Investor Update

Basel, 29 September 2014

Roche presents encouraging update on metastatic bladder cancer data for anti-PDL1 medicine (MPDL3280A)

  • 52% response rate in PD-L1 positive metastatic urothelial bladder cancer (mUBC) patients treated with MPDL3280A
  • Median duration of response in PD-L1 positive UBC patients treated with MPDL3280A not yet reached
  • Median progression-free survival in PD-L1 positive UBC patients treated with MPDL3280A is 24 weeks

Roche (SIX: RO, ROG; OTCQX: RHHBY) presented an update on data for the use of its investigational cancer immunotherapy, MPDL3280A (anti-PDL1 or anti Programmed Death Ligand-1) in patients with mUBC at the European Society for Medical Oncology (ESMO) 2014 Congress in Madrid. With increased observation time in the study, MPDL3280A showed an objective response rate (ORR) of 52% in PD-L1-positive patient populations compared with the ORR presented at ASCO 2014 (43%). MPDL3280A continued to be well tolerated.1

The updated results from a phase I open-label study showed the investigational cancer immunotherapy MPDL3280A (anti-PDL1) shrank tumours (ORR) in 52 percent (17/33) of people who were mostly pretreated for mUBC and whose tumours were characterised as PD-L1 positive by a test being developed by Roche. In the overall mUBC population:

  • Complete responses (CRs) were observed in three patients
  • 19 of 22 (86 percent) responding patients had ongoing responses at the time of data cutoff
  • The median duration of response was not reached
  • Median progression-free survival (PFS) in PD-L1 positive patients treated with MPDL3280A was 24 weeks and was longer than median PFS in PD-L1 negative patients which was 8 weeks.

“The results further reinforce the rationale for using our breakthrough medicine MPDL3280A in advanced bladder cancer, a disease that has seen no major advances in nearly 30 years,” said Sandra Horning, M.D., Chief Medical Officer and head of Global Product Development. “These data also reflect our commitment to advance our clinical development programme in the field of cancer immunotherapy.”

The FDA granted MPDL3280A Breakthrough Therapy Designation in mUBC earlier this year. This designation is intended to expedite the development and review of medicines intended to treat serious diseases and to help ensure patients have access to them through FDA approval as soon as possible. Full results of the mUBC study will be presented at the European Society for Medical Oncology (ESMO) 2014 Congress in Madrid. (Abstract #8080, Monday, September  29, 11am, Sevilla Room)

Results of MPDL3280A in mUBC (Bellmunt et al.)

Efficacy data

  • MPDL3280A had an ORR of 52% observed in mostly platinum-pretreated PD-L1 positive (IHC 2/3) patients with mUBC
  • ORR of 14% (5 out of 36) observed in PD-L1 negative (IHC 0/1) patients
  • Rapid responses have been observed but so far median duration of response has not yet been reached
  • 19 of 22 responding patients had ongoing responses at the time of data cutoff.  Median duration of response has not yet been reached
  • Median progression–free survival (PFS) of 24 weeks so far in IHC 2/3 patients and 8 weeks in IHC 0/1 patients

Safety data

  • MPDL3280A was well tolerated
  • 5% of patients experienced a Grade 3-4 treatment-related adverse event (AE)
  • Renal toxicity has not been observed in MPDL3280A treated patients to date
  • There were no grade 5 treatment-related AEs

About MPDL3280A (anti-PDL1)

MPDL3280A (also known as anti-PDL1) is an investigational monoclonal antibody designed to interfere with a protein called PD-L1. MPDL3280A is designed to target PD-L1 expressed on tumour cells and tumour-infiltrating immune cells, preventing it from binding to PD-1 and B7.1 on the surface of T cells. By inhibiting PD-L1, MPDL3280A may enable the activation of T cells, restoring their ability to effectively detect and attack tumour cells.

About Roche in Cancer Immunotherapy

For more than 30 years, Roche has been developing medicines with the goal to redefine treatment in oncology. Today, we are investing more than ever in our effort to bring innovative treatment options that help a person’s own immune system fight cancer. Our cancer immunotherapy research and development programme comprises more than 20 investigational candidates, which include the evaluation of biomarkers to determine which people may be appropriate candidates for our medicines.

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-four medicines developed by Roche are included in the World Health Organisation Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and chemotherapy.

In 2013 the Roche Group employed over 85,000 people worldwide, invested 8.7 billion Swiss francs in R&D and posted sales of 46.8 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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References
1. Bellmunt J et al., Inhibition of PD-L1 by MPDL3280A leads to clinical activity in patients with metastatic urothelial bladder cancer (UBC), Abstract number: #6984. 29 September 2014, XX CET, Madrid, Spain