Q2 2020 X4 Pharmaceuticals Inc Earnings Call
Greetings and welcome to X four pharmaceuticals second quarter financial and operating results conference call.
Operator: Greetings and welcome to X4 Pharmaceuticals' second quarter financial and operating results conference call. At this time, all participants are in a listen-only mode.
The time all participants are in the listen only mode. A question and answer session will follow the formal presentation.
Operator: The 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, Candace Ellis, Director of Corporate Communications and Investor Relations at X4. You may begin. Thank you, Operator. And good morning, everyone. Thanks so much for joining us.
As a reminder, this conference call is being recorded it is now my pleasure to introduce your host Candace Alice Director of corporate Communications and Investor Relations at X for you may begin.
Thank you operator, and good morning, everyone. Thank so much for joining us.
Candace Ellis: Presenting on today's call will be our Chief Executive Officer, Dr. Paula Ragan, and our Chief Financial Officer, Adam Mostafa. Following prepared remarks by each, we will open the call to your questions, and they will be joined by our Chief Scientific Officer, Renato Scarro, and our Senior Vice President of Technical Operations and Quality, Mary DiBiase. As a reminder, on today's call, we will be making forward-looking statements regarding our Regulatory and Product Development Plans, as well as our research activities. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted. A description of these risks can be found in our most recent Form 10-K, on file with the FCC, and in our forthcoming Form 10-Q. I'd now like to turn the call over to our CEO, Paula Ragan.
Presenting on today's call will be our Chief Executive Officer, Dr. collar Reagan and our Chief Financial Officer Adams Dasa following prepared remarks like each well open the call to your question and they will be joined by our Chief Scientific officer, not escrow in our senior Vice President of technical operations in quality Harry Dibiase.
As a reminder, on today's call will be making forward looking statements regarding our regulatory in product development plan as well as our research activities. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.
A description of these risks can be found in our most recent form 10-K on file with the S. C C.
And in our forthcoming form 10-Q.
I'd now like to turn the call over two or feel Pollard again.
Paula Ragan: Thanks, Candice, and thank you, everyone, for joining us on the call this morning. We hope you are all continuing to stay safe and healthy. We are pleased to report today that, despite the challenges posed by the ongoing COVID-19 pandemic, we were able to achieve important progress during the second quarter. We benefited from what we believe to be two value-adding and de-risking events related to the development of our lead candidate, Maverick Sephora, for the treatment of Wim Syndrome, a rare inherited primary immunodeficiency disease caused by mutations in the As a reminder, Maverix IV is our first-in-class, small-molecule antagonist of the CXCR4 receptor and the first potential disease-modifying treatment for WIM patients. We are currently conducting a pivotal phase 3 trial in patients with Lyme syndrome. The first of these important events was our Virtual Analyst Day that we hosted in early April.
Thanks, Candice and thank you everyone for joining us on the call. This morning, we hope you are all continuing to stay safe and healthy.
We're pleased to report today that despite the challenges posed by the ongoing Kobin 19 pandemic, we were able to in cheat important progress during the second quarter.
We benefited from what we believed to be to value, adding and de risking events related to the development of our lead candidate Mavericks before for the treatment of Wimps syndrome, a rare inherited primary immunodeficiency disease.
By any patients and the chemo kind receptor CX here for a receptor that plays a key role and enabling the healthy trafficking of immune cells and effective immuno surveillance.
As a reminder, Mavericks for is our first in class small molecule antagonist of the CX year for receptor.
And the first potential disease modifying treatment for when patients.
We are currently conducting a pivotal phase three trial in patients with him syndrome.
The first of these important events with our virtual analyst day that we hosted in early April.
Paula Ragan: At this event, we presented an overview of Wim Syndrome and increased our estimate related to the prevalence of this disease in the U.S. based on in-depth primary market research that we conducted in 2019 and earlier this year. As we discussed at that time, our updated research now indicates the range of diagnosed and undiagnosed Lyme patients in the United States to be greater than 3,500, a significant increase from the prior estimates of approximately 1,000 genetically diagnosed patients. Our research also indicated a concentrated target physician population primarily consisting of immunologists, hematologists, and infectious disease specialists that we believe we could cover with a small targeted sales force. This positive market snapshot was followed by a second key event related to the MavRxiv4 WHIM indication, a presentation of additional Phase II data at the European Hematology Association's (EHA) Annual Congress in June.
At this event, we presented an overview of women syndrome and increase our estimate related to the prevalence of this disease in the U.S. based on an in depth primary market research that we conducted in 2019 and earlier this year.
As we discussed at that time, our updated research now indicates the range of diagnosed and undiagnosed when patients in the United States to be greater than 3500.
A significant increase from the prior estimate an approximate a thousand genetically diagnosed patients.
Our research also indicated a concentrated target physician population, primarily consisting of immunologist hematologist and infectious disease specialists that we believe we could cover with a small targeted sales force.
This positive market snapshot was followed by a second key events related to the Mavericks and for women indication our presentation of additional phase two data at the European Hematology Association or E. AJ annual Congress in June.
