What changed in this filing

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Item 1. Business

2026-03-04 compared with 2025-03-05 · 153 added, 130 removed, 519 unchanged (35% of the section changed)

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Aquestive is a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies.

We are developing pharmaceutical products to deliver complex molecules through alternative administrations to invasive and inconvenient standard of care therapies.

We have a proprietary commercial product, Libervant® (diazepam) Buccal Film for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern in patients with epilepsy between two to five years of age, which was launched in April 2024.

We are also advancing a product pipeline for the treatment of severe allergic reactions, including anaphylaxis, under the trade name "Anaphylm™", and our Adrenaverse epinephrine prodrug pipeline platform.

We have five licensed commercialized products which are marketed by our licensees in the U.S.

We are developing pharmaceutical products to deliver complex molecules through administrations that are alternatives to invasive and inconvenient standard of care therapies.

We are advancing our late stage non-device based epinephrine prodrug product candidate for the treatment of severe allergic reactions, including anaphylaxis, under the "Anaphylm™" trade name, and our AdrenaVerse™ epinephrine prodrug pipeline platform.

We have four licensed commercialized products which are marketed by our licensees in the U.S.

and around the world.

We are the exclusive manufacturer of these licensed products.

We also collaborate with pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and have proven drug development and commercialization capabilities.

Aquestive 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.

Our production facilities are located in Portage, Indiana, and our corporate headquarters and primary research laboratory facilities are based in Warren, New Jersey.

We manufacture licensed products at our facilities and anticipate that our current manufacturing capacity is sufficient for commercial quantities of our proprietary and licensed products and product candidates currently in development.

We manufacture licensed products at our facilities and anticipate that our current manufacturing capacity is sufficient for commercial quantities of our licensed products and proprietary product candidates currently in development.

Our facilities have been inspected by the FDA, TGA, and DEA, and are subject to inspection by all applicable health agencies, including ANVISA and EMA.

Not all proprietary or collaborative or licensed products of the Company that may be commercially launched in the future will necessarily be manufactured by us.

PharmFilm® – Our Oral Film Technology

We are presently the worldwide leader in oral film drug delivery and manufacturing, having historically supplied the substantial majority of the world’s oral films for prescription pharmaceutical use, and we have the capability to produce more than one billion commercial doses a year.

We are presently the worldwide leader in oral film drug delivery and manufacturing, having historically supplied the substantial majority of the world’s oral films for prescription pharmaceutical use and having shipped more than two billion doses to patients worldwide.

We developed our PharmFilm technology to provide meaningful clinical and therapeutic advantages over other existing dosage forms and, in turn, to improve the lives of patients and caregivers.

PharmFilm is protected by our patent portfolio, which currently includes at least 110 issued patents worldwide, of which at least 28 are U.S.

patents, and more than 150 pending patent applications worldwide.

Several of the patents in this intellectual property portfolio are utilized in each of our proprietary pipeline products and certain of these patent and patent application cover non-film applications (topical).

We are continuing to develop additional intellectual property and know-how related to the applications and engineering of PharmFilm alone or in combination with other technologies to create product capabilities that have compelling value propositions.

PharmFilm is comprised of proprietary polymer compositions that serve as film formers to APIs and excipients in place.

Proprietary and patent-protected compositions, formulations and manufacturing techniques and technology are employed to ensure that the API is distributed uniformly throughout the film and that target absorption levels are achieved.

Proprietary and patent-protected compositions, formulations and manufacturing techniques and technology, and methods of treatment are employed to ensure that the API is distributed uniformly throughout the film and that target absorption levels are achieved.

Our proprietary technology and manufacturing processes enable PharmFilm to be engineered to fit a variety of target product profiles in order to best address unmet patient needs present within specific disease states.

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• customizable taste profiles.

A drenaverse ™ – Our Epinephrine Prodrug Technology

A drenaVerse ™ – Our Epinephrine Prodrug Platform Technology

Our AdrenaVerse™ platform 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.

Epinephrine plays a critical role in immune suppression but, until now, its role has been limited due to issues in the absorption and conversion of epinephrine in the human body.

We believe that our Adrenaverse platform has demonstrated the ability to harness the therapeutic potential of epinephrine through highly differentiated prodrug formulations, which can achieve absorption, provide sustained local exposure and avoid systemic exposure.

We believe that our AdrenaVerse platform has demonstrated the ability to harness the therapeutic potential of epinephrine through highly differentiated prodrug formulations, which can achieve absorption, provide sustained local exposure and reduce systemic exposure.

The AdrenaVerse platform makes it possible to deliver epinephrine locally across mucosal surfaces and the skin and, therefore, we believe that it has the potential to yield multiple product candidates focused on treating a range of diseases.

The Company’s pipeline includes Anaphylm™ (epinephrine) Sublingual Film and AQST-108 (epinephrine) Topical Gel, which are both product candidates emerging from the Company’s Adrenaverse epinephrine prodrug platform.

The Company’s pipeline includes Anaphylm™ (dibutepinephrine) sublingual film and AQST-108 (epinephrine) topical gel, which are both product candidates emerging from the Company’s AdrenaVerse epinephrine prodrug platform.

Our Product Portfolio and Pipeline

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All other registered trademarks referenced herein are the property of their respective owners.

The FDA conditionally accepted the proprietary name Anaphylm™ (pronounced “ana-film”) as the proposed brand name for Anaphylm.

Final approval of the Anaphylm proprietary name is conditioned on FDA approval of Anaphylm, if any.

Proprietary Growth Drivers

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The active programs in our complex molecule pipeline portfolio are:

• Anaphylm (epinephrine) Sublingual Film – the first and only non-device based, orally delivered epinephrine product candidate in development that has shown clinical results comparable to auto-injectors (such as EpiPen ® and Auvi-Q ® ) for the emergency treatment of allergic reactions, including anaphylaxis.

Epinephrine is the standard of care in the treatment of anaphylaxis and is typically administered via intramuscular injection (IM), including manual auto-injectors such as EpiPen and Auvi-Q, which require patients or their caregivers to inject epinephrine into the patient’s thigh during an emergency allergic reaction.

• Anaphylm ® (dibutepinephrine) sublingual film – the first and only non-device based, orally delivered epinephrine prodrug product candidate in development that has shown clinical results comparable to auto-injectors (such as EpiPen ® and Auvi-Q ® ) for the emergency treatment of allergic reactions, including anaphylaxis.

Epinephrine is the standard of care in the treatment of anaphylaxis and is typically administered via intramuscular injection, including manual auto-injectors such as EpiPen and Auvi-Q, which require patients or their caregivers to inject epinephrine into the patient’s thigh during an emergency allergic reaction.

As a result of this route of administration, many patients and their caregivers are reluctant to use injectable products.

