Genethon Presents Two Year Consolidated Results of Its Gene Therapy Trial for Duchenne Muscular Dystrophy: Maintenance of Motor Functions and Significant, Sustained Reduction in CPK Levels in Patients Treated at the Effective Dose at ASGCT 2025

Genethon Presents Two Year Consolidated Results of Its Gene Therapy Trial for Duchenne Muscular Dystrophy: Maintenance of Motor Functions and Significant, Sustained Reduction in CPK Levels in Patients Treated at the Effective Dose at ASGCT 2025




Genethon Presents Two Year Consolidated Results of Its Gene Therapy Trial for Duchenne Muscular Dystrophy: Maintenance of Motor Functions and Significant, Sustained Reduction in CPK Levels in Patients Treated at the Effective Dose at ASGCT 2025

  • GNT0004 product confirms its clinical efficacy with stabilization of motor functions in patients treated at the effective dose for up to 2 years.
  • Sustained significant reduction in levels of creatine phosphokinase (CPK), a biomarker of muscle damage, with an average decrease of over 75% at 18 months in the 3 patients treated at the effective dose.
  • Pivotal phase planned for mid-2025 in Europe and the United States.

PARIS–(BUSINESS WIRE)–Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, May 13 – 17, 2025. Five patients, aged 6 to 10 years, were treated, 2 at the first dose level and 3 at the second dose level (3×10¹³ vg/kg). The initial part of the clinical trial aimed at selecting the optimal dose (dose escalation phase), evaluating the tolerance and preliminary efficacy of the treatment and determined the effective dose for the pivotal phase of the GNT-016-MDYF trial, which is expected to start in mid-2025 (3×10¹³ vg/kg).


Genethon CEO Frederic Revah observed, “The results of our gene therapy GNT0004 are very positive in patients treated at the dose of 3×10¹³ vg/kg, both in terms of microdystrophin expression and clinical efficacy criteria. Besides these results, the advantage of our product lies in the selected dose for the pivotal phase, which is lower than those used in other gene therapy trials for Duchenne Muscular Dystrophy. We are currently preparing the pivotal phase that we will conduct in Europe and the US.”

Safety, efficacy, and pharmacodynamic results show good tolerance of GNT0004 associated with transient immunological prophylactic treatment, as well as efficacy data in terms of microdystrophin expression, CPK reduction, and clinical criteria (NSAA, timed tests). Patients treated at the effective dose show prolonged improvement or stabilization of motor functions and significant persistent reduction in creatine kinase (CPK) levels, a key marker of muscle damage.

One-year post-treatment, the comparison of the three patients treated at the effective dose with a group of 34 untreated patients, matched by age and followed in the same centers and by the same practitioners, shows a difference of +4.7 points in the score obtained using the internationally recognized clinical evaluation scale NSAA between treated and untreated patients.

At 24 months post-treatment, key observations include:

  • For the 2 patients who reached 2 years post-treatment out of the 3 treated at the effective dose, the trial shows stabilization of motor functions measured by the NSAA scale , while untreated patients from the parallel natural history study showed a continuous and significant average decline in NSAA. For one treated patient, the observed improvement allowed reaching the maximum score of 34 at 12 months, confirmed at 24 months post-treatment.
  • Stabilization of CPK reduction between 50% and 87% on average: >75% at 18 months post-treatment (data from the 3 patients treated at the effective dose), and persistent (up to 24 months follow-up for the first two patients treated at this dose).
  • The reassuring safety profile of the gene therapy drug is confirmed two years after injection, without the occurrence of serious adverse effects at the selected dose, which is notably lower than that used for other gene therapy products under development for Duchenne Muscular Dystrophy.

About GNT0004 and the trial

The GNT0004 gene therapy is composed of an AAV8 (adeno-associated virus) vector and the optimized hMD1 transgene, a shortened but functional version of the gene encoding dystrophin, the protein deficient in people with DMD. This vector is designed to be expressed in muscle tissue and also in the heart, thanks to a tissue-specific Spc5-12 promoter sequence. GNT0004 is administered by a single intravenous injection. It was developed by Genethon, in partnership with the teams of Prof. Dickson (University of London, Royal Holloway) and the Institut de Myologie (Paris). The trial, sponsored by Genethon, combines Phases 1/2/3, a dose-escalation phase followed by a pivotal phase at the dose finally chosen. The trial is being carried out in France and the UK and includes boys aged 6 to 10 with DMD who have retained their ability to walk.

About Duchenne muscular dystrophy

DMD is a rare, progressive genetic disease affecting all the body’s muscles, and mainly boys (1 in 5000). It is due to abnormalities in the gene responsible for producing dystrophin, a structural protein essential for the stability of muscle fiber membranes and their metabolism. The absence of dystrophin leads to progressive degeneration of skeletal and cardiac muscles, loss of walking and respiratory capacity, cardiomyopathy and death between the ages of 20 and 40.

About Genethon

A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a nonprofit organization created by the AFM-Téléthon. The first gene therapy to treat spinal muscular atrophy, incorporating technologies developed at Genethon, is marketed worldwide. With over 240 scientists and professionals, Genethon pursues its goal of developing innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen products from Genethon’s R&D or collaborations are in clinical trials for diseases of the liver, blood, immune system, muscles and eyes. A further seven products could enter clinical trials in the next five years. To find out more visit: http://www.genethon.com/.

Genethon made five oral presentations and seven poster presentations by 12 researchers, scientists, engineers and medical experts at ASGCT 2025. Learn more https://urlr.me/kTPBtW

Contacts

Press contact:
Stephanie Bardon

communication@genethon.fr
06.45.15.95.87

Jason Hancock Joins BioRewards as Head of IT and Digital Operations, Advancing Tech-Driven Plasma Supply Experience

Jason Hancock Joins BioRewards as Head of IT and Digital Operations, Advancing Tech-Driven Plasma Supply Experience




Jason Hancock Joins BioRewards as Head of IT and Digital Operations, Advancing Tech-Driven Plasma Supply Experience

SCOTTSDALE, Ariz.–(BUSINESS WIRE)–#BiotechStartupBioRewards Plasma Services (“BioRewards”), a next-generation U.S.-based plasma collection network, is pleased to announce that Jason Hancock has joined the company as Head of IT and Digital Operations. Hancock brings over two decades of healthcare technology leadership, most recently serving as Senior Director of IT at one of the world’s top four global plasma fractionators.


Hancock’s appointment reflects BioRewards’ rapid evolution from a strong startup into a technology-forward partner of choice for global fractionators seeking dependable plasma supply. In his role, Hancock will lead the design and implementation of BioRewards’ digital infrastructure— overseeing platform architecture, systems integration, and the application of data analytics to optimize the donor experience.

“BioRewards exists to help ensure the global plasma-derived medicines supply chain remains strong and resilient,” says Charles Auger, CEO of BioRewards. “Jason’s leadership will help us scale efficiently while deepening our commitment to innovation and donor-centric operations.”

As BioRewards advances its plan to open more than 20 plasma centers across the U.S., Hancock’s expertise will support the company’s goals of operational excellence, regulatory compliance, and technology-enabled donor engagement.

