Maternal and Postnatal Outcomes Study (MOS) A Worldwide Decentralized Observational Registry to Evaluate the Safety in Women With Fabry Disease and Their Infants Exposed to Elfabrio® (Pegunigalsidase Alfa-iwxj/Pegunigalsidase Alfa) During Pregnancy and/or Lactation

The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation.
The main objectives are to:
* Assess pregnancy outcomes, including maternal and infant health.
* Evaluate the occurrence of congenital malformations and other neonatal outcomes.
This is a global, decentralized, single-arm, prospective and retrospective registry planned to enroll participants over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data will be collected through a secure web-based platform, allowing patients and physicians to enter information via electronic case report forms (eCRFs).
Pregnancy and clinical outcomes will be documented throughout pregnancy and up to 12 months post-birth. Data from self-enrolled patients will be confirmed by their primary care or attending physician. This registry is observational and does not impact clinical care or treatment decisions.

Start Date01 Jun 2025

Sponsor / Collaborator

CHIESI Farmaceutici SpA

[+1]

NCT05541159

/ WithdrawnPhase 1

A Phase 1, Open-label, Single-dose, Multi-center, Parallel Group Study to Evaluate the Pharmacokinetics of TNO155 in Participants With Mild, Moderate, or Severe Renal Impairment Compared to Matched Healthy Control Participants

The purpose of this Phase 1 study is to evaluate the effect of various degrees of renal impairment on plasma pharmacokinetics (PK), safety and tolerability of TNO155. The results of this study will guide the Novartis recommendation regarding whether or not a dose adjustment may be needed when treating patients with renal impairment

Start Date19 Mar 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

[+1]

NCT05490030

/ WithdrawnPhase 1

A Phase 1, Open-label, Single-dose, Multi-center, Parallel Group Study to Evaluate the Pharmacokinetics of TNO155 in Participants With Mild, Moderate, or Severe Hepatic Impairment Compared to Matched Healthy Control Participants

The purpose of this study is to evaluate the effect of various degrees of hepatic impairment on plasma pharmacokinetics (PK), safety and tolerability of TNO155.
The results of this study will guide the Novartis recommendation regarding whether or not a dose adjustment may be needed when treating patients with hepatic impairment.

Start Date06 Mar 2025

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

[+1]

100 Clinical Results associated with ICON plc

0 Patents (Medical) associated with ICON plc

527

Literatures (Medical) associated with ICON plc

01 May 2025·Diabetic Medicine

Protocol for a feasibility and acceptability study for UK general population paediatric type 1 diabetes screening—the EarLy Surveillance for Autoimmune diabetes (ELSA) study

Article

Author: Lepley, Amanda ; Dias, Renuka P. ; Greenfield, Sheila M. ; Gardner, Christine ; Boiko, Olga ; Garstang, Joanna ; Barrett, Tim ; Zakia, Fatima ; Gkoutos, Georgios ; Shukla, David ; Narendran, Parth ; Quinn, Lauren M. ; Boardman, Felicity ; Faustini, Sian ; Connop, Clair ; Burt, Christine ; Dayan, Colin ; Litchfield, Ian ; Acharjee, Animesh ; Oram, Richard ; Richter, Alex G.

AbstractAimThe EarLy Surveillance for Autoimmune (ELSA) study aims to explore the feasibility and acceptability of UK paediatric general population screening for type 1 diabetes.MethodsWe aim to screen 20,000 children aged 3–13 years for islet‐specific autoantibodies through dried blood spot sample collection at home, hospital or community settings. Children with two or more autoantibodies are offered metabolic staging via oral glucose challenge testing. Feasibility assessments will compare recruitment modalities and uptake according to demographic factors (age, gender, ethnicity, level of deprivation and family history of diabetes) to determine optimal approaches for general population screening. The study is powered to identify 60 children (0.3%) with type 1 diabetes (stage 1–3). Parents are invited to qualitative interviews following ELSA completion (child screened negative or positive, single autoantibody or multiple, stage 1–3) to share their screening experience, strengths of the programme and any areas for improvement (acceptability assessments). Parents who decline screening or withdraw from participation are invited to interview to explore any concerns. Finally, we will interview professional stakeholders delivering the ELSA study to explore barriers and facilitators to implementation.ConclusionEarly detection of type 1 diabetes allows insulin treatment to be started sooner, avoids diagnosis as an emergency, gives families time to prepare and the opportunity to benefit from future prevention trials and treatments. ELSA will provide essential feasibility and acceptability assessments for UK general population screening to inform a future national screening programme for paediatric type 1 diabetes.

