Arrowhead Pharmaceuticals is intensifying its focus on Familial Chylomicronemia Syndrome (FCS), a rare genetic disorder, through a multi-pronged approach encompassing disease awareness and therapeutic development. The company recently launched the "We'll Get There Soon" campaign to raise awareness about FCS and provide hope to affected individuals. Concurrently, Arrowhead is advancing the development of Plozasiran, an investigational therapy for FCS, which has received Breakthrough Therapy designation from the FDA. These efforts are supported by a recent $500 million strategic financing agreement, positioning Arrowhead to potentially launch its first commercial product in 2025.
"We'll Get There Soon" Campaign
The "We'll Get There Soon" campaign aims to amplify the voices of individuals living with FCS and to foster a sense of hope within the rare disease community. FCS is characterized by extremely high levels of triglycerides in the blood, leading to a range of serious and potentially life-threatening complications, including pancreatitis, hepatosplenomegaly, and lipemia retinalis. The diagnostic journey for FCS patients can be lengthy and challenging, often spanning many years. Julie Klueckman, an FCS patient collaborating with Arrowhead, emphasized the difficulties in diagnosis, stating that it took a decade of research and multiple hospitalizations before she received an accurate diagnosis.
Alexandra Roeser, director of patient advocacy at Arrowhead, highlighted the company's commitment to patients, stating that Julie’s presence in their offices reminds them of why they do what they do every day and reaffirms one of Arrowhead’s core values: patients–making people’s lives better.
Plozasiran: A Potential Breakthrough Therapy
Plozasiran, Arrowhead's investigational therapy for FCS, has received Breakthrough Therapy designation from the FDA. This designation is intended to expedite the development and review of drugs for serious or life-threatening conditions where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. There are currently no FDA-approved therapies specifically for FCS, leaving physicians with limited options to manage the condition.
Chris Anzalone, PhD, president and CEO of Arrowhead, noted the significance of the Breakthrough Therapy designation, stating that results from clinical studies of investigational plozasiran have been highly encouraging and strongly supportive of further development and commercialization in multiple patient populations. He added that receiving FDA Breakthrough Therapy designation for plozasiran provides important benefits and the potential to expedite the process of getting plozasiran to the patients who need it.
Strategic Financing to Support Commercialization
Arrowhead has secured $500 million in strategic financing with Sixth Street to support the potential commercial launch of Plozasiran and to fund further growth and innovation across its pipeline. The financing agreement is structured to align outflows with future inflows, allowing Arrowhead to maintain financial flexibility during a critical period of growth. The company anticipates a potential commercial launch of Plozasiran in 2025, pending regulatory approval.
Jeff Pootoolal, partner at Sixth Street, and Michael Reslinski, managing director, expressed confidence in Arrowhead's platform technology and pipeline assets, stating that Arrowhead Pharmaceuticals has an impressive platform technology, attractive pipeline assets with promising commercial opportunities, and multiple partnerships maturing toward commercialization. They added that they are pleased to utilize our firm’s scale, expertise and flexibility to help Arrowhead meet its strategic objectives through this innovative financing solution, and we hope to continue strengthening our relationship with the company as it rapidly becomes one of the most exciting emerging commercial-stage biopharmaceutical companies.
