After BioCryst fizzle, a new oral HAE player emerges
A new player in the search for an oral hereditary angioedema pill has received a major boost and show of confidence.
Pharvaris announced $66 million in Series B financing for its B2 receptor antagonist pill, nearly tripling the funding Attune Pharmaceuticals recently received in Series B for its own hereditary angioedema (HAE) pill. The money will fuel the company’s Phase I trial on its lead compound, PHA121, on healthy volunteers. The first subjects have already been dosed.
The news comes three-and-a-half months after BioCryst’s great angioedema fizzle. Although the Durham outfit’s oral drug BCX7353 technically passed a Phase III trial, the drug failed to cut the attack rate by at least 50%, the cutoff at which Wall Street analysts considered the drug marketable, and the biotech’s stock tumbled.
Hereditary angioedema, a potentially life-threatening genetic disorder, affects 1 in every 50,000 people. It is characterized by episodes of extreme swelling across the body. It can cause vomiting and nausea when manifest in the gut and can even be fatal if it appears in the airways and restricts breathing.
Several therapies and injections exist to curb the attacks, but last year Shire’s Takhzyro (lanadelumab) emerged as one of the hottest and most effective drugs, contributing to an 87% reduction in mean HAE attack frequency in a Phase III trial (versus a 0% reduction in the placebo group) that ushered it toward market. The drug is on track to become a blockbuster. Clairvoyant Analytics projects $1.15 billion in sales by 2022.
But because Takhzyro is an injection, the search is still on for an oral medication that can curb the disorder at similar rates. BioCryst continues to develop its oral drug, while Attune Pharmaceuticals remains in the early stage.
HAE is caused by low levels of or dysfunctioning C1 esterase inhibitor protein. Most available therapies work by inhibiting C1 esterase, fulfilling the function of the missing protein. But Pharvaris’ Ph121 works like the FDA-approved icatibant injection, which functions as an antagonist of bradykinin B2 receptors – i.e. it attacks the molecules that directly cause the swelling. The founders of Pharvaris include an inventor of icatibant and a former member of the leadership at Jerini AG, the biopharmaceutical that sells the injection.
