Business Wire

BeiGene Receives Positive CHMP Opinion for BRUKINSA ® (Zanubrutinib) for the Treatment of Adults with Waldenström’s Macroglobulinemia

Share

BeiGene (NASDAQ: BGNE; HKEX: 06160) today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending approval of BRUKINSA (zanubrutinib) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM) who have received at least one prior therapy or first-line treatment for patients unsuitable for chemo-immunotherapy.

“Bruton’s tyrosine kinase (BTK) inhibitors have emerged as a promising treatment for WM, yet treatment discontinuation due to lack of response or side effects remains a concern,” said Prof. Christian Buske, Medical Director at the University Hospital Ulm, Germany, and a trial investigator of the ASPEN study. “The ASPEN trial demonstrated that BRUKINSA provided deep and durable responses and offered substantial improvements in safety and tolerability over standard therapy. Patients in Europe with WM may soon have a new treatment option that can offer improved outcomes.”

The positive CHMP opinion is based on results from the randomized, Phase 3 ASPEN clinical trial, evaluating BRUKINSA compared to ibrutinib in patients with relapsed or refractory (R/R) or treatment-naïve (TN) WM who are unsuitable for chemo-immunotherapy. Based on the modified Sixth International Workshop on Waldenström’s Macroglobulinemia (IWWM-6) response criteria (Treon 2015), the combined complete response (CR) +VGPR rate in the overall intention-to-treat (ITT) population was 28.4% with BRUKINSA (95% CI: 20, 38), compared to 19.2% with ibrutinib (95% CI: 12, 28). While this difference was not statistically significant, BRUKINSA did achieve numerically higher VGPR rates and trends towards increased response quality.1

BRUKINSA demonstrated a more favorable safety profile compared to ibrutinib with lower frequency of certain adverse events, including atrial fibrillation or flutter (2.0% vs. 15.3%) minor bleeding (48.5% vs 59.2%) and major hemorrhage (5.9% vs 9.2%).1 Of the 101 patients with WM treated with BRUKINSA, four percent of patients discontinued due to adverse events, and adverse events leading to dose reduction occurred in 14% of patients.

“The positive CHMP opinion reflects BRUKINSA’s potential role in the WM therapeutic landscape as a selective inhibitor designed to deliver sustained and continuous inhibition of BTK, offering patients the potential for reduced frequency of certain cardiovascular events like atrial fibrillation compared to ibrutinib, and underscores our bold approach to R&D,” said Jane Huang, M.D., Chief Medical Officer, Hematology, at BeiGene. “We are committed to advancing the global registration of BRUKINSA and, if approved, believe it will become the preferred BTK inhibitor for patients with WM.”

“We have a strong team in Europe who are excited for the opportunity to further work with the many investigators who have participated in BRUKINSA trials conducted in Europe to-date. Looking to the future, we have built a team in Europe and they are poised to help patients access BRUKINSA following its expected approval,” said Gerwin Winter, Senior Vice President, Head of Commercial, Europe, at BeiGene. “We look forward to continuing our work with the European health authorities to bring BRUKINSA to patients living with this rare, incurable blood cancer.”

Following the CHMP positive opinion, the European Commission will consider BeiGene’s marketing application, with a final decision expected within 67 days of receipt of the CHMP opinion. The decision will be applicable to all 27 member states of the EU plus Iceland and Norway.

About Waldenström’s Macroglobulinemia

WM is a rare lymphoma representing approximately one percent of all non-Hodgkin’s lymphomas and typically progresses slowly after diagnosis.2 The disease usually affects older adults and is primarily found in the bone marrow, although lymph nodes and the spleen may be involved.3 Throughout Europe, the estimated incidence rate of WM is approximately seven for every one million men and four for every one million women.4

About the ASPEN trial

The Phase 3 randomized, open-label, multicenter ASPEN clinical trial (NCT03053440) evaluated zanubrutinib versus ibrutinib in people with relapsed or refractory (R/R) or treatment-naïve (TN) WM. The primary objective was to establish superiority of zanubrutinib compared to ibrutinib as demonstrated by the proportion of people achieving complete response (CR) or very good partial response (VGPR). Secondary endpoints included major response rate, duration of response and progression-free survival, and safety, measured by incidence, timing and severity of treatment-emergent adverse events. The pre-specified analysis populations for the trial included the overall population (n=201) and R/R patients (n=164). Exploratory endpoints included quality of life measures.