Paula Ragan: I won't go into many specifics here, as we reviewed the data in a detailed call on our June EHA call, but I do want to highlight several key takeaways, as they serve to strengthen our confidence in Mavrixifor's potential to be successful in our ongoing phase 3 trial and deliver a disease-modifying therapy in patients with Wim Syndrome. The data presented were from the open-label extension of our Phase 2 clinical trial evaluating the long-term safety and efficacy of Maverix for inpatients with Wim Syndrome. Most importantly, the data support the Phase III selection of the dose of 400 mg once daily, which demonstrated a greater than 4.5-fold increase in the metric of time above threshold for absolute neutrophil counts, or TATANC, as we abbreviate it, over the low doses explored in the study. The metric TAT-ANC is the primary endpoint defined by the U.S. and European regulators.
We'll go into many specifics here as we review the data in a detailed call on our June E AJ call, but I do want to highlight several key takeaways as they serve to strengthen our confidence and Mavericks, a fourth potential to be successful and our ongoing phase three trial and liver disease modifying therapy in patients with women syndrome.
The data presented at were from the open label extension of our phase two clinical trial evaluating the long term safety and efficacy of Mavericks for in patients with when syndrome.
Most importantly, the data support the phase three selection of the dose of 400 milligrams once daily which demonstrated a greater than 4.5 fold increase in a metric of time above threshold for absolute neutrophil counts for T.H.T., a and C. As we abbreviated.
Over the low doses explored and the study.
The metric T.A.T. and see is the phase three studies defined primary endpoint agreed upon by the U.S. and European regulators.
Paula Ragan: Additionally, the Phase 2 data showed sustained dose-dependent increases in white blood cells, absolute neutrophil counts, and absolute lymphocyte counts at the medium follow-up time of 16.5 months. Also important to note, these hematologic improvements correlated with a direct clinical benefit, with patients treated for at least 6 months experiencing a 54% decrease in annualized infection rates versus the infection rates in the 12 months prior to treatment. Additionally, patients experienced a 75% reduction in cutaneous warts versus baseline, suggesting a favorable impact on the HPV-related disease risk of Lyme syndrome.
Additionally, the phase two data showed sustained dose dependent increases and white blood cells absolute neutrophil counts and absolute lymphocyte count at the median follow up time of 16.5 months.
Also important to note these hematologic improvements correlated with a direct clinical benefit.
With patients treated for at least six month experiencing a 54% decrease in annualized infection rate versus the infection rates and the 12 months prior to treatment.
Additionally, patients experience, a 75% reduction and cutaneous warts frissons baseline, suggesting a favorable impact on the each PV related disease risk of limb syndrome.
Paula Ragan: Rate of Infection and Warp Burden are two important secondary endpoints of our Phase 3 trial. Enrollment in our Phase 3 trial is ongoing and making progress as certain countries and sites have reopened in the context of COVID. We remain on track to report top-line Phase 3 results in WIM in 2022. Our second ongoing Maverick-SIFOR clinical program is a Phase 1b trial in patients with severe congenital neutropenia, or SCN. It's a 14-day proof-of-concept trial designed to assess the safety and tolerability of daily oral Maverick-SIFOR in up to 45 patients with SCN and other selected congenital neutropenia disorders. We continue to expect initial data in 2021. We intend to provide further clarity around these timelines for both WIM and SDN at a future point.
Right and I've infection and weren't burden, our two important secondary endpoints of our phase three trial.
Enrollment in our phase three trial is ongoing and making progress a certain countries and sites have reopened in the context of Cove. It we remain on track to report topline phase three results in win in Twentytwenty too.
Our second ongoing Mavericks for clinical program is a phase one be trial in patients with severe congenital neutropenia or SDN <unk>.
If they 14 day proof of concept trial designed to assess the safety and Tolerability of daily oral Maverick before and up to 45 patients with Essien and other selected congenital neutropenia disorders, and we continue to expect initial data in 2021.
We intend to provide further clarity around these timelines for both win and SDN had a feature points.
While our public facing presentations during the first half. The 2020 were focused primarily on when syndrome. We've also continued to make progress in our ongoing phase one be study a maverick before in patients with Waldenstrms Macroglobulinemia, a rare form of non Hodgkin's lymphoma.
Paula Ragan: While our public-facing presentations during the first half of 2020 were focused primarily on Wimp syndrome, we've also continued to make progress in our ongoing Phase 1B study of maverick before and patients with Waldenstrom's macroglobulinemia, a rare form of non-Hodgkin's lymphoma. This trial is expected to enroll between 12 and 18 patients with Waldenstrom's and is a multi-center, open-label, dose-escalation trial assessing the safety and tolerability of Mavrix X4 in combination with the standard of care treatment, Abrutinib, a tyrosine kinase inhibitor. The trial is being conducted as part of a collaboration with the Leukemia and Lymphoma Society to accelerate the development of Maverixifor for the treatment of Waldenström's. Enrollment is continuing in this trial as well, and we continue to anticipate announcing initial Phase 1b clinical results towards the end of this year.
This trial is expected to enroll between 12 and 18 patients with Waldenstrom and as a multicenter open label dose escalation trial assessing the safety and Tolerability of Maverick before in combination with a standard of care treatment Ibrutinib I tyrosine kinase inhibitor.
The trial is being conducted as part of a collaboration with the leukemia and lymphoma society to accelerate the development of Mavericks before for the treatment of Waldenstrom.
