Q4 2019 Earnings Call
Good morning, and welcome to the Corpus Pharmaceuticals fourth quarter and yearend December 31st 2019 earnings Conference call.
Operator: Good morning, and welcome to the Corbus Pharmaceuticals fourth quarter and year-end December 31st, 2019 earnings conference call. At this time, all participants are in a listen-only mode. The question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Ted Jenkins, Senior Director of Investor Relations and Corporate Communications. Please go ahead, sir.
So I'm all participants are in listen only mode. A question answer session will follow the formal presentation.
If anyone should the car operator systems during the conference. Please press Star zero on your telephone keypad as a reminder, this conference is being recorded.
It's now my pleasure to introduce your host Jenkins Senior Director Investor Relations Corporate Communications. Please go ahead Sir.
Ted Jenkins: Thank you. Good morning, everyone. At this time, I'd like to remind our listeners that remarks made during this call may state management's intentions, hopes, beliefs, expectations, or projections of the future. These are forward-looking statements that involve risks and uncertainty. Forelook and statements on this call are made pursuant to the Safe Harbor provisions of the Federal Securities Law. These forward-looking statements are based on Corbus's current expectations, and actual results could differ materially. As a result, you should not place undue reliance on any forward-looking statements. Some of the factors that could cause actual results to differ materially from those contemplated by such forward-looking statements are discussed in the periodic reports Corbus files with the Securities and Exchange Commission.
Thank you good morning, everyone.
At this time I'd like to remind listeners that remarks made during this call next day management's intentions hopes beliefs expectations projections in the future. These forward looking statements involve risks and uncertainties.
Statements on this call are made pursuant to the safe Harbor provision with federal Securities laws.
These forward looking statements are based on corpus its current expectations and actual results could differ materially.
As a result, you should not place undue reliance on any forward looking statements.
Factors that could cause actual results could differ materially from those contemplated by such forward looking statements are discussed in the current reports corpus files with the Securities Exchange Commission.
Ted Jenkins: The documents are available in the Investors section of the company's website and on the Securities and Exchange Commission's website. We encourage you to review these documents carefully. Joining me on the call today are Dr. Yuval Cohen, our Chief Executive Officer; Dr. Barbara White, our Chief Medical Officer and Head of Research; Sean Moran, our Chief Financial Officer; and Craig Millian, our Chief Commercial Officer. With that, it is my pleasure to turn the call over to you all. Thank you, Ted.
Talking to sort of album Investor section of the company's website and on the Securities and exchange Commission's website.
We encourage you to review these documents carefully.
Joining me on the call today are Dr., you've all Cohen, our Chief Executive Officer, Dr. Barber White, our Chief Medical Officer, and head of research, Sean ran our Chief Financial Officer, and Craig Williams, Our Chief commercial officer with that is my pleasure to turn call over do you bought.
Thank you Ted good morning, everyone and thank you for joining us on this call. This morning.
Dr. Yuval Cohen: Good morning, everyone, and thank you for joining us on this call this morning. Overall, 2019 was marked by multiple clinical and corporate achievements and important progress towards realizing our vision to become the leader in drug development targeting the endocannabinoid system. We presented new data, including oral presentations, at the American College of Rheumatology 2019 Annual Meeting, where we introduced the two-year Phase II open-label extension data that showed continued favorable safety and durable outcomes in our studies of linabicin for systemic sclerosis and for Dermatomyositis. In addition, we completed enrollment in both Resolve-1, our Phase III systemic sclerosis study, and in our Phase IIb cyst I'd like to remind you all of our vision as a company.
Overall 2019 was marked by multiple clinical in corporate achievements and important progress towards realizing our vision to become the leader in drug development targeting Endocannabinoid system.
We presented new data, including all presentations at the American College of Rheumatology, 2019th annual meeting, where we introduced the true your face to open label extension data that showed continued favorable safety and durable outcomes.
Our study Avlon Addison for systemic sclerosis.
And our study for dramatic <unk> sites.
In addition, we completed enrollment in both resolved one are phased squeezed systemic sclerosis study.
And in our phase to be cystic fibrosis study.
Position us positioning us well for topline data this coming summer.
I'd like to remind you all of our vision as a company.
Dr. Yuval Cohen: We believe that targeting the body's endocannabinoid system, also known as the ECS, holds the potential to provide new therapies to treat inflammatory, fibrotic, and metabolic diseases. We are focused on developing potential novel compounds that modulate this powerful biological... We have deep expertise in medicinal chemistry, endocannabinoid system biology, regulatory and patent strategy, as well as a proven track record of executing on our clinical development This past year, we've also been focusing on laying the important groundwork for having the commercial expertise necessary to execute a successful product launch. As we look ahead to this year, we are on track for Linnabis and top-line data from our Phase 3 study in systemic sclerosis this summer, to be followed by our Phase 2B study in cystic fibrosis. We also expect top-line data from our 100-patient Phase II study of lanavisin in systemic lupus erythematosus in the latter part of this year.
We believe the targeting the bodies endocrinologists system also known as the easiest holds the potential to provide new therapies to treat inflammatory.
Right and metabolic diseases.
We are focused on developing potential novel medicines that Mark you make this powerful biological system.
We have deep expertise in the digital chemistry.
Endocannabinoid system biology.
Richard regulatory and patent strategy as well as a proven track record of executing our clinical development plan.
This past year, we've also been focusing on baby important groundwork for having the commercial expertise necessary to execute a successful product launch.
As we look ahead to this year, we're on track for the now the some topline data from our phase three studies systemic sclerosis. This summer to be followed by our phase Twob study in cystic fibrosis.
We also expect topline data from our 100 patient phase two study I'm in Addison if systemic lupus the rest mattel's written that's houses in the latter part of this year.
Dr. Yuval Cohen: That study is funded and run by the National Institutes of Health. Finally, we expect to launch our Phase 1 study of CRB4001 this year and look forward to the key safety data the study will generate. Before Dr. White provides comments on our clinical programs, I do want to say a few words on the coronavirus, or COVID-19, situation and how we're dealing with this rapidly evolving situation. At present, we are not experiencing significant impact or delays from the coronavirus on our business or operations. Like many of our peers, we have put in place a robust risk mitigation plan to ensure the safety of our workforce and to deal with possible effects on our clinical trials. Supply Chain and Research Studies We are monitoring the situation carefully and are following guidance from local and federal health authorities. With that, I'd like to turn the call over to our Chief Medical Officer and Head of Research, Dr. Barbara White, to provide you with a quick update on our clinical and research programs.
That's study is funded and run by the National Institutes of health.
Finally, we expect to launch course phase one study CRB 4001, this year and look forward to to be she safety data the study will generate.
Before Dr. White provides content on our clinical programs I do want to say a few words on the corona virus or cold feet 19 situation and how we're dealing with this rapidly evolving situation.
At present, we are not experiencing significant impact or delays from the corona virus on our business or operations.
Like many of our peers, we have put in place a robust risk mitigation plan to ensure the safety you ever workforce.
And to deal with possible effects to our clinical trials.
