Savara watched their stock crater in June as they an­nounced a late-stage fail­ure on their lead com­pound. But now, based on the same Phase III tri­al, the FDA is giv­ing the biotech and its rare lung dis­ease drug break­through ther­a­py des­ig­na­tion.

The des­ig­na­tion comes for Mol­gradex, an in­halant meant to treat au­toim­mune pul­monary alve­o­lar pro­teinosis (aPAP). It was the lead com­pound in Savara’s 2016 buy­out of Seren­dex. The dis­ease is caused by au­toan­ti­bod­ies that neu­tral­ize a pro­tein called GM-CSF in the lungs, and the in­halant re­plen­ish­es the lungs with a re­com­bi­nant form of the pro­tein.

Savara said in June that the drug had failed to sig­nif­i­cant­ly im­prove oxy­gen lev­els in tri­al pa­tients’ alve­o­lar, the parts of the lung ob­struct­ed in PAP pa­tients. Savara tried to sal­vage the study, point­ing to sec­ondary and “ex­plorato­ry” end­points and blam­ing the pri­ma­ry fail­ure on an un­ex­pect­ed­ly high place­bo ef­fect. In­vestors didn’t buy it, tank­ing their stock price $SVRA 75%.

The FDA, though, ap­pears to think there’s a sto­ry there. It’s im­pos­si­ble to know the agency’s think­ing, but de­tailed slides Savara re­leased this fall em­pha­sized “a pat­tern of im­prove­ment” on sev­er­al met­rics in the 139-per­son tri­al and ar­gued that the “to­tal­i­ty of out­come da­ta” sup­port Mol­gradex over place­bo.

That da­ta in­clud­ed sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments on the Saint Georges Res­pi­ra­to­ry Ques­tion­naire and CT scans. The biotech al­so ar­gued the pri­ma­ry end­point (tech­ni­cal­ly called A-aDO2), as well as a walk­ing test called 6MWT, he­mo­glo­bin lev­els and the time be­fore res­cue surgery “trend­ed” to­ward sig­nif­i­cance.

Bruce Trap­nell

Mol­gradex was “ef­fec­tive as shown by changes in lung pathol­o­gy, phys­i­ol­o­gy, health sta­tus, func­tion,” lead in­ves­ti­ga­tor Bruce Trap­nell wrote.

That still leaves a lot of ques­tions about the ben­e­fit for pa­tients, and though in­vestors came back with the break­through des­ig­na­tion, they didn’t re­turn Savara to its pre-June sta­tus. The stock near­ly tripled on Mon­day, from $1.73 per share to $4.91. But that’s still less than half of the $10.57 price on June 12.

The news may al­so bol­ster con­fi­dence in Savara’s oth­er pro­grams. Those in­clude a Phase II tri­al ap­ply­ing Mol­gradex to non­tu­ber­cu­lous my­cobac­te­ria, with re­sults ex­pect­ed ear­ly this year, and a now-en­rolling study ap­ply­ing the drug to non­tu­ber­cu­lous my­cobac­te­ria in cys­tic fi­bro­sis pa­tients. There’s al­so a van­comycin in­halant for MR­SA in­fec­tions in CF pa­tients, with Phase III re­sults ex­pect­ed in about a year.

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.