Relmada Therapeutics Inc (RLMD) 2025 Q4 法說會逐字稿

Good afternoon and welcome to the Relmada Therapeutics fourth-quarter and full-year 2025 earnings conference call. (Operator Instructions) As a reminder, this conference call is being recorded and will be available for replay on the Relmada website.

I would now like to turn the call over to Brian Ritchie from LifeSci Advisors. Please go ahead, Mr. Ritchie.

Good day, everyone, and thank you for joining us today. This afternoon, Relmada issued a press release providing a business update and outlining its financial results for the three months and year-ended December 30, 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, Relmada'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.

These forward-looking statements are qualified by the cautionary statements contained in Relmada's press release issued today and the company's SEC filings, including in the annual report on Form 10-K for the year ended December 31, 2025, filed after the close today.

This conference call also contains time-sensitive information that is accurate only as of the date of this live broadcast on March 19, 2026. Relmada 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 Relmada's CEO, Dr. Sergio Traversa, who will briefly provide a summary of recent business highlights; Dr. Raj Pruthi, Relmada's CMO Oncology, who will provide an NDV-01 program update and Relmada's CFO, Maged Shenouda, who will provide an update on sepranolone and a review of the company's Q4 financial results. After that, we will open the line for a brief Q&A session.

Now I would like to hand the call over to Sergio Traversa. Sergio?

Thank you, Brian. Good afternoon, and welcome, everyone, to the Relmada fourth-quarter and year-ended 2025 conference call. 2025 has been a transformational year for Relmada, marked by significant progress for our lead program, NDV-01. As a reminder, NDV-01 is a sustained release formulation of gemcitabine and docetaxel. We are developing this investigational product candidate for the treatment of non-muscle invasive bladder cancer or NMIBC.

Most recently, we reported compelling responses and durable 12 months efficacy data for our ongoing Phase 2 study of NDV-01. We achieved FDA alignment for our planned registration of Phase 3 RESCUE programs. We fortified our team, and we substantially strengthened our balance sheet.

As we reflect on our recent accomplishments and planned next steps, I would like to highlight four key areas. First, NDV-01. We believe that the strength of the recently reported 12 months follow-up data could position NDV-01 as a potential best-in-class therapy for the treatment of NMIBC. Furthermore, the strength of the clinical data and the unique easy-to-administer sustained-release formulation gives us confidence that NDV-01 has the potential to provide what urologists and patients with NMIBC need a simple, durable effective treatment that readily fits into real-world practice setting.

We plan to initiate the Phase 3 RESCUE program in the middle of this year. Our Phase 3 regulatory strategy agreed upon with the FDA includes two independent registrational pathways. Pathway one is focused on adjuvant therapy following TURBT in patients with intermediate-risk bladder cancer, which affects about 75,000 patients in the United States. Pathway two is focused on second-line treatment BCG unresponsive patients, which represent about 5,000 patients in the United States.

Second, sepranolone. Sepranolone has previously demonstrated proof of concept in Tourette syndrome. This is a disorder characterized by compulsive behavior. We are getting ready to begin a proof-of-concept study in Prader-Willi syndrome in the middle of this year.

And third, our team. We substantially strengthened our development team with the appointment of Dr. Raj Pruthi, a highly regarded physician, scientist and urologic oncologist as Chief Medical Officer, Oncology. In addition, we established a scientific Advisory Board comprised of similarly distinguished peers to further support the NDV-01 program, Dr. Yair Lotan from the University of Texas Southwestern Medical Center; and Dr. Max Kates from John Hopkins University School of Medicine.

Fourth and last, financial strength. We just completed a successful EUR160 million private financing. Based on existing forecasts, these funds, plus our existing cash balance, provide Relmada with capital through 2029 and importantly, through the completion of the planned NDV-01 program.

Looking ahead, 2026 is poised to be another important year of value creation for Relmada, with the initiation of our Phase 3 RESCUE program for NDV-01 in bladder cancer, and the Phase 2 proof-of-concept trial for sepranolone in Prader-Willi syndrome.

With that, I also would like to express my appreciation for the trust and support of our investors, employees, collaborators and the patients who participate in our studies.