01 Mar 2025·Journal of Biological Chemistry

A predictive chromatin architecture nexus regulates transcription and DNA damage repair

Review

Author: Bhan, Sonali ; Nandi, Sandhik ; Pandita, Tej K ; Ramos, Keneth S ; Sarkar, Parthas S ; Kabiraj, Aindrila ; Das, Chandrima ; Sopori, Shreya ; Pandita, Shruti ; Dhar, Arti ; Tainer, John A ; Bhat, Audesh ; Kumar, Rakesh ; Pandita, Raj K

Genomes are blueprints of life essential for an organism's survival, propagation, and evolutionary adaptation. Eukaryotic genomes comprise of DNA, core histones, and several other nonhistone proteins, packaged into chromatin in the tiny confines of nucleus. Chromatin structural organization restricts transcription factors to access DNA, permitting binding only after specific chromatin remodeling events. The fundamental processes in living cells, including transcription, replication, repair, and recombination, are thus regulated by chromatin structure through ATP-dependent remodeling, histone variant incorporation, and various covalent histone modifications including phosphorylation, acetylation, and ubiquitination. These modifications, particularly involving histone variant H2AX, furthermore play crucial roles in DNA damage responses by enabling repair protein's access to damaged DNA. Chromatin also stabilizes the genome by regulating DNA repair mechanisms while suppressing damage from endogenous and exogenous sources. Environmental factors such as ionizing radiations induce DNA damage, and if repair is compromised, can lead to chromosomal abnormalities and gene amplifications as observed in several tumor types. Consequently, chromatin architecture controls the genome fidelity and activity: it orchestrates correct gene expression, genomic integrity, DNA repair, transcription, replication, and recombination. This review considers connecting chromatin organization to functional outcomes impacting transcription, DNA repair and genomic integrity as an emerging grand challenge for predictive molecular cell biology.

20 Feb 2025·Journal of Clinical Oncology

Large-Scale Pharmacogenomics Analysis of Patients With Cancer Within the 100,000 Genomes Project Combining Whole-Genome Sequencing and Medical Records to Inform Clinical Practice

Article

Author: Odhams, Christopher A. ; Elgar, Greg S. ; Daugherty, Louise C. ; Stuckey, Alex ; Murugaesu, Nirupa ; Caulfield, Mark J. ; Williams, Eleanor M. ; Sosinsky, Alona ; McDonagh, Ellen M. ; Rueda-Martin, Antonio ; Leong, Ivone U.S. ; Turner, Richard M. ; Moutsianas, Loukas ; Giess, Adam ; Walker, Susan ; Welland, Matthew J. ; Rhodes, Daniel J. ; Marks, Lauren ; Bleda, Marta ; Hill, Sue ; Ross, Paul J. ; Pickard, Alexandra ; Sanghvi, Sonali ; Cipriani, Valentina ; Brown, Matthew A. ; Chan, Georgia ; Barnes, Michael R. ; Scott, Richard H. ; Deans, Zandra ; Rendon, Augusto ; Foulger, Rebecca E. ; Snow, Catherine ; Niblock, Olivia ; Leigh, Sarah E.A. ; Cabrera, Claudia P. ; Pasko, Dorota

PURPOSEAs part of the 100,000 Genomes Project, we set out to assess the potential viability and clinical impact of reporting genetic variants associated with drug-induced toxicity for patients with cancer recruited for whole-genome sequencing (WGS) as part of a genomic medicine service.METHODS Germline WGS from 76,805 participants was analyzed for pharmacogenetic (PGx) variants in four genes ( DPYD , NUDT15 , TPMT , UGT1A1 ) associated with toxicity induced by five drugs used in cancer treatment (capecitabine, fluorouracil, mercaptopurine, thioguanine, irinotecan). Linking genomic data with prescribing and hospital incidence records, a phenome-wide association study (PheWAS) was performed to identify whether phenotypes indicative of adverse drug reactions (ADRs) were enriched in drug-exposed individuals with the relevant PGx variants. In a subset of 7,081 patients with cancer, DPYD variants were reported back to clinicians and outcomes were collected. RESULTS We identified clinically relevant PGx variants across the four genes in 62.7% of participants in our cohort. Extending this to annual prescription numbers in England for the drugs affected by these PGx variants, approximately 14,540 patients per year could potentially benefit from a reduced dose or alternative drug to reduce the risk of ADRs. Validating PGx associations in a real-world data set, we found a significant association between PGx variants in DPYD and toxicity-related phenotypes in patients treated with capecitabine or fluorouracil. Reported DPYD variants were deemed informative for clinical decision making in a majority of cases. CONCLUSIONReporting PGx variants from germline WGS relevant to patients with cancer alongside primary findings related to their cancer can be clinically informative, informing prescribing to reduce the risk of ADRs. Extending the range of actionable variants to those found in patients of non-European ancestry is important and will extend the potential clinical impact.

