Dermatomyositis

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

Retrieved on: 
Thursday, February 29, 2024

“argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.

Key Points: 
  • “argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
  • Clinically, we generated significant data through multiple study readouts, achieving key milestones for both the CIDP and MMN patient communities and importantly advancing our second molecule, empasiprubart.
  • Looking forward to 2024, we will act with a continued sense of purpose to expand our patient reach.
  • In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS).

Galapagos announces full year 2023 results and outlook for 2024

Retrieved on: 
Thursday, February 22, 2024

In addition, we entered into a strategic collaboration with BridGene Biosciences to advance our growing early-stage pipeline in precision oncology.

Key Points: 
  • In addition, we entered into a strategic collaboration with BridGene Biosciences to advance our growing early-stage pipeline in precision oncology.
  • Signed an agreement with Boston-based Landmark Bio and started the technology transfer for the decentralized production of Galapagos’ CAR-T cell therapy candidates.
  • Further streamlined our operations with a reduction of approximately 100 positions across the Galapagos organization to align with the Galapagos’ renewed focus on innovation.
  • Signed a strategic collaboration and license agreement with BridGene Biosciences to further strengthen Galapagos' growing early-stage oncology precision medicine pipeline.

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

Retrieved on: 
Monday, January 8, 2024

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.

argenx Highlights 2024 Strategic Priorities

Retrieved on: 
Monday, January 8, 2024

argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.

Key Points: 
  • argenx is planning to reach more patients commercially in 2024 through its multi-dimensional expansion efforts.
  • In addition, ARGX-119, a muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a studies in congenital myasthenic syndrome and amyotrophic lateral sclerosis in 2024.
  • As of December 31, 2023, argenx had approximately $3.2 billion in cash, cash equivalents and current financial assets*.
  • Based on its current operating plans, argenx expects its combined R&D and SG&A expenses in 2024 to be less than $2 billion.

Roivant and Priovant Announce Results from Phase 2 Study of Oral Brepocitinib in Systemic Lupus Erythematosus

Retrieved on: 
Monday, November 27, 2023

Priovant plans to disclose the study data at a future date.

Key Points: 
  • Priovant plans to disclose the study data at a future date.
  • “We saw some of the highest SRI-4 responder rates ever observed in a lupus study in the active arm of this trial, along with a favorable safety and tolerability profile.
  • Unfortunately, we also saw the highest placebo response rate observed in any significant SLE study, and as such it was not possible to truly assess the impact of the drug, or to establish sufficient differentiation from other therapies in lupus patients.
  • Oral brepocitinib has demonstrated statistically significant clinically meaningful benefit in six completed placebo-controlled Phase 2 studies in psoriasis, psoriatic arthritis, alopecia areata, hidradenitis suppurativa, ulcerative colitis, and Crohn’s disease.

Northwell Health Pediatric Rheumatologist Recognized for Lifetime Achievement by the American College of Rheumatology

Retrieved on: 
Tuesday, November 14, 2023

For her dedication to research and leadership in rheumatology, Northwell Health’s Cohen Children’s Medical Center (CCMC) pediatric rheumatologist and Feinstein Institutes for Medical Research scientist Barbara Anne Eberhard, MD , has been awarded top honors by the American College of Rheumatology (ACR) and the Association of Rheumatology Professionals (ARP).

Key Points: 
  • For her dedication to research and leadership in rheumatology, Northwell Health’s Cohen Children’s Medical Center (CCMC) pediatric rheumatologist and Feinstein Institutes for Medical Research scientist Barbara Anne Eberhard, MD , has been awarded top honors by the American College of Rheumatology (ACR) and the Association of Rheumatology Professionals (ARP).
  • Dr. Eberhard was recognized, and named an “ACR Master,” during the ACR Convergence 2023 annual conference which is being held November 10-15 in San Diego, California.
  • View the full release here: https://www.businesswire.com/news/home/20231114668794/en/
    Dr. Barbara Anne Eberhard was recognized for lifetime achievement by the American College of Rheumatology.
  • (Credit: Feinstein Institutes)
    The ACR Master recognition is awarded to physician-researchers who have made significant contributions to the field of rheumatology.

