Q4 2020 Tricida Inc Earnings Call
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Hello, and welcome to the Tri State of fourth quarter financial for yourselves and business update conference. My name is Michelle and I will be the operator for today's call.
At this time all participants are in a listen only mode. Later, we will conduct a question and answer session and during the question and answer session. If you have a question. Please press Star then one on your Touchtone phone.
I'd now like to turn the call over to Jacky Cosman Tracy the senior Vice President of Investor Relations and Communications Ms. Coffman you may begin.
Thank you Michelle good afternoon, and thank you for joining the Tracy the fourth quarter 2020 financial results and business update conference call and today's call Garrett clearance of our founder CEO and President will discuss the response from the office of new drugs or OND.
Of the U S food and drug administration or FDA to our formal dispute resolution request or S. T. R. R also referred to as the appeal.
And Jeff Parker, our CLO and CFO will then discuss our financial results for the fourth quarter and our financial guidance.
Please note that in today's call, we will be making various statements that will include forward looking statements as defined under applicable securities laws forward. Looking statements include our anticipated activities related to our ongoing clinical trials, our secret D, which we'll refer to a seller our interactions and communications with the FDA, our plans and expectations regarding the.
Potential pathway to approval of the family by the FDA, including potentially available through the accelerated approval program and our expectations regarding our financial runway management's assumptions expectations and opinions reflected in these forward looking statements are subject to risks and uncertainties that may cause actual results to differ materially from.
Any results.
Performance or achievements discussed in or implied by such forward looking statements true.
Tracey the can give no assurance that these statements will prove to be correct and we do not intend and undertake no duty to update. These statements. We also urge you to read the risks and uncertainties associated with our business that are described in our filings with the Securities and Exchange Commission.
We issued two press releases. This afternoon, just after the close of market for copies of our press releases. Please go to www Dot Tracy the dot com and follow the link to our Investor Relations page at this time I will turn the call over to Gary.
Thank you Jackie and thank you all for joining us today.
Our primary goal for this call is to discuss the response from the <unk>.
<unk> in December of last year on <unk>.
Right and who the decision and the key points from the appeal of the night letter or <unk> from.
From the OND.
By way of background, we submitted the appeal solely requesting that the only defined that the magnitude of serum bicarbonate changes seen in the tier 301, and the POC a 301 of your trial. The drove a photo of 301 for you on he is reasonably likely to predict clinical benefit in the treatment of metabolic acidosis in patients with <unk> and the trial.
And then for serve as the basis for accelerated approval.
In summary, the OLED Tonight on on appeal.
The already addressed the issue of magnitude of standby, calling the change we had focused on but also address the other deficiencies identified on the zero, namely the reliability of the data from the tier ones of any trial due to the non risk due to the disproportionate impact of data from a single high enrolling clinical sites on the trials resolved and.
The ability of the trials of the U S patient population given that the majority of the subjects on the trial enrolled in sites outside of the United States or regions of the FDA does not consider U S like such as eastern Europe.
On the issue that was the focus of our FTR. The OND concluded, but the magnitude of the increases in standby covenant level of shown in the tier one for you when he trough, but not of sufficient size of durability to establish the treatment of the them I would be reasonably likely to provide the discernible reduction and think of the progression. In addition on the phone.
That the intended confirmatory of drove us to think of the U S.
I'm not part of the confirm a 13% reduction in floating uptick of the progression that level of risk reduction was derived from the two pieces of information and the initial NDA submission specifically the original of predictor of M. A model of the placebo subtracted L. S mean change from baseline to week 52, and standby call. It observed in the fear of wanting trial.
Come back for the topic of the part of Us.
The other later.
In addition, the <unk>.
And you indicated that there were concerns that I, particularly around an NDA supported by a single Registrational trial, such as of the assurance of adequate the blinding the disproportionate impact on the single high end well inside of the thought it was all of them. The majority of sites being in eastern Europe with differences in patient management, such as the concomitant medications diet might affect the treatment of respond to the very much.
And raise the concern of the pick of body two U S patient population.
I would note that with regard to the highest in line, but the idea of completing an inspection of the site and there was no FTA for employee three issued from the infection.
With respect the blinding the only does not suggest that there was a specific on blinding issue and the 300 ones here when he trough, but no the concerns around the adequate blinding of debt while the measures have been put in place. The protect the study's line of trial are reasonable they may not have optime protect of the blind the.
