For the benefit of 90% of patients with cystic fibrosis, the new drug advances into phase 3 clinical

For the benefit of 90% of patients with cystic fibrosis, the new drug advances into phase 3 clinical

February 05, 2018 Source: WuXi PharmaTech

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Recently, Vertex Pharmaceuticals announced the selection of two new generations of the corrective agents VX-659 and VX-445 into Phase 3 development as part of two different cystic fibrosis (CF) triple therapy.

CF is a rare genetic disease that shortens lifespan and affects approximately 75,000 people in North America, Europe and Australia. CF is caused by a defect or deletion of CFTR protein caused by mutation of the CFTR gene. Children must inherit two defective CFTR genes - one for each parent to develop CF. There are about 2000 known mutations in the CFTR gene, some of which cause CF by producing inactive or too little CFTR protein on the cell surface, which can be determined by genetic testing or genotyping. Defects or deletions in the function of CFTR proteins result in an imbalance of salt and water influx and outflow in many organ cells. In the lungs, this causes abnormally thick mucus build-up, causing chronic lung infections and progressive lung damage, and ultimately leading to death. The median age of death was around 20 years. Therefore, they urgently need a drug to stop the progress of the disease and save lives.

VX-659 and VX-445 can increase the amount of mature protein on the cell surface by targeting defects in processing and trafficking of the F508del CFTR protein. The triple therapy with these two corrective agents is currently undergoing a phase 2 evaluation, in combination with tezacaftor and ivacaftor in the treatment of F508del/F508del patients, and in combination with tezacaftor and VX-561 in patients with F508del/Min.

â–²A number of clinical trials involving VX-659 and VX-445 (Source: Vertex Pharmaceuticals official website)

The decision to advance the VX-659 and VX-445 for Phase 3 development is based on preliminary data from Phase 2 studies. They showed that patients with a F508del mutation and F508del/Min mutation predicted a forced expiratory volume in 1 second after 4 weeks of triple therapy with VX-659 (400 mg QD) or VX-445 (200 mg QD). The average absolute improvement (ppFEV1) was 13.3 and 13.8 percentage points higher than the baseline, respectively. A regulatory discussion is currently underway to complete the design of the VX-659 and VX-445 Phase 3 studies. Vertex plans to launch a Phase 3 study in the first half of 2018 to evaluate the efficacy of VX-659 in combination with tezacaftor and ivacaftor. In addition, the company plans to launch another phase 3 study in mid-2018 to evaluate the efficacy of VX-445 in combination with tezacaftor and VX-561 as a one-day regimen. Vertex discussed Phase 2 research data and Phase 3 R&D strategies in its recent fourth quarter and full year financial performance reports.

â–² Dr. Jeffrey Chodakewitz, Executive Vice President and Chief Medical Officer of Vertex (Source: Vertex Pharmaceuticals Official Website)

"These results support the selection of VX-659 and VX-445 triple therapy and emphasize that these options can provide significant clinical benefit for up to 90% of CF patients," said Dr. Jeffrey Chodakewitz, executive vice president and chief medical officer at Vertex. I look forward to discussions with regulators and launch Phase 3 R&D in the first half of this year, with the goal of providing patients with triple therapy as soon as possible."

"The data from the triple therapy demonstrates that we have made rapid progress in the underlying causes of CF," said Dr. Jennifer Taylor-Cousar, co-chair of the Vertex Triple Therapy Steering Committee. "To date, all Phase 2 data provide further evidence. Adding the next generation of corrective agents to tezacaftor and ivacaftor may provide substantial clinical benefit for patients with F508del mutations and a minimal functional mutation, and there are currently no drugs that can cure the underlying causes of their CF. This is also at least one Patients with F508del mutations offer additional benefits and they are already eligible for CFTR modulator therapy."

We look forward to Vertex's decision to bring new treatment options to CF patients as soon as possible.

Reference materials:

[1] Vertex Selects Two Next-Generation Correctors, VX-659 and VX-445, to Advance into Phase 3 Development as Part of Two Different Triple Combination Regimens for People with Cystic Fibrosis

[2] Vertex advances drugs that would reach far broader swath of cystic fibrosis patients

[3] Vertex official website

Fixative (dye-Fixing Agent)

Active Ingredient: Quaternary ammonium salt cationic surfactant
Chemical Name: Formaldehyde-free Fixing Agent
Chemical Family: PolyDADMAC

CAS No.: 26062-79-3

1.Formaldehyde-free Fixing Agent

2.Has no free formaldehyde,no peculiar smell,no irritation to skin, accord to eco request

[Description]:

Formaldehyde-free Fixative Color Fixing Agent is belongs to polymerization polyDADMAC, a cationic polymer. It can make up of macromolecular compound with dye, then achieve to improve soaping and rub fastness of fabrics. It can be used for color fixing for dyeing and printing of reactive dyed fabrics as cotton, linen, silk, and direct dyed fabrics.

[Specification]:

Appearance: Brownish yellow viscous liquid

Ionic character: Cationic

PH Value: 5-8 (1% aqueous solution)

Dissolution: Dissolve in cold water easily.

Composition: Special polymer compound

[Dilution Method]:

With 4-6 times the water and stir to dissolve dilution.

[Direction for Usage]:(To dilute finished product as an example)

A. Padding Process: 5-20g/L, temperature 40°C-60°C, PH Value:5-8, One-dip-one-nip or Double-dip-double-nip.

B. Dipping process: 1-4% (O.W.F), Room temperature 40°C-60°C, PH Value:5-8, bath ratio:1:8-12,Time: 20-30 min.

[Properties]:

1. Concentrated Formaldehyde-free Fixative Fixing Agent has good color fixing property, can obviously improve the dyeing or printing of fabric soaping, washing fastness and wet rubbing fastness.

2. Can be used for much of the association between dye fixation, anti fading property, does not affect the dyeing or printing of fabric vividness. No effect for the original feel, processing fabric has excellent hydrophilic.

3. Formaldehyde-free Fixative widely used for dyeing or printing of the direct, active dye fixing finishing, good fixation effect, especially for the red dye and Cuilan dyes, particularly prominent, fixation is better than the general Formaldehyde-free Fixative.

4. The dye PH value for a wide range, acid, alkali resistance, resistance to hard water, high temperature resistance, an environment-friendly products, does not contain formaldehyde.

Specifications:
Appearance:	Colorless, Transparent Colloid
Viscosity:	100cps-80000cps
Solid Content:	40%min.
PH (30% solution):	3 - 7
Ionic Nature:	Cationic
Specific Gravity:	About 1.1

Package and Storage: It is harmless, no-flammable and non-explosive, it can be stored at room temperature, it can not be placed in the sun. It is packaged in plastic drums with each containing 25kg, 210kg, 1100kg , or others according to the requirements of clients. Storage temperature: 5-30°C. The product would show slightly stratification or appear white at low temperature. There is no negative impact on the performance after mixing up.

Fixative,Dye-Fixing Agent,Organic Dye-Fixing Agent,Reactive Dye-Fixing Agent

Shandong Tiancheng Chemical Co., Ltd. , https://www.tianchengchemical.com