About HAE
HAE is a rare, debilitating genetic inflammatory condition, which causes episodes of swelling in the face, extremities, and GI tract and can be life threatening. It is estimated that 30-40% of patients afflicted by HAE in the U.S. and EU remain undiagnosed, creating a significant growth opportunity in this area. Further, prophylactic treatment is likely underutilized with roughly 40% of patients only treating their attacks acutely on an as-needed basis.

Information on DX-2930

Through the transaction, Shire will acquire DX-2930, a Phase 3-ready novel long-acting highly potent human monoclonal antibody inhibitor of pKal, which has patent protection and anticipated regulatory exclusivity beyond 2030. Proof of concept was demonstrated in a multi-center, randomized, double-blind, placebo-controlled, multiple ascending dose Phase 1B study in HAE patients, based on patients in 300mg, 400mg and placebo groups, who reported having at least two HAE attacks in the three months prior to study entry. Each patient received two treatments of DX-2930 separated by 14 days. During the pre-specified, primary efficacy interval of six weeks (Day 8 to 50), the HAE attack rate was reduced by over 90% in the DX-2930 combined 300mg and 400mg arms, with 0 attacks in the 300mg group (n=4; p < 0.0001) and 0.045 attacks per week in the 400 mg group (n=11; p =0.005), compared to 0.37 attacks per week in the placebo group (n=11). DX-2930 was well tolerated at all dose levels with no evidence of dose-limiting toxicity up to 400 mg. The most common adverse events were HAE attacks, injection site pain, and headache, which were not appreciably higher in the DX-2930 arms compared with placebo. In the study, a total of 37 patients were randomized to active drug or placebo in a 2:1 ratio across 4 dosing groups of 30, 100, 300, or 400mg. Each patient received two doses of DX-2930 or placebo, separated by 14 days, and was followed for 15 weeks after the second dose.

A post hoc analysis of a subgroup of four patients who participated in the Phase 1B study from the 300mg (1) and 400mg (3) cohorts with severe HAE (9-36 attacks in the prior 3 months) had no breakthrough attacks on DX-2930 during the observation period (Day 8 to 50).

There were no deaths or patient discontinuations due to an adverse event, no serious adverse events in patients treated with DX-2930 and no evidence of dose-limiting toxicity was observed. There was no safety signal in treatment-emergent adverse events, clinical laboratory results, vital signs, or electrocardiograms. Subcutaneous injection was well tolerated.

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