The study includes two cohorts, a randomized cohort (cohort 1) consisting of 201 patients with a MYD88 mutation (MYD88MUT) and a non-randomized cohort (cohort 2) in which 28 patients with MYD88 wild-type (MYD88WT) received zanubrutinib because they have historically responded poorly to ibrutinib therapy.

The randomized cohort 1 enrolled 102 patients (including 83 relapsed or refractory (R/R) patients and 19 TN (patients) in the zanubrutinib arm and 99 patients (including 81 R/R patients and 18 TN patients) in the ibrutinib arm. Patients in the zanubrutinib arm were assigned to receive zanubrutinib 160 mg twice daily (BID) and patients in the ibrutinib arm received 420 mg of ibrutinib once daily (QD).

Results of cohort 2 were previously presented at the 24th Congress of European Hematology Association (EHA) and showed an overall response rate (ORR) of 80.8%, a major response rate (MRR; partial response or better) of 53.8% and a VGPR rate of 23.1%.

About BRUKINSA

BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.

BRUKINSA is currently approved in several regions for various indications.* To-date, more than 30 marketing authorization applications in multiple indications have been submitted in the United States, China, the European Union, and more than 20 other countries or regions.

BeiGene Oncology

BeiGene is committed to advancing best and first-in-class clinical candidates internally or with like-minded partners to develop impactful and affordable medicines for patients across the globe. We have a growing R&D team of approximately 2,300 colleagues dedicated to advancing more than 90 clinical trials involving more than 13,000 patients and healthy volunteers. Our expansive portfolio is directed by a predominantly internalized clinical development team supporting trials in more than 40 countries. Hematology-oncology and solid tumor targeted therapies and immuno-oncology are key focus areas for the Company, with both mono- and combination therapies prioritized in our research and development. We currently market three medicines discovered and developed in our labs: BTK inhibitor BRUKINSA in the United States, China, Canada, and additional international markets; and non-FC-gamma receptor binding anti-PD-1 antibody tislelizumab and PARP inhibitor pamiparib in China.

BeiGene also partners with innovative companies who share our goal of developing therapies to address global health needs. We commercialize a range of oncology medicines in China licensed from Amgen and Bristol Myers Squibb. We also plan to address greater areas of unmet need globally through our collaborations including with Amgen, Bio-Thera, EUSA Pharma, Mirati Therapeutics, Seagen, and Zymeworks. BeiGene has also entered into a collaboration with Novartis granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.

About BeiGene

BeiGene is a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide. With a broad portfolio of more than 40 clinical candidates, we are expediting development of our diverse pipeline of novel therapeutics through our own capabilities and collaborations. We are committed to radically improving access to medicines for two billion more people by 2030. BeiGene has a growing global team of over 7,000 colleagues across five continents. To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding future development and potential commercialization of BRUKINSA in the European Union and other markets, the potential for BRUKINSA to be a best-in-class BTK inhibitor, the potential for zanubrutinib to provide improved clinical benefit with advantages in safety, and the potential commercial opportunity for BRUKINSA. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its medicines and technology; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGene’s limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates; the impact of the COVID-19 pandemic on the Company’s clinical development, commercial and other operations, as well as those risks more fully discussed in the section entitled “Risk Factors” in BeiGene’s most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

* BRUKINSA is approved in the following indications and regions:

  • For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)a;
  • For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020) b;
  • For the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who have received at least one prior therapy (China, June 2020) b;
  • For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021);
  • For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021);
  • Registered and reimbursed for the treatment of MCL in patients who have received at least one prior therapy (Israel, April 2021);
  • For the treatment of adult patients with WM who have received at least one prior therapy (China, June 2021)b;
  • For the treatment of MCL in adult patients who have received at least one prior therapy (Canada, July 2021);
  • For the treatment of MCL in adult patients who have received at least one prior therapy (Chile, July 2021);
  • For the treatment of adult patients with MCL who have received at least one previous therapy (Brazil, August 2021); and
  • For the treatment of adult patients with WM (United States, August 2021).

a. This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

b. This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.

