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Clinical Trial News

FDA Approves Two Bispecific T-Cell Engagers for Relapsed/Refractory B-Cell Lymphomas

Drug ApprovalOncology
  • The FDA has granted accelerated approval to epcoritamab-bysp (Epkinly), the first T-cell engaging bispecific antibody for relapsed/refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy.
  • Epcoritamab demonstrated a 61% overall response rate with a 38% complete response rate in the EPCORE NHL-1 trial, with a median duration of response of 15.6 months.
  • The FDA also approved glofitamab-gxbm (Columvi), another CD20/CD3-targeted bispecific T-cell engager, showing a 56% overall response rate and 43% complete response rate in clinical trials.
  • Both therapies carry boxed warnings for serious immune-related adverse reactions including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.

First-in-Human (FIH) Trial in Patients With Relapsed, Progressive or Refractory B-Cell Lymphoma

NCT03625037Active, Not RecruitingPhase 1
Genmab
Posted 6/26/2018

FDA Approves Krystal Biotech's Vyjuvek, a Topical Gene Therapy for Dystrophic Epidermolysis Bullosa

  • The FDA has approved Vyjuvek, Krystal Biotech's topical gene therapy, marking the first treatment of its kind for dystrophic epidermolysis bullosa.
  • Vyjuvek is approved for patients aged six months and older with either recessive or dominant forms of dystrophic epidermolysis bullosa.
  • Clinical trials showed Vyjuvek completely healed wounds in approximately 65% of patients, compared to 21.6% in the placebo group.
  • Krystal Biotech anticipates Vyjuvek will be available in the U.S. in Q3 2023, with plans to provide access to all U.S. patients.

AI-Powered Clinical Trial Matching Platforms Show Promise in Addressing Patient Recruitment Challenges

• Clinical trial recruitment faces significant challenges with 86% of trials failing to meet enrollment timelines, but AI-powered matching platforms are emerging as an effective solution for connecting patients to suitable trials.
• Companies like Belong.Life and myTomorrows have developed AI platforms that demonstrate high accuracy in patient matching, with one Australian study showing over 90% accuracy in eligibility assessment.
• While AI matching platforms show promise, experts emphasize the continued importance of human oversight, data standardization, and privacy protection in implementing these technologies effectively.

FDA Accepts Satsuma Pharmaceuticals' NDA for STS101 Migraine Treatment

  • The FDA has accepted Satsuma Pharmaceuticals' 505(b)(2) NDA for STS101, a novel nasal powder formulation of dihydroergotamine mesylate (DHE) for acute migraine treatment.
  • STS101 is designed for easy self-administration and rapid achievement of high drug plasma levels, potentially offering advantages over existing DHE products.
  • The NDA is supported by Phase 1 and Phase 3 trial results, including the SUMMIT trial, which demonstrated robust and sustained effects on migraine endpoints.
  • Satsuma Pharmaceuticals is in the process of being acquired by Shin Nippon Biomedical Laboratories, Ltd. (SNBL).

Siren Biotechnology Unveils Promising Preclinical Data for Novel Cancer Treatment Combining AAV Gene Therapy with Cytokine Immunotherapy

  • Siren Biotechnology's Universal AAV Immuno-Gene Therapy demonstrated rapid tumor reduction in high-grade glioma organoids and significant survival benefits in multiple mouse models, with complete responses in up to 60% of cases.
  • The novel approach combines AAV gene therapy with engineered interferon cytokines, causing widespread tumor cell apoptosis within 48 hours and complete tumor eradication by day 7, with no evidence of residual proliferating cells.
  • Led by CEO Dr. Nicole Paulk, Siren is initially focusing on brain and eye cancers where systemic treatments have historically failed, with ambitions to establish their therapy as a standard of care for solid tumors.

Elevar Therapeutics Seeks FDA Approval for Rivoceranib and Camrelizumab Combination in Unresectable Hepatocellular Carcinoma

  • Elevar Therapeutics has submitted an NDA to the FDA for rivoceranib in combination with camrelizumab as a first-line treatment for unresectable hepatocellular carcinoma (uHCC).
  • The NDA submission is based on Phase 3 CARES 310 study results, which demonstrated statistically significant improvements in overall and progression-free survival compared to sorafenib.
  • The combination therapy showed a median overall survival of 22.1 months versus 15.2 months for sorafenib, with a hazard ratio of 0.62 (95% CI 0.49-0.80; p<0.0001).
  • Rivoceranib and camrelizumab combination was approved in China in February 2023 as a first-line treatment for liver cancer.

A Study to Evaluate SHR-1210 in Combination With Apatinib as First-Line Therapy in Patients With Advanced HCC

NCT03764293CompletedPhase 3
Jiangsu HengRui Medicine Co., Ltd.
Posted 6/10/2019

Dog Aging Project Explores Rapamycin to Extend Lifespan and Improve Healthspan

  • The Dog Aging Project is studying over 44,000 dogs to understand aging and age-related diseases, aiming to improve both canine and human healthspan.
  • Researchers are investigating rapamycin, a drug known to extend lifespan in lab animals, to see if it can promote healthy aging and longevity in dogs.
  • Early trials of rapamycin in dogs showed improvements in heart function and increased activity levels, with no significant side effects reported by owners.
  • The ongoing TRIAD study, involving 580 dogs, is assessing the impact of rapamycin on lifespan, with results expected in approximately three years.

NIH Awards $7.5M Grant to Advance Vaxxas' HD-MAP Vaccine Technology

  • Vaxxas receives a $7.5 million grant from the NIH to further develop its high-density microarray patch (HD-MAP) vaccine technology.
  • The funding follows promising Phase 1/2 clinical trial results published in The Lancet, supporting the potential of HD-MAP for improved vaccine delivery.
  • The grant will support advanced development and manufacturing scale-up of Vaxxas' needle-free vaccine delivery system.
  • Vaxxas' HD-MAP technology offers the potential for enhanced immune responses and simplified vaccine administration, addressing key challenges in global immunization.

Tucatinib Plus Trastuzumab Shows Promise in HER2-Positive Metastatic Colorectal Cancer

• Tucatinib plus trastuzumab demonstrates clinically meaningful anti-tumor activity in patients with chemotherapy-refractory, HER2-positive metastatic colorectal cancer. • The combination therapy achieved a confirmed objective response rate of 38.1% per blinded independent central review (BICR) in the full analysis set. • Diarrhea was the most common adverse event, with hypertension being the most frequent grade 3 or worse adverse event. • This regimen is the first FDA-approved anti-HER2 therapy for metastatic colorectal cancer, offering a new treatment option.

Tucatinib Plus Trastuzumab in Patients With HER2+ Colorectal Cancer

NCT03043313CompletedPhase 2
Seagen Inc.
Posted 6/23/2017

Viaskin Peanut Allergy Patch Shows Promise in Toddlers

  • A clinical trial reveals that a skin patch, Viaskin, significantly increases peanut tolerance in toddlers with peanut allergies.
  • Approximately 67% of children aged one to three wearing the Viaskin patch for a year could safely ingest more peanut protein than before.
  • The study, involving 362 toddlers across eight countries, offers hope for a new treatment option where few exist for young children.
  • The Viaskin patch represents a potential alternative to strict avoidance, which can significantly impact the quality of life for children and families.

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