Q3 2019 Earnings Call
For the Corpus Pharmaceuticals quarterly update conference call and webcast at this time, all participants certainly listen only mode. A question and answer session will follow the formal presentation. If anyone should acquire operator assistance. During the conference. Please press star zero enter telephone keypad. As a reminder, this conference is being recorded it is on my pleasure to.
Introduce your host Ted Jenkins Senior Director Investor Relations and corporate Communications. Please go ahead Sir.
Good morning, everyone. At this time, we'd like to remind our listeners that remarks made during this call me state management's intentions hopes beliefs expectations are projected to the future.
Forward looking statements and involve risks and uncertainties forward looking statements on this call are made pursuant to the safe Harbor provisions in the federal Securities laws.
These forward looking statements are based on corpus as 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 corpus files with the Securities and Exchange Commission.
These documents are available in the 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.
John Moran, our Chief Financial Officer, and Craig Williams, our Chief commercial officer with that is my pleasure to turn call over you've all thank you did and thank you everyone for joining the call. This morning in the third quarter of 2019, we continued to build our R&D engine.
Execute on our clinical development program.
Prepare for transformational 2020 and start laying the foundation for successful commercial launch.
I want to start by reminding you all of our vision as a company.
We believe the targeting the body's endocannabinoid system also known as the easiest holds the potential to provide new therapies to treat inflammatory fibrotic and metabolic diseases.
We are focused on developing potential novel medicines that modulate this powerful biological system.
We have a deep expertise in medical additional chemistry, Endocannabinoid system biology, regulatory and patent strategy as well as clinical development.
More recently, our attention has turned to laying the groundwork for commercialization.
It's exciting to see other pharmaceutical companies engaged in the development of compounds that modulate functions of the easy yes.
Recent examples include Lebeck, Takeda Roche and Johnson and Johnson.
It is our belief that medicines targeting the endocrinologists and have the potential to reshape the treatment paradigm for inflammatory antibiotic diseases, giving you hope to patients.
We expect data from our phase three study in systemic sclerosis, and our phase Twob study in cystic fibrosis next summer.
In addition, we have an additional candidate being prepared for phase, one and a growing portfolio of unique molecules designed to moderate the functions of the Endocannabinoid system.
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 programs Barbara. Thank you Paul.
Let's now listen it's an oral small molecule cbtwo agonist that reduces inflammation and fibrosis in cellular and animal models of disease.
Promising safety and efficacy data were observed in our initial phase two studies and systemic sclerosis, cystic fibrosis sender Madam last item.
Our resolved one global phase three study unless Madison and systemic sclerosis is fully enrolled with 365 patients ghost.
Topline data are on schedule and expected next summer.
Yes data are positive and the safety profile is acceptable we plan on having discussions with regulatory authorities in the U.S. Europe and Asia.
Filing applications for marketing authorizations for analysis.
We are optimistic about the upcoming topline data with systemic sclerosis.
Our optimism is based on the results of mechanistic studies phase two safety efficacy and biomarker data and supportive safety efficacy and biomarker data in the phase two study of analysis and the related rare autoimmune disease dramatic myositis.
Our optimism.
Is further supported by two year data for our ongoing phase two open label extension study of lab, assuming systemic sclerosis to follow the double blind placebo controlled study.
The long term safety profile avlon, absolutely favorable to date.
80% of this subject to energy open label study remain in it at two years.
Durable improvement in clinical outcome measures has been seen in this open label study.
There will be an oral presentation of the latest open label data at the HCR 2019 annual meeting this Sunday results and presentations will be available on our website.
The determine global phase three study of line ABA cylinder matter myositis is actively enrolling subjects.
We are delighted with a high level of engagement enthusiasm from patients and investigators enrollment goals are currently being met.
We expect to complete enrollment in 2020 with topline results in 2021.
Again, we are optimistic that the determine phase three study will show positive efficacy data and to support a safety profile Berlin Apis and Intermountain mastitis for similar reasons a systemic sclerosis.
Our optimism is supported by data from our ongoing phase two open label extension study of Annapolis and under matter Myositis that followed the double blind placebo controlled study.
The safety profile as Lynn Alison International Mastitis has also been favorable to date.
90% of subjects, who entered the open label study remain in this study at two years.
That's in the systemic sclerosis open label study durable improvement in clinical outcome measures has been seen.
There will be an all presentation of these latest open label data at a CR on Tuesday, well results and presentations will also be available on our website.
The 415 patient phase Twob study is ongoing in people with.
Cystic fibrosis, who are at high risk for recurrent pulmonary exacerbations.
I'm very pleased to announce the patient screening for this study has ended.
Topline data for this 28 week study are expected in summer of 2020 .
As a reminder, pulmonary exacerbations in cystic fibrosis, our accused events have increased slung inflammation with clinical manifestations of worsening respiratory signs and symptoms.
Often including a significant worsening in lung function.
Failure to fully recover lung function after a pulmonary exacerbation cautious about half of long term decline in lung function experienced by people with cystic fibrosis.
Despite exciting advances in the treating us is in the treatment of such.
The fibrosis.
We see F T R targeting therapy.
People with cystic fibrosis.
Still have exacerbations.
There remains an unmet need.
For additional approaches for the prevention of pulmonary exacerbations.
