Latest news with #BacillusCalmette-Guérin
Yahoo
28-05-2025
- Business
- Yahoo
enGene Names Amy Pott as Chief Global Commercialization Officer
BOSTON & MONTREAL, May 28, 2025--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN or "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, today announced the appointment of Amy Pott as Chief Global Commercialization Officer. In this role, Ms. Pott will serve as the Company's first dedicated executive for commercialization planning and execution, reporting to enGene's Chief Executive Officer, Ron Cooper. This appointment marks a significant milestone, as the Company expects to file a Biologics License Application (BLA) with the FDA in mid-2026 for detalimogene voraplasmid, its lead investigational agent in Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC), following the completion of the pivotal cohort of the LEGEND trial. "Amy brings a unique combination of P&L, full commercialization, gene therapy, deep analytics, and global experience to enGene," said enGene CEO, Ron Cooper. "We are thrilled to welcome Amy as our Chief Global Commercialization Officer and believe her leadership and track record of success will be a tremendous asset in preparing and executing the planned launch of detalimogene." Ms. Pott joins enGene from Astellas Pharma, where she most recently served as Senior Vice President (SVP), Strategic Brand Marketing, Ophthalmics and Rare Diseases, and previously as Head of Commercial, Gene Therapies. Prior to Astellas, she was President, North America for Swedish Orphan Biovitrum, and Global Vice President (GVP) U.S. Franchise Head for Internal Medicine and Oncology, as well as GVP, U.S. Commercial Operations at Shire. Before joining Shire, Ms. Pott was Vice President, Strategy, Planning and Analytics at Baxalta, Inc. Ms. Pott holds a Master of Science in European Studies from the London School of Economics and a Bachelor of Arts in History from the University of Bristol. "I am excited to join enGene at such a pivotal moment in its journey," said Ms. Pott. "I look forward to working with this talented team to advance the mission to help transform the treatment landscape for bladder cancer patients with our innovative genetic medicine." About Detalimogene Detalimogene is a novel, investigational, non-viral genetic medicine for patients with high-risk, non-muscle invasive bladder cancer (NMIBC), including Bacillus Calmette-Guérin (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 carcinoma in situ (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 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 voraplasmid (also known as detalimogene, and previously EG-70) 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. To learn more, please visit and follow us on LinkedIn, X and BlueSky. 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 and our expectations as to the timing and anticipated results of the LEGEND study, the future growth of enGene, the potential benefits of detalimogene, and the potential benefits of medicines developed with the DDX platform. 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 Contacts For media contact: media@ For investor contact: investors@ Error while retrieving data Sign in to access your portfolio Error while retrieving data
Business Wire
28-05-2025
- Business
- Business Wire
enGene Names Amy Pott as Chief Global Commercialization Officer
BOSTON & MONTREAL--(BUSINESS WIRE)--enGene Holdings Inc. (Nasdaq: ENGN or 'enGene' or the 'Company'), a clinical-stage, non-viral genetic medicines company, today announced the appointment of Amy Pott as Chief Global Commercialization Officer. In this role, Ms. Pott will serve as the Company's first dedicated executive for commercialization planning and execution, reporting to enGene's Chief Executive Officer, Ron Cooper. This appointment marks a significant milestone, as the Company expects to file a Biologics License Application (BLA) with the FDA in mid-2026 for detalimogene voraplasmid, its lead investigational agent in Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC), following the completion of the pivotal cohort of the LEGEND trial. 'Amy brings a unique combination of P&L, full commercialization, gene therapy, deep analytics, and global experience to enGene,' said enGene CEO, Ron Cooper. 