Arthur Kuan, CG Oncology CEO

AACR22: Af­ter Fer­Gene blowup, a start­up touts even bet­ter blad­der can­cer gene ther­a­py re­sults in tiny study

A pri­vate Cal­i­for­nia biotech is rais­ing some eye­brows on the fi­nal day of AACR22 as it re­port­ed in­ter­im da­ta for its ex­per­i­men­tal drug in com­bi­na­tion with Keytru­da.

Look­ing at 18 pa­tients with non-mus­cle-in­va­sive blad­der can­cer who are un­re­spon­sive to bacil­lus Cal­mette-Guérin (an FDA-ap­proved agent for blad­der car­ci­no­mas), CG On­col­o­gy found that 16 of the 18 achieved a com­plete re­sponse af­ter just three months. On top of this, 13 main­tained their re­sponse through six months, nine through nine months and eight af­ter a year — the study’s pri­ma­ry end­point.

That ini­tial three-month mark is good for an 89% CR rate. The two oth­er pa­tients did not re­spond to the ther­a­py at all, drop­ping out at three months and six months, re­spec­tive­ly, ac­cord­ing to a pre­sen­ta­tion from Mof­fitt Can­cer Cen­ter’s Roger Li.

CG On­col­o­gy’s Phase II study, like many ear­ly- and mid-stage tri­als in the on­col­o­gy space, is small and open-la­bel, and no firm con­clu­sions can be drawn just yet. The biotech is plan­ning on en­rolling 35 to­tal pa­tients for the study, and it’s pos­si­ble the re­sponse rate — and com­plete re­sponse rate — di­min­ish over time.

But the 89% fig­ure rep­re­sents a head-turn­ing num­ber nonethe­less, par­tic­u­lar­ly as Keytru­da monother­a­py achieved on­ly a 41% com­plete re­sponse rate in the study Mer­ck used to sup­port FDA ap­proval. The green light came back in Jan­u­ary 2020 for the same NIM­BC pa­tients who are BCG re­sis­tant and who are in­el­i­gi­ble for, or chose not to un­der­go, cys­tec­to­my.

CG On­col­o­gy al­so found no Grade 3 or high­er side ef­fects in the study so far, not­ing most of the ad­verse events in­clud­ed fre­quent or painful uri­na­tion, blood in the urine, blad­der spasms, fa­tigue and chills.

The ex­per­i­men­tal drug in ques­tion is known as CG0070, an on­colyt­ic im­munother­a­py us­ing a mod­i­fied ade­n­ovirus de­signed to de­stroy blad­der tu­mor cells through the de­fec­tive retinoblas­toma path­way, the biotech says. CG0070 con­tains a “GM-CSF trans­gene” that prompts an im­mune re­sponse af­ter the tu­mor cells rup­ture, re­leas­ing anti­gens and GM-CSF to be tar­get­ed.

It’s a sim­i­lar ap­proach to the one tak­en by Fer­Gene — both com­pa­nies used ade­n­ovirus vec­tors, but dif­fer­ent trans­genes to gar­ner the im­mune re­spons­es. CG On­col­o­gy will like­ly want to avoid Fer­Gene’s pit­falls, how­ev­er: Fer­Gene re­ceived a CRL on its drug due to sev­er­al man­u­fac­tur­ing and CMC is­sues, prompt­ing waves of lay­offs and C-suite de­par­tures.

CG On­col­o­gy has re­mained pri­vate since be­ing found­ed in 2010, and its most re­cent fundraise came in De­cem­ber 2020. The biotech put to­geth­er a $47 mil­lion Se­ries D to fund a Phase III NIM­BC monother­a­py study, as well as the Keytru­da com­bo tri­al.

In ad­di­tion to Mer­ck, CG On­col­o­gy has al­so part­nered with Bris­tol My­ers Squibb to pair CG0070 with Op­di­vo for mus­cle-in­va­sive blad­der can­cer and with Roche to at­tack sol­id tu­mors in com­bi­na­tion with Tecen­triq.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Andrew Phillips, Nexo Therapeutics CEO

Scoop: Ver­sant, NEA launch new biotech helmed by ex-CEO of pro­tein de­grad­er C4 Ther­a­peu­tics

Long-time biotech venture firms Versant and New Enterprise Associates are backing a new startup run by former C4 Therapeutics chief executive Andrew Phillips.

The fledgling biotech has raised at least $30 million so far, according to paperwork filed with the SEC this week. The round could balloon to $60 million.

Phillips, who left protein degradation startup C4 in 2020 to be a managing director at Cormorant Asset Management, is running the show of the new venture as president, the SEC filing outlines. He also served as interim CEO of Cormorant-backed and Hansoh Pharmaceutical-partnered Blossom Bioscience last year.

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Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.