Mile­stone Phar­ma had all the ear­marks of a clas­sic biotech IPO sto­ry when it went pub­lic last year.

The lit­tle 12-year-old biotech had pos­i­tive mid-stage da­ta to tout to in­vestors that un­der­scored the po­ten­tial of a nasal spray treat­ment to halt parox­ys­mal supraven­tric­u­lar tachy­car­dia — or PSVT — events. A big syn­di­cate put up $80 mil­lion to help them lay a com­mer­cial foun­da­tion. And the stock peaked soon af­ter the com­pa­ny de­buted on Nas­daq as they raised an­oth­er $82 mil­lion.

But the Mile­stone team — and the stock $MIST — crashed at an ug­ly twist in the Phase III de­vel­op­ment path. Their drug, etri­pamil, a fast-act­ing cal­ci­um chan­nel block­er, failed the pri­ma­ry. Shares were rout­ed, free-falling 72% ahead of the bell.

The biotech, based in Mon­tre­al, was look­ing to of­fer an at-home so­lu­tion to a rapid heart­beat that usu­al­ly is un­threat­en­ing but sends some peo­ple back to the ER over and over again. At Phase II, re­searchers said they tracked suc­cess rates of 65% to 95%, beat­ing out the 35% place­bo score.

The pri­ma­ry end­point was a com­par­i­son of the nasal ther­a­py with place­bo over 5 hours in turn­ing SVT to si­nus rhythm, and it fell far short of the mark on sta­tis­ti­cal sig­nif­i­cance with a val­ue of 0.12. It al­so fell short on re­duc­ing trips for emer­gency care, with the same p val­ue.

Joseph Olive­to Mile­stone

Mile­stone, though, isn’t go­ing to give up at this point with­out a strug­gle. There was a pos­i­tive hit on pa­tient-re­port­ed treat­ment sat­is­fac­tion, a pos­i­tive “trend” on the ER mea­sure and they drilled down to make the case on the drug’s ear­ly im­pact. And that’s what they’re tak­ing to the FDA now for ex­plorato­ry talks.

“(O)ut­comes af­ter 100 min­utes, which were af­fect­ed by a very small num­ber of place­bo pa­tients re­main­ing in the study at that time, sug­gest that the de­sign and analy­sis plan used in NODE-301 neg­a­tive­ly im­pact­ed the study’s out­come,”  said CEO Joseph Olive­to in a state­ment.

There’s more da­ta to come, but the com­pa­ny is sound­ing a note of cau­tion on how the Covid-19 out­break might af­fect tri­al op­er­a­tions at this stage.

So­cial im­age: Joseph Olive­to, Mile­stone Phar­ma

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

Ocular Therapeutix $OCUL has had its ups and downs in the 7 years since it went public. Friday was one of those down days.

The Bedford, MA-based biotech reported that its lead experimental eye drug, OTX-CSI (cyclosporine intracanalicular insert), failed a Phase II trial for dry eye disease. And the stock experienced one of its periodic meltdowns, dropping more than 30% ahead of the bell.

The therapy flat failed the primary endpoint: increased tear production at 12 weeks as measured by the Schirmer’s Test compared to the vehicle control group. And while investigators called out an improvement from baseline in “signs of dry eye disease as measured by total corneal fluorescein staining (CFS) and symptoms of dry eye disease as measured by the visual analogue scale (VAS) eye dryness in subjects treated with the OTX-CSI insert,” it wasn’t statistically significant.

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

Sanofi and Regeneron can boast of another inflammatory disease where Dupixent has proven effective.

The best-selling drug, which targets both IL-4 and IL-13, has delivered a clean sweep in a Phase III trial for prurigo nodularis, a chronic disease characterized by itch so intense that it can affect patients’ sleep and psychology. Thick skin lesions can cover most of the body.

On the primary endpoint, 37% of patients taking Dupixent saw a clinically meaningful reduction in itch compared to 22% of those on placebo (p=0.0216) at week 12. All secondary endpoints were also met, including clearance of skin lesions and improvement in quality of life.