<unk> also discussed the physical functioning with us in the phase one for you any truck. It stated that the trial results showed improvement of two patient reported measures. The Kitty cure physical function survey and the repeated chair stand test and the <unk> noted that the results. Eventually eventually of established for one of more of the show trials would indicate the potential meaning.
For the benefit of the very much treatment, especially in CCAR, the patients who have physical functional functioning impairment. However, the only do you view of this data from the 301 for you when the trial of the skepticism in the absence of data from a second trial of a similar results and none of that both endpoints would require a vigorous bonding to support robust conclusions.
I would also note that the separate from the area. We previously with the feedback from the division of clinical outcome assessment. The reliance on these physical function of endpoints for approval may require further validation.
Now moving to the path forward.
Based on the feedback from the or the we believe that we now have greater clarity on the potential path for approval of the very most of the accelerated for the program.
The suggested that we need for the division to discuss submission of the week 52 serum bicarbonate of results from the full randomized trial of population of valor and that the trial should include a substantial proportion of patients from the United States of from regions with U S like patients if the reserve.
Also as in all of the from Baltic of do you want to demonstrate that the variable provides the meaningfully larger treatment effect and that's seen in the phase one for you when he trial than the volume trial, along with the three of them out there of any data could address the deficiencies race and the Sierra.
Over the R&D set debt, whether the extent of increase in serum bicarbonate and any subsequent submissions based on the other thing that he would support accelerated approval remains a review issue and we're in part reflect the division's assessment of the adequacy I E power.
To detect the anticipate the treatment effect of speak of the progression in the reasonable timeframe.
Moreover, based on the concerns of express we believe that the FDA could require additional trial the trials the confirm the magnitude of durability of effect of applicability to the U S population for Resubmission of the very man the sort of the accelerated approval of program.
One important point that I would like to clarify is that the only did not review of evaluate the outdated powering assumptions for the <unk> included in our protocol Amendment from Q4 last year that provide us with greater confidence that the trial of successfully achieved its primary endpoint of slowing seeking the progression based on the D 40, the there's renewed us yesterday.
Confirmed grading for a 40% reduction in Egfr. We've discussed is doing the year end update call, but to recap on our powering assumptions now rely on time, depending on predictive model based upon the cohort of more than 24000 U S patients with metabolic acidosis and see the results from this model show on eight 4% lower risk of seeking the progression for each one milliequivalent higher.
Serum bicarbonate.
So as we've described before we believe that the magnitude of the the very much treatment effect in the 301 for me. When you study is not best described by the between the difference of that what that means because of the data on not normally distributed in this case between group difference in the medians of more appropriate the week 52 of median placebo effect of the treatment effect.
The $3, one five milligrams per meter infill on it.
Based on the median treatment effect of that magnitude and using the time to pay the predictive model, we predict the hazard ratio of <unk> 76 for the valor <unk> confirmatory outcome trial, so where the sample size of 600 on subjects and 511 primary endpoint of event the trial, 87% power to show of 24% difference.
And primary endpoint event. Therefore, we believe that value I think it is adequately design and it's currently powered the confirmed the clinical benefit of of Verma treatment and slowing sick of the progression.
These current assumptions for the powering of the volatile not considered by the R&D and their response to the appeal and we have not yet received FDA comments on this revised protocol.
As such we continue to believe that the development of their most of the valor trial is warranted.
I believe at the time line to meet the requirements described in the ADL for accelerated approval may not result in the most rapid development path for development as discussed previously our current volatile protocol on interim analyses for early stopping for efficacy after the accrual of 150 and 250 subjects with <unk>.
Every endpoint of events.
As of February 22021, the voucher.
While it is randomized for 10 33 of fixing of subjects, but on average treatment duration of approximately one year and as the crude 69 subjects of positively adjudicated primary endpoint of events.
Based on the current rate of event. The crew. We believe the trial will have the crude hundred 50 events in the second half of this year and 250 event sometime on mid 2022.
Our approach to the interim analyses was to keep the majority of the offer for the final analysis and direct meaningful for the 250 of an analysis with very little off of direct it towards the high end for Stephen in terms of be seen in the dry run for 250 event into it. This is in line with the limitations of early Interims due to high around the variability the sofa.
Few events.
