Patients Say They Want to Switch to Less Frequent Therapies

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Patients with hereditary angioedema (HAE) are willing to try new therapies that can effectively lessen disease burden with less frequent dosing, according to an interview-based study conducted by Astria Therapeutics.

The findings were shared in a poster, titled “Burdens of Disease and Treatment in Hereditary Angioedema: Interview Insights from HAE Patients,” presented at the virtual 2021 NORD Rare Diseases and Orphan Products Breakthrough Summit, which ran Oct. 18–19.

“Our learnings from these findings support that there is a substantial need for new HAE treatments, and that HAE patients are open to trying new therapies that could reduce their disease and treatment burdens,” Andrew Komjathy, chief commercial officer at Astria, said in a press release.

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The study was conducted with 10 HAE patients, and covered their experiences with diagnosis, disease, and treatment management, as well as advocacy and their perceptions about potential improvements on preventive, or prophylactic, therapies. Structured interviews were conducted for one hour over the phone.

Symptoms started from age four to 16 for the participants, and delays in diagnosis were 16 years on average. For many, this delay stemmed from poor HAE awareness among physicians they had visited over the years.

“It was a very long time to diagnosis. It was unnecessary surgeries, trying different medications, ‘it’s all in her mind.’ I missed 2 years of school,” said one of the interviewed patients.

Patients reported trying two to three preventive treatments on average, and often ended up choosing the one that had the most convenient mode of administration. Most admitted they would feel compelled to try a therapy if dosing was required every two months or less often.

However, despite preventive treatment, half of the participants said they were constantly or often thinking about future disease attacks. Three patients stated that being on a preventive therapy helped them worry less about future attacks.

All those interviewed would switch to a new therapy if it offered similar efficacy with less frequent dosing.

“I’d choose ‘efficacy,’ but I don’t know how much more effective it could get — Takhzyro is supposed to be 80%–90% effective. But if something is equally effective, and it’s easier to integrate into my life, that would make me change,” said one of the patients.

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Earlier this year, Astria launched its preclinical program led by STAR-0215, an antibody-based medicine designed to prevent HAE attacks by suppressing the activity of the enzyme kallikrein. STAR-0215 was originally developed by Quellis Biosciences, which was later acquired by Astria, formerly known as Catabasis Pharmaceuticals.

Kallikrein is overly active in HAE patients due to the lack of another protein, called C1-inhibitor, which is responsible for controlling its activity. Excess kallikrein activity increases the levels of an inflammatory molecule, called bradykinin, which in turn causes blood vessels to expand and tissues to swell.

The mechanism of action of STAR-0215 is similar to that of the approved HAE therapy Takhzyro (lanadelumab). However, STAR-0215 proved to be 10 times more potent at suppressing the enzyme’s activity by 90% and to be more stable in the blood than Takhzyro, according to Astria.

Also, STAR-0215 has additional potential benefits, including requiring less frequent dosing, allowing for the use of smaller injection volumes, and enabling patients to remain free of breakthrough attacks for extended periods of time, the company stated.

“Our goal with our preclinical program, STAR-0215, is to provide the most patient-friendly preventative treatment option for people living with HAE with dosing every three months or longer,” said Komjathy.

“If we achieve our goals of demonstrating reduction of treatment burden while maintaining efficacy, we are hopeful that we can give patients the opportunity to focus their time and energy on what matters most to them, rather than managing their disease,” he added.

Astria expects to file an application by mid-2022 to launch a Phase 1 proof-of-concept trial of STAR-0215. Results are expected by the end of next year.