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Thursday, Mar. 19, 2026 at 4:30 p.m. ET
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Relmada Therapeutics (NASDAQ:RLMD) reported that clinical progress for NDV01 includes durable, high 12-month complete response rates in both broad and BCG-unresponsive NMIBC populations, with favorable safety data. The company confirmed FDA-aligned registrational design and projected timelines for both an intermediate-risk and a BCG-unresponsive bladder cancer pathway, outlining competitive differentiation in patient selection and clinical positioning. Major financing steps have extended the operational runway past program milestones, and leadership enhancements include the addition of clinical and advisory experts to support late-stage execution.
With me on today's call are Relmada Therapeutics, Inc.'s CEO, Dr. Sergio Traversa, who will briefly provide a summary of recent business highlights; Dr. Raj S. Pruthi, Relmada Therapeutics, Inc.'s CMO, Oncology, who will provide an NDV01 program update; and Relmada Therapeutics, Inc.'s CFO, Maged S. Shenouda, who will provide an update on sopranolone 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?
Sergio Traversa: Thank you, Brian. Good afternoon and welcome everyone to the Relmada Therapeutics, Inc. Fourth Quarter and Year End 2025 Conference Call. 2025 has been a transformational year for Relmada Therapeutics, Inc., marked by significant progress for our lead program NDV01. As a reminder, NDV01 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-month efficacy data for our ongoing Phase II study of NDV01. We achieved FDA alignment for our planned registrational Phase III RESCUE programs. We fortified our team and we substantially strengthened our balance sheet.
As we reflect on our recent accomplishment and planned next steps, I would like to highlight four key areas. First, NDV01. We believe that the strength of the recently reported 12-month follow-up data could position NDV01 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 give us confidence that NDV01 has the potential to provide what urologists and patients with NMIBC need: a simple, durable, effective treatment that readily fits into real-world practice settings. We plan to initiate the Phase III RESCUE program in the middle of this year. Our Phase III 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 in BCG-unresponsive patients, which represents about 5,000 patients in the United States. Second, sopranolone. Sopranolone has previously demonstrated proof of concept in Tourette syndrome. It 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 S. 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 NDV01 program: Dr. Yair Lotan from the University of Texas Southwestern Medical Center and Dr. Moussa “Moe” Ates from Johns Hopkins University School of Medicine. Fourth and last, financial strength. We just completed a successful $160,000,000 private financing. Based on existing forecasts, these funds plus our existing cash balance provide Relmada Therapeutics, Inc. with capital through 2029, and importantly, through the completion of the planned NDV01 program.
Looking ahead, 2026 is poised to be another important year of value creation for Relmada Therapeutics, Inc., with the initiation of our Phase III RESCUE program for NDV01 in bladder cancer and the Phase II proof-of-concept trial for sopranolone 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 S. Pruthi, who will provide a review of the NDV01 program, including 12-month follow-up data from the ongoing Phase II study and the summary of our Phase III plans. Raj?
Raj S. Pruthi: Thank you, Sergio. Good afternoon, everyone. It is a privilege to share an update on the clinical progress we have made this year, headlined by the truly compelling and best-in-class results for NDV01 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 treatments 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 NDV01 12-month data. Two, a summary of our planned Phase III program. And three, a discussion of how NDV01 might fit into the practice of urologic oncology.
As Sergio noted, NDV01 is a novel, sustained-release intravesical formulation of two chemotherapy agents, gemcitabine and docetaxel, or gem-dose, as we say. Our program builds on physicians' established familiarity with the efficacy and safety profile of conventional gem-dose. More specifically, in patients who are unresponsive to BCG, this combination offers a salvage, bladder-sparing option that may help avoid a radical cystectomy. Moving on to the 12-month data, we are pleased to report that NDV01 has demonstrated a high response rate and durable 12-month efficacy from the ongoing Phase II study. We believe these data stand out in comparison to other benchmark programs that could position NDV01 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 six biweekly doses every other week times six, followed by monthly maintenance for up to one year. Patients undergo regular assessments with cystoscopy, pathology, 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 are 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 III registrational program, which we are calling RESCUE. The program will evaluate NDV01 in both second-line BCG-unresponsive disease and in intermediate-risk bladder 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-unresponsive disease, we see a CR rate at any time of 94%. Given the burdensome nature of recurrent bladder cancer treatment, safety is a critical element of our product profile. We continue to be encouraged by the favorable safety profile observed for NDV01 across our clinical program.