“Jason’s background with a top-tier global plasma fractionator gives BioRewards a major advantage,” added Chris Barber, Chief Operating Officer of BioRewards. “He will lead the implementation of technologies that enhance both quality and cost-efficiency across our national footprint.”

This leadership addition follows BioRewards’ recent formal launch and more than $100 million in strategic funding from Alpha-1 Plasma Holdings. The company is uniquely positioned to help fractionators meet rising global demand for immunoglobulins and other PDMPs by delivering high-quality source and hyperimmune plasma through a digitally optimized collection network.

About BioRewards Plasma Services

BioRewards Plasma Services is an emerging leader in the independent plasma collection industry, dedicated to delivering high-quality source and hyperimmune plasma material to pharmaceutical companies worldwide. With a leadership team boasting over 100 years of combined experience, BioRewards leverages cutting-edge technology to enhance the donor experience, improve operational efficiency, and ensure a stable high-quality plasma supply chain.

Contacts

Media Contact
Company Name: BioRewards Plasma Services

Contact Person: Media Relations

Email: Info@BioRewards.com
City: New York City

State: New York

Country: United States of America

www.BioRewards.com

Innate Pharma Highlights Abstracts Selected for ASCO 2025 Annual Meeting

Innate Pharma Highlights Abstracts Selected for ASCO 2025 Annual Meeting




Innate Pharma Highlights Abstracts Selected for ASCO 2025 Annual Meeting

  • Long term follow-up results of TELLOMAK Phase 2 trial in Sézary syndrome and mycosis fungoides
  • Trial in Progress poster on IPH4502, Innate’s differentiated ADC in development in advanced solid tumors expressing Nectin-4
  • AstraZeneca to present a poster on updated results from the Phase 2 trial NeoCOAST-2 in resectable NSCLC

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


Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) announced today that four abstracts with Innate’s drugs in clinical development have been accepted for the American Society of Clinical Oncology (ASCO) 2025 Annual Meeting, taking place May 30-June 3, 2025 in Chicago, Illinois.

We are looking forward to participating in the ASCO Annual Meeting 2025, where four abstracts with Innate’s drugs in clinical development have been selected. In particular, we are pleased to share the long-term follow-up data from the TELLOMAK Phase 2 study, which underscores our continued commitment to advancing innovative therapies for patients,” commented Dr Sonia Quaratino, Chief Medical Officer of Innate Pharma.

ASCO abstract details:

Lacutamab

Abstract: 2522
Abstract Title: Lacutamab in patients with relapsed and refractory Sézary syndrome: Long term follow-up from the TELLOMAK phase 2 trial

Session Type: Poster Session

Session Title: Developmental Therapeutics—Immunotherapy

Session Date and Time: Monday, June 2, 2025 – 1:30 – 4:30 PM CDT

Abstract: 2523
Abstract Title: Lacutamab in patients with relapsed and/or refractory mycosis fungoides: Long-term follow-up and translational data from the TELLOMAK phase 2 trial

Session Type: Poster Session

Session Title: Developmental Therapeutics—Immunotherapy

Session Date and Time: Monday, June 2, 2025 – 1:30 – 4:30 PM CDT

IPH4502

Abstract: TPS3159
Abstract Title: A phase 1, open-label, multi-center study of the safety, tolerability, and efficacy of IPH4502 as a single agent in advanced solid tumors

Session Type: Poster Session

Session Title: Developmental Therapeutics—Molecularly Targeted Agents and Tumor Biology

Session Date and Time: Monday, June 2, 2025 – 1:30 – 4:30 PM CDT

Monalizumab (partnered with AstraZeneca)

Abstract: 8046
Abstract Title: Neoadjuvant durvalumab (D) + chemotherapy (CT) + novel anticancer agents and adjuvant D ± novel agents in resectable non-small-cell lung cancer (NSCLC): Updated outcomes from NeoCOAST-2.

Session Type: Poster Session

Session Title: Lung Cancer—Non–Small Cell Local-Regional/Small Cell/Other Thoracic Cancers

Session Date and Time: Saturday, May 31, 2025 – 1:30 – 4:30 PM CDT

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

ISIN code
Ticker code
LEI

FR0010331421

Euronext: IPH Nasdaq: IPHA

9695002Y8420ZB8HJE29

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,” “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

Savara Presented New Data From Pivotal Phase 3 IMPALA-2 Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at American Thoracic Society Conference (ATS) 2025

Savara Presented New Data From Pivotal Phase 3 IMPALA-2 Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at American Thoracic Society Conference (ATS) 2025




Savara Presented New Data From Pivotal Phase 3 IMPALA-2 Trial of Molgramostim Inhalation Solution (Molgramostim) in Patients With Autoimmune Pulmonary Alveolar Proteinosis (aPAP) at American Thoracic Society Conference (ATS) 2025

LANGHORNE, Pa.–(BUSINESS WIRE)–Savara Inc. (Nasdaq: SVRA) (the Company), a clinical-stage biopharmaceutical company focused on rare respiratory diseases, today announced new data in two poster presentations at the ATS International Conference 2025. Data presented were from the Phase 3 IMPALA-2 clinical trial of molgramostim in aPAP and demonstrated that molgramostim reduces surfactant burden and improves health-related quality of life outcomes in patients with aPAP.


ATS 2025 Posters

Poster Title: Molgramostim Reduces Surfactant Burden and Number of Whole Lung Lavage Procedures in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP): Results From the IMPALA-2 Phase 3 Clinical Trial

Presenter: Tisha S. Wang, M.D., Professor of Clinical Medicine, Senior Executive Clinical Vice Chair, University of California Los Angeles Department of Medicine

Poster Number: 918

Poster Location: PD05, Room 3009/3011 West Building, Level 3

Summary:

  • Molgramostim reduced surfactant burden as measured by ground-glass opacification (GGO) scores, a radiological measure of surfactant burden. The mean reduction in GGO score from baseline to Week 24 was greater in the molgramostim group (n=78) than in the placebo group (n=79) (-2.1 vs. -1.1; P=0.0004)
  • Fewer patients in the molgramostim group required rescue whole lung lavages (WLL) compared with placebo. During the 48-week double-blind treatment period, 6 patients (7.4%) in the molgramostim group underwent a total of 15 WLLs and 11 patients (13.3%) in the placebo group underwent a total of 24 WLLs
  • Molgramostim reduces pulmonary surfactant burden, which drives the clinical manifestations of aPAP, and provides support for the potential beneficial treatment effect of molgramostim

Poster Title: The Effects of Molgramostim on Respiratory Health-related Quality of Life and Patient-reported Outcomes in Patients with Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Presenter: Ali Ataya, M.D., Associate Professor of Medicine, University of Florida, Division of Pulmonary and Critical Care Medicine

Poster number: P31

Poster Location: TP22, Area A, Hall F (North Building, Exhibition Level)

Summary:

  • Molgramostim showed benefit on measures of health-related quality of life (HRQoL) and patients’ assessment of breathing problems and physical activity, including changes from baseline in St. George’s Respiratory Questionnaire (SGRQ) Impact and Symptom scores, the EuroQol 5 Dimensions, 5 Levels (EQ-5D-5L), Patient Global Impression of Severity (PGIS), and Patient Global Impression of Change (PGIC) at Weeks 24 and 48, which were included as exploratory endpoints in the trial
  • Molgramostim improved respiratory HRQoL as measured by changes from baseline to Week 24 in SGRQ Impact (P=0.0084) and Symptom scores compared with placebo, and the EQ-5D-5L, a generic HRQoL instrument comprised of a short descriptive system questionnaire that allows patients to rate their health across 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Odds ratios of responses on the EQ-5D-5L numerically favored the molgramostim group on 3 of the 5 dimensions (mobility, self-care, and usual activities) at Weeks 24 and 48
  • Molgramostim reduced the severity of breathing problems, as assessed by PGIS, at both Weeks 24 (P=0.0305) and 48 (P=0.0049). Additionally, more molgramostim patients reported improvements in overall change in daily physical activity level, as measured by more patients assessing themselves as “Much better” or “A little better” compared with placebo at Week 24 (P=0.0368) and Week 48 (P=0.0193)

The abstracts were published in a supplement of the American Journal of Respiratory and Critical Care Medicine. For more details about the ATS International Conference please visit their website.

