Alnylam’s RNAi Product Patisiran Has More Positive News

The Market

The Stock Market is Still under pressure, which is frightening common investors, pressuring them to sell. We see no real reasons for the stock market to be acting as a Bear when the criteria of bullishness are obvious and several. We see no need for us to be scared, overreact and unwillingly contribute to the selloff.

In the News


Alnylam (ALNY) announced positive results from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for hereditary ATTR (hATTR) amyloidosis.

According to Eric Green, Vice President and General Manager of TTR Program at Alnylam, the exploratory analysis of the Phase 3 APPOLO results demonstrated a significant decrease in the composite rate of all-cause hospitalization and mortality in patients receiving patisiran compared to placebo.

These results, along with previously presented APOLLO data, which has demonstrated a successful Patisiran’s halting or reversal of neuropathy progression in most patients cement the evidence that patisiran, if approved, will have the potential to be a transformative treatment for patients with all forms of hereditary ATTR amyloidosis.

Analysis of the results revealed a 50 percent decrease in the composite rate of all-cause hospitalization and mortality over 18 months in patisiran-treated patients, relative to placebo. A similar finding was also observed regarding cardiac hospitalization and all-cause of mortality, showing an approximately 45 percent decrease with patisiran, relative to placebo.

There were significant improvements in disability, gait speed, autonomic neuropathy symptoms, and overall quality of life are observed as early as nine months.

The most commonly reported frequent adverse events (AEs) were peripheral edema and infusion-related reactions (IRRs) and were generally mild to moderate in severity.

Overall, there were 13 deaths in the APOLLO study; none were considered related to study drug and the frequency of deaths was lower in the patisiran group (4.7 percent) as compared with placebo (7.8 percent).

Adverse Events (AEs) leading to treatment discontinuation were lower in patisiran-treated patients (4.7 percent) compared with placebo-treated patients (14.3 percent).

For more detailed information about the results, you can read the firm’s press release.  

The beneficial results support the rationale for early treatment with patisiran. The early treatment is expected to potentially halt or improve neuropathy progression or impairment, respectively.

Today, April 25, 2018, detailed results regarding patisiran’s effect on quality of life will also be presented in a separate oral presentation.


Patisiran is an investigational, intravenously administered RNAi therapeutic targeting transthyretin (TTR) being developed for hereditary ATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made.


Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is a hereditary progressive debilitating, and sometimes fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.

hATTR amyloidosis represents a major unmet medical need.  The disease affects around 50,000 people worldwide. The median survival is 4.7 years following diagnosiswith a reduced survival (3.4 years) for patients presenting with cardiomyopathy.

Currently, the only available treatment options for the early-stage disease are liver transplantation and, in some countries, tafamidis (approved in Europe, Japan, and certain countries in Latin America, specific indication varies by region). As such, there is a significant need for novel therapeutics to help treat patients with hATTR amyloidosis.

Prohost Observations

Patisiran is currently under Priority Review as a Breakthrough Therapy with the FDA and under accelerated assessment by the European Medicines Agency (EMA) for hATTR amyloidosis. The FDA has set a PDUFA date of August 11, 2018.

The safety and efficacy of patisiran have not been evaluated by the FDA, the EMA or any other health authority.

Although we did lower our fair assessment of Alnylam’s value, the firm is expected to put on the market products for genetic diseases that have yet to be treated as no product could reach the root cause of the diseases, the genes. RNAi blocks the messenger RNA (mRNA) before its message results in the production of the protein that causes the progressive debilitating and deadly diseases.

Soon, some of Alnylam’s products will be granted approvals and will be marketed and sold before other competing technologies such as gene therapies or gene editing would create drugs that would compete with the RNAi approach and challenge the antisense procedure.

We will continue to follow up on Alnylam’s products and act on time upon whatever we sense is in favor of boosting the stock or in favor of knocking it down.

Again, we say, the Biotechnology sector is speeding up its revolution. As a results, we will witness more and more products that improve on the currently existing drugs. We will also witness novel created products for diseases that have no treatment yet on the drug market.

Now, ALNY is trading at around $95. Our new 1st target is $100.  

Prohost Forward-Looking: Material presented here is for informational purposes only. Nothing in this article should be taken as a solicitation to purchase or sell securities. Before buying or selling any stock you should do your own research and reach your own conclusion. Further, these are our ‘opinions’ and we may be wrong. We may have positions in securities mentioned in this article. You should take this into consideration before acting on any advice given in this article. If this makes you uncomfortable, then do not listen to our thoughts and opinions. The contents of this article do not take into consideration your individual investment objectives. Also, investing in stocks includes certain risks. Please consult with your own financial adviser before making an investment.

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