Histiocytosis

Electra Therapeutics highlights ELA026 Phase 1b study in sHLH and presents preclinical data at ASH Annual Meeting

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Monday, December 12, 2022
Oncology, Health, Stem Cells, Clinical Trials, Pharmaceutical, Biotechnology, ADCP, Therapy, Histiocytosis, Lymphocyte, Primate, MD, Monocyte, Immunotherapy, Infection, Poster, PK, Pharmacology, Protein, Society, SIRP, Biomarker, Patient, Trial of the century, Cancer, Multiple organ dysfunction syndrome, Splenomegaly, Myelocyte, Death, Incidence, Autoimmune disease, Roger Hawkins (drummer), Clinical trial, Safety, Hepatomegaly, ADCC, Immunosuppression, ASH, PD, Cytopenia, Pharmaceutical industry, Vaccine, Medical device

Electra also announced today that a Phase 1b global study of ELA026 in sHLH patients has been initiated and is ongoing (ClinicalTrials.gov identifier: NCT05416307 ).

Key Points: 
  • Electra also announced today that a Phase 1b global study of ELA026 in sHLH patients has been initiated and is ongoing (ClinicalTrials.gov identifier: NCT05416307 ).
  • The Phase 1b study is highlighted as a clinical trial in progress poster at ASH, titled A Phase 1b study of ELA026 in patients with secondary hemophagocytic lymphohistiocytosis.
  • The Phase 1b study is an open-label, single-arm, multicenter study which will evaluate the safety and efficacy of ELA026, assess biomarkers and identify a dose for Phase 2/3 testing.
  • We have created a robust design for this Phase 1b study with the aim of progressing rapidly toward a Phase 2/3 study so we can accelerate ELA026 as a potential treatment option for sHLH patients in need.

HLH Awarded “Best for the World” Honoree for 2021

Retrieved on: 
Thursday, July 15, 2021
Histiocytosis, Hemophagocytic lymphohistiocytosis, HLH

B Corporations are accelerating a global culture shift to redefine success in business and build a more inclusive sustainable economy.

Key Points: 
  • B Corporations are accelerating a global culture shift to redefine success in business and build a more inclusive sustainable economy.
  • HLH earned a score in the top 5 percent of all B Corporations in its size and category.
  • About HLH: HLH uses state-of-the-art technology backed by decades of experience to preserve thousands of years of history, culture, and natural resources.
  • For more on Legacy Tree Planting Tours visit www.HawaiianLegacyTours.com and for more on sponsoring a Legacy Tree, visit www.LegacyTrees.org .

GenSight Biologics Appoints Country Leads for France, Germany and the UK in Preparations for LUMEVOQ® Commercial Launch

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Wednesday, May 19, 2021
Biotechnology, Pharmaceutical, Health, Companies, Histiocytosis, Blood disorders, Medical specialties, Hemophagocytic lymphohistiocytosis, Health, HLH, Hematologic disease, Swedish Orphan Biovitrum, Genzyme, Sanofi

The European Medicines Agency decision is expected in H1 2022, with the company expecting to achieve UK approval in the same time frame.

Key Points: 
  • The European Medicines Agency decision is expected in H1 2022, with the company expecting to achieve UK approval in the same time frame.
  • Early engagement with authorities and payers is critical in markets such as Germany and the UK, where commercial sales can be generated before the reimbursed price is finalized.
  • As Head of the Rare Diseases & Rare Blood Disorders unit at Sanofi Genzyme, she oversaw the successful launch of a number of rare disease products developed or acquired by the company.
  • He led the commercial launch of Sobi\xe2\x80\x99s treatments for hemophilia in the UK and Ireland and the launch planning for primary hemophagocytic lymphohistiocytosis (HLH) and thrombocytopenia.