Next, I will turn the call over to Dr. Raj Pruthi, who will provide a review of the NDV-01 program, including 12 months follow-up data from the ongoing Phase 2 study and the summary of our Phase 3 plans. Raj?

Thank you, Sergio. Good afternoon, everyone. It's a privilege to share an update on the clinical progress we've made this year, headlined by the truly compelling and best-in-class results for NDV-01 in NMIBC.

Bladder cancer is a high-frequency cancer that has a major impact on the lives of patients. generally diagnosed in their early- to [mid-70s]. High recurrence rates and burdensome treatment disrupt quality of life at a time when patients are eager to enjoy life.

I want to touch on three topics during today's call. One, an overview of the NDV-01 12-month data; two, a summary of our planned Phase 3 program; and three, a discussion of how NDV-01 might fit into the practice of a urologic [oncology].

Sergio noted that NDV-01 is a novel sustained release intravesical formulation of two chemotherapy agents, gemcitabine and docetaxel or gem/doce, as we say. Our program builds on physicians establish familiarity with the efficacy and safety profile of conventional gem/doce. More specifically in patients who are unresponsive to BCG, this combination offers a salvage, a bladder-sparing option, that may help avoid a radical cystectomy.

Moving on to the 12-month data. We are pleased to report that NDV-01 has demonstrated a high response rate and durable 12-month efficacy from the ongoing Phase 2 study. We believe these data stand out in comparison to the other benchmark programs and I could position NDV-01 as a best-in-class treatment option for patients with bladder cancer, if approved.

The study is an open-label single-arm trial in patients with high-risk NMIBC. Patients receive biweekly doses every other week times six, followed by monthly maintenance for up to one year. Patients undergo regular assessments with cystoscopy, typology and, if needed, biopsy.

The study was designed to enroll up to 70 patients with high-risk NMIBC. The primary endpoints are safety and complete response rate at 12 months, [secondary] endpoints or duration of response and event-free survival. The data demonstrated a 12-month complete response rate of 76% with a favorable safety profile.

Notably, the study also showed a 12-month complete response rate of 80% in the BCG [unresponsive] population, one of the most difficult-to-treat segments of NMIBC. These findings support the advancement into the Phase 3 registrational program, which we are calling RESCUE. The program will evaluate NDV-01 in both second-line BCG unresponsive diseases and in intermediate risk or cancer as an adjuvant therapy following TURBT.

When you look at the complete responses, or CR, at any time, in the overall population, we see a CR any time of 95% based on 38 patients. Among those with BCG and responsive disease, we see a CR rate at any time of 94%. Given the [burdensome] nature of recurrent bladder cancer treatment [BCG] is a critical element of our product profile. We continue to be encouraged by the favorable safety profile observed for NDV-01 across our clinical program.

In the 12-month data set for NDV-01, no patients had progression to muscle-invasive disease. No patients underwent a radical cystectomy. No patients had a Grade 3 or higher treatment-related adverse events. No interruptions or discontinuations or treatment due to adverse events occurred. And most treatment-related adverse events were, I think, Grade 1 level.

Moving on to the planned Phase 3 RESCUE program. We believe our 12-month response and durability data compare [a] points favorably to the current commercial and development stage platform. We have constructed our Phase 3 registrational pathways to maximize our probability of success and create the most efficient path to FDA approval.

The RESCUE registration program was designed in alignment with the FDA to provide two separate approval pathways. We expect to secure a US IND clearance and initiate the Phase 3 RESCUE program in the middle of this year.

Let's review the two studies that form the RESCUE program. Registrational pathway one focuses on the evaluation of NDV-01 in patients with intermediate-risk bladder cancer as an adjuvant therapy following TURBT surgeries. We estimate there are about 75,000 patients each year in the US in [esthetics]. This study is planned to be an open-label, randomized controlled trial. As there are no approved treatments for adjuvant intermediate risk NMIBC, the study will evaluate NDV-01 versus observation. The primary endpoint is disease-free survival, or DFS.

Secondary endpoints included high-grade recurrence-free survival, progression-free survival and quality of life metrics. We feel that the opportunity to incorporate NDV-01 into patient care post TURBT is very attractive, and it could pave the way for important clinical indication and broader adoption.

Registration pathway number two is focused on the valuation of NDV-01 in the second-line setting in patients who are BCG unresponsive with carcinoma in situ, or CIS, for refractory to first-line therapies approved or in development. We estimate that there are about 5,000 patients per year in the US in this set.

