Graviton BioScience Corporation Announces Publication of Positive Ph1b Trial Results for GV101 (TDI01) in Chronic Graft versus Host Disease in Signal Transduction and Targeted Therapy, by Beijing Tide Pharmaceutical

Rhea-AI Summary

Graviton BioScience reported peer-reviewed Phase Ib/II data for GV101, a selective ROCK2 inhibitor, in 60 patients with moderate to severe chronic graft versus host disease (cGvHD) who had failed one to five prior systemic therapies. At 24 weeks, best overall response rate was 86.2% at 400 mg and 67.9% at 200 mg, with failure‑free survival of 89.4% and 78.6%, respectively; overall survival was 96.6% (400 mg) and 100% (200 mg). Median time to first response was 30.0 days at 400 mg and 44.5 days at 200 mg, and complete responses occurred in all organ systems except lower GI. Safety findings showed mainly transient bilirubin elevations and headache, with no grade ≥3 leukopenia, thrombocytopenia, or anemia, one grade 3 neutropenia, and no CMV infections. Graviton is planning a Phase III randomized controlled trial in cGvHD and intends to advance the higher dose.

Positive

• bORR 86.2% at 400 mg and 67.9% at 200 mg at 24 weeks in heavily pretreated cGvHD
• Failure‑free survival 89.4% at 400 mg and 78.6% at 200 mg at 24 weeks; median FFS not reached
• Overall survival 96.6% at 400 mg and 100% at 200 mg; only one death, attributed to COVID‑19 pneumonia
• Rapid onset of response with median time to first response of 30.0 days (400 mg) and 44.5 days (200 mg)
• No grade ≥3 hematopoietic toxicity; no grade ≥3 leukopenia, thrombocytopenia, or anemia and only one grade 3 neutropenia
• Phase III RCT planning underway in cGvHD, with plans to carry forward the 400 mg dose

Negative

• High incidence of bilirubin elevation: transient bilirubin elevation in 81.7% of patients
• Grade ≥3 bilirubin laboratory events included unconjugated bilirubin elevation in 16.7%, total bilirubin elevation in 15.0%, and conjugated bilirubin elevation in 5.0% of patients
• Pulmonary response rate of 23.7% (95% CI: 11.4 to 40.2) based on 2014 NIH lung symptom score, lower than some other organ response rates

News Explained

The company says GV101’s 24-week response and failure-free-survival rates exceeded published six-month rates for all approved steroid-refractory cGvHD therapies, adding comparative context to the reported trial results.

Market Context

Recent insider activity was classified as Net Selling, adding a non-clinical context to this publica...

Analysis

Recent insider activity was classified as Net Selling, adding a non-clinical context to this publication. The efficacy results were favorable, while bilirubin elevations and ongoing Phase III planning remained areas to monitor.

Key Figures

bORR at 400 mg: 86.2% bORR at 200 mg: 67.9% Enrolled patients: 60 patients +5 more

8 metrics

bORR at 400 mg 86.2% 24 weeks in cGvHD patients

bORR at 200 mg 67.9% 24 weeks in cGvHD patients

Enrolled patients 60 patients Phase Ib/II trial; 30 at each dose

FFS at 400 mg 89.4% 24 weeks

FFS at 200 mg 78.6% 24 weeks

OS at 400 mg 96.6% 24 weeks

OS at 200 mg 100% 24 weeks

Transient bilirubin elevation 81.7% Adverse event occurring in at least 20% of patients

Historical Context

5 past events · Latest: Aug 13 (Positive)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Aug 13	Earnings report	Positive	-2.2%	Strategic progress and major partnering revenue accompanied second-quarter results.
Aug 11	IND clearance	Positive	+0.2%	FDA clearance enabled a randomized Phase 2 Friedreich's ataxia trial.
Aug 6	Inducement grant	Negative	+2.2%	New-employee options and RSUs created potential equity compensation dilution.
Jul 07	Strategic financing	Positive	+5.5%	Oberland Capital committed up to $400 million for development and commercialization.
May 14	Inducement grant	Negative	-5.5%	Executive hiring included options and RSUs subject to multi-year vesting.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Across recent general-news events, positive developments aligned twice and diverged once, while negative inducement-grant news aligned once and diverged once.

