Unit­ed Ther­a­peu­tics bags a new OK on Ty­va­so, with block­buster ex­pec­ta­tions; Gilead goes back to Ab­Celler­a's well with a set of fresh tar­gets

As ex­pect­ed, Unit­ed Ther­a­peu­tics {UTHR} snagged an OK from the FDA to sell the in­haled ver­sion of its block­buster drug Ty­va­so for a new in­di­ca­tion: pul­monary hy­per­ten­sion as­so­ci­at­ed with in­ter­sti­tial lung dis­ease. And as ex­pect­ed, they im­me­di­ate­ly launched their new mar­ket­ing ef­fort af­ter care­ful­ly lay­ing the ground­work.

Unit­ed COO Michael Benkowitz not­ed:

We’ve al­ready ex­pand­ed our field-based teams by 40% to ed­u­cate the ILD com­mu­ni­ty on the ben­e­fits of Ty­va­so and how to prop­er­ly di­ag­nose PH-ILD. We ex­pect rapid up­take of Ty­va­so in this in­di­ca­tion and ex­pect to dou­ble the num­ber of pa­tients on Ty­va­so ther­a­py by the end of 2022, bar­ring any COVID-re­lat­ed de­lays.

The news couldn’t have come as a big sur­prise to Wall Street, but Cowen an­a­lyst Joseph Thome breathed a sigh of re­lief on the OK, ob­serv­ing grow­ing fears that the FDA’s get-tough pen­chant could have spurred a sur­prise re­jec­tion. Now he’s count­ing the ex­tra cash ex­pect­ed to come in.

We mod­el peak rev­enues of $1.6B in 2025 for UTHR’s in­haled fran­chise, which may prove to be con­ser­v­a­tive if the PH-ILD pop­u­la­tion is in fact un­der­diag­nosed.

— John Car­roll

Gilead heads back to Ab­Cellera with a fresh set of dis­cov­ery tar­gets

Carl Hansen

Less than 2 years af­ter sign­ing their first pact, Gilead has head­ed back up­stream in the R&D process to ink a new dis­cov­ery deal with Van­cou­ver-based Ab­Cellera.

This time Ab­Cellera, which part­nered with Eli Lil­ly on the EUA-ap­proved Covid-19 drug bam­lanivimab, will be throw­ing some new tech­nolo­gies in­to the arrange­ment, which cov­ers an 8-tar­get slate for Gilead. The up­front and mile­stones are stay­ing un­der wraps, but these deals are typ­i­cal­ly heav­i­ly back-end­ed.

Ab­Cellera, one of the mar­ket dar­lings in the pan­dem­ic, was quick to tout its ex­pand­ed ca­pa­bil­i­ties. CEO Carl Hansen not­ed:

We are par­tic­u­lar­ly pleased to see the val­ue cre­at­ed for our part­ners by quick­ly in­te­grat­ing the Tri­an­ni Mouse and Or­thoMab plat­forms, which we ac­quired in sec­ond half of 2020.

— John Car­roll

Agios piv­ots to mi­tapi­vat and stock re­pur­chase plan as Servi­er hands over $1.8B

Jack­ie Fouse

Agios had no soon­er re­ceived its $1.8 bil­lion cash up­front for the sale of its on­col­o­gy drugs to Servi­er than the biotech im­me­di­ate­ly start­ed buy­ing up its own shares.

Agios CEO Jack­ie Fouse wast­ed no time in buy­ing back 7.1 mil­lion shares of its stock from Bris­tol My­ers Squibb for $344.5 mil­lion. An­oth­er $844.5 mil­lion of stock is yet to be ac­quired, and Agios ex­pects it will gain the bulk of those shares ahead of the end of this year.

Agios now will com­plete it piv­ot to mi­tapi­vat, with fil­ings be­ing read­ied for in adults with PK de­fi­cien­cy in both the US and Eu­rope.

Al­so in the time­line: “(I)ni­ti­at­ing two Phase 3 stud­ies of mi­tapi­vat in trans­fu­sion de­pen­dent and non-trans­fu­sion de­pen­dent tha­lassemia; ini­ti­at­ing a Phase 2/3 study of mi­tapi­vat in sick­le cell dis­ease; pre­sent­ing the first da­ta from the healthy vol­un­teer study of AG-946, the next gen­er­a­tion PKR ac­ti­va­tor; and pri­or­i­tiz­ing new in­di­ca­tions for PKR and pyru­vate ki­nase M2 (PKM2) ac­ti­va­tor clin­i­cal de­vel­op­ment.”

— John Car­roll

Eloxx ac­quires Zikani to team up in non­sense mu­ta­tions

Eloxx Phar­ma­ceu­ti­cals is pro­ceed­ing with a new buy­out.

Sum­it Ag­gar­w­al

The Mass­a­chu­setts com­pa­ny is ac­quir­ing Zikani Ther­a­peu­tics in an all-stock trans­ac­tion, it an­nounced Thurs­day morn­ing. As part of the move, Zikani CEO Sum­it Ag­gar­w­al will take the reins of Eloxx, while Zikani CSO Vi­jay Mod­ur be­comes Eloxx’s new R&D chief.

Eloxx’s strat­e­gy cen­ters around non­sense mu­ta­tions, which they say cause rough­ly 10% to 12% of rare in­her­it­ed dis­eases. Eloxx and its new part­ner plan to push for­ward with ELX-02, cur­rent­ly in Phase II tri­als in cys­tic fi­bro­sis for pa­tients af­fect­ed by non­sense mu­ta­tions in the CFTR gene.

The com­pa­ny al­so hopes to file an IND next year for what they say could be the first oral ther­a­py for pro­tein restora­tion for pa­tients with non­sense mu­ta­tions in re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa and junc­tion­al epi­der­mol­y­sis bul­losa. — Max Gel­man

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.”

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.

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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A cell ther­a­py biotech finds a de­liv­ery part­ner; FDA re­moves clin­i­cal hold on Ocu­gen's Covid can­di­date

Umoja Biopharma will pair its so-called VivoVec particles with Lupagen’s Side CAR-T delivery system to target certain cancers, the companies said Monday.

As part of the deal, Umoja’s cell therapies could be delivered via an additional route thanks to Lupagen’s extracorporeal in vivo tech. The Dallas gene therapy company’s tech is expected to provide efficiency and “highly controlled viral vector targeting of T cells,” the companies added.

Phillip Gomez, SIGA CEO

UP­DAT­ED: On the back of SIGA Tech­nolo­gies' win with the FDA, the mon­key­pox virus sees the com­pa­ny spring­ing to fur­ther ac­tion

As the cases of monkeypox now sit at well over 100 worldwide and have spread to multiple continents, the orders for any type of vaccine against monkeypox are seeing nations and medical bodies looking to get their hands on anything and everything. And now SIGA Technologies seems to be getting in on the action.

According to Euronews, SIGA Technologies, a pharmaceutical company that is focused on providing medical countermeasures to biological and chemical attacks, is now in talks with several European authorities looking to stockpile its antiviral that can counter monkeypox. The drug known as tecovirimat or Tpoxx was approved by the FDA in 2018 as a vaccine for smallpox but was approved by the European Medicines Agency to also act against monkeypox, cowpox and complications from immunization with vaccinia.