Af­ter a check­ered mid-stage pro­gram marred by pa­tient deaths, Au­rinia emerged vic­to­ri­ous on Wednes­day with phase III lu­pus nephri­tis da­ta that demon­strat­ed its lead drug is safe and ef­fec­tive for a pa­tient pop­u­la­tion that has few op­tions.

Pe­ter Green­leaf CEO, Au­rinia

Au­rinia’s shares sky­rock­et­ed in Thurs­day morn­ing pre­mar­ket trad­ing, as the Cana­di­an biotech un­veiled plans to sub­mit a mar­ket­ing ap­pli­ca­tion next year.

The 357-pa­tient place­bo-con­trolled AU­RO­RA tri­al test­ed the com­pa­ny’s drug, vo­closporin, in com­bi­na­tion with my­cophe­no­late and low-dose cor­ti­cos­teroids to eval­u­ate whether the ther­a­py could im­prove the speed of and over­all re­nal re­sponse rates in pa­tients with lu­pus nephri­tis, where the au­toim­mune dis­ease rav­ages the kid­neys, trig­ger­ing in­flam­ma­tion that could lead to blood and/or pro­tein in the urine, high blood pres­sure, poor kid­ney func­tion, or kid­ney fail­ure.

Pa­tients giv­en vo­closporin record­ed re­nal re­sponse of 40.8%, while those on the con­trol arm ex­pe­ri­enced a rate of 22.5% — meet­ing the main goal of the study (p < 0.001) at 52 weeks. The Au­rinia ther­a­py al­so in­duced sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments across all pre­spec­i­fied sec­ondary end­points in­clud­ing re­nal re­sponse at 24 weeks, par­tial re­nal re­sponse at dif­fer­ent time points and time to achieve uri­nary pro­tein-to-cre­a­ti­nine ra­tio.

Source: Au­rinia

Click on the im­age to see the full-sized ver­sion

On the safe­ty side, vo­closporin da­ta was al­so de­ci­sive.

Six deaths oc­curred in the study, one in the vo­closporin arm and five in the con­trol group. Se­ri­ous ad­verse events were re­port­ed in 20.8% of vo­closporin pa­tients, ver­sus 21.3% in the con­trol arm.

In 2016, Au­rinia hit a ma­jor road­block with its vo­closporin pro­gram af­ter 13 deaths were record­ed in its phase IIb study – 10 in the low-dose arm, 2 in the high-dose arm and on­ly one in the con­trol group. How­ev­er, 40% of the 265 pa­tients in the study were en­rolled in sites in Asia, where treat­ment stan­dards are dif­fer­ent, Au­rinia had sug­gest­ed at the time.

On Wednes­day, SVB Leerink’s Joseph Schwartz sug­gest­ed Au­rinia’s lat­est da­ta was in-line with his “op­ti­mistic ex­pec­ta­tions.”

“AUPH has ac­cord­ing­ly avoid­ed prob­lem­at­ic sites in Ph.3 AU­RO­RA and to­day’s re­sult shows the fruits of that ef­fort,” he said. “With a clear mor­tal­i­ty ad­van­tage in fa­vor of vo­closporin treat­ment, we think the safe­ty con­tro­ver­sy will take a per­ma­nent back­seat for vo­closporin in lu­pus nephri­tis (LN).”

Au­rinia’s shares flew up 137% to $19.89 in pre­mar­ket trad­ing.

The sys­temic au­toim­mune dis­ease, in which the body’s im­mune sys­tem launch­es an at­tack on its own tis­sues and or­gans, af­fects about 1.5 mil­lion Amer­i­cans, ac­cord­ing to The Lu­pus Foun­da­tion of Amer­i­ca. Rough­ly 60% of lu­pus pa­tients de­vel­op lu­pus nephri­tis and 1 in 4 progress to end-stage re­nal dis­ease.

Lu­pus is a drug de­vel­op­er’s night­mare. In the last six decades, there has been just one FDA ap­proval, with the field re­sem­bling a grave­yard in re­cent years.  Last Oc­to­ber, UCB and Bio­gen’s an­ti-CD40L drug failed in a late-stage study, months af­ter Xen­cor and Sanofi’s Abl­ynx al­so con­ced­ed de­feat in their pro­grams. In Au­gust, how­ev­er, As­traZeneca of­fered a glim­mer of hope af­ter its drug, an­i­frol­um­ab, cleared a Phase III study.

The on­ly bi­o­log­ic so far to win ap­proval for lu­pus is GSK’s Benlysta — which was cleared for adult use in 2011 and for rare cas­es of child­hood lu­pus this year. GSK is in the midst of test­ing Benlysta in com­bi­na­tion with Roche’s rit­ux­imab in the hope the com­bi­na­tion will have a more po­tent ef­fect on the dis­ease ver­sus Benlysta monother­a­py. And last month, Roche said it was work­ing on putting to­geth­er a late-stage study for its mon­o­clon­al an­ti­body Gazy­va in pa­tients with lu­pus nephri­tis.

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
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What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

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But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

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Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.