UroGen Reports 77.8% Three-Month Complete Response Rate from Phase 3 UTOPIA Trial of UGN-103 and Receives FDA Agreement on NDA Submission Strategy in Recurrent LG-IR-NMIBC Based on UTOPIA Trial

On November 6, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported robust preliminary results from its ongoing Phase 3 UTOPIA trial, demonstrating a 77.8% three-month complete response (CR) rate (95% CI, 68.3%, 85.5%) with UGN-103 (mitomycin) for intravesical solution in patients with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). This result is consistent with the 79.6% three-month CR rate (95% CI, 73.9%, 84.5%) observed following treatment with ZUSDURI in the pivotal ENVISION trial. In addition, the U.S. Food and Drug Administration (FDA) agreed that CR and durability results from the UTOPIA trial can support the submission of a New Drug Application (NDA) for UGN-103 for recurrent LG-IR-NMIBC.

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"The robust 77.8% three-month CR rate observed in the UTOPIA trial is highly encouraging and reinforces the potential of UGN-103 to deliver meaningful benefits to patients," said Liz Barrett, President and Chief Executive Officer of UroGen. "In addition, the FDA’s agreement that the UTOPIA trial can support the submission of an NDA for UGN-103 represents a significant regulatory milestone and a strong validation of our clinical strategy. Together, these achievements give us significant momentum and a clear path toward potential approval, positioning UGN-103 as a key growth driver and marking an important advancement in expanding our leadership in uro-oncology. We look forward to working closely with the FDA as we complete the UTOPIA trial and prepare for an NDA submission in 2026."

UGN-103 is designed to offer key improvements over ZUSDURI (mitomycin) for intravesical solution, the first and only FDA-approved treatment for adults with recurrent LG-IR-NMIBC. These include a shorter manufacturing process and a simplified reconstitution procedure, while maintaining UroGen’s established approach that enables prolonged drug exposure within the bladder. UroGen has two U.S. patents with claims relating to the combination of the Company’s RTGel technology combined with medac’s licensed proprietary lyophilized mitomycin formulation. These patents cover UGN-103 as well as the use of UGN-103 for the treatment of LG-IR-NMIBC and expire in December 2041.

About UTOPIA

The UTOPIA trial is a single-arm, multicenter study evaluating the efficacy and safety of UGN-103 in 99 patients across global sites. Enrolled patients received 75 mg of UGN-103 via intravesical instillation once weekly for six weeks. The primary endpoint is complete response rate at three months, with responders entering a follow-up phase of up to 12 months to assess durability of response. For more information on the UTOPIA study, please visit View Source

About UGN-103
In January 2024, UroGen entered into a licensing and supply agreement with medac to develop UGN-103 for LG-IR-NMIBC. UGN-103 is an innovative mitomycin formulation that aims to streamline manufacturing and reconstitution processes while providing intellectual property coverage until December 2041. It utilizes UroGen’s RTGel technology for prolonged mitomycin exposure.

About ZUSDURI

ZUSDURI (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin approved for the treatment of adults with recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology (a sustained release, hydrogel-based formulation), ZUSDURI is delivered directly into the bladder by a trained healthcare professional using a urinary catheter in an outpatient setting, thereby enabling the treatment of tumors by non-surgical means.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include transurethral resection of bladder tumor (TURBT) as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence, and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.

(Press release, UroGen Pharma, NOV 6, 2025, View Source [SID1234659592])

UroGen Reports Third Quarter 2025 Financial Results as ZUSDURI™ Launch Gains Momentum

On November 6, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, reported financial results for the third quarter ended September 30, 2025, and provided an overview of recent developments.

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"Our launch of ZUSDURI, the first and only FDA-approved medicine for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer, continues to gain momentum," said Liz Barrett, President and Chief Executive Officer of UroGen. "Despite slower than anticipated new patient starts, we are encouraged by the patient demand reflected in our patient enrollment forms. Strong enthusiasm and engagement from urologists, growing physician awareness, and broad reimbursement coverage are expanding patient access. Early launch indicators reflect robust interest and confidence in ZUSDURI’s clinical value, reinforcing our belief in the significant commercial opportunity ahead and our ability to fully capitalize on it. The strong complete-response rate for UGN-103 and the FDA’s agreement with our NDA submission plan supports our strategy for the next-generation medicines that are expected to enhance supply, improve manufacturing and preparation efficiencies and provide opportunity for lifecycle extensions. With a strong financial position, we are committed to driving a successful launch of ZUSDURI and advancing our pipeline in ways that deliver lasting impact for patients and long-term value for shareholders."

