TransAlta Q2 2024 Earnings Call Transcript

Quartr
Provided by Quartr
August 7, 2024

There are 13 speakers on the call.

Operator

Good morning. My name is Gigi, and I'll be your conference operator today. I would like to welcome everyone to the SpringWorks Therapeutics Second Quarter 2024 Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' remarks, there will be a question and answer session.

Operator

Please limit your questions to 1. Thank you. I would now like to hand the conference over to Samantha Sandler, Senior Director of Investor Relations at SpringWorks Therapeutics. Samantha, you may now begin the conference.

Speaker 1

Thank you, and good morning, everyone. Welcome to SpringWorks Therapeutics' Q2 2024 Earnings Conference Call. This morning, we issued a press release, which outlines our financial and operational results for the Q2. You can access the press release as well as the slides that we will be presenting today by going to the Investors and Media section of our website

Speaker 2

atwww.springworkstx.com.

Speaker 1

Joining me today are Saqib Islam, Chief Executive Officer Babesh Asher, Chief Commercial Officer Doctor. Jim Cassidy, Chief Medical Officer Frank Perrier, Chief Financial Officer and Doctor. Badri Din Idris, Chief Operating Officer. Before we begin, I would like to remind you that some of the statements made during the call today are forward looking statements that are subject to a number of risks and uncertainties. This may cause our actual results to differ materially, including those described in our reports filed with the SEC.

Speaker 1

You are cautioned not to place any undue reliance on these forward looking statements, and SpringWorks disclaims any obligation to update such statements. I will now turn the call over to Saket.

Speaker 3

Thank you, Sam, and thank you all for joining this morning. I'm pleased to share our 2nd quarter results and how we are delivering on our objective to make a profound impact on the lives of patients with devastating diseases. Starting with on ZIVIO for patients with Desmoid tumors, we are encouraged by the strong momentum of our U. S. Launch.

Speaker 3

In the Q2 of 2024, we reported $40,200,000 in net product revenue. This robust and continued growth is driven by the transformative clinical benefits from ONXIVIO, high demand from patients and physicians across treatment settings and strong commercial execution. Since our FDA approval last November, ONZYVO has rapidly become the standard of care systemic therapy for patients with desmoid tumors. As Bhavesh will discuss in more detail, we are seeing steady growth in new patient starts as well as strong persistence from those who previously initiated ONXIVIO therapy. Importantly, patients are reporting rapid and meaningful symptomatic relief, which is contributing to their overall improved quality of life and their enthusiasm for OXIVIO for their treatment.

Speaker 3

We are pleased with our early launch experience and believe that we have only reached a small proportion of people with desmoid tumors who can benefit from ONXIVIO. Our confidence in the overall patient population has continued to increase as we have gained further insights from the launch and from emerging desmoid tumor specific claims data. Our conviction remains high in the opportunity we have in front of us to serve an increasing number of desmoid tumor patients. Turning to our investigational MEK inhibitor, mirdimetinib. In the Q2, we completed our NDA submission to the FDA for the treatment of children and adults with neurofibromatosis type 1 associated plexiform neurofibromas or NS1PN.

Speaker 3

Our NDA submission is based on positive data from our pivotal Phase 2b RENEW trial, which support the potential for miradamatinib to be a best in class therapy for both children and adults living with these devastating lifelong tumors. There is a substantial unmet need for the approximately 40,000 people living with NF1PN in the United States alone, 30,000 of whom are adults and 10,000 are pediatric patients. Challenges with administration and tolerability limit the use of currently available options for children and there are no FDA approved therapies for adults. We look forward to working with the FDA throughout our NDA review process and to advancing our commercial preparations in anticipation of our second medicine introduced to patients in 2025. We are also excited about the opportunities in our emerging portfolio.

Speaker 3

We have several important efforts in development to serve additional patient populations with high unmet needs, including patients with ovarian granulosa cell tumors, multiple myeloma and MAPK mutant solid tumors. In addition, we recently began enrollment in our Phase 1 study of our investigational T inhibitor SW682 in hipomutant solid tumors, which represent up to 10% of cancers. Finally, we have durable IP protections for our lead molecules and a robust balance sheet that we expect to fund us through profitability. I'll now hand the call to Bhavesh to discuss our progress with ONXIVIO for patients with desmoid tumors. Bhavesh?

Speaker 4

Thank you, Saket. I'm happy to share an update on the U. S. Launch of OXIVIO. 2 full quarters into our launch, Oxivio is already the most prescribed systemic therapy for adults with desmoid tumors.

Speaker 4

As Sakiv highlighted, in the Q2 of 2024, we generated $40,200,000 in net product revenue, driven by strong demand for Obsivio by patients and physicians across treatment sites, real world experience of Obsidio's meaningful benefits and focused commercial execution in delivering the 1st and only FDA approved medicine to the desmoid tumor community. We're seeing robust adoption with broad use aligned with our FDA approved label, including in newly diagnosed patients and in those seeking their next line of therapy after unsatisfactory outcomes with previous interventions. Physicians at Centers of Excellence were strong early adopters of Obsivio. The majority of them have now treated multiple patients and continue to add new patients. We're also pleased with the continued uptake amongst physicians at other academic and community centers, where we are also seeing steady new patient starts.

Speaker 4

In addition, we've received consistent feedback from both patients and physicians on Oxivio's life altering impact. Patients are experiencing rapid and significant symptom relief, notably pain reduction, helping them to get back to their daily lives. Enhancing the patient experience with Obsivio has been an area of focus this quarter. We're pleased with the successful introduction of the 150 milligram and 100 milligram strength OXIVIO tablets in convenient blister packaging. The new product format has seen strong adoption to date, validating the importance of this innovation for patients.

Speaker 4

Patients prescribed OXIVIO continue to have broad access with reimbursement across payers. Commercial and government insurers are broadly reimbursing Ocideo and formal coverage policies are in place for over 90% of commercially covered lives. Across the payer landscape, there is clear recognition of the clinical value of our medicine as an NCCN Category 1 preferred treatment. And in June, the Desmoid Tumor Working Group guidelines were published in JAMA Oncology, highlighting Oxidio's incorporation into the treatment algorithm as the first and only approved drug for desmoid tumors. In just 7 months on market, OXIDIO has become the systemic standard of care for desmoid tumors based on our analysis of prescription data, and we're excited to leverage this position of strength to reach many more patients with this disease.

Speaker 4

We've also continued to receive highly positive feedback from prescribers and patients on their experiences with Obsidio. In a recent survey of 110 oncologists, 95% of Obsivio prescribers expressed satisfaction with their experience. More than 85% of prescribers prefer it over off label systemics, which are known to have inconsistent efficacy and tolerability challenges, further supporting OXIVIO's position as the systemic therapy of choice. In addition, most physicians said that they are likely to use Obsivio as a frontline treatment, indicating treatment with Obsivio at the earliest opportunity for their adult patients. Further, we're hearing consistent feedback from patients of profound symptom relief on OXOVIO, including patients experiencing rapid and meaningful improvements in pain within days of initiating treatment.

Speaker 4

The strength of the real world feedback, which is consistent with our clinical trial experience, will continue to solidify OXIVIO as the standard of care systemic treatment for patients with desmoid tumors. Turning now to the opportunity we see ahead of us. There are up to 16 50 newly diagnosed patients annually. Our market research supports that the vast majority of physicians are likely to use OXIDEO as a frontline treatment and we're already seeing uptake of OXIVIO as the first intervention. Our research has previously pointed to up to 7,000 actively managed patients in the U.

