RARE Daily

Freeline Slashes Staff, Pauses Development of Fabry Disease Candidate in Restructuring

April 5, 2023

Rare Daily Staff

Freeline Therapeutics said a part of a restructuring to extend its cash runway it will pause development of its Fabry disease experiential therapy and prioritize its gene therapy candidate for Gaucher disease type 1 as it cuts it workforce by nearly 30 percent.

The decision comes after the company assessed its strategic priorities based on its current financial position and the capital needs associated with advancing two clinical-stage programs in parallel. As the company moves to reduce operating expenses it said it will reduce its headcount to approximately 65 employees.

“While we remain encouraged by the data on FLT190 in Fabry disease, we have paused its development and are further streamlining the organization to extend our cash runway and focus on FLT201 in Gaucher disease,” Michael Parini, CEO of Freeline.

Parini said FLT201, the company experimental gene therapy for Gaucher disease, has the potential to deliver a longer-acting engineered version of the enzyme missing in people with the condition and the potential to penetrate deeper tissues, which current therapies do not adequately reach.

Gaucher disease is a rare, inherited lysosomal disorder characterized by the toxic accumulation of glucosylceramide and glucosylsphingosine in macrophages. Macrophages enlarged with these fatty substances are called Gaucher cells and amass primarily in the spleen, liver, and bone marrow. This results in a variety of potential symptoms, including grossly enlarged liver and spleen, bone issues, fatigue, low hemoglobin levels and platelet counts, and an adjusted lifetime relative risk of developing Parkinson’s disease that may be more than 20 times greater than the general population. Even on enzyme replacement therapy, the current standard of care, people with Gaucher disease typically have a shortened life expectancy and may experience debilitating symptoms that significantly reduce their quality of life.

FLT201 delivers a novel transgene that was developed by Freeline scientists. The transgene encodes for a rationally engineered variant of glucocerebrosidase (GCase), the enzyme deficient in people with Gaucher disease. The variant has demonstrated a substantially longer half-life than wildtype GCase, which may allow it to penetrate difficult-to-reach organs, including bone and lungs. FLT201 has led to high GCase expression at low doses in preclinical models, as well as increased uptake of GCase and greater clearance of harmful substrates in disease-affected tissues than the current standard of care.

Freeline is actively screening patients for dosing in its GALILEO-1 phase 1/2 clinical trial of FLT201 in Gaucher disease and now expects to report initial data, including assessments of safety and enzyme activity, from the first cohort of GALILEO-1 in the third quarter of 2023.

As of December 31, 2022, Freeline said it had unrestricted cash and cash equivalents of $47.3 million, compared to $117.7 million as of December 31, 2021. With the proceeds from the sale of the company’s German subsidiary, Freeline Therapeutics GmbH, received in February 2023 upon closing of the transaction, and the actions announced today, Freeline expects that its current level of cash and cash equivalents will enable the company to fund its planned operations into the second quarter of 2024.

Photo: Michael Parini, CEO of Freeline Therapeutics

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