'We are thrilled to welcome Amy as our Chief Global Commercialization Officer and believe her leadership and track record of success will be a tremendous asset in preparing and executing the planned launch of detalimogene.' Ms. Pott joins enGene from Astellas Pharma, where she most recently served as Senior Vice President (SVP), Strategic Brand Marketing, Ophthalmics and Rare Diseases, and previously as Head of Commercial, Gene Therapies. Prior to Astellas, she was President, North America for Swedish Orphan Biovitrum, and Global Vice President (GVP) U.S. Franchise Head for Internal Medicine and Oncology, as well as GVP, U.S. Commercial Operations at Shire. Before joining Shire, Ms. Pott was Vice President, Strategy, Planning and Analytics at Baxalta, Inc. Ms. Pott holds a Master of Science in European Studies from the London School of Economics and a Bachelor of Arts in History from the University of Bristol. 'I am excited to join enGene at such a pivotal moment in its journey,' said Ms. Pott. 'I look forward to working with this talented team to advance the mission to help transform the treatment landscape for bladder cancer patients with our innovative genetic medicine.' About Detalimogene Detalimogene is a novel, investigational, non-viral genetic medicine for patients with high-risk, non-muscle invasive bladder cancer (NMIBC), including Bacillus Calmette-Guérin (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 carcinoma in situ (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 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 voraplasmid (also known as detalimogene, and previously EG-70) 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. To learn more, please visit and follow us on LinkedIn, X and BlueSky. 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 and our expectations as to the timing and anticipated results of the LEGEND study, the future growth of enGene, the potential benefits of detalimogene, and the potential benefits of medicines developed with the DDX platform. 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.
Associated Press
20-05-2025
- Business
- Associated Press
Theralase Provides Corporate Update
Toronto, Ontario--(Newsfile Corp. - May 20, 2025) - Theralase® Technologies Inc. (TSXV: TLT) (OTCQB: TLTFF) ('Theralase®" or the 'Company'), a clinical stage pharmaceutical company dedicated to the research and development of light, radiation, sound and/or drug-activated small molecules and their formulations, intended for the safe and effective destruction of various cancers, bacteria and viruses, is pleased to provide a corporate update outlining the Company's strategic objectives. 1) Bacillus Calmette-Guérin ('BCG')-Unresponsive Non-Muscle Invasive Bladder Cancer ('NMIBC') Carcinoma In-Situ ('CIS') Registrational Clinical Study ('Study II') Theralase® has made steady progress on the completion of Study II by enrolling and providing the primary study procedure for 82 patients out of a target of 90 patients (91% enrollment). According to the clinical study design, a patient is considered to have completed Study II, if they receive the study procedure (study drug activated by study device) and have been assessed by the Principal Investigator ('PI') for up to 15 months or they have been prematurely removed from the clinical study by the PI for failure to respond or failure to comply with the clinical study design. According to this definition, 69 patients have completed Study II (with 13 patients on study with pending clinical data) resulting in the following interim clinical data in support of the Study II endpoints. [This table cannot be displayed. Please visit the source.] [This table cannot be displayed. Please visit the source.] [This table cannot be displayed. Please visit the source.] For 82 patients treated in Study II, there have been no Serious Adverse Events ('SAEs') directly related to the Study Drug or Study Device. Outside of the defined endpoints of Study II, Theralase® has demonstrated a duration of CR at extended time points, as follows: [This table cannot be displayed. Please visit the source.] Note: Not all patients have been assessed at these extended time points. As more clinical data is collected, the duration of CR at 2 and 3 years may increase. If approved by the regulatory authorities, the interim clinical data obtained could significantly benefit patients who are faced with a radical cystectomy (removal of their bladder), as the Theralase® treatment provides a strong initial CR and an equally strong duration of that CR over time. It is made even more impressive by the fact that this clinical data was achieved with only one study procedure in the majority of cases. Theralase® is on track to complete enrollment in Study II by the summer of 2025. This will allow the Company to report on 75 patients who have completed Study II in December 2025 and to report on all 90 patients by September 2026. Upon follow-up of all patients, the Company plans to submit a New Drug Application ('NDA') to Health Canada