Monday, January 21, 2019
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Roche announces FDA grants Venclexta accelerated approval for people with newly-diagnosed acute myeloid leukaemia or those who are ineligible for intensive induction chemotherapy

  • Approval based on two studies that showed durable remissions in people with newly-diagnosed acute myeloid leukaemia, who are age 75 years or older, or for those ineligible for intensive induction chemotherapy
  • Venclexta represents a new treatment option for people with acute myeloid leukaemia regardless of subtypes 

Basel, 21 November 2018 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the US Food and Drug Administration (FDA) has granted accelerated approval to Venclexta® (venetoclax), in combination with a hypomethylating agent (azacitidine or decitabine), or low-dose cytarabine (LDAC), for the treatment of people with newly-diagnosed acute myeloid leukaemia (AML), who are age 75 years or older, or for those ineligible for intensive induction chemotherapy due to coexisting medical conditions. AML is the most common type of aggressive leukaemia in adults and has the lowest survival rate for all types of leukaemia.
“Today’s approval marks a significant advance for people with acute myeloid leukaemia, a highly aggressive and difficult-to-treat blood cancer,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “Many people with acute myeloid leukaemia are unable to tolerate standard intensive chemotherapy, and the Venclexta combination regimens represent important new options for these patients.”

This accelerated approval was based on results from the M14-358 study and the M14-387 study in people newly-diagnosed with AML including those who were ineligible for intensive induction chemotherapy. In M14-358, the rate of complete remission (CR) was 37% (n=25/67) and the rate of complete remission with partial blood count recovery (CRh) was 24% (n=16/67) for those who received Venclexta plus azacitidine. For those who received Venclexta plus decitabine, the rate of CR was 54% (n=7/13) and the rate of CRh was 8% (n=1/13). M14-387 showed a CR rate of 21% (n=13/61) and a CRh rate of 21% (n=13/61) for those who received Venclexta in combination with LDAC.

The most common serious side effects of these regimens (occurring in at least 5% of patients) were low white blood cell count with fever, pneumonia, bacteria in the blood, inflammation of tissue under the skin,  device-related infection, diarrhoea, fatigue, bleeding, localized infection,  multiple organ dysfunction syndrome, and respiratory failure.
The FDA’s Accelerated Approval Program allows conditional approval of a medicine that fills an unmet medical need for a serious condition. This approval of Venclexta is based on surrogate endpoints that are reasonably likely to predict clinical benefit, including CR and CRh. Continued approval for this indication may be contingent upon verification and description of clinical benefit observed in confirmatory trials.
The supplemental New Drug Application (sNDA) was granted Priority Review, a designation given to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a disease. In addition, the FDA previously granted two Breakthrough Therapy Designations for Venclexta in people with previously untreated AML ineligible for intensive chemotherapy, either in combination with a hypomethylating agent or LDAC, based on results from these two studies. With this approval, Venclexta is available in the US for two forms of blood cancer.

Venclexta is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the US and commercialised by AbbVie outside of the US.

About the M14-358 study

The M14-358 study (NCT02203773) is an open-label, non-randomised, Phase Ib dose escalation and expansion study evaluating the safety and efficacy of Venclexta in combination with hypomethylating agents, azacitidine or decitabine, in newly-diagnosed people with AML who were 60 years or older, or ineligible to receive intensive induction chemotherapy due to coexisting medical conditions. Study endpoints included complete remission rates, overall survival and safety.

  • In M14-358, the rate of CR was 37% and the rate of CRh was 24% for those who received Venclexta plus azacitidine. The median follow-up for this group was 7.9 months (0.4-36 months). At the time of analysis, for patients who achieved a CR, the median observed time in remission was 5.5 months (0.4-30 months).
  • For those who received Venclexta plus decitabine, the rate of CR was 54% and the rate of CRh was 8%. The median follow-up for this group was 11 months (0.7-21 months). At the time of analysis, for patients who achieved a CR, the median observed time in remission was 4.7 months (1.0-18 months).
  • The observed time in remission for these regimens was defined as the time from the start of CR to the time of the data cut-off date or relapse from CR.
  • The most common adverse reactions with Venclexta plus decitabine were low white blood cell count with or without fever, constipation, fatigue, low platelet count, stomach (abdominal) pain, dizziness, bleeding, nausea, pneumonia, infection in the blood, cough, diarrhoea, low blood pressure, pain in muscles or back, sore throat, swelling in the arms, legs hand and feet, fever and rash.

About the M14-387 study

The M14-387 study (NCT02287233) is an open-label, single-arm, Phase I/II dose escalation and expansion study evaluating the safety and efficacy of Venclexta in combination with LDAC in newly-diagnosed people with AML who were 60 years or older, or ineligible to receive intensive induction chemotherapy due to coexisting medical conditions. Study endpoints included complete remission rates, overall survival and safety.

  • The study showed the rate of CR and CRh was 21% for those who received Venclexta plus LDAC. The median follow-up for this group was 6.5 months (0.3-34 months). At the time of analysis, for patients who achieved a CR, the median observed time in remission was 6.05 months (0.3-25 months). The observed time in remission for this regimen was defined as the time from the start of CR to the time of the data cut-off date or relapse from CR.
  • The most common adverse reactions with Venclexta in combination with LDAC were nausea, low platelet count, bleeding, low white blood cell count with or without fever, diarrhoea, fatigue, constipation and difficulty breathing.

About Venclexta (venetoclax)

Venclexta is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers and other tumours, BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venclexta blocks the BCL-2 protein and works to restore the process of apoptosis.

umours, BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venclexta blocks the BCL-2 protein and works to restore the process of apoptosis.

Venclexta is being developed by AbbVie and Roche. It is jointly commercialised by AbbVie and Genentech, a member of the Roche Group, in the US and by AbbVie outside of the US. Together, the companies are committed to research with Venclexta, which is currently being studied in clinical trials across several types of blood and other cancers.

In the US, Venclexta has been granted four Breakthrough Therapy Designations by the FDA: in combination with Rituxan for people with relapsed or refractory chronic lymphocytic leukaemia (CLL); as a monotherapy for people with relapsed or refractory CLL with 17p deletion; in combination with hypomethylating agents (azacitidine or decitabine) for people with untreated acute myeloid leukaemia (AML) ineligible for intensive chemotherapy; and in combination with low-dose cytarabine for people with untreated AML ineligible for intensive chemotherapy.

Venclexta is approved in more than 50 countries. Roche and AbbVie are currently working with regulatory agencies around the world to bring this medicine to additional eligible patients in need.

