| Friday, February 28, 2014, 17:00 Hrs [IST] |
TeraDiscoveries, Inc., a company designs new drugs and drug candidates and accelerates the process of drug discovery by substituting new innovative cloud computing techniques, has entered into a strategic partnership with Egenix, Inc., a privately held New York based biotechnology firm. The two companies are working together to design new drugs that are effective against cancer and autism.
TeraDiscoveries will use its Inverse Design software platform to design new drug molecules to effectively help Egenix accelerate its drug discovery and development process. Egenix is providing funding and target data that TeraDiscoveries will apply to identify and optimize inhibitors for certain cancer indications and autism. Egenix will develop the drugs through phase 2 while TeraDiscoveries retains a minority stake in the two drugs. Inverse Design runs computational models to quickly scan a vast chemical space to find the strongest inhibitors of a specific biological target, ones that also have good druggable properties and are predicted to have low toxic side effects. Inverse Design also has filters for many important drug properties including toxic side-effect risk assessment, off-target effects assessment, synthesizability, solubility, freedom to operate assessment. Specialized filtering can assess if a molecule crosses the blood-brain barrier. "Designing new drugs that bind to a specified protein target requires finding the best match among millions of molecules and peptides. Our approach executes the search much more efficiently and with greater accuracy than traditional methods,” says Ed Addison, CEO of TeraDiscoveries. “Outside reviewers have claimed that TeraDiscoveries saves over 75% of the time and cost of drug discovery, while providing novel, patentable composition of matter.” “They say genetics loads the gun and environment pulls the trigger. We expect that our partnership with TeraDiscoveries will help create drugs that will stop the trigger from being pulled,” says Donald Fresne, Egenix’s CEO. “We believe TeraDiscoveries to have the best in silico drug discovery platform available. Plus, TeraDiscoveries has the unique ability to filter lead compounds that cross the blood-brain barrier, which is critical for our eIF4e autism inhibitor drug.” Companies can use Inverse Design as a service and run it via the Windows Azure cloud or a private cloud to design, discover, or optimize new small molecule or peptide drugs for their targets. TeraDiscoveries also partners and licenses assets in its pipeline which include compounds for many targets including Jak2, Jak3, hdac8, Aurora A, 11b-HSD1, hsp90, MAPK10, pfDHFR-Ts, ABL1, ABL3, ROCK1, TYK2, c-Kit, among others. |
sabato 1 marzo 2014
TeraDiscoveries, Egenix partner to design new drugs for cancer and autism
Meditope receives US patent for its monoclonal antibody drug conjugates
| Friday, February 28, 2014, 18:00 Hrs [IST] |
Meditope Biosciences, Inc., a biotechnology company developing novel antibody-drug conjugates using its proprietary monoclonal antibody (mAb) technology platform, has received first US patent No. 8,658,774, entitled “Novel Meditopes and Related Meditope-Monoclonal Antibody Delivery Systems, Synthesis and Therapeutic Uses Thereof.”
“We view the issuance of this patent, about two years from initial filing, as validation of the novelty of our platform and an important step in our strategy to build and protect a strong and highly differentiated antibody technology,” said Stephanie Hsieh, Meditope’s president and chief executive officer. “Antibody drug conjugates are an area of significant opportunity in drug development and we look forward to leveraging our unique technology to address the industry’s needs.” |
Novartis marks Rare Disease Day with call for more research to understand and find treatments for rare diseases
| Saturday, March 01, 2014, 10:00 Hrs [IST] |
Novartis joins the global rare disease community in recognition of Rare Disease Day by calling for a global exchange of ideas to deepen the understanding of rare diseases and help address a significant unmet medical need.
Rare Disease Day is an annual, international awareness-raising event coordinated globally by EURORDIS and in the US by the National Organization for Rare Disorders (NORD). The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives. "Novartis is pleased to help raise the dialogue about rare diseases and the need for more research to understand these diseases better," said Dr Mark Fishman, president of the Novartis Institutes for BioMedical Research (NIBR). "Our focus on rare diseases flows from our desire to help patients underserved by today's medicines. In addition, research into rare diseases teaches us fundamental mechanisms of human biology and disease, often applicable to more prevalent disorders." For more than 50 years, Novartis has been a leader in the discovery and development of innovative therapies to treat rare diseases, from rare forms of cancer to debilitating genetic diseases. The focus on rare diseases began in 1963, when Ciba - one of the two predecessor companies of Novartis - launched Desferal, a life-saving therapy for rare hemopathies. Today Novartis has nine marketed drugs that have been designated orphan drugs as well as a robust clinical pipeline including more than 40 active preclinical and clinical research projects in the rare diseases area. While these diseases may be rare, their impact is great. There are more than 6,000 rare diseases affecting more than 60 million people across Europe and North America and millions more worldwide. For this reason, investment in both fundamental discovery and translational research is crucial. To successfully drive innovation that leads to treatments for rare diseases, Novartis believes that global efforts must continue to be focused towards understanding basic processes that go awry in such disorders, and to better understand how to translate such discoveries to new medicines. Knowledge derived from the thorough analysis of a rare disease has high scientific and societal value, because insights into rare disorders can also provide scientists with a clear understanding of disease mechanisms that can be useful to treat more common disorders. To this end, Novartis is also sponsoring and hosting RE(ACT) Congress 2014, the second international conference on research of rare and orphan diseases which is organized by the Gebert Rüf Foundation and the Blackswan Foundation. The meeting is held from March 5 to 8, 2014 at the Novartis Basel Campus. This Congress will provide the global research community with a much-needed platform to connect, exchange ideas and deepen fundamental understanding of rare diseases. This international congress brings together scientists from different disciplines - including stem cell researchers, geneticists, biochemists, clinicians and pharmacists - to discuss rare diseases and the quest for potential treatments. Novartis is also proud to be a global collaborator of choice for biotechnology companies and academic centres seeking to discover and develop drugs for a range of inadequately treated diseases. Novartis has established productive alliances with more than 300 collaborators, both academic and industrial, many focused on rare diseases. "We continue to look for new ways to combine our own scientific knowledge and expertise with that of the rest of the research community, to find the shortest path to new treatments for rare diseases," said Dr Fishman. Novartis is committed to finding treatments for rare diseases that improve the quality of life for patients living with a rare disease as well as to raising awareness of and supporting patients with rare diseases. Novartis is leading in researching and developing innovative therapies to help address the high unmet medical need in rare diseases. Rare diseases are a key strategic focus of the research strategy at NIBR where scientists are currently investigating treatments for more than 40 rare diseases. These research programs have more than 20 active orphan drug designations from the US Food and Drug Administration and European Medicines Agency or both, targeting disorders ranging from aggressive systemic mastocytosis and Cushing's disease to sporadic Inclusion Body Myositis (sIBM) and spinal muscular atrophy. Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. |
EMA accepts Cubist's tedizolid MAA for review
| Saturday, March 01, 2014, 11:00 Hrs [IST] |
The European Medicines Agency (EMA) has accepted to review Cubist Pharmaceuticals' Marketing Authorization Application (MAA) for its investigational antibiotic tedizolid phosphate. Cubist is seeking approval of tedizolid for the treatment of complicated skin and soft tissue infections (cSSTI), with a decision from the European Commission (EC) expected during the first half of 2015.
Tedizolid is a once daily oxazolidinone being developed for both intravenous (IV) and oral administration for the treatment of serious infections caused by certain Gram-positive bacteria, including those caused by methicillin-resistant Staphylococcus aureus (MRSA). The MAA submission is based on positive data from two global phase III clinical studies of tedizolid, which met the primary and secondary endpoints defined by the EMA and US Food and Drug Administration (FDA). “We are very pleased to receive MAA acceptance for tedizolid and to work with the EMA on this important review process,” said Steve Gilman, executive vice president of Research and Development and chief scientific officer of Cubist Pharmaceuticals. “As we continue to build out our global infrastructure, this is a significant development in our ongoing mission to advance potential new antibiotics for patients facing serious bacterial infections, such as MRSA, around the world.” Prior to the EMA acceptance of the MAA, the FDA accepted the Company’s New Drug Application (NDA) for tedizolid with Priority Review and assigned an action date of June 20, 2014. The FDA has asked Cubist to participate in a meeting of its Anti-Infective Drugs Advisory Committee (AIDAC) on March 31, 2014. The AIDAC will review data supporting the Company’s NDA for tedizolid, for which Cubist is seeking approval in acute bacterial skin and skin structure infections (ABSSSI). Additionally, Cubist expects to submit a New Drug Submission (NDS) to Health Canada during the first half of 2014 in acute bacterial skin and skin structure infections (ABSSSI). Complicated skin and soft tissue infections (cSSTI) and acute bacterial skin and skin structure infections (ABSSSI) are a significant and growing problem throughout the world. These infections involve deeper tissue or require surgical intervention (e.g., cellulitis, major cutaneous abscesses and infected wounds) or are associated with a significant underlying disease (e.g., diabetes or systemic immunosuppression) that complicates response to therapy. Cubist has a growing commitment to global public health through its leadership in the R&D of antibiotics to treat serious and life-threatening infections caused by a broad range of increasingly resistant bacteria. Cubist Pharmaceuticals, Inc. is a global biopharmaceutical company focused on the research, development, and commercialization of pharmaceutical products that address significant unmet medical needs in the acute care environment. |
GlobalData report says gout treatment market opportunities bolstered by rise of obesity, type 2 diabetes and lack of physician awareness
| Saturday, March 01, 2014, 14:00 Hrs [IST] |
Apart from having some leading rheumatologists’ opinion that gout is curable with available therapies, a lack of physician education, combined with restricted access to the generic uricosurics which means that new add-on therapies, such as AstraZeneca’s lesinurad, will be highly successful among the difficult-to-treat gout population, says Valentina Gburcik, analyst at GlobalData.
