Relmada Therapeutics Q1 2025 Earnings Call Transcript

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May 12, 2025

There are 7 speakers on the call.

Operator

Good afternoon. Welcome to Ramada Therapeutics First Quarter twenty twenty five Earnings Call. At this time, all participants are in a listen only mode. After the prepared remarks, we will conduct a question and answer session. As a reminder, this conference is being recorded and will be available for replay on the location website.

Operator

I would like to turn the call over to Brian Ritchie from LightSideAvaya. Please go ahead Mr. Ritchie.

Speaker 1

Good day, and thank you everyone for joining us today. This afternoon, Ramada issued a press release providing a business update and outlining its financial results for the three months ended 03/31/2025. Please note that certain information discussed on the call today is covered under the Safe Harbor provision of the Private Securities Litigation Reform Act. We caution listeners that during this call, Ramada's management team will be making forward looking statements. Actual results could differ materially from those stated or implied by these forward looking statements due to risks and uncertainties associated with the company's business.

Speaker 1

These forward looking statements are qualified by the cautionary statements contained in Realmada's press release issued today and the company's SEC filings, including in the annual report on Form 10 ks and today's Form 10 Q for the quarter ended 03/31/2025 filed after the close today. This conference call contains time sensitive information that is accurate only as of the date of this live broadcast, 05/12/2025. Realmada undertakes no obligation to revise or update any forward looking statements to reflect events or circumstances after the date of this conference call. With me on today's call are Realmada's CEO, Doctor. Sergio Traversa, who will briefly provide a summary of recent business highlights and Realmada's CFO, Maga Cinauda, who will provide a review of the company's Q1 financial results.

Speaker 1

After that, we will open the line for a brief Q and A session. Now, I would like to turn the call over to Sergio Traverso. Sergio?

Speaker 2

Thank you, Brian, as always, and good afternoon and welcome everyone to the Renmada first quarter twenty twenty five conference call. Twenty twenty five is off to a good start for Renmada. We added two unique product candidates with very encouraging Phase two data and large addressable markets to our portfolio NDV-one for bladder cancer and sopranolone for Prader Willi syndrome, Tourette syndrome and potentially other CNS indications. Reported initial proof of concept Phase two data for our lead product candidate NDV-one at the American Urology Association. And we made progress towards our objective of bringing each program to patients as soon as possible with preparation underway to begin the next set of studies for NDV-one and Cefrano.

Speaker 2

With two innovative product candidates that have shown promise to improve a concept data, 27,000,000 cash balance, clean balance sheet and disciplined approach to development plan, we are in a good position to advance our pipeline to important clinical milestones. During today's call, I will provide a snapshot of our two programs, including a review of the initial Phase two data for NDVYOD-one at the AUA meeting two weeks ago. After that, Maged will review our financial results. I will make a few closing remarks and then we will take your questions. We also invited on the call today Doctor.

Speaker 2

Yair Lota, Chief of Udo Oncology at the University of Texas Southwestern Medical Center in Dallas who can answer your clinical question regarding NDV-one. We are encouraged by the potential of the diversified pipeline that we are building at Valvada. Starting with NDV-one, we believe the program is an excellent fit with our strategic plan and has the potential to meaningfully improve the care of patients with bladder cancer. Our decision to in license NDV-one was based on strong science, strong field data, and the anticipation of positive phase two data at the upcoming American Urology Association meeting or AUA twenty twenty five. I'm pleased to report that positive top line proof of concept data presented at AUA twenty twenty five supported our initial enthusiasm for NDV-one's potential to be the class leading bladder sparing chemotherapy for non muscle invasive bladder cancer.

Speaker 2

During today's call, I will touch on the market opportunity, the mechanism of action, the data and the next steps. Starting with the market, sources indicated that there are about seventy five thousand new cases of bladder cancer diagnosed each year in The US. About half or fifty percent of those cases have high grade disease that has a high risk of recurrence. It is a very high recurrence rate for the six hundred thousand people approximately in The US living with bladder cancer. Moving to mechanism of action, NDV-one is a novel sustained release intravesical formulation of two chemotherapy agents, gemcitabine and docetaxel or gemdosing.

