Vertex declines after announcing stock sale
BY MARLEY SEAMAN 09.18.08, 1:44 PM ET
Shares of Vertex Pharmaceuticals Inc. fell Thursday after the drug maker said it will sell 7.5 million shares of new stock to raise cash.
The Cambridge, Mass., company said late Wednesday that it would sell the stock, and early Thursday it priced the offering at $25.50 per share, which would allow it to raise $191.3 million. Vertex said it will use the funds to develop its late stage drug candidates for hepatitis C and cystic fibrosis, as well as for general corporate purposes.
Its shares have been trading slightly higher than that price, closing Wednesday at $26.57. In afternoon trading Thursday, Vertex shares gave up $1.37, or 5.2 percent, to $25.20 and fell as low as $24.62 earlier in the session.
The stock last traded at $25.50 on Sept. 10. Vertex had 140.6 million shares outstanding as of May 6, according to a filing with the Securities and Exchange Commission.
Cowen and Company analyst Rachel McMinn said the move was necessary, and will give Vertex financial flexibility in 2009.
"While the timing appears unusual in the midst of an economic meltdown, Vertex would have needed access to capital in the next 12 months to continue development of its lead pipeline product telaprevir, for hepatitis C, and its follow on product, VX-770 for cystic fibrosis," she said.
She said she thinks the company will need to raise more money before it becomes profitable, however.
Goldman Sachs (nyse: GS - news - people ) will manage the books for the stock offering.
jueves, 18 de septiembre de 2008
martes, 16 de septiembre de 2008
He tenido 2 hijos y tengo Fibrosis Quística
Dad with CF beats infertility, hits a double
Brad Hildebrandt was told he'd never have kids. Today, he has twins.
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By John Baiata
Senior Editor
NBC News
updated 9:48 a.m. ET Sept. 16, 2008
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The left-fielder charges the softball and cocks his arm to make the cut-off throw toward third base.
Then, seeing the runner has rounded second base by just a couple of steps, Brad Hildebrandt whipsaws his arm and fires a dart to the second baseman. Runner out. Rally killed. Inning over.
Perhaps the only thing sweeter than this play is the fact that his children are there to witness it. As Brad jogs off the field and accepts congratulatory high-fives from his teammates, he allows himself a slight smile. He has a lot to smile about.
As Brad’s teammate over the past decade, I've seen him purposely approach a ball too slowly in the outfield — goading the runner into trying to take an extra base — and then rifle a throw to erase him on the base path. He is probably the fastest runner on the team, and yet I don't recall ever seeing him visibly winded, even after rounding the bases at full speed.
But he hasn’t lost the ability to amaze me. When he recently confided in me that he has cystic fibrosis, I was stunned.
Cystic fibrosis, or CF, is an inherited, chronic disease that clogs the lungs and other internal organs with a thick mucus. Among other symptoms, CF patients suffer from frequent chest and sinus infections, problems with their digestive systems and poor growth. It affects some 30,000 people in this country, though an additional 10 million are carriers for the disease. When Brad was born, the median age of survival for CF patients was somewhere between 10 and 16 years of age.
Thanks to remarkable advancements in treatment, the median age in 2007 was 37.
Brad is 44.
"I was told I wouldn't live to see my 20s," Brad recalls. That sobering news was delivered to him when he was 14. "It was horrible. I started counting the days, the months, the years."
Three decades later, he's long past measuring his life out by the spoonful.
"I was also told there was a 99 percent chance I'd never have kids,” he says. “I'd been told that pretty much my whole life."
Today, he is happily married with two children, 9-year-old twins Bradley and Sadie.
Brad's double-header
That Brad and his wife, Pam, were able to conceive children at all is a lesson in perseverance, and the possibilities of modern medicine. They both wanted children, and had discussed it prior to marrying in 1997. The odds were not in their favor. About 95 percent of men who have CF are infertile due to an abnormality of the vas deferens, the vehicle by which sperm is carried from the testes to the urethra.
