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Clinical Trial News

Regor Therapeutics to Present Phase 2a Data for Oral GLP-1 Agonist RGT-075 at ADA 2025

  • Regor Therapeutics will present Phase 2a trial results for RGT-075, an oral small molecule GLP-1 receptor agonist, at the American Diabetes Association's 85th Scientific Sessions in Chicago.
  • RGT-075 demonstrated preliminary weight reduction in Phase 1 studies with no new safety signals observed in healthy volunteers and diabetes patients.
  • The company is currently conducting the COMO-1 Phase 2b trial with approximately 240 participants, evaluating multiple doses over 36 weeks with topline data expected by end of 2025.
  • RGT-075 represents a potential breakthrough as an orally bioavailable, once-daily treatment for obesity and overweight patients with weight-related comorbidities.

Food Deserts Compound Clinical Trial Access Barriers for Breast Cancer Patients

  • A study of over 1.3 million breast cancer patients found that living in food deserts significantly reduces clinical trial participation rates.
  • Patients facing both food deserts and clinical trial deserts showed a 27% lower likelihood of trial enrollment compared to 19% for food desert residents alone.
  • The research demonstrates a compounding negative effect when patients live in areas with both limited healthy food access and distant trial sites.
  • Interventions such as establishing satellite facilities, telehealth options, and patient navigation programs could help address these systemic barriers.

FDA Delays KalVista's Sebetralstat Approval Decision Due to Resource Constraints

Drug Approval
  • The FDA has extended its review of KalVista's sebetralstat for hereditary angioedema by four weeks past the June 17 deadline due to heavy workload and limited resources.
  • Sebetralstat would become the first on-demand oral treatment for hereditary angioedema if approved, offering a convenient alternative to injectable therapies.
  • The delay represents the first instance directly attributed to FDA resource constraints following agency restructuring under the Trump administration.
  • KalVista maintains confidence in near-term approval as the FDA has not requested additional data or raised safety concerns.

Intravenous Rehydration Shows Safety in Severely Malnourished Children, Challenging WHO Guidelines

  • A randomized controlled trial involving 292 children across four African countries found that intravenous rehydration was as safe as oral rehydration for children with severe acute malnutrition and gastroenteritis.
  • The study revealed no cases of heart failure or fluid overload with IV treatment, contradicting long-held concerns about cardiac complications in malnourished children.
  • Children receiving standard oral rehydration experienced more complications, including increased need for nasogastric tubes, vomiting episodes, and emergency IV fluids for shock.
  • The findings could prompt a review of WHO guidelines that currently distinguish between rehydration strategies for malnourished and non-malnourished children.

FDA Delays Sebetralstat Approval Decision for Hereditary Angioedema Treatment Due to Resource Constraints

Drug ApprovalRare Disease
  • The FDA will not meet the June 17, 2025 PDUFA goal date for KalVista's sebetralstat NDA due to heavy workload and limited resources, with a decision expected within four weeks.
  • Sebetralstat represents a potential breakthrough as the first oral on-demand treatment for hereditary angioedema attacks in patients aged 12 and older.
  • The FDA has not requested additional data or raised safety concerns, with only labeling finalization remaining under review according to KalVista.
  • The delay affects patients with hereditary angioedema who currently rely on intravenous or subcutaneous treatments for managing painful and potentially life-threatening attacks.

Olutasidenib Maintenance Therapy Demonstrates Sustained Remission in IDH1-Mutated AML Patients

  • Olutasidenib maintenance therapy achieved an 83% relapse-free survival rate at 4 months and 71% at 12 months in IDH1-mutated AML patients who achieved complete remission following induction therapy.
  • The phase 2 trial enrolled 18 patients with a median treatment duration of 19.6 months, demonstrating clinically meaningful activity as a switch maintenance strategy for patients with persistent minimal residual disease.
  • Overall survival rates remained high at 89% for both 12 and 24 months, with a manageable safety profile and no treatment discontinuations due to adverse events.
  • Two patients with prior venetoclax exposure converted from incomplete remission to complete remission during olutasidenib treatment, supporting its therapeutic potential in this patient population.

