Vista­Gen Ther­a­peu­tics’ AV-101 has flunked an ex­plorato­ry Phase II, quash­ing hopes that the drug could work as a monother­a­py for treat­ment-re­sis­tant de­pres­sion.

To be sure, these are tough to treat pa­tients: On av­er­age the 19 pa­tients en­rolled in the study had tried 7.8 dif­fer­ent an­ti­de­pres­sants over their life­time to treat their TRD, which has last­ed an av­er­age of 8.6 years.

Shawn Singh

Af­ter wan­ing these pa­tients off their an­ti­de­pres­sants, in­ves­ti­ga­tors ad­min­is­tered ei­ther AV-101 or place­bo for 14 days, then fol­low­ing a wash out pe­ri­od swapped the drug and place­bo arms. At the end, the drug did not in­duce a sig­nif­i­cant change from base­line in the Hamil­ton De­pres­sion Rat­ing Scale — the pri­ma­ry end­point.

Nev­er­the­less, Vista­Gen $VSTN said the tri­al (spon­sored by and con­duct­ed at the Na­tion­al In­sti­tute of Men­tal Health) con­firmed the safe­ty pro­file of their drug, which they are con­tin­u­ing to test. Topline re­sults from the oth­er study are ex­pect­ed lat­er this year.

“In con­trast to the NIMH study, our on­go­ing EL­E­VATE study is de­signed to eval­u­ate AV-101 as a nov­el ad­junc­tive ther­a­py in a sig­nif­i­cant­ly dif­fer­ent pop­u­la­tion of MDD pa­tients, name­ly those whose cur­rent de­pres­sive episode is less than 2 years,” CEO Shawn Singh said.

In­vestors don’t seem as pa­tient. Vista­Gen’s share price, which was hov­er­ing above $1, plunged 44% Thurs­day fol­low­ing the an­nounce­ment, throw­ing the mi­cro­cap biotech in­to pen­ny­s­tock ter­ri­to­ry.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.