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Hildebrandt family photo Cystic fibrosis patient Brad Hildebrandt and his wife, Pam, conceived their twins, Sadie and Bradley, with the help of in vitro fertilization and a sperm-extraction technique. |
As Pam tells it: "We went to Brad's physician and told him we wanted our own biological children. We were told it wouldn't happen."
Undaunted, Pam began her own exhaustive research. She found that nearby Saint Barnabus Medical Center in Livingston, N.J., could extract the sperm using an aspiration technique and had one of the better success rates in the country with in vitro fertilization.
Before moving ahead, Pam decided to have herself screened for CF. Cystic fibrosis is caused by a recessive gene — meaning that two copies of the gene need be present for the disease to manifest itself. In other words, both mother and father must be carriers. If Pam was found to be a carrier, then their children would have a one-in-four chance of inheriting the disease.
Though Pam was found not to be a carrier, they learned that some mutations of the disease are not detected by the screening. They chose to go ahead with the procedures.
At the time, those procedures cost upwards of $15,000. None of it was covered by health insurance, and the Hildebrandts had to borrow the money from family members. "We figured if it didn't work the first time, we probably couldn't afford to do it again," says Brad, who works as an industrial designer. "Some of the couples we met were on their third or fourth try. There was no way we could afford that."
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The process only added to their anxieties. The sperm aspiration was a success, as was the retrieval of Pam's eggs. But aspirated sperm are not as mature, and therefore not as strong. And retrieved eggs quickly begin to die off. Ultimately, the insemination technique is a calculation of how many fertilized eggs will survive, if any. "It was nerve-racking," says Pam. "We had gone through this whole procedure and only three eggs made it through." Those three fertilized eggs were implanted, and the wait began.
An ultrasound soon revealed Pam was pregnant with twins.
The decision to have children is not one the Hildebrandts dwell on. Asked whether she had considered the fact that she could one day be a single parent raising her children, Pam responds, "Not really. I don't think about it. He's healthy. We live day to day."
Click for related content |
Dr. Bruce Marshall, vice president of clinical affairs with the Cystic Fibrosis Foundation in Bethesda, Md., says the foundation, whose work has been critical in advancing life expectancy for CF adults, does counsel adult patients on the decision to have children: "We remind them of the great rewards of child-rearing, but also remind them that they may not live to see their children full grown."
Watching the two Hildebrandt children play after games, it's clear they've inherited their father's athleticism. Bradley looks to be a baseball star in the making.
Thankfully, the inheritance stops there. Neither child has CF. Even that welcome news was prefaced by some apprehension: “When Sadie was about a year old,” recalls Pam “she started having a lot of colds, and we knew we had to get them tested for our own peace of mind.”
Nuevo Medicamento Genético para 2010
Este medicamento va al origen del problema, pero sólo funcionará para determinadas mutaciones genéticas.
| New wonder drug for genetic conditions could be on the market by 2010 |
| 16 September 2008 |
RESEARCHERS in the States hope a new drug which would revolutionise the treatment of genetic disorders such as muscular dystrophy and haemophilia will be available within two years.
Early trials into PTC Therapeutics’ PTC124 formula show the simple packet of powder can make cells less sensitive to the genetic mutations associated with around 2,400 health problems including cystic fibrosis (CF). Given early enough, there is even evidence the drug can halt the progress of some genetic illnesses, many of which are incurable.
Researcher Dr Stuart Peltz explained: “Many inherited diseases are caused by mutations in genes, which stop cells from making vital proteins and, at present, there are many serious illnesses of this type for which there is currently no effective treatment. We hope to be able to bring this new drug to market, not only quickly for cystic fibrosis, but over time to many different patients with many different diseases for which there is currently no treatment.”
In cystic fibrosis a mutation stops the body’s production of a protein called CTFR. Its absence causes the body’s fluids to become abnormally thick and sticky, causing breathing and digestive problems.
In tests on CF sufferers, PCT124 boosted production of the protein and improved breathing. It is thought the drug could help 10 per cent of the 8,000 UK CF sufferers.
Tests on youngsters with Duchenne muscular dystrophy have also proved promising.
