Q1 2021 ImmunoGen Inc Earnings Call
Operator: Good morning, and welcome to the Immunegence first quarter of 2021 Financial and Operating Results Conference Call. Today's conference is being recorded. At this time, I'd like to turn the call over to Courtney O'Conick, Senior Director of Corporate Communications and Investor Relations. Madam, please go ahead.
Good morning, and welcome to day and unit James first quarter of 2021 financial and operating results Conference call. Today's conference is being recorded and at this time I'd like to surgical rich R. A T Kearney Iconix senior director of corporate Communications and <unk>.
And after releasing ma'am. Please go ahead.
Courtney O'Conick: Good morning, and thank you for joining today's call. Earlier today, we issued a press release that includes a summary of our recent progress in our first quarter 2021 financial results. This press release and a recording of this call can be found under the Investors and Media section of our website at Immuningen.com. With me today are Mark Eniddy, our president and CEO; Anna Birkenblit, our chief medical officer; and Susan L.
Good morning, and thank you for joining today's call earlier today, and we issued a press release that includes a summary of our recent progress and first quarter 2021 financial results. This press release and a recording of this call can be found under the investors and media section of our website and immunogen Dot Com with me today are Mark Kennedy, our president and CEO and <unk>.
And Brad our Chief Medical Officer, and Susan Altshuler, our CFO during.
Courtney O'Conick: During today's call, we will review key accomplishments for the business over the last three months, our financial results, and anticipated upcoming events. During the discussion, we will use forward-looking statements, and our actual results may differ materially from such statements. Descriptions of the risks and uncertainties associated with an investment in immunogen are included in our SEC filings. And with that, I'll turn the call over to Mark. Thanks, Courtney.
During today's call, we will review key accomplishments for the business over the last three months, our financial results and anticipated upcoming events during the discussion and we will use forward looking statements and our actual results may differ materially from such statements descriptions of the risks and uncertainties associated with an investment and immunogen are included in our SEC filings and with that I'll turn the call.
Mark Thanks, Courtney and good morning, everyone and thank you for joining us today I'll begin with our lead program or if it talks about and recently completed enrollment and so right and which is our pivotal single arm study in platinum resistant disease, which has shifted the anticipated timing of top line data from the third into the fourth quarter of this year.
Mark Joseph Enyedy: Good morning, everyone, and thank you for joining us today. I'll begin with our lead program, Mervatuximab. We've recently completed enrollment in Saraya, which is our pivotal single-arm study in platinum-resistant disease, which has shifted the anticipated timing of top-line data from the third to the fourth quarter of this year and the projected submission of the BLA into the first quarter of 2022. We've also experienced some COVID-related impact on accrual for our confirmatory Mirosol trial and correspondingly now expect the readout on the primary endpoint to move from the second to the third quarter of 2022. Anna will provide some more color around the updated timelines in a moment.
And the projected submission of the BLA and to the first quarter of 2022, we've also experienced from COVID-19 related impact on accrual for our conform for confirmatory Mirasol trial, and correspondingly and now expect the readout on the primary endpoint and moved from the second to the third quarter of 2022, and we'll provide some.
More color around the updated timelines and a moment and importantly with positive results from these studies, we continue to anticipate potential accelerated approvals mirv and talks a mab and 2022 and full approval in 2023 beyond platinum resistant ovarian cancer, we submitted a protocol for a single arm study of merger talks and map.
Mark Joseph Enyedy: Importantly, with positive results from these studies, we continue to anticipate potential accelerated approval of Mervatuximab in 2022 and full approval in 2023. Beyond platinum-resistant ovarian cancer, we submitted a protocol for a single-arm study of myrotoxamab monotherapy in recurrent platinum sensitive disease and anticipates initiating patient enrollment for this cohort in the second half of this year. This study further expands our approach to displacing single-agent chemotherapy in later lines of treatment in ovarian cancer.
Mono therapy, and recurrent platinum sensitive disease, and anticipate initiating patient enrollment for this cohort and the second half of this year. This study further expands our approach to displacing single agent chemotherapy and later lines of treatment and ovarian cancer.
Mark Joseph Enyedy: Turning to Mervituximab in combinations, we were pleased to learn that mature data from our Phase 1B forward 2 doublet of Mervatum and patients with Platinum agnostic disease were accepted for an oral presentation at ASCO this year. In addition, a 70 patient IST, led by Dr. Rebecca Aaron at UAB, is now underway evaluating Mervituximab in combination with carboplatin in the neo Finally, we're supporting a randomized study comparing Myrtotoxymaab combined with carboplatin to standard of care and patients with recurrent platinum sensitive disease in a large IST led by Philip Harder at the AGO in Germany, with patient enrollment expected to start in the second half of this year. We continue to advance our second pivotal program, IMGN 632, and CD-123 positive hematologic malign
Turning to Mervyn talks and Mab and combinations, we were pleased to learn that mature data from our phase one b for two doublet of mirv. It talks a mab plus a vast and in patients with platinum agnostic disease were accepted for an oral presentation and I'd ask though this year and addition of 70 patient I S. T led by Dr. Rebecca Erin and.
A D is now underway evaluating bavituximab in combination with Carboplatin and the neo adjuvant setting and finally, we're supporting a randomized study comparing murdo talks a mab combined with Carboplatin to standard of care and patients with recurrent platinum sensitive disease, and a large I S. T led by Philip harder and the a G O and Germany.
And with patient enrollment expected to start and the second half of this year. We continue to advance our second pivotal program I M. G and 632 and C. D 123 positive hematologic malignancies, having and established a path for full approval and V. P. D. C and we are progressing in our phase one expansion cohort and frontline patients and.
Mark Joseph Enyedy: Having established a path to full approval in BPDCN, we are progressing our phase one expansion cohort in frontline patients and expect to generate top-line data in the first half of 2022 with potential approval next year. We are also advancing 632 in combination studies with Aesididine and Venetaclax in relaps and refractory and frontline AML patients and look forward to presenting data from these combinations at Ash in Moving to our earlier stage pipeline, we presented preclinical data at AACR for our first-in-class, Adam 9 targeting ADC, IMGC 936, which we are co-developing with macrogenic.
And expect to generate topline data and the first half of 2022 with time with potential approval next year. We are also advancing 632 and combination studies with Azacitidine, and Geneva, clocks, and relapsed and refractory and frontline AML patients and look forward to presenting data from these combinations at ash in December.
Moving to our earlier stage pipeline and presented preclinical data at ACR and for our first in class Adam nine targeting ADC I M. G. C 93, six which we are co developing with Macrogenics.
Mark Joseph Enyedy: We also continued I&D enabling activities for our next generation foliate receptor alpha targeting ADC, IMGN 151, and expect to submit an application by year end. 2020 and 2022 are critical years in immunogen evolution as we prepare to bring two products to the market and further our innovative portfolio of transformative ADCs for solid tumors and hematologic malignancies. We look forward to keeping you apprised of our progress as we generate clinical data and prepare for commercialization. With that, I'll turn the call over to Anna to provide some additional color on our clinical programs. Anna. Thanks, Mark. I'll start with Saraya.
We also continued our IND, enabling activities for our next generation folate receptor alpha targeting ADC, IMG and 151, and it's and expect to submit an application by year end.
2021, and 2022 are critical years, and immunogen evolution as we prepare to bring two products to the market and further our innovative portfolio of transformative adcs for solid tumors and hematologic malignancies. We look forward to keeping you apprised of our progress as we generate clinical data and prepare for commercialization and wood.
That I will turn the call over to Anna to provide some additional color on our clinical programs Anna.