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However, Anaphylm would, if approved by the FDA, allow a patient to simply place a dissolvable strip, approximately the size and weight of a postage stamp, under the tongue, providing an appropriate medication where it is needed and when it is needed.

The FDA conditionally accepted the proprietary name Anaphylm™ (pronounced “ana-film”) as the proposed brand name for Anaphylm.

Final approval of the Anaphylm proprietary name is conditioned on FDA approval of Anaphylm, if any.

Recent Regulatory Updates

Aquestive completed the Anaphylm NDA submission to the FDA in the first quarter of 2025.

On June 16, 2025, the NDA submission was accepted by the FDA and a PDUFA target action date of January 31, 2026 was assigned.

The Company was informed in September that the FDA would not hold an Advisory Committee meeting regarding the approval of Anaphylm.

Throughout the review period the Company was engaged with and responded to information requests from the FDA.

As the PDUFA data approached, the FDA notified the Company that it had identified deficiencies in the NDA that precluded discussion of labeling and post-marketing commitments and that the FDA review remained ongoing.

The FDA did not provide further information about the deficiencies, despite the Company's efforts to be informed of and resolve the issues.

On January 30, 2026, the Company received a CRL that focused on administration and labeling guidance.

The FDA cited deficiencies in the Anaphylm HF validation study.

These included instances of difficulty opening the pouch and incorrect film placement which, if unaddressed, the FDA believes could cause significant safety issues in the setting of anaphylaxis.

To resolve the FDA’s concerns, the Company has modified the pouch opening, instructions for use, pouch and carton labeling, and plans to conduct a new HF validation study with these modifications.

The Company also plans to further address potential tolerability issues in its resubmission of the NDA.

Clinical trial results submitted as part of the NDA regarding comparability to approved auto-injectors ( such as EpiPen ® and Auvi-Q ® ) , such as bracketing, repeat dose, and sustainability, were not questioned in the CRL.

In addition, there were also no CMC issues noted in the CRL.

Due to the requirements related to HF, FDA's clinical pharmacology division requested a single PK study to understand the impact of any modifications to packaging and labeling.

The Agency indicated that the HF and PK studies can be conducted in parallel.

No additional studies were requested in the CRL.

The Company plans to closely work with the Agency to achieve approval for Anaphylm as expeditiously as possible.

As an initial step, the Company requested a Type A meeting with the FDA to discuss the most efficient path forward for resubmission.

Based on its initial review of the CRL, the Company estimates resubmission of the NDA in Q3 2026, assuming completion of the HF and PK studies and expected typical response times from the FDA.

The Company plans to request accelerated review of the resubmission by the FDA, but no expedited review by the FDA can be guaranteed.

The Company is concurrently pursuing regulatory strategies outside the United States.

The Company received positive feedback from the EMA that no further clinical trials are needed prior to regulatory approval submission.

Aquestive is working towards submission of its marketing authorization application in Europe as well as its NDS in Canada in 2026.

The Company also expects to receive feedback from the MHRA in the United Kingdom in the coming weeks.

The Company believes that these markets represent important opportunities to expand access to the Company's non-invasive epinephrine therapy globally.

Clinical Development of Anaphylm

The Company believes that the original Anaphylm NDA submission is supported by a comprehensive clinical development program consisting of eleven independent clinical studies with approximately 967 total administrations across 411 subjects, including 840 single-dose and 127 repeat-dose exposures of Anaphylm.

As part of the clinical development program, Aquestive conducted a first-of-its-kind oral allergy syndrome study, which demonstrated Anaphylm's performance in a real-world, allergen-induced setting.

The program demonstrated that Anaphylm delivers a PK profile comparable to the leading epinephrine auto-injectors.

These studies showed that Anaphylm was generally well-tolerated and had a safety profile similar to that of epinephrine.

On February 24, 2022, following a Phase 1 clinical study conducted by the Company outside of the U.S., the FDA cleared our IND for Anaphylm, allowing for clinical investigation of Anaphylm in the U.S.

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These studies demonstrated that treatment with Anaphylm was well tolerated, with no serious adverse events, significant medical events, or treatment-related severe adverse events reported.

The data from these clinical studies formed the basis for the EoP2 meeting with the FDA in December of 2022, which provided clarity as to the FDA’s

expectations regarding key clinical program areas for design of revised dosing instructions expected for use in our pivotal clinical trial.

The data from these clinical studies formed the basis for the EOP2 meeting with the FDA in December of 2022, which provided clarity as to the FDA’s expectations regarding key clinical program areas for design of revised dosing instructions expected for use in our pivotal clinical trial.

In the fourth quarter of 2023, we received comments from the FDA on the protocol for our pivotal clinical study for Anaphylm, which comments indicated that our proposed endpoints, sample size, and statistical analysis for the proposed pivotal clinical study were reasonable and provided clarity on PK sustainability with repeat-dose requirements.

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In those comments, the FDA did not outline any new clinical development requirements for the Anaphylm program.

The FDA reserved judgement on the sufficiency of the Anaphylm clinical development program until completion of ongoing and planned studies, the results of which were presented at a pre-NDA interaction with the FDA on November 22, 2024.

The FDA reserved judgment on the sufficiency of the Anaphylm clinical development program until completion of ongoing and planned studies, the results of which were presented at a pre-NDA interaction with the FDA on November 22, 2024.

In March 2024, we released topline data from our pivotal clinical study for Anaphylm.

The two-part, Phase 3, single-center, open-label, randomized study was designed to compare the PK and PD of single and repeat doses of Anaphylm versus single and repeat doses of the IM injection and epinephrine autoinjectors (EpiPen® and Auvi-Q®) in healthy adult subjects.

The results of this study demonstrated that the primary endpoint of epinephrine PK biocomparability of the single administration of Anaphylm to the single administration of Adrenalin (epinephrine IM injection) and epinephrine autoinjectors in healthy adult subjects was met, as well as the secondary endpoints, which included evaluating the PK sustainability of Anaphylm following repeat administration and the safety and tolerability of Anaphylm following single and repeat administrations versus epinephrine IM injection and epinephrine autoinjectors.

The results of this study demonstrated that the primary endpoint, epinephrine PK biocomparability of the single administration of Anaphylm to the single administration of Adrenalin (epinephrine IM injection) and epinephrine autoinjectors in healthy adult subjects was met.

The study also met its secondary endpoints, which included evaluating the PK sustainability of Anaphylm following repeat administration, as well as its safety and tolerability of Anaphylm following single and repeat administrations versus epinephrine IM injection and epinephrine autoinjectors.

I n June 2024, we reported positive topline PK data from the Company's temperature / pH study of Anaphylm.

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In July 2024, we reported positive topline data from the self-administration PK study of Anaphylm.

