SAN FRANCISCO — As the world’s leading biotech executives descended on the annual J.P. Morgan Healthcare Conference this week to talk about gene editing, precision medicine, and other cutting-edge treatments, the folks at tiny Waltham-based Proteon Therapeutics came to promote a less sexy goal.
They want to prevent the vein that surgeons create in the arm of kidney patients on dialysis from clogging.
The firm of 17 employees has been at it, despite major setbacks, for more than 12 years.
For decades, doctors have recommended a simple operation so dialysis patients can hook up to a machine that cleans their blood of wastes that their kidneys don’t remove. Surgeons create a lifeline called an arteriovenous fistula by stitching an artery to a vein in the arm or the wrist.
The fistula creates a more robust vein with greater blood flow. It enables blood to travel from a patient’s arm through a needle to a dialysis machine. Treatments take a few hours, three times a week. The machine removes waste and then returns the filtered blood through another needle in the arm of the patient.
Unfortunately, executives of Proteon say, about half of the surgeries fail to create a usable blood vessel. Many patients end up needing a catheter inserted in the neck, which results in much higher rates of infection and mortality.
As Dr. Steven Burke, Proteon’s chief medical officer, put it, the fistula is for many patients a “lifeline and their Achilles heel.”
Approximately 468,000 patients are on dialysis in the United States, according to the National Kidney Foundation.
In the late 1990s, an interventional radiologist at Johns Hopkins Hospital, Dr. Nicholas Franano, came up with the idea of applying a drug made from a recombinant protein to the newly created fistula to prevent it from clogging. That became the idea behind Proteon, which Franano helped found in 2006.
The company went public in 2014, raising $61 million in the hopes of developing and winning approval of the experimental drug, vonapanitase. Timothy P. Noyes, Proteon’s chief executive, is meeting with investors in San Francisco this week to tell them that he still believes it could be a game changer.
“At the end of the day, the most exciting part about scientific innovation is discovering, developing and launching a drug that you give to people and makes their lives better,” Noyes, 57, said. “I don’t know what can be sexier.”
Noyes, a veteran biopharma executive who worked at GelTex Pharmaceuticals before it was bought by Genzyme in 2000 for about $1 billion, said the protein works by enabling the blood vessel to expand.
Wall Street took a decidedly dimmer view in December 2016.
Shares of Proteon plunged nearly 75 percent after the company reported that the experimental drug failed to meet its primary goal in a late-stage clinical trial. The stock price fell from $7.40 to $2.50 on the Nasdaq exchange. That’s about what it is trading for now.
Nonetheless, Proteon pressed forward with a second late-stage clinical at more than 40 sites, including at Brigham and Women’s Hospital in Boston and UMass Memorial Medical Center in Worcester.
Noyes said Tuesday that vonapanitase failed the first late-stage trial’s main goal — keeping blood flowing without need for another medical procedure. But, he said, the drug succeeded in a secondary trial goal — increasing the length of time between when a fistula is created and when it finally fails. Proteon said that in patients treated with its drug in the first late-stage trial, the risk of this failure fell by 34 percent.
When the larger second late-stage trial concludes in March, Noyes said, he believes the results will show that the medicine prolongs the life of the blood vessel, benefiting patients and reducing medical costs.
“We’ve always maintained that there are several ways that we could demonstrate a potential benefit to patients,” he said. “That’s not changing the goal posts, that’s drug development in a novel space.”
Jonathan Saltzman can be reached at firstname.lastname@example.org.