Q3 2025 and Recent Business Highlights:

ZUSDURI (mitomycin) for intravesical solution:

Following U.S. Food and Drug Administration (FDA) approval on June 12, 2025, UroGen launched ZUSDURI (formerly UGN-102), the first and only FDA-approved medicine for adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC), marking a major milestone in bladder cancer care.
ZUSDURI was assigned a unique, permanent Healthcare Common Procedure Coding System J-code (J9282) by the Centers for Medicare & Medicaid Services. The J-code is expected to be effective January 1, 2026.
ZUSDURI is now broadly accessible to patients through Commercial, Medicare, and Medicaid insurance programs, with open access for more than 95% of covered lives and approximately 296 million eligible patients.
ZUSDURI achieved net product revenue of $1.8 million in Q3 2025; October 2025 preliminary demand revenue estimate of $4.5 million demonstrates accelerating commercial uptake and growing physician adoption.
From launch on July 1, 2025 through October 31, 2025, UroGen reports:
592 activated sites of care
54 unique ZUSDURI prescribers
16 repeat ZUSDURI prescribers
The article "Review of UGN-102: A Reverse Thermal Gel Containing Mitomycin for the Treatment of Recurrent, Low-Grade, Intermediate-Risk Non-Muscle Invasive Bladder Cancer" was published in Reviews in Urology and highlights durable efficacy and manageable safety profile of ZUSDURI in patients with recurrent LG-IR-NMIBC.

JELMYTO (mitomycin) for pyelocalyceal solution in low-grade upper tract urothelial cancer (LG-UTUC)

Generated net product revenue of $25.7 million in the quarter ended September 30, 2025, compared to $25.2 million (which included CREATES Act sales of $2.6 million) reported for same quarter in 2024. Underlying demand revenue grew by approximately 13% year-over-year.

Next-generation novel mitomycin-based formulation for urothelial cancers

Enrollment is complete in the ongoing Phase 3 UTOPIA clinical trial of investigational drug UGN-103 (mitomycin) for intravesical solution in patients with LG-IR-NMIBC. UroGen reported a three-month complete response rate (CRR) of 77.8% (95% CI, 68.3% to 85.5%), consistent with results from the ENVISION clinical trial.
The FDA agreed with the regulatory plan to submit an NDA based on the data from the single-arm Phase 3 UTOPIA trial to support potential approval of UGN-103. UroGen anticipates submitting an NDA for UGN-103 in the second half of 2026 with potential approval anticipated in 2027.
UGN-103 is a next generation product designed to offer certain improvements over ZUSDURI (mitomycin) for intravesical solution, including a shorter manufacturing process and simplified reconstitution procedure. UGN-103 combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH. For more information on the UTOPIA trial, refer to clinicaltrials.gov/NCT06331299.
The Phase 3 clinical trial to explore the safety and efficacy of UGN-104 is ongoing. UGN-104 is an investigational next-generation mitomycin product for LG-UTUC. Like UGN-103, UGN-104 combines UroGen’s RTGel technology with a novel mitomycin formulation licensed from medac GmbH.

UGN-301 (zalifrelimab), an anti-CTLA4 antibody for use in high-grade non-muscle invasive bladder cancer

UroGen has made the strategic decision to discontinue development of UGN-301 (zalifrelimab) following completion of its Phase 1 dose escalation study. While the study confirmed proof of concept for RTGel as a viable platform for local delivery of complex immunotherapies, UGN-301’s overall clinical profile did not meet UroGen’s internal benchmarks for advancement to Phase 2. The program achieved key proof of concept objectives, including sustained bladder exposure with minimal systemic absorption and was generally well tolerated, demonstrating the ability to mitigate CTLA-4–related toxicities, and encouraging efficacy signals. These findings further reinforce the versatility and potential of RTGel technology to enable localized delivery of immunotherapy candidates.
UroGen provided notice to Agenus Inc. of termination for convenience of the License Agreement between the parties. In accordance with the terms of the agreement, termination will become effective upon the later of (i) expiration of the 180-day notice period, or (ii) completion of all required wind-down activities, including the delivery of any Agenus Improvements as defined in the License Agreement.

