Positive Late-Breaking Data for Incyte’s First-in-Class mutCALR-targeted therapy INCA033989 in Essential Thrombocythemia Presented at EHA2025




Positive Late-Breaking Data for Incyte’s First-in-Class mutCALR-targeted therapy INCA033989 in Essential Thrombocythemia Presented at EHA2025

• Data demonstrates the potential for INCA033989 to modify disease by directly inhibiting and eliminating oncogenic mutCALR cells, while sparing healthy cells and restoring normal blood cell production
• In high-risk patients with essential thrombocythemia (ET) with a CALR mutation (mutCALR), 86% of INCA033989-treated patients at doses 400 mg and above achieved a complete or partial hematologic response with the majority (82%) realizing a complete response
• A reduction in peripheral blood mutCALR variant allele frequency (VAF) was observed in 89% of evaluable patients correlating with hematologic response
• Initial results demonstrate a favorable safety profile – no dose limiting toxicities were reported, a maximum tolerated dose was not reached and 98% of patients remained on treatment
• Incyte will host an in-person analyst and investor event highlighting this data at EHA today, Sunday, June 15, 2025, from 6:00 – 7:30 a.m. EDT (12:00 -1:30 p.m. CEST)

WILMINGTON, Del.–(BUSINESS WIRE)–$INCY–Incyte (Nasdaq:INCY) today announced the first clinical data from two studies evaluating the safety, tolerability and efficacy of INCA033989, a novel, first in class, Incyte-discovered, targeted monoclonal antibody in patients with mutant calreticulin (mutCALR)-expressing myeloproliferative neoplasms (MPNs). These data – featured today in the Late-Breaking Oral Session (#LB4002) at the European Hematology Association 2025 (EHA2025) Congress in Milan, Italy – focus on the dose escalation portion of the studies in patients with high risk essential thrombocythemia (ET) who are resistant/intolerant to prior cytoreductive therapy.

The studies evaluated the safety and efficacy of INCA033989 in patients with ET as measured by hematologic response and reduction in mutCALR variant allele frequency (VAF).

Results as of April 4, 2025, showed rapid and durable normalization of platelet counts across all dose levels, with a trend toward improved responses in higher doses (>400 mg), in patients with ET treated with INCA033989. Notably, 86% of patients at doses 400 mg and above achieved a complete or partial hematologic response, with the majority (82%) of patients achieving complete response. Eighty-nine (89) percent of evaluable patients (34/38) showed a reduction in mutCALR VAF from baseline. A partial molecular response (>50% VAF reduction) was observed in 21% of evaluable patients (8/38) after only 3 cycles of treatment.

An exploratory study using single-cell DNA (scDNA) sequencing showed that INCA033989 directly targets and reduces cells carrying mutCALR. This reduction was seen in early blood-forming (CD34-positive) cells and cells in the myeloid-erythroid (ME) lineage. At the same time, there was a clear increase in healthy (wild-type CALR) cells, suggesting that the treatment supports the return of normal blood production. Bone marrow biopsies further confirmed these effects showing fewer megakaryocytes with mutCALR protein and a notable increase in megakaryocytes without mutCALR protein. Together, these findings demonstrate the selectivity of INCA033989, allowing for normalization of healthy hematopoiesis and disease modification.

“The late-breaking data presented today highlight the impact of INCA033989, a novel agent that selectively targets mutant CALR, to inhibit and eliminate cancer-causing cells in patients with essential thrombocythemia (ET), while sparing healthy cells and normalizing healthy blood production,” said Pablo J. Cagnoni, M.D., President, Head of Research and Development, Incyte. “These findings, and the further development of INCA033989, offer the potential to significantly transform the treatment of patients with CALR-mutant myeloproliferative neoplasms (MPNs).”

The results (N=49) showed that INCA033989 was well tolerated across all dose cohorts (24 to 2,500 mg), with no dose-limiting toxicities observed. Only one (1) patient discontinued treatment, and only one (1) dose reduction due to treatment-emergent adverse events (TEAEs) was observed. No infusion interruptions due to TEAEs were reported, and a maximum tolerated dose was not reached. Forty-two (42) patients across the dose cohorts reported a TEAE. The most common TEAEs were fatigue (26.5%) and upper respiratory tract infection (20.4%), all of which were Grade ≤2. Thirteen (13) patients had Grade >3 TEAEs, with transient asymptomatic lipase increase as the most common (6%).

“mutCALR is the second most common oncogenic driver of MPNs, yet the therapeutic landscape lacks a targeted agent for mutCALR expressing MPNs. Currently, ET treatments aim to prevent vascular complications and improve symptoms but are limited by toxicity and tolerability issues,” said John Mascarenhas, M.D., Professor of Medicine at the Icahn School of Medicine at Mt. Sinai and Director, Center of Excellence for Blood Cancers and Myeloid Disorders, The Tisch Cancer Institute. “These data support the hypothesis that INCA033989 has the potential not only to normalize platelet counts and provide rapid and durable hematologic responses – but to induce molecular responses, which could potentially change the natural history of the disease.”

Additional data from the INCA033989 study in patients with myelofibrosis will be submitted for presentation at a future medical meeting. Discussions with regulatory authorities are planned with the goal to initiate a Phase 3 study by early 2026.

More information regarding the EHA2025 Congress and the data from Incyte’s hematology/oncology portfolio being featured at the meeting can be found on the EHA website: https://ehaweb.org/congress/eha2025-congress.

Incyte Conference Call and Webcast

Incyte will host an in-person analyst and investor event on Sunday, June 15, 2025, from 6:00 – 7:30 a.m. ET (12:00 – 1:30 p.m. CEST) to discuss key mutCALR data presented at EHA.

The event will be webcasted and can be accessed via the Events and Presentations tab of the Investor section of Incyte.com and it will be available for replay for 30 days.

About Myeloproliferative Neoplasms

Myeloproliferative neoplasms (MPNs) are a closely related group of blood cancers in which the bone marrow functions abnormally. The bone marrow is where the body’s blood cells are made. MPNs are progressive blood cancers that can strike anyone at any age, but they are more common in older adults. Estimates of the prevalence of MPNs vary, but analysis of claims data suggests there may be as many as 200,000 people in the U.S. living with the most prevalent MPNs: myelofibrosis, polycythemia vera or essential thrombocythemia (ET).¹

About Mutations in Calreticulin (mutCALR)

Calreticulin (CALR) is a protein involved in the regulation of cellular calcium levels and normal protein folding. Somatic, or non-inherited, DNA mutations in the CALR gene (mutCALR) can result in abnormal protein function and lead to the development of myeloproliferative neoplasms (MPNs),² a closely related group of clonal blood cancers in which the bone marrow functions abnormally, overproducing blood cells.^3,4 Among two types of MPNs, essential thrombocythemia (ET) and myelofibrosis (MF), mutCALR drives 25-35% of all cases.^2,3 There are approximately 60,000 patients in the U.S. and Europe with mutCALR positive ET.⁵

Incyte is at the forefront of developing novel therapies for patients with mutCALR ET or MF that target only malignant cells, sparing normal cells, including INCA033989, a first-in-class, mutCALR-specific therapy.

About the INCA033989 Trial Program

The clinical trial program for INCA033989 includes two multicenter, open-label Phase 1 studies, INCA33989-101 (NCT05936359) and INCA33989-102 (NCT06034002), enrolling ~225 patients outside of the U.S. and ~140 patients in the U.S., respectively. The studies are evaluating the safety, tolerability, dose-limiting toxicity (DLT) and maximum tolerated dose (MTD) and/or recommended dose(s) for expansion (RDE) of INCA033989 administered as a monotherapy or in combination with ruxolitinib in patients with myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET) and myelofibrosis (MF). The intent of Part 1A (dose escalation) is to identify the MTD and/or the RDE of INCA033989 among patients with MF and ET. In Part 1A INCA033989 is administered intravenously every two weeks at a protocol defined dose ranging from 24 mg. to 2,500 mg. In Part 1B (dose expansion), INCA033989 is administered at the RDE(s) identified during Part 1A.

The primary endpoint of the studies focuses on safety and tolerability as measured by: the number of participants with DLTs up to 28 days, the number of participants with treatment-emergent adverse events (TEAEs) up to 3 years and 60 days, and the number of participants with TEAEs leading to dose modification or discontinuation up to 3 years and 60 days. Secondary endpoints include response rates, mean change of ET total symptom score from baseline, percentage of MF patients achieving spleen volume reduction, MF patient anemia response, mean change in disease-related allele burden and various pharmacokinetics measures up to 3 years and 60 days.

For more information on the study, please visit: https://clinicaltrials.gov/study/NCT05936359 and https://clinicaltrials.gov/study/NCT06034002.

About Incyte

A global biopharmaceutical company on a mission to Solve On., Incyte follows the science to find solutions for patients with unmet medical needs. Through the discovery, development and commercialization of proprietary therapeutics, Incyte has established a portfolio of first-in-class medicines for patients and a strong pipeline of products in Oncology and Inflammation & Autoimmunity. Headquartered in Wilmington, Delaware, Incyte has operations in North America, Europe and Asia.

For additional information on Incyte, please visit Incyte.com or follow us on social media: LinkedIn, X, Instagram, Facebook, YouTube.

Forward-Looking Statements

Except for the historical information set forth herein, the matters set forth in this press release, including statements regarding the presentation of data for Incyte’s anti-mutCALR monoclonal antibody (INCA033989), the potential this monoclonal antibody offers for patients, and expectations regarding ongoing and future clinical trials contain predictions, estimates, and other forward-looking statements.

