Onconova Therapeutics, Inc. (ONTX) CEO Steve Fruchtman on Q1 2022 Results - Earnings Call Transcript

May 11, 2022 7:32 PM ETOnconova Therapeutics, Inc. (ONTX)
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Onconova Therapeutics, Inc. (NASDAQ:ONTX) Q1 2022 Earnings Conference Call May 11, 2022 4:30 PM ET

Company Participants

Avi Oler - Senior Vice President-Corporate Development and General Counsel

Steve Fruchtman - President and Chief Executive Officer

Mark Gelder - Chief Medical Officer

Mark Guerin - Chief Financial Officer

Conference Call Participants

Charles Zhu - Guggenheim

Ahu Demir - Ladenburg

Joe Pantginis - H.C. Wainright

Robert LeBoyer - Noble Capital Markets

Operator

Ladies and gentlemen, thank you for standing by. Welcome to the Onconova Therapeutics' First Quarter 2022 Financial Results and Business Update Conference Call. At this time, all participants are in a listen-only mode. Following management's prepared [Technical Difficulty] [Operator Instructions] As a reminder, this call is being recorded today, May 11,022.

At this time, I'd like to turn the call over to Avi Oler, Senior Vice President of Corporate Development and General Counsel.

Avi Oler

Thank you operator, and welcome everyone to Onconova's first quarter 2022 financial results and business update conference call. Earlier this afternoon, we issued a press release reporting our financial results and business progress. If you have not yet seen this press release, it is available on the Investors and Media section of our website at www.onconova.com.

Today's call will begin with our President and CEO, Dr. Steve Fruchtman providing an overview of our recent progress and corporate strategy. Dr. Fruchtman will then ask our Chief Medical Officer, Dr. Mark Gelder to review our clinical programs. Our CFO, Mark Guerin will then report our first quarter financial results before we move on to a question-and-answer session.

Before turning the call over to Steve, I’d like to remind everyone that statements made by management during this conference call will include forward-looking statements under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties that can cause actual results to differ materially. Forward-looking statements speak only as of the date they are made as the underlying facts and circumstances may change. Except as required by law, Onconova disclaims any obligation to update these forward-looking statements to reflect future information, events or circumstances. For more information on forward-looking statements, please review the disclaimer in today’s press release and the risk factors in the company’s SEC filings.

With that, it is my pleasure to turn the call over to Steve.

Steve Fruchtman

Thank you, Avi and good afternoon everyone. Our clinical programs continued to make strong progress over the past months as we efficiently executed on the corporate strategy we've spoken about on our past earnings calls. This strategy has us internally focused on our nerazacyclib program, while leveraging relationships with thought leaders from industry and academia to continue the development of rigosertib through investigator-sponsored studies, based on the mechanism of action of rigosertib to target cancer indications of interest.

As many listening may recall, nerazacyclib is a orally available multi-targeted kinase inhibitor, it has a low nanomolar affinity for CDK4/6 and other kinases that play an important roles in tumor cell proliferation, metastasis and survival. It can also stimulate anti-cancer immunity through the inhibition of the CSF1 receptor and disrupt several pathways linked to the described mechanisms of drug resistance for this class of drugs. We believe this inhibitory profile positions nerazacyclib to be a highly differentiated and potentially a best-in-class therapy. And we look forward to the publication of an abstract at the upcoming ASCO 2020 meeting that describes this topic further.

Currently two ongoing Phase I trials are evaluating nerazacyclib in patients with solid tumors. These trials are collectively designed to inform the development and latest stage studies, and also seek to begin clinically demonstrating the advantages of nerazacyclib’s differentiated pharmacologic profile. Dr. Mark Gelder will be speaking about these studies in more depth. So for now, I'll just remind everyone that they evaluate both a continuous dosing schedule and a three weeks, one week we off schedule. These dosing regimens mimic the drug administration schemes for those of the FDA approved CDK4/6 inhibitors.

I'm pleased to say today that each of nerazacyclib’s Phase I trials remains on track and that we expect to identify the optimal recommended Phase II dose in the second half of this year. As these trials continue to progress, the indications and treatment regimens we plan to pursue in the latest stage studies are coming into focus. While it would be premature to talk about the specifics of these studies before they are finalized, I will say now that indications, such as hormone receptor positive HER2-negative breast cancer, multiple myeloma and mantle cell lymphoma among those in addition to others that are drawing our interest.