UGN-501 (investigational next-gen oncolytic virus) for use in high-grade non-muscle invasive bladder cancer (

UGN-501 is a potent and fast-replicating investigational next generation oncolytic virus therapy being developed as a locally administered treatment for bladder cancer and other specialty cancers. Investigational New Drug (IND)-enabling studies are currently ongoing, with the goal of submitting an IND and initiating a Phase 1 trial in 2026.

Third quarter 2025 Financial Results

Revenue: Total revenues for the third quarter ended September 30, 2025 were $27.5 million. JELMYTO generated net product revenue of $25.7 million for the quarter ended September 30, 2025, compared to $25.2 million (which included $2.6 million of CREATES Act sales) in the same period of 2024, reflecting approximately 13% underlying year-over-year demand driven revenue growth. ZUSDURI achieved net product revenue of $1.8 million in its first quarter on the market, with October 2025 preliminary demand revenue estimate of $4.5 million demonstrating growing early commercial momentum.

R&D Expenses: Research and development (R&D) expenses for the third quarter of 2025 were $14.0 million, including non-cash share-based compensation expense of $0.7 million as compared to $11.4 million, including non-cash share-based compensation expense of $0.6 million, for the same period in 2024. The increase in R&D expenses of $2.6 million was primarily driven by costs associated with the Phase 3 UTOPIA trial for UGN-103, partially offset by lower clinical trial costs, manufacturing costs, and regulatory expenses in connection with ZUSDURI.

SG&A Expenses: Selling, general and administrative expenses (SG&A) for the third quarter of 2025 were $37.6 million, including non-cash share-based compensation expense of $2.3 million. This compares to $28.9 million, including non-cash share-based compensation expense of $2.9 million, for the same period in 2024. The increase in SG&A expenses of $8.7 million was primarily driven by ZUSDURI commercial launch activities as well as an increase in overall commercial operation costs.

Financing on Prepaid Forward Obligation: UroGen reported non-cash financing expense related to the prepaid forward obligation to RTW Investments of $4.6 million in the third quarter of 2025, compared to $5.9 million in the same period in 2024. The decrease was primarily driven by changes in underlying assumptions for remeasuring the effective rate.

Interest Expense on Long-Term Debt: Interest expense related to the $125 million term loan facility with funds managed by Pharmakon Advisors was $3.4 million in the third quarter of 2025, compared to $2.7 million in the same period in 2024. The increase was primarily attributable to the interest expense on the third tranche of the loan that was funded in September 2024.

Net Loss: UroGen reported a net loss of $33.3 million or ($0.69) per basic and diluted share in the third quarter of 2025 compared with a net loss of $23.7 million or ($0.51) per basic and diluted share in the same period in 2024.

Cash and Equivalents: As of September 30, 2025, cash, cash equivalents and marketable securities totaled $127.4 million.

For further details on the Company’s financials, refer to Form 10-Q, filed with the SEC.

2025 JELMYTO Revenue and Company Operating Expense Guidance: Guidance for full-year 2025 net product revenues for JELMYTO remains unchanged and is expected to be in the range of $94 to $98 million. This implies a year-over-year growth rate of approximately 8% to 12% over the $87.4 million in demand driven JELMYTO sales in 2024, which excludes the $3.0 million in CREATES Act sales reported in 2024. Continue to expect full-year 2025 operating expenses to be in the range of $215 to $225 million, including non-cash share-based compensation expense of $11 million to $14 million.

Conference Call & Webcast Information: Members of UroGen’s management team will host a live conference call and webcast today at 10:00 AM Eastern Time to review UroGen’s financial results and provide a general business update.

The live webcast can be accessed by visiting the Investors section of the Company’s website at View Source Please connect at least 15 minutes prior to the live webcast to ensure adequate time for any software download that may be needed to access the webcast.

(Press release, UroGen Pharma, NOV 6, 2025, View Source [SID1234659591])

Sana Biotechnology Reports Third Quarter 2025 Financial Results and Business Updates

On November 6, 2025 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on creating and delivering engineered cells as medicines, reported financial results and business highlights for the third quarter 2025.

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Sana announced it will prioritize future development activity for SC451, a HIP-modified stem cell-derived pancreatic islet cell therapy for type 1 diabetes, and for SG293, an in vivo CAR T with CD8-targeted fusogen delivery of a CD19-directed CAR. The company continues to see meaningful progress in its type 1 diabetes program, particularly in advancing SC451 toward an IND. In addition, Sana improved the potency and manufacturability of its next-generation in vivo CAR T product platform, and the company is incorporating these updates into SG293 and expects to file an IND for this program as early as 2027. The company has suspended development of its two allogeneic cell therapy CAR T programs – SC291 in B-cell mediated autoimmune diseases and SC262 in oncology.

