Faron Pharmaceuticals (FARN) investor relations material

Pipeline overview and program updates

• BEXERA, a randomized Phase IIb study in first-line high-risk MDS, was moving into site activation; BEXMAB enrollment was closed and follow-up continued.

• BLAZE combines bexmarilimab with nivolumab in PD-1-refractory metastatic NSCLC and melanoma; two patients had started treatment, with first results expected in H2 2027.

• BEXAR combines bexmarilimab with first-line doxorubicin in soft-tissue sarcoma; its first cohort was full and the second opened, with results targeted for 2027.

• Additional investigator-led studies include bexmarilimab plus oral decitabine/cedazuridine in r/r MDS and a post-transplant MRD-positive AML study; protocols were being finalized or submitted.

• Management expected data during 2027 from four, potentially five, indications; further investigator-led proposals exceeded current sponsorship capacity.

Clinical trial data and development milestones

• Frontline BEXMAB showed 45% CR by IWG 2006 and 41% CR+CReq by IWG 2023; median CR duration was 16.1 months, 57% of transfusion-dependent patients became independent, and 67% of CR patients were MRD-negative.

• In 33 r/r MDS patients, BEXMAB showed 64% overall response, 12% CR+PR, 18% bridged to transplant and estimated median OS of 14.5 months; its small, single-arm design limits comparative conclusions.

• BEXERA is a randomized, double-blind, placebo-controlled Phase 2b in ~90 treatment-naïve HR-MDS patients, comparing bexmarilimab 1 or 3 mg/kg plus azacitidine with placebo plus azacitidine; primary objectives include dose selection and CR+CR-equivalent.

• BEXERA was planned across 35 U.S., European and U.K. sites; first treatment was expected in Q4 2026, with dose selection and a readout targeted for 2027.

• MATINS showed 25%–35% benefit in some solid-tumor cohorts; CLEVER-1-positive macrophages emerged as a candidate response biomarker requiring validation in BEXERA and BLAZE.

R&D strategy and innovation priorities

• Bexmarilimab binds CLEVER-1 to reprogram immunosuppressive myeloid cells, increase antigen presentation and effector T-cell activity, and make azacitidine-resistant blast cells more sensitive.

• Patient and bone-marrow analyses indicated immune and hematopoietic reprogramming, reduced suppressive monocyte-lineage cells, progenitor activation, increased lymphocyte abundance and function, and greater target engagement in patients achieving CR.

• BEXERA incorporates lessons from prior HMA-combination failures, including realistic effect assumptions, placebo control, stratification by TP53, blast percentage and IPSS-M, dose comparison, and adaptive sample-size planning.

• Development prioritizes HR-MDS through Phase 2b and Phase 3, alongside phased proof-of-concept studies in solid and hematologic tumors, including sarcoma and PD-1-refractory melanoma and lung cancer.