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.

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

Avance Clinical is the specialist Australian CRO, with CGT accreditation, for international biotechs that leverages Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend.

Learn more about Avance ClinicReady here.
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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Joan Perelló, Sanifit CEO

Joan Perel­ló set out 17 years ago to de­vel­op a drug. And to­day he's be­ing re­ward­ed with a $424M biotech buy­out

Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.

Working on the far perimeter of the big US/European drug development scene, he took a drug born out of his PhD work and got enough seed cash to get started. That’s one near miracle. In the second near miracle he gathered a previously unheard of venture raise in Spain — helping build an industry ecosystem from scratch — to pursue a successful search for solid human data for his drug, SNF472. And while gathering a virtual team of developers from Europe and the US, the CEO/co-founder steered it into the late-stage arena.

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