Indeed, we are in the future as we reiterated time and time again based on our biotech firms advancement on the road towards treating diseases at their root origin and personalized basis. Great news from Alnylam’s (ALNY) has come announcing positive top-line results from its ongoing Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting the transthyretin (TTR) gene for the treatment of TTR-mediated amyloidosis (ATTR). The RNAi drug achieved statistically significant (p<0.01) knockdown of serum TTR protein levels of greater than 80% in healthy volunteer subjects, in line with results for ALN-TTRsc previously reported in non-human primates.
While we were on the verge of posting an article about Halzyme Therapeutics (HALO), whose technology enables the transformation of intravenously administered drugs Into subcutaneous drugs, we learned that news we were waiting for about results from Alnylam’s subcutaneously transformed drug ALN-TTRsc will be soon announced. We decided to wait for Alnylam’s drug results before we post the HALO article, maybe we could bring a more convincing argument about the virtue of subcutaneous transformation of drugs with Halozyme’s proprietary drug delivery technology. Indeed, Alnylam’s GalNAc-siRNA SC technology has been the primary cause for the success of its RNAi drug and has become ALNY’S primary approach for development of RNAi therapeutics.
The study results demonstrated ALN-TTRsc was able to induce a statistically significant knockdown of serum TTR to levels greater than 80% in treated subjects, results that are in line with the previous non-human primate experience. Alnylam’s drug data are very meaningful not only for the continued advancement of ALN-TTRsc, but also for the continued execution on "Alnylam’s entire 5x15" product strategy.
In addition to offering proof of concept of Alnylam’s technological capability to turn TNAi drugs into safe and effective therapeutics inducing their action at the root-cause of diseases, the results brought hope for the first time to patients suffering from ATTR. This inherited disease is progressively debilitating, and fatal. It is caused by mutations in the TTR gene that produces the culprit TTR protein primarily in the liver.
The mutations of the TTR gene causes the accumulation of abnormal amyloid proteins that damage body organs and tissue, including peripheral nerves and heart, resulting in peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy - morbid conditions with significant mortality. familial amyloidotic polyneuropathy (FAP) patients have a life expectancy of 5 to 15 years from symptom onset. Currently, the only treatment options for early stage FAP disease are liver transplantation and tafamidis (approved in Europe). The mean survival for familial amyloidotic cardiomyopathy (FAC) patients is approximately 2.5 years, and there are no approved therapies. There is a significant need for novel therapeutics to treat patients who have inherited mutations in the TTR gene.
Alnylam is hosting an R&D Day today from 8:30 a.m. – 12:00 p.m. ET at the Sofitel New York in New York City. Alnylam scientists and management are reviewing the progress with the company’s “Alnylam 5×15” product strategy for the development of RNAi therapeutics. The event will be webcast live on the News & Investors section of the company’s website. An audio replay of the event will be available on the Alnylam website approximately 90 minutes after the event.
RNA Interference (RNAi) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells. It is a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
We Congratulate the patients, the physicians who will be able to give hope to patients that had none of it, to Alnylam’s for its continued efforts to push science faster and faster into the future and to the investors who deserve to enjoy the fruits of their investment in an excellent achieving biotech. Their stock is now trading at 43.42 UP 5.82 (15.48%)
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