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

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Fireside chat between Hal Barron and John Carroll, UKBIO19

It’s time we talked about bio­phar­ma — live in Lon­don next week

Zoom can only go so far. And I think at this stage, we’ve all tested the limits of staying in touch — virtually. So I’m particularly happy now that we’ve revved up the travel machine to point myself to London for the first time in several years.

Whatever events we have lined up, we’ve always built in plenty of opportunities for all of us to get together and talk. For London, live, I plan to be right out front, meeting with and chatting with the small crowd of biopharma people we are hosting on October 12 at Silicon Valley Bank’s London headquarters. And there’s a lengthy mixer at the end I’m most looking forward to, with several networking openings between sessions.

Take­da to pull key hy­poparathy­roidism drug from the mar­ket af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024, but the entire inventory will be available until depleted or expired, a company spokesperson said via email.

Pfizer and BioNTech's original Marvel comic book links evolving Covid vaccine science to Avengers' evolving villain-fighting tools.(Source: Pfizer LinkedIn post)

Pfiz­er, BioN­Tech part­ner with Mar­vel for Avengers and Covid-fight­ing com­ic book

Pfizer and BioNTech are collaborating with Marvel to celebrate “everyday” people getting Covid-19 vaccines in a custom comic book.

In the “Everyday Heroes” digital comic book, an evolving Ultron, one of the Avengers’ leading villains, is defeated by Captain America, Ironman and others. The plotline and history of Ultron is explained by a grandfather who is waiting with his family at a clinic for Covid-19 vaccinations.

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FDA+ roundup: Ad­comm date set for Cy­to­ki­net­ics heart drug; New gener­ic drug guid­ance to re­duce fa­cil­i­ty de­lays

The FDA on Wednesday set Dec. 13 as the day that its Cardiovascular and Renal Drugs Advisory Committee will review Cytokinetics’ potential heart drug, meaning regulators aren’t likely to meet the Nov. 30 PDUFA date that was previously set.

The drug, known as omecamtiv mecarbil, read out its first Phase III in November 2020, hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as the key to breaking into the market, failing to significantly differ in reducing cardiovascular death from placebo.

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Car­olyn Bertozzi (Illustration: Assistant editor Kathy Wong for Endpoints News)

Car­olyn Bertozzi, re­peat biotech founder and launch­er of a field, shares in chem­istry No­bel win

Carolyn Bertozzi, predicted by some to become a Nobel laureate, clinched one of the world’s top awards in the wee hours of Wednesday, winning the Nobel Prize in Chemistry alongside a repeat winner and a Copenhagen researcher.

The Stanford professor, Morten Meldal of University of Copenhagen and 2001-awardee K. Barry Sharpless of Scripps shared the prize equally. The Nobel is sometimes split in quarters and/or halves.

Eli Lil­ly and Te­va pre­pare for court bat­tle over mi­graine med ri­val­ry

It looks like Eli Lilly and Teva Pharmaceuticals are going to trial.

A federal appeals court on Monday refused to invalidate three of Teva’s patents for its migraine treatment Ajovy, while also declining to issue a summary judgment in favor of either company, which would effectively end the case without a full trial.

Teva filed suit against Lilly back in 2018, alleging that the company infringed upon nine patents with its rival migraine drug Emgality. The rival drugs were both approved in September 2018 for the preventative treatment of migraine, and are designed to block calcitonin gene-related peptide (CGRP), a protein associated with the onset of migraine pain.

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Rep. Vern Buchanan (R-FL) (Bill Clark/CQ Roll Call via AP Images)

Af­ter cov­er­age re­stric­tions for Alzheimer's drugs, bi­par­ti­san House bill would force CMS to re­view drugs in­di­vid­u­al­ly

When Biogen’s controversial Alzheimer’s drug Aduhelm was hit with a national decision from CMS that restricted coverage to only randomized trials, practically guaranteeing a commercial flop in the near term, questions surfaced over why CMS also included all amyloid-targeted monoclonal antibodies for Alzheimer’s disease.

With Eisai and Biogen’s second Alzheimer’s drug, lecanemab, now showing it can slow the rate of cognitive decline versus placebo, lining up for a likely full approval next spring, the question now turns to whether that data, which is being presented at the Clinical Trials on Alzheimer’s Congress in San Francisco in late November, will be enough for CMS when it asks, “Does the anti-amyloid mAb meaningfully improve health outcomes (i.e., slow the decline of cognition and function) for patients in broad community practice?”

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