By Ed Silverman
In a development that greatly bolsters its standing in the cystic fibrosis market, Vertex Pharmaceuticals late yesterday released data showing a combination of its existing Kalydeco treatment and an experimental medication called VX-661 greatly improved patient breathing. Specifically, lung functioning rose 9 percent and 7.5 percent in the two highest dose groups compared with a placebo over 28 days (read more here).
By Brian Orelli, The Motley Fool
Filed under: Investing
If one “Breakthrough Therapy Designation” is good, three must be great.
That is, if we knew what the designation was really worth.
Johnson & Johnson announced on Monday that it had received a third Breakthrough Therapy Designation for the blood cancer drug ibrutinib that it’s developing with Pharmacyclics . The designation covers patients with chronic lymphocytic leukemia or small lymphocytic lymphoma who have a deletion of the short arm of chromosome 17.
In February, the Food and Drug Administration gave the drug the designation for patients with relapsed or refractory mantle cell lymphoma that failed prior therapy and for patients with Waldenstrom’s macroglobulinemia, a type of cancer affecting B cells.
The Breakthrough Therapy Designation was part of the last year’s FDA Safety and Innovation Act, which is supposed to speed up development review time — potentially requiring less data for approval — for drugs that treat serious diseases where there isn’t a treatment or where the product in development is a vast improvement over current options.
But Congress, in its infinite wisdom, left exactly how to implement the benefits up to the FDA. That was probably a good idea in the long run since the FDA knows what drugmakers need, but the agency isn’t particularly quick in establishing rules.
Companies are left making announcements about a designation that they don’t know exactly how they’ll benefit from. “The implications of Breakthrough Therapy Designation cannot be determined at this time,” read the press release by Johnson & Johnson’s subsidiary Janssen Research & Development.
Vertex Pharmaceuticals had the exact same wording — maybe their flack went to the same law school — in the announcement that the FDA had given Breakthrough Therapy Designation to its cystic fibrosis drug Kalydeco as both a monotherapy and in combination with VX-809.
The biotech added that it’s working with the FDA and other regulatory authorities to figure out exactly how it’ll affect the development. It sure doesn’t look like the designation will help reduce the size of the clinical trials required for approval. Vertex subsequently announced that it’s started two 500-patient phase 3 trials testing VX-809 with Kalydeco in cystic fibrosis patients with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.
The designation might not have much benefit, but don’t ignore the drugs involved; both Vertex’s combination treatment and ibrutinib have a lot of promise. Just this week, promising data from a clinical trial testing ibrutinib in hart-to-treat chronic lymphocytic leukemia patients was presented at the American Association for Cancer Research annual meeting.
At this point, the Breakthrough Therapy Designation is like wearing a broken luxury watch; it’s a nice status symbol, but it isn’t particularly useful. Some will argue that it says that the FDA thinks the drug has promise, but that should be obvious to most investors anyway. The drugs still have to prove their worth in clinical trials to get approved.
Is bigger really better?
Involved in everything from baby powder to biotech, Johnson & …read more
Source: FULL ARTICLE at DailyFinance
By Business Wirevia The Motley Fool
Filed under: Investing
Vertex Enters Agreement with Bristol-Myers Squibb for Phase 2 All-Oral Studies of VX-135 in Combination with Daclatasvir for the Treatment of Hepatitis C
-Two Phase 2 studies to evaluate once-daily combination of Vertex’s investigational nucleotide analogue VX-135 and BMS’ investigational NS5A replication complex inhibitor daclatasvir-
-Study in people with genotype 1 hepatitis C planned to begin in second quarter of 2013-
-Study in people with genotypes 1, 2 and 3 hepatitis C, including people with cirrhosis, planned for second half of 2013-
CAMBRIDGE, Mass.–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (NAS: VRTX) today announced it has entered into a non-exclusive agreement with Bristol-Myers Squibb Company (NYS: BMY) to conduct Phase 2 studies of once-daily all-oral treatment regimens containing Vertex’s nucleotide analogue hepatitis C virus (HCV) polymerase inhibitor VX-135 and Bristol-Myers Squibb’s NS5A replication complex inhibitor daclatasvir for the treatment of hepatitis C. As part of the agreement, Vertex plans to conduct two Phase 2 studies of the combination, including an initial study in treatment-naïve people with genotype 1 HCV infection planned for the second quarter of 2013. Vertex plans to begin a subsequent study in treatment-naïve people infected with genotype 1, 2 or 3 HCV, including those with cirrhosis, in the second half of 2013, pending data from the initial study.
“With more than 170 million people infected worldwide, there is a critical need for new hepatitis C medicines that can offer people simpler and more tolerable treatment regimens that provide high cure rates,” said Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical Officer at Vertex. “These studies with daclatasvir will provide the first opportunity to evaluate VX-135 as part of all-oral regimens in people with multiple hepatitis C genotypes and in people with cirrhosis.”
Clinical Development Plans for VX-135 with Daclatasvir
Under the terms of the agreement, Vertex will conduct two Phase 2 studies of VX-135 in combination with daclatasvir. The first study will enroll approximately 20 non-cirrhotic, treatment-naïve people with chronic genotype 1 HCV infection and is expected to begin in the second quarter of 2013. In the second half of 2013, …read more
Source: FULL ARTICLE at DailyFinance
Nestle SA (NESN.VX) is withdrawing its beef pasta meals from sale in Italy, Spain and France after finding traces of horse DNA in them. …read more
Source: FULL ARTICLE at Fox Business Headlines
A team of high-producing brokers has joined UBS AG’s (UBS, UBSN.VX) retail brokerage division in California from Morgan Stanley’s (MS) wealth-management unit.
Source: FULL ARTICLE at Fox Business Headlines
The European Union proposed tough restrictions on three insecticides suspected of harming honey bees, raising the stakes in a fight between authorities and the chemicals’ manufacturers, Syngenta AG (SYT, SYNN.VX) and Bayer AG (BAYRY, BAYN.XE).
Source: FULL ARTICLE at Fox Business Headlines
A judge has ordered U.S. tax authorities to seek information from UBS AG (UBS, UBSN.VX) on American taxpayers who might hold accounts at Swiss banks with the aim of evading federal income taxes, the U.S. Attorney for the Southern District of New York‘s office said Monday.
Source: FULL ARTICLE at Fox Business Headlines
Swatch Group (UHR.VX) said Monday it had agreed to buy U.S. watch-and-accessory company HW Holdings Inc., owner of the Harry Winston brand, for $1 billion, in an acquisition that strengthens its presence in the U.S. market and helps it expand into jewelry.
Source: FULL ARTICLE at Fox Business Headlines