About Acute Myeloid Leukaemia
Acute myeloid leukaemia (AML) is an aggressive form of leukaemia that starts in immature forms of blood-forming cells, known as myeloid cells, found in the bone marrow.[1] AML is the most common type of aggressive leukaemia in adults. It has the lowest survival rates of all types of leukaemia. [2] Even with the best available therapies, older patients aged 65 and over have survival rates comparable to patients with advanced lung cancer, with a five year overall survival rate of <5%.[3;4] Approximately 20,000 people in the US and 18,000 in Europe are diagnosed with AML each year. [5;6]

About Roche
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 personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.
Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the tenth consecutive year, Roche has been recognised as the most sustainable company in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 billion. 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.

References
[1] American Cancer Society: What is acute myeloid leukemia? [Internet; cited 2018]. Available from: https://www.cancer.org/cancer/acute-myeloid-leukemia/about/what-is-aml.html
[2] Leukemia & Lymphoma Society: Facts and statistics overview – Leukemia. [Internet; cited 2018]. Available from: http://www.lls.org/http%3A/llsorg.prod.acquia-sites.com/facts-and-statistics/facts-and-statistics-overview/facts-and-statistics#Leukemia.
[3] Sekeres MA. Treatment Of Older Adults With Acute Myeloid Leukemia: State Of The Art And Current Perspectives. Haematologica 2008;93:1769-1772
[4] Cancer Research UK: Survival statistics for acute myeloid leukaemia (AML). [Internet; cited 2018]. Available from: http://www.cancerresearchuk.org/about-cancer/acute-myeloid-leukaemia-aml/survival?_ga=2.239561667.1384102361.1500450925-993068745.1500450925
[5] National Cancer Institute. Adult Acute Myeloid Leukemia Treatment (PDQ®)-Health Professional Version [Internet; cited 2018 May]. Available from: https://www.cancer.gov/types/leukemia/hp/adult-aml-treatment-pdq.
[6] Visser O, et al. (RARECARE Working Group). Incidence, survival and prevalence of myeloid malignancies in Europe. Eur J Cancer. 2012;48:3257-3266.

Roche Group Media Relations
Phone: +41 61 688 8888 / e-mail: media.relations@roche.com
– Nicolas Dunant (Head)
– Patrick Barth
– Ulrike Engels-Lange
– Simone Oeschger
– Anja von Treskow

Efficient Flow Chemistry Scale-up of Photoredox Coupling Reactions

The renowned Innovative Technology Centre (ITC) at the University of Cambridge, UK has used a Uniqsis PhotoSyn flow photoreactor to efficiently scale-up a radical mediated boronic acid coupling under photoredox conditions using a novel organic acridium-based photocatalyst.

The acridium-based organic photocatalyst, developed by the University of Cambridge, is demonstrated to be an efficient replacement for iridium-based photocatalysts used to oxidise boronic acid derivatives by a single electron radical coupling process.

By eliminating the control, scale-up and safety problems associated with traditional batch photoreactors – the Uniqsis PhotoSyn LED photochemical flow reactor enabled synthetic chemists at the University of Cambridge to straightforwardly scale a photoredox cross-coupling of boronic acids in 81% yield with gram per hour throughput. In the described work, the researchers also used a Uniqsis Flow-UV inline UV-Vis spectrometer to conveniently monitor steady state.

Using a pair of enclosed large format LED arrays to concentrate the light inwards onto a central coil reactor, the Photosyn is able to provide sufficient light to facilitate reactions in coil reactors (up to 50 ml) enabling efficient photochemical synthetic work at larger scales.

For a copy of the University of Cambridge paper please visit http://www.uniqsis.com/fcPublications.aspx#2109 (select publication 62). For further information on Photosyn flow photoreactor please contact Uniqsis on +44-845-864-7747 / info@uniqsis.com.

Since 2007, Uniqsis has specialised in the design and supply of mesoscale continuous flow chemistry systems for a wide range of applications in chemical and pharmaceutical research. The company’s aim is to make flow chemistry easily accessible to both novices and experienced users.

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Further Information:

Uniqsis Ltd
29 Station Road
Shepreth
Cambridgeshire CB7 5RJ
UK

tel: +44-845-864-7747
email:  info@uniqsis.com
web: www.uniqsis.com

—–

Media

Dr Bill Bradbury    Primetek Solutions
+44-208-546-0869  /  info@primetek-solutions.com

 

 

 

 

 

 

ONCOdianova: The future of tumor tissue biomarkers

New antibodies for the detection of immune-checkpoint-proteins in tumor tissues support the development of novel therapeutic strategies.

Hamburg, August 2018. New immunotherapies give rise to hope for a breakthrough in the treatment of cancer. Cancer cells use immune-checkpoint-proteins to protect themselves against the attack of the immune system. Over the last few years, several immunotherapy-medication, which inhibit checkpoint-proteins, have been approved. An inhibition of those checkpoints leads to a strengthened body’s own defense against the tumor.

The ONCOdianova GmbH (www.oncodianova.com), a newly founded Biotechnology Company from Hamburg, focuses on the specific analysis of immune-checkpoint-proteins in tumor tissues using antibodies. ONCOdianova (www.oncodianova.com) has the vision to provide the best primary antibodies for the detection of immune-checkpoint-marker in the tumor tissue. The aim: To support the development of new cancer medication at the best.

According to the cancer information service experts expect around 493.600 new cases of cancer in Germany alone in 2018[1]. For the development of suitable therapies a differentiating analysis of the endogenous processes is decisive. ONCOdianova knows this and has set up a future-oriented development concept for antibodies in the field of immuno-oncology.

 

 

Antibodies against Checkpoint-Marker enable differentiated analyses of formalin-fixed tumor tissues

 

The use of antibodies in tissue-based studies, apart from the detection of the marker, has another great advantage: It is possible to examine which cells are expressing the marker within the tumor or tumor microenvironment in a morphological context. Clone TG1 is the first monoclonal antibody for the immunohistochemically detection of the important checkpoint-protein TIGIT, in routine formalin-fixed, paraffin-embedded (FFPE) tumor tissue. TG1 enables to identify TIGIT-proteins in the tumor “microenvironment“ and detects invading immune cells, so-called tumor-infiltrating T-cells. Examination of human tissue samples using TG1 can provide a valuable contribution to the clinical research community and tissue diagnostic, since potential medical-approaches can be better tailored to the TIGIT-mediated Tumor-immune-checkpoint.

 

 

The idea of ONCOdianova

 

“The idea behind our company ONCOdianova is to allow the best possible detection of Checkpoint biomarker in the tumor tissue. Our goal is to offer the best antibodies for the combined use in immunohistochemical panels”, explains ONCOdianova CEO Jürgen Frerichs. The first product, anti-TIGIT clone TG1, was presented at the annual meeting of the American Association for Cancer Research (AACR) in the USA in April 2018 as well as in recent studies about TG1 by teams of the University Hospital Hamburg-Eppendorf und the University of Colorado/Denver.

