CLINICAL TRIALS PROFILE FOR RILUTEK

All Clinical Trials for RILUTEK

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00026052 ↗	Riluzole to Treat Major Depression	Completed	National Institute of Mental Health (NIMH)	Phase 2	2001-11-01	This study will examine the safety and effectiveness of the drug riluzole (Rilutek® (Registered Trademark)) for short-term treatment of depression symptoms, such as depressed mood, psychomotor retardation, and excessive sleeping. Despite the availability of a wide range of antidepressant drugs, studies indicate that 30 to 40 percent of patients with major depression do not respond to first-line antidepressant treatment with drugs such as fluoxetine, upropion, venlafaxine and others. Riluzole, which is approved by the Food and Drug Administration (FDA) for amyotrophic lateral sclerosis (ALS), causes chemical changes in the brain that may also have antidepressant properties. Patients between 18 and 70 years of age with major depressive disorder without psychotic features may be eligible for this 2-stage 7-week study. Candidates will be screened with a medical history and physical examination, including an electrocardiogram (EKG), blood and urine tests, and a psychiatric evaluation. A blood or urine sample will be tested for illegal drugs.Women of childbearing potential will have a pregnancy test. Participants will complete stage 1 of the study, which lasts 1 week, and may then continue with stage 2 for an additional 6 weeks. At the start of the study, patients will be tapered off all psychiatric medicines and will begin treatment with a placebo (a sugar pill formulated to look like the active drug). At some point, they will be switched from placebo to riluzole. In addition, participants will undergo the following procedures: - Physical examination and electrocardiograms (EKG) at the beginning and end of the study, with vital signs (temperature, blood pressure and heart rate) checked daily - Weekly 1-hour interviews consisting of psychiatric and psychomotor rating scales to assess treatment response - Weekly blood tests to measure blood levels of riluzole and evaluate drug side effects At the end of the study, participants' psychiatric status will be reassessed and appropriate long-term psychiatric treatment arranged. Patients, ages 18 to 70 with a diagnosis of major depression without psychotic features, will in this pilot study (single arm, single blind) receive riluzole (50-200 mg/day) for a period of 6 weeks. Acute efficacy will be determined by demonstrating a greater response rate using specified criteria. Approximately 25 patients will enter the study to obtain 22 subjects who complete the 6 weeks of acute riluzole treatment. Therefore, if 7/22 patients or greater have greater than 50% improvement on the primary efficacy measure, then based on statistically guidelines from the Optimal Two Stage Design for Clinical Trials, a controlled trial would be indicated to scientifically confirm the signal observed in the single arm trial.
NCT00047723 ↗	Minocycline to Treat Amyotrophic Lateral Sclerosis	Completed	National Institute of Neurological Disorders and Stroke (NINDS)	Phase 3	2003-01-01	The purpose of this trial is to test the safety, tolerability, and effectiveness of minocycline compared to placebo in patients with amyotrophic lateral sclerosis (ALS).
NCT00054704 ↗	Riluzole to Treat Depression in Bipolar Disorder	Terminated	National Institute of Mental Health (NIMH)	Phase 2	2003-02-01	This study examines if Riluzole, FDA approved for ALS, will improve symptoms of depression in Bipolar Disorder. Purpose: This study will examine the safety and effectiveness of riluzole (Rilutek trademark) for short-term treatment of depression symptoms, such as depressed mood, psychomotor retardation, and excessive sleeping in patients with bipolar disease. Riluzole is approved by the Food and Drug Administration (FDA) to treat amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease). Preliminary findings of a study using riluzole to treat acute depression in patients with unipolar depression indicate that it may have antidepressant properties in some patients. Patients between 18 and 70 years of age with bipolar I or II disorder without psychosis may be eligible for this 8-week study. Candidates must be currently depressed, must have had at least one previous major depressive episode, and must have failed to improve with prior treatment with at least one antidepressant. They will be screened with a medical history, physical examination, electrocardiogram (EKG), blood and urine tests, and psychiatric evaluation. A blood or urine sample will be analyzed for illegal drugs. Women of childbearing potential will have a pregnancy test. Participants will begin an 8-week course of treatment, starting with a placebo (a sugar pill formulated to look like the active drug) and, at some point, switching to riluzole. In addition to drug treatment, participants will undergo the following procedures: Physical examination and electrocardiogram (EKG) at the beginning and end of the study; Weekly check of vital signs (temperature, blood pressure and heart rate); Weekly 1-hour interviews consisting of psychiatric and psychomotor rating scales to assess treatment response; Weekly blood tests to measure blood levels of riluzole and evaluate drug side effects. At the end of the study, participants' psychiatric status will be reassessed and appropriate long-term psychiatric treatment arranged. Atendemos pacientes de habla hispana. We enroll eligible participants locally and from around the country. Travel arrangements are provided and costs covered by the National Institute of Mental Health (NIMH). (Arrangements vary by distance and by specific study.) After completing the study participants receive short-term follow-up care while transitioning back to a provider.
NCT00251303 ↗	Riluzole to Treat Child and Adolescent Obsessive-Compulsive Disorder With or Without Autism Spectrum Disorders	Completed	National Institute of Mental Health (NIMH)	Phase 2	2005-08-01	This study will examine the effectiveness of riluzole for treating Obsessive-Compulsive Disorder in Youth, Including those with Autism Spectrum Disorders.
NCT00353665 ↗	Memantine for Disability in Amyotrophic Lateral Sclerosis (MEDALS)	Completed	H. Lundbeck A/S	Phase 2/Phase 3	2005-07-01	The purpose of this trial is to study the effect of Memantine (uncompetitive, moderate affinity, NMDA receptor antagonist that binds to the NMDA receptor channel, and regulates the calcium influx into the neurons), a drug used to treat Alzheimer´s disease, on the progression of Amyotrophic Lateral Sclerosis (ALS). Memantine is added to riluzole (the single drug approved to treat ALS).
NCT00353665 ↗	Memantine for Disability in Amyotrophic Lateral Sclerosis (MEDALS)	Completed	University of Lisbon	Phase 2/Phase 3	2005-07-01	The purpose of this trial is to study the effect of Memantine (uncompetitive, moderate affinity, NMDA receptor antagonist that binds to the NMDA receptor channel, and regulates the calcium influx into the neurons), a drug used to treat Alzheimer´s disease, on the progression of Amyotrophic Lateral Sclerosis (ALS). Memantine is added to riluzole (the single drug approved to treat ALS).

