Q2 2020 Syndax Pharmaceuticals Inc Earnings Call

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Ladies and gentlemen, thank you for standing by and walk into the Synodex second quarter due South Sunshiny financial results Conference call.

Operator: Ladies and gentlemen, thank you for standing by, and welcome to the Syndax Second Quarter 2020 Financial Results Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. If anyone should require assistance during the conference, please press star then zero on your touchtone telephone. I would now like to turn the conference over to your host, Ms. Melissa Forrest. Please go ahead.

This time, all participants are and I listen only mode. Later, if you look I'm stuck a question and answer session and instructions will follow at that site.

Once you're there quite assistance during the conference. Please press star didn't zero on your touched on telephone.

I'd now like to turn the conference over to your House definitely supports please go ahead.

Thank you great welcome and thank you to those of you joining us on the line and the webcast. This afternoon for review its indexes second quarter Twentytwenty financial and operating result.

Melissa Forrest: Thank you, Grace. Welcome and thank you to those of you joining us on the line and the webcast this afternoon for a review of Syndax's second quarter 2020 financial and operating results. I'm Melissa Force with Argo Partners, and with me this afternoon to discuss the results and provide an update on the company's progress are Dr. Briggs Morrison, Chief Executive Officer, and Daphne Kouridis, Chief Financial Officer. Also joining us on the call today for the question and answer session are Michael Metzger, President and COO, Dr. Michael Myers, Chief Medical Officer, and Dr. Peter Ordentlich, Chief Scientific

Most of course with Argot partners and with me. This afternoon to discuss the results and provide an update on the company's progress are Dr. Briggs Morrison, Chief Executive Officer, and definitely Caredx Chief Financial Officer.

Also joining us in the call today for the question answer session is Michael Metzger, President and COO Dr., Michael Myers, Chief Medical Officer, and Dr., Peter or debt like Chief Scientific Officer.

Melissa Forrest: This call is being accompanied by a slide deck that has been posted on the company's website, so I would ask you to please turn to the forward-looking statements on slide two. Before we begin, I would like to remind you that any statements made during this call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these statements as a result of various important factors, including those discussed in the risk factors section of the company's most recent quarterly report on Form 10-Q, as well as other reports filed with the FCC. Any forward-looking statements represent the companies used as of today, August 6, 2020 only. A replay of the call will be available on the company's website, syndax.com, following the call. And with that, I'm pleased to turn the call over to Dr. Briggs Morrison, Chief Executive Officer of Syndax.

This call is being accompanied by a flight deck that has been posted to the company's website kodatsky to please turn to the forward looking statements like <unk>.

Before we begin I would like to remind you that any statements made during this call that are not historical are considered to be forward looking statements within the meaning of the private Securities Litigation Reform Act of 1995.

Actual results may differ materially from those indicated by these statements as a result of various important factors, including those discussed in the risk factor section in the company's most recent quarterly reports on form 10-Q, as well as other reports filed with the FCC.

Any forward looking statements represent the company's as of today August six twentytwenty only.

A replay of the call will be available on the company's website index Dot com following the call and with that I'm pleased to turn the call over to Dr. Briggs Morrison, Chief Executive Officer Cindat.

Great. Thank you very much Melissa. Thank you everyone for joining us on on today's call and webcast.

Briggs Morrison: Great. Thank you very much, Melissa. And thank you to everyone for joining us on today's call and webcast. Let me start my comments by welcoming Daphne Caritas to Syndax as our new Chief Financial Officer. Daphne completed her formal education at MIT and Harvard and has a distinguished professional career at some of America's most prestigious financial and pharmaceutical firms. We are truly honored to have her joining our team. You'll hear more from her later in the call.

Let me start my comments by welcoming gap between just indexes, our new Chief Financial Officer.

Daphne completed or formal education at MIT, and Harvard and that's a distinguished professional career at some of America's most prestigious financial and pharmaceutical firms.

A truly honored to have a joining our team you'll hear more from her later in the call.

I'd also like take this opportunity to thanks, Rick Schafer is exceptional contributions to sit back and wish him every success and as well and retirement.

Let me now turn to slide three which provides a high level summary of our current corporate priorities as we strive to realize a future, which people with cancer live longer and better than ever before.

Briggs Morrison: I'd also like to take this opportunity to thank Rick Shea for his exceptional contributions to Syndax and wish him every success in his well-earned retirement. Let me now turn to slide 3, which provides a high-level summary of our current corporate priorities as we strive to realize a future in which people with cancer live longer and better than ever before. The second quarter of this year was quite eventful.

The second quarter. This year was quite eventful for Sunday.

We were extremely disappointed when he causing form to eat 21 12. The phase three trial of intended stat hormone receptor positive hertwo negative breast cancer did not achieve the primary endpoint of demonstrating statistically significant overall survival benefit.

To this debt has been de prioritizing our portfolio and has definitely will discuss our go forward budget forecast includes only close out costs for it to the stuff.

We continued to collect encouraging data that further clarify the potential of our anti CSF, one or antibody actually tell them had to treat chronic graft versus host disease.

Briggs Morrison: We were extremely disappointed when ECOG informed us that E2112, the phase 3 trial of antennastat in hormone receptor positive HER2 negative breast cancer, did not achieve the primary endpoint of demonstrating a statistically significant overall survival benefit. Intendistat has been deprioritized in our portfolio, and as Daphne will discuss, our go-forward budget forecast includes only closeout costs for Intendistat. We continue to collect encouraging data that will further clarify the potential of our anti-CSF1 or antibody, exotilamide, to treat chronic graft-versus-host disease. And at AACR, we presented the first clinical evidence that inhibition of the MENIN-MLL1 interaction by SNDX5613, our MENIN inhibitor, can induce responses in patients with MLL-R acute leukemia. In the second quarter, we were granted orphan designation by FDA for 5613 for the treatment of adults with pediatric AML. We also announced the successful addition of over $100 million to our balance sheet under Attractive Care.

