Traws Pharma Q3 2023 Earnings Call Transcript

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November 14, 2023

There are 10 speakers on the call.

Operator

Ladies and gentlemen, thank you for standing by. Welcome to Onconova Therapeutics Third Quarter 2023 Financial Results and Business Update Conference Call. At this time, all participants are in a listen only mode. Following management's prepared remarks, we will hold a question and answer session. Followed by number 1 on your touchtone phone.

Operator

As a reminder, this call is being recorded today, November 14, 2020 3. At this time, I would like to turn the call over to Bruce Mankle of LifeSci Advisors.

Speaker 1

Thank you, operator, and welcome everyone to Onconova's Q3 2023 financial results and business update conference call. Earlier this afternoon, Onconova issued a press release reporting its financial results and business progress. If you have not yet seen this press release, it is available in the Investors and Media section of the company's website at www dotonconova.com. Following my introduction, we will hear from Onconova's President and CEO, Doctor. Steve Fruchman Chief Medical Officer, Doctor.

Speaker 1

Victor Moyo and Chief Operating Officer and Chief Financial Officer, Mark Yerin. Onconova's VP of Global Medical Affairs and Research and Development, Mina Arora, will also be available during a Q and A session following the prepared remarks. Before we begin, I would like to remind everyone that statements made 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.

Speaker 1

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, I will now turn the call over to Onconova's President and CEO, Doctor. Steve Froman.

Speaker 2

Thanks, Bruce, and thanks to everyone who is joining us today for this call. I'll open by saying that Onconova has made excellent progress in the Q3 of 2023. During the call today, our Chief Medical Officer, Doctor. Victor Moyo and our Chief Operating Officer and Chief Financial Officer, Bob Gurn. And I will provide you with an update on our lead program, nerazacycline review the data from our recent scientific presentation, outline our upcoming milestones, provide a brief update on the rigosertib development program, summarize our 3rd quarter financial results and outlook on our cash runway and and of course importantly open the call for your questions.

Speaker 2

As you know, Our mission remains to develop novel, proprietary products for patients with hard to treat cancer and with an unmet medical need. Starting with our lead program, Our internal efforts are dedicated to these preparations for nurazaciclib, our proprietary protein kinase inhibitor of CD8.6 as well as other pioneering sulfurcellgrowthanddnasynphasis. The CDK4six class of inhibitory drugs has changed the face of cancer care in several indications, particularly in hormone receptor positive and 2 negative metastatic breast cancer. NERAZACYCLIN was designed by Onconova scientists as a potent next generation entered into this multi $1,000,000,000 drug class. In addition to its multi kinase activity, We believe niragaciclib may be differentiated from the other approved CDK4six inhibitors because of the potential for an improved tolerability profile and based on our preclinical studies improved efficacy as well.

Speaker 2

The ability to target to act on targets beyond CDK4 and 6 may lower the risk of drug resistance based by other CDK4six inhibitors. Our expansive preclinical studies have helped to define nurazaciclib's ability to impact on additional vital targets involved in cell signaling and cancer survival, including the proteins ARK5, also known as NuAct-1, CSF-1R and BUB-1, all important in how cancer establishes its presence in sites of metastatic disease as well as proliferation of the cancer cells. We have selected low grade endometrioid endometrial cancer to be our lead registration indication for nuragocycline in combination with the hormone therapy, letrozole. We believe this is the right choice in endometrial cancer for two main reasons. Number 1, The proposed registrational trial has a high technical and regulatory success probability of success.

Speaker 2

Publication of positive studies with other CDK4 and 6 inhibitors and letrozole combinations and compendia data indicating off label use support the potential for a high probability of technical success. These data and the lack of an approved label underscore the unmet medical need and support the potential for high regulatory success. The second reason is the initial data from our dose ranging studies suggest eragaciclib has the potential to be safer than the other DDK4six approved inhibitors with a wide dosing window that supports an excellent therapeutic index. Note that during our call today, we may use the acronym LG to describe this indication and endometrial cancer. To be registrational trial ready, We plan to, 1, complete the dose escalation segment of our Phase onetwo programs and define a recommended Phase 2 dose for RP2D.

