Sum­mit shares rock­et up, fu­eled by an $842M pact with Duchenne leader Sarep­ta

Sum­mit CEO Glyn Ed­wards

With its mar­ket cap swelled by the FDA’s ap­proval of the con­tro­ver­sial Duchenne mus­cu­lar dy­s­tro­phy drug eteplirsen, Sarep­ta is now wheel­ing and deal­ing its way to new com­bi­na­tion ap­proach­es.

The Cam­bridge, MA-based biotech $SRPT has scored an $842 mil­lion deal to part­ner with Ox­ford, UK-based Sum­mit $SMMT Ther­a­peu­tics, bag­ging Eu­ro­pean-plus rights to a lead ther­a­py that fol­lows a com­ple­men­tary path­way to treat DMD.

The news trig­gered a big ral­ly for Sum­mit’s shares, send­ing the stock rock­et­ing up 111%. Ahead of the an­nounce­ment the mi­cro cap biotech had a mar­ket cap of $136 mil­lion.

In ex­change for re­gion­al com­mer­cial rights, start­ing in Eu­rope, Sum­mit earned a $40 mil­lion up­front, an­oth­er $42 mil­lion in de­vel­op­ment mile­stones – in­clud­ing a $22 mil­lion pay­ment when the last pa­tient is dosed in an on­go­ing Phase II. There’s al­so an­oth­er $440 mil­lion for re­lat­ed de­vel­op­ment mile­stones —for the lead prod­uct and sec­ond-gen prod­ucts — and a fur­ther $330 mil­lion for sales mile­stones, ac­cord­ing to SEC doc­u­ments.

It adds up to $842 mil­lion. And Sarep­ta al­so agreed to share R&D ex­pens­es in the deal.

The deal comes just days af­ter Sarep­ta struck a sep­a­rate col­lab­o­ra­tion deal with Cataba­sis to ex­plore the de­vel­op­ment of com­bi­na­tion drugs for Duchenne mus­cu­lar dy­s­tro­phy.

Sarep­ta is buy­ing in­to the fu­ture of ezutro­mid, as well as the next-gen drugs Sum­mit has been work­ing on. While Sarep­ta’s eteplirsen has fo­cused on us­ing ex­on-skip­ping tech to spur pro­duc­tion of dy­s­trophin to strength­en the boys slow­ly crip­pled by DMD, Sum­mit has been pur­su­ing utrophin mod­u­la­tion with the same goal in mind.

Utrophin is an­oth­er pro­tein the bears sim­i­lar­i­ties with dy­s­trophin. Work­ing on in­sights gained in the lab of Ox­ford’s Kay Davies, the the­o­ry is that adding utrophin can sub­sti­tute for dy­s­trophin. Sum­mit is look­ing for some clear ev­i­dence that it can work in pa­tients, though it’s al­so prepar­ing to go through the long haul and prove it in Phase III. Sum­mit is helmed by CEO Glyn Ed­wards, a high-pro­file fig­ure in the UK’s biotech com­mu­ni­ty.

Sarep­ta bull Bri­an Sko­r­ney at Baird gave the deal a quick thumbs up: “Though we think the deal has in­ter­est­ing po­ten­tial, near term we think this has more read-through to Sarep­ta’s con­fi­dence in an eteplirsen ap­proval in Eu­rope.”

Sarep­ta won an ex­tra­or­di­nary FDA ap­proval for eteplirsen based on a tiny tri­al and some con­tro­ver­sial bio­mark­er da­ta. The in­ter­nal dis­cus­sion over the drug’s fu­ture trig­gered a high-lev­el show­down at the agency, which Janet Wood­cock won by per­se­ver­ing over ve­he­ment ob­jec­tions to the drug from a group that in­clud­ed the agency’s chief sci­en­tist.

Sum­mit CEO Glyn Ed­wards said:

“This agree­ment pro­vides us with ac­cess to Sarep­ta’s de­vel­op­ment, reg­u­la­to­ry and com­mer­cial­i­sa­tion ex­per­tise for the con­tin­ued ad­vance­ment of our promis­ing utrophin mod­u­la­tor pipeline. We look for­ward to this part­ner­ship and work­ing to­geth­er to bring great ad­vances to pa­tients and fam­i­lies liv­ing with DMD.”

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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