The 100 patients space too low Nablus and study in people with systemic lupus era stem is ptosis continues to enroll.
Jack.
This study is funded in managed by the National Institutes itself.
We anticipate study completion and top line data in 2020 .
The second drug in our pipeline CRP.
One.
It's a peripherally restricted she'd be one inverse agonist designed to avoid the central nervous system size affects seamless from on a bar.
We are targeting Nash with fibrosis as the potential first indication for CRD 4001.
[noise] preclinical data show beneficial effect on energy metabolism.
Inflammation and fibrosis.
A phase one safety study is scheduled for read out in 2020.
Lastly, we highlighted eight promising compounds at our R&D day in June .
These are new Cbtwo agonist as well as CB one inverse.
Agonists.
These compounds are progressing through candidates all action.
We are excited about the progress we have made today.
We look forward to continuing to share our data.
Upcoming conferences and through scientific publications.
I will now turn the call back to evolve Thank you Barbara.
Even though we are scheduled.
Data readout.
From the route so all foreign systemic sclerosis study.
And phase to be cystic fibrosis study next summer it's critical that we begin planning for success.
Laying the groundwork and preparing for the potential approval and then commercial launch.
I would now like to turn the call over to our Chief commercial Officer Craig million.
We'll provide you with an update on our commercial activities.
Yeah.
Thank you to evolve and good morning.
Let's take a few minutes to highlight the progress we're making preparing for this excess launch of loan assets.
As we prepare for upcoming data we are building that thing class commercial capabilities that will give us maximum flexibility to commercialize ourselves.
Or partner out specific geography, such as Asia.
At this point in pre launch planning, we're focused on three critical elements to ensure a success.
First.
Building, our commercial leadership team in capabilities.
Second establishing a strong foundation each market insights.
And third communicating a compelling and concise narrative that will provide an appropriate scientific context.
Okay and have a potential regulatory approval.
We are continuing to build an experience leading cross functional team.
That will drive is successful in that that some launch.
Over the past six months Corpus has hired team leaders from marketing supply chain.
And medical Affairs.
And then September we hired keep why has that our vice president of market access.
He most recently served as VP of market access and pricing it intercept pharmaceuticals, where he led global managed to access functions for the launch and commercialization OCO leap up in PBC.
Keith is already digging into healthy economics.
I live in pricing, okay, or engagement and distribution channel strategies.
Yes, we recently completed and that paging journey research.
Well I hinted interviews were completed with systemic sclerosis patients and with academic and community based rheumatologists.
They told us about the devastating impact of this disease on patients' lives.
And they're frustration with guarding the lack of treatment option that all through the course of this disease.
There was an acknowledgment that there are a limited number of available treatments usually immunosuppressants.
Or others selected based on specific organ involvement.
There was consensus that there are no therapies that address the totality of this disease.
These insights will be foundational to developing or go to market strategies product positioning now.
Value proposition and market segmentation.
Not surprisingly when we shared the blinded target efficacy and safety profile profiles for low NAV. This.
There was considerable interest and enthusiasm from both patients and physicians.
Arms with these insights we are now finalizing an unbranded disease education campaign.
I will give voice to the unmet need for new treatments and systemic sclerosis.
This food Vecamyl dialogue around new scientific approaches, including E C S.
To address the totality of this devastating disease.
This disease Education campaign will start in Q1 2020.
In conjunction with an increased president at medical conferences and engagement with thought leaders.
In addition, systemic sclerosis, we've been preparing for Lin absence potential opportunity in cystic fibrosis.
As such we conducted market research with Pulmonologists from C F treatment centers.
We just come back from the North American cystic fibrosis annual meeting, where we think advances and see a treatment which celebrated.
Conversations with experts at this meeting combined with the recently completed market research.
From that remains a need for new anti inflammatory treatments.
Particularly for patients with established lung disease, who continue to exacerbate.
To sum up our market research reinforces the opportunity we have within these first two indications.
We are ensuring strong execution around commercial launch fundamentals.
And I look forward to engaging with you further in the coming months as we advance our commercial capabilities and prepare for lunch.
With that I'll turn the call back over to evolve.
You Craig.
Blood to provide a brief updates on our financial position.
Corbett has a strong balance sheet.
Having ended the quarter with approximately $55 million in cash.
Given the additional milestone payments expected from the cystic fibrosis Foundation.
For our phase to be cystic fibrosis study.
Our capital should be sufficient to support our operations.
Topline data read outs and into the third quarter of 2020.
We have important catalyst ahead.
We will deliver key data readout from too late stage studies of the Annapolis and in this emerging 2020.
We anticipate.
First clinical data in lupus.
The first human data in CRB four 001 will also be available in 2020.
And lastly, we look forward to increasing our global reach.
Continuing to explore additional partnerships abroad.
Such as those in Asia.
In closing I would like to re emphasize corpus is pioneering trends.
[noise] formative medicines.
The target the endocrinologist.
We believe that this biological system holds the potential to improve the treatment of inflammatory and find a body diseases.
We're pivoting towards executing the successful launch of our first product.
With that I'd like to say Q3 over time and attention.
And turn it over to the operator for any questions from our listeners today.
Operator.
Thank you will not be firmed up to your question answer session. If you like you play. Some good question can you. Please press star one or telephone keypad.
