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)

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

GSK has landed a new first from UNICEF — the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

As the ink dries on CSL’s $11.7 billion Vifor buyout, the company posted a dip in profits, due in part to a drop in plasma donations amid the pandemic.

However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.