References:

1. Tam, et al. A randomized phase 3 trial of zanubrutinib vs ibrutinib in symptomatic Waldenström macroglobulinemia: the ASPEN study. Blood. October 2020. 136(18): 2038-2050.

2. Lymphoma Research Foundation. Getting the Facts: Waldenström Macroglobulinemia. Available at https://lymphoma.org/wp-content/uploads/2020/09/LRF_Factsheet_Waldenstro%CC%88m-Macroglobulinemia_090920.pdf. Accessed April 2021.

3. Lymphoma Research Foundation. Available at https://lymphoma.org/aboutlymphoma/nhl/wm/. Accessed December 2020.

4. Buske, C, et al. Treatment and outcome patterns in European patients with Waldenström’s macroglobulinaemia: a large, observational, retrospective chart review. The Lancet Haematology 2018; 5: e0299-309.

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Investor Contact
Gabrielle Zhou
+86 10-5895-8058 or +1 857-302-5189
ir@beigene.com

Media Contact
Liza Heapes or Vivian Ni
+1 857-302-5663 or +1 857-302-7596
media@beigene.com

About Business Wire

Business Wire
Business Wire



Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Janssen Receives Positive CHMP Opinion for RYBREVANT ® (amivantamab) for the Treatment of Patients with Advanced Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations After Failure of Platinum-Based Therapy15.10.2021 18:30:00 CEST | Press release

FOR EU TRADE AND MEDICAL MEDIA ONLY. NOT TO BE DISTRIBUTED TO UK AND BENELUX BASED MEDIA The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a conditional marketing authorisation for the medicinal product amivantamab, intended for the treatment of adult patients with advanced NSCLC with activating epidermal growth factor receptor (EGFR) exon 20 insertion mutations, after failure of platinum-based therapy.3 If approved, amivantamab will be the first treatment in the European Union specifically targeting EGFR exon 20 insertion mutations for advanced NSCLC.1 “This group of patients often face a poor prognosis as there are currently no targeted therapy options for this specific mutation nor does it typically respond to existing therapies used to treat more common EGFR mutations,” 1,4,5 commented Catherine Taylor, M.D., Vice President, Medical Affairs

Leading Researchers Present 24-Plex Digital PCR Assay and Novel Multiplex Applications Uniquely Enabled by Stilla’s 6-Color naica ® system15.10.2021 15:08:00 CEST | Press release

Stilla Technologies, the multiplex digital PCR company, joined researchers from SAGA Diagnostics in presenting a 24-plex digital PCR assay generated on Stilla’s six-color naica® system. To mark the platform’s global commercial launch at the American Society of Human Genetics (ASHG) 2021 Virtual Meeting, Stilla’s Cofounder and Chief Technology Officer Rémi Dangla, PhD, and researchers from SAGA Diagnostics, Fred Hutchinson Cancer Research Center, and University of Athens highlighted the new data during a discussion on how highly sensitive, multiplex digital PCR technology is transforming complex genomic data into actionable insights across a breadth of research and clinical applications. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211015005140/en/ Stilla’s six-color naica® system, the industry’s first six-color digital PCR system, answers market needs for higher performance and multiplexing technology. Learn more here: www

Boston Metal Wins S&P Global Platts Global Metals Award15.10.2021 15:04:00 CEST | Press release

Boston Metal won the New Technology for the Metals and Mining Industry award at the 2021 S&P Global Platts Global Metals Awards. Boston Metal is commercializing ground-breaking technology to decarbonize primary steelmaking. The company’s technology combines patented innovations with best practices from the aluminum and steel industries to deliver a revolutionary solution to the carbon emissions challenge facing the steel value chain. Powered by renewable electricity, molten oxide electrolysis converts iron ore into liquid metal and oxygen. The modular, scalable platform produces no CO2 emissions, is highly energy efficient, and works with a wide range of iron ore grades. “We congratulate all the winners and finalists for persevering through unique challenges and continuing to drive performance while embracing change,” said Saugata Saha, president of S&P Global Platts. Dave Ernsberger, Global Head of Pricing and Market Insight, S&P Global Platts, said: “Not surprisingly, but certainly e

