Amsterdam, The Netherlands, 7 September, 2012 – uniQure B.V., a leader in the field of human gene therapy, today provided an update on its half year trading for the period to June 30, 2012.
Jörn Aldag, CEO of uniQure, commented: “We believe that the gene therapy business of uniQure is now well placed to deliver on the significant value which we believe has been built up over recent years. The validation of Glybera as the first gene therapy product to pass the CHMP assessment underpins the expertise developed at uniQure and the value of the other programs in our pipeline. We are extremely excited by this significant success.”
Following the reorganisation at the end of 2011, and the corporate reorganisation to transfer the business and assets to uniQure as a new entity from the old AMT, the business is now well positioned to take advantage of the opportunities arising from the positive opinion of the CHMP on Glybera. The company will address the challenges arising from the enforced operational reductions which were implemented at the end of 2011. uniQure is now evaluating the options for commercialising Glybera, and making it available to patients.
Separately, the Hemophilia B program continues to make strong progress following the very encouraging publication at the end of 2011 in the New England Journal of Medicine.
The remaining pipeline assets have also performed strongly and each of our Porphyria, Sanfilippo B and Parkinson’s disease programs are expected to enter clinical trials within the coming 12 months.
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.
The Marketing Authorisation Application (MAA) for Glybera was submitted in December 2009, and in July 2012 the CHMP issued its positive opinion.
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 to the European Commission Standing Committee for endorsement, which represents the final step in the approval process. It is encouraging that already many doctors and patients are actively seeking access to Glybera after hearing fo the approval.
During the period until the transfer to uniQure, AMT took steps to bring in non-dilutive financing to cover some or all of the costs associated with its remaining programs, in order to reduce its cash expenditure.
AMT continued to work with St Jude’s Children’s Hospital in the USA, which is currently financing and conducting a clinical study in US and UK. Initial results are promising, with patients showing stable and persistent expression of the Factor IX clotting protein, and able to reduce or stop their administrations of protein replacement therapy, which is the current standard of care and requires intravenous infusion up to three times per week.
By contrast, the hemophilia B gene therapy requires a single administration to provide lasting benefit – the earliest patient was treated almost 24 months ago and so far has shown no detectable lessening of the benefit from this treatment. This is the second gene therapy program that AMT was involved with to show clinical benefit from a single treatment and established AMT as the leading gene therapy company worldwide. The initial results of the study were described in the New England Journal of Medicine in December 2011.
Acute Intermittent Porphyria
This program, in collaboration with the University of Navarra and Digna Biotech in Spain, is making encouraging progress. Observational studies have been underway for several months, and the first patients are expected to be enrolled for treatment in a clinical study in the second half of 2012. The majority of expenditure at this time for this program is covered by an EU grant.
uniQure secured a collaborative agreement with two leading neurology experts to develop further a gene therapy incorporating uniQure’s GDNF (glial cell derived neurotrophic factor) gene for the treatment of Parkinson’s disease.
Professor Krystof Bankiewicz at the University of California, San Francisco (UCSF), a world expert in GDNF gene therapy, and Professor Howard Federoff of Georgetown University, a preeminent physician-neuroscientist, have developed a product approved to start clinical trials in the U.S. using uniQure’s GDNF gene incorporated into an adeno-associated virus-2 (AAV-2) delivery vector. The GDNF gene contains the information to produce a protein necessary for the development and survival of nerve cells. The positive effect of GDNF on nerve cells has already been demonstrated in early research by uniQure in collaboration with the University of Lund, Sweden.
UCSF entered into a collaboration with Dr. Russell Lonser, neurosurgeon and Chief of the Neurosurgical Branch of the NINDS, a division of the National Institutes of Health, to commence a Phase I study of the gene therapy in patients with Parkinson’s disease. Patient enrollment is expected to begin mid-2012. Collaborating on the study will be Drs. Krystof Bankiewicz of UCSF, Howard Federoff of Georgetown University and NINDS co-investigator neurologists Drs. Mark Hallett and Walter Koroshetz.
Under an agreement signed at the beginning of 2011, uniQure is collaborating with a consortium led by Institut Pasteur in the clinical development of a novel gene therapy to treat Sanfilippo B. This rare genetic disease affecting new-born children leads to progressive neuronal degeneration and death. There is no approved therapy currently available.
On behalf of the Consortium, Institut Pasteur will lead the development program and will also sponsor the initial Phase I/II clinical study. uniQure, has taken on manufacturing and supplying the adeno-associated virus, serotype 5 (AAV5) gene therapy product to the Consortium. The Phase I/II clinical study is scheduled to begin in the first half of 2013.
Other Research and Development
AMT demonstrated the advantage of its AAV vector delivery technology for the efficient delivery of short and micro RNA to inhibit disease by RNA interference in two further pre-clinical disease models, for hypercholesterolemia and Huntington’s disease. RNAi-based therapeutic strategies are considered highly promising in the industry, but so far, effective delivery has been elusive. Two other important research projects are intended to greatly enhance the value of the platform developed by AMT: gene expression control and re-administration.
At the EGM held on Friday July 20, 2012 shareholders approved the appointments of Mr. Ferdinand Verdonck as a Director (Supverisory Board member B) and Chairman of the Supervisory Board, and Mr. Edwin de Graaf as a Director (Supervisory Board member A).Outlook
The positive success of Glybera supports the company’s underlying value. The original business plan announced at the time of the restructuring and transfer of the business to uniQure excluded Glybera. The further progression of Glybera to full commercialization will require additional investment and the company is currently evaluating its options and the anticipated investment required. This investment may be financed through partnerships (either on Glybera or Hemophilia B) or through additional investment. .For further enquiries:
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