Q3 2019 Earnings Call
Greetings and welcome to X four Pharmaceuticals third quarter 2019 financial results and business update conference call.
Operator: Greetings and welcome to X4 Pharmaceuticals' 3rd Quarter 2019 Financial Results and Business Update Conference Call. At this time, all participants are in a listen-only mode. A 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, Stephanie Carrington, Managing Director of Investor Relations at Westpac. You may begin.
At this time, all participants are in listen only mode.
A question and answer session will follow the formal presentation. As a reminder, this conference call this being recorded.
It is now my pleasure to introduce your host Stephanie Carrington, managing director of Investor Relations, Ed Westlake you may begin.
Thank you operator.
Stephanie Carrington: Thank you, Operator. Good morning, everyone, and welcome to X4 Pharmaceuticals' third quarter 2019 Financial Results and Business Update conference call. I'm joined today by X4 Pharmaceuticals Chief Executive Officer, Dr. Paula Ragan, Chief Medical Officer, Dr. Lynn Kelly, and Chief Financial Officer, Adam Mostafa. Now turning to today's call, Paula will provide an overview of recent developments, Lynn will discuss clinical development progress, and Adam will recap the financial results for the third quarter. We will then open the call to your questions.
Good morning, everyone and welcome to export Pharmaceuticals third quarter 2019 financial results in business update conference call.
I'm joined today by export Pharmaceuticals, Chief Executive Officer, Dr. Paulo, Reagan, Chief Medical Officer, Dr., Lin Kelly and Chief Financial Officer, Adam must also.
Now turning to today's call Paul will provide an overview of recent developments.
Clinical development program and Adam.
Financial results for the third quarter.
Well then open the call to your question.
Stephanie Carrington: Before we begin, I would like to remind you that X4 will be making certain forward-looking statements during this call. Forward-looking statements include, but are not limited to, statements regarding X4's plans or the development of Mavrixifor X4P001 or any of its other product candidates, including with respect to clinical trials X4 plans to initiate, the design, rate of patient enrollment, and clinical site initiation for its clinical trials. Potential benefits of mesverixifor, including as a treatment for advanced renal cell carcinoma, WIM, Waldenstrom's, or severe congenital neutropenia
Before we begin I would like to remind you that X four we'll be making certain forward looking statements. During this call.
Looking statements include.
Not limited to statements regarding exports plans.
For the development, a matter except for X four pcrs or one or any of its other product candidates, including with respect to clinical trial exports plans to initiate the design rate of patient enrollment and clinical site initiation for its clinical trials.
Potential benefits Emas works before including as a treatment for advanced renal cell carcinoma, when Walden troms or severe congenital neutropenia.
Stephanie Carrington: The safety, durability, and efficacy of Maverix for X4's plans to announce future trial results and X4's plans to partner with any current or future third parties. Any statements made in this call that are not statements of historical fact may be deemed to be forward-looking statements. Forward-looking statements involve certain risks and uncertainties that could cause actual results to differ significantly from those projected, including, without limitation, the risks and uncertainties detailed in X4's most recent annual report on Form 10-K, filed with the Securities and Exchange Commission, or SEC, as updated by X4's current report on Form 8-K, filed with the SEC on April 11, 2019, and in all other filings X4 makes with the SEC. All forward Now turning to today's call, X4 Pharmaceuticals CEO, Paula Ragan. Go ahead, Paula.
The safety sterility and efficacy of networks for exports plans to announce future trial results and exports plans to partner with any.
Current or future third parties.
Any statements made in this call that are not statements of historical fact, maybe deemed to be forward looking statement for live and statements involve certain risks and uncertainties that could cause actual results to differ significantly from those projected and including without limitation, the risks and uncertainties detailed index for as much.
As an annual report on Form 10-K filed with the Securities Exchange Commission worse easy as indicated by exports current report on form 8-K filed with the FCC on April 11, 2019, and in all other filings as for mix.
Did you see see from fight that from time to time.
All forward looking statements made during this call are based on information available to export as of today, an export specifically disclaims any obligation to update. These statements are provided on this call as a result of future events or otherwise.
Now turning today's call export Pharmaceuticals C O Paulo Reagan go ahead Paul.
Thank you Stephanie and thank you everyone for joining us on this morning's call. They're very pleased to be speaking with all of you. Today. This is our first business update call. Since we completed our reverse merger with our sauna and export Pharmaceuticals, Inc., Matt commenced trading as a public company on NASDAQ in March between 19.
