The neurology sector advanced across five distinct fronts today, spanning rare neurodevelopmental disorders, neurotologic disease, neuromuscular care, neuroimmunology access, and biomarker-driven Alzheimer’s diagnostics. While each update reflects progress within its own domain, together they signal a tightening convergence across clinical innovation, regulatory acceleration, commercial readiness, and early disease interception. The through-line is clear, neurology stakeholders are investing earlier, aiming higher, and executing faster.
Korea expands access to Ultomiris for generalized myasthenia gravis
Access progress also advanced in neuroimmunology as Korea’s National Health Insurance approved reimbursement for AstraZeneca’s Ultomiris (ravulizumab) for adults with generalized myasthenia gravis (gMG) who are AChR-antibody positive. The criteria remain narrow, MGFA II–IV, MG-ADL ≥6, prior myasthenic crisis requiring PLEX or IVIg, and failure of corticosteroids plus two non-steroidal immunosuppressants, but the decision marks a significant expansion for a population limited by chronic steroid toxicity and suboptimal long-term control. Evidence from the CHAMPION-MG Phase 3 trial underpins the decision. Ultomiris achieved a 3.1-point MG-ADL improvement versus 1.4 points with placebo (P<0.001), with durability demonstrated in long-term extension data. Clinicians welcomed the move but underscored that broader criteria are needed to ensure meaningful real-world patient impact.
Mirum’s Phase 2 BLOOM trial in Fragile X syndrome
Mirum Pharmaceuticals opened the week by enrolling the first participant in its BLOOM Phase 2 clinical study evaluating MRM-3379, a selective PDE4D inhibitor designed to enhance cAMP signaling in Fragile X syndrome (FXS). With FXS representing the most common inherited cause of intellectual disability and autism spectrum disorder, and with no approved therapies, Mirum’s move is strategically significant.
The Phase 2 design incorporates a three-dose randomized cohort for males aged 16–45 and an open-label adolescent arm, signaling Mirum’s intent to interrogate both safety and early cognitive signals across age segments. The key secondary endpoint is the NIH Toolbox Crystallized Cognition Composite, reflects a deliberate orientation toward measurable cognitive improvement rather than behavioural proxies.
Preclinical data demonstrating gains across cognitive and behavioral domains create a foundation for this proof-of-concept effort, and community advocacy leaders have already expressed strong engagement. With topline results expected in 2027, BLOOM stands as one of the more closely watched mid-stage neurodevelopmental trials, marking Mirum’s expansion beyond its hepatology core into neuroscience-driven rare disease.
Breakthrough Therapy Designation propels SPI-1005 forward in Meniere’s disease
Regulatory momentum accelerated in neurotology, where Sound Pharmaceuticals secured FDA Breakthrough Therapy Designation for SPI-1005, an oral formulation of ebselen. This marks the first-ever BTD for Meniere’s disease and the first BTD globally for sensorineural hearing loss of any aetiology.
The designation was supported by the STOPMD-3 Phase 3 trial, which demonstrated clinically meaningful improvements in pure-tone audiometry and words-in-noise discrimination, an accomplishment few auditory therapies have achieved. A second Phase 3 is currently accruing expanded long-term safety data to support chronic dosing.
BTD not only validates SPI-1005’s mechanism, which targets glutathione peroxidase pathways to repair injured inner-ear cells, but also elevates Sound Pharmaceuticals as a frontrunner in a field long constrained by a lack of approved pharmacologic treatments. With sensorineural hearing loss representing one of the most prevalent neurologic disorders of aging, the potential market impact reaches far beyond Meniere’s disease itself.
Upsher-Smith launches KYMBEE to strengthen DMD corticosteroid access
On the commercial front, Upsher-Smith announced the U.S. launch of KYMBEE (deflazacort) Tablets for Duchenne muscular dystrophy (DMD). The launch is deliberately engineered around access stabilization in a market often challenged by insurance variability, prior authorization complexity, and high caregiver burden.
Distributed through Orsini Specialty Pharmacy and backed by the company’s Promise of Support Program, KYMBEE arrives with payer-oriented scaffolding designed to minimize interruptions in therapy. The support model includes co-pay assistance, insurance navigation, and structured pathways to maintain continuity even in the face of coverage disruption.
By offering a U.S.-manufactured deflazacort across four dosage strengths, Upsher-Smith is positioning KYMBEE as a reliability-driven alternative within the corticosteroid backbone of DMD care. In a landscape where gene therapies and exon-skipping strategies dominate headlines, corticosteroids remain the foundation for maintaining function, delaying progression, and managing the multisystem complications inherent to Duchenne. KYMBEE’s launch reinforces the necessity of strengthening that foundation.
Circular Genomics raises $15M to accelerate circRNA Alzheimer’s diagnostics
The final development of the day came not from the clinic but from the neurodiagnostics investment front. Circular Genomics closed a $15 million Series A to advance its proprietary circular RNA (circRNA) biomarker platform aimed at early Alzheimer’s detection.
Unlike amyloid or tau-focused assays, circRNAs provide a more holistic readout of the molecular dysfunction underlying neurodegeneration, spanning neuroinflammation, oxidative stress, synaptic remodelling, and pathway-level disruptions. Early data benchmarked against PET and CSF modalities show strong predictive performance, including identification of pre-symptomatic individuals at risk of progressing to symptomatic Alzheimer’s.
With disease-modifying therapies now approved, demand for scalable, primary-care–deployable diagnostic tools is intensifying. Circular Genomics’ platform is engineered not just for detection but also for patient stratification, longitudinal monitoring, and therapeutic decision support, placing it squarely within the emerging precision-neurology infrastructure.