Core Insights - Relmada Therapeutics has made significant advancements in its lead program NDV-01, which shows promise as a best-in-class therapy for non-muscle invasive bladder cancer (NMIBC) [2][5][12] - The company successfully completed a $160 million PIPE financing, enhancing its financial position to support the development of NDV-01 [2][5] - Relmada is on track to initiate the Phase 3 RESCUE program for NDV-01 in mid-2026, following regulatory alignment with the FDA on two registrational pathways [2][3][4] Pipeline Progress - NDV-01 demonstrated a 95% complete response (CR) rate at any time and a durable 76% CR rate at 12 months in high-risk NMIBC patients, with no patients experiencing progression to muscle-invasive disease [5][6] - The FDA has confirmed alignment on two registrational pathways for NDV-01, which includes an open-label randomized controlled trial and a single-arm trial for BCG-unresponsive patients [3][4][7] Financial Performance - For the fourth quarter of 2025, the company reported a net loss of $19.9 million, or $0.27 per share, compared to a net loss of $18.6 million, or $0.62 per share, in the same period of 2024 [10][24] - The total operating expenses for the year ended December 31, 2025, were $59.1 million, a decrease from $83.9 million in 2024, primarily due to reduced research and development costs [10][24] - The cash balance as of December 31, 2025, was $93.0 million, bolstered by the recent PIPE financing, which is expected to fund operations through 2029 [5][10][26] Upcoming Milestones - The company plans to initiate the Phase 3 RESCUE program for NDV-01 in mid-2026, with initial data expected by the end of 2026 [10][12] - Sepranolone Phase 2 initiation in Prader-Willi syndrome is also expected in mid-2026 [10][12]

C O R P O R A T E O V E R V I E W Unlocking Life Changing Therapies March 2026 Disclosures The Private Securities Litigation Reform Act of 1995 provides a safe harbor for forward-looking statements made by us or on our behalf. This press release contains statements which constitute "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Any statement that is not historical in nature is a forward-looking statement an ...

Core Insights - Relmada Therapeutics announced promising 12-month interim data from its Phase 2 trial of NDV-01 for high-risk non-muscle invasive bladder cancer (NMIBC), showing a complete response (CR) rate of 76% and an 80% CR rate in the BCG-unresponsive population, indicating potential for a best-in-class profile [1][2][6] Clinical Results - The Phase 2 trial demonstrated a 12-month complete response rate of 76% in the overall population and 80% in the BCG-unresponsive subgroup [2][6] - The complete response rates at various time points were: 95% at any time, 87% at 3 months, 86% at 6 months, 85% at 9 months, and 76% at 12 months [4] - In the BCG-unresponsive subgroup, the complete response rates were 94% at any time, 82% at 3 months, 86% at 6 months, and 91% at 9 months [5] Safety Profile - NDV-01 exhibited a favorable safety profile with no patients experiencing Grade 3 or higher treatment-related adverse events and no treatment-related discontinuations [6][12] - Among the 48 patients, 63% experienced treatment-related adverse events, primarily transient dysuria [12] Future Development Plans - The interim results support advancing NDV-01 into the Phase 3 RESCUE registrational program, expected to initiate in mid-2026, with two pathways: one for adjuvant intermediate-risk NMIBC and another for second-line BCG-unresponsive NMIBC [3][8] - The Phase 3 program aims to address significant unmet needs in the NMIBC market, particularly for patients with limited treatment options [13] Product Overview - NDV-01 is a sustained-release formulation of gemcitabine and docetaxel designed for intravesical administration, allowing for gradual drug release over 10 days [9] - The formulation is convenient to administer in approximately 5 minutes without the need for anesthesia or specialized equipment, enhancing its potential for broad adoption in clinical settings [9]

Protara Therapeutics Conference Call Summary Company Overview - **Company**: Protara Therapeutics (NasdaqGM:TARA) - **Industry**: Biotechnology, focusing on rare diseases and oncology - **Key Areas**: Non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations Core Points and Arguments Oncology Focus - Protara is developing therapies for non-muscle invasive bladder cancer, with two late-stage programs targeting BCG unresponsive and BCG naive patients [3][4] - The ADVANCED-2 study is a significant registrational study for BCG-unresponsive patients, with expected enrollment completion by the end of 2026 [4] - A randomized controlled trial (RCT) for BCG-naive patients has been approved by the FDA, marking a first in the NMIBC setting [4][11] Lymphatic Malformations Program - Protara's TAR 002 program targets macrocystic lymphatic malformations, showing a 100% clinical success rate at the eight-week evaluation point [7][14] - The company received breakthrough therapy designation and fast-track designation from the FDA, indicating a significant unmet need in this area [15][16] Market Opportunities - In NMIBC, 80% of patients are treated in community settings, where safety and tolerability are critical [9] - Protara's 002 therapy is positioned as a best-in-class option due to its safety profile and ease of administration [10] - The market opportunity for lymphatic malformations is estimated at around 1,000 patients annually in the U.S., with a focus on macrocystic cases [17][20] Clinical Data and Expectations - Upcoming data release in February for the ADVANCED-2 study is anticipated to show a complete response (CR) rate similar to previous findings of 72.4% at six months and 50% at twelve months [6][27] - The company aims to demonstrate that BCG-experienced and BCG-refractory patients respond similarly to TAR 002 [8] Competitive Landscape - Protara aims to position its therapies as alternatives to BCG, particularly for patients who cannot receive BCG [25] - The company is focused on generating level one evidence through its RCT, which will enhance its standing with payers and in treatment guidelines [12][13] Future Developments - Protara expects to begin receiving FDA approvals for its programs starting in 2027, with a robust pipeline of late-stage developments [22] - The IV Choline Chloride program is also in phase three, targeting a patient population of approximately 40,000 in the U.S. [21] Additional Important Content - The company emphasizes the importance of patient advocacy in raising awareness about new treatment options for lymphatic malformations [19] - Protara's leadership reflects on the journey of developing their portfolio, highlighting the progress made since going public in 2020 [34][35]

