AstraZeneca’s Q1 2025 Financial Results

On April 29, 2025 AstraZeneca reported Q1 2025 Financial Results (Press release, AstraZeneca, APR 29, 2025, View Source [SID1234652346]).

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Revenue and EPS summary

Q1 2025

% Change

$m

Actual

CER1

– Product Sales

12,875

6

9

– Alliance Revenue

639

40

42

Product Revenue2

13,514

7

10

Collaboration Revenue

74

64

Total Revenue

13,588

7

10

Reported EPS ($)

1.88

34

32

Core3 EPS ($)

2.49

21

21

Key performance elements for Q1 2025

(Growth numbers at constant exchange rates)

Total Revenue up 10% to $13,588m, driven by double-digit growth in Oncology and BioPharmaceuticals
Growth in Total Revenue across all major geographic regions
Core Operating profit increased 12%
Core Tax rate of 16% in the quarter due to timing of settlements. Expectations for the full year Core tax rate are unchanged at 18-22%
Core EPS increased 21% to $2.49
Five positive Phase III readouts and 13 approvals in major regions since the prior results
Pascal Soriot, Chief Executive Officer, AstraZeneca, said:

"Our strong growth momentum has continued into 2025 and we have now entered an unprecedented catalyst-rich period for our company.

Already this year we have announced five positive Phase III study readouts, including most recently the highly anticipated DESTINY-Breast09 for Enhertu, as well as SERENA-6 for camizestrant and MATTERHORN for Imfinzi; the latter two of these will feature in the ASCO (Free ASCO Whitepaper) 2025 plenary sessions, reflecting the significance of these data to the oncology community.

Our company is firmly committed to investing and growing in the US and we continue to benefit from our broad-based source of revenue and global manufacturing footprint, including eleven production sites in the US covering small molecules, biologics as well as cell therapy. Additionally, we have even greater US investment in manufacturing and R&D planned, leveraging our two large R&D sites in Gaithersburg MD and Cambridge MA.

Overall, we are making excellent progress toward our ambition of eighty billion dollars in Total Revenue by 2030."

See Table 1 for details of clinical trial results since the prior earnings announcement, including DESTINY-Breast09, MATTERHORN, and SERENA-6.

See Note 4 for the locations of the eleven US manufacturing sites.

Guidance

AstraZeneca reiterates its Total Revenue and Core EPS guidance5 for FY 2025 at CER, based on the average foreign exchange rates through 2024.

Total Revenue is expected to increase by a high single-digit percentage

Core EPS is expected to increase by a low double-digit percentage

– The Core Tax rate is expected to be between 18-22%

– If foreign exchange rates for April 2025 to December 2025 were to remain at the average rates seen in March 2025, it is anticipated that compared to the performance at CER, FY 2025 Total Revenue would incur a low single-digit percentage adverse impact (unchanged from prior guidance), and Core EPS would incur a low single-digit percentage adverse impact (previously mid single-digit).

Results highlights

Table 1. Milestones achieved since the prior results announcement

Phase III and other registrational data readouts

Medicine

Trial

Indication

Event

Enhertu

DESTINY-Gastric04

HER2-positive gastric/GEJ cancer (2nd-line)

Primary endpoint met

Enhertu

DESTINY-Breast09

HER2-positive metastatic breast cancer (1st line)

Primary endpoint met for combination arm

Imfinzi

MATTERHORN

Resectable gastric/GEJ cancer

Primary endpoint met

camizestrant

SERENA-6

HR+ HER2- metastatic breast cancer (1st line switch on emergence of ESR1m)

Primary endpoint met

eneboparatide

CALYPSO

Chronic hypoparathyroidism

Primary endpoint met, trial continues to 52 weeks

Regulatory approvals

Medicine

Trial

Indication

Region

Calquence

ACE-LY-004

Relapsed/refractory MCL

EU

Calquence

ChangE

CLL/SLL

CN

Datroway

TROPION-Breast01

HR+ HER2- breast cancer (2nd-line)

EU

Enhertu

DESTINY-Breast06

HER2-low and -ultralow HR+ breast cancer (2nd-line+)

EU

Imfinzi

AEGEAN

Resectable early-stage (IIA-IIIB) NSCLC

EU, CN

Imfinzi

NIAGARA

MIBC

US

Imfinzi ± Imjudo

ADRIATIC

SCLC (limited-stage)

