In lat­est set­back for stem cell sec­tor, Celyad fails PhI­II— and the biotech claims a vic­to­ry

At first glance, the idea that stem cells can be used to strength­en a dam­aged heart has a po­tent at­trac­tion. It sounds sen­si­ble enough in the panoply of re­gen­er­a­tive med­i­cine. But Celyad’s pro­gram to prove that it has the right ap­proach with Cure-C just failed in Phase III, of­fer­ing a new set­back to a trou­bled field and a body blow to the biotech com­pa­ny.

For Celyad’s ($CYAD) part, the fact that the Phase III failed was dis­pensed with quick­ly so the com­pa­ny could im­me­di­ate­ly move on to its an­a­lyt­i­cal break­down, point­ing to a sig­nif­i­cant sub­pop­u­la­tion of pa­tients the com­pa­ny in­sists were helped by C-Cure. That analy­sis is giv­ing them grounds to ap­proach the EMA on fil­ing the drug while flag­ging their ea­ger­ness to do a deal on the project, look­ing for an­oth­er com­pa­ny to fund de­vel­op­ment and back a com­mer­cial­iza­tion move.

That will be a hard ar­gu­ment to make with in­vestors. Heart dis­ease in spe­cif­ic – which af­fects mil­lions of peo­ple – is one of the most de­mand­ing fields in R&D, with a very high bar for prov­ing ef­fi­ca­cy and safe­ty to reg­u­la­tors. And in­vestors ini­tial re­sponse was to flee, send­ing Celyad’s shares down about 40% this morn­ing.

When you want to dress up a Phase III fail­ure, the first thing you point to is a “pos­i­tive trend” across the pa­tients treat­ed. Celyad didn’t miss that point. In­ves­ti­ga­tors then ze­roed in on a group of pa­tients “cat­e­go­rized by their End Di­as­tolic Vol­ume (EDV) at in­clu­sion, sig­nif­i­cance was met for the pri­ma­ry end­point (p = 0.015). Most im­por­tant­ly, in this sub­group, a strong trend or a sta­tis­ti­cal sig­nif­i­cant pos­i­tive dif­fer­ence was seen in all in­di­vid­ual el­e­ments of the com­pos­ite pri­ma­ry end­point (Mor­tal­i­ty, Wors­en­ing Heart Fail­ure Events, Qual­i­ty of Life, 6 min­utes Walk­ing Test, End Sys­tolic Vol­ume and Ejec­tion Frac­tion).”

There was no break­down on which the­o­ret­i­cal end­points were hit by the sub­pop­u­la­tion.

Phase III stud­ies, though, aren’t de­signed to iden­ti­fy pa­tient pop­u­la­tions. They’re in­tend­ed to pro­vide con­clu­sive ev­i­dence that a treat­ment is ei­ther ef­fec­tive or safe, or it isn’t.

The Phase III fail­ure fol­lows a de­ci­sion by Te­va just days ago to with­draw from its stem cell pact with Mesoblast, which bad­ly dam­aged the biotech’s share price. And days be­fore that Bio­Car­dia was forced to pull its $50 mil­lion IPO – de­signed to back its own late-stage stem cell treat­ment for heart dis­ease – af­ter it failed to gain enough sup­port for the move.

Celyad – which changed its name in an ef­fort to high­light a new fo­cus on the trendy CAR-T field – sus­pend­ed trad­ing for its shares un­til af­ter a morn­ing press con­fer­ence.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Hal Barron, GSK R&D chief (Endpoints News)

Hal Bar­ron gam­bles $625M cash on high-wire TIG­IT act, throw­ing Glax­o­SmithK­line in­to heat­ed race and com­plet­ing next-gen I/O trin­i­ty

Count Hal Barron and GlaxoSmithKline in for the TIGIT fight.

