Company Remains on Track to Advance Amigal(TM) for Fabry Disease into Phase 3 in Second Quarter
CRANBURY, N.J., May 7 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced financial results for the first quarter 2009.
On a reported basis calculated in accordance with U.S. Generally Accepted Accounting Principles (GAAP), Amicus announced a net loss attributable to common stockholders of $0.55 per share ($0.46 per share on a non-GAAP basis) for the three months ended March 31, 2009. As of March 31, 2009, cash, cash equivalents, and marketable securities totaled $109 million.
'Our first quarter was highlighted by positive Phase 2 extension data and continued progress towards our final Phase 3 development plan for Amigal in Fabry disease,' said John F. Crowley, Amicus' President and CEO. 'We look forward to announcing our final protocol over the upcoming weeks and starting the trial later this quarter.'
Clinical Program Updates
Amigal(TM) (migalastat hydrochloride) for the treatment of Fabry disease
In the first quarter of 2009, Amicus continued to work closely with the U.S. and E.U. regulatory authorities in an effort to finalize its Phase 3 development plan for Amigal. The Company remains on track to announce the final Phase 3 protocol intended to support U.S. approval and to initiate Phase 3 development of Amigal in the second quarter of 2009.
Additionally, in March 2009 at the American College of Medical Genetics (ACMG) 2009 Annual Meeting in Tampa, FL, the Company presented positive results from its Phase 2 extension study with Amigal.
Amicus reported that treatment with Amigal was generally well-tolerated, with no drug-related serious adverse events. The most common adverse events were headache, arthralgia and diarrhea.
Subjects identified as responders to Amigal at the completion of the Phase 2 studies continued to maintain elevated levels of the target enzyme (α-Gal A), as measured in white blood cells, and reduced levels of the target substrate (kidney GL-3), as measured in urine.
A reduction of GL-3 levels was also observed in interstitial capillary cells from kidney biopsies. Previously reported Phase 2 results indicated that little to no GL-3 was detected in these cells in most subjects prior to treatment with Amigal. The new data were obtained from the retesting of biopsies using an improved methodology.
Preliminary results from the evaluation of modified doses and a new dosing regimen were also presented.
Plicera(TM) (afegostat tartrate) for the treatment of Gaucher disease
A Phase 2 clinical trial of the Company's investigational drug Plicera is ongoing. This 6-month study is designed to evaluate safety and to demonstrate trends of efficacy, as measured by the standard endpoints in Gaucher disease. The Company expects to report the results late in the third quarter of 2009.
Amicus will continue to work closely with its partner, Shire HGT, to prepare for Phase 3 development of Plicera pending the results of the ongoing Phase 2 trial.
Furthermore, during the first quarter, the Company announced the issuance of United States Patent No. 7,501,439, titled 'Tartrate Salt of Isofagomine and Methods of Use.' The patent covers the tartrate salt form of isofagomine, the active ingredient in the Plicera, and its use for the treatment of Gaucher disease. The patent will expire in 2027.
AT2220 (1-deoxynojirimycin HCl) for the treatment of Pompe disease
As previously reported, the Company suspended enrollment for the Phase 2 clinical trial of its investigational drug AT2220 for the treatment of Pompe disease and received notice from the U.S. Food and Drug Administration (FDA) that the trial is on clinical hold.
The Company is currently evaluating all data and continues to work closely with the FDA to determine appropriate next steps for advancing the development of AT2220.
The events have no impact on Amicus' ongoing studies with Amigal for Fabry disease and Plicera for Gaucher disease.
Preclinical Chaperone Programs
Amicus continues to invest in research and development to assess the potential for applying its versatile chaperone technology platform to the treatment of a broader range of human genetic diseases. As part of this effort, Amicus continues to conduct preclinical studies in Parkinson's disease and is investing in new research aimed at evaluating disease targets for other neurodegenerative and genetic disorders.
Shire HGT Collaboration
In November 2007, Amicus entered into a strategic collaboration with Shire Human Genetic Therapies, Inc., a wholly-owned subsidiary of Shire plc, to jointly develop Amicus' three lead pharmacological chaperone compounds for lysosomal storage disorders, Amigal, Plicera and AT2220. Under the terms of the collaboration Shire will pay development and sales milestones up to a maximum of $390 million, and will also pay tiered, double digit royalties on net sales of the products. Shire also reimburses world-wide development costs on a 50/50 basis, and in return Shire received rights to commercialize these products outside of the U.S. while Amicus retains all rights to commercialize these products in the U.S. In addition, Amicus leads development operations through the end of Phase 2 clinical trials.