Speaker 2

Secondly, engage with the FDA on the pivotal trial design under construction. Number 3, work with key external clinical experts on the design and conduct of This trial, including the Gynecologic Oncology Group or GOG and the European Network for guiding the colologic oncology trials or NGOT. Our plan is to conduct the registration trial as part of our ongoing collaboration with both of these very prestigious groups. We intend to provide more information on each of these steps over the next few quarters. As you know, there are 3 early stage studies underway in the nuragocycline program, including monotherapy and combination trials.

Speaker 2

Given nurazaciclib's multi kinase mechanism of action, we have conducted our dose escalation studies very carefully, while Victor will summarize the status of these studies based on the clinical and biological target engagement and acceptable overall safety we are observing, we have decided to dose escalate to at least 1 more dosing cohort to fully evaluate the safety profile of nurajaciclib and achieved the optimal recommended Phase 2 dose. As a result of the Phase III program for nurazaciclib may continue into the Q1 of 2024. We believe this is very good news for the program because it underscores the potential for niragaciclib to have a differentiated safety profile and a wide therapeutic index. Changing gears, I would like to touch briefly on our second program that is rigosertib. As you know, in keeping with our focus on capital efficiency, we have been exploring the clinical utility of evosertib through a series of signal finding investigator sponsored trials focused on solid tumor indications driven by legal surgeons impact on 1 of 2 mechanisms.

Speaker 2

The first is the PLK1 pathway involved in squamous cell carcinoma, complicating recessive dystrophic epidermolysis bullosa and the 2nd mutated KRAS protein seen in KRAS mutated non small cell lung cancer in combination with checkpoint inhibitors. As outlined in our second quarter call, we are focused on mapping out a registration study plan for the ultra rare indication of RDEB associated squamous cell carcinoma. Note that we will define that as RDEB associated CC in this call. We selected this to be the lead registrational indication based on the very impressive clinical responses we have seen to date in previously refractory patients and with the significant unmet medical need for this desperate patient population. As you know, we had a Type B meeting with the FDA in June.

Speaker 2

Based on this meeting and feedback from the agency And as outlined in our Q2 call, we intend to develop a protocol for a registrational trial following interactions with the rare disease group at FDA in pursuit of an orphan designation for RDEB associated squamous cell. We plan to provide an update on next steps in the first half of twenty twenty four. Looking ahead to the rest of this year and into 2024, we are focusing on achieving the following milestone. The nurazocycline, we intend to 1, continue the dose escalation segment of the Phase onetwo program, which will bring us and which may bring us into the Q1 of 2024, so we can add at least one more dosing cohort to this study. Number 2, provide a readout on their active site of safety and pharmacology in the first half of twenty twenty four once we have completed the dose escalation studies.

Speaker 2

Number 3, provide an update on our registrational trial readiness over the next couple of quarters, including the definition of the recommended Phase 2 dose, our engagement with the FDA and the pivotal trial design and obtaining rare disease indication, orphan disease indication and our work with internal external clinical experts, including the GOG and Endyte. Achievement of these milestones will also enable us to establish a solid foundation to expand the program to include other indications such as breast cancer and ovarian cancer. For rigosertib, as I just noted in the first half of twenty twenty four, we continue to plan to provide an update on the next steps to obtain orphan drug designation and for the registrational program. Before I hand the call over, I would like to warmly congratulate Victor, formally taking on the role of Chief Medical Officer and warmly welcome Nina Arroyo as Vice President of Global Medical Affairs Research and Development. Victor and I previously worked together at J&J, where he was instrumental in getting approval with some of the most revolutionary and impactful drugs in both supportive care and hematology oncology.

Speaker 2

Both of them are accomplished experts in their fields and bring significant and wide depth of experience in drug development. I believe Victor's extensive track record as a clinical researcher in drug development in oncology and MENA's unique medical affairs expertise in rare diseases and oncology will be instrumental to the company's success as we prepare the clinical plan and regulatory strategy for nurazaciclib and rigosertib. Now I would like to turn the call over to Victor to provide some more details on the neuradisciptib program. Victor?

