Faron Pharmaceuticals announced follow-up data from its BEXMAB Phase I/II trial and provided an update on its drug development pipeline during its Capital Markets Day 2024. The trial focuses on bexmarilimab, a novel immunotherapy, in combination with azacitidine for relapsed and refractory myelodysplastic syndrome (r/r MDS). The results indicate a high overall response rate and prolonged survival compared to existing treatments.
BEXMAB Trial: Sustained Response and Survival
The BEXMAB trial results indicated a 79% overall response rate (ORR) among r/r MDS patients treated with bexmarilimab plus azacitidine (11 out of 14 patients). This is notably higher than the 0-20% ORR reported for similar patient cohorts treated with existing alternatives. The median overall survival (mOS) remained at 13.4 months after a median follow-up of 275 days, which is significantly longer than the 5-6 months typically expected with standard care.
"We’ve continued to see extremely encouraging data from our ongoing BEXMAB trial," said Dr. Petri Bono, CMO of Faron.
Notably, three patients have proceeded to bone marrow transplant, considered the only potential curative treatment for r/r MDS. The median time on treatment in the BEXMAB trial is currently 7.9 months, exceeding initial expectations. The treatment continues to be well-tolerated based on the latest safety follow-up.
Business Development and Partnering
Following a EUR 30.7 million share offering in June 2024, Faron is focused on obtaining regulatory feedback from the FDA, completing BEXMAB Phase II enrollment, and securing a global partnership deal to fund Phase III clinical research and commercialize bexmarilimab. The company anticipates having sufficient funding until the latter half of March 2025.
In July 2024, the FDA provided positive feedback regarding the registrational study plan for bexmarilimab in relapsed and refractory high-risk MDS (HR MDS) and granted Fast Track Designation (FTD) in August 2024. Faron plans to recruit additional frontline MDS patients, aiming for a total enrollment of 32 r/r MDS patients and 20 frontline HR MDS patients, with full BEXMAB enrollment expected by January 2025.
Faron is actively engaged in discussions with potential partners to fund Phase III development and commercialization. While no exclusive agreements have been made, Faron is evaluating the received terms. The company may consider strengthening its financial position before finalizing partnering discussions to maintain flexibility and ensure high-quality Phase II BEXMAB efficacy data.
Scientific Advisory Board Renewal
Faron has renewed its Scientific Advisory Board (SAB) to align with its current drug development pipeline. The SAB comprises internationally recognized clinical scientists with expertise in anti-cancer clinical development within hematological neoplasms and solid tumors. Members include Dr. Toni Choueiri, Dr. Tom Powles, Dr. Amer Zeidan, Dr. Naval G. Daver, Dr. Mika Kontro, and Dr. Christophe Massard.
The SAB will provide guidance on translational activities and the clinical portfolio. "Now we’ve a world-leading Scientific Advisory Board supporting us, the likes of which I have never seen before, and I am very excited about what future holds," added Dr. Bono.
Solid Tumor Development
Faron is also advancing its development plan for bexmarilimab in solid tumors, exploring its potential as a macrophage reprogrammer in various anti-cancer treatments. The company is focusing on innovative approaches to improve tumor cell recognition and prevent immunosuppression.
