pharma · Epilepsy · Allergic Reaction · AQST
Aquestive Therapeutics is a pharma organization headquartered in Vancouver, CA. It trades on NYSE under ticker AQST. Primary therapeutic focus areas include Epilepsy, Allergic Reaction, Nausea and Vomiting, Postoperative
Phase 2 · small molecule · ALS
Riluzole Oral Soluble Film (internal code 17MO1R-0016) is an oral formulation of riluzole, a small-molecule nervous system therapeutic developed by Aquestive Therapeutics for amyotrophic lateral sclerosis (ALS). The program represents a reformulation strategy applied to an established active pharmaceutical ingredient w
Internal code 17MO1R-0016
Riluzole Oral Soluble Film (internal code 17MO1R-0016) is an oral formulation of riluzole, a small-molecule nervous system therapeutic developed by Aquestive Therapeutics for amyotrophic lateral sclerosis (ALS). The program represents a reformulation strategy applied to an established active pharmaceutical ingredient with a long regulatory history. Riluzole itself has been approved in multiple jurisdictions including the United States, European Union, and Australia, where it is marketed under various brand names including PHARMACOR RILUZOLE. Aquestive's soluble film delivery technology aims to optimize the pharmacokinetic or patient convenience profile of the existing molecule.
The riluzole oral soluble film program reached Phase 2 clinical development, with the most recent disclosed milestone dated 13 May 2019. The program has since been terminated, indicating that Aquestive discontinued further development. The termination occurred after Phase 2 evaluation, suggesting that efficacy, safety, or commercial considerations led to the decision to halt progression. Clinical trial activity is documented under NCT03457753, though detailed results and the specific rationale for termination have not been disclosed in the provided facts.
Riluzole remains an approved therapy for ALS across major markets. The competitive landscape for ALS includes agents such as QALSODY (recently approved), NUEDEXTA, and other neurological therapeutics. Aquestive's strategy of applying its soluble film technology to established molecules has been applied to other programs, though this particular riluzole formulation did not advance to regulatory approval.
Amyotrophic lateral sclerosis is a progressive neurodegenerative disorder with limited therapeutic options and significant unmet medical need. Riluzole is one of the few disease-modifying agents available, making optimization of its delivery and tolerability clinically relevant. Patient convenience and adherence remain challenges in ALS management, particularly for patients with progressive motor dysfunction affecting swallowing and oral intake. A soluble film formulation could theoretically address administration barriers for patients with dysphagia or difficulty swallowing tablets.
The commercial significance of ALS therapeutics has increased with recent approvals and expanded treatment paradigms. However, the termination of this program suggests that the clinical or commercial case for a riluzole soluble film formulation was insufficient to justify continued investment. The decision may reflect competitive pressures from newer ALS agents, limited differentiation over existing riluzole formulations, or Phase 2 efficacy or safety findings that did not support advancement.
Aquestive's soluble film technology platform has been applied to multiple neurological and other indications. The termination of the riluzole program does not necessarily reflect on the viability of the platform itself, but rather on the specific commercial and clinical calculus for this particular indication and formulation. The ALS market continues to evolve with new mechanism classes and combination approaches, which may have influenced the decision to discontinue this reformulation effort.
Drug Class: Nervous system therapeutic (ATC N07); established small-molecule agent.
Modality: Small molecule.
Route of Administration: Oral soluble film (reformulation of conventional oral riluzole).
Mechanism of Action: Not disclosed in the facts; riluzole is known clinically to have neuroprotective properties in ALS, though the precise mechanism remains incompletely understood.
Target: Not disclosed in the facts.
Related Therapies: Riluzole is available in multiple formulations and from multiple manufacturers globally, including PHARMACOR RILUZOLE (Pharmacor Pty Limited, Australia), and approved generic formulations in the United States from manufacturers including Alkem Labs, Apotex, Azurity, Daito Pharma, Glenmark, Impax, Italfarmaco, Kenton, Pharmobedient, and Sun Pharma. A liquid suspension formulation of riluzole (Tiglutik) has also been approved in some markets.
First Approval: Riluzole was first approved in the United States under NDA020599 (original innovator application). Australian approvals date from 2003 onwards. European approvals include EMEA/H/C/000109 (Sanofi Winthrop Industrie) and EMEA/H/C/002622 (Zentiva k.s., authorised 24 July 2024).
Patent Status: Not disclosed in the facts.
