Top 5 PCSK9 inhibitor stocks to watch in 2025: From injectables to gene editing

The global market for PCSK9 inhibitors is undergoing a significant transformation, with both established biologics and next-generation oral and gene-editing therapies poised to reshape the cholesterol-lowering landscape. As cardiovascular disease continues to be the leading cause of death globally, the demand for effective lipid-lowering drugs remains urgent. With long-standing barriers around adherence to injectable therapies, newer entrants in the space—including oral peptides and siRNA-based approaches—are beginning to capture both market share and investor attention.

In this feature, we examine five publicly traded companies—Amgen Inc. (NASDAQ: AMGN), Regeneron Pharmaceuticals Inc. (NASDAQ: REGN) in partnership with Sanofi S.A. (NASDAQ: SNY), Merck & Co., Inc. (NYSE: MRK), Novartis AG (NYSE: NVS), and Verve Therapeutics Inc. (NASDAQ: VERV)—to assess their positioning, pipeline momentum, and investor sentiment heading into the second half of 2025.

What is the status of Amgen and next steps for Repatha?

Amgen Inc. remains the legacy leader in the PCSK9 class with Repatha (evolocumab), the first-in-class monoclonal antibody approved in 2015. Repatha delivered robust sales in 2024, generating $606 million in fourth-quarter revenue and achieving a 45 percent year-over-year growth rate. Full-year sales rose 36 percent, signaling continued physician adoption across high-risk cardiovascular patients.

However, legal risks have recently surfaced. A U.S. jury ordered Amgen to pay $406 million in an antitrust case related to bundling practices involving Repatha and other Amgen products. The verdict, which Amgen is appealing, could result in increased scrutiny over pricing and formulary placement strategies. Despite this challenge, analysts remain bullish on Repatha’s continued revenue contribution through at least 2027, given its entrenched position in clinical guidelines and widespread global formulary access.

How are Regeneron and Sanofi positioning Praluent amid legal volatility?

Regeneron Pharmaceuticals and Sanofi co-developed and market Praluent (alirocumab), another injectable monoclonal antibody PCSK9 inhibitor. Praluent has often been overshadowed by Repatha in terms of sales, with estimated annual revenues of $241 million in 2024. However, the recent dismissal of Amgen’s patent enforcement case in Düsseldorf removes a longstanding legal hurdle, potentially paving the way for a stronger European and global pricing strategy.

Both Sanofi and Regeneron have been repositioning Praluent in higher-risk patient categories, including familial hypercholesterolemia and patients with established ASCVD. While the product’s market share remains limited, especially in the United States, analysts believe the newfound legal clarity and targeted pricing strategies could allow Praluent to gain share incrementally in regions where payer resistance to biologics remains strong.

Why Merck’s enlicitide may redefine the oral PCSK9 category

Merck & Co., Inc. has quickly emerged as the most closely watched PCSK9 disruptor due to the development of enlicitide (MK-0616), a first-in-class oral macrocyclic peptide targeting the PCSK9 protein. In June 2025, Merck announced positive topline data from two pivotal Phase 3 trials—CORALreef HeFH and CORALreef AddOn—which evaluated enlicitide’s safety and efficacy in both familial hypercholesterolemia and broader hyperlipidemia populations.

The trials met their primary and secondary endpoints, demonstrating LDL-C reductions in the 50–60 percent range, which rival those seen with injectable monoclonal antibodies. This oral format, requiring once-daily dosing, could revolutionize patient adherence and eliminate a key barrier that has historically limited PCSK9 uptake.

Institutional sentiment has grown more favorable following these trial readouts. Merck stock rose nearly one percent after the announcement, with analysts at Jefferies and Morgan Stanley issuing updated notes calling enlicitide “a pipeline-defining asset” and projecting peak global sales between $4 and $6 billion if approved. Regulatory filings are expected by early 2026, and Merck has already begun infrastructure preparations for a large-scale launch targeting primary care and cardiologist segments.

