Company Of The Year – Roche: Surging Ahead Via Innovation
Roche’s Pharmaceuticals and Diagnostics Divisions continue to generate strong performances significantly driven by new product launches, including four new oncology treatments and the company’s first cancer immunotherapy medicine.
F. Hoffmann-La Roche ltd.
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|Product||2016 Sales||2015 Sales|
All sales are in millions of dollars and were translated using the Federal Reserve Board’s average rate of exchange in 2015: SFr 0.9848.
|1H 2017||1H 2016|
All sales are in millions of dollars and were translated using the Federal Reserve Board’s average rate of exchange in 2015: SFr 0.9848.
As Med Ad News’ Company of the Year for 2017, Roche is just the third industry entity to garner that distinction at least three times in this magazine’s 25-year history of citing such recognition.
Roche, which remains atop the oncology field, is expected by industry analysts to challenge all pharma/biotech companies for the global sales throne in 2022. Roche currently leads the biologics market, holds the top spot of the “pipeline value creation” ranking proving its leadership ambitions beyond oncology, and is forecast to be the largest spender on pharmaceutical R&D in 2022. The impact of biosimilars will be a decisive factor for the future of Roche and its trio of mega-blockbuster anti-cancer brands that accounted for three of the world’s top eight prescription drug sellers in 2016. Newer products with blockbuster potential include Tecentriq (atezolizumab), Venclexta (venetoclax), and Ocrevus (crelizumab).
In 2016, Roche invested CHF 9.92 billion ($10.07 billion) in research and development, and generated sales of CHF 50.58 billion ($51.36 billion). All of Roche’s financial targets for 2016 were achieved. Pharmaceuticals sales increased 3 percent, and Diagnostics sales rose 7 percent – again well above market growth, according to management. Positive financial results continued to be posted by Roche throughout the first half of 2017, with the second-half results anticipated to continue on a growth trend.
The Roche Group is active in 100-plus countries and employed more than 94,000 people during last year. U.S.-based Genentech is a wholly owned member of the Roche Group. In addition, Roche is the majority shareholder of Japan’s Chugai Pharmaceutical, which is recognized within this magazine as one of the industry’s top 50 companies. Roche provides 100-plus medicines, over 140 diagnostic instruments, and more than 850 different tests to patients, laboratories, and healthcare professionals.
Roche’s product pipeline has made “very good advances” during the past couple of years, according to management. Within a short period during 2016, the company launched four new medicines to treat aggressive cancers – Cotellic for skin cancer, Alecensa and Tecentriq for lung cancer, Venclexta for leukemia, and Tecentriq for bladder cancer. The Diagnostics Division also introduced various key new instruments and tests, including the successful 2016 launch of the immunochemistry instrument cobas e 801. Additionally, Roche was granted five FDA breakthrough therapy designations during 2016.
Management says Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalized healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible, company executives say. Roche remains the world’s largest biotechnology company and a global leader in in vitro diagnostics and tissue-based cancer diagnostics, as well as a front-runner in diabetes management.
Roche is regarded as the world leader in oncology as the company strives to advance new approaches to better treat cancer. During 2016, the Group’s first immunotherapeutic medicine Tecentriq won FDA approval for the treatment of bladder and lung cancer. Representing the first major improvement in bladder cancer treatment options in 30 years, this medicine additionally appreciably extends the life expectancy of people with advanced-stage lung cancer.
Roche has undertaken a broad, ongoing effort to advance the personalization of cancer immunotherapy by delivering treatment options tailored to the specific immune biology associated with a person’s tumor. In pursuit of this goal, Roche is developing 20 cancer immunotherapy medicines across nine types of cancer and in more than 50 combinations with other medicines. Roche is dedicated to advancing the science of cancer immunotherapy and exploring multiple biomarker approaches including PD-L1 immunohistochemistry, tumor gene expression, RNA sequencing, and tumor mutational burden (TMB).
For the ninth consecutive year, Roche was recognized during September 2017 as Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry index of the Dow Jones Sustainability Indices (DJSI). This recognition is based on an in-depth analysis of economic, social, and environmental performance. The DJSI family of indices serves as a benchmark for investors who integrate sustainability considerations into their portfolios. Roche performed particularly well in the categories addressing the burden of healthcare costs, ethical marketing practices and climate strategy.