Emapalumab's efficacy confirmed by sensitivity analysis presented at ESID2020

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Saturday, October 17, 2020
Medical specialties, Health, Medicine, Histiocytosis, Hemophagocytic lymphohistiocytosis, Clinical trials, Medical statistics, Medical terminology, Clinical endpoint, Response rate, Glucocorticoid

"We remain committed to making emapalumab accessible to patients affected by this severe condition around the world."

Key Points: 
  • "We remain committed to making emapalumab accessible to patients affected by this severe condition around the world."
  • A high unmet medical need exists among these patients despite today's standard-of-care therapy, glucocorticoids and etoposide, with or without cyclosporine.
  • The pivotal study used clinically objective response criteria to define the primary endpoint of overall response rate to emapalumab in primary HLH.
  • The sensitivity analysis supports the use of the clinically objective ORR as a primary endpoint in studies of primary HLH.

Expression Therapeutics Announces Success in Developing a Stem Cell Lentiviral Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH)

Retrieved on: 
Monday, August 24, 2020
Branches of biology, Biology, Life sciences, Biotechnology, Applied genetics, Genetic engineering, Medical genetics, Hemophagocytic lymphohistiocytosis, Histiocytosis, Gene therapy, Therapy, Lentivirus

Expression Therapeutics has developed a promising and potentially curative gene therapy candidate for familial hemophagocytic lymphohistiocytosis (HLH) type 3 (FHL3).

Key Points: 
  • Expression Therapeutics has developed a promising and potentially curative gene therapy candidate for familial hemophagocytic lymphohistiocytosis (HLH) type 3 (FHL3).
  • "We are excited to announce this expansion of our gene and cell therapy pipeline beyond our lead stem cell lentiviral gene therapy candidate for hemophilia A that is entering Phase 1 clinical testing.
  • Proof of concept for stem cell lentiviral gene therapy of FHL3 was demonstrated using primary patient cells and a genetic mouse model of FHL3.
  • Third, with stem cell-based lentiviral gene therapy there will be no wait time to find a sufficiently human leukocyte antigen-matched donor," said Trent Spencer, Ph.D., President of Expression Therapeutics.

Hemophagocytic Lymphohistiocytosis (HLH) Markets, 2017-2030 - Epidemiology, Drugs, Pipeline, Reimbursement Scenario, Competitive Intelligence

Retrieved on: 
Wednesday, July 29, 2020
Histiocytosis, Hemophagocytic lymphohistiocytosis, HLH, HPLH1

The Hemophagocytic Lymphohistiocytosis epidemiology chapters provide insights about historical and current Hemophagocytic Lymphohistiocytosis patient pool and forecasted trend for every seven major countries.

Key Points: 
  • The Hemophagocytic Lymphohistiocytosis epidemiology chapters provide insights about historical and current Hemophagocytic Lymphohistiocytosis patient pool and forecasted trend for every seven major countries.
  • Hemophagocytic Lymphohistiocytosis epidemiology is segmented by Incidence of Hemophagocytic Lymphohistiocytosis (Familial and secondary HLH), Familial HLH cases by Mutation Types and Secondary HLH cases by Etiologies.
  • Drug chapter segment of the Hemophagocytic Lymphohistiocytosis report encloses the detailed analysis of Hemophagocytic Lymphohistiocytosis pipeline drug.
  • The report covers the detailed information of Hemophagocytic Lymphohistiocytosis collaborations, acquisition and merger, licensing, patent details and other information for Hemophagocytic Lymphohistiocytosis emerging therapies.

Hemophagocytic Lymphohistiocytosis Market Insights, Epidemiology, and Forecast to 2030 - ResearchAndMarkets.com

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Tuesday, July 28, 2020
Infectious diseases, Pharmaceutical, Health, Histiocytosis, Hemophagocytic lymphohistiocytosis, HLH, HPLH1

The "Hemophagocytic Lymphohistiocytosis Market Insights, Epidemiology, and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Hemophagocytic Lymphohistiocytosis Market Insights, Epidemiology, and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • The Hemophagocytic Lymphohistiocytosis epidemiology chapters provide insights about historical and current Hemophagocytic Lymphohistiocytosis patient pool and forecasted trend for every seven major countries.
  • Hemophagocytic Lymphohistiocytosis epidemiology is segmented by Incidence of Hemophagocytic Lymphohistiocytosis (Familial and secondary HLH), Familial HLH cases by Mutation Types and Secondary HLH cases by Etiologies.
  • The Drug chapter segment of the Hemophagocytic Lymphohistiocytosis report encloses the detailed analysis of Hemophagocytic Lymphohistiocytosis pipeline drug.

