am excited to lead the company in its next chapter as we seek to expand exposure impact across indications and geographies.
Michael P. Mason: The company expects that its existing cash, cash equivalents, and investments, and the revenue it expects to generate from Expovia product sales and other licensed revenues, will be sufficient to fund its planned operations into late 2020. We are not providing revenue guidance for 2021 today as we remain in the initial launch phase of Expovio and its expanded indication, but we do expect to see meaningful growth in 2021 relative to 2020, with the ramp of sales increasing in the second half of the year, as we expect to see the benefit of a longer duration of treatment for multiple myeloma patients being prescribed Expovio earlier in their treatment course and or in combination with Velpe. I'll now turn the call over to Richard for some of his thoughts about Karyopharm's future. Richard.
I look forward to leveraging my experience and global product commercialization and organizational leadership to help further advance <unk> impact in helping improve patient outcomes.
And I'm excited to work alongside of both Michael and Sharon.
On behalf of our patients.
Boys partners and shareholders.
Regarding my initial priorities in the near term I think we have a great opportunity to expand our breadth and depth in the multiple myeloma treatment landscape where.
Where we have really just begun to scratch the surface regarding where we can compete and help patients.
And as we continue to generate additional combination data with other myeloma drugs.
We believe exposure has the opportunity to become an increasingly important backbone therapy.
And moving beyond multiple myeloma, I believe <unk> can become a portfolio and a pill.
With the potential to have clinically meaningful utility across a host of cancers.
Richard A. Paulson: Thank you, Mike. Let me begin by saying how excited I am to be leading the Karyopharm organization in such inspiring times. Of course, Karyopharm is not new to me, as I have served on its board of directors since February of 2020. And first, I would like to acknowledge and thank Dr. Michael Kaufman for his immense contributions to the scientific and initial commercial success achieved by Karyopharm.
Including many solid tumor indications both as a single agent and importantly, as a future combination partner of choice, but other cancer medicines.
Allowing for synergistic opportunities to further help patients battling cancer.
Now before we open the call up to your questions.
Let me highlight a few of the key commercial clinics.
Clinical and regulatory milestones that we have already achieved so far in 2021.
Richard A. Paulson: Dr. Kauffman, both as a clinician and drug developer, has truly enabled our company to reach amazing scientific and clinical achievements. During my tenure on the board, I've been extraordinarily impressed with what this amazing company has been able to do in such a short period of time. Now, with three separate FDA approvals and one conditional European marketing authorization grant, all having been achieved in just the past few years. Of course, all of these approvals were preceded by many years of careful and thoughtful clinical development for Exspobia.
And others that we expect for the remainder of the year and shown on slide 17.
First we have made significant progress in Europe, where we recently received conditional marketing authorization for an exposure.
And have submitted a type two variation application based on the phase III Boston study.
Which was recently validated by the EMA.
Next while we've made some progress ex <unk> ex increasing exposure sales in U S.
We are far from content year and remain committed to further penetrating the U S market throughout the remainder of this year and beyond.
This is where I intend to spend much of my time in the weeks and months ahead.
Richard A. Paulson: And Karyopharm remains deeply rooted in innovative science with a passion for improving the lives of patients battling cancer, something that is very near and dear to me as a passion I work toward. Importantly, I believe the best is yet to come for both Karyopharm and for the patients we aim to serve. However, I know that a lot of work still remains.
And finally in the second half of this year, we expect top line data from the phase III <unk> study in endometrial cancer.
The initiation of multiple clinical trials and the presentation of additional combination data with <unk> and other cancer therapies at a variety of medical meetings.
I look forward to updating the investment community on our continued progress in the months and quarters ahead.
Richard A. Paulson: As Karyopharm has now effectively transitioned to a commercial-stage organization, I am excited to lead the company in its next chapter as we seek to expand Expovio's impact across indications and geography. I look forward to leveraging my experience in global product commercialization and organizational leadership to help further advance Karyopharm's impact in helping improve patient outcomes. And I'm excited to work alongside both Michael and Sharone on behalf of our patients, employees, partners, and shareholders.
And with that I would now like to ask the operator to open the call up to your question natural portion of today's presentation.
Operator.
Thank you we will now begin the question and answer session to ask a question you May Press Star then one on your Touchtone phone if youre using a speakerphone. Please pick up your handset before pressing the keys to withdraw your question. Please press Star then two please.
Please ask one question and one follow up.
Our first question comes from Brian Abrahams from RBC capital markets. Please go ahead.
Richard A. Paulson: Regarding my initial priorities, in the near term, I think we have a great opportunity to expand our breadth and depth in the multiple myeloma treatment landscape, where we have really just begun to scratch the surface regarding where we can compete and help patients. And as we continue to generate additional combination data with other myeloma drugs, we believe Exfovio has the opportunity to become an increasingly important backbone therapy.
Hi, there thanks for taking my questions and I guess first off congratulations to Michael and Sharon on all your accomplishments I want to wish you. The best of luck in your new roles and congratulations to Richard as well.
Thanks, Brian.
Yeah. So first off I, just I'm curious if you could maybe provide a little bit more color around the <unk> use patterns in the U S. In the earlier line population maybe.
