Scleroderma

Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Finger, Growth, Face, Blood, Disease, Inflammation, Patient, Committee, Knowledge, Regulation, Systemic scleroderma, EMA, IRIS, Protein, Scleroderma, CB2, European, Collagen, COMP, Connective tissue, Skin, Heart, Sponsor, European Commission, Immune system, The, Safety, Swelling, Marketing, Pharmaceutical industry

Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn

Key Points: 


Orphan designation: (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn

Cabaletta Bio Announces FDA Granted Orphan Drug Designation to CABA-201 for Treatment of Systemic Sclerosis

Retrieved on: 
Wednesday, March 20, 2024
ODD, Diagnosis, Face, Survival, Disease, Patient, Scleroderma, Clinical trial, Skin, Systemic scleroderma, Marketing, FDA, Food, Pharmaceutical industry

PHILADELPHIA, March 20, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of systemic sclerosis (SSc).

Key Points: 
  • PHILADELPHIA, March 20, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the treatment of systemic sclerosis (SSc).
  • CABA-201 is in development as a potential treatment for autoimmune diseases driven by B cells.
  • “Based on the role of B cells and the recently published academic clinical data with CD19-CAR T therapy in systemic sclerosis, we believe CABA-201 may transform the treatment for systemic sclerosis.
  • This designation qualifies Cabaletta for certain incentives, which may include partial tax credit for clinical trial expenditures, waived user fees and potential eligibility for seven years of marketing exclusivity.

Corvus Pharmaceuticals Provides Business Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, March 19, 2024
Atopic dermatitis, Head, T helper cell, HNSCC, Patient, PTCL, Lung cancer, Ipilimumab, Asthma, CTCL, ALCL, Fungal infection, Pulmonary fibrosis, Lymphocyte, RCC, Clinical trial, CRS, Marketing, B-cell lymphoma, Research, PR, PTCL-NOS, Psoriasis, T-cell lymphoma, ASH, Webcast, Immunomodulation, Pembrolizumab, PRS, Exposition, Cancer, Nivolumab, Safety, Scleroderma, Food, NSCLC, Teva Pharmaceuticals, Modified, Lymphoma, Knowledge, Allergy, Immunodeficiency, Time management, Peripheral, Poster, FDA, ITK, Conference call, Kidney cancer, T helper 17 cell, Society, Preprint, BioRxiv, Autoimmunity, Physician, Total, Pharmaceutical industry

BURLINGAME, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS) (GLOBAL NEWSWIRE), a clinical-stage biopharmaceutical company, today provided a business update and reported financial results for the fourth quarter and year ended December 31, 2023.

Key Points: 
  • ET / 1:30 p.m. PT
    BURLINGAME, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (Corvus or the Company) (Nasdaq: CRVS) (GLOBAL NEWSWIRE), a clinical-stage biopharmaceutical company, today provided a business update and reported financial results for the fourth quarter and year ended December 31, 2023.
  • We have several exciting opportunities in immunology, including plans for a randomized, placebo controlled clinical trial of soquelitinib in atopic dermatitis.
  • Mr. Arcara previously served as senior vice president, head global marketing & portfolio and strategy for the innovative medicines and biosimilars business at Teva Pharmaceuticals.
  • ET (1:30 p.m. PT), during which time management will provide a business update and discuss the fourth quarter and full year 2023 financial results.

Early Pulmonary Fibrosis Detection Holds the Key to Better Health Outcomes for Canadians Living with COVID-19 and Rheumatoid Arthritis

Retrieved on: 
Wednesday, February 28, 2024
Rare disease, Oxygen, Connective tissue disease, Research, Scleroderma, Physician, St. Joseph's Health Centre, Lupus, Crackle, Myositis, Pulmonary fibrosis, Life, CPFF, COVID-19, Signs, Rheumatoid arthritis, COVID, Patient, Disease, Diagnosis, Connective tissue, Interview, Face, Pharmaceutical industry

Canadians who have had COVID-19, and other chronic diseases, are experiencing Pulmonary Fibrosis (PF) at significantly higher rates than others, seemingly providing a causal link that researchers are investigating.

Key Points: 
  • Research is shedding light on the growing rate of pulmonary fibrosis (PF) being found in people suffering from long-term COVID-19.
  • One study, Post COVID-19 pulmonary fibrosis; a meta-analysis study , found that almost 45% (44.9%) of study participants began suffering from PF after contracting COVID-19.
  • Canadians suffering from long COVID are encouraged to watch for the early signs of pulmonary fibrosis, a disease that is growing in this population.
  • Dr. Janet Pope's webinar includes explanations for both physicians and thousands of people living with connective tissue diseases of what pulmonary fibrosis symptoms to watch for.

