Q4 2019 Earnings Call

Good day, everyone and welcome to the dice affair.

Matthew L. Sherman: As Steve mentioned, we have a robust pipeline of novel kinase switch control inhibitors, all derived from our proprietary discovery research platform and all designed to address unmet medical needs and difficult-to-treat cancer. First, I would like to review our progress with the most advanced of our investigational broad-spectrum kit in PBGFR-alpha inhibitors. We work toward the potential approval and launch for prenatal patients with advanced chest pain. Previous therapies include Aleve, Imatinib, Sunitinib, and Regorafinib. We continue to explore other possible uses of Imprimidib, including in second-line gestation.

Pharmaceuticals fourth quarter and full year 2019 financial results Conference call. Today's call is being recorded at this time I would like to turn the call over to Gen Robinson, Vice President Investor Relations Jen.

Thank you Michelle welcome and thank you for joining us today to discuss the Cypress fourth quarter and full year 2019 financial results.

John Robinson, Vice President Investor Relations at the site.

Matthew L. Sherman: Intrigue is our ongoing Phase III study of recruitment in patients with second-line GIFs compared to the current standard of care assumed. Both site activation and patient enrollment continue to go very well, and we currently have 111 sites activated in 20 countries. As we recently disclosed, due to an early trend of a higher-than-expected number of center patients, we are planning to increase the total number of patients in the study to help achieve the pre-specified number of events in a timely fashion. The anticipated increase does not change the total number of required events.

With me. This afternoon, just got the financial results and provide a general corporate update our ski Berger.

Chief Executive Officer, not Sherman, Chief Medical Officer, Dan Martin, Chief Commercial Officer, and Soccer, Kelly Chief Financial Officer.

Before we begin I would like to remind you that any statements. We make on this call but are not historical facts are forward looking statements, reflecting the current beliefs and expectations of management made pursuant to the safe Harbor provision of the private Securities Litigation Reform Act 1995.

Examples of forward looking statements made during this conference call include the status and our expected timelines for a preclinical and clinical studies review of our and he submission and potential crushed commercial launch.

Matthew L. Sherman: The Statistical Powering of the Study and our Current Guidance for Achieving Full Enrollment in the Second Half of this Year. We remain confident in the potential for recruitment to become an important and effective therapy for patients with GIST after treatment with a matinee. The data we presented last fall from the Second Line GIST cohort in our ongoing Phase I study continue to show highly encouraging clinical activity based on the median progression-free survival of 46 weeks for 10.7 months and a 19% confirmed objective response rate. As you will recall, published data from the centrally read Pivotal Study for Sunitinib show the median PFS of 24.1 weeks, or 5.6 months, and a confirmed objective response rate of 6.8%. Outside of JIST, we expect to present data from at least one of the expansion cohorts of our ongoing Phase I study of recruitment in the second half of 2017.

<unk> looking statements made on this call involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward looking statements and we cannot assure you that our expectations will be achieved.

Such risks and uncertainties [laughter] clinical trial and timing of study data the actions of regulatory agencies and those set forth in our most recent annual report on form 10-K, as well as our other S E filing Mr. No obligation to update or revise any forward looking.

Following this call replay will be available on the company's website W.W. dot decipher dot com, but that's I will now turn the call over you heard or President and Chief Executive Officer.

Thank you Jay good afternoon, everyone and thank you for joining us on this call to discuss our fourth quarter and full year 29, chief financial results as well as clinical and Businessobjects 29 chain. It was a year of exceptional execution by the junior decipher most significantly we reported positive results from Invictus our pivot.

Matthew L. Sherman: Beyond creatinib, we are focused on rapidly developing DCC3014, our highly selective inhibitor of CSF1R, for patients with tenosynovial giant pill tumor, or TGCT. TGCT is a rare disease with an estimated annual incidence of the localized form of 13,000 and approximately 1300 for the diffuse type of disease in the U.S. Annual incidence rates may not provide a full picture since these patients typically present relatively early in adulthood and can live with the disease for a long time, indicating that the prevalent population may be substantially greater, and only systemic therapy for patients is approved.

<unk> phase three study, which represented in patients with advanced chess, whose previous therapies include at least a magnet sumit that Andrew Berger wrapping up.

These results formed the basis of our first N D. A submission which was recently accepted by the FDA wouldn't priority review and decide to do for target action date of August 13. This year.

The reported another India is being reviewed under the Ft age real time oncology review or arch or pilot program.

Matthew L. Sherman: Small Molecule Inhibitor of Multiple Kinases, including CSF-1R, which was approved in August of last year and is subject to a rent program due to hepatotoxicity, which is believed to be off-target. We believe that DCC 3014 has the potential to fulfill the unmet medical need for an effective treatment with a more favorable safety profile for patients. Last year, we presented preliminary clinical proof-of-concept data on the initial three patients using type PGCT at the Connective Tissue Oncology Society Annual Meeting.

The order for program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible.

In addition, with the reporting that India. We're also participating in the FDA project work was an issue.

Under this program we have received priority review for new drug submission with health, Canada, and say marketing authorization application with the therapy therapeutic goods administration in Australia, former President and advance Jets.

We plan to submit a marketing authorization application to the European Medicines agency in the second half of this year.

Matthew L. Sherman: Encouragingly, all three patients showed preliminary anti-tumor activity as of their first scan. At their first tumor reassessment at Cycle 3, Day 1, tumor reductions from baseline were 48, 25, and 24%, respectively. At the data cutoff date of September 10, 2019, one patient had a confirmed partial response, which was ongoing for nine months. Tumor Reduction from Baseline of 84% as of Cycle 10, Day 1. Symptomatic improvement in mobility and reduced pain, as reported by the investigator, were also observed. ACC 3014 was generally well-tolerated.

We're very proud of our execution on the recruitment program in Jets, where we took the compound from I.M.D. filings you Andy a submission and just over four years. The team designed and executed a clinical and regulatory strategy that allowed us to move rapidly to multiple registration studies generating compelling datasets that have resulted in our inclusion.

In a number of regulatory designations and programs for breakthrough therapy designation to the real time Oncology review pilot program to project work and.

And we believe arching can direct these capabilities to the rest of our clinical and preclinical pipeline to continue to generate value beyond for pregnant.

Importantly, and 29 team. We also made significant progress with our two other wholly owned clinical stage product candidates DCC 30, 14, Andrew Bassinet.

Matthew L. Sherman: No grade 3 or higher treatment emergent adverse events were observed in diffuse-type TGCT patients. In 2020, we expect to select a phase 2 dose and open a phase 2 expansion portion of the study in TGCT patients. We are continuing to enroll TDCT patients in the ongoing dose escalation portion of the Phase I study and look forward to presenting additional data on these patients in the second half of this year. Next in the pipeline is rivacinib, our potent and selected type 2 inhibitor, which we're exploring in two clinical studies in combination with chemotherapy. One with paclitaxel and one with carboplatin. In January, we announced that we had selected a Phase II dose of 100 mg twice daily of rivacinib in combination with carboplatin and activated Part II in the study, where we were evaluating the combination in patients with breast cancer, ovarian cancer, and mesothelioma. Last year, we presented preliminary data from the Phase 1B2 study of the redaction and decarbonation of Paco Tax.

And we introduced our newest preclinical product candidate DCC 31 16.

We remain focused on leveraging our proprietary research platform to build a deep pipeline of novel kinase, which control inhibitors to address unmet medical needs for patients with cancer.

Building off the tremendous work done in 29 chain, we look forward to a transformational year for decipher of this year, we're well positioned for success as we seek to transition from an R&D organization to a commercial stage company based on the potential launch of our first approved products in the U.S. later this year.

We believe 2020 would be an exciting year for our pipeline with significant milestones expected across our clinical and preclinical programs as Matt will discuss in more detail. We expect to have important clinical updates with data on additional Dennis Inovio Jain cell tumor patients with DC Cdthirty 14 data updates for our.

Matthew L. Sherman: We are starting to encourage pulmonary activity with objective responses in patients who have previously received treatment with paclitaxel across multiple solid tumor types. We are actively enrolling the expansion cohort to Part 2 of the study in patients with breast, ovarian, and endometrial cancers. In the second half of this year, we expect to report data from both of the paclitaxel and carboplatinum studies. Finally, our discovery and research engine continues to generate new product candidates for novel targets. And we disclosed last year that the next target we'll be pursuing is the ultra kinase for patients with mutant RAS cancer. The Oak kinase is the initiating factor in the autophagy pathway, which is a cellular recycling pathway that's been shown to be upregulated in mutant RAF cancers and mediates resistance to inhibitors of the RAF kinase signaling pathway. DCC 3116 is our potential first-in-class bulk kinase inhibitor that we intend to develop for the treatment of mutant raft cancer, and we're working toward filing the IMD for 3116 later this year. Deciphera is a company founded on deep insight into kinase biology and focused on developing important new medicines for the treatment of cancer.

Bastion of development program, and an eye Andy filing for DCC 31, 16, a potential first in class Oak inhibitor targeting you can rest cancers.

To review these programs in greater detail I will now turn the call over to match Sherman, Our Chief Medical Officer, Matt.

Thank you Steve.