The Ministry of Justice makes public the evidence supporting of the Republic of Kazakhstan’s case against Anatolie and Gabriel Stati15.10.2021 13:46:00 CEST | Press release

The Ministry of Justice has launched a new website to spotlight evidence gathered by the Republic of Kazakhstan regarding the fraud committed by Anatolie and Gabriel Stati: www.kzarbitration.com. The purpose of the website is to present the facts of the ongoing and complex legal case and provide easy access to the corresponding evidence. This public repository will increase transparency around the case and dispel the disinformation and lies perpetrated by these individuals against the Republic of Kazakhstan and its people. The evidence - in the form of court rulings, expert opinions, and other publicly available documents - confirms that the Statis have been engaged in a complex and multi-faceted fraud since at least 2006. The website explains how Anatolie and Gabriel Stati defrauded international investors of their money, falsified financial statements, fraudulently obtained audit opinions and pursued an international arbitration against the Republic of Kazakhstan to recoup from the S

TP Awards Schlumberger Sakarya Offshore Gas Field Phase-1 Contract15.10.2021 13:00:00 CEST | Press release

Schlumberger announced today a significant contract award by Turkish Petroleum (TP) for the engineering, procurement, construction and installation (EPCI) of end-to-end production solutions for the Sakarya gas field, Turkey’s largest gas reserve. The contract is awarded to Schlumberger and Subsea 7, as part of a consortium. The integrated project scope will cover subsurface solutions to onshore production, including well completions, subsea production systems (SPS), subsea umbilicals, risers, flowlines (SURF), and an early production facility (EPF). Schlumberger will deliver the well completions scope and the design, construction, and commissioning of the early production facility capable of handling up to 350 MMscfd of gas. The SPS and SURF scope will be delivered by OneSubsea®, the subsea technologies, production, and processing systems division of Schlumberger, and Subsea 7. “Schlumberger is uniquely positioned to integrate solutions from the subsurface to the processing facility, a

2021 Jeonju Bibimbap Festival Kicks Off Under the Theme of 'World Bibim Week'15.10.2021 09:00:00 CEST | Press release

Jeonju, a UNESCO Creative City of Gastronomy, announced that the 2021 Jeonju Bibimbap Festival, the typical gourmet festival of the city, is to be held on every weekend during October 9 - 31, 2021 in the vicinity of Jeonju Hyanggyo in Jeonju Hanok Village and online. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20211015005015/en/ UNESCO Creative City of Gastronomy Jeonju city has opened the 2021 Jeonju Bibimbap Festival. Under the title of World Bibim Week, this year's festival is designed and supervised by Ryu Jae-hyeon, Executive Director, focusing on a food festival to deliver the best taste while promoting sociability that is represented by ‘mixture’. It is to be held on every weekend during October 9 - 31, 2021 in the vicinity of Jeonju Hyanggyo in Jeonju Hanok Village and online. The four week-festival features ‘day’ programs on each weekend under different themes including Bibim Week (Day for Couples), Vitamin Week (D

Announcing the 2021 .eu Web Awards Winners!15.10.2021 09:00:00 CEST | Press release

Last night the 2021 .eu Web Awards ended in epic fashion at the Teatro Antico in Taormina, Italy. Hosted by EURid, the .eu Web Awards are a competition for the best .eu, .ею and .ευ websites. After last year’s virtual only event, we were pleased to once again celebrate the finalists and winners in person. View the hybrid event here. Hosted by Sally Bundock, esteemed journalist for BBC world news, the gala night brought us sincere joy reflected by the winners, combined with amazing performances by featured artists like THePETEBOX, Women Orchestra, MM Dance Company, Compagnia Joculares and the GAP’s Orchestra. The magical event ended with fireworks. “Being able to see real faces and raw emotion conveyed by the guests again made the night unforgettable” noted Giovanni Seppia, EURid’s External Relations Manager. “This is what the Web Awards is all about, bringing together people from various industries and backgrounds and honouring them for having chosen a .eu for their online presence.” P

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom