In a first, Chi­na’s BeiGene scores FDA ‘break­through’ sta­tus for BTK in­hibitor — po­ten­tial ri­val for Im­bru­vi­ca

BeiGene may have made its rep as a PD-1 pi­o­neer in Chi­na, but it’s com­ing to the US with more can­cer drugs in its ar­se­nal.

The biotech $BGNE says its Bru­ton’s ty­ro­sine ki­nase (BTK) in­hibitor, zanubru­ti­nib, now en­joys break­through sta­tus grant­ed by the FDA — specif­i­cal­ly, in the treat­ment of adult pa­tients with man­tle cell lym­phoma who have been treat­ed with at least one pri­or ther­a­py.

It’s the first can­cer ther­a­py from main­land Chi­na to re­ceive this en­dorse­ment, BeiGene pres­i­dent and gen­er­al man­ag­er of Chi­na Xi­aob­ing Wu told Bio­Cen­tu­ry.

At the ASH an­nu­al meet­ing in De­cem­ber, BeiGene read out a Phase II tri­al in man­tle cell lym­phoma, re­port­ing an over­all re­sponse rate of 84%, with 59%, or 50 pa­tients, achiev­ing com­plete re­sponse. Twen­ty-one pa­tients, how­ev­er, dis­con­tin­ued zanubru­ti­nib.

“Zanubru­ti­nib has been de­signed to max­i­mize BTK oc­cu­pan­cy and min­i­mize off-tar­get ef­fects,” chief med­ical of­fi­cer Jane Huang said in a state­ment.

The most ad­vanced tri­al for the drug in­volves pa­tients with Walden­ström macroglob­u­line­mia — a rare and in­cur­able form of non-Hodgkin lym­phoma — and pits zanubru­ti­nib di­rect­ly against J&J and Ab­b­Vie’s block­buster Im­bru­vi­ca (ibru­ti­nib), cur­rent­ly the on­ly FDA-ap­proved BTK in­hibitor for the in­di­ca­tion. BeiGene has se­cured fast track des­ig­na­tion for that use.

Oth­er late-stage stud­ies un­der­way tar­get un­treat­ed chron­ic lym­pho­cyt­ic leukemia (CLL)/small lym­pho­cyt­ic lym­phoma (SLL); re­lapsed/re­frac­to­ry (R/R) fol­lic­u­lar lym­phoma; and R/R CLL/SLL.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Common performs onstage, December 2020 (Getty Images)

Com­mon, Jamie Foxx among celebs stand­ing up for clin­i­cal tri­als in star-stud­ded can­cer group's pan­dem­ic push

Healthcare screenings and clinical trial enrollment were battered by the pandemic. But the well-known celebrity-backed Stand Up To Cancer non-profit, along with pharma and advocacy partners, has been working to reverse that and make up lost ground, by stepping up awareness campaigns.

Twelve campaigns launched in 2020 and another five in 2021 amplify the need for cancer screening and care, especially for underserved communities. While pharma companies have long been donors to the cancer research group, Covid brought new support — and increased awareness efforts.

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

UP­DAT­ED: Am­gen halts some drug dis­counts for safe­ty net hos­pi­tals as SCO­TUS takes on 340B case

Amgen will soon be the 10th biopharma company to pull back on offering drug discounts to contract pharmacies of safety-net hospitals under a federal program. Like its peers, Amgen argues that the growth of these contract pharmacies has ballooned in recent years and needs to be reigned in.

Beginning Jan. 3, 2022, Amgen’s policy will only allow 340B covered hospitals to designate a single pharmacy location, with the exception of federal grantees and contract pharmacies wholly owned by a 340B hospital, or that have common ownership with a health system.

Drug­mak­ers cut prices on av­er­age by more than 60% to get on Chi­na's 2022 NDRL list — re­port

China’s National Reimbursement Drug List (NRDL) is a crystal clear example of the country’s bargaining power in the biotech and pharma market, as more firms have reportedly agreed to cut their prices for 67 new medicines to be included in its national medical insurance coverage starting in January.

Being on the list is lucrative. Essentially, if a biotech or pharma company gets on this list, they’re covered by the biggest insurance network in the country. Given China’s vast population, the Chinese government has significant leverage to decide which medicines can make a profit. While domestic drugmakers are quite willing to play that game, cutting prices significantly in exchange for getting on the list, international companies don’t do it as often.

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.