On March 9, 2026 enGene Holdings Inc. (Nasdaq: ENGN, "enGene" or the "Company"), a clinical-stage, non-viral genetic medicines company, reported its financial results for the first quarter ended January 31, 2026, and provided a business update.

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"As data from LEGEND’s pivotal cohort in high-risk, BCG-unresponsive NMIBC continues to mature, we look forward to providing an update at a spring medical conference," said Ron Cooper, President and Chief Executive Officer, enGene. "On the heels of our successful November follow-on offering, we entered 2026 in a position of financial strength and ready to execute. With RMAT and CDRP designations for detalimogene, we are in active dialogue with the FDA to ensure regulatory and manufacturing readiness as we plan for a BLA submission in the second half of this year and potential launch in 2027."

Recent Corporate Updates

Expanded debt facility: In January 2026, the Company expanded its existing loan and security agreement with Hercules Capital, Inc. (NYSE: HTGC) ("Hercules"), for up to $125 million. Access to the additional non-dilutive capital strengthens enGene’s balance sheet in preparation for its planned Biologics License Application ("BLA") to the U.S. Food and Drug Administration ("FDA") for detalimogene voraplasmid as a treatment for high-risk, Bacillus Calmette-Guérin ("BCG")-unresponsive non-muscle invasive bladder cancer ("NMIBC") with carcinoma in situ ("CIS") in the second half of 2026, and the potential commercial launch of detalimogene should it receive FDA approval.

Successful completion of public offering: In November 2025, the Company successfully closed an underwritten public offering of its common shares and pre-funded warrants at an offering price of $8.50 per share and $8.4999 per pre-funded warrant. The offering totaled $140.1 million in net proceeds, following the full exercise of the underwriters’ option to purchase additional shares.

LEGEND pivotal cohort update: In November 2025, enGene reported preliminary data from LEGEND’s pivotal cohort for patients enrolled following a protocol amendment implemented in the fourth quarter of 2024. The preliminary analysis of the post-amendment patients with at least one post-baseline disease assessment included 62 patients at 3 months and 37 patients at 6 months and demonstrated:

63% complete response (CR) rate at any time (n=62);
56% CR rate at 3 months (n=62);
62% CR rate at 6 months (n=37), including 4 patients who successfully converted to CR post reinduction; and
All 5 patients who completed the 9-month assessment had a CR.
The Company completed enrollment of 125 patients in total. Data from these patients demonstrated a generally favorable tolerability profile:

42% of patients experienced a treatment-related adverse event (TRAE);
1.6% of patients experienced dose interruptions due to TRAEs; and
0.8% of patients experienced dose discontinuations due to TRAEs.
enGene continues to believe that detalimogene’s emerging profile supports its potential first-line use in patients with high-risk, BCG-unresponsive non-muscle invasive bladder cancer with CIS.

CMC Development and Readiness Pilot (CDRP) Program: In November 2025, the Company was selected as one of nine companies to participate in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program. The FDA created the CDRP Program to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy. Acceptance into the program further supports enGene’s confidence in detalimogene’s potential and aligns with enGene’s commitment to strong manufacturing practices as clinical development advances.

Anticipated Milestones

Update from LEGEND pivotal cohort planned for a spring 2026 medical conference.
12-month complete response data from LEGEND pivotal cohort expected in 2H 2026.
Planned BLA filing for LEGEND’s pivotal cohort in the second half of 2026.
First Quarter 2026 Financial Results

As of January 31, 2026, cash, cash equivalents and marketable securities were $312.5 million. The Company expects that its cash, cash equivalents and marketable securities will fund operating expenses, debt obligations and capital expenditures into the second half of 2028.

Total operating expenses were $31.2 million for the three months ended January 31, 2026, compared to $26.6 million for 2025. Research and development expenses increased by $2.3 million, primarily driven by increased personnel and clinical costs related to our LEGEND trial and in preparation for our planned Biologics License Application submission in the second half of 2026. General and administrative expenses increased by $2.3 million, primarily driven by personnel and facilities costs as the Company scales its general and administrative function to support the operation of a public company.