Clinical Trial Conditions for RILUTEK

Condition Name

Condition Name for RILUTEK
Intervention	Trials
Amyotrophic Lateral Sclerosis	9
Amyotrophic Lateral Sclerosis (ALS)	2
Depression	2
Bipolar Disorder	2

Condition MeSH

Condition MeSH for RILUTEK
Intervention	Trials
Amyotrophic Lateral Sclerosis	12
Sclerosis	11
Motor Neuron Disease	11
Depression	7

Clinical Trial Locations for RILUTEK

Trials by Country

Trials by Country for RILUTEK
Location	Trials
United States	80
Canada	19
Germany	3
France	3
United Kingdom	3

Trials by US State

Trials by US State for RILUTEK
Location	Trials
Maryland	8
New York	4
Massachusetts	4
Ohio	4
North Carolina	3

Clinical Trial Progress for RILUTEK

Clinical Trial Phase

Clinical Trial Phase for RILUTEK
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	1
Phase 4	3

Clinical Trial Status

Clinical Trial Status for RILUTEK
Clinical Trial Phase	Trials
Completed	21
Recruiting	5
Active, not recruiting	2

Clinical Trial Sponsors for RILUTEK

Sponsor Name

Sponsor Name for RILUTEK
Sponsor	Trials
National Institute of Mental Health (NIMH)	4
AB Science	2
Johns Hopkins University	2

Sponsor Type

Sponsor Type for RILUTEK
Sponsor	Trials
Other	39
Industry	11
NIH	7

RILUTEK Market Analysis and Financial Projection

Last updated: May 4, 2026

Rilutek (riluzole): Clinical Trials Update and Market Analysis Projection

What is Rilutek and what products does it map to?

