UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549

FORM 8-K

CURRENT REPORT

PURSUANT TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934

Date of Report (Date of earliest event reported): January 12, 2020

Aquestive Therapeutics, Inc.
(Exact name of Registrant as specified in its charter)

Delaware
001-38599
82-3827296
(State or Other Jurisdiction of Incorporation or Organization)
(Commission File Number)
(I.R.S. Employer Identification No.)

30 Technology Drive
Warren, NJ 07059
(908) 941-1900
(Address, Including Zip Code, and Telephone Number, Including Area Code, of Registrant’s Principal Executive Offices)

Not Applicable
(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class
Trading Symbol(s)
Name of each exchange on which registered
Common Stock, par value $0.001 per share
AQST
Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☒

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 8.01. Other Events

On January 12, 2020, Aquestive Therapeutics, Inc. (the “Company”) issued a press release reporting that the U.S. Food and Drug Administration (FDA) issued a letter (“Response”) dated January 10, 2020 denying the Company’s Citizen’s Petition received by the FDA on November 1, 2019, including the supplement to the Citizen’s Petition received by the FDA on December 4, 2019 (Docket No. FDA-2019-P-5121) (“Petition”).  In the Response, the FDA indicated that it had approved Neurelis’s NDA for Valtoco® on January 10, 2020.  Valtoco has received orphan drug exclusivity from the FDA’s Center for Drug Evaluation and Research commencing as of January 10, 2020.  A copy of the press release is attached hereto as Exhibit 99.1 and incorporated herein by reference.

The Company is also updating its risk factors to include the following:

We will be required to demonstrate to the FDA that our drug candidate Libervant® provides a “major contribution to patient care” relative to the approved drugs with the same active moiety for the same indication, Diastat® and Valtoco, and there can be no assurance that we will be successful.

We obtained orphan drug designation in the United States for our drug candidate Libervant (diazepam) Buccal Film for the treatment of selected, refractory patients with epilepsy who are on stable regimens of anti-epileptic drugs, or AEPs, and who require intermittent use of diazepam to control bouts of increased seizure activity, or acute repetitive seizure activity.  We completed the rolling submission for our NDA filing with the FDA for Libervant on November 27, 2019.  On January 10, 2020 Neurelis, Inc. obtained FDA approval of its drug candidate Valtoco (diazepam nasal spray) for its formulation of diazepam for the same indication as for our drug candidate Libervant. The FDA’s Center for Drug Evaluation and Research granted Valtoco orphan-drug exclusivity on January 10, 2020.  A company that obtains FDA approval for a designated orphan drug receives orphan drug marketing exclusivity for that drug for the designated indication for a period of seven years from the grant date in the United States.  This orphan drug exclusivity approval limits a subsequently approved product from being marketed during the exclusivity period for the same active moiety for the same orphan drug indication (Diastat and Valtoco), except in a case where the drug candidate sponsor is able to demonstrate, and the FDA concludes, that the later approved drug is clinically superior to the approved products, e.g., safer, more effective, or providing a major contribution to patient care. In assessing whether a sponsor has demonstrated that a drug candidate provides a “major contribution to patient care” relative to the approved drugs, which is evaluated on a case by case basis, the FDA may consider a number of factors and there is no one objective standard.

We will closely review the FDA approval of Valtoco and its FDA-approved label when it is available, and we plan to seek to demonstrate to the FDA that our product candidate Libervant is clinically superior to the two currently FDA-approved products for the same indication as Libervant, Valtoco and Diastat, as a major contribution to patient care within the meaning of the FDA regulations.  There can be no assurance that we will be successful in these efforts.  Any failure to obtain FDA approval of or demonstrate clinical superiority for Libervant would have a material adverse effect on our business, financial condition and results of operations.

Item 9.01. Financial Statements and Exhibits

(d)  Exhibits.

Exhibit Number
 
Description
     
 
Press Release, dated January 12, 2020.

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the Registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

Dated: January 12, 2020
Aquestive Therapeutics, Inc.
     
