Revisiting Aquestive: CNS drugmaker shows progress
Aquestive Therapeutics, Inc., a pharmaceutical company focused on developing and commercializing differentiated products that address patients’ unmet needs and solve therapeutic problems for central nervous system (CNS) disorders, recently reported financial results for the third quarter ended September 30, 2021 and provided an update on recent developments in its business.
Keith Kendall, chief executive officer of Aquestive reported that the company continues to interact with the FDA regarding the NDA submission for Libervant™, a buccally, or inside of the cheek, administered soluble film formulation of diazepam, a benzodiazepine intended for rapid treatment of acute uncontrolled seizures in selected, refractory patients with epilepsy on stable regimens of AEDs who require intermittent use of diazepam to control bouts of increased seizure activity. Aquestive has responded to several information requests, having a recent inspection of its post-marketing adverse event capabilities, regarding changes in language relating to its packaging, approval of the product trade name and an update to the patent information included in the resubmission. The company is continuing to prepare for commercialization with payer and sales force planning underway.
The company believes that Libervant, if approved by the U.S. Food and Drug Administration (FDA) for U.S. market access, will enable a larger share of patients to receive more appropriate treatment by providing consistent therapeutic dosing in a non-invasive and innovative treatment form for epileptic seizures. Aquestive is developing Libervant as an alternative to more invasive, inconvenient and difficult to administer device driven products, including a rectal gel and nasal spray, for patients with refractory epilepsy. Most patients do not access either one of these existing products and remains underserved with regard to having an important medication where they need it, when they need it and in a form they prefer.
Aquestive continues to actively engage with the FDA regarding its accepted New Drug Application (NDA) for Libervant ahead of the Prescription Drug User Fee Act (PDUFA) target goal date of December 23, 2021. Aquestive has again spoken with and provided additional information to the FDA Office of Orphan Products Development (OOPD) that the company believes provides a clear position of clinical superiority as a major contribution to patient care as compared to the device driven rectal gel and nasal spray alternatives.
According to Kendall, “We recently demonstrated clinical results comparable to the autoinjectors in the recently completed Phase 1 trial for AQST-109 (epinephrine prodrug sublingual film), potentially the first orally administered epinephrine product for the emergency treatment of allergic reactions, including anaphylaxis. Before year end, we anticipate receiving written feedback from the FDA following our pre-IND meeting request and plan to commence an adaptive design crossover study that will determine the final formulation and dose strength leading to our pivotal PK study in 2022.”
AQST-109 utilizes Aquestive’s PharmFilm® technologies. The company reported positive topline data from its first-in-human Phase 1 pharmacokinetic (PK) trial for AQST-109. Findings from this study support AQST-109's potential as the first orally administered and transformative epinephrine-based product for the treatment of allergic reactions including anaphylaxis with safety, tolerability, PK and pharmacodynamics (PD) measures comparable to those of the standard of care autoinjectors, such as EpiPen® and Auvi-Q®. These products require patients or caregivers to inject epinephrine into their thighs during an emergency allergic reaction. However, AQST-109 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, again providing an appropriate medication where it is needed, when it is needed and in a form preferred by patients.
Aquestive has submitted its request for a pre-Investigational New Drug (IND) meeting with the FDA and anticipates receiving a written response from the FDA before year end 2021. The company is on track to conduct a crossover study using an adaptive design for AQST-109 beginning in the fourth quarter 2021. This study will determine the final formulation and dose for AQST-109 and allow the company to move forward to the manufacture of registration batches and a pivotal PK study in 2022.
The company’s proprietary product Sympazan® (clobazam), an oral film for the treatment of seizures associated with Lennox-Gastaut syndrome, continued to grow in shipment volumes to the prescriber base for the third quarter 2021. Sympazan has grown quarterly over the past eleven sequential quarters since its launch.
Aquestive’s total revenues were $13.3 million in the third quarter 2021, compared to $8.3 million in the third quarter 2020. For the third quarter 2021 compared to the prior year period, the company saw an 18 percent increase in Sympazan net revenue and a 77 percent increase in manufacture and supply revenue.
The company’s net loss for the third quarter 2021 was $14.6 million, or $0.37 loss per share. The net loss for the third quarter 2020 was $16.6 million, or $0.49 loss per share. The year-over-year change in net loss was driven by higher revenue, lower costs and expenses, offset by an increase in non-cash interest expense related to the KYNMOBI® monetization transaction, which does not represent a cash output or monetary obligation at any time during the life of the transaction.
LMR Partners LLP acquired a new stake in shares of Aquestive Therapeutics, Inc. in the second quarter. The firm acquired 12,090 shares of the company’s stock, valued at approximately $48,000.
A number of other institutional investors have also recently made changes to their positions in the business. PDT Partners LLC purchased a new position in Aquestive Therapeutics during the second quarter valued at $325,000. Alberta Investment Management Corp purchased a new position in Aquestive Therapeutics during the second quarter valued at $596,000. Morgan Stanley lifted its position in Aquestive Therapeutics by 54.7 percent during the second quarter. Morgan Stanley now owns 553,727 shares of the company’s stock valued at $2,198,000 after buying an additional 195,685 shares during the period. Tibra Equities Europe Ltd purchased a new position in Aquestive Therapeutics during the second quarter valued at $310,000. Finally, Goldman Sachs Group Inc. lifted its position in Aquestive Therapeutics by 45.3 percent% during the second quarter. Goldman Sachs Group Inc. now owns 68,030 shares of the company’s stock valued at $270,000 after buying an additional 21,204 shares during the period. 40.16 percent of the stock is owned by institutional investors and hedge funds.