"Given our recent progress and the potentially transformative impact with SC451 in type 1 diabetes, as well as with our in vivo CAR T platform across a range of diseases, now is the time to concentrate our efforts in these programs," said Steve Harr, President and CEO of Sana. "Our goal for SC451 in type 1 diabetes is a single treatment leading to normal blood glucose with no need for further insulin treatment or immunosuppression, and the past several quarters of clinical results, manufacturing progress, and regulatory developments have solidified our confidence that this is possible. Additionally, as we progress toward our goals of filing an IND and beginning our Phase 1 clinical trial next year, we believe now is the time to free up resources to invest in scaling this important therapy. Separately, we have increased the potency of in vivo CAR T platform, and based upon preclinical data, believe we have the opportunity to develop best-in-class therapies with a single treatment and no conditioning chemotherapy for a range of B-cell cancers and B-cell mediated autoimmune diseases. Based upon our momentum and given the resources required to fully exploit these opportunities, we have made the difficult decision to suspend our allogeneic CAR T programs, including SC291 and SC262. While these programs increased our confidence in our HIP platform, we believe the impact we can have for patients and shareholders is now greater with increased focus on SC451 and in vivo CAR T cells."

Recent Corporate Highlights

Published positive results from an investigator-sponsored, first-in-human study transplanting UP421, an allogeneic primary islet cell therapy engineered with hypoimmune platform (HIP) technology, into a patient with type 1 diabetes without the use of any immunosuppression.

UP421 is a primary human HIP-modified pancreatic islet cell therapy for patients with type 1 diabetes. The goal of this investigator-sponsored trial (IST) is to understand safety, immune evasion, islet cell survival, and beta cell function, as measured by C-peptide production, of HIP-modified pancreatic islet cells transplanted into type 1 diabetes patients without the use of any immunosuppression. The trial is being conducted under a clinical trial authorization at Uppsala University Hospital with Dr. Per-Ola Carlsson as the principal investigator.
Results of the study through 6 months after cell transplantation demonstrate the survival and function of pancreatic beta cells as measured by the presence of circulating C-peptide, a biomarker indicating that transplanted beta cells are producing insulin. C-peptide levels also increase with a mixed meal tolerance test at these timepoints, consistent with insulin secretion in response to a meal. 12-week PET-MRI scanning demonstrated islet cells at the transplant site. The study identified no safety issues, and the HIP-modified islet cells evaded immune detection.
The New England Journal of Medicine (NEJM) published a journal article titled "Survival of Transplanted Allogeneic Beta Cells with No Immunosuppression" (DOI: 10.1056/NEJMoa2503822). The article discusses 12-week results from this study. NEJM also published an accompanying editorial that further describes both the Sana technology and progress in the field (DOI: 10.1056/NEJMe2507578).
Sana and Uppsala University Hospital expect to report additional data from the IST, including longer-term follow-up.
Advancing our focused pipeline across two platforms:

Hypoimmune Platform – Type 1 diabetes – Sana continues pre-clinical development of SC451, an O-negative, HIP-modified, iPSC-derived pancreatic islet cell therapy, which uses the same HIP technology as UP421. Sana has had multiple interactions with regulators over the past several months, including an FDA INTERACT meeting, the results of which increase confidence in moving forward with our HIP-edited master cell bank for GMP manufacturing and our nonclinical testing plan. Sana expects to file an IND and begin our Phase 1 clinical trial for SC451 as early as 2026.
Fusogen Platform – In vivo CAR T cells – SG293 is the next-generation version of our prior SG299 product candidate and was renamed to reflect an updated construct and manufacturing process. SG293 is a CD8-targeted fusosome that delivers to CD8+ T cells the genetic material to make CD19-directed CAR T cells while avoiding potentially troublesome delivery to tissues such as the liver. SG293 builds on data from SG299, showing cell-specific delivery and deep B-cell depletion – as measured by depletion in circulating and lymph node B cells as well as a phenotypic reset when B cells return – in non-human primates without the use of any lymphodepleting chemotherapy. Sana intends to explore SG293 in both B-cell cancers and B-cell mediated autoimmune diseases. The company expects to file an IND for SG293 as early as 2027.
SC291 (HIP-modified CD19-directed allogeneic CAR T) in autoimmune diseases and SC262 (HIP-modified CD22-directed allogeneic CAR T) in oncology – Sana recently published in the journal Cell Stem Cell an article titled "Hypoimmune CD19 CAR T cells evade allorejection in patients with cancer and autoimmune disease," which provides additional clinical data showing how HIP-modified cells avoid immune attack (doi.org/10.1016/j.stem.2025.07.009). Nevertheless, as the company seeks to further prioritize resources and follow promising data in the SC451 and fusogen programs, Sana has suspended enrollment and further internal investment in the respective Phase 1 trials.
Raised aggregate gross proceeds of $133.2 million from sales of common stock through Sana’s at-the-market offering facility (ATM) and equity financing in the third and fourth quarters of 2025; expected cash runway into late 2026.

