Linda Marbán, Capricor CEO

Capri­cor hits pri­ma­ry end­point in Duchenne ex­ten­sion study, ready­ing its FDA pitch

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.

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.

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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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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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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Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

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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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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President Joe Biden signs the Democrats' landmark climate change and health care bill. From L-R: Sen. Joe Manchin (D-WV), Senate Majority Leader Chuck Schumer (D-NY), House Majority Whip James Clyburn (D-SC), Rep. Frank Pallone (D-NJ) and Rep. Kathy Castor (D-FL). (Susan Walsh/AP Images)

Pres­i­dent Biden signs ma­jor drug pric­ing re­forms in­to law: What's com­ing for bio­phar­ma?

President Joe Biden yesterday afternoon signed into law historic, decades-in-the-making new drug pricing reforms as part of a wider reconciliation bill that will likely take a chunk out of biopharma companies’ profits for some blockbusters just prior to generic or biosimilar competition.

The partisan bill (all Democrats in the House and Senate voted for it, and all Republicans voted against it) includes not only Medicare price negotiations — which won’t kick off until 2026, leaving ample time for a legal challenge — but mandatory inflation-related rebates, and a $2,000 annual cap on what seniors’ pay for their prescription drugs.

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Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

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.