Jeff Leiden, Vertex via YouTube

Jump­ing in­to grow­ing queue of buy­ers, Ver­tex chief tar­gets a slate of new and 'larg­er' deals ahead

Resh­ma Ke­wal­ra­mani Ver­tex

Af­ter mak­ing it crys­tal clear that Jeff Lei­den plans to keep his hand di­rect­ly on the wheel of busi­ness de­vel­op­ment fol­low­ing his move from CEO to the ex­ec­u­tive chair­man’s spot, Lei­den him­self stepped up on Wednes­day to per­son­al­ly map out plans for a se­ries of new deals he ex­pects to or­ches­trate to beef up the pipeline.

Dur­ing the Q2 call with an­a­lysts, Lei­den her­ald­ed the near-term ap­proval ex­pect­ed for their triple com­bi­na­tion for cys­tic fi­bro­sis. And he made it abun­dant­ly clear that a string of new deals re­gard­ing gene edit­ing pro­grams for Duchenne mus­cu­lar dy­s­tro­phy — buy­ing Ex­on­ics and more — are just a pre­lude to more M&A pacts in the very near fu­ture as Resh­ma Ke­wal­ra­mani preps a move to the CEO suite.

Right at the in­tro, Lei­den not­ed:

These agree­ments pro­vide us with de­vel­op­ment can­di­dates that have shown promis­ing pre­clin­i­cal re­sults and al­so en­able us to in­te­grate cut­ting-edge sci­en­tif­ic tech­nol­o­gy and ex­per­tise in dis­eases that are high­ly aligned with our busi­ness strat­e­gy. We plan to ex­e­cute more of these types of fields as we fur­ther ex­pand our pipeline of trans­for­ma­tive med­i­cines over the com­ing months and years.

In high­light­ing his con­tin­ued pres­ence in the day-to-day op­er­a­tions, Lei­den spelled out his role in “busi­ness de­vel­op­ment, help­ing to get deals done and se­cure our ac­cess to ex­ter­nal in­no­va­tion in prod­ucts.”

In the Q&A, Lei­den was ex­plic­it that Ver­tex’s cash flow po­si­tion gives them plen­ty of fire­pow­er for do­ing more, and larg­er deals ahead, af­ter spend­ing $600 mil­lion over the past 12 months. What they won’t do, though, is buy up com­mer­cial or late-stage pro­grams clos­ing in on an ap­proval.


As we said be­fore, first of all, we are ac­cu­mu­lat­ing sig­nif­i­cant fi­nan­cial fire­pow­er cap­i­tal in our bal­ance sheet, and so you should ex­pect to see us do more deals and po­ten­tial­ly larg­er deals. But the strat­e­gy will re­main the same as it’s been for the last four years. And as you know, we fo­cus on three ar­eas. Any­thing in CF that could be com­ple­men­tary or were ad­di­tive to what we’re do­ing now is triple. Ob­vi­ous­ly, we’re not see­ing any of those be­cause the triple has set such a high bar, but we con­tin­ue to look at every­thing out there.

The sec­ond one is tech­nolo­gies — our tech­nol­o­gy plat­forms that would al­low us to bet­ter treat the kinds of dis­eases which you’ve heard about to­day ei­ther alone or po­ten­tial­ly in com­bi­na­tion with small mol­e­cules. And you’ve seen us do the CRISPR deal, the Mod­er­na deal, the Ar­bor deal, the X-Chem deal, all of those fall in­to that cat­e­go­ry.

And then the third area is look­ing for as­sets most­ly pre­clin­i­cal and ear­ly clin­i­cal as­sets that will com­ple­ment our pipeline in the dis­eases we’re in­ter­est­ed in. In a way, Ex­on­ics was a part of that be­cause DMD and DM1 are two dis­eases we’re in­ter­est­ed in and we con­tin­ue to look for those as­sets.

Ver­tex’s de­ci­sion to go af­ter deals comes dur­ing one of the busiest M&A sea­sons we’ve seen in years, with a host of ma­jor play­ers open­ing up their check­books to buy new drugs for the pipeline. Most, Like Ver­tex, are fo­cused clear­ly on ear­ly and mid-stage as­sets, where they hope to find a few rel­a­tive bar­gains in the R&D bin. In just the last few days we’ve seen Daniel O’Day at Gilead as well as Al­bert Bourla at Pfiz­er high­light their in­ter­est in more deals. So he’ll have plen­ty of com­pa­ny at the bar­gain­ing ta­ble. All of this will al­so con­tin­ue to feed in­to the ven­ture cy­cle that’s been see­ing more and big­ger funds come along to back star­tups.

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.

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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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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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

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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