Aeter­na Zen­taris shares evis­cer­at­ed by its lat­est late-stage drug dis­as­ter

David Dodd, Aeter­na Zen­taris

When Aeter­na Zen­taris $AEZS planned a move to Charleston, SC, a cou­ple of years ago, the biotech promised to cre­ate a batch of new jobs as it wres­tled to make a come­back fol­low­ing a sting­ing FDA re­jec­tion of its lead drug. This morn­ing, though, the com­pa­ny sim­ply added to its litany of set­backs.

Billed by at least one an­a­lyst as the fu­ture of the com­pa­ny, the biotech an­nounced that its drug Zop­trex failed a Phase III study for ad­vanced en­dome­tri­al can­cer. And now it’s sim­ply wash­ing its hands of the drug and go­ing back to see if they can wres­tle an ap­proval for an old drug that’s al­ready been re­ject­ed by the FDA.

Shares of Aeter­na Zen­taris were evis­cer­at­ed, drop­ping 57% in pre-mar­ket trad­ing.

In­ves­ti­ga­tors said the biotech’s drug looked re­mark­ably like the dox­oru­bicin com­par­i­son arm. Zop­trex scored a 10.9 month over­all sur­vival rate com­pared to 10.8 months for dox­oru­bicin. The sec­ondary end­points were al­so a match.

That re­sult fol­lows a Phase III fail­ure of their lead drug Macrilen in Jan­u­ary, which the FDA re­ject­ed three years ago. Aeter­na Zen­taris said it was con­sid­er­ing what next steps, if any, it would take on the drug af­ter the con­fir­ma­to­ry study end­ed in fail­ure. Then weeks lat­er the com­pa­ny added that the FDA had spelled out the ad­di­tion­al da­ta need­ed that could lead to an ap­proval. And this morn­ing, faced with a sep­a­rate dis­as­ter, they are now talk­ing not on­ly about pur­su­ing the new drug ap­pli­ca­tion, but mar­ket­ing plans as well.

It’s a tough sale, though, as the last bit of cred­i­bil­i­ty at the com­pa­ny takes a bruis­ing hit.

CEO David Dodd had this to say:

Our fo­cus has now shift­ed en­tire­ly to fil­ing our new drug ap­pli­ca­tion for Macrilen and, if the prod­uct is ap­proved, to its com­mer­cial launch as soon as pos­si­ble. We will al­so op­ti­mize our re­sources to be con­sis­tent with our fo­cus on Macrilen-re­lat­ed ef­forts. We con­tin­ue to be­lieve in the po­ten­tial that Macrilen pro­vides for us to be­come a fo­cused spe­cial­ty phar­ma­ceu­ti­cal com­pa­ny. Our in­ten­tion is to sub­mit the Macrilen NDA in the third quar­ter of 2017 and, if the prod­uct re­ceives FDA ap­proval, to com­mer­cial­ly launch the prod­uct in the first quar­ter of 2018.

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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.

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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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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.

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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.

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