Genelux Corporation Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Business Updates

On March 19, 2026 Genelux Corporation (NASDAQ: GNLX), a late clinical-stage immuno-oncology company, reported fourth quarter and full year 2025 financial results and business updates.

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"Looking ahead in 2026, Genelux is entering a pivotal period of key clinical readouts for Olvi-Vec and defining milestones for the Company. With the Phase 3 OnPrime/GOG-3076 registrational trial expected to yield topline data in the second half of the year, we are focused on continued disciplined execution. Our productive interactions with the FDA, most recently in January 2026, have informed our next steps and strengthen our conviction in the development of Olvi-Vec for cancer patients with limited alternatives," said Thomas Zindrick, President, CEO and Chairman of Genelux. "Encouraging interim data reported in January 2026 from our systemic lung cancer programs provided additional insight into the potential of Olvi-Vec. In platinum-relapsed or refractory advanced SCLC, systemically delivered Olvi‑Vec demonstrated partial responses, and in advanced or metastatic recurrent NSCLC, early signals of anti-tumor activity were observed. Collectively, these findings further support our strategy of developing Olvi-Vec as a potential platinum resensitizing agent across multiple platinum-treated solid tumors."

"In parallel, we are advancing our manufacturing and operational capabilities to support the long-term development of Olvi-Vec and lay the groundwork for potential commercial readiness following successful registration, if obtained. These efforts are intended to ensure appropriate supply, quality systems, and organizational infrastructure as we advance our registration-directed programs," concluded Mr. Zindrick.

Clinical Program Highlights

Olvi-Vec in Platinum-Resistant/Refractory Ovarian Cancer:
Genelux continues to advance Olvi-Vec toward potential registration in platinum-resistant/refractory ovarian cancer (PRROC). Intraperitoneal administration of Olvi-Vec enables high and condensed dosing in PRROC, where patients need options that can deliver anti-tumor activity and effectively reverse platinum resistance:

The ongoing Phase 3 OnPrime/GOG-3076 registrational trial (NCT05281471) of Olvi-Vec in platinum-resistant/refractory ovarian cancer is being conducted at sites across the United States, with topline data anticipated in the second half of 2026.
The OnPrime/GOG-3076 study is a multi-center, randomized open-label phase 3 study evaluating the safety and efficacy of Olvi-Vec in combination platinum-doublet chemotherapy and bevacizumab compared to the active comparator arm with physician’s choice of chemotherapy and bevacizumab in women diagnosed with platinum-resistant/refractory ovarian cancer (includes fallopian tube cancer and primary peritoneal cancer).
As of its most recent assessment in February 2026, the Independent Data Monitoring Committee recommended study continuation without modification.
Olvi-Vec in Lung Cancer:
Genelux is advancing two ongoing trials of systemically delivered Olvi-Vec in lung cancer. These trials are designed to demonstrate that Olvi-Vec’s oncolytic immunotherapy mechanism can extend beyond intraperitoneal delivery into a systemic setting across multiple solid tumor types and showcase Olvi-Vec’s potential to resensitize tumors to platinum-based chemotherapy:

The Phase 1b/2 study (OLVI-VEC-SCLC-202) in SCLC (NCT07136285) is evaluating Olvi-Vec in combination with platinum and etoposide chemotherapy in SCLC patients with platinum-resistant or relapsed disease after failing previous treatment with platinum and etoposide chemotherapy. The trial is being conducted by the Company’s licensing partner, Newsoara HYK Biopharmaceuticals Co., Ltd., in China. Data from the dose escalation cohorts are expected to support determination of a systemic dose for Phase 2. The following preliminary findings were reported in January 2026:
Partial responses in 3 of 9 SCLC patients (33%), including two responses in the highest dose cohort with ~55% and ~85% tumor shrinkage from baseline.
The disease control rate was 67% (6/9 patients), with tumor shrinkage ranging from 24–85% from baseline among patients achieving disease control.
Durability signals were observed, including one patient with ongoing progression‑free survival (PFS) of 12.1 months and another patient with PFS of 7.7 months, the latter exceeding their prior line of therapy by 5.8 months (PFS of 7.7 months vs. 1.9 months).
The Phase 2 VIRO-25 study (NCT06463665) is assessing Olvi-Vec in combination with platinum-based chemotherapy and an immune checkpoint inhibitor (ICI) in patients with advanced or metastatic recurrent NSCLC who failed standard frontline treatment of platinum chemotherapy and an ICI. The trial is being conducted in the United States.
In preliminary findings reported in January 2026, Olvi‑Vec demonstrated a 60% disease control rate (3/5 evaluable patients), with tumor size changes of 8.9%, -18.9%, and -22.7% respectively, as compared to baseline.
Olvi‑Vec was generally well tolerated across the SCLC and NSCLC studies as of their data review cutoff dates of December 23, 2025 and December 31, 2025, respectively.
Additional dose‑finding updates from both the SCLC Phase 1b/2 and NSCLC Phase 2 VIRO‑25 trials are expected throughout 2026, aligning with the Company’s strategy to optimize a systemic dosing regimen to inform future registrational development.

Business Updates

Chief Medical Officer

Jason Litten, M.D., joined the Company as Chief Medical Officer in January 2026. Dr. Litten brings more than 20 years of experience across academia, large pharmaceutical organizations, and innovative biotechnology companies. He has led the design, execution, and interpretation of Phase 1-4 clinical trials in both liquid and solid tumors, with expertise across biologics, small molecules, and cellular therapies, and will oversee Genelux’s clinical development strategy.

