The Axon – Issue #14, July 2025

Happy August, and welcome to The Axon!

As the summer winds down, neurology continues to heat up. In this month’s roundup, we highlight the latest clinical milestones, FDA approvals, and expert perspectives driving the field forward. From exciting updates in neurodegeneration and autoimmune disease to advancements in digital diagnostics and sleep science, NeurologyLive has you covered. Whether you’re catching up on new research or diving into commentary from thought leaders, this edition is packed with content to keep your practice current and connected.

FDA News

August brought another wave of impactful FDA activity across the neurology space, continuing to shape the treatment landscape for both rare and prevalent conditions. Below, catch up on some of the most notable agency decisions, including new approvals in Parkinson disease and autoimmune conditions such as chronic inflammatory demyelinating polyneuropathy. In addition, the team covered some notable industry news, such as the updated labeling of donanemab, an approved treatment for early-stage Alzheimer disease, and the concerns around Sarepta's gene therapy program following another patient death.

July 9 - FDA Approves Updated Label for Alzheimer Therapy Donanemab to Lower ARIA-E Risk

• Eli Lilly and Company announced that the FDA approved an updated label for donanemab (Kisunla), its amyloid-targeting therapy for early-stage Alzheimer disease, to include a new titration-based dosing schedule. Originally approved in 2024 for patients with mild dementia and confirmed amyloid pathology, donanemab was initially dosed at 700 mg IV every 4 weeks for three doses, then 1400 mg every 4 weeks. The updated regimen introduces a more gradual titration, aiming to reduce the risk of amyloid-related imaging abnormalities (ARIA) while maintaining efficacy in lowering amyloid plaques and phosphorylated tau 217 (p-tau).

July 9 - Exablate Neuro Gains FDA Approval for Staged Bilateral Focused Ultrasound in Advanced Parkinson Disease

• The FDA approved INSIGHTEC undefined ’s Exablate Neuro platform for staged bilateral pallidothalamic tractotomy in patients with advanced Parkinson disease, covering both initial and follow-up procedures to enable comprehensive bilateral treatment. This expanded indication provides a new option for patients with severe motor symptoms who have not responded to prior therapies. Exablate Neuro delivers MRI-guided focused ultrasound to the pallidothalamic tract through an incisionless, hardware-free procedure with a lower risk profile than traditional surgery. Insightec announced plans for a limited launch at select centers in 2025, alongside efforts to support reimbursement and broaden access.

July 18 - FDA Shuts Down Sarepta's Distribution of Gene Therapy Elevidys Following Patient Deaths

• Following the deaths of three patients, the FDA requested Sarepta Therapeutics to suspend distribution of Elevidys, its approved gene therapy for Duchenne muscular dystrophy (DMD), and pause clinical trials involving its gene therapy candidates. The most recent death involved a 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3 enrolled in a phase 1 study of SRP-9004 (patidistrogene bexoparvovec), who died from liver failure—also the cause of the two prior deaths. In response, the FDA revoked Sarepta’s platform technology designation, citing insufficient safety evidence for its AAVrh74 platform, and requested a voluntary halt to Elevidys shipments, which the company declined.

July 21 - FDA Clears HyHub and HyHub Duo Devices for Easier Administration of Immune Globulin Infusion, Hyqvia

• The FDA granted 510(k) clearance to Takeda ’s HyHub and HyHub Duo devices for patients aged 17 and older, enabling needle-free transfer of Hyqvia—an approved treatment for chronic inflammatory demyelinating polyneuropathy (CIDP)—from vials in home or clinical settings. These devices function as docking stations for Hyqvia’s dual vial units, which include immune globulin and recombinant human hyaluronidase, and are designed to reduce preparation steps by up to 50% compared with pooling bags. In addition to streamlining setup and minimizing ancillary supplies, Takeda noted that a dedicated carrier bag will be available to support mobility during administration. The devices are expected to launch in the U.S. in the second half of fiscal year 2025.

Mind Moments

This month, NeurologyLive released three new episodes of our biweekly podcast, Mind Moments, spotlighting timely conversations with leading experts across the neurology field. Among them was a special FDA-focused edition featuring migraine specialist Stewart Tepper, MD, who broke down the recent approvals of CT-132 and STS101 for acute migraine treatment. New episodes drop every other Friday at 7 AM ET, with special editions covering breaking news and major meeting highlights. To catch up on past episodes or subscribe, visit neurologylive.com/mindmoments.