The single-dose, three-period, randomized crossover study was designed to compare the PK and PD of Anaphylm self-administered, Anaphylm healthcare provider (HCP)-administered, and Adrenalin IM injection HCP- administered.

The single-dose, three-period, randomized crossover study was designed to compare the PK and PD of Anaphylm self-administered, Anaphylm HCP-administered, and Adrenalin IM injection HCP-administered.

The primary PK parameters were the Cmax and the AUC exposures, at predefined time points after dosing in 36 healthy adult subjects.

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The safety profile was favorable, with all adverse events classified as mild to moderate and resolving without medical intervention.

Also in October 2024, at the ACAAI 2024 Annual Meeting in Boston, we presented results from a subsequent analysis of our pivotal study data demonstrating Anaphylm's consistent PK and PD profile regardless of variable placement or intraoral movement.

Also in October 2024, at the American College of Allergy, Asthma and Immunology 2024 Annual Meeting, we presented results from a subsequent analysis of our pivotal study data demonstrating Anaphylm's consistent PK and PD profile regardless of variable placement or intraoral movement.

The analysis showed that 87.5% of subjects maintained consistent film placement during disintegration.

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On November 22, 2024, we received positive pre-NDA written response feedback from the FDA prior to our planned NDA submission in the first quarter of 2025.

The FDA did not indicate in those responses that any additional adult clinical trials would be necessary for submitting the NDA for Anaphylm.

The FDA did not indicate in those responses that any additional adult clinical trials would be necessary for submitting the NDA for Anaphylm, although there can be no guarantee that the FDA will not require that additional clinical studies be performed for approval of Anaphylm.

In addition, the FDA agreed with our planned NDA content and format for the submission, planned safety evaluation, and planned pediatric trial.

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In addition, the FDA requested minor modifications to the pediatric trial protocol, which requested modifications were incorporated in the final pediatric trial protocol.

Finally, the FDA noted that due to the new route of administration and the data supporting this route of administration, an advisory committee meeting may be necessary prior to FDA approval and we are preparing for this potential request from the FDA.

We commenced the NDA submission to the FDA in the first quarter of 2025.

The pediatric study in subjects from the ages of 7 to 17 (weight greater than or equal to 30 kgs) is currently underway and the initial study results appear in line with expectations.

We are planning on initiating a product launch of Anaphylm, if approved by the FDA, in the first quarter of 2026.

This is based on completing the NDA submission with the FDA in the coming weeks.

• AQST-108 (epinephrine) Topical Gel – Our product candidate, AQST‑108 is composed of the prodrug dipivefrin which is enzymatically cleaved into epinephrine after administration.

AQST‑108, is a topically delivered adrenergic agonist prodrug, which we believe has the potential to support the re-establishment of immune privilege in the hair follicle and we are pursuing its development for the treatment of alopecia areata, which is an autoimmune disease leading to hair loss on the scalp, face and, in more severe cases, other body areas.

Finally, the FDA noted that due to the new route of administration and the data supporting this route of administration, an advisory committee meeting may be necessary prior to FDA approval, although the FDA recently confirmed as more fully described below that it would not hold an advisory committee.

The pediatric study in subjects from the ages of 7 to 17 (weight greater than or equal to 30 kgs) was completed with positive topline data reported on April 1, 2025.

A total of thirty-two patients completed the study.

The PK results were consistent with previous adult studies.

Anaphylm was shown to be safe and well-tolerated with no serious adverse events reported in this pediatric study.

• AQST-108 (epinephrine) topical gel – Our product candidate, AQST‑108 is generated from our 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.

Epinephrine plays a critical role in immune suppression but, until now, its role has been limited due to issues in the absorption and conversion of epinephrine in the human body.

AQST-108 is composed of the prodrug dipivefrin which is enzymatically cleaved into epinephrine after administration.

AQST‑108 is a topically delivered adrenergic agonist prodrug, which we believe has the potential to support the re-establishment of immune privilege in the hair follicle and we are pursuing its development for the possible treatment of alopecia areata, which is an autoimmune disease leading to hair loss on the scalp, face and, in more severe cases, other body areas.

We completed the first human clinical trial for AQST-108.

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In Part 2, the calculated percentage of AQST-108 observed in the skin remained consistent across all studied formulations and zero post-dose AQST-108 concentrations in plasma were observed.

We received pre-IND FDA feedback in the fourth quarter of 2024 to align on the Phase 2a clinical trial design and plan to open and IND and initiate the Phase 2a clinical trial in the second quarter of 2025.

We opened an IND for this product candidate in the fourth quarter of 2025.

We are continuing pre-clinical development and toxicology studies for AQST-108.

Dosing of subjects in a second Phase 1 clinical trial was successfully completed in the first quarter of 2026 and the data readout from the study is expected in Q2 2026.

This study is intended to further characterize the safety, tolerability, and pharmacologic profile of the program and to inform potential future development opportunities, including indication selection.

Proprietary CNS Product

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• Libervant ® – a buccally, or inside of the cheek, administered soluble film formulation of diazepam, Libervant was developed as an alternative to device-dependent rescue therapies currently available to patients with refractory epilepsy, which are a rectal gel and nasal sprays.

On April 26, 2024, the FDA approved Libervant for U.S.

On April 26, 2024, the FDA approved Libervant ® (diazepam) Buccal Film for U.S.

market access 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 in patients with epilepsy between two to five years of age.

, seizure clusters, ARS) that are distinct from a patient’s usual seizure pattern in patients with epilepsy between two to five years of age.

Libervant is the first and only orally administered rescue product for the treatment of seizure cluster in patients between ages two to five.

The only other current FDA approved product for these ARS patients between two to five years of age is a diazepam rectal gel.

The only other current FDA approved products for these ARS patients between two to five years of age is a diazepam rectal gel and a diazepam nasal spray.

In October 2024, Libervant 5mg, 7.5mg, 10mg, 12.5mg and 15 mg for ARS patients between two and five years of age became available through multiple retail distribution channels.

Market access activities have broadened coverage.

Libervant is available nationwide with retail distribution capabilities in place.

For Medicaid patients, Libervant is available in all states.

Commercial patient access to Libervant continues to expand based on health plan reviews and Pharmacy Benefit Manager agreements.

Prior to the FDA approval of Libervant for ARS patients between two to five years, the FDA granted tentative approval in August 2022 for Libervant for the same indication in patients with epilepsy 12 years of age and older, finding that Libervant had met all required quality, safety, and efficacy standards for approval.

However, due to an existing FDA regulatory grant of orphan drug market exclusivity for a diazepam nasal spray product sold by another

company for use in ARS patients 6 years of age and older, the FDA determined that Libervant was not yet eligible for marketing in the United States for this patient population of 12 years of age and older.

We expect to file for FDA approval for use of Libervant for these ARS patients aged between 6 and 12 years prior to the expiration of the orphan drug marketing exclusivity block of the nasal spray product.

