Q4 2020 X4 Pharmaceuticals Inc Earnings Call
Greetings and welcome to X for Pharmaceuticals, fourth quarter financial and operating results conference call. At this time, all participants on a listen only mode.
Question and answer session will follow the formal presentation.
As a reminder, this conference call is being recorded it is now my pleasure to introduce your host Danbury of lifestyle advisors. Please begin.
Thank you operator and.
Good morning, everyone. Thanks for joining us.
Presenting on today's call will be X Force, Chief Executive Officer, Dr. Paula Ragan.
And the company's Chief Financial Officer, Adam and stuff.
Following prepared remarks by each we will open up the call to your questions.
And we will be joined by a Doctor Diego Covid, Chief Medical Officer, <unk>, Chief Scientific Officer, and Mary do you foresee senior Vice President technical operations and quality.
As a reminder, on today's call the company will be making forward looking statements regarding regulatory and product development plans.
As well as research activities.
These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.
Description of these risks can be found and X force most recent filing with the SEC.
I'd now like to turn the call over to Paula Ragan Paula.
Thanks, Dan and thank you everyone for joining us on the call. This morning, we hope that you're all safe and healthy.
And with many companies 2020 through some unexpected and significant challenges our way certainly the COVID-19 pandemic with chief among them.
And working within a community where many patients are immuno compromised that resulted and challenges for the enrollment of our clinical trials. However.
However, I'm extremely proud of the work done by our team and mitigating. These obstacles, we made significant progress and the clinical development of our lead candidate Maverick before and have an experienced leadership team and a place to execute on our 2021 goals and beyond.
As a reminder, maverick before it is our first in class small molecule antagonist and the chemo kind receptor C X C. R. Four being developed as a once daily oral therapy to treat a number of conditions caused by excessive signaling and the CX. Therefore see X L 12 pathway.
Our most advanced Mavericks four program is and whim syndrome as you may recall.
Carl when is a rare inherited primary immunodeficiency diseases caused by gain of function <unk> mutations that prevents the healthy trafficking of immune cells and effective and you know surveillance.
We believe Maverick and four has the potential to be the first disease modifying therapy for them more than 3500 estimated diagnosed and undiagnosed when patients and the U S.
We are currently conducting a global phase III clinical trial and women syndrome. The four women trial is a randomized double blinded placebo controlled multicenter study designed to evaluate the safety and efficacy of Maverick before and genetically confirmed whim patients.
The primary efficacy endpoint for the trial will compare the level of circulating neutrophils relative to the clinically meaningful threshold and response to treatment with maverick before versus placebo.
Over the course of 52 weeks.
Secondary endpoints include infection rates, wart burden and assessments of immune system function and quality of life.
Given the current pace of enrollment and the fact that this is a 52 week study we continue to anticipate top line data from this phase III trial in 2020 two.
Notably during the fourth quarter of 2020, we received both fast track and rare pediatric designation from the FDA from Maverick before for the treatment of women syndrome.
And these F D. A designation further recognized whim as a serious condition with a clear unmet need and both adult and pediatric populations and underscore the importance of our efforts to advance this program as rapidly as possible.
Through the fast track program, we will be eligible for more frequent meetings with the FDA to discuss the drug development plan and protocols and the clinical data that would support Mavericks and fourth potential approval for whim.
As you May recall Maverick before was previously granted breakthrough therapy designation by the FDA as well as orphan drug status by the FDA and the European Commission for the treatment of Whim syndrome.
With the rare pediatric designation and we may qualify and the future for a priority review voucher, which if awarded could potentially be monetized.
In the meantime, we expect additional data this year to further support our confidence and the outcome of the four whim trial, notably the open label extension of our phase two clinical trial and whim has continued to dose patients. We anticipate presenting detailed updated results from this study within the next year.
Since we expect that Mavericks, where it will be used as a chronic therapy. If approved these long term data are quite meaningful to the patient and physician communities.
We are also continuing our research and to whim prevalence characterizations of specific when mutations and compiling patient case studies offer the potential presentation at upcoming medical conferences and other venues in 2020, one and beyond.
Again with a goal of better understanding of the wind market for future commercialization and also increasing our visibility in the whim patient physician and K O L community is.
Turning now to our other ongoing maverick before our clinical programs and.
