Fac­ing ques­tions on pa­tient deaths, Au­rinia shares crater in wake of lu­pus study

Au­rinia Phar­ma­ceu­ti­cals man­aged to kick up a brief spike in its share price this morn­ing af­ter re­leas­ing some up­beat num­bers from a Phase IIb study of its lu­pus drug vo­closporin. But it didn’t last long. The shares $AUPH quick­ly cratered, plung­ing by 48%, as ques­tions turned to a clus­ter of deaths tracked in its two dosage arms as well as the cost of the piv­otal study need­ed for an ap­proval.

At first blush, the num­bers in the 265-pa­tient study looked sol­id. The low dose of the drug hit the pri­ma­ry end­point on com­plete re­spons­es for the lu­pus pa­tients. But dur­ing the fol­lowup call, an­a­lysts ques­tioned why the low dose would beat out the high dose on CRs. They al­so ze­roed in on 13 deaths record­ed in the study – 10 in the low-dose arm, 2 in the high-dose arm and on­ly one in the con­trol group.

Forty per­cent of all the pa­tients in the study were en­rolled in Asia, com­pa­ny ex­ecs re­spond­ed, where most of the deaths oc­curred. The deaths were not re­lat­ed to the drug, they said in the call, and might be at­trib­uted to the kind of treat­ment stan­dards in dai­ly prac­tice in Asia. In the state­ment, the com­pa­ny con­ced­ed that:

The over­all rate of se­ri­ous ad­verse events (SAEs) was high­er in both vo­closporin groups but the na­ture of SAEs is con­sis­tent with high­ly ac­tive LN.

Mary Anne Doo­ley, the chief in­ves­ti­ga­tor for the study, main­tained that vo­closporin “could po­ten­tial­ly change the cur­rent treat­ment par­a­digm for LN.”

In­ves­ti­ga­tors com­bined vo­closporin to the cur­rent stan­dard of care of my­cophe­no­late mofetil, com­par­ing it to a com­bo us­ing a place­bo.

The drug, though, wasn’t al­ways that much bet­ter than the place­bo/SoC arm. In the study, 32.6% of pa­tients on low dose achieved CR, com­pared to 27.3% on high dose and 19.3% in the con­trol arm. Leerink’s Joseph Schwartz not­ed that the deaths will need to be stud­ied more, adding that the pos­i­tive re­sults al­so left some­thing to be de­sired. He added:

“At first glance, the ef­fi­ca­cy seen in the drug arms looks low­er than what we had ex­pect­ed, but still re­spectable. The study was 80% pow­ered to de­tect a re­mis­sion rate of 41% for the drug ver­sus 20% for place­bo based on a planned sam­ple size of 258 sub­jects. CR rates as low as 35% vs. 15% or as high as 47% ver­sus 25% would al­so main­tain 80% pow­er. The re­verse dose re­sponse was sur­pris­ing, par­tic­u­lar­ly since this would have been pulled down by ac­count­ing for the rel­a­tive­ly high num­ber of deaths in the drug arm.”

The Cana­di­an biotech not­ed that it had $12.1 mil­lion in cash at the end of H1, leav­ing it look­ing for fresh funds with a bat­tered stock price.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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Elizabeth Nabel speaks at a news conference, Oct. 7, 2019 (Elise Amendola/AP Images)

Brigham and Wom­en's pres­i­dent Eliz­a­beth Nabel fol­lows Mon­cef Slaoui off Mod­er­na's board

Amid recent scrutiny on how Moderna’s top executives have been cashing out their increasingly valuable shares, the biotech is parting ways with a board member who’s also heading a hospital where its Covid-19 vaccine is being tested.

Elizabeth Nabel — the president of Brigham and Women’s Hospital — has followed in Moncef Slaoui’s footsteps in resigning from Moderna’s board of directors. She took the role in 2015, two years before the Operation Warp Speed leader did; and as with Slaoui and MIT professor Robert Langer, her term was due to expire in 2021.

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Roche de­clares a PhI­II fail­ure for Covid-19 as the IL-6 re­pur­pos­ing the­o­ry bites the dust

Another big IL-6 drug has failed to move the needle for Covid-19 patients, leaving that particular field of repurposed drug R&D on the ropes for the pandemic.

This morning it was Roche’s turn to outline a Phase III failure for Actemra, adding compelling data that have now all but extinguished the theory that an IL-6 drug could significantly help the most severely afflicted patients. That comes just weeks after Regeneron and Sanofi hit the red light on their trial for Kevzara after getting back-to-back readouts that made Roche’s trial a long shot at best.

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Tony Coles, Cerevel Therapeutics CEO

Adding $445M, Tony Coles and his big Pfiz­er neu­ro spin­out hitch a ride to Wall Street on Per­cep­tive’s SPAC

Two years ago, after Pfizer abruptly shut down its entire neuroscience division, Bain Capital bet $350 million that those assets were still worth something and packaged them into a new biotech: Cerevel Therapeutics. A year later, they got seasoned executive Tony Coles, who had recently jumped back into the C-suite of another neuroscience startup, to run the company.

Now Coles is steering Cerevel public, in what he says is the largest ever transaction of its kind. Cerevel has agreed to merge with Perceptive Advisors’ specialty acquisition company ARYA II. Between the roughly $125 million Perceptive raised through ARYA and an additional investment of $320 million Bain Capital, Perceptive and — yes, really — Pfizer, among others, Cerevel will now move forward with an added $445 million in its coffers.

