Autoimmune hemolytic anemia

HUTCHMED Initiates Registration Stage of the ESLIM-02 Phase II/III Trial of Sovleplenib for Warm Antibody Autoimmune Hemolytic Anemia in China

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Friday, March 22, 2024
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HONG KONG and SHANGHAI, China and FLORHAM PARK, N.J., March 22, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“ HUTCHMED ”) (Nasdaq/AIM:HCM; HKEX:13) today announces that it has initiated the registration stage of the Phase II/III clinical trial of sovleplenib in adult patients with warm antibody autoimmune hemolytic anemia (“wAIHA”) in China.

Key Points: 
  • HONG KONG and SHANGHAI, China and FLORHAM PARK, N.J., March 22, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“ HUTCHMED ”) (Nasdaq/AIM:HCM; HKEX:13) today announces that it has initiated the registration stage of the Phase II/III clinical trial of sovleplenib in adult patients with warm antibody autoimmune hemolytic anemia (“wAIHA”) in China.
  • This follows positive data from the proof-of-concept Phase II stage of the trial and subsequent consultation with the China National Medical Products Administration (“NMPA”).
  • If positive, the data from the trial may be used to support a future New Drug Application (“NDA”) filing.
  • The objective of the registration stage of the study is to confirm the safety and efficacy of sovleplenib in adult patients with wAIHA.

BioCryst R&D Day Highlights New Diversified Pipeline of First-in-Class/Best-in-Class Therapies with Five Programs Expected in Clinical Development in Next 24 Months

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Friday, November 3, 2023
GMG, BioCryst Pharmaceuticals, FDA, Research, Patient, Drug design, IgA nephropathy, Plasma, CAD, Lupus nephritis, KLK5, Genetic disorder, RESEARCH, C2, DME, Eculizumab, Infection, Injection, Skin, Diabetes, Autoantibody, Ryan Murphy, Edema, Netherton, R, Autoimmune hemolytic anemia, HAE, Kallikrein, Growth, Disease, Dehydration, Muscle weakness, Immune system, Hemolysis, Solubility, Netherton syndrome, Clinical trial, Paratriathlon, Eye, Ravulizumab, Cold agglutinin disease, PARK, IP, SPINK5, Fine chemical, Pharmaceutical industry, Vaccine

A live webcast of the event will be available online in the investors section of the company website at www.biocryst.com .

Key Points: 
  • A live webcast of the event will be available online in the investors section of the company website at www.biocryst.com .
  • The team is excited to share our approach, and our expanded pipeline of new programs, at our R&D Day,” said Jon Stonehouse, president and chief executive officer of BioCryst.
  • The disease is caused by the deficiency of a natural inhibitor (SPINK5) of KLK5, a serine protease responsible for regulating skin shedding.
  • Netherton syndrome can be life threatening, especially during infancy when patients are vulnerable to dehydration and recurrent infections.

Immunovant Reports Financial Results and Recent Business Updates for the Quarter Ended December 31, 2022

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Friday, February 3, 2023
IPR, Research, WAIHA, IND, Common stock, D, Calendar, Graves' ophthalmopathy, Degenerative disease, TED, Clinical trial, Autoimmune hemolytic anemia, CIDP, Information technology, Pharmaceutical industry, Airline

R&D Expenses: Research and development expenses were $42.3 million for the three months ended December 31, 2022, compared to $29.8 million for the three months ended December 31, 2021.

Key Points: 
  • R&D Expenses: Research and development expenses were $42.3 million for the three months ended December 31, 2022, compared to $29.8 million for the three months ended December 31, 2021.
  • G&A Expenses: General and administrative expenses were $11.8 million for the three months ended December 31, 2022, compared to $11.5 million for the three months ended December 31, 2021.
  • Financial Highlights for Fiscal Nine Months Ended December 31, 2022:
    R&D Expenses: Research and development expenses were $108.4 million for the nine months ended December 31, 2022, compared to $69.8 million for the nine months ended December 31, 2021.
  • Net loss for the nine months ended December 31, 2022 and 2021 included $24.8 million and $22.4 million, respectively, related to non-cash stock-based compensation expense.

Annexon Highlights Strategic Focus to Advance Four Flagship Complement Programs through Late-Stage Development and Progress Across Three Therapeutic Franchises

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Sunday, January 8, 2023
Well, Severe cognitive impairment, SAD, Autoimmune disease, Complement component 1q, Autoimmune hemolytic anemia, Disease, Growth, LN, Plasma, Medicine, Neurodegeneration, Investment, The First Half of My Life, GA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Brain, HIV disease progression rates, Lupus nephritis, HD, Nephritis, AFL, Physician, Eye, Pivotal trial, FDA, Autoimmunity, Geographic atrophy, GBS, Anemia, Food, ALS, Continuous Progress Mathematics, Time-invariant system, Therapy, Webcast, Nerve, Hemolysis, CAD, MAD, Multifocal motor neuropathy, Cold agglutinin disease, Nerve Damage, Patient, Ophthalmology, MMN, Conference, Pharmaceutical industry, Waiha, Amyotrophic lateral sclerosis

BRISBANE, Calif., Jan. 08, 2023 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today reported progress across its broad portfolio of complement therapies and outlined its focus on four flagship programs to support its advancement to a late-stage biopharmaceutical company developing first-in-class treatments for complement-mediated diseases of the body, brain and eye.