and the FDA in 4Q2026, with a decision expected by the respective regulatory authorities on a marketing approval by 1Q2027. As Theralase® completes enrollment in Study II, it is actively searching for commercialization partners for international marketing and sales of Ruvidar®. To this end, Theralase® is in various stages of initial and advanced discussions with international pharmaceutical companies for various geographical territories concerning: In recent discussions with the FDA, the Company has decided that since Study II is 91% complete, that the best course of action is not to pursue Break Through Designation, but to complete Study II and submit the clinical data to the FDA in a formal NDA. At the end of the meeting, the FDA made a comment that they were impressed that the interim clinical data obtained to date was able to be achieved with only one clinical treatment, in the majority of cases. Ruvidar® has demonstrated 10 years shelf life, strongly supporting the stability of the molecule and the ability of clinics to store the small molecule for extended periods of time. 2) Glio Blastoma Multiforme ('GBM') Brain Cancer Treatment Theralase® has successfully completed pre-clinical research to develop Ruvidar® for the destruction of GBM. The Company expects to complete Good Laboratory Practice ('GLP') toxicology in 4Q2025 to allow commencement of a Phase I/II adaptive clinical study in 1Q2026. As an example of the effectiveness of Rutherrin® (Intra Venous ('IV') formulation of Ruvidar®) activated by radiation in the destruction of brain cancer, refer to Figure 1. [ This image cannot be displayed. Please visit the source: ] Figure 1: Destruction of human glioma cells treated with radiation-activated Rutherrin® versus radiation alone To view an enhanced version of this graphic, please visit: 3) Non-Small Cell Lung Cancer ('NSCLC') Treatment Theralase® has finished conducting pre-clinical research to develop Ruvidar® for the destruction of NSCLC. Mice treated with x-ray activated Rutherrin® in a Lewis Lung Cancer ('LLC1") orthotopic model have demonstrated up to a 4-fold slower tumour progression, based on the Magnetic Resonance Imaging assessment of tumour volumes. [ This image cannot be displayed. Please visit the source: ] Figure 2: Tumour volume analysis in mice after tumour inoculation and treatment with either radiation alone or a combined treatment of Rutherrin ® and radiation treatment To view an enhanced version of this graphic, please visit: As shown in Figure 2, there is a significant delay in tumour progression in mice treated with x-ray activated Rutherrin® versus radiation alone (p> 0.001). Mice treated with x-ray activated Rutherrin® demonstrate that the tumour is regressing over time. The Company expects to complete GLP toxicology in 4Q2025 to allow commencement of a Phase I/II adaptive clinical study in 1Q2026. 4) Muscle Invasive Bladder Cancer ('MIBC') Treatment Theralase® is conducting pre-clinical research to develop Ruvidar® for the destruction of MIBC as an intravenous treatment for patients that are inflicted with this disease. The Company expects to complete GLP toxicology in 4Q2025 to allow commencement of a Phase I/II adaptive clinical study in 1Q2026. [ This image cannot be displayed. Please visit the source: ] Figure 3: Full depth tumour necrosis also occurs in muscle invasive bladder tumours, with no damage to healthy muscle tissue (Light-activated Ruvidar®) To view an enhanced version of this graphic, please visit: 5) Leukemia, Lymphoma and Myeloma Treatment Theralase® is conducting pre-clinical research to develop Ruvidar® for the destruction of Leukemia, Lymphoma and Myeloma as an extracorporeal treatment for patients that are inflicted with these diseases. Theralase® plans to develop this technology in 2026. [ This image cannot be displayed. Please visit the source: ] Figure 7. Evaluation of Leukemia in a mouse model To view an enhanced version of this graphic, please visit: 6) Herpes Simplex Virus ('HSV-1") Topical Treatment for Cold Sore Lesions Theralase® is conducting pre-clinical research to develop Ruvidar® for the inactivation of HSV-1 as a topical treatment for the billions of patients worldwide that are inflicted with cold sores on an annual basis. The Company is using its internal research laboratory to develop the topical formulation to be used in preclinical and clinical evaluation. The Company expects to complete GLP toxicology in 4Q2025 to allow commencement of a Phase I/II adaptive clinical study in 1Q2026. The research, which demonstrated that Ruvidar TM was effective at inactivating both enveloped and non-enveloped viruses, alone and when light