The FDA's in­sid­er view on Aerie’s glau­co­ma drug: Yes on ef­fi­ca­cy, but…

Aerie CEO Vi­cente Anido

The FDA had some en­cour­ag­ing words for Aerie’s new glau­co­ma drug Rho­pres­sa, but there’s a sig­nif­i­cant caveat that might come back to haunt the biotech as it hunts an ap­proval.

In the in­ter­nal re­view of­fered up by the agency two days ahead of the mis­sion-crit­i­cal pan­el dis­cus­sion and vote, FDA in­sid­ers give the da­ta a thumbs up on ef­fi­ca­cy, but note that the com­para­tor ther­a­py was bet­ter than the drug in the more se­vere­ly af­flict­ed pa­tients in­clud­ed in their Phase III stud­ies.

Look­ing over the da­ta from three Phase III stud­ies, the FDA re­view­ers stat­ed:

(W)e con­cur with the Ap­pli­cant’s over­all ef­fi­ca­cy con­clu­sion that ne­tar­sudil 0.02% once-dai­ly (QD) (al­so re­ferred to as ne­tar­sudil QD) is ef­fi­ca­cious for the re­duc­tion of el­e­vat­ed in­traoc­u­lar pres­sure (IOP). How­ev­er, ne­tar­sudil QD is less ef­fi­ca­cious com­pared to the com­para­tor, tim­o­lol oph­thalmic so­lu­tion 0.5% twice dai­ly (BID), for sub­jects with high­er max­i­mum base­line IOP (≥ 25 mmHg).

In­vestors didn’t seem to mind the is­sue, at least at first glance. Aerie’s stock shot up 14% as the re­view made the rounds.

The FDA’s note on ef­fi­ca­cy im­me­di­ate­ly drew the at­ten­tion of Mizuho’s Difei Yang. But Yang says the sug­gest­ed la­bel doesn’t re­strict an OK to mild to mod­er­ate pa­tients, paving the way to wide­spread use. Yang notes:

We view FDA’s mod­i­fi­ca­tions to Aerie’s pro­posed lan­guage to be very mi­nor. Ad­di­tion­al­ly, we are en­cour­aged by the lan­guage used in the in­di­ca­tion and us­age sec­tion where there is no men­tion of Rho­pres­sa be­ing used for mild to mod­er­ate glau­co­ma pa­tients on­ly. The cur­rent pro­posed la­bel reads as fol­lows, “RHO­PRES­SA (ne­tar­sudil oph­thalmic so­lu­tion) 0.02% is a Rho ki­nase in­hibitor in­di­cat­ed for the re­duc­tion of el­e­vat­ed in­traoc­u­lar pres­sure in pa­tients with open-an­gle glau­co­ma or oc­u­lar hy­per­ten­sion.” We be­lieve this to be a best case sce­nario for Rho­pres­sa.

Aerie, though, still has to clear a bar on the man­u­fac­tur­ing side. Its drug is man­u­fac­tured at the Bausch + Lomb man­u­fac­tur­ing fa­cil­i­ty in Tam­pa, which re­cent­ly failed to mea­sure up to fed­er­al stan­dards for the sec­ond time. The Valeant com­pa­ny, though, fol­lowed up with a vow that it will soon have its house in or­der, en­cour­ag­ing in­vestors as short sell­ers hov­ered about in hopes of a set­back.

A bull­ish Aerie CEO Vi­cente Anido has al­ready tout­ed Rho­pres­sa as a like­ly block­buster able to earn more than $1 bil­lion a year, even though the biotech had to get the FDA’s per­mis­sion to change the end­point for its sec­ond late-stage study of Rho­pres­sa in or­der to avoid back-to-back fail­ures.

Aerie is al­so two for two on its piv­otal Phase III stud­ies for Ro­cla­tan, a com­bo drug that com­bines the gener­ic la­tanoprost to Rho­pres­sa.

John Hood [file photo]

UP­DATE: Cel­gene and the sci­en­tist who cham­pi­oned fe­dra­tinib's rise from Sanofi's R&D grave­yard win FDA OK

Six years after Sanofi gave it up for dead, the FDA has approved the myelofibrosis drug fedratinib, now owned by Celgene.

The drug will be sold as Inrebic, and will soon land in the portfolio at Bristol-Myers Squibb, which is finalizing a deal to acquire Celgene.

