On May 7, 2026 enGene Therapeutics Inc. (Nasdaq: ENGN or "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, reported additional interim results from the pivotal cohort of its ongoing, Phase 2 LEGEND trial of detalimogene voraplasmid (also known as detalimogene) in high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) patients with carcinoma in situ (CIS) with or without concomitant papillary disease, which enrolled 125 patients. These data, as of April 21, 2026, demonstrated that patients treated with detalimogene achieved a 54% complete response (CR) at any time and a 43% CR rate at six months. The progression rate to muscle-invasive or advanced disease was low, at 3.2%. Detalimogene was generally well tolerated with 55% of patients having experienced a treatment-related adverse event, mostly mild (Grade 1 and 2).

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"These updated data continue to reinforce the favorable safety and tolerability profile of detalimogene and its clinical activity in a heavily pretreated, high-risk NMIBC patient population with limited therapeutic options. Importantly, the low rate of progression to muscle-invasive disease leaves patients eligible for other bladder-sparing therapies," said Ron Cooper, President and Chief Executive Officer, enGene. "While durability outcomes to date are not what we hoped, these data are preliminary. We are focused on evaluating the totality of the data as it evolves and plan to continue to engage with the FDA and the medical community."

LEGEND Pivotal Cohort Data Update

The interim analysis is based on the 125 patients enrolled in Cohort 1. Patients who discontinued without any disease evaluation or who discontinued after a CR were considered not evaluable for subsequent landmark CR analysis.

Efficacy overview:

54% (95% CI: 45%, 63%) CR at any time (67/124)
91% of responses occurred at first disease assessment
43% (95% CI: 34%, 52%) CR rate at 6 months (52/121) with 14% (6/43) of patients having successfully converted from non-CR to CR post re-induction
Two re-induced patients have not yet had a 6-month assessment
Of the 52 responders at 6 months:
37/44 patients who had a 9-month assessment were in CR; an additional 8 patients are pending evaluation
13/22 patients who had a 12-month assessment were in CR; an additional 11 patients are pending evaluation
A maximum of 21 patients, including the 2 currently undergoing re-induction, still have the potential to achieve a CR at 12 months
The Kaplan-Meier (KM) estimate of the 12-month duration of response (DoR) is 25% (95% CI: 11%, 41%)
The KM estimate for median DoR is 37.3 weeks (range: 31.6-43.9 weeks)
Low rate of progression to muscle invasive or more advanced disease (3.2%)
Safety overview:

55% of patients experienced a treatment-related adverse event (TRAE)
Among patients with any TRAE, 91% experienced Grade 1-2 TRAEs
Grade ≥3 TRAEs were reported in 6 patients (4.8%)
Low percentage of patients experienced TRAEs leading to treatment interruption (2.4%) and treatment discontinuation (2.4%)
Among the 32 patients who had their first disease assessment after the last data analysis (October 24, 2025, reported November 11, 2025), CR rates were lower than previously reported results. The CR rate at any time was 39% and at 6 months was 32% for these patients. A preliminary subgroup analysis has not revealed any material differences in demographics or key disease characteristics. A more comprehensive analysis of these patients, including potential contributing factors, is ongoing.

As a beneficiary of FDA’s RMAT designation and a participant in the FDA’s CDRP program, enGene has completed the required FDA manufacturing validation batches and submitted a Statistical Analysis Plan (SAP) to the agency. The Company plans further engagement with the FDA as it approaches a potential Biologics License Application (BLA) filing and plans to provide an update in the second half of 2026.

The Company plans to share these data with the broader medical community at a Plenary Presentation at the upcoming American Urological Association meeting on May 15, 2026.

Safety Information

The overall tolerability profile associated with detalimogene is favorable. Of the 125 patients assessed for safety in Cohort 1, 69 patients (55%) experienced at least one TRAE, which were mainly Grade 1/2 in severity. The most common TRAEs were fatigue (22%), dysuria (14%), micturition urgency (12%), pollakiuria (12%), and bladder spasm (11%). Six patients experienced Grade ≥3 TRAEs, including one Grade 4 TRAE, which has resolved. There were no Grade 5 TRAEs reported. TRAEs leading to dose discontinuations (2.4%) and dose interruptions (2.4%) were rare.

