Data presented today for the first time at the American Autonomic Society (AAS) annual meeting

POTS (postural orthostatic tachycardia syndrome) is a debilitating condition characterized by a marked increase in heart rate upon standing and associated orthostatic symptoms, with no FDA-approved treatments available

A single dose of baloncibart reduced the exaggerated heart rate response to standing and improved orthostatic symptoms through four weeks; biomarker changes were consistent with plasma volume expansion, the intended effect of NPR1 blockade

Potentially pivotal Phase 3 trial planned for first half of 2027

TARRYTOWN, N.Y., Nov. 12, 2026 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced results from the Phase 2 PINNACLE trial in adults with postural orthostatic tachycardia syndrome (POTS) evaluating baloncibart, a novel, investigational, first-in-class monoclonal antibody that blocks natriuretic peptide receptor 1 (NPR1). The data were presented for the first time at the American Autonomic Society’s (AAS) 37th International Symposium on the Autonomic Nervous System in San Diego, CA.

POTS is a chronic disorder in which heart rate rises abnormally upon standing. Patients may experience palpitations, dizziness, fatigue, exercise intolerance, cognitive difficulties, gastrointestinal symptoms and chronic pain. Although historically underdiagnosed, recent estimates suggest it affects between 500,000 and several million people in the U.S., and the vast majority are young women. While the cause of POTS is unknown, it is believed to be associated with low blood volume. Baloncibart is designed to increase the volume of blood in circulation in an effort to reduce the increase in heart rate and symptoms that occur in people with POTS.

“POTS causes debilitating symptoms that can profoundly disrupt daily life, and patients have no FDA-approved treatment options. In the trial, a single injection of baloncibart 450 mg reduced the exaggerated heart rate response to standing and improved orthostatic symptoms through four weeks,” said Satish R. Raj, M.D., Professor of Cardiac Science at the University of Calgary’s Cumming School of Medicine, and investigator in the trial. “The results are consistent with the rationale for NPR1 blockade in POTS, with biomarker changes pointing to an increase in blood volume alongside lower heart rate and better symptoms.”

The randomized, double-blind, Phase 2 PINNACLE trial evaluated a single subcutaneous dose of baloncibart 150 mg (n=27) or 450 mg (n=28) compared to placebo (n=27) in patients with POTS. The primary endpoint was the change in heart rate from lying down to standing (DeltaHR) at day 8; no formal hypothesis testing was prespecified. Improvements were observed with baloncibart on DeltaHR and other key measures of the disease (i.e., symptoms and function) as assessed by the Living With POTS Questionnaire (LWPQ). The LWPQ is a novel patient-reported outcome tool to assess POTS symptoms and function in clinical trials, which is being developed by Regeneron in accordance with the FDA patient-focused drug development guidance.

Baloncibart 450 mg reduced DeltaHR versus placebo at day 8 (primary endpoint) with a least-squares (LS) mean difference of -11.4 beats per minute (bpm; nominal p=0.0007), and at day 29 (secondary endpoint) with an LS mean difference of -7.4 bpm (nominal p=0.0124). Baloncibart 150 mg reduced DeltaHR versus placebo at day 8 (LS mean difference: -9.5 bpm; nominal p=0.0045), but the effect was not maintained at day 29.

Baloncibart 450 mg improved orthostatic symptoms, physical functioning, and activities of daily living versus placebo at day 29, as measured by the LWPQ. Domain scores range from 0 to 100, with lower scores indicating fewer symptoms or less impairment. The LS mean differences in change from baseline for each domain were as follows:

• Orthostatic symptoms (baseline mean: 45.5): -12.9 points (nominal p=0.0096)
• Physical functioning (baseline mean: 55.1): -13.8 points (nominal p=0.0073)
• Activities of daily living (baseline mean: 48.7): -8.7 points (nominal p=0.1035)
• Overall across the three domains, there was no obvious improvement with baloncibart 150 mg compared to placebo
  
  

Additionally at day 8, both doses of baloncibart led to changes in biomarkers consistent with the anticipated beneficial plasma volume expansion driven by NPR1 blockade, including an increase in NT-proBNP and reductions in standing norepinephrine and hemoglobin. Average standing systolic blood pressure increased for both doses, with observed mean changes from baseline at day 29 of 8.7 mmHg to a mean of 118.8 mmHg for baloncibart 450 mg and 5.4 mmHg to a mean of 114.0 mmHg for baloncibart 150 mg.

In the trial, single doses of baloncibart up to 450 mg were generally well tolerated. Treatment-emergent adverse events (TEAEs) were mostly mild or moderate and occurred in 13 patients (48.1%) receiving placebo, 18 patients (66.7%) receiving baloncibart 150 mg, and 19 (67.9%) receiving baloncibart 450 mg; one severe TEAE occurred in the trial in the placebo group. TEAEs that occurred in ≥10% of patients in any treatment group, including placebo, baloncibart 150 mg and baloncibart 450 mg, were: injection site reaction (14.8%, 3.7%, and 17.9%), headache (0%, 11.1%, and 10.7%), syncope (11.1%, 14.8%, and 3.6%), heavy menstrual bleeding (0%, 11.1%, and 0%), and nasopharyngitis (11.1%, 0%, and 0%). Two serious TEAEs were reported: one case of headache with baloncibart 150 mg and one case of syncope with baloncibart 450 mg. No TEAE led to discontinuation.

“POTS is a condition that disproportionately impacts younger women and has been under-recognized and under-served for too long, which is why it was critical to incorporate the patient experience in our trial design and measure symptoms that reflect how patients actually feel and function,” said Boaz Hirshberg, M.D., Senior Vice President, Clinical Development, Internal Medicine at Regeneron. “These initial results show the potential of baloncibart for people living with POTS and we look forward to advancing this novel therapy into a Phase 3 clinical trial in 2027.”

The safety and efficacy of baloncibart have not been evaluated by any regulatory authority.

About Regeneron’s VelocImmune Technology
Regeneron’s VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce fully optimized human antibodies. When Regeneron’s Co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor, Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite^® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved fully human monoclonal antibodies. This includes Dupixent^® (dupilumab), Libtayo^® (cemiplimab-rwlc), Praluent^® (alirocumab), Kevzara^® (sarilumab), Evkeeza^® (evinacumab-dgnb), Inmazeb^® (atoltivimab, maftivimab and odesivimab-ebgn), Veopoz^® (pozelimab-bbfg), and Pasatru^® (garetosmab-grts). In addition, REGEN-COV^® (casirivimab and imdevimab) had been authorized by the FDA during the COVID-19 pandemic until 2024. A total of 11 antibody and bispecific products have been approved or authorized worldwide that were invented or derived using VelocImmune technologies.

About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases.

Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite^®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center^® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.

For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X.

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