activated was accepted in a peer-reviewed publication, Heliyon, and can be reviewed at An example of the efficacy of Ruvidar® versus standard of care therapies (i.e.: Acyclovir or Abreva) is demonstrated below: [ This image cannot be displayed. Please visit the source: ] Figure 4. Acyclovir (5%) (5 days of treatment x 5 times per day) To view an enhanced version of this graphic, please visit: [ This image cannot be displayed. Please visit the source: ] Figure 5. Abreva (10% Docosanol) (6 days of treatment x 5 times per day) To view an enhanced version of this graphic, please visit: [ This image cannot be displayed. Please visit the source: ] Figure 6. Ruvidar TM (1%) (5 days of treatment x once per day) To view an enhanced version of this graphic, please visit: 7) Cross Listing to a US Exchange The Company has raised $CAN 6.3 million over the last 2 years in support of it research and development programs. It is currently investigating the use of a full-service investment bank in the United States to advise on potential financings and US listing opportunities. Information on any future financings will be released once available in accordance with applicable securities laws. 2025/2026: Potentially Transformative Years Theralase® is positioning itself to deliver on a series of significant clinical and corporate milestones in 2025; including: These efforts should collectively support long-term shareholder value and reinforce Theralase®'s leadership in light / radiation-activated drug therapy for the destruction of various cancers and viruses. About Study II: Study II utilizes the therapeutic dose of the patented study drug (Ruvidar®) (0.70 mg/cm 2 ) activated by the proprietary study device (TLC-3200 Medical Laser System). Study II is focused on enrolling and treating approximately 90 BCG-Unresponsive NMIBC CIS patients in 12 clinical study sites located in Canada and the United States. About Ruvidar®: Ruvidar®(TLD-1433) is a small molecule, able to be activated by light, radiation, sound and/or other drugs, intended for the safe and effective destruction of various cancers, bacteria and viruses. About Theralase® Technologies Inc.: Theralase® is a clinical stage pharmaceutical company dedicated to the research and development of light, radiation, sound and/or drug-activated small molecule compounds, their associated drug formulations and the light systems that activate them, with a primary objective of efficacy and a secondary objective of safety in the destruction of various cancers, bacteria and viruses. Additional information is available at and Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release. Forward Looking Statements: This news release contains Forward-Looking Statements ('FLS') within the meaning of applicable Canadian securities laws. Such statements include; but, are not limited to statements regarding the Company's proposed development plans with respect to small molecules and their drug formulations. FLS may be identified by the use of the words 'may, 'should', 'will', 'anticipates', 'believes', 'plans', 'expects', 'estimate', 'potential for' and similar expressions; including, statements related to the current expectations of the Company's management regarding future research, development and commercialization of the Company's small molecules; their drug formulations; preclinical research; clinical studies and regulatory approvals. These statements involve significant risks, uncertainties and assumptions; including, the ability of the Company to fund and secure the regulatory approvals to successfully complete various clinical studies in a timely fashion and implement its development plans. Other risks include: the ability of the Company to successfully commercialize its small molecule and drug formulations; the risk that access to sufficient capital to fund the Company's operations may not be available on terms that are commercially favorable to the Company or at all; the risk that the Company's small molecule and drug formulations may not be effective against the diseases tested in its clinical studies; the risk that the Company fails to comply with the terms of license agreements with third parties and as a result loses the right to use key intellectual property in its business; the Company's ability to protect its intellectual property; the timing and success of submission, acceptance and approval of regulatory filings. Many of these factors that will determine actual results are beyond the Company's ability to control or predict. Readers should not unduly rely on these FLS, which are not a guarantee of future performance. There can be no assurance that FLS will prove to be accurate as such FLS involve known and unknown risks, uncertainties and other factors which may cause actual results or future events to differ materially from the FLS are made as of the date hereof and are subject to change. Except as required by law, the Company assumes no obligation to update such FLS. For investor information on the Company, please feel to reach outInvestor Inquiries - Theralase Technologies. For More Information: (843-5273) (5273) Kristina Hachey, CPA Chief Financial Officer X 224 [email protected] To view the source version of this press release, please visit