As I said earlier, our powering assumptions the yield of the hazard ratio of <unk> 76 based on the placebo subtract the treatment effect observed at week 52, and three of <unk> and the time, the predictive model of that hazard ratio.
They are of a 1% chance of stopping at 150 island and the 21% chance of stopping at the interim to the 65% power of of meaning for the final analysis, which is when 511 per of endpoint events out of dedicated.
So the first two interims the of accumulative probability of stopping by final analysis of 87%.
The interim looks for the stopping for efficacy.
Most relevant in the context of us on the estimating the <unk> ability to slow progression of spaghetti that is the true hazard ratio of for example, where of 0.7 O. Instead of <unk> 76, this would double the possibility of stepping away from approximately 20% to 40%.
Also to illustrate what we would have to observe and each of interim analysis to be successful if we observe the hazard ratio of less than 550 events.
Stop the trial early.
If we observe the hazard ratio of <unk>, six seven or less.
If you could stop the trial at 250 minutes.
Given some of the academic trials.
Now onto the <unk> study, we consider the interim to a worthwhile look an awful lot of spend.
If either of the planned interim analysis for early stopping for efficacy in the.
The trial was also positive outcomes data.
Also on positive outcomes data this data could be available before the 52 week serum bicarbonate data from the fully enrolled.
Trial as suggested by the on D.
That is why I said earlier that we believe at the time line to meet the requirements described in the ADL for accelerated approval may not result in the most rapid development path for them on.
Given the extent of timeline are we know of faceless folks are at approval. We are evaluating several options with respect to the balance of the kidney trial that are focused on obtaining prior to the end of 2022 additional data on the effect of of Irma on the CCAR the progression physical functioning and serum bicarbonate.
In addition to the currently specified ends of in August for early stopping for efficacy I've. Just discussed options also include the possibility of stopping the trial early for administrative reasons.
Which would allow analysis of data using our alpha of remaining at that time.
Provide an example of adoption of the trial was stopped at <unk> 50 of events assuming of true hazard ratio of <unk> 76 for you.
Half of 39% power and if instead of stopping at 150 of the trial of stop at 250 event the power increases to 58% the.
To provide you with the instilled in us as of the treatment effect is actually larger than the true hazard ratio of <unk> seven the power of 59% and 81% respectively for the two interim analyses.
Switching from power to observed hazard of based on statistics, we could be successfully the hand 50 of advent. If we observe the hazard ratio of <unk> 72, or 250 events. If you observe the hazard ratio of <unk> 78.
If stopping early for administrative reasons for it.
Here the positive result, it could potentially from the basis for Resubmission of the NDA, who the traditional approval pathway.
In summary, we believe data from a volatile on the very important and furthering on understanding of the regulatory path for approval of the very much for either the accelerate on traditional approval pathways.
Note Afghans acceptance of the valor data in support of an NDA submission, including the acceptability of the data from non U S countries or regions, which will comprise a substantial proportion of the data from the trial will ultimately be a review issue.
Further the FDA may require additional clinical data beyond debt provided by the Valor trial, Inc.
Concluding my remarks, I would like to extend my appreciation for the full team of Tri Cedar for the amazing work of difficult circumstances in 2020.
Like all of you on this call we've had to deal with the enormous complexity for the complexities of running a business during the COVID-19 from a mode of locations.
As of the days and weeks and months of zoom calls given our of regulatory setback. The also have to work for a significant company restructuring last year.
Today, the smaller number of commitment to continue to develop some of them of for patients with metabolic acidosis remained strong.
That I will turn the call over to Jeff Our new Chief operating officer on C. Chief Financial Officer, who has got expanded responsibilities from corporate and business development and he's going to tell you about our financial results and outlook.
Thank you, Eric and thanks, everyone for joining us on the call today.
Research and development expense was $27 $3 million for the three months ended December 31, 2020, and $148 $4 million for the year ended December 31 2020.
General and administrative expense was $21 8 million for the three months ended December 31, 2020, and $103 million for the year ended December 31 2020.
Net loss was $264 $8 million for the year ended December 31, 2020, non-GAAP net loss was $214 $4 million for this period.
As of December 31, 2020, cash cash equivalents and investments were $332 $3 million.
We currently have $75 million from a debt facility with Hercules, which is interest only until April of 2022 and has a final maturity of eight of October 2023.
<unk> $200 million in convertible senior notes due in 2027.