EU, JP

Truqap

CAPItello-291

Biomarker-altered HR+ HER2- metastatic breast cancer

CN

Wainzua

NEURO-TTRansform

ATTRv-PN

EU

Beyonttra (acoramidis)

NCT04622046

ATTR-CM

JP

Ultomiris

CHAMPION-MG

gMG

CN

Regulatory submissions or acceptances* in major regions

Medicine

Trial

Indication

Region

Enhertu

DESTINY-Breast06

HER2-low and -ultralow HR+ breast cancer (2nd-line+)

CN

Imfinzi

PACIFIC-5

Locally advanced NSCLC

CN

Imfinzi + Imjudo

HIMALAYA

Unresectable HCC

CN

Imfinzi

HIMALAYA

Unresectable HCC

CN

Imfinzi

DUO-E

Primary advanced or recurrent endometrial cancer with mismatch repair deficiency

CN

Fasenra

MANDARA

EGPA

CN

Tezspire

WAYPOINT

CRSwNP

US, EU, JP, CN

Koselugo

KOMET

NF1-PN (adults)

US, CN

* US, EU and China regulatory submissions denotes filing acceptance

Other pipeline updates

For recent trial starts and anticipated timings of key trial readouts, please refer to the Clinical Trials Appendix, available on www.astrazeneca.com/investor-relations.html.

Table 2: Key elements of financial performance in Q1 2025

Item

Reported

Change

Core

Change

$m

Act

CER

$m

Act

CER

Product Revenue

13,514

7

10

13,514

7

10

■ See Tables 3 and 24 for medicine details of Product Revenue, Alliance Revenue and Product Sales

Collaboration Revenue

74

64

64

74

64

64

■ See Table 4 for details of Collaboration Revenue

Total Revenue

13,588

7

10

13,588

7

10

■ See Tables 5 and 6 for Total Revenue by Therapy Area and by region

Gross Margin (%)

84

+1pp

84

+1pp

+ Fluctuations in foreign exchange rates

− Pricing adjustments, for example to sales reimbursed by the Medicare Part D programme in the US

■ See ‘Reporting changes’ below for the definition of Gross Margin6

■ Variations in Gross Margin can be expected between periods, due to fluctuations in foreign exchange rates, product seasonality, Collaboration Revenue, and other effects

R&D expense

3,159

13

15

3,088

14

16

■ Core R&D: 23% of Total Revenue

+ Positive data read-outs for high value pipeline opportunities that have ungated late-stage trials

+ Investment in platforms, new technology and capabilities to enhance R&D capabilities

SG&A expense

4,492

3

3,457

1

4

■ Core SG&A: 25% of Total Revenue

Other operating income and expense7

113

71

71

115

79

78

+ Upfront receipt on a divestment

Operating Margin (%)

27

+2pp

+2pp

35

+1pp

Net finance expense

265

(12)

(11)

215

(12)

(11)

+ Debt issued in 2024 at higher interest rates

− Adjustment relating to tax settlements (see below)

Tax rate (%)

14

-8pp

-8pp

16

-6pp

-6pp

− Updates to estimates of prior period tax liabilities following settlements with tax authorities

EPS ($)

1.88

34

32

2.49

21

21

For monetary values the unit of change is percent; for Gross Margin, Operating Margin and Tax rate the unit of change is percentage points.

In the expense commentary above, the plus and minus bullets denote the directional impact of the item being discussed, e.g. a ‘+’ symbol beside an R&D expense comment indicates that the item resulted in an increase in the R&D spend relative to the prior year.

China

In April 2025, there are following developments in relation to the China investigations:

First, in relation to the illegal drug importation allegations, AstraZeneca received an Appraisal Opinion from the Shenzhen City Customs Office regarding suspected unpaid importation taxes amounting to $1.6 million. To the best of AstraZeneca’s knowledge, the importation taxes referred to in the Appraisal Opinion relate to Enhertu. A fine of between one and five times the amount of unpaid importation taxes may also be levied if AstraZeneca is found liable.

Second, in relation to the personal information infringement allegation, AstraZeneca received a Notice of Transfer to the Prosecutor from the Shenzhen Bao’an District Public Security Bureau (the ‘PSB’) regarding suspected unlawful collection of personal information. The Company has been informed that there was no illegal gain to the Company resulting from personal information infringement.

AstraZeneca continues to fully cooperate with the Chinese authorities.