Steve mentioned, we have a robust pipeline of novel cardiac switch control inhibitors, all derived from our proprietary discovery research platform well designed to address unmet medical needs difficult to treat cancers.

First I would like to review our progress was pregnant Oh investigation broad spectrum, Kip and PV Jaafar elephant inhibitor.

As we work towards a potential approval and launch for patients with advanced just because previous therapies, including leaf and that live sunitinib and we're hoping that we continue to explore other possible uses of unpredicted, including in second line just patients.

Intrigue is our ongoing phase three study ever present in patients with second line June compared to the current standards of care Sumit.

Both site activation and patient enrollment continues to go very well.

And we currently have 111 sites activated 20 countries.

As we recently disclosed due to an early trends have a higher than expected members censored patients requiring to increase the total number of patients.

Daniel C. Martin: We have demonstrated the ability to discover and develop novel drug candidates based on our proprietary research engine centered on kinase switch control inhibition. I look forward to updating you on the progress with our clinical and preclinical programs throughout the year. I will now turn the call over to Dan Martin, our Chief Commercial Officer, and we will discuss our commercial preparations for the potential launch of Recruitment in the U.S. Thank you, Matt.

Nope achieved a pre specified number of exams and timely fashion.

The anticipated increase does not change the total number of required events. The statistical powering of the study and our current guidance with achieving full enrollment the second half this year.

We remain confident in the potential problem premiums to be covenant importing and effective therapy for patients with just after treatment with the maximum.

Daniel C. Martin: We are tremendously excited about the potential for recombinant to address critical unmet needs for patients battling, As many of you know, just as a rare cancer, with significant unmet needs for patients who develop resistance to first line of medicine. Current 2nd and 3rd line treatments confirm modest progression-free survival benefits and no overall survival benefit compared to previous treatments. Furthermore, there are no

David We presented last fall from the second line, just cohort and our ongoing phase one study.

Turning to show highly encouraging clinical activity based the median progression free survival 46 weeks, a 10.7 months, 19% confirmed objective response rate.

As you will recall published data from essentially rent pivotal study for significant shows a median PFS of 24.1 weeks or 5.6 months and the confirmed objective response rate of 6.8%.

Daniel C. Martin: Currently Approved Tribunal, the vast majority of patients who progress on third-line therapy, except for the 5-6% Blue Harbor or rare PDGFR-alpha exonating mutations. Determining the precise number of patients who receive treatment for GIST after progressing on imatinib is challenging given that existing therapies are more widely used in non-GIST indications, and GIST epidemiological studies have typically focused on patients with newly diagnosed GIST as Analyses of U.S. claims data have helped to refine our understanding of the patient journey in advanced genetics. Based on these analyses, we estimate that in the U.S., there are approximately 2,000 incident patients eligible for second-line treatment. Second, line is the patient population that we always consider as the primary long-term opportunity for repregnancies in the post-immatric setting and is the focus of the phase three intrigue study. Beyond the second line, we estimate a reduction in the incident treatment-eligible population of approximately 20 to 30 percent.

I'm sorry, just we expect to present data from at least one of the expansion cohorts of our ongoing phase one study ever printed in the second half of Twentytwenty.

Younger printing and we're focused on rapidly developing DCC 30, 40 hour highly selective inhibitor CSF one are for patients with tenants Inovio triumphal tumor for TG city.

TGC teams are rare disease.

Committed annual incident localized form of 13000.

Approximately 1300 for the diffuse type of the season than us.

Annual incidents rates may not provide a full picture since these patients typically present loans, we really in adulthood and can live with a disease for a long time, indicating that the prevalent population in substantially greater.

The only improve systemic therapy for patients UGC tea is Texas darts.

Daniel C. Martin: Subsequent line therapy due to participation in clinical trials. Treatment discontinuation or DEP. In addition to these claims analyses, we've conducted extensive market research to better understand what gist-treating physicians are looking for in a new therapy, and to what extent they think Repretinib could address those needs. Irrespective of line of therapy, GIST patients have consistently ranked overall and progression-free survival, as well as safety and tolerability, as their most valued markers of clinical benefit. And that's a review of blinded product profiles for represent... As well as other therapeutic options, only treating physicians conveyed high interest in the representative profile due to its efficacy and safety.

Small molecule inhibitor multiple cases, including some one off which was approved in August of last year and subject to a rems program due to have hepatotoxicity.

This is believed to be off target.

We believe that DCC 30, 40 has potential to fulfill the unmet medical need for an effective treatment for more favorable safety profile for patients with TGC too.

Last year, we presented preliminary clinical proof of concept data. The initial three patients with diffused type two DCT and the connective tissue oncology Society annual meeting.

Encouragingly Encouragingly all three patients showed preliminary anti tumor activity as of the first scans.

At the first tumor reassessment cycle three genuine tumor reductions from baseline were 48 25, 24% respectively.

Daniel C. Martin: as well as the fact that INVICTUS was a Phase III placebo-controlled study, which they consider to be the gold standard, further enhancing their confidence. Preparations are on track for our first commercial launch, pending FDA approval for print. Under Matt's leadership, our medical affairs team is in place, and our MSLs have been engaging in sarcoma. Scientific Exchange.

The data cutoff date of September 10, 29 team one patient have confirmed partial response, which was ongoing for nine months for tumor reduction from baseline that 84% as a single tenant did one.

Symptomatic improvements in mobility and reduced came as reported by the investigator. We're also observed.

Daniel C. Martin: On the commercial side, our marketing team launched a disease education program to raise awareness of the unmet need and gist and has defined our go-to-market strategies for launch. MRP access teams focused on ensuring optimal patient access on approval, including building out our patient assistance program. As you know, these programs are important for any oncology drug, Specialty in Oral Oncology Drugs, often as a result of the patient affordability challenges that can arise due to the Medicare Part D Benefit Design. Regarding our sales organization,

DCC 30, 14 was generally well tolerated no grade three or higher treatment emergent adverse events observed diffuse type two GCG patients.

In 2020, we expect to select the fees to dose and open the phase two expansion portion of the study introducing two patients.

We're continuing to roll TGC few patients and the ongoing dose escalation portion of the phase one study.

And look forward to presenting additional data on these patients in the second half of this year.

Daniel C. Martin: We've identified who the top creators of GIST are and where they practice. We've defined our sales force size and structure. And we've built an incredibly seasoned sales leadership team.

Next in the pipelines rebel estimate our potent selective tied to inhibitor, which were exploring and two clinical studies in combination with chemotherapy, one with Paclitaxel and one with carbo platinum.

Daniel C. Martin: And we've launched our recruiting of sales representatives. We've been extremely pleased with the deep and talented pool, which includes candidates from virtually every leading oncology company in the industry. And it's composed of individuals with extensive oral oncology knowledge.

In January we announced that we had selected the phase two dose of 100 milligrams twice daily over investment in combination with carbon platinum inactivated part two of the study.

Daniel C. Martin: We're tremendously excited about the commercial team and capabilities we're building and very grateful to have the opportunity, pending approval, to help bring Repretinib to patients. I will now turn the call over to Tucker Kelly, our Chief Financial Officer, to review the financial... Thanks, Dan. We'll be taking a minute to discuss a few highlights from our full quarter and full year 2019 financial results. We remain well-capitalized to execute on the compelling opportunity we have with the potential

Where we are evaluating combination to patient breast cancer ovarian cancer and these affiliate.

Last year, we presented preliminary data from the phase one be two study ever definitely the combination of Paclitaxel.

Sorry, encouraging preliminary activity with objective responses in patients, but didn't previously received treatment with paclitaxel across multiple solid tumor types.

We are actively enrolling the expansion cohorts part two of the study in patients with breast ovarian and endometrial cancer.

Thomas Patrick Kelly: and a strong clinical and research pipeline.

In the second half of this year, we expect to report data from both of the Paclitaxel and carbon platinum studies.

Thomas Patrick Kelly: We ended 2019 with cash, cash equivalents, and marketable securities of approximately 580 million, and in February

Finally, our discovery research engine continues to generate new product candidates for novel targets and we disclosed last year, but the next target will be pursuing the bulk of timings for patients with new breast cancers.

Thomas Patrick Kelly: We have completed a follow-on public offering that raised approximately $188 million in net proceeds. Based on our current plans, we expect that our cash will be sufficient to pay for all of our services.

Okay kinase initiating factor and the of talking to pathway.

Which is a cellular imaging recycling pathway has been shown to be up regulated new reps cancers and mediates from assistance to inhibitors the progress can be significantly.

Thomas Patrick Kelly: Operations and CapEx into the second half of 2022.

Thomas Patrick Kelly: In the fourth quarter of 2019, our total operating expenses increased to approximately $70 million from $58 million in the prior quarter. This was due to increased investments to support our potential commercial launch of a pregnancy test, as well as increased clinical development activities across the pipeline. Research and Development Expenses were approximately $47 million, and Selling, General, and Administrative Expenses were approximately $24 million for the fourth quarter of 2020.

If you see 31 16 is our potential first in class both kinds inhibitor that we intend to comes off for the treatment immune rest cancers.

And we're working towards filing 90 for 31 16 later this year.

Decipher as a company founded and deep insights into clinics biology, and focused on developing importantly, new medicines for the treatment of cancer.