“Roche is strongly positioned for the future,” says Dr. Severin Schwan, CEO of Roche. “We are the world’s largest biotech company, the leader in cancer therapy, equipped with one of the best product pipelines in the industry and No. 1 in laboratory diagnostics. I am confident that Roche will continue its 120-year success story.”
2017 Performance & Outlook
During the first six months of 2017, Roche’s Group sales increased 5 percent to CHF 26.34 billion ($26.75 billion). Diluted core EPS advanced 6 percent year over year to CHF 8.23 ($8.36), ahead of sales. According to management, core EPS growth reflects the strong underlying business performance. IFRS net income improved 2 percent compared to first-half 2016 to CHF 5.58 billion ($5.66 billion). IFRS net income was impacted by intangible asset impairments. These increased by CHF 0.7 billion ($711 million) net of taxes, notably from the partial impairment of the Esbriet product intangible.
In the Pharmaceuticals Division, combined sales for the first two quarters of 2017 rose 5 percent to CHF 20.52 billion ($20.84 billion). Roche’s recently launched medicines Tecentriq, Ocrevus, and Alecensa contributed CHF 0.5 billion ($508 million) of new sales, accounting for half of the division’s growth. In addition, Perjeta continued to generate a strong sales performance. Growth was partially offset by decreased sales of Tarceva, Tamiflu, and Pegasys. U.S. sales increased 8 percent, paced by Tecentriq, Xolair, Rituxan/MabThera, and Ocrevus, which was recently launched for treating relapsing and primary progressive forms of multiple sclerosis. In Europe, first-half 2017 sales were stable, according to management. Growth of Perjeta and Actemra/RoActemra sales was offset by decreased sales of Avastin. In the International region, sales improved 5 percent, led by the Latin America and Asia-Pacific subregions. Sales in Japan during first-half 2017 were stable. Growth of Alecensa sales (+42 percent) was partially offset by Avastin (-3 percent), which was negatively affected by the biannual government price cuts during April 2016.
Diagnostics Division sales improved 5 percent to CHF 5.82 billion ($5.91 billion) for the first six months of 2017. Centralized and Point of Care Solutions (+8 percent) was the top contributor, led by the growth of its immunodiagnostics business (+13 percent). In regional terms, growth was paced in particular by Asia-Pacific (+13 percent), with continued strong growth in China (+20 percent). According to Roche, sales rose 3 percent in EMEA, 8 percent in Latin America, 1 percent in North America, and 2 percent in Japan.
Core operating profit advanced by 3 percent in the Pharmaceuticals Division and by 5 percent in the Diagnostics Division during first-half 2017. The growth rates of each division were impacted by the base effect of income during 2016 from changes to the Group’s Swiss pension plans, partially offset by income from older medicine divestments in the first six months of 2017.
Based on the first-half results, Roche expects full-year 2017 sales to grow by mid-single digits, at constant exchange rates. Additionally, core earnings per share are targeted to grow broadly in line with sales, at constant exchange rates. Management expects Roche to further increase its dividend in Swiss francs.
Roche’s key pharmaceutical products in 2017 include Herceptin, Perjeta, and Kadcyla for HER2-positive breast cancer and HER2-positive metastatic gastric cancer (Herceptin only). Together the products generated 6 percent growth in the first six months of 2017. Herceptin sales increased 3 percent to CHF 3.54 billion ($3.6 billion), led by growth in the United States and Brazil, as well as by additional reimbursement approvals and broader use in China. Sales of Perjeta – up 17 percent globally to CHF 1.07 billion ($1.09 billion) – grew in all regions following increased demand in the neoadjuvant and metastatic settings, particularly in Europe, the United States, and the International region. Kadcyla worldwide sales advanced 9 percent over first-half 2016 to CHF 443 million ($450 million), led by the International region (+47 percent).
For forms of blood cancer, rheumatoid arthritis, and certain types of vasculitis, Rituxan/MabThera global sales continued to rise, up 3 percent to CHF 3.84 billion ($3.9 billion). The performance was driven by growth in the oncology and immunology segments. The largest sales increases were recorded in the United States and the International region. Growth in China was supported by additional reimbursement approvals as well as expanded regional access.