Thanks, Mark I'll start with Syria, while we have recently fully enrolled this study accrual was more variable relative to our prior experience due to a combination of factors, including COVID-19, as well as screening activity and some trials tapering in recent weeks as they anticipated imminent completion of enrollment.
Anna Berkenblit: While we have recently fully enrolled the study, accrual was more variable relative to our prior experience due to a combination of factors, including COVID-19, as well as screening activity in some trials tapering off in recent weeks as they anticipated imminent completion of enrollment. These factors contributed to a roughly six-week delay, which, as Mark mentioned, has shifted the timing for reporting top-line data for Saraya into the fourth quarter. The net effect has also moved our plan's submission of the BLA into the first quarter of 2022.
These factors contributed to a roughly six week delay, which as Mark mentioned has shifted the timing for reporting topline data for Sorel and into the fourth quarter and the net effect is also moved our planned submission of the BLA into the first quarter of 2022.
Anna Berkenblit: Despite Miratol having had an earlier start, COVID-19 has also affected site activation and patient accrual in this trial. While we initially expected top line data in the latter part of the first half of 2022, we now expect top line data in the third quarter of 2022, with a submission of the supplemental BLA for full approval planned by the end of 2020. Consisting the Suraya and Miritzol studies are the highest priority for the business, and we look forward to the opportunity to offer a new therapeutic option to patients living with ovarian cancer.
Despite mirasol, having had an earlier start COVID-19 has also affected site activation and patient accrual in this trial, while we initially expected topline data in the latter part of the first half of 2022, we now expect top line data in the third quarter of 2022 with the submission of the supplemental BLA.
For full approval plans by the end of 2020 two.
Getting the Syria, and Mirasol studies are the highest priority for the business and we look forward to the opportunity to offer a new therapeutic options for patients living with ovarian cancer.
With the continued uptake of PARP inhibitors as maintenance in the frontline and recurrent platinum sensitive setting there is an increasing population of ovarian cancer patients for whom and non platinum based regimen is needed.
Anna Berkenblit: With the continued uptake of PARP inhibitors as maintenance in the frontline and recurrent platinum sensitive settings, there is an increasing population of ovarian cancer patients for whom a non-platinum-based regimen is needed. The initial data we have for Mervatuximab monotherapy in heavily pre-treated platinum-sensitive patients are encouraging, and we have therefore submitted a protocol to the FDA for a single-arm trial in this population. Operational activities are underway to enable the initiation of the study in the second half of this year.
And the initial data we have for Murdo talks about monotherapy in heavily pretreated platinum sensitive patients are encouraging and we have therefore submitted a protocol to the FDA for a single arm trial and this population operational activities are underway to enable initiation of the study and the second half of this year.
To expand into earlier lines of ovarian cancer treatment. We are pursuing combination studies with Carboplatin and investigator sponsored trial and the Neo adjuvant setting is now underway at the University of Alabama with Doctor Rebecca Arent. This is our first opportunity to assess and <unk> in the upfront setting.
Anna Berkenblit: To expand into earlier lines of ovarian cancer treatment, we are pursuing combination studies with Carboplatin. An investigator-sponsored trial in the neoadjuvant setting is now underway at the University of Alabama with Dr. Rebecca Aran. This is our first opportunity to assess mervituximab in the upfront setting. Importantly, this trial will support our goal of incorporating folate receptor alpha testing at diagnosis as part of standard of care. In addition to this trial, we are also supporting a randomized study comparing mervituximab in combination with carvolatin to standard of care in recurrent platinum sensitive disease with Dr. Philip Harder in Germany. This trial is slated to start in the second half of the year.
Importantly, this trial will support our goal of incorporating folate receptor alpha testing a diagnosis of <unk>.
Part of standard of care.
In addition to this trial. We are also supporting a randomized study comparing <unk> in combination with Carboplatin to standard of care and recurrent platinum sensitive disease with Doctor Philipp Harter in Germany. This trial is slated to start and the second half of the year.
In addition to starting from trials in combination with Carboplatin and we're pleased to have mature data from our <unk> plus avastin cohort in recurrent ovarian cancer, regardless of platinum status and selected for a virtual oral presentation at <unk> and June Dr.
Dr. David O'malley from the Ohio State University, well present mature safety and efficacy data from our phase one b forward two cohort of <unk>, plus Avastin, which we believe will bring us a step closer to achieving our goal of establishing Margaret talked and that as the combination agent of choice in ovarian cancer and support.
Anna Berkenblit: In addition to starting these new trials in combination with carbo-platin, we are pleased to have mature data from our Mervitopsinab plus Avastin cohort in recurrent ovarian cancer, regardless of platinum status, selected for a virtual oral presentation at ASCO in June. Dr. Dave O'Malley from Ohio State University will present mature safety and efficacy data from our Phase 1B Forward 2 cohort of Mervituximab Plus of Aston, which we believe will bring us a step closer to achieving our goal of establishing Mervituximab as the combination agent of choice in ovarian cancer and supporting its use in earlier lines of therapy.
And its use in earlier lines of therapy.
In addition, two posters from our collaborators were accepted for presentation at <unk> data from an expansion cohort from the city of hope trial evaluating bavituximab in combination with Gemcitabine and patients with folate receptor alpha positive platinum resistant ovarian cancer, and a trial and progress poster.
And from Dana Farber evaluating Marvin talked for that in combination with <unk> and patients with advanced or recurrent microsatellite stable endometrial cancer.
Moving to IMG see 93, six hour Adam nine targeted ADC, our partners at Macrogenics identified Adam nine and known member of the matrix Matala protein Nathan disintegrate and family as an attractive target for ADC development because it is over expressed in multiple solid tumors, but many.
Anna Berkenblit: In addition, two posters from our collaborators were accepted for presentation at ASCO. Data from an expansion cohort from the City of Hope trial evaluating Mervatuximab in combination with gem cytobine in patients with folate receptor alpha positive platinum resistant ovarian cancer, and a trial in progress poster from Dana Farber evaluating Mervitoximab in combination with pembrylizumab in patients with advanced or recurrent, micro Moving to IMGC 936, our Adam 9 targeted ADC, Our partners at Macrogenics identified Adam 9, a known member of the Matrix Metalloprdeonase and Disintegring Family, as an attractive target for ADC development because it is overexpressed in multiple solid tumors but minimally expressed in normal tissue, and anti-adam-9 antibodies are efficiently internalized and degraded by tumor cell lines.
Emily expressed and normal tissue and anti Adam nine antibodies are efficiently internalize and degraded by tumor cell lines.
At ACR and April we presented data on Adam nine expression and solid tumors and evaluated the activity of IMG see 93, six and clinically relevant patient derived xenograft or pdx models with Adam nine expression similar to that observed in humans and solid tumors IMT.
<unk> and 93, six demonstrated compelling activity against a broad panel of pdx models, including non small cell lung triple negative breast and gastric and pancreatic cancers.
We are actively enrolling patients with solid tumors known to express Adam nine and a phase one dose escalation study and collecting tumor tissue for retrospective Adam nine expression assessment by Immunohistochemistry, and we're excited about the potential of this program and we look forward to presenting data at a major medical meeting likely in early 2000.
Anna Berkenblit: At AICR in April, we presented data on atom-9 expression in solid tumors and evaluated the activity of IMGC-936 in clinically relevant patient-derived xenographed or PDX models, with atom-9 expression similar to that observed in human solid tumors. IMGC-936 demonstrated compelling activity against a broad panel of PDX models, including non-small cell lung, triple negative breast, gastric, and pancreatic cancer We are actively enrolling patients with solid tumors known to express atom nine in a phase one dose escalation study and collecting tumor tissue for retrospective atom nine expression assessment by immunohistochemistry.
