World R&D Market for UPS Research Expected to Top $5.5 Billion by 2018
By Mia Burns
In five years, BCC Research expects the global ubiquitin proteasome R&D market to surpass $5.5 billion, according to a new report. As a publisher of technology market research reports based in Wellesley, Mass., BCC Research also expects the market to register a five-year compound annual growth rate of 14.2 percent for the period 2013 to 2018. The value for the market this year was about $2.9 billion.
Ubiquitin proteasome system represents one of the most studied and challenging areas in basic and applicable science, according to BCC Research. The scientists who discovered this cellular phenomenon were awarded the Nobel Prize in Chemistry in 2004. Since then, ubiquitin ligases and proteasome function have become one of the major targets for pharmaceutical and drug development companies. However, despite the accumulation of basic knowledge about the ubiquitination process and proteasome function, progress in drug screening and therapeutic development has been delayed. Until 2012, there was only one approved product on the market, Millennium Pharmaceuticals’ Velcade, a proteasome inhibitor.
Velcade is a therapeutic treatment for patients with multiple myeloma, a type of bone marrow cancer that can damage the immune system. According to BCC Research, the global market value of Velcade was more than $2 billion in 2012. The market is expected to reach $2.6 billion by 2013 and $4.5 billion by 2018, registering a CAGR of 11.4 percent for the period 2013 to 2018.
Onyx Pharmaceuticals markets the new second-generation proteasome inhibitor on Kyprolis, an immunomodulatory. FDA approved Kyprolis last year to treat patients who suffer from multiple myeloma and have received at least two prior therapies, including treatment with Velcade. Many analysts predict that Kyprolis sales will increase dramatically in the next five years, depending on further applications and regulatory approvals. According to BCC Research, the global market value of Kyprolis was $62 million in 2012. This market is expected to reach $260 million by 2013 and further increase to $1 billion by 2018, to register a CAGR of 30.9 percent for the period 2013 to 2018.
BCC Research market analyst Marianna Tcherpakov does not anticipate that Kyprolis will pose a dire threat to the Velcade market share at this time. “Kiprolis is approved as second/third line treatment after Velcade has failed and when patient’s condition is worth and not improving,” she told Med Ad News Daily. “Although it is a promising drug, more clinical trials are needed to prove that Kiprolis indeed improves patient’s survival. For now, there is not enough data to consider this drug a threat to Velcade. Nevertheless, Kiprolis was accepted in a positive light by analysts and medical community. If clinical data will show better survival and response to this drug, it can be approved in the future as alternative to Velcade and other treatments like Revlimid. For now, this is far away from happening and may take a number of years for this scenario. Also, it is hard to beat Velcade, since the drug was proven itself in the last couple of years as preferential choice. For now, Kiprolis revenues will come as second choice alternative when other drugs failed and can still generate billions in revenues.”
Regarding the delay in progress for drug screening and therapeutic development for the ubiquitination process and proteasome function, Tcherpakov outlines several factors, including the complexity of the biological system. “Ubiquitination process requires at least three types of enzymes,” she told Med Ad News Daily. “Some of the enzymes like E1 are very conserved, which makes the drugs screening process problematic since compound may not be selective enough against damaged cells and can affect healthy tissues. On the other hand, E3 ligases are represented by hundreds of genes and some of them require specific factors which may not be known yet and more research is required. Proteasome is also complex structure so regulation and function of this machinery is still not fully understood. More importantly, successful drug screening requires efficient and reliable assay development which is not optimal yet for ubiqutination reaction, therefore making it very hard to find good drug candidates.”
The actual commercial potential of this research area has yet to be fully acknowledged. “Overall, success of Velcade and Kiprolis already proved how profitable this area can be, even when the drugs are not ‘prefect’”, Tcherpakov says. “Further success story, meaning approval of other ubiquitination related drug candidate such as inhibitor against E3 ligase or deubiquitinase can encourage pharma and drug discovery industry to invest more in this area. For now, there are no drugs approved against ubiquitination enzymes or deubiquitinases.”
In addition, other big pharmas have different R&D projects that focus on drug candidates in ubiquitination process, Tcherpakov told Med Ad News Daily. “For example, Genentech (Roche) is known for its scientific contribution in research of different E3 ligases, Celgene is working on compounds against Cereblon, Roche and J&J have programs on Mdm2/p53 drug candidates,” she says. “There are smaller companies that make contribution for ubiquitin sector drug development: Proteologics from Israel or Progenra. There are some new players in this area like Cleave Biosciences or Resolve Therapeutics that may have interesting data in the future.”
For now, the sector is in a steady state, according to Tcherpakov. “There are number of projects in development, but except for Kiporlis, no major breakthrough were reported in the last couple of years,” she says. “Deubiquitinases are becoming one of the potential trends since assay development in this sector is easier for screening for drug candidates. Indeed, there are new companies like Cleave Biosciences, Proteostasis Therapeutics, or Mission Therapeutics along with older companies like Progenra that focus on deubiquitinases. It seems that it may take at least five years till industry will see new approved drug against ubiquitination or deubiquitination enzyme on the market. But scientific or even clinical breakthroughs may come earlier, which gives hope for further drug development in this sector. For example, if E1 inhibitor from Millennium Pharmaceuticals will be approved, it will definitely boost this sector for further investment in drug development.”
Posted: August 2013