Pharmacology

Larimar Therapeutics Announces the Dosing of the First Patient in Long-term Open Label Extension Study for Nomlabofusp in Patients with Friedreich’s Ataxia

Retrieved on: 
Monday, March 11, 2024

Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to address the root cause of Friedreich's ataxia (FA) by delivering frataxin to mitochondria.

Key Points: 
  • Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to address the root cause of Friedreich's ataxia (FA) by delivering frataxin to mitochondria.
  • “We are pleased to dose the first patient in our OLE study, further advancing the nomlabofusp clinical program and building on the successful completion of our Phase 2 dose escalation study.
  • Based on our Phase 1 and Phase 2 findings, we expect to continue daily dosing throughout the study,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar.
  • Initial data from the OLE study is expected in Q4 2024.

FibroGen Appoints Deyaa Adib, M.D. as Chief Medical Officer

Retrieved on: 
Monday, March 11, 2024

SAN FRANCISCO, March 11, 2024 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced the appointment of Deyaa Adib, M.D., an executive leader with almost three decades of oncology development experience, as Senior Vice President & Chief Medical Officer to oversee all global clinical development activities.

Key Points: 
  • SAN FRANCISCO, March 11, 2024 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced the appointment of Deyaa Adib, M.D., an executive leader with almost three decades of oncology development experience, as Senior Vice President & Chief Medical Officer to oversee all global clinical development activities.
  • “Deyaa’s leadership and expertise in oncology research and clinical development, particularly in bringing oncology therapeutics to market, will be key to accelerating our oncology pipeline and bringing novel cancer therapies to patients in need,” said Thane Wettig, Chief Executive Officer of FibroGen.
  • “We will benefit immensely from Deyaa’s broad and deep experience in oncology drug development, including his experiences in both pancreatic and prostate cancers.”
    “I am excited to join FibroGen and lead the clinical development organization at this important time for our company,” commented Deyaa Adib, M.D., Chief Medical Officer of FibroGen.
  • Prior to joining FibroGen, he was the Chief Medical Officer of Triumvira Immunologics Inc. where he led the transition of two novel cell therapy programs into clinical development.

Qu Biologics and TransBIOTech Awarded $450K Applied Research and Development Grant Funding

Retrieved on: 
Monday, March 11, 2024

TransBIOTech, a dedicated Applied Research and Technology Center in Quebec that is focused on supporting biotechnology companies and deepening expertise in this sector in Canada, will partner on this work.

Key Points: 
  • TransBIOTech, a dedicated Applied Research and Technology Center in Quebec that is focused on supporting biotechnology companies and deepening expertise in this sector in Canada, will partner on this work.
  • Dr. Shirin Kalyan, Immunologist and Qu’s Vice President of Scientific Innovation, stated, “In partnership with TransBIOTech, we are very pleased to have been awarded this Applied Research and Development grant to support the expansion of our preclinical and translational programs while we [Qu Biologics] focus on completing enrollment of our two important Phase 2 clinical trials currently underway.
  • We at TransBIOTech aim to nurture the Canadian biotech ecosystem and to help generate value for innovative Canadian biotechnology companies, such as Qu Biologics.
  • Please subscribe here to receive future news and updates from Qu Biologics.

Vigil Announces Oral Presentation on Small Molecule TREM2 Agonist VG-3927 as a Potential Disease-Modifying Therapeutic at AD/PD 2024

Retrieved on: 
Wednesday, March 6, 2024

WATERTOWN, Mass., March 06, 2024 (GLOBE NEWSWIRE) -- Vigil Neuroscience, Inc. (Nasdaq: VIGL), a clinical-stage biotechnology company committed to harnessing the power of microglia for the treatment of neurodegenerative diseases, today presented preclinical data on the profile of VG-3927 in an oral presentation at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Diseases being held March 5 – March 9 in Lisbon Portugal.

Key Points: 
  • WATERTOWN, Mass., March 06, 2024 (GLOBE NEWSWIRE) -- Vigil Neuroscience, Inc. (Nasdaq: VIGL), a clinical-stage biotechnology company committed to harnessing the power of microglia for the treatment of neurodegenerative diseases, today presented preclinical data on the profile of VG-3927 in an oral presentation at the AD/PD™ 2024 International Conference on Alzheimer’s and Parkinson’s Diseases being held March 5 – March 9 in Lisbon Portugal.
  • The presentation outlines preclinical data on the agonist pharmacology of VG-3927, its effect on AD-associated neuropathological endpoints, and its potential as a disease-modifying therapeutic for the treatment of AD.
  • “As the first and only small molecule TREM2 agonist to enter clinical development, we are thrilled to have an opportunity to further demonstrate the differentiated profile for VG-3927 and how it could represent a significant treatment advancement for those living with AD,” said David Gray, PhD, Chief Science Officer at Vigil.
  • “Having recently commenced dosing in our Phase 1 healthy volunteer clinical trial evaluating VG-3927, we look forward to further investigating this mechanism of action and its potential as a disease-modifying therapeutic.”
    Title: Characterization of the First Small Molecule TREM2 Agonist, VG-3927, for Clinical Development in Alzheimer’s Disease

Wave Life Sciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 6, 2024

In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.

