Protalix Navigates Shifting Gaucher Disease Market, Bets on Pipeline Growth

NEW YORK, NY – November 13, 2025

Revenue Dip Masks Long-Term Growth Strategy

Protalix BioTherapeutics reported third-quarter revenues of $17.9 million, a slight decrease compared to the same period last year. The company attributes the fluctuation primarily to purchasing patterns of its commercial partners – Chiesi, Pfizer, and Fiocruz – rather than a significant shift in patient demographics. Despite the quarterly dip, Protalix posted a net income of approximately $2.4 million, demonstrating a continued focus on managing costs and prioritizing research and development. The company is actively investing in its pipeline, particularly its lead candidate, PRX-115, a potential breakthrough treatment for uncontrolled gout.

“The company’s partners control their own inventory,” explained a source close to the company. “Those purchasing patterns inherently create some quarterly variability.” The quarterly figures mask a longer-term focus on expanding beyond its established Gaucher disease therapy, Elelyso.

Competitive Landscape in Gaucher Disease Treatment

The Gaucher disease market remains competitive, with established players like Sanofi and Takeda vying for market share alongside innovative therapies. While enzyme replacement therapies (ERTs) remain the mainstay of treatment, substrate reduction therapies (SRTs) are gaining traction, and the prospect of gene therapies looms on the horizon. Protalix's Elelyso, a plant cell-expressed recombinant glucocerebrosidase, holds a position in the ERT space, but faces increasing competition and evolving patient preferences.

“There’s a constant push for improved dosing regimens and more convenient administration methods,” said an industry analyst. “Patients are looking for therapies that fit seamlessly into their lives.” Sanofi and Takeda have both introduced enhanced formulations, increasing the pressure on existing treatments. The anticipated arrival of gene therapies could further disrupt the market, offering potentially curative options for some patients. The global Gaucher Disease Treatment Market is estimated to reach $3.7 billion by 2034.

Pipeline Focus: PRX-115 as a Potential Game Changer

Protalix is placing significant emphasis on PRX-115, a PEGylated recombinant uricase designed for the treatment of uncontrolled gout. Recent Phase 1 data demonstrated encouraging results, prompting the company to submit an Investigational New Drug (IND) application to the FDA. An IND was approved in October, and plans are underway to initiate a Phase 2 clinical trial later this year.

“We are particularly excited about PRX-115,” stated a company spokesperson. “We believe it has the potential to be a best-in-class therapy with a long-acting profile, addressing a significant unmet need for patients who do not respond adequately to existing treatments.” The company estimates that the Phase 2 trial will require over $20 million in expenses.

Analysts are cautiously optimistic about PRX-115’s potential. “If the Phase 2 data is positive, this could be a major catalyst for Protalix,” said an industry observer. “Gout is a large and growing market, and a long-acting, effective therapy would be highly valued.” The company’s decision to prioritize PRX-115 reflects a strategic shift towards diversifying its pipeline and reducing its reliance on Elelyso.

Analyst Sentiment and Financial Outlook

Despite the recent quarterly results, analysts remain largely positive on Protalix’s long-term prospects. One analyst recently downgraded their price target from $15.00 to $12.00, citing a review of the company’s financial positioning, but maintained a ‘Buy’ rating. The average price target from analysts is $12.50, with a range of $10.00 to $15.00.

The company’s focus on pipeline development is seen as a key driver of future growth. “Protalix is investing in the right areas,” said an investment strategist. “PRX-115 has the potential to be a blockbuster drug, and the company is also exploring other promising candidates.”

The company’s cash and cash equivalents stood at $29.4 million at the end of the quarter, providing a sufficient runway for continued research and development efforts. Management believes that the current cash position will support operations for at least 12 months. A successful Phase 2 trial for PRX-115 could attract additional investment and accelerate the company’s growth trajectory. The company also anticipates a decision regarding a proposed dosing regimen for its Gaucher disease treatment, Elfabrio, with its European partner, Chiesi, in the first quarter of 2026.