Paula Ragan: Thank you, Stephanie. And thank you, everyone, for joining us on this morning's call. We are very pleased to be speaking with all of you today. This is our first business update call since we completed our reverse merger with Arsanis and X4 Pharmaceuticals, Inc., commenced trading as a public company on NASDAQ in March of 2019 and the completion of our follow-on equity offering in April of 2019. Since that time, our team has been very active on multiple fronts.
And the completion of our follow on equity offering in April 2019, our team has been very active on multiple fronts since that time.
Paula Ragan: As a reminder, we are advancing the clinical development of Mavarixifor, a potentially first-in-class, once-daily, oral, small-molecule antagonist of the chemokine receptor CXCR4 across multiple indications. In the third quarter of 2019, we received notice that the European Commission had granted orphan drug designation to Mavarixifor for the treatment of Wim's syndrome, which followed the same designation received from the FDA Our core development plans for Maverix IV are focused on three rare disease areas as follows. We initiated a pivotal phase three global clinical trial for Mavarixifor for the treatment of WIM syndrome in June 2019. We are currently enrolling patients in this trial and are on track to fully enroll the trial by the middle of 2020. We also remain on track to provide a WIM prevalence and patient identification update by the end of the first quarter of 2020.
As a reminder, we are advancing the clinical development of Mavericks before potentially first in class once daily oral small molecule antagonist of the chemo kind receptor CXC, our four across multiple indications.
During the third quarter of 2019, we received notice that the European Commission granted orphan drug designation to Mavericks before for the treatment of when syndrome, which followed the same designation received from the FDA in October 2018.
Our core development plans for matter, except for our focused on three rare disease area that's fallen.
We initiated a pivotal phase three global clinical trial for Maverick before for the treatment of women syndrome. In June 2019, we're currently enrolling patients in this trial and are on track to fully enroll the trial by the Middle of 2020. We also remain on track to provide a win prevalence and patient identification update.
By the end of the first quarter of 2020.
Paula Ragan: We initiated a Phase 1b clinical trial of Maverix IV for the treatment of severe congenital neutropenia earlier this week, and preparations are underway to initiate a Phase 1b clinical trial of Maverix X4 for the treatment of Waldenström's macroglobulinemia during the final quarter of 2019. Lynn will elaborate shortly on the progress that we're making across each of these trials. On September 30th, at the European Society for Medical Oncology 2019 Congress in Barcelona, we released positive results from the Phase 2a portion of our 65-patient open-label Phase 1-2 clinical trial of Mavrixifor in combination with Exitinib or Enlita in patients with advanced clear cell renal cell carcinoma. We believe these promising results demonstrated a meaningful improvement in median progression-free survival in a heavily pre-treated patient population with poor prognoses and provided clinical evidence that continues to support Maverix IV's potential role in inducing immune-related antitumor responses. Ann Bolster is the published evidence that has shown a generally favorable safety and tolerability profile.
We initiated a phase one be clinical trial, a mavericks before for the treatment of severe congenitally neutropenia earlier this week.
And preparations are underway to initiate the phase one be clinical trial Mavericks before for the treatment of Waldenstrom macroglobulinemia during the final quarter Htwo 2019.
Let me elaborate shortly regarding the progress that we're making across each of these trials.
On September Thirtyth of European Society for medical Oncology 2019 Congress in Barcelona, We released positive results from the phase two a portion of our 65 patient open label Phase one two clinical trial of Mavericks. If we're in combination with exact NIM or in later in patients with advanced.
Clear so renal cell carcinoma.
We believe these promising results demonstrated a meaningful improvements in median progression free survival any heavily pre treated patient population with poor prognoses.
And provided clinical evidence that continues to support Mavericks worth potential rule and inducing immune related.
Anti tumor responses.
And bolsters the published evidence, which is shown in generally favorable safety and Tolerability profile.
Paula Ragan: We are encouraged by these data, including data from the 8 patients, or 12%, who remain on combination therapy with Maverix X4 and Exit Nib for more than 12 months past the primary endpoint. We are actively engaging in partnering discussions as we look forward to continuing to explore the potential benefit of Mavrixifor in underserved cancer patients with solid tumors, including as a potential triple combination agent in addition to tyrosine kinase inhibitors and checkpoint inhibitor therapy or in combination with other standard of care treatments. Also, in the third quarter of 2019, we entered into an agreement with Abisko Therapeutics to develop and commercialize Maverix X4 in combination with checkpoint inhibitors or other agents in Greater China with a focus on solid tumor oncology indications.
We are encouraged by these data, including the data from the eat patients or 12% remain on comedies and therapy with Mavericks before any sitting in for more than 12 months past the primary endpoint.
