Regeneron Reports First Quarter 2025 Financial and Operating Results

On April 29, 2025 Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) reported financial results for the first quarter of 2025 and provided a business update (Press release, Regeneron, APR 29, 2025, View Source [SID1234652324]).

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"Regeneron has one of the most exciting pipelines in the industry, with unmatched diversity, scientific distinction, and potential to help millions of patients," said Leonard S. Schleifer, M.D., Ph.D., Board co-Chair, President and Chief Executive Officer of Regeneron. "We are laser focused on fulfilling the promise of this pipeline by advancing our clinical programs, and expect several important data readouts this year. We are also working to ensure our four blockbuster medicines reach even more patients who could benefit, with year-to-date progress including regulatory approvals for Dupixent in CSU in the U.S. and COPD in Japan."

Financial Highlights

($ in millions, except per share data) Q1 2025 Q1 2024 % Change
Total revenues $ 3,029 $ 3,145 (4 %)
GAAP net income $ 809 $ 722 12 %
GAAP net income per share – diluted $ 7.27 $ 6.27 16 %
Non-GAAP net income(a) $ 928 $ 1,116 (17 %)
Non-GAAP net income per share – diluted(a) $ 8.22 $ 9.55 (14 %)

"We have made meaningful progress across our pipeline so far in 2025, including four regulatory approvals, nine regulatory submissions, and are poised to report pivotal or proof-of-concept data across programs in immunology, oncology, hematology, internal medicine, and rare diseases later this year," said Christopher Fenimore, Executive Vice President, Finance and Chief Financial Officer of Regeneron. "We continue to deploy capital with the goal of maximizing long-term shareholder value, with a focus on internal investment in our research, development, and commercial capabilities, along with returning capital directly to our shareholders through opportunistic share repurchases and our dividend program, which we initiated earlier this year."

Business Highlights

Key Pipeline Progress
Regeneron has approximately 45 product candidates in clinical development, including a number of marketed products for which it is investigating additional indications. Updates from the clinical pipeline include:

Dupixent (dupilumab)

In April 2025, the U.S. Food and Drug Administration (FDA) approved Dupixent for the treatment of adults and adolescents aged 12 years and older with CSU who remain symptomatic despite antihistamine treatment.
In March 2025, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved Dupixent for the treatment of patients with COPD.
The Company and Sanofi presented positive results from the pivotal Phase 2/3 trial in adults with moderate-to-severe bullous pemphigoid at the 2025 American Academy of Dermatology (AAD) Annual Meeting. In February 2025, the FDA accepted for priority review the supplemental Biologics License Application (sBLA) for Dupixent in bullous pemphigoid, with a target action date of June 20, 2025. A regulatory application has also been submitted in the European Union (EU).

EYLEA HD (aflibercept) 8 mg

In April 2025, the FDA accepted for priority review an sBLA for both the treatment of macular edema following RVO, and broadening the dosing schedule to include every 4-week (monthly) dosing across approved indications. The FDA target action date is August 19, 2025, following the use of a priority review voucher.
The FDA issued a Complete Response Letter (CRL) for the EYLEA HD pre-filled syringe on April 23, 2025. The Company held several teleconferences with the FDA to better understand the contents of the CRL, and believes the key outstanding issue relates to a question posed by the FDA to a third-party component supplier. This component supplier has expeditiously responded to FDA requests for information. The CRL did not identify any issues with the safety or efficacy of EYLEA HD, the usability of the device, proposed labelling, or pre-approval inspection findings.
In April 2025, the FDA issued a CRL regarding the sBLA for the addition of extended dosing intervals. The FDA indicated that the submitted data did not support extended dosing intervals greater than every 16 weeks. The Company is evaluating the FDA’s decision.
The Company presented positive results from the Phase 3 QUASAR trial for the treatment of patients with macular edema following RVO, including those with central, branch, and hemiretinal vein occlusions, at the Angiogenesis, Exudation, and Degeneration (Angiogenesis) 2025 annual meeting.
The Company announced positive three-year (156-week) results from an extension study of the Phase 3 PULSAR trial in patients with wet age-related macular degeneration (wAMD). Similar to the three-year results for the pivotal PHOTON trial in diabetic macular edema (DME), the longer-term wAMD data demonstrated the vast majority of patients who entered the extension study sustained the visual gains and anatomic improvements achieved by the end of the second year, while also achieving substantially longer treatment intervals. The results were presented at the Angiogenesis 2025 annual meeting.

