Mer­ck KGaA, Pfiz­er's Baven­cio set to seal blad­der can­cer ap­proval af­ter con­fir­ma­to­ry tri­al scores

Baven­cio could use the win.

More than two years af­ter its mak­ers — Ger­many’s Mer­ck KGaA and Pfiz­er — se­cured FDA ap­proval for a form of blad­der can­cer, the com­pa­nies un­veiled that the con­fir­ma­to­ry study test­ing the PD-L1 in­hibitor had per­formed as ex­pect­ed, help­ing pa­tients live longer as a main­te­nance treat­ment ver­sus stan­dard-of-care.

The drug was grant­ed ac­cel­er­at­ed ap­proval on the ba­sis of tu­mor re­sponse and du­ra­tion of re­sponse in May 2017 in pa­tients with lo­cal­ly ad­vanced or metasta­t­ic urothe­lial car­ci­no­ma (UC) whose dis­ease has pro­gressed dur­ing or af­ter plat­inum-con­tain­ing chemother­a­py. On Mon­day, the com­pa­nies said that da­ta from the con­fir­ma­to­ry JAVELIN Blad­der 100 tri­al showed pa­tients treat­ed with Baven­cio lived sig­nif­i­cant­ly longer than those who re­ceived stan­dard care.

A sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in over­all sur­vival was demon­strat­ed in the Baven­cio arm in each of the co-pri­ma­ry pop­u­la­tions: all ran­dom­ized pa­tients and pa­tients with PD-L1–pos­i­tive tu­mors, the com­pa­ny added, with­out dis­clos­ing the num­bers. The da­ta will be hand­ed over to the FDA.

Baven­cio was late to the check­point in­hibitor game. Mer­ck’s mar­ket-lead­ing Keytru­da and Bris­tol-My­ers’ pi­o­neer­ing Op­di­vo were the first two PD-1/L1 drugs to make the cut, and have since scored var­i­ous ap­provals across im­muno-on­col­o­gy and now reap in bil­lions in sales. Re­gen­eron, Roche and Chi­na-based drug de­vel­op­ers al­so have their own check­points — PARP in­hibitors are al­so the mix.

Last Sep­tem­ber, Cowen an­a­lysts shaved their 2024 pro­jec­tions for Baven­cio by $170 mil­lion to $480 mil­lion, cit­ing flat growth and rel­a­tive­ly few cat­a­lysts ahead. In com­par­i­son, Op­di­vo is ex­pect­ed to gen­er­ate a meaty $12.4 bil­lion that year, and king Keytru­da a hefty $20.6 bil­lion, the an­a­lysts es­ti­mat­ed.

Baven­cio, for which Pfiz­er paid $850 mil­lion up­front in 2014 to part­ner with Mer­ck KGaA on, has had some se­ri­ous set­backs in its quest to make a mark. The part­ners were forced to ad­mit de­feat in ovar­i­an can­cer in March last year af­ter three straight tri­al fail­ures. Months lat­er in No­vem­ber, the drug al­so fiz­zled as a first-line main­te­nance ther­a­py for gas­tric can­cer pa­tients, years af­ter fal­ter­ing in a gas­tric can­cer study to im­prove over­all sur­vival. In 2018, Baven­cio al­so botched a key study in the lu­cra­tive field of lung can­cer.

The com­pa­nies, how­ev­er, did have some luck in kid­ney can­cer. Baven­cio was grant­ed ap­proval in May 2019 in com­bi­na­tion with Pfiz­er’s ty­ro­sine ki­nase in­hibitor In­ly­ta for use as a front­line ther­a­py for re­nal cell car­ci­no­ma, af­ter late-stage da­ta showed the com­bi­na­tion demon­strat­ed a sig­nif­i­cant pro­gres­sion-free sur­vival ben­e­fit. It is al­so ap­proved for use in Merkel cell car­ci­no­ma.

UC ac­counts for about 90% of all blad­der can­cer cas­es. When blad­der can­cer is metasta­t­ic, the five-year sur­vival rate is 5%, ac­cord­ing to Amer­i­can Can­cer So­ci­ety es­ti­mates.

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.

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.

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.

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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.”

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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.

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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.”

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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.

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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.