Q4 2020 CTI Biopharma Corp Earnings Call
Ladies and gentlemen, and thank you for standing by and welcome to the Q4 2020 C. T. I Biopharma Corp earnings Conference call. At this time all participant lines are in a listen only mode. After the speaker's presentation. There will be a question and answer session to ask a question. During the session you will need to press star one.
And on your telephone please be advised that today's conference is being recorded if you require any further assistance. Please press star zero and I would now like to hand, the conference over to your speaker today, Dr. Adam Craig. Thank you. Please go ahead Sir.
Thank you Gigi and welcome to this afternoons conference call. Joining me today are David <unk>, Chief Financial Officer, and Bruce Seeley, Chief Operating officer. Following formal remarks, the conference call will be opened for questions. Before we begin. Please note that during this call we will be making forward looking statements based on current expert.
Patients such forward looking statements are not guarantees of future performance and are subject to risks and uncertainties that may cause actual results to differ materially from those anticipated by the forward looking statements additional information concerning these risks and uncertainties are contained in today's press release.
<unk>.
This quarter C. G I executed on critical activities and further advance per <unk> towards a U S approval and commercial launch.
Pulling NDA submission for the use of Chris and he'd been myelofibrosis patients with severe thrombocytopenia, such as platelet counts of less than 50000 per liter is nearly complete and we remain on track to finish the submission by the end of this month.
As we have previously discussed the formats of the NDA was agreed with the FDA and will consist of a submission package and based upon the available data from the completed phase III persist and persist two studies and the phase two part two or three study with a focus on the severely thrombocytopenic patients enrolled in these studies.
Including frontline treatment naive patients and patients with prior exposure to JAK inhibitors and.
And persist two in patients with severe thrombocytopenia, who were treated with <unk> 200 milligrams twice a day 29 percentage of patients had a reduction and spleen volume of at least 35 per cent compared to three percentage of patients receiving best available therapy, a statistically significant findings and the.
Same population for <unk>, and it was well tolerated with reversible and manageable side effects.
If the FDA accepts the NDA and granted priority review and we could receive and accelerated approval for Christmas and by the end of 2021.
And at that point, the ongoing phase III Pacific is study is expected to be completed as opposed to approval confirmatory study.
<unk> and this trial has recently improved in part due to the abatement of the COVID-19 pandemic and we continue to expect to report topline primary efficacy data in 2023.
Our preparations for the commercial launch of progressing hip all progressing well.
Over third and if the existing myelofibrosis population has severe thrombocytopenia approximately 7000 patients.
To address this unmet medical need we have now hired key leadership in marketing sales medical affairs and market access over the coming months, we will focus on our disease awareness campaign market access and customer engagement and the deployments of our field force.
Last year, we launched the study and Procrit nabe and severe COVID-19 patients and response to the pandemic. This study prevent is a randomized double blind placebo controlled multi center phase III study of <unk> and they've been hospitalized patients with severe COVID-19, comparing <unk> plus standard of care.
Care versus placebo plus standard of care and hospitalized patients.
Primary endpoint of the trial will assess the proportion of patients who progressed to invasive mechanical ventilation and ore extra corporal membrane oxygenation or die by day 28, we expect to reports and interim analysis from this study in mid 2021.
Before I turn the call over to David to review iPhone, all financials I want to touch on and additional indications that we have been investigating that is the use of <unk> and the prevention of acute graft versus host disease or gvhd.
And at Ash, The American Society of Hematology meeting and December data was presented from and investigator sponsored phase one and two study showing that and even pre Christmas to the standard prophylaxis of civil lineup and low dose Tucker limas resulted in a significant reduction and the expected acute graft vs.
Host rates and patients within the first 100 days of therapy as compared to historical data without comprise compromising transplantation outcomes and without any new safety concerns.
The phase II component of this studies non rolling she is interested and in this indication remains high and we look forward to providing an update on the progress of the phase III trial later in the year with that I'll now turn the call over to David to review, our quarterly financials, David Thank you Adam.
As of December 31, and 2020, our cash and cash equivalents totaled $52 5 million compared to 33.7.
<unk> 7 million as of December 31, 2019, we expect current cash and cash equivalents enable us to fund our operations into the second quarter of this year.
Operating loss was $14 8 million and $47 8 million for the three months and year ended December 31 2020, respectively.
Compared to an operating loss of $9 5 million and $40 7 million for the respective periods in 2019.
No revenues were reported for the three months and year ended December 31, and 2020 as well as for the three months ended December 31, 2019, while revenues of 3.3 million were recognized for the year ended December 31 2019.
Net loss for the three months ended December 31, 2020 was 15 million or 20.
For basic and diluted loss per share compared to net loss of $8 2 million or 14 for basic and diluted loss per share for the same period in 2019 net loss for the year ended December 31, 2020 was 52 five.
Million or <unk> 74 per basic and diluted loss per share compared to a net loss of $40 million or <unk> 69 cents per basic and diluted loss per share for the same period and 2019.
