Aquestive focuses on redesigned products for CNS conditions
Solving therapeutic problems
Aquestive Therapeutics, a pharmaceutical company that advances and commercializes medicines to solve critical therapeutic problems, especially those of the central nervous system (CNS), uses innovative drug delivery technology and scientific expertise to redesign important medicines. Recently, the company announced its first quarter results and revealed its plans for the rest of 2021.
Keith Kendall, president and chief executive officer of Aquestive, said, “We are focused on continuing to make progress this year in advancing our proprietary products. In response to feedback from the FDA, we are developing additional analyses of the existing clinical data in the NDA for Libervant and expect to refile by the end of the second quarter of 2021. We recently initiated the first-in-human Phase 1 PK study with our second-generation epinephrine candidate AQST-108 and anticipate reporting top-line data in the second half of the year.”
Libervant™, which is administered buccally, or inside of the cheek, is a soluble film formulation of diazepam, a benzodiazepine intended for rapid treatment of acute uncontrolled seizures. The drug is intended for use in selected, refractory patients with epilepsy on stable regimens of AEDs who require intermittent use of diazepam to control episodes of increased seizure activity. Aquestive is developing Libervant as an alternative to more invasive, inconvenient and difficult-to-administer, device-driven products, including a rectal gel, for patients with refractory epilepsy. Because of these issues, a large portion of the patient population does not receive adequate treatment or foregoes treatment altogether. Libervant, if approved by the FDA for U.S. market access, could enable a larger share of these patients to receive more appropriate treatment by providing consistent therapeutic dosing in a non-invasive and innovative treatment form.
Aquestive previously received a Complete Response Letter (CRL) from the FDA in September 2020. Since September, Aquestive has engaged with the FDA and developed a plan to address the deficiencies noted in the CRL. The company continues to believe that no additional clinical studies will be required to resubmit the NDA. It anticipates a six-month review process and PDUFA action date by the end of 2021.
Aquestive continues to advance its two product candidates, AQST-108 and AQST-109, for the potential treatment of severe allergic reactions, including anaphylaxis, utilizing Aquestive’s PharmFilm® technologies. In March the company initiated first-in-human Phase 1 pharmacokinetic (PK) study for the AQST-109 epinephrine sublingual film candidate. It began a two-part Phase 1 PK trial of AQST-109 in Canada and continues to conduct non-clinical modeling analysis of AQST-108 in anticipation of engaging regulators in the second half of 2021 after the readout from both of these activities.
According to Dan Barber, the company’s chief operating officer, “Aquestive is, we believe, the first and only company that has successfully demonstrated a repeatable and predictable capability for orally administering a film for the delivery of systemic epinephrine as indicated by the results from the two completed Phase 1 PK trials with AQST-108. We have taken the learnings from the AQST-108 studies regarding absorption and conversion and have applied them to the development of our second generation prodrug, AQST-109. We plan to commence a Phase 1 PK trial with AQST-109 and anticipate a topline data readout in the second half of 2021.”
“Today is an exciting day for the Aquestive team,” said Kendall. “Now that we have completed the submission of our initial patent applications, we are thrilled to provide our various stakeholders more insight into our scientific and clinical efforts to develop treatments for anaphylaxis. Patients, caregivers and healthcare providers remain highly interested in non-invasive routes of delivery for systemic epinephrine. Our surveys show that 80 percent of patients surveyed are interested in PharmFilm replacing their current medical device and 96 percent of patients surveyed believe that PharmFilm would be easier to administer during an emergency situation when compared to their current medical device.”
Despite the continued limitations on provider in-person interactions caused by the COVID-19 pandemic, the company’s proprietary product Sympazan® (clobazam), an oral film for the treatment of seizures associated with Lennox-Gastaut syndrome, continues to meet key performance metrics, according tto company officials. Shipment volume has grown 13 percent sequentially, quarter over quarter, and 40 percent year over year. Sympazan saw continued growth in the prescriber base, with over 30 percent penetration into the company’s focused group of prescribers, with approximately 80% of those prescribers writing multiple prescriptions.
Aquestive’s total revenues were $11.1 million in the first quarter 2021, compared to $8.8 million in the first quarter 2020. This year-over-year increase reflected higher license and royalty revenue and growth in Sympazan revenue. Aquestive saw net revenue growth in the first quarter 2021, compared to the prior year period, of 56 percent for Sympazan, the first of its proprietary products to be launched.
The company’s net loss for the first quarter 2021 was $14.7 million, or $0.41 loss per share. The net loss for the first quarter 2020 was $16.5 million, or $0.49 loss per share. The year-over-year change in net loss was driven by higher revenue and reductions in costs and expenses, partly offset by increased interest expense related to the sale of future revenue. This increase in interest expense was due to the accounting associated with the KYNMOBI® monetization transaction which closed on November 3, 2020, and does not represent a cash output or monetary obligation at any time during the life of the transaction. Adjusted EBITDA loss was $6.3 million in the first quarter 2021, compared to $11.2 million in the first quarter of 2020. The year-over-year change in adjusted EBITDA loss was driven by higher revenue and reductions in costs and expenses.
As of March 31, 2021, cash and cash equivalents were $27.5 million. During the first quarter 2021, Aquestive accessed capital under its "At-The-Market" (ATM) facility resulting in net proceeds of $9.9 million.
Aquestive is maintaining its full year 2021 financial outlook. The company expects: total revenues of approximately $38 million to $42 million, non-GAAP adjusted gross margins of approximately 70 to 75 percent on total revenues and non-GAAP adjusted EBITDA loss of approximately $42 to $45 million.