The posters are available on the Congresses & Publications page of the Company’s corporate website.

About the IMPALA-2 Trial

IMPALA-2 is a global, pivotal, Phase 3, 48-week, randomized, double-blind, placebo-controlled clinical trial designed to compare the efficacy and safety of molgramostim 300 mcg self-administered once daily by inhalation with matching placebo in patients with aPAP. The trial is being conducted at 43 clinical trial sites across 16 countries, including the U.S., Canada, Japan, South Korea, Australia, and countries in Europe, including Turkey. The primary efficacy assessment was diffusing capacity of the lungs for carbon monoxide (DLCO), a gas exchange measure, and the primary endpoint was change from baseline to Week 24 in percent predicted DLCO, with a secondary endpoint of change from baseline to Week 48 in percent predicted DLCO. Three additional secondary efficacy variables evaluated clinical measures of direct patient benefit: St. George’s Respiratory Questionnaire (SGRQ) Total score, SGRQ Activity score, and exercise capacity using a treadmill test, with each endpoint measured at Weeks 24 and 48. The primary time point for efficacy assessments was at Week 24; however, efficacy was assessed through Week 48 to evaluate durability of effect. Safety was assessed through Week 48. All patients who completed the 48-week double-blind treatment period continued into a 96-week open-label period during which they are receiving molgramostim 300 mcg administered once daily.

About Autoimmune Pulmonary Alveolar Proteinosis (aPAP)

Autoimmune PAP is a rare lung disease characterized by the abnormal build-up of surfactant in the alveoli of the lungs. Surfactant consists of proteins and lipids and is an important physiological substance that lines the alveoli to prevent them from collapsing. In a healthy lung, excess surfactant is cleared and digested by immune cells called alveolar macrophages. Alveolar macrophages need to be stimulated by granulocyte-macrophage colony-stimulating factor (GM-CSF) to function properly in clearing surfactant, but in aPAP, GM-CSF is neutralized by antibodies against GM-CSF, rendering macrophages unable to adequately clear surfactant. As a result, an excess of surfactant accumulates in the alveoli, causing impaired gas exchange, resulting in clinical symptoms of shortness of breath, often with cough and frequent fatigue. Patients may also experience episodes of fever, chest pain, or coughing up blood, especially if secondary lung infection develops. In the long term, the disease can lead to serious complications, including lung fibrosis and the need for a lung transplant.

About Savara

Savara is a clinical-stage biopharmaceutical company focused on rare respiratory diseases. Our lead program, molgramostim inhalation solution (molgramostim), is a recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) in Phase 3 development for autoimmune pulmonary alveolar proteinosis (aPAP). Molgramostim is delivered via an investigational eFlow® Nebulizer System (PARI Pharma GmbH) specifically developed for inhalation of a large molecule. Our management team has significant experience in rare respiratory diseases and pulmonary medicine, identifying unmet needs, and effectively advancing product candidates to approval and commercialization. More information can be found at www.savarapharma.com and LinkedIn.

Contacts

Media and Investor Relations Contact
Savara Inc.

Temre Johnson, Executive Director, Corporate Affairs

ir@savarapharma.com

IMVARIA Reports Multi-Site Clinical Experience With FDA-Authorized AI Diagnostic Service for Idiopathic Pulmonary Fibrosis at ATS 2025

IMVARIA Reports Multi-Site Clinical Experience With FDA-Authorized AI Diagnostic Service for Idiopathic Pulmonary Fibrosis at ATS 2025




IMVARIA Reports Multi-Site Clinical Experience With FDA-Authorized AI Diagnostic Service for Idiopathic Pulmonary Fibrosis at ATS 2025

Presenting real-world use of Fibresolve, the first-of-its-kind AI-powered adjunctive diagnostic service supporting assessment of suspected Interstitial Lung Disease (ILD) and Idiopathic Pulmonary Fibrosis (IPF)

BERKELEY, Calif.–(BUSINESS WIRE)–#ATSIMVARIA Inc., a health tech company pioneering AI-driven digital biomarker solutions, today reported results from multi-site clinical experiences with IMVARIA’s diagnostic referral service, where pulmonologists send cases for AI-supported diagnostic evaluation of suspected Interstitial Lung Disease (ILD) and Idiopathic Pulmonary Fibrosis (IPF). Built by medical doctors with software engineering expertise, Fibresolve is the first ever FDA-authorized AI adjunctive diagnostic service of any type in lung fibrosis. Clinical data from use around the U.S. will be presented by pulmonary experts from Harvard’s Mass General Hospital at ATS 2025 International Conference, focusing on respiratory diseases, held on May 16-21, 2025 in San Francisco.

“We’re excited that our clinical users are sharing real-world experience with Fibresolve at the ATS Conference,” said Joshua Reicher, MD, Co-founder and CEO of IMVARIA. “At IMVARIA, we’ve taken a different approach to AI – one that makes it far easier for pulmonologists to benefit from this new technology without changing workflows or installing complex systems. As practicing medical doctors, my co-founder Dr. Michael Muelly and I designed Fibresolve to meet the highest medical standards, deliver new insights, and make it easy for clinicians to use AI with confidence and minimal burden. We’re proud to see that approach working in real clinical settings.”

IMVARIA’s Fibresolve received FDA authorization in early 2024 and has gone through a rigorous process to make it useful and reliable for pulmonologists. Fibresolve is available through IMVARIA’s centralized service that uses AI to help guide safe, non-invasive diagnoses. Fibresolve also has the distinction as the first FDA Breakthrough-Designated AI diagnostic tool with simultaneously adopted CPT billing codes by the American Medical Association (AMA) in any disease.

IMVARIA is additionally presenting data on ScreenDx and Bronchosolve, two more AI solutions in its pulmonary portfolio. ScreenDx, FDA-cleared in 2025, is the first AI technology authorized to assess interstitial lung findings compatible with ILD. Bronchosolve is an investigational tool designed to support more accurate assessment of indeterminate lung nodules and is currently under research investigation.