These [few] patients have few if any effective treatment alternatives to radical cystectomy. The study is designed as a single-arm open-label trial. The primary endpoint is CR any time. Secondary endpoints will include the duration of response, or DOR, progression-free survival and recurrence-free survival amongst responders. We expect to report the initial three-month response data from this study by the end of 2026. We're excited about this pathway because it could offer a rapid route to approval.

Before I hand the call over to Maged, I'd like to make a note of how the NDV-01 [might] in clinical practice. NDV-01 is formulated to create a soft matrix in the bladder to enhance local bladder urothelial exposure and minimize systemic toxicity. It is delivered in the office in less than five minutes. This simple formulation and administration model has the potential to optimize the delivery experience for patients and providers offering a level of simplicity and time savings that stands up amongst the others.

Before I hand the call over to our CFO, Maged Shenouda, our Phase 2 data gives us high confidence in our registrational program. By addressing a clear unmet need with a unique sustained delivery profile, we believe NDV-01 is uniquely positioned to redefine the standard of care in bladder cancer. We look forward to initiating the RESCUE registrational program at an estimated 80 sites in North America in the middle of this year as we work to bring NDV-01 in [broader] cancer patients as soon as possible.

Maged?

Thanks, Raj, and good afternoon, everyone. Today, I'll spend a few minutes on sepranolone and then provide you with an overview of our fourth-quarter 2025 financial results. Because supranalone modulates GABA, one of the most important neurotransmitters, it is defined as a GAMSA, or GABA modulating steroid antagonist.

Sepranolone's novel action on the GABA neurotransmitter pathway gives it the potential to normalize the activity of the GABA A receptor and alleviate the repetitive symptoms of compulsivity disorders. These disorders affect millions of people around the world and include indications such as obsessive compulsive disorder, Tourette syndrome and Prader-Willi syndrome.

We are preparing to initiate a proof-of-concept study in Prader-Willi syndrome in mid-2026. Our immediate efforts are dedicated to completing study preparations, including engaging with the FDA on our proposed trial design and establishing a robust supply chain.

Moving now to our financial results. As noted earlier by Brian, this afternoon, Relmada issued a press release announcing our business and financial results for the fourth quarter and 12 months ended December 31, 2025. During this call, I'll review our fourth-quarter 2025 financial results and refer you to our press release and 10-K filing issued this afternoon for financial information for the last 12 weeks.

Starting with our cash balance. Relmada closed 2025 with a cash balance of $93 million. This includes net proceeds of approximately $94 million from an underwritten stock offering announced on November 5, 2025. This compares to cash, cash equivalents and short-term investments of approximately $45 million at December 31, 2024.

On March 9, 2025, the company announced a $160 million of private financing with net proceeds of approximately $150 million. This financing, along with our cash balance as of December 31, 2025, is expected to provide sufficient resources to fund company operations through 2029, including completion of the Phase 3 RESCUE program for NDV-01.

Moving through our fourth-quarter financial results. Research and development expense for the three months ended December 31, 2025, and totaled $8.1 million compared to $11 million for the three months ended December 31, 2024, a decrease of $2.9 million. The decrease was primarily driven by a decrease in study costs associated with the completion of two Phase 3 trials for REL-1017, partially offset by increased costs related to the startup of the Phase 3 NDV-01 trials in Phase 2b sepranolone study and additional R&D personnel.

General and administrative expense for the three months ended December 31, 2025, totaled $12.3 million compared to $8.1 million for the three months ended December 31, 2024, an increase of approximately $4.2 million. The increase was primarily driven by an increase in compensation costs partially offset by a decrease in stock compensation costs.

Net cash used in operating activities for the three months ended December 31, 2025, totaled $14.6 million compared to $8.8 million for the three months ended December 31, 2024. The net loss for the three months ended December 31, 2025, was $19.9 million or $0.27 per basic and diluted share compared to a net loss of $18.7 million or $0.62 per basic and diluted share for the three months ended December 31, 2024.

Before we open the call for questions, I'll turn it back to Sergio for some closing comments. Sergio?

