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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STAT+: Zap in a cap: How one neurotech startup is using a hat to treat depression

Wake up. Brush your teeth. Wash your face. 

And put on your lifesaving baseball hat.

That’s right. If you have treatment-resistant depression, this could be the regular morning routine in your future. The hat would activate a blueberry-sized device implanted in your skull that sends a pulse of electricity into your brain.

This is Jacob Robinson’s vision — and it got closer to reality on Friday after the Food and Drug Administration approved a request from Robinson’s startup, Motif Neurotech, to start an initial feasibility trial to test the efficacy of their device in treating depression that hasn’t responded to other treatments. Scientists have been zapping brains to alleviate depression for decades through a method called transcranial magnetic stimulation, or TMS. Motif wants to do the same thing, but with a twist.

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NIH-funded research lags in reporting sex differences, new study finds

Fewer than half of papers published by NIH-funded researchers analyze or report their data by sex, which could make it harder to know what the results mean for men and women, a new study found.

Over a decade ago, the National Institutes of Health set out to promote sex-inclusivity in study design by introducing the expectation that research it funded consider sex as a biological variable (SABV). The guidelines are broad, asking researchers to consider SABV in their design, analysis, and reporting, without mandating that sex differences be examined in the results. 

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Genetic risk for neurodevelopmental disorders as a potential factor affecting antipsychotic responsiveness in schizophrenia: a postmortem brain study

IntroductionSchizophrenia (SCZ) is a highly heritable neuropsychiatric disorder. Its genomic architecture reportedly overlaps with that of neurodevelopmental disorders, such as attention deficit hyperactivity disorder (ADHD) and autism spectrum disorder (ASD). However, the effect of genomic risk for ADHD and ASD on SCZ symptoms remains unclear.MethodWe obtained genome-wide association study (GWAS) data from the postmortem brains of 24 patients with SCZ and 48 controls and calculated the polygenic risk scores (PRSs) for ADHD (ADHD-PRS) and ASD (ASD-PRS) using publicly available GWAS data. For 19 patients with SCZ whose antemortem clinical information was available, we conducted correlation analyses between PRSs, severity of SCZ symptoms, and the antipsychotic responsiveness score (ARS). Additionally, we divided the patients into two subgroups based on ADHD-PRS (high and low ADHD-PRS groups) and performed exploratory gene expression analyses and subsequent pathway analysis in the prefrontal cortex.ResultsThe ARS of positive symptoms (ARS-PS) demonstrated a suggestive negative correlation with ADHD-PRS and a positive correlation with ASD-PRS although these associations did not survive multiple testing correction. No correlation was observed between the ARS of general psychopathology or the ARS of negative symptoms and either ADHD-PRS or ASD-PRS. Gene expression analysis identified 1,773 DEGs, including neuropsychiatric disorder-related genes including CHRNB2. These DEGs were enriched in pathways associated with the neuronal system and mitochondrial function.DiscussionOur findings suggest that the genomic risk for neurodevelopmental disorders may affect the antipsychotic responsiveness of patients with schizophrenia and implicate translational alterations in potential marker molecules in this phenotype. Due to the limited sample size in the current study, further investigation on the large cohort is required to verify our exploratory findings.