Enrollment is continuing in this trial as well and we continue to anticipate announcing initial phase one be clinical results towards the end of this year.
Paula Ragan: These results will contain both safety data and important efficacy signals. We expect these data to inform future FDA discussions we plan to hold regarding a registration trial in Waldenstrom. Lastly, we wanted to acknowledge here the promotion of Dr. Renato Scurll to the position of Chief Scientific Officer of X4. Renato was one of the scientific founders of X4 and a co-inventor of Maverix X4 and has more than 25 years of experience leading the discovery and development of disease-modifying small molecule drugs to treat genetically defined rare diseases. In this expanded role, he is leading all research and non-clinical development functions at the company, overseeing our operations in Vienna, Austria, as well as our efforts to advance and expand our pipeline targeting additional genetic rare disease indications. I'm now going to turn it over to Adam to discuss our financial results for the quarter. Adam?
These results will contain both safety data and important efficacy signals.
We expect these data to inform future ft aid discussions we plan to whole regarding a registration trial and waldenstrom.
Lastly, we wanted to acknowledge here the promotion of Dr. anatomy girls the position of Chief Scientific Officer of X four.
We're not show was one of the scientific founders of X four and a Colin inventor of Mavericks before it has more than 25 years of experience, leading the discovery and development of disease modifying small molecule drugs to treat genetically defined rare diseases.
And this expanded role he is leading all research and non clinical development functions that the company overseeing our operations in Vienna, Austria.
Well as our efforts to advance and expand our pipeline targeting additional genetic rare disease indications.
I'm now going to turn it over to add them to discuss our financial results for the quarter Adam.
Adam S. Mostafa: Adam, thank you Paula, and thanks to all of you on the call today. As presented in our press release this morning, I will summarize our financial activities and results for the second quarter of 2020. As of June 30, 2020, X4 had $105.6 million in cash. I do expect that our cash and cash equivalents will fund our operations into early 2022.
Thank you Paula and thanks to all of you on the call today.
As presented in our press release. This morning, I will summarize our financial activities and results for the second quarter 2020.
As of June Thirtyth, 2020, Excor had $105.6 million in cash.
Do you expect that our cash and cash equivalents will fund our operations into early 2022.
Note that this guidance does not include the $25 million potential proceeds from our amended Hercules debt facility or any cash exercise proceeds from investors holding our outstanding warrants.
Operator: Proceeds from our amended Hercules Debt Facility or any cash exercise proceeds from investors holding our outstanding Warrants. Research and development expenses were $9.3 million for the second quarter of 2020, as compared to $8.9 million for the comparable period in 2019. General and administrative expenses were $5.3 million for the second quarter of 2020, as compared to $4.6 million for the comparable period in 2019, and our net loss was $15.1 million for the second quarter of 2020 compared to a net loss of $13.4 million for the comparable period in 2019.
Research and development expenses were $9.3 million for the second quarter of 2020.
As compared to $8.9 million for the comparable period in 2019.
General and administrative expenses were $5.3 million for the second quarter 2020.
As compared to $4.6 million than a comparable period and 29 team.
And our net loss was $15.1 million for the second quarter of 2020 compared to a net loss of $13.4 million for the comparable period between 19.
With that.
Operator: With that, why don't we open it up to questions? Operator? Thank you, sir. As a reminder, to ask a question, you would need to press star 1 on your telephone. To withdraw your question, press the pound key. Please stand by while we compile the Q&A list. I show our first question comes from the line of Stephen Wiley from Stiefel. Please go ahead. Hi, this is Ellen Sands on for Steve. Thanks for taking the question. So my first question is, is there any scenario whereby you decided to escalate the dose beyond 600 milligrams in the Waldenstrom's trial?
Why don't we opened it up to questions.
Right.
Thank you Sir.
As a reminder to ask a question you would need to press star one on your telephone to withdraw your question personal key.
Please standby, while we've compiled acuity roster.
Oh sure first question comes from the line of Steven Why Lee from Stifel. Please go ahead.
Hi, This is Alan sands on for Steve.
Thanks for taking my question. So my first question is is there any scenario whereby you decided dose escalate beyond 600 milligrams gnomes trends trial.
Paula Ragan: Hi. Thanks, Ellen. This is Paula.
Hi, Thanks, Alan the so Paulo just to answer your question I think that's a insightful I think at this point, we think Thats, a 600 milligram exposure or certainly gets us well above the IC 90, a at the trough level, but we can consider doing that given the safety profile. The drugs. So I think there'll be some flux.
Paula Ragan: Just to answer your question, I think that's insightful. I think at this point, we think that the 600 milligram exposure certainly gets us well above the IC90 at the trough level, but we can consider doing that given the safety profile of the drug. So I think there'll be some flexibility there in terms of safety, but likely not given what we're already dosing the patients with.
Ability there in terms of safety, but likely not given what we're already dosing patients with.
Okay makes sense and then.
Paula Ragan: Okay, makes sense. And then, maybe just two more for me. So, can you remind us if you've predefined a given number of representative genotypes that you want to specifically enroll into the Phase 1b for SCN, or is it all-comers? I know you're looking at a genetic panel for the patients.
Maybe just two more for me. So can you remind us if he pre defined given number of representative genotype that you want to specifically enrolling the phase one dvrs yen or is it all comers I know you're looking at genetic panel for patients.