 

 

The future for ONCOdianova

 

“We believe that new antibodies for the immunohistochemical evaluation of tumors are essential for the development of optimal diagnostic algorithms”, says Jürgen Frerichs. The Biotech-Company focus strongly on the highly complex “TIGIT-axis” which is used for interaction between various immune-checkpoint-proteins and so allows the communication between tumor and immune system. Next to the development of further TIGIT-antibody-clones, ONCOdianova also concentrates on other marker for the TIGIT-axis such as CD155, CD226, CD112 and CD112R. “We see a great diagnostic potential and believe that immunohistochemical studies of immune-checkpoint markers in tumor tissues continue to gain importance. Future medication will be adapted to patients individual needs based on these studies and could provide optimal medical treatment.”, explains Frerichs.

 

The homepage ONCOdianova.com shows the latest news of the development, the project progress and further background information.

[1] https://www.krebsinformationsdienst.de/grundlagen/krebsstatistiken.php

Roche announces global availability of blood-based genomic profiling test, FoundationOne Liquid

  • Liquid biopsy test can identify 70 of the most commonly mutated genes in solid tumours, as well as microsatellite instability
  • Many cancer patients have insufficient or inadequate tissue for genomic testing and so may benefit from FoundationOne Liquid
  • FoundationOne Liquid joins FoundationOne CDx to deliver a complementary portfolio which provides insights to personalise a patient’s treatment plan

Basel, 24 September 2018 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today the global availability of FoundationOne®Liquid, a liquid biopsy test. FoundationOne Liquid can identify circulating tumour DNA in the blood of people living with cancer and can identify 70 of the most commonly mutated genes in solid tumours, including microsatellite instability, a genomic signature which may help inform cancer immunotherapy based treatment decisions. [1,2] From a single blood sample, the liquid biopsy offers a quick and convenient option for some patients with solid tumours. [3]

FoundationOne Liquid meets a compelling need for comprehensive genomic profiling for people who have insufficient or inadequate tissue, including those with advanced non-small cell lung cancer, where an estimated 15% of patients are not eligible for tissue biopsy and approximately 10% have a biopsy size that is insufficient to evaluate. [4-6] FoundationOne Liquid complements FoundationOne CDx, a tissue-based genomic profiling test launched in the US earlier this year, to deliver a portfolio of comprehensive genomic profiling services for healthcare professionals.
“Cancer is a disease of the genome and genomic profiling of every patient’s tumour at the start of their treatment journey will provide transformative outcomes for patients,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “At Roche, we believe the use of innovative profiling technologies like FoundationOne Liquid and FoundationOne CDx will improve access to targeted cancer treatments for patients and enrich the future development of targeted medicines.”
About FoundationOne Liquid

FoundationOne Liquid is a liquid biopsy circulating tumour DNA (ctDNA) test that complements

FoundationOne CDx, a tissue-based test. The blood sample is sent to a Foundation Medicine lab where   the test is performed using next generation sequencing to analyse the four main classes of genomic alterations as well as microsatellite instability, an indicator that may help inform immunotherapy treatment decisions using ctDNA isolated from plasma derived from peripheral whole blood.
About FoundationOne CDx

FoundationOne CDx is based on the first US Food and Drug Administration-approved broad companion diagnostic assay to assess the four main classes of genomic alterations and identify patients with advanced cancer who are likely to respond to targeted therapies based on their individual genomic profile. The tissue sample is sent to a Foundation Medicine lab where the test is performed using next generation sequencing to analyse the four main classes of genomic alterations, as well as microsatellite instability and tumour mutational burden, using DNA isolated from formalin-fixed paraffin embedded (FFPE) tumour tissue specimens. The test has been validated with 2,100 clinical samples and 4,200 analytical samples. [7] FoundationOne CDx is intended to be used by clinicians as decision-making support in consideration of a patient’s genomic profile for therapy selection and patient management according to professional guidelines in oncology for cancer patients.
 

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient’s unique cancer. The company, a member of the Roche Group, offers a full suite of comprehensive genomic profiling tests to identify the molecular alterations in a patient’s cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicine’s molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer.

For more information, please visit http://www.foundationmedicine.com or follow Foundation Medicine on Twitter (@FoundationMedicineATCG).
About Roche

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 personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the tenth consecutive year, Roche has been recognised as the most sustainable company in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).
The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 billion. 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.

References

[1] Foundation Medicine. FoundationOne Liquid Technical Specifications [Internet: accessed September 2018]. Available from: https://www.foundationmedicine.com/genomic-testing/foundation-one-liquid.

[2] Dudley JC, et al. Microsatellite Instability as a Biomarker for PD-1 Blockade. Clin Cancer Res. 2016; 22:813-20.

[3] Clark TA, et al. Analytical Validation of a Hybrid Capture-Based Next-Generation Sequencing Clinical Assay for Genomic Profiling of Cell-Free Circulating Tumor DNA. J Mol Diagn. 2018; S1525-1578(17)30527-5 [Epub ahead of print].

[4] Rafael OC, et al. Molecular Testing in Lung Cancer: Fine-Needle Aspiration Specimen Adequacy and Test Prioritization Prior to the CAP=IASLC=AMP Molecular Testing Guideline Publication. Cancer Cytopathology. 2014; 122(6):454-458.

[5] Douillard JY, et al. Gefitinib Treatment in EGFR Mutated Caucasian NSCLC. J Thorac Oncol. 2014; 9:1345-1353.

[6] Kim ES, et al. Gefitinib versus docetaxel in previously treated non-small-cell lung cancer (INTEREST): a randomised phase III trial. Lancet. 2008; 372:1809-18.

[7] Foundation Medicine website: FoundationOne CDx – “Go Beyond FoundationOne®” [Internet: accessed September 2018]. Available from: https://www.foundationmedicine.com/genomic-testing/foundation-one-cdx.

Roche Group Media Relations

Phone: +41 61 688 8888 / e-mail: media.relations@roche-global.com
– Nicolas Dunant (Head)

– Patrick Barth

– Ulrike Engels-Lange

– Simone Oeschger

– Anja von Treskow

Positive phase III results for Roche’s Hemlibra for haemophilia A without factor VIII inhibitors published in New England Journal of Medicine

  • Hemlibra prophylaxis significantly reduced bleeds compared to no prophylaxis
  • Hemlibra is the first medicine to demonstrate superior efficacy to prior factor VIII prophylaxis based on a statistically significant reduction in treated bleeds in an intra-patient comparison
  • Hemlibra is currently under Priority Review by the FDA for people with haemophilia A without factor VIII inhibitors

Basel, 30 August 2018 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that pivotal data from the phase III HAVEN 3 study, which evaluated Hemlibra® (emicizumab) prophylaxis administered every week or every two weeks in adults and adolescents aged 12 years or older with haemophilia A without factor VIII inhibitors, were published in the 30 August 2018 issue of the New England Journal of Medicine (NEJM).

