No­van’s shares in melt­down fol­low­ing "dis­cor­dant" PhI­II stud­ies of a new ac­ne drug

Nathan Stasko, No­van

Of­fi­cial­ly, No­van $NOVN put out some mixed da­ta from two Phase III stud­ies of their lead drug for ac­ne, with one suc­cess and one fail­ure. But even the pos­i­tive da­ta from one tri­al failed to stand out from the con­trol arm in a ma­jor way, putting this com­pa­ny un­der a cloud.

Its stock plunged 80% this morn­ing.

On the up side, in­ves­ti­ga­tors say that SB204 hit all three co-pri­ma­ry end­points for NI-AC302, with sig­nif­i­cant – though far from im­pres­sive – re­sults for re­duc­ing the num­ber of le­sions and ac­ne clear­ance. Here are the num­bers, for both stud­ies:

•The ab­solute change from base­line in the num­ber of non-in­flam­ma­to­ry le­sions in NI-AC301 was -15.4 for SB204 and -13.4 for ve­hi­cle (p=0.030), and in NI-AC302 was -14.9 for SB204 and -12.3 for ve­hi­cle (p=0.001).

•The ab­solute change from base­line in the num­ber of in­flam­ma­to­ry le­sions in NI-AC301 was -12.1 for SB204 and -11.1 for ve­hi­cle (p=0.114), and in NI-AC302 was -12.9 for SB204 and -10.6 for ve­hi­cle (p<0.001).

•The pro­por­tion of pa­tients with IGA suc­cess in NI-AC301 was 13.4% for SB204 and 13.8% for ve­hi­cle (p=0.866), and in NI-AC302 was 18.9% for SB204 and 14.3% for ve­hi­cle (p=0.032).

A to­tal of 2,639 pa­tients ages 9 and old­er with mod­er­ate to se­vere ac­ne were en­rolled across a to­tal of 110 sites in the Unit­ed States.

The sec­ondary end­points looked quite sim­i­lar, leav­ing Mor­risville, NC-based No­van count­ing its cash. There’s enough mon­ey on hand to make it through the end of the year, when it’s hop­ing to have some bet­ter da­ta from an up­com­ing read­out on an an­ti-fun­gal pro­gram.

No­van raised $45 mil­lion with its IPO last fall, ear­mark­ing the mon­ey for an NDA for this drug, which it billed as the first new chem­i­cal en­ti­ty to come along for ac­ne in 20 years. Those plans may well have to change now. The biotech end­ed the day yes­ter­day with a mar­ket cap of $298 mil­lion.

“While we are pleased with the re­sults of the NI-AC302 tri­al that met the reg­u­la­to­ry re­quire­ment for sta­tis­ti­cal­ly sig­nif­i­cant ef­fi­ca­cy of SB204, we are dis­ap­point­ed with the dis­cor­dant re­sults of NI-AC301. Our team has not yet re­ceived the full da­ta set and we in­tend to pro­vide an up­date on the SB204 pro­gram af­ter our com­plete analy­sis,” said Nathan Stasko, PhD, Pres­i­dent and CEO of No­van. “De­spite these dis­cor­dant re­sults, we be­lieve in the po­ten­tial of ni­tric ox­ide’s mul­ti­ple, well-doc­u­ment­ed mech­a­nisms of ac­tion and the da­ta we have re­cent­ly gen­er­at­ed for our SB206 an­ti-vi­ral and SB414 an­ti-in­flam­ma­to­ry prod­uct can­di­dates. We con­tin­ue to look for­ward to near term clin­i­cal re­sults from our SB208 an­ti-fun­gal pro­gram in the sec­ond quar­ter of 2017 and ad­vanc­ing our pipeline of in­no­v­a­tive ther­a­pies for pa­tients suf­fer­ing from skin dis­eases.”

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Vas Narasimhan. Getty Images

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David Liu, Liu Group

David Liu un­veils newest ad­vance­ment in CRISPR tech: Prime edit­ing

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David Liu, base editing pioneer and founder of Beam Therapeutics, published the findings in Nature alongside Andrew Anzalone. They estimated that the breakthrough “in principle” puts 89% of human diseases in purview — although experts cautioned that human therapies were a long way off.

Bhaskar Chaudhuri. Frazier Healthcare Partners

Fra­zier Health­care Part­ner­s' der­ma­tol­ogy up­start at­tracts a mar­quee syn­di­cate, $94M+ for 'in-be­tween' top­i­cal drug

For the past three years Frazier Healthcare Partners’ Bhaskar Chaudhuri has been carefully and quietly grooming Arcutis Therapeutics, a new dermatology play he co-founded to deliver topical formulations of well-known drugs. Now that the biotech is poised to enter Phase III, he’s being joined by a marquee syndicate for its $94.5 million Series C.

HBM Healthcare Investments, Vivo Capital, BlackRock, Omega Funds, Pivotal BioVentures, and Goldman Sachs jumped on board, joining Bain Capital Life Sciences, OrbiMed and RA Capital Management in backing Arcutis’ lead topical cream for plaque psoriasis.

A new com­pa­ny en­ters the Tec­fidera fight, of­fer­ing to kill two birds

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Sometime in the 1st century AD, a patient presented to Arataeus looking like a varicose ghost. He was “emaciated and atrophied, pale, feeble and incapable of performing any of his accustomed works,” the Greek physician wrote, with hollow temples and huge veins running all over his body.

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Just weeks after Bristol-Myers Squibb defended its failed pivotal study pitting Opdivo against Nexavar in liver cancer, Roche says it’s beat the frontline challenge with a combination of their PD-L1 Tecentriq with Avastin. And now they’re rolling their regulatory teams in the US, Europe and China in search of a new approval — badly needed to boost a trailing franchise effort.
Given their breakthrough and Big Pharma status as well as the use of two approved drugs, FDA approval may well prove to be something of a formality. And the Chinese have been clear that they want new drugs for liver cancer, where lethal disease rates are particularly high.
Researchers at their big biotech sub, Genentech, say that the combo beat Bayer’s Nexavar on both progression-free survival as well as overall survival — the first advance in this field in more than a decade. We won’t get the breakdown in months of life gained, but it’s a big win for Roche, which has lagged far, far behind Keytruda and Opdivo, the dominant PD-1s that have captured the bulk of the checkpoint market so far.
Researchers recruited hepatocellular carcinoma — the most common form of liver cancer — patients for the IMbrave150 study who weren’t eligible for surgery ahead of any systemic treatment of the disease.
Roche has a fairly low bar to beat, with modest survival benefit for Nexavar, approved for this indication 12 years ago. But they also plan to offer a combo therapy that could have significantly less toxicity, offering patients a much easier treatment regimen.
Cowen’s Steven Scala recently sized up the importance of IMbrave150, noting:

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