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.

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.

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.