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Clinical Trial News

Cassava Sciences' Simufilam Fails Phase 3 Alzheimer's Trial, Halting Further Development

  • Cassava Sciences' Phase 3 ReThink-ALZ trial of simufilam in mild-to-moderate Alzheimer's disease did not meet its co-primary endpoints, indicating no significant cognitive or functional benefit.
  • Due to the disappointing results, Cassava Sciences is discontinuing its second Phase 3 trial, ReFocus-ALZ, and the Open Label Extension study, effectively halting simufilam's development.
  • Despite the setback, simufilam maintained a favorable safety profile throughout the trial, and the company reported $149 million in cash reserves as of Q3 2024.
  • The company intends to present detailed analyses of both studies in the future, while also expressing gratitude to patients, families, and investigators involved in the clinical program.

Simufilam 100 mg for Mild-to-Moderate Alzheimer's Disease

NCT04994483CompletedPhase 3
Cassava Sciences, Inc.
Posted 11/3/2021

Palatin Technologies Advances PL8177 for Ulcerative Colitis and Bremelanotide for Obesity

  • Palatin Technologies has completed enrollment in its Phase 2 study of oral PL8177 for ulcerative colitis, with topline data expected in Q1 2025.
  • Preclinical data suggests PL8177 may resolve damaging inflammation in the colon, offering a potential alternative to immunosuppressants.
  • Palatin also completed a Phase 2 trial of bremelanotide with tirzepatide for obesity, with topline data anticipated later this quarter.
  • The combination therapy targets the MC4R pathway, potentially leading to additive weight loss effects beyond GLP-1/GIP drugs.

Phase 2a to Evaluate PL-8177 in Subjects with Active Ulcerative Colitis (UC)

NCT05466890Active, Not RecruitingPhase 2
Palatin Technologies, Inc
Posted 9/15/2022

A Phase 2 Study Evaluating the Co-Administration of Bremelanotide with Tirzepatide for the Treatment of Obesity

NCT06565611Active, Not RecruitingPhase 2
Palatin Technologies, Inc
Posted 8/5/2024

Cassava Sciences' Simufilam Fails to Meet Endpoints in Phase 3 Alzheimer's Trial; Program Discontinued

  • Cassava Sciences' Phase 3 ReThink-ALZ trial of simufilam in mild-to-moderate Alzheimer's disease did not meet its co-primary endpoints.
  • The co-primary endpoints were change in cognition and function assessed by ADAS-COG12 and ADCS-ADL scales at week 52.
  • Due to these results, Cassava Sciences has discontinued its second Phase 3 trial, ReFocus-ALZ, and the open-label extension study.
  • Simufilam demonstrated a favorable safety profile, and Cassava plans to present detailed analyses from both studies at a future medical meeting.

Simufilam 50 mg or 100 mg for Mild-to-Moderate Alzheimer's Disease

NCT05026177TerminatedPhase 3
Cassava Sciences, Inc.
Posted 11/18/2021

Simufilam 100 mg for Mild-to-Moderate Alzheimer's Disease

NCT04994483CompletedPhase 3
Cassava Sciences, Inc.
Posted 11/3/2021

Anavex's Blarcamesine Phase IIb/III Alzheimer's Trial Data Accepted for Publication

  • Anavex Life Sciences announced the acceptance of a peer-reviewed manuscript detailing Phase IIb/III trial results of oral blarcamesine for early Alzheimer's disease.
  • The manuscript, titled 'Blarcamesine for the treatment of Early Alzheimer's Disease,' is expected to be published in Q4 2024/Q1 2025.
  • Anavex is on track for regulatory submission of oral blarcamesine to the European Medicines Agency (EMA) in the current quarter of 2024.

Icotrokinra Shows Promise in Phase 3 Trials for Plaque Psoriasis

  • Icotrokinra, an oral peptide targeting the IL-23 receptor, met primary endpoints in two Phase 3 trials for moderate-to-severe plaque psoriasis.
  • The ICONIC-LEAD trial demonstrated that 64.7% of patients achieved clear or almost clear skin at week 16, significantly higher than placebo.
  • In the ICONIC-TOTAL trial, icotrokinra also met the primary endpoint of achieving clear or almost clear skin at week 16 compared to placebo.
  • Icotrokinra, developed by Johnson & Johnson and Protagonist Therapeutics, could offer a convenient once-daily oral therapy option.

