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Clinical Trial News

Pharmacists Lead Implementation of Advanced Cell and Gene Therapies Across Major Health Systems

Drug Approval
  • Pharmacists are uniquely positioned to lead the implementation of advanced cell and gene therapies, serving as both clinical and operational experts in health systems managing these complex treatments.
  • Major health systems are already delivering widespread advanced therapeutics, with Mayo Clinic offering 9 active therapies, UC San Diego Health providing 12, and Children's Hospital of Philadelphia making 12 available to eligible patients.
  • Significant challenges include high costs, manufacturing variability, patient hesitancy toward gene manipulation, and the need for new governance structures and educational frameworks.
  • The first CRISPR-based medicine, exagamglogene autotemcel (Casgevy), was approved in 2023 for treating sickle cell disease and transfusion-dependent β-thalassemia, marking a milestone in gene therapy applications.

Iambic Therapeutics Showcases AI-Driven Drug Discovery Platform at Multiple Investor Conferences

AIDrug Discovery
  • Iambic Therapeutics, a clinical-stage biotechnology company, will present its AI-driven drug discovery platform at six major investor conferences between April and June 2025.
  • The company's platform features advanced technologies including Enchant, a multimodal transformer model that predicts clinical outcomes, and NeuralPLexer for protein structure prediction.
  • Iambic has demonstrated the ability to complete design-make-test cycles on a weekly cadence through integration of AI-generated molecular designs with automated experimental execution.
  • The San Diego-based company, founded in 2020, is advancing a pipeline of potential best-in-class and first-in-class clinical assets to address unmet patient needs.

Precision Medicine Approaches Transform Bronchiectasis Management as Research Centers Expand Nationwide

Precision Medicine
  • Bronchiectasis management is shifting from standardized treatment algorithms to precision medicine approaches that target specific patient phenotypes and symptoms.
  • The COPD Foundation has established 27 bronchiectasis and nontuberculous mycobacteria Centers of Excellence with plans to expand to over 150 centers nationwide.
  • Current clinical trials are investigating targeted therapies including DPP1 inhibition, Type 2 inflammation treatments, and CFTR modulators for non-cystic fibrosis bronchiectasis.
  • A "treatable traits" approach focusing on individual patient characteristics is improving management outcomes and quality of life for bronchiectasis patients.

Ultra-Processed Foods Linked to Increased Asthma and Allergy Risk in Children, Research Shows

  • A systematic review published in Pediatric Allergy and Immunology found significant associations between ultra-processed food consumption and the development of allergies and asthma in children.
  • Ultra-processed foods now comprise over 50% of dietary energy intake in high-income countries, making their health impacts a critical public health concern.
  • Research demonstrates that ultra-processed foods alter the gut microbiome and cause immune dysregulation, potentially explaining their connection to respiratory diseases.
  • Socioeconomic disparities limit access to fresh, healthy foods in low-income areas, contributing to higher rates of metabolic dysfunction and respiratory conditions like asthma.

Oral Ritlecitinib Combined with Phototherapy Shows Enhanced Repigmentation in Nonsegmental Vitiligo

  • Combination therapy of oral ritlecitinib plus narrowband UV-B phototherapy demonstrated superior repigmentation results compared to ritlecitinib monotherapy in nonsegmental vitiligo patients.
  • The combination treatment achieved 69.6% mean improvement in facial repigmentation versus 55.1% with ritlecitinib alone in phase 2b clinical trial results.
  • Ritlecitinib, a JAK3/TEC family kinase inhibitor already approved for severe alopecia areata, is advancing through phase 3 trials for vitiligo treatment.
  • The dual-step therapeutic approach addresses both inflammation reduction and melanocyte regeneration, offering new hope for patients with this psychologically impactful autoimmune condition.

A 104-Week Study of Ritlecitinib Oral Capsules in Adults With Nonsegmental Vitiligo (Active and Stable) Tranquillo 2

NCT06072183Active, Not RecruitingPhase 3
Pfizer
Posted 11/8/2023

A Phase 2b Study To Evaluate The Efficacy And Safety Profile Of PF-06651600 And PF-06700841 In Active Non-segmental Vitiligo Subjects

NCT03715829CompletedPhase 2
Pfizer
Posted 11/26/2018

A 52-Week Study to Learn About the Safety and Effects of Ritlecitinib in Participants With Nonsegmental Vitiligo

