| |   | And hello. Today, Adam Feuerstein catches us up on a new RNA therapy from Arrowhead Pharmaceuticals, we've got a STAT investigation into Covid-19 test maker Cue Health, and more.
- Meghana | | | Does Cue Health have anything else to offer? Before the pandemic, diagnostics maker Cue Health didn’t have a single product on the market. But in July 2020, it inked a deal with the NBA to provide a Covid-19 test to the league’s many players. By that October, it won a $481 million deal with the Trump administration to provide its Covid tests to people all around the country. The company soon went public with a $3 billion valuation. But Cue Health’s financial health is quite precarious, a STAT investigation finds. It had a difficult time scaling up manufacturing and delivering its tests on time. The company told its employees last week that it would lay off 170 manufacturing workers — about 10% of its workforce. Cue Health has little to offer beyond basic Covid-19 tests. And as the demand for these tests dwindles, the company is finding itself in a difficult position. Read more. | Arrowhead Pharma’s RNA treatment reduced toxic protein levels An experimental RNA-based treatment sharply reduced levels of a toxic, mutated protein that causes liver damage in patients with an inherited disease called alpha-1 antitrypsin deficiency, or AATD, according to results from a mid-stage clinical trial presented Saturday by its maker Arrowhead Pharmaceuticals. The treatment, called fazirsiran (formerly ARO-AAT) is being developed in a partnership with the Japanese drugmaker Takeda Pharmaceuticals. All 16 patients in the study given injections of fazirsiran responded to the treatment, with a median reduction of mutant AAT protein reaching 83% at week 24 or 48. All patients showed corresponding reductions in related protein and enzyme biomarkers associated with liver damage. At the highest doses of fazirsiran tested in the study, seven of 12 patients had reversal of liver fibrosis, including two patients with cirrhosis. The study results were also published Saturday in the New England Journal of Medicine. AATD is a genetic disease that prevents the liver from making a protein called AAT needed to protect lung tissue from being damaged by other enzymes. People born with AATD either don’t make enough AAT, or the protein is abnormal and isn’t released into the bloodstream. A buildup of abnormal AAT damages the liver. Serious lung injury caused by the absence of the protective protein typically follows. Arrowhead and Takeda are developing fazirsiran to address the liver damage caused by AATD. The drug works by silencing the defective gene that produces the abnormal, or mutated AAT protein. Fazirsiran is unable to address the need for protective AAT protein to reach the lungs, so patients also require regular infusions of replacement AAT protein. Vertex Pharmaceuticals has taken a different approach to treating AATD with drugs designed to produce healthy, functional AAT protein, but so far, the work has not been successful. A larger, potentially pivotal, placebo-controlled clinical trial of fazirsiran is currently underway. Arrowhead and Takeda expect results to read out later this year. | Democrats' Last Chance for Drug Pricing Reform? The drug pricing debate remains a central conversation in Congress. On July 20, STAT sits down with key decision-makers about the latest developments from Capitol Hill and the White House. Join them. | Gilead wins 'colossal financial fraud' lawsuit Gilead Sciences has reached a $33 million settlement with a Florida clinic that was, according to the company, engaged in a “dangerous and fraudulent scheme” to exploit a patient assistance program for HIV medications. A number of clinics, pharmacies, and lab testing facilities were working together to illegally obtain and resell thousands of bottles of two different HIV prevention drugs — collectively pocketing $43 million dollars in profits. The settlement was with one such clinic, Well Care, which allegedly used homeless and low-wage earners to apply frequently to the patient assistance program to receive free HIV prevention pills. The clinic would repurchase them for as little as $10, repackage them, and then sell them to other program participants or on the black market. The clinic, and others it worked with, could then make more than $1,000 per bottle. Read more. | Pfizer’s new booster protects against Omicron Pfizer’s updated Covid-19 boosters prompted a stronger antibody response against the Omicron variant. The results are similar to those released by Moderna earlier this month, and data from both companies will be evaluated Tuesday by an FDA advisory committee. Regulators are trying to determine which Covid strains should be included in booster shots for the fall, and the companies will need advance notice to adequately prepare. Pfizer’s new booster “improves the neutralizing antibody response to Omicron BA.1 compared to the prototype vaccine” while not impacting efficacy against the original incarnation of the virus, FDA briefing documents say. However, there are “caveats to be noted relevant to the interpretation of these data.” Read more. | More reads - As muscular dystrophy drug meets key goal, Italfarmaco plans march forward to regulators. MedCity News
- BioMarin’s hemophilia gene therapy recommended for approval in Europe. BioPharma Dive
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