Another Roche blockbuster oncology medicine is Avastin for advanced colorectal, breast, lung, kidney, cervical and ovarian cancer, and relapsed glioblastoma (a type of brain tumor). First-half 2017 worldwide sales for the anti-cancer medicine were down 1 percent to CHF 3.41 billion ($3.46 billion). In the United States, where Avastin is already broadly used in its approved indications, sales decreased 3 percent, mainly due to growing use of cancer immunotherapy medicines in lung cancer. Sales continued to grow in the International region (+11 percent), especially in China, where sales improved due to broader market penetration in the lung and colorectal cancer setting. Sales in Europe, down 5 percent, were impacted by the removal of the reimbursement for breast cancer in France.
Marketed for rheumatoid arthritis, forms of juvenile idiopathic arthritis and giant cell arteritis, Actemra/RoActemra global sales in the first six months of 2017 climbed up 13 percent to CHF 922 million ($936 million). Sales growth was reported for all regions, notably in the United States (+16 percent) and Europe (+15 percent). In Japan, sales rose 7 percent due to steady growth in demand for the subcutaneous formulation. According to Roche, increasing use of Actemra/RoActemra (tocilizumab) as a single agent and of the subcutaneous formulation remained key growth drivers worldwide. Actemra/RoActemra remained the leading rheumatoid arthritis monotherapy in the five largest EU markets.
Sales for the idiopathic pulmonary fibrosis medicine Esbriet improved 16 percent to CHF 418 million ($424 million). First-half 2017 sales continued to expand, fueled by growth in the United States (+19 percent) and the International region (+34 percent). According to Roche, increased investments in patient education about urgency to treat is a key area in the U.S. and the EU and resulted in growth driven by penetration into moderate and mild patient segments.
Global sales during the 2017 first half for Gazyva/Gazyvaro – indicated for chronic lymphocytic leukemia (CLL) and rituximab-refractory follicular lymphoma – rose 44 percent to CHF 133 million ($135 million). Sales expanded in all regions where the product has been introduced, despite increasing competition in the CLL marketplace.
Recently launched Roche medicines recorded strong sales performances during first-half 2017. Tecentriq sales amounted to CHF 237 million ($241 million), driven mainly by uptake in the United States in metastatic bladder cancer and in metastatic non-small cell lung cancer (NSCLC). Ocrevus, which was launched in the United States during April 2017, met strong demand in both indications, relapsing and primary progressive multiple sclerosis. The product’s sales totaled CHF 192 million ($195 million). Roche says Ocrevus is being used across various stages of these diseases and is a key U.S. growth driver. Alecensa sales reached CHF 148 million ($150 million) for first-half 2017. Alecensa demonstrated very good uptake in the United States and sales growth remained strong in Japan. The product is intended for people with ALK-positive advanced NSCLC whose disease has progressed on, or who are intolerant to crizotinib.
Centralized and Point of Care Solutions was the largest contributor to the Diagnostics Division’s first-half 2017 sales performance – up 8 percent to CHF 3.46 billion ($3.51 billion) – led by the Asia-Pacific region. Integrated Serum Work Area solutions, comprising the immunodiagnostics (+13 percent) and clinical chemistry (+3 percent) segments, were the primary growth drivers. The cobas e 801 module, for immunodiagnostics, was introduced to the U.S. market during the 2017 first half. According to Roche, cobas e 801 is a core element of the integrated laboratory that doubles the currently available immunochemistry testing capacity over the same floor space, requires only low sample volume, and delivers fast results. The module has been very well received by customers and contributes to the strong performance of this business area, management notes.
First-half 2017 sales in Molecular Diagnostics rose 1 percent to CHF 920 million ($934 million), with 2 percent growth in the underlying molecular business and a decrease in the sequencing business. Regional growth was spurred on by the North America and EMEA markets. Sales in the blood screening and HPV screening businesses advanced 3 percent and 2 percent respectively. In virology, which includes Roche’s portfolio for diagnosis and monitoring of hepatitis B, hepatitis C, and HIV, sales decreased 1 percent, impacted by a base effect from prior-year strong HCV sales.
Tissue Diagnostics sales increased 13 percent to CHF 485 million ($492 million) versus the first-half 2016 performance, driven by strong sales in North America and EMEA. Sales in the advanced staining and primary staining portfolios increased 9 percent and 15 percent respectively. The companion diagnostics business rose 40 percent in first-half 2017. The CINtec Histology and CINtec PLUS Cytology tests recorded continued good growth (+18 percent) according to Roche, also supported by the recent U.S. market introduction of the CINtec Histology 510(k) product that was granted FDA approval in April.