In the 12-month data set for NDV01, 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 event, no interruptions or discontinuations of treatment due to adverse events occurred, and most treatment-related adverse events were at the grade 1 level. Moving on to the planned Phase III RESCUE program. We believe our 12-month response and durability data compare quite favorably to the current commercial and development-stage therapies. We have constructed our Phase III registrational pathways to maximize our probability of success and create the most efficient path to FDA approval.
The entire RESCUE registration program was designed in alignment with the FDA to provide two separate approval pathways. We expect to secure U.S. IND clearance and initiate the Phase III RESCUE program in the middle of this year. Let us review the two studies that form the RESCUE program. Registrational pathway one focuses on the evaluation of NDV01 in patients with intermediate-risk bladder cancer as an adjuvant therapy following TURBT surgery. We estimate there are about 75,000 patients each year in the U.S. in this setting. This study is planned to be an open-label, randomized controlled trial. Since there are no approved treatments for adjuvant intermediate-risk NMIBC, the study will evaluate NDV01 versus observation.
The primary endpoint is disease-free survival, or DFS. Secondary endpoints include high-grade recurrence-free survival, progression-free survival, and quality-of-life metrics. We feel that the opportunity to incorporate NDV01 into patient care post-TURBT is very attractive, and it could pave the way for an important clinical indication and broader adoption. Registrational pathway number two is focused on the evaluation of NDV01 in the second-line setting in patients who are BCG-unresponsive with carcinoma in situ, or CIS, or refractory to first-line therapies approved or in development. We estimate that there are about 5,000 patients per year in the U.S. in this setting.
Since these 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 among responders. We expect to report the initial three-month response data from this study by the end of 2026. We are excited about this pathway because it could offer a rapid route to approval. Before I hand the call over to Maged, I would like to make a note about how we feel NDV01 might fit into clinical practice.
NDV01 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 out among the others. And I will hand the call over to our CFO, Maged S. Shenouda. Our Phase II data give us high confidence in our registrational program. By addressing a clear unmet need with a unique sustained delivery profile, we believe NDV01 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 NDV01 to bladder cancer patients as soon as possible. Maged?
Maged S. Shenouda: Thanks, Raj, and good afternoon, everyone. Today, I will spend a few minutes on 2025 financial results. Because sopranolone modulates GABA, one of the most important neurotransmitters, it is defined as a GABA, or GABA-modulating steroid antagonist. Sopranolone's novel action on the GABA neurotransmitter pathway gives it the potential to normalize the activity of the GABAA 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's 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 Therapeutics, Inc. issued a press release announcing our business and financial results for the fourth quarter and twelve months ended December 31, 2025. During this call, I will review our fourth quarter 2025 financial results and refer you to our press release and Form 10-K filing issued this afternoon for financial information for the last twelve months. Starting with our cash balance. Relmada Therapeutics, Inc. closed 2025 with a cash balance of $93,000,000.
This includes net proceeds of approximately $94,000,000 from an underwritten stock offering announced on 11/05/2025. This compares to cash, cash equivalents, and short-term investments of approximately $45,000,000 at 12/31/2024. On 03/09/2025, the company announced a $160,000,000 private financing with net proceeds of approximately $150,000,000. This financing, along with our cash balance as of 12/31/2025, is expected to provide sufficient resources to fund company operations through 2029, including completion of the Phase III RESCUE program for NDV01. Moving through our fourth quarter financial results. Research and development expense for the three months ended 12/31/2025 totaled $8,100,000 compared to $11,000,000 for the three months ended 12/31/2024, a decrease of $2,900,000.
The decrease was primarily driven by a decrease in study costs associated with the completion of two Phase III trials for REL-1017, partially offset by increased costs related to the start-up of the Phase III NDV01 trials and Phase 2b sopranolone study, and additional R&D personnel. General and administrative expense for the three months ended 12/31/2025 totaled $12,300,000 compared to $8,100,000 for the three months ended 12/31/2024, an increase of approximately $4,200,000. 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 12/31/2025 totaled $14,600,000 compared to $8,800,000 for the three months ended 12/31/2024.
The net loss for the three months ended 12/31/2025 was $19,900,000, or $0.27 per basic and diluted share, compared to a net loss of $18,700,000, or $0.62 per basic and diluted share, for the three months ended 12/31/2024. Before we open the call for questions, I will turn back to Sergio for some closing comments. Sergio?
