Alex­ion doles out $22M up­front for two pre­clin­i­cal RNAi drugs from Dicer­na

For­mer biotech ace Alex­ion Phar­ma­ceu­ti­cals is still shop­ping to fat­ten its pipeline as it con­tin­ues to shake off a sales prac­tices scan­dal linked to its ex­pen­sive rare dis­ease drug Soliris that cul­mi­nat­ed in an ex­ec­u­tive ex­o­dus and top-to-bot­tom re­or­ga­ni­za­tion. On Wednes­day, the Boston, MA-based com­pa­ny said it would fork out $22 mil­lion up­front to co-de­vel­op two pre­clin­i­cal RNAi ther­a­pies owned by Dicer­na Phar­ma­ceu­ti­cals $DR­NA.

The agree­ment fol­lows a Sep­tem­ber deal to buy pri­vate­ly held Syn­tim­mune for up to $1.2 bil­lion, and the ac­qui­si­tion of Swe­den’s Wil­son Ther­a­peu­tics for $855 mil­lion in April.

RNAi ther­a­pies of­fer the po­ten­tial to over­come key lim­i­ta­tions of tra­di­tion­al ap­proach­es to treat­ment, and has long in­ter­est­ed drug de­vel­op­ers, but has fre­quent­ly been met with clin­i­cal fail­ure. RNA in­ter­fer­ence (RNAi) is a process in which cer­tain dou­ble-strand­ed RNA mol­e­cules in­hib­it the ex­pres­sion of dis­ease-caus­ing genes by de­stroy­ing the mes­sen­ger RNAs (mR­NAs) of those genes. Rather than tar­get­ing and bind­ing to pro­teins to in­hib­it their ac­tiv­i­ty like mon­o­clon­al an­ti­bod­ies, RNAi ex­erts its ef­fects one step ear­li­er in the gene si­lenc­ing process by tar­get­ing the mR­NA, the in­struc­tion set that di­rects the build­ing of dis­ease-caus­ing pro­teins. Ear­li­er this year, the FDA ap­proved the first-ever RNAi drug, On­pat­tro, for the treat­ment of the hered­i­tary transthyretin-me­di­at­ed (hAT­TR) amy­loi­do­sis, which is a rare, pro­gres­sive life-threat­en­ing dis­ease.

Dou­glas Fam­brough

Dicer­na is fo­cus­ing on liv­er-tar­get­ed dis­eases that have not been amenable to con­ven­tion­al ther­a­pies, and says its GalXC RNAi plat­form has fu­eled a pipeline of ther­a­pies de­signed to con­quer chal­lenges posed by pri­or gen­er­a­tions of RNAi ther­a­peu­tics — the tech­nol­o­gy is de­signed to si­lence the ex­pres­sion of dis­ease-dri­ving genes in a way that is “high­ly spe­cif­ic, gen­er­al­ly well tol­er­at­ed, and al­lows for con­ve­nient, in­fre­quent sub­cu­ta­neous ad­min­is­tra­tion,” ac­cord­ing to CEO Dou­glas Fam­brough.

“These ther­a­pies mi­grate to the liv­er where com­ple­ment pro­teins are pro­duced and de­grade the mes­sen­ger RNA be­fore the com­ple­ment pro­tein can be trans­lat­ed. As such, these treat­ments would op­er­ate more up­stream than Alex­ion’s cur­rent com­ple­ment an­ti­bod­ies that block the com­ple­ment pro­teins af­ter they have been cre­at­ed, and the mech­a­nism should al­so pro­vide a longer du­ra­tion of ef­fect than an­ti­bod­ies,” not­ed Leerink’s Ge­of­frey Porges in a note.

As part of the deal, Dicer­na is el­i­gi­ble to re­ceive po­ten­tial de­vel­op­ment and reg­u­la­to­ry mile­stone pay­ments of up to $105 mil­lion per tar­get, in ad­di­tion to ag­gre­gate sales mile­stones of up to $160 mil­lion and roy­al­ties on fu­ture prod­uct sales. In ad­di­tion, Alex­ion is mak­ing a con­cur­rent $15 mil­lion eq­ui­ty in­vest­ment in Dicer­na at a price of 17.95 per share, a pre­mi­um of near­ly 47% to its Tues­day close. Alex­ion, which al­so re­port­ed high­er-than-ex­pect­ed Q3 earn­ings and rev­enue that just missed an­a­lyst es­ti­mates on Wednes­day, has the right to ex­er­cise op­tions on two ad­di­tion­al GalXC RNAi mol­e­cules from Dicer­na un­der the agree­ment.

Ri­val RNAi drug de­vel­op­er — and mak­er of On­pat­tro — Al­ny­lam Phar­ma­ceu­ti­cals $AL­NY an­nounced this April that Dicer­na had agreed to fork out $25 mil­lion in cash and stock to set­tle a law­suit filed in 2015 claim­ing it stole in­tel­lec­tu­al prop­er­ty re­lat­ed to Al­ny­lam’s gene-si­lenc­ing tech­nol­o­gy to de­vel­op ther­a­pies, open­ing the door for Dicer­na to forge new al­liances.

Oth­er play­ers in the RNAi field in­clude Ar­row­head Phar­ma­ceu­ti­cals $AR­WR, RXi Phar­ma­ceu­ti­cals $RXII, Si­lence Ther­a­peu­tics (LSE: $SLN) and iTher­a­peu­tics.

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Af­ter the field left her be­hind, a sci­en­tist-turned-in­vestor gets her first R&D job; Nous­com finds new lead­er­ship for its can­cer vac­cines

Before she boarded the plane, Cristina Ghenoiu spent most school day afternoons at Bucharest’s National Museum of Natural History, studying endangered animals Romanian scientists had brought back from around the country, or the world. The communist government sponsored a wide range of programs for kids so both parents could work. Her sister danced; Ghenoiu fell in love with biology.

And she was good, at least good enough to win several national awards and then, at 15, a spot as Romania’s representative to an international school in Canada that accepted about one person per country.

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Brian Stuglik (file photo)

Turn­ing fo­cus to clin­i­cal work, Ve­rastem ax­es 31 jobs, scales back can­cer drug pro­mo­tion af­ter dis­ap­point­ing sales

Months after taking the helm at Verastem Oncology, Brian Stuglik has a plan to take the biotech in a “new strategic direction” — but not before some layoffs.

Left out of an upbeat press release spelling out its clinical plans, and buried below news of a $100 million private placement in an SEC filing, is a planned restructuring that will claim 31 jobs. Alongside some other cost-saving measures, Verastem expects to cut expenses down by $70 million to $80 million per year.

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Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

Sage con­firms sus­pen­sion of 2 de­pres­sion tri­als af­ter PhI­II flop; Es­pe­ri­on fol­lows up maid­en ap­proval with com­bo OK

→ In the wake of a flop in the crucial Phase III MOUNTAIN study, Sage Therapeutics confirmed in its quarterly update that it’s suspended enrollment in two other pivotal trials for the oral depression drug SAGE-217 (or zuranolone) as it awaits guidance from the FDA. While REDWOOD (measuring relapse) and RAINFOREST (for patients with both major depressive disorder and insomnia) are on hold pending amendments, though, the open-label SHORELINE has completed enrollment. CEO Jeff Jonas remained tight-lipped about what specific tweaks they are considering for the program, reiterating only there have been issues with compliance and room for a higher dose.

Dan O'Day (AP Images)

UP­DAT­ED: A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisition, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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