Diabetes Care sales in the 2017 first half decreased 4 percent to CHF 962 million ($977 million), impacted by challenging market conditions in North America (-18 percent). Sales improved 3 percent in Latin America, but were down 1 percent in EMEA, 5 percent in the Asia-Pacific and 5 percent in Japan. Rollout of the Accu-Chek Guide system, Roche’s new blood glucose monitoring platform initially launched during 2016, extended into additional major markets including the United States and Germany.
Roche acquired all shares of mySugr GmbH – which specializes in all-around care for people with diabetes – as reported at the end of June. With the acquisition of mySugr, Roche is further building its platform for digital diabetes management, company executives say. This is a new patient-centered digital health service in diabetes care, which helps healthcare providers using existing Accu-Chek tools to make better and faster therapy decisions while improving patient communication.
Product Approvals/Launches & Pipeline Updates During 2017
Roche received important new medicine approvals in Pharmaceuticals from various regulatory agencies during 2017. For example, Tecentriq won FDA accelerated approval in April for treating people with locally advanced or metastatic urothelial carcinoma who are not eligible for cisplatin chemotherapy. Tecentriq is the first cancer immunotherapy approved in advanced bladder cancer as initial treatment for those unable to receive cisplatin chemotherapy. The medicine was previously FDA-approved for people with locally advanced or mUC who have disease progression during or following any platinum-containing chemotherapy, or within 12 months of receiving chemotherapy before surgery (neoadjuvant) or after surgery (adjuvant).
The European Commission on Sept. 22 granted marketing authorization for Tecentriq as a monotherapy for treating people with locally advanced or metastatic NSCLC after they have been previously treated with chemotherapy regardless of PD-L1 status. Individuals with EGFR-activating mutations or ALK-positive tumor mutations should additionally have received targeted therapy before receiving Tecentriq. The approval is based on results from the large randomized Phase III Oak clinical trial and the randomized Phase II Poplar study.
Also on Sept. 22, the European Commission granted marketing authorization for Tecentriq as a monotherapy for treating people with locally advanced or metastatic urothelial carcinoma who have been previously treated with a platinum-containing chemotherapy or who are considered ineligible for cisplatin chemotherapy, regardless of PD-L1 status. This approval is based on results from the randomized Phase III IMvigor211 trial and cohorts 1 and 2 from the single-arm Phase II IMvigor210 study.
The Phase III IMvigor211 trial did not meet its primary endpoint of overall survival versus chemotherapy. The results observed in patients treated with Tecentriq in IMvigor211 were generally consistent with those observed in a similar group of people in the Phase II IMvigor210 study.
Tecentriq is designed to bind with a protein known as PD-L1. The monoclonal antibody is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable T-cell activation. Roche says the medicine has the potential to be used as a foundational combination partner with cancer immunotherapies, targeted medicines and various chemotherapies across a broad range of cancers.
Another EU approval occurred on Sept. 22 as the European Commission granted marketing clearance of Gazyvaro (obinutuzumab) in combination with chemotherapy, followed by Gazyvaro maintenance in people achieving a response, as a new treatment option for previously untreated advanced follicular lymphoma. This approval is based on results from the GALLIUM clinical trial, the first Phase III study in previously untreated follicular lymphoma to demonstrate superior progression-free survival (PFS) over MabThera (rituximab)-based treatment, the current standard of care.
The engineered monoclonal antibody Gazyvaro is designed to attach to CD20, a protein expressed on certain B cells, but not on stem cells or plasma cells. The drug is designed to attack and destroy targeted B cells both directly and together with the body’s immune system. Stem cells (B-cell progenitors) in bone marrow lack the CD20 antigen, enabling healthy B cells to regenerate after treatment and return to normal levels within a few months.
Gazyvaro is marketed as Gazyva outside the EU and Switzerland. Gazyva/Gazyvaro is available in more than 80 countries in combination with chlorambucil for people with previously untreated CLL and in combination with bendamustine for individuals with certain types of previously treated follicular lymphoma. The approvals in certain types of previously treated follicular lymphoma were based on the Phase III GADOLIN trial, in people with follicular lymphoma who did not respond to or who progressed during or within six months of prior MabThera-based therapy, demonstrating a significant improvement in PFS and overall survival (OS) with Gazyvaro-based therapy compared to bendamustine alone.