<unk> is progressing and our phase one b trial investigating the safety and efficacy of Maverick before and combination with Ibrutinib for the treatment of a subset of sic see our formula and patience with Walden from macro globule anemia.
A rare form of non Hodgkin's lymphoma.
We look forward to presenting initial data from this study and the first half of 2021 with a more robust dataset expected to be per cent and later this year.
As previously discussed while I'm from results from a somatic mutation and that M. Y D. Eight eight gene that is present and more than 90% of patients approximately 30% to 40% of these patients have a second mutation and the CXC are forging.
These double meat and patients with both the M Y D. Eight eight and CXC are four mutations tip.
And typically present with a more severe disease profile and do not achieve the same depth and robustness and responses as and patients with the single N. <unk> eight mutation treated with the current standard of care.
Our targeting is high need double mutant subset of Walden from patients as we assess the potential of maverick's first treatments in combination with Ibrutinib and our ongoing phase one day trial.
The phase one B multicenter open label dose escalation clinical trial is expected to enroll approximately 12 to 18 patients in.
In addition to safety dose and Pharmacodynamic markers. The study is designed to evaluate changes and serum immunoglobulin M and hemoglobin from baseline both key biomarkers of clinical response and Walden trauma patients.
And lastly regarding our phase one b trial in severe congenital neutropenia, we continued to anticipate initial phase one data from this study later in 2021.
As you can see we expect that 'twenty 'twenty, one will be a year of key value driving catalysts for X four across clinical data read outs enrollment completions and breath of opportunity initiatives.
On our last call. We briefly introduced you to Dr Art to virus, who had just joined as our new Chief Scientific Officer.
As he mentioned he has a wealth of experience and discovering and developing novel next generation <unk> antagonist.
<unk> is an ideal fit to lead our R&D initiatives and has already had significant positive impact on our research activities and on our earlier stage pipeline initiatives.
Following art's joining in November we also welcomed Doctor Diego Ocado viewed as our new Chief Medical Officer, having.
And having previously served and key senior medical leadership roles at Fulcrum Therapeutics, and Biogen Diego has brought us broad and deep clinical experience and the rare disease space. He.
He has also proven and valuable to us already as we continue to advance our multiple clinical programs in parallel and further advance our preclinical pipeline towards the clinic.
With that summary, I'll now turn the call over to Adam to discuss our financial results for the quarter Adam.
Thanks, Paul and thanks to all of you on the call today.
As presented in our press release this morning.
And will summarize our financial activities and results for the fourth quarter and full year ended December 31, two.
And in 'twenty.
As of the end of the year X four at $80 7 million and cash cash equivalents and restricted cash.
We continue to expect that our cash and cash equivalents will fund our operations into 2022.
Research and development expenses were $12 3 million and $41 9 million for the fourth quarter and full year ended December 31, 2020, respectively as.
Compared to $7 1 million and $30 2 million for the comparable periods in 2019, respectively.
General and administrative expenses were $5 4 million and $20 9 million for the fourth quarter and full year ended December 31 2020, respectively.
As compared to $3 9 million from $17 6 million for the comparable periods in 2019, respectively.
Finally X four reported net losses of $18 4 million and $62 1 million for the fourth quarter and full year 2020, as compared to net losses of $10 8 million and $52 8 million and a comparable periods in 2019, respectively.
Let's now open up the call for questions operator.
Thank you as a reminder to ask a question you will need to press star one on your telephone.
Joe Your question press the pound key please standby, while we compile the Q&A roster.
Our first question comes from Stephen Willey with Stifel. Your line is now open.
Yeah. Good morning, guys. Thanks for taking the question.
I guess, Paul and maybe you can just kind of frame up expectations for us in terms of what this first windows or Walden streams and disclosure might look like just in terms of.
Yes.
Maybe timing and theirs.
Potential bad news on the medical conference and the first half of the year.
What we might be able to expect in terms of just patient numbers and then also with a median duration.
A follow up on those patients.
Sure. Thanks, Steve So the plan.
Plan is to present the data at a medical conference of course, that's not always and our control, but we are like me to aim for one of them more visible conferences like <unk>.
And there's some other conferences around that same time frame that.
And that could be a good fit as well.