Poster Presentations at ATS 2025 Conference

Fibresolve

Title: Clinical Experience with the First FDA-Authorized Artificial Intelligence Tool in Interstitial Lung Disease and Idiopathic Pulmonary Fibrosis

Session: A46 – New Research in Biomarkers and Imaging for ILD

Poster: P1547

Date and Time: Sunday, May 18, 2025: 9:15 AM – 4:15 PM

ScreenDx

Title: Automated Al Detection of Interstitial Lung Disease by Computed Tomography (CT) in the COPDGene Trial; Subanalysis and Characteristics of Accurately Detected Cases

Session: A57 – Late Breaking Abstracts in Clinical Problems

Poster: P1006

Date and Time: Sunday, May 18, 2025: 9:15 AM – 4:15 PM

Bronchosolve

Title: Closed Loop, Full Automation of Suspicious Lung Nodule Risk Assessment with AI in Screening Cases

Session: B110 – The Road to Early Detection: Advancing Lung Cancer Screening Through AI, Risk Models, And Real-World Data

Poster: 619

Date and Time: Monday, May 19, 2025: 2:15 PM – 4:15 PM

Title: Age-Stratified Subanalysis of a Closed Loop, Fully Automated Lung Nodule Risk Assessment Al Software

Session: B80-1 – From Bench to Bedside: Innovative Biomarkers, Screening Approaches, And Personalized Treatments In Lung Cancer

Poster: P804

Date and Time: Monday, May 19, 2025: 9:15 AM – 4:15 PM

Together, these presentations reflect IMVARIA’s mission to empower clinicians to make the best decisions through clinically meaningful AI.

For more information about IMVARIA, click here.

About IMVARIA Inc.

IMVARIA is a health tech company pioneering AI-driven solutions that empower clinicians to make accurate diagnoses and prognoses at earlier stages of disease. Founded in 2019 by physician-engineers from Google and Stanford University, the company operates its AI Lab with automated, machine-learning algorithm technology to transform clinical decision-making into data science. IMVARIA is based in Berkeley, CA. For more information, go to www.imvaria.com.

Contacts

Media Contact
Anthony Petrucci

Bioscribe

anthony@bioscribe.com

Be Biopharma Announces New Preclinical Data for BE-102, a B Cell Medicine for the Potential Treatment of Hypophosphatasia

Be Biopharma Announces New Preclinical Data for BE-102, a B Cell Medicine for the Potential Treatment of Hypophosphatasia




Be Biopharma Announces New Preclinical Data for BE-102, a B Cell Medicine for the Potential Treatment of Hypophosphatasia

  • Preclinical research demonstrates that a single administration of BE-102 provides continuous secretion of active alkaline phosphatase (ALP) in vivo out to 6 months
  • No safety findings observed in long-term pharmacology studies
  • Data presented at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Be Biopharma, Inc. (“Be Bio”), a clinical-stage company pioneering the discovery and development of engineered B Cell Medicines (BCMs), today presented results from new preclinical research demonstrating a single administration of BE-102, a BCM for the potential treatment for Hypophosphatasia (HPP), produces continuous levels of active ALP long-term in vivo. The findings will be presented during an oral presentation at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting on Saturday, May 17, at 10:45 AM CT.

HPP is a genetic disease caused by deficient ALP activity, resulting from pathogenic mutations in the ALPL gene, which leads to multi-systemic clinical complications including deficient bone mineralization. Enzyme replacement therapy (ERT) is the only approved treatment for HPP which requires frequent lifelong injections, and is only available for perinatal/infantile- and juvenile-onset forms of HPP. BE-102 was developed to address these limitations by providing continuous secretion of active ALP from a single infusion, with the flexibility to be titratable and re-dosable as needed. BE-102 is manufactured from primary human B cells by isolating, activating, and precision engineering with CRISPR/Cas9 followed by AAV-mediated delivery of a DNA donor template for the insertion of human ALPL gene into the CCR5 locus (a safe harbor locus) followed by expansion and differentiation in culture into ALP-secreting B lymphocyte lineage cells.

“These studies demonstrate the potential of our B cell medicine platform to deliver B cell derived active ALP with durability out to six months,” said Rick Morgan, Chief Scientific Officer of Be Biopharma. “BE-102 offers a novel and durable approach that may overcome the limitations of current enzyme therapy and does not require pre-conditioning, offering flexibility for re-dosing.”

The presentation highlights both in vivo and in vitro data supporting the target product profile for BE-102. In vivo studies were conducted in immune-deficient NOG-hIL6 mice, confirming long-term engraftment and continuous production of B cell derived active ALP (>175 days) following a single IV administration of BE-102. No BE-102 related adverse events have been observed across multiple in vivo studies. In vitro pharmacology data presented today demonstrates that BE-102 secretes active ALP, which is capable of rescuing calcium deposit inhibited by inorganic pyrophosphate (PPi), a potent inhibitor of bone mineralization and an ALP substrate which accumulates in people with HPP. Be Bio’s in vitro and in vivo pharmacology and safety data established preclinical proof-of-concept that BE-102 has the potential to be a disease-modifying therapy for people with HPP by providing continuous secretion of ALP, with the flexibility to be titratable and re-dosable as needed. A robust package of preclinical studies is planned in anticipation of submission of an IND for a first-in-human clinical trial for people with HPP.

About BE-102

BE-102 is a first-in-class BCM that has been engineered using artificial intelligence-guided protein design to modify primary human B cells to produce ALP, an enzyme deficient in people living with HPP. A single infusion of BE-102 has the potential to provide continuous secretion of therapeutic ALP with the flexibility to be titrated and/or re-dosed, if needed, and without the need for pre-conditioning. BE-102 has been selected as a Development Candidate and has the potential to transform the standard of care for people living with HPP.

About Engineered B Cell Medicines – A New Class of Cellular Medicines

The B cell is a powerful cell that produces thousands of proteins per cell per second at constant levels, and over decades. Precision genome editing can now be used to engineer B Cells that produce therapeutic proteins of interest, driving a new class of cellular medicines – Engineered B Cell Medicines (BCMs) – with the potential to be durable, allogeneic, re-dosable, and administered without pre-conditioning. The promise of BCMs could transform therapeutic biologics with broad application — across protein classes, patient populations and therapeutic areas.

About Be Biopharma

Be Biopharma (“Be Bio”) is pioneering Engineered B Cell Medicines (BCMs) to dramatically improve the lives of patients who are living with Hemophilia B and other genetic diseases, cancer, and other serious conditions. With eyes locked on the patient, our team of purpose-driven scientists, technologists, manufacturing experts and business builders collaborate to create a bold new class of cell therapies. Be Bio was founded in October 2020, and is backed by ARCH Venture Partners, Atlas Venture, RA Capital Management, Nextech, Alta Partners, Longwood Fund, Bristol Myers Squibb, Takeda Ventures, Seattle Children’s Research Institute, Pathway to Cures (the venture philanthropy fund for the National Bleeding Disorders Foundation) and others to re-imagine medicine based on the power of Engineered B cells. For more information, please visit us at Be.Bio and our LinkedIn page.

