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.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

News brief­ing: Bausch Health clos­ing in on deal to ac­quire Al­le­gro as­sets; PharmAbcine strikes deal with Sam­sung Bi­o­log­ics to de­vel­op an­ti­body pro­gram

Bausch Health is closing in on a deal that would allow it to buy out all of Allegro Ophthalmics’ eye-related assets — including the rights to lead candidate risuteganib — for $50 million.

The payment would be made in two tranches: $10 million at signing, and $40 million in 2021.

Risuteganib is in clinical development for intermediate dry Age-related Macular Degeneration (AMD). It’s expected to enter two concurrent Phase III trials for that indication in the next year. The drug is also being tested in patients with diabetic macular edema (DME), and last year met the primary endpoint in a Phase II study, with 48% of patients gaining 8 or more letters in visual acuity from baseline at week 28, compared to 7% in the control group at week 12.

UP­DAT­ED: Two wild weeks for Grail end in $8B Il­lu­mi­na buy­out

Grail’s whirlwind two weeks have ended in the wealthy arms of its former founder and benefactors.

Illumina has shelled out $8 billion to reacquire the closely-watched liquid biopsy startup they spun out just 5 years ago and sold off much of its shares just 3 years ago. The deal comes nearly two weeks after the well-heeled startup filed for a potentially massive IPO — one that was disrupted just a week later when Bloomberg reported that Illumina was in talks to buy their former spinout for up to $8 billion.

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Roche vaults to the front of the NL­RP3 clin­i­cal race, pay­ing $448M up­front to bag In­fla­zome

Roche is going all in on NLRP3.

The pharma giant is putting down $448 million (€380 million) upfront to snatch Novartis-backed Inflazome, which makes it a clinical player in the space overnight.

Dublin and Cambridge, UK-based Inflazome is the second NLRP3-focused biotech Roche has acquired in less than two years, and although no numbers were disclosed in the Jecure buyout, this is almost certainly a much larger deal.

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Donald Trump, AP

Covid-19 roundup: Trump sug­gests Pfiz­er vac­cine could be first ap­proved; VBI Vac­cines inks de­vel­op­ment deal with Cana­da

President Donald Trump commented Monday morning that Pfizer’s Covid-19 vaccine candidate could be the first to win approval by regulators.

During an interview on a Fox News’ morning show, the president said Pfizer was doing “very well” when asked which candidate could be approved, according to a Reuters report. He added that J&J could follow up afterward, saying “they’ll probably be a little later.”

Is­raeli biotech rais­es $57M to go where cur­rent BRAF in­hibitors can't, with back­ing from No­var­tis, SR One

For the blockbuster potential of Novartis’ Tafinlar and Pfizer’s Braftovi, all the BRAF inhibitors on the market so far only target V600 mutations — which accounts for roughly 50% of patients.

Israeli biotech Novellus now has $57 million to develop a drug that they say can help the other 50% who have everything else.

The Series C will fund a Phase II trial for PLX-8394, a “paradox breaker” that could block RAF without activating MAPK signaling. In a Phase I trial, a patient with a BRAF fusion saw their tumor go away after taking the drug, allowing Novellus to hit the ground running.

Clay Siegall (Life Science Washington via YouTube)

#ES­MO20: Seat­tle Ge­net­ics eyes 4th ap­proval with new da­ta in a crowd­ed field

Does Seattle Genetics have another approval on its hands?

The last 12 months, not so great for the world, has been great for Seattle Genetics. The company landed two separate FDA approvals, signed a $4.5 billion deal with Merck and watched antibody-drug conjugates — the technology they spent years developing to broad industry skepticism — emerge suddenly as one of the most popular approaches in oncology. And on Monday at ESMO, the company and their partners at Genmab unveiled the data behind the ADC it hopes will provide its next major FDA approval.