Sarep­ta posts ear­ly, rosy gene ther­a­py da­ta for limb gir­dle mus­cu­lar dy­s­tro­phy — fu­el­ing $165M buy­out of part­ner

Sarep­ta Ther­a­peu­tics $SRPT has re­vealed a promis­ing, ear­ly snap­shot of re­sults for its sec­ond gene ther­a­py pro­gram in pa­tients with a pro­gres­sive mus­cle de­gen­er­a­tive dis­or­der — months af­ter re­port­ing en­cour­ag­ing da­ta for its Duchenne gene ther­a­py, as the com­pa­ny for­ti­fies its sta­tus as a bonafide gene ther­a­py play­er. And the re­sults in­spired a $165 mil­lion buy­out.

Doug In­gram

On Wednes­day, Sarep­ta di­vulged da­ta from three pa­tients in the first co­hort of an open-la­bel Phase I/II study test­ing the use of an ex­per­i­men­tal gene ther­a­py — MYO-101 — in limb gir­dle mus­cu­lar dy­s­tro­phy (LGMD), a group of rare pro­gres­sive ge­net­ic dis­or­ders char­ac­ter­ized by wast­ing and weak­ness of the vol­un­tary mus­cles of the hip and shoul­der ar­eas (limb-gir­dle area) that have no ap­proved treat­ments.

The ther­a­py — which in­fus­es the cor­rec­tive gene us­ing a virus as a ve­hi­cle — is de­signed to treat LGMD2E, al­so known as be­ta-sarco­gly­canopa­thy, a se­vere form of LGMD char­ac­ter­ized by pro­gres­sive mus­cle fiber loss, in­flam­ma­tion and mus­cle fiber re­place­ment with fat and fi­brot­ic tis­sue.

MYO-101, along with four oth­er ex­per­i­men­tal LGMD gene ther­a­pies, was in-li­censed for $60 mil­lion by Sarep­ta last May from a pri­vate biotech called My­onexus (which burst on­to the scene with $2.5 mil­lion in seed fund­ing in 2017). On Wednes­day, Sarep­ta said it had ex­er­cised its op­tion to buy the com­pa­ny for $165 mil­lion.

Tri­al da­ta on the three pa­tients showed the ther­a­py re­ju­ve­nat­ed the pro­duc­tion, by an av­er­age of 51%, of be­ta-sarco­gly­can, the pro­tein re­quired for mus­cle func­tion that is miss­ing in this pa­tient pop­u­la­tion. The da­ta were mea­sured fol­low­ing a mus­cle biop­sy 60 days fol­low­ing in­fu­sion.

Whether the en­hanced pro­duc­tion of the be­ta-sarco­gly­can trans­lates to func­tion­al im­prove­ments re­mains to be seen, and the com­pa­ny will pro­vide those da­ta as it an­a­lyzes them at an up­com­ing med­ical meet­ing.

Cred­it Su­isse’s Mar­tin Auster pre­dict­ed that the ev­i­dence of po­ten­tial func­tion­al im­prove­ments ver­sus nat­ur­al his­to­ry da­ta would be made at the MDA con­fer­ence sched­uled for April 13 to 17.

How­ev­er, based on pre­clin­i­cal da­ta, Sarep­ta said that func­tion­al im­prove­ments are seen with more than a 20% im­prove­ment in be­ta-sarco­gly­can pro­duc­tion.

In the tri­al, one of the sec­ondary end­points was the re­duc­tion in cre­a­tine ki­nase (CK), an en­zyme found in the blood that is typ­i­cal­ly el­e­vat­ed as a re­sult of mus­cle dam­age. Ini­tial da­ta showed that MYO-101 dra­mat­i­cal­ly re­duced CK lev­els on av­er­age by 90%, Sarep­ta said.

“We think every mea­sure of ef­fect ex­ceeds ex­pec­ta­tions and like­ly puts this pro­gram on a fast track to ap­proval,” Baird’s Bri­an Sko­r­ney wrote in a note.

Un­like Duchenne pa­tients, those af­flict­ed with LGMD are typ­i­cal­ly not treat­ed with steroids as stan­dard, back­ground ther­a­py. In this tri­al, pa­tients were start­ed on steroids be­fore they were in­fused with the gene ther­a­py.

