New partnership aims to speed development of CF therapy
Thermo Fisher to give Arcturus support for planned Phase 3 trial of ARCT-032
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Arcturus Therapeutics is partnering with Thermo Fisher Scientific to support the development of ARCT-032, Arcturus’ experimental inhaled therapy for cystic fibrosis (CF).
Under the agreement, Thermo Fisher will provide Arcturus with manufacturing and clinical trial support for a planned Phase 3 study testing ARCT-032. And if the inhaled therapy is ultimately approved for commercial use, Thermo Fisher will also support commercialization activities, including having exclusive rights to manufacture a commercial supply of the drug.
“Thermo Fisher brings together manufacturing, clinical research, and commercialization expertise to help customers simplify development, reduce operational complexity, and accelerate the delivery of innovative therapies to patients. Our collaboration with Arcturus demonstrates the value of this integrated model,” Mike Shafer, executive vice president and president of biopharma services at Thermo Fisher Scientific, said in a press release from Arcturus.
ARCT-032 designed to deliver mRNA encoding healthy CFTR protein
CF is caused by mutations in the gene that encodes the CFTR protein. Without a functional version of this protein, the body produces unusually thick and sticky mucus, which builds up in the lungs and other organs to drive most disease symptoms. A class of medicines called CFTR modulators can boost the activity of the defective protein, but modulators only work in people with certain CF-causing mutations, and some patients can’t take modulators due to side effects or accessibility constraints.
When genes are read to make protein, the genetic code is copied from a cell’s DNA into a temporary molecule called messenger RNA (mRNA), which is then used as the template for protein production. ARCT-032 is designed to deliver mRNA encoding healthy CFTR protein to cells in the lungs, allowing lung cells to produce a functional version of the protein that can help normalize mucus production. Since this strategy doesn’t rely on a patient’s existing CFTR protein, it is expected to work equally well regardless of each individual’s specific underlying mutations.
ARCT-032 is currently being tested in a Phase 2 clinical trial called LunairCF (NCT06747858). Early data suggested that the inhaled therapy has an acceptable safety profile and hinted that it may help reduce the buildup of lung mucus.
The Phase 2 study is still recruiting participants at several sites across the U.S., and is open to adults with CF who are not taking CFTR modulators. All participants in the Phase 2 study will be treated with ARCT-032 at various doses for one to three months, with the main goal of evaluating the experimental therapy’s safety.
Assuming that the Phase 2 study yields positive results, Arcturus is then planning to launch a Phase 3 trial to further test the therapy. Phase 3 trials generally include more participants and are designed to prove whether or not a therapy is effective, in addition to collecting safety data.
Under the new agreement, Thermo Fisher will be providing Arcturus with manufacturing, clinical research, and related services for the planned Phase 3 trial, as well as potentially supporting commercialization efforts.
“We are pleased to collaborate with Thermo Fisher to utilize its impressive CDMO [contract development and manufacturing organization] capabilities, global infrastructure, and proven ability to support complex mRNA programs from Phase 3 through potential commercialization,” said Joseph Payne, president and CEO of Arcturus. “Their capabilities are aligned with Arcturus’ goal of efficiently advancing our cystic fibrosis program into Phase 3 and preparing for potential commercial supply.”
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