Speaker 3

Thank you, Steve, and good afternoon, everyone. Today, I'd like to provide you with an update on the narsociclib Phase III program and touch briefly on our recent and upcoming medical meeting presentation. Starting with naresaciclib, as you may recall, there are 3 studies in the Phase III program in advanced solid tumors in patients. There's a Phase 1 monotherapy dose escalation study underway with our partner Hanex In China, is a Phase 1 monotherapy dose escalation study underway at 3 centers in the U. S.

Speaker 3

A Phase III combination dose escalation and dose expansion study, which is underway at 6 centers in the United States. This study is evaluating of increasing narsaciclib doses and a fixed 2.5 milligram per day dose of the widely prescribed anti estrogen agent, Letrozole. This study is being conducted in patients with L, weak and other gynecological tumors who are undergoing 2nd and third line treatment. As of November 13, 2023, we have dosed about 30 patients across the entire Phase III program. In the U.

Speaker 3

S. Phase 1 monotherapy study, we are dosing patients in the 7th cohort at 280 milligrams per day. In the Phase III combination study, we are starting the 2nd cohort 200 milligrams per day of narazaciclib with 2.5 milligrams per day of letrozole and have identified patients who could enroll on the next cohort. Based on the data we have observed to date, We have 3 important initial observations. Number 1, naresaciclib is safe and well tolerated to date.

Speaker 3

I'd like to make 2 points about the emerging safety characteristics of narsaciclib. 1st, based on the initial safety data pharmacokinetic studies, it may be possible to dose nirazaciclib once daily without the need for time off during treatment. This is important because it may address and or the need for a 3 week on and 1 week off dosing strategy that is required for bone marrow recovery following treatment with the most commonly prescribed CDK4six inhibitors. We believe this is an important potential feature of nirazaciclib because the requirement for a 3 weeks on, 1 week off dosing strategy may also permit tumor cell proliferation in between dosing. Secondly, initial data from our dosing studies suggest that narsaciclib may be able to avoid the difficult adverse event of diarrhea.

Speaker 3

We believe that these two distinctive characteristics may enable a wide dosing window that supports and excellent therapeutic index for narazaciclib. Number 2, we are seeing target engagement based on the observation of Grade 1 and 2 neutropenia in patients receiving a dose of at least 120 milligrams per day. Number 3, we are seeing Biological target engagement based on the results of the thymidine kinase assay at doses of 200 milligrams and above. As indicated by Steve, based on the good target engagement and overall safety that we are observing, we believe that it would be reasonable to dose escalate to at least one more dosing cohort. In order to fully explore the safety profile of nirazatript and to enable us to achieve the optimal recommended Phase 2 dose.

Speaker 3

This will extend the Phase III program for niragaciclib into the Q1. I would like to echo Steve's comment that our decision to add at least one more dosing cohort to the dose escalation study is very good news for the program because it underscores the potential for nirazaciclib to have a differentiated safety profile and wide therapeutic index. We expect to provide a readout on safety and pharmacology in the first half of twenty twenty four, once we've completed the dose escalation study. This approach will help define the optimal Recommended Phase 2 dose and we believe it is also in keeping with the FDA guidance from Project OPTIMA for dose optimization prior to approval. Also in the Q3, there were 2 medical meeting presentations related to our programs.

Speaker 3

This included presentation number 1 of Clinical data affirming the potential for rigosertib in RDEB associated squamous cell carcinoma, which was made by our colleagues from the University Hospital in Salzburg, Austria and Thomas Jefferson in Philadelphia. This data were presented as a late breaker at the European Academy of Dermatology and Veneralogy or EADV. The second presentation of promising Clinical data in combination with the of nirazaciclib in combination with Ibrutinib in the treatment of Sensitive and resistant mental cell lymphoma cell line was given at the European MCL or Mental Cell Network Lymphoma Annual Meeting. Each of these presentations underscore the biology and activity for nirazacitib and rigosertib. Looking ahead to December, we plan to present 2 additional preclinical abstract.

Speaker 3

The first one is at San Antonio at the Breast Cancer Symposium, often called SABCS. One of our collaborators will present a poster titled narsaciclib's differentiated targets and kinase inhibitory activity contribute to the enhanced inhibition of tumor growth in preclinical model. We have a second presentation at the American Society For Hematology or ASH, where our collaborators will present a poster titled narazaciclib, a differentiated CDK4six antagonist prolongs cell cycle arrest in metabolic reprogramming, enabling restoration of ibrutinib sensitivity in BTKI resistant mental cell lymphoma. We will share more of these abstracts in the coming weeks. In closing, I am optimistic about our programs, our progress and the outlook for rigosatib and narsasanquilib In 2024.