Yahoo
20-05-2025
- Business
- Yahoo
Theralase Provides Corporate Update
Toronto, Ontario--(Newsfile Corp. - May 20, 2025) - Theralase® Technologies Inc. (TSXV: TLT) (OTCQB: TLTFF) ("Theralase®" or the "Company"), a clinical stage pharmaceutical company dedicated to the research and development of light, radiation, sound and/or drug-activated small molecules and their formulations, intended for the safe and effective destruction of various cancers, bacteria and viruses, is pleased to provide a corporate update outlining the Company's strategic objectives. 1) Bacillus Calmette-Guérin ("BCG")-Unresponsive Non-Muscle Invasive Bladder Cancer ("NMIBC") Carcinoma In-Situ ("CIS") Registrational Clinical Study ("Study II") Theralase® has made steady progress on the completion of Study II by enrolling and providing the primary study procedure for 82 patients out of a target of 90 patients (91% enrollment). According to the clinical study design, a patient is considered to have completed Study II, if they receive the study procedure (study drug activated by study device) and have been assessed by the Principal Investigator ("PI") for up to 15 months or they have been prematurely removed from the clinical study by the PI for failure to respond or failure to comply with the clinical study design. According to this definition, 69 patients have completed Study II (with 13 patients on study with pending clinical data) resulting in the following interim clinical data in support of the Study II endpoints. Primary Endpoint Performance (CR at any Point in Time)# % Confidence Interval (95%) Complete Response ("CR") 43/69 62.3% [43.7, 80.9] Total Response (CR and IR) 48/69 69.6% [49.9, 89.2] Secondary Endpoint Performance (Duration of CR) (15 months)# % Confidence Interval (95%) Complete Response ("CR") 18/43 41.9% [22.5, 61.2] Tertiary Endpoint Performance (Safety) (15 months)# % Safety 69/69 100.0% For 82 patients treated in Study II, there have been no Serious Adverse Events ("SAEs") directly related to the Study Drug or Study Device. Outside of the defined endpoints of Study II, Theralase® has demonstrated a duration of CR at extended time points, as follows: Duration of CR Time # % Confidence Interval (95%) 2 Years 10/43 23.3% [8.8, 37.7] 3 Years 9/43 20.9% [7.3, 34.6] 7 Years 1/43 2.3% [0.0, 6.9] Note: Not all patients have been assessed at these extended time points. As more clinical data is collected, the duration of CR at 2 and 3 years may increase. If approved by the regulatory authorities, the interim clinical data obtained could significantly benefit patients who are faced with a radical cystectomy (removal of their bladder), as the Theralase® treatment provides a strong initial CR and an equally strong duration of that CR over time. It is made even more impressive by the fact that this clinical data was achieved with only one study procedure in the majority of cases. Theralase® is on track to complete enrollment in Study II by the summer of 2025. This will allow the Company to report on 75 patients who have completed Study II in December 2025 and to report on all 90 patients by September 2026. Upon follow-up of all patients, the Company plans to submit a New Drug Application ("NDA") to Health Canada and the FDA in 4Q2026, with a decision expected by the respective regulatory authorities on a marketing approval by 1Q2027. As Theralase® completes enrollment in Study II, it is actively searching for commercialization partners for international marketing and sales of Ruvidar®. To this end, Theralase® is in various stages of initial and advanced discussions with international pharmaceutical companies for various geographical territories concerning: Licensing of the light-activated Ruvidar® for BCG-Unresponsive NMIBC CIS Collaborative research focused on investigating light-activated Ruvidar® in the treatment of NMIBC Collaborative research focused on combining Ruvidar® with other FDA approved drugs In recent discussions with the FDA, the Company has decided that since Study II is 91% complete, that the best course of action is not to pursue Break