Alex­ion preps an FDA pitch on Soliris suc­ces­sor while sig­nal­ing more deals ahead for rare dis­ease drugs

Alex­ion used its Q1 up­date Thurs­day to un­veil an­oth­er set of Phase III non-in­fe­ri­or stats for its Soliris suc­ces­sor, adding to its reg­u­la­to­ry pack­age for new ap­provals that should ship lat­er this year.

Fol­low­ing ear­li­er proof of non-in­fe­ri­or­i­ty for treat­ment-naive pa­tients, the new Phase III high­light­ed their suc­cess­ful switch­ing of Soliris pa­tients to ALXN1210, a shift from dos­ing every two weeks to eight weeks.

Lud­wig Hantson

To be sure, the in­ves­ti­ga­tors un­der­scored the same kind of im­prove­ments they found over Soliris on key mea­sures, but there was noth­ing sta­tis­ti­cal­ly sig­nif­i­cant about it. That will be good for shoring up their de­fense of the fran­chise drug for years to come — com­pa­ny ex­ecs talked of patents stretch­ing out to 2035 — as ri­vals press along with new drugs they hope can beat the stan­dard ther­a­py for parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH).

Now the biotech is prep­ping reg­u­la­to­ry pitch­es that should ar­rive in a mat­ter of months.

Ear­li­er this month Alex­ion an­nounced a deal to buy a Stock­holm-based biotech for $855 mil­lion in cash, bag­ging a new drug for rare cas­es of Wil­son dis­ease. The com­pa­ny is us­ing that as a mod­el for more deals just like it.

Paul Clan­cy

CFO Paul Clan­cy not­ed:

I would char­ac­ter­ize the bias to­wards prod­ucts, not to­wards plat­forms. Nev­er say nev­er, but this is rare dis­ease. Wil­son rep­re­sents a good ex­am­ple of our bias. Rare dis­ease, dev­as­tat­ing dis­eases, a po­ten­tial prod­uct to trans­form the dis­ease, and we’re build­ing up our abil­i­ty to do that in­side the com­pa­ny.

CEO Lud­wig Hantson sought to re­as­sure an­a­lysts that they were sat­is­fied that they could con­vert a large num­ber of pa­tients to the new drug, which some an­a­lysts be­lieve will have to come with a dis­count for pay­ers. Dur­ing the call with an­a­lysts, the CEO said:

We have a dif­fer­en­ti­at­ed pro­file with 1210. I don’t think any­body will dis­pute that. And, for sure, we’re go­ing to try to get the best la­bel. And we be­lieve that the da­ta that we have will be re­flect­ed in the clin­i­cal tri­al sec­tion. The way I look at it, we don’t need the su­pe­ri­or­i­ty claim to be suc­cess­ful for a fast con­ver­sion. We have a strong, dif­fer­en­ti­at­ed clin­i­cal pro­file, a ro­bust da­ta pack­age.

It’s not ide­al, but an­a­lysts like Ge­of­frey Porges saw rea­sons for ap­plause.

Alex­ion not­ed that ‘1210 was nu­mer­i­cal­ly su­pe­ri­or on both the pri­ma­ry and all key sec­ondary end­points. We are al­so en­cour­aged by the dis­clo­sure that ze­ro sub­jects treat­ed with ‘1210 ex­pe­ri­enced break­through he­mol­y­sis ver­sus 5 sub­jects treat­ed with Soliris. In terms of safe­ty, there were no dis­con­tin­u­a­tions due to ad­verse events, neu­tral­iz­ing an­ti­bod­ies, or cas­es of meningo­coc­cal in­fec­tion.

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.