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

Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.