Merck's Winrevair Demonstrates Significant Morbidity and Mortality Reduction in PAH Patients

  • Merck's Winrevair (sotatercept-csrk) met the primary endpoint in the Phase 3 ZENITH trial, showing a significant reduction in morbidity or mortality events in PAH patients.
  • The independent data monitoring committee recommended early termination of the ZENITH trial due to Winrevair's overwhelming efficacy, offering all participants access to the drug.
  • Winrevair is already approved in the U.S. and 36 other countries, with plans to present ZENITH results to regulatory authorities for further approvals.
  • The ZENITH trial results indicate that Winrevair has the potential to be practice-changing in the management of pulmonary arterial hypertension.

FDA Approves BridgeBio's Attruby (acoramidis) for Transthyretin Amyloid Cardiomyopathy

• The FDA has approved BridgeBio's Attruby (acoramidis) for adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and life-threatening heart condition. • Attruby is the first and only approved treatment that specifies near-complete stabilization of transthyretin (TTR), addressing the root cause of ATTR-CM. • Clinical trials demonstrated that Attruby significantly reduced cardiovascular death and hospitalization, while also improving the quality of life for patients. • BridgeBio's Attruby is poised to compete with Pfizer's Vyndaqel in the ATTR-CM market, offering a new treatment option for patients.

China Approves MSD's Welireg (belzutifan) for VHL Disease-Associated Tumors

  • China's NMPA has approved MSD's Welireg (belzutifan) for treating specific von Hippel-Lindau (VHL) disease-associated tumors in adults, including RCC, CNS hemangioblastomas, and pNET.
  • The approval was based on the Phase II LITESPARK-004 trial, which demonstrated promising objective response rates (ORR) and duration of response (DOR) in patients with VHL-associated tumors.
  • Welireg offers the first systemic therapy option for Chinese adults with VHL disease-associated tumors who are not candidates for immediate surgery, addressing a significant unmet need.
  • LITESPARK-004 trial data showed ORRs of 49% for RCC, 63% for CNS hemangioblastomas, and 83% for pNET, highlighting the drug's potential across various VHL-related tumor types.

Eflornithine Combo Fails to Meet Primary Endpoint in Phase III Brain Cancer Trial

• Orbus Therapeutics' eflornithine combination with lomustine did not meet the primary endpoint of overall survival in the Phase III STELLAR trial for recurrent IDH mutant astrocytoma. • The combination therapy showed clinically meaningful improvements in overall survival (OS) and progression-free survival (PFS) compared to lomustine alone. • The STELLAR trial involved 343 patients and the combination therapy was well-tolerated, with common adverse events related to myelosuppression and hearing impairment. • Eflornithine, which blocks ornithine decarboxylase, previously received FDA orphan drug and breakthrough designations for anaplastic glioma.

Study to Evaluate Eflornithine + Lomustine vs Lomustine in Recurrent Anaplastic Astrocytoma (AA) Patients

NCT02796261Unknown StatusPhase 3
Orbus Therapeutics, Inc.
Posted 7/1/2016

Edwards Lifesciences' SAPIEN 3 RESILIA Valve Shows Strong One-Year Outcomes in TAVI Trial

  • Edwards Lifesciences reports that patients treated with the SAPIEN 3 RESILIA valve experienced outstanding one-year outcomes, including lower mortality rates.
  • The PARTNER II trial, involving over 9,000 patients, demonstrated low reintervention rates and larger effective orifice areas with the SAPIEN 3 Ultra RESILIA valve.
  • The study found no paravalvular leak in 84.4% of cases using the SAPIEN 3 Ultra RESILIA valve, indicating improved valve performance and patient outcomes.
  • These real-world data reinforce the benefits of the SAPIEN valve platform, particularly in low surgical risk patients with longer life expectancies.

PARTNER II Trial: Placement of AoRTic TraNscathetER Valves II - XT Intermediate and High Risk

NCT01314313CompletedNot Applicable
Edwards Lifesciences
Posted 3/1/2011

RHEIA (Randomized researcH in womEn All Comers With Aortic Stenosis)

NCT04160130Active, Not RecruitingNot Applicable
Optimapharm
Posted 11/29/2019

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