These forward-looking statements are based on Incyte’s current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials and the ability to enroll subjects in accordance with planned schedules; determinations made by the FDA, EMA and other regulatory agencies; Incyte’s dependence on its relationships with and changes in the plans of its collaboration partners; the efficacy or safety of Incyte’s products and the products of Incyte’s collaboration partners; the acceptance of Incyte’s products and the products of Incyte’s collaboration partners in the marketplace; market competition; unexpected variations in the demand for Incyte’s products and the products of Incyte’s collaboration partners; the effects of announced or unexpected price regulation or limitations on reimbursement or coverage for Incyte’s products and the products of Incyte’s collaboration partners; sales, marketing, manufacturing and distribution requirements, including Incyte’s and its collaboration partners’ ability to successfully commercialize and build commercial infrastructure for newly approved products and any additional products that become approved; greater than expected expenses, including expenses relating to litigation or strategic activities; variations in foreign currency exchange rates; and other risks detailed in Incyte’s reports filed with the Securities and Exchange Commission, including its annual report on form 10-K and our quarterly report on Form 10-Q for the quarter ended March 31, 2025. Incyte disclaims any intent or obligation to update these forward-looking statements.

Contacts

Incyte Contacts:
Media
media@incyte.com

Investors
ir@incyte.com

Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne




Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne

– The Company is developing an enhanced immunosuppressive regimen in consultation with a panel of multi-disciplinary clinical experts and engaging with regulators

– Shipments of ELEVIDYS for infusions in non-ambulatory patients in commercial setting are suspended until enhanced regimen is approved and in place

– ENVISION study is paused while seeking a protocol amendment to incorporate additional immunosuppression

– Sarepta to host investor call on June 16, 2025, at 8:00 am Eastern time

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the Company is taking to strengthen the safety profile in non-ambulatory patients. These steps follow a second reported case of acute liver failure (ALF) resulting in death. The cases of ALF to date have both occurred in non-ambulatory individuals with Duchenne. Sarepta extends its deepest sympathies to the affected families and care teams.

Key Safety Initiatives

Evaluating and Enhancing Immunosuppressive Regimen: As part of a comprehensive review of safety data, Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients. Sarepta is working to immediately convene an independent group of leading experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for ELEVIDYS. This panel will evaluate data and assess our proposed regimen, which includes sirolimus and is supported by preclinical data demonstrating the effectiveness of additional immunosuppression in moderating liver enzyme elevations, a key factor in mitigating potential safety events. Sarepta will share the panel’s recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance.

Suspending Shipments of ELEVIDYS for Non-Ambulatory Patients: Sarepta is temporarily suspending shipments of ELEVIDYS for non-ambulatory patients while an enhanced immunosuppressive regimen is evaluated, discussed with regulatory bodies, and put in place.

For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after ELEVIDYS infusion, along with post-treatment monitoring, remains the same.

ENVISION Study Paused: Sarepta has voluntarily paused dosing in the ENVISION clinical study (also known as Study SRP-9001-303). FDA concurs with this action. The pause will allow for the evaluation of a protocol amendment to incorporate an enhanced immunosuppressive regimen for the non-ambulatory patient cohort and incorporate any additional feedback from the FDA. Regulatory alignment is needed before screening and dosing in ENVISION may resume.

ENVISION is a global, randomized, double-blind, placebo-controlled trial evaluating ELEVIDYS in older ambulatory and non-ambulatory individuals living with Duchenne muscular dystrophy. In the U.S., it serves as the confirmatory trial required under the FDA’s accelerated approval pathway for non-ambulatory patients.

“Our paramount priority is the safety and well-being of the patients we serve. We are taking immediate, decisive steps to better understand and mitigate the risk of acute liver failure, including enhancing the immunosuppressive regimen, for those with Duchenne who are non-ambulatory,” said Louise Rodino-Klapac, Ph.D., chief scientific officer and head of research & development, Sarepta. “We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient’s family and his care team during this incredibly difficult time. Duchenne muscular dystrophy is a devastating disease that profoundly affects lives and often cuts them far too short. With more than 900 individuals treated to-date, we know how much hope families place in new treatment options like ELEVIDYS – and we are committed to honoring that hope by acting swiftly, guided by scientific rigor and the insights of leading experts, to strengthen safety for all future patients.”

Commitment to Long-Term Safety and Understanding

Sarepta remains committed to a thorough approach and the highest standards of patient safety and scientific rigor. The event has been reported to FDA and global health authorities and will inform ongoing discussions around a potential label update to reflect the risk of severe ALF and additional immune management strategies for non-ambulatory patients. While elevated liver enzymes are a known class effect of all AAV-based gene therapies, the exact mechanism behind AAV-related liver toxicity remains unclear. Current evidence suggests it is likely driven by an adaptive immune response. The Company will provide additional updates as appropriate.

Investor Conference Call Details

Sarepta will be hosting a conference call and webcast to discuss this update and provide an update on the Company’s business on Monday, June 16, 2025, at 8:00 am Eastern time. The event will be webcast live under the investor relations section of Sarepta’s website at: https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

About ELEVIDYS (delandistrogene moxeparvovec-rokl)

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle.

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.

• For patients who are ambulatory and have a confirmed mutation in the DMD gene

• For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

WARNINGS AND PRECAUTIONS:

Infusion-related Reactions:

• Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate.

• ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.

• Discontinue infusion for anaphylaxis.

Acute Serious Liver Injury:

• Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks.

• Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.

• Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels).

• Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.

Immune-mediated Myositis:

• In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed.

• Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.

• Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur.

Myocarditis:

• Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.

• If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath.

• Advise patients to contact a physician immediately if they experience cardiac symptoms.

Preexisting Immunity against AAVrh74:

• In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies.

• Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.

• ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400.

Adverse Reactions:

• The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.

Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

For further information, please see the full Prescribing Information.

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.

Internet Posting of Information

We routinely post information that may be important to investors in the ‘For Investors’ section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Forward-Looking Statements

This statement contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, clinical trials, ELEVIDYS, the potential benefits of an enhanced immunosuppression regimen in dosing in non-ambulatory patients, and expected plans and milestones, including providing additional updates as appropriate and engaging with regulators on an enhanced immunosuppressive regimen for dosing in non-ambulatory patients.

Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, these data may not be sufficient to support approval by the FDA or other global regulatory authorities; success in clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or with advisory committee recommendations, or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading “Risk Factors” in our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.

Contacts

Investor Contact:
Ian Estepan

617-274-4052

iestepan@sarepta.com

Media Contacts:
Tracy Sorrentino

617-301-8566

tsorrentino@sarepta.com

Kara Hoeger

617-710-3898

KHoeger@sarepta.com

High Throughput Process Development Strategic Business Report 2025: Market to Reach $24.6 Billion by 2030 – Surge in Demand for Personalized Medicine Strengthens Opportunities – ResearchAndMarkets.com




High Throughput Process Development Strategic Business Report 2025: Market to Reach $24.6 Billion by 2030 – Surge in Demand for Personalized Medicine Strengthens Opportunities – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “High Throughput Process Development – Global Strategic Business Report” report has been added to ResearchAndMarkets.com’s offering.

The global market for High Throughput Process Development was valued at US$15.3 Billion in 2024 and is projected to reach US$24.6 Billion by 2030, growing at a CAGR of 8.3% from 2024 to 2030. This comprehensive report provides an in-depth analysis of market trends, drivers, and forecasts, helping you make informed business decisions. The report includes the most recent global tariff developments and how they impact the High Throughput Process Development market.

What Factors Are Driving the Growth in the High Throughput Process Development Market?

The growth in the high throughput process development market is driven by several factors, including the increasing complexity of biologic drugs, the rising demand for personalized medicines, and advancements in automation and data analytics. As the biopharmaceutical industry shifts toward more complex therapeutics, such as gene and cell therapies, the need for efficient process optimization is more critical than ever. HTPD allows companies to streamline the development of these therapies, reducing both time and costs.

The growing demand for biosimilars, which require rapid process development to compete with branded biologics, is also contributing to market growth. Additionally, the adoption of automation and machine learning in process development is further enhancing the efficiency and accuracy of bioprocess optimization, driving the market’s expansion.

High throughput process development (HTPD) is a critical tool in the biopharmaceutical industry, enabling faster, more efficient development of biologic drugs. HTPD involves the use of automated, miniaturized experiments that allow researchers to test multiple process conditions simultaneously, significantly accelerating the optimization of production processes for therapeutic proteins, vaccines, and monoclonal antibodies.

The ability to screen numerous variables at once reduces the time and resources needed to scale up bioprocesses, from lab-scale to commercial manufacturing. As biopharmaceutical companies face increasing pressure to reduce costs and bring therapies to market faster, HTPD has become indispensable in ensuring both process efficiency and product quality.

How Are Technological Innovations Shaping High Throughput Process Development?

Technological innovations, particularly in automation, robotics, and data analytics, have revolutionized high throughput process development. Automation systems now allow for the simultaneous running of hundreds of experiments, enabling researchers to optimize cell culture conditions, purification processes, and formulation strategies more efficiently. The integration of advanced data analytics and machine learning into HTPD platforms has further enhanced process optimization, allowing for predictive modeling and real-time decision-making. Microfluidic technologies, which allow for the precise control of small volumes of liquids, are also playing a critical role in reducing reagent use and increasing the speed of bioprocess optimization. These technologies are enabling biopharmaceutical companies to streamline their development timelines and reduce production costs.

How Do Market Segments Define the Growth of the High Throughput Process Development Market?

Product types include consumables, software, and instruments, with consumables like reagents and microplates holding the largest market share due to their constant need in HTPD workflows. Services such as contract research and process development outsourcing are also expanding, as biopharmaceutical companies increasingly seek external expertise to accelerate drug development. The biopharmaceutical industry, particularly companies focused on biologics such as monoclonal antibodies, is the primary end-user of HTPD, as it enables rapid scale-up from R&D to manufacturing. Other key users include academic and research institutes involved in biotechnology research.

Report Scope

Report Features:

• Comprehensive Market Data: Independent analysis of annual sales and market forecasts in US$ Million from 2024 to 2030.
• In-Depth Regional Analysis: Detailed insights into key markets, including the U.S., China, Japan, Canada, Europe, Asia-Pacific, Latin America, Middle East, and Africa.
• Company Profiles: Coverage of players such as Agilent Technologies, Aurora Biomed, Axxam S.P.A., Bio-Rad Laboratories, Danaher Corporation and more.
• Complimentary Updates: Receive free report updates for one year to keep you informed of the latest market developments.