Supply chain and research studies.
We are monitoring the situation carefree and our following guidance from local and federal health authorities.
With that I'd like to turn the call over to our Chief Medical Officer, and head of research Dr. Barbara <unk> to provide you with a quick update on our clinical and research program.
Dr. Barbara White: Thank you, Yuval. Lonavisin is an oral small molecule CB2 agonist that has been shown to reduce inflammation and fibrosis in a variety of preclinical and human models. Promising safety, efficacy, and biomarker data have been demonstrated in our initial Phase II studies in systemic sclerosis, cystic fibrosis, and dermatomyositis patients. At the American College of Rheumatology (ACR) annual meeting in November 2019, we presented Phase II OLE data that showed chronic dosing with linabasam continued to have an acceptable safety and efficacy profile after 25 months of The median ACR, Combined Response Index in Diffuse Cutaneous Systemic Sclerosis, or ACR CRISP score, remained at or above 0.95 out of a maximum score of 1. However, improvement from baseline in the Modified Rodent Skin score reached about minus 9 points at the time the data were presented.
Thank you you fall.
Well now this is an oral small molecule cbtwo agonist that has been shown to reduce inflammation and fibrosis in a variety of preclinical and human model.
Promising safety efficacy and biomarker data happened its drugs have been demonstrated in our initial phase two studies, it's just that make sclerosis, cystic fibrosis under matter mastitis patient.
At the American College of Rheumatology or HCR annual meeting in November.
What do you 19.
We presented data to a well lead data that showed chronic dosing with low Nablus. Some continued to have an acceptable safety and efficacy profile after 25 months of treatment.
The median HCR combined response index and diffused Catania systemic sclerosis, or you see our credit score remained at or above <unk> 0.95 out of a maximum score one.
Improvement from baseline in the modified broadening skin score reached about minus nine point at the time to data were presented.
Our 12 month global Phase three resolved one study of metabolism for the treatment of systemic sclerosis is fully enrolled with 365 <unk> go.
Dr. Barbara White: Our 12-month Global Phase III Resolve I Study of Lenavisin for the Treatment of Systemic Sclerosis is fully enrolled with 365 subjects dosed. Top-line data are on schedule and expected this summer. You will be pleased to learn that 98% of the eligible subjects who completed the double-blind randomized placebo control part of the Resolve-1 study to date have enrolled in the Phase 3 Open Label Extension. We remain optimistic that the upcoming Phase III top-line data will show favorable safety and positive treatment benefits for labicum in this rare, life-threatening disease. This optimism is based on consistency between the biological activities of lanabicin and the underlying disease mechanisms in systemic sclerosis, as well as encouraging Phase 2 safety, efficacy, and biomarker data. The similarities in baseline disease characteristics between Phase 2 and Phase 3 subjects and use of the same primary CN point, the ACR CRISP score, in the Phase II and III studies.
Topline data are on schedule and expected this summer.
You will be pleased to learn that 98% or be eligible subjects, who completed a double blind randomized placebo controlled part of the resolved one study to date have enrolled in the phase three open label extension.
We remain optimistic that the upcoming phase three topline data will show favorable safety and positive treatment benefits for the lab system in this rare life threatening disease.
This optimism is based on consistency.
Tween, the biologic activity, it's a one abbott's them and the underlying disease mechanisms in systemic sclerosis.
Our encouraging phase two safety efficacy and biomarker data.
Similarities in baseline disease characteristics between phase two in phase three subjects.
And use the same quite.
The endpoint DHR, Chris Corr, the phase two and three study.
If the resolved one efficacy data are positive and the safety profile remains acceptable we plan to hold discussions with regulatory authorities in U.S.
Dr. Barbara White: If the Resolve-1 efficacy data are positive and the safety profile remains acceptable, we plan to hold discussions with regulatory authorities in the U.S., Europe, and Asia about filing for marketing authorization. Our second global phase three study is the determined study of lenabicin for the treatment of dermatomyositis. We are very pleased with the rate of enrollment, expecting to be fully enrolled later this year with study completion in 2021.
Europe, and Asia about filing for marketing authorization.
Our second global Phase three study is to determine study of Lin Abbott some for treatment of dramatic my side.
We're very pleased.
With the rate of enrollment expecting to be fully enroll later this year, we study completion and 2021.
Dr. Barbara White: The open-label extension of this study is already active. We are optimistic that the Determine Phase III study will show positive efficacy in support of safety data for similar reasons as systemic sclerosis. Data in an oral presentation at the ACR Annual Meeting in November 2019 highlighted continued acceptable safety of linalisin in subjects with dermatomyositis who received chronic dosing through 23 months. Continued improvement was seen in active skin disease in this open-label extension, with a mean improvement from baseline of minus 20.9 points in the cutaneous dermatomyositis activity and severity index. CDASI activity score 23 months. 18 of the 20 subjects remained enrolled in the Phase 2 OLE study at that time.
The open label extension of this study is already active.
We are optimistic that to determine phase three study will show positive efficacy in support of safety data for similar reasons a systemic sclerosis.
Data in an oral presentation at Bcr annual meeting in November 2019 highlighted continued acceptable safety analysis in subjects with dramatic outsiders.
Organic dosing through 23 month.
Continued improvements was seen in active skin disease. In this open label extension with the mean improvement from baseline of minus 20.9 points into cutaneous dramatic myositis activity in severity.
Index.
Or see Dasti activity score 23 months.
18 of the 20 subjects remained enrolled in the face too early study at that time.
Dr. Barbara White: Turning to Cystic Fibrosis, our 28-week Phase IIb study of linabasam in 426 CF patients at high risk for recurrent pulmonary exacerbations, an acute event of increased lung inflammation with clinical manifestations of worsening respiratory signs and symptoms, often including a significant worsening in lung function, completed enrollment last November. We believe lenabasim represents a potential new anti-inflammatory option for reducing rates of pulmonary exacerbations in people with cystic fibrosis, without regard to CFTR mutation for current background therapy. Top-line data from our CF study are expected following the ResolveOne study. Our second asset, CRB4001, is a CB1 inverse agonist designed to have minimal access to the brain in order to avoid psychiatric effects, similar to Ramanujan. CRB 4001 has demonstrated potent, potentially beneficial effects on glucose tolerance, insulin sensitivity, lipid metabolism, body fat, and hepatic fat, in Animal Models of Disease with robust literature supporting these metabolic effects.
Turning to cystic fibrosis, or 28 week phase to be study of analysis in 426, CF patients at high risk for recurrent pulmonary exacerbation complete enrollment last November.
Pulmonary exacerbations in CF, our acute events have increased long inflammation with clinical manifestations of worsening respiratory signs and symptoms often including a significant worsening in lung function.
We believe will now this represents a potential new anti inflammatory option for reducing rates of pulmonary exacerbations in people cystic fibrosis.
Without regard to see STR mutation or current background therapy.
Topline data from our CF study are expected following the resolved one study.
Our second aspect CRB 4001 is the CB one inverse agonist designed to have minimal access to the brain in order to avoid psychiatric effect.
