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Clinical Trial News

Amphista Therapeutics Hits Second Milestone in Bristol Myers Squibb Protein Degradation Partnership

Drug Discovery
  • Amphista Therapeutics achieved the first milestone in its second discovery programme with Bristol Myers Squibb, triggering a milestone payment under their strategic collaboration.
  • The achievement follows a previous milestone reached in May 2023 for the first discovery programme, demonstrating consistent progress across both active programmes.
  • The collaboration, valued at up to $1.25 billion in potential milestone payments, focuses on developing small molecule protein degraders using Amphista's EclipsysTM platform.
  • Amphista's targeted protein degradation approach shows potential for CNS penetration, expanding therapeutic applications beyond traditional modalities.

Actinogen Optimizes XanaMIA Phase 2b Alzheimer's Disease Trial Design

Actinogen Medical Limited has announced optimizations to its XanaMIA Phase 2b trial for mild-to-moderate Alzheimer's disease, reducing costs by AUD 30 million and speeding up initial results. The trial now focuses on a 10 mg dose group, enrolling 220 patients, and includes Australian sites for the first 100 patients. Additionally, the XanaCIDD trial for cognitive impairment associated with depression has expanded to include UK sites, with results expected by mid-2024.

Xeltis Initiates Pivotal Trial for aXess Graft in Hemodialysis Patients

• Xeltis has commenced a pivotal European clinical trial to assess the safety and efficacy of its aXess graft in patients with end-stage renal disease requiring hemodialysis. • The prospective, single-arm study aims to enroll 110 patients across up to 25 European centers, monitoring them over five years post-implantation. • aXess, designed for immediate use and to transform into a living blood vessel, has shown promising preliminary results in a first-in-human trial. • The trial seeks to validate aXess as a potentially safer and longer-lasting solution, reducing complications associated with repeated dialysis punctures.

Resminostat Receives Orphan Drug Designations in US and EU for CTCL Treatment

  • Resminostat (Kinselby), a histone deacetylase (HDAC) inhibitor developed by 4SC AG, has been granted Orphan Drug Designation (ODD) by the FDA for cutaneous T-cell lymphoma (CTCL).
  • The European Medicines Agency (EMA) has also granted Orphan Drug Designation to resminostat for the treatment of CTCL, providing 10 years of market exclusivity in the EU.
  • 4SC anticipates submitting a Marketing Authorisation Application (MAA) to the EU for Kinselby (resminostat) in Q1 2024, marking a significant step toward potential approval.

Novel Targets and Strategic Sequencing: The Evolving Landscape of T-Cell Redirection Therapies in Multiple Myeloma

OncologyCAR-T
  • CAR T-cell therapies and bispecific antibodies targeting BCMA and GPRC5D are transforming multiple myeloma treatment, with ciltacabtagene autoleucel showing unprecedented efficacy with a median PFS of 34.9 months in heavily pretreated patients.
  • Disease progression rate is a critical factor in therapy selection, with rapidly progressing patients better suited for "off-the-shelf" bispecific antibodies, while patients with indolent disease may benefit more from CAR T-cell therapies despite longer manufacturing times.
  • Strategic sequencing of therapies is crucial, as prior BCMA-directed bispecific antibody treatment significantly reduces the efficacy of subsequent BCMA-targeted CAR T-cell therapy, highlighting the importance of preserving T-cell fitness and considering antigen loss.

A Study of Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Ciltacabtagene Autoleucel Versus Daratumumab, Bortezomib, Lenalidomide and Dexamethasone (DVRd) Followed by Autologous Stem Cell Transplant (ASCT) in Participants With Newly Diagnosed Multiple Myeloma

NCT05257083RecruitingPhase 3
Stichting European Myeloma Network
Posted 10/10/2023

A Study of CAR-T Cells Targeting Both BCMA and GPRC5D in Treatment of Relapsed or Refractory Multiple Myeloma

NCT05325801Not Yet RecruitingPhase 1
Zhejiang University
Posted 4/1/2022

A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma

NCT03548207CompletedPhase 1
Janssen Research & Development, LLC
Posted 6/29/2018

A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-cell Maturation Antigen (BCMA) in Participants With Multiple Myeloma

NCT04133636RecruitingPhase 2
Janssen Research & Development, LLC
Posted 11/7/2019

Safety and Efficacy of ALLO-715 BCMA Allogenic CAR T Cells in in Adults With Relapsed or Refractory Multiple Myeloma (UNIVERSAL)

NCT04093596Active, Not RecruitingPhase 1
Allogene Therapeutics
Posted 9/23/2019

Efficacy and Safety Study of bb2121 in Subjects With Relapsed and Refractory Multiple Myeloma

NCT03361748CompletedPhase 2
Celgene
Posted 12/13/2017

Efficacy and Safety Study of bb2121 Versus Standard Regimens in Subjects With Relapsed and Refractory Multiple Myeloma (RRMM)

NCT03651128Active, Not RecruitingPhase 3
Celgene
Posted 4/16/2019

A Study of Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT04557098Active, Not RecruitingPhase 2
Janssen Research & Development, LLC
Posted 9/17/2020

Dose Escalation Study of Talquetamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT03399799Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 12/16/2017

A Study of MCARH109 and MCARH125 in People With Multiple Myeloma

NCT05431608Active, Not RecruitingPhase 1
Memorial Sloan Kettering Cancer Center
Posted 6/20/2022