According to Valentina Gburcik, a GlobalData’s Analyst covering Cardiovascular & Metabolic Disorders, physicians and patients both display a low awareness of gout’s seriousness, despite its status as one of the oldest recognized disorders in humans. Indeed, Key Opinion Leaders (KOLs) interviewed by GlobalData agree that physicians need to take the disease more seriously, with some even questioning the need for novel therapies. Gburcik says: “The gout drug market is very mature and highly genericized. Allopurinol, a first-line, urate-lowering therapy (ULT), has been used for almost 40 years in over 90% of ULT-treated gout patients. Takeda’s Uloric, which reached the market in 2009, managed to steal only about 10% of allopurinol’s patient share, as the much lower cost and general effectiveness of the latter drug presented a stiff entry barrier to the branded therapy. “Despite Uloric’s struggle to penetrate the market, pharmaceutical companies perceive that the difficult-to-treat population is growing and that novel treatments are necessary.” Gburcik believes that gout’s increasing prevalence may be explained by the increasing frequency of risk factors for hyperuricemia, such as obesity, type 2 diabetes and hypertension. Patients who are unresponsive to or intolerant of standard gout therapies are also rising in number, and several companies have been trying to develop novel drugs targeting this challenging patient pool. On the other hand, KOLs interviewed by GlobalData have indicated that while novel therapies are welcome, improved physician education remains a more significant unmet need in gout treatment, as the disease is practically curable with the current standard of care. However, as Gburcik continues: “While we agree that raised awareness would support proper disease management with available therapies, consequently limiting the opportunity for novel agents, there is no sign that gout management will improve significantly over the coming years. Drug developers, such as AstraZeneca, therefore have the potential to achieve great success in this market.” GlobalData is a leading global research and consulting firm offering advanced analytics to help clients make better, more informed decisions every day. |
Anacor Pharma completes end-of-phase II meeting with US FDA for treatment of mild-to-moderate atopic dermatitis with AN2728
| California Saturday, March 01, 2014, 15:00 Hrs [IST] |
Anacor Pharmaceuticals has successfully completed an End-of-Phase II meeting with the United States Food and Drug Administration (FDA) for the topical treatment of mild-to-moderate atopic dermatitis with AN2728 Ointment, 2%, a novel boron-based phosphodiesterase-4 (PDE-4) inhibitor. Atopic dermatitis is a chronic rash characterized by inflammation and itch and affects 10 to 20 per cent of infants and young children.
"We have reached agreement with the FDA on all major parameters for the phase III trials, which we expect to initiate in the next 60 days," said David Perry, chief executive officer of Anacor Pharmaceuticals. "Based on the clinical studies conducted to date, we believe AN2728 has the potential to offer patients and physicians a safe and effective topical treatment option for mild-to-moderate atopic dermatitis, providing a potential alternative to treating with topical corticosteroids or topical calcineurin inhibitors." Anacor will conduct two multi-centre, double-blind, placebo-controlled trials with approximately 750 subjects per trial randomized 2:1 (active:vehicle). Both studies will be conducted at multiple sites and enroll subjects ages two years and up with mild-to-moderate atopic dermatitis. Mild-to-moderate atopic dermatitis is defined as an Investigator Static Global Assessment (ISGA) score of 2 ("mild") or 3 ("moderate"). The ISGA is a 5-point scale from 0 ("clear") to 4 ("severe"). AN2728 Ointment, 2% will be applied twice daily for 28 days. The primary efficacy endpoint will be treatment success at Day 29, defined as an ISGA of "Clear" or "Almost Clear" with at least a 2-grade improvement from baseline. Secondary endpoints will include an ISGA of "Clear" or "Almost Clear" at Day 29 as well as time to treatment success. Safety evaluation will include reported adverse events, safety laboratory tests, and vital signs. Anacor will also initiate a long-term safety trial to evaluate the safety of intermittent use of AN2728 Ointment, 2% for up to 12 months. Subjects who complete either phase III trial will have the option to roll into the long-term safety trial until approximately 500 subjects are enrolled. At least 100 subjects will be enrolled for 12 months and at least 300 subjects will be enrolled for six months, during which time subjects will be treated as needed under the direction of the investigator. The following four clinical studies of AN2728 in mild-to-moderate atopic dermatitis support the basis for our phase III study design: AD-202 - a double-blind, bilateral, vehicle-controlled study in 25 adults in which 52% of AN2728-treated lesions achieved total or partial clearance, defined as an Atopic Dermatitis Severity Index (ADSI) score < 2 versus 16% for vehicle after 28 days of twice-daily treatment. The ADSI score is the sum of the severity scores of five clinical features (erythema, pruritus, exudation, excoriation and lichenification) from 0 (none) to 3 (severe) for each feature, for a total score of 0 to 15. Anacor originally reported the results of this study on December 12, 2011. AD-203 - an open-label, whole-body study in 23 adolescents in which 74% of patients achieved an ISGA score of 0 ("clear") or 1 ("almost clear") after four weeks of twice-daily treatment. 35% of patients achieved an ISGA score of 0 ("clear") or 1 ("almost clear") with a minimum 2-grade improvement after four weeks of twice-daily treatment. Anacor originally reported the results of this study on December 10, 2012. AD-204 - a double-blind, bilateral, dose-ranging study in 86 adolescents in which twice daily treatment with AN2728 Ointment, 2% demonstrated the greatest improvement in treating atopic dermatitis lesions. 62% of lesions in this treatment group achieved total or partial clearance, as defined by the ADSI. Anacor originally reported the results of this study on March 21, 2013. AD-102 - a maximal use systemic exposure (MUSE) study in patients, ages two to 17 years with atopic dermatitis affecting a large percentage of their body surface area. 47% of patients achieved an ISGA score of 0 ("clear") or 1 ("almost clear") with a minimum 2-grade improvement after four weeks of twice-daily treatment with AN2728 Ointment, 2%. 65% of patients achieved an ISGA score of 0 ("clear") or 1 ("almost clear") after four weeks of twice-daily treatment with AN2728 Ointment, 2%. Anacor originally reported results of this study on November 12, 2013. In addition to these four studies, AN2728 has demonstrated safety and efficacy in 14 other phase I and phase II studies. In all studies, AN2728 was generally considered safe and well-tolerated. Most adverse events were mild and largely unrelated to study drug. Anacor is a biopharmaceutical company focused on discovering, developing and commercializing novel small-molecule therapeutics derived from its boron chemistry platform. |
sabato 15 febbraio 2014
martedì 11 febbraio 2014
venerdì 7 febbraio 2014
Thalidomide victims receive $89m compensation Settlement ends a long battle with the drug’s distributor and will compensate more than 100 Australians and New Zealanders
An $89m compensation payment for people left with birth defects after their mothers took thalidomide has been approved.
More than 100 Australian and New Zealand victims will be compensated in the landmark settlement after the Victorian supreme court signed off on the settlement on Friday.
Lawyer Peter Gordon, acting for the victims, told the court the settlement was a fair and compassionate resolution.
He said there were no objectors and no objections.
“No one has submitted any complaint,“ Gordon said.
The $89m will be paid by the drug’s distributor, Diageo, with thalidomide’s manufacturer Grunenthal not included in the agreement.
The settlement ends a long compensation battle by the thalidomide victims, many of whom were born with missing or shortened limbs.
Thalidomide, a drug to counter morning sickness, was withdrawn from sale in 1961.
The drug was distributed in Australia and New Zealand around 1960 and 1961 by Distillers, which became part of Diageo in 1997.
EffRx inks pact with 3 pharma firms to distribute its osteoporosis drug, Binosto in Italy, Spain & Portugal
EffRx Pharmaceuticals SA has signed exclusive distribution agreements with three leading local pharmaceutical companies in Italy, Spain and Portugal for Binosto - EffRx’s innovative osteoporosis medication. Under the terms of the agreement, EffRx grants exclusive marketing authorization and distribution rights to Abiogen Pharma SpA for Italy, Lacer SA for Spain, and Laboratorios Atral SA for Portugal.
Binosto is the first and only buffered solution for the treatment of osteoporosis, delivering fracture-risk reduction at the hip & spine. Binosto is administered as an effervescent tablet for oral solution and offers convenience for the patient. Binosto is currently marketed in the United States, and approved in Europe and Australia; approvals in other territories are pending. Binosto was originally developed by EffRx through an agreement with Merck & Co, Inc. granting EffRx worldwide rights to all Merck effervescent and related patents protecting alendronate.
