2021-01-12| R&D

Alnylam Aims to Obtain “Top 5 Biotech” Tag By 2025

by Ruchi Jhonsa
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After delivering a stellar performance last year, Alnylam, a leading RNAi therapeutics company, is now eyeing a position in the top 5 biotech companies in market cap by 2025. At the 39th Annual JP Morgan Healthcare Conference, the company announced a 5-year strategy dubbed Alnylam P5x25, which is aimed at driving sustainable innovation of drugs for rare and common diseases and reaping exceptional financial benefits for the company.

The 5 Ps of the strategy are patients, products, pipeline, performance, and profitability. As part of the strategy, the company is looking forward to catering to over half a million patients with 6 or more marketed products in rare and prevalent diseases. It also plans to launch additional 8 clinical programs and file 4 or more regulatory submissions per year. With these many additional clinical programs and products in the pipeline, the company expects to see a 40% or higher revenue growth. Alnylam strongly believes this plan will help it achieve sustainable non-GAAP profitability in the next 5 years.


FDA Approved RNAi Therapeutics

Since its inception 19 years back, Alnylam has successfully launched four products in the market in just over 2 years. In 2018, the company secured the USFDA approval for Onpattro, followed by the second approval for Givlaari in 2019 and the third one for Oxlumo just a month back. “These products are or are expected to become market-leading products for many years to come,” said John Maragnore, CEO of Alnylam.

In terms of revenue, Onpattro, an RNAi drug for the treatment of ATTR amyloidosis, leads the pack. The drug generated a whopping $306 million during pandemic-representing 80% growth from 2019. The second to bag approval, Givlaari, which is used for treating rare liver disease, acute hepatic porphyria, generated $55 million last year. For the most recent approval, Oxlumo, the company has already started seeing strong early demand and generated sales worth $300,000 in 2020.

Besides the three FDA-approved drugs, the company is getting its hopes high on the fourth medication, vutrisiran, which is nearing regulatory submission in the US. The company believes that vutrisiran, the company’s second ATTR amyloidosis medication, can significantly grow their opportunity beyond Onpattro, especially in people with mixed ATTR phenotype.

Excited about the company’s progress, John Maragnore notes, “In addition to our four currently marketed products, we can expect two additional product launches over the next 12 to 24 months, meaningfully increasing the number of revenue-generating assets, all of which are in a growth phase for the foreseeable future. We believe this profile of organic growth generation of commercial medicines is nearly unprecedented in biotech history, and we expect to be positioned to expand on this profile in the years to come.”


Extension of Drug Labels

Maraganore pointed out that one way by which they are planning to achieve their 2025 goal is by extending the labels of the existing medications. Onpattro is currently approved for hATTR patients with polyneuropathy. But it is being tested in ATTR patients who predominantly present with cardiomyopathy (CM) symptoms (APOLLO-B study). Following the completion of the trials, Onpattro is expected to be available for ATTR-CM patients. Besides, vutrisiran is also being tested in ATTR patients with cardiomyopathy. Oxlumo, which recently got the FDA approval, will be extended to severe cases of PH1 disease.


Sustainable Innovation

Alnylam has a strong organic product engine, which is the source of sustainable innovation. Around 25 preclinical programs are running at the moment that spans rare and common diseases such as hypertension, gout, and diabetes. Alnylam envisions that RNAi therapeutics, with their durable effects and infrequent dosing, will be the future of common and rare diseases. This will help make the company self-sustainable and reduce the need to tap external funding sources for driving the projects.

“Because of the Alnylam platform we built, the opportunities for the future are plentiful, if not boundless by drug discovery standards, and include leveraging our human genetics capabilities for enhanced success rates, concrete delivery toward new tissues like tumors for RNA therapeutics and cancer novel combinations with monoclonal antibodies, creating market disruptive therapies, dual targeting bis-RNAi technology to treat diseases in fundamentally new ways, controllable pharmacology with our Reversir technology and alternative, less invasive modes of delivery like Oral or trans blood-brain barrier approaches. So I think you can expect sustainable and disruptive innovation from Alnylam for many years to come,” said Maraganore.

Related Article: Alnylam Nears Fourth Approval with Successful Trial Data for RNAi Drug



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