Closed public offering in August 2025 of 24.3 million shares of Sana’s common stock, including 3.4 million shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, and pre-funded warrants to purchase 1.5 million shares of Sana’s common stock. The gross proceeds from this offering were $86.3 million before deducting underwriting discounts and commissions and offering expenses.
Raised gross proceeds of $29.5 million in the third quarter of 2025 and an additional $17.4 million in the fourth quarter of 2025 from sales of common stock through Sana’s ATM.
Third Quarter 2025 Financial Results

GAAP Results

Cash Position: Cash, cash equivalents, and marketable securities as of September 30, 2025 were $153.1 million compared to $152.5 million as of December 31, 2024. The increase of $0.6 million was primarily driven by net proceeds from equity financings of $109.7 million and other cash inflows of $2.4 million, partially offset by cash used in operations of $111.2 million.
Research and Development Expenses: For the three and nine months ended September 30, 2025, research and development expenses, inclusive of non-cash expenses, were $30.1 million and $97.1 million, respectively, compared to $53.2 million and $170.5 million for the same periods in 2024. The decreases of $23.1 million and $73.5 million for the three and nine months ended September 30, 2025 compared to the same periods in 2024, respectively, were primarily due to the portfolio prioritization announced in the fourth quarter of 2024, which resulted in lower research, laboratory, and clinical development costs, lower personnel-related costs, including non-cash stock-based compensation, and a decrease in facility and other allocated costs. Research and development expenses include non-cash stock-based compensation of $3.2 million and $12.0 million, respectively, for the three and nine months ended September 30, 2025, and $6.5 million and $19.5 million for the same periods in 2024.
Research and Development Related Success Payments and Contingent Consideration: For the three and nine months ended September 30, 2025, Sana recognized non-cash expenses of $3.1 million and $15.3 million, respectively, compared to a non-cash gain of $5.5 million and a non-cash expense of $4.6 million for the same periods in 2024, in connection with the change in the estimated fair value of the success payment liabilities and contingent consideration in aggregate. The value of these potential liabilities fluctuates significantly with changes in Sana’s market capitalization and stock price.
General and Administrative Expenses: General and administrative expenses for the three and nine months ended September 30, 2025, inclusive of non-cash expenses, were $10.3 million and $32.1 million, respectively, compared to $14.1 million and $46.8 million for the same periods in 2024. The decreases of $3.8 million and $14.7 million for the three and nine months ended September 30, 2025, respectively, compared to the same periods in 2024 were primarily due to lower personnel-related costs, including non-cash stock-based compensation, due to a decrease in headcount in connection with the portfolio prioritization announced in the fourth quarter of 2024, and decreased legal and consulting fees. General and administrative expenses include non-cash stock-based compensation of $2.3 million and $7.1 million for the three and nine months ended September 30, 2025, respectively, compared to $4.3 million and $11.8 million for the same periods in 2024.
Impairment of Long-Lived Assets: For the nine months ended September 30, 2025, non-cash impairment of long-lived assets was $44.6 million, compared to zero for the same period in 2024. The non-cash impairment, recorded in the second quarter of 2025, was primarily related to Sana’s manufacturing facility in Bothell, Washington and certain laboratory and office space in Seattle, Washington. Because of the increased availability of manufacturing capacity at third-party contract development and manufacturing organizations (CDMOs) for cell and gene therapy products as well as progress in understanding its near-term manufacturing needs, Sana expects to use CDMOs to meet its manufacturing needs at present and has suspended further build-out of internal manufacturing capabilities.
Net Loss: Net loss for the three and nine months ended September 30, 2025 was $42.2 million, or $0.16 per share, and $185.3 million, or $0.75 per share, respectively, compared to $59.9 million, or $0.25 per share, and $217.7 million, or $0.95 per share, for the same periods in 2024.
Non-GAAP Measures

