Nature Medicine

Elicio Therapeutics to Present Updated Clinical T Cell and Antigen Spreading Response Data from the Ongoing AMPLIFY-201 Phase 1 Study of ELI-002 and Preclinical Data on ELI-007 and ELI-008 at the AACR Annual Meeting

Retrieved on: 
Friday, April 5, 2024
CD4, CD8, AMP, Development, Antigen, Immunotherapy, Colorectal cancer, Memory, Patient, Lung, GM-CSF, Biomarker, DNA, Epitope, Cancer, Granzyme B, BRAF, Lymph, IL2, Circulating tumor DNA, Phenotype, Neoplasm, Research and development, Immunization, Therapy, Vaccination, CRC, V600E, Immune system, Melanoma, Granzyme, Phase 1, Amphetamine, Nature Medicine, Annual general meeting, Vaccine

Preclinical data on vaccine candidates, ELI-007 and ELI-008, investigational peptide vaccines targeting BRAF and p53-driven cancers, respectively, will also be shared.

Key Points: 
  • Preclinical data on vaccine candidates, ELI-007 and ELI-008, investigational peptide vaccines targeting BRAF and p53-driven cancers, respectively, will also be shared.
  • A majority of patients who received the booster immunizations maintained or increased mKRAS-specific T cell responses relative to baseline.
  • The mKRAS-specific CD4 and CD8 T cells generated by ELI-002 exhibited increased cytotoxic function and development of favorable memory phenotype.
  • "Earlier data published in Nature Medicine demonstrate that our off-the-shelf lymph node-targeted cancer vaccine candidate, ELI-002, induces memory T cell responses.

Mustang Bio Announces Publication in Nature Medicine of Data from Phase 1 Trial Evaluating MB-101 IL13Rα2-targeted CAR T-Cells in High-Grade Glioma

Retrieved on: 
Thursday, March 7, 2024
T cell, Glioblastoma, Arms, ICT, ICV, Traffic barrier, Toxicity, Headache, Biopsy, DSM-IV codes, Cerebrospinal fluid, City, Cancer, Central nervous system, CXCL10, TME, Cerebral edema, Dual, Hope, GBM, Safety, CXCL9, Fatigue, Hypertension, Translation, CNS, Tumor microenvironment, Patient, Arm, Spinal cord, Survival, Brain, Nature Medicine, Infrared spectroscopy correlation table, Vaccine

WORCESTER, Mass., March 07, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (Nasdaq: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for difficult-to-treat cancers and rare genetic diseases, today announced Phase 1 clinical data were published in Nature Medicine that demonstrated the promising safety and clinical activity of Mustang’s MB-101 (IL13Ra2-targeted CAR T-cells) for the treatment of patients with recurrent and refractory malignant glioma, including glioblastoma.

Key Points: 
  • MB-101 was developed by City of Hope, one of the largest cancer research and treatment organizations in the United States, and exclusively licensed to Mustang.
  • Central nervous system (CNS) increases in inflammatory cytokines, including IFNγ, CXCL9, and CXCL10, were associated with CAR T-cell administration and bioactivity.
  • Primary endpoints were safety and feasibility, with secondary endpoints measuring therapy-related cytokine dynamics, CAR T-cell persistence and clinical outcomes.
  • Dr. Brown has a financial interest in Mustang and has previously been a paid consultant for the company.

Day One Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Progress

Retrieved on: 
Monday, February 26, 2024
BRAF, Pfizer, Research, New Drug Application, Doctor of Philosophy, MD, Investment, FDA, Food, Priority review, Day One, Event, Patient, Medicine, Clinical trial, Nature Medicine, NDA, Head, Ageing, MEK, Pharmaceutical industry

BRISBANE, Calif., Feb. 26, 2024 (GLOBE NEWSWIRE) -- Day One Biopharmaceuticals (Nasdaq: DAWN) (“Day One” or the “Company”), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, today announced its fourth quarter and full year 2023 financial results and highlighted recent corporate achievements.

Key Points: 
  • “We have a monumental year ahead of us at Day One with the upcoming PDUFA date for tovorafenib,” said Jeremy Bender, Ph.D., chief executive officer of Day One.
  • The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2024.
  • In November 2023, Nature Medicine published the registrational Phase 2 FIREFLY-1 trial results investigating tovorafenib in patients with BRAF-altered, relapsed or progressive pediatric low-grade glioma (pLGG).
  • In the fourth quarter of 2023, Day One continued its commercial preparedness for the approval and launch of tovorafenib with the hiring of 18 sales representatives in the U.S.