Oncology Programs

The Company submitted an sBLA to the FDA and a regulatory application in the EU for Libtayo (cemiplimab) in adjuvant CSCC based upon data from a Phase 3 trial which demonstrated that adjuvant treatment with Libtayo was the first and only immunotherapy that led to a statistically significant and clinically meaningful improvement in the primary endpoint of disease-free survival (DFS) in patients with high-risk CSCC after surgery.
The FDA accepted for review the resubmission of the BLA for odronextamab, a bispecific antibody targeting CD20 and CD3, in relapsed/refractory (R/R) follicular lymphoma, with a target action date of July 30, 2025.
The FDA accepted for review the resubmission of the BLA for linvoseltamab, a bispecific antibody targeting BCMA and CD3, in R/R multiple myeloma, with a target action date of July 10, 2025.
The European Commission (EC) granted conditional marketing approval of Lynozyfic (linvoseltamab) to treat adults with R/R multiple myeloma. The indication is specific to those who have received at least three prior therapies including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy.
Enrollment was completed in a Phase 3 confirmatory trial (LINKER-MM3) for linvoseltamab in R/R multiple myeloma.

Other Programs

The Company presented updated data from the Phase 1/2 trial of DB-OTO, an AAV-based gene therapy, in children with profound genetic hearing loss due to variants of the otoferlin (OTOF) gene at the Association for Research in Otolaryngology’s (ARO) 48th Annual MidWinter Meeting. The data showed that 11 out of 12 children treated demonstrated a notable response, with improved hearing at various decibel hearing levels. This included updated results from the first child dosed, who received treatment at 10 months of age and at 48 weeks post-treatment showed continued near-normal levels of hearing, as well as formal speech perception improvements between 48- and 72-weeks post-treatment.
A Phase 3 study for itepekimab, an antibody to IL-33, in chronic rhinosinusitis with nasal polyposis (CRSwNP) was initiated.
A Phase 2 study for REGN5381, an agonist antibody to NPR1, in uncontrolled hypertension was initiated.
A Phase 2 study for REGN7544, an antagonist antibody to NPR1, in sepsis-induced hypotension was initiated.
The FDA granted Orphan Drug designation for mibavademab, an agonist antibody to leptin receptor (LEPR), in generalized lipodystrophy; a Phase 3 study is ongoing.

Corporate Updates

The Company reached resolution of its patent infringement litigation related to Biocon’s EYLEA (aflibercept) Injection 2 mg biosimilar product. The settlement precludes Biocon from launching its biosimilar product in the United States until the second half of 2026. All intellectual property-related litigation with Biocon in the United States has been dismissed.
The Company announced a 10-year agreement with FUJIFILM Diosynth Biotechnologies (Fujifilm) to manufacture and supply Regeneron’s commercial bulk drug product at Fujifilm’s North Carolina campus. The agreement is anticipated to nearly double the Company’s large-scale manufacturing capacity in the United States.
First Quarter 2025 Financial Results

Revenues

($ in millions) Q1 2025 Q1 2024 % Change
Net product sales:
EYLEA HD – U.S. $ 307 $ 200 54 %
EYLEA – U.S. 736 1,202 (39 %)
Total EYLEA HD and EYLEA – U.S. 1,043 1,402 (26 %)
Libtayo – U.S. 192 159 21 %
Libtayo – ROW** 93 105 (11 %)
Total Libtayo – Global 285 264 8 %
Praluent – U.S. 57 70 (19 %)
Evkeeza – U.S. 31 24 29 %
Inmazeb – ROW — 1 (100 %)
Total net product sales 1,416 1,761 (20 %)

Collaboration revenue:
Sanofi 1,183 910 30 %
Bayer 344 356 (3 %)
Other 4 1 *
Other revenue 82 117 (30 %)
Total revenues $ 3,029 $ 3,145 (4 %)

* Percentage not meaningful
** Rest of world (ROW)

Net product sales of EYLEA HD increased in the first quarter of 2025, compared to the first quarter of 2024, primarily due to higher sales volumes.

