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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Gene Wang, Immetas co-founder and CEO (file photo)

Im­metas Ther­a­peu­tics nabs $11M Se­ries A to nar­row their bis­pe­cif­ic work tar­get­ing in­flam­ma­tion in age-re­lat­ed dis­eases

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.