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.

Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Hal Barron, GSK's president of R&D and CSO, speaks to Endpoints News founder and editor John Carroll in London at Endpoints' #UKBIO19 summit on October 8, 2019

[Video] Cel­e­brat­ing tri­al fail­ures, chang­ing the cul­ture and al­ly­ing with Cal­i­for­nia dream­ers: R&D chief Hal Bar­ron talks about a new era at GSK

Last week I had a chance to sit down with Hal Barron at Endpoints’ #UKBIO19 summit to discuss his views on R&D at GSK, a topic that has been central to his life since he took the top research post close to 2 years ago. During the conversation, Barron talked about changing the culture at GSK, a move that involves several new approaches — one of which involves celebrating their setbacks as they shift resources to the most promising programs in the pipeline. Barron also discussed his new alliances in the Bay Area — including his collaboration pact with Lyell, which we covered here — frankly assesses the pluses and minuses of the UK drug development scene, and talks about his plans for making GSK a much more effective drug developer.

This is one discussion you won’t want to miss. Insider and Enterprise subscribers can log-in to watch the video.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?

Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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UP­DAT­ED: J&J's Xarel­to, Amar­in's Vas­cepa are cost-ef­fec­tive, not bud­get friend­ly — ICER

ICER, an increasingly influential cost-effectiveness watchdog in the United States, has concluded in its review of treatments for cardiovascular disease that while the cost of J&J’s Xarelto and Amarin’s Vascepa meet its benchmark for value pricing — the two treatments will not likely treat as many patients as hoped without surpassing the annual budget threshold calculated by ICER for each therapy.

Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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