Spleen

Nurix Therapeutics Reports First Clinical Evidence of CNS Activity of NX-5948, a Brain-Penetrant, Orally Available, BTK Degrader in Development for B Cell Malignancies

Retrieved on: 
Tuesday, April 9, 2024
Histology, Brain, Rituximab, Chronic lymphocytic leukemia, CLL, Lymphoma, Disease, CSF, Central nervous system, SAN, CNS, Patient, PCNSL, BCL2, Cancer, Leukemia, Inflammation, Cerebrospinal fluid, NHL, Lymph, AACR, Proteolysis targeting chimera, Neoplasm, Therapy, MYC, BTK, Myenteric plexus, Lymphoid leukemia, Spleen, Medical imaging

SAN FRANCISCO, April 09, 2024 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, today announced the presentation of the first findings of clinical responses in the brain for NX-5948, an orally available, selective degrader of Bruton’s tyrosine kinase (BTK). The presentation included case studies for two patients, one with CLL with CNS involvement and the other with PCNSL, each demonstrating clinically meaningful responses. The presentation also provided evidence of measurable drug levels in the CNS of multiple patients in the ongoing Phase 1 trial who had CNS tumor involvement. These data were presented by Gwenn M. Hansen, Ph.D., chief scientific officer of Nurix, as part of the Major Symposium session Molecular Glues, PROTACs, and Next-Gen Degraders: Discovery and Early Preclinical Advances at the AACR 2024 Annual Meeting, which is being held from April 5-10, 2024, in San Diego, CA.

Key Points: 
  • The presentation included case studies for two patients, one with CLL with CNS involvement and the other with PCNSL, each demonstrating clinically meaningful responses.
  • The presentation also provided evidence of measurable drug levels in the CNS of multiple patients in the ongoing Phase 1 trial who had CNS tumor involvement.
  • “These data are the first demonstration of clinical activity in the brain of a targeted protein degrader, opening the door for new therapeutic strategies to treat leukemias and lymphomas with CNS involvement,” said Dr. Hansen.
  • “The CLL patient with CNS involvement showed an impressive durable response with NX-5948 as single agent therapy in this setting.

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, B cell, Gaucher's disease, Cell, CD34, Bone marrow, Bleeding, Disease, Seizure, Bruise, Patient, Committee, Knowledge, Fracture, Spleen, Regulation, Fatigue, Autotransplantation, EMA, IRIS, Human, Life expectancy, Eye, Bone pain, Anemia, European, COMP, Sponsor, European Commission, Safety, PPD, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Cullinan Oncology Provides Corporate Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Thursday, March 14, 2024
Research, EGFR, NSCLC, Disease, AML, Cullinan, Patient, Tissue, Sale, Multiple myeloma, G&A, CMC, Enrollment, IND, Lymph, Investment, MDS, FDA, Lymphoma, Spleen, CD19, CGEM, Pharmaceutical industry

CAMBRIDGE, Mass., March 14, 2024 (GLOBE NEWSWIRE) -- Cullinan Oncology, Inc. (Nasdaq: CGEM; “Cullinan”), a biopharmaceutical company focused on developing modality-agnostic targeted oncology therapies, today reported on recent and upcoming business highlights and announced its financial results for the fourth quarter and full year ended December 31, 2023. The company also announced that Chief Financial Officer Jeff Trigilio will depart the company effective March 29. Following his departure, Jeff has agreed to support the company through a transition period.

Key Points: 
  • R&D Expenses: Research and development (R&D) expenses were $34.8 million for the fourth quarter of 2023, compared to $33.8 million for the third quarter of 2023.
  • R&D expenses for the fourth and third quarters of 2023 included $2.7 million and $3.2 million of equity-based compensation expenses, respectively.
  • G&A Expenses: General and administrative (G&A) expenses were $10.6 million for the fourth quarter of 2023, compared to $11.0 million for the third quarter of 2023.
  • Net Loss: Net loss (before items attributable to noncontrolling interest) for the fourth quarter of 2023 was $25.6 million, compared with net loss of $39.2 million for the third quarter of 2023.

SecondWave Systems Announces Professor Paul Peter Tak as New Advisory Board Member and Secures $3M Additional Financing to Accelerate Development of Novel Ultrasound-Based Anti-Inflammatory Therapy

Retrieved on: 
Wednesday, March 27, 2024
Research, Medical Devices, Clinical Trials, Health Technology, Public Policy, Government, Biotechnology, White House, Federal Government, Managed Care, Health, Science, ARPA-H, Rheumatoid arthritis, Partnership, Patient, Trial of the century, ARPA, Inflammation, DARPA, Spleen, Pharmaceutical industry

SecondWave Systems, Inc. , a pioneer in the emerging field of bio-ultrasonic medicine, welcomes Professor Paul Peter Tak as the inaugural member of its newly established advisory board.