Speaker 2

Endo one forms a spherical soft matrix within the bladder that sequesters gemdoce and releases these two agents as a matrix gradually dissolves. The formulation was specifically designed to maximize local gem dosage concentration and also provide prolonged exposure while minimizing systemic toxicity. Published clinical studies have shown that gemcitabine and docetaxel achieves a response rate and recurrence free survival that comparable to or better than the historical standard of care, bacillus calmetgarin or BCG. However, the administration of conventional chemotherapy is cumbersome. The two chemotherapy agents require special handling and preparation in a controlled hospital pharmacy setting.

Speaker 2

In addition, the two chemotherapies administer sequencing over three to five hours with limited tumor exposure time. In contrast, NBV-one sustained release formulation is intended to be dosed in office as a ready to use therapy that is administered in less than ten minutes without the need for anesthesia or new or dedicated equipment. What is really exciting about NDVO data is the data presented at AUA twenty twenty five two weeks ago. The presentation was based on data from an ongoing single arm, single center ex US Phase two study evaluating NBV-one in patients with high grade MIBC. Twenty six patients have been enrolled as of the data of the last data cut off.

Speaker 2

The AUA presentation was based on the results for the first twenty patients. The group included two patients with carcinoma in situ CIS and eighteen patients with papillary disease TA and T1. Of the papillary disease patients, eight were BCG naive and twelve were BCG unresponsive. The efficacy data were presented based on three and six months assess. In addition, the highest response rate at any time point was also reported.

Speaker 2

Based on the three months assessment, dosing of NDV-one resulted in overall response rate of eighty five percent or seventeen out of twenty patients. High grade recurrence free survival in patients with papillary disease of eighty three percent or fifteen out of eighteen patients. A complete response in calcium in situ patients recognizing that the number is small was 100 or two out of two patients. For data reported at any time point, the overall response rate was ninety percent or eighteen out of twenty patients. High grade recurrence free survival in papillary disease was eighty nine percent for sixteen out of eighteen patients.

Speaker 2

Complete response in carcinoma in situ patients remains one hundred percent for two out of two patients. Importantly, seven patients were evaluated at six months. One hundred percent of these patients achieved disease free status. This group includes one patient with CIS and six patients with papillary disease characterized as TA or T1. One of these patients was retreated at three months and responded to the second treatment.

Speaker 2

From a safety perspective, NDV-one was well tolerated with no treatment related adverse events greater than grade one. We were very pleased with the reception of the data received at AUA. We believe that the results suggest that NDV-one has the potential to significantly improve the care of patients with NM IVC. NDV-one is currently in continues the Phase two single arm study to assess safety and efficacy in patients with high grade non muscle invasive bladder cancer. Our goal is to bring NDVO-one to patients as soon as possible.

Speaker 2

Looking ahead to the second half of twenty twenty five, our effort will focus on securing a U. S. IND clearance. Turning briefly to Cipranolone. In February, we acquired the rights of Cipranolone from Azerina Pharma.

Speaker 2

Our decision was based on Cipranolone's broad safety database and promising Phase two results in Tourette syndrome. I would like to touch on four topics for Cefrano, the market opportunity, the mechanism of action, the data and the next steps. Starting with the market, we believe Cefrano is well suited to treat disorders marked by compulsive behavior and excessive activity of the GABA neurotransmitter pathway, including Prader Willi syndrome and Tourette syndrome. The assumed neurobehavioral disorder can manifest through repetitive behavior and positivity and represents sizable underserved markets. Prader Willi syndrome or Prader Willi is our first candidate indication for soprano.

Speaker 2

Prader Willi is a complex genetic disorder often defined by persistent anger and overeating hyperphagia. Current treatment is focused on improving obsessive compulsive behavior and other medical complications. Prader Willi is estimated to affect approximately three hundred and fifty thousand people worldwide, including approximately twenty thousand people in The US. Turning to the mechanism of action, Ceprano is first in class endogen neurosteroid. It's a member of a new subgroup of neurosteroid called GABA modulating steroid antagonist.

Speaker 2

GABA selectively act on GABA to alleviate the repetitive symptom of compulsive disorder. We were attracted to Zepranolone because of its unique mechanism of action and promising proof of concept data. Phase two, the results from the originator AZARINA showed that Zepranolone demonstrated a competitive peak reduction of 28% with a P value of 0.051 in its primary clinical endpoint as measured by the YGTSS standardized to Rett scale. The data also showed that sopranolone treatment produced an improved quality of life without any off target CNS effects. These data provide a strong foundation to study sopranolone in compulsion related disorders such as PWS or Prader Willi syndrome.

