Yesterday I was on my way back from Marblehead and the Tufts Cycle for Life event and stopped by Boston for meetings.
While standing on the pavement contemplating my schedule, I checked out my industry news feeds to see what was happening.
Beantown was hopping around me yesterday!
Announced the phase II trial of it's multi-kinase inhibitor, AP24534, which targets the T315I mutation in CML and is now named ponatinib. The drug also inhibits FLT3-ITD and the FGFR pathway.
According to Ariad:
"The PACE (Ponatinib Ph+ ALL and CML Evaluation) trial is designed to provide definitive clinical data for regulatory approval of ponatinib in this setting."
The goal will be to focus on the refractory setting after failure of Sprycel or Tasigna:
"The PACE trial is a global, single-arm clinical study of oral ponatinib in 320 patients with chronic phase, accelerated phase, or blast phase CML, as well as Ph+ ALL. Patients resistant or intolerant to dasatinib (Sprycel) or nilotinib (Tasigna), or with T315I mutation of BCR-ABL, will be enrolled in the trial."
Originally, this would have meant ponatinib would be used in 3rd line after imatinib, the dominant front-line therapy, and then either dasatinib or nilotinib in 2nd line. However, as the 2nd generation therapies move up to first-line, with nilotinib already approved in this indication and dasatinib due to be reviewed by the FDA soon, this could well mean that ponatinib could be used in 2nd line in the future should it garner good data and approval from the FDA.
This is a particularly promising agent because it is the leading compound being evaluated for the T315I mutation in CML, which none of the existing drugs target.
While all attention is on the seemingly never ending dance between Sanofi-Aventis and Genzyme over the possible buyout deal and price, an interesting press release from the biotech firm quietly announced the divesting of the genetic testing business to Lab Corp:
"LabCorp will purchase the business in its entirety, including all testing services, technology, intellectual property rights, and its nine testing laboratories. LabCorp is committed to offer employment to the unit’s approximately 1900 employees upon closing, including senior management. The agreement is subject to customary closing conditions, including the Hart-Scott-Rodino Antitrust Improvements Act of 1976, with the goal of closing before the end of the year."
This comes after the announcement late last week that 1,000 jobs will be lost over the next 2 years, which was rolling over the local news channels over the weekend. The mood in MA was less the buoyant as a result. The fallout from the manufacturing problems and subsequent fines are clearly having a major impact on the troubled biotech. I wonder what will be next to go?
Last but not least, a more uplifting development yesterday was the announcement by Ironwood and Forest Labs regarding positive top-line results from the first of two Phase 3 clinical trials assessing the efficacy and safety of their investigational drug, linaclotide, in patients with irritable bowel syndrome with constipation (IBS-C).
According to the press release:
"The two co‐primary endpoints required by the European Medicines Agency (EMA) were met in this study, showing statistical significance and clinically relevant improvement for linaclotide treated patients both for abdominal pain/abdominal discomfort responder and IBS degree of relief responder over the three‐month period. Significant improvement was also achieved for all pre‐specified main secondary endpoints (stool frequency, stool consistency, straining, and bloating).
The safety results were consistent with those observed in previous linaclotide clinical trials, with diarrhea being the most common adverse event in linaclotide‐treated patients."
This is very good news indeed for people suffering with the condition, especially as two huge global drug makers (GSK and Novartis) both tried, but failed, to bring a drug to market and keep it there.
Bear in mind that IBS is a debilitating and inconvenient condition that affects a lot lot of people, perhaps as many as 30 million suffer from the effects. It might sound a little dramatic, but IBS has unfortunately become a sort of sword-in-the-stone challenge to big Pharma. Ironwood may well be the young 'Arthur' for millions of people suffering with IBS.
If the safety and efficacy data hold up with the regulatory authorities, an approval for linaclotide would be very good news indeed for people suffering from this condition.
Photo Credit: Werner Kunz