And with remarkable.
CRB 4001 has demonstrated potent.
Essentially beneficial effects on glucose tolerance insulin sensitivity lifted metabolism body fat, that's a pad exam.
In animal models of disease with robust literature supporting these metabolic effects.
Dr. Barbara White: We have identified additional potential beneficial effects on inflammation and in fibrosis assays. Dr. Tan and colleagues reported last month that CRB 4001 blocked liver fibrosis. We are considering CRB4001 as a potential treatment for NASH with fibrosis, with potential to be used in other diseases such as diabetic nephropathy. We plan to start our CRB4001 Phase 1 study in Q3, to evaluate the safety, tolerability, and pharmacokinetics of CRB4001. We plan that the Phase I testing will also include a PET scan study to test whether therapeutic exposures to CRB4001 will lead to significant binding of CRB4001 to CB1 in the brain. Lastly, our research team anticipates selection of our next candidate compound this year, representing the output of our growing research team of medicinal chemists, DMPK specialists, toxicologists, modelers, and biologists. And now, I'll turn the call back to you all.
We have identified additional potential benefit beneficial effect on inflammation and in fibrosis assay.
Dr. Chan and colleagues reported last month that CRB 4001 block.
Liver fibrosis.
We are considering CRB 4001, it's a potential treatment for Nash with fibrosis.
With potential will be used in other diseases, such as diabetic nephropathy.
We plan to start a CRB 4001 phase one study in Q3.
To evaluate the safety Tolerability and pharmacokinetics of CRB 4001.
We plan that the phase one testing will also include the pet Dan study to test what a therapeutic exposure to CRB 4001 will lead to significant binding.
CRB 4001 to see the one in the brain.
Lastly, our research team anticipate selection of our next candidate compound this year.
Representing the output of our growing research team medicinal chemistry, the M.T.K. specialists toxicologist Motherson biology.
Now I'll turn the call back to useful. Thank you Barbara in 2019, we began to prepare for the potential approval of the Madison and subsequently its commercial launch.
Dr. Yuval Cohen: Thank you, Barbara. In 2019, we began to prepare for the potential approval of Linatisim and, subsequently, its commercial launch. We are making very good progress with our initiatives, including the initiation of our disease educational campaign for systemic sclerosis last week. I would now like to turn the call over to our Chief Commercial Officer, Craig Millian, who will provide you an update on our commercial activities.
We are making very good progress with our initiatives, including the initiation of our disease educational campaign in systemic sclerosis last week.
Now I'd like to turn the call over to our Chief Commercial Officer, Craig million Who'll provide you an upbeat on our commercial activities. Thank evolve and good morning, everyone on our last call I highlighted that at this point if we launch planning we're focused on three critical elements to ensure success first building our commercial leadership.
Craig Millian: Thank you, Yuval, and good morning, everyone. On our last call, I highlighted that at this point in prelaunch planning, we're focused on three critical elements to ensure success. First, building our commercial leadership team and capabilities. Second, establishing a strong foundation of deep market insight. And third, communicating a compelling narrative that provides an appropriate scientific context ahead of a potential regulatory approval. Starting with building out our team.
Stephen capabilities second establishing a strong foundation of deep market insights.
And third communicating a compelling narrative that provides an appropriate scientific context ahead of a potential regulatory approval.
Starting with building out our team we have established a talented group of capable leaders to drive to successful launch including leads for marketing market access Medical affairs commercial analytics and operations supply chain public relations and patient advocacy.
Craig Millian: We have established a talented group of capable leaders to drive a successful launch, including leaders from marketing, market access, medical affairs, commercial analytics and operations, supply chain, public relations, and patient advocacy. We continue to develop a strong foundation of market insights in both systemic sclerosis and cystic fibrosis, and have conducted market research with patients, physicians, and payers in both the U.S. and Europe. Many of these insights have been incorporated into our most recent investor deck, available on the Corbus website.
We continue to develop a strong foundation of market insights in both systemic sclerosis end cystic fibrosis and have conducted market research with patients physicians and payers in both the U.S. and in Europe.
Many of these insights have been incorporated into our most recent investor deck available on the Corpus Web site.
Craig Millian: Now I'd like to take a moment to highlight some of our key learnings specific to systemic sclerosis. The greatest unmet need is in patients with early or active diffuse systemic sclerosis. These patients, once diagnosed, often have considerable challenges managing their disease, and the impact of scleroderma on their lives is profound. From a go-to-market perspective, there are roughly 50 scleroderma standards of excellence, and we estimate that fewer than 2,000 rheumatologists at these centers and also in the community treat the majority of scleroderma patients. Upon FDA approval, we believe we can efficiently reach potential prescribers with a small customer-facing team augmented by targeted multi-channel outreach. Rheumatologists who manage scleroderma patients do the best they can with available treatments, but they have no single approved treatment to address the totality of the disease.
Now I'd like to take a moment to highlight some of our key learnings specific to systemic sclerosis.
The greatest unmet need is in patients with early or active use systemic sclerosis.
These patients once diagnosed often have considerable challenges managing their disease and the impact of scleroderma on their lives is profound.
From a go to market perspective, there are roughly 50 scleroderma centers of excellence.
We estimate that you would have in 2000 Rheumatologists at these centers and also in the community treat the majority of scleroderma patients.
Upon FDA approval, we believe we can efficiently reach potential prescribers with a small customer facing team augmented by targeted multi channel outreach.
Rheumatologist to manage scleroderma patients do the best they can't would available treatment, but they have no single approved treatment to address the totality of disease.
Craig Millian: Currently used therapies address symptoms or specific organ complications but not the underlying disease progression and generally come with the added burden of immunosuppression. In our market research, we've also tested a blinded target product profile from Linabasim based on our Phase 2 data, as well as our Phase 3 study design. This profile was met with positive interest by patients, rheumatologists, and payers. Most recently, we completed market access research with payers in both the U.S. and Europe. Payers appreciate that systemic sclerosis is a rare disease with substantial unmet need. Recognizing the limitations of current treatments, payers are highly receptive to the need for and potential value of new treatment options. After presenting a potential product profile for Linabasim, we explored how payers might approach access and reimbursement decisions. Based on disease burden and lack of approved treatments, payers acknowledged the overall potential value of lenabasum as being consistent with treatments for other serious rare diseases.
Currently used therapy, the dress symptoms or specific organ complications, but not the underlying disease progression and generally comment the added burden of immunosuppression.
In our market research. We've also tested a blinded target product profile from Atlanta habits, and based on our phase two data as well as our phase three study design.
This profile was met with positive interest by patients Rheumatologist and payers.
Most recently, we completed market access research with payers in both the U.S. and in Europe.
Payers appreciate that systemic sclerosis is a rare disease with substantial unmet need.
Recognizing the limitations of current treatments payers are highly receptive to the need for and potential value of new treatment options.
After presenting a potential product profile, so what happens.
We explored how payers might approach access and reimbursement decisions.
Based on disease burden and lack of approved treatments payers acknowledge the overall potential valuable Annapolis as being consistent with treatments for other series rare diseases.
