Stephen Farr Zo­genix

F-Prime’s Ben Aus­pitz has turned a mod­est in­vest­ment in his new­ly launched biotech Modis Ther­a­peu­tics in­to a quick score on the M&A side. And the deal comes with some lessons on the speed rare dis­ease biotechs can move these days, as well as the val­ue they can cre­ate in short or­der.

Ra­zor fo­cused on rare mi­to­chon­dr­i­al DNA de­ple­tion dis­or­ders, Oak­land, CA-based Zo­genix $ZGNX has come up with a $400 mil­lion deal to bag the com­pa­ny and its fledg­ling pipeline — just 10 months af­ter Aus­pitz put to­geth­er a $30 mil­lion A round. The buy­out terms in­clude $250 mil­lion in cash and stock along with $150 mil­lion for reg­u­la­to­ry ap­provals in the US and Eu­rope.

The deal gives Zo­genix chief Stephen Farr the chance to get this one right from the start. The com­pa­ny was em­bar­rassed by a refuse-to-file no­tice from the FDA last April for their drug to treat Dravet syn­drome. Reg­u­la­tors called out the com­pa­ny for slop­py and in­ad­e­quate work.

Mi­chio Hi­ra­no Co­lum­bia

Aus­pitz gam­bled on re­search by Mi­chio Hi­ra­no of Co­lum­bia Uni­ver­si­ty Irv­ing Med­ical Cen­ter and Ra­mon Martí at Barcelona’s Vall d’He­bron Re­search In­sti­tute. They came up with MT1621, the lead drug for thymi­dine ki­nase 2 de­fi­cien­cy (TK2d). 

The VC is shar­ing the wealth with the rest of the syn­di­cate, which in­cludes Or­biMed, Ac­eras Life Sci­ences and Os­age Uni­ver­si­ty Part­ners.

While they were dis­cussing an­i­mal da­ta for the launch round, the drug has since com­plet­ed a Phase II piv­otal pro­gram in 38 pa­tients. Modis re­ports a strong p val­ue (0.004) in sur­vival, com­par­ing their da­ta to a his­tor­i­cal con­trol group. And Modis is al­so boast­ing about hit­ting key goals for “func­tion­al abil­i­ties, in some cas­es re-ac­quir­ing pre­vi­ous­ly lost mo­tor mile­stones.”

Ra­mon Martí VHIR

In re­cent months the biotech al­so picked up break­through sta­tus at the FDA and PRIME sta­tus at the EMA, en­cour­ag­ing plans for a quick di­a­logue with the FDA.

The ther­a­py is not free of safe­ty pit­falls, though.

Modis ex­ecs say that 2 adult-on­set pa­tients “stopped treat­ment due to asymp­to­matic in­creas­es in amino­trans­feras­es, with­out in­creas­es in biliru­bin, which re­solved up­on dis­con­tin­u­a­tion of treat­ment.”

It’s all in Zo­genix’s hands now.

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.”

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.