This interest is based both on preclinical data and the fact that hormone receptor positive HER2-negative breast cancer is the only invitation in which CDK4/6 inhibitors are currently FDA approved. But with the knowledge of the unfortunate reality that resistance to the currently approved drugs are very commonly seen.

Shifting gears. I’ll now briefly mention just some high level updates on rigosertib, which targets the Ras and PLK1 pathways. Rigosertib also acts as an immune modulation due to its ability to generate novel antigens on cancer cell membranes which promotes the infiltration of autologous T cells into the tumor microenvironment and thus recognize the cancer cells as foreign and they have the opportunity to thus control their proliferation.

There are currently two investigator-sponsored studies of rigosertib ongoing with a third expected to begin by the end of June. Two of these studies seek to leverage rigosertib’s immunomodulatory affects by examining it in combination with a PD-1 checkpoint inhibitor. Last year we reported preliminary data from the first of these studies, which is in KRAS mutated non-small cell lung cancer. These data were highly encouraging as we are observed responses with different KRAS mutations, who had failed prior checkpoint inhibitor therapies.

This finding differentiates rigosertib from other approved KRAS targeted agent since responses as seen in different KRAS mutations. And thus, demonstrates rigosertib’s potential to be those KRAS mutation independent and to also overcome the mechanisms driving checkpoint inhibitor resistance in KRAS mutated non-small cell lung cancer. We look forward to the continued progress of this trial and we expect to report additional data at a major medical meeting by the end of this year.

We expect the second trial of the rigosertib checkpoint inhibitor combination therapy to begin later this quarter. This study will enroll patients with metastatic melanoma who are refractory to checkpoint blockade. I'm pleased to report today that the FDA recently approved this trial to proceed after reviewing its design, which Dr. Mark Gelder will speak about shortly.

In addition to the ongoing and planned investigator-sponsored studies I just mentioned, rigosertib is also being evaluated as a monotherapy and an investigator-sponsored study in recessive dystrophic epidermolysis bullosa, also known RDEB, complicated by squamous cell carcinoma of the skin. Unlike the two combination studies I mentioned, this trial seeks to take advantage of rigosertib’s ability to potently inhibit the PLK1 pathway.

On our last earnings call, I discussed some very exciting initial data from the trial which showed a durable complete response of over a year in an extensively pre-treated patients with RDEB as metastatic refractory squamous cell carcinoma who remains in complete remission today. Those single patient data was always be viewed with caution. It's important to realize that RDEB associated squamous cell carcinoma is an ultra-rare disease that tragically is invariably fatal.

Additionally, we think these initial data could have an important read through [indiscernible] several more prevalent squamous cell indications, which is a concept that Mark Gelder will discuss in a few moments. However, before Mark begins his contribution to today's call, I'd like to first extend my thanks to those responsible for the progress we will be discussing. This includes all of our shareholders, employees, partners and investigators. Of course, most importantly, this list also includes the brave patients who participated in our clinical trials, as well as their families. It's the unmet needs of patients that inspire the Onconova team as we work to develop our clinical programs based on expert science.

With that, I'll now turn the call over to Mark Gelder. Mark?

Mark Gelder

Thank you, Steve, and good afternoon everyone. It is my pleasure to provide an update on our recent clinical progress and future plans. Starting with a discussion of our lead nerazacyclib program. As Steve mentioned, nerazacyclib is currently being advanced in two complementary Phase I dose escalation studies in patients with solid tumors, one in the United States and one in China where we are collaborating with HanX Biopharmaceuticals. The US trial is evaluating a continuous daily dosing schedule in patients with advanced solid tumors. Enrollment remains ongoing in this trials fourth cohort, which is evaluating a 160 milligram dose of nerazacyclib administered orally each day.

The safety findings coming out of this study continue to be very promise. As we have not observed any dose limiting toxicities or clinically meaningful cases of neutropenia. Although, we are beginning to see anticipated on target effects. If the final data from our US study confirm nerazacyclib favorable safety profile with daily dosing, it will provide an important point of differentiation between nerazacyclib and the approved CDK4/6 inhibitors, palbociclib and ribociclib.

