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First Commercial Brain Gene Therapy Treatments Delivered in US Using ClearPoint Neuro's SmartFlow Cannula

FDA ApprovalRare DiseaseNeurology
  • The first commercial gene therapy treatments ever delivered directly to the brain in the United States have been successfully performed using ClearPoint Neuro's SmartFlow Neuro Cannula to administer KEBILIDI for AADC deficiency.
  • KEBILIDI (eladocagene exuparvovec-tneq) is delivered directly to the putamen region of the brain through stereotactic neurosurgery, with the SmartFlow cannula being the only FDA-authorized device for this administration.
  • ClearPoint Neuro reported record clinical activity in July with 17 global patients treated across 11 different drug platforms, while nine of the company's partners have now been accepted into FDA expedited review programs.
  • New ICD-10 codes specific to neuro infusions will become effective October 1, 2025, to assist in tracking commercial use of brain-delivered therapies.

Evestia Clinical Merges with Atlantic Research Group to Create Global Specialist CRO

NeurologyOncology
  • Evestia Clinical (formerly EMAS Pharma) has merged with US-based Atlantic Research Group to create an independent global specialist CRO group in fast-growing markets.
  • The merger more than doubles Evestia Clinical's size by headcount and service delivery capability while expanding its US footprint and therapeutic offerings to include neurology and late-stage clinical trials.
  • The combined group will provide full clinical trial services from Phase 1 through Phase 3 and beyond, with enhanced technologies and specialized services across multiple therapeutic areas.
  • Following integration, the enlarged group will be headquartered in the UK (Letchworth) and USA (Charlottesville, VA) with global capabilities through its network of offices and expert partners.

Oragenics Raises $20 Million to Advance Concussion Treatment Clinical Trials

Neurology
  • Oragenics completed a $20 million preferred stock offering to fund ongoing ONP-2 concussion clinical trials and related research and development activities.
  • The biotechnology company focuses on developing intranasal pharmaceuticals for neurological disorders, including treatments for mild traumatic brain injury (mTBI).
  • The offering includes 800,000 shares of Series H Convertible Preferred Stock at $25 per share, convertible to common stock at $2.50 per share.
  • Proceeds will also support repayment of a $3 million bridge note and general corporate purposes as the company advances its neurology pipeline.

Brandon Capital Opens Second $5M Funding Round for Dementia Innovation Program

Neurology
  • Brandon Capital has launched the second funding round for its CUREator+ Dementia and Cognitive Decline incubator program, offering up to $5 million per project to support innovative dementia solutions.
  • The program targets the development of therapeutics, diagnostics, medical devices, and digital health technologies for Australia's 433,300 dementia patients and 1.7 million caregivers.
  • Funded by Australia's Medical Research Future Fund, the initiative provides non-dilutive grants through a competitive merit-based process with applications closing July 10, 2025.
  • Eligible projects span early diagnosis, progression prevention, symptom management, and quality of life improvements across preclinical, clinical, and commercial development stages.

World-First Octopus Trial Offers New Hope for Progressive Multiple Sclerosis Patients

Neurology
  • The innovative Octopus trial represents the world's first multi-armed, multi-staged clinical study specifically designed to test multiple potential treatments for progressive multiple sclerosis simultaneously.
  • The trial is evaluating metformin and lipoic acid against placebo in progressive MS patients, utilizing adaptive design methodology previously successful in cancer and COVID-19 research.
  • More than 75,000 people in the UK suffer from progressive MS, a devastating condition with virtually no available treatments that causes gradual worsening without remission.
  • The MS Society is funding this groundbreaking research through their Stop MS Campaign, aiming to address the critical unmet medical need in progressive forms of the disease.

Sun Pharma Invests $25 Million in Pharmazz, Expanding Stake to 22.7% and Securing Key Drug Rights

Neurology
  • Sun Pharmaceutical Industries Ltd has announced a strategic $25 million investment in US-based Pharmazz Inc, increasing its ownership stake to 22.7% in the biopharmaceutical company.
  • The investment will be completed in stages, with an initial $10 million tranche plus $7.5 million from a previous agreement by May 2025, and an additional $15 million expected by November 2025.
  • Through this investment, Sun Pharma secures exclusive rights to market Sovateltide in select emerging markets and gains options to negotiate licensing rights in certain developed countries.

Assess the Safety and Efficacy of Sovateltide in Patients With Acute Cerebral Ischemic Stroke

NCT05691244RecruitingPhase 3
Pharmazz, Inc.
Posted 7/24/2025

Curatis Reveals Corticorelin as Active Substance in C-PTBE-01 with Billion-Dollar Market Potential

OncologyNeurology
  • Curatis has disclosed that Corticorelin is the active substance in its C-PTBE-01 therapy for peritumoral brain edema (PTBE), with potential to reduce or eliminate steroid use in treatment.
  • The company has significantly increased its market size estimates, now projecting over 500,000 PTBE patients globally and a market opportunity exceeding USD 1 billion.
  • Curatis is targeting an FDA meeting in Q3 2024 to finalize the design of a pivotal Phase 3 trial planned for 2026, while strengthening its team with key appointments in clinical development and technical operations.

Texas A&M Researcher's Umbilical Cord Cell Therapy Shows Promise for ALS Treatment in Clinical Trials

NeurologyFDA Approval
  • Dr. Simrit Parmar at Texas A&M University has developed an innovative umbilical cord blood-derived T regulatory cell therapy that has demonstrated safety and efficacy in treating over 80 patients with ALS and other inflammatory diseases.
  • The therapy addresses inflammation using Treg cells that do not require donor-recipient matching and can be manufactured at scale from a single cord blood unit, with products remaining viable for up to three years when frozen.
  • Recent studies published in NEJM Evidence showed functional improvement in ALS patients following multiple infusions, while a historic partnership with Saudi Arabia's King Faisal Specialist Hospital will launch new clinical trials.
  • Dr. Parmar aims to secure FDA regulatory approval within two years for aplastic anemia treatment while seeking expanded funding for larger ALS patient studies in the United States.

Ulefnersen Shows Unprecedented Recovery in Young Patients with Rare FUS-ALS

Rare DiseasePrecision MedicineNeurology
  • Columbia University researchers report that ulefnersen, an experimental drug, demonstrated remarkable efficacy in treating FUS-ALS, a rare genetic form of ALS affecting young people.
  • In a small case series of 12 patients, two showed exceptional responses, including one young woman who regained the ability to walk and breathe independently after treatment.
  • The therapy reduced neurofilament light, a biomarker of nerve damage, by up to 83% after six months, suggesting potential for not just slowing but reversing functional losses in early intervention.

A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis

NCT04973137TerminatedPhase 3
Prothena Biosciences Ltd.
Posted 8/30/2021

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)

NCT04512235Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 11/12/2020

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)

NCT04504825Active, Not RecruitingPhase 3
Alexion Pharmaceuticals, Inc.
Posted 2/2/2021

Ruxolitinib Shows Promise in Treating CAR T-Cell Therapy-Induced Parkinsonism in Multiple Myeloma

CAR-TNeurologyOncology
  • Researchers report successful treatment of ciltacabtagene autoleucel-induced parkinsonism in multiple myeloma patients using the JAK inhibitor ruxolitinib, offering a potential solution for this serious complication.
  • Both patients developed immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS) alongside parkinsonism, suggesting a possible connection between these conditions in CAR T-cell therapy recipients.
  • The findings are particularly significant as CAR T-cell therapy expands to earlier treatment lines for multiple myeloma, highlighting the urgent need for effective management strategies for neurological complications.

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