Key Insights:

• Market Growth: Understand the significant growth trajectory of the Monoclonal Antibodies segment, which is expected to reach US$17.8 Billion by 2030 with a CAGR of a 9.1%. The Other Molecule Types segment is also set to grow at 6.5% CAGR over the analysis period.
• Regional Analysis: Gain insights into the U.S. market, valued at $3.9 Billion in 2024, and China, forecasted to grow at an impressive 12.6% CAGR to reach $6.4 Billion by 2030. Discover growth trends in other key regions, including Japan, Canada, Germany, and the Asia-Pacific.

Segments

• Molecule Type (Monoclonal Antibodies, Other Molecule Types)
• Component (Tools & Systems, Consumables, Software, Other Components)
• Technology (Chromatography, Ultraviolet-Visible Spectroscopy, Other Technologies)
• End-Use (Biopharmaceutical Companies, Contract Research Organizations, Academic Research Institutes)

Tariff Impact Analysis: Key Insights for 2025

What’s Included in This Edition:

• Tariff-adjusted market forecasts by region and segment
• Analysis of cost and supply chain implications by sourcing and trade exposure
• Strategic insights into geographic shifts

Buyers receive a free July 2025 update with:

• Finalized tariff impacts and new trade agreement effects
• Updated projections reflecting global sourcing and cost shifts
• Expanded country-specific coverage across the industry

Key Attributes:

Key Topics Covered:

MARKET OVERVIEW

• Influencer Market Insights
• Tariff Impact on Global Supply Chain Patterns
• Global Economic Update
• High Throughput Process Development – Global Key Competitors Percentage Market Share in 2024 (E)
• Competitive Market Presence – Strong/Active/Niche/Trivial for Players Worldwide in 2024 (E)

MARKET TRENDS & DRIVERS

• Rising Demand for Biopharmaceuticals Drives Growth in High Throughput Process Development
• Technological Advancements in Automation and Robotics Propel Innovation in High Throughput Process Development
• Increasing Focus on Speed and Efficiency in Drug Discovery Expands Addressable Market for High Throughput Solutions
• Growing Adoption of High Throughput Technologies in Vaccine Development Spurs Market Growth
• Surge in Demand for Personalized Medicine Strengthens Business Case for High Throughput Process Development
• Increasing Use of High Throughput Screening in Protein and Antibody Discovery Expands Market Potential
• Technological Innovations in Microfluidics and Lab Automation Propel Market Innovation
• Growing Focus on Reducing Time-to-Market for Pharmaceuticals Drives Adoption of High Throughput Processes
• Rising Use of Artificial Intelligence (AI) in High Throughput Data Analysis Strengthens Competitive Advantage
• Increasing Focus on Process Optimization and Scalability in Biopharmaceutical Production Drives Innovation
• Rising Demand for High Throughput Analytical Techniques in Quality Control Expands Market Opportunities

FOCUS ON SELECT PLAYERS: Some of the 33 companies featured in this report

• Agilent Technologies
• Aurora Biomed
• Axxam S.P.A.
• Bio-Rad Laboratories
• Danaher Corporation
• Eppendorf
• GE Healthcare
• Hamilton Company
• Merck Millipore
• Perkinelmer
• Sartorius Stedim Biotech
• Tecan Group
• Thermo Fisher Scientific, Inc.

For more information about this report visit https://www.researchandmarkets.com/r/sge48r

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Nutraceutical Excipients Global Strategic Business Report 2025: Growing Use of Probiotics and Prebiotics Expands Opportunities for Specialized Nutraceutical Excipients – Global Forecast to 2030 – ResearchAndMarkets.com




Nutraceutical Excipients Global Strategic Business Report 2025: Growing Use of Probiotics and Prebiotics Expands Opportunities for Specialized Nutraceutical Excipients – Global Forecast to 2030 – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Nutraceutical Excipients – Global Strategic Business Report” report has been added to ResearchAndMarkets.com’s offering.

The global market for Nutraceutical Excipients was valued at US$5.0 Billion in 2024 and is projected to reach US$7.2 Billion by 2030, growing at a CAGR of 6.2% from 2024 to 2030. This comprehensive report provides an in-depth analysis of market trends, drivers, and forecasts, helping you make informed business decisions.

Nutraceutical excipients are inactive ingredients used in the production of nutraceutical products such as dietary supplements, functional foods, and fortified beverages. These excipients serve a crucial role by aiding in the stabilization, preservation, and delivery of active ingredients like vitamins, minerals, and bioactive compounds.

Growth in the Nutraceutical Excipients Market Is Driven by Several Factors

Growth in the nutraceutical excipients market is driven by several factors, including innovations in excipient technology, evolving consumer preferences, and increasing regulatory scrutiny. Advances in excipient formulations, such as the development of multifunctional and bioenhancing excipients, are expanding the range of nutraceutical products available to consumers.

The growing popularity of clean-label and organic products is also fueling demand for natural excipients that meet consumer expectations for purity and safety. Additionally, the rise in chronic health conditions and aging populations is increasing the need for functional and targeted nutraceuticals, further driving demand for excipients that improve the bioavailability and stability of active ingredients. Regulatory requirements for product safety and quality are pushing manufacturers to innovate, ensuring excipients meet the highest standards for efficacy and safety.

How Are Innovations in Nutraceutical Excipients Shaping the Market?

Technological advancements in excipient development are driving significant changes in the nutraceutical market. The demand for excipients that are organic, non-GMO, and free from allergens is on the rise, reflecting consumer preferences for clean-label products. New innovations in multifunctional excipients, which can perform multiple roles such as improving both bioavailability and stability, are gaining traction.

Additionally, advancements in nanotechnology are enabling the development of excipients that enhance the solubility and absorption of poorly soluble nutraceutical ingredients. These innovations are leading to the production of more effective, faster-acting, and longer-lasting nutraceutical products, appealing to consumers who prioritize both efficacy and natural ingredients.

How Are Consumer Preferences and Regulatory Trends Impacting Demand for Nutraceutical Excipients?

The growing focus on health and wellness has spurred consumer demand for nutraceutical products that are safe, effective, and free from artificial additives. This shift is influencing the nutraceutical excipients market, as manufacturers are increasingly opting for natural, organic, and clean-label excipients that align with consumer values.

Regulatory bodies are also playing a key role by setting stringent guidelines for excipient safety and quality, particularly in regions like North America and Europe. As a result, excipient manufacturers are investing in research and development to create products that comply with these regulations while offering superior functionality. Moreover, the rise of personalized nutrition is driving demand for excipients that enable the production of customized nutraceutical solutions, expanding the market’s scope.

Report Scope

Report Features:

• Comprehensive Market Data: Independent analysis of annual sales and market forecasts in US$ Million from 2024 to 2030.
• In-Depth Regional Analysis: Detailed insights into key markets, including the U.S., China, Japan, Canada, Europe, Asia-Pacific, Latin America, Middle East, and Africa.
• Company Profiles: Coverage of players such as ABF Ingredients, Cargill, Inc., Dow, Inc., DuPont de Nemours, Inc., EGGLE Wasserburg Verwaltungs GmbH and more.
• Complimentary Updates: Receive free report updates for one year to keep you informed of the latest market developments.

Key Insights:

• Market Growth: Understand the significant growth trajectory of the Binders segment, which is expected to reach US$2.4 Billion by 2030 with a CAGR of a 7.6%. The Fillers & Diluents segment is also set to grow at 5.5% CAGR over the analysis period.
• Regional Analysis: Gain insights into the U.S. market, valued at $1.3 Billion in 2024, and China, forecasted to grow at an impressive 9.5% CAGR to reach $1.7 Billion by 2030. Discover growth trends in other key regions, including Japan, Canada, Germany, and the Asia-Pacific.

Segments:

• Functionality (Binders, Fillers & Diluents, Coating Agents, Lubricants, Disintegrants, Flavoring Agents)
• Form (Dry, Liquid)

Tariff Impact Analysis: Key Insights for 2025

What’s Included in This Edition:

• Tariff-adjusted market forecasts by region and segment
• Analysis of cost and supply chain implications by sourcing and trade exposure
• Strategic insights into geographic shifts

Buyers receive a free July 2025 update with:

• Finalized tariff impacts and new trade agreement effects
• Updated projections reflecting global sourcing and cost shifts
• Expanded country-specific coverage across the industry

Key Attributes:

Key Topics Covered:

MARKET OVERVIEW

• Influencer Market Insights
• Tariff Impact on Global Supply Chain Patterns
• Global Economic Update
• Nutraceutical Excipients – Global Key Competitors Percentage Market Share in 2024 (E)
• Competitive Market Presence – Strong/Active/Niche/Trivial for Players Worldwide in 2024 (E)

MARKET TRENDS & DRIVERS

• Rising Consumer Demand for Functional Foods and Dietary Supplements Drives Growth in Nutraceutical Excipients Market
• Technological Advancements in Drug Delivery Systems Propel the Adoption of Innovative Nutraceutical Excipients
• Increasing Preference for Clean-Label and Natural Ingredients Expands the Market for Organic Nutraceutical Excipients
• Growing Focus on Enhanced Bioavailability and Solubility Strengthens Demand for Advanced Excipient Formulations
• Rising Awareness of Preventative Healthcare and Wellness Fuels Growth in Nutraceutical Supplements, Driving Excipient Demand
• Technological Innovations in Multi-Functional Excipients Propel Growth in Nutraceutical Manufacturing Efficiency
• Increased Regulatory Scrutiny on Ingredient Safety and Quality Standards Spurs Demand for Compliant Excipients
• Growing Use of Probiotics and Prebiotics Expands Opportunities for Specialized Nutraceutical Excipients
• Consumer Trends Toward Sugar-Free and Low-Calorie Supplements Accelerate Demand for Sweetening and Flavoring Excipients
• Expansion of Sports Nutrition and Weight Management Products Throws the Spotlight on Performance-Enhancing Excipients
• Rising Popularity of Plant-Based and Vegan Nutraceuticals Strengthens the Business Case for Non-Animal Derived Excipients
• Growth in Elderly Population and Demand for Age-Related Supplements Propel Market Adoption of Digestive-Friendly Excipients

FOCUS ON SELECT PLAYERS

• ABF Ingredients
• Cargill, Inc.
• Dow, Inc.
• DuPont de Nemours, Inc.
• EGGLE Wasserburg Verwaltungs GmbH
• Hilmar Ingredients
• IMCD Group BV
• Ingredion, Inc.
• Innophos Holdings, Inc.
• Jrs Pharma GmbH Co. KG
• Kerry Group plc
• Roquette Freres S.A.
• Sensient Pharmaceutical

For more information about this report visit https://www.researchandmarkets.com/r/3j2d84

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

press@researchandmarkets.com
For E.S.T Office Hours Call 1-917-300-0470

For U.S./ CAN Toll Free Call 1-800-526-8630

For GMT Office Hours Call +353-1-416-8900

Pharmacy Automation Strategic Research Report 2025: Market to Reach $9.9 Billion by 2030 – Integration of IoT in Pharmacy Systems Generates Demand for Smart Solutions – ResearchAndMarkets.com




Pharmacy Automation Strategic Research Report 2025: Market to Reach $9.9 Billion by 2030 – Integration of IoT in Pharmacy Systems Generates Demand for Smart Solutions – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Pharmacy Automation – Global Strategic Business Report” report has been added to ResearchAndMarkets.com’s offering.