Non-GAAP Operating Cash Burn: Non-GAAP operating cash burn for the nine months ended September 30, 2025 was $108.0 million compared to $153.1 million for the same period in 2024. Non-GAAP operating cash burn is the decrease in cash, cash equivalents, and marketable securities, excluding cash inflows from financing activities, costs related to the portfolio prioritizations in the fourth quarters of 2024 and 2023, and the purchase of property and equipment.
Non-GAAP Net Loss: Non-GAAP net loss for the three and nine months ended September 30, 2025 was $39.0 million, or $0.15 per share, and $125.4 million, or $0.51 per share, respectively, compared to $64.7 million, or $0.27 per share, and $208.3 million, or $0.91 per share, for the same periods in 2024. Non-GAAP net loss excludes non-cash expenses and gains related to the change in the estimated fair value of contingent consideration and success payment liabilities, and non-cash impairment losses recorded in 2025.

(Press release, Sana Biotechnology, NOV 6, 2025, View Source [SID1234659590])

Replimune Reports Fiscal Second Quarter 2026 Financial Results and Provides Corporate Update

On November 6, 2025 Replimune Group, Inc. (Nasdaq: REPL), a clinical stage biotechnology company pioneering the development of novel oncolytic immunotherapies, reported financial results for the fiscal second quarter ended September 30, 2025 and provided a business update.

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The Company announced on October 20, 2025, that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) resubmission of RP1 for the treatment of advanced melanoma with a Prescription Drug User Fee Act (PDUFA) target action date set for April 10, 2026. The resubmission is considered by the FDA to be a complete response to the complete response letter received on July 21, 2025. In the Type A meeting minutes, the FDA indicated that the IGNYTE-3 trial could potentially support approval.

"After a collaborative dialogue and productive engagement with the FDA we are encouraged by the acceptance of our BLA resubmission for RP1 in combination with nivolumab," said Sushil Patel, Ph.D., CEO of Replimune. "We are currently partnering with the agency on the ongoing review to bring this important therapy to patients."

Program Highlights & Milestones

RP1 (vusolimogene oderparepvec)

The global Phase 3 trial, IGNYTE-3 assessing RP1 in combination with nivolumab is ongoing. The trial is expected to enroll approximately 400 patients globally and is evaluating RP1 in combination with nivolumab versus a control arm of physician’s choice in patients with advanced melanoma who have progressed on anti-PD-1 and anti-CTLA-4 therapies or are ineligible for anti-CTLA-4 treatment. The primary endpoint of this trial is overall survival, and key secondary endpoints are progression free survival and overall response rate.
Acral melanoma data for RP1 plus nivolumab was recently presented at the ESMO (Free ESMO Whitepaper) Congress 2025. The analysis of acral melanoma data from the IGNYTE anti-PD-1 failed melanoma cohort showed treatment with RP1 combined with nivolumab resulted in an objective response rate (ORR) of 44% (8/18) with a median duration of response of 11.9 months. The safety profile was favorable with generally transient grade 1 and 2 treatment related adverse events.
Additionally, a poster from ESMO (Free ESMO Whitepaper) featuring data from the IGNYTE clinical trial showed that RP1 plus nivolumab provided responses across multiple advanced non-melanoma skin cancer (NMSC) tumor types, including anti–PD-1 naïve and failed disease, as well as both in locally advanced and metastatic disease. The ORR was 100.0%, 33.3%, 66.7%, and 56.3% in patients with anti–PD-1 naïve MCC, BCC, angiosarcoma, and CSCC, respectively. The ORR was 26.3%, 30.0%, 37.5%, and 15.2% in patients with anti–PD-1 failed MCC, BCC, angiosarcoma, and CSCC, respectively. The IGNYTE clinical trial cohort in NMSC is ongoing.
Data from the ongoing ARTACUS Phase 2 trial evaluating the potential of RP1 as monotherapy in cutaneous squamous cell carcinoma patients following organ transplant were recently presented during an oral session at the Society for Melanoma Research 22nd International Congress. A publication for ARTACUS is planned for 2026.

