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Clinical Trial News

CD47-Targeted Immunotherapies Show Promise in Cancer Treatment

• CD47-targeted therapies are emerging as a novel cancer immunotherapy approach, inhibiting the "don't eat me" signal that cancer cells use to evade immune surveillance. • Several CD47 inhibitors, like magrolimab and evorpacept, are undergoing clinical trials, showing encouraging results in hematological and solid tumors, often in combination with other treatments. • Regulatory bodies are supportive, granting fast track designations and IND clearances, indicating a rapidly expanding CD47 market driven by the rising incidence of cancer. • Pharmaceutical companies and research institutions are actively involved, with the United States leading the sector, while countries like China are increasing their engagement in clinical studies.

KalVista's Sebetralstat Shows Consistent Efficacy for Hereditary Angioedema

  • KalVista Pharmaceuticals presented data at the Bradykinin Symposium 2024, highlighting sebetralstat's consistent efficacy and safety in treating hereditary angioedema (HAE) attacks.
  • Clinical trials showed sebetralstat provided rapid symptom relief, with a median time to relief of 1.8 hours for all attacks and 1.3 hours for laryngeal attacks.
  • The oral administration of sebetralstat may offer advantages over current injectable treatments, potentially improving patient compliance and earlier treatment initiation.
  • Sebetralstat demonstrated a safety profile comparable to placebo, reinforcing its potential as a transformative on-demand treatment for HAE.

Multiple Myeloma Survivor and Physician Advocate Drives Progress in Clinical Trials

  • Dr. Jim Omel, a multiple myeloma survivor, has leveraged his experience as a physician and patient to advocate for others and contribute to clinical trial design.
  • Omel's insights were invaluable to the DETERMINATION study, which explored stem cell transplants for newly diagnosed multiple myeloma patients and included a significant number of African American participants.
  • The DETERMINATION study revealed potential differences in treatment response between African American and white patients, highlighting the need for tailored approaches.
  • Omel's advocacy extends to raising awareness about early detection and genetic abnormalities associated with multiple myeloma, emphasizing the importance of tests like SPE.

Blood Biomarkers Show Promise in Predicting Suicidality and Guiding Personalized Treatment

  • Researchers have identified blood biomarkers that can predict suicidal ideation and future hospitalization for suicidality, offering a new tool for risk assessment.
  • The study used a multi-cohort approach, including discovery, validation, and testing phases, to ensure the robustness of the identified biomarkers.
  • Pathway analysis revealed that these biomarkers are involved in key biological processes related to psychiatric disorders, providing insights into the underlying mechanisms of suicidality.
  • The findings support the development of personalized treatment strategies based on an individual's biomarker profile, potentially improving outcomes for those at risk.

Neutrophil Dysfunction Linked to Psoriatic Arthritis Severity

  • Researchers found that neutrophils from PsA patients show reduced activation, ROS production, and phagocytic ability upon TNF stimulation.
  • PsA neutrophils release fewer granular enzymes and NET biomarkers compared to healthy controls, indicating impaired function.
  • Serum levels of neutrophil-related mediators and NET biomarkers are elevated in PsA patients, suggesting a distinct neutrophil signature.
  • CitH3 shows potential as a diagnostic biomarker, accurately distinguishing PsA patients from healthy individuals with high specificity and sensitivity.

Strides Pharma Receives USFDA Approval for Generic Theophylline Extended-Release Tablets

  • Strides Pharma's subsidiary secures USFDA approval for generic Theophylline extended-release tablets, used for chronic asthma and lung diseases.
  • The approved drug, a generic version of Theo-Dur, addresses symptoms like wheezing and shortness of breath by relaxing bronchial muscles.
  • Theophylline extended-release tablets, with a market size of $11.5 million, will be manufactured in Bangalore, enhancing Strides Pharma's portfolio.
  • Strides Pharma's stock price surged following the announcement, reflecting investor confidence in the company's growth and market position.

FDA Gears Up for Key Approval Decisions in Q4 2024

• The FDA is set to decide on dasiglucagon for congenital hyperinsulinism, with a PDUFA date of October 8, potentially benefiting pediatric patients. • Nivolumab-based regimens for resectable NSCLC are under review, supported by Phase 3 data showing a 42% reduction in disease recurrence, with a PDUFA date of October 8. • Acoramidis for transthyretin amyloid cardiomyopathy shows promise, with Phase 3 results indicating statistically significant improvements and a PDUFA date of November 29. • Zanidatamab for HER2-amplified biliary tract cancer is under review, supported by Phase 2b data showing a 41.3% objective response rate, with a PDUFA date of November 29.

A Randomized, Double-Blind Study to Assess the Safety, Efficacy and Tolerability of Oral DFD-29 Capsules for the Treatment of Rosacea.