And in terms of the data it's an open label phase one b as you know with three different cohorts. So we'll present me presenting data, which will support a demonstration of obviously safety and dose escalation and then patients have joined the trial at various time points. So we will be providing them you know.
A range of treatment times for that initial cohort of patients.
And also be providing I G M changes as well as hemoglobin changes, which are indicative of activity I think the most important thing for people that are recognizable aimed to provide some relevant benchmark. So people can put our data and context, what standard of care and so I think that's a that's the plan at this point.
Okay, that's helpful and.
I guess as you continue to characterize some of these additional.
<unk> mutations.
Have you gained and the any insight as to whether or not.
Certain mutations within Walden streams are more oncogenic just in terms of the.
Pathogenicity.
Yeah. So there there are theres, one referenced and our literature that talks about sort of two different buckets of mutations and Walton from some possibly a little bit more impactful than others and we certainly reflect on that and I think that's important and set of information to be tracking at this point, we're tracking that with.
And our patience, but well use our own data set to better inform us on how to think about variant mutations and how they may or may not impact clinical outcomes. I think just overall the data are still early across the board and so we look forward to contribute and hang that to that total dataset with our own study.
Okay, and then just lastly, and.
And maybe just talk about some of the pediatric work that you do.
And they have plans for.
I guess, specifically and the context of that and the.
And the voucher now potentially available and I think I think the age cut off and for Windows 12 years, if I remember correctly.
Yes, that's right. So the ongoing study does capture the adolescent population in terms of expanding our studies beyond the age of 12 were currently evaluating that certainly our goal is to always.
Move forward and a safe way for patients and then expand as appropriate and so we're learning that is certainly on our sort of near term list of priorities and we'll be glad to update you on the a better clarity on how we'll be initiating studies and younger populations.
Alright, thanks for taking questions.
Okay. Thanks, Steve.
Thank you. Our next question comes from Joel Beatty with Citi. Your line is now open.
Hi, Thanks for taking the questions first one thanks for that and the previous question you talked about what to expect from this phase one one from steady coming around from later in the first half could you compare that with what to expect and the data set that is expected towards the end of 2021.
Sure. So most phase one studies and Walden strong obviously, our focus on safety and dose, but predictably. They also are assessing changes and IGN as well as hemoglobin as a measure of activity against the disease. So the early part of this year, we'll be looking.
At changes and and those two metrics and addition to safety and dose over time.
And longer term outcomes enable us to measure response rates, which are more robust in terms of their requirements, but typically that includes imaging for patients who have systemic disease as well as bone marrow assessments. So the end of the year will include response rate information because the study will be much more mature across a broad.
And number of our patients the earlier data will be.
The leading indicators that are certainly highly correlate with response rates. For example, IGN is one of the major components of response rate. So it's a nice correlation for the long term outcomes.
Okay, Great and then for one from Drome could you help us think about the enrollment speed and seems to be a little bit slower than first expected and and how to think of that in light of what that mean and mean for patient interest and.
And I ask because you've done a lot of work characterizing the size of the patient population out there and it seems like it's more than 3500 patients and in the U S alone and and there is no approved therapies like this so it seems like it and.
Perhaps.
And the need for interest and enrollment might be a little bit faster, then and what we've been seeing so can you help us kind of reconcile those two thoughts thanks.
Sure No. It's a great question and I wish enrollment, but I think most of us and the industry have wished enrollment with correlate with patient numbers. Unfortunately, that's not the case.
Patients and.
And site needs to be lined up the lead time for opening sites and a geographic region that supports patients and minimize if their travel requirements, especially during the COVID-19 pandemic with or with that before COVID-19 hit.
Hit us so really this is a story of matching patients to open sites supporting their travel and risk profiles for Covid, we identified patients well in advance of opening our sites to make sure we could optimize across the globe and terms of how we would logistically open. These trials so I don't have any.
Thing to offer other than to suggest that the connectivity is not there for the industry and unfortunately, it's not there for us as well. The good news is we've known our patients now for quite a period of time, they're ready, they're there and 2021 will be the year, we successfully enroll this trial and communicate that to the street.
Great. Thank you very much.
Thank you. Our next question comes from Leland <unk> with Oppenheimer. Your line is now open.
Hey, good morning.
