Positive Phase 2 Interim Results from BEXMAB Trial

Key highlights

• Interim Phase 2 read-out from the BEXMAB Trial confirms earlier positive Phase 1 & 2 findings in MDS patients with prior HMA failure
• In Phases 1 & 2, 20 MDS patients who are refractory or relapsed on HMA (r/r MDS) and have no effective treatment options, continue to show high objective response rate (ORR) at 80%
• The BEXMAB Phase 1 & 2 MDS patients with prior HMA failure are experiencing an estimated median overall survival (mOS) of approximately 13.4 months currently, compared to the 5-6 months that would typically be expected under standard of care historically

TURKU, FINLAND - Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company focused on tackling cancers via novel immunotherapies, today provides Interim Phase 2 results of the ongoing BEXMAB trial in myelodysplastic syndrome (MDS) patients that have failed a hypomethylating agent (HMA), also known as relapsed/refractory MDS (r/r MDS). Full analysis of the data will be presented at the 66th American Society of Hematology (ASH) Annual Meeting on 9 December 2024 in San Diego, US.

The initial BEXMAB Phase 2 results have already indicated a high ORR of 79% (11/14) amongst HMA-failed MDS patients treated with a combination of bexmarilimab + azacitidine. There is now a total of 20 HMA-failed MDS patients evaluable for read-out with this novel combination. The treatment has been well tolerated, without any dose-limiting toxicity. The ORR in this otherwise untreatable population is 80% (16/20). Similar size patient cohorts treated with existing alternatives have reported 0-20% ORR, without deep and durable remissions. The estimated median overall survival of the 20 r/r MDS patients remains 13.4 months.

In summary, the updated BEXMAB results show very encouraging efficacy and robust treatment benefit for the r/r MDS patients. The detailed efficacy, safety and biomarker results of the 20 r/r MDS patients treated in the BEXMAB trial will be presented at the 66th American Society of Hematology Annual Meeting. The BEXMAB trial is continuing to enroll patients as planned with the next efficacy data readout for the fully recruited BEXMAB trial patients expected around the end of Q1 2025.

• Petri Bono, Chief Medical Officer of Faron, said: "r/r MDS is a life-threatening haematological malignancy with limited treatment options and high unmet medical need. Our updated trial results in r/r MDS further enforces bexmarilimab's ability to overcome treatment leading to clinically meaningful deep responses. We look forward to sharing the detailed results with the haematology community and discussing these data with health authorities in H1 2025."
• Juho Jalkanen, Chief Executive Officer of Faron, said: "It is remarkable seeing the ORR continuing to be so strong even as the patient population grows, as it would typically be expected to settle at a lower level. For patients, I believe these results are truly exciting as we take another step closer to providing an additional option for their poorly met treatment needs. With our repeatedly strong data, we are very much looking forward to our continuing discussions with regulatory agencies and partner candidates."

Faron will be hosting a virtual webinar to discuss the full analysis of data on Tuesday, December 10, 2024 at 16.00 EET/9am ET.

To register for the event visit: BEXMAB Study Update

The ASH Annual Meeting will take place from 7-10 December 2024, in San Diego, California and virtually. The poster will contain updated clinical data from the trial.

Poster presentation details:

Title: Encouraging Efficacy of Bexmarilimab with Azacitidine in Relapsed or Refractory MDS in Bexmab Ph1/2 Study

Session Time: Monday, 9 December 2024, 6:00 PM - 8:00 PM PST

Session Title: Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster III

Location: San Diego Convention Center, Halls G-H

Lead Authors: Dr. Mika Kontro, MD, PhD, Associate Professor at the University of Helsinki; Dr. Naval Daver, MD, Associate Professor of Leukemia at The University of Texas MD Anderson Cancer Center

Abstract Number: 4265

The full poster will be available on the Company's website at https://www.faron.com/investors once presented at ASH.

About bexmarilimab