Limbic Raises $14M to Bring AI-Powered Mental Health Support, Proven in the World’s Largest Public Health System, to U.S. Providers

Retrieved on: 
Tuesday, March 5, 2024
Technology, Mental Health, Medical Devices, LGBTQ+, Health Technology, Health, Consumer, General Health, Artificial Intelligence, Mental health, AI, Intelligence, Generative, NHS, Khosla Ventures, Synthetic media, Supercell, Patient, Acceleration, Mental, United, Burnout, Workforce, Nature Medicine, Vinod Khosla, Brain, Nursing

Limbic’s mental health AI has been used by over 260,000 patients across the United Kingdom’s National Health Service (NHS) Talking Therapies.

Key Points: 
  • Limbic’s mental health AI has been used by over 260,000 patients across the United Kingdom’s National Health Service (NHS) Talking Therapies.
  • Limbic’s clinical AI has demonstrated increased access to mental health services, acceleration in patient onboarding, reduced wait times, and improved quality of care.
  • The funding will expand the availability of Limbic’s clinical mental health AI to U.S. health providers.
  • This included a 179% increase among people who identified as non-binary, 39% for Asian and 40% for Black mental health patients.

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

Retrieved on: 
Wednesday, February 28, 2024
Biotechnology, Pharmaceutical, Health, Oncology, CCNE1, Quarter, Adolescence, Research, Arm, PPP2R1A, PARP, FBXW7, Net, FOLFIRI, AACR, Conference, BMS, ADP, PLK4, Clinical trial, Safety, Therapy, Patient, RECIST, Nature Medicine, IND, ATR, WEE1, Partnership, Pharmaceutical industry

Repare Therapeutics Inc. (“Repare” or the “Company”) (Nasdaq: RPTX), a leading clinical-stage precision oncology company, today reported financial results for the fourth quarter and full year ended December 31, 2023.

Key Points: 
  • Repare Therapeutics Inc. (“Repare” or the “Company”) (Nasdaq: RPTX), a leading clinical-stage precision oncology company, today reported financial results for the fourth quarter and full year ended December 31, 2023.
  • Repare will sponsor the global clinical trial as a new arm in the ongoing MYTHIC trial, with costs being shared equally by Debiopharm and Repare.
  • Disclose additional camonsertib clinical development plans beyond the TRESR and ATTACC clinical trials sponsored by Repare in the second quarter of 2024.
  • Fourth Quarter and Full Year 2023 Financial Results:
    Cash, cash equivalents, and marketable securities: Cash, cash equivalents, and marketable securities as of December 31, 2023 were $223.6 million.

New Publication in Nature Medicine Supports Therapeutic Potential of Senolytics to Provide Long-lasting, Disease-modifying Intervention in Vision Loss

Retrieved on: 
Tuesday, February 6, 2024
Biotechnology, Paper, SAN, Cell, Research, VEGF, Therapy, Ageing, Environment, Patient, Diabetic retinopathy, Hyperglycemia, Unity Biotechnology, Retina, Homeostasis, Visual acuity, UBX, Cellular senescence, Diabetes, Endothelium, DME, University, Eye, Metalloproteinase, Nature Medicine, Université de Montréal, Pharmaceutical industry

Sustained hyperglycemia from diabetes induces cellular senescence which damages the retina, including the delicate vasculature in the eye.

Key Points: 
  • Sustained hyperglycemia from diabetes induces cellular senescence which damages the retina, including the delicate vasculature in the eye.
  • This can lead to fluid accumulation and retinal thickening – a key feature of DME.
  • They demonstrated that these senescent cells contribute to loss of barrier function, which can cause leaky blood vessels.
  • This research further supports the potential for senolytic medicines to address the significant unmet need with current standard of care.”

Deciphera Pharmaceuticals Announces Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, February 6, 2024
Biotechnology, Pharmaceutical, Health, Oncology, OS, KIT, European, Investigational New Drug, Trial of the century, DNA, Research, Patient, IND, Autophagy, ASCO, GIST, Public expenditure, FDA, EMA, MAA, Part, Food, Society, TGCT, Gastrointestinal stromal tumor, European Medicines Agency, Sunitinib, Neoplasm, Cancer, INSIGHT, Tenosynovial giant cell tumor, NDA, Nature Medicine, Pharmaceutical industry

Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, today announced financial results for the fourth quarter and year ended December 31, 2023 and provided a corporate update.

Key Points: 
  • Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, today announced financial results for the fourth quarter and year ended December 31, 2023 and provided a corporate update.
  • Expects to present updated results from the Phase 1/2 study of vimseltinib in TGCT in the second half of 2024.
  • Expects to initiate a Phase 2 proof-of-concept study of vimseltinib for the treatment of chronic graft versus host disease (cGVHD) in the fourth quarter of 2024.
  • Cost of Sales: Cost of sales were $1.8 million in the fourth quarter of 2023, which includes $0.9 million in cost of product sales, compared to cost of product sales of $0.7 million for the fourth quarter of 2022.