“In the HAVEN 3 study, Hemlibra showed a significant and clinically meaningful reduction in bleeds in people with haemophilia A without factor VIII inhibitors, while offering multiple subcutaneous dosing options,” said Johnny Mahlangu, Faculty of Health Sciences, University of the Witwatersrand and NHLS, Johannesburg, South Africa. “The publication of these results in the New England Journal of Medicine represents a major advance for haemophilia research and reinforces the potential of Hemlibra to change the standard of care for people with haemophilia A.”

“Current prophylactic treatment options for people with haemophilia A can require frequent intravenous infusions, and despite treatment, many continue to have bleeds that can lead to long-term joint damage,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “Given the challenges many people face managing their haemophilia, we believe Hemlibra could make a meaningful difference, and we are working with health authorities to hopefully make this treatment available to people with haemophilia A without factor VIII inhibitors as soon as possible.”

Data from the HAVEN 3 study showed that Hemlibra prophylaxis administered subcutaneously every week or every two weeks significantly reduced treated bleeds by 96% (rate ratio [RR]=0.04; p<0.0001) and 97% (RR= 0.03; p<0.0001), respectively, compared to no prophylaxis. Results also showed that 55.6% (95% CI: 38.1; 72.1) of people treated with Hemlibra every week and 60% (95% CI: 42.1; 76.1) of people treated with Hemlibra every two weeks experienced zero treated bleeds, compared to 0% (95% CI: 0.0; 18.5) of people treated with no prophylaxis. In an intra-patient comparison of people who previously received factor VIII prophylaxis in a prospective non-interventional study and switched to Hemlibra prophylaxis, Hemlibra demonstrated a statistically significant reduction of 68% (RR=0.32; p<0.0001) in treated bleeds, making it the first medicine to show superior efficacy to prior factor VIII prophylaxis treatment, the current standard of care. There were no unexpected or serious adverse events (AEs) related to Hemlibra in the HAVEN 3 study, and the most common AEs were consistent with previous studies. The most common AEs occurring in 5% or more of people were injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza.
Earlier this year, the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation and Priority Review to Hemlibra for people with haemophilia A without factor VIII inhibitors based on data from the HAVEN 3 study. The FDA is expected to make a decision on approval by 4 October 2018. Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate substantial improvement over existing therapies. Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease. In addition, the company’s Marketing Authorisation Application (MAA) variation of Hemlibra for haemophilia A without factor VIII inhibitors is under review by the European Medicines Agency (EMA). Submissions to other regulatory authorities around the world are ongoing.
 

About HAVEN 3 (NCT02847637)

HAVEN 3 is a randomised, multicentre, open-label, phase III study evaluating the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis versus no prophylaxis (episodic/on-demand factor VIII treatment) in people with haemophilia A without factor VIII inhibitors. The study included 152 patients with haemophilia A (12 years of age or older) who were previously treated with factor VIII therapy either on-demand or for prophylaxis. Patients previously treated with on-demand factor VIII were randomised in a 2:2:1 fashion to receive subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk for at least 24 weeks (Arm A), subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 3 mg/kg/2wks (Arm B) for at least 24 weeks or no prophylaxis (Arm C) for at least 24 weeks. Patients previously treated with factor VIII prophylaxis received subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study (Arm D). Episodic treatment of breakthrough bleeds with factor VIII therapy was allowed per protocol.

The phase III HAVEN 3 study in people with haemophilia A without factor VIII inhibitors met its primary         endpoint and key secondary endpoints. Data from the study showed:

  • Hemlibra prophylaxis every week or every two weeks resulted in a 96% (RR=0.04; p<0.0001) and 97% (RR=0.03; p<0.0001) reduction in treated bleeds, respectively, compared to no prophylaxis.
  • 55.6% (95% CI: 38.1, 72.1) of people treated with Hemlibra every week and 60% (95% CI: 42.1, 76.1) of people treated with Hemlibra every two weeks experienced zero treated bleeds, compared to 0% (95% CI: 0.0; 18.5) of people treated with no prophylaxis.
  • 91.7% (95% CI: 77.5, 98.2) of people treated with Hemlibra prophylaxis every week and 94.3% (95% CI: 80.8, 99.3) of people treated with Hemlibra prophylaxis every two weeks experienced three or fewer treated bleeds, compared to 5.6% (95% CI: 0.1, 27.3) of people treated with no prophylaxis.
  • Hemlibra prophylaxis every week or every two weeks resulted in a 95% (RR=0.05; p<0.0001) and 95% (RR=0.05; p<0.0001) reduction in treated target-joint bleeds, respectively, compared to no prophylaxis.
  • Hemlibra prophylaxis every week or every two weeks resulted in a 95% (RR=0.05; p<0.0001) and 94% (RR=0.06; p<0.0001) reduction in all bleeds, respectively, compared to no prophylaxis.
  • Hemlibra prophylaxis every week demonstrated a statistically significant reduction of 68% (RR=0.32; p<0.0001) in treated bleeds compared to prior factor VIII prophylaxis based on an intra-patient comparison of people who were previously enrolled in a prospective non-interventional study.
  • There were no unexpected or serious AEs related to Hemlibra, and the most common AEs were consistent with previous studies. No thrombotic events or cases of thrombotic microangiopathy were observed. The most common AEs occurring in 5% or more of people were injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza.
  • There were no unexpected or serious AEs related to Hemlibra, and the most common AEs were consistent with previous studies. No thrombotic events or cases of thrombotic microangiopathy were observed. The most common AEs occurring in 5% or more of people were injection site reactions, joint pain (arthralgia), common cold symptoms (nasopharyngitis), headache, upper respiratory tract infection and influenza.

About Hemlibra® (emicizumab)

Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for people with haemophilia A. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly. The clinical development programme is assessing the safety and efficacy of Hemlibra and its potential to help overcome current clinical challenges: the short-lasting effects of existing treatments, the development of factor VIII inhibitors and the need for frequent venous access. Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech. It is marketed in the United States by Genentech as Hemlibra (emicizumab-kxwh) for people with haemophilia A with factor VIII inhibitors, with kxwh as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the US Food and Drug Administration.

About haemophilia A

Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia A affects around 320,000 people worldwide, [1,2] approximately 50-60% of whom have a severe form of the disorder. [3] People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their disorder, people with haemophilia A can bleed frequently, especially into their joints or muscles.[1] These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility, and long-term joint damage. [4] A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. [5] Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII,[6] making it difficult, if not impossible to obtain a level of factor VIII sufficient to control bleeding.
About Roche in haematology

For more than 20 years, Roche has been developing medicines that redefine treatment in haematology. Today, we are investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. In addition to approved medicines MabThera®/Rituxan® (rituximab), Gazyva®/Gazyvaro® (obinutuzumab), and Venclexta®/Venclyxto(TM) (venetoclax) in collaboration with AbbVie, Roche’s pipeline of investigational haematology medicines includes Tecentriq® (atezolizumab), an anti-CD79b antibody drug conjugate (polatuzumab vedotin/RG7596) and a small molecule antagonist of MDM2 (idasanutlin/RG7388). Roche’s dedication to developing novel molecules in haematology expands beyond malignancy, with the development of Hemlibra® (emicizumab), a bispecific monoclonal antibody for the treatment of haemophilia A.

