MDS

Geron Corporation Reports Business Highlights and Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 2, 2023
Biotechnology, Pharmaceutical, Health, FDA, Research, New Drug Application, PDUFA, MAA, CHMP, MDS, CRISPR, European Directive on Traditional Herbal Medicinal Products, European Medicines Agency, Anemia, GAAP, Acquisition, Exercise, Imetelstat, Momenta, Society, Impress, ASH, Genome, Treasurer, Clinical trial, Geron Corporation, Risk, Webcast, Retirement, EMA, Corporation, Depreciation, Editas Medicine, NDA, Johnson & Johnson, Patient, Transfusion-dependent anemia, SET, Pharmaceutical industry, Cryptocurrency, Rare-earth element

Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported business highlights and financial results for the third quarter of 2023.

Key Points: 
  • Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company developing a first-in-class telomerase inhibitor, imetelstat, to treat hematologic malignancies, today reported business highlights and financial results for the third quarter of 2023.
  • Ms. Robertson brings to Geron over 30 years of financial and commercial operations experience.
  • In the third quarter of 2023, the Company received $28.3 million upon the cash exercise of outstanding warrants.
  • Geron will host a conference call at 9:00 am ET on Thursday, November 2, 2023 to discuss business updates and third quarter 2023 financial results.

Geron IMerge Phase 3 Presentations at Upcoming ASH Annual Meeting Reinforce Significant Durability and Breadth of Effect of Imetelstat in Lower Risk MDS

Retrieved on: 
Thursday, November 2, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, International Prognostic Scoring System, IPSS, Survival, FDA, RUNX1, H. Lee Moffitt Cancer Center & Research Institute, VAF, Food, TP53, Thrombocytopenia, Literature, EZH2, MDS, European Medicines Agency, Regulation of tobacco by the U.S. Food and Drug Administration, Imetelstat, Society, ASXL1, SF3B1, Transfusion-dependent anemia, Geron Corporation, Risk, Abstract, TI, EMA, Corporation, LR, Anemia, ETV6, Patient, Neutropenia, Mutation, Pharmaceutical industry, Communications satellite, ASH

Four abstracts have been accepted for presentation at the 65th American Society of Hematology (ASH) Annual Meeting taking place from December 9-12 in San Diego, California and virtually.

Key Points: 
  • Four abstracts have been accepted for presentation at the 65th American Society of Hematology (ASH) Annual Meeting taking place from December 9-12 in San Diego, California and virtually.
  • “Additionally, our abstract showcasing a real-world data population level analysis of over 5,000 lower risk MDS patients adds to the significant literature suggesting a correlation between TI and improvement in survival.
  • This analysis suggests imetelstat has clinical activity in lower risk MDS patients independent of risk categories.
  • Achievement of durable TI was associated with improved survival, supporting the clinical benefit of achieving transfusion independence in lower risk MDS.

Rigel Announces Poster Presentations at the 65th American Society of Hematology Annual Meeting and Exposition

Retrieved on: 
Thursday, November 2, 2023
CR, IVO, VEN, Haematopoiesis, Constipation, Platelet, Acute myeloid leukemia, CRC, Prior, Thrombocytopenia, Vomiting, MDS, Diarrhea, Treatment, Rigel, Fostamatinib, Bone marrow, Hematopoietic stem cell transplantation, CRH, CRi, Inflammation, Azacitidine, Nausea, Society, Progress, ASH, Poster, Myelodysplastic syndrome, Cell death, Relapse, ITP, SAN, ORR, University, Safety, Venetoclax, IRAK, Cellular, MD Anderson Cancer Center, Cytopenia, Transplant, Neutropenia, AML, HSCT, Fatigue, Pharmaceutical industry, Dentistry, Dietary supplement, Bookkeeping, Vaccine, Patient, Cancer

SOUTH SAN FRANCISCO, Calif., Nov. 2, 2023 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced the upcoming presentation of four posters highlighting data from their commercial and clinical-stage hematology-oncology portfolio at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition being held December 9-12, 2023, in San Diego, California and virtually.

Key Points: 
  • "We are also excited by the compelling data in patients with mIDH1 MDS and look forward to evaluating this potential opportunity further.
  • On top of the olutasidenib data, we are delighted to share other updates and data across our development portfolio, demonstrating our presence in the hematology-oncology space."
  • These results support the use of fostamatinib as a second-line treatment in patients with primary ITP.
  • To learn more about Rigel Pharmaceuticals and their clinical and commercial hematology/oncology portfolio visit Booth #2805 during ASH 2023.

