Sud­den death forces Sophiris to halt fol­lowup ther­a­py in prostate can­cer study — shares crushed

Small-cap biotech Sophiris $SPHS has hit the brakes on its dos­ing sched­ule for pa­tients in its Phase IIb prostate can­cer study af­ter one of the sub­jects in the tri­al died short­ly af­ter re­ceiv­ing a sec­ond dose. 

There’s not much to go on here. 

Sophiris’ state­ment says on­ly that in­ves­ti­ga­tors study­ing top­salysin halt­ed any fur­ther sec­ond dos­es when one of the most re­cent pa­tients to get a sec­ond dose died on the same day. Ex­ecs not­ed the change-up af­ter tout­ing some of the re­spons­es they’ve been track­ing among a few dozen pa­tients in the tri­al. 

We have no de­tails of what killed the pa­tient. Dose-lim­it­ing tox­i­c­i­ties, though, rep­re­sent a threat to any drug study.

Sophiris shares cratered on the news, plung­ing 49%.

San Diego-based Sophiris hasn’t re­ceived much at­ten­tion over the years, but the team there has been work­ing on this drug for 14 years af­ter ini­tial­ly li­cens­ing it from Johns Hop­kins. It’s de­scribed as a po­tent med that is ac­ti­vat­ed on­ly be en­zymes found in prostate spe­cif­ic anti­gen. The com­pa­ny re­port­ed a suc­cess­ful Phase III in be­nign prostate hy­per­pla­sia in late 2015, with a weak p = 0.043, but said it couldn’t do a sec­ond piv­otal with­out a deal or sig­nif­i­cant new mon­ey. That’s when they went in­to mid-stage stud­ies on prostate can­cer.

The biotech al­so not­ed to­day that the in­de­pen­dent mon­i­tor­ing board had giv­en re­searchers a green light on the study, af­ter eval­u­at­ing re­spons­es among 38 pa­tients, in­clud­ing 7 who had re­ceived a sec­ond course of ther­a­py. The pa­tient who died was in a group of 3 who re­ceived a fol­lowup treat­ment af­ter that.

The CEO said he was sad­dened, but sought to re­as­sure in­vestors, as­sert­ing their suc­cess so far.

“We are very en­cour­aged by the safe­ty and biop­sy re­sults from a sin­gle ad­min­is­tra­tion of top­salysin in the Phase IIb study. Biop­sy re­sults im­proved from what we saw in the Phase IIa proof of con­cept tri­al and safe­ty and tol­er­a­bil­i­ty re­mains in-line with what we have seen his­tor­i­cal­ly,” said CEO Ran­dall Woods in a pre­pared state­ment. “We be­lieve that the safe­ty and biop­sy da­ta from the first ad­min­is­tra­tion of top­salysin sup­ports mov­ing for­ward in­to po­ten­tial reg­is­tra­tion stud­ies. We will con­tin­ue to eval­u­ate whether fu­ture clin­i­cal de­vel­op­ment will in­clude an op­tion to ad­min­is­ter a sec­ond dose as we re­ceive more in­for­ma­tion about the pa­tient death and ad­di­tion­al in­for­ma­tion from the 10 pa­tients who re­ceived a sec­ond dose. We will be able to eval­u­ate this to­wards the end of this year.”

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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Emma Walmsley, GlaxoSmithKline CEO (Kevin Dietsch/Pool via CNP/Alamy)

Glax­o­SmithK­line hus­tles the 7th PD-1 past the fin­ish line with Jem­per­li. But how big will up­take be?

Everything came up sevens for GlaxoSmithKline on Thursday as the pharma notched the seventh PD-1 approval seven years after the first such drugs were OK’ed in Keytruda and Opdivo. But will it bring GSK good fortune?

The FDA granted accelerated approval to dostarlimab, to be branded Jemperli, to treat recurrent or advanced endometrial cancer in a specific subset of patients following platinum-based chemo. It’s a drug that came to GSK through its buyout of Tesaro, which it snapped up for $5.1 billion back in December 2018.

JP Gabriel, Ocugen

JP Gabriel watched from the bleach­ers as the pan­dem­ic raged. Now head of sup­ply chain at Ocu­gen, he's ready to bat

The world was in the middle of the most pressing public health risk his generation had ever seen, and JP Gabriel felt like he was sitting on the sidelines. As a VP of biologics and mRNA manufacturing at Ultragenyx, Gabriel watched from the sidelines as players like Pfizer/BioNTech and Moderna used mRNA tech to chase their own Covid-19 vaccines.

This month, Gabriel got the chance to get his hands dirty against the pandemic — but it won’t be with mRNA.

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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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