We have demonstrated the ability to discover and develop novel drug candidates based on our proprietary research engine centered on Chinese which control. In addition, I look forward to updating you on the progress with our clinical and preclinical programs throughout the year.

Thomas Patrick Kelly: 2019

Steven L. Hoerter: We expect our expenses to grow over the coming quarters as we build our commercial organization and expand our clinical development. With that, I'll now turn the call back over to you. Thank you, Tucker. I'd like to take a moment to thank the entire team here at Decipher for their dedication to our mission of developing important new medicines for the treatment of cancer. We're very proud of the progress we made in 2019 and excited about the prospects for 2020 as we look to transition into a commercial stage company and aggressively develop our exciting clinical and research pathways. With that, Michelle, I'd like to open the call to questions. Operator.

Ill now turn the call both again, Martin our Chief commercial officer to discuss our commercial preparations for potential launch of recruitment in the us.

Yes.

Thank you Matt.

We are tremendously excited about the potential for recruitment to address critical unmet needs for patients battling advanced chips.

As many of you know just as a rare cancer with significant unmet need for patients who develop resistance to first line amendment.

Turning to second and third line treatments confirmed modest progression free survival benefit and no overall survival benefit compared to placebo.

Further there are no currently approved treatment options for the vast majority of patients who are progressing third line therapy.

Except for the 5% to 6% who harbor around our PDGF, our Alf X any indication.

Jessica Fye: In order to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key. Please stand by while we compile the Q&A roster. And your first question comes from the line of Jessica Fye from J.P. Morgan. Hey guys, good afternoon. Thanks for taking my question. With the enrollment completion for Intrigue expected in the back half of this year, what's your latest estimate on when we'll see top-line data for that trial?

Determining the precise number of patients who receive treatment for just after progressing on that challenge given that existing therapies are more widely used in non just indications and just epidemiological studies have typically focused on patients with newly diagnosed chest as opposed to those with metastatic disease.

And now she's of U.S. claims data has helped to refine our understanding the patient journey in advanced Jets.

Steven L. Hoerter: Hey Jess, it's Steve. Thanks for the question. So, you know, we have not changed our guidance in terms of time to completing enrollment, which is in the second half of this year. And when we reach full enrollment, our intent would then be to disclose what we anticipate as being the timing for a potential readout of the study.

Based on these analyses we estimate that any of US there are approximately 2000 incident patients eligible for second line treatment.

Second minus the patient population that we have always viewed as the primary long term opportunity for recruitment in the post mountain setting and is the focus of the phase three intrigue study.

Steven L. Hoerter: Okay, maybe I can get a second one. What will you be watching for when the Avopretinib Voyager study reads out? Is it hazard ratio, safety profile, absolute months of improvement, or better than control? What are you most focused on in that data set?

The on second line, we estimate the reduction in the incident treatment eligible population of approximately 20% to 30%.

Each subsequent line therapy due to participate batiste.

Steven L. Hoerter: Yeah, good question, Jeff. We're really interested in the totality of the data. You know, based on our understanding, the company intends to report top-line data. So depending on what level of detail they get into, we'll be interested in the total view of the data, the risk benefit profile, and the like, and how that compares with the current label for pregnant women. So we look forward to seeing the data here in the coming quarter.

We should in clinical trials.

Treatment discontinuation or death.

In addition to these claims analyses we conducted extensive market research to better understand with just treating physicians are looking for new therapy and to what extent I think were pregnant can address those needs.

Irrespective of line of therapy, just treaters have consistently ranked overall and progression free survival as well as safety and tolerability as their most valued markers of clinical benefit.

Christopher Joseph Raymond: Great, thank you. And again, if you'd like to ask a question, that's star one on your telephone keypad. And your next question comes from the line of Chris Raymond from Piper Sandler. Thanks, guys. Just a question, I guess, on intrigue.

And after reviewing blinded product profiles for pregnant.

As well as other therapeutic options just trading conditions conveyed high interest in the recruitment profile due to the efficacy and safety data from investments as well as the fact invictus for the phase three placebo controlled study, which take considered to be the gold standard further enhancing their comps to date.

Christopher Joseph Raymond: I know you had a disclosure about the censoring rate being a little higher than expected in the upsizing, and you talked about this in your prepared comments, but I guess I'm wondering if you can give a little bit more sort of color or indication of your communication cadence going forward. So, when you actually do decide to upsize it, will you announce that, and should we expect to see that perhaps on clintrials.gov? Or would you sort of announce that? You know, in some other fashion, if you will, and I'm just kind of curious as to how you intend to communicate, you know, any sort of changes that actually get enacted going forward.

Our preparations are on track for our first commercial launch pending FDA approval for printing.

Under match leadership, our medical Affairs team is in place and I am itself and engaging succumb to experts in scientific exchange on the commercial side. Our marketing team has launched a disease education program to raise awareness of young eating just and as defined or go to market strategies for launch.

Okay access teams focused on ensuring optimal patient access upon approval of accretive including building out our patient assistance program.

Steven L. Hoerter: Yeah, thanks, Chris. Thanks. Thanks for the question. I mean, first, let me just say that our confidence in the website www.clinicaltrials.gov.au

As you know these programs are important for any oncology drug.

Especially in oral oncology trials, often at the result of the patient affordability challenges that can arise due to the Medicare part D benefit design.

Steven L. Hoerter: Great, okay. And then, maybe, I'm not sure if you're going to want to answer this, but just talking about your launch preparedness, you've got a couple different scenarios I think that you'll be launching into. You know, we're pregnant into. You either have a competitor with just a label for PDGFR-alpha Exon 18 patients, or one with a broader fourth line label. I guess the question is, how different are you planning for both scenarios? I would imagine you're planning for both scenarios, but how different are those plans?

Regarding our sales organization, we've identified who the top treaters exist are and where they practice, we've defined our salesforce sized structure. We built an incredibly seasoned sales leadership team and we've launched our recruiting of sales representatives, we've been extremely pleased with the deep and talented applicant.

Pool, which includes candidates from virtually every leading oncology company in the industry and it's composed of individuals with extensive oral oncology launch experience.

Were tremendously excited about the commercial team and capabilities, we're building and very grateful to have the opportunity pending approval to help bring the patent to patients this year.

Steven L. Hoerter: Yeah, thanks, Chris. Good question. Let me make a few comments on that, and I'll flip it over to Dan to add some more.

I'll now turn the call over to cover Kelly, Our Chief Financial Officer to review the financial results.

Steven L. Hoerter: As we think about the profile of a pregnant woman, we view the data that we have to support the product, the basis for the NDA.

Thanks, Dan will be taken in it to discuss a few highlights from our fourth quarter in full year 2019 financial results.

Steven L. Hoerter: as supporting the drugs being best-in-class in this setting. It's not common that you see a drug in a pivotal study, a randomized study, generate a hazard ratio of 0.15; in our case, the hazard ratio for PFS is 0.15, or for overall survival, for that matter, of 0.36.

We remain well capitalized to execute on the compelling opportunity we have the potential launch for president and a strong clinical research pipeline.

We ended 2019 with cash cash equivalents and marketable security of approximately 580 million and in February we completed a follow on public offering that raised approximately 188 million net proceeds.

Steven L. Hoerter: So this, I think, signals a very compelling profile in terms of efficacy. And then we know from the tolerability profile of the drug that the drug is generally well-tolerated, and Invictus was very consistent with the broad phase one set of data that we've already had. So we have a high degree of confidence in terms of how physicians will view those data. Dan, I think, mentioned in his prepared remarks some of the research that he's done with physicians and potential prescribers. And Dan, maybe you want to add some additional color. Yeah. Thanks, Steve. And Chris, a good question.

Based on our current plans, we expect that our cash will be sufficient to fund operations and capex into the second half of 2022.

In the fourth quarter of 29 team. Our total operating expenses increased to approximately 70 million from 58 million in the prior quarter based on increased investments, Florida potential commercial launch of upright and as well as increased clinical development activities across the pipeline.

Research and development expenses were approximately 47 million and selling general and administrative expenses were approximately 24 million for the fourth quarter 2019.

We expect our expenses to grow over the coming quarters as we build our commercial organization and expand our clinical development activities with that I'll now turn the call back over to Steve.

Daniel C. Martin: So, you know, of course, it's great to have more options for patients, but when we think about how the treatment paradigm may shape up in the days to come, we really focus on what our market research has helped educate us about, which is what treating physicians say they're really looking for. And I mentioned it in my prepared remarks, but it has endpoints, progression-free survival, and, really unique in space, overall survival relative to placebo. As Steve mentioned, in addition, a clean safety and tolerability profile. That's what they tell us they're looking for. And, you know, when we did the research to show blinded profiles across Repregnant as well as other treatment options in the space, what came back really consistently was their interest level in a product like Repregnant.

Thank you checker I'd like to take a moment to thank the entire team here of decipher for their dedication to our mission of developing important new medicines for the treatment of cancer.

We're very proud of the progress we made in 2019 and are excited about the prospects for 2020, as we look to transition into a commercial stage company and aggressively develop our exciting clinical and research platform.

With that Michelle I'd like to open the call for questions.

