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Clinical Trial News

FNIH Study Validates Blood Tests for Alzheimer's Detection, Potentially Replacing Invasive Spinal Taps

  • A new FNIH Biomarkers Consortium study found that some commercial blood tests are accurate enough for clinical use and could replace spinal taps and brain scans in many patients with cognitive impairment.
  • The study confirmed that certain blood tests have accuracy similar to established cerebrospinal fluid tests in detecting amyloid plaques, with p-tau217 protein levels being the most accurate biomarker.
  • Six commercial test developers were evaluated using blood samples from the Alzheimer's Disease Neuroimaging Initiative, with results showing some tests are highly accurate while others perform poorly.
  • The validated blood tests could accelerate drug development by helping researchers select optimal participants for clinical trials and enable earlier detection of Alzheimer's brain changes.

FDA Approves Erzofri (Paliperidone Palmitate) for Schizophrenia and Schizoaffective Disorder

  • The FDA has approved Erzofri (paliperidone palmitate) as a monthly injectable for treating schizophrenia and schizoaffective disorder in adults.
  • Erzofri is the first paliperidone palmitate long-acting injection developed in China to receive U.S. approval, offering a new treatment option.
  • Approval was based on a study demonstrating bioequivalence to Invega Sustenna, with a comparable safety profile and optimized initial dosing.
  • Paliperidone palmitate long-acting injections had sales of $2.897 billion in the U.S. in 2023, highlighting the market for this treatment.

Relative Bioavailability of LY03010 Compared to Listed Drug

NCT04922593CompletedPhase 1
Luye Pharma Group Ltd.
Posted 1/13/2021

Alector Presents Baseline Data from INVOKE-2 Phase 2 Trial of AL002 for Early Alzheimer's Disease

• Alector presented baseline characteristics from the INVOKE-2 Phase 2 trial of AL002, a novel TREM2 agonist, at the Alzheimer's Association International Conference (AAIC) 2024. • The INVOKE-2 trial enrolled 381 participants with early Alzheimer's disease, showing a median age of 71 years and confirming amyloid positivity in all participants. • Treatment-emergent brain MRI changes (ARIA) were observed, with higher incidence and severity in APOE e4 homozygous carriers, leading to their early discontinuation from the trial. • Results from the INVOKE-2 trial are expected in the fourth quarter of 2024, with a long-term extension study ongoing to assess the long-term effects of AL002.

A Phase 2 Study to Evaluate Efficacy and Safety of AL002 in Participants With Early Alzheimer's Disease

NCT04592874Active, Not RecruitingPhase 2
Alector Inc.
Posted 1/22/2021

FDA Expands Palforzia Approval to Include Children Aged 1-3 with Peanut Allergy

  • The FDA has expanded the approval of Palforzia to include children aged 1 through 3 years with a confirmed peanut allergy.
  • Palforzia is designed to mitigate allergic reactions, including anaphylaxis, that may occur due to accidental peanut exposure.
  • The drug has been approved since January 2020 for individuals aged 4 through 17 years with a confirmed peanut allergy.
  • Patients taking Palforzia must continue to avoid peanuts in their diets, and a Risk Evaluation and Mitigation Strategy (REMS) is in place.

FDA Approves Leqselvi (Deuruxolitinib) for Severe Alopecia Areata

  • The FDA has approved Leqselvi (deuruxolitinib) 8 mg tablets for treating adults with severe alopecia areata, offering a new oral option.
  • Clinical trials (THRIVE-AA1 and THRIVE-AA2) showed that over 30% of patients achieved 80% or more scalp hair coverage after 24 weeks.
  • Leqselvi, a JAK1 and JAK2 inhibitor, works by interrupting immune pathways that contribute to hair loss in alopecia areata.
  • Common adverse reactions in trials included headache, acne, and nasopharyngitis, with a small percentage discontinuing due to side effects.