Richard A. Paulson: And moving beyond multiple myeloma, I believe Expovio can become a portfolio in a pill, with the potential to have clinically meaningful utility across a host of cancers, including many solid tumor indications, both as a single agent and, importantly, as a future combination partner of choice with other cancer medicines, allowing for synergistic opportunities to further help patients battling cancer. Now, before we open the call up to your questions, let me highlight a few of the key commercial, clinical, and regulatory milestones that we've already achieved so far in 2021 and others that we expect for the remainder of the year, as shown on slide 17.
Can you help characterize the blend of the penta refractory versus earlier line Boston population being put on drug based on your market research.
What's your sense as to how <unk> is fitting into the current competitive landscape across lines versus previous quarters, and then I had a follow up thanks.
Sure Ken This is John Demaree, our Chief commercial officer, Don Thanks, Michael.
Sales growth, we saw Brian with the cross lines of therapy with the majority coming from later line patients. It's hard to tell for sure is prescription data we receive from our specialty pharmacies and distributors does not capture and what line of therapy patients are receiving <unk>.
However from secondary market research data repurchase our own market research with prescribers and AD boards are sensitive that's still much of the business is coming from later lines EG fourth line in later lines, though many of those patients are now receiving the triplet combination with exposure.
Richard A. Paulson: First, we have made significant progress in Europe, where we recently received conditional marketing authorization for Nexovio and have submitted a type two variation application based on the phase three Boston study, which was recently validated by the EMA.
Richard A. Paulson: Next, while we have made some progress increasing expovial sales in the U.S., we are far from content here and remain committed to further penetrating the US market throughout the remainder of this year and beyond. This is where I intend to spend much of my time in the weeks and months ahead. And finally, in the second half of this year, we expect top-line data from the Phase 3 Siendo study in endometrial cancer, the initiation of multiple clinical trials, and the presentation of additional combination data with Exscobio and other cancer therapies at a variety of medical meetings.
And another myeloma backbone instead of just exposure.
That's the message out which was more commented in the past, indicating we're getting trial of the new triplet indication and we would expect them to try it in later lines and then move it up into earlier lines as they have success. So while the majority of patients are still in later lines, we are making inroads with some physicians who are starting to prescribe exposure to their second third line patients.
Got it that makes it a lot of sense and then maybe just a follow up just maybe a bigger picture strategic question for Richard Selinexor, Obviously, that's true.
Signals in a number of different tumor types, where do you see the most promising opportunities for the drug do you see any opportunity to further refine the development strategy or even maybe a partner and some of these combo indications you mentioned to split economics.
Richard A. Paulson: I look forward to updating the investment community on our continued progress in the months and quarters ahead. And with that, I would now like to ask the operator to open the call up to the question and answer portion of today's presentation. Operator.
To enable any further cost savings on the R&D side.
Jason: Thank you. We will now begin the question and answer session. To ask a question, you may press star then 1 on your touch-tone phone. If you are using a speakerphone, please pick up your handset before pressing the keys.
Thank you Brian.
We'll get into that.
Future share with share with you and the other analysts but.
In the near term, we have a great opportunity to really expand our breadth and depth and multi myeloma treatment landscape.
And as you know, we've just begun there and John talk to that as we're starting to move into earlier lines, where we can compete and help more patients.
Jason: To withdraw your question, please press star then 2. Please ask one question and one follow-up. Our first question comes from Brian Abrahams from RBC Capital Markets. Please go ahead. Hi there.
As you heard from Michael we are generating new data and the ex.
This study as we continue to generate additional combination data with other myeloma drugs I do believe we have the opportunity to be a backbone.
Brian Corey Abrahams: Thanks for taking my questions. And I guess, first off, congratulations to Michael and Sharon on all your accomplishments. I want to wish you the best of luck in your new roles. And congratulations to Richard as well.
In many areas.
And as we move forward and as we heard a little bit around D&O moving beyond multiple myeloma.
I really believe this opportunity to be a portfolio in a pill.
As clinically meaningful force and will allow us to have utility across a host of cancers. So we'll have to explore moving forward, how we bring that to life.
Brian Corey Abrahams: Thanks Brian. My pleasure. Yeah, so first off, I just I'm curious if you can maybe provide a little bit more color around Expovio use patterns in the U.S. and in the earlier line population, maybe Can you help characterize the blend of the pentarefractory versus earlier line Boston population being put on drugs based on your market research and What's your sense as to how Expovio is fitting into the current competitive landscape across lines versus you know in previous quarters and then how to follow up?
But when you look at many solid tumor indications both as a single agent and importantly, as you touched on is a combination partner from our southern cancer medicines, it's going to allow us to really synergistically.
<unk> from more patients battling cancer moving forward.
Okay.
Got it thanks, so much.
The next question comes from Maury Raycroft from Jefferies. Please go ahead.
Morning, everyone and I would like to add my congrats to Michael Sharon and Richard too.
Brian Corey Abrahams: Sure, I'll turn that to John Demery, our Chief Commercial Officer. John. Thanks, Michael. As sales grew, we saw Brian with the cross lines of therapy, with the majority coming from later line patients. It's hard to tell for sure as prescription data we receive from our specialty pharmacies and distributors does not capture in what line of therapy patients are receiving Expovio. However, from the secondary market research data we purchase, and our own market research with prescribers and ad boards, our sense is that much of the business is still coming from later lines, e.g., fourth line and later lines.
Maybe first question is just if you can talk more about the higher gross to net discount in <unk> compared to <unk> 20 can you say what the discount was in <unk> and we'll just continue going forward or how should we think about it.
