HONG KONG – Takeda Pharmaceutical Co. Ltd. is preparing to take Takhzyro (lanadelumab) to the world stage after announcing its interim results for a phase III hereditary angioedema long-term prophylaxis (HELP) study.

Donatello Crocetta, Takeda’s global medical head of rare immunology and metabolic diseases, told BioWorld the Japanese company has “an ambitious registration and launch plan” for the drug, aiming to launch it in “over 20 new countries … within the next year … to ensure access to Takhzyro for as many patients as possible worldwide.”

Successful launches have already taken place in the U.S., Switzerland, Germany, Austria, the U.K., Israel and the United Arab Emirates. The company has already initiated access schemes in France, Greece, Finland, Norway and Sweden.

Takeda released the results of its HELP study on June 6 at the 2020 European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress. A second Japanese company, Astellas Pharma Inc, released positive results from its DOLOMITES study two days later. The latter study compared the efficacy and safety of roxadustat to darbepoetin alfa in treating anemia in nondialysis-dependent, stage 3 to 5 chronic kidney disease (CKD) patients.

The HELP open-label extension suggested that Takhzyro was well-tolerated and could prevent hereditary angioedema attacks over an extended treatment period, with sustained and consistent reductions in monthly attack rates across a range of different patient subgroups, according to Takeda. The company also said that monthly attack rates were at 0.18 in the extension against 0.26 at the end of the original study.

“Data from the interim analyses of the HELP study open-label extension are very encouraging and showed the safety and efficacy profile of Takhzyro was consistent with that of the original findings from the phase III HELP study,” Crocetta said. “The analyses we have recently presented suggest that Takhzyro is well-tolerated and can prevent HAE attacks over an extended treatment period, with reduction in monthly attack rates across different patient subgroups.”

That “evidence helps us understand the potential of Takhzyro to provide sustained prevention and reduce attack frequencies long-term, across a range of patient demographics and clinical characteristics,” he added.

The original HELP study evaluated 125 patients, ages 12 and older, over 26 weeks, with the extension further testing Takhzyro’s long-term safety and efficacy by evaluating 212 patients between May 2016 and August 2018. Of the patients who participated in the extension, 109 took part in the original study and 103 patients did not participate in the study but had at least one hereditary angioedema attack in the 12 weeks prior to the study commencement date. The extension was completed in November 2019.

Takeda will also be testing Takhzyro against other indications to broaden its potential use. A phase III study to test the drug’s efficacy in treating pediatric hereditary angioedema is currently underway, with a clinical study in other forms of bradykinin-mediated angioedema set to be initiated soon.

The company is also evaluating the potential for Takhzyro, alongside icatibant, as a potential therapy for the COVID-19 virus. Crocetta said clinical trials had been “initiated or planned” but did not provide further details.

Takhzyro is a human monoclonal antibody that binds and decreases plasma kallikrein activity when injected. Developed by Shire plc, it is produced in Chinese hamster ovary cells through recombinant DNA technology. Shire obtained FDA approval for the drug in August 2018, with the European Commission (EC) giving its approval three months later.

Takeda completed its acquisition of Shire in January 2019, after it saw its proposals rejected four times, and eight months after it finally announced the $58.66 million deal in May 2018. Takeda shareholders approved the deal at an extraordinary shareholder meeting in December 2018.

The company also recently announced the results of the phase II trials for its NEDD8-activating enzyme (NAE) inhibitor, pevonedistat-2001, at the 56th American Society of Clinical Oncology meeting as well as the 25th European Hematology Association meeting, with both virtual events taking place in May.

It followed up the pevonedistat-2001 results by inking a license agreement with Switzerland’s Debiopharm SA to develop microbiome therapeutics to treat gastrointestinal disorders. Its most recent FY 2019 financial results also reveal an active pipeline and an expanding footprint in China, with more than 15 approvals planned over the next five years.

The pipeline also includes 12 “Wave 1” best-in-class/first-in-class new molecular entities with potential approvals through the 2024 fiscal year; nine ongoing registration-enabling studies and around 30 clinical stage early development “Wave 2” new molecular entities, plus increasing investment in next-generation platforms for sustained growth through the 2025 fiscal year and beyond.

Meanwhile, Takeda shares (Tokyo:4502) were down 1.29% to close at ¥4,062 (US$38.04) on June 11.

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