FDA grants breakthrough therapy designation for investigational medicine venetoclax in combination with MabThera/Rituxan in relapsed or refractory chronic lymphocytic leukaemia

• First in a new class of medicines designed specifically to activate apoptosis, allowing cancer cells to self-destruct
• Tenth breakthrough therapy designation for Roche’s portfolio of medicines

Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the US Food and Drug Administration (FDA) has granted breakthrough therapy designation to venetoclax in combination with MabThera/Rituxan (rituximab) for the treatment of people with relapsed or refractory chronic lymphocytic leukaemia (CLL). Venetoclax is an investigational medicine being developed in partnership with AbbVie.

The designation was based on results from the M13-365 study, which were presented at the Annual Meeting of the American Society of Hematology (ASH) in December 2015 (abstract #830). Venetoclax was previously granted breakthrough therapy designation by the FDA in April 2015 for the treatment of people with previously treated (relapsed or refractory) CLL with 17p deletion.

Breakthrough therapy designation is designed to expedite the development and review of medicines intended to treat serious or life-threatening diseases and to help ensure people have access to them through FDA approval as soon as possible. The combination of venetoclax and MabThera/Rituxan for the treatment of people with relapsed or refractory CLL is being further evaluated in the ongoing MURANO (GO28667) study.

About Venetoclax (RG7601, GDC-0199/ABT-199)

Venetoclax is an investigational small molecule designed to selectively bind and inhibit the BCL-2 protein, which plays an important role in a process called apoptosis (programmed cell death). It is believed that blocking BCL-2 may restore the signalling system that tells cancer cells to self-destruct. The BCL-2 protein is linked to the development of resistance in certain blood cancers and is expressed in chronic lymphocytic leukaemia (CLL) and non-Hodgkin’s lymphoma (NHL). In collaboration with AbbVie, venetoclax is being evaluated in a robust development program as a single agent or in combination with other medicines. There are ongoing Phase II and III studies for venetoclax in CLL, and Phase I and II studies are also ongoing in several other blood cancers, including indolent NHL, diffuse large B-cell lymphoma (DLBCL), acute myeloid leukaemia (AML) and multiple myeloma (MM).

About Roche in haematology

For more than 20 years, Roche has been developing medicines that redefine treatment in haematology. Today, we’re investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. In addition to approved medicines MabThera/Rituxan (rituximab) and Gazyva/Gazyvaro (obinutuzumab), Roche’s pipeline of investigational haematology medicines includes an anti-PDL1 antibody (atezolizumab/MPDL3280A), an anti-CD79b antibody drug conjugate (polatuzumab vedotin/RG7596), a small molecule antagonist of MDM2 (idasanutlin/RG7388) and in collaboration with AbbVie, a small molecule BCL-2 inhibitor (venetoclax/RG7601/GDC-0199/ABT-199). Roche’s dedication to developing novel molecules in haematology expands beyond oncology, with the development of the investigational haemophilia A treatment emicizumab (ACE910).

About Roche

Headquartered in Basel, Switzerland, Roche is a leader in research-focused healthcare with combined strengths in pharmaceuticals and diagnostics. Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and neuroscience. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Roche’s personalised healthcare strategy aims at providing medicines and diagnostics that enable tangible improvements in the health, quality of life and survival of patients. Founded in 1896, Roche has been making important contributions to global health for more than a century. Twenty-nine medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and chemotherapy.

In 2014, the Roche Group employed 88,500 people worldwide, invested 8.9 billion Swiss francs in R&D and posted sales of 47.5 billion Swiss francs. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com.

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