There's there's sort of two two buckets there patients that don't have an existing genetic profile, but do qualify from the inclusion price criteria. So we'll be screening, though to better understand their genetic profile.
Paula Ragan: There are sort of two buckets. There are patients that don't have an existing genetic profile but do qualify from the inclusion criteria, so we'll be screening those to better understand their genetic profile. There will be some that are already defined with certain genetic phenotypes or genetic profiles, such as GATA2 or G6PC3, but we will still actually genetically panel those as well in case there's additional genetic alterations that we need to understand, so I think there's just flexibility there. Everyone will get genotypes, but some have a bit of a better understanding entering the study. Okay, great. And then last one, are there any updates on potential collaborative partners for Mavericks IV and solid tumors? Adam, do you want to take that?
There will be some that are already defined with certain genetic phenotype or genetic profile such as gotta to emerging <unk> P. C. But we will still actually genetically panel those as well in case, there's additional genetic alterations that we need to understand so I think there's flexibility there everyone will get genotypes, but some have a bit about a better understand.
Entering the study.
Okay, Great and then last one are there any updates around central collaborative partners for remember export in solid tumors.
Adam you want to take that.
Sure.
Adam S. Mostafa: Sure. It's a good question.
Good question.
Adam S. Mostafa: So we have our partnership with Abisko, which is progressing nicely. They recently announced a crowdfunding campaign, and that's for Chinese rights outside of China.
So we have a partnership with the Bisco, which is progressing nicely. They recently announced a fund raising and that's for China rights.
Outside of China.
Adam S. Mostafa: We don't currently have any updates on that strategic front. We continue to focus on rare oncology with our Waldenstrom's trial, with data expected later this year. Okay, great. Thank you for taking the questions. Thank you. Our next question comes from Olinda Lee from Canaccord. Please go ahead. Good morning. It's Ben Shim for Arlinda. Thanks for taking my questions. Paula, just a couple general ones. Can you give us an idea of what the scope of the Phase 1B Walden Institute data will be? What will be provided? Maybe we can nail down a little bit more specific timing for that.
Currently have any updates on the strategic front.
Continue to focus on where oncology.
Their wounds Troms trial with data expected later this year.
Okay, great. Thank you for taking the questions.
Thank you.
Next question comes from all Linda Lee from Canaccord. Please go ahead.
Good morning extension for Arlinda, Thanks for taking my questions.
Paul just a couple gelatin one so can you give us an idea of what the scope of the fees wouldn't be waldenstrms data will be provided.
Maybe kneel down little bit more specific timing for that.
Sure. Thanks, Ben So the fee is one D is a dose escalating trial and think about it almost in two parts every patient will initiate at 200 milligrams and assuming safe a safety supports that they'll be dose escalated to the next level. So 400, and then 600 after 20.
Paula Ragan: Sure. So, CS1B is a dose-escalating trial, and think about it in almost two parts. Every patient will start at 200 milligrams, and assuming safe safety supports that, they'll be dose-escalated to the next level, so 400, and then 600 after 28 days of safety. And that's, again, 12 to 18 patients, so that's kind of Part 1. Part 2 will be for any given patient reaching their maximum tolerated dose. They'll continue on in the study for at least 3 more months, and then roll into an open-label extension. So, the way we think about the data is in those two parts. The first part enables us to better understand safety PK PD profiles, and additionally, we'll be looking at IgM-level drops.
He is a safety.
And that's again 12 18 patients. So that's kind of the part one part two will be for any given patient reaching their maximum tolerated dose continue on in the study for at least three three more months and then roll into an open label extension. So the way we think about the data are in those two part the first part enables us to.
A better understand safety PK PD profile and Additionally, we'll be looking at a G.M. level drops.
Paula Ragan: I.
Paula Ragan: IGM-level changes certainly have been shown to occur in the first two to three months of dosing with Ibrutinib in the single mutant population. That's much more difficult in the double mutant population, so we're hopeful that seeing meaningful drops in the IGM would be indicative of clinical activity of Maverix 4 in that double mutant patient population.
I'd n. level changes certainly have been shown to occur in the first two to three months of dosing with Ibrutinib and these single me in population that's much more difficult in the double mutant population. So we're hopeful that seeing meaningful drops Nigerian would be indicative of clinical activity of Mavericks pour in that.
I mean in patient population.
Okay great.
Paula Ragan: Okay, great. Just a second, kind of general question.
Just a second quarter general question.
As we get closer to getting hopefully qubic 19 vaccine as early as this fourth quarter.
Paula Ragan: As we get closer to getting hopefully a COVID-19 vaccine as early as this fourth quarter, my question to you is, how directly relevant do you think this vaccine will be to your patient populations in each of the three indications you're studying, directly or indirectly? Is this recalibrating maybe your expectations for enrollment and potential data readout?
Yes. My question to you as home Directory relevant do you think this vaccine will be to your patient populations and each of the threeq.
Indications, you're studying it directly or indirectly again [laughter] is this recalibrating, maybe your expectations for enrollment and.
Central data read outs. Thanks.
So it's a great question I think anything related to cope with 19 data in a when patients is a challenging certainly we were not.
Paula Ragan: So it's a great question. I think anything related to COVID-19 data and when patients are challenging, because certainly we are not.