Key Terms

rock2 inhibitor, failure-free survival, ugt1a1, oatp1b3

4 terms

rock2 inhibitor medical

"GV101, a novel, highly selective ROCK2 inhibitor, yielded an 86.2%"

A ROCK2 inhibitor is a drug or compound that blocks the activity of the enzyme Rho-associated coiled-coil containing protein kinase 2 (ROCK2), which helps control cell shape, movement, inflammation, and tissue scarring. For investors, the term signals a specific class of therapeutic candidates and research programs: progress, trial results, or approvals for a ROCK2 inhibitor can affect a biotech company’s development prospects and commercial potential, much like a key part passing or failing quality testing on a factory line.

failure-free survival medical

"Failure-free survival (FFS) was 83.9% at 24 weeks"

Failure-free survival is a clinical-trial endpoint that measures how long a patient goes from the start of treatment until a predefined “failure” event occurs, such as disease progression, relapse, starting a new therapy, or death, depending on the study’s protocol. It matters to investors because it summarizes how durable and effective a treatment appears in delaying negative outcomes—like timing how long a car runs before any major problem—although the exact events counted vary by trial.

ugt1a1 medical

"reversible enzymatic (UGT1A1) and transporter (OATP1B3) inhibition"

UGT1A1 is a liver enzyme encoded by a gene that helps the body chemically modify and clear certain drugs and bilirubin, acting like a cleanup crew that tags substances so they can be removed. Genetic differences in UGT1A1 can change how fast people process medications, affecting drug safety, dosing and effectiveness; that makes the gene important to investors because it can drive demand for testing, influence regulatory labels, and affect a drug’s market success.

oatp1b3 medical

"reversible enzymatic (UGT1A1) and transporter (OATP1B3) inhibition"

A liver membrane protein that transports a variety of drugs and natural compounds from blood into liver cells. Like a gatekeeper controlling what enters the liver, OATP1B3 affects how quickly drugs are removed from circulation, how they are distributed in the body, and whether they interact with other medicines. Its activity can influence a drug’s dose, safety profile, and regulatory testing, so it matters for drug development and valuation of related therapies.

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• GV101, a novel, highly selective ROCK2 inhibitor, yielded an 86.2% best overall response rate (bORR) at 400 mg and a 67.9% bORR at 200 mg at 24 weeks in chronic Graft versus Host Disease (cGvHD) patients who had failed at least two prior lines of therapy.
• GV101's 24-week bORR exceeds published 6-month bORRs for all existing, approved steroid-refractory cGvHD therapies.
• Failure-free survival (FFS) was 83.9% at 24 weeks and reached 89.4% at 400 mg (78.6% at 200 mg); this FFS also exceeds published 6-month FFS probabilities for all approved steroid-refractory cGvHD therapies.
• Complete responses were observed in all but one organ system (only one participant had lower GI involvement).
• Favorable safety profile with no evidence of hematopoietic toxicity and no increased incidence of infections.
• The single Grade ≥3 adverse event occurring in ≥5% of participants was benign bilirubin elevation without transaminase elevation. This laboratory abnormality is related to reversible enzymatic (UGT1A1) and transporter (OATP1B3) inhibition by GV101.

NEW YORK, Aug. 26, 2026 /PRNewswire/ -- Graviton BioScience Corporation, a privately held, clinical-stage biotechnology company, announced the publication of peer-reviewed data from a multicenter Phase Ib/II multi-dose, open-label clinical trial evaluating the safety, tolerability, and efficacy of GV101 in patients with moderate to severe chronic Graft versus Host Disease (cGvHD). The data, published in Signal Transduction and Targeted Therapy, a Springer Nature Portfolio journal, provide efficacy and findings from the 60 enrolled patients, 30 at 200 mg and 30 at 400 mg, who had previously received one to five prior systemic therapies for cGvHD.

Key efficacy results: At 24-weeks, GV101 yielded a bORR of 86.2% (95% CI: 68.3 to 96.1) at 400 mg and 67.9% (95% CI: 47.6 to 84.1) at 200 mg; FFS was 89.4% at 400 mg and 78.6% at 200 mg. Median FFS was not met at the time of analysis. OS was 96.6% at 400 mg and 100% at 200 mg; a single participant died, attributed to COVID-19 pneumonia. Median time to first response was 30.0 days (range 28-115 days) at 400 mg and 44.5 days (range 28-284 days) at 200 mg, consistent with a rapid onset. Median duration of response was not met at the time of analysis. Complete responses were observed in all organ systems except lower GI (only 1 participant had lower GI involvement). By 24-weeks, notable organ response rates included liver (52.6%), esophagus (50.0%), and upper gastrointestinal tract (50.0%); pulmonary response rate based on the 2014 NIH Consensus Criteria lung symptom score was 23.7% (95% CI: 11.4 to 40.2).

Safety: AEs occurring in ≥20% of patients included transient bilirubin elevation (81.7%) and headache (23.3%). GV101-induced bilirubin elevations are benign, related to UGT1A1 inhibition (unconjugated) and OATP1B3 inhibition (conjugated), were generally transient and not associated with elevations in liver transaminases. Grade ≥3 TRAEs (reported in ≥5% of total patients) included only laboratory events: unconjugated bilirubin elevation (n = 10; 16.7%), total bilirubin elevation (n = 9; 15.0%), and conjugated bilirubin elevation (n = 3; 5.0%). There was no grade ≥3 leukopenia, thrombocytopenia, or anemia, and only one grade 3 neutropenia. There were no CMV infections.