U.S. FDA AWARDS BOTH RARE PEDIATRIC DISEASE AND ORPHAN DRUG DESIGNATIONS TO HG204, A CRISPR RNA-EDITING THERAPY, FOR THE TREATMENT OF MECP2 DUPLICATION SYNDROME

Retrieved on: 
Tuesday, October 31, 2023
FDA, Ageing, Orphan Drug Act of 1983, Food, Rare, RNA, Sponsor, MDS, CRISPR, Greater China, Prevalence, MECP2, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Frasier, ODD, Speech, Neurodevelopmental disorder, Clinical trial, RNA editing, Messenger, MECP2 duplication syndrome, Therapy, Patient, GM, Pharmaceutical industry

"Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.

Key Points: 
  • "Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.
  • The FDA defines rare pediatric diseases as rare diseases (with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals aged under 18.
  • When the FDA awards rare pediatric disease designation (RPDD), the Sponsor of the trial firstly is granted a 6-months accelerated review of the drug candidate irrespective of indication, and which may be sold to another company.
  • "MDS is a rare and fatal childhood disease with the progressive of neurological regression such as loss of motor skills and speech.

U.S. FDA AWARDS BOTH RARE PEDIATRIC DISEASE AND ORPHAN DRUG DESIGNATIONS TO HG204, A CRISPR RNA-EDITING THERAPY, FOR THE TREATMENT OF MECP2 DUPLICATION SYNDROME

Retrieved on: 
Tuesday, October 31, 2023
FDA, Ageing, Orphan Drug Act of 1983, Food, Rare, RNA, Sponsor, MDS, CRISPR, Greater China, Prevalence, MECP2, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Frasier, ODD, Speech, Neurodevelopmental disorder, Clinical trial, RNA editing, Messenger, MECP2 duplication syndrome, Therapy, Patient, GM, Pharmaceutical industry

"Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.

Key Points: 
  • "Although there is currently no clinical trial evaluating MDS, this is a sign of recognition for MDS and raises hopes for children and teens with this fatal, devastating disorder.
  • The FDA defines rare pediatric diseases as rare diseases (with fewer than 200,000 cases in the United States) that are serious or life threatening and primarily affect individuals aged under 18.
  • When the FDA awards rare pediatric disease designation (RPDD), the Sponsor of the trial firstly is granted a 6-months accelerated review of the drug candidate irrespective of indication, and which may be sold to another company.
  • "MDS is a rare and fatal childhood disease with the progressive of neurological regression such as loss of motor skills and speech.

Servier Announces FDA Approval of TIBSOVO® (ivosidenib tablets) for the Treatment of IDH1-Mutated Relapsed or Refractory (R/R) Myelodysplastic Syndromes (MDS)

Retrieved on: 
Tuesday, October 24, 2023
CR, Survival, Abbott, FDA, Food, MDS, Hope, IDH, News, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Syndrome, Priority review, Caregiver, Disease, Risk, Commercial, Breakthrough therapy, ORR, IDH1, Safety, Overalls, Patient, Diagnosis, Prognosis, Mutation, Pharmaceutical industry, Laboratoires Servier

BOSTON, Oct. 24, 2023 /PRNewswire/ -- Servier, a leader in oncology committed to bringing the promise of tomorrow to the patients we serve, today announced the U.S. Food and Drug Administration (FDA) has approved TIBSOVO® (ivosidenib tablets) for the treatment of patients with isocitrate dehydrogenase 1 (IDH1)-mutated relapsed or refractory (R/R) myelodysplastic syndromes (MDS). This is the fifth indication for TIBSOVO across IDH1-mutated cancers, and the first and only approved targeted therapy for people diagnosed with R/R MDS within this molecularly defined subset.

Key Points: 
  • "Servier is proud to lead the way in mutant IDH inhibition through continued innovations that support patients living with difficult and hard-to-treat cancers," said Arjun Prasad, Head of Commercial, Servier Pharmaceuticals.
  • "As the first and only targeted therapy available for patients with IDH1-mutated relapsed or refractory myelodysplastic syndromes, today's FDA approval for TIBSOVO reinforces our commitment to deliver significant advances in areas of high unmet need and bring the right treatment, to the right patient, at the right time."
  • "This new indication in IDH1-mutated relapsed or refractory myelodysplastic syndromes reinforces the importance of mutational testing to inform treatment decisions and potentially improve patient outcomes."
  • "Before today, there were no approved targeted therapies available to relapsed or refractory MDS patients harboring the IDH1-mutation.

Amylyx Pharmaceuticals to Present Clinical Trial Design of ORION, a Phase 3 Global Study of AMX0035 in Progressive Supranuclear Palsy (PSP), at the Neuro2023 PSP and CBD International Research Symposium

Retrieved on: 
Thursday, October 19, 2023
Biotechnology, Pharmaceutical, Health, Clinical Trials, PSP, ORION, Progressive supranuclear palsy, Movement disorder, Pan Pacific Hotels and Resorts, Medication, MDS, Neurology, AMX0035, Disease, Aggregation, Ludwig Maximilian University of Munich, Unfolded protein response, HIV disease progression rates, LMU, Quality of life, Department of Neurology, Xuanwu hospital, Caregiver burden, Safety, Hospital, Pharmaceutical industry, Medical imaging, Amylyx Pharmaceuticals, MD

Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced an upcoming presentation at Neuro2023 on the design of ORION, a planned global, Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) in progressive supranuclear palsy (PSP).