Key Points: 
  • SecondWave Systems, Inc. , a pioneer in the emerging field of bio-ultrasonic medicine, welcomes Professor Paul Peter Tak as the inaugural member of its newly established advisory board.
  • This new funding enables further advancement of the MINI technology platform for implementation in a pivotal clinical trial for at-home treatment of RA.
  • “Despite advances in treatment, there is still a significant unmet need for better treatments for chronic inflammatory diseases, like RA”, said Dr. Tak.
  • “One area of unmet need is for therapies that specifically target the cholinergic anti-inflammatory pathway, a natural mechanism in the body that helps control inflammation.

PANTHERx®Rare Selected by Edenbridge Pharmaceuticals as Specialty Pharmacy for Yargesa® (miglustat 100 mg capsules)

Retrieved on: 
Monday, April 1, 2024
Disease, Capsule, Patient, Pharmacy, Bone pain, Hepatomegaly, Enzyme replacement therapy, Anemia, Gaucher, Thrombocytopenia, Liver, Spleen, Pharmaceutical industry

Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.

Key Points: 
  • Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.
  • Through this process, Yargesa® reduces the buildup of harmful glycosphingolipids and symptoms of the disease.
  • This accumulation leads to manifestations including but not limited to enlarged spleen or liver, hepatomegaly, anemia, thrombocytopenia, and bone pain.
  • "We appreciate Edenbridge Pharmaceuticals' commitment to making a difference in the lives of patients with Type 1 Gaucher disease.

Kymera Therapeutics Presents Preclinical Data for STAT6 and TYK2 First-In-Class, Oral Degrader Immunology Programs at the American Academy of Dermatology Annual Meeting

Retrieved on: 
Friday, March 8, 2024
Janus, Model, Spleen, STAT6, Doctor of Philosophy, Inflammation, Atopic dermatitis, Immunoglobulin E, Resource, JAK, Therapy, Inflammatory bowel disease, Protein, TYK2, TPD, Stem cell, Biology, Serum, Phase 1, American Academy, Pharmaceutical industry

WATERTOWN, Mass., March 08, 2024 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing a new class of small molecule medicines using targeted protein degradation (TPD), today announced that its preclinical data demonstrating the therapeutic potential of its potent and selective heterobifunctional degraders of STAT6 (KT-621) and TYK2 (KT-294) are being presented in the poster session at the American Academy of Dermatology’s Annual Meeting in San Diego, California. Kymera’s oral STAT6 and TYK2 degraders have the potential to address multiple immune-mediated diseases and overcome the limitations of existing technologies and agents. Today’s poster presentations mark the first time that data from a STAT6 targeted agent and a TYK2 degrader have been shared at a major medical meeting. Based on the results generated to date, Kymera intends to initiate Phase 1 testing for KT-621 and KT-294 in the in the second half of 2024 and the first half of 2025, respectively. Data from both Phase 1 trials are expected to be reported in 2025.

Key Points: 
  • Kymera’s oral STAT6 and TYK2 degraders have the potential to address multiple immune-mediated diseases and overcome the limitations of existing technologies and agents.
  • Today’s poster presentations mark the first time that data from a STAT6 targeted agent and a TYK2 degrader have been shared at a major medical meeting.
  • “Our differentiated strategy to targeted protein degradation has resulted in an industry-leading immunology pipeline of oral degrader medicines, each with the potential to treat multiple complex immuno-inflammatory diseases.
  • At low daily oral doses, preclinical studies with KT-621 demonstrated near full in vivo STAT6 degradation in disease-relevant tissues that was well-tolerated.

Immix Biopharma Announces “Be Proactive in AL” AL Amyloidosis Awareness Initiative

Retrieved on: 
Tuesday, March 5, 2024
Spleen, Liver, Bangladesh Technical Education Board, Autoimmune disease, NIH, Physician, Extrapyramidal system, Weight loss, Dizziness, AL amyloidosis, Amyloidosis, Diagnosis, Joint, Hypoesthesia, Fatigue, Patient, Heart, Tissue, Mortality

LOS ANGELES, CA, March 05, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and autoimmune disease, today announced its new AL Amyloidosis awareness campaign Be Proactive in AL™.

Key Points: 
  • LOS ANGELES, CA, March 05, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and autoimmune disease, today announced its new AL Amyloidosis awareness campaign Be Proactive in AL™.
  • The campaign seeks to increase awareness about the critical need to recognize and diagnose AL Amyloidosis early, while also educating AL Amyloidosis patients about available treatment options.
  • AL amyloidosis is a life-threatening immunological disorder in which an abnormal protein called amyloid builds up in tissues and organs.
  • “Today, it is common for AL Amyloidosis patients to see 3 or 4 different physicians and wait months to years before being definitively diagnosed with AL amyloidosis,” said Dena Heath, Amyloidosis Research Consortium Board Secretary and Facilitator, Northern California Amyloidosis Support Group.