RP2

The registration-directed Phase 2/3 REVEAL trial of RP2 in metastatic uveal melanoma is currently enrolling. The clinical trial is expected to enroll approximately 280 patients with metastatic uveal melanoma who are immune checkpoint inhibitor-naïve and evaluate RP2 in combination with nivolumab versus ipilimumab in combination with nivolumab. The primary endpoints of the trial are overall survival and progression free survival, and key secondary endpoints are overall response rate and disease control rate.
The Phase 2 clinical trial of RP2 combined with atezolizumab and bevacizumab in anti-PD-1/PD-L1 progressed hepatocellular carcinoma is currently enrolling. The protocol is being amended to include RP2 as monotherapy with data planned by the end of 2026. The trial is being conducted under a collaboration and supply agreement with Roche. The Company also expects to enroll its first patient in the fourth quarter of 2025 in a cohort evaluating RP2 in patients with biliary tract cancer. This cohort will evaluate RP2 combined with durvalumab.

Upcoming Events

Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2025 40th Annual Meeting being held November 5th to 9th, 2025:
Late-Breaking Oral Presentation: Biomarker and updated clinical data for RP1 plus nivolumab in anti-PD-1 failed melanoma from the IGNYTE trial demonstrate reversal of mechanisms of resistance to immune checkpoint blockade (Abstract 1327, November 7th, 4:45 pm ET)
Poster: RP1 plus nivolumab in patients with and without prior BRAF-directed therapy: A subgroup analysis of patients with anti–PD-1 failed BRAF-mutant melanoma from the IGNYTE clinical trial (Poster 611, November 7, 5:35-7:00 pm ET)
Poster: Retreatment with RP1 in combination with nivolumab in patients with advanced anti–PD-1 failed melanoma (Poster 600, November 8, 5:10-6:35 pm ET)

Financial Highlights

Cash Position: As of September 30, 2025, cash, cash equivalents and short-term investments were $323.6 million, as compared to $483.8 million as of fiscal year ended March 31, 2025. The decrease in cash balance was a result of cash burn related to operating activities in advancing the company’s clinical development plans.

Based on the current operating plan, the Company believes that existing cash, cash equivalents and short-term investments, as of September 30, 2025 will enable the Company to fund operations late into the fourth quarter of 2026 which includes the potential commercialization of RP1 in skin cancers and for working capital and general corporate purposes and excludes any potential revenue.
R&D Expenses: Research and development expenses were $57.9 million for the fiscal second quarter and $43.4 million for the fiscal second quarter ended September 30, 2024. This increase was primarily due to an increase in RP1 direct research costs related to the IGNYTE-3 confirmatory study and other study costs including lab and operating supplies, as well as increased RP2 study costs. In addition, personnel-related costs increased as we continued to prepare for a potential commercial launch of RP1. Research and development expenses included $4.4 million in stock-based compensation expenses for the fiscal second quarter ended September 30, 2025.

S,G&A Expenses: Selling, general and administrative expenses were $26.4 million for the fiscal second quarter ended September 30, 2025, as compared to $15.5 million for the fiscal second quarter ended September 30, 2024. Selling, general and administrative expenses included $4.0 million in stock-based compensation expenses for the fiscal second quarter ended September 30, 2025.
Net Loss: Net loss was $83.1 million for the fiscal second quarter ended September 30, 2025 and $53.1 million for the fiscal second quarter ended September 30, 2024.

About RP1

RP1 (vusolimogene oderparepvec) is Replimune’s lead product candidate and is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death, and the activation of a systemic anti-tumor immune response.

About RP2

RP2 is based on a proprietary strain of herpes simplex virus engineered and genetically armed with a fusogenic protein (GALV-GP R-) and GM-CSF intended to maximize tumor killing potency, the immunogenicity of tumor cell death and the activation of a systemic anti-tumor immune response. RP2 additionally expresses an anti-CTLA-4 antibody-like molecule, as well as GALV-GP R- and GM-CSF. RP2 is intended to provide targeted and potent delivery of these proteins to the sites of immune response initiation in the tumor and draining lymph nodes, with the goal of focusing systemic-immune-based efficacy on tumors and limiting off-target toxicity.

(Press release, Replimune, NOV 6, 2025, View Source [SID1234659589])

Relay Therapeutics Reports Third Quarter 2025 Financial Results and Corporate Updates

On November 6, 2025 Relay Therapeutics, Inc. (Nasdaq: RLAY), a clinical-stage, small molecule precision medicine company developing potentially life-changing therapies for patients living with cancer and genetic disease, reported third quarter 2025 financial results and corporate updates.

