Fre­quen­cy Ther­a­peu­tics pre­sent­ed re­sults from a longer-term dura­bil­i­ty study of its ac­quired sen­sorineur­al hear­ing loss can­di­date, say­ing FX-322 showed sig­nif­i­cant im­prove­ment for some pa­tients in key mea­sures of hear­ing that were sus­tained for up to 21 months.

The da­ta build up­on Phase I/II re­sults from 2019 where the pro­gram demon­strat­ed sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ments in word recog­ni­tion scores in pa­tients with chron­ic SNHL, Fre­quen­cy said. That study last­ed for on­ly 90 days, where­as Tues­day’s new da­ta were part of sub­se­quent test­ing of a sub­set of these pa­tients be­tween 13 and 21 months af­ter ini­tial dos­ing.

Ac­quired SNHL typ­i­cal­ly re­sults from dam­age to the hair cells of the in­ner ear. These au­di­to­ry sen­so­ry hair cells may be lost due to chron­ic noise ex­po­sure, ag­ing, cer­tain vi­ral in­fec­tions or ex­po­sure to drugs that are tox­ic to the ear. FX-322 is still be­ing stud­ied in a Phase IIa tri­al to de­ter­mine ap­pro­pri­ate dos­ing lev­els and con­firm the ear­li­er re­sults.

Kiniksa nets ODD for gi­ant cell ar­teri­tis pro­gram

The FDA has grant­ed a new or­phan drug des­ig­na­tion to a pro­gram from Kiniksa Phar­ma­ceu­ti­cals.

Tues­day’s win­ner is mavril­i­mum­ab, a mon­o­clon­al an­ti­body in­hibitor be­ing stud­ied to treat gi­ant cell ar­teri­tis, an in­flam­ma­to­ry dis­ease of medi­um-to-large ar­ter­ies. The com­pound tar­gets gran­u­lo­cyte macrophage colony stim­u­lat­ing fac­tor re­cep­tor al­pha, or GM-CS­FRα.

Chron­ic GCA in the head area can typ­i­cal­ly re­sult in re­cur­rent headaches and jaw cramp­ing, as well as con­sti­tu­tion­al symp­toms of fever and fa­tigue. The con­di­tion can al­so re­sult in per­ma­nent vi­sion loss from di­min­ished blood flow to the eye. The large ves­sel form of GCA af­fects the branch­es of the aor­ta sup­ply­ing blood to the rest of the body.

Mavril­i­mum­ab is cur­rent­ly in Phase II world­wide stud­ies in GCA and pneu­mo­nia re­lat­ed to Covid-19.

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

The biotech Monopar Therapeutics is putting a kibosh on a study for its drug to prevent severe oral mucositis (SOM) in patients who are going through chemoradiotherapy for oropharyngeal cancer.

The interim analysis for Monopar’s Phase IIb/III had around 50% of the planned patients enrolled. An independent data safety monitoring board let Monopar know that the trial did not meet the threshold for efficacy. The trial was looking for a 15% of absolute difference in SOM prevention between its drug, Validive, and placebo. Monopar will now discontinue the study as well as the development of Validive.