Diazepam

Updated top-line results demonstrate improved outcomes in adults with narcolepsy or idiopathic hypersomnia treated with Xywav as observed in the Phase 4 DUET (Developing Understanding of Hypersomnia by Evaluating Low-Sodium Oxybate Treatment) study Novel analysis of real-world Epidiolex treatment patterns underscore importance of dose optimization for improved patient persistence For U.S. media and investors only DUBLIN, March 19, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that seven abstracts from across its neuroscience portfolio will be featured at the 77th Annual American Academy of Neurology Meeting (AAN) being held April 5-9, 2025, in San Diego. Data presented at the meeting includes an updated presentation of top-line results of the open-label, single-arm, Phase 4 DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) trial, evaluating the effectiveness and safety of low-sodium oxybate, Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution, on key sleep outcomes and daytime symptoms, including excessive daytime sleepiness (EDS), in adults with narcolepsy or idiopathic hypersomnia (IH). Notably, the results from DUET demonstrated statistically significant improvements from baseline to end of treatment in Epworth Sleepiness Scale (ESS) scores, reduced sleep stage shifts, increased deep sleep and reduced number of awakenings among adults with narcolepsy treated with Xywav. In adults with IH, Xywav treatment also showed improvements in ESS and Idiopathic Hypersomnia Severity Scale scores. Further, new patient-reported data from DUET demonstrated the association of Xywav treatment with improvements in daytime symptoms and functional impacts, including cognitive complaints, among adults with narcolepsy or IH. Xywav is indicated for the treatment of EDS or cataplexy in patients 7 years of age and older with narcolepsy and for adults with idiopathic hypersomnia. "This year's presentations at the AAN Annual Meeting reinforce our ongoing commitment to addressing debilitating neurological disorders, with limited or no treatment options including narcolepsy, idiopathic hypersomnia and epilepsy," said Sarah Akerman, MD, head of neuroscience global medical and scientific affairs of Jazz Pharmaceuticals. "We continually strive to expand our understanding, research and capabilities in neuroscience through ongoing data generation for Xywav and Epidiolex to ensure patients' lived experiences and needs are reflected holistically. By listening to our patients, their care teams and leading industry experts, we aim to better address the unmet needs of those living with these difficult-to-treat conditions and ultimately help redefine possibilities for their lives." Highlights at the 2025 AAN Annual Meeting include: Two presentations showcasing updated results from the open-label, single-arm, Phase 4 DUET trial of adults with narcolepsy or IH, which evaluated the effectiveness and safety of Xywav on key sleep outcomes, daytime symptoms, and functional impacts, including cognitive complaints, work productivity, and daily activities. Novel analysis of real-world Epidiolex® (cannabidiol) treatment patterns from U.S. specialty pharmacy data found the overall probability of persistence at one year was nearly 70% (69.9%) among new patients and underscores the importance of dose optimization. The analysis shows how healthcare providers optimize dosing over time, with half (52%) of patients taking dosages >15 mg/kg/day at 12 months and those taking an average of >20 mg/kg/day having the lowest likelihood of discontinuation. This real-world data demonstrates the importance of dose optimization for long-term persistence, which could lead to improved seizure control and tolerability. The 2025 AAN Annual Meeting abstracts are available online at index.mirasmart.com/AAN2025. A full list of Jazz Pharmaceuticals' presentations follows below: About Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution Xywav is the only low-sodium oxybate approved by the U.S. Food and Drug Administration (FDA) for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy. The Office of Orphan Product Development (OOPD) at the FDA also published its summary of clinical superiority findings for Xywav for the treatment of cataplexy or EDS in patients 7 years of age and older with narcolepsy by means of greater cardiovascular safety compared to Xyrem® (sodium oxybate) oral solution. The decision of the OOPD is based on the FDA findings that Xywav provides a greatly reduced chronic sodium burden compared to Xyrem. Xywav has 131 mg of sodium at the maximum recommended nightly dose whereas other high sodium oxybates have 1640 mg at the equivalent dose. Xywav is comprised of a unique composition of cations resulting in 92% less sodium, or a reduction of approximately 1,000 to 1,500 mg/night at the recommended dose range of 6 g to 9 g/night. Xywav is the only oxybate therapy that does not carry a warning in the label related to use in patients sensitive to high sodium intake. Xywav is also the first and only U.S. FDA-approved treatment option for idiopathic hypersomnia in adults. The FDA recognized seven years of Orphan Drug Exclusivity for Xywav for the treatment of idiopathic hypersomnia in adults. Xywav is the only FDA-approved treatment studied across the multiple symptoms of idiopathic hypersomnia, such as EDS, sleep inertia (severe grogginess or confusion when waking up), long sleep duration and cognitive impairment. Xywav can be administered as a twice- or once-nightly regimen for the treatment of idiopathic hypersomnia in adults. The exact mechanism of action of Xywav in the treatment of adults with idiopathic hypersomnia and of cataplexy and EDS in narcolepsy is unknown. It is hypothesized that the therapeutic effects of Xywav are mediated through GABAB actions during sleep at noradrenergic and dopaminergic neurons, as well as thalamocortical neurons.1 The U.S. Drug Enforcement Agency (DEA) has designated Xywav as a Schedule III medicine. The DEA defines Schedule III drugs, substances, or chemicals as drugs with a moderate to low potential for physical and psychological dependence.1,2 Because of the risks of central nervous system (CNS) depression and abuse and misuse, Xywav is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the XYWAV and XYREM REMS. Important Safety Information for Xywav Contraindications XYWAV is contraindicated in combination with sedative hypnotics or alcohol and in patients with succinic semialdehyde dehydrogenase deficiency. Warnings and Precautions Central Nervous System Depression The concurrent use of XYWAV with other CNS depressants, including but not limited to opioid analgesics, benzodiazepines, sedating antidepressants or antipsychotics, sedating anti-epileptic drugs, general anesthetics, muscle relaxants, and/or illicit CNS depressants, may increase the risk of respiratory depression, hypotension, profound sedation, syncope, and death. If use of these CNS depressants in combination with XYWAV is required, dose reduction or discontinuation of one or more CNS depressants (including XYWAV) should be considered. In addition, if short-term use of an opioid (eg, post- or perioperative) is required, interruption of treatment with XYWAV should be considered. After first initiating treatment and until certain that XYWAV does not affect them adversely (eg, impair judgment, thinking, or motor skills), caution patients against hazardous activities requiring complete mental alertness or motor coordination such as operating hazardous machinery, including automobiles or airplanes. Also caution patients against these hazardous activities for at least 6 hours after taking XYWAV. Patients should be queried about CNS depression-related events upon initiation of XYWAV therapy and periodically thereafter. Abuse and Misuse XYWAV is a Schedule Ill controlled substance. The active moiety of XYWAV is oxybate, also known as gamma-hydroxybutyrate (GHB), a Schedule I controlled substance. Abuse of illicit GHB, either alone or in combination with other CNS depressants, is associated with CNS adverse reactions, including seizure, respiratory depression, decreases in the level of consciousness, coma, and death. The rapid onset of sedation, coupled with the amnestic features of GHB particularly when combined with alcohol, has proven to be dangerous for the voluntary and involuntary