Acasti Pharma(ACST)2022-08-10 16:00Drug Development and Pipeline - Acasti completed the acquisition of Grace on August 27, 2021, positioning itself as a late-stage specialty pharmaceutical company focused on rare and orphan diseases[90]. - The company has three advanced drug candidates: GTX-104 for Subarachnoid Hemorrhage (SAH), GTX-102 for Ataxia Telangiectasia (A-T), and GTX-101 for Postherpetic Neuralgia (PHN), all of which have received Orphan Drug Designation (ODD)[95][98]. - Acasti's drug development strategy leverages the 505(b)(2) regulatory pathway, potentially shortening the time to market for reformulated drugs[92]. - The company anticipates starting Phase 3 safety studies for GTX-104 in the first half of 2023, following successful PK bridging study results[98]. - GTX-102's PK bridging study results are expected in the second half of 2022, with Phase 3 initiation planned for the second half of 2023[98]. - GTX-101's single-dose study results are expected in the second half of 2022, with a Multiple Ascending Dose study to follow in 2023[98]. - Acasti's therapeutic pipeline includes three unique clinical stage assets supported by over 40 granted and pending patents worldwide[93]. - GTX-102, a concentrated oral-mucosal spray of betamethasone, aims to improve neurological symptoms of Ataxia Telangiectasia (A-T), a rare genetic disorder affecting approximately 4,300 patients annually in the U.S.[115][117]. - GTX-101, a non-narcotic topical bupivacaine spray, targets postherpetic neuralgia (PHN) with a total addressable market of 200 million for PHN pain[132][135]. Financial Performance - The net loss for the three months ended June 30, 2022, was 1.406 million from the net loss of 2.590 million, a significant increase of 469,000 for the same period in 2021[150]. - Total assets as of June 30, 2022, amounted to 64.478 million from 104.403 million as of June 30, 2022, up by 53.457 million in the previous year[150]. - General and administrative expenses for the three months ended June 30, 2022, totaled 757,000 from 38,377 as of June 30, 2022, a net decrease of 40,975 at June 30, 2021[168]. - Operating activities used cash of 3,401 for the same period in 2021[169]. - Total fully diluted shares increased to 50,462,426 as of June 30, 2022, from 49,018,292 as of March 31, 2022[167]. Market Potential - The total addressable market for SAH is estimated at over 150 million based on the number of treatable patients in the U.S.[117]. - Approximately 40% of patients using lidocaine patches experience insufficient pain relief, highlighting the need for alternatives like GTX-101[132]. Clinical Study Results - In a clinical trial, oral liquid betamethasone reduced ataxia symptoms by a median of 13 points (28% decrease) in the intent-to-treat population, with significant results compared to placebo (P = 0.01)[119]. - GTX-102 achieves similar blood levels at only 1/70th the volume of an oral solution of betamethasone, which is crucial for patients with swallowing difficulties[121]. - GTX-101 has shown to be well absorbed through the skin with no evidence of skin irritation in Phase 1 studies, indicating its safety and tolerability[138]. - The company believes that non-opioid products like GTX-101 will be attractive options for PHN pain relief due to faster onset and sustained relief[140]. Regulatory and Compliance - Acasti plans to submit the final PK bridging study report to the FDA in Q3 2022 and expects to initiate the Phase 3 Safety Study in the first half of 2023, which will take approximately 18 months to complete[114]. - The company received a notification from Nasdaq on July 27, 2022, for failing to maintain a minimum bid price of 2.8 million under the RKO supply agreement, which is currently disputed with Aker Biomarine Antarctic[190]. - The total contractual obligations as of June 30, 2022, amounted to 3,062 due within one year[188]. - The company has a fixed value commitment of $3.1 million for the RKO supply agreement with Aker to purchase raw krill oil product for CaPre[202].