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"We are pleased with the clinical progress we’ve made across all three of our RLY-2608 trials in breast cancer and vascular malformations," said Sanjiv Patel, M.D., President and Chief Executive Officer of Relay Therapeutics. "In conjunction with our company’s focus on clinical execution, we are pleased to welcome to our Board, Mr. Coats and Mr. Dable, whose extensive experiences in late-stage development and commercialization will help to guide us as we advance our programs."

Board of Directors Updates

Effective November 4, 2025, Lonnel Coats and Habib Dable were appointed to Relay Therapeutics’ Board of Directors.

Lonnel Coats is the former Chief Executive Officer and director of Lexicon Pharmaceuticals, Inc., a biopharmaceutical company. Prior to his time at Lexicon, from 1996 to 2014, Mr. Coats served in a series of leadership positions at Eisai Inc. and Eisai Corporation of North America, U.S. subsidiaries of Tokyo-based Eisai Co., Ltd., a Japanese pharmaceutical company, including as Chief Executive Officer of Eisai Inc. from 2010 to 2014 and as President and Chief Operating Officer of Eisai Inc. from 2004 to 2010. As President and Chief Executive Officer of Eisai, Mr. Coats oversaw the commercialization of Eisai products in the therapeutic areas of oncology, neurology, gastro-intestinal, epilepsy and metabolic disorders. Prior to joining Eisai, Mr. Coats spent eight years with Janssen Pharmaceuticals, Inc., a division of Johnson & Johnson, where he held a variety of management and sales positions. Mr. Coats is a former member of the board of directors of Blueprint Medicines and Verve Therapeutics. Mr. Coats holds a B.S. in Public Administration from Oakland University.

Habib Dable has over 30 years of experience in the healthcare industry and is currently an advisor at RA Capital Management, L.P. Most recently, Mr. Dable was President and Chief Executive Officer of Acceleron Pharma Inc., a biopharmaceutical company targeting leading-edge therapies for patients with serious and rare diseases, until its acquisition by Merck in 2021. Prior to joining Acceleron in 2016, Mr. Dable spent 22 years at Bayer AG where he served as President of U.S. Pharmaceuticals; Executive Vice President, Global Head Specialty Medicine; Vice President, Ophthalmology; Global Launch Team Head, EYLEA; Global Head, Neurology and Ophthalmology; and Vice President, Regional Head, Hematology

and Cardiology. He also serves on the board of directors of Day One Biopharmaceuticals, PepGen Inc. and BioLink.org. Mr. Dable is also a former member of the board of directors of Blueprint Medicines, Millendo Therapeutics, Aerovate Therapeutics and Albireo Pharma. Mr. Dable received a B.B.A and M.B.A. from the University of New Brunswick.

RLY-2608 Highlights


Breast Cancer Clinical Trial Execution
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Continued Phase 3 ReDiscover-2 trial of RLY-2608 + fulvestrant in PI3Kα-mutated, CDK4/6 pre-treated, HR+/HER2- advanced breast cancer
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Continued Phase 1/2 ReDiscover trial, advancing the ongoing triplet cohorts with RLY-2608 + fulvestrant + atirmociclib, palbociclib, or ribociclib

Vascular Malformations Clinical Trial
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Continued execution of ongoing Phase 1/2 ReInspire clinical trial in vascular malformations

Third Quarter 2025 Financial Results

Cash, Cash Equivalents and Investments: As of September 30, 2025, cash, cash equivalents and investments totaled $596.4 million, as compared to $781.3 million as of December 31, 2024. The company expects its current cash, cash equivalents, and investments will be sufficient to fund its operating expenses and capital expenditure requirements into 2029.

R&D Expenses: Research and development expenses were $68.3 million for the third quarter of 2025, as compared to $76.6 million for the third quarter of 2024. The decrease of $8.4 million was primarily due to the series of strategic choices made to streamline the research organization throughout 2024 and 2025, as well as cost avoidance on continued development of lirafugratinib after execution of the license agreement with Elevar Therapeutics, Inc. in December 2024, offset by increases in costs for the ReDiscover-2 Trial and ReInspire Trial.

G&A Expenses: General and administrative expenses were $12.1 million for the third quarter of 2025, as compared to $19.8 million for the third quarter of 2024. The decrease of $7.6 million was primarily due to a decrease in stock compensation expense, as well as other employee compensation costs.

Net Loss: Net loss was $74.1 million for the third quarter of 2025, or a net loss per share of $0.43, as compared to a net loss of $88.1 million for the third quarter of 2024, or a net loss per share of $0.63.

(Press release, Relay Therapeutics, NOV 6, 2025, View Source [SID1234659588])