Press Release: New Phase 2b results for amlitelimab support potential for best-in-class maintenance of response in atopic dermatitis

Retrieved on: 
Monday, March 11, 2024
Eosinophil, Fever, Week, Headache, OX40L, Conjunctivitis, Scleroderma, Teaching hospital, Atopic dermatitis, Upper respiratory tract infection, Part, Asthma, TARC, Conference, AAD, Safety, Biomarker, Part Two, Hidradenitis suppurativa, IGA, Patient, Inflammation, Chills, Moa, Serum, American Academy, Pharmaceutical industry

The safety profile was consistent with Part 1 of the study with amlitelimab being well-tolerated and no new safety concerns identified.

Key Points: 
  • The safety profile was consistent with Part 1 of the study with amlitelimab being well-tolerated and no new safety concerns identified.
  • Overall rates of treatment-emergent adverse events (TEAEs) were 69.8% for continued amlitelimab treatment, 71.9% for the amlitelimab withdrawal-arm and 66.7% for placebo.
  • TEAEs more commonly observed included headache (11.6% amlitelimab continuation, 3.9% amlitelimab withdrawal, 6.7% placebo), upper respiratory tract infection (9.3% amlitelimab continuation, 5.5% amlitelimab withdrawal, 20% placebo).
  • Amlitelimab is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.

aTyr Pharma Announces Expanded Access Program (EAP) for EFZO-FIT™ Clinical Trial Participants

Retrieved on: 
Wednesday, February 21, 2024
SAN, Sarcoidosis, Scar, Small RNA, Pi, ATyr Pharma, FDA, Inflammation, Food, Scleroderma, Risk, Myelocyte, Health, Safety, Principal investigator, Expanded access, Neuropilin, Systemic scleroderma, ILD, OLE, Patient, Fibrosis, Clinical trial, EAP, Pharmaceutical industry

SAN DIEGO, Feb. 21, 2024 (GLOBE NEWSWIRE) --  aTyr Pharma, Inc. (Nasdaq: LIFE) (aTyr or the Company), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced plans to initiate an Individual Patient Expanded Access Program (EAP) for its lead therapeutic candidate, efzofitimod, for patients with pulmonary sarcoidosis. The Individual Patient EAP is intended to allow access for patients who complete the Phase 3 EFZO-FIT™ study and wish to receive treatment with efzofitimod outside of the clinical trial.

Key Points: 
  • Individual Patient EAP allows access to efzofitimod for patients who complete the Phase 3 EFZO-FIT™ study in pulmonary sarcoidosis.
  • Company initiating program based on blinded EFZO-FIT™ study investigator and patient participant feedback.
  • The Individual Patient EAP is intended to allow access for patients who complete the Phase 3 EFZO-FIT™ study and wish to receive treatment with efzofitimod outside of the clinical trial.
  • “We are pleased to make efzofitimod available to patients beyond the duration of the EFZO-FIT™ clinical trial through this Individual Patient EAP,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr.

Certa Therapeutics’ FT011 Granted US FDA Fast Track for the Treatment of Systemic Sclerosis

Retrieved on: 
Monday, February 19, 2024
Fast Track, EMA, Biotechnology, Scleroderma, FDA, Food, Treatment, Marker beacon, Therapy, GPCR, Patient, Acceleration, Fibrosis, Clinical trial, Disease, NDA, Pharmaceutical industry

MELBOURNE, Australia, Feb. 19, 2024 (GLOBE NEWSWIRE) -- Certa Therapeutics (Certa), a biotechnology company developing innovative precision therapies for patients with inflammatory and fibrotic diseases, today announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for its investigational therapy FT011 for the treatment of systemic sclerosis (scleroderma), having previously granted Orphan Drug Designation.