Operator, we'd like to kind of orderly progression quarter in order to ask a question you want me to press Star one on your telephone to withdraw your question press the pound key.

Please standby, we compile the Q and a roster.

I'm sure first question comes from the line of Jessica Fye from JP Morgan.

Daniel C. Martin: In fact, in one study we did, about 97% of the respondents said they'd be interested in a product with Receptnant's profile for the treatment of patients with GIST. So, we really are focused on how we can best get Receptnant to patients as quickly as possible because we think it can have a real positive impact as they battle their disease. Thanks a lot.

Hey, guys. Good afternoon, Thanks for taking my question.

With the enrollment completion for intrigue expected in the back half of this year. What's your latest estimate on one we'll see topline data for that trial.

Daniel C. Martin: And again, in order to ask a question, that's star number 1. And your next question comes from the line of Eun Yang from Jeffries. Thank you. So for the second line gist, when you look at the phase one study, you were shown more than 20 weeks of PFS improvement compared to what Sunitinib has shown in phase three. But in the clinic, based on your conversation with oncologists, what would be the kind of minimum PFS benefits that they would consider clinically meaningful compared to Sunitinib?

And just to see thanks. Thanks for the question. So we have not changed our guidance in terms of time to completing enrollment which is in the second half of this year and when we reach full enrollment our intent within due to disclose what we anticipate as being the timing for a potential readout of the site.

Okay, maybe they can get a second on [laughter].

What will you be watching for when the ever put NIM. Voyager study reads out is it hazard ratio safety profile absolute month improvement over control what are you most focused on that.

Steven L. Hoerter: Yeah, thanks for the question. So you're right about second line cohort phase one, with those data showing that based on our recent update, last year was a 10.7 month progression-free survival for that cohort. Of course, that's in a non-randomized setting, but if you do the cross-study comparison that you just mentioned and look at the SUTEMP label, that does represent a really substantial difference relative to what SUTEMP offers, and I think Matt commented on this in his prepared remarks. So in the context of intrigue, we think that a clinically meaningful difference in progression-free survival would be in the range of two to three months, and we think, certainly, based on the data that we've generated in the phase one study so far, we have a lot of optimism in terms of how we view the potential for repregnant but to offer benefits to patients in that setting.

Yes. Good question, Jeff we're interested really on the totality of the data.

Based on our understanding of the company intends reporting topline data so depending on what level of detail they get into will be interested in the total view of the data the risk benefit profile and the like and how that compares with the current label for a critical.

If we look forward to seeing the data here in the coming up coming quarter.

Great. Thank you.

And again, if you'd like to ask a question that star one on your telephone keypad on your next question comes from the line of Chris Raymond from Piper Sandler.

Thanks, guys just.

I shouldn't I guess, then and intrigue and I know.

Steven L. Hoerter: Okay, thanks. And then one question on 3014-CSF1R inhibitors. So when you move into the registration trial, do you think you would need to compare it to PEX-DARC-INIB in order to recruit patients faster?

You had a disclosure about.

The censoring rate they were higher than expected in the upside you talked about this in your prepared comments, but I guess I am wondering if you can give a little bit more sort of color or indication of your communication cadence going forward. So when you actually do decide to upsize. It will you announce that.

Steven L. Hoerter: Yeah, thanks, Eun, for the question. So, it's not clear to us yet whether or not the design for that pivotal study would be. And as we said, we'll provide some additional guidance on that toward the end of this year as we provide a data update from Phase I. You know, I think there are potentially a variety of different options. One could be a placebo-controlled study, and another could be a head-to-head study. But we, of course, need to generate some more data, have some discussions with the regulators, and then determine what our pivotal study strategy is going to be. And as I said, we'll have some additional information on that, we think, at the end of this year.

And you know should we expect to see that perhaps and Clin trials dot Gov or would you sort of announce that.

In some other fashion if you will.

And yeah, I'm, just kind of curious as to how you intend to communicate any sort of changes that actually get enacted going forward.

Yes, thanks, Chris. Thanks. Thanks for the question I mean first let me just say that our confidence in a treat remains unchanged. So when we make the final decision on number of patients that we'll be adding to the study will make a disclosure on that probably both on clinical trials outcomes as well as updating our investor deck I just make sure that.

Steven L. Hoerter: Thank you. And your next question comes from the line of Michael Schmidt from Guggenheim. Hey, guys, thanks for taking my questions. I just wanted to follow up on the intrigue trial disclosure. We've gotten a fair amount of questions on sort of, you know, maybe you could help us understand again, sort of what's been driving the higher than expected rate of discontinuations and what your confidence level is that there is no imbalance in those dropouts in either of the arms.

The information is out there and then as I mentioned earlier once we reach for enrollment our guidance remains the same for the second half I will then provide at that time some additional guidance on when we expect the study to report out.

Great, Okay and then.

<unk> I'm not sure if you don't want to answer this but just to talking about your launch preparedness.

You got a couple of different scenarios I think that you'll be launching.

Michael Werner Schmidt: Yeah, thanks, Michael. Steve, thanks for the question.

Repression of into you either have a competitor with just a.

Steven L. Hoerter: You know, as I noted earlier, our confidence and intrigue are unchanged. I also noted that our guidance remains the same in terms of enrollment. And I think it's also important to remember that there's been no change in the number of events required to reach in the study or for powering the study. So this is really about enabling a readout in a timely fashion for the study. You know, in terms of censoring seen in the study, this is a phenomenon in any event-driven study in oncology where you see censoring. So it's not something that's surprising to us that we see this dynamic, and we have confidence that by adding a number of subjects to the study, we can ensure a timely readout for intrigue. So we look forward to, of course, generating the data. And as I noted, when we reach full enrollment, we'll then provide some additional disclosure on the potential time to read out for the study.

Label for PDGF, our Alpha X on 18 patients, so or one with a broader fourth line.

Label.

Yeah, I guess the question is.

How different are I would imagine you're planning for both scenarios, but how different are those plants.

Yes. Thanks for asking question really make a few comments on that but I'll flip it over to advance to add some more color.

As we think about the profile of upgrading that we view that the data that we have to support the product the basis for the FDA as supporting the drugs being best in class in this setting to treat this disease. It's not comment that you see a drug in a pivotal study a randomized study generate a hazard ratio of 0.15.

Our case hazard ratio for PFS, 1.154th overall survival for that matter point to reset. So this was I think signals a very compelling profile in terms of efficacy and then we know from the the Tolerability profile. The drug drug is generally well tolerated and Invictus was very consistent with the broad phase one set of data that we've been.

Steven L. Hoerter: Okay. Great. Thanks. And then, you know, regarding the update from the Phase I study of reprednib in other cancer types that you guided to in the second half of this year, I guess, should we think about this predominantly as an update from the systemic mastocytosis arm or potentially from some of the other cohorts that you've been enrolling? And I guess what type of data would you like to see there in order to justify potential further, you know, next steps and additional trials in any of these indications?

Oh, sorry, published so we have a high degree of confidence in terms of how physicians will view those data Dan I think mentioned in his prepared remarks, some of the research that you've done with physicians with potential prescribers and then maybe you would add some additional color, yes, I think Steve and Chris Good question.

So you know of course, it's great to have more options for patients, but when we think about how the treatment paradigm the shape up and negates the come we really focus on what our market research has.

Help to educate us about which is what I'm just treating physician say there, they're really looking for and I mentioned in my prepared remarks, but its these endpoints.

Steven L. Hoerter: Yeah, thanks, Michael. As you noted, we have a number of expansion cohorts in the ongoing phase one, including a cohort in mastocytosis. We have a cohort in kid-driven melanoma. We have a GPM cohort. So a number of cohorts where we're investigating the potential for Repregnant to play a role in other tumor types. As you noted, our guidance is to provide an update from at least one of those cohorts toward the end of the year. And our hope is that mastocytosis could be one of those cohorts and potentially a lot of further data as well. But this is, of course, dependent upon getting patients enrolled and generating data. So it's really premature for me to comment on what the bar would be in any of these cohorts to warrant further study. I think we need to see the data mature, understand the data, and, of course, present the data. And then, at that time, I think we'd be in a position to offer some additional color on where we might go from there.

Progression free survival and really unique in the space overall survival relative to placebo.

As Steve mentioned in addition, a clean safety and Tolerability profile, that's what they tell us they're looking for and.

When we did the research to show blinded profiles across our footprint and as well as other treatment options in the space. What came back really consistently was their interest level on a product like refresh and if in fact.

In one study we did about 97% of the respondents said to be interested in.

Hey, our product with Britain its profile for treatment of patients in jets. So.

We really are focused on how we can best scan for printing them to patients as quickly as possible because we think it.

And have a real positive impact as they've got all their disease.

Great. Thanks, a lot.

And again in order to ask a question that star one and your next question comes from the line of Ian Yang from Jefferies.

Steven L. Hoerter: And then you did provide some updated information on the, you know, identified or treated patient population for GIST in the US. How should we think about the potential market opportunity outside the US or maybe more specifically in Europe for GIST?

Thank you.

The line kids. So when you look at phase one study, a usually showing more than 20 weeks silver PFS improvement compared to.

