It has been a very bumpy ride for Capri­cor Ther­a­peu­tics over the past sev­er­al years, in­clud­ing a halt­ed J&J part­ner­ship and ven­ture in­to pen­ny stock sta­tus. How­ev­er, the lat­est news from the San Diego-based biotech shows that its ex­per­i­men­tal Duchenne drug may be a durable, long-term treat­ment.

The com­pa­ny post­ed the one-year re­sults from its open-la­bel ex­ten­sion study for their can­di­date dubbed CAP-1002. The ex­ten­sion from its Phase II study, named HOPE-2, was con­duct­ed in boys and young men with lat­er-stage Duchenne mus­cu­lar dy­s­tro­phy who paused and then re­sumed treat­ment af­ter a year. Re­searchers mea­sured pa­tients in the one-year fol­low-up against the orig­i­nal study’s treat­ment arm ef­fect and in two sub­sets of the orig­i­nal place­bo group.

Capri­cor’s drug hit the pri­ma­ry end­point on an up­per limb mus­cle func­tion scale when com­pared to all three groups. When pit­ted against the treat­ment arm, CAP-1002 in­duced a sta­tis­ti­cal­ly sig­nif­i­cant dif­fer­ence in the scale, good for a p-val­ue of p=0.023. Against the HOPE-2 place­bo group, the ex­ten­sion tri­al record­ed a p-val­ue of p=0.015. Against the group of place­bo pa­tients who re­mained off-treat­ment, CAP-1002 post­ed a val­ue of p=0.006.

The biotech said CAP-1002 was made avail­able to all 20 pa­tients orig­i­nal­ly en­rolled in the HOPE-2 study. Of those, 13 en­tered the ex­ten­sion and 12 com­plet­ed the first year of fol­low-up. As in HOPE-2, CAP-1002 was ad­min­is­tered quar­ter­ly and the cur­rent re­sults are from the one-year analy­sis.

“The open-la­bel ex­ten­sion phase of the HOPE-2 study is fair­ly unique in its de­sign in that all pa­tients were off CAP-1002 or place­bo for a mean of ap­prox­i­mate­ly one year be­fore re­sump­tion or ini­ti­a­tion of ther­a­py. These da­ta sug­gest pa­tients on CAP-1002 ac­cu­mu­late ben­e­fit over time where their skele­tal mus­cle func­tion is bet­ter pre­served which may in­di­cate a long-term po­ten­tial ben­e­fit of CAP-1002,” said Lin­da Mar­bán, Capri­cor’s CEO, in a state­ment.

Capri­cor’s can­di­date con­sists of al­lo­gene­ic car­dios­phere-de­rived cells, or CD­Cs, a type of prog­en­i­tor cell to ex­ert po­tent im­munomod­u­la­to­ry ac­tiv­i­ty and is be­ing in­ves­ti­gat­ed for its po­ten­tial to mod­i­fy the im­mune sys­tem’s ac­tiv­i­ty to en­cour­age cel­lu­lar re­gen­er­a­tion. The biotech is charg­ing ahead with the can­di­date, plan­ning to present these da­ta to the FDA.

In March, the re­sults from the Phase II were post­ed in The Lancet, fa­vor­ing CAP-1002 over place­bo in the mus­cle func­tion scale with a val­ue of p=0.014. Car­diac MRI as­sess­ments showed im­prove­ments in heart func­tion and struc­ture with CAP-1002 treat­ment.

In Jan­u­ary, the com­pa­ny signed a col­lab­o­ra­tion with Japan­ese phar­ma com­pa­ny Nip­pon Shinyaku. Capri­cor saw a $30 mil­lion up­front pay­ment to run the Phase III tri­al for CAP-1002 while leav­ing the door open for $705 mil­lion in po­ten­tial mile­stones.

The lat­est news out of Capri­cor has seemed to bring in­vestors some pos­i­tiv­i­ty as the com­pa­ny $CAPR has seen its stock rise 41% since the start of the year.

But Capri­cor has not been with­out set­backs. In 2018, the biotech not­ed that it had vol­un­tar­i­ly halt­ed a clin­i­cal tri­al of a stem cell ther­a­py the com­pa­ny pre­vi­ous­ly hoped would tamp down on the dam­ag­ing in­flam­ma­tion as­so­ci­at­ed with DMD. The halt was called fol­low­ing a “se­vere al­ler­gic re­ac­tion” that oc­curred dur­ing in­fu­sion.

And in 2019, the com­pa­ny said it would ex­plore strate­gic al­ter­na­tives for one or more of its prod­ucts and cut 21 jobs to keep fi­nan­cial­ly afloat, but had re­sumed dos­ing in its DMD tri­al.

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.