Corporate and business development

FibroGen
In February 2025, FibroGen announced the sale of FibroGen China to AstraZeneca.

Under the terms of the agreement, FibroGen will receive an enterprise value of $85m plus FibroGen net cash held in China at closing, estimated at the date of signing to be approximately $75m, totalling approximately $160m. The transaction is expected to close by mid-2025, pending customary closing conditions, including regulatory review in China.

Upon closing, AstraZeneca will obtain all rights to roxadustat in China, including manufacturing in China.

EsoBiotec
In March 2025, AstraZeneca entered into a definitive agreement to acquire EsoBiotec, a biotechnology company pioneering in vivo cell therapies that has demonstrated promising early clinical activity. The EsoBiotec Engineered NanoBody Lentiviral (ENaBL) platform could offer many more patients access to transformative cell therapy treatments delivered in minutes rather than the current process which takes weeks.

AstraZeneca will acquire all outstanding equity of EsoBiotec for a total consideration of up to $1bn, on a cash and debt-free basis. This will include an initial payment of $425m on deal closing, and up to $575m in contingent consideration based on development and regulatory milestones. The transaction is expected to close in the second quarter of 2025, subject to customary closing conditions and regulatory clearances.

Alteogen Inc
In March 2025, AstraZeneca and Alteogen Inc. entered into an exclusive license agreement for ALT-B4, a novel hyaluronidase utilising Hybrozyme platform technology. Under the terms of the agreement, AstraZeneca has acquired worldwide rights to use ALT-B4 to develop and commercialise subcutaneous formulations of several oncology assets. Alteogen will be responsible for clinical and commercial supply of ALT-B4 to AstraZeneca. AstraZeneca has made an upfront payment to Alteogen and may make additional payments, conditional on achievement of specific development, regulatory and sales-related milestones. Additionally, Alteogen will receive royalties on the sales of the commercialised products.

Beijing R&D centre
In March 2025, AstraZeneca announced it will establish its sixth global strategic R&D centre, to be located in Beijing, China. It will be AstraZeneca’s second R&D centre in China, following the opening of the Shanghai R&D centre, and will advance early-stage research and clinical development, enabled by a state-of-the-art artificial intelligence and data science laboratory. The new R&D centre will be located near leading biotech companies, research hospitals, and the National Medical Products Administration in the Beijing International Pharmaceutical Innovation Park (BioPark).

Harbour BioMed
In March 2025, AstraZeneca executed a global strategic collaboration with Harbour BioMed to discover and develop next-generation multi-specific antibodies for immunology, oncology and beyond. The strategic collaboration includes an option to license multiple programs utilizing Harbour BioMed’s proprietary fully human antibody technology platform in multiple therapeutic areas, together with an equity investment in Harbour BioMed, which closed in April 2025. Upfront payments for the collaboration and equity investment total $175m. AstraZeneca may incur additional fees and contingent milestones for each program it elects to license, along with tiered royalties on future net sales.

BioKangtai
In March 2025, BioKangtai and AstraZeneca entered into a strategic partnership to establish a joint venture that focus on researching, developing, and producing innovative vaccines.

The joint venture will serve as AstraZeneca’s first and only vaccine production hub in China, with a registered capital of RMB 345m (approx. $50m) and a total investment of approx. $400m (RMB 2.76bn). BioKangtai and AstraZeneca will each hold 50% equity in the venture.

Syneron Bio
In March 2025, AstraZeneca executed a strategic collaboration with Syneron Bio to develop potential first-in-class macrocyclic peptides for the treatment of chronic diseases. Under this collaboration, AstraZeneca will gain access to Syneron Bio’s innovative macrocyclic peptide drug research and development platform to support research programmes exploring possible future treatments of chronic diseases, including rare, autoimmune, and metabolic disease. AstraZeneca will pay an upfront payment of $55m, with option exercise fees and contingent milestones of over $3bn if all programs are optioned, along with tiered royalties on future net sales. AstraZeneca will also make an equity investment in Syneron Bio.

Tempus AI and Pathos AI
In April 2025, AstraZeneca, Tempus AI, Inc. (Tempus) and Pathos AI, Inc. (Pathos) entered into a series of agreements regarding the development of a foundational large multimodal model in the field of oncology. The model will be used to gather biological and clinical insights, discover novel drug targets, and develop therapeutics. AstraZeneca will pay Tempus a fee, and a syndicate of investors, including AstraZeneca, will contemporaneously execute a stock purchase agreement with Pathos.