Craig Millian: Of course, more work will be done post-data to establish our value platform before finalizing pricing strategies. Finally, before turning the call back to you, Val, I'd like to update you on our recently-launched systemic sclerosis disease education campaign that targets rheumatology. This campaign is based on our market insights, conversations with KOL advisors, and, of course, the scientific literature. It calls for healthcare professionals to evaluate the totality of systemic sclerosis. The insight behind the campaign is that systemic sclerosis is a complex, devastating disease driven by both inflammation and fibrosis. However, current approaches using immunosuppressive or antifibrotic agents address only symptoms or specific organ complications.
Of course more work will be done post data to establish our value platform before finalizing pricing strategy.
Finally, before turning the call back to evolve I'd like to update you on our recently launched systemic sclerosis disease education campaign that targets Rheumatologists.
This campaign is based on our market insights conversations with cable advisors and of course, the scientific literature.
The campaign called for health care professionals to evaluate the totality of systemic sclerosis.
The insight behind the campaign is that systemic sclerosis is a complex devastating disease, driven by both inflammation and fibrosis.
However, current approaches using immunosuppressive or anti fibrotic agents address only symptoms or specific organ complications.
Craig Millian: This campaign also emphasizes the total burden of systemic sclerosis on the patient, including increased mortality risk and disability. A central feature of the campaign is a website that unravels the complexity of systemic sclerosis and also considers the potential of targeting novel mechanisms, including the CB2 receptor. This website was launched last week, and I encourage you to visit totalssc.com, T-O-T-A-L-S-S-C.com, to learn more. In the coming weeks, we will launch the full campaign, including paid search and ads on social media and relevant medical websites, all aimed at reaching the rheumatology audience. In addition, the campaign will be highlighted at upcoming medical conferences. This is our initial step. And throughout 2020, we plan to increase our investment to add content and expand reach. In summary, we are focused on ensuring strong execution around prelaunch fundamentals. We look forward to providing future updates as we advance our commercial capabilities and progress toward the loan. Let me turn the call back to you, Val, for the financial discussion.
This campaign also emphasize it the total burden of systemic sclerosis on the patient.
Looting increased mortality risk and disability.
Essential feature of the campaign is a website that unravels the complexity of systemic sclerosis, and also considers the potential of targeting novel mechanisms, including the Cbtwo receptor.
This website was launched last week and I encourage you to visit total FC Dotcom T O T. L. S. S C dotcom to learn more.
In the coming weeks, we will launch the full campaign, including paid search and adds on social media and relevant medical web sites, all aimed at reaching the rheumatology audience.
In addition, the campaign will be highlighted at upcoming medical Congresses.
This is our initial steps throughout 2020, we plan to increase our investment to add content and expand reach.
In summary, we are focused on ensuring strong execution around pre launch fundamentals.
We look forward to providing future updates as we advance our commercial capabilities and progress toward launch let me turn the call back to you ball for the financial discussion. Thank you Craig I'd like to provide a brief update on our financial position.
Dr. Yuval Cohen: Thank you, Craig. I'd like to provide a brief update on our financial position. Corbus has strengthened its balance sheet. Last month, we raised $46 million in gross proceeds from a public offering, bringing the total capital raised over the past 12 months to $86 million. We expect a cash on hand of $31 million as of December 31, 2019, together with the $43 million in net proceeds from the public offering we just did and the remaining $7.5 million in milestone payments from the Cystic Fibrosis Foundation Award, to fund operations into the fourth quarter of 2020. In closing, I want to highlight again that our vision of focusing on novel compounds that target the endocannabinoid system has the potential to yield transformative medicines This year, with our key multiple data readouts, will be our most important year to date. We look forward to what it brings. With that, I'd like to thank you all for your time and attention and turn it over to the operator for any questions from our listeners today.
I just have strengthened its balance sheet.
In February last month, we raised $46 million in gross proceeds from the public offering bringing the total capital raised over the past 12 months to $86 million.
We expect the cash on hand up $31 million as of December 30, Onest 2019, together with the 43 million in net proceeds from the public offering we just did and the remaining seven and a half million dollars in milestone payments from the cystic Fibrosis Foundation Award to fund operations into the fourth quarter of 2020.
In closing I want to highlight again that our vision of focusing on novel compounds that target. The endocannabinoid system has the potential to yield transformative medicines that could improve the treatment of inflammatory and fiber optic diseases.
This year with our key multiple data readout will be our most important year to date.
We look forward to what it brings.
With that I'd like to thank you for your time and attention and turn it over to the operator for any questions from our listeners today.
Thank you.
Operator: Thank you. If you'd like to ask a question, please press star 1 on your telephone keypad, and a confirmation tone will indicate your line is in the question queue. You may press star 2 if you would like to remove your question from the queue. For participants that are using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.
At this time will now be conducting a question answer session.
If she wants to ask a question. Please press star one from your telephone keypad and the confirmation Tama indicate your line is the question Q.
Let me first start to feel like to move your question from the Q.
I've listened studies next week or equipment, it may be necessary to pick up your handset before pressing the star keys.
Operator: One moment, please, while we poll for questions. Thank you. Our first question is from the line of Brian Abrams with RBC Capital Markets. Hi there.
One moment, please while we pull for questions.
Thank you. Our first question is from the line of Brian Abrams with RBC capital markets, especially with your question.
Brian Abrams: Thanks so much for taking my questions. I guess first off, on the Phase III systemic sclerosis trial, how are you guys feeling about the overall conduct as we get closer to the potential readout? It sounds like a lot of patients are rolling into the open label extension. What's the retention like there? Is it as high as in Phase II, and what are your thoughts on the key CRIS subcomponents that might also be important from both a clinical and regulatory perspective? And then I had a follow-up.
Hi, there. Thanks, so much for taking my questions.
I guess first off on the phase three a systemic sclerosis trial.
How are you guys feeling about the overall conduct as we get closer to the potential.
Readout it sounds like a lot of patients are rolling into the open label extension, what's the retention looked like there as it is hires in the phase two and what's your sense as to the key Chris sub components that might also be important from both the clinical and regulatory perspective, then I had a follow up.
Dr. Barbara White: Thanks, Brian. I'll try and remember all of those.
Thanks, Brian I'll try and remember all of those first how's the trial going great.
Dr. Barbara White: First, how's the trial going? Great. We're near the end. Most of the subjects are reaching, you know, at month 11 out of 12, two-thirds of the subjects are out of this study now, so we're really closing in on the end of this study. So far, we've been pleased with the conduct, and we've been especially pleased with the subject. And as part of that, I think you can see that if we have, you know, 98, 99% of the eligible people rolling over into the open label, we could not be more delighted. In terms of retention in the Open Label, I think we've got over 200 in it now, and we've had, I think, maybe 1% drop out. And some of them have been in for a year or so. So again, the retention rate to date is really quite good, and the enrollment rate is great. The retention rate is great
More near the end I'm most of the subjects are reaching you know this month 11, now 12 that two thirds of the subjects about our out of this study now so we're really closing in on the finish up. This study so far we've been pleased with the conduct and we've been especially.
