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Clinical Trial News

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

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

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

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

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

Truqap Plus Abiraterone Shows Promise in PTEN-Deficient Metastatic Prostate Cancer

  • AstraZeneca's Truqap, combined with abiraterone and ADT, significantly improved radiographic progression-free survival in PTEN-deficient metastatic hormone-sensitive prostate cancer.
  • The Phase III CAPItello-281 trial demonstrated a clinically meaningful benefit compared to abiraterone and ADT with placebo in this specific patient population.
  • An early trend towards improved overall survival was observed, though data is still immature, with the trial continuing to assess this key secondary endpoint.
  • This combination marks the first AKT inhibitor to show benefit in this aggressive prostate cancer subtype, addressing a critical unmet need.

Scemblix Demonstrates Superior Efficacy Over TKIs in First-Line CML Treatment: 96-Week ASC4FIRST Trial Results

  • Scemblix (asciminib) shows significantly higher major molecular response (MMR) rate compared to investigator-selected tyrosine kinase inhibitors (TKIs) at 96 weeks in newly diagnosed chronic myeloid leukemia (CML) patients.
  • The ASC4FIRST trial demonstrates a favorable safety profile for Scemblix, with fewer discontinuations due to adverse events compared to TKIs.
  • These findings support Scemblix as a potential first-line treatment option for CML, offering improved efficacy and tolerability.
  • The 96-week data reinforce the potential of Scemblix to change the treatment paradigm for CML patients requiring first-line therapy.

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