Weekly News Update

What have you done for your lungs lately? 

A new therapeutic approach to fibrotic conditions of the lungs has been approved for clinical trials in idiopathic pulmonary fibrosis (IPF) patients and a new therapeutic approach to cystic fibrosis is approved. These conditions were previously difficult to treat and usually have very poor prognosis.  

Indalo Initiates Dosing in IPF Patients with Antifibrotic Drug Candidate (Posted: October 21, 2019).
Indalo Therapeutics announced the first IPF patient has been dosed in a clinical trial of the company’s lead drug candidate IDL-2965. Indalo is studying IDL-2965 administered orally once daily in an adaptive multi-part Phase 1 clinical trial. Indalo recently completed the first two parts of the protocol: single- and multiple-ascending dose (MAD) studies in normal healthy volunteers (NHVs). The NHV studies, which explored a 120-fold dose range in 80 NHVs, demonstrated steady-state plasma concentrations at multiples of predicted efficacious exposures and a favorable safety profile. Dosing has now begun in the third part of the protocol: a MAD study in IPF patients designed to measure the effect of IDL-2965 on safety, pharmacokinetics, and biomarkers over 28 days. 

IDL-2965 is an oral, selective antagonist of αvβ1, αvβ3, and αvβ6 that uniquely inhibits multiple fibrogenic processes, including the local activation of TGF-β (a central regulator of pathologic fibrosis) as well as the ability of stiff extracellular matrix to promote fibroblast migration and survival. This approach provides robust antifibrotic activity across a broad range of tissue types in preclinical models without having to rely on null-like suppression of a single target. 
This new approach to this underserved medical problem demonstrates a mechanistic approach to problem solving. The therapeutic agent is targeted at a pathology present in different tissue types and is not restricted to one tissue type. Medical advances in therapy based on mechanistic studies will provide benefits to patients who were previously facing a daunting fight for survival. 

The entire article can be found at this link: https://www.americanpharmaceuticalreview.com/1315-News/518817-Indalo-Initiates-Dosing-in-IPF-Patients-with-Antifibrotic-Drug-Candidate/.  

FDA Approves New Therapy for Cystic Fibrosis

(Posted: October 22, 2019)

The U.S. Food and Drug Administration (FDA) has approved Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population. 

Cystic fibrosis, a rare, progressive, life-threatening disease, results in the formation of thick mucus that builds up in the lungs, digestive tract, and other parts of the body. It leads to severe respiratory and digestive problems. Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene. While there are approximately 2,000 known mutations of the CFTR gene, the most common mutation is the F508del mutation. 

Trikafta is a combination of three drugs that target the defective CFTR protein. It helps the protein made by the CFTR gene mutation function more effectively. The efficacy of Trikafta in patients with cystic fibrosis aged 12 years and older was demonstrated in two trials. In each trial, the primary analysis looked at increases in the percent predicted forced expiratory volume in one second, known as ppFEV1, which is an established marker of cystic fibrosis lung disease progression. Trikafta increased the ppFEV1 in both trials. The safety profile of Trikafta is based on data from the 510 cystic fibrosis patients in the two trials. The safety profile was generally similar across all subgroups of patients with very few if any serious adverse effects. 

Patients with cystic fibrosis should speak with a healthcare professional and have tests performed to understand which gene mutations they have. The presence of at least one F508del mutation should be confirmed using an FDA-cleared genotyping assay prior to treatment. 

The FDA granted this application Priority Review, in addition to Fast Track and Breakthrough Therapy Designation. Trikafta also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The approval of Trikafta was granted to Vertex Pharmaceuticals Incorporated, which will receive a Rare Pediatric Disease Priority Review Voucher for developing this therapy.

The entire article can be found at this link: https://www.americanpharmaceuticalreview.com/1315-News/518840-FDA-Approves-New-Therapy-for-Cystic-Fibrosis/

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Dr. Joe Nieusma and the Superior Toxicology & Wellness Team

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