ESPR To Submit NDAs In Q1, GRFS' Alzheimer's Study Brings Hope, RTRX On Watch

pharma 020917 28oct18

Today's Daily Dose brings you news about Anavex's encouraging results from its Alzheimer's study; Esperion's promising phase III clinical development program evaluating the LDL-C-lowering efficacy of bempedoic acid; Grifol's AMBAR trial; Retrophin's long-term data from DUET study, and Ultagenyx's failed glucose transporter type-1 deficiency syndrome (Glut1 DS) trial.

Read on...

Anavex Life Sciences Corp. (AVXL), on Friday, presented new promising phase 2a clinical data of ANAVEX2-73 in mild-to-moderate Alzheimer's patients.

At 148 weeks into the five-year extended phase IIa study, there was a significant association between higher ANAVEX2-73 concentration and maintained activities of daily living and reduced cognitive decline in Alzheimer's disease patients, according to the Company. ANAVEX2-73 demonstrated continued favorable safety and tolerability through 148 weeks.

A phase 2b/3 study of ANAVEX2-73 was recently initiated by the Company.

AVXL closed Friday's trading at $2.52, up 4.13%.

Esperion (ESPR) has completed its phase III clinical development program evaluating the LDL-C-lowering efficacy of bempedoic acid, and has reported positive cumulative results.

The program consisted of four, pivotal, phase III studies to evaluate the LDL-C lowering efficacy and safety and tolerability of bempedoic acid 180 mg compared to placebo in high cardiovascular risk patients including atherosclerotic cardiovascular disease (ASCVD) and/or heterozygous familial hypercholesterolemia (HeFH) patients.

According to the trial results, bempedoic acid achieved additional 18% to 31% LDL-C lowering in patients on maximally tolerated statins and provided additional 19% to 33% hsCRP reduction. High sensitivity C Reactive Protein, or hsCRP, is a biomarker of the underlying inflammation associated with cardiovascular disease risk.

In the phase III program, the 5-component MACE events in the Bempedoic Acid arm were 4.0% compared to 4.6% for placebo. In the subset of patients with diabetes, the reduction in HbA1c was 0.19% to 0.31% compared to placebo.

Esperion plans to submit New Drug Applications to the FDA for bempedoic acid and the bempedoic acid/ezetimibe combination pill for LDL-C-lowering indications during the first quarter of 2019, and submit Marketing Authorization Applications to the European Medicines Agency during the second quarter of 2019.

ESPR closed Friday's trading at $41.88, up 13.22%.

Grifols S.A.'s (GRFS) top line results from its phase IIb/III trial, dubbed AMBAR, has demonstrated, with statistical significance, the ability to slow down the progression of the disease in moderate Alzheimer's disease patients.

In the AMBAR trial, the combination of Plasmapheresis (a well-known and safe procedure used in plasma exchange) with Albutein 20% demonstrated a 61% reduction in the progression of moderate Alzheimer's disease.

"We are very pleased with the results and we celebrate them as a breath of fresh air that brings hope to the Alzheimer's patients and their families. This is the most significant development in the treatment of patients with moderate Alzheimer's disease in over 15 years," said Mercé Boada, Director of Fundació ACE.

GRFS closed Friday's trading at $19.44, up 5.37%.

Related Reading:

Will These Alzheimer's Drugs In Phase 3 Clinical Trials Cross The Finish Line?

Retrophin Inc.'s (RTRX) long-term data from an open-label extension of phase II study of Sparsentan for the treatment of Focal Segmental Glomerulosclerosis, dubbed DUET, has yielded encouraging results.

The long-term data involves results from an 84-week analysis of the DUET study. The top-line results from the 8 week analysis of the DUET study were reported in September 2016.

According to the new findings, in patients who received Sparsentan as part of the original eight-week, double-blind treatment period, median urine protein-to-creatinine ratio (UP/C) was reduced from 2.8 g/g at baseline (week 0) to 0.9 g/g at week 84. Patients who crossed over to Sparsentan from the original Irbesartan control group experienced additional and sustained reduction in proteinuria during the treatment period, with median UP/C decreasing from 2.3 g/g at crossover (week eight) to 1.1 g/g at week 84.

In patients who received Sparsentan as part of the original eight-week, double-blind treatment period, the proportion of patients who achieved *FPRE increased from 28 percent at week eight to 60 percent at week 84.

The proportion of patients who crossed over to Sparsentan from the original Irbesartan control group and achieved *FPRE increased from nine percent at week eight to 50 percent at week 84.

*FSGS partial remission of proteinuria endpoint, or FPRE, is defined as urine protein-to-creatinine ratio of less than or equal to 1.5 g/g and more than 40 percent reduction of proteinuria from baseline.

RTRX closed Friday's trading at $24.98, up 1.26%.

Shares of Ultragenyx Pharmaceutical Inc. (RARE) slumped 18% on Friday, following its failed phase III study of UX007 in patients with glucose transporter type-1 deficiency syndrome experiencing disabling paroxysmal movement disorders.

The study did not achieve its primary endpoint of demonstrating a statistically significant reduction in the frequency of paroxysmal movement events with UX007 treatment compared to placebo, and did not demonstrate a meaningful difference between treatment groups.

The Company has planned to discontinue the Glut1 DS development program.

RARE closed Friday's trading at $48.62, down 18.09%.

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