Due to their associated bone marrow toxicity that leads to neutropenia, these two drugs are administered with a three week on, one week off dosing schedule to a lot of time for bone marrow recovery. We believe that avoiding an interrupted dosing schedule with nerazacyclib could potentially contribute to enhanced efficacy. Alongside our progress in the US, we have also seen nerazacyclib’s Phase I trial in China proceeding as planned. The study continues to enroll patients into its fifth cohort, which is evaluating a 200 milligram oral dose of nerazacyclib administered each day with a three weeks on, one week off dosing schedule.

Given the favorable safety data seen in this trial to date HanX is currently preparing a protocol amendment that will allow for continued dose escalation. Thanks to the progress we've made in both of nerazacyclib’s Phase I dose escalation programs. We remain on track to identify a recommended Phase II dose later this year.

Looking ahead, we expect to utilize this dose in future trials, including a Phase II basket study enrolling patients with various cancers, such as CDK4/6 inhibitor refractory hormone receptor positive HER2-negative metastatic breast cancer. Our planned future trials also include studies that will be designed to evaluate nerazacyclib alone and in combination with other anti-cancer agents in indications where CDK4/6 inhibitors are not currently approved.

Before moving on from our discussion of nerazacyclib, I'd like to briefly highlight the robust preclinical data set supporting our development plan. Some key aspects of this data include results suggesting nerazacyclib potently inhibit CDK2, which is essential to DNA replication and one of several reported drivers of resistance to the currently available CDK4/6 inhibitor therapies. Additionally, nerazacyclib has been shown to inhibit [ARC5] (ph), also known as New Act-1 which is a kinase that promote cancer cell survival in hypoxic micro environments and serves an important role in cell adhesion and metastasis.

As Steve mentioned, nerazacyclib also has strong affinity for the CSF1 receptor, the inhibition of which leads to activation of an anti-cancer immune response. Beyond data on nerazacyclib’s inhibitory activity, our preclinical dataset also includes results indicating they it can suppress the growth of cancer cell lines that are resistant to palbociclib, which is currently the most widely prescribed CDK4/6 inhibitor. Additionally, we are also highly encouraged by more recently generated in-vitro data which shows nerazacyclib killing breast cancer cells that lack the retinal blastomere gene. This is a significant finding as the expression of retinal blastomere protein is critical to the anti-cancer activity of the current CDK4/6 inhibitors and [indiscernible] of the retinal blastomere gene is another resistance pathway.

Therefore, we believe these results further position nerazacyclib to be studied in areas beyond those where palbociclib and other anti-CDK4/6 therapies are currently approved, as well as in patients resistant the currently approved CDK4/6 inhibitors. I also mentioned that nerazacyclib was shown to cause less bone marrow toxicity than palbociclib in mirroring studies. As mentioned earlier, palbociclib’s cycle association with bone marrow toxicity that necessitates its three week on, one week off dosing schedule. Collectively, these preclinical findings support our enthusiasm for nerazacyclib’s development following the identification of the recommended Phase II dose.

Through the progression of our ongoing and planned trials, we aim to show how nerazacyclib’s differentiated pharmacologic profile may lead to improved patient outcomes. We look forward to the programs in advancement and to providing more details on our future clinical plans once they have been finalized.

Moving on, I'd now like to speak about rigosertib’s investigator sponsored program, starting with the anti-PD1 combination studies. Steve mentioned earlier, these include an ongoing Phase I/II trial evaluating rigosertib and nivolumab in combination in KRAS mutated non-small cell lung cancer and a planned study designed to evaluate rigosertib in combination with pembrolizumab in patients with refractory metastatic melanoma.

An important point to note about each of these trials is that, the inclusion criteria limits their respective patient populations to those who have previously failed therapy with a checkpoint inhibitor. Rigosertib’s potential to overcome checkpoint inhibitor resistance is supported by previously reported preclinical data highlighting its immunomodulatory effects. These data show rigosertib reversing the cold immunosuppressive tumor microenvironment that often underlie checkpoint inhibitor resistance by promoting the expression of novel antigens, such as CD40 on cancer cells. This recruits immune effector cells into the tumor, which can then be stimulated by checkpoint inhibitors to mediate cancer cells there.

