Latest news with #NMIBC
Medscape
a day ago
- Health
- Medscape
Q&A: Bladder Cancer Treatment Challenges
Kyle A. Richards, MD Bladder cancer is a prevalent malignancy affecting the urinary system, with symptoms including gross or microscopic hematuria, urinary frequency, and pelvic pain. Medscape spoke with Kyle A. Richards, MD, an associate professor in the Department of Urology at University of Wisconsin School of Medicine and Public Health, about the current challenges in bladder cancer diagnosis and treatment. What are the challenges with managing early stage bladder cancer? Most patients are diagnosed with early stage bladder cancer, or non-muscle invasive bladder cancer (NMIBC). Once the initial cancer is removed via transurethral resection of bladder tumor (TURBT), lifelong surveillance is needed to detect recurrence, which may occur at any time in a high rate of patients. For high-risk NMIBC, monitoring using office cystoscopy is recommended every 3 months for the first 2 years after diagnosis. Noninvasive urine biomarkers aim to reduce this significant burden. One such biomarker, CxMonitor, has shown promise in early phase clinical studies, but additional trials are needed. Another challenge is the difficulty of detecting all the cancer burden in the bladder following TURBT using the typical 'white light' source. Narrow band imaging and blue light cystoscopy aim to address this. Though the data are mixed, these technologies appear to improve detection rates and possibly the completeness of TURBT. However, a randomized trial comparing blue-light-guided and standard white-light-guided TURBT showed no difference in recurrence rates. How do you determine the staging of bladder cancer? Bladder cancer staging is based on the pathology report from TURBT plus axial imaging of the chest, abdomen, and pelvis to evaluate for metastatic disease. FDG PET scan may help assess for distant metastasis, and CT urogram often helps evaluate for upper tract urothelial tumors. For localized NMIBC, we typically assess the need for adjuvant therapy following TURBT to reduce the risk for recurrence or progression. Standard-of-care treatment for high-risk NMIBC is bacillus Calmette-Guérin (BCG). Because of the BCG shortage worldwide and lack of responsiveness to BCG in some patients, additional treatments have been explored, with sequential chemotherapy — specifically gemcitabine + docetaxel (gem/doce) — gaining the most traction. We have no reliable tools to predict which patients are less likely to respond to BCG or gem/doce. Computational histologic artificial intelligence has been used to develop assays to help predict response to BCG, preserve BCG for patients most likely to respond, and route others to different treatments. More investigation is needed to see how these tests perform prospectively. How do you manage patients with bladder cancer? Surgery is often needed to remove the primary bladder tumor. Adjuvant intravesical therapies following a complete TURBT are recommended for intermediate- or high-risk patients with NMIBC to help prevent recurrence and progression. For patients with suspected low-risk NMIBC, a single dose of gemcitabine immediately after TURBT reduced the risk for recurrence from 47% to 35%. Complications following TURBT are typically minor. In a prospective study of 159 patients undergoing TURBT, 10% had an unplanned emergency room visit, 79% experienced hematuria, and one third had bladder spasms and incontinence. Patient education and preoperative counseling may help reduce the burden of complications and postoperative readmission. Can you tell us about recently approved bladder cancer therapies? Perioperative durvalumab was studied in combination with gemcitabine and cisplatin (GC) for neoadjuvant therapy in patients with muscle invasive bladder cancer. This three-drug regimen was compared to the standard GC regimen in the NIAGARA trial. Overall survival at 2 years following cystectomy was improved by 8% in the durvalumab arm. Nogapendekin alfa inbakicept was evaluated in patients with NMIBC unresponsive to BCG. This drug modulates the local immune microenvironment in the bladder as an IL-15 super-agonist and must be given with BCG. In the pivotal study, 71% of patients had an initial complete response with a median duration of 26 months. Mitomycin intravesical solution has been investigated in patients with low-grade intermediate-risk NMIBC, who often must undergo frequent TURBTs that can negatively affect quality of life. This agent, given intravesically, delivers a slow release of chemotherapy that ablates the tumors. With this strategy, 80% of patients were tumor free without requiring surgery at 3 months, and 82% remained free of cancer 12 months later. It has similar efficacy to TURBT but involves six weekly instillations vs one trip to the operating room. Can you tell us more about upcoming therapies and clinical trial results in the near future? I will be excited to see the results from the BRIDGE trial (ECOG-ACRIN EA8212), which met its patient accrual in July 2025. This trial randomized over 700 newly diagnosed patients who were BCG naive with high-grade NMIBC to BCG vs gem/doce. The primary objective is to assess event-free survival. Patient-reported outcomes are also being collected. TAR-200, which is under FDA review, involves a novel drug delivery system that is inserted in the bladder and slowly releases gemcitabine to treat early stages of bladder cancer. A July 2025 press release cited an 82% complete response rate against bladder cancer, with 52% remaining cancer free for at least 1 year.
