Blue­bird shares tank af­ter FDA puts an­oth­er hold on its long de­layed sick­le cell gene ther­a­py

Bluebird bio has been hit with another hold on one of its gene therapy programs.

The company announced Monday that the FDA had placed a partial hold on its sickle cell gene therapy trials for children under the age 18, after one adolescent patient was diagnosed with persistent anemia.

The biotech’s scientists are still trying to determine the cause of the patient’s anemia, but the company emphasized the individual is in otherwise good health and has not suffered any of the pain crises that are a hallmark of the disease. The anemia, they noted, is not severe enough to require transfusions.

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George Scangos (L) and Marianne De Backer

Pi­o­neer­ing biotech icon George Scan­gos hands in his re­tire­ment pa­pers — and this time it’s for re­al

George Scangos, one of the all-time great biotech CEOs, says the time has come to turn over the reins one last time.

The 74-year-old biotech legend spent close to three decades in a CEO post. The first was at Exelixis — which is still heavily focused on a drug Scangos advanced in the clinic. The second “retirement” was at Biogen, where he and his team were credited with a big turnaround with the now fading MS blockbuster Tecfidera. And the third comes at Vir, where he traded in his Big Biotech credentials for a marquee founder’s role back on the West Coast, hammering out a Covid-19 alliance with Hal Barron — then R&D chief at GSK — and breaking new ground on infectious diseases with some high-powered venture players.

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FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

Jeanne Loring, director of the Center for Regenerative Medicine (Credit: Jamie Scott Lytle)

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last July, Jeanne Loring stood on a dirt road surrounded by Florida swampland and watched as a nearby SpaceX rocket blasted into the sky. The payload included a very personal belonging: cell clusters mimicking parts of her brain.

For more than two decades, Loring has been at the forefront of a stem cell field that always seems on the brink of becoming the next thing in medicine, but has been slow to lift off.

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Pa­tient death spurs tri­al halt for Ma­gen­ta Ther­a­peu­tics

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

Bris­tol My­ers claims win with CAR-T ther­a­py Breyanzi in leukemia

Bristol Myers Squibb is looking to expand Breyanzi into more indications — and the pharma’s newest data readout makes progress on that front.

The Big Pharma put out word Thursday that the CAR-T cell therapy met the primary endpoint of complete response rate compared to historical control in a subset of patients with relapsed or refractory chronic lymphocytic leukemia (CLL) that were refractory to a BTK inhibitor and pretreated with a BCL-2 inhibitor.

FDA takes next step in Tor­rent Phar­ma­ceu­ti­cal­s' trou­bled In­dia plant saga, is­sues OAI

The FDA has handed Torrent Pharmaceuticals an official action indicated (OAI) status for a previously inspected manufacturing facility in India.

Torrent Pharma sent a letter to the National Stock Exchange of India earlier this week with word that the manufacturer has received a “communication from the FDA determining the inspection classification as ‘Official Action Indicated’ (OAI)” for one of its sites. An OAI classification from the FDA comes after the agency has completed an inspection and determines if the facility complies with the applicable laws and regulations. Being given an OAI classification means that regulatory or administrative actions will be recommended to Torrent. However, the details on the recommended actions have not been given.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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Vu Truong, Aridis Pharmaceuticals CEO (Aridis/Nasdaq)

Aridis' mon­o­clon­al an­ti­body fails PhI­II, but plans for sec­ond tri­al any­way

Aridis Pharmaceuticals’ monoclonal antibody missed the bar in a Phase III test in ventilator-associated pneumonia caused by the gram-positive bacteria S. aureus, the company announced Wednesday. 

But Aridis is planning for a second Phase III study anyway once it discusses the findings with the FDA and the European Medicines Agency. Execs blamed recruitment challenges stemming from Covid-19 and Russia’s invasion of Ukraine for the miss, cutting their enrollment target in half.