Please with the enthusiasm Oh the patients.
And the subjects and as part of that I think you can see that if we have you know 90, 899% of the eligible people rolling over into the open label, we couldn't be more delighted.
In terms of retention in the open label I think we've.
You know, we've got over 200, and it now and we've had I think maybe 1% dropped out and some of them opinion from a year or so so again the retention rate to date is really quite good. The enrollment rates is quite retention rate is great and this is important because it's going to give us additional.
Dr. Barbara White: And this is important because it's going to give us additional safety data. It's important to provide long-term safety data. To date, it's been quite acceptable in other OLEs, and we're going to have even more patients in this one. And we think that will help support the...
Safety did it's important to provide long term safety data to date, it's been quite acceptable in the other allele and we're going to have even more patients and this one we think that will help support the.
Dr. Barbara White: The study approvability based on a single trial to have that much additional information. Not only on safety but also on outcomes, we think that regulators across the globe will look at all of the components of the ACR CRIS. The ACR CRIS gives us a tool to address the totality of the disease. Just what Craig pointed out was so important.
The study Approvability based on a single trial to help that much additional information <unk> safety, but also on outcomes.
We think that the regulator so across the globe, we'll look at the components of the a CR, Chris Yes, Youre, Chris gives us a tool to address the totality of the disease, just what Craig pointed out what's so important.
It is a measure of patients overall, better or not and it is a regulatory outcome in terms of what's important to the patients. We think each of the individual components on we think the regulators will be.
Dr. Barbara White: It is a measure of whether the patient is overall better or not, and it is a regulatory outcome. In terms of what's important to patients, we think each of the individual components is, and we think the regulators will be interested in all of them. The improvement in MRSS or skin thickening, change in lung function, change in patient global assessment, change in patient function, as well as what the physician thinks. Do they think the patient has improved, or not? So we expect them to pay attention to all of them, and we will certainly do a variety of analysis to support that.
Interested in all of them the improvement in Emerus Astro skin thickening change in lung function change and patient global change in patient function as well as what the position. Thanks do they take the patients improved or not so we expect them to pay attention to all and we'll certainly do a variety of analysis to support.
Thanks, So much Barbara and then maybe a question for Rep for Craig you you had talked about some of the interesting findings from the initial market research that you've done in systemic sclerosis and.
Craig Millian: Thanks so much, Barbara. And then, maybe, a question for Craig.
Craig Millian: You talked about some of the interesting findings from the initial market research that you've done in systemic sclerosis. And I guess, obviously, before the data and the label, it's premature to talk about pricing specifically. But you did mention that some of the payers in your access work were expressing receptivity to the potential value of linabasum and viewing it as consistent with certain other treatments for rare diseases. I was wondering if you could be maybe a little bit more specific and help us understand sort of the types of treatments that payers are viewing the value as being consistent with, just to provide some framework for how they're thinking about value.
I guess I, obviously, it's been.
Before the data and the label, it's premature to talk about pricing, specifically, but you did mention that some of the payers.
And your access work.
Expressing receptivity for the potential value.
Nab assuming.
And doing it is consistent with certain other treatments for rare diseases. I was wondering if you're just maybe a little bit more specific can help us understand sort of that the types of treatments that payers are viewing the value as being consistent with just to provide some framework for how they're thinking about value and ill jump back in queue. Thanks.
Craig Millian: And I'll jump back into Hugh. Thanks.
Yeah.
Craig Millian: Yeah, um, so... So thanks for the question, Brian.
So.
So thanks for the question Brian.
Craig Millian: We are obviously very much engaged with the pricing strategy, and it is early. So I appreciate that I'm not going to get into specific numbers. But I'd be happy to share some general thoughts in terms of the research we've conducted. First, we're focusing on optimizing our value proposition in systemic sclerosis. And the emphasis is on patients with diffuse disease, which, as you're aware, is the most serious form of scleroderma. And as well as the population in which our drug has been studied. So the prevalence of diffuse disease is about nine diffuse systemic sclerosis patients per 100,000 in the U.S., and that patient population clearly falls within the rare disease category and, obviously, not the ultra-rare but kind of this new concept of the medium rare disease category, which is a term that I've heard used.
We are obviously very much engaged with the pricing strategy and it isn't it is early so.
Reshape that you know I'm not going to get into specific numbers, but I'd be happy to share. Some general thoughts in terms of the research conducted.
Our first we're focusing on optimizing our value proposition in systemic sclerosis, and the emphasis is on patients with diffuse disease, which as you're aware as the most serious form of scleroderma as well as the population which are drugs been study.
So the prevalence of of diffuse disease is about nine diffuse systemic sclerosis patients per 100000 in the U.S. and that patient population clearly falls within the.
The rare disease category, and obviously not the ultra rare, but I kind of this new concept of in the medium rare disease category.
And then this is a term that that I've heard used.
Craig Millian: We, our market research was conducted independently by Clearview Consulting, and the payers agreed that systemic sclerosis is a serious rare disease with limited treatment options. They're aware that there are no specific uh... treatments indicated for systemic sclerosis overall, and you know, I'll leave you with this thought. Based on their reaction to the blinded Lenabisin product profile, payers suggested that there would be, uh... that there would be quite a bit of flexibility when considering levels of access. And again, I'd say their thought is it would be consistent with treatment analogs from other serious diseases with prevalence rates similar to SFC. For example, diffuse is 9 per 100,000; overall, SFC is about 30 per 100,000.
We are market research was conducted independently by Clearview consulting and the payers agreed that systemic sclerosis is a serious rare disease with limited treatment options. They are aware that there were no specific.
Treatments indicated for systemic sclerosis, overall, and I'll leave it with pissed off based on the reaction to the blinded Lin absent product profile pay your suggested that there would be that there would be quite a bit of flexibility when considering levels of access throughout abroad pricing range and again I'd say there.
Our thought as it would be consistent with treatment and lots from other serious diseases with prevalence rates similar to SFC and again diffuse has nine per 100000 overall SSC is about 30 30 500000. So you at this point and of course as you're aware, it's not gotten dry in terms of access levels, so depending on price.
Craig Millian: So at this point, and, of course, as you're aware, it's not gotten dry in terms of access levels. So depending on the pricing strategy, different levels of access and reimbursement are achieved. So a lot of that depends on the goals we set. But certainly, we plan to leverage these insights from payer research. We're just beginning our work in terms of health economic modeling. As you mentioned, until we have our data and a final label, we really can't determine our final value proposition. But we'll certainly combine those insights with our health economic modeling, and further down the road, we'll determine an optimal pricing strategy.
I think strategy.
Different levels of access and reimbursement or achieves a lot of that depends on the goals, we set but certainly we plan to leverage these insights and to pay a rate research. We're just beginning our work in terms of health economic modeling as you mentioned until we have our data in a final label, we really can't determine our final value.
Proposition, but but we'll certainly combine those insights with our health economic modeling and further down the road will.
A little determinant optimal pricing value strategy.
Great. Thank you so much.