Conference Call

enGene will host a conference call and live webcast at 8:00 a.m. ET today, May 7, 2026. Individuals interested in listening to the conference call may do so by clicking the following link, which is also available at the "Investors" section of the Company’s website at www.engene.com/investors. Following the live webcast, an archived version of the call will also be available on the Company’s website for 90 days.

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 the 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 present as papillary outgrowths from the bladder wall, which are typically resected, or as carcinoma in situ (CIS), which consists of flat, multifocal lesions that cannot be resected. The two forms can also 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 potentially subject to full removal of the bladder (cystectomy) as a curative but life-altering next step.

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. These designations are intended to expedite the development and review of drugs to serious or life-threatening conditions and fill an unmet medical need. Detalimogene has also been selected for the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program, designed to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy.

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 125 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).

(Press release, enGene, MAY 7, 2026, View Source [SID1234665361])

On May 7, 2026 AbCellera (Nasdaq: ABCL) reported that the Company will present at the following investor conferences:

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Bank of America Health Care Conference, May 13, 2026
Jefferies Global Healthcare Conference in New York, June 4, 2026

A live audio webcast of each presentation may be accessed through a link that will be posted on AbCellera’s Investor Relations website closer to date. A replay will be available through the same link following the presentation.

(Press release, AbCellera, MAY 7, 2026, View Source [SID1234665360])

On May 7, 2026 Repertoire Immune Medicines, a biotechnology company pioneering the discovery and development of programmable T cell-targeted immune medicines, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to RPTR-1-201 for the treatment of HLA-A*02:01-positive adult patients with unresectable or metastatic triple-negative breast cancer (TNBC) after progression on, or intolerance to, available standard therapies. RPTR-1-201 is a novel T cell receptor (TCR) bispecific immune medicine that is currently in a Phase 1/2 trial for the treatment of multiple advanced solid tumors.

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"Patients with advanced TNBC often face limited options after progression on standard therapies," said Robert Andtbacka, MD, CM, Chief Medical Officer of Repertoire Immune Medicines. "Fast Track Designation underscores the seriousness of this disease and the unmet needs for new treatment options. RPTR-1-201 is designed to redirect T cells by targeting a tumor-selective epitope on tumor cells in selected patients, and we appreciate the FDA’s engagement as we evaluate its potential in this population."

"RPTR-1-201 reflects our ability to discover tumor-selective epitopes and intentionally engineer TCR bispecifics designed to engage them with high affinity and specificity," said Anthony Coyle, PhD, President and Head of Research and Development at Repertoire Immune Medicines. "Derived from our DECODE platform, RPTR-1-201 couples an epitope-specific TCR with an anti-CD3 domain to engage and redirect T cells. Fast Track Designation supports closer dialogue with the FDA as we advance the program and continue its clinical evaluation."

The FDA’s Fast Track program is intended to facilitate the development and expedite the review of investigational therapies that treat serious conditions and address unmet medical needs. The designation may enable more frequent interactions with the FDA and may allow rolling review of a future marketing application if relevant criteria are met.

RPTR-1-201 is a TCR bispecific molecule comprised of an engineered TCR that binds with high affinity and precision to a tumor-selective epitope and an anti-CD3 moiety that engages and redirects T cells to kill tumor cells. RPTR-1-201 is derived from Repertoire’s DECODE platform, which maps the immune synapse to identify TCR-epitope pairs and translate these insights into T cell-targeted immune medicines.

RPTR-1-201 is currently being evaluated in a Phase 1/2 clinical trial as monotherapy and in combination with an anti-PD-1 therapy in participants with advanced solid tumors (ClinicalTrials.gov Identifier: NCT07293754). Repertoire plans to discuss development options in the Fast Track indication with the FDA as the program advances.

(Press release, Repertoire, MAY 7, 2026, View Source [SID1234665359])

On May 7, 2026 Photocure ASA (OSE: PHO) reported Hexvix/Cysview revenues of NOK 139.0 million in the first quarter of 2026 (Q1 2025: NOK 125.3 million), and an adjusted EBITDA of NOK 15.3 million (Q1 2025: NOK 9.7 million) for the company. In 2026, Photocure expects product revenue growth in the range of 7% to 11% on a constant currency basis and adjusted EBITDA margin expansion.

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"Photocure delivered a solid start to 2026, with strong growth across all territories and continued execution across both our commercial and strategic priorities. Revenue growth was robust in North America and Europe, reflecting accelerating adoption of blue-light cystoscopy and increasing procedural penetration in key markets," says Dan Schneider, President & Chief Executive Officer of Photocure.

