Ver­tex con­tin­ues shop­ping, spend­ing $20M up­front to ac­cess Ri­bometrix's RNA-drug­ging plat­form

Ver­tex, flush with $4 bil­lion in cash and cash equiv­a­lents (as of June 30), has been on the prowl to park its ex­cess cap­i­tal in places with po­ten­tial. Its lat­est in­jec­tion is in Durham, North Car­oli­na-based Ri­bometrix — a start­up work­ing on tar­get­ing RNA with small mol­e­cule drugs.

Kevin Weeks UNC

Cre­at­ed by Kevin Weeks, a chemist at the Uni­ver­si­ty of North Car­oli­na at Chapel Hill, and his for­mer un­der­grad­u­ate stu­dent Katie Warn­er — the com­pa­ny is part of a slate of drug de­vel­op­ers such as Sky­hawk Ther­a­peu­tics, Ar­rakis and Ex­pan­sion Ther­a­peu­tics, as well as No­var­tis $NVS and Mer­ck $MRK who have sim­i­lar lofty am­bi­tions.

While most ex­ist­ing drugs fo­cus on pro­teins, Ri­bometrix is work­ing on chang­ing the be­hav­ior of dis­ease-caus­ing RNA em­broiled in neu­rode­gen­er­a­tive dis­eases and can­cer. Ri­bometrix’s tech­nol­o­gy sug­gests strands of RNA im­pli­cat­ed in dis­ease are char­ac­ter­ized by clefts and crevices in­to which small-mol­e­cule drugs can bind, which in turn can mod­u­late RNA ac­tiv­i­ty.

Ear­li­er this month, Ri­bometrix raised $7.8 mil­lion, to add to the $30 mil­lion Se­ries A fund­ing round — backed by Mer­ck and Am­gen — un­veiled in No­vem­ber 2018. Ver­tex has now put in $20 mil­lion up­front — in­clud­ing an eq­ui­ty in­vest­ment — in a deal that will see the two en­ti­ties work to­geth­er on three ther­a­peu­tic pro­grams, in­clud­ing one on­go­ing dis­cov­ery pro­gram from Ri­bometrix.

Ri­bometrix is el­i­gi­ble to re­ceive more than $700 mil­lion in mile­stones pay­ments, in ad­di­tion to po­ten­tial net glob­al sales on any prod­ucts that re­sult from the col­lab­o­ra­tion.

For Ver­tex $VRTX, the deal is valu­able as it looks to ex­pand its tech­no­log­i­cal prowess be­yond its block­buster slate of cys­tic fi­bro­sis (CF) treat­ments. As it awaits the ap­proval of its CF triplet — which is en­gi­neered to treat a much broad­er set of pa­tients — the com­pa­ny has sig­naled its ap­petite for deals.

Er­ic Ol­son UT South­west­ern

Ear­li­er this year, Ver­tex agreed to buy gene-edit­ing pi­o­neer Ex­on­ics — born in the lab of UT South­west­ern in­ves­ti­ga­tor Er­ic Ol­son — for $245 mil­lion, while set­ting aside an­oth­er $175 mil­lion to ex­pand its work with Swiss-based gene-edit­ing com­pa­ny CRISPR Ther­a­peu­tics. In Sep­tem­ber, Ver­tex agreed to spend close to a bil­lion dol­lars to pur­chase Sem­ma Ther­a­peu­tics — a biotech com­pa­ny work­ing on a cure for type I di­a­betes, based on decades of work by Har­vard pro­fes­sor Doug Melton.

The Boston biotech’s CEO Jef­frey Lei­den is al­so plan­ning to tran­si­tion to the chair­man’s role next spring, to make room for cur­rent CMO Resh­ma Ke­wal­ra­mani.

So­cial im­age: Ver­tex


UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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Vas Narasimhan. Getty Images

UP­DAT­ED: Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and briefly spread the chill to a high-pro­file biotech

Back in July, during an investor call where Novartis execs ran through an upbeat assessment of their Q2 performance, CEO Vas Narasimhan and development chief John Tsai were pressed to predict which of the two looming Phase III readouts — involving cardio drug Entresto and asthma therapy fevipiprant, respectively — had a higher likelihood of success. Tsai gave the PARAGON-HF study with Entresto minimally better odds, but Narasimhan emphasized that their strategy of giving fevipiprant to more severe patients gave them confidence.