EffRx expects the launch in these markets beginning in the second quarter 2014. Italy, Spain and Portugal are three major markets for osteoporosis treatment in Europe. EffRx is confident that the promise of Binosto will also become true in other European countries with the appointment of distribution partners that have a sizeable footprint as well as in-depth understanding of their local markets.
Christer Rosén, chairman and CEO of EffRx Pharmaceuticals said, “We are thrilled to announce the partnerships with Abiogen, Lacer, and Atral. These partners, with their well-established sales forces, are well positioned to drive Binosto to a market leadership position.”
EffRx is committed to making Binosto available to osteoporosis patients around the world and is currently discussing the commercialization of Binosto with pharmaceutical companies in other key Regions and countries.
EffRx Pharmaceuticals is an innovative specialty pharmaceutical company that exploits its proprietary technology platform to create novel therapeutic entities.
Binosto is the first and only buffered solution for the treatment of osteoporosis, delivering fracture-risk reduction at the hip & spine. Binosto is administered as an effervescent tablet for oral solution and offers convenience for the patient. Binosto is currently marketed in the United States, and approved in Europe and Australia; approvals in other territories are pending. Binosto was originally developed by EffRx through an agreement with Merck & Co, Inc. granting EffRx worldwide rights to all Merck effervescent and related patents protecting alendronate.
EffRx expects the launch in these markets beginning in the second quarter 2014. Italy, Spain and Portugal are three major markets for osteoporosis treatment in Europe. EffRx is confident that the promise of Binosto will also become true in other European countries with the appointment of distribution partners that have a sizeable footprint as well as in-depth understanding of their local markets.
Christer Rosén, chairman and CEO of EffRx Pharmaceuticals said, “We are thrilled to announce the partnerships with Abiogen, Lacer, and Atral. These partners, with their well-established sales forces, are well positioned to drive Binosto to a market leadership position.”
EffRx is committed to making Binosto available to osteoporosis patients around the world and is currently discussing the commercialization of Binosto with pharmaceutical companies in other key Regions and countries.
EffRx Pharmaceuticals is an innovative specialty pharmaceutical company that exploits its proprietary technology platform to create novel therapeutic entities.
'Indian pharma sector may undergo major consolidation in key segments'
Indian pharma sector is expected to witness a major consolidation within the industry, especially in the API sector to meet the growing market demand and challenges globally, according to Singhi Advisors, a Mumbai based global investment banking firm. Understanding the changing dynamics of the Indian pharma industry, the company expressed keen interest in providing their expertise and services in mergers and acquisitions (M&A) and capital raising initiatives to those interested in expanding their business worldwide.
Interestingly, to get better exposure in the local market, multi-nationals and larger domestic players are understood to be scouting for collaborative initiative with mid market players. Simultaneously, various mid size companies are looking at the possibility of joint ventures and M&As to enhance their chance at getting global acceptance by associating with well established players. Singhi Advisors an expert in this field will be dolling out their expertise in this area by identifying companies with growth potential but low financial and investment backing.
Mahesh Singhi, managing director of Singhi Advisors pointed out this will be a win-win situation for all especially the small and mid market cos as they will be able to get easy access to the required capital, organisational capability and exposure to advanced technologies and processes needed to upgrade their existing capacity, while opening up a great market opportunity both domestically and globally for all the parties. Singhi stressed, “Our focus will be on filling out the gaps prevalent in this area by inward integration of pharma sector by providing wider platform to our clients. This we plan to do by identifying their bottlenecks so that we can help them with proper advice by finding key partners to explore further business proposition for expanding and scaling up their business further.”
Experts inform that low entry barriers in the market have led to rapid growth of unorganised sector in the county resulting in inefficiency and duplicability of the process. Industry insiders feel that such collaborative efforts will help nullifying competition from the local market as small and mid size companies with potential can work out a proposition to either enter into a JV or merge with bigger players, which will be mutually beneficial for all.
Gopal Agrawal, partner, Singhi Advisors informs that their focus will be on identifying such companies who have high potential but lack the technological knowhow to make it into the global market, while at the same time have a very strong base in the local market. “There is a changing trend in the pharma sector, wherein various large and median companies are increasingly coming into focus due to their strong presence in the local or domestic market. This is a great sign as India has lot of mid market or even large companies with high potential who get side tracked due to lack of technological capabilities. With correct knowledge and proper valuation, we can sure integrate the best of both, to boost their business interest in a growing market,” Agrawal stressed.
Singhi Advisors have recently advised two Indian Companies viz Span Diagnostic and Sunways India in identifying strategic investors namely ARKRAY Inc. and Rohto Pharmaceuticals Inc. respectively. Both the strategic investors are from Japan which again is the key focus country with Singhi where they have done five transactions over last one year, being highest by any Advisory firm in India.
The company also plans to focus on applying this strategy in the medical device and biotech sector, as most of the key players in the domestic market especially in the medical device sector belong to the SMEs with a few global giants dominating the market.
Interestingly, to get better exposure in the local market, multi-nationals and larger domestic players are understood to be scouting for collaborative initiative with mid market players. Simultaneously, various mid size companies are looking at the possibility of joint ventures and M&As to enhance their chance at getting global acceptance by associating with well established players. Singhi Advisors an expert in this field will be dolling out their expertise in this area by identifying companies with growth potential but low financial and investment backing.
Mahesh Singhi, managing director of Singhi Advisors pointed out this will be a win-win situation for all especially the small and mid market cos as they will be able to get easy access to the required capital, organisational capability and exposure to advanced technologies and processes needed to upgrade their existing capacity, while opening up a great market opportunity both domestically and globally for all the parties. Singhi stressed, “Our focus will be on filling out the gaps prevalent in this area by inward integration of pharma sector by providing wider platform to our clients. This we plan to do by identifying their bottlenecks so that we can help them with proper advice by finding key partners to explore further business proposition for expanding and scaling up their business further.”
Experts inform that low entry barriers in the market have led to rapid growth of unorganised sector in the county resulting in inefficiency and duplicability of the process. Industry insiders feel that such collaborative efforts will help nullifying competition from the local market as small and mid size companies with potential can work out a proposition to either enter into a JV or merge with bigger players, which will be mutually beneficial for all.
Gopal Agrawal, partner, Singhi Advisors informs that their focus will be on identifying such companies who have high potential but lack the technological knowhow to make it into the global market, while at the same time have a very strong base in the local market. “There is a changing trend in the pharma sector, wherein various large and median companies are increasingly coming into focus due to their strong presence in the local or domestic market. This is a great sign as India has lot of mid market or even large companies with high potential who get side tracked due to lack of technological capabilities. With correct knowledge and proper valuation, we can sure integrate the best of both, to boost their business interest in a growing market,” Agrawal stressed.
Singhi Advisors have recently advised two Indian Companies viz Span Diagnostic and Sunways India in identifying strategic investors namely ARKRAY Inc. and Rohto Pharmaceuticals Inc. respectively. Both the strategic investors are from Japan which again is the key focus country with Singhi where they have done five transactions over last one year, being highest by any Advisory firm in India.
The company also plans to focus on applying this strategy in the medical device and biotech sector, as most of the key players in the domestic market especially in the medical device sector belong to the SMEs with a few global giants dominating the market.
Omeros' OMS824 Huntington's disease programme receives US FDA fast track designation
The US Food and Drug Administration (FDA) has granted Fast Track designation to Omeros Corporation's phosphodiesterase 10 (PDE10) inhibitor, OMS824 for the treatment of cognitive impairment in patients with Huntington's disease. OMS824 selectively inhibits PDE10, an enzyme expressed in areas of the brain linked to a wide range of diseases that affect cognition, including Huntington's disease and schizophrenia.
Omeros has conducted successful clinical trials assessing the safety, tolerability, pharmacokinetics and target engagement of a wide range of doses of OMS824 in its phase I programme. Positive data from the company's OMS824 phase IIa schizophrenia trial were recently announced, and Omeros expects to begin enrolling patients this quarter in its phase II trial evaluating OMS824 for Huntington's disease.
FDA's Fast Track programme facilitates the development of drugs intended to treat serious or life-threatening conditions and that have the potential to address unmet medical needs. A drug programme with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval. Many drugs that receive Fast Track designation are also considered appropriate to receive Priority Review, and their respective New Drug Applications (NDAs) may be accepted by the FDA as a "rolling submission" in which portions of an NDA are reviewed before the complete application is submitted. Priority Review and rolling submission can each provide further acceleration of FDA's approval process.
"FDA's Fast Track designation of OMS824 for Huntington's disease reflects the unmet need associated with cognitive impairment in Huntington's patients and recognizes the drug's potential to treat this condition," stated Gregory A Demopulos, MD, chairman and chief executive officer of Omeros. "Together with the orphan drug status previously awarded OMS824 by the FDA for Huntington's, we have the opportunity to streamline the development of this promising compound. We look forward to enrolling patients in our OMS824 phase II clinical trial for Huntington's disease within the next few weeks."