Confirmation totaling indicate your line is in the question Q.
You mean press star to feed like Trooper question from the Q.
For participants choosing speaker equipment, and maybe this is already to pickup or handset before pressing star one.
One moment, please for we pull for questions.
Our first question today is coming from Brian Abrams from RBC. Your line is now lives.
Hi, Thanks for taking my questions and thanks for all the updates.
Particularly on the on the market research progress there I guess my first question is I'm curious to know.
Key learnings have been asked for patients who have now been.
Systemic sclerosis patients has now been on treatment.
For for multiple years, and maybe if you could frame expectations for what we might look for.
In the upcoming a CRM data and now with though the last caught.
You were starting to see.
Disease stabilization and just curious what.
We should be looking for there.
Oh.
So Brian Thanks for the question is just Barbara.
I think I'm delighted to say you shouldn't expect rather soon.
You should expect to see.
Oh really favorable long term safety profile from are these patients.
Okay and severity of learning.
The double blinded and tying the.
Open label on some of them beyond what Weve pork genius.
There are multiple years on the drug.
Acceptable safety and now I want to position that importance for that because that is important to the an extension study with important if you remember as Greg pointed out.
Many of these patients right.
Mmm suppresses treatment armed guards, it's not in this process and the ability to deliver.
'cause C.
Without any suppression incontact standard of care is super important.
Secondly, the efficacy data.
Our beautifully.
Accordingly persist.
They are doing well, Paul we couldnt, yeah fragrance anymore than that so I think.
You should expect Laura this name and we are delighted.
Right and then bonds, so yes I.
I was curious if you could talk a little put about the impact you might expect from some of the.
Likely very recent changes.
In the standard of care and in terms of the impact to that the conduct of the phase to be study.
And maybe your ability.
To teach out a benefit and ensure that the arms are balanced.
Sure I'll be glad to do that so first of all the.
As a sad that we've identified all the patients this study.
The U.S. patients.
Came in first.
Cause that.
The way decides to go after day.
So you patients are and they have skin in the study.
Many of 'em someone more out of this.
All righty.
With that triple therapy being available.
It is only appropriate that if it is in subjects best interest that they are allowed to begin triple therapy.
That that is medically appropriate.
Okay, and we're also looking forward to having no gain.
Because we believe.
Yeah, well now outlets NIM will offer treatment benefit on top on these very important standard.
Hair treatments for the patients.
We actually look forward to having started some subjects get on triple combo drawings and he will have a.
Patients on the others here John modulators.
So we're hopeful that impact some of these patients U.S. will actually start triple therapy.
We don't think will be a law thatll be some I think it's less likely both subjects for me.
Well get on Triple therapy direct contact.
Right.
And I think that it will be dealt with you when in the terms of sensitivity analysis from us.
In place.
Let's turn it over to Craig first from Oh.
Comments about the importance yeah, yeah. Thanks prior to the question and we did our market research, which we conducted with about 20.
Pulmonologists from sea of treatment centers we.
Yeah really built into the the.
The dialogue or the questions you know.
Anticipating the introduction of Triple combo on what that would mean.
For their patients and unmet need.
And specifically for a a product with the profiles will now this.
And you know based on that research as well as frankly conversations we even just had last weekend and they see us see.
With a with leading pulmonologists.
We're very confident that even with the reach in the approval of.
Right CAFTA, there will likely remain a sizable number patients.
Who would potentially benefit.
From a safe and effective anti inflammatory medicine.
As far as said, yes, many adults with cystic fibrosis have established lung disease with a considerable infectious and inflammatory component.
And it's not clear that CFTR modulators alone will entirely prevent these patients from experiencing pulmonary exacerbations in the future that's what we heard.
From physicians in the market research and at that meeting.
Many physicians you we spoke with express their strong belief that those patients with established lung disease, who continue to exacerbate will almost certainly still need novel therapies.
Yeah, and Brian if I can share.
An anecdote so I was the.
Booth today, if you if the and.
We had a patient they're not many patients of course that any if you've got a patient approached us and she's on the triple combo. She is going on it for a while who expanded access.
Her long functions every low.
And.
It was it was remarkable to cure from her just how key she was about the potential.
Being on on the mabus into here on the one hand, absolutely the effects of Triple combo add on her.
But on the other and.
We're just seeing with your own eyes, the the gap that were still left.
In her.
In her treatment.
Specialty around pulmonary exacerbations.
That's helpful. One more for me if I if I could.
Any updates on the formulation work for four 001 and the timelines for when we might see that had stayed in next year and I know some ladders and the NIH his hands anything but just curious if you had anymore.
Visibility on that and I'll hop back in the queue. Thanks.
Sure, Yes, I'm Anja.
Delighted to say that well, we've got a formulation to take into.
Thanks for the question this is John Barbara.
There are a lot of data upon which to develop a range of specific binding to CB, one and those data largely come actually from a monobore. We noted that the 20 milligram dose of the non amount was associated with.
Just unacceptable CNS adverse events and the five milligram has not so and their data in the literature about how much receptor occupancy there would be at least in rodent brains and when can extrapolate human range, so without going through all those calculations I would say, yes, we have an internal set of range that.
It.