Craig Millian: Great, thank you so much. The next question is coming from the line of Maury Raycroft with Jefferies. Hi, good morning everyone, and thanks for taking my questions. To start, in systemic sclerosis or any of your other late-stage trials, are you taking regular blood samples from patients and assessing those for inflammatory or possibly fibrosis biomarkers in the blood on a blinded basis? And can you comment on what you're seeing qualitatively?
Your next question is coming from the line of their Maury Raycroft with Jefferies. Please proceed with your question.
Hi, good morning, everyone and thanks for taking my questions to start for systemic sclerosis or any of your other late stage trials are you taking regular blood samples for patients in assessing those four laboratory or possibly fibrosis biomarkers in the blood on a blinded basis and it can you comment.
And on what you're seeing qualitatively what was it.
So personal yet we are taking.
Maury Raycroft: So, first of all, we are taking samples, but we're actually taking them from involved tissue. So we think that that's probably a little more relevant.
Samples, but we're actually taking them from involved tissue. So we think that that's probably a little more relevant we found in the phase. Two study was quite informative that in fact, many of the biomarkers are not ever elevated because our patients run back on the Mr. presses in all about 80% of them are so at least some background immunosuppressive him that group of subjects.
Dr. Barbara White: We found in the Phase II study, it was quite informative, that in fact, many of the biomarkers are not elevated because our patients are on background immunosuppressors. You know, about 80% of them are on at least some background immunosuppressors. And in that group of subjects, it's less common to find these markers elevated in the blood.
It's let's call them to find these markers elevated in the blood. That's why we're looking at all tissue. So we will look at histology and gene expression in the skin of patients with systemic sclerosis enter Madam myositis and we are certainly looking at upsells and more because of inflammation and.
Dr. Barbara White: That's why we're looking at involved tissue. So we will look at histology and gene expression in the skin of patients with systemic sclerosis and dermatomyositis. And we are certainly looking at cells and markers of inflammation in the sputum of patients with CF. As you may recall, Mark, we certainly saw very encouraging data in the Phase II study in these same samples with a reduction of inflammation and fibrosis in the scleroderma skin, a reduction of cells and inflammatory mediators in the DM skin, and a reduction in cells and inflammatory mediators in the CF sput
Sputum of patients with CF is you may recall more we certainly saw very encouraging data in the phase two study and the same samples with reduction of inflammation fibrosis and this clarity on the skin reduction of cells inflammatory mediators and the d. and skin and a reduction in July and inflammatory mediators Mcf skill.
Got it and are you taking those on a serial.
Dr. Barbara White: Got it. And are you taking those on a serial basis over time? I guess, are there different time points where you're getting the samples?
Basis over time, I guess are there different time points that you're getting this here.
Dr. Barbara White: You know, Mark, because they're biopsies, we're doing them at the baseline and at 12 months. We're not doing them more often than that, except in the DM study, I think some patients may volunteer for three. But mostly it's just the beginning and end of the study. The CFs are easier to come by, so they're being done a little more frequently.
Mark because they're biopsies, we're doing them, but they find them at 12 months, we're not doing them more often are not except the dam study I think some patients may volunteer for three but mostly it's just getting into the study to see if you are easier to combine their being down a little more frequently.
Got it Okay, and then for fours years or a one just clarifying what.
Dr. Barbara White: And then for 4001, just clarifying, will you include some obese individuals in your initial FAD, MAD, safety, and PK study? and uh...
Well you include some obese individuals in your initial I say D Mad safety and PK study.
And.
Okay.
Dr. Barbara White: Go ahead, sorry.
Go ahead sorry.
Dr. Barbara White: And then, will you just do one PET scan or multiple PET scans on those individuals? And have you established a threshold for how much receptor occupancy would be acceptable at different time points to move to the next step to test a drug in a larger study?
And then what are you just do one pet scan or multiple pet scans.
On those individuals and have you establish a threshold for how much receptor occupancy would be acceptable at different price points.
To move to the next steps to test driving larger study.
Dr. Barbara White: Sure, great questions. First, a question about testing safety, tolerability, and pharmacokinetics in obese individuals. That is part of the Phase I study. There's a potential that those parameters might be different than they would be in a normal or overweight individual. So we will test that because if we're targeting NASH, we're going to be dealing with a lot of obese people. So that's a straightforward early safety
Great questions first question about testing safety and Tolerability pharmacokinetics and obese individuals that is part of the phase one study.
As a potential that those parameters might be different than they would be an abnormal or overweight individual. So we will tell us because if we're targeting Nash we're gonna be dealing with a lot of obese people. So that's a straightforward early safety.
Dr. Barbara White: And PK, understanding that we need. We will move from there. After we've got those studies done, we will move into doing the PET scan at the NIH. We've been working quite closely with Dr. George Kunos and his colleagues there. The way this study is done is the patients will get multiple doses of 4001 first. They'll be loaded with it, and these will be obese individuals. And then they will be injected with a specific CB1 ligand that is for PET scanning and will light up their brain at baseline before they receive anything. We know that to see how much of the ligand binds in their brain. They'll get the drug, and then they'll have another study, and we'll see if there's any displacement of the binding of the ligand.
And PK understanding that we need.
We will move from their after we've got those studies done we will move into doing the pet scan at the NIH, we've been working quite closely with Dr. George pools and his colleagues. They are the way. This study is done is the patients will get some multiple doses of 4001 first they'll be loaded with it and these will be obese individuals.
And then we will and they will be injected with a specific CB one like Dan.
That does for pet scanning then we'll light up the brain. The first have a study.
At baseline before they receive anything you know that.
To see how much of the like an bonds in the brain they'll get the drug and then we'll have another study and we'll see if theres any displacement of the binding on blogs.
Dr. Barbara White: So the results will be compared in a cohort that gets placebo for seven days and a cohort that gets 4001 for seven days. And we will look for whether or not 4001 displaces ligand binding better than placebo. The data that are available in the literature suggest that 4001 is a better treatment for depression than placebo for depression.
Results will be compared on a cohort that gets placebo for seven days and a cohort that guess 4001 for seven days and we will look for whether or not all 4001 displaces lie Dan.
Finding better than placebo more than placebo.
The data that are available in the literature suggests that.
We are less likely to see the CNS adverse events, if they levels of displacement that would be specific binding are probably in that 10% or less range, which could be some variability around that.
Dr. Barbara White: We're less likely to see CNS adverse events because if the levels of displacement, that would be specific binding, are probably in the 10% or less range. Of course, there'll be some variability around that. Certainly, we would be less comfortable if we saw specific binding in the 30%, 40% range. That would certainly be room for caution. Somewhere in between, I think, there would be. We would need to interpret and really think about that. A little bit, we would move forward, I think, and a lot, no, and in between, we'll think about it.
Certainly we would be less comfortable if we saw specific binding and a gain of 30 40 range from 40% range that wouldn't that would certainly be room for caution somewhere in between I think would be.
We would need to interpret and really think about but.
A little bit we wouldn't move forward I think and a lot snowing in between we'll think about <unk>.