As a result of the orphan drug marketing exclusivity granted by the FDA to the nasal spray product, the FDA cannot give final approval for U.S.

market access for Libervant for any age group of 6 and above until the expiration or inapplicability of the orphan drug market exclusivity.

However, overcoming the orphan drug marketing exclusivity determination is difficult to establish, with limited precedent, and there can be no assurance that the FDA will agree with our position seeking to overcome such market exclusivity and approve Libervant for U.S.

market access for this age group of 6 years and older earlier than January 2027, the scheduled date for expiration of orphan drug market exclusivity for the FDA nasal spray product.

See “Licensed Commercial Products, Product Candidates and Other Products – Libervant” for a discussion of the licensing arrangement for Libervant.

In the fourth quarter of 2024 the FDA granted seven years of ODE to Libervant® (diazepam) Buccal Film for ARS patients between two to five years of age.

Libervant was originally granted Orphan Drug Designation on November 10, 2016 and the ODE now granted to Libervant for patients ages two to five extends to April of 2031.

In the fourth quarter of 2024, the FDA granted seven years of ODE to Libervant for ARS patients between two to five years of age.

Libervant was originally granted Orphan Drug Designation on November 10, 2016.

On February 14, 2025, in a lawsuit brought by Neurelis, Inc.

("Neurelis") against the FDA (Neurelis, Inc.

("Neurelis"), the owner of the FDA approved nasal spray Valtoco, against the FDA (Neurelis, Inc.

Califf, for which the Company joined as a Defendant Intervenor) challenging the FDA's approval of Libervant for ARS patients aged between two and five years, the U.S.

District for the District of Columbia issued a final appealable order entering a judgment in favor of Neurelis's motion for summary judgement and vacating the FDA’s approval of Libervant.

The District Court's ruling was not based on grounds of safety or efficacy of Libervant, but rather on the grounds that the law granting ODE to the FDA approved nasal spray Valtoco for patients aged six and older should be interpreted to extend to children aged two to five, despite that Valtoco was not approved by the FDA to treat these younger patients.

The Company is appealing this ruling and has filed a motion to stay the court's ruling pending the outcome of the appeal.

The Company is continuing to market and sell Libervant in the ordinary course, pending a final non-appealable ruling in this matter.

On February 24, 2025, Aquestive filed a request with the FDA that the FDA also confirms approval of Libervant for ARS patients aged between two and five years on the FDA regulatory grounds of clinical superiority over the other currently existing FDA approved ARS drugs, including confirming the FDA grant of ODE for Libervant for ARS patients aged between two and five years.

District for the District of Columbia issued a final appealable order entering a judgment in favor of Neurelis's motion for summary judgment and vacating the FDA’s approval of Libervant.

The District Court's ruling was not based on grounds of safety or efficacy of Libervant, but rather on the grounds that the law granting ODE to the FDA approved nasal spray Valtoco for patients aged six years and older should be interpreted to extend to children aged two to five years, despite that Valtoco was not approved by the FDA to treat these younger patients at the time the FDA approved Libervant for this pediatric age group.

The FDA is appealing this ruling.

As a result of the District Court ruling, the FDA converted the approval of Libervant for patients aged between two and five years to a "tentative approval" and Aquestive has ceased marketing activities for Libervant in the United States.

On February 24, 2025, Aquestive filed a request with the FDA that the FDA also confirm approval of Libervant for ARS patients aged between two and five years on the FDA regulatory grounds of clinical superiority over the other currently existing FDA approved ARS drugs.

FDA’s orphan drug regulations define a “clinically superior” drug as “a drug shown to provide a significant therapeutic advantage over and above that provided by an approved orphan drug (that is otherwise the same drug)” in one of three ways:

the basis of greater efficacy or safety, or providing a major contribution to patient care.

While the Company is not able 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, the Company intends to vigorously defend against any decision that jeopardizes the on-label patient access to Libervant.

See Part II, Item 8.

Financial Statements and Supplementary Data, Note 23, Contingencies.

Licensed Commercial Products and Product Candidates and Other Products

The FDA has taken this request under advisement and has not yet provided a response to the Company.

However, due to the existing FDA regulatory grant of ODE for Valtoco® for use in ARS patients 6 years of age and older, the FDA determined that Libervant was not yet eligible for marketing in the United States for this patient population of 12 years of age and older.

We expect to file for FDA approval for use of Libervant for these ARS patients aged between 6 and 11 years prior to the expiration of the ODE for Valtoco.

However, as a result of the ODE granted by the FDA to Valtoco and the District Court's ruling, the FDA cannot give final approval for U.S.

market access for Libervant for any age group until the expiration of the ODE or a determination by the FDA of inapplicability of the ODE for Libervant, unless the District Court's ruling vacating the FDA approval of Libervant for ARS patients aged between two and five years is overturned on appeal.

In the event that the District Court's ruling is reversed without further right of appeal, and the tentative approval of Libervant for ARS patients aged between two and five is converted to a final approval by the FDA, the Company would only be able to market Libervant for ARS patients aged between two and five years and would continue to be restricted from market access of Libervant for older ARS patients until the expiration of the ODE for Valtoco.

market access for any age group earlier than January 2027, the scheduled date for expiration of ODE for Valtoco.

See “ Licensed Commercial Products, Product Candidates and Other Products – Libervant ” for a discussion of the licensing arrangement for Libervant.

See " Note 23, Contingencies - Neurelis FDA Lawsuit" for a discussion of the FDA case and new legislation clarifying the Orphan Drug Act.

Licensed Commercial Products, Product Candidates and Other Products

Our portfolio also includes other products and product candidates that we have licensed, or will seek to license, or for which we have licensed our intellectual property for commercialization.

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• Suboxone ® – a sublingual film formulation of buprenorphine and naloxone, respectively an opioid agonist and antagonist, that is marketed in the United States and internationally for the treatment of opioid dependence.

Suboxone was launched by our licensee, Indivior Inc., or Indivior, in 2010.

Suboxone was launched by our licensee, Indivior, in 2010.

Suboxone is the most prescribed branded product in its category and was the first sublingual film product for the treatment of opioid dependence.

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Zambon is responsible for the regulatory approval and marketing of Emylif in the countries where Zambon seeks to market the product and Aquestive is responsible for the development and manufacture of the product.

During the second quarter of 2023, Aquestive received a $0.5 million milestone payment in connection with the first commercial sale in the first country in the licensed territory for Emylif pursuant to the terms of the license agreement with Zambon.

During the second quarter of 2025, Aquestive earned a $500 milestone payment in connection with the sale of Emylif pursuant to the terms of the license agreement with Zambon.

In January 2021, we announced that we granted an exclusive license to MTPA for the commercialization in the United States of Exservan.

