CuraAX aims to address debilitating blood pressure drops and cognitive symptoms of nOH

CuraSen Therapeutics, Inc., a clinical-stage biopharmaceutical company developing drug candidates with broad applicability in neurodegenerative and neuropsychiatric diseases, today announced dosing of the first patient with CuraAX (CST-3056) in a Phase 2a proof-of-concept trial in neurogenic orthostatic hypotension (nOH).

nOH is a serious condition characterized by a sudden drop in blood pressure when a person moves from sitting to standing, or from a supine position to sitting. This leads to debilitating dizziness, fainting, falls, hospitalizations and cognitive impairment due to reduced blood flow to the brain. Nearly 700,000 patients with Parkinson’s disease (PD) and related alpha-synuclein disorders, such as Dementia with Lewy Bodies (DLB), Multiple System Atrophy (MSA) and Pure Autonomic Failure (PAF), are diagnosed with nOH in the U.S. Diagnosis of nOH is associated with higher morbidity and mortality among these patients.

CuraAX, given as an oral tablet, is a selective partial α_1A-adrenergic receptor agonist that is also CNS-penetrant. It is designed to stabilize blood pressure and maintain cerebral blood flow.

“Dosing our first patient in this Phase 2a trial marks an important milestone for CuraAX and for patients living with nOH,” said Kathleen Sereda Glaub, chief executive officer, CuraSen Therapeutics. “Our Phase 1 trial in healthy volunteers showed encouraging safety, tolerability and pharmacologic activity. Unlike current treatments, which are limited by non-selective prodrug mechanisms, variable pharmacokinetics, burdensome dosing regimens and significant side effects, CuraAX has the potential to deliver stable, durable efficacy, improved safety and meaningful cognitive benefit. This represents an important best-in-class therapeutic opportunity for patients.”

The Phase 2a, proof-of-concept, dose-ranging study will evaluate safety, tolerability, and impact of CuraAX treatment on orthostatic signs and symptoms in patients with nOH and Parkinson’s disease, or Pure Autonomic Failure. Participants are blinded to study medication and will receive placebo and multiple dose levels of CST-3056 over five consecutive days. The primary endpoint is change in standing systolic blood pressure; secondary endpoints include the patient-reported outcome, known as the Orthostatic Hypotension Symptom Assessment (OHSA), which is a registrational endpoint.

The study is expected to enroll up to 12 patients and is taking place at sites in New Jersey and New York, and at the Vanderbilt Autonomic Dysfunction Center. The principal investigator for the study is Italo Biaggioni, MD, professor of medicine and pharmacology, and an internationally recognized nOH expert.

IND-enabling and Phase 1 studies for CuraAX were supported with funding from the Alzheimer’s Drug Discovery Foundation (ADDF).

In addition to CuraAX, CuraSen’s development programs include CuraCN (CST-103/CST-107) and CuraXN (CST-2032/CST-107), both oral, CNS-penetrant β₂-adrenergic receptor agonists combined with nadolol, a peripherally restricted beta-blocker. Designed to restore adrenergic function that is lost early in neurodegenerative diseases, both drug candidates have shown cognitive benefit in Phase 2 proof-of-concept studies in Parkinson’s and Alzheimer’s disease, with additional trials planned in progressive supranuclear palsy (PSP) and Alzheimer’s.

About CuraSen Therapeutics

CuraSen is focused on the development of new treatments for neurodegenerative and neuropsychiatric diseases targeting mechanisms with broad applicability, including neurogenic orthostatic hypotension, Parkinson’s and Alzheimer’s disease, and certain orphan indications. CuraSen’s drugs are small molecules, given as oral tablets, designed to directly activate certain receptor populations in the brain and periphery to compensate for decline of the adrenergic system driven by aging and neurologic disease. For more information, please visit www.curasen.com.

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The Phase 2a, proof-of-concept, dose-ranging study will evaluate safety, tolerability, and impact of CuraAX treatment on orthostatic signs and symptoms in patients with nOH and Parkinson’s disease, or Pure Autonomic Failure