Contacts

Investor Contact:
ir@be.bio

Media Contact:
media@be.bio

AMVUTTRA® (vutrisiran) Significantly Reduces Mortality and a Range of Important Cardiovascular Events in Patients with ATTR Amyloidosis with Cardiomyopathy: Additional Data from HELIOS-B

AMVUTTRA® (vutrisiran) Significantly Reduces Mortality and a Range of Important Cardiovascular Events in Patients with ATTR Amyloidosis with Cardiomyopathy: Additional Data from HELIOS-B




AMVUTTRA® (vutrisiran) Significantly Reduces Mortality and a Range of Important Cardiovascular Events in Patients with ATTR Amyloidosis with Cardiomyopathy: Additional Data from HELIOS-B

– Analysis Presented at the Heart Failure 2025 Congress Supports Primary Findings, Highlighting Impact of AMVUTTRA, which Delivers Rapid Knockdown of Transthyretin –

– Additional 42-Month Data Reinforce Vutrisiran’s Impact on the Risk of All-Cause Mortality and Further Underscore the Effect on Cardiovascular Mortality –

Vutrisiran Demonstrated Substantial Benefit Across a Range of Cardiovascular Events, Notably Reducing Urgent Heart Failure Visits by 46% in the Overall Patient Population During the Double-Blind Period –

– Findings Simultaneously Published in JACC –

– Details of TRITON-CM Phase 3 Study Evaluating Alnylam’s Investigational Next-Generation TTR Silencer, Nucresiran, to be Presented at Congress –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today presented the most contemporary analysis of the HELIOS-B Phase 3 study of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) as a late-breaking abstract at the Heart Failure 2025 Congress, a scientific congress of the European Society of Cardiology, taking place May 17-20 in Belgrade, Serbia. The results demonstrate that vutrisiran, which rapidly knocks down transthyretin, reduces key cardiovascular (CV) events such as CV hospitalizations, and heart failure (HF) hospitalizations. Additionally, in the analysis, urgent HF visits were reduced by 46% (95% CI: 0.30, 0.98; p = 0.041) in the overall population during the double-blind period, compared to placebo. These CV events often precede all-cause mortality (ACM) and are key indicators of disease progression.


Importantly, results from the November 2024 data cut, including further follow up through up to 42 months, reinforce the primary HELIOS-B analysis showing vutrisiran’s effect on ACM, and further demonstrate that vutrisiran reduces CV mortality. Through 42 months, the risk of ACM was reduced by 36% (95% CI: 0.46, 0.88; p = 0.007) and the risk of CV mortality was reduced by 33% (95% CI: 0.47, 0.96; p = 0.038) in the overall population, compared to placebo. For both the primary analysis and the current analysis, vital status through 42 months was ascertained for over 99% of all randomized patients from the HELIOS-B study, underscoring the robustness of the results.

The study was conducted in a contemporary patient population with patients receiving robust background therapy, inclusive of treatment with a TTR stabilizer and SGLT2 inhibitors. The analysis of the HELIOS-B Phase 3 study, including mortality data through up to 42 months, was simultaneously published in JACC.

“From the primary analysis of HELIOS-B, we know that AMVUTTRA profoundly impacts all-cause mortality, while preserving patients’ functional capacity and quality of life,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “These new data—including the impact on mortality, on cardiovascular events and on urgent heart failure visits, the latter of which was reduced by nearly half—add to the story of consistency and magnitude of benefit. I remain impressed by the HELIOS-B results, which are noteworthy given the substantial use of heart failure treatments in the study population, and I believe they continue to reinforce AMVUTTRA as a clinically differentiated, first-line option for patients with ATTR-CM.”

The results from the analysis underscore the rapid and sustained benefits of vutrisiran in treating ATTR-CM across key endpoints:

Impact of Vutrisiran Versus Placebo

Overall Population

N=654

Reduction in Urgent HF Visits through 36 Months

46%

RR: 0.54

P = 0.041

Reduction in HF Hospitalizations through 36 Months

33%

RR: 0.67

P = 0.002

Reduction in Risk of CV Events through 36 Months

27%

RR: 0.73

P = 0.001

Reduction in CV Hospitalizations through 36 Months

25%

RR: 0.75

P = 0.004

Reduction in Risk of ACM through 42 Months

36%

HR: 0.64

P = 0.007

Reduction in CV Mortality through 42 Months

33%

HR: 0.67

P = 0.038

In a separate presentation on Tuesday, May 20, Alnylam will share findings from a subgroup analysis of HELIOS-B evaluating the impact of vutrisiran on ACM and recurrent CV events among patients identified by investigators as having experienced disease progression while being treated with tafamidis.

Also at the Heart Failure 2025 Congress, Alnylam will present the study design and rationale for TRITON-CM, a Phase 3, randomized, double-blind, study of nucresiran in patients with ATTR-CM. Nucresiran is an investigational next-generation RNAi therapeutic targeting TTR that has been shown to deliver rapid knockdown of TTR greater than 95% with twice-annual dosing in a Phase 1 study. TRITON-CM is an event-driven CV outcomes trial with a primary endpoint of composite ACM and CV events. The study is on track to initiate in the first half of 2025 and will enroll approximately 1,200 patients with wild-type or variant TTR and confirmed cardiomyopathy, including those receiving background stabilizer therapy. Additional details of the study’s secondary endpoints and key inclusion and exclusion criteria will be shared on Monday, May 19.

AMVUTTRA® (vutrisiran) was approved by the U.S. Food and Drug Administration (FDA) and the Brazilian Health Regulatory Agency (ANVISA) for treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of vutrisiran for the same indication. A formal regulatory decision by the European Commission of the EMA is expected by the third quarter of 2025. Vutrisiran is currently under review for the treatment of ATTR-CM by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025 and beyond.

For additional information on Alnylam’s presentations at the Heart Failure 2025 Congress, please visit Capella.

Indications and Important Safety Information

Indications Approved by the U.S. FDA

AMVUTTRA® (vutrisiran) is indicated for the treatment of the:

  • cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
  • polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation

AMVUTTRA treatment leads to a decrease in serum vitamin A levels.

Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions

In a study of patients with hATTR-PN, the most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%).

In a study of patients with ATTR-CM, no new safety issues were identified.

For additional information about AMVUTTRA, please see the full U.S. Prescribing Information (revised March 2025)

About AMVUTTRA® (vutrisiran)

AMVUTTRA® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of variant and wild-type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, vutrisiran is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. In Europe, it is administered as a subcutaneous injection once every three months, either by a healthcare professional, or self-administered by patients or their caregivers. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease.

About ATTR

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.1-4

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

Alnylam Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam’s expectations regarding the safety and efficacy of vutrisiran for the treatment of ATTR-CM, including the ability of vutrisiran to reduce mortality and cardiovascular events in ATTR-CM patients and to preserve patients’ functional capacity and quality of life; the potential for vutrisiran to become a first-line therapy for ATTR-CM; the timing of the initiation of the TRITON-CM study and the number of patients who will be enrolled in that study; the timing of additional global regulatory submissions for vutrisiran; the timing or receipt of any additional regulatory approvals for vutrisiran for ATTR-CM; Alnylam’s ability to execute on its “Alnylam P5x25” strategy and to deliver transformative medicines in both rare and common diseases benefit patients around the world through sustainable innovation and exceptional financial performance; and Alnylam’s ability to have a leading biotech profile should be considered forward-looking statements.

Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam’s ability to successfully execute on its “Alnylam P5x25” strategy; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing Alnylam’s views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638.

2 Gertz MA. Am J Manag Care. 2017;23(7):S107-S112.

3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9.

4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.

Contacts

Alnylam Pharmaceuticals, Inc.


