Bayer receives U.S. FDA breakthrough therapy designation for copanlisib for the treatment of marginal zone lymphoma

BayerBayer announced today that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for copanlisib (Aliqopa™) for the treatment of adult patients with relapsed marginal zone lymphoma (MZL) who have received at least two prior therapies. MZL is an indolent form of non-Hodgkin's Lymphoma (iNHL) and accounts for about 10% of all non-Hodgkin's Lymphoma in the U.S. The Breakthrough Therapy Designation was based on data from the MZL subgroup of the pivotal Phase II trial CHRONOS-1 that led to accelerated approval of copanlisib in the U.S. for the treatment of adult patients with relapsed follicular lymphoma (FL, the most common histological subtype of iNHL) who have received at least two prior systemic therapies. Copanlisib is a pan-class intravenous phosphatidylinositol-3-kinase (PI3K) inhibitor with inhibitory activity predominantly against the PI3K-alpha and PI3K-delta isoforms expressed in malignant B cells. The compound is currently not approved by the European Medicines Agency (EMA) or other authorities outside of the U.S.

"The clinical evidence suggests that copanlisib may address an unmet medical need by providing physicians and MZL patients with a therapy in a relapsed setting where there are no approved treatments," said Dr. Scott Z. Fields, M.D., Senior Vice President and Head of Oncology Development at Bayer's Pharmaceutical Division. "We will continue working closely with the FDA in order to bring copanlisib to these underserved patients as soon as possible."

The FDA's Breakthrough Therapy Designation is intended to expedite the development and review of drug candidates that treat serious or life-threatening diseases or conditions, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies.

Bayer is conducting two additional Phase III studies - CHRONOS-3 and CHRONOS-4 - to evaluate the efficacy and safety of copanlisib in combination with other therapies in iNHL (including MZL) patients who have relapsed following one or more prior therapies.

Clinical evidence for copanlisib in MZL

In the Phase II study 16439 part B (CHRONOS-1), copanlisib was investigated in iNHL patients including 23 patients with relapsed or refractory MZL, who have received at least two prior therapies. An 18 month follow-up analysis of CHRONOS-1 showed an ORR in the full set analysis (FAS) population of 60.6% and in the MZL histology an ORR of 78.3% (n=23). The overall safety of copanlisib confirmed the expected tolerability and manageable safety profile.

About marginal zone lymphoma (MZL)

MZL is an indolent form of non-Hodgkin's Lymphoma (iNHL) and accounts for about 10% of all non-Hodgkin’s Lymphoma in the U.S. Typically chemotherapy and/or immunotherapy are often chosen for the treatment of MZL. While initial therapies are often successful, there continues to be an unmet need for relapsed (advanced) MZL.

About copanlisib (Aliqopa) injection

Copanlisib is a pan-PI3K inhibitor with predominant activity against the PI3K-alpha and PI3K-delta isoforms expressed in malignant B-cells, developed by Bayer. Copanlisib is approved in the U.S. for the treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies.

The U.S. FDA approved copanlisib under the brand name Aliqopa™, in September 2017 for the treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies. Accelerated approval was granted for this indication based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Copanlisib has been shown to induce tumor cell death by apoptosis and inhibition of proliferation of primary malignant B cell lines. Copanlisib inhibits several key cell-signaling pathways, including B-cell receptor signaling, CXCR12 mediated chemotaxis of malignant B-cells, and NFκB signaling in lymphoma cell lines.

The broad clinical development program for copanlisib also includes ongoing Phase III studies in indolent NHL (iNHL) patients who have relapsed or are refractory to prior therapies. The CHRONOS-3 Phase III study is evaluating copanlisib in combination with rituximab in relapsed iNHL and the CHRONOS-4 Phase III study is evaluating copanlisib in combination with standard immunochemotherapy in relapsed iNHL. More information about these trials can be found at www.clinicaltrials.gov.

Copanlisib is currently not approved outside of the U.S.

About Oncology at Bayer

Bayer is committed to delivering science for a better life by advancing a portfolio of innovative treatments. The oncology franchise at Bayer includes five marketed products and several other assets in various stages of clinical development. Together, these products reflect the company's approach to research, which prioritizes targets and pathways with the potential to impact the way that cancer is treated.

About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2018, the Group employed around 117,000 people and had sales of 39.6 billion euros. Capital expenditures amounted to 2.6 billion euros, R&D expenses to 5.2 billion euros.

Most Popular Now

AstraZeneca takes next steps towards broad and equ…

AstraZeneca has taken the next steps in its commitment to broad and equitable global access to the University of Oxford’s COVID-19 vaccine, following landmark agreements ...

Johnson & Johnson announces acceleration of it…

Johnson & Johnson (NYSE: JNJ) (the Company) today announced that through its Janssen Pharmaceutical Companies (Janssen) it has accelerated the initiation of the Phase 1/2...

Low-cost dexamethasone reduces death by up to one …

In March 2020, the RECOVERY (Randomised Evaluation of COVid-19 thERapY) trial was established as a randomised clinical trial to test a range of potential treatments for C...

Sanofi invests to make France its world class cent…

Sanofi detailed plans on how the Company will make significant investments in France to increase its vaccines research and production capacities, and contribute in respon...

Calquence showed promising clinical improvement in…

Results published in Science Immunology showed that Calquence (acalabrutinib), a Bruton’s tyrosine kinase (BTK) inhibitor, reduced markers of inflammation and improved cl...

Super-potent human antibodies protect against COVI…

A team led by Scripps Research has discovered antibodies in the blood of recovered COVID-19 patients that provide powerful protection against SARS-CoV-2, the coronavirus ...

New consortium EUbOPEN will provide tools to unloc…

Almost twenty years after deciphering the human genome, our understanding of human disease is still far from complete. One of the most powerful and versatile tools to bet...

AstraZeneca to supply Europe with up to 400 millio…

AstraZeneca has reached an agreement with Europe's Inclusive Vaccines Alliance (IVA), spearheaded by Germany, France, Italy and the Netherlands, to supply up to 400 milli...

Up to 45 percent of SARS-CoV-2 infections may be a…

An extraordinary percentage of people infected by the virus behind the ongoing deadly COVID-19 pandemic never show symptoms of the disease, according to the results of a ...

Researchers identify potent antibody cocktail to t…

Researchers at the University of Maryland School of Medicine (UMSOM) evaluated several human antibodies to determine the most potent combination to be mixed in a cocktail...

Gilead announces results from Phase 3 Trial of rem…

Gilead Sciences, Inc. (Nasdaq: GILD) announced topline results from the Phase 3 SIMPLE trial in hospitalized patients with moderate COVID-19 pneumonia. This open-label st...

Mayo finds convalescent plasma safe for diverse pa…

Mayo Clinic researchers and collaborators have found investigational convalescent plasma to be safe following transfusion in a diverse group of 20,000 patients. The findi...