Sergio Traversa: Thank you, Maged. In closing of our prepared remarks, I would like to share that I am very confident and optimistic about our clinical programs and the long-term prospects for Relmada Therapeutics, Inc. As we are getting ready to initiate the RESCUE registrational program for NDV01, 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: Thank you. We will now be conducting a question-and-answer session. If you would like to ask a question, please press 1 on your telephone keypad. One moment, please, while we poll for questions. Our first question is from Uy Sieng Ear with Mizuho Securities.
Uy Sieng Ear: Hey, guys. Congrats on the great data and the PIPE. Yeah. It seems like you guys did a lot of work this quarter. So maybe, you know, maybe we are getting some questions on whether you will present additional data from your Phase II study. Should we kind of expect, going forward, maybe updates every three months? And will you also have data, I guess, at AUA by any chance? So that is the first question.
And the second question that we have been kind of getting is there has 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 what you are doing exactly to ensure that those patients are truly second-line patients. And the third question is, you indicated that you have three-month data from your Phase III BCG-unresponsive second-line unresponsive patients. Will this be from the entire patient population, or would this be sort of interim and as partial or a portion of the number of patients that you expect to enroll? Thanks.
Sergio Traversa: Thank you, Uy, for the questions. I think Raj can answer these questions. I pass it to Raj.
Raj S. Pruthi: Yeah. Thank you, Sergio. Good to hear from you, Uy. Regarding the data, we will be presenting updated data, this 12-month data, at the AUA. It has been accepted to that. We will 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-unresponsive second-line group, starting at the end of the year. So as we start the trial in midyear, we should have some three-month data to be able to share externally by the end of the year as it is a single-arm, open-label study.
And our thought is to then actually, at a cadence of about every three months, share the data as we get six-, nine-, and twelve-month follow-up on that data set. I think you provide 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 had one line of therapy and still have been, for example, BCG-unresponsive, while allowing a second line of those to be intravesical chemotherapy. We are also allowing a maximum of two prior lines.
We are also going to look at 15 patients at three months for those who received one prior line of therapy versus two just to make sure there is nothing concerning that would suggest that those should be excluded, and this is reflective of the conversations we have had with the FDA. I think it is a good question. Limiting the number of kind of third or fourth lines.
Uy Sieng Ear: Thank you. Right. Thank you.
Operator: Our next question is from Farzin Hak with Jefferies. Congrats on the progress, and thank you for taking my questions.
Farzin Hak: So I have one on the operational standpoint. The NMIBC space is becoming congested with active trials and drugs approved too. So are there, like, what is your expectation for enrollment cadence across your two studies? And can the drug’s in-office profile potentially serve as a recruitment advantage?
Sergio Traversa: Thank you, Farzin. Raj, do you want to take that?
Raj S. Pruthi: Yeah. Thank you. Yeah, Farzin. Good to hear from you. I think you are right. I think the high-risk, BCG-unresponsive setting has been a crowded space, but I think ours, coming in as a second-line therapy, provides a unique advantage. And there are no drugs that have been approved in that setting and none that I am aware of that are even being investigated as a pivotal study in that setting. So I think we have a competitive advantage in that we can go to sites that, even with drugs in development, can follow them in this unique path. And same for intermediate risk. I think right now, it is becoming a bigger, expansive interest.
But what I have seen, for example, CG Oncology has an intermediate-risk trial that looks like it is ahead of schedule and accrued very rapidly. I think there is a lot of interest from investigators in looking at intermediate-risk patients. So I expect us to enroll that pretty rapidly, ahead of schedule like CG did. Thanks for the question, Farzin.
Farzin Hak: Got it. And then for the second-line high-grade settings, 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 submitting the NDA?
Raj S. Pruthi: Another great question. They have not required a minimum of follow-up. They said they want to see CR any time, as you said, and durability of response, or duration of response. I think the wording they used, which I think is important, is they want to see 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 have not specified what that number is.
Farzin Hak: Got it. Thank you so much.
Operator: Thank you. Thank you, Farzin. Your next question is from Christopher with Lucent.
Christopher: Hey, guys. Thanks for the question. I was just wondering, given the population differences between your Phase II and Phase III 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?
Sergio Traversa: Thank you, Christopher. Raj, do you want to take this one as well?