The global market for Pharmacy Automation was valued at US$6.6 Billion in 2024 and is projected to reach US$9.9 Billion by 2030, growing at a CAGR of 6.9% from 2024 to 2030. This comprehensive report provides an in-depth analysis of market trends, drivers, and forecasts, helping you make informed business decisions. The report includes the most recent global tariff developments and how they impact the Pharmacy Automation market.

What Factors Are Driving Growth in the Pharmacy Automation Market?

The growth in the pharmacy automation market is driven by several factors. Firstly, the increasing demand for efficient and error-free medication dispensing systems is propelling the adoption of automation technologies. Technological advancements in robotics, AI, and IoT are enhancing the capabilities of pharmacy automation systems, making them more reliable and efficient. The rising incidence of chronic diseases and an aging population are increasing the volume of medications dispensed, necessitating more efficient pharmacy operations.

Additionally, the growing emphasis on reducing medication errors and improving patient safety is driving the demand for automated systems. The expansion of hospital infrastructure and the growth of retail pharmacies are also contributing to market growth. Furthermore, regulatory pressures for compliance with drug safety standards are encouraging the adoption of pharmacy automation solutions. These factors collectively support the robust growth and continuous innovation within the pharmacy automation market.

Revolutionizing Medication Management: The Emergence of Pharmacy Automation

Pharmacy automation refers to the use of technology and machinery to handle and streamline various tasks in a pharmacy setting, such as medication dispensing, packaging, labeling, and inventory management. This automation significantly reduces the risk of human error, enhances operational efficiency, and allows pharmacists to focus more on patient care and consultation.

In hospital and retail pharmacies, automated systems ensure the accurate dispensing of medications, which is critical for patient safety. Automated processes also improve the management of drug inventories, reducing waste and ensuring that medications are always in stock. Overall, pharmacy automation represents a transformative shift in the pharmaceutical industry, improving accuracy, efficiency, and patient safety.

How Is Technology Transforming Pharmacy Operations?

Technological advancements have profoundly impacted pharmacy operations, driving the adoption of sophisticated automation systems. Modern pharmacy automation solutions include robotic dispensing systems, automated medication storage and retrieval systems, and electronic prescription processing.

Robotics and AI-driven systems can handle repetitive tasks with high precision, drastically reducing dispensing errors and freeing up pharmacists for more clinical duties. Integration of electronic health records (EHR) with pharmacy automation systems ensures seamless data flow, enhancing the accuracy of medication dispensing and patient safety. Additionally, advancements in software and analytics enable better tracking of medication usage patterns, aiding in inventory management and forecasting. These technologies collectively enhance the overall efficiency of pharmacy operations, contributing to improved healthcare delivery.

What Role Does Pharmacy Automation Play in Healthcare Compliance?

Pharmacy automation plays a vital role in ensuring compliance with healthcare regulations and standards. Automated systems are designed to adhere to stringent guidelines for medication dispensing, labeling, and documentation, minimizing the risk of non-compliance. They provide accurate records of medication transactions, which are essential for audits and regulatory reporting.

Automation also supports adherence to drug safety protocols, such as monitoring for potential drug interactions and ensuring proper dosing. Furthermore, automated systems facilitate compliance with privacy laws, such as HIPAA, by securing patient information and limiting access to authorized personnel only. By ensuring adherence to regulatory requirements, pharmacy automation enhances the overall safety and reliability of pharmaceutical services.

Report Scope

Key Insights:

• Market Growth: Understand the significant growth trajectory of the Medication Dispensing Systems segment, which is expected to reach US$3.3 Billion by 2030 with a CAGR of a 6.6%. The Packaging & Labeling Systems segment is also set to grow at 9.2% CAGR over the analysis period.
• Regional Analysis: Gain insights into the U.S. market, valued at $1.8 Billion in 2024, and China, forecasted to grow at an impressive 6.4% CAGR to reach $1.5 Billion by 2030. Discover growth trends in other key regions, including Japan, Canada, Germany, and the Asia-Pacific.

Report Features:

• Comprehensive Market Data: Independent analysis of annual sales and market forecasts in US$ Million from 2024 to 2030.
• In-Depth Regional Analysis: Detailed insights into key markets, including the U.S., China, Japan, Canada, Europe, Asia-Pacific, Latin America, Middle East, and Africa.
• Company Profiles: Coverage of players such as ARxIUM, Inc., Baxter International, Inc., Becton, Dickinson and Company, Capsa Healthcare, Cerner Corporation and more.
• Complimentary Updates: Receive free report updates for one year to keep you informed of the latest market developments.

Segments:

• Medication Dispensing Systems
• Packaging & Labeling Systems
• Tabletop Counters
• Storage & Retrieval Systems
• Other Products

Tariff Impact Analysis: Key Insights for 2025

What’s Included in This Edition:

• Tariff-adjusted market forecasts by region and segment
• Analysis of cost and supply chain implications by sourcing and trade exposure
• Strategic insights into geographic shifts

Buyers receive a free July 2025 update with:

• Finalized tariff impacts and new trade agreement effects
• Updated projections reflecting global sourcing and cost shifts
• Expanded country-specific coverage across the industry

Key Attributes:

Key Topics Covered:

MARKET OVERVIEW

• Influencer Market Insights
• Tariff Impact on Global Supply Chain Patterns
• Pharmacy Automation – Global Key Competitors Percentage Market Share in 2024 (E)
• Competitive Market Presence – Strong/Active/Niche/Trivial for Players Worldwide in 2024 (E)
• Global Economic Update

MARKET TRENDS & DRIVERS

• Increasing Demand for Efficient and Error-Free Medication Dispensing Spurs Growth
• Technological Advancements in Robotics Strengthen Business Case for Automation
• Rising Focus on Reducing Medication Errors Drives Adoption of Automated Systems
• Growing Need for Workflow Optimization in Pharmacies Expands Market Opportunity
• Integration of IoT in Pharmacy Systems Generates Demand for Smart Solutions
• Development of Compact and User-Friendly Automation Solutions
• Growth in Retail and Hospital Pharmacies Sustains Demand
• Expansion of Centralized Pharmacy Models Boosts Market Growth
• Demand for Remote and Telepharmacy Services Drives Technology Adoption
• Increasing Focus on Cost Reduction and Efficiency in Pharmacies
• Rising Awareness and Adoption of Automated Medication Management Systems

FOCUS ON SELECT PLAYERS

• ARxIUM, Inc.
• Baxter International, Inc.
• Becton, Dickinson and Company
• Capsa Healthcare
• Cerner Corporation
• KUKA AG
• Omnicell, Inc.
• Parata Systems, LLC.
• RxSafe, LLC.
• ScriptPro LLC
• Talyst
• Tcgrx Pharmacy Workflow Solutions

For more information about this report visit https://www.researchandmarkets.com/r/1xpzbx

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

press@researchandmarkets.com

For E.S.T Office Hours Call 1-917-300-0470

For U.S./ CAN Toll Free Call 1-800-526-8630

For GMT Office Hours Call +353-1-416-8900

FDA Approves KEYTRUDA® (pembrolizumab) for PD-L1+ Resectable Locally Advanced Head & Neck Squamous Cell Carcinoma as Neoadjuvant Treatment, Continued as Adjuvant Treatment Combined With Radiotherapy With or Without Cisplatin Then as a Single Agent




FDA Approves KEYTRUDA® (pembrolizumab) for PD-L1+ Resectable Locally Advanced Head & Neck Squamous Cell Carcinoma as Neoadjuvant Treatment, Continued as Adjuvant Treatment Combined With Radiotherapy With or Without Cisplatin Then as a Single Agent

Approval introduces the first perioperative anti-PD-1 treatment regimen for adults with resectable locally advanced head and neck squamous cell carcinoma whose tumors express PD-L1 (CPS ≥1)

RAHWAY, N.J.–(BUSINESS WIRE)–$MRK #MRK–Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA^® (pembrolizumab), Merck’s anti-PD-1 therapy, for the treatment of adult patients with resectable locally advanced head and neck squamous cell carcinoma (HNSCC) whose tumors express PD-L1 (Combined Positive Score [CPS] ≥1) as determined by an FDA-approved test, as a single agent as neoadjuvant treatment, continued as adjuvant treatment in combination with radiotherapy (RT) with or without cisplatin and then as a single agent.

The approval is based on data from the pivotal Phase 3 KEYNOTE-689 trial, the results of which were presented at the American Association for Cancer Research (AACR) Annual Meeting on April 27, 2025. At the trial’s first pre-specified interim analysis, KEYTRUDA before surgery (neoadjuvant), then continued after surgery (adjuvant) in combination with standard of care (SOC), RT with or without cisplatin, followed by KEYTRUDA alone, reduced the risk of event-free survival (EFS) events (defined as disease recurrence, disease progression, or death) by 30% (HR=0.70 [95% CI, 0.55–0.89]; p=0.00140) in patients whose tumors expressed PD-L1 (CPS ≥1) compared to adjuvant SOC. Among the CPS ≥1 population, median EFS was 59.7 months (95% CI, 37.9-not reached) in the KEYTRUDA arm versus 29.6 months (95% CI, 19.5-41.9) in the SOC arm.

