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Clinical Trial News

Real-World Data Show Less Robust Weight Loss with GLP-1 Drugs Compared to Clinical Trials

• Real-world studies indicate that weight loss from GLP-1 agonists is less pronounced than reported in clinical trials, with patients achieving only a 3.7% average weight loss after one year. • Patient persistence with GLP-1s is a significant challenge, as discontinuation rates can reach as high as 85% after two years of treatment, impacting long-term health outcomes. • Factors such as medication supply shortages, adverse effects, high costs, and unmet expectations contribute to non-persistence in real-world GLP-1 usage. • To achieve the health benefits of GLP-1s, sustained use is crucial to realize clinical success and avoid potential weight regain after discontinuing the medication.

Evolving Landscape of MASLD: New Insights and Therapeutic Approaches

• Metabolic dysfunction-associated steatotic liver disease (MASLD) is now the preferred term for non-alcoholic fatty liver disease (NAFLD), reflecting a shift towards metabolic dysfunction as the primary driver. • Research is focusing on the pathogenesis of MASLD, including the roles of genetics, senescent cells, and the intersection with alcohol-related liver disease. • Emerging therapies, such as PNPLA3 siRNA, resmetirom, and tirzepatide, are showing promise in treating MASLD and associated liver fibrosis. • Non-invasive diagnostic tools like vibration-controlled transient elastography are increasingly important for monitoring disease progression and predicting liver-related events.

Exscientia Highlights AI and Robotics Integration in Streamlined Drug Discovery

• Exscientia detailed its automated drug discovery workflow, integrating robotics and AI to enhance efficiency and flexibility in assay testing. • Early data indicates AI-designed molecules have an 80% Phase I success rate, significantly higher than the industry average of 15%. • The company's robotic lab in Oxfordshire uses AI to design molecules with reduced human involvement, increasing overall productivity. • AI-led drug pipelines are attracting higher investment, exemplified by Bristol-Myers Squibb's $1.2bn AI drug discovery deal with Exscientia.

Dr. Reddy's and Hetero Partner with Gilead to Produce Generic HIV Drug Lenacapavir

• Dr. Reddy's and Hetero have signed non-exclusive licensing agreements with Gilead to manufacture and market generic lenacapavir in 120 countries. • Lenacapavir, marketed as Sunlenca, is approved for multi-drug resistant HIV-1 and is under investigation for HIV prevention (PrEP). • The agreement allows Dr. Reddy's to handle technology transfer, clinical studies, and product launches in the specified markets. • This collaboration aims to provide affordable access to lenacapavir in low- and middle-income countries with high HIV prevalence.

FDA Authorizes Phase 2a Trial of Progerinin for Hutchinson-Gilford Progeria Syndrome

  • The FDA has authorized a Phase 2a clinical trial for Progerinin, a novel drug developed by PRG Science & Technology for Progeria treatment.
  • The trial will evaluate Progerinin in combination with Zokinvy, the current standard of care, in 10 patients with Progeria and Progeroid Laminopathies.
  • Progerinin has shown promise in preclinical studies, potentially increasing lifespan in a Progeria mouse model by 50%.
  • This trial represents a collaborative effort between PRG S&T, The Progeria Research Foundation, Boston Children’s Hospital, and Amarex CRO.

Ionis Pharmaceuticals' ION582 Receives FDA Fast Track Designation for Angelman Syndrome Treatment

• ION582, developed by Ionis Pharmaceuticals, has been granted Fast Track designation by the FDA for treating Angelman syndrome. • This designation aims to expedite the development and review of ION582, addressing an unmet need for Angelman syndrome patients. • ION582 is designed to target and reduce the expression of the UBE3A-AS transcript in individuals with Angelman syndrome. • The FDA's decision underscores the potential of ION582 to significantly improve outcomes for those affected by this rare neurological disorder.

Rapamycin Counteracts Valproate's Harmful Effects on Fetal Spinal Cord Development

  • Researchers found that Sodium Valproate, an epilepsy drug, can cause spinal malformations in fetuses during pregnancy.
  • Using human mini-spinal cords (organoids), they discovered that Valproate changes the cells that form the spinal cord, leading to malformations.
  • Co-treatment with Rapamycin prevented the negative effects of Valproate on the organoids, potentially allowing safer use of the epilepsy drug during pregnancy.

Onyda XR, First Liquid Non-Stimulant for ADHD, Now Available in the US

  • Tris Pharma's Onyda XR (clonidine hydrochloride), the first liquid, non-stimulant medication for ADHD, is now available in the U.S. for pediatric patients aged 6 and older.
  • Approved by the FDA in May 2024, Onyda XR can be used as a monotherapy or as an adjunctive therapy with CNS stimulants for ADHD treatment.
  • Clinical trials supporting approval included placebo-controlled studies demonstrating efficacy and safety in pediatric patients, with common adverse reactions including somnolence and fatigue.
  • Onyda XR offers a new treatment option for ADHD patients who struggle with pills or need longer-acting therapy, addressing a significant unmet need in ADHD management.

Neoantigen Vaccine Shows Promise in Early-Stage Lymphoplasmacytic Lymphoma

  • A first-in-human neoantigen DNA vaccine for lymphoplasmacytic lymphoma (LPL) demonstrated stable disease or better in patients with untreated asymptomatic LPL.
  • The vaccine was well-tolerated, with no dose-limiting toxicities, suggesting its potential as an early intervention strategy.
  • The trial showed a reduction in clonal tumor subpopulations and altered gene-expression pathways in the mature B-cell compartment following vaccination.
  • Early intervention with the vaccine nearly doubled the disease-free progression time to an average of just under seven years.

Vaccine Therapy in Treating Patients With Lymphoplasmacytic Lymphoma

NCT01209871Active, Not RecruitingPhase 1
M.D. Anderson Cancer Center
Posted 2/26/2015

Triveni Bio Secures $115 Million in Series B Funding to Advance Atopic Dermatitis Drug

  • Triveni Bio raised $115 million in Series B funding, led by Goldman Sachs Alternatives, to advance its pipeline of antibody drugs for inflammatory diseases.
  • The company's lead candidate, TRIV-509, is being developed for atopic dermatitis and targets kallikreins 5 and 7, enzymes overexpressed in most atopic dermatitis patients.
  • Triveni aims to initiate its first clinical trial for TRIV-509 in early 2025, with preclinical data suggesting it may be more potent than IL-4R targeting drugs like Dupixent.
  • The funding will support Triveni's operations through 2027 and the development of additional candidates, including a bispecific antibody and a treatment for hereditary pancreatitis.

Evaluation of NM26-2198 in Healthy Subjects and in Patients With Moderate-to-severe Atopic Dermatitis (AD)

NCT05859724TerminatedPhase 1
Yellow Jersey Therapeutics AG
Posted 5/10/2023

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