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Clinical Trial News

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.

ARCHER Trial Opens Enrollment to Test Shortened Radiation Therapy for Muscle Invasive Bladder Cancer

  • The phase 3 ARCHER trial (NRG-GU015) is now enrolling 486 patients to compare ultra-hypofractionated stereotactic body radiation therapy (5 treatments) versus standard hypofractionated radiotherapy (20 treatments) for muscle invasive bladder cancer.
  • Up to 20% of muscle invasive bladder cancer patients currently forego curative treatment due to logistical concerns related to frequent visits to specialized centers over several months.
  • The study aims to determine if the shortened radiation regimen can maintain bladder-intact event-free survival at 3 years while reducing patient burden and improving quality of life.
  • Translational research components include circulating tumor DNA analysis as a biomarker for predicting disease recurrence and guiding future personalized treatment approaches.

Testing Shorter Duration Radiation Therapy Versus the Usual Radiation Therapy in Patients Receiving the Usual Chemotherapy Treatment for Bladder Cancer, ARCHER Study

NCT07097142Not Yet RecruitingNot Applicable
NRG Oncology
Posted 8/14/2025

Citizen Health Raises $30M Series A to Develop AI Advocate for Rare Disease Patients

AIRare Disease
  • Citizen Health secured $30 million in Series A funding led by 8VC to develop an AI Advocate platform for rare disease patients, bringing total funding to $44 million since December 2023.
  • The platform has built over 60 communities across 123 rare conditions and achieved a 98.3% patient data-sharing rate, with data already accepted by the FDA for regulatory submissions.
  • The company plans to launch its AI Advocate to select communities in Q3 2025, focusing on transforming fragmented healthcare into proactive, personalized patient experiences.
  • With rare diseases affecting over 400 million people globally and 95% lacking approved treatments, the platform addresses critical unmet needs in rare disease care and research.

Rubicon Research Secures ₹250 Crore Pre-IPO Investment from Amansa, Reduces Fresh Issue Size Ahead of Year-End Public Listing

  • Amansa Investments has completed a ₹250 crore pre-IPO investment in pharmaceutical formulations company Rubicon Research, acquiring shares at ₹484.47 per share.
  • The investment reduces Rubicon Research's fresh IPO issue size from ₹500 crore, with the company expected to launch its maiden public offering by the end of 2024.
  • General Atlantic Singapore remains the majority shareholder with 54.01% stake after transferring 51.60 lakh equity shares to Amansa Investments.
  • Rubicon Research operates two US FDA-inspected R&D facilities and maintains a portfolio of 69 USFDA-approved products targeting regulated markets, particularly the United States.

Repare Therapeutics Reports 52% Reduction in Q2 Loss Amid Strategic Pivot to Licensing Model

  • Repare Therapeutics narrowed its Q2 2025 GAAP loss per share to $0.39, significantly beating analyst estimates of -$0.56, primarily driven by a $5.7 million gain from technology platform licensing deals.
  • The precision oncology company reduced operating expenses by 38.3% year-over-year while securing major partnerships, including a $10 million upfront licensing deal with Debiopharm for its PKMYT1 inhibitor lunresertib.
  • Two key clinical trials, POLAR for RP-3467 targeting DNA polymerase theta and LIONS for RP-1664 targeting PLK4 kinase, are expected to report initial findings by the end of 2025.

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