And 'twenty two.
With that I'll turn the call over to Susan to cover our financials and Susan.
Thanks, Anna for the first quarter of 2020, once we generated $15 $7 million and revenue nearly all of which came from noncash royalty revenue.
Operating expenses were $44 6 million comprised of $34 $4 million and research and development expenses compared with $27 4 million for the first quarter of 2020.
This increase was due to the greater year over year clinical development costs related to advancing our ceramic mirasol and M. D C and <unk> hundred six study and.
And greener external manufacturing costs.
G&A expenses were $10 2 million compared to $8 9 million for the first quarter of 2020, primarily due to increased professional fees and personnel cost and we ended the first quarter was $283 $1 million and cash and our financial guidance for 2021 for remains unchanged, we expect revenue to be between 65.
Susan L. Schiller: We're excited about the potential of this program, and we look forward to presenting data at a major medical meeting, likely in early 2022. With that, I'll turn the call over to Susan to cover our financials. Thanks, Anna.
Susan L. Schiller: For the first quarter of 2021, we generated $15.7 million in revenue, nearly all of which came from non-cash royalty revenue. Operating expenses were $44.6 million, comprised of $34.4 million of research and development expenses compared with $27.4 million for the first quarter of 2020. This increase was due to greater year-over-year clinical development costs related to advancing our Saraya, Miracol, and IMGC-936 studies and greater external manufacturing costs. GNA expenses were $10.2 million compared to $8.9 million for the first quarter of 2020, primarily due to increased professional fees and personnel costs.
And $75 million operating expenses to be between 202 hundred $10 million and cash and cash and equivalents at year end to be between 140 and $150 million. We expect our current cash to fund operations into the second half of 2022.
And look forward to what and what is ahead of us and with that I'll open the call for questions.
Thank you for sensors to ask a question you need to press star one on your telephone to withdraw your question press the pound key.
Your first question comes from John Newman of Canaccord. Your line is now open.
Hi, guys. Good morning, Thanks for all the updates and things.
And thanks for taking my question.
So mark I just wondered.
Now that the topline data for Surya.
Susan L. Schiller: We ended the first quarter with $283.1 million in cash. Our financial guidance for 2021 remains unchanged. We expect revenues to be between $65 and $75 million, operating expenses to be between $200 and $210 million, and cash and cash equivalents at your end to be between $140 and $150 million. We expect our current cash to fund operations into the second half of 2020.
It looks like it will surface and the fourth quarter of 'twenty one.
Might that allow you to give us a little bit more detail regarding the durability of responses when you present, the topline data. Thanks.
Yeah, and I'll ask Andrew to comment and further but the answer is yes, yes, that's exactly right John given the pace of enrollment over the entire course of the study, including the last few months, we anticipate that we will have not only top line or our data for the primary endpoint, but we will also have a reasonable estimate.
Duration of response.
Okay, great. Thank you and then.
And just one follow up question sort of on a different topic. So it's been a lot of activity recently with the FDA regarding.
Operator: We look forward to what is ahead of us, and with that, I'll open the call to: Thank you, presenters. To ask a question, you need to press star 1 on your telephone. To withdraw your question, press the Pound. Your first question comes from John Newman of Canada Horde. Your line is now open. Hi guys, good morning. Thanks for all the updates and thanks for taking my question. So, Mark, I just wondered, now that the top line data for Saraya looks like it will surface in the fourth quarter of 21, might that allow you to give us a little bit more detail regarding the durability of responses?
Accelerated approval.
Our understanding is that the agency actually views studies companies that have studies already running.
At the time and accelerated approval that would be confirmatory.
As a significant positive I just wanted to if you could comment on your thoughts there. It seems like if you've already got your confirmatory study up and running the agencies more comfortable just curious as to how you think about that thanks.
Yeah, maybe just at a high level I mean, that's in fact, the regulatory guidance. So when you talk to the FDA about and accelerated approval study. The first thing. They say to you is great, but we want to make sure you've completed enrollment and your confirmatory study at the time and regulatory actions. So that's formal guidance that they provide every.
Mark Joseph Enyedy: Responses when you present the top line data.
Anna Berkenblit: Yeah, and I'll ask Anna to comment any further, but the answer is yes. Yeah, that's exactly right, John. Given the pace of enrollment over the entire course of the study, including the last few months, we anticipate that we'll have not only top-lined ORR data for the primary endpoint, but we will also have a reasonable estimate of the duration of results.
Applicants so well.
And what happens and practices that not everybody achieves that objective, we expect to be right in that place and then you saw the.
<unk> on some of the accelerated approvals, which and I had the opportunity to to sit through and and I think we were very pleased with the overall disposition of the Odessa relative to some of these so called hanging hanging approval. So.
Mark Joseph Enyedy: Okay, great, thank you. I had just one follow-up question, sort of on a different topic. So there's been a lot of activity recently with the FDA regarding accelerated approval. Our understanding is that the agency actually views studies, companies that have studies already running.
Excellent great. Thank you.
Your next question comes from Mr. Michael Schmidt of Guggenheim sorry.
Mark Joseph Enyedy: at the time of accelerated approval, that would be confirmatory as a significant positive. I just wanted to ask you to comment on your thoughts there.
Your line is now open.
Hey, guys. Good morning, Thanks for taking my questions.
I had a question on.
And both ongoing study soraya and Mirasol and.
Mark Joseph Enyedy: It seems like if you've already got your confirmatory study up and running, the agency's going to
And perhaps related to your comments about the increased use of PARP inhibitors and recent years, just wondering how you think and.
Mark Joseph Enyedy: He's more comfortable; I'm just curious as to how you think about that. Thanks.
Mark Joseph Enyedy: Maybe just at a high level. That's, in fact, the regulatory guidance. So when you talk to the FDA about an accelerated approval study, the first thing they say to you is, great, but we want to make sure you've completed enrollment in your confirmatory study at the time of regulatory action. So that's formal guidance that they provide every applicant. But what happens in practice is that not everybody achieves that objective. We expect to be right in that place.
<unk> might perform in and are more heavily pretreated patient population with PARP inhibitors specifically.
Sure Michael So parts are being incorporated into frontline maintenance for recurrent platinum sensitive maintenance therapy, and we've absolutely seen with each trial that we have enrolled over the past six years and increasing percentage of patients receiving a prior PARP and what I can tell you.
As we've looked across each study and <unk> and have really has nice activity regardless of prior PARP use it or not and you know that makes sense given net biologically theres no reason to expect any kind of cross resistance between our tubular and directed payload and and a PARP inhibitor, which impairs the ability to repair DNA damage.
Mark Joseph Enyedy: And then, you know, you saw the ODAC on some of the accelerated approvals which Anna had the opportunity to sit through. And I think we were very pleased with the overall disposition of the ODAC relative to some of these so-called hanging approvals.
Operator: Excellent. Great. Thank you.
Operator: Your next question comes from Mr. Michael Smith of Guggenheim. Sorry, your line is now open. Hey guys, good morning, thanks for taking my questions. I had a question about both ongoing studies, Saria Mirosol, and perhaps related to your comments about the increased use of PARP inhibitors in recent years, just wondering how you think myrvitizum might perform in a more heavily-treated patient population.
Makes sense and then a question on your new single arm study in recurrent platinum sensitive patients. Just wondering if you could help us maybe outline just over that the size of the study potential timelines and also the size of the commercial opportunity, perhaps relative to the <unk> patient population.
