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® Marketing Authorisation Application remains on track with European Medicines Agency (EMA);
o Responses to Day 120 questions submitted in November 2010;
o Day 180 list of Outstanding Issues now received;
· Raised €14.3 million through private placement;
· Novel biomarker for Glybera® 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 € 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.
Jörn Aldag, Chief Executive Officer of AMT, commented: “We are excited by the key milestones achieved in 2010: our lead product Glybera® 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® 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® with the European Medicines Agency (EMA) as a treatment for lipoprotein lipase deficiency (LPLD), the agency conducted its initial review of the Glybera® 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®, 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® 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 € 3.3 million for this project has been secured from the EU, with €1.1 million assigned to AMT, supporting our financial commitment to this project.
-Duchenne Muscular Dystrophy
The DMD program continues to progress, with up to € 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 Française 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 € 14.3 million of new funds, before expenses (€13.3 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 € 1.4 million, a € 1.0 million increase compared to the total net income for the year ended December 31, 2009, which amounted to € 0.4 million. These revenues represent grant income from the Dutch government and the European Union.
Operating costs
Research and development expenditure totaled € 16.4 million, compared to € 13.2 million in 2009, an increase of € 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 € 0.5 million of impairment charges and the largest part of the € 0.5 million increase in charges relating to share-based incentive schemes, both of which represent non-cash items.
General and administrative costs decreased to € 4.1 million, from € 4.9 million in 2009. This decrease reflected the lower level of advisory costs in 2010, compared to 2009, as well as the fact that 2009 contained certain reorganization costs.
Operating result
AMT’s operating loss rose to € 19.1 million for 2010, from € 17.8 million for 2009, an increase of € 1.3 million. This increase can be largely accounted for by certain non-cash items amounting to € 1.0 million, comprising an increase of € 0.5 million in charges relating to share-based incentive schemes, a € 0.3 million charge relating to impairment charges on certain intangible assets following the termination of a 2008 collaboration agreement, and a € 0.2 million charge in respect of the impairment of certain leasehold improvements. After excluding for these non-cash, non-recurring items, the operating loss in 2010 would have been broadly equivalent to the operating loss for 2009.
Finance income and costs
Net finance income fell to € 0.5 million compared to € 0.6 million in 2009, reflecting the lower average cash balances of the Group during 2010 during a period when interest rates available on cash deposits remained low. In addition, finance costs increased to € 0.5 million compared to € 0.0 million, reflecting the interest charge payable on the 2009 convertible bond, together with foreign exchange movements on currency accounts. Of these finance costs, € 0.2 million relates to non-cash items.
Result for the Year and Loss per Share
Total net loss for the year ended December 31, 2010 amounted to € 19.1 million, compared to the net loss for the year ended December 31, 2009, which amounted to € 17.2 million, an increase of € 1.9 million. The increase in the net loss includes an increase in non-cash charges of € 1.2 million described within the ‘Operating loss’ and ‘Finance income and costs’ paragraphs above. The loss per share amounted to € 1.13 for 2010 compared to € 1.17 for 2009. The basic and diluted loss per share are the same because the company is loss-making in both periods.
Cash Flow and Cash Position
Cash and cash equivalents amounted to € 17.9 million at December 31, 2010, a decrease of € 4.8 million or 21% compared to € 22.6 million at December 31, 2009. The decrease in cash and cash equivalents is mainly the result of cash used in operating activities amounting to € 17.7 million in 2010 (2009: €16.5 million), offset by net cash generated from financing activities of € 13.4 million.
The cash used in operating activities represents our operational loss adjusted for non-cash items such as share-based payment expenses and changes in working capital.
The cash flow from financing activities amounted to € 13.4 million reflecting the issue of new shares in October 2010, compared to € 4.8 million in 2009, which principally represented the drawdown of the convertible loan.
Equity
Shareholders’ equity amounted to € 13.5 million at December 31, 2010 compared to € 18.4 million at December 31, 2009. A total number of 23,512,225 shares were issued and outstanding at December 31, 2010.
Outlook
The company expects its existing cash resources, together with the net cash inflows that it expects to generate during 2011 from partnering activities and from the commercialization of Glybera® that would follow a successful MAA submission, to be sufficient to fund its operations for at least the next 12 months from the date of publication of the audited consolidated annual accounts, which is expected to be on March 4, 2011. In reaching this conclusion, the Management of AMT has considered the uncertainty inherent in forecasting future net cash inflows, and believes that its expectations, as described above, are reasonable.
To discuss these results, AMT’s management will conduct a conference call at 3:30 PM CET, which will also be webcast. To participate in the conference call, please call one of the following telephone numbers 15 minutes prior to the event, using access code 3705648: +44 (0)20 7806 1956 for the UK; +1 212 444 0413 for the US; and +31 (0)20 707 5510 for the Netherlands. Following the results presentation, the lines will be opened for a question and answer session. A replay of the call will be available following the event.
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