Yahoo
08-08-2025
- Business
- Yahoo
CG Oncology Reports Second Quarter 2025 Financial Results and Provides Business Updates
- Announced best-in-disease durability data in BOND-003 Cohort C and promising early signal in Cohort P for cretostimogene grenadenorepvec at the American Urological Association Annual Meeting - - Initiated CORE-008 Cohort CX evaluating the combination of cretostimogene and gemcitabine in patients with high-risk (HR) BCG-exposed NMIBC - - Announced unanimous verdict that CG Oncology owes no future royalties or other payments to ANI Pharmaceuticals - IRVINE, Calif., Aug. 08, 2025 (GLOBE NEWSWIRE) -- CG Oncology, Inc. (NASDAQ: CGON), a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients with bladder cancer, today reported financial results for the second quarter ended June 30, 2025, and provided business updates. 'In the second quarter, we announced best-in-disease durability and tolerability from the Phase 3 BOND-003 Cohort C registrational trial, building upon the body of evidence demonstrating the power of cretostimogene's unique dual mechanism of action, and its potential to treat intermediate-risk and high-risk NMIBC,' said Arthur Kuan, Chairman & Chief Executive Officer at CG Oncology. 'We remain laser focused on bringing forward cretostimogene, our potentially breakthrough backbone treatment to patients. The recent positive outcome of the ANI litigation allows us to continue to focus our resources and energy on delivering this innovative therapy. We are poised to initiate our BLA submission for cretostimogene in the fourth quarter of the year for the treatment of patients with HR NMIBC unresponsive to BCG.' Corporate Highlights Presented Best-in-Disease Durability and Tolerability Data in BOND-003 Cohort C and Promising Early Signal in Cohort P for Cretostimogene at a Plenary Session at the American Urological Association (AUA) Annual Meeting: On April 26th at the AUA Annual Meeting, the Company presented best-in-disease durability and tolerability data from Cohort C of the Phase 3 BOND-003 clinical trial that showed a 75.5% complete response (CR) at any time, with 34 confirmed CRs at 24 months and 9 patients pending their 24-month assessment as of the cutoff date of March 14, 2025. The 12- and 24-month CR rates are 50.7% and 42.3% by K-M estimation respectively. Median duration of response is 28 months and is ongoing. Notably, 97.3% of patients were free from progression to muscle invasive disease at 24 months. Additionally, Cohort P, which is in patients with BCG-unresponsive Ta/T1 papillary disease without carcinoma in situ (CIS), showed an estimated 90.5% high-grade recurrence-free survival at 3 and 9 months in 24 treated patients. Initiated CORE-008 Cohort CX clinical trial of cretostimogene + gemcitabine in HR BCG-exposed NMIBC: In April, the Company initiated CORE-008 Cohort CX evaluating the combination of cretostimogene and gemcitabine, given either concurrently or sequentially, in patients with HR BCG-exposed NMIBC, including patients with CIS and with or without Ta/T1 disease and patients with only Ta/T1 disease. Announced Unanimous Verdict that CG Oncology Owes No Future Royalties or Other Payments to ANI Pharmaceuticals: On July 29th, the Company announced that a jury in the Superior Court of the State of Delaware unanimously found in favor of CG Oncology on all claims in a March 2024 lawsuit brought by ANI Pharmaceuticals, Inc. (ANI). The jury unanimously rejected all of ANI's claims for unjust enrichment damages after Judge Sheldon K. Rennie, on July 16, 2025, had ruled in favor of CG Oncology that, as a matter of law, ANI was not entitled to any royalties on future sales of cretostimogene grenadenorepvec. As a result, CG Oncology will not owe ANI a future royalty of 5% on commercial sales of cretostimogene, no damages have been awarded to ANI, and there are no further payments due to ANI under the 2010 agreement between ANI and CG Oncology. The Company will continue to vigorously defend any post-trial motions and appeals brought by ANI. Anticipated Upcoming Milestones PIVOT-006 (intermediate-risk NMIBC): Phase 3 enrollment completion in 3Q'25 Initiation of BLA submission in 4Q'25 for cretostimogene monotherapy in HR BCG-unresponsive NMIBC with CIS with or without Ta/T1 disease BOND-003 Cohort P (HR BCG-unresponsive NMIBC in Ta/T1 disease without CIS): Topline data from the Phase 3 clinical trial of cretostimogene monotherapy in 4Q'25 CORE-008 Cohort A (HR BCG-naïve NMIBC with CIS +/- Ta/T1): Topline data from the Phase 2 clinical trial of cretostimogene monotherapy in 4Q'25 CORE-008 Cohort CX (HR BCG-exposed NMIBC): Topline data from the Phase 2 clinical trial of the combination of cretostimogene with gemcitabine in 1H'26 Second Quarter 2025 Financial Highlights Cash Position: Cash and cash equivalents and marketable securities as of June 30, 2025, were $661.1 million, compared with $688.4 million as of March 31, 2025. Based on current operating plans, the Company expects its existing cash, cash equivalents and marketable securities will be sufficient to fund operations into the first half of 2028. Research and Development (R&D) Expenses: R&D expenses for the three months ended June 30, 2025 were $31.3 million compared with $18.5 million for the three months ended June 30, 2024. The increase was primarily due to an increase in clinical trial expenses and an increase in compensation costs due to increased headcount. General and Administrative (G&A) Expenses: G&A expenses for the three months ended June 30, 2025 were $17.4 million compared with $7.5 million for the three months ended June 30, 2024. The increase was primarily attributed to an increase in personnel-related expenses, including compensation costs from increased headcount and an increase in legal expenses. Net Loss: Net loss was $41.4 million, or ($0.54) per share, for the three months ended June 30, 2025, compared to a net loss of $18.9 million, or ($0.28) per share, for the three months ended June 30, 2024. About Cretostimogene GrenadenorepvecCretostimogene is an investigational, intravesically delivered oncolytic immunotherapy that has been studied in a clinical development program, which includes more than 400 patients with Non-Muscle Invasive Bladder Cancer (NMIBC). This program includes two Phase 3 clinical trials: BOND-003 for high-risk BCG-unresponsive NMIBC and PIVOT-006 for intermediate-risk NMIBC. CG Oncology also has a Phase 2 trial, CORE-008, evaluating the safety and efficacy of cretostimogene in high-risk NMIBC. Additionally, we have