We are actively engaging in partnering discussions as we look forward to continuing to explore the potential benefit Mavericks before an underserved cancer patients with solid tumors, including as a potential triple combination agent. In addition to tyrosine kinase inhibitors and checkpoint inhibitor therapies or in combination with.
The other standard of care treatment.
Also in the third quarter of 2019, we entered into an agreement with a bisco therapeutics to develop and commercialize Mavericks before in combination with checkpoint inhibitors were other Asian in greater China with a focus on solid tumor oncology indications.
Paula Ragan: We retained full rest-of-world rights to develop and commercialize Mavericks outside of greater China for all indications and the ability to utilize any data generated from this collaboration for X4's rest-of-world development program. We have continued to build out our organization on multiple fronts to prepare to deliver our milestones in 2020 and beyond. Over the past month, we have strengthened our executive team, as exemplified by our recent hires. Renato Skurl, PhD, was appointed as our Senior Vice President of Research and Development in September. Dr. Skurl has over 25 years of experience.
We retained full rest of world rights to develop.
And commercialize Mavericks before outside a greater China for all indication and the ability to utilize any data generated from this collaboration for ethane exports rest of World development program.
We've continued to build out our organization on multiple fronts to prepare to deliver our milestones in 2020 M. beyond.
Over the back half months, we have strengthened our executive team as exemplified by our recent hires.
We're not a girl Phd was appointed as our senior Vice President of research and development in September doctors girls has over 25 years of experience.
Leading the discovery and development of small molecule drugs to treat rare diseases cancer infection in neuro degenerative diseases.
Paula Ragan: In addition, he was one of the original founders of X4 Pharmaceuticals, and it's great to have him join as an R&D leader for our talented team in Vienna. Derek Meisner has joined us this week as General Counsel.
In addition, he was one of the original founders of X, where pharmaceuticals, and it's great to have him join as an R&D leader for our talented team Indiana.
Derek Meissner has joined US this week as general counsel 'cause legal case career has spanned over two decades any the season that legal veteran brings in house experience at both biotechnology related companies and security from.
Paula Ragan: His legal career spans over two decades, and he is a seasoned legal veteran who brings in-house experience at both biotechnology-related companies and security firms. In addition, Bill Elitsky joined our Board of Directors in the third quarter. He brings more than two decades of biopharmaceutical executive leadership experience at both public and private companies with significant expertise in global rare disease commercialization, including a particular focus on commercial strategy, pricing, reimbursement, and market access. We are very pleased to have Bill at this exciting time for the company as we begin to lay the groundwork for a commercial organization. I'd now like to turn the call over to our Chief Medical Officer, Dr. Lynn Kelley.
In addition, the Alisky joined our board of directors in the third quarter. He brings more than two decades of bio pharmaceutical executive leadership experience at both public and private companies with significant expertise in global rare disease commercialization, including a particular focus on commercial strategy pricing right.
Imbursement end market access we're very pleased to have bill at this exciting time for the company as we began laying the groundwork for a commercial organization.
I'd now like to turn the call over to our Chief Medical Officer, Dr. Lin Kelly.
Unknown Attendee: Thank you very much, Paula. Our Pivotal Phase III Global Clinical Trial for the Treatment of Wim Syndrome is well underway. As you may recall, the Global 4-WIM Trial is a 52-week, randomized, double-blind, placebo-controlled, multi-center study designed to evaluate the safety and efficacy of maverixifor in genetically-confirmed WIM patients. The trial is designed to enroll 18 to 28 subjects in approximately 20 countries, followed by an open-label extension trial. The primary efficacy endpoint for the trial will compare the level of circulating neutrophils relative to a clinically meaningful threshold of 500 cells per microliter in response to Mavrixiv4 versus placebo measured during multiple 24-hour periods over the course of 52 weeks. Secondary endpoints include infection rates, wart burden, and assessment of immune system function and quality of life.
Thank you very much color, our pivotal phase three global clinical trial for the treatment of when syndrome is well underway as you may recall the global for wind trial is a 52 week randomized double blind placebo controlled multicenter study designed to evaluate the safety and efficacy of never before genetically confirmed.
Armed when patients the trials designed to enroll 18 to 20 subjects in approximately 20 countries followed by an open label extension trial.
The primary efficacy endpoint for the trial will compare the level of circulating neutrophils relative to a clinically meaningful threshold of 500 sells for Michael leader in response to never acts of war versus placebo measured during multiple 24 hour periods over the course of 52 weeks secondary endpoints include infection rates.
