Relmada Therapeutics Inc (RLMD) 2025 Q3 法說會逐字稿

Good afternoon, and welcome to Relmada Therapeutics third-quarter 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 of LifeSci Advisors. Please go ahead.

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 ended September 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-Q for the quarter ended September 30, 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 November 13, 2025. 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, 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 Q3 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. Sergio, please go ahead.

Thank you, Brian, as always, and good afternoon, and welcome, everyone, to the Relmada third-quarter 2025 conference call. 2025 is shaping up to be a standout year for Relmada with excellent product development progress driven by the effort of our outstanding team and strengthened by our recent successful capital raise.

We are developing one late-stage and one mid-stage clinical program that we believe could be life-changing for patients. Each program has the potential to be the best-in-class treatment. Our lead program is NDV-01, a sustained release formulation of gemcitabine docetaxel or Gem/Doce in development for non-muscle invasive bladder cancer, or NMIBC, which affects about 68,000 new patients each year in the US and has a prevalence of approximately 744,000 patients in the US only.

Our second program is sepranolone that is intended to normalize GABAA receptor activity in compulsive disorder. Sepranolone is in development for Prader-Willi syndrome, which has a US prevalence of approximately 20,000 patients.

Here are the three key messages that we will cover today. Number one, we reported nine months follow-up data from the Phase II study of NDV-01 in patients with NMIBC. In brief, the study showed a 92% overall response rate at any time with favorable overall safety. We are very pleased by the encouraging and consistent data.

Number two, we are pleased to have secured FDA alignment on the key elements of the Phase III program for NDV-01. It is intended to enable two distinct and independent registrational tracks for NDV-01 in NMIBC. This is an important key derisking milestone for the program that opens the door to a broad market opportunity in NMIBC.

Number three, with the recently completed $100 million underwritten financing, we are well capitalized. The recent offering provides the resources to support our planned operations into 2028 and drive forward the planned registration studies for NDV-01 and the Phase II study for sepranolone in PWS.

We are preparing to initiate these studies in 2026. For NDV-01, we expect to begin two separate registrational studies for NMIBC starting in the first half of 2026. For sepranolone, we anticipate starting a Phase II study in PWS also in the first half of 2026.

We are well positioned to advance our pipeline, thanks to our expanding clinical team. Earlier this year, we appointed Dr. Raj Pruthi as Chief Medical Officer, Uro-oncology. Dr. Pruthi is a highly respected expert in bladder cancer and urologic oncology, who brings vast experience advancing novel therapies for NMIBC.

We have also established a Clinical Advisory Board to provide additional guidance for the pivotal program for NDV-01. The Board is comprised of prominent leaders in NMIBC and chaired by Dr. Yair Lotan, a renowned urologic oncologist. In October, we were pleased to welcome Dr. Max Kates to our Advisory -- Clinical Advisory Board. Dr. Kates bring a wealth of experience from chairing the landmark Phase III BRIDGE study and leading several other practice-changing studies.

I am very pleased with Relmada work this year to derisk our pipeline and advance two potentially life-changing therapies. We are looking ahead to 2026 with enthusiasm with several value inflection catalysts ahead. Next, Raj is going to provide an update on the NDV-01 development program, including the nine-month follow-up data from the Phase II and a summary of the key highlights from the recent Type B pre-IND meeting with the FDA. Raj?

Thank you, Sergio, and good afternoon, everyone. I believe this is a very exciting time for our patients based on our excellent progress with the NDV-01 development program. I want to touch on three items today: an overview of the patient care journey in non-muscle invasive bladder cancer, or NMIBC; a review of the nine-month data; and a summary of the FDA meeting highlights.

Let's start with the NMIBC and the patient journey. There are about 85,000 new cases of bladder cancer diagnosed each year in the United States and 744,000 people living with bladder cancer. About 80% of bladder cancer patients have NMIBC and recurrence rates over five years are about 60% to 80%.

