Fol­low­ing job cuts and R&D re­struc­tur­ing, mi­cro­cap mi­croR­NA play­er mi­Ra­gen brings in new CEO to right the ship

Four years af­ter rid­ing the shell of a strug­gling mi­cro­cap biotech to Nas­daq with $40 mil­lion in cash, mi­croR­NA play­er mi­Ra­gen Ther­a­peu­tics is fac­ing a reck­on­ing of its own.

Af­ter the mar­ket close Thurs­day, mi­Ra­gen dis­closed a flur­ry of changes at the com­pa­ny: CEO William Mar­shall has re­signed, leav­ing COO Lee Rauch in charge as the new chief to over­see a “re­view of strate­gic al­ter­na­tives” — of­ten a sign that the board is ea­ger for a sale, al­though in-li­cens­ing and oth­er deals are al­so on the ta­ble.

Lee Rauch

Rauch, a biotech vet who joined just three months ago, is tak­ing over a year in­to a cost re­struc­tur­ing plan that Mar­shall be­gan last Au­gust, which in­volved slash­ing the work­force by half and elim­i­nat­ing 44 po­si­tions, main­ly in R&D and ad­min­is­tra­tive teams. Dis­cov­ery re­search was to go on the back­burn­er while mi­Ra­gen push­es the four core pro­grams it al­ready had.

In con­junc­tion with the shake­up, mi­Ra­gen no­ti­fied in­vestors that it’s crown­ing the pre­clin­i­cal pro­gram MRG-229 its lead com­pound, pri­or­i­tiz­ing it over the three clin­i­cal-stage drugs.

The idea is to treat id­io­path­ic pul­monary fi­bro­sis by re­vers­ing the ab­nor­mal­ly low lev­els of mi­croR­NA-29 with the con­ju­gat­ed mim­ic.

“We be­lieve that fi­bro­sis is an ide­al dis­ease process for the use of mi­croR­NA ther­a­peu­tics,” Rauch said in a state­ment. “This in­cludes MRG-229, a re­place­ment for miR-29, which tar­gets the im­por­tant genes and path­ways that are cen­tral to the de­vel­op­ment of ab­nor­mal ex­tra­cel­lu­lar ma­trix de­po­si­tion re­sult­ing in fi­bro­sis.”

William Mar­shall

Rem­larsen, which works in a sim­i­lar way, is in Phase II for cu­ta­neous fi­bro­sis and be­ing ex­plored for oc­u­lar fi­bro­sis. The dif­fer­ence is that it is de­signed for lo­cal ad­min­is­tra­tion, while MRG-229 had po­ten­tial ap­pli­ca­tions in pul­monary, re­nal and he­pati­tis fi­bro­sis with its sys­temic mech­a­nism.

Al­so de­pri­or­i­tized for now are cobo­marsen, the mi­croR­NA-155 in­hibitor po­si­tioned for cu­ta­neous T-cell lym­phoma and adult T-cell leukemia/lym­phoma, as well as MRG-110, an in­hibitor of mi­croR­NA-92 de­signed to tar­get heart fail­ure, wound heal­ing and oth­er is­chemic dis­ease.

With $30.6 mil­lion of cash and cash equiv­a­lents plus $8.1 mil­lion of out­stand­ing debt by the end of H1, mi­Ra­gen had re­cent­ly out­lined a run­way to Q3 2021.

Board chair­man Jeff Hat­field said mi­Ra­gen is now bank­ing on Rauch’s “ex­ten­sive ex­pe­ri­ence in com­pa­ny build­ing, cor­po­rate strat­e­gy and busi­ness de­vel­op­ment” to set a course for the fu­ture.

Some in­vestors aren’t stick­ing around. Shares $MGEN fell 5.32% pre-mar­ket, al­though that means lit­tle when the stock is just trad­ing at $0.89.

Un­lock­ing ESG strate­gies for growth with Gilead Sci­ences

RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

What con­tro­ver­sy? Eli Lil­ly plots Alzheimer's BLA fil­ing lat­er this year as FDA taps more an­ti-amy­loid drugs as break­throughs

The FDA is keeping the good news coming for Alzheimer’s drug developers. And Eli Lilly is taking them up on it.

Amid continued controversy around whether Biogen’s new flagship drug, Aduhelm, should have been approved at all — and swelling, heated debates surrounding its $56,000 price tag — the agency had no issue handing them and their Japanese partner Eisai a breakthrough therapy designation for a second anti-amyloid beta antibody, lecanemab, late Wednesday.