Worked burden and assessment of immune system function and quality of life. The trial is currently enrolling patients and we are in the process of activating sites globally. We remain on track to fully enroll the trial in the middle of 2020 timeframe. We would also like to remind you that there's a win fees to open label trial update clean for next year.
Unknown Attendee: The trial is currently enrolling patients, and we are in the process of activating sites globally. We remain on track to fully enroll the trial in the middle of 2020. We would also like to remind you that there is a WIM Phase 2 Open Label Trial update planned for next year. Earlier this week, we initiated a Phase 1b clinical trial in Severe Congenital Neutropenia, or SCN. This is a rare blood disorder characterized by abnormally low levels of certain white blood cells.
Sure.
Earlier this week, we initiated a phase one be clinical trial and severe congenital neutropenia, whereas C. N. This is a rare blood disorder characterized by abnormally low levels of certain with blood cells.
Unknown Attendee: This trial is planned as a 14-day exploratory trial to assess the genetic profiles of the SCN patients at baseline, assess safety and tolerability, and examine responses to Mavrixifor in up to 45 patients across SCN and exploratory subpopulations. One additional benefit of this trial is that it may enable us to correlate genetic profiles with potential maverick CIFOR responses, supporting future development of maverick CIFOR as a targeted therapy in a genetically predefined population of SCN. We are also on schedule to commence a Phase 1b clinical trial in Waldenström's macroglobulinemia during this final quarter of 2019. As a reminder, Waldenstrom's is a rare form of non-Hodgkin's lymphoma and has an estimated prevalence of greater than 13,000 patients in the United States and European Union.
This trial is planned as a 14 day exploratory trial to assess for genetic profiles of the S. C N patients at baseline assess safety and Tolerability and examiner responses to never acts of war in up to 45 patients across S. C. N an exploratory sub populations one additional benefit of this.
Trial is that it may enable us to quarterly genetic profiles with potential never to support responses supporting future development of Mavericks before as a targeted therapy in a genetically predefine population of S. C N.
We're also one scheduled to commence phase one be clinical trial in Waldenstrms Macroglobulinemia. During this final quarter of 2019 as a reminder, waldenstrom is a rare form of non Hodgkin's lymphoma and has an estimated prevalence of greater than 13000 patients in the United States and her European.
In Union.
Unknown Attendee: 30-40% of Waldstrom's patients have a WIM-like mutation in a CXER4 gene. This trial is currently being designed as a multinational study to assess Mazarixavir in combination with Ibrutinib in patients with MyD88 plus CXER4 mutations. We will be examining safety to determine the optimum dose and will gain some insights into activity through assessing IgM levels in the blood. High IgM levels are a hallmark of the disease, and we believe any decrease seen will enable us to gain an early understanding of the potential of Maverick support to provide benefit in the CXER4 mutant-focused patient population. Thank you all, and I will now turn the call over to our Chief Financial Officer, Adam Mostafa.
30% to 40% of Wall Trust patients have a win like mutation in the CX here for gene. This trial is currently being designed as a multinational study to assess Mavericks afore in combination with the Britain in patients with Mydeighty eight plus CX here for mutations we will be examining safety to determine the optimum.
And we'll gain some insights into activity through assessing ITM levels in the blood.
Hi, Hi, Jim levels, our homework biomarker of the disease and we believe any decrease seen will be enable us to gain in early understanding of the potential of never acts of war to provide benefit in the CX here for music focused patient population.
Fuel and I will now turn the call over to our Chief Financial Officer, Adam myself.
Adam S. Mostafa: Thank you very much, Lynn. As of September 30th, 2019, cash, cash equivalents, and restricted cash were $77 million, as compared to $96 million as of June 30th, 2019. This decrease of approximately $19 million reflected cash used to fund operating activities during the third quarter and included a $6.5 million cash payment for full repayment of X4's loans with FFG. Note that this repayment was part of the debt refinancing executed with Hercules in June 2019, under which we still have $5 million in borrowing availability.
Thank you very much Lynn as of September Thirtyth, 2019, cash cash equivalents unrestricted cash were $77 million as compared to $96 million as of June Thirtyth 2019.
This decrease of approximately $19 million reflected cash used to fund operating activities during the third quarter.
And included a 6.5 million dollar cash payments for full repayment of Exforge loans with F. G.
No. This repayment was part of the debt refinancing executed with Hercules in June 2019, under which we still have $5 million in borrowing availability.
Research and development expenses were $8.6 million for the third quarter 2019, as compared to $8.9 million for the second quarter 2019.