A Study of the Combination of Talquetamab and Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT04586426Active, Not RecruitingPhase 1
Janssen Research & Development, LLC
Posted 12/15/2020

A Study of CAR-T Cells Targeting GPRC5D in the Treatment of r/r Multiple Myeloma

NCT05016778Active, Not RecruitingEarly Phase 1
Zhejiang University
Posted 6/8/2021

MagnetisMM-3: Study Of Elranatamab (PF-06863135) Monotherapy in Participants With Multiple Myeloma Who Are Refractory to at Least One PI, One IMiD and One Anti-CD38 mAb

NCT04649359Active, Not RecruitingPhase 2
Pfizer
Posted 2/2/2021

MCARH109 Chimeric Antigen Receptor (CAR) Modified T Cells for the Treatment of Multiple Myeloma

NCT04555551Active, Not RecruitingPhase 1
Memorial Sloan Kettering Cancer Center
Posted 9/8/2020

A Study of Bortezomib, Lenalidomide and Dexamethasone (VRd) Followed by Cilta-cel, a CAR-T Therapy Directed Against BCMA Versus VRd Followed by Lenalidomide and Dexamethasone (Rd) Therapy in Participants With Newly Diagnosed Multiple Myeloma for Whom ASCT is Not Planned as Initial Therapy

NCT04923893Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 8/19/2021

A Study of Talquetamab in Participants With Relapsed or Refractory Multiple Myeloma

NCT04634552RecruitingPhase 2
Janssen Research & Development, LLC
Posted 2/1/2021

A Study Comparing JNJ-68284528, a CAR-T Therapy Directed Against B-cell Maturation Antigen (BCMA), Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd) in Participants With Relapsed and Lenalidomide-Refractory Multiple Myeloma

NCT04181827Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 6/12/2020

Safety and Efficacy of Anti-BCMA/GPRC5D CAR-T Cell Therapy in Treating Relapsed and Refractory Multiple Myeloma(rr/MM)

NCT05509530RecruitingPhase 2
Xuzhou Medical University
Posted 5/1/2022

AI-Powered HASTEN Tool Accelerates Virtual Drug Screening 10-Fold

  • Finnish researchers have developed an ML-powered tool called HASTEN that reduces virtual drug screening time from 6 months to under 10 days for 1.56 billion molecules.
  • The system successfully identified 90% of top-scoring drug candidates when screening just 1% of the compound library against bacterial and viral targets.
  • Orion Pharma and University of Eastern Finland researchers have made their datasets public to advance computational drug discovery research.

Leucid Bio's Lateral NKG2D CAR-T Therapy LEU011 Receives MHRA Approval for Solid Tumor Clinical Trial

  • Leucid Bio has received MHRA clinical trial authorization for LEU011, a novel lateral CAR-T therapy targeting NKG2D ligands expressed in over 80% of human tumors.
  • The Phase 1/2 AERIAL trial will evaluate LEU011 in patients with relapsed or refractory solid tumors, with the first patient expected to be dosed in Q4 2023.
  • LEU011 previously received the Innovation Passport designation from MHRA, highlighting its potential as a breakthrough therapy for solid tumors with limited treatment options.

FDA Grants Orphan Drug Designation to Immix Biopharma's NXC-201 for AL Amyloidosis

  • Immix Biopharma's NXC-201, a CAR-T cell therapy, receives Orphan Drug Designation from the FDA for treating Amyloid Light Chain (AL) Amyloidosis.
  • The designation provides Immix Biopharma with benefits including potential market exclusivity, tax credits, and a waiver for prescription drug user fees.
  • NXC-201 is currently in Phase 1b/2a clinical trials and has demonstrated a 100% hematologic response rate in AL amyloidosis patients in early trials.
  • AL Amyloidosis, a rare disorder affecting 30,000-40,000 individuals in the US and Europe, has a market projected to reach $6 billion by 2025.

NXC-201 (formerly HBI0101) Multiple Myeloma

NCT04720313Active, Not RecruitingPhase 1
Hadassah Medical Organization
Posted 1/1/2021

Study of IMX-110 in Combination With Tislelizumab in Patients With Advanced Solid Tumors

NCT05840835RecruitingPhase 1
Immix Biopharma, Inc.
Posted 2/3/2023

Travere Therapeutics' Filspari Nears, But Misses, Primary Endpoint in IgA Nephropathy Trial

  • Travere Therapeutics' Filspari narrowly missed statistical significance in a Phase 3 trial for IgA nephropathy, a rare kidney disorder, with a p-value of 0.058.
  • The trial involved 404 patients randomized to receive Filspari or irbesartan, an existing blood pressure medication used to manage the disease.
  • While early data showed a 50% reduction in urine protein levels, the 24-month data on kidney function decline narrowly missed the primary endpoint.
  • Filspari received accelerated approval in February based on earlier biomarker data, with full approval contingent on these confirmatory trial results.

First RoActemra Biosimilar Tyenne Receives EU Approval, Marking Milestone for Fresenius Kabi

  • The European Commission has granted approval for Tyenne, the first biosimilar version of Roche's blockbuster drug RoActemra (tocilizumab), developed by Fresenius Kabi for multiple inflammatory conditions and severe COVID-19.
  • Tyenne is approved for all RoActemra indications, including rheumatoid arthritis, systemic juvenile idiopathic arthritis, and cytokine release syndrome, representing a significant advancement in accessible treatment options.
  • This approval marks Fresenius Kabi's third biosimilar success and aligns with their Vision 2026 strategy to generate €1 billion in additional revenue every three years.

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