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

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

LLS backs 5 new can­cer drug projects with up to $50M; Trodelvy con­tin­ues to im­press with more TNBC da­ta

The Leukemia and Lymphoma Society has tapped 5 new early-stage projects to back with up to $10 million each in fresh investments. The 5 biotechs are:

— Caribou, headed by Rachel Haurwitz and co-founded by Jennifer Doudna, is working on next-gen, off-the-shelf CAR-Ts to replace the patient-derived cells now in use.

— The LLS supported NexImmune’s IPO, helping fund its work on nanoparticles that can gin up an immune response directed at cancer cells. The biotech has 2 projects now in Phase I trials.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.