Underwritten Public Offering of Common Stock

In January 2026, the Company closed an underwritten follow-on public offering of 6,666,667 shares of common stock at a price of $3.00 per share, generating net proceeds of $18.5 million, after deducting underwriting discounts, commissions and offering expenses. Net proceeds are expected to be used for general corporate purposes, which may include research and development expenses, clinical trial expenses, capital expenditures, and working capital.

Fourth Quarter and 2025 Financial Results

Cash, cash equivalents, marketable securities and restricted cash were $14.6 million as of December 31, 2025. Subsequently, on January 8, 2026, the Company raised an additional $18.5 million in net proceeds through an underwritten offering of common stock, resulting in a pro forma balance of $33.1 million as of December 31, 2025. Based on its current operating plan, the Company expects the combined cash, cash equivalents, marketable securities and restricted cash will fund operations into the first quarter of 2027.

Research and development (R&D) expenses were $19.9 million and $19.0 million for the years ended December 31, 2025 and 2024, respectively, an increase of $0.9 million. The increase was primarily driven by increased clinical trial costs associated with the Company’s Phase 3 On Prime/GOG-3076 registrational trial in 2025.

General and administrative (G&A) expenses were $13.4 million and $12.7 million for the years ended December 31, 2025 and 2024, respectively, an increase of $0.7 million. The increase was primarily driven by an increase in employee compensation due to the combination of annual salary increases and changes to headcount required to support the Company’s operations, as partially offset by a decrease in consulting services.

Net loss was $32.1 million or $0.86 per share for the year ending December 31, 2025, as compared to $29.9 million or $0.95 for the year ended December 31, 2024.

(Press release, Genelux, MAR 19, 2026, View Source [SID1234663771])

Cellectis Reports Full Year 2025 Financial Results and Provides a Business Update

On March 19, 2026 Cellectis (the "Company") (Euronext Growth: ALCLS – NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene editing platform to develop life-saving cell and gene therapies, reported financial results for the fourth quarter and full year 2025, ending December 31, 2025 and provided a business update.

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"Lasme-cel demonstrated a potentially transformative efficacy profile in one of oncology’s most challenging settings, achieving 100% overall response rate in the target Phase 2 population. Critically, lasme-cel converted all patients in the target population into transplant-eligible candidates. The pivotal Phase 2 is now enrolling, and with a BLA submission anticipated in 2028, lasme-cel is on a clear regulatory path to potentially becoming the first off-the-shelf CAR-T therapy to address this high unmet medical need" said André Choulika, Ph.D., Co-Founder and Chief Executive Officer of Cellectis. "With interim Phase 2 data for lasme-cel in r/r B-ALL, and full Phase 1 data for eti-cel in r/r NHL, both expected in Q4 2026, we are entering an important year for Cellectis, as we advance our ambition to bring life-saving off-the-shelf CAR-T therapies to patients who have run out of options".

________________________________
1 Cash, cash equivalents and fixed-term deposits include restricted cash of $4.4 million as of December 31, 2025 classified as current and non-current financial assets and fixed-term deposits of $144.8 million as of December 31, 2025, classified as current financial assets.

Allogeneic CAR-T Pipeline

Lasme-cel in relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) – BALLI-01

In October 2025, Cellectis presented full Phase 1 lasme-cel clinical data at the Cellectis’ R&D Day. The presented data position lasme-cel as a potentially game-changing therapy for patients with r/r B-ALL. Data highlighted:

Strong efficacy:

68% overall response rate (ORR) with lasme-cel Process 2, manufactured internally (n=22)
83% ORR at the recommended Phase 2 dose (RP2D) (n=12)
100% ORR in the target Phase 2 population (n=9)

In the target Phase 2 population, the complete response or complete remission with incomplete hematology recovery (CR/Cri) rate was 56%, with approximately 80% of these patients achieving minimum residual disease (MRD)-negative status

Favorable safety profile:

Low rates of ≥ grade 3 cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) at 2.5% and 5% respectively

Transplant eligibility in target Phase 2 population:

All patients became eligible for transplant

Strong survival benefit:

14.8 months median overall survival (OS) in patients who achieved MRD-negative CR/CRi

The first interim analysis for the pivotal Phase 2 of the BALLI-01 trial is expected in Q4 2026 (n=40). Cellectis anticipates submitting a Biologics License Application (BLA) in 2028.

Eti-cel in relapsed or refractory non-Hodgkin lymphoma (r/r NHL) – NATHALI-01

In December 2025, Cellectis presented encouraging Phase 1 preliminary data of eti-cel at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting. The data showcased the potential of eti-cel in r/r NHL patients who have relapsed following multiple lines of therapy including, for 93% of patients, an autologous CD19 CAR-T, with an 88% ORR and 63% CR at the current dose level (n=8).
Cellectis is initiating patient enrollment in the cohort with low dose interleukin-2 (IL-2) support to evaluate the potential to further enhance the already high response rates and durability of response in patients with r/r NHL.
Cellectis expects to present the full Phase 1 dataset in 2026, including results from the IL-2 combination.

Circular single-stranded DNA (cssDNA) as a non-viral template for gene therapy

In November 2025, Cellectis published a Nature Communications article establishing cssDNA as a highly efficient non-viral DNA donor template, for gene insertion in hematopoietic stem and progenitor cells (HSPCs).

While viral vectors such as AAV6 are commonly used for gene insertion, they raise safety and efficacy concerns. Over the past decade, non-viral DNA templates delivery has emerged as promising alternatives.

Cellectis’ research results mark a pivotal advance toward next-generation non-viral cell and gene therapies.

