Wednesday, December 21, 2016
Alnylum Pharmaceutical UPDATE
At the request of Desiree Lyon:
Alnylum Pharmaceutical has nearly completed Phase I of the new porphyria drug ALN-AS1 with very promising results. Alnylum specializes in RNA interference (RNAi) treatments which target specific cells that are the root cause of various diseases. Alnylum targets rare diseases that currently have limited treatments, especially blood disorders such as hemophilia and acute porphyria.
Researchers and doctors worldwide agree that porphyria is an extremely rare genetic disease in which the patient does not properly produce heme, an important molecule in blood, which causes a build up of naturally occurring neurotoxins ALA and PBG. These neurotoxins build up in the liver and eventually spill into other areas of the body causing a variety of symptoms including severe abdominal pain, nausea, vomiting, constipation, diarrhea, painful body aches, muscle weakness, paralysis and in some cases psychosis, encephalopathy and death.
ALN-ASI or Givosiran (gi-VOH-sir-an) targets liver cells that are responsible for heme production and the root cause of acute porphyria. By attaching an RNAi to a sugar molecule, Givosiran goes directly to the liver cells where it slows down heme production. Early results show a 74% decrease in ALA and PBG buildup and a 74% decrease of porphyria attacks in patients. There has only been one adverse event reported and that is skin irritation at the injection site. This is a very common occurrence with any injection treatment. Givosiran is injected once every 4 weeks using the same procedure diabetics use to inject insulin. Potentially, Givosiran will be able to be self-administered at home and not require a doctor's appointment.
The results have been so promising, Alnylum believes it can safely bypass Phase II testing and will be seeking this exception from the Food and Drug Administration (FDA) in early 2017 in hopes of starting large scale human trials (Phase III) by the end of 2017.
Alnylum did extensive patient research before developing the drug and discovered how limited current treatments are for porphyria patients. Panhematin has been the most widely used treatment for acute porphyria patients and has been in use for over thirty years. It requires 3 to 11 intravenous treatments over consecutive days during acute attacks or can be used prophylactic requiring patients to receive the treatment anywhere from once a month to twice a week. Treatments take up to an hour and usually require patients to have an intravenous access port surgically installed. It is extremely exciting to see a company take a modern medicine approach to treat a disease that affects nearly 5,000 patients worldwide.
Alnylum believes Givosiran has the potential to greatly reduce or eliminate the rarely spoken about chronic aspects of porphyria, the frequency and severity of attacks, as well as reducing the risk of liver cancer which is common in long time sufferers of porphyria.
The American Porphyria Foundation (APF) has played an important role in helping Alnylum develop Givosiran. By creating the Porphyria Consortium, Alnylum was able to move quickly through the research and expert recruitment phase of the drug development. The APF has assisted Alnylum recruit patients as well as asymptomatic high excreters of ALA and PBG who volunteered for Phase I. In addition, The APF has sent porphyria patients to meet with the development teams at Alnylum to provide insight into battling porphyria on a daily basis as well as providing advice to help recruit patients for Phase III and address concerns they may have. Finally, the APF will play a critical role in assisting the recruitment of the more than 100 patients worldwide to participate in Phase III.
Emergency Room Guidelines for Acute Porphyrias http://www.porphyriafoundation.com/sites/default/files/ERGuidelinesAcutePorphyria%201.pdf ...
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