Investigational New Drug

Synthekine Announces U.S. FDA Clearance of IND Application for CD19 CAR-T and Orthogonal IL-2 Combination Therapy, SYNCAR-001 + STK-009, for Treatment of Lupus Without Lymphodepletion

Retrieved on: 
Tuesday, March 26, 2024
Biotechnology, FDA, Health, Pharmaceutical, Clinical Trials, LN, Risk, Patient, NYU, SLE, IND, Investigational New Drug, Safety, Lupus nephritis, Inc., CD19, Food, Medicine, Pharmaceutical industry

SYNCAR-001 + STK-009 is a cytokine-inducible cell therapy regimen built on Synthekine’s proprietary orthoIL-2 technology.

Key Points: 
  • SYNCAR-001 + STK-009 is a cytokine-inducible cell therapy regimen built on Synthekine’s proprietary orthoIL-2 technology.
  • The SYNCAR-001 + STK-009 combination therapy is in a Phase 1 study ( NCT05665062 ) in CD19+ hematologic malignancies.
  • “However, successful treatment with CD19 CAR-Ts requires patients to be lymphodepleted, a significant burden on the patient, physician and healthcare system.
  • By leveraging Synthekine’s orthoIL-2 technology, STK-009 has the potential to drive SYNCAR-001 cell engraftment and controlled cell expansion without lymphodepletion.

InnoCare Announces Approval of Clinical Trial of BCL2 Inhibitor ICP-248 in Combination with Orelabrutinib as First-Line Therapy for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma in China

Retrieved on: 
Wednesday, March 13, 2024
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, SLL, CLL, Hematology, E-3, Incidence, Patient, Malignancy, BCL2, Cancer, Leukemia, Death, IND, Clinical trial, Investigational New Drug, HKEX, BTK, Safety, Marketing, NRDL, Lymphoma, CD19, Pharmaceutical industry

This multicenter, randomized-controlled, open-label clinical study is designed to evaluate the efficacy and safety of ICP-248 combined with orelabrutinib versus immunochemotherapy in treatment-naive patients with CLL/SLL.

Key Points: 
  • This multicenter, randomized-controlled, open-label clinical study is designed to evaluate the efficacy and safety of ICP-248 combined with orelabrutinib versus immunochemotherapy in treatment-naive patients with CLL/SLL.
  • ICP-248 is a novel, orally bioavailable BCL2-selective inhibitor, which aims to treat hematologic malignancies as a monotherapy or in combination with other therapies.
  • BCL2 is an important regulatory protein of apoptosis pathway, and its abnormal expression is related to the development of various hematologic malignancies.
  • ICP-248 has an anti-tumor effect by selectively inhibiting BCL2 and restoring the mechanism of programmed cell death.

Legend Biotech Reports Fourth Quarter and Full Year 2023 Results and Recent Highlights

Retrieved on: 
Monday, March 11, 2024
Research, FDA, Clinical Trials, Other Health, Biotechnology, Pharmaceutical, Health, Science, Oncology, Other Science, Johnson & Johnson, Growth, Drug discovery, Exercise, Partnership, Webcast, Patient, Committee, Collaboration, Nature, Senior, Lenalidomide, Investment, Investigational New Drug, Delta, ODAC, Janssen, CHMP, Food

Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, today reported its fourth quarter and full year 2023 unaudited financial results and key corporate highlights.

Key Points: 
  • Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, today reported its fourth quarter and full year 2023 unaudited financial results and key corporate highlights.
  • Financial Results for Quarter and Year Ended December 31, 2023
    As of December 31, 2023, Legend Biotech had approximately $1.3 billion of cash and cash equivalents, time deposits, and short-term investments.
  • There was no license revenue for the three months ended December 31, 2023, and December 31, 2022.
  • License revenue for the year ended December 31, 2023, was $35.2 million, compared to $50.0 million for the year ended December 31, 2022.

MOMA Therapeutics Appoints Jullian G. Jones, Ph.D., J.D., MBA, as Chief Business Officer

Retrieved on: 
Monday, March 11, 2024
Biotechnology, Pharmaceutical, Health, Oncology, MOMA, Georgetown University Law Center, Eli Lilly, Growth, Learning, Science, Partnership, Boehringer Ingelheim, Patient, SVP, Roche, Acquisition, Cancer, IPO, Thought, MBA, Doctor of Philosophy, Investigational New Drug, Johns Hopkins University, Therapy, Heart, Eli Lilly and Company, FDA, Knowledge

MOMA Therapeutics, a biopharmaceutical company discovering and developing a new generation of precision therapeutics via insights derived from the company’s KnowledgeBase platform, today announced that Jullian G. Jones, Ph.D., J.D., MBA, has been appointed as the company’s chief business officer.