So with that I will hand, it back to you Adam. Thank you David So in summary, we're delighted today to be close to the completion of the NDA submission and.
And for the potential from the acceleration of the approval of <unk> by the end of the year.
For use in patients with severe and modified myelofibrosis with severe thrombocytopenia.
Currently underserved patient group.
That concludes our formal remarks, operator, please open the call for questions.
As a reminder to ask a question you will need to press star one on your telephone to Luis.
So on your question press the pound key.
And the standby, while we compile the Q&A roster.
Our first question comes from the line of Ren Benjamin from JMP Securities. Your line is now open.
Okay.
Ren Benjamin from JMP Securities. Your line is now open please check your mute button.
Hey, Good afternoon, guys can you hear me okay, yes.
Yes, we can and high risk.
Hey, Thanks for taking the questions and congrats on all the progress.
Maybe just starting off with the with the application.
You know you mentioned that it will be completed by the end of this month have there been any sort of further discussions with the regulatory agency on the way to the completion of this application Adam or is it pretty much all and balls and Euro court at this point.
And then there's as you as the application gets reviewed do you anticipate you know sort of back and forth and and ultimately I think I've asked you. This from the past and I want to know if there's any any changes you anticipate any sort of a panel.
Discussions potentially coming out from it and ultimately by the end of the year.
I'm, assuming and approval can.
Can you just remind us what sort of how we should be thinking about the commercial opportunity as you as you build it out you know I know youre doing the disease awareness campaigns and Theres a lot of things happening right, now, which I'd love to get some more color on but ultimately when you're launching how does that launch look for you guys.
Yes, so now I'll go through the questions. Thanks for all of US are rent and.
To start with about the application. The FDA was very clear we have a very good pre NDA meeting and it was very clear to us what was required of us they.
And there was a great deal of clarity during that meeting they specifically wanted us to focus on presenting data around the 200 milligram twice a day dosing of <unk> and severely thrombocytopenic patients. So that's what we that has been the sense and the focus of the NDA and we will be the focus of the label the draft label.
We will submit very shortly and there has been some correspondence with the FDA, but really mainly during the review process classifications of points there hasnt been any major dialog with them with regard to the.
And the NDA, because I think as I said they were extremely clear to assess to what was expected of us and we have addressed I believe we have addressed all of their concerns and and application that is very thorough and very quickly.
With respect to the panel and.
Currently I think the day test shows that we all are very similar to the other drugs and the disease area and other tyrosine kinase inhibitors with respect to our safety profile and efficacy data, we think is pretty clear with the 2009 versus 3%.
So you are right that I just referred to so at the moment, we don't see that being a panel. However is at the discretion of the FDA and.
And it will be low.
And we all we will prepare for that eventuality until we hear otherwise from the FDA currently if we file by the end of the month, we should hear from them by the end of May whether they've accepted the filing and at that point they should indicate whether they believe that should be a panel and nuts and.
And then the commercial opportunity I'd, rather not go into too much detail about the commercial opportunity at the moment, because Bruce Seeley, who is with me still working on that at the moment. We're suffice to say we have hired the key leadership in sales marketing Medical Affairs and.
Market access and we have done a considerable amount of work identifying al why the patients. All so we understand how we can deploy our field force over and over the coming months, but at this point I'd, rather not go into too much detail on that and just leave at that.
Okay Fair.
Fair enough.
And as long as you don't mind I'll, probably ask that the next conference call.
As you get more clarity I guess, just leaving one last question from me regarding Gvhd, you mentioned updated data.
Later this year is it is it fair to assume by Ash of this year is that how we should be thinking about it and then.
Maybe related to that how does.
We have recruited threads, the needle and M and but there's the landscape is changing quite a bit and gvhd can you talk a little bit about how you envision procrit and threading the needle and gvhd.
Well as I said and the opening remarks, I with very interested and the graft versus host data publication for that data is going to come out very shortly and I think it's exciting opportunity for us to expand the use and.
And potentially expand the label of per Chris' nib.
The data of the study is being conducted by the University of Minnesota, and the Moffitt Center and they've indicated on the enrollment of the.
On phase II study is going well and I would expect there to be some data by ash to to answer your question with respect to the broader use of and Q acute graft graft versus host I think these the proof of concept of using per Chris' nib in this setting.
It has been very positive and we are as the team expanding other ways of using the drug and graft versus host but for the for the timing of our main focus at the moment is the acute graft versus host indication.
And.
Perfect. Thanks, very much for taking my questions.
Thank you Ryan.
Thank you. Our next question comes from the line of Chad Messer from Needham and company. Your line is now open.
Great. Thanks, Good evening, Thanks for taking my question and.
Congrats on being so close to completing a potential filing.
You had mentioned.
Labeling and I'm just wondering what you know.
Whatever is appropriate and that can be discussed at this point, given where you are about your thoughts on labeling I know and.
This was discussed as a way of.
