Todd Brady, Aldeyra CEO

Aldeyra shares spi­ral af­ter a late-stage fail­ure in dry eye dis­ease — but that won't stop its pur­suit of an NDA

Aldeyra Ther­a­peu­tics’ shares tanked in pre­mar­ket trad­ing af­ter the com­pa­ny ad­mit­ted its dry eye can­di­date re­prox­alap missed the pri­ma­ry end­point in a topline Phase III read­out — but who needs the pri­ma­ry?

De­spite the late-stage fail­ure, Aldeyra is charg­ing ahead with an­oth­er Phase III tri­al, and CEO Todd Brady is con­fi­dent a sec­ondary end­point will lead the can­di­date to suc­cess.

“We con­tin­ue to ad­vance re­prox­alap to­ward NDA sub­mis­sion as we fo­cus on the com­ple­tion of TRAN­QUIL­I­TY-2 and en­roll­ment in the 12-month safe­ty tri­al,” he said in a state­ment.

In­vestors weren’t as con­fi­dent. $ALDX shares slipped more than 38% in pre­mar­ket trad­ing Tues­day, pric­ing in at $4.39 — a long fall from yes­ter­day’s clos­ing price of $7.13.

While re­prox­alap met the pri­ma­ry end­point of oc­u­lar red­ness in a Phase II tri­al, the end­point didn’t reach sta­tis­ti­cal sig­nif­i­cance in the Phase III TRAN­QUIL­I­TY read­out, Aldeyra an­nounced on Mon­day with­out pro­vid­ing any hard num­bers.

It did, how­ev­er, meet the sec­ondary end­point for dry eye dis­ease based on the Schirmer test, which de­ter­mines whether a pa­tient’s eye pro­duces enough tears. Again with­out pro­vid­ing the ac­tu­al re­sult, Aldeyra said the p-val­ue there was 0.0001.

The Schirmer test has been ac­cept­ed by the FDA as part of the ba­sis for ap­proval of oth­er dry eye prod­ucts, the Lex­ing­ton, MA-based com­pa­ny said. So it’s go­ing forth with an­oth­er Phase III study, dubbed TRAN­QUIL­I­TY-2 — ex­cept this time, it’s mod­i­fy­ing the tri­al so that the pri­ma­ry end­point will be met if sta­tis­ti­cal sig­nif­i­cance is achieved in ei­ther oc­u­lar red­ness or the Schirmer test.

In ad­di­tion, tar­get en­roll­ment has been upped from 300 to 400 pa­tients, ac­cord­ing to Aldeyra. Top-line re­sults there are ex­pect­ed in mid-2022.

“Fol­low­ing the achieve­ment of sta­tis­ti­cal sig­nif­i­cance in oc­u­lar red­ness in our re­cent Phase 2 clin­i­cal tri­al, the achieve­ment of sta­tis­ti­cal sig­nif­i­cance of Schirmer test in TRAN­QUIL­I­TY may pro­vide an ad­di­tion­al op­tion to sat­is­fy the re­main­ing ob­jec­tive sign re­quire­ment for dry eye dis­ease NDA sub­mis­sion,” Brady said in a state­ment.

In­vestors cheered on Aldeyra a few years ago when re­prox­alap achieved sta­tis­ti­cal sig­nif­i­cance in a Phase IIb read­out for two dos­es rat­ed on the Four-Symp­tom Oc­u­lar Dry­ness Score and the Over­all Oc­u­lar Dis­com­fort Symp­tom Score. How­ev­er, some care­ful ob­servers no­ticed that the high dose hit at week 8, then lost sta­tis­ti­cal sig­nif­i­cance at week 12, while the low dose out­per­formed the high dose at week 12 — rais­ing some ques­tions about dura­bil­i­ty and dose re­sponse.

Pend­ing the en­roll­ment of an on­go­ing 12-month safe­ty study and the out­come of TRAN­QUIL­I­TY-2, Brady says an NDA sub­mis­sion could come as soon as mid-2022.

This isn’t the in­dus­try’s on­ly fail­ure this year in dry eye dis­ease.

In Oc­to­ber, Oc­u­lar Ther­a­peu­tix an­nounced its lead ex­per­i­men­tal eye drug, OTX-CSI (cy­closporine in­tra­canalic­u­lar in­sert), failed to hit the pri­ma­ry end­point in a Phase II tri­al: in­creased tear pro­duc­tion at 12 weeks as mea­sured by the Schirmer test com­pared to the ve­hi­cle con­trol group. Aerie’s dry eye dis­ease drug al­so missed the pri­maries in a Phase IIb tri­al back in Sep­tem­ber, but the com­pa­ny said it would plunge right in­to two Phase III tri­als any­way.

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Tom Barnes, Orna Therapeutics CEO

UP­DAT­ED: 'We have failed to fail': Mer­ck gam­bles $250M cash on a next-gen ap­proach to mR­NA — af­ter punt­ing its big al­liance with Mod­er­na

Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Marisol Peron, Genmab SVP of communications and corporate affairs

Gen­mab launch­es cor­po­rate cam­paign am­pli­fy­ing its ‘knock your socks off’ an­ti­bod­ies

Genmab often talks about its “knock-your-socks-off” antibodies — and now the term is getting its own logo and corporate campaign.

The teal and purple logo for the acronym KYSO — Genmab pronounces it “ky-so” — debuts on Wednesday and comes on the heels of Genmab’s newly announced 2030 vision. That aspiration aims to expand Genmab’s drug development beyond oncology to include other serious diseases, while also doubling down on its own drug development.

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