A cou­ple years af­ter li­cens­ing Novim­mune’s ema­palum­ab and turn­ing around a quick FDA OK, Stock­holm-based So­bi is bet­ting up to $1.2 bil­lion for rights to an­oth­er rare dis­ease drug.

The com­pa­ny is shelling out $250 mil­lion up­front and adding up to $915 mil­lion in mile­stones for rights to de­vel­op and com­mer­cial­ize Apel­lis Phar­ma­ceu­ti­cals’ drug pegc­eta­coplan out­side the US. To­geth­er, the com­pa­nies will see the sys­temic C3 ther­a­py through five reg­is­tra­tional tri­als in hema­tol­ogy, nephrol­o­gy and neu­rol­o­gy.

The tri­als, to run in par­al­lel, in­clude two new reg­is­tra­tional pro­grams in cold ag­glu­tinin dis­ease (CAD) and hematopoi­et­ic stem cell trans­plan­ta­tion-as­so­ci­at­ed throm­bot­ic mi­croan­giopa­thy (HSCT-TMA), both slat­ed to be­gin next year. The biotechs will al­so push for­ward with ex­ist­ing pro­grams in parox­ys­mal noc­tur­nal he­mo­glo­bin­uria (PNH); im­mune com­plex mem­bra­nopro­lif­er­a­tive glomeru­lonephri­tis (IC-MPGN) and C3 glomeru­lopa­thy (C3G); and ALS.

In ad­di­tion to the up­front and mile­stones, So­bi is sweet­en­ing the deal with $80 mil­lion in re­im­burse­ment pay­ments over a four-year pe­ri­od for R&D. Plus, Apel­lis co-founder and CEO Cedric Fran­cois said there are “dou­ble-dig­it” roy­al­ties on the ta­ble.

“This col­lab­o­ra­tion fur­ther strength­ens our al­ready strong bal­ance sheets, which we be­lieve will ex­tend our cash run­way in­to the sec­ond half of 2022,” Fran­cois said in a con­fer­ence call on Tues­day morn­ing. He added that the com­pa­ny plans to lever­age So­bi’s in­ter­na­tion­al in­fra­struc­ture, and their “strong pres­ence in key glob­al mar­kets.”

Apel­lis $APLS is keep­ing US com­mer­cial­iza­tion rights for all in­di­ca­tions, and world­wide rights for what Fran­cois said is their “largest in­di­ca­tion”: ge­o­graph­ic at­ro­phy (GA). Pegc­eta­coplan is cur­rent­ly in two Phase III tri­als for GA.

Gui­do Oelk­ers

A joint de­vel­op­ment com­mit­tee will over­see progress on the oth­er in­di­ca­tions, Fran­cois said dur­ing the call. PNH is the most ad­vanced of the 5 in­di­ca­tions — Apel­lis has al­ready sub­mit­ted an NDA, and ex­pects to hear about a PDU­FA date by the end of this year.

“Over­all, we be­lieve this deal with SO­BI is net-net a pos­i­tive one for APLS, as it should sup­port the ro­bust com­mer­cial launch of sys­temic pegc­eta­coplan in PNH and be­yond,” Baird an­a­lysts Mad­hu Ku­mar and Robert Paler­mo wrote in a note.

The up­front, they wrote, will like­ly al­low for “rapid ex­pan­sion” in­to CAD and HSCT-TMA, “where re­views sug­gest com­ple­ment ac­ti­va­tion via C3 is a key ac­ti­va­tion node and clin­i­cal case stud­ies sug­gest C5 mAbs like (Alex­ion’s) Soliris might not work where there are al­ter­na­tive path­way mu­ta­tions.”

Fran­cois said Apel­lis chose So­bi, which he called a “glob­al leader in hema­tol­ogy and rare dis­eases,” be­cause the col­lab­o­ra­tion “will be as im­por­tant to them as it will be to us.”

Two years ago, So­bi put down $50 mil­lion up­front and up to $400 mil­lion in mile­stones for the rights to ema­palum­ab — one of its stars af­ter re­struc­tur­ing to fo­cus on hema­tol­ogy and im­munol­o­gy. It lat­er ac­quired the whole pro­gram, with mile­stones adding up to $518 mil­lion. The drug, mar­ket­ed as Gam­i­fant, is ap­proved in the US for he­mo­phago­cyt­ic lym­pho­his­ti­o­cy­to­sis (HLH), and raked in $26.7 mil­lion in the first half of 2020. But in Ju­ly, the EMA re­ject­ed So­bi’s pitch for ap­proval for rea­sons CEO and pres­i­dent Gui­do Oelk­ers didn’t elab­o­rate on.

Re­gard­ing the prospects for pegc­eta­coplan, Oelk­ers said in a state­ment:

Giv­en the cen­tral role of C3 in the com­ple­ment cas­cade, pegc­eta­coplan has the po­ten­tial to be­come the foun­da­tion for a broad­er plat­form in rare dis­eases. With pos­i­tive Phase 3 da­ta in PNH, pegc­eta­coplan can el­e­vate the stan­dard of care for this de­bil­i­tat­ing blood dis­or­der.

Bristol Myers Squibb has a new R&D partner, one to which they’re paying a pretty penny to use their discovery platform.

The pharma company is doling out $55 million upfront to Schrödinger $SDGR to work on up to five small molecules, with the potential for $2.7 billion in milestone payments. Schrödinger’s initial targets include HIF-2 alpha and SOS1/KRAS for a type of kidney cancer and KRAS-driven cancers, respectively.

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.