Study of AG-120 (Ivosidenib) vs. Placebo in Combination With Azacitidine in Participants With Previously Untreated Acute Myeloid Leukemia With an IDH1 Mutation

NCT03173248Active, Not RecruitingPhase 3
Institut de Recherches Internationales Servier
Posted 6/26/2017

Open-label Study of FT-2102 With or Without Azacitidine or Cytarabine in Patients With AML or MDS With an IDH1 Mutation

NCT02719574CompletedPhase 1
Forma Therapeutics, Inc.
Posted 4/30/2016

Thermo Fisher Scientific Explores $4 Billion Diagnostics Unit Sale to Streamline Portfolio

  • Thermo Fisher Scientific is reportedly planning to divest portions of its diagnostics business valued at approximately $4 billion to offload lower-growth assets.
  • The company has enlisted advisers to explore private equity interest in parts of its diagnostics unit, including the microbiology division that produces infectious disease testing equipment.
  • This strategic move comes amid healthcare sector volatility and follows the company's recent $4.1 billion acquisition of Solventum's purification and filtration business.
  • The auction process does not guarantee a sale, as Thermo Fisher may ultimately decide to retain these assets depending on market conditions and strategic considerations.

NeuroEM Therapeutics Secures $1.33 Million in Series A Funding to Advance Drug-Free Alzheimer's Treatment

  • NeuroEM Therapeutics has closed initial rounds totaling $1.33 million of a $5 million Series A equity raise led by BlueLake.vc to commercialize its Transcranial Electromagnetic Treatment (TEMT-RF) technology.
  • The company's innovative approach uses radio frequencies delivered through a lightweight cap worn twice daily to target cognitive decline and restore brain function in Alzheimer's patients.
  • NeuroEM received the first FDA Breakthrough Device status for treating Alzheimer's disease and has demonstrated safety and cognitive enhancement in early clinical studies.
  • The funding will support continued progress toward commercialization of the drug-free treatment, which aims to prevent and treat cognitive decline caused by aging and neurodegenerative diseases.

Ponatinib Shows Superior Survival Outcomes in Ph+ ALL Patients with Persistent MRD After Induction

Drug Approval
  • Post hoc analysis of the PhALLCON trial demonstrates that ponatinib achieved higher rates of deep molecular responses compared to imatinib in newly diagnosed Ph+ ALL patients who remained MRD-positive after induction therapy.
  • Patients with persistent MRD who continued ponatinib beyond cycle 3 showed significantly improved event-free survival with a median not reached versus 24.8 months for imatinib (HR 0.198, P = 0.0004).
  • The analysis supports continuing ponatinib treatment in Ph+ ALL patients who fail to achieve MRD negativity after initial induction, though researchers caution about the small patient numbers and potential selection bias.

A Study of Ponatinib Versus Imatinib in Adults With Acute Lymphoblastic Leukemia

NCT03589326Active, Not RecruitingPhase 3
Takeda
Posted 10/4/2018

Nectero Medical Secures CPT Codes for Investigational AAA Treatment as Phase 2/3 Trial Shows Promise

  • Nectero Medical received two Category III CPT codes from the American Medical Association for its investigational EAST system, facilitating future billing and reimbursement for treating small- to medium-sized abdominal aortic aneurysms.
  • The EAST system delivers pentagalloylglucose directly into aneurysmal walls via a dual-balloon catheter, designed as a one-time endovascular treatment to slow or stabilize AAA growth.
  • One-year follow-up data from the stAAAble trial showed 91% of patients experienced aneurysm growth rates below expected levels compared to historical controls.
  • The approval represents a significant milestone for addressing an unmet medical need, as no proven therapeutic interventions currently exist for small- to medium-sized AAAs.

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