Key findings:

Superior efficiency: cssDNA achieved over 40% knock-in efficiency, outperforming linear DNA by 3-5 times.
Versatility: the process successfully targets multiple loci in HSPCs and primary T cells.
Better persistence: in murine models, cssDNA-edited cells showed superior engraftment and edit maintenance compared to AAV6-edited cells.

TALE base editors (TALEB) safety and precision

At ESGCT 2025, Cellectis presented a comprehensive safety study on TALE base editors (TALEB), which enable precise C-to-T DNA editing without causing double-strand breaks.

Key study highlights:

Safety assessment: researchers used advanced bioinformatics and experimental models to track potential off-target effects in the nuclear genome of primary T cells.
No bias detected: the study found no evidence of unintended editing at CTCF binding sites, which are critical for genome organization and gene expression.

These research results provide a strong framework for the safe development of TALEB in therapeutic cell engineering, supporting their potential for future nuclear and mitochondrial applications.

Partnerships

AstraZeneca – Joint Research and Collaboration Agreement

Activities are progressing under the Joint Research and Collaboration Agreement with AstraZeneca, which leverages Cellectis’ gene editing expertise and manufacturing capabilities to develop up to 10 novel cell and gene therapy products for areas of high unmet medical need, including oncology, immunology and rare genetic disorders.

Servier (through its sublicensee Allogene) – Anti-CD19 CAR-T

Under the Servier Agreement, Cellectis is eligible to up to $340 million in development and sales milestones as well as low double-digit royalties on sales.

In December 2025, an arbitral tribunal has issued its decision in the arbitration proceedings against Les Laboratoires Servier and Institut de Recherches Internationales Servier IRIS SARL ("Servier"), relating to the License, Development and Commercialization Agreement entered into between Servier and Cellectis on March 6, 2019, as amended (the "Servier Agreement"). The Tribunal ruled on a partial termination of the License Agreement with respect to product UCART19 V1 (also referred to as "ALLO-501" by Allogene) and provided that Cellectis shall, at Allogene’s request, engage in good-faith discussions regarding the granting of a direct license to product UCART19 V1. All other claims brought by the parties were dismissed.

Allogene – Anti-CD70 CAR-T

According to Allogene, the TRAVERSE trial in renal cell carcinoma has completed enrollment in its Phase 1b cohort, and Allogene is currently exploring partnering opportunities to advance the asset.

Iovance

According to Iovance, new data across several pipeline programs is anticipated throughout 2026, including a Phase 1/2 trial investigating IOV-4001, a PD-1 inactivated TIL therapy, in previously treated advanced melanoma and NSCLC.

Corporate

Annual Shareholders Meeting

On June 26, 2025, Cellectis held a Shareholders General Meeting. At the meeting, during which approximately 57% of voting rights were exercised, resolutions 1 through 23 and resolutions 25 and 26 were adopted, while resolution 24 was rejected, consistent with the recommendations of the Board of Directors. The detailed results of the vote and the resolutions are available on Cellectis’ website: View Source

Board composition

The Cellectis Shareholders’ Meeting appointed Mr. André Muller as a director of the Company’s Board of Directors. At the close of this meeting, the term of Mr. Axel-Sven Malkomes expired, and the previously announced resignation of Mr. Pierre Bastid became effective. In connection with these changes to the Board of Directors, the Board of Directors appointed Mr. André Muller, Dr. Donald Bergstrom, and Dr. Rainer Boehm as the members of the Company’s Audit Committee.

2025 Financial Results

Cash: As of December 31, 2025, Cellectis had $211 million in consolidated cash, cash equivalents, restricted cash and fixed-term deposits classified as current-financial assets. The Company believes its cash, cash equivalents, and fixed-term deposits will be sufficient to fund its operations into H2 2027.

This compares to $264 million in consolidated cash, cash equivalents, restricted cash and fixed-term deposits classified as current-financial assets as of December 31, 2024. This $53 million change includes $36.9 million of cash-in from our revenue, $8.4 million of interest received from our financial and cash-equivalent investments, $2.2 million cash-in from R&D tax credit, $3.2 million cash-in from VAT credit payments, and a $4.8 million foreign currency translation impact offset by cash payments from Cellectis to suppliers of $50.5 million, Cellectis’ wages, bonuses and social expenses paid of $40.0 million, the payments of lease debts of $10.8 million, the repayment of the Prêt Garanti par l’Etat (PGE) loan for $5.4 million and the payments of capital expenditures for $3.5 million.

We currently foresee focusing our cash spending at Cellectis in supporting the development of our pipeline of product candidates, including the manufacturing and clinical development expenses of lasme-cel, eti-cel and potential new product candidates, and operating our state-of-the-art manufacturing capabilities in Paris (France) and Raleigh (North Carolina).

Revenues and Other Income: Consolidated revenues and other income were $79.6 million for the year ended December 31, 2025 compared to $49.2 million for the year ended December 31, 2024. This $30.4 million increase between the years ended December 31, 2024 and 2025 is mainly driven by the evolution of activities performed in connection with the research plans and fulfillment of our performance obligations under the Joint Research and Collaboration Agreement signed with AstraZeneca. Revenues as recorded in the year ended December 31, 2024 included a $5.4 million development milestone under the License Agreement signed with Servier.

R&D Expenses: Consolidated R&D expenses were $93.5 million for the year ended December 31, 2025, compared to $90.5 million for the year ended December 31, 2024. This $3.0 million increase is mainly due to (i) a $4.2 million increase in personnel expenses driven by an evolution of our R&D headcount consistent with our roadmap, higher fair market value of stock-based compensation instruments due to underlying stock dynamics, and foreign exchange effects; (ii) a $0.3 million increase in depreciation and amortization; compensated by (iii) a $1.5 million decrease in purchases and external expenses.