Is likely to be acceptable I don't think it has to be nothing but it has to be low enough.
To have reasonable.
Assumptions that we shouldn't see that type of CNS side effects. So our I'll stop there, but say, yes, we can anticipate that and later on again, if I can but I see you know I often find it really interesting to juxtapose on Addison Weve CRB 4001 in the following week with low now December it really is shaping up to be.
The seminal question is does it work is the efficacy data going to be positive at the end this clinical study coming up.
I don't think too many people are losing sleep over the safety profile going out of some at least to date of course CRB 4001 is the polar opposite of it.
The seven or question here the crux of it is does it have a meaningful engagement with CB one in the brain if it happens that engagement and it's not worth pursuing but if it doesn't then oddly enough the clinical efficacy of it.
Yes, I wouldn't say it's predictable.
But it's very close to that remain about was a very very potent drug for a variety of things. It's critical efficacy was never in dispute.
So.
Webcast at this time, all participants certainly listen only mode.
Question and answer session will follow the formal presentation, if they didn't want to acquire operator assistance. During the conference. Please press star zero under telephone keypad.
As a reminder, this conference is being recorded it is on my pleasure to introduce your host Ted Jenkins Senior Director Investor Relations in corporate Communications. Please go ahead Sir.
Good morning, everyone.
This time, we'd like to remind our listeners that remarks made during this call me speak management's intentions hopes beliefs expectations are projected to the future.
These are forward looking statements and involve risks and uncertainties forward looking statements on this call are made pursuant to the safe Harbor provisions of the federal Securities laws.
These forward looking statements are based on core versus 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 corpus files with the Securities and Exchange Commission.
These documents are available that 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., you've all Cohen, our Chief Executive Officer, Dr. Barber White, our Chief Medical Officer and head of research.
John Moran, our Chief Financial Officer, and Craig Williams, our Chief commercial officer with that is my pleasure to turn the call over to evolve.
Thank you did and thank you everyone for joining the call. This morning in the third quarter of 2019, we continued to build our R&D engine execute on our clinical development program.
Pair for transformational 2020 and start laying the foundation for successful commercial launch.
I want to start by reminding you all of our efficient as a company.
We believe the targeting the body's endocannabinoid system also known as the easiest holds the potential to provide new therapies to treat inflammatory fiber optic and metabolic diseases.
We are focused on developing potential novel medicines that modestly this powerful biological system.
We have a deep expertise in medical additional chemistry, Endocannabinoid system biology, regulatory and patent strategy as well as clinical development.
Recently, our attention has turned to laying the groundwork for commercialization.
Exciting to see other pharmaceutical companies engage in the development of compounds that modulator function of the E. Yes.
Recent examples include Lubeck, Takeda Roche and Johnson and Johnson.
It is our belief that medicines targeting the endocrinologists and have the potential to reshape the treatment paradigm for inflammatory antibiotic diseases, giving you hope to patients.
We expect data from our phase three study in systemic sclerosis in our phase Twob study in cystic fibrosis next summer.
In addition, we have an additional candidate.
I think repaired for phase, one and a growing portfolio of unique molecule designed to moderate the functions of the Endocannabinoid system.
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 programs Barbara. Thank you Paul.
Well in Addison, it's an oral small molecule cbtwo agonist that reduces inflammation and fibrosis in cellular and animal models that disease.
Promising safety and efficacy data were observed in our initial phase two studies.
Good luck sclerosis, cystic fibrosis under Madam last item.
Our resolve one global phase three study unless Madison and systemic sclerosis is fully enrolled with 365 patients dosed.
Applying data are on schedule and expected next summer.
Yes data are positives and the safety profile is acceptable we plan on having discussions with regulatory authorities in the U.S. Europe and Asia.
Piling applications for marketing authorizations for Olin Amisom.
We are optimistic about the upcoming topline data with systemic sclerosis.
Our optimism is based on the results of mechanistic studies phase two safety efficacy and biomarker data.
And supportive safety efficacy and biomarker data in the phase two study of analysis and the related rare autoimmune disease dramatic last items.
Our optimism.
Is further supported by two year data for our ongoing phase two open label extension study of lab instruments systemic sclerosis, the followed the double blind placebo controlled study.
The long term safety profile of one absolute is favorable to date.
And 80% of this subject to enter the open label study remain in it at two years.
Durable improvement in clinical outcome measures has been seen in this open label study.
There will be an oral presentation of the latest open label data at the AC Our 2019 annual meeting this Sunday results and presentations will be available on our website.
The determine global phase three study of land ABA cylinder matter myositis is actively enrolling subjects.
We are delighted with the high level of engagements enthusiasm from patients and investigators enrollment goals are currently being met.
We expect to complete enrollment in 2020 with topline results in 2021.
Again, we are optimistic that the determine phase three study will show positive efficacy data and to support a safety profile Berlin Amisom Intermountain mastitis for similar reasons a systemic sclerosis.
Our optimism is supported by data from our ongoing phase two open label extension study of an advocate and then dramatic myositis. The followed the double blind placebo controlled study.
The safety profile as Len Alison International Mastitis has also been favorable to date, 90% of subjects, who entered the open label study remain in this study at two years.
That's in the systemic sclerosis open label study durable improvement in clinical outcome measures husbands gene.