Additional combination trials investigating Gazyvaro with other approved or investigational medicines, including cancer immunotherapies and small molecule inhibitors, are under way across various blood cancers.
New data from additional analyses of the pivotal Phase III GALLIUM clinical trial in people with previously untreated follicular lymphoma confirmed that the improvement in PFS with Gazyva/Gazyvaro-based treatment over Rituxan/MabThera-based treatment was sustained, irrespective of chemotherapy regimen. According to Roche, this updated analysis – which was reported in June – includes a further six months of follow-up.
In yet another Sept. 22 EU marketing clearance for Roche, the European Commission approved Actemra/RoActemra as the first treatment for the potentially life-threatening autoimmune condition giant cell arteritis (GCA). On May 22, the FDA approved Actemra for treating GCA, a chronic condition that had not seen any new treatment options in more than 50 years. New Zealand’s Medsafe also granted regulatory clearance for this indication during May.
At the end of August, Actemra became the first FDA-approved treatment for severe or life-threatening cytokine release syndrome (CRS) induced by chimeric antigen receptor (CAR) T cell therapy. CAR T cell therapy is an immunotherapy designed for treating certain cancers. CRS is caused by an overactive immune response.
U.S. regulators granted Priority Review and Orphan Drug Designation to Actemra/RoActemra for the treatment of CAR T cell-induced CRS based on the rare, severe, and life-threatening nature of CRS and available data on the safety and efficacy of Actemra/RoActemra.
The CAR T cell-induced cytokine release syndrome marketing clearance represents the seventh FDA approval for Actemra/RoActemra since the product’s U.S. launch during 2010. Actemra/RoActemra represents the first approved anti-IL-6 receptor biologic available in both intravenous and subcutaneous formulations for treating adult patients with moderate to severe active rheumatoid arthritis.
Actemra/RoActemra is part of a joint-development agreement with Chugai Pharmaceutical and has been approved in Japan since April 2005. Available in 116 countries, Actemra/RoActemra is also being investigated in a global Phase III multicenter, randomized, double-blind, placebo-controlled study (NCT02453256) for patients with systemic sclerosis, also known as scleroderma. Actemra/RoActemra was granted Breakthrough Therapy Designation by the FDA during June 2015 for scleroderma.
The FDA during June approved Rituxan Hycela (rituximab and hyaluronidase human) for subcutaneous injection as a treatment for adults with specific types of blood cancer. The new formulation combines the monoclonal antibody that is used in intravenous Rituxan/MabThera with hyaluronidase, an enzyme that enables injection of the medicine underneath the skin. Treatment can be administered in five to seven minutes, compared to 1.5 hours or longer for intravenous Rituxan.
Rituxan Hycela is a co-formulation of the same monoclonal antibody as intravenous Rituxan/MabThera and Halozyme Therapeutics’ proprietary hyaluronidase human, an FDA-approved enzyme that facilitates the delivery of a large volume of medicine under the skin.
The therapeutic monoclonal antibody Rituxan initially received U.S. marketing clearance for treating relapsed indolent non-Hodgkin lymphoma (NHL) in 1997 and was the first targeted cancer medicine to be granted FDA approval. MabThera was approved in the EU during June 1998, and has since been used to treat more than 2.7 million individuals with specific blood cancers. For more than 15 years, the efficacy and safety of Rituxan/MabThera has been documented in more than 300 Phase II/III clinical trials. The product has been approved for treating several blood cancers, specifically, certain types of NHL and for chronic lymphocytic leukemia. Rituxan/MabThera continues to be studied in other types of blood cancers and disease fields where CD20-positive cells are believed to play a role.
The medicine is marketed as Rituxan in the United States, Japan, and Canada. Genentech and Biogen collaborate on Rituxan in the United States, and Roche markets MabThera in the rest of the world, except Japan, where the drug is jointly marketed by Chugai and Zenyaku Kogyo.