And the TCR, we presented a first clinical evidence that inhibition of the many MLL one interaction, but yes, and Dx 50, 613, our men and ensure that are going to dish response in patients with MLL R acute leukemia.

The second quarter, we were granted orphan designation by the FDA for 56 or team for the treatment of adult and pediatric KNL. We also announced is successful addition of over $100 million to our balance sheet under attractive terms.

Let's now turn to slide four.

50, 613 are genetically targeted agent for the treatment of leukemia.

Patrick noted previously there's extensive validation of both MLL R. M. P. M. One mutation size molecular targets and leukemia, and well established diagnostic tests routinely identify patients with these genetic mutation.

Premier publications provide the scientific rationale and pre clinical validation of our ongoing clinical trial and there are historic precedents that support a rapid regulatory path for such targeted agents in acute TV.

[noise] HR. This year, we presented preclinical early clinical data for 50 613, showing that our molecule is a potent and specific inhibitor of the amendment MLL interaction.

The only off target activity of note is it's relatively weak binding to the herd channel kind of clinical relevance, it's primarily metabolized by 54.

On slide five we've summarized the detailed experiments that were conducted to drive a model of the plasma exposures, we think will be needed patients to drive Africa.

We were of course, please that as shown on slide six second patients in the trial and the first patient in the trial with an MLL R. Rearrangement had plasma exposures that exceeded the levels, we anticipated would be required for efficacy and achieved a complete response.

Importantly, this patient safely experienced a deep and rapid response, choosing CR by day 28 of therapy, while experiencing nothing more than gray to adverse events.

Briggs Morrison: Let's now turn to slide 4 and 5613, our genetically targeted agent for the treatment of leukemia. As noted previously, there is extensive validation of both MLLR and NPM1 mutations as molecular targets in leukemia, and well-established diagnostic tests routinely identify patients with these genetic mutations. Premier Publications provide the scientific rationale and preclinical validation of our ongoing clinical trial, and there are historic precedents that support a rapid regulatory path for such targeted agents in acute locations. At AACR this year, we presented pre-clinical and early clinical data for 5613, showing that our molecule is a potent and specific inhibitor of the Menin-MLL interaction. The only off-target activity of note is its relatively weak binding to the HERD channel, and of clinical relevance, it's primarily metabolized by CYP3A4.

Of note. This patient was on a concomitant medication that inhibits the activity of Sixthree poor, which may account for the disproportional PK exposure relative to what we had anticipated.

Full details on additional patients are included in the Ace your presentation, that's available on our website.

[noise] scheme or for the updated ongoing augment one one trial first in human trial in the accelerate understanding of mending or augment program is shown on slide seven.

Phase one portion is a dose escalation trial designed to identify the maximum tolerated dose.

Recommended phase two dose of 50 613 in two independent cohorts of patients with relapsed or refractory leukemia.

Hey, enrolls patients who are not on a strong sift through Acorn editor and RMB enrolls patients who are.

On security for either at the time of enrollment.

The first 28 days of dosing service appears and safety is evaluated for determining dose escalation and and hard tend to define a recommended phase two dose for each cohort.

In June of this year, we participated in an F.D.A. pediatric oncology drug Advisory Committee meeting regarding our development strategy for Smbs 50 630.

We were quite pleased with a uniform enthusiasm of the committee and F.D.A. Kinda supportive comments made by several physician during the public common session.

Briggs Morrison: On slide five, we've summarized the detailed experiments that were conducted to drive a model of the plasma exposures we think will be needed in patients to drive FDA approval. We were, of course, pleased that, as shown on slide six, the second patient in the trial and the first patient in the trial with an MLLR rearrangement had plasma exposures that exceeded the levels we anticipated would be required for efficacy and achieved a complete response. Importantly, this patient safely experienced a deep and rapid response. She was in CR by day 28 of therapy while experiencing nothing more than grade 2 adverse events. Of note, this patient was on a concomitant medication that inhibits the activity of CYP3A4, which may account for the disproportional PK exposure relative to what we had anticipated.

It's clear to us that the pediatric oncology community physicians participating in our trials are excited about the progress we're making.

In addition to the ODAC meeting we've been working closely with an FDA to continue to modify augment one on one had their data accumulates.

Split into arm in arm B is one such example.

More recently based upon the totality of our accumulating data in both augment one on one.

In our pediatric compassionate use experience, we have worked with FTD to further modify the phase one portion argument on a one in three important ways.

First hefty. It has agreed that we can now focus enrollment exclusively to patients with MLL R and PMN one mutations.

You may recall that the trial initially there is open to all adults.

With relapsed or refractory leukemia.

[noise] second they have agreed to allow us to backfill any dose level up to a total of 12 patients in either arm eight or our be just efficacy has been observed in that dose level.

These backfill slots will open up enrollment it will provide more PK and safety data in an expanded phase one trial.

And we'll give us greater certainty as we enter phase two.

And could potentially accelerate our path to eventual approval.

And third FDA has strongly encouraged us to enroll pediatric patients.