Rilutek is a brand of riluzole, an oral small-molecule drug used in amyotrophic lateral sclerosis (ALS). The active ingredient is riluzole (CAS 99792-79-7).
Key regulatory positioning: riluzole is an established therapy for ALS in multiple major markets; the commercial footprint is driven by historical adoption rather than new-generation differentiation.

Core drug identity | Item | Value | |---|---| | Trade name | Rilutek | | Generic name | riluzole | | Indication | ALS (disease treatment positioning in practice; prescribing varies by label language by jurisdiction) | | Route | Oral | | Drug class | ALS therapy (glutamate-modulating profile per label narratives) |

What is the current clinical trials status for riluzole?

Riluzole is not a new entrant drug, so the clinical landscape is dominated by: (1) comparative effectiveness work versus other ALS agents, (2) combination regimens, and (3) translational studies seeking biomarkers and patient subgroups rather than new monotherapy pivotal programs.

What is consistently observable from the public clinical-trial record is that riluzole appears as background therapy in many ALS studies rather than as the investigational primary. Where riluzole is the investigational arm, studies generally target dosing schedules, combination approaches, or endpoint expansion (functional scales, survival, biomarkers). This pattern holds across the ALS trial ecosystem because riluzole functions as standard-of-care in many geographies.

Clinical-trial “where it shows up” pattern

• Background standard therapy: trials of agents targeting neuroinflammation, excitotoxicity pathways, mitochondrial function, or growth signaling frequently keep riluzole use stable while evaluating add-on effect.
• Combination trials: riluzole often remains one of multiple concomitant therapies in regimen designs.
• Observational and registry studies: riluzole prescribing patterns and real-world outcomes are commonly evaluated in ALS cohorts.

Implication for “trials update”

• No evidence pattern indicates a new, late-stage riluzole monotherapy pivotal program that would typically reset market expectations.
• The clinical activity that does exist tends to support incremental label refinements (where applicable) and competitive positioning in combination strategies rather than a new exclusivity event driver.

What is the commercial market context for riluzole (Rilutek)?

Riluzole is a mature therapy with long-standing use. Market performance generally depends on:

• ALS prevalence and diagnosis rate growth (population-level demand)
• Treatment adherence and persistence (drug continuity in chronic disease)
• Pricing dynamics (generic erosion and payer pressure in major markets)
• Formulation and access (where brand vs generic share shifts)
• Competitive pressure (new ALS therapies with differentiated mechanisms)

Across most markets, riluzole faces a structural reality: its commercial economics are heavily influenced by genericization. Even in markets where a brand name exists, the net market outcome is often determined by generic pricing and formulary positioning rather than brand-led innovation.

Competitive set and switching pressure (practical drivers)

• Disease-modifying entrants: newer ALS agents can change payer behavior and clinician prescribing patterns, but most regimens still allow concomitant riluzole use.
• Formulary design: many formularies treat riluzole as a core option and require higher-evidence thresholds for newer agents, limiting total substitution.
• Combination logic: because ALS is chronic and multifactorial in trial design, many new therapies add to rather than replace riluzole.

How large is the riluzole opportunity and what is the base-case projection?

Riluzole demand scales with ALS patient volume. Projection methodology in this category typically anchors on:

• Incidence and prevalence trends for ALS
• diagnostic awareness and neurologist referral patterns
• treatment penetration of standard-of-care ALS meds
• net price trajectory (brand to generic mix, rebate intensity, tender outcomes)

Given riluzole’s maturity and generic pressure, the base-case usually shows:

• Volume stability or modest growth aligned to incidence/prevalence
• Revenue growth capped or declining in nominal terms unless price effects stabilize

Base-case market behavior (directional) | Driver | Expected direction for riluzole revenue | |---|---| | ALS patient population | Up | | Treatment penetration | Stable to up (gradual) | | Net price (brand compression, generic mix) | Down | | Net revenue | Flat to down nominal; stable in real terms |

What projection range is appropriate for 2026-2030?

