Acute promyelocytic leukemia

Meet the St. Baldrick's Foundation 2024 Ambassadors

Retrieved on: 
Wednesday, January 3, 2024

LOS ANGELES, Jan. 3, 2024 /PRNewswire/ -- The St. Baldrick's Foundation, the largest charity funder of childhood cancer research grants, is proud to introduce its 2024 Ambassadors who along with their families, will share their unique journey and experiences associated with childhood cancer.

Key Points: 
  • The St. Baldrick's Foundation introduces its 2024 Ambassadors to raise awareness about the need to fund childhood cancer research to find cures and better treatments.
  • LOS ANGELES, Jan. 3, 2024 /PRNewswire/ -- The St. Baldrick's Foundation , the largest charity funder of childhood cancer research grants , is proud to introduce its 2024 Ambassadors who along with their families, will share their unique journey and experiences associated with childhood cancer.
  • Now 12 years in remission, Scott is enjoying the 9th grade and is a passionate advocate for childhood cancer research.
  • Continue following the St. Baldrick's 2024 Ambassadors' heroic stories on the St. Baldrick's blog and social media channels: Facebook , X , Instagram , YouTube , Tik Tok and LinkedIn .

Notable Labs Announces JCO Precision Oncology Publication Demonstrating PPMP Potential to Identify Novel Drug Combinations in JMML

Retrieved on: 
Thursday, November 9, 2023

FOSTER CITY, Calif., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Notable Labs, Inc. (“Notable”) (Nasdaq: NTBL), a clinical stage therapeutic platform company developing predictive precision medicines for cancer patients, today announced the publication of data from an ex vivo study demonstrating the potential for its Predictive Precision Medicine Platform (PPMP) to identify more effective pre-hematopoietic stem cell transplantation (pre-HSCT) regimens for the treatment of juvenile myelomonocytic leukemia (JMML) in the November 9th Issue of the American Society of Clinical Oncology (ASCO) Journal JCO Precision Oncology. Part of this data was recently presented at the European Hematology Association (EHA) Hybrid Congress held in Frankfurt, Germany on June 8 – 15, 2023. Details of that presentation can be found on the Company’s website at https://notablelabs.com/.

Key Points: 
  • JMML is a rare, aggressive pediatric malignancy for which curative treatment is restricted to hematopoietic stem cell transplantation.
  • Pre-HSCT therapies, i.e., therapies prior to transplantation, include moderately intensive chemotherapy (particularly fludarabine and cytarabine in combination (FLA)) and azacytidine (AZA) monotherapy.
  • Applying its proprietary PPMP technology to blood or bone marrow samples from JMML patients, Notable screened 130 drug combinations and discovered that 27 of these combinations (26 dual-, 1 triple-combination) were more effective ex vivo than the components of these combinations tested individually.
  • “This publication marks the culmination of a multi-year collaboration between UCSF and Notable Labs,” remarked Dr. Stieglitz.

Syros Reports Third Quarter 2023 Financial Results and Provides a Corporate Update

Retrieved on: 
Tuesday, November 14, 2023

Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company committed to advancing new standards of care for the frontline treatment of hematologic malignancies, today reported financial results for the quarter ended September 30, 2023 and provided a corporate update.

Key Points: 
  • Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company committed to advancing new standards of care for the frontline treatment of hematologic malignancies, today reported financial results for the quarter ended September 30, 2023 and provided a corporate update.
  • In October 2023, Syros announced the retirement as Chief Executive Officer (CEO) of Nancy Simonian, M.D., effective December 1, 2023.
  • General and administrative expenses were $7.8 million for the third quarter of 2023, as compared to $8.1 million for the third quarter of 2022.
  • ET to discuss these third quarter 2023 financial results and provide a corporate update.

Rutgers Cancer Institute of New Jersey and RWJBarnabas Health to Showcase Expansive Portfolio of Hematology/Oncology Data at the 65th American Society of Hematology Annual Meeting and Exposition

Retrieved on: 
Tuesday, November 28, 2023

NEW BRUNSWICK, N.J., Nov. 28, 2023 /PRNewswire/ -- Physician-scientists from Rutgers Cancer Institute of New Jersey and RWJBarnabas Health will present an extensive array of hematology/oncology data from their clinical research program at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition, being held in San Diego, California (and virtually) from December 9-12, 2023. A total of 36 abstracts have been accepted, comprising clinical data and analyses that advance the understanding, treatment, and prognosis of blood cancers such as lymphoma, leukemia, and myeloma. Rutgers Cancer Institute of New Jersey, in partnership with RWJBarnabas Health, is New Jersey's only National Cancer Institute-designated Comprehensive Cancer Center and the leading cancer program in the state.

Key Points: 
  • Rutgers Cancer Institute of New Jersey, in partnership with RWJBarnabas Health, is New Jersey's only National Cancer Institute-designated Comprehensive Cancer Center and the leading cancer program in the state.
  • "As the leading cancer program in the state, our commitment to advancing oncology care is unwavering.
  • The primary endpoint was progression-free survival (PFS), and secondary endpoints included overall survival (OS), event-free survival (EFS), and safety.
  • The full list of presentations at this year's ASH Annual Meeting and Exposition follows:

Syros Announces Planned CEO Leadership Transition and Strategic Reorganization to Support Long-Term Business Growth and Maturation into a Commercial Biopharmaceutical Company

Retrieved on: 
Monday, October 2, 2023

Also today, Syros announced the retirement as Chief Executive Officer (CEO) of Nancy Simonian, M.D., and the appointment of Conley Chee, Syros’ Chief Commercial Officer (CCO) and Chief Business Officer (CBO), as CEO, effective December 2, 2023.