Key Points: 
  • MOMA Therapeutics, a biopharmaceutical company discovering and developing a new generation of precision therapeutics via insights derived from the company’s KnowledgeBase platform, today announced that Jullian G. Jones, Ph.D., J.D., MBA, has been appointed as the company’s chief business officer.
  • “We are thrilled to welcome Jullian to MOMA.
  • She has an exceptional track record of thinking and acting strategically and creatively across different business dimensions, while leading with heart and a passion for mentorship,” said Asit Parikh, M.D., Ph.D., chief executive officer of MOMA.
  • “Jullian’s expertise is perfectly aligned with MOMA’s business needs at this stage of our growth and positions us more strongly for success.

IASO Bio Announces U.S. FDA Approval of Investigational New Drug Application for BCMA CAR-T Equecabtagene Autoleucel for Generalized Myasthenia Gravis

Retrieved on: 
Thursday, April 4, 2024
Vital capacity, MuSK protein, Glucocorticoid, NMPA, Hormone, GMG, Disease, Chinese, MRS, BCMA, Nervous system, Patient, SAN, Enzyme inhibitor, History, Female, Multiple myeloma, CRS, B cell, Biotechnology, IND, Phenotype, Investigational New Drug, QMG, Safety, Thymectomy, FDA, Food

The Chinese IND for this indication of Equecabtagene Autoleucel was approved by the NMPA in January this year.

Key Points: 
  • The Chinese IND for this indication of Equecabtagene Autoleucel was approved by the NMPA in January this year.
  • The 2 subjects were treated with a single infusion of Eque-cel at the doses of 1.0×106 CAR-T/Kg, respectively.
  • No immunomodulatory therapy other than low dose pyridostigmine (90 mg/day and 60 mg/day, respectively) was used during the follow-up period.
  • Anti-AChR antibodies, anti-Titin antibodies, and anti-MuSK antibodies decreased rapidly and maintained at very low levels in both subjects after infusion.

Verismo Therapeutics Announces Submission of IND Application to the FDA for SynKIR™-310 in Relapsed/Refractory B-cell NHL

Retrieved on: 
Wednesday, April 3, 2024
Malignancy, MCL, Patient, B-cell lymphoma, Standard of care, Follicular lymphoma, Trial of the century, NHL, IND, Verismo, Clinical trial, Investigational New Drug, Therapy, Mantle cell lymphoma, FDA, NK, DLBCL, B cell, CD19, Food, Bone marrow, Pharmaceutical industry

PHILADELPHIA, April 3, 2024 /PRNewswire/ -- Verismo Therapeutics, a clinical-stage CAR T company developing the novel KIR-CAR platform technology, today announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its Phase 1 clinical trial of SynKIR™-310, for the treatment of relapsed/refractory (r/r) B-cell Non-Hodgkin Lymphomas (B-cell NHL), including Diffuse Large B Cell lymphoma (DLBCL), Follicular Lymphoma (FL), Mantle Cell Lymphoma (MCL), and Marginal Zone Lymphoma (MZL).

Key Points: 
  • Commercially approved CAR T cell therapies have shown impressively high initial response rates in blood cancers.
  • Over time, however, these therapies result in relapse in an estimated 40-50% of patients1.
  • There are currently very limited treatment options for patients with r/r DLBCL who relapse following treatment with commercial CAR T cell therapies.
  • Initiation of the clinical trial is the culmination of years of focused research and diligent work by the Verismo team."

SpliSense Announces FDA Clearance of Investigational New Drug Application for Phase 2 Initiation of SPL84 for the Treatment of Cystic Fibrosis

Retrieved on: 
Wednesday, April 3, 2024
Mutation, Disease, RNA, Doctor of Philosophy, IND, Life expectancy, Asthma, IPF, Idiopathic pulmonary fibrosis, Investigational New Drug, Bronchiectasis, CFTR, COPD, FDA, Cystic fibrosis, Food, Pharmaceutical industry