Additionally during June, a new tablet formulation of Esbriet (pirfenidone) – available as an 801-mg or 267-mg tablet – was approved by the European Commission for treating mild to moderate idiopathic pulmonary fibrosis (IPF) following U.S. approval in early 2017. The new 801-mg tablet makes therapy easier for patients, who now have the option of taking only one tablet three times per day versus three capsules three times daily. The new film-coated 267-mg tablet is smaller than the original 267-mg capsule and may be easier to swallow.
Esbriet was available in 40 countries as of September. The mechanism of action of the oral medicine is not fully understood, although it is believed to interfere with the production of transforming growth factor (TGF)-beta, a small protein in the body involved in how cells grow and produce scars (fibrosis), and tumor necrosis factor (TNF)-alpha, a small protein that is involved in inflammation. Esbriet has been granted Orphan Drug designation. The drug was approved for use in Europe during 2011 in adults with mild-to-moderate IPF and in the United States for IPF in October 2014.
Esbriet was initially approved on the basis of the largest clinical study program in IPF to date, including three Phase III studies (ASCEND and CAPACITY 004 and 006) with a total of 1,247 people with IPF.
Esbriet is conditionally recommended for use in people with IPF in the ATS/ERS/JRS/ALAT treatment guidelines that were published during July 2015. Pirfenidone has been available under the trade name Pirespa since 2008 in Japan and since 2012 in South Korea by Shionogi & Co. Ltd. Under different brand names, pirfenidone is approved for the treatment of IPF in China, India, Argentina and Mexico. Roche acquired InterMune and its lead asset Esbriet during September 2014 and continues to expand access to the medicine in additional countries.
Results of a six-month study combining Esbriet and nintedanib treatment were revealed during September showing a similar safety profile for the combination treatment to that expected for each treatment individually. According to Roche, the majority of the 89 patients included in the clinical study tolerated the combination treatment. The study additionally suggested that during the six-month period, lung function change from baseline was small, and quality of life scores did not deteriorate in patients who completed the 6 months of combination treatment.
The FDA approved Roche’s Lucentis (ranibizumab injection) in April for diabetic retinopathy, the leading cause of blindness among working-age adults in the United States. Lucentis became the first medicine approved to treat all forms of diabetic retinopathy. Priority Review Designation was granted by the FDA based on analysis of results from a National Institutes of Health-funded collaborative group study.
The vascular endothelial growth factor (VEGF) inhibitor Lucentis is designed to bind to and inhibit VEGF-A, a protein thought to play a critical role in the formation of new blood vessels (angiogenesis) and the hyperpermeability (leakiness) of the vessels. Lucentis is FDA-approved for treating patients with wet age-related macular degeneration (AMD), macular edema after retinal vein occlusion (RVO), diabetic macular edema (DME), diabetic retinopathy, and myopic choroidal neovascularization (mCNV).
Lucentis was developed by Roche, which retains U.S. commercial rights. Novartis holds exclusive commercial rights for the rest of the world.
Outside the United States, Lucentis is approved in more than 110 countries to treat patients with wet AMD, for treating DME, and due to macular edema secondary to both branch retinal vein occlusion (BRVO), central retinal vein occlusion (CRVO) and visual impairment due to choroidal neovascularization (CNV).
The Australian Therapeutic Goods Administration in July approved Ocrevus as a treatment for relapsing forms of multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS). Australia became the second country to approve Ocrevus following FDA’s marketing clearance in March 2017. As of mid-July, marketing applications were under review in more than 50 countries globally, including in Europe, Latin America, and the Middle East.
The humanized monoclonal antibody Ocrevus was developed to target CD20-positive B cells, a specific type of immune cell believed to be a key contributor to myelin (nerve cell insulation and support) and axonal (nerve cell) damage. Such nerve cell damage can lead to disability in people with multiple sclerosis. Ocrevus is administered as an intravenous infusion every six months.
During August, Roche announced that the FDA accepted the company’s Biologics License Application (BLA) and granted Priority Review for emicizumab prophylaxis (preventative) as a once-weekly subcutaneous treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. A U.S. regulatory approval decision is expected by Feb. 23, 2018. Data from HAVEN 1 and HAVEN 2 have additionally been filed for approval consideration to the European Medicines Agency and are being reviewed under accelerated assessment. Further studies evaluating emicizumab in people with hemophilia A both with and without inhibitors and exploring less frequent dosing regimens are under way.