PDE10 is an enzyme that is expressed in areas of the brain linked to diseases that affect cognition and psychomotor functions, including Huntington's disease and schizophrenia. Huntington's disease is a hereditary neurodegenerative disorder that leads to movement, cognition, and behavioral abnormalities and premature death. Schizophrenia is a group of severe brain disorders characterized by an abnormal interpretation of reality, which can manifest as delusions, hallucinations, and/or disordered thinking and behavior. Cognitive dysfunction is responsible for substantial disability in both of these diseases and is not meaningfully improved by current medications.
Omeros' proprietary compound OMS824, currently in phase II clinical programmes, inhibits PDE10 and is being developed for the treatment of cognitive disorders. In addition to potential benefits on cognition, OMS824 could also improve the motor and psychiatric abnormalities in Huntington's disease as well as the positive (e.g., hallucinations) and negative (e.g., flat affect) symptoms of schizophrenia. Omeros has been awarded both Orphan Drug and Fast Track Designations by the US FDA to evaluate OMS824 in Huntington's disease, and a Fast Track application to the FDA for the evaluation of OMS824 in schizophrenia is currently under review.
Omeros is a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics targeting inflammation, coagulopathies and disorders of the central nervous system.
Omeros has conducted successful clinical trials assessing the safety, tolerability, pharmacokinetics and target engagement of a wide range of doses of OMS824 in its phase I programme. Positive data from the company's OMS824 phase IIa schizophrenia trial were recently announced, and Omeros expects to begin enrolling patients this quarter in its phase II trial evaluating OMS824 for Huntington's disease.
FDA's Fast Track programme facilitates the development of drugs intended to treat serious or life-threatening conditions and that have the potential to address unmet medical needs. A drug programme with Fast Track status is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval. Many drugs that receive Fast Track designation are also considered appropriate to receive Priority Review, and their respective New Drug Applications (NDAs) may be accepted by the FDA as a "rolling submission" in which portions of an NDA are reviewed before the complete application is submitted. Priority Review and rolling submission can each provide further acceleration of FDA's approval process.
"FDA's Fast Track designation of OMS824 for Huntington's disease reflects the unmet need associated with cognitive impairment in Huntington's patients and recognizes the drug's potential to treat this condition," stated Gregory A Demopulos, MD, chairman and chief executive officer of Omeros. "Together with the orphan drug status previously awarded OMS824 by the FDA for Huntington's, we have the opportunity to streamline the development of this promising compound. We look forward to enrolling patients in our OMS824 phase II clinical trial for Huntington's disease within the next few weeks."
PDE10 is an enzyme that is expressed in areas of the brain linked to diseases that affect cognition and psychomotor functions, including Huntington's disease and schizophrenia. Huntington's disease is a hereditary neurodegenerative disorder that leads to movement, cognition, and behavioral abnormalities and premature death. Schizophrenia is a group of severe brain disorders characterized by an abnormal interpretation of reality, which can manifest as delusions, hallucinations, and/or disordered thinking and behavior. Cognitive dysfunction is responsible for substantial disability in both of these diseases and is not meaningfully improved by current medications.
Omeros' proprietary compound OMS824, currently in phase II clinical programmes, inhibits PDE10 and is being developed for the treatment of cognitive disorders. In addition to potential benefits on cognition, OMS824 could also improve the motor and psychiatric abnormalities in Huntington's disease as well as the positive (e.g., hallucinations) and negative (e.g., flat affect) symptoms of schizophrenia. Omeros has been awarded both Orphan Drug and Fast Track Designations by the US FDA to evaluate OMS824 in Huntington's disease, and a Fast Track application to the FDA for the evaluation of OMS824 in schizophrenia is currently under review.
Omeros is a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics targeting inflammation, coagulopathies and disorders of the central nervous system.
Indian oncologists upset with Roche’s move to stall marketing of biosimilar drug for breast cancer trastuzumab
Indian oncologists are annoyed over the Swiss drug major Roche's recent move to stall marketing of biosimilar drug for breast cancer trastuzumab by the biosimilar manufacturing majors Biocon and Mylan. They are upset that the poor breast cancer patients in the country cannot have access to affordable drugs.
It is reported that in 2012, the global sales for Herceptin is US$ 6.4 billion and in India it witnessed sales of US$ 21 million.
Standard treatments for breast cancer include surgery, chemotherapy, hormone therapy and targeted therapies, including biologics. The survival rate for breast cancer patients in the US has improved to nearly 90 per cent after the introduction of biologics, improved screening, and other treatment enhancements.
The concept of affordable cancer drugs being made available to the poor Indian patients has not been acceptable by the multinational company, said a section of pharma consultants.
Disallowing patient’s access to affordable and quality drugs is not the right move. The multinational companies have done it again and want to indicate their muscle power in the cancer medication space. All MNC drugs are expensive and unaffordable to the poor patients, stated a section of junior doctors from the Kidwai Institute of Oncology who did not want to be named.
“Biocon and Mylan are pedigreed, world class companies with stellar R&D facilities, staffed by very capable scientists who are known for innovation. They are not new to the world of biologicals and have several biological products to their credit such as Insulin analogues, and growth factors available across the world, serving the cause of millions of disadvantaged,” points out Dr Vineet Gupta, director, HealthCare Global Enterprises.
Both Canmab and Hertraz are molecules that every Indian should be proud of, as they offer hope to thousands of women with breast cancer who otherwise would not have been able to afford the multinational molecule such as Herclone or Biceltis, added Dr Gupta.
How many women must die before affordable trastuzumab is available, and when will the government wake up and heed to the voice of the feeble, questions Dr Gupta
It is reported that in 2012, the global sales for Herceptin is US$ 6.4 billion and in India it witnessed sales of US$ 21 million.
Standard treatments for breast cancer include surgery, chemotherapy, hormone therapy and targeted therapies, including biologics. The survival rate for breast cancer patients in the US has improved to nearly 90 per cent after the introduction of biologics, improved screening, and other treatment enhancements.
The concept of affordable cancer drugs being made available to the poor Indian patients has not been acceptable by the multinational company, said a section of pharma consultants.
Disallowing patient’s access to affordable and quality drugs is not the right move. The multinational companies have done it again and want to indicate their muscle power in the cancer medication space. All MNC drugs are expensive and unaffordable to the poor patients, stated a section of junior doctors from the Kidwai Institute of Oncology who did not want to be named.
“Biocon and Mylan are pedigreed, world class companies with stellar R&D facilities, staffed by very capable scientists who are known for innovation. They are not new to the world of biologicals and have several biological products to their credit such as Insulin analogues, and growth factors available across the world, serving the cause of millions of disadvantaged,” points out Dr Vineet Gupta, director, HealthCare Global Enterprises.
Both Canmab and Hertraz are molecules that every Indian should be proud of, as they offer hope to thousands of women with breast cancer who otherwise would not have been able to afford the multinational molecule such as Herclone or Biceltis, added Dr Gupta.
How many women must die before affordable trastuzumab is available, and when will the government wake up and heed to the voice of the feeble, questions Dr Gupta
giovedì 6 febbraio 2014
martedì 4 febbraio 2014
Trend towards advanced specialty healthcare accelerating in the Middle East
Industry leaders at Arab Health 2014 to review delivery of healthcare services in the Middle East
Dubai, UAE: Although science and technology will undoubtedly play a significant role in enhancing
and enabling better healthcare provision in the future, it will be equally important for countries in
the Middle East to focus on improving the delivery of healthcare services and associated business
processes such as the development of primary care and specialised care services. This issue will be debated at the Leaders in Healthcare Conference during the 39th Arab Health
Exhibition & Congress from 27 -30 January 2014 at the Dubai International Convention & Exhibition
Center.
According to Professor Lord Darzi of Denham, Hamlyn Chair of Surgery, Director, Institute of Global
Health Innovation, Imperial College London, UK, and speaker at the Leaders in Healthcare
Conference, “The Middle East should move towards specialised care, but in parallel to developing
effective primary care services which should form the basic foundation of a healthcare system. There
is a need to build efficient and disease-specific models of prevention and specialised care that
deliver quality outcomes in the most cost-effective way possible. One example of this is Qatar’s
National Cancer Strategy. This looks at all aspects of cancer prevention and management drawing on
robust international evidence, and is reshaping the treatment and support patients receive. The
Middle East is also increasingly attracting international academic and clinical research talent and
emerging as a location for biomedical research. We are starting to see this feed into the
development of specialised care services and the creation of disease-specific research strategies.”
Healthcare spend in the region is expected to reach $133.19bn in 20181
. Lord Darzi believes that
regional investment opportunities include the development of regional specialist providers,
enhanced primary care services, the development of regionally based capacity to manufacture
pharmaceuticals and medical equipment, and the development of a regional talent pool to reduce
the reliance on imported human capital. These are all growth areas in the region’s healthcare sector. Commenting on this growth trajectory, Steven J. Thompson, Senior Vice President, Johns Hopkins
Medicine and CEO, Johns Hopkins Medicine International says, “Over the past decade Johns Hopkins
Medicine has engaged in a number of exciting and highly successful health care collaborations in the
Middle East. Many government agencies and leading companies in the region have placed a high
priority on increasing the accessibility, quality and safety of health care delivery, as well as on adding
advanced specialty care. More hospitals in the Middle East are becoming world class institutions,
and that trend seems only to be accelerating.”
