Sarep­ta’s eteplirsen spurned as Eu­ro­pean reg­u­la­tors turn thumbs down on con­tro­ver­sial Duchenne MD drug

Sarep­ta’s ap­proval for eteplirsen — or Ex­ondys51 — in the US re­mains one of the most con­tro­ver­sial OKs in FDA his­to­ry, earn­ing a rare la­bel that says the biotech has yet to pro­duce ev­i­dence the drug works.

But light­ning won’t strike twice on the reg­u­la­to­ry path­way for this biotech.

The biotech re­port­ed Thurs­day af­ter­noon that the CHMP is prep­ping a neg­a­tive vote, bar­ring the drug from the con­ti­nent. And Sarep­ta shares im­me­di­ate­ly tum­bled 5% in af­ter-mar­ket trad­ing.

Doug In­gram

The drug, which has a sol­id set of sup­port­ers in the DMD com­mu­ni­ty, is get­ting snubbed de­spite the Eu­ro­peans’ ac­cep­tance of PTC Ther­a­peu­tics’ ri­val drug, which has failed re­peat­ed piv­otal stud­ies. But in DMD, reg­u­la­tors have a ten­den­cy to make things up as they go along, fre­quent­ly bend­ing the rules to ac­com­mo­date a pas­sion­ate group of pa­tients and par­ents — or not. PTC has been re­peat­ed­ly slapped down by the FDA, even af­ter Janet Wood­cock over­ruled a host of agency in­sid­ers with her in­sis­tence on ap­prov­ing eteplirsen.

Sarep­ta CEO Doug In­gram had this to say:

Un­for­tu­nate­ly, the CHMP’s trend vote was neg­a­tive. Based on dis­cus­sions with CHMP rep­re­sen­ta­tives, it is our un­der­stand­ing that the CHMP did not con­clude that eteplirsen is in­ef­fec­tive for ex­on 51 amenable pa­tients, but rather that Sarep­ta has not yet met the reg­u­la­to­ry thresh­old for con­di­tion­al ap­proval, in part due to the use of ex­ter­nal con­trols as com­para­tors in the stud­ies. Sarep­ta plans to file for re-ex­am­i­na­tion and will re­quest that a Sci­en­tif­ic Ad­vi­so­ry Group (SAG), which is made up of DMD and neu­ro­mus­cu­lar spe­cial­ists, be con­vened to pro­vide ex­pert guid­ance and in­sight in­to, among oth­er things, the va­lid­i­ty of the ex­ter­nal con­trols used and the im­por­tance of slow­ing pul­monary de­cline in pa­tients with DMD.

Hop­ing to take some of the sting out of the news, while dis­ap­point­ing some an­a­lysts with its Q1 per­for­mance, Sarep­ta al­so an­nounced a deal to part­ner with My­onexus Ther­a­peu­tics on its work de­vel­op­ing gene ther­a­pies for Limb-gir­dle mus­cu­lar dy­s­tro­phies. Sarep­ta paid $60 mil­lion up­front and of­fered $45 mil­lion more in mile­stones for the deal, which al­so pro­vides the biotech with a buy­out op­tion at proof-of-con­cept.

“My­onexus and its fo­cus on gene ther­a­py us­ing the AAVrh.74 vec­tor to treat forms of LGMD aligns bril­liant­ly with our vi­sion to emerge as one of the most mean­ing­ful glob­al pre­ci­sion ge­net­ic med­i­cine com­pa­nies by fo­cus­ing on the use of ge­net­ic med­i­cine to im­prove the lives of those with rare fa­tal dis­eases,” said In­gram in a pre­pared state­ment.

Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.

Sanjiv Patel, Relay CEO

Re­lay reaps $400M IPO wind­fall af­ter draw­ing the cur­tain on mo­tion-based drug de­sign pipeline

A red-hot venture market back in 2018 propelled Relay Therapeutics to a whopping $400 million crossover round. And now, the biotech is riding on a red-hot IPO market to a public debut that brought in the same exact amount.