<unk> currently has the financial resources to fund its operations into at least mid 2022 prior to modify any of its material agreements. We are in advanced discussions to modify certain of these agreements and if successful would extend the company's financial resources beyond mid 2022.
As Gary has discussed we are evaluating several options with respect to the valor trial that are focused on obtaining prior to the end of 2022 additional data on the effective of armor on security progression physical functioning and serum bicarbonate.
These options include the possibility of stopping the trial early for administrative reasons.
Which would allow analysis of the data using all alpha remaining at that time.
Our goal is to obtain this of data the data from the valor trial within the timeframe of our existing capital resources.
With that I'll turn the call over to Gary for some final comments.
Yeah. This is obviously a.
A disappointing outcome that does provide greater clarity from the FDA on the accelerated approval path.
And I have to really highlight debt.
The term accelerated here.
As the regulatory term and not a temporal term I think that the message. We want you to walk away with is that the providing potential for traditional approval on the basis of.
Off of positive renal outcome data is really what what this team here is truly believing in and is working on while keeping all of the options.
On a viable. So this is really a key time for the company and for.
Bringing our health and believe two of the 3 million patients of the United States with metabolic acidosis and chronic kidney disease.
Our ultimate goal and we'll continue to work towards that end, we are as excited and we stay on by I think the strength of our data and of our compound.
With that operator of it can now open up the call for questions.
Thank you Sir we will now begin the question and answer session. If you have a question. Please press Star then one on your touched on the phone.
If you wish to be removed from the queue. You may press the pound sign of our the hash key.
Also if you think your speaker phone you may need to pick up on your handset first before pressing the numbers.
Once again to ask a question. Please press star one at this time.
Yeah.
The first question in the queue. It comes from Greg sect, the niche from Goldman Sachs. Please.
Please go ahead, Sir your line is open.
Hi, everyone. This is <unk>.
Jack on the line for Greg.
Really appreciate it.
All of the color on the ongoing discussions with the agency.
I'm curious, though it sounds.
Kind of the main concern with 301 of 300 line was really you know the regional makeup of the trials whether it was.
The U S patient population of enough so with that in mind I mean.
Do you feel like.
Serum Bicarb change is still potentially an approvable endpoint.
Or do you really think that disease progression is the way to kind of at this point and then a follow up.
The drag on that.
I think we.
We've characterized I think our view has not changed that debt the key.
The key concerns and issues that we had to address from FDA were around the magnitude of the fact, the durability of effect and the pick about age of the ice population I think that that Hasnt changed and we think that the you know the particular issue.
Around the the the thing aside.
As part of that debt overarching kind of concern.
We believe that debt.
There still is Inc.
On.
On the basis of serum Bicarb is of surrogate.
I think sort of you know.
All right.
The current state of play of per OND, instead, we would need all randomized subjects 69 subjects with one of your data and in our book that is a likelihood that that could occur later than the potential early readout from.
In terms of of the interim analysis in terms of being a provision so I hope that answers your question.
Yes, yes, that's very helpful.
And then maybe more specifically on some of the OND concerns about sort of the powering assumptions behind valor.
You know lets say theoretically there potentially right about there maybe not being as much wiggle room.
How was that.
In the form potentially stopping the trial early in 2022 of them you know if it turns out that the trial at that point might be now.
The 50% powered or less would you still think about going that route of wood or is there some kind of the.
Threshold of percent, Howard, where you'd feel comfortable stopping the trial for administrative reasons.
Yes, just to be clear the powering assumptions as obviously are those of the upfront the assumptions and what we've learned is that Unfortunately, you know we've always had a very interactive.
And if you know our relationship with with the cardio renal division sort of advancing some of the various models. However, the F. D. R V.
We really have to focus on only on inflammation, that's in that that the knee in the NDA and the original NDA submission and so we really feel strongly that debt. Some of those earlier versions of of the powering assumptions and the interpretation of the same bicarb results.
We're not the most up to date view of it and that was.
The more of up to date view, we are very confident that debt is a good probability of of stopping on them.
Alright, thank you.
Thank you and the next question in the queue comes from Jessica Fye with J P. Morgan. Your line is open. Please proceed.
Hey, guys. Thanks for taking my questions.
I guess first I just thought one of the follow up on.
Craig's question.
You have any plans to provide the FDA with 52 week Bicarb data from valor.