Sustainability highlights

In preparation for new reporting regulations, AstraZeneca combined its 2024 sustainability and annual reporting into one integrated publication. Details of performance against targets can be found in the 2024 Sustainability Data Annex.

AstraZeneca published its first Taskforce on Nature-related Financial Disclosures report, and its Sustainable use and sourcing of raw materials report.

Reporting calendar

The Company intends to publish its H1 and Q2 2025 results on 29 July 2025.

Conference call

A conference call and webcast for investors and analysts will begin today, 29 April 2025, at 11:45 UK time. Details can be accessed via astrazeneca.com.

Bayer Initiates Phase I Study Targeting GPC3 With Actinium-225 Radiopharmaceutical in Patients With Advanced Hepatocellular Carcinoma

On April 29, 2025 Bayer reported initiation of a Phase I clinical trial with 225Ac-GPC3 (BAY 3547926), an investigational targeted alpha radiopharmaceutical being developed to treat tumors expressing Glypican-3 (GPC3) in patients with advanced hepatocellular carcinoma (HCC) (Press release, Bayer, APR 29, 2025, View Source [SID1234652345]). Oncofetal protein GPC3 is a membrane-associated proteoglycan which is overexpressed in 70-75% of HCC lesions making it an attractive target for targeted radionuclide therapy.1,2 The first-in-human, dose escalation study (NCT06764316) will evaluate the safety, tolerability and preliminary efficacy of BAY 3547926 alone, and as a combination therapy in patients with advanced HCC.

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"Hepatocellular carcinoma continues to be a devastating disease for millions of patients and a pressing unmet need in cancer care. The launch of the Phase I trial using the 225Ac-GPC3 radionuclide therapy marks an important milestone in our commitment to develop new medicines targeting cancer cells with high effect size and precision to improve the lives of people living with cancer," said Dominik Ruettinger, M.D., Ph.D., Global Head of Research and Early Development for Oncology at Bayer’s Pharmaceuticals Division. "Through continued research innovation we can unlock the full potential of targeted alpha therapies which is an emerging class of targeted radionuclide therapy and a strategic focus area for Bayer’s precision oncology development portfolio."

Liver cancer, including hepatocellular carcinoma, is the third leading cause of cancer-related deaths in the world with almost 900,000 new cases annually.3 It is the most rapidly growing cause of cancer deaths in the US accounting for approximately 2% of new cases and 5% of cancer deaths.4 Despite recent scientific advancements, many doctors are not satisfied with the therapeutic benefits from the currently available treatments.

Targeted alpha therapy (TAT) has the potential to address high unmet medical need across various cancer types. Bayer’s growing TAT portfolio combines alpha particle-emitting radionuclides with different targeting moieties. 225Ac-GPC3 is the third TAT program in clinical development and the first investigational targeted radiopharmaceutical for Bayer in HCC. The newly disclosed targeted alpha conjugate joins 225Ac-Pelgifatamab and 225Ac-PSMA-Trillium, which are currently in Phase I clinical trials in patients with advanced metastatic castration resistant prostate cancer.

On April 28, 2025 Bayer introduced 225Ac-GPC3 in an oral presentation during the "New Drugs on the Horizon" session at the AACR (Free AACR Whitepaper) (American Association of Cancer Research) Annual Meeting, showcasing preclinical characterization of the asset including the low uptake and fast clearance from normal organs as well as induction of tumor regression in in vivo models. Recognition in this special symposium highlights the company’s commitment for precision oncology development portfolio.

1

Kolluri A and Ho M (2019), The Role of Glypican-3 in Regulating Wnt, YAP, and Hedgehog in Liver Cancer. Front. Oncol. 9:708. doi: 10.3389/fonc.2019.00708.
2

Yongle Wu, Hui Liu, Huiguo Ding, GPC-3 in hepatocellular carcinoma: current perspectives, Journal of Hepatocellular Carcinoma 2016:3, 63-67.
3

Global cancer statistics 2022: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries, Freddie Bray BSc, MSc, PhD; Mathieu Laversanne MSc; Hyuna Sung PhD; Jacques Ferlay ME; Rebecca L. Siegel MPH; Isabelle Soerjomataram MD, MSc, PhD; Ahmedin Jemal DVM, PhD.
4

SEER Cancer Stat Facts: Liver and Intrahepatic Bile Duct Cancer. National Cancer Institute. Bethesda, MD, View Source

About BAY 3547926
BAY 3547926 is an investigational targeted alpha radiopharmaceutical being developed to treat tumors expressing Glypican-3 (GPC3) in patients with advanced hepatocellular carcinoma (HCC). It is composed of a GPC3 targeting high affinity antibody radiolabeled with actinium-225 (225Ac). 225Ac-GPC3 delivers highly potent alpha-particles to the GPC3-expressing cancer cells, with the potential to inducing DNA double-strand breaks, reducing cancer cell viability which may potentially cause anti-tumor activity.