For the three months ended January 31, 2026, net loss attributable to common shareholders was approximately $29.8 million, or $0.44 per share, compared to approximately $24.6 million, or $0.48 per share, for the three months ended January 31, 2025. The increase in net loss is mainly attributed to the increase in operating expenses, partially offset by net interest income earned during the period.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

Non-muscle invasive bladder cancer (NMIBC) is a disease that poses a significant burden on both patients and clinics and has a massive economic impact on our healthcare system. NMIBC occurs when cancer cells grow in the tissues that line the interior of the bladder, but the cancer has not yet penetrated the muscle of the bladder wall. NMIBC can 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. 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. Detalimogene has also been selected to participate in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) program. The FDA created the CDRP Program 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). The LEGEND trial is actively enrolling patients with sites participating in the USA, Canada, Europe, and the Asia-Pacific region.

(Press release, enGene, MAR 9, 2026, View Source [SID1234663384])

On March 9, 2026 Lyell Immunopharma, Inc. (Nasdaq: LYEL), a late-stage clinical company advancing a pipeline of next-generation chimeric antigen receptor (CAR) T-cell therapies for patients with cancer, reported it has closed the sale of an additional $50 million of shares of its common stock to investors from its July 2025 equity private placement of up to $100 million, following achievement of a clinical milestone within its PiNACLE pivotal trial evaluating rondecabtagene autoleucel (ronde-cel) in patients with relapsed/refractory large B-cell lymphoma (LBCL) in the third- or later-line setting. The Company also announced the appointment of Smital Shah as its Chief Financial and Business Officer, effective March 9, 2026.

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"Smital’s deep expertise in navigating complex financial landscapes and her proven track record of strategic leadership in the biotechnology industry make her the ideal partner to guide Lyell’s financial strategy as we enter a transformative year of execution," said Lynn Seely, M.D., President and CEO of Lyell. "The attainment of the second $50 million tranche of the private placement is evidence of Lyell’s continued advancement toward significant value inflection points. Smital’s insights will be invaluable in ensuring we remain well-capitalized and operationally disciplined to deliver on our mission of bringing next-generation cell therapies to patients with cancer."

Closing of Additional $50 Million Equity Private Placement Following Achievement of Clinical Milestone for Ronde-cel
Under the terms of the securities purchase agreement for the July 2025 equity private placement of up to $100 million, and following the initial closing of $50 million, Lyell held the right to require investors to purchase an additional $50 million of common stock upon the achievement of a clinical milestone for ronde-cel, a dual-targeting CD19/CD20 CAR T-cell therapy in pivotal clinical development for patients with LBCL. Following the successful achievement of this clinical milestone, Lyell exercised this right and the investors purchased these shares from Lyell at a purchase price per share of $25.61, resulting in gross proceeds of approximately $50 million, bringing the total equity private placement proceeds to approximately $100 million. With the closing of this additional tranche, Lyell expects its cash, cash equivalents and marketable securities to be sufficient to meet working capital and capital expenditure needs into the second quarter of 2027.

The offer and sale of the foregoing securities were made in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended (the "Securities Act"), or any applicable state securities laws. Accordingly, the securities may not be reoffered or resold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements of the Securities Act and such applicable state securities laws. The investors have been granted customary resale Form S-3 registration rights for the shares of common stock issued to them in the financing.

This press release shall not constitute an offer to sell, or a solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.

Smital Shah Appointed Chief Financial and Business Officer
Ms. Shah joins Lyell with over two decades of extensive leadership experience spanning corporate finance, capital markets and strategic operations within the biopharmaceutical industry. Most recently, she operated as an independent Chief Financial Officer and Chief Business Officer consultant, providing strategic guidance to multiple life sciences organizations. From 2014 to 2022, Ms. Shah served as the Chief Business and Financial Officer at ProQR Therapeutics (Nasdaq: PRQR) where she was responsible for directing all business functions, including finance, communications, commercial strategy, business development and legal.