Relmada is focused on high-risk NMIBC and on intermediate risk NMIBC, representing about 80% of NMIBC cases or 54,000 people per year. In brief, the patient care journey most commonly begins when a patient presents with blood in the urine or hematuria. Suspected bladder cancer cases are diagnosed using cystoscopy and cytology. Treatment begins with a surgical procedure called transurethral resection of the bladder tumor, or TURBT. This procedure allows surgeons to classify the patient's disease stage and risk category and define the treatment plan.

After surgery, patients with high-risk disease receive intravesical adjuvant therapy with standard of care immunotherapy known as Bacillus Calmette-Guérin or BCG. Patients are then monitored with regular cystoscopies and urine cytology every three months to assess for recurrence. Patients with recurrent disease are treated with repeat surgery, alternating with intravesical treatments.

NDV-01 is a novel, sustained-release, intravesical formulation of two chemotherapy agents, gemcitabine and docetaxel or Gem/Doce, as we say. It was designed to build on data from numerous studies conducted over the past decade, showing that combination use of these two agents receives -- achieves response rates and recurrence-free survival that are comparable to or better than historical standard of care BCG. And for those who are unresponsive to BCG, it can provide a second-line bladder sparing option to avoid radical cystectomy.

The sustained release formulation of NDV-01 will be provided to study sites in a ready-to-use format that does not require a specialized pharmacy or biocontainment hood to formulate the Gem/Doce combination. NDV-01 is intended to be instilled into the bladder through a regular catheter in the office in a less than five-minute intravesical installation. Upon administration, the formulation creates a soft matrix that is intended to enhance local exposure and minimize systemic toxicity.

Moving to the nine-month data. We're pleased to report that NDV-01's continued positive Phase II performance, strongly supporting its potential to transform the treatment of NMIBC. The study is a single-arm, single-center, ex-US trial in patients with high-risk NMIBC.

Patients are treated with NDV-01 in a six biweekly induction phase, followed by monthly maintenance for up to one year. Patients receive regular assessments with cystoscopy, cytology and if needed, biopsy. The study is designed to enroll up to 70 patients with localized high-risk NMIBC.

The primary endpoints are safety and complete response at 12 months. Secondary efficacy endpoints are duration of response and event-free survival. Efficacy assessments for the nine-month follow-up included analysis of data at nine months and at any time point. These are the same safety and efficacy parameters that were applied to the six-month data reviewed during our Q2 call in August and the three-month data presented at the American Urological Association meeting in April.

Looking at the data, we observed a complete response rate of 92% at any time based on 25 patients. Amongst patients with BCG unresponsive disease, we see a 91% CR any time. At the nine-month assessment, we observed a complete response rate of 85%. No patients had progression to muscle invasive disease and no patients underwent a radical cystectomy. Patients who have been reinduced had a 60% complete response rate.

The study also includes certain defined subpopulations. For example, patients with BCG unresponsive disease, we saw a 91% CR anytime and a nine-month CR rate of 88%. NDV-01 continues to demonstrate favorable safety consistent with our expectations and known efficacy and safety of Gem/Doce. There were no reported new safety signals, no patients who had treatment-related adverse events that were Grade 3 or higher and no patients discontinued treatment due to adverse events.

The most common treatment-related adverse events were transient dysuria and hematuria and asymptomatic positive urine cultures and incidental finding observed in 9% of patients with hematuria only seen at 7%. All patients with dysuria were grade 1 and resolved within 24 hours.

Our goal is to bring NDV-01 to patients as soon as possible. We intend to initiate the Phase III program for NDV-01 in the first half of 2026. The recent positive Type B FDA meeting is a key milestone that reinforces our confidence in the path forward for NDV-01.

I'd like to summarize the key outcomes from the FDA meeting, a very positive, constructive and pragmatic discussion with them. Relmada has secured FDA alignment on certain key elements of the planned Phase III pivotal program for NDV-01, incorporating two separate and distinct registrational paths. Number one, high-risk second-line BCG unresponsive NMIBC patients; and number two, intermediate risk NMIBC in the adjuvant setting.