Next: Graviton is currently in Phase III RCT planning for GV101 in cGvHD and plans to carry the high dose forward.

About Chronic Graft versus Host Disease

cGvHD is a rare, serious and potentially fatal complication of allogeneic stem cell transplantation that affects about 18,700 people in the US and has a global incidence of about 53,000 new cases per year. In cGvHD, donor immune cells attack healthy tissues and organs in the transplant recipient. The disease is characterized by persistent immune dysregulation, inflammation, and fibrosis that can affect multiple organ systems, significantly impair quality of life, and cause death. Despite advances in treatment, many patients experience inadequate responses, disease recurrence, and intolerable AEs. There is significant unmet need for an effective early-stage therapy that targets the key drivers of cGvHD, fibrosis and immune dysregulation, whilst preserving immune function.

About GV101

GV101 is a clinical-stage, best-in-class selective inhibitor of Rho/Rho-associated coiled-coil containing protein kinase 2 (ROCK2). ROCK2 is a key regulator of multiple cellular pathways involved in inflammation, fibrosis, metabolism, and gene expression. GV101 has been evaluated in preclinical and clinical studies and is being developed for multiple potential indications.

About Graviton BioScience Corporation

Graviton BioScience Corporation is a clinical-stage drug discovery and development company dedicated to engineering and developing best-in-class therapeutics for treating metabolic, CNS, inflammatory, fibrotic, and other disease indications. Graviton has developed a portfolio of selective ROCK2 inhibitors among other safe, innovative, and novel therapies for patients with fibrotic, autoimmune, and CNS diseases. GV101 is in clinical studies, with additional assets advancing through the pipeline. Leading the Company is Dr. Samuel Waksal, the founder and former Chairman and CEO of Kadmon Pharmaceuticals (acquired by Sanofi). Dr. Waksal is also the founder and former CEO of ImClone Systems (acquired by Eli Lilly) and a founder of MeiraGTx (NASDAQ: MGTX).

Forward-Looking Statements

This press release contains forward-looking statements regarding Graviton's development plans, regulatory strategy, and potential future clinical applications for its selective ROCK2 inhibitors. Such statements are subject to risks and uncertainties, including, but not limited to, biological, clinical, regulatory, financial, and operational risks. Actual results may differ materially.

Graviton BioScience Contact

Melanie Glickman
Senior Associate, Operations Strategy
Melanie.Glickman@gravitoncorp.com

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SOURCE Graviton BioScience Corporation

FAQ

What were the key Phase Ib/II results for GV101 in chronic graft versus host disease reported by Graviton BioScience?

GV101 achieved high response and survival rates in cGvHD. According to Graviton BioScience, 24‑week bORR reached 86.2% at 400 mg and 67.9% at 200 mg, with failure‑free survival of 89.4% and 78.6% and overall survival up to 100%.

How does the safety profile of GV101 look in the Phase Ib/II cGvHD trial mentioned alongside MeiraGTx (NASDAQ: MGTX)?

GV101 showed a generally favorable safety profile with mainly laboratory bilirubin elevations. According to Graviton BioScience, 81.7% had transient bilirubin elevation, but there was no grade ≥3 leukopenia, thrombocytopenia, or anemia, only one grade 3 neutropenia, and no CMV infections reported.

What is GV101 (TDI01) and how does it work in chronic graft versus host disease?

GV101 is a selective ROCK2 inhibitor targeting pathways in inflammation and fibrosis. According to Graviton BioScience, ROCK2 regulates multiple cellular processes, and GV101 is being developed as a best‑in‑class therapy for fibrotic, autoimmune, CNS, and other disease indications including cGvHD.

What organ response rates did GV101 achieve at 24 weeks in the Graviton BioScience cGvHD study?

GV101 produced complete responses in all organ systems except lower GI. According to Graviton BioScience, 24‑week organ response rates included liver 52.6%, esophagus 50.0%, upper GI 50.0%, and a pulmonary response rate of 23.7% based on the 2014 NIH lung symptom score.

What are the next clinical development steps for GV101 after the Phase Ib/II cGvHD results?

Graviton plans to move GV101 into Phase III testing. According to Graviton BioScience, the company is currently planning a Phase III randomized controlled trial in chronic graft versus host disease and intends to carry the higher 400 mg dose forward.

How many patients were enrolled in the GV101 Phase Ib/II trial in cGvHD highlighted near MeiraGTx (MGTX)?

The trial enrolled 60 patients with moderate to severe cGvHD. According to Graviton BioScience, 30 patients received 200 mg and 30 received 400 mg of GV101, all having previously undergone one to five prior systemic therapies for chronic graft versus host disease.

How quickly did patients respond to GV101 treatment in the Graviton BioScience cGvHD study?

Responses to GV101 occurred within weeks of treatment. According to Graviton BioScience, median time to first response was 30.0 days at the 400 mg dose and 44.5 days at the 200 mg dose, indicating relatively rapid onset of clinical effect.