initiated an Expanded Access Program for cretostimogene in North America for patients who are unresponsive to BCG and meet certain program eligibility requirements. Cretostimogene is an investigational candidate, and its safety and efficacy have not been established by the FDA or any other health authority. About CG OncologyCG Oncology is a late-stage clinical biopharmaceutical company focused on developing and commercializing a potential backbone bladder-sparing therapeutic for patients afflicted with bladder cancer. CG Oncology sees a world where urologic cancer patients may benefit from our innovative immunotherapies to live with dignity and have an enhanced quality of life. To learn more, please visit: Forward-Looking StatementsCG Oncology cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. The forward-looking statements are based on our current beliefs and expectations and include, but are not limited to, statements regarding our anticipated cash runway, future results of operations and financial position; the anticipated timing and conduct of our ongoing and planned clinical trials and preclinical studies for cretostimogene, including anticipated next milestones in our development pipeline; the timing and likelihood of regulatory filings and approvals for cretostimogene; the potential therapeutic benefits of cretostimogene for high-risk and intermediate-risk NMIBC patients; and that cretostimogene has best-in-disease durability and tolerability data. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: interim results of a clinical trial are not necessarily indicative of final results and one or more of the clinical outcomes may materially change as patient enrollment continues, following more comprehensive reviews of the data, and as more patient data becomes available; potential delays in the commencement, enrollment and completion of clinical trials, including the BOND-003 and PIVOT-006 trials; we may use our capital resources sooner than expected and they may be insufficient to allow us to achieve our anticipated milestones; our dependence on third parties in connection with manufacturing, shipping and clinical and preclinical testing; results from earlier clinical trials and preclinical studies not necessarily being predictive of future results; unexpected adverse side effects or inadequate efficacy of cretostimogene that may limit its development, regulatory approval, and/or commercialization; and other risks described in our filings with the Securities and Exchange Commission (SEC), including under the heading 'Risk Factors' in our annual report on Form 10-K, as supplemented in Part II, Item 1A, 'Risk Factors' of our quarterly report on Form 10-Q for the quarter ended June 30, 2025, and other filings that we make with the SEC from time to time (which are available at You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Contacts:Media Sarah ConnorsVice President, Communications and Patient Advocacy, CG Oncology(508) Investor RelationsMegan KnightVice President, Investor Relations, CG Oncology(619) CG ONCOLOGY, INC. Condensed Consolidated Statements of Operations and Comprehensive Loss(In thousands, except share and per share amounts)(unaudited) Three Months EndedJune 30, Six Months EndedJune 30, 2025 2024 2025 2024 Revenues License and collaboration revenue $ — $ 111 $ 52 $ 640 Operating expenses Research and development 31,331 18,470 58,799 35,680 General and administrative 17,410 7,494 32,198 13,282 Total operating expenses 48,741 25,964 90,997 48,962 Loss from operations (48,741 ) (25,853 ) (90,945 ) (48,322 ) Other income (expense), net: Interest income, net 7,319 6,943 15,066 12,487 Other expense (income), net (4 ) 8 1 (1 ) Total other income, net 7,315 6,951 15,067 12,486 Net loss and comprehensive loss $ (41,426 ) $ (18,902 ) $ (75,878 ) $ (35,836 ) Net loss per share, basic and diluted $ (0.54 ) $ (0.28 ) $ (1.00 ) $ (0.63 ) Weighted average shares of common stock outstanding, basicand diluted 76,226,829 66,649,443 76,207,333 56,857,104 CG ONCOLOGY, INC. Consolidated Balance Sheet Data(In thousands) June 30,2025(unaudited) December 31,2024 Cash, cash equivalents, and marketable securities $ 661,052 $ 741,998 Total assets 701,445 754,797 Total liabilities 31,087 21,420 Total stockholders' equity 670,358 733,377 Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
25-07-2025
- Business
- Business Wire
ImmunityBio Reports 60% Increase in Revenue in Q2 2025, with Year-to-Date Sales of $43 Million and 246% Unit Growth Since J-Code with Regulatory Updates
CULVER CITY, Calif.--(BUSINESS WIRE)--ImmunityBio, Inc. (NASDAQ: IBRX) Q2 2025 Revenue Growth with Continued Strong Sales Momentum: $26.4 million, up 60% from Q1 2025, with year-to-date sales of ~$43 million. ANKTIVA ® Unit Growth Since J-Code: 246% unit sales volume growth in 1H 2025 compared to 2H 2024. Cash Position: $153.7 million in cash, cash equivalents and marketable securities as of June 30, 2025. NSCLC: Initiated randomized clinical trial (RCT) ResQ201A with N-803 + tislelizumab in 2 nd line lung cancer; US sites initiated, submitted clinical trial applications in EU, UK; Canada and Asia filings planned. Lymphopenia: FDA supportive of new data; reaffirmed RMAT/EAP; collaborative discussion outlining regulatory endpoints, trial design and registrational pathways to full approval for the treatment of lymphopenia with randomized trial design in progress. Full Enrollment Reached in the Randomized NCI Cancer Prevention Clinical Trial Using ANKTIVA + Adenovirus Vaccine in 186 Patients with Lynch Syndrome. United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA) marketing authorization application of ANKTIVA approved. Papillary NMIBC: Discussion with FDA regarding filing status of supplemental BLA. ImmunityBio performing ongoing evaluation of next steps to address the Refuse to File decision by the FDA, following June Type A meeting with the Agency. New updated data on Papillary disease provided to the FDA and discussions with Agency to continue. Separately, ImmunityBio has applied to the National Comprehensive Cancer Network (NCCN) to seek expansion of the BCG-unresponsive NMIBC guidelines to include papillary-only disease. Financial Overview In the second quarter of 2025, ImmunityBio reported $26.4 million in revenue, representing a 60% increase from $16.5 million in the first quarter of 2025. This growth reflects continued commercial traction of ANKTIVA + BCG in BCG-unresponsive non-muscle invasive bladder cancer with CIS with or without Papillary tumors. The 1H 2025 