 
By:
/s/ John T. Maxwell
   
Name: John T. Maxwell
   
Title: Chief Financial Officer


Exhibit 99.1

Aquestive Therapeutics Receives FDA Response to Citizen’s Petition

Warren, NJ, January 12, 2020 – Aquestive Therapeutics, Inc. (NASDAQ: AQST), a specialty pharmaceutical company focused on developing and commercializing differentiated products that meet patients’ unmet needs and solve therapeutic problems, today reported that the U.S. Food and Drug Administration (FDA) issued a response letter (Response ) dated January 10, 2020 denying Aquestive’s Citizen’s Petition received by the FDA on November 1, 2019, including the supplement to the Citizen’s Petition received by the FDA on December 4, 2019 (Docket No. FDA-2019-P-5121) (Petition). The Petition requested, among other things, that the FDA stay approval of a New Drug Application for Valtoco® (diazepam nasal spray) submitted by Neurelis, Inc. until additional clinical studies were conducted. In the Response, the FDA indicated that it had approved Neurelis’s NDA for Valtoco on January 10, 2020. Valtoco has received orphan drug exclusivity from the FDA’s Center for Drug Evaluation and Research commencing as of January 10, 2020 for the labeled indication of 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 six years of age and older.

“This patient population has been underserved for some time with little choice beyond the rectally administered gel and choice is important. We believe that our candidate drug Libervant™ (diazepam) Buccal Film will, if approved by the FDA, further expand patient choice as the first orally administered dosage form for this patient population,” said Keith J. Kendall, Chief Executive Officer of Aquestive.

“We appreciate that the FDA has confirmed in the Response its guidance that 505(b)(2) drugs can be approved without proving bioequivalence if they demonstrate “relative bioavailability” to the reference drug. The FDA stated in the Response, when granting exclusivity based upon “major contribution to patient care” over and above already approved products for the indication, it may 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 making this determination for Valtoco, the FDA indicated in the Response that the intranasal route of administration provides a major contribution to patient care over the rectal route of administration by providing a significantly improved ease of use.”

“We look forward to working with the FDA in the coming months to demonstrate why we believe that our product candidate Libervant, as an orally delivered product for this indication, has one or more of the attributes required by the FDA to be considered a major contribution to patient care relative to the currently approved products,” concluded Mr. Kendall.

Although we cannot be assured of FDA approval of Libervant, Aquestive remains committed to helping people affected by seizure clusters by looking to bring important innovative products to the market that will improve the lives of patients.

We provided preliminary 2020 revenue guidance in our press release dated January 10, 2020. As a reminder, we did not include any Libervant revenues in that guidance.

About Aquestive Therapeutics
Aquestive Therapeutics is a specialty pharmaceutical company that applies innovative technology to solve therapeutic problems and improve medicines for patients. Aquestive is advancing a late-stage proprietary product pipeline to treat CNS conditions and provide alternatives to invasively administered standard of care therapies. The Company also collaborates with other pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven capabilities for drug development and commercialization.

