Oc­u­lar Ther­a­peu­tix's last-minute gam­bit fails to sal­vage its sec­ond FDA ap­pli­ca­tion, shares plunge

An 11th hour bid by Oc­u­lar Ther­a­peu­tix to sal­vage its FDA ap­pli­ca­tion for their drug/de­vice eye treat­ment Dex­ten­za got shot down in a hur­ry on Tues­day.

A year af­ter Oc­u­lar re­ceived its first re­jec­tion of the eye ther­a­py based on man­u­fac­tur­ing is­sues, the Bed­ford, MA-based com­pa­ny was hand­ed its sec­ond com­plete re­sponse let­ter af­ter in­spec­tors flagged is­sues dur­ing a pre-NDA in­spec­tion a cou­ple of months ago.

Oc­u­lar shares $OCUL took a nose dive on the news, drop­ping more than 30%.

Oc­u­lar ex­ecs tried to win a re­prieve by fil­ing a re­sponse to the FDA’s man­u­fac­tur­ing con­cerns on Mon­day, the day ahead of the PDU­FA date. They want­ed reg­u­la­tors to flag the re­sponse as a ma­jor amend­ment to their ap­pli­ca­tion and ex­tend the dead­line, but the FDA stiff-armed the biotech with­out even tak­ing a look — ac­cord­ing to Oc­u­lar’s ver­sion of events.

Amar Sawh­ney

Dex­ten­za has had a mixed Phase III record in the clin­ic, suc­ceed­ing with its hy­dro­gel plug in de­liv­er­ing dex­am­etha­sone over an ex­tend­ed pe­ri­od in re­duc­ing post-sur­gi­cal pain, but fail­ing at sig­nif­i­cant­ly re­duc­ing oc­u­lar itch­ing for al­ler­gic con­junc­tivi­tis. An­a­lysts have al­so been scratch­ing their heads over the com­mer­cial prospects of a ther­a­py avail­able in cheap eye drops.

None of that gets an­swered, though, be­fore the com­pa­ny wins a reg­u­la­to­ry green light. And now the biotech, which is part­nered with Re­gen­eron on an ex­tend­ed dos­ing ap­proach for Eylea, will once again have to go back to the draw­ing board over its man­u­fac­tur­ing woes as it preps for a third shot at an OK.

Said Oc­u­lar Ther­a­peu­tix CEO Amar Sawh­ney:

Im­por­tant­ly, there were no clin­i­cal is­sues iden­ti­fied in the CRL per­tain­ing to ef­fi­ca­cy or safe­ty re­lat­ed to the post-sur­gi­cal pain in­di­ca­tion. We be­lieve that DEX­TEN­ZA can be ap­proved once these open man­u­fac­tur­ing items are re­solved.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

UP­DAT­ED: Bris­tol My­ers Squibb com­mits $300 mil­lion to com­bat racial dis­par­i­ties, but de­clines to re­lease own de­mo­graph­ic da­ta

After the police killing of George Floyd, a flurry of pharma and biotech companies, executives and investors jumped out to make statements, either expressing support for Black Lives Matter and the protests or condemning systemic racism.

Now, a Big Pharma company is publicly putting some teeth behind those statements. This morning, Bristol Myers Squibb announced they would spend $300 million on a broad effort to reduce racial health disparities, and diversify both their clinical trials and their own executive team and workforce.