Christine Regan Lindenboom

(Investors and Media)

+1-617-682-4340

Josh Brodsky

(Investors)

+1-617-551-8276

Atara Biotherapeutics Appoints James Huang and Nachi Subramanian to Board of Directors

Atara Biotherapeutics Appoints James Huang and Nachi Subramanian to Board of Directors




Atara Biotherapeutics Appoints James Huang and Nachi Subramanian to Board of Directors

THOUSAND OAKS, Calif.–(BUSINESS WIRE)–Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, announced the appointment of James Huang and Nachi Subramanian to its Board of Directors, effective following the completion the Company’s previously announced $16 million offering that was announced on May 15, 2025. The offering closed on May 16, 2025.


Mr. Huang has over 37 years of biotech experience and is the Founder and Managing Partner of Panacea Venture. Prior to Panacea Venture, Mr. Huang was a Managing Partner at Kleiner Perkins (KPCB) China where he focused on the firm’s life sciences practice, and a managing partner at Vivo Ventures where he led numerous investments in China. He was also the president of Anesiva, Inc., a biopharmaceutical company focused on pain-management treatments. Earlier in his career, he held senior roles in business development, sales, marketing, and research and development with Tularik Inc. (acquired by Amgen), GlaxoSmithKline LLC, Bristol-Myers Squibb and ALZA Corp. (acquired by Johnson & Johnson). Additionally, Mr. Huang serves as a member of the board of directors of Kindstar Globalgene Technology, Inc., Connect Biopharma Holdings Limited, Lee’s Pharmaceutical Holdings Limited and several private companies. He received an M.B.A. from the Stanford Graduate School of Business and a B.S. degree in chemical engineering from the University of California, Berkeley.

Nachi Subramanian has served as a Managing Director at the Redmile Group since December 2021. He previously spent fourteen years at J.P. Morgan, where he held senior roles across the Private Markets and Global Cash Equities businesses. Nachi began his career in Institutional Equities at Bear Stearns. He holds a B.A. in Political Science and Economics from the University of California, Irvine.

About Atara Biotherapeutics, Inc.

Atara is a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr Virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases. Atara is headquartered in Southern California.

Contacts

Investor and Media Relations
Amber Daugherty

Sr. Director, Strategy and Operations

adaugherty@atarabio.com

IFF Announces Pricing of Tender Offers For Certain Outstanding Series of Notes

IFF Announces Pricing of Tender Offers For Certain Outstanding Series of Notes




IFF Announces Pricing of Tender Offers For Certain Outstanding Series of Notes

NEW YORK–(BUSINESS WIRE)–IFF (NYSE: IFF) announced today the Total Consideration (as defined below) payable in connection with its previously announced tender offers to purchase for cash: (i) up to $1,100,000,000 aggregate purchase price, excluding accrued and unpaid interest (the “Amended Pool 1 Maximum Amount”), of its 1.230% Senior Notes due 2025 (the “2025 Notes”), 1.832% Senior Notes due 2027 (the “2027 Notes”), 4.450% Senior Notes due 2028 (the “2028 Notes”) and 2.300% Senior Notes due 2030 (the “2030 Notes” and collectively with the 2025 Notes, the 2027 Notes and the 2028 Notes, the “Pool 1 Notes”) and (ii) up to $900,000,000 aggregate purchase price, excluding accrued and unpaid interest (the “Amended Pool 2 Maximum Amount” and, together with the Amended Pool 1 Maximum Amount, the “Amended Maximum Amounts”), of its 3.268% Senior Notes due 2040 (the “2040 Notes”), 4.375% Senior Notes due 2047 (the “2047 Notes”), 5.000% Senior Notes due 2048 (the “2048 Notes”) and 3.468% Senior Notes due 2050 (the “2050 Notes” and collectively with the 2040 Notes, the 2047 Notes and the 2048 Notes, the “Pool 2 Notes” and, together with the Pool 1 Notes, the “Notes”), subject to prioritized acceptance levels listed in the table below (“Acceptance Priority Levels”), Series Tender Caps (as defined below), if applicable, and the terms and conditions of the tender offers.


The table below sets forth, among other things, the aggregate principal amount of each series of Notes validly tendered and not validly withdrawn as of 5:00 p.m., New York City time, on May 15, 2025 (the “Early Tender Date”) and expected to be accepted for purchase in each tender offer, the approximate proration factor for such Notes and the Total Consideration for each series of such Notes, as calculated at 10:00 a.m., New York City time, today, May 16, 2025.

Pool 1 Tender Offers

Amended Pool 1 Maximum Amount: $1,100,000,000(1(a))

Title of

Security

CUSIP/ISIN

Principal

Amount

Outstanding

Acceptance

Priority

Level(2)

Series

Tender

Cap(3)

U.S.

Treasury

Reference

Security

Bloomberg

Reference

Page

Fixed

Spread

Reference

Yield

Principal

Amount

Tendered at

Early Tender

Date

Principal

Amount

Expected to

be Accepted

Approximate

Proration

Factor

Total

Consideration

(4)(5)

1.230% Senior Notes due 2025

459506AN1

 

U45950AE9

$1,000,000,000

1

$500,000,000

5.000% UST due 9/30/25

FIT3

+ 0 bps

4.385%

$849,172,000

$500,000,000

58.91%

$988.67

1.832% Senior Notes due 2027

459506AP6

 

U45950AF6

$1,200,000,000

2

$400,000,000

3.750% UST due 4/30/27

FIT1

+ 75 bps

3.936%

$540,985,000

$400,000,000

73.97%

$935.84

2.300% Senior Notes due 2030

459506AQ4

 

U45950AG4

$1,500,000,000

3

N/A

3.875% UST due 4/30/30

FIT1

+ 110 bps

4.019%

$524,136,000

$266,678,000

50.90%

$867.44

Pool 2 Tender Offers

Amended Pool 2 Maximum Amount: $900,000,000(1(b))

Title of

Security

CUSIP/ISIN

Principal

Amount

Outstanding

Acceptance

Priority

Level(2)

Series

Tender

Cap(3)

U.S.

Treasury

Reference

Security

Bloomberg

Reference

Page

Fixed

Spread

Reference

Yield

Principal

Amount

Tendered at

Early Tender

Date

Principal

Amount

Expected to

be Accepted

Approximate

Proration

Factor

Total

Consideration

(4)(5)

3.468% Senior Notes due 2050

459506AS0

 

U45950AJ8

$1,500,000,000

1

$649,114,000

4.500% UST due 11/15/54

FIT1

+ 135 bps

4.884%

$649,114,000

$649,114,000

N/A

$648.85

3.268% Senior Notes due 2040

459506AR2

 

U45950AH2

$750,000,000

2

$450,000,000

4.625% UST due 2/15/35

FIT1

+ 165 bps

4.397%

$417,599,000

$417,599,000

N/A

$723.10

4.375% Senior Notes due 2047

459506AE1

 

US459506AE19

$500,000,000

3

N/A

4.750% UST due 2/15/45

FIT1

+ 145 bps

4.889%

$103,796,000

$103,796,000

N/A

$768.50

5.000% Senior Notes due 2048

459506AL5

 

US459506AL51

$800,000,000

4

N/A

4.750% UST due 2/15/45

FIT1

+ 140 bps

4.889%

$294,828,000

$115,138,000

39.11%

$843.23

(1) (a) Represents the maximum aggregate purchase price payable, excluding the applicable accrued and unpaid interest, in respect of the Pool 1 Notes.

