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

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.
Hap­py to share very promis­ing re­sults from CORE-1 tri­al com­bin­ing CG0070 and pem­bro for #BC­Gun­re­spon­sivedis­ease, pre­sent­ed by Dr. Uchio @ #AACR22! 14/16 CR, all 6 with durable re­sponse who reached 12mo! @garys­tein­bergmd @UroDocAsh @tbi­vala1 @pcvblack @Kua­nArthur @cgon­col­o­gy https://t.co/B2zPu­cUhYH
— Roger Li (@Uroger­liMD) April 12, 2022

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.
Nearly every clinical trial, regardless of indication or phase, should be agile—incorporating technology and decentralized approaches—to make drug and therapy development faster, easier and more representative.
For more than 60 years, the traditional clinical trial model has relied on a disparate network of independent investigator sites to find, enroll, and matriculate patients through a study to determine drug efficacy and safety. To participate, each of these sites must establish their own processes, people, and technology to accommodate clinical trials, which creates a significant barrier to entry. As a result, less than three percent1 of all healthcare providers participate as clinical trial investigators.
As a drug formerly abandoned by Gilead finally looks set to complete its tortuous journey toward FDA approval, GlaxoSmithKline is shelling out $1.9 billion to snap it up.
Sierra Oncology gets the credit for salvaging momelotinib — which Gilead first obtained through acquiring YM BioSciences for $510 million, but offloaded in 2018 following late-stage failure — and steering it to a positive Phase III readout earlier this year. Started by the crew that used to run YM, Sierra had paid only $3 million in cash to scoop up the drug, in addition to $192 million in biobucks that Gilead will likely pocket given its success.
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AbbVie president and vice chair Michael Severino is leaving the Chicago-area pharma giant on May 31 to become a CEO-partner at prolific biotech creator Flagship Pioneering in June. He’s the third CEO-partner to be named at the incubator, which is behind the likes of Moderna, in the past six days.
He notified the Humira drugmaker on April 8 of his new career ambitions, according to an AbbVie SEC filing Wednesday morning. He’s departing after three and a half years in the president and vice chair posts. Prior to that, he spent nearly five years as CSO.
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The whole premise of the FDA’s accelerated approval pathway rests on the idea that clinical benefit will be established after the approval, via a confirmatory trial, proving the drug was worth authorizing early on.
In many cases, the confirmatory trial does just that, confirming the benefits that seemed promising at first, while in others — 112 of the 253 drugs authorized under the accelerated pathway have not been confirmed as clinically effective, per this BMJ investigation — still need to fulfill their promise.
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Relative to things like oncology and gene therapy, the field of hearing loss is small in the biotech world. When looking at hearing loss and regenerative medicine together, the space shrinks even further.
Now, the small, Oklahoma City-based Autigen is making its way into the ring — and looking to be the first biotech to finally crack regenerating hair cells in the inner ear.
Autigen announced a collaboration with the Boehringer Ingelheim early Wednesday to go after SNHL, or sensorineural hearing loss. The company nabbed an undisclosed upfront payment from Boehringer, along with an unspecified amount for research support and an unspecified duration of said collaboration.
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A group of 30 bipartisan lawmakers sent letters to 7 naloxone manufacturers, calling on them to apply for over-the-counter status for their opioid overdose antidotes and open up supplies further as the opioid crisis continues in the US with record levels of overdoses and deaths.
Citing a Massachusetts study that found substantially increased access to naloxone reduced opioid overdose mortality rates by 46%, the senators and representatives called on Pfizer, Teva Pharmaceuticals, Adamis Pharmaceuticals, Akorn, Amphastar Pharmaceuticals, Emergent BioSolutions, and Hikma Pharmaceuticals to “submit applications to make naloxone available over the counter without delay.”
A handful of prominent oncology pharmas are teaming up on a next-gen diagnostics initiative, with the ambitious vision of testing for all cancer patients. Bayer, GlaxoSmithKline, Novartis and Roche are leading the Precision Cancer Consortium’s drive to open up biomarker testing on a global scale.
The new group will try to boost biomarker testing in two ways — by increasing awareness about the value of genomic testing and by working to improve genomic biomarker testing in clinical trials, it said in a press release.
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Following reports that McKinsey engaged in abusive and deceptive business practices to stoke the flames of the opioid epidemic, the House Oversight committee on Wednesday released a new report documenting how the consulting company leveraged its work with the FDA to drive new business with opioid manufacturers.
Documents uncovered by the committee also show how opioid juggernaut Purdue Pharma explicitly tasked McKinsey with providing advice on how to influence the FDA’s decisions.
Merck hasn’t sold Zostavax in the US since 2020, but that’s not stopping the lawsuits. Thousands of cases charging Zostavax side effects, efficacy issues and advertising and marketing complaints are still working their way through courts across the country.
Estimates of more than 2,000 cases stand, although Merck has been picking up some wins lately. Its most recent dismissal on Tuesday absolved the drugmaker in a class action case in Ohio charging that Merck’s “aggressive marketing campaign” for Zostavax contained false claims around effectiveness and protection duration. It alleged, for instance, that Merck promoted a 51% effectiveness rate, when it was actually lower for older age groups. The lawsuits claimed Merck violated Ohio consumer sales practice laws.
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The University of Kansas
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