Net product sales of EYLEA in the first quarter of 2025, compared to the first quarter of 2024, were negatively impacted by (i) lower volume as a result of continued competitive pressures (as described below), loss in market share to compounded bevacizumab due to patient affordability constraints, and the continued transition of patients to EYLEA HD, and (ii) a lower net selling price.

In addition, total EYLEA and EYLEA HD net product sales were negatively impacted by lower wholesaler inventory levels at the end of the first quarter of 2025 compared to the end of the fourth quarter of 2024. Total EYLEA and EYLEA HD net product sales decreased 30% on a sequential basis; however, physician unit demand decreased sequentially by 11%.

EYLEA net product sales have been, and are likely to continue to be, negatively impacted by increased competition from other anti-VEGF products, including biosimilars, as well as the transition of patients from EYLEA to EYLEA HD. In addition, if independent not-for-profit patient assistance funds that provide copay assistance are unable to support eligible patients, this will likely have a continued negative impact on patient affordability resulting in lower utilization of higher-cost anti-VEGF agents.

Sanofi collaboration revenue increased in the first quarter of 2025, compared to the first quarter of 2024, due to an increase in the Company’s share of profits from the commercialization of antibodies, which were $1.018 billion and $804 million in the first quarter of 2025 and 2024, respectively. The change in the Company’s share of profits from commercialization of antibodies was driven by higher profits associated with an increase in Dupixent sales.

Refer to Table 4 for a summary of collaboration revenue.

Operating Expenses

GAAP %
Change
Non-GAAP(a) %
Change

($ in millions) Q1 2025 Q1 2024 Q1 2025 Q1 2024
Research and development (R&D) $ 1,327 $ 1,248 6 % $ 1,186 $ 1,122 6 %
Acquired in-process research and development (IPR&D) $ 12 $ 7 71 % * * n/a
Selling, general, and administrative (SG&A) $ 633 $ 689 (8 %) $ 537 $ 584 (8 %)
Cost of goods sold (COGS) $ 266 $ 240 11 % $ 217 $ 196 11 %
Gross margin on net product sales(c) 81% 86% 85% 89%
Cost of collaboration and contract manufacturing (COCM) $ 199 $ 193 3 % * * n/a
Other operating expense (income), net $ — $ 15 (100 %) * $ — — %

* GAAP and non-GAAP amounts are equivalent as no non-GAAP adjustments have been recorded

GAAP and non-GAAP R&D expenses increased in the first quarter of 2025, compared to the first quarter of 2024, driven by the advancement of the Company’s clinical pipeline and higher personnel-related costs.
GAAP and non-GAAP gross margin on net product sales was adversely impacted by higher inventory write-offs and reserves in the first quarter of 2025 compared to the first quarter of 2024.
Other Financial Information

GAAP other income (expense), net included the recognition of net unrealized gains on equity securities of $140 million in the first quarter of 2025, compared to $196 million of net unrealized losses in the first quarter of 2024.

In the first quarter of 2025, the Company’s GAAP effective tax rate (ETR) was 10.6%, compared to (3.0%) in the first quarter of 2024. The GAAP ETR increased in the first quarter of 2025, compared to the first quarter of 2024, due to lower tax benefits from less stock option exercises. In the first quarter of 2025, the non-GAAP ETR was 11.6%, compared to 6.1% in the first quarter of 2024.

A reconciliation of the Company’s GAAP to non-GAAP results is included in Table 3 of this press release.

Capital Allocation

In February 2025, the Company’s board of directors authorized a new share repurchase program to repurchase up to an additional $3.0 billion of the Company’s common stock. During the first quarter of 2025, the Company repurchased shares of its common stock and recorded the cost of the shares, or $1.052 billion, as Treasury Stock. As of March 31, 2025, an aggregate of $3.874 billion remained available for share repurchases under the Company’s share repurchase programs.

In April 2025, the Company’s board of directors declared a cash dividend of $0.88 per share on the Company’s common stock and Class A stock, payable on June 6, 2025 to shareholders of record as of May 20, 2025.

Rakovina Therapeutics Showcases Preclinical Results of Novel AI-Discovered Cancer Therapies at AACR 2025

On April 29, 2025 Rakovina Therapeutics Inc. (TSX-V: RKV) (FSE: 7JO), a biopharmaceutical company advancing next-generation cancer therapies through artificial intelligence (AI)-powered drug discovery, reported the presentation of new preclinical data from two of its lead programs at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 (Press release, Rakovina Therapeutics, APR 29, 2025, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-showcases-preclinical-results-of-novel-ai-discovered-cancer-therapies-at-aacr-2025 [SID1234652322]).

