No­var­tis-al­lied Oph­thotech blitzed af­ter a pair of PhI­II dis­as­ters for wet-AMD fran­chise drug

Opthotech CEO David R. Guy­er

Oph­thotech $OPHT had every­thing go­ing for it when it struck a $1 bil­lion-plus part­ner­ship with No­var­tis on its late-stage wet-AMD drug Fo­vista back in 2014. The an­ti-PDGF — de­signed to go hand-in-hand with an an­ti-VEGF like Lu­cen­tis — had suc­cess­ful­ly nav­i­gat­ed a pos­i­tive Phase IIb. And a suc­cess­ful IPO in 2013 added cash as in­vestors bought in­to its promise. No­var­tis it­self agreed to pay $330 mil­lion in up­front and near-term cash to get the deal done — a siz­able sum.

But the odds-on win­ner turned in­to a dis­ap­point­ing los­er this morn­ing as Oph­thotech and No­var­tis spelled out a se­ri­ous fail­ure in two late-stage stud­ies, send­ing its share price reel­ing. Its stock cratered in­stant­ly, plung­ing 82% in pre-mar­ket trad­ing.

In one of the Phase III stud­ies, the Fo­vista/Lu­cen­tis com­bo group ac­tu­al­ly did slight­ly worse on the pri­ma­ry vi­su­al acu­ity score than Lu­cen­tis alone. Com­bine the two Phase III stud­ies and there was on­ly a tiny, in­con­se­quen­tial gain in vi­su­al acu­ity.

The com­bi­na­tion ther­a­py al­so failed to dis­tin­guish it­self on pre-spec­i­fied sec­ondary end­points, com­plet­ing a rout for the part­ners.

The re­veal marks a bit­ter set­back for No­var­tis as well, which count­ed Fo­vista as one of its top late-stage ef­forts that would help the phar­ma gi­ant counter grow­ing gener­ic com­pe­ti­tion.

There’s still one more shot on goal for this drug. A Phase III com­bo study with Eylea is set to read out next year. But Re­gen­eron re­cent­ly ex­pe­ri­enced a mid-stage set­back on the same PDGF/VEGF ap­proach, and washed their hands of the whole thing af­ter re­peat­ed­ly dis­count­ing their chances of a suc­cess to an­a­lysts. An­oth­er fail­ure by Oph­thotech would like­ly ice the com­bo the­o­ry al­to­geth­er, leav­ing the biotech in the dust.

Re­gen­eron has shift­ed its gaze to Ang2 as the most like­ly bet on im­prov­ing on Eylea. “We now look to Eylea/Ang2 com­bi­na­tion to bol­ster the fran­chise,” notes Jef­feries’ Biren Amin, “for which the PII tri­al ONYX con­tin­ues to en­roll pts.”

“We are very dis­ap­point­ed in the re­sults from these tri­als, par­tic­u­lar­ly for pa­tients af­flict­ed with wet AMD,” com­ment­ed Opthotech CEO David R. Guy­er. “We are thank­ful to the pa­tients and clin­i­cal in­ves­ti­ga­tors and their staff for par­tic­i­pat­ing in the tri­als. We will con­tin­ue to an­a­lyze the da­ta from these two stud­ies to bet­ter un­der­stand the tri­al re­sults.”

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 commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Sanofi and GSK reach deal with Cana­da for 72 mil­lion vac­cine dos­es

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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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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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.

Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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