Thank you, Maged. In closing of our prepared remarks, I would like to share that I'm very confident and optimistic about our clinical programs and the long-term prospects for Relmada. As we are getting ready to initiate the RESCUE registrational program for NDV-01, we are focused on execution and looking forward to updating you on our progress in the coming quarters.

Operator, I would now like to open the call for questions.

(Operator Instructions) Uy Ear, Mizuho Securities.

Congrats on the great data and the pipe. It seems like you guys did a lot of work this quarter. So maybe there's been getting some questions on whether you will present additional data from your Phase 2 study. Should we kind of expect going forward maybe updates every three months? And will you also have data, I guess, add AUA by any chance? So that's the first question.

And the second question that we've been kind of getting is, there's been -- I guess, some investors are a little bit concerned that the second-line patients may not be necessarily second line, but maybe by the time they get to your regimen, it could be their third line. Maybe just help us understand how -- what you're doing exactly to ensure that those patients are truly second-line patients.

And the third question is you indicated that you have three months data from your Phase 3 BCG unresponsive second line in responsive patients. Will this be from the entire patient population? Or would this be sort of interim and is part portion of the number of patients that you expect to enroll?

Thank you, Uy, for the questions. I think Raj is -- you can answer these questions. I pass it to Raj.

Yeah. Thank you, Serge. Good to hear for you. Regarding the data, we will be presenting an updated data this 12-month data at the AUA. It has been acceptance of that. We'll also be presenting a trial in progress as we get RESCUE going.

Our plan is to focus on introducing data -- and I think this is your last question, in the BCG responsive second-line group starting at the end of the year. So as we start the trial in the midyear, we should have some three-month data to be able to share externally by the end of the year as a single-arm open label. And our thought is to then actually, at a cadence of about every three months, share the data as we get 6, 9 and 12 months on that data set.

I think you provided an excellent question on second line, third line, fourth line. And we are indeed limiting the number of prior therapy lines to two. So you can have one live therapy at silicon, for example, and while allowing the second of those [still be] followed by ANKTIVA. We'll also allow a maximum of two. We're also going to look at 15 patients at three months of those who received one first-line therapy versus two, just to make sure there -- it's an open-label study that there's nothing concerning that we want there, those should be excluded.

And this is reflective of the conversations we've had with the FDA. But I think it's a good question. We made a number of kind of third or fourth lines.

Farzin Haque, Jefferies.

Congrats on the progress. So I had one on the operational standpoint. NMIBC space is becoming congested with active trials and drugs approved too. So like what is your expectation for enrollment cadence across your two studies? And can the drug in office profile potentially serve as a recruitment advantage?

Thank you, Farzin. Raj, do you want to take that?

Yeah, yeah. Thank you. Yeah, Farzin, good to hear from you. I think you're right. It's -- I think the high-risk BCG unresponsive has been a crowded space, but I think our is coming in as a second-line therapy provides a unique advantage and there's no drugs that have been approved in that setting and none that I'm aware of that are even being investigated as a pivotal study in that setting. So I think we have a competitive advantage and that we can go to sites that even in development drugs in development and follow them in this unique path.

And same for intermediate risk. I think right now, there's -- it's becoming a bigger expansive interest. But what I've seen in, for example, CG Oncology has an intermediate risk trial that looks like it's ahead of schedule and accrued very rapidly. I think there's a lot of interest in -- from investigators and looking at intermediate risk patients. I think what we -- I expect us to enrolled that pretty rapidly ahead of schedule like CT did.

Thanks for the question, Farzin.

Got it. And then for the second-line, high-grade fittings, beyond the primary endpoint of CR rate at any time, has the FDA stipulated a minimum duration of follow-up required for all patients by just submitting the NDA?

Another great question. They haven't required a minimum of follow-up. They said they want us to CR any, as you said, and durability of response or duration of response. I think the wording to use what I think is important is they've kind of seen the totality of the data.

So they want to see do you have a response? And is there some level of durability? I mean they haven't specified what that number is.

Christopher Liu, Lucid.

Thanks for the question. I was just wondering, given the population differences between your Phase 2 and Phase 3 RESCUE, what are you expecting to see in terms of the CR rate at the three-month mark as well as what we should be benchmarking against with the status?

Thank you, Chris. Raj, do you want to take this one as well?

