Spain's Sanifit Laboratoris SL, a company developing treatments for calcification disorders, has dosed the first patient in a phase III trial of its lead asset, SNF-472, for the treatment of the rare and sometimes deadly disease calciphylaxis, a calcium accumulation disorder.
New York-based Hoth Therapeutics Inc. said preliminary preclinical data on a topical nanoparticle formulation of the endocannabinoid anandamide it licensed from Zylö Therapeutics Inc. represents "a positive step toward underlining the transformative potential" of the early stage therapy for the skin disease cutaneous lupus erythematous.
Pediatric gene editing specialist Logicbio Therapeutics Inc. has revealed an FDA clinical hold on a planned phase I/II trial of its lead candidate, LB-001, an investigational therapy for rare inherited metabolic disorder methylmalonic acidemia (MMA).
LONDON – The genomes of 38 different tumor types and the 47 million mutations that fostered their growth are revealed in unprecedented detail in 23 studies published in Nature and other journals on Feb. 6, 2020.
A high-profile test of two experimental medicines in people with autosomal dominant Alzheimer’s disease (ADAD), a rare inherited form of the disease, found that neither drug significantly slowed rates of cognitive decline vs. placebo, the primary endpoint.
Top-line results from Biohaven Pharmaceutical Holding Co.’s phase III trial of troriluzole against placebo in treating generalized anxiety disorder (GAD) failed to hit its primary endpoint, prompting the company CEO to say the poor results support halting development plans for the glutamate modulator as a monotherapy in GAD.
With the Simpliciti-T1 phase II trial testing new-mechanism TTP-399 as adjunct therapy in type 1 diabetes (T1D), High Point, N.C.-based Vtv Therapeutics Inc. nailed the HbA1c endpoint with none of the safety concerns foiling developers of type 2 diabetes (T2D) drugs who sought to broaden their labels.
LONDON – Oculis SA reported positive results for its topical nanoparticle formulation of dexamethasone in the treatment of diabetic macular edema (DME), showing the eye drops reduced central macular thickness (CMT) in the phase II proof-of-concept trial.
The advent of transcatheter aortic valve replacement (TAVR) changed the framework for dealing with aortic valve stenosis, but some clinicians might argue there was a corresponding and inappropriate rush away from surgical aortic valve replacement (SAVR).
Houston-based Nanospectra Biosciences Inc. has kicked off a pivotal U.S. study of its Aurolase therapy in the targeted destruction of prostate tumors using nanomedicine technology. The IDE study, with an estimated completion date of December 2022, will support a de novo 510(k) submission to the U.S. FDA. The first two patients were treated at the University of Michigan, which also participated in the first-in-human pilot study of Aurolase.