The first in vi­vo CRISPR/Cas9 gene edit­ing pro­gram has some new dura­bil­i­ty da­ta show­ing sus­tained re­duc­tion of a tox­ic pro­tein in AT­TR amy­loi­do­sis at all four dose lev­els in a small 15-pa­tient study.

In­tel­lia Ther­a­peu­tics pre­sent­ed the much-an­tic­i­pat­ed da­ta for its Re­gen­eron-col­lab­o­rat­ed NT­LA-2001 Fri­day morn­ing, adding to the ini­tial Phase I re­sults it first de­liv­ered al­most a year ago to the day.

The Cam­bridge, MA, biotech’s shares $NT­LA climbed near­ly 6% be­fore the open­ing bell Fri­day.

In­tel­lia is now in talks with the FDA and oth­er reg­u­la­tors about tri­al de­sign for a po­ten­tial piv­otal study, the Jen­nifer Doud­na-found­ed biotech said on an in­vestor call Fri­day morn­ing. The much-tracked sin­gle-dose pro­gram, which ed­its genes di­rect­ly in­side the body, is be­ing in­ves­ti­gat­ed for two dif­fer­ent man­i­fes­ta­tions of AT­TR amy­loi­do­sis — polyneu­ropa­thy, where pa­tients have pe­riph­er­al nerve dam­age, and car­diomy­opa­thy, where pa­tients’ heart mus­cles don’t work well.

All re­sults since last June have been in polyneu­ropa­thy, with Fri­day’s da­ta drop show­ing sus­tained dura­bil­i­ty in the rare con­di­tion in which tox­ic pro­tein ac­cu­mu­lates in a pa­tient’s or­gans.

Back in Feb­ru­ary, In­tel­lia had said that at the high­est dose lev­el (1.0mg/kg), six pa­tients had shown a 93% mean re­duc­tion in the tox­ic pro­tein. In the new da­ta, three of those pa­tients reached the nine-month fol­low-up point and main­tained that lev­el of TTR re­duc­tion.

The mean re­duc­tion for the three pa­tients at the sec­ond-high­est dose (0.7mg/kg) was 86%. For the longest-tracked group, dose lev­el two at 0.3mg/kg, the three pa­tients had a mean re­duc­tion of 89%.

Part II of the study, which is on­go­ing, is test­ing a dose of 80mg, the fixed-dose equiv­a­lent of the high­est dose group in Part I, the com­pa­ny said.

Headaches, in­fu­sion-re­lat­ed re­ac­tions, back pain, rash and nau­sea were the most fre­quent ad­verse events across all four dose lev­els, the biotech said. Eleven of the 15 pa­tients re­port­ed a max­i­mal ad­verse event of grade 1. The most se­ri­ous ad­verse event, which In­tel­lia said was “pos­si­bly re­lat­ed” to its treat­ment, was grade 3 vom­it­ing in a pa­tient with a med­ical his­to­ry of gas­tro­pare­sis.

“Based on the in­ter­im da­ta shared to­day, we be­lieve NT­LA-2001’s po­ten­tial to be a trans­for­ma­tion­al treat­ment for pa­tients with AT­TR amy­loi­do­sis is be­com­ing clear­er. The safe­ty, depth of serum TTR re­duc­tion and dura­bil­i­ty pro­file demon­strat­ed thus far high­lights its po­ten­tial for halt­ing and re­vers­ing the dis­ease af­ter a sin­gle dose,” said CEO and pres­i­dent John Leonard in a state­ment.

The com­pa­ny will com­plete en­roll­ment for both the polyneu­ropa­thy and car­diomy­opa­thy arms of the tri­al lat­er this year and will present the first car­diomy­opa­thy re­sults by year’s end, In­tel­lia said.

In­tel­lia is try­ing to leapfrog Al­ny­lam’s On­pat­tro and re­cent­ly ap­proved Amvut­tra, as well as Io­n­is’ Tegse­di. Those are all RNA-tar­get­ing drugs, but re­quire con­stant dos­ing com­pared to In­tel­lia’s one-time ther­a­py. On­pat­tro and Tegse­di have shown re­duced mu­tant pro­tein ex­pres­sion by about 80%.

Al­ny­lam is pur­su­ing la­bel ex­pan­sions of its On­pat­tro and Amvut­tra in AT­TR with car­diomy­opa­thy, as well.

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room:

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.