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)

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Paul Hudson, Sanofi CEO (Getty Images)

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David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Af­ter star­ring at ASH last fall, Gilead’s new Forty Sev­en crew col­ors in more promis­ing da­ta for ma­grolimab at AS­CO

We now know the full, early-stage story behind the drug that inspired Gilead CEO Dan O’Day’s recent $5 billion acquisition of Forty Seven.

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Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

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Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

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The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

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Iron­wood, Ab­b­Vie kick de­layed-re­lease Linzess for­mu­la­tion to the curb af­ter tri­al fail­ure

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The experimental drug, MD-7246, failed to help patients with abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D) in a mid-stage study, prompting the partners to abandon the therapy.

First approved in 2012, Linzess (known chemically as linaclotide) enhances the activity of the intestinal enzyme guanylate cyclase-C to increase the secretion of intestinal fluid and then transit through the intestinal tract, as well as reduce visceral pain, to relieve pain and constipation associated with IBS.