SG&A Expenses: Consolidated SG&A expenses were $19.8 million for the year ended December 31, 2025 compared to $19.1 million for the year ended December 31, 2024. The $0.7 million change is mainly due to a $0.6 million increase in purchases and external expenses.

Other operating income and expenses: Other operating income and expenses decreased slightly by $0.2 million between the years ended December 31, 2024 and 2025, from $0.8 million in 2024 to $0.6 million in 2025.

Net financial gain (loss): Net financial loss was $34.9 million for the year ended December 31, 2025, compared to a $22.8 million net financial gain for the year ended December 31, 2024. This $57.7 million difference reflects mainly a $28.3 million decrease in financial income and a $29.5 million increase in financial expenses between the years ended December 31, 2024 and 2025.

The decrease in financial income is mainly attributable to (i) a $14.3 million gain in change in fair value of the derivative instrument component of the SIA, which was recorded last year before derecognition of the derivative in May 2024; (ii) a $7.2 million decrease in foreign exchange gains; (iii) a $1.8 million decrease in income from cash, cash equivalents and financial assets in line with the evolution of interest rates in 2025, (iv) a $ 5.7 million gain recognized in the year ended December 31, 2024 on the fair value measurement of the Tranches A, B and C warrants issued to the European Investment Bank ("EIB"), partially offset by (v) a $0.8 million increase in FX derivatives fair value gains.

The increase in financial expenses is mainly attributable to a (i) $22.2 million increase in foreign exchange loss over the period due to the devaluation of the USD against the Euro, (ii) a $14.7 million loss on the fair value measurement of the Tranches A, B and C warrants issued to the EIB, (iii) a $0.7 million increase in interest on our financial and lease liabilities, partially offset by (iv) a $7.8 million decrease in the loss on fair value measurement of our investment in shares of Cibus which was entirely sold in Q1 2025.

Net Income (loss) Attributable to Shareholders of Cellectis: Consolidated net loss attributable to shareholders of Cellectis was $67.6 million (or a $0.67 loss per share) for the year ended December 31, 2025, compared to a $36.8 million loss (or a $0.41 loss per share) for the year ended December 31, 2024. The $30.8 million change in net loss was primarily driven by (i) a $30.4 million increase in revenues and other income, offset by (ii) a $3.9 million increase in operating expenses and other operating income and (iii) a $57.7 million change from a net financial gain of $22.8 million as of December 31, 2024 to a net financial loss of $34.9 million as of December 31, 2025.

Adjusted Net Income (Loss) Attributable to Shareholders of Cellectis: Consolidated adjusted net loss attributable to shareholders of Cellectis was $61.5 million (or a $0.61 loss per share) for the year ended December 31, 2025, compared to a net loss of $33.6 million (or a $0.37 loss per share) for the year ended December 31, 2024.

The year-end consolidated financial statements of Cellectis have been prepared in accordance with International Financial Reporting Standards, as issued by the International Accounting Standards Board ("IFRS").

Please see "Note Regarding Use of Non-IFRS Financial Measures" for reconciliation of GAAP net income (loss) attributable to shareholders of Cellectis to adjusted net income (loss) attributable to shareholders of Cellectis.

(Press release, Cellectis, MAR 19, 2026, View Source [SID1234663770])

TRIANA Biomedicines Announces First Patient Dosed in a Phase 1/2 Trial Evaluating TRI-611 for the Treatment of ALK Positive Non-small Cell Lung Cancer

On March 19, 2026 TRIANA Biomedicines, Inc. (TRIANA), a leading biopharmaceutical company focused on advancing a target-first and proximity-first molecular glue discovery platform to address difficult to drug disease targets, reported that the first patient has been dosed in a Phase 1/2 clinical trial evaluating TRI-611, a novel ALK-fusion molecular glue degrader, in patients with anaplastic lymphoma kinase–positive (ALK+) non-small cell lung cancer (NSCLC).

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NSCLC is the most common type of lung cancer, accounting for about 85 percent of all lung cancer cases. ALK+ NSCLC represents an important subset of lung cancer, characterized by a dependence on ALK fusion proteins for its growth and survival. Despite therapeutic advances with ALK tyrosine kinase inhibitors (TKI), resistance to TKI-based therapies and limited combination therapy options remain major clinical challenges for these younger, non-smoking patients, underscoring the profound and long-lasting impact of this disease. Addressing this persistent unmet need represents a significant opportunity to elevate the standard of care and meaningfully improve outcomes for people living with ALK+ NSCLC.

The Phase 1/2 clinical trial is a global, first-in-human, open-label study designed to evaluate the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of TRI-611 in patients with ALK+ NSCLC. The Phase 1 portion will consist of a dose escalation design, enrolling ALK+ NSCLC patients, who have been previously treated with standard of care ALK TKI therapies. The Phase 2 portion will further evaluate and characterize the efficacy and safety of TRI-611 across different patient cohorts.

"We are excited to have TRI-611 in the clinic and to advance this new potential therapy for people living with lung cancer," said Dr. Caroline Germa, Chief Medical Officer of TRIANA. "Despite progress in treatments, many patients continue to face limited options. This study represents an important step in our mission to develop innovative therapies that may ultimately improve outcomes for ALK+ NSCLC patients and their families."

"Dosing of the first patient with TRI-611 marks an important milestone for TRIANA and for the ALK+ NSCLC patient community," said Dr. Patrick Trojer, President and CEO of TRIANA. "This study reflects our commitment to treating diseases in entirely new ways by applying our molecular glue technology to highly relevant cancer targets. We are excited to advance TRI-611 and bring its potential impact one step closer to patients."