About Roche

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 personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management. Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry nine years in a row by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 billion. 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.

References

[1] WFH. Guidelines for the management of haemophilia. 2012 [Internet; cited 2018 July]. Available from:

http://www1.wfh.org/publications/files/pdf-1472.pdf

[2] Berntorp E, Shapiro AD. Modern haemophilia care. The Lancet 2012; 370:1447-1456.

[3] Marder VJ, et al. Hemostasis and Thrombosis. Basic Principles and Clinical Practice. 6th Edition, 2013. Milwakee, Wisconsin. Lippincott Williams and Wilkin.

[4] Franchini M, Mannucci PM. Haemophilia A in the third millennium. Blood Rev 2013; 179-84.

[5] Gomez K, et al. Key issues in inhibitor management in patients with haemophilia. Blood Transfus. 2014; 12:s319-s329.

[6] Whelan, SF, et al. Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of haemophilia A patients. Blood 2013; 121:1039-48.

Roche Group Media Relations

Phone: +41 61 688 8888 / e-mail: media.relations@roche-global.com
– Nicolas Dunant (Head)

– Patrick Barth

– Ulrike Engels-Lange

– Simone Oeschger

– Anja von Treskow

 

 

 

 

 

 

PRIME designation granted by European Medicines Agency for RG6042 for treatment of Huntington’s disease

  • European Medicines Agency PRIME (PRIority MEdicines) status is granted to medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options
  • RG6042 has the potential to be the first therapy targeting the underlying cause of Huntington’s disease, a fatal neurodegenerative rare disease
  • Third PRIME designation for a Roche medicine 

Basel, 3 August 2018 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) has granted PRIME (PRIority MEdicines) designation for the company’s investigational medicine RG6042 (formerly known as IONIS-HTTRx) for the treatment of people with Huntington’s disease (HD). RG6042 has demonstrated its ability to reduce the toxic mutant huntingtin protein (mHTT), which is believed to be the underlying cause of HD, in a Phase I/IIa study.[1] PRIME is a designation implemented by the EMA to support data generation and development plans for promising medicines, providing a pathway for accelerated evaluation by the agency, and thus potentially enable them to reach patients earlier.[2]

“We are very pleased that the European Medicines Agency has granted PRIME designation for RG6042, as there is an urgent medical need to find treatment options for families affected by Huntington’s disease,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “Preliminary data on RG6042 were the first to show that levels of toxic mutant huntingtin protein can be lowered in adults with Huntington’s disease, and we are working closely with the EMA and other health authorities to initiate a global phase III study as soon as possible.”

PRIME designation for RG6042 is primarily based on the data from an exploratory Phase I/IIa trial of RG6042 that demonstrated a significant reduction in mHTT, which breaks down the nerve cells in the brain.[1] The study demonstrated a mean 40% (up to 60%) reduction of the specific HD protein in the cerebrospinal fluid (CSF) of adult patients treated with RG6042 for three months at the two highest doses. Furthermore, levels of mHTT measured in the CSF were still declining in the majority of treated patients (~70%) as of the last measurement in the study.[3] RG6042 was well tolerated in this short initial study.[1] These data were shared at the CHDI 13th Annual HD Therapeutics Conference in March 2018,[3] and updated results were presented at the American Academy of Neurology (AAN) Annual Meeting in April 2018.[4]

Roche will initiate a pivotal phase III study to evaluate RG6042 in a larger patient population to further characterise the safety profile and determine if it can slow the progression of HD in adults.

About RG6042

RG6042 is a second-generation modified antisense oligonucleotide (ASO) designed to reduce the production and levels of mHTT protein by targeting human HTT mRNA.[5]RG6042 is the result of a comprehensive drug discovery programme between Roche and Ionis Pharmaceuticals focused on optimising the potency, specificity and tolerability of an ASO targeting human HTT mRNA. RG6042 is the most advanced compound in clinical development to target toxic mutant huntingtin protein (mHTT), which is believed to be the underlying cause of HD. Treatment with RG6042 has the potential to slow or stop disease progression in all people with HD.[1]

About Huntington’s disease

Huntington’s disease is a rare genetic, progressive condition that causes the nerve cells in the brain to break down, which severely affects a person’s everyday functions such as mobility and thinking.[6] It has a devastating impact on people living with the disease, and the hereditary nature of HD means it profoundly affects entire families.[6] As the disease progresses, people with HD may develop personality changes, difficulty walking and swallowing, as well as having a significant cognitive impact.[6] Survival ranges from approximately 10-20 years following motor onset of the disease.[6]

There is no known cure for HD and no approved therapies that treat the underlying cause. The estimates for the number of people affected by Huntington’s vary between geographic regions. Huntington’s disease is the most common monogenic neurological disorder in the developed world, with an estimated prevalence of ~3.5-7/100,000 in North America, Western Europe, and Australia.[7]

About Roche in neuroscience

Neuroscience is a major focus of research and development at Roche. The company’s goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases. Roche has more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, Alzheimer’s disease, spinal muscular atrophy, Parkinson’s disease, Huntington’s disease and autism spectrum disorder.

About Roche

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 personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.

Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines.

Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry nine years in a row by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 billion. 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.

References

[1] Ionis Pharmaceuticals. 2018. Press release: IONIS-HTT Rx (RG6042) top-line data demonstrate significant reductions of disease-causing mutant Huntingtin protein in people with Huntington’s disease. [Internet; cited 2018 July]. Available from: http://ir.ionispharma.com/node/23401/pdf

[2] European Medicines Agency. PRIME Designation. [Internet; cited 2018 July]. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/

[3] CHDI 13th Annual HD Therapeutics Conference 2018. Press Release: Ionis Pharmaceuticals Licenses IONIS-HTT Rx to Partner Following Successful Phase 1/2a Study in Patients with Huntington’s Disease. [Internet; cited 2018 July]. Available from: https://chdifoundation.org/ionis-pharmaceuticals-licenses-ionis-htt-rx-to-partner-following-successful-phase-12a-study-in-patients-with-huntingtons-disease/.  (Data on file)

[4] Ionis Pharmaceuticals. 2018. Press release: New Data from IONIS-HTT Rx Phase 1/2 Study Demonstrates Correlation Between Reduction of Disease-causing Protein and Improvement in Clinical Measures of Huntington’s Disease. [Internet; cited 2018 July]. Available from: http://ir.ionispharma.com/node/23661/pdf.
[5] Leavitt B, Tabrizi S, Kordasiewicz H et al. Discovery and early clinical development of ISIS-HTTRx, the first HTT-lowering drug to be tested in patients with Huntington’s disease (PL01.002). Neurology 2016;86(Suppl. 16):PL01.002. [Internet; cited 2018 July]. Available from: http://n.neurology.org/content/86/16_Supplement/PL01.002.
[6] NHS Choices. Huntington’s disease: Overview. [Internet; cited 2018 July]. Available from: https://www.nhs.uk/conditions/huntingtons-disease/.
[7] Rawlins M, et al. The Prevalence of Huntington’s Disease. Neuroepidemiology 2016;46:144-153.