In the setting of high-risk second-line BCG unresponsive disease, the FDA stated that a single-arm trial might be acceptable in a more refractory patient population. We're excited about this approach because it could offer a rapid route to approval. In the indication of intermediate risk in the adjuvant setting, the FDA agreed that a proposal to randomize patients post TURBT to adjuvant NDV-01 versus observation, evaluating a time-to-event endpoint is generally acceptable.

We feel that the opportunity to incorporate NDV-01 into patient care after TURBT is very attractive. It could pave the way for an additional indication and broader clinical adoption. Importantly, the FDA indicated that no further clinical -- nonclinical studies are required to support a 505(b)(2) NDA. This is very good news. We look forward to working with the FDA to complete the study design and initiate the registrational program in the first half of 2026.

Our efforts in the coming months will also focus on transferring production to contract manufacturers to complete scale-up and production of clinical batches. As I hand the call over to our CFO, Maged Shenouda, I am optimistic about the NDV-01 clinical development program based on the excellent nine-month results, positive outcomes with the FDA meeting and our ongoing Phase III preparation. 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 third-quarter 2025 financials. Sepranolone is a member of a new subgroup of neurosteroids called GAMSAs or GABAA Modulating Steroid Antagonist. We believe sepranolone's novel action on the GABAA neurotransmitter pathway gives a unique potential to normalize GABAA receptor activity and alleviate the repetitive symptoms in disorders where compulsive behaviors are a common feature.

These disorders affect millions of people in the US and around the world and include indications such as Prader-Willi syndrome and Tourette syndrome. We have selected Prader-Willi Syndrome, or PWS, as the first clinical indication that we will evaluate for sepranolone. It affects approximately 350,000 people worldwide, including approximately 20,000 people in the US. PWS is a complex genetic disorder, often defined by persistent hunger and overheating. Current treatment is focused on improving the obsessive compulsive behaviors and other medical complications that characterize the disorder.

Phase II data from a study in patients with Tourette syndrome established sepranolone's initial efficacy in a compulsivity disorder with good overall tolerability. We intend to initiate a proof-of-concept study in PWS in the first half of 2026. Our immediate efforts are dedicated to completing study preparations, including engaging with the FDA and our proposed trial design -- 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 third quarter and nine months ended September 30, 2025.

As of September 30, 2025, Relmada had cash, cash equivalents and short-term investments of approximately $13.9 million compared to $44.9 million as of December 31, 2024. Notably, this excludes net proceeds of approximately $94 million from our $100 million underwritten offering of common stock and prefunded warrants, which the company closed on November 5, 2025. Based on current plans, the company believes that its current cash balance, including net proceeds from the offering, is sufficient to support planned expenses into 2028.

Cash used in operations in the third quarter ended September 30, 2025, was $6.7 million compared to $16.7 million for the same period in 2024. During today's call, I'll review our third quarter 2025 financial results. Information regarding the nine months results are included in our press release and 10-Q filings issued this afternoon.

Research and development expense for the third quarter of 2025 totaled $4 million compared to $11.1 million for the third quarter of 2024, a decrease of $7.1 million. The lower spend was primarily driven by lower study costs with the wind down of clinical trials for REL-1017, partially offset by an increase in manufacturing and drug storage costs associated with the ramp-up of NDV-01 and sepranolone studies and an increase in R&D employees.

General and administrative expense for the third quarter of 2025 totaled $6.3 million compared to $11.9 million for the third quarter of 2024, a decrease of approximately $5.6 million. The decrease was primarily driven by a decrease in stock-based compensation expense as well as direct employee and administrative expense. The net loss for the third quarter of 2025 was $10.1 million or $0.30 per basic and diluted share compared with a net loss of $21.7 million or $0.72 per basic and diluted share for the third quarter of 2024.