user (eg, assault victim). Physicians should carefully evaluate patients for a history of drug abuse and follow such patients closely. XYWAV and XYREM REMS Because of the risks of central nervous system depression and abuse and misuse, XYWAV is available only through a restricted distribution program called the XYWAV and XYREM REMS. Notable requirements of the XYWAV and XYREM REMS include the following: Healthcare Providers who prescribe XYWAV are specially certified XYWAV will be dispensed only by the central pharmacy that is specially certified XYWAV will be dispensed and shipped only to patients who are enrolled in the XYWAV and XYREM REMS with documentation of safe use Further information is available at or 1-866-997-3688. Respiratory Depression and Sleep-Disordered Breathing XYWAV may impair respiratory drive, especially in patients with compromised respiratory function. In overdoses of oxybate and with illicit use of GHB, life-threatening respiratory depression has been reported. Increased apnea and reduced oxygenation may occur with XYWAV administration in adult and pediatric patients. A significant increase in the number of central apneas and clinically significant oxygen desaturation may occur in patients with obstructive sleep apnea treated with XYWAV. Prescribers should be aware that sleep-related breathing disorders tend to be more prevalent in obese patients, in men, in postmenopausal women not on hormone replacement therapy, and among patients with narcolepsy. Depression and Suicidality In Study 1, the randomized-withdrawal clinical trial in adult patients with narcolepsy (n=201), depression and depressed mood were reported in 3% and 4%, respectively, of patients treated with XYWAV. Two patients (1%) discontinued XYWAV because of depression. In most cases, no change in XYWAV treatment was required. In Study 2, the randomized-withdrawal clinical trial in adult patients with idiopathic hypersomnia (n=154), depression and depressed mood were reported in 1% and 3%, respectively, of patients treated with XYWAV. All patients continued XYWAV treatment. Two suicides and two attempted suicides occurred in adult clinical trials with oxybate (same active moiety as XYWAV). One patient experienced suicidal ideation and two patients reported depression in a pediatric clinical trial with oxybate. These events occurred in patients with and without previous histories of depressive disorders. The emergence of depression in patients treated with XYWAV requires careful and immediate evaluation. Monitor patients for the emergence of increased depressive symptoms and/or suicidality while taking XYWAV. Other Behavioral or Psychiatric Adverse Reactions In Study 1, confusion and anxiety occurred in 1% and 5% of patients with narcolepsy treated with XYWAV, respectively. One patient experienced visual hallucinations and confusion after ingesting approximately 9 grams of XYWAV. In Study 2, confusion and anxiety occurred in 3% and 16% of patients with idiopathic hypersomnia, respectively. One patient experienced visual hallucinations, which led to discontinuation of XYWAV. Other neuropsychiatric reactions reported with oxybate (same active moiety as XYWAV) in adult or pediatric clinical trials and in the postmarketing setting include hallucinations, paranoia, psychosis, aggression, agitation, confusion and anxiety. The emergence or increase in the occurrence of behavioral or psychiatric events in patients taking XYWAV should be carefully monitored. Parasomnias Parasomnias can occur in patients taking XYWAV. In Study 1 and Study 2, parasomnias, including sleepwalking, were reported in 6% and 5% of adult patients treated with XYWAV, respectively. In a clinical trial of XYREM (same active moiety as XYWAV) in adult patients with narcolepsy, five instances of sleepwalking with potential injury or significant injury were reported. Parasomnias, including sleepwalking, have been reported in a pediatric clinical trial with sodium oxybate (same active moiety as XYWAV) and in postmarketing experience with sodium oxybate. Episodes of sleepwalking should be fully evaluated and appropriate interventions considered. Most Common Adverse Reactions The most common adverse reactions (occurring in ≥5% of XYWAV-treated patients in adult clinical trials in either narcolepsy or IH) were nausea, headache, dizziness, anxiety, insomnia, decreased appetite, hyperhidrosis, vomiting, diarrhea, dry mouth, parasomnia, somnolence, fatigue, and tremor. In the pediatric clinical trial with XYREM (same active moiety as XYWAV) that included pediatric patients 7 to 17 years of age with narcolepsy, the most common adverse reactions (≥5%) were nausea (20%), enuresis (19%), vomiting (18%), headache (17%), weight decreased (13%), decreased appetite (9%), dizziness (8%), and sleepwalking (6%). The overall adverse reaction profile of XYREM in the pediatric clinical trial was similar to that seen in the adult clinical trial program. The safety profile in pediatric patients with XYWAV is expected to be similar to that of adult patients treated with XYWAV and to that of pediatric patients treated with XYREM. Additional Adverse Reactions Adverse reactions that occurred in 2-<5% of adult patients treated with XYWAV in the Open­ Label Titration and Stable Dose Periods of the randomized-withdrawal study in adult patients with narcolepsy with cataplexy (Study 1) were fatigue, dry mouth, depressed mood, enuresis, irritability, paresthesia, depression, tremor, somnolence, and muscle spasms. Adverse reactions occurring in 2-<5% of patients treated with XYWAV in the IH study include balance disorder, muscle spasms, fall, paresthesia, snoring, weight decreased, bruxism, confusional state, depressed mood, feeling drunk, and irritability. Adverse reactions that occurred in ≥2% of patients in clinical studies with oxybate (but not in Study 1) and which may be relevant for XYWAV, were pain, feeling drunk, pain in extremity, cataplexy, disturbance in attention, sleep paralysis, and disorientation. Discontinuation: In Study 1, 9 of 201 patients (4%) reported adverse reactions that led to withdrawal from the study (anxiety, decreased appetite, depressed mood, depression, fatigue, headache, irritability, nausea, pain in extremity, parasomnia, somnolence, and vomiting). The most common adverse reaction leading to discontinuation was nausea (1.5%). In Study 2, 17 of 154 (11%) patients across all study periods (excluding placebo during the DB RWP) (up to 42 weeks) reported adverse reactions that led to withdrawal from the study (anxiety, nausea, insomnia, vomiting, fatigue, feeling abnormal, fall, decreased appetite, dizziness, paresthesia, tremor, parasomnia, confusional state, hallucination visual, and irritability). The most common adverse reaction leading to discontinuation was anxiety (3.2%). In Study 1 and Study 2, the majority of adverse reactions leading to discontinuation began during the first few weeks of treatment. In the pediatric clinical trial with XYREM (same active moiety as XYWAV), 7 of 104 patients reported adverse reactions that led to withdrawal from the study (hallucination, tactile; suicidal ideation; weight decreased; sleep apnea syndrome; affect lability; anger, anxiety, depression; and headache). Drug Interactions XYWAV is contraindicated in combination with alcohol or sedative hypnotics. Use of other CNS depressants may potentiate the CNS-depressant effects of XYWAV. Concomitant use of sodium oxybate with divalproex sodium results in an increase in systemic exposure to GHB, which was shown to cause a greater impairment on some tests of attention and working memory in a clinical study. A similar increase in exposure is expected with concomitant use of XYWAV and divalproex sodium; therefore, an initial dose reduction of XYWAV is recommended when used concomitantly with divalproex sodium. Prescribers are advised to monitor patient response closely and adjust dose accordingly if concomitant use of XYWAV and divalproex sodium is warranted. Pregnancy and Lactation There are no adequate data on the developmental risk associated with the use of XYWAV or sodium oxybate in pregnant women. XYWAV should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. GHB is excreted in human milk after oral administration of sodium oxybate. There is insufficient information on the risk to a breastfed infant, and there is insufficient information on milk production in nursing mothers. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for XYWAV and any potential adverse effects on the breastfed infant from XYWAV or from the underlying maternal condition. Pediatric Use The safety and effectiveness of XYWAV for the treatment of cataplexy or excessive daytime sleepiness in pediatric patients 7 years of age and older with narcolepsy have been established. XYWAV has not been studied in a pediatric clinical trial for narcolepsy or IH. Use of XYWAV in pediatric patients 7 years of age and older with narcolepsy is supported by evidence from an adequate and well­controlled study of sodium oxybate in pediatric patients 7 to 17 years of age, a study in adults showing a treatment effect of XYWAV similar to that observed with sodium oxybate, pharmacokinetic data of sodium oxybate from adult and pediatric patients, and pharmacokinetic data of XYWAV from healthy adult volunteers. Safety and effectiveness of XYWAV in pediatric patients below the age of 7 years with narcolepsy have not been established. Safety and effectiveness of XYWAV for the treatment of idiopathic hypersomnia in pediatric patients have not been established. Geriatric Use In general, dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy. Hepatic Impairment The starting dose of XYWAV should be reduced in patients with liver impairment. Dosage Modification in Patients with Hepatic Impairment: The recommended starting dosage in patients with hepatic impairment is one-half of the original dosage per night, administered orally, divided into two doses. Dependence and Tolerance There have been case reports of withdrawal, ranging from mild to severe, following discontinuation of illicit use of GHB at frequent repeated doses (18 g to 250 g per day) in excess of the recommended dosage range. Signs and symptoms of GHB withdrawal following abrupt discontinuation included insomnia, restlessness, anxiety, psychosis, lethargy, nausea, tremor, sweating, muscle cramps, tachycardia, headache, dizziness, rebound fatigue and sleepiness, confusion, and, particularly in the case of severe withdrawal, visual hallucinations, agitation, and delirium. These symptoms generally abated in 3 to 14 days. In cases of severe withdrawal, hospitalization may be required. In the clinical trial experience with XYREM in narcolepsy/cataplexy patients at recommended doses, two patients reported anxiety and one reported insomnia following abrupt discontinuation at the termination of the clinical trial; in the two patients with anxiety, the frequency of cataplexy had increased markedly at the same time. In the XYWAV clinical trial in adult narcolepsy/cataplexy patients at recommended doses, one patient reported insomnia following abrupt discontinuation of XYWAV. In the XYWAV clinical trial in adult idiopathic hypersomnia patients at recommended doses, six patients reported insomnia, two patients reported early insomnia, and one patient reported visual and auditory hallucinations following abrupt discontinuation of XYWAV. Tolerance to XYWAV has not been systematically studied in controlled clinical trials. There have been some case reports of symptoms of tolerance developing after illicit use at dosages far in excess of the recommended XYWAV dosage regimen. About Epidiolex®/Epidyolex® (cannabidiol) Epidiolex/Epidyolex is a prescription, plant-derived cannabis-based medicine administered as an oral solution which contains highly purified cannabidiol (CBD). Cannabidiol, the active ingredient in Epidiolex, is a cannabinoid that naturally occurs in the Cannabis sativa L. plant. The precise mechanisms by which Epidiolex exerts its anticonvulsant effect in humans are unknown. Epidiolex was approved by the U.S. Food and Drug Administration (FDA) for use in the U.S., the European Commission (EC) for use in Europe, the Medicines and Healthcare products Regulatory Agency (MHRA) for use in Great Britain, the Therapeutic Goods Administration for use in Australia, Swissmedic for use in Switzerland, the Food & Nutrition Services of the Israel Ministry of Health for use in Israel, and the New Zealand Medicines and Medical Devices Safety Authority for use in New Zealand, is an oral solution which contains highly purified cannabidiol (CBD). In the U.S., Epidiolex is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) or tuberous sclerosis complex (TSC) in patients one year of age and older. Epidiolex has received approval in the European Union under the tradename Epidyolex for adjunctive use in conjunction with clobazam to treat seizures associated with LGS and DS in patients two years and older, and for adjunctive use to treat seizures associated with TSC, in patients two years of age and older. Epidiolex has received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of seizures associated with LGS, DS, and TSC. Similarly, Epidyolex received ODD from the European Medicines Agency (EMA) for the same indications. Important Safety Information & Indications CONTRAINDICATION: HYPERSENSITIVITY EPIDIOLEX (cannabidiol) oral solution is contraindicated in patients with a history of hypersensitivity to cannabidiol or any ingredients in the product. WARNINGS & PRECAUTIONS Hepatic Injury: EPIDIOLEX can cause dose-related transaminase elevations. Concomitant use of valproate and elevated transaminase levels at baseline increase this risk. Obtain transaminase and bilirubin levels prior to starting treatment, at 1, 3, and 6 months after initiation of treatment, and periodically thereafter, or as clinically indicated. Resolution of transaminase elevations occurred with discontinuation of EPIDIOLEX, reduction of EPIDIOLEX and/or concomitant valproate, or without dose reduction. For patients with elevated transaminase levels, consider dose reduction or discontinuation of EPIDIOLEX or concomitant medications known to affect the liver (e.g., valproate or clobazam). Dose adjustment and slower dose titration is recommended in patients with moderate or severe hepatic impairment. Consider not initiating EPIDIOLEX in patients with evidence of significant liver injury. There have been postmarketing reports of cholestatic or mixed patterns of liver injury. Elevated ammonia levels were reported in some patients with transaminase elevations; most taking concomitant valproate, clobazam, or both. Consider discontinuation or dose adjustment of valproate or clobazam if ammonia is elevated. Somnolence and Sedation: EPIDIOLEX can cause somnolence and sedation that generally occurs early in treatment and may diminish over time; these effects occur more commonly in patients using clobazam and may be potentiated by other CNS depressants. Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including EPIDIOLEX, increase the risk of suicidal thoughts or behavior. Inform patients, caregivers, and families of the risk and advise them to monitor and report any signs of depression, suicidal thoughts or behavior, or unusual changes in mood or behavior. If these symptoms occur, consider if they are related to the AED or the underlying illness. Withdrawal of Antiepileptic Drugs: As with most AEDs, EPIDIOLEX should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. ADVERSE REACTIONS: The most common adverse reactions in patients receiving EPIDIOLEX (≥10% and greater than placebo) include transaminase elevations; somnolence; decreased appetite; diarrhea; pyrexia; vomiting; fatigue, malaise, and asthenia; rash; insomnia, sleep disorder and poor-quality sleep; and infections. Hematologic abnormalities were also observed. PREGNANCY: EPIDIOLEX should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Encourage women who are taking EPIDIOLEX during pregnancy to enroll in the EPIDIOLEX Pregnancy Surveillance Program and the North American Antiepileptic Drug (NAAED) Pregnancy Registry. DRUG INTERACTIONS: Strong inducers of CYP3A4 and CYP2C19 may affect EPIDIOLEX exposure. EPIDIOLEX may affect exposure to CYP2C19 substrates (e.g., clobazam, diazepam, stiripentol), orally administered P-gp substrates, or other substrates (see full Prescribing Information). Consider dose reduction of orally administered everolimus, with appropriate therapeutic drug monitoring, when everolimus is combined with EPIDIOLEX. A lower starting dose of everolimus is recommended when added to EPIDIOLEX therapy. Concomitant use of EPIDIOLEX and valproate increases the incidence of liver enzyme elevations. Pneumonia was observed more frequently with concomitant use of EPIDIOLEX and clobazam. Dosage adjustment of EPIDIOLEX or other concomitant medications may be necessary. INDICATIONS: EPIDIOLEX (cannabidiol) oral solution is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 1 year of age and older. Please read the EPIDIOLEX full Prescribing Information for additional important information here . About Jazz Pharmaceuticals Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit for more information. Contacts: Media: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 Investors: Jeff Macdonald Executive Director, Investor Relations Jazz Pharmaceuticals plc [email protected] Ireland +353 1 634 3211 U.S. +1 650 496 2717 References: Xywav (calcium, magnesium, potassium and sodium oxybates) oral solution. Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc. 2021. United States Drug Enforcement Agency. Drug Scheduling. . Accessed March 2025. SOURCE Jazz Pharmaceuticals plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Initiated Anaphylm™ (epinephrine) Sublingual Film NDA filing process with FDA Anaphylm pre-commercial activities underway; launch expected in Q1 2026, if approved by the FDA Initial Anaphylm pediatric clinical trial results in subjects 7-17 years of age in line with expectations AQST-108 (epinephrine) Topical Gel Phase 2a clinical trial on track to begin in Q2 2025 Proforma cash and cash equivalents as of December 31, 2024 approximately $93.0 million Company to host investment community conference call on March 6, 2025 WARREN, N.J., March 05, 2025 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST) (“Aquestive” or the “Company”), a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies, today reported financial results for the fourth quarter and full year ended December 31, 2024, and provided a progress update on the key 2025 objectives previously outlined by the Company. "We are thrilled to announce today the start of our Anaphylm application process with the FDA. We anticipate completion of this process in the first quarter of 2025 and achievement of the FDA acceptance milestone before the end of the second quarter of 2025,” said Daniel Barber, President and Chief Executive Officer of Aquestive. "This is a major moment in the Company’s history, and we are excited to move significantly closer to the day when patients would benefit from a non-device, non-invasive based treatment option for severe allergic reactions, including anaphylaxis. In parallel, we are also excited to advance our AQST-108 (epinephrine) Topical Gel for patients suffering from alopecia areata into Phase 2a clinical trial development. We believe that we are well-positioned to execute on our strategic priorities and key milestones for 2025." Anaphylm™ (epinephrine) Sublingual FilmAquestive is advancing the development of Anaphylm, a non-device based epinephrine product candidate that has demonstrated clinical results comparable to autoinjectors (such as EpiPen® and Auvi-Q®) for the emergency treatment of severe allergic reactions, including anaphylaxis. In the fourth quarter of 2024, the Company successfully completed all adult clinical trials for Anaphylm and received positive feedback in its pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA). The pediatric clinical trial for Anaphylm will be finalized shortly and the initial topline data from this clinical trial is in line with expectations. With the completion of the Anaphylm clinical program, the Company has started the NDA submission, which will include the necessary pediatric data to support a product label, if approved by the FDA, that would align to the weight and age parameters of the existing 0.3 mg EpiPen® autoinjector. The Company expects to receive acceptance of its NDA for Anaphylm by the FDA in the second quarter of 2025. In the pre-NDA meeting minutes received from the FDA in the fourth quarter of 2024, the Agency noted that a Pulmonary-Allergy Drugs Advisory Committee may be necessary during the review process for Anaphylm. The advisory committee meeting process would provide Aquestive with the opportunity to further highlight the potential benefits of Anaphylm for patients. Aquestive is actively preparing for this potential Advisory Committee discussion. Aquestive continues to prepare for the launch of Anaphylm, a potentially transformative product for the emergency treatment of severe allergic reactions, including anaphylaxis. The initial focus of the Company's launch preparation has been optimizing Anaphylm's market access for patients, if approved by the FDA. Aquestive is also increasing awareness of Anaphylm through the continued execution of its medical affairs strategy, including presenting scientific data at medical forums throughout 2025. The company remains committed to a successful launch of Anaphylm in the first quarter of 2026, if approved by the FDA. AQST-108 (epinephrine) Topical GelAquestive is advancing the development of AQST-108 (epinephrine) Topical Gel for the treatment of alopecia areata (AA). The Company successfully completed a pre-Investigational New Drug Application (IND) meeting with the FDA in December 2024. The written response received from the FDA was supportive of continued development of AQST-108. The Company remains on track to begin its Phase 2a clinical trial of AQST-108 in patients with AA in the second quarter of 2025. An estimated 6.7 million people in the United States have been affected by AA. Of those affected, 43% are considered severe. The existing therapies for alopecia areata are janus kinase (or JAK) inhibitors. These systemic treatments with known side effects come with a "black box" warning and are expensive for patients. Even with these limitations, the current estimated market opportunity for JAK inhibitors is over One Billion U.S. dollars. In the Company’s first-in-human Phase 1 clinical trial, AQST-108 demonstrated no serious adverse events or topical adverse events. Since AQST-108 is topical and there is evidence that it acts at the application site, it may not have systemic side effects. As a result of these conditions, AQST-108, if approved by the FDA as a treatment for AA, has the potential to capture meaningful market share for the treatment of these patients. Libervant® (diazepam) Buccal FilmAquestive received FDA approval of Libervant for patients aged between two and five years in April of 2024. Libervant is the first and only FDA approved orally administered rescue product for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) (or ARS) in patients between two and five years of age. This milestone ensured that younger ARS patients in this age group have access to this essential treatment. In December 2024, the Company received Orphan Drug Exclusivity (ODE) for Libervant for ARS patients between two and five years of age until April 2031. Aquestive launched Libervant for ARS patients aged between two and five years in 2024 with a small, targeted sales force and access for the product has been expanding since its launch. Demand for Libervant in this labeled patient population continues to increase and in the fourth quarter of 2024 it became fully available through national retail distribution. In February 2025, the U.S. District Court for the District of Columbia ruled that the FDA erred in approving Libervant for ARS patients between the ages of two and five years as a result of the FDA’s prior grant of ODE to the previously approved Valtoco nasal spray product for ARS patients aged six years and older. The Company is appealing the Court's ruling. Aquestive intends to continue to take action to keep Libervant on the market for these young patients and to engage with the FDA to protect these young patients. The NDA for Libervant for ARS patients twelve years of age and older was tentatively approved by the FDA in August 2022 and is currently subject to the ODE block of Valtoco until January 2027. Commercial CollaborationsAquestive continues to manufacture products for the licensing and supply collaborations that it has established. The Company manufactured approximately 43 million doses in the fourth quarter 2024, compared to approximately 45 million doses in the fourth quarter 2023. The Company continues to see consistent order demand for the manufacture of Indivior’s Suboxone® Sublingual Film product and