With reasonable safety, Ofev (nintedanib) slowed lung function decline in Japanese patients with progressive fibrosing interstitial lung diseases (ILDs), according to a subgroup analysis of the Phase 3 INBUILD trial. Overall, this analysis provides further assurance to clinicians in Japan on Ofev’s benefits to patients there, with “no new…
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A large and multi-ethnic database, reported to be the first of its kind for rare lung diseases, is now compiling real-world clinical and imaging data on people with pulmonary fibrosis (PF) and other interstitial lung diseases (ILDs) from centers across the globe. Launched by the Open Source Imaging Consortium (OSIC),…
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
A single dose of the investigational oral therapy PLN-74809 for idiopathic pulmonary fibrosis (IPF) achieved therapeutic target engagement of up to 98% in the lungs of patients, according to interim results of a Phase 2a trial. At all doses tested, the anti-fibrotic, or anti-scarring, therapy engaged its intended target…
Blocking the activity of a cell death-suppressing protein called Bcl-2 promoted the death of macrophages — a type of immune cell involved in pulmonary fibrosis (PF) — and reversed established lung scarring in a mouse model, a study showed. Notably, Bcl-2’s anchoring at the surface of macrophage mitochondria was…
Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…
A trio of global companies is teaming up to develop an inhaled dry powder formulation of SF2523, an investigational small molecule that blocks the activity of two fibrosis-driving proteins that may help block tumor growth and ease lung scarring and inflammation. SF2523 is being developed as a treatment for…
In preclinical studies, PRS-220, Pieris Pharmaceuticals’ experimental inhaled therapy for idiopathic pulmonary fibrosis (IPF), showed stronger lung tissue penetration and anti-scarring effects than pamrevlumab — an into-the-vein, mechanistically similar IPF therapy currently being tested in a Phase 3 clinical trial. These promising findings, which support Pieris’…
A new service, Bionews Clinical is seeking to bring more patients into relevant clinical trials by helping all involved — scientists, pharmaceutical companies, people with diseases — regard these studies as part of continuing care. “Today what happens is that the conversation about patients participating in a clinical trial is…
The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…