Speaker 2

Our effort to progress Zepranilone are expected to include planned FDA interaction and further development of product supply with plans to advance into clinical development in early twenty twenty six. Now I would like to turn the call over to our Chief Financial Officer, Megan Chenouda to talk about our financial results. Megan.

Speaker 3

Thanks, Sergio. With two innovative product candidates that have shown promising proof of concept data, a $27,100,000 cash balance, a clean balance sheet and a disciplined development plan, we're in a good position to advance our pipeline milestones. Turning to our financial results. As noted by Brian, this afternoon, we issued a press release announcing our business and financial results for the first quarter ended 03/31/2025. As of 03/31/2025, Realmada had cash, cash equivalents and short term investments of approximately $27,100,000 compared to $44,900,000 as of 12/31/2024.

Speaker 3

Cash used in operations in the first quarter ended 03/31/2025, was $18,100,000 compared to $13,000,000 for the same period in 2024. Our efforts in 2025 are dedicated to advancing NDV-one and sopranolone through key development milestones. Over the coming months, as we finalize our clinical and regulatory strategy for each program, we expect to have better visibility into our requirements and runway. Moving through our first quarter twenty twenty five financial results. Research and development expense for the first quarter twenty twenty five totaled $12,000,000 compared to $13,300,000 for the first quarter of twenty twenty four, a decrease of $1,300,000 The lower spend was primarily driven by lower study costs, with the completion of clinical trials for REL-ten seventeen for major depressive disorder, offset by payments for the sopranolone acquisition and the NDV-one in licensing.

Speaker 3

General and administrative expense for the first quarter of twenty twenty five totaled $6,300,000 compared to $9,700,000 for the first quarter of twenty twenty four, a decrease of approximately 3,400,000 The decrease was primarily driven by a decrease in stock based compensation expense. The net loss for the first quarter of twenty twenty five was $17,600,000 or $0.58 per basic and diluted share compared with a net loss of $21,800,000 or $0.72 per basic and diluted share for the first quarter of twenty twenty four. Before we open the call for questions, I'll turn back to Sergio for some closing comments. Sergio?

Speaker 2

Thank you, Magid. I would like to leave you with these key messages from today's call before we enter in the Q and A section. 2025 is off to a strong start with the addition of two unique product candidates with proof of concept Phase two data and large addressable market to our portfolio. NDV-one for bladder cancer and sopranolone for Prader Willi syndrome and Tourette syndrome. Reported positive initial proof of concept Phase two data for our lead product candidate NDVU-one at AUA, and we made progress toward our objective of bringing each program to patient as soon as possible.

Speaker 2

With preparation underway to begin the next set of studies for NDV-one and Siprano. With two innovative product candidates, they've shown promising proof of concept data, 27,000,000 cash balance, a clean balance sheet, and the disciplined approach to development plan, we are in a good position to advance our pipeline to important clinical milestones. As we prepare to advance our two clinical program, we want to thank our investors for your support and for taking time to join today's call. Operator, I would like now to open the call for questions.

Operator

Thank you. Our first question is from Oi Eir with Mizuho Securities. Please proceed.

Speaker 4

Hey, guys. Yeah. Thanks for taking our questions, and congrats on the quarter, the recent data. So, maybe the first question from us is, you indicate you'll be approaching the FDA to speak for them to, in order to move forward. I guess what gives you confidence that the current data from the Phase two study would be sufficient for the FDA to agree for NDV-one to move into registrational study.

Speaker 4

I guess the second question maybe is, you indicate that you're going to scale up supply. Could you sort of elaborate what you mean by that? Are they commercial products? Are they scaling up for clinical study only? Thanks.

Speaker 2

Thank you, Oi, for the question. Let me answer the first one, and I believe Doctor. Lawton, should have joined the call and, anything you would like to add would be very welcome. But what make us confident that the conversation with the FDA will drive like the beginning or the start of registration of a program. But the couple of things, one is the combination of drug itself, right?