NCT06163326RecruitingPhase 3
Pfizer
Posted 1/19/2024

A 52-Week Study of Ritlecitinib Oral Capsules in Adults and Adolescents With Nonsegmental Vitiligo (Active and Stable) Tranquillo

NCT05583526Active, Not RecruitingPhase 3
Pfizer
Posted 12/1/2022

EpiVax Appoints Dr. Vibha Jawa as Chief Scientific Officer to Lead Immunogenicity Strategy

  • EpiVax, a leader in preclinical immunogenicity assessment for biologic therapeutics and vaccines, has appointed Dr. Vibha Jawa as Chief Scientific Officer, succeeding founder Dr. Annie De Groot who transitions to Chief Medical Officer.
  • Dr. Jawa brings over two decades of experience in biologics, vaccine, and gene therapy development from senior roles at Bristol Myers Squibb, Merck, and Amgen, contributing to more than 20 successful regulatory filings.
  • The appointment marks a key milestone in EpiVax's transformation that began in 2024, with Dr. Jawa expected to expand the company's reach into new therapeutic areas and advance immunogenicity science through AI and immunoinformatics integration.

Global Study Links COVID-19 Infection and Adenoviral Vaccines to Guillain-Barré Syndrome Risk, Clears mRNA Vaccines

  • A multinational study analyzing data from over 230 million people across 20 global sites found that SARS-CoV-2 infection increases Guillain-Barré syndrome risk by approximately three times within six weeks of infection.
  • Adenoviral vector COVID-19 vaccines (AstraZeneca, Janssen/Johnson & Johnson) showed increased GBS risk, while mRNA vaccines (Pfizer-BioNTech, Moderna) and inactivated vaccines (Coronavac/Sinovac) did not demonstrate elevated risk.
  • The research reinforces that COVID-19 infection poses greater neurological health risks than vaccination, supporting continued vaccination as a public health tool.
  • The Global Vaccine Data Network study emphasizes the importance of continuous vaccine safety monitoring and provides evidence-based guidance for different vaccine types.

Trump Administration Cancels $590 Million Moderna Bird Flu Vaccine Contract Amid mRNA Technology Review

  • The Trump administration has terminated Moderna's $590 million contract for developing H5N1 bird flu vaccines, citing failure to meet scientific and safety standards for continued federal investment.
  • HHS officials stated that mRNA technology remains "under-tested" and criticized the previous administration for concealing safety concerns, despite mRNA vaccines being administered nearly 2 billion times globally.
  • The cancellation eliminates what experts consider the most rapid vaccine production platform available for pandemic response, as bird flu has infected 70 people in the past year.
  • Moderna reported positive interim data from mid-stage trials showing "rapid, potent and durable immune response" in approximately 300 healthy adults, and plans to explore alternative funding paths.

A Study of mRNA-1018 Pandemic Influenza Candidate Vaccines in Healthy Adults

NCT05972174CompletedPhase 1
ModernaTX, Inc.
Posted 7/10/2023

A Study of mRNA-1010 Compared With a Licensed Influenza Vaccine in Adults ≥50 Years of Age

NCT06602024CompletedPhase 3
ModernaTX, Inc.
Posted 9/16/2024

Supernus Faces Generic Competition as Third Parties File ANDA Challenges for ADHD Drug Qelbree

  • Supernus Pharmaceuticals received Paragraph IV Notice Letters from third parties who submitted Abbreviated New Drug Applications (ANDAs) for generic versions of Qelbree (viloxazine extended-release capsules) in 100, 150, and 200 mg strengths.
  • The company plans to vigorously enforce its intellectual property rights, with six issued U.S. patents covering Qelbree that expire between September 2029 and April 2035.
  • This generic challenge represents a significant threat to Supernus's ADHD treatment franchise, as generic competition could substantially impact future revenue streams for the CNS-focused biopharmaceutical company.

Eisai and Merck Secure Patent Victory, Blocking Lenvima Generic Until 2036

  • Eisai and Merck successfully defended a key patent for cancer drug Lenvima against Shilpa Medicare's challenge, with a federal judge ruling the patent describes the invention clearly and in sufficient detail.
  • The patent victory covers purified lenvatinib mesylate and blocks Shilpa's generic version from FDA approval until the patent expires in February 2036.
  • Lenvima generated over $1 billion in alliance revenues for MSD last year and $1.51 billion in US sales for Eisai, making it a significant blockbuster cancer treatment.
  • The drug is widely used alongside Keytruda as first-line therapy for kidney cancer and second-line treatment for endometrial cancer, having treated over half a million patients worldwide.

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