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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Try­ing to shake up the Parkin­son's par­a­digm, Ab­b­Vie sub­mits NDA for con­tin­u­ous, 24-hour in­fu­sion ther­a­py

AbbVie is approaching the FDA with a new therapy to potentially treat Parkinson’s disease, using prodrugs of two medications commonly used for the condition.

The Big Pharma submitted its NDA for ABBV-951, a solution of levodopa and carbidopa prodrugs being evaluated in advanced Parkinson’s patients who don’t respond well to oral therapy, AbbVie announced Friday morning. Researchers are hoping a positive Phase III study that reads out in late October will help move things along quickly at the agency.

Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Emer Cooke, EMA director (AP Photo/Geert Vanden Wijngaert)

Ahead of FDA, EMA rec­om­mends au­tho­riz­ing new gene ther­a­py treat­ment for ul­tra-rare dis­ease

Aromatic amino acid decarboxylase (AADC) deficiency is an ultra-rare genetic disease that leaves patients unable to produce certain hormones in the brain, such as dopamine and serotonin, usually leading to developmental delays, weak muscle tone and inability to control the movement of the limbs. It can also lead to multiple organ failure.

To date, there have been no treatments approved for AADC deficiency, which has been identified in less than 150 patients.

Ather­sys tries to post-hoc-an­a­lyze its way out of an­oth­er tri­al fail for stroke stem cell ther­a­py

Athersys’ stem cell therapy has failed yet again.

In a 206-person trial conducted in Japan, Athersys’ stem cell therapy for stroke failed its primary endpoint of “excellent outcome,” a combined measure of three stroke recovery scores.

While a greater percentage of patients in the treatment group reached the primary endpoint compared to placebo, that difference was not statistically significant.

Siddhartha Mukherjee (Brian Ach/Getty Images for The New Yorker)

All Blue's $733M bid to ac­quire Zymeworks turns hos­tile as board bat­tles back — af­ter a biotech celebri­ty jumps in

Yesterday, the team at All Blue Capital — bent on the takeover of a badly battered Zymeworks — brought in celebrated oncologist, Pulitzer prize-winning writer and biotech exec Siddhartha Mukherjee to add some glitz to their proposed board. But they’re still not winning over any converts.

This morning, Zymeworks’ board officially turned this acquisition offer into a hostile showdown, rejecting the unsolicited offer and marshaling its forces to prevent a buyout at $10.50 per share.

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