Investor Update

Basel, 16 May 2013

Roche to present data on investigational cancer immunotherapy anti-PDL1 antibody MPDL3280A at ASCO 2013

  • Encouraging results from six ASCO abstracts, including biomarker results, to be presented
  • MPDL3280A to proceed directly into pivotal studies

Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the company will present important new data on its anti-PDL1 antibody MPDL3280A (also known as RG7446) at the 49th Annual Meeting of the American Society of Clinical Oncology (ASCO), May 31 to June 4, 2013, in Chicago. MPDL3280A is an investigational medicine designed to make cancer cells more vulnerable to the body’s own immune system by interfering with a protein called PD-L1 (Programmed Death-Ligand 1). It was highlighted as part of ASCO’s official press programme.

“Activating the immune system to eliminate cancer cells has long been a goal of cancer research and we are excited to be at the forefront of investigating this approach,” said Ira Mellman Ph.D., vice president, Research Oncology at Genentech Research and Early Development. “We chose to target the PD-L1 protein on tumour cells because this approach has the potential to maximise durable response and allows for the most direct connection to PD-L1 for the purposes of biomarker identification and diagnostic testing. We have also modified the binding region of our antibody to eliminate a process called antibody-dependent cell-mediated cytotoxicity, or ADCC. This modification has the potential to enhance efficacy and safety.”

Data on MPDL3280A will be presented in five oral presentations and one poster presentation:

  • A study of MPDL3280A, an engineered PD-L1 antibody in patients with locally advanced or metastatic tumours. Abstract #3000. ASCO press briefing Wednesday, May 15. Oral abstract presentation, Monday, June 3, 3:00 PM – 3:15 PM CDT in S406.
  • Biomarkers and associations with the clinical activity of PD-L1 blockade in a MPDL3280A study. Abstract #3001. Oral abstract presentation, Monday, June 3, 3:15 PM – 3:30 PM in S406.
  • Clinical activity, safety and biomarkers of MPDL3280A, an engineered PD-L1 antibody in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC). Abstract #8008. Oral abstract presentation, Monday, June 3, 5:30 PM – 5:45 PM CDT in E Hall D2.
  • Clinical activity, safety and biomarkers of MPDL3280A, an engineered PD-L1 antibody in patients with locally advanced or metastatic melanoma (mM). Abstract #9010. Clinical Science Symposium abstract presentation, Sunday, June 2, 10:15 AM – 10:30 AM CDT in E Arie Crown Theatre.
  • Clinical activity, safety and biomarkers of MPDL3280A, an engineered PD-L1 antibody in patients with metastatic renal cell carcinoma (mRCC). Abstract #4505. Oral abstract presentation, Saturday, June 1, 2:45 PM – 3:00 PM CDT in E Arie Crown Theatre.
  • Clinical activity, safety, and biomarkers of MPDL3280A, an engineered PD-L1 antibody in patients with locally advanced or metastatic CRC, gastric cancer (GC), SCCHN, or other tumours. Abstract #3622. Poster presentation, Sunday, June 2, 8:00 AM – 11:45 AM in S Hall A2.

Full session details of the 2013 Annual Meeting can be found through the ASCO iPlanner, here: http://iplanner.asco.org/am2013.

Based on the results of these studies, Roche and Genentech are initiating pivotal studies investigating MPDL3280A in non-small cell lung cancer (NSCLC). These studies will incorporate an investigational companion diagnostic. Roche and Genentech are also exploring additional studies of MPDL3280A in other cancer types, alone and in combination with other medicines, including Avastin (bevacizumab) and Zelboraf (vemurafenib).

Selected efficacy data to be presented at ASCO are included in the tables below. In addition to these data, updated data in other tumours (abstract #3622) will be presented.


Overall Response Rates 1)
Overall Phase 1 experience (Abstract #3000)21% (29/140)
Metastatic Non-Small Cell Lung Cancer (Abstract #8008)22% (9/41)
Metastatic Melanoma (Abstract #9010)29% (11/38)
Renal Cell Carcinoma (Abstract #4505)13% (6/47) 2)

1 Efficacy evaluable subjects dosed prior to August 1, 2012; data cutoff February 1, 2013.
2 Renal cell carcinoma data not yet mature

Overall Response Rates 1)

All-comers 2)PD-L1 positive 3)PD-L1 negative 3)
Overall Phase 1 experience (Abstract #3000)21% (29/140)36% (13/36)13% (9/67)

1 Efficacy evaluable subjects dosed prior to August 1, 2012; data cutoff February 1, 2013.
2 All-comers includes patients with unknown PD-L1 status
3 PD-L1 status based on Roche PD-L1 IHC

Among the 29 responders, 26 continued to respond at the last assessment (time on study of 3 to 15+ months).

No maximum tolerated dose, dose-limiting toxicities or treatment-related deaths were observed during dose escalation. The majority of adverse events were transient Grade 1/2 events. The most common events included increased liver enzymes (AST or ALT), inflammation of the large intestine (colitis), and high blood glucose (hyperglycemia). Selected safety data to be presented at ASCO are shown below:

Grade 3/4 Adverse Events Regardless of Attribution* – All Dose Cohorts (n=171)
Grade 3/4 Adverse Events% (n)
All Grade 3/4 Events43% (73/171)*
Hyperglycemia5% (9/171)
Fatigue4% (7/171)
Increased ALT3% (5/171)
Dyspnea3% (5/171)
Hypoxia3% (5/171)

*Adverse events regardless of whether they were assessed as being due to, disease, other factors or MPDL3280A. Data cut off Feb 1, 2013.

About cancer immunotherapy and MPDL3280A

Cancer immunotherapy is the use of the body’s own immune system to attack and eliminate cancer cells. Research at Roche’s Genentech and elsewhere adds to a growing body of evidence that suggests cancer immunotherapy warrants further clinical study.

Anti-PDL1 antibody MPDL3280A is an investigational medicine designed to make cancer cells more vulnerable to the body’s immune system by interfering with a protein called PD-L1. PD-L1 is found on the surface of cells in tumours and is believed to act as a “stop sign,” preventing the immune system from destroying cancer cells. MPDL3280A is being studied in clinical trials to understand whether blocking PD-L1 (“removing the stop sign”) will help the immune system respond to cancer.

MPDL3280A is being studied in clinical trials alone and with other medicines that directly interfere with how cancer grows and spreads.

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 www.roche.com.

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