MuSK protein

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).

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.

ZILBRYSQ® (zilucoplan) Is Now Commercially Available in the U.S. for the Treatment of Generalized Myasthenia Gravis (gMG) in Adult Patients Who Are Anti-Acetylcholine Receptor (AChR) Antibody Positive

Retrieved on: 
Wednesday, January 3, 2024

ZILBRYSQ® (zilucoplan) is now commercially available by prescription in the United States and dispensed by UCB's selected exclusive specialty pharmacy PANTHERx® Rare Pharmacy.

Key Points: 
  • ZILBRYSQ® (zilucoplan) is now commercially available by prescription in the United States and dispensed by UCB's selected exclusive specialty pharmacy PANTHERx® Rare Pharmacy.
  • The most common side effects of ZILBRYSQ include injection site reactions, upper respiratory tract infections, and diarrhea.
  • This support includes refresher injection coaching and information on symptom tracking and ongoing treatment support, as well as help in understanding insurance coverage.
  • If an eligible patient or caregiver is looking for support, please visit http://www.UCBONWARD.com or call 1-844-ONWARD.

NMD Pharma Raises €75 Million (~$80 million) in a Series B Financing

Retrieved on: 
Wednesday, November 15, 2023

The financing was led by current investor Jeito Capital and includes investments from other current NMD Pharma investors: Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, and the Roche Venture Fund.

Key Points: 
  • The financing was led by current investor Jeito Capital and includes investments from other current NMD Pharma investors: Novo Holdings, Lundbeckfonden BioCapital, INKEF Capital, and the Roche Venture Fund.
  • NMD Pharma is developing first-in-class small molecule inhibitors of the skeletal muscle specific chloride ion channel (ClC-1) to enhance neuromuscular transmission with the potential to restore muscle function in a range of rare neuromuscular diseases.
  • Proceeds from the financing will be used to complete three Phase 2 studies with NMD670, the Company’s lead ClC-1 inhibitor.
  • Furthermore, NMD Pharma will continue to expand its pipeline of ClC-1 inhibitor molecules and pursue undisclosed targets for the treatment of other neuromuscular diseases.

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.

UCB announces U.S. FDA approval of ZILBRYSQ® (zilucoplan) for the treatment of adults with generalized myasthenia gravis

Retrieved on: 
Tuesday, October 17, 2023

It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.

Key Points: 
  • It is the only once-daily gMG target therapy for self-administration by adult patients with anti-AChR antibody-positive gMG.
  • Patients were randomized in a 1:1 ratio to receive daily subcutaneous injections of 0.3 mg/kg ZILBRYSQ or placebo for 12 weeks.
  • The most common adverse reactions (≥10%) in patients with gMG were injection site reactions, upper respiratory tract infection, and diarrhea.
  • The primary endpoint for the RAISE study was change from baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score.

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

Retrieved on: 
Thursday, August 10, 2023

PHILADELPHIA, Aug. 10, 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 second quarter ended June 30, 2023, and provided a business update.

Key Points: 
  • PHILADELPHIA, Aug. 10, 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 second quarter ended June 30, 2023, and provided a business update.
  • Cabaletta anticipates generating 3-month clinical data on efficacy and tolerability in initial CABA-201 treated patients by the first half of 2024.
  • 2023 Cantor Global Healthcare Conference, which is being held from September 26-28, 2023 in New York, NY.
  • As of June 30, 2023, Cabaletta had cash, cash equivalents and short-term investments of $176.3 million, compared to $106.5 million as of December 31, 2022.

PANTHERx® Selected by UCB as part of the Limited Specialty Pharmacy Network for RYSTIGGO®(rozanolixizumab-noli), for the Treatment of Generalized Myasthenia Gravis (gMG)

Retrieved on: 
Wednesday, August 2, 2023

Generalized Myasthenia Gravis is a rare, chronic autoimmune disease that results from pathogenic autoantibodies impairing synaptic transmission at the neuromuscular junction.

Key Points: 
  • Generalized Myasthenia Gravis is a rare, chronic autoimmune disease that results from pathogenic autoantibodies impairing synaptic transmission at the neuromuscular junction.
  • 2-3 These autoantibodies primarily consist of AChR or MuSK antibodies.
  • 4 The resultant damage manifests as muscle fatigue and weakness.
  • We are happy to be able to help some individuals living with Generalized Myasthenia Gravis with the distribution of RYSTIGGO," said Rob Snyder, CEO of PANTHERx® Rare.

KabaFusion Selected by UCB as a Limited Distribution Partner for RYSTIGGO® (rozanolixizumab-noli)

Retrieved on: 
Wednesday, August 2, 2023

CERRITOS, Calif. and LEXINGTON, Mass., Aug. 02, 2023 (GLOBE NEWSWIRE) -- KabaFusion, a nationally recognized leader in home infusion, has been selected by UCB as a limited distribution partner to provide RYSTIGGO® (rozanolixizumab-noli).

Key Points: 
  • CERRITOS, Calif. and LEXINGTON, Mass., Aug. 02, 2023 (GLOBE NEWSWIRE) -- KabaFusion, a nationally recognized leader in home infusion, has been selected by UCB as a limited distribution partner to provide RYSTIGGO® (rozanolixizumab-noli).
  • RYSTIGGO® (rozanolixizumab-noli) is for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.
  • “KabaFusion is proud to be a leader in home infusion and providing access to patients with new drugs that will improve their quality of life.
  • We have a well-established history of successfully managing patients with myasthenia gravis and neuromuscular conditions and we are proud to have been selected by UCB as a limited distribution partner to provide RYSTIGGO to patients and providers,” said Dr. Sohail Masood, founder and CEO of KabaFusion.

argenx Reports Half Year 2023 Financial Results and Provides Second Quarter Business Update

Retrieved on: 
Thursday, July 27, 2023

VYVGART® Hytrulo now available in the U.S. with first vials shipped in July

Key Points: 
  • VYVGART® Hytrulo now available in the U.S. with first vials shipped in July
    Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced its half year 2023 financial results and provided a second quarter business update.
  • “We are thrilled to see the momentum continue across all aspects of our business, with a catalyst-rich first half of the year.
  • Our ambition level is high and we are positioning argenx for long-term growth with VYVGART, empasiprubart, and our pipeline of immunology solutions.
  • argenx expects to nominate one new pipeline candidate in 2023.