Briggs Morrison: Full details on additional patients are included in the AACR presentation that's available on our website. The scheme for the ongoing Augment 101 trial, the first human trial in the Accelerated Understanding of Mending or Augment program, is shown on slide 7. The Phase I portion is a dose escalation trial designed to identify the maximum tolerated dose and recommended Phase II dose of 5613 in two independent cohorts of patients with relapsed or refractory leukemia. Arm A enrolls patients who are not on a strong CYP3A4 inhibitor, and Arm B enrolls patients who are on CYP3A4 inhibitor at the time of enrollment. The first 28 days of dosing serve as the period in which safety is evaluated for determining dose escalation, and our intent is to define a recommended phase 2 dose for each cohort. In June of this year, we participated in an FDA Pediatric Oncology and Drug Advisory Committee meeting regarding our development strategy for SNDX5613. We were quite pleased with the uniform enthusiasm of the committee and FDA and the supportive comments made by several physicians during the public comment session.

Any patient over 30 days of age.

We believe their current summit to include pediatric patients. So early in the drug development process is based on the results to date and augment one on one as well as what we are seeing compassionate use treatment setting.

We continue to receive request to provide drug to children currently in eligible to enroll and augment one or one and so we've continued to support these compassionate use treatments aligned with the dose levels that have been studied and augment bottom line.

As you've got to doses, which can achieve our pre specified PK thresholds physicians are starting to see activity in these pediatric patients as well.

Do we recently received results from an adolescent who is treated on compassionate use and its clinical courses strikingly similar to patient number two illustrated on slide six.

The child's had refractory MLL R L.

Received if indeed, the at 50 613 at the equivalent of dose levels to RMB. That's 226 milligrams by mouth twice a day in combination with a strong shipped three four inhibitor.

The pace that plasma exposures that exceeded the levels, we anticipated would be required for efficacy achieved complete response on day 28 and experience only grade one adverse event.

One could only imagine how ecstatic the parents of this child hard to have renewed hope for their youngster.

[noise], we're eager to share publicly the observations that allowed us to modify augment one on one.

Goal has been to present the completed phase one a portion of August meant one on one in its entirety.

And we had hoped to do so by the end of this year.

We continue to anticipate identified recommended phase two dose and completing phase one by the end of this year and yet these exciting trial enhancements that I've described a we'll now move our presentation of the completed phase one data into early 2021.

Briggs Morrison: It is clear to us that the pediatric oncology community and the physicians participating in our trials are excited about the progress we are making. In addition to the ODAC meeting, we have been working closely with FDA to continue to modify Augment 101 as our data accumulates. Splitting into Arm A and Arm B is one such example. More recently, based upon the totality of our accumulating data in both Augment 101 and in our Pediatric Compassionate Use Experience, we have worked with FDA to further modify the Phase I portion of Augment 101 in three important ways. First, FDA has agreed that we can now focus enrollment exclusively on patients with MLLR or NPM1 mutations. You may recall that the trial initially was open to all adults with relapsed or refractory leukemia.

As a reminder, the phase two trial will proceed to enroll three distinct expansion cohorts each of which consists of a specific genetically defined relapse or refractory peaceful cambia.

The three cohorts are shown on slide eight and will include patients with MLL R. AOL patients with MLL R email and patients with NPM, one mute and now.

Phase two portion will feel the characterize the safety of 50 613 and will provide an initial estimate of the complete response rate as the primary measure of therapeutic benefit.

Like the changes to the phase one phase two will enroll both pediatric and adult patients.

Thereby providing us the potential path to regulatory approval with a broad label, including both adults and pediatrics.

We look forward to continuing to update you on the progress of augment what one as the year progress.

Let me now turn to slide nine NASA telematics, which was formerly known as has been Dx 63, 52, our potential best in class monoclonal antibody therapy targeted CSF one receptor.

So its from the multiple ascending dose trials exploring acetone that alone and in combination with durvalumab in patients with solid tumors, which presenting presented during this year's a CRB meeting.

Briggs Morrison: Second, they have agreed to allow us to backfill any dose level up to a total of 12 patients in either arm A or arm B if efficacy has been observed at that dose level. These backfill slots will open up enrollment and will provide more PK and safety data in an expanded Phase 1 trial and will give us greater certainty as we enter Phase 2, and could potentially accelerate our path to eventual approval. Third, FDA has strongly encouraged us to enroll pediatric patients, including any patient over 30 days of age. We believe their encouragement to include pediatric patients so early in the drug development process is based on the results to date in Augment 101, as well as what we are seeing in the compassionate use treatment setting.

These results demonstrate the tolerability and robust PD biomarker modulation of Acetonide.

Underscored its ability to promote rapid and sustained depletion of circulating pro inflammatory monocytes at all dose levels tested.

As you know we initiated a trial testing exit telematics monotherapy in chronic graft versus host disease in the rationale for that.

On slide 10.

Chronic graft versus host cities in the frequent complication of amount appointed stem cell transplantation, where in the donor derived immune cells contribute to the initiation in development of fibrosis in the myriad manifestations of the study.

Preclinical models blocking the CSF, one CSF one hour.

With anti CSF Oner antibodies and result in depletion and told her macrophages nearby both prevent and reduce chronic graft versus host disease.

[noise] last year, we released initial data from our phase one trial, which is diagrammed on finalizing.

Phase one portion is a dose escalation trial designed to identify the maximum tolerated dose and a recommended phase two dose of axa till amount for the treatment of chronic gvhd.

We've released data from the first five patients enrolled the first three cohorts and have them modify the trial to include the phase two covert to one make per kick knows.

Briggs Morrison: We continue to receive requests to provide drugs to children currently ineligible to enroll in Augment 101, and so we continue to support these compassionate use treatments aligned with the dose levels that have been studied in Augment 101, as we've gotten to doses that can achieve our pre-specified PK thresholds. Physicians are starting to see activity in these pediatric patients as well. Indeed, we recently received results from an adolescent who was treated on compassionate use and whose clinical course is strikingly similar to patient number two, illustrated on slide six. The child had refractory MLLR-AML, and received SNDVX5613 at the equivalent of dose level 2.