219

News (Medical) associated with ICON plc

11 Apr 2025

·www.prnewswire.com

Class Action Filed Against ICON Public Limited Company (ICLR) Seeking Recovery for Investors - Contact Levi & Korsinsky

NEW YORK, April 11, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in ICON Public Limited Company ("ICON " or the "Company") (NASDAQ: ICLR) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of ICON investors who were adversely affected by alleged securities fraud between July 27, 2023 and January 13, 2025. Follow the link below to get more information and be contacted by a member of our team: ICLR investors may also contact Joseph E. Levi, Esq. via email at [email protected] or by telephone at (212) 363-7500. CASE DETAILS: The filed complaint alleges that defendants made false statements and/or concealed that: (a)ICON was suffering from a material loss of business due to customer cost reduction measures and other widespread funding limitations impacting the Company's client base; (b)ICON's purported functional service provision and hybrid model offerings were insufficient to shield the Company from the adverse effects of a significant market downturn; (c) the requests for proposals ICON received from its biotechnology customers during the Class Period were used in substantial part as price discovery tools, and thus were not indicative of underlying client demand; (d)ICON's customers had canceled contracts, limited or reduced engagements, delayed clinical trial work, and/or failed to enter into new contracts with ICON for additional clinical trial work at historical rates once existing projects ended (or were scheduled to end) in 2024; (e)ICON's two largest customers were diversifying their clinical research organization providers away from the Company; (f)as a result of (a)-(e) above, ICON's reported net new business awards and book-to-bill metrics materially misrepresented client demand for ICON's services; and (g)as a result of (a)-(f) above, ICON was tracking materially below the 2024 revenue and EPS guidance issued during the Class Period and such guidance lacked a reasonable factual basis. WHAT'S NEXT? If you suffered a loss in ICON during the relevant time frame, you have until April 11, 2025 to request that the Court appoint you as lead plaintiff. Your ability to share in any recovery doesn't require that you serve as a lead plaintiff. NO COST TO YOU: If you are a class member, you may be entitled to compensation without payment of any out-of-pocket costs or fees. There is no cost or obligation to participate. WHY LEVI & KORSINSKY: Over the past 20 years, the team at Levi & Korsinsky has secured hundreds of millions of dollars for aggrieved shareholders and built a track record of winning high-stakes cases. Our firm has extensive expertise representing investors in complex securities litigation and a team of over 70 employees to serve our clients. For seven years in a row, Levi & Korsinsky has ranked in ISS Securities Class Action Services' Top 50 Report as one of the top securities litigation firms in the United States. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 17th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Patent Infringement

08 Apr 2025

·www.prnewswire.com

Shareholders that lost money on ICON Public Limited Company(ICLR) should contact Levi & Korsinsky about pending Class Action - ICLR

NEW YORK, April 8, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in ICON Public Limited Company ("ICON " or the "Company") (NASDAQ: ICLR) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of ICON investors who were adversely affected by alleged securities fraud between July 27, 2023 and January 13, 2025. Follow the link below to get more information and be contacted by a member of our team: ICLR investors may also contact Joseph E. Levi, Esq. via email at [email protected] or by telephone at (212) 363-7500. CASE DETAILS: The filed complaint alleges that defendants made false statements and/or concealed that: (a)ICON was suffering from a material loss of business due to customer cost reduction measures and other widespread funding limitations impacting the Company's client base; (b)ICON's purported functional service provision and hybrid model offerings were insufficient to shield the Company from the adverse effects of a significant market downturn; (c) the requests for proposals ICON received from its biotechnology customers during the Class Period were used in substantial part as price discovery tools, and thus were not indicative of underlying client demand; (d)ICON's customers had canceled contracts, limited or reduced engagements, delayed clinical trial work, and/or failed to enter into new contracts with ICON for additional clinical trial work at historical rates once existing projects ended (or were scheduled to end) in 2024; (e)ICON's two largest customers were diversifying their clinical research organization providers away from the Company; (f)as a result of (a)-(e) above, ICON's reported net new business awards and book-to-bill metrics materially misrepresented client demand for ICON's services; and (g)as a result of (a)-(f) above, ICON was tracking materially below the 2024 revenue and EPS guidance issued during the Class Period and such guidance lacked a reasonable factual basis. WHAT'S NEXT? If you suffered a loss in ICON during the relevant time frame, you have until April 11, 2025 to request that the Court appoint you as lead plaintiff. Your ability to share in any recovery doesn't require that you serve as a lead plaintiff. NO COST TO YOU: If you are a class member, you may be entitled to compensation without payment of any out-of-pocket costs or fees. There is no cost or obligation to participate. WHY LEVI & KORSINSKY: Over the past 20 years, the team at Levi & Korsinsky has secured hundreds of millions of dollars for aggrieved shareholders and built a track record of winning high-stakes cases. Our firm has extensive expertise representing investors in complex securities litigation and a team of over 70 employees to serve our clients. For seven years in a row, Levi & Korsinsky has ranked in ISS Securities Class Action Services' Top 50 Report as one of the top securities litigation firms in the United States. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 17th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Patent Infringement

