Alnylam is an American company whose cornerstone is its interference or interference RNA technology, a way to silence genes and block proteins that cause disease. From the beginning, they have used it to develop orphaned treatments (for rare pathologies). However, they are now investigating to open it to other pathologies such as hypertension or Alzheimer’s, among others. Thus, he tells Its general director in Spain, Sergio Bullónto electionomista.es.
To begin with, how much have you invoiced at 2024?
The company, worldwide, has closed the year with more than 1,000 million billing. Spain is a country important and contributes a small part of this income. We belong to the international part, which covers Europe, Japan and Canada. In total, all that region has obtained 400 million.
¿What percentage of income comes from Spain?
ANDntre the 30 and 40 millionaccording to our estimates.
AND regard to benefit?
Today, Alnylam is a company that does not give benefits. However, we wait and set a goal for five years that it was to become profitable and sustainable at the end of this year. We are doing very well along the way.
“At the end of 2025, the company will be profitable and sustainable, as we set out”
What is the company’s roadmap for this year in the country?
In Spain, we have achieved the financing of four products: two for hereditary amyloidosis by transport, one for the type 1 primary hyperoxaluria and another for acute liver porphyria. The three diseases are rare. At the moment, we have launched these four medications and, last year, we had positive results to expand amvuttra to a new indication: cardiac amyloidosis, which although it remains an orphan pathology, is more prevalent. The road map for this year would be that this new indication obtains regulatory approval at European level and we can work with local authorities to receive their authorization as well.
When would it get to Spain?
At European level, we estimate that it is more or less in the summer. In Spain, we would expect about a year or a year. I think that in 2026 an agreement could be reached with the Ministry of Health. It will also depend on the environment and the new regulation that is in Spain, with new reais decrees and the new drug law.
And the company expects any investment in the country this year?
Alnylam has opted for Spain since 2017 and has made investments, mainly at the level of personnel hiring. The company has been growing until it has around 30 employees. Future investments at the factory storage level and others is not the idea. It is true that we have some agreements with different companies that help us to packaging and others of the product. In Spain, we work with movement, which distributes nationwide.
“Our road map is that the new indication of Amvuttra receives European approval this year”
Before ha mentioned Personnel hiring. In 2025, the template will be expanded?
If this new indication of Amvuttra comes to fruition we hope to start new contracts to cover this new release. How much would it increase? It is a question that we still have no resolved, but I think that at least between 10 and 20%, that is, about five or ten people.
Lthe medications that are marketed here, ¿Where are they manufactured?
All development is carried from the United States. There, we have three floors.
Do you plan to open Some other Factory outsideThe American country?
It will depend on the volume we start working. We dedicate ourselves mainly to rare diseases, which are pathologies of very low prevalence and manufacturing is lower. I think the plan can be to open something out of the United States, but in the long term.
Let’s talk about politics. The Ministry of Health He works in the Royal Decree of Health Technology Evaluation. This aims to reduce the time in which treatments land in Spain. Do you think you will get itn?
We are living a unique moment at the legislative level, but not only in Spain, but also in Europe. The main objective is that all processes be expedited for the patient to receive the treatment in time. Everything that the ministry is doing is in this line and I am optimistic in which it will come to fruition. According to Aelmhu’s report, we are still far from an optimal scenario. Today, we are talking about financing times between 20 and 30 months. It is important that this is expedited. It is true that progress has been made. Last year there were drugs that were approved very quickly, but this has to be embodied in legislation that is optimal for everyone. I believe that health authorities have to understand the complexity that entails investigating and developing drugs in orphan diseases we are talking about a prevalence of five patients per 100,000 inhabitants.
Do you think you would have toBer A Royal Decree only For medicines orphans?
I don’t think it is necessary. Nour vision is that The real decrees have some special wink or politics for orphan medications for this complexity that commented.
“Our vision is that the real decrees have a special wink or politics for orphan medications”
There is also the Plan of the pharmaceutical industry in Spain. ANDn As for rare diseases,qCan you contribute to the name?
Alnylam is a small company, but we are betting on bring clinical trials to Spain. It will allow patients to access innovation in very early stages of research, an increase in knowledge of the disease and the entire clinical development part that will have a positive impact on Spanish centers. The tests of the products that I have commented and of our future medications will be carried out in Spain. We are talking about hundreds of patients and dozens of hospitals who are participating.
¿How many eStudios goesn attract?
The idea is that in 2025 we bring one for hypertension and another for hereditary amyloidosis by transport. At least two should start this year. In addition, it has been proposed that for the second mentioned, between 10 and 15 centers participate.
On the other hand, what are the next releases?
Our main launch is the amvuttra indication expansion. The company also has great programs and is making a great effort in the entire research part. We have studies at the cardiometabolic level, which are advancing at a very good pace. This is for hypertension. It is not a rare disease, but we are going to open our technology to new areas that such as The aforementioned, the central nervous system (Alzheimer’s, Hunginton disease, etc.). We are also trying to reach other fabrics. They are the areas in which the company is working at a slightly preclinical level and others.
Are these clinical trials are performing In Spain?
Not yet. We are talking about preclinical phases and some phases I, but the idea is to continue advancing with them and logically they will arrive in Spain.
“Alnylam is a company that is betting on bringing clinical trials to Spain”
Of all of them, ¿What is the most advanced?
The most advanced is that of hypertension. We are already starting phase III. It is a very ambitious essay in which we have signed an agreement globally with Roche to develop it. In the United States, this company and Alnylam will be marketed, and outside the country the Swiss company. We hope you receive the approval of the authorities between 2029 and 2030 and arrive in Spain.
¿And what other treatments are part of your R&D pipeline?
ANDWe investigate for hepatitis B, but it is not a rare disease. Also everything that opens the possibility of reaching new tissues such as muscle or adipose, we can open the range of new therapies.
¿And they are investigating medications with further companies?
Yes, with Sanofi-Aventis we are collaborating on the issue of hemophilia. They expect the approval of the FDA this year to Fitusiran and in Europe the same. It is an Alnylam product, but an agreement was reached long ago in which they were responsible for all clinical development and marketing. Leqvio also, a drug for the family hypercholesterolemia of Novartis, originally developed it to the American laboratory The Medicine Company, which was acquired by the Switzerland. From that product we receive a series of royalties.
So, his business strategy is to develop medicines and then sign an agreement with some company to finish developing or marketing it
Yes. We do not want to stop innovation, but this requires many resources and investment. Therefore, we reach strategic agreements with different entities to terminate certain clinical development programs. For example, we also have an agreement with Regeneron.
And this 2025 goesn To sign more toSISTS?
I do not know this whole part. The innovation and development team from the United States takes it.
Finally, how can new technologies in the investigation of treatments for rare diseases?
The whole boom of artificial intelligence that we are living right now will have an important role in clinical development. For example, in order to advance faster when choosing therapeutic targets, working on models, knowing better potential diseases or responses to certain medications. I always say that rare disease patients have to touch the lottery three times. The first, to name the pathology it has; the second, that there is a clinical development of a solution; And the third, who has access to a medicine
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