On September 3, 2025 enGene Holdings Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral gene therapy company, reported it has achieved its target enrollment milestone of 100 patients for the pivotal cohort of its ongoing, open-label, multi-cohort Phase 2 LEGEND trial of detalimogene voraplasmid ("detalimogene" and previously EG-70) in patients with high-risk, non-muscle invasive bladder cancer (NMIBC) (Press release, enGene, SEP 3, 2025, View Source [SID1234655749]). LEGEND’s pivotal Cohort 1 is studying detalimogene in patients with high-risk NMIBC with carcinoma in-situ (CIS) with or without concomitant papillary disease. Patients in the screening process remain eligible for potential enrollment in the trial. The Company expects to overenroll in its pivotal cohort resulting in an adjustment in guidance for a BLA submission to 2H 2026.

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"Achieving our target enrollment goal for detalimogene in LEGEND’s pivotal cohort represents an important milestone for enGene. It brings us a step closer to our goal of providing patients and physicians with the first non-viral gene therapy that offers a unique balance of efficacy, safety, and ease-of-use," said Ron Cooper, Chief Executive Officer of enGene. "We are grateful to study participants, investigators, and our clinical organization for their contributions to advancing the development of detalimogene."

About Detalimogene Voraplasmid

Detalimogene is a novel, investigational, non-viral gene therapy 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, reduce complexities related to safe handling and cold storage, and streamline both manufacturing processes and administration paradigms.

Detalimogene has received Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations 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. The RMAT program is intended to expedite the development and review of regenerative medicine therapies for serious or life-threatening conditions, where preliminary clinical evidence suggests potential to address unmet medical needs. Similarly, 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 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.

On September 3, 2025 Beyond Air, Inc. (NASDAQ: XAIR) ("Beyond Air" or the "Company"), a commercial stage medical device and biopharmaceutical company focused on harnessing the power of nitric oxide (NO) to improve the lives of patients, reported that Steve Lisi, Chairman and Chief Executive Officer of Beyond Air, will participate in the H.C. Wainwright 27th Annual Global Investment Conference being held September 8-10, 2025 in New York, NY (Press release, Beyond Air, SEP 3, 2025, View Source [SID1234655736]).

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An on-demand webcast of the Company’s presentation will be available beginning at 7:00 a.m. ET on Friday, September 5, 2025, on the H.C. Wainwright Conference platform.

In addition, members of management will be available for meetings with investors at the conference. If you are interested in requesting a one-on-one meeting at the conference, please contact your respective representative at H.C. Wainwright to schedule accordingly.

On September 3, 2025 Immunome, Inc. (Nasdaq: IMNM), a biotechnology company focused on developing first-in-class and best-in-class targeted cancer therapies, reported that Immunome management will present at the H.C. Wainwright 27th Annual Global Investment Conference on Tuesday, Sept. 9, 2025 at 1:00 p.m. Eastern Time (Press release, Immunome, SEP 3, 2025, View Source [SID1234655750]).

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Interested parties can access the live audio webcast for this conference from the Investor Relations section of the company’s website at www.immunome.com. The webcast replay will be available after the conclusion of the live presentation for approximately 30 days.

On September 3, 2025 Boehringer Ingelheim reported HERNEXEOS (zongertinib tablets) has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for first-line treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations (Press release, Boehringer Ingelheim, SEP 3, 2025, View Source [SID1234655737]).

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"Exploring accelerated review pathways is part of Boehringer’s strategy to have an unprecedented and generational impact on people facing cancer. We are incredibly pleased that HERNEXEOS has received Breakthrough Therapy Designation for first-line use in patients living with HER2-mutant non-small cell lung cancer," said Vicky Brown, Senior Vice President and Head of Immunology, Oncology, and Eye Health, Boehringer Ingelheim. "This pathway was designed to expedite the development and review of promising medicines for serious diseases, and clearly highlights the potential of HERNEXEOS."

Breakthrough Therapy Designation is given to expedite the development and review of medicines for serious conditions where preliminary clinical evidence indicates that the medicine may demonstrate substantial improvement over currently available therapies.

HERNEXEOS was recently approved by the U.S. FDA for adult patients with unresectable or metastatic non-squamous NSCLC whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy. This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Boehringer will present new and updated HERNEXEOS data at the upcoming World Conference on Lung Cancer (WCLC) in September and European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) in October.

About HERNEXEOS
HERNEXEOS is an irreversible tyrosine kinase inhibitor (TKI) that selectively inhibits HER2 while sparing wild-type EGFR, thereby limiting associated toxicities. Boehringer Ingelheim is investigating HERNEXEOS across a range of advanced solid tumors with HER2 alterations.

