Following recent reports of new onset or worsening peripheral neuropathy in patients with ATTR amyloidosis with cardiomyopathy (hATTR-CM) treated with Alnylam’s (ALNY) RNAi –based drug revusiran, The firm requested that the Data Monitoring Committee (DMC) review the reports and the Clinical trial data on an unblinded basis. After reviewing the reports, the DMC advised suspending the dosing of revusiran.
However, the DMC stated that It did not find conclusive evidence for a drug-related neuropathy signal in the trial. It informed the Company, though, that the benefit-risk profile for revusiran no longer supports continued dosing.
Alnylam chose to discontinue revusiran’s development while actively monitoring patients across revusiran studies to ensure their safety and to further evaluate ENDEAVOUR data to understand the potential cause of these findings.
Alnylam tried to explain the reasons why observers, including investors should not consider this negative news should suggest that the firm’s technology is not safe and so are its pipeline products. As expected, though, frightened investors were not ready to listen. They raced into selling the stock, causing its tumbling.
Prohost Observations
We carefully listened to what Alnylam had to say and we will tell it all. But first, we intend to express our feeling that the worst losers from ending the development of revusiran are the sufferer from hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM) who, unfortunately will remain with no effective treatments for a deadly hereditary disease.
Here is what those who caused the selloff didn’t want to hear or if they heard, they did not want to believe.
The Data Monitoring Committee’s Announcement:
There is no conclusive evidence for revusiran-related neuropathy in the trial.
Alnylam’s Following Statements:
– The decision to discontinue revusiran’s does not affect our other drugs, including patisiran, which is currently in Phase 3 development for another condition known as hATTR amyloidosis with polyneuropathy (hATTR-PN). Patisiran utilizes a lipid nanoparticle delivery formulation.
– All other clinical programs in Alnylam’s pipeline use Enhanced Stabilization Chemistry (ESC) GalNAc delivery technology, while the stopped product revusiran uses Standard Template Chemistry (STC) GalNAc delivery technology.
– The decision does not affect any of of the other RNA interference (RNAi) programs in Alnylam’s pipeline.
– Based on a current assessment of the safety data across the Alnylam’s other programs, no evidence of drug-related neuropathy signal have shown in over 800 treated subjects and patients with exposure of up to 34 months. This observation applies also to the ALN-PCSsc program partnered with The Medicines Company.
– The Company reaffirmed its “Alnylam 2020” guidance and remains committed to the advancement of these investigational RNAi therapeutics for treatment of diseases with high unmet medical need.
In summary, the origin of the problems that occurred with revusiran treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM) is not yet figured out.
Having said that, we have to also understand investors’ rush to sell the stock. It is obvious that it is extremely difficult to convince investors not to panic when bad news comes from a product in clinical trial by a company that has no marketed products and its products are based on a novel difficult technology.
Bottom line, betting on a novel product expected to conquer a killer disease that has yet to find safe and effective treatment requires insurance. In the stock market. This this kind of insurance is obtained through placing a stop-loss order that would limit the losses in a security.
Those who are condemning Alnylam, or offering any other opinion do not yet have any proof that would back their negative, neutral, or positive opinion. So, rushing into accumulating the stock on weakness in this condition is premature. It is better if we take our time and follow up on the firm’s news. It would be great if we can still place a stop-loss order.
For those who ask whether IONS would be affected, our answer is NO when it comes to Science, and YES, but for a few days, when it comes to daily traders.
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 so consult with your own financial adviser before making an investment decision. Investing includes certain risks including loss of principal.
News & Comments
October 6, 2016
Alnylam: Some Acted Without Listening and Some Listened But Did Not Believe
Following recent reports of new onset or worsening peripheral neuropathy in patients with ATTR amyloidosis with cardiomyopathy (hATTR-CM) treated with Alnylam’s (ALNY) RNAi –based drug revusiran, The firm requested that the Data Monitoring Committee (DMC) review the reports and the Clinical trial data on an unblinded basis. After reviewing the reports, the DMC advised suspending the dosing of revusiran.
However, the DMC stated that It did not find conclusive evidence for a drug-related neuropathy signal in the trial. It informed the Company, though, that the benefit-risk profile for revusiran no longer supports continued dosing.
Alnylam chose to discontinue revusiran’s development while actively monitoring patients across revusiran studies to ensure their safety and to further evaluate ENDEAVOUR data to understand the potential cause of these findings.
Alnylam tried to explain the reasons why observers, including investors should not consider this negative news should suggest that the firm’s technology is not safe and so are its pipeline products. As expected, though, frightened investors were not ready to listen. They raced into selling the stock, causing its tumbling.
Prohost Observations
We carefully listened to what Alnylam had to say and we will tell it all. But first, we intend to express our feeling that the worst losers from ending the development of revusiran are the sufferer from hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM) who, unfortunately will remain with no effective treatments for a deadly hereditary disease.
Here is what those who caused the selloff didn’t want to hear or if they heard, they did not want to believe.
The Data Monitoring Committee’s Announcement:
There is no conclusive evidence for revusiran-related neuropathy in the trial.
Alnylam’s Following Statements:
– The decision to discontinue revusiran’s does not affect our other drugs, including patisiran, which is currently in Phase 3 development for another condition known as hATTR amyloidosis with polyneuropathy (hATTR-PN). Patisiran utilizes a lipid nanoparticle delivery formulation.
– All other clinical programs in Alnylam’s pipeline use Enhanced Stabilization Chemistry (ESC) GalNAc delivery technology, while the stopped product revusiran uses Standard Template Chemistry (STC) GalNAc delivery technology.
– The decision does not affect any of of the other RNA interference (RNAi) programs in Alnylam’s pipeline.
– Based on a current assessment of the safety data across the Alnylam’s other programs, no evidence of drug-related neuropathy signal have shown in over 800 treated subjects and patients with exposure of up to 34 months. This observation applies also to the ALN-PCSsc program partnered with The Medicines Company.
– The Company reaffirmed its “Alnylam 2020” guidance and remains committed to the advancement of these investigational RNAi therapeutics for treatment of diseases with high unmet medical need.
In summary, the origin of the problems that occurred with revusiran treatment of hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM) is not yet figured out.
Having said that, we have to also understand investors’ rush to sell the stock. It is obvious that it is extremely difficult to convince investors not to panic when bad news comes from a product in clinical trial by a company that has no marketed products and its products are based on a novel difficult technology.
Bottom line, betting on a novel product expected to conquer a killer disease that has yet to find safe and effective treatment requires insurance. In the stock market. This this kind of insurance is obtained through placing a stop-loss order that would limit the losses in a security.
Those who are condemning Alnylam, or offering any other opinion do not yet have any proof that would back their negative, neutral, or positive opinion. So, rushing into accumulating the stock on weakness in this condition is premature. It is better if we take our time and follow up on the firm’s news. It would be great if we can still place a stop-loss order.
For those who ask whether IONS would be affected, our answer is NO when it comes to Science, and YES, but for a few days, when it comes to daily traders.
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 so consult with your own financial adviser before making an investment decision. Investing includes certain risks including loss of principal.
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