About Targeted Alpha Therapy
Targeted alpha therapy (TAT) is an emerging class of radionuclide therapy that can be used against a variety of tumors. It delivers alpha particle radiation directly to the tumor inside the body, either via its bone-seeking property (radium-223) or by combining alpha radionuclides, such as actinium-225, with specific targeting moieties.

Actinium-225 is an alpha particle–emitting radionuclide with a 9.9-day half-life. Alpha particles deposit highly ionizing radiation over a short range. This localized delivery of the radioactive payload induces irreparable DNA double-strand breaks, often resulting in cell death. At the same time, because the energy travels a short range, damage to nearby normal tissues is much reduced.

AbCellera Presents Data Showing Preclinical Efficacy of PSMA x CD3 T-Cell Engagers at AACR 2025

On April 29, 2025 AbCellera (Nasdaq: ABCL) reported new data on its PSMA x CD3 T-cell engagers (TCEs), presented as a poster at the American Association for Cancer Research (AACR) (Free AACR Whitepaper)Ⓡ (AACR) (Free AACR Whitepaper) 116th Annual Meeting at the McCormick Place Convention Center in Chicago, Illinois, taking place April 25 to 30, 2025 (Press release, AbCellera, APR 29, 2025, View Source [SID1234652344]).

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Targeting prostate-specific membrane antigen (PSMA) with a CD3 TCE could provide an effective treatment option for metastatic castration-resistant prostate cancer (mCRPC). In its poster presentation at AACR (Free AACR Whitepaper), AbCellera demonstrated that its PSMA x CD3 TCEs show promising preclinical activity, including:

Potent in vitro tumor-cell killing, with one molecule showing ~10x higher EC50 than benchmark
Sustained in vitro activity across four rounds of serial T-cell killing
Preclinical in vivo efficacy, with significant tumor growth inhibition in a xenograft mouse model
Potential for enhanced potency when combined with costimulatory PSMA x CD28 bispecifics
"The preclinical data package presented at AACR (Free AACR Whitepaper) is the first demonstration that our novel CD3-binders can be used to generate molecules with potent anti-tumor efficacy in vivo," said Adam Clarke, Ph.D., SVP, Discovery at AbCellera. "The poster underscores the strength of our TCE platform strategy, which is to combine diverse CD3- and tumor-binding antibodies and use empirical testing to engineer optimized therapeutic candidates. In addition, the data strongly support the use of costimulation to further increase anti-tumor activity."

Cellworks’ Study Predicts Immune Checkpoint Inhibitor Resistance in Patients with High Microsatellite Instability

On April 29, 2025 Cellworks Group Inc., a leader in Personalized Therapy Decision Support and Best-in-Class PTRS, reported compelling results from a new study demonstrating the ability of the Cellworks Platform to identify patients with high microsatellite instability (MSI-H) who may not respond to immune checkpoint inhibitors (ICIs), despite MSI-H status (Press release, Cellworks, APR 29, 2025, View Source [SID1234652343]). Results from the study were showcased in a poster presentation titled, Use of Biosimulation to Predict Immune Checkpoint Inhibitor Resistance in Patients with High Microsatellite Instability as part of the AACR (Free AACR Whitepaper) Annual Meeting 2025 taking place April 25-30, 2025 at the McCormick Place Convention Center in Chicago.

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While immune checkpoint inhibitors (ICIs) such as pembrolizumab are considered a standard-of-care for MSI-H cancers, MSI-H status alone is not a definitive predictor of treatment success. In this study, Cellworks applied its unique mechanistic Computational Biology Model (CBM) to biosimulate patient-specific responses to ICIs. The computational biosimulation process in the study uncovered molecular signatures of resistance in MSI-H patients who were predicted to have poor response to ICIs, providing a deeper understanding of why some MSI-H patients fail to benefit from immunotherapy.

