Es­ca­lat­ing R&D woes spur fresh M&A chat­ter for cash-rich Gilead

Gilead CSO Nor­bert Bischof­berg­er

Four years af­ter Gilead ac­quired mo­melo­tinib in its $510 mil­lion buy­out of YM Bio­Sciences, the big — and what ap­pears to be in­creas­ing­ly un­lucky — biotech was forced to re­port that the drug per­formed poor­ly in its two Phase III myelofi­bro­sis tri­als. And the fourth straight pipeline flop quick­ly spurred fresh spec­u­la­tion that Gilead will soon have to tap in­to its big cash re­serves for a ma­jor ac­qui­si­tion.

The JAK in­hibitor scored on a non-in­fe­ri­or­i­ty show­down with Jakafi (In­cyte’s rux­oli­tinib) in spleen re­duc­tion, but blew the key sec­ondary end­point of non-in­fe­ri­or­i­ty for the to­tal symp­tom score that was used to mea­sure the drugs. And it failed a sep­a­rate Phase III su­pe­ri­or­i­ty com­par­i­son against best avail­able treat­ment at week 24.

It all adds up to a fresh pipeline mess at Gilead, which saw its shares $GILD de­cline 1.25% Thurs­day morn­ing, shav­ing off more than a bil­lion dol­lars of its $100 bil­lion mar­ket cap. In­cyte {IN­CY}, mean­while, saw its shares shoot up 7% on the news, as spec­u­la­tion mount­ed that Gilead may now be pressed hard to make a bid for the com­pa­ny.

By the num­bers: mo­melo­tinib: 6.7%; BAT: 5.8%; 95 per­cent CI: -8.9% to +10.2%; p=0.90. And be­cause it didn’t suc­ceed on su­pe­ri­or­i­ty on the pri­ma­ry score, in­ves­ti­ga­tors didn’t run the num­bers on sec­ondary end­points.

That’s not what the com­pa­ny want­ed or need­ed to see. But af­ter high­light­ing some pos­i­tive trends from the two late-stage stud­ies, Gilead plans to see if reg­u­la­tors are feel­ing gen­er­ous about its prospects.

“The re­sults from both the SIM­PLI­FY-1 and SIM­PLI­FY-2 stud­ies in­di­cate that mo­melo­tinib pro­vides some treat­ment ben­e­fit, in­clud­ing ben­e­fit on ane­mia-re­lat­ed end­points,” said Nor­bert Bischof­berg­er, PhD, Ex­ec­u­tive Vice Pres­i­dent of Re­search and De­vel­op­ment and Chief Sci­en­tif­ic Of­fi­cer. “We plan to dis­cuss these re­sults with reg­u­la­to­ry au­thor­i­ties to de­ter­mine the next steps.”

The set­back on mo­melo­tinib comes near the end of what has turned in­to a grim year for Gilead. A re­cent run­down of its work on the NASH drug GS-4997 sparked con­sid­er­able skep­ti­cism from an­a­lysts, who want to see much more com­pelling num­bers be­fore they buy in.

Gilead re­cent­ly wrote off sim­tuzum­ab, along with the late-stage drug GS-5745 for ul­cer­a­tive col­i­tis and Crohn’s. And it’s top car­dio prospect — ele­clazine (GS-6615) — failed a late-stage study as well, sig­nif­i­cant­ly re­duc­ing its chances of be­com­ing the big new drug that Gilead needs as hep C wanes.

As the R&D hole keeps get­ting deep­er, some won­der if Gilead will be forced back to the deal ta­ble to buy some­thing big. Its hep C fran­chise may be wan­ing, but it cre­at­ed a huge wind­fall in cash re­serves. And Leerink’s Ge­of­frey Porges be­lieves that this lat­est black cloud comes with a sil­ver lin­ing, adding In­cyte to the list of big takeover tar­gets. On Thurs­day morn­ing he not­ed:

GILD in­di­cat­ed that they plan to dis­cuss these re­sults with reg­u­la­to­ry au­thor­i­ties to de­ter­mine the next steps, but it seems like­ly that mo­melo­tinib, which has been in de­vel­op­ment since 2009, will be writ­ten off, end­ing the po­ten­tial of yet an­oth­er small “bolt on” ac­qui­si­tion. One con­se­quence of this dis­ap­point­ment, how­ev­er, is that it does re­store IN­CY to the list of fea­si­ble can­di­dates for ac­qui­si­tion by Gilead, since they would no longer face the oblig­a­tion to di­vest one of the over­lap­ping JAK pro­grams.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

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Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Af­ter safe­ty re­view, EMA mir­rors FDA with up­dat­ed rec­om­men­da­tions for JAK in­hibitors

The EMA released updated recommendations today for the use of JAK inhibitors (JAKi) after reviewing data from several clinical trials that showed increased incidents of issues in certain patients who have rheumatoid arthritis and other risk factors.

The EMA noted malignancy, major adverse cardiovascular events (MACE), serious infections, venous thromboembolism (VTE) and mortality in some patients.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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No longer ‘dead or just hi­ber­nat­ing,’ drug­mak­ers re­turn to heart med­i­cines

In 2015, now-FDA Commissioner Robert Califf joined industry, academic and regulatory representatives in Washington to discuss why more drugs weren’t in development for cardiovascular diseases, the leading US cause of death and once a mainstay of pharmaceutical industry blockbusters.

The group pointed to many reasons. Clinical trials could take years and testing was expensive. Wide availability of generic drugs made the commercial prospects uncertain. Their paper title summed up the mood: “Cardiovascular Drug Development: Is it Dead or Just Hibernating?”

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