Two pa­tients were fol­lowed up af­ter 90 days had el­e­vat­ed liv­er en­zymes — an is­sue that has cropped up in oth­er gene ther­a­py tri­als such as AveX­is’ (No­var­tis) SMA pro­gram — with one deemed as a se­ri­ous ad­verse event. How­ev­er, Sarep­ta said the lofty liv­er sig­nal oc­curred when the pa­tients were ta­pered off oral steroids, and symp­toms re­solved up­on sup­ple­men­tal steroid treat­ment.

The co­hort da­ta, al­though ear­ly, have a po­ten­tial read-through to all the limb-gir­dle pro­grams, Sarep­ta CEO Doug In­gram said on a con­fer­ence call with an­a­lysts on Wednes­day. The com­pa­ny’s shares $SRPT jumped about 8% in ear­ly trad­ing.

The promise of gene ther­a­pies is in­tox­i­cat­ing as they are po­ten­tial one-shot cures for pre­vi­ous­ly un­touch­able dis­eases. In just a lit­tle over a year now, SanofiNo­var­tis and Roche have all in­vest­ed in with multi­bil­lion-dol­lar gene ther­a­py M&A deals, snap­ping up the pi­o­neers in the field. Ear­li­er this week, Roche mag­ni­fied the en­thu­si­asm with its $4.3 bil­lion bet on Spark Ther­a­peu­tics $ONCE, a com­pa­ny that has ef­fec­tive­ly helped a cat­e­go­ry of blind pa­tients see again.

“While im­me­di­ate readthrough (of the Sarep­ta da­ta) goes to LGMD2B and 2C, the promise goes be­yond LGMD. We think this is what ul­ti­mate­ly dri­ves long-term val­ue here as it makes the foun­da­tion be­hind Roche’s $4.8B Spark ac­qui­si­tion seem enor­mous­ly spec­u­la­tive and lim­it­ed in con­trast,” Sko­r­ney said.

Nick Leschly via Getty

UP­DAT­ED: Blue­bird shares sink as an­a­lysts puz­zle out $1.8M stick­er shock and an un­ex­pect­ed de­lay

Blue­bird bio $BLUE has un­veiled its price for the new­ly ap­proved gene ther­a­py Zyn­te­glo (Lenti­Glo­bin), which came as a big sur­prise. And it wasn’t the on­ly un­ex­pect­ed twist in to­day’s sto­ry.

With some an­a­lysts bet­ting on a $900,000 price for the β-tha­lassemia treat­ment in Eu­rope, where reg­u­la­tors pro­vid­ed a con­di­tion­al ear­ly OK, blue­bird CEO Nick Leschly said Fri­day morn­ing that the pa­tients who are suc­cess­ful­ly treat­ed with their drug over 5 years will be charged twice that — $1.8 mil­lion — on the con­ti­nent. That makes this drug the sec­ond most ex­pen­sive ther­a­py on the plan­et, just be­hind No­var­tis’ new­ly ap­proved Zol­gens­ma at $2.1 mil­lion, with an­a­lysts still wait­ing to see what kind of pre­mi­um can be had in the US.

Ted Love. HAVERFORD COLLEGE

Glob­al Blood Ther­a­peu­tics poised to sub­mit ap­pli­ca­tion for ac­cel­er­at­ed ap­proval, with new piv­otal da­ta on its sick­le cell dis­ease drug

Global Blood Therapeutics is set to submit an application for accelerated approval in the second-half of this year, after unveiling fresh data from a late-stage trial that showed just over half the patients given the highest dose of its experimental sickle cell disease drug experienced a statistically significant improvement in oxygen-wielding hemoglobin, meeting the study's main goal.

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News­mak­ers at #EHA19: Re­gen­eron, Ar­Qule track progress on re­sponse rates

Re­gen­eron’s close­ly-watched bis­pe­cif­ic con­tin­ues to ring up high re­sponse rates

Re­gen­eron’s high-pro­file bis­pe­cif­ic REGN1979 is back in the spot­light at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion sci­en­tif­ic con­fab. And while the stel­lar num­bers we saw at ASH have erod­ed some­what as more blood can­cer pa­tients are eval­u­at­ed, the re­sponse rates for this CD3/CD20 drug re­main high.

A to­tal of 13 out of 14 fol­lic­u­lar lym­phomas re­spond­ed to the drug, a 93% ORR, down from 100% at the last read­out. In 10 out of 14, there was a com­plete re­sponse. In dif­fuse large B-cell lym­phoma the re­sponse rate was 57% among pa­tients treat­ed at the 80 mg to 160 mg dose range. They were all com­plete re­spons­es. And 2 of these Cars were for pa­tients who had failed CAR-T ther­a­py.