Key Findings

Efficacy Scores Significantly Higher in MSI-H Patients. MSI-H patients demonstrated significantly higher pembrolizumab efficacy scores compared to microsatellite stable (MSS) patients in both STAD (average ES: 20.5 vs. 3.2, p < 0.001) and CRC (average ES: 13.4 vs. 2.4, p < 0.001).
Large Subset of MSI-H Patients Predicted to Have Low ICI Response. Despite being MSI-H, 59% of STAD and 81% of CRC patients were identified as low pembrolizumab responders.
Molecular Drivers of Resistance Identified. In MSI-H patients classified as low pembrolizumab responders, higher rates of NOTCH2, EGFR, and EZH2 amplifications, along with TP53 loss-of-function mutations, were identified. In MSI-H/ES-L CRC patients, MYC amplification was significantly enriched (p < 0.05).
"These findings highlight the power of using patient-specific drug response methods to move beyond MSI-H status and identify critical molecular drivers of immune checkpoint inhibitor resistance," said Dr. James Wingrove, Chief Development Officer at Cellworks and presenting author of the study. "By identifying patients unlikely to respond to ICIs, we can help oncologists personalize treatment strategies and improve outcomes for MSI-H patients who may otherwise receive ineffective therapies."

"This study demonstrates the importance of looking beyond MSI status to understand immune checkpoint inhibitor resistance at a molecular level," said Dr. Michael Castro, Chief Medical Officer at Cellworks. "Our biosimulation revealed that MSI-H patients with low predicted response to pembrolizumab frequently harbored alterations such as NOTCH2, EGFR, and EZH2 amplifications, as well as TP53 loss-of-function mutations in STAD, and MYC amplifications in CRC. Identifying these resistance-associated biomarkers can help guide clinicians in selecting more effective, personalized treatment strategies for MSI-H patients who may not benefit from ICIs alone."

Study Design

Cellworks developed a mechanistic Computational Biology Model (CBM) that can be personalized based on a patient’s tumor-based genomic profile, revealing signaling pathway dysregulation and patient-specific drug response. Output from the model was used to identify MSI-H patients who may have a poorer response to ICIs. Computational biosimulation was performed using real-world retrospective cohorts of 423 STAD patients and 534 CRC patients (TCGA). MSI measurements were provided by TCGA. Efficacy scores based on biosimulated composite cell growth in response to disease and therapy were generated on all patients for pembrolizumab. Molecular rationales for ICI resistance were identified for MSI-H patients with low pembrolizumab efficacy scores.

The Cellworks Platform

The Cellworks Platform performs computational biosimulation of protein-protein interactions, enabling in silico modeling of tumor behavior using comprehensive genomic data. This allows for the evaluation of how personalized treatment strategies interact with the patient’s unique tumor network. Multi-omic data from an individual patient or cohort is used as input to the in silico Cellworks Computational Biology Model (CBM) to generate a personalized or cohort-specific disease model.

The CBM is a highly curated mechanistic network of 6,000+ human genes, 30,000 molecular species and 600,000 molecular interactions. This model along with associated drug models are used to biosimulate the impact of specific compounds or combinations of drugs on the patient or cohort and produce therapy response predictions, which are statistically modeled to produce a qualitative therapy response score for a specific therapy. The Cellworks CBM has been tested and applied against various clinical datasets with results provided in over 125 presentations and publications with global collaborators.

Ensoma to Present Preclinical Data and Manufacturing Advancements for In Vivo HSC Engineering Platform to Treat Cancer, Immune Disorders and Genetic Diseases at ASGCT 28th Annual Meeting

On April 29, 2025 Ensoma, a genomic medicines company advancing the future of medicine through one-time, in vivo therapies designed to precisely and durably engineer the hematopoietic system to treat chronic diseases, reported it will present preclinical data and manufacturing advancements at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting, hosted May 13-17 in New Orleans (Press release, Ensoma, APR 29, 2025, View Source [SID1234652342]).

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The company’s poster presentations will highlight key programs that leverage its in vivo HSC engineering platform. Ensoma combines a unique delivery system with an advanced gene engineering toolkit, offering the potential for durable, transformative therapies to address chronic diseases, improve patient access and significantly reduce treatment burden. The platform uses virus-like particles (VLPs) that preferentially bind to HSCs, efficiently deliver DNA to the nucleus and de-target the liver. With a 35-kilobase cargo capacity, these VLPs carry a diverse range of genomic engineering constructs, including control elements that enable specific cargo expression in targeted cells, from single base edits to large multi-gene insertions.