Group 1 - The call is focused on the updated interim results from the ongoing Phase II open-label ADVANCED-2 trial of TARA-002, which targets patients with non-muscle invasive bladder cancer [2] - Justine O'Malley, Senior Vice President of Investor Relations and Corporate Affairs, is leading the update call [2] - The conference is being recorded, and participants are reminded to disconnect if they have objections [1] Group 2 - Forward-looking statements will be made during the call, representing the company's views as of the current date [3] - Actual results may differ from these forward-looking statements due to various factors, as outlined in the Risk Factors section of the company's most recent annual report [4] - The company disclaims any obligation to update these forward-looking statements except as required by law [4]

Core Insights - Relmada Therapeutics announced positive 9-month follow-up safety and efficacy data for NDV-01 in non-muscle invasive bladder cancer (NMIBC), achieving a 92% overall response rate [1][3] - The FDA provided feedback supporting two potential registrational trials for NDV-01, one for 2nd line refractory BCG-unresponsive NMIBC and another for intermediate-risk NMIBC [1][4] - The company plans to initiate Phase 3 pivotal programs for NDV-01 in the first half of 2026 [2][8] FDA Meeting Outcomes - The FDA confirmed that no additional non-clinical studies are required for the 505(b)(2) New Drug Application (NDA) [4] - A single-arm trial for high-grade, 2nd line BCG-unresponsive NMIBC patients is considered acceptable by the FDA [4] - A randomized controlled trial proposal for intermediate-risk NMIBC was also deemed generally acceptable [4] Clinical Results - The 9-month follow-up data showed a complete response rate of 92% (23/25) for all patients, with 84% (21/25) at 3 months and 87% (20/23) at 6 months [3][5] - In the BCG-unresponsive subpopulation, the complete response rates were 91% (10/11) at any time, 82% (9/11) at 3 months, and 78% (7/9) at 6 months [6][12] Safety Profile - No new safety signals were reported, with no patients experiencing Grade 3 treatment-related adverse events (AEs) [11] - Among 36 enrolled patients, 61% experienced treatment-related AEs, primarily transient dysuria (62%) [11] Product Overview - NDV-01 is a sustained-release formulation of gemcitabine and docetaxel designed for intravesical administration, allowing for gradual drug release over 10 days [8] - The product is convenient to administer in-office, requiring less than 10 minutes and no specialized equipment [8] Market Opportunity - NMIBC accounts for 75-80% of all bladder cancer cases, with a significant market opportunity due to high recurrence rates and limited treatment options [9]

Core Insights - Relmada Therapeutics reported a 91% overall response rate for NDV-01 in non-muscle invasive bladder cancer during a 6-month follow-up of a Phase 2 study, indicating strong efficacy and safety [1][3][4] - The company is preparing to initiate a Phase 3 registration trial for NDV-01 in the first half of 2026 and plans to start a Phase 2 study for sepranolone in Prader-Willi syndrome in the same timeframe [1][5] Clinical Results - The Phase 2 study of NDV-01 involved 29 patients, with a median age of 73 years. The study reported a complete response rate of 91% at any time point, 83% at 3 months, and 90% at 6 months [2][3] - No patients progressed to muscle invasive disease or underwent radical cystectomy, and no patients experienced Grade 3 treatment-related adverse events [3][4] Pipeline Developments - NDV-01 is a sustained-release formulation of gemcitabine and docetaxel, designed for intravesical administration, which enhances drug retention and minimizes systemic toxicity [10][12] - Sepranolone is a first-in-class GABAA Modulating Steroid Antagonist targeting disorders linked to excess GABAergic activity, with plans for further clinical development [5][13] Financial Performance - For the second quarter ended June 30, 2025, Relmada reported a net loss of $9.9 million, or $0.30 per share, compared to a net loss of $17.8 million, or $0.59 per share, in the same period of the previous year [5][24] - Research and development expenses were $2.8 million for the quarter, down from $10.7 million in Q2 2024, primarily due to reduced trial costs [5][24] Market Opportunity - The U.S. market for high-grade/intermediate-grade non-muscle invasive bladder cancer is significant, with approximately 600,000 prevalent cases, highlighting the potential for NDV-01 as a frontline or salvage therapy [7][12] - Prader-Willi syndrome, targeted by sepranolone, has an estimated U.S. prevalence of 20,000 patients, representing an orphan disease market [7][14]