Gene ther­a­pies seize the top of the list of the most ex­pen­sive drugs on the plan­et — and that trend has just be­gun

Anyone looking for a few simple reasons why the gene therapy field has caught fire with the pharma giants need only look at the new list of the 10 most expensive therapies from GoodRx.

Two recently approved gene therapies sit atop this list, with Novartis’ Zolgensma crowned the king of the priciest drugs at $2.1 million. Right below is Luxturna, the $850,000 pioneer from Spark, which Roche is pushing hard to acquire as it adds a gene therapy group to the global mix.

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Neil Woodford, Woodford Investment Management via YouTube

Un­der siege, in­vest­ment man­ag­er Wood­ford faces an­oth­er in­vest­ment shock

Em­bat­tled UK fund man­ag­er Neil Wood­ford — who has con­tro­ver­sial­ly blocked in­vestors from pulling out from his flag­ship fund to stem the blood­let­ting, af­ter a slew of dis­ap­point­ed in­vestors fled fol­low­ing a se­ries of sour bets — is now pay­ing the price for his ac­tions via an in­vestor ex­o­dus on an­oth­er fund.

Har­g­reaves Lans­down, which has in the past sold and pro­mot­ed the Wood­ford funds via its re­tail in­vest­ment plat­form, has re­port­ed­ly with­drawn £45 mil­lion — its en­tire po­si­tion — from the in­vest­ment man­ag­er’s In­come Fo­cus Fund.

Search­ing for the next block­buster to fol­low Darza­lex, J&J finds a $150M an­ti-CD38 drug from part­ner Gen­mab

Now that J&J and Genmab have thrust Darzalex onto the regulatory orbit for first-line use in multiple myeloma, the partners are lining up a deal for a next-gen follow-on to the leading CD38 drug.


Janssen — J&J’s biotech unit — has its eyes on HexaBody-CD38, a preclinical compound generated on Genmab’s tech platform designed to make drugs more potent via hexamerization.


Genmab is footing the bill on studies in multiple myeloma and diffuse large B-cell lymphoma; once it completes clinical proof of concept, Janssen has the option to license the drug for a $150 million exercise fee. There’s also $125 million worth of milestones in play.

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Savara shares are crushed as PhI­II tri­al flunks pri­ma­ry, key sec­on­daries — but they can’t stop be­liev­ing

In­vestors are in no mood to hear biotechs tout the suc­cess of a “key” sec­ondary end­point when the piv­otal Phase III flunks the pri­ma­ry goal. Just ask Savara. 

The Texas biotech $SVRA went look­ing for a sil­ver lin­ing as com­pa­ny ex­ecs blunt­ly con­ced­ed that Mol­gradex, an in­haled for­mu­la­tion of re­com­bi­nant hu­man gran­u­lo­cyte-macrophage colony-stim­u­lat­ing fac­tor (GM-CSF), failed to spur sig­nif­i­cant­ly im­proved treat­ment out­comes for pa­tients with a rare res­pi­ra­to­ry dis­ease called au­toim­mune pul­monary alve­o­lar pro­teinosis, or aPAP.

As an­oth­er an­tibi­otics biotech sinks in­to a cri­sis, warn­ings of a sec­tor ‘col­lapse’

Another antibiotics company is scrambling to survive today, forcing the company’s founding CEO to exit in a reorganization that eliminates its research capabilities as the survivors look to improve on minuscule sales of their newly approved treatment. And the news — on top of an alarming series of failures — spurred at least one figure in the field to warn of a looming collapse of the antimicrobial resistance research field.

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Fol­low­ing CAR-T pi­o­neer­s' foot­steps, Tes­sa launch­es Chi­na JV in $120M deal

These days just about every biotech se­ri­ous about glob­al de­vel­op­ment — and not just com­mer­cial­iza­tion — has a Chi­na strat­e­gy. Tes­sa Ther­a­peu­tics, a Bay­lor as­so­ci­at­ed out­fit based out of Sin­ga­pore, is no ex­cep­tion.

Tak­ing a page out of the CAR-T pi­o­neers’ play­book, Tes­sa is es­tab­lish­ing a joint ven­ture with Chi­na-Sin­ga­pore Guangzhou Knowl­edge City, which is ini­tial­ly putting down $40 mil­lion for a 13% stake with $40 mil­lion more to come in a sec­ond stage. The biotech, which now re­tains an 87% con­trol, is al­so rolling out its own con­tri­bu­tions in two phas­es, start­ing with $20 mil­lion and all its tech­nol­o­gy li­cense rights for Chi­na.