Mi­a­mi biotech Longeveron has sold it­self to in­vestors on the promise of us­ing cell ther­a­py to im­prove the lives of ag­ing pa­tients un­able to walk for long dis­tances, but new da­ta don’t look promis­ing at all.

Longeveron’s lome­cel-B, a cell ther­a­py for ag­ing frailty, flopped the key six-minute walk test end­point of a Phase 2b tri­al of the drug, the biotech said Fri­day, and in­vestors weren’t pleased.

At 180 days, pa­tients re­ceiv­ing the drug post­ed no sig­nif­i­cant im­prove­ment on a walk test com­pared to place­bo, Longeveron said, “af­ter ad­just­ing for mul­ti­ple com­par­isons us­ing the Hochberg method” to de­ter­mine false dis­cov­ery. Lome­cel-B is an off-the-shelf prod­uct us­ing “med­i­c­i­nal sig­nal­ing cells” de­rived from healthy donors’ bone mar­row.

But all hope is not lost in Longeveron’s telling: At that same 180-day mark, the drug post­ed a sta­tis­ti­cal­ly sig­nif­i­cant dose-re­sponse curve com­pared with place­bo — a pos­i­tive sign, it said. Mean­while, Longeveron said pa­tients did post a sig­nif­i­cant im­prove­ment on the walk test at day 270 us­ing what it called a pre-spec­i­fied analy­sis, but was not part of the pri­ma­ry end­point.

What those sup­port­ing fig­ures could mean in the long term or whether they have any ef­fect on clin­i­cal out­comes is un­clear, but Longeveron said it would re­view the da­ta with an in­de­pen­dent steer­ing com­mit­tee and con­sid­er the best path for­ward for the pro­gram.

Mean­while, Longeveron tout­ed safe­ty da­ta from the study but those da­ta weren’t avail­able in the topline re­lease.

Shares of $LGVN were trad­ing down around 25% late Fri­day af­ter­noon.

Ed­i­tor’s Note: This sto­ry has been up­dat­ed.

Kunwoo Lee was a graduate student at UC-Berkeley when gene editing pioneer Jennifer Doudna — who happened to work in the same building where he studied — published a paper on CRISPR/Cas9. So he did what any aspiring bioengineer would do: He ran to her lab, and grabbed a postdoc there.

“We started really thinking about the future coming (for) gene therapy and gene editing,” he said.

Lee’s research with Doudna led him to co-found a small San Francisco-based biotech called GenEdit in 2016, the same year he graduated. After five quiet years, the team is now unveiling a $26 million Series A round with support from some big names like Eli Lilly to fund their work on one of the most pressing challenges in gene therapy: what Lee calls the “delivery problem.”

When Thomas Braun was starting out as a young professor at Germany’s University of Würzburg in 1997, he decided to try his hand at a new field: heart regeneration, a sci-fi-esque premise that could offer a way to treat patients recovering from a heart attack. He thought it would take a few years before they got results.

“We were,” he acknowledges now, “rather naïve.”

But on Thursday, after two and a half decades of fitful starts and abandoned leads, Braun and a team of researchers at the Max Planck Institute showed that they could reprogram heart cells in mice and get the animals to regenerate cardiac tissue after a heart attack. The breakthrough, published in Science, adds new evidence that it will eventually be possible to help patients recover muscle lost in heart attacks and gives another boon to anti-aging researchers who want to one day apply these rejuvenation techniques across much of the body.

In an attempt to get its house of accelerated approvals in order, the FDA is holding its second adcomm of 2021 to review cancer drugs that won accelerated approvals but failed to confirm clinical benefit in subsequent trials or have taken a long time to read those data out.

On Dec. 2, the FDA’s Oncologic Drugs Advisory Committee will review two accelerated approvals from Secura Bio’s Farydak (panobinostat), a third-line multiple myeloma drug, and Acrotech Biopharma’s Marqibo, as a third-line drug for adult patients with Philadelphia chromosome negative acute lymphoblastic leukemia. Both drugs have been marketed for more than five years under their accelerated approvals but have recorded negligible sales in their respective indications in recent years.