AllRock Bio Presents Phase 1 Data for Lead Program, ROC-101, at the ERS Congress

ROC-101 exhibits favorable pharmacokinetics for once-daily dosing

Data support the potential therapeutic efficacy of ROC-101; justify further development via a Phase 2, proof-of-concept trial in patients with PAH or ILD-PH slated to begin Q4 2025

NATICK, Mass., Sept. 29, 2025 /PRNewswire/ -- AllRock Bio, Inc., a clinical stage biotechnology company focused on advancing impactful therapies for cardiopulmonary and fibrotic diseases, today announced encouraging Phase 1 data for its first-in-class oral pan-rho-associated protein kinase (ROCK) inhibitor, ROC-101, at the European Respiratory Society (ERS) Congress, held September 27 to October 1, 2025, in Amsterdam.

The first-in-human study met its primary endpoint, demonstrating that ROC-101 is safe, well-tolerated, does not cause hypotension, and achieves target engagement—supporting its continued evaluation in patients with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (ILD-PH).

Rho kinases play an integral role in multiple aspects of fibrotic disease pathogenesis. ROCK1 and ROCK2 are differentially upregulated in a variety of disorders; therefore, inhibition of both ROCK2 and ROCK1 is key to addressing the inflammatory, proliferative, and fibrotic disease-associated remodeling in PAH and ILD-PH, two serious conditions with approximate five-year survival rates of 57% and 38%, respectively.

"The results from our Phase 1 trial confirm that ROC-101 is a therapeutically promising compound," said William Marshall, MD, CMO of AllRock. "ROC-101 was specifically engineered to target ROCK1 and ROCK2, which play nonredundant roles in driving the pathology of PAH and ILD-PH. Our goal is to create a more effective treatment option that improves upon the clinical outcomes of existing therapies."

ROC-101's mechanism of action is complementary to other disease-modifying therapies in the pulmonary hypertension space. AllRock Bio's Phase 2a ROCSTAR clinical trial will evaluate ROC-101 in combination with standard of care in PAH and ILD-PH patients and is expected to initiate in late 2025.

"By designing a forward-thinking clinical trial, we're generating the robust data that clinicians and patients need," said Catherine Pearce, DHSc, MBA, AllRock's CEO and co-founder. "This approach allows us to address the significant unmet needs that persist and ultimately provides a viable way to improve patient outcomes."

The Phase 1 study—a randomized, double-blind, single and multiple ascending dose trial—was conducted in 92 healthy volunteers. Key findings include:

• Strong safety and tolerability: ROC-101 was safe and well-tolerated at all doses tested, up to 40 mg once daily. The study found no evidence of hypotension at any dose.
  • Among those receiving ROC-101, the most common treatment emergent adverse events (TEAEs) were headache (n=2) and constipation (n=3).
  • There were no serious AEs, discontinuations due to AEs, or clinically significant treatment-emergent alterations in the electrical activity of the heart as recorded by an electrocardiogram.
• Favorable pharmacokinetics (PK): PK data showed dose-proportional increases in drug exposure. ROC-101 was rapidly absorbed, and its half-life supports once-daily dosing.
• Effective target engagement: Pharmacodynamic (PD) and biomarker data demonstrated a dose-dependent decrease in ROCK1 and ROCK2 activity.
• Superior exposure compared to other pan-ROCK inhibitor: Modeling showed ROC-101 achieved >20 times exposure than that of fasudil (a pan-ROCK inhibitor approved for subarachnoid hemorrhage in Japan and China), at the fasudil dose linked to significantly improved survival in PAH/PH patients in previous academic clinical trials.

About ROC-101
ROC-101 is a first-in-class, oral pan-ROCK inhibitor in clinical development for the treatment of pulmonary arterial hypertension (PAH) and pulmonary hypertension with interstitial lung disease (ILD-PH). With favorable pharmacokinetics that support once-daily dosing, ROC-101 is designed to selectively and potently inhibit both ROCK1 and ROCK2. By targeting the distinct, non-redundant roles of these kinases that are central to the inflammatory, proliferative, and fibrotic remodeling seen in cardiopulmonary diseases, ROC-101 has the potential to address significant unmet needs. Its mechanism of action is complementary to other approved and investigational therapies for pulmonary hypertension. In a Phase 1 study in healthy volunteers, ROC-101 was found to be safe, well tolerated, and showed no evidence of hypotension.

About PAH
Pulmonary arterial hypertension (PAH) is a severe condition characterized by elevated blood pressure in the pulmonary arteries, impacting both the lungs and the right side of the heart. In PAH, the small pulmonary vessels undergo progressive narrowing, leading to elevated pulmonary arterial pressure. This heightened pressure impedes blood flow through the lungs, necessitating increased effort from the right ventricle to maintain adequate circulation, ultimately placing significant strain on the heart. Despite 16 approved drugs—including four vasodilator classes—there continues to exist an unmet need for disease-modifying treatments in the current landscape.

About ILD-PH
Pulmonary hypertension (PH) associated with Interstitial Lung Disease (ILD-PH) is a distinct form of PH where the blood pressure in the lungs increases in the setting of ILD. ILD encompasses a broad group of diseases, such as idiopathic pulmonary fibrosis, that impair the structure of the lungs, making it progressively harder to breathe. ILD-PH is associated with increased mortality and morbidity with worsening symptoms over time, and very limited treatment options are available.

About AllRock Bio
AllRock Bio is a clinical stage biotechnology company focused on the development of transformative therapies for cardiopulmonary diseases. The company's lead candidate, ROC-101, is a first-in-class, oral pan-ROCK inhibitor being developed to address the urgent unmet need in pulmonary arterial hypertension (PAH) and other life-threatening fibrotic diseases. With a seasoned leadership team and a commitment to addressing unmet need, AllRock is dedicated to bringing a new class of disease-modifying therapies to patients. For more information, please visit www.allrockbio.com. Follow us on Linkedin.

Media contact:
[email protected]

SOURCE AllRock Bio