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The following post was written and/or published as a collaboration between Benzinga’s in-house sponsored content team and a financial partner of Benzinga.
Just over a year ago, biopharmaceutical company Acasti Pharma Inc. ACST ACST refocused its efforts following an unsuccessful trial of its drug candidate, CaPre, which was under development for the treatment of hypertriglyceridemia.
Acquisition Opens New Doors?
Fast forward a year, after an extensive and in-depth strategic review process during which the company raised more than $60M in capital, it completed a major acquisition of Grace Therapeutics Inc. in August 2021. Acasti intended the acquisition to position itself as a late-stage specialty pharma company and secured novel drug delivery technologies and a pipeline of three promising drugs in the clinic to treat rare and orphan diseases.
Now, Acasti is targeting three underserved orphan diseases: subarachnoid hemorrhage (SAH), a rare and life-threatening medical emergency in which bleeding occurs over the surface of the brain in the subarachnoid space between the brain and skull; Ataxia-Telangiectasia (A-T), a progressive, neurodegenerative genetic disease that primarily impacts children and causes severe disability; and postherpetic neuralgia (PHN), a persistent and often debilitating neuropathic pain caused by nerve damage from the varicella zoster virus that can cause shingles - all of which may represent significant market opportunities.
Acasti’s new pipeline aims to improve clinical outcomes by applying proprietary formulation and drug-delivery technologies to marketed pharmaceutical compounds to achieve improvements over the current standard of care in rare diseases that are underserved by available therapies or currently have no approved therapies.
Taking Advantage of Orphan Drug Designation
The company reports it is taking advantage of the Orphan Drug Designation that it has already received from the FDA, which provides each drug, once approved, with seven years of market exclusivity in the U.S. and ten years in Europe. These drug candidates will also benefit from additional protection from over 40 granted and pending patents that extend beyond 2036. These patents cover composition of matter claims for a novel oral mucosal spray, a topical spray, and an intravenous infusion, as well as method of use claims, in most major global markets.
Orphan drugs are those developed by pharmaceutical companies for the treatment of rare diseases, which is defined by the FDA as one where fewer than 200,000 people in the U.S. are affected. Developing and commercializing orphan drugs come with major incentives such as market exclusivity, a faster regulatory review process, significant tax credits, and premium pricing. Some of the world’s largest pharmaceutical companies such as AstraZeneca plc AZN and Sanofi S.A. SNY are major producers of orphan drugs.
“We believe that the acquisition of Grace’s assets represents a transformative opportunity for Acasti. In addition to advancing our current clinical pipeline, our novel drug-delivery technologies could be used to develop new therapies that improve upon other marketed compounds with known safety profiles and could provide additional attractive paths to drug development and commercialization,” Acasti CEO Jan D’Alvise said at the time.
Significant Milestones in 2022
While all at different stages of development, the three clinical drug candidates are expected to achieve significant development milestones in 2022 such that the Company expects to have 2 drugs in Phase 3 and a third in Phase 2 by early 2023. Importantly, Acasti has two years of capital on its balance sheet, which it plans to use to fund the completion of clinical development for its lead asset (GTX-104), and significantly advance the next two assets in the pipeline.
The preceding post was written and/or published as a collaboration between Benzinga’s in-house sponsored content team and a financial partner of Benzinga. Although the piece is not and should not be construed as editorial content, the sponsored content team works to ensure that any and all information contained within is true and accurate to the best of their knowledge and research. This content is for informational purposes only and not intended to be investing advice.
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