sales of $42.9 million represents a 246% increase in unit volume during the first two quarters of 2025 since the J-code approval versus the last two quarters of 2024. The Company ended the quarter with $153.7 million in cash, cash equivalents and marketable securities as of June 30, 2025. Non-Muscle Invasive Bladder Cancer (Papillary NMIBC) ImmunityBio conducted a Type A meeting with the FDA in June to discuss its program targeting papillary-only NMIBC and the Agency's response to the supplemental BLA filing. Contrary to the advice the FDA gave the Company in January 2025 to submit the supplemental BLA, the FDA responded with a Refuse-to-File (RTF) notice in May on the basis of requiring a randomized controlled trial (RCT) against chemotherapy. At the June meeting, ImmunityBio provided new data regarding the updated results since the initial BLA filing of papillary only data as well as real-world data of chemotherapy just published in this indication. In the papillary only NMIBC new data based on 26 of the 100 subjects in Cohort A and 80 subjects in Cohort B (Papillary Alone) of our QUILT-3.032 trial, demonstrated long-term (36-month) progression free survival and bladder sparing with ANKTIVA + BCG. ImmunityBio presented the newly published real-world data which demonstrates that compared to chemotherapy, ANKTIVA + BCG led to improved outcomes of progression free survival and cystectomy avoidance at 36-months. To our knowledge, the results to date of ANKTIVA + BCG represent the longest duration of follow-up with the longest duration of bladder sparing in these subjects. The Company indicated at the meeting that it would seek a new meeting request with this new data and withdraw the prior supplemental BLA filing; however, the Company is re-evaluating this approach in consultation with its regulatory counsel and may seek to amend the initial filing with the new data rather than withdrawing it, with a commitment to initiate a randomized controlled trial of chemotherapy free ANKTIVA + BCG versus chemotherapy in the papillary alone indication. Separately, ImmunityBio has applied to the National Comprehensive Cancer Network (NCCN) to seek expansion of the BCG-unresponsive NMIBC guidelines to include papillary-only disease, in addition to the currently recognized CIS with or without papillary disease. The NCCN is expected to review the submission at its August 2025 meeting. Non-Small Cell Lung Cancer (NSCLC) ImmunityBio has launched ResQ201A, a randomized controlled trial, in the United States, evaluating its IL-15 superagonist N-803 in combination with tislelizumab, a PD-1 checkpoint inhibitor from BeOne Medicines in patients with 2 nd line lung cancer who were progressing on checkpoint inhibitors. The Company has also submitted clinical trial applications for ResQ201A in the European Union and the United Kingdom, with Canada expected to be submitted in early Q3 2025, and with plans underway to submit in Asia. Lymphopenia The Company also met with the Division of Non-Malignant Hematology at the FDA in June to present updated data from its lymphopenia program. The Division was supportive of the findings including the underlying science of stimulating lymphocytes with ANKTIVA and expressed a desire to support an efficient path to approval, noting that additional time will be required to finalize the appropriate development plan. Expanded Access Program (EAP) authorization has been activated for the indication for all solid tumors in patients who have failed first-line treatment on chemotherapy, radiotherapy or immunotherapy and exhibit low Absolute Lymphocyte Counts (ALC < 1,000/μL). Disclaimer Regarding Financial Overview The information and amounts presented above, including under the caption 'Financial Overview,' reflects the Company's preliminary estimates based solely upon information available to it as of the date of this press release, and the amounts reported are not a comprehensive statement of its financial results or position as of June 30, 2025. Any actual amount that the Company reports in its Quarterly Report on Form 10-Q for the period ended June 30, 2025 will be subject to its financial closing procedures and any final adjustments that may be made prior to the time its financial results for the period ended June 30, 2025 are finalized. As a result, these preliminary estimates may differ materially from the actual results that will be reflected in the Company's condensed consolidated financial statements for the quarter when they are completed and publicly disclosed. About ANKTIVA The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of key immune cells—NK and CD8+ killer T cells—that are involved in killing cancer cells. By activating NK cells, ANKTIVA overcomes the tumor escape phase of clones resistant to T cells and restores memory T cell activity with resultant prolonged duration of complete response. ANKTIVA is a first-in-class IL-15 agonist IgG1 fusion complex, consisting of an IL-15 mutant (IL-15N72D) fused with an IL-15 receptor alpha, which binds with high affinity to IL-15 receptors on NK, CD4+, and CD8+ T cells. This fusion complex of ANKTIVA mimics the natural biological properties of the membrane-bound IL-15 receptor alpha, delivering IL-15 by dendritic cells and drives the activation and proliferation of NK cells with the generation of memory killer T cells that have retained immune memory against these tumor clones. The proliferation of the trifecta of these immune killing cells and the activation of trained immune memory results in immunogenic cell death, inducing a state of equilibrium with durable complete responses. ANKTIVA has improved pharmacokinetic properties, longer persistence in lymphoid tissues, and enhanced anti-tumor activity compared to native, non-complexed IL-15 in-vivo. ANKTIVA was approved by the FDA in 2024 and by UK MHRA in 2025 for BCG-unresponsive non-muscle invasive bladder cancer CIS with or without papillary tumors. For more information, visit About ImmunityBio ImmunityBio is a vertically-integrated commercial stage biotechnology company developing next-generation therapies that bolster the natural immune system to defeat cancers and infectious diseases. The Company's range of immunotherapy and cell therapy platforms, alone and together, act to drive and sustain an immune response with the goal of creating durable and safe protection against disease. Designated an FDA Breakthrough Therapy, ANKTIVA is the first FDA-approved immunotherapy for non-muscle invasive bladder cancer CIS that activates natural killer cells, T cells, and memory T cells for a long-duration response. The Company is applying its science and platforms to treating cancers, including the development of potential cancer vaccines, as well as developing immunotherapies and cell therapies that we believe sharply reduce or eliminate the need for standard high-dose chemotherapy. These platforms and their associated product candidates are designed to be more effective, accessible, and easily administered than current standards of care in oncology and infectious diseases. For more information, visit (Founder's Vision) and connect with us on X (Twitter), Facebook, LinkedIn, and Instagram. