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Clinical Trial News

Evoke Pharma Secures Patent Extension for GIMOTI Nasal Spray, Extending Market Exclusivity to 2036

  • Evoke Pharma received a Notice of Allowance from the USPTO for a patent application covering GIMOTI use in patients with moderate to severe gastroparesis symptoms.
  • The patent extends market exclusivity for GIMOTI, the only FDA-approved non-oral metoclopramide formulation, by an additional 6 years to December 2036.
  • GIMOTI is indicated for treating acute and recurrent diabetic gastroparesis in adults, offering a critical non-oral treatment option for patients whose oral medication absorption is compromised.
  • The patent allowance drove Evoke Pharma's stock price up over 163% in trading, reflecting investor confidence in the drug's extended market protection.

Celularity Partners with Fountain Life to Deploy Stem Cell Therapies Under New Florida Law

  • Celularity Inc. has entered a strategic partnership with Fountain Life to supply placental-derived stem cell therapies under Florida's new law effective July 1, 2025.
  • The Florida Statute § 458.3245 authorizes qualified physicians to treat patients with stem cell products for orthopedic conditions, wound healing, and pain management without FDA IND approval.
  • Fountain Life operates four longevity centers across the US, including two in Florida, positioning them to implement the new regulatory framework immediately.
  • Celularity maintains substantial inventory of clinical-grade stem cell therapies at its FDA-registered GMP facility, ensuring "Day One" readiness for the partnership.

Neuros Medical Secures $56 Million Series D to Commercialize FDA-Approved Post-Amputation Pain Treatment

FDA Approval
  • EQT Life Sciences co-led a $56 million Series D financing round for Neuros Medical to support U.S. commercialization of Altius®, an FDA-approved direct electrical nerve stimulation system.
  • Post-amputation pain affects up to 80% of amputees in the U.S., representing a severely underserved indication with few effective non-opioid treatment options available.
  • Altius® is currently the only FDA-approved solution for post-amputation pain, delivering targeted nerve stimulation through patient-controlled, on-demand therapy.
  • The oversubscribed funding round will enable Neuros Medical to scale commercial operations and expand access to this breakthrough non-opioid treatment for amputees.

Experimental CoQ10 Precursor Treatment Shows Dramatic Results in 8-Year-Old with Rare Fatal Disease

Rare Disease
  • An 8-year-old boy with HPDL deficiency, a rare mitochondrial disease causing paralysis and often death, showed dramatic improvement after receiving an experimental treatment with 4-hydroxybenzoate (4-HB).
  • The treatment, developed by NYU Langone researchers, bypasses the defective HPDL enzyme by providing CoQ10 precursors directly, allowing cells to produce the essential antioxidant needed for mitochondrial function.
  • Within two months of treatment, the child progressed from needing a wheelchair to walking long distances and running, representing the first successful demonstration of reversing neurological symptoms in primary CoQ10 deficiency.
  • The breakthrough emerged from mouse studies showing 90% survival rates when treated with CoQ10 precursors, compared to death within 15 days without treatment.

AstraZeneca Launches PRIMULA Preg Study to Evaluate Anifrolumab Safety During Pregnancy in SLE Patients

Monoclonal Antibody
  • AstraZeneca has initiated the PRIMULA Preg study, a prospective observational cohort study to assess anifrolumab safety during pregnancy in patients with systemic lupus erythematosus.
  • The study fulfills an FDA post-marketing requirement and compares outcomes between pregnant SLE patients exposed to anifrolumab versus those who are not exposed.
  • Anifrolumab is a human monoclonal antibody targeting the type 1 interferon receptor, approved by FDA and EMA for treating moderate to severe SLE in adults.
  • The study began on August 29, 2025, and aims to provide crucial safety data that could potentially expand anifrolumab's market use during pregnancy.