About TRI-611

TRI-611 is a novel oral, small molecule, investigational therapy designed to target and degrade ALK fusion proteins in patients with ALK+ NSCLC. TRI-611 is a highly selective, potent, brain-penetrant, molecular glue degrader that brings ALK and the E3 ligase cereblon together through a unique binding mechanism that works independently of the kinase active site. TRI-611 harnesses the body’s innate protein-degradation machinery to selectively eliminate the ALK fusion protein and is designed to overcome the limitations observed with currently available ALK inhibitors.

(Press release, Triana Biomedicines, MAR 19, 2026, View Source [SID1234663769])

TALZENNA Plus XTANDI Significantly Improves Radiographic Progression-Free Survival in Metastatic Prostate Cancer

On March 19, 2026 Pfizer Inc. (NYSE: PFE) reported positive topline results from the Phase 3 TALAPRO-3 study of TALZENNA (talazoparib), an oral poly ADP-ribose polymerase (PARP) inhibitor, in combination with XTANDI (enzalutamide), an androgen receptor pathway inhibitor (ARPI), in people with homologous recombination repair (HRR) gene-mutated metastatic castration-sensitive prostate cancer (mCSPC), also known as metastatic hormone-sensitive prostate cancer (mHSPC).

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The study met its primary endpoint, with TALZENNA plus XTANDI demonstrating a statistically significant and clinically meaningful improvement in radiographic progression-free survival (rPFS), compared to placebo plus XTANDI. The results markedly exceeded the pre-specified target hazard ratio of 0.63, with the majority of patients remaining progression-free at the time of analysis. Consistent efficacy benefit was also observed in patients whose tumors harbored BRCA and non-BRCA HRR gene alterations.

"Current treatment approaches leave many patients with HRR gene-mutated metastatic castration-sensitive prostate cancer vulnerable to early disease progression," said Neeraj Agarwal, M.D., FASCO, Professor and Presidential Endowed Chair of Cancer Research at Huntsman Cancer Institute at the University of Utah, and global lead investigator for TALAPRO-3. "The TALAPRO-3 results demonstrate that treatment with TALZENNA in combination with XTANDI earlier in the disease course significantly extends the time patients can live without their cancer worsening."

At the time of the interim analysis, results showed a strong trend toward improved overall survival (OS), a key secondary endpoint. Benefits were also observed in other secondary endpoints, including overall response rate, duration of response, and time to Prostate-Specific Antigen (PSA) progression. The safety of TALZENNA plus XTANDI was consistent with the known safety profile of each medicine, and no new safety signals were identified.

Prostate cancer is the second most common cancer in men worldwide, with an estimated 1.4 million new cases diagnosed globally in 20221 and 330,000 new cases anticipated in the United States in 2026.2 mCSPC, is a form of advanced prostate cancer that has spread beyond the prostate but is still sensitive to androgen inhibition.3 Despite recent treatment advances, 50% to 65% of patients with mCSPC progress to metastatic castration-resistant prostate cancer (mCRPC) within two years, with increased risk in HRR gene-mutated patients.4-6

"Alterations in DNA damage repair genes, such as HRR genes, are found in approximately 25% of metastatic prostate cancers and associated with a worse prognosis and are less responsive to current standards of care, representing a group with a high unmet need," said Jeff Legos, Chief Oncology Officer, Pfizer. "TALZENNA plus XTANDI is already a standard of care in HRR gene-mutated metastatic castration-resistant prostate cancer, and these unprecedented results demonstrate the potential to deliver benefit earlier in the disease course. These findings underscore Pfizer’s leadership in precision medicine and commitment to bringing more personalized treatment options to people living with prostate cancer."

TALZENNA plus XTANDI in HRR gene-mutated mCSPC is an investigational treatment regimen. The TALAPRO-3 results will be submitted for presentation at an upcoming medical congress and will be discussed with global health authorities for potential regulatory submissions.

TALZENNA plus XTANDI is currently approved in 60 countries, including in the United States for adults with HRR gene-mutated mCRPC and in the European Union for adults with mCRPC in whom chemotherapy is not clinically indicated.

About TALAPRO-3

The Phase 3 TALAPRO-3 trial is a multicenter, randomized, double-blind, placebo-controlled study that enrolled 599 patients with mCSPC [with ≤3 months of ADT (chemical or surgical) with or without an approved ARPI in the mCSPC setting] at sites in the U.S., Canada, Europe, South America, and the Asia-Pacific region. Patients with histologically/cytologically confirmed adenocarcinoma of the prostate without neuroendocrine differentiation, small cell, or signet cell features and with alterations in one or more HRR genes (as per HRR12 gene panel) in the trial were randomized to receive TALZENNA 0.5 mg/day plus XTANDI 160mg/day, or placebo plus XTANDI 160mg/day.

The primary endpoint of the trial is investigator-assessed radiographic progression-free survival (rPFS), defined as the time from the date of randomization to radiographic progression in soft tissue per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1), or in bone per Prostate Cancer Working Group 3 (PCWG3) criteria by investigator assessment, or death, whichever occurs first. Secondary endpoints include overall survival (OS), objective response rate (ORR), duration of response (DOR), prostate-specific antigen (PSA) response, and patient-reported outcomes.

For more information on the TALAPRO-3 trial (NCT04821622), go to www.clinicaltrials.gov.

About TALZENNA (talazoparib)

TALZENNA is an oral inhibitor of poly ADP-ribose polymerase (PARP), which plays a role in DNA damage repair. Preclinical studies have demonstrated that TALZENNA blocks PARP enzyme activity and traps PARP at the site of DNA damage, leading to decreased cancer cell growth and cancer cell death.

