Less than a year af­ter rais­ing $90 mil­lion in its sec­ond round of fi­nanc­ing, for­mer Sarep­ta chief Ed­ward Kaye is mak­ing the leap to the Nas­daq on Wednes­day with an up­sized of­fer­ing for his pre­clin­i­cal an­ti­sense biotech, Stoke Ther­a­peu­tics.

Adri­an Krain­er Cold Spring Har­bor Labra­to­ries

The com­pa­ny in-li­censed its tech­nol­o­gy: Tar­get­ed Aug­men­ta­tion of Nu­clear Gene Out­put (TAN­GO) from Cold Spring Har­bor Lab­o­ra­to­ry’s Adri­an Krain­er, who is cred­it­ed as the in­ven­tor of Bio­gen’s $BI­IB spinal mus­cu­lar at­ro­phy drug Spin­raza. Krain­er co-found­ed Stoke and serves on its board.

As it preps an ap­pli­ca­tion to test its lead ex­per­i­men­tal ther­a­py next year, Stoke raised gross pro­ceeds of $142 mil­lion by of­fer­ing 7.9 mil­lion shares at $18, above the range of $14 to $16. It had pre­vi­ous­ly filed to of­fer 6.7 mil­lion shares, and plans to list on the Nas­daq un­der the sym­bol “$STOK.”

The holy grail of gene ther­a­pies is to de­vel­op a treat­ment that is cu­ra­tive with a sin­gle dose, but so far, re­search has shown that the lev­el of pro­tein pro­duced as a re­sult of this type of ther­a­peu­tic in­ter­ven­tion can fluc­tu­ate from pa­tient to pa­tient, its dura­bil­i­ty re­mains in ques­tion and is ex­pen­sive to man­u­fac­ture be­cause sci­en­tists must en­gi­neer virus­es to de­liv­er the gene ther­a­py to the pa­tient.

Stoke is de­vel­op­ing an­ti­sense oligonu­cleotide med­i­cines, by tak­ing aim at dis­eases ar­eas that are of­ten not amenable to tra­di­tion­al gene ther­a­py — be­cause the gene size is too big — or to gene edit­ing, as the ther­a­peu­tic needs to be titrat­able. It is bet­ting on sta­bi­liz­ing the pro­tein man­u­fac­tured by treat­ed cells with its TAN­GO tech­nol­o­gy, which is de­signed to iden­ti­fy gene seg­ments that can be tar­get­ed by drugs to di­al up the lev­el of pro­tein pro­duced. The Bed­ford, Mass­a­chu­setts-based com­pa­ny then de­vel­ops RNA drugs to har­ness these tar­gets.

Like GW Phar­ma­ceu­ti­cals’ $GW­PH keen­ly watched and 2018 ap­proved cannabis-de­rived ther­a­peu­tic Epid­i­olex, Stoke is tar­get­ing Dravet syn­drome, a rare and cat­a­stroph­ic form of in­tractable epilep­sy that be­gins in in­fan­cy.

Con­trol­ling the amount of pro­tein pro­duced fol­low­ing ther­a­py is par­tic­u­lar­ly sig­nif­i­cant in au­to­so­mal dom­i­nant dis­eases — such as ge­net­ic caus­es of epilep­sy — in which mu­ta­tions in just one copy of a gene ad­verse­ly im­pact pro­tein pro­duc­tion. An over-pro­duc­tion of pro­tein in such cas­es could be just as sin­is­ter. Stoke’s drug is de­signed to up-reg­u­late pro­duc­tion of a pro­tein miss­ing in pa­tients with Dravet syn­drome by tar­get­ing RNA splic­ing. The an­ti­sense oligonu­cleotide is sus­pend­ed in saline and de­liv­ered via spinal in­jec­tion, and will like­ly be ad­min­is­tered every four months or so.

If the FDA sanc­tions an in-hu­man study, the biotech ex­pects to ini­ti­ate a Phase I/II clin­i­cal tri­al in chil­dren and ado­les­cents in the first half of 2020 and an­tic­i­pates to have clin­i­cal da­ta, in­clud­ing pre­lim­i­nary ef­fi­ca­cy da­ta, in 2021.

As of March 31, Stoke has raised over $130 mil­lion in fund­ing, in­clud­ing in­vest­ments from Ap­ple Tree Part­ners, RTW In­vest­ments, RA Cap­i­tal Man­age­ment, Cor­morant As­set Man­age­ment, Per­cep­tive Ad­vi­sors and funds man­aged by Janus Hen­der­son In­vestors, Red­mile Group, Sphera Funds Man­age­ment and Alexan­dria Ven­ture In­vest­ments.

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.