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.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.

As court case looms, Bris­tol My­ers touts la­bel ex­pan­sion for Breyanzi

As Bristol Myers Squibb braces for a court battle over a costly delay — at least for Celgene shareholders — for its CAR-T lymphoma treatment Breyanzi, the pharma giant is touting a label expansion in the second-line setting.

Breyanzi, also known as liso-cel, snagged a win on Friday in adults with large B-cell lymphoma (LBCL) who: don’t respond to chemotherapy, or relapse within 12 months; don’t respond or relapse after 12 months; or are not eligible for hematopoietic stem cell transplant after chemo due to their age or comorbidities.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.