Positive FDA Feedback

• Faron had a formal meeting with the FDA to discuss the registrational clinical development plan for bexmarilimab in the treatment myelodysplastic syndrome (MDS).
• The FDA acknowledged the difficulties of running a randomized study with a comparator in the relapsed / refractory setting (r/r) and instead proposed that Faron conduct a confirmatory Phase III study in frontline high-risk MDS (HR MDS), that would not require a separate Phase III in r/r MDS.
• This FDA guidance is part of Project Frontrunner, an initiative intended to bring promising new cancer treatments as early as possible to a broader patient population.
• The Phase III suggested by the FDA targets a significantly larger patient population with potential for faster approval earlier than anticipated, speeding up and increasing our sales forecast for bexmarilimab.

TURKU, Finland - Faron Pharmaceuticals Ltd. (AIM: FARN, First North: FARON), a clinical-stage biopharmaceutical company pursuing a CLEVER-1 receptor targeting approach to reprogramming myeloid cells to activate anti-tumor immunity in hematological and solid tumor microenvironments, today provides information on the result of its formal Type D Scientific Advice Meeting with the USA Food and Drug Administration (the FDA) regarding the registrational study plan for its drug candidate bexmarilimab in relapsed and refractory high risk MDS (r/r MDS).

Given the previously reported promising results of treating r/r MDS using a combination of bexmarilimab + azacitidine to overcome primary or developed resistance to azacitidine, Faron had proposed to move into a randomized registrational Phase III study for the treatment of r/r MDS using bexmarilimab + azacitidine against the investigator's choice of a hypomethylating agent (HMA). Instead, given the encouraging efficacy already seen in both frontline and r/r HR MDS and the well-established safety profile of bexmarilimab, the FDA proposed that after the ongoing Phase II BEXMAB study in r/r MDS, Faron should move directly into a registrational blinded randomized frontline HR MDS study investigating bexmarilimab + azacitidine against placebo + azacitidine. The FDA noted that given the relatively modest efficacy of single agent azacitidine and the current response rates with bexmarilimab that the size of such a frontline study may not have to be substantially larger than the proposed study in the r/r setting.

Subject to continued positive results, the FDA's feedback means that a separate Phase III in r/r MDS would not be required and Faron's ongoing BEXMAB Phase II study could be the registrational trial for patients with r/r MDS, given that the benefit of bexmarilimab + azacitidine against azacitidine alone will be confirmed in an interim read-out of the response rate from a Phase III in frontline HR MDS study. Accelerated approval for frontline HR MDS would come from the response rate of this single Phase III study and the full approval from the survival read-out of the same study.

"Faron is now adjusting its development plan accordingly", says Dr. Juho Jalkanen, Chief Executive Officer of Faron. "This is very positive feedback and exceeds our expectations. The FDA's proposal significantly reduces development costs and timelines to bring bexmarilimab therapy to all HR MDS patients. This feedback underlines that the FDA sees the high unmet need in HR MDS, a condition for which new treatment options are urgently needed. The FDA's proposal has provided Faron with clear guidance on the path to approval that will confirm the highly encouraging results bexmarilimab has already obtained in overcoming resistance to azacitidine. We are extremely grateful for this feedback and will work hard to deliver on this recommendation."

"The suggested Phase III targets a significantly bigger patient population sooner than anticipated, speeding up and increasing our sales forecast for bexmarilimab. This does not significantly impact our ongoing activities and cash runway, as the Phase II in r/r MDS continues as planned. In addition, we will enroll more frontline HR MDS patients into the Phase I part of BEXMAB to better understand the effect size, which will enable us to successfully power and design the proposed frontline Phase III study. We believe we can offset this additional clinical investment through other savings, so that it will not have a significant impact on our cash runway. The only deviation from the original plan is that instead of a Phase III in r/r MDS, we will start preparations for a Phase III in frontline HR MDS, which is a remarkable achievement.", continues Dr. Jalkanen.

Faron will be hosting a virtual webinar to discuss the FDA feedback and updated clinical development plans July 15th, at 15.00 EEST / 13.00 BST.

To register for the event visit: https://faron.videosync.fi/fda-feedback-update or contact the IR team for more information at investor.relations@faron.com.

About Bexmarilimab

About Faron Pharmaceuticals Ltd.