News announcing that small biotech firms are filing for approval of breakthrough drugs targeting deadly diseases is worth admiration, not cynicism. Humans look for miracles; yet, they impassively overlook hundreds of human handmade marvels taking place every second around them. Serendipity does not make miracles. Human curiosity, learning and determination to erase whatever obstacles might block the road to great accomplishments create marvels. Discovering the structure of the DNA is a miracle, mapping the human genome is a miracle, switching genes on and off with a pill is working on a miracle and the advancement in biological sciences in the past two decades is a basket full of miracles. The miracle of miracles, though, was that in spite of labeling the biotechnology industry high risk since its inception, investors never abandoned investing in it. Thousands generated lots of profit on it, especially those who managed to select the best of the best of the stocks and stick to them. Those investors, no doubt, contributed a great deal in creating many of the miracles that are filling what a quarter of a century ago was an empty basket.  More...

The time has come for redefining the meaning of “creating shareholders value” in the biotech sector. Pinpointing the real value has become imperative. It is the road to a fair assessment of innovative biotech firms. It helps investors reap the fruits of the real value of excellent firms at the right time, rather than rush to sell extremely promising stocks while still undervalued. It makes investors think twice before throwing their shares of firms that are installing one by one the building blocks of their vision. Creating shareholders value is investing in the future. The cost of this investment shouldn’t be cause for downgrading the stocks because of overspending, which has become a normal pattern. We still see all development-stage biotech stock prices slashed following the announcement of their quarterly results. There is a big difference between spending on the future and spending because the money has been made available. Investors can know the difference. The first thing to look for is whether the firm has any program in development that is worth spending on, including buying firms having a technology that is badly required for the successful outcome of the firm’s program. This kind of investment should not be punished or treated as unnecessary spending. More...

On Monday May 7, 2012, Vertex announced an interim analysis of data from an ongoing Phase 2 study of VX-809 in combination with KALYDECO™ (ivacaftor). The analysis demonstrated significant improvements in lung function (FEV₁) among adults with cystic fibrosis (CF) who have two copies (homozygous) of the most common mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, F508del.   More...