The company continued to execute on its plan to expand blue-light cystoscopy (BLC) use in Q1 2026 with the installation of 11 new Saphira towers in the U.S. — 4 new accounts and 7 blue light tower upgrades. Photocure had 413 active accounts in the U.S. at the end of the quarter, an increase of 21% versus the first quarter of 2025. Across Europe, a total of 75 Olympus Visera Elite III BLC capable systems were installed since the launch in Q1 2025.

Total revenues ended at NOK 264.6 million in the first quarter of 2026, an increase from NOK 125.3 million in Q1 2025. The total revenue in Q1 2026 includes recognized milestone payments of NOK 125.6 million for the approval of Cevira in China and the acceptance of the marketing authorization approval request in Europe. Reported EBITDA was NOK 128.3 million (NOK 1.8 million). EBIT ended at NOK 120.7 million (NOK -5.6 million). Cash and cash equivalents were NOK 192.7 million at the end of the period.

"As a very important regulatory and strategic update, the U.S. Food and Drug Administration has provided clarity on the reclassification pathway for OAY-related equipment (Diagnostic Endoscopic Light Source Systems) following its response to the Karl Storz Citizen Petition and has confirmed plans to initiate a proposed reclassification process in the second half of 2026. This marks an important step towards a more structured and predictable regulatory framework for BLC equipment in the U.S. market. For Photocure, reclassification has the potential to be a step-change driver for the business, unlocking a significantly larger commercial opportunity, as we move towards double-digit penetration across the expanded market relative to where we are today," Schneider adds.

Photocure’s partners Richard Wolf and Asieris achieved a significant milestone with the April approval of the System blue BLC platform in China, which will be commercialized alongside Hexvix following its prior approval by the National Medical Products Administration (NMPA) in November 2024, enabling a fully integrated drug–device offering. At the same time, the recent CE mark and early commercial traction of blue light–compatible systems in Europe from the leading global medtech company Stryker, reinforce the growing recognition of BLC as an important standard in bladder cancer management and supporting broader adoption over time.

"Cevira, originally developed by Photocure and out-licensed to Asieris, was approved in China by the NMPA in March as a first-in-class non-invasive therapy for cervical precancerous lesions. Shortly thereafter, Cevira was endorsed with Level 1A evidence in expert consensus guidelines in China, reinforcing its clinical adoption potential. In Europe, the European Medicines Agency accepted the Marketing Authorization Application for Cevira during the quarter as well. The approval of Cevira in China and the EMA acceptance serve as milestones with contractual payments owed to Photocure in the amounts of 11.0 million and 2.0 million dollars respectively. The NMPA approval milestone is in dispute, with Asieris having paid 6.6 million of the 11.0 million dollars owed. Photocure believes its legal position to collect the full amount is strong and intends to engage in discussions with Asieris to explore potential pathways forward," Schneider says, and continues:

"Furthermore, in addition to our Hexvix/Cysview base business and partnered developments mentioned above, Photocure also remains committed to advancing a strategy of building an integrated diagnostics platform and leveraging our existing strong commercial footprints in North America and Europe. The uro-oncology landscape is rapidly evolving toward more personalized and data-driven care pathways, increasing the importance of multi-modal precision diagnostics tools. During the quarter, we made a targeted 3.0 million dollar minority investment in Vesica Health, a company within precision diagnostics, developing and launching a multi-omic urine biomarker test for early detection of bladder cancer with best-in-class performance. Our initiatives in flexible cystoscopy with Richard Wolf, AI-enabled software with Claritas/ISC, biomarkers with Vesica Health, and other innovations are progressing as planned, with the goal of improving early detection, diagnostic confidence, surveillance and treatment decision-making."

Photocure sees multiple drivers supporting continued growth in its base business, including sustained procedural adoption, expansion of installed equipment, increased utilization across existing accounts, and continued upgrade cycles to next-generation imaging systems. In addition, several strategic catalysts will further enhance its trajectory, including FDA reclassification of BLC to bring additional rigid equipment manufacturers to the U.S. and the reintroduction of flexible BLC solutions.