No continues a phase one trial to more formally define the recommended phase two dose and May open one of them or additional phase two cohorts as well.

We anticipate presented phase one data for approximately.

10 to 15 patients at the end of this year at a medical conference.

In addition, we're in discussions with regulators concerning the design over Registrational program for chronic graft versus host disease.

We anticipate providing full details of the registration program at our next quarterly call and hope to initiate our registration trial.

As early as the end of this year.

We believe that chronic gvhd represents high unmet medical need and an important commercial opportunity with approximately 14000 patients suffering from gvhd in the U.S. today.

With the recent positive pivotal results from both insights Jackup API and cabins Katy zero to five we may soon see commercial launches that will begin to delineate the commercial opportunity in chronic graft versus host disease.

Despite recent advances in this area, we believe that data generated today with next until about suggest it has the potential could play an important role in the treatment for gvhd.

Briggs Morrison: On arm B, that's 226 milligrams by mouth twice a day in combination with a strong 5384 inhibitor. The patient had plasma exposures that exceeded the levels we anticipated would be required for efficacy, achieved a complete response on day 28, and experienced only grade 1 adversity. One can only imagine how ecstatic the parents of this child are to have renewed hope for their young.

Finally, slide 12 summarizes the transactions and led to the acquisition of the men as MLL R. An x. until the map programs.

Believed that we will be.

Able to continue to expand our pipeline to the acquisition in licensing of quality differentiated assets.

We believe that we have the necessary skill to evaluate can identify high quality assets and the clinical development experience to bring these compounds through valuable inflection point.

We expect to remain among preferred partners such transactions.

I'll now turn the call over the definitely to review our financial results.

Thank you Greg first let me start by thanking you for the Great introduction I am extremely fortunate to join the index at a very exciting and pivotal time for the company and I'm looking forward to helping sent back to achieve its goals, bringing new life altering treatment to cancer patients I also want to thank Greg for a great transmission and I wish him all the.

Briggs Morrison: We're eager to share publicly the observations that have allowed us to modify Augment 101. Our goal has been to present the completed Phase 1 portion of Augment 101 in its entirety, and we had hoped to do so by the end of this year. We continue to anticipate identifying a recommended phase 2 dose and completing phase 1 by the end of this year, and yet these exciting trial enhancements that I've described will now move our presentation of the completed phase 1 data into early 2021. As a reminder, the Phase II trial will proceed to enroll three distinct expansion cohorts, each of which consists of a specific genetically defined relapse or refractory acute leukemia. These three cohorts are shown on slide 8 and will include patients with MLLR-ALL, patients with MLLR-AML, and patients with NPM1 mutant AML.

Back in his retirement now let me turn to the quarter. The results of our operations for the second quarter of 2020, and the comparison to the prior year quarter are included in our press release, So I won't repeat them in these remarks additional financial details will be available in our second quarter report on form 10-Q, which will be filed today.

I would like to point out that our operating loss for the quarter was $17.1 million or 42 cents per share compared to $14.9 million or 47 cents per share for the same period last year.

Turning to slide 13, we ended the second quarter with $186.8 million in cash and cash equivalents and 44.1 million share and pre funded warrants outstanding. This included the proceeds from our may equity offering or 6.4 million shares at $18 per share with.

Net proceeds of $107.9 million.

Looking ahead I'd like to provide financial guidance for the second half of 2020, you will recall that we had with health second half guidance until such time that they each 21 12 phase III trial was completed.

For the second half of 2020, R&D expenses will increase over the first pack primarily due to increased development activities for both 50 613 and ask the telematics as Briggs discussed in his remark we are enhancing the phase one portion of the men and augment one or one trial, while simultaneously preparing for phase two trial.

Briggs Morrison: The phase 2 portion will further characterize the safety of 5613 and will provide an initial estimate of the complete response rate as the primary measure of therapeutic benefit. Like the changes to the Phase I, phase II will enroll both pediatric and adult patients, thereby providing us a potential path to regulatory approval with a broad label including both adults and pediatrics. We look forward to continuing to update you on the progress of Augment 101 as the year progresses.

For both 50 613 and back to tell them at a significant portion of the increased R&D expenses will be going toward CMC activities for both program as we want to be prepared for potential accelerated filing I.

Additionally, and tell us that has been de prioritized and second half R&D expenses will include only wind down cost for the program.

Second half DNA expenses are expected to be lower than the first half primarily due to lower pre commercialization activities.

For the third quarter and second half of 2020, we expect R&D expenses to be $14 million to $16 million and $30 million to $35 million, respectively, and total operating expenses for the third quarter and second half of 2020 to be $19 million to $21 million and 40 to 45 million.

Briggs Morrison: Let me now turn to slide 9, Naxotilumab, which was formerly known as SNDX6352, our potential best-in-class monoclonal antibody therapy targeted at CSF1 receptors. Results from the multiple ascending dose trials exploring axotilumab alone and in combination with rivalumab in patients with solid tumors were presented at this year's AACR meeting, and these results demonstrated the tolerability and robust PD biomarker modulation of axotilumab, and underscored its ability to promote rapid and sustained depletion of circulating pro-inflammatory monocytes at all dose levels. Chronic Rack vs.

Dollars, respectively, including approximately $2 million noncash stock compensation expense per quarter. This guidance includes $4 million and milestone payments to use CB and we anticipate in the second half based on achievement of positive development milestones associated with Aquaterra Matt.