Sure, Yes, I think so with interest.
<unk> guided that we would end up from a range of 15 to 20 per cent for growth. So that we ended up just slightly north of that in Q1 net at 21% in pretty typical you know where the first quarter with the copay resets et cetera. So we do expect that to come down into our into our range of 15 to 20 per cent as we think about.
The entire year.
Some lumpiness quarter to quarter.
Got it that's helpful and then as far as access for the sales team goes can you provide any more specifics or quantify how much access they have currently and where you aim to get in the second half of 'twenty one.
John Demery: So many of those patients are now receiving the triplet combination with Expovio and another myeloma backbone instead of just Expovio plus dexamethasone, which was more common in the past, indicating we're getting trials of the new triplet indication, and we would expect them to try it in later lines and then move it up into earlier lines if they have success. So while the majority of patients are still in later lines, we are making inroads with some physicians who are starting to prescribe Expovio to their second and third line patients. Got it; that makes a lot of sense. And then maybe just the follow-up, just maybe a bigger picture strategic question for Richard. Selenix obviously has shown signals in a number of different tumor types.
Sure John I'll take that.
So in terms of access to customers.
COVID-19 has been a challenge it's created an impact for patients for HCP and the entire industry and we do still have some access challenges, we do see that growing.
It's growing at a different pace in different parts of the country. The last secondary data report that we saw that suggested that about 20 to 25 per cent of our interactions are in person and.
75% to 85% of our interactions are still virtual we're providing our teams tools to engage with their customers in both the virtual and in person environments. So they continue to drive the Boston message as we go forward, we expect as we go throughout the remainder of the quarter and about the remainder of the year debt access in person.
Richard A. Paulson: Where do you see the most promising opportunities for the drug, and do you see any opportunity to further refine the development strategy or even maybe partner in some of these combo indications that you mentioned to split the economics to enable any further cost savings on the R&D side? Thanks.
Our customers will continue to grow substantially.
Got it okay. Thank you for taking my question.
Richard A. Paulson: You know, we'll get into that in the future and share with you and the other analysts. But, you know, I think in the near term, we have a great opportunity to really expand our breadth and depth in the multiple myeloma treatment landscape. And as you know, we've just begun there, and John talked about that, as we're starting to move into earlier lines where we can compete and help more patients. As you heard from Michael, we are generating new data in the XPD study as we continue to generate that additional combination data with other myeloma drugs. You know, I do believe we have the opportunity to be a backbone in many areas.
Thank you.
The next question comes from Peter Let Us from Barclays. Please go ahead.
Okay.
Michael just always been a pleasure speaking with you flip with next steps.
First question just for Richard just on.
If we get really transfixed on first 100 days, so just if I could.
Walk us through what you're thinking for.
The initial steps are cared for.
Yeah. Thanks, Thanks Peter.
And the initial steps I think over the next 100 days I mean really what I'm Super excited about with zero harm is your opportunity to help patients.
Richard A. Paulson: And as we move forward, and as we heard a little bit about Siendo, moving beyond multiple, I really believe this opportunity to be a portfolio and a pill is clinically meaningful for us and will allow us to have utility across a host of cancers. So we'll have to explore, moving forward, how we bring that to life. When we look at many solid tumor indications, you know, both as a single agent and, importantly, as you touched on, as a combination partner of course to other cancer medicines, it's going to allow us to really synergistically bring exfobio to more patients battling cancer moving forward. Thanks so much.
As you know exposure I think is uniquely positioned with its broad mechanism of action and its ability to be combined with a with a variety of other cancer drugs.
And enable us to make a tremendous impact on patients battling cancer.
In the near term as we touched on our focus is.
Continuing to move up past the initial launch of exposure and enter a refractory multiple myeloma.
I shall launch was successful, but as we expand into earlier line settings, it's more complex and it's going to require us to really expand our breadth and depth. So that is a key area of focus for me in the near term.
And then also should the phase III <unk> study in endometrial cancer prove successful.
Maurice Thomas Raycroft: The next question comes from Maurice Raycroft from Jefferies. Please go ahead. Thank you for joining us, everyone, and I would like to add my Maybe the first question is just... We can talk more about the higher growth in that. Unknown Speaker 1-1-1, Sure, yeah, I think we've got it that we end up in a range of 15 to 20% for growth and that we ended up just slightly north of that in Q1 at 21%. And pretty typical, you know, with the first quarter with the copay resets, etc.
We will need to serve entrepreneurs relations early index.
The next little while so and this is potentially a significant commercial opportunity for us moving forward.
Going to need to see how we bring that to life over the near term. So those are a couple of areas.
Near term focus.
And finally as we're in the process of discussing and evaluating potential partners in Europe and Japan.
With the recent marketing authorizations in EU as.
As well as the acceptance of the MAA for Boston mutation, which is where we know much of our value to us and potential partners will be a generator.
Michael P. Mason: So we do expect that to come down into our, you know, our range of 15 to 20%. As we think about, you know, for the entire year, with some lumpiness in a quarter. And then as far as access goes, can you provide any more? John, I'll take that.
We need to continue to explore those partnership opportunities.
The immediate priority is around sharing we bring an exposure to the EU and multiple myeloma.
However, potential partners have also expressed very high interest in our solid tumor pipeline.
