CRANBURY, N.J., May 13 Amicus Therapeutics(Nasdaq: FOLD), a biopharmaceutical company developing small molecule,orally-administered pharmacological chaperones for the treatment of humangenetic diseases, today announced financial results for the first quarter of2008. On a reported basis calculated in accordance with U.S. GenerallyAccepted Accounting Principles (GAAP), Amicus announced a net lossattributable to common stockholders of $0.34 per share ($0.28 per share on anon-GAAP basis) for the three months ended March 31, 2008. As of March 31,2008, cash, cash equivalents, and marketable securities totaled $154.6million.
"In the first quarter of 2008, we continued to advance our three leadclinical development programs. We also reported data from multiple clinicaltrials that add to the growing body of evidence demonstrating that treatmentwith our oral pharmacological chaperone drug candidates has the potential tobenefit patients with Fabry, Gaucher and Pompe disease," said John F. Crowley,President and CEO of Amicus Therapeutics. "In the months ahead in 2008, wewill be focused on advancing the global regulatory plan for Amigal Phase 3development, evaluating opportunities for additional clinical studies inGaucher and Pompe disease, as well as on identifying new therapeutic areas forapplying our pharmacological chaperone technology platform."
Amigal(TM) (migalastat hydrochloride) is an investigational, oraltherapeutic being developed for the treatment of Fabry disease. In March,clinical investigators presented positive results from Phase 2 clinical trialsof Amigal at the American College of Medical Genetics (ACMG) meeting. The datashowed that Amigal was generally safe and well-tolerated at all dosesevaluated and no drug-related serious adverse events were reported. Inaddition, Amigal increased the level of the enzyme deficient in Fabry patientsin 24 of 26 study subjects, and in a majority of study subjects the treatmentresulted in a reduction of kidney GL-3 as measured in urine.
Amicus, along with its partner Shire, has initiated discussions with theU. S. Food and Drug Administration (FDA) and the European Medicines Agency(EMEA) regarding its plans for Phase 3 clinical evaluation of Amigal. Amicusexpects to complete these interactions and provide an update in the secondhalf of 2008, and subject to the outcome of the discussions, the Company plansto initiate the Phase 3 clinical trial in the first half of 2009.
In parallel with the regulatory process, 23 of the original 26 patientscontinue to be treated with Amigal in the voluntary Phase 2 extension study tomonitor long term safety and efficacy. In addition, the Company will evaluatemodified doses and dose regimens in these 23 patients. Data from thisextension study are expected to be available by Q1 2009, prior to finalizationof the Phase 3 protocol.
Plicera(TM) (isofagomine tartrate) is an investigational, oral therapeuticbeing developed for the treatment of Gaucher disease. At the ACMG meeting inMarch, clinical investigators presented full data from a 4 week Phase 2 studyin Gaucher patients who switched from enzyme replacement therapy (ERT) withimiglucerase to the pharmacological chaperone Plicera. Results showed thatPlicera was generally safe and well tolerated at all doses and increasedtarget enzyme activity levels in a majority of patients. In the trial, GCaseactivity, as measured in white blood cells, was increased in 20 of the 26patients with evaluable GCase data, and 5 of the 6 patients without a clearincrease were either in the lowest dose cohort or the cohort dosed leastfrequently. As expected in this short term study, the levels of relevanthematological markers of Gaucher disease remained stable.
Amicus has amended the protocol for the 6-month Phase 2 clinical trial ofPlicera patient