Speaker 3

For rigosertib, the promising activity that we have observed in Our DEB associated squamous cell cancer is distinct and could address a critical medical need for this ultra rare condition. These data plus its unique activity on PLK1 and KRAS pathways make rigosertib a very Interesting anticanceragent. Now that we have better defined the indications where we believe we'll be more likely to Successful based on its mechanism of action and studying cancers where these targets can be engaged by rigosertib. For niravaciclib, CDK4six inhibitors have substantially changed the face of cancer for the better. There is consistent positive clinical data on the class in endometrial cancer.

Speaker 3

In addition to target engagement, our program is showing acceptable initial safety, which could give narsaciclib a unique profile. Based on our expansive large preclinical studies, we have defined a multi kinase profile That enabled that could result in differentiated activity and the potential for broader clinical utility. These features are the cornerstone of my enthusiasm and optimism for narsaciclib. With that, I'll conclude my portion of the call and hand it off to Mark.

Speaker 4

Thanks very much, Victor, and good afternoon, everyone. Onconova closed the 3rd quarter of 2023 with cash and cash equivalents of $25,200,000 compared to $38,800,000 as of December 1, 2022. Based on our current projections, we believe our current cash position will be sufficient to fund our ongoing clinical trials and business operations into the Q3 of 2024. Research and development expenses for the Q3 of 2023 were $2,500,000 compared to $3,600,000 for the same period in 2022. General and administrative expenses for the Q3 of 2023 were $2,700,000 and this compares with $2,100,000 for the same period in 2022.

Speaker 4

Net loss for the Q3 of 2023 was $4,700,000 or $0.23 per share on 21,000,000 weighted average shares outstanding. This compares with a net loss for the Q3 of 2022 of 5,400,000 or $0.26 per share on 20,900,000 weighted average shares outstanding. The change in net loss for the Q3 of 2023 compared with the same period in 2022 was primarily result of the timing of manufacturing batch production and clinical trial expenses, partially offset by higher general and administrative costs related to our AGM in the 2023 period. From a corporate development perspective, we continue to actively engage in a range of discussions related to partnering opportunities to support the progression of our programs. With my financial review complete, I'll now hand the call back to Steve for his concluding remarks.

Speaker 2

Thank you, Mark. In closing, we are enthusiastic about the excellent progress that has been made for both miragacyclib and rigosertib in the Q3. We look forward to building on that progress as we wrap up 2023 and begin the New Year. Looking ahead, we are focused on achieving the following milestones. For our differentiated multi kinase inhibitor nirazaciclib, We intend to 1, present 2 abstracts at the December important medical meetings 2, continue the dose escalation segment of the Phase III program, which may bring us into the Q1 of 2024 3, provide a readout on Niranisypant safety and pharmacology and the first half of twenty twenty four.

Speaker 2

4, provide an update on our Phase 3 readiness Over the next couple of quarters, including definition of our recommended Phase 2 dose, our engagement with the FDA on the PIM penetration trial design and plans to work to continue our work with external clinical experts, including the gynecologic oncology group, GOG and the European Network for Gynecologic Oncology trials, ENGINE. Our plan is to conduct a registration trial as part of our ongoing collaboration with both of these groups. These milestones will provide a strong foundation for the next steps for niragocycline and with added potential indications including breast cancer, ovarian cancer and mantle seldom follow-up. For rigosertib, our program has utilized an investigator sponsored trial strategy with several solid tumor indications underway. Our main effort here is to remain focused on odd data associated squamous cell carcinoma and we continue to plan to provide an update on the next steps to obtain orphan drug designation and a registrational program in the first half of twenty twenty four.

Speaker 2

In closing, I want to recognize the diligence and dedicated work of our management team, employees, partners and investigators and most important as well as the brave and dedicated patients to participate in our clinical trials and the investment of the investment community For the support of Onconova and our very important work, we look forward to updating you on our continued progress. With that, we'll begin today's question and answer session. Operator?

