Promising execution in the clinic with programs in hemophilia B, Fabry disease and Gaucher disease; potential to deliver transformative therapies driven by Freeline’s differentiated platform
Data anticipated
Promising execution in the clinic with programs in hemophilia B, Fabry disease and Gaucher disease; potential to deliver transformative therapies driven by Freeline’s differentiated platform
Data anticipated across all programs by mid-2022
FLT180a B-LIEVE trial for hemophilia B launched in Q4 2021, one quarter ahead of latest guidance
FLT201 Phase 1/2 trial for Gaucher disease Type 1 on track for trial site initiation by year-end 2021; dosing in Q1 2022FLT190 MARVEL-1 trial for Fabry disease progressing in the clinic;
next patient expected to be dosed in H1 2022
Company continues to prioritize investments on the highest value activities; hones corporate focus with deprioritization of hemophilia A program and proposed 25% workforce reduction
LONDON, Dec. 13, 2021 (GLOBE NEWSWIRE) — Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the “Company” or “Freeline”), a clinical-stage biotechnology company developing transformative AAV-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced its 2022 corporate priorities and provided updated guidance.
“Today we are announcing a tightening of our focus to deliver on the promise of Freeline to advance therapies that have the highest potential to transform the lives of patients suffering from debilitating diseases. Leveraging our highly potent capsid and unique platform capabilities, we believe we can deliver best-in-class or first-in-class treatments, positioning Freeline for long-term success as a leader in gene therapy,” said Michael Parini, Chief Executive Officer of Freeline. “2021 was a transformational year at Freeline as we made progress across our three strategic priorities – execution, prioritization and innovation – to lay the foundation for success in 2022 and beyond.”
“Starting with execution, we are on track to initiate our Phase 1/2 dose-finding study of FLT201 in Gaucher disease by year-end, in line with prior guidance, and look forward to dosing the first patient in the first quarter of 2022. Our compelling preclinical data suggest that the potency of our AAVS3 capsid combined with our proprietary protein engineering positions FLT201 as a potentially transformative therapy for Gaucher disease Type 1, a serious, life-long disease with significant unmet medical need. FLT201 will be our third program in the clinic and is poised to be the first AAV gene therapy program for Gaucher disease Type 1 to enter the clinic.”
“We continue to make strong progress in hemophilia B, initiating our Phase 1/2 dose-confirmation B-LIEVE study one quarter ahead of latest guidance. We are rapidly advancing FLT180a for hemophilia B, having already identified a sufficient number of patients to fully enroll the trial via the ECLIPSE run-in study. We look forward to dosing patients in the first quarter of 2022. The most recent data we released at the American Society of Hematology (ASH) Annual Meeting strengthens our belief that FLT180a has the potential to be a best-in-class gene therapy treatment for hemophilia B.”
“We also will advance FLT190 for Fabry disease throughout 2022, with additional patients to be dosed and data expected through the year. Based on the encouraging data from the first two patients dosed in our ongoing MARVEL-1 trial, we continue to believe that FLT190 has the potential to deliver high levels of α-Gal A expression to keep Fabry patients off enzyme replacement therapy at relatively low doses compared to other treatments. We expect to dose the third patient in our ongoing clinical trial in the first half of 2022.”
Mr. Parini continued, “Successful execution of these programs requires focused and strategic allocation of resources. To drive continued advancement of FLT180a, FLT190 and FLT201, all of which have the potential to be first or best-in-class, Freeline has undertaken a detailed strategic review of our programs and operations. Following this review, the Company has decided to halt further development of our preclinical work for FLT210 in hemophilia A, given the additional work needed. Freeline will seek to explore potential third-party partnership opportunities to progress this important work, which will allow us to focus our time and resources on the programs most likely to return transformative benefits for patients.
“Today we also announced that, in an effort to streamline our operations, the Company is implementing an approximate 25% reduction in the size of our workforce. This change follows our program optimization and will enable us to better focus our time and resources on the highest value activities. We are confident these steps will create a more efficient and sustainable company, positioning Freeline for long-term success across its clinical programs and platform technology. I would like to express my sincere gratitude to our departing colleagues who are impacted by this decision. Their dedication and contributions have been invaluable to advancing our mission, and we will honor their work through continued focus on delivering our three core programs to the patients waiting for functional cures for their diseases.”
“We are excited about our programs and what’s to come in 2022. These decisions to focus our efforts will result in a reduction in operating expenses that extends our cash runway by an additional quarter to the second quarter of 2023 and provides a solid financial foundation to support the future success of our programs and Company. We are ready, with increased focus and commitment, to deliver the next generation of innovations for patients,” said Mr. Parini.
Recent Updates, Key 2022 Priorities and Program Guidance
Gaucher Disease Type 1
- Phase 1/2 dose-finding trial of FLT201 on track to be initiated in Q4 2021.
- The Company expects to dose two patients in the first dose cohort in the first half of 2022, with initial data expected in Q3 2022. Data from additional patients expected prior to year-end 2022.
Hemophilia B
- Reported long-term follow-up data from its Phase 1/2 B-AMAZE dose-finding trial of FLT180a for the treatment of hemophilia B at the 63rd American Society of Hematology (ASH) Annual Meeting. Results suggest that a dose of 7.7e11 vg/kg, coupled with a short course of prophylactic immune management, has the potential to achieve durable factor IX (FIX) activity in the normal range (50-150%).
- Initiated the B-LIEVE study, a Phase 1/2 dose-confirmation study of FLT180a manufactured at commercial scale and using a starting dose of 7.7e11 vg/kg and a short course of prophylactic immune management, in Q4 2021.
- The Company expects to complete dosing of the first dose cohort in the B-LIEVE study and report data from the first dose cohort in the first half of 2022.
Fabry Disease
- Reported data from the lowest dose cohort in MARVEL-1 trial of FLT190 for Fabry disease, demonstrating durable α-Gal A expression over two years in the first patient and promising activity with near normal α-Gal A levels in the second patient, who remains off enzyme replacement therapy more than 16 weeks post-dosing.
- The Company expects to dose the third patient at the at 7.5e11 vg/kg dose level in H1 2022, with updated data from the first two patients dosed, and initial data from the third, in the first half of 2022. Escalation to next dose level is expected in H2 2022, along with updated data.
Corporate
The company intends to reduce expenses and extend its existing cash runway through a prioritization of its programs and reduction in workforce:
- The Company expects to incur charges relating to its proposed workforce reduction between $2.1 and $2.6 million in the fourth quarter of 2021, which is expected to result in $1.3 to $1.6 million of cash expenditures in the first quarter of 2022 and $0.8 to $1.0 million of cash expenditures in the second quarter of 2022.
- The Company estimates the proposed reduction in force will decrease aggregate cash expenditures by between $4.7 and $5.1 million in 2022 and between $7.0 and $7.6 million in 2023.
- As a result of these initiatives, the Company expects its current level of cash and cash equivalents will enable the Company to fund its operating expenses into the second quarter of 2023.
Recent Comments