Biotech Strategy Blog

Commentary on Science, Innovation & New Products with a focus on Oncology, Hematology & Cancer Immunotherapy

Posts tagged ‘rheumatoid arthritis’

Tocilizumab approved for systemic juvenile rheumatoid arthritis in children

By on April 18, 2011

Juvenile rheumatoid arthritis (JRA) is a disease that results in chronic joint inflammation, and is the most common rheumatology disease in children.

The American College of Rheumatology classifies juvenile RA into 3 subtypes, one of which is systemic JRA.  The news that the FDA just approved tocilizumab (Actemra®) from Roche for pediatric systemic JRA (SJRA) is therefore good news for several reasons:

1. Very few drug companies obtain pediatric indications for their drug since registration trials are more routinely undertaken with adult patients.

2. In several adult phase III clinical trials, tocilizumab was also shown to delay joint damage as measured by ACR20, ACR50 and ACR70 responses.  An ACR 20 response requires a patient to have a 20% reduction in the number of swollen and tender joints.

If you look at the adult phase III clinical data that has been published for the AMBITION, LITHE, OPTION, TOWARD, RADIATE studies, what is noticeable to me is the high percentage of patients across the studies who had an ACR20 response within 24 weeks (70%, 56%, 59%, 61%, 50%); in all cases a significant improvement over placebo plus methatraxate or other disease modifying anti-arthritic drug (DMARD).

Source: Roche data on adult phase III trial results

However, it’s clear from the above data that fewer patients had deep responses e.g. ACR50, ACR70.  Tocilizumab is a treatment option for those who have failed previous RA therapies, and while not a cure, can provide symptom relief and improve joint function in those suffering from moderate to severe RA.

According to the Roche press release, data from the pediatric study known as TENDER, showed that 64/75 children (85%) of children with SJRA experienced an ACR30 improvement.  Given the fact the drug is already approved for adult use, and has extensive phase III trial data, a small pediatric sample size is not unexpected.

Summary:

A pediatric approval is good news, as all too often pharma and biotech companies neglect this market.  I plan to write more in future posts about RA drugs in development as I think this is a market that may continue to evolve new treatment options over the next few years.

AB Science – realizing the potential of masitinib in cancer and inflammation

Following on from yesterday’s news that Gilead had acquired Calistoga and CAL-101, another company that is exploring the interface between cancer and inflammation is Paris based AB Science.

Pharma Strategy Blog has an excellent interview with the CEO, Alain Moussy.  AB Science is an emerging French biopharmaceutical company, and I previously wrote about its IPO.

The company has adopted a unique market entry strategy of obtaining approval first in animal health for their tyrosine kinase inhibitor, masitinib.  In 2008, AB Science gained European approval for canine mast cell tumors and in December 2010 FDA approval.

The company recently announced that on February 8, 2011 it had its first US sale of masitinib to vets.

Masitinib is in fact a multi-kinase inhibitor that inhibits wild type and mutant forms of stem cell factor receptor (c-KIT, SCFR), platelet-derived growth factor (PDGFR), fibroblast growth factor 3 (FGFR3) and to a lesser degree, focal adhesion kinase (FAK).

Sally Church on the Pharma Strategy Blog has written about how AB Science’s strategy makes sense – if you look at Pfizer, they obtain more revenue from animal health than they do from oncology.  AB Sciences’ Masivet® in Europe, Kinavet® in the United States competes against Pfizer animal health’s tyrosine kinase inhibitor, Palladia® (toceranib), which also targets mast cell cancer in dogs.

Not only does this growth strategy generate revenue for an early-stage company like AB Science, it also allows the company to build a sales and marketing infrastructure in the United States and Europe while waiting for the results of pivotal phase 3 studies in humans.

The phase 2 clinical trial data for masitinib in combination with gemcitabine in pancreatic cancer were impressive (28% survival at 18 months).  The phase 3 clinical trial results are expected this year.  The clintrials.gov listing shows the date for the estimated primary completion date (Overall Survival) as November 2010 with study completion in November 2011.  Obviously the exact timing depends on how fast subjects were accrued, but I would be surprised if we didn’t see some data presented at ASCO or ESMO, especially if positive.

In terms of targeting inflammation, masitinib is in phase III development for mastocytosis, rheumatoid arthritis (RA) and asthma.  AB Science announced on January 27, 2011 the first patient recruited into their phase 3 study in severe asthma.

The company’s new product development strategy is way ahead of many of its competitors in identifying the links between cancer and inflammation, and choosing to target market opportunities in both areas.

AB Science is an exciting company to watch, and I expect that we will see important new data come out at major scientific meetings this year.

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