Aer’s inhaled formulation targets the mucus plugs obstructing COPD patients’ airways.
Pictured: A doctor holds a chest x-ray/courtesy of iStock
A small Raleigh-based startup hopes to shake up the chronic obstructive pulmonary disease (COPD) market with a treatment for an underserved target—airway mucus plugs. Aer Therapeutics closed a $36 million Series A Thursday to take its inhaled mucolytic to the clinic.
The new company is a spin-out from John Fahy’s lab at UCSF. Human airways have three areas of pathological problems, Fahy told BioSpace. There are already treatments available for two of them.
The smooth muscle tightening that causes bronchospasm (wheezing) is treated with a bronchodilator, and airway inflammation is treated with anti-inflammatory drugs. The third issue occurs when the natural mucus in the airways becomes completely occlusive, forming mucus plugs that obstruct airways.
Very few drugs have specifically and effectively targeted the mucus component, Fahy said.
Approximately 5 million COPD patients and 2 million asthma patients in the U.S. are estimated to have a mucus plug-high disease subtype. AER-01 is designed to liquefy these mucus plugs.
“This is currently a ‘hot’ area and certainly important to patients,” Bruce Miller, chief scientific officer of the COPD Foundation, told BioSpace.
He called Aer’s approach sensible and said the test will be whether it brings positive clinical outcomes for patients.
Lack of Effective Treatments
There is currently no cure for COPD. Common treatments involve dual and triple therapies that combine a corticosteroid to reduce inflammation with one or two types of bronchodilators, in addition to rescue inhalers.
But these treatments do not address the airway obstruction caused by mucus plugs. Fahy’s lab, in collaboration with academic partners in Dublin, discovered they could put thiol chemistry on a sugar scaffold to create an effective mucolytic. The result turned a normally inert sugar into a tool that can cleave mucus plugs.
Delivering this agent via the airway is the key with AER-01, Fahy explained. Current drugs marketed to break up mucus have limited efficacy because when taken orally, sufficient concentrations will not reach the obstructed areas through the bloodstream to break up the plugs. Fahy said the principle of using an oral medication to treat airway mucus plugs is “doomed at the door.”
Cash for the Clinic
The first-in-human study will launch this year, most likely in June, Aer President and CEO Jim Shaffer told BioSpace. This Series A round, which included Canaan, OrbiMed and Hatteras Venture Partners, will give the startup a cash runway of about 2.5 years—enough to complete the Phase I study and a Phase IIa proof-of-concept trial, Shaffer said.
Phase I will enroll healthy volunteers in Australia with a single ascending dose, then multiple ascending dose arms, before shifting to COPD patients. The drug will first be administered via nebulizer, with plans to develop a dry powder inhaler formulation for convenience.
Phase IIa will follow, with plans to dose moderate-to-severe COPD patients with mucus plugs over four weeks in mid-2024.
Shaffer has expanded his one-man team to hire consultants as full-time staffers, with a target headcount of eight for now. Aer will lean on contract manufacturers to accomplish its mission in a cost-effective way, he said.
The World Health Organization ranks COPD as the third leading cause of death worldwide. If successful in this first indication, Shaffer said Aer has the potential to expand into other lung diseases like asthma, bronchiectasis and cystic fibrosis.
Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.
