Close to a year-and-a-half af­ter tap­ping the brakes on one of its pre­clin­i­cal pro­grams to do some added ge­net­ic en­gi­neer­ing work on their next-gen CAR-T, Anixa $ANIX Ther­a­peu­tics says the reg­u­la­to­ry light is flash­ing red on their IND.

The San Jose, CA-based biotech — which changed its name from ITUS in 2018 — ex­plained in late 2019 that they were tak­ing a knee for at least a year so that re­searchers could go back and amp up the ex­pres­sion of fol­li­cle stim­u­lat­ing hor­mone on T cells to im­prove tar­get­ing of the FSH re­cep­tor on a spe­cif­ic set of ovar­i­an cells. That re­quired new vec­tor en­gi­neer­ing work by their part­ners at Mof­fitt Can­cer Cen­ter.

Jose Cone­jo-Gar­cia

The work, though, has ev­i­dent­ly spurred ques­tions at the FDA, which slapped a clin­i­cal hold on the pro­gram, the biotech an­nounced Mon­day morn­ing.

Their shares plunged 28% on the news.

Ac­cord­ing to Anixa:

The study un­der the In­ves­ti­ga­tion­al New Drug ap­pli­ca­tion has been placed on clin­i­cal hold pend­ing sub­mis­sion of ad­di­tion­al in­for­ma­tion re­quest­ed by the FDA. With­in the next 30 days, it is ex­pect­ed that the FDA will pro­vide a let­ter to MCC with de­tailed and spe­cif­ic in­for­ma­tion re­quest­ed.  MCC will as­sem­ble and sub­mit in­for­ma­tion ad­dress­ing the re­quest as soon as pos­si­ble there­after. Suc­ces­sive to the sub­mis­sion, the FDA will con­tin­ue its re­view of the IND.

Jose Cone­jo-Gar­cia, the chair of the de­part­ment of im­munol­o­gy at Mof­fitt, is cred­it­ed as the chief in­ven­tor of the CAR-T tech that Anixa is us­ing.

So­cial: Mof­fitt Can­cer Cen­ter web­site

When Biolyse, an Ontario-based manufacturer of sterile injectables, forged a deal with Bolivia last week to manufacture up to 50 million J&J Covid-19 vaccine doses, the agreement kicked off what will prove to be a test case for how difficult the system of compulsory licenses is to navigate.

The first problem: When Biolyse asked J&J, via a March letter, to license its Covid-19 vaccine, manufacture it in Canada and pay 5% royalties on shipments to needy, low-income countries, J&J rejected the offer, refusing to negotiate. J&J also did not respond to a request for comment.

Novartis’ Entresto started the ACC weekend off rough with a trial flop in heart attack patients, slowing the drug’s push into earlier patients. Now, an NIH-sponsored study is casting doubt on Entresto’s use in the most severe heart failure patients, another black mark on the increasingly controversial drug’s record.

Entresto, a combination of sacubitril and valsartan, could not beat out valsartan alone in an outcomes head-to-head for severe heart failure patients with a reduced ejection fraction (HFrEF), according to data presented Monday at the virtual American College of Cardiology meeting.

As Incyte prepares to potentially hit the market with a topical formulation of its cash cow ruxolitinib in atopic dermatitis, the Wilmington, DE-based company is beefing up its data package for another indication: vitiligo.

Incyte released Phase III results from two of its clinical vitiligo programs Monday morning, saying both studies met their primary endpoints of patients achieving at least 75% improvement from baseline in repigmentation of the face. The data will likely lead Incyte to ask for approval in both the US and Europe for those older than 12 before the end of the year.

While Cytokinetics’ heart drug technically met its primary endpoint back in November, it missed a key secondary endpoint — reduction in cardiovascular death — which eventually cost the company two partnerships. Now the team is back with data suggesting the drug works better in sicker patients, and it’s planning a trip to the FDA.

In a post-hoc analysis, which can be a very difficult sale at the FDA, Cytokinetics separated patients from the Phase III GALACTIC-HF study into four quartiles based on ejection fraction, a measurement of how well the left ventricle pumps blood with each heartbeat. Patients in the lower two quartiles — those with an EF of 22% or lower, and between 29% to 32% — saw a 15% and 17% relative risk reduction of heart failure events and cardiovascular death combined, Cytokinetics reported at ACC. No difference was seen in the upper two quartiles.