A wave of activity across neurodegeneration, epilepsy, schizophrenia, Parkinson’s research, and rare pediatric oncology underscores how capital, mechanistic innovation, and maturing clinical programs are tightening the path from discovery to late-stage evidence. Today’s developments span targeted protein degradation, glutamatergic modulation, neuromodulation in generalized epilepsy, RNAi delivery across the blood–brain barrier, serotonergic approaches in Dravet syndrome, lymphatic drug delivery, and open-science drug discovery.
Zai Lab Secures NRDL Renewals for VYVGART, NUZYRA, and ZEJULA
China’s 2025 NRDL update renewed reimbursement for VYVGART in AChR+ generalized myasthenia gravis, NUZYRA (oral) in CABP and ABSSSI, and ZEJULA across first-line and recurrent platinum-sensitive ovarian cancer. With six products now on NRDL, Zai Lab continues to expand affordability and access across immunology, infectious diseases, and oncology, reinforcing its commercial scale in Greater China.
Convergen Raises $10M to Advance TrimTAC Platform in Neurodegeneration
Convergen closed a $10M seed financing led by Qiming Venture Partners to accelerate its TrimTAC platform, next-generation bifunctional degraders leveraging TRIM21 to selectively clear multimeric pathogenic aggregates, a central driver of multiple neurodegenerative diseases. The platform addresses key limitations of classical degraders, including dependence on a narrow set of E3 ligases and limited activity against multimeric proteins. The company’s founders, including noted degradation expert Dr. Ting Han, position TrimTAC as a potentially category-defining TPD engine for aggregate-driven disorders.
MJFF Launches First Validation Projects Under T2T, Targeting High-Priority Parkinson’s Biology
The Michael J. Fox Foundation initiated the first wave of target-validation programs under its multi-year T2T Initiative after reviewing more than 280 nominated targets and narrowing the field to 21 priorities. Initial funded teams will advance targets such as NOD2, OGA, and lysosomal pathways (TRPML1, TMEM175, ATP13A2). MJFF has committed $7.5M to date and simultaneously released ~59 public target profiles via the new T2T Target Explorer platform, creating a transparent evidence map to accelerate investment and translational decisions across the field.
Newron Initiates U.S. Arm of ENIGMA-TRS 2, a Global Phase III Trial of Evenamide in TRS
Newron has opened U.S. enrollment for ENIGMA-TRS 2, a 12-week, randomized Phase III study evaluating evenamide as add-on therapy in treatment-resistant schizophrenia. Evenamide, a first-in-class glutamate modulator targeting aberrant VGSC-driven glutamate release, previously delivered clinically meaningful responses in inadequate and treatment-resistant patients, with strong tolerability. ENIGMA-TRS 1 (one-year study) and ENIGMA-TRS 2 together aim to support the first approved add-on therapy for TRS, with topline data expected in 2026.
Harmony Biosciences Shows 50% Median Motor Seizure Reduction in EPX-100 Dravet OLE
Initial open-label extension data from Harmony’s Phase 3 ARGUS trial revealed ~50% median reduction in countable motor seizure frequency over 28 days, with half of the participants achieving ≥50% reduction. EPX-100 was well tolerated over multi-year exposure, with minimal GI effects and no new safety signals. Topline ARGUS results are expected in 2026.
Merz Therapeutics Previews >20 Data Readouts for TOXINS 2026, Spanning Spasticity and Dystonia
Merz Therapeutics will present more than 20 abstracts at TOXINS 2026, including pediatric lower-limb spasticity results (ELLIE), real-world durability data in cervical dystonia (RELY-CD), a Phase 2 design in chronic peripheral neuropathic pain (PaiNT), and systematic immunogenicity evaluations across major botulinum toxin products. The breadth of data signals ongoing investment to reinforce leadership in neurotoxin science and multimodal spasticity management.
Arrowhead Doses First Subjects in Phase 1/2a ARO-MAPT Study, Bringing RNAi Across the BBB
Arrowhead has initiated dosing in the first-in-human trial of ARO-MAPT, an siRNA therapeutic designed to silence the MAPT gene for Alzheimer’s disease and other tauopathies. Using a new delivery system capable of crossing the blood–brain barrier after subcutaneous administration, the program demonstrated deep and durable tau knockdown in primates. The study will assess both healthy volunteers and early-stage AD patients, with initial data in 2H 2026.
Arrowhead Doses First Subjects in Phase 1/2a ARO-MAPT Study, Bringing RNAi Across the BBB
Arrowhead has initiated dosing in the first-in-human trial of ARO-MAPT, an siRNA therapeutic designed to silence the MAPT gene for Alzheimer’s disease and other tauopathies. Using a new delivery system capable of crossing the blood–brain barrier after subcutaneous administration, the program demonstrated deep and durable tau knockdown in primates. The study will assess both healthy volunteers and early-stage AD patients, with initial data in 2H 2026.
Agora Open Science Trust Nominates M4K2009 as Lead Candidate for DIPG
In a landmark for open-science drug development, Agora nominated ALK2 inhibitor M4K2009 as its lead development candidate for Diffuse Intrinsic Pontine Glioma following extensive global collaboration. The molecule demonstrated strong potency, selectivity, and brain penetration, enabling progression to IND-enabling studies. The program supported by Conscience’s DMOS initiative, OICR, and multiple clinical and academic partners illustrates how non-proprietary, open-data models can advance therapeutics in market-failure conditions such as rare pediatric oncology.
Seaport Therapeutics Extends Glyph Validation with CBD Prodrug Showing 3× Dose Efficiency
Preclinical data presented at AES 2025 show that GlyphCBD, Seaport’s lymphatic-targeted prodrug of cannabidiol, achieved seizure protection at a median effective CBD dose three times lower than standard oral CBD. The findings highlight the platform’s ability to bypass first-pass metabolism and reduce hepatotoxicity risks, addressing a well-recognized limitation of currently approved CBD therapies for Dravet and Lennox-Gastaut syndromes.
NeuroPace Reports 77% Median Reduction in GTC Seizures at 18 Months in IGE
Preliminary 18-month data from the NAUTILUS trial demonstrated a 77% median reduction in generalized tonic-clonic seizures in patients with drug-resistant idiopathic generalized epilepsy treated with the RNS System. The reduction exceeds that observed in the company’s original pivotal focal epilepsy study and strongly supports NeuroPace’s planned PMA supplement submission for IGE by year-end. Clinical Global Impression of Change exceeded 80% across physicians and patients.
Related: Last week’s NeuroPulse Daily recap offers further background on emerging CNS trends.
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