Through Designation, but to complete Study II and submit the clinical data to the FDA in a formal NDA. At the end of the meeting, the FDA made a comment that they were impressed that the interim clinical data obtained to date was able to be achieved with only one clinical treatment, in the majority of cases. Ruvidar® has demonstrated 10 years shelf life, strongly supporting the stability of the molecule and the ability of clinics to store the small molecule for extended periods of time. 2) Glio Blastoma Multiforme ("GBM") Brain Cancer Treatment Theralase® has successfully completed pre-clinical research to develop Ruvidar® for the destruction of GBM. The Company expects to complete Good Laboratory Practice ("GLP") toxicology in 4Q2025 to allow commencement of a Phase I/II adaptive clinical study in 1Q2026. As an example of the effectiveness of Rutherrin® (Intra Venous ("IV") formulation of Ruvidar®) activated by radiation in the destruction of brain cancer, refer to Figure 1. Figure 1: Destruction of human glioma cells treated with radiation-activated Rutherrin® versus radiation alone To view an enhanced version of this graphic, please visit: 3) Non-Small Cell Lung Cancer ("NSCLC") Treatment Theralase® has finished conducting pre-clinical research to develop Ruvidar® for the destruction of NSCLC. Mice treated with x-ray activated Rutherrin® in a Lewis Lung Cancer ("LLC1") orthotopic model have demonstrated up to a 4-fold slower tumour progression, based on the Magnetic Resonance Imaging assessment of tumour volumes. Figure 2: Tumour volume analysis in mice after tumour inoculation and treatment with either radiation alone or a combined treatment of Rutherrin ® and radiation treatment To view an enhanced version of this graphic, please visit: As shown in Figure 2, there is a significant delay in tumour progression in mice treated with x-ray activated Rutherrin® versus radiation alone (p> 0.001). Mice treated with x-ray activated Rutherrin® demonstrate that the tumour is regressing over time. The Company expects to complete GLP toxicology in 4Q2025 to allow commencement of a Phase I/II adaptive clinical study in 1Q2026. 4) Muscle Invasive Bladder Cancer ("MIBC") Treatment Theralase® is conducting pre-clinical research to develop Ruvidar® for the destruction of MIBC as an intravenous treatment for patients that are inflicted with this disease. The Company expects to complete GLP toxicology in 4Q2025 to allow commencement of a Phase I/II adaptive clinical study in 1Q2026. Figure 3: Full depth tumour necrosis also occurs in muscle invasive bladder tumours, with no damage to healthy muscle tissue (Light-activated Ruvidar®) To view an enhanced version of this graphic, please visit: 5) Leukemia, Lymphoma and Myeloma Treatment Theralase® is conducting pre-clinical research to develop Ruvidar® for the destruction of Leukemia, Lymphoma and Myeloma as an extracorporeal treatment for patients that are inflicted with these diseases. Theralase® plans to develop this technology in 2026. Figure 7. Evaluation of Leukemia in a mouse model To view an enhanced version of this graphic, please visit: 6) Herpes Simplex Virus ("HSV-1") Topical Treatment for Cold Sore Lesions Theralase® is conducting pre-clinical research to develop Ruvidar® for the inactivation of HSV-1 as a topical treatment for the billions of patients worldwide that are inflicted with cold sores on an annual basis. The Company is using its internal research laboratory to develop the topical formulation to be used in preclinical and clinical evaluation. The Company expects to complete GLP toxicology in 4Q2025 to allow commencement of a Phase I/II adaptive clinical study in 1Q2026. The research, which demonstrated that RuvidarTM was effective at inactivating both enveloped and non-enveloped viruses, alone and when light activated was accepted in a peer-reviewed publication, Heliyon, and can be reviewed at An example of the efficacy of Ruvidar® versus standard of care therapies (i.e.: Acyclovir or Abreva) is demonstrated below: Figure 4. Acyclovir (5%) (5 days of treatment x 5 times per day) To view an enhanced version of this graphic, please visit: Figure 5. Abreva (10% Docosanol) (6 days of treatment x 5 times per day) To view an enhanced version of this graphic, please visit: Figure 6. RuvidarTM (1%) (5 days of treatment x once per day) To view an enhanced version of this graphic, please visit: 7) Cross Listing to a US Exchange The Company has raised $CAN 6.3 million over the last 2 years in support of it research and development programs. It is currently investigating the use of a full-service investment bank in the United States to advise on potential financings