Key Points: 
  • Annexon has prioritized resources and execution of late-stage development of its four flagship programs: Guillain-Barré syndrome (GBS), Huntington’s disease (HD), geographic atrophy (GA) and its first-in-kind oral small molecule, ANX1502.
  • Our mission is to free the body, brain and eye from diseases driven by the classical complement cascade,” said Douglas Love, president and CEO of Annexon.
  • Following an assessment of the market opportunity in wAIHA and a range of additional autoimmune indications, Annexon has determined not to advance development in wAIHA.
  • A replay of the webcast will be archived on the Annexon website for 30 days following the presentation.

HUTCHMED Initiates a Phase II/III Trial of Sovleplenib for Warm Antibody Autoimmune Hemolytic Anemia in China

Retrieved on: 
Monday, October 10, 2022
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HONG KONG and SHANGHAI, China and FLORHAM PARK, N.J., Oct. 10, 2022 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited ( HUTCHMED ) (Nasdaq/AIM:HCM; HKEX:13) today announces that it has initiated a Phase II/III trial of sovleplenib in adult patients with warm antibody autoimmune hemolytic anemia (wAIHA) in China.

Key Points: 
  • HONG KONG and SHANGHAI, China and FLORHAM PARK, N.J., Oct. 10, 2022 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited ( HUTCHMED ) (Nasdaq/AIM:HCM; HKEX:13) today announces that it has initiated a Phase II/III trial of sovleplenib in adult patients with warm antibody autoimmune hemolytic anemia (wAIHA) in China.
  • The Phase II stage of the study is to evaluate the safety and preliminary efficacy of sovleplenib in adult patients with wAIHA.
  • If results of the Phase II stage are positive, the Phase III stage will be initiated to confirm such efficacy and safety.
  • Sovleplenib is a novel, investigational, selective small molecule inhibitor for oral administration targeting the spleen tyrosine kinase, also known as Syk.

Rigel Announces Top-line Results from FORWARD Phase 3 Clinical Trial of Fostamatinib in Patients with Warm Autoimmune Hemolytic Anemia

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Wednesday, June 8, 2022
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SOUTH SAN FRANCISCO, Calif., June 8, 2022 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced top-line efficacy and safety data from the FORWARD Phase 3 clinical trial, a global, multi-center, randomized, double-blind, placebo-controlled trial of fostamatinib in patients with warm autoimmune hemolytic anemia (wAIHA). The trial did not demonstrate statistical significance in the primary efficacy endpoint of durable hemoglobin response in the overall study population. In a post-hoc regional analysis of U.S., Canadian, Australian, and Western European trial sites, patients treated with fostamatinib had a favorable durable hemoglobin response compared to placebo, whereas in the Eastern European trial sites patients did not. Rigel plans to continue analyzing the data to understand the geographical differences in patient disease characteristics and outcomes and discuss these findings with the U.S. Food and Drug Administration (FDA).

Key Points: 
  • On behalf of the entire Rigel team, we are grateful to the patients, their caregivers, and the healthcare professionals who participated in the trial."
  • Of the 90 patients that completed the FORWARD Phase 3 study, 71 (79%) enrolled in the open-label extension study.
  • Fostamatinib is currently being evaluated in a Phase 3 randomized, double-blind, placebo-controlled clinical study in 90 patients with wAIHA who have failed at least one prior treatment.
  • Fostamatinib is currently being studied in a Phase 3 clinical trial( NCT03764618 )for the treatment of warm autoimmune hemolytic anemia (wAIHA)2; a Phase 3 clinical trial( NCT04629703 )for the treatment of hospitalizedhigh-riskpatients withCOVID-192; and an NIH/NHLBI-sponsored Phase 3 clinical trial (ACTIV-4 Host Tissue Trial, NCT04924660 ) for the treatment of COVID-19 in hospitalized patients.

Knight Therapeutics Enters into Exclusive License and Supply Agreements with Rigel Pharmaceuticals to Commercialize Fostamatinib in Latin America

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Tuesday, May 24, 2022
People, CLR, Severe acute respiratory syndrome coronavirus 2, Platelet, Risk, Death, Shock, Organ dysfunction, Autoimmune hemolytic anemia, Thermoregulation, Waiha, Patient, Common, Antibody, Macrovascular disease, GUD, AIHA, Endothelial dysfunction, Therapy, Macrophage, Nature, COVID-19, Jean-Louis Vincent, TSX, Bruise, Intention, Infection, Partnership, Bleeding, ITP, SYK, Splenectomy, Pathology, Neutrophil extracellular traps, Acute kidney injury, Neutrophil, NETS, Monocyte, Museo-laboratorio della Civiltà Contadina (Museum-workshop of the Peasant Culture), Immune system, Steroid, Knight Therapeutics, Fostamatinib, CEO, Annual report, GLOBE, Pharmaceutical industry, Vaccine, Generic drug, Rigel, Uniting Medically Supervised Injecting Centre, Knight

MONTREAL, May 24, 2022 (GLOBE NEWSWIRE) -- Knight Therapeutics Inc. (TSX: GUD), a pan-American (ex-USA) specialty pharmaceutical company, today announced that it has entered into exclusive license and supply agreements with Rigel Pharmaceuticals granting Knight the rights to commercialize fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, in Latin America.