Dr. Barbara White: Got it. Very good. And will you announce when you start dosing individuals in that study?
Got it very good and where you announce when you start dosing.
Individuals in that study.
Dr. Barbara White: Sure, we will announce it. And as I said, we're on target, and we expect it to start on Tuesday. So, so far, so good. Okay.
Sure we want we will announce in and as I said, we're on target we expected to start and she is so so so far so good.
Got it okay. Thank you very much.
Leland Gershaw: Okay, thank you very much. Our next question is from the line of Leland Gershaw with Oppenheimer. Good morning, everyone. I have a two-part question, which is, it relates to the variety of mechanisms and drugs that are used as anti-inflammatories, anti-fibrotics, versus Linabizim, and I'm wondering, Barbara, if you could comment on which of those other therapeutics that are often used for the kinds of conditions that you are looking at could either work very well with Linabizim or potentially interfere with the dysfunction And then I have a second part. Thanks.
Our next question is from the line of Leland Gershell with Oppenheimer. Please proceed with your question.
Hi, good morning, everyone I'm, having a two part question.
Question, which is as it relates to the variety of mechanisms and drugs that are used take anti inflammatory. It's I brought its versus when they have this in them and wondering Barbara to comment on.
Which of those other therapeutics that are often used for the kinds of conditions that youre looking at.
You know could either worked pretty well that didn't tiering with with habits anymore.
Essentially this year with it its function given that some of these agents Tim can actually go against resolution and then at the second part thanks.
Okay.
So I think if I'm, if I may repaid repeat that question. It is.
Dr. Barbara White: So I think, if I may repeat that question, it is, what is the potential to use lenavisin in combination with other therapies? What might be synergistic efficacy or antagonistic effects? And might there be drug-drug interactions that would be a safe signal? So, as you know, we are studying patients on their background treatment, whatever it is. That's always been the approach, and we felt comfortable doing that because the expression of CB2 really just on activated immune cells goes away when they're not.
What's the potential to use when that have some in combination with other therapies what might be.
Synergistic efficacy or antagonistic effects and might there be drug drug interaction.
That work against a signal so.
As you know we are sitting patients on their background treatment or whatever do yes, it's always been the approach and we felt comfortable doing that because oh expression of C. B to really just on activated immune cells goes away when they're not that increases the safety profile and the fact to date, we've not seen significant well.
Dr. Barbara White: That increases the safety profile, and the fact that to date we've not seen significant clinical evidence of drug-drug interactions or abnormalities in lab tests, et cetera, mechanistically, I think it's possible that we could see even better efficacy in some of the patients who might be on background immunosuppressants such as MMS. We'll know obviously when the trial is over and the subset analyses are done. But, for example, the approach that we have of activating the resolution of information is absolutely novel. Nobody else is doing that. This is brand new.
Well evidence of drug drug interactions or abnormalities in the lab tests et cetera.
Mechanistically I think it's possible that we could see even better efficacy in some of the patients who might be on background immunosuppressive such as MMS well know obviously when the truck when that when the when the trial is over in the subset analyses are done but for example.
The approach that we have of activate resolution of inflammation is absolutely novel Nobody else is doing that this is brand new this provides a whole new approach to treating these chronic inflammatory antibiotic diseases.
Dr. Barbara White: This provides a whole new approach to treating these chronic inflammatory and fibrotic diseases. So, if you view the disease mechanisms sort of as a mountain, you have a climb up the hill and a climb down the hill. Lenatacin activates the going down the hill, the turning of things off, although there certainly are some effects on the activation phases, but it's potential if you pair things, you might even see better efficacy. However, we don't know that. We'll find out, and I have no belief that it needs to be that way.
So if you do you the disease mechanisms sort of is the mountain you open up a.
Climb up the hill in the climb down the Hill Lenovo some activates the going down the health of turning of things off.
Although there certainly are some effect on the activation stays as soon as potential with you've hair thing you might even see better efficacy. However.
We don't know that we'll find out and I have I have no belief that it needs to be that way by itself.
Dr. Barbara White: By itself, and we've tested lenabazine by itself, and we're testing it in the current studies without concomitant suppressives, we have every expectation that we will see really substantial clinical benefits. So I think that the way that we think it will be used will be either as monotherapy in early patients who have early active disease to bring it under early control. Craig can talk about that more, as well as in combination therapy depending upon certain organ involvements and preferences of the patients and the physicians. Drug-drug interaction studies, we've done some significant modeling of that, consistent with what the FDA likes to see. And while there are some potential drug-drug interactions with some types of compounds, they're not the compounds that patients usually use, so we're not expecting significant issues there. Craig, I didn't know if you wanted to comment about your views of commercially the use of the owner in combination.
We tested well now doesn't buy a cellphone testing it in the current studies without some comedy aims to process. We have every expectation that we will see I'm really substantial clinical benefit. So so I think that's the way that we think it will be used will be either as monotherapy in early patients early active disease.
Bringing under only control I can talk about that more as well as in combination therapy, depending upon no certain organ involvement some preferences with the patients and physicians.
The drug drug interaction studies, we've done some significant modeling Oh, that's consistent with what the FDA likes to see and while there are some potential drug drug interactions with some types of compound so not the compounds the patient usually use so we're not expecting significant issues there.
Craig I didn't know if you wanted to comment about your views of commercially if you sell onerous in combination.
Craig Millian: And obviously, as Barbara said, it will come down to the data. Certainly, I think there's... a significant opportunity for both. Certainly, most patients are on some sort of background immunosuppression, and certainly
Yeah, and obviously as Barbara stadiums, good will come down to the data.
Certainly I think there's.
Significant opportunity for both certainly most patients are on some sort of background immunosuppression and certainly.
Craig Millian: with the opportunity to add lanavisim without adding to the overall, based on the safety and tolerability profile that we expect that we've seen to date, not adding a significant treatment burden while adding significant incremental efficacy will be very compelling. Certainly, there are also patients who experience problematic side effects on immunosuppressants. They and their healthcare provider may want to try to titrate down to some degree the amount of immunosuppression, and obviously, the opportunity there even for patients further along in their disease and have more stable disease, there's certainly going to be a nice opportunity for Linneapolitan there as well. And certainly the de novo patient who's first diagnosed; we would love to be top of mind in terms of the first drug that they consider for a newly diagnosed patient as well.
Adding.
The opportunity to add lit Apis and without adding to the overall based on this safety and Tolerability profile. We expect that we've seen to date, you know not adding significant treatment burden, while adding significant incremental efficacy will be very.
Very compelling.
Certainly there are also patients who experience.
Problematic side effects on Immunosuppressants, they are paying their healthcare provider may want to try to tight treat down to some degree the amount of immunosuppression and obviously the opportunity there even for patients further along in their disease and have more stable disease is certainly though to be.
Andy.
Filling out some there as well and certainly the de Novo occasion.
Whose first diagnose.
We would love to be.
Top of mind in terms of the first drug that they consider but for a newly diagnosed patients as well and certainly as we think about how we construct our go to market.