Addressing.
<unk>.
Pat safety concerns just just wanted to make sure we have your latest thoughts on that.
Yes. Thank you Chad, it's nice to speak to you again.
The labor and we spend an awful lot of time and the labeling and we've gone through the safety day to the entire day to safety.
Data set included primarily the data from the less and 50000 population, which is what the FDA is most interested in and we think in the labeling we can address previous safety concerns that the FDA and the warnings and precautions section, particularly around any bleeding or any cardiac events.
After reviewing the data and a great deal of detail. We do not think there is a justification for black box warning. So we have in our proposal trough label to the FDA, we're not going to include a black box warning for hemorrhage.
And obviously this will be a negotiation once the FDA has started to review the data, but overall I was very when we looked at the integrated day to all the data together I was very encouraged and very pleased to see that the side effect profile of Procrit and <unk> was very manageable and very predictable and.
We think we can address the concerns and with normal label language as others have within the warnings and precautions section of the label.
Okay, great. Thank you very helpful and very encouraging.
We've touched base on this before but just wondering you know here on debt on the eve of a filing.
Filing with FDA.
You have had any further thoughts on on just.
General EU strategy.
Well the the EU.
EU is <unk>.
As indicated previously that they would like the Pacifica phase III trial for completion to be complete before we submit we're continuing to review our position on that and obviously with the UK.
Pricing away from Europe. The UK is now as a separate entity.
The focus to be honest with you given the size of our company the focus over the last six months has been the NDA, but once the NDA is in place myself from my regulatory colleagues colleagues, we will take some time and looked at.
Reassess, our regulatory strategies and other territories.
Okay Fair enough and then and then.
Just and you.
I'd like to get your thoughts here on on cash position, obviously index into two Qs not.
Super enviable, but.
Just looking back at my notes from from the last quarter that that runway appears to have shortened.
Just wondering if there's a particular reason for that.
Anything you can say about the options, you're considering too to address yes.
Yeah, we're not going to comment on that today, except for the <unk>.
And that David has already presented chat, but thank you for your question and just one point Chad in terms of R. R.
Accelerating.
Costs, that's associated with the commercialization and as we built on that debt infra.
Infrastructure.
Alright, great. Okay fair enough. Thank you guys.
Thank you. Thank you just a reminder, too.
Yes.
As a reminder to ask a question you will need to press star one on your telephone to withdraw your question press the pound key.
Our next question comes from the line of Thomas Flaten from Lake Street Capital. Your line is now open Hey.
You guys and let me add my congrats to just a couple of housekeeping on prevent and Gvhd on the prevent study Adam I think you had increased the interim patient group up to 200 patients is that still the right number we should be thinking about.
Yes, a scratch on US Okay, and then could you remind us.
With the study going on and Gvhd.
Your options are.
Do you have full access to all the data that's being generated by the by the U of M and Moffitt team or can you just remind us what your relationship is with that team doing that research.
Good relationship we do we do have access while we're talking and have currently we don't have access on the phase III, but we will be when the data is mature we will bear to look and say that's and the agreement with the Moffitt and Minnesota, who who are who have who could partner and say they want to move this forward as much as we do.
So we will be able to look at the data and then the options for US as you know at that point and the truckload reached b at the end of phase two we're going to look at the data set and we are going to see if there is a potential approval pathway for acute graft versus host with Procrit and.
And that may be based on phase II data that may be the current phase II day. So it may be based on us generating additional data we won't know until we have a discussion with the FDA.
And then just a couple on the on the run up to a potential launch here by the year and as we're thinking about spending what's your trigger for kind of mass hiring of sales reps and kind of junior marketing folks that I'm sure there's going to be a bolus of people coming into the company is that going to be once you have and acceptance to file it.
The bulk of the hiring is after we have acceptance for ha high.
Let Bruce comment on that briefly and.
And so you're right.
Gate and predominantly the validation of the filing as Adam mentioned at the end of May and then as far as the sales force is concerned we're going to bring them on.
Closer to the premium per day.
And just on that have you guys settled on a on a final number of sales reps that you need on on if you've completed that exercise with the ups or whoever you're using.
No we have not.
Finalize that.
And process now.
And then just one final one from me with respect to drug supply how do we stand with that.
Manufacturing has always been.
A very well established a very strong component of the company.
We had plenty of dry product actually podium manufactured some years ago and.
And we have no concerns about the manufacturing there'll be plenty of drug.
Supply patients within the first year, and then as things Firstly, a commercialization and if things progress we'll stop manufacturing additional.
Drug.
Excellent I appreciate you guys, taking the questions and congrats again thanks.
Thomas.
Thank you at this time I am showing no further questions I would now like to turn the call back over to you and foster Adam Craig for closing remarks.
Thank you Gigi I'd just like to thank everyone for joining the call today, we look forward to further conversations over the coming months.
Ladies and gentlemen, this concludes today's conference call. Thank you for participating you may now disconnect.
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