Adam S. Mostafa: Research and development expenses were $8.6 million for the third quarter of 2019 as compared to $8.9 million for the second quarter of 2019. General and administrative expenses were $4.4 million for the third quarter of 2019 as compared to $4.6 million for the second quarter of 2019. During the third quarter of 2019, we transferred the rights to develop and commercialize the ASN 200, 300, and 500 programs acquired through the merger with Arsonis.
General and administrative expenses were $4.4 million for the third quarter 2019, as compared to $4.6 million for the second quarter 2019.
During the third quarter 2019, we transferred the rights to develop and commercialize the isn't 200 305 hundred programs acquired through the merger with our saunas.
Adam S. Mostafa: Accordingly, we de-recognized our IPR&D intangible assets and recorded a $4 million charge during the three months ended September 30, 2019. The charge reflects the write-off of the IP R&D balance of $4.9 million, less $900,000 of cash received from the sale during the quarter. The net loss was $17.7 million for the third quarter of 2019, or a net loss per basic and diluted share of $1.22. As compared to a net loss of $13.4 million for the second quarter of 2019, or a net loss per basic and diluted share of $1.02. Excluding the IP R&D charge, the net loss for the third quarter 2019 was approximately the sum of the R&D and G&A expenses in the quarter, as mentioned. We will now turn the call over to the operator for Q&A.
Accordingly, we derecognized, our IP R&D intangible assets and recorded 4 million dollar charge during the three months ended September Thirtyth 2019.
The charge reflects the write off of the IP R&D balance of $4.9 million less $900000 of cash received from the sale during the quarter.
Net loss was $17.7 million for the third quarter of 2019 or net loss per basic and diluted share of $1.22 cents as compared to a net loss of $13.4 million for the second quarter of 2019 or net loss per basic and diluted share of one dollar and two cents.
Yes.
Excluding the IP R&D charge net loss for the third quarter 2019 was approximately the some of the R&D and GE and expenses in the quarter as mentioned.
We will now turn the call over to the operator for today.
Thank you as a reminder to ask a question you would need to press star one on your telephone.
Operator: Thank you. 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 roster. I will show our first question. It comes from Marc Frahm from Cowan & Company. Please go ahead.
Your question first the pelkey. Please standby were compiled the culinary roster.
I show our first question comes from Marc Frahm from Cowen and company. Please go ahead.
Hi, Thanks for taking my questions. This morning, maybe on the CN trial that you just started.
Marc Alan Frahm: Hi, thanks for taking my questions this morning. Maybe on the SCN trial that you just started, you could give some detail. I assume you're looking for some particular families of mutations within SCNs. You can maybe give some of the breakdown of maybe how many mutations you expect to enroll within the 45 patients and if there are specific sub-cohorts that you're looking for.
Just to give some detailed I assume you're looking for.
Some particular families of mutations with NFC and.
You can maybe give some of the breakdown of yeah, maybe how many mutation do you expect to enroll within that 45 patients and you know if there are specific.
Of course that you're looking for.
Thanks, Mark for the question. This is Paul inland will add commentary so were looking pre defined we're looking at certain mutation called gathered to GE six PC three and a few other very recent genetically defined immunodeficiencies that appear.
Paula Ragan: Thanks, Mark, for the questions. This is Paula, and Lynn will add commentary. So, we're looking – predefined, we're looking at certain mutations called GATA2, G6PC3, and a few other very recent genetically-defined immunodeficiencies that appear to have some overlap with the CXCR4 pathway. We're also using a large panel that would cover additional genes associated with immunodeficiencies and screening all patients against that, so we can fully understand the breadth of these patients' mutations and correlate that with potential response to Mavirixa4.
Here to have some overlap with the seeks care pathway for also using a large panel that would cover additional genes associated with Immunodeficiencies and screening all patients against that so we can fully understand the breadth of these patients mutations and correlate that with potential in response to matter at the four.
Okay great.
Marc Alan Frahm: Okay. All right. Great.
Thank you.
Stephen Douglas Willey: Our next question comes from Stephen Wiley from Stifo. Please go ahead.
Our next question comes from Stephen Willey from Stifel. Please go ahead.
Hi, This is Alan on for Steve I'm, just one question there wasn't ash abstract yesterday released evaluating families with when syndrome that I believe sound serious infections are fine hospitalization to be relatively rare I think it was about one per year I know infection rate as a secondary endpoint was the phase III trial. So.