An investigational bispecific monoclonal antibody, emicizumab is designed to bring together factors IXa and X, proteins required to activate the natural coagulation cascade and restore the blood-clotting process. Emicizumab is administered by an injection of a ready-to-use solution under the skin (subcutaneously) once per week. Emicizumab is being studied in pivotal Phase III trials in people 12 years of age and older, both with and without inhibitors to factor VIII, and in children younger than 12 years of age with factor VIII inhibitors. Other studies are exploring less frequent dosing schedules.
Roche announced in September that the Phase III MURANO trial, which assessed Venclexta/Venclyxto in combination with Rituxan/MabThera in people with relapsed or refractory CLL, met its primary endpoint and demonstrated a statistically significant improvement in the time people lived without their disease progressing when treated with Venclexta/Venclyxto plus Rituxan/MabThera versus bendamustine plus Rituxan/MabThera. No new safety signals or increase in known toxicities of Venclexta/Venclyxto were observed with the treatment combination of Venclexta/Venclyxto with Rituxan/MabThera.
Roche revealed new data from multiple studies of Venclexta/Venclyxto at the 22nd European Hematology Association Annual Congress in June. The presentation confirmed Venclexta/Venclyxto monotherapy benefit in certain patients with high-risk chronic lymphocytic leukemia and its potential in other difficult-to-treat blood cancers. The first-in-class BCL2-specific oral inhibitor represents a potential new way of treating various blood cancers.
The small molecule Venclexta/Venclyxto is designed to selectively bind and inhibit the BCL-2 protein, which plays a significant role in a process called apoptosis (programmed cell death). Overexpression of the BCL-2 protein in chronic lymphocytic leukemia has been associated with resistance to certain therapies. It is believed that blocking BCL-2 may restore the signaling system that informs cells, including cancer cells, to self-destruct.
Venclexta/Venclyxto is being jointly developed by Roche and AbbVie. The product is being tested in Phase III studies for the treatment of relapsed, refractory, and previously untreated CLL, along with clinical trials in several other cancers. Venclexta/Venclyxto is jointly commercialized by AbbVie and Genentech in the United States and is commercialized by AbbVie outside of the United States.
Roche during September presented data from a worldwide Phase III study demonstrating significant clinical benefit of Alecensa (alectinib) in later-line advanced ALK-positive lung cancer. The ALUR trial shows 85 percent reduction in risk of disease worsening or death versus chemotherapy in patients with advanced ALK-positive NSCLC who had progressed on chemotherapy and crizotinib.
The Phase III Alex study demonstrated that Alecensa reduced the risk of disease worsening or death by more than half (53 percent) compared to crizotinib when given as initial (first-line) treatment for people with anaplastic lymphoma kinase (ALK)-positive advanced NSCLC.
Alecensa is approved as a monotherapy for individuals with ALK-positive NSCLC who have progressed on or are intolerant to crizotinib in the United States, Europe, Kuwait, Israel, Hong Kong, Canada, South Korea, Switzerland, India, Australia, Singapore, Thailand, and Taiwan. The drug is also approved in Japan for patients whose tumors were advanced, recurrent, or could not be removed completely through surgery (unresectable). Alecensa was granted FDA accelerated approval during December 2015 for treating patients with ALK-positive NSCLC who have progressed on or are intolerant to crizotinib.
Roche reported during June that the primary analysis of the Phase III Haven 1 study in adults and adolescents and interim analysis of the Phase III Haven 2 study showed positive data. The studies assessed once-weekly subcutaneous emicizumab prophylaxis for treating adults and adolescents or children with hemophilia A and inhibitors to factor VIII. The primary endpoint was treated bleeds, and results demonstrated a statistically significant and clinically meaningful reduction in bleed rate of 87 percent (Haven 1) with emicizumab prophylaxis versus on-demand treatment with bypassing agents.
Based on data presented at EAN during June, Ocrevus significantly reduced disease activity and disability progression in patients with RMS and PPMS, as measured by No Evidence of Progression or Active Disease (NEPAD), a novel composite endpoint in MS. The company says Ocrevus significantly reduced the risk of RMS and PPMS patients requiring mobility aids versus comparators. In patients with PPMS, Ocrevus reduced the risk of more severe forms of disability progression compared to placebo.
