Aqualung Therapeutics, a Tucson-based drug startup headed by a University of Arizona pulmonologist, has won a major investment to help take its flagship drug into initial human clinical trials for treatment of life-threatening lung inflammation.
And that may be just the start for Aqualung, which last week announced promising preclinical study results for use of its drug to treat prostate cancer and is looking at a variety of diseases that may respond to the therapy.
Aqualung, which was founded by UA pulmonologist Dr. Joe G.N. “Skip” Garcia, recently announced it recently closed a $2.5 million investment round led by JSR Life Sciences, a Silicon Valley biopharma services and manufacturing company.
In addition to the investment, which was in the form of a convertible note, JSR has committed another $1.5 million to a planned $10 million Series A venture-capital fundraising round, the companies said.
The recently announced investment will help Aqualung launch its first human clinical trial of its drug, a monoclonal antibody called ALT-100, for treatment of Acute Respiratory Distress Syndrome.
Garcia, Aqualung’s CEO, said he’s excited about seeing the foundational research he began more than 30 years ago take a major step toward patients’ bedsides.
“The ‘Holy Grail’ for any physician-scientist is to make a discovery in the laboratory which successfully translates to the clinic or hospital to reduce human suffering and to improve human health,” Garcia said. “We believe the ALT-100 (monoclonal antibody) fits that description very well and have moved very effectively as a company to bring this therapeutic to a clinical reality.”
The drug is based on foundational research Garcia began decades ago, with continual grant support from the National Institutes of Health.
Garcia, who joined the UA in 2013 as senior vice president of health services, said the key discovery came while he was chief of pulmonary and critical care medicine at Johns Hopkins University from 1998 to 2005.
Using new genomic technology, Garcia and his colleagues for the first time identified an enzyme called extracellular Nicotinamide phosphoribosyltransferase or eNAMPT, as a important factor in animal models of acute respiratory distress syndrome (ARDS), an often fatal buildup of fluid in the lungs.
“Although there were no papers in the entire world’s scientific literature about NAMPT, we courageously pursued this target with the hope it may be a relevant target in humans with ARDS,” said Garcia, Dr. Merlin K. Duval Endowed Professor of Medicine at the UA.
Garcia and his team at Johns Hopkins had a eureka moment when they discovered eNAMPT was present in human ARDS patients, Garcia said.
Later, while Garcia was chair of the University of Chicago Department of Medicine, researchers were able to show that eNAMPT was a master regulator of inflammation that could potentially be courted with drugs.
Garcia and his lab then created antibodies to neutralize eNAMPT that proved to be significantly protective in reducing lung injury in animal models.
Aqualung says its preclinical studies show its monoclonal antibody drug can suppress eNAMPT to lessen runaway inflammation that can lead to death in ARDS patients.
The convertible-note investment will help fund a Phase Ia clinical trial in which ALT-100 will be given to a small cohort of healthy humans to gauge its safety and tolerance in patients, said Stan Miele, president of Aqualung Therapeutics.
The company plans to complete its $10 million fundraising within about six months, meanwhile completing the Phase Ia study in Australia, a popular trials venue for early-stage companies because of its generous tax breaks for drug development, Miele said.
Aqualung plans to file an investigational new drug application with the U.S. Food and Drug Administration within the next couple of months as it wraps up required animal toxicology studies, he said.
Confident in the safety of its drug, Miele said, the company is already planning a Phase II clinical trial in ARDS patients at five sites in the U.S. and one in Australia and hopes to complete that trial within the next two years.
Miele said Aqualung is well-positioned to take its drug through trials, after publishing seven peer-reviewed papers on its preclinical studies and manufacturing more than 3,000 doses for human clinical trials.
The company uses Garcia’s UA lab for drug development and has a tech-transfer agreement with the school to cover any intellectual property developed there.
Just the beginning
ARDS treatment is just the start for Aqualung’s drug, which the company says affects a main immunity pathway involved in many life-threatening inflammatory conditions, including ventilator-induced lung injury, radiation-induced lung injuries, intrauterine infections during pregnancy, prostate cancer, pulmonary hypertension, autoimmune disorders and both pulmonary and hepatic fibrosis.
Last week, Aqualung announced that its ALT-100 monoclonal antibody significantly reduced the severity of aggressive prostate cancer, Miele noted.
As an immunotherapy to check inflammation, Aqualung’s drug could eventually be used to treat a range of diseases.
“This targets sort of the central, innate immunity pathway, like a drug like Humira, that has multiple indications, whether it’s for Crohn’s disease or psoriatic arthritis, they focus on an innate immunity pathway as well,” Miele said.
But taking Aqualung’s first drug through trials and to market approval will still take years and more funding rounds in the tens of millions of dollars, Miele said.
Aqualung has a found a strong partner in JSR Life Sciences, a Sunnyvale, California, provider of drug development and manufacturing services.
JSR is working with other experienced life science investment groups to coordinate a syndicated series A capital round within the coming months, Aqualung said.
“As early investors in this novel target of eNAMPT, we have seen how ALT-100 can potentially bring significant value to the industry and lead to therapeutic breakthroughs for patients,” Jason Zhang, JSR Life Sciences’ Corporate Venture Fund, said in announcing the investment. “We believe that the Aqualung team has developed a truly innovative platform technology that can have an impact on reducing hospitalization for those with ARDS, but also save lives of ARDS patients.”
