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Clinical Trial News

Ascelia Pharma Nears Orviglance NDA Submission for Orphan Liver Imaging Agent

Orphan Drug
  • Ascelia Pharma is finalizing its New Drug Application (NDA) for Orviglance, a first-in-class oral MRI contrast agent for liver imaging in kidney-impaired patients.
  • The pivotal Phase 3 SPARKLE trial demonstrated statistically significant improvements in lesion visualization compared to unenhanced MRI across three independent readers (p < 0.001).
  • Orviglance addresses a $300-450 million annual U.S. market opportunity with no direct competitors, targeting 100,000 liver MRI procedures in renal-impaired patients.
  • The drug has received Orphan Drug Designation from the FDA, providing seven years of market exclusivity and expedited review pathways upon approval.

Pfizer's Inclacumab Fails Phase III Trial for Sickle Cell Disease, Marking Second Setback from Global Blood Therapeutics Acquisition

Monoclonal Antibody
  • Pfizer's investigational P-selectin inhibitor inclacumab failed to meet its primary endpoint of reducing vaso-occlusive crises in a Phase III trial for sickle cell disease.
  • The 48-week THRIVE-131 study enrolled 241 participants aged 16 and older but showed no significant difference in VOC rates compared to placebo.
  • This represents the second major disappointment from Pfizer's $5.4 billion acquisition of Global Blood Therapeutics, following Oxbryta's market withdrawal in 2024.
  • Despite the setback, Pfizer maintains its commitment to developing treatments for sickle cell disease patients and advancing research in this therapeutic area.

A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease

NCT05348915Active, Not RecruitingPhase 3
Pfizer
Posted 3/29/2022

A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises

NCT04935879CompletedPhase 3
Pfizer
Posted 10/4/2021

FDA Approves Precigen's PAPZIMEOS as First Gene Therapy for Recurrent Respiratory Papillomatosis

Drug ApprovalRare Disease
  • The FDA granted full approval to Precigen's PAPZIMEOS (zopapogene imadenovec-drba), marking the first and only treatment approved for adults with recurrent respiratory papillomatosis (RRP).
  • In clinical trials, 51% of patients achieved complete response with no surgeries needed for at least one year after treatment, with most remaining surgery-free two years later.
  • The approval triggered a dramatic pre-market surge of over 50% in Precigen's stock price, reflecting investor confidence in the company's innovative gene therapy platform.
  • PAPZIMEOS represents a breakthrough for approximately 27,000 U.S. adults suffering from RRP, a condition caused by HPV that previously required repeated surgical interventions.

AstraZeneca's FluMist Home Becomes First Self-Administered Flu Vaccine Available for Home Delivery in US

Drug Approval
  • AstraZeneca's FluMist Home is now available for home delivery in 34 US states, marking the first time Americans can self-administer a flu vaccine at home.
  • The nasal spray vaccine can be used by adults under 50 and children as young as 2 with parental assistance, following FDA approval for self-administration in September 2024.
  • The home delivery option aims to address low vaccination rates, as less than half of Americans received flu vaccines during the severe 2024-2025 season that caused up to 82 million illnesses.
  • AstraZeneca's usability study demonstrated 100% of intended users successfully administered a full dose without healthcare provider supervision.

AI Model COMET Accelerates RNA Medicine Development, Cutting Timelines from Months to Weeks

AIDrug Discovery
  • Researchers from NTU and MIT developed COMET, an AI model that predicts optimal lipid nanoparticle formulations for RNA medicine delivery, published in Nature Nanotechnology.
  • The transformer-based neural network can identify the best nanoparticle designs among millions of possibilities without extensive laboratory testing, reducing development timelines from months to weeks.
  • COMET-designed lipid nanoparticles outperformed clinically approved benchmarks in both laboratory and animal studies, demonstrating superior delivery performance.
  • The technology extends beyond pandemic preparedness to enable RNA therapies targeting specific organs and cell types for treating cancers and metabolic disorders.

Sol-Gel Completes Phase 3 Enrollment for Gorlin Syndrome Treatment SGT-610, Targets $300M Peak Revenue

Rare Disease
  • Sol-Gel Technologies has completed patient enrollment for its pivotal Phase 3 clinical trial of SGT-610, a topical hedgehog pathway inhibitor for preventing basal cell carcinomas in Gorlin syndrome patients.
  • SGT-610 could become the first approved therapy for preventing new BCC lesions in this rare genetic disorder, with the company targeting potential peak revenue exceeding $300 million annually.
  • The company's Phase 1b trial of SGT-210 for Darier disease continues despite recruitment challenges, with market potential estimated between $200-300 million.
  • Sol-Gel secured $16 million from selling U.S. rights to EPSOLAY and TWYNEO to Mayne Pharma, extending its cash runway into the first quarter of 2027.

UMass Chan Gene Therapy Shows Biochemical Correction in GM2 Gangliosidosis Phase I/II Trial

Rare Disease
  • A Phase I/II clinical trial at UMass Chan Medical School demonstrated biochemical correction of GM2 gangliosidosis using dual vector gene therapy with minimal adverse reactions.
  • Trial participants maintained oral feeding for longer periods and experienced fewer, more controllable seizures compared to typical disease progression.
  • All nine participants showed increased HexA enzyme production surpassing two times the lower limit of normal, though therapeutic levels were not achieved.
  • Researchers plan to modify the approach to a single vector system to double therapeutic DNA delivery and enable earlier treatment.

RESTEM Receives FDA Orphan Drug Designation for Stem Cell Therapy in Polymyositis Treatment

Rare Disease
  • RESTEM, a clinical-stage biotech company, received FDA Orphan Drug Designation in December 2024 for its umbilical cord outer lining stem cell program targeting polymyositis and dermatomyositis.
  • The polymyositis therapeutic pipeline includes over 7 companies developing treatments, with emerging therapies like HuABC2, PF1801, PN-101, and M5049 advancing through clinical trials.
  • Polymyositis is a rare autoimmune disease causing inflammation and weakness of skeletal muscles, with current treatment options limited and high drug costs creating market barriers.

AI-Designed Antibiotics Show Promise Against Drug-Resistant Gonorrhea and MRSA in Laboratory Studies

AI
  • MIT researchers used generative AI to design two novel antibiotics capable of killing drug-resistant gonorrhea and MRSA in laboratory and animal tests.
  • The AI system analyzed 36 million compounds using two innovative approaches to create completely new molecular structures distinct from existing antibiotics.
  • While promising, only two of 80 AI-generated gonorrhea treatments could be synthesized, highlighting manufacturing challenges before human clinical trials can begin.
  • Experts believe this breakthrough could herald a "second golden age" of antibiotic discovery in the fight against antimicrobial resistance.

Delaware Court Rules Pfizer COVID-19 Vaccine Does Not Infringe Alnylam's Nanoparticle Patents

  • The U.S. District Court for the District of Delaware entered a final judgment on July 30, 2025, ruling that Pfizer's COVID-19 vaccine did not infringe any of Alnylam's patents covering nanoparticle technology.
  • The court previously denied Alnylam's motion for additional construction of the term "cationic lipid" on April 28, 2025, noting that Alnylam was dissatisfied with its own proposed definition.
  • Following the court's denial, Alnylam withdrew its opposition to Pfizer's summary judgment motion and requested to stay all pre-trial and trial deadlines.
  • The final judgment also dismissed all of Pfizer's affirmative defenses and counterclaims without prejudice, concluding the patent dispute between the two pharmaceutical companies.

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