We also have early clinical evidence of rigosertib’s ability to overcome checkpoint inhibitor resistance. Thanks to preliminary data from the non-small cell lung cancer trial I just mentioned. A preliminary update from the trial reported last September showed a 29% overall response rate and a 43% disease control rate. Given that all of the patients enrolled in this trial had previously failed treatment with the standard of care PD-1 checkpoint inhibitor, these data suggest that rigosertib may enhance the efficacy of anti-PD-1 therapy and strongly support the activity of the studied doublet.

I should also emphasize a point made earlier, which is that responses were observed in patients with different KRAS mutation. This sets rigosertib apart from other approved KRAS agents designed to only target patients with a particular KRAS mutation, such as G12C. Since our last update, the trials dose expansion phase has continued to enroll patients. While data from prior patients has been maturing, we expect to provide updated results from the trial later this year. And additional study that will evaluate the same rigosertib nivolumab doublet, but with potentially higher doses of rigosertib is also being considered since we have yet to reach a maximum tolerated -- tolerated dose in the current trial.

Moving on, the second investigator sponsored trial I’ll speak about today is the upcoming study designed to evaluate rigosertib in combination with the anti-PD-1 one antibody pembrolizumab in advanced metastatic melanoma. As mentioned earlier, this trial will also exclusively enroll patients who have failed prior checkpoint inhibitor therapy. There is a pressing unmet need in this population as approximately half of metastatic melanoma patients lack effective treatment options after progressing on a checkpoint inhibitor.

This is a quite frequent occurrence in this indication as 40% to 60% of metastatic melanoma patients will show acquired or primary resistance to PD-1 inhibition. To attempt to address this need we and the investigator sponsoring the trial will be collaborating on an open-label two-stage single-arm Phase II study. The primary endpoint of the trial will be overall response rate, while key secondary endpoints will include progression-free survival, overall survival and biomarker assessments intended to shed light on rigosertib ability to remodel the tumor microenvironment.

If pre-specified response criteria are met during the first stage of the trial, the study will proceed to stage two where additional patients will be enrolled. The design of the study was recently approved by the FDA and is now awaiting IRB approval at the academic center where it will be conducted. We expect this to occur later this quarter, which will allow the trial to open for enrollment.

The last investigator-sponsored study I mentioned today is the ongoing trial evaluating rigosertib monotherapy in the RDEB associated squamous cell carcinoma, which has shown a durable complete response in a heavily pre-treated patient. Since we spoke about the background of this trial and its initial data on our last earnings call, today I will focus on what we believe [indiscernible] mean from a broader perspective.

Let me start by quickly recapping the scientific rationale behind this trial. RDEB is caused by a lack of Type 7 collagen protein, which leads to extreme skin fragility and chronic wound formation. Over time many RDEB patients develop squamous cell carcinoma with the over expression of protein PLK1. This led the trials investigator who is one of the world's leading experts on RDEB to run a drug screening for inhibitors of PLK1. Of all of the agents tested in this extensive screen, rigosertib was found to be the most potent inhibitor of PLK1, suggesting it may have activity against cancer driven by PLK1 over expression. With the observed complete response in the ongoing investigator-sponsored trial, we now have clinical proof of concept supporting this hypothesis.

Given the wide array of cancers with PLK1 overexpression, we believe these recent proof of concept data highlight rigosertib’s potential to address unmet needs beyond just RDEB associated squamous cell carcinoma. The recent data is now driving conversations with key opinion leaders interested in additional investigator-sponsored studies in more prevalent indications, such as other subtypes of squamous cell carcinoma. In parallel, the ongoing trial in RDEB patients continues to advance.

Lastly, I'd like to once again emphasize that rigosertib’s clinical development will be pursued primarily through investigator-sponsored studies and that we plan to continue dedicating our internal resources primarily [Technical Difficulty].

With that, I will now let Mark begin his discussion of our recent financial results.