Yes, sure. Yeah. Thanks, Chris. Thanks for the question. So in the intermediate risk, we are looking at -- well, we structured the trial to look at a -- the statistics around it, a two-year RFS of 75%. And that actually is reflected in the literature with [Jetsen]. And I think with what we've seen in this population and with sustained release, we should exceed that, but that's our target number that drives the statistics. It's an event-driven study. So that's kind of the 128 events as a target.

Regarding the BTG on responses to line, it is -- we have looked at that, what is the -- it's interesting that in first line, the first drug approved was valrubicin in 1998 at 8% 12 months CR, followed by KEYTRUDA and 19% [at shown] 24%.

So I think -- I'm glad that there is not -- the FDA wasn't fixated on a certain number. But I think that number should be at those levels or lower than what we've seen in first-line [better] as a precedent if that makes sense.

(Operator Instructions) Kelsey Goodwin, Piper Sandler.

Congrats on the recent clinical update. Regarding, I guess, specifically to that update regarding the patient baseline characteristics and the CIS versus papillary split, I guess how should we be comparing this data set to that of competitors with primarily CIS patients there? And I guess any data to support that gem/doce similar in CIS in papillary patients.

Kelsey, Sergio here. Raj, it seems that this one also is for you.

Yeah. Great question, Kelsey. Regarding -- so starting with the last part of your question first, there is -- in fact, it's interesting as a clinician, you often think, okay, the patient is BCG unresponsive, this might be more virulent. But we don't [at think is] CIS versus papillary. Some people show pure [CIS] behaves better than T1 papillary were. So it's a mixed bag.

It's actually interesting for gem/doce, there's a hard hold by Steinberg in 2020 in the Journal of Urology that looked at heavily pretreated, basically BCG [thoughtful] patients. And at 12 months, the RFS in those patients are CRs with 60% in the CIS population and 61% in pathways. So there isn't a market difference typically in between that.

But even if you go back and look at our data of what we've showed, ours at 12-month is 80%, 12-month came at 84%. And that does include four patients with at 12 months for patients with CIS. We had four out of four with complete response at any time, two out of two or 12 months.

Completely understood these are small numbers, but I think it still compares quite favorably. Even if you take NDV-01 and the BCG unresponsive papillary, our entire population, including CS, and compare it to the best-in-class papillary, which is in J&J's INLEXZO, I think their 12-month TAM was 74%. So [even taking our entire over], we have -- we're significantly higher. So I think it's still best in class. I hope I answered your question.

Yeah. No, that's super helpful. And then maybe just one follow-up, if I can. With respect to the intermediate risk setting and in that kind of market overall, I guess, how much do you think that market might need to be built out by these early launches, just given we haven't had an approved agent until last year.

Kelsey, another great question, if you don't mind me jumping in Sergio.

No, no, go ahead.

Right now -- I mean, we mentioned it's about 75,000 to 80,000 patients with intermediate risk disease. And if you look at the data analysis, only about 35% of those patients will receive adjuvant therapy. What's important in our studies and in CV study is that in our interest population, we do include small less than 3-centimeter [PA] high-grade patients.

I think that's very important because 20% of the intermediate risk disease in these high-grade PA patients, and those are the ones that probably more than anybody needs adjuvant therapy to prevent recurrence. So we go back to (technical difficulty) to [35%] receive an adjuvant therapy. I think it's exactly what you're alluding to.

Right now, there isn't an approved therapy. There isn't a lot of data. And there is a lot of agents that are -- can be reimbursed to the urologist in a buy and build model, for example. So I think that 35% number is going to only grow as you've seen data from [moon rise rest of] our intermediate study as we get data and gives patients confidence that, hey, here is an agent that might reduce my risk of having another TURBT and give your relative confidence that I have an agent that I can deliver in my office that will reduce TURBT risk for live patients. It's only going to grow.

Thank you. This concludes our question-and-answer session. I would now like to hand the floor back over to Sergio Traversa for any closing remarks.

Well, closing remarks is a big thank you to everybody that is allowed Relmada to get where we are now. We create data with a drug that can really help patient with bladder cancer. So looking forward to update everybody on our progress. Thank you, and enjoy the rest of the day.

Thank you.

This concludes today's conference. You may disconnect your lines at this time. Thank you again for your participation.