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FDA Grants Fast Track Designation to AVB-114 for Crohn Disease Perianal Fistulas
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Key Takeaways
- AVB-114 is a cell therapy designed to improve healing of Crohn's perianal fistulas, which are often resistant to standard treatments.
- The phase 1 STOMP-I trial showed AVB-114's safety and efficacy, with significant clinical healing and no treatment-related serious adverse events.
The designation was granted after positive results from the phase 1 STOMP-I clinical trial (NCT01915927).
The FDA granted a fast track designation to AVB-114 (Avobis Bio) for the treatment of Crohn perianal fistulas. The designation was a result of the results from the phase 1 STOMP-I (NCT01915927) clinical trial.1,2
Image credit: Jo Panuwat D | stock.adobe.com
AVB-114 is an implantable cell therapy that aims to overcome the impaired healing of perianal fistulas resulting from Crohn disease. Perianal fistulas are painful tunneling wounds connecting the rectum or anus to the skin. About 2 out of 3 patients do not experience durable fistula healing with the current standard of care, resulting in elevated and prolonged suffering because of disease progression and complications resulting from surgeries. AVB-114 consists of living cells and a plug-shaped bioabsorbable material that combine to generate tissue and produce healing signals in a local therapeutic treatment.1
In the prospective, single-arm, phase 1 STOMP-I clinical trial (NCT01915927), AVB-114 demonstrated its ability to safely and effectively deliver cell-based therapy for patients with single-tract fistulizing perianal Crohn disease. A total of 20 patients aged 18 to 65 years (median: 36 years) who failed conventional therapy were enrolled. Additionally, patients were enrolled if they met the following criteria: US residents; diagnosed with Crohn disease with single or multiple draining complex perianal fistulae for at least 3 months despite standard therapy; a colonoscopy within the last 12 months to rule out malignant or premalignant condition; have no contraindications to MR evaluations (eg, pacemaker or magnetically active metal fragments); will comply with protocol; and/or failed standard medical therapy, including anti-tumor necrosis factor (TNF) agents. Additionally, patients were permitted to take concurrent therapies with corticosteroids, 5-aminosalicylic acid drugs, thiopurines, methotrexate, antibiotics, and anti-TNF therapy.2,3
Patients received AVB-114 and were then followed up for 8 visits through a 12-month duration. Safety was the trial’s primary end point, and clinical hearing and MRI response were secondary end points.2,3
Complete clinical healing was observed in 14 of 18 patients at the 6-month period and 13 of 17 at the 12-month period. Additionally, MRI response was observed in 12 of 18 patients at 6 months. Through the 12-month duration, none of the patients experienced a serious adverse event (AE) related to investigational treatment; however, 7 serious AEs and 22 AEs were reported by 4 and 12 patients, respectively, but these were considered unrelated to treatment.3 Currently, AVB-114 is undergoing evaluation in the phase 2 STOMP-II (NCT04847739)4 trial, with primary end point results expected in mid-2025, according to a news release.1
"We are grateful for the FDA's recognition that perianal fistulas are a serious condition for many patients living with Crohn disease and that AVB-114 has the potential to bring meaningful improvement to their lives," Tiffany Brown, PhD, CEO of Avobis Bio, said in a news release. "Receiving [a] fast track designation will enable us to work closely with FDA in reviewing the STOMP-II primary end point results later this year and aligning on the most expeditious commercialization pathway for AVB-114."1