Operator

One moment please for the first question. Our first question comes from the line of Charles Zhu from Guggenheim Partners. Your line is now open.

Speaker 5

Hi, everyone. This is Edward on for Charles Zhu at Guggenheim. Maybe I have to start off with just a question on how your how the Nerazaciclib plus alectrozole combination, how that trial is tracking and maybe when we could see Initial efficacy data, I think you talked about in the past with for a data update at 4Q 2023. So I'm just wondering how you're thinking about that now?

Speaker 2

Sure. Victor, would you like to take that?

Speaker 3

Yes. Thank you for that question. Right now, we are still in the process of dose Escalation, as both Steve and I mentioned, we are seeing a wider therapeutic index requiring us to go to higher doses Before we go into expansion mode. So This study will extend into 2024 as a result as well along with the monotherapy study.

Speaker 2

And just to add to what Vic just said, I want to remind everybody that the approved CDK4six inhibitors were approved not on response Prolongation of progression free survival and overall survival. The reason for that, our CDK4six inhibitors are not cytotoxic drugs. They don't cause response. That could prevent tumor proliferation, thus improvement hopefully in PFS and overall survival,

Speaker 5

Great. And maybe just a follow-up question, if I can. On the monotherapy dose escalation, it sounds like you have both the neutropenia and the thiamine kinase assay. I guess how much room based on at least on the PKPD characterization, do you think you have to keep escalating? Do you think you still have sufficient room to gain efficacy by escalating further?

Speaker 5

Or how are you thinking

Speaker 3

Yes. Ultimately, To make a decision

Speaker 2

of

Speaker 3

whether we've reached an optimal dose For the Phase 2, we would look at a combination of the safety and the PK characteristics as well as the pharmacodynamic studies. You do raise a good you do point out that since we might not With the same degree of neutropenia and diarrhea as some of the other PD4 ks inhibitors. We will be focusing on data coming from the PK Including the tolerability of course, but as well as pharmacodynamic. We have had to expand the last couple of cohorts. And usually, when you see that, it would suggest that we are close to reaching what might be Really, the optimal dose or the maximum tolerated dose.

Operator

Our next question comes from the line of Pao Demia from Ladenburg Thalmann. Please go

Speaker 6

ahead. Good afternoon, team. Thank you for taking my questions and providing helpful updates. I have Three questions on nirazulaciclib and if I have time I'll ask one more on RDEB program. My first question is has 2 portions.

Speaker 6

First one is, what how many patients are we expecting to see data from the data portion of the study? And the second part of the question is following up on adverse question. When do we expect to see any efficacy data? And given your comments Steve, on the PFS versus response rate, when would you envision the data would mature to show any clinical activity on the PFS?

Speaker 2

I'll give Victor a rest and take the second part of your question first, if I may. So without a control arm, we can the PFS, I'm going to say Palbo and letrozole in breast cancer is 8.3 months. We're not going to wait 8.3 months in what the PFS will be as part of this Phase 1 study. The purpose of the Phase 1 is to just clearly establish what the recommended Phase 2 dose should be and thus what the dose for our registration trial ultimately will be. And if our PFS is 12 months or 8 months or 10 months, whatever it may be, it's very hard to interpret that without a control army in this patient population.

Speaker 2

So the goal is really to just determine what the dose of will be in combination with letrozome for our registration trial, which clearly will have a control arm And thus we are able to compare the combination of neuroendocrine plus net result PFS to what the control arm that the FDA will tell us that they accept. So we really They see some efficacy data if there is a response. And again, these are not cytotoxic drugs, but sometimes you will see Tumor shrinkage are more likely to register not more likely the registration trial will be determined by both PFS and overall response overall survival rather than response data. And I don't remember the first part of your question, Arun. I don't know if I answered it.

Speaker 2

Would you like to repeat it?

Speaker 6

This was helpful. The first part was the safety portion of the study, how many patients, What are we going to see? Are we going to see any target engagement and other analysis as well?

Speaker 2

Okay. Victor, I think you've had enough of a rest. So could you take that please?

