Hemophagocytic lymphohistiocytosis

Danaher, Jennifer Doudna, and Innovative Genomics Institute Launch Danaher-IGI Beacon for CRISPR Cures with Aim to Address Hundreds of Diseases Using Gene-editing Platform Solution

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Tuesday, January 9, 2024
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The center, known as the Danaher-IGI Beacon for CRISPR Cures, aims to use CRISPR-based gene editing to permanently address hundreds of diseases with a unified research, development and regulatory approach.

Key Points: 
  • The center, known as the Danaher-IGI Beacon for CRISPR Cures, aims to use CRISPR-based gene editing to permanently address hundreds of diseases with a unified research, development and regulatory approach.
  • The collaboration, which enables a substantial new research program at IGI, is the largest Danaher Beacon to date.
  • The Beacons program funds pioneering academic research with the goal of developing innovative technologies and applications for human health.
  • The unique nature of CRISPR makes it ideal for developing and deploying a platform capability for CRISPR cures on demand.

Innate Pharma Announces U.S. FDA Lifts Partial Clinical Hold on Lacutamab Clinical Program

Retrieved on: 
Thursday, January 4, 2024
Oncology, Health, FDA, Clinical Trials, Pharmaceutical, Biotechnology, Food, HLH, Hemophagocytic lymphohistiocytosis, Euronext Paris, IND, Disease, IPH, Death, IPHA, Pharmaceutical industry

Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold placed on the lacutamab IND.

Key Points: 
  • Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold placed on the lacutamab IND.
  • On October 5, Innate announced that the lacutamab IND has been placed on partial clinical hold by FDA following a recent patient death in the TELLOMAK study.
  • The FDA decision to lift the partial clinical hold is based on the FDA review of the fatal case which Innate, together with a steering committee of independent experts, determined to be related to aggressive disease progression and lacutamab unrelated.
  • “We have worked closely with the FDA to diligently resolve the partial clinical hold on the lacutamab IND, which included an in-depth analysis of the fatal case which was due to progression of an aggressive form of the disease,” said Dr Quaratino, Chief Medical Officer of Innate Pharma.

Sobi to present new data at ASH 2023 Annual Meeting

Retrieved on: 
Monday, November 20, 2023
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STOCKHOLM, Nov. 20, 2023 /PRNewswire/ -- Sobi® will present new data at the 65th Annual Meeting of the American Society of Hematology (ASH) in San Diego from the 9th to the 12th of December 2023.

Key Points: 
  • STOCKHOLM, Nov. 20, 2023 /PRNewswire/ -- Sobi® will present new data at the 65th Annual Meeting of the American Society of Hematology (ASH) in San Diego from the 9th to the 12th of December 2023.
  • During the meeting several analyses will be presented in patients with haemophilia A, paroxysmal nocturnal hemoglobinuria (PNH), immune thrombocytopenia (ITP), relapsed or refractory diffuse large b-cell lymphoma, myelofibrosis, and haemophagocytic lymphohistiocytosis.
  • "We take pride in our expanded commitment to individuals affected by rare diseases, which we are pleased to highlight at this year's ASH meeting, with research that spans a range of rare and debilitating conditions.
  • "We look forward to collaborating and connecting in person at this year's meeting."

Sobi to present new data at ASH 2023 Annual Meeting

Retrieved on: 
Monday, November 20, 2023
Blood type, ITP, Swedish Orphan Biovitrum, Society, Ruxolitinib, Research, PNH, Patient, Hemophagocytic lymphohistiocytosis, RDMA, MBA, MD, Research development, ASH, Medical Affairs Bureau

STOCKHOLM, Nov. 20, 2023 /PRNewswire/ -- Sobi® will present new data at the 65th Annual Meeting of the American Society of Hematology (ASH) in San Diego from the 9th to the 12th of December 2023.

Key Points: 
  • STOCKHOLM, Nov. 20, 2023 /PRNewswire/ -- Sobi® will present new data at the 65th Annual Meeting of the American Society of Hematology (ASH) in San Diego from the 9th to the 12th of December 2023.
  • During the meeting several analyses will be presented in patients with haemophilia A, paroxysmal nocturnal hemoglobinuria (PNH), immune thrombocytopenia (ITP), relapsed or refractory diffuse large b-cell lymphoma, myelofibrosis, and haemophagocytic lymphohistiocytosis.
  • "We take pride in our expanded commitment to individuals affected by rare diseases, which we are pleased to highlight at this year's ASH meeting, with research that spans a range of rare and debilitating conditions.
  • "We look forward to collaborating and connecting in person at this year's meeting."

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

Retrieved on: 
Wednesday, October 11, 2023
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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.

Innate Pharma Provides Update on Lacutamab Clinical Program

Retrieved on: 
Thursday, October 5, 2023
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Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the lacutamab IND leading to a pause in new patient enrollment to the Company’s ongoing lacutamab trials IPH4102-201 (Phase 2 TELLOMAK) and 102 (Phase 1b PTCL).

Key Points: 
  • Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the lacutamab IND leading to a pause in new patient enrollment to the Company’s ongoing lacutamab trials IPH4102-201 (Phase 2 TELLOMAK) and 102 (Phase 1b PTCL).
  • The partial clinical hold follows one fatal case of hemophagocytic lymphohistiocytosis (HLH), a rare hematologic disorder.
  • TELLOMAK, Innate Pharma’s ongoing Phase 2 trial of lacutamab in cutaneous T-cell lymphoma (CTCL), completed enrollment in Q2 2023 (n=170 patients).
  • Innate Pharma is on track for final data from the Phase 2 TELLOMAK trial and preliminary data on PTCL in Q4 2023.