Mark Guerin

Thank you, Mark, and thanks to everyone who has joined today's call. I'm pleased to say that Onconova remains in a strong financial position. As of March 31, 2022, the company had cash and cash equivalents of $50.8 million, compared to $55.1 million as of December 31, 2021. Based on our current projections, we believe our current cash position will be sufficient to fund our ongoing clinical trials and business operations, including the pursuit of corporate development opportunities for more than 18 months. This runway is expected to take us through the achievement of multiple important milestones.

Turning now to a review of our first quarter financial results. Research and development expenses for the first quarter of 2022 were $2 million compared to $1.9 million for the first quarter of 2021. General and administrative expenses for the first quarter of 2022 were $2.2 million and this compares with $2.2 million for the first quarter of 2021.

We reported a net loss for the first quarter of 2022 of $4.1 million or $0.20 per share on 20.9 million weighted average shares outstanding. This compares with a net loss for the first quarter of 2021 of $4.7 million or $0.32 per share on 14.6 million weighted common shares outstanding.

This completes my financial review. I'll now turn the call back over to Steve to provide a summary of our anticipated milestones before we move on to Q&A.

Steve Fruchtman

As always, thank you, Mark. We expect to achieve several important clinical milestones over the upcoming months. Starting with nerazacyclib, we remain on track to establish its recommended Phase II dose in the second half of this year. This then enable the subsequent initiation of a Phase II basket trial and a number of important indications, including hormone receptor positive HER2-negative refractory metastatic breast cancer. With nerazacyclib we expect the investigator-sponsored trial evaluating rigosertib pembrolizumab combination therapy in melanoma to open for enrollment later this quarter. We also expect the continued advancement of the ongoing Phase I/IIA trial of rigosertib plus nivolumab in advanced KRAS mutated non-small cell lung cancer. Updated data from this study are expected with great anticipation by year's end.

Finally, we continue to assess opportunities to potentially expand our pipeline through strategic licensing and collaborations. With regards to these assets, I will emphasize two points. First, any action here will be data driven based on the science and market size underlying the opportunity. Second, we intend to take a highly selective approach in these efforts as we continue to have very strong conviction around the therapeutic and commercial potential of our current assets.

With that, we will now open the call for questions. Operator?

Question-and-Answer Session

Operator

[Operator Instructions] Our first question comes from Charles Lu with Guggenheim.

Charles Zhu

Good evening, everyone, and thanks for taking the question. Regarding CDK4/6 inhibition, to what extent is neutropenia an on target effect? And do you need to observe neutropenia in order to also see a therapeutic effect associated with your inhibitor? Thank you.

Steve Fruchtman

Mark Gelder, would you like to take that?

Mark Gelder

Sure. And thank you for the question. So neutropenia is an on target effect of CDK4/6, particularly CDK6 inhibitors. If you look at the Phase I data from palbo, ribo and abemaciclib, you'll see that neutropenia once the dose limiting toxicity or DLP associated with both palbociclib and ribociclib, and it was really the neutropenia that was responsible for the ultimate dose selected to move forward in their Phase II and III clinical trials and it was also responsible for the necessity of a three week on, one week off schedule. Abemaciclib, also a very potent CDK4/6 inhibitor also has some associated neutropenia. But if you look at the Phase I data with abemaciclib, diarrhea was their primary DLT. And because they had some [indiscernible] but it was not actually the dose limiting toxicity and the neutropenia that was observed did not necessitate a three week on, one week off dose schedule. Abemaciclib has a continuous daily dosing.

So do we anticipate seeing some neutropenia, as we continue with our dose escalation? Absolutely we do, we will not be surprised at all, we expected. Based on our preclinical data do we think that the neutropenia associated with the nerazacyclib will be as severe or profound as that with palbociclib or riboside. No, we do not. And what do we anticipate will be the DLT associated with the nerazacyclib. We don't really know yet what our DLT will be. We have not seen a DLT in our study here in the US, but do we anticipate we will see some neutropenia? Yes we do and I’m hopeful. Are we all hopeful that the neutropenia will not facilitate a three week on, one week off schedule, but the jury is still on.

Charles Zhu

Got it. Great, thanks for that color and detail.

Operator

Our next question comes from Ahu Demir with Ladenburg.