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Ludwig Hantson, Alexion CEO

UP­DAT­ED: The lead drug in Alex­ion’s $930M buy­out deal last fall just flopped — adding in­jury to an­a­lysts’ M&A in­sults

When Alexion $ALXN put down $930 million in cash last fall to buy Achillion, the biotech’s top execs were particularly proud of 2 clinical-stage assets, with a spotlight on the lead drug danicopan (ACH-4471) in Phase II. That drug, along with a companion therapy in Phase I, fit right in their R&D wheelhouse, noted CEO Ludwig Hantson.

But now the lead drug, redubbed ALXN2040, is being washed out and repositioned after failing 2 Phase II trials for C3 Glomerulopathy.

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Robert Nelsen (Illustration by Emma Kumer for Endpoints News)

Af­ter Big Phar­ma aban­doned in­fec­tious dis­eases, 5 biotech con­trar­i­ans de­cid­ed to go all in. Then Covid-19 changed every­thing

Bob Nelsen had been quietly wondering how to eradicate viruses for years, before one day in 2015, he welcomed a pair of immunologists into the ARCH Venture Partners offices on the 34th floor of Seattle’s Wells Fargo Building.

Louis Picker and Klaus Früh, professors at Oregon Health & Science University, had by then spent 5 years running around the country in search of funding for their startup, TomegaVax, and Früh, at least, was nearing wit’s end. The Gates Foundation was interested but told them they needed other investors. Investors told them to come back with more data, pharmaceutical executives said they’re in the wrong game — too little money to be made fighting infectious disease. Still, a well-connected board member named Bob More landed them a meeting with the coveted venture capitalist, and so, in a narrow conference room overlooking the Puget Sound, Picker prepared to again explain the idea he had spent 15 years on: re-engineering a benign microbe into the first vaccines for HIV and better ones for hepatitis and tuberculosis.

“This lightbulb went on his head,” Picker recalled in a recent interview. “Most of them just didn’t get it. And Bob’s hit.”

By that point, Nelsen was more than just a venture capitalist. Scraggly and greying but no less opinionated at 52, he was mobbed at biotech conferences, having earned a reputation for crass wisdom and uncanny foresight, for making big bets on big ideas that changed medicine. Those ideas included DNA sequencing, which he first cut a check for in the 90s, and leveraging the immune system to tackle cancer. He earned millions making billion-dollar companies.

Yet for years he had harbored an almost singular obsession: “I hate viruses,” he told Forbes in 2016. He told me he was “pissed off” at them. The obsession drove him to his first biotech investment in 1993, for an inhalable flu vaccine approved a decade later and still in use. And it drove him to invest in CAR-T as a potential cure for HIV, years before it proved a wildly effective treatment for some cancers.

Now, listening to Picker talk about T cells and antibodies and the curious biology of cytomegalovirus, Nelsen began wondering if it was time for another bet. Picker’s technology was not only promising, he reasoned, it could be the basis of a company that changed how researchers approached viruses. Instead of trying to come up with an antidote for every pathogen, you could do what cancer researchers had learned to do, and harness the immune system to do the work for you.

This wasn’t a popular opinion at the time. “It’s like the least trendy idea in the world,” Nelsen told me. “People would say, ‘Why the hell are you going into infectious disease?’”

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Severin Schwan, Roche CEO (Georgios Kefalas/Keystone via AP Images)

UP­DAT­ED: Genen­tech slash­es near­ly 500 jobs in strate­gic shift to 'lo­cal health­care ecosys­tems.' Clin­i­cal spe­cial­ists make up the biggest group

Deep into the pandemic, the executives at Roche are executing an unusual plan to lay off nearly 500 workers at South San Francisco-based Genentech.

The huge Roche subsidiary handed in their WARN notice to the state a few days ago, noting that 474 staffers were being permanently cut from the payroll.

A spokesperson for the company told me via e-mail:

The majority of these roles are from our field organization and, though formally associated with our South San Francisco headquarters, are actually located throughout the United States. These position eliminations are not related to the COVID-19 pandemic, the clinical trials for which Actemra is being studied in COVID-19 pneumonia, or any specific therapeutic area.

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi prunes its pipeline, tak­ing the ax to a line­up of drug, vac­cine pro­grams

Sanofi swept away a lineup of drug and vaccine programs in its Q2 update, starting with a late-stage vaccine play with Daiichi Sankyo and running right through setbacks on their CD38 drug, the anti-inflammatory Kevzara, as well as a troubled alliance with Denali on ALS.

Gone is a longstanding Phase III development program with Daiichi Sankyo for a pediatric pentavalent vaccine in Japan, though there’s no word on what prompted the pullback.

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Test re­sults in hand, Thrive rais­es $257M to push liq­uid biop­sy to­ward ap­proval

Three months after announcing the results of a landmark trial, Thrive Earlier Detection has raised $257 million to put their liquid biopsy cancer test into a pivotal trial.

Thrive started raising for the Series B immediately after the study results were published in Science at the end of April. That study, run across 10,000 women at the Geisinger Health System, showed for the first time that a blood test could help doctors diagnose certain types of cancer in patients who did not yet show symptoms, more than doubling the percentage of cancers that were detected.