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.

Lei­den:

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.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Novartis CEO Vas Narasimhan [via Bloomberg/Getty]

I’m not per­fect: No­var­tis chief Vas Narasimhan al­most apol­o­gizes in the wake of a new cri­sis

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But he couldn’t quite get there.

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FDA to Sarep­ta: Your wide­ly an­tic­i­pat­ed fol­lowup to Ex­ondys 51 is not get­ting an ac­cel­er­at­ed OK for Duchenne MD

In one of the least anticipated moves of the year, the FDA has rejected Sarepta’s application for an accelerated approval of its Duchenne MD drug golodirsen after fretting over safety issues.

In a statement that arrived after the bell on Monday, Sarepta explained the CRL, saying:

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Levi Garraway. Broad Institute via Youtube

Roche raids Eli Lil­ly for its next chief med­ical of­fi­cer as San­dra Horn­ing plans to step down

We found out Monday morning where Levi Garraway was headed after he left Eli Lilly as head of oncology R&D a few days ago. Roche named Garraway as their new chief medical officer, replacing Sandra Horning, who they say is retiring from the company.

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Af­ter a posse of Wall Street an­a­lysts pre­dict a like­ly new win for Sarep­ta, we're down to the wire on a crit­i­cal FDA de­ci­sion

As Bloomberg notes, most of the Wall Street analysts that cover Sarepta $SRPT are an upbeat bunch, ready to cheer on the team when it comes to their Duchenne MD drugs, or offer explanations when an odd setback occurs — as happened recently with a safety signal that was ‘erroneously’ reported last week.

Ritu Baral Cowen
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UP­DAT­ED: No­var­tis spin­off Nabri­va fi­nal­ly scores its first an­tibi­ot­ic ap­proval

In May, Nabriva Therapeutics suffered a setback after the FDA rejected its antibiotic for complicated urinary tract infections — the Novartis spinoff has now had some better luck with the US agency, which on Monday approved its other drug for community-acquired bacterial pneumonia.

The drug, lefamulin, has been developed as an intravenous and oral formulation and been tested in two late-stage clinical trials. The semi-synthetic compound, whose dosing can be switched between the two formulations, is engineered to inhibit the synthesis of bacterial protein by binding to a part of the bacterial ribosome.

Saqib Islam. CheckRare via YouTube

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SpringWorks and Satsuma — both biotech spinouts that have closed B rounds in April — are loading up with IPO cash to boost their respective late-stage plans.
SpringWorks

Bain-backed SpringWorks is the better-known company of the two, and it’s gunning for a larger windfall of $115 million to add to $228 million from previous financings. In the process, the Stamford, CT-based team is also drawing the curtains on the partnerships it has in mind for the pair of assets it had initially licensed from Pfizer.

Mi­nor­i­ty racial groups con­tin­ue to be dis­mal­ly rep­re­sent­ed in can­cer tri­als — study

Data reveal that different racial and ethnic groups — by nature and/or nurture — can respond differently in terms of pharmacokinetics, efficacy, or safety to therapeutics, but this disparity is not necessarily accounted for in clinical trials. A fresh analysis of the last decade of US cancer drug approvals suggests the trend continues, cementing previous research that suggests oncology trials are woefully under-representative of the racial makeup of the real world.

Van­da shares slide af­ter FDA spurns their big end­point and re­jects a pitch on jet lag re­lief

Back in the spring of last year, Vanda Pharmaceuticals $VNDA served up a hot stew of mixed data for a slate of endpoints related to what they called clear evidence that their melatonin sleep drug Hetlioz (tasimelteon) could help millions of travelers suffering from jet lag.

Never mind that they couldn’t get a planned 90 people in the study, settling for 25 instead; Vanda CEO Mihael H. Polymeropoulos said they were building on a body of data to prove it would help jet-lagged patients looking for added sleep benefits. And that, they added, would be worth a major upgrade from the agency as they sought to tackle a big market.