Key Points: 
  • In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.
  • Revenue was $29.1 million for the fourth quarter of 2023 as compared to $1.2 million in the prior year quarter.
  • Net loss was $16.3 million for the fourth quarter of 2023 as compared to $43.7 million in the prior year quarter.
  • ET to review the fourth quarter and full year 2023 financial results and pipeline updates.

Rezolute Reports Validation of the Potential Use of RZ358 for Treatment of Non-Islet Cell Tumor Hypoglycemia (NICTH)

Retrieved on: 
Wednesday, March 6, 2024

Tumor hyperinsulinism (HI) may be caused by a variety of different tumor types, resulting in islet cell tumor hypoglycemia (ICTH) and NICTH.

Key Points: 
  • Tumor hyperinsulinism (HI) may be caused by a variety of different tumor types, resulting in islet cell tumor hypoglycemia (ICTH) and NICTH.
  • The Company previously reported on the successful use of RZ358 under its Expanded Access Program (EAP) to treat patients with insulin-producing pancreatic islet cell tumors (ICTs), or insulinomas, causing severe and uncontrolled hypoglycemia.
  • The therapeutic potential of RZ358 in this setting was anticipated given that ICTH is mediated by insulin and that RZ358 is known to work at the insulin receptor to decrease excess insulin binding and activity.
  • The inclusion of NICTH patients in a potential addressable market for RZ358 in tumor HI would more than double the population.

Harvard Bioscience to Showcase Latest Solutions for Preclinical and Organoid Applications at Society of Toxicology Meeting

Retrieved on: 
Wednesday, March 6, 2024

Complementing an already extensive product line, the new innovations provide exciting new opportunities for advancing preclinical and organoid-focused therapy development.

Key Points: 
  • Complementing an already extensive product line, the new innovations provide exciting new opportunities for advancing preclinical and organoid-focused therapy development.
  • The Company’s industry-leading GLP-compliant Ponemah™ preclinical data management platform now includes integrations with the new SoHo™ implantable telemetry solution and the VivaMARS™ high-capacity behavior monitoring system.
  • The integrated platform also opens new opportunities for the use of emerging machine learning-based algorithms to efficiently analyze large data pools.
  • The new Mesh MEA platform is designed for the emerging applications of organoids in research and discovery, safety pharmacology and toxicology.

Sitryx Therapeutics makes key appointments to strengthen business operations and progress immunometabolism-targeting pipeline into clinical development

Retrieved on: 
Monday, March 4, 2024

At Sitryx, Ben joins the Company’s senior leadership team and will oversee the Company’s day-to-day operations and finance.

Key Points: 
  • At Sitryx, Ben joins the Company’s senior leadership team and will oversee the Company’s day-to-day operations and finance.
  • Before becoming Head of Clinical Operations at Sitryx, Gordon was Vice President of Development Operations at biotech company Mission Therapeutics, where he oversaw the design and execution of the company’s clinical development assets.
  • Leading Sitryx’s clinical operations, Gordon will work with the development team as it progresses its assets beyond pre-clinical research and into clinical-stage drug development.
  • Dr Ravi Rao, Chief Medical Officer of Sitryx, commented: “Sitryx has made excellent progress with its pipeline of potential first- and best-in-class, disease-modifying therapeutics.

Wave Life Sciences Announces Upcoming Presentations at MDA Conference that Highlight Best-in-Class Potential for WVE-N531 in Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, February 27, 2024

CAMBRIDGE, Mass., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced its upcoming presentations at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 3-6 in Orlando, FL.

Key Points: 
  • Wave’s poster presentations will highlight the best-in-class potential of WVE-N531 in Duchenne muscular dystrophy (DMD), which is currently being evaluated in the Phase 2, potentially registrational FORWARD-53 study.
  • The presentations will also illustrate the impact of Wave’s novel PN chemistry on pharmacology of its exon skipping oligonucleotides.
  • These data suggest that WVE-N531 muscle concentrations in the clinic may be higher in heart and diaphragm than in skeletal muscle.
  • “At Wave, we increasingly continue to regard exon skipping as the preferred mechanism for altering DMD disease progression in those amenable to this approach.

Arch Biopartners Has Pre-IND Meeting With FDA to Discuss Repurposing Cilastatin as a New Treatment to Prevent Acute Kidney Injury

Retrieved on: 
Tuesday, February 27, 2024

The PIND meeting provided the Arch team with guidance from the FDA for the content of a future IND application for cilastatin.

Key Points: 
  • The PIND meeting provided the Arch team with guidance from the FDA for the content of a future IND application for cilastatin.
  • An IND application is a request to the FDA for authorization to administer a new drug to patients in a human trial.
  • Arch has method of use patents in several jurisdictions for repurposing cilastatin as a treatment for AKI.
  • We look forward to working with our research and clinical collaborators to establish cilastatin and DPEP-1 inhibition as a new treatment to prevent toxin-related AKI.”