Less than a month af­ter pub­lish­ing pos­i­tive long-term da­ta for an ex­per­i­men­tal gene ther­a­py, Or­chard Ther­a­peu­tics has qui­et­ly ter­mi­nat­ed its li­cens­ing agree­ment over the pro­gram.

In an 8-K fil­ing to the SEC last Fri­day, Or­chard re­port­ed that it is end­ing the li­cense re­lat­ed to its OTL-101 can­di­date, in de­vel­op­ment to treat adeno­sine deam­i­nase se­vere com­bined im­mun­od­e­fi­cien­cy or ADA-SCID, known col­lo­qui­al­ly as “bub­ble boy syn­drome.” It’s a rare ge­net­ic dis­ease where pa­tients have al­most no im­mune sys­tem what­so­ev­er.

The move comes af­ter Or­chard pub­lished long-term re­sults in the New Eng­land Jour­nal of Med­i­cine show­ing all 50 pa­tients who re­ceived the treat­ment were still alive af­ter at least two years. Event-free sur­vival was al­so high, with 97% of US pa­tients and 95% of UK par­tic­i­pants not need­ing to re­turn to pri­or ther­a­pies.

Or­chard had been de­vel­op­ing the ther­a­py af­ter sign­ing the agree­ment with UCLA and Uni­ver­si­ty Col­lege Lon­don back in Feb­ru­ary 2016. The biotech has an­oth­er ther­a­py for ADA-SCID known as Strimvelis, ap­proved in Eu­rope in 2016, but halt­ed dos­ing af­ter a pa­tient treat­ed un­der com­pas­sion­ate use be­gan see­ing treat­ment for leukemia.

The idea be­hind OTL-101 is to use a lentivirus with hematopoi­et­ic stem cells, ad­min­is­tered ex vi­vo, to in­sert a func­tion­al copy of the ADA gene in­to pa­tients’ cells. Or­chard had been look­ing for an ap­proval for a while, hav­ing pre­vi­ous­ly won break­through ther­a­py des­ig­na­tion for OTL-101 from the FDA.

An FDA green­light had elud­ed Or­chard with the Covid-19 pan­dem­ic forc­ing them to push back plans to sub­mit a rolling ap­proval ap­pli­ca­tion. But last week’s SEC re­port may have been in the works for at least a year.

Back in May 2020, Or­chard an­nounced a new op­er­a­tional strat­e­gy in­volv­ing lay­ing off 25% of its staff and re­struc­tur­ing its pipeline to fo­cus less on rare dis­eases. Part of that re­struc­tur­ing, Or­chard said at the time, was to re­duce in­vest­ment in OTL-101 and an­oth­er pro­gram — steer­ing re­sources in­stead to­ward four oth­er can­di­dates clas­si­fied as “top near-term pri­or­i­ties.”

The pro­grams in­volved are be­ing set up to treat: metachro­mat­ic leukody­s­tro­phy (OTL-200), Wiskott-Aldrich syn­drome (OTL-103), Mu­copolysac­cha­ri­do­sis type I (OTL-203) and Mu­copolysac­cha­ri­do­sis type II­IA, al­so known as San­fil­ip­po syn­drome type A (OTL-201).

Or­chard went pub­lic in 2018, rid­ing a $200 mil­lion IPO af­ter two oth­er nine-fig­ure rounds with a $110 mil­lion Se­ries B and $150 mil­lion Se­ries B. The biotech worked with Glax­o­SmithK­line, too, ac­quir­ing the Big Phar­ma’s rare dis­ease unit — in­clud­ing Strimvelis — in ex­change for rough­ly 20% eq­ui­ty, al­so in 2018. The lack of an FDA-ap­proved prod­uct at this stage, how­ev­er, has left an­a­lysts with ques­tions.

So­cial im­age: Bob­by Gas­par, Or­chard Ther­a­peu­tics CEO

As the Biden administration tries to wrangle the details of its infrastructure bill, Senate Finance Committee Chair Ron Wyden (D-OR) took a concrete step forward on drug pricing reforms on Tuesday and unveiled five principles for such reforms, including providing Medicare with the ability to negotiate prices.

“Allowing the Secretary of HHS to negotiate the price Medicare will pay creates a much needed mechanism to achieve fairer prices when the market has failed to do so,” Wyden wrote.

Endpoints News turned five years old over the weekend. I wanted to mark the happy occasion by extending our deepest gratitude to Endpoints’ premium subscribers while outlining several other ways to support us as we go broader and get bigger this year and beyond.

Same as any business, we’ve got to create value and get paid for delivering it. So if you depend on Endpoints to stay abreast on biopharma developments, we depend on you too.

More than a year ago, Roche and Eli Lilly were forced to contend with a Phase II/III failure of their respective Alzheimer’s drugs. But while Eli Lilly essentially threw in the towels, Roche wasn’t ready to give up yet.

The Swiss drugmaker now has some biomarker data to spotlight as investigators continue monitoring patients in an open-label extension study.

Dubbed DIAN-TU, the study had been designed to see whether Roche’s gantenerumab and Lilly’s solanezumab could spur a cognitive benefit for a group of patients who had a rare, inherited form of Alzheimer’s that’d tied to early-onset. In short, they didn’t: Both failed the primary endpoint.