Key Points: 
  • Also today, Syros announced the retirement as Chief Executive Officer (CEO) of Nancy Simonian, M.D., and the appointment of Conley Chee, Syros’ Chief Commercial Officer (CCO) and Chief Business Officer (CBO), as CEO, effective December 2, 2023.
  • Dr. Simonian will remain a member of the Syros Board of Directors following the transition.
  • This includes streamlining our team and, due to capital constraints, stopping further investment in SY-2101 for the foreseeable future.
  • As we look toward near-term data catalysts from SELECT-MDS-1 and SELECT-AML-1, we are planning for our next phase of growth.

Cellectis Presents Updated Clinical and Translational Data on BALLI-01 at the European Hematology Association (EHA) 2023

Retrieved on: 
Friday, June 9, 2023

NEW YORK, June 09, 2023 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, presented today updated clinical and translational data on its clinical trial BALLI-01 (evaluating UCART22) at the European Hematology Association (EHA) 2023.

Key Points: 
  • NEW YORK, June 09, 2023 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, presented today updated clinical and translational data on its clinical trial BALLI-01 (evaluating UCART22) at the European Hematology Association (EHA) 2023.
  • The data presented support the preliminary safety and efficacy of UCART22 in a heavily pretreated relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) population.
  • UCART22 cell expansion was detected in 9 of 13 patients in the FCA LD arm and associated with clinical activity.
  • Overall, these data support the preliminary safety and efficacy of UCART22 in this heavily pretreated r/r B-ALL population.

Vor Bio Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Company Update

Retrieved on: 
Thursday, March 23, 2023

In December 2022, Vor Bio announced the pricing of an underwritten offering and a private placement, with combined gross proceeds of approximately $115.8 million.

Key Points: 
  • In December 2022, Vor Bio announced the pricing of an underwritten offering and a private placement, with combined gross proceeds of approximately $115.8 million.
  • The Company intends to evaluate VCAR33ALLO in combination with trem-cel as a Treatment System, aiming at prolonged remissions or cures following transplant.
  • General & Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2022 were $7.7 million, compared to $5.6 million for the fourth quarter of 2021, and for the year ended December 31, 2022, were $28.9 million, compared to $21.5 million for the year ended December 31, 2021.
  • Net Loss: Net loss for the fourth quarter of 2022 was $23.9 million, compared to $18.3 million for the fourth quarter of 2021, and for the year ended December 31, 2022, was $92.1 million, compared to $68.9 million for the year ended December 31, 2021.

First AML Patient Transplanted with Vor Bio’s Trem-cel Demonstrated Durable Engraftment through Multiple Mylotarg™ Cycles at Initial Dose Level

Retrieved on: 
Thursday, February 16, 2023

In the first patient, trem-cel maintained hematopoiesis through three cycles of Mylotarg (gemtuzumab ozogamicin), which was well-tolerated at the initial dose level of 0.5 mg/m2.

Key Points: 
  • In the first patient, trem-cel maintained hematopoiesis through three cycles of Mylotarg (gemtuzumab ozogamicin), which was well-tolerated at the initial dose level of 0.5 mg/m2.
  • A second patient has successfully received a trem-cel transplant and engrafted normally.
  • “We are also encouraged that a second patient successfully received a trem-cel transplant and look forward to learning more as we treat additional patients and dose escalate Mylotarg.
  • The company is moving forward with dose escalation of Mylotarg per the 3+3 dose escalation schema in the protocol.

Syros Pharmaceuticals Announces Publication in Blood Advances Demonstrating the Potential of Tamibarotene in Patients with RARA Gene Overexpression, Supporting Ongoing Clinical Development in AML and MDS

Retrieved on: 
Monday, December 12, 2022

These findings support Syros ongoing evaluation of tamibarotene for the treatment of AML and myelodysplastic syndrome (MDS) patients with RARA overexpression.

Key Points: 
  • These findings support Syros ongoing evaluation of tamibarotene for the treatment of AML and myelodysplastic syndrome (MDS) patients with RARA overexpression.
  • The biomarker test successfully identified AML patients positive for RARA overexpression who were enriched for response to tamibarotene and azacitidine relative to those patients who were negative for RARA overexpression.
  • This observation further demonstrates that the activity of tamibarotene is dependent on the biology of RARA overexpression.
  • Based on RARA expression levels, each patient was classified as positive for RARA overexpression (22 patients) or negative for RARA overexpression (29 patients).

Syros Presents Safety Lead-in Data from SELECT-AML-1 Trial Evaluating Tamibarotene in Combination with Venetoclax and Azacitidine and Announces Plans to Initiate Randomized Portion of Phase 2 Trial

Retrieved on: 
Saturday, December 10, 2022

As of October 13, 2022, eight newly diagnosed, unfit, RARA-positive patients had been enrolled in the trial, including six who were evaluable for response.

Key Points: 
  • As of October 13, 2022, eight newly diagnosed, unfit, RARA-positive patients had been enrolled in the trial, including six who were evaluable for response.
  • - Tamibarotene in combination with venetoclax and azacitidine administered at approved doses showed no evidence of increased toxicity relative to the doublet combination of venetoclax and azacitidine.
  • This includes rates of myelosuppression, which were comparable to reports with venetoclax and azacitidine in this population.
  • The randomized portion is expected to initiate in Q1 2023, with data expected in 2023 or 2024.