1 Arabianindustry.com 3 June 2013 http://bit.ly/1bcT9WjScience and technology will play a significant role in enhancing and enabling better healthcare
provision in the future and managing spiraling healthcare costs. “For example, we are already seeing the impact that smartphones and digital applications are having
as a cost effective and patient targeted method of managing chronic diseases like diabetes. These
will change the landscape of primary and community care delivery over the next decade. Personalised medicine will revolutionise healthcare by allowing for the tailoring of treatments to
each patient. Gone will be the days of trial and error; prescribing courses of treatment that do not
work. Through a combination of genomic information, tissue imaging data and clinical outcomes, we
will know what will work for a particular patient with a certain genomic profile,” says Lord Darzi.
-END- Note to Editors
About Arab Health Exhibition & Congress: Arab Health Exhibition & Congress is the largest healthcare event in the Middle East. Established 39
years ago, Arab Health provides a platform for the world’s leading manufacturers, wholesalers and
distributors to meet the medical and scientific community in the Middle East and subcontinent. Arab
Health, organised by Informa Life Sciences Exhibitions, takes place from 27-30 January 2014 at the
Dubai International Convention & Exhibition Centre. With more than 3900 exhibiting companies
from 63 countries and 19 healthcare conferences with an estimated 9000 delegates, Arab Health is a
much anticipated addition to the 2014 medical event calendar. Website: www.arabhealthonline.com
About Institute of Global Health Innovation:
Website: http://www3.imperial.ac.uk/global-health-innovation
About Johns Hopkins Medicine International:
Website: http://www.hopkinsmedicine.org/international/
For press enquires please contact:
Inga Stevens
Senior Communication Manager, Informa Life Sciences Exhibitions
T: +971 4 407 2743
inga.stevens@informa.com
Dubai, UAE: Although science and technology will undoubtedly play a significant role in enhancing
and enabling better healthcare provision in the future, it will be equally important for countries in
the Middle East to focus on improving the delivery of healthcare services and associated business
processes such as the development of primary care and specialised care services. This issue will be debated at the Leaders in Healthcare Conference during the 39th Arab Health
Exhibition & Congress from 27 -30 January 2014 at the Dubai International Convention & Exhibition
Center.
According to Professor Lord Darzi of Denham, Hamlyn Chair of Surgery, Director, Institute of Global
Health Innovation, Imperial College London, UK, and speaker at the Leaders in Healthcare
Conference, “The Middle East should move towards specialised care, but in parallel to developing
effective primary care services which should form the basic foundation of a healthcare system. There
is a need to build efficient and disease-specific models of prevention and specialised care that
deliver quality outcomes in the most cost-effective way possible. One example of this is Qatar’s
National Cancer Strategy. This looks at all aspects of cancer prevention and management drawing on
robust international evidence, and is reshaping the treatment and support patients receive. The
Middle East is also increasingly attracting international academic and clinical research talent and
emerging as a location for biomedical research. We are starting to see this feed into the
development of specialised care services and the creation of disease-specific research strategies.”
Healthcare spend in the region is expected to reach $133.19bn in 20181
. Lord Darzi believes that
regional investment opportunities include the development of regional specialist providers,
enhanced primary care services, the development of regionally based capacity to manufacture
pharmaceuticals and medical equipment, and the development of a regional talent pool to reduce
the reliance on imported human capital. These are all growth areas in the region’s healthcare sector. Commenting on this growth trajectory, Steven J. Thompson, Senior Vice President, Johns Hopkins
Medicine and CEO, Johns Hopkins Medicine International says, “Over the past decade Johns Hopkins
Medicine has engaged in a number of exciting and highly successful health care collaborations in the
Middle East. Many government agencies and leading companies in the region have placed a high
priority on increasing the accessibility, quality and safety of health care delivery, as well as on adding
advanced specialty care. More hospitals in the Middle East are becoming world class institutions,
and that trend seems only to be accelerating.”
1 Arabianindustry.com 3 June 2013 http://bit.ly/1bcT9WjScience and technology will play a significant role in enhancing and enabling better healthcare
provision in the future and managing spiraling healthcare costs. “For example, we are already seeing the impact that smartphones and digital applications are having
as a cost effective and patient targeted method of managing chronic diseases like diabetes. These
will change the landscape of primary and community care delivery over the next decade. Personalised medicine will revolutionise healthcare by allowing for the tailoring of treatments to
each patient. Gone will be the days of trial and error; prescribing courses of treatment that do not
work. Through a combination of genomic information, tissue imaging data and clinical outcomes, we
will know what will work for a particular patient with a certain genomic profile,” says Lord Darzi.
-END- Note to Editors
About Arab Health Exhibition & Congress: Arab Health Exhibition & Congress is the largest healthcare event in the Middle East. Established 39
years ago, Arab Health provides a platform for the world’s leading manufacturers, wholesalers and
distributors to meet the medical and scientific community in the Middle East and subcontinent. Arab
Health, organised by Informa Life Sciences Exhibitions, takes place from 27-30 January 2014 at the
Dubai International Convention & Exhibition Centre. With more than 3900 exhibiting companies
from 63 countries and 19 healthcare conferences with an estimated 9000 delegates, Arab Health is a
much anticipated addition to the 2014 medical event calendar. Website: www.arabhealthonline.com
About Institute of Global Health Innovation:
Website: http://www3.imperial.ac.uk/global-health-innovation
About Johns Hopkins Medicine International:
Website: http://www.hopkinsmedicine.org/international/
For press enquires please contact:
Inga Stevens
Senior Communication Manager, Informa Life Sciences Exhibitions
T: +971 4 407 2743
inga.stevens@informa.com
Big Data to drive reduced healthcare costs and improved quality of care in GCC
International case studies to be presented at Big Data Conference at Arab Health 2014
Dubai, UAE: The GCC has all of the necessary factors to enable Big Data to launch with success.
There are several large healthcare systems and regulators bringing together enormous amounts of
data in real time about their patients and populations. The region also has access to cutting edge IT
and mobile technologies to collect and manage the data. More importantly, it is equipped with the
tools and growing talent to perform analytics in the healthcare sector. A panel of international and regional experts will highlight the global outcomes of implementing Big
Data in healthcare during the Big Data Conference at the 39th Arab Health Exhibition & Congress
from 28-29 January 2014 at the Dubai International Convention & Exhibition Centre.
There is a worldwide divide between laboratory scientists who continuously produce new research
findings and medical professionals in their clinical settings who need to be aware of these results.
The same applies to patient medical records that need to be accessible across a number of
specialties. This divide is more pronounced in the developing world due to the lack of conscious
effort to translate research findings into clinical practice through analytics. Bridging this gap through
sharing the latest health information will have a positive impact on patients and can even be
lifesaving.
According to Daniel Whitehead, MENA Healthcare Lead, Booz Allen Hamilton, “Using data analytics
on electronic health records in real time can offer enormous benefits for patients in this region. For
example, predictive analytics can be used to identify which patients are more at risk of life
threatening complications in the ER. Moreover, in the management of chronic diseases using mobile
monitoring applications, Big Data can help identify which behaviours are associated with negative
outcomes and which interventions are most effective.”
“There has been a lot of buzz around the potential for ‘Big Data’ to enable better decision making,”
says Mike Swinford, CEO, GE Global Healthcare Services. “Adding in human insight and process
control with analytical structures is what GE calls the ‘Industrial Internet’; Intelligent Machines –
Intelligent Information – Intelligent People. This is what the Industrial Internet is all about. In
healthcare, this strategic approach is a way to drive optimisation of assets, reduce operational
inefficiencies and improve clinical quality, all while reducing costs.”
According to Dr Abdul Rezzak Hamzeh, Senior Scientific Coordinator, Centre for Arab Genomic
Studies, Dubai, UAE, “A working example of using Big Data in healthcare in the GCC is through the
CTGA (Catalogue for Transmission Genetics in Arabs) database at the Centre for Arab Genomic
Studies in Dubai. The CTGA database is the largest ethnic-based genetic database worldwide and it
currently hosts a collection of over 1600 records of genetic disorders and their related genes. These
records provide good quality coverage of both clinical and molecular aspects of the geneticdisorder, and more importantly they offer comprehensive and updated coverage of research results
on these disorders in Arab populations.”
-END- Note to Editors
About Arab Health Exhibition & Congress
Arab Health Exhibition & Congress is the largest healthcare event in the Middle East. Established 39
years ago, Arab Health provides a platform for the world’s leading manufacturers, wholesalers and
distributors to meet the medical and scientific community in the Middle East and subcontinent. Arab
Health, organised by Informa Life Sciences Exhibitions, takes place from 27-30 January 2014 at the
Dubai International Convention & Exhibition Centre. With more than 3900 exhibiting companies
from 63 countries and 19 healthcare conferences with an estimated 9000 delegates, Arab Health is a
much anticipated addition to the 2014 medical event calendar.
