J&J turns its back on Capri­cor and its failed stem cell tech, tak­ing its mon­ey and rep with it

A lit­tle more than three years ago, J&J stepped up with a $12.5 mil­lion up­front to rent a front row seat on Capri­cor Ther­a­peu­tics’ $CAPR Phase II stem cell study, look­ing to see if their off-the-shelf ap­proach could mend a dam­aged heart. At the time, CEO Lin­da Mar­bán told me that it was well worth the year-long re­view ef­fort need­ed to bring the phar­ma gi­ant to the ta­ble, look­ing to roll ahead with $325 mil­lion in mile­stones.

Lin­da Mar­bán

“It says, OK, some­body very large and pow­er­ful is tak­ing a look at this tech­nol­o­gy and say­ing there’s some­thing there, and that’s the most ex­cit­ing thing for me,” she not­ed.

Thurs­day evening, two months af­ter the biotech not­ed that their 142-pa­tient study was head­ed to fail­ure, J&J said they weren’t in­ter­est­ed. They washed their hands of the part­ner­ship, hand­ed back full rights, and walked away from the deal.

Capri­cor shares dropped more than 10% Fri­day morn­ing.

Capri­cor is left with a stock that has been ham­mered down to pen­ny stock size, look­ing to ral­ly in­vestors around a plan to shift fo­cus to Duchenne mus­cu­lar dy­s­tro­phy with a plan to use their stem cell tech to tamp down on the in­flam­ma­tion that wreaks hav­oc on boys. LA-based Capri­cor — which ini­tial­ly set out to test the po­ten­tial of tech­nol­o­gy Lin­da Mar­bán’s hus­band, Ed­uar­do Mar­bán, de­vel­oped at Johns Hop­kins — not­ed in May that it would be cut­ting its work force.

Even three years ago, it was clear that the big play­ers had a wan­ing in­ter­est in stem cells. Once the sub­ject of in­tense spec­u­la­tion, stem cells and the com­pa­nies that de­vel­op them have been shunt­ed aside as re­peat­ed stud­ies fell far short of ex­pec­ta­tions — par­tic­u­lar­ly in heart re­pair. The Cal­i­for­nia In­sti­tute for Re­gen­er­a­tive Med­i­cine, which pro­vid­ed sig­nif­i­cant sup­port to Capri­cor, has been left de­fend­ing a long track record with lit­tle to show for it. There has been some re­newed hope by Bay­er, a few Japan­ese play­ers, and some oth­ers, though, that the tech­nol­o­gy has pro­gressed to the point where the field can start to pro­duce suc­cess­es.

The CEO now says they can get start­ed with a Duchenne study in the sec­ond half. And Mar­bán isn’t giv­ing up. In a pre­pared state­ment, she said:

Al­though Janssen’s de­ci­sion re­moves a po­ten­tial cor­po­rate part­ner for Capri­cor, this de­ci­sion al­so re­solves un­cer­tain­ty con­cern­ing the scope of the li­cense for CAP-1002 and pro­vides Capri­cor the free­dom to en­ter in­to new li­cens­ing and/or busi­ness de­vel­op­ment op­por­tu­ni­ties around this promis­ing ther­a­peu­tic can­di­date.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs covers a wide range of indications that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so.

The flu virus (CDC)

Roche tacks on an­oth­er Xofluza in­di­ca­tion as flu sea­son meets pan­dem­ic

Xofluza was heralded as the first new flu drug in 20 years when it got the FDA OK back in 2018. But even so, Roche saw tough competition from cheaper Tamiflu generics that appeared to be nearly as — if not just as — effective.

Now, the pharma says the drug also can be used to prevent influenza after exposure, snagging a new approval and adding to Xofluza’s appeal as flu season meets the pandemic.

A poll sug­gests vac­cine da­ta boost­ed Pfiz­er's pub­lic im­age, but oth­er da­ta point to long road ahead

For much of the pharmaceutical industry, the pandemic presented an opportunity: to prove their value to the world and turn public opinion around on a business much of the country had come to disdain.

That theory — that helping pull the country from a pandemic could neutralize years of anger over high drug prices — was put to its biggest test this month, as three different drugmakers announced data from their Covid-19 vaccines, offering the first major evidence that industry-built inoculations could turn the tide of the outbreak in the US.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Covid-19 roundup: Rus­sia prices vac­cine 't­wo or more times cheap­er' than mR­NA shots; Sino­vac PhI­II da­ta ex­pect­ed in ear­ly De­cem­ber

The world can now purchase its first registered vaccine — at less than $10 per dose.

RDIF, the Russian sovereign wealth fund and an avid backer of Sputnik V, said the vaccine will be available internationally starting from March 2021. A two-dose regimen of the adenovirus-based vaccine, which it has said is more than 90% effective, will cost less than $20.

And they are not shy about inserting themselves right into a rivalry with Western frontrunners, namely Pfizer/BioNTech and Moderna.

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