Steven L. Hoerter: Good question, Michael. Let me start off with an answer to that, and then I'll ask Dan to offer some additional color if he has any. So, in this disease, we wouldn't expect that the incident population of GIST is any different in the U.S. versus any other geography around the world. So, I think the best way to generate an estimate for GIST in Europe is really to look at it on a population basis relative to the U.S. And so, based on that, we would expect to see not only a larger number of patients in Europe relative to the U.S.

So let me turn it hasn't shown on page is today.

But in in the clinic based on your conversation with them colleges.

Well, what do we the kind of minima PFS of benefits that they would you consider a clinically meaningful compared to <unk>.

Yes, they understood. Thanks for the question so you're right second line cohort phase one with those data has shown based on our recent update from the end of last year is a 10.7 month progression free survival for that cohort of course, that's on a non randomized setting, but if you do the cost set a comparison.

Daniel C. Martin: Dan, I don't know if you have any additional color you'd offer on that. Yeah, sure. Just a little bit. Thanks, Steve. When you extend the incident rate that Steve was mentioning to a European and Japanese population, whereas in the U.S., we think there are probably 4,000 to 6,000 incident patients with GIST, newly diagnosed GIST, as per the American Cancer Society, we think in Europe and Japan together, that would bring you to approximately 8,000 new patients, incident new patients in those parts of the world.

You just mentioned that look at the suits that label on that does represent a really substantial difference relative to what suits have offers and I think Matt commented on this in his prepared remarks. So in the context of of industry, because we think that a clinically meaningful difference and progression free survival within the range of two to three months on and we.

I think certainly based on the data that we generated in the phase one study. So far we have a lot of optimism in terms of how we view the potential for rhopressa to offer benefits to patients and that site.

Okay. Thanks, and then but my question on cities that are one for cancer when our Uh huh.

Daniel C. Martin: And when we think about the second through fourth line, would that be a similar, I guess, haircut, so to speak, to get to those numbers, XUS?

HM two registration trial.

Thank you the need to compare to the extent to maybe you know her to recruit patients a faster.

Daniel C. Martin: Yeah, well, we haven't done any primary research in those territories yet, Michael, to provide further guidance, but we would assume that they would be substantially similar in terms of what you'd see in the treatment paradigm as patients receive treatment with one line of therapy and then progress and need to receive treatment with the next line.

Yes. Thanks again for the question. So it's it's not clear to us yet whether what the design for that pivotal study would be it as we said we will provide some additional guidance on that towards the end of this year was we provided data update from the phase one.

Michael Werner Schmidt: Okay, great. Thanks for clarifying.

I think there are essentially a variety of different options in the one can be a a placebo controlled study I know there could be a head to head study I believe course, new generate some more data have some discussions with the regulators and then determine what our pivotal studies strategy is going to be as I said, we'll have some additional information on that we think at the end of this year.

Steven L. Hoerter: [inaudible]

Robin Karanowskis: And your next question comes from Robin Karanowskis from SunTrust. Hi guys, thanks for taking my question. Just a couple follow-ups. Maybe first on the NCCN guidelines, Ava Pritten got on there pretty quickly. [inaudible] Transcription by Transcription Outsourcing, LLC.

Steven L. Hoerter: Hey Robin, it's Steve. Thanks for the tripartite question. Let me try and knock these off one by one. So, in terms of the MCCN guidelines, you know, we think it's very encouraging that the MCCN chose to act so quickly to update the treatment guidelines in the compendium post the approval of Ava Pridnev. And I think that is reflective in part of the unmet need generally speaking in GIST and how physicians are excited about the potential for new treatment options for their patients given that unmet need. So, our expectation would be that the panel moves quickly upon a potential approval of Ava Pridnev in the U.S. to evaluate our data and then make a determination of how to reflect that in the MCCN guidelines. So I think the second part of your question was related to the expanded access program that we've launched. And maybe I can provide just a brief update on the EAP. So we're very pleased with the pace of sites opening and patients enrolling in the EAP.

Thank you.

Yes.

Thank you.

And your next question comes from the line of Michael Schmidt from Guggenheim.

Hey, guys. Thanks for taking my question I, just wanted to follow up on the intrigue trial.

Sure.

In a fair amount of questions on sort of you know maybe you could help us understand a gas of what's been driving the higher than expected rate of Discontinuations I suppose and what your confidence level is that there is now imbalance and dropouts in either of the arm.

Yes. Thanks, Michael Thanks for the question you know as I noted earlier, our confidence in intrigue is unchanged.

Right I noted in our guidance remains the same in terms of enrollment and I think it's also important to remember that there's been no change in the number of events required.

To reaching the study or the powering the study. So this is really about enabling a read out in a timely fashion for the study.

In terms of censoring CNO study. This is a phenomenon in any event driven study in oncology, where you see censoring.

Steven L. Hoerter: I can offer that we've seen patients enroll in a variety of different jurisdictions in the U.S., in Europe, and in a variety of different other geographies beyond the U.S. and Europe. And again, our view of the number of subjects that we now have on the EAP is that this is, again, just reflective of the general unmet medical need in this population. So we're pleased to see that patients have the ability to access RepredNIP in that way. So then I think the third part, Robin, had to do with intrigue, and I think your question was, why is it that, you know, we find ourselves adding subjects to the study? And I think there's kind of an easy way to think about this.

So it's not something that's surprising to us that we see this dynamic and and we have confidence that by adding a number of subjects to the study that we can ensure a finally read out a foreign trade. So we look forward to of course generating data and as I noted when we reach full enrollment will then provide some additional disclosure on the potential time to read.

For the study.

Okay, great. Thanks, and then regarding the the update from the Phase one study of free press Nab and other cancer types that you you've guided to in a second half of this year.

Yes. It should we think about this predominantly as a an update from the systemic mastocytosis arm or potentially from some of the other cars that you've been a enrolling and I guess, what what type of data, but do you like to see that in order to justify potential further.

Steven L. Hoerter: You know, generally, with event-driven studies in oncology, as I noted, you have this phenomenon where patients are censored. So when you set out and you initiate a study, you make a certain set of assumptions in terms of how many subjects might be censored, and therefore, what should your overall end be in the population? And it's probably pretty low.

Our next steps and additional trials than any in any of these indications.

Yes, Thanks, Michael So as you noted we have a number of expansion cohorts and the ongoing phase one, including a cohort and mastocytosis, we have a cohort and capture them melanoma, we have a GBM cohorts or a number of cohorts, where we're investigating the potential for recruitment to play a role and other tumor types. So.

Steven L. Hoerter: [inaudible] It's not entirely common that companies come to this realization in the course of a study's conduct early enough to be able to actually add subjects to the study. So oftentimes, what you'll see companies do is simply announce that there's a delay in the report out of the study as they're waiting for the number of events to accumulate. So, in our case, we made a certain assumption when the study, when the protocol was written. It turns out, based on some early indications from Intrigue, that that set of assumptions may have been a little bit off of the mark. And so that's the reason we've taken this approach to just ensure we get to the requisite number of events in a timely fashion.

He noted our guidance is to provide an update from at least one of those cohorts for this year and to our hope is that best buy ptosis could be one of those awards of essentially a lot of further data as well and this use of course dependent upon getting patients enrolled in generating data. So it's really premature for me to comment on what the bar.

Would be at any of these cohorts such warrant further study I think we need to see other data mature understand the data was presented data and then at that time I think we'd be in a position to offer some additional color on where we might go from there.

Reni John Benjamin: Thank you. And your next question comes from the line of Ren Benjamin from JMP Securities. Hey, good afternoon, guys. Thanks for taking the questions. Could you talk a little bit about how many patients might be added to the Intrigue study? Are we talking about a handful, tens, you know, hundreds? And maybe what is the gating factor that remains before you implement such a change? And kind of how long does it take once you've decided on the change to actually get the trial sites to implement it?

Understood and then he did provide some updated information on the you know identified are treated patient population just in the U.S.

How should we think about be the potential market opportunity Akcea, maybe more specifically in Europe for just.

That's one of these are good question, Michael Let me, let me start off of an answer for that and we lost stands offer some additional color. If he has a so in this disease. We wouldn't expect that the incident population of jus is any different in the view Ross versus any other geography around the world. So I think the best way to generate.

Steven L. Hoerter: Yeah, thanks for the question. So we haven't yet disclosed that number as we're still working through the amendment. And there is, as you probably know, a process one has to go through to get the amendment written, to get it reviewed appropriately, and get it out to sites. And so as soon as we're on the back end of that process, we'll then update our disclosures, as I mentioned earlier.

And estimate for just in Europe is really to look at an all population basis relative to the yourselves.

So based on that will be with respect to see are not only larger number of patients in Europe relative to the you asked just based on population differences, but again I don't know if you have any additional how would you offer upon that yes, sure just a little bit anything Steve So.

Steven L. Hoerter: Okay, and then one of the things that we noticed, at least with the Orbis initiative, is that, I guess, a couple of additional countries have come on board. If we read it right, Singapore and potentially Switzerland. Does the application for Retinib kind of automatically get filed in those countries, or is that something that you need to apply for?

When you extend the incident rate.

And Steve was mentioning to our European and Japanese population, whereas in units, we think Theres, probably 4000 to 6000 incident patients with just a newly diagnosed just as per the American cancer Society.