Also known as: ALS, Charcot disease, Lou Gehrig disease
Prevalence: Point prevalence: 1-9 / 100 000 (Europe) — source: Orphanet, validated.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by progressive muscular paralysis reflecting degeneration of motor neurons in the primary motor cortex, corticospinal tracts, brainstem and spinal cord.
ClinicalTrials.gov lists 700 registered studies for Amyotrophic Lateral Sclerosis (AACT aggregate).
Phase breakdown: NA (363), PHASE2 (127), PHASE1 (77), PHASE1/PHASE2 (51), PHASE3 (38), PHASE2/PHASE3 (27), EARLY_PHASE1 (12), PHASE4 (5)
Common investigational therapies:
Disease data sourced from MONDO Disease Ontology (MONDO:0004976), Orphanet — amyotrophic lateral sclerosis, NCT00004457, NCT00004771, NCT00005674, NCT00005766, NCT00007722, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, Open Targets Platform (CC BY 4.0).
Latest disclosed milestone
Most recent program activity; specific milestone details not disclosed.
Program termination
Program terminated after Phase 2; exact termination date and rationale not disclosed.
The ALS therapeutic landscape includes multiple approved agents and recent entrants. QALSODY (tofersen, Biogen) represents a newer mechanism class (antisense oligonucleotide targeting SOD1) and was recently approved, expanding treatment options beyond riluzole. NUEDEXTA (dextromethorphan/quinidine, Amneal Pharma Europe) addresses pseudobulbar affect in ALS patients. Other neurological agents listed in the competitive set—including AMVUTTRA, AUSTEDO, DAYBU, ONPATTRO, XYREM, NERVENTRA, B-PATCH, and VYNDAMAX—represent different therapeutic classes and indications, though some may be used off-label or in overlapping patient populations.
Riluzole itself remains widely available as generic formulations from multiple manufacturers, creating a highly commoditized market for the active ingredient. Aquestive's soluble film formulation would have competed against established riluzole products and newer mechanism-based therapies. The termination of this program suggests that differentiation through formulation alone was insufficient to justify Phase 3 investment and regulatory pursuit, particularly given the availability of alternative riluzole formulations (including liquid suspension) and the emergence of disease-modifying agents with distinct mechanisms.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| AMVUTTRA | Lacuna Pharma Pty Ltd | — | approved |
| AUSTEDO | Teva Pharma GmbH | — | approved |
| DAYBU | Acadia Pharmaceuticals B.V. | — | approved |
| ONPATTRO | Lacuna Pharma Pty Ltd | — | approved |
| XYREM | Amneal Pharma Europe Ltd | — | approved |
| NERVENTRA | Teva Pharma GmbH | — | approved |
| NUEDEXTA | Amneal Pharma Europe Ltd | — | approved |
| B-PATCH | Indivior Pty Ltd | — | approved |
| PHARMACOR VARENICLINE | Alphapharm Pty Ltd | — | approved |
| VIOKAT | SOLENO THERAPEUTICS INC | — | approved |
| VYNDAMAX | Pfizer Australia Pty Ltd | — | approved |
| QALSODY | — | — | approved |
| TOFERSEN | — | SOD1 mRNA inhibitor | Approved |
| RILUZOLE | — | Sodium channel alpha subunit blocker | Approved |
| VALPROIC ACID | — | Succinate semialdehyde dehydrogenase inhibitor | Phase 3 |
| VALPROATE SODIUM | — | Succinate semialdehyde dehydrogenase inhibitor | Phase 3 |
| TIRASEMTIV | — | Fast skeletal troponin complex activator | Phase 3 |
| RAVULIZUMAB | — | Complement C5 inhibitor | Phase 3 |
| QUINIDINE | — | Sodium channel alpha subunit blocker | Phase 3 |
| MECASERMIN | — | Insulin-like growth factor I receptor agonist | Phase 3 |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
United States: Riluzole is approved via NDA020599 (original innovator) and multiple ANDA applications (generic formulations) from Alkem Labs, Apotex, Azurity, Daito Pharma, Glenmark, Impax, Italfarmaco, Kenton, Pharmobedient, and Sun Pharma. Aquestive holds an approved ANDA (ANDA206045) for a riluzole formulation. The oral soluble film program (17MO1R-0016) did not advance to FDA approval; regulatory status is terminated.
European Union: Riluzole is approved under EMEA/H/C/000109 (Sanofi Winthrop Industrie) and EMEA/H/C/002622 (Zentiva k.s., authorised 24 July 2024). The soluble film formulation did not receive EMA approval.