“The introduction of KEYTRUDA as a perioperative treatment option for certain patients with resectable locally advanced head and neck squamous cell carcinoma represents a potentially significant shift in how we manage this disease,” said Dr. Ravindra Uppaluri, the study’s overall principal investigator, director of Head and Neck Surgical Oncology, Brigham and Women’s Hospital and Dana-Farber Cancer Institute. “With this approval, we can now offer appropriate patients with resectable locally advanced head and neck squamous cell carcinoma a new treatment regimen that has been shown to reduce the risk of recurrence, progression, or death by 30%, compared with standard of care adjuvant chemoradiotherapy or radiotherapy alone.”

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue and can affect more than one body system simultaneously. Immune-mediated adverse reactions can occur at any time during or after treatment with KEYTRUDA, including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, dermatologic reactions, solid organ transplant rejection, other transplant (including corneal graft) rejection, and complications of allogeneic hematopoietic stem cell transplantation. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions. Early identification and management of immune-mediated adverse reactions are essential to ensure safe use of KEYTRUDA. Based on the severity of the adverse reaction, KEYTRUDA should be withheld or permanently discontinued and corticosteroids administered if appropriate. KEYTRUDA can also cause severe or life-threatening infusion-related reactions. Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. For more information, see “Selected Important Safety Information” below.

“As the first perioperative anti-PD-1 treatment option for appropriate patients with resectable locally advanced head and neck squamous cell carcinoma, this new treatment regimen has the potential to shift the treatment paradigm for patients and their families affected by this disease,” said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories. “Based on these trial results, KEYTRUDA as part of this regimen shows potential to change long-standing standards of care for treating certain patients with locally advanced HNSCC.”

This approval was reviewed under Project Orbis, an initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent review of oncology drugs among its international partners. Under Project Orbis, marketing authorization applications for KEYTRUDA based on the results of KEYNOTE-689 are under review by health authorities in Israel, Canada, Australia, Singapore, Brazil and Switzerland. Marketing Authorization Applications are also under review by regulatory authorities worldwide, including Europe and Japan.

In the U.S., KEYTRUDA is currently approved as monotherapy and in combination regimens for certain patients with recurrent or metastatic HNSCC as follows:

• KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC;
• KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test; and
• KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

Study design and additional data supporting the approval

KEYNOTE-689 is a randomized, multicenter, open-label, active-controlled Phase 3 trial (ClinicalTrials.gov, NCT03765918) evaluating KEYTRUDA as neoadjuvant treatment, continued as adjuvant treatment in combination with standard of care (SOC), radiotherapy (RT) with or without cisplatin, then as a single agent in patients with resectable locally advanced (Stage III-IVA) head and neck squamous cell carcinoma (HNSCC). Patients with active autoimmune disease that required systemic therapy within two years of treatment or a medical condition that required immunosuppression were ineligible. Randomization was stratified by primary tumor site (oropharynx/oral cavity vs. larynx vs. hypopharynx), tumor stage (III vs. IVA) and PD-L1 status (Tumor Proportion Score [TPS] ≥ 50% vs. TPS<50%). The study enrolled 714 patients who were randomized 1:1 to receive:

• Neoadjuvant KEYTRUDA 200 mg for 2 cycles prior to surgical resection. Within 6 weeks following surgery, 3 cycles of adjuvant KEYTRUDA 200 mg every 3 weeks in combination with RT with or without 3 cycles of cisplatin 100 mg/m² every 3 weeks. This was followed by KEYTRUDA 200 mg every 3 weeks for up to 12 cycles; or
• No neoadjuvant treatment prior to surgery. Within 6 weeks following surgery, adjuvant RT with or without 3 cycles of concurrent cisplatin 100 mg/m² every 3 weeks.

On both treatment arms, patients received cisplatin with adjuvant RT if high-risk pathological features (i.e., positive margins <1 mm or extranodal extension) were present at surgery.

Treatment with KEYTRUDA continued until disease progression by RECIST v1.1 per BICR during the neoadjuvant phase that precluded surgery, local or metastatic recurrence during the adjuvant phase, completion of treatment, or unacceptable toxicity. Assessment of tumor status was performed prior to surgery at Week 6 in the neoadjuvant phase. Following the start of the adjuvant phase, assessment of tumor status was performed 12 weeks after end of RT with or without cisplatin treatment and then every three months until the end of Year 3; then every six months thereafter up to the end of Year 5.

The trial was not designed to isolate the effect of KEYTRUDA in each phase (neoadjuvant or adjuvant) of treatment.

The major efficacy outcome measure was event-free survival (EFS) by BICR defined as the time from randomization to the first occurrence of any of the following events: progression of disease that precluded definitive surgery, local or distant disease progression or recurrence, or death due to any cause. Among the CPS ≥1 population, at the time of the first pre-specified interim analysis, the EFS hazard ratio (HR) was 0.70 (95% CI, 0.55-0.89; p=0.00140) and the number of events was 128 (37%) in the KEYTRUDA arm versus 156 (47%) in the SOC arm. In an exploratory subgroup analysis of patients with PD-L1-positive (Combined Positive Score [CPS] ≥1) hypopharyngeal tumors who were randomized (n=51), the EFS HR was 2.28 (95% CI, 0.79-6.56).

Additional efficacy outcome measures were major pathological response (mPR) as assessed by BIPR, and overall survival (OS). While OS results were not mature at this interim analysis, with 76% of pre-specified OS events in the CPS ≥1 population, no trend towards a detriment was observed.

The most common adverse reactions (≥ 20%) on the KEYTRUDA arm were stomatitis (48%), radiation skin injury (40%), weight loss (36%), fatigue (33%), dysphagia (29%), constipation (27%), hypothyroidism (26%), nausea (24%), rash (22%), dry mouth (22%), diarrhea (22%), and musculoskeletal pain (22%).

The median duration of exposure to KEYTRUDA in the neoadjuvant phase was 3.1 weeks (range: 1 day to 4.9 weeks). The median duration of exposure to KEYTRUDA in the adjuvant phase was 42 weeks (range: 1 day to 82 weeks).

Of the 361 patients who received KEYTRUDA as neoadjuvant treatment, 11% (n=38) did not receive surgery. Of the 351 patients randomized to SOC, 12% (n=43) did not receive surgery. In the adjuvant phase of the KEYTRUDA arm, 100 patients received KEYTRUDA and cisplatin with concurrent RT while 154 patients received KEYTRUDA alone with concurrent RT. In the SOC arm, 139 patients received cisplatin with concurrent RT while 136 patients received RT alone. For the KEYTRUDA arm, a total of 222 patients received single-agent KEYTRUDA following RT.

Of the 361 patients who received at least one dose of single agent KEYTRUDA as neoadjuvant treatment, 11% of patients experienced serious adverse reactions. Serious adverse reactions that occurred in more than one patient were pneumonia (1.4%), tumor hemorrhage (0.8%), dysphagia (0.6%), immune mediated hepatitis (0.6%), cellulitis (0.6%), and dyspnea (0.6%).

Of the 255 patients who received at least one dose of KEYTRUDA in the adjuvant phase, 38% experienced serious adverse reactions. The most frequent serious adverse reactions reported in ≥1% of KEYTRUDA-treated patients were pneumonia (2.7%), pyrexia (2.4%), stomatitis (2.4%), acute kidney injury (2.0%), pneumonitis (1.6%), COVID-19 (1.2%), death not otherwise specified (1.2%), diarrhea (1.2%), dysphagia (1.2%), gastrostomy tube site complication (1.2%), and immune-mediated hepatitis (1.2%).

About head and neck cancer

Head and neck cancer describes a number of different tumors that develop in or around the throat, larynx, nose, sinuses and mouth. In the U.S., it is estimated there will be approximately 72,680 new cases of head and neck cancer diagnosed and more than 16,680 deaths from the disease in 2025. These data include cancers of the oral cavity, pharynx and larynx. Most head and neck cancer is squamous cell carcinoma which begins in the flat, squamous cells that make up the thin mucosal lining of the head and neck. Locally advanced head and neck squamous cell carcinoma (LA-HNSCC) is cancer that has spread from where it started to nearby tissue or lymph nodes but has not yet spread to distant parts of the body. There are several factors that greatly increase the risk of developing head and neck cancer, including tobacco and alcohol use and human papillomavirus (HPV).

About KEYTRUDA^® (pembrolizumab) injection, 100 mg

KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD- L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,600 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient’s likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA^® (pembrolizumab) Indications in the U.S.

Head and Neck Squamous Cell Cancer

KEYTRUDA is indicated for the treatment of adult patients with resectable locally advanced head and neck squamous cell carcinoma (HNSCC) whose tumors express PD-L1 [Combined Positive Score (CPS) ≥1] as determined by an FDA-approved test, as a single agent as neoadjuvant treatment, continued as adjuvant treatment in combination with radiotherapy (RT) with or without cisplatin and then as a single agent.

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [Combined Positive Score (CPS) ≥1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.

See additional selected KEYTRUDA indications in the U.S. after the Selected Important Safety Information.

Selected Important Safety Information for KEYTRUDA

Severe and Fatal Immune-Mediated Adverse Reactions

KEYTRUDA is a monoclonal antibody that belongs to a class of drugs that bind to either the programmed death receptor-1 (PD-1) or the programmed death ligand 1 (PD-L1), blocking the PD-1/PD-L1 pathway, thereby removing inhibition of the immune response, potentially breaking peripheral tolerance and inducing immune-mediated adverse reactions. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue, can affect more than one body system simultaneously, and can occur at any time after starting treatment or after discontinuation of treatment. Important immune-mediated adverse reactions listed here may not include all possible severe and fatal immune-mediated adverse reactions.

