Myelin Protective Small Molecule Lucid-MS Shows Promising Early-Stage Data in Healthy Volunteers

In a phase 1 trial of healthy volunteers, treatment with investigational Lucid-21-302 (Quantum Biopharma), a first-in-class compound targeting demyelination, was shown to be safe and well-tolerated at daily doses of 150 and 300 mg p.o. All told, the study investigators concluded that this agent represents an important drug candidate to advance into a phase 2 trial of patients with multiple sclerosis (MS), a demyelinating disorder.¹

Lucid-21-302, also called Lucid-MS, is a non-covalent inhibitor of protein arginine deiminase 2 (PAD2), an enzyme expressed in the central nervous system that catalyzes the citrullination of myelin proteins. The phase 1 trial was a multiple-ascending dose (MAD), randomized, double-blind, placebo-controlled trial testing Lucid-21-302 vs placebo, given orally for 7 days. In the study, healthy volunteers aged 18-60 years received the investigational agent at doses of 150 mg (Cohort 1) and 300 mg p.o. (Cohort 2).

Led by Andrzej Chruschinski, MD, PhD, vice-president, scientific and clinical affairs at Quantum Biopharma, the trial reported 38 treatment emergent adverse events (TEAEs) by 12 of the 40 participants who received Lucid-21-302 or placebo. Overall, most of the TEAEs were mild in nature, there were no severe TEAEs, and there were no reports of serious AEs as well. Notably, none of the TEAEs reported in Lucid-21-302-treated patients were considered related to the study drug.

Presented at the 2026 Americas Committee for Treatment & Research in Multiple Sclerosis (ACTRIMS) Forum, held February 5-7 in San Diego, the phase 1 trial also examined pharmacokinetic (PK) properties of the study drug. Results showed that exposure increased in a dose-dependent manner, with C_max rising from 1.5 to 2.1 µg/mL at 150 mg to 2.8 to 3.3 µg/mL at 300 mg, and Area under the curve (AUC)_0-Last increasing from 4.2 to 5.3 to 9.8 to 11.6 hr·µg/mL, respectively.

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The study authors also provided a comparison of these data to previous nonclinical studies. Overall, the bioavailability for Lucid-21-302 was 79% in rats, and not currently available in humans yet. The AUC_0-Last of Lucid-21-302 was 8.5 hr·µg/mL in mice, while 3-month chronic toxicity studies identified no observed adverse effects level (NOAEL) exposures of 250 hr·µg/mL at 300 mg/kg in rats and 158 hr·µg/mL at 150 mg/kg in dogs.

Following these data, the drug is expected to be moved into a phase 2 trial. In mid-2025, Quantum Biopharma, the drug’s manufacturer, signed a manufacturing agreement with a “leading” contract development and manufacturing organization to produce the drug. At the time of the agreement, Chruscinski said this is “an important step in the clinical development of Lucid-MS (Lucid-21-302).”²

Months later, in December 2025, Quantum announced completion of dosing in 180-day repeated dose oral toxicity and toxicokinetic studies for the investigational agent. At the time, the company noted that these studies will support the investigational new drug application with the FDA, including the design of a phase 2 trial testing Lucid-MS in patients with MS.³

To date, there are no FDA-approved therapies for MS that have remyelination or demyelination properties. Recently, in late 2025, the clinical community was met with negative data from a promising phase 2 study, dubbed VISTA (NCT06083753), that tested a remyelinating drug in patients with relapsing-remitting MS. Although the proof-of-concept study failed, Continuem, the drug manufacturers for PIPE-307, are planning to take a more detailed look at the exploratory end points of the study.⁴

Click here for more 2026 ACTRIMS Forum coverage.

REFERENCES
1. Chruscinski A, Joshi A, Stys P, Antel J, Kotra L. P131 / P131 - Results of a Phase 1 Multiple Ascending Dose Trial of Lucid-21-302 (Lucid-MS) in Healthy Volunteers: A Novel Myelin Protective Small Molecule to Prevent Disease Progression in Multiple Sclerosis. Presented at: 2026 ACTRIMS Forum; February 5-7; San Diego, CA. ABSTRACT P131.
2. Quantum BioPharma Signs Agreement to Manufacture Oral Drug Formulation of its Potential Breakthrough Drug Lucid-21-302 (Lucid-MS) for Use in its Upcoming Phase 2 Multiple Sclerosis Trial. News release. Quantum BioPharma. August 11, 2025. Accessed February 5, 2026. https://feeds.issuerdirect.com/news-release.html?newsid=8238648156541045&symbol=QNTM
3. Quantum Biopharma Announces Completion of Dosing in 180-Day Repeated Dose Oral Toxicity and Toxicokinetic Studies for Lucid-21-302 (Lucid-MS). News release. Quantum BioPharma. December 23, 2025. Accessed February 5, 2026. https://finance.yahoo.com/news/quantum-biopharma-announces-completion-dosing-130000424.html
4. Contineum Therapeutics Reports Topline Data From Its Phase 2 PIPE-307 VISTA Trial for the Treatment of Relapsing-Remitting Multiple Sclerosis (RRMS). News release. November 20, 2025. Accessed February 5, 2026. https://ir.contineum-tx.com/news-releases/news-release-details/contineum-therapeutics-reports-topline-data-its-phase-2-pipe-307