John Demery: Yeah, so in terms of access to customers, you know, COVID has been a challenge. We've created an impact for patients, for HCPs, and the entire industry, and we do still have some access challenges. We do see that growing, though it's growing at a different pace in different parts of the country.
So as we touched on making sure we find the right strategy moving forward in Europe is also going to be a key focus.
Thank you and then just from your prior experience, which should put parallels do you have with drug similar.
John Demery: The last secondary data report that we saw suggested that about 20-25% of our interactions are in-person, and 75-85% of our interactions are still virtual. We're providing our teams tools to engage with their customers in both the virtual and in-person environments, so that you continue to drive the Boston message as we go forward. We expect, as we go throughout the remainder of the quarter and the remainder of the year, that access in-person to our customers will continue to grow substantially.
To selinexor.
Yes, Thanks Peter.
In the past I've had the opportunity to bring drugs to market and multiple myeloma, specifically working on Kyprolis and I think as we know it.
Many areas when you first launch and later later stages of the disease and then work to move up.
We need to continue to work with physicians to help them better understand the medicine.
Some have understand how to initiate and manage patients.
And that's a process that takes time, but one which we're committed to moving forward.
Maurice Thomas Raycroft: Got it. Okay, thank you for taking my question. Thank you. The next question comes from Peter Laws from Barclays. Please go ahead. Hey Michael, it's always been a pleasure speaking with you and best of luck with the next one. And maybe the first question just for Richard just on because we get really transfixed on the first 100 days, so I just wonder if you could walk us through what you're thinking for the initial steps at Karyopharm.
Perfect. Thanks, so much congratulations Quebec and thank you.
The next question comes from Eric Joseph from Jpmorgan. Please go ahead.
Thanks for taking the questions good morning.
Together the dog Richard I will do my best to not volume Paul Richardson.
Thank you goodbye.
Especially in multiple myeloma.
Right exactly.
Just a couple of questions.
Peter Richard Lawson: Thanks. Thanks, Peter.
Richard A. Paulson: You know, in the initial steps, I think over the next 100 days, really, what I'm super excited about with Karyopharm is the opportunity to help patients. As you know, Expovio, I think, is uniquely positioned with its broad mechanisms of action and its ability to be combined with a variety of other cancer drugs. Now, it's going to enable us to make a tremendous impact on patients battling cancer. You know, in the near term, as we touched on, our focus is to continue to move past the initial launch of exfobio and pancreas-refractory multiple myeloma.
Recent.
From friends and looking forward here exiting first quarter growth seems pretty strong I guess can you comment on demand trends through April so far and are you able to.
Perhaps guide us a little bit.
On second quarter expectations.
Then just a.
Follow up on sort of where youre seeing used per guidance.
The once weekly.
Sorry, with most of that a bad day for.
The once weekly regimen Justin.
Combination use.
Is there any expectation there.
True to longer average duration on therapy or is it really more about trying to.
Expand into.
Earlier line use.
Richard A. Paulson: I think the initial launch was successful, but as we expand into earlier line settings, it's more complex, and it's going to require us to really expand our breadth and depth. So that is a key area of focus for me in the near term. And then also, should the phase three Siendo study in endometrial cancer prove successful, we will need to start launch preparations early. In the next little while, so this is potentially a significant commercial opportunity for us moving forward.
EBIT.
Yes.
You can no longer type of therapeutic range.
Thanks for taking the question.
Eric It's Michael I'm listen, we're not going to comment on on this quarter's growth.
At all at this point and we'll update you obviously, when we can but I will turn it over to John from the second part of the question.
Now looking at our dosing mix. Thanks for your question. There are just as an example last year in Q1 over 50% of our new patients who are being started on the twice a week 80 milligram dose 160 milligrams per week, which was the starting dose in the storm population in this most recent quarter, but only 15% of patients where.
Richard A. Paulson: We're going to need to see how we bring that to life over the near term. So those are a couple areas of my near-term focus. And finally, you know, as we're in the process of discussing and evaluating potential partners in Europe and Japan. With the recent marketing authorization for EU, you know, as well as the acceptance of the MAA for Boston communication, which is where we know much of our value to us, potential partners will be a generator.
<unk> started on that dose with the remaining 85% do you started on a once weekly dose most commonly either 100 milligrams or 80 milligrams and while we can't be exactly share which line of treatment. These prescriptions are being generated in syndicated market research. We purchased suggest many of them are being prescribed with other multiple myeloma drugs such as <unk>.
Okay from the Boston study or with <unk> or <unk>, which are in the <unk> guidelines are with Kyprolis, which has been studied in the stomp trial.
We continue to see that the triplet use expand most likely in the later line population now and we do expect that to move early area of patients get more comfortable using exposure with the different triple combinations of course, I mentioned, a couple of different drugs listed <unk> as a company will only promote <unk> indication, which is our on label indication.
Richard A. Paulson: We need to continue to explore those partnership opportunities, and the immediate priority is around ensuring we bring Nexpovial to the EU and multiple myeloma. However, potential partners have also expressed very high interest in our solid tumor pipeline. So, as we touched on, making sure we find the right strategy moving forward in Europe is also going to be a key focus.
Yeah.
Okay got it thats, taking the questions.
The next question comes from David Leibowitz from Morgan Stanley. Please go ahead.
Thank you very much for taking my question is it fair to say that the.
Richard A. Paulson: And then just from your prior experience, Richard, what parallels do you have with drugs, similar to some of the. Thanks, Peter.