Monitor patients closely for symptoms and signs that may be clinical manifestations of underlying immune-mediated adverse reactions. Early identification and management are essential to ensure safe use of anti–PD-1/PD-L1 treatments. Evaluate liver enzymes, creatinine, and thyroid function at baseline and periodically during treatment. For patients with TNBC treated with KEYTRUDA in the neoadjuvant setting, monitor blood cortisol at baseline, prior to surgery, and as clinically indicated. In cases of suspected immune-mediated adverse reactions, initiate appropriate workup to exclude alternative etiologies, including infection. Institute medical management promptly, including specialty consultation as appropriate.

Withhold or permanently discontinue KEYTRUDA depending on severity of the immune-mediated adverse reaction. In general, if KEYTRUDA requires interruption or discontinuation, administer systemic corticosteroid therapy (1 to 2 mg/kg/day prednisone or equivalent) until improvement to Grade 1 or less. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider administration of other systemic immunosuppressants in patients whose adverse reactions are not controlled with corticosteroid therapy.

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis. The incidence is higher in patients who have received prior thoracic radiation. Immune-mediated pneumonitis occurred in 3.4% (94/2799) of patients receiving KEYTRUDA, including fatal (0.1%), Grade 4 (0.3%), Grade 3 (0.9%), and Grade 2 (1.3%) reactions. Systemic corticosteroids were required in 67% (63/94) of patients. Pneumonitis led to permanent discontinuation of KEYTRUDA in 1.3% (36) and withholding in 0.9% (26) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Pneumonitis resolved in 59% of the 94 patients.

Pneumonitis occurred in 8% (31/389) of adult patients with cHL receiving KEYTRUDA as a single agent, including Grades 3-4 in 2.3% of patients. Patients received high-dose corticosteroids for a median duration of 10 days (range: 2 days to 53 months). Pneumonitis rates were similar in patients with and without prior thoracic radiation. Pneumonitis led to discontinuation of KEYTRUDA in 5.4% (21) of patients. Of the patients who developed pneumonitis, 42% interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.

Pneumonitis occurred in 7% (41/580) of adult patients with resected NSCLC who received KEYTRUDA as a single agent for adjuvant treatment of NSCLC, including fatal (0.2%), Grade 4 (0.3%), and Grade 3 (1%) adverse reactions. Patients received high-dose corticosteroids for a median duration of 10 days (range: 1 day to 2.3 months). Pneumonitis led to discontinuation of KEYTRUDA in 26 (4.5%) of patients. Of the patients who developed pneumonitis, 54% interrupted KEYTRUDA, 63% discontinued KEYTRUDA, and 71% had resolution.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis, which may present with diarrhea. Cytomegalovirus infection/reactivation has been reported in patients with corticosteroid-refractory immune-mediated colitis. In cases of corticosteroid-refractory colitis, consider repeating infectious workup to exclude alternative etiologies. Immune-mediated colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (1.1%), and Grade 2 (0.4%) reactions. Systemic corticosteroids were required in 69% (33/48); additional immunosuppressant therapy was required in 4.2% of patients. Colitis led to permanent discontinuation of KEYTRUDA in 0.5% (15) and withholding in 0.5% (13) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, 23% had recurrence. Colitis resolved in 85% of the 48 patients.

Hepatotoxicity and Immune-Mediated Hepatitis

KEYTRUDA as a Single Agent

KEYTRUDA can cause immune-mediated hepatitis. Immune-mediated hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.4%), and Grade 2 (0.1%) reactions. Systemic corticosteroids were required in 68% (13/19) of patients; additional immunosuppressant therapy was required in 11% of patients. Hepatitis led to permanent discontinuation of KEYTRUDA in 0.2% (6) and withholding in 0.3% (9) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement; of these, none had recurrence. Hepatitis resolved in 79% of the 19 patients.

KEYTRUDA With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider monitoring more frequently as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased alanine aminotransferase (ALT) (20%) and increased aspartate aminotransferase (AST) (13%) were seen at a higher frequency compared to KEYTRUDA alone. Fifty-nine percent of the patients with increased ALT received systemic corticosteroids. In patients with ALT ≥3 times upper limit of normal (ULN) (Grades 2-4, n=116), ALT resolved to Grades 0-1 in 94%. Among the 92 patients who were rechallenged with either KEYTRUDA (n=3) or axitinib (n=34) administered as a single agent or with both (n=55), recurrence of ALT ≥3 times ULN was observed in 1 patient receiving KEYTRUDA, 16 patients receiving axitinib, and 24 patients receiving both. All patients with a recurrence of ALT ≥3 ULN subsequently recovered from the event.

Immune-Mediated Endocrinopathies

Adrenal Insufficiency

KEYTRUDA can cause primary or secondary adrenal insufficiency. For Grade 2 or higher, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold KEYTRUDA depending on severity. Adrenal insufficiency occurred in 0.8% (22/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.3%) reactions. Systemic corticosteroids were required in 77% (17/22) of patients; of these, the majority remained on systemic corticosteroids. Adrenal insufficiency led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.3% (8) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Hypophysitis

KEYTRUDA can cause immune-mediated hypophysitis. Hypophysitis can present with acute symptoms associated with mass effect such as headache, photophobia, or visual field defects. Hypophysitis can cause hypopituitarism. Initiate hormone replacement as indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Hypophysitis occurred in 0.6% (17/2799) of patients receiving KEYTRUDA, including Grade 4 (<0.1%), Grade 3 (0.3%), and Grade 2 (0.2%) reactions. Systemic corticosteroids were required in 94% (16/17) of patients; of these, the majority remained on systemic corticosteroids. Hypophysitis led to permanent discontinuation of KEYTRUDA in 0.1% (4) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement.

Thyroid Disorders

KEYTRUDA can cause immune-mediated thyroid disorders. Thyroiditis can present with or without endocrinopathy. Hypothyroidism can follow hyperthyroidism. Initiate hormone replacement for hypothyroidism or institute medical management of hyperthyroidism as clinically indicated. Withhold or permanently discontinue KEYTRUDA depending on severity. Thyroiditis occurred in 0.6% (16/2799) of patients receiving KEYTRUDA, including Grade 2 (0.3%). None discontinued, but KEYTRUDA was withheld in <0.1% (1) of patients.

Hyperthyroidism occurred in 3.4% (96/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (0.8%). It led to permanent discontinuation of KEYTRUDA in <0.1% (2) and withholding in 0.3% (7) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. Hypothyroidism occurred in 8% (237/2799) of patients receiving KEYTRUDA, including Grade 3 (0.1%) and Grade 2 (6.2%). It led to permanent discontinuation of KEYTRUDA in <0.1% (1) and withholding in 0.5% (14) of patients. All patients who were withheld reinitiated KEYTRUDA after symptom improvement. The majority of patients with hypothyroidism required long-term thyroid hormone replacement. The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC, occurring in 16% of patients receiving KEYTRUDA as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. The incidence of new or worsening hypothyroidism was higher in 389 adult patients with cHL (17%) receiving KEYTRUDA as a single agent, including Grade 1 (6.

Contacts

Media Contacts:

Julie Cunningham

(617) 519-6264

Sienna Choi

(908) 873-4311

Investor Contacts:

Peter Dannenbaum

(732) 594-1579

Steven Graziano

(732) 594-1583

Read full story here

Ethris Signs Strategic Collaboration with Thermo Fisher Scientific to Provide Access to mRNA Technology Platforms




Ethris Signs Strategic Collaboration with Thermo Fisher Scientific to Provide Access to mRNA Technology Platforms

Partnership further validates Ethris’ novel technology platform based on stabilized non-immunogenic mRNA, which overcomes the innate instability and immunity of mRNA

MUNICH–(BUSINESS WIRE)–Ethris GmbH, a clinical-stage biotechnology company pioneering next-generation RNA therapeutics and vaccines, today announced a strategic collaboration with Thermo Fisher Scientific, the world leader in serving science, to provide a fully integrated mRNA solution to biopharmaceutical developers.

The collaboration brings together Ethris’ comprehensive mRNA technology platforms, including its proprietary Stabilized Non-Immunogenic mRNA (SNIM^® RNA), minimal UTR and mRNA manufacturing technologies with Thermo Fisher’s industry-leading end-to-end GMP-compliant manufacturing capabilities. Broadening global access to Ethris’ comprehensive suite of mRNA technology platforms, the partners will facilitate the ability of biopharmaceutical developers to rapidly advance candidate mRNA medicines from research to clinical proof-of-concept.

“Thermo Fisher Scientific has a long-standing track record of supporting the development of advanced therapies,” said Dr. Carsten Rudolph, CEO of Ethris. “By combining our clinically validated mRNA platforms with Thermo Fisher’s manufacturing expertise, we are creating a powerful offering for biopharma partners. This collaboration addresses the increasing demand for scalable, high-quality mRNA technologies and enables us to serve a broader set of collaborators worldwide.”

The collaboration will also support the continued optimization of Ethris’ platform technologies, which have already demonstrated positive pharmacodynamic effects, safety and targeted engagement in Phase 1 topline data of its lead candidate, ETH47. Designed for local administration via nasal spray to address the upstream trigger of asthma exacerbations, ETH47 showed dose-dependent, localized production of the encoded protein, interferon lambda at the site of administration, with no systemic bioavailability. The study confirmed that ETH47 activated downstream signaling, demonstrating the expressed protein’s functional activity. Given its non-immunogenic nature, SNIM® RNAs can be administered repeatedly, leading to sustained production of therapeutically active proteins within the human body. Eligible for multiple routes of administration, the technology can replace or augment missing or non-functional proteins that cause a disease, introduce new proteins to modulate the course of the disease or its symptoms and be used to develop vaccines.

“Thermo Fisher is committed to supporting the rapid translation of breakthrough therapies into clinical application,” said Ben Castro, Vice President and General Manager, Large Molecule, Drug Substance, Thermo Fisher Scientific. “By partnering with Ethris, we are expanding our services to include cutting-edge mRNA manufacturing, empowering our customers to bring life-changing treatments to patients faster and more efficiently.”