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Aquestive was the exclusive sole manufacturer and supplier for Exservan in China.

Under the terms of the license agreement with Haisco, as amended, Aquestive received a $7.0 million upfront payment in September 2022, and was to receive regulatory milestone payments, double-digit royalties on net sales of Exservan in China, and earn manufacturing revenue upon the sale of Exservan in China.

Under the terms of the license agreement with Haisco, as amended, Aquestive received a $7,000 upfront payment in September 2022, and was to receive regulatory milestone payments, double-digit royalties on net sales of Exservan in China, and earn manufacturing revenue upon the sale of Exservan in China.

In June 2024, Aquestive and Haisco mutually agreed to terminate the Haisco Agreement.

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Under the Pharmanovia Agreement, Pharmanovia will lead the regulatory and commercialization activities for Libervant in the Territory and Aquestive will serve as the exclusive sole manufacturer and supplier of Libervant in the Territory.

We received $3.5 million upon agreement execution.

received $3,500 upon agreement execution.

Effective March 27, 2023, we amended the Pharmanovia Agreement to expand the scope of the licensed territory for Libervant to cover the rest of the world, excluding the U.S., Canada and China.

Pharmanovia will be responsible for seeking appropriate regulatory approval in the expanded territories.

Pursuant to the terms of the Pharmanovia Amendment, we received a non-refundable payment of $2.0 million from Pharmanovia on execution of the Pharmanovia Amendment.

Pursuant to the terms of the Pharmanovia Amendment, we received a non-refundable payment of $2,000 from Pharmanovia on execution of the Pharmanovia Amendment.

• Sympazan ® – an oral soluble film formulation of clobazam used for the treatment of seizures associated with a rare, intractable form of epilepsy known as Lennox-Gastaut syndrome, or LGS, in patients aged two years of age or older, was approved by the FDA on November 1, 2018.

We commercially launched Sympazan in December 2018.

On October 26, 2022, we entered into a License Agreement with Otter Pharmaceuticals, LLC, a subsidiary of Assertio Holdings, Inc., pursuant to which we granted an exclusive, worldwide license of its intellectual property for Sympazan to Assertio during the term of that agreement for an upfront payment of $9.0 million.

Additionally, we subsequently received from Assertio a $6.0 million milestone payment upon its receipt of a notice of allowance from the United States Patent and Trademark Office of its patent application U.S.

Additionally, we subsequently received from Assertio a $6,000 milestone payment upon its receipt of a notice of allowance from the United States Patent and Trademark Office of its patent application U.S.

16/561,573, and payment of the related allowance fee.

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• KYNMOBI ® – a sublingual film formulation of apomorphine, which is a dopamine agonist, was developed to treat episodic off-periods in Parkinson’s disease.

We licensed our intellectual property to Cynapsus Therapeutics, Inc., a company that was acquired by Sunovion for the commercialization of KYNMOBI under the Sunovion License

We licensed our intellectual property to Cynapsus Therapeutics, Inc., a company that was acquired by Sunovion for the commercialization of KYNMOBI under the Sunovion License Agreement.

KYNMOBI was approved by the FDA on May 21, 2020 and commercially launched by Sunovion in September 2020.

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Pursuant to the terms of the March 2012 agreement with Zevra, we began to receive milestone and royalty revenues for Azstarys.

See Part II Item 8.

Financial Statements and Supplementary Data, Note 7, Material Agreements for details.

Market Overview

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Auto-injectors can be inconvenient to transport and many patients and caregivers dislike injections as a delivery method.

In August 2024, a non-injection-based epinephrine delivery device, Neffy ®, was approved by the FDA and EMA for the treatment of severe allergic reactions, including anaphylaxis, and is in the early phase of its commercial launch.

In August 2024, a non-injection-based epinephrine delivery device, n effy ®, was approved by the FDA and EMA for the treatment of severe allergic reactions, including anaphylaxis, and is available to patients in the United States and certain other countries.

Proper dosing and the ability to effectively administer epinephrine in a timely, reliable manner is critical for patients experiencing anaphylaxis.

However, we believe that the inability to administer complex molecules via oral administration has limited the development of treatments that have the potential to provide significant patient benefit.

However, we believe that the inability to administer complex molecules via oral administration has

limited the development of treatments that have the potential to provide significant patient benefit.

We designed Anaphylm, a “first of its kind” oral sublingual film formulation delivering systemic epinephrine, as a rescue medicine for the treatment of anaphylaxis, using Aquestive’s proprietary PharmFilm technologies.

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Chronic medicines are used on a daily basis to suppress seizure activity.

Approximately 1.1 million of those suffering from epilepsy will continue to suffer

with breakthrough seizures and may require an acute (rescue) management strategy.

Patients are routinely prescribed antiepileptic drugs, or AEDs, as “maintenance” therapy to control chronic seizure activity.

Approximately 1.1 million of those suffering from epilepsy will continue to suffer with breakthrough seizures and may require an acute (rescue) management strategy.

Patients are routinely prescribed AEDs, as “maintenance” therapy to control chronic seizure activity.

Most AEDs specifically target neuronal excitation or neuronal inhibitory pathways.

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Rectal administration of this drug presents a particular challenge for patients.

We developed our product candidate Libervant as an alternative to the device-dependent rescue therapies currently available to patients with refractory epilepsy and Libervant® is currently available for patients between ages two to five.

We developed our product candidate Libervant as an alternative to the device-dependent rescue therapies currently available to patients with refractory epilepsy.

See “ Our Product Portfolio and Pipeline ” above and “ Competition ” below in this Item 1.

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Patients with LGS are often drug resistant, predisposing them to recurrent seizures, and are typically prescribed a combination of antiepileptic medications, which often includes clobazam.

Clobazam (branded name Onfi) is available in both a tablet and suspension formulation.

Clobazam (branded name Ondif) is available in both a tablet and suspension formulation.

Generic versions of the clobazam tablet and suspension formulation are available to patients, as well.

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Manufacturing and Product Supply

We operate two manufacturing and primary packaging facilities located in Portage, Indiana, where we currently manufacture our licensed products, Suboxone, Emylif, Ondif and Sympazan, and our proprietary product, Libervant for patients between two to five years of age, on an exclusive basis.

We operate two manufacturing and primary packaging facilities located in Portage, Indiana, where we currently manufacture our licensed products, Suboxone, Emylif, Ondif and Sympazan.

These facilities are expected to have a combined capacity to accommodate the production of our proprietary product pipeline candidate and licensed products, without any current need for additional infrastructure.

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We will also continue to consider our anticipated facilities and infrastructure needs as our product development grows.

We have produced over 1.0 billion doses in the last five years.

We have produced over 2.5 billion doses since the Company's inception.

The cGMP manufacturing operations in Indiana are registered with the DEA for Schedule II - V.