Key Points: 
  • MONTREAL, May 24, 2022 (GLOBE NEWSWIRE) -- Knight Therapeutics Inc. (TSX: GUD), a pan-American (ex-USA) specialty pharmaceutical company, today announced that it has entered into exclusive license and supply agreements with Rigel Pharmaceuticals granting Knight the rights to commercialize fostamatinib, an oral spleen tyrosine kinase (SYK) inhibitor, in Latin America.
  • In return, Knight receives exclusive rights to fostamatinib in all potential indications, including chronic ITP, wAIHA, and COVID-19 in Latin America.
  • We are excited to be working with Rigel to provide access to an innovative, first-in-class treatment option to patients across Latin America with chronic ITP.
  • TAVALISSE and TAVLESSE are registered trademarks of Rigel Pharmaceuticals, Inc.
    Investor Contact for Knight Therapeutics Inc.:

Annexon Biosciences Highlights Portfolio Progress and Key Anticipated Milestones, and Reports First Quarter 2022 Financial Results

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Monday, May 9, 2022
COVID-19, Amyotrophic lateral sclerosis, Forward-looking statement, Investment, Nerve Damage, HD, Private Securities Litigation Reform Act, Immune system, Brain, Nasdaq, GLOBE, ARCHER, Autoimmunity, GBS, Securities Exchange Act of 1934, Nephritis, Securities Act of 1933, Biomarker, Research, Eye, Geographic atrophy, CTA, Patient, Autoimmune disease, Complement component 1q, Program, Neurodegeneration, Severe cognitive impairment, Inflammation, Lupus nephritis, Drug Quality and Security Act, Degenerative disease, R, Waiha, Disease, Humour, IV, SEC, Terminology, G&A, Guillain, Autoimmune hemolytic anemia, ALS, Central nervous system, GA, Pharmaceutical industry, Vaccine, Risk management

BRISBANE, Calif., May 09, 2022 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today announced progress across its broad pipeline of product candidates and reported first quarter 2022 financial results.

Key Points: 
  • I am very proud of the strong execution by the Annexon team in advancing our classical complement platform across multiple indications driven by aberrant complement activity.
  • Research and development (R&D) expenses: R&D expenses were $27.0 million for the quarter ended March 31, 2022, compared to $20.7 million for the quarter ended March 31, 2021.
  • General and administrative (G&A) expenses: G&A expenses were $8.4 million for the quarter ended March 31, 2022, compared to $5.5 million for the quarter ended March 31, 2021.
  • Annexon is rigorously developing a pipeline of diversified product candidates across multiple mid- to late- stage clinical trials, with clinical data anticipated throughout 2022 and 2023.

FDA approves Enjaymo™ (sutimlimab-jome), first treatment for use in patients with cold agglutinin disease

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Friday, February 4, 2022
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Enjaymo is the only approved treatment to inhibit red blood cell destruction in CAD and help stop the chain reaction from the start.

Key Points: 
  • Enjaymo is the only approved treatment to inhibit red blood cell destruction in CAD and help stop the chain reaction from the start.
  • For the first time, we have a treatment that targets complement-mediated hemolysis, which is the underlying cause of the red blood cell destruction in many CAD patients.
  • Dosage interruptions due to an adverse reaction occurred in 17 percent (4/24) of patients who received Enjaymo.
  • Following the completion of the 26-week treatment period of CARDINAL (Part A), eligible patients continued to receive Enjaymo in an extension study.

Spleen Tyrosine Kinase Inhibitors Pipeline Market Research Report 2022 - ResearchAndMarkets.com

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Monday, January 31, 2022
Health, Pharmaceutical, Hematological Cancer Research Investment and Education Act, III, BCR, Autoimmune hemolytic anemia, Clinical trial, Neutrophil, Factor X, Discovery, Acute myeloid leukemia, Signal transduction, TRN, Tyrosine-protein kinase SYK, Macrophage, MEIS1, BTK, COVID-19, II, PI3K, Fostamatinib, HOXA9, Entospletinib, Phase II, SYK, News, Degenerative disease, B cell, FC, ENTO, Therapy, PTK, AML, Monocyte, TNF, Vaccine, Pharmaceutical industry, Spleen

The "Spleen tyrosine kinase inhibitors - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Spleen tyrosine kinase inhibitors - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • This "Spleen tyrosine kinase inhibitors - Pipeline Insight, 2022" report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Spleen tyrosine kinase inhibitors pipeline landscape.
  • Spleen tyrosine kinase (Syk) is a cytosolic non-receptor protein tyrosine kinase (PTK) and is mainly expressed in hematopoietic cells.
  • The companies which have their Spleen tyrosine kinase inhibitors drug candidates in the most advanced stage, i.e.