Hemophagocytic lymphohistiocytosis

Elixiron Immunotherapeutics Announces Orphan Drug Designation Granted to CSF-1R Inhibitor, EI-1071 for the Treatment of Idiopathic Pulmonary Fibrosis

Retrieved on: 
Tuesday, August 2, 2022

SAN FRANCISCO, Aug. 02, 2022 (GLOBE NEWSWIRE) -- Elixiron Immunotherapeutics, a clinical stage biotech company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its colony stimulating factor1 receptor (CSF1R) inhibitor, EI1071 for the treatment of idiopathic pulmonary fibrosis (IPF).

Key Points: 
  • SAN FRANCISCO, Aug. 02, 2022 (GLOBE NEWSWIRE) -- Elixiron Immunotherapeutics, a clinical stage biotech company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its colony stimulating factor1 receptor (CSF1R) inhibitor, EI1071 for the treatment of idiopathic pulmonary fibrosis (IPF).
  • Idiopathic pulmonary fibrosis is a chronic interstitial lung disease characterized by tissue fibrosis and progressive decline in lung function.
  • CSF1 signaling plays an important role in the regulation of pulmonary macrophages which drive inflammatory damage and tissue fibrosis in IPF.
  • CSF1R blockade with EI1071 shows promising therapeutic effects, ameliorating lung fibrosis and improving respiratory functions in preclinical models of IPF.

Elixiron Immunotherapeutics Receives Grant From the Alzheimer's Association to Trial Microglia-Targeting Immunotherapy

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Tuesday, July 12, 2022

This follows the successful completion of a first-in-human phase 1 study that was also made possible in part thanks to an earlier Alzheimer's Association Part the Cloud-Bill Gates Partnership program grant.

Key Points: 
  • This follows the successful completion of a first-in-human phase 1 study that was also made possible in part thanks to an earlier Alzheimer's Association Part the Cloud-Bill Gates Partnership program grant.
  • "If successful, this trial will support the advancement of a large-scale trial to see if EI-1071 can halt disease progression, to maintain cognitive function."
  • "Through a second grant, the Alzheimer's Association is excited to propel this treatmentavenue into a phase 2 study, and we look forward to the results of that trial."
  • Elixiron Immunotherapeutics is driven by an international team with a shared vision to produce next-generation immunotherapies for unmet needs in immunological and degenerative diseases.

Sobi Announces Launch of the INTO-HLH Registry, a Disease Registry for Patients with Hemophagocytic Lymphohistiocytosis (HLH)

Retrieved on: 
Tuesday, June 28, 2022

WALTHAM, Mass., June 28, 2022 (GLOBE NEWSWIRE) -- Sobi North America today announced the launch of the INTO-HLH Registry, a new disease registry for patients with hemophagocytic lymphohistiocytosis (HLH).

Key Points: 
  • WALTHAM, Mass., June 28, 2022 (GLOBE NEWSWIRE) -- Sobi North America today announced the launch of the INTO-HLH Registry, a new disease registry for patients with hemophagocytic lymphohistiocytosis (HLH).
  • The INTO-HLH Registry (Insight into the Natural history and Treatment Outcomes of Hemophagocytic Lymphohistiocytosis) will enroll pediatric and adult patients with HLH, a rare and life-threatening disease characterized by hyperinflammation affecting multiple organs of the body.
  • The INTO-HLH Registry is designed to define the natural history of HLH, including the full diagnostic and treatment journey that patients with HLH experience.
  • More broadly, we hope that INTO-HLH Registry will create a community of hope for physicians, scientists, patients, and patient organizations.

UPDATE -- Revive Therapeutics Expands Bucillamine Research to Treat Omicron Variant and Adds Inflammatory Markers to FDA Phase 3 Clinical Trial

Retrieved on: 
Friday, December 3, 2021

The Company supported recent research to explore the utility of thiol-based drugs under its sponsored research agreement with the University of California, San Francisco (UCSF) in the laboratory of Dr. John Fahy.

Key Points: 
  • The Company supported recent research to explore the utility of thiol-based drugs under its sponsored research agreement with the University of California, San Francisco (UCSF) in the laboratory of Dr. John Fahy.
  • There is evidence that Bucillamine inhibits pro-inflammatory cytokine production and transendothelial T-cell migration, both of which could further dampen disease course in COVID-19 (Horowitz LD.
  • Currently, the Company is exploring the use of Bucillamine for the potential treatment of infectious diseases, with an initial focus on severe influenza and COVID-19.
  • With its acquisition of Psilocin Pharma Corp., Revive is advancing the development of Psilocybin-based therapeutics in various diseases and disorders.

Revive Therapeutics Expands Bucillamine Research to Treat Omicron Variant and Adds Inflammatory Markers to FDA Phase 3 Clinical Trial

Retrieved on: 
Friday, December 3, 2021

The Company supported recent research to explore the utility of thiol-based drugs under its sponsored research agreement with the University of California, San Francisco (UCSF) in the laboratory of Dr. John Fahy.

Key Points: 
  • The Company supported recent research to explore the utility of thiol-based drugs under its sponsored research agreement with the University of California, San Francisco (UCSF) in the laboratory of Dr. John Fahy.
  • There is evidence that Bucillamine inhibits pro-inflammatory cytokine production and transendothelial T-cell migration, both of which could further dampen disease course in COVID-19 (Horowitz LD.
  • Currently, the Company is exploring the use of Bucillamine for the potential treatment of infectious diseases, with an initial focus on severe influenza and COVID-19.
  • With its acquisition of Psilocin Pharma Corp., Revive is advancing the development of Psilocybin-based therapeutics in various diseases and disorders.

HLH Awarded “Best for the World” Honoree for 2021

Retrieved on: 
Thursday, July 15, 2021

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

Retrieved on: 
Wednesday, May 19, 2021

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.

AB2 Bio to Participate in Orphan Disease Panel Discussion at the LifeSci Partners 10th Annual Healthcare Corporate Access Event

Retrieved on: 
Tuesday, January 5, 2021

The panel is entitled Rare Advantages and Unique Challenges Faced by Orphan Companies.

Key Points: 
  • The panel is entitled Rare Advantages and Unique Challenges Faced by Orphan Companies.
  • AB2 Bio is building a late-stage clinical pipeline with Tadekinig alfa, a novel IL-18 binding protein with established clinical proof-of-concept in three, life-threatening orphan autoinflammatory indications.
  • Tadekinig alfa is currently in late-stage development for the treatment of severe orphan autoinflammatory diseases, including primary and secondary HLH and Stills disease.
  • AB2 Bio is building a late-stage clinical pipeline with Tadekinig alfa, a novel IL-18 binding protein with established clinical proof-of-concept in three, life-threatening orphan autoinflammatory indications.

Emapalumab's efficacy confirmed by sensitivity analysis presented at ESID2020

Retrieved on: 
Saturday, October 17, 2020

"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

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.