The $400 million raise breaks down to 20 million shares sold at $20 each — above even the amended proposed range of $16 to $18.

It’s also double what Relay had penciled in with the initial S-1 filing, in yet another illustration that those figures are often nothing more than a placeholder these days.

BioAt­la rakes in $72.5M Se­ries D, ad­vanc­ing re­search for pH-de­tect­ing can­cer treat­ments

A little over a year after agreeing to a worldwide collaboration with hefty Chinese biotech BeiGene worth up to $270 million, BioAtla is ready for another haul.

The San Diego-based biotech pulled in a $72.5 million haul for their Series D financing, which the company announced Wednesday. Funds will go toward their four main clinical programs developing cancer treatments, two of which are currently in Phase II, BioAtla president and former Celgene COO Scott Smith said.

Mene Pangalos, AstraZeneca R&D chief (AstraZeneca via YouTube)

A day af­ter Mod­er­na vac­cine re­sults, ru­mors swirl of pend­ing As­traZeneca da­ta

A day after Moderna and the NIH published much-anticipated data from their Phase I Covid-19 vaccine trial, attention is turning to AstraZeneca which, according to a UK report, is expected to publish its own early data tomorrow.

ITV’s Robert Peston reported that AstraZeneca will publish the Phase I data in The Lancet. 

AstraZeneca and Moderna represent the two most ambitious Covid-19 vaccine efforts, having set the quickest timelines for approval (though they were recently joined in that regard by the Pfizer-BioNTech partnership) and some of the loftiest goals in total doses. Yet there is even less known about AstraZeneca’s vaccine’s effect on humans than there was about Moderna’s before yesterday. Although, in a controversial move, Moderna released some statistics from its Phase I in May, AstraZeneca has yet to say anything about what it saw in its Phase I trial — a move consistent with the scientific convention to withhold data until it can be published in a peer-reviewed journal.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

‘Plan­ning to vac­ci­nate every­one in the US,’ Mod­er­na out­lines ef­forts to sup­ply their Covid-19 vac­cine as man­u­fac­tur­ing ramps up ahead of PhI­II

Twelve days from the planned start of their Phase III pivotal trial, the executive crew at Moderna has set up the manufacturing base needed to begin production of the first 500,000 doses of their Covid-19 vaccine with plans to feed it into a global supply chain. But the initial batches will likely be ready in the US first, where company CEO Stéphane Bancel plans to be able to vaccinate everyone.

“We have started making commercial product at-risk, and will continue to do so every day and every week of the month,” Bancel told analysts during their morning call on the Phase I data just published in the New England Journal of Medicine.

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Covid-19 roundup: Vac­cine by end of 2020? Ken Fra­zier warns hype do­ing 'grave dis­ser­vice'

When it comes to setting expectations about a Covid-19 vaccine, Ken Frazier does not mince words.

Over a month after first casting doubts on the aggressive 12- to 18-month timeframe championed by the US government and his biopharma peers, the Merck CEO again cautioned against any hype around a quick vaccine approval.

In a wide-ranging interview with Harvard Business School professor Tsedal Neeley that touched other big topics such as race, Frazier emphasized that vaccines take a long time to develop. He would know: Out of the seven new vaccines introduced around the world in the past 25 years, four came from Merck.

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Jeff Albers, Blueprint CEO

Di­ag­nos­tic champ Roche buys its way in­to the RET ti­tle fight with Eli Lil­ly, pay­ing $775M in cash to Blue­print

When Roche spelled out its original $1 billion deal — $45 million of that upfront — with Blueprint to discover targeted therapies against immunokinases, the biotech partner’s RET program was still preclinical. Four years later, pralsetinib is on the cusp of potential approval and the Swiss pharma giant is putting in much more to get in on the commercial game.

Roche gains rights to co-develop and co-commercialize the drug, with sole marketing responsibility for places outside the US and China (where CStone has staked its claim).

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