Multifocal motor neuropathy

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.

Dianthus Therapeutics Enhances Leadership Team with Appointment of Jeffrey Stavenhagen, Ph.D., as Chief Scientific Officer

Retrieved on: 
Wednesday, November 29, 2023

NEW YORK and WALTHAM, Mass., Nov. 29, 2023 (GLOBE NEWSWIRE) -- Dianthus Therapeutics, Inc. (Nasdaq: DNTH), a clinical-stage biotechnology company dedicated to advancing the next generation of antibody complement therapeutics to treat severe autoimmune diseases, today announced the appointment of Jeffrey Stavenhagen, Ph.D., as Chief Scientific Officer. In this role, Dr. Stavenhagen will lead the Company’s discovery and preclinical research and translational science initiatives.

Key Points: 
  • In this role, Dr. Stavenhagen will lead the Company’s discovery and preclinical research and translational science initiatives.
  • “I’m thrilled to welcome Jeff to the Dianthus team as we advance next-generation antibody therapeutics for patients living with severe autoimmune diseases.
  • He was most recently Chief Scientific Officer at Therini Bio, where he directed R&D initiatives leading to the development of a first-in-class monoclonal antibody for the treatment of neurodegenerative diseases.
  • She earned a master’s degree in biotechnology from Northwestern University and a bachelor’s degree in biology from the University of Iowa.

Dianthus Therapeutics Highlights Recent Business Achievements and Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 9, 2023

Top-line Phase 1 data across seven single and multiple ascending dose cohorts with 52 healthy volunteers establish DNTH103’s best-in-class potential.

Key Points: 
  • Top-line Phase 1 data across seven single and multiple ascending dose cohorts with 52 healthy volunteers establish DNTH103’s best-in-class potential.
  • Research and development expenses for the quarter ended September 30, 2023 were $8.0 million, inclusive of $0.4 million of stock-based compensation.
  • Net loss for the quarter ended September 30, 2023 was $14.8 million or $3.78 net loss per share (basic and diluted).
  • For additional information on the Company’s financial results for the quarter ended September 30, 2023, please refer to the Form 10-Q filed with the SEC.

NEUROPATHY ACTION FOUNDATION (NAF) TO HOST FREE VIRTUAL PATIENT EDUCATION EVENT ON MULTIFOCAL MOTOR NEUROPATHY (MMN)

Retrieved on: 
Thursday, October 12, 2023

SANTA ANA, Calif. , Oct. 12, 2023 /PRNewswire/ -- The Neuropathy Action Foundation (NAF), a 501(c)(3) non-profit dedicated to providing neuropathy patients with the resources necessary to improve their quality of life, today announced that it will host a free one-hour virtual webinar to educate participants about Multifocal Motor Neuropathy (MMN), including signs and symptoms, incidences and causes, and the importance of accurate and early diagnosis. Details for the event include:

Key Points: 
  • Details for the event include:
    "The process to detect and treat Multifocal Motor Neuropathy can be lengthy and difficult, and MMN patients are often misdiagnosed, which presents additional challenges for patients experiencing symptoms," said NAF President Dominick Spatafora.
  • "The mission of the Neuropathy Action Foundation is to equip neuropathy patients and their families with the resources necessary to improve their quality of life, and this free educational event is a continuation of our decades-long commitment to empowering people living with MMN."
  • Multifocal Motor Neuropathy (MMN) is a rare condition in which multiple motor nerves are attacked by one's immune system.
  • Most patients are in their 40s to 60s, although MMN has been described between the ages of 20 and 80.

Dianthus Therapeutics Announces Positive Top-line Phase 1 Data and Debuts Trading on Nasdaq as DNTH

Retrieved on: 
Tuesday, September 12, 2023

NEW YORK and WALTHAM, Mass., Sept. 12, 2023 (GLOBE NEWSWIRE) -- Dianthus Therapeutics, Inc. (“Dianthus”), a clinical-stage biotechnology company dedicated to advancing the next generation of antibody complement therapeutics to treat severe autoimmune diseases, today announced positive top-line Phase 1 data for DNTH103 validating its best-in-class potential as a selective classical pathway inhibitor targeting only the active form of the C1s protein. Additionally, Dianthus will begin trading on the Nasdaq Capital Market today as DNTH, following the successful completion on September 11, 2023 of the previously announced merger with Magenta Therapeutics (“Magenta”).