and US listing opportunities. Information on any future financings will be released once available in accordance with applicable securities laws. 2025/2026: Potentially Transformative YearsTheralase® is positioning itself to deliver on a series of significant clinical and corporate milestones in 2025; including: Completing Study II and submitting an NDA to Health Canada and the FDA Commencing Phase I/II adaptive clinical studies in: GBM, NSCLC, MIBC, HSV and Leukemia Evaluating strategic financing and listing opportunities in the US market These efforts should collectively support long-term shareholder value and reinforce Theralase®'s leadership in light / radiation-activated drug therapy for the destruction of various cancers and viruses. About Study II:Study II utilizes the therapeutic dose of the patented study drug (Ruvidar®) (0.70 mg/cm2) activated by the proprietary study device (TLC-3200 Medical Laser System). Study II is focused on enrolling and treating approximately 90 BCG-Unresponsive NMIBC CIS patients in 12 clinical study sites located in Canada and the United States. About Ruvidar®:Ruvidar®(TLD-1433) is a small molecule, able to be activated by light, radiation, sound and/or other drugs, intended for the safe and effective destruction of various cancers, bacteria and viruses. About Theralase® Technologies Inc.:Theralase® is a clinical stage pharmaceutical company dedicated to the research and development of light, radiation, sound and/or drug-activated small molecule compounds, their associated drug formulations and the light systems that activate them, with a primary objective of efficacy and a secondary objective of safety in the destruction of various cancers, bacteria and viruses. Additional information is available at and Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release. Forward Looking Statements:This news release contains Forward-Looking Statements ("FLS") within the meaning of applicable Canadian securities laws. Such statements include; but, are not limited to statements regarding the Company's proposed development plans with respect to small molecules and their drug formulations. FLS may be identified by the use of the words "may, "should", "will", "anticipates", "believes", "plans", "expects", "estimate", "potential for" and similar expressions; including, statements related to the current expectations of the Company's management regarding future research, development and commercialization of the Company's small molecules; their drug formulations; preclinical research; clinical studies and regulatory approvals. These statements involve significant risks, uncertainties and assumptions; including, the ability of the Company to fund and secure the regulatory approvals to successfully complete various clinical studies in a timely fashion and implement its development plans. Other risks include: the ability of the Company to successfully commercialize its small molecule and drug formulations; the risk that access to sufficient capital to fund the Company's operations may not be available on terms that are commercially favorable to the Company or at all; the risk that the Company's small molecule and drug formulations may not be effective against the diseases tested in its clinical studies; the risk that the Company fails to comply with the terms of license agreements with third parties and as a result loses the right to use key intellectual property in its business; the Company's ability to protect its intellectual property; the timing and success of submission, acceptance and approval of regulatory filings. Many of these factors that will determine actual results are beyond the Company's ability to control or predict. Readers should not unduly rely on these FLS, which are not a guarantee of future performance. There can be no assurance that FLS will prove to be accurate as such FLS involve known and unknown risks, uncertainties and other factors which may cause actual results or future events to differ materially from the FLS. Although the FLS contained in the press release are based upon what management currently believes to be reasonable assumptions, the Company cannot assure prospective investors that actual results, performance or achievements will be consistent with these FLS. All FLS are made as of the date hereof and are subject to change. Except as required by law, the Company assumes no obligation to update such FLS. For investor information on the Company, please feel to reach out Investor Inquiries - Theralase Technologies. For More Information: (843-5273) (5273) Kristina Hachey, CPAChief Financial Officer X 224khachey@ To view the source version of this press release, please visit