Prior to her tenure at ProQR, Ms. Shah managed multi-billion-dollar debt, cash and investment portfolios at Gilead Sciences, Inc. She developed financial expertise through her tenure as an investment banker at Leerink Partners and J.P. Morgan, where she focused on capital raising and complex strategic transactions across the biotechnology sector. She began her career in various research and development roles at Johnson & Johnson.

Ms. Shah served on the Board of Directors of Pliant Therapeutics until June 2025 and as a Board Member and Chair of the Audit Committee at Graphite Bio until its merger with LENZ Therapeutics in 2024. She holds a B.S. in Chemical Engineering from the University of Mumbai, an M.S. in Chemical Engineering from Virginia Tech, as well as an M.B.A. in Finance from the University of California, Berkeley, Haas School of Business.

(Press release, Lyell Immunopharma, MAR 9, 2026, View Source [SID1234663367])

On March 9, 2026 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported results from the Phase III persevERA Breast Cancer study evaluating investigational giredestrant in combination with palbociclib for people with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer. The study did not meet its primary objective of a statistically significant improvement in progression-free survival in the intent-to-treat population versus letrozole plus palbociclib, but a numerical improvement was observed. The adverse events for the giredestrant combination were manageable and consistent with the known safety profiles of each individual treatment.

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"While persevERA didn’t meet its primary objective, we are confident in the potential of giredestrant to become a new standard-of-care endocrine therapy in early and advanced ER-positive breast cancer," said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. "We believe there is a path forward for combining giredestrant with a CDK4/6 inhibitor in the adjuvant setting and we are conducting further studies. The efficacy demonstrated in evERA and lidERA provides clear validation of the clinical activity of giredestrant and reinforces the strength of our expanding clinical development program."

The giredestrant clinical development program is made up of distinct studies designed to reflect the specific disease biology of each stage of breast cancer. Genentech will continue to advance the clinical development program to identify the people with ER-positive breast cancer who can derive the greatest benefit from giredestrant.

Giredestrant Phase III clinical development program

Trial

Indication

Regimen

lidERA

Breast Cancer

Adjuvant ER+/HER2- breast cancer

Giredestrant vs. standard-of-care endocrine therapy (SoC ET)

persevERA Breast Cancer

1L ER+/HER2- metastatic breast cancer

(endocrine-sensitive)

Giredestrant + palbociclib vs. letrozole plus palbociclib

pionERA Breast Cancer

1L ER+/HER2- metastatic breast cancer

(endocrine-resistant)

Giredestrant + physician’s choice of CDK4/6 inhibitor vs. fulvestrant + physician’s choice of CDK4/6 inhibitor

evERA

Breast Cancer

2L+ ER+/HER2- metastatic breast cancer

Giredestrant + everolimus vs. SoC ET + everolimus

heredERA Breast Cancer

1L maintenance ER+/HER2+ metastatic breast cancer

Giredestrant + dual HER2 blockade vs. HER2 blockade

evERA was the first positive Phase III readout for giredestrant, followed by lidERA in the early-stage setting. The scientific rationale for lidERA was supported by prior results in the neoadjuvant setting, including the Phase II coopERA trial showing that giredestrant was superior to an aromatase inhibitor in reducing malignant cell division (Ki67 levels). This growing body of evidence underscores the potential of giredestrant to become a new standard-of-care endocrine therapy across ER-positive early-stage and advanced breast cancer.

persevERA is the first of two distinct Phase III studies in the first-line setting; the pionERA study of giredestrant in combination with physician’s choice of cyclin-dependent kinase (CDK)4/6 inhibitor in endocrine-resistant ER-positive, HER2-negative breast cancer is expected to readout in 2027.

The United States Food and Drug Administration (FDA) recently accepted the New Drug Application based on the evERA data. In the coming weeks, Genentech will submit the giredestrant Phase III lidERA data in early-stage breast cancer to the FDA.

The full results from persevERA will be presented at an upcoming medical meeting.

About the persevERA Breast Cancer study

persevERA Breast Cancer [NCT04546009] is a Phase III, randomized, double-blind, placebo-controlled, multicenter study evaluating the efficacy and safety of giredestrant plus palbociclib versus letrozole plus palbociclib as first-line treatment for people with estrogen receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer. The study enrolled 992 patients globally.

