FDA Ruling On DVAX This Week, XNCR On Watch, It's Impressive But Early For AVXL

(RTTNews) - Today's Daily Dose brings you news about Alnylam's promising preliminary phase I trial results of investigational Lumasiran for the treatment of Primary Hyperoxaluria Type 1; an update on Anavex Life's ANAVEX2-73 trial results in mild-to-moderate Alzheimer's patients; Dynavax's upcoming regulatory catalyst; Retrophin's new data from its DUET study and impressive results from Xenecor's IgG4-related disease trial.

Read on...

Alnylam Pharmaceuticals Inc.'s (ALNY) ongoing phase 1/2 study with investigational Lumasiran for the treatment of Primary Hyperoxaluria Type 1 has achieved substantial reductions in urinary oxalate levels in all patients.

Three patients in the first cohort received monthly subcutaneous doses of Lumasiran for three months while one patient received placebo. The preliminary results showed that the treatment group experienced greater than 50 percent decreases in urinary oxalate excretion relative to baseline.

Primary Hyperoxaluria Type 1, or PH1, is a devastating disease, usually manifesting in early childhood, in which excessive hepatic oxalate production leads to renal failure and often death.

ALNY closed Friday's trading at $132.36, down 0.92%.

Anavex Life Sciences Corp. (AVXL) made a presentation on Saturday at the 2017 Clinical Trials on Alzheimer's disease Meeting, which included new results from a phase 1 study of ANAVEX2-73, phase 2a study of ANAVEX2-73 and data from the first year of the Phase 2a long-term extension study of ANAVEX2-73 in mild-to-moderate Alzheimer's patients.

An analysis of clinical data from the phase I and phase 2a trials of ANAVEX2-73 revealed that there was a strong drug concentration / response relationship for exploratory secondary endpoints cognition and function and that Alzheimer's patients with milder disease stage tended to respond better to ANAVEX2-73 than patients with more advanced disease stage. But that's not all.

The data analysis has also demonstrated that ANAVEX2-73 administration does not prolong QTc interval. Prolonging the QT interval can lead to potentially fatal arrhythmias.

AVXL closed Friday's trading at $4.35, up 2.59%.

Dynavax Technologies Corp.'s (DVAX) HEPLISAV-B, a vaccine candidate for immunization against hepatitis B infection in adults aged 18 years and older, faces the FDA decision on November 9, 2017.

An FDA panel, on July 28, 2017, voted 12 to 1 that the safety data for HEPLISAV-B supports its approval. In the recent review, the panel was not asked to vote on the immunogenicity (efficacy) of the vaccine.

In November 2012, the panel had voted 13 to one that HEPLISAV-B demonstrated immunogenicity (efficacy), but voted eight to five with one abstention that there was insufficient data to support its safety.

HEPLISAV-B has been turned down twice by the FDA, i.e., in February 2013 and November 2016.

DVAX closed Friday's trading at $21.60, down 0.92%.

Jazz Pharmaceuticals plc (JAZZ) has sought approval for its acute myeloid leukemia med Vyxeos in the European Union, and a Marketing Authorization Application for the same, submitted to the European Medicines Agency's Committee for Medicinal Products for Human Use, is under accelerated assessment.

Vyxeos was approved by the FDA for the treatment of adults with two types of acute myeloid leukemia (AML), newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC), on August 3, 2017, much earlier than expected.

JAZZ closed Friday's trading at $137.89, up 0.69%.

Retrophin Inc. (RTRX) on Friday announced new positive data from the ongoing open-label extension of phase II study of Sparsentan for the treatment of focal segmental glomerulosclerosis (FSGS), a rare kidney disorder that often leads to end-stage renal disease.

Focal segmental glomerulosclerosis, or FSGS, is a rare kidney disease that scars the kidneys, causing them to lose functionality. FSGS is a common cause of proteinuria, a condition associated with abnormal amount of protein in the urine.

According to the new findings from the phase II study, dubbed DUET, patients with FSGS who remained on Sparsentan for 40 weeks during the open-label period achieved progressive reduction in proteinuria.

The initial findings from the DUET study, reported last September, demonstrated that the Sparsentan treatment group achieved statistical significance in the primary efficacy endpoint, demonstrating a greater than two-fold reduction in proteinuria compared to Irbesartan, after an eight-week, double-blind treatment period. Irbesartan is part of a class of drugs used to manage FSGS in the absence of an FDA-approved pharmacologic treatment.

The Company is planning to initiate a phase III pivotal study of Sparsentan in focal segmental glomerulosclerosis in the near future.

RTRX closed Friday's trading at $24.96, up 2.17%.

Xencor Inc. (XNCR), on Saturday, reported impressive final results from its phase II study of XmAb5871 in patients with active IgG4-related disease, or IgG4-RD.

IgG4-RD is a rare fibro-inflammatory autoimmune disorder that is estimated to impact up to 40,000 patients in the United States.

As per the final results, 12 of 15 patients completed the study, and all 12 achieved the primary endpoint of at least a two-point reduction in the IgG4-RD Responder Index (IgG4-RD RI) on Day 169. More than 50% of the patients achieved remission and 14 of the 15 patients achieved at least a five-point reduction in disease activity.

XmAb5871 has also been found to be well tolerated in patients with active IgG4-RD, noted the Company.

Based on the promising results, the Company plans to advance XmAb5871 into a phase III trial in the second half of 2018.

XNCR closed Friday's trading at $20.21, up 4.77%.