Speaker 3

Yes. The phase The dose escalation portion will be is driven by, of course, the number of core That we ultimately enroll. So that is a variable number and we give An approximation of where we might end up. The Phase 2 POTION is slated to enroll up to 30 patients in that Phase IIa portion.

Speaker 6

Thank you, Victor. My next question is on the safety profile. So having not much Or not high impact diarrhea or neutrophenia is new? And the information that you mentioned, Taking the 1 week off portion in the clinical setting is open label formation. What are the steps you need to take to remove that 1 week portion of?

Speaker 6

What are the discussions like with the agency? How are you going to move forward with that part?

Speaker 3

Well, first off, we are still to have our discussions with the agents. Right now, what we That in our setting is that we are dosing continuously. And as long as that safety is supported for continuous dosing. Our recommended Phase II dose will include continuous dosing, and that would be provide the basis of the discussions that we hold with the agency subsequently. So right now, we are not holding that at we are not doing the 3 weeks On 1 week on that some of the CDK4six inhibitors have to do.

Speaker 3

We are dosing continuously.

Speaker 6

I see. That's very helpful. Thank you. I'll keep my RDEB question. I'll drop in the queue to be mindful of the others.

Speaker 6

Thank you so much for answering my question.

Speaker 3

You're welcome.

Speaker 2

Thank you, I hope.

Operator

Our next question comes from Joe Pantginis with H. C. Wainwright. Your line is now open.

Speaker 7

Hi, this is Josh on for Joe. Thank you I had a question about the enrollment and how that's looking and the number of patients For the Phase 2 study for rigustertib in the CKI resistant KRAS non small cell lung cancer?

Speaker 2

Victor?

Speaker 3

Yes. We since the last update, We enrolled 3 more patients on that study. We are looking to have an date in terms of outcomes subsequently once we obtain a report from the investigator.

Speaker 7

Thank you. And for the naradociclib trial in solid tumors, Are you seeing any specific solid tumor indications like more of a certain kind or has been pretty uniform across the solid tumors After the monotherapy trial?

Speaker 3

We have enrolled a truly diverse Set of patients with solid tumors. And so there isn't a Particular pattern in the monotherapy that I can describe as standing out. Our initial Our patients tended to be more on the GI, gastrointestinal type of tumors, but there really isn't anything that stands out as being unique in terms of

Speaker 7

All right. Thank you. Thank you again for the update.

Speaker 3

You're welcome.

Operator

Our next question comes from the line of Robert D. Moore from Noble Capital Markets. Your line is now open.

Speaker 3

Since you're going to be doing an additional cohort in the neurazociclib solid tumors, what is your projected Start date for Phase 2 at this point.

Speaker 2

So again, to give Rick Victor a rest, I'll take that. So That's a great question, Robert. If we see additional DLTs In 2023, at the 280 milligram cohort, we may be done. If not, Again, we may go into the Q1 to establish the recommended Phase 2 dose. We can't meet with the agency until we know the dose of the registration trial because they want to see the safety of that dose.

Speaker 2

So we have to go into the first Quarter of 'twenty four, to establish the recommended Phase 2 dose and meet with the agency, We estimate that the registration trial will begin in first half of twenty twenty four in low grade endometrial cancer.

Speaker 3

Okay. And if there are no DLTs in the cohort to be added, Would you add another second cohort in 2024?

Speaker 2

It's always hard to predict these things, It depends on what we see regarding as Victor explained, we have a pharmacodynamic marker, The timing kinase marker, we already see engagement and as we dose escalate more engagement, We know we're engaging in the kinase involved stem cell proliferation, because we see some mild neutropenia. It really depends, it also depends on the PK, are we maxing out as we dose escalate, is PK going up or is it staying at a maximum level, so there's no need to just escalate. So all those things Victor alluded to go into the decision. Are we done at 280? Meaning do we have DLTs at 280, which means Recommended Phase 2 dose would be 2 40.

Speaker 2

So we're seeing enough evidence that 280 is safe with target engagement with From PK to claim that 280 will be the recommended Phase 2 dose. So those to say it's easier to answer that question with data that And hypothetically, I answered it to the best of my ability.

Speaker 3

Okay, great. Thank you very much.

Operator

Our next question comes from the line of James Molloy from Alliance Global Partners. Your line is now open.