Speaker 4

S. Each year. Our early launch results as well as desmoid tumor specific ICD-ten claims data have continued to validate the size of this currently addressable patient pool. And we now view this estimate as conservative. The role of systemic therapy is continuing to increase among patients who require active treatment with surgery being deprioritized in treatment guidelines.

Speaker 4

With Obsivio as the 1st choice systemic, we're confident that there continues to be a substantial opportunity for growth in this patient population. In addition, there's a sizable diagnosed prevalent population of 30,000 people overall. We know that the majority of desmoid tumor patients receive active intervention over the course of their disease, and hence a meaningful proportion of these patients who are currently on the sidelines could potentially be addressed with a new treatment option. We have seen robust adoption to date and continue to have strong conviction in the sizable opportunity ahead of us to continue serving adults with desmoid tumors at all stages of treatment. Our success thus far positions us well for continued momentum in the second half of the year and beyond.

Speaker 4

We're gratified to have earned the advocacy of desmoid tumor experts through their collaboration in the DEFY study as well as engagement following the approval of Oxavil. Our commercial efforts aim to amplify their voices to educate and improve outcomes for patients. This comes in the form of providing opportunities for these leaders to educate the broader desmoid tumor community, including physicians, nurses, advocates, patients and caregivers on guideline recommended treatment approaches. Importantly, this includes educating on the role of systemics, the limitations of surgical intervention and the availability of Obsivio as an FDA approved and NCCN Category 1 preferred treatment option. Education and peer to peer information and experience sharing are critical avenues to increasing awareness of Obsivio and will continue to support increased breadth and depth of prescribing.

Speaker 4

Continuing to address the needs of patients also remains paramount and we're highly focused on supporting positive experiences with Obsidio. In mid May, successfully introduced our 150 milligram and 100 milligram strength tablets in convenient blister packaging. This new product format was developed as an innovation for patients to increase convenience and adherence by reducing the daily pill intake and simplifying morning and evening dosing. We have seen a strong transition to this new format through the Q2 and expect this transition to continue over the next 3 months. Additionally, we're expanding our educational resources and delivering best in class patient services through SpringWorks Care Connections to further support the treatment journey with OXIDEO.

Speaker 4

We also continue to generate data to support the strong clinical profile of OXIVIO and are advancing our efforts to expand Obsivio into additional geographies. In the second quarter, we presented additional data from our Phase 3 DEFI trial at ASCO that reinforced and manageable safety profile of Obsidio, including in hard to treat subgroups such as patients with characteristics associated with poor prognosis and those with APC mutations, which can be a more aggressive form of the disease. In an oral presentation at ASCO, the PHY was also showcased as an exemplar of one of the most comprehensive assessments of ovarian function in an oncology clinical trial to date and a best practice for evaluating a drug's effect on ovarian function for future cancer trials in accordance with ASCO guidelines. This analysis included updated data on investigator reported resolution of ovarian toxicity in DEFI, which further supported the transient nature of ovarian toxicity in both the majority of patients who stay on OXIVIO therapy and all those who stop treatment for any reason. Turning to upcoming milestones, We're working closely with the European regulators as they review our marketing authorization application and we are excited by the the a medical conference before the end of the year.

Speaker 4

In our Phase 1 and Phase 2 studies, patients remain on Obsivio for a median of over 4 years. And at the time of the Phase 3 DEFI trial data cutoff, median treatment duration was approaching 2 years. With these long term follow-up data from DEFI, we plan to provide an update on the continued anti tumor activity and overall clinical benefit provided by Obsivio with longer durations of treatment. The ability for patients to stay on therapy is critical given the persistent morbidities associated with this disease, and we believe these longer term results from DEFI will further support the potential for extended treatment durations. I'll now turn over to Doctor.

Speaker 4

Jim Cassidy, our Chief Medical Officer, to discuss the progress we're making with our MEK inhibitor miralumetinib in children and adults with NF1 PM. Jim?

Speaker 5

Thanks, Bhavesh. I'm glad to provide an update on our progress developing mildametinib as a potentially best in class therapy for patients with NF1PN, which is a highly morbid and lifelong disease that affects both children and adults. To start, there's a significant opportunity to improve outcome for these patients that are approximately 100,000 people in the United States living with NF1. These individuals have a 30% to 50% lifetime risk of developing clicks from neurofibromas, which are tumors that grow along peripheral nerve sheaths and can cause severe disfigurement, pain and functional impairment. We estimate that there are approximately 40,000 patients with NF1PN in the U.

Speaker 5

S. Today, the majority of whom are adults who currently do not have an approved therapy. As there is no specific demographic range for this disease, we estimate that there are a proportionate number of people living with NF1Pn outside of the United States as well. While systemic therapies are treatment of choice and MYC inhibitors have been clinically validated as a class in this indication, there are no formal treatment guidelines and our market research shows that only a small portion of NF1PN patients have been treated with a targeted therapy. We have done considerable work to understand the disease state, current treatment paradigm and physician and patient preferences.

Speaker 5

And taken as a whole, our research supports that patients are in need of new options. This is illustrated by a highly fragmented treatment landscape with significant use of off label therapies, even in pediatric patients for whom there is an approved medicine. People living with NF1PN have substantial needs that are not met by current options and bringing to market a new systemic therapy could allow many more patients to be treated. As we'll discuss, the positive results from our pivotal Phase IIb RENEW trial support nivnimetimib's potential to be a 1st in class therapy for adults with NF1PN and the best in class option for pediatric patients. The renewed results were presented at 3 medical conferences in the Q2, including during an oral session at ASCO.

Speaker 5

These presentations are improving awareness and building enthusiasm for our positive results amongst the physician community. The efficacy data across both adult and pediatric cohorts in RENEW showed significant reductions in the size of PN tumors with robust objective response rates confirmed by blinded independent central review. In addition, we saw very deep responses that have not been seen before in published studies of this disease. The majority of patients with a confirmed objective response experienced a tumor reduction of greater than 50% and reductions of up to 90% were seen in both cohorts. This is particularly significant when you consider the hard to treat patients that were enrolled in our study.

Speaker 5

Importantly, physician feedback suggests that these are unprecedented data and have the potential to be a meaningful differentiator. In both children and adults, nidometinib demonstrated a manageable safety profile with low rates of Grade 3 adverse events and low discontinuation rates due to AEs. We believe nadametinib's tolerability profile supports the potential for extended treatment duration, which is important in a chronic disease like NF1Pn, where there are high rates of recurrence after stopping therapy. In RENEW across both cohorts, the median time on treatment was approaching 2 years with some patients on merimedinib therapy for nearly 4 years at the time of the primary analysis. Nearly all patients that completed the treatment phase continued on therapy in the long term follow-up portion of the study.

Speaker 5

The potential for mivinumetinib to enhance patient quality of life is also very meaningful. Pain is a common symptom in NF1PN and current clinical practice recommendations indicate that this is a critical factor in treatment initiation decisions. Further, in many cases, the primary goal of treatment is improvement of such PN associated morbidities. Pain was the most commonly reported baseline morbidity in the RENEW trial, So it's significant that both adults and children, including those with the most ability to take in pain, reported early sustained and clinically significant reductions in pain severity and pain interference over the course of mivimetinib's treatment. Lastly, we believe that nivimetinib's convenient dosing regimen, which provides a built in treatment holiday and a pediatric friendly dispersible formulation to further optimize the patient experience and potentially enhance compliance.