And Ed and thanks for taking my question and just wanted to ask in terms of the open label update on the phase two and when would we also be targeting ehealth or.
Would it be later in the year just.
I wanted to give some color around the timing.
And also wanted to ask just in terms of the earlier pipeline and.
And just wanted to check in and see how any progress maybe going away from the earlier Ken that you have.
And the pipeline. Thank you.
Sure Diego would you like to comment on the open label extension and then art would you like to comment on the pipeline.
Go ahead Diego.
Yeah. Thank you Paula.
Yeah, we are.
Planning for later this still we should all do a face to win open label extension and.
But most likely later in the year or early next year.
On art would you like to comment on the pipeline.
Sure. Thank you Diego click and call.
Yes, the pipeline actually I'm pretty excited about where we're going with that we've been looking at some of those profiles and various molecules.
And very deep.
Target series, and we're looking for opportunities and so this year I think we're going to be able to push something towards into the IND, enabling studies with the hope of getting something into the clinic next year. So that's the goal.
Got some pretty good profiles were able to penetrate into the brain.
And that leaves us with opportunities for some green related diseases that are associated with six year four of regulation.
And then we have compounds.
It looks like the target various lymphocyte populations. So there are opportunities for Lymphocytopenia type diseases.
So more to come and all the score.
There's a lot more information as we go.
Excellent. Thank you for taking my questions congratulations.
Thank you. Our next question comes from RK with H C. Wainwright. Your line is now open.
Thank you good morning, Paul on that.
Couple of quick questions. The first one on world and storms.
I think since we Blue Tonight.
Doug.
Oh got approved as a combination therapy as well along with Rituxan and <unk> in 2018. The combination has been kind of moving more into the first line.
First line therapy.
But you are doing your studies just us in combination with Ibrutinib. So how do you see youll.
Nomination and working.
And within the therapeutic.
Total pudic paradigm for this disease.
Thanks, RK I appreciate the question.
I think there are some geographic differences. So so what we've been hearing is certainly a brute and nib.
And it's.
And there's still used single agent, sometimes on the frontline setting and certainly and our second line setting and the U S and has some shifts to do that as well as in Europe, we recognize the prudent and plus rituximab.
On a frontline paradigm.
And you look at the data and also we hear some input from our X U S. Clinicians I think most people experienced protects them lab offering very little to the combination.
And effectively are prioritizing ibrutinib and in the frontline setting so I think that could actually bode well for us. It is still early and you mentioned 2018, and it's really only about two years and do it and it does take some time for the paradigm to settle and but I think we're actually and very good shape with our initially partnering with Ibrutinib even.
And in the event that the paradigm shifts I think CX airport antagonism partners and more broadly with other types of mechanisms and agents and Walden from then and even more broadly lymphoma. So on either regard I think we're well positioned with its early dataset to just show the power of <unk> four antagonism and move forward from there.
Great. Thank you for that and then.
The second question is on SCM.
So just.
And just as you discussed a little bit more in terms of debt expectations.
From the.
And from from the other indication and the middle of the ear.
From modern storms and.
So here and you'll see and.
I know youre not.
Telling us specifically when in 2021.
But what sort of.
And what sort of B C and efficacy data on all of them.
Just.
B B some safety.
And on dosing and.
Dose escalation.
Sure. So let me just remind you of the strategic importance of MTN. So severe congenital neutropenia is.
Ideally what we hope.
Assuming women's approved.
Certainly like to explore the application of maverick's, four and other patient and flip these profound neutropenia isn't lymphopenia and SDN is a natural first place to explore.
Strategically the timing on the data still continues to fit well with the overall timing of our global efforts around Maverick before so as we as we think about the data this year.
And the S and trial has probably been the most complicated and duty.
Due to Covid because of the duration of the trial. It is a two week dosing period, which offers little opportunity to provide clinical benefit for patients and we respect that it is really a translational study that will correlate a patient's genetic profile with a response after two weeks of dosing. So at the end of the day we.
You need to be respectful of the patient dynamics, given the global and once in 100 ear and I make that we're all experiencing.
So we so we will have some initial data towards the end of the year and we'll provide more color on that as we gain better clarity.
Perfect. Thank you very much and good.
Good luck.
Thank you so much okay.
Thank you and our next question comes from my England Tani with B Riley Securities. Your line is now open.