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Hervé Hoppenot, Incyte CEO (Jeff Rumans)

ODAC echoes FDA con­cern over In­cyte PD-1, as Paz­dur sig­nals broad­er shift for ac­cel­er­at­ed ap­proval

After the FDA lambasted their PD-1 ahead of an adcomm earlier this week, Incyte ran into new trouble Thursday as ODAC panelists voted against an accelerated OK by a wide margin.

Members of the Oncologic Drugs Advisory Committee recommended with a 13-4 vote to defer a regulatory decision on Incyte’s retifanlimab until after more data can be collected from a placebo-controlled trial. The PD-1 therapy is due for a PDUFA date in late July after receiving priority review earlier this year.

Karen Flynn, Catalent

Q&A: When the pan­dem­ic struck, Catal­en­t's CCO had just joined the team

Karen Flynn came aboard Catalent’s team just in time.

The company was going through a surge of changes, and she had been brought over from her role as CCO of West Pharmaceutical Services to serve in the same capacity for the New Jersey-based CDMO. Then a few months later, the pandemic was in full-force.

Since then, Catalent’s been in hyper-expansion mode. In early May, it acquired Promethera’s Hepatic Cell Therapy Support SA subsidiary and its 32,40-square-foot facility in Gosselies, Belgium. Prior to that, the company acquired Belgian CDMO Delphi Genetics, wrapped up the expansion of an already-existing site in Madison, WI and added an ultra-low temperature freezer partner in Sterling. As Emergent has botched millions of doses of AstraZeneca’s vaccine, the company has swooped in to move that production to its Maryland plant as well.

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New FDA doc­u­ments show in­ter­nal dis­sent on Aduhelm ap­proval

In a lengthy review document and a pair of memos from top officials, the FDA released on Tuesday night its most detailed argument yet for approving Biogen’s intensely controversial Alzheimer’s drug aducanumab.

The documents amount to an agency attempt to quench the firestorm their decision kindled, as outside advisors members resigned and experts warned that an unproven drug now could stretch Medicare’s budget to a breaking point. Ultimately, the documents show how CDER director Patrizia Cavazzoni and Office of New Drugs director Peter Stein both concurred with FDA neuroscience head Billy Dunn on the accelerated approval while the staff at FDA’s Office of Biostatistics did not think an approval was warranted.

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Richard Pazdur (vis AACR)

FDA en­cour­ages in­clud­ing in­cur­able can­cer pa­tients in tri­als, re­gard­less of pri­or ther­a­pies

The FDA on Thursday called to include those with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in appropriate clinical trials, regardless of whether they have received existing alternative treatments.

Historically, many cancer clinical trials have required that participating patients previously received multiple therapies, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence.

On heels of Aduhelm ap­proval, Bris­tol My­ers jumps back in­to Alzheimer's race

Bristol Myers Squibb last put major resources behind an Alzheimer’s drug nearly a decade ago, when their own attempt at targeting amyloid flamed out in mid-stage studies. They invented another molecule, a Tau-targeted antibody, but jettisoned it to Biogen in 2017 as they dropped out of neuroscience altogether.

But on Thursday, the New York pharma announced they were getting back in the game. Bristol Myers exercised an $80 million option to bring a tau-targeted antibody from Prothena into a Phase I study. The opt-in, which Bristol Myers triggered ahead of analyst expectations, opens the door for another $1.7 billion in milestones down the road.

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James Peyer, Cambrian

Can a cell ther­a­py treat mus­cu­lar dy­s­tro­phy? A Ger­man bil­lion­aire's an­ti-ag­ing start­up is try­ing to find out

Gene therapy companies have faced huge hurdles trying to deliver healthy genes into muscular dystrophy patients’ muscle cells, so here’s an idea: Why don’t we just replace the muscle cells themselves?

Over the last two years, Vita Therapeutics has been exploring that possibility, building on early stem cell work from Johns Hopkins professor Peter Andersen. And on Tuesday they announced a $32 million Series A to begin to move their first therapy into the clinic, where they hope it will help rebuild muscle in patients with a type of dystrophy that afflicts the arms and legs.

Alexis Borisy (file photo)

EQRx and Ex­sci­en­tia, a pair of self-styled dis­rup­tors, team up to over­turn the drug pric­ing ap­ple cart

The biotech industry has seen no shortage of innovation in recent years, but in one area — drug pricing — the field has been anything but innovative. Now, two brash startups taking different roads to upset the drug pricing model will partner up to create a sort of “super-disruptor.”

EQRx and UK-based AI specialist Exscientia will team up on a discovery-through-commercialization collaboration the partners hope will work better than the sum of its parts to bring cheaper medicines to patients faster, the companies said Thursday.