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 Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Lian­Bio an­nounces terms for IPO next week; NIH and White House of­fi­cials dis­cuss hy­po­thet­i­cal bio-med re­search agency

LianBio, a biotech that has operations in both the US and China, announced the terms yesterday for its initial public offering.

The biotech plans to raise $325 million by offering 20.3 million shares priced between $15 and $17.

At the midpoint of the proposed range, LianBio could command a fully diluted market value of $1.8 billion, based on a number of expected outstanding shares.

The two year old biotech has focused on in-licensing and commercialization of therapeutics in China, Hong Kong, Taiwan, Macau and other Asian markets. The company currently has nine programs across five therapeutic areas, including oncology, cardiovascular, and inflammatory diseases.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Bris­tol My­ers pledges to sell its Ac­celeron shares as ac­tivist in­vestors cir­cle Mer­ck­'s $11.5B buy­out — re­port

Just as Avoro Capital’s campaign to derail Merck’s proposed $11.5 billion buyout of Acceleron gains steam, Bristol Myers Squibb is leaning in with some hefty counterweight.

The pharma giant is planning to tender its Acceleron shares, Bloomberg reported, which add up to a sizable 11.5% stake. Based on the offer price, the sale would net Bristol Myers around $1.3 billion.

To complete its deal, Merck needs a majority of shareholders to agree to sell their shares.

Boost­er bo­nan­za: FDA en­dors­es 'mix-and-match' scheme, and Mod­er­na and J&J too

The FDA late Wednesday signed off on authorizing the use of heterologous — or what FDA calls a “mix and match” of a primary vaccine series and different booster doses — for all currently available Covid-19 vaccines, in addition to separately authorizing Moderna and J&J boosters.

On the mix-and-match approach, which FDA officials insisted isn’t too confusing in a press conference, the agency offered the example of an 18-year-old who received the J&J shot at least two months ago and may now receive a single booster of the J&J, a half dose of the Moderna, or the Pfizer-BioNTech booster.