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.

Digital therapeutics company Better Therapeutics announced on Thursday that it’s cutting 35% of its staff as it awaits FDA clearance for its first product.

The company, which launched eight years ago, is one of a growing group of companies seeking a digital alternative to traditional medicine. The space saw a record $7.5 billion in investments in 2021, according to Chris Dokomajilar at DealForma, with uses spanning ADHD, PTSD and other indications. However, private insurers have been slow to hop on board.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.