Speaker 5

In RENEW, children and caregiver reports were highly positive in this regard with maximum acceptability scores for ease of swallowing and willingness to continue to take the dispersible tablet. The totality of the renewed data reinforces our belief that mirmimetinib can address strong desire amongst both physicians and patients for a new therapy that offers robust tumor and symptomatic control, a manageable safety profile and convenient dosing regimen, all of which we expect will enable patients to remain on therapy for an extended period of time. Looking ahead, we are making meaningful progress advancing ridimetinib towards potential regulator approval in 2025. Mirdametinib was granted orphan drug designation by both the FDA and the European Commission and the FDA also granted Fast Track and rare pediatric disease designations for NF1PN. We completed our NDA submission to the FDA at the end of June and we anticipate announcing our PDUFA date in the coming weeks.

Speaker 5

We're also on track to complete the submission of our European marketing authorization within this half. In addition, the renewed results will continue to be showcased at upcoming medical congresses, and we expect the results to be published in a peer reviewed journal later this year. And now Frank Perrier, our Chief Financial Officer will discuss our Q2 financial results. Frank?

Speaker 6

Thank you, Jim. I'll now summarize a few highlights from our Q2 2024 financial results. Starting with revenues,

Speaker 7

we recorded

Speaker 6

$40,200,000 of OXIVIO net product revenue in the 2nd quarter. This brings our 2024 year to date OXIVIO net product revenue to $61,200,000 Our total operating expenses increased compared to the second quarter and the first half of twenty twenty three, driven by the commercial activities to support the U. S. Launch of OXIVIO and the anticipated U. S.

Speaker 6

Launch of mirimetinib in both adults and children with NF1PN. We have a strong balance sheet supporting our clear path to profitability with $522,000,000 in cash, cash equivalents and marketable securities as of the end of the second quarter. Lastly, we have a durable operating plan designed to fund multiple global product launches and to enable the continued investment and expansion opportunities across our pipeline. With that, I'll hand the call back over to Sakam.

Speaker 3

Thank you, Frank. As we've discussed this morning, we are very pleased with the continued strong momentum of our U. S. Launch of ONCEVIO for adults with desmoid tumors and are working with urgency to bring mirdimetinib to children and adults with NF1Pn. In parallel with these commercial efforts, we are enthusiastic about our emerging portfolio.

Speaker 3

Nirogastatin is being studied in Phase II trial in ovarian granulosa cell tumors, which is fully enrolled. These tumors account for approximately 5% of all ovarian cancers. And like desmoid tumors, this is a meaningful patient population for which there are no FDA approved therapies. In multiple myeloma, we've established clinical proof of concept for niragastat in combination settings with several BCMA directed agents and are continuing to collaborate with a number of industry and academic partners to further this strategy. We're also pursuing expansion opportunities for miradamatinib in several monotherapy and combination therapy settings.

Speaker 3

Promising data from a Phase III study of miradamatinib in pediatric low grade glioma were recently presented at the International Symposium on Pediatric Neuro Oncology Meeting and several combination studies are currently ongoing, including a trial with MAPTURE's brimurafenib in advanced solid tumors with MAP kinase mutations and with BeiGene's lifurafenib in NRAS mutant solid tumors. Bimberafenib is also being explored as a monotherapy in adults with the RAF mutant solid tumors, and we're looking to confirm the efficacy signal we reported last year in the dose escalation study in several ongoing dose expansion cohorts. Additionally, SW682, our oral TID inhibitor is now in the clinic. This program is designed to treat tumors with hippo pathway mutations, which can occur in up to 10% of cancers, including mesothelioma and head and neck cancer. We believe that there is a meaningful opportunity with SW-six eighty two for SpringWorks to develop a novel medicine for these cancers.

Speaker 3

Lastly, we are also advancing several internally discovered preclinical stage programs and we look forward to sharing more about these efforts as they progress. To close, we are very pleased with our accomplishments in the first half of this year and have multiple milestones and data readouts ahead that we look forward to sharing. These include long term follow-up data from our DEFY trial, which we believe will support the benefits of extended treatment durations with OXIVIO in desmoid tumors. It also includes initial data from our Phase 2 trial of niragastat in ovarian granulosa cell tumors, and we expect the full RENEW trial results to be published in a peer reviewed journal. Our focus for the remainder of the year will be to continue building on ONZIVIO's momentum in the U.

Speaker 3

S, while working to bring this medicine to patients in Europe and other geographies. To advance our regulatory and commercial preparations for mirdumetinib in anticipation of serving patients with NF1Pn in the U. S. And Europe and to progress our diversified emerging portfolio in an efficient manner. We are confident that our strong foundation will support our continued success as we deliver on our mission to change the lives of people suffering from devastating diseases.

Speaker 3

As always, I would like to thank the patients and investigators who participated in our clinical trials, our patient advocacy partners and collaborators and our team of spring workers. Are now happy to take questions. Operator?

Operator

Thank you. We will now open the call for questions. Our first question will come from Anupam Rama at JPMorgan.

Speaker 8

Hey, guys. Congrats on all the progress. Just a quick one for me. What are you seeing from both new prescribers and repeat prescribers for Obsivio? And in the early innings, the wait and watch population that's coming in to seek treatment for Obsivio?

Speaker 8

Thanks so much.

Speaker 3

Thanks, Anupam. I'll let you take that.

Speaker 9

Yes. So thanks, Anupam. On the repeat versus new, obviously, we saw a good amount of new prescribers in the initial months of the launch. As you'd expect, we've started to see refills coming in at a regular pace from these initial starts, but supported by continued new patient starts from new patients being acquired in the Q2 as well. As you'd expect, the total proportion of business coming from existing patients versus new patient starts is increasing over time.

Speaker 9

And so that's a positive development. From a which and what perspective, we do know obviously that the proportion of the DT patient population, there is is under active surveillance at any point in time. However, we do know that over 90% of these patients receive some active intervention during the course of their disease. Now, with the availability of the 1st and only FDA approved treatment, the opportunity ahead of us and supported by treatment guidelines which favor systemic therapy supported by the positive experiences that patients and physicians are seeing without Xivio in the early innings of the launch, the opportunity ahead of us is to try and drive faster treatment for these patients under active surveillance. And so we do expect the proportion of active surveillance patients to be dropping over time.

Operator

Our next question comes from the line of Yaron Werber from TD Cowen.

Speaker 10

Great. Thanks for taking my question. Congrats also on a nice quarter. Maybe I have a kind of a couple of interrelated questions. One, can you just give us a sense on inventories for the quarter for Exzeovio?

Speaker 10

And then secondly, just a follow on Anupam's question, it's one would imagine that the academic centers are probably the early adopters and they're moving kind of quickly as patients are coming in to use Obsivio. What are you seeing in the community? Maybe just give us a little bit of sense of kind of the breadth of usage? Thank you.

Speaker 3

Prabhush, I'll turn to you again

Speaker 5

for that. Yes.

Speaker 9

Thank you. So from an inventory standpoint, the overall impact of inventory in the second quarter was minimal. The stocking levels were generally aligned with the demand that we're seeing. And similar to prior quarters, inventory was not a key driver of performance this quarter. From a community dynamic perspective, 1st of all, I'll say that from as we said in our prepared comments, there is a deeper pool of patients overall than we originally estimated.

Speaker 9

And we started to break into this aggregate pool of patients that are of greater than 7,000 actively managed patients. As you highlighted, we're pleased with the uptake in the centers of excellence. We saw breadth of prescribing in the early months of the launch. We saw depth of prescribing in the Q2. Specifically addressing your question around community, we've started to see good adoption in the community centers where we continue to drive breadth and but we still have an opportunity, right.

