Roche nabs front­line OK for Avastin/Tecen­triq in com­mon liv­er can­cer, best­ing an old Bay­er drug

For the first time in 12 years, the FDA has ap­proved a new front­line treat­ment for the most com­mon form of liv­er can­cer.

The agency okayed a com­bi­na­tion of Roche’s an­ti-VEGF an­ti­body Avastin and their im­munother­a­py Tecen­triq for pa­tients with un­re­sectable or metasta­t­ic he­pa­to­cel­lu­lar car­ci­no­ma (HCC). The ap­proval comes two weeks af­ter Roche and their big biotech sub Genen­tech pub­lished Phase III re­sults show­ing the com­bo im­proved both pro­gres­sion-free sur­vival and, cru­cial­ly, helped pa­tients live longer than the long-run­ning stan­dard-of-care, Bay­er’s Nex­avar.

In that study, 84% of the 336 pa­tients tak­ing Avastin and Tecen­triq com­bo were alive at 6 months, com­pared to 67.2% of the 165-pa­tient Nex­avar arm. At 12 months, 72.2% of the Roche pa­tients were alive vs 54.6% for Nex­avar.

Richard Finn

“For the first-time we have a reg­i­men that marked­ly im­proves sur­vival over so­rafenib, the stan­dard of care for first-line he­pa­to­cel­lu­lar car­ci­no­ma since 2007, and of­fers pa­tients the op­por­tu­ni­ty for im­proved dis­ease con­trol with a fa­vor­able tol­er­a­bil­i­ty pro­file,” lead in­ves­ti­ga­tor Richard Finn said in a state­ment, us­ing the chem­i­cal name for Nex­avar.

The ap­proval is a ma­jor boon for Roche’s im­munother­a­py pro­gram, which, while pro­duc­ing a block­buster, has nonethe­less long lagged be­hind the mar­ket-lead­ing drugs from Bris­tol My­ers Squibb and Mer­ck. No­tably, Bris­tol My­ers’ Op­di­vo failed last year to give pa­tients sta­tis­ti­cal­ly sig­nif­i­cant im­proved sur­vival over Nex­avar in the same set­ting, al­though that tri­al was as a monother­a­py and the NJ phar­ma in­di­cat­ed they may try to pur­sue oth­er front­line us­es for the drug in HCC.

The new in­di­ca­tion could al­so make a larg­er im­pact, both in rev­enue and for pa­tients, than last week, when Tecen­triq won ap­proval as a monother­a­py in non-small cell lung can­cer by show­ing it could beat chemother­a­py — years af­ter Mer­ck’s keytru­da had done the same. Roche has been oc­cu­py­ing a sim­i­lar po­si­tion in triple-neg­a­tive breast can­cer, af­ter it be­came the first im­munother­a­py ap­proved for that in­di­ca­tion last year.

For Roche, though, the big ques­tions now cen­ter on whether they can show that their an­ti-TIG­IT mol­e­cule tiragolum­ab — the first of the sec­ond-gen­er­a­tion check­point in­hibitor and an area where Roche is ahead of the pack — im­proves on ex­ist­ing ther­a­pies. So far, the da­ta have been mixed.

Pho­to: Richard Finn (Glob­al On­col­o­gy Acad­e­my)

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Luke Miels, GSK chief commercial officer

GSK picks up Scynex­is' FDA-ap­proved an­ti­fun­gal drug for $90M up­front

GSK is dishing out $90 million cash to add an antifungal drug to its commercial portfolio, in a deal spotlighting the pharma giant’s growing focus on infectious diseases.

The upfront will lock in an exclusive license to Scynexis’ Brexafemme, which was approved in 2021 to treat a yeast infection known as vulvovaginal candidiasis, except in China and certain other countries where Scynexis already out-licensed the drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

See­los Ther­a­peu­tics 'tem­porar­i­ly' stops study in rare neu­ro dis­or­der for busi­ness rea­sons

Microcap biotech Seelos Therapeutics is halting enrollment of its study in spinocerebellar ataxia type 3 (also known as Machado-Joseph disease) because of “financial considerations,” and in order to focus on other studies, the company said today, adding that the pause would be temporary.

The study will continue with the patients who have already enrolled, and the data from them will be used to decide whether to continue enrolling others in the future.

Alec­tor cuts 11% of work­force as it dou­bles down on late-stage neu­ro pro­grams part­nered with GSK, Ab­b­Vie

A month after revealing plans to concentrate on its late-stage immuno-neurology pipeline, Alector is trimming its headcount by 11%.

The layoffs will impact around 30 employees across the organization, the company disclosed in an SEC filing, adding that the plan will “better align the company’s resources” with the new strategy. With $712.9 million in cash, cash equivalents and investments as of the end of 2022, Alector believes the reserves will now get it through 2025.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

FDA ap­proves Nar­can opi­oid over­dose re­ver­sal spray for over-the-counter sale

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.

J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,900+ biopharma pros reading Endpoints daily — and it's free.

No longer ‘dead or just hi­ber­nat­ing,’ drug­mak­ers re­turn to heart med­i­cines

In 2015, now-FDA Commissioner Robert Califf joined industry, academic and regulatory representatives in Washington to discuss why more drugs weren’t in development for cardiovascular diseases, the leading US cause of death and once a mainstay of pharmaceutical industry blockbusters.

The group pointed to many reasons. Clinical trials could take years and testing was expensive. Wide availability of generic drugs made the commercial prospects uncertain. Their paper title summed up the mood: “Cardiovascular Drug Development: Is it Dead or Just Hibernating?”

Endpoints Premium

Premium subscription required

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