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.

When AstraZeneca trumpeted “momentous” and “transformative” results for Tagrisso earlier this year at ASCO, some practitioners threw cold water on the ADAURA fervor. Sure, the disease-free survival data look good, but overall survival is the endpoint that matters when it comes to choosing adjuvant therapy for non-small cell lung cancer patients, the experts said.

The OS data still aren’t here, but AstraZeneca is back at ESMO to bolster their case with a look at brain metastasis data.

Late in August, as negotiations on a pricing deal with President Trump reached a boiling point, PhRMA president Stephen Ubl sent an email update to the 34 biopharma chiefs that sit on his board. He wrote that if the industry did not agree to pay for a $100 “cash card” sent to seniors before November, White House chief of staff Mark Meadows was going to tell the news media Big Pharma was refusing to “share the savings” with the elderly — and that all of the blame for failed drug pricing negotiations would lie squarely on the industry.