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.

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Novartis’ sickle cell drug, approved in 2019 and branded as Adakveo, has failed an ongoing Phase III, according to preliminary results.

The Swiss pharma giant unveiled early data from the ongoing STAND Phase III study on Friday, saying that crizanlizumab showed no statistically significant difference between the drug at two different dose levels compared to placebo in annualized rates of vaso-occlusive crises that lead to a healthcare visit over the first year since being randomized into the trial.

Merck’s blockbuster cancer treatment Keytruda has been handed another indication by the FDA.

The US regulator announced on Thursday that it has approved Keytruda to serve as an adjuvant treatment for non-small cell lung cancer (NSCLC), which is its fifth indication in NSCLC and 34th indication overall.

According to a Merck release, the approval is based on data from a Phase III trial, dubbed Keynote-091, which measured disease-free survival in patients who received chemotherapy following surgery. The data from Merck displayed that Keytruda cut down on the risk of disease recurrence or death by 27% versus placebo.

Israeli biotech Sol-Gel Technologies announced Friday that it got its hands on a rare disease drug candidate from PellePharm for almost $75 million, amid claims that the drug has the potential to reach a $300 million market.

Execs said on a conference call Friday morning that patidegib, a hedgehog signaling pathway blocker, is being investigated to treat Gorlin syndrome, a rare genetic disorder that increases the risk of developing certain kinds of cancer such as basal cell skin cancer and medulloblastoma, a type of brain cancer. The disease affects around one in every 31,000 people, and an estimated 70,000 people worldwide.

J&J and Legend Biotech’s next step in turning their CAR-T therapy Carvykti into a potential megablockbuster has succeeded, the companies said Friday.

Carvykti achieved the primary endpoint — progression-free survival — in an open-label Phase III study testing the treatment in second- to fourth-line multiple myeloma patients. The CARTITUDE-4 trial, for which there aren’t any hard data yet, represents the biggest development for Carvykti’s ability to compete with Bristol Myers Squibb’s Abecma since its approval last February.