Craig Millian: And certainly, as we think about how we construct our go-to-market strategy and the insights that these patients are being diagnosed and treated often by the community rheumatologist, and when you think about the profile of Linneapolitan being oral, again, safety and tolerability to date look quite reasonable, and obviously, the efficacy we would consider to be very attractive potentially as a first-line agent. So we think it really covers multiple bases.
Strategy and the insights that.
These patients are being diagnosed and treated often by the community Rheumatologist and when you think about the profile of one outstanding oral.
Again safety Tolerability to date looking quite reasonable and obviously the efficacy is going to we would consider to be very attractive potentially.
As a first line agency. So yeah, we think it really covers multiple multiple basis.
Thanks, that's very helpful, but just a quick follow up.
Leland Gershaw: Thanks. That's very helpful. But just a quick follow-up. As we look toward the Resolve 1 readout in cispinospirosis, can you give indication at this point if we're going to see in the top line breakdown of the CRIS subcomponents, the secondary endpoints? Will it be limited more to just truly the top line, or is it... to where they are going to look at that indication, printing the data? Thanks.
We look toward the result, one.
Readout insist desperate just can you give indication at this point, we're gonna see in the topline.
Break down of the critical components. The secondary endpoints you won't be limited more too just to the topline or or is it.
It's too early I mean looking at the integration between the good thing.
Definitely you'll see the primary we'll see the Chris and I think it would be reasonable to.
Dr. Barbara White: Definitely, you'll see the primary. You will see the CRIS. And I think it would be reasonable to expect most, if not all, of the secondaries, and that's the components of the CRIS. You'll... Because they each individually address how an Addison could provide benefit to the totality of the illness that we're treating. So, I would... At this point, I can't promise, but I think it would be our intent to show you what the overall score and the components look like, and that covers the secondaries.
Expect.
[music].
Most if not all of the secondary is the components of the Chris fuel.
Within each individually.
A dress.
Well in Addison could provide benefit to the totality of the illness that we're treating so I would at this point I can't promise that I think it would be our intent.
To show you what the overall score that component supply can that covers the secondary.
Lisa Vaco: Great. Thanks very much for your time. Our next question is from the line of Lisa Vaco with J&P Securities. Please proceed with your question. Hi John. This is on for Lisa. Thanks for taking the questions. I guess, following up on a previous question, in Resolve 1, when you're looking at these biopsies and biomarkers, can you discuss how important it is to see correlation between the biomarker changes and what you're seeing clinically in patients?
Great. Thanks, very much Susan.
Our next question is from the line of recent Baker with JMP Securities. Please proceed with your question.
Hi, John on for Lisa Thanks for taking my questions.
I guess following up on a previous question and resolved one when you're looking at.
Biopsies and Biomarkers can you discuss.
The importance of seeing correlation between the biomarker changes in what you're seeing clinically in patients.
You know I think there will be.
Dr. Barbara White: You know, I think there will be, certainly, I would expect it to correlate. But I'm not sure I would expect to see a fabulously strong correlation, because that's the difference between, you know, skin and clinical outcomes, and as we've had many discussions, there is, there's variability in clinical outcomes, and so forth and so on. But I would certainly expect to see some correlation; that's why we do it, so that we can support that the efficacy outcomes we have are very sensible. Can you look at the impact of this drug on the underlying disease pathogenesis?
I would expect it to correlate I'm not sure I would expect to see a fabulously strong correlation because that's the difference between you know scanning clinical outcomes and as we've had many discussions there are.
There's variability in the clinical outcomes and so forth insulin, but I would certainly expect to see some correlation that's why we do it.
We can support that the efficacy outcomes. We have are very sensible when you looked at look at Impax up this drug on the underlying disease pathogens.
Thanks, I guess, just one more for Craig you discuss targeting about 2000 rheumatologists.
John: Thanks. And I guess just one more for Craig.
Craig Millian: You discussed targeting about 2,000 rheumatologists. Can you discuss how you're tiering those docs and then kind of your thoughts on the size of your sales force? Thanks.
Discuss how you're hearing those docs and then kind of your thoughts on the size of your sales force. Thanks.
Sure.
Craig Millian: Sure. Yeah, so just to take a step back, at launch our goal is going to be clearly to build awareness and understanding with both the academic and community-based rheumatologists who are treating scleroderma patients. And we talked about the product profile, we think lending itself well for both those who treat at the centers of excellence as well as those community rheumatologists who often are diagnosing and getting patients. So, you know, let me caveat this by saying we haven't done a formal sales force sizing exercise yet, but, you know, we would envision a national footprint, ensuring coverage first and foremost of the roughly 50 scleroderma centers of excellence, and we would envision that being duly covered by a commercial customer-facing team as well as a medical, a field medical, We would also want to have the capacity to call on those community-based rheumatologists who are treating a sizable number of scleroderma patients in their practice.
Yeah. So so just to take a step back I mean that launch our goal is gonna be clearly to build awareness and understanding with both the academic and community based rheumatologists.
We're treating scleroderma patients and we talked about the of the product profile, we think you'll lending itself well for both.
Those who treat at the centers of excellence as well as those community Rheumatologists, who often are diagnosing and getting patients our goal would be to get patients started on treatment immediately upon diagnosis.
So let me caveat this by saying we haven't done a formal salesforce sizing exercise yet, but you know we would envision a national footprint.
Ensuring coverage first and foremost of the roughly 50 glitter during that centers of excellence and we would envision that being duly.
Covered by a commercial customer facing team as well as a medical field medical team.
We would also one out of capacity to call on those community based rheumatologist, we're treating a sizable number of scleroderma patients in their practice.
Craig Millian: And, you know, right now, we estimate that number to be between 1,500 and 2,000 rheumatologists, and the cutoff we're using preliminarily is that they have roughly 10 scleroderma patients or more in their practice. But again, this is quite preliminary, and there's gonna be additional work done to validate that number. So, I would expect a fairly modestly-sized specialty sales team of no more than 50, but with national reach. And then, as I mentioned, we want to augment that with innovative multi-channel marketing approaches and, obviously, also have a strong field-based medical team.
And.
So right now we estimate that number to be between 1500 in 2000, Rheumatologists and a cut off were using preliminarily is if they have.
Roughly 10, 10, scleroderma patients or more in their practice, but again this is quite preliminary and theres going to be additional work done to validate that number so.
I would expect a fairly modestly sized specialty sales team, but no more than 50.
But with national reach and then.
Mentioned, we'd want to augment that with innovative multichannel marketing approaches and obviously also have a a strong.
Field based medical team.
John: Great, thanks for taking the questions and congratulations on the progress. Thank you. Thank you. Thank you, everyone. This concludes our question and answer session and our conference for today. Thank you for your participation. You may now disconnect your lines at this time and have a wonderful day.
Great. Thanks for taking the questions and congrats on the progress.
Thank you.
Thank you everyone. This concludes our question answer session at our conference for today. Thank you for your participation. You may now disconnect your lines to so I would have a wonderful day.
Operator: Thank you, everyone. Take care. Stay safe.
Thank you everyone take care thanks, Dave.