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

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.