Elicio Therapeutics to Present ELI-002 7P (AMPLIFY-7P) Trial in Progress Poster on Phase 1/2 Study of Lymph Node-Targeted Vaccine at ASCO GI Symposium

Retrieved on: 
Wednesday, January 17, 2024
Tumor microenvironment, Development, Death, Pancreatic cancer, Patient, Therapy, Cancer, Neoplasm, DNA, Pancreas, Standard of care, Survival, AMP, Poster, University, Lymphocyte, Risk, HLA, Safety, UCLA, Lymph, CA19-9, Immunization, University of California, PDAC, Research and development, Observation, Biopsy, Colorectal cancer, Nature Medicine, RAS, Pharmaceutical industry

The poster describes AMPLIFY-7P, a Phase 1 and randomized Phase 2 study of ELI-002 7P, an investigational therapeutic cancer vaccine, administered as an adjuvant monotherapy treatment for patients with KRAS-mutated pancreatic ductal adenocarcinoma (“PDAC”).

Key Points: 
  • The poster describes AMPLIFY-7P, a Phase 1 and randomized Phase 2 study of ELI-002 7P, an investigational therapeutic cancer vaccine, administered as an adjuvant monotherapy treatment for patients with KRAS-mutated pancreatic ductal adenocarcinoma (“PDAC”).
  • “We are progressing our AMP-powered, lymph node-targeted cancer vaccine, ELI-002 7P, as a monotherapy in an adjuvant setting for patients with pancreatic cancer, and recently dosed the first patient in the randomized Phase 2 cohort.
  • AMPLIFY-7P is a Phase 1 and Phase 2 study of ELI-002 7P in patients with RAS mutated pancreatic and colorectal tumors after locoregional treatment.
  • A Phase 2 interim analysis is planned using group sequential design for control of overall alpha 0.10.

Deciphera Pharmaceuticals Presents Long-Term Follow-Up Results from INTRIGUE Phase 3 Clinical Study in Second-Line GIST Patients at the 2024 American Society of Clinical Oncology Gastrointestinal Cancers Symposium

Retrieved on: 
Thursday, January 18, 2024
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, FDA, Clinical Trials, Patient, Progression-free survival, KIT, Neoplasm, OS, Hypertension, ASCO, Fatigue, Society, Cancer, Myalgia, Survival rate, Public health, INSIGHT, AP, PFS, Safety, Diarrhea, GIST, Sunitinib, Nature Medicine, Preventive healthcare, Pharmaceutical industry

There were fewer patients with Grade 3/4 drug-related treatment emergent adverse events (TEAE) with QINLOCK (26.5%) compared with sunitinib (55.2%).

Key Points: 
  • There were fewer patients with Grade 3/4 drug-related treatment emergent adverse events (TEAE) with QINLOCK (26.5%) compared with sunitinib (55.2%).
  • Dose interruptions and reductions as well as treatment discontinuations due to TEAEs were lower with QINLOCK versus sunitinib.
  • Patients with mutations in KIT exon 11 and 17/18 had improved progression-free survival, objective response rate, and overall survival with QINLOCK versus sunitinib.
  • Based on the results of this prespecified exploratory objective in INTRIGUE, the Company is enrolling the INSIGHT pivotal Phase 3 clinical study of QINLOCK in second-line GIST patients with mutations in KIT exon 11 and 17/18 only.

Elicio Therapeutics Announces First Patient Dosed in Randomized Phase 2 Pancreatic Cancer Study

Retrieved on: 
Thursday, January 11, 2024
Development, Pancreatic cancer, Death, Patient, Therapy, Cancer, Neoplasm, Risk, PDAC, Research and development, Medical research, Biomarker, Nature Medicine, Pharmaceutical industry

This is in contrast to other KRAS-targeted therapeutics in development — particularly small molecule KRAS inhibitors — which target fewer mutations, potentially limiting the number of patients that can be treated and also limiting duration of benefit due to development of escape mutants.

Key Points: 
  • This is in contrast to other KRAS-targeted therapeutics in development — particularly small molecule KRAS inhibitors — which target fewer mutations, potentially limiting the number of patients that can be treated and also limiting duration of benefit due to development of escape mutants.
  • “ELI-002 represents a cancer vaccine approach that could potentially address the much broader spectrum of pancreatic cancer KRAS mutations.
  • The Phase 2 study builds on positive findings from our 2-peptide formulation of ELI-002 published in Nature Medicine demonstrating a significantly improved decrease in tumor biomarkers, along with strong T cell responses that correlated with a reduced risk of relapse and death.
  • Initial interim data on ELI-002 7P monotherapy from Phase 1A arm will be shared in the first half of 2024.