Trial testing safety, effectiveness of add-on PH treatment kicks off
First patients dosed in small study of ROC-101 plus Winrevair, other drugs
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Building on positive data from an earlier study, Allrock Bio has dosed the first patients in a small clinical trial testing ROC-101, its oral treatment candidate for pulmonary hypertension (PH).
The open-label Phase 2a ROCSTAR study (NCT07175038) is recruiting patients across the U.S., Canada, and Europe — including as many as 10 individuals with PH associated with interstitial lung disease (PH-ILD). The trial is primarily enrolling participants, as many as 30, with pulmonary arterial hypertension (PAH). Eligible participants must be in World Health Organization functional class II or III.
Supported by a $50 million fundraising round, the study is evaluating the safety, tolerability, and effectiveness of ROC-101 when added to standard-of-care therapies, including in a group of patients receiving Winrevair (sotatercept-csrk).
“We have successfully dosed our first patients with ROC-101, including in combination with a stable dose of [Winrevair],” Kate Steiner, MBBS, vice president and head of clinical development at Allrock, said in a company press release noting the milestone.
“We believe ROC-101, with its oral, once-daily dosing, has differentiated potential to address underlying disease mechanisms, further reduce pulmonary vascular resistance and improve functional outcomes for patients on top of existing therapies,” Steiner said.
ROC-101 aims to block 2 enzymes involved in inflammation and scarring
PH is marked by abnormally high pressure in the blood vessels that carry blood from the heart to the lungs. In PAH, this is driven by narrowing and remodeling of the pulmonary arteries. In PH-ILD, the condition develops in the setting of interstitial lung disease, which is marked by lung scarring and inflammation.
ROC-101 is designed to inhibit both ROCK1 and ROCK2, two enzymes involved in processes of inflammation, cell growth, and scarring, which are believed to contribute to PH. According to Allrock, blocking both targets may complement the way existing PH therapies work.
The study’s primary goal is to assess changes in the pulmonary vascular resistance, a measure of how hard the heart must work to pump blood through the lungs, from its start, or baseline, to week 24 (about six months). This will be evaluated by right heart catheterization, the main test for PH. In that procedure, a thin, flexible tube is inserted into a blood vessel and guided to the right side of the heart and pulmonary artery, where it directly measures pressure.
Key secondary goals include changes in six-minute walk distance, a measure of exercise capacity, NT-proBNP, a biomarker of heart strain, and WHO functional class. Other blood flow measures will also be evaluated.
The trial follows data from a Phase 1 study in healthy volunteers in which ROC-101 showed a favorable safety profile, with pharmacological properties supporting once-daily dosing. The company also reported no evidence of low blood pressure in that trial, helping support the decision to move the candidate into Phase 2a testing.