"We remain confident in Photocure’s momentum and continued positive trajectory. We expect strong underlying revenue growth across all regions, with product revenue growth of 7% to 11% on a constant currency basis, supported by sustained commercial execution. As operating leverage improves, we anticipate further expansion in adjusted EBITDA margin, reflecting the scalability of our platform and disciplined execution across the base business alongside a strategic platform extension. Our focus remains on delivering consistent execution and building long-term shareholder value," Schneider concludes.

(Press release, PhotoCure, MAY 7, 2026, View Source [SID1234665358])

On May 7, 2026 MEDSIR, an international company and a leader in oncology research, reported the results of the PHERGain and PHERGain-2 studies at the ESMO (Free ESMO Whitepaper) Breast 2026 scientific congress. Both studies evaluate de-escalation strategies based on targeted therapies in HER2-positive early breast cancer, with the aim of identifying which patients could safely avoid standard chemotherapy without compromising therapeutic benefit. Taken together, these studies reinforce a more personalized approach focused on reducing treatment-related toxicity while better preserving patients’ quality of life.

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PHERGain is an international, multicenter, phase II clinical trial in which 356 patients were enrolled across 45 hospitals in seven European countries, including Spain. Following publication of the primary results in leading scientific journals such as The Lancet, MEDSIR has now presented the five-year survival outcomes at ESMO (Free ESMO Whitepaper) Breast 2026. The objective of the study was to further validate a therapeutic de-escalation strategy in patients with HER2-positive early breast cancer, based on PET imaging and adapted to pathological complete response (pCR), allowing chemotherapy to be omitted without compromising disease control or survival.

The study demonstrated the efficacy of the combination of trastuzumab and pertuzumab within this adaptive strategy, showing that approximately 30% of patients with localized HER2-positive early breast cancer could avoid chemotherapy. The results presented at the congress confirm the robustness of this approach, with close to 90% of patients remaining recurrence-free five years after surgery. These findings support the validity of this adaptive therapeutic strategy as a safe and durable alternative over time, prioritizing patient well-being by avoiding conventional chemotherapy and its associated toxicities.

In addition, the company presented a translational analysis of the PHERGain study showing that circulating tumor DNA detected through liquid biopsy represents a precise, rapid, and non-invasive tool with the potential to identify patients who will remain disease-free in the long term.

As highlighted during the congress by Dr. Javier Cortés, principal investigator of the study and Director of the International Breast Cancer Center in Madrid and Barcelona: "In addition to the clinical outcomes, with nearly 90% of patients free from relapse five years after surgery, we see that the analysis of circulating tumor DNA in blood is emerging as a key tool to enable early identification of patients with a better prognosis and those who may benefit from more intensive treatment."

PHERGain-2: De-escalation with a focus on quality of life

PHERGain-2, presented at ESMO (Free ESMO Whitepaper) Breast 2026 and published simultaneously in Annals of Oncology, is an international, multicenter, phase II trial that enrolled 396 patients across 47 centers in Spain, Italy, Denmark, Hungary, Poland, and Bulgaria. The study aims to demonstrate the efficacy of a de-escalation strategy in HER2-positive early breast cancer in selected low-risk patients. Specifically, it focuses on patients with tumors measuring between 5 and 30 mm, with no nodal involvement at diagnosis and high HER2 protein expression.

The trial explores a chemotherapy-free, pCR-guided strategy based on the targeted therapies trastuzumab and pertuzumab, incorporating T-DM1 within the treatment regimen. In addition, quality of life is established as the primary safety objective, placing patient-reported outcomes at the center of treatment evaluation. The results presented suggest that more than half of the patients maintain a satisfactory quality of life throughout the first year of treatment, reinforcing the notion that avoiding chemotherapy contributes to a reduction in long-term adverse effects.

With regard to efficacy, the primary endpoint is still pending. Nevertheless, the observed pCR rate of 60% is encouraging, particularly considering that treatment is initiated without chemotherapy. These findings support the clinical potential of this strategy in this patient population.

As stated by Dr. Antonio Llombart-Cussac, principal investigator of the study and Head of the Medical Oncology Department at Hospital Arnau de Vilanova in Valencia: "The results of PHERGain-2 reinforce the idea that it is possible to offer patients a therapeutic alternative that prioritizes quality of life by avoiding side effects that negatively impact physical and emotional well-being, such as fatigue, hair loss, or other health problems associated with chemotherapy."

(Press release, MedSIR, MAY 7, 2026, View Source [SID1234665357])