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

Opthotech CEO David R. GuyerOpthotech 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.”

Janet Woodcock (AP Images)

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Janet Woodcock is set to be the most powerful person at the FDA in less than a week.

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Janet Woodcock (AP Images)

Janet Wood­cock is in the run­ning for FDA com­mis­sion­er — what does that mean for the agen­cy's fu­ture?

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The decision, as the initial wave of reactions suggest, could have dramatic implications for where the agency is headed in the next four years — if not beyond.

Woodcock, the longtime CDER director, is being vetted alongside former FDA principal deputy commissioner Joshua Sharfstein, Bloomberg reported. Already tapped as acting head of the agency, she’s set to take over from Stephen Hahn right after Biden’s inauguration next week.

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CEO Brett Monia (Ionis)

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Terry Rosen, Arcus CEO

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David Kessler in April 2009 (Eric Risberg/AP Images)

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Friday the European agency put out the 5th in a series of statements about the hackers who broke into their system, noting that some of the information on vaccines that was gleaned in the attack is showing up online — altered to raise questions about the Covid-19 vaccines now in use.

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Peter Thiel, Getty (Photographer: Kiyoshi Ota/Bloomberg)

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