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Clinical Trial News

BioNTech Shuts Down Maryland Cell Therapy Manufacturing Following CAR-T Trial Failure

CAR-TOncology
  • BioNTech will lay off 63 employees and wind down cell therapy manufacturing at its Gaithersburg, Maryland facility by the end of 2025 following disappointing Phase 1 trial results.
  • The company discontinued development of its CAR-T candidate BNT211 targeting CLDN6 in testicular cancer and germ cell tumors due to insufficient efficacy data.
  • Despite the setback, BioNTech continues studying BNT211 in other CLDN6-expressing cancers including ovarian, sarcoma, endometrial, and gastric cancers.
  • The facility closure is part of broader oncology pipeline restructuring as BioNTech realigns resources and reduces global workforce by up to 1,350 jobs by 2027.

FDA Issues Critical Safety Warning for Scopolamine Patch Following 13 Global Hyperthermia Cases

  • The FDA has identified 13 worldwide cases of hyperthermia associated with scopolamine patches, including two deaths and four hospitalizations, with most cases occurring in vulnerable populations under 17 or over 60 years old.
  • Three patients experienced dangerous body temperatures exceeding 105°F, with symptoms typically appearing within 72 hours of initial patch application and often accompanied by other anticholinergic effects.
  • The agency now requires updated labeling warnings and urges clinicians to exercise particular caution when prescribing to high-risk groups or during hot weather conditions.
  • Most cases involved off-label pediatric use for managing drooling and secretions in neurological conditions, despite the patch lacking FDA approval for children.

Kindeva Partners with Rocket Science Health to Advance Novel Intranasal CNS Drug Delivery Platform

  • Kindeva and Rocket Science Health have formed a non-exclusive partnership to develop the RSH Olfactory Delivery Device, a dual nostril intranasal platform that precisely targets the olfactory clefts for direct CNS drug delivery.
  • The innovative device overcomes complex nasal anatomy challenges by ejecting a laminar stream rather than conventional plume sprays, with proprietary dispensing tips extending past the nasal valve for optimal drug deposition.
  • The technology is designed with human factors considerations for accessibility and accurate dosing, particularly benefiting cognitively and physically impaired patients requiring self-administration.
  • Kindeva's CDMO expertise and regulatory pathway knowledge will support RSH's journey toward FDA approval and eventual commercial distribution of this breakthrough intranasal delivery innovation.

FDA Halts Clinical Trials Sending American Cells to Foreign Labs for Genetic Engineering

  • The FDA announced an immediate review of clinical trials that send American citizens' living cells to China and other hostile countries for genetic engineering, often without patient knowledge or consent.
  • The practice was enabled by a Biden Administration exemption to export controls that allowed biological samples including DNA to be processed overseas, even by companies partially controlled by the Chinese Communist Party.
  • The FDA will require companies to demonstrate full transparency, ethical consent, and domestic handling of sensitive biological materials before new trials can proceed.
  • NIH is reviewing its entire research portfolio to identify federally funded trials that may have utilized the exemption and compromised patient data or national security.

AI Model Reveals 46% Cognitive Decline Reduction in Alzheimer's Trial Reanalysis

AIPrecision Medicine
  • Cambridge researchers used AI to reanalyze a failed Alzheimer's clinical trial, finding the drug lanabecestat slowed cognitive decline by 46% in patients with early-stage, slow-progressing mild cognitive impairment.
  • The AI model stratified patients into slow and rapid progressors with three times greater accuracy than standard clinical assessments using memory tests, MRI scans, and blood tests.
  • This precision medicine approach could accelerate drug discovery by identifying optimal patient populations for treatment, potentially reducing the 95% failure rate and $43 billion spent on unsuccessful dementia research.
  • The findings demonstrate that while the drug cleared beta amyloid protein in both patient groups, only slow-progressing patients showed symptomatic improvements in the AMARANTH trial.

GeneCentric Secures $8M Series C to Commercialize RNA-Based Liquid Biopsy Platform

  • GeneCentric Therapeutics closed an $8 million Series C financing round led by Hatteras Venture Partners to commercialize its GenomicsNext platform, which combines gene expression analysis with DNA variant detection in liquid biopsies.
  • The company's ExpressCT technology enables measurement of gene expression from circulating tumor DNA, addressing limitations of current liquid biopsy assays that focus solely on DNA mutations.
  • GenomicsNext is expected to launch commercially in 2025 as a research tool for biopharmaceutical companies, with funding providing runway through 2026.

Avalo Therapeutics Appoints Rita Jain to Board as AVTX-009 Phase 2 Trial for Hidradenitis Suppurativa Progresses

Monoclonal Antibody
  • Avalo Therapeutics has appointed Rita Jain, M.D., a rheumatologist with over two decades of biopharmaceutical leadership experience, to its Board of Directors.
  • The appointment comes as the company advances AVTX-009, a high-affinity anti-IL-1β monoclonal antibody, through a Phase 2 LOTUS trial for hidradenitis suppurativa.
  • Dr. Jain previously served as Chief Medical Officer at ChemoCentryx, where she advanced Tavneos (avacopan), a first-in-class treatment for ANCA-associated vasculitis.
  • The Phase 2 LOTUS trial results for hidradenitis suppurativa are expected to read out in the middle of next year.

