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.

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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Fol­low­ing US, Chi­na hos­pi­tal ef­forts, Gilead plots its own PhI­II tri­als for close­ly watched Covid-19 drug

Gilead is launching its own Phase III trials of remdesivir, the repurposed antiviral that a WHO official called the “one drug right now we think may have real efficacy” against Covid-19 as the novel coronavirus originating from Wuhan, China ravages the world.

Announced just a day after the NIH and the University of Nebraska Medical Center registered their US-based trial online, Gilead’s program will comprise two studies enrolling around 1,000 patients beginning in March. They will recruit primarily in Asian countries but will also include patients from other locations with “high numbers of diagnosed cases,” the company said.

Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Anthony Fauci (AP Images)

UP­DAT­ED: NIH-part­nered Mod­er­na ships off its PhI-ready coro­n­avirus vac­cine can­di­date to a sea of un­cer­tain­ty

Off it goes.

Moderna has shipped the first batch of its mRNA vaccine against SARS-CoV-2 from its manufacturing facility in Norwood, Massachusetts, to the National Institute of Allergy and Infectious Diseases in Bethesda, Maryland, for a pioneering Phase I study.

It’s a hectic race against time. In the 42 days since Moderna selected the sequence they would use to develop their vaccine — a record time, no less — the number of confirmed cases around the world has surged astronomically from a few dozen to over 80,000, per WHO and Johns Hopkins estimates.

The candidate that they came up with, mRNA-1273, encodes for a prefusion stabilized form of the spike protein, which gives the virus its crown shape and plays a key role in transmission. The Coalition for Epidemic Preparedness Innovations, the Oslo-based group better known as CEPI, funded the manufacture of this batch.

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In at least one life-sci hub, gen­der and di­ver­si­ty ini­tia­tives haven’t made a dent

Gender and racial diversity at the top of UK life science companies has hardly budged over the last seven years despite repeated advocacy efforts, according to a new report.

The report, from the recruiting firm Liftstream, found that 14.8% of directors on life sciences boards were women and 21.1% of top executives were women. That’s a modest bump from the 9.8% of directors and 18.1% of executives Liftstream identified in their last report from 2014. The percentage of women CEOs moved from 8% to 9.8%.

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Will a 'risk-of­f' mind­set has­ten cell ther­a­py M&A? Io­vance surges on buy­out chat­ter

Is it time for some cell therapy M&A?

Investors of Iovance Biotherapeutics certainly thought so, sending its stock $IOVA up as much as 40% after Bloomberg reported that the cancer-focused biotech is talking to potential buyers.

While 2019 saw a number of high-profile gene therapy company takeovers — led by Roche’s $4.3 billion bid of Spark as Astellas went for Audentes, Biogen snapped up Nightstar and Vertex absorbed Exonics — large players appeared to prefer partnering on the cell therapy front, particularly when it comes to cancer. Hal Barron put his weight behind Rick Klausner’s startup as he rebuilt GlaxoSmithKline’s cancer pipeline. Takeda turned to MD Anderson to license their natural killer cell therapy.

One less ri­val for Im­muno­vant, as Alex­ion aban­dons FcRn in­hibitor

Less than one year after Alexion parted with $25 million upfront to secure access to a second anti-FcRn asset, it is abandoning the experimental drug. The discontinuation, disclosed at the SVB Leerink Global Healthcare Conference in New York during a fireside chat, bodes well for rival Immunovant.

The drug (ABY-039), partnered for development with Sweden’s Affibody, was forsaken on the basis of early-stage data that was not viewed favorably, Baird and SVB Leerink analysts noted.