Anna Berkenblit: patient population with PARP inhibitors specifically.
Yeah. So we have designed this study of single agent and <unk> in patients with recurrent later line platinum sensitive disease to really address and increasing unmet need with a larger population of patients who technically are still platinum sensitive by that rather arbitrary definition of recurring greater than <unk>.
Anna Berkenblit: Sure, Michael. So, PARPs are being incorporated into frontline maintenance or recurrent platinum sensitive maintenance therapy. And we've absolutely seen, with each trial that we have enrolled over the past six years, an increasing percentage of patients receiving prior PARP. And what I can tell you is we've looked across each study, and MERVituxemab really has nice activity, regardless of whether prior PARP use is present or not. And, you know, that makes sense, given that biologically there's no reason to expect any kind of cross-resistance between our tubule and directed payload and a PARP inhibitor which impairs the ability to repair DNA damage.
Six months after the last dose of platinum, but we know they're at high risk for allergic reactions or their their bone marrow, maybe a little bit tired and they need other well tolerated active therapies. So from our phase one study way back we had a handful of patients for more than that actually we had ah patients enrolled in a couple of our cohorts.
And the biopsy cohort and then other.
Cohort with three to four prior lines of therapy, who indeed had platinum sensitive disease and we when we look at those patients the activity of <unk> and that looks quite nice and so that has supported the study that we have just with just a bit and the protocol to FDA and.
Anna Berkenblit: And then a question on your new single-arm study in recurrent platinum-sensitive patients. Just wondering if you could help us maybe outline just sort of the size of the study, potential timelines, and also the size of the commercial opportunity, perhaps, relative to the Soraya patient population. Yeah, so we've designed this study of single agent mervitoxamab in patients with recurrent later-line platinum-sensitive disease to really address an increasing unmet need with a larger population of patients who technically are still platinum sensitive by that rather arbitrary definition of recurring greater than six months after the last dose of platinum, but we know they're at high risk for allergic reactions or their bone marrow may be a little bit tired, and they need other well-collarated active therapy.
You can think of it basically is a two stage design study and so once we are ready to open the study will share more details.
Michael just in terms of the market opportunity or DRG data tell us that third line platinum sensitive patients are about 2000 patients obviously, we're targeting and only a subset of those folks.
And evolving segment of the marketplace. So we won't get all 2000 and of those patients and.
And it will depend.
To a large degree on the efficacy that we generate relative to what you see with platinum based combinations.
Anna Berkenblit: So from our phase one study way back, we had a handful of patients, well more than that actually. We had patients enrolled in a couple of our cohorts, the biopsy cohort, and then the cohort with three to four prior lines of therapy who indeed had platinum sensitive disease.
In that setting and the data there are actually a little bit scarce, but.
Based on what we've seen preliminarily, we think.
Mirv monotherapy can compete quite effectively there both in terms of efficacy, but equally important.
Anna Berkenblit: And when we look at those patients, the activity of myrvituxinab looks quite nice, and so that has supported the study that we have just submitted the protocol to FDA. You can think of it basically as a two-stage design study.
Our ability and to some degree convenience as well.
Okay, Great and then I know you did speak about a few new investigator sponsored studies and I was just wondering if you could perhaps speak to your evolving thinking about.
Anna Berkenblit: And so, you know, once we are ready to open the study, we'll share more detail. Yeah, Michael, just in terms of the market opportunity, our DRG data tell us that third-line platinum-sensitive patients are about 2,000 patients. Obviously, we're targeting only a subset of those folks, but it's an evolving segment of the marketplace. So, you know, we won't get all 2,000 of those patients, and it will depend to a large degree on the efficacy that we generate relative to what you see. with platinum-based combinations in that setting, and the data there are actually a little bit scarce.
Potentially supporting company sponsored studies and earlier stage patients.
Sure so.
And I mentioned, we're really excited about this and neo adjuvant study.
A study being conducted by a doctor are and let out of UAV for the reasons that you described one being in the frontline setting having.
Having access to assess essentially fresh tumor tissue and.
And being able to assess fully receptor alpha levels and and those patients. So I think we're excited about that and.
Mark Joseph Enyedy: But based on what we've seen preliminarily, we think Merv Monotherapy can compete quite effectively there, both in terms of efficacy, but equally important, tolerability, and to some degree, convenience as well. Okay, great. And then I know you did speak about a few new investigator-sponsored studies. I was just wondering if you could perhaps speak to your evolving thinking about potentially supporting company-sponsored studies in an earlier stage. Sure, so as Anna mentioned, we're really excited about this neoadjuvant study being conducted by Dr. Aaron led out of UAB for the reasons that she described. One, being in the front-line setting, you know, having access to, you know, essentially fresh tumor tissue, and being able to fully assess receptor alpha levels in those patients. So I think we're excited about that.
And then.
And we're dealing with Philip harder and the age Yo is you know a randomized controlled study of upwards of 140 patients. So we expect to have a pretty strong.
Strong signal there in terms of you know a direct comparison against the standard of care and so we are evaluating our company sponsored effort in that setting and working through the details of the design as we speak.
Great. Thanks, so much.
Yeah.
Your next question comes from bars.
Boris Beaker of COVID-19.
And is now open.
Hi, good morning.
I just wanted to probe maybe initially in terms of the delay and Missouri study how is COVID-19 COVID-19 impacting ovarian cancer I'm just trying to understand since you aren't relying on new patient diagnosis. So it shouldnt all the patients for the survey of trial already be kind of diagnosed and and the system.
Mark Joseph Enyedy: And then, you know, the study we're doing with Philip Harder and the AGO is, you know, a randomized controlled study of upwards of 140 patients. So we expect to have a pretty strong signal there in terms of, you know, direct comparison against the standard of care. And so we are evaluating a company-sponsored effort in that setting and working through the details of the design. Great. Thanks so much.
Yeah, and I give you a little more color, but to start with.
We're working with <unk> as our CRO and what they tell us is that and more than 70% of their studies, which include other ovarian cancer studies, they saw a decline and accrual rates and the first quarter of this year and comparison to Q4 and.
Operator: Your next question comes from Boris Speaker of Colin. So your lines are now open. Good morning.
Last year, so there was a COVID-19 impact.
Operator: I just wanted to probe, maybe initially, in terms of the delay in the serraea study. How is COVID-im impacting ovarian cancer? I'm just trying to understand since you aren't relying on new patients' diagnosis, so shouldn't all the patients for the syria trial already be kind of diagnosed and in the systems? Yeah, I'll let Anna give you a little more color, but to start with, we're working with Icubia as our CRO, and what they tell us is that in more than 70% of their studies, which include other ovarian cancer studies, they saw a decline in accrual rates in the first quarter of this year in comparison to Q4 of last year.
Part of what we see as well is there is some variability. So we're normally we're accustomed to seeing a sort.
Sort of.
And a direct correlation between the number of sites that we have active and our accruals throughout the study and what we've seen and in particular was acute during the early spring with some variability and.
And and accrual which.
And and the CRO attributed to to COVID-19.
Got it and it's interesting.
I guess for my second question is on V. P D sand and just again, what do you need to show and that study for it to be considered successful.
Yeah. So the P. P D C and frontline cohort to support full approval is enrolling and we've aligned with SBA debt.
Mark Joseph Enyedy: So there was a COVID impact. Part of what we see as well is, you know, there's some variability. So we're normally, we're accustomed to seeing, you know, sort of a direct correlation between the number of sites that we have active and are monitored throughout the study. And what we've seen, in particular, which was acute during the early spring, was some variability in accrual, which, you know, we and the CRO attribute to COVID. Josh. I guess my second question is about the PDCN. Reminds you again what you need to do.