The primary endpoint is investigator-assessed progression-free survival. Key secondary endpoints include overall survival, objective response rate, duration of response and safety.

About giredestrant

Giredestrant is an investigational, oral, potent next-generation selective estrogen receptor degrader and full antagonist.

Giredestrant is designed to block estrogen from binding to the estrogen receptor (ER), triggering its breakdown (known as degradation) and stopping or slowing down the growth of cancer cells.

Giredestrant has an extensive clinical development program and is being investigated in five company-sponsored Phase III clinical trials that span multiple treatment settings and lines of therapy to benefit as many people as possible:

Giredestrant versus standard-of-care endocrine therapy (SoC ET) as adjuvant treatment in ER-positive, human epidermal growth factor receptor 2 (HER2)-negative early-stage breast cancer (lidERA Breast Cancer; NCT04961996)
Giredestrant plus everolimus versus SoC ET plus everolimus in ER-positive, HER2-negative, locally advanced or metastatic breast cancer (evERA Breast Cancer; NCT05306340)
Giredestrant plus palbociclib versus letrozole plus palbociclib in ER-positive, HER2-negative, endocrine-sensitive, recurrent locally advanced or metastatic breast cancer (persevERA Breast Cancer; NCT04546009)
Giredestrant plus investigator’s choice of a cyclin-dependent kinase (CDK)4/6 inhibitor versus fulvestrant plus a CDK4/6 inhibitor in ER-positive, HER2-negative advanced breast cancer resistant to adjuvant endocrine therapy (pionERA Breast Cancer; NCT06065748)
Giredestrant plus dual HER2 blockade versus dual HER2 blockade in ER-positive, HER2-positive locally advanced or metastatic breast cancer (heredERA Breast Cancer; NCT05296798)
About estrogen receptor (ER)-positive breast cancer

Globally, the burden of breast cancer continues to grow, with 2.3 million women diagnosed and 670,000 dying from the disease every year. Breast cancer remains the number one cause of cancer-related deaths amongst women, and the second most common cancer type.

ER-positive breast cancer accounts for approximately 70% of breast cancer cases. In the U.S. and EU5, an estimated 273,000 people are diagnosed in the early-stage setting, 88,000 people are diagnosed in first-line and 106,000 in the second and third-line setting combined.

A defining feature of ER-positive breast cancer is that its tumor cells have receptors that attach to estrogen, which can contribute to tumor growth.

Despite treatment advances, ER-positive breast cancer remains particularly challenging to treat due to its biological complexity. In the early-stage setting, up to a third of people eventually experience disease recurrence on or after adjuvant endocrine therapy treatment. Additionally, many have to interrupt or stop treatment early due to safety or tolerability issues, thereby increasing the risk of death. In advanced settings, resistance to endocrine therapy – particularly following treatment with cyclin-dependent kinase inhibitors – increases the risk of disease progression and is associated with poor outcomes.

There is an urgent need for more effective treatments that can delay clinical progression and reduce the burden of treatment on people’s lives.

(Press release, Genentech, MAR 9, 2026, View Source [SID1234663385])

On March 9, 2026 AstraZeneca and Daiichi Sankyo reported its supplemental Biologics License Application (sBLA) for Enhertu (trastuzumab deruxtecan) has been accepted and granted Priority Review in the US for the treatment of adult patients with HER2-positive breast cancer who have residual invasive disease after neoadjuvant HER2-targeted treatment.

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The Food and Drug Administration (FDA) grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available treatment options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance. The Prescription Drug User Fee Act date, the FDA action date for its regulatory decision, is anticipated during the third quarter of 2026.

Enhertu was recently granted Breakthrough Therapy Designation (BTD) by the FDA in this setting. BTD accelerates the development and regulatory review of potential new medicines intended to treat a serious condition and address a significant unmet medical need.

The sBLA also is being reviewed under Project Orbis, which provides a framework for concurrent submission and review of oncology medicines among participating international partners.