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UP­DAT­ED: The FDA sets a reg­u­la­to­ry speed record, pro­vid­ing a snap OK for Ver­tex's break­through triplet for cys­tic fi­bro­sis

The FDA has approved Vertex’s new triplet for cystic fibrosis at a record-setting speed.

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IM­brave150: Roche’s reg­u­la­to­ry crew plans a glob­al roll­out of Tecen­triq com­bo for liv­er can­cer as PhI­II scores a hit

Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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UP­DAT­ED: Clay Sie­gall’s $614M wa­ger on tu­ca­tinib pays off with solid­ly pos­i­tive piv­otal da­ta and a date with the FDA

Back at the beginning of 2018, Clay Siegall snagged a cancer drug called tucatinib with a $614 million cash deal to buy Cascadian. It paid off today with a solid set of mid-stage data for HER2 positive breast cancer that will in turn serve as the pivotal win Siegall needs to seek an accelerated approval in the push for a new triplet therapy.

And if all the cards keep falling in its favor, they’ll move from 1 drug on the market to 3 in 2020, which is shaping up as a landmark year as Seattle Genetics prepares for its 23rd anniversary on July 15.

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David Liu, Liu Group

David Liu un­veils newest ad­vance­ment in CRISPR tech: Prime edit­ing

The researcher behind base-editing is out with what some scientists are hailing as the biggest advancement in CRISPR technology since that 2016 breakthrough: “prime editing.” The new molecular gadget is capable of erasing any base pair and stenciling in another and cutting or adding long segments of DNA without breaking both strands of the helix.

David Liu, base editing pioneer and founder of Beam Therapeutics, published the findings in Nature alongside Andrew Anzalone. They estimated that the breakthrough “in principle” puts 89% of human diseases in purview — although experts cautioned that human therapies were a long way off.

Bhaskar Chaudhuri. Frazier Healthcare Partners

Fra­zier Health­care Part­ner­s' der­ma­tol­ogy up­start at­tracts a mar­quee syn­di­cate, $94M+ for 'in-be­tween' top­i­cal drug

For the past three years Frazier Healthcare Partners’ Bhaskar Chaudhuri has been carefully and quietly grooming Arcutis Therapeutics, a new dermatology play he co-founded to deliver topical formulations of well-known drugs. Now that the biotech is poised to enter Phase III, he’s being joined by a marquee syndicate for its $94.5 million Series C.

HBM Healthcare Investments, Vivo Capital, BlackRock, Omega Funds, Pivotal BioVentures, and Goldman Sachs jumped on board, joining Bain Capital Life Sciences, OrbiMed and RA Capital Management in backing Arcutis’ lead topical cream for plaque psoriasis.

A new com­pa­ny en­ters the Tec­fidera fight, of­fer­ing to kill two birds

The remedy for the most common side effect for one of the most common multiple sclerosis drugs is simple: aspirin.

Taking aspirin with Biogen’s Tecfidera will reduce the flush, a sometimes painful form of red skin irritation, many patients experiences. The problem is that the aspirin has to be taken at least 30 minutes before Tecfidera, turning a simple twice-a-day, one-dose oral drug into a staggered two-drug regimen.

UP­DAT­ED: Bris­tol-My­ers makes Op­di­vo pitch for front­line lung can­cer with open la­bel PhI­II study

Despite a head start, when Bristol-Myers Squibb and its pioneering checkpoint inhibitor Opdivo suffered a key lung cancer setback in 2016, they found themselves relegated to the backseat as Merck’s Keytruda seized the wheel on the road to immunotherapy stardom. Bristol-Myers has since suffered blow after blow in its quest to take a big slice of the lucrative market, peppered with some small successes. On Tuesday, the New Jersey drugmaker touted positive data from a Phase III open-label study in a bid to carve itself a piece of the frontline lung cancer market.