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, such as statements regarding discussions and meetings with the U.S. FDA, including with respect to the previously reported RTF letter received by the Company and potential implications thereof, potential next steps, decisions and timeline related to the Company's regulatory submissions and strategy, financial results anticipated to be reported in future SEC filings, clinical trial data and potential results and implications to be drawn therefrom, the expectation that the EAP described herein will enable patients to have access to ANKTIVA for the indication described, the RMAT designation as previously reported and potential results therefrom and regulatory submissions in connection therewith, the belief that ALC levels and NLR levels obtained from a CBC are predictors of clinical benefit and outcomes relating to overall survival, clinical trial and expanded access program enrollment, timing, data and potential results to be drawn therefrom, anticipated components of ImmunityBio's Cancer BioShield TM platform, anticipated review timeline for the Company's NCCN guidelines submission in NMIBC papillary only and potential implications therefrom, the development of therapeutics for cancer and infectious diseases, potential benefits to patients, potential treatment outcomes for patients, the described mechanism of action and results and contributions therefrom, potential future uses and applications of ANKTIVA alone or in combination with other therapeutic agents for the prevention or reversal of lymphopenia, potential future uses and applications of ANKTIVA alone or in combination with other therapeutic agents across multiple tumor types and indications and for potential applications beyond oncology, potential regulatory pathways and the regulatory review process and timing thereof, the application of the Company's science and platforms to treat cancers or develop cancer vaccines, immunotherapies and cell therapies that have the potential to change the paradigm in cancer care, and ImmunityBio's approved product and investigational agents as compared to existing treatment options, among others. Statements in this press release that are not statements of historical fact are considered forward-looking statements, which are usually identified by the use of words such as 'anticipates,' 'believes,' 'continues,' 'goal,' 'could,' 'estimates,' 'scheduled,' 'expects,' 'intends,' 'may,' 'plans,' 'potential,' 'predicts,' 'indicate,' 'projects,' 'is,' 'seeks,' 'should,' 'will,' 'strategy,' and variations of such words or similar expressions. Statements of past performance, efforts, or results of our preclinical and clinical trials, about which inferences or assumptions may be made, can also be forward-looking statements and are not indicative of future performance or results. Forward-looking statements are neither forecasts, promises nor guarantees, and are based on the current beliefs of ImmunityBio's management as well as assumptions made by and information currently available to ImmunityBio. Such information may be limited or incomplete, and ImmunityBio's statements should not be read to indicate that it has conducted a thorough inquiry into, or review of, all potentially available relevant information. Such statements reflect the current views of ImmunityBio with respect to future events and are subject to known and unknown risks, including business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about ImmunityBio, including, without limitation, (i) risks and uncertainties regarding the FDA regulatory submission, filing and review process and the timing thereof, (ii) whether the RMAT designation will lead to an accelerated review or approval, of which there can be no assurance, (iii) risks and uncertainties regarding commercial launch execution, success and timing, (iv) risks and uncertainties regarding participation and enrollment and potential results from the expanded access clinical investigation program described herein, (v) whether clinical trials will result in registrational pathways and the risks, (vi) whether clinical trial data will be accepted by regulatory agencies, (vii) the ability of ImmunityBio to continue its planned preclinical and clinical development of its development programs through itself and/or its investigators, and the timing and success of any such continued preclinical and clinical development, patient enrollment and planned regulatory submissions, (viii) potential delays in product availability and regulatory approvals, (ix) ImmunityBio's ability to retain and hire key personnel, (x) ImmunityBio's ability to obtain additional financing to fund its operations and complete the development and commercialization of its various product candidates, (xi) potential product shortages or manufacturing disruptions that may impact the availability and timing of product, (xii) ImmunityBio's ability to successfully commercialize its approved product and product candidates, (xiii) ImmunityBio's ability to scale its manufacturing and commercial supply operations for its approved product and future approved products, (xiv) whether the NCCN will review and/or approve the Company's submission described herein on the anticipated timeline or at all, and (xv) ImmunityBio's ability to obtain, maintain, protect, and enforce patent protection and other proprietary rights for its product candidates and technologies. More details about these and other risks that may impact ImmunityBio's business are described under the heading 'Risk Factors' in the Company's Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on March 3, 2025, and the Company's Form 10-Q filed with the SEC on May 12, 2025, and in subsequent filings made by ImmunityBio with the SEC, which are available on the SEC's website at ImmunityBio cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date hereof.
