I recently returned from a few days in Boston & Cambridge, so today, in memory of the late Alastair Cooke and his Letter from America, broadcast for 58 years from 1946 to 2004, I wanted to share with you my “Letter from Boston”.
New England is the No 1 biotechnology region on the East Coast of the United States and the Boston/Cambridge area of Massachusetts is the hub.
What makes Boston/Cambridge so attractive as a biotech region? Amongst many, I’d suggest 3 factors stand out to me:
- Access to World-Class Science with an Entrepreneurial Focus. With over 50,000 students in the Boston/Cambridge area it is a city with a focus on higher education. Harvard, MIT, Boston University, Northeastern, Tufts, Massachusetts General Hospital are but a few of the many research institutions. However, what strikes me about the researchers in Boston/Cambridge area is the entrepreneurial focus they have. The idea of starting up a company, commercializing an innovation or finding the application of science is something a lot of people want to do. This entrepreneurial focus is key to the success of industry/academic colloboration in the area.
- Critical Mass of Industry infrastructure. There’s a range of companies in the Boston/Cambridge area. From start-ups such as Blueprint Medicines to more established companies such as Ariad, Vertex and Millennium-Takeda, what Boston/Cambridge offers is a critical mass of talent and people. Those working in the area have sufficient opportunities to move to new companies and positions, that it’s not a major career risk to move to the area. There’s also a lot of early stage infrastructure such as the Novartis Institute of Biomedical Research that bridges the gap between basic research and early stage commercial development.
- Geographic Location. Finally, what stands out for me is the excellent location that Boston has. You can easily reach New York’s investors and analysts, Washington Policy Makers or New Jersey big pharma without too much difficulty. At the same time, Boston is easily accessible for European companies, and the travel time to London can be less than going to the West Coast.
Pfizer recently announced further R&D investment in the Longwood Medical area, Harvard are building a new science campus in Allston and Vertex recently broke ground on a new headquarters in the South Boston innovation district.
For biotechnology companies at all stages of development there are a lot of opportunities in the Boston/Cambridge area.
Vertex today announced positive phase III clinical trial results for VX-770 in cystic fibrosis, a drug that I had previously written about.
I don’t see any point in rehashing the press release, but the data is good news for the company and Cystic Fibrosis patients.
As an update to this morning’s blog post that mentioned Vertex’s VX-770, the company have just announced their key business objectives for 2011. Further information will be included in the presentation by Vertex at the JP Morgan Healthcare conference scheduled for later today.
The news in Cystic Fibrosis is that if the phase 3 clinical trial data is positive the NDA for VX-770 is expected in the second half of 2011. The following are the relevant sections from the press release:
Cystic Fibrosis: Phase 3 Registration Program for VX-770 Nears Completion
VX-770 NDA Submission Planned for Second Half of 2011
- Three trials of the novel cystic fibrosis transmembrane conductance regulator protein (CFTR) potentiator VX-770 are fully enrolled and ongoing as part of a global Phase 3 registration program focused on patients with the G551D mutation. The G551D mutation is present in approximately four percent of people with CF.
- The first Phase 3 data for VX-770 are expected in the first quarter of 2011 and will come from the Phase 3 STRIVE trial in people aged 12 and older with at least one copy of the G551D mutation. Data from the Phase 2 DISCOVER trial, which was primarily a safety study that enrolled people aged 12 and older with two copies of the F508del mutation, are also expected in the first quarter of 2011.
- Data from the Phase 3 ENVISION trial in people aged six to 11 with at least one copy of the G551D mutation are expected in mid-2011.
- If positive, the results from the Phase 3 program for VX-770 could support the submission of an NDA for VX-770 in the second half of 2011.
In addition, Vertex announced that they expected interim data in the first half of 2011 from the phase 2 trial that combines VX-770 with VX-809:
Combination of Two CFTR Modulators for the Treatment of People with the Most Common Mutation of Cystic Fibrosis
- Vertex is conducting a Phase 2a clinical trial to evaluate multiple combination regimens of its lead CFTR Modulators – VX-770, a CFTR potentiator, and VX-809, a CFTR corrector – in people with the most common mutation of CF, known as F508del. Enrollment is ongoing in Part One of the trial, which is designed to evaluate VX-809 (200 mg), or placebo, dosed alone for 14 days and in combination with VX-770 (150 mg or 250 mg), or placebo, for 7 days. Vertex expects to obtain interim data from Part One of the trial in the first half of 2011.
2011 looks to be an interesting year for Cystic Fibrosis and it is certainly positive to see biotechnology companies such as Vertex developing new products for this debilitating illness.
One of the exciting things about the biotechnology industry is its ability to innovate and translate developments in basic science into potential new drugs.
I previously wrote about denufosol in cystic fibrosis (CF), a disease that affects about 30,000 people in the United States and 70,000 worldwide. The disease is characterized by the accumulation of mucus that leads to bacterial overgrowth and chronic lung infections. Mucus cannot be removed from the lung in CF due to abnormal mucociliary transport resulting from impaired epithelial chloride secretion and sodium hyperabsorption. This is now known to be due to defective cystic fibrosis transmembrane regulator (CFTR) protein. A good overview of this can be found in the 2006 New England Journal of Medicine Editorial by Felix Ratjen, “Restoring Airway Surface Liquid in Cystic Fibrosis.”
A good overview of the pipeline of new drugs in development for CF can be found on the Cystic Fibrosis Foundation web site. Vertex in particular has two drugs (VX-809, VX-770) in late stage development that are cystic fibrosis transmembrane conductance regulators, aimed at increasing CFTR function. Phase 3 registration data for VX-770 is expected in the first half of 2011. I look forward to writing about the results.
Recently, a team from Johns Hopkins led by Neeraj Vij published a paper in the January 2011 issue of Journal of Immunology on the “Critical Modifier Role of Membrane-Cystic Fibrosis Transmembrane Conductance Regulator-Dependent Ceramide Signaling in Lung Injury & Emphysema.”
The researchers found that lung damage in mice was associated with changes in the amount of CFTR in the cell surface membrane. Decreases in the amount of CFTR were associated with increased ceramide, a trigger of inflammation of cell-death. Or as the the paper describes it:
“CFTR expression inversely correlates with severity of emphysema and ceramide accumulation in chronic obstructive pulmonary disease subjects compared with control subjects.”
The emergence of inflammation as a key role in chronic disease was the subject of a previous blog post about diabetes, so is interesting to see another area where it is involved.
This basic research shows that developing drugs that target CFTR and mediate ceramide may have an important role to play in the treatment of emphysema, a chronic obstructive pulmonary disease (COPD) that affects 2 million Americans. Translational medicine that can take basic science and apply it to clinical practice is key to the long term success of the biotechnology industry.