JERUSALEM, April 3, 2024 /PRNewswire/ -- SpliSense, a clinical-stage biotechnology company focused on the development of transformative RNA-based therapies for pulmonary diseases including cystic fibrosis (CF), muco-obstructive diseases like COPD, asthma, non-CF bronchiectasis (NCFB) and idiopathic pulmonary fibrosis (IPF), today announced that the U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application for the initiation of a Phase 2 study for SPL84 in CF. SPL84 is the Company's lead antisense oligonucleotide (ASO) product for the treatment of people with CF carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Key Points: 
  • SPL84 is the Company's lead antisense oligonucleotide (ASO) product for the treatment of people with CF carrying the 3849+10 kilobase (Kb) C->T splicing mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
  • The Company has also announced today that it has secured funding from the CF Foundation and other existing investors to support the SPL84 Phase 2 study and additional pulmonary programs.
  • "We are excited to initiate the Phase 2 study for SPL84 in CF with the support of the CF Foundation and other existing investors," said Gili Hart, PhD, Chief Executive Officer of SpliSense. "
  • Our lead product, SPL84, has been shown to fully restore CFTR activity in the CF gold standard pharmacological model.

RemeGen's Telitacicept (RC18) Granted Fast Track Designation by United States FDA for Treatment of Primary Sjögren's Syndrome

Retrieved on: 
Wednesday, April 3, 2024
Rheumatoid arthritis, Autoimmunity, Lupus, Risk, Rheumatism, Patient, PSS, SLE, Diabetic nephropathy, Myasthenia gravis, IND, Mouth, Investigational New Drug, Cytokine, B-cell activating factor, Iga, FDA, FTD

The FDA grants FTD to accelerate the development and review of new drugs that have the potential to treat life-threatening conditions and address unmet medical needs.

Key Points: 
  • The FDA grants FTD to accelerate the development and review of new drugs that have the potential to treat life-threatening conditions and address unmet medical needs.
  • Telitacicept prevents abnormal differentiation and maturation of B-cells through a two-pronged approach, thereby treating various autoimmune diseases mediated by B-cells.
  • In December 2023, Telitacicept was granted Investigational New Drug (IND) by the FDA for the treatment of active pSS in adults.
  • Dr. Jianmin Fang, CEO of RemeGen, commented, "As the first fusion protein drug of its kind independently developed by RemeGen, we are delighted the FDA has recognized Telitacicept for fast track designation.

MIRA Pharmaceuticals Welcomes Dr. Itzchak Angel, former Head of Pharmacology at Synthelabo, as Chief Scientific Advisor

Retrieved on: 
Thursday, March 21, 2024
Discovery, Research, Depression, Epilepsy, MIRA, Development, Shire (pharmaceutical company), Stroke, Litoxetine, IND, VPA, Sanofi, Clinical trial, Research and development, Investigational New Drug, Drug development, Marketing, Pharmaceutical industry

MIAMI, March 21, 2024 /PRNewswire/ -- MIRA Pharmaceuticals Inc. (NASDAQ: MIRA) ("MIRA" or the "Company"), an innovative pre-clinical-stage pharmaceutical company focused on the development of novel treatments for neurologic and neuropsychiatric disorders, proudly announces the appointment of distinguished pharmaceutical executive Dr. Itzchak Angel as its new Chief Scientific Advisor.

Key Points: 
  • Dr. Angel brings over 40 years of preclinical, clinical and regulatory acumen to MIRA.
  • He has significantly contributed to the research, development and regulatory approval of groundbreaking medications over his remarkable career.
  • Additionally, his activities contributed to the development and marketing of Mizollen (mizolastine), Xatral (alfuzosin) and Ambien (zolpidem).
  • "We are truly honored to have Dr. Angel on our team," stated Erez Aminov, Chairman & CEO of MIRA.

Sanyou Bio and BioGeometry join forces to create a next-generation Antibody Drug Discovery Platform driven by AIGC

Retrieved on: 
Tuesday, March 19, 2024
Software as a service, Drug discovery, Intelligence, Partnership, Research, AI, IND, Investigational New Drug, Drug development, Fed, Fine chemical

In this strategic cooperation, Sanyou Bio will integrate BioGeometry's proprietary generative AI antibody design platform (GeoBiologics).

Key Points: 
  • In this strategic cooperation, Sanyou Bio will integrate BioGeometry's proprietary generative AI antibody design platform (GeoBiologics).
  • Moreover, BioGeometry has achieved a closed-loop between dry and wet lab on its generative AI antibody discovery SaaS platform, GeoBiologics.
  • Dr. Guojun Lang, founder and CEO of Sanyou Bio, remarked: "BioGeometry's generative AI technology has enabled Sanyou to achieve intelligent and digital transformation of our antibody drug R&D platform, further strengthening our advantages in new drug discovery and preclinical integration.
  • Dr. Jian Tang, founder and CEO of BioGeometry, stated: "We look forward to jointly creating with Sanyou Bio a cutting-edge AI-enabled antibody discovery platform combining dry and wet labs.