Af­ter spend­ing a year look­ing at Voy­ager’s next-gen AAV cap­sids, No­var­tis has picked two of them to be the de­liv­ery vec­tor for its gene ther­a­py pro­grams tar­get­ing two neu­ro­log­ic dis­eases.

The li­cense trig­gers a $25 mil­lion pay­ment — $12.5 mil­lion for each tar­get — and opens up a road to $600 mil­lion in to­tal mile­stones. But it al­so leaves some mon­ey off the ta­ble as No­var­tis, which had the op­tion to li­cense cap­sids against a to­tal of three tar­gets, de­cid­ed not to act on the third.

“What I imag­ine they’ve done is to take some of their trans­genes, com­bine that with our cap­sids, and see how it does in their hands,” Al San­drock told End­points News.  The for­mer Bio­gen R&D chief took up the CEO man­tle at Voy­ager just days af­ter the No­var­tis deal was an­nounced.

Com­ing af­ter Pfiz­er al­so se­lect­ed a cap­sid in a sep­a­rate deal and Neu­ro­crine paid $175 mil­lion up­front to al­ly on the de­vel­op­ment of cer­tain gene ther­a­pies, No­var­tis’ move of­fers a boost to the big turn­around San­drock was brought in to over­see. The biotech, which emerged in 2014 as one of the flashy new gene ther­a­py play­ers out to tack­le big dis­eases, suf­fered a se­ries of set­backs with their clin­i­cal pro­grams and ul­ti­mate­ly de­cid­ed to go back to the draw­ing board in 2021, piv­ot­ing back to dis­cov­ery.

At the last meet­ing that he joined in on, San­drock said, sci­en­tists were in­ter­est­ed in trans­duc­tion ef­fi­cien­cy by brain re­gion — what pro­por­tion of neu­rons or glial cells gets trans­duced de­pend­ing on the cap­sids.

The abil­i­ty to reach the brain, in­clud­ing dif­fi­cult-to-reach re­gions, and reach bet­ter gene ex­pres­sion there com­pared to tra­di­tion­al AAV vec­tors is a key sell­ing point for the li­brary of cap­sids gen­er­at­ed from Voy­ager’s screen­ing plat­form.

The process be­gan with mil­lions of vari­ants of AAV9 and AAV5, and Voy­ager sift­ed it down to about a dozen. San­drock reck­oned that No­var­tis might have eval­u­at­ed half a dozen or 10 cap­sids be­fore de­cid­ing on the cur­rent ones.

It’s un­clear what stage the No­var­tis pro­grams are at cur­rent­ly; a No­var­tis spokesper­son de­clined to pro­vide de­tails.

Allen Nun­nal­ly

Un­der the deal struc­ture, No­var­tis can swap out the cap­sids it’s se­lect­ed for a sub­sti­tute over the next two years, added chief busi­ness of­fi­cer Allen Nun­nal­ly, as Voy­ager comes up with new­er ones.

No­var­tis may al­so add on op­tions to li­cense cap­sids for up to two rare cen­tral ner­vous sys­tem tar­gets for an ad­di­tion­al $18 mil­lion per tar­get — with the ex­act fi­nan­cials down the road ex­act­ly mir­ror­ing the ini­tial deal.

San­drock sees Voy­ager ink­ing more such deals to li­cense out its cap­sids for var­i­ous use. The com­pa­ny will still keep an in­ter­nal pipeline, but it will seek to part­ner out pro­grams for com­mon dis­eases such as Alzheimer’s or Parkin­son’s.

“We’d love it if our cap­sids are the most com­mon­ly used in the en­tire in­dus­try for CNS dis­or­ders,” he said.

Belgian biotech Confo Therapeutics has landed $183 million, plus potential royalties, in a drug-discovery deal with Daiichi Sankyo.

Early Thursday, Confo Therapeutics put out word of the deal that will be focused on small molecule antagonists to go after an undisclosed target that the company says is associated with CNS diseases.

Confo CEO Cedric Ververken told Endpoints News that Daiichi originally reached out to learn about the biotech’s technology. He added that Confo, founded in 2015, will use its platform to drug a GPCR target that Daiichi has struggled with internally.

Otonomy may be shutting down, but the lessons learned there will live on at another biotech working on new treatments for hearing loss.

San Francisco-based Spiral Therapeutics has bought certain assets related to three of Otonomy’s programs, ranging from data, patent rights, and know-how to inventory. That includes data around Otonomy’s twice-failed lead program, OTO-104 (Otividex), a sustained-exposure formulation of dexamethasone.

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”