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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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