TALZENNA was initially approved in the U.S., EU, and multiple other regions as a single agent for the treatment of adult patients with deleterious or suspected deleterious gBRCAm HER2-negative locally advanced or metastatic breast cancer.

TALZENNA in combination with XTANDI was approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with HRR gene-mutated mCRPC in June 2023. The combination was also approved by the European Commission in January 2024 for the treatment of adult patients with mCRPC in whom chemotherapy is not clinically indicated. TALZENNA in combination with XTANDI is approved in 60 countries, indications vary by country.

TALZENNA (talazoparib) Indication in the U.S.

TALZENNA is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated for:

HRR gene-mutated mCRPC:

In combination with enzalutamide for the treatment of adult patients with homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC).
Breast Cancer:

As a single agent, for the treatment of adult patients with deleterious or suspected deleterious germline BRCA-mutated (gBRCAm) HER2-negative locally advanced or metastatic breast cancer. Select patients for therapy based on an FDA-approved companion diagnostic for TALZENNA.
TALZENNA (talazoparib) Important Safety Information

WARNINGS and PRECAUTIONS

Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML), including cases with a fatal outcome, has been reported in patients who received TALZENNA. Overall, MDS/AML has been reported in 0.4% (3 out of 788) of solid tumor patients treated with TALZENNA as a single agent in clinical studies. In TALAPRO-2, MDS/AML occurred in 2 out of 511 (0.4%) patients treated with TALZENNA and enzalutamide and in 0 out of 517 (0%) patients treated with placebo and enzalutamide. The durations of TALZENNA treatment in these 5 patients prior to developing MDS/AML were 0.3, 1, 2, 3, and 5 years. Most of these patients had received previous chemotherapy with platinum agents and/or other DNA damaging agents including radiotherapy.

Do not start TALZENNA until patients have adequately recovered from hematological toxicity caused by previous chemotherapy. Monitor blood counts monthly during treatment with TALZENNA. For prolonged hematological toxicities, interrupt TALZENNA and monitor blood counts weekly until recovery. If counts do not recover within 4 weeks, refer the patient to a hematologist for further investigations including bone marrow analysis and blood sample for cytogenetics. If MDS/AML is confirmed, discontinue TALZENNA.

Myelosuppression consisting of anemia, neutropenia, and/or thrombocytopenia, have been reported in patients treated with TALZENNA. In TALAPRO-2, Grade ≥3 anemia, neutropenia, and thrombocytopenia were reported, respectively, in 48%, 19%, and 9% of patients receiving TALZENNA and enzalutamide. Forty-two percent of patients (216/511) required a red blood cell transfusion, including 25% (127/511) who required more than one transfusion. Discontinuation due to anemia, neutropenia, and thrombocytopenia occurred, respectively, in 8%, 3%, and 0.4% of patients.

Withhold TALZENNA until patients have adequately recovered from hematological toxicity caused by previous therapy. Monitor blood counts monthly during treatment with TALZENNA. If hematological toxicities do not resolve within 28 days, discontinue TALZENNA and refer the patient to a hematologist for further investigations including bone marrow analysis and blood sample for cytogenetics.

Embryo-Fetal Toxicity TALZENNA can cause fetal harm when administered to pregnant women. Advise male patients with female partners of reproductive potential or who are pregnant to use effective contraception during treatment and for 4 months following the last dose of TALZENNA.

ADVERSE REACTIONS

Serious adverse reactions reported in >2% of patients included anemia (9%) and fracture (3%). Fatal adverse reactions occurred in 1.5% of patients, including pneumonia, COVID infection, and sepsis (1 patient each).

The most common adverse reactions (≥ 10%, all Grades), including laboratory abnormalities, for patients in the TALAPRO-2 study who received TALZENNA with enzalutamide vs patients receiving placebo with enzalutamide were hemoglobin decreased (79% vs 34%), neutrophils decreased (60% vs 18%), lymphocytes decreased (58% vs 36%), fatigue (49% vs 40%), platelets decreased (45% vs 8%), calcium decreased (25% vs 11%), nausea (21% vs 17%), decreased appetite (20% vs 14%), sodium decreased (22% vs 20%), phosphate decreased (17% vs 13%), fractures (14% vs 10%), magnesium decreased (14% vs 12%), dizziness (13% vs 9%), bilirubin increased (11% vs 7%), potassium decreased (11% vs 7%), and dysgeusia (10% vs 4.5%).

Clinically relevant adverse reactions in <10% of patients who received TALZENNA with enzalutamide included abdominal pain (9%), vomiting (9%), alopecia (7%), dyspepsia (4%), venous thromboembolism (3%) and stomatitis (2%).

DRUG INTERACTIONS

Coadministration with P-gp inhibitors The effect of coadministration of P-gp inhibitors on talazoparib exposure when TALZENNA is taken with enzalutamide has not been studied. Monitor patients for increased adverse reactions and modify the dosage as recommended for adverse reactions when TALZENNA is coadministered with a P-gp inhibitor.

Coadministration with BCRP inhibitors Monitor patients for increased adverse reactions and modify the dosage as recommended for adverse reactions when TALZENNA is coadministered with a BCRP inhibitor. Coadministration of TALZENNA with BCRP inhibitors may increase talazoparib exposure, which may increase the risk of adverse reactions.

USE IN SPECIFIC POPULATIONS

Males of Reproductive Potential Based on animal studies, TALZENNA may impair fertility.

Renal Impairment The recommended dosage of TALZENNA for patients with moderate renal impairment (CLcr 30 – 59 mL/min) is 0.35 mg taken orally once daily with enzalutamide. The recommended dosage of TALZENNA for patients with severe renal impairment (CLcr 15 – 29 mL/min) is 0.25 mg taken orally once daily with enzalutamide. No dose adjustment is required for patients with mild renal impairment. TALZENNA has not been studied in patients requiring hemodialysis.

