Investor Update

Basel, 09 December 2014

Roche presents data for hemophilia A therapy ACE910

  • Phase I data indicate antibody therapy ACE910 is well tolerated and has a promising efficacy profile in severe hemophilia A
  • Early data indicate encouraging reduction in bleeding rates in all patients
  • ACE910 shows promise as a preventive treatment for hemophilia A, irrespective of the presence of factor VIII inhibitors

Roche (SIX: RO, ROG; OTCQX: RHHBY) presented data from a first-in-patient study of the novel humanised bispecific antibody ACE910 (RG6013) in hemophilia A patients during the 56th American Society of Hematology (ASH) Annual Meeting. Results from the open-label phase I study, conducted by Chugai, showed once-weekly subcutaneous administration of ACE910 was well tolerated and possessed a promising efficacy profile in hemophilia A patients. Results showed that ACE910 significantly reduced the annualised bleeding rate (ABR) during treatment, compared to the rate seen in the six-month period prior to study enrolment.

Importantly, ACE910 was effective irrespective of the presence of inhibitors to blood clotting factor VIII (FVIII). FVIII is an essential blood clotting protein required for the normal functioning of the coagulation system.  People with hemophilia A lack FVIII and therefore need to get it injected into their bloodstream. Approximately 30% of severe hemophilia A patients develop inhibitors to injected FVIII, rendering current FVIII replacement therapies ineffective.

As detailed in the table below, all patients with prior bleeding events experienced reductions in their ABR, with the rate of reduction reaching 100% for patients in each of the three cohorts.

“ACE910, designed to overcome the limitations of intravenous FVIII replacement therapy, may offer an effective, convenient prophylactic treatment option for hemophilia A patients with or without inhibitors to factor VIII,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “This exciting expansion into hemophilia A represents Roche’s commitment to benign as well as malignant hematology, and based on the study outcome, we plan to move into more advanced clinical trials of this promising compound in 2015.”

The objectives of the phase I investigations were to assess the safety and efficacy of ACE910 in this patient group, as well as the pharmacokinetics and pharmacodynamics of the drug. Full results of the data set were presented at the American Society of Hematology (ASH) Annual Meeting 2014 in San Francisco (Abstract #691).

About ACE910

ACE910 is an investigational humanised bispecific monoclonal antibody engineered to simultaneously bind factors IXa and X and thereby mimic the FVIII cofactor function in hemophilia A patients who are deficient in this blood clotting protein. By mimicking FVIII cofactor function, ACE910 may promote the blood coagulation reaction under FVIII-deficient conditions and help overcome medical problems such as the development of FVIII inhibitors and the need for frequent venous access which are regularly encountered in hemophilia A patients. ACE910 was created by Chugai Pharmaceutical Co., Ltd. and was opted into by Roche earlier in 2014.

About Roche in hematology

For more than 20 years, Roche has been developing medicines with the goal of redefining treatment in hematology. 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 products MabThera/Rituxan and Gazyva/Gazyvaro, Roche’s pipeline of investigational hematology medicines includes the antibody-drug conjugate polatuzumab vedotin, a small molecule antagonist of MDM2 and, in collaboration with AbbVie, the small molecule BCL-2 inhibitor venetoclax.

Furthermore, Roche’s dedication to developing novel molecules in hematology expands beyond oncology, with the development of the investigational hemophilia A treatment ACE910.

About Roche in Oncology

Roche has been working to transform cancer care for more than 50 years, bringing the first specifically designed anticancer chemotherapy drug, 5-Flourouracil (5-FU), to patients in 1962. Roche’s remains committed to developing innovative medicines and diagnostics for cancer.

The Roche Group’s portfolio of innovative cancer medicines includes Avastin (bevacizumab), Erivedge (vismodegib), Gazyva/Gazvyaro (obinutuzumab), Herceptin (trastuzumab), Kadcyla (trastuzumab emtamsine), MabThera/Rituxan (rituximab), Perjeta (pertuzumab), Tarceva (erlotinib), Xeloda (capecitabine) and Zelboraf (vemurafenib). In addition, the Group has a robust oncology pipeline focusing on new therapeutic targets and novel combination strategies.

In addition to its innovative portfolio of cancer medicines, Roche is constantly developing new diagnostic tests that will have a significant impact on disease management for cancer patients. Within Roche there are more than 350 pharma and diagnostics collaborations, most of which are in the field of oncology. With a broad portfolio of tumour markers for prostate, colorectal, liver, ovarian, breast, stomach, pancreatic and lung cancer, as well as a range of tissue and molecular oncology tests that contribute to personalised cancer care today, Roche is leading a new era of innovation in the fight against cancer.

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.

All trademarks used or mentioned in this release are protected by law.