Breakthrough therapy

Madrigal Pharmaceuticals Announces EMA Validation of its Marketing Authorization Application for Resmetirom for the Treatment of NASH/MASH with Liver Fibrosis

Retrieved on: 
Tuesday, March 5, 2024

NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.

Key Points: 
  • NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.
  • Resmetirom is a once-daily, oral, liver-directed THR-β agonist designed to target key underlying causes of NASH.
  • The clinical development program for resmetirom is comprised of 18 clinical studies supporting the MAA: twelve Phase 1 studies, two Phase 2 studies, and four Phase 3 studies.
  • Without treatment, the disease can lead to cirrhosis, liver failure, liver cancer and premature death,” said Becky Taub, M.D., Chief Medical Officer and President of Research & Development of Madrigal.

Tonix Pharmaceuticals Announces Presentation at The 6th International Congress on Controversies in Fibromyalgia

Retrieved on: 
Thursday, February 29, 2024

The presentation will include additional data from the recently reported positive Phase 3 RESILIENT trial of Tonmya™ (also known as TNX-102 SL) for the management of fibromyalgia.

Key Points: 
  • The presentation will include additional data from the recently reported positive Phase 3 RESILIENT trial of Tonmya™ (also known as TNX-102 SL) for the management of fibromyalgia.
  • A copy of the Company’s presentation will be available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com following the conference.
  • Tonix is a biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering.
  • All of Tonix's forward-looking statements are expressly qualified by all such risk factors and other cautionary statements.

HUTCHMED Reports 2023 Full Year Results and Provides Business Updates

Retrieved on: 
Wednesday, February 28, 2024

HONG KONG and SHANGHAI, China and FLORHAM PARK, N.J., Feb. 28, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”, the “Company” or “we”) (Nasdaq/AIM:​HCM; HKEX:​13), the innovative, commercial-stage biopharmaceutical company, today reports its financial results for the year ended December 31, 2023 and provides updates on key clinical and commercial developments. HUTCHMED to host results call and webcasts today at 7:30 a.m. EST / 12:30 p.m. GMT / 8:30 p.m. HKT in English, and at 8:30 a.m. HKT in Chinese (Putonghua) on Thursday, February 29, 2024.

Key Points: 
  • HUTCHMED is committed to progressively embedding sustainability into all aspects of our operations and creating long-term value for our stakeholders.
  • The 2023 Sustainability Report will be published alongside our 2023 Annual Report in April 2024 and will include further information on HUTCHMED sustainability initiatives and their performance.
  • Net Income attributable to HUTCHMED for 2023 was $100.8 million compared to Net Loss attributable to HUTCHMED of $360.8 million in 2022.
  • The net income attributable to HUTCHMED in 2023 was $0.12 per ordinary share / $0.59 per ADS49, compared to net loss attributable to HUTCHMED of $0.43 per ordinary share / $2.13 per ADS in 2022.

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

Retrieved on: 
Tuesday, February 27, 2024

Alector and GSK are co-developing AL101 for the potential treatment of more prevalent neurodegenerative diseases, including AD and Parkinson’s disease.

Key Points: 
  • Alector and GSK are co-developing AL101 for the potential treatment of more prevalent neurodegenerative diseases, including AD and Parkinson’s disease.
  • In September 2023, Alector completed enrollment of 381 participants in the randomized, double-blind, placebo-controlled, dose-ranging, INVOKE-2 Phase 2 clinical trial.
  • In July 2023, Alector presented an update on INVOKE-2 at the Alzheimer’s Association International Conference (AAIC).
  • Alector’s management team will host a conference call discussing Alector’s results for the fourth quarter and full year 2023 and provide a business update.

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

Retrieved on: 
Tuesday, February 27, 2024

-- Significant Progress in Establishing Global Commercial Manufacturing Capacity for Vaxcyte's PCV Candidates, Including Expanded Collaboration with Lonza --

Key Points: 
  • ET / 1:30 p.m. PT --
    SAN CARLOS, Calif., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Vaxcyte, Inc. (Nasdaq: PCVX), a clinical-stage vaccine innovation company engineering high-fidelity vaccines to protect humankind from the consequences of bacterial diseases, today announced financial results for the fourth quarter and full year ended December 31, 2023, and provided a business update.
  • Additionally, as part of ongoing Chemistry, Manufacturing and Controls (CMC)-focused discussions, Vaxcyte received encouraging input from the FDA regarding the VAX-24 adult licensure requirements.
  • The six-month safety data from both studies showed safety and tolerability results for VAX-24 similar to PCV20 at all doses studied.
  • Vaxcyte will host a conference call and webcast to discuss this announcement today, February 27, 2024, at 4:30 p.m.