Key Points: 
  • Additionally, Dianthus will begin trading on the Nasdaq Capital Market today as DNTH, following the successful completion on September 11, 2023 of the previously announced merger with Magenta Therapeutics (“Magenta”).
  • The Phase 1 healthy volunteer study was designed to validate the extended half-life, potent classical pathway inhibition, and potentially differentiated safety profile of DNTH103.
  • Additional information about our Phase 1 data and DNTH103 can be found in our corporate presentation at investor.dianthustx.com .
  • “These results, combined with our transition to the public markets, represent a major milestone in our growth trajectory.

argenx Initiates Second Cohort of Phase 2 ARDA Study of Empasiprubart in Multifocal Motor Neuropathy

Retrieved on: 
Tuesday, June 20, 2023

“We are encouraged by the favorable safety profile and early efficacy signals from the ARDA study, as well as the IDMC recommendation to advance the study to the next cohort.

Key Points: 
  • “We are encouraged by the favorable safety profile and early efficacy signals from the ARDA study, as well as the IDMC recommendation to advance the study to the next cohort.
  • The IDMC confirmed a favorable safety and tolerability profile of empasiprubart consistent with results from the Phase 1 study and recommended advancing to the second cohort.
  • The study’s objective, in addition to assessing safety and efficacy of empasiprubart, is to populate a PK/PD model to inform the Phase 3 study dose selection.
  • The Phase 2 ARDA study is a randomized, double-blinded, placebo-controlled multicenter study to evaluate the safety and tolerability, efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity of two dose regimens of empasiprubart in adults with multifocal motor neuropathy (MMN).

Takeda Delivers Strong Revenue and Profit Growth in FY2022; Updated Capital Allocation Policy Reflects Deleveraging Progress and Confidence in Growth Outlook

Retrieved on: 
Thursday, May 11, 2023

Rare Diseases, with 723.4 billion yen in reported revenue, grew 4.8% on a CER basis.

Key Points: 
  • Rare Diseases, with 723.4 billion yen in reported revenue, grew 4.8% on a CER basis.
  • Plasma-Derived Therapies (PDT) Immunology, with 678.4 billion yen in reported revenue, delivered outstanding growth of 15.3% on a CER basis.
  • ALUNBRIG® (for non-small cell lung cancer) also grew 35% on a CER basis with strong demand in Europe, China, and Growth & Emerging Markets.
  • This approval marks the first approval of QDENGA in Latin America as Takeda continues to progress additional regulatory filings in other dengue-endemic countries.

Annexon Reports Fourth Quarter and Year-End 2022 Financial Results and Reiterates Anticipated Milestones

Retrieved on: 
Monday, March 6, 2023

BRISBANE, Calif., March 06, 2023 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today outlined anticipated upcoming milestones across its portfolio of complement therapies and reported fourth quarter and full year 2022 financial results.

Key Points: 
  • “2022 was a year of focused execution at Annexon, establishing a strong foundation on which to drive near- and long-term value across our deep portfolio of complement therapeutics,” said Douglas Love, president and CEO of Annexon.
  • To date, ANX1502 has been generally well-tolerated in the ongoing single-ascending dose (SAD) trial with no dose-limiting safety signals observed.
  • Annexon continues to expect its cash, cash equivalents and marketable securities as of December 31, 2022, to be sufficient to fund the company’s current operating plan into 2025.
  • Net loss: Net loss was $34.4 million for the quarter ended December 31, 2022, and $141.9 million for the year ended December 31, 2022, compared to $37.4 million for the quarter ended December 31, 2021, and $130.3 million for the year ended December 31, 2021.

Annexon Highlights Strategic Focus to Advance Four Flagship Complement Programs through Late-Stage Development and Progress Across Three Therapeutic Franchises

Retrieved on: 
Sunday, January 8, 2023

BRISBANE, Calif., Jan. 08, 2023 (GLOBE NEWSWIRE) -- Annexon, Inc. (Nasdaq: ANNX), a clinical-stage biopharmaceutical company developing a new class of complement medicines for patients with classical complement-mediated autoimmune, neurodegenerative and ophthalmic disorders, today reported progress across its broad portfolio of complement therapies and outlined its focus on four flagship programs to support its advancement to a late-stage biopharmaceutical company developing first-in-class treatments for complement-mediated diseases of the body, brain and eye.

Key Points: 
  • Annexon has prioritized resources and execution of late-stage development of its four flagship programs: Guillain-Barré syndrome (GBS), Huntington’s disease (HD), geographic atrophy (GA) and its first-in-kind oral small molecule, ANX1502.
  • Our mission is to free the body, brain and eye from diseases driven by the classical complement cascade,” said Douglas Love, president and CEO of Annexon.
  • Following an assessment of the market opportunity in wAIHA and a range of additional autoimmune indications, Annexon has determined not to advance development in wAIHA.
  • A replay of the webcast will be archived on the Annexon website for 30 days following the presentation.