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AnnJi Pharmaceutical Reports Promising Phase 1/2a Results for AJ201 in SBMA, a Rare Neuromuscular Disease

Rare DiseaseNeurology
  • AnnJi Pharmaceutical's AJ201 demonstrated positive results in a Phase 1/2a trial for Spinal and Bulbar Muscular Atrophy (SBMA), showing improvements in physical function and muscle biomarkers after 12 weeks of treatment.
  • The investigational drug reduced mutant androgen receptor levels by more than 50% in over half of treated patients and activated the Nrf2 pathway, supporting its proposed mechanism of action.
  • With no FDA-approved treatments currently available for SBMA, AnnJi plans to advance AJ201 to Phase 3 trials, potentially offering hope for approximately 1 in 40,000 males affected by this rare genetic disorder.

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

NCT05517603CompletedPhase 1
AnnJi Pharmaceutical Co., Ltd.
Posted 2/28/2023

ONWARD Medical Advances Brain-Computer Interface Technology with Fifth Successful Implant for Spinal Cord Injury Patients

AINeurology
  • ONWARD Medical has successfully implanted its ARC-BCI® Therapy in two additional patients, bringing the total to five individuals with spinal cord injuries who can now potentially control movement through thought.
  • The innovative DigitalBridge™ system creates a wireless connection between brain and spinal cord, using AI to decode neural signals and translate intention into movement, bypassing damaged neural pathways.
  • With FDA Breakthrough Device Designation and growing clinical evidence, ONWARD's less invasive BCI approach is positioning the company as a leader in neurotechnology for restoring function after paralysis.

Protagenic Therapeutics Secures $3.1 Million to Advance CNS Drug Pipeline Following Phytanix Bio Merger

Neurology
  • Protagenic Therapeutics (Nasdaq: PTIX) has raised $3.1 million through warrant exchanges and exercises, bolstering its financial position to advance peptide-based drug candidates for CNS disorders.
  • The funding comes immediately after Protagenic's business combination with Phytanix Bio on May 19, 2025, creating a unified company with six complementary drug programs targeting stress disorders, epilepsy, and obesity.
  • The merged entity combines Protagenic's expertise in peptide therapeutics with Phytanix's specialization in cannabinoid and cannabinoid-like molecules, potentially strengthening their competitive position in neurological drug development.

Nitrous Oxide Breakthrough Enables Safer Gene Therapy Delivery to Brain

Neurology
  • UT Southwestern researchers discovered that nitrous oxide can safely enhance gene therapy delivery to the brain by improving blood-brain barrier permeability when combined with focused ultrasound.
  • The novel approach required up to 1,000 times lower microbubble concentrations and significantly reduced ultrasound pressure compared to conventional methods, dramatically minimizing tissue damage risk.
  • Mouse model studies demonstrated significantly improved gene uptake in targeted brain regions, with the team now preparing for clinical trials to treat neurological diseases.
  • This breakthrough could revolutionize treatment approaches for brain disorders including Alzheimer's disease, multiple sclerosis, and brain tumors by overcoming the blood-brain barrier challenge.

FDA Approves First Blood Test for Alzheimer's Diagnosis, Boosting Roche and BioArctic Shares

FDA ApprovalNeurologyPrecision Medicine
• The U.S. Food and Drug Administration has approved the first blood test for diagnosing Alzheimer's disease, potentially revolutionizing patient identification and treatment pathways.
• The diagnostic breakthrough is expected to drive market activity for pharmaceutical companies Roche and BioArctic, whose shares showed positive movement following the regulatory announcement.
• This blood test could significantly streamline the Alzheimer's diagnostic process, making it more accessible and efficient compared to traditional methods that rely on expensive brain scans and invasive procedures.

Johnson & Johnson and Karolinska Institutet Celebrate Decade of Collaboration Advancing Medical Innovations

OncologyNeurology
  • The ten-year partnership between Johnson & Johnson Innovative Medicine and Karolinska Institutet has yielded significant scientific advances across nine disease areas, with researchers working side by side since 2015.
  • Over 150 researchers have participated in the collaboration, producing more than 30 scientific papers and 50 presentations at international conferences, focusing on critical medical needs in oncology, neurology, immunology, and ophthalmology.
  • The partnership exemplifies successful innovation through academic-industry collaboration, with both organizations now exploring new opportunities to develop innovative therapies for complex conditions like Alzheimer's disease and cancer.

ImmuPharma Advances P140 Autoimmune Program with Novel Non-Immunosuppressive Mechanism

Neurology
  • ImmuPharma has made significant scientific breakthroughs with its P140 program, confirming a unique non-immunosuppressive mechanism of action for treating autoimmune conditions.
  • The company plans to increase P140 dosage up to 20 times higher than previously tested, following FDA guidance and pharmacokinetic studies, potentially enhancing efficacy without steroid-like side effects.
  • ImmuPharma is in active discussions with several global commercial partners while also advancing its anti-infective portfolio targeting fungal and bacterial infections.

Sleep Disturbances in ICU Patients: New Research Reveals Impact on Recovery and Outcomes

Neurology
  • New research presented at ATS 2025 reveals that sleep patterns in ICU patients may serve as important biomarkers for predicting patient outcomes, with atypical N3 sleep linked to longer ICU stays.
  • A study of ICU patients found that hypersomnia (sleeping more than 11 hours in 24 hours) was associated with higher mortality rates, longer hospital stays, and lower rates of discharge to home.
  • Over 60% of ICU survivors with acute respiratory failure experience insomnia, with pre-existing mental health conditions and neurologic causes of respiratory failure significantly increasing this risk.

SP-102 Shows Clinically Meaningful Results for Sciatica Treatment in CLEAR Trial Analysis

Neurology
  • Scilex Holding Company presented post-hoc analysis of the C.L.E.A.R. trial demonstrating clinically meaningful safety and efficacy of SP-102 (SEMDEXA™) for lumbosacral radicular pain at the ASIPP Annual Meeting.
  • The analysis showed clear separation between SP-102 and placebo in multiple endpoints, with enhanced results in the modified intent-to-treat population that received confirmed study drug.
  • SP-102's viscous gel formulation was specifically designed as a safer alternative to current off-label products that carry warnings about potentially dangerous and life-threatening adverse events.

Acadia Pharmaceuticals Wins Patent Infringement Case Against Aurobindo for Parkinson's Drug Nuplazid

Neurology
  • A U.S. District Court ruled that Aurobindo Pharma infringes on two key patent claims held by Acadia Pharmaceuticals for its Parkinson's disease drug Nuplazid.
  • The court rejected Aurobindo's arguments that Acadia's patent claims were invalid, providing significant intellectual property protection for Acadia's flagship product.
  • Following the favorable ruling, Acadia's stock soared by over 34% to a one-year high of $23.61, reflecting investor confidence in the company's market position.

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