Key Points: 
  • Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the “Company”) today announced an upcoming presentation at Neuro2023 on the design of ORION, a planned global, Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) in progressive supranuclear palsy (PSP).
  • Neuro2023: The PSP and CBD International Research Symposium will take place on October 19-20, 2023, at the Pan Pacific Hotel in London, UK.
  • The ORION Phase 3 trial was designed and planned in collaboration with key global academic leaders, people living with PSP, and industry advocacy groups.
  • Additional details regarding trial enrollment and eligibility criteria will be shared upon initiation of the trial, which is anticipated to start by the end of 2023.

Inhibikase Therapeutics Highlights Unblinded Functional Analysis from the 201 Trial of Risvodetinib in Untreated Parkinson’s Disease and Provides Update on Ongoing Enrollment

Retrieved on: 
Monday, October 16, 2023
S&E, Schwab, Therapy, Data, Safety, Disease, Congress, Placebo, Patient, Part, PD, MDS, Pharmaceutical industry

These participants were withdrawn from the trial following the FDA’s temporary clinical hold in November, 2022 that was lifted in January, 2023.

Key Points: 
  • These participants were withdrawn from the trial following the FDA’s temporary clinical hold in November, 2022 that was lifted in January, 2023.
  • The Company also provided an update on the enrollment progress and recruitment tools being used in the ongoing 201 Trial.
  • In August 2023, Inhibikase presented an analysis of the primary and secondary endpoints performed on these eleven participants at the Movement Disorders Society Congress.
  • The Company also detailed changes in the functional assessments of motor and non-motor features using a hierarchical analysis of fifteen secondary endpoints.

Inside information: Faron Announces Positive BEXMAB Study Update in Relapsed/Refractory AML and HMA-Refractory MDS Patients

Retrieved on: 
Wednesday, October 11, 2023
Majesty of the Seas, Nasdaq First North, Faron Pharmaceuticals, Quality of life, Optimus, Capillary leak syndrome, Standard of care, ORR, AML, EST, Webcast, Acute myeloid leukemia, IR, Bone marrow, Hemophagocytic lymphohistiocytosis, AIM, HMA, Syndrome, Cryptogenic organizing pneumonia, SOC, MDS, Prognosis, Patient, Phase II, Pharmaceutical industry, Online shopping, Fårö

TURKU, Finland and BOSTON, Oct. 11, 2023 (GLOBE NEWSWIRE) -- Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company pioneering macrophage reprogramming for effective anticancer immunotherapies, today announces updated data from the Phase 1/2 BEXMAB study investigating bexmarilimab in combination with standard of care (SoC) in relapsed/refractory (r/r) acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) patients having failed hypomethylating agents (HMAs).

Key Points: 
  • The most recent data includes read outs from a total of 22 patients (r/r AML with 12 patients, MDS frontline and MDS HMA-failed patients with five patients each) who have completed at least two or more treatment cycles.
  • Eight of 11 patients achieved complete remission in the bone marrow with or without blood count recovery.
  • The highest single indication-specific ORR was observed among HMA-failed MDS patients (4 out of 5 patients; 80%).
  • Faron plans to increase the number of US clinical sites from two to five sites to accelerate study recruitment.

Foghorn Therapeutics to Present Clinical and Pre-Clinical Data from Multiple Programs Across Its Diverse Pipeline at AACR-NCI-EORTC International Conference

Retrieved on: 
Wednesday, October 4, 2023
Synovial sarcoma, AACR, UM, CBP, DLBCL, Foghorn, NGS, Chromatin, BRM, BRG1, Mum, Gene expression, NSCLC, Prostate, Biomarker, Lung cancer, EP300, Acute myeloid leukemia, B cell, MDS, Malignancy, AML, BAF, Patient, Lymphoma, Neoplasm, BRD9, AR, Conference, Prostate cancer, Pharmaceutical industry, Vaccine, Science

The conference will be held October 11–15, 2023, in Boston, Massachusetts.

Key Points: 
  • The conference will be held October 11–15, 2023, in Boston, Massachusetts.
  • Foghorn will highlight data from its FHD-286 program demonstrating its potential as a broad-based hematologic and solid tumor differentiation agent, including clinical data in acute myeloid leukemia (AML), metastatic uveal melanoma (mUM), and pre-clinical data in non-small cell lung cancer (NSCLC) and prostate cancer.
  • Also at AACR-NCI-EORTC, the Company will present data from its selective EP300 program, including in vitro selective degradation and antiproliferation in AR+ prostate and DLBCL cell lines.
  • The EP300 program targets CBP mutant cancers and subsets of EP300 dependent malignancies, which include bladder, NSCLC, and various leukemias and lymphomas.