Business Wire
12-06-2025
- Business
- Business Wire
enGene Reports Second Quarter 2025 Financial Results and Provides Business Update
BOSTON & MONTREAL--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN, or 'enGene' or the 'Company'), a clinical-stage, non-viral genetic medicines company, today announced its financial results for the second quarter ended April 30, 2025, and provided a business update. 'We have seen strong enrollment in the pivotal cohort of our LEGEND study,' said Ron Cooper, Chief Executive Officer of enGene. 'This positions us to stay on track for our planned trial updates across all cohorts in the second half of 2025 and a potential BLA filing in mid-2026, advancing our goal to introduce a novel, non-viral therapy that could redefine treatment for patients with high-risk non-muscle invasive bladder cancer.' Recent Corporate Updates Key executive hires and management appointments: In May 2025, the Company announced the appointment of Amy Pott as Chief Global Commercialization Officer. Ms. Pott joined enGene from Astellas Pharma, where she most recently served as Senior Vice President, Strategic Brand Marketing, Ophthalmics and Rare Diseases, and previously as Head of Commercial, Gene Therapies. She will serve as the Company's first dedicated executive for commercialization planning and execution. LEGEND study enrollment update: Over the course of the first and second quarters of 2025, the Company expanded its clinical footprint for the LEGEND study with the addition of trial sites in Europe and Asia. The pivotal cohort evaluating detalimogene voraplasmid (also known as detalimogene, and previously EG-70) in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) remains on track for our planned BLA in mid-2026. The Company expects to provide an update from LEGEND's pivotal cohort in the second half of 2025. European Medicines Agency (EMA) Scientific Advice: The Company shared detalimogene preclinical and clinical data through the EMA Scientific Advice process. During the dialogue, EMA indicated that it broadly agrees that these data could be suitable for a Conditional Marketing Authorization Application (CMA) submission for detalimogene in BCG-unresponsive NMIBC with CIS assuming a positive benefit-risk ratio. BIOTECanada Gold Leaf Biotech Company of the Year Award Winner: The Biotech Company of the Year Award recognizes a company that is transforming the future of healthcare through groundbreaking advancements. The Company is proud and honored to be recognized with this distinction. Second Quarter 2025 Financial Results As of April 30, 2025, cash, cash equivalents and marketable securities were $251.5 million. The Company expects that its existing cash, cash equivalents and marketable securities will fund operating expenses, debt obligations and capital expenditures into 2027. Three Months ended April 30, 2025 Total operating expenses were $27.1 million for the three months ended April 30, 2025, compared to $17.3 million for the three months ended April 30, 2024. Research and development expenses increased by $10.4 million, mainly due to increasing manufacturing and clinical costs related to our pivotal LEGEND study and personnel-related costs. General and administrative expenses decreased by $0.5 million, primarily driven by decreased reliance on professional services to support the Company's operation as a publicly traded company. For the three months ended April 30, 2025, net loss attributable to common shareholders was approximately $25.8 million, or $0.51 per share, compared to approximately $15.0 million, or $0.38 per share, for the same period for the three months ended April 30, 2024. The increase in net loss is mainly attributed to the increase in operating expenses, partially offset by net interest income earned during the period. About Non-Muscle Invasive Bladder Cancer (NMIBC) Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on our healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can take the form of papillary outgrowths from the bladder wall, which are typically resected, or carcinoma in situ (CIS), flat, multifocal lesions that are unable to be resected, and the two can co-occur. About 75-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50-70%) and are subject to full removal of the bladder (cystectomy) as a curative but life-altering next step. About Detalimogene Detalimogene is a novel, investigational, non-viral genetic medicine for patients with high-risk, NMIBC, including BCG-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response. Detalimogene was developed using the Company's Dually Derivatized Oligochitosan® (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, simplify safe handling and cold storage complexities, and streamline both manufacturing processes and administration paradigms. Detalimogene has received Fast Track designation from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive CIS NMIBC, with or without resected papillary tumors, who are unable to undergo cystectomy. Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. About the Pivotal LEGEND Trial Detalimogene is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk, NMIBC. LEGEND's pivotal cohort (Cohort 1) consists of approximately 100 patients with high-risk, BCG-unresponsive NMIBC with CIS (with or without papillary disease) and is designed to serve as the basis of the Company's planned Biologics License Application (BLA) filing. In addition to this pivotal cohort, LEGEND includes three additional cohorts, including NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3). The LEGEND trial is actively enrolling patients with sites participating in the USA, Canada, Europe, and the Asia-Pacific region. About enGene enGene is a clinical-stage biotechnology company mainstreaming genetic medicines through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene's lead program is detalimogene for patients with Non-Muscle Invasive Bladder Cancer (NMIBC) – a disease with a high clinical burden. Detalimogene is being evaluated in the ongoing multi-cohort LEGEND Phase 2 study, which includes a pivotal cohort studying detalimogene in Bacillus Calmette-Guérin (BCG)-unresponsive patients with carcinoma in situ (CIS). Detalimogene was developed using enGene's proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA. For more information, visit Forward-Looking Statements Certain statements contained in this press release may constitute 'forward-looking statements' within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and 'forward-looking information' within the meaning of Canadian securities laws (collectively, 'forward-looking statements'). enGene's forward-looking statements include, but are not limited to, statements regarding enGene's management teams' expectations, hopes, beliefs, intentions, goals, or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words 'anticipate', 'appear', 'approximate', 'believe', 'continue', 'could', 'estimate', 'expect', 'foresee', 'intends', 'may', 'might', 'plan', 'possible', 'potential', 'predict', 'project', 'seek', 'should', 'would', and similar expressions (or the negative version of such words or expressions) may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: our plans regarding the timing of our planned BLA submission to the Food and Drug Administration, our expectations as to the timing and anticipated results of the LEGEND study, including the timing of preliminary data or other updates, our expectations regarding a potential CMA submission to the EMA, our expectations regarding completion of enrollment in the LEGEND study, including the timing, the potential benefits of detalimogene, including its potential impact on the treatment landscape and attractiveness to patients and physicians, the potential benefits of medicines developed with the DDX platform and the expected period over which we estimate our cash, cash equivalents and marketable securities will be sufficient to fund our current operating plan. Many factors, risks, uncertainties and assumptions could cause the Company's actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, the Company's ability to recruit and retain qualified scientific and management personnel, establish clinical trial sites and enroll patients in its clinical trials, execute on the Company's clinical development plans and ability to secure regulatory approval on anticipated timelines, and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission ('SEC') on EDGAR, including those described in the 'Risk Factors' section of the Company's Annual Report on Form 10-K for the fiscal year ended October 31, 2024 (copies of which may be obtained at or You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene's assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. enGene Holdings Inc. Condensed Consolidated Balance Sheet Information (unaudited) (Amounts in thousands of USD)
Associated Press
12-06-2025
- Business
- Associated Press
enGene Reports Second Quarter 2025 Financial Results and Provides Business Update
BOSTON & MONTREAL--(BUSINESS WIRE)--Jun 12, 2025-- enGene Holdings Inc. (Nasdaq: ENGN, or 'enGene' or the 'Company'), a clinical-stage, non-viral genetic medicines company, today announced its financial results for the second quarter ended April 30, 2025, and provided a business update. 'We have seen strong enrollment in the pivotal cohort of our LEGEND study,' said Ron Cooper, Chief Executive Officer of enGene. 'This positions us to stay on track for our planned trial updates across all cohorts in the second half of 2025 and a potential BLA filing in mid-2026, advancing our goal to introduce a novel, non-viral therapy that could redefine treatment for patients with high-risk non-muscle invasive bladder cancer.' Recent Corporate Updates Key executive hires and management appointments: In May 2025, the Company announced the appointment of Amy Pott as Chief Global Commercialization Officer. Ms. Pott joined enGene from Astellas Pharma, where she most recently served as Senior Vice President, Strategic Brand Marketing, Ophthalmics and Rare Diseases, and previously as Head of Commercial, Gene Therapies. She will serve as the Company's first dedicated executive for commercialization planning and execution. LEGEND study enrollment update: Over the course of the first and second quarters of 2025, the Company expanded its clinical footprint for the LEGEND study with the addition of trial sites in Europe and Asia. The pivotal cohort evaluating detalimogene voraplasmid (also known as detalimogene, and previously EG-70) in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) remains on track for our planned BLA in mid-2026. The Company expects to provide an update from LEGEND's pivotal cohort in the second half of 2025. European Medicines Agency (EMA) Scientific Advice: The Company shared detalimogene preclinical and clinical data through the EMA Scientific Advice process. During the dialogue, EMA indicated that it broadly agrees that these data could be suitable for a Conditional Marketing Authorization Application (CMA) submission for detalimogene in BCG-unresponsive NMIBC with CIS assuming a positive benefit-risk ratio. BIOTECanada Gold Leaf Biotech Company of the Year Award Winner: The Biotech Company of the Year Award recognizes a company that is transforming the future of healthcare through groundbreaking advancements. The Company is proud and honored to be recognized with this distinction. Second Quarter 2025 Financial Results As of April 30, 2025, cash, cash equivalents and marketable securities were $251.5 million. The Company expects that its existing cash, cash equivalents and marketable securities will fund operating expenses, debt obligations and capital expenditures into 2027. Three Months ended April 30, 2025 Total operating expenses were $27.1 million for the three months ended April 30, 2025, compared to $17.3 million for the three months ended April 30, 2024. Research and development expenses increased by $10.4 million, mainly due to increasing manufacturing and clinical costs related to our pivotal LEGEND study and personnel-related costs. General and administrative expenses decreased by $0.5 million, primarily driven by decreased reliance on professional services to support the Company's operation as a publicly traded company. For the three months ended April 30, 2025, net loss attributable to common shareholders was approximately $25.8 million, or $0.51 per