NCT05296629CompletedPhase 3
Journey Medical Corporation
Posted 3/14/2022

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

NCT03860935CompletedPhase 3
Eidos Therapeutics, a BridgeBio company
Posted 3/19/2019

Oral Sulopenem Versus Amoxicillin/Clavulanate for Uncomplicated Urinary Tract Infection in Adult Women

NCT05584657CompletedPhase 3
Iterum Therapeutics, International Limited
Posted 10/18/2022

A Trial to Assess the Long-term Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly

NCT04125836CompletedPhase 3
Camurus AB
Posted 10/10/2019

A Study Evaluating the Efficacy and Safety of Inavolisib + Palbociclib + Fulvestrant vs Placebo + Palbociclib + Fulvestrant in Participants With PIK3CA-Mutant, Hormone Receptor-Positive, HER2-Negative, Locally Advanced or Metastatic Breast Cancer

NCT04191499Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 1/29/2020

A Study of ZW25 (Zanidatamab) in Subjects With Advanced or Metastatic HER2-Amplified Biliary Tract Cancers

NCT04466891CompletedPhase 2
Jazz Pharmaceuticals
Posted 10/1/2020

A Study of Neoadjuvant Chemotherapy Plus Nivolumab Versus Neoadjuvant Chemotherapy Plus Placebo, Followed by Surgical Removal and Adjuvant Treatment With Nivolumab or Placebo for Participants With Surgically Removable Early Stage Non-small Cell Lung Cancer

NCT04025879Active, Not RecruitingPhase 3
Bristol-Myers Squibb
Posted 11/5/2019

A Two-Period Open-label Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism

NCT03777176CompletedPhase 3
Zealand Pharma
Posted 2/7/2019

Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon in Children With Congenital Hyperinsulinism

NCT03941236Active, Not RecruitingPhase 3
Zealand Pharma
Posted 5/1/2019

A Study to Assess the Safety, Efficacy and Tolerability of Oral DFD-29 Capsules for the Treatment of Rosacea (MVOR-2)

NCT05343455CompletedPhase 3
Journey Medical Corporation
Posted 3/29/2022

Trial Evaluating Efficacy and Safety of Dasiglucagon in Children With Congenital Hyperinsulinism

NCT04172441CompletedPhase 2
Zealand Pharma
Posted 6/19/2020

A Trial to Assess Efficacy and Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly

NCT04076462CompletedPhase 3
Camurus AB
Posted 8/19/2019

IN8bio Restructures to Focus on INB-100 AML Program Following $12.4 Million Private Placement

  • IN8bio is prioritizing its INB-100 program for acute myeloid leukemia (AML) due to promising results and a challenging financing environment.
  • The company is reducing its workforce by approximately 49% and pausing its glioblastoma multiforme (GBM) program to cut costs.
  • A $12.4 million private placement will fund the expansion of the INB-100 Phase I trial, with enrollment expected to complete in H1 2025.
  • Early data from the INB-100 trial shows 100% relapse-free survival in AML patients, supporting the shift in focus and investment.

Novel Gamma-Delta (γδ)T Cell Therapy for Treatment of Patients with Newly Diagnosed Glioblastoma

NCT04165941Active, Not RecruitingPhase 1
University of Alabama at Birmingham
Posted 2/11/2020

A Phase 1b / 2 Drug Resistant Immunotherapy With Activated, Gene Modified Allogeneic or Autologous γδ T Cells (DeltEx) in Combination With Maintenance Temozolomide in Subjects With Recurrent or Newly Diagnosed Glioblastoma

NCT05664243Active, Not RecruitingPhase 1
In8bio Inc.
Posted 9/8/2023

Expanded/Activated Gamma Delta T-cell Infusion Following Hematopoietic Stem Cell Transplantation and Post-transplant Cyclophosphamide

NCT03533816RecruitingPhase 1
University of Kansas Medical Center
Posted 1/31/2020

Sun Pharma and Moebius Medical's MM-II Receives FDA Fast Track Designation for Osteoarthritis Knee Pain

  • The FDA granted Fast Track Designation to MM-II for osteoarthritis knee pain, expediting its development and review process.
  • MM-II is a non-opioid product using liposomes to reduce joint friction, offering a novel approach to pain relief.
  • Phase 2b data showed a single MM-II injection provided greater pain relief than placebo for up to 26 weeks.
  • Sun Pharma and Moebius Medical are advancing MM-II to Phase 3 trials and seeking CE Mark for EU market entry.

Efficacy and Safety of MM-II for Treatment of Knee Pain in Subjects With Symptomatic Knee Osteoarthritis

NCT04506463CompletedPhase 2
Sun Pharmaceutical Industries Limited
Posted 12/22/2020

GSK's Nucala Shows Promise in Reducing COPD Flare-Ups

  • GSK's Nucala demonstrated a statistically significant and clinically meaningful reduction in moderate to severe flare-ups in COPD patients compared to placebo.
  • The advanced trial treated patients for up to two years, indicating a potential long-term benefit in managing this chronic lung condition.
  • Nucala, initially approved for severe asthma, could open a new growth avenue for GSK, expanding its application to include COPD treatment.
  • The trial involved 806 participants, using Nucala in conjunction with inhaled maintenance therapy, with full results to be published later.

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