Amsterdam Molecular Therapeutics Reports Full Year Results 2011
AMSTERDAM, April 26, 2012
Amsterdam Molecular Therapeutics (AMT 0.00%, news) Holding N.V. (in liquidation) (Euronext: AMT), today reported its results for the year to December 31, 2011.
Key Highlights
• Glybera® Marketing Authorisation Application received a positive assessment from the Committee for Advanced Therapies and the Scientific Advisory Group on reexamination;
• CHMP maintains negative position based on clinical benefit; however no issues raised with respect to safety or CMC manufacturing;
• Latest CHMP vote was 16 in favour vs 15 opposed; however a positive vote of 17 is required for an approvable opinion
• Positive article in New England Journal of Medicine regarding AMT's Hemophilia B gene therapy
• Raised € 2.5 million through private placement;
• New collaboration initiated with consortium led by Institut Pasteur to develop Sanfilippo gene therapy;
• Following rejection of Glybera by CHMP in October 2011, AMT initiated strategic reorganization, reducing headcount by 50% to some 45 FTEs;
• AMT initiated strategic review process leading to the transaction announced in February 2012 to transfer all AMT's gene therapy business to a new, unlisted company, uniQure BV. The transaction completed on April 5, 2012, and AMT received Depositary Receipts in uniQure BV in consideration;
• AMT entered into liquidation on April 5, 2012;
• On April 26, 2012, the uniQure BV depositary receipts were distributed to AMT's shareholders by way of an advance distribution. It is not expected that there will be any further (final) distributions made to AMT shareholders;
• AMT is expected to cease to exist and delisting to take place in summer 2012
Jörn Aldag, Liquidator of AMT, commented: "We believe that the gene therapy business developed by AMT offers significant promise. We are very disappointed that whilst the majority of CHMP members voting followed the advice of the experts at the CAT and the SAG, there were still a sufficient number of blocking votes to prevent Glybera advancing for the benefit of patients. Nevertheless we are encouraged that no safety of manufacturing issues were raised in the latest CHMP assessment. We believe that, under uniQure's ownership, the future of gene therapy development is very encouraging."
Operations
The progression of AMT's lead product, Glybera® through the assessment process was the key focus of the Group's activities during 2011 until the outcome of the reexamination process of the CHMP in October. Thereafter, the Group went through a strategic reorganization to reduce costs and secure additional funds. This resulted in a 50% reduction in headcount and the transfer of AMT's operations and programs to a newly created company uniQure BV. uniQure received an initial € 6.0 m in new financing from one of AMT's investors, Forbion. Since completion of the transaction, on April 5, 2012, another of AMT's investors, Gilde Healthcare, has invested a further € 1.0 m into uniQure.
Lipoprotein Lipase Deficiency (LPLD)
AMT has developed Glybera for the treatment of Lipoprotein Lipase Deficiency (LPLD), a rare and very severe disease. In patients with mutations in the LPL gene, dietary fat (triglyceride molecules) cannot be broken down and so causes chylomicrons, which carry triglycerides around the body, to accumulate in the blood. This may result in recurrent extremely painful and life-threatening episodes of pancreatitis. Pancreatitis, or inflammation of the pancreas, is a major clinical symptom of LPLD. It causes severe abdominal pain and often leads to hospitalization of patients as well as other complications such as diabetes and early atherosclerosis.
AMT submitted the Marketing Authorisation Application (MAA 0.00%, news) for Glybera in December 2009; in June 2011 the CHMP published its opinion that Glybera was not approvable at that time. In July 2011, AMT filed for reexamination of the dossier. Two new rapporteurs examined the application for approval under exceptional circumstances, and both issued positive assessments. The Scientific Advisory Group (SAG), an expert panel specifically selected to evaluate clinical results and the science of the product, and the Committee for Advanced Therapies (CAT 0.00%, news), which provides guidance on advanced therapeutics such as gene and cell therapy, after extensive review and analysis of the data advised the CHMP that Glybera should be approved now under exceptional circumstances. The CHMP Rapporteurs, SAG and the CAT concluded that data from three Glybera clinical trials demonstrated meaningful evidence of clinical efficacy, without any major safety concerns. However, the CHMP was not bound to follow this advice, and maintained its negative opinion.
In January 2012 AMT announced that the European Commission Standing Committee, which makes the final determination on approvability of novel therapies, requested further information from CHMP, which resulted in a further assessment of Glybera in a limited patient population. In April 2012 AMT announced that the CAT remained positive and that a majority of CHMP voting members were also positive on Glybera. However the CHMP vote was only 16 - 15 in favour and consequently Glybera failed to achieve the level of 17 positive votes required for an approval.
Since originally submitting the MAA, AMT has generated significant additional data, including results from a long-term efficacy study of Glybera showing that improved chylomicron metabolism could be used as a biomarker for increased LPL activity in those patients missing the gene that produces this protein. Data showed that breakdown of chylomicrons produced after meals was greatly and significantly improved at both 14 and 52 weeks following one-time Glybera administration.
It was also shown that Glybera significantly reduces the risk of pancreatitis in LPLD patients. By reducing the incidence of pancreatitis episodes substantially, Glybera has the potential to help "normalize" the day to day lives of patients affected by this disease and prevent the often frequent trips to hospital that patients otherwise experience.
The application will now go back to the European Commission Standing Committee for further consideration.