And it will include up to 20 frontline patients these are patients.
Who may have had local therapy surgery or radiation, but have not had any systemic therapy and so the study is designed to rule out a CR slashed CRC or clinical CR rate of 10 per cent.
Great. Thank you very much for taking my questions.
Sure.
Mark Joseph Enyedy: Again, what do you need to show in that study for it to be considered successful?
Your next question comes from and the shy of William Blair. Your line is now open.
Anna Berkenblit: Yeah, so the BPDCN frontline cohort to support full approval is enrolling, and we've aligned with FDA that it will include up to 20 frontline patients. These are patients who may have had local therapy, surgery, or radiation, but have not had any systemic therapy. And so the study is designed to rule out a CR slash CRC or clinical CR rate of 10%.
Yes.
Andy.
Andy are you there.
Yeah.
Operator, I think we probably should go to the next question.
Hello, Andy.
Yeah.
Yes.
Why don't we take the next question.
Alright next question comes from Jessica Fye of JP Morgan.
Anna Berkenblit: Great. Thank you very much for taking my question. Your next question comes from Andy Shai of William Blair. Your line's now open. Andy?
Yeah.
Yeah.
Yeah.
Jeff.
Operator can you check the communications here because it seems like we're missing two people already.
Operator: Andy, are you there? Operator, I think we probably should go to the next question. Hello, Andy.
Yes, just give me a second here Sir.
Operator: Why don't we take the next question? All right, the next question comes from Jessica Fye of JP Morgan. Operator, can you check the communications here? Because it seems like we're missing two people already.
Hello, Ms. Jessica Your line is now open.
Hello can you guys hear me.
Sounds like that's Andy.
No I'm sorry, I'm sorry. This is this is a look brennan on for Jess.
Operator: Give me a second here, sir. Hello, Ms. Jessica. Your line is now open. Hello, can you guys hear me? Sounds like that to Andy. No, sorry, sorry, this is Luke Brennan on for Jess. Oh, great. Okay, hey, Luke, good.
Okay, Hey look good.
And your questions. This morning.
So with the FDA feedback that you guys are.
Anna Berkenblit: Yeah, thanks for the questions this morning. So with the FDA feedback that you guys expect on the protocol, is the goal for the FDA to sign off on that single-arm trial in later-line platinum sensitive patients as the basis for approval? No, so basically, when we are getting ready to start a study, we submit the protocol to FDA, and there's a 30-day waiting period.
Specced on the other protocol as the goal for the FDA to sign off on that single arm trial, and later line platinum sensitive patients as the basis for approval.
No. So basically when we are getting ready to start a study we submit the protocol to FDA, There's a 30 day waiting period and so there's a good chance actually that we may not hear anything back from FDA. We of course are waiting so that if they do have feedback we will have the opportunity to incorporate it before.
Anna Berkenblit: And so, you know, there's a good chance that we may not actually hear back from FDA. We, of course, are waiting so that if they do have feedback, we'll have the opportunity to incorporate it before we enroll the first patient in the study. Okay, so could you guys just get a little bit more color on the dynamics of starting, you know, both the combo therapy and the monotherapy trials in the same setting?
We enrolled the first patient in this study.
Okay. So I guess could you guys just give a little bit more color on the dynamics between starting you know both the combo.
Therapy, and the monotherapy trials and and the same setting.
Anna Berkenblit: So the monotherapy study is in patients with two or more prior lines of platinum therapy. So with at least two, so two to three prior lines of platinum therapy. And that really is a population that, again, is still technically platinum sensitive, but needs something else. We are also planning to allow patients who've had one prior line of platinum-based therapy but who had a hypersensitivity reaction that would make them inappropriate for further platinum therapy.
So the mono therapy study is in patients with two or more prior lines.
For platinum.
So with at least two so two to three prior lines of platinum therapy, and that really is a population that again, its still technically platinum sensitive but needs need something else. We are also planning to allow patients who've had one prior line of platinum based therapy, but who had.
Hypersensitivity reactions that would make them inappropriate for further platinum. So that is a setting again, we think will be increasing in frequency just because there are more patients with later line platinum sensitive disease.
Anna Berkenblit: So that is a setting, again, we think will be increasing in frequency just because there are more patients with later Lyme Platinum sensitive disease. Our carboplatin combinations, one is the neoadjuvant setting, so that's up front. And so those are patients who have not had any therapy before.
And Carboplatin combinations, one as the neo adjuvant setting so that's upfront.
And so those are patients who have not had any therapy before and we're very excited about that because that'll be our first opportunity to study <unk> and that in untreated patients.
Anna Berkenblit: We're very excited about that because that will be our first opportunity to study myveratuximab in untreated patients. And then the randomized phase two study, the IST that Mark mentioned being done in Germany, that's going to be a randomized study of myrbitoxinab plus carboplatin versus standard, basically platinum-based doublets with standard of care maintenance therapy as appropriate for each patient. Okay, thank you very Thank you. Your next question comes from Andy Shai of William Blair. Sir, your lines are now open. Oh, great. Thanks for taking my question. Sorry about the technical difficulty.
And then the randomized.
<unk> to study the ISP that Mark mentioned being done and Germany, that's gonna be a randomized study of <unk> plus carboplatin versus standard basically platinum based doublet with standard of care maintenance therapy as appropriate for each patient.
Yeah.
Yeah.
Okay.
Thank you very much.
Thank you. Your next question comes from Andy shy of William Blair Sir your.
Your line is now open.
Oh, great. Thanks for taking my question and sorry about the technical difficulty.
Operator: And congratulations on the Sora enrollment completion. I have two questions regarding the kind of label expansion opportunities. It might be a little bit... early to answer this question, but the new kind of platinum agnostic study that you're proposing, do you foresee Marisol potentially serving as a confirmatory trial for that opportunity? And also, Mark, I'm just wondering if you could educate us on the compendia listing process. I'm just curious about how much interaction that you might have back and forth, kind of relative to the regular FDA review.
And congratulations on this aura.
Enrollment completion.
I have two questions regarding kind of the label expansion opportunities.
Might be a little bit.
Early to answer this question, but the new.
Kind of platinum agnostic study that Youre proposing do you foresee marisol potentially serving as a confirmatory trial for that opportunity.
And also Mark I'm, just wondering if you can educate.
Educators on the compendium listing process I'm, just curious about how much interaction that you might have back and forth.
Relative to the regular FDA review process.
Anna Berkenblit: review process
Anna Berkenblit: Yeah, so I'll start. So the new study that we are getting ready to start later this year of Merv Monotherapy and recurrent platinum-sensitive disease, it's not exactly platinum agnostic, Andy. These are patients who technically are still platinum sensitive, having recurred greater than six months after the last dose of platinum. Again, these patients need other non-platinum-based options. So that study is slated to get going later this year. I think it's unlikely that Mirosol would serve as a confirmatory study for that.
Yeah, So I'll start.
So the new study that we are getting ready to start later this year of mirv monotherapy and recurrent platinum sensitive disease, and it's not exactly platinum agnostic Andy These are patients who.
Technically are still platinum sensitive, having recurred greater than six months from the last dose of platinum.
And these patients need other non platinum based options.
So that study is slated to get going later this year I think it's unlikely that mirasol would serve as a confirmatory study for that and Mirasol will likely readout prior to us completing the.