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This industry is highly competitive and new products and technologies evolve and come to market at a rapid pace.

The companies operating in this market include multinational organizations,

established biotechnology companies, single product pharmaceutical and biotechnology companies, specialty pharmaceutical companies, and generic drug companies.

The companies operating in this market include multinational organizations, established biotechnology companies, single product pharmaceutical and biotechnology companies, specialty pharmaceutical companies, and generic drug companies.

Many of the larger, established organizations currently have commercialization capabilities in-house, and may have partnership or license agreements in place with smaller companies for commercialization rights.

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Among these are needle device-based treatments that have been accepted as standard of care treatment for many years, including autoinjectors such as EpiPen ® marketed by Teva, and generics of EpiPen marketed by Viatris, Inc., Adrenaclick ® marketed by Amneal Pharmaceuticals, LLC, and Auvi-Q ® marketed by Kaleo Inc.

and syringe device Symjepi® marketed by US Worldmeds.

and syringe device Symjep ® marketed by U.S.

In August 2024, ARS Pharmaceuticals, Inc.

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There are other non-needle device based treatments in earlier phases of development that have not yet been approved for marketing in the United States by the FDA.

Anaphylm is the first and only orally delivered epinephrine product candidate in development to demonstrate clinical results comparable to autoinjectors such as EpiPen and Auvi-Q for the emergency treatment of allergic reactions, including anaphylaxis.

We believe that Anaphylm is the first and only orally delivered epinephrine prodrug product candidate in development to demonstrate clinical results comparable to autoinjectors such as EpiPen and Auvi-Q for the emergency treatment of allergic reactions, including anaphylaxis.

We believe Anaphylm is well positioned as a non-device based treatment option for this indication, if approved by the FDA, due to its comparable efficacy to existing auto-injector and nasal spray products, easy portability and convenience of use.

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, safer, more effective, or providing a major contribution to patient care) within the meaning of FDA regulations and guidance.

In assessing whether a drug candidate sponsor has demonstrated that its drug candidate provides a “major contribution to patient care” over and above the currently approved drugs, which is evaluated by the FDA on a case by case basis, there is no single objective standard and the FDA may, in appropriate circumstances, consider such factors as convenience of treatment location, duration of treatment, patient comfort, reduced treatment burden, advances in ease and comfort of drug administration, longer periods between doses, and potential for self-administration.

In assessing whether a drug candidate sponsor has demonstrated that its drug candidate provides a “major contribution to patient care” over and above the currently

approved drugs, which is evaluated by the FDA on a case by case basis, there is no single objective standard and the FDA may, in appropriate circumstances, consider such factors as convenience of treatment location, duration of treatment, patient comfort, reduced treatment burden, advances in ease and comfort of drug administration, longer periods between doses, and potential for self-administration.

In August 2022, the FDA granted tentative approval for Libervant 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 in patients with epilepsy 12 years of age and older.

The FDA concluded that Libervant had met all required quality, safety, and efficacy standards for approval.

However, as a result of the orphan drug marketing exclusivity previously granted by the FDA to a nasal spray product, the FDA cannot give final approval for U.S.

However, as a result of the orphan drug marketing exclusivity previously granted by the FDA to Valtoco, the FDA cannot give final approval for U.S.

market access for Libervant for any age group of 6 years and above until the expiration or inapplicability of the orphan drug market exclusivity scheduled to occur in January, 2027.

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market access for ARS patients between two and five years of age.

Libervant is the first and only orally administered rescue product for the treatment of ARS patients between ages two and five years.

The only other current FDA approved product on the market for these epilepsy patients between two and five years of age is a diazepam rectal gel.

On December 18, 2024, the FDA granted seven years of ODE to Libervant for the treatment of ARS patients between two and five years of age.

In October 2024, Libervant 5mg, 7.5mg, 10mg, 12.5mg and 15 mg for ARS patients between two and five years of age became available through multiple retail distribution channels.

Market access activities have broadened coverage.

Libervant is available nationwide with retail distribution capabilities in place.

For Medicaid patients, Libervant is available in all states.

Commercial patient access to Libervant continues to expand based on health plan reviews and Pharmacy Benefit Manager agreements.

Libervant is the first and only orally administered rescue product for the treatment of ARS patients between ages two and five years.

The only other current FDA approved product on the market for these epilepsy patients between two and five years of age at the time of approval of Libervant for this pediatric age group was a diazepam rectal gel.

On February 14, 2025, in a lawsuit brought by Neurelis, Inc.

("Neurelis") against the FDA ( Neurelis, Inc.

Califf, for which the Company joined as a Defendant Intervenor) challenging the FDA's approval of Libervant for ARS patients aged between two and five years, the U.S.

District for the District of Columbia issued a final appealable order entering a judgment in favor of Neurelis's motion for summary judgement and vacating the FDA’s approval of Libervant.

The District Court's ruling was not based on grounds of safety or efficacy of Libervant, but rather on the grounds that the law granting ODE to the FDA approved nasal spray for ARS patients aged six and older should be interpreted to extend to children aged two to five, despite that Valtoco was not approved by the FDA to treat these younger patients.

The Company is appealing this ruling and has filed a motion to stay the court's ruling pending the outcome of the appeal.

The Company is continuing to market and sell Libervant in the ordinary course, pending a final non-appealable ruling in this matter.

Califf, for which the Company joined as a Defendant Intervenor) challenging the FDA's approval of Libervant for ARS patients aged between two and five years, the court ruled in favor of Neurelis, granting its summary judgment motion, and against the FDA's and the Company's cross-motions for summary judgment.

In an accompanying opinion, the court directed the FDA to vacate the approval of Libervant.

As a result of the District Court's ruling, the FDA converted the approval of Libervant to a "tentative approval" and the Company has ceased marketing activities in the United States.

See Part II, Item 8.

Financial Statements and Supplementary Data, Note 23, Contingencies.

Financial Statements and Supplementary Data, Note 23, Contingencies - Neurelis FDA Lawsuit" for a discussion of the FDA case and new legislation clarifying the Orphan Drug Act.

Material Agreements

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patents, and more than 110 pending patent applications worldwide.

These issued patents and pending patent applications provide both process of making and composition of matter protection for our PharmFilm technology and products and product candidates, including Suboxone, Libervant and Anaphylm and our PharmFilm formulations of diazepam, clobazam, riluzole and epinephrine.

These patents and, if issued as patents, pending patent applications will likely expire between 2024 and 2044.

The pending patent applications will provide composition of matter and process of making protection for our PharmFilm dosage formulations of diazepam and epinephrine and, if issued as patents, will likely expire by 2040 and 2044, respectively.