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Forward-Looking Statement
This press release includes 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 may include, but are not limited to, statements regarding therapeutic benefits and plans and objectives for regulatory approvals of Libervant and our other product candidates; ability to obtain FDA approval and advance Libervant and our other product candidates to the market; statements about our growth and future financial and operating results and financial position, regulatory approval and pathways, clinical trial timing and plans, our and our competitors’ orphan drug approval and resulting drug exclusivity for our products or products of our competitors, short-term and long-term liquidity and cash requirements, cash funding and cash burn, 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 the Company's development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and plans; risk of delays in FDA approval of Libervant and our other drug candidates or failure to receive approval; risk that a competitor obtains FDA orphan drug exclusivity for a product with the same active moiety as the orphan drug product for which we are seeking FDA approval and that such earlier approved competitor orphan drug blocks our product in the U.S. for seven years for the same indication; risk of our ability to demonstrate to the FDA “clinical superiority” within the meaning of FDA regulations of our drug candidate Libervant® (diazepam) Buccal Film relative to the FDA-approved Valtoco® (diazepam nasal spray) and Diastat® (diazepam rectal gel) including by establishing a major contribution to patient care within the meaning of FDA regulations relative to the approved product and there can be no assurance that we will be successful; risk inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks and regulatory limitations); risk of development of our sales and marketing capabilities; risk of legal costs associated with and the outcome of our patent litigation challenging third party at risk generic sale of our proprietary products; risk of sufficient capital and cash resources, including access to available debt and equity financing and revenues from operations, to satisfy all of our short-term and longer term cash requirements and other cash needs, at the times and in the amounts needed; risk of failure to satisfy all financial and other debt covenants and of any default; risk related to government claims against Indivior for which we license, manufacture and sell Suboxone and which accounts for the substantial part of our current operating revenues; risks associated with Indivior’s announcement of its intention to cease production of its authorized generic buprenorphine naloxone film product, including the impact from loss of orders for the authorized generic product and risk of eroding market share for Suboxone and risk of sunsetting product; risks related to the outsourcing of certain sales, marketing and other operational and staff functions to third parties; risk of the rate and degree of market acceptance of our products and product candidates; the success of any competing products, including generics; risk of the size and growth of our product markets; risk of 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 the Company's products; risk of unexpected patent developments; the impact of existing and future legislation and regulatory provisions on product exclusivity; legislation or regulatory action affecting pharmaceutical product pricing, reimbursement or access; claims and risks that may arise regarding the safety or efficacy of the Company's products and product candidates; risk of loss of significant customers; risks related to legal proceedings, including patent infringement, investigative and antitrust litigation matters; changes in governmental laws and regulations; risk of product recalls and withdrawals; uncertainties related to general economic, political, business, industry, regulatory and market conditions and other unusual items; and other risks and uncertainties affecting the Company including those described in the “Risk Factors” section and in other sections included in the Company's Annual Report on Form 10‑K filed with the SEC on March 14, 2019, in our quarterly reports on Form 10-Q, and in the Form 8-K filed on January 13, 2020. Given these 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 us or any person acting on our 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.

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Additional Information Regarding Orphan Drug Exclusivity

The Response includes the following in discussing orphan drug exclusivity:

Section 527 of the [Federal Food, Drug, and Cosmetic Act] defines “clinically superior” to mean “the drug provides a significant therapeutic advantage over and above an already approved or licensed drug in terms of greater efficacy, greater safety, or by providing a major contribution to patient care.” The orphan-drug regulations elaborate on the definition of “clinically superior” as follows:

Clinically superior means that a drug is shown to provide a significant therapeutic advantage over and above that provided by an approved drug (that is otherwise the same drug) in one or more of the following ways:

Greater effectiveness than an approved drug (as assessed by effect on a clinically meaningful endpoint in adequate and well controlled clinical trials). Generally, this would represent the same kind of evidence needed to support a comparative effectiveness claim for two different drugs; in most cases, direct comparative clinical trials would be necessary; or

Greater safety in a substantial portion of the target populations, for example, by the elimination of an ingredient or contaminant that is associated with relatively frequent adverse effects. In some cases, direct comparative clinical trials will be necessary; or

In unusual cases, where neither greater safety nor greater effectiveness has been shown, a demonstration that the drug otherwise makes a major contribution to patient care.

Because of the diverse ways in which drugs may qualify as clinically superior under these criteria, FDA evaluates clinical superiority on a case by case basis. Specifically, with respect to the major contribution to patient care prong of the clinical superiority definition, the FDA has further stated:

There is no way to quantify such superiority in a general way. The amount and kind of superiority needed would vary depending on many factors, including the nature and severity of the disease or condition, the quality of the evidence presented, and diverse other factors;

and

The following factors, when applicable to severe or life-threatening diseases, may in appropriate cases be taken into consideration when determining whether a drug makes a major contribution to patient care: convenient 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.

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The Aquestive logo is a registered trademarks of Aquestive Therapeutics, Inc. All other registered trademarks referenced herein are the property of their respective owners.

Media Inquiries:
Mark Corbae
e: mark.corbae@icrinc.com
p: 858.213.7956

Investor Inquiries:
Stephanie Carrington
stephanie.carrington@icrinc.com


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