Au­rinia bounces back, shares soar on pos­i­tive da­ta for lu­pus drug

Brad Rovin, OSU Wexn­er Med­ical Cen­ter

Last sum­mer, Au­rinia $AUPH found it­self be­tween a rock and a hard place as it sought to ex­plain a trou­bling clus­ter of deaths in the two dos­ing co­horts — as well as an odd flip-flop in re­sults you might ex­pect to see from the low and high dos­es — used to test their drug vo­closporin for lu­pus nephri­tis. Un­sat­is­fied by the case Au­rinia made for ef­fi­ca­cy and safe­ty at 24 weeks, in­vestors pulled out af­ter look­ing over the fall­out, cra­ter­ing the stock.

Since then, the stock has man­aged to re­trieve much of what it lost that tu­mul­tuous day. And now, Au­rinia will try to make a good im­pres­sion with 48 week da­ta from the Phase IIb study, which post­ed with even bet­ter ef­fi­ca­cy da­ta. And this time the com­pa­ny in­sists it’s ready to step up to a late-stage test to prove it has a drug that can make a dif­fer­ence for pa­tients.

The key da­ta point: Af­ter 48 weeks, the low-dose drug group achieved a com­plete re­mis­sion rate of 49%, up from 33% at 24 weeks, ac­cord­ing to Au­rinia. That co­hort still man­aged to do bet­ter than the high dose arm, with a 40% CR rate. The con­trol arm hit a mere 24%.

Track­ing par­tial re­mis­sions, the low dose hit 68% and 72% in the low and high dose groups, on­ly slight­ly changed from the 24 week re­sults. The con­trol arm al­so held steady with a 48% PR rate.

This time, in­vestors seemed hap­py to ac­cept a suc­cess. Au­rinia’s shares spiked 65%.

The biotech like­ly breathed a sigh of re­lief in as­sess­ing se­ri­ous ad­verse events, with no new deaths to re­port in ei­ther drug arm and 3 deaths and one ma­lig­nan­cy to cite in the con­trol arm. Last sum­mer an­a­lysts 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 at the time, where most of the deaths oc­curred. The deaths were not re­lat­ed to the drug, they said in the ear­li­er call, and might be at­trib­uted to the kind of treat­ment stan­dards in dai­ly prac­tice in Asia. In their state­ment last Au­gust, 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.

As far as the com­pa­ny is con­cerned now, they’ve an­swered all the ques­tions they need to set out in a piv­otal Phase III tri­al.

Brad Rovin, di­rec­tor of nephrol­o­gy and vice chair­man of de­search for the De­part­ment of In­ter­nal Med­i­cine at the Ohio State Uni­ver­si­ty Wexn­er Med­ical Cen­ter, had this to say:

“The AU­RA tri­al’s long-term re­sults con­vinc­ing­ly demon­strate that the ad­di­tion of vo­closporin to stan­dard-of-care treat­ment is su­pe­ri­or to stan­dard-of-care alone. These da­ta are not on­ly sta­tis­ti­cal­ly sig­nif­i­cant, but clin­i­cal­ly im­por­tant. Twice as many pa­tients giv­en 23.7 mg vo­closporin twice dai­ly achieved a com­plete re­nal re­sponse com­pared to those treat­ed with place­bo. This is an im­pres­sive re­nal re­sponse rate and these re­sults may shift the treat­ment par­a­digm of LN. Based on these en­cour­ag­ing da­ta, I am look­ing for­ward to the Phase III tri­al of vo­closporin in LN.”

That Phase III is slat­ed to be­gin next quar­ter, re­gard­less of what the an­a­lysts say next.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Michel Vounatsos, Getty Images

UP­DAT­ED: Stay tuned: Bio­gen’s num­bers are great — it’s their wor­ri­some fu­ture that leaves an­a­lysts skit­tish

Biogen came out with an upbeat assessment of their Q2 numbers today, discounting the arrival of a key rival for its blockbuster Spinraza franchise. But the top execs remain grimly determined to not say much anything new about the sore points that have dragged down its stock, including the future of its big investment in Alzheimer’s or how it plans to invest the considerable cash that the big biotech continues to reap.

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Why wait? Cel­gene re­struc­tures a big Jounce pact — ze­ro­ing in on new I/O path­way with $530M deal and bump­ing ICOS

Celgene’s business team isn’t waiting for the big merger with Bristol-Myers Squibb to go through before syncing its strategy with the new mother ship.

Tuesday evening the big biotech unveiled a $530 million deal — $50 million in upfront cash — to amend their alliance with Jounce Therapeutics $JNCE to gain worldwide rights to JTX-8064, an antibody that targets the LILRB2 receptor on macrophages. Their old, $2.6 billion deal is being scrapped, leaving Jounce with a pipeline that includes the lead drug, the ICOS-targeting vopratelimab.

PACT Phar­ma says it's per­fect­ed the tech to se­lect neoanti­gens for per­son­al­ized ther­a­py — now on­to the clin­ic

At PACT Pharma, the lofty goal to unleash a “tsunami” of T cells personalized for each patient has hinged on the ability to correctly identify the neoantigens that form something of a fingerprint for each tumor, and extract the small group of T cells primed to attack the cancer. It still has a long way to go testing a treatment in humans, but the biotech says it has nailed that highly technical piece of the process.

UP­DAT­ED: My­ovan­t's uter­ine fi­broid drug looks com­pet­i­tive in PhI­II — but can they van­quish mighty Ab­b­Vie?

Vivek Ramaswamy’s Myovant $MYOV has closely matched its positive first round of Phase III data for their uterine fibroid drug relugolix, setting up a head-to-head rivalry with pharma giant AbbVie as the little biotech steers to the market with a planned filing in Q4.

Here’s how Myovant plans to prevail over the AbbVie $ABBV empire.

In the study, 71.2% of women receiving once-daily relugolix combination therapy achieved the clinical response they were looking for, compared to only 14.7% in the control arm. The data comfortably reflected the same outcomes in the first Phase III — 73.4% of women receiving once-daily oral relugolix combination therapy achieved the responder criteria compared with 18.9% of women receiving placebo — which will reassure regulators that they are getting the carefully randomized data that qualifies for the FDA’s gold standard for success.

Lit­tle Mar­i­nus sees its shares eclipsed as the Sage ri­val fails to com­pare on PPD in PhII

The executive team at Sage $SAGE have skirted another potential pitfall on its way to racking up a big future for its depression drug Zulresso.

Little Marinus Pharmaceuticals $MRNS had sought to challenge the Sage drug with an IV formulation — followed by an oral version — of ganaxolone for postpartum depression. But researchers say their Phase II study failed to positively differentiate itself from a placebo at 28 days — leaving them to hold up “clinically meaningful” data within the first day of administration compared to the control arm.

Roche cuts loose Tam­i­flu OTC rights, hand­ing Sanofi the keys as the phar­ma gi­ant dou­bles down on Xofluza

Roche set out to make a better flu medicine than Tamiflu as that franchise was headed to a generic showdown. Now they’ll see just how well Xofluza stacks up against the mainstay drug after handing off over-the-counter rights in the US to Sanofi.

Sanofi $SNY says it will now step in to negotiate a deal with the FDA to steer Tamiflu into the OTC market, a role that could well involve new studies to ease passage of the drug out of doctor’s hands and into the consumer end of the market. And the French pharma giant will have first dibs over “selected” OTC markets around the world as they push ahead.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.