A leader in the development of RNAi therapeutics, Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) will present the outcome of the open label extension studies of the drug givosiran in phase 1 as well as phase 1/ 2 trials at the 53rd Annual Global Liver Congress. The summit will be held in Paris during April 11 and 15 2018. The collaborators and scientists of Alnylam will show case the findings of the innovative drug in clinical trials involving patients suffering from acute hepatic porphyrias (ACP).
A brief outline of the presentation at the European Association to Study the Liver on April 14 2018, is given below:
An oral presentation of phase 1/ 2 placebo controlled/ randomized and an open label extension study of an investigational RNAi – Givosiran in patients with ACP.
Exploring the natural history of patients with ACP worldwide with repeated attacks.
An insight into how ACP affects the work life and quality life of the patients.
A deep insight into the utilization of health-care for people suffering from ACP in the EU and the US.
The lead authors of the above presentation are DM Bissell, M Balwani, L Gouya, J Bloomer,P Harper, and E Sardh.
An investigational RNAi therapeutic – Givosiran is mainly targeted at the liver ALAS1 mRNA. It will be administered to the patients through a subcutaneous injection to cure ACP. According to the study, the patents receiving this therapy monthly will be able to reduce induced liver ALAS1 levels gradually and bring down the porphobilinogen and aminolevulinic acid to almost normal levels.
Therefore, patients receiving Givosiran therapy at the prescribed levels will be able to get safeguarded from life threatening and severe attacks and mitigate chronic symptoms. The innovative formulation utilizes the ESC-GalNAc conjugate technology of Alnylam to provide durable and potency levels.
The US FDA has given the breakthrough therapy designation for the drug. EMA has accorded the prime designation for the innovative drug. The effectiveness and safety of the drug will be evaluated in Phase 3 trial.
Alnylam Pharmaceuticals, Inc. Showcases the Clinical Outcome of Patisiran
The company will exhibit the results of Apollo phase 3 trial for the Patisiran drug at 16th ISA during March 26 and 29, 2018. The summit will be held in Komato Japan to explain the effectiveness of Patisuran to cure hereditary ATTR (hATTR) amyloidosis.