June 21, 2024

Wall Street eyes Alnylam’s RNAi therapeutics potential By Investing.com

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Alnylam Pharmaceuticals, a leader in the biopharmaceutical industry, has been making significant strides in the development of RNA interference (RNAi) therapeutics. With a series of clinical trials and strategic partnerships underway, the company stands at the forefront of a potentially transformative era in the treatment of serious diseases.

Company Overview

Alnylam, known for its focus on RNAi therapeutics, has four FDA/EMA-approved drugs and a pipeline rich with potential. The company’s efforts are geared towards rare diseases, with a keen interest in conditions such as transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM) and hypertension.

Clinical Trials and Pipeline Developments

Analysts are closely monitoring Alnylam’s Phase III HELIOS-B study on AMVUTTRA for ATTR-CM, with top-line data expected in early 2024. This follows the positive Phase II KARDIA-1 study results, which demonstrated zilebesiran’s efficacy in significantly reducing systolic blood pressure. The Phase II KARDIA-2 data, examining zilebesiran combined with standard hypertension medication, is also highly anticipated in the first half of 2024. Additionally, plans to initiate the KARDIA-3 study in mid-2024 targeting higher-risk patients are underway.

Alnylam’s collaboration with Roche has been a boon, bringing in a $310 million upfront payment and potential milestones up to $2.8 billion, plus profit-sharing in the U.S. This partnership, along with one with Regeneron (NASDAQ:), is set to bolster Alnylam’s pipeline, which is expected to see 9 wholly-owned and 6 partnered Investigational New Drug (IND) filings by the end of 2025.

Financial Health

The company’s financial position is robust, with estimated pro forma cash of $2.51 billion and debt of $1.02 billion. Revenue estimates for 2023 have been increased from $1,412 million to $1,773 million, with EPS estimates for 2024 showing a positive trend from a loss in 2023 to earnings in 2024.

Market Position and Competitive Landscape

Alnylam’s competitive edge lies in its RNAi technology, which has shown potential across multiple therapeutic areas. The company’s once-yearly subcutaneous regimen for ATTR-CM with ALN-TTRsc04 could offer a competitive advantage, and its expansion into obesity and oncology markets could drive future revenue growth.

Bear Case

What are the risks facing Alnylam Pharmaceuticals?

The biopharmaceutical sector is inherently risky, with clinical trials and regulatory approval processes presenting significant hurdles. Alnylam faces competition for similar treatments, and any clinical or regulatory setbacks could impact its stock value. Revenue figures have fallen short of estimates, and uncertainties, such as the Complete Response Letter (CRL) for Onpattro in ATTR-CM, have posed challenges to the company’s ATTR franchise.

Could Alnylam’s debt affect its future growth?

While Alnylam’s cash reserves are solid, the management of its debt levels relative to these reserves will be crucial. The company’s strategic investments and pipeline development must be balanced against the need to maintain financial stability and manage liabilities effectively.

Bull Case

How could upcoming clinical data releases affect Alnylam’s stock value?

Positive outcomes from upcoming clinical trials, such as the HELIOS-B study and KARDIA-2 data, could significantly boost Alnylam’s stock value. The potential for an sNDA filing in mid-2024 to expand treatment access for AMVUTTRA in ATTR-CM is a particularly noteworthy catalyst.

What strategic partnerships could enhance Alnylam’s pipeline development?

Alnylam’s partnerships with industry giants like Roche and Regeneron are expected to enhance pipeline development and provide financial support. These collaborations validate Alnylam’s technology and could lead to further strategic alliances, bolstering the company’s position in the market.

SWOT Analysis


  • Advanced RNAi technology with multiple therapeutic applications.
  • Strong pipeline with potential IND filings indicating future growth.
  • Strategic partnerships providing financial and technological support.


  • Risks associated with clinical trials and regulatory processes.
  • Competition in the biopharmaceutical sector for RNAi therapeutics.
  • Management of debt in relation to cash reserves.


  • Upcoming clinical data releases could prove to be significant stock catalysts.
  • Expansion into new therapeutic areas like oncology and obesity.
  • Potential for pipeline drugs to capture substantial market share.


  • Clinical or regulatory setbacks could negatively impact stock value and investor confidence.
  • Market penetration challenges and pricing pressures.
  • Uncertainties surrounding patent expirations and market exclusivity.

Analysts Targets

  • Piper Sandler: Overweight rating, price target of $210.00 (November 30, 2023).
  • BMO Capital Markets: Outperform rating, price target of $234.00 (January 2, 2024).
  • H.C. Wainwright & Co: Buy rating, price target of $395.00 (November 6, 2023).
  • Cantor Fitzgerald: Neutral rating, price target of $165.00 (December 14, 2023).
  • Barclays Capital Inc.: Overweight rating, price target of $236.00 (December 14, 2023).

The timeframe for the data used in this article ranges from November to January 2024.

InvestingPro Insights

As Alnylam Pharmaceuticals continues to innovate in the realm of RNA interference therapeutics, investors and analysts are keeping a keen eye on the company’s financial health and market performance. Here are some insights based on real-time data from InvestingPro that could provide additional context for those following the biopharmaceutical leader.

The company’s market capitalization stands at a robust $23.09 billion, reflecting investor confidence in its long-term strategy and pipeline potential. However, it’s important to note that Alnylam is not expected to be profitable this year, with a reported P/E ratio of -44.89 and an adjusted P/E ratio for the last twelve months as of Q3 2023 at -45.9. This aligns with analyst expectations and underscores the fact that the company is currently prioritizing growth and development over immediate profitability.

Despite this, Alnylam demonstrates financial prudence with its liquid assets exceeding short-term obligations and operating with a moderate level of debt. This suggests that the company is well-positioned to manage its financial commitments while continuing to invest in its promising pipeline of RNAi therapeutics.

Revenue growth figures are particularly striking, with a 79.37% increase over the last twelve months as of Q3 2023, and an even more impressive quarterly revenue growth of 183.96% in Q3 2023. This indicates that Alnylam is effectively monetizing its approved drugs and advancing its pipeline, which could translate into long-term value for shareholders.

For those considering a deeper dive into Alnylam’s financials and market prospects, InvestingPro offers a comprehensive suite of tools and additional InvestingPro Tips. Subscribers can access more than five additional tips that may provide further guidance on investment decisions. Currently, InvestingPro subscription is on a special New Year sale with a discount of up to 50%. To take advantage of this offer, use coupon code SFY24 to get an additional 10% off a 2-year InvestingPro+ subscription, or SFY241 to get an additional 10% off a 1-year InvestingPro+ subscription.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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