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Clinical Trial News

FDA Panel Rejects MDMA Therapy for PTSD, Raising Concerns About Data and Safety

  • An FDA advisory panel voted against approving MDMA-assisted therapy for PTSD, citing concerns about data credibility and research practices.
  • Issues raised include potential bias due to unblinding in trials and strong prior beliefs about treatment benefits among participants and therapists.
  • Concerns were also noted regarding a lack of diversity in trials, adverse effect data, and allegations of unethical conduct within the therapy model.
  • Despite the setback, research into psychedelic therapies for mental health conditions continues, with the VA funding further studies.

Pfizer's DMD Gene Therapy Fails to Meet Primary Endpoint in Phase III Trial

  • Pfizer's fordadistrogene movaparvovec gene therapy for Duchenne muscular dystrophy (DMD) did not meet the primary endpoint in the Phase III CIFFREO study.
  • The study, involving patients aged 4-8, assessed motor function improvement using the North Star Ambulatory Assessment (NSAA) scale after one year.
  • Key secondary endpoints, including 10-meter run/walk velocity and time to rise from the floor, also showed no significant difference compared to placebo.
  • Pfizer plans to share more detailed results from the CIFFREO study at an upcoming medical meeting.

Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

NCT04281485Active, Not RecruitingPhase 3
Pfizer
Posted 11/5/2020

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372Active, Not RecruitingPhase 2
Pfizer
Posted 8/8/2022

UroGen Pharma's UGN-102 Shows Promise in Phase 3 Trial for Bladder Cancer

• UroGen Pharma's UGN-102 met its primary endpoint in the Phase 3 ENVISION trial, demonstrating a high complete response rate at three months for low-grade non-muscle invasive bladder cancer. • Many patients in the ENVISION trial maintained a complete response to UGN-102 at the 12-month mark, indicating durable efficacy. • UroGen Pharma is advancing with a New Drug Application to the FDA for UGN-102, which could offer a significant alternative to current standard procedures. • The potential market opportunity for UGN-102 is estimated to exceed $5 billion, highlighting the unmet need and potential impact of this novel treatment.

UroGen Pharma's UGN-102 Shows Promise in Bladder Cancer Treatment, Faces Market Cautiousness

• UroGen Pharma's UGN-102 demonstrated promising 12-month durability results in a Phase 3 ENVISION study, exceeding expectations for bladder cancer treatment. • Goldman Sachs analyst Paul Choi maintains a Hold rating on UroGen Pharma, citing uncertainty regarding UGN-102's ability to replace the standard TURBT surgery. • Despite positive clinical data and potential for priority review, caution is advised due to the entrenched nature of current surgical practices among urologists. • Increased peak sales and market penetration projections for UGN-102 are noted, pending further regulatory information and assessment of UroGen Pharma's performance.

Formycon Advances Phase III Trial for Keytruda Biosimilar FYB206 in Non-Small Cell Lung Cancer

Monoclonal Antibody
  • Formycon AG has enrolled the first patient in its Phase III "Lotus" trial comparing FYB206 with Keytruda in non-small cell lung cancer patients.
  • The double-blind, multicenter study will evaluate best overall response rate over 17 treatment cycles within 52 weeks, followed by an additional 12 months of therapy.
  • FYB206 targets the world's best-selling drug Keytruda, which generated $25 billion in sales in 2023 and could reach $30 billion by 2026.
  • The biosimilar candidate is expected to enter the market after Keytruda's exclusivity expires in 2029 (USA) and 2030 (EU), with Phase III results anticipated in 2027.

Avidity's Delpacibart Etedesiran Granted FDA Breakthrough Therapy Designation for Myotonic Dystrophy Type 1

  • Avidity Biosciences' delpacibart etedesiran (del-desiran; AOC 1001) receives Breakthrough Therapy Designation from the FDA for treating myotonic dystrophy type 1 (DM1).
  • The designation is supported by positive long-term data from the Phase 2 MARINA-OLE trial, demonstrating improvements in muscle strength, myotonia, and activities of daily living.
  • Del-desiran, an antibody oligonucleotide conjugate (AOC), targets DMPK mRNA to address the underlying cause of DM1, a rare and debilitating muscle disease.
  • A global Phase 3 HARBOR study is planned to begin in the second quarter of 2024, with endpoints including video hand opening time and muscle strength measurements.

Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS)

NCT02970162CompletedPhase 3
Catalyst Pharmaceuticals, Inc.
Posted 11/1/2016

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia

NCT05579691CompletedPhase 2
Larimar Therapeutics, Inc.
Posted 9/21/2022

A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)

NCT01377922CompletedPhase 3
Catalyst Pharmaceuticals, Inc.
Posted 6/1/2011

A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)

NCT04993768Active, Not RecruitingPhase 2
Transposon Therapeutics, Inc.
Posted 10/15/2021

Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients

NCT05479981Active, Not RecruitingPhase 2
Avidity Biosciences, Inc.
Posted 8/4/2022

Setrusumab Shows Sustained Fracture Reduction in Osteogenesis Imperfecta Patients

  • Setrusumab demonstrates a 67% reduction in annualized fracture rate over 14 months in osteogenesis imperfecta (OI) patients.
  • Treatment with setrusumab led to a significant 22% increase in lumbar spine bone mineral density (BMD) from baseline.
  • The mean improvement in lumbar spine BMD Z-score was +1.25, indicating substantial normalization in bone density.
  • No treatment-related serious adverse events were observed, supporting the safety profile of setrusumab.

Setrusumab vs Placebo for Osteogenesis Imperfecta

NCT05125809Active, Not RecruitingPhase 2
Ultragenyx Pharmaceutical Inc
Posted 2/21/2022

EHA 2024: Monoclonal Antibodies Show Promise in Multiple Myeloma Treatment

  • The DREAMM-8 trial demonstrated that Blenrep combined with pomalidomide and dexamethasone significantly improved progression-free survival in relapsed/refractory multiple myeloma (RRMM) patients compared to pomalidomide, bortezomib, and dexamethasone.
  • Linvoseltamab monotherapy in the LINKER-MM1 study showed substantial efficacy in RRMM patients, with a high objective response rate and durable responses, marking it as a promising treatment option.
  • The GMMG-HD7 trial indicated that adding Sarclisa to standard-of-care induction therapy resulted in deeper responses and higher rates of minimal residual disease negativity in newly diagnosed multiple myeloma patients.

A Study to Evaluate Daratumumab in Transplant Eligible Participants With Previously Untreated Multiple Myeloma

NCT02541383CompletedPhase 3
Intergroupe Francophone du Myelome
Posted 9/1/2015

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

Daratumumab, VELCADE (Bortezomib), Lenalidomide and Dexamethasone Compared to VELCADE, Lenalidomide and Dexamethasone in Subjects With Previously Untreated Multiple Myeloma

NCT03710603Active, Not RecruitingPhase 3
Stichting European Myeloma Network
Posted 12/14/2018

IMMUNOPLANT for Newly Diagnosed Multiple Myeloma

NCT06376526RecruitingPhase 2
Dickran Kazandjian, MD
Posted 8/21/2024

Study Comparing Daratumumab, Lenalidomide, Bortezomib, and Dexamethasone (D-RVd) Versus Lenalidomide, Bortezomib, and Dexamethasone (RVd) in Subjects With Newly Diagnosed Multiple Myeloma

NCT02874742CompletedPhase 2
Janssen Research & Development, LLC
Posted 8/29/2016

Aldeyra Therapeutics Completes Enrollment in Phase III Dry Eye Disease Trial of Reproxalap

  • Aldeyra Therapeutics has finished enrolling 132 subjects for its Phase III clinical trial evaluating topical ocular 0.25% reproxalap for dry eye disease.
  • The trial assesses the primary endpoint of ocular discomfort to support a potential resubmission of a New Drug Application (NDA) in the second half of 2024.
  • Reproxalap previously demonstrated a statistically significant reduction in ocular discomfort compared to a vehicle in combined data from four completed dry eye chamber trials.
  • Aldeyra aims to propose a draft label highlighting both chronic and acute symptom improvement, along with a reduction in ocular redness, for reproxalap.

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