"We look forward to presenting preclinical data at ASGCT (Free ASGCT Whitepaper) that underscore the potential of our in vivo HSC engineering platform to address complex genetic disorders and cancer," said Robert Peters, Ph.D., chief scientific officer of Ensoma. "Exciting new findings from our non-human primate studies reinforce the precision and effectiveness of our VLPs, showcasing their ability to safely deliver genetic therapies with targeted biodistribution and expression. With this strong progress, Ensoma remains on track to initiate our first clinical trial in the second half of 2025—marking a major step toward bringing this groundbreaking therapy to patients."

"Building on this momentum, our novel adenovirus production process is a significant milestone for Ensoma, ensuring the scalable and high-quality production of VLPs needed for clinical trials," said Dan Leblanc, chief technology officer of Ensoma. "This robust suspension manufacturing process is delivering consistent product yield and quality to support the clinical use of EN-374 for the treatment of X-CGD. It’s incredibly rewarding to see our platform move closer to delivering potentially transformative therapies to patients."

Poster Presentations at ASGCT (Free ASGCT Whitepaper) 28th Annual Meeting:

Title: In Vivo Engineering of Hematopoietic Stem Cells with Virus-like Particles to Generate Multi-Lineage CAR Immune Cell Therapy for Cancer (1783)
Poster Presentation Time/Date: 5:30-7:30 p.m. CT, Thursday, May 15
Location: Poster Hall I2
Presenter: Chirayu Chokshi, Ph.D., Ensoma

Data Summary: Ensoma will present updated data from its preclinical HER2 CAR program. An HSC-targeted VLP encoding lineage-specific regulatory elements to direct CAR expression resulted in robust generation of HSC-derived CAR-M, NK and T cells in vivo. This lineage-restricted, multi-cellular CAR therapy mediated tumor control and microenvironment remodeling, supporting Ensoma’s technology as a highly differentiated approach to addressing solid tumors.

Title: Novel In Vivo Gene Therapy Approach to Hematopoietic Stem Cell (HSC) Engineering Creates Durable HSC-Derived Neutrophils to Treat X-Linked Chronic Granulomatous Disease (1780)
Poster Presentation Time/Date: 5:30-7:30 p.m. CT, Thursday, May 15

Location: Poster Hall I2

Presenter: Sravya Kattula, Ph.D., Ensoma

Data Summary: Ensoma will present updated preclinical data from its X-CGD program with EN-374, which highlights the use of its VLP platform to efficiently modify HSCs in vivo and restore neutrophil function. In this preclinical study in a CGD mouse model, EN-374 provided durable gene correction in neutrophils to restore CYBB protein expression and activity. This study sets the foundation for Ensoma’s in vivo HSC gene therapy to reach the clinic and be applied to a range of genetic disorders that thus far have only been addressed with ex vivo gene therapies.

Title: Acute Safety and Biodistribution Profile of Hematopoietic Stem Cell (HSC) Targeting Virus-like Particles Based on Helper-dependent Adenovirus Serotype 5/35++ in Non-human Primates (1779)
Poster Presentation Time/Date: 5:30-7:30 p.m. CT, Thursday, May 15
Location: Poster Hall I2
Presenter: Patrick Au, Ph.D., Ensoma

Data Summary: Ensoma will present data from a non-human primate study evaluating the safety, biodistribution and transgene expression profiles of its HSC-targeting VLPs. Results showed favorable tolerability without any clinical signs of toxicity and a well-characterized biodistribution, supporting the continued development of Ensoma’s VLP-based platform as a safe and effective solution for in vivo HSC engineering.

Title: Development and Scale-up of a Novel Adenovirus Production Process (2016)
Poster Presentation Time/Date: 6:00-7:30 p.m. CT, Tuesday, May 13
Location: Poster Hall I2
Presenter: Chapman Wright, Ph.D., Ensoma

Data Summary: Ensoma will present the establishment of a novel, serum-free suspension manufacturing process for large-scale, high-efficiency VLP manufacturing. The presentation will address adaptation of an adherent cell line to a serum-free suspension cell culture, followed by a design-of-experiment strategy for efficient development of a clinical-scale production process.