Rewiring Stroke Recovery: What the Glymphatic System Can Teach Us

Our first episode of the month featured Joyce Lee-Iannotti , MD, PhD, division chief of both the Stroke Center and the Comprehensive Sleep Center at Barrow Neurological Institute , who discussed her presentation from the 2025 SLEEP Annual Meeting focused on the glymphatic system’s role in stroke. In the episode, she outlines the science behind this brain-cleansing pathway, its dependence on sleep quality, and how its dysfunction may worsen outcomes in both ischemic and hemorrhagic stroke. Lee-Iannotti also details practical, evidence-backed strategies for optimizing glymphatic clearance—from sleep posture and sleep apnea treatment to emerging pharmacologic options like orexin antagonists. In addition, she highlights major gaps in clinician awareness and offers calls to action for improving sleep assessments in stroke care.

Special Episode: Breaking Down the FDA’s Latest Migraine Therapy Approvals with Stewart Tepper, MD

Stewart Tepper, MD, professor of neurology at the Geisel School of Medicine at Dartmouth , unpacks the FDA’s recent approvals of STS101 (Atzumi; Satsuma Pharmaceuticals, Inc. ) and CT-132 (Click Therapeutics) for the acute and preventive treatment of migraine. In this two-part episode, he explores the pharmacologic characteristics and clinical role of DHE—the active agent in STS101—and how advances in delivery methods could broaden its use in acute migraine management. Tepper also reviews the supporting data for CT-132, the first FDA-approved prescription digital therapeutic for migraine, and considers its potential impact on both patients and providers. Throughout, he emphasizes the importance of clinician education in navigating an evolving treatment landscape, where emerging therapies promise improved outcomes, fewer side effects, and greater accessibility.

Overviewing Donanemab’s New Dosing Regimen: Lowering ARIA Risk in Alzheimer Care

In our third episode, John Sims, MD, head of medical development for donanemab at Eli Lilly and Company , joins NeurologyLive to discuss the FDA’s recently updated label for donanemab (Kisunla), an anti-amyloid therapy for Alzheimer disease. He highlights key safety findings from the TRAILBLAZER-ALZ-6 study (NCT05738486) that supported the approval of a new titration-based dosing schedule designed to reduce the risk of amyloid-related imaging abnormalities (ARIA). Sims also offers practical insights for clinicians treating early-stage AD, including guidance on genotype-based risk stratification, ARIA monitoring, and patient counseling. He concludes by reflecting on donanemab’s clinical impact and its role in shaping the next chapter of anti-amyloid therapy and Alzheimer disease management.

Clinical Trial in Focus

Each month, NeurologyLive® will spotlight an innovative and clinically relevant trial in a long-form feature designed for the neurology community. These in-depth articles delve into the study’s design, its clinical significance, and the drug under investigation, while also exploring the supporting clinical trial data that has shaped its development.

This month's trial in focus is the AHEAD 3-45 Study (NCT04468659), a groundbreaking investigation of lecanemab (Leqembi; Eisai US ) in cognitively unimpaired individuals at risk for Alzheimer disease (AD). As the first secondary prevention trial to use plasma-based biomarkers to streamline participant screening, AHEAD 3-45 is exploring whether lecanemab can slow tau accumulation and delay cognitive decline during the preclinical stage of AD. Led by principal investigator Michael S. Rafii, MD, PhD, professor of neurology at the University of Southern California , the trial consists of two parallel studies: the phase 2 A3 trial in patients with intermediate amyloid burden and the phase 3 A45 trial in those with elevated amyloid. Both trials employ amyloid PET–guided dosing strategies tailored to disease stage, reflecting a precision medicine approach that aims to maximize efficacy while minimizing exposure in asymptomatic individuals.

To read the July iteration of Clinical Trial in Focus, head here: Designing a Prevention Trial for Alzheimer Disease Using Lecanemab: The AHEAD 3-45 Study

Features & Clinician Submissions

Eyeing Neurology Drug Development: 2H Trial Readouts to Watch for in 2025

With the second half of 2026 approaching, attention across the neurology community is turning to a new round of anticipated clinical trial readouts that may shape the next chapter of treatment innovation. These upcoming data span a diverse mix of conditions and therapeutic approaches—from disease-modifying agents in neurodegeneration to cutting-edge strategies in rare and refractory disorders. In this month’s feature, NeurologyLive® highlights the most closely watched trials slated for readout in late 2026, offering insight into their design, endpoints, and the potential impact these therapies may have on clinical decision-making and patient outcomes.

