Oys­ter Point Phar­ma — a small Prince­ton, NJ-based biotech look­ing to dis­rupt a mas­sive eye drug mar­ket large­ly mo­nop­o­lized by two ther­a­pies — has pos­i­tive piv­otal tri­al da­ta as it march­es on a path to­wards US ap­proval.

Oys­ter, which went pub­lic in an $80 mil­lion IPO last year, has a nasal for­mu­la­tion of vareni­cline —a for­mu­la­tion of Pfiz­er’s smok­ing-ces­sa­tion drug Chan­tix — it is de­vel­op­ing for dry eye dis­ease, a chron­ic, episod­ic dis­ease af­fect­ing the tears and oc­u­lar sur­face that af­fects some 33 mil­lion Amer­i­cans.

The con­di­tion cur­rent­ly rep­re­sents a glob­al mar­ket of more than $5 bil­lion and is ex­pect­ed to grow to about $8 bil­lion by 2025, ac­cord­ing to Need­ham an­a­lysts. The ther­a­peu­tic mar­ket is dom­i­nat­ed by an­ti-in­flam­ma­to­ry eye drops: Al­ler­gan’s Resta­sis — al­though gener­ic com­pe­ti­tion is eat­ing in­to sales as Ab­b­Vie buys the large drug­mak­er — and Shire’s Xi­idra (pur­chased last year by No­var­tis in a $5.3 bil­lion deal), as well as over-the-counter ar­ti­fi­cial tears.

Two dos­es of Oys­ter’s for­mu­la­tion, called OC-01, (0.6 mg/ml and 1.2 mg/ml) were test­ed against a place­bo in 758 pa­tients with mild, mod­er­ate, and se­vere dry eye dis­ease in the ON­SET-2 study.

Both dos­es met the main goal of the study — a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in sub­jects gain­ing > 10 mm in Schirmer’s score (a test de­signed to as­sess a per­son’s eyes make enough tears to keep the eyes moist) at week 4, ver­sus the con­trol arm. In the small­er dose group, the per­cent­age of pa­tients hit­ting that score was 44%, and on the high­er group the per­cent­age of pa­tients hit­ting the goal was 47% — in the con­trol group, the per­cent­age was 26%.

In ad­di­tion, in the small­er dose group, the mean change in Schirmer’s score was 11.0 mm. In the high­er dose group the mean change in score was 11.2 mm — in the con­trol group, the change was 5.9 mm. Key sec­ondary goals, in­clud­ing an im­prove­ment in eye dry­ness, were al­so met. The most com­mon side ef­fect, ex­pe­ri­enced by half the pa­tients, was sneez­ing.

The ex­ist­ing crop of drugs such as Xi­idra and Resta­sis can take weeks or months to take ef­fect. Eye con­sul­tants in­di­cate that Resta­sis and Xi­idra have fail­ure rates of 50%+ and tol­er­a­bil­i­ty is­sues which fur­ther lim­it their use, and nei­ther prod­uct is com­plete­ly ef­fec­tive for all pa­tients, Cowen’s Ken Cac­cia­tore wrote in a note in late March, es­ti­mat­ing OC-01 rep­re­sents a $1 bil­lion-plus op­por­tu­ni­ty giv­en the need for new treat­ment al­ter­na­tives.

The ON­SET-2 study adds to mixed but large­ly pos­i­tive mid-stage tri­al da­ta on OC-01 — and Oys­ter is prep­ping to sub­mit a mar­ket­ing ap­pli­ca­tion in the sec­ond quar­ter.

“The abil­i­ty to show sta­tis­ti­cal­ly sig­nif­i­cant sign and symp­tom end­points with­in the same clin­i­cal tri­al has been elu­sive in dry eye dis­ease,” said com­pa­ny chief Jef­frey Nau in a state­ment. “The abil­i­ty to meet this high bar in the ON­SET-2 pop­u­la­tion con­sist­ing of mild, mod­er­ate, and se­vere sub­jects is even more no­table and speaks to the broad ap­plic­a­bil­i­ty of OC-01 to treat dry eye pa­tients.”

For ex­am­ple, mixed tri­al re­sults prompt­ed the FDA to ask for ad­di­tion­al ef­fi­ca­cy da­ta on Xi­idra, which were even­tu­al­ly sup­plied. On­ly then did the agency sanc­tion ap­proval in 2016.

An­oth­er po­ten­tial dry eye dis­ease drug, Kala Phar­ma­ceu­ti­cals’ Ey­su­vis, which is be­ing de­vel­oped for acute use, al­so had a mixed dataset, which prompt­ed a re­jec­tion last Au­gust. In March, the com­pa­ny — one of the pletho­ra of biotechs that trace their roots to the pro­lif­ic lab at MIT led by Bob Langer — post­ed da­ta from an­oth­er late-stage study as it looks to re­sub­mit its mar­ket­ing ap­pli­ca­tion.

Novartis’ Entresto started the ACC weekend off rough with a trial flop in heart attack patients, slowing the drug’s push into earlier patients. Now, an NIH-sponsored study is casting doubt on Entresto’s use in the most severe heart failure patients, another black mark on the increasingly controversial drug’s record.

Entresto, a combination of sacubitril and valsartan, could not beat out valsartan alone in an outcomes head-to-head for severe heart failure patients with a reduced ejection fraction (HFrEF), according to data presented Monday at the virtual American College of Cardiology meeting.

When Biolyse, an Ontario-based manufacturer of sterile injectables, forged a deal with Bolivia last week to manufacture up to 50 million J&J Covid-19 vaccine doses, the agreement kicked off what will prove to be a test case for how difficult the system of compulsory licenses is to navigate.

The first problem: When Biolyse asked J&J, via a March letter, to license its Covid-19 vaccine, manufacture it in Canada and pay 5% royalties on shipments to needy, low-income countries, J&J rejected the offer, refusing to negotiate. J&J also did not respond to a request for comment.

As Incyte prepares to potentially hit the market with a topical formulation of its cash cow ruxolitinib in atopic dermatitis, the Wilmington, DE-based company is beefing up its data package for another indication: vitiligo.

Incyte released Phase III results from two of its clinical vitiligo programs Monday morning, saying both studies met their primary endpoints of patients achieving at least 75% improvement from baseline in repigmentation of the face. The data will likely lead Incyte to ask for approval in both the US and Europe for those older than 12 before the end of the year.

While Cytokinetics’ heart drug technically met its primary endpoint back in November, it missed a key secondary endpoint — reduction in cardiovascular death — which eventually cost the company two partnerships. Now the team is back with data suggesting the drug works better in sicker patients, and it’s planning a trip to the FDA.

In a post-hoc analysis, which can be a very difficult sale at the FDA, Cytokinetics separated patients from the Phase III GALACTIC-HF study into four quartiles based on ejection fraction, a measurement of how well the left ventricle pumps blood with each heartbeat. Patients in the lower two quartiles — those with an EF of 22% or lower, and between 29% to 32% — saw a 15% and 17% relative risk reduction of heart failure events and cardiovascular death combined, Cytokinetics reported at ACC. No difference was seen in the upper two quartiles.