Alex­ion clinch­es aHUS ap­proval for Ul­tomiris as the clock ticks on Soliris con­ver­sion

Alex­ion has racked up a sec­ond ap­proval for Ul­tomiris, the suc­ces­sor ther­a­py to Soliris, as its main­stay block­buster ther­a­py faces a patent re­view process that could dras­ti­cal­ly short­en its patent ex­clu­siv­i­ty.

John Orloff

The FDA OK for atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) on Fri­day was wide­ly ex­pect­ed af­ter Alex­ion post­ed a full slate of pos­i­tive Phase III da­ta in Jan­u­ary. But reg­u­la­tors al­so flagged con­cerns about se­ri­ous meningo­coc­cal in­fec­tions, slap­ping a black box warn­ing on the la­bel and man­dat­ing a REMS.

The late-stage re­sults, which in­clud­ed on­ly adults, were “broad­ly com­pa­ra­ble to ex­ist­ing Soliris da­ta in sim­i­lar pa­tient pop­u­la­tions, but with added con­ve­nience of every 8 week dos­ing,” SVB Leerink an­a­lysts Ge­of­frey Porges not­ed at the time.

Among 56 en­rolled, 53.6% demon­strat­ed a com­plete re­sponse af­ter 26 weeks, mea­sur­ing up to a 56% his­toric re­sponse rate for Soliris.

While an­oth­er Phase III in pe­di­atric and ado­les­cent pa­tients is still on­go­ing, the com­pa­ny dis­closed that in the ini­tial treat­ment pe­ri­od 71% of chil­dren demon­strat­ed a com­plete re­sponse. Out of 16 pa­tients, 14 were eval­u­at­ed for that in­ter­im analy­sis. The FDA went ahead and cleared it for use by in­fants as young as one month old, mark­ing the first pe­di­atric ap­proval for the drug.

Drugs for aHUS — a chron­ic, pro­gres­sive ul­tra-rare dis­ease that can have dev­as­tat­ing ef­fects on the kid­neys — are mea­sured by their abil­i­ties to clear blood clots in small blood ves­sels through­out the body, or throm­bot­ic mi­croan­giopa­thy (TMA).

Ul­tomiris works by in­hibit­ing un­con­trolled com­ple­ment ac­ti­va­tion, specif­i­cal­ly block­ing the C5 path­way. It’s al­so ap­proved in the US and Eu­rope as a ther­a­py for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria.

“The con­se­quences of un­con­trolled com­ple­ment ac­ti­va­tion, like or­gan fail­ure and po­ten­tial­ly death, cre­ate sig­nif­i­cant chal­lenges and un­cer­tain­ty for peo­ple and fam­i­lies fac­ing aHUS,” said John Orloff, head of R&D, in a state­ment. “Based on the Phase 3 da­ta, which demon­strat­ed clin­i­cal­ly mean­ing­ful ben­e­fits in peo­ple with aHUS, we be­lieve UL­TOMIRIS has the po­ten­tial to be­come the new stan­dard of care for this dev­as­tat­ing dis­ease.”

Con­vinc­ing physi­cians and pa­tients would be cru­cial. In Au­gust, the US Patent Tri­als Ap­peal Board (PT­AB) agreed to con­duct an in­ter partes re­view on Soliris af­ter Am­gen, which is push­ing a biosim­i­lar to the com­ple­ment in­hibitor, launched an ef­fort to over­turn patents on the ac­tive in­gre­di­ent.

If Am­gen pre­vails, Soliris’ ex­clu­siv­i­ty could ex­pire as ear­ly as 2022. Cred­it Su­isse’s Mar­tin Auster has pre­vi­ous­ly mod­eled a rough­ly 80% con­ver­sion to Ul­tomiris in three to four years across the PNH and aHUS fran­chis­es.

The ba­ton is now in the com­mer­cial team’s hands.

Porges ex­pects US con­ver­sion ef­forts for Ul­tomiris to be­gin im­me­di­ate­ly, with the EU and Japan to fol­low next year. From his note out late Fri­day:

We es­ti­mate 2020 US Ul­tomiris aHUS rev­enues of ~$140mm, in­creas­ing to ~$640mm in 2025, rep­re­sent­ing ~72% of com­bined US Soliris/Ul­tomiris aHUS sales. We con­tin­ue to re­main op­ti­mistic about the prospects for Alex­ion’s com­ple­ment fran­chise and be­lieve fur­ther in­di­ca­tions of rapid con­ver­sion across in­di­ca­tions and ge­o­gra­phies, as well as fur­ther life-cy­cle man­age­ment op­por­tu­ni­ties from the Achillion (ACHN, MP) ac­qui­si­tion (as­sum­ing FTC clear­ance), could in­crease Alex­ion’s growth out­look over both the near- and longer-term; and po­ten­tial­ly draw in­vestors back in­to the stock over the next 6-12 months.

Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

A low-pro­file biotech bests Re­gen­eron in high-pro­file patent suit

For nearly a decade now, the low-profile Cambridge biotech Kymab has been battling in US, UK, Japanese and Australian courts with the biotech behemoth Regeneron.

Regeneron has turned itself into a $70 billion company off of a platform of transgenically humanized mice they can use to make antibodies for anything from Ebola to colorectal cancer. The technology took decades and billions to build, 20 years from the company’s founding to the first approved drug. And the company guards and touts it zealously, breaking their production process down into various branded components — Velocimmune, Velocigene, Velocimouse and four other Velocis — and sometimes suing would-be copycats. In 2014, most notably, they sued two Pfizer-backed entities for patent infringement.

Bull­ish biotech mar­ket pro­pels Pli­ant to $144M IPO — as No­var­tis pro­vides a $10M boost

After pharma partner Novartis boosted its IPO with a $10 million private placement, Pliant Therapeutics has wrapped its journey to the Nasdaq on a high note.

Pliant had penciled in a $86 million raise back in May. But as has become the norm in recent months, that initial number has turned out to be a mere placeholder, making way for the final haul of $144 million.

The South San Francisco biotech did so by pricing at $16, the high end of the range, while bringing the number of shares offered up to 9 million.

Cameron Durrant, Humanigen CEO (Columbia University Technology Ventures via YouTube)

Cameron Dur­rant hus­tled his way from the OTC side­lines right in­to the Covid-19 drug race. Death or glo­ry lies straight ahead

Over the past few months, Covid-19 has gone from being a monolithic threat to one of the biggest overnight boons the biopharma industry has ever seen. And amid all the furor over Moderna’s swelling stock price, plenty of chatter over what new drugs and vaccines will cost and investors’ uninhibited zeal for all things related to pandemic products, it’s been one little biotech’s golden ticket back from the land of the living dead.

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Covid-19 roundup: BAR­DA sup­ports Op­er­a­tion Warp Speed with big $628M con­tract to ser­vice Amer­i­ca's vac­cine pro­duc­tion needs

Another BARDA contract designed to service America’s Covid-19 vaccine needs has been deployed.

The White House-led initiative designed to bankroll development to bring a vaccine to the American public by this fall — Operation Warp Speed — has via BARDA handed a meaty contract to the maker of an FDA-licensed anthrax vaccine to open up its manufacturing apparatus to shore up production of Covid-19 vaccines.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

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