ACST Plunges On Data Delay, MREO On Watch, AQST Faces FDA In Sep.

Today’s Daily Dose brings you news about an update on Acasti Pharma’s TRILOGY 2 study, Biohaven’s disappointing results from Generalized Anxiety Disorder trial, Aquestive Therapeutics’ date with FDA, the clinical hold on LogicBio Therapeutics’ methylmalonic acidemia trial and Mereo BioPharma’s funding transactions.

Read on…

Shares of Acasti Pharma Inc. (ACST) plunged to a new low on Monday, following news that there is going to be a delay in the reporting of TRILOGY 2 topline results.

TRILOGY 2 is one of the two phase III trials of the Company’s prescription drug candidate CaPre for the treatment of severe hypertriglyceridemia. Initially, the topline results from this study were slated to be announced by mid-February 2020. But now, the Company expects to report the results sometime only in the third quarter of this year.

Last month, the Company reported disappointing topline results from the first phase III trial of CaPre, dubbed TRILOGY 1. Although there was a 30.5% and 36.7% reduction in triglyceride levels, compared with baseline, among patients receiving CaPre at 12 and 26 weeks respectively, as well as 42.2% reduction in triglyceride levels among patients receiving CaPre while on background statin therapy at 12 weeks, the TRILOGY 1 study did not reach statistical significance due to unusually large placebo effect.

ACST closed Monday’s trading at $0.57, down 24.60%. The stock touched a new all-time low of $0.48 in intraday trading.

Aquestive Therapeutics Inc.’s (AQST) New Drug Application for Libervant Buccal Film for the management of seizure clusters has been accepted for review by the FDA – with a decision date set for September 27, 2020.

Libervant is a buccally, or inside of the cheek, administered soluble film formulation of diazepam, a benzodiazepine. The Company is developing Libervant as an alternative to Diastat, the current standard of care rescue therapy for patients with refractory epilepsy, which is a rectal gel.

If approved by the FDA, Libervant will be the first oral diazepam-based therapy approved for management of seizure clusters in the population of 1.2 million refractory epilepsy patients, the Company noted.

AQST closed Monday’s trading at $4.67, up 17.93%.

Biohaven Pharmaceutical Holding Company Ltd.’s (BHVN) phase III clinical trial evaluating Troriluzole compared to placebo for the treatment of patients with Generalized Anxiety Disorder has yielded disappointing results.

There was no difference between Troriluzole monotherapy at 100mg twice daily and placebo on the primary endpoint of the mean change from baseline on the Hamilton Anxiety Rating Scale (HAM-A) after eight weeks of treatment.

While the results from this trial do not support the development of Troriliuzole as a monotherapy in Generalized Anxiety Disorder, the drug candidate continues to be tested in other disease indications and with different dosing paradigms.

Near-term Catalysts:

— A pivotal Phase 2/3 trial of Troriluzole dosed at 280mg in Alzheimer’s disease is underway, with the trial expected to be completed in the fourth quarter of this year.
— A Phase 2/3 trial of Troriluzole adjunctive therapy in subjects with Obsessive Compulsive Disorder is ongoing, with topline data is anticipated in the second quarter.
— A phase III trial of Troriluzole in adult subjects with Spinocerebellar Ataxia is ongoing, with topline data expected in 2021.
— The Company’s most advanced drug candidate is Rimegepant Zydis orally disintegrating tablet (ODT) for the treatment of migraine. The FDA decision is expected in the first quarter of 2020.
— A phase III study of Rimegepant in the preventive treatment of migraine is also underway, with topline data anticipated this quarter.
— A phase III clinical trial for Verdiperstat for the treatment of Multiple System Atrophy is ongoing, with topline results expected in 2021.

BHVN closed Monday’s trading at $47.06, down 8.18%.

Shares of LogicBio Therapeutics, Inc. (LOGC) slumped over 25% in extended trading on Monday as the FDA placed a clinical hold on the Company’s Investigational New Drug submission for LB-001 for the treatment of methylmalonic acidemia pending the resolution of certain clinical and nonclinical questions.

The Investigational New Drug submission was made in January 2020 to support the initiation of a Phase 1/2 clinical trial in patients with methylmalonic acidemia, an inherited disorder in which the body is unable to process certain proteins and fats.

The Company expects to resolve the issue within 30 days.

LOGC closed Monday’s trading at $10.45, down 5.00%. In after-hours, the stock fell another 25.84% to 7.75.

Mereo BioPharma Group plc (MREO) has entered into a $5 million convertible equity financing with Novartis Pharma (NVS), in addition to entering a securities purchase agreement with Aspire Capital Fund, LLC for up to $28 million.

The transactions are expected to provide the Company with an efficient and flexible source of capital for the continued advancement of its pipeline in 2020.

The Company’s lead drug candidate is Setrusumab, a potential treatment for osteogenesis imperfecta, also known as brittle bone disease. Setrusumab was acquired from Novartis in 2015.

A Phase 2b ASTEROID study with Setrusumab in adult patients with osteogenesis imperfecta was successfully completed last month.

The Company is preparing for its upcoming Type B End-of-Phase 2 meeting with the FDA to discuss the positive data from the ASTEROID study as well as its proposed pivotal study design in pediatric osteogenesis imperfect patients.

MREO closed Monday’s trading at $2.00, up 27.39%.

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