STAT+: Intellia says CRISPR-based treatment for rare disease reduced swelling attacks in pivotal trial

Intellia Therapeutics said Monday that a single dose of its gene editing treatment dramatically reduced swelling attacks in patients with a rare genetic disorder in a Phase 3 trial, setting up a potential approval.

The therapy, known as lonvo-z, would be the second approved CRISPR-based medicine, after Vertex Pharmaceutical’s sickle cell treatment Casgevy. Intellia has already initiated a rolling submission with the agency. It would be the first in vivo treatment, meaning it edits patients’ DNA directly in the body. 

In the 80-patient study, volunteers with hereditary angioedema (HAE) who received lonvo-z saw their attack rates drop 87%, relative to placebo. Just over 60% were entirely attack-free over the period, compared to 11% for the placebo patients.

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STAT+: Veradermics’ hair loss drug succeeds in late-stage trial

An oral medicine for hair loss successfully spurred hair growth in a late-stage trial, startup Veradermics announced Monday.

Veradermics assessed the pill in two ways: by how many hairs grew within a square centimeter of the scalp, on average, and by how satisfied participants were with the results. Over the course of six months, men who took the drug, known as VDPHL01, either once or twice daily had between 30 and 33 more hairs per square centimeter of scalp. Men in the placebo group grew approximately seven additional hairs.

Between 79% and 86% of men taking VDPHL01 said they saw improvement, along with between 72% and 84% of the clinical trial investigators — results that pleased Reid Waldman, a dermatologist turned Veradermics’ chief executive.

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STAT+: Oruka’s long-acting psoriasis therapy posts strong results in mid-stage study

A long-acting injectable treatment for plaque psoriasis helped 63% of patients achieve complete skin clearance in a mid-stage clinical trial, its maker, Oruka Therapeutics, reported Monday. 

Separately, Oruka said an updated analysis of blood exposure levels supported the potential for the drug, ORKA-001, to be injected just once per year. 

Oruka still needs to conduct late-stage clinical trials, but Monday’s results bolster ORKA-001’s potential to become a new treatment for the autoimmune skin disease with remission rates equal to or greater than current commercial blockbusters Skyrizi and Bimzelx, while requiring fewer injections.

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