Almost all of the incremental increase in prescriptions is from the expanded label or is there some incremental increase.
Richard A. Paulson: You know, in the past, I've had the opportunity to bring drugs to market for multiple myeloma, specifically working on Kyprolis. And I think, as we know, in many areas, when you first launch a drug in later, later stages of the disease and then work to move up, you need to continue to work with physicians to help them better understand the medicine, help them understand how to initiate and manage patients. And that's a process that takes time, but one which we're committed to moving forward with. Thank you so much.
From the D L B C L and and maybe some additional from the initial indication.
Yes, I think it's fair to say that the 17% demand growth was driven primarily by new patient starts in multiple myeloma with most of that being an increase in triplet usage, so driven by the new indication that we've seen.
How do you expect.
I guess, if there's any way to qualitatively.
Peter Richard Lawson: Congratulations. Thank you. The next question comes from Eric Joseph from J.P. Morgan. Please go ahead.
I guess estimate how how the shift in.
And the environment as patients are starting to see their doctors a little bit more actively.
Eric William Joseph: Thanks for taking the questions. Good morning, and congratulations again on the role, Richard. I will do my best not to call you Paul Richardson.
With vaccination is going up as the year goes on.
Eric William Joseph: Just a couple of questions on recent prescription trends and looking forward here. From the first quarter, growth seems pretty strong. Can you comment on demand trends through April so far? And are you able to perhaps guide us a little bit on second quarter expectations? and then just follow up on sort of where you're seeing use right now, you know that with the once weekly regimen, with most of the demand being for the once weekly regimen just as a combination use, you know, expand into earlier line use where you might see the benefit of, Thanks for taking the time to join us. Eric, it's Michael.
How could that number shift going forward.
And how should we look at that when we model as.
As far as what type of demand.
Could change.
Going forward.
I think it was interesting to see and the first quarter. When you look at all the multiple myeloma brands that have reported this quarter to date as Michael mentioned, it seems to be a down quarter in terms of multiple myeloma. We would expect as patients are vaccinated that patients would return to see there.
Michael P. Mason: Listen, we're not going to comment on this quarter's growth at all at this point, and we'll obviously update you when we can, but I will turn it over to John for the second part of the question. Now, looking at our dosing, thanks for your question there, Eric. Just as an example, last year in Q1, over 50% of our new patients were being started on the twice-a-week 80-milligram dose, or 160 milligrams per week, which was the starting dose in the storm population. In this most recent quarter, only 15% of patients were started on that dose, with the remaining 85% being started on a once-weekly dose, most commonly either 100 milligrams or 80 milligrams.
Our physicians that they will be getting these therapies. So we would expect the market to return to growth, we would expect to benefit from that market growth as well as obviously incremental growth above that with the continued launch and success in driving uptake in earlier lines with the Boston indication.
Thanks for answering my questions.
Thanks for your question.
The next question comes from Jonathan Chang from SVP Leerink. Please go ahead.
Good morning, and thanks for taking my questions.
First question given it sounds like the majority of revenue currently is still coming from the later lines storm setting.
John Demery: And while we can't be exactly sure which line of treatment these prescriptions are being generated in, syndicated market research we purchased suggests many of them are being prescribed with other multiple myeloma drugs, such as Velcade from the Boston study, or with Pomelos or Darzalex, which are in the NCCN guidelines, or with Kyprolis, which has been studied in the STOP trial. So we continue to see triplet use expand, most likely in later-line populations now, and we do expect that to move earlier as patients get more comfortable using Expovia with the different triplet combinations. Of course, I mentioned a couple of different drugs listed in NCCN. As a company, we will only promote the XBD indication, which is our own label. Okay, I got it.
Or are the later line setting period can you speak to reasons for confidence that you'll be able to successfully penetrate the earlier line settings and see the benefits for longer duration of treatment in the second half sales numbers.
So maybe I'll start and then I'll turn it to John Richard May have a comment.
The main the main driver here is the education of physicians physicians for the last five years and have assembled a series of multiple different triplet therapies typically that they use and per our second third fourth line settings, and they're comfortable with those where a new entry.
While this is not the unmet medical need that we had when we went into penta refractory. We do have one of the simplest if not the simplest.
Eric William Joseph: Thanks for taking the question. The next question comes from David Leibowitz from Morgan Stanley. Please go ahead.
Triplet therapy in terms of administration time required for a patient to come into clinic and so on of all the drugs. So what youre doing now is having to educate doctors that theres a new kid on the block and then it's quite simple it's easy.
David Leibowitz: Thank you very much for attending. Is it fair to say that almost all the... from the expanded label, or is there some? I think it's fair to say that the 17% demand growth was driven primarily by new patient starts in multiple myeloma, with most of that being an increase in triplet usage. So driven by the new indication that we've seen. How do you expect, um, I guess. Is there any way to qualitatively...
And with the proper prophylaxis it can be very manageable with patients on this combination as you know for over two years now.
So that's the main driver here and I think as John mentioned with with our sales force getting back face to face.
David Leibowitz: I guess estimate how the shift will affect the environment as patients are starting to see their doctors a little bit more actively. Unknown Speaker.
Unknown Speaker: Unknown Speaker How could that number, far as what type of, I think it was interesting to see in the first quarter when you look at all the multiple myeloma brands that have reported this quarter to date, as Michael mentioned, it seems to be a down quarter in terms of multiple myeloma. We would expect, as patients are vaccinated, that patients would return to see their physicians, and that they will be getting new therapies.