Raj S. Pruthi: Yes. Sure. Yeah. Thanks, Christopher. Thanks for the question. So in the intermediate risk, we are looking at, and we have structured the trial to look at, the statistics around a two-year RFS of 75%. And that actually is reflected in the literature with gem-dose. I think with what we have seen in this population and with sustained release, we should exceed that, but that is our target number. That drives the statistics. It is an event-driven study. So that is kind of the driver. And with 128 events, it is the target.
Regarding the BCG-unresponsive second-line, it is interesting that in first line, the first drug approved was valrubicin in 1998 at 8% 12-month CR, followed by Keytruda at 19% and then other agents at 24%. So I think I am glad that the FDA was not fixated on a certain number. But I think that number should be at those levels or lower than what we have seen in first-line therapy, just as a precedent. If that makes sense. Thanks for the question.
Christopher: Got it.
Operator: Our next question is from Kelsey Goodwin with Piper Sandler.
Kelsey Goodwin: Hey, thanks so much for taking my question, and congrats on the recent clinical update. Regarding, I guess, specifically to that update, regarding the patient baseline characteristics and the CIS versus papillary split, how should we be comparing this data set to that of competitors with primarily CIS patients there? And any data to support that gem-dose looks similar in CIS and papillary patients?
Sergio Traversa: Hi, Kelsey. It is Sergio here. Raj, it seems that this one also is for you.
Raj S. Pruthi: Yeah. Great question, Kelsey. Starting with the last part of your question first. It is interesting as a clinician, you often think, okay, if the patient is BCG-unresponsive, this might be more virulent. But we do not often, I think, ask is it CIS versus papillary? Some people show pure CIS behaves better, but T1 actually worsens outcomes. So it is a mixed bag. It is actually interesting for gem-dose. There is an article by Steinberg in 2020 in the Journal of Urology that looked at heavily pretreated, basically BCG-unresponsive patients, and at twelve months, the RFS in those patients, or CRs, was 60% in the CIS population and 61% in papillary.
So there is not a marked difference typically between them. But even if you go back and look at our data of what we showed, ours at twelve months is 80%, twelve-month CR at 84%. That does include four patients with CIS. We had four out of four with complete response at any time, two out of two at twelve months. Completely understood these are small numbers, but I think it still compares quite favorably. Even if you take NDV01 and the BCG-unresponsive population, our entire population including CIS, and compare it to the best-in-class categories, I think their twelve-month CR was 74%.
So even taking our entire cohort, we have significantly higher, so I think it is still best in class. I hope I answered your question.
Kelsey Goodwin: Yeah. No. That is super helpful. Thank you so much for that. And then maybe just one follow-up if I can. With respect to the intermediate-risk setting and that kind of market overall, how much do you think that market might need to be built out by these early launches, just given we have not had an approved agent until last year? Thanks so much.
Raj S. Pruthi: You bet, Kelsey. Another great question. Right now, we mentioned it is about 75,000 to 80,000 patients with intermediate-risk disease. And if you look at the data now, only about 35% of those patients will receive adjuvant therapy. What is important in our studies and in CG’s study is that in our intermediate-risk population, we do include small, less than three-centimeter Ta high-grade patients. I think that is very important because 20% of the intermediate-risk disease is these high-grade Ta patients, and those are the ones probably more than anybody who need adjuvant therapy to prevent recurrence.
So if we go back to what we call about 35% receiving an adjuvant therapy, I think it is exactly what you are alluding to. Right now, there is not an approved therapy. There is not a lot of data. There are not a lot of agents that can be reimbursed to the urologist in a buy-and-bill model, for example. I think that 35% number is going to only grow as you see data from programs like Moonlight, or from PIVOT-006, or from our intermediate study as we get data.
It gives patients confidence that, hey, here is an agent that might reduce my risk of having another TURBT and gives urologists confidence that I have an agent that I can deliver in my office that will reduce TURBT risk for my patients. It is only going to go up.
Kelsey Goodwin: That is great. Thank you so much.
Sergio Traversa: Thank you, Kelsey.
Operator: 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.
Sergio Traversa: Well, the closing remarks is a big thank you to everybody that has allowed Relmada Therapeutics, Inc. to get where we are now. We created data with a drug that can really help patients with bladder cancer. We are looking forward to updating everybody on our progress. Thank you, and enjoy the rest of the day. Thank you.
Operator: This concludes today's conference. You may disconnect your lines at this time. Thank you again for your participation.
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