Speaker 9

With the broad pool of patients, we have an opportunity both to continue to drive breadth of prescribing, but also those who experience, who do prescribe OXIVIO to drive depth of prescribing. Now I'll highlight a couple of things in that regard. First of all, our ability to be in the right place at the right time for these community clinics has been significantly enhanced by the availability of the desmoid tumor specific ICD-ten codes. And so we're able to direct our sales team to be in those clinics when a patient is available for treatment. And then secondly, the educational efforts that we're driving both around the availability of ObsEva as the first and only treatment option for desmodemar patients, but also treatment guidelines which do favor systemic therapy gives us confidence in the ability for us to drive depth in that community segment as well.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Corinne Johnson from Goldman Sachs.

Speaker 2

Hey, good morning guys. I guess you've talked a little bit about having some more visibility on this patient population given the ICD-ten codes. And potentially as you kind of move to these blister packs in the forward. I guess as you look at that, where have you identified the pockets where you see kind of the greatest opportunity for near and intermediate term growth? And what are you doing tactically to reach that patient population and keep them on therapy?

Speaker 2

And then I did want to ask also on meridamatinib, maybe with the launch next year, where you see kind of like the low hanging fruit with respect to initial adoption? And how should we think about the trajectory of the early launch compared to what you've accomplished here with Exhibio?

Speaker 3

Thanks, Corinne for the series of questions, but we're going to try and answer all of them. I think as we think of miradamatinib, we view that opportunity is actually quite meaningful. And as I think of low hanging fruit, I think we're very comfortable saying that this is an opportunity that is actually at least as large as what we see in Deshpande tumors. And that's driven by a number of factors. 1, by the size of the patient's population, right?

Speaker 3

We talked about 40,000 patients in the U. S. And a proportionate number outside the U. S. And a third of those are excuse me, a quarter of those are pediatric and 3 quarters of those are adults.

Speaker 3

So the opportunity for us to have a best in class treatment for pediatric and a first in class treatment for adults, we think gives us a meaningful target. 2nd, the physicians in this space actually themselves even in the pediatric setting believe that their patients are not being served as broadly as they could be. And so we think there's an opportunity there. And third, you can use KOSILUCO revenues, which now run rate just in the pediatric setting of about $500,000,000 annually, as a proxy for the opportunity just in that one quarter of the market. And as you know, we're filing for both adults and kids.

Speaker 3

So we think the opportunity beyond, I think low hanging fruit implies that it's all easy, none of it is, but you've got a medicine that people are waiting for an opportunity that is sizable and data that we believe supports a potentially best in class profile for mirinib for this disease. Abhishek, I'll turn to you to talk about OXIVIO.

Speaker 9

Yes. I'd just reiterate a couple of the points. 1st and foremost, we're fortunate to have with Obsivio to be in a position of delivering a drug with transformative benefit to desmonterumor patients, right? That is the foundation for the high awareness we have, the high satisfaction that we shared in the prepared comments as well as the strong preference that we see amongst prescribers and patients supported by broad access by the payer community. So with just 7 months on the market, we become the systemic standard of care and the most prescribed systemic therapy for adult patients with desmoid tumors.

Speaker 9

And so the opportunity for us just to continue to drive depth within the centers of excellence as well as drive depth and breadth in the community segment as I mentioned before. With the large pool of 7,000 more than 7,000 actively managed patients, we do see that we have a significant opportunity ahead of us to see continued growth for Acelvia.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Peter Lawson from Barclays.

Speaker 4

Great. Thanks for taking the questions. Thanks for the update as well and congrats on the progress. First question is, it's really just about the patient trends, how they can look in month over month and particularly into July, and then whether you're seeing any patients that are discontinuing? Thank you.

Speaker 3

Yes. Thanks for the question, Peter. I think we aren't seeing any exceptional discontinuations. It's what we would have expected from the DEFY study. And I think that gives us confidence going into not just the Q3, but the Q4 as well.

Speaker 3

So I think that the trend holds well. As Paves highlighted, we think the patient pool is actually deeper than what we had originally estimated. And I think that portends well for the aggregate opportunity here. Fundamentally, where we are perhaps most excited is the feedback that we're getting though. We've got patients seeing deep immediate and persistent benefits to their pain that is causing them to want to get on therapy and want to stay on therapy.

Speaker 3

So we believe that the opportunity here continues to be sizable.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Michael Schmidt from Guggenheim.

Speaker 11

Hey guys, good morning and congrats on a strong second quarter result. Just a follow-up on Obsivio. As we think about the Q3 here coming up, some other products sometimes experience seasonality over the summer months. Is that something you may expect as well with OXIVIO? And then a follow-up on mirdometinib as we think about the launch next year.

Speaker 11

Obviously, you have already a product on the market. Do you expect initial switching from coselugo over to mirdamatinib? Or would you expect a predominantly penetration into the currently untreated patients with NF1 as we think about that next year? Thanks so much.

Speaker 3

Thank you, Michael. So I'll take the OTIVIO question. I'll pass to Doctor. Yatrice to take the Mirabativ question. Yes, I don't think that we're immune from seasonality.

Speaker 3

I think as we see in many other products, I would expect to see some seasonality in the summer months. But obviously, as we've said from the outset, we think the pool is deep and we think the opportunity is significant. Anthony?

Speaker 7

Yes. So with respect to our opportunity within NF1PN for miritamatinib, I'll remind you that our NDA filing is for both pediatric and adult patients, the latter of whom represent 3 quarters of the market and do not have an approved therapy. So we think there's quite a bit of white space there with respect to being able to serve patients who are previously untreated. Within the pediatric population, I think there will certainly be some patients who have been treatment experienced over the years that are seeking their next line of therapy, but our market research shows that that market is minimally penetrated as well with respect to systemic therapy. So we think there's a significant number of pediatric patients who are also looking for their 1st systemic therapy option when mirabegnatib becomes available.

Operator

Thank you. One moment for our next question. Our last question will come from the line of Alex Stranahan from Bank of America.

Speaker 12

Hey guys, thanks for taking my question and congrats from us on the strong print. Any framing you can provide around the focus of the longer term follow-up data from DeFi later this year? And maybe one quick one if I can sneak it in. Is your expectation that you will receive accelerated review for mirvetinib? Just to clarify.

Speaker 12

Thank you.

Speaker 3

Thank you, Alec. I'll turn to Bethany for the answers.

Speaker 7

Sure. So with respect to the longer term follow-up data, Alec, I'll remind you that at the time of our primary analysis, the median time on treatment for patients that were randomized to the niragastat arm was just under 2 years and the majority of those patients were remaining on therapy. And so what we hope to be able to share an update on later this year is certainly a more mature median time on treatment figure for niryacastat treated patients as well as the trends that we see in terms of responses, how they evolve over time and the benefits that we are seeing with a longer dated data cut on the patient reported outcomes as well. So that's what we're gearing up to be able to share. And I'll remind you that in Phase 1 and Phase 2, our median time on treatment was over 4 years in each of those studies.

Speaker 7

So there's the opportunity to really present longer term data.

Operator

Thank you.

Speaker 7

With respect to any expectations on review timing and then review designations for the mirinib filing, we have not given any guidance on that topic. We'll be in a position to receive the PDUFA date from FDA about 60 days from filing. So that'll be sometime late in

Operator

August. Thank you. This concludes today's call. Thank you for joining. You may now disconnect.