We will be able to change practice and guide physicians that there is a new option out there John do you want to yeah. I think Michael is point about education is critical and we're also deploying a number of new tools to help continue to reinforce and drive that education. So we're deploying interactive content that can be used in person or via virtual <unk>.
Unknown Speaker: So we would expect the market to return to growth. We would expect to benefit from that market growth as well as obviously incremental growth above that with the continued launch and success and driving uptake in earlier lines with the Boston indication. Thanks, Francis.
<unk> by both our sales force and our marketing team. In addition, we recently made significant investments in our digital ecosystem to improve that physician education, including website enhancements K O L videos banner ads and search engine marketing search engine optimization and digital media social networking edited media.
Unknown Speaker: Thanks for your question. The next question comes from Jonathan Chang from SVB LearLink. Please go ahead.
Jonathan Chang: Good morning, and thanks for taking my questions. First question, given it sounds like the majority of revenue currently is still coming from the later line setting or the later line setting period, can you speak to reasons for confidence that you'll be able to successfully penetrate the earlier line settings and see that benefits for longer duration of treatment in the second half? And maybe I'll start and then I'll turn it to John and Richard may have a comment. The main driver here is education.
Pilots and plan to continue to expand its substantially going forward to drive that education that Michael spoke to as being critical.
So investing in significant peer to peer education via both live and digital channels and finally beyond just branded we're continuing to enhance disease state education around the role of ex <unk> inhibition in cancer, including in multiple myeloma. There are a number of things that we're doing to continue to drive that education in the second half of the year.
Michael P. Mason: Physicians for the last five years have assembled a series of multiple different triplet therapies, typically, that they use in first, second, third, fourth line settings, and they're comfortable with those. We're a new entry. While this is not the unmet medical need that we had when we went into Pentarepractory, we do have one of the simplest, if not the simplest, triplet therapy in terms of administration, time required for a patient to come into the clinic, and so on, of all the drugs.
Second quarter and second half.
Got it. Thank you and just one follow up to that can you give us a sense of what you can and can't track and teasing out where the sales are coming from thank you.
Got it.
Yes.
Yes.
Well I'll just this is that because you can get Jonathan Yeah, I know not much has really changed there from for those who've been following our story for some time.
Michael P. Mason: So what you're doing now is having to educate doctors that there is a new kid on the block, and that it's quite simple, it's easy, and with the proper prophylaxis, it can be very manageable with patients on this combination, as you know, for over two years now. So that's the main driver here, and I think, as John mentioned, with our sales force getting back face-to-face, we will be able to change practice and guide physicians that there is a new option out there. John, do you want to go?
Essentially we're able to attract from from our distributor network, but certainly for about half of our business that goes through specialty distributors.
We can see them essentially just what what dose of drug is being prescribed and how much per account and then for the other half of our business, we're able to see a bit more data where we can see are these new patients are they refills and that's where we get our refill data from and we can see if our these multiple myeloma patients or these D. L. B C. L P.
John Demery: Yeah, I think Michael's point about education is critical, and we're also deploying a number of new tools to help continue to reinforce and drive that education. For example, we're deploying interactive content that can be used in person or via virtual engagement by both our sales force and our marketing team. In addition, we recently made significant investments in our digital ecosystem to improve physician education, including website enhancements, KOL videos, banner ads, search engine marketing, search engine optimization, digital media, social networking, and competitive media pilots, and plan to continue to expand this substantially going forward to drive that education that Michael spoke to as being critical. We're also investing in significant peer-to-peer education via both live and digital channels.
But none of this data Unfortunately gives us specifically what line of treatment. These patients are from and it doesn't tell us what specifically combination drugs.
Are being prescribed <unk>.
So all of that information, we garner through to general market research or our intelligence with our sales force et cetera.
Yes.
The next question comes from Ed White from H C. Wainwright. Please go ahead.
Good morning, and congratulations to Richard and.
Thank you and congratulations to Michael and Sharon as well.
So just two pipeline questions. The first is on export.
03503, or four in Myelofibrosis I was just wondering if we can get a status update and perhaps.
John Demery: And finally, beyond just branded, we're continuing to enhance disease state education around the role of XCO1 inhibition in cancer, including in multiple myeloma. So a number of things that we're doing to continue to drive that education in the second half of the year. Second quarter and second half.
When we can see data from those studies and the second question is on the solid tumor studies and just wanted to get an update there.
G B M.
Non small cell lung cancer and.
Jonathan Chang: Got it. Thank you. And just one follow-up to that. Can you give us a sense of what you can and can't track in teasing out where these sales are coming from? Thank you. Yeah. Well, this is Ian.
Colorectal cancer any any kind of update you can give us there on status.
Timing to data would be appreciated as well thank you.
Ian Karp: Jonathan, not much has really changed there for those who have been following our story for some time. Essentially, we're able to track from our distributor network, certainly for about half of our business that goes through special distributors, we can see essentially just what dose of drug is being prescribed and how much per account. And then for the other half of our business, we're able to see a bit more data where we can see, are these new patients, are they refills? And that's where we get our refill data from.
Hi, Ed.
We will update on the I know that I know the 34 and 35 Myelofibrosis studies are on clinical trials Gov.
When we have our first patients enrolled we will announce those as typical.