  

(b) Represents the maximum aggregate purchase price payable, excluding the applicable accrued and unpaid interest, in respect of the Pool 2 Notes.

  

(2) Subject to the Amended Maximum Amounts, the Series Tender Caps (as define below) and proration, if applicable, the principal amount of each series of Notes that is purchased in each tender offer will be determined in accordance with the applicable Acceptance Priority Level (in numerical priority order) specified in this column.

  

(3) The 1.230% Senior Notes due 2025 will be subject to an aggregate principal amount sublimit of $500,000,000 (the “2025 Notes Series Tender Cap”) and the 1.832% Senior Notes 2027 will be subject to an aggregate principal amount sublimit of $400,000,000 (the “Amended 2027 Notes Series Tender Cap”). The 3.268% Senior Notes due 2040 will be subject to an aggregate principal amount sublimit of $450,000,000 the “2040 Notes Series Tender Cap”) and the 3.468% Senior Notes due 2050 will be subject to an aggregate principal amount sublimit of $649,114,000 (the “Amended 2050 Notes Series Tender Cap” and, together with the 2025 Notes Series Tender Cap, the Amended 2027 Notes Series Tender Cap and the 2040 Notes Series Tender Cap, the “Series Tender Caps”).

  

(4) Per $1,000 principal amount of Notes validly tendered prior to or at the Early Tender Date and accepted for purchase.

  

(5) The Total Consideration for each series of Notes validly tendered prior to or at the Early Tender Date and accepted for purchase is calculated using the applicable fixed spread specified in the table above and is inclusive of the Early Tender Payment of $30.00 per $1,000 principal amount of Notes. The Total Consideration for each series of Notes does not include accrued and unpaid interest, which will be payable in addition to the applicable Total Consideration.

The tender offers are being made upon the terms, and subject to the conditions, previously described in the Offer to Purchase dated May 2, 2025, as amended and supplemented by IFF’s news release on May 16, 2025 (as so amended, the “Offer to Purchase”). IFF refers investors to the Offer to Purchase for the complete terms and conditions of the tender offers.

Withdrawal rights for the Notes expired at 5:00 p.m., New York City time, on the Early Tender Date. The tender offers for the Notes will expire at 5:00 p.m., New York City time, on June 2, 2025, or any other date and time to which IFF extends the applicable tender offer, unless earlier terminated. As previously announced, IFF expects to elect to exercise its right to make payment on May 20, 2025 (the “Early Settlement Date”) for Notes that were validly tendered prior to or at the Early Tender Date and that are accepted for purchase. IFF intends to fund the purchase of validly tendered and accepted Notes with the cash proceeds from the sale of its Pharma Solutions business, which was completed on May 1, 2025.

Because the Pool 1 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date have an aggregate purchase price, excluding accrued and unpaid interest, that exceeds the Amended Pool 1 Maximum Amount, IFF does not expect to accept for purchase all Pool 1 Notes that have been validly tendered and not validly withdrawn prior to or at the Early Tender Date. Rather, subject to the Amended Pool 1 Maximum Amount, the Series Tender Caps and the Acceptance Priority Levels set forth in the table above, in each case as further described in the Offer to Purchase, IFF expects to accept for purchase $500,000,000 aggregate principal amount of the 2025 Notes and $400,000,000 aggregate principal amount of 2027 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date. IFF expects to accept for purchase $266,678,000 aggregate principal amount of the 2030 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date on a prorated basis using a proration factor of approximately 50.90%. IFF does not expect to accept for purchase any amount of the 2028 Notes. Because the aggregate principal amount of the 2025 Notes and the 2027 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date exceeds the 2025 Series Tender Cap and Amended 2027 Series Tender Cap, IFF expects to accept for purchase an amount equal to the applicable Series Tender Cap in each case validly tendered and not validly withdrawn prior to or at the Early Tender Date on a prorated basis using a proration factor of approximately 58.91% and 73.97%, respectively. As described further in the Offer to Purchase, Notes tendered and not accepted for purchase will be promptly credited to the tendering holder’s account. Additionally, because the Pool 1 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date have an aggregate purchase price, excluding accrued and unpaid interest, that exceeds the Amended Pool 1 Maximum Amount, IFF does not expect to accept for purchase any Pool 1 Notes tendered after the Early Tender Date on a subsequent settlement date.

Because the Pool 2 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date have an aggregate purchase price, excluding accrued and unpaid interest, that exceeds the Amended Pool 2 Maximum Amount, IFF does not expect to accept for purchase all Pool 2 Notes that have been validly tendered and not validly withdrawn prior to or at the Early Tender Date. Rather, subject to the Amended Pool 2 Maximum Amount, the Series Tender Caps and the Acceptance Priority Levels set forth in the table above, in each case as further described in the Offer to Purchase, IFF expects to accept for purchase $649,114,000 aggregate principal amount of 2050 Notes, $417,599,000 aggregate principal amount of 2040 Notes, and $103,796,000 aggregate principal amount of 2047 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date. Because the aggregate principal amount of the 2050 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date is equal to the Amended 2050 Series Tender Cap, IFF expects to accept for purchase all of the 2050 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date. IFF expects to accept for purchase all of the 2040 Notes and 2047 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date. IFF expects to purchase an aggregate principal amount of $115,138,000 of the 2048 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date on a prorated basis using a proration factor of approximately 39.11%. As described further in the Offer to Purchase, Notes tendered and not accepted for purchase will be promptly credited to the tendering holder’s account. Additionally, because the Pool 2 Notes validly tendered and not validly withdrawn prior to or at the Early Tender Date have an aggregate purchase price, excluding accrued and unpaid interest, that exceeds the Amended Pool 2 Maximum Amount, IFF does not expect to accept for purchase any Pool 2 Notes tendered after the Early Tender Date on a subsequent settlement date.

The applicable Total Consideration listed in the table above (the “Total Consideration”) will be paid per $1,000 principal amount of each series of Notes validly tendered and accepted for purchase pursuant to the applicable tender offer on the Early Settlement Date. Only holders of Notes who validly tendered and did not validly withdraw their Notes prior to or at the Early Tender Date are eligible to receive the applicable Total Consideration for Notes accepted for purchase. Holders will also receive accrued and unpaid interest on Notes validly tendered and accepted for purchase from the applicable last interest payment date up to, but not including, the Early Settlement Date.

All Notes accepted for purchase will be retired and cancelled and will no longer remain outstanding obligations of IFF.

IFF’s obligation to accept for payment and to pay for Notes validly tendered and not validly withdrawn in the tender offers is subject to the satisfaction of certain conditions described in the Offer to Purchase. IFF reserves the right, subject to applicable law, to (i) waive any and all conditions to any of the tender offers, (ii) extend or terminate any of the tender offers, (iii) further increase or decrease either of the Amended Maximum Amounts and/or increase, decrease or eliminate any of the Series Tender Caps (other than the 2040 Series Tender Cap), or (iv) otherwise further amend any of the tender offers. IFF may take any action described in clauses (i) through (iv) above with respect to one or more tender offers without having to do so for all tender offers.

