Pearl Huang. Cygnal

'We're ripe': Cyg­nal draws the cur­tain on Flag­ship's lat­est bet on ex­oneur­al bi­ol­o­gy — and $65M in cash

No mat­ter how many times one’s heard Flag­ship Pi­o­neer­ing’s ideation process de­scribed, there al­ways seems to be an el­e­ment of evo­lu­tion­ary won­der: bold, new con­cepts that are “sev­er­al stan­dard de­vi­a­tions away from what is known,” put through a rig­or­ous vet­ting process first aimed at try­ing to kill the idea, and on­ly the fittest sur­vive.

Noubar Afeyan

That’s per­haps why Pearl Huang found its lat­est cre­ation, Cyg­nal Ther­a­peu­tics, and its fo­cus on the pe­riph­er­al ner­vous sys­tem “ir­re­sistibly at­trac­tive.” While Huang’s ap­point­ment as CEO back in Jan­u­ary was well-pub­li­cized, Cyg­nal is just spelling out the de­tails on its plat­form to­day, with $65 mil­lion — most­ly from Flag­ship — to boast.

Be­fore she de­cid­ed to jump from Roche to take on the role, Huang did her home­work on the field that Flag­ship is call­ing ex­oneur­al bi­ol­o­gy.

“When you look back through the lit­er­a­ture, for ex­am­ple, in can­cer bi­ol­o­gy, the pe­riph­er­al nerves were de­scribed to be a part of tu­mors in the late 1800s,” she said. “So the knowl­edge was al­ready out there.”

Avak Kah­jelian

But the gen­er­al pre­oc­cu­pa­tion with the cen­tral ner­vous sys­tem and the brain, as well as a lack of meth­ods to il­lu­mi­nate the roles at pe­riph­er­al nerves play in dis­eases, rel­e­gat­ed the PNS to a wiring di­a­gram in charge of ex­e­cut­ing or­ders in many sci­en­tists minds. It wasn’t un­til bet­ter imag­ing tech­niques came around in re­cent years that they could see just how ex­ten­sive that sys­tem is in the body and in mul­ti­ple dis­ease states. And it’s al­so what at­tract­ed Flag­ship’s Noubar Afeyan, Avak Kahve­jian and Jor­di Ma­ta Fink to launch the ven­ture.

“We see that the pe­riph­er­al ner­vous sys­tem it branch­es and goes as deeply in­to tis­sue as your vas­cu­lar sys­tem,” she said. And through Cyg­nal’s work, “we can see that non-neur­al cells and tis­sues in the dis­ease state are ac­tu­al­ly coopt­ing the lan­guage of the neu­rons. They are now ex­press­ing neur­al genes and ac­ti­vat­ing neur­al path­ways but they are non-neur­al in ori­gin them­selves.”

There are six com­po­nents in Cyg­nal’s ef­fort to de­code the role the PNS plays in dis­eases and how drug hunters can use it to their ad­van­tage:

  1. Neu­roimag­ing
  2. Cul­ture tech­nolo­gies to test re­duc­tion­ist neu­ro­bi­ol­o­gy ideas where “we grow dis­ease tis­sue / cell types in the pres­ence of pri­ma­ry neu­rons to dis­sect the sig­nal­ing”
  3. Chem­i­cal ge­net­ics
  4. Iden­ti­fy causal­i­ty for a group of 2,000 ge­net­ic tar­gets dubbed the “neu­rome,” via CRISPR-Cas9
  5. Bioin­for­mat­ics plat­form fo­cused on neur­al sig­nals and neur­al path­ways
  6. A neu­rophar­ma­copia with 1,000 mol­e­cules de­signed to treat CNS dis­or­ders
Jor­di Ma­ta Fink

“Right now in this place in time, we’re the on­ly peo­ple — the on­ly com­mer­cial or­ga­ni­za­tion on the plan­et that can take those six tech­nolo­gies, put them to­geth­er, throw one ques­tion in­to that plat­form, get six dif­fer­ent an­swers and then con­nect the dots be­tween those an­swers to get nov­el in­sights in­to bi­ol­o­gy,” Huang said.

That will, in turn, lead to the dis­cov­ery of new small and large mol­e­cules — ad­mit­ted­ly tra­di­tion­al modal­i­ties that Cyg­nal be­lieves will do the job. With 41 staffers on hand (and a plan to ex­pand the of­fice to ac­com­mo­date 80), the goal is to nom­i­nate two pro­grams for de­vel­op­ment this year, most like­ly for can­cer and in­flam­ma­tion. But down the line, Huang can see plug­ging in any dis­ease from fi­bro­sis and en­dometrio­sis to wound heal­ing and obe­si­ty in­to their plat­form and yield­ing new drugs.

So why come out of stealth mode now?

“It’s kind of over­due, don’t you think?” Huang said. “It’s just time. We’re ripe.”

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RBC Capital Markets explores what is material in ESG for biopharma companies with the ESG leads at Gilead Sciences. Gilead has long focused on sustainability but recognized a more robust framework was needed. Based on a materiality assessment, Gilead’s ESG strategy today focuses first on drug access and pricing, while also addressing D&I and climate change. Find out why Gilead’s board is “acutely aware” of the contribution that ESG makes to firm’s overall success.