There will be an oral presentation of these latest open label data at a CR on Tuesday results and presentations will also be available on our website.
The 415 patient phase Twob study is ongoing in people with.
Cystic fibrosis, who are at high risk for recurrent pulmonary exacerbations.
I'm very pleased to announce the patient screening for this study has ended.
Topline data for this 28 week study are expected in summer of 2020 .
As a reminder, pulmonary exacerbations in cystic fibrosis, our accused of events.
<unk> increased slung inflammation with clinical manifestations of worsening respire, Tory signs and symptoms, often including a significant worsening in lung function.
Failure to fully recover lung function after a pulmonary exacerbation cautious about half of long term decline in lung function experienced by people with cystic fibrosis.
Despite exciting advances in the treating ups is in the treatment of cystic fibrosis, we see STR targeting therapies.
People with cystic fibrosis still have exacerbations, there remains an unmet need for additional approaches for the prevention of pulmonary exacerbations.
The 100 patient phase two letting advices study in people with systemic lupus erythema ptosis continues to enroll subjects.
This study is funded in managed by the National Institute of Health.
We anticipate study completion and topline data in 2020 .
The second drug in our pipeline CRB 4001 is the peripherally restricted CB one inverse agonist designed to avoid the central nervous system side effects seen with from Audubon.
We are targeting Nash with fibrosis as a potential first indication for CRD 4001.
Preclinical data show beneficial effects on energy metabolism inflammation and fibrosis.
A phase one safety study is scheduled for readout in 2020 .
Lastly, we highlighted eight promising compounds at our R&D day in June .
As our new Cbtwo agonist as well as CB, one inverse agonist.
These compounds are progressing through candidate selection.
We are excited about the progress we have made today.
We look forward to continuing to share our data at upcoming conferences and through scientific publications.
I'll now turn the call back Tivo, Thank you Barbara.
Given that we are scheduled for data readout from the resolve on systemic sclerosis study and phase Twob cystic fibrosis study next summer. It is critical that we begin planning for success laying the groundwork and preparing for the potential approval and then commercial launch.
I would now like to turn the call over to our Chief Commercial Officer, Craig millions, who will provide you with an update on our commercial activities Greg.
Thank you you evolves and good morning.
Like to take a few minutes to highlight the progress, we're making and preparing for the successful launch of one abbott's them.
As we prepare for upcoming data we are building best in class commercial capabilities that will give us maximum flexibility to commercialize ourselves or partner out specific geographies such as Asia.
At this point in pre launch planning, we're focused on three critical elements to ensure success.
First building, our commercial leadership team and capabilities.
Second establishing a strong foundation deep market insights.
And third communicating a compelling and concise narrative that will provide an appropriate scientific context ahead of a potential regulatory approval.
We are continuing to build an experienced lean cross functional team that will drive a successful when after some launch.
Over the past six months Corbis was hired key leaders for marketing supply chain and medical affairs.
And in September we hired Keith White, as our vice president of market access.
Keith most recently served as VP of market access and pricing it intercept pharmaceuticals, where he led global managed access functions for the launch and commercialization of Ocaliva in PBC.
He is already digging into health economics value in pricing.
Engagement and distribution channel strategies.
Next we recently completed index patient journey research.
Brian did interviews were completed with systemic sclerosis patients and with academic and community based rheumatologists.
They told us about the devastating impact of this disease on patients lives in the frustration regarding the lack of treatment options that all through the course of disease.
There was acknowledgment that there are a limited number of available treatments, usually immunosuppressants or others selected based on specific organ involvement.
There was consensus that there are no therapies that address the totality of this disease.
These insights will be foundational to developing or go to market strategies product positioning value proposition in market segmentation.
Not surprisingly when we shared the blinded target efficacy and safety product profile Berlin Abis.
There was considerable interest and enthusiasm from both patients and physicians.
Armed with these insights we are now finalizing an unbranded disease education campaign that will give voice to the unmet need for new treatments and systemic sclerosis.
This will begin at dialogue around new scientific approaches, including Dcs to address the totality of this devastating disease.
This disease Education campaign will start in Q1 2020 in conjunction with an increased presence at medical conferences and engagement with thought leaders.
In addition to systemic sclerosis, we've been preparing for an absence potential opportunity in cystic fibrosis.
As such we've conducted market research with Pulmonologists from CF treatment centers.
We just come back from the North American cystic fibrosis annual meeting, where recent advances and see a treatment which celebrated.
Conversations with experts at this meeting combined with the recently completed market research affirm that there remains a need for new anti inflammatory treatments, particularly for patients with establish lung disease, who continue to exacerbate.
To sum up our market research reinforces the opportunity we have within these first two indications.
We are ensuring strong execution around commercial launch fundamentals.
And I look forward to engaging with you further in the coming months as we advance our commercial capabilities and prepare for launch.
With that I'll turn the call back over to evolve. Thank you Craig I'd like to provide a brief update on our financial position.
Corbis has a strong balance sheet, having ended the quarter with approximately $55 million in cash.
Given the additional milestone payment expected from the cystic fibrosis Foundation for our phase Twob cystic fibrosis study, our capital should be sufficient to support our operations from top line data read outs and into third quarter of 2020.
We have important catalyst ahead.
We will deliver key data readouts from too late stage studies of the in Addison in the summer of 2020.