Operator

Good morning. My name is Gigi, and I'll be your conference operator today. I would like to welcome everyone to the SpringWorks Therapeutics Second Quarter 2024 Earnings Conference Call. At this time, all participants are in a listen only mode. After the speakers' remarks, there will be a question and answer session.

Operator

Please limit your questions to 1. Thank you. I would now like to hand the conference over to Samantha Sandler, Senior Director of Investor Relations at SpringWorks Therapeutics. Samantha, you may now begin the conference.

Speaker 1

Thank you, and good morning, everyone. Welcome to SpringWorks Therapeutics' Q2 2024 Earnings Conference Call. This morning, we issued a press release, which outlines our financial and operational results for the Q2. You can access the press release as well as the slides that we will be presenting today by going to the Investors and Media section of our website

Speaker 2

atwww.springworkstx.com.

Speaker 1

Joining me today are Saqib Islam, Chief Executive Officer Babish Asher, Chief Commercial Officer Doctor. Jim Cassidy, Chief Medical Officer Frank Perrier, Chief Financial Officer and Doctor. Badgerdin Idris, Chief Operating Officer. Before we begin, I would like to remind you that some of the statements made during the call today are forward looking statements that are subject to a number of risks and uncertainties. This may cause our actual results to differ materially, including those described in our reports filed with the SEC.

Speaker 1

You are cautioned not to place any undue reliance on these forward looking statements, and SpringWorks disclaims any obligation to update such statements. I will now turn the call over to Saket.

Speaker 3

Thank you, Sam, and thank you all for joining this morning. I'm pleased to share our Q2 results and how we are delivering on our objective to make a profound impact on the lives of patients with devastating diseases. Starting with ONXIVIO for patients with desmoid tumors, we are encouraged by the strong momentum of our U. S. Launch.

Speaker 3

In the Q2 of 2024, we reported $40,200,000 in net product revenue. This robust and continued growth is driven by the transformative clinical benefits from ONXIVIO, high demand from patients and physicians across treatment settings and strong commercial execution. Since our FDA approval last November, ONZYVIO has rapidly become the standard of care systemic therapy for patients with desmoid tumors. As Bhavesh will discuss in more detail, we are seeing steady growth in new patient starts as well as strong persistence from those who previously initiated OXIVIO therapy. Importantly, patients are reporting rapid and meaningful symptomatic relief, which is contributing to their overall improved quality of life and their enthusiasm for OXIVIO for their treatment.

Speaker 3

We are pleased with our early launch experience and believe that we have only reached a small proportion of people with desmoid tumors who can benefit from ONXIVIO. Our confidence in the overall patient population has continued to increase as we have gained further insights from the launch and from emerging desmoid tumor specific claims data. Our conviction remains high in the opportunity we have in front of us to serve an increasing number of desmoid tumor patients. Turning to our investigational MEK inhibitor, mirdometinib. In the second quarter, we completed our NDA submission to the FDA for the treatment of children and adults with neurofibromatosis type 1 associated plexiform neurofibromas or NS1PN.

Speaker 3

Our NDA submission is based on positive data from our pivotal Phase 2b RENEW trial, which support the potential for miradamatinib to be a best in class therapy for both children and adults living with these devastating lifelong tumors. There is a substantial unmet need for the approximately 40,000 people living with NF1PN in the United States alone, 30,000 of whom are adults and 10,000 are pediatric patients. Challenges with administration and tolerability limit the use of currently available options for children and there are no FDA approved therapies for adults. We look forward to working with the FDA throughout our NDA review process and to advancing our commercial preparations in anticipation of our second medicine introduced to patients in 2025. We are also excited about the opportunities in our emerging portfolio.

Speaker 3

We have several important efforts in development to serve additional patient populations with high unmet needs, including patients with ovarian granulosa cell tumors, multiple myeloma and MAPK mutant solid tumors. In addition, we recently began enrollment in our Phase 1 study of our investigational T inhibitor SW682 in hipomutant solid tumors, which represent up to 10% of cancers. Finally, we have durable IP protections for our lead molecules and a robust balance sheet that we expect to fund us through profitability. I'll now hand the call to Bhavesh to discuss our progress with ONXIVIO for patients with desmoid tumors. Bhavesh?

Speaker 4

Thank you, Saket. I'm happy to share an update on the U. S. Launch of OXYvio. 2 full quarters into our launch, Oxivio is already the most prescribed systemic therapy for adults with desmoid tumors.

Speaker 4

As Sakiv highlighted, in the Q2 of 2024, we generated $40,200,000 in net product revenue, driven by strong demand for Obsivio by patients and physicians across treatment sites, real world experience of Obsivio's meaningful benefits and focused commercial execution in delivering the first and only FDA approved medicine to the desmoid tumor community. We're seeing robust adoption with broad use aligned with our FDA approved label, including in newly diagnosed patients and in those seeking their next line of therapy after unsatisfactory outcomes with previous interventions. Physicians at centers of excellence were strong early adopters of Obsidio. The majority of them have now treated multiple patients and continue to add new patients. We're also pleased with the continued uptake amongst physicians at other academic and community centers, where we are also seeing steady new patient starts.

Speaker 4

In addition, we've received consistent feedback from both patients and physicians on Oxivio's life altering impact. Patients are experiencing rapid and significant symptom relief, notably pain reduction, helping them to get back to their daily lives. Enhancing the patient experience with OXIVIO has been an area of focus this quarter. We're pleased with the successful introduction of the 150 milligram and 100 milligram strength OXIVIO tablets in convenient blister packaging. The new product format has seen strong adoption to date, validating the importance of this innovation for patients.

Speaker 4

Patients prescribed OXIVIO continue to have broad access with reimbursement across payers. Commercial and government insurers are broadly reimbursing Ocivio and formal coverage policies are in place for over 90% of commercially covered lives. Across the payer landscape, there is clear recognition of the clinical value of our medicine as an NCCN Category 1 preferred treatment. And in June, the Desmoid Tumor Working Group guidelines were published in JAMA Oncology, highlighting Oxidio's incorporation into the treatment algorithm as the 1st and only approved drug for desmoid tumors. In just 7 months on market, OXIDIO has become the systemic standard of care for desmoid tumors based on our analysis of prescription data and we're excited to leverage this position of strength to reach many more patients with this disease.

Speaker 4

We've also continued to receive highly positive feedback from prescribers and patients on their experiences with Obsivio. In a recent survey of 110 oncologists, 95% of Obsivio prescribers expressed satisfaction with their experience. More than 85% of prescribers prefer it over off label systemics, which are known to have inconsistent efficacy and tolerability challenges, further supporting OXIVIO's position as the systemic therapy of choice. In addition, most physicians said that they are likely to use Oxivio as a frontline treatment, indicating treatment with Oxivio at the earliest opportunity for their adult patients. Further, we're hearing consistent feedback from patients of profound symptom relief on OXIVIO, including patients experiencing rapid and meaningful improvements in pain within days of initiating treatment.

Speaker 4

The strength of the real world feedback, which is consistent with our clinical trial experience, will continue to solidify OXIVIO as the standard of care systemic treatment for patients with desmoid tumors. Turning now to the opportunity we see ahead of us. There are up to 16 50 newly diagnosed patients annually. Our market research supports that the vast majority likely to use OXIVIO as a frontline treatment and we're already seeing uptake of OXIVIO as the first intervention. Our research has previously pointed to up to 7,000 actively managed patients in the U.

