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Amicus Therapeutics (FOLD) Updates on FY15 Outlook; Plans Migalastat HCl Marketing App Submission; Names New COO

January 12, 2015 9:05 AM EST

Amicus Therapeutics (NASDAQ: FOLD) provided its full-year 2015 strategic outlook and financial guidance.

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "2014 was a transformational year for Amicus. We enter 2015 with great momentum and equally high expectations. We expect many important firsts for Amicus in 2015 including our first regulatory submissions for our novel oral Fabry monotherapy as well as our first human clinical study of our highly differentiated, proprietary next-generation ERT for Pompe disease. These milestones will significantly advance our goal of building one of the world's leading biotechnology companies focused on the treatment of rare and orphan diseases and improving the lives of people living with rare disorders around the world. I am very optimistic and look forward to leading Amicus for many years to come as we fulfill this great vision."

Key 2015 Highlights

  • Migalastat monotherapy FDA interaction planned in 1Q15. MAA submission on track for mid-2015 to seek marketing approval for Fabry patients with amenable mutations.
  • Initiation of longer-term Phase 2 Fabry co-administration study anticipated in 2015 in support of Fabry franchise strategy to develop migalastat in combination with ERT for Fabry patients with non-amenable mutations.
  • Next-generation Pompe ERT (ATB200 + chaperone) set to enter clinic in 2H15. Additional details on Pompe program to be highlighted at 33rd Annual J.P. Morgan Healthcare Conference.
  • Strong balance sheet supports current development programs, biologics manufacturing capabilities, and initial commercial infrastructure to establish leading global rare disease company
    • Full-year 2015 net cash spend expected to range between $73 million and $83 million
    • Current cash ($169.1 million at December 31, 2014) projected to fund operating plan into 2017

Mr. Crowley will discuss Amicus' corporate objectives and key milestones in a presentation at the 33rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 13, 2015 at 10:30 a.m. PT (1:30 p.m. ET). A live webcast of the presentation can be accessed through the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicustherapeutics.com/events.cfm, and will be archived for 90 days.

Highlights and Milestones by Program

Fabry Franchise

Amicus is preparing to submit marketing applications for the oral pharmacological chaperone migalastat HCl ("migalastat") as a monotherapy for Fabry patients who have amenable mutations. Positive Phase 3 data in both treatment naïve and ERT switch patients have shown that treatment with migalastat has resulted in reductions in disease substrate, stability of kidney function and improvement in a key cardiac parameter (left ventricular mass index, or LVMI) in patients with amenable mutations. For all other Fabry patients who do not have amenable mutations and cannot take monotherapy, Amicus is advancing migalastat in combination with ERT.

Anticipated 2015 Fabry Franchise Milestones:

  • FDA meeting to discuss migalastat monotherapy in 1Q15
  • Migalastat monotherapy MAA submission in mid-2015
  • Initiation of longer-term Phase 2 study of oral migalastat co-administered with currently marketed ERTs in 2H15
  • Internal development underway of next-generation ERT (bio-better Fabry ERT cell line for co-formulation with migalastat)

Next-Generation ERT for Pompe Disease (ATB200 + Chaperone)

Amicus is leveraging its biologics capabilities to develop a bio-better, uniquely engineered rhGAA enzyme with an optimized carbohydrate structure (designated ATB200) to enhance uptake, in combination with a pharmacological chaperone to improve activity and tolerability. In preclinical studies ATB200 has demonstrated greater enzyme uptake into tissues and further substrate reduction compared to the current standard of care Pompe ERT (Myozyme/Lumizyme). Clinical studies of pharmacological chaperones in combination with currently marketed ERTs have established initial human proof-of-concept that a chaperone can stabilize enzyme activity and potentially improve ERT tolerability.

Anticipated 2015 Pompe Program Milestones:

  • GMP batch of ATB200 in 1Q15
  • Pre-IND meeting in mid-2015
  • Phase 1/2 study initiation in 2H15

2015 Financial Guidance

Cash, cash equivalents, and marketable securities totaled $169.1 million at December 31, 2014 compared to $82.0 million at December 31, 2013. The Company's balance sheet was strengthened during 2014 with a $40 million at-the-market (ATM) financing as well as a $103.5 million public offering. Amicus expects full-year 2015 net cash spend between $73 million and $83 million. The current cash position is projected to fund operations into 2017.

Company Leadership

Amicus also announced that Bradley L. Campbell has been promoted to President and Chief Operating Officer. Mr. Campbell will continue to report to Mr. Crowley and will also continue to oversee a broad range of activities at Amicus including: Research and Development; Technical (Manufacturing) Operations; Program Management; and Patient Advocacy and Public Policy. He was previously named Chief Operating Officer in 2013 and prior to that he has held a number of positions at Amicus, including Chief Business Officer.



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