Familial Chylomicronemia Syndrome is a rare condition that can have deadly symptoms.
Arrowhead Pharmaceuticals announced a new campaign titled “We’ll Get There Soon,” which is focused on providing awareness for Familial Chylomicronemia Syndrome (FCS).1 The condition is a rare disease that can lead to serious and life threatening symptoms.
In a press release, Arrowhead included a statement from Julie Klueckman, a woman living with FCS. In the release, she said, “My journey to diagnosis was a difficult one that ended after a decade-long diagnostic process and countless hours of research, learning about FCS, and finally presenting the idea to my doctor. Despite my eventful history with this condition, including countless hospitalizations, multiple surgeries, a radical hysterectomy, relocating and moving my family to be closer to my trusted doctors, I remain optimistic about the future for FCS patients.” Excited by what the future holds, Julie is also working with the Arrowhead team contributing patient insights to support and inform efforts for people with FCS because she believes, “Hope is on the horizon.”
Alexendra Roeser, director of patient advocacy at Arrowhead, also said, “We are humbled to play a small role in amplifying the voices of those affected by FCS and bringing them hope. Julie’s presence in our offices reminds us of why we do what we do every day and reaffirms one of Arrowhead’s core values: patients–making people’s lives better.”
In September, Arrowhead announced that FDA had granted breakthrough therapy designation for Plozasiran, a treatment for FCS.2
In a press release issued at the time, Chris Anzalone, PhD, president and CEO of Arrowhead, said, “There are currently no FDA-approved therapies to specifically treat FCS, leaving physicians with very few options to help their patients. Results from clinical studies of investigational plozasiran have been highly encouraging and strongly supportive of further development and commercialization in multiple patient populations. 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.”
In August, the company announced that it had secured $500 million in strategic financing with Sixth Street.3 At the time, Anzalone said, “This deal and its favorable structure and economics allow us to confidently build our commercial capabilities to support our first commercial launch in 2025, provided we receive regulatory approval for plozasiran in familial chylomicronemia syndrome. This transaction is non-dilutive and has an attractive repayment structure, which largely aligns outflows with future inflows, allowing us to keep more cash in the business at a critical time of growth. In addition, because of the flexible repayment mechanics during the seven-year term, it has important risk sharing features. Not only does the facility allow us to support plozasiran as we move toward a commercial launch, it also expands our ability to fund growth and innovation across our pipeline.”
Jeff Pootoolal, partner at Sixth Street and Michael Reslinski, managing director added, “Arrowhead Pharmaceuticals has an impressive platform technology, attractive pipeline assets with promising commercial opportunities, and multiple partnerships maturing toward commercialization. We 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.”
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