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

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).

The Houston-based biotech Salarius Pharmaceuticals is lifting the cover on data from a Phase I/II trial for a drug currently on voluntary hold after a patient death, and the results appear to have underwhelmed investors.

Salarius’ candidate, dubbed seclidemstat, is an oral LSD1 inhibitor that is meant to treat Ewing sarcoma and FET-rearranged sarcomas in patients under 12 years old. The biotech had presented data with 13 patients with “first- and second-relapse Ewing sarcoma” who were treated in combination with topotecan and cyclophosphamide.