continues to support its other global collaborations including the recent launch of Emylif® (riluzole) Oral Film product by Zambon S.p.A in Europe. Sales of royalty-based products, inclusive of Sympazan® (clobazam) Oral Film for the treatment of seizures associated with Lennox-Gastaut Syndrome in patients two years of age and older and Azstarys® for the treatment of Attention Deficit Hyperactivity Disorder (ADHD) in patients six years of age and older, continued to provide a steady stream of revenue in the fourth quarter of 2024. Fourth Quarter 2024 FinancialsTotal revenues decreased to $11.9 million in the fourth quarter 2024, from $13.2 million in the fourth quarter 2023. This 10% decrease in revenue was primarily driven by decreases in license and royalty revenue due to $1.0 million of milestone royalty revenue for Azstarys from Zevra Therapeutics recognized in the prior year. Manufacture and supply revenue decreased to $10.7 million in the fourth quarter 2024 from $11.0 million in the fourth quarter 2023, primarily due to a decrease in Suboxone revenues, partially offset by an increase in Ondif® revenues from Hypera Pharma due to timing of orders. License and royalty revenue decreased to $0.8 million in the fourth quarter 2024 from $1.9 million in the fourth quarter 2023, primarily due to $1.0 million of milestone royalty revenue for Azstarys from Zevra Therapeutics recognized in the prior year. Research and development (R&D) expenses increased to $4.9 million in the fourth quarter 2024 from $2.9 million in the fourth quarter 2023. The increase in R&D expenses was primarily due to an increase in clinical trial costs associated with the continued advancement of the Anaphylm development program. Selling, general and administrative expenses increased to $16.0 million in the fourth quarter 2024 from $9.6 million in the fourth quarter 2023, primarily due to increased commercial spending for both Libervant and Anaphylm, regulatory fees related to the approval of Libervant, severance costs of $1.8 million, and higher legal expenses of $1.5 million, partially offset by a decrease in insurance fees. Aquestive’s net loss for the fourth quarter 2024 was $17.1 million, or ($0.19) for both basic and diluted loss per share, compared to the net loss for the fourth quarter 2023 of $8.1 million, or ($0.12) for both basic and diluted loss per share. The increase in net loss was primarily driven by increases in selling, general and administrative expenses, R&D expenses, non-cash interest expense related to amortization of debt and royalty obligation discounts, and decreases in revenues partially offset by increases in interest income and other income, net, and decreases in manufacture and supply expenses and loss on the extinguishment of debt. Non-GAAP adjusted EBITDA loss was $11.0 million in the fourth quarter 2024, compared to Non-GAAP adjusted EBITDA loss of $2.8 million in the fourth quarter 2023. Non-GAAP adjusted EBITDA loss excluding adjusted R&D expenses was $6.6 million in the fourth quarter 2024, compared to a Non-GAAP adjusted EBITDA loss excluding adjusted R&D expenses of $0.1 million in the fourth quarter 2023. Full Year 2024 FinancialsTotal revenues increased to $57.6 million for the full year 2024, from $50.6 million for the full year 2023. This 14% increase in revenue was primarily driven by increases in license and royalty revenue due to the recognition of deferred revenue from the termination of certain licensing and supply agreements, and increases in co-development and research fees, partially offset by decreases in manufacture and supply revenue. Manufacture and supply revenue decreased to $40.0 million for the full year 2024 from $43.8 million for the full year 2023, primarily due to decreases in Suboxone and Sympazan revenues, partially offset by an increase in Emylif® revenues from Zambon and an increase in Ondif revenues. Excluding the one-time retroactive price increase of $1.7 million recognized in the prior year, manufacture and supply revenue decreased to $40.0 million for the full year 2024 from $42.1 million for the full year 2023. R&D expenses increased to $20.3 million for the full year 2024 from $13.1 million in the full year 2023. The increase in R&D expenses was primarily due to clinical trial costs and product research expenses associated with the continued advancement of the Anaphylm and AQST-108 development programs, increases in personnel costs, and an increase in share-based compensation. Selling, general and administrative expenses increased to $50.2 million for the full year 2024 from $31.8 million for the full year 2023. Selling, general and administrative expenses excluding changes to the allocation methodology for manufacture and supply costs increased to $45.6 million for the full year 2024 from $31.8 million for the full year 2023. This was primarily driven by higher commercial and regulatory spending for Libervant and Anaphylm of approximately $6.2 million, one-time severance costs of $2.9 million, higher shared-based compensation expenses of $2.3 million, and higher non-commercial personnel costs of approximately $2.0 million. Aquestive's net loss for the full year 2024 was $44.1 million, or ($0.51) for both basic and diluted loss per share, compared to the net loss for the full year 2023 of $7.9 million, or ($0.13) for both basic and diluted loss per share. The increase in net loss was primarily driven by increases in selling, general and administrative expenses, R&D expenses, non-cash interest expenses related to amortization of debt and royalty obligation discounts, and decreases in interest income and other income, net partially offset by increases in revenues and decreases in manufacture and supply expenses and loss on the extinguishment of debt. Non-GAAP adjusted EBITDA loss was $23.0 million for the full year 2024, compared to Non-GAAP adjusted EBITDA loss of $11.6 million for the full year 2023. Non-GAAP adjusted EBITDA loss excluding adjusted R&D expenses was $4.0 million for the full year 2024, compared to Non-GAAP adjusted EBITDA income excluding adjusted R&D expenses of $1.0 million for the full year 2023. As of December 31, 2024, cash and cash equivalents were $71.5 million. Proforma cash and cash equivalents were approximately $93.0 million, which includes capital net proceeds of $21.4 million received on February 14, 2025 through the Company's "At-the-Market" (ATM) facility. 2025 OutlookAquestive is providing its full year 2025 financial outlook. The Company expects: Our revenue guidance for 2025 includes Libervant for ages two to five and some level of erosion in the demand for Suboxone. As a reminder, our 2024 revenue included one-time recognition of deferred revenue related to the termination of certain licensing and supply agreements. Our Non-GAAP adjusted EBITDA loss guidance for 2025 includes significant pre-commercial spending for Anaphylm as well as costs associated with the submission of the Anaphylm NDA (and related filing fee), completion of the Anaphylm pediatric clinical trial, preparations for a potential advisory committee if required by the FDA for approval of Anaphylm, commencing the AQST-108 Phase 2a clinical trial in second quarter of 2025, and continued commercialization of Libervant for epilepsy patients aged between two and five years. Tomorrow’s Conference Call and Webcast ReminderThe Company will host a conference call at 8:00 a.m. EST on Thursday, March 6, 2025. In order to participate, please register in advance here to obtain a local or toll-free phone number and your personal pin. A live webcast of the call will be available on the "Events and Presentations" page of the Investor section of Aquestive’s website: Fourth Quarter 2024 Earnings Call. The webcast will be archived for 30 days. About Anaphylm™Anaphylm™ (epinephrine) Sublingual Film is a polymer matrix-based epinephrine prodrug product. Anaphylm is similar in size to a postage stamp, weighs less than an ounce, and begins to dissolve on contact. No water or swallowing is required for administration. The primary packaging for Anaphylm is thinner and smaller than an average credit card, can be carried in a pocket, and is designed to withstand weather excursions such as exposure to rain and/or sunlight. The Anaphylm trade name for AQST-109 has been conditionally approved by the FDA. Final approval of the Anaphylm proprietary name is