Shares of Bellerophon Therapeutics Inc. (NASDAQ:BLPH), rescued from potential delisting by a recent reverse stock split, shot 168.4% higher to $9.20 on Feb. 18 as new top-line data from a small phase II study showed its inhaled nitric oxide delivery system, Inopulse, delivered significant improvements in blood flow for people with pulmonary hypertension (PH) associated with pulmonary fibrosis (PF).
Privately held Zhittya Genesis Medicine Inc. received approval from Mexico’s Comisión Federal para la Protección contra Riesgos Sanitarios (Cofepris) to begin a phase I trial at the Zambrano Hospital in Monterrey for patients with Parkinson’s disease.
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).
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.
Emeryville, Calif.-based Zogenix Inc.’s positive top-line data from the phase III study with Fintepla (fenfluramine oral solution) in Lennox-Gastaut syndrome (LGS) failed to charm Wall Street, which by day’s end trimmed the shares (NASDAQ:ZGNX) by $20.50, or 39%, putting the final price at $32.12.