Steven L. Hoerter: Yeah, I think that's right. So FDA's intent, at least based on what we've seen, based on interviews that have been given by Dr. Pazdur, is to try to broaden this program over time. You know, I think I may have read the same article that you did about Singapore and Switzerland potentially being included in the future. But in our case, the two countries that are within scope are the ones that I mentioned. So Canada and Australia, where we've now received priority review. And so we don't have any expectation that the number of countries is going to increase from those two. So you can expect it would be just Canada and Australia that would be part of this project or this recruitment application.

We think in Europe, and Japan, together that would bring you to approximately 8000 on new patients into infinity patients in nodes parts of the world.

And then we think about second through fourth line with that'd be a similar I guess haircuts, so speak to get to those numbers ex U.S.

Yeah, well, we haven't done in the primary research in those territories, Yeah, Michael to provide further guidance that we would assume that it would be substantially similar in terms of what you'd see in the treatment paradigm as size patients.

Thank you for digital plus a lot of therapy in progress and need to receive trigger the next month.

Okay, great. Thanks for clarifying.

You got.

And your next question comes from the line of Robyn Karnauskas from Suntrust Suntrust.

Steven L. Hoerter: Got it. And then just one final question is, have you already had discussions with EMEA as you were preparing for this following the second half, or are you waiting on anything in particular before having it?

Hi, guys. Thank for taking my question I'm, just a couple of follow up.

Maybe first on the NCCN guidelines I'm interpreting that got on their pretty quickly maybe give your thought ride any hurdles you think about getting on the guidelines sit here.

Steven L. Hoerter: Yeah, we were working towards, as I noted, a filing in the second half of the year. And so we haven't talked about specific interactions with specific regulators.

Once you want to your approach and then second how is your early access program going and then third anything like you're not you're not surprised that you know these.

Steven L. Hoerter: We're just working toward getting that filing in, and that'll be the next disclosure that you see from us related to the MAA. There's a disclosure related to the filing in the second half.

That you may have to upsize trial, because I've I've.

Censoring that you're saying, but why weren't those assumptions, including original protocol with his help us understand like maybe some on differences between your original assumptions and maybe what you were saying in the blinded data. Thank you.

Steven L. Hoerter: Great, thanks for taking the question.

Yeah, Hey, Robert that Steve Thanks for the the try parts a bank question, Let me, let me try and knock these off one by one also in terms of yet in terms of the NCCN guidelines. We think it's very encouraging that the NCCN chose to act so quickly to update the treatment guidelines in the compendium post the approval.

Peter Richard Lawson: You got it. Thanks, Ryan.

Steven L. Hoerter: And your next question comes from the line of Peter Lawson from Barclays. Hey Steve, just wondering if you have a good handle on the censoring events, or is that still kind of playing out, is that still evolving?

All of it and turn them and I think that is reflective in part of the unmet need generally speaking in Jess and how how physicians are excited about the potential for new treatment options for their patients given that unmet need.

Steven L. Hoerter: Yeah, thanks. Thanks, Peter, for the question. I mean, we have a very good sense of what [inaudible] We took the decision that we did to add a number of patients to the study, but we don't have any expectation that that trend is going to change over time. You know, it's still early, so I suppose that, with the addition of more subjects, we might see fewer censoring events. Kind of difficult to predict, but it's not anything that's concerning to us. We have a pretty straightforward remedy for this, and that is to add more subjects to the study. So we're working on that.

So our expectation would be the panel.

Moves quickly upon a potential approval of accretive in the Ross to evaluate our data and then make a determination of of how to reflect that in the NCCN guidelines.

I think there's the second part of your question I think it was related to the expanded access program that we've launched and maybe I can provide just a brief update on the fee. So we're very pleased with with the pace of sites opening and patients in rolling on the EAP I can offer that we've seen patients enrolled in a very.

I'd have different things addictions, you us in Europe engine, a variety of different other geographies beyond view us in Europe and again, our our view of the number of subjects that we now have on the is that this is again just reflective of the general unmet medical need in this population.

Steven L. Hoerter: But will there be any changes to the enrollment criteria for those new subjects, or will that stay the same?

Steven L. Hoerter: No, it stays absolutely the same. Yeah, so there aren't any other changes to the study. This is just about enrolling subjects to get to the requisite number of events in a timely fashion.

Daniel C. Martin: Thank you. Just on, how should we think about pricing, discounting, any expectations around net? Just metrics we should be thinking about.

So we're pleased to see that patients have the ability to access.

Were accretive in that context.

So then I think the third part Robyn had to do with intrigue and I think your question was.

Steven L. Hoerter: Yeah, so I think I can make a couple of remarks and then I'll turn it over to you. So, as we look at the pricing landscape, of course, we're out talking to payers right now, sharing with them the product profile of a pregnant attorney and an understanding of how they view value. Certainly, we're doing the same with physicians and sharing as much of the product profile with them as possible and understanding how they view the data. And that will eventually guide a pricing decision for us. And Dan, I don't know if you want to offer any more color there.

Why is that we find ourselves adding subjects for the study I think it's Kevin easy way to think about Thats going to generally with event driven studies in oncology as I noted you you have this phenomenon where patients are sensors. So when you set out in your initiate a study so I think a certain set of assumptions in terms of how many subjects might be set.

Answered and therefore, what what's your overall NBG and the population.

And if it's probably.

Daniel C. Martin: Sure, yeah, a couple of points. Peter, thank you for the question. So, from a research perspective, we have, of course, been engaged with payers to understand their view of GIST and the unmet need, and we've been really pleased to see in the research that payers actually really do appreciate the degree of unmet need in GIST. You don't always see that in this kind of research, and so we're really encouraged to see that. They also, when responding to these blinded product profiles, really seem to have a positive view of the recombinant profile and what that can mean for patients. So we're confident that payers ultimately will appreciate the value that a recombinant can bring to their patients, and beyond that, it's a little premature for us to comment on price. As it relates, you mentioned discounts. While we wouldn't comment on gross to net per se, one thing that we have tried to emphasize is the importance of patient assistance programs, these free drug programs in the space for any oral oncology drug. These programs are important just given the patient affordability challenges that can arise as a result.

Not.

Not entirely comment that the companies come to this realization in the course of the studies conduct early enough to be able to actually as subjects in the studies oftentimes we will see companies do is simply announced the there is a delay in the report out of a study as they're waiting for the number of against you accumulate.

So in our case, we made a certain assumption.

When the study protocol was written it turns out based on some early indications from intrigue that that set of assumptions may have been a little bit off of tomorrow and so thats. The reason we've taken this approach to just ensure we get to the requisite number of events in a timely fashion.

Okay. Thank you.

I'm sorry next question comes from the line of Ren Benjamin from JMP Securities.

Hey, good afternoon, guys. Thanks for taking the questions.

Could you talk a little bit about how many patients might be added to the intrigue study, we're talking about a handful tens hundreds and maybe what is the gating factor that remains before you implement such a change and how long does it take you know once you've decided on the change to actually get the.

Daniel C. Martin: So that's something that you want to keep in mind as you're thinking about your models.

Christopher Joseph Raymond: Great. And your next question comes from the line of Christopher Moret from Nomura: Hi, good afternoon.

Trial sites to implement this.

Yes. Thanks for the question. So we havent yet disposals that number is we're still working through the amendment in there as you probably know there's a process. One has to go through to get dividend Britain ticketed reviewed appropriately going it helps us sites and so as soon as we're on the on the back into that process will then update our disclosures.

Steven L. Hoerter: Thanks for taking the questions. First, just with respect to RATNF and future, you know, potential studies, maybe elaborate on when you might start or if you're thinking of starting, you know, earlier blinded setting trials. In particular, some physician checks suggested combinations within that that might be interesting to try. I was curious if you'd initiated or when you might initiate some trials in that regard.

As I mentioned earlier.

Okay, and then one of the things that we noticed at least with the Orbis initiative is I guess a couple of additional countries have come on board. If we were at a REIT, Singapore and potentially Switzerland is it does the application with retina kind of automatically get filed to those countries or is that something that you need to apply for.

Steven L. Hoerter: Yeah, sure, Chris. Thanks for the question and Steve. So I'll take that one.

Steven L. Hoerter: So, yes, we're certainly thinking through other places in GIST, for example, where we think repregnant might be able to offer benefit to patients. And I think you named some of the possibilities. Certainly, there's a lot of investigator enthusiasm for the Invictus data and for the potential of repregnant in this disease, and that could include combinations of other therapies. Certainly, the tolerability profile for repregnant based on the Invictus data suggests that the drug might be a good combination partner. And I think there are other settings in the disease where we could generate meaningful data and potentially demonstrate that the drug can offer benefits to patients. So we intend to actively pursue those now that we have the Invictus data behind us and determine next steps in GIST specifically.

Separately.

Yes, and I think that's right. So that FDA agents has always been summit, we've seen based on interviews that have been given by Dr. Pazdur hedges and sounds I think is to try to broaden was program overtime I think I maybe read the same are the same article that you did about Singapore in Switzerland, potentially being included in future, but in our case the.

