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

AstraZeneca once projected its Brilinta drug to peak at $3.5 billion in sales, and though the blood thinner never reached that lofty goal, it received the latest positive signs in a string of recent good news.

The pharma released full details from its THALES study Thursday morning, which measured the effects of Brilinta and aspirin against aspirin alone in treating patients who had an acute ischemic stroke or transient ischemic attack. When taken twice daily with once-a-day aspirin for 30 days, the Brilinta combo reduced the risk of stroke and death by 17 percent, meeting the primary endpoint of the study.

Three months after Merck published in the New England Journal of Medicine data that left doctors and investors divided over just how well its experimental heart drug worked, the FDA has handed that drug priority review. A decision is now due by January 20, 2021.

Merck first announced the drug, known as vericiguat, as a Phase III success last November. In 2016, Merck had paid $1 billion upfront for US rights to the Bayer-developed drug. Early projections foresaw a few hundred million a year in sales, but the unspecified late-stage success raised the possibility for far more. After all, Novartis’s flagship heart drug, Entresto, was earning $1.7 billion per year and was expected to reach up to $4 billion in annual sales.

After spending 4 years in a senior post with GlaxoSmithKline’s star team positioning Shingrix for a blockbuster approval, Guillaume Pfefer is headed back to the biotech world — in style.

Pfefer has signed on to join Noubar Afeyan’s busy group of partners at Flagship, and he’s taking the helm of an upstart — which today is being merged with another Flagship startup — with some grand plans of its own. The announcement this morning notes that Pfefer will run Kintai Therapeutics, one of the grads of the Flagship labs.

→Merck KGaA has taken an early option on an immuno-oncology program developed at F-star Therapeutics. This is their second option in the collaboration. And they added a pair of preclinical discovery programs to the alliance as well.

→ Any biotech going public these days wouldn’t feel right if they didn’t upsize the offering. And that’s just what Phase I biotech Pandion Therapeutics did. The autoimmune company is now selling 7 million shares, a 1.5 million share bump, for $16 to $18 a share.

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.