FTD

Lexeo Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Operational Highlights

Retrieved on: 
Monday, March 11, 2024

NEW YORK, March 11, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to pioneering treatments for genetically defined cardiovascular diseases and APOE4-associated Alzheimer’s disease, today reported fourth quarter and full year 2023 financial results and provided operational highlights.

Key Points: 
  • Data to date suggest that FA patients may have lower FXN levels in the heart versus peripheral tissues.
  • Across all three cardiac biopsies, we observed an increase in FXN levels as measured by liquid chromatography mass spectrometry relative to pre-treatment baseline levels.
  • Lexeo anticipates the gross proceeds from the private placement to be approximately $95.0 million, before deducting any offering related expenses.
  • G&A expenses were $15.4 million for the year ended December 31, 2023, compared to $12.0 million for the same period in 2022.

CervoMed Announces Presentation of Biomarker Data from the AscenD-LB Phase 2a Trial and Preclinical Data Supporting Potential of Neflamapimod in Tau-Mediated Disease at AD/PD ™ 2024

Retrieved on: 
Tuesday, March 5, 2024

BOSTON, March 05, 2024 (GLOBE NEWSWIRE) -- CervoMed Inc. (NASDAQ: CRVO), a clinical stage company focused on developing treatments for degenerative diseases of the brain, today announced the presentation of biomarker data from the AscenD-LB Phase 2a trial of neflamapimod in patients with dementia with Lewy bodies (DLB), demonstrating that neflamapimod reduces plasma levels of glial fibrillary acidic protein (GFAP) compared placebo, and that the effects of neflamapimod on GFAP were inversely correlated to change in CDR-SB (reduction in GFAP associated with improvement in CDR-SB, and increase in GFAP associated with worsening in CDR-SB). These data will be featured in a poster presentation at the 18th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™) 2024, being held both virtually and in Lisbon, Portugal from March 5–9, 2024. In addition, academic researchers from UCL will be presenting data in a separate poster at the meeting demonstrating that p38MAPK inhibition, including with neflamapimod specifically, improves tau-induced axonal transport defects both in vitro and in a tauopathy mouse model.

Key Points: 
  • These data will be featured in a poster presentation at the 18th International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD™) 2024, being held both virtually and in Lisbon, Portugal from March 5–9, 2024.
  • “The effects on GFAP, particularly the association between GFAP response and clinical outcomes, further support that neflamapimod is clinically efficacious in patients with DLB,” said John Alam, MD, Chief Executive Officer of CervoMed.
  • A PDF copy of the GFAP poster presentation will be available on the “ Presentations and Publications ” section of the CervoMed website.
  • Title: NEW INSIGHTS IN THE DEVELOPMENT OF BIOMARKERS, IMAGING, AND THERAPY OF ALPHA-SYNUCLEIN, LRKK2, AND GBA PATHOLOGIES

Passage Bio Reports Fourth Quarter and Full-Year 2023 Financial Results and Provides Recent Business Highlights

Retrieved on: 
Monday, March 4, 2024

"In 2023, we achieved a significant milestone by announcing encouraging data from three patients in Cohort 1 of our FTD-GRN program.

Key Points: 
  • "In 2023, we achieved a significant milestone by announcing encouraging data from three patients in Cohort 1 of our FTD-GRN program.
  • We eagerly anticipate a catalyst-rich 2024, supported by our robust balance sheet, bringing us closer to improving patient outcomes across neurodegenerative diseases."
  • Additionally, Dose 1 of PBFT02 continued to be generally well-tolerated in patients who received an enhanced steroid regimen for immunosuppression.
  • Cash Position: Cash, cash equivalents and marketable securities were $114.3 million as of December 31, 2023, as compared to $189.6 million as of December 31, 2022.

Merus Announces Financial Results for the Fourth Quarter and Full Year 2023 and Provides Business Update

Retrieved on: 
Wednesday, February 28, 2024

Merus also continues to evaluate patients with untreated advanced PD-L1+ HNSCC treated with petosemtamab 1500 mg in combination with pembrolizumab.

Key Points: 
  • Merus also continues to evaluate patients with untreated advanced PD-L1+ HNSCC treated with petosemtamab 1500 mg in combination with pembrolizumab.
  • Merus plans to report initial interim efficacy and safety data from this cohort in the second quarter of 2024.
  • Merus achieved a milestone and received a payment of $2.5 million related to the advancement of this program in the third quarter of 2023.
  • Merus also achieved an additional milestone of $1 million for candidate nomination in the fourth quarter of 2023.

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.

Coya Therapeutics Presents Biomarker Data on Neuroinflammatory Pathways in Frontotemporal Dementia (FTD) at the AD/PD 2024 Conference

Retrieved on: 
Wednesday, March 6, 2024

The poster presentation was shared for the first time today at the AD/PD 2024 Conference in Lisbon, Portugal and can be found here .