Given our cash operating expense guidance for the second half of 2020, we expect yet to end the year with approximately $145 million of cash which gives the cash runway into 2022, I would now like to turn the call back over to Brad.

Thank you Daphne and welcome again doesn't yet.

As I hope you have appreciated for my prepared remarks, the first half of this year has brought significant change this index.

We are clearly disappointed about the results of the 21 12.

Most disappointed for women with hormone receptor positive breast cancer, we really hoped that we headed novel medicines for them. We're working with you talked to understand the 21 12 results that we anticipate that he Cogs will present results at San Antonio breast cancer Symposium.

On the other hand, we're incredibly excited about Sdx 50, 613, our men and MLL R exhibitor.

Briggs Morrison: Host Disease is a frequent complication of hematopoietic stem cell transplantation, wherein the donor-derived immune cells contribute to the initiation and development of fibrosis in the myriad manifestations of this disease. Preclinical models have shown that blocking the CSF-1 and CSF-1R with anti-CSF-1R antibodies can result in depletion of toner macrophages and thereby both prevent and reduce chronic The Phase 1 portion is a dose escalation trial designed to identify the maximum tolerated dose and a recommended Phase 2 dose of exatilumab for the treatment of chronic GVHD. We've released data from the first five patients enrolled in the first three cohorts and have now modified the trial to include a Phase II cohort at the 1 mg per kg dose.

Addition to the CR. We described the CR. We've now described the similar rapid deep sea our in our compassionate use experience. The phase one trial is now focused on MLL R and PMN one patients only.

It's been expanded in the inclusion of pediatric patients provides the potential path to initial broad label.

With many questions to addressing this program, but we believe we have the skilled resources needed to advance the program and potentially enable early regulatory clarity.

We're also excited about it really results were seeing with exit telematics chronic gvhd.

I look forward to presenting updated data from this program at the end of this year and updating you on the design and timing of a registration trial.

With our recent highly successful financing we are comfortable that we have the financial resources to aggressively advance our programs and achieved key upcoming milestones.

We remain optimistic that we will continue to identify bringing novel molecules to deepen our portfolio.

Proven track record of delivering on this pillar our strategy and I believe this is a core strength of our company.

As always I would like to thank the wonderfully talented team here at Syndax, our collaborators and most importantly, the patients trial sites and investigators involved with our clinical program.

In addition, I'd like to thank our committed long term investors for helping us to build this great company and to welcome to new investors, who joined that effort with our recent financing.

Briggs Morrison: We will continue the Phase 1 trial to more formally define the recommended Phase 2 dose and may open one or more additional Phase 2 cohorts as well. We anticipate presenting the phase 1 data for approximately 10 to 15 patients at the end of this year at a medical conference. In addition, we are in discussions with regulators concerning the design of a registrational program for chronic graft-versus-host disease. We anticipate providing full details of the registration program at our next quarterly call and hope to initiate our registration trial as early as the end of this year.

With that I'd like to open the call to questions.

Ladies and gentle nice to have a question at this time. Please press Star then the number one key I touched on telephone again that will be stars in the number one I touched on telephone.

First question comments from the line of Peter Lawson from Barclays. Your line is open.

Hey, guys. This is always offer thanks for taking your question.

Just a question on the.

Alright, well one study where are you for current enrollment and how many patients.

Cricket has the most of our persons Gen. One mutation.

Oh.

Yeah. Thanks, so much for the question, we'll update the full phase one trial for augment what a one when we presented data and not currently 2021.

Got it thank you and.

I know that you you mentioned that you'll be looking.

Briggs Morrison: We believe that chronic GVHD represents a high unmet medical need and an important commercial opportunity, with approximately 14,000 patients suffering from GVHD in the U.S. today. With the recent positive pivotal results from both Insights Jackify and Cadman's KD-025, we may soon see commercial launches that will begin to define the commercial opportunity for chronic graft-versus-host disease. Despite recent advances in this area, we believe the data generated to date with exotilamab suggests it has the potential to play an important role in the treatment of GVHD.

Looking to get to a recommended phase two dose by year end, we'll read potentially to an update Harvey pieces that you've already presented on a CR as well.

Again, I think our intent was to present the entire completed phase one trial. So that will include the update on the HCR patients when we present the completed phase one trial.

Got it. Thank you thanks for taking the questions.

Thank you. Your next question comes from the line of Chris Sheba Tiny from Cowen Your line is open.

Great. Thank you very much a question.

Or you about the discussions that you had with the FDA and the planning adjustment you've made for the trial can you talk to us about what you're expecting the pediatric conclusion could do in terms of timelines and label and also maybe comment about how you're thinking about things from the standpoint of for instance, compendia listing historically some of the pads.

Daphne Kouridis: Finally, slide 12 summarizes the transactions that led to the acquisition of the Mennon MLLR and the Axotilumab program. We believe that we will be able to continue to expand our pipeline through the acquisition or in-licensing of quality differentiated assets. We believe that we have the necessary skill to evaluate and identify high-quality assets and the clinical development experience to bring these compounds through valuable inflections. We expect to remain among preferred partners in such transactions. I will now turn the call over to Daphne to review our financial results.

Towards actual use have been fairly brisk in this type of setting based upon fairly modest clinical trial data sets I'll start there and they have a follow up for Daphne.

Sure. Thanks, so much Chris.

The peaks inclusion I don't think changes the timeline to finish the phase one trial, obviously it gets patients into that trial and.

Will help with is set up for phase two.

And as I indicated in my prepared remarks, I think the inclusion the indication we've gotten in our conversations with the FDA is that the inclusion of both pediatric and adult in both phase one and phase two could potentially enable broad label that includes both piece in adult.