Information relating to the tender offers

Barclays Capital Inc., BNP Paribas Securities Corp. and BofA Securities, Inc. are the lead dealer managers for the tender offers. The other dealer managers for the tender offers are Citigroup Global Markets Inc., Mizuho Securities USA LLC, Wells Fargo Securities, LLC, ING Financial Markets LLC, U.S. Bancorp Investments, Inc. and SMBC Nikko Securities America, Inc. Investors with questions regarding the terms and conditions of the tender offers may contact Barclays Capital Inc. at (800) 438-3242 or by email at us.lm@barclays.com, BNP Paribas Securities Corp. at (888) 210-4358 or by email at dl.us.liability.management@us.bnpparibas.com or BofA Securities, Inc. at (888) 292-0070 or (980) 387-3907 or by email at debt_advisory@bofa.com. D.F. King & Co., Inc. is the tender and information agent for the tender offers. Investors with questions regarding the procedures for tendering Notes may contact the tender and information agent by email at IFF@dfking.com, or by phone at (212) 269-5550 (for banks and brokers only) or (877) 478-5045 (for all others, toll-free). Beneficial owners may also contact their broker, dealer, commercial bank, trust company or other nominee for assistance.

The full details of the tender offers, including complete instructions on how to tender Notes, are included in the Offer to Purchase. Holders are strongly encouraged to read carefully the Offer to Purchase, including materials incorporated by reference therein, because they contain important information. The Offer to Purchase may be obtained from D.F. King & Co., Inc., free of charge, by calling (212) 269-5550 (for banks and brokers only) or (877) 478-5045 (for all others, toll-free).

This news release does not constitute an offer to purchase, or a solicitation of an offer to sell, or the solicitation of tenders with respect to the Notes. No offer, solicitation, purchase or sale will be made in any jurisdiction in which such an offer, solicitation or sale would be unlawful. The tender offers are being made solely pursuant to the Offer to Purchase made available to holders of the Notes. None of IFF or its affiliates, their respective boards of directors, the dealer managers, the tender and information agent or the trustee, with respect to any series of Notes, is making any recommendation as to whether or not holders should tender or refrain from tendering all or any portion of their Notes in response to the tender offers. Holders are urged to evaluate carefully all information in the Offer to Purchase, consult their own investment and tax advisors and make their own decisions whether to tender Notes in the tender offers, and, if so, the principal amount of Notes to tender.

Cautionary Statement Under The Private Securities Litigation Reform Act of 1995

This press release contains “forward-looking statements” within the meaning of the federal securities laws, including Section 27A of the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended (the “Exchange Act”). Forward-looking statements often address expected future business and financial performance and financial condition, and often contain words such as “”plan”, “expect,” “anticipate,” “intend,” “believe,” “seek,” “see,” “will,” “would,” “target,” similar expressions, and variations or negatives of these words. Forward-looking statements by their nature address matters that are, to different degrees, uncertain. The forward-looking statements included in this release are made only as of the date hereof, and we undertake no obligation to update the forward-looking statement to reflect subsequent events or circumstances.

Welcome to IFF

At IFF (NYSE: IFF), we make joy through science, creativity and heart. As the global leader in flavors, fragrances, food ingredients, health and biosciences, we deliver groundbreaking, sustainable innovations that elevate everyday products—advancing wellness, delighting the senses and enhancing the human experience. Learn more at iff.com, Linkedln, Instagram and Facebook.

© 2025 by International Flavors & Fragrances Inc. IFF is a Registered Trademark. All Rights Reserved.

Contacts

Media Relations:

Paulina Heinkel

332.877.5339

Media.request@iff.com

Investor Relations:

Michael Bender

212.708.7263

Investor.Relations@iff.com

SmithRx Named to Modern Healthcare’s Best Places to Work in Healthcare in 2025 List

SmithRx Named to Modern Healthcare’s Best Places to Work in Healthcare in 2025 List




SmithRx Named to Modern Healthcare’s Best Places to Work in Healthcare in 2025 List

Modern pharmacy benefits leader has grown headcount by 450% in 3 years

SAN FRANCISCO–(BUSINESS WIRE)–SmithRx, a modern pharmacy benefits manager (PBM) committed to transparency and a universal pass-through model, has been recognized by Modern Healthcare as one of the 2025 Best Places to Work in Healthcare. The award is based largely on employee feedback from a confidential survey administered by a third-party organization, and measures employee sentiment on key issues including growth opportunities, benefits, company culture, and more.


Founded in 2018, SmithRx has intentionally built a mission-driven and collaborative culture that drives employees to do their best work for clients and each other. Each SmithRx team member strongly believes in the company’s mission of transforming U.S. healthcare and its transparent, universal pass-through model, which helps significantly lower pharmacy costs for employer clients. Paired with its innovative savings programs, SmithRx’s approach to pharmacy benefits drives cost reduction for clients by an average of 30% or more.

“The people behind SmithRx have always been our secret sauce, with their contributions supporting our efforts to pioneer a smarter, fairer healthcare system in this country,” said Jake Frenz, Founder and CEO of SmithRx. “It is our highest priority to empower our employees as they grow their careers, collaborate on complex problems, and find satisfaction in the work. We’re so pleased that our team feels supported at SmithRx.”

SmithRx has experienced extensive growth since its inception. The company brought its total customer base to over 4,000 clients in 2024, and achieved more than $200M in savings for clients with minimal member disruption. This momentum has required an expanded team as well; SmithRx has grown its headcount by 450% over the past three years.

Modern Healthcare’s Best Places to Work in Healthcare awards program identifies and recognizes outstanding employers in the healthcare industry nationwide. Modern Healthcare partners with Workforce Research Group on the assessment process, which includes an extensive employee survey. The complete list of this year’s winners, in alphabetical order, is available at modernhealthcare.com/bestplaceslist.

“Being recognized as a 2025 Best Place to Work in Healthcare is a powerful testament to how these organizations value their people,” said Dan Peres, President of Modern Healthcare. “In a time of constant change and challenge, this year’s winners have shown a deep commitment to creating environments where employees feel supported, heard, and inspired to do their best work. That kind of culture doesn’t happen by accident — it’s intentional, and it’s worth celebrating.”

About SmithRx

SmithRx is a modern PBM dedicated to reducing the cost and complexity of pharmacy benefits. The company empowers organizations of all sizes to take control of their pharmacy spend with a radically transparent, universal pass-through model, innovative cost-saving programs, and intuitive technology. Unlike legacy PBMs, SmithRx eliminates hidden fees, passes through 100% of rebates and discounts, and provides real-time prescription pricing and detailed savings reports. This gives clients the insights they need to make informed decisions and achieve meaningful savings for both their business and their employees. Experience the difference transparency, trust, and fairness can make in building a more equitable healthcare system. Learn more at www.smithrx.com.

About Modern Healthcare

Modern Healthcare is the most trusted business news and information brand in the healthcare industry. Modern Healthcare empowers healthcare leaders and influencers to make timely and informed business decisions. To learn more or subscribe, go to www.modernhealthcare.com/subscriptions

Contacts

Media Contact:
Claire Schillings

claire.schillings@smithrx.com