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The first presentation highlighted Rakovina’s PARP1-selective inhibitor program. Using the proprietary Deep Docking and generative platforms , Rakovina previously screened more than four billion potential drug-like molecules to identify a shortlist of top candidates. These compounds were synthesized and evaluated in Rakovina’s laboratory facilities at the University of British Columbia.

Data presented at AACR (Free AACR Whitepaper) demonstrated a new class of PARP1 inhibitors with significantly improved metabolic stability, including the lowest in vitro clearance rates and the longest half-life compared to other candidates currently in development. Early animal studies revealed strong plasma exposure and a promising pharmacokinetic profile suggestive of central nervous system (CNS) penetration — a potentially significant advantage in treating brain-involved malignancies.

The second presentation showcased progress from Rakovina’s ATR-specific inhibitor program, developed in partnership with Variational AI. Researchers identified a focused set of lead candidates predicted to be highly potent and selective against ATR, a key DNA damage response target. These candidates are also designed with the potential to cross the blood-brain barrier, an important feature for addressing cancers affecting the CNS. The top candidates are currently being synthesized for laboratory validation.

"Our ability to screen billions of molecules and advance top candidates to in vitro and in vivo validation within months — rather than years — exemplifies the transformative power of AI in drug discovery," said Jeffrey Bacha, Executive Chairman of Rakovina Therapeutics. "This acceleration gives us the opportunity to significantly reduce the time and cost required to bring new life-saving cancer treatments to patients."

The results presented at AACR (Free AACR Whitepaper) reinforce the robustness of Rakovina’s AI-enabled discovery platform. Moving forward, Rakovina will leverage these findings to further refine and train its AI models, with the goal of advancing best-in-class lead candidates for both the PARP1 and ATR programs into clinical development. The Company intends to collaborate with pharmaceutical partners to accelerate the path to the clinic and deliver novel therapies to patients in need.

Pasithea Therapeutics Announces Completion of Enrollment and Initial Dosing of Patients in Cohort 6 from its Phase 1 Trial of PAS-004 in Advanced Cancer Patients

On April 29, 2025 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, for the treatment of neurofibromatosis type 1 (NF1) and other MAPK pathway driven cancer indications, reported the Company has completed enrollment and initial dosing of three subjects in Cohort 6 with 30 mg capsules of PAS-004 (Press release, Pasithea Therapeutics, APR 29, 2025, View Source [SID1234652321]).

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"We are pleased to have recruited, enrolled and commenced dosing of the initial three subjects in Cohort 6 more rapidly than anticipated and we currently expect to complete enrollment of all patients in the trial by the end of 2025," said Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea.

The ongoing Phase 1 clinical trial is a multi-center, open-label, dose escalation 3+3 study design to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of PAS-004 in patients with MAPK pathway driven advanced solid tumors with a documented RAS, NF1 or RAF mutation or patients who have failed BRAF/MEK inhibition (NCT06299839).

Nuvalent Announces Publication in Molecular Cancer Therapeutics Reinforcing Rational Molecular Design of Zidesamtinib as a Novel ROS1-Selective Inhibitor

On April 29, 2025 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported the publication of a manuscript in Molecular Cancer Therapeutics, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper), which supports the rational molecular design of zidesamtinib, its novel and selective ROS1 inhibitor (Press release, Nuvalent, APR 29, 2025, https://investors.nuvalent.com/2025-04-29-Nuvalent-Announces-Publication-in-Molecular-Cancer-Therapeutics-Reinforcing-Rational-Molecular-Design-of-Zidesamtinib-as-a-Novel-ROS1-Selective-Inhibitor [SID1234652317]). Zidesamtinib is currently being evaluated in the ongoing ARROS-1 Phase 1/2 trial for patients with advanced ROS1-positive non-small cell lung cancer (NSCLC) and other solid tumors, which is designed with registrational intent for tyrosine kinase inhibitor (TKI) pre-treated and TKI-naïve patients with advanced ROS1-positive NSCLC.