Speaker 2

Gencitabine plus docetaxel has been used, has been currently used and has been used for some time by many, many urologists everywhere. And everybody is convinced about the efficacy and the safety of the local administration of two drugs. The reason that there's not been more widely used is the limitation in the practicality. The very few doctor office can prepare So it has to be prepared by pharmacies authorized used to handle chemotherapy, most of the cases in clinics.

Speaker 2

So, and the administration that requires a sequential just either been in docetaxel one after the other, and it takes time. So you have to keep by the doctor office or the clinic occupied for three, four, five hours and for the patient too, because he has to sit in the clinic, holding and for one or two hours, each of the two preparations. That's even if a patient affected bladder cancer is willing to go through a lot to avoid to take the bladder off is still a convoluted process. And so that's one of the reasons that we feel confident that the combination of chemotherapy is not new, is well known is in use and is recognized as one of the most effective, not the most effective, pharmacological treatment of bladder cancer. The second one that comes from these data and, is the safety of the formulation.

Speaker 2

We have no one single patient interrupting the study for side effect and all the side effect registered all grade one. So it seems it's very, very, well tolerated. And so you put the two things together and the advantage also of the administration in the doctor office, no anesthesia less than ten minutes is a prefilled syringe that doesn't need any handling. So all the things together should make the FDA like willing to let us go into a larger registration study. Of course, there is always until we get the direct the minutes from the FDA, you cannot never be sure, but, we believe there is a very good chance that we'll be okay with that.

Speaker 2

And I know if Doctor. Lozano has been able to join the call. He was in a surgery. Yes,

Speaker 5

good afternoon. Can you hear me okay?

Speaker 2

Yes, absolutely. Very well. Thank you. So

Speaker 5

I think I can address the issue to some degree. First of all, intravesical chemotherapy has been routinely used for treating both intermediate risk and high risk bladder cancer for decades. Now it's interesting because the therapies that are currently used, mitomycin, gemcitabine, docetaxel, are all being used as off label use, but but they are, reimbursed and commonly utilized. The biggest challenge for you all to know is that you need a hood to mix these formulations. And unless you have a cancer pharmacy, you can't give it in your office.

Speaker 5

Immune therapies like BCG come in a vial and a powder that you can reformulate, but intramedical chemotherapy, you can't. Medical oncologists who give IV doses of the chemotherapy are not typically giving intravesical therapies in their offices. They're not familiar with placing catheters. There's little reimbursement. And so you have a bit of a catch point too.

Speaker 5

If you're a patient, you can't really get it into your urologist's office, and you can't get it at your medical oncologist's office. Now the formulation of gemcitabine, docetaxel, is actually one of the more commonly used drugs in BCGN responsive, which is a space with a lot of development between nattoferagene and Keytruda and TAR two hundred and Crittostimogene. In ANKiva, there's a lot of drugs being developed in this space, and yet many people are still using gemcitabine, docetaxel because the other drugs are well, some of them are not approved yet. Some of them are more problematic to give in the clinic. But many patients are kind of out in the cold.

Speaker 5

They're not able to get access to these drugs, either the newer drugs or drugs like gemcitabine, docetaxel. Now in terms of efficacy, as mentioned, there are many studies looking at intravesical chemotherapy and demonstrating efficacy. However, we know that formulations like TAR two hundred, which elute over three weeks, work better than single agents. And I think that this combination, which has advantage of both being easy to deliver and sustainable release in the bladder over two weeks, will be superior, potentially over agents that stay in your bladder for just one hour. So there are several, potential advantages for this both in terms of ease of use and the potential, increased efficacy.

Speaker 2

Thank you, Doctor. Lawton. Did I answer your question?

Speaker 4

Yeah. So, maybe just some follow-up on what you guys said in response to potential differentiation. So, Doctor. Loudon, if you're still there, maybe one of the feedbacks that we've gotten from investors is that this is kind of a crowded market. So, just help us understand how you see NVD-one fit in the treatment paradigm when it comes to market.

Speaker 4

Have BCG, you have CG oncology and other potential competitors who could be ahead? Thanks.