The stakes are as high as the risks: While a growing pack of Big Pharma rivals is lending credence to the hypothesis that TIGIT will be the next big immune checkpoint and cancer drug target, the first clinical trials have shown response rates that can be described as modest at best. But Barron’s bet is on the whole “axis” that the receptor sits on, with an eye on testing its new anti-TIGIT antibody not just in combo with PD-1 but also in triplets.

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As Covid-19 shifts the world's at­ten­tion to biotech, Noubar Afeyan's Flag­ship builds $3.4B fund to fu­el new in­ven­tions. Here's the plan

A little more than a year ago, Flagship Pioneering rolled out a monster fund with $1.1 billion in it to bankroll the platform companies they were creating inside their own labs. But it turns out, that was just the prelude to a much, much larger raise, as both current investors — who’ve been reaping the rewards of some booming biotech stocks — join in with new investors betting on more in the years to come.

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Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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Geoffrey Porges, SVB Leerink

Top an­a­lyst maps a rocky road for Ver­tex — un­less they adopt a $10B M&A cam­paign to save the pipeline

After repeatedly poking Vertex $VRTX execs with pointed criticism of their R&D strategy, top SVB Leerink analyst Geoffrey Porges is now turning up the heat to a full-scale roasting.

In a note out early Monday morning, Porges spotlights the impact of Vertex’s recent follow-up failure on AATD — their treatment hit the endpoint but missed on commercial prospects — rewriting his recommended scenario for CEO Reshma Kewalramani as she grapples with the setback.

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Stephen Hahn (AP Images)

UP­DAT­ED: Ex-FDA com­mish Stephen Hahn joins Flag­ship, a ven­ture group that spawned Covid-19 vac­cine mak­er Mod­er­na

That revolving door between the FDA and industry is spinning even faster than usual.

Former FDA commissioner Stephen Hahn is joining Flagship Pioneering, the venture outfit that founded Moderna — which raced its way to an FDA EUA for a Covid-19 vaccine that is making billions of dollars– as the new CMO of its Preemptive Medicine and Health Security initiative.

Flagship confirmed the hire — first reported in The Washington Post — in a statement out late Monday.

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Patrizia Cavazzoni, CDER

FDA’s Cavaz­zoni calls for ad­comms to ‘get back to the sub­stance’

While her comments were recorded prior to the FDA’s recent approval of Biogen’s controversial Alzheimer’s drug, CDER Director Patrizia Cavazzoni presciently called for substantiating reforms to the advisory committee process at the agency.

Short on examples, Cavazzoni said at a BIO event aired on Monday that some recent adcomms show “how they can be swayed by emotion in the face of hard facts,” but they need to “get back to the fundamentals, which is listening to thoughtful input from experts in response to thoughtful questions that we ask them.”

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Terns Phar­ma­ceu­ti­cals touts safe­ty da­ta from an ex-Eli Lil­ly can­di­date in the hunt for NASH treat­ment

While many others have tried — and failed — to get a NASH candidate across the finish line, Terns Pharmaceuticals thinks its FXR agonist will eventually earn its wings without the safety issues that have slowed others down. Now, a mid-stage safety readout could help add some validity to those hopes.

No patients in the Phase IIa LIFT study discontinued TERN-101 due to side effects, CMO Erin Quirk said during a call with investors on Monday morning. That includes pruritus, an uncomfortable itching sensation that frequently leads patients to drop out of other FXR agonist studies.

Paul Burton, incoming Moderna CMO (J&J/file photo)

Look­ing be­yond the pan­dem­ic, Mod­er­na ap­points J&J vet­er­an Paul Bur­ton as new CMO

Moderna is turning to one of its Covid-19 vaccine competitors to fill its open CMO slot, but this time, it’s not the vaccine experience they’re after.

Paul Burton, who’s spent 16 years at J&J, most recently as chief global medical affairs officer of Janssen, will take over as Moderna CMO on July 6.

With an eye toward a future beyond the pandemic, the mRNA biotech went with Burton, who earned his MD and PhD degrees in London, as he offers experience on a range of therapeutic areas, as well as work as a cardiothoracic surgeon and leading tech projects with Apple.

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