Bren­dan Frey set out, when he be­gan the project to pick out a lead pro­gram for Deep Ge­nomics, to prove that the ar­ti­fi­cial in­tel­li­gence sys­tems his lab has de­signed can iden­ti­fy new drug tar­gets and find a win­ning can­di­date much faster than tra­di­tion­al meth­ods. Now that they have ze­roed in on an an­ti­sense oligonu­cleotide ex­on-skip­ping ther­a­py for a sub­type of Wil­son dis­ease — se­lect­ed out of 2,400 ail­ments and 120,000 un­der­ly­ing ge­net­ic mu­ta­tions — as their face case, the Toron­to-based biotech is ready to delve in­to new fron­tiers with their AI tech.

Deep Ge­nomics plans to spend rough­ly half of its new $40 mil­lion on clin­i­cal de­vel­op­ment of DG12P1, whose first-in-hu­man tri­al is slat­ed for 2021; and the oth­er half on strength­en­ing var­i­ous com­po­nents of its ma­chine learn­ing sys­tems, from ro­bot­ics to as­says. Fu­ture Ven­tures, an in­vestor in Tes­la and SpaceX, led the Se­ries B round while Am­pli­tude Ven­tures, Mag­net­ic Ven­tures, Khosla Ven­tures and True Ven­tures joined.

While they took a splice mod­u­lat­ing ap­proach for their first AI-dis­cov­ered com­pound akin to what Bio­gen has with Spin­raza, Deep Ge­nomics is next fo­cused on hap­loin­suf­fi­cien­cies — dis­or­ders where in­creased ex­pres­sion lev­els of one func­tion­al gene would be valu­able. In oth­er words, where­as the pre­vi­ous chal­lenge lied in find­ing the right mu­ta­tion to tin­ker, the new puz­zle is how best to boost a known tar­get.

It’s a rel­a­tive­ly new ap­pli­ca­tion of oligonu­cleotides with no ap­proved drugs, which is al­so be­ing ex­plored at Stoke Ther­a­peu­tics.

“There are many dif­fer­ent mech­a­nisms that could be rel­e­vant, and so when you look at all these dif­fer­ent mech­a­nisms and all these dif­fer­ent re­gions of the gene that you could de­sign the oli­go for, there are tens of thou­sands, or even hun­dreds of thou­sands of pos­si­ble com­pounds,” says Frey, a star re­searcher that in­sil­i­co’s Alex Zha­voronkov con­sid­ers “with­out doubt in top 10 sci­en­tists in this field in the world.”

Frey elab­o­rates:

That could be in the 5’ UTR, it could be in the in­tron, it could be in an ex­on, in­tron­ic se­quences are very large, could be in a 3’ UTR, dif­fer­ent kinds of mech­a­nisms may be in­volved. It could be a mat­ter of al­ter­ing the up­stream open read­ing frame, or it could be a mat­ter of an in­tron or ten­sion bot­tle neck, it could be a mat­ter of chang­ing the polyadeny­la­tion site, it could be a com­pound that al­ters sec­ondary struc­tures.

New tools will be re­quired to test all these paths they can po­ten­tial­ly trav­el down, and Deep Ge­nomics’ team of 40-plus is al­ready per­fect­ing one mod­el de­signed to pre­dict polyadeny­la­tion pat­terns.

“Mul­ti­lin­gual­ism is an im­por­tant core val­ue for us,” he pre­vi­ous­ly said. “Every­one at the com­pa­ny has had AI train­ing, and every­one at the com­pa­ny has done wet lab work. In fact I ac­tu­al­ly se­quenced the ge­nom­ic DNA for the Wil­son tar­get to val­i­date it once we edit­ed the cell line to put the pa­tient mu­ta­tion in­to the cells.”

In ad­di­tion to deep­en­ing the clin­i­cal bench, Frey is al­so re­cruit­ing for the busi­ness de­vel­op­ment unit.

“Our pipeline is over­flow­ing, and so we’re fo­cused on sign­ing some busi­ness deals,” he tells End­points News in the lead­up to the an­nu­al JP Mor­gan con­fab in San Fran­cis­co.

That in­cludes two ad­di­tion­al pro­grams for Wil­son’s dis­ease that, to­geth­er with the lead pro­gram, would ad­dress up to a quar­ter of the 230,000 pa­tients world­wide. There’s one oth­er undis­closed meta­bol­ic can­di­date for which Frey plans to sub­mit an IND this year, some com­pounds for retinopa­thy, and a dozen hap­loin­suf­fi­cient projects in de­vel­op­ment, he says.

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

As the Covid-19 pandemic made conventional trials impossible for some drugmakers, more and more companies moved to decentralize their clinical studies, accelerating business for tech developers like Science 37. Leveraging that boost, the company is on the verge of a SPAC merger, landing unicorn status and its very own stock ticker.