Speaker 5

Right. I think yeah, I'm happy I'm happy to respond. First of all, you know, I think that, you know, if you ask patients, they want to keep their bladder. And in the BCG unresponsive space, which I completely agree, there's probably three or four potential treatments, know, TAR two hundred and creatosimogene, both will likely be accepted by the FDA. But nonetheless, patients are frequently gonna want two or three lines of therapy, and, they're gonna want to get sort of the most efficient, effective treatments.

Speaker 5

I don't necessarily think that that's gonna be the best first place to go with with this drug, mainly because as you say, it's gonna be a bit of a busy space even though I suspect that since many people are already using gemcitabine, docetaxel as their main treatment off label if they actually have an approved compound that they're familiar with, with durable, excretion of drug and an easier mechanism of delivery, then they'll be very open to giving that drug that they're familiar with over some of the other agents. But, another hand in the intermediate risk space, which is has a higher prevalence by far than the BCGN responsive place, there really aren't drugs, that are commonly used. Intravesical gemcitabine is you know, is available, not approved, but available. But as I said, it's hard to get access to. So and academic centers, we give intravesical chemotherapy, but many community sites don't.

Speaker 5

And so it it would be a very natural fit to give intravesical chemotherapy, such as this formulation for intermediate risk patients. It has potential in the chemo ablative space as well, which even though that's not sort of a a place that we commonly use drugs, but, UGN one zero two is doing a did a chemoablation trial, and it's going to FDA. And it's a single drug, mitomycin. This is actually a combination, which I think could potentially compete nicely if it has a good performance. And there's a bridge trial comparing gemdosi to BCG that's being enrolled right now.

Speaker 5

And if it shows equivalence or superiority, then this drug could fit in the BCG naive space. And the other drugs that you're mentioning, TAR two hundred, Crudo Simogene are not competing in that space. And the trials that have been completed with BCG and checkpoint inhibitors have shown Crest has been reported, had about a seven percent increase reduction reduction in recurrence at eighteen months, but about a fifteen percent rate of grade three SAEs and no improvement in progression, no improvement in survival. So I don't think any of the checkpoint inhibitors are gonna compete in the BCG naive space. But if the BRIDGE trial shows equivalence of efficacy, this drug could actually fit in the BCG naive space without much competition from some of these newer agents.

Speaker 5

I see many potential uses right now.

Speaker 2

Okay. Thank you. Thank you, doctor Lawton. And, wait, your second question was regarding the manufacturing and sorry. Was for, sepranolone or for NDV01?

Speaker 2

For the NDV

Speaker 4

o Right.

Speaker 2

Yeah. The the clearly, the quantity needed for commercial will be large. So we'll like, we will and we we always want to have two manufacturer at minimum. So we are looking for like capacity and a second manufacturer for like risk management. It's not a complicated product to make.

Speaker 2

It's gel and so they're all known components.

Speaker 4

Okay, thank you.

Speaker 2

Thank you, Wei. Our

Operator

next question is from Andrew Tye with Jefferies. Please proceed.

Speaker 6

Good afternoon. This is Matt Barcus on for Andrew. Thanks for taking our questions. I guess regarding the latest dataset for NDV01 presented at the AUA meeting earlier, when should we look forward to you sharing the complete response rate for the entire population? And how do you anticipate sharing the future updates from the program?

Speaker 6

Like what more can we look forward to in those datasets throughout the year? And what are your expectations for success?

Speaker 2

Well, can answer part of it and maybe Doctor. Lothan can expand. So the next data point will be the six months. We had seven patients now at the AUA with a 100% complete response. We'll have six months data of the 20 patients somewhere around June, July.

Speaker 2

We'll present that and then date that and then we'll give a nine and twelve months of the 20 patients. And so these are the expectation of the data like the, we can only look at what we have now that is like ninety percent at three months and one hundred percent of the seven patients at six months, but they look pretty good. And no surprise because it's known that the combination JENDOZA has very efficacious. And even if like with the duration of tumor contact of a couple hours, we use the same dose and it stays there for ten days and is done six times in three months. So the expectation that the results are good are definitely there.

Speaker 2

And you want to add something, Doctor. Luton?