04 Apr 2025

·www.prnewswire.com

ICON Public Limited Company Securities Fraud Class Action Lawsuit Pending: Contact Levi & Korsinsky Before April 11, 2025 to Discuss Your Rights - ICLR

NEW YORK, April 4, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in ICON Public Limited Company ("ICON " or the "Company") (NASDAQ: ICLR) of a class action securities lawsuit. CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of ICON investors who were adversely affected by alleged securities fraud between July 27, 2023 and October 23, 2024. Follow the link below to get more information and be contacted by a member of our team: ICLR investors may also contact Joseph E. Levi, Esq. via email at [email protected] or by telephone at (212) 363-7500. CASE DETAILS: The filed complaint alleges that defendants made false statements and/or concealed that: (a)ICON was suffering from a material loss of business due to customer cost reduction measures and other widespread funding limitations impacting the Company's client base; (b)ICON's purported functional service provision and hybrid model offerings were insufficient to shield the Company from the adverse effects of a significant market downturn; (c) the requests for proposals ICON received from its biotechnology customers during the Class Period were used in substantial part as price discovery tools, and thus were not indicative of underlying client demand; (d)ICON's customers had canceled contracts, limited or reduced engagements, delayed clinical trial work, and/or failed to enter into new contracts with ICON for additional clinical trial work at historical rates once existing projects ended (or were scheduled to end) in 2024; (e)ICON's two largest customers were diversifying their clinical research organization providers away from the Company; (f)as a result of (a)-(e) above, ICON's reported net new business awards and book-to-bill metrics materially misrepresented client demand for ICON's services; and (g)as a result of (a)-(f) above, ICON was tracking materially below the 2024 revenue and EPS guidance issued during the Class Period and such guidance lacked a reasonable factual basis. WHAT'S NEXT? If you suffered a loss in ICON during the relevant time frame, you have until April 11, 2025 to request that the Court appoint you as lead plaintiff. Your ability to share in any recovery doesn't require that you serve as a lead plaintiff. NO COST TO YOU: If you are a class member, you may be entitled to compensation without payment of any out-of-pocket costs or fees. There is no cost or obligation to participate. WHY LEVI & KORSINSKY: Over the past 20 years, the team at Levi & Korsinsky has secured hundreds of millions of dollars for aggrieved shareholders and built a track record of winning high-stakes cases. Our firm has extensive expertise representing investors in complex securities litigation and a team of over 70 employees to serve our clients. For seven years in a row, Levi & Korsinsky has ranked in ISS Securities Class Action Services' Top 50 Report as one of the top securities litigation firms in the United States. CONTACT: Levi & Korsinsky, LLP Joseph E. Levi, Esq. Ed Korsinsky, Esq. 33 Whitehall Street, 17th Floor New York, NY 10004 [email protected] Tel: (212) 363-7500 Fax: (212) 363-7171 SOURCE Levi & Korsinsky, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Patent Infringement

100 Deals associated with ICON plc

100 Translational Medicine associated with ICON plc

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Pipeline

Pipeline Snapshot as of 08 May 2025

The statistics for drugs in the Pipeline is the current organization and its subsidiaries are counted as organizations,Early Phase 1 is incorporated into Phase 1, Phase 1/2 is incorporated into phase 2, and phase 2/3 is incorporated into phase 3

Phase 1 Clinical

Other

Current Projects

Drug(Targets)	Indications	Global Highest Phase

JCXH-105	Herpes Zoster More	Phase 1
BMS-986196 ( BTK )	Multiple Sclerosis More	Phase 1
MMV367	Malaria, Falciparum More	Phase 1
Sulfaphenazole ( DHPS )	Bacterial Infections More	Withdrawn
Warfarin Potassium ( VKOR )	-	Withdrawn

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