These issued patents and pending patent applications provide process of making, composition of matter and method of treatment protection for our PharmFilm technology and products and product candidates, including Suboxone®, Libervant® and Anaphylm™ and our PharmFilm formulations of clobazam and riluzole.

These granted patents will likely expire between 2036 and 2037.

The pending patent applications will provide composition of matter, process of making protection and method of treatment for our PharmFilm dosage formulations of diazepam and epinephrine and, if issued as patents, will likely expire by 2037 and 2045, respectively.

The projected expiration dates exclude any patent term adjustment or patent term extension.

PharmFilm ® – Our Oral Film Technology

Our PharmFilm technology is covered by at least 12 patent families.

These patent families provide process, composition of matter protection for our PharmFilm technology, and comprise at least 49 issued patents worldwide, of which at least 12 are U.S.

patents, and related pending patent applications worldwide.

The patents and pending patent applications, if issued as patents, will likely expire between 2024 and 2044, excluding any patent term adjustment or patent term extension.

The PharmFilm ® technology patents and/or patent applications also generically and specifically protect the technology utilized in the products in our CNS programs, our complex molecule programs, as well as our licensee programs.

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Also encompassed within our platform technology is coverage for our complex molecule program which includes molecules such as epinephrine and technology applicable to our product candidate Anaphylm.

Our platform technology patents and/or patent applications further cover the products Libervant, Suboxone and Ondif, as well as our formulations of the molecules apomorphine, which is part of our licensed programs.

Our platform technology patents and/or patent applications further cover the product candidate Libervant™ and Suboxone® and Ondif® licensed products, as well as our formulations of the molecules apomorphine, which is part of our licensed programs.

The expiration dates for patents covering PharmFilm products and product candidates, and for pending applications if issued as patents, extend from 2025 to 2044, excluding any patent term adjustment or patent term extension.

We note that several of our issued patents are or have been involved in administrative proceedings, such as reexamination and inter partes review at the U.S.

We note that several of our issued patents have been involved in administrative proceedings, such as reexamination and inter partes review at the U.S.

Patent and Trademark Office, or USPTO, and opposition at the European Patent Organization, or EPO.

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We may then initiate a patent infringement lawsuit in response to the notice of the Paragraph IV certification, which would automatically prevent the FDA from approving the ANDA or 505(b)(2) NDA until the earlier of 30 months, expiration of the patent, settlement of the lawsuit, or a decision in the infringement case that is favorable to the ANDA or 505(b)(2) NDA applicant.

AdrenaVerse™– Our Epinephrine Prodrug Platform Technology

Our AdrenaVerse™ technology is covered by at least 4 patent families.

These patent families provide process, composition of matter protection for our AdrenaVerse™ technology.

The patents and pending patent applications, if issued as patents by the PTO, will likely expire between 2037 and 2045, excluding any patent term adjustment or patent term extension.

The rest of our patent portfolio largely relates to patents and applications owned by us and directed to our product development portfolio and other product candidates and related compositions and/or manufacturing processes.

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• submission to the FDA of an NDA;

• satisfactory completion of an FDA pre-approval inspection of the facility or facilities at which the product is manufactured to assess compliance with the FDA’s current cGMP regulations to assure that the facilities, methods and controls are adequate to preserve the drug’s identity, strength, quality and purity;

• satisfactory completion of potential clinical site inspection(s) to assure studies are conducted in accordance with Good Clinical Practice (GCP) regulations;

• satisfactory completion of a potential FDA pre-approval inspection(s) of the facility or facilities at which the product is manufactured to assess compliance with the FDA’s current cGMP regulations to assure that the facilities, methods and controls are adequate to preserve the drug’s identity, strength, quality and purity;

• satisfactory completion of a potential review at a public FDA Advisory Committee meeting;

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Under federal law, the submission of most NDAs is subject to a substantial application user fee, and applicant under an approved NDA is also subject to an annual program fee for each prescription drug product, which beginning in Fiscal Year 2018 replaced the product and establishment fees.

The PDUFA defines the metrics FDA must meet as a result of the fee payment.

The FDA has 60 days from its receipt of an NDA to determine whether the application will be accepted for filing based on the Agency’s threshold determination that it is sufficiently complete to permit substantive review.

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Once the submission is accepted for filing, the FDA begins an in-depth substantive review.

Under PDUFA, the FDA has agreed to certain performance goals in the review of NDAs through a two-tiered classification

system, Standard Review and Priority Review.

Under PDUFA, the FDA has agreed to certain performance goals in the review of NDAs through a two-tiered classification system, Standard Review and Priority Review.

Priority Review designation is given to drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists.

The FDA endeavors to review applications subject to Standard Review within ten to twelve months, whereas the FDA’s goal is to review Priority Review applications within six to eight months.

Before approving an NDA, the FDA will typically inspect one or more clinical sites to assure compliance with GCP requirements.

The FDA will not approve the product unless it determines that the manufacturing process and facilities are in compliance with cGMP requirements and are adequate to assure consistent production of the product within required specifications and the NDA contains data that provide substantial evidence that the drug is safe and effective in the indication studied.

After the FDA evaluates the NDA and the manufacturing facilities and possibly conducts a sponsor inspection, it issues either an approval letter or a complete response letter.

A complete response letter generally outlines the deficiencies in the NDA and may require substantial additional testing, or information, in order for the FDA to reconsider the application.

In issuing the complete response letter, the FDA may recommend actions that the applicant might take to place the NDA in condition for approval, including requests for additional information or clarification.

The FDA may delay or refuse approval of an NDA if applicable regulatory criteria are not satisfied, require additional testing or information and/or require post-marketing testing and surveillance to monitor safety or efficacy of a product.

Even with submission of this additional information, the FDA may ultimately decide that an application does not satisfy the regulatory criteria for approval.

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The requirement for a REMS can materially affect the potential market and profitability of a drug.

Moreover, product approval may require substantial post-approval testing and surveillance to monitor the drug’s safety or efficacy, and the FDA has the authority to prevent or limit further marketing of a product based on the results of these post-marketing programs.

marketing programs.

Once granted, product approvals may be withdrawn if compliance with regulatory standards is not maintained, or problems are identified following initial marketing.

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In addition, any distribution of prescription drug products must comply with the PDMA, a part of the FDCA.

In addition, Title II of the Federal Drug Quality and Security Act of 2013, known as the DSCSA has imposed new “track and

trace” requirements on the distribution of prescription drug products by manufacturers, distributors, and other entities in the drug supply chain.

In addition, Title II of the Federal Drug Quality and Security Act of 2013, known as the DSCSA has imposed new “track and trace” requirements on the distribution of prescription drug products by manufacturers, distributors, and other entities in the drug supply chain.

These requirements are being phased in over a ten-year period.

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Orphan designation must be requested before submitting an NDA.

After the FDA grants orphan product designation, the identity of the therapeutic agent and its potential orphan use are disclosed publicly by the FDA.

