Brendan Frey, Deep Genomics

SpaceX in­vestor backs Toron­to AI up­start's jour­ney in­to the 'dark re­gion' of ge­net­ic dis­eases

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.

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

Daniel O'Day (AP Images)

Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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