BioMarin, Cyclerion slash workforce in the name of strategic focus

Published: Oct 07, 2022

By Tristan Manalac

BioSpace

Rare disease biotech BioMarin Pharmaceuticals Inc. announced Friday it was shedding around 4% of its global workforce, or around 120 jobs, in an effort to improve its operational efficiency.

This strategic reorganization comes ahead of the anticipated U.S. launch of BioMarin’s Roctavian (valoctocogene roxaparvovec), its gene therapy hopeful for hemophilia A. The downsizing will result in annual savings of around $50 million starting next year, which the company plans to reinvest into its early-stage clinical development programs.

“We are continuing to invest in our current clinical development portfolio, as well as bolster investment in our next generation of commercial products,” Debra Charlesworth, vice president of corporate communications at BioMarin, told BioSpace.

She added, “Our early-stage pipeline has a rich abundance of candidate therapies in cardiovascular, CNS, hematologic & metabolic, and musculoskeletal therapeutic areas.”

The savings will also be used to support product launches and fund BioMarin’s operations, allowing it to boost profitability and expand margins.

According to the California company, affected employees were notified of their termination this week. The transition is set to be complete within the year, with most of the layoffs coming from BioMarin’s U.S. operations. The company expects to incur $20 to $25 million in severance payments and termination benefits.

Roctavian delivers a functional copy of the gene that enables the body to produce Factor VIII, a protein crucial for blood clotting. Compared with currently available therapies and prophylaxes, which require continued dosing, Roctavian is a one-time infusion and substantially eases treatment burden.

BioMarin’s gene therapy was approved by the European Commission in August but remains under review by the FDA. The company resubmitted its Biologics License Application late last month, banking on robust new data to bolster its regulatory chances.

In the first quarter of this year, BioMarin posted $519 million in total earnings, which the company labeled “record first quarter revenues” at the time. Nearly $20 million of these proceeds came from Voxzogo (vosoritide), which in November last year became the first FDA-approved drug for kids with the rare genetic disorder achondroplasia. According to BioMarin, savings from Friday’s restructuring will also support Voxzogo product launches.

Cyclerion Down to 16 Employees

On Thursday, small Massachusetts-based biotech Cyclerion Therapeutics also revealed it was undergoing strategic restructuring, downsizing by 45% and leaving only 16 full-time employees onboard. 

The lay-offs will cost Cyclerion some $1.9 million in one-time employee payments but will help it save $4.1 million yearly. The company plans on channeling this money into CY6463, its potentially disease-modifying candidate for serious conditions of the central nervous system. 

Cyclerion is looking to develop CY6463 for the treatment of patients with rare genetic mitochondrial diseases.

The candidate has recently emerged with positive data from an open-label clinical study of patients with mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), a complex orphan disease that affects the CNS and other organ systems. MELAS patients treated with CY6463 saw improvements in disease markers, including inflammation, functional brain connectivity and mitochondrial function.

Due to this promising performance and an encouraging safety profile, Cyclerion has decided to make CY6463 its top priority and focus its expertise and resources on advancing the candidate. The company is set to meet with the FDA for guidance on the CY6463 development program.

Aside from downsizing, Cyclerion is also looking to forge strategic partnerships and out-license its other assets to support its pivot to mitochondrial diseases. One of its compounds, praliciguat, has already been licensed to fellow Massachusetts biopharma Akebia Therapeutics and is currently being assessed in renal diseases.

Source: BioSpace