Novartis Secures Exclusive Option for Sironax's Blood-Brain Barrier Platform in $175M Deal

  • Sironax has granted Novartis an exclusive option to acquire its proprietary Brain Delivery Module (BDM) platform, a differentiated blood-brain-barrier crossing technology designed to enhance brain delivery of therapeutics.
  • The strategic agreement allows Novartis to evaluate the BDM platform within an option period, with Sironax eligible to receive up to $175 million in upfront and near-term payments upon option exercise.
  • Sironax retains rights to continue developing selected therapeutic assets using the platform while Novartis would acquire full global rights to the BDM technology if the option is exercised.
  • The collaboration addresses one of drug discovery's most significant challenges, as effectively delivering therapeutics across the blood-brain barrier remains a critical obstacle in neuroscience drug development.

KALA BIO Completes Phase 2b PCED Trial Enrollment, No FDA-Approved Options Exist

Orphan DrugRare Disease
  • KALA BIO has completed enrollment of 79 patients in the CHASE Phase 2b trial evaluating KPI-012 for persistent corneal epithelial defect (PCED), with topline results expected by Q3 2025.
  • The trial represents a significant milestone for treating an estimated 100,000 Americans with PCED, as no FDA-approved products currently exist with broad indication for all underlying etiologies.
  • KPI-012, a human mesenchymal stem cell secretome therapy, has received both Orphan Drug and Fast Track designations from the FDA for PCED treatment.
  • The CHASE trial could potentially serve as a pivotal study to support a Biologics License Application submission, contingent on positive results and regulatory discussions.

Four-Company Consortium Launches AI-Driven Drug Discovery for Subarachnoid Hemorrhage Complications

Drug Discovery
  • Proteros biostructures, Qanatpharma, Zuse Institute Berlin, and Enamine have formed a research collaboration to develop novel therapeutics for delayed cerebral ischemia, a life-threatening complication of subarachnoid hemorrhage.
  • The partnership leverages generative AI technology from ZIB to accelerate early-stage drug discovery targeting cerebrovascular resistance mechanisms compromised in SAH patients.
  • Compound screening efforts are already underway, with in-vitro validation studies planned for the second half of 2025, representing the most significant effort to address microvascular dysfunction-driven diseases.
  • The collaboration addresses a critical unmet medical need, as current standard of care with nimodipine fails to target the molecular mechanism underlying delayed cerebral ischemia.

Revolution Medicines Partners with Iambic Therapeutics in $25M AI-Driven Drug Discovery Collaboration for RAS-Addicted Cancers

AIDrug DiscoveryOncologyTargeted Therapy
  • Revolution Medicines and Iambic Therapeutics announced a multi-year collaboration in July 2025 to develop novel drug candidates using AI-driven discovery platforms targeting RAS-addicted cancers.
  • The partnership leverages Iambic's NeuralPLexer protein structure prediction model and PropANE neural network, trained on Revolution's proprietary data to accelerate discovery of therapies for challenging oncology targets.
  • Under the agreement, Iambic will receive up to $25 million through upfront payments, performance-based milestones, and R&D reimbursements, while both companies retain rights to exclusive targets.
  • The collaboration aims to address RAS mutations that drive approximately 30% of all cancers but remain among the most challenging drug targets for conventional discovery methods.

Exactech Receives FDA Clearance for First-to-Market Scapula Reconstruction System Targeting Post-Surgical Fractures

  • Exactech's Equinoxe Scapula Reconstruction System has received FDA 510(k) clearance as the first device specifically designed to treat acromial and scapular spine fractures following reverse total shoulder arthroplasty.
  • The system addresses a significant unmet clinical need, as acromial and scapular fractures occur in an average of 2.8% of patients after reverse shoulder replacement, with rates reaching up to 10.9% in certain patient populations.
  • The novel dual plating system features anatomically contoured plates with integral hooks designed to counteract deltoid muscle pull and biomechanical loading from prosthetic implants.
  • The device will enter pilot launch with limited U.S. availability later this year, representing a new treatment paradigm for managing these challenging post-surgical complications.

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