Paula Ragan: I don't think we're aware of any data yet that kind of elucidates what their risk profile is. I actually have been following COVID in the broader sense of immunocompromised patients, and they do have some, you know. The most recent Nature publication suggests they do have some modest risk, of course, but not to the extremes that we've seen in various key populations. So, again, with that backdrop, I think, you know, what we can control is just keeping focused on the trial and making sure our patients are safe. We have an independent safety monitoring board that, of course, assesses their infections. So, I think in the context, and then to get back to the context of vaccines, every vaccine is different, and different patient populations respond differently. So, we would obviously need to better study that, and I think it's just too much uncertainty and too many unknowns at this point to really conjecture.
Aware of any data yet that kind of elucidates what their risk profile is actually had been falling code in a broader sense of immunocompromised patients and they do have some you know that the needs. Most recent needs. Your publications suggest they do have some modest risk of course, but but not the extremes that we've seen in various keep popular.
Jason.
So again with with that backdrop I think you know as what we can control just keeping focused on the trial and making sure. Our patients are seats, we have a D. A an independent safety monitoring board that of course assesses their infection. So.
I think under contract and then to get just get back the context of vaccine.
Every vaccine is different in and different patient populations respond differently. So we would obviously need to better study that I think it's just a.
<unk> too much uncertainty in too much and knows at this point really conjecture.
Paula Ragan: Thank you very much, Paula. Good luck to you.
Okay. Thank you very much Paul good luck to you.
Operator: Thanks, Ben; take care.
Yeah, Thanks, Ben take care.
Thank you.
Operator: Thank you. Our next question comes from Leland Gershel from Oppenheimer. Please go ahead. Hey, good morning guys.
Next question comes from Leland Gershell from Oppenheimer. Please go ahead.
Hey, good morning, guys. Thank you for the update my question I actually wanted to ask on me earlier.
Operator: Thank you for the update. Thanks for the question. I actually wanted to ask about the earlier pipeline behind MAV on 0203, just wanted to check in if we'll be hearing any news flow on those compounds, the programs for those, later this year or perhaps in 21. Thanks.
Type wind behind.
So to over three just wanted to check and it will be hearing a news flow and on those.
Programs for those.
Later, this year or perhaps 21 thanks.
Renato Scarro: Sure, Renato, do you want to take that?
Sure and I know you want to take that as soon thanks I'll take that question. So.
Renato Scarro: Yes, yeah, thanks. I'll take that question.
Renato Scarro: So we're scheduled to basically move one of those compounds into development later in the year. We're currently just looking at some properties of these molecules to make a decision in terms of which molecule we would move forward with. So we're on track to basically move one of the molecules into development and possibly another molecule next year. Right, I think it was GBM 402. Correct. So that's the molecule that crosses the blood-brain barrier. I show our next question comes from the line of Joel Beattie from Citi, please go ahead. Hi, thanks for taking the questions. For the Waldenstrom's data coming out later this year, could you describe what would be next for the program if the data is successful?
Yeah were scheduled to basically moved one of those compounds into.
Because I don't want later than you would currently just looking that some properties of these molecules to make a decision in terms of which molecule we wouldn't be forward. So we're on track the but as we move one of them all to something about potentially another molecule next year.
Indications.
Thank you is GBM.
Yeah.
Correct.
So this is my which are the crosses the blood brain barrier.
Thanks.
Thank you.
I show next question comes from the line of Joel Beatty from Citi. Please go ahead.
Hi, thank for taking the questions for the Waldenstrom stuff coming out later this year could you describe what would be next for the program. If the data is successful.
Sure. Thanks, Joel, Yes, or you know we no. So much about her molecule already and then certainly seeing signs of clinical activity in the face on be would support directly moving into what we would be with a phase two three trial that would likely be a controlled trial I'm you know combination.
Paula Ragan: Sure, thanks, Joel. Yes, we're, you know, we know so much about our molecule already, and certainly, seeing signs of clinical activity in Phase 1b would support directly moving into what we would view as a Phase 2-3 trial. That would likely be a controlled trial, you know, combination versus likely standard of care or single treatment with brutinib, but we still need to have conversations with the FDA on that. But we're in great shape, I think, in terms of knowing so much about our drug from other trials that we would feel quite comfortable, you know, seeing strong signals to move forward with a registration trial subsequent to Phase 1b.
One versus likely standard of care or single single treatment with Ibrutinib, but we still need to have conversations with the FDIC on that.
But we're in great shape I think in terms of we know so much about our drug from other trials that we would feel quite comfortable or you know seeing strong signals coming forward with a registration trial subsequent to the phase one be.
Paula Ragan: Okay, that's helpful. And then, would that Phase 2, Phase 2-3 trial be in the current Cash Fundway guidance?
Through the health woman with the phase Twob I'm pleased to three trial be and the current pet friendly again.
No it would not I'll, let Adam expand on that.
Adam S. Mostafa: No, it would not. I'll let Adam expand on that.
Would it be into it and Joe just to clarify and the current runway guidance without the question.
Adam S. Mostafa: Would it be in the, Joe, just to clarify, the current runway guidance, without the question? Yeah, the cash runway to early 2022. Yeah, no, it's not currently contemplated. In our current runway guidance to early 2022 and the nature of that trial, the cost of that trial, et cetera, would be informed in part by the data that we'll plan to put out later this year and further discussions with the FDA.