Speaker 4

S. Each year. Our early launch results as well as desmoid tumor specific ICV10 claims data have continued to validate the size of this currently addressable patient pool, and we now view this estimate as conservative. The role of systemic therapy is continuing to increase among patients who require active treatment with surgery being deprioritized in treatment guidelines. With Obsivio as the 1st choice systemic, we're confident that there continues to be a substantial opportunity for growth in this patient population.

Speaker 4

In addition, there's a sizable diagnosed prevalent population of 30,000 people overall. We know that the majority of desmoid tumor patients receive active intervention over the course of their disease, and hence a meaningful portion of these patients who are currently on the sidelines could potentially be addressed with a new treatment option. We have seen robust adoption to date and continue to have strong conviction in the sizable opportunity ahead of us to continue serving adults with desalination tumors at all stages of treatment. Our success thus far positions us well for continued momentum in the second half of the year and beyond. We're gratified to have earned the advocacy of desmoid tumor experts through their collaboration in the DEFY study as well as engagement following the approval of Oxavil.

Speaker 4

Our commercial efforts aim to amplify their voices to educate and improve outcomes for patients. This comes in the form of providing opportunities for these leaders to educate the broader desmoid tumor community, including physicians, nurses, advocates, patients and caregivers on guideline recommended treatment approaches. Importantly, this includes educating on the role of systemics, the limitations of surgical intervention and the availability of Obsivio as an FDA approved and NCCN Category 1 preferred treatment option. Education and peer to peer information and experience sharing are critical avenues to increasing awareness of Obsivio and will continue to support increased breadth and depth of prescribing. Continuing to address the needs of patients also remains paramount and we're highly focused on supporting positive experiences with Obsivio.

Speaker 4

In mid May, we successfully introduced our 150 milligram and 100 milligram strength tablets in convenient blister packaging. This new product format was developed as an innovation for patients to increase convenience and adherence by reducing the daily pill intake and simplifying morning and evening dosing. We have seen a strong transition to this new format through the Q2 and expect this transition to continue over the next 3 months. Additionally, we're expanding our educational resources and delivering best in class patient services through SpringWorks Care Connections to further support the treatment journey with Oxivio. We also continue to generate data to support the strong clinical profile of Obsivio and are advancing our efforts to expand Obsivio into additional geographies.

Speaker 4

In the Q2, we presented additional data from our Phase 3 DEFI trial at ASCO that reinforced the robust efficacy and manageable safety profile of Auxidio, including in hard to treat subgroups such as patients with characteristics associated with poor prognosis and those with APC mutations, which can be a more aggressive form of the disease. In an oral presentation at ASCO, the file was also showcased as an exemplar of one of the most comprehensive assessments of ovarian function in an oncology clinical trial to date and a best practice for evaluating a drug's effect on ovarian function for future cancer trials in accordance with ASCO guidelines. This analysis included updated data on investigator reported resolution of ovarian toxicity in DEFI, which further supported the transient nature of ovarian toxicity in both the majority of patients who stay on OXIVIO therapy and all those who stop treatment for any reason. Turning to upcoming milestones. We're working closely with the European regulators as they review our marketing authorization application, and we are excited by the potential to receive approval from the European Commission in 2025.

Speaker 4

We also look forward to sharing long term follow-up data from Difei at a medical conference before the end of the year. In our Phase 1 and Phase 2 studies, patients remain on Observeo for a median of over 4 years. And at the time of the Phase 3 DEFI trial data cutoff, median treatment duration was approaching 2 years. With these long term follow-up data from DEFI, we plan to provide an update on the continued antitumor activity and overall clinical benefit provided by Obsivio with longer durations of treatment. The ability for patients to stay on therapy is critical given the persistent morbidities associated with this disease, and we believe these longer term results from DEFY will further support the potential for extended treatment durations.

Speaker 4

I'll now turn over to Doctor. Jim Cassidy, our Chief Medical Officer, to discuss the progress we're making with our MEK inhibitor mirabematinib in children and adults with NF1 PM. Jim?

Speaker 5

Thanks, Bhavesh. I'm glad to provide an update on our progress developing mabdometinib as a potentially best in class therapy for patients with NF1Pn, which is a highly morbid and lifelong disease that affects both children and adults. To start, there is a significant opportunity to improve outcome for these patients that are approximately 100,000 people in the United States living with NF1. These individuals have a 30% to 50% lifetime risk of developing flexible neurofibromas, which are tumors that grow along peripheral nerve sheaths and can cause severe disfigurement, pain and functional impairment. We estimate that there are approximately 40,000 patients with NF1PN in the U.

Speaker 5

S. Today, the majority of whom are adults who currently do not have an approved therapy. As there is no specific demographic ring for this disease, we estimate that there are a proportionate number of people living with NF1Pn outside of the United States as well. While systemic therapies are treatment of choice and MYC inhibitors have been clinically validated as a class in this indication, there are no formal treatment guidelines and our market research shows that only a small portion of NF1PN patients have been treated with a targeted therapy. We have done considerable work to understand the disease state, current treatment paradigm and physician and patient preferences.

Speaker 5

And taken as a whole, our research supports that patients are in need of new options. This is illustrated by a highly fragmented treatment landscape with significant use of off label therapies, even in pediatric patients for whom there is an approved medicine. People living with NF1PN have substantial needs that are not met by current options and bringing to market a new systemic therapy could allow many more patients to be treated. As we'll discuss, the positive results from our pivotal Phase IIb RENEW trial support nivnimetamib's potential to be a 1st in class therapy for adults with NF1PN and the best in class option for pediatric patients. The renewed results were presented at 3 medical consultancies in the Q2, including during an oral session at ASCO.

Speaker 5

These presentations are improving awareness and building enthusiasm for our positive results amongst the physician community. The efficacy data across both adult and pediatric cohorts in RENEW showed significant reductions in the size of PN tumors with robust objective response rates confirmed by blinded independent central review. In addition, we saw very deep responses that have not been seen before in published studies of this disease. The majority of patients with a confirmed objective response experienced a tumor reduction of greater than 50% and reductions of up to 90% were seen in both cohorts. This is particularly significant when you consider the hard to treat patients that were enrolled in our study.

Speaker 5

Importantly, physician feedback suggests that these are unprecedented data and have the potential to be a meaningful differentiator. In both children and adults, nivamatinib demonstrated a manageable safety profile with low rates of Grade 3 adverse events and low discontinuation rates due to AEs. We believe nadametinib's tolerability profile supports the potential for extended treatment duration, which is important in a chronic disease like NF1Pn, where there are high rates of recurrence after stopping therapy. In RENEW across both cohorts, the median time on treatment was approaching 2 years with some patients on mirimedinib therapy for nearly 4 years at the time of the primary analysis. Nearly all patients that completed the treatment phase continued on therapy in the long term follow-up portion of the study.

Speaker 5

The potential for mivinumetinib to enhance patient quality of life is also very meaningful. Pain is a common symptom in NF1PN and current clinical practice recommendations indicate that this is a critical factor in treatment initiation decisions. Further, in many cases, the primary goal of treatment is improvement of such PN associated morbidities. Pain was the most commonly reported baseline morbidity in the RENEW trial, So it's significant that both adults and children, including those with the most ability to take in pain, reported early sustained and clinically significant reductions in pain severity and pain interference over the course of mabimetinib's treatment. Lastly, we believe that mivamatinib's convenient dosing regimen, which provides a built in treatment holiday and a pediatric friendly dispersible formulation to further optimize the patient experience and potentially enhance compliance.

