Some mixed news this week on the new drug development front for GlaxoSmithKline (NYSE: GSK):

  • Fabry Disease Candidate Fails

Amicus Therapeutics shares fell the most ever after the company said its experimental treatment for Fabry disease failed in a clinical trial. It is partnered with GSK on the effort.

Amicus dropped 47 percent to $3.06 on Thursday, the biggest one-day drop since it began trading in 2007. Amicus is based in Cranbury, N.J.

Fabry disease is a rare genetic disorder in which patients can’t break down fats that accumulate in the body’s organs. A six-month result from the 67-patient trial showed that the experimental drug didn’t perform better than a placebo, Amicus and Glaxo said in a statement Wednesday.

The study, in the third and final stage of testing typically required for regulatory approval, will continue for a full year, the companies said.

No serious adverse events were reported in the clinical trial, the companies said.

However, in statements from the companies, the firms said they are not giving up on the drug.

John F. Crowley, Chairman and Chief Executive Officer of Amicus: “Consistent with our Phase II experience, the six-month results from Study 011 demonstrate notable trends in kidney interstitial capillary GL-3 reduction in favour of migalastat HCl monotherapy compared to placebo. We look forward to announcing additional six-month results at the WORLD Symposium in February, including a presentation of important secondary and tertiary endpoints in this study. We also anticipate 12-month results from this study in the first half of 2013. Once we have the 12-month data, we intend to meet with FDA to discuss a U.S. approval pathway. We continue to believe that migalastat HCl may become an important treatment option as an oral monotherapy drug for men and women with Fabry disease who have amenable mutations.”

Marc Dunoyer, Global Head of GSK Rare Diseases: “GSK and Amicus are committed to advancing migalastat HCl as a monotherapy in Fabry patients with amenable mutations. While these 6-month data are encouraging, there is additional work to be done. We continue to analyse the six-month results and look forward to receiving the 12-month results from this study. In addition, the results of Study 012, our second Phase 3 Fabry monotherapy study, will add to the totality of our data and give us a more complete picture of the clinical effect of migalastat HCl. This study, an 18-month comparison of migalastat to ERT, with iohexol GFR as the primary endpoint, is fully recruited and due to report in 2014.”

  • GSK Partners with Swiss Firm

GSK says it will collaborate with GlycoVaxyn AG to develop new anti-bacterial conjugate vaccines, an $8 billion market.

The closely held company will receive an upfront payment and an equity investment from GSK, which has an option to obtain an exclusive license on vaccines they work on during a three-year collaboration period, Schlieren, Switzerland-based GlycoVaxyn said in a statement today. The company is also eligible to receive milestone payments and royalties if the vaccines win regulatory approval.

Financial terms weren’t disclosed.

GlycoVaxyn focuses on vaccines against common severe bacterial infections. Its most advanced program is a vaccine against the Shigella bacterium, which causes dysentery and bloody diarrhea. Pre-clinical candidates include vaccines to prevent hospital infections and E. coli.

“Collaboration with a vaccine industry leader confirms the potential of our platform to generate new complex vaccines,” GlycoVaxyn Chief Executive Officer Philippe Dro said in the statement.
GlycoVaxyn uses recombinant DNA technology that allows it to target bacterial diseases for which effective vaccines don’t yet exist, according to the company. Investors in GlycoVaxyn include Sofinnova Partners, Index Ventures and Edmond de Rothschild Investment Partners.

GSK operates its North American headquarters in RTP.

[GSK ARCHIVE: Check out 10 years of GSK stories as reported in WRAL Tech Wire.]

(Bloomberg contributed to this report.)