Sarep­ta halts its golodirsen Duchenne MD study in UK af­ter pa­tient ex­pe­ri­ences ad­verse event

Sarep­ta said this morn­ing that it has halt­ed dos­ing its Duchenne mus­cu­lar dy­s­tro­phy drug golodirsen at all its UK sites af­ter one of the pa­tients in the tri­al ex­pe­ri­enced an ad­verse event. But the biotech swift­ly in­di­cat­ed that it doesn’t ex­pect the halt to last for long.

The news was first re­port­ed by EP Van­tage writer Ja­cob Pli­eth, who not­ed chat­ter about the dos­ing halt on Face­book cit­ing is­sues with rhab­domy­ol­y­sis side ef­fects — a break­down of mus­cle tis­sue that trig­gers se­vere pain.

Doug In­gram, Sarep­ta CEO

Sarep­ta’s shares $SRPT slid 6.5% in pre-mar­ket trad­ing as the re­port cir­cu­lat­ed.

In a state­ment sent around af­ter the EP Van­tage sto­ry hit, Sarep­ta said that UK tri­al rules re­quired a stop­page at those sites, even though an in­de­pen­dent re­view board had looked over the safe­ty da­ta and con­clud­ed that the study could con­tin­ue. And it is — out­side of the UK. The com­pa­ny sent around a state­ment say­ing:

Pa­tients en­rolled in the study at UK study sites have tem­porar­i­ly stopped dos­ing due to UK spe­cif­ic stop­ping rules, trig­gered by one se­ri­ous ad­verse event (SAE) that could pos­si­bly be re­lat­ed to the in­ves­ti­ga­tion­al drug prod­uct.

The biotech is now seek­ing a green light to get dos­ing start­ed again in the UK.

As Sarep­ta not­ed, rhab­domy­ol­y­sis is not an un­com­mon symp­tom of DMD, which caus­es boys to steadi­ly weak­en be­fore it kills them. It’s a rare, dead­ly dis­ease, and Sarep­ta has tout­ed ear­ly ev­i­dence that the drug can pro­duce small quan­ti­ties of dy­s­trophin, hop­ing that the FDA will see enough cause to of­fer an ear­ly OK.

That’s a tall or­der, though, af­ter Sarep­ta won a con­tro­ver­sial ap­proval for Ex­ondys51 that re­quired Janet Wood­cock to car­ry it past a gamut of skep­tics at the agency who had been harsh­ly crit­i­cal of the biotech’s de­vel­op­ment ef­forts.

Here’s the full state­ment from Sarep­ta:

Study 4045-301 (ESSENCE) is a glob­al, ran­dom­ized dou­ble-blind, place­bo-con­trolled study eval­u­at­ing ef­fi­ca­cy and safe­ty in pa­tients with Duchenne mus­cu­lar dy­s­tro­phy (DMD) amenable to skip­ping ex­ons 45 or 53. Pa­tients en­rolled in the study at UK study sites have tem­porar­i­ly stopped dos­ing due to UK spe­cif­ic stop­ping rules, trig­gered by one se­ri­ous ad­verse event (SAE) that could pos­si­bly be re­lat­ed to the in­ves­ti­ga­tion­al drug prod­uct. The study re­mains blind­ed and the ad­verse event ob­served is con­sis­tent with those seen in pa­tients with DMD. The safe­ty da­ta from all pa­tients in the ESSENCE tri­al were re­viewed by an in­de­pen­dent Da­ta Mon­i­tor­ing Com­mit­tee (DMC). The DMC deemed that dos­ing could con­tin­ue for all pa­tients. How­ev­er, based on the UK spe­cif­ic stop­ping rules of the study, the Med­i­cine and Health­care prod­ucts Reg­u­la­to­ry Agency (MHRA) re­quired that dos­ing stop at all UK sites. Sarep­ta is cur­rent­ly sub­mit­ting an amend­ment to the MHRA. Fol­low­ing ap­proval from the MHRA, dos­ing can be reini­ti­at­ed at the UK sites.

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.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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