Theresa Heah, Kevin Slawin, Chris Garabedian (AsclepiX)

Bet­ter than Eylea, as durable as gene ther­a­py? Per­cep­tive, Xon­toge­ny boost oph­thalmic star­tup's shot for best of all worlds

What’s the biggest break­through in the oph­thal­mol­o­gy space since Lu­cen­tis and Eylea?

No­var­tis’ Beovu, launched last No­vem­ber for wet age-re­lat­ed mac­u­lar de­gen­er­a­tion, has been ham­pered by safe­ty is­sues. Ko­di­ak Sci­ences sports $2.74 bil­lion on the promise of an an­ti­body biopoly­mer con­ju­gate that can be dosed every 16 weeks — re­duc­ing the treat­ment bur­den. Then there’s the line of gene ther­a­py play­ers from re­genxbio to Ad­verum, fol­low­ing in the foot­steps of Spark Ther­a­peu­tics.

But As­cle­piX be­lieves it has an even bet­ter an­swer.

Johns Hop­kins pro­fes­sors Alek­sander Popel and Jor­dan Green found­ed the start­up af­ter de­sign­ing and run­ning an ar­ti­fi­cial in­tel­li­gence al­go­rithm to mine the hu­man pro­teome for any se­quences that seem an­ti-an­gio­genic. They got a hit in the non-cod­ing se­quences for col­la­gen 4: a 20mer pep­tide they named AXT107, which ex­hib­it­ed an­ti-VEGF prop­er­ties like the com­mon eye drugs.

It stood out for two oth­er rea­sons. It al­so ac­ti­vat­ed the an­giopoi­etin re­cep­tor Tie2, and when in­ject­ed in­to the vit­re­ous, it turns in­to a slow-re­lease gel with a half life of 5 to 7 months.

That could mean once-a-year or even less fre­quent dos­ing. Since then, the small and scat­tered team at As­cle­piX has con­firmed the drug’s ef­fect in turn­ing off an­gio­gen­e­sis through in­te­grins in 10 an­i­mal mod­els and found no side ef­fects.

“This was one of the most ro­bust pre­clin­i­cal datasets we saw across ther­a­peu­tic ar­eas and they were well done, and they were ref­er­ence­able, mean­ing that oth­ers use sim­i­lar mod­els to ac­cess the ef­fi­ca­cy and safe­ty of their com­pounds,” said Chris Garabe­di­an, whose Per­cep­tive Xon­toge­ny Fund is lead­ing a $35 mil­lion Se­ries A.

AXT107 owes much of its de­sir­able prop­er­ties to the fact that it’s a nat­u­ral­ly oc­cur­ring pep­tide that’s “evolved over mil­lions of years” as part of the home­osta­sis mech­a­nism, added Kevin Slaw­in, an ear­ly in­vestor and in­ter­im CEO.

“This is not a drug where 10 phar­ma­ceu­ti­cal em­ploy­ees went in­to a lab and had or­ders to drug a cer­tain tar­get, and went ahead and tried and then came up with some­thing that of­ten had side ef­fects like in­flam­ma­tion,” he said.

As­cle­piX is work­ing with a con­tract man­u­fac­tur­er in Italy — one of three in the world equipped with the pep­tide pro­duc­tion and fil­tra­tion ca­pac­i­ty through com­mer­cial stage — to fi­nal­ize the batch of drug for the up­com­ing trio of Phase I/IIa stud­ies. The process is straight­for­ward enough that Slaw­in doesn’t en­vi­sion any CMC is­sues.

The first-in-hu­man tri­als will be­gin lat­er this year, si­mul­ta­ne­ous­ly test­ing in three pa­tient pop­u­la­tions: di­a­bet­ic mac­u­lar ede­ma (DME), wet age-re­lat­ed mac­u­lar de­gen­er­a­tion (wet AMD) and mac­u­lar ede­ma sec­ondary to reti­nal vein oc­clu­sion (RVO).

“So next year we will have proof of con­cept, or at least a sig­nal on the three in­di­ca­tions,” CMO There­sa Heah said. “These are 3 very large in­di­ca­tions from a glob­al mar­ket size per­spec­tive. We are talk­ing 10 bil­lion dol­lar-plus-plus-plus right now. If we are able to demon­strate once-a-year in­jec­tion, we will be en­ter­ing an­oth­er larg­er in­di­ca­tion that’s not been touched by any­body else, which is di­a­bet­ic retinopa­thy.”

Steve Altschuler

Pos­i­tive da­ta there could open up count­less doors — strate­gic part­ner­ships, a big­ger fi­nanc­ing, IPO, you name it. Es­pe­cial­ly with Per­cep­tive’s main funds on the syn­di­cate along­side Slaw­in’s Rapha Cap­i­tal Man­age­ment and Cel­gene co-founder Sol Bar­er’s Bar­er & Son Cap­i­tal.

“We have a very safe prod­uct,” Slaw­in added. “It’s still in­trav­it­re­al, we don’t need sub­reti­nal in­jec­tions or any­thing like that, yet we have the longevi­ty to match any kind of gene ther­a­py.”

Heah, an oph­thal­mol­o­gist by train­ing and bio­phar­ma vet who helped Bay­er launch Eylea out­side the US, brought both her own sub­ject ex­per­tise and a group of sea­soned drug de­vel­op­ers to As­cle­piX. From her perch in New Jer­sey, she man­ages two oth­er staffers in the area, two in Bal­ti­more and an­oth­er two in Cal­i­for­nia. Covid-19 has changed lit­tle.

“I’m used to run­ning teams in Japan, Aus­tralia, Eu­rope, US East Coast, West Coast,” she said. “In fact we re­al­ized we got more done with­out the trav­els.”

Slaw­in, who spoke to End­points News just be­fore a flight from Hous­ton to Flori­da, might be the ex­cep­tion. De­pend­ing on how the CEO search goes, he might con­tin­ue lead­ing the com­pa­ny or re­treat back to a board now led by Spark co-founder Steve Altschuler, along­side Garabe­di­an and Ben Askew, an R&D part­ner at Xon­toge­ny.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA’s work­load buck­les un­der the strain, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

David Berry (Flagship)

Flag­ship's next big tech­no­log­i­cal bet? The cloud

Earlier this month, Flagship announced their big bet on the software half the industry is talking about, launching the AI and machine learning startup. Now, they and a couple other investors are gambling $100 million on a software that much of the public generally thinks of as a cool, IT afterthought: cloud computing.

The idea, says founder and Flagship partner David Berry, is one of scale: The sheer magnitude of biological data that you can store on cloud technology is unprecedented. And that size, when leveraged properly, can allow you to ask questions and form insights that are similarly unprecedented.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.