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

Thank you, Maged. Before we go to the Q&A session, I would like to share that I'm very pleased with Relmada work this year to advance and derisk our portfolio of potentially life-changing therapy for patients. With our progress comes our gratitude for your support and for taking time to join today's call. 2026 is shaping up to be another very important year for the company, and we look forward to updating you on our continued progress.

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

(Operator Instructions) Uy Ear, Mizuho Securities.

Yeah, congrats on all the progress that you've made over the last nine months, yeah. It takes a lot of doing. Maybe the first question we have is maybe just help us understand a little bit about the different potential market opportunity for the two indications that you are going after?

And maybe also help us understand the sequence of it. Will you start the study at the same time? And when you think that one study will finish before the other? And if you can maybe provide some guidance on when each of the study could complete.

So maybe talk about the potential number of patients in the refractory second-line setting versus the potential number of patients in low-grade intermediate risk who would -- who could benefit from the adjuvant combination of NDV-01? First question. Thank you.

Thank you, Uy, for the question. I believe Raj that runs the clinical program can answer your question appropriately. Raj, do you want to try?

Yeah, of course. So as I mentioned, there's two proposed indications. One is in BCG unresponsive refractory to first-line therapy. And I'll talk -- let me talk a little bit about this population, then the intermediate risk, and then we'll talk about timelines.

So this is a relatively smaller population. There are about 8,000 patients per year. Now with current therapies, 55% to 80% of those patients will recur after first-line therapy. So there's a growing number of patients that are needed in the second-line indication. So from 8,000, you can take that down to 55% to 80% each year that will be BCG unresponsive that fail a primary therapy.

Now the intermediate risk population, and this is high-grade and low-grade intermediate risk, is a much larger patient population, estimated about 80,000 incident and prevalent patients each year in the United States with intermediate-risk NMIBC. A significant number of them, probably over half will receive an adjuvant therapy. And so that's about 40,000. So that represents a significant market for us to address. And I think if you look at surveys of urologists, chemotherapy and Gem/Doce chemotherapy is the preferred choice.

Now regarding your question on timing, our plan is to initiate both of these trials, although they are separate indications, both trials at about the same time in the second quarter of 2026. I think this will provide for operational efficiencies and contracting and addressing sites. And I think for the sites will be easier as they kind of know how to do a clinical trial one side or the other.

In the unresponsive patient population, the first patient in being Q2 '26, will likely have clinical data, three-month data by Q4 '26 to provide internally and externally. And then the endpoint is going to be a 12-month CR. So that will be Q2 '27 with top line data in Q2 '28.

In the intermediate risk study also initiating in Q2 '26, that's an open-label but randomized study that will take probably about 15 months to complete enrollment. With that completed enrollment, we'll need probably about 18 to 24 months of follow-up. It's -- I think it's a little bit trickier. We plan to do an interim analysis at 70% events.

Regarding the ability to provide data before then, I think that's a conversation we'll have to have with the FDA, although it's an open-label study, we certainly wouldn't want to expand alpha along the way. So I hope that gives you an idea of the size of the populations and the timelines.

Yeah, that was super helpful. So maybe just help us with the other element. So with J&J and Lexo, I think the price is $69,000 per dose or per one of those pretzel tubes. And the induction phase is -- I think it's -- you need eight of those. So that sort of rounds up to about $550,000 a year.

Does that sort of make sense in terms of -- I know it's probably too early to sort of speak about pricing. Just wanted to maybe get your view on what potential pricing could look like?

Yeah. Let me actually take a quick answer to that, and then I'd like to ask our CEO, Sergio, to comment. So yeah, I think you're right, if you actually add up the induction phase and maintenance phase in the first year for -- in Lexo, it approaches $700,000. So that certainly has now set the new benchmark above Antiva for -- if you look at one year of therapy.