Please see full U.S. Prescribing Information and Patient Information for TALZENNA (talazoparib) at www.TALZENNA.com.

About XTANDI (enzalutamide)

XTANDI (enzalutamide) is an androgen receptor pathway inhibitor. XTANDI is a standard of care and has received regulatory approvals in one or more countries around the world for use in people with metastatic hormone-sensitive prostate cancer (mHSPC), metastatic castration-resistant prostate cancer (mCRPC), non-metastatic castration-resistant prostate cancer (nmCRPC) and non-metastatic hormone-sensitive prostate cancer (nmHSPC) with high-risk biochemical recurrence (BCR). XTANDI is currently approved for one or more of these indications in more than 80 countries, including in the United States, European Union and Japan. Over 1.5 million patients have been treated with XTANDI globally.7

About XTANDI (enzalutamide) and Important Safety Information

XTANDI (enzalutamide) is indicated for the treatment of patients with:

castration-resistant prostate cancer (CRPC)
metastatic castration-sensitive prostate cancer (mCSPC)
nonmetastatic castration sensitive prostate cancer (nmCSPC) with biochemical recurrence at high risk for metastasis (high-risk BCR)
Important Safety Information

Warnings and Precautions

Seizure occurred in 0.6% of patients receiving XTANDI in eight randomized clinical trials. In a study of patients with predisposing factors for seizure, 2.2% of XTANDI-treated patients experienced a seizure. It is unknown whether anti-epileptic medications will prevent seizures with XTANDI. Patients in the study had one or more of the following predisposing factors: use of medications that may lower the seizure threshold, history of traumatic brain or head injury, history of cerebrovascular accident or transient ischemic attack, and Alzheimer’s disease, meningioma, or leptomeningeal disease from prostate cancer, unexplained loss of consciousness within the last 12 months, history of seizure, presence of a space occupying lesion of the brain, history of arteriovenous malformation, or history of brain infection. Advise patients of the risk of developing a seizure while taking XTANDI and of engaging in any activity where sudden loss of consciousness could cause serious harm to themselves or others. Permanently discontinue XTANDI in patients who develop a seizure during treatment.

Posterior Reversible Encephalopathy Syndrome (PRES) There have been reports of PRES in patients receiving XTANDI. PRES is a neurological disorder that can present with rapidly evolving symptoms including seizure, headache, lethargy, confusion, blindness, and other visual and neurological disturbances, with or without associated hypertension. A diagnosis of PRES requires confirmation by brain imaging, preferably MRI. Discontinue XTANDI in patients who develop PRES.

Hypersensitivity reactions, including edema of the face (0.5%), tongue (0.1%), or lip (0.1%) have been observed with XTANDI in eight randomized clinical trials. Pharyngeal edema has been reported in post-marketing cases. Advise patients who experience any symptoms of hypersensitivity to temporarily discontinue XTANDI and promptly seek medical care. Permanently discontinue XTANDI for serious hypersensitivity reactions.

Ischemic Heart Disease In the combined data of five randomized, placebo-controlled clinical studies, ischemic heart disease occurred more commonly in patients on the XTANDI arm compared to patients on the placebo arm (3.5% vs 2%). Grade 3-4 ischemic events occurred in 1.8% of patients on XTANDI versus 1.1% on placebo. Ischemic events led to death in 0.4% of patients on XTANDI compared to 0.1% on placebo. Monitor for signs and symptoms of ischemic heart disease. Optimize management of cardiovascular risk factors, such as hypertension, diabetes, or dyslipidemia. Discontinue XTANDI for Grade 3-4 ischemic heart disease.

Falls and Fractures occurred in patients receiving XTANDI. Evaluate patients for fracture and fall risk. Monitor and manage patients at risk for fractures according to established treatment guidelines and consider use of bone-targeted agents. In the combined data of five randomized, placebo-controlled clinical studies, falls occurred in 12% of patients treated with XTANDI compared to 6% of patients treated with placebo. Fractures occurred in 13% of patients treated with XTANDI and in 6% of patients treated with placebo.

Embryo-Fetal Toxicity The safety and efficacy of XTANDI have not been established in females. XTANDI can cause fetal harm and loss of pregnancy when administered to a pregnant female. Advise males with female partners of reproductive potential to use effective contraception during treatment with XTANDI and for 3 months after the last dose of XTANDI.

Dysphagia or Choking Severe dysphagia or choking, including events that could be life-threatening requiring medical intervention or fatal, can occur due to XTANDI product size. Advise patients to take each capsule or tablet whole with a sufficient amount of water to ensure that all medication is successfully swallowed. Consider use of a smaller tablet size of XTANDI in patients who have difficulty swallowing. Discontinue XTANDI for patients who cannot swallow capsules or tablets.

Interference with Immunoassay Measurement of Digoxin XTANDI can interfere with certain digoxin immunoassays (e.g., Chemiluminescent Microparticle Immunoassays), resulting in falsely elevated digoxin plasma concentration results. Notify the laboratory conducting the digoxin plasma concentration assay to use an appropriate method in patients receiving XTANDI and digoxin.

Adverse Reactions (ARs)

In the data from the five randomized placebo-controlled trials, the most common ARs (≥ 10%) that occurred more frequently (≥ 2% over placebo) in XTANDI-treated patients were musculoskeletal pain, fatigue, hot flush, constipation, decreased appetite, diarrhea, hypertension, hemorrhage, fall, fracture, and headache. In the bicalutamide-controlled study, the most common ARs (≥ 10%) reported in XTANDI-treated patients were asthenia/fatigue, back pain, musculoskeletal pain, hot flush, hypertension, nausea, constipation, diarrhea, upper respiratory tract infection, and weight loss.