Yeah look of the care friendly way too early equally too.
Yeah, I know it's not.
Currently contemplated.
In our current runway guidance to early 20 to 2022.
Right and the nature that trial the cost of that trial scenario would be informed in part by the data that will we'll plan to put out later this year.
Further discussions with the <unk>.
It sounds great. Thank you.
Adam S. Mostafa: It sounds great. Thank you. Thank you. Our next question comes from the line. Swayampakula Ramakanth from HC Wainwright, please go ahead. Thank you. Good morning, Paula and Adam.
Thank you.
Our next question comes from the line of small I am punctually Ramakanth from H.C. Wainwright. Please go ahead.
Thank you good morning pull on a them most of my questions are being.
Paula Ragan: Most of my questions have been answered. However, just want to check on a couple of things. Any update you can provide on enrollment stats in your clinical studies, given the current environment? And the second question is about the collaboration with NVIDIA for generating the genetic test, the diagnostic test that you want to generate.
Oh, we're just wanted to go on couple of things and they have good you can provide on on and enrollments that and your clinical studies given given the current environment.
The second question is.
On the collaboration within me to or generating the agenda to just Oh, the diagnostic does or do you want goods in the.
Yes.
Paula Ragan: Sure. Thanks, R.K.
Sure.
Paula Ragan: So, the enrollment status, I think we're maintaining our current guidance. Maybe just to provide a little bit more nuance, I am encouraged that the patients are still there. We've appreciated this through surveys, through patient advocacy groups. Our KOLs are very much engaged.
Thanks, Okay. So the b enrollment status I think I'm, we're maintaining our current guidance.
Maybe just to provide a little bit more nuance.
I am encouraged that the patients are still there weve appreciated this through surveys to patient advocacy groups are kale all they're very much engaged I think that continued challenge keeps relying on you know different countries and different hospitals, even have a local requirements that are.
Paula Ragan: I think the continued challenge continues to be that different countries and different hospitals even have local requirements that are, you know, of varying degrees of restriction in terms of COVID. So, we're working very closely with them. We feel the engagement is strong and continues, but we just need to continue to wait out how these various sites and countries kind of return to a quasi-normal profile. And then, in terms of the NVPAY collaboration, that's going well. But we don't yet have specific guidance on providing sort of a relevant output from that, as we're still building the broader funnel to capture as many patients as possible. Obviously, a larger patient pool helps us generate confidence in sort of the overall profile of the data, so stay tuned for next year on that one. So, thank you.
You know a varying degree the restriction in terms of Cove. It. So we're working very closely with them. We feel be engagement as is strong and continues but we just need to continue to wait out how these various sites and countries.
Kinda returned back to acquire a normal profile for patients.
And then in terms of the envy today collaboration that's going well.
We don't yet have specific guidance on providing sort of a relevant output from that as we're still building the broader fund all the capture as many patients as possible, obviously, a larger patient pool helps generate confidence in sort of the overall profile of the data. So stay tuned for next year on that one.
Thank you. Thank you book.
Adam S. Mostafa: Thank you, thank you both. Thank you. Our next question comes from the line of Laura Christensen from Cowen.
Thank you.
Next question comes from the line of Laura Christiansen from Cowen. Please go ahead.
Operator: Please go ahead. Thanks. Good morning. Thanks for taking my question. Just following up on the earlier question about the Waldenstrom's trial and expectations for the readout, I just was wondering if you could provide any more clarity about maybe the number of patients that will be at the 600 milligram dose and how robust the IgM reduction data might be, just given that we don't know exactly when the patients were enrolled and cut off for when you'll be presenting the data.
Thanks. Good morning, Thanks for taking my question just following up on the earlier, one about the Walton trends trial and expectations for the read out I. Just was wondering if you can provide any more clarity about maybe the number of patients that we'll be at the 600 milligram dose and you know.
How much how robust the I'd young production data might be just given that we don't know exactly when the patients enrolled and a cut off or.
When you'll be presenting the data.
Paula Ragan: Yeah, I mean, I don't think we can give anything more clear. Laura, we're enrolling.
Yeah, I mean, I don't think we can give anything.
More clear LER were enrolling.
Paula Ragan: It's very difficult to forecast in a Phase 1b trial how it all comes together. And Brutonib has its own safety profile, and we have not, you know, tested, you know, completed this testing in combination with Brutonib, so just you'll have to wait and see when the data comes. I think, you know, we are excited that there is a lot of room for improvement in terms of the IgM-level drop in the single mutant population. You know, their drop is maybe 50%, 60% after three months from their baseline, so I think if we can, and then the double mutants actually fare much worse than that, so I think there's opportunity to see changes. We just need to complete the study and share that with everyone when we have that data set.
It's very difficult to forecast and if he is one be how it all comes together Ibrutinib has its own safety profile and we've not you know tested you know completed the testing and commissioned to Britain and so just walked a wait and see when the data comes.
I think you know we are excited that those a lot of room for improvement in terms of IDN level drop and the single me and population you know there drop is maybe you know 50, 60% after three months from their baseline. So I think if we can and then that the double me is actually very much worse.
