Uniqure NV rolled out stock-pleasing safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase I/II trial with AMT-130 for the treatment of Huntington’s disease (HD), but investors must wait for details on MRI scans. “We have communicated that we will discuss the MRI findings in the middle of next year,” CEO Matt Kapusta told investors during a conference call. “That’s all I can say.”
Shares of Athira Pharma Inc. (NASDAQ:ATHA) plunged almost 66.3%, or $5.60, to close at $2.85 on June 22 after the company disclosed top-line results from its exploratory phase II study with fosgonimeton (ATH-1017, fosgo) in mild-to-moderate Alzheimer’s disease (AD).
After an up-and-down day – mostly up, toward the end – during which the phrase “totality of the data” got air time aplenty, shares of PTC Therapeutics Inc. (NASDAQ:PTCT) closed at $34.07, a rise of $5.66, or almost 20%, on word of top-line data from Study 041 with Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy (DMD).
The U.S. FDA’s Psychopharmacologic Drugs Advisory Committee took up the complicated matter of Acadia Pharmaceuticals Inc.’s sNDA for Nuplazid (pimavanserin) to treat hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP).
The FDA’s Psychopharmacologic Drugs Advisory Committee posted briefing documents related to the June 17 meeting, set to consider Acadia Pharmaceuticals Inc.’s sNDA for Nuplazid (pimavanserin) to treat hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP). Although shares of the San Diego-based firm (NASDAQ:ACAD) stayed in the black, closing at $18.77, up $2.52, or 15.5%, the briefing docs did not bring uniformly good news, echoing some of the concerns spelled out in an earlier complete response letter.
With Alnylam Pharmaceuticals Inc.’s FDA clearance for Amvuttra (vutrisiran) in the rearview mirror, investors are looking ahead to potentially label-widening phase III data related to another, already approved drug: Onpattro (patisiran).
Day One Biopharmaceuticals Inc.’s phase II data with tovorafenib from the Firefly-1 study in pediatric brain cancer dazzled Wall Street, and shares (NASDAQ:DAWN) closed $14.96, up $8.34, or 126%.
After surprising Wall Street by unanimously voting in favor of the gene therapy elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy from Bluebird Bio Inc., the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee met again June 10, this time to examine the risk-benefit profile of the company’s betibeglogene autotemcel (beti-cel) for people with transfusion-dependent beta-thalassemia.
A paper published March 22 in the journal Chest highlighted research that found patients with interstitial lung disease (ILD) who had poor quality of life specifically related to cough were more likely to be hospitalized for respiratory issues, need a lung transplant and die. The findings in 1,447 patients matter because they back previous investigations and provide a way of predicting the advance of ILD, of which idiopathic pulmonary fibrosis (IPF) is a prominent type. A handful of companies are working on drugs to tackle cough in IPF, often an early symptom of the disease.