Roche Group Media Relations

Phone: +41 61 688 8888 / e-mail: media.relations@roche-global.com
– Nicolas Dunant (Head)

– Patrick Barth

– Ulrike Engels-Lange

– Simone Oeschger

– Anja von Treskow

 

Shri J P Nadda addresses 8th BRICS Health Ministers’ Meeting at Durban; highlights the achievements of India in the health sector

“India affirms strong support for TB cooperation plan in every aspect as agreed upon in the 6th BRICS Health Ministers’ meeting held in Delhi in 2016 and in the first High-Level Meeting of the UN General Assembly on Ending Tuberculosis.”This was stated by Shri J P Nadda, Union Minister of Health and Family Welfare during his address at the ‘8th BRICS Health Ministers’ Meeting at Durban, South Africa, today. Shri J P Nadda further said that to improve healthcare, there is a need for increasing accessibility of affordable, quality, effective and safe drugs, vaccines and diagnostics for TB patients. “I would like to state that India is
committed to eliminating TB by 2025,” the Union Health Minister further emphasized.

In his address, Shri Nadda said that India today is more firmly committed to achieving Universal Health Coverage (UHC) as articulated in its National Health Policy. “India has fast-tracked many initiatives aimed at achieving all the core tenets of Universal Health Coverage i.e, strengthening health systems, improving access to free medicines and diagnostics and reducing catastrophic healthcare spending,” he added. He stated that to translate the vision of UHC, Prime Minister of India,Shri Narendra Modi has recently launched an ambitious programme called ‘Ayushman Bharat’ i.e. Long Live India. “The programme rests on the twin pillars of Health and Wellness Centres for provision of comprehensive primary healthcare services and National Health Protection Mission for secondary and tertiary care that aims to cover around 100 million families,” Shri Nadda said.

Highlighting the accomplishments of India in the health sector, Shri Nadda stated that India’s achievement in
reducing the maternal mortality ratio (MMR) by 77%, from 556 per 100000 live births in 1990 to 130 per 100
000 live births in 2016 was commended by Regional Director, WHO SEARO.

“This achievement puts the country on track to achieve the Sustainable Development Goal (SDG) target of
MMR below 70 by 2030. I would like to mention schemes like Pradhan Mantri Surakshit Matritva Abhiyan,
allowing women access to antenatal check-ups, obstetric gynaecologists and to track high-risk pregnancies,
etc., and Janani Shishu Suraksha Karyakram,”Shri Nadda stated.

During his address, the Union Health Minister said that to reduce the burden of NCDs, India has already initiated universal screening for prevention and management of five common NCDs including hypertension, diabetes and three common cancers of oral cavity, breast and cervix at pan India level. “He further said that India has started a unique initiative called AMRIT Deendayal pharmacies, an acronym for ‘Affordable Medicines & Reliable Implants for Treatment’ – Centres that provide medicines for cancer & cardiovascular diseases and cardiac implants at significantly reduced prices. “We must, therefore, renew our resolve and commitment to fight NCDs together on the BRICS platform, through R &D and innovative strategies,” Shri Nadda said.

Shri Nadda further said that there is need to promote strategies for use of traditional or alternative forms of medicine, like Ayurveda in India and the Chinese traditional remedies.“It is important that within the framework of BRICS, we carry out not only an exchange of technologies and the development of joint ventures, but also the co-generation of knowledge because, only together can we solve the most significant health problems,” the Union Health Minister added while reiterating the need for enhanced cooperation

Cabinet approves establishment of Medical College, Salempur at Deoria, Uttar Pradesh

The Cabinet Committee on Economic Affairs chaired by Prime Minister Shri Narendra Modi has approved the
proposal of the State Government of Uttar Pradesh for establishment of new medical college at Deoria at a cost of Rs.250 crore under Phase-II of the Centrally Sponsored Scheme.

Under Phase-II of the Centrally Sponsored Scheme for establishment of new medical colleges attached with
existing District/Referral hospitals, the criteria for one medical in every three Parliamentary Constituencies and one Government medical college in each State was adopted. Accordingly, a requirement of 24 additional medical colleges including eight medical colleges in Uttar Pradesh has been approved.

Out of the eight identified Blocks in Uttar Pradesh, the Block-6 consists of (70) Ghosi, (71) Salempur and (72)
Ballia Parliamentary Constituencies. The State Government of Uttar Pradesh has recommended for
establishment of Medical Colleges, Salempur at Deoria by up-gradation of District Hospital, Deoria.
The State Government has informed that Salempur is part of District Deoria and Salempur Parliamentary
Constituency is partly covered under District Deoria. The District Hospital, Deoria is also suitable as it has
sufficient number of beds and other facilities including availability of land as per MCI norms for the establishment of medical college of all possible locations in the identified Block.

  • Late-breaking Phase II exploratory analysis of investigational crenezumab to show impact on amyloid beta oligomer levels in CSF
  • Two year open-label extension updates for investigational gantenerumab will include data on effects of higher doses in reducing amyloid PET load and long-term safety

Basel, 20 July 2018 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that it will present 16 new data presentations from across its Alzheimer’s disease (AD) pipeline at this year’s Alzheimer’s Association International Conference (AAIC) from 22-26 July in Chicago, Illinois. Roche’s AD pipeline includes two late stage investigational molecules, crenezumab and gantenerumab, which are both in Phase III clinical trials, and an anti-tau molecule in Phase II.

“The range of data that Roche is presenting at AAIC is a testament to our commitment to bring new treatments to help the many millions of people living with Alzheimer’s disease,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development.

In a late-breaking session, an exploratory analysis is being presented on crenezumab from the completed phase II BLAZE and ABBY clinical trials that will show the impact of crenezumab treatment over the whole trial duration on amyloid beta oligomer levels in cerebrospinal fluid (CSF) in people with mild to moderate AD. Baseline data from the CREAD 1 study in prodromal to mild AD will also be presented. Crenezumab is an investigational, monoclonal antibody designed to preferentially bind to and promote removal of oligomers, a form of amyloid beta.

Additionally, updates from open-label extension studies of gantenerumab, including data on the effects of higher doses of gantenerumab in reducing amyloid PET load at 24-months, as well as long-term safety data, will be presented. Data on the effects of low doses of gantenerumab on amyloid and tau biomarkers in cerebrospinal fluid will also be presented. Gantenerumab is an investigational, monoclonal antibody designed to bind to aggregated beta-Amyloid and remove amyloid beta plaques. Two recently initiated Phase III GRADUATE clinical studies are evaluating the safety and efficacy of gantenerumab for the treatment of early AD.