The therapy was conditionally approved for use as a monotherapy by China’s National Medical Products Administration (NMPA) for the treatment of adult patients with unresectable, locally advanced or metastatic NSCLC whose tumors have activating HER2 (ERBB2) mutations and who have received at least one line of prior systemic therapy. It also recently received Breakthrough Therapy Designation from the Center for Drug Evaluation (CDE) of China’s NMPA for the first line treatment of adult patients with unresectable or metastatic NSCLC harboring activating mutations in the HER2 TKD. This orally administered, targeted therapy has also been granted Orphan Drug Designation in Japan.

About non-small cell lung cancer (NSCLC)
Lung cancer claims more lives than any other cancer type and the incidence is set to increase to over 3 million cases worldwide by 2040.1,2 NSCLC is the most common type of lung cancer.3 Due to a lack of symptoms and misdiagnoses,4 most patients with NSCLC present at stage III or IV, where the disease has metastasized locally or to other organs.5 The estimated 5-year survival rate historically has been less than 10% for metastatic disease.6,7,8 People living with advanced NSCLC can experience a detrimental physical, psychological, and emotional impact on their daily lives.9,10,11

About the Beamion clinical trial program
Beamion LUNG-1 (NCT04886804): An open-label, Phase I dose escalation trial, with dose confirmation and expansion, of zongertinib as monotherapy in people with unresectable or metastatic solid tumors with HER2 alterations. The study has 2 parts. The first part is open to adults with different types of advanced cancer with HER2 alterations that include mutations, amplifications, over-expression and fusions for whom previous treatment was not successful. The second part is open to people with HER2-mutant non-small cell lung cancer.

Beamion LUNG-2 is a Phase III, open label, randomized, active-controlled study that will enroll 416 patients with unresectable, or metastatic non-squamous NSCLC harboring HER2 tyrosine kinase domain mutations to evaluate zongertinib compared with standard of care. 

On September 3, 2025 First Ascent Biomedical (FA), an innovative biotech company specializing in transforming cancer treatment through functional precision medicine, reported a new collaboration with the Fight Colorectal Cancer Foundation (Fight CRC) to launch a prospective feasibility study aimed at transforming colorectal cancer treatment (Press release, First Ascent Biomedical, SEP 3, 2025, https://firstascentbiomedical.com/first-ascent-biomedical-and-fight-colorectal-cancer-foundation-announce-groundbreaking-study-to-advance-precision-oncology-for-colorectal-cancer-patients/?utm_source=rss&utm_medium=rss&utm_campaign=first-ascent-biomedical-and-fight-colorectal-cancer-foundation-announce-groundbreaking-study-to-advance-precision-oncology-for-colorectal-cancer-patients [SID1234655876]). Funded by a $350,000 grant from Fight CRC, and spearheaded by Anjee Davis, chief executive officer, this innovative study will leverage First Ascent’s cutting-edge xDRIVE tumor profiling technology to deliver rapid, personalized treatment insights for patients fighting colon cancer in Minnesota.

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"Fight CRC is committed to empowering patients through innovation," said Davis. "This grant underscores our strategic focus on enhancing care through advanced research, particularly for those with limited treatment options."

The study, set to begin enrollment in April or May 2025, will focus on 25 patients with metastatic colorectal cancer. By integrating functional drug testing, genetic analysis, and artificial intelligence, First Ascent’s xDRIVE platform will provide oncologists with detailed tumor profiling data and tailored treatment options, aiming to enhance patient outcomes.

Leading the clinical efforts are Hao Xie, MD, PhD, a gastrointestinal cancer specialist and precision medicine expert, and Dr. Boardman, whose work in cancer genetics and early detection has advanced the field of colorectal cancer care. Noah Berlow, PhD, First Ascent’s Chief Technology Officer and the study’s principal investigator, brings his expertise in AI-driven cancer research to oversee the project. Together, this team is poised to demonstrate the power of rapid, individualized tumor profiling to better understand each patient’s unique cancer.

"This collaboration with the Fight Colorectal Cancer Foundation is another important step in transforming how we approach cancer treatment," said Jim Foote, founder and chief executive officer, First Ascent Biomedical. "We are honored to be part of this effort."

The study is expected to complete enrollment within nine months and will provide actionable data to oncologists for patients requiring advanced treatment options. Patients interested in participating must be seen in Rochester, MN. Information will soon be available on ClinicalTrials.gov.