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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Nurses star in J&J's campaign centered on the importance of nurses who are increasingly stressed, burnt out and quitting the profession (via Johnson & Johnson)

Thank­ful for nurs­es: J&J's new cam­paign aims to re­set pan­dem­ic clock back to grat­i­tude

In the early days of the pandemic, people cheered for nurses – delivering food, writing thank you notes and ringing bells nightly to show their appreciation. But something shifted this summer, and now Johnson & Johnson wants to remind people of the gratitude that nurses still deserve.

Call it politics or pandemic weariness or the result of almost two years of a deadly pandemic, but nurses today face threats and mistreatment from patients and their angry family members. And nurses are leaving the profession in record numbers.

Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Drug­mak­ers cut prices on av­er­age by more than 60% to get on Chi­na's 2022 NDRL list — re­port

China’s National Reimbursement Drug List (NRDL) is a crystal clear example of the country’s bargaining power in the biotech and pharma market, as more firms have reportedly agreed to cut their prices for 67 new medicines to be included in its national medical insurance coverage starting in January.

Being on the list is lucrative. Essentially, if a biotech or pharma company gets on this list, they’re covered by the biggest insurance network in the country. Given China’s vast population, the Chinese government has significant leverage to decide which medicines can make a profit. While domestic drugmakers are quite willing to play that game, cutting prices significantly in exchange for getting on the list, international companies don’t do it as often.