Around one in five breast cancers are considered HER2-positive, a subtype that is often associated with aggressive disease and poor prognosis.1-3 Currently, approximately half of patients with HER2-positive early breast cancer have residual disease following neoadjuvant treatment (before surgery), putting them at an increased risk of disease recurrence.4-9 Despite receiving additional treatment in the post-neoadjuvant setting with current standards of care, some patients still experience tumour progression to metastatic disease, where the five-year survival rate drops from nearly 90% to approximately 30%.10,11

Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: "While there has been significant progress in treating HER2-positive early breast cancer, managing patients at a higher risk of recurrence remains challenging. With this Priority Review, we move closer to bringing Enhertu to the post-neoadjuvant setting, offering more patients the opportunity for sustained long-term outcomes and a potential path to cure."

Ken Takeshita, Global Head, R&D, Daiichi Sankyo, said: "For patients with residual invasive disease after neoadjuvant therapy, identifying additional treatments following surgery is critical to help further reduce the risk of recurrence and help prevent progression to metastatic disease. This Priority Review reinforces the potential of Enhertu to become a new standard of care for HER2-positive early breast cancer based on the results of DESTINY-Breast05."

The sBLA is based on data from the DESTINY-Breast05 Phase III trial presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2025 Congress and subsequently published in The New England Journal of Medicine.1

In the trial, Enhertu significantly reduced the risk of invasive disease recurrence or death (invasive disease-free survival [IDFS]) by 53% compared with trastuzumab emtansine (T-DM1; based on a hazard ratio [HR] of 0.47; 95% confidence interval [CI] 0.34-0.66; p<0.0001) as a post-neoadjuvant treatment for patients with HER2-positive breast cancer. Enhertu demonstrated a three-year IDFS rate of 92.4% compared with 83.7% with T-DM1. IDFS findings were consistent across all prespecified subgroups.

Enhertu also significantly reduced the risk of disease recurrence or death (disease-free survival [DFS]), a key secondary endpoint, by 53% versus T-DM1 (HR 0.47; 95% CI 0.34-0.66; p<0.0001). Further, Enhertu lowered the risk of distant disease recurrence (distant recurrence-free interval) by 51% and the risk of brain metastases (brain metastasis-free interval) by 36% compared with T-DM1 (HR 0.64; 95% CI 0.35-1.17).

The safety profile of Enhertu observed in DESTINY-Breast05 was consistent with its known profile with no new safety concerns identified.

Regulatory submissions for Enhertu based upon DESTINY-Breast05 are also under review in the EU and Japan. In addition, an sBLA for Enhertu followed by paclitaxel, trastuzumab and pertuzumab (THP) currently is under review in the US for the neoadjuvant treatment of patients with HER2-positive early breast cancer based on results from the DESTINY-Breast11 trial.

Enhertu is already approved in more than 90 countries as a treatment for patients with HER2-positive metastatic breast cancer.

Enhertu is a specifically engineered HER2-directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.

Notes

Post-neoadjuvant treatment for HER2-positive early breast cancer
Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide.12 More than two million breast cancer cases were diagnosed in 2022, with more than 665,000 deaths globally.12 In the US, more than 320,000 cases of breast cancer are diagnosed annually with more than 42,000 deaths.13

HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumours including breast cancer.14 HER2 protein overexpression may occur as a result of HER2 gene amplification and is often associated with aggressive disease and poor prognosis in breast cancer.14 Approximately one in five cases of breast cancer are considered HER2-positive.2-3

For patients with HER2-positive early breast cancer, achieving pCR with neoadjuvant treatment is the earliest indicator of improved long-term survival.9 However, approximately half of patients who receive neoadjuvant treatment do not experience pCR, putting them at increased risk of disease recurrence.4-9

Despite receiving additional treatment for residual disease in the post-neoadjuvant setting, some patients still experience invasive disease or death, and current treatment options have shown limited impact on central nervous system recurrence.11 In the US, around 16,000 patients with HER2-positive early breast cancer receive treatment in the post-neoadjuvant setting (after surgery) each year.15

DESTINY-Breast05
DESTINY-Breast05 is a global, multicentre, randomised, open-label, Phase III trial evaluating the efficacy and safety of Enhertu (5.4mg/kg) versus T-DM1 in patients with HER2-positive early breast cancer with residual invasive disease in breast or axillary lymph nodes following neoadjuvant therapy and a high risk of recurrence. High risk of recurrence was defined as presentation with inoperable cancer (prior to neoadjuvant therapy) or pathologically positive axillary lymph nodes following neoadjuvant therapy.