conditioned on FDA approval of the product candidate. About AQST-108AQST-108 (epinephrine) Topical Gel is a topically delivered adrenergic agonist prodrug gel product candidate. Aquestive completed a first in human study for AQST-108 that measured the amount of epinephrine that remained on the skin or was found in circulation in the human body over time after the application of the topical gel and without any serious or topical adverse events. AQST-108 is based on Aquestive’s Adrenaverse™ platform which contains a library of over twenty epinephrine prodrug product candidates intended to control absorption and conversion rates across a variety of possible dosage forms and delivery sites. About Libervant®Libervant® (diazepam) Buccal Film is a buccally, or inside of the cheek, administered film formulation of diazepam, a benzodiazepine intended for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) (or ARS) that are distinct from a patient’s usual seizure pattern, approved by the FDA for ARS patients between two and five years of age. Aquestive developed Libervant as an alternative to the device-based products currently available for patients with refractory epilepsy, including a rectal gel and nasal spray products. The FDA granted tentative approval in August 2022 for Libervant for treatment of ARS patients twelve years of age and older, with U.S. market access for Libervant for this age group of patients subject to the expiration of the existing orphan drug market exclusivity of a previously FDA approved nasal spray drug scheduled to expire in January 2027. In December 2024, the Company received ODE for Libervant for ARS patients between two and five years of age until April 2031. In February 2025, the U.S. District Court for the District of Columbia ruled that the FDA erred in approving Libervant for ARS patients between the ages of two and five years as a result of the FDA’s prior grant of ODE to the previously approved Valtoco nasal spray product for ARS patients aged six years and older. The Company is appealing this ruling and has requested an immediate stay of the ruling. The Company intends to continue to take action to keep Libervant on the market for these young patients and to engage with the FDA to protect these young patients. The Company is not able at this time to determine or predict the ultimate outcome of these proceedings or provide a reasonable estimate or range of estimates of the possible outcome in these matters. Important Safety InformationDo not give Libervant to your child between the ages of two and five if your child is allergic to diazepam or any of the ingredients in Libervant or has an eye problem called acute narrow angle glaucoma. What is the most important information I should know about Libervant? Libervant is a benzodiazepine medicine. Taking benzodiazepines with opioid medicines, alcohol, or other central nervous system (CNS) depressants (including street drugs) can cause severe drowsiness, breathing problems (respiratory depression), coma, and death. Get emergency help right away if any of the following happens: shallow or slowed breathing, breathing stops (which may lead to the heart stopping), excessive sleepiness (sedation). shallow or slowed breathing, breathing stops (which may lead to the heart stopping), excessive sleepiness (sedation). Do not allow your child to drive a motor vehicle, operate heavy machinery, or ride a bicycle until you know how taking Libervant with opioids affects your child. Risk of abuse, misuse, and addiction. Libervant is used in children 2 to 5 years of age. The unapproved use of Libervant has a risk for abuse, misuse, and addiction, which can lead to overdose and serious side effects including coma and death. Serious side effects including coma and death have happened in people who have abused or misused benzodiazepines, including diazepam (the active ingredient in Libervant). These serious side effects may also include delirium, paranoia, suicidal thoughts or actions, seizures, and difficulty breathing. Call your child’s healthcare provider or go to the nearest hospital emergency room right away if you get any of these serious side effects. Your child can develop an addiction even if your child takes Libervant as prescribed by your child’s healthcare provider. Give Libervant exactly as your child’s healthcare provider prescribed. Do not share Libervant with other people. Keep Libervant in a safe place and away from children. Physical dependence and withdrawal reactions. Libervant is intended for use if needed in order to treat higher than usual seizure activity. Benzodiazepines, including Libervant, can cause physical dependence and withdrawal reactions, especially if used daily. Libervant is not intended for daily use. Do not suddenly stop giving Libervant to your child without talking to your child’s healthcare provider. Stopping Libervant suddenly can cause serious and life-threatening side effects, including, unusual movements, responses, or expressions, seizures that will not stop (status epilepticus), sudden and severe mental or nervous system changes, depression, seeing or hearing things that others do not see or hear, homicidal thoughts, an extreme increase in activity or talking, losing touch with reality, and suicidal thoughts or actions. Call your child’s healthcare provider or go to the nearest hospital emergency room right away if your child gets any of these symptoms. Some people who suddenly stop benzodiazepines have symptoms that can last for several weeks to more than 12 months including, anxiety, trouble remembering, learning, or concentrating, depression, problems sleeping, feeling like insects are crawling under your skin, weakness, shaking, muscle twitching, burning, or prickling feeling in your hands, arms, legs or feet, and ringing in your ears. Physical dependence is not the same as drug addiction. Your child’s healthcare provider can tell you more about the differences between physical dependence and drug addiction. Do not give your child more Libervant than prescribed or give Libervant more often than prescribed. Your child can develop an addiction even if your child takes Libervant as prescribed by your child’s healthcare provider. Give Libervant exactly as your child’s healthcare provider prescribed. Do not share Libervant with other people. Keep Libervant in a safe place and away from children. Do not suddenly stop giving Libervant to your child without talking to your child’s healthcare provider. Stopping Libervant suddenly can cause serious and life-threatening side effects, including, unusual movements, responses, or expressions, seizures that will not stop (status epilepticus), sudden and severe mental or nervous system changes, depression, seeing or hearing things that others do not see or hear, homicidal thoughts, an extreme increase in activity or talking, losing touch with reality, and suicidal thoughts or actions. Call your child’s healthcare provider or go to the nearest hospital emergency room right away if your child gets any of these symptoms. Some people who suddenly stop benzodiazepines have symptoms that can last for several weeks to more than 12 months including, anxiety, trouble remembering, learning, or concentrating, depression, problems sleeping, feeling like insects are crawling under your skin, weakness, shaking, muscle twitching, burning, or prickling feeling in your hands, arms, legs or feet, and ringing in your ears. Physical dependence is not the same as drug addiction. Your child’s healthcare provider can tell you more about the differences between physical dependence and drug addiction. Libervant can make your child sleepy or dizzy and can slow your child’s thinking and motor skills. Do not allow your child to drive a motor vehicle, operate machinery, or ride a bicycle until you know how Libervant affects your child. Do not give other drugs that may make your child sleepy or dizzy while taking Libervant without first talking to your child’s healthcare provider. When taken with drugs that cause sleepiness or dizziness, Libervant may make your child’s sleepiness or dizziness much worse. Like other antiepileptic medicines, Libervant may cause suicidal thoughts or actions in a small number of people, about 1 in 500. Call a healthcare provider right away if your child has any of these symptoms, especially if they are new, worse, or worry