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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Anthony Fauci (AP Images)

A press of­fi­cer at An­tho­ny Fau­ci’s NI­AID was un­masked as a hard-right Covid troll. He just re­tired to­day

William B Crews had been a public affairs specialist at the NIH’s National Institute of Allergy and Infectious Diseases.

That ended today when he informed the agency of his decision to retire, after he was identified as the managing editor at RedState, a prominent Trump loyalist website.

Crews’ RedState duties are performed under the alias streiff. While enjoying the benefits of pseudonymity, he disparaged and worked against NIAID with an incendiary level of rhetoric in the midst of a pandemic.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Frank Zhang (AP Images)

Rocked by cus­toms in­ves­ti­ga­tion, Leg­end's CFO takes over as CEO Frank Zhang placed un­der house ar­rest

When Frank Zhang stepped down from GenScript — the contract research group he’s run for 18 years — to take up the CEO post at its CAR-T focused spinout Legend Biotech, he assured analysts that he was in for the long haul.

Just 49 days later, though, he’s been forced to hand back the title.

In a dramatic turn of events, Legend disclosed that Zhang is under house arrest in China as part of a customs investigation involving GenScript. While he remains the chairman, CFO Ying Huang has been tapped to double as interim CEO.

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UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Donald Trump, AP

Covid-19 roundup: Trump sug­gests Pfiz­er vac­cine could be first ap­proved; VBI Vac­cines inks de­vel­op­ment deal with Cana­da

President Donald Trump commented Monday morning that Pfizer’s Covid-19 vaccine candidate could be the first to win approval by regulators.

During an interview on a Fox News’ morning show, the president said Pfizer was doing “very well” when asked which candidate could be approved, according to a Reuters report. He added that J&J could follow up afterward, saying “they’ll probably be a little later.”

Is­raeli biotech rais­es $57M to go where cur­rent BRAF in­hibitors can't, with back­ing from No­var­tis, SR One

For the blockbuster potential of Novartis’ Tafinlar and Pfizer’s Braftovi, all the BRAF inhibitors on the market so far only target V600 mutations — which accounts for roughly 50% of patients.

Israeli biotech Novellus now has $57 million to develop a drug that they say can help the other 50% who have everything else.

The Series C will fund a Phase II trial for PLX-8394, a “paradox breaker” that could block RAF without activating MAPK signaling. In a Phase I trial, a patient with a BRAF fusion saw their tumor go away after taking the drug, allowing Novellus to hit the ground running.