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.”

In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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UP­DAT­ED: Have a new drug that promis­es to fight Covid-19? The FDA promis­es fast ac­tion but some de­vel­op­ers aren't hap­py

After providing an emergency approval to use malaria drugs against coronavirus with little actual evidence of their efficacy or safety in that setting, the FDA has already proven that it has set aside the gold standard when it comes to the pandemic. And now regulators have spelled out a new approach to speeding development that promises immediate responses in no uncertain terms — promising a program offering the ultimate high-speed pathway to Covid-19 drug approvals.

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Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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The race to de­vel­op Covid-19 drugs and vac­cines is on — here’s what’s hap­pen­ing in the UK

Weeks away from the results of ongoing US and China trials testing its experimental antiviral remdesivir, Gilead is going to trial the failed Ebola drug in a small group of coronavirus patients in England and Scotland. The United Kingdom is also home to a range of other therapeutic efforts, as the pandemic rages on across the globe.

On Tuesday, Southampton, UK-based startup Synairgen kicked off a mid-stage placebo-controlled study testing its experimental drug, SNG001 — an inhaled formulation of interferon-beta-1a — that has previously shown to be safe and effective in improving lung function in asthma patients with a respiratory viral infection in a pair of Phase II trials.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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‘There was a grow­ing weari­ness’: Rush­ing against a pan­dem­ic clock, As­pen Neu­ro­sciences se­cures $70M Se­ries A

Just before Christmastime, Howard Federoff got a tip from Washington: There was a new virus in China. And this one could be bad.

News report of the virus had not yet appeared. Federoff, a neuroscientist, was briefed because years before, he was vetted as part of a group — he didn’t give a name for the group — to consult for the US government on emerging scientific issues. His day job, though, was CEO of Aspen Neurosciences, a Parkinson’s cell therapy startup that days before had come out of stealth mode and gave word to investors they were hoping to raise $70 million. That, Federoff realized, would be difficult if a pandemic shut down the global economy.

FDA puts pe­di­atric aGVHD drug on pri­or­i­ty re­view lane — will they go vir­tu­al with the ad­comm?

Despite worries about regulatory delays due to new work arrangements under Covid-19, the FDA appears intent to go full speed ahead with its everyday work, not only granting priority review to a stem cell therapy for acute graft versus host disease but also plotting an advisory committee meeting for it.

With a PDUFA date of September 30, the journey of the drug — remestemcel-L, or Ryoncil — could shed light on the agency’s capacity to facilitate drug development unrelated to Covid-19.

Covid-19 roundup: Trump push­es his new fa­vorite, untest­ed drug; CRISPR out­lines crip­pling im­pact of Covid-19

President Trump has a new favorite Covid-19 drug.

After a conversation with Japanese Prime Minister Shinzo Abe, Politico reports, the president is pressuring the FDA to issue emergency use authorization for favipiravir, a flu drug that showed glimpses of success in China but remains unproven and carries a list of worrying side effects. The push comes after a week-plus in which the White House touted a potentially effective but unproven malaria medication despite the concerns of scientific advisors such as NIAID director Anthony Fauci. And Trump ally Rudy Giuliani has been talking up unproven cell therapy efforts on Twitter.

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