→ Al­most a year af­ter it was ap­proved for rheuma­toid arthri­tis, Ab­b­Vie’s JAK in­hibitor Rin­voq was shown for the first time to work as a monother­a­py in atopic der­mati­tis. In a Phase III study, two dif­fer­ent dos­es of the drug beat place­bo on all pri­ma­ry and sec­ondary end­points. Of pa­tients in the high dose group, 80% saw at least 75% skin clear­ance ver­sus 16% for place­bo. That num­ber was 70% in the low dose group.

→ Dicer­na has re­ceived a rare pe­di­atric dis­ease des­ig­na­tion from the FDA for its lead drug, ne­dosir­an, be­ing de­vel­oped for pri­ma­ry hy­per­ox­aluria. The des­ig­na­tion makes Dicer­na el­i­gi­ble for a pri­or­i­ty re­view vouch­er they can sell or use for any fu­ture mar­ket­ing ap­pli­ca­tion. Dicer­na is com­pet­ing with their RNAi ri­val and part­ner Al­ny­lam in this in­di­ca­tion. Al­ny­lam al­so re­ceived the pe­di­atric des­ig­na­tion, along with pri­or­i­ty re­view as they await FDA de­ci­sion. Dicer­na has not yet filed.

→ Beef­ing up its nascent cell and gene ther­a­py unit, Sarep­ta Ther­a­peu­tics has en­tered a li­cense pact with Se­lec­ta Bio­sciences that gives it ac­cess to Se­lec­ta’s im­mune tol­er­ance plat­form. Imm­TOR is de­signed to min­i­mize or pre­vent the for­ma­tion of neu­tral­iz­ing an­ti­bod­ies to AAV — the vi­ral vec­tor Sarep­ta de­ploys to de­liv­er its gene ther­a­pies for Duchenne and limb-gir­dle mus­cu­lar dy­s­tro­phies — there­by mak­ing re­dos­ing pos­si­ble.

“As we build our en­dur­ing gene ther­a­py en­gine, we in­tend not on­ly to rapid­ly ad­vance treat­ments for rare, life-end­ing dis­eases, but at the same time, to ad­vance the state of ge­net­ic med­i­cine sci­ence by con­tin­u­al­ly im­prov­ing the util­i­ty of gene ther­a­py,” Sarep­ta CEO Doug In­gram said in a state­ment.

Two weeks after joining Verastem Oncology as chief medical officer, Frank Neumann is leaving the company for another job.

Neumann had joined Verastem after leaving bluebird bio, which surprisingly split into two companies last week, one in oncology and one in rare diseases. It’s not yet clear to where Neumann is headed next, but he noted in a statement that Verastem’s data and strategy were “truly exciting.”

Finally freed from the restraints of a partial FDA clinical hold on its lead HER2-positive solid tumor candidate, Pieris Pharmaceuticals is now racing toward Phase II.

The FDA slapped a partial hold on Pieris’ PRS-343 back in July, restricting the biotech from enrolling new patients in a Phase I trial. While Pieris was allowed to continue dosing patients who were already enrolled, the agency requested they conduct an additional “in-use and compatibility study” before recruiting any more.

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

A month after filing the IND application for its human extracellular matrix designed to regenerate knee cartilage, Histogen has hit a roadblock.

The FDA on Tuesday verbally notified the San Diego-based biotech that it was placing a clinical hold on the planned Phase I/II clinical trial of HST-003 due to pending CMC information and additional questions needed to complete their review.

Histogen had planned to test the safety and efficacy of implanting hECM within microfracture interstices and related cartilage defects to regenerate that cartilage in conjunction with a microfracture procedure. The company said in a press release that it expects to receive written notice of the clinical hold from the FDA by Feb 12.

Gritstone Oncology has had a big week, and it’s only Wednesday.

On Tuesday, the biotech revealed plans to start clinical testing of an experimental Covid-19 vaccine — in tandem with NIAID — that can also target other coronaviruses, with the goal of preventing future pandemics should SARS-CoV-2 prove difficult to cure with current vaccines. Then, on Wednesday morning, Gritstone licensed lipid nanoparticle technology from Genevant Sciences to develop what it’s calling “self-amplifying RNA vaccines” against Covid-19.