In EMBARK, the placebo-controlled study of nonmetastatic CSPC (nmCSPC) with high-risk biochemical recurrence (BCR) patients, Grade 3 or higher adverse reactions during the total duration of treatment were reported in 46% of patients treated with XTANDI plus leuprolide, 50% of patients receiving XTANDI as a single agent, and 43% of patients receiving placebo plus leuprolide. Permanent treatment discontinuation due to adverse reactions during the total duration of treatment as the primary reason was reported in 21% of patients treated with XTANDI plus leuprolide, 18% of patients receiving XTANDI as a single agent, and 10% of patients receiving placebo plus leuprolide.

Lab Abnormalities: Lab abnormalities that occurred in ≥ 5% of patients, and more frequently (> 2%) in the XTANDI arm compared to placebo in the pooled, randomized, placebo-controlled studies are hemoglobin decrease, neutrophil count decreased, white blood cell decreased, hyperglycemia, hypermagnesemia, hyponatremia, hypophosphatemia, and hypercalcemia.

Hypertension: In the combined data from five randomized placebo-controlled clinical trials, hypertension was reported in 14.2% of XTANDI patients and 7.4% of placebo patients. Hypertension led to study discontinuation in < 1% of patients in each arm.

Drug Interactions

Effect of Other Drugs on XTANDI Avoid coadministration with strong CYP2C8 inhibitors. If coadministration cannot be avoided, reduce the dosage of XTANDI. Avoid coadministration with strong CYP3A4 inducers. If coadministration cannot be avoided, increase the dosage of XTANDI.

Effect of XTANDI on Other Drugs Avoid coadministration with certain CYP3A4, CYP2C9, and CYP2C19 substrates for which minimal decrease in concentration may lead to therapeutic failure of the substrate. If coadministration cannot be avoided, increase the dosage of these substrates in accordance with their Prescribing Information. In cases where active metabolites are formed, there may be increased exposure to the active metabolites.

Please access this link for XTANDI’S US Full Prescribing Information for additional safety information.

(Press release, Pfizer, MAR 19, 2026, View Source [SID1234663768])

Ractigen Therapeutics Announces China NMPA IND Approval for Phase II Clinical Trial of saRNA Therapy RAG-01 in Non-Muscle Invasive Bladder Cancer

On March 19, 2026 Ractigen Therapeutics, a pioneering clinical-stage biotechnology company developing innovative small activating RNA (saRNA) therapeutics, reported that China’s National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) has approved its Investigational New Drug (IND) application to initiate a Phase II clinical trial of RAG-01 for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC).

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This significant milestone makes RAG-01 the second saRNA therapeutic to receive IND approval in China, both of which were developed by Ractigen. This approval not only solidifies Ractigen’s global leadership in saRNA technology but also serves as a critical clinical validator for the company’s broader RNA activation (RNAa) platform and its LiCOTM delivery technology.

RAG-01 is a novel saRNA therapeutic designed to upregulate p21, a key regulator of cell cycle arrest and cellular senescence, to inhibit the abnormal proliferation of bladder cancer cells. This innovative saRNA therapeutic aims to treat NMIBC by increasing p21 mRNA and protein levels.

The progression of RAG-01 into Phase II clinical development follows highly encouraging Phase I data from Australia, which demonstrated clear target engagement (p21 protein upregulation), a favorable safety profile, and promising complete response (CR) signals.

"We are very pleased to receive IND approval from the China NMPA CDE for the Phase II trial of RAG-01," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "This approval is an important validation of both the RAG-01 program and the broader potential of saRNA therapeutics. RAG-01 reflects our innovation strategy of activating endogenous disease-relevant genes that have long been considered difficult to drug. We believe this differentiated mechanism, together with local bladder delivery, may offer a meaningful new treatment option for patients with NMIBC. We look forward to working closely with investigators and regulatory authorities to advance this program."

The Phase II study is a randomized, controlled, multi-center trial designed to evaluate the efficacy and safety of RAG-01 as monotherapy and in combination with chemotherapy in patients with intermediate- and high-risk NMIBC. The China study builds on encouraging preliminary data from the ongoing Phase I clinical trial in Australia, where RAG-01 has demonstrated favorable safety, clear target engagement, and anti-tumor activity.

About RAG-01

RAG-01 is a pioneering saRNA therapeutic candidate designed to activate the tumor suppressor gene p21 via RNA activation (RNAa). The product is administered through intravesical instillation using Ractigen’s proprietary LiCO delivery technology. In the Phase I clinical trial conducted in Australia, RAG-01 has shown encouraging preliminary safety, target engagement, and efficacy data. In 2024, RAG-01 received IND clearance from the U.S. Food and Drug Administration (FDA) and was granted Fast Track designation.

About NMIBC

Non-muscle invasive bladder cancer (NMIBC) is a common malignancy confined to the lining of the bladder. The standard first-line treatment is transurethral resection of the bladder tumor (TURBT) followed by intravesical BCG or chemotherapy. However, a significant proportion of patients experience treatment failure or multiple recurrences, highlighting the need for new and effective treatment options.

About RNAa

RNA activation is a clinically validated platform technology developed by Dr. Long-Cheng Li and his team. It utilizes saRNAs to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This innovative technology holds vast potential for developing therapeutic drugs across various diseases, particularly where traditional methods fall short.

(Press release, Ractigen, MAR 19, 2026, View Source [SID1234663766])