PharmaEssentia Announces Pipeline Presentation During Upcoming American Society of Hematology Annual Meeting

Company will update on its trial of ropeginterferon alfa-2b for essential thrombocythemia, which continues to enroll in the United States and Asia, and expands into Canada

BURLINGTON, Mass.–(BUSINESS WIRE)–PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced plans to present new updates on its pipeline during the 63^rd American Society of Hematology Annual Meeting, December 11-14, 2021.

During the meeting, the company will provide an update on SURPASS ET, a Phase 3 pivotal clinical trial of its investigational ropeginterferon alfa-2b (P1101), a novel mono-pegylated proline interferon under evaluation for the treatment of essential thrombocythemia (ET). PharmaEssentia continues to enroll participants in the trial at sites throughout the United States and Asia, and has now expanded with new sites in Canada.

�People diagnosed with a myeloproliferative neoplasm (MPN), and particularly those with ET, have limited therapeutic options that can help them manage the progressive effects of the disease, so through this study, we will evaluate whether our mono-pegylated interferon could represent a useful alternative to currently available options,” said Raymond Urbanski, M.D., Ph.D., U.S. Head of Clinical Development and Medical Affairs. “Since we initiated the study in the U.S. earlier this year, we have heard from clinicians outside the U.S. who are eager to consider a new investigational option to support their ET patients. We are pleased to partner with these renowned MPN specialists to participate in this important study.”

The SURPASS ET open-label, randomized, active-controlled study is comparing the efficacy, safety, and tolerability and pharmacokinetics of P1101 as second line therapy for ET as compared with anagrelide, a commonly used oral platelet-reduction therapy. The study aims to enroll approximately 160 patients, who will be randomized to receive either P1101 subcutaneously every two weeks or anagrelide at a standard dose. Eligible patients include high-risk ET patients, those who are resistant to or intolerant of hydroxyurea and who have not received prior therapy with interferon.

The primary efficacy endpoint for the study employs the modified European Leukemia Net (ELN) criteria. Secondary efficacy endpoints include time to and durability of response, response rates based on hematologic parameters and symptomatic improvements. Changes in CALR, MPL, and JAK2 allelic burden over time is also being investigated. Topline data are expected by late 2022. More details on the study design and sites are available at www.clinicaltrials.gov (NCT04285086) or at www.SURPASSET.com.

PharmaEssentia continues to build momentum with its pipeline to deliver new solutions in MPNs. The company is seeking approval for its first indication in polycythemia vera (PV) in the U.S. and expects a decision by the U.S. Food and Drug Administration (FDA) in November 2021.

About Ropeginterferon alfa-2b

Ropeginterferon alfa-2b is a long-acting, mono-pegylated proline interferon aimed to be administered once every two weeks or longer. Ropeginterferon alfa-2b has Orphan Drug designation for treatment of polycythemia vera (PV) in the United States. Marketed as Besremi® in Europe, the product was approved by the European Medicines Agency (EMA) in 2019. Ropeginterferon alfa-2b was discovered and is manufactured by PharmaEssentia in its Taichung plant, which was cGMP certified by TFDA in 2017 and by EMA in January 2018.

About Essential Thrombocythemia

Essential thrombocythemia (ET) is a myeloproliferative neoplasm (MPN) characterized by an overproduction of platelets in the blood that results from a genetic mutation; data indicates a JAK2 gene mutation is present in approximately half of diagnosed patients. ET is estimated to affect up to 57 per 100,000 people in the U.S. The disease is most commonly diagnosed through routine blood work, and is most common in people over the age of 50, with women 1.5 more times more likely to be diagnosed than men. As a chronic, progressive disease, ET requires regular monitoring and appropriate treatment. Over time, the disease may progress into more deadly conditions such as myelofibrosis or acute leukemia.^1,2

About PharmaEssentia

PharmaEssentia Corporation (TPEx: 6446), based in Taipei, Taiwan, is a rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, the company aims to deliver effective new biologics for challenging diseases in the areas of hematology and oncology, with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today the company is expanding its global presence with operations in the U.S., Japan, China and Korea, along with a world-class biologics production facility in Taichung. For more information, visit our website or find us on LinkedIn and Twitter.

Forward Looking Statement

Some of the statements included in this press release, particularly those relating to the results of clinical trials, the clinical benefits to be derived from ropeginterferon alfa-2b, regulatory submissions and the timing of any such review, approvals, the commercial opportunity and competitive positioning, and any business prospects for ropeginterferon alfa-2b, may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995 and similar legislation and regulations under Taiwanese law. Among the factors that could cause our actual results to differ materially are the following: acceptance of the BLA filing does not represent final evaluation of the adequacy of the data submitted in the BLA; whether the FDA will complete its review of the BLA on a timely basis; the risk that the FDA ultimately denies approval of the BLA; whether, ropeginterferon alfa-2b, if approved, will be successfully launched and marketed; and other risk factors identified from time to time in our reports filed with any global securities regulator or agency. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. The information found on our website, and the FDA website, is not incorporated by reference into this press release and is included for reference purposes only.

¹ Mehta J, Wang H, Iqbal SU, Mesa R. Epidemiology of myeloproliferative neoplasms in the United States. Leuk Lymphoma. 2014 Mar;55(3):595-600

² “What is Essential Thrombocythemia?” MPN Research Foundation. 2020. Available at: http://www.mpnresearchfoundation.org/Essential-Thrombocythemia

Contacts

Kellie Hotz, kellie_hotz@pharmaessentia.com