share, compared to approximately $15.0 million, or $0.38 per share, for the same period for the three months ended April 30, 2024. The increase in net loss is mainly attributed to the increase in operating expenses, partially offset by net interest income earned during the period. About Non-Muscle Invasive Bladder Cancer (NMIBC) Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on our healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can take the form of papillary outgrowths from the bladder wall, which are typically resected, or carcinoma in situ (CIS), flat, multifocal lesions that are unable to be resected, and the two can co-occur. About 75-80% of new bladder cancer diagnoses are NMIBC. Patients suffering from high-risk NMIBC who are unresponsive to the standard of care, Bacillus Calmette-Guérin (BCG), face high rates of disease recurrence (50-70%) and are subject to full removal of the bladder (cystectomy) as a curative but life-altering next step. About Detalimogene Detalimogene is a novel, investigational, non-viral genetic medicine for patients with high-risk, NMIBC, including BCG-unresponsive disease. It is designed to be instilled in the bladder and elicit a powerful yet localized anti-tumor immune response. Detalimogene was developed using the Company's Dually Derivatized Oligochitosan® (DDX) platform, a technology designed to transform how gene therapies are accessed by patients and utilized by clinicians. Medicines developed with the DDX platform can potentially overcome the limitations of viral-based gene therapies, simplify safe handling and cold storage complexities, and streamline both manufacturing processes and administration paradigms. Detalimogene has received Fast Track designation from the U.S. Food and Drug Administration (FDA) based on its potential to address the high unmet medical need for patients with BCG-unresponsive CIS NMIBC, with or without resected papillary tumors, who are unable to undergo cystectomy. Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. About the Pivotal LEGEND Trial Detalimogene is being evaluated in the ongoing, open-label, multi-cohort, Phase 2 LEGEND trial to establish its safety and efficacy in high-risk, NMIBC. LEGEND's pivotal cohort (Cohort 1) consists of approximately 100 patients with high-risk, BCG-unresponsive NMIBC with CIS (with or without papillary disease) and is designed to serve as the basis of the Company's planned Biologics License Application (BLA) filing. In addition to this pivotal cohort, LEGEND includes three additional cohorts, including NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive high-risk NMIBC patients with papillary-only disease (Cohort 3). The LEGEND trial is actively enrolling patients with sites participating in the USA, Canada, Europe, and the Asia-Pacific region. About enGene enGene is a clinical-stage biotechnology company mainstreaming genetic medicines through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene's lead program is detalimogene for patients with Non-Muscle Invasive Bladder Cancer (NMIBC) – a disease with a high clinical burden. Detalimogene is being evaluated in the ongoing multi-cohort LEGEND Phase 2 study, which includes a pivotal cohort studying detalimogene in Bacillus Calmette-Guérin (BCG)-unresponsive patients with carcinoma in situ (CIS). Detalimogene was developed using enGene's proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA. For more information, visit Forward-Looking Statements Certain statements contained in this press release may constitute 'forward-looking statements' within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and 'forward-looking information' within the meaning of Canadian securities laws (collectively, 'forward-looking statements'). enGene's forward-looking statements include, but are not limited to, statements regarding enGene's management teams' expectations, hopes, beliefs, intentions, goals, or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words 'anticipate', 'appear', 'approximate', 'believe', 'continue', 'could', 'estimate', 'expect', 'foresee', 'intends', 'may', 'might', 'plan', 'possible', 'potential', 'predict', 'project', 'seek', 'should', 'would', and similar expressions (or the negative version of such words or expressions) may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: our plans regarding the timing of our planned BLA submission to the Food and Drug Administration, our expectations as to the timing and anticipated results of the LEGEND study, including the timing of preliminary data or other updates, our expectations regarding a potential CMA submission to the EMA, our expectations regarding completion of enrollment in the LEGEND study, including the timing, the potential benefits of detalimogene, including its potential impact on the treatment landscape and attractiveness to patients and physicians, the potential benefits of medicines developed with the DDX platform and the expected period over which we estimate our cash, cash equivalents and marketable securities will be sufficient to fund our current operating plan. Many factors, risks, uncertainties and assumptions could cause the Company's actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, the Company's ability to recruit and retain qualified scientific and management personnel, establish clinical trial sites and enroll patients in its clinical trials, execute on the Company's clinical development plans and ability to secure regulatory approval on anticipated timelines, and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission ('SEC') on EDGAR, including those described in the 'Risk Factors' section of the Company's Annual Report on Form 10-K for the fiscal year ended October 31, 2024 (copies of which may be obtained at or ). You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene's assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. View source version on CONTACT: For media:[email protected] For investors:[email protected] KEYWORD: UNITED STATES NORTH AMERICA CANADA MASSACHUSETTS INDUSTRY KEYWORD: HEALTH GENETICS RESEARCH PHARMACEUTICAL SCIENCE BIOTECHNOLOGY SOURCE: enGene Holdings Inc. Copyright Business Wire 2025. PUB: 06/12/2025 08:00 AM/DISC: 06/12/2025 07:58 AM