Anna Berkenblit: Mirosol will likely read out prior to us completing the study that we're planning to start later this year. Moving to Compendia listing, typically, you need two separate studies to support compendial listing. And I think the best example for us right now is our combination data with myrbituxamab plus Bevacizumab. You may recall we have one cohort specifically in platinum-resistant disease that was presented at ASCO. A couple years ago now, twice.
The study that we're planning to start later this year moving.
Moving to compendium listing typically you need two separate studies.
To support compendium listing and I think the Best example for US right now is our combination data with <unk> plus Bevacizumab you may recall, we have one cohort specifically and platinum resistant disease that was presented at <unk> a couple of years ago now twice.
Anna Berkenblit: And then we have our second cohort in Platin Magnostics, so a broader population of patients with recurrent ovarian cancer. That is another cohort of 60 patients. And again, we anticipate that the safety and efficacy data from those two separate cohorts will support compendia listing. It may very well be the case that with Mervitoxinab plus carboplatin, based on the data we're planning to generate for that doublet, we could also gain compendia listing in advance of a form from a label expansion from a registration trial. Got it, that's very helpful. And then the last question.
And then we have our second cohort and platinum agnostic. So a broader population of patients with recurrent ovarian cancer that is another cohort 60 patients and again, we anticipate that the safety and efficacy data from those two separate cohorts will support compendium listing.
It may very well be the case that with nerve Rituximab plus carboplatin based on the data and we're planning to generate for that doublet. We could also gained compendium listing in advance of the formal.
Label expansion from a registration trial.
Got it that's very helpful and then last question.
Anna Berkenblit: Anna, I'm just curious about the investigators' learning curve and using Merv in the context of a clinical trial. So specifically, I'm just wondering if you expect to see some sort of safety or efficacy benefit from clinical sites that participated in Forward 1 and are concurrently recruiting patients for Soraya and Marisol. Yeah, so we have a wealth of data from the Forward One study, Andy, and it gives me a lot of comfort because, you know, with Forward 1, we went from, you know, 10 or 15 Phase 1 sites to over 100 sites for the Phase 3 study.
And.
And I'm just curious about your thoughts on investigators learning curve and using Merv in the context of a clinical trial.
So specifically I'm just wondering if you expect to see some sort of.
Safety or efficacy benefit from clinical sites that.
Had participated and forward one and are currently recruiting patients for some Ryan Marisol.
Yeah. So we have a wealth of data from the forward one study Andy and it gives me a lot of comfort because you may recall with forward. One we went from you know 10 or 15 phase one sites to over 100 sites for the Phase III study and the safety profile of <unk> and forward one and.
Anna Berkenblit: And the safety profile of Mervitoxamad in Forward 1, in 243 patients who were dosed with Mervatuxinad looked quite good. For me, the bellwether there is that only one of those 243 patients discontinued due to blurred vision. And that tells you that new sites, new investigators were very well informed and educated. We really did focus on that in Forward 1, like we did in Suraya and Mirosol, to manage patients appropriately on marvatumab.
And 243 patients who were dosed with <unk> and that looked quite good for me. The bellwether. There is that only one of those 243 patients discontinued due to blurred vision and that tells you that new sites new investigators.
And we're very well and.
<unk> formed and educated we really did focus on that and forward. One like we are in Soraya and mirasol to manage patients appropriately on Mark <unk> and I would expect no different and the Soraya and Mirasol studies.
I will say, you're absolutely right we picked.
Anna Berkenblit: And I would expect no different in the Suraya and Mirosol studies. I will say you're absolutely right. We picked the best sites from Forward 1 to be included in Syraea, and I think that was a really important strategy for us, both in terms of getting accrual completed and ensuring that we have optimal data and
The best sites from forward one to be included in Syria.
And I think that was a really important strategy for us both in terms of getting accrual completed and ensuring that we have optimal data integrity.
I see that makes total sense. Thank you very much.
Thank you. Your next question comes from.
Anna Berkenblit: That makes total sense. Thank you very much.
Operator: Thank you. Your next question comes from Sayamakula, Ramana, from H.C. Wayne Rutt.
Yeah, Mark cooler Romana.
From each C. Wainwright your line is now open.
Operator: Your line is now open. Thank you. This is RK from Hitzewen.
Thank you Ms RK from Pennsylvania.
Mark Joseph Enyedy: A couple of quick questions. Mark, as you embark on the regulatory strategy with, you know, the two studies, Soria for accelerated approval, and Mirosol for full approval, you're also trying to evergreen MyvertoxMab, you know, with various combination regiments being tested in the Forward program. So what gives you the confidence and comfort to put a lot of resources behind this molecule when some could think this is a big, bold step with no approval yet in any indication?
Couple of quick questions.
Mark as you.
Embark day regulatory strategy.
And over the two studies for for accelerated approval and Mirasol for full approval.
You're also trying to evergreen.
From a rituximab.
They just combination regimens and can start and the forward.
And Hmm.
So what gives you the confidence.
And comfort.
To put a lot of resources behind this molecule.
And then some good thing for us.
Big bold steps, but no.
No approval, yet and any indication.
Mark Joseph Enyedy: So, the first thing is, you know, we are looking at a relatively accelerated approval timeline for Myrotoxamab with the BLA submission in the first quarter of 2022. We have fast track designation for this product, and I think we could reasonably expect priority review, which gives us a relatively short window for review and approval. Our objective then is to ensure that Mervatoxyab can be used as widely as the data warrant.
Yes.
So.
For the first thing is we are looking at a relatively accelerated approval timeline for Marvel talks and map with the BLA submission and the first quarter of 2022 and fast track designation for this product and I think we could reasonably expect.
Priority review, which gives us a relatively short window for review.
And approval. Our objective then is to ensure that mark.
And it talks a mab or it can be used as widely as the day to warrant and so this is why we placed an emphasis on the companion listing there could be available to us at the time of our initial approval and so the goal here is really to accelerate the penetration.
Mark Joseph Enyedy: And so this is why we place some emphasis on the compendia listings that could be available to us at the time of initial approval. And so, you know, the goal here is really to accelerate the penetration of MIRV both within its label and then as physicians decide at their own discretion to use the product consistent with the compendial listing that that's available to them so that reimbursement would not be a barrier to that. So we don't view that as a high-risk strategy, particularly in light of the data that we've already generated.
And mirv, both within its label and then as physician decide and their own discretion and use the product consistent with companion listing that that's available to them. So that reimbursement would not be a barrier to that so I don't we don't view that as a high risk strategy, particularly in light of the.
Data that we've already generated so.
Mark Joseph Enyedy: So, you know, there's no further work really needed other than generating the actual publications from Mervyn in combination with Avastin. And then, you know, when we look at the data that we've generated to date, particularly in some of these earlier line patients from some of our phase one work, the data looked quite compelling in terms of the response rates that we generated relative to current benchmarks. And so you may remember from, you know, it's a small end, but our initial data in patients with high levels of expression was an 80% response rate and progression-progressive-free survival was 15 months.
No further work really needed and other than generating the actual publications.
For Mirv and combination with a vast and and then when we look at the data that we've generated.
To date and <unk>.
Particularly in some of these earlier line patients from some of our you know phase one work that day will look quite compelling in terms of the response rates that we've generated relative to current benchmarks and so you may remember from.
Small and but our initial cargo data and patients with high levels of expression was and 80% response rate and progression free survival of 15 months. If you look at our combination and our triplets that were published at ESMO. Similarly, very high response rates and so you know our goal.