The full range of data from Roche’s Alzheimer’s clinical development program, including investigational medicines and diagnostics, being presented at AAIC include:

Investigational

Medicine

Abstract Title

Abstract Number (type), Presentation Date, Time

Crenezumab

LATE BREAKER: Target Engagement in an AD Trial: Crenezumab Lowers Abeta Oligomer Levels in CSF

Selkoe D (oral)

 

Session: DT-02-03 Developing Topics: Recent Developments in Biomarkers,

Wednesday, July 25, 20184:45-5:00 PM

Baseline characteristics from a phase 3 trial of crenezumab in prodromal to mild Alzheimer’s disease (CREAD) Lin H, et al.

Lin H (oral)

 

Session: 01-02 Clinical Prevention and Early Alzheimer’s Disease Trials, Sunday, July 22, 2018: 8:45 AM – 9:00 AM

Gantenerumab

The Effect of Low Doses of Gantenerumab on Amyloid and Tau Biomarkers in Cerebrospinal Fluid (CSF) in the Marguerite Road Study

Voyle N (oral)

 

Session: O1-09 Clinical: Clinical Investigations in Symptomatic AD Using Abeta Targeted Therapeutics, Sunday, July 22, 2018: 2:15 – 2:30 PM

24-Month Amyloid PET Results of the Gantenerumab High-Dose Open Label Extension Studies

Klein G (oral)

 

Session: O1-09 Clinical: Clinical Investigations in Symptomatic AD Using Abeta Targeted Therapeutics, Sunday, July 22, 2018: 2:30 – 2:45 PM

Update on the Safety and Tolerability of Gantenerumab in the Ongoing Open-Label Extension (OLE) of the Marguerite Road Study in Patients with Mild Alzheimer’s Disease (AD) after Approximately Two Years of Study Duration

Abi-Saab D (oral)

 

Session: O1-09 Clinical: Clinical Investigations in Symptomatic AD Using Abeta Targeted Therapeutics, Sunday, July 22, 2018: 2:45 – 3:00 PM

Update on the Safety and Tolerability of Gantenerumab in the Ongoing Open-Label Extension of the Scarlet Road Study in Patients with Prodromal Alzheimer’s Disease after Approximately 2 Years of Study Duration

Andjelkovic M (oral)

 

Session: O1-09 Clinical: Clinical Investigations in Symptomatic AD Using A-Beta Targeted Therapeutics, Sunday, July 22, 2018: 3:00 – 3:15 PM

 

CSF Biomarkers

LATE BREAKER: Detecting brain amyloid status using fully automated plasma Abeta biomarker assays

 

Hansson O (oral)

Session: DT-02-04 Developing Topics: Recent Developments in Biomarkers,

Wednesday, July 25, 20185:00-5:15 PM

Analysis of cerebrospinal fluid (CSF) biomarkers to predict risk of clinical decline and progression to dementia in patients with mild cognitive impairment and mild cognitive symptoms

Shaw LM (poster)
Session: P3-06 Diagnosis and Prognosis: Biomarkers (non-neuroimaging) Tuesday, July 24, 20189:30 AM – 4:15 PM, McCormick Place, Hall F1

Multicenter evaluation of the analytical characteristics of the Elecsys® Total-Tau cerebrospinal fluid (CSF) and Elecsys® Phospho-Tau (181P) CSF immunoassays

Kollmorgen G (poster)
Session: P1-07 Diagnosis and Prognosis: Biomarkers (non-neuroimaging) Sunday, July 22, 2018: 9:30 AM – 4:15 PM, McCormick Place, Hall F1

Technical validation and multicenter evaluation of the Elecsys® beta-Amyloid 1-40 prototype immunoassay for quantitation in cerebrospinal fluid (CSF)

Wiegers AK (poster)
Session: P1-07 Diagnosis and Prognosis: Biomarkers (non-neuroimaging) Sunday, July 22, 2018: 9:30 AM – 4:15 PM, McCormick Place, Hall F1

A Unified Pre-Analytical Protocol for Handling of CSF Samples before Analyses of AD Biomarker Levels

Hansson O (oral) 
Session: O2-09 Biomarkers: Methods and Quality, Monday, July 23, 2018: 2:00 PM – 3:30 PM

CSF biomarkers in the general population: associations with demographics and APOE genotype

Van Harten AC (oral)

 

02-04 Biomarkers: Longitudinal Changes: Monday, July 23, 3018: 9:00 AM – 9:15 AM,

Diagnostic performance of Elecsys® immunoassays for cerebrospinal fluid Alzheimer’s disease biomarkers in a non-academic multicentre memory clinic cohort: the ABIDE project

Willemse E (oral)
Session: O2-09 Biomarkers: Methods and Quality, Monday, July 23, 2018: 2:00 PM – 3:30 PM

 

PET Tau Tracers

Tau Burden Measured Using [18F]GTP1 Correlates with CSF Tau Phosphorylation at Sites T217 and T205 More Closely Than T181

Wildsmith K (oral)

 

Session: O3-14 Biomarkers: Novel Biomarkers in Cerebrospinal Fluid (CSF), Tuesday, July 24, 20184:30 PM – 4:45 PM

 

Baseline Tau Burden Measured By [18f]GTP1 Imaging Is Associated with Subsequent Cognitive Decline in Prodromal to Mild Alzheimer’s Disease

Teng E (poster)

 

Session: P4-17 Developing Topics, Wednesday, July 25, 20189:30 AM – 4:15 PM, McCormick Place, Hall F1

Non-molecule

P# 25259 Estimand in Early Alzheimer’s Disease: Progress Update from the International Alzheimer’s Disease Scientific Working Group (AD SWG) Substream.

Delmar P (poster)

 

Session:  P4-01 [Posters Wed] Therapeutics: Clinical, Wednesday, July 25, 20189:30 AM – 4:15 PM, Hall F1


About crenezumab
Crenezumab is an investigational, monoclonal antibody designed to preferentially bind to and promote removal of neurotoxic oligomers, a form of amyloid beta. Crenezumab has an antibody backbone (IgG4) designed to minimize the inflammatory response in the brain, which may result in a lower risk of MRI abnormalities. It is currently being studied in two phase III, two-year, randomized, double-blind, placebo-controlled, multicenter clinical trials (CREAD 1 and 2) in early AD.  Based on the learnings from two completed phase II trials, the CREAD studies are using higher doses of crenezumab and have enrolled people with early AD who have confirmed AD pathology. These studies are now fully enrolled.
Crenezumab is also being studied in a landmark Alzheimer’s Prevention Initiative (API) trial of cognitively healthy individuals in Colombia with an autosomal dominant mutation who are at risk to develop early-onset AD. Crenezumab is being developed by Roche and Genentech and was discovered by Swiss biotechnology company AC Immune SA.

