Calgary, Alberta and Amsterdam, Netherlands--(Newsfile Corp. - October 27, 2025) - Zenith Epigenetics Ltd. ("Zenith" or the "Company") is pleased to announce that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug designation to ZEN-3694 for treatment of NUT carcinoma. NUT carcinoma is a rare, aggressive form of cancer with no currently approved treatments and significant unmet medical need. The Company recently announced Fast Track designation for ZEN-3694 and has stated its intention to pursue Breakthrough Therapy designation.
"Orphan Drug status underscores the unmet need for novel treatment options in this rare disease, where patients face poor prognoses and currently have no approved targeted therapies," said Donald McCaffrey, President & CEO of Zenith Epigenetics. "We believe ZEN-3694, through its epigenetic mechanism and combinatorial approach, has the potential to significantly improve outcomes and survival for people with NUT carcinoma. Orphan Drug designation, and the recently announced Fast Track designation, help us advance this program with the goal of making ZEN-3694 available to those who may benefit from it."
Orphan Drug Designation
The purpose of the Orphan Product program is to support the development and evaluation of new treatments for rare diseases. FDA grants Orphan Drug designation to investigational therapies addressing conditions that affect fewer than 200,000 people in the US. Incentives for sponsors of Orphan designated drugs include:
Granting of Orphan Drug status comes as representatives of the Company travel to attend the World Orphan Drug Congress ("WODC") 2025, in Amsterdam, the Netherlands, October 27-29. WODC is the largest and most established orphan drug & rare disease meeting with over 2000 professionals in the rare disease community attending, including pharmaceutical and biotechnology executives, as well as patient advocates.
NUT Carcinoma
NUT carcinoma is a highly aggressive type of cancer affecting adults and children and typically occurs in the midline area of the body including head, neck, and thoracic areas. While NUT carcinoma is a rare cancer, it is currently underdiagnosed – due to lack of awareness and testing – and the actual incidence is estimated at 10,000 cases per year in G8 countries. However, growing awareness among clinicians, as well as new biomarker testing methods, are improving detection. With rapidly increasing diagnoses, no approved therapies, and a median overall survival of about 6 months, there is a significant unmet need for new therapeutic options in NUT carcinoma.
ZEN-3694
Our lead compound, ZEN-3694, is a potent and selective BET bromodomain inhibitor (BETi). Taken orally once daily, it is currently in clinical development for multiple oncology indications, in combination with approved targeted therapies. Over 550 patients to date have been dosed with ZEN-3694, yielding robust evidence of on-target safety – enabling chronic dosing, combinability with other targeted drugs, as well as clinical efficacy. We believe ZEN-3694 is well differentiated as compared to other BET inhibitors and places us at the forefront of development of BETi combination therapies for the treatment of cancers in patient populations with a high unmet need.
NUT Carcinoma Clinical Program
ZEN-3694, is currently being evaluated in two (2) active NUT carcinoma clinical trials in combination with abemaciclib (ClinicalTrials.gov ID: NCT05372640) and cisplatin & etoposide (ClinicalTrials.gov ID: NCT05019716). In NUT carcinoma, the NUTM1 gene is fused with a transcriptional regulator – most commonly a BET protein – and drives expression of cancer-causing genes, leading to unchecked growth of tumors. Through disrupting the activity of NUT fusion protein, ZEN-3694 has demonstrated both single-agent and combination efficacy in treating NUT carcinoma. To date, the combination of abemaciclib plus ZEN-3694 has shown superior response rate and duration of response compared to single agent BET inhibitors by preventing therapy resistance.
Metastatic Castration Resistant Prostate Cancer Clinical Program
Zenith and our partner Newsoara initiated a 200 patient Phase 2b randomized trial to evaluate the efficacy and tolerability of the combination of ZEN-3694 + enzalutamide vs. single agent enzalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC). The trial dosed its first patient in December 2021, and to date, approximately 178 patients have been enrolled into the study. If the trial's endpoint is met, we intend to advance the program to a randomized, Phase 3, registration-enabling study.