To read more about the most notable 2H trials to watch, head here: https://www.neurologylive.com/view/eyeing-neurology-drug-development-2h-trials-watch-for-in-2025

Collateral Damage to Patients From Formulary Restrictions in Multiple Sclerosis: Perspectives from an MS Specialist, a Patient, and a Neurologist in Training

We introduced a new clinician specialist to our close circle, Dr. William Kilgo, MD, FAAN of the University of South Alabama ! In his first feature with NeurologyLive, Kilgo, a multiple sclerosis specialist, highlights the devastating impact of insurance-based treatment denials on patients with chronic neurologic disease. Through the story of Dana Campbell, a U.S. Navy veteran whose stable MS condition was upended after a change in insurance resulted in a forced switch from effective therapy, Dr. Kilgo illustrates how opaque step therapy policies can lead to avoidable relapses, hospitalizations, and long-term harm. He also shares the perspectives of a neurology resident on the growing burden of administrative barriers, calling for reform to protect both patients and the clinicians who care for them.

To read Collateral Damage to Patients From Formulary Restrictions in Multiple Sclerosis: Perspectives from an MS Specialist, a Patient, and a Neurologist in Training, head here: https://www.neurologylive.com/view/collateral-damage-patients-from-formulary-restrictions-multiple-sclerosis-ms-perspectives-from-ms-specialist-patient-neurologist-in-training

The Transition of Care from Pediatric to Adult Neurology: Leaving the Cocoon

Our second newly introduced clinician author is Susan Duberstein , associate director of child neurology at the Jacobi Medical Center. This thoughtful feature by Duberstein explores the often-overlooked challenges that young patients with chronic neurological conditions face when transitioning from pediatric to adult care. Drawing from clinical experience, Dr. Duberstein outlines the critical components of a successful handoff—including healthcare continuity, early patient education, psychosocial support, and multidisciplinary collaboration. Through real-world insights and practical guidance, the piece highlights the need for proactive, individualized planning to ensure patients maintain access to the care and services they need during this pivotal stage of life.

To read The Transition of Care from Pediatric to Adult Neurology: Leaving the Cocoon, head here: https://www.neurologylive.com/view/the-transition-of-care-from-pediatric-to-adult-neurology-leaving-the-cocoon

SLEEP 2025: Analyzing High-Level, Late-Breaking Data

In this special video series from NeurologyLive®, sleep experts Eric Olson, MD, and Anita Shelgikar, MD—president-elect of the AASM—break down key late-breaking data from the 2025 SLEEP Annual Meeting. The panel covers emerging insights in narcolepsy and hypersomnia, from high-dose efficacy and cardiovascular safety of low-sodium oxybate to real-world prescribing trends and social determinants of health. They also spotlight investigational therapies like TAK-861 and ALKS 2680, offering timely context on the future of care for central disorders of hypersomnolence.

Topic presentations covered:

• Impact of Switching from High- to Low-Sodium Oxybate on Ambulatory Blood Pressure in Patients with Narcolepsy - LBA 1647
• Effectiveness and Safety of Low-Sodium Oxybate Dosages Greater Than 9 Grams in Study Participants With Narcolepsy - LBA 1648
• Social Determinants of Health and Clinical Burden in Narcolepsy: A Retrospective Cohort Analysis - LBA 1656
• Vibrance-3: Study Design and Methods for a Phase 2, Randomized, Placebo Controlled, Parallel-Group Study Evaluating the Safety and Efficacy of ALKS 2680 in Patients With Idiopathic Hypersomnia - LBA 1661
• Accelerometry Assessment of Factors Related to how TAK-861 Affects Disrupted Sleep in People with Narcolepsy Type 1 - LBA 1670
• Narcolepsy Treatment Trends and Change in Alerting Agent Use After Low-Sodium Oxybate Initiation - LBA 1674

For the entire series, head here: https://www.neurologylive.com/special-report/sleep-2025-analyzing-high-level-late-breaking-data