Hi, Good morning theme is this all hotels from Miami.
Grasp on all the progress a couple of brief questions from us could you discuss a little bit about the AGM response kinetics and kind of how you see the levels or how you think about the levels potentially evolving over time from this.
The initial readout relative to kind of what we can.
To see at the end of the year and then briefly on web if you could just.
Give us a little bit of color on the enrollment dynamics in terms of U S versus ex U S and he considerations for the pandemic.
Alluded to and the SDN program.
And as it relates to maybe missed doses.
Sure. So I think the first question was around sort.
And sort of IGF level kinetics, and benchmark to help you appreciate and Britney and but that did I catch that correctly, yes exactly.
Okay.
So I'll begin and then I'll I'll ask Diego to also chime in but there are actually some very.
Good references and.
Britain. It has been well studied in the Walden from population Dr. Steve tree on its considered one of the global experts in this area and thankfully. He is one of the key.
Investigators on our trial.
Dr. Tran translational work has shown that patients with the X here for and mutations have IGN drops that are dramatically different than those patients without <unk> and just a few references for those folks on the phone and Theres a great publication by Dr tree on and blood of 2017 that actually split that the kinetic.
And of the AGM vs time for the two different types of population.
So it'll be a great benchmark for us because we understand what patients do on Ibrutinib with the C X, therefore mutation and without and ideally our treatment with Maverick sport could correct for that and change these patients from doubled maintenance to look more like the kinetics of single mirror and so and that's some of the.
Efforts that were going on now to measure and benchmark.
So I hope that was helpful. I think the second question was a little bit around the pandemic impact on our women trial. So I do think there are differences there certainly the whim trial provides the potential for clinical benefit if we reflect on our phase II data. That's been published it has certainly strong suggestion.
The reductions and infection rate and some of the quality of having whim syndrome. So there is reason to be motivated and committed to this trial, which we've certainly seen our brave patients step forward with and because we certainly had these broad relationships with kols over the years and their patients when you feel like that relationship.
Is certainly one of the key things that we continue to respect and appreciate and support our hitting our enrollment targets, which are nicely on track for this year.
That's very helpful. Thanks for taking my questions.
Absolutely.
Thank you. Our next question comes from debt the Java with Roth Capital Partners. Your line is now open.
Good morning, guys just wanted to quickly confirm Paula let's work on and see any kind of highlights on them.
Walton from data before a presentation and a medical meeting and then also a weighted.
Any update on a GAAP progress operating a partnership with the best deals this year.
So why don't I invite Diego and to comments about our plans around the Walden from day to given his connection with our investigators and then I'll take the other question.
Yes, Thank you Paula.
So we are looking to do any Michelle and Paul.
Disclosure of their wallet and strong phase <unk> data.
Towards the end of each one will slightly at <unk>.
And we'll report on initial safety PK and PD, we have a very good pharmacodynamic marker.
And some initial data on the seed on my Jam on Haemoglobin, which are really helpful and biomarkers.
And later in the year, most likely at Ash, we plan to have a second presentation on a.
Shooting to more patients and longer follow up and tooling on assessment of responses and cooling and bone marrow and whole body piece on both of these presentations and we.
We'll be able to be compared with the benchmarks from Ibrutinib monotherapy in double mutant population. So we believe that will help us.
Our sales day emerging efficacy on mobile except 40 and these these type of lymphoma.
Which has obviously a high unmet need is Seattle day.
Really the sickest and.
And use that information to decide on next steps.
Regarding the partnership with obese Coppola could you take that one.
Thank you Diego yes.
The fiscal partnership is advancing.
I would bet that we'll be able to provide more clear update towards the end of 2021, but it's launching nicely and as we gain clarity, we'll look forward to sharing more details at a future point.
Got it Paul and thanks for the update.
Thank you.
Thank you I'm not showing any further questions at this time I would now like to turn the call back over to Paula Ragan for closing remarks.
Thank you so much for joining everyone. This morning, I Hope you all continue to remain safe and healthy and if you have any follow up questions, we'd be happy to answer those offline. Thank you again and I hope you enjoy the rest of your day.
Goodbye.
Ladies and gentlemen, this concludes today's conference call. Thank you for participating you may now disconnect.
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