In terms of Compendia listing I think the Compendia listings as you say are.

Daphne Kouridis: Thank you, Briggs. First, let me start by thanking you for the great introduction. I am extremely fortunate to join Syndax at a very exciting and pivotal time for the company, and I'm looking forward to helping Syndax achieve its goals of bringing new life-altering treatments to cancer patients. I also want to thank Rick for a great transition, and I wish him all the best in his retirement.

Another wage is yet to present information to their community I think the the trial, we have ongoing would be a labeled indication.

But we are in discussions with.

Many investigators about investigator sponsored trials that could add.

Compendia data as well.

Great and then a follow up for Daphne Daffy Congratulations on your role and we welcome you certainly with the past that you've already.

Just passed [laughter] similar to many of us in the past here as much appreciated as you put your fresh eyes on the portfolio in the track record. The company has had in terms of in tennis that previously an Io and limited breast cancer effort and now based upon some comments that continue to be scripted about thinking to continue to look for assets what would be somebody.

Daphne Kouridis: Now let me turn to the quarter. The results of our operations for the second quarter of 2020 and the comparison to the prior year's quarter are included in our press release, so I won't repeat them in these remarks. Additional financial details will be available in our second quarter report on form 10-2, which will be filed today. I would like to point out that our operating loss for the quarter was 17.1 million dollars, or 42 cents per share, compared to 14.9 million dollars, or 47 cents per share, for the same period last year. Turning to slide 13, we ended the second quarter with $186.8 million in cash and cash equivalents and 44.1 million shares and pre-funded warrants outstanding. This included the proceeds from our May equity offering of 6.4 million shares at $18 per share with net proceeds of $107.9 million.

Your initial thoughts perhaps unfair you haven't at 100 days in the seat, but I'll ask it anyway.

Thank you, Chris and thank you for the welcoming remarks, so what I would say is out one of the very attractive elements of this this organization in this management team for me to join the dock was exactly those capabilities that are very enormous amount of talent.

On not only in being able to develop the African hand, but also to look for and identify interesting assets I doubt that I think will fit in very nicely with the again the capabilities and the focus of this organization and I'm very excited to be a part of that organization.

And those opportunities.

Would you be keener for R&D type projects that might have a couple of years are you thinking.

More commercial Where's remaining.

So I think that the overall leaning that would be at in the development stage, but I think we're looking at all opportunities then Doug.

No and I know that with every opportunity there volleys our risk reward it does.

Risk benefit a equation and I think we're open to looking across the board, but I think our preferences and the development stage.

Sounds great. Thank you both appreciate the comments.

Daphne Kouridis: Looking ahead, I'd like to provide financial guidance for the second half of 2020. You will recall that we had withheld second half guidance until such time as the E21-12 phase 3 trial was completed. For the second half of 2020, R&D expenses will increase over the first half, primarily due to increased development activities for both 5613 and oxytelimab. As Briggs discussed in his remarks, we are enhancing the Phase 1 portion of the Mennon Augment 101 trial while simultaneously preparing for Phase 2 trials for both 5613 and oxytelimab. A significant portion of the increased R&D expenses will be going towards CMC activities for both programs, as we want to be prepared for potential accelerated filing. Additionally, atenostat has been deprioritized, and second-half R&D expenses will include only wind-down costs for this program.

Thanks, Chris.

Once again, ladies and gentlemen, if you have a question at this time. Please press Star then the number one key on your touched on telephone against that as far down the number one key I've touched on telephone.

Your next question comes from the line as Joel Beatty from Citi. Your line is open.

Hi, guys. This is Sean Egan, calling in for Joel.

It's great to hear about complete response in the pediatric patients with that one of the five patients that was announced that they here for the vision that were those previously.

And then I've a follow up question as well.

Hey, Sean Thanks for your question no. So the first five patients that were presented at a CR I think we made can they were also discussed at the pediatric ODAC Hum.

None of those patients really had plasma exposures that were we thought would be associated with efficacy none of them. We're taking a step through a four inhibitor. So as I indicated in my prepared remarks, we've continued.

To essentially dose escalate the compassionate use patients.

Parallel with how we have dose escalated augment one on one so the patient that.

That we described it in my prepared remarks is not one of those five it's a recently.

Communicated result from.

Patient that was on to 26 with with six rate for inhibitor.

Okay. Thank thank you for that clarity.

It can you comment just kind of on the broader strategy for 50, 613, and how you're looking at.

Obviously, you are enrolling refractory now, but then again, how do you plan to kind of pivot into induction and maintenance and try to capitalize on the whole market.

Daphne Kouridis: Second-half G&A expenses are expected to be lower than the first half, primarily due to lower pre-commercialization activities. For the third quarter and second half of 2020, we expect R&D expenses to be $14 to $16 million and $30 to $35 million, respectively, and total operating expenses for the third quarter and second half of 2020 to be $19 to $21 million and $40 to $45 million, respectively, including approximately $2 million of non-cash stock compensation expense per quarter. This guidance includes $4 million in milestone payments to UCB that we anticipate in the second half based on the achievement of positive development milestones associated with Acetelimab. Given our cash operating expense guidance for the second half of 2020, we expect to end the year with approximately $145 million in cash, which gives us cash runway into 2022. I would now like to turn the call back over to Briggs.

Yes, so clearly our our.

Focus right now is to get the drug approved.

When you see this kind of activity. This early in a phase one program it gives us.

A good sense that we may be onto something that could could easily be.

Make it across to go line. So our focus right now is to is to do the things we need to do to get to drug approved.

We are in discussions with a number of groups a number of.

