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Areteia gets $350M to turn failed ALS drug into a therapy for severe asthma

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The available treatments for eosinophilic asthma come with a wide range of side effect risks. Areteia Therapeutics is developing an oral therapy designed to match the blockbuster biologic drugs that treat this severe form asthma, but with a potentially better safety profile. On Tuesday, the new company unveiled its approach along with up to $350 million in financial commitments to support pivotal tests of its experimental medicine.

Like other forms of asthma, eosinophilic asthma causes inflamed airways that are blocked by mucus, making it difficult to breathe. But eosinophilic asthma stems from a type of white blood cell called an eosinophil. In high levels, eosinophils cause inflammation in the airways.

Areteia’s lead drug candidate is dexpramipexole, a first-in-class small molecule designed to block the maturation and release of eosinophils in the bone marrow, where these cells are produced. In a placebo-controlled Phase 2 test enrolling patients with moderate-to-severe eosinophilic asthma, company said that treatment with its drug led to significant, dose-dependent reduction in eosinophil levels in the blood at each of the three doses tested. Furthermore, the once-daily pill was well tolerated by patients and no serious adverse effects were reported. With the new capital, Areteia plans to advance to Phase 3 testing. Success there could bring redemption for a drug that previously fell short in amyotrophic lateral sclerosis (ALS).

Dexpramipexole comes from the labs of Pittsburgh-based Knopp Biosciences. A decade ago, that privately held drug discovery company was developing dexpramipexole for ALS in a research partnership with Biogen. In 2013, Biogen reported that the drug failed its Phase 3 clinical trial. Under Knopp, the drug continued clinical development in eosinophilic asthma.

The first line of eosinophilic asthma treatments are typically inhaled steroids. If the symptoms are severe, oral steroids offer another option. But long-term use of these drugs raises the risks of developing osteoporosis, weight gain and diabetes. Injectable biologic drugs offer yet another treatment option. These antibody drugs are designed to block interleukin 5, a signaling protein that plays a key role in the maturation of eosinophils. These drugs can also cause hypersensitivity reactions and raise the risk of infections, but that hasn’t stopped them from becoming big sellers. AstraZeneca’s Fasenra and GSK’s Nucala accounted for $1.2 billion and £1.14 billion (about $1.15 billion) respectively in 2021 revenue, according to company financial reports. Teva Pharmaceutical Industries markets Cinqair for eosinophilic asthma; that company’s annual report does not break out sales for individual products.

Areteia was created by Knopp and Population Health Partners, a private equity firm that forms new companies around late-stage drug candidates with the potential to address prevalent diseases. If dexpramipexole can win regulatory approval as the first oral drug for eosinophilic asthma, the company said that it could become an alternative to biologic drugs with the potential of also finding use as an earlier line of therapy.

The Series A round of funding announced Tuesday was led by Bain Capital Life Sciences. With the financing, dexpramipexole transfers from Knopp to Areteia. Other investors include Access Biotechnology, GV, Arch Venture Partners, Saturn Partners, Sanofi, Maverick Capital, and Population Health Partners.

Photo credit: Jackie Niam, Getty Images

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