On a roll, Dicer­na notch­es $100M up­front as Eli Lil­ly eyes RNAi ap­pli­ca­tions in neu­rode­gen­er­a­tion, pain

Just days af­ter sell­ing co-de­vel­op­ment rights to two of its pre­clin­i­cal RNAi ther­a­pies to Alex­ion for a cool $22 mil­lion, Dicer­na has scored an­oth­er Big Phar­ma col­lab­o­ra­tion — this time mov­ing fur­ther up the re­search stream but scor­ing a much big­ger up­front.

Eli Lil­ly is pay­ing $100 mil­lion for dibs on some new ap­pli­ca­tions of Dicer­na’s GalXC plat­form — neu­rode­gen­er­a­tion and pain — as well as the car­diometa­bol­ic work that’s be­come sta­ple in the RNAi field. Add that to Lil­ly’s $100 mil­lion eq­ui­ty in­vest­ment ($18.47 per share, a 42% pre­mi­um from Dicer­na’s $DR­NA clos­ing price on Fri­day) and the $350 mil­lion in mile­stones for each of the 10 tar­gets that the part­ners ex­pect to pur­sue, and Dicer­na is look­ing at a pact po­ten­tial­ly worth $3.7 bil­lion.

Dan Skovron­sky

“We are ex­cit­ed to col­lab­o­rate with Dicer­na and uti­lize their RNAi ex­per­tise to study tar­gets that up un­til now have proven to be very tech­ni­cal­ly chal­leng­ing,” said Lil­ly CSO Dan Skovron­sky in a state­ment. “RNAi has the po­ten­tial to treat an ar­ray of dis­eases that are of strate­gic im­por­tance to Lil­ly.”

For each of the GalXC mol­e­cule iden­ti­fied, Dicer­na will be re­spon­si­ble for the pre­clin­i­cal de­vel­op­ment un­til Lil­ly takes over to con­duct clin­i­cal tri­als and po­ten­tial com­mer­cial­iza­tion, Dicer­na CEO Dou­glas Fam­brough told me.

The re­search and li­cens­ing pact rep­re­sents a big leap since Dicer­na got “ear­ly val­i­da­tion” from Boehringer In­gel­heim last No­vem­ber, in a deal that came with $10 mil­lion up­front and $191 mil­lion in po­ten­tial pay­ments.

Dou­glas Fam­brough

Build­ing on the premise that RNAi can si­lence dis­ease-caus­ing genes by de­stroy­ing their mes­sen­ger RNA be­fore they can give in­struc­tions to make dis­ease-caus­ing pro­teins, the GalXC plat­form is de­signed to pro­duce mol­e­cules that op­er­ate in “the most spe­cif­ic and po­tent fash­ion.”

While the cur­rent par­a­digm — as well as its ef­forts so far — has fo­cused on liv­er cells, Dicer­na be­lieves it is well equipped to ex­plore oth­er forms of RNAi de­liv­ery.

“It is al­ready es­tab­lished that oligonu­cleotide ther­a­peu­tics can be uti­lized suc­cess­ful­ly in neu­ro­log­i­cal con­di­tions, most no­tably the Spin­raza treat­ment for spinal mus­cle at­ro­phy,” Fam­brough wrote in an email. “We an­tic­i­pate col­lab­o­rat­ing with Lil­ly on the use of dif­fer­ent tar­get­ing lig­ands and chemistries to op­ti­mize de­liv­ery to neu­ro­log­i­cal cells. Due to chal­lenges cross­ing the blood-brain bar­ri­er, we an­tic­i­pate that ini­tial­ly the mol­e­cules will be in­ject­ed in­trathe­cal­ly.”

Once caught up in a bit­ter le­gal bat­tle over trade se­crets with ri­vals at the pi­o­neer­ing RNAi biotech Al­ny­lam $AL­NY, Cam­bridge, MA-based Dicer­na has since set­tled the law­suits and turned its at­ten­tion to deal­mak­ing as its lead as­set en­tered the clin­ic.