We anticipate the first clinical data in lupus.
The first human data in CRB four 001 will also be available in 2020.
And lastly, we look forward to increasing our global reach by continuing to explore additional partnerships abroad, such as those in Asia.
In closing I would like to reemphasize at Corpus is pioneering transformative medicines the target the endocrinologists system.
We believe that this biological system holds the potential to improve the treatment of inflammatory antibiotic diseases.
We're pivoting towards executing the successful launch of our first product.
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.
Operator.
Thank you will not be conducting a question and answer session if you'd like to be placed in the question can you. Please press star one on your telephone keypad, a confirmation Tony will indicate your line is in the question Q.
Let me Prostar too if you'd like to remove your question from the Q.
Participants using speaker equipment and may be necessary to pick up or has had before pressing star one.
One moment, please will be poll for questions.
Our first question today is coming from Brian Abrams from RBC. Your line is allies.
Hi, there thanks for taking my questions and thanks for all the updates, particularly on the on the market research progress there.
I guess my first question is I'm curious to know what key learnings have been off for patients who have now been.
Systemic sources patients who have now been on treatment.
For multiple years, and maybe if you could frame expectations for what we might look for.
In the upcoming HCR data I know with the last cut you were starting to see.
See stabilization and just curious what what we should be looking for there to follow.
So Brian Thanks for the question this is Barbara.
I think I'm delighted to say you should expect to see where other soon you should expect to see a totally favorable long term safety profile for these patients.
Considering the amount of time in the double blind and the amount of time in the.
Open label on some of them well beyond what we'll report continue after multiple years on the drug to have an acceptable safety and I want to position that importance of that because that is important to the an extension study. It's important if you remember as Chris pointed out.
Many of these patients on immunosuppressive treatments and our drug is not immune suppressive any ability to deliver efficacy without immune suppression in the context standard of care is super important.
Secondly, the efficacy data our beautifully boringly per system. They are doable, we couldn't ask for anything more than that so I think.
You should expect more at the same and we are delighted.
Great and then on C. F. I was curious if you could talk a little bit about the impact you might expect from some of the.
Likely very recent changes.
The standard of care.
In terms of the impact to that the conduct of the phase Twob study.
Maybe your ability to tease out of benefit and ensure that the arms are balanced.
Sure I'll be glad to do that so first of all the.
As I said that we've identified all the patients for the study.
The U.S. patients came in first because that's just the way the sites where activated so U.S. patients are and they have skin in the study.
Many of them some of them are out at this time already.
With that triple therapy being available on it is only appropriate that if it is in the subjects best interest that they are allowed to begin triple therapy. During study that is medically appropriate.
And we're also looking forward to having no state.
Because we believe.
That blend Alison will offer treatment benefit on top on these very important standard of care treatments for the patients we actually looks forward to having some some subjects get on triple combo. During the study we will have a patients on the other CFTR modulators so well.
Awful, but in fact some of the patients in U.S. will actually start triple therapy. We don't think will be along that will be some I think it's less likely that the subjects from New York well, we'll get on triple therapy during the context of the study.
And I think that it will be dealt with you when in the terms of sensitivity analysis from a statistical standpoint, so I'm going to turn it over to Craig for some current comments about the importance yeah. Yeah. Thanks. Prior to the question you and limited our market research, which we conducted with about 20 pulmonologists from sea of treatment.
Centers, we really built into the the.
The dialogue or the could the questions you know.
Anticipating the introduction of Triple combo, what that would mean for their patients and unmet need and specifically for a product with the profile of when Allison.
And based on that research as well as frankly conversations we even just had last week at end HCFC with with leading Pulmonologists.
We're very confident that even with the reach an approval of try CAFTA.
Likely remain a sizable number of patients who would potentially benefit from a safe and effective anti inflammatory medicine.
As far as said many adults with cystic fibrosis have established lung disease with a considerable with infectious and inflammatory component and it's not clear that CFTR modulators alone will entirely prevent these patients from experiencing pulmonary exacerbations in the future if thats, what we heard from physicians in the.
Market research and at that meeting.
In fact, many physicians you we spoke with express their strong belief that that those patients with establish lung disease, who continue to exacerbate will almost certainly still need novel therapies.
And Brian if I can share up an anecdote. So I was at the booth at any CFC and.
We had a patient or not many patients of course, if any if you've got a patient approach us and she's on the triple combo. She has been on it for a while through expanded access.
Prolong functions very low and.
It was it was remarkable to hear from her just how key she was about the potential of being on on the Matheson.
Into here on the one hand, absolutely the effect the triple combo add on her but on the other hand, just seeing with your own eyes. The the gap there was still left.
In her.
In her treatment, especially around pulmonary exacerbations.
That's really helpful. One more for me if I, if I could any updates on the formulation work for four 001 and the timelines for when we might see that pet data next year I know the latter is in the NIH. His hands I think but just curious if you had anymore.
Visibility on that and I'll hop back in the queue. Thanks.
Sure, Yes, I'm on Jeff.
Delighted to say that well, we've got a formulation to take into our phase. One study we have some real experts and they have.
Driven.
On the formulation to allow us to have the desired solubility and dose response relationship that we need to enter the clinic. So we got this data and we're very pleased with them and as I said, we expect to have the read out from the phase one.