Mark Joseph Enyedy: If you look at our combination, our tripletes, our triplet, we look at our patient, we're a patient, we're a response rate that was published at ESMO, similarly, very high response rates. And so, you know, our goals here are really to leverage that. So, I mean, we have a lot of conviction that this drug should be the combination agent of choice for both Vastin and Carbo. And, you know, I think it's our responsibility to accelerate that work. And part of that's just driven by, you know, our overall conviction about initial approval and the availability of this drug in 2022. Thank you, on IMG at 632.
And here are really two lever that so I mean, we have a lot of conviction that this drug should be the combination agent of choice for both the vast and and cargo.
And I think it's our responsibility to accelerate that work and then and part of Thats driven by.
Our overall conviction about and initial approval and the availability of this drug in 2020 two.
Perfect. Thank you.
And on <unk> and 632.
Mark Joseph Enyedy: You know, while certainly this drug could target multiple liquid tumors, you know, your initial focus is on BPDCN followed by AML. So do you, or should we ask, you know, following what you're trying to do with broadening, broadening the profile of 632 in additional liquid tumors, what would be your plans for that? Right, so, I mean, high level with our portfolio, our ethos in terms of pursuing development, is fast-to-market strategies where we can use single-arm studies to support accelerated approval in the case of myrbitoxyab. In the case of 632 and BPDCA, we'll have full approval.
And why.
Suddenly this.
Drug and also can target multiple liquid tumors and R E D.
Our initial focus.
And is on BP D C and followed by AML.
So do you.
Or should we expect.
And our following what you're trying to do it.
Broadening.
Broadening the profile of 632, and our additional liquid tumors.
And what would be your plans on that.
Right. So I mean high level with our portfolio our ethos in terms of pursuing development is fast to market strategies, where we can use single arm studies to support accelerated approval and in cases and remember it talks about and in the case of 632, Mvpds, Yeah, and we will have full approval, but you may recall that we got.
Mark Joseph Enyedy: But you may recall that we got breakthrough therapy designation for that program in relapse-refractory patients and aligned with FDA around an additional cohort in front-line patients, which we are pursuing as we speak. But yes, our goal is to CD-123 is expressed across a range of hematologic malignancies, and our principal foray there has been in acute myeloid leukemia. Initially, it was monotherapy where we saw some interesting activity, but clearly, the benchmark has been set in that disease with the combination of acesididine and venetaclachs.
Breakthrough therapy designation.
And for that program, and relapsed refractory and aligned with FDA around and additional cohort and frontline patients, which we are pursuing as we speak.
But yes, our goal is to CD 120, threes and expressed across a range and hematologic malignancies and our principal for a there has been and acute myeloid leukemia initially as monotherapy, where we saw some interesting activity.
But clearly the bench Mark has been set and that disease with the combination of <unk> Decitabine and <unk>.
And who need a class and so our goal was to integrate and 632 into that doublet to create and looking at both doublets and most importantly, I think a triplet.
Mark Joseph Enyedy: So the goal was to integrate 632 into that doublet to create, you know, looking at both doublets and, most importantly, I think, a triplet to see whether we can generate response rates and duration of response that would make that regimen competitive. We're working through that as we speak, and we expect to have data at Ash at the end of the year with the triplet. But yes, that represents a, you know, significant potential expansion beyond the BPDCN label.
And see whether we can generate response rates and duration of response that would make that regimen and competitive we're working through that as we speak and we expect to have data at ash at the end of the year.
With with the triplet, but yes that represents a significant potential expansion beyond the BDC and label.
Mark Joseph Enyedy: Thank you, Mark. Thanks for taking my questions. Your next question comes from Kenan McKay of RBC Capital Markets. Your line is now open. Hey, thanks, too.
Thank you Mark Thanks for taking my questions sure.
Your next questions come from homes from Keenan Mckay of RBC capital markets. Your line is now open.
Operator: Hey, thanks. Thank you. Thank you.
Okay Great question.
Yeah.
Operator: This is my name here. I'm in time. I'm in a long spot in a meeting. I, N.D. is, I'm ready to be soft here in this. And they're going to be soft here in this.
Yeah.
Okay.
Great.
Okay.
Hey, Kevin.
Operator: Kenan, you're breaking up substantially. I couldn't; I only could make out about every third word.
Sure.
Ken and Youre, breaking up substantially I couldnt I only could make out about every third word.
Operator: You don't operate off at one people for not because that have merged.
Oh for sure.
Mark.
Yes.
Good day.
Operator: I did not understand that, Kenan, my apology. So maybe we can follow up, um,
Got it Mark.
And I did not get that Ken and I apologies. So maybe we can follow up.
Offline.
Operator: Parkes closed. Sorry, yep. Operator, for the next question.
Perfect.
Sorry for me up.
Operator next question.
Okay.
Operator: The next question comes from Joe Katanzaro of Pfeiffer Sandler. Your line is now open. Hey guys, thanks so much for taking my questions.
Next question comes from Joe Catanzaro of Piper Sandler Your line is now open.
Hey, guys. Thanks, so much for taking my questions. Maybe just one quick one for me to follow up on the and the single arm monotherapy trial and platinum sensitive patients. So and you had mentioned that way back in the phase one experience you actually had enrolled platinum sensitive patients I'm wondering if maybe you could elaborate a little bit more and on what exactly you saw and those patients and.
Operator: Maybe just one quick one for me to follow up on the single arm monotherapy trial and platinum sensitive patients. So, Anna, you had mentioned that way back in the phase one experience, you actually had enrolled platinum sensitive patients. I'm wondering if maybe you could elaborate a little bit more on what exactly you saw in those patients and specifically how it relates to folate receptor expression levels and the various cutoffs that you utilized back in that phase one trial.
And specifically how it relates to folate receptor.
Expression levels and the various cutoffs debt you utilized back and that phase one trial.
Anna Berkenblit: Phase 1 trial.
Anna Berkenblit: Yeah, sure, Joe. So as I mentioned, we had the biopsy cohort, which was silent on platinum status, and we also had what we affectionately called the eyedrop cohort in patients with three to four prior lines of therapy regardless of platinum status. You know, that was back in the day when Olaparib got its accelerated approval initially as treatment in patients with three to four prior lines of therapy, so we were sort of following that model.
Yeah sure Joe So and so as I mentioned, we had the biopsy cohort, which was silent on platinum status and we also had.
What we affectionately called the eye drop cohort in patients with three to four prior lines of therapy, regardless of platinum status, you know that was back and the day when a elaborate got it for accelerated approval initially as treatment in patients with three to four price. So we were sort of following that model as a result, even though our intent at the time was.
Anna Berkenblit: As a result, even though we were, our intent at the time was really not to focus on platinum sensitive disease, we did enroll enough patients across those two cohorts with later-line disease. I mean, these were patients with three prior lines of therapy and had a very nice, the FRA Alpha High patients had a very nice response. So, you know, these are internal data that we're using to support the design of the single-arm study that is on track to get open later this year. And, you know, we're very pleased with the data that we have from the system.
Really not to focus on platinum sensitive disease.
We did enroll enough patients that across those two cohorts with later and Lyme disease and initial patients with three prior lines of therapy.
And had a very nice day fr Alpha high patients had a very nice response.
And so.
And these are our internal data that we're using to support.
The design of the single arm study that is on track to open later this year.
And you know, we're very pleased with the data that we have from and from that phase one experience and so again, we're going to have a it's basically a two stage design and you know once once the study is up and running we can sort of talk more about the actual design statistics and timeline.
Anna Berkenblit: from that phase one experience, and so again, we're going to have a it's basically a two-stage design, and you know, once the studies are up and running, we can sort of talk more about the actual design statistics and timeline. Okay, got it. Thanks for taking my question. The next question comes from Kelly Shai of Jeffries. Your line is now open.