Results From Pivotal Phase 2/3 Study of Emapalumab in Patients With Primary HLH Published in New England Journal of Medicine

Retrieved on: 
Thursday, May 7, 2020
Health, Drugs, Hemophagocytic lymphohistiocytosis, Emapalumab, HLH, Clinical trial, Swedish Orphan Biovitrum, Histiocytosis

STOCKHOLM, May 7, 2020 /PRNewswire/ -- Sobi announced today that the results from the pivotal phase 2/3 study evaluating the efficacy and safety of emapalumab in patients with primary haemophagocytic lymphohistiocytosis (HLH) were published in the New England Journal of Medicine on 7 May 2020.

Key Points: 
  • STOCKHOLM, May 7, 2020 /PRNewswire/ -- Sobi announced today that the results from the pivotal phase 2/3 study evaluating the efficacy and safety of emapalumab in patients with primary haemophagocytic lymphohistiocytosis (HLH) were published in the New England Journal of Medicine on 7 May 2020.
  • Emapalumab is the first therapy approved by the US Food & Drug Administration (FDA) for primary HLH and is under review by the European Medicines Agency (EMA).
  • "The results further advance our understanding of primary HLH and the role of interferon gamma in its pathogenesis.
  • This pivotal clinical study is the first study in primary HLH to prospectively assess and report treatment responses using predefined comprehensive objective clinical and laboratory criteria.

AB2 Bio Appoints Michael Soldan CEO

Retrieved on: 
Tuesday, March 3, 2020
Hemophagocytic lymphohistiocytosis, HLH, Medical specialties, Fresenius, École Polytechnique Fédérale de Lausanne, NLRC4, Histiocytosis, Soldan, Economy of Germany

Prior to Fresenius Kabi, Dr. Soldan was with Merck Group, where he spent two years heading the biosimilars business.

Key Points: 
  • Prior to Fresenius Kabi, Dr. Soldan was with Merck Group, where he spent two years heading the biosimilars business.
  • I am very pleased to welcome Michael Soldan to AB2 Bio, where he will bring the benefit of his impressive and wide-ranging career in the pharmaceuticals industry.
  • AB2 Bio is conducting a pivotal Phase 3 clinical trial of Tadekinigalfa in primary, interleukin-18 driven Hemophagocytic Lymphohistiocytosis (HLH) patientswith NLRC4 mutation or XIAP deficiency.
  • AB2 Bio Ltd ( http://www.ab2bio.com/ ) is a private advanced clinical-stage biotech company located in the Innovation Park at the Ecole polytechnique federale de Lausanne (EPFL), Switzerland.

Data to be Highlighted at the American Society of Hematology 2019 Annual Meeting

Retrieved on: 
Thursday, December 5, 2019
Health, Drugs, Breakthrough therapy, Monoclonal antibodies, Hemophagocytic lymphohistiocytosis, Histiocytosis, Emapalumab, Food and Drug Administration, Pediatric Trials Network, Phases of clinical research

The adaptive design of a study evaluating the efficacy, safety and pharmacokinetics of emapalumab in adult patients with secondary HLH will be presented.

Key Points: 
  • The adaptive design of a study evaluating the efficacy, safety and pharmacokinetics of emapalumab in adult patients with secondary HLH will be presented.
  • The initial phase will enrol 10 adult patients and the study is expected to open later this year.
  • The latest clinical data and ongoing trials with the potential to impact the care of patients with HLH will also be reviewed.
  • The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899).