Single ascending dose and multiple ascending dose.
In 2020, I have been an extension extensive discussions and have visited the NIH a couple of times to.
Well move forward.
The design and the commitment of the NIH for the pet scan so because it's the NIH, we're not there quite yet I am actually hesitant to commit on timelines and I will just say that we appreciate the need to address our with human data.
Whether or not 4001 lines, a significant number of CB one receptors in the brain wanted to deliver that therapeutic levels.
We will do that but I asked for a little more time to be from on timelines with that with the studies to them and Brian if I can but in.
And I don't know how many people pick this up I think some of you folks have.
If you remember.
We've always had a competitor in the form of Johnson and Johnson.
Is there a bird rock monoclonal antibody targeting CB, one so same mechanism, but obviously very different compound.
And it was really intriguing to see I think about two three weeks ago. Another enter into two that field, which was takeda with gold fish.
Again targeting the same CD one receptor a different organ. This time, the kidney very sensible CB, one sees a pretty much the same.
Those areas rolling the kidney that it doesn't deliver so this is an area focusing on the endocannabinoid system, which seems to be moving very very rapidly. They are now three companies, including ourselves again the differences we have an oral systemic drug.
The other two entrance have monoclonal antibodies.
Thanks, so much.
Thank you as a reminder, ladies and gentlemen that star one to be placed in the question Q1 moment. Please what we pull for questions.
His question is coming from Murray Wright from Jefferies. Your line is now lives.
Hey, everyone. Good morning, and congrats on the project progress. Thanks for taking my questions I'm, just wondering poor HCR coming up.
What we should be focused on their with some of the update you guys have and is there anything on their competitive landscape and we should be looking up or at the meeting and then I was just wondering also or youre eventual filing for systemic scleroderma.
Well you include long term safety data from the open label extension study in a in that filing.
Sorry, let me just.
With the last one first because I can remember it and then ill [laughter] first on.
So in terms of the data that we will submit to support AC at the time of on any potential filing that of course will need to be discuss with FDA any M&A and PMTA, we will engage in those discussions that we certainly think it's likely because that will be represent long term.
Safety exposure and certainly not in a placebo group, but in a in a treated group. So it would be our expectation that that will go in and helps support.
Take a good look it's a safety findings.
In the poster and again I think what we've got it HCR are a couple of things.
To remind you we got all presentations, we were just delighted that the HCR leadership felt that the data were important enough to warrant oral presentation of both of these open label extensions in the matter Matthias and in systemic sclerosis, we think that emphasizes the unmet need that way.
Support for novel treatments in these diseases, so pay attention to safety and efficacy I Hope you will be delightfully board.
With what you've seen here because you can seem more favorable safety in more durable efficacy.
We also are going to present.
In a poster form the baseline characteristics that subjects in the phase three that should be of interest to you because it's going to show that they look a lot like the folks who are in the phase two study all the global these will be middle aged white, ladies in large part who are sick who have a lot.
A lot of disease activity as assessed by baseline MRF Seth.
Hi, a global assessment by the patients by the physicians by their functional disability by their own pulmonary involvement is are sick people from background in the suppresses the ones that are in very much in NIM treatment ones for whom we believe we showed potential clinical benefit and thanks to so I think that was.
An important for you said just line up yes. This is what they expected that's I think.
That's why should take a look at the poster I think in terms of competitors.
When we look over it.
Again, I think we're pretty far advanced we should be the first out with that.
Assuming.
We find efficacy and safety, we should see the first took a win and look for approval to address the totality of disease, that's as Craig thought and I think to that.
And we've had a lot of discussions in the past about our primary efficacy outcome, Chris and I think you should note that some of that the the companies are studies that are just a bit behind us our using this now using the same outcomes.
In their attempt to say is there efficacy here is there any early read maybe that's what would move for us. So I would just say keep your eyes open for the early studies what are they doing what are they using and I would say keep an eye out for discussions about the HR christen estimating.
Great. That's very helpful and second question just on the reclaim general molecules just if theres any.
More specifics on when those could potentially move into the clinic or when you potentially partner does our partner one of those are there anything on the strategic side you can comment on those sure sure I'll I'll start it's not just generally we have our own compounds that we've made ourselves the cbtwo agonist that we're looking at.
Come from and terminal I think that.
We certainly are making progress internally, we reported on the eight we've got our own favorites.
Those eight theres some front runners.
We look forward to formally selecting the first candidate from our pipeline that will occur in the next few months and we would anticipate moving forward them to do the necessary studies to prepare prepare to enter clinic and 2021.
And more what I'm really excited about is.
This gives us.
A bunch of thing it creates a a real viable platform.
No longer depending on just one asset.
It also uses that asset to validate the rest of the platform, which I think will be a very powerful synergy, but last but not least it gives us the ability to start having dialogue with potential partners at the preclinical stage to look at indications that we ourselves would not be the appropriate company to develop for example, we're.
Really really good and our year towards rare disease commercialization, that's really our bread and butter. That's what we're excited about especially obviously North America versus Asia, where were commercializing that the platform has application for multiple inflammatory fiber optic and even metabolic diseases, most of which are not going to be rare.
