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Clinical Trial News

Ractigen's RAG-21 Receives FDA Orphan Drug Designation for FUS-ALS Treatment

  • Ractigen Therapeutics' RAG-21, a novel siRNA therapy targeting the FUS gene, has received FDA Orphan Drug Designation for amyotrophic lateral sclerosis (ALS).
  • RAG-21 leverages RNA interference to reduce FUS protein levels, addressing motor neuron degeneration in FUS-ALS, a particularly aggressive ALS subtype.
  • Preclinical studies demonstrate RAG-21's efficacy and safety in mitigating FUS cytoplasmic mis-localization and aggregation, offering hope for improved outcomes.
  • The FDA's designation provides incentives, including tax credits and marketing exclusivity, to accelerate RAG-21's development for this rare and severe disease.

Tislelizumab Gains Category 1 Recommendation in NCCN Guidelines for Hepatocellular Carcinoma

• Tislelizumab-jsgr (Tevimbra) has been added to the NCCN guidelines as a category 1 recommended regimen for first-line systemic therapy in HCC. • The NCCN guidelines are updated regularly, and healthcare professionals should ensure they are using the latest version to inform their practice. • Identifying biomarkers to predict response to checkpoint inhibitors remains a key challenge in HCC treatment, as many patients do not benefit from current therapies.

FDA Approves Attruby (Acoramidis) for Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

• The FDA has approved Attruby (acoramidis) for treating transthyretin amyloid cardiomyopathy (ATTR-CM), offering a new hope for patients with this rare heart condition. • Attruby, a TTR stabilizer, demonstrated significant reduction in cardiovascular death and hospitalization in the ATTRibute-CM Phase 3 trial. • The approval provides an additional treatment option alongside existing therapies, potentially improving heart health and quality of life for ATTR-CM patients. • BridgeBio offers patient support programs like ForgingBridges™ to assist with access, insurance, and financial aid for Attruby treatment.

Merck's Winrevair Shows Promise in Pulmonary Hypertension, While Biohaven and Cassava Face Setbacks

  • Merck's Winrevair significantly reduced the risk of death in patients with pulmonary arterial hypertension, marking a potential breakthrough in treating this rare lung condition.
  • Biohaven's spinal muscular atrophy treatment failed to meet primary endpoints in a pivotal study, leading to a notable decline in the company's stock value.
  • Cassava Sciences reported that its Alzheimer's drug did not achieve primary targets, representing another setback in the company's research efforts.
  • Shortages of obesity drugs like Wegovy persist, with over 200,000 prescriptions for copies being filled monthly, prompting FDA consideration of the drug's shortage status.

Merck's Winrevair Shows Positive Topline Results in Phase 3 ZENITH Study for High-Risk PAH Patients

  • Merck's WINREVAIR (sotatercept-csrk) met its primary endpoint in the Phase 3 ZENITH study, demonstrating a statistically significant reduction in morbidity or mortality events in high-risk PAH patients.
  • The ZENITH study evaluated WINREVAIR in adults with pulmonary arterial hypertension (PAH) functional class III or IV at high risk of mortality.
  • An independent data monitoring committee recommended stopping the ZENITH study early due to the strength of the results, offering all participants WINREVAIR in an open-label extension study.
  • WINREVAIR is already approved in the U.S. and 36 countries based on the Phase 3 STELLAR trial, and has been submitted for approval in Japan.

AI-Discovered Drug INS018_055 Enters Phase 2 Trials for Idiopathic Pulmonary Fibrosis

• INS018_055, an AI-discovered drug developed by Insilico Medicine, has advanced to Phase 2 clinical trials for Idiopathic Pulmonary Fibrosis (IPF). • The drug targets TNIK, an antifibrotic protein identified using Insilico's PandaOmics and Chemistry42 AI platforms, demonstrating safety and tolerability in Phase 1 trials. • Phase 2a trials are underway in both China and the US, each enrolling 60 patients, to evaluate the drug's effectiveness and monitor short-term side effects. • IPF affects 13 to 20 individuals per 100,000, imposing significant direct medical costs, highlighting the urgent need for effective treatments.

iiCON-Supported Phase II Trial Aims to Combat Pneumococcal Disease with Novel Vaccine

  • A new £3.2 million Medical Research Council-funded trial, managed by iiCON, will advance the development of a novel vaccine against pneumococcal disease, addressing antibiotic resistance and global health concerns.
  • The phase II study will assess ImmBio's PnuBioVax, a protein-based vaccine, in a controlled human infection model (CHIM) trial in Malawi, comparing its efficacy against PCV13 and a placebo in preventing SPN3 carriage.
  • The trial seeks to prevent transmission of serotype 3 pneumococcus (SPN3), which has become a dominant strain with high antimicrobial resistance, particularly impacting children and immunocompromised individuals in low- and middle-income countries.
  • The low-cost vaccine, if successful, may be manufactured and distributed in Africa, potentially boosting the local economy and ensuring access to communities in need.

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.

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

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

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

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

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