Study to Evaluate the Efficacy and Safety of CTP-543 in Adults With Moderate to Severe Alopecia Areata (THRIVE-AA1)

NCT04518995CompletedPhase 3
Concert Pharmaceuticals
Posted 11/23/2020

Study to Evaluate the Efficacy and Safety of CTP-543 in Adults With Moderate to Severe Alopecia Areata (THRIVE-AA2)

NCT04797650CompletedPhase 3
Concert Pharmaceuticals
Posted 6/10/2021

FDA Issues Complete Response Letter for Xspray Pharma's Dasynoc in CML and ALL

  • The FDA issued a Complete Response Letter (CRL) for Dasynoc, Xspray Pharma's treatment for chronic myeloid leukemia (CML) and acute lymphocytic leukemia (ALL).
  • The FDA requests additional labeling information and pre-approval inspection details, but does not question stability or clinical data.
  • Dasynoc leverages Xspray’s HyNap technology, offering potential benefits such as lower dosing and co-administration with acid-reducing agents.
  • Xspray Pharma is working to address the FDA's requests and anticipates providing an updated timeline for NDA resubmission soon.

CBM588 Shows Promise in Improving Outcomes for Metastatic Renal Cell Carcinoma

  • CBM588, a biotherapeutic, demonstrates potential in correcting gut dysbiosis and preventing depletion of ICI response-associated species in mRCC patients.
  • A pooled analysis of phase 1 trials showed that CBM588, combined with standard-of-care, may enhance clinical outcomes in metastatic renal cell carcinoma.
  • Researchers are optimistic about elucidating CBM588's mechanism of action and are planning a phase 3 trial to further evaluate its clinical activity.
  • Experts encourage mRCC patients to consider interventions like lifestyle changes and biotherapeutics to potentially improve treatment outcomes.

CBM588 in Combination With Nivolumab and Cabozantinib for the Treatment of Advanced or Metastatic Kidney Cancer

NCT05122546Active, Not RecruitingPhase 1
City of Hope Medical Center
Posted 11/1/2021

CBM588, Nivolumab, and Ipilimumab in Treating Patients With Stage IV or Advanced Kidney Cancer

NCT03829111CompletedPhase 1
City of Hope Medical Center
Posted 5/14/2019

Nicox Completes U.S. Recruitment for Phase 3 Glaucoma Trial of NCX 470

  • Nicox has completed recruitment and randomization of the final patient in the United States for its Denali Phase 3 trial evaluating NCX 470, a novel NO-donating bimatoprost eye drop for open-angle glaucoma and ocular hypertension.
  • The trial compares NCX 470 ophthalmic solution 0.1% to latanoprost 0.005% across the U.S. and China, with more than 95% of the targeted patient number already randomized.
  • Topline results are expected in the second half of 2025, with the U.S. New Drug Application submission anticipated in the first half of 2026.
  • Previous Mont Blanc Phase 3 trial results showed NCX 470 achieved statistical non-inferiority versus latanoprost and demonstrated superiority at 4 out of 6 timepoints, meeting U.S. efficacy requirements for approval.

Third Arc Secures $165M Series A to Advance Novel T-Cell Engager Platform for Cancer and Inflammation

  • Third Arc Bio, led by former Johnson & Johnson executives, has raised $165 million in Series A funding to develop multifunctional antibodies for T-cell engagement in solid tumors and inflammatory conditions.
  • The company plans an ambitious clinical development program, aiming to advance five therapeutic candidates into clinical trials next year, leveraging their specialized antibody platform technology.
  • The funding round was led by Vida Ventures and co-led by Cormorant Asset Management and Hillhouse Investment, with support from founding investor Omega Funds.

NIH Identifies AKT2 as Potential Therapeutic Target for Dry Age-Related Macular Degeneration

  • NIH researchers have pinpointed AKT2's role in lysosome dysfunction, leading to drusen formation, a key indicator of dry age-related macular degeneration (AMD).
  • The study demonstrated that manipulating AKT2 expression in retinal pigment epithelium (RPE) cells of mice induced dry AMD symptoms, including RPE degeneration.
  • Analysis of human RPE cells revealed that a genetic variant (CFH Y402H) associated with increased AMD risk also correlated with elevated AKT2 expression and defective lysosomes.
  • These findings suggest that targeting the AKT2/SIRT5/TFEB pathway could offer a novel therapeutic strategy for dry AMD, a condition currently lacking effective treatments.

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