Because riluzole is a mature molecule and the market is structurally shaped by generic economics, the most decision-useful approach is to forecast range outcomes rather than a single point estimate. In the absence of new riluzole-specific exclusivity catalysts, the plausible scenarios cluster around generic pricing and treatment penetration.

Projection frame (not a point forecast)

• Conservative scenario: continued net price compression outpaces volume gains, producing nominal revenue decline.
• Base scenario: ALS prevalence growth offsets price erosion, yielding flat nominal revenue or mild growth in select markets.
• Upside scenario: payer coverage stabilizes, and uptake increases (real-world adherence/persistence improvements), narrowing the price gap.

Indicative outcome bands | Horizon | Conservative | Base | Upside | |---|---:|---:|---:| | 2026-2027 | Decline in nominal revenue | Flat | Mild growth | | 2028-2030 | More pronounced nominal decline | Flat to mild growth | Mid-single digit growth in pockets |

Does riluzole face a major regulatory risk or label discontinuation risk?

For an established ALS standard, major discontinuation risk is typically tied to:

• failure of post-market safety surveillance,
• withdrawal in key markets,
• label restriction changes.

No pattern in the public regulatory ecosystem suggests a broad discontinuation of riluzole. The drug remains referenced in ALS care pathways. The main business risk is less “regulatory termination” and more “commercial erosion” through generic price competition and formulary changes.

What is the likely impact of “clinical trial activity” on riluzole revenue?

In mature molecules with background standard positioning, new trials usually have limited direct upside for standalone riluzole revenue because:

• they do not create new exclusivity,
• they often accept riluzole as background rather than investigational,
• uptake is already high where it is standard-of-care.

The clinical activity can still affect:

• treatment adherence guidelines,
• which patient subgroups are encouraged to use riluzole,
• persistence through clinical pathways.

But the mechanism is indirect and typically slow, so it tends to show up as incremental volume or persistence rather than sharp revenue steps.

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Key Takeaways

• Rilutek is riluzole, an established ALS therapy; the commercial story is driven by ALS population demand and generic pricing dynamics rather than new riluzole-specific exclusivity.
• Clinical trial activity exists primarily as background therapy or combination design logic in ALS programs, with less evidence of a new late-stage riluzole pivotal monotherapy development cycle.
• Market projection for 2026-2030 should be modeled as flat-to-declining nominal revenue with volume growth offset by net price pressure. Upside is possible in pockets if payer coverage and persistence improve, not from breakthrough clinical differentiation.

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FAQs

1) Is riluzole still used as standard-of-care in ALS trials?

Yes. Riluzole frequently appears as background standard therapy in ALS clinical studies, with investigational interventions assessed on top of existing treatment.

2) What has the biggest effect on riluzole revenue: volume or price?

Net price (brand-to-generic mix, rebates, and formulary pressure) typically dominates short-to-medium horizon revenue behavior. Volume growth from prevalence/incidence trends usually cannot fully offset price compression.

3) Are there late-stage clinical programs that could reset riluzole’s market?

No consistent pattern suggests an imminent riluzole-specific late-stage pivotal monotherapy catalyst that would change the market structure.

4) Does riluzole compete head-to-head with newer ALS therapies?

Often it does not. Many newer therapies are used in combination or as add-on options, so switching is limited by regimen logic and payer coverage patterns.

5) What should investors watch for in riluzole going forward?

The key watch items are ALS epidemiology trends, treatment penetration and persistence, and net price trajectory by market (generic mix and payer formulary outcomes).

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References

[1] ClinicalTrials.gov. Search results for riluzole in amyotrophic lateral sclerosis (ALS). U.S. National Library of Medicine.
[2] European Medicines Agency (EMA). Riluzole product information and public assessment materials relevant to ALS indications.
[3] U.S. Food and Drug Administration (FDA). Riluzole prescribing information and ALS label history for riluzole-containing products.