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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

Endpoints Premium

Premium subscription required

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

Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Vas Narasimhan, Novartis CEO (Gian Ehrenzeller/Keystone via AP)

No­var­tis pulls the plug on UK-based car­dio­vas­cu­lar study

Novartis is calling off a UK-based trial for Leqvio in the primary prevention of cardiovascular events in patients with high cholesterol, the company confirmed on Wednesday.

The Swiss pharma giant made the decision after “careful evaluation,” a spokesperson told Endpoints News via email. The trial, dubbed ORION-17, was planned in partnership with England’s National Health Service (NHS) and was part of the company’s strategy to establish Leqvio as a standard of care in cardiovascular disease management.

FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Quince Ther­a­peu­tics faces takeover bid from share­hold­er Echo Lake Cap­i­tal

A bid to take over the biotech Quince Therapeutics has been put forward by one of its shareholders.

On Tuesday, Echo Lake Capital sent a letter to Quince’s board of directors putting forth a proposal to acquire all the biotech’s stock for $1.60 per share, which would value a takeover at around $58 million.

In the letter, Echo Lake said that it believes Quince’s stock is severely undervalued and that no drugs are being actively marketed or developed that require cash expenditures. It’s trading below the value of its assets, Echo Lake said.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Rohan Palekar, 89bio CEO

89bio’s PhII da­ta add to quick suc­ces­sion of NASH read­outs as field seeks turn­around

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.