Speaker 5

In RENEW, children and caregiver reports were highly positive in this regard with maximum acceptability scores for ease of soloing and willingness to continue to take the dispersible tablet. The totality of the renewed data reinforces our belief that mamimetinib can address strong desire amongst both physicians and patients for a new therapy that offers robust tumor and symptomatic control, a manageable safety profile and convenient dosing regimen, all of which we expect will enable patients to remain on therapy for an extended period of time. Looking ahead, we are making meaningful progress advancing ridimetinib towards potential regulator approval in 2025. Mridametinib was granted orphan drug designation by both the FDA and the European Commission and the FDA also granted fast track and rare pediatric disease designations for NF1PN. We completed our NDA submission to the FDA at the end of June and we anticipate announcing our PDUFA date in the coming weeks.

Speaker 5

We're also on track to complete the submission of our European marketing authorization within this half. In addition, the renewed results will continue to be showcased at upcoming medical congresses and we expect the results to be published in a peer reviewed journal later this year. And now Frank Perrier, our Chief Financial Officer, will discuss our Q2 financial results. Frank?

Speaker 6

Thank you, Jim. I'll now summarize a few highlights from our Q2 2024 financial results. Starting with revenues, we recorded $40,200,000 of OXIVIO net product revenue in the 2nd quarter. This brings our 2024 year to date OXIVIO net product revenue to $61,200,000 Our total operating expenses increased compared to the second quarter and the first half of twenty twenty three, driven by the commercial activities to support the U. S.

Speaker 6

Launch of OXBIO and the anticipated U. S. Launch of mirinib in both adults and children with NF1PN. We have a strong balance sheet supporting our clear path to profitability with $522,000,000 in cash, cash equivalents and marketable securities as of the end of the second quarter. Lastly, we have a durable operating plan designed to fund multiple global product launches and to enable the continued investment and expansion opportunities across our pipeline.

Speaker 6

With that, I'll hand the call back over to Saket.

Speaker 3

Thank you, Frank. As we've discussed this morning, we are very pleased with the continued strong momentum of our U. S. Launch of ONCEVIO for adults with Desjoint tumors and are working with urgency to bring mirdometinib to children and adults with NF1Pn. In parallel with these commercial efforts, we are enthusiastic about our emerging portfolio.

Speaker 3

Nirogastatin is being studied in a Phase 2 trial in ovarian granulosa cell tumors, which is fully enrolled. These tumors account for approximately 5% of all ovarian cancers. And like desmoid tumors, this is a meaningful patient population for which there are no FDA approved therapies. In multiple myeloma, we've established clinical proof of concept for niragastat in combination settings with several BCMA directed agents and are continuing to collaborate with a number of industry and academic partners to further this strategy. We're also pursuing expansion opportunities for mirdometinib in several monotherapy and combination therapy settings.

Speaker 3

Promising data from a Phase III study of mirdometinib in pediatric low grade glioma were recently presented at the International Symposium on Pediatric Neuro Oncology Meeting, and several combination studies are currently ongoing, including a trial with MAP cures for nerafenib in advanced solid tumors with MAP kinase mutations and with BeiGene's lifurafenib in NRAS mutant solid tumors. Bimberafenib is also being explored as a monotherapy in adults with RAS mutant solid tumors, and we're looking to confirm the efficacy signal we reported last year in the dose escalation study in several ongoing dose expansion cohorts. Additionally, SW682, our oral TED inhibitor is now in the clinic. This program is designed to treat tumors with hippo pathway mutations, which can occur in up to 10% of cancers, including mesothelioma

Speaker 11

and head and

Speaker 3

neck cancer. We believe that there is a meaningful opportunity with SW682 for SpringWorks to develop a novel medicine for these cancers. Lastly, we are also advancing several internally discovered preclinical stage programs and we look forward to sharing more about these efforts as they progress. To close, we are very pleased with our accomplishments in the first half of this year and have multiple milestones and data readouts ahead that we look forward to sharing. These include long term follow-up data from our DEFI trial, which we believe will support the benefits of extended treatment durations with OXIVIO in desmoid tumors.

Speaker 3

It also includes initial data from our Phase 2 trial of niragastat in ovarian granulosa cell tumors, and we expect the full RENEW trial results to be published in a peer reviewed journal. Our focus for the remainder of the year will be to continue building on ONXIVIO's momentum in the U. S. While working to bring this medicine to patients in Europe and other geographies to advance our regulatory and commercial preparations for mirdimetinib in anticipation of serving patients with NF1Pn in the U. S.

Speaker 3

And Europe and to progress our diversified emerging portfolio in an efficient manner. We are confident that our strong foundation will support our continued success as we deliver on our mission to change the lives of people suffering from devastating diseases. As always, I would like to thank the patients and investigators who participated in our clinical trials, our patient advocacy partners and collaborators and our team of spring workers. We're now happy to take questions. Operator?

Operator

Thank you. We will now open the call for questions. Our first question will come from Anupam Rama at JPMorgan.

Speaker 8

Hey, guys. Congrats on all the progress. Just a quick one for me. What are you seeing from both new prescribers and repeat prescribers for Obsivio? And in the early innings, the wait and watch population that's coming in to seek treatment for Obsivio?

Speaker 8

Thanks so much.

Speaker 3

Thanks, Anupam. Bob, I shall let you take that.

Speaker 9

Yes. So thanks, Anupam. On the repeat versus new, obviously, we saw a good amount of new prescribers in the initial months of the launch. As you'd expect, we've started to see repos coming in at a regular pace from these initial starts, but supported by continued new patient starts from new patients being acquired in the Q2 as well. As you'd expect, the total proportion of business coming from existing patients versus new patient starts is increasing over time.

Speaker 9

And so that's a positive development. From a wait and watch perspective, we do know obviously that the proportion of the DP patient population, there is is under active surveillance at any point in time. However, we do know that over 90% of these patients received some active intervention during the course of their disease. Now, with the availability of the first and only FDA approved treatment, the opportunity ahead of us and supported by treatment guidelines which favor systemic therapy supported by the positive experiences that patients and physicians are seeing with Obsivio in the early innings of the launch, the opportunity ahead of us is to try and drive faster treatment for these patients under active surveillance. And so we do expect the proportion of active surveillance patients to be dropping over time.

Operator

Our next question comes from the line of Yaron Werber from TD Cowen.

Speaker 10

Great. Thanks for taking my question and congrats also on a nice quarter. Maybe I have a kind of a couple of interrelated questions. One, can you just give us a sense on inventories for the quarter for Exivio? And then secondly, just a follow on Anupam's question, it's one would imagine that the academic centers are probably the early adopters and they're moving kind of quickly as patients are coming in to use Obsivio.

Speaker 10

What are you seeing in the community? Maybe just give us a little bit of sense of kind of the breadth of usage? Thank you.

Speaker 3

Bhavesh, I'll turn to you again for that. Yes.

Speaker 9

Thank you. So from an inventory standpoint, the overall impact of inventory in the second quarter was minimal. The stocking levels were generally aligned with the demand that we're seeing. And similar to prior quarters, inventory was not a key driver of performance this quarter. From a community dynamic perspective, 1st of all, I'll say that from as we said in our prepared comments, there is a deeper pool of patients overall than we originally estimated.

Speaker 9

And we started to break into this aggregate pool of patients that are of greater than 7,000 actively managed patients. We are as you highlighted, we are pleased with the uptake in the centers of excellence. We saw breadth of prescribing in the early months of the launch. We saw depth of prescribing in the Q2. Specifically addressing your question around community, we've started to see good adoption in the community centers where we continue to drive breadth and but we still have an opportunity, right.

