Keiichi Fukuda, Heartseed CEO

Fresh off $598M deal with No­vo Nordisk, a Japan­ese stem cell com­pa­ny is on its way to the clin­ic with a dif­fer­ent ap­proach to treat­ing heart fail­ure

A com­mon ap­proach to treat­ing heart fail­ure with in­duced pluripo­tent stem cells in­volves graft­ing sheets of cells on­to the sur­face of the heart to im­prove vas­cu­lar­iza­tion and blood flow. It’s the eas­i­est method of trans­plan­ta­tion — but you run the risk of not mak­ing an elec­tri­cal con­nec­tion with the heart and the cells not syn­chro­niz­ing with the pa­tient’s heart mus­cle.

So what if you could in­ject spher­i­cal clus­ters of heart cells di­rect­ly in­to the heart mus­cle wall? For Heart­seed, that’s now the $37 mil­lion ques­tion.

The Tokyo-based biotech un­veiled a mod­est Se­ries C round on Fri­day morn­ing, bring­ing its to­tal raise to about $75 mil­lion. The news comes just a week and a half af­ter No­vo Nordisk struck an up to $598 mil­lion deal for ex­clu­sive rights to de­vel­op, man­u­fac­ture and com­mer­cial­ize the com­pa­ny’s lead can­di­date HS-001 world­wide, ex­cept in Japan where Heart­seed is keep­ing the de­vel­op­ment rights.

Heart­seed is run by Kei­ichi Fuku­da, a pro­fes­sor in Keio Uni­ver­si­ty’s de­part­ment of re­gen­er­a­tive med­i­cine and ad­vanced car­diac ther­a­peu­tics and a long­time re­searcher. It’s named af­ter a species of house­plant which re­sem­bles the spher­i­cal mi­cro­tis­sue of heart cells the team cre­ates in the lab.

HS-001 con­sists of donor iP­SC-de­rived car­diomy­ocyte spher­oids. Pluripo­tent es­sen­tial­ly means the cell can be dif­fer­en­ti­at­ed in­to any cell you want. And in Heart­seed’s case, it’s car­diomy­ocytes — the cells re­spon­si­ble for gen­er­at­ing con­trac­tile force in the heart.

The biotech in­jects the car­diomy­ocyte spher­oids di­rect­ly in­to the my­ocardi­um, where they grow and elec­tri­cal­ly cou­ple with oth­er heart cells.

Back in March, the com­pa­ny got the OK from Japan’s Phar­ma­ceu­ti­cals and Med­ical De­vices Agency to be­gin a Phase I/II tri­al, dubbed LAPiS, in se­vere heart fail­ure as­so­ci­at­ed with is­chemic heart dis­ease. The tri­al is ex­pect­ed to be­gin in the sec­ond half of this year.

The No­vo deal in­cludes $55 mil­lion in up­front and near-term mile­stone pay­ments, and if the drug gets ap­proved, Heart­seed is el­i­gi­ble for “sin­gle-dig­it to low dou­ble-dig­it” roy­al­ties on an­nu­al sales out­side Japan. No­vo Nordisk has the rights to co-com­mer­cial­ize the drug in Japan, where it would split the prof­its and cost 50/50 with Heart­seed.

“We be­lieve that the part­ner­ship with No­vo Nordisk is very valu­able as we seek to dis­sem­i­nate our Japan-ori­gin in­no­va­tion glob­al­ly as ear­ly as pos­si­ble,” Fuku­da said in a state­ment at the time.

The news comes about a year and a half af­ter Heart­seed pulled in a $26 mil­lion Se­ries B round. A slate of new and old in­vestors par­tic­i­pat­ed in the Se­ries C, in­clud­ing UTokyo In­no­va­tion Plat­form Co., Med­ical In­cu­ba­tor Japan, Keio In­no­va­tion Ini­tia­tive, Sum­it­o­mo Mit­sui Trust In­vest­ment, SBI Group, Nis­say Cap­i­tal, SM­BC Cap­i­tal, Medi­pal Hold­ings, and Itochu Chem­i­cal Fron­tier.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

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By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

The IPO 4-1-1: Four fil­ings, a pric­ing and a with­draw­al head­line this week's Nas­daq ac­tion as raise ap­proach­es $7.5B

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

Another week, another horde of biotechs is doing the Nasdaq dance.

This week saw four companies file their SEC paperwork ahead of expected debuts, another hit Nasdaq on Friday and a sixth formally withdrew its bid to go public. Aerovate Therapeutics, Ocean Biomedical and Acumen Pharmaceuticals all penciled in initial raises of $100 million, while Dermata Therapeutics is estimating a modest $18 million raise.

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Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)

An­a­lysts hound Bio­gen with ques­tions over Aduhelm's $56K price point as news of Alzheimer's OK rip­ples through glob­al mar­kets

It’s been nearly 24 hours since the FDA handed down its controversial decision to approve Biogen’s Aduhelm drug for Alzheimer’s disease, and reactions from around the industry are pouring in as analysts try to size up the ramifications of Monday’s news.

Biogen hosted an investor call Tuesday morning, providing a forum for a number of topics that have been top of mind. The call came as the news of Aduhelm’s approval in the US rippled through global markets, while $BIIB stock soared to around $380 apiece (up from $286 on Friday), as Biogen’s partners at Eisai seek new approvals in Asia.

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Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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