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FDA Panel Nod For GSK's Shingles Vaccine, FOLD Beyond Essence, FENC Is Red Hot


Today's Daily Dose brings you news about encouraging interim analysis of Alexion's REGAIN study of Soliris; Fennec's uplisting and preliminary results of SIOPEL 6; Amicus' failed epidermolysis bullosa trial; Vanda's disappointing atopic dermatitis trial results and FDA panel's recommendation for approval of GlaxoSmithKline's investigational shingles vaccine Shingrix.

Read on...

Alexion Pharmaceuticals Inc. (ALXN) has announced that interim analysis of its ongoing phase III open-label extension study of the pivotal, placebo-controlled REGAIN study of Soliris for the treatment of patients with refractory generalized myasthenia gravis show sustained treatment benefits.

The treatment benefits for patients treated with Soliris in REGAIN study through 26 weeks were maintained in the extension study across all four assessment scales for an additional 52 weeks (78 weeks in total), the company noted.

For patients who received placebo in REGAIN and then were treated with Soliris in the extension study, significant treatment benefits occurred within 1 to 4 weeks and were sustained through 52 weeks across all four assessment scales.

The assessment scales include MG-ADL - MG-Activities of Daily Living, a patient-reported assessment of functional ability to carry out daily activities; QMG - Quantitative MG, a clinical assessment of muscle strength by physicians; MGC - MG Composite, a patient- and physician-reported assessment of functional ability and signs and symptoms of MG and MG-QoL 15 - MG Quality of Life 15, a patient-reported assessment of MG-specific quality of life.

Soliris is already indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis, and for the treatment of patients with atypical hemolytic uremic syndrome.

The drug generated total sales of $1.59 billion in the first half of 2017, and its sales are expected to range between $3.07 billion to $3.12 billion for the full year.

ALXN closed Wednesday's trading at $146.49, up 1.68%.

Amicus Therapeutics Inc.'s (FOLD) phase III clinical study of novel topical wound-healing agent SD-101 in participants with epidermolysis bullosa, dubbed ESSENCE, did not meet the primary endpoints or secondary endpoints.

Epidermolysis bullosa is a rare, devastating genetic disorder that leads to severe skin blistering and open wounds often beginning at birth. There are no approved therapies to treat epidermolysis bullosa, which affects tens of thousands of children and adults throughout their lifetime.

An important regulatory catalyst to watch out for is the Company's filing of New Drug Application for Galafold for Fabry disease by the end of this year. If approved, the drug may hit the U.S. market shelves in the second half of 2018.

Galafold was approved by the European Commission last May, and has already been launched in Europe.

FOLD closed Wednesday's trading at $13.51, up 1.73%.

Fennec Pharmaceuticals Inc. (FENC) (FRX.TO), which was uplisted to the NASDAQ on Wednesday (Sept.13, 2017), has announced preliminary results of SIOPEL 6.

SIOPEL 6 is a phase III study of Pedmark (Sodium Thiosulphate, or STS) in reducing ototoxicity in patients receiving Cisplatin chemotherapy for standard risk hepatoblastoma.

According to the preliminary results of SIOPEL 6, the addition of sodium thiosulfate (STS) significantly reduces the incidence of cisplatin-induced hearing loss without any evidence of tumor protection. Among the first 94 evaluable patients, hearing loss occurred in 65.9% under Cisplatin Arm and in 38.0% under Cisplatin + STS Arm.

The final results from SIOPEL 6 are expected in the fourth quarter of 2017.

FENC closed Wednesday's trading at $12.00, up 35.59%. The stock is up 461% year-to-date.

An FDA panel has unanimously recommended approval of GlaxoSmithKline plc's (GSK) investigational Shingrix vaccine for the prevention of herpes zoster in people aged 50 years or over.

Shingrix contains Agenus Inc.'s (AGEN) proprietary immune adjuvant, QS-21 Stimulon.

The only shingles vaccine currently approved for use in the United States is Merck & Co. Inc.'s (MRK) Zostavax.

The effectiveness of Zostavax at preventing shingles is 70% in adults aged 50-59 years and just around 18% in adults aged 80 years or older. In contrast, Shingrix has demonstrated over 90% efficacy against shingles, independent of age even after four years of administration.

The FDA's final decision on approval of Shingrix is expected in October of this year.
If approved, sales of Shingrix would reach more than $1 billion by 2022, according market intelligence firm Evaluate.

GSK closed Wednesday's trading at $40.10, down 0.67%.

ImmunoCellular Therapeutics Ltd.'s (IMUC) plan to regain compliance with the NYSE MKT LLC's continued listing standards has been accepted by the exchange.

On June 29, 2017, the Company was notified by NYSE MKT that it was not in compliance with certain continued listing standards relating to stockholders' equity. The Company has time until December 23, 2018 to regain compliance, failing which the NYSE MKT staff may initiate delisting proceedings as appropriate.

IMUC closed Wednesday's trading at $0.31, up 9.07%.

Vanda Pharmaceuticals Inc.'s (VNDA) phase II clinical study of Tradipitant as a monotherapy in the treatment of chronic pruritus in patients with atopic dermatitis has failed to meet the pre-specified primary endpoint of Average Itch Visual Analog Scale.

Although Tradipitant showed improvement over placebo, this improvement was not significant due to high placebo effect and the lack of sensitivity of the Average Itch Visual Analog Scale, noted the Company.

However, Tradipitant was shown to improve the intensity of the worst itch patients experienced, as well as atopic dermatitis disease severity.

Vanda believes that if these results are further confirmed in future studies, Tradipitant has the potential to become a first line pharmacological option in the treatment of patients with atopic dermatitis in need of systemic treatment.

The Company expects to meet with the FDA in the near future to chart a clinical development path towards registration of Tradipitant in the treatment of patients with atopic dermatitis.

VNDA closed Wednesday's trading at $17.00, down 1.45%.

by RTTNews Staff Writer

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