Australia: Riluzole is approved and listed on the PBS (Pharmaceutical Benefits Scheme) under multiple codes (11662T, 14393M, 14429K, 8664B) from sponsors including Asteri Pharma, Pharmacor Pty Limited, Sandoz, and Sanofi-Aventis Australia. First listings date from 2003 onwards.
China (NMPA): Riluzole is in clinical trials in China (NCT04950933); regulatory approval status for the soluble film formulation is not disclosed.
Japan (PMDA): Regulatory status not disclosed in the facts.
Riluzole oral soluble film is a reformulated version of riluzole, an established ALS therapeutic, using Aquestive Therapeutics' soluble film delivery technology. The program was designed to optimize the administration or pharmacokinetic profile of the active ingredient.
Riluzole is used to treat amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease affecting motor neurons. It is one of the few disease-modifying agents available for ALS.
No. The riluzole oral soluble film program (17MO1R-0016) was terminated after Phase 2 development and did not receive regulatory approval in any jurisdiction.
Aquestive Therapeutics developed the riluzole oral soluble film program using its proprietary soluble film formulation technology.
The program reached Phase 2 clinical development before being terminated. The most recent disclosed milestone was 13 May 2019.
Clinical trial NCT03457753 was associated with the program; however, detailed trial design, results, and outcomes are not disclosed in the available facts.
Yes. Riluzole is approved for ALS in the United States, European Union, Australia, and other jurisdictions. It is available as generic formulations from multiple manufacturers and under brand names such as PHARMACOR RILUZOLE.
Aquestive's soluble film technology is a proprietary drug delivery platform designed to optimize the administration and pharmacokinetic profile of oral medications. The riluzole program represents one application of this technology.
The specific rationale for termination is not disclosed in the available facts. Possible reasons may include Phase 2 efficacy or safety findings, commercial considerations, or competitive pressures from generic riluzole and newer ALS agents.
Competitors include QALSODY (tofersen, a newer antisense oligonucleotide), NUEDEXTA (dextromethorphan/quinidine for pseudobulbar affect), and other neurological agents. Riluzole also competes with generic formulations from multiple manufacturers.
Yes. Tiglutik (riluzole oral suspension) is an approved liquid formulation of riluzole available in some markets, providing an alternative to tablet formulations.
Riluzole is known clinically to have neuroprotective properties in ALS, though the precise mechanism of action remains incompletely understood and is not disclosed in the available facts.
Riluzole is manufactured by multiple companies globally, including Alkem Labs, Apotex, Azurity, Daito Pharma, Glenmark, Impax, Italfarmaco, Kenton, Pharmobedient, Sun Pharma, Sanofi, Zentiva, Sandoz, and others.
Riluzole was first approved in the United States under NDA020599 (date not specified in facts). Australian approvals date from 2003 onwards; European approvals include authorisations from 2024.
No. The program has been terminated and is no longer in active development by Aquestive Therapeutics.
Riluzole is classified as a nervous system therapeutic (ATC N07) and is a small-molecule drug administered orally.
Riluzole Oral Soluble Film → Drug → Target → Indication → Company → Trials → Competitors
Strategic Implications: Aquestive's decision to terminate the riluzole soluble film program after Phase 2 reflects the commercial and clinical challenges of reformulation-based strategies in mature, generic-dominated markets. Riluzole's status as a widely available, low-cost generic limits the pricing power and market differentiation available to a new formulation, even if it offers convenience advantages. The Phase 2 termination suggests either insufficient efficacy/safety signals or a commercial assessment that Phase 3 investment and regulatory pursuit would not yield adequate return.
Competitive Implications: The termination does not materially affect the ALS market, as riluzole remains available from multiple sources. Newer agents such as QALSODY represent more substantial competitive threats to riluzole-based therapies through distinct mechanisms and disease-modifying potential. Aquestive's platform technology (soluble film) may be better suited to molecules with stronger intellectual property protection, higher therapeutic value, or less competitive generic pressure.
Future Catalysts: No further development milestones are expected for this program. Aquestive's pipeline focus has likely shifted to other indications or formulations with greater commercial potential.
Expected Milestones: None; program is terminated.
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Intelligence compiled from public regulatory and clinical sources (FDA, EMA, ClinicalTrials.gov and company disclosures). Figures may be editorial or analyst estimates; verify against primary sources before relying on them.