“Dicer­na’s busi­ness strat­e­gy is to re­tain full or sub­stan­tial own­er­ship stake in its key rare dis­ease pro­grams, while pur­su­ing part­ner­ships for more com­plex dis­eases with mul­ti­ple gene dys­func­tions and larg­er pa­tient pop­u­la­tions,” he said. “No­tably, and con­sis­tent with our strat­e­gy, Dicer­na re­tains rights to de­vel­op prod­ucts in­de­pen­dent­ly for or­phan neu­rode­gen­er­a­tion and pain in­di­ca­tions.”

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.

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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As­traZeneca pub­lish­es Covid-19 vac­cine PhI­II pro­to­cols in lock­step with Mod­er­na and Pfiz­er. How are they dif­fer­ent?

Following in the steps of Moderna and Pfizer, the other two American drugmakers currently in Phase III trials for their Covid-19 vaccines, AstraZeneca posted its own study protocols over the weekend. The move is the latest in a series of rare peeks behind the curtain, as such blueprints are typically shared once such trials are completed.

“Given the unprecedented global impact of the Coronavirus pandemic and the need for public information, AstraZeneca has published the detailed protocol and design of our AZD1222 clinical trial. As with most clinical development, protocols are not typically shared publicly due to the importance of maintaining confidentiality and integrity of trials. AstraZeneca continues to work with industry peers to ensure a consistent approach to sharing timely clinical trial information,” the company said in a statement.

Sebastian Nijman (file photo)

Roche looks to ge­net­ic mod­i­fiers for new drug tar­gets, team­ing up with Dutch biotech in $375M deal

Roche is gambling on a new way of discovering drug targets and, ultimately, promising to infuse more than $375 million into a small biotech if all goes well.

A spinout of the Netherlands Cancer Institute and Oxford University, Scenic Biotech set out to pioneer a field that’s gaining some traction among top VCs in the US: to harness the natural protecting powers of genetic modifiers — specific genes that suppress a disease phenotype.

Donald Trump and White House chief of staff Mark Meadows, before boarding Marine One (Getty Images)

Pric­ing deal col­laps­es over Big Phar­ma's re­fusal to is­sue $100 'cash card­s' be­fore the elec­tion — re­port

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.

#ES­MO20: As­traZeneca aims to spur PRO­found shift in prostate can­cer treat­ment with Lyn­parza OS da­ta

AstraZeneca has unveiled the final, mature overall survival data that cemented Lynparza’s first approval in prostate cancer approval — touting its lead against rivals with the only PARP inhibitor to have demonstrated such benefit.

But getting the Merck-partnered drug to the right patients remains a challenge, something the companies are hoping to change with the new data cut.

The OS numbers on the subgroup with BRCA1/2 or ATM-mutated metastatic castration-resistant prostate cancer are similar to the first look on offer when the FDA expanded the label in May: Lynparza reduced the risk of death by 31% versus Xtandi and Zytiga. Patients on Lynparza lived a median of 19.1 months, compared to 14.7 months for the anti-androgen therapies (p = 0.0175).

Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

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Greg Friberg (File photo)

#ES­MO20: Am­gen team nails down sol­id ear­ly ev­i­dence of AMG 510’s po­ten­tial for NSCLC, un­lock­ing the door to a wave of KRAS pro­grams

The first time I sat down with Amgen’s Greg Friberg to talk about the pharma giant’s KRAS G12C program for sotorasib (AMG 510) at ASCO a little more than a year ago, there was high excitement about the first glimpse of efficacy from their Phase I study, with 5 of 10 evaluable non-small cell lung cancer patients demonstrating a response to the drug.

After decades of failure targeting KRAS, sotorasib offered the first positive look at a new approach that promised to open a door to a whole new approach by targeting a particular mutation to a big target that had remained “undruggable” for decades.

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