Sarep­ta read­ies FDA pitch af­ter a small study spot­lights 1% dy­s­trophin add for golodirsen in Duchenne MD

Af­ter win­ning an FDA ap­proval for Ex­ondys51 as a new ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy with­out hav­ing to ac­tu­al­ly pro­duce da­ta to prove it worked, Sarep­ta $SRPT fol­lowed up this new morn­ing with a fresh set of da­ta from a small study un­der­scor­ing the abil­i­ty of its ex­per­i­men­tal fol­lowup ex­on-skip­ping drug golodirsen to trig­ger added pro­duc­tion of dy­s­trophin.

Doug In­gram, Sarep­ta CEO

The num­bers it cites, though, are tiny. De­spite that, Sarep­ta is telling an­a­lysts to­day that it plans to see if the FDA could be will­ing to give it an ac­cel­er­at­ed green light.

Prin­ci­pal in­ves­ti­ga­tor Francesco Muntoni not­ed:

All (25) treat­ed boys showed the an­tic­i­pat­ed ex­on skip­ping af­ter treat­ment and this re­sult­ed in a mean in­crease of dy­s­trophin pro­tein, as mea­sured by West­ern blot, from 0.095 per­cent at base­line to 1.019 per­cent of nor­mal af­ter at least one-year of treat­ment with golodirsen.

To un­der­score, that’s a claimed win with slight­ly more than 1% of nor­mal dy­s­trophin. And it will ar­rive at the FDA af­ter an in­ter­nal war was fought over Ex­ondys51, which Janet Wood­cock even­tu­al­ly won by shov­ing aside a line­up of reg­u­la­tors that tried to get in her way.

Some of the an­a­lysts found the out­come to be more than a lit­tle lean. Not­ed RBC’s Matthew Eck­ler:

To­day’s re­lease con­firms the abil­i­ty of Sar­pe­ta’s ex­on-skip­ping ap­proach to in­crease dy­s­trophin in the mus­cles of DMD pa­tients, in our view. How­ev­er, re­call that dur­ing FDA re­view of Ex­ondys 51’s NDA, the agency raised con­cerns around the clin­i­cal ben­e­fit of pro­duc­ing small amounts of dy­s­trophin (cit­ing 10% as the thresh­old for clin­i­cal ben­e­fit). In­deed, one of the bear cas­es we hear for SRPT is that the small amount of dy­s­trophin pro­duc­tion and lack of a con­firmed clin­i­cal ben­e­fit leave the door open to pay­or push­backs and de­nials. We’d still ex­pect some push­back around the head­line “1% dyst. How­ev­er re­gard­less of the ul­ti­mate clin­i­cal ben­e­fit, we think that with the cur­rent da­ta re­lease (and pos­si­bil­i­ty of ac­cel­er­at­ed ap­proval), in­vestors will start as­crib­ing val­ue to Sarep­ta’s pipeline, and that shares will bet­ter re­flect the in­trin­sic val­ue of the com­pa­ny be­yond Ex­ondys 51.

Bri­an Sko­r­ney at Baird likes it. His rea­sons in­clude:

Re­call Sarep­ta sub­mit­ted base­line and 48-week treat­ment dy­s­trophin analy­ses to the FDA last year from the PRO­MOVI study, which showed eteplirsen in­creased dy­s­trophin from 0.16% of nor­mal to 0.44% at week 48, a 0.28% ab­solute in­crease. To­day’s SRP-4053 re­sults are clear­ly more im­pres­sive, with an in­crease from 0.095% to 1.019% (p<0.001), al­most 3.5x the dy­s­trophin pro­duc­tion seen in the PRO­MOVI pa­tients. Get­ting across the 1% of nor­mal thresh­old ap­pears to be an im­por­tant psy­cho­log­i­cal thresh­old, as crit­ics have of­ten cit­ed the “frac­tion of a per­cent” is­sue for eteplirsen.

The com­pa­ny is out to prove that its ex­on-skip­ping tech does work ef­fec­tive­ly, but the biotech still has a very long way to go on that score. Nev­er­the­less, with some help­ful cheer­lead­ing on the side­lines, its stock jumped 15% on the re­lease.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

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.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,200+ biopharma pros reading Endpoints daily — and it's free.