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Clinical Trial News

Aelis Farma's AEF0117 Fails Phase 2B Trial for Cannabis Use Disorder

• Aelis Farma's AEF0117 did not meet its primary endpoint of reducing cannabis use to one day or less per week in patients with moderate to severe cannabis use disorder (CUD). • The Phase 2B trial also failed to meet secondary endpoints, including complete cessation of marijuana use or reduction to two days or less per week. • Indivior, which had an option to license AEF0117, announced it does not currently expect to exercise this option due to the lack of separation from placebo in the trial. • AEF0117 was well-tolerated, with adverse events similar across treatment groups, including placebo, indicating no significant safety concerns.

NIAID's NextGen COVID-19 Vaccine Program Advances with Phase 1 Trial

  • Leidos Biomedical is seeking a CDMO for fill & finish services for an mRNA-LNP COVID-19 vaccine candidate.
  • The vaccine, developed under NIAID's NextGen program, will be used in a Phase 1 clinical trial in the US.
  • The fill & finish services are expected to begin in November-December 2024, using ready-to-fill bulk drug product.
  • This initiative supports regulatory filing and evaluation of the vaccine candidate, aiming to enhance COVID-19 protection.

Moderna's Updated COVID-19 Vaccine Targeting JN.1 Receives Positive CHMP Opinion

  • Moderna's updated Spikevax vaccine, targeting the SARS-CoV-2 variant JN.1, has received a positive recommendation from the EMA's CHMP.
  • The vaccine is intended for active immunization against COVID-19 in individuals aged six months and older, aligning with EMA guidance.
  • Approvals for the JN.1-targeting vaccine have already been secured in Japan, Taiwan, and the UK, with pending authorization from the European Commission.
  • Moderna is participating in the EU's HERA tendering process for mRNA COVID-19 vaccines, potentially expanding its reach across member states.

ProKidney Prioritizes US Market for Rilparencel, Discontinuing Ex-US Phase III CKD Trial

  • ProKidney will discontinue its ex-US Phase III trial (REGEN-016) of rilparencel for chronic kidney disease (CKD) to focus on the US market.
  • The strategic shift aims to expedite potential US registration and commercial launch of rilparencel, saving the company $150M-$175M.
  • Rilparencel, a regenerative cell therapy, has RMAT designation from the FDA and targets patients with type 2 diabetes and CKD.
  • Positive interim data from the REGEN-007 Phase 2 trial supports rilparencel's potential to preserve kidney function in CKD patients.

A Study of Renal Autologous Cell Therapy (REACT) in Participants With Type 2 Diabetes and Chronic Kidney Disease

NCT05286853RecruitingPhase 3
Prokidney
Posted 7/17/2024

Study of Renal Autologous Cell Therapy (REACT) in Subjects With Type 1 or 2 Diabetes and Chronic Kidney Disease (REGEN-007)

NCT05018416CompletedPhase 2
Prokidney
Posted 7/27/2021

Proact: A Study of REACT in Subjects With Type 2 Diabetes Mellitus and Chronic Kidney Disease

NCT05099770RecruitingPhase 3
Prokidney
Posted 1/5/2022

CBD Reduces Daytime Fatigue in Hypertensive Patients: A Randomized Trial

  • A recent study published in Cannabis and Cannabinoid Research indicates that CBD administration significantly reduces daytime fatigue in patients with primary hypertension.
  • The randomized, placebo-controlled trial assessed the impact of CBD on participants' quality of life over a five-week period, showing improvements in ESS scores.
  • Researchers observed that CBD intake led to enhanced fatigue/vitality and psychological well-being, though overall sleep quality improvements were not reported.
  • The findings suggest CBD may offer a therapeutic avenue for managing daytime sleepiness associated with hypertension, warranting further investigation.