Okay got it thanks for taking my question.
Sure.
Next question.
And <unk> comes from Kelly shy of Jefferies. Your line is now open.
Operator: Thank you for taking my questions. I have one question from one of your early face trials. So the 936, I'm targeting ADC, you recently showed a nice proof of concept data in Asia. I'm wondering, do you think Adam 9 is predictive for treatment efficacy based on its biology? And also, are we going to screen patients after the escalation phase? If there is any companion diagnostic development ongoing at immunogen. And also, lastly, what has been learned from the previous item targeting antibodies, if there are any?
And I. Thank you for taking my questions I have one question from a one off deal or anything.
Trials are.
And so my three six and I'm targeting ADC you recently showed a nice proof of concept data at ACR and wondering do you think at EM and predictive for the freedom and advocacy and based on its biology and I'm sorry go ahead and to screen patients after dose escalation phase and.
And there are any and companion diagnostic a developmental and colleague and telling me antigen.
And also lastly, what has been and learning from previous Ah, Adam targeting and have a day. So yeah. Thank you.
Operator: Thank you. Sure, Adam Nine is a member of the Matrix, Metallop Proteinase, and Dysinthagrin family, and I certainly remember the days when we were targeting MMPs, Matrix Metacototroteinases, to try to prevent metastasis and invasion. And those mechanistically oriented approaches really didn't turn out so well for cancer patients.
Sure So Adam nine as a member of the matrix and the talent proteinase and disintegrated family and I certainly remember the days that we were targeting and then pes matrix and <unk>.
And to try to prevent.
Metastasis and invasion and those mechanistically oriented approaches really didn't turn out so well for cancer patients. However.
Anna Berkenblit: However, Adam 9 is really, we're using it as a zip code to target our very potent next generation mechanizenoid toxic payload to tumor cells and to spare normal tissue. So it's really not, I wouldn't think of it as a biological mechanistic rationale, but really more targeting our toxic payload to tumor cells. In terms of your next question around screening, we are generating data now in phase one dose escalation to inform whether or not we will need to select patients for atom nine expression or not.
However, Adam nine is really we're using it as a ZIP code to target are very potent next generation maintains annoyed toxic payload to tumor cells and to spare normal tissue. So it's really not I wouldnt think of it as a biological mechanistic rationale, but really more.
For targeting our toxic payload to the tumor cells.
In terms of your next question around and screening.
We are generating data now in phase, one dose escalation to inform whether or not and we will need to select patients for Adam nine expression or not as you saw and our ACR poster out of nine is actually reasonably highly expressed across a broad range of tumors, but we will be poised.
Anna Berkenblit: As you saw in our AACR poster, atom nine is actually reasonably highly expressed across a broad range of tumors. But we will be poised to select patients by atom nine expression and develop a CDX with a cutoff, a validated cutoff if needed. And, you know, right now we're doing all of this. The spade work to get us to that point, if needed.
<unk> to select patients by Adam nine expression and develop a C. Dx with a cutoff of validated cutoff if needed and you know right now we're doing all of the spade work to get us to that point if needed.
Anna Berkenblit: And then your last question around learnings from prior atom nine targeted antibodies. I really can't speak to that because this is a novel target. Necrogenics brought the target and their antibody to us, and collaboratively, we've developed this ADC. So we're really excited.
And then your last question around learnings from prior Adam nine targeted antibodies I really can't speak to that because this is a novel target macrogenics brought the target and their antibody to us.
And collaboratively we've developed this ADC. So we're really excited the phase one dose escalation trial is enrolling and we look forward to sharing data in early 2022.
Anna Berkenblit: The phase one dose escalation trial is enrolling, and we look forward to sharing data in early 2020. Thank you very much. It's very informed.
And I think it all right. Thank you very much and Marion from do you also have another a quick question regarding the upcoming.
Anna Berkenblit: I also have another quick question regarding the upcoming ESCO update regarding the Merv-Avvvvastin combo in platinum agnostic patients. I just wonder what the estimated ORL and the PFS achieved by standard of care are in this folate receptor alpha-high subpopulation. Or put another way, I should ask, what should be the benchmark for this selective patient population? Thank you. Yeah, so there are two separate populations, if you will, platinum resistant and recurrent platinum sensitive.
And I'll come and ask her update.
For Marvell and <unk>.
I think humble in the platinum agnostic patients I just wonder what is the estimated oh are and the PFS and achieved by standard of care in these sniffle folate receptor Alpha high some population.
And that OE I should ask him what should it be the benchmark for all day so for now.
And the Ah patient population. Thank you.
Yeah. So there's two separate populations, if you will platinum resistant and and recurrent platinum sensitive in unselected patients.
Anna Berkenblit: In unselected patients with platinum-resistant disease, I would point you to the Aurelia study that looked at Avastin plus chemotherapy, which had a 27 or 28% confirmed response rate and a medium TFS of 6.7 months. They did not assess folate receptor alpha in orelia, so I don't know how the FRA-A-A high patients did, but what I can tell you is there are studies that have been published, mostly retrospective Alpha is potentially a poor prognostic.
Platinum resistant disease, I would point you to the or really a study that looked at a vast and plus chemotherapy, which had a 27 or 28% confirmed response rate and a median PFS of six seven months. They did not assess for folate receptor alpha and.
So I don't know how the fr Alpha high patients did but what I can tell you is there are studies that have been published mostly retrospective.
Looking at Fr Alpha is potentially a poor prognostic factor and if you look at our exploratory analyses from forward one at ESMO and 2019, and you'll see there there may be something to that but you know certainly not not anything that I would use to design a study assuming high fr Alpha patients would do worse than the overall population and standard with standard of care.
Anna Berkenblit: factor and if you look at our exploratory analyses from forward one at ESMO in 2019 you'll see there there may be something to that but you know certainly not not anything that I would use to design a study assuming high f r alpha patients would do worse than the overall population and standard with standard of care moving to recurrent platinum sensitive disease I would point you to the GOG 213 study and the ocean study looking at patients with one prior line of therapy and there the response rate is in the mid-50s and the PFS for platinum-based doublitz is somewhere between 8 and 1?2 months.
Sure.
Moving to recurrent platinum sensitive disease I would point you to the <unk> 2014 study and the oceans study looking at patients with one prior line of therapy and there. The response rate is and the mid fifties and the PFS for platinum based doublet is somewhere between eight eight and to happen 10, and a half months. So keep that in the back of your mind when you see our day.
Operator: So keep that in the back of your mind when you see our data from our mature MIRVBBBB cohort that Dr. O'Malley will be presenting in an oral presentation at ASCO. Thank you. Now I would like to hand the call back to our presenters for their final comments. Presenters, please go ahead. Thank you. We appreciate your time this morning. We look forward to keeping you apprised of our products and progress during the year, and in particular, seeing you at, or at least on Zoom anyway, for ASCO and the data updates there. This concludes today's conference call. Thank you all for joining us. You may now disconnect.
<unk> from our mature mirv Bev.
For the Doctor O'malley will be presenting and an oral presentation at Africa.
Okay. Thank you.
Now I would like to hand, the call over about two hour per centers for this.
Final comments presenters. Please go ahead.
Thank you well we appreciate your time. This morning, we look forward to keeping you apprised of our product and our progress and the year and in particular and seeing you at <unk>.
And on zoom anyway for for <unk>, and <unk> and the data updates there. Thanks.
This concludes today's conference call. Thank you all for joining you may now disconnect.
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