The primary endpoint of DESTINY-Breast05 is investigator-assessed IDFS. IDFS is defined as the time from randomisation until first invasive local, axillary or distant recurrence or death from any cause. The key secondary endpoint is investigator-assessed disease-free survival. Other secondary endpoints include overall survival, distant recurrence-free interval, brain metastases-free interval and safety.

DESTINY-Breast05 enrolled 1,635 patients in Asia, Europe, North America, Oceania and South America. For more information about the trial, visit ClinicalTrials.gov.

Enhertu
Enhertu is a HER2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, Enhertu is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. Enhertu consists of a HER2 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

Enhertu (5.4mg/kg) in combination with pertuzumab is approved in the US as a 1st-line treatment for adult patients with unresectable or metastatic HER2-positive (IHC 3+ or ISH+) breast cancer, as determined by an FDA-approved test based on the results from the DESTINY-Breast09 trial.

Enhertu (5.4mg/kg) is approved in more than 90 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2-positive (IHC 3+ or ISH+) breast cancer who have received a prior anti-HER2-based regimen, either in the metastatic setting or in the neoadjuvant or adjuvant setting, and have developed disease recurrence during or within six months of completing therapy based on the results from the DESTINY-Breast03 trial.

Enhertu (5.4mg/kg) is approved in more than 90 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy based on the results from the DESTINY-Breast04 trial.

Enhertu (5.4mg/kg) is approved in more than 60 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-low (IHC 1+ or IHC 2+/ ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer, as determined by a locally or regionally approved test, that have progressed on one or more endocrine therapies in the metastatic setting based on the results from the DESTINY-Breast06 trial.

Enhertu (5.4mg/kg) is approved in more than 70 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumours have activating HER2 (ERBB2) mutations, as detected by a locally or regionally approved test, and who have received a prior systemic therapy based on the results from the DESTINY-Lung02 and/or DESTINY-Lung05 trials. Continued approval in China and the US for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Enhertu (6.4mg/kg) is approved in more than 80 countries/regions worldwide for the treatment of adult patients with locally advanced or metastatic HER2-positive (IHC 3+ or IHC 2+/ISH+) gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01, DESTINY-Gastric02 and/or DESTINY-Gastric06 trials. Continued approval in China for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Enhertu (5.4mg/kg) is approved in more than 10 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HER2-positive (IHC 3+) solid tumours who have received prior systemic treatment and have no satisfactory alternative treatment options based on efficacy results from the DESTINY-PanTumor02, DESTINY-Lung01 and DESTINY-CRC02 trials. Continued approval for this indication in the US may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Enhertu clinical development programme
A comprehensive global clinical development programme is underway evaluating the efficacy and safety of Enhertu as a monotherapy, in combination or sequentially with other cancer medicines across multiple HER2-targetable cancers.

(Press release, AstraZeneca, MAR 9, 2026, View Source [SID1234663345])

On March 9, 2026 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported that clinical data and updates from key programs within its hemato‑oncology pipeline will be presented at the 2026 EBMT Annual Meeting, taking place March 22–25, 2026 in Madrid, Spain.

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An oral presentation, scheduled during the Presidential Plenary Session, will detail the final results, including overall survival data, from the Company’s lead asset MaaT013, which is currently under review by the European Medicines Agency (EMA) following the submission of a Marketing Authorization Application in June 2025.

In addition, MaaT Pharma will present three poster communications, featuring:

Key results from CHRONOS, a multicenter retrospective cohort study describing real‑world outcomes in third‑line acute gastrointestinal GvHD, and
The study designs of two clinical trials (PHOEBUS and THRASSA – pediatric study announced in March 2025) advancing the Company’s therapeutic pipeline.
With this broad clinical data presentation at the 2026 EBMT Annual Meeting, MaaT Pharma reaffirms its position in hemato‑oncology across the full care continuum of HCT and its commitment to improving outcomes for patients undergoing allogeneic transplantation.

