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.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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Ugur Sahin, AP Images

As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

Robert Spurr, President Salix Pharmaceuticals

Bausch Health’s Sal­ix pi­lots study to shine light on chron­ic liv­er dis­ease and push back on stereo­types

October is both breast cancer awareness and liver disease awareness month. While there’s no doubt which condition draws more attention during the month, Salix wants to change that.

Salix, Bausch Health’s gastroenterology arm, piloted its first chronic liver disease report and physician survey with results out this week aimed at raising awareness and dispelling stereotypes.

While 4.5 million people have chronic liver disease or cirrhosis – which is even more than 3.8 million women diagnosed with breast cancer – the research found chronic liver disease “has not received the attention or level of effort needed for adequate prevention, diagnosis, and standardization of its management.”

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Katie Fanning, Mozart Therapeutics CEO

Mozart Ther­a­peu­tics makes its of­fi­cial de­but, jump­ing in­to the hot Treg R&D field with some big-name in­vestors back­ing it

Treg cells have been getting more and more attention recently among autoimmune specialists. There’s been Jeff Bluestone’s Sonoma, the $157 million launch of GentiBio this summer and Egle Therapeutics — which launched just last week — to name a few.

Now, there’s a new Treg player jumping in that wants to distinguish itself in the market: Mozart Therapeutics. Today, the biotech is emerging from stealth in its official debut with a $55 million Series A — with a bunch of A-list Big Pharma names on board a syndicate led by ARCH.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

With San­doz con­tin­u­ing to drag on No­var­tis, Vas Narasimhan says he may fi­nal­ly be ready for a sale or spin­off

After years of rehab work aimed at getting Sandoz in fighting trim to compete in a market overshadowed by declining prices, CEO Vas Narasimhan took a big step toward possibly selling or spinning off the giant generic drug player.

The pharma giant flagged plans to launch a strategic review of the business in its Q3 update, noting that “options range from retaining the business to separation.”

Analysts have been poking and prodding Novartis execs for years now as Narasimhan attempted to remodel a business that has been a drag on its performance during most of his reign in the CEO suite. The former R&D chief has made it well known that he’s devoted to the innovative meds side of the business, where they see the greatest potential for growth.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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