One year on, Capri­cor's stem cell ther­a­py ap­pears to help DMD pa­tients in small study, but in­vestors balk at the da­ta

Re­peat­ed set­backs aside, lit­tle Capri­cor has sug­gest­ed it has gen­er­at­ed some long-term da­ta to sup­port its pur­suit to gar­ner ap­proval for its stem cell ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy, al­though some of the da­ta ap­peared to un­der­whelmed in­vestors.

The da­ta from the small, place­bo-con­trolled mid-stage study, HOPE-2, tracked the ef­fects of the com­pa­ny’s stem cell ther­a­py CAP-1002, which is de­signed to tem­per the in­flam­ma­tion as­so­ci­at­ed with DMD, in 8 boys and young men who are in ad­vanced stages of DMD. The re­main­ing 12 en­rolled pa­tients re­ceived the place­bo.

The main goal of the study was a mea­sure that eval­u­ates shoul­der, arm and hand strength in pa­tients who are gen­er­al­ly non-am­bu­lant (per­for­mance of the up­per limb (PUL) 2.0), as sug­gest­ed by the FDA, Capri­cor said. It is one of sev­er­al ways Capri­cor quan­ti­fied skele­tal mus­cle im­prove­ment in the tri­al.

The in­tra­venous in­fu­sion of CAP-1002, giv­en every 3 months, in­duced a sta­tis­ti­cal­ly mean­ing­ful im­prove­ment of 2.4 points (p=0.05) ver­sus the place­bo group, in which pa­tient de­clines were con­sis­tent with nat­ur­al his­to­ry da­ta. How­ev­er, on an­oth­er mea­sure of up­per limb func­tion, the trend was in fa­vor of the Capri­cor drug, but did not hit sta­tis­ti­cal sig­nif­i­cance.

The com­pa­ny’s shares $CAPR were down near­ly 13% to $6.89 in morn­ing trad­ing.

Source: Capri­cor, 2020

Click on the im­age to see the full-sized ver­sion

Mean­while, there were al­so some en­cour­ag­ing da­ta on car­diac func­tion — the ge­net­ic con­di­tion is char­ac­ter­ized by pro­gres­sive weak­ness and chron­ic in­flam­ma­tion of the skele­tal, heart and res­pi­ra­to­ry mus­cles.

As re­flect­ed above, CAP-1002 elicit­ed an im­prove­ment across dif­fer­ent mea­sures of car­diac func­tion, al­though the ef­fect was not al­ways sta­tis­ti­cal­ly sig­nif­i­cant. In par­tic­u­lar, the drug al­so caused a re­duc­tion in the lev­els of the bio­mark­er CK-MB, an en­zyme that is on­ly re­leased when there is car­diac mus­cle cell dam­age.

Armed with these da­ta and an RMAT and or­phan drug des­ig­na­tion from the FDA, Capri­cor is now hop­ing to eke out a plan with the FDA for mar­ket­ing ap­proval.

Ed­uar­do Mar­bán

LA-based Capri­cor ini­tial­ly set out to test the po­ten­tial of tech­nol­o­gy that Ed­uar­do Mar­bán, CEO Lin­da Mar­bán’s hus­band, de­vel­oped at Johns Hop­kins. But re­peat­ed set­backs clob­bered the com­pa­ny, which in 2014 trad­ed north of $14 a share. In 2017, J&J walked away from a col­lab­o­ra­tion on a stem cell ther­a­py for dam­aged hearts af­ter it flopped in the clin­ic.

In late 2018, the com­pa­ny vol­un­tar­i­ly halt­ed a DMD clin­i­cal tri­al, fol­low­ing a “se­vere al­ler­gic re­ac­tion” that oc­curred dur­ing in­fu­sion. In Feb­ru­ary 2019, the com­pa­ny said it is ex­plor­ing strate­gic al­ter­na­tives for one or more of its prod­ucts and cut­ting 21 jobs to keep fi­nan­cial­ly afloat, but had re­sumed dos­ing in its DMD tri­al.

The first batch of pos­i­tive da­ta on CAP-1002, which con­sists of prog­en­i­tor cells de­rived from donor hearts and is de­signed to ex­ude ex­o­somes that ini­ti­ate mus­cle re­pair by sup­press­ing in­flam­ma­tion and dri­ving im­munomod­u­la­tion, came last Ju­ly when the com­pa­ny an­nounced the drug had gen­er­at­ed a pos­i­tive ef­fect at the in­ter­im analy­sis junc­ture of HOPE-2. Capri­cor is now work­ing on to flex­ing its ther­a­peu­tic mus­cle with CAP-1002 to fight the Covid-19 pan­dem­ic.

DMD is a rare mus­cle-wast­ing dis­ease caused by the ab­sence of dy­s­trophin, a pro­tein that helps keep mus­cle cells in­tact. It dis­pro­por­tion­ate­ly af­fects boys — and af­fects rough­ly 6,000 in the Unit­ed States.

Pa­tients are es­sen­tial­ly treat­ed with steroids. Sarep­ta Ther­a­peu­tics now has two ex­on-skip­ping drugs de­signed to treat cer­tain sub­sets of the dis­ease, al­though the mag­ni­tude of their ef­fect is con­tro­ver­sial giv­en that ap­provals were not based on place­bo-con­trolled da­ta. Mean­while, Sarep­ta and oth­ers are al­so pur­su­ing one-time cures in the form of gene ther­a­pies to re­place the miss­ing dy­s­trophin gene in pa­tients.

So­cial: Lin­da Mar­bán, Capri­cor CEO (Twit­ter)

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Tom Barnes, Orna Therapeutics CEO

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Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

FDA ap­proves one of the prici­est new treat­ments of all time — blue­bird's gene ther­a­py for be­ta tha­lassemia

The FDA on Wednesday approved the first gene therapy for a chronic condition — bluebird bio’s new Zynteglo (beti-cel) as a potentially curative treatment for those with transfusion-dependent thalassemia.

The thumbs up from the FDA follows a unanimous adcomm vote in June, with outside experts pointing to extraordinary efficacy, with 89% of subjects with TDT who received beti-cel having achieved transfusion independence.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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