A tough year for mes­enchy­mal stem cell com­pa­nies just got tougher.

Pluris­tem, the NASA-al­lied Is­raeli stem cell biotech, an­nounced Mon­day that a da­ta mon­i­tor­ing com­mit­tee de­ter­mined the Phase III tri­al for a lead pro­gram wouldn’t meet the pri­ma­ry end­point. The com­pa­ny will aban­don the ef­fort, which had fo­cused on pre­vent­ing am­pu­ta­tions in peo­ple with crit­i­cal limb is­chemia.

They blamed a low num­ber of am­pu­ta­tions in the place­bo group, which they ar­gued made it more dif­fi­cult to prove that their stem cells were pro­vid­ing a ben­e­fit.

Pluris­tem’s stock $PSTI was cut near­ly in half on the news, falling from $11.46 to $6.60 pre-mar­ket.

The hit makes Pluris­tem the sec­ond mes­enchy­mal de­vel­op­er to take a beat­ing on the mar­ket this year, af­ter the Aus­tralia-based Mesoblast $MESO saw shares drop off in Au­gust. They had risen in an­tic­i­pa­tion of an FDA de­ci­sion on their graft-ver­sus-host-dis­ease ap­pli­ca­tion, and fell when the agency de­liv­ered a CRL.

Mesoblast re­cov­ered, though, af­ter No­var­tis bought in­to their ef­fort to ap­ply their stem cells in peo­ple with se­vere Covid-19 and oth­er pa­tients with acute res­pi­ra­to­ry dis­tress syn­drome.

Yaky Yanay

Pluris­tem has its own Covid-19 pro­gram — one of over a dozen such ef­forts in the US to use stem cells as a way of mod­u­lat­ing the over­ac­tive im­mune re­sponse in se­vere pa­tients — and CEO Yaky Yanay sought to turn at­ten­tion to it on a call with in­vestors Wednes­day morn­ing.

He not­ed that they were like­ly to com­plete en­roll­ment in the first quar­ter of 2021 and an­nounce da­ta around 60 days lat­er. He al­so point­ed to read­outs next year in mus­cle re­gen­er­a­tion fol­low­ing hip frac­ture and in pa­tients ex­pe­ri­enc­ing in­com­plete hematopoi­et­ic re­cov­ery fol­low­ing hematopoi­et­ic cell trans­plan­ta­tion.

Asked, though, about their work in in­ter­mit­tent clau­di­ca­tion, a con­di­tion that af­fects the ar­ter­ies and can progress to crit­i­cal limb is­chemia, Yanay made clear they were mov­ing on. He said that they would de­vote their ef­forts to in­di­ca­tions where they be­lieve they have the best chance of suc­ceed­ing.

But not every­one agrees that Covid is one of those in­di­ca­tions. Mes­enchy­mal stem cells, short-lived stem cells that can pass through the body safe­ly, have been in the clin­ic for two decades in a range of dis­eases, but re­searchers have strug­gled to show ben­e­fits in large, place­bo-con­trolled stud­ies

“In a way, it’s like giv­ing as­pirin for Covid,” Jeanne Lor­ing, an ear­ly stem cell pi­o­neer and a pro­fes­sor emer­i­tus at the Scripps Re­search Cen­ter for Re­gen­er­a­tive Med­i­cine told End­points News in Sep­tem­ber. “It’s not go­ing to hurt them, but the chances of it help­ing them? It’ll be a mir­a­cle.”

So­cial im­age: Yaky Yanay, Pluris­tem (Twit­ter)

As Congress considers whether to adopt sweeping new legislation to lower prescription drug prices across the board, the Kaiser Family Foundation is out with a new report on Monday showing how a more targeted approach on a subset of drugs might be a more efficient way to save government funds.

“This analysis shows that Medicare Part D and Part B spending is highly concentrated among a relatively small share of covered drugs, mainly those without generic or biosimilar competitors,” wrote Juliette Cubanski, deputy director of the program on Medicare policy at KFF, and Tricia Neuman, SVP of KFF. “Focusing drug price negotiation or reference pricing on a subset of drugs that account for a disproportionate share of spending would be an efficient use of administrative resources, though it would also leave some potential savings on the table.”

After a turbulent couple of years, Ovid is officially doing away with a program it had once championed in the face of heavy analyst skepticism.

Ovid has discontinued development of OV101, or gaboxadol, in Angelman syndrome and will not pursue further clinical trials for Fragile X syndrome, the company announced Monday morning. The news comes after Ovid had previously paused development in Angelman when the compound flunked a Phase III trial in December.

Close to a year-and-a-half after tapping the brakes on one of its preclinical programs to do some added genetic engineering work on their next-gen CAR-T, Anixa $ANIX Therapeutics says the regulatory light is flashing red on their IND.

The San Jose, CA-based biotech — which changed its name from ITUS in 2018 — explained in late 2019 that they were taking a knee for at least a year so that researchers could go back and amp up the expression of follicle stimulating hormone on T cells to improve targeting of the FSH receptor on a specific set of ovarian cells. That required new vector engineering work by their partners at Moffitt Cancer Center.

It’s always a surprise when a court has to step in to tell the FDA that it erred in performing one of its main duties: classifying whether a medical product is drug or a device.

But that’s what the US Court of Appeals for the District of Columbia did on Friday, making clear to the world’s top drug regulator that Genus Medical Technologies’ contrast agent barium sulfate (also known as Vanilla SilQ) should not be considered a drug, as the FDA had said, but a medical device.