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.

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.