What con­tro­ver­sy? Eli Lil­ly plots Alzheimer's BLA fil­ing lat­er this year as FDA taps more an­ti-amy­loid drugs as break­throughs

The FDA is keeping the good news coming for Alzheimer’s drug developers. And Eli Lilly is taking them up on it.

Amid continued controversy around whether Biogen’s new flagship drug, Aduhelm, should have been approved at all — and swelling, heated debates surrounding its $56,000 price tag — the agency had no issue handing them and their Japanese partner Eisai a breakthrough therapy designation for a second anti-amyloid beta antibody, lecanemab, late Wednesday.

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James Peyer, Cambrian

Can a cell ther­a­py treat mus­cu­lar dy­s­tro­phy? A Ger­man bil­lion­aire's an­ti-ag­ing start­up is try­ing to find out

Gene therapy companies have faced huge hurdles trying to deliver healthy genes into muscular dystrophy patients’ muscle cells, so here’s an idea: Why don’t we just replace the muscle cells themselves?

Over the last two years, Vita Therapeutics has been exploring that possibility, building on early stem cell work from Johns Hopkins professor Peter Andersen. And on Tuesday they announced a $32 million Series A to begin to move their first therapy into the clinic, where they hope it will help rebuild muscle in patients with a type of dystrophy that afflicts the arms and legs.

Hervé Hoppenot, Incyte CEO (Jeff Rumans)

ODAC echoes FDA con­cern over In­cyte PD-1, as Paz­dur sig­nals broad­er shift for ac­cel­er­at­ed ap­proval

After the FDA lambasted their PD-1 ahead of an adcomm earlier this week, Incyte ran into new trouble Thursday as ODAC panelists voted against an accelerated OK by a wide margin.

Members of the Oncologic Drugs Advisory Committee recommended with a 13-4 vote to defer a regulatory decision on Incyte’s retifanlimab until after more data can be collected from a placebo-controlled trial. The PD-1 therapy is due for a PDUFA date in late July after receiving priority review earlier this year.

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In a lengthy review document and a pair of memos from top officials, the FDA released on Tuesday night its most detailed argument yet for approving Biogen’s intensely controversial Alzheimer’s drug aducanumab.

The documents amount to an agency attempt to quench the firestorm their decision kindled, as outside advisors members resigned and experts warned that an unproven drug now could stretch Medicare’s budget to a breaking point. Ultimately, the documents show how CDER director Patrizia Cavazzoni and Office of New Drugs director Peter Stein both concurred with FDA neuroscience head Billy Dunn on the accelerated approval while the staff at FDA’s Office of Biostatistics did not think an approval was warranted.

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From left: Rajul Jain, Stefan Vitorovic, Arjun Goyal, Arie Belldegrun, Jean-Philippe (JP) Kouakou-Zebouah, Helen Kim

Arie Bellde­grun's Vi­da Ven­tures goes back to the well with $825M mega­fund and its eyes set on more in­no­v­a­tive meds

Among the list of bright names in biopharma, few shine brighter than Kite founder and serial entrepreneur Arie Belldegrun, who has rattled off a remarkable run of success in recent years. Now, a Belldegrun investment team is locking up a massive third fund to keep chasing the cutting edge in therapeutics.

Vida Ventures closed its third investment fund at a whopping $825 million — its largest yet — as the ever-expanding VC firm hits 30 companies in its portfolio developing new routes to hard-to-treat diseases, the company said Thursday.

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Karen Flynn, Catalent

Q&A: When the pan­dem­ic struck, Catal­en­t's CCO had just joined the team

Karen Flynn came aboard Catalent’s team just in time.

The company was going through a surge of changes, and she had been brought over from her role as CCO of West Pharmaceutical Services to serve in the same capacity for the New Jersey-based CDMO. Then a few months later, the pandemic was in full-force.

Since then, Catalent’s been in hyper-expansion mode. In early May, it acquired Promethera’s Hepatic Cell Therapy Support SA subsidiary and its 32,40-square-foot facility in Gosselies, Belgium. Prior to that, the company acquired Belgian CDMO Delphi Genetics, wrapped up the expansion of an already-existing site in Madison, WI and added an ultra-low temperature freezer partner in Sterling. As Emergent has botched millions of doses of AstraZeneca’s vaccine, the company has swooped in to move that production to its Maryland plant as well.

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Richard Pazdur (vis AACR)

FDA en­cour­ages in­clud­ing in­cur­able can­cer pa­tients in tri­als, re­gard­less of pri­or ther­a­pies

The FDA on Thursday called to include those with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in appropriate clinical trials, regardless of whether they have received existing alternative treatments.

Historically, many cancer clinical trials have required that participating patients previously received multiple therapies, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence.

On heels of Aduhelm ap­proval, Bris­tol My­ers jumps back in­to Alzheimer's race

Bristol Myers Squibb last put major resources behind an Alzheimer’s drug nearly a decade ago, when their own attempt at targeting amyloid flamed out in mid-stage studies. They invented another molecule, a Tau-targeted antibody, but jettisoned it to Biogen in 2017 as they dropped out of neuroscience altogether.

But on Thursday, the New York pharma announced they were getting back in the game. Bristol Myers exercised an $80 million option to bring a tau-targeted antibody from Prothena into a Phase I study. The opt-in, which Bristol Myers triggered ahead of analyst expectations, opens the door for another $1.7 billion in milestones down the road.

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