Countries that are we going so for the ones that I mentioned, so Canada, and Australia, where we've now received priority review and so we don't have any expectation that the number of countries is going to increase from those too. So you can expect it would be just Canada and Australia that would be part of this project one of us for the recruitment of application.

Got it and then just one final question is have you already had discussions with a the EMEA and as you are or preparing for this filing the second half Where's the are you waiting on anything in particular before having the discussions.

Steven L. Hoerter: When might we hear from you on the initiation of those studies, and when might they begin?

Steven L. Hoerter: So we have it. I don't have specific guidance for you, Chris, on that. It's a little early for us to talk about planning for any additional studies. So at the right time, we'll certainly provide an update on our thinking there and on potential study initiation.

Yeah, we.

We're working towards as I noted a filing in the second half of the year and so we haven't talked about specific interactions with specific regulators. We're just working toward getting a filing in and that will be the next disclosure that you see from us related to the M&A as a disclosure related to the filing in the second half.

Steven L. Hoerter: Will it be contingent upon the second line data in your mind? in terms of setting up those studies and starting them?

Steven L. Hoerter: Yeah, I think, you know, from our point of view, based on the strength of the Invictus data, it's very clear that we have a very active drug in this disease. So I wouldn't see any need for us to wait for any additional

Great. Thanks for taking the questions.

You got thanks.

And your next question comes from the line of Peter Lawson from Barclays.

Hey, Steve.

I just wonder if you do you have a good handle as we think about intrigues you have a good handle on the sensor and events or is that still kind of playing out still evolving.

Steven L. Hoerter: We have a lot of data, and I think we have the basis of information we need to consider additional clinical steps in the disease.

[laughter].

Steven L. Hoerter: Okay, and if we could just shift then to 3014 for a second, you guided us to providing some phase two dose selection updates and some data updates in the second half of the year. How many patients with data might we see there? And then secondarily, you know, it occurred to me that this trial has, it looks like, been running since 2017, early 2017. So I was just wondering what's been the delay in accruing patient data here? Correct me if I'm wrong about the study start time. But anyway, details on both of those would be great. Thank you.

Yes. Thanks, Thanks, Peter for the question I mean, it would we have a very good sense of of what we see in the early data and that was the reason that we took the decision that we did two to add a number of patients study.

But we don't have any expectation of that trend is going to change over time, though it's still early so I suppose that with the addition of more subjects that John we might see fewer censoring events and difficult to predict but it's not anything that's concerning to us.

We have a pretty straightforward remedy for for this and that is at a number of subjects to the study. So we're working on that.

Steven L. Hoerter: Yeah, thanks Chris. Thanks for the question. So you know, we recorded phase one at the triple meeting, and this was in a solid tumor patient population. So as you might recall, it wasn't until early last year that the company disclosed a move into Tennessee Mobile Giant. So the way to think about it is, you know, we made that declaration or that disclosure early last year, then started to get sites open that had these patients to enroll. And that was the result then of the data we presented on the initial three patients at the Connective Tissue Oncology Society conference in Tokyo in November, the one that Matt referenced in his prepared remarks. So that's been kind of the history of the study.

Would it be any changes to the enrollment criteria for those new subject. So that stays the same looked at.

Now I'd say is absolutely the saying, yes, so there aren't any other changes to the study. This is just about enrolling subjects to get to the requisite number of events.

In a timely fashion no other changes and study.

Thank you just on how should we think about pricing discounting any expectations around that.

Just metrics that we should be thinking about.

Yes, I can make a couple of remarks, and then I'll turn it over to Dan just to comment on so.

As we as we look at the pricing landscape of course, we're out talking to payers right now by sharing with them the product profile for pregnant turned to use an understanding of how they view value certainly work, we're doing the same with with physicians and sharing as long as product profile with them and understanding how they.

Steven L. Hoerter: So we're actively opening additional sites, frankly. We have, over time, opened additional sites, focusing on tenosynovial giant cell tumors, and actively enrolling subjects with TGCT in the Phase I trial. And while I can't provide guidance now specifically to a number of patients that we might report on in the second half, we expect to have a number of additional patients enrolled in the study from the additional sites that we've opened. So we think we'll have a good number of patients, and again, as Matt mentioned in his prepared remarks, the intent here is to get to a recommended Phase II dose, open an expansion cohort, and then, based This is, again, a disease that we know is driven by a genetic translocation that results in overproduction of the ligand for the receptors, so the biology, we think, is very straightforward. And we also know that 3014 is a highly selective inhibitor for this target. So we think it's very well suited for us to move relatively briskly with 3014 in this disease to a potential future registration.

The data and that will eventually guide of pricing decision for us and Dan I don't know if you will offer any more color there sure yeah couple of points Peter Thank you for the question.

So feeling from a research perspective, we.

Of course been engaged with payers to understand.

Their view of just and the unmet need and you've been really pleased.

You see in the research that payers actually.

Really do appreciate the degree of unmet need a ingest you don't always see that and it's kind of research and so we're really encouraged to see that.

They also when responded these blinded product profiles really seem to have a positive view of the recruitment profile and what that could mean for patients. So we're confident that.

Payers ultimately will appreciate the value that reprinted can bring.

To there to their patients and.

Beyond that its little premature for us to comment on price as it relates you mentioned discounts, while we wouldn't be.

A comment on gross to net per say one thing that we have tried to.

Steven L. Hoerter: Great. That's very helpful.

Steven L. Hoerter: And then, just to follow up on some of the clarity you provided, the data you'll share in the second half won't be sufficient in your mind to progress into physical trials just yet. So I assume, you know, you said you'd be opening the expansion cohorts. We'll need to see some data from the expansion cohorts. How much longer might we have to wait for that data? And then would that data be comparable in your mind to, you know, some data we have for Pexidartan currently to help you make your decisions? And is that the right way to think about it? Thank you.

Underscores the importance of.

Patient assistance programs these free drug programs in the space.

Any or oncology drug.

These programs are important just given the.

Patient affordability challenges the Cana rise as a result of the Medicare part D benefit design. So that's something that you want to keep in mind.

As for thinking about.

Your models.

Great. Thanks much.

And your next question comes from the line of Christopher Marai from Nomura.

Steven L. Hoerter: Yeah, thanks, Chris. You know, that's exactly the right question to ask, I think, and it's just premature for me to comment at this time on when we might be ready, because it depends on data, and as data comes in and data matures, that will then determine when we're ready to launch and do a potential pivotal study with 3014 and this disease. So it is just difficult for me to forecast that from where we are today.

Hi, good afternoon, thanks for taking the questions.

First just with respect to Radnet, then and future potential studies, maybe elaborate on when you might start or if you're thinking of starting.

Earlier fine setting trials one in particular comes from sufficient check suggested combinations with and that it might be interesting to try I was curious it or if you initiated or when you might initiate some trials.

Metric artisanal a follow up thank you.

Steven L. Hoerter: Okay, thanks. Your next question comes from the line of Andrew Berens. Hi, thanks.

Yeah sure Chris Thanks for the question Steve.

Ill take that one so yes, we're certainly thinking through other places in just for example, where we think were pregnant might be able to offer benefits to patients and I think you named some of the possibility certainly theres a lot of investigator enthusiasm for the Invictus data and for the potential for repression of in this disease.

Andrew Scott Berens: Congratulations on all the progress, guys. A couple of questions on where we might see some data presented, assuming that there are going to be any medical conferences in the near future. [inaudible] Are we still planning, you may have said this in the prepared comments, but I missed it, are we still planning to see the systemic mastocytosis data this year? And I do have

That could include combinations with other therapies certainly the tolerability profile for credit that based on the Invictus data.

Steven L. Hoerter: Yeah, sure, Andy. Thanks. Thanks for the question. It's Steve.

Suggests that the drug might be a good combination partner I think there other settings in the disease, where we could generate meaningful data potentially demonstrate that but the drug can offer benefit to patients. So we intend to actively pursue those now that we have the invictus data behind us and determine next steps and just specific.

Steven L. Hoerter: So yes, what we've said and guided to since the beginning of the year is that we would expect to have data from one or more expansion cohorts from phase one, which were pretentive, and that would include systemic mastocytosis. So our hope, of course, is that there are medical conferences during the course of the year, and that would enable us to speak with experts and, of course, get data published. So that's what our focus is.

Right.

Well when might we hear from from you on initiation of those studies and when might they initiate.

So we have it I don't have specific guidance, where you Chris on that it's a little early for us to so talk about planning for any additional studies. So at the right time, we'll certainly provide an update on our thinking there and on potential study initiation.

Steven L. Hoerter: I don't believe we've seen those data presented anywhere. So, when are we going to see the patients that have more than the normal 150 QD? And then also, how should we think about those patients, potentially, on the label?

Will it be contingent upon the second line.

Data in your mind, Turkey.

Steven L. Hoerter: Yeah, good, good question. So you're right. In the INVICTA study, patients who started off on the Recretinib arm upon central PD had the opportunity to dose escalate to 150 BID. And actually, similarly, in the Phase I study, patients in the Phase I study also had the opportunity to dose escalate to 150 BID. So that's an analysis that we intend to publish. I can't guide you specifically when we might have that analysis ready for publication, but it certainly is, we think, going to be an interesting set of data. So that's something that the team is working on in terms of the analysis and publication plan for the data. Okay.