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The publication, entitled "Zidesamtinib Selective Targeting of Diverse ROS1 Drug-Resistant Mutations," is published online and can be accessed here: View Source

"Zidesamtinib was specifically designed with the goal of addressing the combined medical needs of treating tumors that have developed resistance, treating brain metastases and avoiding off-target adverse events. Structural studies play a critical role in the development and optimization of novel therapeutics, particularly when aiming to solve for multiple, and at times competing, challenges. To date, structural studies for ROS1-positive cancers have been hindered by a lack of ROS1 G2032R crystal structures, despite G2032R being the most commonly occurring ROS1 resistance mutation," said first author Anupong Tangpeerachaikul, Ph.D., Director, Biology at Nuvalent. "With this publication in Molecular Cancer Therapeutics, we are pleased to have shared what is, to our knowledge, the first structure of ROS1 G2032R, or any ROS1 mutation, offering a framework for understanding ROS1 TKI activity against these important drivers of disease progression. This structure further illustrates the intentional design of zidesamtinib and adds to the growing body of preclinical data supporting its ROS1-selective and TRK-sparing design."

The manuscript explores the activity of zidesamtinib and other approved or investigational ROS1 TKIs at clinically relevant concentrations against ROS1 resistance mutations, including the most commonly occurring resistance mutation, ROS1 G2032R, in preclinical mutagenesis screens and an intracranial ROS1 G2032R xenograft model. Findings presented in the manuscript show that, at clinically relevant concentrations, zidesamtinib suppressed on-target resistance in ENU mutagenesis screens simulating first-line and later-line treatment and inhibited ROS1 G2032R brain tumors more effectively than the other ROS1 TKIs evaluated. This favorable preclinical activity suggests the potential for zidesamtinib to delay tumor progression, both peripherally and intracranially.

In addition, the manuscript details the first crystal structure of ROS1 G2032R in complex with zidesamtinib, which further supports zidesamtinib’s molecular design and provides structural insights into how the ROS1 G2032R mutation affects TKI binding. Zidesamtinib was designed with the goal of being active against ROS1 and ROS1 resistance mutations while avoiding the inhibition of the structurally related tropomyosin receptor kinase (TRK) family in the central nervous system (CNS), which has been associated with neurological adverse events that can be dose limiting. The crystal structure elucidates zidesamtinib’s preclinical affinity for ROS1 G2032R and selectivity for ROS1 over TRK.

The company expects to report pivotal clinical data for TKI pre-treated patients with advanced ROS1-positive NSCLC from the ARROS-1 Phase 1/2 trial in the first half of 2025 in support of an anticipated New Drug Application submission by mid-year 2025, with an initial target indication of TKI pre-treated patients with advanced ROS1-positive NSCLC.

About Zidesamtinib
Zidesamtinib is a novel brain-penetrant ROS1-selective inhibitor created with the aim to overcome limitations observed with currently available ROS1 inhibitors. Zidesamtinib is designed to remain active in tumors that have developed resistance to currently available ROS1 inhibitors, including tumors with treatment-emergent ROS1 mutations such as G2032R. In addition, zidesamtinib is designed for central nervous system (CNS) penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ROS1 inhibitors and to drive deep, durable responses for patients across all lines of therapy. Zidesamtinib has received breakthrough therapy designation for the treatment of patients with ROS1-positive metastatic non-small cell lung cancer (NSCLC) who have been previously treated with 2 or more ROS1 tyrosine kinase inhibitors and orphan drug designation for ROS1-positive NSCLC.

Nouscom Presents Positive Final Results from Completed Phase Ib/II Study of Neoantigen Immunotherapy NOUS-209 at AACR 2025, Demonstrating a Highly Potent and Durable Immune Response in Lynch Syndrome Carriers

On April 29, 2025 Nouscom, a clinical-stage biotech company developing next-generation neoantigen-targeted off-the-shelf and personalized cancer immunotherapies, reported complete safety and immunogenicity results from a Phase Ib/II study evaluating NOUS-209 in individuals with Lynch Syndrome (LS) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025 (Press release, NousCom, APR 29, 2025, View Source;utm_medium=rss&utm_campaign=nouscom-presents-positive-final-results-from-completed-phase-ib-ii-study-of-neoantigen-immunotherapy-nous-209-at-aacr-2025-demonstrating-a-highly-potent-and-durable-immune-response-in-lynch-syndrome [SID1234652315]). The study found that NOUS-209 monotherapy was safe, well-tolerated and induced potent, broad and durable immune responses in all LS carriers evaluated.

