Cystic Fibrosis-related ProQR Names New Therapy Development Chief

David M. Rodman is the new chief development strategy officer at ProQR Therapeutics, a leader in RNA therapies for cystic fibrosis (CF) and other diseases.

Rodman, a medical doctor, will be tasked with advancing ProQR’s treatment candidates.

Dr. David M. Rodman. Photo credit: ProQR

A key therapy the company is working on is QR-010. The RNA-based compound is intended to correct the ΔF508 mutation of CFTR, the gene that is defective in CF. The drug repairs messenger RNA (mRNA), the copy of a gene that creates a corresponding protein.

QR-010 is administered by nebulizer, a device that produces a fine mist that is inhaled into the lungs.

A Fast Track Designation in the United States is helping ProQR develop the therapy.

The first exploratory proof of concept Phase 1 study (NCT02564354) of QR-010 involved 18 CF patients with the F508del mutation. The study evaluated whether QR-010 could increase CFTR functioning. Researchers looked at patients’ nasal potential difference (NPD), a test that measures chloride transport through the CFTR protein.

In October 2016, the company announced that NPD tests suggested that QR-010 had restored CFTR activity.

QR-010 is being evaluated in a Phase 1b trial (NCT02532764) in CF patients with two copies of the F508del mutation. Results for multiple ascending doses are expected by mid-year.

“At ProQR we are just beginning to capitalize on the power of RNA-based therapeutics. We believe RNA therapeutics offers a powerful therapeutic approach to severe genetic disease. We believe the RNA approach has advantages over other approaches, and we are excited to fully explore the possibilities for patients. By adding Dave to our leadership, we will be able to strengthen our portfolio and strategically build our pipeline of RNA approaches to treating disease,” Noreen R. Henig, MD, ProQR’s chief medical officer, said in a news release.

“There are very few opportunities like ProQR where a great team, cutting-edge science and the passion for patients come together,” Rodman said. “In joining ProQR, I look forward to continue to make an effort for CF patients, but also on making a big impact for patients suffering from other rare diseases.”

ProQR’s portfolio also includes QR-110, a drug for treating Leber’s congenital amaurosis Type 10 (LCA10), the most common cause of genetic blindness in childhood. QR-110 has received Orphan Drug designation from the U.S. Food and Drug Administration and the European Medicines Agency.

In addition to its other candidates, ProQR is developing QR-313, a treatment for a rare disease called dystrophic epidermolysis bullosa.

Rodman, an expert in drug development, has held leadership roles in translational medicine, rare disease drug development, and RNA therapies. Translational medicine is an interdisciplinary approach to biomedicine.

One of Rodman’s positions was vice president and head of respiratory clinical development at Vertex Pharmaceuticals, where he led teams developing such CF therapies as Orkambi.

He most recently served as chief medical officer and executive vice president of research and development at miRagen Therapeutics and Nivalis. He also held management positions at the Novartis Institutes for Biomedical Research.

“In the ProQR tradition of only working with the best of the best, I’m very pleased that Dave is joining our team,” said Daniel de Boer, chief executive officer of ProQR.