Investor Update

Basel, 03 June 2013

Promising data from the Bcl-2 inhibitor GDC-0199/ABT-199 in hematology presented at ASCO

Late stage development underway in chronic lymphocytic leukemia

Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced promising phase 1 data from a study of GDC-0199/ABT-199, also known as RG7601, in people with relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL) and relapsed/refractory (R/R) non-Hodgkin lymphoma (NHL). Data were presented at the 49th Annual Meeting of the American Society of Clinical Oncology (ASCO) in Chicago. Relapsed cancer refers to a cancer that returns after a period of remission while refractory cancer refers to a cancer that is no longer responding to treatment.

GDC-0199/ABT-199 is designed to work by interfering with the process by which some cancer cells survive, thereby promoting a natural death process known as apoptosis.

In the R/R CLL arm of the study (55 evaluable patients), the investigators concluded that GDC-0199/ABT-199 is highly active achieving an 84% overall response rate. 18% of patients achieved a complete remission (CR) or CR with incomplete count recovery and 65% achieved a partial remission (PR). It is important to note that these results were independent of high-risk markers such as 17p deletion and fludarabine (F)-refractory disease.  In fact, 81% and 78% of patients with 17p deletion and F-refractory CLL, respectively, achieved at least a PR. The most common adverse events (AEs), which occurred in more than 15% of patients, were diarrhea (41%), neutropenia (39%), nausea (38%), fatigue (29%), upper respiratory tract infection (27%), cough (23%) and thrombocytopenia (18%). Grade 3/4 AEs occurring in more than 5 patients were neutropenia (38%), thrombocytopenia (11%) and tumor lysis syndrome (TLS) (11%). Adverse events of TLS occurred in 3/3 patients in cohort 1 and 3/53 patients with the modified stepped dosing schedule.  One of the modified schedule events was fatal and occurred within 48 hours of dose-escalation to 1200 mg in a patient with laboratory evidence of TLS.

“In hematology, we have a strong heritage thanks to our experience with MabThera/Rituxan and we are building on this heritage by looking for innovative ways to improve patient outcomes, especially for those for whom current treatments offer little benefit,” said Richard Scheller, Head, Genentech Research and Early Development. “For people with CLL who have specific and difficult to treat disease, we are looking to initiate our registration trial with GDC-0199 as soon as possible.”

In the R/R NHL arm of the study (32 patients), the investigators concluded that GDC-0199/ABT-199 is highly active, particularly in mantle cell lymphoma (MCL) and that further investigation of GDC-0199/ABT-199 as a single-agent and in combination is warranted. For the 29 patients evaluated for efficacy, the overall response rate was 53%, with 9% experiencing CRs and 44% experiencing PRs.  In mantle cell lymphoma (MCL) the overall response rate was 100%. The most common AEs occurring in 15% of patients or more, were nausea (41%), diarrhea (28%), vomiting (19%), fatigue (19%), pyrexia (19%), cough (19%) and neutropenia, thrombocytopenia, constipation, dyspepsia, back and extremity pain, each in 16% of patients.

The U.S. Food and Drug Administration (FDA) recently accepted Genentech and AbbVie’s amended clinical trial protocols for studies of GDC-0199/ABT-199 in patients with CLL and enrollment for GDC-0199/ABT-199 clinical trials in CLL, NHL and multiple myeloma has been re-instated. Genentech and AbbVie expect to move GDC-0199/ABT-199 into later-stage clinical trials in the near future.

About GDC-0199/ABT-199

GDC-0199/ABT-199, also known as RG7601, is a novel small molecule being developed in collaboration with AbbVie and is designed to selectively block the function of Bcl-2 proteins and with the goal of enhancing cell death activity (apoptosis). Bcl-2 proteins play a central role in enhancing cell death activity and are thought to impact tumor formation, growth and resistance. They are expressed at high levels in non-Hodgkin lymphoma (NHL), chronic lymphocytic leukemia (CLL) and in other B-cell neoplasms.

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, infectious diseases, inflammation, metabolism 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 diagnostic tools that enable tangible improvements in the health, quality of life and survival of patients. In 2012 Roche had over 82,000 employees worldwide and invested over 8 billion Swiss francs in R&D. The Group posted sales of 45.5 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

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