We won't be able to give much in the way of data expectations, just given accrual rates and so on and so forth.
But we're quite encouraged about.
The underlying basis for doing those studies and hopefully this year you guys will see some data in that regard for the solid tumor studies. The same thing applies the colorectal and lung studies are ongoing and we hope to provide updates at medical meetings went up when appropriate hopefully this year.
Ian Karp: And we can see, are these multiple myeloma patients, or are these DLBCL patients? But none of this data, unfortunately, gives us specifically what line of treatment these patients are on. And it doesn't tell us what specifically combination drugs are being prescribed. So all of that information we garner through general market research or intelligence with our sales force, et cetera. The next question comes from Ed White from H.C. Wainwright. Please go ahead.
And force the GBM study similar although I think we've just initiated as you know some of the new cohorts and frontline combination.
And in relapsed in combination with a lot less team. So those will take a little bit longer and I would expect maybe next year to see them, but we'll have to see.
Okay.
Great.
The next question comes from calling Cosi from Baird. Please go ahead.
Edward Patrick White: Good morning and congratulations to Richard and, you know, and a thank you and congratulations to Michael and Sharon as well. So just two pipeline questions. The first is on export 035 and 034 in myelofibrosis. I was just wondering if we can get a status update and, perhaps, when we can see data from those studies. And the second question is about solid tumor studies, and I just wanted to get an update there on GBM, non-small cell lung cancer, and colorectal cancer.
Hi, good morning, Thanks, so much for taking our questions and congrats L team.
If you could comment on what the inventory stocking levels as of the end of <unk> and then when you're thinking about competition in the later lines of multiple myeloma and what are some of the features that expose neo or debt.
Teachers have exposure that really resonates with the prescribers from thinking relative to competitors.
But Mike Mason will discuss the inventory sure. So as Michael was earlier on the call, we had 17% demand growth, but 7% ex factory go from part of that was gross to net and part of the other difference. It was some stocking that happened in December of 2020, Yes. If you recall we've got the.
Edward Patrick White: Any kind of updates you can give us there on the status and time to data would be appreciated as well. Hi Ed, we will update on the. I know that I know the 34 and 35 myelofibrosis studies are on clinicaltrials.gov. When we have our first patients enrolled, we will announce those as typical. We won't be able to give much in the way of data expectations, just given the accrual rates and so on and so forth.
The Boston approval in mid to mid to late December yourself from stocking in December so that.
Sorry, we didn't see much of a benefit of that in Q1, so that led us somewhere around four ish weeks of inventory, which is pretty consistent where we've had in prior quarters.
Great John will take the second part.
And in terms of what.
Customers are looking for you asking and later lines of therapy actually you know our goal with the day Boston data is to move into earlier lines of therapy, and what we've seen there in multiple myeloma physicians. Many physicians believe it is important to treat with different mechanisms as early as possible in the patients of course of disease and Thats one of the key.
Michael P. Mason: But we're quite encouraged about the underlying basis for doing those studies, and hopefully, this year, you guys will see some data in that regard. For the solid tumor studies, the same thing applies. The colorectal and lung studies are ongoing, and we hope to provide updates at medical meetings when appropriate, hopefully this year, and for the GBM study, similar, although I think we've just initiated, as you know, some of the new cohorts in frontline combination and in relapsed in combination with the Lomas team. So those will take a little bit longer, and I would expect maybe next year to see them, but we'll have to see.
Vantage is exposed to a <unk> is a novel mechanism of action that is synergistic with Proteasome inhibitors like Velcade in the indicated ex P D regimen.
We also know that they are very focused on efficacy and we're able to communicate with the ex U D regimen rapid and sustained PFS benefit as well as clinically significant durable responses all attributes that are very important to the customers.
Colleen Margaret Kusy: Great. The next question comes from Colleen Kusy from Baird. Please go ahead. Hi, good morning.
Colleen Margaret Kusy: Thanks so much for taking our questions and congratulations to the whole team. If you could comment on what the inventory stocking level is as of the end of 1Q, and then when you're thinking about competition in the later lines of multiple myeloma, what are some of the features of Expovio that really resonate with prescribers when thinking relative to competitors? Mike Mason will discuss the inventory. Sure. So as Michael mentioned earlier in the call, you know, we had 17% demand growth but 7% X factor growth, and part of that was gross net.
I do think it's important to note when you look at the landmark Boston study, it's the only trial out there to date study Velcade weekly the way, it's actually used by clinicians and clinical practice and despite using that weekly dose or half the dose of other trials still produced very strong data per patient.
Those are some of the attributes that are physicians are looking for and we're highlighting as they work to move ex EDF in earlier lines of therapy.
Great. Thank you that's very helpful and if I can quickly ask a follow up if it's the end of the data are positive how much incremental investment would you expect you'll need to make in your commercial infrastructure to expand into solid tumors.
Michael P. Mason: And part of the other difference was some stocking that happened in December of 2020. If you recall, we got the Boston approval in mid to late December. So we saw some stocking in December.
Yeah I'll go ahead Jonathan.
Michael P. Mason: So we didn't see much of a benefit of that in Q1. So that led us somewhere around, you know, four-ish weeks of inventory, which is pretty consistent with what we've had, you know, in prior quarters. Great, John will take the second part.
We're actually doing that assessment right now.
To look at overlap of accounts and the size of the commercial platform and footprint that we would need to fully maximize that that's something that we'll be able to provide more data to you at a later point in time.