About Zenith
Zenith Epigenetics Ltd., a wholly owned subsidiary of Zenith Capital Corp., is a clinical stage biotechnology company focused on the discovery and development of novel therapeutics for the treatment of cancer and other disorders with significant unmet medical need. Zenith Epigenetics is developing various novel combinations of BET inhibitors with other targeted agents. Our lead compound, ZEN-3694, is in clinical development for various oncologic indications such as metastatic castration resistant prostate cancer, NUT carcinoma, ovarian cancer, colorectal cancer, breast cancer, squamous cell lung cancer and other solid tumors. Several of these studies are sponsored by the National Cancer Institute ("NCI") under the NCI-Zenith Cooperative Research & Development Agreements ("CRADA") and CRADAs between NCI and other NCI collaborators.
Forward-Looking Statement
This news release may contain certain forward-looking information as defined under applicable Canadian securities legislation, that are not based on historical fact, including without limitation statements containing the words "believes", "anticipates", "plans", "intends", "will", "should", "expects", "continue", "estimate", "forecasts" and other similar expressions. In particular, this news release includes forward-looking information relating to the Company's development activities involving ZEN-3694 in NUT carcinoma, prostate cancer, ovarian cancer, lung cancer, breast cancer, colorectal cancer, and other tumor types, as a single agent, or in combination with chemotherapies, as well as our partnerships, agreements, and collaborations in furtherance of these development activities. Our actual results, events or developments could be materially different from those expressed or implied by these forward-looking statements. We can give no assurance that any of the events or expectations will occur or be realized. By their nature, forward-looking statements are subject to numerous assumptions and risk factors including those discussed in our most recent MD&A which are incorporated herein by reference and are available through SEDAR+ at www.sedarplus.ca. The forward-looking statements contained in this news release are expressly qualified by this cautionary statement and are made as of the date hereof. Zenith disclaims any intention and has no obligation or responsibility, except as required by law, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
For further information, please contact:
Investor Relations
Phone: 587-390-7865
Email: info@zenithepigenetics.com
Website: www.zenithepigenetics.com
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/271991
"Orphan Drug status underscores the unmet need for novel treatment options in this rare disease, where patients face poor prognoses and currently have no approved targeted therapies," said Donald McCaffrey, President & CEO of Zenith Epigenetics. "We believe ZEN-3694, through its epigenetic mechanism and combinatorial approach, has the potential to significantly improve outcomes and survival for people with NUT carcinoma. Orphan Drug designation, and the recently announced Fast Track designation, help us advance this program with the goal of making ZEN-3694 available to those who may benefit from it."
Orphan Drug Designation
The purpose of the Orphan Product program is to support the development and evaluation of new treatments for rare diseases. FDA grants Orphan Drug designation to investigational therapies addressing conditions that affect fewer than 200,000 people in the US. Incentives for sponsors of Orphan designated drugs include:
- Potential seven years of market exclusivity after approval
- Tax credits for qualified clinical trials
- Exemption from user fees (paid by the company when submitting drug applications for review)
Granting of Orphan Drug status comes as representatives of the Company travel to attend the World Orphan Drug Congress ("WODC") 2025, in Amsterdam, the Netherlands, October 27-29. WODC is the largest and most established orphan drug & rare disease meeting with over 2000 professionals in the rare disease community attending, including pharmaceutical and biotechnology executives, as well as patient advocates.
NUT Carcinoma
NUT carcinoma is a highly aggressive type of cancer affecting adults and children and typically occurs in the midline area of the body including head, neck, and thoracic areas. While NUT carcinoma is a rare cancer, it is currently underdiagnosed – due to lack of awareness and testing – and the actual incidence is estimated at 10,000 cases per year in G8 countries. However, growing awareness among clinicians, as well as new biomarker testing methods, are improving detection. With rapidly increasing diagnoses, no approved therapies, and a median overall survival of about 6 months, there is a significant unmet need for new therapeutic options in NUT carcinoma.