Kaiser Permanente Launches FDA-Approved Gene Therapy Program for Pediatric Sickle Cell Disease

FDA ApprovalRare Disease
  • Kaiser Permanente Southern California will launch an FDA-approved gene therapy program for pediatric sickle cell disease patients through a partnership with Downey Medical Center and City of Hope.
  • The program will serve approximately 220 children aged 12 and above currently treated by Kaiser Permanente, with the first patient scheduled for treatment this summer.
  • The gene therapy process involves extracting stem cells from the patient's blood, modifying them in a laboratory, and reinfusing them to provide a cure without requiring donor matching.
  • This treatment addresses a critical unmet need, as sickle cell disease affects approximately 100,000 Americans and reduces life expectancy by more than 20 years compared to the national average.

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

NCT03263559CompletedPhase 2
Medical College of Wisconsin
Posted 10/3/2017

FDA Approves Tafasitamab Triple Combination for Relapsed/Refractory Follicular Lymphoma

Drug ApprovalMonoclonal Antibody
  • The FDA has approved tafasitamab-cxix (Monjuvi) in combination with lenalidomide and rituximab for adult patients with relapsed or refractory follicular lymphoma.
  • The phase 3 inMIND trial demonstrated a median progression-free survival of 22.4 months with the tafasitamab combination versus 13.9 months with placebo plus lenalidomide and rituximab.
  • This approval provides the first CD19- and CD20-targeted immunotherapy combination for this patient population, offering a chemotherapy-free treatment option.
  • Serious adverse effects were reported in 33% of patients receiving tafasitamab, with serious infections occurring in 24% of patients.

A Study Evaluating Venetoclax in Combination With Azacitidine in Participants With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

NCT02942290Active, Not RecruitingPhase 1
AbbVie
Posted 1/12/2017

Phase I/II Study of AD-PluReceptor Plus Tafasitamab-cxix and Lymphodepleting Chemotherapy in Patients With Autoimmune Disorders

NCT06434363RecruitingPhase 1
M.D. Anderson Cancer Center
Posted 7/31/2024

A Phase 3 Study to Assess Efficacy and Safety of Tafasitamab Plus Lenalidomide and Rituximab Compared to Placebo Plus Lenalidomide and Rituximab in Patients With Relapsed/Refractory (R/R) Follicular Lymphoma or Marginal Zone Lymphoma.

NCT04680052Active, Not RecruitingPhase 3
Incyte Corporation
Posted 4/15/2021

Study to Evaluate Imetelstat (GRN163L) in Participants With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

NCT02598661Active, Not RecruitingPhase 2
Geron Corporation
Posted 1/12/2016

MInimal Residual Disease Adapted Strategy

NCT04934475Active, Not RecruitingPhase 3
Intergroupe Francophone du Myelome
Posted 12/8/2021

Tafasitamab and Zanubrutinib for the Treatment of Patients With Newly Diagnosed Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma, TaZA CLL Study

NCT05718869RecruitingPhase 2
City of Hope Medical Center
Posted 5/18/2023

Talazoparib + Enzalutamide vs. Enzalutamide Monotherapy in mCRPC

NCT03395197Active, Not RecruitingPhase 3
Pfizer
Posted 12/18/2017

Open Label Study to Evaluate the Safety and Efficacy of Lenalidomide With MOR00208 in Patients With R-R DLBCL

NCT02399085CompletedPhase 2
MorphoSys AG
Posted 3/29/2016

Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity In Adult Participants With Newly Diagnosed Higher-Risk Myelodysplastic Syndrome

NCT04401748Active, Not RecruitingPhase 3
AbbVie
Posted 9/10/2020

Eccogene Initiates Phase 1b Trial of Novel Oral GLP-1 Receptor Agonist AZD5004/ECC5004 in China

  • Eccogene has dosed the first patient in a Phase 1b clinical trial of AZD5004/ECC5004, an investigational oral small molecule GLP-1 receptor agonist, in China.
  • The drug is being developed for type 2 diabetes and obesity or overweight with at least one comorbidity, designed as a convenient once-daily oral therapy with no food-related restrictions.
  • AstraZeneca is currently conducting two global Phase 2b trials (VISTA and SOLSTICE) to evaluate the drug's efficacy and safety in obesity and type 2 diabetes patients.
  • The China trial represents a key milestone to support local regulatory requirements, with Eccogene retaining co-development and co-commercialization rights in the region.

Efficacy, Safety, and Tolerability of Once Daily Oral Administration of AZD5004 Versus Placebo for 26 Weeks in Adults With Type 2 Diabetes Mellitus.

NCT06579105Active, Not RecruitingPhase 2
AstraZeneca
Posted 10/8/2024

Effects of AZD5004 in Adults Who Are Living With Obesity or Overweight With at Least 1 Weight-related Comorbidity

NCT06579092Active, Not RecruitingPhase 2
AstraZeneca
Posted 10/8/2024

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