Speaker 5

No. I I you know, I think this is, obviously, you know, an interesting cohort from an efficacy standpoint. I actually think the the more important, aspect of it is actually the safety standpoint because a lot of there's a lot of data about efficacy of gem dosage formulations, and we know that, you know, you could look even at tar 200 data and see what happens when you give gemcitabine over a sustained period of time. But the safety is actually the more important component because you worry whether or not prolonged exposure of the bladder to the chemotherapy might cause, you know, irritation, frequency, urgency, pain. And so far, we haven't seen that, and that's probably if you had asked me at the beginning of this, what would that be my biggest concern?

Speaker 5

It would not have been a efficacy concern. It would have been a safety concern. And so that's probably the most reassuring aspect of this. It's a heterogeneous population, so it's gonna be a little challenging to compare this to some of the mature trials like SUNRISE one or BOND three in terms of efficacy because only some of these patients have CIS BCGN responsive CIS. This is still, you know, phase two with a heterogeneous population.

Speaker 5

But, at some point, obviously, you know, once the you know, after conversation with FDA, we can decide on which indication to actually do a a larger cohort. But, but the safety profile is obviously quite reassuring.

Speaker 2

Thank you. Thank you, doctor Lotham. Did I answer your question? That was the first part that I didn't catch entirely.

Speaker 6

No, yeah, yeah, you caught it. Thanks.

Speaker 1

Oh, you.

Operator

Then I guess

Speaker 6

like as you're thinking about talking with FDA on with these data and the design of the phase three, I guess what would you want the phase three to look like in terms of time points, endpoints and the types of patients you're thinking about enrolling?

Speaker 2

Doctor. Lothan that here you can add a lot of value because Doctor. Lothan is helping us very closely to design the the phase three program. Do you wanna answer that?

Speaker 5

Sure. I think there's, you know, there are easier routes and there are harder routes. I think somebody highlighted the challenge with the BCG unresponsive route. The benefits of that route are that you that the FDA has approved single arm phase two trials for approval in CIS alone to look at efficacy. The challenge is that these patients are relatively rare, and it takes many sites and quite a bit of time to enroll.

Speaker 5

I think there's two easier routes. One route would be to go through a single arm chemoablation route, similar to what UroGen with the Envision trial. I think we're gonna learn a lot later on this month when it goes to ODAC. And if if the drug their combination gets approved with a single agent mitomycin that stays in your bladder about four hours, and then a single arm trial in that setting with our formulation makes a lot of sense. It'd be probably the quickest route to approval.

Speaker 5

And if if that doesn't if there's reasonable rationale from the FDA that they won't approve such an approach, then a randomized trial like PIVOT-six, an intermediate risk, randomizing NDV-one to placebo or observation would probably be the next quickest route. That trial actually enrolled extremely quickly in The US. I think that this formulation would actually be more attractive than an oncolytic virus, but that type of trial design was enrolling very rapidly. They're almost done with enrollment, and I think somewhere around fifteen to eighteen months. And I think that would be the next approach, especially since the FDA approved a randomization against placebo, which is easy to do a superiority trial against nothing in a population of patients who have high risk for recurrence.

Speaker 6

Great. Yeah, thanks for the color.

Speaker 2

Thank you.

Operator

There are no further questions at this time. So this will conclude today's conference. You may disconnect your lines at this time and thank you for your participation.

Speaker 2

Thank you all. Thank you very much. Thank you, Doctor. Lodan.

Speaker 5

Thank you.

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Key Takeaways

  • Ramada added two novel candidates—NDV-one for non-muscle invasive bladder cancer and sopranolone for Prader-Willi and Tourette syndromes—after in-licensing and acquisition, bolstering its pipeline with large addressable markets.
  • In its Phase 2 proof-of-concept trial, NDV-one achieved an 85% overall response rate at three months (83% recurrence-free in papillary disease; 100% complete response in CIS) and was well tolerated with no ≥Grade 2 adverse events.
  • NDV-one’s ready-to-use intravesical gel allows office-based, 10-minute dosing—avoiding complex pharmacy prep and prolonged administration—while maximizing local drug exposure and minimizing systemic toxicity.
  • The company plans to secure U.S. IND clearance and engage the FDA on a registrational trial, exploring both single-arm chemoablation and randomized intermediate-risk study designs to accelerate approval.
  • Financially, Ramada ended Q1 with $27.1 million in cash, reported a net loss of $17.6 million (vs. $21.8 million YoY), and continues to allocate disciplined R&D spending toward key pipeline milestones.
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