Aurinia CEO Peter Greenleaf

Af­ter los­ing race for first to mar­ket in lu­pus nephri­tis, Au­rinia taps Ot­su­ka for vo­closporin li­cens­ing pact abroad

Tiny biotech Au­rinia Phar­ma­ceu­ti­cals is look­ing to slay a gi­ant in Glax­o­SmithK­line’s Benlysta — which just scored a big ap­proval in lu­pus nephri­tis — with its late-stage vo­closporin on the cusp of an FDA ap­proval. Look­ing to take its com­mer­cial plans glob­al, Au­rinia has tapped a Japan­ese phar­ma to ac­cel­er­ate its po­ten­tial vo­closporin launch.

Au­rinia will hand over li­cens­ing rights for lu­pus nephri­tis can­di­date vo­closporin in the EU, Japan and else­where to Ot­su­ka Phar­ma­ceu­ti­cal as the late-stage drug rapid­ly ap­proach­es its FDA re­view date, the com­pa­nies said Thurs­day.

The com­mer­cial tie-up comes with­in hours of British drug­mak­er Glax­o­SmithK­line get­ting the FDA green­light for its Benlysta as the first-ap­proved ther­a­py for LN. Au­rinia’s $AUPH share prices were trad­ing down 12.7% on Thurs­day to around $13.24 at press time, un­der­scor­ing in­vestors’ neg­a­tive re­ac­tion to the news.

Au­rinia will re­ceive an up­front cash pay­ment of $50 mil­lion as part of its deal with Ot­su­ka as well as the pos­si­bil­i­ty for an ad­di­tion­al $50 mil­lion con­tin­gent on sales and reg­u­la­to­ry mile­stones. Au­rinia will then pull in tiered roy­al­ties of 10% to 20% on sales in the EU, Japan, UK, Rus­sia, Nor­way, Switzer­land, Be­larus, Ice­land, Licht­en­stein and Ukraine.

To meet its end, Ot­su­ka ex­pects to file a mar­ket­ing ap­pli­ca­tion for vo­closporin with the EMA in the sec­ond quar­ter and plans to sub­mit in Japan “at a lat­er date,” the com­pa­nies said. Vo­closporin is fac­ing an FDA re­view date of Jan. 22.

Au­rinia, a biotech with no ap­proved ther­a­pies in its port­fo­lio, has a lot rid­ing on LN, a se­vere form of lu­pus that can lead to end-stage re­nal fail­ure, dial­y­sis and kid­ney re­place­ment. But be­ing sec­ond to mar­ket af­ter Big Phar­ma GSK and Benlysta, a nine-year vet­er­an in lu­pus with es­tab­lished re­la­tion­ships with nephrol­o­gists, will be no easy task.

Af­ter ear­li­er test­ing marred by pa­tient deaths, Au­rinia re­leased Phase III da­ta in De­cem­ber 2019 show­ing vo­closporin in com­bi­na­tion with my­cophe­no­late and low-dose cor­ti­cos­teroids im­proved the speed of and over­all re­nal re­sponse rates in pa­tients with LN over place­bo.

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. Vo­closporin al­so post­ed sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments across all pre-spec­i­fied sec­ondary end­points, in­clud­ing re­nal re­sponse at 24 weeks, par­tial re­nal re­sponse at dif­fer­ent time points and time to achieve uri­nary pro­tein-to-cre­a­ti­nine ra­tio.

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.

Au­rinia has had a rough go of it this year, cut­ting an­oth­er late-stage pro­gram for vo­closporin oph­thalmic so­lu­tion (VOS) in No­vem­ber af­ter flop­ping a Phase II/III study in dry-eye dis­ease. Au­rinia por­trayed that for­mu­la­tion as a next-gen fol­low-up to Resta­sis, but the drug couldn’t make its mark in im­prov­ing pa­tients’ con­di­tion.

A win in LN would help turn the tide in Au­rinia’s fa­vor, but it’s al­so rac­ing time as an­oth­er big play­er, Roche’s Gazy­va, looks to en­ter the mar­ket. In June 2019, Roche post­ed Phase II da­ta show­ing Gazy­va on top of stan­dard-of-care ther­a­py best­ed stan­dard of care alone in achiev­ing com­plete re­nal re­sponse for lu­pus nephri­tis pa­tients. The drug earned the FDA’s break­through tag in Sep­tem­ber 2019.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Tom Barnes (Orna)

The mR­NA era is here. MPM be­lieves the fu­ture be­longs to oR­NA — and Big Phar­ma wants a seat at the ta­ble

If the ultra-fast clinical development of Covid-19 vaccines opened the world’s eyes to the promises of messenger RNA, the subsequent delays in supply offered a crash course on the ultra-complex process of producing them. Even before the formulation and fill-finish steps, mRNA is the precious end product from an arduous journey involving enzyme-aided transcription, modification and purification.

For Bristol Myers Squibb, Novartis Institutes for Biomedical Research, Gilead’s Kite and Astellas, it’s time to rethink the way therapeutic RNA is engineered.

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Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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S&P ex­pects steady ero­sion in Big Phar­ma's cred­it pro­file in 2021 as new M&A deals roll in — but don't un­der­es­ti­mate their un­der­ly­ing strength

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Tal Zaks, Moderna CMO (AP Photo/Rodrique Ngowi, via still image from video)

CMO Tal Zaks bids Mod­er­na a sur­prise adieu as biotech projects $18.4B in rev­enue, plots post-Covid ex­pan­sion

How do you exit a company after six years in style? Developing one of the most lucrative and life-saving products in pharma history is probably not the worst way to go.

Tal Zaks, Moderna’s CMO since 2015, will leave the mRNA biotech in September, the biotech disclosed in their annual report this morning. The company has already retained the recruitment firm Russell Reynolds to find a replacement.

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Glax­o­SmithK­line re­thinks strat­e­gy for Covid-19 an­ti­body — not the Vir ones — af­ter tri­al flop. Is there hope in high-risk pa­tients?

In the search for a better Covid-19 therapeutic, GlaxoSmithKline and Vir have partnered up on two antibodies they hope have a chance. GSK is also testing its own in-house antibody, and early results may have shut the door on its widespread use.

A combination of GSK’s monoclonal antibody otilimab plus standard of care couldn’t best standard of care alone in preventing death and respiratory failure in hospitalized Covid-19 patients after 28 days, according to data from the Phase IIa OSCAR study unveiled Thursday.

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Bio­phar­ma's suc­cess rate in bring­ing drugs to mar­ket has long been abysmal. Can new tools help rewrite that trou­bled past?

In 2011, a team of researchers at British drugmaker AstraZeneca had a problem they were looking to solve.

For years, drug discovery and development were a wasteland for innovation. Novel drugs largely fell into one of two categories — monoclonal antibodies and small molecules — and new therapeutic modalities were hard to come by. After a rush of promising approvals in the late 1990s — including then-Biogen’s CD20 targeting antibody breakthrough Rituxan — the field stagnated and attrition rates stayed sky-high. What exactly is the industry doing wrong? AstraZeneca asked itself.

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