Website: www.arabhealthonline.com
About Booz Allen Hamilton: Website: www.boozallen.com
About GE Global Healthcare:
Website: www.gehealthcare.com
About Arabic Genetic Centre:
Website: www.cags.org.ae
For press enquires please contact:
Weaam El-Ataya
PR & Social Media Executive
Informa Life Sciences Exhibitions
T: +971 4 408 2813
weaam.elataya@informa.com
Dubai, UAE: The GCC has all of the necessary factors to enable Big Data to launch with success.
There are several large healthcare systems and regulators bringing together enormous amounts of
data in real time about their patients and populations. The region also has access to cutting edge IT
and mobile technologies to collect and manage the data. More importantly, it is equipped with the
tools and growing talent to perform analytics in the healthcare sector. A panel of international and regional experts will highlight the global outcomes of implementing Big
Data in healthcare during the Big Data Conference at the 39th Arab Health Exhibition & Congress
from 28-29 January 2014 at the Dubai International Convention & Exhibition Centre.
There is a worldwide divide between laboratory scientists who continuously produce new research
findings and medical professionals in their clinical settings who need to be aware of these results.
The same applies to patient medical records that need to be accessible across a number of
specialties. This divide is more pronounced in the developing world due to the lack of conscious
effort to translate research findings into clinical practice through analytics. Bridging this gap through
sharing the latest health information will have a positive impact on patients and can even be
lifesaving.
According to Daniel Whitehead, MENA Healthcare Lead, Booz Allen Hamilton, “Using data analytics
on electronic health records in real time can offer enormous benefits for patients in this region. For
example, predictive analytics can be used to identify which patients are more at risk of life
threatening complications in the ER. Moreover, in the management of chronic diseases using mobile
monitoring applications, Big Data can help identify which behaviours are associated with negative
outcomes and which interventions are most effective.”
“There has been a lot of buzz around the potential for ‘Big Data’ to enable better decision making,”
says Mike Swinford, CEO, GE Global Healthcare Services. “Adding in human insight and process
control with analytical structures is what GE calls the ‘Industrial Internet’; Intelligent Machines –
Intelligent Information – Intelligent People. This is what the Industrial Internet is all about. In
healthcare, this strategic approach is a way to drive optimisation of assets, reduce operational
inefficiencies and improve clinical quality, all while reducing costs.”
According to Dr Abdul Rezzak Hamzeh, Senior Scientific Coordinator, Centre for Arab Genomic
Studies, Dubai, UAE, “A working example of using Big Data in healthcare in the GCC is through the
CTGA (Catalogue for Transmission Genetics in Arabs) database at the Centre for Arab Genomic
Studies in Dubai. The CTGA database is the largest ethnic-based genetic database worldwide and it
currently hosts a collection of over 1600 records of genetic disorders and their related genes. These
records provide good quality coverage of both clinical and molecular aspects of the geneticdisorder, and more importantly they offer comprehensive and updated coverage of research results
on these disorders in Arab populations.”
-END- Note to Editors
About Arab Health Exhibition & Congress
Arab Health Exhibition & Congress is the largest healthcare event in the Middle East. Established 39
years ago, Arab Health provides a platform for the world’s leading manufacturers, wholesalers and
distributors to meet the medical and scientific community in the Middle East and subcontinent. Arab
Health, organised by Informa Life Sciences Exhibitions, takes place from 27-30 January 2014 at the
Dubai International Convention & Exhibition Centre. With more than 3900 exhibiting companies
from 63 countries and 19 healthcare conferences with an estimated 9000 delegates, Arab Health is a
much anticipated addition to the 2014 medical event calendar.
Website: www.arabhealthonline.com
About Booz Allen Hamilton: Website: www.boozallen.com
About GE Global Healthcare:
Website: www.gehealthcare.com
About Arabic Genetic Centre:
Website: www.cags.org.ae
For press enquires please contact:
Weaam El-Ataya
PR & Social Media Executive
Informa Life Sciences Exhibitions
T: +971 4 408 2813
weaam.elataya@informa.com
domenica 2 febbraio 2014
WT1 as a biomarker to predict relapse in patients with acute leukemia and MDS
Relapse is a major obstacle to maximizing curative potential of allogeneic HCT (aHCT) therapy for acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL)) and myelodysplastic syndrome (MDS). It has been shown that relapse can be detected through longitudinal application of quantitative PCR (qPCR) measurement techniques before patients (Pts) develop symptoms and that early intervention to delay or stop relapse can improve patient outcome. However, such tests apply only to a small percentage of Pts with fusion gene transcripts and specific mutations. WT1 has been investigated as a tumor marker in a variety of studies which have shown its association with disease progression and relapse. In our pilot work, based on WT1 kinetics data in a small cohort of Pts, we have shown the striking correspondence of increasing WT1 transcript levels with future relapse and the specific time frame between molecular relapse detected by the WT1 PCR test and hematologic relapse confirmed by clinical observations. Our plan is to establish ranges of WT1 transcript levels characteristic of Pts after aHCT that do not relapse, a high threshold level that can serve as a specific biomarker for relapse with close to or 100% specificity, and a less stringent threshold level with lower specificity to identify more Pts that relapse and at earlier times. We will also define the time interval when relapse is detected molecularly by qPCR methods, and later confirmed morphologically by standard clinical methods. Our hypothesis is that many of the Pts that will ultimately relapse will have a period of minimal residual disease that may be detected by repeated elevations of WT1 not exceeding the determined WT1 threshold levels. The ultimate purpose of our study is to apply this important biomarker as part of standard diagnostic procedures for detection of relapse in the highest-risk individuals – acute leukemia and MDS Pts undergoing aHCT.
FIRST v4.0 - Financial Impact of Recurrence Score Testing
FIRST v4.0 has been designed to
- Predict the financial implications to a health care plan of adopting the Oncotype DX® breast cancer assay for use in women with ER-positive early-stage invasive breast cancer.
- To permit plans to input their own characteristics and expected use of the Oncotype DX breast cancer assay in determining the expected costs to the plan.
Inputs and data sources
Table 3. Inputs used in FIRST v4.0, separated into 8 categories.
Data to inform the default values for many of the inputs were obtained from published materials, either from systematic review of the peer-reviewed literature or from publicly available data from government sources. The Navigation bar of FIRST v4.0 provides user access to information on source data (Figure 6).
Many of the inputs values are assumed to be plan specific, and the user is permitted to change these inputs to fit their individual plan’s characteristics. Ranges have been provided for several of the variables, designed to reflect the variability found in the published sources.
Results and computations
Table 4. FIRST v4.0 outputs
User interface
There are two default value options that differ in their data sources for the frequency of RS risk groups. Figure 6 below shows the base case inputs given default values in which the RS risk group frequencies are based on a meta-analysis of published literature.2, 4, 10, 21-32
Figure 6. FIRST v4.0: Budget impact model inputs
Showing default values: risk group frequency values from literature meta-analysis, base case.
Showing default values: risk group frequency values from literature meta-analysis, base case.
Choosing Genomic health sales data default values changes the RS risk group frequency values (Input no. 4) to 57%, 32%, and 11% for low, intermediate and high risk groups in node negative patients. For node positive patients, the risk group frequencies would be, in the same order, 59%, 33%, and 8%.
Scenario analysis tools include lower bound, upper bound and breakeven analyses. The lower bound tool button models a scenario in which test utilization (Input no. 2) is 80% and the costs associated with chemotherapy (Input no. 7) are increased by 25%. In the upper bound scenario, test utilization is 50% and the costs associated with chemotherapy are decreased by 25%. Figure 7 below shows the results of the base case, lower bound and upper bound scenario analyses for both default values options in an abbreviated and condensed presentation of the results interface.
Figure 7. FIRST v4.0 interface. Budget impact model results
A. Default risk group fequency values: Literature meta-analysis
A. Default risk group fequency values: Literature meta-analysis
B. Default risk group frequency values: Genomic Health sales data
All computations – except for computing cost of recurrence – involve simple algebra. For example, the projected number of women per year with node-negative, ER-positive invasive breast cancer is the product of
- number of covered lives in the plan
- age-adjusted incidence of invasive breast cancer
The projected number of women per year to be tested is product of
- number of covered lives in the plan
- age-adjusted incidence of invasive breast cancer
- percent of women to be tested with the Oncotype DX breast cancer assay
The costs associated with change in chemotherapy use are computed as product of
- net change in chemotherapy use due to the Oncotype DX breast cancer assay
- associated costs for each cost component (drugs, supportive care, or adverse events)
Estimating the effect of the Oncotype DX breast cancer assay on late costs of recurrence involves somewhat more detailed computations. FIRST v4.0 predicts when recurrence occurs, assigns a cost to that recurrence, and then adjusts the cost to net present value in 2010 US dollars, using a fixed annual discount rate of 3%.
Key findings (Budget impact model)
- Every scenario examined results in a reduction in total costs (Table 5).