Unknown Attendee: Hi, this is Ellen on behalf of Steve. Just one question. There was an ASH abstract released yesterday evaluating families with Winn syndrome that I believe found serious infections requiring hospitalization to be relatively rare. So I think it was about one per year. I know infection rate is a secondary endpoint in the phase three WIM trial, so I was just wondering if you expect serious infections to be as low as that abstract suggests. And if so, just how will you evaluate any type of delta resulting from treatment on mavericks before with hospitalizations related to infections? Thanks.
I was just wondering if you expect serious infections to be as low as that Abcheck suggests and its so just how will you evaluate any type of delta, resulting from treatment on my first before wed like hospitalizations related to affections. Thanks.
So show a we've seen we just saw the abstract for that yesterday D. A mutation in Russia autosomal dominant with very little penetration. So I think theres, a breadth of patients that present with somewhat very serious life threatening infections and some with milder chronicle chronic infections that result in some of the long terms.
Paula Ragan: So, we have seen, we just saw the abstract for that yesterday. The mutation is actually autosomal dominant with variable penetration, so I think there's a breadth of patients that present with some with very serious, life-threatening infections and some with mild or chronic infections that result in some of the long-term sequelae. In the setting of the clinical trial, we have prospectively put together an infection adjudication committee that is independent of the company and composed of three experts that will be helping to assess the infections that happen both in the placebo group as well as potentially in the treatment arm. And so, we are looking at that. We expect, we've done some previous work looking at infection rates in Phase 2 patients, and then we'll be sharing that later on in 2020 when we share the Phase 2 report.
Equally we in the setting in the clinical trial have prospectively put together an infection adjudication committee that is independent from the company of three experts.
That will be helping to assess the infections that happened both in the placebo group as well as potentially in the treatment arm and so we are looking at that we expect a we've done some previous work looking at a infection rates in the phase two patients and then we'll be sharing that later on in 2020.
When we share the a phase to report.
Unknown Attendee: Okay, great. And then just one more question for me. What kind of differences, if any, do you expect to see in response between patients that have SCN versus other types of congenital neutropedia disorders? Thank you.
Okay, Great and then just one more question for me what kind of differences do if any do you expect to see in response [laughter] between patients that have s. yen versus other types of congenital neutropenia disorders. Thank you [laughter].
I think we expect a d., we know that never support is able to mobilize white blood cells, and especially neutrophils. So we wouldn't expect anything different I think what we're expecting to learn is all about the different mutations and whether or not the responsiveness.
Unknown Attendee: I think we expect the We know that Maverix X4 is able to mobilize white blood cells and especially neutrophils, so we wouldn't expect anything different. I think what we're expecting to learn is all about the different mutations and whether or not they're responsiveness, so it really is an informed Phase 1B trial in terms of our movement forward. Paula, do you have anything to add?
So it really isn't inform a phase one be trial in terms of our movement forward. Paul do you have anything to add.
Paula Ragan: No, I think we just want to comment that we are looking for the magnitude of response. We do expect responses in essentially all patients given the mechanism of action. The important correlation is the magnitude of the response with the correlating genetic profile.
No I think we just want to comment that we are looking for magnitude of response, we do expect responses and essentially all patients given the mechanism of action important correlation is the magnitude of the response with the correlating genetic profile.
Okay, great. Thank you.
Unknown Attendee: Okay, great. Thank you.
Thank you. Our next question comes from Joel Beatty from Citi. Please go ahead.
Joel Beattie: Thank you. Our next question comes from Joel Beattie from Citi. Please go ahead.
Hi, Thanks for taking my questions first one is on the when patient identification update in the first quarter next year could you provide maybe a little more information on the types of analyses are approach that you're using to.
Paula Ragan: Hi. Thanks for taking the questions. The first one is on the WIM patient identification update in the first quarter of next year. Could you please provide maybe a little more information on the types of analyses or approaches that you're using to help identify patients?
Okay.
Sure we haven't explicitly shared the tool that X four as using although I'll highlight what other rare disease companies typically used to approach how to triangulate prevalent.
Paula Ragan: Sure, we haven't explicitly shared the tools that X4 is using, although it will highlight what other rare disease companies typically use to approach how to triangulate prevalence. The typical tools are electronic medical records research. They are also looking at existing genetic databases, either housing a kind of retrospective samples or forward-looking samples. And we have disclosed the collaboration with Invitae, where we're prospectively providing genetic screening for physicians for patients of unknown diagnosis. So across those three types of approaches, we'll be able to give you updates on some of those initiatives that X4 is pursuing.
The typical tools, our electronic medical Records research. They are also looking at existing genetic databases, either housing kind of retrospective samples or forward looking samples and we have disclosed the collaboration with envied, hey, where where prospective we.