ZEN-3694
Our lead compound, ZEN-3694, is a potent and selective BET bromodomain inhibitor (BETi). Taken orally once daily, it is currently in clinical development for multiple oncology indications, in combination with approved targeted therapies. Over 550 patients to date have been dosed with ZEN-3694, yielding robust evidence of on-target safety – enabling chronic dosing, combinability with other targeted drugs, as well as clinical efficacy. We believe ZEN-3694 is well differentiated as compared to other BET inhibitors and places us at the forefront of development of BETi combination therapies for the treatment of cancers in patient populations with a high unmet need.
NUT Carcinoma Clinical Program
ZEN-3694, is currently being evaluated in two (2) active NUT carcinoma clinical trials in combination with abemaciclib (ClinicalTrials.gov ID: NCT05372640) and cisplatin & etoposide (ClinicalTrials.gov ID: NCT05019716). In NUT carcinoma, the NUTM1 gene is fused with a transcriptional regulator – most commonly a BET protein – and drives expression of cancer-causing genes, leading to unchecked growth of tumors. Through disrupting the activity of NUT fusion protein, ZEN-3694 has demonstrated both single-agent and combination efficacy in treating NUT carcinoma. To date, the combination of abemaciclib plus ZEN-3694 has shown superior response rate and duration of response compared to single agent BET inhibitors by preventing therapy resistance.
Metastatic Castration Resistant Prostate Cancer Clinical Program
Zenith and our partner Newsoara initiated a 200 patient Phase 2b randomized trial to evaluate the efficacy and tolerability of the combination of ZEN-3694 + enzalutamide vs. single agent enzalutamide in patients with metastatic castration-resistant prostate cancer (mCRPC). The trial dosed its first patient in December 2021, and to date, approximately 178 patients have been enrolled into the study. If the trial's endpoint is met, we intend to advance the program to a randomized, Phase 3, registration-enabling study.
About Zenith
Zenith Epigenetics Ltd., a wholly owned subsidiary of Zenith Capital Corp., is a clinical stage biotechnology company focused on the discovery and development of novel therapeutics for the treatment of cancer and other disorders with significant unmet medical need. Zenith Epigenetics is developing various novel combinations of BET inhibitors with other targeted agents. Our lead compound, ZEN-3694, is in clinical development for various oncologic indications such as metastatic castration resistant prostate cancer, NUT carcinoma, ovarian cancer, colorectal cancer, breast cancer, squamous cell lung cancer and other solid tumors. Several of these studies are sponsored by the National Cancer Institute ("NCI") under the NCI-Zenith Cooperative Research & Development Agreements ("CRADA") and CRADAs between NCI and other NCI collaborators.
Forward-Looking Statement
This news release may contain certain forward-looking information as defined under applicable Canadian securities legislation, that are not based on historical fact, including without limitation statements containing the words "believes", "anticipates", "plans", "intends", "will", "should", "expects", "continue", "estimate", "forecasts" and other similar expressions. In particular, this news release includes forward-looking information relating to the Company's development activities involving ZEN-3694 in NUT carcinoma, prostate cancer, ovarian cancer, lung cancer, breast cancer, colorectal cancer, and other tumor types, as a single agent, or in combination with chemotherapies, as well as our partnerships, agreements, and collaborations in furtherance of these development activities. Our actual results, events or developments could be materially different from those expressed or implied by these forward-looking statements. We can give no assurance that any of the events or expectations will occur or be realized. By their nature, forward-looking statements are subject to numerous assumptions and risk factors including those discussed in our most recent MD&A which are incorporated herein by reference and are available through SEDAR+ at www.sedarplus.ca. The forward-looking statements contained in this news release are expressly qualified by this cautionary statement and are made as of the date hereof. Zenith disclaims any intention and has no obligation or responsibility, except as required by law, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
For further information, please contact:
Investor Relations
Phone: 587-390-7865
Email: info@zenithepigenetics.com
Website: www.zenithepigenetics.com
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/271991