- Net reduction of adjuvant chemotherapy use results in savings in the costs of chemotherapy drugs, administration, supportive care, and adverse events. (per patient tested per year)
- Literature-based RS risk group frequency: $5,603 (range $4,202 to $7,004) savings.
- GHI sales data: $6,744 (range $5,058 to $8,430) savings.
- The humanistic benefits of avoiding adjuvant chemotherapy include preventing early toxicities (nausea and vomiting, alopecia) and late toxicities (development of new primary tumors, ovarian failure, and cognitive dysfunction).
Table 5. Scenarios results summary for a 2 million member plan
References
2. Albain KS, Barlow WE, Shak S, et al. Prognostic and predictive value of the 21-gene recurrence score® assay in postmenopausal women with node-positive, oestrogen-receptor-positive breast cancer on chemotherapy: a retrospective analysis of a randomised trial. Lancet Oncol 2010;11:55-65.
4. Goldstein LJ, Gray R, Badve S, et al. Prognostic utility of the 21-gene assay in hormone receptor-positive operable breast cancer compared with classical clinicopathologic features. J Clin Oncol 2008;26:4063-71.
10. Klang SH, Hammerman A, Liebermann N, Efrat N, Doberne J, Hornberger J. Economic implications of 21-gene breast cancer risk assay from the perspective of an Israeli-managed health-care organization. Value Health 2010;13:381-7.
21. Lo SS, Mumby PB, Norton J, et al. Prospective multicenter study of the impact of the 21-gene recurrence score assay on medical oncologist and patient adjuvant breast cancer treatment selection. J Clin Oncol 2010;28:1671-6.
22. Asad J, Jacobson AF, Estabrook A, et al. Does Oncotype DX recurrence score affect the management of patients with early-stage breast cancer? Am J Surg 2008;196:527-9.
23. Erb C, Fox K, Patel M. Evaluation of practice patterns in the treatment of node-negative, hormone-receptor positive breast cancer patients with the use of the Oncotype DX assay at the University of Pennsylvania. Abstract #3082. In: 30th Annual San Antonio Breast Cancer Symposium. San Antonio, TX; 2007.
24. Habel LA, Shak S, Jacobs MK, et al. A population-based study of tumor gene expression and risk of breast cancer death among lymph node-negative patients. Breast Cancer Res 2006;8:R25.
25. Liang H, Brufsky A, Lembersky B. A retrospective analysis of the impact of Oncotype DX low recurrence score results on treatment decisions in a single academic breast cancer center. Abstract #2061. In: 30th Annual San Antonio Breast Cancer Symposium. San Antonio TX; 2007.
26. Liebermann N, Baehner FL, Soussan-Gutman L, Klang S, Yoshizawa C, Shak S. Evaluation of Recurrence Score, nodal status and traditional clinicopathologic metrics in a large ER positive patient cohort. Abstract #1420. In: 2011 European Society for Medical Oncology. Providence, RI; 2011.
27. Oratz R, Kim B, Chao C, et al. Physician survey of the effect of the 21-gene recurrence score assay results on treatment recommendations for patients with lymph node-positive, estrogen receptor-positive breast cancer. J Oncol Pract 2011;7:94-9.
28. Oratz R, Paul D, Cohn AL, Sedlacek SM. Impact of a commercial reference laboratory test recurrence score on decision making in early-stage breast cancer. J Oncol Pract 2007;3:182-6.
29. Paik S, Shak S, Tang G, et al. A multigene assay to predict recurrence of tamoxifen-treated, node-negative breast cancer. N Engl J Med 2004;351:2817-26.
30. Paik S, Tang G, Shak S, et al. Gene expression and benefit of chemotherapy in women with node-negative, estrogen receptor-positive breast cancer. J Clin Oncol 2006;24:3726-34.
31. Partin JF, Mamounas EP. Impact of the 21-gene recurrence score assay compared with standard clinicopathologic guidelines in adjuvant therapy selection for node-negative, estrogen receptor-positive breast cancer. Ann Surg Oncol 2011;18:3399-406.
32. Thanasoulis T, Brown A, Frazier T. The role of Oncotype DX assay on appropriate treatment for estrogen positive, lymph node negative invasive breast cancer. In: American Society of Breast Surgeons Annual Meeting. New York, NY; 2008.
Validity Assessment of Oncotype DX breast cancer assay economic analyses
Economic validity refers to the completeness, quality, and reliability of the analyses used to assess the economic implications of novel technologies. In 2003, Weinstein et al. outlined the criteria that should be considered when evaluating economic analysis.6 Four criteria have been established for systematically evaluating the quality and validity of economic evaluations, details of which are found in the appendix.
Six papers and one letter have been published in peer-reviewed journals on the potential economic implications of the Oncotype DX® breast cancer assay in four countries (Table 1). Furthermore, six additional studies, representing five countries, have been presented as abstracts. In total, the implications of the Oncotype DX breast cancer assay have been evaluated in eight different countries.
Table 1. Summary of Health Economic Evidence
Economic Implications of Oncotype DX® in the United States
In the first economic analysis of the Oncotype DX breast cancer assay in node-negative, ER-positive invasive breast cancer patients receiving tamoxifen, Hornberger et al. performed cost-utility analyses using a decision analytic model.7Using a Markov model, they forecasted overall survival, costs, and cost-effectiveness of using the Oncotype DX breast cancer assay results (Recurrence Score) in patients classified as having low or high risk of distant recurrence based on 2004 National Comprehensive Cancer Network® (NCCN®) clinical guidelines. Data from a large multicenter clinical trial (NSABP B-14) were analyzed to derive risk classifications based on guideline criteria and the Oncotype DX breast cancer assay. The effect of adjuvant chemotherapy (CT) on distant recurrence-free survival (DRFS) was based on published meta-analyses of CT trials. The analysis took a societal perspective, considering survival, quality of life, and relevant costs. Results showed fifty-three patients (8%) were classified as having low risk of distant recurrence by NCCN guidelines and the Oncotype DX breast cancer assay reclassified 15 of these patients (28%) to the intermediate or high risk groups. The remaining 615 patients (92%) were classified as having high risk of distant recurrence by NCCN guidelines and the Oncotype DX breast cancer assay reclassified 300 of these patients (49%) to low risk. Among a hypothetical cohort of 100 patients with node-negative, ER-positive early-stage invasive breast cancer (7.9% of whom are NCCN-classified as low risk for distant recurrence), use of the Oncotype DX breast cancer assay was expected to reclassify two patients from low risk to intermediate or high risk and 45 patients from high risk to low risk. Assuming patients with intermediate or high risk receive chemotherapy and patients at low risk do not, the Oncotype DX breast cancer assay was predicted to, on average, increase quality-adjusted survival by 8.6 years and reduce overall costs by $202 828 (Figure 3).
Figure 3. Cost effectiveness of the Oncotype DX breast cancer assay applied to cohort of 100 patients with node-negative, ER-positive early-stage invasive breast cancer
From Hornberger et al. Am J Manag Care 2005.7
From Hornberger et al. Am J Manag Care 2005.7
The Oncotype DX breast cancer assay was cost saving in more than two-thirds of probabilistic simulations, with cost-effectiveness most influenced by the propensity to administer CT based on the Oncotype DX breast cancer assay results, and by the proportion of patients at low risk as defined by NCCN guidelines. Researchers concluded that the Oncotype DX breast cancer assay predicts recurrence risk in node-negative, ER-positive patients with early-stage invasive breast cancer more accurately than current guidelines.20 If applied appropriately, the assay is predicted to increase quality-adjusted survival and save costs.
In a 2007 economic analysis of the cost-effectiveness of the Oncotype DX breast cancer assay, Lyman et al. assessed the efficacy of therapy guided by the Oncotype DX breast cancer assay results (Recurrence Score).8 Using data from NSABP studies B-14 and B-20, patients were classified as high (RS ≥31), intermediate (RS 18-30), or low (RS < 18) risk for distant recurrence at 10 years. Cost-effectiveness ratios were estimated for therapy guided by the Oncotype DX breast cancer assay compared with either tamoxifen alone or combined chemotherapy and tamoxifen. Therapy guided by the Oncotype DX breast cancer assay was associated with a gain in individual life expectancy of 2.2 years compared with tamoxifen alone, and with similar life expectancy to that seen with combined chemotherapy and tamoxifen. Therapy guided by the Oncotype DX breast cancer assay was estimated to provide a net cost savings of $2,256 compared with chemotherapy and tamoxifen with an incremental cost-effectiveness ratio of $1,944 per life year saved compared with tamoxifen alone (Figure 4).
Figure 4. Cost-effictiveness of Oncotype DX® breast cancer assay
From Lyman et al. Cancer 2007.8
From Lyman et al. Cancer 2007.8
Researchers concluded that for patients with lymph node-negative, estrogen receptor-positive early-stage invasive breast cancer, treatment decisions based on therapy guided by the Oncotype DX breast cancer assay is associated with greater efficacy and acceptable cost-effectiveness ratios compared with tamoxifen alone. Compared with combined chemotherapy and tamoxifen, therapy guided by the Oncotype DX breast cancer assay is associated with similar efficacy and lower cost.