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

Retrieved on: 
Tuesday, February 20, 2024

Auvelity® net product sales were $49.0 million and $130.1 million for the fourth quarter and full year of 2023, the first full year of launch.

Key Points: 
  • Auvelity® net product sales were $49.0 million and $130.1 million for the fourth quarter and full year of 2023, the first full year of launch.
  • Total costs of revenue were $7.4 million and $26.1 million for the fourth quarter and full year of 2023, respectively.
  • Approximately 84,000 prescriptions were written for Auvelity in the fourth quarter of 2023, representing a 23% sequential increase versus the third quarter of 2023.
  • Axsome will host a conference call and webcast today at 8:00 AM Eastern to discuss fourth quarter and full year 2023 financial results as well as to provide a corporate update.

Tonix Pharmaceuticals to Participate in the 2024 BIO CEO & Investor Conference

Retrieved on: 
Tuesday, February 20, 2024

CHATHAM, N.J., Feb. 20, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals, will present at the BIO CEO & Investor Conference on February 26, 2024 at 1:30 p.m.

Key Points: 
  • CHATHAM, N.J., Feb. 20, 2024 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals, will present at the BIO CEO & Investor Conference on February 26, 2024 at 1:30 p.m.
  • Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering.
  • Tonix intends to meet with the FDA in the first half of 2024 and submit an NDA for the approval of Tonmya for the management of fibromyalgia in the second half of 2024.
  • A Phase 2 study of TNX-1300 is expected to be initiated in the first quarter of 2024.

Ligand Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, February 27, 2024

Royalties for the fourth quarter of 2023 were $22.5 million, compared with $22.0 million for the same period in 2022.

Key Points: 
  • Royalties for the fourth quarter of 2023 were $22.5 million, compared with $22.0 million for the same period in 2022.
  • Costs of Captisol sales were $1.6 million for the fourth quarter of 2023, compared with $21.6 million for the same period in 2022, with the decrease due to lower total Captisol sales during the fourth quarter of 2023 and $9.8 million in accelerated depreciation on Captisol manufacturing equipment during the fourth quarter of 2022.
  • Amortization of intangibles was $8.3 million for the fourth quarter of 2023, compared with $8.5 million for the same period in 2022.
  • Travere also announced fourth quarter 2023 results reporting that it received 459 new patient start forms for FILSPARI in the fourth quarter of 2023 and net product sales of $14.9 million for the fourth quarter.

Bayer Receives U.S. FDA Breakthrough Therapy Designation for BAY 2927088 for Non-Small Cell Lung Cancer Harboring HER2 Activating Mutations

Retrieved on: 
Monday, February 26, 2024

Bayer announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for BAY 2927088 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have received a prior systemic therapy.

Key Points: 
  • Bayer announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for BAY 2927088 for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have received a prior systemic therapy.
  • The Breakthrough Therapy designation is supported by preliminary clinical evidence from the Phase I, open-label, multicenter first-in-human study (NCT05099172) evaluating the safety, pharmacokinetics and preliminary efficacy of BAY 2927088 in adult patients with advanced NSCLC harboring HER2 or EGFR.
  • “Early clinical evidence suggests that BAY 2927088, our investigational novel oral tyrosine kinase inhibitor, has the potential to benefit patients with NSCLC harboring a HER2 mutation that have progressed on a prior systemic therapy and currently have no other approved treatment available,” said Dominik Ruettinger, M.D., Ph.D., Head of Research and Early Development for Oncology at Bayer’s Pharmaceuticals Division.
  • “This Breakthrough Therapy designation is a significant milestone in our relentless efforts to develop innovative therapies for the treatment of lung cancer characterized by specific genomic markers.

U.S. Food and Drug Administration (FDA) Grants Priority Review of Epcoritamab (EPKINLY®) for Difficult-to-Treat Relapsed or Refractory Follicular Lymphoma (FL)

Retrieved on: 
Tuesday, February 27, 2024

NORTH CHICAGO, Ill., Feb. 27, 2024 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review of the supplemental Biologics License Application (sBLA) for epcoritamab-bysp, a subcutaneously administered T-cell engaging bispecific antibody for the treatment of adult relapsed or refractory (R/R) follicular lymphoma (FL) after two or more lines of therapy. If approved, epcoritamab-bysp (EPKINLY®) will be the first and only subcutaneous bispecific antibody to treat adults with R/R FL after two lines of prior therapy, marking its second indication following FDA and European Medicines Agency (EMA) approval of R/R third-line diffuse large B-cell lymphoma (DLBCL) treatment.

Key Points: 
  • The FDA grants Priority Review to investigational therapies that, if approved, may offer significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications.
  • This designation shortens the review period to six months compared to 10 months for Standard Review.
  • "Together with our partner Genmab, we are committed to further advancing epcoritamab as a core lymphoma therapy."
  • Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.