Key Points: 
  • MELBOURNE, Australia, Feb. 19, 2024 (GLOBE NEWSWIRE) -- Certa Therapeutics (Certa), a biotechnology company developing innovative precision therapies for patients with inflammatory and fibrotic diseases, today announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for its investigational therapy FT011 for the treatment of systemic sclerosis (scleroderma), having previously granted Orphan Drug Designation.
  • [4]
    Professor Darren Kelly, Certa Therapeutics CEO and founder said, “We are thrilled to have received Fast Track Designation which supports further acceleration of the FT011 clinical development program.
  • Certa is progressing preparations toward a pivotal clinical trial of FT011 as a treatment for scleroderma.
  • C Denton, W Stevens, N Kruger, M Papadimitriou, F Khong, M Bradney, D Kelly, R Lafyatis “FT011 for the Treatment of Systemic Sclerosis.

Corvus Pharmaceuticals Presents Soquelitinib Preclinical Data at the Keystone Symposia on Systemic Autoimmune and Autoinflammatory Diseases

Retrieved on: 
Monday, February 12, 2024
Atopic dermatitis, Immunodeficiency, Psoriasis, Infection, SQL, ITK, Scleroderma, Preprint, Asthma, T helper cell, Pulmonary fibrosis, T helper 17 cell, Venn, Lymphocyte, Cytokine, Rheumatoid arthritis, Immunosuppression, Dermatitis, Publication, Lymphoma, Research, Immunomodulation, Vaccine

BURLINGAME, Calif., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that preclinical data for soquelitinib, the Company’s lead ITK inhibitor program, was presented in a poster session at the Keystone Symposia on Systemic Autoimmune and Autoinflammatory Diseases, which took place February 8-11, 2024.

Key Points: 
  • BURLINGAME, Calif., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that preclinical data for soquelitinib, the Company’s lead ITK inhibitor program, was presented in a poster session at the Keystone Symposia on Systemic Autoimmune and Autoinflammatory Diseases, which took place February 8-11, 2024.
  • These preclinical product candidates exhibit specific biologic properties that are anticipated to enable more precise inhibition of Th1, Th2 and/or Th17 cell function.
  • Atopic dermatitis (also called eczema) and asthma are thought to be mediated primarily by Th2 lymphocytes.
  • The soquelitinib preclinical data and information on the Company’s next-generation ITK inhibitor candidates was presented by Rahul Pawar, Ph.D., Senior Scientist at Corvus, in poster session #2 (poster #2008) at the Keystone Symposia.

AnaMar Announces US and EU Orphan Drug Designation for AM1476 for Treating Systemic Sclerosis

Retrieved on: 
Monday, February 5, 2024
Inflammation, Systemic scleroderma, Biomarker, Patient, Diagnosis, FDA, EMA, Marketing, Scleroderma, Interstitial lung disease, Skin, Pulmonary fibrosis, Disability, Disease, Fibrosis, European Medicines Agency, Death, ODD, Autoimmune disease, ILD, Organ dysfunction, Pharmaceutical industry

AM1476 offers a unique dual-action approach to treat both skin and lung manifestations of systemic sclerosis.

Key Points: 
  • AM1476 offers a unique dual-action approach to treat both skin and lung manifestations of systemic sclerosis.
  • Systemic sclerosis is a chronic, progressive, autoimmune disease characterized by inflammation and fibrosis, i.e.
  • AnaMar's Chief Executive Officer, Dr. Ulf Ljungberg, said: “We are delighted with the FDA’s and EMA’s decisions to grant orphan drug designation to AM1476 for SSc.
  • Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralized approval process.

aTyr Pharma to Present Posters Highlighting Importance of Neuropilin-2 in Immune Regulation at Keystone Symposia on Myeloid Cell Diversity

Retrieved on: 
Monday, January 29, 2024
Patient, Medicine, Fibrosis, Faculty, Scar, SAN, NRP2, Scleroderma, University, Small RNA, Disease, Neuropilin, Nebraska Medicine, ATyr Pharma, Inflammation, Systemic scleroderma, Sarcoidosis, Myelocyte, Immune system, Pharmaceutical industry

SAN DIEGO, Jan. 29, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced that the company will present two posters at the Keystone Symposia on Myeloid Cell Diversity: From Fundamental Biology to Disease States, which is being held January 28 – 31, 2024, in Banff, Alberta, Canada.

Key Points: 
  • Findings further demonstrate that efzofitimod modulates myeloid cells via the neuropilin-2 (NRP2) receptor to promote a unique anti-inflammatory mechanism.
  • Role of NRP2 in immune system validated by activity of NRP2 blocking antibody in preclinical models.
  • aTyr Pharma, San Diego; Faculty of Health and Medical Sciences, Copenhagen, Denmark.
  • These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.