After the FDA grants orphan product designation, the identity of the therapeutic agent and its

potential orphan use are disclosed publicly by the FDA.

Orphan drug designation, however, does not convey any advantage in, or shorten the duration of, the regulatory review and approval process.

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Other states prohibit various marketing-related activities, such as the provision of certain kinds of gifts or meals.

Still other states require the reporting of certain pricing information, including information pertaining to and justification of price increases, or prohibit prescription drug price gouging.

Still other states require the reporting of certain pricing information, including information pertaining to and justification of price increases, or prohibit

prescription drug price gouging.

In addition, states such as California, Connecticut, Nevada, Massachusetts and the District of Columbia require pharmaceutical companies to implement compliance programs and/or marketing codes.

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In the event governmental authorities conclude that our business practices did not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations, they may impose sanctions under these laws, which are potentially significant and may include civil monetary penalties, damages, exclusion of an entity or individual from participation in government health care programs, criminal fines and individual imprisonment, additional reporting requirements if we become subject to a corporate integrity agreement or other settlement to resolve allegations of violations of these laws, as well as the potential curtailment or restructuring of our operations.

Further, we may be

subject to contractual damages and reputational harm as result of such non-compliance.

Further, we may be subject to contractual damages and reputational harm as result of such non-compliance.

Even if we are not determined to have violated these laws, government investigations into these issues typically require the expenditure of significant resources and generate negative publicity.

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Some states have elected not to expand their Medicaid programs to individuals with an income of up to 133% of the federal poverty level, as is permitted under the PPACA.

For each state that does not choose to expand its Medicaid program,

there may be fewer insured patients overall, which could impact our sales of products for which we receive regulatory approval, business and financial condition.

For each state that does not choose to expand its Medicaid program, there may be fewer insured patients overall, which could impact our sales of products for which we receive regulatory approval, business and financial condition.

Where new patients receive insurance coverage under any of the new Medicaid options made available through the PPACA, the possibility exists that manufacturers may be required to pay Medicaid rebates on drugs used under these circumstances, a decision that could impact manufacturer revenues.

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Such scrutiny has resulted in several recent Congressional inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for products.

For example, the Inflation Reduction Act of 2022 (“IRA”), signed into law by President Biden, included a number of significant drug pricing reforms, which include the establishment of a drug price negotiation program within the HHS (beginning in 2026) that requires manufacturers to charge a negotiated “maximum fair price” for certain selected drugs or pay an excise tax for noncompliance, the establishment of rebate payment requirements on manufacturers under Medicare Parts B and D to penalize price increases that outpace inflation, and a redesign of the Part D benefit, a part of which requires manufacturers to provide discounts on Part D drugs starting in 2025.

Congress has further indicated that they will continue to pursue new legislative and/or administrative measures to control drug costs, including price or patient reimbursement constraints, discounts, restrictions on certain access and marketing cost disclosure and transparency measures, and, in some cases, laws designed to encourage importation from other countries and bulk purchasing.

Each recent presidential administration has enacted and repealed Executive Orders that have impacted prescription drug prices.

In his first term, President Trump enacted Executive Orders directing the Secretary of HHS to eliminate protection under an Anti-Kickback Statute safe harbor for certain retrospective price reductions, ensure that payment by the Medicare program for certain Medicare Part B drugs is not higher than the payment by other comparable countries, allow certain low-income individuals purchase insulin and epinephrine, and implement a rulemaking plan to test a payment model, pursuant to which Medicare would receive most-favored-nation prices ( i.e ., the lowest price) for certain pharmaceutical products.

In his first term, President Trump enacted Executive Orders directing the Secretary of HHS to eliminate protection

under an Anti-Kickback Statute safe harbor for certain retrospective price reductions, ensure that payment by the Medicare program for certain Medicare Part B drugs is not higher than the payment by other comparable countries, allow certain low-income individuals purchase insulin and epinephrine, and implement a rulemaking plan to test a payment model, pursuant to which Medicare would receive most-favored-nation prices ( i.e ., the lowest price) for certain pharmaceutical products.

President Biden enacted Executive Orders to, among other things, reduce barriers to the PPACA marketplace, increase the affordability of the marketplace and increase eligibility for and coverage from the marketplace and Medicaid.

On the first day of President Trump’s second term, he revoked many of President Biden’s Executive Orders, including such Orders impacting Medicaid and the PPACA.

During President Trump’s second term, he revoked many of President Biden’s Executive Orders, including such Orders impacting Medicaid and the PPACA.

It is difficult at this time to predict what future executive or legislative initiatives may impact drug pricing.

At the state level, legislatures are increasingly passing legislation and implementing regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures and, in some cases, designed to encourage importation from other countries and bulk purchasing.

In January 2024, the FDA authorized Florida's Agency for Health Care Administration's drug importation program, which is the first step toward Florida facilitating importation of certain prescription drugs from Canada.

Authorization of other state programs may follow.

We expect that healthcare reform measures that may be adopted in the future could have a material adverse effect on our industry generally and on our ability to maintain or increase sales of products for which we receive regulatory approval or to successfully commercialize our product candidates, if approved.

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Additionally, discounted pricing or rebates on purchases of pharmaceutical products must be offered under various federal and state healthcare programs, including:

the Centers for Medicare & Medicaid Services’ Medicaid Drug Rebate

Program, Medicare Part B Program and Medicare Part D Manufacturer Discount Programs, the U.S.

the Centers for Medicare & Medicaid Services’ Medicaid Drug Rebate Program, Medicare Part B Program and Medicare Part D Manufacturer Discount Programs, the U.S.

Department of Veterans Affairs’ Federal Supply Schedule Program, and the Health Resources and Services Administration’s 340B Drug Pricing Program.

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We are also subject to various laws and regulations regarding laboratory practices, the experimental use of animals, and the use and disposal of hazardous or potentially hazardous substances in connection with our research.

While we believe we are in compliance with applicable environmental and other regulations, in each of these areas, as above, the FDA and other government agencies have broad regulatory and enforcement powers, including, among other things, the ability to levy fines and civil penalties, suspend or delay issuance of approvals, seize or recall products, and withdraw approvals, any one or more of which could have a material adverse effect on us.

and civil penalties, suspend or delay issuance of approvals, seize or recall products, and withdraw approvals, any one or more of which could have a material adverse effect on us.

Human Capital

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All of our colleagues were employed in the U.S.

Of these colleagues, 22 are directly involved in research and development, 93 are involved in manufacturing operations, and 27 are involved in business development and general and administrative activities.

Of these colleagues, 22 are directly involved in R&D, 90 are involved in manufacturing operations, and 35 are involved in business development and general and administrative activities.

Our colleagues are not represented by a labor union.

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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 1. Business