On that so I think there's opportunity to see changes, we just need to complete the study and share that with everyone. Let me when we have that dataset.
Paula Ragan: Okay. Thanks. Yes, thank you. Thank you. Our next question comes from Zegva Jalaf from Rock Capital Partners. Please go ahead. Good morning.
Understood Okay. Thanks.
Yes. Thank you.
Thank you I'm next question comes from Zach of a job loss from Roth Capital Partners. Please go ahead.
Good morning, I feel like on the questions have been asking a and B update was in line with ACA Peyton No. Just a quick question your with gliding thoughts on partnership now that you've added some when data you're gonna have waldenstrom coming up soon I know there rare diseases, but in aggregate at its a large opportunities I was just thinking.
Adam S. Mostafa: I feel like a lot of the questions have been asked, and I think the update was in line with occupation. So, just a quick question here regarding thoughts on partnership. Now that you've added some WIM data, you're going to have Waldenstrom coming up soon.
Adam S. Mostafa: I know they're rare diseases, but in aggregate, it's a large opportunity. So, I was just wondering, in terms of partnerships, what were your thoughts there? And then, you did say that the efforts from EBT haven't fully materialized. Yeah, but I was just wondering if you had any learnings that you could communicate as you're, you know, going after these patient awareness efforts and things like that.
Tim to partnerships at what we I thought there and then you did state that the atherectomy BT hasn't fully materialize, yet but was just wondering if he had any brining that you could communicate as you you know I'm going after these patient awareness African perform.
Sure Adam you want to take the first one.
Adam S. Mostafa: Sure, Adam, you want to take the first one?
Adam S. Mostafa: Sure. Yeah, so, our strategic focus is to really own and commercialize in the rare disease markets, and that's the trajectory and path we're currently on. Like any company, we'll be optimistic and consider situations as they arise, whether that be geographic or otherwise.
Sure.
Yes so.
Strategic focus is to.
Owning commercialize you know the rare disease markets and that's the trajectory and perhaps a we're currently on like any company will be opportunistic and consider situations as they arise.
Whether that be geographic or otherwise.
And then I'll I'll take a quick comment on envy taste question, then aspirin outage join in but I mean, I think the exciting thing about a lot of these genetic a assessments are really doing a more thorough understanding of the full.
Paula Ragan: And then I'll make a quick comment on EnvyTaste's question and ask Renato to join in. But, I mean, I think the exciting thing about a lot of these genetic assessments is really getting a more thorough understanding of the full mutation frequencies. You may be aware that in certain genes, there are so-called variations of unknown significance, and the question is whether those are pathogenic or not, so that actually is the subject of Renato's team's focus, and, you know, maybe Renato, you can provide some commentary on why that's actually exciting for us.
Mutation frequencies he may be aware that in certain genes, there's they're called variations on the unknown significance. In the question is are those pathogenic or not so that actually is a subject of of or not a team focused and you know maybe we're not or you can provide some commentary on and why that's actually exciting for us.
Sure. So yes, one of the things that we're doing from a research respective them just to remind everyone researches Dr. Lynn you don't Austria.
Renato Scarro: Sure. So, you know, one of the things that we're doing from a research perspective, and just to remind everyone, our research is conducted in Vienna, Austria, we're looking to basically characterize all the known pathogenic variants, of which there are about a dozen of those, as well as the variants of unknown significance that Paula just mentioned, and we're looking to characterize them in terms of their functional ability regarding signaling, specifically internalization, And then we're also looking more deeply into some of the secondary roles in terms of B-cell phenotyping and some of the other signaling through the ERK and the KT pathway. This is, you know, obviously exciting in the sense that no one's actually done a comprehensive analysis of all these different variants, and ultimately, what we're looking to do is connect them to the phenotype that's presented clinically.
Looking to basically Turkey was all the known as of June adherence, which here about a dozen of those.
As was the variances on phones secretly considerable were just mentioned and we're looking to characterize it becomes the functional capability [noise] regarding signaling.
Typically internalization constant flux et cetera, and then we're also looking more deeply into some of the second degree levels in terms of B cell phenotype in and some of the other certainly through the.
Katy.
This is obviously exciting in the sense it no one's actually done the comprehensive analysis will be different variants and ultimately what we're looking to do it was connect them to the phenotype that's presented clinically.
Yeah, I think I'm just to bring it all back around there, they're basically might be more kind of undiscovered when patients because nobody because there yet have not been an understanding of the full paradigm of all the mutations that do caused disease. So that's an area of interest for us that has kinda spooled up from our.
Paula Ragan: Yeah, I think just to bring it all back around, there basically might be more kind of undiscovered WIM patients because nobody, because there yet has not been an understanding of the full paradigm of all the mutations that do cause disease. So that's an area of interest for us that has kind of spewed out from our genetic testing work. But stay tuned on that. Certainly, Renato's team's doing an excellent job. And in the future, certainly when we have some more meaningful updates to share, we'll look forward to doing that.
Genetic testing work, but stay tuned on that that certainly or not as team's doing excellent job and in the future will certainly when we have some more meaningful updates to share will afford to doing that.
Okay. Thanks, guys.
Thank you.
