News brief­ing: Fre­quen­cy posts ex­tend­ed pos­i­tive re­sults for hear­ing loss pro­gram; Kiniksa earns ODD for GCA can­di­date

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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Dif­fu­sion to hand Nas­daq spot to EIP Phar­ma for PhI­Ib de­men­tia study of ex-Ver­tex drug

One of the more than a dozen bidders for Diffusion Pharmaceuticals’ spot on Nasdaq has prevailed.

Boston biotech EIP Pharma will merge with Diffusion in an all-stock deal, with plans to start a Phase IIb clinical trial in the coming months in a common form of dementia with no approved treatments. The combined company will be renamed CervoMed.

The nine-year-old privately-held EIP is working on a former Vertex drug that it will test in a 160-person Phase IIb in patients with dementia with Lewy bodies, or DLB. The National Institute on Aging is expected to fund that trial with a $21 million grant. With the reverse merger, slated for closing in the middle of this year, EIP will be funded through that readout in the second half of 2024. EIP’s equity and debt holders will own about 77.25% of the combined company.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

CSL CEO Paul McKenzie (L) and CMO Bill Mezzanotte

Q&A: New­ly-mint­ed CSL chief ex­ec­u­tive Paul McKen­zie and chief med­ical of­fi­cer Bill Mez­zan­otte

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 re­ports ro­bust sales led by type 2 di­a­betes and pul­monary drugs, promis­es more to come high­light­ing obe­si­ty

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.”

Monopar puts a stop to its chemo com­pli­ca­tion drug; Ex­i­cure ex­plores fu­ture trans­ac­tions

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.

Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.