Kezar Life Sciences Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Monday, November 13, 2023

Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company developing breakthrough treatments for immune-mediated and oncologic disorders, today reported financial results for the third quarter ended September 30, 2023 and provided a business update.

Key Points: 
  • Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company developing breakthrough treatments for immune-mediated and oncologic disorders, today reported financial results for the third quarter ended September 30, 2023 and provided a business update.
  • In addition, Kezar recently announced a strategic restructuring to prioritize long-term growth and focus resources on its clinical-stage programs.
  • Actions to prioritize clinical programs and implement cost saving measures are expected to extend Kezar’s cash runway into late 2026.
  • Research and development expenses for the third quarter of 2023 increased by $9.8 million to $23.7 million compared to $13.9 million in the third quarter of 2022.

Cabaletta Bio Reports Third Quarter 2023 Financial Results and Provides Business Update

Retrieved on: 
Thursday, November 9, 2023

PHILADELPHIA, Nov. 09, 2023 (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 reported financial results for the third quarter ended September 30, 2023, and provided a business update.

Key Points: 
  • In November 2023, Cabaletta announced the Company’s fourth IND application for CABA-201 was cleared by the FDA for a Phase 1/2 study in patients with generalized myasthenia gravis (gMG).
  • Cabaletta will present new preclinical data for CABA-201 in a poster presentation and Cabaletta Bio Scientific Advisory Board members Carl June, M.D., and Georg Schett, M.D.
  • Cabaletta plans to participate in the following upcoming investor conferences:
    Stifel 2023 Healthcare Conference, which is being held from November 14-15, 2023 in New York, NY.
  • As of September 30, 2023, Cabaletta had cash, cash equivalents and short-term investments of $164.4 million, compared to $106.5 million as of December 31, 2022.

Galapagos reports third quarter 2023 results and releases new encouraging data from CAR-T studies for presentation at ASH 2023

Retrieved on: 
Thursday, November 2, 2023

Overall, 11 of 12 patients responded to treatment (ORR of 92%) and 9 of 12 patients achieved a CRR (75%).

Key Points: 
  • Overall, 11 of 12 patients responded to treatment (ORR of 92%) and 9 of 12 patients achieved a CRR (75%).
  • Overall, 11 of 13 patients responded to treatment (ORR of 85%) and 9 of 13 patients achieved a CRR (69%).
  • New preliminary data (data cut-off: 26 April 2023), for 12 patients enrolled in EUPLAGIA-1, will be presented at ASH (see ASH abstract and poster presentation details below).
  • New preliminary data (data cut-off: 2 May 2023) for 14 patients enrolled in ATALANTA-1 will be presented at ASH (see ASH abstract and poster presentation details below).

Mallinckrodt Announces Publication of Two Journal Manuscripts with Clinical and Economic Evidence for Acthar® Gel (Repository Corticotropin Injection)

Retrieved on: 
Tuesday, October 10, 2023

DUBLIN, Oct. 10, 2023 /PRNewswire/ -- Mallinckrodt plc (OTCMKTS: MNKTQ), a global specialty pharmaceutical company, today announced the publication of two journal manuscripts reinforcing the clinical and economic evidence supporting the efficacy, safety profile, continued use, and cost-effectiveness of Acthar Gel for appropriate patients across a range of FDA-approved autoimmune and inflammatory disease indications.1,2 

Key Points: 
  • Mallinckrodt's health economics manuscript titled "Acthar Gel (RCI): A Narrative Literature Review of Clinical and Economic Evidence" was published on June 24, 2023 in ClinicoEconomics and Outcomes Research.
  • Acthar is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides.
  • Acthar Gel is approved by the U.S. Food and Drug Administration (FDA) for the treatment of several autoimmune disorders and medical conditions known to cause inflammation.3
    Please see indications and Important Safety Information below.
  • Outcomes may be influenced by therapies not evaluated in the study and the clinical outcomes may not be solely attributable to Acthar.