Speaker 9

With the broad pool of patients, we have an opportunity both to continue to drive breadth of prescribing, but also those who experience, who do prescribe OXIVIO to drive depth of prescribing. Now I'll highlight a couple of things in that regard. First of all, our ability to be in the right place at the right time for these community clinics has been significantly enhanced by the availability of the desmodium or specific ICD-ten codes. And so we're able to direct our sales team to be in those clinics when a patient is available for treatment. And then secondly, the educational efforts that we're driving both around the availability of Obsivio as the 1st and only treatment option for desmoid tumor patients, but also treatment guidelines which do favor systemic therapy gives us confidence in the ability for us to drive depth in that community segment as well.

Operator

Our next question comes from the line of Corinne Johnson from Goldman Sachs.

Speaker 2

Hey, good morning guys. I guess you've talked a little bit about having some more visibility on this patient population given the ICD-ten codes. And potentially as you kind of move to these blister packs in the forward. I guess as you look at that, where have you identified the pockets where you see kind of the greatest opportunity for near and intermediate term growth? And what are you doing tactically to reach that patient population and keep them on therapy?

Speaker 2

And then I did want to ask also on meridamatinib, maybe with the launch next year, where you see kind of like the low hanging fruit with respect to initial adoption? And how should we think about the trajectory of the early launch compared to what you've accomplished here with Exvio?

Speaker 3

Thanks, Karen, for the series of questions, but we're going to try and answer all of them. I think as we think of miradamatinib, we view that opportunity is actually quite meaningful. And as I think of low hanging fruit, I think we're very comfortable saying that this is an opportunity that is actually at least as large as what we see in test point tumors. And that's driven by a number of factors. 1, by the size of the patient population, right?

Speaker 3

We talked about 40,000 patients in the U. S. And a proportionate number outside the U. S. And a third of those are excuse me, a quarter of those are pediatric and 3 quarters of those are adults.

Speaker 3

So the opportunity for us to have a best in class treatment for pediatric and a first in class treatment for adults, we think gives us a meaningful target. 2nd, the physicians in this space actually themselves even in the pediatric setting believe that their patients are not being served as broadly as they could be. And so we think there's an opportunity there. And third, you can use Kosiluco revenues, which now run rate just in the pediatric setting of about $500,000,000 annually, as a proxy for the opportunity just in that one quarter of the market. PE.

Speaker 3

So we think the opportunity beyond, I think low hanging fruit implies that it's all easy, none of it is, but you've got a medicine that people are waiting for an opportunity that is sizable and data that we believe supports a potentially best in class profile for mirinib for this disease. Abhishe, I'll turn to you to talk about OXIVIO.

Speaker 9

Yes. I'd just reiterate a couple of the points. 1st and foremost, we're fortunate to have with Obsivio to be in a position of delivering a drug with transformative benefit to desmonterumor patients, right? That is the foundation for the high awareness we have, the high satisfaction that we shared in the prepared comments as well as the strong preference that we see amongst prescribers and patients supported by broad access by the payer community. So with just 7 months on the market, we become the systemic standard of care and the most prescribed systemic therapy for adult patients with desmoid tumors.

Speaker 9

And so the opportunity for us is to continue to drive depth within the centers of excellence as well as drive depth and breadth in the community segment as I mentioned before. With the large pool of 7,000 more than 7,000 actively managed patients, we do see that we have a significant opportunity ahead of us to see continued growth for Obsidian.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Peter Lawson from Barclays.

Speaker 4

Great. Thanks for taking the questions. Thanks for the update as well and congrats on the progress. First question is really just about the patient trends, how they've been looking month over month and particularly into July, and then whether you've seen any patients discontinuing? Thank you.

Speaker 3

Yes. Thanks for the question, Peter. I think we aren't seeing any exceptional discontinuations. It's what we would have expected from the DEFY study. And I think that gives us confidence going into not just the Q3, but the Q4 as well.

Speaker 3

So I think that the trend holds well. As Bhavesh highlighted, we think the patient pool is actually deeper than what we had originally estimated. And I think that portends well for the aggregate opportunity here. Fundamentally, where we are perhaps most excited is the feedback that we're getting though. We've got patients seeing deep immediate and persistent benefits to their pain that is causing them to want to get on therapy and want to stay on therapy.

Speaker 3

So we believe that the opportunity here continues to be sizable.

Operator

Thank you. One moment for our next question. Our next question comes from the line of Michael Schmidt from Guggenheim.

Speaker 11

Hey guys, good morning and congrats on a strong second quarter result. Just a follow-up on Obsivio. As we think about Q3 here coming up, some other products sometimes experience seasonality over the summer months. Is that something you may expect as well with OXIVIO? And then a follow-up on mirdometinib as we think about the launch next year.

Speaker 11

Obviously, you have already product on the market. Do you expect initial switching from Koselugo over to mirdamatinib? Or you expect a predominantly penetration into the currently untreated patients with NF1 as we think about that next year? Thanks so much.

Speaker 3

Thank you, Michael. So I'll take the Obsivio question. I'll pass to Doctor. Atrice to take the nerdivertiv question. Yes, I don't think that we're immune from seasonality.

Speaker 3

I think as we see in many other products, I would expect to see some seasonality in the summer months. But obviously, as we've said from the outset, we think the pool is deep and we think the opportunity is significant. Anthony?

Speaker 7

Yes. So with respect to our opportunity within NF1PN for miritamatinib, I'll remind you that our NDA filing is for both pediatric and adult patients, the latter of whom represent 3 quarters of the market and do not have an approved therapy. So we think there's quite a bit of white space there with respect to being able to serve patients who are previously untreated. Within the pediatric population, I think there will certainly be some patients who have been treatment experienced over the years that are seeking their next line of therapy, but our market research shows that that market is minimally penetrated as well with respect to systemic therapy. So we think there's a significant number of pediatric patients who are also looking for their 1st systemic therapy option when mirabenib becomes available.

Operator

Our last question will come from the line of Alex Stranahan from Bank of America.

Speaker 12

Hey guys, thanks for taking my question and congrats from us on the strong print. Any framing you can provide around the focus of the longer term follow-up data from DeFi later this year? And maybe one quick one if I can sneak it in. Is your expectation that you will receive accelerated review for mirvetinib? Just to clarify.

Speaker 12

Thank you.

Speaker 3

Thank you, Alec. I'll turn to Bethany for the answers.

Speaker 7

Sure. So with respect to the longer term follow-up data, Alec, I'll remind you that at the time of our primary analysis, the median time on treatment for patients that were randomized to the niragastat arm was just under 2 years and the majority of those patients were remaining on therapy. And so what we hope to be able to share an update on later this year is certainly a more mature median time on treatment figure for niryacostat treated patients as well as the trends that we see in terms of responses, how they evolve over time and the benefits that we are seeing with a longer dated data cut on the patient reported outcomes as well. So that's what we're gearing up to be able to share. And I'll remind you that in Phase 1 and Phase 2, our median time on treatment was over 4 years in each of those studies.

Speaker 7

So there's the opportunity to really present longer term data.

Operator

Thank you.

Speaker 3

With

Speaker 7

respect to any expectations on review timing and then review designations for the mirinib filing, we have not given any guidance on that topic. We'll be in a position to receive the PDUFA date from FDA about 60 days from filing. So that'll be time late in

Operator

August. Thank you. This concludes today's call. Thank you for joining. You may now disconnect.

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