The other end of the spectrum to me is Zosduri, which is in low-grade intermediate chemoablation, which the yearly cost there is about $120,000. So I think the numbers will fall somewhere between. But Sergio, do you mind if I ask you to comment on that?

Yeah. No, sure. Thanks, Raj. And look, it's a bit early to talk about pricing because we have to -- will be data-driven. Depending on what the data will look like, we will price accordingly to the value added for patients.

And -- but we do have a luxury to watch what the uptake and the penetration of J&J and the other chemotherapy UroGen with their pricing, and we'll base our decision based on also how their pricing is -- will be received by the urology community. So (inaudible) I hope I answered your question the best way I can, but we don't really have any like a specific pricing orientation for now.

Yeah. Thanks for that. I know it's probably -- it's way too early. And you're right, you need to look at the data, but it's -- I guess it's kind of encouraging that the pricing is interesting.

So maybe a last question. Maybe just help us to kind of understand a little better with respect to the differentiation and -- from the conventional Gem/Doce. I think on the call, you mentioned that you need a special biochemical hood and you need a special pharmacists, I guess someone who maybe even licensed who needs to put the product into syringes to be used.

Yeah, just help us understand like -- because of this hurdle, where the product is currently used? Is it mostly in academic center? And if all of this goes away, like what -- how does it open up the market for you if it does?

Yeah, that's a wonderful question. Yeah, and I think has been the hurdle of Gem/Doce, right? It's -- we know it works as urologists. We know it works well, like I mentioned for a decade. The obstacles for the community urologists where 70% to 80% of these patients are taken care of in the community is you need a specialized pharmacist.

And if you look at the overall procedural time of sequential gemcitabine followed by docetaxel, it's upwards of four hours. So that's a very -- that's easy to do in an academic center, and I think that's where most of the uptake has, been very difficult in the community, lack of specialized pharmacy and room or chair time and the staff for that for four hours. So I think by having prefilled syringes avoiding the specialized pharmacy and having a five-minute or so instillation to a catheter, removing the catheter, watching the patient, allowing them to go home, I think opens the door.

This is an opportunity for academic centers as well, but I think also to meet the patients where they're at and to meet the urologists where they're at as well. So I think this -- it opens up the market significantly.

Okay. Sorry, maybe just one additional question. So I know you're going after just the two indications, which is actually quite broad, particularly in the intermediate risk section. But there's an ongoing study in BCG-naive patients called, I guess, the BRIDGE study. If this study succeeds, like what does that do to the potential opportunity for this product to be used off label, even though you won't be promoting it because every doc will probably know about your product.

Yeah, Raj, do you want to give your view?

Yeah. Thank you, Sergio. So that's a very insightful question, Uy. And actually, Max Kates is Head of the BRIDGE trial. So the BRIDGE trial is a randomized study of BCG versus Gem/Doce in high-risk disease. And it's an 800-patient trial, cooperative group trial that is near end of enrollment and will read out in two years.

So the timing is nice for us. It's meant to look if Gem/Doce is noninferior to BCG. So we know that the obstacles with BCG now are supply issues, and that's been ongoing for 15 years in the US globally. And also, it does have toxicity to it. It's effective, but it does have toxicity.

So if now you introduce the ability of Gem/Doce to substitute in for BCG, Uy, I think that especially as you said, in an off-label use, an easier way to give it, I think that opens the market significantly. That's a tremendous opportunity for Relmada. Great question. Thank you.

Ladies and gentlemen, this concludes our question-and-answer session. I will now hand over to Sergio Traversa for closing remarks.

Thank you very much, and thank you, everyone. And just an extended thank you to investors, patients, and employees, collaborators, consultants that has helped us to get where we are now, and they will continue to help us to get where we want to be, that is to bring NDV-01 and sepranolone available for doctors and patients. Thank you very much, and I wish everyone a great evening for the rest of the day. Thank you.

Thank you, sir. Ladies and gentlemen, that concludes today's call. Thank you for joining us, and you may now disconnect.