The Phase III Aphinity trial showed adjuvant treatment with the combination of Perjeta, Herceptin, and chemotherapy (the Perjeta-based regimen) significantly reduced the risk of breast cancer recurrence or death in people with HER2-positive early breast cancer versus Herceptin and chemotherapy alone, as reported by Roche in June. At the time of the primary analysis, with median follow-up of 45.4 months, the reduction in risk of invasive breast cancer recurrence with the Perjeta-based regimen was highest in people with lymph node-positive or hormone receptor-negative disease.
Perjeta targets the HER2 receptor, a protein found on the outside of many normal cells and in high quantities on the outside of cancer cells in HER2-positive cancers. The medicine is designed specifically to prevent the HER2 receptor from pairing (or ‘dimerizing’) with other HER receptors (EGFR/HER1, HER3 and HER4) on the surface of cells, a process believed to play a role in tumor growth and survival. Binding of Perjeta to HER2 may additionally signal the body’s immune system to destroy the cancer cells. The mechanisms of action of Perjeta and Herceptin are thought to complement each other, as both bind to the HER2 receptor, but to different locations. The combination of Perjeta and Herceptin is believed to provide a more comprehensive, dual blockade of HER signaling pathways, thus preventing tumor cell growth and survival.
On the diagnostics front, the Elecsys HIV combi PT assay received FDA clearance during second-quarter 2017. This highly sensitive and specific assay further expands Roche’s broad testing menu for infectious diseases in the United States. With the addition of this assay, labs are able to screen for common co-infections, including hepatitis C and syphilis, which can be tested simultaneously with HIV, reducing the need for sample splitting and additional analyzers. The U.S. regulatory agency additionally granted approval for the cobas CMV (cytomegalovirus) test for use on the fully automated cobas 6800 and cobas 8800 systems. Healthcare professionals use the CMV test to evaluate how transplant patients on therapy are responding to treatment.
The cobas MRSA/SA nucleic acid test for use on the cobas Liat System for the qualitative detection and differentiation of methicillin-resistant Staphylococcus aureus (MRSA) and S. aureus (SA) was launched during July in countries accepting the CE mark. This nucleic acid test is regarded as ideal for use at the point of care. This menu expansion serves specific needs of European markets, according to Roche. This is the first real-time PCR test that simultaneously detects MRSA and SA with one sample in under 30 minutes. MRSA and SA are considered significant sources of healthcare-associated and community-associated infections.
Introduced globally in May were the Avenio ctDNA (ctDNA – circulating tumor DNA) analysis kits for oncology research. These are the first blood-based distributed oncology tests and provide accurate insights into different stages and forms of cancer.
According to Roche, they represent a portfolio of three next-generation sequencing (NGS) liquid biopsy assay kits for oncology research: the Avenio ctDNA Targeted Kit, Expanded Kit and Surveillance Kit.
The kits include all reagents, bioinformatics and software to make ctDNA-testing accessible to every NGS laboratory.
Roche introduced during the second quarter the Avenio Millisect System, which uses an automated, digitally assisted process to reliably and efficiently isolate clinically relevant cells from tissue biopsies for diagnostic testing in the United States and countries accepting the CE mark. The system allows for efficient tissue dissection and maximizes medical value for all molecular downstream applications.
Roche announced in early April the launch of the cobas Liat PCR System with four assays into CE markets. This is the first 20-minute real-time PCR nucleic acid test to detect Clostridium difficile, which supports time-sensitive diagnoses in hospitals, urgent-care settings, and physician offices. Infections with C. difficile can quickly become life threatening to high-risk patients. The cobas Liat PCR System additionally includes tests for strep A, influenza A/B, and influenza A/B and RSV.
Roche announced in March the introduction of the cobas HPV on the cobas 6800/8800 Systems for cervical cancer screening in markets accepting the CE mark. The cobas HPV assay helps provide screening in identifying women at risk, before pre-cancer or cancer develops. The cobas 6800/8800 Systems deliver full automation, helping labs meet the throughput that high volume, HPV DNA screening programs demand.
The company entered a new era in hematology testing with the January launch of the new cobas m 511 analyzer. A dedicated hematology testing solution, the cobas m 511 integrated hematology analyzer was made available for countries accepting the CE Mark. The launch marked the entry for Roche Diagnostics in the hematology field with a Roche developed system.