Oh for us to develop let alone commercialized common diseases, I think doesnt make a lot of since this allows us to have those discussion with them to present, what we think our unique compounds. We obviously fresh IP around them that I think we'll be more and more interesting, especially as we see more and more big pharma coming in.
Through the endocrinologists.
Got it was very helpful. Thanks, again for taking my question.
Yes.
Thank you. Our next question today is coming from Leland Gershell from Oppenheimer. Your line is our lives.
Hi, good morning, Evault and team. Thanks for taking my questions just a quick one back on 4001.
If you could remind us has there been said a.
Sort of an upper limit of acceptability for penetration into the brain as as we wait the.
<unk> data and if so.
What that might be in terms of permitting a go forward development. Thanks.
Thanks for the question this is that Barbara.
There are a lot of data upon which to develop a range of specific finding TCB won and those data largely come action from among them on we noted that the 20 milligram dose from online was associated with.
Just on acceptable CNS adverse events and the five milligrams must not so and their data in the literature about how much receptor occupancy there would be probably slowed and brains and when can extrapolate human rights.
Without going through all those calculations I would say, yes, we have an internal sort of range that is likely to be acceptable I don't think it has to be nothing but it has to be low enough.
To have reasonable.
Assumptions that we shouldn't see that type of CNS side effects. So I'll I'll stop there, but say, yes, we can anticipate that and later on again, if I can but I think you know I often find it really interesting to juxtapose the Madison Weve CRB 4001 in the following Wade will now this and it really is shaping up to.
The the seminal question is does it work is weak efficacy data going to be positive at the end this clinical study coming up.
I don't think too many people are losing sleep over the safety profile going out of some at least to date of course CRB 4001 is the polar opposite of it.
The seven no question here the crux of it is does it having meaningful engagement with CB one in the brain. If it has is that engagement and it's not worth pursuing but if it doesn't then oddly enough the clinical efficacy of it.
Yes, I wouldn't say it's predictable.
But it's very close to that remain about was a very very potent drug for a variety of things its clinical efficacy was never in dispute. So this is a program, which I think is a little bit unusual because.
I think the value inflection oddly enough you is going to be all about the safety once we clear the safety.
The rest of it becomes much more easy to predict.
And then just as a brief though technical scientific remind our I forgot latency share.
Our drug from our drug 4001.
Also differs from them on a bond in its binding affinity for the splice variants.
CB one call CD Wendy that is prefer that preferentially expressed in the liver.
No one that we think is important in driving some of them.
Pathogenic Nash and that is really just not expressed in the brain and so we think that at levels, which we can have adequate receptor occupancy.
By 4001, CB, one being the liver that that that differential finding will actually also improved safety profile improve safety margin that we have because that's the when you need to engage more than the regular season one.
Okay. Thanks for the comprehensive answer and look forward to use your.
Excellent.
Thank you. Our next question today is coming from Liisa Bayko from JMP Securities. Your line is now lives.
Hi, Thanks for taking my question John I'm for Lisa I was hoping you could tell us a little bit more about the preclinical programs that you won't be able to do R&D day summer, what's kind of looking promising anymore characterization is there something that looks like we'd ahead of the rest and then I also.
The reason.
Kidney.
Partnership first would be one I guess I was hoping if you can discuss kind of maybe branching out in the more nephrology. Thanks.
Sure Yeah, we we right now have a current paying for it.
Yes.
One called for 96, Okay. That's it's number.
And it is a CD running it is just.
Keeping one person and it's got some very favorable.
Biologic activity in formation in fibrosis assets, and some favorable characteristics and we're really pleased with it so far it's got a long road to.
So far which we acknowledge that but it's it's probably the elite horse right now.
I think in terms as we think through.
Potential indications I couldn't agree more beauty of the targeting inevitably system in Sydney, one inverse agonist in particular is there potential efficacy in fibrosis in multiple organ, whether it's the liver kidney along the heart, there's just a lot of data testing.
That dish has the potential be efficacious, so as we move.
The one inverse agonist forward and right now our lead horses for 96 that could change.
We are thinking about where the best initial indications are for that.
And what John if I could but in.
Well first of accrued as I think you're the one who actually has the heads up on on that one so that was really really interesting.
Talking about CB to for a second.
I think I mentioned, a little bit earlier, so what's happening there is.
Pretty much out of the Blue Roche came up with a publication looking out to Cbtwo agonist. So conceptually similar to the Nab as Tim.
Preclinical data early days looking at the mouse model for Uveitis and my guess your Eve.
They are constrained to a topical application that's not unusual with some of these synthetic comm Ave. It's that if there are difficult to formulate otherwise.
But that was really interesting uveitis is a very very logical approach to dealing with inflammation in the I threw CB too.
It's interesting to see.
How big pharma again is dipping their toes into this biology, placing bets on compound and what I really excites me about our preclinical pipeline is we have all of that covered.
For a big pharma, that's interesting targeting or neutralizing CB. One we have those compounds for big pharma interested in agonizing, we're activating CB too we have those covered as well and again with patent or there are typically much fresher vendors.
Thanks for the coming up these guys.
Thank you, we reach and of our question and answer session and ladies and gentlemen that does conclude today's teleconference and webcast. You may disconnect. Your lines at this time and have a wonderful day. We thank you for your participation today [noise].