Ethris’ SNIM® RNA platform sets new standards for mRNA therapeutics by enabling safe, efficacious and stable drug products as well as scalable manufacturing, expanding the potential of mRNA medicines.

About Ethris

Ethris, a clinical-stage biotechnology company, has paved a new path from genes to therapeutic proteins, using its proprietary RNA and lipidoid nanoparticle technology platform to discover, design and develop innovative therapies. With more than a decade as an mRNA pioneer, Ethris is a global leader in delivering stabilized mRNAs directly to the respiratory system via optimised formulation and nebulisation technologies. The company is rapidly advancing its mRNA pipeline of immuno-modulation, protein replacement therapies, and differentiated vaccines, with the ultimate goal of improving patients’ lives.

For more information, visit www.ethris.com.

Contacts

Ethris contact:
Dr. Philipp Schreppel

+49 89 244 153 042

schreppel@ethris.com

City of Hope Developed a Foundational Map of Tumor Cells for Personalized Brain Cancer Treatments




City of Hope Developed a Foundational Map of Tumor Cells for Personalized Brain Cancer Treatments

Scientists developed a new framework to improve the ability of doctors to use precision medicine to identify therapeutic targets and potentially spotlight ways to prevent disease recurrence

LOS ANGELES–(BUSINESS WIRE)–City of Hope^®, one of the largest and most advanced cancer research and treatment organizations in the U.S. with its National Medical Center named top 5 in the nation for cancer by U.S. News & World Report, co-led the first study to demonstrate that characterizing genetic material near chromosomes forecasts how mutated, cancer-causing genes reengineer DNA and alter the tumor microenvironment. The leading-edge brain cancer research provides foundational knowledge that one day will improve the practice of precision medicine and allow oncologists to deliver more personalized therapies to cancer patients.

Tiny DNA molecules outside of chromosomes were once disregarded, but in the past decade research has revealed that these circles called extrachromosomal DNA, or ecDNA, fuel cancer by breaking the laws of biology.

“Our study offers new insights into the interplay between different ecDNA. Importantly, when there is a prevalence of ecDNA and cancer-causing ingredients like the EGFR protein or tumor protein p53, the tumor microenvironment becomes hypoxic. It falls into a state of reduced oxygen, which has been linked to cancer progression, resistance to therapy and poor clinical outcomes,” said David Craig, Ph.D., professor and chair of the Department of Integrative Translational Sciences at City of Hope and co-corresponding author of a study published today in Nature Communications.

Spatial transcriptomics (measuring and mapping of DNA activity) combined with genomic data can help identify groups of cells within a tumor that share a common ancestor but have acquired additional mutations. How they are distributed spatially informs the understanding of tumor evolution. The new translational science data underpins City of Hope’s precision medicine research, which applies innovation to create better outcomes with fewer side effects for more patients. City of Hope aspires to cure more cancer patients.

City of Hope researchers led a team that performed bulk RNA sequencing, tumor/normal DNA sequencing and spatial transcriptomics in a small sample of gliomas — tumors that develop in the brain or spinal cord. Through varied experiments and validation cohorts, they were able to identify common and distinct characteristics of the tumor microenvironment, developing an integrated analysis framework that can be leveraged by others.

“While our paper solely evaluated different types of brain cancers, the spatial transcriptomic principles and genome sequencing techniques we outlined will one day enable physicians to provide more personalized therapies for cancer patients,” said Gabriel Zada, M.D., a neurosurgeon with Keck Medicine of USC, professor of neurological surgery and physiology and neuroscience at the Keck School of Medicine of USC, co-director of the USC Brain Tumor Center and co-corresponding study author. “Cancer and its treatment is not one-size-fits-all. Understanding the molecular activity of ecDNA near inheritable and non-inheritable cells provides deep insights into potential therapeutic targets and risk of cancer recurrence.”

The researchers demonstrated that ecDNA drives rapid cancer cell (oncogene) proliferation outside of chromosomes, the thread-like structures inside the cell nucleus that houses DNA and RNA. EcDNA contributes to the development of gliomas, genetic instability and distinct tumor cell populations within a single tumor, making cancer more difficult to eliminate.

The dynamic nature of ecDNA may be what enables cancer cells to adapt and reprogram their genomes, driving tumor progression in response to changes in their microenvironment, including changes resulting from therapies.

“We have now demonstrated how cancer cells dynamically reprogram their own genome to control and respond to the tumor microenvironment. By uncovering these mechanisms, we are paving the way for more precise and effective treatments tailored to the unique biology of each patient,” Dr. Craig said.

The Nature Communications study entitled “Resolving Subclonal Genomic Heterogeneity in Gliomas by Integrating Spatial Transcriptomics with Loss of Heterozygosity and Extrachromosomal DNA Analysis” was supported by the National Center for Advancing Translational Science of the U.S. National Institutes of Health (KL2TR001854).

About City of Hope

City of Hope’s mission is to make hope a reality for all touched by cancer and diabetes. Founded in 1913, City of Hope has grown into one of the largest and most advanced cancer research and treatment organizations in the U.S., and one of the leading research centers for diabetes and other life-threatening illnesses. City of Hope research has been the basis for numerous breakthrough cancer medicines, as well as human synthetic insulin and monoclonal antibodies. With an independent, National Cancer Institute-designated comprehensive cancer center that is ranked top 5 in the nation for cancer care by U.S. News & World Report at its core, City of Hope’s uniquely integrated model spans cancer care, research and development, academics and training, and a broad philanthropy program that powers its work. City of Hope’s growing national system includes its Los Angeles campus, a network of clinical care locations across Southern California, a new cancer center in Orange County, California, and cancer treatment centers and outpatient facilities in the Atlanta, Chicago and Phoenix areas. City of Hope’s affiliated group of organizations includes Translational Genomics Research Institute and AccessHope^TM. For more information about City of Hope, follow us on Facebook, X, YouTube, Instagram and LinkedIn.

Contacts

Letisia Marquez

626-476-7593

lemarquez@coh.org

MaaT Pharma Presents Updated Positive Data in Early Access Program for Xervyteg® at the EHA Congress Validating High Efficacy Observed in Pivotal ARES Study in Acute Graft-versus-Host Disease




MaaT Pharma Presents Updated Positive Data in Early Access Program for Xervyteg® at the EHA Congress Validating High Efficacy Observed in Pivotal ARES Study in Acute Graft-versus-Host Disease

• Oral presentation highlights updated data in Early Access Program (EAP) for 173 patients with acute Graft-vs-Host Disease (aGvHD) treated with Xervyteg^®
• Independent dataset from EAP reinforces the findings from the pivotal ARES trial and has also been included in the EMA Marketing Authorization Application submitted on June 2^nd, 2025
• EAP for Xervyteg^® in aGvHD is currently running in 11 countries* providing expanded access to patients with high unmet medical needs

LYON, France–(BUSINESS WIRE)–$MAAT #Cancer–Regulatory News:

MaaT Pharma (EURONEXT: MAAT – the “Company”), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem Therapies^TM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today announced that Professor Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University, will present updated data for Xervyteg^® (MaaT013) in treating acute Graft-versus-Host Disease (aGvHD) under the Early Access Program (EAP) at the European Hematology Association (EHA) Annual Congress 2025. This independent EAP dataset further supports the efficacy and safety profile of Xervyteg^® previously shown in the pivotal ARES trial. It also confirms the breakthrough potential of Xervyteg^® for aGvHD patients with limited treatment options and it also serves as supportive data within the Marketing Authorization Application (MAA) recently submitted to the European Medicines Agency (EMA).

Key highlights:

• aGvHD is a major cause of morbidity and mortality following allogeneic hematopoietic stem cell transplantation. The patients (N=173) treated in EAP previously failed 1 to 6 aGvHD systemic treatment lines and most had grade III (49%) or IV (38%) aGvHD. The real-world data presented underscores the favorable safety profile of Xervyteg^® the strong and durable responses, translating into increased overall survival:

• Gastrointestinal Overall Response Rate (GI-ORR) of 53% at D28, with Complete Response (CR) observed in 30% of patients; all-organ Overall Response Rate (ORR) was 50% with 26% CR.

• Response is maintained at D56 indicating a long-term disease control with a GI-ORR of 47% and an ORR considering all organs of 46%.

• Overall Survival (OS) in all patients was 55% at 6 months, 48% at 12 months, 44% at 24 months.

• Xervyteg^® displayed a good overall safety profile in the EAP population.

• OS was significantly higher in patients who responded to Xervyteg^® (MaaT013) compared to non-responders (69% versus 25% at 12 months, and 61% versus 25% at 24 months).

• Median survival in all patients was 312 days. In responder patients, median survival was 834 days vs 69 days in non-responders.

A subset of patients (n=70) failing both steroid resistant (SR) and ruxolitinib resistant (RR) and thus resembling the cohort enrolled in the pivotal Phase 3 ARES trial (NCT04769895), exhibited a significant and consistent efficacy profile:

• At both Day 28 and Day 56, Xervyteg^® demonstrated durable efficacy in SR/RR aGvHD patients, with GI-ORRs of 57% and Complete Response (CR) observed in 44% of patients at D28 and 51% at D56. All-organs ORR was 54% with 41% CR at D28, and 55% with 48% CR at D56.

• OS was 55% at 6 months, 51% at 12 months, 40% at 24 months.

• OS was significantly higher in patients who responded to Xervyteg^® compared to non-responders (77% versus 14% at 12 months, and 59% versus 14% at 24 months).

• Median survival in all 70 patients was 445 days. In responder patients, median survival was 834 days vs 53 days in non-responders.

The complete data may be found here.

“The consistency between the real-world Early Access Program data and our pivotal ARES trial underscores Xervyteg^®’s clinical benefit for patients with severe, treatment-resistant aGvHD,” said Dr. Gianfranco Pittari, PhD, Chief Medical Officer at MaaT Pharma. “This is particularly meaningful for clinicians and patients, as it confirms the potential of microbiome therapies to deliver long-term survival benefits in a population with historically poor outcomes.”

