Systemic scleroderma

Chemomab Reports New Peer-Reviewed Publication Reinforcing the Clinical Association of Its CCL24 Target with Disease Severity and Mortality in Patients with Systemic Sclerosis

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星期四, 四月 18, 2024
Mortality, University, Fibrosis, University of Leeds, TEL, Systemic scleroderma, Synovitis, Nature, Skin, CMMB, Primary sclerosing cholangitis, Prognosis, Communication, Serum, Vascular disease, Physiology, Doctor of Philosophy, Calcinosis, Rheumatology, History, ILD, IND, PSC, Disease, Therapy, Risk, Patient, CSO, Scleroderma, Lung, Clinical trial, CCL24, Pharmaceutical industry

TEL AVIV, Israel, April 18, 2024 (GLOBE NEWSWIRE) -- Chemomab Therapeutics Ltd. (Nasdaq: CMMB), (Chemomab), a clinical stage biotechnology company developing innovative therapeutics to treat rare fibro-inflammatory diseases with high unmet need, today announced the publication of a new study that further confirms the key role of its novel soluble protein target CCL24 in systemic sclerosis (SSc). The study, “Serum CCL24 as a Biomarker of Fibrotic and Vascular Disease Severity in Systemic Sclerosis,” was published in the current edition of the journal Arthritis Care and Research.1

Key Points: 
  • It explored the relationship between serum CCL24 levels and SSc severity and prognosis.
  • One in four patients in a real-life SSc population was found to have a high CCL24 serum concentration, despite standard of care treatment with immunosuppressive therapy.
  • The analysis found that higher CCL24 levels were linked to critical clinical variables associated with the most severe forms of SSc.
  • Crucially, high serum CCL24 was predictive of lung deterioration and a higher baseline CCL24 level was associated with higher 10-year SSc-related mortality.

Zura Bio Announces Oversubscribed $112.5 Million Private Placement

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星期四, 四月 18, 2024
Professional Services, Health, Finance, Clinical Trials, Pharmaceutical, Biotechnology, Sale, Prospectus, Systemic scleroderma, HS, Private, Hidradenitis suppurativa, Guggenheim Partners, Cantor, Inflammation, Placement, Autoimmunity, Security (finance)

Zura Bio Limited (Nasdaq: ZURA) (“Zura Bio”), a clinical stage, multi-asset immunology company developing novel dual-pathway antibodies for autoimmune and inflammatory diseases, today announced that it has entered into subscription agreements for a private placement that is expected to result in gross proceeds of approximately $112.5 million, before deducting placement agent fees and offering expenses (the “Private Placement”).

Key Points: 
  • Zura Bio Limited (Nasdaq: ZURA) (“Zura Bio”), a clinical stage, multi-asset immunology company developing novel dual-pathway antibodies for autoimmune and inflammatory diseases, today announced that it has entered into subscription agreements for a private placement that is expected to result in gross proceeds of approximately $112.5 million, before deducting placement agent fees and offering expenses (the “Private Placement”).
  • The Private Placement is being conducted in accordance with applicable Nasdaq rules and was priced to satisfy the “Minimum Price” requirement (as defined in the Nasdaq rules).
  • The private placement is expected to close on April 22, 2024, subject to satisfaction of customary closing conditions.
  • Piper Sandler, Guggenheim Securities, and Cantor served as lead placement agents for the Private Placement.

FDA Grants Orphan Drug Designation for the Treatment of Scleroderma

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星期四, 四月 11, 2024
Collagen, Pain, FDA, Mortality, University, Patient, RFU, Systemic scleroderma, Risk, Skin, Food, Death, Helix, BLR, Research, Disease, Scleroderma, Rheumatology, Fatigue, Fibrosis, Public, Rheumatism, Multimedia, Myofibroblast, Pharmaceutical industry

NORTH CHICAGO, Ill., April 11, 2024 /PRNewswire/ -- BLR Bio, an emerging biotechnology company in Rosalind Franklin University's Helix 51 biomedical incubator , announced that the U.S. Food and Drug Administration (FDA) granted an Orphan Drug Designation for its investigational therapy BLR-200 for the treatment of systemic sclerosis (SSc), also known as scleroderma.

Key Points: 
  • NORTH CHICAGO, Ill., April 11, 2024 /PRNewswire/ -- BLR Bio, an emerging biotechnology company in Rosalind Franklin University's Helix 51 biomedical incubator , announced that the U.S. Food and Drug Administration (FDA) granted an Orphan Drug Designation for its investigational therapy BLR-200 for the treatment of systemic sclerosis (SSc), also known as scleroderma.
  • Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the U.S.
  • "The FDA's granting of Orphan Drug Designation to BLR-200 highlights the urgent need for new and innovative therapeutic options for patients afflicted with the disease."
  • Dr. Ronald Kaplan, RFU executive vice president for research, said the orphan designation represents an important milestone in the development of BLR Bio's drug candidate.

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

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星期二, 四月 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 Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

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星期四, 三月 21, 2024
Research, MPV, IIM, MuSK protein, Toxicity, Conference, Disease, Dermatomyositis, Juvenile dermatomyositis, Federation, Patient, NOL, Observation, CRS, SLE, Health Canada, IND, Immunoglobulin therapy, Pemphigus, Investment, Investigational New Drug, Myopathy, Systemic scleroderma, Marketing, FDA, Clinical trial, Cytokine release syndrome, Cyclophosphamide, CD19, Food, Autoimmune disease, Pharmaceutical industry

In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.

Key Points: 
  • In October 2023, Cabaletta received Investigational New Drug (IND) application clearance from the U.S. Food and Drug Administration (FDA) for the Phase 1/2 RESET-SSc trial.
  • In November 2023, Cabaletta announced that its IND application for CABA-201 was allowed to proceed by the FDA for the Phase 1/2 RESET-MG trial.
  • Cabaletta anticipates reporting initial clinical data from the Phase 1/2 RESET-MG trial in the second half of 2024.
  • As of December 31, 2023, Cabaletta had cash, cash equivalents and short-term investments of $241.2 million, compared to $106.5 million as of December 31, 2022.

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

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星期三, 三月 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.

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

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星期三, 二月 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.

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

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星期一, 二月 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

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星期一, 一月 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.

aTyr Pharma Announces Howard University President Emeritus Dr. Wayne A. I. Frederick as Advisor

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星期四, 一月 18, 2024
Mortality, ILD, Patient, Outline of health sciences, Wayne A. I. Frederick, Sarcoidosis, Research, Biology, Fibrosis, Associate, Scar, ACS, Dean (education), Howard University, SAN, Knowledge, University, National academy, Humana, Scleroderma, Business, Small RNA, American Surgical Association, Human, Disease, American College, Neuropilin, ATyr Pharma, Health, Inflammation, Organization, MD Anderson Cancer Center, Systemic scleroderma, College, Howard University Hospital, Abstract, Myelocyte, American Cancer Society, Pharmaceutical industry

Dr. Frederick is President Emeritus of Howard University, having served as President from 2014 to 2023.

Key Points: 
  • Dr. Frederick is President Emeritus of Howard University, having served as President from 2014 to 2023.
  • “We are honored to welcome a distinguished physician executive such as Dr. Frederick as an advisor to aTyr,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr.
  • He is also the distinguished Charles R. Drew Professor of Surgery at the Howard University College of Medicine and a practicing cancer surgeon at Howard University Hospital.
  • Dr. Frederick earned a B.S., M.D., and completed his surgical residency training at Howard University Hospital.