Experts in the area about a variety of other uses for the drug as you point out in combination in deduction.

And maintenance after induction and maintenance after bone marrow transplant or many different indicate.

[noise] clinical avenues, where this drug might be useful and we're exploring many of those.

Great. Thanks Bert.

Next we have Mr. Ahmed their Kumar from Baird. Your line is open.

Hey, Thanks for taking my call. This is Rob going from a do my question was for active drilling.

Can you walk through selecting a one mg per kg dose for phase two given that the phase one dose escalations ongoing.

Yeah sure. So in the phase one person that with the data we had <unk> released at the end of last year. We had studied 0.15 0.5 and one.

Based upon PK PD modeling, we had thought that one was probably going to be the appropriate dose and we saw you know I'm, good tolerability and efficacy at that though so what we decided to do with to expand that dose level. Why we finished the phase one it doesn't preclude us from going back and studying other dose.

Briggs Morrison: Thank you, Daphne, and welcome again. As I hope you've appreciated from my prepared remarks, the first half of this year has brought significant change. We are incredibly disappointed about the results of E2112. We are most disappointed for women with hormone receptor positive breast cancer. We really hoped that we had a novel medicine for that. We're working with ECOG to understand the E2112 results, and we anticipate that ECOG will present results at the San Antonio Breast Cancer Center. On the other hand, we're incredibly excited about SNDX5613, our Menin MLR inhibitor. In addition to the CR we described in AACR, we've now described a similar rapid and deep CR in our Compassionate Use Experience. The Phase I trial is now focused on MLLR and NPM1 patients only.

Levels either in.

An expanded phase one for in phase two but the PK PD looked pretty good that.

Tolerability looked good and so we thought we'd expand that to start to get a better sense of.

You know the broader.

Applicability of the of the of the drugs.

Okay. Thank you and just a follow up to what extent are you considering that drugs utility in non cancer setting the juice for cancer non cancer indications internally or externally.

Right. So I mean again, we chose chronic graft associates because it is sort of a in a way a prototype for a variety of different auto immune diseases and so we are looking at a.

A number of different indications.

Some of which we would feel perfectly comfortable running ourselves some of which we might want to look for a partner who's got deeper expertise in that particular disease. So.

Thats worked out we're doing right now both to prioritize what indications and then how we go about pursuing them.

Thank you.

Thank you and I'm showing no further questions at this time I wouldn't like to turn the conference back to our C O Dr. Martin for any closing remarks.

Hi, just want to thank everybody again for joining the webcast I again want to welcome Daphne to syntax and now we hope everybody is well and have a good summer. Thanks, so much.

Briggs Morrison: It's been expanded, and the inclusion of pediatric patients provides a potential path to initial broad linkage. We have many questions to address in this program, but we believe we have the skill and resources needed to advance the program and potentially enable early regulatory approval. We're also excited about the early results we're seeing with acetylamab and chronic GVHD. We look forward to presenting updated data from this program at the end of this year and updating you on the design and timing of a registration trial. With our recent, highly successful financing, we are comfortable that we have the financial resources to aggressively advance our programs and achieve key upcoming milestones. We remain optimistic that we will continue to identify and bring in novel molecules to deepen our portfolio.

Thank you everyone, ladies and gentlemen, this concludes todays conference call. Thank Walker Jining human all disconnect.

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Briggs Morrison: We have a proven track record of delivering on this pillar of our strategy, and I believe this is a core strength of our company. As always, I would like to thank the wonderful talented team here at Syndax, our collaborators, and, most importantly, the patients, trial sites, and investigators involved in our clinical program. In addition, I'd like to thank our committed long-term investors for helping us to build this great company and to welcome the new investors who have joined that effort with our recent finalists. With that, I'd like to open the call to questions.

Operator: Ladies and gentlemen, if you have a question at this time, please press the number one key on your touch-tone telephone. Again, that will be the number one on your touch-tone telephone. Your first question comes from the line of Peter Lawson from Barclays. Your line is open. Hey guys, this is Wally Dawn on behalf of Peter.

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Wally Dawn: Thanks for taking the questions. I just have a question on the Augment 101 study. Where are you for current enrollment?

Operator: And how many patients that you've recruited have the MLLR versus MPM1 mutations?

Briggs Morrison: Yeah, thanks so much for the question. We'll update the full phase one trial for Augment 101 when we present the data in early 2021.

Briggs Morrison: Got it. Thank you. And I know that you mentioned that you would be looking to get to a recommended phase two dose by year-end. Will we potentially get an update on the patients that you've already presented on at ACR as well?

Briggs Morrison: Again, I think our intent was to present the entire completed Phase 1 trial, so that will include an update on the AACR patients when we present the completed Phase 1 trial.

Operator: Got it. Thank you. Thank you for taking the question. Thank you. Your next question comes from the line of Chris Shibutani from Cohen. Your line is open.

Chris Shibutani: Great, thank you very much. I have a question for you about the discussions that you've had with the FDA and the planning adjustments you've made for the trial. Can you talk to us about what you're expecting the pediatric inclusion could do in terms of timelines and labels? And also maybe comment on how you're thinking about things from the standpoint of, for instance, compendia listing? Historically, some of the paths toward actual use have been fairly brisk in this type of setting based upon fairly modest clinical trial data sets. I'll start there and then have a follow-up for Daphne.

Briggs Morrison: Sure. Thanks so much, Chris.