About gantenerumab
Gantenerumab is an investigational, monoclonal antibody designed to bind to aggregated amyloid beta and remove amyloid beta plaques. It is being investigated in two phase III studies (GRADUATE 1 and 2) for the treatment of early AD. In completed phase III clinical studies, gantenerumab removed beta amyloid plaques, which have been shown to be toxic to the brain. Ongoing open-label extension studies have informed the design of the GRADUATE program. The new studies, which are enrolling people with early AD with confirmed AD brain pathology, include higher doses of gantenerumab. The target dose is achieved through a titration regimen to optimize safety. Gantenerumab is also being studied as part of the DIAN-TU trial, a worldwide clinical study evaluating multiple compounds in individuals at risk for or with a type of early-onset AD caused by a genetic mutation. Gantenerumab is being developed by Roche and Genentech and was identified and optimized by phage display technology in cooperation with MorphoSys AG, a Munich-based Biotech.

About Alzheimer’s disease
Alzheimer’s disease is a progressive, fatal disease of the brain that gradually destroys memory, thinking skills and problem solving and impairs daily functioning such as the ability to manage one’s own activities. Biological changes are believed to start decades before clinical symptoms of Alzheimer’s disease become evident. In the early stages (prodromal to mild dementia), people may have difficulty remembering things, but daily function may or may not be impaired. In the later stage of the disease, people increasingly become reliant on others for even simple day-to-day tasks. Dementia affects 44 million people worldwide with 7.7 million new cases each year, of which Alzheimer’s disease is the most common form. There is no cure for Alzheimer’s disease. Current treatments focus on alleviating symptoms and are unable to stop Alzheimer’s from progressing because they do not affect the disease’s underlying causes.

Hear from Rachelle Doody, Roche’s Global Head of Neurodegeneration, about our strength in both diagnosis and treatment.


About Roche in neuroscience
Neuroscience is a major focus of research and development at Roche. The company’s goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases. Roche has more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, Alzheimer’s disease, spinal muscular atrophy, Parkinson’s disease, Huntington’s disease and autism spectrum disorder.

About Roche
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 personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.
Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry nine years in a row by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 billion. 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.

Roche Group Media Relations
Phone: +41 61 688 8888 / e-mail: media.relations@roche-global.com
– Nicolas Dunant (Head)
– Patrick Barth
– Ulrike Engels-Lange
– Simone Oeschger
– Anja von Treskow

20180720-Roche-MR_Alzheimers_AAIC_en

Basel, 20 July 2018 – Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today, that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Device Designation to Elecsys® beta-Amyloid (1-42) CSF and Elecsys® Phospho-Tau (181P) CSF. These in vitro diagnostic immunoassays are for the measurement of the beta-Amyloid (1-42) and Phospho-Tau concentrations in cerebrospinal fluid (CSF) in adult patients with cognitive impairment who are being evaluated for Alzheimer’s disease (AD) or other causes of dementia.

Currently, the diagnosis of AD is largely based on clinical symptoms, including cognitive testing, with a significant number of patients diagnosed when their disease has already advanced. A diagnosis of AD based on cognitive measures alone is only correct in 70 – 80 percent of cases. Measuring biomarkers with CSF immunoassays, associated with AD pathology, increases certainty of a diagnosis of AD and can help to evaluate the progression of the disease. The Breakthrough Device Designations are for indication of use with Elecsys beta-Amyloid (1-42) CSF and Elecsys Phospho-Tau (181P) CSF in concordance with amyloid PET visual read result and risk of cognitive or functional decline. The Breakthrough Devices Program is a voluntary program for certain medical devices that provide for more effective treatment or diagnosis of a life-threatening or irreversibly debilitating disease or condition. This program is designed to expedite the development and review of these medical devices.

“We are excited about FDA’s recognition of the potential clinical benefit the Elecsys CSF assays can bring to clinicians, laboratories and their patients in diagnosing AD at an early stage,” said Roland Diggelmann, CEO of Roche Diagnostics. “Roche was one of the first companies to use biomarkers in clinical trials and we will continue to explore high-performing diagnostic and disease-monitoring solutions.”

About Elecsys® CSF immunoassays
Establishing the presence of degenerative diseases of the central nervous system is important when it comes to distinguishing age-related memory disorders from early-stage dementia. Deposits of beta-amyloid peptides (1-42) and neurofibrillary tangles in the brain are the two earliest changes associated with the development of Alzheimer’s disease and can be detected by different methods. Biomarkers in the cerebrospinal fluid (CSF) help clinicians detect Alzheimer’s disease, the most common form of dementia, from an earlier stage. Currently available for countries accepting the CE Mark*, the Elecsys beta-Amyloid (1-42) CSF, Elecsys Phospho-Tau (181p) CSF and Elecsys(R) Total-Tau CSF immunoassays are in vitro tests for the quantitative determination of concentrations of the beta-amyloid (1-42), phospholylated tau (181P) protein and total Tau protein in human cerebrospinal fluid. [1-3] The markers can be used alone or in combination. [1-3] The ElectroChemiLuminescence Immunoassay “ECLIA” is intended for use on Elecsys and cobas e immunoassay analyzers. [1-3]
*
Local product availability may vary independently from CE Mark approval.

About Alzheimer’s disease
Alzheimer’s disease is a progressive, fatal disease of the brain that gradually destroys memory, thinking skills and problem solving and impairs daily functioning such as the ability to manage one’s own activities. Biological changes are believed to start decades before clinical symptoms of Alzheimer’s disease become evident. In the early stages (prodromal to mild dementia), people may have difficulty remembering things, but daily function may or may not be impaired. In the later stage of the disease, people increasingly become reliant on others for even simple day-to-day tasks. Dementia affects 44 million people worldwide with 7.7 million new cases each year, of which Alzheimer’s disease is the most common form. There is no cure for Alzheimer’s disease. Current treatments focus on alleviating symptoms and are unable to stop Alzheimer’s from progressing because they do not affect the disease’s underlying causes.

About Roche
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 personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.

Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.
Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. Thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Roche has been recognised as the Group Leader in sustainability within the Pharmaceuticals, Biotechnology & Life Sciences Industry nine years in a row by the Dow Jones Sustainability Indices (DJSI).

The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2017 employed about 94,000 people worldwide. In 2017, Roche invested CHF 10.4 billion in R&D and posted sales of CHF 53.3 billion. 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.

References
[1] Roche Diagnostics. Elecsys beta-Amyloid (1-42) CSF: Method sheet. 2018. Last accessed: June 2018.
[2] Roche Diagnostics. Elecsys Phospho-Tau CSF: Method sheet. 2018. Last accessed: June 2018.
[3] Roche Diagnostics. Elecsys Total-Tau CSF: Method sheet. 2018. Last accessed: June 2018.

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20180720-Roche_MR_Diagnostics_AAIC_en