Key Points: 
  • The poster presentation was shared for the first time today at the AD/PD 2024 Conference in Lisbon, Portugal and can be found here .
  • We intend to file an Investigational New Drug (IND) application with the FDA for COYA 302 in FTD later this year and initiate a Ph.
  • 2 trial in FTD patients shortly thereafter,” stated Fred Grossman, Chief Medical Officer at Coya Therapeutics.
  • In addition, several inflammation transcripts in monocyte genes known to be involved in neuroinflammatory signaling pathways were dysregulated in FTD, compared to controls.

Coya Therapeutics to Present Data on Immune System and Regulatory T Cell (Treg) Contributions in Frontotemporal Dementia (FTD) Patients at AD/PD 2024 Conference

Retrieved on: 
Thursday, February 29, 2024

The AD/PD 2024 Conference will be held March 5-9, 2024 in Lisbon, Portugal.

Key Points: 
  • The AD/PD 2024 Conference will be held March 5-9, 2024 in Lisbon, Portugal.
  • The poster, titled “Deciphering the Role of the Peripheral Immune System in the Pathology of Frontotemporal Dementia,” will be presented on March 6 and 7.
  • The work was funded by a grant from the Houston Methodist Clinical Scholar Award Program.
  • This presentation will highlight the complexity of neuroinflammatory pathways, providing the rationale for our planned IND filing for COYA 302, our combination therapeutic candidate, in FTD patients in 4Q24.”

Coya Therapeutics Issues Letter to Stockholders Highlighting Expansion of COYA 302 into Alzheimer’s Disease and Coya’s Pathway to a “Pipeline in a Product”

Retrieved on: 
Wednesday, February 21, 2024

Today, we announce that we are further expanding the pipeline for COYA 302, adding Alzheimer’s disease (AD) to its growing list of indications expected to be validated in the clinic.

Key Points: 
  • Today, we announce that we are further expanding the pipeline for COYA 302, adding Alzheimer’s disease (AD) to its growing list of indications expected to be validated in the clinic.
  • Beyond COYA 302, our therapeutic platform includes additional drug product combinations using COYA 301 (our proprietary LD IL-2) as their backbone.
  • 2 investigator-initiated trial with COYA 301 in AD that we expect will support the development of COYA 302 in that same indication.
  • I look forward to providing investors with additional periodic updates on our research, clinical, corporate, and commercial progress.

QurAlis to Present Data That Show its Splice-Switching ASOs Restore UNC13A Function in ALS and Frontotemporal Dementia

Retrieved on: 
Monday, March 4, 2024

CAMBRIDGE, Mass., March 4, 2024 /PRNewswire/ -- QurAlis Corporation, a clinical-stage biotechnology company driving scientific breakthroughs into powerful precision medicines that will alter the trajectory of amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, today announced preclinical data that showed the company's UNC13A splice-switching antisense oligonucleotides (ASOs) modulate UNC13A splicing and restore normal synaptic activities in ALS and FTD. QurAlis' ASOs prevented cryptic exon inclusion in UNC13A transcripts, increased UNC13A protein levels, and normalized localization of UNC13A protein at the synapse.

Key Points: 
  • QurAlis' ASOs prevented cryptic exon inclusion in UNC13A transcripts, increased UNC13A protein levels, and normalized localization of UNC13A protein at the synapse.
  • A defining feature of both sporadic and familial disease is the cytoplasmic mis-localization of TAR DNA Binding Protein-43 (TDP-43).
  • "QurAlis has identified ASOs that modulate UNC13A splicing and restore normal synaptic activities.
  • QurAlis' ASOs restored all or some aspects of SNARE complex assembly and synaptic vesicle fusion and release.

FDA Grants Fast Track Designation to 9MW2821

Retrieved on: 
Tuesday, February 27, 2024

SHANGHAI, Feb. 27, 2024 /PRNewswire/ -- Mabwell (688062.SH), an innovative biopharmaceutical company with entire industry chain, announces that its self-developed novel ADC drug targeting Nectin-4 (R&D Code: 9MW2821) has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) for the treatment of advanced, recurrent, or metastatic esophageal squamous cell carcinoma (hereinafter referred to as "ESCC").

Key Points: 
  • SHANGHAI, Feb. 27, 2024 /PRNewswire/ -- Mabwell (688062.SH), an innovative biopharmaceutical company with entire industry chain, announces that its self-developed novel ADC drug targeting Nectin-4 (R&D Code: 9MW2821) has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA) for the treatment of advanced, recurrent, or metastatic esophageal squamous cell carcinoma (hereinafter referred to as "ESCC").
  • 9MW2821 is the world's first Nectin-4-targeting drug to disclose clinical efficacy data for the indication of EC.
  • After injection, 9MW2821 can specifically bind to Nectin-4 on the cell membrane surface, be internalized and release cytotoxic drug, and induce the apoptosis of tumor cells.
  • 9MW2821 is also the first to disclose preliminary clinical efficacy data for the indication of CC among drugs with the same target in the world.