Danuta Jeziorska, Nucleome Therapeutics CEO

Drug dis­cov­ery biotech look­ing in­to the ‘dark’ re­gions of the hu­man genome picks up a $40M+ Se­ries A

While the com­bo of ma­chine learn­ing and drug dis­cov­ery has man­aged to rake in a lot of cap­i­tal for com­pa­nies this year, one biotech is look­ing to the dark side of the genome to stand out.

Nu­cle­ome Ther­a­peu­tics, a UK-based com­pa­ny found­ed in 2019 and spun off from Ox­ford Uni­ver­si­ty, is seek­ing to cre­ate pre­ci­sion med­i­cines by de­cod­ing “dark mat­ter” from the hu­man genome to find tar­gets no one else has gone af­ter. The com­pa­ny has now gar­nered a £37.5 mil­lion ($42.4 mil­lion) Se­ries A to ad­vance its dis­ease pro­grams and build out its plat­form.

In an in­ter­view with End­points News, Nu­cle­ome CEO and co-founder Danu­ta Jeziors­ka said when the hu­man genome was se­quenced, sci­en­tists found that around 98% of the DNA that we have did not con­tain genes. While they didn’t ful­ly ap­pre­ci­ate what it was, the sci­en­tif­ic com­mu­ni­ty has come to un­der­stand that with­in this space there is an “in­struc­tion man­u­al of sorts.” The area es­sen­tial­ly does not en­code pro­teins but con­tains a large num­ber of ge­net­ic changes as­so­ci­at­ed with dis­ease.

“So, if you think in each cell, you have the same DNA, you have two me­ters of DNA, but then you have hun­dreds of cell types,” she said. “So how the cell knows that from the 22,000 genes that are there, which ones to turn on and which ones to turn off, and al­so in which amounts? And this is the dark genome — it’s like the in­struc­tion man­u­al.”

If this area is af­fect­ed there could be ei­ther not enough or too much pro­tein pro­duced, or an onco­gene can be turned on in the wrong place. Jeziors­ka of­fers the dis­ease al­pha tha­lassemia as an ex­am­ple of the role the dark genome can play when af­fect­ed.

One of the rea­sons that oth­ers have not pur­sued the dark genome, Jeziors­ka said, is that pre­vi­ous tools were not present to un­der­stand its func­tion and were not pre­cise enough, but that us­ing ma­chine learn­ing and the com­pa­ny’s 3D tech­nol­o­gy might get the job done.

The com­pa­ny uti­lizes “3D genome tech­nol­o­gy” in its plat­form along with ma­chine learn­ing to dis­cov­er genes that are linked to dis­eases, with the even­tu­al goal of find­ing drug­gable can­di­dates. This tech­nol­o­gy was in­vent­ed by the found­ing team at Nu­cle­ome and al­lows the team to look at how DNA is fold­ed in the cell from a 3D per­spec­tive and help to un­lock the ge­net­ic vari­ants in the “dark” ar­eas, which will help elu­ci­date their func­tion, Jeziors­ka ex­plained.

By us­ing this tech­nol­o­gy com­bined with ma­chine learn­ing, the com­pa­ny is cur­rent­ly look­ing at tar­gets in the au­toim­mune dis­ease space, how­ev­er, the com­pa­ny does not have a can­di­date pre­pared yet.

So far, the com­pa­ny has raised £5.2 mil­lion ($5.8 mil­lion) in seed fund­ing and cur­rent­ly has a team of around 20. This lat­est in­jec­tion will give the com­pa­ny around three years of run­way to build out its plat­form and get a can­di­date to­geth­er. The com­pa­ny will al­so look to dou­ble its head­count and will look to build part­ner­ships with larg­er phar­ma com­pa­nies.

The round was led by M Ven­tures, the VC arm of Mer­ck KGaA. John­son and John­son In­no­va­tion, Pfiz­er Ven­tures, British Pa­tient Cap­i­tal and Ox­ford Sci­ence En­ter­pris­es all par­tic­i­pat­ed in the round.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Tim Pearson, Carrick Therapeutics CEO

Pfiz­er backs $60M in­fu­sion in­to Car­rick, teams up on breast can­cer treat­ment

In a big week for Carrick Therapeutics, the company announced $60 million in funding for its lead breast cancer drug and development of a second program, as well as a collaboration with Pfizer for combo development.

The $35 million from Pfizer comes with an agreement under which Pfizer will support Carrick’s Phase II study of samuraciclib in combination with Pfizer’s Faslodex for advanced breast cancer. Along with the investment, Adam Schayowitz, vice president and development head of breast cancer, colorectal cancer and melanoma at Pfizer global product development, will join Carrick’s scientific advisory board.

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Thomas Gad, Y-mAbs Therapeutics founder and interim CEO

FDA re­jects Y-mAbs’ neu­rob­las­toma drug af­ter tak­ing is­sue with clin­i­cal tri­al de­sign

Uncertainty about clinical trial evidence has led the FDA to hand down a complete response letter for Y-mAbs’ neuroblastoma drug, casting a cloud on the future of a candidate that had gone through a long development journey in a rare pediatric cancer.

Y-mAbs said it’s disappointed “but not surprised” given that the agency’s oncology drug advisory committee had voted 16-0 against its drug’s approval a few weeks ago.

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Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with Rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Philip Tagari switch­es Am­gen's dis­cov­ery lab for in­sitro's ma­chine learn­ing tools; CEO Joaquin Du­a­to to chair J&J's board

In February, Philip Tagari will take a few days of retirement and then immediately return to industry. He won’t be leading the therapeutics discovery unit for a large biopharma, though.

He’ll trade in his Amgen hat for chief scientist at a machine learning startup that has reeled in hundreds of millions in capital to lay the groundwork for a much-hyped new model of drug discovery that aims to speed up the time to new clinical assets.

Raul Rodriguez, Rigel Pharma CEO

Rigel Phar­ma scores FDA ap­proval for leukemia, kick­ing off show­down with Servi­er in IDH1

When Rigel Pharma bought olutasidenib from Forma Therapeutics, it acquired a drug that already secured a PDUFA date at the FDA — for February 2023. But regulators are ready to give their OK sooner than that.

The FDA has approved the IDH1 inhibitor as a treatment for adult patients with relapsed or refractory acute myeloid leukemia who have a susceptible IDH-1 (isocitrate dehydrogenase-1) mutation as detected by an FDA-greenlit test. Rigel will market it as Rezlidhia.

Uğur Şahin, BioNTech CEO (ddp images/Sipa USA/Sipa via AP Images)

BioN­Tech bets on dif­fi­cult STING field via small mol­e­cule pact with a Pol­ish biotech

BioNTech is beefing up its relatively thin small molecule pipeline by adding weight to a clinically difficult corner of oncology R&D: STING agonists. To do so, BioNTech is teaming up with a 15-year-old Polish biotech and doling out €40 million, about $41.5 million, to start.

The deal is broken into two parts: First, BioNTech obtains an exclusive global license to develop and market Ryvu Therapeutics’ STING agonist portfolio as small molecules, whether alone or in combination with other agents.

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