What makes Novartis’s inclisiran a compelling siRNA disruptor?

Novartis AG has taken a differentiated approach to PCSK9 inhibition with inclisiran, a small-interfering RNA (siRNA) therapy approved under the brand name Leqvio. Unlike monoclonal antibodies or oral peptides, inclisiran offers a biannual injection schedule, improving adherence without requiring daily dosing. While its LDL-C reduction is slightly lower than that of Repatha or Praluent, its ease of administration makes it an attractive option for patients averse to frequent dosing.

As of mid-2025, inclisiran has achieved double-digit market share in several European countries and is gaining ground in the United States following broader payer acceptance. Novartis has reported steady uptake across hospital systems and outpatient cardiovascular clinics. Analysts estimate inclisiran could reach $3 billion in annual sales by 2027, particularly if real-world evidence continues to support its effectiveness and cost-efficiency.

Novartis stock has responded modestly to inclisiran’s success, with the cardiovascular franchise seen as a stable, long-term growth pillar rather than a breakout catalyst. Nevertheless, the company’s ability to combine siRNA innovation with broad commercial reach positions it as a resilient competitor in the PCSK9 space.

How is Verve Therapeutics transforming the game with gene-editing?

Verve Therapeutics Inc. is the most speculative of the five names, but also perhaps the most transformative. The American biotech company is developing a once-and-done PCSK9 therapy using CRISPR-based base editing. Its lead candidate, VERVE-101, targets the PCSK9 gene directly, aiming to permanently reduce LDL-C after a single infusion. Preclinical and early human data suggest the potential for lifelong lipid management without ongoing dosing.

However, Verve has encountered regulatory complexity, including a recent clinical hold by the U.S. Food and Drug Administration pending additional safety data. Patient enrollment has resumed in Europe under ex-U.S. regulatory pathways, with interim Phase 1 results expected by early 2026. Analysts from SVB Securities and Piper Sandler view Verve as a high-risk, high-reward play, with potential peak sales exceeding $10 billion if the therapy succeeds—though significant clinical and regulatory hurdles remain.

Institutional interest remains strong, particularly among biotech-focused hedge funds and long-only growth managers. Verve’s market capitalization hovers near $1.2 billion, reflecting speculative enthusiasm around the gene-editing platform.

What does the broader PCSK9 market outlook suggest?

The global PCSK9 inhibitor market, valued at around $2 billion in 2023, is expected to grow to $2.8 billion in 2025 and potentially exceed $8 billion by 2032. This expansion is driven by persistent unmet need in LDL-C management, increased cardiovascular risk stratification, and rising guideline support for aggressive lipid-lowering strategies. Analysts also expect the market to bifurcate into injectable versus non-injectable modalities, with oral and RNA-based therapies likely gaining share in the primary prevention space.

From a pricing and access perspective, value-based models are becoming increasingly important, especially in single-payer markets and U.S. Medicare Advantage plans. Companies with flexible pricing strategies, superior adherence data, and scalable manufacturing capabilities are expected to lead the next phase of PCSK9 market evolution.

Which PCSK9 inhibitor stocks offer the best investment potential in 2025?

Amgen Inc. remains the dominant player in terms of revenue and established clinical credibility, though legal liabilities may temper valuation expansion. Regeneron and Sanofi are positioned to defend Praluent’s modest but stable market share, especially now that patent risks have eased. Merck & Co., Inc. holds the most disruptive potential with enlicitide, a product that could significantly alter adherence dynamics and access in the cholesterol market. Novartis AG, while not headline-grabbing, is strategically positioned with inclisiran as a long-term, low-frequency alternative. Verve Therapeutics offers the highest upside, but with corresponding execution risk tied to novel gene-editing science.

For investors seeking stability with growth potential, Amgen and Novartis offer defensible positioning. Those seeking disruptive upside may find Merck and Verve compelling, while Regeneron–Sanofi remains a value play with renewed optionality.