Systemic 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acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn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acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn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acid Treatment of systemic sclerosis, 12/01/2017 Withdrawn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 Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 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

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.

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.

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.

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

Retrieved on: 
Thursday, January 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.

Cabaletta Bio Receives Additional FDA Fast Track Designations for CABA-201 in Dermatomyositis and Systemic Sclerosis

Retrieved on: 
Monday, January 8, 2024
Dermatomyositis, Food, GMG, IND, Research, FDA, SLE, Organ dysfunction, Scleroderma, Investigational New Drug, Systemic scleroderma, Myositis, Patient, Lupus nephritis, Pharmaceutical industry

PHILADELPHIA, Jan. 08, 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 separate Fast Track Designations to CABA-201, an investigational 4-1BB-containing fully human CD19-CAR T cell therapy, for the treatment of patients with dermatomyositis to improve disease activity and for the treatment of patients with systemic sclerosis (SSc) to improve associated organ dysfunction.

Key Points: 
  • “The additional Fast Track Designations for CABA-201 in both dermatomyositis and systemic sclerosis, the second and third Fast Track Designations for CABA-201, provide the opportunity for expedited development and review of CABA-201 for the treatment of these autoimmune indications where there is a significant unmet need, despite currently available therapies,” said David J. Chang, M.D., Chief Medical Officer of Cabaletta.
  • “We believe these designations potentially accelerate our ability to launch the first targeted, and potentially curative, cell therapy for autoimmune diseases driven by B cells.
  • To date, Cabaletta has received clearance from the FDA for Investigational New Drug (IND) applications for CABA-201 in multiple autoimmune conditions including systemic lupus erythematosus (SLE), myositis, SSc and generalized myasthenia gravis (gMG).
  • Cabaletta is conducting four Phase 1/2 clinical trials with a total of nine cohorts that can advance simultaneously, employing a similar parallel cohort design and starting dose of 1 x 106 cells/kg without a dose escalation requirement.

aTyr Pharma Presents Poster Demonstrating Preclinical Effects of Efzofitimod in Rheumatoid Arthritis and Rheumatoid Arthritis-Associated ILD at the ACR Convergence 2023

Retrieved on: 
Monday, November 13, 2023
NRP2, SAN, Neuropilin, Inflammation, Scar, Sarcoidosis, ILD, Scleroderma, Systemic scleroderma, Patient, Myelocyte, Rheumatoid arthritis, American College of Rheumatology, ATyr Pharma, American College, Small RNA, ACR, Poster, Fibrosis, Pharmaceutical industry

The poster will be available on the aTyr website once presented.

Key Points: 
  • The poster will be available on the aTyr website once presented.
  • The poster presents findings from preclinical models of rheumatoid arthritis (RA) and RA-associated ILD (RA-ILD), where NRP2, efzofitimod’s binding partner, is known to be expressed on pro-inflammatory synovial macrophages.
  • An animal knockout model for NRP2 demonstrated that NRP2 deficiency exacerbated disease pathology in preclinical models of inflammatory disease.
  • 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 Third Quarter 2023 Results and Provides Corporate Update

Retrieved on: 
Thursday, November 9, 2023
NRP2, Research, Safety, SAN, Neuropilin, PST, D, BLA, Sarcoidosis, ILD, Scleroderma, International, Investment, Systemic scleroderma, ERS, Clinical trial, Patient, European Respiratory Society, DSMB, Rheumatoid arthritis, ATyr Pharma, American College, American College of Rheumatology, Small RNA, The Journal, Frontiers, Skin, ACR, Poster, Biologics license application, Pharmaceutical industry, Pharmacology, Kyorin

SAN DIEGO, Nov. 09, 2023 (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 third quarter 2023 results and provided a corporate update.

Key Points: 
  • Ended the third quarter of 2023 with $105.6 million in cash, cash equivalents and investments.
  • SAN DIEGO, Nov. 09, 2023 (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 third quarter 2023 results and provided a corporate update.
  • Based on current enrollment projections, the Company expects to complete enrollment in the study early in the second quarter of 2024.
  • G&A Expenses: General and administrative expenses were $2.6 million for the third quarter of 2023.