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UP­DAT­ED: AveX­is sci­en­tif­ic founder was axed — and No­var­tis names a new CSO in wake of an ethics scan­dal

Now at the center of a storm of controversy over its decision to keep its knowledge of manipulated data hidden from regulators during an FDA review, Novartis CEO Vas Narasimhan has found a longtime veteran in the ranks to head the scientific work underway at AveXis, where the incident occurred. And the scientific founder has hit the exit.

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Ab­b­Vie gets its FDA OK for JAK in­hibitor upadac­i­tinib, but don’t look for this one to hit ex­ecs’ lofty ex­pec­ta­tions

Another big drug approval came through on Friday afternoon as the FDA OK’d AbbVie’s upadacitinib — an oral JAK1 inhibitor that is hitting the rheumatoid arthritis market with a black box warning of serious malignancies, infections and thrombosis reflecting fears associated with the class.

It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

The top 10 fran­chise drugs in bio­phar­ma his­to­ry will earn a to­tal of $1.4T (tril­lion) by 2024 — what does that tell us?

Just in case you were looking for more evidence of just how important Amgen’s patent win on Enbrel is for the company and its investors, EvaluatePharma has come up with a forward-looking consensus estimate on what the list of top 10 drugs will look like in 2024.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

ICER has some strong words for PTC, Sarepta and the FDA as the US drug price watchdog concludes that as currently priced, their respective new treatments for Duchenne muscular dystrophy are decidedly not cost-effective.

The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

UP­DAT­ED: Sci­en­tist-CEO ac­cused of im­prop­er­ly us­ing con­fi­den­tial in­fo from uni­corn Alec­tor

The executive team at Alector $ALEC has a bone to pick with scientific co-founder Asa Abeliovich. Their latest quarterly rundown has this brief note buried inside:

On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

There’s no explicit word in the filing on what kind of confidential info was involved, but the proceeding got started 2 days ahead of Abeliovich’s IPO.

Abeliovich, formerly a tenured associate professor at Columbia, is a top scientist in the field of neurodegeneration, which is where Alector is targeted. More recently, he’s also helped start up Prevail Therapeutics as the CEO, which raised $125 million in an IPO. And there he’s planning on working on new gene therapies that target genetically defined subpopulations of Parkinson’s disease. Followup programs target Gaucher disease, frontotemporal dementia and synucleinopathies.

But this time Abeliovich is the CEO rather than a founding scientist. And some of their pipeline overlaps with Alector’s.

Abeliovich and Prevail, though, aren’t taking this one lying down.

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Chi­na has be­come a CEO-lev­el pri­or­i­ty for multi­na­tion­al phar­ma­ceu­ti­cal com­pa­nies: the trend and the im­pli­ca­tions

After a “hot” period of rapid growth between 2009 and 2012, and a relatively “cooler” period of slower growth from 2013 to 2015, China has once again become a top-of-mind priority for the CEOs of most large, multinational pharmaceutical companies.

At the International Pharma Forum, hosted in March in Beijing by the R&D Based Pharmaceutical Association Committee (RDPAC) and the Pharmaceutical Research and Manufacturers of America (PhRMA), no fewer than seven CEOs of major multinational pharmaceutical firms participated, including GSK, Eli Lilly, LEO Pharma, Merck KGaA, Pfizer, Sanofi and UCB. A few days earlier, the CEOs of several other large multinationals attended the China Development Forum, an annual business forum hosted by the research arm of China’s State Council. It’s hard to imagine any other country, except the US, having such drawing power at CEO level.

As dis­as­ter struck, Ab­b­Vie’s Rick Gon­za­lez swooped in on Al­ler­gan with an of­fer Brent Saun­ders couldn’t say no to

Early March was a no good, awful, terrible time for Allergan CEO Brent Saunders. His big lead drug had imploded in a Phase III disaster and activists were after his hide — or at least his chairman’s title — as the stock price continued a steady droop that had eviscerated share value for investors.

But it was a perfect time for AbbVie CEO Rick Gonzalez to pick up the phone and ask Saunders if he’d like to consider a “strategic” deal.

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CEO Pascal Soriot via Getty Images

As­traZeneca's jug­ger­naut PARP play­er Lyn­parza scoops up an­oth­er dom­i­nant win in PhI­II as the FDA adds a 'break­through' for Calquence

AstraZeneca’s oncology R&D group under José Baselga keeps churning out hits.

Wednesday morning the pharma giant and their partners at Merck parted the curtains on a successful readout for their Phase III PAOLA-1 study, demonstrating statistically significant improvement in progression-free survival for women with ovarian cancer in a first-line maintenance setting who added their PARP Lynparza to Avastin. This is their second late-stage success in ovarian cancer, which will help stave off rivals like GSK.

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