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.

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.