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.

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

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James Sabry (Roche)

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

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Testing 3 different doses of their drug, which relies on ADAMTS-5 inhibition, investigators concluded that none of them triggered a statistically significant response — as measured by cartilage thickness.

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Covid-19 roundup: Pars­ing Bourla, a top an­a­lyst sees im­proved chances for Pfiz­er vac­cine; Fau­ci: No sur­prise that Trump was hit by Covid-19

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