During this year’s Congress, Clinigen, MaaT Pharma’s strategic commercial partner for MaaT013’s Early Access Program and commercialization in Europe pending EMA approval, will host a dedicated symposium on Advancing Management of Steroid Refractory aGvHD with GI Involvement: From Unmet Need to a Potential Third Line Option. MaaT013 is currently under regulatory review and not yet approved.

The symposium will be chaired by Florent Malard, MD, PhD, hematology professor at Saint-Antoine Hospital and Sorbonne University and Fabio Ciceri, MD, PhD, Associate Professor of Hematology, University Vita-Salute San Raffaele. It will also feature renowned hematology experts such as Zinaida Peric, MD, hematologist University of Rijeka (Croatia); Chair, GvHD Subcommittee, Transplant Complications Working Party, EBMT, Ernst Holler, Professor at University Medical Center Regensburg (Germany) and Jaime Sanz, MD, hematologist and coordinator of the Bone Marrow Transplant Unit at the University Hospital La Fe in Valencia (Spain). The details of the Symposium are below:

Title: Advancing Management of Steroid Refractory aGvHD with GI Involvement: From Unmet Need to a Potential Third Line Option
Date: Sunday, March 22, 2026
Time: 12:30 – 14:00 CET
Room: N101–102
Professor Malard will also speak at a workshop session dedicated to the Current state of microbiota transplantation in allogeneic HCT, independently from any activities related to the Company, on Monday, March 23, 2026, from 11:00am – 11:20am CET in Velazquez location (W02 Workshop | GVHD beyond immunosuppression – Regulation, repair, and innovation).

EBMT abstracts are now available at www.ebmt.org. Details of the MaaT Pharma presentations are as follow:

Oral presentation:

Title: MaaT013 for Ruxolitinib-Refractory Acute Graft-versus-Host Disease with Gastrointestinal Involvement: Final Results from the ARES Phase III Trial
Abstract number: GS2-8
Session: GS2 Presidential Symposium
Session Date/Time: Monday, March 23, 2026 – 17:33 – 17:42 CET
Location: VELAZQUEZ
Presenter: Prof. Florent Malard, MD, PhD, hematology professor at Saint-Antoine Hospital and Sorbonne University and ARES Trial lead investigator
Poster Presentations:

PHOEBUS

Title: MaaT033 for Gut Microbiota Optimization To Improve Survival after Allogeneic HCT: the Phoebus Trial
Abstract number: A093
Session: Transplant and Cellular Therapies – Clinical
Session Date/Time: Monday, March 23, 2026 – 18:00 – 19:00 CET
Location: VELAZQUEZ
Presenter: Prof. Florent Malard, MD, PhD, hematology professor at Saint-Antoine Hospital and Sorbonne University and PHOEBUS Trial lead investigator
CHRONOS

Title: Key results from CHRONOS, a multicenter retrospective cohort study describing real-world outcomes in third-line acute gastrointestinal GvHD
Abstract number: B005
Session: Graft-versus-Host Disease – Clinical
Session Date/Time: Tuesday, March 24, 2026 – 18:00 – 19:00
Location: VELAZQUEZ
Presenter: Johannes Clausen, MD, hematologist at Ordensklinikum Linz Elisabethinen, Hematology Department, Linz, Austria
THRASSA

Title: THRASSA, a Multicenter Open-label Study Evaluating the Safety, Tolerability and Efficacy of MaaT013 in Ruxolitinib-Refractory or Intolerant Paediatric/Adolescent Participants with Gastrointestinal Acute Graft-versus-Host Disease
Abstract number: P222
Session: Paediatrics – Clinical
Session Date/Time: Sunday, March 22, 08:30 – 18:00 CET
Location: e-Poster area
Presenter: Marion Bruelle, Clinical Scientist at MaaT Pharma

(Press release, MaaT Pharma, MAR 9, 2026, View Source [SID1234663368])