In 2010, Lo et al. at Loyola University Medical Center published the first prospective study on the decision impact of the Oncotype DX breast cancer assay.21 It inquired about reanalysis of the economic implications of the Oncotype DX breast cancer assay with their data. In response, Hornberger et al. applied the data reported by Lo et al. in the previously published decision analytic model and found a saving of more than $300 per patient tested.9
Hornberger et al. conducted a subsequent study in collaboration with Humana Inc., a large United States health insurance company, to examine the economic implications of adopting the Oncotype DX breast cancer assay within its health plan.15 Using published decision impact study of the Oncotype DX breast cancer assay and cost data obtained from retrospective claims analyses of 952 invasive breast cancer patients who were tested with the Oncotype DX breast cancer assay, the study found that, for Humana, the adoption of the Oncotype DX breast cancer assay was cost-saving (Figure 5).
Figure 5. Cost-effectiveness of Oncotype DX® breast cancer assay adoption to Humana, Inc.
From Hornberger et al. Am J Manag Care 2011.15
From Hornberger et al. Am J Manag Care 2011.15
To view the Hornberger et al. 2005 article please visit:http://www.ajmc.com/files/articlefiles/AJMC05MayHornbergr313to.pdf
To view the Lyman et al. 2007 article please visit: http://www3.interscience.wiley.com/cgi-bin/abstract/114124513/ABSTRACT
To view the Hornberger et al. 2011 article please visit: http://jop.ascopubs.org/content/7/3S/e38s.abstract?sid=7c228324-299f-44ff-bfb5-f15726ddfd19
National Comprehensive Cancer Network® and NCCN® are registered trademarks of NCCN, which does not endorse any product or therapy.Economic implications of the Oncotype DX® breast cancer assay outside the United States
The economic implications of the Oncotype DX breast cancer assay have been evaluated in seven countries outside the United States (Table 2).
Table 2. Summary of health economic evidence outside the United States
References
6. Weinstein MC, O'Brien B, Hornberger J, et al. Principles of good practice for decision analytic modeling in health-care evaluation: report of the ISPOR Task Force on Good Research Practices--Modeling Studies. Value Health 2003;6:9-17.
7. Hornberger J, Cosler LE, Lyman GH. Economic analysis of targeting chemotherapy using a 21-gene RT-PCR assay in lymph-node-negative, estrogen-receptor-positive, early-stage breast cancer. Am J Manag Care 2005;11:313-24.
8. Lyman GH, Cosler LE, Kuderer NM, Hornberger J. Impact of a 21-gene RT-PCR assay on treatment decisions in early-stage breast cancer: an economic analysis based on prognostic and predictive validation studies. Cancer 2007;109:1011-8.
9. Hornberger J, Lyman GH, Chien R. Economic implications of 21-gene recurrence score assay: US multicenter experience. J Clin Oncol 2010;28:e382; author reply e3.
15. Hornberger J, Chien R, Krebs K, Hochheiser L. US insurance program’s experience with a multigene assay for early-stage breast cancer. Am J Manag Care 2011;17:e194-202.
20. Goldstein LJ, Gray R, Badve S, et al. Prognostic Utility of the 21-Gene Assay in Hormone Receptor-Positive Operable Breast Cancer Compared With Classical Clinicopathologic Features. J Clin Oncol 2008.
21. Lo SS, Mumby PB, Norton J, et al. Prospective multicenter study of the impact of the 21-gene recurrence score assay on medical oncologist and patient adjuvant breast cancer treatment selection. J Clin Oncol 2010;28:1671-6.
The Oncotype DX Breast Cancer Assay for DCIS Patients
The Oncotype DX Breast Cancer Assay for DCIS is a multi-gene diagnostic assay designed to support personalized treatment planning for patients with DCIS following local excision. The assay provides an individualized estimate of the 10-year risk of local recurrence (DCIS or invasive carcinoma) to help guide treatment decision making in women with ductal carcinoma in situ treated by local excision, with or without tamoxifen.
The Oncotype DX Breast Cancer Assay is performed in the licensed Genomic Health laboratory where the assay was developed. All H&E's are reviewed by board certified surgical pathologists and the DCIS tumor is marked for manual microdissection. The dissected, enriched tumor sample then undergoes RNA extraction. Next, the RNA is analyzed using a technique called real-time RT-PCR (reverse transcriptase-polymerase chain reaction). Finally, the DCIS Score™ result is calculated from the gene expression results.
Advantages of RT-PCR
The Oncotype DX Breast Cancer Assay analyzes the expression of a panel of 21 genes from a tumor specimen using a technique called RT-PCR. A high-throughput, real-time RT-PCR method was developed to analyze the expression of select genes. Unlike routine histopathologic H&E slide review or assessment of immunohistochemical stains for hormone receptors, RT-PCR is sensitive, specific, highly reproducible, and has a wide dynamic range. RT-PCR is a mature technology that is routinely utilized in several clinical applications including viral load testing for HIV.
To quantify gene expression, RNA is extracted from formalin-fixed, paraffin-embedded (FPET) tumor tissue and subjected to DNase I treatment. Total RNA content is measured and the absence of DNA contamination is verified. Reverse transcription is performed and is followed by quantitative TaqMan® (Roche Molecular Systems, Inc.) RT-PCR reactions in 384-well plates. The expression of each of 16 cancer related genes is measured in triplicate and then normalized relative to a set of five reference genes.
The Oncotype DX Breast Cancer Assay for DCIS patients standardized testing methods have been optimized to minimize variability due to:
- Sources of pre-analytic variability: delay to fixation, choice of fixative, duration of fixation, age of sample
Oncotype DX Assay Development
The Oncotype DX Breast Cancer Assay for DCIS patients was developed in four steps. Each of these steps is described in detail below.
- Optimization of methods for quantifying gene expression in formalin-fixed, paraffin-embedded tissue (FPET)
- Selection of 250 candidate genes from the human genome
- Testing of candidate genes to identify an optimal gene panel for clinical validation
- Prospective clinical validation of the gene panel and DCIS Score result calculation
Step 1. Optimization of methods for quantifying gene expression in formalin-fixed, paraffin-embedded tissue
The ability to work with FPET samples is critical in the U.S., as this is the standard method for tumor preservation and storage. When tissue is preserved in paraffin, the RNA is fragmented. However, the relative ratio of RNA between genes is unchanged. By utilizing RT-PCR techniques, the expression of most genes—relative to a set of reference genes—can be measured. To develop the Oncotype DX Breast Cancer Assay, Genomic Health researchers optimized RT-PCR technology 1) for high-throughput, real-time quantitation of specific RNA in FPET, and 2) to be reproducible regardless of the variability inherent in tumor blocks.
Step 2. Selection of 250 candidate genes from the human genome
Genomic Health researchers relied on numerous sources to identify 250 candidate genes—those possibly associated with breast cancer tumor behavior—from among the approximately 25,000 genes in the human genome.
Step 3. Testing of candidate genes to identify an optimal gene panel for clinical validation
The 250 candidate genes were analyzed in a total of 447 patients from three independent clinical studies in order to identify a panel of genes strongly correlated with distant recurrence-free survival. The selection of the 16 cancer genes used for the Oncotype DX Breast Cancer Assay was based on the results of the three clinical trials, which demonstrated a consistent and strong statistical link between these genes and distant breast cancer recurrence. Five reference genes were identified to normalize the expression of these cancer-related genes. The 21- gene panel described above has been optimized for the DCIS Score, which is calculated from a subset of the genes using a DCIS-specific algorithm and coefficients.
Step 4. Prospective clinical validation of the DCIS Score
The Oncotype DX Breast Cancer Assay gene panel and DCIS Score result calculation were validated in a large, independent, multicenter clinical trial (ECOG E5194). The endpoints and analysis plan were prospectively defined. The results of this study were presented at the San Antonio Breast Conference 2011 and the results of the ECOG E5194 clinical validation study will be published in the near future.
21-Gene Panel Used to Calculate Recurrence Score® Result
The Oncotype DX Breast Cancer Assay gene panel was selected from the published literature, a genomics database and experiments based on DNA arrays performed on fresh-frozen tissue. The DCIS Score is obtained by performing the Oncotype DX Breast Cancer Assay, using a distinct DCIS algorithm and coefficients that was pre-specified because of its ability to predict recurrence in patients with DCIS regardless of whether adjuvant tamoxifen therapy was given.
Development of the DCIS Score algorithm was based on published results for the Oncotype DX Breast Cancer Assay showing similarity in the expression profiles of the Recurrence Score genes between DCIS and Invasive Breast Cancer (IBC) when both are present within the same patient tumor. The DCIS Score algorithm was developed based on published data obtained from the Kaiser Permanente and NSABP B-14 studies in which the proliferation gene group, PR and GSTM1 were found to predict distant recurrence regardless of whether adjuvant tamoxifen therapy was given. This DCIS Score was subsequently validated as a predictor of local recurrence in patients from the ECOG E5194 study. These results were presented at the San Antonio Breast Conference 2011.
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