Regeneron’s mid-stage success in postural orthostatic tachycardia syndrome has the company gearing up to launch a Phase 3 study in the first half of 2027.
A single dose of Regeneron Pharmaceuticals’ investigational monoclonal antibody reduced orthostatic heart rate at eight days for patients with a common autonomic nervous system disorder that causes abnormal heart rate elevations when standing.
Regeneron’s Phase 2 Pinnacle trial enrolled 82 patients with postural orthostatic tachycardia syndrome (POTS), a chronic condition that is also associated with lightheadedness, brain fog and fatigue. There are currently no FDA-approved treatments available to treat the disorder.
Pinnacle met its primary goal measuring the change in heart rate from lying down to standing at day eight, according to data presented Thursday at the American Autonomic Society annual meeting in San Diego.
Patients receiving a single 450-mg injection of baloncibart—also known as REGN7544—experienced a mean drop in heart rate of 11.4 beats per minute compared to placebo, while a 150-mg injection reduced heart rate by 9.5 beats per minute.
Patients receiving the high dose continued to experience a significant decrease in heartbeat at day 29, while those on the lower dose did not.
Regeneron also recorded improvements for the 450-mg arm on measures like physical functioning and daily living activities at day 29, as reported by patients. No improvements were observed for the 150-mg cohort on these measures compared to placebo.
The experimental 450-mg dose was generally well tolerated, with treatment-emergent adverse events (TEAEs) mostly mild or moderate. One severe TEAE occurred in the placebo arm, while one serious TEAE of headache occurred in the 150-mg cohort and one case of fainting was reported for the 450-mg dose. None of the TEAEs prompted discontinuation from the study.
Regeneron now plans on moving baloncibart into late-stage testing, with a trial launch slated for the first half of next year.
“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,” Boaz Hirshberg, senior vice president of clinical development and internal medicine at Regeneron, said in a release.
The cause of POTS remains unclear, though it is believed to be tied to low blood volume, according to Regeneron. Baloncibart is an NPR1-blocking monoclonal antibody designed to increase the volume of blood in circulation to reduce the increased heart rate and subsequent symptoms.
Both doses of baloncibart at day eight were associated with “biomarkers consistent with the anticipated beneficial plasma volume expansion driven by NPR1 blockade,” according to the company.
Regeneron also studied baloncibart as a treatment for sepsis-induced low blood pressure, completing a Phase 2 trial for the indication earlier this year. The company has not publicly shared results from the study, called ESTELLA, or announced further development plans in the space. The company’s pipeline doesn’t include the asset for that indication.
The drug candidate stems from Regeneron’s VelocImmune technology, which uses a genetically engineered mouse platform to develop human antibodies. The platform has played a role in the creation of several now-approved monoclonal antibodies, including Sanofi-partnered inflammatory blockbuster Dupixent.
In the most recent quarter, Dupixent continued to shine, recording a 35% year-over-year increase with $6.5 billion in global sales—the first time the drug cleared the $5 billion mark. As key patent expiries creep closer for Dupixent, Sanofi and Regeneron renewed their long-term partnership, with the latter fronting $1 billion to advance four long-acting, next-gen antibodies for immune-mediated conditions.
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