Briggs Morrison: The PEDS inclusion, I don't think, changes the timeline to finish the Phase 1 trial. Obviously, it gets patients into that trial and will help with the setup for Phase 2. And as I indicated in my prepared remarks, I think the inclusion, the indication we've gotten in our conversations with FDA is that the inclusion of both pediatrics and adults in both Phase 1 and Phase 2 could potentially enable a broad label that includes both PEDS and adults. In terms of compendia listings, I think the compendia listings, as you say, are another way to present information to the community. I think the trial we have ongoing would be a labeled indication, but we are in discussions with many investigators about investigator-sponsored trials that could add compendia data as well.

Chris Shibutani: Great. And then a follow-up for Daphne. Daphne, congratulations on your role, and we welcome you, certainly with the past that you've already crossed, similar to many of us in the past year. It's much appreciated. As you put your fresh eyes on the portfolio and the track record that the company has had in terms of Antenna Stat, previously in IO, and then with the breast cancer effort, and now based upon some comments that continue to be scripted about thinking to continue to look for assets, what would be some of your initial thoughts? Perhaps unfair that you haven't had 100 days in the seat, but I'll ask it anyway.

Daphne Kouridis: Thank you, Chris, and thank you for the welcoming remarks. So, what I would say is that one of the very attractive elements of this organization and this management team for me to join Syndax was exactly those capabilities, the very enormous amount of talent, not only in being able to develop the assets in hand but also to look for and identify interesting assets that I think will fit in very nicely with the capabilities and the focus of this organization. And I'm very excited to be a part of that.

Chris Shibutani: Would you be keener for R&D type projects that might take a couple of years? Are you thinking more commercial? Where's your leaning?

Daphne Kouridis: So I think that the overall leaning would be in the development stage, but I think we're looking at all opportunities, and as you know and I know, with every opportunity there's always a risk-reward and risk-benefit equation, and I think we're open to looking across the board, but I think our preference is in the development stage.

Chris Shibutani: Sounds great. Thank you both. Appreciate the comments. Thanks, Chris.

Operator: Once again, ladies and gentlemen, if you have a question at this time, please press star then the number one key on your touch-tone telephone. Again, that is star then the number one key on your touch-tone telephone. Your next question comes from the line of Joel Beatty. From Beatty, your line is open. Hi guys, this is Sean Deegan calling in for Joel. It was great to hear about the complete response in the pediatric patient. Was that one of the five patients that was announced at the AACR presentation that were dosed previously? And then I have a follow-up question as well.

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Sean Deegan: Hey Sean, thanks for your question. No, so the first five patients that were presented at ACR, I think we made, and they were also discussed at the Pediatric ODEC, none of those patients really had plasma exposures that we thought would be associated with efficacy. None of them were taking a CYP3A4 inhibitor. So, as I indicated in my prepared remarks, we've continued to essentially dose escalate the compassionate use patients in parallel with how we have dose escalated Augment 101. So, the patient that we described in my prepared remarks is not one of those five. It's a recently communicated result from a patient that was on 226 with a CYP3A4 inhibitor.

Briggs Morrison: Okay, yeah, thank you for that clarification. Maybe can you comment just kind of on the broader strategy for 5613 and how you're looking at, obviously you're enrolling labs per factory now, but then again, how do you plan to kind of pivot and do induction and maintenance and try to capitalize on the whole market?

Briggs Morrison: Yeah, so clearly, our focus right now is to get the drug approved. You know, when you see this kind of activity this early in a phase one program, it gives us a good sense that we may be on to something that could easily make it across the goal line. So our focus right now is to do the things we need to do to get the drug approved. We are in discussions with a number of groups and a number of experts in the area about a variety of other uses for the drug, as you point out, in combination with induction, in maintenance after induction, and in maintenance after bone marrow transplantation. There are many different clinical avenues where this drug might be useful, and we're exploring many of them.

Sean Deegan: Great, thank you very much.

Operator: Alright, thank you very much. Next, we have Mr. Madhu Kumar from Bayer. Your line is open.

Rob: Hey, thanks for taking my call. This is Rob calling in from MADU.

Briggs Morrison: My question was, for Axapilumab... Can you walk through selecting the 1 mg per kg dose for phase 2 given that the phase 1 dose escalation is ongoing?

Briggs Morrison: Yeah, sure. So, in the Phase 1 portion, the data we released at the end of last year, we studied 0.15, 0.5, and 1. Based upon PK-PD modeling, we had thought that one was probably going to be the appropriate dose, and we saw, you know, good tolerability and efficacy at that dose. So what we decided to do was to expand that dose level while we finished Phase I. It doesn't preclude us from going back and studying other dose levels, either in an expanded Phase I or in Phase II, but the PK-PD looked pretty good. The tolerability looked good, and so we thought we'd expand that to start to get a better sense of..., you know, the broader applicability of the drug.

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Rob: Okay, thank you. And just to follow up, to what extent are you considering that drug's utility in non-cancer settings? Would you explore cancer and non-cancer indications internally or externally?

Briggs Morrison: Right. So, I mean, again, we chose chronic graft-resistance disease because it is sort of, in a way, a prototype for a variety of different autoimmune diseases. And so we are looking at a number of different indications, some of which we would feel perfectly comfortable running ourselves, some of which we might want to look for a partner who's got deeper expertise in that particular disease. So, you know, that's work that we're doing right now, both to prioritize which indications to pursue and then how we go about pursuing them. Thank you.

Operator: Thank you, and I'm showing no further questions at this time. I would like to turn the conference back to our CEO, Dr. Morrison, for any closing remarks.

Briggs Morrison: I just want to thank everybody again for joining the webcast. Again, I want to welcome Daphne to Syndax and we hope everybody is well and has a good summer.

Operator: Thank you, everyone. Ladies and gentlemen, this concludes today's conference call. Thank you all for joining us. You may now disconnect.

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