Providing genetic screening for.
Physicians for patients of unknown diagnosis, so across the three types of approaches will be able to give you updates on some of those initiatives that xplore is pursuing.
Right.
Paula Ragan: Great. Another question on phase 3 in Wimps Syndrome. I know it's still early in enrollment, but could you give us a sense of confidence in the timing of enrollment and if there were some initial patients that were kind of waiting to get in that trial that were able to enroll early? Or is it some type of enrollment pattern that may be ramping up over time as sites get online?
Another question on the phase three and one son Jerome I know, it's still early on in enrollment I guess could you give a sense of other confidence and the timing of enrollment and sort of if there were some initial patients every kind of waiting to get an IDE trial that we're able to enroll early ours is gone.
On pattern, and then maybe <unk> wrapping up overtime as folks get online.
I think you hit the nail on the head in terms of your sites online we we have identified.
Unknown Attendee: I think you hit the nail on the head in terms of your sites online. We have identified patients, and our biggest hurdle right now is the paperwork and regulatory aspect of the different sites globally. And so we are actively, not only right now recruiting patients, but we are actively getting those sites up online globally to enroll the patients. We are confident that we will have full enrollment by the mid-2020 time period, as we've always given guidance.
Patients and our biggest hurdle right now is the paperwork and regulatory aspect of the different.
Sites globally, and so we are actively not only right now recruiting patients, but we are actively getting those sites up online globally to enroll the patients. We are confident that we will have full enrollment by the mid 2020 time period as we've always given guidance.
Great and then one last question that's kind of a size question on mechanism of action and various indications.
Joel Beattie: Great. And then there is one last question.
Joel Beattie: It's kind of a science question on the mechanism of action and various indications. It seems like Mavirixifor for neutropenias is increasing the mobilization of neutrophils. And I'm wondering if you could help reconcile that with what you saw in the oncology setting, where it seems like it was decreasing mobilization of MDSCs, which I believe largely consist of neutrophils. But it seems like the effect on neutrophils may be a little bit different between those two settings.
It seems like never before for neutropenia.
There is increasing the mobilization of neutrophils.
And I'm wondering if you could help reconcile that with what you saw.
Oncology setting where it seems like it was decreasing mobilization of fees, which I believe largely consists of neutrophils. So it seems like the effect on neutrophils, maybe a little bit different between those two setting.
Sure. Thanks to all this is Paula so the the mechanism of action is actually are that the root causes a disease is somewhat different in those two different settings. So on the tumor microenvironment, there is and evidence to support their isn't overproduction of the Axiall 12, which has the full like in a six year for.
Paula Ragan: Sure. Thanks, Joel. This is Paula.
Paula Ragan: So, the mechanism of action is actually, or the root cause of the disease is somewhat different in those two different settings. For example, in the tumor microenvironment, there is evidence to support that there is an overproduction of CXCL12, which is the sole ligand of CXCR4, and that overproduction causes the differential chemotaxis of MDSCs versus other non-immunosuppressive cells. Publications in Nature Medicine support the differentiated attraction of immunosuppressive cells in the tumor microenvironment based on that chemokine. So, in some ways, it's an overproduction of CXCL12 as the root cause of the problem in the tumor microenvironment, and when you disturb that signaling, you're able to reestablish the normal immune cell trafficking with CD8-positive T cells, which we have shown with In Wimp syndrome, the ligand is normal, and the mutated receptor keeps the white blood cells trapped in the bone marrow where they're initially produced. So, it's still a trafficking issue related to aberrant signaling; it's just a different origin of the lack of trafficking response. I hope that this helps answer your question.
And that overproduction causes that differential chemo, Texas of Mds season versus other non immunosuppressive cells publications and nature medicine support actually the differentiated attraction of immuno suppressive cells and the tumor microenvironment based on that chemo quynh. So.
In some ways. It's an it's an overproduction of CX IL 12, as the root causes of the problem and the tumor microenvironment and when you disturb that signaling you're able to reestablish the normal immune cell trafficking with kiera <unk>, sorry, Cdeight positive T cells, which we have shown with our phase one be trial in melanoma.
In a win syndrome, it's at the inverted problem. The ligand is normal and the mutated receptor keeps the white blood cells trapped in the bone marrow, whether initially produced so it's still a trafficking issue related to the aberrant signaling is shut the different origin of the.
A lack of trafficking response I hope that helps answer your question.
Joel Beattie: Yeah, I appreciate it. Great. Thank you very much.
Yeah I appreciate it thank you very much.
Thank you.
