Mer­ck dou­bles down on Sky­hawk's tech to drug RNA, promis­ing $600M per au­toim­mune, meta­bol­ic tar­get

Ear­ly col­lab­o­ra­tions with big-name part­ners lent Sky­hawk Ther­a­peu­tics both the en­dorse­ments and the cap­i­tal to pur­sue a small mol­e­cule plat­form for drug­ging RNA. And they soon want­ed more. When Mer­ck signed on to dis­cov­er can­di­dates against neu­rode­gen­er­a­tion and can­cer last Ju­ly, Bio­gen was just ex­pand­ing the list of neu­ro tar­gets they want to go af­ter.

Now Mer­ck is com­ing back for its own round two, adding au­toim­mune and meta­bol­ic dis­eases to the menu.

Kath­leen Mc­Carthy

As in the orig­i­nal deal, the phar­ma gi­ant isn’t break­ing down the up­front, stick­ing to the $600 mil­lion per pro­gram fig­ure to il­lus­trate their in­ter­est here. Li­cens­es to any IP aris­ing from the col­lab­o­ra­tion goes to Mer­ck, which would take over de­vel­op­ment once it ex­er­cis­es an op­tion.

At the core of Sky­hawk’s plat­form is RNA splic­ing mod­i­fi­ca­tion — a way to coax dys­func­tion­al genes in­to pro­duc­ing func­tion­al pro­teins with­out al­ter­ing them per­ma­nent­ly. Kath­leen Mc­Carthy, its co-founder and CSO, was part of a team that hit on their phe­nom­e­non by re­verse en­gi­neer­ing how ris­diplam, an SMA drug now shep­herd­ed by Roche and PTC Ther­a­peu­tics, worked.

The ap­proach of­fers a han­dle on un­drug­gable tar­gets, said Dean Li, SVP of dis­cov­ery sci­ences and trans­la­tion­al med­i­cine at Mer­ck Re­search Lab­o­ra­to­ries.

Bill Haney

Bill Haney, the an­gel in­vestor and CEO, bet that it could ap­ply broad­ly be­yond rare dis­eases. Mul­ti­ple drug de­vel­op­ers have since chimed in over $300 mil­lion up­front to match the $8 mil­lion seed round he ini­tial­ly put to­geth­er with some un­con­ven­tion­al back­ers.

“Mer­ck has been a won­der­ful part­ner in dis­cov­er­ing nov­el drug can­di­dates for neu­ro­log­i­cal dis­eases and can­cer,” Haney said in a state­ment.

Oth­er biotechs have sprung up in the RNA mod­u­la­tion space, in­clud­ing Ri­bometrix, which counts Mer­ck as an in­vestor, as well as Ar­rakis, where Michael Gilman has stuck to a more mea­sured ap­proach on the BD front.

Daphne Koller, Getty

Bris­tol My­er­s' Richard Har­g­reaves pays $70M to launch a neu­rode­gen­er­a­tion al­liance with a star play­er in the ma­chine learn­ing world

Bristol Myers Squibb is turning to one of the star upstarts in the machine learning world to go back to the drawing board and come up with the disease models needed to find drugs that can work against two of the toughest targets in the neuro world.

Daphne Koller’s well-funded insitro is getting $70 million in cash and near-term milestones to use their machine learning platform to create induced pluripotent stem cell-derived disease models for ALS and frontotemporal dementia.

Ar­cus and As­traZeneca part­ner on a high stakes an­ti-TIG­IT/PD-L1 PhI­II can­cer study, look­ing to im­prove on a stan­dard of care

For AstraZeneca, the PACIFIC trial in Stage III non-small cell lung cancer remains one of the big triumphs for AstraZeneca’s oncology R&D group. It not only made their PD-L1 Imfinzi a franchise player with a solid advance in a large niche of the lung cancer market, the study — which continues to offer data on the long-range efficacy of their drug — also helped salve the vicious sting of the failure of the CTLA-4 combo in the MYSTIC study.

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized Covid-19 patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $812 million.

Q32 Bio grabs $60M to kick off hu­man stud­ies for next-gen com­ple­ment drugs — with some Covid-19 tweaks along the way

For a company that launched in the early months of the pandemic, Q32 Bio had its fair share of run-ins with the new normals under Covid-19.

The original plan, for instance, was to conduct first-in-human studies of the IL-7 receptor antibody it licensed from Bristol Myers Squibb in the Netherlands. But they realized shortly after that while the country was beginning to open up clinical trials, there were additional restrictions on drugs that tampered with immunological mechanisms.

No­var­tis buys a new gene ther­a­py for vi­sion loss, and this is one pre­clin­i­cal ven­ture that did­n't come cheap

Cyrus Mozayeni got excited when he began to explore the academic work of Ehud Isacoff and John G. Flannery at UC Berkeley.

Together, they were engaged in finding a gene therapy approach to pan-genotypic vision restoration in patients with photoreceptor-based blindness, potentially restoring the vision of a broad group of patients. And they did it by using a vector to deliver the genetic sequence for light sensing proteins.

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Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Hal Barron, GSK R&D chief

GSK's Hal Bar­ron ax­es a once-prized drug from J&J, con­tin­u­ing shift away from res­pi­ra­to­ry

Hal Barron’s revamp of the GlaxoSmithKline pipeline continued yesterday, as the British pharma announced they axed an asthma drug they once promised over $200 million to acquire.

Then led by CEO Andrew Witty and R&D chief Patrick Vallance, GSK picked up the drug, known elegantly as GSK3772847, from J&J in 2016, hoping to expand on the beachhead in asthma they had established the year prior with Breo Ellipta. They promised up to $227 million in upfront payments and milestones.

Konstantin Poukalov

Per­cep­tive re­cruits A-list in­vestors to back its in-house Chi­na start­up with a mam­moth $310M raise

It took two years for Perceptive Advisors to conceive and boot up LianBio, its big bet on a new kind of in-licensing model for China, seeding it with enough cash to set up two anchoring deals with MyoKardia and BridgeBio. The result was a startup that was all ready to go, reaping $310 million just a little over two months after official launch.

Homegrown Chinese biotechs — many of them boasting of US ties and execs with overseas credentials — have been raking in mega-venture rounds in 2020, both from influential local backers and overseas VC firms that have been loading up new cash. As with IPOs, the deal flow might be slower but the amounts are often more staggering. LianBio’s latest round, unusually, is branded both a Series A and crossover.

Dan O'Day, Gilead CEO (Getty Images)

Covid-19 roundup: Mod­er­na posts $1.1B in Covid-19 vac­cine de­posits; Gilead CEO sees po­ten­tial need for Vek­lury on a 'sea­son­al ba­sis'

Moderna’s $MRNA executive team is clearly itching to get a near-term EUA for their Covid-19 vaccine.

The mRNA wunderkind posted an update on their pivotal trial for mRNA-1273, with a note that they have $1.1 billion in customer deposits for the vaccine.

Right now Moderna CEO Stéphane Bancel and his team are neck-and-neck with Pfizer/BioNTech in a race to unveil data on what could be the first vaccines for the pandemic. And he is not one to downplay the company’s prospects.

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