Is that still kind of on the table at the Interims don't hit and you kind of get to that time point.
Yeah, I mean definitely I didn't get the.
It's something that are that is.
It's one of the the potential auctions in the past that we are pursuing it's more I think would be one of the highlight the sort of the misnomer of you.
Of accelerated in the context of timing of a potential readouts. So so it doesn't mean that we don't believe in it or we wouldn't pursue it. It just means that we think that the interim analysis.
Happen earlier.
Okay.
On it and maybe related to that is there any reason to expect the stronger treatment effect on serum bicarb in dollar than what you saw in San Juan and free of money.
Yes.
We've improved screening criteria.
To ensure that we have truly astronautic patients in the study.
And there are other reasons that the we can comment on because of the ongoing trial, but we are confident that you would see.
You know the significant.
The increase.
The increase serum bicarb of effects some of the or larger blocks of five cup of effects on the median of sleep on the one five.
And Geoff it's Jeff I would comment that for our powering assumptions, though we don't need to assume anything greater than 315.
What was actually observed in the median difference and the 301 study of $3. One five of that goes into our analysis, we're not assuming anything greater than that when we think about either of the interims or the stopping.
The the differences the the percent reduction in renal events per one mill equivalent debt really is the.
Is the factor that you need to take into account and based on the time dependent model of course, that's $8 four per cent.
Okay got it and maybe just the last one given the way.
It seems like the large proportion of ex U S patients.
Would empower already how do you think about the ftes willingness to accept those results. It's successful for example, if you do hit on one of these interims.
Yes, I think you know having done this before a couple of times and the team many of them.
The team members here income.
On a go on development I've worked on the about half of the program that the light to greater than 90% on a.
On the eastern European data and really having looked at the patient by patient a patient level data and for once everyone. He believes that the dose patients that you know after the same underlying disease and the same comorbidities and treatments.
You know as you asked like patients, but let's be clear of the the feedback from.
From OND.
Is is quite definitive on the concern and you know we we can assume that this is only going to be in the context of accelerated approval and Thats I think thats why why are we on.
On focusing on recruiting additional U S like patients here and the remaining yeah.
152 on the subject.
Yeah.
Got it thank you.
Thanks for that.
Thank you and the last question in the queue comes from any thought of proof of Terra with Cowen. Your line is open. Please proceed.
Hi, Thank you so much for taking my call I just had a question about the renal event rate for Tyler.
Has it been consistent with the on expectations and what kind of factors could cause it to the theory.
Yes, I think we are.
For the current number of 69.
For the 511 events you know I think that's in line with all of our expectations and that the it keeps us on track for the.
The the timing in the second half.
Of this year for the $1 50, and second half of next year for the $2 50 now the X.
Vacation for our steering committee and the world experts of run multiple of his trials of is that the rate is going to increase as patients are in the study for a longer period of time, it's well known that in the first year or.
Or to the pure of elements.
Because of better care of just being part of the study of being seen more often by a physician and then it really sort of goes up significantly.
And so our expectation is that we continue to see an increase in that in that range and we'll monitor it carefully and update on that periodically.
Kind of things that can impact it of.
Of course, you know our studies of specific you know, we obviously have to make sure that.
We keep of our patients in the study and you know we also monitor COVID-19 related events. So.
Those are all the things that we watch very.
Carefully to ensure that all patients remain on the study and again the accrual of the right number of events to get interpretable data.
Great. Thank you and then just a follow up from what you mentioned about Covid time has it affected your ability to monitor patients and also has it affected the maybe the timing of the Interims.
So on the timing of the interim we.
We don't believe it has affected the.
So far.
And and yes.
We've put specific COVID-19 measures in place.
Oh really independent.
To allow sort of home delivery of study drug and the other things and and we're managing that very actively and.
I think that's obviously given given that sort of such such a major a worldwide pandemic that's debt.
High on the list of things to to consider them for all ongoing clinical trials.
Okay. Thank you very much.
Yeah.
And we have no further questions at this time, so I'll turn the call back over to Jacky Kauffman for any closing remarks. Thank you Michelle and thank you all for joining us on the call today as always if you have additional questions. Please don't hesitate to email us at IR at <unk> Dot com. Thank you very much and goodbye.
Ladies and gentlemen. This concludes today's teleconference. Thank you for participating you may now disconnect.
Yeah.
Okay.
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