Pantheon Vision Pioneers Bioengineered Solutions to Combat Global Corneal Blindness

  • Pantheon Vision, led by industry veteran John Sheets, PhD, is developing innovative bioengineered alternatives to traditional corneal transplants to address global corneal blindness.
  • The company aims to reduce dependence on donated corneal tissues while improving graft acceptance, vision restoration, and long-term outcomes for patients.
  • Corneal blindness presents a significant public health challenge, particularly affecting low- and middle-income countries where access to eye care remains limited.

Mindstate's AI-Designed Psychedelic MSD-001 Receives FDA and EMA Nod for Human Trials

  • Mindstate Design Labs has received FDA and EMA approval to begin Phase 1 human trials for its AI-designed psychedelic compound, MSD-001.
  • MSD-001 is designed to selectively target serotonin receptors, offering a more controlled and predictable psychoactive effect compared to traditional psychedelics.
  • The Phase 1 trials will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of MSD-001 in healthy participants.
  • Mindstate's Osmanthus AI platform synthesizes extensive drug reports and biochemical data to design targeted states of consciousness, potentially expanding therapeutic applications.

Acadia Pharmaceuticals' Phase 3 Trial Investigates Carbetocin Nasal Spray for Hyperphagia in Prader-Willi Syndrome

  • Acadia Pharmaceuticals is enrolling patients in a Phase 3 clinical trial (COMPASS-PWS) to evaluate carbetocin nasal spray for hyperphagia in Prader-Willi Syndrome (PWS).
  • The trial includes 170 participants aged 5 to 30 with PWS, comparing carbetocin to placebo over 12 weeks in a double-blind study.
  • Carbetocin, a synthetic analogue of oxytocin, may reduce hyperphagia by binding to oxytocin receptors, potentially addressing oxytocin deficiency in PWS.
  • Participants completing the 12-week study can enroll in an open-label extension receiving carbetocin nasal spray for up to 3 years.

SOPHiA GENETICS' ecDNA Assay Validated for Boundless Bio's Clinical Trial

• SOPHiA GENETICS and Boundless Bio collaborate with Precision for Medicine to validate a novel clinical trial assay for detecting extrachromosomal DNA (ecDNA). • The ecDNA Solution (ECS) algorithm identifies patients with ecDNA-amplified tumors for Boundless Bio's POTENTIATE Phase 1/2 study of BBI-355. • Precision for Medicine adapts its diagnostic workflow to integrate with the SOPHiA DDM™ Platform, enhancing patient selection for clinical trials. • The collaboration aims to expedite clinical trial enrollment and support the development of new ecDNA-directed therapies for cancer patients.

BTK Inhibitors Show Promise and Face Challenges in Multiple Sclerosis Trials

  • Sanofi's tolebrutinib showed mixed results in Phase III trials for relapsing MS, missing primary endpoints in two studies but delaying disability progression in a third.
  • Roche's fenebrutinib demonstrated near-total elimination of disease activity in a Phase II study for relapsing MS, with 96% of patients relapse-free after one year.
  • Both Sanofi's tolebrutinib and Roche's fenebrutinib have faced partial clinical holds due to concerns over liver safety, highlighting a key challenge for oral BTK inhibitors.
  • Despite setbacks, oral BTK inhibitors are viewed as promising for MS treatment, with neurologists eagerly awaiting their arrival and analysts predicting significant future sales.

A Study to Evaluate the Efficacy and Safety of Fenebrutinib Compared With Ocrelizumab in Adult Participants With Primary Progressive Multiple Sclerosis

NCT04544449Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 10/26/2020

Study to Evaluate the Efficacy and Safety of Fenebrutinib Compared With Teriflunomide in Relapsing Multiple Sclerosis (RMS)

NCT04586023Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 3/24/2021

A Study to Evaluate the Efficacy and Safety of Fenebrutinib Compared With Teriflunomide in Relapsing Multiple Sclerosis (RMS)

NCT04586010Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 3/17/2021

A Study to Investigate the Efficacy of Fenebrutinib in Relapsing Multiple Sclerosis (RMS)

NCT05119569Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 3/1/2022

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