Gideon Bollag, Opna Bio CEO

Plexxikon vets kick off new drug dis­cov­ery out­fit — with old as­sets and new tar­get

Dai­ichi Sankyo shut down its South San Fran­cis­co sub­sidiary Plexxikon ear­li­er this year. Now a new start­up is look­ing to forge its own path — with 10 Plexxikon vet­er­ans, five as­sets from the now-de­funct biotech and a fo­cus around a new tar­get.

Op­na Bio, led by ex-Plexxikon CEO Gideon Bol­lag, an­nounced its emer­gence from stealth this morn­ing — com­bined with a $38 mil­lion Se­ries A round.

Plexxikon, bought out by Dai­ichi in 2011 for $935 mil­lion, was shut down ear­li­er this year as the Japan­ese phar­ma planned to stream­line ef­forts to fur­ther R&D in­to En­her­tu and two oth­er AD­Cs. The com­pa­ny, which was found­ed more than two decades ago, pushed two can­cer ther­a­pies all the way past the FDA: the Roche-part­nered Zelb­o­raf for melanoma, and Tu­ralio for tenosyn­ovial gi­ant cell tu­mors in 2019.

Dou­glas Hana­han

Op­na Bio, ac­cord­ing to Bol­lag, was found­ed back in 2019 — and there is more con­nec­tion to Bol­lag’s pre­vi­ous com­pa­ny, Onyx Phar­ma­ceu­ti­cals, than Plexxikon. Dou­glas Hana­han, one of Op­na’s co-founders and head of the biotech’s sci­en­tif­ic ad­vi­so­ry board, knew Bol­lag from his time at Onyx.

“And re­al­ly in­de­pen­dent­ly of Plexxikon, at the very be­gin­ning of the pan­dem­ic, when I was still CEO at Plexxikon, Doug called and had this news sto­ry and asked if I would be in­ter­est­ed in it, and looked in­to it. It was very ex­cit­ing,” Bol­lag said.

What that sto­ry was — and what dis­tin­guish­es Op­na from oth­er biotechs — is a pro­tein called frag­ile X men­tal re­tar­da­tion pro­tein, or FM­RP, and us­ing it in on­col­o­gy drug de­vel­op­ment. Hana­han and his lab pub­lished a pa­per last week in Sci­ence about the pro­tein be­ing ex­pressed in can­cer and they’re hy­poth­e­siz­ing that it is in­volved in help­ing tu­mor cells evade the im­mune sys­tem.

The pro­tein, en­cod­ed by the FMR1 gene, has nor­mal­ly been as­so­ci­at­ed with neu­rons, Bol­lag tells End­points News. How­ev­er, as Hana­han and lab point­ed out, in­creased lev­els of FM­RP were not­ed in cer­tain can­cers, in­clud­ing pan­cre­at­ic, colon, breast, prostate and lung can­cer.

That dis­cov­ery led Hana­han’s lab to try to find out why can­cer cells are ex­press­ing it since nor­mal­ly they should not be able to, Bol­lag point­ed out. And as it turns out, FM­RP is a mas­ter reg­u­la­tor of trans­la­tion and mR­NA sta­bil­i­ty, and that plays a role in can­cer evad­ing de­tec­tion by the im­mune sys­tem. Ad­di­tion­al­ly, that might lend a hand to the treat­ment of cer­tain can­cers that are im­mune to check­point in­hibitors.

Bol­lag added that the whole idea is that if that pro­tein can be blocked by a small mol­e­cule, then the host im­mune sys­tem should be much more able to go af­ter and de­stroy the tu­mor.

So far, can­di­date screen­ing and se­lec­tion are still on­go­ing.

On top of that ef­fort, Op­na Bio said it ac­quired five on­col­o­gy can­di­dates from Plexxikon — in­clud­ing two clin­i­cal stage pro­grams. One, BET in­hibitor OPN-2853, is in a Phase I/II tri­al for myelofi­bro­sis, and the oth­er (OPN-7486) is a CSF1 in­hibitor planned to start Phase II some­time next year. The re­main­ing three can­di­dates are still in pre­clin­i­cal de­vel­op­ment.

In the mean­time, the 15-per­son biotech says it has a goal to find a part­ner, and that emerg­ing out of stealth will en­able the com­pa­ny to have more dis­cus­sions with more pos­si­ble in­ter­est­ed par­ties. The Se­ries A will last Op­na well in­to 2024.

In­vestors in that round in­clud­ed Lon­gi­tude Cap­i­tal, North­pond Ven­tures and Men­lo Ven­tures.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Tim Pearson, Carrick Therapeutics CEO

Pfiz­er backs $60M in­fu­sion in­to Car­rick, teams up on breast can­cer treat­ment

In a big week for Carrick Therapeutics, the company announced $60 million in funding for its lead breast cancer drug and development of a second program, as well as a collaboration with Pfizer for combo development.

The $35 million from Pfizer comes with an agreement under which Pfizer will support Carrick’s Phase II study of samuraciclib in combination with Pfizer’s Faslodex for advanced breast cancer. Along with the investment, Adam Schayowitz, vice president and development head of breast cancer, colorectal cancer and melanoma at Pfizer global product development, will join Carrick’s scientific advisory board.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls



Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Thomas Gad, Y-mAbs Therapeutics founder and interim CEO

FDA re­jects Y-mAbs’ neu­rob­las­toma drug af­ter tak­ing is­sue with clin­i­cal tri­al de­sign

Uncertainty about clinical trial evidence has led the FDA to hand down a complete response letter for Y-mAbs’ neuroblastoma drug, casting a cloud on the future of a candidate that had gone through a long development journey in a rare pediatric cancer.

Y-mAbs said it’s disappointed “but not surprised” given that the agency’s oncology drug advisory committee had voted 16-0 against its drug’s approval a few weeks ago.

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Philip Tagari switch­es Am­gen's dis­cov­ery lab for in­sitro's ma­chine learn­ing tools; CEO Joaquin Du­a­to to chair J&J's board

In February, Philip Tagari will take a few days of retirement and then immediately return to industry. He won’t be leading the therapeutics discovery unit for a large biopharma, though.

He’ll trade in his Amgen hat for chief scientist at a machine learning startup that has reeled in hundreds of millions in capital to lay the groundwork for a much-hyped new model of drug discovery that aims to speed up the time to new clinical assets.

Raul Rodriguez, Rigel Pharma CEO

Rigel Phar­ma scores FDA ap­proval for leukemia, kick­ing off show­down with Servi­er in IDH1

When Rigel Pharma bought olutasidenib from Forma Therapeutics, it acquired a drug that already secured a PDUFA date at the FDA — for February 2023. But regulators are ready to give their OK sooner than that.

The FDA has approved the IDH1 inhibitor as a treatment for adult patients with relapsed or refractory acute myeloid leukemia who have a susceptible IDH-1 (isocitrate dehydrogenase-1) mutation as detected by an FDA-greenlit test. Rigel will market it as Rezlidhia.

Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with Rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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Uğur Şahin, BioNTech CEO (ddp images/Sipa USA/Sipa via AP Images)

BioN­Tech bets on dif­fi­cult STING field via small mol­e­cule pact with a Pol­ish biotech

BioNTech is beefing up its relatively thin small molecule pipeline by adding weight to a clinically difficult corner of oncology R&D: STING agonists. To do so, BioNTech is teaming up with a 15-year-old Polish biotech and doling out €40 million, about $41.5 million, to start.

The deal is broken into two parts: First, BioNTech obtains an exclusive global license to develop and market Ryvu Therapeutics’ STING agonist portfolio as small molecules, whether alone or in combination with other agents.

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