John Demery: Yeah, in terms of what customers are looking for, you ask in later lines of therapy. Actually, you know, our goal with the Boston data is to move into earlier lines of therapy. And what we've seen there in multiple myeloma, physicians, many physicians believe it's important to treat with different mechanisms as early as possible in the patient's course of disease. And that's one of the key advantages Expovio brings is a novel mechanism of action that is synergistic with proteasome inhibitors like Velcade and the indicated XVD regimen.
Great. Thank you.
The next question comes from Arlinda Lee from Canaccord. Please go ahead.
Hi, guys. Thanks for taking my questions.
I guess I was curious about on the education front.
I'm curious what you guys are encountering is what the disconnect is always I heard a list from prescribers to prescribe more into the Boston trial or Paul.
John Demery: We also know that they're very focused on efficacy and were able to communicate with the XVD regimen's rapid and sustained PFS benefit, as well as clinically significant durable responses, all attributes that are very important to customers. And I do think it's important to note when you look at the landmark Boston study, it's the only trial out there to date to study Velcade weekly the way it's actually used by clinicians in clinical practice. And despite using that weekly dose or half the dose of other trials, they still produce very strong data for patients. So those are some of the attributes that our physicians are looking for.
Austin patient population.
Yes.
Yeah, I'll start and John can add I don't think there's really a disconnect again I think it really has to do with debt.
Debt you do things that you are used to doing doctors are very much.
You know theyre confident with what regimens they add prior to the Boston approval, they've been working on them and owning them for the last five years, which is really the last time that a major second line new indication, our new drug came out and.
So they're just comfortable it's a little like before we all use seatbelts nobody really wanted to put it on but you eventually get used to it and then it becomes second nature. So what we have to do is make the Boston regimen second nature to physicians and that's going to take a little bit of time.
Colleen Margaret Kusy: And we're highlighting this as we work to move XVD up into earlier lines of therapy. Great, thank you. That's really helpful. And if I can quickly ask a follow-up question, if CNDA data are positive, how much incremental investment would you expect you'll need to make in your commercial infrastructure to expand into solid tumors? We're actually doing that assessment right now to look at overlap of accounts and the size of the commercial platform and footprint that we would need to fully maximize that. So that's something that we'll be able to provide more data to you at a later point. Great, thank you. The next question comes from Arlinda Lee from Canaccord.
But I don't think there is a problem here at all when the doctors typically most doctors when they use this regimen. They are pleasantly surprised when they use proper prophylaxis, we get a number of doctors, calling us and saying well the patients didn't have any nausea.
And we asked them what they did a nice day, while I used what you suggested in and that's how it all works. So we know how to we know how to prevent the side effects from this drug and a lot of them.
Arlinda Lee: Hi, guys. Thanks for taking my questions. Um, I guess I was curious about on the education front. I'm curious what you guys are encountering, what the disconnect is or the hurdle is for prescribers to prescribe more into the Boston trial or Boston patient population. Thank you.
Our reversible and we just have to change.
Habits.
I think Michael absolutely right. Some physicians have the habit based on the storm data as you know that debt.
Michael P. Mason: I'll start, and John can add. I don't think there's really a disconnect. Again, I think it really has to do with doing things that you're used to doing. Doctors are very much, you know, confident with what regimens they had prior to Boston approval. They've been working on them and honing them for the last five years, which is really the last time that a major second line in a new indication or new drug came out. And so they're just comfortable. It's a little like, you know, before we all used seatbelts; nobody really wanted to put them on, but you eventually got used to them.
Once weekly dosing regimen, and the Boston is better tolerated it shows a higher level of efficacy. So it's just getting physicians to try that new regimen versus the original regimen. We watch list continue that education process.
Okay.
There are no more questions from the queue. This concludes our question and answer session I would like to turn the conference back over to Richard Polson for any closing remarks.
Michael P. Mason: And then it becomes second nature. So what we have to do is make the Boston regimen second nature to physicians, and that's gonna take a little bit of time. But I don't think there's a problem here at all.
I'd like to thank everybody again for joining today's call and we look forward to updating you on our progress as soon as again. Thank you operator from close to Paul.
Michael P. Mason: Typically, most doctors, when they use this regimen, they're pleasantly surprised. When they use proper prophylaxis, we get a number of doctors calling us and saying, well, the patient didn't have any nausea. And we, you know, we asked them what they did, and they said, well, I used what you suggested, and that's how it all works. So we know how to prevent these side effects from this drug. A lot of them, they are reversible, and we just have to change people's habits.
The conference has now concluded. Thank you for attending today's presentation you may now disconnect.
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John Demery: I think Michael is absolutely right. Some physicians have a habit based on the STORM data. As you know, the once weekly dosing regimen in Boston is better tolerated and shows a higher level of efficacy.
John Demery: So it's just getting physicians to try that new regimen versus the original regimen we launched with to continue that education. There are no more questions in the queue. This concludes our question and answer session. I'd like to turn the conference back over to Richard Paulson for any closing remarks. I'd like to thank everybody again for joining today's call, and we look forward to updating you on our progress as soon as we can. Thank you, operator. We'll close the call. The conference is now concluded. Thank you for attending today's presentation. You may now disconnect. The Bulletproof Executive, 2013
Richard A. Paulson: For more information visit www.publichealth.org ??? ??? ??? ??? ??? ??? ??