In the news release, "Amsterdam Molecular Therapeutics Reports Full Year Results 2010" issued on 16 Feb 2011 06:00 GMT, by Amsterdam Molecular Therapeutics B.V AEX:AMT over PR Newswire, we are advised by a representative of the company that the full year figures should have been included in the dissemination. Complete, corrected release follows
AMSTERDAM, February 16, 2011 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today reported its results for the year to December 31, 2010.
Key Highlights
- Glybera(R) Marketing Authorisation Application remains on track with
European Medicines Agency (EMA);
- Responses to Day 120 questions submitted in November 2010;
- Day 180 list of Outstanding Issues now received;
- Raised EUR14.3 million through private placement;
- Novel biomarker for Glybera(R) activity identified;
- Diagnostic gene chip for LPLD developed by AMT's collaborator,
Progenika, obtained CE Mark;
- Hemophilia Phase I/II study underway, being led by St Jude Children's
Hospital and UCL Hospital; data from initial patients showed safe,
Stable expression;
- DMD program progressing with US Orphan Drug Status granted and up to
EUR 4 million Senter Novem funding;
- Porphyria program funding through EU grant now finalized;
- GDNF program progressing following renegotiation of collaboration
agreement with Amgen; and
- New collaboration initiated with consortium led by Institut Pasteur to
develop Sanfilippo gene therapy.
Jorn Aldag, Chief Executive Officer of AMT, commented: "We are excited by the key milestones achieved in 2010: our lead product Glybera(R) is progressing towards market approval and we are looking forward, with confidence, to the Regulatory Agency's decision. We have continued, also, to make good progress in our other pipeline programs, on both the R&D and partnering fronts."
Operations
Lipoprotein Lipase Deficiency (LPLD)
The advance of AMT's lead product, Glybera(R) towards market approval was the key focus of the Group's activities during 2010. The regulatory process continues to progress on schedule. Following the filing of Glybera(R) with the European Medicines Agency (EMA) as a treatment for lipoprotein lipase deficiency (LPLD), the agency conducted its initial review of the Glybera(R) registration dossier in early 2010 and sent the Day 120 List of Questions to AMT in May. AMT submitted its response to the EMA in November 2010, based in part on additional data and analyses from patients previously treated with Glybera(R), including new data available from the last clinical trial and its one-year extension. The EMA restarted evaluation on November 26 (Day 121), and following the normal centralized review procedure, AMT has received, at Day 180, a significantly reduced list of outstanding items. We are now in the process of compiling the responses and expect to submit answers to the EMA by end of the first quarter and believe to be on track for a mid 2011 response on marketing approval.
Because AMT's technology can be applied equally to a wide range of other genetic diseases, the success of Glybera(R) would validate AMT's approach for its other pipeline products, targeting a range of orphan diseases and diseases with large patient populations, in each case where there is high unmet medical need, including Parkinson's disease, hemophilia, Duchenne muscular dystrophy (DMD) and acute intermittent porphyria (AIP).
Hemophilia B
The hemophilia B program has entered a Phase I/II clinical study, led by our partners St Jude Children's Research Hospital, Memphis, Tennessee, and University College London (UCL) Hospital, London, UK. Initial data presented at the American Society of Hematology (ASH) conference in Florida in December 2010 showed that dose-dependent, safe, stable, and persistent expression in patients has been observed.
Porphyria
Our collaboration with FIMA in Pamplona, Spain, to develop a gene therapy for acute intermittent porphyria (AIP) is progressing very well, and has generated positive pre-clinical data. The program is now entering toxicology studies and a Phase I/II study is scheduled to begin in early 2012. Total grant funding of EUR 3.3 million for this project has been secured from the EU, with EUR1.1 million assigned to AMT, supporting our financial commitment to this project.
Duchenne Muscular Dystrophy
The DMD program continues to progress, with up to EUR 4 million of financial support from Senter Novem, which covers 35% of development costs through to completion of the Phase I/II study. In September 2010, the US FDA granted our gene therapy for DMD Orphan Drug status. This complements the Orphan Drug status granted to the same program by the EMA covering Europe in 2009.
GDNF
AMT has successfully renegotiated the terms of its collaborative agreement with Amgen covering the use of GDNF in gene therapies. AMT now has the ability to explore development of additional indications, including orphan diseases, alongside the development of Parkinson's disease. The University of Lund, Sweden, is performing preclinical studies in Parkinson's disease models for this program, the results of which are encouraging.
SanfilippoB
Immediately after the year-end, AMT announced a collaboration with Institut Pasteur and the Association Francaise contre les Myopathies (AFM) to develop a gene therapy for the rare lysosomal disorder, Sanfilippo B for which Institut Pasteur has achieved preclinical proof of concept. Under the agreement, AMT will receive funding to manufacture the product using its AAV vector technology to progress the program through a Phase I/II study. Thereafter, AMT will have an option to acquire full commercial rights to the program.
Financing
In October 2010, AMT successfully raised EUR 14.3 million of new funds, before expenses (EUR13.3 million net of costs) via a private placement of ordinary shares. These funds together with our existing cash resources take AMT comfortably through the assessment process of Glybera. The Company also expects to generate significant additional revenue from commercializing Glybera and from developing its collaborations with other parties. Taking these additional funding sources into account, AMT believes it will have sufficient cash resources to meet its operational requirements.
Results
Revenues
The total net income for the year ended December 31, 2010 amounted to EUR 1.4 million, a EUR 1.0 million increase compared to the total net income for the year ended December 31, 2009, which amounted to EUR 0.4 million. These revenues represent grant income from the Dutch government and the European Union.
Operating costs
Research and development expenditure totaled EUR 16.4 million, compared to EUR 13.2 million in 2009, an increase of EUR 3.2 million reflecting the ongoing level of activity to support the filing of Glybera, as well as ongoing development on AMT's other projects, including the Duchenne program (which is partly funded by the investment credit from Senter Novem) and reflecting the activities financed by the grants described above. In addition, research and development expenditure in 2010 included the EUR 0.5 million of impairment charges and the largest part of the EUR 0.5 million increase in charges relating to sha