In terms of setting up those studies and starting.

Yes, I think from our point of view based on the strength of the Invictus data, it's very clear that we have a very active drug in this disease.

So I wouldn't see any need for us to wait for any additional clinical data to accrue to guide us to to look at other strategies with recruitment than just but as you know in addition to Invictus of course, we have the 178 patient experience from the phase one so we generated a lot of data with regretted that adjusts and I think we.

I have the basis of information that we need to consider additional clinical steps in the disease.

Steven L. Hoerter: Sorry, Andrew, was there a second part to that question? Yeah, yeah, would those appear on the label, potentially?

Okay, and if we could just shift then too.

30, 14 for a second.

Steven L. Hoerter: Yeah, it's not, you know, it's, it's, it's too early for me to comment on that, of course, as you know, the FDA is going to be the decision maker in terms of what appears on the label. I think a base case assumption would be that it's going to be based on the 150 QD dose. But we'll have to see how that discussion goes and what the final label ends up looking like with potential approval.

You guided to providing some phase two dose selection.

Dates and some data updates in the second half of the year.

How many patients with the data we see there.

And then secondarily it occurred to me that this trial is it looks like in running since 2017 early 2017. So I was just wondering what's been the delay in accruing a patient data here on correct me if I'm wrong about that study start time.

Anyway, a detail some of those would be great. Thank you.

Steven L. Hoerter: Okay, thanks a lot. I appreciate it. You got it. Thanks, Andy. And your last question comes from the line of Arlinda Lee from Canaccord. Hi guys, thanks for taking my questions.

Yes, thanks, Chris So thanks for the question. So you know Weve reported for the first time the phase one at the Triple meeting and this was a solid tumor patient population.

So as you might recall it wasn't until I think early last year that the company disclosed a move into tenets of you'll jives cell tumor.

Steven L. Hoerter: Um, I just wanted to clarify on the phase one update for Reprechnib. Is that data only going to be for non-GIST indications, or might we see any additional GIST data there? And is that data going to be sufficient to decide on a go-to-expansion phase?

So the way to think about it as we made that that declaration of that disclosure and early last year. Then started to get sites open that had these patients to enroll and that was the result, then was the data we presented in the initial three patients at the connective tissue Oncology Society conference in Tokyo in November the one that Matt referenced.

Steven L. Hoerter: Yeah, hi Dara, Linda, it's Steve. Thanks for the question. So that's right. So what we have is that we have some data from at least one of the expansion cohorts during the course of this year in the second half of the year. And so it's too early for me to comment on where those data may or may not lead us. So we have to look at the totality of the data. And once those data are mature, and we present them in the second half, I think at that time, at the time of data presentation, we'd be in a position to then provide some additional color on where we might go with that.

In his prepared remarks, so that's been kind of the history on the study. So we are actively.

Opening additional sites frankly, we have overtime open additional sites focusing on tenants Inovio jives cell tumor actively enrolling subjects with TGC tea in the phase one and while I can't provide guidance now specifically to a number of patients that we might report on in the second half we expect to have a number.

We have additional patients enrolled on the study from the additional sites that we've opened so we think we'll have a good number of patients and again as Matt mentioned in his prepared remarks, we intend areas to get to a recommended phase two dose opened an expansion cohort and then based on the data make a determination about next clinical steps.

Steven L. Hoerter: And then, similarly, for the scope of the CSF1R inhibitor data at year-end, is that going to be sufficient to help decide on an expansion? Thanks.

Steven L. Hoerter: Yeah, so I, you know, Chris, I think, had the same question a couple of minutes ago. And, you know, it will depend on the totality of the data that we have. So I think, you know, it's premature for us to comment on what we might have at year end and whether that's going to be sufficient for us to decide on the next clinical step. But I can just offer that Matt and his team, broadly, whether it be the clinical operations team or the development team, are very actively working on the balance of our clinical stage portfolio, getting the requisite sites open, getting patients put on study. So we're looking forward to having additional data, more mature data, in the second half that we can share with investors and with the clinical community to guide the next potential steps with each of these programs.

For the drug in TGC. This is again a disease that we know is driven by a genetic translocation that results in over production of the ligand for the receptor. So the biology, we think is very straightforward and we also know that 30 14 is a highly selective inhibitor for this target. So we think it's very well suited for us to move right.

Relatively briskly of with 30 14 in this disease, so potential future registration.

Great. That's helpful and then I guess just.

To follow up on some of the clarity you provided the data you'll share in second half will be sufficient to near mines to progress into pivotal trials. Just just yet so I assume you said youll be opening expansion cohorts will need to see some data from the expansion cohorts how much longer might we have to wait for that data.

Steven L. Hoerter: Thanks. Thank you. And this does conclude the Q&A portion. I would now like to hand the call back over to Steve Hoerter.

And then with that data be comparable in your mind to Nielsen data, we have protected Darden currently to help you make your decision is and is that the right when it got it. Thank you.

Steven L. Hoerter: Yeah, thanks everyone for joining us on the call this afternoon. Thanks for your continued interest and support of us here at Decipher. I wish you all a wonderful evening. Thank you very much.

Yes. Thanks, Thanks, Kristina that's exactly the right question to ask I think it has just premature for me to comment.

At this time on when we might be ready because it depends on data and as data comes in and data matures and that will then determine when we're ready to to launch into a potential pivotal study with 30 14 in this disease. So just difficult for me to forecast, that's where we are today.

Okay. Thank you.

Right.

Your next question comes from the line up Andrew Baron.

Hi, Thanks, Congrats on all the Kroger occurs.

And ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect. [inaudible] BF-WATCH TV 2021

A couple questions on where we might see some data presented.

You mean that there are going to be any medical conferences in the near future.

Are we still planning you may have said this in the prepared comments, but I missed that who still planning to see the systemic mastocytosis data.

This year and I'd be able to follow up on Invictus too.

Yes already thanks. Thanks for the question, it's Steve So yes, what we what we said in guidance you since the beginning of the years that we would expect to have data from one or more expansion cohorts from the phase one which were accretive and that would include specific mastocytosis. So our hope of courses that are higher medical conferences.

During the course of the year.

And that would enable us to speak with experts and of course cut data published so thats, our that's our focus.

Okay. Thanks, and then in regards Invictus My recollection, there was an extension phase where patients that progress I believe on either on we're able to our remaining Kurt to enter the extension phase I to double the does that were prevnar adult, but we've seen the data presented anywhere so.

Well when are we going to see the patients that had more than the.

Good morning, Onefifty QB and that also how should we think about those patients potentially on the label.

Yes. Good good question, so you're right in the Invictus study patients who started off on their accretive arm upon central PD had the opportunity to dose escalate to 150 VIP.

And then actually similarly in the in the Phase one study patients in the phase. One also had the opportunity to dose escalate to 150, VIP. So thats an analysis that that we intend on publishing I cant guided specifically when we might have that analysis ready for publication, but it certainly is we think going again.

Interesting set of data so that's something with the team is working on in terms of analysis and publication plan for those data.

Okay second sorry, I knew that are stuck in parts of my question.

Yes, just label I think with yet yeah, I would those appear in the label potentially.

Yeah, it's not it's too early for me to comment on that of course, you as you know the FDA is going to be the decision maker in terms of what appears in the label I think at the base case assumption would be that it's going to be based on the 152 dose, but we'll have to see how now that discussion goes and what the final label.

Ends up looking like with the potential approval.

Okay. Thanks, a lot appreciate it.

You bet. Thanks any.

I'm sorry last question comes from the line of Arlinda Lee from Canaccord.

Hi, Thanks for taking my question.

I just wanted to clarify on the.

Phase one update for their pets now.

Is that data.

I'm only going to be for non just indications or might we see any additional just data there and is that data going to be sufficient to decide on it.

Go to expansion phase.

Yes, hi, dialing that Steve Thanks for the question. So that's right. So what we've guided to is if we did have some data from at least one of the expansion cohorts. During the course of this year in the second half of the year and so it's too early for me to comment on where those data may or may not lead us. So we have to look.

At the totality of the data.

And once those data are mature and we present them in the second half and I think at that time at the time of data presentation, we'd be in a in a position to then provide some additional color on on where we might go with that.

And then similarly for the double the CSF one our into their data at year end is that going to be sufficient to decide on.

Help decide on that.

An expansion thanks.

Yes so.

Chris I think had the same question a couple of minutes ago and it will depend on the totality of the data that we have so I think you know it's premature for us to comment on what we might have at year end and whether thats going to be sufficient for us to decide on next clinical staff I can just offer that Matt and his team broadly.

Whether it be the clinical operations team or the development team very actively working on the balance of our clinical stage portfolio getting the requisite sites open by getting patients but on study. So we're looking forward to having additional data more mature data in the second half that we can share.

With investors and with the clinical community to guide next potential SAP split that each of these programs.

Thank you.

And this does conclude the Kunaev portion I would now like to hand, the call back over to Steve harder.

Yes, thanks, everyone for joining us on the call. This afternoon and thanks for your continued interest in support of US here Decipher I wish you all are wonderful evening. Thank you very much.

And ladies and gentlemen. This concludes today's conference call. Thank you for participating you may now disconnect.

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