Swayampakula Ramakanth: As a reminder, to ask a question, you will need to press Star 1 on your telephone. To withdraw your question, press the pound key. Our next question comes from Swayampakula Ramakanth from HC Wainwright. Please go ahead.
As a reminder to ask a question you would need to press star one on your telephone.
Question first the county.
Our next question comes from Swayam part cool off Ramakanth from H.C. Wainwright. Please go ahead.
Unknown Attendee: Thank you. Good morning, folks. Thanks for taking my questions. To start off, on Waldenstrauntz, what needs to be accomplished before the start of the Phase 1B trial that you're planning to start this quarter?
Thank you.
Good morning folks thanks for taking my questions.
To start off.
On on Waldenstrms.
Trial work needs to be accomplished before the start of the creates long beach, so to your planning to stuff or this quarter.
Hey, it's really a we're in a we've already submitted the protocol to the FDA and we already have our primary sites identified and we're getting through the paperwork for the IR be submissions. So there is nothing I'm, we're just marching through the typical cadence.
Unknown Attendee: It's really, we're in a, we've already submitted the protocol to the FDA, and we already have our primary sites identified, and we're getting through the paperwork for the IRB submission. So there is nothing, we're just marching through the typical cadence of any clinical trial, and we have worked with the investigators closely already and think we're in good shape.
Any clinical trial and we have worked with the investigators closely already and I think we're in good shape.
HM staying on the topic.
Unknown Attendee: Staying on the topic, I'm just trying to understand, you know, what sort of confidence you have in Averix for this indication since we have seen quite a few clinical candidates not be successful for Walden Street Syndrome.
Just one on those and a walk the I'm sort of confidence you have.
For.
For this indication since we have seen quite a few clinical candidates yeah, you know not being successful for wireless phones.
Syndrome.
Unknown Attendee: Well, I think we're confident of the success, and I think there's an unmet clinical need that has been both reinforced by discussions with the agency and investigators, as the combination double mutants with the MITEI-88 and the CXCR4 mutation certainly have a shallower and a worse outcome, and there is no alternative trial right now that will be enrolling patients with that profile. And so, while there are other drugs, such as Abrutinib and Xanabrutinib trials that exist or have been completed, there's nothing that addresses the double mutant population right now.
Well I think I think we're confident of the success and I think theres, an unmet clinical need that has been both reinforced by discussions with the agency and investigators.
As a the combination double meetings with the Mighty 88, and the CX here for mutation certainly has a shallower and a worse outcome and there is no alternative trial right now that will be enrolling patients with that profile and so while there is other.
Drugs, such as a brutal <unk> and Zana Bruton of trials that exist or have been completed there's nothing that addresses a double mutant population right now.
Swayampakula Ramakanth: Okay, thank you. And then the last question from me is the collaboration, the relationship that you have initiated with Abisko in China. Could you give us some color as to how Abisko is planning to run this development program and when we could potentially hear about initiating clinical studies in China, if they need to be done there?
Okay. Thank you and then the Colossus or for me is there.
Collaboration though the relationship you have initiated its itself this quarter in China <unk> could you give us some color as to how or this gross planning to under development program and then could.
Potentially here, but initiation of clinical studies in China.
And then it didn't there.
Unknown Attendee: So, we've created a joint steering committee. We are working on the appropriate protocols and the cadence. We're not ready to share at this time what those are, but the collaboration between ourselves and the clinical and business sides of the teams on both sides of the ocean is actually going forward quite nicely. Thank you. Thank you very much
So we are a we've created a joint steering committee, we are working on the appropriate protocols and the cadence we're not ready to share at this time, what those are but the collaboration between ourselves and the clinical and business side of the teams on both sides of the Ocean Uh Huh.
Her a actually going forward quite nicely.
Swayampakula Ramakanth: Thank you. Thank you for taking time to answer all my questions.
Thank you thanks for taking all the questions.
Thank you.
Paula Ragan: I have no further questions than the queue. At this time, I'd like to turn the call over to Paula Ragan, CEO, for closing remarks. Please go ahead.
I show no further questions in the queue at this time, all like to turn the call over to Apollo Reagan CEO for closing remarks. Please go ahead.
Paula Ragan: Thank you very much for joining the call today. We look forward to continuing to share progress as it evolves, and I wish everyone a great day. Bye-bye.
Thank you very much for joining the call today, we look forward to continuing to share progress as it evolves and I wish everyone a great day.
Right.
Ladies and gentlemen, this concludes today's conference call. Thank you for participating you may now disconnect.
Operator: Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.
Operator: BF-WATCH TV 2021