Ahu Demir

Hello team. Thank you very much for taking my question. My question will be on nerazacyclib as well. I'm curious to hear what is the extent of preclinical models we are expecting to see at ASCO? Any additional color would be much appreciated.

Steve Fruchtman

I'll ask Mark Gelder again to take that. Mark?

Mark Gelder

So we submitted an abstract to ASCO describing the kinase inhibitory profile associated with nerazacyclib, we did not submit an abstract that describes any of its activity in terms of preclinical models, such as PDX or xenograft date.

Ahu Demir

That's helpful. My follow-up question will be again on nerazacyclib. How many active sites is currently open? And do you plan to open additional sites? Curious on the enrollment status. I'm sorry, I joined late, I hope I'm not asking something you already covered.

Steve Fruchtman

Mark?

Mark Gelder

Yeah. So again, thank you for the question. In our Phase I study here in the US, we have three active Phase I sites who have all been enrolling very well. And do we anticipate opening additional sites for the dose escalation -- the Phase I dose escalation study? No, we do not. As we open additional studies as part of the basket trial, once we achieved the recommended Phase II dose, well we open additional sites for the basket studies? Absolutely, we will. Yes.

Ahu Demir

That's helpful. My last question will be, are there any rational combination of strategies you could use to pair it with nerazacyclib? Are you considering any of those given the safety profile has to match, of course, but just curious if there are any rationale matches with nerazacyclib?

Steve Fruchtman

Mark?

Mark Gelder

Yeah. So again, thank you for the question. Great question. And I think that if you look at where the currently approved CDK4/6 is, where their approval is, i.e. hormone receptor positive HER-2 negative metastatic breast cancer and you look at the combination therapies employed with the three approved agents, i.e. either a raw mutation inhibitors, surge, et cetera. We will be doing combination studies with one or more raw mutation inhibitors, we'll be doing combination studies with one or more [SERDs] (ph). We will -- we are also planning some additional combination studies that we think make a very good mechanistic sense and we will be outlining those in the future. But yes, we are planning combination studies with at least two other, if not more anti-cancer agents.

Steve Fruchtman

And just to give –

Ahu Demir

Great. Thank you very much.

Steve Fruchtman

And I would just to give Mark Gelder a break. As you know, mantle cell based on the mechanism of action of nerazacyclib and as we mentioned it is a great interest to us and based on the anticipated profile and perhaps some preclinical studies planned, drugs like ibrutinib in mantle cell are the standard of cash, so we could anticipate looking at combinations with the typical anti-mantle cell drug use, such as ibrutinib as well.

Ahu Demir

Very helpful. Thank you very much. Much appreciate you taking my questions.

Operator

Our next question comes from Joe Pantginis with H.C. Wainwright.

Joe Pantginis

Hey guys, good afternoon. Thanks for taking the question and the details today. So sticking with nerazacyclib a little bit, obviously everything we're discussing today is the completely classical route and going to identify the RP2D in the second half. So I wanted to ask my question this way, with all the initiatives that FDA, how are you going to look to reconcile, if that's the right word, the initiatives with the FDA to identify the minimal effective dose?

Steve Fruchtman

Well, I tried to give Mark a break, but I can’t. Mark Gelder, would you like to handle that.

Mark Gelder

What I can tell you is that, we have recently completed and finalized a protocol amendment for the ongoing Phase I dose escalation study, where we are going to incorporate a PD, pharmacodynamic marker, so that we will not only be looking at the safety profile with each dose escalation, but we will also be looking with the PD marker. And this will allow us not only to identify the “MTD” or maximally tolerated dose, but also a dose where we achieved maximal biological effect based on the PD market.

Joe Pantginis

Got it.

Mark Gelder

So that protocol amendment has been finalized, that's been signed and the amendment has been sent to the three sites for submission for their IRB has been submitted to the FDA as well.

Joe Pantginis

Okay. And then -- so you're talking about markers. That's a good segue for my next question. I guess earlier in your prepared comments when you talked about on target effects that you're seeing already and of course, you already have the discussion about neutropenia, any other details about what other on target affects you are seeing? And how you're measuring them?

Steve Fruchtman

Go ahead Mark. Please.