you: thoughts about suicide or dying new or worse depression feeling agitated or restless trouble sleeping (insomnia) acting aggressive, being angry or violent other unusual changes in behavior or mood attempts to commit suicide new or worse anxiety or irritability an extreme increase in activity and talking (mania) new or worse panic attacks acting on dangerous impulses Pay attention to any changes, especially sudden changes in mood, behaviors, thoughts, or feelings. Keep all follow-up visits with your child’s healthcare provider as scheduled. Call your child’s healthcare provider between visits as needed, especially if you are worried about symptoms. Suicidal thoughts or actions can be caused by things other than medicines. If your child has suicidal thoughts or actions, your child’s healthcare provider may check for other causes. thoughts about suicide or dying new or worse depression feeling agitated or restless trouble sleeping (insomnia) acting aggressive, being angry or violent other unusual changes in behavior or mood attempts to commit suicide new or worse anxiety or irritability an extreme increase in activity and talking (mania) new or worse panic attacks acting on dangerous impulses What are the possible side effects of Libervant? The most common side effects of Libervant are sleepiness and headache. These are not all the possible side effects of Libervant. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1 800 FDA-1088. For more information about Libervant, talk to your doctor, and see Product Information: Medication Guide and Instructions For Use. About Aquestive Therapeutics, Inc.Aquestive is a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies. We are developing orally administered and topical gel products to deliver complex molecules, providing novel alternatives to invasive and inconvenient standard of care therapies. Aquestive has five commercialized products marketed by the Company and its licensees in the U.S. and around the world and is the exclusive manufacturer of these licensed products. The Company also collaborates with pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven drug development and commercialization capabilities. Aquestive is advancing a late-stage proprietary product candidate for the treatment of severe allergic reactions, including anaphylaxis, and an early-stage epinephrine prodrug topical gel product candidate for the treatment of possible various dermatology conditions, including alopecia areata. For more information, visit Aquestive.com and follow us on LinkedIn. Non-GAAP Financial InformationThis press release and our webcast earnings call regarding our quarterly financial results contains financial measures that do not comply with U.S. generally accepted accounting principles (GAAP), such as non-GAAP adjusted EBITDA loss, non-GAAP adjusted EBITDA loss excluding adjusted R&D expenses, non-GAAP adjusted gross margins, non-GAAP adjusted costs and expenses and other adjusted expense measures, because such measures exclude, as applicable, share-based compensation expense, interest expense, interest expense related to the sale of future revenue, interest income, depreciation, amortization, and income taxes.  Specifically, the Company adjusts net income (loss) for loss on the extinguishment of debt; certain non-cash expenses, including share-based compensation expenses; depreciation and amortization; and interest expense related to the sale of future revenue, interest income and other income (expense), net and income taxes, with a result of adjusted EBITDA loss.  Similarly, manufacture and supply expense, R&D expense, and selling, general and administrative expense were adjusted for certain non-cash expenses of share-based compensation expense and depreciation and amortization. Adjusted EBITDA loss and these non-GAAP expense categories are used as a supplement to the corresponding GAAP measures to provide additional insight regarding the Company’s ongoing operating performance.  These measures supplement the Company’s financial results prepared in accordance with GAAP. Aquestive management uses these measures to analyze its financial results, and its future manufacture and supply expenses, gross margins, R&D expense and selling, general and administrative expense and to help make managerial decisions. In management’s opinion, these non-GAAP measures provide added transparency into the operating performance of Aquestive and added insight into the effectiveness of our operating strategies and actions. The Company may provide one or more revenue measures adjusted for certain discrete items, such as fees collected on certain licensed products, in order to provide investors added insight into our revenue stream and breakdown, along with providing our GAAP revenue. Such measures are intended to supplement, not act as substitutes for, comparable GAAP measures and should not be read as a measure of liquidity for Aquestive. Adjusted EBITDA loss and the other non-GAAP measures are also likely calculated in a way that is not comparable to similarly titled measures reported by other companies. Non-GAAP OutlookIn providing the outlook for non-GAAP adjusted EBITDA and non-GAAP gross margin, we exclude certain items which are otherwise included in determining the comparable GAAP financial measures. In order to inform our outlook measures of non-GAAP adjusted EBITDA and non-GAAP gross margin, a description of the adjustments which have been applicable in determining non-GAAP Adjusted EBITDA and non-GAAP gross margin for these periods are reflected in the tables below. In providing outlook for non-GAAP gross margin, the Company adjusts for non-cash share-based compensation expense and depreciation and amortization. The Company is providing such outlook only on a non-GAAP basis because the Company is unable to predict with reasonable certainty the totality or ultimate outcome or occurrence of these adjustments for the forward-looking period such as share-based compensation expense, income tax, amortization, and certain other adjusted items, which can be dependent on future events that may not be reliably predicted. Based on past reported results, where one or more of these items have been applicable, such excluded items could be material, individually or in the aggregate, to reported results. Forward-Looking StatementCertain statements in this press release include “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “believe,” “anticipate,” “plan,” “expect,” “estimate,” “intend,” “may,” “will,” or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding the advancement and related timing of our product candidate Anaphylm™ (epinephrine) Sublingual Film through clinical development and approval by the FDA, including the timing of submission of pediatric clinical studies, and filing and acceptance of the NDA for Anaphylm with the FDA and potential approved label indications, and the following launch of Anaphylm, if approved by the FDA; the results of the Company’s clinical studies for Anaphylm and the ability of such results to support submission of the NDA for approval of Anaphylm to the FDA; Anaphylm's potential as a non-device, non-invasive based treatment option for severe allergic reactions, including anaphylaxis, if Anaphylm is approved by the FDA; the advancement, growth and related timing of our Adrenaverse™ pipeline of epinephrine prodrug product candidates, including AQST-108 (epinephrine) Topical Gel through clinical development and FDA regulatory approval process, including design and timing of clinical studies including those necessary to support the targeted indication of alopecia areata for AQST-108 and the potential market share that AQST-108 may achieve, if approved by the FDA and launched; the increased market demand for our product Libervant® (diazepam) Buccal Film for the label indicated epilepsy patients between the ages of two and five years; the advancement and related timing of our product candidate Libervant® (diazepam) Buccal Film for the label indicated epilepsy patient population aged between 6 and 11 years through FDA regulatory approval and the following launch of Libervant for this patient population, if approved by the FDA upon the expiration of the orphan drug market exclusivity of an FDA approved nasal spray product of another company scheduled to expire in January 2027; the potential benefits