Yahoo
29-04-2025
- Health
- Yahoo
New data from J&J's bladder cancer drug-device trial strengthens support for NDA
Johnson & Johnson (J&J) has reported more data supporting the efficacy of its intravesical drug release system TAR-200 in patients with Bacillus Calmette-Guérin (BCG)-unresponsive, high-risk non-muscle-invasive bladder cancer (HR-NMIBC). Data, announced during the Paradigm-Shifting, Practice-Changing Clinical Trials in Urology plenary session at the American Urological Association (AUA) Annual Meeting 2025, showed a high response rate in one of the cohorts from the Phase IIb SunRISe-1 trial (NCT04640623). J&J announced new data from both cohort two and cohort four at the conference. TAR-200, trademarked as GemRIS, is a drug-device combination product that continuously delivers gemcitabine chemotherapy directly to the bladder over a week. It can be inserted without the need for anaesthesia or sedation. J&J acquired TAR-200 and the drug delivery technology during its purchase of Taris Biomedical in 2019. In the new data from cohort two, which enrolled 85 BCG-unresponsive NMIBC patients with carcinoma in situ, 82.4% achieved a complete response (CR), with this translating into sustained disease control, with 52.9% of responders maintaining CR at one year. In the same cohort, the median duration of response (DOR) was 25.8 months and at 12 months, 86.6% of patients who responded to treatment remained cystectomy-free. Meanwhile, patients in cohort four, 52 HR-NMIBC patients with papillary disease-only, 85.3% and 81.1% disease-free survival (DFS) rates at six and nine months. Also, 94.2% of patients avoided cystectomy at a median follow-up of 12.8 months. J&J also reported progression-free (PFS) and overall survival (OS) rates of 95.6% and 98% at nine months, respectively. In both cohorts, TAR-200, the treatment was well-tolerated, with most adverse events (AEs) being mild urinary symptoms. These findings indicate that TAR-200 offers a highly effective and durable treatment option for patients with certain types of BCG-unresponsive HR-NMIBC. Most treatment-related AEs were mild and manageable. Between both cohorts, seven patients discontinued treatment due to treatment-related AEs, and there were no treatment-related deaths. Disease area leader of bladder cancer for J&J Innovative Medicine, Dr. Christopher Cutie, said: 'Bladder cancer is one of the ten most common cancers worldwide, yet treatment options have remained largely unchanged for over 40 years, leaving patients with few choices if initial BCG therapy does not work. TAR-200 is designed to allow for sustained delivery of medication directly into the bladder through a brief and routine procedure, which benefits patients. These data now show patients can remain cancer-free for a meaningful period of time, marking a significant step forward for those facing this challenging disease.' The new data, announced on Saturday 26 April comes after J&J filed a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for TAR-200 in January 2025 based on previous data announced from the SunRISe-1 trial. The application is for the use of TAR-200 in NMIBC patients with carcinoma in situ who are BCG-unresponsive, with or without papillary tumours. This submission is being reviewed through the Real-Time Oncology Review (RTOR) programme, which allows the FDA to review data before the complete application is formally submitted. Earlier results from cohort two were presented at the European Society of Medical Oncology (ESMO) Congress 2024 and the AUA 2024 Meeting. TAR-200 did not, however, show benefit in a Phase III trial in patients with muscle-invasive bladder cancer (MIBC) not undergoing radical cystectomy. The Phase III SunRISe-2 study (NCT04658862) was terminated by J&J after an independent data monitoring committee said the trial was unlikely to meet its primary endpoint of bladder intact-event free survival (BI-EFS). The Phase II SunRISe-4 trial, also in patients with MIBC, is ongoing. The SunRISe-1 trial is ongoing, and TAR-200 is also being investigated in NMIBC in two Phase III studies, SunRISe-3 (NCT05714202) and SunRISe-5 (NCT06211764). Also at the AUA Annual Meeting 2025, Pfizer presented positive data from a Phase III trial of sasanlimab and BCG, with the combination therapy improving event-free survival (EFS) in BCG-naïve high-risk NMIBC patients compared to subjects who were only given BCG, meeting the trial's primary endpoint. "New data from J&J's bladder cancer drug-device trial strengthens support for NDA" was originally created and published by Medical Device Network, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