Mark Gelder

So the other on target effects that we're seeing are, again, primarily related to bone marrow and bone marrow precursors. And so looking at hematocrit hemoglobin levels looking at platelet counts, again, looking at white blood cell counts, etcetera. So we are -- this is primarily through looking at different laboratory values and the CBC is the most sensitive indicator of CDK6 inhibition.

Joe Pantginis

Got it. And then my last question, just switching gears, thanks for the patients. When you look at the potential for a Phase II in RDEB, obviously, there is no standard of care really. And I was just wondering at this point could you take any broad strokes as to the design or size of this study one would guess that it wouldn't be that big.

Steve Fruchtman

Well, I'll take that. I think actually Joe, it could be tiny. This is such an ultra-rare disease, 100 patients -- new patients [indiscernible] per year in the US, most of them die and foresee a wound and infection before they develop squamous cell carcinoma, but the fact that we have one complete remission, rate limiting step in my view is to get more patients, because of the ultra-rare nature of the disease. I don't think we would have to do a controlled Phase II trial. I think if we have few more patients we have the dose, we have the dose for few more patients on the current trial, if the responses are seen the plan would be to go directly to the FDA to ask for approved in this rare indication based on the response rate observed and how durable the response has been to date.

So that would be our plan. Get a few more patients has robust response that we've seen to date and then go to the FDA with our data and have a discussion with the FDA about an approval or what next steps would it take to get such an [indiscernible].

Joe Pantginis

Got it. Thanks a lot. And I’m looking forward to the updates later this year.

Operator

Our next question comes from Robert LeBoyer with Noble Capital Markets.

Robert LeBoyer

I'm opposed to giving Dr. Gelder a break. So I would like to ask a question about the rigosertib trial that's coming up. Based on the discussion earlier, it sounds it's going to do a combination trial of rigosertib with KEYTRUDA and metastatic melanoma. And this is going to start with a phase using a single-arm in a single center and then go to a second stage with additional patients. Could you elaborate as much as you could on the number of patients, treatment time and time for data or starting the second Phase.

Steve Fruchtman

Sure.

Mark Gelder

Steve, do you want me to handle that or do you want to go [indiscernible]

Steve Fruchtman

No please, Rob, has good taste and he sounds like he prefers you, so go ahead, Mark, please.

Mark Gelder

So the study is going to be conducted, it’s a Phase II study or Phase I2 study. It's going to be conducted at a single academic center that has a very high volume melanoma patient population. It will -- the inclusion criteria are such that, all patients will have been required to have failed prior checkpoint inhibitor therapy and by that, I mean, progressed on prior therapy. It's not going to include patients who are intolerant, because this is a combination of rigosertib and pembrolizumab. And in Stage 1 there will be 10 patients enroll. If a certain response rate is met, as defined in the protocol, it will then progress on to Stage 2. Currently Stage 2 will take place at the same institution. They feel confident that they have a very adequate patient population to enroll this trial quickly. The Stage 2 will enroll an additional approximately 20 patients.

And if all goes well and as planned and looking at the volumes that they do have, we do believe that they can meet the enrollment targets, but if all goes well, we should have data from these first stage by the first part of next year. We believe that we will have Stage 1 fully enrolled by the end of the year and then we should have some response data in the first part of next year. Remember, the primary endpoint is response rate per resist. And so, it's going to take a minimum two cycles, some patients may have responses early i.e. after one or two cycles, but it may be that we don't see responses in other patients with three or four cycles. So we won't -- we don't anticipate having results of the first stage until the first part of next year.

Robert LeBoyer

Great. Thank you very much.

Mark Gelder

But this is a -- this is going to be conducted at an academic center with a very high volume of melanoma.

Operator

I'm showing no further questions in the queue. At this time, I'd like to turn the call back to the speakers for any closing remarks.

Steve Fruchtman

Thank you, operator. I’d like to once again thank all the those participating today and for your very insightful questions. We are excited about our recent progress and look forward to the continued execution of our corporate and clinical plans as outlined today. Please stay safe and have a nice evening and take care.

Operator

Ladies and gentlemen, thank you for your participation in today's conference call. This concludes the event. You may now disconnect.

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