Institut Pasteur is leading development of the treatment and, along with a supporting research consortium, has contracted Amsterdam Molecular Therapeutics (AMT) to manufacture Phase I and II trial supplies.
To fulfil the €1.8m ($2.3m) deal AMT will perform current good manufacturing practice (cGMP) compliant production of the Sanfilippo B treatment at its 375 m sq facility. The site has sufficient capacity for commercial production of its lead candidate and trial supplies of pipeline products.
Muriel Eliaszewicz, medical director at Institut Pasteur, said: "AMT is one of the only companies in the world that has a proven ability in manufacturing cGMP quality gene therapy products, not only in batches sufficient for clinical development but also in support of a potential regulatory approval.”
In-house activities have given AMT experience of production and regulation of adeno-associated viral (AAV) vectors for gene therapy. Glybera, the lead product at AMT, is now being reviewed by the European Medicines Agency (EMA) for treatment of lipoprotein lipase deficiency (LPLD).
Pipeline product
AMT may choose to bolster its pipeline by acquiring full commercial rights to the Sanfilippo B treatment it is producing on a contract basis. Phase I and II trials are scheduled to begin before 2013 and if these demonstrate proof-of-concept AMT may acquire full commercial rights.
Including the option to acquire the treatment “supports our strategy to build a pipeline of orphan and ultra-orphan indications”, said Jorn Aldag, CEO of AMT. Other pipeline compounds at AMT target hemophilia B, acute intermittent porphyria and Duchenne muscular dystrophy.