Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) is a pioneering biopharmaceutical company committed to developing novel and life-extending treatments for people living with progressive neurodegenerative diseases. Founded in 2013 by two undergraduate students at Brown University, Joshua Cohen and Justin Klee, Amylyx began with a powerful vision: to disrupt the status quo in neurological research by targeting the common underlying causes of neuronal death. From that bold academic idea emerged a clinical-stage company that would go on to challenge long-held limitations in the treatment of diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s, Wolfram syndrome, and other rare neurological disorders.
Amylyx first rose to prominence through the development of AMX0035, an innovative oral therapy designed to simultaneously target mitochondrial dysfunction and endoplasmic reticulum stress—two key contributors to neuronal degeneration. The company’s most significant milestone came in 2022, when AMX0035 was approved by the U.S. Food and Drug Administration (FDA) under the brand name Relyvrio for the treatment of ALS. This approval marked a major inflection point not just for Amylyx, but for the broader ALS community, which had long been underserved with limited therapeutic options.
While Amylyx faced a critical turning point in early 2024 after the Phase 3 PHOENIX trial failed to confirm Relyvrio’s survival benefit in ALS—leading to the drug’s voluntary withdrawal from the market—the company demonstrated clinical integrity, fiscal responsibility, and an unwavering commitment to patients. Rather than retreat, Amylyx used the moment to recalibrate its long-term strategy, accelerate new trials, and expand its research into other neurodegenerative and metabolic diseases with high unmet needs.
Today, Amylyx is advancing a robust and diversified pipeline that reflects its broader mission of transforming outcomes for patients with historically intractable conditions. This includes the continued investigation of AMX0035 in Wolfram syndrome, a rare genetic disorder with neurological and metabolic manifestations, and the development of Avexitide, a promising therapy for post-bariatric hypoglycemia (PBH) currently in Phase 3. In addition, the company is exploring next-generation neuroprotective candidates such as AMX0114, leveraging its foundational science and platform technologies.
Amylyx operates with a patient-first philosophy that is embedded in every aspect of its work—from its rigorous clinical trial designs to its transparent communication with stakeholders and regulatory bodies. With a headquarters in Cambridge, Massachusetts and a growing team of neuroscientists, clinicians, and regulatory experts, the company continues to embody its founding spirit of curiosity, urgency, and accountability.
Now at a critical turning point in its corporate journey, Amylyx is actively reshaping its identity—not as a single-drug company, but as a neuroscience-focused biotech platform with multiple paths to growth. Backed by significant analyst support, strong institutional interest, and several upcoming clinical readouts, Amylyx is well positioned to re-emerge as a key player in the fight against neurodegeneration.
Analyst Upgrades Signal a Major Shift in Wall Street Sentiment
Recent analyst activity suggests that institutional confidence in Amylyx is on the rise. On June 17, 2025, Citigroup’s Geoff Meacham initiated coverage on Amylyx with a “Buy” rating and a 12-month price target of $12.00. This initiation marks a turning point for the company, as Citi’s bullish outlook reflects broader belief in Amylyx’s evolving pipeline and upcoming clinical milestones. Citi’s endorsement is not an outlier—it builds on a growing body of bullish sentiment from other brokerages, including HC Wainwright, Mizuho, and Leerink Partners.
According to aggregated Wall Street data, Amylyx currently holds a consensus price target of $10.80, with the highest estimate at $16.00 and a low estimate of $8.00. These projections imply a potential upside of nearly 100% from current trading levels around $5.40. The average brokerage recommendation currently sits at 1.7, which falls firmly into “Outperform” territory on the standard 1-to-5 scale. This string of endorsements points to renewed institutional interest in Amylyx’s long-term story and provides a validation of its strategic pivot following Relyvrio’s withdrawal.
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Diversified Pipeline: From ALS to Rare Metabolic Disorders
While AMX0035’s path in ALS may be paused, the compound is not dead. Amylyx is continuing to explore its potential in Wolfram syndrome, a rare, inherited neurodegenerative and metabolic disorder. Preliminary data from the Phase 2 HELIOS study has shown encouraging results, and the company expects pivotal data from the next trial stage in Q3 2025. Success here could provide an unexpected comeback narrative for AMX0035 in a niche orphan indication with regulatory and pricing advantages.
Meanwhile, Amylyx is expanding its reach through Avexitide, a GLP-1 receptor antagonist designed to treat post-bariatric hypoglycemia (PBH)—a debilitating condition with no approved therapies. Avexitide is currently being evaluated in the pivotal LUCIDITY Phase 3 trial, with topline data expected in the first half of 2026. If successful, Avexitide could become a first-in-class treatment and a meaningful commercial asset.
Further enhancing the pipeline is AMX0114, a next-generation neuroprotective candidate for ALS. The company has already dosed the first patient in the Phase 1 LUMINA trial, and while it is still early, AMX0114 is based on a refined mechanism of action informed by Amylyx’s work with AMX0035. Collectively, these programs position Amylyx with a multi-asset pipeline targeting high-unmet-need indications, each with significant market potential and limited direct competition.
Financial Position and Market Recovery Potential
Amylyx remains in a stable financial position, having wisely preserved cash following the withdrawal of Relyvrio. The company has taken steps to reduce operational expenses while continuing to support key clinical programs. This lean approach, coupled with the company’s potential for orphan drug designations and favorable regulatory pathways, gives Amylyx a capital-efficient runway to deliver meaningful milestones over the next 12 to 18 months.
Shares of AMLX are still trading at distressed levels relative to the company’s long-term potential, largely reflecting lingering caution after the ALS setback. However, institutional inflows are returning, with fund holdings increasing and options activity skewing bullish. Technically, the stock appears to be stabilizing and trending upward, trading above its 50-day moving average with momentum building into upcoming data catalysts.
Multiple Catalysts Set to Drive Revaluation in 2025–2026
Investors who follow biotech turnarounds understand that value is often unlocked not at approval, but at the point of re-rating—when the market begins to acknowledge that a company’s narrative has changed. Amylyx is rapidly approaching that inflection point. Three key catalysts could drive substantial upside over the next year: the Phase 2b/3 data for AMX0035 in Wolfram syndrome, topline Phase 3 results for Avexitide in PBH, and early clinical signals from AMX0114 in ALS.
If even one of these programs delivers positive results, Amylyx’s valuation could be rerated substantially higher, especially given that Wall Street consensus already sees a 2x return from current levels. The company’s focus on orphan indications also means that a relatively small patient population can translate into high pricing power and regulatory incentives such as fast-track approval and market exclusivity.
Conclusion: Amylyx Is Rebuilding Its Identity—and Its Upside
Amylyx Pharmaceuticals is no longer just “the Relyvrio company.” It is evolving into a platform-driven biotech with multiple high-value clinical programs, strong leadership, and growing analyst support. While past challenges cannot be ignored, they have prompted a necessary realignment that may ultimately strengthen the company’s long-term trajectory. The addition of Citigroup’s “Buy” rating and $12 price target reflects a renewed belief in Amylyx’s science, strategy, and market opportunity.
For biotech investors seeking asymmetric return potential, Amylyx presents a compelling speculative opportunity. With an average price target nearly 100% above current levels, several late-stage trials on deck, and a leaner, smarter operating strategy in place, AMLX could become one of the most surprising turnaround stories in biotech over the next 12 months.
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