San Diego-based aTyr Pharma recently raised $86.3 million in what some characterized as a dramatic turnaround for the company.
After a failed attempt to develop a treatment for a rare neuromuscular disease, aTyr is having far more success with a new drug known as ATYR1923, meant to treat pulmonary fibrosis – a disease that occurs when lung tissue becomes damaged and scarred.
This thickened, stiff tissue makes it more difficult for the lungs to work properly. As pulmonary fibrosis worsens, people become progressively more short of breath, according to the Mayo Clinic.
“It’s a very scary disease to get,” said Sanjay Shukla, aTyr president and CEO. “Oftentimes, you need a lung transplant.”
The most common treatment for the disease is to take steroids that have harmful side effects over time, Shukla said.
“Steroids can be used kind of in the short term to calm down inflammation. When you take them time after time, day after day, year after year, it just wrecks your body,” Shukla said. “You gain weight, there’s just a host of things.”
Better Than Expected
So far, aTyr’s new drug has performed well in initial clinical testing.
“We’ve been fortunate to have very outstanding data that has been the culmination of my last five years as CEO,” Shukla said. “It’s been a journey for the past four years where we have learned more about how the drug works, tested it in vitro, which is basically in the dish.”
Moving on to animal studies, the drug “appeared really well in preventing and sometimes reversing lung inflammation and fibrosis,” Shukla said.
Subsequent tests on human volunteers were equally encouraging.
“It performed better than expected,” Shukla said. “It did an outstanding job in letting patients reduce their steroid use.”
More importantly, volunteers taking the drug felt better.
“That’s important to me. The patient has to feel better,” Shukla said. “The fact that we’re improving symptoms has us really feeling good at aTyr.”
In January 2020, aTyr entered into a license with Kyorin Pharmaceutical Co., Ltd. for the development and commercialization of ATYR1923 in Japan.
“We are moving now into the last phase of clinical development,” Shukla said, adding the goal is to gain final FDA approval in 2024 or 2025.
aTyr was founded in 2005 by Paul Schimmel and Xian-Lei Young, professors of molecular medicine at Scripps Research Institute.
“He (Schimmel) should win the Nobel Prize. It’s probably the only thing he hasn’t won at this point,” Shukla said.
Shukla took over as president and CEO of aTyr in November 2017 after serving as the company’s chief medical officer from March 2016 to November 2017 and had worked for the company as a consultant from January 2016 to March 2016.
He characterized the company’s issues with its earlier drug as a learning experience.
“Early data looked good. It didn’t trend in the right direction. We had to make the painful decision to shut that down,” Shukla said. “We learned some things about the biology that told us it might be better for us to look in other areas, other diseases.”
The company had to regroup and downsize by about a third, according to Shukla.
“There’s a challenge here in translating this science from a real prominent San Diego research institution and essentially trying to make a medicine in a new area of biology,” Shukla said. “Lab technology is risky.”
Building on the success of the new drug, Shukla said he expects aTyr to grow substantially
“I think we can build the type of large, anchor biotech company that can rival the companies in Boston and San Francisco,” Shukla said. “We do think we are a company that has a pipeline of new biology.”
Boston and San Francisco are the leading life science centers in the nation, with San Diego ranked third.
“We’re poised to be a much bigger company,” Shukla said. “I think the future for us is very bright.”
The company is back up to 60 employees, Shukla said. In a year, he expects to have a staff of 80 to 100.
“We have learned and we have advanced our science,” Shukla said, also noting the company is working on a drug for the treatment of breast cancer and lung cancer that will be moving into a clinical trial in 2022.
CEO: Sanjay S. Shukla
Headquarters: La Jolla
Business: pharmaceutical company
Notable: aTyr was founded on scientific discoveries from the lab of Paul Scimmel, biophysical chemist and professor of molecular medicine at The Scripps Research Institute