Operator: As a reminder, to ask a question, you would need to press star 1 on your telephone. To withdraw your question, press the pound key. I'm sure our next question comes from Mayank Mantani from B. Riley. Please go ahead. Good morning.
As a reminder to ask a question you would need to press star one on your telephone to which are your question first the pound key.
I sure. Our next question comes from my young on Tommy from B. Riley. Please go ahead.
Good morning, Thanks for taking my question and passing water boarding deduction d. that indeed IDH each in congrats to they got accounted for the promotion.
Renato Scarro: Thanks for taking my question and for the impressive word introduction data indeed at EHA, and congrats to Renata also for the promotion. Can you just remind us about the status of the manuscript for this data? And I know you talked a lot about vitae. Just any near-term learnings from that that could also help the SCN phase 1, phase 1-2 study enrollment? If you could comment on that, they're not open at this point, yeah.
Can you just remind us about the status of the man. It's good for you for these data and I know you Doug a lot about NBD, just any any need downloading from that but we also have the at the end of phase one phase one doing study enrollment.
If you could comment on that.
Sure there not only on the going out.
Sorry.
Paula Ragan: Yeah, so I mean, I think we're aiming to basically have the publication up in the second half of the year, which is still basically on track. Regarding the NVTAE data, I think, again, one of the things I didn't mention earlier is that we can increase the denominator in terms of the potential number of patients that we can treat with Maverix X4. And the, you know, what we're putting in place in terms of the platform regarding this, you know, characterization of these different variants, we can also apply to SCM. So this is a platform that we can actually apply to other potential non-CXCR4-related genes that can impact primary immunodeficiency, so we're building this capability in-house to really expand our capability in the primary immunodeficiency space. Okay, great. And then on the VIM Phase 3 study, can you comment on the proportion of sites between the U.S., ex-U.S., and even within the U.S., how many might be in the South?
So I mean, I think regarding to basically the publication in the second half you, which is supposedly on track.
Regarding any detail you do everything you're getting another thing that had mentioned new leaders that we can increase in denominated when it comes with potential number of patients that we can treat with my Brooks tool.
And the.
What we're putting in place in terms of the platform regardless.
Characterization of these different buildings, we can also apply to see yet.
So this is a platform that we can actually applied it into other.
Potential non CXC off will related genes that can impact probably be a deficiency. So we're building it's good and build the in house that really stands out that Buzzi <unk>.
The primary really consistency space.
Okay, Great and then on the damn phase three study.
It can you comment on the proportion of sides of the Green U.S. X U.S. and even within you as how many by being the indices out any any color you can give.
Paula Ragan: Any color you could give?
You know I think it's about a third U.S. two thirds ex the U.S. and then you know beyond that I think.
Paula Ragan: I think it's about a third U.S., two-thirds ex-U.S., and then beyond that, I think... I think the relevant thing is, are the sites, you know, engaged in enrolling? And our answer is, yes, where it's possible. I am excited that we've been, you know, enrolling patients, and they are there. We just have to be mindful that these are immunocompromised patients, and ultimately, it's always the physician and the patient's decision about when the right time is, you know, when it's the right time. But we're making progress. We maintain our guidance, and we'll look forward to, you know, sharing more clarity when we achieve it.
I think the relevant thing is are the sites you know engaged in enrolling and our answer is yes, where it's possible I am excited that we've been in <unk>, you know enrolling patients than they are there. We just have to be mindful that beat our immunocompromised patients and ultimately it's always the physician and the patient decision about when the right you know when it's the right.
Time, but we're making progress we maintain our guidance and we'll look forward to sharing more clarity when we achieved that.
Okay, Great and this final question on while even stronger.
Paula Ragan: Okay, great. And just a final question on Waldenstrom's.
Paula Ragan: Can you comment on, like, how many or what proportion of patients are in part two that you just talked about? Paula.
Yeah.
He didn't get any common done like.
How many or.
What proportion of patients are out in the bought do that gear. This dog the bugs.
Paula.
Yeah that they'll all be given the option I mean that the phase one be as part one and two so all patients will pass through both parts of the trial us along a safety permits.
Paula Ragan: Yeah, they'll all be given the... I mean, phase 1B is Part 1 and 2, so all patients will pass through both parts of the trial, as long as safety permits.
Okay, great. Thanks for taking my question.
Paula Ragan: Okay, great. Thanks for taking my question. Thank you. I have no further questions in the queue. At this time, I'd like to turn the call over to Paula Ragan, CEO, for closing remarks.
Thank you.
I show no further questions in the queue at this time I like to turn the call over to Apollo Reagan CEO for closing remarks.
Well. Thank you so much for joining us today, we look forward to continuing to provide updates on our progress of the approach. Our next key milestone. If you have any further questions. Please don't hesitate to reach out to Kansas and we hope you all stay safe and healthy enjoy the rest of the summer. Thank you again and have a great day.
Paula Ragan: Well, thank you so much for joining us today. We look forward to continuing to provide updates on our progress as we approach our next key milestones. If you have any further questions, please don't hesitate to reach out to Candice. And we hope you all stay safe and healthy and enjoy the rest of the summer. Thank you again, and have a great day.
Operator: Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect. My Outro for My 20th Birthday, BF-WATCH TV 2021
Ladies and gentlemen, this concludes today's conference call. Thank you for participate and you may now disconnect.
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