Speaker 8

Hi, good afternoon. Thank you for taking my question. My question is on the rigosertib, Getting the orphan designation, I know that we discussed before some of the challenges on that. Can you walk through sort of the steps you have to take we get the rigoservosortumorphan designation and how the clinical trial plan might look, should you get that?

Speaker 2

Sure. And Nina, as our RDEB squamous cell expert An expert in rare diseases, would you like to take that question of how we introduce the concept Of odd dead squamous cell to the Orphan Group and FDA? Nina? Maenie is calling in From the U. K, perhaps she was the connection was lost.

Speaker 2

So I'll take that, if I may. I need to go this is clearly an orphan disease, right? There are 60 to 100 patients. Right. There are 60 to 100 patients per year that develop RDEB in the U.

Speaker 2

S. Once squamous cell develops the patients, 50% of the patients by within 2.5 years. So clearly, this is an orphan disease. Our goal at the orphan group Explain to FDA why this is different than what I'll call spontaneous squamous cell, For instance, sun damaged squamous cell. It's different because 1, RDEB squamous cell develops In late teenage years, early 20s, some damaged squamous cell develops in the elderly.

Speaker 2

Some damaged squamous cells are very good prognosis usually removed surgically and that's the end of the therapy required. Our DEBS squamous cell is a very aggressive disease as I just mentioned. And the cynical known that differentiates spontaneous squamous So from IDEV squamous cell is the fact that squamous cell complicating IDEV has a mutation of the collagen 7 gs, which spontaneous squamous cell does not have. So our goal at The Orphan Group is to explain why this disease rdebs squamous cell is different in common squamous cell, which in fact is not by their definition is often disease. Clearly, our DEFCWAY missile is and our goal is to interact with the agency to explain In greater detail what I just said.

Speaker 9

Apologies, my line dropped. Thank you for answering that. I've got no further things to add apart from we have also presented last month The EADB meeting is a late breaking abstract, and we'll be taking learnings from the initial patient experience and moving that into the clinical development registrational program as well and into discussions. So in addition to the discussion we've mentioned about the orphan drug designation, we'll be also doing that. Apologies, my line dropped there.

Speaker 2

The media did sound so much informed and I did only based on accents in my view. Thank you.

Speaker 8

Thank you very much for that. I guess the one follow-up question will be, is it more sort of a Yes, check various boxes or do you have to go make a presentation and sort of pitch them on the disease or is it just kind of a form to fill out? How does that sort of mechanically work?

Speaker 2

We'll basically do it anyway the organization group wants us to. But hopefully, there'll be a chance for an interaction with them. We think that's much more value. The FDA offering group will make that call. You will request a face to face meeting.

Speaker 2

My understanding is they're doing most of these virtually And they may just respond to a written briefing book on why we believe we deserve off of this. It's up to them.

Speaker 8

Thank you for taking the questions.

Operator

I'm showing no further questions in the queue. Ladies and gentlemen, thank you for your participation on today's conference call. This concludes today's event. You may now disconnect.

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Key Takeaways

  • Nurazaciclib is advancing as a differentiated next‐generation CDK4/6 inhibitor with multi‐kinase targeting and early safety data suggesting continuous once‐daily dosing and lower diarrhea risk, with Phase 1/2 dose escalation extended into Q1 2024 to establish the recommended Phase 2 dose.
  • The lead registration trial for nurazaciclib in low-grade endometrioid endometrial cancer plus letrozole is being designed in collaboration with FDA, GOG and ENGOT to maximize technical and regulatory success.
  • Rigosertib is poised for an orphan designation and registrational study in ultra-rare RDEB-associated squamous cell carcinoma, building on investigator-sponsored trial signals and FDA rare disease feedback, with an update due in H1 2024.
  • Financials: Q3 2023 cash was $25.2 million (runway into Q3 2024), R&D spend fell to $2.5 million, and the net loss narrowed to $4.7 million (–$0.23/share) versus a $5.4 million loss in Q3 2022.
  • Upcoming milestones include December 2023 abstracts at SABCS and ASH, a Q1 2024 nurazaciclib safety/pharmacology readout, and FDA engagement on pivotal trial readiness.
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