Dr Peltz said: “The difference between this and other drugs is it doesn’t just treat the symptoms, it treats the underlying cause. The drug allows the body to make the lost protein.”
It is hoped PCT124, which would have to be taken daily for life, could be on the market as early as 2010 as widespread trials continue.
martes, 9 de septiembre de 2008
Sarah Walters ha cumplido 49 años con Fq
La gran Sarah Walters, de Reino Unido, ha cumplido 49 años, leer su web es toda una alegría para todos los afectados de Fq. Esta mujer es a sus 49 años(sin trasplantar) un ejemplo a seguir en Fq.
Es médico, motera, heavy metalera y hace cantidad de ejercicio todos los días, es instructora de sky y de varios deportes. Os recomiendo su web.
http://www.docsquid.com/aboutme.htm
Es médico, motera, heavy metalera y hace cantidad de ejercicio todos los días, es instructora de sky y de varios deportes. Os recomiendo su web.
http://www.docsquid.com/aboutme.htm
Últimos Avances en Fq De Vertex Farmaceutics y su Vx-770
A new drug therapy may represent a tremendous step forward in the treatment of some 70,000 cystic fibrosis (CF) patients worldwide, Dr David Sheppard from the University of Bristol told an audience at the BA Festival of Science in Liverpool today [9 September]. Speaking at the conference, Dr Sheppard said: 'The early results with VX-770 suggest that drug therapies which target defects at the root of the disease have the potential to improve greatly the quality of life of CF patients.' At the moment there is no cure for CF - a common single-gene disorder in the UK. The disease, which affects about 8,000 people in the UK and 70,000 people worldwide, is due to a defective gene that causes ducts and tubes in the body to become blocked by thick, sticky mucus. This mucus affects the lungs, pancreas, the intestines, the liver and the reproductive organs. One of the most recognisable symptoms of CF is 'salty sweat', caused by the failure of the sweat ducts to reabsorb salt. Existing treatments only alleviate symptoms, for example, physiotherapy to clear the air passages, antibiotics for lung infections and enzymes to aid the digestion of food. The defective gene disables or destroys a protein known as CFTR. To date around 1,500 genetic defects have been found in this protein. In general, genetic defects cause harm in two ways - some stop the protein from travelling to its correct destination in cells, whereas others prevent the protein from working properly. Dr Sheppard's research group, supported by the Cystic Fibrosis Trust, investigates how new drugs restore function to defective CFTR proteins and EuroCareCF, of which Dr Sheppard is the coordinator, works to promote CF therapy development in Europe. The new drug therapy (VX-770) was developed by Vertex Pharmaceuticals and will tackle the 'functional' defect. It has been tested on CF patients in the US who carry a genetic defect known as G551D. European trials are expected in the future. Early results are very encouraging. Patients who received 150mg twice a day saw the concentration of salt in their sweat decrease by almost 50 per cent and lung function improve by 10 per cent. The Cystic Fibrosis Foundation funded the discovery and early development work of VX-770 in collaboration with Vertex Pharmaceuticals. To date the Foundation has invested $79 million in the project. Results of the trials can be found here. Dr Sheppard's research group and other academic groups and companies are also working to develop new drugs that tackle defects at the root of CF. Current life expectancy for CF patients is between 30 and 40 years of age in Western Europe and the US but is considerably lower in other parts of the world. One in 25 people in the UK is a carrier of a defective CF gene, making CF one of the most common life-shortening, childhood-onset, inherited diseases. http://www.bristol.ac.uk/
lunes, 11 de agosto de 2008
El día 23 de Julio fuimos recibidos por el Presidente de la Xunta de Galicia
El pasado día 23 de Julio, nuestra Presidenta Angeles Campos fue recibida por el Presidente de La Xunta Emilio Pérez Touriño.
Todos confiamos en que todas estas reuniones con altos dirigentes de la escala Pública tengan pronto sus resultados positivos plasmados en avances, ayudas, subvenciones para nuestro colectivo.
Muchas Gracias
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