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LS is a common inherited condition that significantly increases a person’s risk of developing microsatellite instability (MSI)-associated cancers over their lifetime. Managing LS is limited to frequent screenings or elective organ removal surgery. NOUS-209 is an investigational off-the-shelf cancer immunotherapy that targets tumors with mismatch repair deficiency (dMMR) and/or MSI. These tumors produce markers known as frameshift peptide neoantigens (FSPs). NOUS-209 is a pioneering approach to cancer interception comprising two proprietary viral vectors that deliver 209 shared FSP neoantigens and train the immune system to recognize and attack cancerous and pre-cancerous cells before tumors can fully develop.

The completed Phase Ib/II trial evaluated safety and immunogenicity in 45 LS carriers demonstrated;

Favorable Safety Profile: NOUS-209 was well tolerated, with no serious treatment-related adverse events across the entire study population.
Potent and Durable Immunogenicity Profile: Neoantigen-specific T cell responses were reported in 100% of evaluable participants (n=37). Responses were robust, reaching a mean of ~1100 interferon-gamma (IFN-γ) spot forming cells (SFC) per million of Peripheral Blood Mononuclear Cells (PBMCs). Immune responses were durable, with tumor-specific T cells detected after 1 year in >85% of participants (n=33).
Broad, Polyspecific T Cell Response: Immune responses were confirmed against 115 different FSP neoantigens to date, validating the ability of NOUS-209 to elicit broad polyspecific immune responses.
Desired T Cell Phenotype: NOUS-209 induced neoantigen-specific CD8 and CD4 T cells, exhibiting an effector memory phenotype associated with long-lived immunity critical for sustained surveillance and ability to eliminate emerging tumor cells.
Demonstration of Tumor Cell Killing: T cells induced by NOUS-209 were shown to directly kill tumor cells ex vivo, confirming functional anti-cancer activity.
Target Validation in LS-Associated Premalignant and Cancer Lesions: The vast majority of the immunogenic FSP neoantigens were shown to be present in both pre-cancerous as well as cancer lesions from independent cohorts of LS carriers.
These data support the further clinical development of NOUS-209 as a monotherapy in LS carriers. Following positive Type B and C meetings with the US Food and Drug Administration (FDA), Nouscom has a clear path forward for the advancement of NOUS-209 to a potentially registration-enabling Phase 2/3 clinical study for cancer interception in those living with LS.

"Currently, individuals with Lynch Syndrome rely on frequent screenings, such as colonoscopies, to manage their markedly increased risk of developing cancer. These latest data are a step toward a completely new approach – leveraging the immune system for cancer interception," said the study’s principal investigator, Eduardo Vilar-Sanchez, M.D., Ph.D., Professor of Clinical Cancer Prevention at The University of Texas MD Anderson Cancer Center. "NOUS-209 has shown clear evidence that the T cells it activates can persist over time, effectively target and kill tumor cells and develop into memory cells that support long-term immune protection. These findings support NOUS-209’s potential as a cancer interception strategy."

"Nouscom’s proprietary viral vector platform is uniquely positioned to deliver rapid, potent and broad immune activation against a large number of shared neoantigens, with minimal reactogenicity and durable immune responses," said Dr. Elisa Scarselli, Chief Scientific Officer of Nouscom. "These compelling Phase Ib/II data further reinforce our confidence in NOUS-209 monotherapy to safely and effectively prime the immune system to recognize and intercept pre-malignant and cancer lesions before they progress into tumors in LS carriers."

Dr. Marina Udier, Chief Executive Officer of Nouscom, added: "We are enormously pleased with these robust Phase Ib/II data and look to progress toward a potentially registration-enabling Phase 2/3 clinical study for cancer interception in LS. We remain deeply committed to advancing NOUS-209 and bring better solutions for people living with LS who urgently need and deserve a better way to manage their cancer risk."

The study was led by researchers at MD Anderson, in collaboration with the Cancer Prevention Clinical Trials Network and sponsored by the National Cancer Institute (grant # UG1CA242609) of the National Institutes of Health. These data were presented during the Hot Topics in Cancer Prevention session, and the abstract is available on the AACR (Free AACR Whitepaper) website here.