Bellicum Presents Early Phase 1 Results for BPX-601 in Prostate Cancer at ASCO GU Cancers Symposium

Retrieved on: 
Thursday, February 16, 2023
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HOUSTON, Feb. 16, 2023 (GLOBE NEWSWIRE) -- Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, today will present early Phase 1 results for BPX-601 at the American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO GU) in San Francisco and virtually. The poster titled “Early Results from a Phase 1, Multicenter Trial of PSCA-Specific GoCAR T® Cells (BPX-601) in Patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC)” presents initial data from the first two cohorts (n=8) treated with BPX-601. These interim results demonstrated preliminary efficacy of BPX-601 PSCA-directed GoCAR-T cells in combination with rimiducid in heavily pre-treated patients.

Key Points: 
  • The poster titled “Early Results from a Phase 1, Multicenter Trial of PSCA-Specific GoCAR T® Cells (BPX-601) in Patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC)” presents initial data from the first two cohorts (n=8) treated with BPX-601.
  • These interim results demonstrated preliminary efficacy of BPX-601 PSCA-directed GoCAR-T cells in combination with rimiducid in heavily pre-treated patients.
  • “We believe these encouraging initial clinical results in mCRPC support the potential of BPX-601 and the GoCAR-T® platform,” stated Rick Fair, President and Chief Executive Officer, Bellicum Pharmaceuticals.
  • GoCAR-T cells are designed to function optimally with repeat dosing of rimiducid to induce the co-activation molecules MyD88 and CD40.

Enlivex Announces Issuance of Israeli Patent Covering the Use of Allocetra™ to Prevent Cytokine Release Syndrome (CRS) Resulting from CAR T-Cell Therapy, Infectious Diseases or Any Non-Infectious Source of CRS

Retrieved on: 
Wednesday, February 15, 2023
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Nes-Ziona, Israel, Feb. 15, 2023 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, today announced the issuance of an Israeli patent, numbered 284985, entitled, “Combination Immune Therapy and Cytokine Control Therapy for Cancer Treatment.” The patent provides added intellectual property protection in Israel into at least 2037, with claims covering the use of for Allocetra™ for prevention or amelioration of cytokine storms in cancer patients receiving CAR-T therapy, as well as in patients whose cytokine storms result from infectious diseases or non-infectious sources.

Key Points: 
  • Nes-Ziona, Israel, Feb. 15, 2023 (GLOBE NEWSWIRE) -- Enlivex Therapeutics Ltd. (Nasdaq: ENLV, the “Company”), a clinical-stage macrophage reprogramming immunotherapy company, today announced the issuance of an Israeli patent, numbered 284985, entitled, “Combination Immune Therapy and Cytokine Control Therapy for Cancer Treatment.” The patent provides added intellectual property protection in Israel into at least 2037, with claims covering the use of for Allocetra™ for prevention or amelioration of cytokine storms in cancer patients receiving CAR-T therapy, as well as in patients whose cytokine storms result from infectious diseases or non-infectious sources.
  • CAR T-cells are T-cells that have been genetically modified to include a receptor that allows them to specifically target and destroy cancer cells.
  • While certain CAR T-cell treatments were recently approved by the FDA in several cancer indications, such treatments have been associated in many patients with a side effect named cytokine release syndrome, which describes a collection of potentially severe or life-threatening symptoms that stem from over-activation of immune pathways.
  • Preclinical data indicate that Allocetra™ has the potential to prevent or ameliorate cytokine release syndrome associated with CAR T-cell therapies.

Sobi to Present Data from REAL-HLH Study at the ACR Convergence 2022 Meeting

Retrieved on: 
Thursday, October 27, 2022
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Since its approval, no study has evaluated the use of Gamifant in a larger cohort of patients in the real-world clinical setting.

Key Points: 
  • Since its approval, no study has evaluated the use of Gamifant in a larger cohort of patients in the real-world clinical setting.
  • Currently, Gamifant is not approved by the FDA for the treatment of patients with secondary HLH.
  • The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899).
  • To report suspected adverse reactions, contact Sobi North America at 1-866-773-5274 or FDA at 1-800-FDA-1088.

Elixiron Immunotherapeutics Announces Orphan Drug Designation Granted to Interferon-Gamma-Neutralizing Antibody, EI-001 for the Treatment of Hemophagocytic Lymphohistiocytosis

Retrieved on: 
Tuesday, September 6, 2022
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SAN FRANCISCO, Sept. 06, 2022 (GLOBE NEWSWIRE) -- Elixiron Immunotherapeutics, a clinical stage biotech company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its interferon-gamma-neutralizing human antibody, EI001 for the treatment of hemophagocytic lymphohistiocytosis (HLH).

Key Points: 
  • SAN FRANCISCO, Sept. 06, 2022 (GLOBE NEWSWIRE) -- Elixiron Immunotherapeutics, a clinical stage biotech company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its interferon-gamma-neutralizing human antibody, EI001 for the treatment of hemophagocytic lymphohistiocytosis (HLH).
  • "The Orphan Drug Designation will definitely accelerate our ability to bring EI001 to market sooner rather than later, to offer patients what we feel will be a better treatment than currently-available options."
  • Elixiron Immunotherapeutics is developinginnovative immunotherapies to address unmet needs in rare and immunological diseases.
  • EI1071 is being developed for the treatment of Alzheimer's disease with funding support in part from the Alzheimer's Association Part the Cloud program.