our products and product candidates could bring to patients; the achievement of clinical and commercial milestones; our future financial and operating results and financial position, including with respect to our 2025 financial outlook; and business strategies, market opportunities, and other statements that are not historical facts. These forward-looking statements are based on our current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks and uncertainties include, but are not limited to, risks associated with our development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and plans, including those relating to Anaphylm (including for pediatric patients), AQST-108, and our other product candidates; risks associated with our distribution work for Libervant, including any delays or changes to the timing, cost and success of our distribution activities and expansion of market access for Libervant to ARS patients aged between two and five years; risk of delays in advancement of the regulatory approval process through the FDA of our product candidates, including the filing of the respective NDAs, for Anaphylm, AQST-108, Libervant for patients aged between 6 and 11 years and other product candidates, or failure to receive FDA approval at all of any of these product candidates; risk of the Company’s ability to generate sufficient clinical data for approval of our product candidates, including with respect to our PK/PD comparability submission for FDA approval of Anaphylm; risks associated with our ability to address the FDA’s comments on our future clinical trials and other concerns identified in the FDA Type C meeting minutes for Anaphylm, including the risk that the FDA may require additional clinical studies for approval of Anaphylm; risks associated with the success of any competing products, including generics; risk that we may not succeed in appealing the ruling of the U.S. District Court of the District of Columbia that the FDA approval of Libervant for the label indicated epilepsy patients between the ages of two and five years was in error as a result of the current orphan drug market exclusivity granted by the FDA for a prior approved nasal spray product of another company for this patient population aged six years and older; risks of not overcome the seven year orphan drug market exclusivity of the FDA approved nasal spray product for these label indicated patients aged six years and older in order for Libervant to be granted U.S. market access for patients aged two years and older prior to expiration of the orphan drug market exclusivity period of the nasal spray product, which is scheduled to expire in January 2027; risks and uncertainties inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks and regulatory limitations); risk of development of a sales and marketing capability for commercialization of our product Libervant and other product candidates, including Anaphylm and AQST-108; risks associated with the potential impact on the value of the Company of the sale or outlicensing of our product and product candidates, including Libervant and Anaphylm and other product candidates; risk of insufficient capital and cash resources, including insufficient access to available debt and equity financing, including under our ATM facility, and revenues from operations, to satisfy all of our short-term and longer-term liquidity and cash requirements and other cash needs, at the times and in the amounts needed, including to fund commercialization activities relating to Libervant for patients between two and five years of age and to fund future clinical development and commercial activities for our product candidates, including Anaphylm, AQST-108 and Libervant for patients aged between 6 and 11 years, should these product candidates be approved by the FDA, and for Libervant patients of 6 years and older upon expiration of the orphan drug marketing exclusivity period of the nasal spray product; risk that our manufacturing capabilities will be insufficient to support demand for Libervant for patients between two and five years of age and for older patients, should Libervant receive U.S. market access for these older patients, and for demand for our licensed products in the U.S. and abroad; risk of eroding market share for Suboxone® as a sunsetting product, which accounts for a substantial part of our current operating revenue; risk of default of our debt instruments; risks related to the outsourcing of certain sales, marketing and other operational and staff functions to third parties; risks associated with the rate and degree of market acceptance in the U.S. and abroad of Libervant for label indicated epilepsy patients between two and five years of age, and for older label indicated epilepsy patients, if approved for U.S. market access and after the expiration of the orphan drug market exclusivity period in January 2027; risk of the rate and degree of market acceptance in the U.S. and abroad of Anaphylm, AQST-108 and our other product candidates, should these product candidates be approved by the FDA, and for our licensed products in the U.S. and abroad; risk associated with the size and growth of our product markets; risk associated with our compliance with all FDA and other governmental and customer requirements for our manufacturing facilities; risks associated with intellectual property rights and infringement claims relating to our products; risk that our patent applications for our product candidates, including for Anaphylm and AQST-108, will not be timely issued, or issued at all, by the U.S. Patent and Trademark Office; risk of unexpected patent developments; risk of legislation and regulatory actions and changes in laws or regulations affecting our business including relating to our products and product candidates and product pricing, reimbursement or access therefor; risk of loss of significant customers; risks related to claims and legal proceedings against us including patent infringement, securities, business torts, investigative, product safety or efficacy and antitrust litigation matters; risk of product recalls and withdrawals; risks related to any disruptions in our information technology networks and systems, including the impact of cybersecurity attacks; risk of increased cybersecurity attacks and data accessibility disruptions due to remote working arrangements; risk of adverse developments affecting the financial services industry; risks related to inflation and changing interest rates; risks related to the impact of other pandemic diseases, such as COVID-19, on our business, including with respect to our clinical trials and the site initiation, patient enrollment and timing and adequacy of those clinical trials, regulatory submissions and regulatory reviews and approvals of our product candidates, availability of pharmaceutical ingredients and other raw materials used in our products and product candidates, supply chain, manufacture and distribution of our products and product candidates; risks and uncertainties related to general economic, political (including the Ukraine and Israel wars and other acts of war and terrorism), business, industry, regulatory, financial and market conditions and other unusual items; risks related to uncertainty about presidential administration initiatives and their impact on our business; and other uncertainties affecting us including those described in the "Risk Factors" section and in other sections included in the Company’s 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K filed with the U.S. Securities and Exchange Commission. Given those uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date made. All subsequent forward-looking statements attributable to the Company or any person acting on its behalf are expressly qualified in their entirety by this cautionary statement. The Company assumes no obligation to update forward-looking statements or outlook or guidance after the date of this press release whether as a result of new information, future events or otherwise, except as may be required by applicable law. PharmFilm®, Libervant®, Sympazan® and the Aquestive logo are registered trademarks of Aquestive Therapeutics, Inc. All other registered trademarks referenced herein are the property of their respective owners. Investor inquiries:Astr PartnersBrian Korbbrian.korb@asterpartners.com