Coya Therapeutics Licenses Intellectual Property Rights for Use of Next Generation Immune Modulatory Biologics in Combination with COYA 301 to Enhance Regulatory T Cells (Tregs) in Inflammatory Diseases

Retrieved on: 
Tuesday, February 13, 2024
Research, Neurology, Biotechnology, Other Health, Health, Pharmaceutical, General Health, Other Science, Science, Transfer, Spleen, Coya, Inflammation, COYA, Nebraska Medicine, Technology transfer, GM-CSF, University, Therapy, Phenotype, Patent, Patient, Gendelman, CD39, Mouse, Immune system, Pharmaceutical industry

Coya Therapeutics, Inc. (NASDAQ: COYA) (“Coya” or the “Company”), a clinical-stage biotechnology company developing multiple therapeutic programs intended to enhance regulatory T cell (Treg) function, has expanded and strengthened its patent estate beyond COYA 302, which is a combination of COYA 301 with CTLA-4 Ig.

Key Points: 
  • Coya Therapeutics, Inc. (NASDAQ: COYA) (“Coya” or the “Company”), a clinical-stage biotechnology company developing multiple therapeutic programs intended to enhance regulatory T cell (Treg) function, has expanded and strengthened its patent estate beyond COYA 302, which is a combination of COYA 301 with CTLA-4 Ig.
  • UNeMed will receive payments upon achievement of certain milestones and will be eligible to receive tiered low single-digit royalty on net sales.
  • This included a 4-to-6-fold higher expression of Tregs in mice treated with the combination compared to treatments with either cytokine alone.
  • Coya’s foundation and progress in low-dose IL-2 research makes them an ideal company to leverage our findings to date.”

Denali Therapeutics Announces New Data and Expansion of Its Blood-Brain Barrier (BBB)-Crossing Enzyme Replacement Therapy Programs at WORLDSymposium™

Retrieved on: 
Wednesday, February 7, 2024
Traffic barrier, Sanfilippo, Genomics, Hearing, Growth, BBB, AFL, Metabolism, Feinberg School of Medicine, Spleen, Dermatan sulfate, Patient, Cerebrospinal fluid, University of North Carolina, FDA, Cognition, COMPASS, Mouse, CSF, Physician, Hunter syndrome, MPS II, Brain, Behavior, MPS, Mucopolysaccharidosis, Hunting, University, SGSH, ETV, Liver, Alfa, Glycogen storage disease, Pharmaceutical industry

Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.

Key Points: 
  • Denali’s BBB-crossing enzyme replacement approach has the potential to prevent cognitive and behavioral manifestations in MPS IIIA.
  • New two-year peripheral biomarker data demonstrate high magnitude and sustained reduction of urine heparan sulfate and dermatan sulfate, including in participants who switched from standard-of-care enzyme replacement therapy to tividenofusp alfa, suggesting enhanced peripheral activity.
  • Denali also announced that dosing has begun in the Phase 1/2 study of DNL126 for the potential treatment of MPS IIIA.
  • “Our goal is to bring effective new medicines to MPS families as soon as possible,” said Carole Ho, M.D., Chief Medical Officer of Denali.

Beta-thalassemia Market to Witness Upsurge in Growth During the Study Period (2019-2032), Evaluates DelveInsight | Leading Companies - Vertex Pharmaceuticals, CRISPR Therapeutics, Agios Pharmaceuticals, Celgene, Forma Therapeutics

Retrieved on: 
Wednesday, January 17, 2024
Vertex Pharmaceuticals, Patient, Biologics license application, Therapy, Endocrine system, TGF, Heart, LAS, HBA, Spleen, Diagnosis, Anemia, Mouse, Thalassemia, Beta thalassemia, HB, CBC, RBC, Pharmacosmos, Disease, Iron overload, TDT, EU4, FDA, Blas, Prevalence, Liver, Novo Nordisk, PDUFA, Red blood cell, CRISPR Therapeutics, Hemoglobin A, Cell, Iron, Pharmaceutical industry

LAS VEGAS, Jan. 17, 2024 /PRNewswire/ -- DelveInsight's Beta-thalassemia Market Insights report includes a comprehensive understanding of current treatment practices, beta-thalassemia emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

Key Points: 
  • According to DelveInsight's analysis, the market size for beta-thalassemia across the 7MM is expected to grow with a significant CAGR by 2032.
  • The promising beta-thalassemia therapies in the pipeline include CTX001, EDIT-301, Mitapivat, ACE-536, Panobinostat, Etavopivat tablets, and others.
  • In September 2023, Pharmacosmos has initiated a Phase II trial of SP-420 in patients with transfusion-dependent β-thalassemia.
  • In June 2023, FDA accepted the Biologics License Application (BLAs) of exagamglogene autotemcel (exa-cel) for transfusion-dependent beta thalassemia (TDT).