Nick Leschly, bluebird bio CEO (Jeff Rumans)

Af­ter good news in sick­le cell, blue­bird bio fol­lows up with pos­i­tive long-term da­ta in Loren­zo’s Oil dis­ease

Less than a week af­ter blue­bird bio said their sick­le cell gene ther­a­py was “very un­like­ly” to have caused a case of acute myeloid leukemia, the com­pa­ny is pre­sent­ing some more good news for a dif­fer­ent pro­gram.

Re­veal­ing long-term da­ta in the rare ge­net­ic dis­or­der cere­bral adrenoleukody­s­tro­phy (CALD), blue­bird said Mon­day that 27 of 30 evalu­able pa­tients in a piv­otal Phase II/III study were alive and free of ma­jor func­tion­al dis­abil­i­ties at the two-year mark. And they’re al­so high­light­ing da­ta from a fol­low-up study where 14 pa­tients re­main un­af­fect­ed by such dis­abil­i­ties through five years.

Blue­bird is hop­ing to pro­vide an al­ter­na­tive to al­lo­gene­ic stem cell trans­plants, which is the cur­rent stan­dard of care for CALD, al­so known as Loren­zo’s Oil dis­ease, the com­pa­ny’s head of rare ge­net­ic dis­ease R&D Richard Colvin said in a state­ment. The five-year da­ta have es­pe­cial­ly “en­cour­aged” the team that the ther­a­py, dubbed Lenti-D, can have a safe and pro­longed treat­ment ef­fect.

Da­ta come from a to­tal of 32 pa­tients, with a me­di­an fol­low-up of 38.6 months. The fol­low-ups ranged from 13.4 to 82.7 months, and in ad­di­tion to the 14 who reached five years since treat­ment, sev­en of those in­di­vid­u­als have hit the six-year mile­stone.

Two pa­tients dropped out at in­ves­ti­ga­tor dis­cre­tion, as blue­bird has pre­vi­ous­ly said. A third pa­tient al­so — blue­bird re­port­ed sev­er­al years ago — ex­pe­ri­enced rapid pro­gres­sion of the dis­ease ear­ly on in treat­ment and ul­ti­mate­ly died.

An­oth­er two ad­di­tion­al pa­tients have not yet reached the slat­ed two-year time­frame, and nei­ther have shown any ev­i­dence of ma­jor func­tion­al dis­abil­i­ties.

CALD is the most se­vere form of adrenoleukody­s­tro­phy; it’s a rare, X-linked meta­bol­ic dis­or­der that af­fects about one in 21,000 male new­borns around the world, blue­bird says. Mu­ta­tions in the ABCD1 re­sult in tox­ic buildup of very long-chain fat­ty acids, usu­al­ly in the parts of the brain and spinal cord.

The dis­ease is as­so­ci­at­ed with six ma­jor func­tion­al dis­abil­i­ties that blue­bird is look­ing to stamp out with its treat­ment. These in­clude the loss of com­mu­ni­ca­tion, cor­ti­cal blind­ness, the need for tube feed­ing, in­con­ti­nence, wheel­chair de­pen­dence and the loss of all vol­un­tary move­ment.

Blue­bird is al­so eval­u­at­ing Lenti-D in a sep­a­rate Phase III tri­al, look­ing at CALD pa­tients af­ter they’ve re­ceived busul­fan and flu­dara­bine con­di­tion­ing. Re­searchers have treat­ed 19 pa­tients with a me­di­an fol­low-up of 8.6 months, and 17 of those 19 have achieved neu­trophil en­graft­ment. Fif­teen of 19 al­so had platelet en­graft­ment as of the No­vem­ber da­ta cut­off date.

Since this study doesn’t yet have any pa­tients who have reached the 24-month mark, blue­bird’s not re­leas­ing any ef­fi­ca­cy da­ta from this tri­al just yet. All re­port­ed da­ta from the Phase II/III and par­al­lel Phase III stud­ies are cur­rent as of Oc­to­ber 2020, blue­bird said.

The Cam­bridge, MA-based biotech has been plagued by set­backs and safe­ty is­sues over the last year, with two se­ri­ous ad­verse events pop­ping up in the sick­le cell pro­gram last month trig­ger­ing a fresh round of con­cerns over its lentivirus de­liv­ery plat­form. But last week’s news proved a mo­men­tary sigh of re­lief as re­searchers con­tin­ue to look in­to a sec­ond event, thought to be myelodys­plas­tic syn­drome.

With the Lenti-D da­ta be­ing pre­sent­ed at a sci­en­tif­ic fo­rum Mon­day, blue­bird re­mains on track to sub­mit its BLA pitch to the FDA in “mid-2021.”

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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From left: James Brown, Michael Chambers, John Ballantyne

Alde­vron founders back a biotech start­up that's look­ing to end the moral de­bate over cell lines once and for all

For millions of Catholics around the world, the development of new vaccines to combat Covid-19 has sparked a moral dilemma. All the approved vaccines in use relied — in some fashion — on cell lines that were derived from aborted fetal tissue.

While church leaders accepted the vaccines and recommended their use to end the pandemic, a number also highlighted their preference for the mRNA vaccines from Pfizer/BioNTech and Moderna over the J&J and AstraZeneca shots, which they noted were more heavily dependent on cell lines that they found morally objectionable.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.