In comparison, historical data from Abedin et al. 2021 demonstrated that in a similar population of patients, i.e. third-line aGvHD patients receiving additional treatment after ruxolitinib failure, the median survival was only 28 days.

“Among patients who responded by Day 28, the majority achieved a complete resolution of aGvHD symptoms — a strong predictor of sustained disease control over time. The overall safety profile is favorable in this high-risk patient population,” outlines Professor Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University.

Details of the Oral Presentation:

• Title: Pooled Fecal Allogeneic Microbiotherapy for Refractory Gastrointestinal Acute Graft-Versus-Host Disease: Results from the Early Access Program in Europe
• Abstract number: S260
• Presenting Author: Mohamad Mohty, Professor of Hematology and Head of the Hematology and Cellular Therapy Department at Saint-Antoine Hospital and Sorbonne University
• Session title: s424 Stem cell transplantation – Session 2
• Date & Time: 13/06/2025 (17:00 – 17:15 CEST) – Brown Hall 3

MaaT Pharma also presented a poster on the design of its ongoing Phase 2b trial (PHOEBUS) evaluating MaaT033 to enhance overall survival in allo-HSCT. This international, multi-center trial (NCT05762211) is the largest randomized controlled study to date of a microbiome-based therapy in oncology, enrolling up to 387 patients across 60 sites.

—

About MaaT Pharma

MaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021.

Forward-looking Statements

All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim”, “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements.

* France, Austria, Belgium, Canada, Germany, Italy, Lebanon, Spain, Sweden, Switzerland & USA

Contacts

MaaT Pharma – Investor Relations

Guilhaume DEBROAS, Ph.D.

Head of Investor Relations

+33 6 16 48 92 50

invest@maat-pharma.com

Rx Communications Group – U.S. Investor Relations

Michael Miller

Managing Director

+1-917-633-6086

mmiller@rxir.com

MaaT Pharma – Media Relations

Pauline RICHAUD

Senior PR & Corporate Communications Manager

+33 6 14 06 45 92

media@maat-pharma.com

Catalytic Agency – U.S. Media Relations

Heather Shea

Media relations for MaaT Pharma

+1 617-286-2013

heather.shea@catalyticagency.com

Innate Pharma Highlights Preclinical Antitumor Activity of IPH6501 in Diffuse Large B-Cell Lymphoma and Follicular Lymphoma at the 2025 European Hematology Association (EHA) Congress




Innate Pharma Highlights Preclinical Antitumor Activity of IPH6501 in Diffuse Large B-Cell Lymphoma and Follicular Lymphoma at the 2025 European Hematology Association (EHA) Congress

• Preclinical data from IPH6501, Innate’s proprietary ANKET^® targeting CD20, demonstrating potent antitumor activity in vitro on patient-derived samples from Diffuse Large B-Cell Lymphoma (DLBCL) and Follicular Lymphoma (FL), will be presented
• Preclinical in vivo models support enhanced antitumor efficacy for the combination of IPH6501 with R-CHOP, the standard of care in untreated DLBCL and FL patients
• IPH6501 is currently under investigation in a Phase 1/2 clinical study in relapsed and/or refractory (R/R) CD20+ B-cell non-Hodgkin lymphoma

MARSEILLE, France–(BUSINESS WIRE)–#ANKET–Regulatory News:

Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced the presentation of preclinical data for IPH6501, its proprietary ANKET^® targeting CD20 currently under investigation in a Phase 1/2 study in relapsed and/or refractory (R/R) B-cell non-Hodgkin lymphoma (B-NHL) (NCT06088654), at the European Hematology Association (EHA) Congress 2025, taking place June 12-15 in Milan, Italy.

R-CHOP is an established standard of care for treatment-naïve patients with diffuse large B cell lymphoma (DLBCL) and follicular lymphoma (FL), two subtypes of B-NHL. The treatment landscape continues to evolve with novel approaches including T cell engagers, antibody-drug conjugates, their combination with standard of care therapies, and CAR-T cells. Yet, there remains an unmet medical need for patients ineligible, refractory to, or relapsing from these therapies.

In preclinical in vitro models, IPH6501 demonstrated potent NK cell proliferation and tumor cell killing activity in DLBCL and FL patient samples, supporting its therapeutic potential in these indications. In preclinical in vivo xenografted mouse tumor models, IPH6501 showed strong activity in a rituximab-resistant model — even in the presence of rituximab highlighting their combination potential. Additionally, when combined with R-CHOP, IPH6501 showed enhanced antitumor efficacy, leading to tumor eradication that was maintained after treatment discontinuation. These findings underscore the potential of IPH6501 in improving standard-of-care R-CHOP treatment in previously untreated DLBCL and FL patients and support further evaluation of additional combination strategies in B-NHL.

“Patients with R/R DLBCL and FL continue to face significant unmet medical needs. The encouraging activity observed in preclinical models with IPH6501, including in rituximab-resistant settings, suggests its potential to offer new treatment options for B-cell non-Hodgkin lymphoma. The enhanced antitumor activity in combination with R-CHOP could support further investigation in untreated patients. These findings support further clinical development aimed at improving outcomes in this challenging patient population,” commented Dr Sonia Quaratino, Chief Medical Officer of Innate Pharma.

The poster will be available in the publication section of Innate Pharma’s website.

Abstract details

Antitumor characterization of IPH6501, a novel il2v-armed tetraspecific NK cell engager targeting CD20 B cells, in DLBCL and FL patient samples, and in preclinical combination with R-CHOP
Abstract Code: PS2004

Session: Poster session 2

Session Date/Time: Saturday, June 14, 2025, 18:30 – 19:30 CEST

About ANKET^®

ANKET^® (Antibody-based NK cell Engager Therapeutics) is Innate’s proprietary platform for developing next-generation, multi-specific natural killer (NK) cell engagers to treat certain types of cancer. This versatile, fit-for-purpose technology is creating an entirely new class of molecules to induce synthetic immunity against cancer.

About IPH6501

IPH6501 is the first Antibody-based NK cell Engager Therapeutic to co-engage activating receptors on NK cells (NKp46 and CD16), IL-2R (but not the alpha subunit) through a variant of human IL-2, and a tumor antigen (CD20) via a single molecule. This unique multispecific design provides targeted proliferation and activation signals to NK cells, promoting their cytotoxic activity against CD20 expressing malignant cells.

IPH6501 has shown better antitumor efficacy compared to approved benchmark antibodies in preclinical tumor models (Demaria, EHA 2023, Carrette, SITC 2024, Demaria et al, Science Immunology 2024).

IPH6501 is currently being evaluated in a Phase 1/2 multicenter trial (NCT06088654), investigating the safety and tolerability of IPH6501 in patients with relapsed and/or refractory CD20-expressing B-cell Non-Hodgkin’s Lymphoma.

About Innate Pharma

Innate Pharma S.A. is a global, clinical-stage biotechnology company developing immunotherapies for cancer patients. Its innovative approach aims to harness the innate immune system through three therapeutic approaches: multi-specific NK Cell Engagers via its ANKET^® (Antibody-based NK cell Engager Therapeutics) proprietary platform and Antibody Drug Conjugates (ADC) and monoclonal antibodies (mAbs).

Innate’s portfolio includes several ANKET^® drug candidates to address multiple tumor types as well as IPH4502, a differentiated ADC in development in solid tumors. In addition, anti-KIR3DL2 mAb lacutamab is developed in advanced form of cutaneous T cell lymphomas and peripheral T cell lymphomas, and anti-NKG2A mAb monalizumab is developed with AstraZeneca in non-small cell lung cancer.

Innate Pharma is a trusted partner to biopharmaceutical companies such as Sanofi and AstraZeneca, as well as leading research institutions, to accelerate innovation, research and development for the benefit of patients.

Headquartered in Marseille, France with a US office in Rockville, MD, Innate Pharma is listed on Euronext Paris and Nasdaq in the US.

Learn more about Innate Pharma at www.innate-pharma.com. Follow us on LinkedIn and X.

Information about Innate Pharma shares

Disclaimer on forward-looking information and risk factors

This press release contains certain forward-looking statements, including those within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. The use of certain words, including “anticipate,” “believe,” “can,” “could,” “estimate,” “expect,” “may,” “might,” “potential,” “intend,” “should,” “will,” or the negative of these and similar expressions, is intended to identify forward-looking statements. Although the Company believes its expectations are based on reasonable assumptions, these forward-looking statements are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those anticipated. These risks and uncertainties include, among other things, the uncertainties inherent in research and development, including related to safety, progression of and results from its ongoing and planned clinical trials and preclinical studies, review and approvals by regulatory authorities of its product candidates, the Company’s reliance on third parties to manufacture its product candidates, the Company’s commercialization efforts and the Company’s continued ability to raise capital to fund its development. For an additional discussion of risks and uncertainties, which could cause the Company’s actual results, financial condition, performance or achievements to differ from those contained in the forward-looking statements, please refer to the Risk Factors (“Facteurs de Risque”) section of the Universal Registration Document filed with the French Financial Markets Authority (“AMF”), which is available on the AMF website http://www.amf-france.org or on Innate Pharma’s website, and public filings and reports filed with the U.S. Securities and Exchange Commission (“SEC”), including the Company’s Annual Report on Form 20-F for the year ended December 31, 2024, and subsequent filings and reports filed with the AMF or SEC, or otherwise made public by the Company. References to the Company’s website and the AMF website are included for information only and the content contained therein, or that can be accessed through them, are not incorporated by reference into, and do not constitute a part of, this press release.

In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by the Company or any other person that the Company will achieve its objectives and plans in any specified time frame or at all. The Company undertakes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

This press release and the information contained herein do not constitute an offer to sell or a solicitation of an offer to buy or subscribe to shares in Innate Pharma in any country.

Contacts

For additional information, please contact:

Investors

Innate Pharma
Henry Wheeler

Tel.: +33 (0)4 84 90 32 88

Henry.wheeler@innate-pharma.fr

Media Relations

NewCap
Arthur Rouillé

Tel.: +33 (0)1 44 71 00 15

innate@newcap.eu