Provention Bio Inc. gained the go-ahead from the U.S. FDA of the BLA for Tzield (teplizumab-mzwv), an intravenously given, anti-CD3-directed antibody, as the first and only immunomodulatory treatment to delay the onset of stage 3 type 1 diabetes (T1D) in adult and pediatric patients ages 8 and older with stage 2 T1D. “This is not another monoclonal antibody that’s treating symptoms better than the previous one,” said CEO Ashleigh Palmer. “This is a game-changing, breakthrough innovation” for patients who previously had “no option other than to wait” for their disease to worsen. Provention priced the compound at $13,850 per vial, which amounts to $193,900 for a 14-day course of treatment.

Northwest glioblastoma therapy extends OS, 'long tail' survival, in phase III trial

An investigational dendritic cell vaccine from Northwest Biotherapeutics Inc., administered with standard of care, extended both median overall survival (OS) and "the 'long tail' of the survival curve" vs. matched external controls in newly diagnosed and recurrent glioblastoma multiforme brain cancer patients, according to results published Nov. 17 in JAMA Oncology. News of the outcomes, top-lined in May, triggered a 15.4% rise in Northwest's shares (OTCQB:NWBO) by midday Nov. 18 as the company continued to prepare regulatory filings for the candidate, Dcvax-L.

Patients, Wall Street Stoked for new drugs in Dravet syndrome

With investors watching to find out whether Stoke Therapeutics Inc.’s positive results with a low, single dose of STK-001 for Dravet syndrome (DS) pans out in more extensive research, a number of players large and small are investigating candidates for the rare but dismal form of epilepsy. DS makes up about 0.17% of all epilepsies, striking one in 15,700 people in the U.S., most of whom bear an SCN1A mutation and a confirmed clinical diagnosis of the disease. Three DS drugs have been approved so far by the U.S. FDA.

Tango success shines light on PRMT5 and synthetic lethality

The stock rally by Tango Therapeutics Inc. over the past month or so has further revved the long-percolating interest in protein arginine methyl transferase 5 and in synthetic lethality (SL), where a number of parties have programs ongoing. Other notable developers include Amgen Inc. and Mirati Therapeutics Inc.

Long lung COVID gives broader insights into fibrotic lung disease

An in-depth investigation of the underlying causes of pulmonary symptoms that in some cases persist for months following recovery from the acute stage of COVID-19 has found a distinctive proinflammatory signature in the plasma and airways of affected patients. The research could provide an explanation for the ongoing interstitial lung disease and fibrosis seen in patients who were hospitalized with severe COVID-19, and also point to neutrophils as a specific therapeutic target.

Also in the news

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