No­vo Nordisk gets PhI­II he­mo­phil­ia tri­als for a Roche ri­val back on track af­ter com­plet­ing work-around on blood clot threat

Af­ter abrupt­ly shut­ting down three he­mo­phil­ia stud­ies back in March due to se­ri­ous safe­ty is­sues, No­vo Nordisk is get­ting things go­ing again.

The Dan­ish phar­ma gave the go-ahead to re­sume Phase III tri­als for its con­cizum­ab can­di­date, which are in­ves­ti­gat­ing sub­cu­ta­neous pro­phy­lax­is treat­ment in he­mo­phil­ia A and B pa­tients re­gard­less of in­hibitor sta­tus. No­vo Nordisk had halt­ed the stud­ies af­ter three pa­tients ex­pe­ri­enced non-fa­tal blood clots.

In a state­ment, the com­pa­ny said it had found a “new path for­ward” for con­cizum­ab. The FDA has agreed to new safe­ty mea­sures and guide­lines and lift­ed the clin­i­cal hold, No­vo Nordisk said.

Asked to ex­pand on these ad­di­tion­al mea­sures, a No­vo spokesper­son said they in­clude “spe­cif­ic rec­om­men­da­tions for the treat­ment of break­through bleed­ing episodes, both in terms of dos­ing and fre­quen­cy, man­dat­ing the use of low­er dos­es of ad­di­tion­al he­mo­sta­t­ic agents, as well as a new con­cizum­ab dos­ing reg­i­men, with an ini­tial dai­ly dose of 0.20 mg/kg con­cizum­ab for all pa­tients.”

These tri­als had ini­tial­ly be­gun in late 2019 af­ter No­vo had re­leased pos­i­tive proof-of-con­cept da­ta. Con­cizum­ab is an an­ti-tis­sue fac­tor path­way in­hibitor that tar­gets Fac­tor Xa, at­tempt­ing to lim­it its ac­ti­va­tion. If the can­di­date works, it could re­store the de­fi­cien­cies present in he­mo­phil­ia A (fac­tor VI­II) and he­mo­phil­ia B (fac­tor IX).

He­mo­phil­ia drugs are de­signed to pre­vent un­con­trolled bleed­ing in pa­tients and the cur­rent mar­ket leader is Roche’s Hem­li­bra, which No­vo had hoped to ri­val. So far in 2020, Hem­li­bra has net­ted Roche over $700 mil­lion in US sales and topped $1 bil­lion glob­al­ly. The drug ac­counts for 23% of US pa­tient share, ac­cord­ing to the phar­ma’s sec­ond quar­ter earn­ings re­port.

But the he­mo­phil­ia are­na has been trend­ing to­ward new­er gene ther­a­py treat­ments for some time. Roche’s own ther­a­py, com­ing out of the biotech Spark Ther­a­peu­tics, ran in­to some bumps back in 2017 for he­mo­phil­ia A and now ex­pects to start a Phase III some­time in 2021. That put it be­hind fron­trun­ner Bio­Marin, which ex­pects an FDA de­ci­sion on its he­mo­phil­ia A pro­gram be­fore the end of Au­gust.

Should Bio­Marin re­ceive ap­proval, it would be the world’s first gene ther­a­py pro­gram ap­proved for he­mo­phil­ia A. But it’s shown signs of trou­ble as well, as mean Fac­tor VI­II ac­tiv­i­ty lev­els have slid in each suc­ces­sive year. CEO JJ Bi­en­aimé has in­di­cat­ed the biotech in­tends to charge some­where in the $2 mil­lion to $3 mil­lion range, a price tag that could make the ther­a­py the most ex­pen­sive sin­gle-use ther­a­py.

Sang­amo and Pfiz­er are al­so in the he­mo­phil­ia gene ther­a­py game, and while they’re be­hind both Bio­Marin and and Spark, stronger da­ta on dura­bil­i­ty could pre­vent com­peti­tors from cap­tur­ing and keep­ing mar­ket share.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

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:

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bay­er plots a ma­jor facelift at Berke­ley cam­pus, un­cork­ing a 30-year, $1.2B plan to dri­ve cell and gene ther­a­pies

Bayer first set roots in Berkeley back in 1974, when it was still operating as Miles Labs. The site has pumped out three hemophilia A treatments for distribution worldwide; but now, as the pharma continues its cell and gene therapy push, it has something bigger in mind.

Bayer is planning a 30-year revamp at the campus, which includes 918,000 square feet in new buildings and double the jobs, according to a report by the Bay Area Council Economic Institute.

LLS backs 5 new can­cer drug projects with up to $50M; Trodelvy con­tin­ues to im­press with more TNBC da­ta

The Leukemia and Lymphoma Society has tapped 5 new early-stage projects to back with up to $10 million each in fresh investments. The 5 biotechs are:

— Caribou, headed by Rachel Haurwitz and co-founded by Jennifer Doudna, is working on next-gen, off-the-shelf CAR-Ts to replace the patient-derived cells now in use.

— The LLS supported NexImmune’s IPO, helping fund its work on nanoparticles that can gin up an immune response directed at cancer cells. The biotech has 2 projects now in Phase I trials.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 107,400+ biopharma pros reading Endpoints daily — and it's free.