Dealmaking and Industry Outlook in Niche Metabolic Diseases
Decision Resources, Inc, August 2010, Pages: 49
Biologics will enjoy 12 years of marketing exclusivity under the newly enacted regulatory pathway for biosimilars in the United States, and opportunities for developing and marketing biobetters and enzyme replacement therapies (ERTs; biologics based on therapeutic recombinant enzymes) are great in metabolic disease markets.
In this report, we look beyond the dominant and well-documented diabetes sector of the metabolic marketplace to analyze opportunities in niche metabolic diseases. We chose lysosomal storage diseases because of their unmet need and existing opportunities for developing and marketing more ERTs. We assessed growth-hormonerelated diseases because they represent additional opportunities for developing biosimilars and biobetters.
The report addresses several sectors of the metabolic disease therapy area, the brand drugs used to treat these diseases, and certain R&D pipelines. We also provide insights from interviews with the CEOs of Astellas Venture Management and Takeda Research Investment, and we examine recent dealmaking activities by pharmaceutical companies and certain corporate venture capital firms in metabolism.
Questions Answered in this Report:
- More than 6,000 rare diseases (defined as a disease that affects fewer than 200,000 people) have been identified that affect a total of 25 million people in the United States alone. What rare diseases exist in the metabolic disease therapy area? What major brands are marketed to treat some of these diseases? What treatment options are in development for these diseases? What manufacturing problems has Genzyme experienced with its enzyme replacement therapies?
- Human growth hormones were among the earliest biosimilars on the market. Why is Novartis more strongly committed to its generics subsidiary Sandoz today than in the past? What are the major marketed growth hormone products? What is the status of growth hormone biosimilars and biobetters? Why will the human growth hormone biosimilar market be slow to develop?
- GlaxoSmithKline’s CEO Andrew Witty told investors that the cost of achieving success in the company’s neuroscience programs was disproportionately high. Why did GlaxoSmithKline disinvest in its neuroscience programs for pain, anxiety, and depression? Why did GlaxoSmithKline establish a new rare disease unit? What recent deals form the basis of this new endeavor? Why does GlaxoSmithKline feel that the risk associated with product discovery and development in rare diseases is generally lower than in other disease areas?
- Astellas Pharma’s senior director for licensing and alliances, Yoshitaka Yoneyama, says that Astellas Venture Management (AVM) promotes direct dialogue between portfolio companies and company headquarters. What are the strategies of corporate venture capital (CVC) groups? Why do CVC groups dislike “pay to play” deals? In which metabolism companies have AVM and Takeda Research Investment invested? What perceptions do CVCs have about metabolic disease drug discovery and development?
- Teva North America CEO Bill Marth says that his company never saw the new U.S. biosimilars pathway as the only way to compete when biologics lose patent protection and that Teva is going the biologics license application (BLA) route for many biologics. Why does Teva feel that the U.S. Congress dropped the ball on biosimilars legislation? Why is Teva planning to sidestep the new U.S. biosimilars pathway? Why is drug development of orphan diseases such as lysosomal storage diseases uniquely suited to take advantage of the 12-year market exclusivity afforded to original biologics in the United States?
Scope:
- Primary research: Interviews with the CEO of Astellas Venture Management, the CEO of Takeda Research Investment, and Astellas Phama’s senior director for licensing and alliances. Decision Resources analysts’ market forecasts of therapies for metabolic diseases, lysosomal storage diseases, and growth-hormone-related diseases.
- Diseases: Metabolic disease, lysosomal storage disorders, mucopolysaccharidoses, Hurler-Scheie disorder, Hunter’s syndrome, Morquio’s syndrome, Sanfi lippo’s syndrome, Sly’s syndrome, Maroteaux-Lamy syndrome, sphingolipidoses, metachromatic leukodystrophy, Tay-Sachs disease, Krabbe’s disease, Wolman’s disease, cholesteryl ester storage disease, Gaucher’s disease, Fabry’s disease, Niemann-Pick disease, glycogen storage disorders, Pompe’s disease, von Gierke’s disease, Andersen’s disease, McArdle’s disease, Cori’s disease, Hers’ disease, Tarui’s disease, Fanconi-
- Bickel’s syndrome, mucolipidoses, cherry-red-spot-myoclonus syndrome, inclusion-cell disease, growth-hormone deficiency, idiopathic short stature, small for gestational age (SGA), acromegaly, AIDS-associated wasting and cachexia, growth failure from chronic renal insuffi ciency (CRI), Noonan’s syndrome, Prader-Willi’s syndrome, short stature homeobox (SHOX) defi ciency, Turner’s syndrome, obesity, type 1 and type 2 diabetes.
- Therapies in development: Enzyme replacement therapy (ERT); substrate reduction therapy (SRT); RNA-targeting drugs; stop codon read through therapy; gene replacement therapy; pharmacological chaperone therapy (PCT); hematopoietic stem cell transplantation (HSCT); enzyme enhancement therapy (EET); gene mutations; disease-causing enzymes; major brands for lysosomal storage disorders, type 1 and type 2 diabetes, and obesity; R&D pipelines for mucopolysaccharidoses, sphingolipidoses, and glycogen storage disorders; international classifi cation codes for endocrine, nutritional, and metabolic diseases; Genzyme’s ERT manufacturing problems, gastrointestinal permeation enhancement technology (GIPET), xenotransplantation, cell regeneration.
- Dealmaking and strategies: Recent metabolic disease deals for 13 companies, new focus on rare diseases, corporate venture capital strategies and deals, biologics strategies, biosimilars, biobetters, biologic market exclusivity opportunities, premium pricing of orphan disease therapies.
Special features of this report include . . .
- Interviews with the CEOs of Astellas Venture Management and Takeda Research Investment
- New rare disease strategies by Big Pharma.
- Deficient genes and defi cient enzymes for lysosomal storage disorders.
- R&D pipelines for mucopolysaccharidoses, sphingolipidoses, and glycogen storage disorders.
- Major brands for lysosomal storage disorders, type 1 and type 2 diabetes, and obesity.
- An appendix of international classifi cation codes for endocrine, nutritional, and metabolic diseases.
Key Terms for this Report: Metabolic, lysosomal storage, mucopolysaccharidosis, sphingolipidosis, glycogen storage, mucolipidosis, growth hormone, corporate venture capital, inlicensing, biologic, biosimilar, market exclusivity, premium pricing, enzyme replacement therapy, substrate reduction therapy.
Executive Summary
- Strategic Considerations
- Stakeholder Implications
Metabolic Disease Market
Lysosomal Storage Disorders–Opportunities for More Enzyme Replacement Therapies
- Major Players and Brands
- Treatment Options in Development for Lysosomal Storage Disorders
- Mucopolysaccharoses–Diseases, Enzymes, Genes, and Pipelines
- Sphingolipidoses (Lipidoses)–Diseases, Enzymes, Genes, and Pipelines
- Glycogen Storage Diseases (Glycogenoses)–Diseases, Enzymes, Genes, and Pipelines
- Mucolipidoses–Diseases, Enzymes, Genes, and Pipelines
- Issues Relating to the Manufacture of Recombinant Enyzmes
Growth-Hormone-Related Disorders–Opportunities for Biosimilars and Biobetters
- Diseases
- Major Brands and Players
- Biobetters
- Biosimilar Human Growth Hormones
Metabolic Disease Dealmaking–Who’s Buying What?
- Amgen
- AstraZeneca
- Boehringer Mannheim
- GlaxoSmithKline
- Ipsen
- Eli Lilly
- Merck & Co.
- Novo Nordisk
- Pfizer
- Recordati
- Roche
- Sanofi -Aventis
- Takeda Pharmaceutical
Corporate Venture Capital Currently Not Big on Metabolic Diseases
- Astellas Venture Management
- NVS Strategic Fund
- Takeda Research Investment
Outlook and Market Drivers
- Evolving Focus on Rare Diseases
- Opportunity Too Good to Refuse
Appendix
- 5AM Ventures
- Abbott Laboratories
- Actelion Pharmaceuticals
- AgaMatrix
- Alantos
- Aldagen
- Alexion
- Alizé Pharma
- Alphapharm
- Ambrx
- Amgen
- Amicus Therapeutics
- Amylin Pharmaceuticals
- ArmaGen Technologies
- Array BioPharma
- Astellas Pharma
- Astellas Venture Management (AVM)
- AstraZeneca
- Aurobindo Pharma
- Bayer
- Bayhill Therapeutics
- BioDecade
- BioMarin Pharmaceutical
- BioPartners
- Biovitrium
- Boehringer Mannheim
- Bristol-Myers Squibb
- Celltrion
- Center for Drug Evaluation and Research (CDER)
- Chinese Diabetes Society
- Cowen and Company
- CureDM
- Daiichi Sankyo
- Dainippon Sumitomo Pharma
- Depomed
- DeveloGen
- Dicerna Pharmaceuticals
- Eli Lilly
- Elixir Pharmaceuticals
- Envoy Therapeutics
- Enzon Pharmaceuticals
- ExSAR
- FASgen
- Galapagos
- Generex Biotechnology
- Genzyme
- GlaxoSmithKline
- GlyGenix Therapeutics
- Hadassah Medical Organization
- Harvard University
- Hebrew University of Jerusalem
- Helix Therapeutics
- Hospira
- Ilypsa
- Increlex
- Interpharm Consultancy
- Invida Group
- Isis Pharmaceuticals
- Ispen
- JCR Pharmaceuticals
- Johnson & Johnson
- Juvenile Diabetes Research Foundation
- Karo Bio
- LG Life Sciences
- Lonza
- MacroGenics
- Mannkind
- Massachusetts General Hospital
- Medingo
- Medtronic
- Merck & Co.
- Merck KGaA
- Merrion Pharmaceuticals
- Metabolex
- Mylan
- National Health Service (NHS)
- National Institute for Health and Clinical Excellence (NICE)
- National MPS Society
- Novartis
- Novo Nordisk
- NVS Strategic Fund
- Offi ce of Orphan Products Development (FDA)
- Ono Pharmaceutical
- Orphan Europe
- PDL BioPharma
- Pfizer
- Plexxikon
- PPL Therapeutics
- Prolor Biotech (formerly Modigene)
- Prosensa
- Protalix Biotherapeutics
- QOL Medical
- Ranbaxy Laboratories
- Rare Disease Therapeutics
- Recordati
- ReGenX Biosciences
- Revivicor
- Rhythm Pharmaceuticals
- Roche
- Roche Venture Fund
- Ryan Foundation for MPS Children
- Sandoz
- Sanofi -Aventis
- Servier
- Shire
- Shire Human Genetic Therapies
- Sigma-Tau Pharmaceuticals
- Synageva BioPharma
- Takeda Pharmaceutical
- Takeda Research Investment (TRI)
- Takeda San Francisco
- Teijin
- Tekmira Pharmaceuticals
- Tercica
- Teva North America
- Teva Pharmaceutical Industries
- Theratechnologies
- Tianjin Lisheng Pharmaceutical
- Tianjin Takeda Pharmaceuticals
- Torrent Pharmaceuticals
- Transition Therapeutics
- U.S. Department of Veterans Affairs
- University of Florida
- University of Michigan
- Vivendy Therapeutics
- Watson Pharmaceuticals
- Wellstat Therapeutics
- Yissum Research and Development
- Zymenex
- ZyStor Therapeutics
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